PharmAust’s breakthrough MND/ALS study showcased at major European medical conference
The success of PharmAust’s (ASX: PAA) Phase 1 MEND study into the treatment of motor neurone disease/amyotrophic lateral sclerosis (MND/ALS) is being showcased at a major global gathering of medical professionals in Stockholm, Sweden.
Texas-based clinical trial design specialists Berry Consultants are presenting data from the Phase 1 MEND study at the annual meeting of the European Network to Cure ALS (ENCALS).
This event is the largest and most significant MND/ALS conference in Europe.
Statistical comparisons
A poster presentation by Drs Philip White and Melanie Quintana is highlighting the statistical comparisons made between patients treated with monepantel (MPL) in PharmAust’s recently completed study.
“To have the clinical data from our successful Phase 1 MEND study presented at a major international conference such as ENCALS is a major achievement for the company,” said PharmAust managing director Dr Michael Thurn.
“We are very grateful to Berry Consultants for independently presenting the data and creating greater awareness about our promising efficacy data within the broader MND/ALS community.”
“We look forward to working with [them] as we move closer to finalising the design of the pivotal adaptive Phase 2/3 STRIKE study planned to commence in the second half of 2024.”
Survival benefits
Earlier this month, PharmAust revealed it had received encouraging survival benefit assessments for its MPL neurodegenerative disease treatment.
The study undertaken by Berry Consultants identified that treatment with MPL significantly reduced the risk of death by 91% compared to untreated matched controls from the pooled resource open-access ALS clinical trials (PRO-ACT) database.
The study concluded that the survival patterns between MPL treatment and PRO-ACT control groups differ significantly for each dataset tested, even using the most conservative analysis dataset.
New OLE study
PharmAust is currently preparing to undertake an open-label extension (OLE) study.
Ten of the 12 patients who completed the Phase 1 MEND study have been successfully enrolled for the next phase to investigate the long-term safety, tolerability and efficacy of MPL.
PharmAust plans to dose eligible patients with 10 milligrams of MPL per kilogram of body weight daily for an additional 12 months.
The OLE study involves two sites in Australia—Calvary Health Care Bethlehem (led by Associate Professor Susan Mathers) and Macquarie University, led by Professor Dominic Rowe.