Cowen & Co. Annual Health Care Conference Camurus. Advancing late stage pipeline with high market potential. Company Presentation, 7 March 2017

Transcription

1 Cowen & Co. Annual Health Care Conference 2017 Camurus Advancing late stage pipeline with high market potential Company Presentation, 7 March 2017

2 Forward-looking statements This presentation contains forward-looking statements that provide our expectations or forecasts of future events such as new product developments and regulatory approvals and financial performance. Camurus is providing the following cautionary statement. Such forward-looking statements are subject to risks, uncertainties and inaccurate assumptions. This may cause actual results to differ materially from expectations and it may cause any or all of our forward-looking statements here or in other publications to be wrong. Factors that may affect future results include currency exchange rate fluctuations, delay or failure of development projects, loss or expiry of patents, production problems, unexpected contract, patent, breaches or terminations, government-mandated or market-driven price decreases, introduction of competing products, Camurus ability to successfully market products, exposure to product liability claims and other lawsuits, changes in reimbursement rules and governmental laws and interpretation thereof, and unexpected cost increases. Camurus undertakes no obligation to update forward-looking statements 2

3 Camurus overview Innovation that delivers Proprietary FluidCrystal technology used in 10 clinical programs Preclinical to NDA/MAA enabling pipeline programs Expansive and rewarding pharma partnerships Emerging European commercial organization Superior products for unmet markets Long-acting medications for better treatment outcomes and quality of life for patients Initial focus on opioid dependence and chronic pain Lean and experienced team 64 employees with 45 in R&D Headquarter in Lund, Sweden LISTED ON NASDAQ STOCKHOLM 3 rd DEC, 2015 MARKET CAP ~450 million USD CASH POSITION 55 million USD END

4 Late-stage, diversified pipeline PARTNERS PRODUCT PRECLINICAL PHASE 1/2 PHASE 3 REGISTRATION CAM2038 Weekly Opioid dependence CAM2038 Monthly Opioid dependence CAM2038 Weekly Chronic pain CAM2038 Monthly Chronic pain CAM2029 Neuroendocrine tumours CAM2029 Acromegaly CAM2032 Prostate cancer CAM4071 Not disclosed CAM2047 CINV* CAM2048 Pain CAM2058 Pain, nausea and vomiting *CINV Chemotherapy nausea and vomiting 4

5 Financials Scope Field Strong collaborations with strategic partners CAM2038, CAM2048, CAM2058 CAM2029, CAM other products CAM4072 Opioid dependence and pain Acromegaly, neuroendocrine tumours and other indications Genetic obesity Exclusive license agreement for North America, with option to China, Japan, Korea, and Taiwan Exclusive, worldwide, collaboration and license agreement Exclusive license to FluidCrystal Injection depot for setmelanotide USD 20 million in upfront license fee received + USD 130 million in total potential development and sales milestones Mid double digit % royalties on sales USD 50 million received in upfront, option exercise and development milestones USD 700 million in total potential development and sales milestones Mid to high single digit % royalties on sales USD 65 million in potential development and sales milestones Mid to mid-high single digit % royalties on sales New Hope in The Search for Treatment for Obesity, WSJ, August 26,

6 R&D highlights from 2016 PRODUCT EVENT TIME CAM2038 Opioid dependence Positive results from pivotal Phase 3 efficacy trial Positive results from Phase 2 opioid challenge trial Completed enrollment in Phase 3 long-term safety trial November May April CAM2038 Chronic pain First patient enrolled in Phase 3 efficacy trial Completed enrollment in pivotal Phase 2 chronic pain trial September April CAM2029 Acromegaly & NET* Positive results from Phase 2 trial in acromegaly and NET July CAM2032 Prostate cancer Positive results from Phase 2 trial in prostate cancer June CAM4071 Not disclosed indication Phase 1 clinical trial completed June CAM2047 CINV** CAM2048 Pain CAM2058 Pain & nausea and vomiting Phase 1 trial started October *NET: Neuroendocrine tumours **CINV: Chemotherapy induced nausea and vomiting 6

7 FluidCrystal technology

8 Leader in lipid science and formulation technologies FluidCrystal injection depot The FluidCrystal technology is based on functional liquid crystal nanostructures +400 PATENTS & APPLICATIONS lipid 1 L 2 I 2 17 CLINICAL TRIALS WITH FC TECHNOLOGY 1 FluidCrystal topical bioadhesive H 2 COMMERCIAL PRODUCT L a water lipid 2 FluidCrystal nanoparticles 8

9 FluidCrystal injection depot for easy and convenient dosing ~1500 SUBJECTS HAVE RECEIVED >10,000 INJECTIONS IN CLINICAL TRIALS Easy and convenient administration of long-acting injectables Good safety and local tolerability Applicable across drug substance classes Manufacturing by standard processes 9

10 Plasma BPN concentration (ng/ml) Clinical performance of FluidCrystal injection depot Weekly or monthly FluidCrystal depot provides buprenorphine exposure within the range of current sublingual products for daily dosing single dose and steady-state Time (days) FluidCrystal weekly depot (exemplified with buprenorphine clinical data) FluidCrystal monthly depot (single dose, exemplified with buprenorphine clinical data) FluidCrystal monthly depot (at steady state, simulated data) Comparator once daily product (exemplified with daily sublingual buprenorphine) 10

11 CAM2038: Weekly and Monthly Buprenorphine Injection Depots for Opioid Use Disorder Treatment

12 Opioid dependence is a growing global health problem EACH DAY Estimated 32 million opioid users globally 1 Largest society burden of all drugs 1 About 4 million diagnosed patients in Europe and the US Less than half in treatment (Europe and US 2,3 ) US overdose epidemic 4 Thousands 6,0 6,2 7,3 12,9 11,7 10,6 9,5 21,3 21,9 18,9 19,7 17,8 16,8 15,8 24,5 30,1 28,6 91 AMERICAN S DIE FROM OPIOID OVERDOSES Escalating opioid crisis 207,400 drug related deaths worldwide 30,000 US opioid overdose deaths in 2015 Growing concerns in Europe A chronic relapsing disease Opioid-use trajectories show that the disease is chronic with less than 30% abstinence achieved over time Opioid dependence mortality continues to increase over time 6 Opioid deaths in Sweden 5 Annual deaths with illicit drugs or opioid pharmaceutics present during forensic examination Heroin Non-heroin opioids Other drugs Source: 1. UNODC, World Drug Report 2015; 2. SAHMSA, National Survey on Drug Use and Health (NSDUH) 2014; 3. EMCDD, European Drug Report Trends and Developments 2015; 4. Center for Disease Control & Prevention 2016; 5. Toxreg 2016; 6. Hser et al. Harvard Review Psychiatry 2015; 23:

13 Medication assisted treatment considered best practice treatment for opioid use disorder Large evidence-base for efficacy Standard of care with daily buprenorphine or methadone medications Demonstrated effective in treating opioid dependence Suppresses withdrawal and reduces cravings Decreases illicit drug use Reduces risky behavior, spreading of blood-borne disease and mortality On the World Health Organization s Essential Medicines List Limited adherence to treatment Poor treatment retention with continued illicit opioid use / use on top Extensive misuse and diversion Safety issues Overdoses Important unmet medical needs Cardiac events (methadone) Need for daily, frequently supervised, dosing Inconvenient, stigma, and reduced quality of life Burdens to the healthcare and criminal justice systems No significant innovation in opioid use disorder treatment for the past 20 years 13

14 CAM2038 a paradigm shift in opioid dependence treatment Buprenorphine FluidCrystal injection depot CAM2038 key attributes Small volume, subcutaneous, liquid injection Encapsulating gel phase formed immediately after injection Long-acting buprenorphine Multiple doses of both weekly and monthly formulations allow flexible, individualized treatment Provided ready for use in a prefilled syringe Room temperature storage Extensive clinical documentation From 7 clinical trials (phase 1, phase 2, phase 3) Pharmacokinetics, pharmacodynamics, efficacy, and safety (4 studies including active controls) On track for NDA and MAA submissions mid-2017 FastTrack designation by FDA Long-acting release and continuous treatment effect Rapid and sustained suppression of withdrawal and blockade of opioid effects Safeguards against diversion, misuse and accidental pediatric exposure Reduced number of doses/decisions from 365 to 12 times per year No daily supervised dosing and related stigma Improved treatment adherence dose given is dose taken 14

15 Flexible and individualized opioid dependence treatment across treatment stages Weekly and monthly CAM2038 are administered as small dose volume subcutaneous (SC) injections by a prefilled syringe with a thin 23G needle Weekly CAM2038 Initiation - induction Weekly CAM2038 Stabilization Monthly or Weekly CAM2038 Maintenance treatment 15

16 Extensive clinical program for CAM2038 in opioid dependence Trial no. No. subjects Key results / Study design Status Extended release of BPN suited for once 60 healthy volunteers weekly dosing. Dose proportional exposure. 6-8 HS Phase 1 under naltrexone block times higher bioavailability versus SL BPN tablets. Good safety Extended release suited for weekly respective 87 healthy volunteers HS Phase 1 and local monthly dosing. 6-8 times higher bioavailability. under naltrexone block Acceptability of CAM2038 dosing higher than SL tablets. tolerability for CAM2038, weekly and Dose proportional extended release further monthly supported by pharmacodynamics results for HS Phase 2 41 patients formulations withdrawal symptoms over time and time to rescue medication. Randomized, placebo controlled opioid HS Phase 2 47 patients challenge study of CAM2038 in opioid dependent patients (US). 7 CLINICAL TRIALS COMPLETED AND UNDER COMPLETION +900 STUDY PARTICIPANTS DOSED WITH CAM2038 OR PLACEBO HS Phase 2 Repeat dose pharmacokinetic study of CAM2038 in opioid dependent pain patients (US), including injections in different subcutaneous injection sites. Positive results HS Phase 3 HS Phase 3 Double blind, double dummy Phase 3 efficacy trial of CAM2038 versus sublingual buprenorphine (US). N=428 Open label Phase 3 long-term safety trial in patients with opioid dependence (EU, US, AUS). N=228 Positive results Under completion 16

17 Peak Score (mm) CAM2038 provides rapid and sustained opioid blockade Pivotal Phase 2 study At this moment, my liking for this drug is Strong Liking 100 Qualification (Days 1-3) (Days 4-6) (Days 1-3) (Days 4-6) Weekly CAM2038 Injection 1 Weekly CAM2038 Injection 2 CAM2038 q1w 24 mg CAM2038 q1w 32 mg 70 Neutral pt. difference Prespecified complete blocking criteria 40 Strong Disliking Hydromorphone (mg, IM) Presentation, S27, ISAM & CSAM-SMCA 2016 Montreal, Canada Oct 20-22, 2016, Sharon L Walsh, Sandra Comer, Michelle Lofwall, Bradley Vince, Debra Kersh, Marion A Coe, Jermaine D Jones, Fredrik Tiberg, Behshad Sheldon, Sonnie Kim. 17

18 Total COWS Score CAM2038 also provides complete suppression of withdrawal Effective suppression of clinical opiate withdrawal scale (COWS) scores from treatment initiation Weekly CAM2038 Injection 1 Weekly CAM2038 Injection 2 CAM mg CAM mg Withdrawal COWS Score <5 Mild Moderate Moderate to Severe Severe > BL Treatment Days 18

19 Phase 3 efficacy study of weekly and monthly CAM2038 Study design 24-week, double-blind, double-dummy, randomized, active-controlled Phase 3 study Treatment-seeking patients with moderate to severe opioid use disorder, not currently on medication assisted treatment (MAT) Assessed from treatment initiation to maintenance Non-inferiority and superiority objectives CAM2038 vs. sublingual buprenorphine/naloxone BPN/NX Standard of Care Key Primary and Secondary Outcomes Primary Endpoint EMA: Negative Urines with NI margin of -11% FDA: Response Rate* with NI margin of -10% Key Secondary Endpoint: EMA: Cumulative Distribution Function (CDF) of urines negative for illicit opioids from Week 5 to 25. (Sampling Weeks) FDA: CDF of negative urines plus self-reports negative for illicit opioids from Week 5 to 25 (Sampling Weeks). Multiple additional secondary and explorative endpoints: retention; craving, morning cravings, withdrawal (SOWS and COWS) * Responder defined as displaying negative urine samples, including self-reports, for; treatment week 12 and month 6, at least 2 of treatment weeks 9, 10 and 11 and 12, and at least 5 of the 6 samples obtained during treatment weeks 12 through

20 CAM2038 met both FDA and EMA primary endpoints of non-inferiority versus SL BPN/NX Standard of Care (ITT) CAM2038 vs SL BPN/NX 95%CI Non-inferiority (NI) EMA: Primary Efficacy Endpoint - Mean % Urine Samples Negative for Illicit Opioids NI margin 11% Non-Inferiority p-value (-0.1%, 13.3%) <0.001 FDA: Primary Efficacy Endpoint - Response Rate NI margin 10% (-3.5%, 10.4%) < ,2-0,1 0 0,1 0,2 Favors SL BPN/NX Favors CAM2038 Difference (95% CI) 20

21 Statistical superiority met for CAM2038 versus SL/BPN/NX for secondary endpoint of CDF for negative urines +/- self-reports throughout the study (ITT) Grace Period Weeks Treatment Weeks Sampling Weeks Superiority p-value (FDA) Superiority p-value (EMA) a b c a Secondary endpoint for FDA and EMA; b Primary endpoint in RB-US ; c Including 2-week open label phase in RB-US CDF cumulative distribution function 21

22 CAM2038 demonstrated non-inferior and superior efficacy across treatment phases versus daily SL BPN/NX Robust efficacy versus Standard of Care Met both FDA and EMA primary endpoints Statistical superiority for the key secondary endpoint of CDF for negative urines Efficacy throughout treatment phases From initiation to maintenance CAM2038 safety profile comparable to daily sublingual buprenorphine/naloxone Fewer SAEs (3.2% vs. 6%) No drug overdoses vs 4 overdoses Good local tolerability; no severe AEs MAA and NDA submissions mid-2017 MAA Market Authorisation Application 22

23 Global strategy for CAM2038 with build-up of own European commercial organization for opioid use disorder Rationale Estimated 32 million opioid users globally Specialist market in EU ~ patients in treatment CAM2038 addresses high unmet need Sizeable socio-economic benefits 1.3m opioid problem users 2.5m diagnosed opioid dependent 1.3m registered heroin users On-going paradigm shift Accessible and concentrated market 187k opioid dependent Cost efficient roll-out and creation of significant value Braeburn markets Camurus markets Braeburn option right Transformative new treatment can support pricing strategy and reimbursement on European markets 23

24 Significant interest in CAM2038 among European physicians Country Physicians willingness to prescribe CAM % patients*: q4w q1w N=51 N=50 86% 94% 31% 30% 36% 25% 700k PATIENTS IN TREATMENT IN EUROPE N=50 86% 27% 43% N= 48 N=50 96% 22% 39% N= 47 Large markets with high willingness to prescribe weekly and monthly depots Source. 1. Market access dynamics in opioid addiction, Decision Resources 2015 * % patients prescribers thought would be prescribed CAM2038 of those currently prescribed medication 24

25 Building the commercial organization in Europe 2016 EU commercial leadership team in place GMs in early reimbursed markets Pricing, market access, medical affairs 2017 Regional leadership teams early reimbursed markets GMs 2 nd wave markets 2018 Regional leadership teams 2 nd wave markets Full key account teams for CAM2038 launch Internationally experienced leadership team Pre-launch activities Specialist pharma leadership Market access Medical affairs Global strategy Opioid use disorder & pain Stepwise build right time, right place principles HEOR, pricing and market access Strategic marketing Medical affairs Policy and education Country operating models 25

26 CAM2029: Next generation subcutaneous octreotide depot for treatment of NET and acromegaly

27 CAM2029 for treatment of acromegaly and neuroendocrine tumors Overview of acromegaly and NET Strong market growth over 15 years 1 Acromegaly is a rare, chronic and insidious hormonal disorder Occurs when the pituitary gland produces excess growth hormone (GH) and insulin-like growth factor-1 (IGF-1) Current gold-standard medical treatment include somatostatin analogues Neuroendocrine tumors (NETs) are malignant neoplasms Somatostatin analogues constitute the current standard of safe and effective medical therapy for symptom control Somatostatin analogues also show anti-tumor effects 339 (USDm) CAGR : Somatuline : 16% Sandostatin : 7% Sandostatin (Novartis) Somatuline (Ipsen) Significant potential in converting Sandostatin LAR patients to CAM2029 Source1. Medtrack 27

28 CAM2029 is a convenient, safe and effective treatment option CAM2029 overview CAM2029 key attributes Ready-to-use, long-acting octreotide for treatment of acromegaly and neuroendocrine tumors (NETs) Exclusive partnership with Novartis "Novartis Oncology continues its commitment to developing a new formulation of octreotide through a collaboration with Camurus Positive Phase 2 results announced in July 2016 Well maintained control of disease symptoms and biomarkers in NET and acromegaly patient when switching from Sandostatin LAR Good safety and local tolerability Phase 3 studies planned to start in 2017 Easy subcutaneous administration using prefilled syringe Self-administration option with significant convenience benefits and cost savings Increased bioavailability (500%) with potential for enhanced treatment efficacy 1 Thin needle and small injection volume Room temperature stability avoiding cold chain distribution and conditioning before use 1. Novartis Q2 and H1 Condensed Interim Financial Report 28

29 Pipeline expansion with new attractive preclinical candidates PARTNERS PRODUCT STATUS PRECLIN PH 1/2 PH 3 REG CAM4072 Genetic obesity CAM2043 PAH CAM2046 Diabetes Early stage collaborations with pharma and biotech partners Rhythm preparing to enter clinical development in 2017 Phase 1 planned to start in 2017 Formulation development Formulation development 29

30 CAM2043 is a novel sustained release treprostinil under development for treatment of pulmonary arterial hypertension Pulmonary arterial hypertension (PAH) is a progressive, life-threatening disease Orphan indication of cases per million Fewer than 200 treatment centers in the US PAH market exceeded 4 billion USD with aggregated treprostinil product sales of about 1.1 billion in 2015 Remodulin (United Therapeutics) sales of ~$572 million in 2015, ~12% CAGR 1 Limitations of current parenteral treatments Infusion site pain in 85% of patients Treatment limiting in 8% of patients Infections and sepsis relating to administration by continuous infusion Need for extra-corporal pump device limiting convenience and quality of life Requiring programming, drug dilution and filling with associated complications in 28% of patients Pump not water resistant Includes meta-cresol preservative Source: 1. PharmaCircle 30

31 CAM2043 has multiple potential treatment benefits versus parenteral infusion for PAH patients Convenient SC depot Weekly target dosing Individualized treatment Steady exposure levels adjustable by dose Within dose range of current products Maintain efficacy of parenteral infusion products Potential for improved local tolerability Less injection site pain Reduced risks of infections and sepsis No need for meta-cresol preservative CAM2043 pharmacokinetic profile Plasma TPN conc (ng/ml) 100 treprostinil SC 22.5mg treprostinil SC 30 mg , Time (hours) Source: Company data (dog PK) 31

32 Significant clinical news flow expected during 2017 PARTNERS PRODUCT EVENT TIME CAM2038 Opioid dependence CAM2038 Chronic pain Phase 3 results, long-term safety study NDA and MAA submissions Phase 2 results Phase 3 efficacy results Q2, 2017 Mid-2017 Q2, 2017 H2, 2017 CAM2029 Acromegaly & NET Phase 3 start 2017 CAM2047 CINV Phase 1 results Q2, 2017 CAM2048 Pain CAM2058 Pain, nausea and vomiting Phase 1 study results Phase 1 study results Phase 2 study start Q2, 2017 CAM2043 PAH Phase 1 study start H

33 Summary De-risked, late-stage, differentiated pipeline Opioid addiction, pain, cancer and acromegaly Successful Phase 3 study results in addiction Attractive multi-billion dollar specialty pharmaceutical markets Concentrated prescriber audiences and active patient advocacy groups Strong collaborations with dedicated partners Novartis, Braeburn Pharmaceuticals, Rhythm, Solasia, R-Pharm US Early project collaborations with global pharma companies Several levers for pipeline and business expansion New product candidates entering clinical development Growth opportunities via proven technology platforms as well as strong business development European commercial organisation Leadership team established Pre-launch activities for CAM2038 ongoing Solid financial position 33

34 Camurus AB, Ideon Science Park, SE Lund, Sweden

35 Solid financial position with increasing investments in late-stage development programs Key figures, musd Q4 Q1-Q4 Q4 Q1-Q4 Revenues Operating result ,5-3.3 Cash Total assets Revenues Operating expenses License payments Milestone payments Net sales; services and products Equity Research & dev Sales & marketing Administration 35