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 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|
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.
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
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
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
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
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|
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
8|
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
9|
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
10 |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)
11 |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
12 |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
13 |Hypothalamic Obesity (HO) 14 |
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
15 |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 metroprololUnique Ion-Channel Drug Discovery Platform 17 |
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
18 |Overview of Strategic Partnerships 19 |
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
20 |Conclusions 21 |
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
22 |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
23 |24 |
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