Guess which ASX healthcare stock just rocketed 20% on a 'significant milestone'

Some very promising study data is getting investors excited on Wednesday.

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PharmAust Limited (ASX: PAA) shares are catching the eye with a strong gain on Wednesday.

At the time of writing, the ASX healthcare stock is up almost 20% to 24.5 cents.

Why is this ASX healthcare stock rocketing?

Investors have been bidding the clinical-stage biotechnology company's shares higher following the release of an announcement this morning.

According to the release, the company's Open-Label Extension (OLE) study has delivered some very promising results.

The OLE study is investigating the long-term safety, tolerability, and efficacy of monepantel (MPL) in patients with Motor Neurone Disease (MND)/Amyotrophic Lateral Sclerosis (ALS).

The study involves two sites in Australia, Calvary Health Care Bethlehem, led by Associate Professor Susan Mathers, and Macquarie University, led by Professor Dominic Rowe.

MPL is a potent and safe inhibitor of the mTOR pathway. The company notes that this pathway plays a central role in the growth and proliferation of cancer cells and degenerating neurons. It 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.

What's the latest?

The ASX healthcare stock revealed that its updated data analysis conducted by Berry Consultants shows a statistically significant survival benefit for MPL compared to untreated matched-controls from the Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) database for patients with MND/ALS.

It notes that treatment with MPL significantly reduced the risk of death by 91% when compared to PRO-ACT matched controls.

In addition, management highlights that the updated analysis of the rate of decline in ALSFRS-R is to include the compassionate use program and continued to show that MPL reduces the rate of disease progression.

Enrolment on to the OLE Study is now complete with 10 of the 12 patients from the Phase 1 MEND Study rolling over.

What's next?

The ASX healthcare stock's managing director, Dr Michael Thurn, believes this is a significant milestone for the company.

He also notes that it sets the company up well ahead of the expected commencement of the Phase 2/3 STRIKE study later this year. Dr Thurn commented:

I'm very pleased that we have completed enrolment in the OLE study as this is a significant milestone for PharmAust. 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. These results provide an exciting backdrop ahead of the anticipated commencement of the pivotal adaptive Phase 2/3 STRIKE study in H2 2024.

Motley Fool contributor James Mickleboro has no position in any of the stocks mentioned. The Motley Fool Australia's parent company Motley Fool Holdings Inc. has no position in any of the stocks mentioned. The Motley Fool Australia has no position in any of the stocks mentioned. The Motley Fool has a disclosure policy. This article contains general investment advice only (under AFSL 400691). Authorised by Scott Phillips.

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