Company Presentation March 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 |
Investment highlights “Saniona has two drugs that work!” An advanced-stage pipeline in the drug tesofensine Tesofensine is progressing toward regulatory approval in the $250M obesity market in Mexico. Other markets are being evaluated Tesomet (a combination of tesofensine and metoprolol) has demonstrated dramatic reductions in craving for food and weight in the rare genetic eating disorder Prader Willi Syndrome and will be progressed to Phase 2b targeting a billion dollar market Tesomet is also being studied in patients with Hypothalamic Obesity, another rare eating disorder with limited treatment options representing a billion dollar market opportunity Discovered and is advancing four first in class ion channel modulators through early clinical and late preclinical development Two internally addressing large market segments as well as rare diseases with high medical need Two with partners for treatment of schizophrenia and ataxia respectively Continue to look for strategic partners where shareholder value can be accelerated Experienced management team with a globally recognized track record of developing product candidates for CNS related disorders and conditions 3 |
Proprietary Pipeline Near term news flow and value generation Product Indication Preclinical Phase 1 Phase 2 Milestone Tesomet Prader Willi Ph2b/3 start 2019/20 tesofensine + Syndrome metoprolol Hypothalamic (monoamine reuptake Ph2a readout Q1 20 inhibitor + beta blocker) obesity Neuropathic pain SAN711 Ph1 initiation Q3 19 Itching ( GABA α3 PAM) Candidate selection IK Program Inflammation, IBD Q2 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 |
Tesofensine – How does it work? 6 |
Tesomet = Tesofensine + “betablocker” 7 |
Tesofensine: 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 2018 2019 2020 2021 2022 Cofepris Mexico Commercialization Phase 3 review Argentina Argentina Commercialization NDA 8 | 8 |
Tesofensine: Successful Phase 3 Study in Mexico by Partner Medix Medix to file for registration in Mexico and Argentina Phase 3 Study Design All endpoints met: Randomized, double-blind, placebo controlled trial in Mexico Primary endpoint: percent change in bodyweight compared to baseline at 372 patients enrolled: 24 weeks - N=124: placebo - N=124: 0.25mg tesofensine Secondary endpoints include: - N=124: 0.50mg tesofensine - Proportions of patients achieving a weight loss of >5 and 10 percent, - 24 weeks treatment period and 12 respectively week follow up - Metabolic including glycemic - All patients prescribed an energy endpoints restricted diet of 1,500-2,000 kcal - Quality of life and physical activity of 20-40 minutes 9 | 9 |
Tesofensine and Tesomet are supported by a large safety database Tesofensine, the key active ingredient in Tesomet, is well studied and has been safe and well tolerated More than 1800 subjects treated to date. 1 Phase 3 study, N= 372 (248 tesofensine) 8 Phase 2 studies, 2 Phase 2 studies* N = 1310 (one ongoing), N=78 (1000 tesofensine) 18 Phase 1 studies, 3 Phase 1 studies* N = 391 (325 tesofensine) (one ongoing), N=105 10 | *Indications include Type 2 diabetes and Prader Willi syndrome
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 11 |
Tesomet: Go-2-Market opportunity in orphan indications Potential for market entry within 4 years >$4B opportunity Prader Willi Syndrome Hypothalamic Obesity Positive Phase 2a in PWS adults Phase 2 study initiated in March 2019 Phase 2a in adolescent patients ongoing Proof of principle from previous compound Life-threatening hyperphagia and obesity Life-threatening hyperphagia and obesity Prevalence: 1/40.000 Prevalence: 1/(50.000-100,000) 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 Syndrome label Hypothalamic FDA Phase 2a Phase 3 filing obesity 12 |
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) 13 |
Causes of death among 312 individuals with PWS Source: HHS Public Access, Merlin G. Butler et al Merlin G. Butler et al: “individuals with PWS show high rates of choking, accidents and GI -perforation presumably related to uncontrolled hyperphagia and food seeking behaviors contributing to about one third of all reported deaths and about one half of the deaths in childhood ” Category N Mean Age SD Respiratory Failure 94 24.6 16 Cardiac 50 32.1 14 GI 30 21.4 16 Infection 29 35.7 16 Obesity 22 30.7 12 Pulmonary Embolism 19 34.1 12 Choking 18 30.1 17 Accident 17 25.0 16 Renal Failure 7 34.2 11 Neurologic 6 18.0 21 Hypothermia 3 30.8 14 Drug Reaction 3 25.1 9 Cancer 4 39.7 27 14 |
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 Conclusion: Second part of Phase 2a study to be conducted at quarter of dose (0.125 mg per day) 15 |
Tesomet: Phase 2a Study in PWS adults Strong effect on hyperphagia and weight (0.5 mg per day) 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 16 |
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) 17 |
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