Excessive Drug Pricing: What It Is, And What It Isn t

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1 Too Little, Too Late For GSK/Janssen s Sirukumab in RA? Data shows early and good separation over placebo in RA, but it will have to work hard to catch up with its more advanced rivals (p3) Forget Teva & Mylan - Big Pharma Owns Generics Teva's acquisition of Allergan's generics division just about moves the needle, but big pharma dominates off-patent business (p18) Scrip scripintelligence.com 24 June 2016 No Orphan Drug Pricing If agreeing on the pricing & reimbursement of new innovative medicines is a tricky business, it s even more so where drugs for rare diseases are concerned (p14) Pharma intelligence informa Excessive Drug Pricing: What It Is, And What It Isn t IAN SCHOFIELD It s difficult to know exactly how to define excessive or unfair pricing where pharmaceutical products are concerned, and many factors have to be taken into account. Even the EU Commission and competition authorities may be reluctant to pursue cases of alleged price gouging, lawyer David Hull told Informa s recent EU Pharma Law Forum in Brussels. Excessive pricing of medicines is never far from the public eye, particularly in times of pressure on healthcare spending, and all the more so since the furore over Martin Shkreli s decision to raise the US price of Turing Pharmaceuticals AG s toxoplasmosis drug Daraprim by more than 5,000% in Shkreli s move was certainly an eyecatching maneuver, and one that most in the pharmaceutical company industry were quick to distance themselves from. There have been many other cases of steep price increases on certain products recently, notably with Valeant Pharmaceuticals International Inc. s Isuprel and Nitropress last year. But what exactly is excessive or unfair pricing, and what are the grounds on which to decide whether a drug should be allowed to have a very high price at launch, or a significant increase in price during its time on the market? The question was examined at Informa s recent EU Pharma Law Forum in Brussels by David Hull of law firm Van Bael & Bellis, who looked at excessive pricing in Europe in terms of EU competition law, particularly the potential abuse of a dominant position. On the one hand, Hull said, high pricing is seen as an evil of monopoly, but high prices also spur innovation and investment, so distinguishing between what are acceptably high prices and what are illegal excessive prices can be a very tricky balance. Looking at how far EU case law and legislation can be of help, Hull noted that according to Article 102 of the Treaty on the Functioning of the European Union, an abuse may consist of directly or indirectly imposing unfair purchase or selling prices for a product. And in a landmark 1978 case involving United Brands, the Court of Justice of the EU said that charging a price that was excessive because it has no reasonable relation to the economic value of the product was abusive. Of course that doesn t say a lot, Hull commented. What is reasonable relation? The court also set out a two-pronged legal test: whether the difference between costs and price is excessive (i.e., whether it leads to excessive profits), and whether the price is unfair either in itself or comparison with competing products. This is actually not that helpful either, Hull said. When looking at the difference between prices and excessive costs, which costs are you talking about? That s very difficult the biggest costs are R&D costs in this sector, and it is difficult to allocate these to a single product. TURN TO PAGE 7 /

2 STOCKWATCH IN THIS ISSUE It s difficult to define excessive or unfair pricing where pharma is concerned 6 Shire is buying Pfizer's monoclonal IgG2 antibody PF because of its promise in latestage trials 10 Pharma, payers and stakeholders need to work more collaboratively when it comes pricing & reimbursement 14 from the editor Pharma is no stranger to the anti-climax. So many drugs progress to late-stage development with the promise of major advances in disease treatment and great returns to their developers, only to crash in Phase III, at regulatory review or even on the market. From bapineuzumab to MAGE-A3 and from evacetrapib to Provenge, the road to pharmaceutical anticlimax is paved with high hopes. In corporate maneuvering also, anti-climax rears its head time and again. How much anticipatory ink has been spilt on mega-mergers Pfizer/AstraZeneca, AbbVie/Shire, Pfizer/Allergan only for the deals in question to evaporate in a puff of legal fees? It falls to me to write this week's letter as we go to press just hours before Britain opens its polling stations to decide whether the UK should remain in the EU. By the time you read this, we will know the outcome. The pharma industry, with its strong presence in the UK, is one that stands to suffer diverse and significant impacts from a Brexit. It's too close to call right now, but a vote to Bremain is one anti-climax that our sector would happily embrace. exclusive online content COVER / Excessive Drug Pricing: What It Is, And What It Isn t 3 Too Little, Too Late For GSK/Janssen s Sirukumab in RA? 4 ADA: Victoza s Broad CV Benefit Leads The Way For Semaglutide 6 Infinity Doesn t Sugarcoat Disappointing Duvelisib Data; Cuts R&D Staff To Preserve Cash 8 Biotrial Says Will Continue To Cooperate As French Probe Manslaughter Angle in Phase I Case 8 GSK s Vaccines Chief Slaoui To Follow Witty Out The Door 9 R&D Bites 10 Shire Buys Pfizer s IBD MAdCAM-1 Candidate For Undisclosed Price 11 Merck Poised To Be First To Market With A PD-1 For First-Line Lung Cancer 14 Orphan Drug Pricing: More Dialogue And Collaboration Needed, Says EURORDIS 16 Policy & Regulation Briefs 17 Better Together? Aegerion And QLT Merge To Solve Problems 18 Expert View: Data Take: Forget Teva and Mylan - Big Pharma Owns Generics 20 Business Bulletin 21 Stockwatch: A Week Of Dashed Expectations 22 Pipeline Watch 23 Appointments Another Sea Change In Myeloma Promised By New Therapies, Combos Darzalex made a splash with impressive Phase III data in relapsed multiple myeloma at the EHA and ASCO meetings, but a range of other drugs are on the way, including a nextgeneration CD38 inhibitor from Sanofi, checkpoint inhibitors and Roche's BCL-2 Venclexta. Italian Drug Maker Menarini Aims To Expand Into US and Japan The corporate licensing director at A. Menarini IFR tells Sten Stovall why and how Italy's largest drug maker plans to expand into the US and Japanese /scripintelligence /scripintelligence /scripintelligence 2 Scrip intelligence 24 June 2016 Informa UK Ltd 2016

3 HEADLINE NEWS Too Little, Too Late For GSK/Janssen s Sirukumab in RA? ALEX SHIMMINGS Full data for the Phase III SIRROUND-D study of GlaxoSmith- Kline PLC/Janssen Pharmaceuticals Inc. s sirukumab show early and good separation over placebo in rheumatoid arthritis, but the third-placed anti-il6 product will have to work hard to catch up with its more advanced rivals, Actemra (tocilizumab) and sarilumab. First filings for GlaxoSmithKline and Janssen s IL-6 inhibitor sirukumab are on track for the third quarter, following the presentation of the full data from the 1,670-patient SIRROUND-D study at the EULAR meeting in London, in which it met its co-primary endpoints of reducing the signs and symptoms of RA and inhibition of radiographic progression in adults with moderately to severely active rheumatoid arthritis. The top-line data were released last December. Sirukumab produced significantly greater inhibition of radiographic progression, or joint destruction, with a mean change from baseline to week 52 in the van der Heijde-Sharp score of 0.50 among 557 patients receiving sirukumab 50 mg every four weeks and 0.46 for 557 patients receiving sirukumab 100 mg every two weeks compared with 3.69 among the placebo group (n=556) (both p<0.001). Significant inhibition of radiographic progression was demonstrated in both patients naïve to biologic therapy and those previously treated with biologics, and was seen as early as week 24. The percentage of patients achieving ACR20 at week 16 was 54.8% and 53.5% of patients receiving 50 mg and 100 mg doses of sirukumab, respectively, compared with 26.4% for placebo (both p < 0.001). The lead investigator, Professor Tsutomu Takeuchi, of the Keio University School of Medicine, Tokyo, Japan said there was a beautiful separation between both doses of sirukumab and placebo, and that the ACR20 responses with both doses occurred early and were sustained throughout the 52-week treatment period compared with placebo. However, when compared with its more advanced rivals, the picture is not so rosy. When asked how the efficacy data for sirukumab compared to that of Roche s Actemra (tocilizumab) and Sanofi/Regeneron Pharmaceuticals Inc. s sarilumab, Takeuchi noted that the ACR20 response was lower for both doses of sirukumab (and placebo) compared with the data seen with the other IL-6 inhibitors. Sirukumab s less favorable/slightly inferior efficacy data to date and anticipated late market entry will restrict its patient share, should it be approved and launched, despite Janssen s experience in the autoimmune field, say analysts at Datamonitor Healthcare. Actemra has been approved for RA in the US since 2010, while sarilumab is currently awaiting approval there. At least the safety profile is reassuring, fitting well with that seen with the other anti-il6 biologics. The most common (at least 8%) adverse events were elevated liver enzymes, upper respiratory tract infection, injection site erythema and nasopharyngitis. Shutterstock: Puwadol Jaturawutthichai Once on the market, based on its clinical performance to date, Datamonitor Healthcare expects sirukumab will be used in late lines of therapy, likely in patients who have also failed treatment with a non-anti-tnf (since Actemra and sarilumab are more efficacious than sirukumab, it will likely be used as a last resort). Another question raised was over the dose used. Takeuchi said he was concerned that the regulators might like to see a lower dose tested, perhaps 25 mg every four weeks, or even 10 mg every four weeks. He was not able to comment on whether the company will conduct further studies to assess the efficacy and safety of a lower dose of sirukumab. All major secondary endpoints were also met for both doses of including change from baseline in the health assessment questionnaire disability index (HAQ-DI), ACR50, percentage of patients with improved disease activity score in 28 joints (DAS28) at week 24, and percentage achieving ACR70 for six consecutive months (major clinical response) by week 52. Other studies in the SIRROUND program include: SIRROUND-T in patients with an inadequate response to anti- TNF agents and other biologic agents. SIRROUND-H study: in patients with an inadequate response or were intolerant to methotrexate (MTX) or for whom MTX was inappropriate. Due to complete in September SIRROUND-M study: a completed study in Japanese patients who had an inadequate response to MTX or sulfasalazine. SIRROUND-LTE study: a long-term extension study for patients completing SIRROUND-D and SIRROUND-T. This study is estimated to complete in With additional reporting by Christina Vasiliou. scripintelligence.com 24 June 2016 Scrip intelligence 3

4 HEADLINE NEWS ADA: Victoza s Broad CV Benefit Leads The Way For Semaglutide LUCIE ELLIS Full cardiovascular (CV) outcomes data for Novo Nordisk AS s leading diabetes therapy, Victoza (liraglutide), show positive effects across a wide range of CV endpoints placing the company in a strong position with the first GLP-1 product and second ever diabetes therapy to show a benefit on CV mortality. The full data from the Liraglutide Effect and Action in Diabetes Evaluation of CV Outcome Results (LEADER) study were presented on June 13 during the American Diabetes Association s annual meeting in New Orleans and the data were simultaneously published in the New England Journal of Medicine. Data from the trial has been eagerly anticipated since Boehringer Ingelheim GMBH and partner Eli Lilly & Co. produced positive CVOT data for their leading diabetes drug Jardiance (empagliflozin) last September which was the first trial to show a heart failure benefit for a diabetes treatment in high-risk patients. While Novo Nordisk s therapy is in a different drug class to Lilly and Boehringer s offering, the two already compete for market space and as such will be using their respective CV data to promote the individual benefits of Victoza and Jardiance. Novo Nordisk AS reported topline data from the LEADER CV outcomes trial in March this year, which left many asking whether the benefit of Victoza is enough to move GLP-1 therapies to the front line for type 2 diabetes treatment. The positive outlook from LEADER also paves the way for Novo Nordisk s follow on GLP-1 product, semaglutide, for which CV data from the Phase III SUSTAIN 6 study will be reported in September at the European Association for the Study of Diabetes (EASD) annual meeting. The injectable drug also is being developed as an oral GLP-1 receptor agonist, so if it is able to meet Victoza s CV benefit, semaglutide is likely to be used much earlier in type 2 diabetes treatment. Steve Bain from Swansea University Medical School, who led the UK arm of Novo Nordisk s study, told Scrip that is what he is most excited to see following the LEADER presentation. The full data for LEADER show that participants taking Victoza experienced a 13% lower risk of time-to-first occurrence of CV death, non-fatal heart attack or non-fatal stroke, compared to those in the placebo group. In addition, LEADER data show a I was applauding EMPA-REG in Stockholm last year because it was a great trial, but I think data from LEADER is even more robust 22% lower risk of CV mortality and a 15% lower risk of all-cause mortality. More surprisingly, patients in the treatment arm had a 22% lower risk of new evidence of advanced diabetic kidney disease. No significant safety issues were reported in the Victoza treatment arm of the study. Patients were assessed for clinical events, compliance with Victoza and other medicines during visits at one, three and six months initially, then every six months for five years. An excess in adjudicated cancers of the pancreatic origin was observed in the Victoza group, but the finding was not significant. Lead investigator of the study, John Buse, Verne S. Caviness Distinguished Professor at the University of North Carolina School of Medicine, said: It is exciting to see such broad-based benefit for patients who took liraglutide, because most prior trials of diabetes medications have not shown such benefits. He said patients should find comfort in the fact that Victoza can safely improve outcomes beyond the core treatment of type 2 diabetes. If Novo Nordisk continues to follow in Boehringer s and Lilly s footsteps, the company could submit the LEADER data to regulatory authorities for a label extension for Victoza. However, it has made no commitment that this will be its next step. Boehringer and Lilly submitted the data from EMPA-REG to US regulatory authorities in January this year as a supplemental new drug application (snda). Novo Nordisk s chief scientific officer Mads Thomsen commented on comparisons between the two CV outcomes studies. He noted that while he likes rival product Jardiance, and was impressed by data from the EMPA-REG study, Lilly and Boehringer s trial saw a greater degree of heterogeneity in its findings than the LEADER study which he described as having very consistent and full-bodied data. They are two very different trials. I was applauding EMPA-REG in Stockholm last year because it was a great trial, but I think data from LEADER is even more robust. In the NEJM paper, co-author Bernard Zinman made a similar observation of the key differences between Victoza and Jardiance. The NEJM paper notes that time to benefit emerged earlier in the EMPA-REG study than in the LEADER trial and the heterogeneity of the direction and magnitude of the effects on components of the composite primary out-come in that trial [EMPA-REG] contrasts with consistency of the effects in the present [LEADER] trial. The paper however highlights that the true reason for positive benefit with Victoza, while other GLP-1 products and several type 2 diabetes therapies with other mechanisms have failed, is still unclear. No obvious single explanation in terms of either the study designs or the included populations is apparent to explain the divergent findings across this body of medical literature, authors of the NEJM paper noted. CLICK Click here for more coverage of the ADA Meeting: 28IH9Gj 4 Scrip intelligence 24 June 2016 Informa UK Ltd 2016

5 INFOGRAPHIC CANCER R & D Products in development 4,422 products in ongoing development for at least one cancer indication 1. The number of new cancer cases worldwide is expected to grow by about 70% over the next two decades 3 but pharma R&D in oncology is hitting unprecedented heights. Recent breakthroughs in immune-oncology have opened up a whole new front of research against the disease, raising hopes for better patient outcomes. Nearly one third of all products currently in development are for a cancer indication ,435 Top 10 2 Products by Sales ($m) 2015 $ 293 Launched* Preregistration Registered 237 Phase III Clinical Trial Phase II Clinical Trial Broken down by most advanced phase of development Phase I Clinical Trial Preclinical 1 2 Avastin $6,944 Herceptin $6, Gleevec $4,658 Velcade $2,901 Most popular therapeutic categories 1 3 Rituxan (oncology) $5,860 8 Alimta $2,493 2,390 1, Targeted Anticancers (non-immunotherapy) Chemotherapy (non-targeted) Immunotherapy Development of immune-oncology drugs has mushroomed and they account for most of the rise in cancer drug candidates in recent years. 4 5 Revlimid $5,801 Neulasta $4, Zytiga $2,231 Sprycel $1,942 Top 10 cancer indications by number of products in development 1 Unspecified cancer Unspecified solid cancer Breast cancer Prostate cancer Brain cancer Colorectal cancer Acute myelogenous leukaemia Non-small cell lung cancer Melanoma Liver cancer ,164 Cancers figure among the leading causes of morbidity and mortality worldwide, with approximately 14 million new cases and 8.2 million cancer-related deaths in New active substance launches for cancer indications have risen steadily this century. 112 New active substances launched since Sources: 1 Citeline's Pharmaprojects interrogated 9 June 2016 (this figure includes launched drugs that are launched still in development for other cancer indications or are still being rolled out onto markets worldwide. *NB These are launched products still in active development for other cancer indications or still being rolledout onto markets worldwide. 2 Source: Datamonitor Healthcare May Source: World Health Organization, February Source: Scrip Research scripintelligence.com 24 June 2016 Scrip intelligence 5

6 HEADLINE NEWS Infinity Doesn t Sugarcoat Disappointing Duvelisib Data; Cuts R&D Staff To Preserve Cash MANDY JACKSON Infinity s hopes for accelerated approval of duvelisib based on the DYNAMO study have been dashed, so as the company plots a new strategy for the PI3K inhibitor with or without its partner AbbVie it has cut discovery research operations, including 46 jobs. Infinity Pharmaceuticals Inc. had high expectations for the Phase II DYNAMO clinical trial testing its PI3K inhibitor duvelisib in indolent non-hodgkin lymphoma (inhl), but the company didn t sugarcoat the results: while the trial s primary endpoint was met, the data were not as good as hoped. The results were so disappointing that Infinity and AbbVie Inc. are discussing potential changes to their partnership and Infinity is rethinking its business strategy, which was dependent on regulatory approvals for duvelisib monotherapy as early as in The company now plans to cut its discovery research organization, resulting in a head count reduction of 21% or 46 jobs a move that President and CEO Adelene Perkins described as a necessary step to preserve financial resources. DYNAMO was a Phase II, single-arm clinical trial testing duvelisib monotherapy in 129 refractory inhl patients whose disease progressed within six months of their last treatment with Roche s Rituxan (rituximab) in combination with chemotherapy or radioimmunotherapy. The overall response rate (ORR) was the primary endpoint, but Infinity and partner AbbVie were looking for an ORR and complete response (CR) rate that exceeded responses observed in patients treated with approved drugs. The ORR was 46% with no CRs only partial responses. ORRs were as high as 68% among the trial s 28 participants with small lymphocytic lymphoma (SLL) and as low as 33% for 18 patients with marginal zone lymphoma; the response rate was 41% among 83 patients with follicular lymphoma. Perkins was brave, frankly, when she said we hoped that treatment with duvelisib as a monotherapy would have provided a larger clinical benefit for patients in DYNA- MO, because her peers at small and midsized biopharma companies often point to the bright side of bad clinical trial results to obscure a drug s failure. Infinity and AbbVie will evaluate the DYNAMO results for positive signals that could support an accelerated US FDA approval, which the companies previously planned to seek based on the pivotal Phase II trial. However, since that seems unlikely now, Infinity s CEO did not lean on any positive subgroup analyses to prop up her company s value when discussing the DYNAMO results. Infinity s stock price fell 69.2% to $1.36 per share on June 14, bringing the company s market cap to $67.3m about onethird of Infinity s cash balance of $193m as of March 31. Investors may have had higher hopes for the DYNAMO study than stock analysts, however, given Wedbush Securities analyst David Nierengarten s June 14 assessment that as expected duvelisib results are not competitive with already available therapies. PRIMARY ENDPOINT MET, BUT COMPETITIVE DATA ARE LACKING The absence of CRs puts duvelisib at a competitive disadvantage to other therapies approved to treat inhl, including the PI3K inhibitor Zydelig (idelalisib) from Gilead Sciences Inc. and the CD20 inhibitors Rituxan and Gazyva (obinutuzumab), both of which were developed by Roche s Genentech Inc. subsidiary. Although the [DYNAMO] primary endpoint was met, these topline results are not strong enough to differentiate duvelisib in an already crowded indication, Sagient Research said in a June 14 Biomedtracker report. Biomedtracker noted that a 65% ORR and 20% CR rate would be necessary to differentiate duvelisib from other PI3K inhibitors, according to a key opinion leader. Zydelig reported a 57% ORR and 6% CR rate in Gilead s Phase III registrational study in refractory inhl patients. While Infinity and AbbVie review their own data to determine whether duvelisib development is worth pursuing as a monotherapy or in combination with other agents, the companies will reassess the terms of their 2014 license agreement, which gave Infinity $275m up front and up to $530m in milestone fees plus royalties. The deal did not include Infinity s rheumatoid arthritis program for duvelisib, which the company shut down four months after announcing the AbbVie agreement based on a Phase II failure. Infinity and AbbVie revealed their cancer-only agreement to co-develop duvelisib two months before AbbVie agreed to pay $21bn for Pharmacyclics Inc., whose primary asset was the BTK inhibitor Imbruvica (ibrutinib), which is approved in four indications, including SLL and chronic lymphocytic leukemia (CLL) another key indication for duvelisib. Equity analysts have questioned whether AbbVie would continue its partnership with Infinity after adding Imbruvica to its portfolio and following FDA approval in April for the big pharma s BCL-2 inhibitor Venclexta (venetoclax) for CLL patients with a 17p deletion genetic mutation. Analysts are saying now that, based on the DYNA- MO data, AbbVie s decision to drop duvelisib from its pipeline is imminent. We note that the high response rates and good durability of response seen from the Phase I/II study boasted best singleagent activity at that time [with a 72% ORR and 33% CR rate] and were the main reason for Infinity to successfully forge the AbbVie collaboration on duvelisib, William Blair analyst Katherine Xu wrote in a June 14 research report, noting later in the analysis that AbbVie is likely to terminate the collaboration. CLICK Read full story at: 6 Scrip intelligence 24 June 2016 Informa UK Ltd 2016

7 HEADLINE NEWS / CONTINUED FROM PAGE 1 First you have to figure out what the R&D costs are, which is not easy, and once you do that you have to figure out, OK, how do I allocate the costs over the time I am selling the product, and also how do you allocate them across countries, to bigger or higher priced markets, or developing markets? But a comparison with competing products is more helpful, he said. Common sense tells you that if you are trying to figure out what excessive means, you must find something to compare it to, you can t just pull it out of the air. But then you have the problem of which products are comparable. Moreover, what of the many R&D projects that fail? What do you do about the cost of that do you allocate it to successful products, and if so which ones? Competition authorities don t like to get into the business of regulating prices TIMING IS KEY The acceptability or otherwise of an excessive price will also depend, Hull continued, on factors such as the type of product involved (e.g., a breakthrough drug for a serious unmet medical need), whether the price in question is a launch price or a price increase, and the competitive environment. In some cases the high price may simply be a result of pricing dynamics working as they should, he said. Looking at the example of the new hepatitis C drugs, Hull noted that in 2014 some members of the European Parliament had raised concerns about the high price of Gilead Sciences Inc. s Sovaldi (sofosbuvir) and its impact on patient access, asking whether Gilead was abusing a dominant position and whether the European Commission intended to intervene. Hull said that generally the competition authorities are reluctant to take action against high prices. Competition authorities don t like to get into the business of regulating prices, and they are particularly concerned that if they step into this sector and start saying what excessive is, they could hurt innovation. In the hepatitis C case, the commission declined to open an investigation. In response to a parliamentary question in late 2014, Competition Commissioner Margrethe Vestager pointed out that the member states are able to take measures to regulate drug prices, and that in any case the market for hepatitis drugs was a rapidly moving therapeutic area, with several new classes of direct-acting antivirals now in advanced stages of development. This would seem to suggest that this is a dynamic market. The commission, Hull said, also noted that the product represents a cure where there wasn t one before and that it was clearly a step up in the treatment of hepatitis C. A subsequent parliamentary question pointed out that Gilead acquired the patent to Sovaldi from another company and so cannot invoke research costs as grounds for the excessive price. Vestager responded to this saying that since the commission s first response, the factual situation surrounding this particular medicine has evolved further. She noted that AbbVie Inc. s Viekira Pak (ombitasvir/paritaprevir/ritonavir) had entered the market to compete with Sovaldi in addition to, for example, Olysio (simeprivir) from Janssen Pharmaceuticals Inc. Furthermore, several member states have concluded or are negotiating pricing and reimbursing agreements with respect to this group of novel hepatitis C medicines. These other entrants did arrive on the market, and now prices are coming down unfortunately for Gilead stock but this is exactly what should be happening, Hull observed. Competitors see the profits Gilead is making, they put a lot of money in, and the price starts going down. Gilead, he said, went out on a limb and took a risk in bringing the product to market, and you don t want to penalize a company when it starts to charge high prices because it is successful. PRICE INCREASES High launch prices are one thing; steep price increases on a product already on the market are another, Hull said, noting two legal cases that are currently under way in Italy and the UK. In the Italian Aspen Pharma case, the competition authority launched an investigation into allegations that Aspen had abused a dominant market position by increasing the prices of its cancer drugs by between 250% and 1,500% because, the authority claimed, the company wanted to limit parallel exports of the drug to cheaper countries. It claimed Aspen threatened to withdraw the marketing authorization if the health authority did not agree to the price increases, Hull said. A hearing is scheduled for July, with a decision expected in September. In the UK case pitting Flynn Pharma Ltd. against Pfizer, the Competition and Markets Authority (CMA) issued a statement of objections in August 2015 to the two companies, alleging excessive pricing for the antiepileptic Epanutin (phenytoin sodium). In 2012, Pfizer had sold the UK marketing rights to Epanutin to Flynn, but continued to manufacture the product and sold it to Flynn at prices between eight and 17 times its historic prices in the UK. Flynn genericized the product and began selling it on to customers in September 2012 at prices that the CMA said were times higher than those historically charged by Pfizer. The result was that in 2013 the NHS spent more than 50m on phenytoin sodium capsules, compared with about 2.3m annually before September The CMA said that its findings on dominance and abuse were provisional and no conclusion can be drawn at this stage that there has, in fact, been any breach of competition law. We will carefully consider any representations from Pfizer and Flynn Pharma before deciding whether the law has been infringed. This case too is still under way. Summing up, Hull said: If you have drastic price increase, you have a lot greater potential for problems. scripintelligence.com 24 June 2016 Scrip intelligence 7

8 HEADLINE NEWS Biotrial Says Will Continue To Cooperate As French Probe Manslaughter Angle in Phase I Case IAN SCHOFIELD French prosecutors are beginning judicial proceedings into possible manslaughter following the Phase I trial tragedy in January this year, although CRO Biotrial says the move is a normal decision in a complex investigation. The Paris Public Prosecutor has opened judicial proceedings against persons unknown for manslaughter and unintentional injury in the case of the French Phase I trial that in January this year led to the death of one volunteer and caused neurological damage in several others. The proceedings, which follow a preliminary inquiry that began on Jan. 15, 2016, are intended to determine whether faults of a criminal nature contributed to the fatality and the injuries, or whether it was a question of scientific chance, the prosecutor said in a statement. Biotrial told Scrip that the launch of the proceedings was a normal decision that was expected as part of a complex investigation. As we have done since the first day of the administrative and judicial investigations, we will continue to cooperate in the utmost transparency with investigators, it declared. The judicial investigation guarantees respect for the adversarial principle and the rights of people, which are highly valued. The study, which involved Portuguese company Bial s experimental FAAH inhibitor BIA , was being conducted by the CRO Biotrial, which is based in Rennes, north-western France. It was suspended on Jan. 11, 2016, after the first volunteer had been hospitalized and later fell into a coma, but not before the remaining five were given a further dose. The prosecutor noted that a number of investigations had already been carried out into the affair, and cited some of the conclusions reached by these inquiries, such as the fact that the mechanism by which the substance caused the adverse effects was unknown, and that endogenous factors specific to each volunteer in the cohort could explain the variability of the effects of the substance on the organism of the six volunteers in the cohort in question. NEW ELEMENT Its statement also noted that the inquiries had shown that the volunteer who died had an occult endocranial vascular pathology well before he took part in the trial, which could explain the fatal outcome in this case. However, it s not clear where this information originated. Biotrial said that this was something new that did not come up in the reports of the other inquiries. The other two investigations into the affair were conducted by the government s social affairs inspectorate (IGAS) and by a temporary specialist scientific committee (CSST) set up by the drug regulatory body ANSM. The IGAS inquiry found that there were questions around the evaluation of the level of risk posed by the product and the latitude given to those running the trial. IGAS final report, published on May 23, blamed Bial and Biotrial over the choice of dosages and the delay in reporting the incidents to the health authorities, and said Biotrial was to blame with regard to its scientific and ethical responsibility for pursuing inquiries into understanding the accident. Biotrial has contested the criticisms in the report, though, saying it retained the right to seek its nullification. GSK s Vaccines Chief Slaoui To Follow Witty Out The Door SUKAINA VIRJI One person definitely not lining up to take over from Sir Andrew Witty when he retires from GlaxoSmithKline PLC in March 2017 is its vaccines chief and former head of R&D Dr. Moncef Slaoui. He is to retire from the company in June Slaoui spearheaded the overhaul of GlaxoSmithKline PLC pharmaceutical R&D and was instrumental in its DPU (discovery performance unit) initiative. Like Andrew Witty, Moncef Slaoui has spent the bulk of his career at GSK in a variety of roles spanning 28 years. Prior to his current role as chairman of vaccines, where he oversaw the integration with the Novartis AG vaccines business, he served as chairman of R&D for eight years. Second in command of vaccines is Luc Debruyne, who holds the position of president of the Vaccines business. Slaoui also oversees GSK s venture capital arm, SR One. When he was head of R&D, Slaoui led a complete overhaul of GSK s R&D activities, deconstructing the discovery organization into 38 focused and accountable DPUs. He is widely seen as transforming GSK s late-stage pipeline, which currently has more than 30 Phase III programs compared with less than 10 in However, he has faced a series of setbacks in recent years, notably the cardiovascular treatment darapladib; cancer vaccine MAGE-A3; and Duchene muscular dystrophy drisapersen. His departure is probably because he is not being considered for the CEO job, believes Andy Smith, Scrip s popular Stockwatch columnist and chief investment officer of Mann Bioinvest. Slaoui will remain a member of GSK s board until 31 March 2017 and from 1 April 2017 until his retirement on 30 June 2017, he will serve as an advisor the company. Slaoui joined GSK in 1988 and the board in Scrip intelligence 24 June 2016 Informa UK Ltd 2016

9 R&D BITES Marathon Looking To Go The Distance In DMD The biotech submitted an NDA to FDA for its DMD drug in hopes of succeeding where other companies have failed. The Duchenne muscular dystrophy community has had quite a ride for the last few months as several of their best hopes for gaining an approved treatment have been dashed by regulatory rejections, but news surfaced June 14 that Marathon Pharmaceuticals LLC is pursuing approval for its own DMD treatment. The Northbrook, Ill.-based biotech announced that it has filed a New Drug Application (NDA) with FDA for its DMD drug deflazacort, a glucocorticoid that has been on the market for several decades as an anti-inflammatory and immunosuppressive treatment. The DMD community believes the steroid could have the same benefits as prednisone in helping muscle weakness in DMD patients, while having fewer side effects like weight gain and behavior issues. Unlike the treatments that have been developed by competitors which act on specific genes, deflazacort would not be intended to modify the disease. Even if the drug is approved in the indication, it could face push back from payers since it is already available for other uses. FDA usually takes up to 60 days to accept a filing before starting the review process. The company has conducted a preclinical and clinical program, testing the drug in approximately 200 DMD patients across seven clinical trials. The company sold off a portfolio of non-core assets including the sedative Amytal (amobarbital sodium for injection), Iprivask (desirudin for injection) for preventing blood clots, Isuprel (isoproterenol HCl injection) for cardiovascular indications, Nitropress (sodium nitroprusside injection) for congestive heart failure, opium tincture for diarrhea, Pepcid (famotidine) tablets for heartburn and Seconal (secobarbital sodium) sedative capsules to Valeant in February 2015 to better concentrate on its pipeline of rare disease products. does intend to continue developing the drug in status epilepticus and pediatric orphan indications. Paratek Antibiotic Meets Non-Inferiority Bar In First Phase III Paratek Pharmaceuticals Inc. has one set of Phase III data for its broad-spectrum antibiotic omadacycline in the treatment of serious skin infections, but it will be about two years before the company can seek US and EU approvals, because it is waiting on results from a second Phase III clinical trial. Boston-based Paratek's omadacycline met the thresholds set by the FDA and the European Medicines Agency (EMA) to show non-inferiority to Pfizer Inc.'s now-generic, narrow-spectrum antibiotic Zyvox (linezolid) in the treatment of acute bacterial skin and skin structure infections (ABSS- SI) in the OASIS trial. However, Boston-based Paratek doesn't expect data from its second Phase III trial, which is testing omadacycline versus moxifloxacin in patients with communityacquired pneumonia, until the third quarter of 2017 with approvals in late 2018 at the earliest. Marinus Nixes Ganaxolone Indication After Trial Fail The biotech vows to continue development of the drug in pediatric indications after a late-stage failure in an adult seizure disorder. Marinus Pharmaceuticals Inc. remains bullish on its lead compound ganaxolone, despite another failure for the drug, but investors are giving up hope. The Radnor, Pa.-based biotech reported topline results from a Phase III study of ganaxolone in adult focal onset seizures on the morning of June 13 that showed the drug failed to meet its primary endpoint of significantly reducing seizures compared with a placebo. The company said during a conference call with analysts that the drug did reduce seizure activity in patients, but that the data was not robust enough to be statistically significant. The median percent reduction of focal onset seizures in the ganaxolone group was 21.28% compared to 10.25% seen in placebo patients during the titration and the 12-week treatment period. Ganaxolone, which is being developed in three formulations including intravenous, capsule and liquid, will no longer advance in this indication, said Marinus. The drug showed a similar safety profile to previous studies with the highest reported adverse events being fatigue, dizziness and tiredness. Yet, there was a higher proportion of patients in the ganaxolone treatment arm that discontinued the study due to adverse events 44 patients (25%) versus 26 patients (14%). The biotech UK's NICE OKs BMS' Opdivo- Yervoy Combo In Advanced Skin Cancer Patients with skin cancer in England and Wales will be the first in Europe to have access to Bristol-Myers Squibb's combo therapy containing the checkpoint inhibitors Opdivo (nivolumab) and Yervoy (ipilimumab), after the National Institute for Health and Care Excellence backed use of the regime in final draft guidance issued June 17. The drugs can stall progression of advanced melanoma by an average of eight months compared with standard treatment, NICE said when issuing its decision, one of the fastest approvals from NICE to date, coming just weeks after the combination was licensed by the European Commission on May 11. scripintelligence.com 24 June 2016 Scrip intelligence 9

10 HEADLINE NEWS Shire Buys Pfizer s IBD MAdCAM-1 Candidate For Undisclosed Price STEN STOVALL Back on the product trail after its mega merger with Baxalta Inc, Shire PLC has bought global rights to Pfizer Inc. s investigational biologic PF for inflammatory bowel disease. Shire PLC said it was buying Pfizer Inc. s PF , a monoclonal IgG2 antibody directed against MAdCAM-1, because of its promise in late-stage trials for treating moderate-to-severe inflammatory bowel disease (IBD). The drug is among several that target white blood cell trafficking in the intestinal mucosa as a way of treating IBD, which includes ulcerative colitis and Crohn s disease - serious, chronic diseases characterized by inflammation of the intestine. Shire must be impressed with the PF s promise given the company s plans to cull some activities in its R&D pipeline after completion this month of the company s combination with Baxalta Inc., which cost some $32bn. Shire CEO Flemming Ornskov told Scrip on June 3 that the combined R&D teams of Shire and Baxalta would meet later this month to start deciding what projects would continue and which would be scrapped, saying, We won t be able to do everything going forward. But Shire gave little insight in the price it paid for PF or its regulatory plans when announcing the pact with Pfizer. Shire s head of clinical development, Howard Mayer, did say that the Dublinbased group look forward to continuing the development of PF , a unique and differentiated biologic that will benefit from our experience in IBD and across the gastrointestinal space. Terms of the deal - which remains contingent of Hart-Scott-Rodino approval - were not disclosed in the group s announcement issued June 14. PF is a fully-human monoclonal antibody designed to directly target a gastrointestinal endothelial adhesion molecule known as mucosal addressin cell adhesion molecule 1 (MAdCAM-1), that binds to the alpha4beta7 integrin on We look forward to continuing the development of PF , a unique and differentiated biologic that will benefit from our experience in IBD and across the gastrointestinal space. Shire s clinical development head Howard Mayer. white blood cells. Most anti-adhesion molecule therapies target the integrin family. Takeda Pharmaceutical Co. Ltd. s currently approved ulcerative colitis and Crohn s disease drug Entyvio (vedolizumab)) and Roche s investigational agent for the condition called etrolizumab both bind to this molecule, as does the multiple sclerosis drug Tysabri (natalizumab). But the experience with Biogen Inc./Elan Corp. PLC s Tysabri which promotes an often fatal brain inflammation known as progressive multifocal leukoencephalopathy (PML), has made gastroenterologists wary of agents sharing any aspect of its mechanism of action. Although no cases of PML have been seen so far with the other antialpha4beta7 agents, the FDA questioned vedolizumab s developer closely about the risk before approving the drug. The Tysabri risk was detected post-market and has been addressed with extensive risk management steps. DRUG TARGETS MADCAM-1 To avoid such concerns, the Pfizer drug that Shire has bought targets a different factor called MAdCAM-1, an adhesion molecule on endothelial cells that interacts with alpha4beta7 and which - unlike Tysabri doesn t influence immune surveillance in the central nervous system and does not bind to VCAM-1, another indirect target of Tysabri that appears to be involved in PML. PF has completed Phase II clinical trials in ulcerative colitis and Crohn s disease, known as TURANDOT and OPERA, respectively. The Pfizer-sponsored TURANDOT Phase II trial met its primary and secondary end points; adult patients with moderate to severe active ulcerative colitis who failed at least one previous treatment who were treated with PF showed an increased rate of remission, response, and mucosal healing at week 12, compared to placebo. The most commonly reported adverse events were consistent with the underlying disease, Shire said in a statement. 10 Scrip intelligence 24 June 2016 Informa UK Ltd 2016

11 HEADLINE NEWS Merck Poised To Be First To Market With A PD-1 For First-Line Lung Cancer JESSICA MERRILL Merck s Keytruda improved progressionfree survival and overall survival in previously untreated non-small cell lung cancer patients in the highly-anticipated KEYNOTE-024 trial, all but guaranteeing a rapid FDA approval in the indication. Positive progression-free survival and overall survival data from Merck & Co. Inc. s high profile KEYNOTE-024 trial testing Keytruda (pembrolizumab) in patients with previously untreated advanced non-small cell lung cancer (NSCLC) positions Keytruda to beat Bristol-Myers Squibb Co. s Opdivo (nivolumab) to a firstline lung cancer indication. The winner long-term in the high stakes market remains very much up in the air, however, as the details of the trial were not released. Bristol s trial in a similar patient population, CheckMate 026, is still ongoing, with data anticipated in the third quarter, and there is still a big question about the optimal threshold for testing tumors for PD-L1 expression. Both KEYNOTE-024 and CheckMate 026 used a different threshold for enrolling patients by PD-L1 expression, which could impact the results. What does seem apparent is that Merck is on a fast track toward having Keytruda approved for first-line lung cancer. In an interview June 15, the day before the data were announced, Head of Global Clinical Development, Merck Research Laboratories Roy Baynes said the company will move as quickly as possible to get the data filed with FDA. Keytruda, like Bristol s Opdivo, is already approved for the second-line treatment of lung cancer and FDA will likely move quickly to approve the drug for first-line use as it has done in other indications for the PD-1 inhibitors. BMO Capital Markets analyst Alex Arfaei predicted an ultra-speedy review within one month after filing. Merck plans to disclose the full data from KEYNOTE-024 at an upcoming medical meeting, expected to be the European Society for Medical Oncology (ESMO) meeting in October. The trial enrolled patients whose tumors expressed high levels of PD-L1 (tumor proportion score of 50% or more) and showed Keytruda was superior to chemotherapy for both the primary endpoint of progression-free survival and the secondary endpoint of overall survival. Demonstrating the overall survival benefit in particular is a positive for Merck at such an early time point. An independent data monitoring committee recommended the trial be halted and that patients receiving chemotherapy be offered Keytruda. BRISTOL AND COMBINATIONS RIGHT ON MERCK S HEELS Merck s lead in the first-line NSCLC market isn t expected to be long-lasting. Bristol s CheckMate 026 trial is on track to read out in the third quarter, pitting the two rivals against each other in a fierce competition for market share in what is believed to be the largest single market for PD-/L1 inhibitors. The timing advantage Merck has over Bristol is not likely very material under the assumption that both sets of results get presented in full at the same meeting, Bernstein analyst Tim Anderson said. Rather, what will matter more is how the two sets of findings compare side by side. Opdivo has thus far dominated in the second-line lung cancer market because it was cleared by FDA without a requirement for PD-L1 testing, unlike Keytruda, leading patients and physicians to choose the more convenient option. Only about 30% of lung cancer patients are being tested for PD-L1 expression, with the majority of those being in the first line, according to Merck, but the firm has played up that Keytruda captures the majority of patients tested. Most of Keytruda s sales have been coming from use in melanoma. The dynamics will be different in the first-line lung cancer market because both drugs are expected to be approved with a requirement for PD-L1 testing. Having the experience in the market with testing could be an advantage for Merck. Merck and Bristol have deployed different strategies in clinical trials when it comes to testing for the biomarker, however. It remains to be seen where the advantage will lie or if there is one at all. KEYNOTE-024 enrolled only the highest expressers of PD-L1, 50% or greater, while CheckMate 026 enrolled patients with a PD-L1 expression of 1% or greater. Although PD-L1 is an imperfect biomarker, high expressers have been shown in clinical studies to generally respond more favorably to treatment with a PD-1 blocker. The design of the trials could yield different outcomes or indications if FDA should choose to limit the use of Keytruda to patients with a PD-L1 expression of 50% or more. That limited marketed represents only about 25-30% of NSCLC patients, according to analysts. But BMO s Arfaei predicted FDA won t restrict the label. Based on the good data from KEYNOTE-001, which in Rx naïve patients with 1-49% PD-L1 expression showed median OS of 19.5 months, and the FDA s unwillingness to restrict the Keytruda 2L+ label to a specific PD-L1 threshold from the single arm KN-001 trial, and the growing body of evidence illustrating the dynamic nature of PD-L1 expression, we believe that Keytruda will likely get an unrestricted 1L label, similar to the wording of its current indication, he said. Labeling for Keytruda currently recommends it for use in PD-L1 positive patients as determined by an FDA-approved test. The clinical studies section reflects data for patients with at least 50% expression of PD-L1, but Merck had submitted a supplemental new drug application (snda) to include data from KEYNOTE-001 with expression levels of just 1% or higher. Merck s Baynes stood by the company s PD-L1 strategy despite the fact the company has given up significant share to Bristol, pointing to the data that has come out of the second-line NSCLC trials. scripintelligence.com 24 June 2016 Scrip intelligence 11

12 HEADLINE NEWS Measuring Physical Activity In Clinical Trials In many clinical trials, improvements in physical activity and mobility are important outcome measures. These are often estimated using in-clinic controlled assessments of exercise capacity such as treadmill tests. Many drug treatments that improve symptoms may also enable increased activity and mobility with corresponding improvements in quality of life. These improvements may be better examined by measuring free-living activity that represents real-life behaviors that have genuine meaning and value to the individual. Accelerometers are key components of the majority of fitness trackers today. Accelerometry enables measurement of tiny gross motor movements and represents a precise, non-invasive, and relatively inexpensive technology that can be used with low patient burden in free-living conditions. It has been used by researchers for over 40 years to study sleep and activity patterns in non-clinical settings. In the area of sleep assessment, actigraphy (the measurement of rest and activity patterns) is already a well-established diagnostic tool reimbursed in many countries, including the US. The first research using this technology to study circadian rhythm and sleep disorder in non-clinical settings was carried out in the 1970s and has led to broad methodology consensus among academics including optimal wear location, wear interval, and accepted derived sleep quality and quantity endpoints. Validation of the technology against gold standard diagnostic tools such a polysomnography has also resulted in the use of actigraphy by sleep laboratories as a diagnostic aid for sleep disorders. Scientific evidence, routine clinical use and globally accepted practice parameters have contributed to the emergence of a standard methodology for the use of actigraphy and have made it easier to incorporate sleep endpoints into clinical trials. Although physical activity monitors have been widely used since 2000 to measure free-living activity in large-scale global community-based studies, the acceptance of standard derived outcomes measures and Sponsored Content ICON s Bill Byrom and Marie McCarthy discuss best practice when choosing, implementing and interpreting data from physical activity monitors in clinical trials. Monitoring free-living activity is more beneficial to the patient than in the clinic. standard methodologies has been less well defined. Many outcome measures have been developed to assess healthy individuals rather than the patient populations routinely seen in clinical trials. Although accelerometers are used to monitor physical activity in today s trials, uncertainty exists as to the methodology to employ. In particular, common questions include: What physical activity outcome measures are going to provide clinically relevant data? Which device will be acceptable to regulatory agencies, appropriate to the patient population and study objectives? How should devices be implemented in a study? Selecting An Appropriate Device Choosing an appropriate actigraphy device depends upon a number of factors. Historically, physical activity monitors were worn on the hip and were less optimal for measurement of sleep parameters; whereas devices designed for sleep studies were worn on the non-dominant wrist, and optimized to generate sleep endpoints. Today, newer generations of devices are likely to be worn on the wrist for 24 hours and incorporate algorithms to generate both sleep and daytime activity endpoints. Patient compliance often depends on patient characteristics such as age, BMI, gender, lifestyle and personal preferences. Elements such as wear location, form factor, and ease of use can influence acceptability for different populations. Study design factors including the duration of the monitoring period, and operational considerations such as data transfer process, data integration and management need to be considered when using physical activity monitors Implementation Standards When you explore the scientific literature, there is a wealth of validation evidence supporting the use of physical activity monitors. However, it is /ICON Plc /ICON plc /ICON plc 12 Scrip intelligence 24 June 2016 Informa UK Ltd 2016

13 HEADLINE NEWS Sponsored Content that study teams sometimes struggle to incorporate actigraphy endpoints into their protocols. Bill Byrom recently worked with David Rowe at Strathclyde University in Glasgow, to derive standards for physical activity monitor use to measure activity in COPD and other less active patient populations by reviewing published studies in COPD and other key scientific work. Their evaluation, featured in Contemporary Clinical Trials (2016) identified that implementation standards varied enormously. For example, the 76 studies included: 27 different activity monitor models, numerous wear locations, required wear intervals ranging from two days to 26 weeks, and minimum required wear time required per day ranging from six to 24 hours. With reference to the nature of activity in this patient population and the wider scientific literature on activity monitoring in less active patient groups, Byrom and Rowe proposed a set of implementation standards for clinical trials in this and similar patient populations (Table 1). In the area of dementia, there is a lack of consensus regarding the role physical activity plays in moderate cognitive impairment, with different scientific reviews presenting opposing arguments on the role physical activity plays in cognitive decline. Marie McCarthy worked with clinical and scientific experts within ICON to identify the promise of actigraphy in Alzheimer s disease clinical trials and review methodology and terminology used in published studies to promote the increased use of actigraphy endpoints in this patient population. McCarthy and colleagues identified that the use of different outcome measures, subjective self-reports and diverse nomenclature makes it difficult to compare studies. An agreed standard selection of outcome measures and physical activity monitoring methodology would help to bring clarity to the area. Data Interpretation And Endpoint Selection In their review of 76 COPD studies, Byrom and Rowe identified that 80 different derived health outcomes measures describing activity and changes in activity were reported by the researchers. Similarly, McCarthy et al identified a variety of outcome measures and terminology used to describe and measure physical activity, exercise, aerobic activity, and sedentary behaviour in Alzheimer s disease trials (featured in JPAD, 2016). This lack of standardization is a potential barrier to drug developers where robust and repeatable methodology and validated outcomes measures are important for acceptance. The two publications we have developed provide a useful starting point for outcome measure selection for different patient populations and clinical objectives. It is clear that, like sleep, physical activity is an important outcome measure that is related to therapeutic efficacy across a broad range of disease indications. There is growing interest in the use of accelerometers to objectively quantify free-living activity in addition to the assessment of sleep in clinical trials. Importantly, free-living activity may be better related to other health outcomes such as quality of life compared to in-clinic functional performance tests. We actively encourage the use of actigraphy in clinical trials where improvements in physical activity and mobility are important to direct or indirect outcome measures, and continue to contribute to increased utilization of this approach through the development of implementation and endpoint selection standards. Table 1 Proposed Implementation standards for COPD clinical trials measuring changes in sedentary behavior and light/ moderate and overall activity Methodology item Activity monitor type Activity Monitor Placement Epoch length Number of days worn Number of hours wear for a valid day Valid interval definition (Number of valid days) Definition of non-wear episode Missing data methodology Proposed approach Preferred: a triaxial accelerometer with access to raw data.accepted: a triaxial accelerometer without access to raw data with sufficient storage capacity for at least 7 days data captured in 15s epochs. Preferred: Thigh Accepted: Waist (if attachment options will not influence devicecompliance and wear time). Record with, or process into, 15s epochs, considered sufficient to measure sedentary behaviour changes in addition to physical activity. 7 days 10 hours or more per waking wear period. 5+ days (no requirement for specific numbers of weekend or week days) 60 minutes of zero counts with up to two 1- minute epochs of up to 100 cpm for ActiGraph, or equivalent for other devices Eliminate non-valid days and participants without sufficient valid days. Source: Contemporary Clinical /ICON Plc /ICON plc /ICON plc scripintelligence.com 24 June 2016 Scrip intelligence 13

14 HEADLINE NEWS Orphan Drug Pricing: More Dialogue And Collaboration Needed, Says EURORDIS IAN SCHOFIELD Pharmaceutical companies, payers and other stakeholders need to work more collaboratively if the problems surrounding the pricing and reimbursement of orphan medicines are to be tackled successfully in Europe, says the rare disease organization EURORDIS. If agreeing on the pricing and reimbursement of new innovative medicines is a tricky business, it s even more so where expensive drugs for rare diseases are concerned. Payers with limited funds may take fright at the high costs of orphan medicines in view of the often limited evidence available at the time of marketing authorization. Rare disease communities in Europe are worried that orphan drugs are increasingly not being reimbursed because of their high costs in relation to their perceived value. An increasing number of cases are being reported across Europe of medicines not being reimbursed following marketing authorisation or even of reimbursement decisions being reversed, says the rare disease organization EURORDIS. One such case of reimbursement reversal was the decision in March this year by the Dutch National Healthcare Institute (ZINL) to no longer pay for Alexion s Soliris (eculizumab), which is used in patients with paroxysmal nocturnal hemoglobinuria (PNH), citing a poor cost-effectiveness ratio. At its recent membership meeting, EU- RORDIS said the fact that drug pricing decisions are taken at national level in the EU leads to unwanted fragmentation and in turn often irrational pricing decisions, and called for more collaboration among national and European authorities and the pharmaceutical industry to streamline the drug pricing process. One way of approaching the issue that is being trialled by the Benelux countries (Belgium, the Netherlands and Luxembourg) is to club together to try to achieve more affordable prices for orphan drugs. The initiative, which began in mid-2015, aims to take a broader, more multi-stakeholder approach involving joint price negotiations with pharmaceutical firms, as well as the exchange of data and sharing of registries. The initiative seems to be gathering pace: the European Commission has just said that Austria is to join the collaboration too, and an announcement to this effect is expected at the June 17 meeting of the Council of the EU. But a March 21 post about the Soliris decision on the online rare disease portal RareConnect, which is run by EURORDIS, noted wryly that despite the emphasis on a wider European approach to orphan reimbursement, the Dutch ZINL clearly missed this message. Jean-Louis Roux, public affairs director at EURORDIS, said that while we are not privy to why the decision was taken on Soliris, there was a good level of certainty that it was taken on purely economic grounds, as the value of the drug itself had not been challenged. Now, most probably to make a point of principle to the company concerned, the payers have decided to stop reimbursing it, Roux told Scrip in an interview. We understand that if new data should come in showing that the product is not delivering the value it says it delivers, such a decision would be in order but hardly so when the value remains unchallenged. LACK OF DIALOGUE One problem, Roux said, was that there is still a continuing lack of genuine dialogue between pharmaceutical companies and payers on how to deal with the orphan question and generate a shared understanding of the value of a product. Instead what we observe boils down to a tug-of-war solely focused on price, and this means that we are not getting anywhere in terms of better health outcomes or a more intelligent way for payers and industry to work together. In order to start a debate on better ways to approach orphan drug pricing, in May 2015 EURORDIS, together with the European Patients Forum (EPF), called for the development of a new platform or table for price negotiation for improving access to medicines while addressing future challenges to the sustainability of healthcare systems. They said this approach could be based on the three core principles of value assessment, volume of patients treated, and the continuous post-approval generation of real-world evidence. Such a platform, they said, might be set up by a core group of member states and later expanded to others. The Benelux initiative seemed to fit that bill. A year on, in May 2016, EURORDIS and the EPF wrote to the authorities in the three countries suggesting that they extend their agreement for joint orphan drug price negotiation to other member states that have since then expressed an interest in such an approach. When we look at what Benelux are doing, there is a lot of promise to break out of national silos and start understanding that, if you seek convergence of efforts, you tend to be stronger, not weaker, Roux said. Noting Austria s decision to join, he stressed that this collaborative initiative was not solely focused on joint procurement of medicines but more constructively on finding ways of increasing access to innovative drugs in an affordable and sustained way and increasing the predictability of uptake for pharmaceutical companies. The aim is not just to squeeze lower prices out of industry but to look also at the scientific aspects and methodologies for assessing value, and to see how the parties can work together more upstream, Roux said. The attention given to price can be misleading. A drug can come at a high price and still be good value for money, but today we tend to get caught in a debate that is broader and affects all of society. Other member states are already working on similar but separate initiatives, for example Romania and Bulgaria, Spain and Portugal, and the Nordic countries, 14 Scrip intelligence 24 June 2016 Informa UK Ltd 2016

15 HEADLINE NEWS although they are not necessarily working with each other. We say the spirit is good but let s avoid falling into another elaborate form of fragmentation, Roux observed. Our hope is that we can unlock these discussions and encourage individual countries to more fully realise the value of broader collaboration at the European level. GETTING THE VALUE RIGHT EURORDIS believes that the failure to recognize the true value of orphans by awarding them reimbursement not only harms patients but also reduces industry s readiness to develop new innovative drugs. A key issue in the pricing and reimbursement debate is that orphan drugs are increasingly being approved with higher levels of uncertainty something that is compounded by the growing complexity of products such as gene and cell therapies and by the fact that some drugs are coming on the market for ultra-rare diseases that may affect only a few dozen people. A case in point is GlaxoSmithKline PLC s Strimvelis, a gene therapy that was given the all-clear by the European Medicines Agency s CHMP in April for for ADA-SCID, which the company said affects only tens of patients. Noting that the development of Strimvelis cost hundreds of millions of dollars, GSK CEO Andrew Witty said that if people want ultra-rare-disease medicines, we re going to have to think creatively about how we manage that. For such products, Roux said, we have to tackle how these drugs work in a far longer time horizon. And there is little visibility as to how the drug is going to affect the patient s health in the medium to long term, e.g., whether it will prove consistently effective in populations that tend to be very heterogeneous (particularly so in rare diseases), whether it might have to be re-administered during the patient s lifetime, etc. These are totally new reflections. And the payers, who are confronted with impossible financing choices to make, obviously do not particularly like this added element of uncertainty. MOCA COORDINATED ACCESS INITIATIVE One answer to these complex conundrums could be to seek earlier and more structured dialogue between stakeholders to help identify and assess the added value of orphan medicines. Ways of doing so are currently being explored via the Mechanism of Coordinated Access to orphan medicines (MoCA), which has been established as part of the Process on Corporate Responsibility in the Field of Pharmaceuticals launched by the European Commission in September Jean-Louis Roux, public affairs director of EURORDIS If you seek convergence of efforts, you tend to be stronger, not weaker MoCA is open to a range of stakeholder groups, including national pricing and reimbursement authorities, patients, marketing authorisation holders and applicants, the pharmaceutical industry in general, and others such as the European Medicines Agency, HTA bodies and experts. A number of pilot projects have been run under MoCA, focusing on matters like company strategy, the target disease, the product and its development, and challenges to market access, with a view to helping speed up access for patients. For companies, the benefits of these pilots are likely to include more predictable uptake of orphan drugs, more effective gathering of real-world outcomes, better predictability of product development, and better understanding of member states expectations regarding the requirements for pricing and reimbursement, according to MoCA s revised Terms of Reference, which were published in January For their part, the pilots could help national authorities to better identify and predict likely patient numbers, gather more data for informed decision-making, and share expertise and knowledge with other countries. The number of pilots has grown sharply of late, and the platform is very close to cruise speed, according to Roux. EUROR- DIS and other individual patient experts are involved in this platform, in order to ensure that the patients voice is properly represented at the table. In so doing, we strive to break the locking of horns between industry and payers. MoCA is a place where national divergences in pricing and reimbursement can be discussed, Roux observed. It is a unique forum in Europe where you have the possibility to take a bird s eye view of the decisions taken by individual countries, and to question whether they make sense at all for example, when access to a product is granted on one side of a border but not on the other. We need better understanding. OTHER INITIATIVES As part of these efforts, EURORDIS is helping to develop a set of principles for value assessment and funding processes for rare diseases, together with a working group on value led by health economist Lieven Annemans. The principles, Roux said, have undergone a public consultation and are now being fine-tuned with a view to publication in a scientific journal. EURORDIS and the EPF are also working on a position statement on access, value, pricing and sustainability with regard to orphan drugs. A preliminary version was published in mid-may, and a more complete statement is expected to be ready for the June 17 Council meeting, where health ministers will examine the Dutch presidency s proposals on strengthening the balance in the EU pharmaceutical system. This position statement will reaffirm the central role of patients and the burning need to better factor their views in pricing and reimbursement processes, but it will also lay out principles and ideas as to how a more robust framework for fair access can be shaped, Roux said. You can expect to hear more from us on the need to defend patients with rare diseases who need access to orphan drugs and would like payers and industry to work better together. scripintelligence.com 24 June 2016 Scrip intelligence 15

16 POLICY & REGULATION BRIEFS Indian Panel Reviews Celsentri Clearances An Indian expert panel has sought data on ViiV Healthcare's HIV entry inhibitor maraviroc (Selzentry/ Celsentri) - among the drugs cleared, some years ago, with a trial waiver in the country and recommended for re-examination by a Parliamentary committee. A subject expert committee (SEC), which advises the Indian regulator on trial-related permissions as part of a layered approval process, recently noted the absence of "India specific" data since maraviroc was launched. ViiV Healthcare is the specialist HIV joint venture established by GlaxoSmithKline PLC and Pfizer Inc. in 2009; Shionogi & Co. Ltd. joined in "GSK was asked for presentation of safety, efficacy data before the SEC. The firm presented global safety and efficacy data (published in 2008). There was no data of Indian patients," details in the minutes of a SEC (antimicrobial and anti-viral) meeting held on May 23 said. The committee said that the details of the product's permissions required to be examined and safety and efficacy data be submitted before it along with reasons for delay in marketing the drug. Pfizer received marketing authorization (MA) for Celsentri in India in November 2009 and subsequently transferred the MA to GSK India. The product was launched in India in January 2014 (marketed as Axentri) and is being manufactured by Emcure. ViiV clarified that GSK does not market maraviroc in India - this is "managed" by Emcure while GSK, as such, provides regulatory support and necessary annual safety reporting. ViiV told Scrip that Indian patients were included in the Expanded Access Programme only and confirmed that maraviroc was approved in India with a trial waiver as was then permitted for drugs indicated in life threatening/serious diseases or diseases of special relevance to the Indian health scenario. A waiver of clinical trials in the Indian population for new drugs which have already Chiasma Responds To FDA Complete Response With 33% Staff Cut With a near-term launch of Mycapssa unlikely, Chiasma cuts its commercial staff while it evaluates how to respond to an April complete response letter. Three months ago, Chiasma Inc. execs spoke of being just around the corner from becoming a commercial company with the expected approval and launch of Mycapssa for acromegaly, but following an FDA "complete response" letter and an apparently unsuccessful end-of-review meeting with the agency, the Boston-area biotech is slashing its headcount 33%, much of it the commercial force it recently put in place. On June 14, Chiasma announced the workforce cut "including substantially all of its commercial personnel" included chief commercial officer Anand Varadan as part of a corporate restructuring to focus on the development of Mycapssa an oral formulation of the somatostatin analogue octreotide (Novartis AG's Sandostatin) for both the US and European markets. The firm is continuing to enroll patients in a Phase III MPOWERED study for EU approval, testing the drug against standard of care monthly somatostatin analogue injections (octreotide or Ipsen's Somatuline (lanreotide)). Chiasma's stock finished trading June 14 down 8% on the day to $2.57 per share. Previously, the stock lost more than half its market value when the "complete response" letter was announced April 18. Mycapssa had an April 15 FDA action date, but in the "complete response" the agency strongly recommended that the company conduct a randomized, doubleblind and controlled trial. The agency further specified that the new trial should enroll patients from the US and "be of sufficiently long duration to ensure that control of disease activity is stable at the time point selected for the primary efficacy assessment" been approved outside India is currently permitted only in cases of "national emergency, extreme urgency, epidemics and for orphan drugs for rare diseases and drugs indicated for conditions/diseases for which there is no therapy. India's Substitution And 'Similar' Generics Poser India's Drugs Technical Advisory Board (DTAB) has rejected a proposal to allow chemists to substitute a drug formulation with a same-ingredient generic or a cut-price brand name product, but the decision has unwittingly raised some interesting questions on how the 'pharmacy of the world' should deal with interchangeable and "similar" generics. Last month, the DTAB, the highest technical body under India's Drugs and Cosmetics Act, turned down a proposal that sought to amend rules to provide that chemists may offer for supply a drug formulation "containing the same ingredients but in generic or other cheaper brand name." The proposal was expected to authorize the chemist to sell the matching salt of branded medicines prescribed by the physician especially at India's "Jan Aushadhi" stores that offer cut-price unbranded generic medicines. Sub-rule 11A of Rule 65 of India's Drugs and Cosmetics Rules, 1945 currently specifies that no person dispensing a prescription containing substances specified in Schedule H and Schedule H1 or X may supply any other preparation, whether containing the same substances or not in lieu thereof. These rules, though, do not always hold on the ground and chemists are known to substitute brands in India. 16 Scrip intelligence 24 June 2016 Informa UK Ltd 2016

17 HEADLINE NEWS Better Together? Aegerion And QLT Merge To Solve Problems LISA LAMOTTA Aegerion and QLT will unite to create a new company dubbed Novelion in a deal that could help both companies solve some long-standing issues. Two floundering companies are joining together in what analysts and investors believe will be a fruitful combination that could solve their cash flow and diversification problems. While the move seems positive for both companies, it could hinge on the success of one late-stage product and ongoing settlements with regulators. Beleaguered Aegerion Pharmaceuticals Inc. announced June 15 that it will merge with Canada's QLT Inc. in an allstock merger that will make Aegerion a direct subsidiary of QLT. Each share of Aegerion common stock will be exchanged for shares of QLT common stock, resulting in QLT shareholders owning 67% of the new company. The resulting new company will be renamed Novelion Therapeutics and will continue to be traded on both the NASDAQ and the Toronto Stock Exchange. The deal is expected to close in the third quarter. "Today's announcement represents our commitment to achieving the transformative impact pillars of the strategy we outlined in February and bolstering our pipeline through business development initiatives," said Aegerion CEO Mary Szela on June 15. "Our global commercial capabilities in rare diseases and our patient support services are important components of the orphan drug business model. And we believe a transaction with QLT that leverages cash position and pipeline assets would create a strong platform for reimagining the Company and accelerating our potential revenue growth and profitability." In conjunction with the deal, a syndicate of old and new investors has agreed to buy 22m QLT shares. The syndicate has agreed to vote in favor of the deal and the investment will help fund the new company. If all goes as planned, Novelion will have an unrestricted cash balance of $100m. The new company will be led by current Aegerion CEO Szela. Novelion's board will have 10 members including four Aegerion designees, four QLT designees and two shareholder representatives, one from Broadfin Capital and the other from Sarissa capital management. Szela told investors that the new company could have $500m in potential revenues, including upto $100m from its currently marketed homozygous familial hypercholesterolemia (HoFH) drug Juxtapid (lomitapide), $200m to $250m from the already approved lipdystrophy drug Myalept (metreleptin) and $200 million from QLT's Phase III-ready zuretinol. Novelion will be headquartered in Vancouver, Canada, but will retain Aegerion operations in Cambridge, Mass. The merger prompted analysts to question motives behind the deal and whether the deal would trigger an advantageous tax rate. Executives on a conference call with analysts admitted that there would be potential future benefits for Novelion, but that the tax structure was not the underlying reason for the deal. GREATER THAN THE SUM OF ITS PARTS The reasons for the deal are fairly easy to discern both companies were plagued with problems. Aegerion has been trying to diversify away from Juxtapid for the last couple years as sales waned and competition from the PCSK9s entered the market. Yet, lagging sales were the least of Aegerion's problems; the company has faced a swath of lawsuits and warnings from regulators related to improper sales and marketing. Former CEO Marc Beer was even issued a warning letter by FDA for his comments on television about the intended use of Juxtapid. (Also see "BioNotebook: Aegerion lawsuit, Juno funding, Concert IPO, Salix layoffs" - Scrip, 17 Jan, 2014.) and (Also see "BUSTED: Aegerion CEO caught doing some fast-talking on 'Fast Money'" - Scrip, 12 Nov, 2013.) Aegerion reached an agreement with the Department of Justice and the Securities and Exchange Commission regarding its activities related to Juxtapid, agreeing to a settlement of $40m in May The settlement is still preliminary, but the deal includes adjustments should those numbers change and executives noted it would not be a roadblock to completing the deal. Beyond giving Aegerion a clean slate, it also gives the company the cash it needs to expand the pipeline and conduct further business development activities. "[The deal] provides us with time to reconfigure Aegerion's core business with the goal to deliver positive operating cash flow in Importantly, the transaction structure does not trigger a repayment obligation with respect to Aegerion's outstanding convertible senior notes," said Szela. For QLT, the merger provides the company with the diversification it needs, both from a product portfolio perspective, but geographically as well. "Since the election of the current QLT Board it's been our strategy to join forces with a strategic industry partner with a diversified portfolio and the infrastructure and market access capabilities to fulfill our objective of leveraging our cash, tax and general corporate assets to maximize the value of our retinoid program," said QLT Interim CEO Geoffrey Cox. Like Aegerion, QLT has had a whole mess of problems over the last few years, including several dashed attempts at previous mergers. QLT has been virtually a shell company. The company ousted its management in 2012 and went through a messy proxy battle that resulted in the selling off of its assets. The biotech has been working the last few years to make itself more palatable to an acquirer by touting its favorable tax rate and solid balance sheet, but with the closing of the tax loophole in the US and a virtually empty pipeline, QLT continued to struggle. The biotech saw several deals fall through last year, including a merger with Auxilium Pharmaceuticals Inc. (Also see "Another Deal Dashed For QLT?" - Scrip, 21 Aug, 2015.) scripintelligence.com 24 June 2016 Scrip intelligence 17

18 EXPERT VIEW Data Take: Forget Teva and Mylan - Big Pharma Owns Generics JOHN HODGSON The pool at the foot of the patent cliff is a dark, dynamic vortex. Teva's imminent acquisition of Allergan's generics division just about moves the needle: but it's big pharma that dominates the off-patent drugs business these days. Table 1. Big pharma sells far more off-patent drugs than big generics (2015 figures) CATEGORY Pharma majors* Large generics companies** PHARMA SALES ($BN) OFF-PATENT SALES ($BN) % OFF-PATENT % % *Pfizer, Sanofi, AstraZeneca, Otsuka Pharmaceutical, Novo Nordisk, Novartis, Merck & Co, GlaxoSmithKline, Bayer, Merck KGaA, Amgen, Bristol-Myers Squibb, Eli Lilly, Johnson & Johnson, Daiichi Sankyo, Boehringer Ingelheim, Biogen Idec, Gilead Sciences, Takeda ** Teva, Mylan, Fresenius Kabi, Allergan, Abbott Laboratories, Endo International, Aspen, Dr Reddy's, Lupin, Apotex, Cipla, STADA, Mallinckrodt, Hikma Pharmaceuticals, KRKA, Perrigo, Glenmark Pharmaceuticals, Alvogen Pharmaceuticals, Impax Laboratories, Strides Arcolab, Wockhardt, Sagent Pharmaceuticals, ANI Pharmaceuticals, Aurobindo, Par Pharmaceutical, Nichi-Iko, Valeant Pharmaceuticals, Cadila, Intas Pharmaceuticals, Amneal Pharmaceuticals, Lannett. Only four of the top ten companies that compete in the generic drugs market Teva Pharmaceutical Industries Ltd., Fresenius Kabi AG, Mylan NV and Sun Pharmaceutical Industries Ltd. - are "generics" companies. The premier league includes six "originators" for whom off patent drugs just happen to account for 31-68% of their drug sales - Pfizer Inc., AstraZeneca PLC, Sanofi, Novartis AG, Novo Nordisk AS, and Otsuka Pharmaceutical Co. Ltd. At least since 2011, a time when the world's biggest ever selling drug, Pfizer's Lipitor, lost a large part of its market exclusivity, the major pharmaceutical companies have been collecting more and more off-patent drugs. Big originator companies make nearly twice as much money from off-patent drugs than big generics companies do (Table 1). According to Scrip's research, 19 pharmaceutical majors (excluding Teva but including Novartis (Sandoz)) sold nearly $95bn worth of patent-expired or patent-expiring drugs in 2015, 26% of their total drug sales. By contrast, the generics sales of the top 30 generics drug companies (those not considered pharmaceutical majors) are around $55.6bn. Most of the pharmaceuticals companies do not think of their offpatent drugs as generics and, of course, by a strict regulatory definition they are not. They are originals, earlier blockbusting brands, formerly products at the edge of the pharmaceutical endeavor. But now they must compete in markets with cost-plus products. Teva is a big company and is best known for its generics sales but it doesn't top the off-patent drugs sales tree. Its Generic Medicines Division accounts for only 49% of the Israeli company's revenue, or $9.5bn. To that, add another $4bn in off-patent sales from its MS drug Copaxone's (glatiramer acetate injection) to get a total of around $13.5bn in off-patent drugs sales. (Including Copaxone under off-patent medicines seems justified even though the company allocates Copaxone's revenues to its Specialty Medicines segment; major elements of the drugs exclusivity have expired and its sales figures have fallen in each of the past two years). Adding all of Allergan PLC's $6.5bn generic division would only take Teva to second in the pecking order with sales of off-patent drugs totaling around $19.5bn, but the subtraction of $2bn in estimated acquisition-associated and other portfolio divestments takes that off-patent figure down to only $17.5bn (~70% of total pro rata drug sales). The difficulty for Teva in becoming #1 in off-patent drugs is that, fast as it makes external acquisitions of generics collections, the major pharma companies keep getting big off-patent windfalls as their former blockbuster products lose market exclusivity. Pfizer is the #1 company in off-patent drugs sales. It now boasts an Established Products division that accounts for $21bn (47% of Pfizer's total drug sales). The division includes several elements: firstly, Lipitor (still a blockbuster, generating nearly $1.9bn) plus another $9bn in 'legacy' products; secondly, another $5.3bn worth of products on the cusp of losing exclusivity; and, thirdly, nearly $4bn in sterile injectables largely from its 2015 Hospira acquisition. The major drug companies also have similar off-patent offerings and equally cute ways of describing them. Novartis, of course, has Sandoz, its $9.2bn generics division; but it also sells $6bn worth of "Established Medicines" for a total of offpatent sales in excess of $15bn. Sanofi, which has a "Generics" division with sales in 2015 of around 1.9bn, also has an "Established Pharmaceutical" division which is six times bigger than its generics operation, turning over 11.6bn: in total, that's $14.98bn at 2015 exchange rates. AstraZeneca distinguishes "Legacy" products worth $8.9bn that encompass a variety of its declining cardiovascular and respiratory products. Merck & Co. Inc., 12th in the generics league table, speaks of $3.9bn worth of "Diversified Brands" that include products such as Singulair and Nasonex. GlaxoSmithKline PLC, at 13th, formed an "Established Products" segment in April 2014 intending to spin it out into a separate company but changing its mind before the year was out. GSK's Established Products now have sales of over $4bn. Bristol-Myers Squibb Co. has $2.6bn in "Mature Products." 18 Scrip intelligence 24 June 2016 Informa UK Ltd 2016

19 EXPERT VIEW Not all major pharmaceutical companies have a separate division for their off-patent products, but they are are nevertheless a growing part of their business, at least in terms of volume. Eli Lilly & Co. is still in recovery from the declining sales of Cymbalta, Evista and Zyprexa. Biologics such as Rebif and Erbitux at Merck KGAA and Epogen and Neupogen at Amgen Inc. face biosimilar competition. And the best word for Otsuka's $5.5bn (68% of sales) exposure to off-patent competition at the moment is simply "Abilify". LOVE THE LEGACY; REVEAL THE GROWTH For businesses that pride themselves on the innovative nature of their products and their potential, pharmaceutical companies can be surprisingly candid about the magnitude of the off-patent portfolio they carry. However, for companies that are constantly in the midst of major, patent-expiry-instigated business renewal and restructuring, cramming the older gear into a warehouse out back does mean that the newer, shiny, patent-protected product lines dominate the shop window and showroom. Annual reports can focus with optimism on the "growth portfolio" while consigning the legacy, established or mature products to a footnote or a short paragraph that explains perfunctorily that lower sales were "due to the impact of competition on product X following loss of exclusivity." It makes sense to put sales of compounds in an off-patent basket when the patents expire. Sometimes, however, the accountants preparing the annual reports appear to indulge in financial sleight of hand that might mislead the unwary. For instance, in Pfizer's 2015 annual report, the "Established Products" division included patent-lapsed drugs such as the nonsteroidal anti-inflammatory Celebrex (celecoxib) and the antibiotic Zyvox (linezolid). The two drugs generated $1.7bn in sales for the Established Products segment during In 2013, according to the K, sales of the two products were worth $3.4bn, revenue that was also attributed to the Established Products segment. However, in Pfizer's annual report for 2013, the revenue for Celebrex was assigned to Pfizer's Primary Care segment and that for Zyvox to Specialty Care, neither division being part of Established Products. Allocating pre-loe revenues to a legacy segment creates an impression of a shrinking off-patent portfolio and boosts the apparent growth rate of the growth segment. Merck & Co does a similar thing in its annual reporting. It report for FY 2014 showed sales for its "Diversified Brand" grouping diminishing rapidly from just under $9bn in 2012 to just under $5bn in Included in the group were the 2012 peak sales for Singulair (montelukast sodium) and the 2013 peak sales for Nasonex (mometasone furoate monohydrate). The post-hoc assignments had the effect of boosting the appearance of Merck's non-diversified growth segment by around $3bn between 2012 and There is nothing sinful in this: companies may assign products to any segment they choose. But caveat lector: readers of annual reports should be wary. PROFITS OF DOOM Sales in the generics markets can be attractive margins. The operating margins of large generics companies like Mallinckrodt PLC and Endo International PLC are in the region of 40% of Table 2. Fat in generics: operating margins * COMPANY OPERATING MARGIN RANGE Pfizer 59-64% Teva Pharmaceutical Industries 52-54% Mallinckrodt 34-51% Endo International 27-42% Hikma Pharmaceuticals 31-36% Aspen 33-35% Actavis 32-34% Lupin 27-34% Aurobindo 28% Dr Reddy's 25-26% Cadila 22% Fresenius Kabi 20-21% KRKA 20% Abbott Laboratories 18-20% Intas Pharmaceuticals 19% Impax Laboratories 10-16% Strides Arcolab 16% Sandoz 16-18% STADA 9-15% Nichi-Iko 7-9% *For "pure" generics firms or companies that report operating profits for an off-patent segment CLICK View the main table showing top 30 companies competing in the generics space: sales. Teva's Generic division consistently reports operating margins closer to 50%. Pfizer's off-patent segment reports an operating margin near 60%, reflecting the presence of products that have only just lost protection in the "Established Products" basket and the kudos and premium associated with original brands. For companies building out international generics businesses from India and other developing markets, operating margins are somewhat tighter but far from asphyxiating. Hikma Pharmaceuticals PLC, Aspen Pharmacare Holdings Ltd., Lupin Ltd., Aurobindo Pharma Ltd., Dr Reddy's Laboratories, Zydus Cadila each have reported generics operating margins in the 20-35% range since There has been a false dichotomy between the makers of generic and "originator" drugs. This may be due in part to the perverse way in which generic drug makers calculate their pecking order. While other pharma companies compare the value of sales, generics companies count prescription numbers. Generic drugs and originator molecules thereby become apples and pears difficult to equate. However, once exclusivity is lost, there is only one market. scripintelligence.com 24 June 2016 Scrip intelligence 19

20 BUSINESS BULLETIN Does Deal With Roche Signal Eleven's Doomed Fate? Eleven Biotherapeutics Inc. more than doubled in value in early trading on June 13 before closing up 27.8% at $2.39 per share after the company said it licensed EBI-031, its lead development program, to Roche a move that Gilde Raises 250m 'Later Stage' Healthcare Fund The Gilde Healthcare IV later stage and growth capital fund has closed having raised a 250m million. The new fund, which was oversubscribed, is targeting investment opportunities in medtech, digital health and therapeutics both in Europe and the US. "It is an extension of the focus that we had in our third fund," said van der Meer. "The only change is that we have developed a bit more interest of an interest in the digital-health space. It used to consist of only home health, but now digital health is broader and has become a very important investment theme for us." Gilde expects to spend around one third of the available capital in each of the three subsectors. The new fund will make single investments of between 15-25m. "For us, later stage means that a particular company or asset can be exited in a three to four-year timeframe," explained van der Meer. "It doesn't mean that a company needs to be in advanced clinical trials; it can also be preclinical if it's a first or best in class biological asset for instance." could signal Eleven's intention to wind down the eye disease-focused company. Cambridge, Massachusetts-based Eleven was left with no clinical candidates back in January after its topical drug isunakinra (EBI-005) failed in Phase III in a second indication, forcing the company to focus on its only named preclinical program the injection EBI-031 for diabetic macular edema (DME) and uveitis. Eleven President and CEO Abbie Celniker said the company will "continue to evaluate additional strategic alternatives" following the deal with Roche, which is worth up to $270m plus royalties. Roche will pay $7.5m up front and up to $262.5m in milestone fees for EBI-005, including a payment when the US FDA validates the investigational new drug (IND) application that Eleven submitted recently to seek permission for clinical testing of EBI-031. The first milestone fee will be $22.5m if the IND becomes effective on or before Sept. 15 or $20m if it becomes effective after Sept. 15. Shutterstock: Dragon Images Dr Reddy's Pumps Complex Generics Play with Teva deal Dr. Reddy's Laboratories Ltd. is acquiring a basket of ANDAs from Teva Pharmaceutical Industries Ltd. as it increasingly leans towards developing and building a portfolio of complex and limited competition generics in the US and hopes to become "more relevant" there. The products being acquired form part of the portfolio that Teva is purging as a precondition to its closing of the $40.5bn acquisition of Allergan PLC s generics business. The Hyderabad-based Dr Reddy's said, over the weekend, that it had entered into a definitive agreement with Teva and an affiliate of Allergan plc to buy eight ANDAs for $350m in cash. The deal is contingent on the closing of the Teva/Allergan generics transaction - expected in June - and approval by the US Federal Trade Commission (FTC) of Dr Reddy s as a buyer. Teva had, at the time of its first quarter results, indicated that it was close to finalizing the divestiture list with the FTC and agreeing on the remedies. Buyers for the majority of the products had been identified, it added. The deal covers a mix of filed ANDAs pending approval and an approved ANDA, and comprises complex generic products across diverse dosage forms, a statement from Dr Reddy's said. Mesoblast Seeks New Partner To Replace Teva Australia-based Mesoblast Ltd. faced angry questions from analysts on June 14 when it tried to put a positive spin on news which followed a10-day halt in trading in its shares - that Teva Pharmaceuticals had ended a research partnership with the stem cell specialist and returned full rights to its experimental therapy for advanced chronic heart failure, which is in late-stage testing. Mesoblast shares sank upon resumption of trading, falling more than 40% from the previous close. Mesoblast had partnered with Teva Pharmaceutical Industries Ltd on the experimental stem cell therapy, called MPC-150-IM, through a 2010 partnership with Cephalon Inc. Teva bought out Cephalon for $6.8bn in 2011, putting it on the hook to fund ongoing development of MPC-150-IM. Teva has now pulled out of the partnership and fully returned the drug to Mesoblast, citing the need to focus on "core therapeutic areas. 20 Scrip intelligence 24 June 2016 Informa UK Ltd 2016

21 STOCKWATCH A Week Of Dashed Expectations ANDY SMITH Last week was another losing week in broad stock markets, driven by fears of a Brexit. The S&P 500 index was depressed by about 1% and in what is becoming an unwelcome recent trend, the NASDAQ Biotech index (NBI) underperformed the broad stock market by finishing the week down over 4%. While some of this underperformance can be attributed to risk-aversion at a time of short-term macroeconomic uncertainty, missed expectations in life science companies did not help matters. Having come a long way (down) since the peak of last summer, investors are interested in when life science stocks will stabilize, and then when they will start to outperform broad stock markets again. Technical analysts or chartists look at share price graphs and pronounce some meaning from the patterns. However, I tend think of technical analysts in the same way as Robert Harris character Cicero (in his historical fiction series on ancient Rome) thought of the auguries and soothsayers of the day. To bring Cicero s quote up to date, no doubt birds and entrails have their place in civilian government, but they sit badly with the prediction of stock market trends. When the sector renaissance occurs, it will be when the positive fundamental drivers of the sector new blockbuster product approvals, profitability, M&A and clinical effectiveness outweigh the factors currently depressing the life science investment proposition drug pricing scrutiny, market access and reimbursement, and valuations. But last week s announcement by Alnylam Pharmaceuticals Inc. at the European Hematology Association (EHA) did nothing to alleviate the mood of the sector. Alnylam s press release on its initial Phase I/II trial results for its interfering RNA product ALN-CC5 for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) was termed "positive" although its 11% share price drop on the week suggested otherwise. Unfortunately for Alnylam, there is already an established and efficacious standard of care in PNH in the form of Alexion Pharmaceuticals Inc.'s Soliris (eculizumab). Compared with Soliris, ALN-CC5's activity in three Soliris-naive patients was described as "underwhelming" by the analysts from Citigroup and "inadequate" by those from Jefferies. ALN-CC5 s place in the treatment of PNH looks to have been relegated to either a combination with Soliris (where the analysts from Jefferies described its activity as "modest") or in Soliris-refractory patients, where a further dicing of the PNH orphan indication seems unlikely to justify Alnylam's $4.8bn market capitalization. Alnylam's supporters might point to its late-stage pipeline as support for its hefty valuation although that pipeline still includes its previous foray into the treatment of RSV infection with ALN-RSV, which missed a primary endpoint in Phase IIb in It is not just 18-year-old companies in the interfering RNA field that appear to have their platforms under question but also the oldest antisense company Ionis Pharmaceuticals, Inc. which recently encountered serious safety issues in a Phase III study, 27 years after its founding. (Also see "Ionis Antisense Platform Safety Questioned After Thrombocytopenia Seen In Studies" - Scrip, 26 May, 2016.). Alnylam wasn t the only company not living up to expectations after a conference presentation last week. After the topline announcement on the benefit of Novo Nordisk AS' Victoza (liraglutide) on cardiovascular outcomes earlier in the year, the full results of the LEADER study in type 2 diabetic patients were presented at the American Diabetes Association meeting. (Also see "ADA: Victoza's 'Broad' CV Benefit Leads The Way For Semaglutide" - Scrip, 13 Jun, 2016.) Victoza demonstrated a 13% reduction in cardiovascular risk which the analysts from Leerink described as "solid & consistent" and those from Citigroup described as "significant". As with Alnylam however, investors appeared to be expecting more, probably in the non-fatal stroke or myocardial infarct endpoint, which was not significant, and the Novo stock price finished the week down 5.8%. Perhaps the most drastic example of missed expectations came from gene therapy company uniqure NV, whose share price finished the week down over 31% after it presented uncompetitive results for its product AMT-060 in five hemophilia patients at the European Hematology Association meeting. Companies need not be in competition with others in order to miss expectations, as Infinity Pharmaceuticals Inc. found when it reported the results of the single-arm Phase II DYNAMO study of duvelisib in refractory indolent non-hodgkin Lymphoma. (Also see "Infinity Doesn't Sugarcoat Disappointing Duvelisib Data; Cuts R&D Staff To Preserve Cash" - Scrip, 15 Jun, 2016.) The study met its primary endpoint of overall response rate. However, none of those responses was complete and the discovery research job cuts that were announced at the same time as the clinical trial results announcement, as well as the 68% drop in Infinity s stock price last week, only confirmed my prejudice against relying upon response rates in oncology clinical trials. This is not Infinity s first rodeo dismount and after such a long line of disappointments, the share price reaction and accompanying retrospective downgrades by sell-side analysts were to be expected. (Also see "Stockwatch: More than one born every minute" - Scrip, 20 Jun, 2013.) There is no sign of an end to the undulating doldrums in which the life science sector finds itself. Even the traditional M&A upside disappointed last week when Ariad Pharmaceuticals Inc. completed its strategic review without an announcement that it was being acquired, which at least some investors may have expected. With myriad missed expectations last week, if the week had been a college assignment for life science companies, the comment on return of the paper would be "could do better". Let s hope they do soon. N.B. The Magna Biopharma Income fund holdings include Alexion. Andy Smith is chief investment officer of Mann Bioinvest. Mann Bioinvest is the investment adviser for the Magna BioPharma Income fund which has no position in the stocks mentioned, unless stated above. Dr Smith gives an investment fund manager's view on life science companies. He has been lead fund manager for four life science specific funds, including International Biotechnology Trust and the AXA Framlington Biotech Fund, and was awarded the Technology Fund Manager of the year for scripintelligence.com 24 June 2016 Scrip intelligence 21

22 PIPELINE WATCH Scrip s weekly Pipeline Watch tabulates the most recently reported late-stage clinical trial and regulatory developments from the more than 10,000 drug candidates currently under active research worldwide. CLICK Visit scrip intelligence.com for the entire pipeline with added commentary. Late-stage clinical developments for the week June 2016 LEAD COMPANY PARTNER COMPANY DRUG INDICATION MARKET REGULATORY APPROVAL PaxVax Inc. Vaxchora vaccine cholera prevention US TaiGen Biotechnology Co. Ltd. Taigexyn (nemonoxacin) community-acquired pneumonia China SUPPLEMENTAL REGULATORY APPROVAL Roche Gazyvaro (obinutuzumab) follicular lymphoma EU Shire PLC Kamada Ltd. Glassia (alpha-1 proteinase inhibitor) emphysema Swedish Orphan Biovitrum AB Orfadin (nitisinone) hereditary tyrosinemia type-1 US REGULATORY FILING ACCEPTED Charleston Laboratories Inc. ORPHAN DRUG DESIGNATION Daiichi Sankyo Co. Ltd. Emergent BioSolutions Inc. FAST-TRACK STATUS CL-108 (promethazine, hydrocodone and acetaminophen) BioThrax (anthrax vaccine adsorbed) moderate to severe pain anthrax GlycoMimetics Inc. GMI-1271 acute myeloid leukemia US ProMetic Life Sciences Inc. Prometic (plasminogen) congenital plasminogen deficiency US COMPLETE RESPONSE LETTER KemPharm Inc. REGULATORY FILING Marathon Pharmaceuticals LLC Apadaz (benzhydrocodone and acetaminophen) acute pain deflazacort Duchenne muscular dystrophy US Perrigo Co. PLC ingenol mebutate gel psoriasis US SUPPLEMENTAL REGULATORY FILING Janssen-Cilag International Eprex (epoetin alfa) PHASE III TRIAL INITIATION Bellerophon Therapeutics Inc. INOpulse (pulsatile inhaled nitric oxide) anemia in low or intermediate-1 risk myelodysplastic syndromes pulmonary arterial hypertension Boehringer Ingelheim GMBH Ofev (nintedanib) idiopathic pulmonary fibrosis SymBio Pharmaceuticals Ltd. The Medicines Co. Ionsys (SyB P-1501) acute post-op pain Japan Vertex Pharmaceuticals Inc. PRODUCT LAUNCH Eisai Inc. Actelion Pharmaceuticals Ltd. Source: Sagient Research s BioMedTracker Nippon Shinyaku Co. Ltd. Orkambi (lumacaftor plus ivacaftor) Fycompa (perampanel) oral suspension cystic fibrosis epilepsy Uptravi (selexipag) pulmonary arterial hypertension Germany US US US US EU US 22 Scrip intelligence 24 June 2016 Informa UK Ltd 2016

23 APPOINTMENTS Bristol-Meyers Squibb has appointed Murdo Gordon executive vice president and chief commercial officer, a newly created role. Gordon has been with the global biopharma company since 1989, holding positions of increasing responsibility including senior vice president for US oncology and immunoscience from , and president of US operations. Most recently, he co-led the commercial organisation of the company as head of worldwide markets. Late-stage biopharma company Soligenix Inc. has appointed Karen Krumeich senior vice president and chief financial officer (CFO), following the retirement of current acting CFO Joseph Warusz at the end of this month. Prior to joining Soligenix, Krumeich served as a consultant providing finance, investor relations and business development services to the company, before which she was vice president and CFO for several development-stage life science companies, including Cerecor Inc. and Mela Sciences Inc. Lysogene, a biotech company specialising in the treatment of central nervous system diseases, has appointed Kimberley S. Gannon chief scientific officer. Gannon joins the business from NeuroPhage Pharmaceuticals Inc., where she was senior vice president of preclinical research and development. Prior to this, she held several leadership positions at other companies, including vice president of research and development at CereMedix, and senior director of biology at EPIX (Predix) Pharmaceuticals. Her expertise spans preclinical pharmacology, translational medicine and nonclinical drug development. Personalised therapy specialist Endocyte Inc. has appointed Mike Sherman president and CEO, succeeding founding CEO Ron Ellis, who has resigned effective immediately. Sherman has been the company s chief financial officer since 2006, and took on the additional role of chief operating officer in Prior to this, held a variety of executive roles, most recently as vice president of finance and strategic planning at Guidant Corporation. Amgen has appointed Esteban Santos executive vice president, operations, succeeding Madhavan Balachandran, who is retiring at the end of the year. Santos was senior vice president, manufacturing since 2013, before which he held several leadership roles at the company, including vice president, drug product, and vice president, manufacturing. Prior to joining Amgen in 2007, he was site general manager for the Johnson & Johnson Cordis operation in Puerto Rico, having previously helped to manage General Electric s industrial and transportation businesses in Puerto Rico, Connecticut and Pennsylvania. Novel small molecule drug developer Arena Pharmaceuticals, Inc. has appointed Kevn R. Lind executive vice president and chief financial officer. Lind has over 15 years of experience in healthcare banking and private equity, most recently at TPG, where he was a principal at TPG Special Situations Partners and initially at TPG-Axon s Pharma Partners Group. Prior to this, he spent the early years of his career as a healthcare investment banker at Lehman Brothers, Inc. Hoopika Biotech AG has appointed Jörn Aldag CEO, replacing founding CEO Katherine Cohen. Aldag joins the immunotherapy company from uniqure N.V., where he was CEO, having previously been president and CEO of Evotec AG from 1997 to Alongside his position leading Hoopika, Aldag will remain chair of molecular partners and a board member of Unum Therapeutics, both of which are developing next generation immune-oncology therapies. Scrip ELEANOR ALEXANDRA DONNA MANDY JOANNE FRANCESCA STEN IAN ASHLEY LISA LUCIE LUBNA PAUL WILKINSON JOHN MIKE PETER JOHN SARAH All stock images in this publication courtesy of unless otherwise stated. Customer Services Tel: +44 (0) or (US) Toll Free: To subscribe, visit scripintelligence.com To advertise, contact Scrip is published by Informa UK Limited. Informa UK Ltd 2016: All rights reserved. ISSN scripintelligence.com 24 June 2016 Scrip intelligence 23

24 Maximize Your Reimbursement Potential The balance of power behind the prescribing decision is changing: payers are ever more in charge. That means that insight into how payers make decisions how they evaluate drugs, one against another will be crucial to any successful drug launch. RxScorecard objectively, authoritatively, and systematically assesses marketed and pipeline drugs in a therapeutic indication from the payer s point of view. Developed by senior medical and pharmacy leaders from major payers and pharmacy benefit managers, RxScorecard delivers practical and powerful insight into your drug s reimbursement potential and how you can maximize it. Transparent, objective, and grounded in payer data, RxScorecard helps you refine your development path, future-proof your market access strategy, and achieve payer acceptance. Discover RxScorecard today. Visit to review the selection of RxScorecards today. Interact with the data. Compare drugs on clinical, safety, and economic metrics. See the payer perspective.

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