Antisense Therapeutics set to widen its scope of treatment options

Following recently reported positive clinical trial results relating to its research into Duchenne Muscular Dystrophy, biopharmaceutical drug development company Antisense Therapeutics (ASX: ANP) has declared that it’s now actively exploring clinical development opportunities in other indications where inflammation plays a key role in disease progression.

The company’s overarching mission is to develop and commercialise novel antisense therapeutics for a variety of drug candidates including Duchenne Muscular Dystrophy (DMD), multiple sclerosis (MS) and acromegaly –  a hormonal disorder that occurs due to excessive amounts of growth hormone in the body.

Late last year, Antisense reported data from a phase 2 clinical trial on its immunomodulatory therapy ATL1102 for DMD and confirmed the drug can have positive effects on the progression of the disease.

The trial aimed to assess the safety and tolerability of 25-milligram doses of ATL1102 administered to nine wheelchair-bound young male participants once a week via subcutaneous injection over six months.

At the end of the trial period, ATL1102 was found to have an “excellent safety profile” with Antisense stating that its drug was “generally safe and well-tolerated” with no serious adverse effects reported and no safety concerns expressed by the Australian Data Safety Monitoring Board.

According to Antisense, ATL1102 is an antisense inhibitor of CD49d, an expression on certain immune cells such as T lymphocytes. Researchers have discovered that DMD patients displaying a greater number of T cells with high levels of CD49d have more severe and rapid disease progression.

Antisense has set out to develop a means of mitigating this inherent relationship and says that inhibition of the CD49d expression has demonstrated activity in several animal models of inflammatory disease including asthma, arthritis and MS.

Moreover, ATL1102 was previously shown to be highly effective in reducing MS inflammatory brain lesions in a phase 2 clinical trial in relapsing remitting MS patients.

With positive trial results stacking up in its favour, Antisense has moved to apply for a an Investigational New Drug application (IND) with the US FDA, which would clear ATL1102 for use in a further phase 2 clinical trial in MS patients at the same 25mg per week dose that has shown activity in the DMD trial.

Treating multiple sclerosis

MS is a life-long chronic disease that progressively destroys the central nervous system. The condition affects around 400,000 people in North America, over 20,000 people in Australia and more than 2 million worldwide.

According to market statistics, MS drug sales totalled US$23 billion in 2018 and are forecast to grow to US$39 billion over the next six years.

In a statement to the market, Antisense reported that it is currently consulting with clinical experts on the appropriate next steps for clinical development in MS while also re-engaging with pharmaceutical companies active in the MS space to discuss partnering opportunities.

One potential avenue the company could take is to expand the scope of its development to include other neuroinflammatory and muscular dystrophy disorders given the expected antisense platform and CD49d target based advantages in these applications.

Antisense is also active in filing new patent applications to protect the use of ATL1102.

Just recently, the company said that its international patent application titled “Methods for treating multiple sclerosis using antisense oligonucleotides” advanced to the national phase in the US, Australia, New Zealand, Canada and Europe.

If granted, this patent family will protect the use of ATL1102 in MS until 2038, with a further five-year extension available in the US, Australia and Europe.