Progress update on Phase 1/2 MND interim analysis
Released
Released
Announcement PDF
Progress update on Phase 1/2 MND interim analysis
• PharmAust confirms that all blood samples from the interim analysis have
been collected and submitted for analysis
• Analysis of how the MPL is absorbed, distributed, metabolised and
eliminated by the body are expected within the week
• An analysis evaluating changes in biomarkers and pharmacodynamics is
still pending
• PharmAust will continue with MPL dose escalation for Cohorts 3 and 4 to
determine the optimum dose for a Phase 2 trial
• A favourable interim analysis will allow PharmAust to prepare for a Phase
2 MND clinical trial and a Human Cancer Phase 2 clinical trial
15 May 2023 – Perth, Australia: PharmAust Limited (ASX: PAA & PAAO), a clinical-stage
biotechnology company, provides a progress update on its Phase 1/2 clinical trial of its lead drug
candidate monepantel (MPL) in Motor Neurone Disease/Amyotrophic Lateral Sclerosis (MND/ALS).
As announced on 2 March 2023, the Principal Investigator recommended undertaking an interim
analysis of preliminary biomarkers and efficacy markers on completion of dosing of the last patient of
Cohort 2.
PharmAust confirms that all blood samples from the interim analysis have been collected from all 12
patients in Cohorts 1 and 2 and have been submitted for analysis. Results will be announced to the
market as they are received. The Company anticipates the pharmacokinetics study results will be
available within the week.
Treatment-related changes from baseline in this safety, tolerability, pharmacokinetic and preliminary
efficacy study will include an analysis of functional rating scales, quality of life and cognitive
assessment. Further, prognostic indicators and several disease-related biomarkers will be measured.
PharmAust will also continue with the MPL dose escalation for Cohorts 3 and 4 during the interim trial
analysis to determine the optimum dose level for a Phase 2 trial.
Subject to approval at the next Safety Committee meeting to be held on 18 May 2023, the patients from
Cohort 1 will be escalated to Cohort 3. Following further approvals, those patients from Cohort 2 will
move to Cohort 4.
The Phase 1/2 MND trial is on track to be completed in Q3 of CY 2023.
MPL is a promising treatment for MND
According to the International Alliance of ALS/MND Associations, MND affects over 350,000 people
globally and kills more than 100,000 people yearly. The disease is invariably fatal, with the average life
expectancy of someone with MND being around 27 months. The MND/ALS addressable market is
US$3.6Bn per annum, with Riluzole already reaching ~US$1Bn annual sales.
The disease is progressive, meaning the symptoms get worse over time. MND has no cure and no
effective treatment to reverse its progression. PharmAust notes that five patients have surpassed the
6-month mark on MPL without any safety issues, and one patient appears “stable”.
PharmAust Limited ABN 35 094 006 023. Suite 116, 1 Kyle Way, Claremont, Western Australia
Tel: 08 9202 6814 Fax: 08 9467 6111
www.pharmaust.com
PharmAust demonstrated in its preclinical programs that MPL has the potential to activate molecular
pathways relevant to the treatment of MND. MPL could reduce the rate of degeneration and loss of
motor neurons in the brainstem's anterior horns and motor nuclei. There are also several surrogate
clinical endpoints to be determined during the trial. PharmAust has developed and manufactured a
bespoke MPL tablet for the trial.
With success in the clinic, PharmAust hopes that MPL could receive orphan drug designation by the
TGA and FDA for motor neurone disease. Such designations come with financial and supportive
benefits and PAA is evaluating this opportunity.
The Phase 1/2 study is being funded by a commitment of $881,085 by FightMND, Australia's largest
independent funder of MND research.
The Board authorises this announcement.
Enquiries:
Anusha Aubert
Investor Relations
investorenquiries@pharmaust.com
P +61 (8) 9202 6814
F +61 (8) 9467 6111
W www.pharmaust.com
About PharmAust Limited:
PharmAust Limited is listed on the Australian Securities Exchange (code: PAA) and the Frankfurt Stock Exchange
(code: ECQ). PAA is a clinical-stage company developing therapeutics for both humans and animals. The company
specialises in repurposing marketed drugs lowering the risks and costs of development. These efforts are supported
by PAA’s subsidiary, Epichem, a highly successful contract medicinal chemistry company which generated $3.4
million in sales of goods & services in FY 2022.
PAA’s lead drug candidate is monepantel (MPL), a novel, potent and safe inhibitor of the mTOR pathway – a
pathway having key influences in cancer growth and neurodegenerative diseases. MPL has been evaluated in
Phase 1 clinical trials in humans and Phase 2 clinical trials in dogs. MPL treatment was well-tolerated in humans,
demonstrating preliminary evidence of anticancer activity. MPL demonstrated objective anticancer activity in dogs.
PAA is uniquely positioned to commercialise MPL for treatment of human and veterinary cancers as well as
neurodegenerative disease as it advances a reformulated version of this drug through Phase 1 and 2 clinical trials.
PharmAust Limited ABN 35 094 006 023. Suite 116, 1 Kyle Way, Claremont, Western Australia
Tel: 08 9202 6814 Fax: 08 9467 6111
www.pharmaust.com
Share your feedback
Discover