PAION. Dr Wolfgang Söhngen, CEO. European Business Development Conference, 23 Sept. 2013

Transcription

1 PAION Dr Wolfgang Söhngen, CEO European Business Development Conference, 23 Sept. 2013

2 Disclaimer It is important to note that this information contains forward-looking statements which are based on the currently held beliefs and assumptions of the management of PAION AG, which are expressed in good faith and, in its opinion, reasonable. Forward-looking statements involve known and unknown risks, uncertainties and other factors, which may cause the actual results, financial condition, performance, or achievements of PAION AG, or industry results, to differ materially from the results, financial condition, performance or achievements expressed or implied by such forward-looking statements. Given these risks, uncertainties and other factors, recipients of this information are cautioned not to place undue reliance on these forward-looking statements. PAION AG disclaims any obligation to update these forward-looking statements to reflect future events or developments. This presentation constitutes neither an offer to sell nor a solicitation to buy any securities in Germany, the United Kingdom, the United States or any other jurisdiction. Neither this presentation nor anything contained herein shall form the basis of, or be relied on in connection with, any offer or commitment whatsoever. In particular, this presentation is not an offer for sale of securities in the United States. Securities may not be offered or sold in the United States absent registration or an exemption from registration under the U.S. Securities Act of 1933, as amended. PAION AG (the "Company") does not intend to register any securities of the Company in the United States. 2

3 Corporate overview PAION AG is transforming its business model from a pure development company to a specialty pharmaceutical company with a focus on anaesthesia products Focus on Remimazolam, a promising ultra-short acting anaesthetic/ sedative with significant market potential 3

4 Corporate Highlights Remimazolam Formal scientific advice meeting in Feb One Phase II and one Phase III trial in General Anaesthesia in addition to the Japanese Phase II/III trial will be sufficient for EU approval Recruitment completed for Phase II/III Study in anaesthesia by Ono in May 2013 Recruitment completed for Phase III Study in anaesthesia (ASA III or higher) by Ono Option agreement with Hana Pharm re license in South Korea in July 2013 Start of Phase II study in EU anaesthesia study in September 2013 GGF2 Positive results of Phase I Study by Acorda in March 2013 FDA has granted Fast Track designation Second GGF2 trial in heart failure planned for 2H

5 Financialsi 5

6 Financial Outlook 2013 Revenues Revenues of EUR 1.5 million EUR 1.2 million for completion of tech transfer with Yichang EUR 0.3 million option fee Hana Pharm If Hana Pharm license can be closed in 2013 additional EUR 0.7 million Income from the potential partnering of Remimazolam is not included in this outlook Costs Reduced cost structure Research and development expenses will be higher than in the previous year SG&A costs will be lower than in the previous year 6

7 Financial Outlook 2013 Net loss Net loss in the mid to high single-digit million range expected for 2013 In case of further out-licensing deals the loss could be reduced Liquidity Cash of EUR 14.2 million as of 30 June 2013 Cash reach until end of Q (based on current structure and including European Phase II study in anaesthesia) 7

8 Product pipeline 8

9 Pipeline overview Compound Remimazolam i.v. anaesthetic/sedative Remimazolam i.v. anaesthetic/sedative Remimazolam i.v. anaesthetic/sedative Remimazolam i.v. anaesthetic/sedative M6G iv i.v. opioid Solulin i.v. thrombomodulin Indication Anaesthesia Anaesthesia Procedural Sedation ICU sedation Peri-operative pain Haemophilia PN13 i.v. thrombomodulin Haemophilia mutants GGF2 i.v. glial growth factor Status of development PC PI PII PIII Ono PAION PAION Ono No development activities No development activities Earliest launch Estimated peak sales (US$m) Partners } Ono (Japan), Yichang (China) 500 Ono (Japan), Yichang (China) 500 Ono (Japan), Yichang (China) Grant by BMBF Heart failure Acorda (ww) 9

10 Remimazolam a Game Changer Opportunity Remimazolam is an ultra short-acting anesthetic/sedative activating the GABAA receptor designed to provide fast onset of sedation with rapid recoveryer Desired sedative profile with rapid recovery to alertness A compelling pharmacoeconomic case can be built from Remimazolam s favorable profile Key design feature is an ester group that is readily cleaved into inactive metabolites by ubiquitous tissue esterases Primary metabolism is not via CYP 450 enzymes Results in a very short half life, predictable pharmacokinetics and faster time to recovery Composition of matter until 2020, extension expected to 2025 Besylate salt patent to 2027 Further patents granted and applications filed, exclusivity expected to Comprehensive preclinical package in place (wide therapeutic window) More than 800 volunteers/patients on drug 10

11 Competitive Product Profile for Remimazolam Procedural Sedation Product Profiles Remimazolam Midazolam Propofol Fospropofol Rapid time to peak effect Rapid offset Predictable recovery time Early discharge? Low respiratory depression Less resources for supervision Early recovery to full cognition Reversal agent available Low re-sedation risk after reversal No (low) pain on injection 11

12 Competitive Product Profile for Remimazolam General anaesthesia Product Profiles Remimazolam Propofol Sevoflurane Rapid time to peak effect Rapid offset Predictable recovery time Early discharge Low respiratory depression Cardiostability Early recovery to full cognition Reversal agent available Low re-sedation risk after reversal No (low) pain on injection Low risk of excitation No risk ik of theatre contamination i No risk of atmospheric pollution Simple equipment to handle during induction of narcosis 12

13 Pricing and Penetration Analogy Dexmedetomidine for ICU Sedation in the US Dexmedetomidine vs. Remimazolam Unmet Needs Remimazolam Dexmed Priced at $300 to $600 per day of Rapid and predictable onset ICU Sedation Rapid and predictable recovery Strong growth in 2009 and 2010 Low risk of accumulation shows acceptance of this higher Easier ICU pat. mgmt. (comm. and coop.) price. US 2010 sales of $123Mio Early discharge (up 67% from 2009) Dexmedetomidine is a vastly Low cardio & respiratory depression inferior product to Remimazolam Fewer resources for supervision (See table on right) Early recovery to full cognition Pi Pricing i in Europe 170 to 204. Reversal agent available Dexmedetomidine has proven that entering a generic market at a No re-sedation risk after reversal significant ifi multiple l to the existing No (low) pain on injection generics (clonidin, midazolam and Appropriate for deep sedation and? propofol) is possible both in the anaesthesia US and EU Sleep-like lik Sedation 13

14 Monitored Anaesthesia Care (MAC) costs are estimated $1.2- $8 billion annually in the US Rand study shows increasing i use of propofol and added d costs by need for supervision with anaesthesiologist Use of anesthesia services for low-risk patients during gastrointestinal endoscopies may have increased steadily to more than $1.1 1 billion per year at the national level. 1 Cohen and colleagues estimate costs of up to $ 8 billion if anesthesia services were used for all 20 million endoscopic procedures. 2 Vargo et alshowed the gastroenterologists were able to perform three colonoscopies under propofol sedation in the time it takes to perform two colonoscopies with conventional sedation. This significant improvement in efficiency i translated t into measurable decreases in the operating costs, nurse requirements, and bed requirements in the recovery area. 3 These costs are not sustainable under any healthcare reform 1 Rand Corporation, Boston 2 Cohen LB, Benson AA, Issues in endoscopic sedation. Gastroenterol Hepatol 2009; 5: Vargo JJ, Bramley T, Meyer K, Nightengale B. Practice efficiency and economics: the case for rapid recovery sedation agents for colonoscopy in a screening population. J Clin Gastroenterol 2007; 41:

15 FDA judges risks with propofol require anesthesiologist In response to J&J s Sedasys Application: The fundamental reason for this warning [i.e. on the label requiring an anesthetist] is the potential for patients to become completely unresponsive to stimulation and lose their inherent capacity to maintain adequate respiratory gas exchange through a patent airway. Also, endogenous reflexes that protect the respiratory tract from aspiration are lost as patients become deeply sedated. A small increment in propofol dose can transition a patient from being capable of self sustaining their vital signs to a state of general anaesthesia where vital signs must be vigilantly monitored and managed. There is no pharmacological reversal agent for an overdose of propofol. Therefore patients who become unresponsive and also lose respiratory integrity require immediate mechanical intervention to prevent a serious adverse event or death of the patient. 15

16 Result of intensive primary market research lead to re-positioning i US Situation Insurance companies pay billions for colonoscopies with propofol Can not be financed with reduced d health budget Situation is aggravated by the increasing shortage of anaesthesiologists Some payers refuse to pay for anaesthesiologists Pressure to use Midazolam reduces efficiency of GI doctors EU Situation Medical need in elderly patients and cardiac surgery Propofol with cardio-depressive activity is sub-optimal Willingness to accept higher price if total cost can be reduced Shortening the time on the ICU Therefore, colonoscopy is U.S. lead indication Therefore, general anaesthesia is EU lead indication 16

17 Remimazolam - over 800 volunteers/patients on drug Completed Studies Anaesthesia (Ono) Procedural Sedation (PAION) Phase I bolus in healthy volunteers (Japan) Phase I infusion in healthy volunteers (Japan) Phase II induction and maintenance of anaesthesia in general surgery (Japan) Phase II/III in surgical patients (Japan) Phase III in ASA III or higher surgical patients (Japan) Earliest launch expected 2015 Planned/Ongoing Studies Phase I single bolus in healthy volunteers (USA) Phase I multiple bolus in volunteers undergoing colonoscopy (USA) Phase IIa single bolus in Upper GI Endoscopy Phase IIb multiple bolus in Colonoscopy Pending to funding earliest launch expected 2016 ICU Sedation (Ono) Phase II in ICU sedation (Japan) under review Phase III in ICU sedation (Japan) - planned Anaesthesia (PAION) Phase II in cardiac surgery patients (Germany) started Phase III in cardiac surgery patients (countries tbd) planned in 2014 pending to funding 17

18 Remimazolam on track for target profiles Rapid onset of sedation Rapid offset of sedation Wake up times are independent of length of infusion and are comparable between procedural sedation and general anaesthesia Linear pharmacokinetics Clearance not related to body weight Reversed by flumazenil Benzodiazepine-like safety profile No serious adverse events and no unusual findings Good cardiovascular and respiratory stability No clinically i ll relevant changes in ECG No (low) pain on injection 18

19 Our Vision 19

20 Milestones 2013 Global and regional partnering discussions ongoing g Evaluation of different funding options to perform Phase III study in EU in general anaesthesia Evaluation of different funding options to perform Phase III study in US in procedural sedation Start of European Phase II trial in cardiac surgery (PAION) Hana Pharm license agreement 2014 Phase III study results in general anaesthesia (ONO) 20

21 Summary PAION has the opportunity to address a medical need by bringing a safer, short-acting, predictable agent to the market with lower development risk given remimazolam s drug class and safety profile and short development timelines with relatively low costs to approval. All great companies have been built on one product with a disruptive potential ti 21

22 Thank you very much for your attention! Contact: PAION AG M ti t Martinstrasse Aachen Germany Phone info@paion.com 22