Developing first in class cardiovascular drugs Biotech Agora Event - November 8 th , 2016
Company highlights Novel therapeutic class - brain aminopeptidase A inhibitors (BAPAIs) - provides antihypertensive effects and cardioprotection Strategy to target patient subgroups in prevalent indications with high unmet medical needs (hypertension and heart failure) Lead program, QGC001 , completed 30-patient phase IIa study in hypertension – postive results have been announced on September 29, 2016 Initiated QGC101 congestive heart failure program: first 3 clinical centers opened mid 2016 in France and Norway for pan-European phase IIa study Broad intellectual property portfolio, multiple patent families granted or filed Experienced management team and Scientific/Clinical Advisory Board 2
Key market data 9.4 million 1/3 Deaths worlwide every year due 1/3 people die due to cardiovascular to complications of high blood disease. With 17 million deaths per year, pressure (1) it is the leading cause of death worldwide (1) At least 1/3 of adults has high blood pressure. “Hyper -pressure contributes to nearly 9.4 million deaths due Proportion to heart disease and stroke every year and, together, increases to one these two diseases are the number one cause of death worldwide. in two for people And, hyper-pressure also increases the risk of kidney failure, above age 50 (1) blindness and several other conditions. It often occurs together with other risk factors like obesity, diabetes and high cholesterol – increasing the health risk even further .” WHO Chief Dr Margaret Chan – World Health Day April 07, 2013 $ 40 billion $ 39 billion Global anti-hypertensive Global heart failure drugs market estimate for 2015 (3) drugs market in 2013 (2) Sources : (1) WHO (World Health Organization) - A global brief on hypertension, Silent killer, global public health crisis (2013), (2) the pharmaletter (June 2014), (3) Heidenreich et al, Circulation Heart Failure (2013) 3 3
Blockbuster patent expiration creates opportunity Blockbuster (I.N.N.) Company Main Patent expiration date Diovan Sept. 2012 (valsartan) Micardis Top-selling Jan. 2014 (telmisartan) antihypertensive Benicar, Olmetec Oct. 2016 drugs (olmesartan) Avapro, Aprovel Mar. 2014 (irbesartan) Blopress Jun. 2012 (candesartan) Antihypertensive drugs account for 5 of 10 top-selling cardiovascular products, Pipeline lacks innovation; each with annual sales >$1 billion majority of late stage programs focused on combination Increasing generic threat creates significant therapies using existing drugs need for innovative antihypertensive therapeutics candidates 4
1 BAPAI : novel therapeutic class
BAPAI : a new therapeutic approach to treat hypertension and associated cardiovascular diseases Hypertension Heart failure Brain Aminopeptidase Other diseases A Inhibitors (BAPAIs) Quantum Genomics, the BAPAI company, is developing first in class treatments targeting a new pharmacological pathway in the brain Benefitting from more than 20 years of leading European academic research Catherine Llorens-Cortes , PhD in Neurobiology, Director of the Central 2014 Category Neuropeptides and cardio-vascular hydro- « Research team » regulation research team – College de France CIRB-CNRS UMR U1050 7241/INSERM 6
A triple mechanism of action with a single drug Inhibition of Aminopeptidase A Angiotensin II Angiotensin III Vasopressin release Sympathetic nerve activity Baroreflex BAPAI Increase of the diuresis Lowering vascular Controlling (urinary elimination) resistance heart rate Mechanism of action described in several peer reviewed BAPAIs are innovative drugs that target academic publications: a new central pharmacological pathway leading to both Bodineau & al – Hypertension – 2008 antihypertensive effects and Marc & al – ProgNeuroscience – 2011 cardioprotection Marc & al – Hypertension - 2012 7
Targeting Low Renin High Vasopressin (LRVH) patients LRHV patients (approximately 30% of total hypertensive population) have mostly uncontrolled or poorly controlled high blood pressure LRHV profile is overexpressed in elderly, Asian, African American and Hispanic populations ACEs and ARBs drugs are not effective for LRHV patients 8
2 Robust pipeline of new drug candidates
Pipeline of four R&D programs Quantum Genomics Partner Identification of Preclinical Clinical studies Clinical studies Clinical studies Clinical studies New drug Commercia- active studies Phase I Phase IIa Phase IIb Phase III approval lization compounds (NDA) QGC001 First-in-class Tolerance, safety and efficacy Treatment of In hypertensive patients hypertension as monotherapy HYPERTENSION QGC011 Combination Regulatory Preclinical results Treatment of Pharmacokinetics and toxicology hypertension (rats and dogs) in combination QGC006 Best-in-class Proof of efficacy (single dose) Optimized treatment in hypertensive rats of hypertension as monotherapy QGC101 HEART FAILURE CONGESTIVE Proof of efficacy repeated doses First-in-class (post infarction rat and dog models) Prevention and and start of phase II treatment of congestive heart failure Typically, identification and preclinical phases last 2-3 years, a phase I 1-2 years, a phase IIa 1-2 years , a Phase IIb 1-2 years, a phase III 2-3 years and new drug approval and commercialization 2-3 years. 10
QGC001, phase I trials Identification of Clinical studies Clinical studies Target discovery Preclinical studies active compounds Phase I Phase II QGC001 First-in-class Stand alone treatment of Hypertension Phase Ia • started in 2012 Positive : overall safety and tolerability Randomized, double blind, placebo controlled study of single ascending doses in 80 healthy volunteers of QGC001 up to 2g Phase Ib • started in 2013 Positive : overall safety and tolerability of QGC001 up to 750mg twice a day Randomized, double blind, placebo controlled study of multiple ascending doses in 44 healthy volunteers and no food interaction Michel Azizi, MD, Phase IIa • started in Q1 2015 Medical Doctor, University Professor – Hospital Initiation of a phase IIa Practitioner Director of CIC 9201 (Cardiovascular, in 30 hypertensive patients renal, endocrine pathology and physiology) 11
Phase IIa for QGC001: trial completed Randomization 1:1 Period P1 Washout Period P2 Tapering and discontinuation of Run-in current HTA Period A QGC001 Placebo Placebo therapy No drug Placebo treatment Period P1 Washout Period P2 B Placebo Placebo QGC001 2 weeks 2 weeks 4 weeks 2 weeks 4 weeks 4 Centers in France all labelled as “ Centers of Excellence” by the European Society of Hypertension. Principal Investigator: Pr. Michel Azizi - Hôpital Européen Georges Pompidou - Paris Safety endpoints : Evaluated from signs, symptoms and laboratory tests at each visit Pharmacokinetics endpoints : Measured twice during each of the two treatment periods Efficacy endpoints include : 24 hours ambulatory blood pressure measurement (ABPM) Home blood pressure measurement (HBPM) Office blood pressure measurement (OBPM) 12 Hormonal measurement of several biomarkers
Positive Top-line results from phase IIa in hypertension Convergence of positive signals on several endpoints Especially regarding the primary endpoint: drop in daytime systolic blood pressure measured as ambulatory pressure in hypertensive patients, treated with QGC001 as compared to placebo Positive result confirmed by in-depth multivariate analysis Quality of methodology & data Global quality of data recognized by investigators Patients are truly hypertensive Excellent quality of ABPM data No placebo effect The full results of the study will be presented at a major medical meeting. In this respect, the Company is targeting the next European Society of Hypertension meeting which will take place in June 2017 in Milan. 13
Planned next clinical trials in hypertension In the US, start of a phase II on a targeted population in 2017 Meeting with FDA : Pre-IND meeting with the FDA Reviewing Division on the Clinical, Non-clinical, and the Chemistry, Manufacturing, Control Development of the study The FDA has reviewed and analyzed the entire QGC001 documentation, including all preclinical and clinical data available to date, including phase IIa trial methodology and efficacy and tolerance data. Expect to submit an IND application for a trial mid-2017 to evaluate QGC001 in a targeted population of hypertensive patients. Data from planned phase II trial intended to support design of phase III clinical program for QGC001 in the US. Enrollment of ethnic/racial minorities in this hypertension study has been encouraged by the FDA Further targeted studies in preparation for Asia and Europe. 14
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