PharmAust just got a pre-IND meeting with the US FDA to kick off ‘24

US FDA grants PharmAust a pre-IND meeting for monepantel for treatment of MND/ALS
Meeting will provide feedback on PharmAust proposed development program to receive accelerated approval
PharmAust on-track for opening an IND application and initiating adaptive Phase 2/3 clinical study

Special Report: PharmAust has kicked off 2024 with promise after the US FDA granted the clinical-stage biotech a pre-investigational new drug (pre-IND) meeting for monepantel to treat motor neurone disease/amyotrophic lateral sclerosis (MND/ALS).

PharmAust (PAA) says 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 the requirements for non-clinical and clinical pharmacology, clinical chemistry, and manufacturing controls.

Reviewers from the FDA’s Office of Neuroscience will provide feedback on PAA’s proposed development program for monepantel.

PAA says the request was submitted on December 15, 2023 and the FDA has committed to provide written responses by February 13, 2024.

Importantly, and given the stage of development of monepantel for the treatment of MND/ALS, the pre-IND meeting provides PAA with an opportunity to seek feedback from the FDA on the design of its planned Phase 2/3 adaptive clinical study.

The company will also gain insights into the FDA’s requirements for monepantel to be potentially granted accelerated approval by the regulator.

PAA says with these considerations in hand it will be able to proceed confidently with its full IND application filing in Q2 2024.

Phase 2/3 study to assess drug efficacy

PAA’s phase 2/3 study is a multicentre, randomised, placebo-controlled, adaptive clinical study evaluating the safety and efficacy of monepantel in patients with MND/ALS over 48 weeks.

The primary aim will be to assess the efficacy of monepantel, as compared to placebo, on the progression of MND/ALS.

PAA says the efficacy will be assessed as a change from baseline disease severity measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) total score and survival.

Being an adaptive study design, an interim analysis will be performed at Week 24 by a team of unblinded statisticians for the potential to stop the study early for either success or futility.

Australia’s leading not-for-profit foundation for motor neurone disease research FightMND recently invited PAA to submit a full grant application to help cover costs of the study.

Up to $1.8 million in support from FightMND is available per grant – and a successful application could play a key role in assisting PAA in development of monepantel.

According to the International Alliance of ALS/MND Associations, MND affects over 350,000 people globally and kills more than 100,000 people yearly.

The disease is invariably fatal, with no cure nor effective treatment to reverse its progression. The average life expectancy of someone with MND is around 27 months from first diagnosis, while independent studies have shown that one-third of patients die within 12 months.

PAA says the MND/ALS addressable market is US$3.6 billion per annum, with the standard of care treatment, Riluzole, only prolonging life on average by 2-3 months.

One study away from accelerated approval

PAA CEO Dr Michael Thurn says the company is looking forward to receiving feedback from the FDA.

“It’s remarkable to think that PharmAust is potentially one clinical study away from receiving accelerated approval and providing MND/ALS patients with a much-needed new therapy for a condition that is invariably fatal,” Thurn says.

“Receiving feedback from the FDA in February positions us well to achieve our near-term milestone of opening an IND in Q2 2024 with the goal of initiating the planned adaptive Phase 2/3 clinical study in patients shortly after that.”

 

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.

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