Breakthrough Prize given to Antisense pioneer

Last week, biopharmaceutical drug discovery and development company, Antisense Therapeutics (ASX:ANP) spiked 217% in one day. ANP has two drug candidates, ATL1102 and ATL1103, which it has been studying in clinical trials for use in three diseases: Duchenne Muscular Dystrophy (DMD), Multiple Sclerosis (MS) (ATL1102) and acromegaly (ATL1103).

ANP is garnering plenty of attention at the moment, not least for its Phase II clinical trial with its flagship drug to treat DMD at the Royal Children’s Hospital in Melbourne.

On the Thursday (17 October), Forbes published an article about two winners of The Breakthrough Prizes, referred to as the ‘Oscars of Science’. Head of research at Ionis Pharmaceuticals, C. Frank Bennett, and his colleague Adrian R. Krainer will share a $3 million Breakthrough Prize for their role in developing Spinraza, a drug for spinal muscular atrophy which was invented by Ionis.

The link to ANP is the fact that the small cap has a worldwide exclusive license from Ionis for their IP on ATL1102 — and Bennett is actually the first named inventor on the original ATL1102 patents.

Ergo, one of the brains behind ANP’s ATL1102 drug candidate is the same man behind the ground-breaking Spinraza drug. Bennett also previously served as non-executive director on ANP’s board.

According to the Forbes report, Bennett joined Ionis three decades ago, when the company’s founder was leaving big pharma to start a smaller biotechnology firm to focus on a technology called antisense.

Antisense technology — which is the focus for Antisense Therapeutics/ ANP — works by using “a strand of nucleotides, the type of chemical in DNA” to stop the functioning of particular genes.

In the clinical trials that led to the approval of Ionis’ drug, Spinraza, 50% of the infants treated with Spinraza reached a ‘major motor milestone’. The drug was also shown to improve motor function in later-onset forms of spinal muscular atrophy.

ANP clinical trials underway

In May, ANP completed a $5 million capital raising, with the backing of two major institutional investors, Australian Ethical Investment and Platinum Asset Management for its all-important Phase II clinical trial of ATL1102 in DMD patients.

The six-month dosing trial of ATL1102 in nine non-ambulant DMD patients aged 10-18 years is now underway at the neuromuscular centre of the Royal Children’s Hospital in Melbourne.

ATL1102 inhibits the expression of protein ‘CD49d’ on certain immune cells (T cells). DMD is more severe and progresses more rapidly in patients who have more T cells with high levels of CD49d. The drug candidate is being developed to treat the inflammation that exacerbates tissue damage and causes fibrosis in DMD patients — a problem which currently can only be managed with corticosteroids.

On being issued a 'speeding ticket’ from the ASX last week, ANP referred to an article published mid last week, which told the small cap’s story to date: ‘ASX Junior’s Flagship Immunomodulatory Drug Could Turn the Tide in Duchenne Muscular Dystrophy Treatment’.

As of close of market yesterday, ANP’s share price is still sitting at around ~182% higher than it was before Wednesday’s rally.