ASARINA PHARMA Annual General Meeting 8 May 2019 Remain in control of your life 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 • Efficacy in humans established for Sepranolone in premenstrual Clinical mid-stage company with pipeline in dysphoric disorder (PMDD) women’s health • Additional indications with high unmet medical need • Novel therapy with unique Mode of Action First-in-class therapy for PMDD – • Substantial unmet medical need: a highly underserved indication Disabling condition affecting 4-5 % of women in fertile age • Significant medical costs for society create large market potential Phase IIb initiated in April 2018. • Phase IIb study with 14 centers in UK, Poland, Germany and Sweden recruiting 200-225 patients Topline results expected early 2020 Phase IIa Proof of Concept study in 80-90 women with Menstrual • Menstrual Migraine: Migraine in 7 centers in Sweden, Denmark and Finland. mid-term significant value inflection point Study initiation July 2019 and read-out Q4 2020 • Building a Scandinavian franchise in women’s health Significant commercial potential – • Potential PMDD annual peak sales: USD 600 million worldwide total peak sales > USD 1.4 billion • Potential for attractive license deals to fund further growth 3
The Asarina team Jakob Dynnes Karin Ekberg Peter Nordkild Hansen COO CEO CFO MD PhD, clinical MSc, MBA Novo Nordisk physiology Novo Nordisk Ferring, Egalet, Creative Peptides Zealand Pharma Pharmexa Umecrine Cognition Evolva, Nordea Sven Göthe Otto Skolling Märta Segerdahl CMC CBO CMO MSc PhD PhD, Med. Sci. Pharmacia & Upjohn Pharmacia & Upjohn Astra Zeneca Siemens Medical Kabi Fresenuis Lundbeck Novozymes Karolinska Development 4
A robust project portfolio 2018 2019 2020 2021 2022 1 st generation EoP PMDD Pha PM Phase e IIb IIb PMDD Pha PM Phase e III (with h aut utoinj njec ector) Sepranolone 2 Me Menst strual Mi Migraine Me Menst strual Mi Migraine SA Phase Pha e IIb – dos dose findi ding Phase Pha e IIa IIa – pr proof oof of of con oncept pt (w (with au autoinjector) 2 nd generation Gel/vagi Ge ginal r ring/ g/sustained d 2nd gener 2nd eneration n de developm pment pr process release/tablet selection pro re rocess Sepranolone CMC & Upscaling API & AP & DP scale le up 5
Sepranolone in Premenstrual Dysphoric Disorder Remain in control of your life 6
Phase IIb: recruitment initiated April 2018. > 3/4 of patients recruited. Readout Q1 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= 200-225 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 7
Sepranolone in Menstrual Migraine Remain in control of your life 8
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 9
Phase IIa clinical Proof of Concept study in Menstrual Migraine baseline / diagnosis 3 cycles treatment follow-up 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 Sepranolone dose 10 mg Secondary endpoints: Duration and severity of migraine attacks Menstrual Screen Randomize Sepranolone migraine 7 centers in N=80-90 dose 16 mg Denmark, Sweden and Diagnosis verified in 3 (Double-blind) Finland baseline cycles Placebo 10
Sepranolone in Tourette´s Syndrome - a new opportunity Remain in control of your life 11
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 Inhibition of positive GABA-A modulating (PAM) Sepranolone 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 12
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 Sepranolone (10 mg/kg, SC) 1.00 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? All movements were scored by personnel blinded to Interaction 7.461 0.0385 * Yes Row Factor 14.12 0.0008 *** Yes treatment and genotype; n=5-7/group Column Factor 42.96 < 0.0001 **** Yes ANOVA table SS DF MS F (DFn, DFd) P value Interaction 0.2326 2 0.1163 F (2, 30) = 3.637 P = 0.0385 Analysis: 2-way ANOVA followed by Tukey’s post-hoc Row Factor 0.4402 1 0.4402 F (1, 30) = 13.77 P = 0.0008 Column Factor 1.339 2 0.6696 F (2, 30) = 20.94 P < 0.0001 tests 13
Tic reduction 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 Finasteride (25 mg/kg, IP) NS 0.75 NS 0.50 NS 0.25 0.00 WT D1CT-7 Source of Variation % of total variation P value P value summary Significant? Interaction 16.23 <0.0001 **** Yes All movements were scored by personnel blinded to Row Factor 23.85 <0.0001 **** Yes treatment and genotype; n=7/group Column Factor 33.18 <0.0001 **** Yes ANOVA table SS (Type III) DF MS F (DFn, DFd) P value Interaction 0.4598 3 0.1533 F (3, 48) = 9.716 P<0.0001 Analysis: 2-way ANOVA followed by Tukey’s post-hoc Row Factor 0.6754 1 0.6754 F (1, 48) = 42.82 P<0.0001 Column Factor 0.9398 3 0.3133 F (3, 48) = 19.86 P<0.0001 tests 14
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 15
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 16
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 17
Value inflection points – external communication 2019-2020 2019 2020 APH204 study UM203 top APH204 last initiation line results patient first PMDD Menstrual dose Migraine IND Last patient UC2016 Proof APH204 top approval for first visit of concept in line results Sepranolone UM203 animals Menstrual in MM PMDD Migraine June 2019 Q 3 - 2020 Q 1 - 2020 Q 3 - 2019 Q 2 - 2020 Q 2 -Q 3 2019 Q 4 - 2020 18
Financials Remain in control of your life 19
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