ASARINA PHARMA Remain in control of your life Corporate presentation JPM 2020 1
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 ”believe”, ”assume”, ”expert” or similar expressions. Such forward-looking statements are subject to known and unknown risks, uncertainties and other factors which may result in a substantial divergence between the actual results, financial situation, development or performance of Asarina and those explicitly or implicitly presumed in these statements Against the background of these uncertainties readers should not rely on • forward-looking statements Asarina assumes no responsibility to update forward-looking statements or to • adapt them to future events or developments 2
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 therapy for PMDD – Novel therapy with unique Mode of Action • a highly underserved indication • Substantial unmet medical need: Disabling condition affecting 4-5 % of women in fertile age Phase IIb randomiza?on finalized dec 2019. Phase IIb study with 14 centers in UK, Poland, Germany and Sweden • recruiting 206 patients completed randomization December 2019 Topline results April 2020 Phase IIa Proof of concept study in 80-90 women with Menstrual • Menstrual Migraine: Migraine in 7 centers in Sweden and Finland mid-term significant value inflection point > 50 % of subjects enrolled after 4 months recruitment Strong Pre-clinical efficacy data on par with antipsychotics but without • Tourette syndrome side effects published in Journal of Neuroendocrinology May 2019 An Orphan opportunity Phase IIa study with 20 subjects to start at Danish Tourette center Q3 2020 Potential PMDD/MM annual peak sales: > USD 2.000 mio worldwide • Significant commercial potential – Potential Orphan Tourette annual peak sales: > USD 1.000 mio worldwide • total peak sales > USD 3 billion Building a Scandinavian franchise in women’s health/neurology • 3
The Asarina team Peter Nordkild Karin Ekberg Jakob Dynnes CEO COO Hansen CFO PhD, clinical MD MSc, MBA physiology Novo Nordisk Novo Nordisk Creafve Pepfdes Ferring, Egalet, Zealand Pharma Umecrine Cognifon Pharmexa Evolva, Nordea Sven Göthe Märta Segerdahl Otto Skolling CMC CMO CBO MSc MD, PhD PhD Pharmacia & Upjohn Pharmacia & Upjohn Astra Zeneca Siemens Medical Lundbeck Kabi Fresenuis Novozymes Karolinska Development 4
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 Fonder (Sweden) 5.1% Länsförsäkringar (Sweden) 4.9% Handelsbanken Fonder (Sweden) 3.3% PEG Capital (Sweden) 2.6% CEO & Founder 3.1% Others (incl. 660 private shareholders) 16,8% Total 100.0% 5
Pipeline Asarina Pharma 2020 2021 2022 2023 2024 PMDD PMDD PMDD Phase III US & EU Regulatory Phase IIb MM Menstrual Migraine Mentrual Migraine Phase III Phase IIa Phase IIb Tourette Tourette Syndrome Preclinical Phase IIa Oral Lead UC2016 Oral Lead UC2016 Preclinical Phase I Preclinical Sepranolone Feasibility Sepranolone Bio Eq. Sepranolone Phase I New administration form New administration form New adm. form 6
Sepranolone normalises GABA A -receptor activity, targeting underlying cause of PMDD Extrasynaptic receptors PMDD patients have increased sensitivity to contain a 𝜀 subunit GABA A steroid allopregnanolone (ALLO), PAM a Cl - GABA b which is elevated during the premenstrual (luteal) phase of the menstrual cycle Extrasynaptic GABA A receptors Novel PAM Sepranolone inhibits the Positive Allosteric binding site Modulation (PAM) effect of ALLO on the Increased tonic GABAergic current GABA A receptor through Postsynapticterminal • Fine tuned receptor activity without overstimulation • High selectivity • Minimal off-target effects 7
Sepranolone in Premenstrual Dysphoric Disorder Remain in control of your life 8
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 concentrating • Occurs only during the late luteal phase of the menstrual cycle • Symptoms are present one to two weeks before menses and disappear within a few days after onset of menstruation • More than a third of PMDD women have suicidal thoughts and are Anxiety/Depression 4 times more likely to attempt suicide • Interferes with work, social activities and relationships • Refractory patients undergo treatment with GnRH agonists or hysterectomy and oophorectomy to eliminate PMDD symptoms Bloa?ng * Diagnostic and statistical manual of Mental Disorders 9
No current drugs directly target the underlying mechanism of PMDD 1,2,3 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 Black Box Warning 46% discontinued in 6 Require hormonal months due to side add-back effects Approved U.S. U.S. U.S. Sepranolone Initial formulary placement: 2nd line therapy Current 1 st line therapies only moderately effective 1. Nevatte T., et al. Arch Women Ment Health. 2003: online at DOI 10.1007/s00737-013-0346-y 2. Yonkers K.et.al. Ob&Gyn 2005;106(3):492. 3. Yaz Full Prescribing Information 10
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 in total premenstrual Placebo n=36 symptom score Active n=70 (p=0.041) compared to placebo Highly stafsfcally significant reducfon in pre-menstrual symptom score in “treated as intended” populafon (p = 0.006) n=26 n=34 Placebo Sepranolone *Total symptom score of 11 symptoms 11
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 injections every other day) Primary Endpoint • Change in premenstrual symptom severity questionnaire (DRSP) range before and during three Sepranolone treatment cycles dose 10 mg Secondary Endpoints • Safety PMDD Screen Randomize Sepranolone (DSM-5) verified • Responder analysis N= ~206 Multicenter dose 16 mg in at least two (Double-blind ) D, UK, PL, S menstrual cycles e-PRO •DRSP according to DSM-5 as Placebo diagnostic screener for PMDD Overwhelming interest/very low drop out rate of < 15% 12
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 completed web-screener on the page 7,514 women chose to register on ClinLife study page ~10% final contact with site for telephone screen ~470 has signed informed consent 206 randomised patients 13
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 producfon and autoinjector • for Phase III are ongoing. Esfmated start H2 2021, approximately 2 years study durafon. 14
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 PMDD according to DSM- • 5 – the Daily Record of Severity of Problems ( DRSP) score – the same as in Phase IIb The study will consist of a baseline/ diagnostic phase of 2 menstrual cycles, followed by • a double-blind treatment of 3 menstrual cycles, then followed by an open label extension phase of 3 menstrual cycles, and a safety follow-up cycle. Treatment will consist of self-administered s.c. injections of Sepranolone every 48 hours during the woman’s luteal phase, starting on Day -14, totalling up to 7 injections 15
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