Michael R. Hayden, Netherlands

Prilenia Therapeutics BV .
Dr. Hayden is a Killam Professor at the University of British Columbia, the highest honour UBC can confer on any faculty member. Only 4 such awards have ever been conferred in the Faculty of Medicine. Dr. Hayden is also Canadian Research Chair in Human Genetics and Molecular Medicine. Dr. Hayden was the President of Global R&D and Chief Scientific Officer at Teva from 2012-2017. During this time, approximately 35 new products were approved in major markets with many for diseases of the CNS and led the approval of Austedo for chorea in HD, the second drug ever to be approved for HD. Dr. Hayden was named one of the 50 Canadians born in the 20th century who have changed the world. Dr. Hayden is the co-founder of five biotechnology companies including: Prilenia, NeuroVir Therapeutics Inc., Xenon Pharmaceuticals Inc., Aspreva Pharmaceuticals Corp, and 89bio. He currently sits on different public and private boards of biotechnologies companies. Author of approximately 900 peer-reviewed publications and invited submissions, Dr. Hayden has focused his research primarily on translational medicine, including genetics, lipoprotein disorders, Huntington disease, predictive, personalized medicine and drug development. Dr. Hayden and his research group have identified 10 disease-causing genes which includes the identification of the major gene underlying high-density lipoprotein (HDL) in humans. Dr. Hayden also identified the first mutations underlying Lipoprotein Lipase (LPL) Deficiency and developed gene therapy approaches to treat this condition resulting in the first approved gene therapy product (Glybera) in a major market. Dr. Hayden has been the most cited author in the world on ABCA1, and Huntington Disease since 2006. Dr. Hayden is the recipient of numerous prestigious honours and awards. Most recently, Dr. Hayden was awarded the David Dubinsky Humanitarian Award from the American Friends of Soroka Medical Center (AFSMC). He was inducted into the Canadian Medical Hall of Fame in 2017. He was named one of PharmaVoice’s “100 of the Most Inspiring People” (2015); awarded an Honorary Doctorate of Science by the University of Gottingen (2014); the Luminary award by the Personalized Medicine World Conference (2014); the Diamond Jubilee Medal (2012), on behalf of HRH Queen Elisabeth II, the Killam Prize by the Canada Council of the Arts (2011), in recognition of his outstanding career achievements; and the Canada Gairdner Wightman award (2011). Dr. Hayden has also been awarded the Order of Canada (2011), and the Order of British Columbia (2010). He was named Canada’s Health Researcher of the Year by CIHR (NIH of Canada) in 2008, and he received the Prix Galien in 2007. Dr. Hayden is committed to empowering others. In addition to mentoring over 100 graduate students and postdocs, he is also a TED mentor.

Presenter of 2 Presentations

THE PROOF-HD STUDY: PRIDOPIDINE’S OUTCOME ON FUNCTION IN HUNTINGTON DISEASE

Session Type
SYMPOSIUM
Date
12.03.2021, Friday
Session Time
12:00 - 14:00
Room
On Demand Symposia B
Lecture Time
13:30 - 13:45
Session Icon
On-Demand

Abstract

Aims

Background: Pridopidine is a well-tolerated, potent, selective sigma-1 receptor (S1R) agonist. The S1R regulates key cellular processes implicated in neurodegeneration including reducing ER stress and enhancing mitochondrial function. Pridopidine is the first drug to find a beneficial effect on TFC, a validated scale measuring functional capacity that is relevant to both patients and clinicians, in long-term HD trials. In post-hoc analysis of the PRIDE-HD trial, pridopidine 45mg bid had improved TFC scores vs placebo in early HD participants (HD 1+2) at 52 weeks (change from placebo 1.16, nominal p=0.0003). Responder analysis shows that pridopidine reduced the probability of TFC decline (1 or more points) by 80% (nominal p=0.02, week 52). Exploratory endpoints cUHDRS (combined score of cognitive, motor and function; nominal p=0.04) and Q-Motor assessment measuring motor function at 26 and 52 weeks (nominal p=0.02, p=0.195, respectively) also suggest beneficial effects.

Aim: To assess the effect of pridopidine 45mg bid on TFC in patients with early stage HD.

Methods

Design: PROOF-HD is a Phase 3, 65-week, randomized, double-blind, placebo-controlled trial. The primary endpoint is the mean change from baseline to week 65 in TFC. Secondary endpoints include change from baseline in Total Motor Score (TMS), cUHDRS, and Q-Motor to week 65.

Results

The target population is early HD (TFC≥7) participants with an expected TFC decline of -0.8 to -1 points/year.

Conclusions

We use data from prior trials where pridopidine has showed clinically meaningful and nominally significant benefit on endpoints to maximize likelihood of success in the design of the PROOF-HD trial.

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