ASARINA PHARMA Annual General Meeting 8 May 2019 Remain in control - - PowerPoint PPT Presentation

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Remain in control of your life

ASARINA PHARMA Annual General Meeting 8 May 2019

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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 Overview

• Building a Scandinavian franchise in women’s health
• Potential PMDD annual peak sales: USD 600 million worldwide
• Potential for attractive license deals to fund further growth
• Phase IIa Proof of Concept study in 80-90 women with Menstrual

Migraine in 7 centers in Sweden, Denmark and Finland. Study initiation July 2019 and read-out Q4 2020 Menstrual Migraine: mid-term significant value inflection point

• Novel therapy with unique Mode of Action
• Substantial unmet medical need:

Disabling condition affecting 4-5 % of women in fertile age

• Significant medical costs for society create large market potential

First-in-class therapy for PMDD – a highly underserved indication

• Phase IIb study with 14 centers in UK, Poland, Germany and Sweden

recruiting 200-225 patients Phase IIb initiated in April 2018. Topline results expected early 2020

• Efficacy in humans established for Sepranolone in premenstrual

dysphoric disorder (PMDD)

• Additional indications with high unmet medical need

Clinical mid-stage company with pipeline in women’s health Significant commercial potential – total peak sales > USD 1.4 billion

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The Asarina team

Peter Nordkild CEO MD

Novo Nordisk Ferring, Egalet, Pharmexa

Jakob Dynnes Hansen CFO MSc, MBA

Novo Nordisk Zealand Pharma Evolva, Nordea

Otto Skolling CBO MSc

Pharmacia & Upjohn Siemens Medical Novozymes Karolinska Development

Karin Ekberg COO PhD, clinical physiology

Creative Peptides Umecrine Cognition

Märta Segerdahl CMO PhD, Med. Sci.

Astra Zeneca Lundbeck

Sven Göthe CMC PhD

Pharmacia & Upjohn Kabi Fresenuis

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Me Menst strual Mi Migraine Pha Phase e IIa IIa – pr proof

• of of
• f con
• ncept

pt 1st generation Sepranolone Me Menst strual Mi Migraine Pha Phase e IIb – dos dose findi ding (w (with au autoinjector) 2018 2019 2020 2021 2022 PM PMDD Pha Phase e IIb IIb 2nd generation Sepranolone

Ge Gel/vagi ginal r ring/ g/sustained d re release/tablet selection pro rocess

PM PMDD Pha Phase e III (with h aut utoinj njec ector) CMC & Upscaling AP API & & DP scale le up

A robust project portfolio

SA

EoP 2

2nd 2nd gener eneration n de developm pment pr process

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Sepranolone in Premenstrual Dysphoric Disorder

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Phase IIb: recruitment initiated April 2018. > 3/4 of patients recruited. Readout Q1 2020

Design

• RCT, double-blinded, placebo-

controlled, with two cycles of diagnosis, three treatment cycles and a follow-up cycle. Treatment cycle will be for 14 days (7 injections every

• ther day)

Primary Endpoint

• Change in premenstrual symptom

severity questionnaire (DRSP) range before and during three treatment cycles

Secondary Endpoints

• Safety
• Responder analysis

e-PRO

• DRSP according to DSM-5 as

diagnostic screener for PMDD Baseline/Diagnosis Two cycles 1 month follow-up 3 treatment cycles

Screen

Multicenter D, UK, PL, S

Sepranolone dose 10 mg Placebo

Randomize

N= 200-225 (Double-blind)

PMDD

(DSM-5) verified in at least two menstrual cycles

Sepranolone dose 16 mg

Overwhelming interest/very low drop out rate

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Sepranolone in Menstrual Migraine

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Migraine related to 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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Phase IIa clinical Proof of Concept study in Menstrual Migraine

TRIAL DESIGN

RCT, double-blinded, placebo-controlled with 80-90 patients in parallel dose groups Three treatment cycles with intermittent 16 days exposure (8 injections every other day with start 14 days prior to next menstruation) Primary endpoint: Change in number of migraine days per cycle measured before and during 3 treatment cycles Secondary endpoints: Duration and severity of migraine attacks Screen

7 centers in Denmark, Sweden and Finland

Sepranolone dose 10 mg

Placebo Randomize

N=80-90 (Double-blind)

Menstrual migraine

Diagnosis verified in 3 baseline cycles

Sepranolone dose 16 mg

baseline / diagnosis 3 cycles treatment follow-up

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Sepranolone in Tourette´s Syndrome

• a new opportunity

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Tourette’s syndrome

Prevalence 0.3 - 0.6 % of children 6 - 17 years of age ~ 600.000 patients in US/EU/Japan Symptoms Repetitive movements and unwanted sounds (tics) Cause Disruption of neuro-transmitters due to abnormal GABA-A receptor system Potential effect of Sepranolone Inhibition of positive GABA-A modulating (PAM) effect Current treatment options Behavioural therapy, dopamine antagonists Stage of program

• Preclinical. Phase IIa to begin recruitment Q2 2020

Commercial potential TS pharmaceutical treatment ~ 200k-400k patient treatments annually in US, EU and Japan Opportunity assessment Orphan drug designation opportunity Large unmet medical need Challenging clinical trial design

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Source of Variation Interaction Row Factor Column Factor ANOVA table Interaction Row Factor Column Factor % of total variation 7.461 14.12 42.96 SS 0.2326 0.4402 1.339 P value 0.0385 0.0008 < 0.0001 DF 2 1 2 P value summary * *** **** MS 0.1163 0.4402 0.6696 Significant? Yes Yes Yes F (DFn, DFd) F (2, 30) = 3.637 F (1, 30) = 13.77 F (2, 30) = 20.94 P value P = 0.0385 P = 0.0008 P < 0.0001

Spatial confinement

WT D1CT-7 0.00 0.25 0.50 0.75 1.00 1.25 1.50 Vehicle (SC) Sepranolone (5 mg/kg, SC) Sepranolone (10 mg/kg, SC)

# Tics/min

P<0.0001 P<0.00001 P<0.0001 NS NS

All movements were scored by personnel blinded to treatment and genotype; n=5-7/group Analysis: 2-way ANOVA followed by Tukey’s post-hoc tests

Dose dependent tic reduction with Sepranolone

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Spatial confinement

WT D1CT-7 0.00 0.25 0.50 0.75 1.00 1.25 1.50 Vehicle (SC) Sepranolone (10 mg/kg, SC) Haloperidol (0.3 mg/kg, IP)

# Tics/min

P<0.00001 P<0.00001 P<0.0001 NS NS

All movements were scored by personnel blinded to treatment and genotype; n=7/group Analysis: 2-way ANOVA followed by Tukey’s post-hoc tests

Source of Variation Interaction Row Factor Column Factor ANOVA table Interaction Row Factor Column Factor % of total variation 16.23 23.85 33.18 SS (Type III) 0.4598 0.6754 0.9398 P value <0.0001 <0.0001 <0.0001 DF 3 1 3 P value summary **** **** **** MS 0.1533 0.6754 0.3133 Significant? Yes Yes Yes F (DFn, DFd) F (3, 48) = 9.716 F (1, 48) = 42.82 F (3, 48) = 19.86 P value P<0.0001 P<0.0001 P<0.0001

NS Finasteride (25 mg/kg, IP) P<0.00001

Tic reduction on par with Haldol and Finasteride

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Market opportunity

• Assumptions:
• 600.000 TS patients in US/EU/Japan
• 200.000 patients in US/EU/Japan eligible for treatment
• Market penetration of 10% at peak sales
• 10% or 20.000 patients being treated with Sepranolone
• Annual Sepranolone pricing e.g. USD 75.000

(Ingrezza for TD: USD 64.400 annually)

• Annual peak sales of Sepranolone e.g. > USD 1 bill
• Lundbeck acquisition of Abide Therapeutics and ABX 1431 –

presently in phase IIa for Tourette´s for USD 250 mio upfront

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Development of Sepranolone for Tourette´s

• Phase IIa, 3 months continuous administration, proof
• f concept study in e.g. 30 male TS´s subjects
• Apply for orphan drug designation in EU and US
• Differentiated pricing will require two distinctly

different formulations e.g. intravaginal ring for PMDD and MM and a gel/tablet for Tourette´s

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Autoinjector

Ypsomed (Ypsomate™) and BD (Intevia™) selected as lead product candidates

• Proof of concept with Sepranolone product
• Detailed analysis ongoing
• Selection of Autoinjector June 2019
• SDS to support the Regulatory process

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Value inflection points – external communication 2019-2020

Q2-Q3 2019

IND approval for Sepranolone in MM APH204 study initiation Menstrual Migraine

June 2019

Last patient first visit UM203 PMDD

Q3 - 2019

UM203 top line results PMDD

Q1 - 2020

UC2016 Proof

• f concept in

animals

Q2 - 2020

APH204 last patient first dose

Q3 - 2020

APH204 top line results Menstrual Migraine

Q4 - 2020

2019 2020

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Financials

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Key financials 2018

MSEK 2018 2017 Net sales

• 1,7

Research and dev. 39,0 23,0 Other external costs 6,2 3,5 Staff costs 6,4 3,9 Depreciation

• 3,9

Financial items (net) 0,0 0,2 Tax (income) 7,6 4,0 Net result 44,0

• 28,3
• Cash position - year-end

141,5 9,4

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The Asarina share

• Number of issued shares: 16.0 million
• Warrant program: 4.7 % of issued shares
• Trading platform: NASDAQ First North (Stockholm)
• First day of trading: 24 September 2018
• IPO proceeds: SEK 150 million
• IPO share price: SEK 21
• Current share price (May 7th 2019): SEK 27

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Major shareholders

> 50% are non-Swedish investors

Kurma Biofund (France) Östersjöstiftelsen (Sweden) Rosetta Capital (UK) Idinvest Patrimonie (France) Swedbank Robur Fonder (Sweden) Sectoral Asset Management (Canada) Catella Fonder (Sweden) Ergomed plc (UK) Handelsbanken Fonder (Sweden) Nordnet Pensionsförsäkring (Sweden) Others (incl. 740 private shareholders) Total 19.6% 14.7% 12.8% 10.2% 7.4% 7.4% 5.2% 2.4% 2.4% 1.7% 16.1% 100.0%

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Summary

• Womens’ health company with experienced management team
• Innovative approach to diseases with serious un-met medical need
• Ongoing phase IIb study in Premenstrual Dysphoric Disorder (PMDD)
• Phase IIa study in Menstrual Migraine to commence in July 2019
• Interesting orphan indication in Tourette´s Syndrome
• Solid share performance since IPO in September 2018