- PharmAust announces positive Phase 1 data in trial to treat MND/ALS
- Preliminary efficacy data shows a 58% reduction in the rate of disease progression
- PAA submitted the new data to US FDA for Orphan Drug Designation
- Company makes key appointments and establishes scientific advisory board
- Development & manufacturing arrangements completed for Phase 2/3 trial
Special Report: PharmAust has made significant progression on the clinical trial front last quarter with the highlight being positive top-line results from its Phase 1 MEND study of primary drug candidate monepantel (MPL) to treat motor neuron disease/amyotrophic lateral sclerosis (MND/ALS).
In February, clinical stage biotech PharmAust (ASX:PAA) announced results from the MEND study revealing MPL’s superior safety profile and tolerability to FDA-approved MND/ALS drug Relyvrio.
Preliminary efficacy data shows a 58% reduction in the rate of disease progression for the high-dose cohort.
MPL and its active metabolite were detected in the cerebrospinal fluid, indicating the compounds’ ability to cross the blood-brain barrier.
PAA says the significant finding has led to identifying an optimal dose for its upcoming pivotal Phase 2/3 clinical study, slated to begin later in CY24.
PAA’s cash position at March 31, 2024 was $3.94m with available funding of $4.88m.
During the quarter, payments for R&D equated to $1.92 million representing costs involved with the development of MPL and preparations for commencement of the Phase 2/3 clinical trial.
PAA submits clinical data to FDA
In March, PAA submitted additional clinical data to the US FDA to support its orphan drug designation (ODD) request for MPL following its previous request for more data, with a response expected by mid-June 2024.
ODD is given to a drug or biological product to prevent, diagnose or treat a rare disease or condition.
For the pharmaceutical company, ODD incentives include market exclusivity for several years along with tax credits for qualified clinical trials.
PAA says recent clinical trial failures of leading FDA-approved drugs for MND/ALS highlight the significant unmet need for effective treatments, positioning MPL as a potential alternative.
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.
Importantly, 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.
MND Extension study starts
During the quarter, PAA commenced an open-label extension (OLE) study for MPL after receiving approval from the Monash Health Human Research Ethics Committee.
The 12-month multicentre OLE study includes patients from the Phase 1 MEND study invited to continue treatment. The patients will receive a daily dose of 10 mg/kg body weight of MPL for a year.
PAA says the OLE study is important to gather long-term safety and tolerability data and to assess both the therapeutic benefits and biomarkers related to the disease.
Berry consultants appointed and board updates
In January PAA announced it was partnering with leading MND/ALS clinical study design and statistical analysis specialists Berry Consultants as it focuses on moving forward toward its Phase 2/3 MND/ALS study later this year.
Berry Consultants has a strong track record of innovative clinical trial design and experience with the FDA.
In February, PAA announced the appointment Dr Thomas Duthy as a non-executive director
to its board, as it works to enhance its governance and business development acumen.
Duthy has extensive experience in the biotech sector along with corporate advisory and investor relations. He is the executive director of Neurotech International (ASX:NTI) and is also on the boards of other ASX-listed life sciences companies.
During the quarter PAA also announced formation of its Scientific Advisory Board (SAB).
Including internationally renowned experts in MND/ALS drug discovery and clinical development, the SAB will provide strategic guidance to PAA in the development of MPL.
Post-quarter updates
In April PAA announced the appointment of Dr Herbert Brinkman as head of manufacturing, bringing more than 30 years of pharmaceutical product development experience.
PAA has also inked manufacturing process development deals with Syngene International and Catalent Pharma Solutions, global leaders in GMP manufacture and commercial supply of pharmaceutical products.
Under the agreements Syngene will manufacture 60kg of GMP monepantel consisting of one 15kg engineering batch followed by three 15kg process validation batches designed to validate the GMP manufacturing process, support product registration and prepare PAA for commercial supply.
Catalent will be responsible for the GMP manufacture of three registration batches, totalling more than a million tablets, which will be required to support product registration and facilitate commercial scale-up activities.
Last week the company announced the early exit of Dr Michael Thurn as CEO.
‘Strong foundation to push forward’
Interim CEO John Clark says the March quarter was particularly important and successful for PAA headlined by its positive Phase 1 MEND trial top-line trial data.
“This gives us a strong foundation to push forward with both the open-label extension study and the pivotal adaptive Phase 2/3 clinical trial,” he says.
“The positive FDA feedback has provided us with a clear path towards potential accelerated approval.”
He says bolstering the broader team in recent months with key appointments to management will equip PAA to continue advancing the clinical development of MPL as planned.
“The early exit of Dr Michael Thurn as CEO has been accommodated within the current team,” he says.
“We remain on schedule for the commencement of our pivotal adaptive Phase 2/3 clinical trial starting in 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.