Antisense Therapeutics Limited (ASX:ANP) recently confirmed positive results from the phase II clinical trial of ATL1102 in Duchenne Muscular Dystrophy (DMD) that affirmed the safety and positive drug effects on disease progression endpoints.
The company is now planning to advance ATL1102 into a potentially pivotal phase IIb clinical trial in DMD.
Antisense is also actively exploring clinical development opportunities in other indications where inflammation plays a key role in disease progression.
DMD market
The global DMD drugs market is expected to reach US$4.11 billion by 2023, according to a report by Grand View Research, Inc.
Sarepta Therapeutics Inc (NASDAQ:SRPT) has a drug called Exonyds 51 (eteplirsen) designed to increase dystrophin levels in about 13% of DMD patients who have a specific mutation of their dystrophin gene.
The drug is currently marketed in the US only and sells for >US$300,000 per patient per annum (some are paying close to $1 million per year for their treatment).
Sarepta’s market cap has grown from about US$60 million in 2012 when they started clinical development on Exonyds 51, to more than US$9 billion now.
Broader value creation potential
ATL1102 is an antisense inhibitor of CD49d, a subunit of VLA-4 (Very Late Antigen-4).
Antisense inhibition of CD49d expression has demonstrated activity in a number of animal models of inflammatory disease including asthma, arthritis and multiple sclerosis (MS).
The company has an Investigational New Drug application (IND) with the US FDA clearing ATL1102 for use in a phase IIb clinical trial in MS patients at the same 25mg per week dose that has shown activity in the DMD trial.
It is worth noting that MS drug sales in 2018 were US$23 billion and forecast to grow to US$39 billion by 2026.
In addition to MS, Antisense sees exciting potential for ATL1102’s use in other neuroinflammatory and muscular dystrophy disorders.
Shares have been up as much as 20% to an intra-day high of 7.2 cents.