PharmAust progresses MPL programs for treatment of motor neurone disease and canine oncology
Biotechnology company PharmAust (ASX: PAA) has progressed its monepantel (MPL) clinical programs for motor neurone disease and canine oncology during the period to end September.
The company’s Phase 1 MEND motor neurone disease study was completed to the third dose level cohort and “encouraging” safety and interim biomarker analyses indicated a positive treatment effect.
Three of the four dose level cohorts have now been completed with no safety concerns and despite some patients receiving treatment continuously for over 12 months.
A safety monitoring committee connected to the trial recommended PharmAust proceed to the fourth and final dose level cohort of 10 milligrams per kilogram bodyweight.
Subsequent to the quarter, patients in that cohort completed dosing with no safety concerns.
They have one more follow-up visit scheduled before data collation and analysis begins.
Top-line data is expected to be released in the new year.
Meanwhile, PharmAust is finalising a 12-month open label extension (OLE) MEND study for ethics approval.
All 12 patients from the Phase 1 arm are expected to take part.
Phase 1 MEND is an open label, multi-centre trial involving 12 patients with the goal of determining a Phase 2 dose of MPL based on safety and preliminary efficacy.
It is supported by an $880,000 grant from independent funding body FightMND, with the final instalment of $150,142 due for receipt this week.
During the quarter, PharmAust provided updates on the progress of a Phase 2 study trialling MPL as a daily treatment for canines with B-cell lymphoma.
The company noted that cancer in one dog had remained stable after 12 months of treatment while other data showed that treatment with MPL can extend survival rates up to a median of 150 days.
“A distinguishing feature of the dogs treated with MPL is the excellent quality of life they can experience… this is in stark contrast to what dogs experience when subjected to expensive chemotherapy regimens,” it said.
Subsequent to the quarter, PharmAust announced the Phase 2 study data had presented “encouraging top-line results” showing that daily treatment with MPL compared favourably to Laverdia, which is the most recent B-cell and T-cell lymphoma treatment approved by the US Food and Drug Administration.
The data will be used to open an investigational new animal drug (INAD) application with the FDA’s Centre for Veterinary Medicine and for the start of MPL registration studies in the new year.
In August, PharmAust appointed Dr Michael Thurn to the role of chief executive officer.
Dr Thurn has been involved with drug discovery, development, regulation and commercialisation activities through leadership roles with research organisations and publicly-listed biotechnology companies.
He is experienced in executing Phase 1 and Phase 2 clinical trials and business development strategies for animal and health products.
Dr Roger Aston remains chairman of the PharmAust board and has transitioned into a non-executive role.
He continues to advise the company using his 40 years of experience in the pharmaceutical and healthcare industry.
PharmAust announced the voluntary liquidation of wholly-owned subsidiary Epichem in August, following the loss of a research contract which funded new treatments for neglected diseases.
As Epichem was unable to secure a new long-term contract to replace the funding, PharmAust opted to dissolve the company so it could focus on the clinical development of MPL.
PharmAust’s cash position at end September was $1.72 million with total available funding for future activities of $2.2 million.
The final FightMND grant instalment is expected this week while a research and development tax incentive payment of $553,000 will be received next month.
PharmAust expects to raise an additional $396,000 under an options offer to existing listed option holders.
The company spent $420,000 during the period on costs associated with the development of the primary drug candidate MPL.