Asarina Pharma AB (publ) — JPM 2020
33 slides from Asarina Pharma AB (publ)'s conference presentation, January 2020
Slide 1: Asarina Pharma Corporate presentation JPM 2020
ASARINA PHARMA Remain in control of your life Corporate presentation JPM 2020 1
Slide 2: Disclaimer
Disclaimer • The shares of Asarina Pharma (”Asarina”) are traded on NASDAQ First North in Stockholm (ticker: ”ASAP”) • This presentation may contain specific forward-looking statements, relating to Asarina´s future business, development and economic performance e.g. statements including terms like ”
Slide 3: Asarina Pharma Overview
Asarina Pharma Overview • Phase IIb Premenstrual Dysphoric Disorder – topline results April 2020 Clinical mid-stage company with pipeline in • Phase IIa study in Menstrual Migraine with topline results Q4 2020 women’s health and neurology • Phase IIa study in Tourette to start Q3 2020 First-in-class
Slide 4: The Asarina team
The Asarina team Peter Nordkild Jakob Dynnes Karin Ekberg CEO Hansen COO CFO MD MSc, MBA PhD, clinical Novo Nordisk Novo Nordisk physiology Ferring, Egalet, Zealand Pharma Creafve Pepfdes Pharmexa Evolva, Nordea Umecrine Cognifon Märta Segerdahl Otto Skolling Sven Göthe CMO CBO CMC MD, PhD MSc PhD A
Slide 5: Major shareholders
Major shareholders +85% are institutional investors Kurma Biofund (France) 17.1% Östersjöstiftelsen (Sweden) 14.5% Idinvest Patrimonie (France) 8.9% Swedbank Robur Fonder (Sweden) 7.3% Fourth Swedish National pension fund 6.2% Rosetta Capital (UK) 5.8% Sectoral Asset Management (Canada) 5.4% Catella
Slide 6: Pipeline Asarina Pharma
Pipeline Asarina Pharma 2020 2021 2022 2023 2024 PMDD PMDD PMDD Phase IIb Phase III US & EU Regulatory Menstrual Migraine Mentrual Migraine MM Phase IIa Phase IIb Phase III Tourette Tourette Preclinical Syndrome Phase IIa Oral Lead UC2016 Oral Lead UC2016 Preclinical Phase I Feasibility Sepranolone
Slide 7: Sepranolone normalises GABAA-receptor activity, targeting underlying cause of PMDD
Sepranolone normalises GABAA-receptor activity, targeting underlying cause of PMDD Extrasynaptic receptors PMDD patients have increased sensitivity to contain a 𝛿 subunit GABAA steroid allopregnanolone (ALLO), a Cl- GABA PAM b which is elevated during the premenstrual (luteal) phase of the…
Slide 8: Sepranolone in Premenstrual Dysphoric Disorder
Sepranolone in Premenstrual Dysphoric Disorder Remain in control of your life 8
Slide 9: PMDD affects > 3.5 mio women in the US
PMDD affects > 3.5 mio women in the US • Defined by WHO in ICD-11 as a Gynecological disease • Diagnostic criteria established in DSM-5 * - Affective: Emotional lability, depressed mood, irritability, anxiety - Somatic: Lethargy, bloating, joint pain, hypersomnia Irritability - Cognitive: Difficulty
Slide 10: No current drugs directly target the underlying mechanism of PMDD
No current drugs directly target the underlying mechanism of PMDD1,2,3 SSRI Antidepressant Hormonal Therapy YAZ oral Agent Fluoxetine GnRH agonists contraceptive Efficacy Moderate (50-60%) Moderate High Often persistent in PMDD Suppress patients hormonal cycles Side Effects 46% discontinued in 6 Bla
Slide 11: Sepranolone meets primary (FDA) endpoint in phase IIa study
Sepranolone meets primary (FDA*) endpoint in phase IIa study • Double-blind, placebo controlled trial in 120 randomized patients • Patients received five doses over 10 days from ovulation • Two doses, 10mg and 16mg tested; pooled data below Statistically significant reduction Placebo n=36 in total p
Slide 12: Fully randomized phase IIb study with topline results in April 2020
Fully randomized phase IIb study with topline results in April 2020 Design • RCT, double-blinded, placebo- controlled, with two cycles of Baseline/Diagnosis 3 treatment cycles 1 month follow-up diagnosis, three treatment cycles and Two cycles a follow-up cycle. Treatment cycle will be for 14 days (7
Slide 13: Overwhelming patient interest
Overwhelming patient interest • All patients recruited via a media campaign through geotargeted advertisement Patient IC Randomisation “News” re PMDD Google Web Telephone Clinic DRSP PMDD & advertisements Ads screener screen visit 1 ePRO diagnosis 1,191,322 visits on study landing page 248,315 compl
Slide 14: PMDD
PMDD • Posifve data from Phase IIa study in 2015 • Phase IIb study reading out in April 2020 - All pafents now randomized - High treatment compliance - Low number of withdrawals • No safety signals, preclinically or clinically • Long-term tox starfng April 2020 • Preparafons including upscaling of p
Slide 15: PMDD Phase III Clinical Program
PMDD Phase III Clinical Program • Asarina Pharma plans for two pivotal studies, randomized, double-blind parallel groups, one dose level vs. placebo One in North America and one in EU/ ROW. The studies will each include ~500 patients • Population: Women age 18-45, fulfilling diagnostic criteria for
Slide 16: PMDD Phase III Clinical Program
PMDD Phase III Clinical Program • The women will rate their PMDD symptoms daily on the DRSP scale using a web based eDiary. Primary endpoint is reduction in the DRSP score during the treatment cycles, compared to baseline • Start of dosing in each cycle will be led by tracking of menstrual flow usin
Slide 17: Market opportunity PMDD
Market opportunity PMDD • Assumptions: > 4% of women suffer from PMDD (~10.600.000) > 25% or 2,5 mio in EU/US/Japan seek treatment > 50% are refractory to present treatment - Sepranolone market introduction end 2024 - Sepranolone market penetration of 20% at peak sales - 250.000 patients being treat
Slide 18: Sepranolone in menstrual migraine
Sepranolone in menstrual migraine Remain in control of your life 18
Slide 19: Menstrual migraine – Prophylaxis is the best cure
Menstrual migraine – Prophylaxis is the best cure • MM occurs from 2 days before to 3 days into menstruafon • MM is predictable but harder to treat and avacks are longer • MM pain does not respond well to state of the art migraine treatment with triptanes and NSAID´s • MM pafents seems to have livle
Slide 20: Migraine attacks during the menstrual cycle
Migraine attacks during the menstrual cycle Menstrual exacerbation of migraine occurs in ~ 50% of women with migraine MacGregor et al., NEUROLOGY 2006;67:2154–2158 Incidence of migraine, urinary estrone-3-glucuronide (E1G) and pregnanediol-3-glucuronide (PdG) levels on each day of the menstrual cycl
Slide 21: Changes in circulating neurosteroid levels are associated with migraine
Changes in circulating neurosteroid levels are associated with migraine 21
Slide 22: > 50% of patients enrolled in phase IIa Proof of Concept study
> 50 % of patients enrolled in phase IIa Proof of Concept study • The ongoing Phase IIa study is a randomized, double blind, parallel group study in women age 18-45 comparing two doses of Sepranolone and placebo. Esfmated top line results in Q4 2020 • The study consists of a diagnosfc/ baseline phas
Slide 23: Market opportunity Menstrual Migraine
Market opportunity Menstrual Migraine • Assumptions: > 6% of women suffer from Menstrual Migraine (~14.000.000) > 50% or 7 mio in EU/US/Japan seek treatment > 30% are refractory to present treatment - Sepranolone market introduction 2026 - Sepranolone market penetration of 10% at peak sales - 200.00
Slide 24: Sepranolone in Tourette Syndrome - a new opportunity
Sepranolone in Tourette Syndrome - a new opportunity Remain in control of your life 24
Slide 25: 2018 Impact Survey by US Tourette Association in 1.000 patients
2018 Impact Survey by US Tourette Association in 1.000 patients Children: - 63% felt discriminated against - 32% have considered suicide/self harming behavior - 40% were forced to miss school - 59% take prescription medications to manage TS - 29% have tried 5 or more different medications - 44% of p
Slide 26: Phase IIa study in Tourette Syndrome
Phase IIa study in Tourette Syndrome • In a mouse model of TS, Sepranolone reduces tic behavior similarly to Haloperidol and Finesteride • Asarina Pharma will in September 2020 initiate a randomized parallel group Phase IIa study in adolescent and adult patients with TS • The study will include 20 m
Slide 27: Dose dependent tic reduction with Sepranolone
Dose dependent tic reduction with Sepranolone Spatial confinement 1.50 Vehicle (SC) P<0.0001 P<0.00001 1.25 Sepranolone (5 mg/kg, SC) P<0.0001 # Tics/min 1.00 Sepranolone (10 mg/kg, SC) NS 0.75 NS 0.50 0.25 0.00 WT D1CT-7 Source of Variation % of total variation P value P value summary Significant?
Slide 28: Efficacy on par with Haldol and Finasteride
Efficacy on par with Haldol and Finasteride Spatial confinement 1.50 Vehicle (SC) P<0.0001 P<0.00001 1.25 Sepranolone (10 mg/kg, SC) P<0.00001 # Tics/min P<0.00001 Haloperidol (0.3 mg/kg, IP) 1.00 NS Finasteride (25 mg/kg, IP) 0.75 NS 0.50 NS 0.25 0.00 WT D1CT-7 Source of Variation % of total variat
Slide 29: Market opportunity Tourette Syndrome
Market opportunity Tourette Syndrome • Assumptions: - 600.000 TS patients in US/EU/Japan > 300.000 patients in US/EU/Japan treated with drugs - Sepranolone market introduction 2025 - Sepranolone market penetration of 10% at peak sales - 10% or 30.000 patients being treated with Sepranolone - Annual
Slide 30: Autoinjector – Convenient and easy administration
Autoinjector AUTO-INJECTO Convenient and easy administration Secondary Ypsomed (Ypsomate™) selected as Autoinjector • Compatible with Sepranolone syringe • Single, fixed dose • Good space • Automatic injection • Disposable • Several op • Provide needle shielding system • Secondary packaging 30
Slide 31: Value generation
Value generation Remain in control of your life 31
Slide 32: Value inflection points – external communication 2019-2020
Value inflection points – external communication 2019-2020 2019 2020 ü Last patient IND Menstrual first visit approval Migraine UM203 Sepranolone last patient PMDD PMDD first dose ü Oral proof of Top line Menstrual Top line results Migraine results concept in Menstrual study initiation PMDD animals
Slide 33: Asarina Pharma - summary
Asarina Pharma - summary ü First treatment to target Premenstrual Dysphoric Disorder and Menstrual Migraine with potential disease modifying effect targeting the origin of these diseases ü Significant unmet medical need in both indications with US market opportunity alone of > USD 1 billion and a si