PAA aims to become a global leader in treatment of neurodegenerative diseases. Pic: Getty Images
Health & Biotech
Special Report: PharmAust raises $10m to finalise preparations for its pivotal adaptive Phase 2/3 STRIKE study.
Clinical stage biotech PharmAust (ASX:PAA) says it has received strong support from new and existing institutional investors for the placement at 19 cents/share, representing a 15.6% discount to the last closing price 22.5 cents/share on June 18 and a 19.8% discount to the five trading day VWAP of 23.7 cents/share.
PAA says directors and management are participating for ~$1m in the placement, subject to shareholder approval.
A share purchase plan (SPP) will be offered to eligible shareholders at the same offer price as the placement.
Placement proceeds will be used to finalise preparations for the pivotal adaptive Phase 2/3 STRIKE study of its lead drug monepantel (MPL) in patients with MND/AML.
Funds raised will also be used for MPL under Good Manufacturing Practice (GMP) , preclinical models, regulatory filings, working capital and offer costs.
PAA CEO and managing director Dr Michael Thurn says strong support from the placement serves as a vote of confidence in the exciting opportunity ahead for the biotech with MPL, along with the quality of its people and strategy.
“PharmAust can now finalise preparations towards commencing a pivotal registration study for monepantel for the treatment of MND/ALS,” he says.
With positive data about survival rates in patients taking MPL, new executive appointments, and a presentation at a major medical conference PAA continues to build momentum as its share price soars more than 100% year to date.
Updated data by Berry Consultants shows patients who took MPL had a 91% lower risk of death from MND/ALS compared to ‘untreated matched-controls’ from the Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) database for patients with MND/ALS.
The PRO-ACT database is a database of historical control data that scientists and clinicians often use to as a source control data given the ethical concerns in recruiting patients for control/placebo arms in life threatening diseases.
Based in Austin, Texas, Berry Consultants specialises in clinical trial design, analysis, execution, and software solutions for the pharmaceutical and medical device industries.
The organisation is renowned for their expertise in Bayesian statistics and aim to set the standard for adaptive clinical trials across all medical fields.
Berry Consultants also examined how MPL affects the decline rate of ALS Functional Rating Score-Revised (ALSFRS-R) scores over time.
The ALSFRS-R measures functional status in MND/ALS patients, covering mobility, self-care, speech, and swallowing.
Berry Consultants used data from PAA’s recently completed Phase 1 MEND study and baseline ALSFRS-R scores of patients who joined the open label extension (OLE) study.
They compared changes in ALSFRS-R scores between MPL-treated patients and those from the PRO-ACT database, similar to their survival analysis method.
The analysis showed that MPL slowed the decline in ALSFRS-R scores by 34%, with a change in slope of 0.40 ALSFRS-R points per month, leading to an estimated decline rate of -0.81 points per month.
“The updated survival analysis conducted by Berry Consultants is extremely encouraging, as is the updated efficacy analysis that indicates MPL continued to slow the rate of disease progression in patients with MND/ALS,” Thurn says.
“These results provide an exciting backdrop ahead of the anticipated commencement of the pivotal adaptive Phase 2/3 STRIKE study in H2 2024.”
PAA’s OLE has now completed enrolment with the study investigating the long-term safety, tolerability, and efficacy of MPL in patients with MND/ALS who previously completed the Phase 1 MEND study.
Eligible patients are receiving a daily dose of 10 mg/kg body weight of MPL for an additional 12 months.
The OLE study is being run from two Australian sites including Calvary Health Care Bethlehem, led by Associate Professor Susan Mathers and Macquarie University, led by Professor Dominic Rowe.
Dr Philip White and Dr Melanie Quintana of Berry Consultants presented data from the Phase 1 MEND study at the annual meeting of the European Network to Cure ALS (ENCALS), held in Stockholm from June 17-19.
PAA says the ENCALS meeting is Europe’s largest and most significant MND/ALS conference attended by physicians, key opinion leaders, pharmaceutical and biotechnology representatives, academics and patient advocacy groups.
The poster presentation highlighted the statistical comparisons made between patients treated with MPL in PAA’s Phase 1 MEND Study to untreated matched-controls from the PRO-ACT database for patients with MND/ALS.
PAA non-executive chairman Sergio Duchini and chief operating officer, John Clark attended ENCALS for a variety of business meetings with industry, global clinical trial service providers and key opinion leaders in MND/ALS.
Thurn says 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,” he says.
“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.”
In May, PAA announced the reappointment of Thurn as its managing director and CEO as it works to become a global leader in neurodegenerative diseases.
Thurn oversaw completion of the Phase 1 MEND study and has been praised for his ability to create and maintain strong shareholder relations, which led to a successful $3.5m capital raising at a 4.8% premium to the 15-day volume weighted average price late last year.
To position PAA for the next phase of clinical development and commercialisation he established a world-class scientific advisory board, made several key management hires and formed a partnership with Berry Consultants.
To advance its treatments for neurodegenerative diseases, PAA has also appointed experienced neuroscientist Dr Nicky Wallis as chief scientific officer.
PAA says Wallis excels in clinical development from pre-clinical stages to Phase 3 trials and has successfully managed international studies in the US, Europe, Australia, and New Zealand, focusing on regulatory compliance, cross-functional collaboration, and vendor management.
Before joining PAA, Wallis consulted for biotechnology companies in Australia, specialising in central nervous system diseases.
Her expertise in MND/ALS at the molecular level and extensive experience in neurodegenerative disease development would be instrumental for exploring MPL’s potential other applications.
More recently, PAA announced experienced US pharmaceutical executive Dr Katie MacFarlane had join the Board of Directors.
MacFarlane has more than 30 years of experience in the development and commercialisation of pharmaceutical products and devices.
Her previous senior executive positions include chief commercial officer at Agile Therapeutics, vice president of marketing, sales and new product planning at Warner Chilcott, and senior director of marketing at Parke-Davis (now Pfizer).
MacFarlane is currently the head of commercial for Arkayli Biopharma, a startup developing a treatment for a rare paediatric disease.
She is a board member of Mayne Pharmaceuticals, an affiliate faculty member of the Purdue University School of Pharmacy and a founding member and advisor to IPhO.
She has also previously served on the board of RespireRx and a non-profit, INMED Partnerships for Children.
“Katie’s has built an impressive and extensive network of pharmaceutical contacts over the years. PAA non-executive chairman Sergio Duchini says.
“PharmAust looks forward leveraging these networks and working with Katie to lift the visibility of the Company in the US.”
June 21 is MND Global Awareness Day and PAA invites everyone to join the International Alliance of ALS/MND Associations’ webinar that will explore the impact of the Ice Bucket Challenge on raising funds for research.
The PAA share price is up 104% YTD and 18% in the past month.
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.
Health & Biotech
Health & Biotech
Health & Biotech
