Pioneering Ion Chanel Development For The Treatments of Rare Diseases Corporate presentation 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 Executive Leadership Team Rami Levin, MBA President, CEO and Board Member Previously, President Sobi Inc., Vice President Marketing and EMD Serono Inc. Managing Director Merck Serono in Scandinavia, Global Marketing Director Merck Serono, Geneva, Switzerland Jørgen Drejer, Ph.D. CSO, Co-Founder and Board Member Board member of 2Curex AB Previously, Executive Vice President, Research Director and co-founder of NeuroSearch A/S, Department Head and Project Manager at Novo Nordisk. Thomas Feldthus, M.Sc., MBA CFO and Co-Founder Board member of Scandion Oncology A/S Previously, CFO and co-founder of Symphogen A/S, Investment manager of Novo A/S, Corporate Development manager of Novo Nordisk A/S 3 |
Saniona Investment Highlights Clinical stage Biopharmaceutical company focused Addressing unmet needs in rare CNS diseases on rare diseases with high unmet medical need Late stage treatment in development for two rare PWS: Will begin phase 2b in H2, 2020 eating disorders: Prader Willi Syndrome (PWS) and HO: Phase 2 results available in Q2, 2020 Hypothalamic Obesity (HO) SAN711 – For rare neuropathic itching disorders (e.g. brachioradial pruritus), entering phase 1 Unique ion-channel drug discovery platform SAN903 – For rare inflammatory and fibrotic disorders (e.g. idiopathic pulmonary fibrosis) Tesofensine for obesity Delivering additional value through strategic CAD-1883 for essential tremor and Ataxia partnerships GABAa5 for schizophrenia An experienced executive leadership team with a Experience in research, development and combined experience of over 80 years in the commercialization of rare disease drugs, both in industry both in the US and the EU the US and EU 4 |
“Weight gain, hyperphagia “Therefore, patients are and obsession with food are more available to other the greatest burden on both activities, and life as a whole patients with Prader-Willi becomes easier for the syndrome and their families. patients and their families. ” Tesomet helps to control Dóra Török weight and appetite and it Primary Investigator decreases preoccupation with food.” Prader-Willi Syndrome (PWS) 5 |
Prader-Willi Syndrome, a Debilitating, Rare Genetic Disorder Patient Population Birth incidence: 1 in 15000 USA Europe Japan Cause 8,000 13,000 3,300 Absence of paternally expressed genes at Chromosome 15 (q11-q13) Disease Characteristics Hyperphagia, insatiable hunger Short life expectancy, median 30-40 years 1 Complications from hyperphagia Obesity related comorbidities Intellectual disabilities, physical deficiencies, behavioral problems Significant burden on caregivers and families 1 Manzardo, A., Loker, J., Heinemann, J. et al. Survival trends from the Prader – Willi Syndrome Association (USA) 40-year mortality survey. Genet Med 20, 24 – 30 (2018) doi:10.1038/gim.2017.92 6 |
Tesomet - a true Triple Monoamine Uptake Inhibitor Controls Eating Tesomet Tesomet increases levels of monoamines by blocking re- uptake Reduces hyperphagia by controlling appetite and craving for food Increases metabolic rate Addresses significant unmet needs in PWS 7 |
Significant reduction in hyperphagia scores in phase 2a PWS study Adults: Tesomet 0.5 mg reduced the hyperphagia Adolescents: Hyperphagia is down to low score to zero in a double-blind study single digits at 0.25 mg per day during OLE 2 N=2 N=2 N=6 N=2 N=6 N=3 N=5 N=2 8 |
Significant reduction in body weight in phase 2a PWS study % change in body weight (adults & adolescents) 3 months 3 months 3 months 6 Tesomet change in weight (%) 3.6 4 0.5 mg 2.9 Placebo (n=9) 2 OLE-2 0.4 0 -2 0.125 mg (n=9) -2.6 -4 0.25 mg (n=3) -6 0.125 mg (n=1) -5.6 adults adolescents ANCOVA, Change from baseline to Day 91, LOCF Double blinded phases of the study: Extension phases of the study: The 0.5 mg in adults showed promising efficacy A weight reduction was seen in OLE 2 where patients were at the target plasma levels of tesofensine The 0.125 mg in adolescents did not show efficacy on primary endpoint • likely due to too low exposures of tesofensine 9 |
Significant correlation between change in body weight and plasma level All individual patients experienced dose-dependent monthly weight loss – highly statistically significant correlation between weight loss and plasma concentration Random Co-Efficient Analysis Monthly change in body weight by PK versus % Monthly Weight Change plasma level in individual patients 7% 6% 5% 4% % change in body weight 3% 2% p=0.003 (intercept) 1% 0% p=0.005 (slope) -1% -2% Expected -3% -4% 95% conf. limits -5% -6% 0.125 mg 0.125 mg 0.25 mg 95% pred. limits 0.25 mg -7% - 1 2 3 4 5 6 7 8 9 10 11 12 Plasma level (ng/ml) 10 |
Conclusions of phase 2a results of Tesomet in Prader Willi Syndrome Main Efficacy Findings Main Safety Findings Study Design - 18 patients dosed and followed - Significant reduction in - 0.125mg and 0.25mg doses up to 9 months hyperphagia score were safe and well tolerated - Three doses investigated: - 2.6% reduction in body weight - Higher than expected drop-out 0.125mg, 0.25mg and 0.5mg on the 0.25mg dose by the end of rates were observed at a dose of the study 0.5mg - Good correlation between efficacy, dose and plasma level 11 |
Pivotal PWS Clinical Program with Overlapping Program Design Study Overview 16-week dose finding study; 36-week open label extension with dose adjustment In total: about 150 patients over a 2-3-year period and a total investment of $30M 2020 2021 2022 Phase 2b FDA filing Phase 3 Tesomet 12 | 12 |
Hypothalamic Obesity (HO) 13 |
Hypothalamic Obesity, an Acquired Eating Disorder Patient Population 50% of patients acquire HO post USA Europe Japan craniopharyngioma 1 3,400 – 6,800 5,500 – 11,000 1,300 – 2,600 Prevalence ~1 in 50,000-100,000 2 Disease characteristics Post surgical obesity and hyperphagia, insatiable hunger Memory impairment, attention, impulse control Depression and suicide 1 Roth, C., Hypothalamic Obesity in Craniopharyngioma Patients, J. Clin. Med. 2015 , 4 (9), 1774-1797; https://doi.org/10.3390/jcm4091774 2 Inferred given prevalence of craniopharyngioma cited by Garnett, M.R., Puget, S., Grill, J. et al. Craniopharyngioma. Orphanet J Rare Dis 2, 18 (2007) doi:10.1186/1750-1172-2-18 14 |
Tesomet: Phase 2a Study in Hypothalamic Obesity Study Overview • Primary endpoint: safety • Secondary endpoints: Change in bodyweight compared to baseline at 24 weeks, appetite score, metabolic including glycemic endpoints, quality of life • Single centre, randomized, double-blind placebo controlled trial • 21 patients enrolled 2020 2021 2022 24-weeks double blind 24-weeks open label Placebo Placebo crossover Phase 2 Tesomet * FDA Phase 3 FDA Phase 3 filing 15 | 15 | *0. 50mg tesofensine + 50mg metroprolol
Unique Ion-Channel Drug Discovery Platform 16 |
Proprietary Pipeline: Multiple Value Drivers Product Indication Preclinical Phase 1 Phase 2a Phase 2b Next steps Tesomet Prader-Willi Ph2b start 2020 tesofensine + Syndrome metoprolol (monoamine Hypothalamic Ph2a completion reuptake inhibitor + Obesity Q2 2020 beta blocker) Rare SAN711 neuropathic Phase 1 ( GABA α3 PAM) itching disorders Rare Filing IND SAN903 inflammatory (IK channel blocker) disorders 17 | 17 | 17 |
Overview of Strategic Partnerships 18 |
Partnered Pipeline and Milestones Product Indication Preclinical Phase 1 Phase 2 Phase 3 Expected approval Tesofensine Obesity and launch in H2 2020 Essential tremor Spinout Minority stake CAD-1883 Royalties Ataxia Upfront: 5M € GABAa5 Schizophrenia Milestones: 85M € Royalties 19 | 19 | 19 |
Conclusions 20 |
Expected Corporate Milestones TIMING EVENT PARTNER EVENT H1 2020 • Tesomet HO: Topline data • • Approval/launch: tesofensine Mexico Tesomet PWS: Initiate phase 2b H2 2020 • • tesofensine NDA filing in Argentina Tesomet PWS: IND filing 21 |
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