FDA offers PharmAust clear guidance for orphan drug designation approval

FDA seeks clinical trial data from PharmAust to support its Orphan Drug Designation in the treatment of MND/ALS
PharmAust to unveil Phase 1 MEND Study clinical data this quarter and will submit an FDA amendment
Company granted option to submit an amendment instead of reapplying with an entirely new application

Special Report: PharmAust says it is encouraged by clear and concise feedback from the US Food and Drug Administration (FDA) on what is required to receive orphan drug designation (ODD) for monepantel (MPL) to treat Motor Neurone Disease (MND) / Amyotrophic Lateral Sclerosis (ALS).

Clinical-stage biotech PharmAust (ASX:PAA) says the US Food and Drug Administration (FDA) Office of Orphan Products Development has requested additional data to support the
MPL ODD application for the treatment of MND/ ALS.

PAA says the request was made due to the absence of preclinical or clinical data to establish the potential for the drug to be effective in MND/ALS.

The FDA grants ODD status to assist and encourage companies to develop safe and effective treatments for rare diseases and disorders (impacting less than 200,000 persons in the US).

Under the US Orphan Drug Act, ODD provides incentives, including tax credits, grants, waiver of some administrative fees for clinical trials and seven years of market exclusivity following drug approval.

PAA submitted the request for ODD in November 2023 to the FDA’s Office of Orphan Products Development.

The application was based on preclinical mechanistic data that demonstrates MPL can induce autophagy in diseased cells, along with consideration of the pathology of the disease.

The FDA’s reply states it could not grant the ODD at this stage and requests preclinical data in a relevant animal model or clinical data from human trials, to establish the potential for the drug to be effective in MND/ALS.

The FDA has provided PharmAust with a pathway to address the absence of clinical data by submitting an amendment to the ODD request, rather than reapplying with an entirely new application.

In pre-clinical programs, PharmAust has already shown that MPL can activate molecular pathways relevant to its development as a MND therapeutic drug. If effective, MPL would reduce the rate of degeneration and loss of motor neurons in the anterior horns and motor nuclei of the brainstem.

MEND study results to address FDA request

PAA’s Phase 1 MEND study top-line results are on track to be released before the end of this current quarter, with the company planning to submit the results to the FDA to support its ODD request.

The Phase 1 MEND Study involved 12 patients with MND/ALS and was completed in December 2023.

All patients have since elected to continue to receive MPL under a compassionate-use program and are eligible to enrol in a 12-month open-label extension study due to start in February 2024.

Patients have been treated with MPL for an average of 13 months, and it continues to be well-tolerated by these patients.

‘Encouraged by the clear and concise feedback’

PAA chief executive Dr Michael Thurn says the company looks forward to releasing its Phase 1 MEND Study clinical data in the coming weeks and responding to the FDA to complete the ODD application.

“The company is encouraged by the clear and concise feedback from the FDA on what is required for the ODD to be granted,” he says.

“We appreciate the FDA’s feedback on our development programs, which allows us to refine our strategies to maximise our opportunities for blockbuster success.

“Over the coming weeks, we will continue our communication strategy with the FDA about the ODD and the pre-IND meeting for our adaptive Phase 2/3 study.”

Need for MND/ALS treatment

According to the International Alliance of ALS/MND Associations, MND affects more than 350,000 people globally with more than 100,000 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 also this month announced it was partnering with leading MND/ALS clinical study design and statistical analysis specialists Berry Consultants as it focuses on moving towards 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 and will oversee design of its adaptive Phase 2/3 trial.

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.