Jefferies 2014 Global Healthcare Conference London, UK November 19, :00 AM. November

Transcription

1 Jefferies 2014 Global Healthcare Conference London, UK November 19, :00 AM November

2 Forward Looking Statements This document contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including certain plans, expectations, goals and projections regarding financial results, product developments and technology platforms. All statements that are not clearly historical in nature are forward-looking, and the words "anticipate," "assume," "believe," "expect," "estimate," "plan," "will," "may," and similar expressions are generally intended to identify forward-looking statements. All forward-looking statements involve risks, uncertainties and contingencies, many of which are beyond our control that could cause actual results to differ materially from those contemplated in such forward-looking statements. These risks include risks that the launch of Bloxiverz will not be as successful as anticipated; our ability to bring other R&D projects of the former Éclat Pharmaceuticals to market may be unsuccessful; FDA may not take action on the status of unapproved versions of neostigmine still on the market; clinical trial results may not be positive or our partners may decide not to move forward; products in the development stage may not achieve scientific objectives or milestones or meet stringent regulatory requirements; products in development may not achieve market acceptance; competitive products and pricing may hinder our commercial opportunities; we may not be successful in identifying and pursuing opportunities to develop our own product portfolio using Flamel's technology; and the risks associated with our reliance on outside parties and key strategic alliances. These and other risks are described more fully in Flamel's Annual Report on Form 20-F for the year ended December 31, 2013 that has been filed with the Securities and Exchange Commission (SEC). All forward-looking statements included in this release are based on information available at the time of the release. We undertake no obligation to update or alter our forward-looking statements as a result of new information, future events or otherwise. November

3 Key Highlights Best-in-class drug delivery platforms creating a broad, proprietary product pipeline 5 products in development using four delivery platforms Generate cash flow through FDA (NDA) approval for currently unapproved marketed drugs (UMDs) with established need Bloxiverz (neostigmine methylsulfate) approved on May 31, 2013 and launched in 3Q 2013 Vazculep (phenylephrine hydrochloride injection) approved on June 30, 2014 and launched in October 2014 Utilize cash flow from UMDs for development of a proprietary pipeline expected to deliver 6-8 regulatory filings through 2017 NDA filing anticipated in 2015 for our third UMD Expect 4-6 regulatory filings in using Flamel s technology Utilizing the 505(b)2 pathway known safe and efficacious drugs Anticipate being cash flow positive in 4Q 2014 November

4 Unapproved Marketed Drug (UMD) Strategy There are many products marketed in the United States that have never received FDA approval and are not covered by DESI (Drug Efficacy Study Implementation) FDA has provided guidance on how it will deal with these situations Guidance for FDA Staff and Industry - Marketed Unapproved Drugs - Compliance Policy Guide Per FDA policy, once an NDA approval has been obtained for an UMD FDA normally intends to allow a grace period of roughly 1 year from the date of approval of the product before it will initiate enforcement action (e.g., seizure or injunction) against marketed unapproved products of the same type. However, the grace period provided is expected to vary from this baseline based upon certain factors In the case of Bloxiverz, on July 30, 2014, 14 months after approval, the FDA ordered all manufacturers of unapproved product to cease manufacturing. Strategy is focused on a limited number of opportunities with an established medical need, few competitors and the potential for a meaningful return on investment Flamel has identified opportunities beyond Bloxiverz, the first of which is Vazculep (phenylephrine HCL injection) approved in June 2014 November

5 Marketed Product BLOXIVERZ FDA approval on May 31, 2013 (first and only FDA-approved neostigmine methylsulfate injection) Launched during third quarter of 2013 Strengths: 0.5 mg/ml or 1 mg/ml (10 ml MDV) Indication: reversal of the effects of non-depolarizing neuromuscular blocking agents after surgery Bloxiverz WAC price is $35.80 per vial There are approximately 5 million vials sold annually in the United States Website: November

6 Marketed Product VAZCULEP FDA approval on June 30, 2014 (only FDA-approved phenylephrine injection available in three vial sizes) Launched in October 2014 Form: 1 ml single use vials, 5 ml and 10 ml pharmacy bulk package vials for intravenous injection (bolus or infusion) (10 mg/ml) Indication: treatment of clinically important hypotension resulting primarily from vasodilation in the setting of anesthesia Recent data show the following approximate annual unit volumes: 1ml vial 5.6m 5ml vial 1.3m 10ml vial 140k Website: November

7 Broad Drug Delivery Portfolio is Distinctive Flamel owns and develops outstanding drug delivery platforms that are able to tackle key challenges in the formulation, in various dosage forms (e.g. capsules, tablets, sachets or oral liquid suspensions; or injectable for subcutaneous administration) of a broad range of drugs (novel, already-marketed, or off-patent): The Company is developing specific applications of its proprietary, versatile drug delivery platforms for developing new formulations internally or with partners Micropump, LiquiTime, Trigger Lock and Medusa are trademarks of Flamel Technologies S.A. November

8 Flamel Pipeline (4Q 2014) Drug/ Technology Indication Pre- Clinical Proof of Concept Dose Ranging Pivotal Under Review Approved Marketed Sales Force Bloxiverz/UMD* Anesthesia Flamel Vazculep/UMD* Anesthesia Flamel UD/UMD* Undisclosed Flamel UD/UMD* Undisclosed Flamel Sodium oxybate /Micropump Narcolepsy Ibuprofen/LiquiTime Pain TBD UD/LiquiTime Respiratory TBD Opioid/Trigger Lock Pain TBD Exenatide XL/Medusa Diabetes hgh/medusa Short Stature TBD *UMD is Flamel s Unapproved Marketed Drugs Strategy, but does not involve patented technology. UD = undisclosed TBD= To Be Determined TBD TBD November

9 Flamel Pipeline Looking Ahead (2Q, 2016) Drug/ Technology Indication Pre- Clinical Proof of Concept Dose Ranging Pivotal Under Review Approved Marketed Sales Force Bloxiverz/UMD* Anesthesia Flamel Vazculep/UMD* Anesthesia Flamel Disclosed/UMD* Disclosed Flamel Disclosed/UMD* Disclosed Flamel Sodium oxybate /Micropump Narcolepsy Ibuprofen/LiquiTime Pain TBD Disclosed/LiquiTime Respiratory TBD Opioid/Trigger Lock Pain TBD Exenatide XL/Medusa Diabetes hgh/medusa Short Stature TBD *UMD is Flamel s Unapproved Marketed Drugs Strategy, but does not involve patented technology. UD = undisclosed TBD= To Be Determined TBD TBD November

10 Upcoming Milestones Technology 2H H 2015 Vazculep (UMD Product #2*) UMD Product #3* UMD Product #4* Launch FDA NDA filing Micropump LiquiTime Trigger Lock Medusa Proof-of-concept study extension data Sodium oxybate Clinical data 1 program (ibuprofen) PK/PD data Sodium oxybate Clinical data 1 program (respiratory) Clinical data 1 program Clinical study initiation 1 program (diabetes) * UMD is our Unapproved Marketed Drugs strategy November

11 LiquiTime Ibuprofen Positive First-in-Man Clinical Trial Results Ibuprofen twice-daily dosing First-in-Man (FIM) clinical study in healthy volunteers (15 subjects) Trial design: open-label, randomized, 3-period, 3-way crossover study Liquitime formulation 1 LiquiTime formulation 2 Immediate release ibuprofen PK results announced in September 2014 Bioequivalence to the immediate-release ibuprofen oral suspension Similar onset versus IR Similar blood levels at 12 hours versus IR No safety nor tolerability issues Target: global OTC (over-the-counter) market Pivotal bioequivalence study to be initiated in 2015 November

12 Sodium Oxybate Micropump Microparticles Positive First-in-Man Clinical Trial Results Sodium oxybate, a Micropump -based formulation for one single dose before bedtime for patients suffering from narcolepsy, eliminating the need for a second dose. The current dosing regimen for the standard of care, Xyrem (sodium oxybate), in the U.S. is two equal, divided doses: the first dose at bedtime and the second dose 2.5 to 4 hours later Flamel s results of its FIM (First-in-Man) clinical study in human subjects, published in April 2014, demonstrated the elimination of the second nighttime dose. The key data for 14 evaluable subjects at a nightly dose of 4.5 g (two doses of 2.25 g for Xyrem) are: Onset of action similar to Xyrem ; C max lower than Xyrem ; and, Mean blood concentration ( g/ml) at hours 7 and 8 similar to Xyrem. The profiles of two selected formulations at 6 g in 13 evaluable subjects were consistent with expectations. The current study will continue to treat subjects at higher doses. Given these results, Flamel plans: 1) a second PK study dosing patients up to 7.5 g with results expected before year end 2014; 2) a new clinical study to further evaluate PK/PD is expected to yield results around mid-year 2015 November

13 Intellectual Property Broad portfolio of patents* Date of expiration of granted patents Platform US Europe Micropump November 2025 July 2023 LiquiTime September 2025 April 2023 Trigger Lock April 2027 May 2026 (pending) Medusa December 2029 May 2028 (pending) *as of December 31, 2013 (source: 20-F for 2013) New patents may be issued targeting each individual program where a Flamel drug delivery platform is applied to a specific molecule November

14 Condensed Consolidated Statement of Operations Unaudited Nine months ended In USD million, except EPS and share data September 30, 2013 September 30, 2014 Total Revenue Total Costs and Expenses (67.3) (85.2) Loss from operations (51.0) (61.0) Net Loss (48.1) (57.8) Diluted EPS (1.89) (1.64) Adjusted Diluted EPS (non GAAP) (0.62) (0.44) Diluted Shares Outstanding November

15 Condensed Balance Sheet (Unaudited) In USD million Cash & marketable securities As of Dec. 31, 2013 As of June 30, 2014 As of September 30, LT debt Other LT liabilities includes government loans for R&D projects + acquisition liability contingent consideration, note and warrant consideration, and facility and royalty agreements concluded in February 2013 and December includes R&D credit tax financing, funding from partner GSK, provision for retirement indemnity and employee service award provision November

16 Senior Management Name Title Joined Flamel Experience Michael S. Anderson Chief Executive Officer years Pharma Siân Crouzet Principal Financial Officer years Financial Steven A. Lisi David Monteith, Ph.D. Phillandas T. Thompson, J.D., M.B.A. SVP, Business and Corporate Development VP, Research and Development years Financial years Pharma SVP, General Counsel years Legal November

17 Key Highlights Best-in-class drug delivery platforms creating a broad, proprietary product pipeline 5 products in development using four delivery platforms Generate cash flow through FDA (NDA) approval for currently unapproved marketed drugs (UMDs) with established need Bloxiverz (neostigmine methylsulfate) approved on May 31, 2013 and launched in 3Q 2013 Vazculep (phenylephrine hydrochloride injection) approved on June 30, 2014 and launched in October 2014 Utilize cash flow from UMDs for development of a proprietary pipeline expected to deliver 6-8 regulatory filings through 2017 NDA filing anticipated in 2015 for our third UMD Expect 4-6 regulatory filings in using Flamel s technology Utilizing the 505(b)2 pathway known safe and efficacious drugs Anticipate being cash flow positive in 4Q 2014 November

18 Advanced Formulation and Delivery Platforms for Better and Safer Drugs Headquarters 33 avenue du Dr. Georges Levy Vénissieux Cedex France Corporate Contact Phone: Fax: Flamel Technologies SA s (NASDAQ: FLML) business model is to blend high-value internally developed products with its leading drug delivery capabilities. The company markets Bloxiverz (neostigmine methylsulfate) and Vazculep (phenylephrine hydrochloride) in the USA and manufactures Micropump-based microparticles under FDA-audited GMP guidelines for Coreg CR (carvedilol phosphate), marketed in the USA by GlaxoSmithKline. The Company has a proprietary pipeline of niche specialty pharmaceutical products, while its drug delivery platforms are focused on the goal of developing safer, more efficacious formulations of drugs to address unmet medical needs. Its pipeline includes chemical and biological drugs formulated with its Micropump (and its applications to the development of liquid formulations LiquiTime and of abusedeterrent formulations Trigger Lock ) and Medusa proprietary drug delivery platforms. Several Medusabased products have been successfully tested in clinical trials. The Company is headquartered in Lyon, France and has operations in St. Louis, Missouri, USA, and manufacturing facilities in Pessac, France. Additional information can be found at November