PharmAust partners with US-based Berry Consultants for Phase 2/3 study
Health & Biotech
Health & Biotech
Special Report: PharmAust teamed up with US-based clinical study design specialists Berry Consultants to design and analyse its planned adaptive Phase 2/3 clinical study for monepantel in patients with Motor Neurone Disease (MND)/Amyotrophic Lateral Sclerosis (ALS).
Clinical-stage biotech PharmAust (ASX:PAA) is partnering with leading MND/ALS clinical study design and statistical analysis specialists Berry Consultants as it focuses on moving forward toward its Phase 2/3 MND/ALS study in 2024.
PAA says Berry Consultants has a strong track record of innovative clinical trial design and experience with the FDA.
The consultancy recently was involved in the advisory committee meeting for the recent US FDA-accelerated approval of NASDAQ-listed Amylyx Pharmaceutical’s Relyvrio for MND/ALS.
Berry Consultants has also played a crucial role in the Healey ALS Platform Trial, a pioneering initiative that tests various experimental treatments concurrently and continuously, an option that may be available to PharmAust in the future.
The Healey ALS Platform Trial saw the Healey & AMG Center for ALS at Massachusetts General Hospital assemble a team of experts to dramatically rethink how early phase trials in MND/ALS are performed and to accelerate a path to effective treatments.
While traditionally each trial evaluates only one drug at a time, and requires lengthy start-up and execution times, platform trials instead are trials where multiple drugs are tested at the same time, using specialized statistical tools. New regimens (drugs) can be added as they become available, thereby decreasing or eliminating the gap in time from identification of a rationale therapy to testing.
More can be read about the Healey ALS Platform Trial here.
Based in Austin, Texas, Berry Consultants is a statistical consulting firm specialising in clinical trial design, analysis, implementation and software solutions for pharmaceutical and medical device sectors.
The consultancy is staffed with globally recognised specialists in Bayesian statistics and focused on establishing benchmarks in adaptive clinical trial design and analysis across medical industry.
PAA says Berry Consultants is overseeing design of PAA’s adaptive Phase 2/3 trial to best position monepantel for FDA approval for the treatment of MND/ALS.
The lead statistician for PAA’s Phase 2/3 study will be Berry Consultants director and senior statistical scientist Dr Melanie Quintana, who is an expert in innovative clinical trial design for rare neurodegenerative diseases.
PAA recently announced that it had secured a Pre-Investigational New Drug (Pre-IND) meeting with the FDA.
The pre-IND meeting formally initiates communications with the FDA regarding developing monepantel to treat MND/ALS and aims to confirm details and acceptability of its proposed ongoing development program, including requirements for non-clinical and clinical pharmacology, clinical chemistry, and manufacturing controls.
According to the International Alliance of ALS/MND Associations, MND affects more than 350,000 people globally with more than 100,000 people deaths annually.
The disease is invariably fatal, with no cure or effective treatment to reverse its progression.
The average life expectancy of someone with MND is around 27 months from first diagnosis, while independent studies showing that one-third of patients die within 12 months.
PAA CEO Dr Michael Thurn says Berry Consultants’ reputation and experience in designing and conducting the statistical analysis of MND/ALS studies is “unmatched”.
“Berry Consultants has partnered with the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital,” he says.
“The Center is currently running the largest ALS study ever undertaken, the Healey ALS Platform Trial, which is focused on accelerating the development of promising new drugs for patients with ALS.
“Berry Consultants was integral in the concept, design, and continual evolution of that study in addition to numerous other MND/ALS programs.”
Berry Consultants director and senior statistical scientist Dr Melanie Quintana says the consultancy is excited to partner with PharmAust in the design of its Phase 2/3 study in MND/ALS.
“ALS is a devastating disease in which there is a clear unmet medical need for treatments that slow and ultimately stop the progression of the disease,” she says.
“Our goal in the design of this study is to rigorously investigate as efficiently as possible whether or not monepantel is effective in slowing ALS disease progression through the use of adaptive design.”
This article was developed in collaboration with PharmAust, a Stockhead advertiser at the time of publishing.
This article does not constitute financial product advice. You should consider obtaining independent advice before making any financial decisions.