InDex Pharmaceuticals The Way Forward Towards Phase III and Q & A December 12, 2019 1
Forward Looking Statement 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. 2
Agenda 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 3
Ulcerative Colitis – a Debilitating Disease with High Unmet Medical Need • 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 “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 4
Clear Need for Safer and More Efficacious Drugs in Moderate to Severe UC • Third line therapies have problems with tolerance and severe side effects Last line Colectomy • >$5 Bn per year of biologics sales globally in UC alone 1 Cobitolimod Biologics • >200,000 UC patients globally receive treatment Third line JAK inhibitors with biologics 1 Immunomodulators 1 Ulcerative colitis disease coverage. Datamonitor Healthcare 2016 Second line Glucocorticosteroids (GCS) First line Aminosalicylates Mesalazine (5-ASA), Sulphasalazine (SP) 5
Cobitolimod – InDex’s Lead Drug Candidate • Cobitolimod is a potential new medication for • Competitive efficacy moderate to severe ulcerative colitis • Excellent safety, very low systemic uptake • Successful phase IIb study CONDUCT • Local treatment, provides rapid onset of action • 4 previous completed clinical studies support • Novel mechanism of action (TLR9 agonist) efficacy and safety demonstrated in CONDUCT • Potential for combination therapy Cobitolimod has high market potential with an outstanding combination of efficacy and safety 6
1. The Succesful Phase IIb Study CONDUCT 7
Phase IIb Study Design • Moderate to severe active Week 0 Week 1 Week 2 Week 3 Week 6 Week 10 left sided UC Cobitolimod Cobitolimod Placebo Placebo • Failed 5-ASA/SP and GCS 30 mg 30 mg • Failed immunomodulators Primary endpoint Cobitolimod Cobitolimod Placebo Placebo 125 mg 125 mg and/or biologics Follow up Allocation • No concomitant biologics Cobitolimod Cobitolimod 1:1:1:1:1 Placebo Placebo 250 mg 250 mg N=213 Cobitolimod Cobitolimod Cobitolimod Cobitolimod 125 mg 125 mg 125 mg 125 mg Placebo Placebo Placebo Placebo Exploratory study to identify best dosing regimen for phase III 8
Successful Topline Results COBITOLIMOD PLACEBO Clinical Remission at Week 6* 30 mg x 2 125 mg x 2 125 mg x 4 250 mg x 2 (n=44) (n=40) (n=43) (n=42) (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 n.s. n.s. n.s. 0.0247 (pre-specified with cut-off <0.10) 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
Competitive Efficacy vs. Current Products 40 35 Placebo Best dose % patients in clinical remissoin 30 25 21,4 20 17,9 16,9 16,6 16,5 15,6 15 9,3 10 6,8 6,4 5,4 5,3 3,6 5 0 Adalimumab§ Golimumab§ Vedolizumab§ Tofacitinib§ Ustekinumab§ Cobitolimod* TNF- α inhibitors Integrin inhibitor JAK inhibitor IL-23/IL-12 inhibitor TLR9 agonist § Full Mayo Score ≤2, *3 - component Mayo Score ≤2. C aution advised when comparing data across clinical studies NOTE: Infliximab excluded from comparison as not comparable phase III patient population 10
Competitive Efficacy vs. Late Stage Pipeline 40 % patients in clinical remission 35 33 Placebo Best dose 30 25 22,6 21,4 21 19,6 20 16,7 16,4 15 8,8 10 6,8 6,2 4,8 5 2,7 0 0 0 Etrolizumab§ SHP647§ Ozanimod§ Etrasimod* Upadacitinib* Mirikizumab* Cobitolimod* Integrin inhibitors S1PR modulators JAK inhibitor 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 11
Excellent Safety Profile COBITOLIMOD Treatment Emergent Adverse Events PLACEBO (n=44) 30 mg x 2 125 mg x 2 125 mg x 4 250 mg x 2 No of patients (%) (n=40) (n=43) (n=42) (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 12
Safety Concerns with Other Drug Classes DRUG CLASS SAFETY PROFILE TNF- α inhibitors Infections, malignancies, skin disorders Integrin inhibitors Infections, hypersensitivity reactions Infections, cancer, tears (perforation) in the stomach or intestines, pulmonary JAK inhibitors embolism IL-23 inhibitors Infections, malignancies S1PR modulators Heart rate effect, elevated liver transaminase Cobitolimod has demonstrated an excellent safety profile 13
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 14
2. The Way Forward Towards Phase III 15
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 16
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 17
Traditional Phase III Program Design • 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 Market approval induction & maintenance Open label feeding study Induction study 1 Active Active or Placebo N=approx 500 Maintenance study Active or Placebo N=approx 200 Induction study 2 Active or Placebo Long-term follow-up study N=approx 500 Active 18
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 Subject to regulatory and other evaluations Induction study 1 Cobitolimod Induction study 2 or Placebo Cobitolimod Open label or Placebo feeding study Cobitolimod Maintenance study Cobitolimod or Placebo Market approval Market approval maintenance induction 19
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 20
Project Value Increases as It Gets Closer to Market CLINICAL DEVELOPMENT VALUE PHASE I PHASE II PHASE III MARKET $$$$$ $$$$ $$$ $$ $ TIME BUSINESS DEVELOPMENT 21
Recommend
More recommend
