Forward Looking Statements



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Transcription:

September 2015 1

Forward Looking Statements This document includes statements concerning our operating results (including product sales), financial condition and product development milestones, which are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements herein that are not clearly historical in nature are forward-looking, and the words anticipate, assume, believe, expect, estimate, plan, will, may, and the negative of these and similar expressions generally identify forward-looking statements. All forward-looking statements involve risks, uncertainties and contingencies, many of which are beyond Flamel s control and could cause actual results to differ materially from the results contemplated in such forwardlooking statements. These risks, uncertainties and contingencies include the risks relating to: our dependence on a small number of products and customers for the majority of our revenues; the possibility that our Bloxiverz and Vazculep products, which are not patent protected, could face substantial competition resulting in a loss of market share or forcing us to reduce the prices we charge for those products; the possibility that we could fail to successfully complete the research and development for the two pipeline products we are evaluating for potential application to the FDA pursuant to our unapproved-to-approved strategy, or that competitors could complete the development of such products and apply for FDA approval of such products before us; our dependence on the performance of third parties in partnerships or strategic alliances for the commercialization of some of our products; the possibility that our products may not reach the commercial market or gain market acceptance; our need to invest substantial sums in research and development in order to remain competitive; our dependence on certain single providers for development of several of our drug delivery platforms and products; our dependence on a limited number of suppliers to manufacture our products and to deliver certain raw materials used in our products; the possibility that our competitors may develop and market technologies or products that are more effective or safer than ours, or obtain regulatory approval and market such technologies or products before we do; the challenges in protecting the intellectual property underlying our drug delivery platforms and other products; our dependence on key personnel to execute our business plan; the possibility that we may cease to qualify as a foreign private issuer, which would increase the costs and expenses we incur to comply with U.S. securities laws; and the other risks, uncertainties and contingencies described in the Company s filings with the U.S. Securities and Exchange Commission, including our annual report on Form 20-F for the year ended December 31, 2014, all of which filings are also available on the Company s website. Flamel undertakes no obligation to update its forward-looking statements as a result of new information, future events or otherwise, except as required by law. September 2015 2

Flamel Technologies Transformed Fully integrated global specialty pharmaceutical company with FDA approved products and a broad pipeline using Flamel s proprietary drug delivery platforms Two FDA approved products marketed by Flamel in the United States will drive significant revenue growth in 2015 Incremental clinical data for several of Flamel s pipeline products over the next 12-18 months Cash flow positive as of Q4 2014 and will be cash flow positive in 2015 and beyond (six months 2015 adjusted net income non GAAP = $18.3m) Net cash position of over $116m on June 30, 2015 provides for a strong balance sheet New strategy: Flamel controls 100% of its drug development September 2015 3

Key Milestones Achieved Launch of Bloxiverz and Vazculep in the United States (July 2013 and October 2014) Completed an equity raise of $113m (net proceeds) (March 2014) Eliminated virtually all debt Divested contract manufacturing facility (December 2014) Clear focus on product development and revenue generation Implemented corporate reorganization (December 2014) Established footprint in Ireland and shifted all intellectual property from France to Ireland Presented positive clinical and preclinical data Micropump sodium oxybate: eliminated middle of the night dose (December 2014) LiquiTime ibuprofen: twice-daily dosing confirmed (September 2014) LiquiTime guaifenesin: twice-daily dosing confirmed (March 2015) Trigger Lock hydromorphone: bioequivalence criteria met (under fasted conditions) (June 2015) Medusa exenatide: PK* profile compatible with release over one week in humans (June 2014) *PK = pharmacokinetics September 2015 4

2015 Expectations Achieve total product sales of $170-185m Submit an application for UMD#3 to the FDA NDA accepted for filing by FDA - September 2015 (PDUFA date: April 30, 2016) Start pivotal study for Micropump sodium oxybate Present human clinical data on at least one additional LiquiTime product Positive LiquiTime guaifenesin First-In-Man data announced in March 2015 Enter into an agreement for one or more LiquiTime OTC products Present PK data and abuse deterrence data on Trigger Lock hydromorphone Positive results from two pilot PK studies (fed and fasted conditions) in healthy volunteers announced in June 2015 Present First-in-Man interim data on Medusa exenatide September 2015 5

Flamel s Goals Upcoming Milestones Evolution of Pipeline September 2015 6

Upcoming Milestones Technology H1 2015 H2 2015 UMD Product #3* NDA filed with FDA Accepted by FDA - September 2015 Micropump LiquiTime Trigger Lock Pre-IND meeting with FDA for sodium oxybate Clinical data for guaifenesin Positive PK results announced in March 2015 Clinical data for hydromorphone Positive PK results in healthy volunteers (fed and fasted conditions) announced in June 2015 Abuse deterrence data generated inhouse Sodium oxybate pivotal clinical initiation Ibuprofen pivotal clinical initiation Abuse deterrence data generated independently by a contract research organization FDA meeting request Medusa Phase I dosing begins in August with first clinical data available around year-end *UMD is Flamel s Unapproved Marketed Drugs Strategy September 2015 7

Flamel s Pipeline (Q3 2015) 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* #3 Undisclosed Flamel UD/UMD* #4 Undisclosed Flamel Sodium oxybate/ Micropump Narcolepsy Ibuprofen/LiquiTime Pain, fever TBD Guaifenesin/LiquiTime Respiratory Hydromorphone/ Trigger Lock Pain Exenatide/Medusa Diabetes TBD TBD TBD TBD *UMD is Flamel s Unapproved Marketed Drugs Strategy that does not involve patented technology UD = undisclosed TBD= To Be Determined September 2015 8

Flamel s Pipeline Looking Ahead (Q3 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* #3 Disclosed Flamel Disclosed/UMD* #4 Disclosed Flamel Sodium oxybate/ Micropump Narcolepsy Ibuprofen/LiquiTime Pain, fever TBD Guaifenesin/LiquiTime Respiratory Hydromorphone/ Trigger Lock Pain Exenatide/Medusa Diabetes TBD TBD TBD TBD *UMD is Flamel s Unapproved Marketed Drugs Strategy that does not involve patented technology TBD= To Be Determined September 2015 9

Flamel s Marketed Products Bloxiverz Vazculep September 2015 10

Marketed Product BLOXIVERZ FDA approval on May 31, 2013 (first FDA-approved neostigmine methylsulfate injection) Strengths: 0.5 mg/ml or 1 mg/ml (10 ml multiple-dose vial) Indication: reversal of the effects of non-depolarizing neuromuscular blocking agents after surgery Bloxiverz WAC price is $98.75 per vial There are approximately 5 million vials sold annually in the United States More details can be found at www.bloxiverz.com September 2015 11

Marketed Product VAZCULEP FDA approval on June 30, 2014 (only FDA-approved phenylephrine injection available in three vial sizes) Form: 1 ml single use vials, 5 ml and 10 ml pharmacy bulk package vials for intravenous injection (bolus or infusion) (strength: 10 mg/ml) Indication: Treatment of clinically important hypotension resulting primarily from vasodilation in the setting of anesthesia WAC Prices for all three sizes are competitive Recent data show the following approximate annual unit volumes: 1mL vial 5.6m 5mL vial 1.3m 10mL vial 140K More details can be found at www.vazculep.com September 2015 12

Flamel s R&D Pipeline Micropump sodium oxybate LiquiTime ibuprofen LiquiTime guaifenesin Trigger-Lock hydromorphone Medusa exenatide September 2015 13

Micropump Sodium Oxybate Treatment of Narcolepsy Single Dose Nightly Formulation Micropump sodium oxybate has been studied in 40 healthy volunteers to date across 2 studies Results of two studies at doses of 4.5g and 6g show the following: Onset of action similar to 4.5g nightly dose of Xyrem (Jazz s sodium oxybate) C max lower than 4.5g nightly dose of Xyrem (6g dose similar to Xyrem 4.5g dose) Mean blood concentration (μg/ml) at hours 7 and 8 similar to 4.5g nightly dose of Xyrem No adverse events or tolerability issues Micropump sodium oxybate 7.5g dose performed in line with expectations relative to the data at lower doses of Micropump sodium oxybate Profile is consistent with the need for only one single dose before bedtime Current dosing regimen for Xyrem, the standard of care 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 has met with the FDA in H1 2015 and is on target to begin pivotal study at year end 2015 September 2015 14

Micropump Sodium Oxybate Market Opportunity 2014 sales for Xyrem were $779m 1 CAGR for Xyrem from 2009-2014 is in excess of 50% The number of narcoleptics in the US is estimated to be in excess of 178,000 patients 2 Jazz reports less than 13,000 patients currently on treatment Micropump sodium oxybate could benefit from the significant number of untreated patients Limited competition to date Jazz is expected to maintain dominant position in the narcolepsy market Micropump sodium oxybate is anticipated to benefit from an improved profile compared with Jazz s drug 1 Jazz s Q4 2014 financial results 2 GlobalData September 2015 15

LiquiTime Ibuprofen Treatment of Pain and Fever Extended-Release Liquid Ibuprofen Ibuprofen oral suspension twice-daily dosing confirmed First-in-Man (FIM) clinical study in healthy volunteers (15 subjects) Trial design: open-label, randomized, 3-way crossover with an immediaterelease ibuprofen control and 2 formulations of LiquiTime ibuprofen PK results announced in September 2014 demonstrated: Bioequivalence to immediate-release ibuprofen Similar onset versus immediate-release ibuprofen Similar blood levels at 12 hours versus immediate-release ibuprofen No safety or tolerability issues US regulatory filing expected in H1 2016 September 2015 16

LiquiTime Guaifenesin Treatment of Cough and Cold Extended-Release Liquid Guaifenesin Guaifenesin oral suspension twice-daily dosing confirmed First-In-Man (FIM) clinical study in healthy volunteers (16 subjects) Trial design: open label, randomized, 4-way crossover with an immediate release guaifenesin control dosed three times over 12 hours and three formulations of LiquiTime guaifenesin PK results announced in March 2015 demonstrated: A profile that is highly likely to succeed in a pivotal bioequivalence study Similar blood levels at 12 hours versus immediate-release guaifenesin No safety or tolerability issues US regulatory filing expected in H2 2016 September 2015 17

LiquiTime OTC Franchise Market Opportunity Cough and cold US market is estimated at $6.5b annually 1 OTC (Over-The-Counter) ibuprofen-containing products recorded sales in the USA beyond $490m including combination products 2 OTC guaifenesin-containing products recorded sales in the USA beyond $440m including combination products 2 These markets are dominated by OTC drugs, many of which are combination of different active ingredients having distinctive actions: Ibuprofen or Acetaminophen/paracetamol (analgesic and antipyretic) Chlorpheniramine or Diphenhydramine (antihistamine) Pseudoephedrine or Phenylephrine (decongestant) Dextromethorphan (antitussive) Guaifenesin (expectorant) LiquiTime allows for the combination of those active ingredients with potentially tailored release profiles for each of them 1 Deutsche Bank 2 IMS September 2015 18

Trigger Lock Hydromorphone Treatment of Pain Extended-Release Abuse-Resistant Opioid: Results and Market Opportunity Positive results from two pilot PK studies in healthy volunteers announced in June 2015 Bioequivalence criteria for area under the curve (AUC) and the peak plasma concentration (Cmax) were met under fasted conditions No notable difference seen in either AUC or Cmax in fed and fasted conditions (comparing the effect of food on the PK parameters) Abuse deterrence data generated in-house and being generated independently by a contract research organization (subject to further announcements) Flamel will meet with the FDA before the end of 2015 Abuse of painkillers is a major public health concern in the USA An estimated 2.1 million people in the US are suffering from substance use disorders related to prescription opioid pain relievers in 2012 Recent FDA policy is positive for anti-abuse platforms such as Trigger Lock Market opportunity: U.S. market for prescription painkillers (all forms) in 2014: $9.4b* OxyContin (extended-release oxycodone, Purdue): $2.5b* ER hydromorphone (Exalgo & generic, Mallinckrodt + generic, Allergan) $204m* 41 million opioid prescriptions for extended- and immediate-release oral opioid-containing products in the US in 2014* * IMS September 2015 19

Medusa Exenatide Treatment of Type 2 Diabetes Positive Preclinical Results and Market Opportunity Successfully tested in minipigs (June 2014) Significantly improved bioavailability versus Bydureon Two successive injections were administered with very similar release profiles No adverse clinical signs Excellent local tolerability PK profile is compatible with a release over one week in humans Interim phase I human clinical data to be reported in late 2015 Market opportunity: GLP-1 (glucagon-like peptide-1) products recorded US sales beyond ~$2.6b* in 2014, including $1.8b for Victoza (once a day liraglutide, Novo Nordisk) $458m for Bydureon (once-a-week exenatide, AstraZeneca) $320m for Byetta (twice-a-day exenatide, AstraZeneca) * IMS September 2015 20

Flamel s Strengths Diversified and proven drug delivery platforms Strong intellectual property Seasoned senior management Healthy financial situation September 2015 21

Diversified and Proven Drug Delivery Platforms 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 (already-marketed, off-patent or novel) Modified/Controlled Release of Solid Oral Drugs Modified/Controlled Release of Liquid Oral Drugs Modified/Controlled Release of Injectable Drugs Abuse-Resistant Modified/Controlled Release Narcotics/Opioid Analgesics Micropump, LiquiTime, Trigger Lock and Medusa are trademarks of Flamel Ireland Ltd. September 2015 22

Strong Intellectual Property Broad portfolio of patents* Date of expiration of granted patents Platform US Europe Micropump July 2027 July 2023 LiquiTime September 2025 April 2023 Trigger Lock April 2027 May 2026 (pending) Medusa June 2031 June 2027 (pending) New patents may be issued targeting each individual product in development where a Flamel drug delivery platform is applied to a specific molecule * 2014 Annual Report on form 20-F published on April 31st, 2015 September 2015 23

Seasoned Senior Management Name Title Appointed Experience Michael S. Anderson Chief Executive Officer 2012 40+ years Pharma Sandy Hatten Senior Vice President, Quality and Regulatory Affairs 2015 30+ years Pharma Phillandas T. Thompson, J.D., M.B.A. Senior Vice President, General Counsel 2013 16+ years Legal Siân Crouzet Principal Financial Officer 2005 17+ years Financial Gregory J. Davis Vice President, Corporate and Business Development 2015 20+ years Pharma David Monteith, Ph.D. Vice President, Research and Development 2014 25+ years Pharma Scott Macke Vice President, Supply Chain and Operations 2012 22+ years Pharma Jean Chatellier, Ph.D. Vice President, Alliance Management and Licensing 2010 15+ years Pharma Séverine Martin, E.M.B.A. Director, Human Resources and Corporate Projects 2014 13+ Human Resources September 2015 24

Condensed Consolidated Statement of Operations (Unaudited) In USD million, except EPS (USD) and shares data (million) Six months ended June 30, 2014 June 30, 2015 Total Revenue 8.9 82.5 Total Costs and Expenses (52.5) (73.5) Profit (loss) from continuing operations Net income (loss) from continuing operations (43.6) 9.0 (47.1) (5.8) Net income (loss) (47.7) (5.8) Diluted EPS from continuing operations (1.41) (0.14) Adjusted Diluted EPS (non-gaap) (0.29) 0.45 Diluted Shares Outstanding 33.4 40.3 September 2015 25

Condensed Balance Sheet (Unaudited) In USD million Cash & marketable securities As of Dec. 31, 2013 As of Dec. 31, 2014 As of March 31, 2015 As of June 30, 2015 7.0 92.8 113.2 116.1 LT debt 1 85.5 118.5 123.2 n/a Other LT liabilities 2 8.3 2.3 2.1 n/a 1 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 2013 2 Includes R&D tax credit financing, funding from former partner GSK, provision for retirement indemnity and employee service award provision September 2015 26

Flamel Technologies Transformed Fully integrated global specialty pharmaceutical company with FDA approved products and a broad pipeline using Flamel s four proprietary drug delivery platforms Two FDA approved products marketed by Flamel in the United States will drive significant revenue growth in 2015 Incremental clinical data for several of Flamel s pipeline products over the next 12-18 months Cash flow positive as of Q4 2014 and will be cash flow positive in 2015 and beyond (six months 2015 adjusted net income non GAAP = $18.3m) Net cash position of over $116m on June 30, 2015 provides for a strong balance sheet New strategy: Flamel controls 100% of its drug development September 2015 27

Headquarters 33 avenue du Dr. Georges Levy 69200 Vénissieux (Lyon) France Flamel Technologies SA (NASDAQ: FLML) is a specialty pharmaceutical company utilizing its core competencies in formulation development and drug delivery to develop safer and more efficacious pharmaceutical products, addressing unmet medical needs and/or reducing overall healthcare costs. Flamel currently has approvals for and markets two previously Unapproved Marketed Drugs ( UMDs ) in the USA, Bloxiverz (neostigmine methylsulfate injection) and Vazculep (phenylephrine hydrochloride injection). The Company intends to add to this branded business by creating additional products, focusing on the development of products utilizing Flamel s proprietary drug delivery platforms. Flamel currently has several products in development utilizing Micropump (oral sustained release microparticles platform) along with its tangent technologies, LiquiTime and Trigger Lock. The lead project for Micropump is sodium oxybate. LiquiTime allows for the extended-release of liquid medicines (such as ibuprofen and guaifenesin) and Trigger Lock is an abuse-resistant iteration of Micropump, designed specifically for long-acting opioids (such as hydromorphone). Additionally, the Company has developed a long acting injectable platform, Medusa, a nanogel depot technology currently being studied with exenatide. Flamel s products are targeting high-value molecules and will utilize either the 505(b)(2) approval process for NDAs or biosimilar pathways ultimately approved by FDA and other regulatory authorities. The Company is headquartered in Lyon, France and has operations in St. Louis, Missouri, USA, and Dublin, Ireland. Additional information can be found at www.flamel.com. Specialty Pharmaceutical Company with Proprietary Drug Delivery Platforms Focused on Improved or Cost-Effective Products Corporate Contact Phone: +33 472 783 434 Fax: +33 472 783 435 E-mail: licensing@flamel.com September 2015 28

Drug Delivery Platforms at a Glance September 2015 29

Micropump Platform at a Glance Extended/delayed release of drugs best absorbed in the small intestine (75% of all small molecules) Precise pharmacokinetics of single or combination of drugs in various formats Numerous Micropump -based products successfully tested in human clinical trials Commercial stage platform approved in the USA and EU Various dosage forms (tablet, capsule, sachet, liquid) Widely used and accepted excipients Combination of multiple release profiles and/or multiple active ingredients Rapid development time Taste-masking properties Strong IP position Cost effective and easy to scale-up September 2015 30

Micropump Microparticles for Controlled/Modified Release Granules drug granulate or layered neutral core Microparticles are dispersed in the stomach and pass into the small intestine, where each microparticle releases the drug at an adjustable rate and over an extended period of time (up to 24 hours) Drug released at an adjustable rate controlled and/or delayed Micropump microparticles can be used separately or together to provide highly specialized delivery profiles September 2015 31

LiquiTime Platform at a Glance LiquiTime is a novel, proprietary and innovative delivery platform allowing the stable Liquid and controlled release formulation of one or several combined drugs over Time LiquiTime meets challenges faced in the treatment of pediatric and geriatric patients and patient populations who have difficulty swallowing tablets or capsules, and may provide better patient compliance This graph illustrates the different near zero-order release profiles which can be tailored for the same drug LiquiTime s versatility allows once- or twice-daily liquid formulations of a wide variety of drugs September 2015 32

LiquiTime for Extended Release Liquid Suspension The liquid suspension contains small coated drug microparticles A dose typically contains 5,000 to 50,000 particles ER microparticles are suspended in the liquid medium Granules drug granulate or layered neutral core Coating controls diffusion keeps its integrity offers good resistance to stress 150-500 µm Each microparticle is individually coated and behaves as an independent micro reservoir September 2015 33

Trigger Lock Platform at a Glance Trigger Lock is a proprietary and innovative delivery platform that enables the controlled release of narcotic and opioid analgesics while deterring their abuse Trigger Lock successfully addresses the issues of narcotic/opioid analgesics tampering: The sustained release Micropump -based microparticles are resistant to crushing: each microparticle retains its polymer coating which is virtually impervious to further crushing Trigger Lock resists extraction attempts (even in boiling liquids) with beverages (alcoholic or not) preventing injection Trigger Lock preserves the bioavailability of the narcotic/opioid analgesics Trigger Lock is compatible with different dosage forms (capsules, tablets) September 2015 34

Trigger Lock SR Microparticles for Abuse Resistance 1. Drug loaded Micropump microparticles Sustained Release (SR) microparticles which are resistant to crushing 2. Viscosifying ingredient(s) To prevent abuse by injection after extraction in a small volume of solvent 3. Quenching ingredient(s) To prevent extraction in large volumes of liquid Each microparticle retains its polymer coating which is virtually impervious to further crushing September 2015 35

Medusa Platform at a Glance Applicable to a wide range of small molecules, peptide and protein drugs Solubilization and stabilization of drugs Safe, non-immunogenic and fully biodegradable Combination of several different drugs in the same formulation Medusa Nanogel Depot Sustained delivery from 1 to 7 days in human Bio-friendly, water-based, solvent-free formulation process Strong IP position September 2015 36

Medusa Nanogel Depot For Injection Made of polyglutamic acid and Vitamin E Amphiphilic and spontaneously forms stable nanoparticles in water Complexes are stable over a wide range of ph COO - Na + COO - Na + COO - Na + COO - Na + COO - Na + COO - Na + COO - Na + Polyglutamate chain Vitamin E Water clear liquid Solution Or Freeze-dried * * Injection * Drug solution or powder 1 2 3 Formulation by simple mixing in water Non-covalent association (reversible hydrophobic and/or electrostatic interaction) of the drug with Medusa nanogel In vivo depot formation Sustained release of the unmodified drug over 1 to 7 days In Vitro In Vivo September 2015 37