InDex Pharmaceuticals The Way Forward Towards Phase III and Q - - PowerPoint PPT Presentation

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InDex Pharmaceuticals The Way Forward Towards Phase III

and

Q & A

December 12, 2019

Forward Looking Statement

This presentation contains certain forward-looking statements reflecting the Company’s current view

• f future events and financial and operational performance. Such forward-looking statements are

associated with both known and unknown risks and circumstances outside the Company’s control. All statements in this presentation other than statements of historical or current facts or circumstances are forward-looking statements. Forward-looking statements are made in several sections of the presentation and can be identified by the use of terms or expressions such as “may”, “could”, “should”, “anticipated”, “estimated”, “expected”, “likely”, “forecasted”, “plans to”, “aims to”, or conjugations of such terms or similar terms. The forward-looking statements only apply as of the date

• f this presentation. The Company has no intent or obligation to publish updated forward-looking

statements or any other information contained in this presentation based on new information, future events etc. other than required by applicable law, regulation or regulatory framework.

• 1. The successful phase IIb study CONDUCT
• 2. The way forward towards phase III
• 3. Strengthened shareholder base
• 4. Q&A

CEO Peter Zerhouni, Chairman Wenche Rolfsen and board member Lennart Hansson

Agenda

Ulcerative Colitis – a Debilitating Disease with High Unmet Medical Need

“I always need to be close to a toilet, which restricts my life significantly. The worst is to be so socially disabled. I would like to be spontaneous and just live my life without worrying about my stomach and toilet visits”,

Emma, 24 years old suffering from ulcerative colitis

• Ulcerative colitis is an inflammatory bowel disease (IBD) with chronic

inflammation of the colonic mucosa leading to ulcers

• Recurrent with active and inactive periods
• Very frequent blood- and mucus-mixed loose stools
• High negative impact on quality of life

Clear Need for Safer and More Efficacious Drugs in Moderate to Severe UC

Colectomy Biologics JAK inhibitors Immunomodulators Glucocorticosteroids (GCS) Aminosalicylates

Mesalazine (5-ASA), Sulphasalazine (SP)

Last line Cobitolimod Third line Second line First line

• Third line therapies have problems with tolerance

and severe side effects

• >$5 Bn per year of biologics sales globally in UC

alone1

• >200,000 UC patients globally receive treatment

with biologics1

Cobitolimod – InDex’s Lead Drug Candidate

• Cobitolimod is a potential new medication for

moderate to severe ulcerative colitis

• Successful phase IIb study CONDUCT
• 4 previous completed clinical studies support

efficacy and safety demonstrated in CONDUCT

• Competitive efficacy
• Excellent safety, very low systemic uptake
• Local treatment, provides rapid onset of action
• Novel mechanism of action (TLR9 agonist)
• Potential for combination therapy

Cobitolimod has high market potential with an outstanding combination of efficacy and safety

• 1. The Succesful Phase IIb Study CONDUCT

Phase IIb Study Design

Primary endpoint Follow up Allocation 1:1:1:1:1 N=213 Week 0 Week 1 Week 2 Week 3 Week 6 Week 10

Cobitolimod 30 mg Cobitolimod 125 mg Cobitolimod 250 mg Cobitolimod 125 mg Placebo Placebo Placebo Placebo Cobitolimod 125 mg Placebo Placebo Placebo Placebo Cobitolimod 125 mg Placebo Cobitolimod 30 mg Cobitolimod 125 mg Cobitolimod 250 mg Cobitolimod 125 mg Placebo

• Moderate to severe active

left sided UC

• Failed 5-ASA/SP and GCS
• Failed immunomodulators

and/or biologics

• No concomitant biologics

Exploratory study to identify best dosing regimen for phase III

Successful Topline Results

Clinical Remission at Week 6* COBITOLIMOD PLACEBO (n=44) 30 mg x 2 (n=40) 125 mg x 2 (n=43) 125 mg x 4 (n=42) 250 mg x 2 (n=42) % of patients

12.5 % 4.7 % 9.5 % 21.4 % 6.8 %

Δ to placebo

5.7 %

• 2.1 %

2.7 % 14.6 %

P-value one-sided test

(pre-specified with cut-off <0.10)

n.s. n.s. n.s. 0.0247

P-value two-sided test

n.s. n.s. n.s. 0.0495

Full analysis set, *Primary Endpoint = Clinical Remission at Week 6 defined as Modified Mayo sub scores: i) rectal bleeding of 0, ii) stool frequency of 0 or 1 and iii) endoscopy score of 0 or 1 (excluding friability)

Sensitivity analyses confirm the robustness of the primary endpoint findings

9,3 6,4 5,4 3,6 5,3 6,8 16,5 17,9 16,9 16,6 15,6 21,4

5 10 15 20 25 30 35 40 Adalimumab§ Golimumab§ Vedolizumab§ Tofacitinib§ Ustekinumab§ Cobitolimod*

% patients in clinical remissoin Placebo Best dose

§Full Mayo Score ≤2, *3-component Mayo Score ≤2. Caution advised when comparing data across clinical studies

NOTE: Infliximab excluded from comparison as not comparable phase III patient population

Competitive Efficacy vs. Current Products

TNF-α inhibitors TLR9 agonist Integrin inhibitor JAK inhibitor IL-23/IL-12 inhibitor

Competitive Efficacy vs. Late Stage Pipeline

2,7 6,2 8,8 4,8 6,8 21 16,7 16,4 33 19,6 22,6 21,4

5 10 15 20 25 30 35 40 Etrolizumab§ SHP647§ Ozanimod§ Etrasimod* Upadacitinib* Mirikizumab* Cobitolimod*

% patients in clinical remission Placebo Best dose Integrin inhibitors JAK inhibitor S1PR modulators IL-23 inhibitor TLR9 agonist

§Full Mayo Score ≤2, *3-component Mayo Score ≤2. Caution advised when comparing data across clinical studies

NOTE: Filgotinib not included as no data reported in UC

Excellent Safety Profile

Treatment Emergent Adverse Events No of patients (%) COBITOLIMOD PLACEBO (n=44) 30 mg x 2 (n=40) 125 mg x 2 (n=43) 125 mg x 4 (n=42) 250 mg x 2 (n=42) Patients with AEs

10 (25.0%) 17 (39.5%) 15 (35.7%) 18 (42.9%) 21 (47.7%)

Patients with Serious AEs

2 (5.0%) 2 (4.8%) 4 (9.5%) 2 (4.5%)

Deaths

1 (2.3%)

Safety analysis set, some patients have reported several adverse events

Safety Concerns with Other Drug Classes

DRUG CLASS SAFETY PROFILE

TNF-α inhibitors Infections, malignancies, skin disorders Integrin inhibitors Infections, hypersensitivity reactions JAK inhibitors Infections, cancer, tears (perforation) in the stomach or intestines, pulmonary embolism IL-23 inhibitors Infections, malignancies S1PR modulators Heart rate effect, elevated liver transaminase

Cobitolimod has demonstrated an excellent safety profile

CONDUCT Fulfilled All Study Objectives

• Met the primary endpoint
• Identified the dose to move forward with
• Competitive efficacy
• Superior safety profile
• Outstanding combination of efficacy and safety with a novel and unique

mechanism of action

• 2. The Way Forward Towards Phase III

Full Speed Ahead Towards Phase III

• Analyze full CONDUCT data set – Q4 2019/Q1 2020
• Regulatory interactions – Q1 2020
• Protocol development – Q1 2020
• CRO initiation/study feasibility – Q1 2020
• Market research – Q1 2020
• Study drug manufacturing – H1 2020
• Additional tox studies – H1 2020
• Planned First-Patient-In – H2 2020

Going Alone – an Attractive Opportunity

• Successful phase IIb study and change in regulatory requirements
• New possibilities for phase III design that InDex can manage on its own
• Enables a faster and more efficient way to market
• Maximizes shareholder value
• Strengthens negotiation position towards potential partners

Traditional Phase III Program Design

Maintenance study Active or Placebo N=approx 200 Induction study 1 Active

• r Placebo

N=approx 500 Induction study 2 Active

• r Placebo

N=approx 500 Long-term follow-up study Active Open label feeding study Active Market approval induction & maintenance

• Two pivotal induction studies and one maintenance study including approximately 1,000 patients
• Optional open label feeding study to reduce # of patients in the induction studies
• Re-randomization to maintenance study

Sequential Phase III Program Tentative Design

• Minimize risk by performing sequential studies
• Allows stepwise financing with additional de-risked inflection points
• Fast to market potential by 2-step approval process
• First label induction of remission, to be followed by extended label for maintenance treatment

Maintenance study Cobitolimod or Placebo Induction study 1 Cobitolimod

• r Placebo

Induction study 2 Cobitolimod

• r Placebo

Market approval induction

Open label feeding study Cobitolimod

Market approval maintenance Subject to regulatory and other evaluations

Arena Pharmaceuticals’ Innovative Phase III Program

• One treat through study for induction & maintenance (N=372). NB! no re-randomization
• Second induction study sequentially (N=330)
• First-Patient-In June 2019

$

Project Value Increases as It Gets Closer to Market

VALUE

$$ $$$ $$$$ $$$$$

PHASE I PHASE II PHASE III MARKET TIME CLINICAL DEVELOPMENT BUSINESS DEVELOPMENT

Partnership Discussions to Continue in Parallel

• Maintain and establish contacts with potential partners
• Compile full business development package
• Scientific communication to maximize impact in scientific community
• Going alone strengthens negotiation position
• Control the timing and circumstances for potential partnerships
• A less extensive phase III program widens the universe of potential partners
• Out-licensing at a later stage of development increases the value of cobitolimod

IBD is a High Value Therapeutic Area

RECENT DEALS IN IBD/INFLAMMATION

YEAR COMPANY PARTNER COMPOUND MOA CLINICAL PHASE TERMS

2015 Receptos Celgene Ozanimod S1PR modulator Phase II $7.2 billion (acquisition) 2015 Galapagos Gilead Filgotinib JAK inhibitor Phase II $300 million upfront + $425 million equity investment + $1.35 billion milestones + tiered royalty starting at 20% 2016 Pfizer Shire SHP647 Integrin inhibitor Phase II $90 million upfront + $460 million milestones + royalty 2016 MedImmune/ Astra Zeneca Allergan Brazikumab IL-23 inhibitor Phase IIa $250 million upfront + $1.27 billion milestones + royalty 2018 Theravance J&J TD-1473 JAK inhibitor Phase I $100 million upfront + $900 million milestones + royalty

• 3. Strengthened Shareholder Base

Directed Share Issue To Finance Phase III Preparations

• Raised SEK 140 million in September to finance phase III preparations
• At market issue price of SEK 6.98/share through accelerated book building (no

discount)

• Wide range of Swedish and international investors including AP4, Bengt Julander

(through Linc AB), and Industrifonden

• Positive interest from institutional investors enable continued financing
• Carnegie and Pareto as financial advisors

Strong Shareholder Base

SHAREHOLDER NUMBER OF SHARES OWNERSHIP % VOTES %

SEB Venture Capital 12 994 367 14.6 14.6 Stiftelsen Industrifonden 12 865 296 14.5 14.5 Linc AB 8 875 650 10.0 10.0 Fjärde AP-fonden 6 400 000 7.2 7.2 Avanza Pension 3 338 907 3.8 3.8 Staffan Rasjö 3 124 718 3.5 3.5 Originat AB 2 700 000 3.0 3.0 SEB Life International 2 321 225 2.6 2.6 Skandinaviska Enskilda Banken S.A. 2 300 000 2.6 2.6 Nordnet Pensionsförsäkring AB 2 001 604 2.3 2.3

10 Largest Shareholders per October 18, 2019

Summary

• Successful phase IIb study demonstrates outstanding combination of efficacy and

safety

• CONDUCT data and regulatory changes provide opportunity to go alone in phase III
• Creates maximum value for the shareholders
• Faster and more efficient way towards the market
• Project value increases as it gets closer to the market
• Strengthened negotiating position towards potential partners
• Strong shareholder base and interest from institutional investors

Q&A