18 September | 2019
Prilenia’s Pridopidine chosen to participate in the first ALS platform trial by the Sean M. Healey & AMG Center for ALS at Mass General
The Sean M. Healey & AMG Center for ALS at Mass General aims to speed up the search for new approaches to help patients with amyotrophic lateral sclerosis (ALS), through this groundbreaking new trial design which allows for rapid testing of multiple drugs through use of a common trial design
[Herzliya & Boston, 18 September 2019] Prilenia, a newly formed biotech company focused on developing novel treatments for neurodegenerative and neurodevelopmental disorders announces that Pridopidine, its lead compound, has been selected, as the first of three potential new treatments to be included in the launch of the first ever platform trial in amyotrophic lateral sclerosis (ALS). Two additional compounds were chosen to join the trial at a later stage.
Pridopidine was chosen by an independent review committee out of 30 competing investigational treatments based on human genetic data, efficacy in preclinical models, favorable safety profile and readiness of drug supply. Pridopidine is a highly selective S1R agonist, and this mechanism has already been shown to provide some benefit in ALS patients.
This innovative platform trial is led by the Sean M. Healey & AMG Center for ALS at Mass General Hospital. The trial design aims to accelerate the development of effective treatments for people living with ALS, by testing and evaluating multiple treatments simultaneously.
“This groundbreaking trial brings hope to ALS patients. The selection of Pridopidine validates the potential for this drug to impact common pathways across neurodegenerative diseases,” says Michael R. Hayden, MD, PhD, Executive Chairman of Prilenia. “We are honored to be a part of this innovative platform trial hosted by the Healey Center and the NEALS organization, a world leader in running ALS clinical trials.”
“A subset of the Healey Science Advisory Committee and NEALS scientific advisory board reviewed the almost 30 applications from 10 countries to select the top candidates ready for testing in people today,” says Merit Cudkowicz, MD, MSc, director of the Sean M. Healey & AMG Center for ALS at Mass General. “We believe that this platform trial approach will accelerate how therapies are tested and look forward to advancing this important work.”
The HEALEY ALS Platform Trial will be the second clinical trial initiated by Prilenia. Earlier this year, Prilenia launched a phase 2 clinical trial in the US to evaluate the safety and efficacy of Pridopidine in treating Levodopa Induced Dyskinesia in patients with Parkinson’s Disease.
Partial financial support to initiate these first treatments is made possible thanks to the generosity of the Healey family and friends and the AMG Charitable foundation along with partners at TackleALS.
Pridopidine a first-in-class drug candidate with an established safety profile and therapeutic potential in several neurodegenerative diseases affecting adults and children. Pridopidine is a highly selective S1R agonist, shown to exerts neuroprotective effects in numerous models of neurodegenerative disorders mediated via the S1R. S1R validation as a therapeutic target for ALS is demonstrated in animal models and in humans.
Compelling preclinical data supports a therapeutic potential for pridopidine in ALS, In ALS SOD1G93A motor neurons (MNs), pridopidine exerts neuroprotective effects via activation of the S1R. Specifically, pridopidine improves BDNF and GDNF axonal transport, restores synaptic activity and the neuro-muscular junction (NMJ) function and increases neuronal survival. In-vivo, pridopidine treatment of SOD1G93A mice reduces toxic protein aggregates and ameliorates muscle fiber wasting. Clinical support for the validity of the S1R as a potential target for ALS can be learned from previous data suggesting S1R activation may enhance bulbar and speech function in ALS patients.
Pridopidine is also the first drug to show a statistically significant effect on maintenance of functional capacity in early HD, as measured by Total Functional Capacity (TFC).
Pridopidine was acquired by Prilenia from Teva in 2018 and is currently in Phase 2 clinical development for the treatment of patients with Parkinson’s Disease suffering from Levodopa Induced Dyskinesia (PD-LID). In addition to the Healey platform trial in ALS, Prilenia is planning to initiate a phase 3 trial in Huntington Disease in the near future.
Background on ALS
Amyotrophic lateral sclerosis, ALS, is the most prevalent adult-onset progressive motor neuron disease, affecting approximately 30,000 people in the U.S. and an estimated 500,000 people worldwide. ALS causes the progressive degeneration of motor neurons, resulting in progressive muscle weakness and atrophy. There are currently three FDA therapies approved specifically for treating ALS—riluzole, nuedexta and edaravone.