https://thewest.com.au/business/biotech/how-pharmaust-is-offering-hope-to-patients-with-mnd-and-als-using-veterinary-drug-monepantel-c-13935712

How PharmAust is offering hope to patients with MND and ALS using veterinary drug monepantel

Cheyanne EncisoThe West Australian

Mon, 1 April 2024 6:00PM

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Cheyanne Enciso

The head of PharmAust says the Perth biotech could offer hope to the hundreds of thousands of patients with motor neurone disease and ALS using a re-purposed veterinary drug approved for sheep drench.

The clinical-stage company completed a phase 1 study in December, with the recent findings revealing the drug monepantel — typically used to remove worm infestations in sheep — reduced the progression of the neuro degenerative diseases by 58 per cent.

ALS is the most common form of MND and is characterised by progressive degeneration of nerve cells in the spinal cord and brain. It affects voluntary control of arms and legs, and leads to trouble breathing.

There is no cure for ALS.

PharmAust said its drug had shown it could provide patients with an additional 13.5 to 56.5 months — or nearly five years — of life, compared to other drugs on the market like the US FDA-approved Relyvrio, which could only prolong life by an extra nine months.

PharmAust chief executive Michael Thurn said MND and ALS sufferers only had an average of just over two years to live after diagnosis.

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With Relyvrio’s manufacturer Amylyx announcing in early March the drug did not show significant benefit in a large clinical trial and may be withdrawn from the market, Mr Thurn said there was “really no very good treatments available for MND/ALS”.

“It’s a hideous disease, it’s characterised by your motor functions shutting down, whether that’s your ability to walk, to swallow, to speak, to breath,” he said.

“There’s so few drugs in development for MND/ALS, it’s been a hard one to crack.

“The first drug that was approved for use in MND was back in 1995 and in 1995 that drug only extended life by two to three months.”

Based on PharmAust’s analysis, Mr Thurn said at least one-third of the 12 participants in its study — conducted in Melbourne and Sydney — should have expected to have died.

“We can say, quite confidently, that those 12 patients have benefited by being on monepantel,” he said.

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PharmAust has now filed the phase 1 study results to the FDA for a so-called orphan drug designation, which offers incentives such as tax credits for certain clinical trials. A decision from the FDA is expected in coming months.

The company is now also gearing up for a phase 2/3 clinical study, set to start towards the middle of this year and will have sites in Australia, Europe and the US.

“This whole plan of making it a global study then enables us to use this one study as the centrepiece to then get approval from the FDA in the US, get approval from the European Medicines Agency and also the Therapeutic Goods Administration,” Mr Thurn said.

“Based on the numbers of patients out there with MND, this drug could be a blockbuster. It could be a drug that makes more than $1 billion in sales.”

PharmAust shares have surged almost 400 per cent in the past six months, with Mr Thurn saying it wanted to reward shareholders.

“We’ve been out there creating awareness about the company, our program, and we’ll continue to do that,” he said.

“This is just the beginning of the journey of the company, we’re hopeful that over the next 12 months we’ll certainly improve the share price position.

https://investorhub.pharmaust.com/announcements/6270398

Announcement summary

The article in The West Australian quoted PharmAust CEO Dr Michael Thurn as saying: “Based on the numbers of patients out there with MND, this drug could be a blockbuster. It could be a drug that makes more than $1 billion in sales.”

PharmAust clarifies that this is not a forecast nor revenue projection. The Company is continuing development of the drug (monepantel) including the forthcoming global pivotal registration Phase 2/3 clinical study scheduled to start mid-2024, and any potential sales of the drug will be subject to these study outcomes, regulatory approval and various pharmaceutical reimbursement schemes.

The quote was provided based on the potential for sales of monepantel for the treatment of motor neurone disease (MND) / amyotrophic lateral sclerosis (ALS), should it be approved in major regulatory jurisdictions such as the United States (US) and Europe. Relevant data on MND/ALS including the prevalence of disease, size of market and the list price of recently approved drugs for orphan diseases and MND/ALS are summarised as follows:

• In the US and western Europe it is estimated there is more than 37,000 and 56,000 patients with MND/ALS, respectively, and more than 268,000 globally (1)
• The MND/ALS treatment market was more than USD $9 billion in 2022 and is estimated to each USD $23 billion by the end of 2035 (2)
• In 2021 the median treatment cost was USD $218,872 for orphan drugs (range USD $237 to USD $1,272,021) (3)
• The last three drugs (QalsodyTM, Radicava®, and RelyvrioTM) that received US Food and Drug Administration (FDA) approval for the treatment of MND/ALS each have an annual cost of between USD $150,000 to USD $200,000 (4)
• RelyvrioTM, referenced in the recent media coverage, generated USD $381 million in sales during 2023, its first year from launch (5)

To the extent that the quote could be considered a financial forecast, PharmAust retracts the statement as it does not have a reasonable basis for the targeted sales figure at this stage. Investors should not rely on the revenue target mentioned in the article as a basis for investment decision on the Company.

1-Park J, Kim J-E and Song T-J (2022) The Global Burden of Motor Neuron Disease: An Analysis of the 2019 Global Burden of Disease Study. Front. Neurol. 13:864339. doi: 10.3389/fneur.2022.864339

2-https://www.researchnester.com/reports/motor-neuron-disease-treatment-market/37813-Althobaiti H, Seoane-Vazquez E, Brown LM, Fleming ML, Rodriguez-Monguio R. Disentangling the Cost of Orphan Drugs Marketed in the United States. Healthcare (Basel). 2023 Feb 13;11(4):558. doi: 10.3390/healthcare11040558. PMID: 36833091; PMCID: PMC9957503.4-https://endpts.com/biogen-prices-new-als-drug-at-more-than-14k-per-dose/

5-https://edition.cnn.com/2024/03/08/health/als-drug-relyvrio-fails-trial/index.html