‘Outstanding milestone’: FDA grants PharmAust orphan drug designation for monepantel

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PharmAust receives US FDA orphan drug designation for monepantel (MPL) to treat motor neurone disease
Application based on preclinical mechanistic data showing that MPL can induce autophagy in diseased cells
PharmAust preparing for upcoming pivotal Phase 2/3 clinical study, forecast to start in H2 CY24


Special Report: PharmAust has been granted orphan drug designation (ODD) by the US Food and Drug Administration (FDA) for its drug monepantel (MPL) aimed at treating motor neurone disease (MND), also known as amyotrophic lateral sclerosis (ALS).

Clinical stage biotech PharmAust (ASX:PAA) has announced it has achieved a key catalyst with lead drug MPL granted FDA approval to treat MND/ALS.

The FDA’s ODD status is designed to support the development of treatments for rare diseases affecting fewer than 200,000 people in the US.

Benefits include tax credits, grants, waived clinical trial fees, and seven years of market exclusivity upon drug approval.

PAA’s ODD application was based on preclinical mechanistic data showing that MPL can induce autophagy in diseased cells and consideration of the pathology of the disease.

The company initially submitted its request to the FDA in November 2023. Following a request for additional data in January 2024, PAA provided positive clinical data from its Phase 1 MEND study in March.


Significant drop in disease progression

In February PAA announced positive top line results involving 12 MND/ALS patients with

preliminary efficacy data showed a 58% reduction in the rate of disease progression for the high-dose cohort.

Furthermore, the results showed that daily administration of MPL over seven to 12 months was well-tolerated with no serious side effects.

MPL and its active metabolite were detected in the cerebrospinal fluid (CSF) NfL biomarkers, indicating the  compounds’ ability to cross the blood-brain barrier and a meaningful clinical effect.

PAA says the significant finding in the MENDS study has led to identifying an optimal dose for its upcoming pivotal Phase 2/3 clinical study, slated to begin later in CY24.


Clear FDA pathway for full MPL approval

Following a productive Pre-Investigational New Drug (Pre-IND) meeting with the FDA in February, PAA also now has a clear pathway towards accelerated and full MPL approval.

The FDA provided positive feedback on PAA’s proposed development program for MPL including specific guidance on requirements for non-clinical and clinical pharmacology, clinical chemistry, and manufacturing controls.

The FDA indicated that the forthcoming adaptive Phase 2/3 clinical study could potentially support accelerated approval of MPL, provided the study demonstrates substantial evidence of effectiveness and an adequate safety profile.

The pivotal and adaptive Phase 2/3 study will be a global study with clinical sites planned in Australia, Europe, and the US.


Great need for treatment

MND/ALS is an invariably fatal nervous system disease that weakens muscles and impacts physical function, which affects more than 350,000 people globally.

Sufferers have an average life expectancy of about 27 months with FDA approved treatments Riluzole and Relyvrio only able to prolong life by about 2-3 months and 10 months respectively.

PAA first identified the potential of MPL to treat MND/ALS after pre-clinical studies found the drug could activate molecular pathways relevant to its development as a MND therapy, leading to launch of the MEND study.

The MND/ALS treatment market was more than US$9 billion in 2022 and is forecast to reach US$23bn by the end of 2035.

In 2021, the median treatment cost was US$218,872 for orphan drugs (ranging from US$237 to US$1,272,021).

PAA says the last three drugs that received FDA approval for the treatment of MND/ALS (Qalsody, Radicava, and Relyvrio) each have an annual cost of between US$150,000-$200,000, with Relyvrio having generated US$381m in sales during 2023, its first year after launch.

In April Amylyx Pharmaceuticals announced that it had begun the process of voluntarily withdrawing Relyvrio from the market after it failed to prove efficacy in a large clinical trial.


‘Outstanding milestone for PharmAust’

PAA managing director John Clark says the ODD is a major milestone for PharmAust as it works to improve treatment options for MND/ALS sufferers globally.

“This is an outstanding milestone for PharmAust  and  monepantel, providing  an even  stronger pathway forward for the drug, particularly  in  light of recent failures of other MND/ALS treatments.

“We are now increasingly optimistic as we progress to our pivotal registration adaptive Phase 2/3 study which will commence in H2 CY2024.”



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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