ASARINA PHARMA Remain in control of your life - Corporate presentation JPM 2020

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ASARINA PHARMA Remain in control of your life - Corporate presentation JPM 2020
ASARINA PHARMA
 Remain in control of your life

 Corporate presentation
 JPM 2020

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ASARINA PHARMA Remain in control of your life - Corporate presentation JPM 2020
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

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ASARINA PHARMA Remain in control of your life - Corporate presentation JPM 2020
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
 Topline results April 2020 recruiting 206 patients completed randomization December 2019

 • 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

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ASARINA PHARMA Remain in control of your life - Corporate presentation JPM 2020
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
 Astra Zeneca Pharmacia & Upjohn Pharmacia & Upjohn
 Lundbeck Siemens Medical Kabi Fresenuis
 Novozymes
 Karolinska Development
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ASARINA PHARMA Remain in control of your life - Corporate presentation JPM 2020
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%
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ASARINA PHARMA Remain in control of your life - Corporate presentation JPM 2020
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 Preclinical Sepranolone Bio Eq. Sepranolone
 Phase I
 New administration form New administration form New adm. form

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ASARINA PHARMA Remain in control of your life - Corporate presentation JPM 2020
Sepranolone normalises GABAA-receptor
 activity, targeting underlying cause of PMDD
 Extrasynaptic receptors

 PMDD patients have increased sensitivity to contain a subunit

 GABAA steroid allopregnanolone (ALLO), a Cl- GABA PAM
 b

 which is elevated during the premenstrual
 (luteal) phase of the menstrual cycle
 Extrasynaptic GABAA
 receptors

 Novel PAM

 Sepranolone inhibits the Positive Allosteric binding site

 Modulation (PAM) effect of ALLO on the
 Increased tonic
 GABAergic current
 Postsynaptic terminal

 GABAA receptor through
 • Fine tuned receptor activity
 without overstimulation
 • High selectivity
 • Minimal off-target effects

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ASARINA PHARMA Remain in control of your life - Corporate presentation JPM 2020
Sepranolone in Premenstrual
 Dysphoric Disorder

 Remain in control of your life
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ASARINA PHARMA Remain in control of your life - Corporate presentation JPM 2020
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
 Anxiety/Depression • More than a third of PMDD women have suicidal thoughts and are
 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
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ASARINA PHARMA Remain in control of your life - Corporate presentation JPM 2020
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 Black Box Warning
 Require hormonal
 months due to side
 add-back
 effects
 Approved U.S. U.S. U.S.

 Sepranolone
 Initial formulary placement: 2nd line therapy
 Current 1st 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
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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 premenstrual
 Active n=70 symptom score
 (p=0.041) compared
 to placebo

 Highly stafsfcally
 significant reducfon in
 pre-menstrual
 symptom score in
 “treated as intended”
 n=26 n=34 populafon (p = 0.006)
 Placebo Sepranolone

 *Total symptom score of 11 symptoms
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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 (DSM-5) verified
 Randomize Sepranolone
 • Responder analysis Multicenter N= ~206
 D, UK, PL, S
 in at least two
 (Double-blind) dose 16 mg
 menstrual cycles
 e-PRO
 • DRSP according to DSM-5 as
 diagnostic screener for PMDD Placebo

 Overwhelming interest/very low drop out rate of < 15%
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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
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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.

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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

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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 using the eDiary

 • In parallel, a clinical pharmacology program for special populations,
 abuse potential, etc. will be conducted. Deferral will be requested
 for pediatric studies
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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 treated with Sepranolone

 - Annual Sepranolone pricing e.g. USD 6.000
 (Aimovig for Migraine: USD 6.900 annually)
 (Elagolix for Endometriosis: USD 10.000 annually)
 (Relugolix for Uterine fibrosis: USD 7.200 annully)

 - Annual ww peak sales of Sepranolone > USD 1.0 bill

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Sepranolone in menstrual migraine

 Remain in control of your life
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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 or no response to prophylacfc treatment
 with CGRP anfbodies
 • 1 out of 5 women migraineurs suffers from menstrual migraine.
 Only symptomafc treatments (triptans, NSAID) are available
 Sepranolone aims to prevent the inifafon of the migraine avack, by
 avenuafng the hyperexcitability of hypothalamic GABAA neurons

 Prof Nissilä: “My experience was that MM a:acks
 were the only kind to keep persis=ng throughout
 CGRP medica=on. Neither triptans nor CGRP
 an=bodies are fully effec=ve against MM”

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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 cycle in 120 cycles from 38 women
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Changes in circulating neurosteroid levels are
 associated with migraine

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> 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 phase of three menstrual cycles,
 followed by three cycles of Sepranolone treatment. Women self-administer
 Sepranolone every 48 hours during the luteal phase of their menstrual cycle
 • Primary endpoint is reducfon from baseline in number of migraine days
 • Enrollment is on track, with half of pafents enrolled at year end 2019
 • Phase IIb will include different dosing regimens, trying to reduce the number
 of injecfons
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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
 - 200.000 patients being treated with Sepranolone

 - Annual Sepranolone pricing e.g. USD 6.000
 (Aimovig, Avojy, Emgality for Migraine: USD 6.900 annually)

 - Annual ww peak sales of Sepranolone > USD 1.0 bill

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Sepranolone in Tourette Syndrome
 - a new opportunity

 Remain in control of your life
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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 parents feel that their childs symptoms are
 not adequately controlled by existing medications

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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 male and female TS patients, ages 14-45, randomized
 to sepranolone or no treatment
 • After a 1-month baseline period, patients will receive sepranolone every other
 day during 3 months*. Endpoint is reduction of tics on the YGTSS** scale

 * Due to natural waxing and waning of tics, too short observation periods tend to overestimate effects
 **Yale Global Tic Severity Scale (YGTSS) (McGuire et al 2018)
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Dose dependent tic reduction with Sepranolone

 Spatial confinement
 1.50
 Vehicle (SC)
 P
Efficacy on par with Haldol and Finasteride

 Spatial confinement
 1.50
 Vehicle (SC)
 P
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 Sepranolone pricing e.g. USD 50.000
 (Ingrezza for TD: USD 64.400 annually)

 - Annual ww peak sales of Sepranolone > USD 1.0 bill

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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

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Value generation

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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
 Migraine

 August 2019 Q1 - 2020 Q3 - 2020
 July 2019 April - 2020 Q3 - 2020 Q4 - 2020

 ü PMDD got its own code in ICD-11 May 2019
 ü IND approval for Sepranolone in Menstrual Migraine July 2019
 APH205 study
 Q3-4 - 2020 initiation
 Tourette’s
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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 similar size market opportunity in Europe and ROW

 ü Topline results in April 2020 in 206 subjects/14 centers from
 Phase IIb PMDD study

 ü > 50 % of 80-90 subjects in 7 centers for Phase IIa study in Menstrual Migraine
 enrolled by December 31st

 ü Tourette Phase IIa study to be initiated Q3 2020 with read out Q3 2021

 ü Strong cash position to finalize all three studies and production scale up for Phase III

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