Company Presentation June 2019 1 |
FORWARD LOOKING STATMENTS This presentation contains forward- looking statements that provide Saniona’s expectations or forecasts of future events such as new product developments, regulatory approvals and financial performance. Such forward looking statements are subject to risks, uncertainties and may be impacted by inaccurate assumptions. This may cause actual results to differ materially from expectations and it may cause any or all of Saniona’s 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, breaches or terminations of contracts, government-mandated or market driven price decreases, introduction of competing products, exposure to product liability claims and other lawsuits, changes in reimbursement rules, changes of laws regulations or interpretation thereof, and unexpected cost increases. Saniona undertakes no obligation to update forward looking statements. 2 | 2 |
Saniona – Investment highlights Treating diseases of the central nervous system and eating disorders Saniona is a leading late stage biotech company addressing diseases of the central nervous system (CNS) and eating disorders. The company has five projects in clinical development and several partnership agreements A world leading R&D team focusing on ion channels and a management team with excellent track record in drug development and from making license deals and partnership agreements with both Big Pharma and VCs A drug that works in obesity and two orphan drug indications with billion dollar potential Tesofensine has demonstrated positive Phase 3-data in obesity, the partner Medix is now planning to launch in Mexico, Saniona will receive milestones and royalties – a $250M market in Mexico alone Tesomet with promising Phase 2a-data in Prader Willi Syndrome (PWS), a rare genetic disorder resulting in a chronic feeling of extreme hunger (hyperphagia) – a $3bn market potential Tesomet in Phase 2a for hypothalamic obesity (HO) , an acquired eating disorder caused by damage to the appetite centre usually following surgical removal a brain tumour – a >$1bn market opportunity Exciting near term news flow with the potential to generate shareholder value Phase 2a data from the open label extension in PWS Phase 2a data in HO Market approval of Tesofensine in Mexico Inititation of Phase 1 with SAN711 for chronic pain and itching Boehringer Ingelheim initiates Phase 1 for Schizophrenia Saniona’s world class technology platform will continue to generate assets and early stage partnership opportunities 3 | 3 |
Proprietary Pipeline Near term news flow and value generation Product Indication Preclinical Phase 1 Phase 2 Milestone Tesomet Prader Willi Ph2a data Q1 19 tesofensine + Syndrome Ph2b/3 start 2019/20 metoprolol Hypothalamic (monoamine reuptake Ph2a start Q1 19 inhibitor + beta blocker) obesity Neuropathic pain SAN711 Ph1 ready Itching ( GABA α3 PAM) Candidate selection IK Program Inflammation, IBD H1 19 4 | 4 | 4 |
Partnered pipeline Near term news flow and non-dilutive cash Product Indication Preclinical Phase 1 Phase 2 Phase 3 Tesofensine Obesity Essential tremor Spinout Minority stake CAD-1883 Royalties Ataxia Upfront: 5M € Not Schizophrenia Milestones: 85M € disclosed Royalties NS2359 off patent; NS2359 Cocaine Addiction financed by US grants 5 | 5 | 5 |
Medix partnership With ~50% market share, Medix is market leader in the $250M Rx Obesity Market in Mexico Regional deal structure Medix holds the rights to tesofensine & Tesomet in Mexico & Argentina Medix finances clinical studies and commercialization Saniona receives double digit royalties Saniona retains rights to rest of the world including exclusive rights to Medix’ clinical data Medix could be on the market in Mexico in 2020 and in Argentina one year later Medix to launch product in Mexico and Argentina following successful completion of Phase 3 study in 20 Tesofensine well tolerated and archived 10% average weight loss in 6 month - better than any of the existing drugs on the market 2018 2019 2020 2021 2022 Cofepris Mexico Commercialization Phase 3 review Argentina Argentina Commercialization NDA 6 | 6 |
Tesomet: tesofensine + metoprolol Tesofensine, in preclinical models and clinical trials, has shown efficacy and safety • Reduction in food intake • Weight loss efficacy • Effects on glycemic parameters relevant for type 2 diabetes • Excellent safety and tolerability COMPOSITION TESOFENSINE METOPROLOL Beta blocker to control slight increase in heart rate Effective weight loss drug Tesomet = tesofensine + beta blocker (metroprolol) • Neutralizes slight heart rate increase observed with tesofensine • Allows for strong intellectual property protection through 2036 7 |
Tesomet: Go-2-Market opportunity in orphan indications Potential for market entry within 4 years – Total investment of $30-40M - >$4B opportunity Prader Willi Syndrome Hypothalamic Obesity Positive Phase 2a in PWS adults Phase 2 study preparations Phase 2a in adolescent patients ongoing Life-threatening hyperphagia and obesity Life-threatening hyperphagia and obesity Prevalence: 1/(50.000-100,000) Prevalence: 1/40.000 Estimated market size: >1B USD Estimated market size: ~3B USD 2018 2019 2020 2021 2022 Prader Willi Open FDA Phase 2a Phase 3 Phase 2b filing label Syndrome Hypothalamic FDA Phase 2a Phase 3 filing obesity 8 | 8 |
Tesomet - lead indication: Prader-Willi syndrome Genetic disease caused by mutations/deletion of genes on chromosome 15 Chronic feeling of extreme hunger (hyperphagia) no matter how much the patient eats Other symptoms and characteristics Mental retardation and behavioural problems Low metabolic rate (50% of normal) Sensitive to some medicines (½ dose prescribed) Medical need } Acute life-threating hyperphagia (choking, bowel rupture) Short life expectation (average in 30s) Life-threatening obesity No effective treatment available today Economic and social costs Quality of life for patients and families Family stress and loss of income Care and medical costs (USD 100-300K per year) 9 | 9 |
PWS opportunity has blockbuster potential Accessible market value equals 3 Billion USD (Analyst estimates) Premium pricing potential Orphan drug status will ensure premium pricing Majority of drugs with less that 10,000 patients in the US tend to be priced above 200K USD per year Large commercial opportunity No drugs approved for treating hyperphagia Low investment Clear endpoint with short studies (Phase 3: 100 patients / 6 months) Straightforward commercialization (most patients are managed by specialists in central centers) 10 |
Summary of Phase 2a studies in patients with PWS Proof-of-concept for Tesomet, which should be given at lower doses as is the case for many other drugs 3-months double blind Phase 2a study in adults with PWS provides proof-of-concept for Tesomet Tesomet provides strong reduction in hyperphagia and weight at optimal dose for obese patient of 0.5 mg/day 0.5 mg/day is too high in some PWS patients because of a different body composition and slow metabolism 3-months double blind study followed by two 3-months open label dose-finding studies in adolescents with PWS Tesomet at 0.125 mg/day is well tolerated but does not provide the intended blood concentration and efficacy Tesomet at 0.25 mg/day initiated in March – to be completed in July Tesomet appears to be well tolerated at 0.25 mg/day and first data point suggests that patients have stabilized in weight with a small weight loss recorded in some patients Conclusion Tesomet is a promising highly effective treatment option for control of hyperphagia and weight in PWS The results provide sufficient information to start a Phase 2b / Phase 3 program Many patients may have full benefit of 0.25 mg per day whereas the optimal dose may be between 0.25 mg and 0.5 mg per day for some patients Potential adaptive design in clinical studies with a starting dose of 0.25 mg per day 11 |
Pivotal PWS clinical program Potential high level overlapping Phase 2b/Phase 3clinical program design Randomisation Fixed dose Fixed dose Flexible dose Last dose from DB Part Phase 2 (pivotal trial #1) Screening Fixed dose – t.b.c. Placebo 1 2 3 4 6 7 8 9 10 11 12 5 Double-blind Part Open Label Randomisation Fixed OR flexible dose Fixed dose Phase 3 (pivotal trial #2) Screening Fixed or fdose – t.b.c. Placebo 1 2 3 4 5 6 7 8 9 10 11 12 Double-blind Part Open Label 12 |
Tesomet: Phase 2a Study in PWS adults Study set up Results from Phase 2a study in February 2018 (0.5 mg per day) Exploratory randomized, Positive effect on key PK and Safety double-blind, placebo- efficacy endpoints controlled 12 weeks study - No SAE in 9 patients - Reduced craving for - AE mainly CNS related food - Half-life longer than - Tesomet 6 - Weight loss expected in PWS patients - Placebo 3 13 |
Tesomet: Phase 2a Study in PWS adults Results PWS hyperphagia score (data show mean and SD) 20 PWS weight loss 15 Week 8 Week 13 Hyperphagia score Tesomet 5.00 % (n=5) 6.75 % (n=2) 10 Placebo 0.46 % (n=2) 0.75 % (n=2) 5 0 0 20 40 60 80 100 days of treatment placebo treatment 14 |
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