Duchenne’s Muscular Dystrophy (DMD) is a devastating generic muscular wasting disorder with a 100% fatality rate. At present, the only treatment for the muscle inflammation that leads to the loss of muscle function and strength, is to put patients on corticosteroids often for the rest of their lives. While corticosteroids do reduce inflammation and thus slow the progression of the disease, long-term use of these drugs can have major side effects such as weight gain, reduced bone density and stunted growth.

Antisense Therapeutics (ASX: ANP) is an Australian biotechnology company that is developing a new drug aimed at slowing the progression of DMD without the side effects of corticosteroids.

“We’re looking at a better way of treating the inflammation than using corticosteroids which were first approved nearly 60 years ago,” said CEO Mark Diamond when he spoke on The Insider.

 

Antisense’s ATL1102 drug is currently undergoing Phase II trials at the Royal Children’s Hospital in Melbourne. Administered to patients with advanced Duchennes over 6 months, the trial showed the drug to be safe, well-tolerated and to also reduce disease progression.

“We were able to improve the strength and muscle function in these patients over the losses you’d expect to see in that timeframe,” said Mark.

 

When the patients’ muscles were assessed under MRI, the drug was shown to have reduced the amount of fat in the muscles. Given increased fat is an indicator of muscle loss, this was a promising development for the company.

Mark stated “Our drug was able to show improvement in upper limb muscle function which is the key parameter for getting a drug approved to treat non-ambulatory patients with Duchennes.”

“We are potentially one study away from being able to commercialise our drug in the second biggest pharmaceutical market in the world,” said Mark, referring to Europe.

 

Antisense is also working to take the next steps in getting the drug developed and commercialised for the US market.

With the DMD market expected to hit $4 billion by 2023, Antisense’s completely novel drug mechanism means there is no direct competition in the non ambulant DMD treatment space and the company is thus well-positioned to lead in this market.

Last week, Antisense’s CEO Mark Diamond joined us for ‘The Insider. This article is a summary of the information Mark shared with us during the session. You can watch a recording of the session below, or you can click here to book into our next session where we will be joined by PlaySide Studios, Nusantara Resources (ASX: NUS) and Nickel Mines (ASX:NIC).

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