PharmAust has received additional MPL tablets to ensure it has enough availability for its Phase 1/2 MND trial and future human trials.
Clinical-stage biotech PharmAust (ASX:PAA) said pre-release technical specifications of the current Good Manufacturing Practice (cGMP) grade monepantel (MPL) tablets demonstrate that the formulation, purity, and stability specification required have been achieved.
Receipt of the roughly 10,000 MPL tablets ensures continued availability for the Phase 1/2 MND trial and ongoing maintenance dosage along with sufficient supply for other human clinical trials.
The study aims to determine the tolerability, safety, pharmacokinetics, and preliminary efficacy of oral MPL for the treatment of Motor Neurone Disease/Amyotrophic Lateral Sclerosis (MND/ALS).
The trial comprises four cohorts with escalating doses – with the tablets being well tolerated by patients so far.
PAA said so far, all patients have elected to remain on MPL post Day 29 and wish to continue receiving the tablets.
Continuation of access and treatment with the investigational product is a permitted option in the protocol.
Hope for MND/ALS sufferers
According to the International Alliance of ALS/MND Associations, MND affects ~350,000 people globally and kills more than 100,000 people annually with no cure.
The average life expectancy of someone who has MND is around 27 months with the disease progressive and symptoms getting worse over time.
The total MND/ALS addressable market is US$3.6 billion per annum with current ALS drug on the market Riluzole already reaching ~US$1 billion in annual sales.
PAA demonstrated in its preclinical programs that MPL could potentially reduce the rate of degeneration, and notes that patients have been dosed with MPL for up to six months in the clinical trial with no signs of material adverse events and are “stable”.
PAA hopes that in due course MPL could receive orphan drug designation by the TGA and FDA for motor neurone disease with such designations coming with financial and supportive benefits.
Multiple avenues to a $1Bn drug
Not only does PharmAust have clinical trials underway in MND in humans and cancer in canines, the Company is set for further trials in human cancer to commence this calendar year. This provides PharmAust with multiple ‘shots on goal’ at a +$1Bn drug success.
PharmAust previously advised that it had identified a Principal Investigator in the United States to evaluate MPL tablets in human Phase 2 cancer trials, as a follow on from the Phase I clinical trial undertaken at the Royal Adelaide Hospital in 2015.
Todays announcement of additional tablets manufactured is another step forward on commencing these trials
Further, the mid-point outcomes in the MND trial which are expected next month, will include information on absorption and pharmacokinetics of MPL and this data will facilitate phase 2 human clinical trials in cancer.
GMP tablet manufacture key for trials
GMP tablet manufacture is a key component for undertaking Good Clinical Practice (GCP) trials and will enable the data emerging from forthcoming trials to be admissible to the US Food and Drug Administration (FDA) to support new drug registration programs.
Furthermore, adoption of GMP standards ensures products meet the highest standards of safety and efficacy.
The MPL tablets were manufactured in collaboration with Syngene International, an integrated research, development and manufacturing services company along with Catalent Pharma Solutions (NYSE:CTLT).
The tablets will be stored in the stability chamber at PAA’s wholly owned subsidiary, Epichem Pty Ltd in Perth.
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.