PharmAust's Phase 1 Study Data Presented at Major Conference
Released
Released
Announcement PDF
Berry Consultants Presents PharmAust’s Phase 1
Study Data at Major International ALS Conference
Highlights:
Dr Philip White and Dr Melanie Quintana of Berry Consultants presented data from
PharmAust’s successful Phase 1 MEND study at the annual meeting of the European
Network to Cure ALS (ENCALS)
The data highlighted the statistically significant survival benefit for patients treated with
monepantel compared to untreated matched-controls from the PRO-ACT database
Representatives from PharmAust will be in attendance to hold a variety of business
meetings
Click here to view the poster being presented at the ENCALS meeting
Click here to watch a video of Dr Philip White presenting the poster
18 June 2024 – Melbourne, Australia: PharmAust Limited (ASX: PAA & PAAOA) (“PharmAust” or “the
Company”), a clinical-stage biotechnology company, is pleased to announce that Berry Consultants
presented data from PharmAust’s successful Phase 1 MEND study at the annual meeting of the
European Network to Cure ALS (ENCALS), being held in Stockholm, Sweden from 17-19 June.
The meeting is the largest and most significant motor neurone disease/amyotrophic lateral sclerosis
(MND/ALS) conference in Europe, attended by physicians, key opinion leaders, pharmaceutical and
biotechnology representatives, academics and patient advocacy groups.
The poster presentation by Dr Philip White and Melanie Quintana highlighted the previously reported
(as announced on 14 and 27 February and 5 June) statistical comparisons made between patients
treated with monepantel (MPL) in PharmAust’s recently completed Phase 1 MEND Study to untreated
matched-controls from the Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) database for
patients with MND/ALS.
The highlights included:
An average 0.48-point improvement in Revised Amyotrophic Lateral Sclerosis Functional Rating
Scale-Revised (ALSFRS-R) score per month for the MPL treatment patients, relative to
untreated matched-controls from the PRO-ACT database
Compared to matched controls from the PRO-ACT database, treatment with MPL results in a
significantly (2=9.39, p=0.0022) longer survival of patients with MND/ALS
Treatment with MPL significantly reduces the risk of death by 91% (HR=0.087, p=0.0154)
compared to PRO-ACT matched controls
PharmAust’s Non-Executive Chairman, Sergio Duchini and the Company’s Chief Operating Officer,
John Clark will be in attendance at ENCALS for a variety of business meetings with industry, global
clinical trial service providers and key opinion leaders in MND/ALS.
PharmAust Managing Director Dr Michael Thurn commented:
“To have Dr Philip White and Dr Melanie Quintana present the clinical data from our successful Phase
1 MEND Study at a major international conference such as ENCALS is a major achievement for the
Company. We are very grateful to Berry Consultants for independently presenting the data and creating
greater awareness about our promising efficacy data to the broader MND/ALS community. We look
forward to working with Berry Consultants as we move closer to finalising the design of the pivotal
adaptive Phase 2/3 STRIKE study, planned to commence in H2 2024.”
PharmAust Limited ABN 35 094 006 023. Level 4, 96-100 Albert Road, South Melbourne VIC 3205
Tel: +61 3 9692 7222
www.pharmaust.com
About the PRO-ACT database
The PRO-ACT database is the largest publicly available repository of merged ALS clinical study data.
Clinical study data were pooled from 16 completed Phase 2/3 ALS/MND clinical studies and one
observational study. Over 8 million de-identified longitudinally collected data points from more than
10,000 persons with ALS were standardised across studies and merged to create the PRO-ACT
database. This database includes demographics, family histories, and longitudinal clinical and
laboratory data.
About Berry Consultants
Berry Consultants is a statistical consulting company based in Austin, Texas USA, specialising in
innovative clinical trial design, analysis, execution, and software solutions for the pharmaceutical and
medical device industry. Berry Consultants employs world-renowned experts in Bayesian statistics and
strives to set the standard for adaptive clinical trial design and analysis across all medical disciplines.
www.berryconsultants.com
This announcement is authorised for release by the Board of Directors of PharmAust Limited.
For further information, please contact:
Dr Michael Thurn
Managing Director
investorenquiries@pharmaust.com
P +61 (3) 9692 7222
W www.pharmaust.com
Media:
Catherine Strong
Morrow Sodali
c.strong@morrowsodali.com
0406 759 268
About PharmAust Limited:
PharmAust Limited is listed on the Australian Securities Exchange (ASX Code: PAA). PAA is a clinical-stage
biotechnology company developing therapeutics for neurodegenerative diseases. The company is focused
on repurposing monepantel (MPL) for amyotrophic lateral sclerosis (ALS). ALS is the most common form of
motor neurone disease (MND) and affects both upper and lower motor neurons.
MPL is a potent and safe inhibitor of the mTOR pathway. This pathway plays a central role in the growth and
proliferation of cancer cells and degenerating neurons. The mTOR pathway regulates the cellular “cleaning
process”, where toxic proteins are broken down into macromolecules to be reused. This autophagic process
is disrupted in most neurodegenerative diseases, including ALS.
The company recently announced positive top-line results for its Phase 1 MEND study in participants with
ALS. PAA is in the planning stages for a registration adaptive Phase 2/3 STRIKE clinical study and anticipates
commencing enrolment in H2 CY 2024. This single pivotal study could potentially lead to accelerated approval
with the US Food and Drug Administration for monepantel for the treatment of ALS in 2026.
In 2024, the Neurodegenerative Disease Market size is estimated to be worth USD 55.12 billion, with a
forecast growth (CAGR) of 7.14% the market size is expected to reach USD 77.82 billion by 2029.1
1
https://www.mordorintelligence.com/industry-reports/neurodegenerative-disease-market
PharmAust Limited ABN 35 094 006 023. Level 4, 96-100 Albert Road, South Melbourne VIC 3205
Tel: +61 3 9692 7222
www.pharmaust.com
PharmAust Investor Hub:
We encourage you to utilise our Investor Hub for any enquiries regarding
this announcement or other aspects concerning PharmAust. This platform
offers an opportunity to submit questions, share comments, and view video
summaries of key announcements.
Access the investor hub by scanning the QR code or visiting:
https://investorhub.pharmaust.com/
PharmAust Limited ABN 35 094 006 023. Level 4, 96-100 Albert Road, South Melbourne VIC 3205
Tel: +61 3 9692 7222
www.pharmaust.com
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