PharmAust completes Phase 1 MEND study and files for US Orphan Drug Designation

Latest News

Clinical-stage biotechnology company PharmAust (ASX:PAA) has announced that all patients have completed its Phase 1 MEND study of monepantel (MPL) for motor neurone disease (MND/ALS).

The company said the release of top-line results remains on track for the first quarter of the 2024 calendar year.

CEO Dr Michael Thurn said, “Excitement is growing knowing that we are only months away from releasing top-line data.

"We have several patients who have now received treatment with monepantel for 13 months or more, with the median treatment duration being 10.6 months for the 12 patients who participated in the study.

"Every month a patient continues on treatment brings enormous satisfaction and excitement around the potential benefit that monepantel may bring to patients, their families and caregivers, and of course the healthcare system as a whole.”

The Phase 1 MEND study is an open-label, multicentre study involving 12 patients with MND/ALS to determine the recommended Phase 2 dose based on safety and preliminary efficacy. The study design involves two cohorts of six patients, each progressively receiving higher dose levels of MPL in a staggered design approach over time.

The study is supported by a drug development grant of $881,085 from FightMND.

PharmAust said it has successfully submitted the protocol for the 12-month Open Label Extension study to the Human Research Ethics Committee for approval. It has also applied for an Orphan Drug Designation with the US FDA.