Antisense Therapeutics (ASX:ANP) opened up 89 per cent today after final clinical trial results suggest its drug could be a blockbuster for a nasty disease affecting little boys.

The biotech’s immunomodulatory therapy, ATL1102 for Duchenne Muscular Dystrophy (DMD), met primary and secondary endpoints in a phase two clinical trial.

The key outcome in the nine-boy trial was that it was safe and worked. The secondary outcomes met included functional capacity around grip and pinch strength. MRI assessments of upper limb muscles showed the drug appeared to stabilise the fat fraction percentage in the muscles of the forearm.

DMD is a debilitating disease that affects one in every 3500 to 5000 boys and causes muscles to deteriorate and breakdown.

Patients are wheelchair-bound by the time they reach eight to 11 years and many do not survive past their 20s.

The usual treatment is a life-long course of corticosteroids — which are supposed to be used for
short periods of time but for sufferers of DMD are a permanent option. Long-term use comes with nasty side effects like diabetes, thinning of the skin and poor wound healing, and weight gain.

Antisense chief Mark Diamond said today the company had met with three European agencies to discuss a phase 2b study to take place in that region. Those agencies suggested that if this trial reproduced the successful results, it could be a pathway to conditional approval.

Diamond is also looking at an entree to the US market. Antisense has started the process for an orphan drug application, an option that provides incentives for companies to develop drugs for rare diseases.

And he said it was looking at adapting the treatment to other diseases. The company has already completed a successful phase 2a trial in Multiple Sclerosis and has Crohn’s disease, arthritis and other inflammatory diseases in its pipeline.


In other ASX biotech news:

TBG Diagnostics (ASX:TDL), still suspended as the ASX demands answers on why news that a COVID-19 diagnostic test had been approved for sale in Europe took four days to be revealed to investors in spite of a massive run up in the share price, has had another test kit approved for sale. This one is a lab kit that tests for active infection.