PharmAust progresses toward accelerated approval for MND therapy
Clinical-stage biotechnology company PharmAust (ASX: PAA) has a number of key activities lined up to further progress testing of its primary drug candidate monepantel (MPL).
The upcoming program follows the early December completion of PharmAust’s phase 1 MEND study used for treating motor neurone disease / amyotrophic lateral sclerosis (MND/ALS).
This milestone was marked by all patients completing the study, with the release of top-line results anticipated in Q1 CY24.
Early in January 2024, PharmAust scheduled a pre-investigational new drug (pre-IND) meeting with the US Food and Drug Administration regarding the use of MPL for the treatment of MND/ALS.
The pre-IND meeting aims to confirm the details and acceptability of PharmAust’s proposed ongoing development program, including the requirements for non-clinical and clinical pharmacology, clinical chemistry and manufacturing controls.
The meeting will provide PharmAust with an opportunity to seek feedback from the FDA on the design of its planned adaptive phase 2/3 clinical study and gain insights into the FDA’s requirements for MPL to be granted accelerated approval.
It also provides PharmAust with the impetus to proceed confidently with filing its full IND application in Q2 CY 2024.
Orphan drug designation
In another interaction with the FDA, PharmAust submitted a request for orphan drug designation (ODD) in November 2023 to the office of orphan products development.
The request was based on pre-clinical mechanistic data that demonstrates MPL can induce autophagy in diseased cells.
The FDA sent a response letter in late January 2024 requesting PharmAust submit additional clinical data in support of granting the ODD for the treatment of MND/ALS.
PharmAust is due to release its phase 1 MEND Study clinical data this quarter and will submit an amendment to the FDA following data release.
Open-label extension study
In a further MPL-related development, PharmAust received approval in late January from Monash Health Human Research Ethics Committee (HREC) to commence an open-label extension (OLE) study of MPL in patients with MND/ALS at Calvary Health Care Bethlehem in Melbourne.
The study is a multi-centre, 12-month analysis designed to allow the 12 patients who participated in the phase 1 MEND study to continue to receive further treatment with MPL.
Under the program, patients will receive a daily dose of 10 milligrams of MPL per kilogram of body weight for 12 months.
The OLE study will further test the hypotheses that MPL administration to individuals living with MND/ALS will safely reduce disease-associated protein accumulation in motor neurons and provide therapeutic benefits.
Phase 1 MEND results
Meanwhile, PharmAust has confirmed that top-line results from the phase 1 MEND study are on-track to be released this coming quarter.
In January 2024, PharmAust announced it would partner with leading clinical study design specialist Berry Consultants to design and analyse the planned adaptive phase 2/3 clinical study for MPL in MND/ALS patients.
Berry Consultants will be involved in the statistical analysis of the phase 1 MEND study data.
New animal drug application
PharmAust has also confirmed it plans to open an investigational new animal drug (INAD) file with the FDA for MPL as a treatment for canine B-cell lymphoma.
Sponsors of new animal drugs will typically submit a request to open an INAD file when they have enough pilot data to start discussing the development process with the FDA and/or they want to begin shipping the drug for use in investigational studies in the US.