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InDex Pharmaceuticals
The Way Forward Towards Phase IIIand
Q & A
December 12, 2019
Forward Looking Statement
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This presentation contains certain forward-looking statements reflecting the Company’s current view of 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 of 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
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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
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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
1Ulcerative colitis disease coverage. Datamonitor Healthcare 2016
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
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1. The Succesful Phase IIb Study CONDUCT
Phase IIb Study Design
Prim
ary
en
dp
oin
t
Fo
llow
up
Allocation1:1:1:1:1
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
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• 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
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Successful Topline Results
Clinical Remission at Week 6*
COBITOLIMODPLACEBO
(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
6,45,4
3,65,3
6,8
16,517,9
16,9 16,615,6
21,4
0
5
10
15
20
25
30
35
40
Adalimumab§ Golimumab§ Vedolizumab§ Tofacitinib§ Ustekinumab§ Cobitolimod*
% p
atie
nts
in c
linic
al r
emis
soin 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
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TNF-α inhibitors TLR9 agonistIntegrin inhibitor JAK inhibitor IL-23/IL-12 inhibitor
Competitive Efficacy vs. Late Stage Pipeline
0
2,7
6,2
8,8
0
4,86,8
21
16,7 16,4
33
19,6
22,621,4
0
5
10
15
20
25
30
35
40
Etrolizumab§ SHP647§ Ozanimod§ Etrasimod* Upadacitinib* Mirikizumab* Cobitolimod*
% p
atie
nts
in c
linic
al r
emis
sio
n
Placebo Best dose
Integrin inhibitors JAK inhibitorS1PR modulators IL-23 inhibitor TLR9 agonist
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§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
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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%) 0 2 (4.8%) 4 (9.5%) 2 (4.5%)
Deaths 0 0 0 0 1 (2.3%)
Safety analysis set, some patients have reported several adverse events
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Safety Concerns with Other Drug Classes
DRUG CLASS SAFETY PROFILE
TNF-α inhibitors Infections, malignancies, skin disorders
Integrin inhibitors Infections, hypersensitivity reactions
JAK inhibitorsInfections, 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
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2. The Way Forward Towards Phase III
Full Speed Ahead Towards Phase III
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• 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
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• 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 studyActive or Placebo
N=approx 200
Induction study 1Active
or PlaceboN=approx 500
Induction study 2Active
or PlaceboN=approx 500
Long-term follow-up studyActive
Open label feeding studyActive
Market approvalinduction &
maintenance
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• 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
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• 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 studyCobitolimod or Placebo
Induction study 1Cobitolimod
or PlaceboInduction study 2
Cobitolimodor Placebo
Market approvalinduction
Open labelfeeding studyCobitolimod
Market approvalmaintenance
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
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$
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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
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• 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 MOACLINICAL
PHASETERMS
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 equityinvestment + $1.35 billion milestones + tiered royalty starting at 20%
2016 Pfizer Shire SHP647 Integrin inhibitor Phase II$90 million upfront + $460 million milestones+ royalty
2016MedImmune/ 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
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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
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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
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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
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