Pioneering Ion Channel Development For The Treatments of Rare Diseases - Corporate presentation March 2020 - Saniona

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Pioneering Ion Channel Development For The Treatments of Rare Diseases - Corporate presentation March 2020 - Saniona
Pioneering Ion Channel Development For
The Treatments of Rare Diseases
Corporate presentation
March 2020
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Pioneering Ion Channel Development For The Treatments of Rare Diseases - Corporate presentation March 2020 - Saniona
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.

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Pioneering Ion Channel Development For The Treatments of Rare Diseases - Corporate presentation March 2020 - Saniona
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.

               Anita Milland
               Interim CFO & Head of IR
               Previously, VP Finance & Administration of Saniona A/S. CFO of NeuroSearch A/S.
               Member of the board of directors NeuroSearch Sweden AB, Poseidon
               Pharmaceuticals A/S and NsExplorer A/S.

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Pioneering Ion Channel Development For The Treatments of Rare Diseases - Corporate presentation March 2020 - Saniona
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
     Hypothalamic Obesity (HO)                           HO: Phase 2 results available in Q2, 2020
                                                         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

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Pioneering Ion Channel Development For The Treatments of Rare Diseases - Corporate presentation March 2020 - Saniona
“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)

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Pioneering Ion Channel Development For The Treatments of Rare Diseases - Corporate presentation March 2020 - Saniona
Prader-Willi Syndrome, a Debilitating, Rare Genetic Disorder

     Patient Population
          Birth incidence: 1 in 15000
     Cause                                                                                         USA           Europe   Japan
                                                                                                  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 years1
                  Complications from hyperphagia
                  Obesity related comorbidities
          Intellectual disabilities, physical deficiencies,
          behavioral problems
          Significant burden on caregivers and families

     1Manzardo, 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
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Pioneering Ion Channel Development For The Treatments of Rare Diseases - Corporate presentation March 2020 - Saniona
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

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Pioneering Ion Channel Development For The Treatments of Rare Diseases - Corporate presentation March 2020 - Saniona
Tesomet: Phase 2a Study Design in Prader-Willi Syndrome

         Study Overview
            Two-center, randomized, double-blind, placebo controlled trial
            Upon completion of adult enrollment (n=9), adolescent patients enrolled (n=9)

                                    Double-blind                              Open-Label Extension 1   Open-Label Extension 2
                                                                                     (OLE 1)                  (OLE 2)

                 Tesomet (0.5 mg)
Adults

                     Placebo
                                           Adolescents

                                                         Tesomet (0.125 mg)

                                                                                  Tesomet (0.125 mg)       Tesomet (0.25 mg)
                                                              Placebo

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Pioneering Ion Channel Development For The Treatments of Rare Diseases - Corporate presentation March 2020 - Saniona
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=3                N=2

      N=6     N=6
                         N=5
                                        N=2

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Pioneering Ion Channel Development For The Treatments of Rare Diseases - Corporate presentation March 2020 - Saniona
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 (%)
                                                   4             0.5 mg                         3.6
                                                                                                       2.9
                                                                  (n=9)                                                                  Placebo
                                                   2                                                                 OLE-2
                                                                         0.4
                                                   0
                                                   -2                                             0.125 mg
                                                                                                    (n=9)              -2.6
                                                   -4
                                                                                                                 0.25 mg (n=3)
                                                   -6        -5.6                                                0.125 mg (n=1)

                                                                 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

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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

                                    Monthly change in body weight by                              Random Co-Efficient Analysis
                                    plasma level in individual patients                       PK versus % Monthly Weight Change
                                  7%
                                  6%
                                  5%
                                  4%
        % change in body weight

                                  3%
                                  2%
                                  1%                                                                               p=0.003 (intercept)
                                  0%
                                  -1%                                                         p=0.005 (slope)
                                  -2%
                                  -3%                                                                                                    Expected
                                  -4%
                                  -5%
                                                                                                                                         95% conf. limits
                                  -6%
                                  -7%               0.125 mg             0.25 mg                        0.125 mg         0.25 mg         95% pred. limits
                                        -   1   2   3   4    5   6   7    8    9   10 11 12
                                                        Plasma level (ng/ml)

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Conclusions of phase 2a results of Tesomet in Prader Willi Syndrome

   Study Design                       Main Efficacy Findings             Main Safety Findings

   - 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

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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

        2020                        2021                          2022
          Phase 2b

                                                                                  FDA filing
          Phase 3

                                                      Tesomet

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Hypothalamic Obesity (HO)

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Hypothalamic Obesity, an Acquired Eating Disorder

       Patient Population
             50% of patients acquire HO post                                                      USA                            Europe                       Japan
             craniopharyngioma1                                                               3,400 – 6,800                  5,500 – 11,000               1,300 – 2,600
             Prevalence ~1 in 50,000-100,0002
       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
       2Inferred 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

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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

16 |         *0. 50mg tesofensine + 50mg metroprolol
Unique Ion-Channel Drug Discovery Platform

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Proprietary Pipeline: Multiple Value Drivers

  Product                Indication          Preclinical   Phase 1   Phase 2a   Phase 2b    Next steps

  Tesomet                Prader-Willi
  tesofensine +                                                                            Ph2b start 2020
                         Syndrome
  metoprolol
  (monoamine
  reuptake inhibitor +
                         Hypothalamic                                                      Ph2a completion
  beta blocker)          Obesity                                                           Q2 2020

                         Rare
  SAN711
                         neuropathic                                                       Phase 1
  (GABA α3 PAM)
                         itching disorders

                         Rare
  SAN903                                                                                   Filing IND
                         inflammatory
  (IK channel blocker)
                         disorders

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Overview of Strategic Partnerships

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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
  CAD-1883                                                            Minority stake
                                                                        Royalties
                Ataxia

                                                                      Upfront: 5M €
   GABAa5       Schizophrenia                                        Milestones: 85M €
                                                                         Royalties

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Conclusions

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Expected Corporate Milestones

                                                               proprietary
                         2020                                      2021                     2022

               H1                    H2                  H1                   H2       H1              H2
       HO topline data      PWS phase 2b          HO phase 3          PWS phase 3              HO phase 3 topline
       (phase 2)            initiation            initiation          initiation               data
                            Tesomet PWS IND       PWS phase 2b                                 PWS phase 3 topline
                            filing                topline data                                 data

                                                                 partnered
                         2020                                      2021                     2022

               H1                    H2                  H1                   H2       H1              H2
                            Approval & launch                         Launch of
                            of Tesofensine in                         Tesofensine in
                            Mexico                                    Argentina
                            Tesofensine NDA
                            filing in Argentina

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Saniona Investment Highlights

  1.    Clinical stage Biopharmaceutical company focused on rare diseases with
        high unmet medical need

  2.    Late stage treatment in development for two rare eating disorders:
        Prader Willi Syndrome and Hypothalamic Obesity

  3.    Unique ion-channel drug discovery platform

   4.   Delivering additional value through strategic partnerships

   5.   An experienced executive leadership team with a combined experience of
        over 80 years in the industry both in the US and the EU

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