ASARINA PHARMA AnnualGeneral Meeting 8 May 2019 · • The shares of Asarina Pharma (”Asarina”)...

23
1 Remain in control of your life ASARINA PHARMA Annual General Meeting 8 May 2019

Transcript of ASARINA PHARMA AnnualGeneral Meeting 8 May 2019 · • The shares of Asarina Pharma (”Asarina”)...

Page 1: ASARINA PHARMA AnnualGeneral Meeting 8 May 2019 · • The shares of Asarina Pharma (”Asarina”) are traded on NASDAQ First North in Stockholm (ticker: ”ASAP”) • This presentation

1

Remain in control of your life

ASARINA PHARMAAnnual General Meeting8 May 2019

Page 2: ASARINA PHARMA AnnualGeneral Meeting 8 May 2019 · • The shares of Asarina Pharma (”Asarina”) are traded on NASDAQ First North in Stockholm (ticker: ”ASAP”) • This presentation

2

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

Page 3: ASARINA PHARMA AnnualGeneral Meeting 8 May 2019 · • The shares of Asarina Pharma (”Asarina”) are traded on NASDAQ First North in Stockholm (ticker: ”ASAP”) • This presentation

3

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

Page 4: ASARINA PHARMA AnnualGeneral Meeting 8 May 2019 · • The shares of Asarina Pharma (”Asarina”) are traded on NASDAQ First North in Stockholm (ticker: ”ASAP”) • This presentation

4

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

Page 5: ASARINA PHARMA AnnualGeneral Meeting 8 May 2019 · • The shares of Asarina Pharma (”Asarina”) are traded on NASDAQ First North in Stockholm (ticker: ”ASAP”) • This presentation

5

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

Page 6: ASARINA PHARMA AnnualGeneral Meeting 8 May 2019 · • The shares of Asarina Pharma (”Asarina”) are traded on NASDAQ First North in Stockholm (ticker: ”ASAP”) • This presentation

6

Sepranolone in Premenstrual Dysphoric Disorder

Remain in control of your life

Page 7: ASARINA PHARMA AnnualGeneral Meeting 8 May 2019 · • The shares of Asarina Pharma (”Asarina”) are traded on NASDAQ First North in Stockholm (ticker: ”ASAP”) • This presentation

7

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

Page 8: ASARINA PHARMA AnnualGeneral Meeting 8 May 2019 · • The shares of Asarina Pharma (”Asarina”) are traded on NASDAQ First North in Stockholm (ticker: ”ASAP”) • This presentation

8

Sepranolone in Menstrual Migraine

Remain in control of your life

Page 9: ASARINA PHARMA AnnualGeneral Meeting 8 May 2019 · • The shares of Asarina Pharma (”Asarina”) are traded on NASDAQ First North in Stockholm (ticker: ”ASAP”) • This presentation

9

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

Page 10: ASARINA PHARMA AnnualGeneral Meeting 8 May 2019 · • The shares of Asarina Pharma (”Asarina”) are traded on NASDAQ First North in Stockholm (ticker: ”ASAP”) • This presentation

10

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

Page 11: ASARINA PHARMA AnnualGeneral Meeting 8 May 2019 · • The shares of Asarina Pharma (”Asarina”) are traded on NASDAQ First North in Stockholm (ticker: ”ASAP”) • This presentation

11

Sepranolone in Tourette´s Syndrome- a new opportunity

Remain in control of your life

Page 12: ASARINA PHARMA AnnualGeneral Meeting 8 May 2019 · • The shares of Asarina Pharma (”Asarina”) are traded on NASDAQ First North in Stockholm (ticker: ”ASAP”) • This presentation

12

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

Page 13: ASARINA PHARMA AnnualGeneral Meeting 8 May 2019 · • The shares of Asarina Pharma (”Asarina”) are traded on NASDAQ First North in Stockholm (ticker: ”ASAP”) • This presentation

13

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

Page 14: ASARINA PHARMA AnnualGeneral Meeting 8 May 2019 · • The shares of Asarina Pharma (”Asarina”) are traded on NASDAQ First North in Stockholm (ticker: ”ASAP”) • This presentation

14

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

Page 15: ASARINA PHARMA AnnualGeneral Meeting 8 May 2019 · • The shares of Asarina Pharma (”Asarina”) are traded on NASDAQ First North in Stockholm (ticker: ”ASAP”) • This presentation

15

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

Page 16: ASARINA PHARMA AnnualGeneral Meeting 8 May 2019 · • The shares of Asarina Pharma (”Asarina”) are traded on NASDAQ First North in Stockholm (ticker: ”ASAP”) • This presentation

16

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

Page 17: ASARINA PHARMA AnnualGeneral Meeting 8 May 2019 · • The shares of Asarina Pharma (”Asarina”) are traded on NASDAQ First North in Stockholm (ticker: ”ASAP”) • This presentation

17

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

Page 18: ASARINA PHARMA AnnualGeneral Meeting 8 May 2019 · • The shares of Asarina Pharma (”Asarina”) are traded on NASDAQ First North in Stockholm (ticker: ”ASAP”) • This presentation

18

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

Page 19: ASARINA PHARMA AnnualGeneral Meeting 8 May 2019 · • The shares of Asarina Pharma (”Asarina”) are traded on NASDAQ First North in Stockholm (ticker: ”ASAP”) • This presentation

19

Financials

Remain in control of your life

Page 20: ASARINA PHARMA AnnualGeneral Meeting 8 May 2019 · • The shares of Asarina Pharma (”Asarina”) are traded on NASDAQ First North in Stockholm (ticker: ”ASAP”) • This presentation

20 20

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

Page 21: ASARINA PHARMA AnnualGeneral Meeting 8 May 2019 · • The shares of Asarina Pharma (”Asarina”) are traded on NASDAQ First North in Stockholm (ticker: ”ASAP”) • This presentation

21

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

Page 22: ASARINA PHARMA AnnualGeneral Meeting 8 May 2019 · • The shares of Asarina Pharma (”Asarina”) are traded on NASDAQ First North in Stockholm (ticker: ”ASAP”) • This presentation

22

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%

Page 23: ASARINA PHARMA AnnualGeneral Meeting 8 May 2019 · • The shares of Asarina Pharma (”Asarina”) are traded on NASDAQ First North in Stockholm (ticker: ”ASAP”) • This presentation

23

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