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Remain in control of your life
ASARINA PHARMAAnnual General Meeting8 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 NordkildCEO
MDNovo Nordisk Ferring, Egalet, Pharmexa
Jakob Dynnes Hansen
CFOMSc, MBANovo Nordisk Zealand PharmaEvolva, Nordea
Otto Skolling CBO
MScPharmacia & UpjohnSiemens MedicalNovozymesKarolinska Development
Karin EkbergCOO
PhD, clinical physiologyCreative Peptides Umecrine Cognition
Märta SegerdahlCMO
PhD, Med. Sci.Astra ZenecaLundbeck
Sven GötheCMC
PhDPharmacia & UpjohnKabi Fresenuis
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Menstrual Migraine Phase IIa – proof of concept
1st generation Sepranolone
Menstrual Migraine Phase IIb – dose finding
(with autoinjector)
2018 2019 2020 2021 2022
PMDD Phase IIb
2nd generation Sepranolone
Gel/vaginal ring/sustained release/tablet selection process
PMDD Phase III (with autoinjector)
CMC & Upscaling API & DP scale up
A robust project portfolio
SA
EoP2
2nd generation development 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 other 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/DiagnosisTwo cycles
1 month follow-up3 treatment cycles
ScreenMulticenterD, UK, PL, S
Sepranolonedose 10 mg
Placebo
RandomizeN= 200-225
(Double-blind)
PMDD (DSM-5) verified in at least two
menstrual cycles
Sepranolonedose 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 migraineMacGregor 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 DESIGNRCT, 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
Screen7 centers in
Denmark, Sweden and Finland
Sepranolonedose 10 mg
Placebo
RandomizeN=80-90
(Double-blind)
Menstrual migraine
Diagnosis verified in 3 baseline cycles
Sepranolonedose 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 needChallenging clinical trial design
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Source of VariationInteractionRow FactorColumn Factor
ANOVA tableInteractionRow FactorColumn Factor
% of total variation7.46114.1242.96
SS0.23260.44021.339
P value0.03850.0008< 0.0001
DF212
P value summary********
MS0.11630.44020.6696
Significant?YesYesYes
F (DFn, DFd)F (2, 30) = 3.637F (1, 30) = 13.77F (2, 30) = 20.94
P valueP = 0.0385P = 0.0008P < 0.0001
Spatial confinement
WT D1CT-70.00
0.25
0.50
0.75
1.00
1.25
1.50Vehicle (SC)Sepranolone (5 mg/kg, SC)Sepranolone (10 mg/kg, SC)
# Ti
cs/m
in
P<0.0001P<0.00001P<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-70.00
0.25
0.50
0.75
1.00
1.25
1.50Vehicle (SC)Sepranolone (10 mg/kg, SC)Haloperidol (0.3 mg/kg, IP)
# Ti
cs/m
in
P<0.00001P<0.00001P<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 VariationInteractionRow FactorColumn Factor
ANOVA tableInteractionRow FactorColumn Factor
% of total variation16.2323.8533.18
SS (Type III)0.45980.67540.9398
P value<0.0001<0.0001<0.0001
DF313
P value summary************
MS0.15330.67540.3133
Significant?YesYesYes
F (DFn, DFd)F (3, 48) = 9.716F (1, 48) = 42.82F (3, 48) = 19.86
P valueP<0.0001P<0.0001P<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 of 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 Sepranolonein 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 of 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 2017Net 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