Antisense Therapeutics (ASX: ANP) and (FRA: AWY) are up over 60% in 2 months.
2 major reasons for this:
Sarpeta Therapeutics announced their experimental gene-therapy drug had failed to significantly improve motor function in patients with Duchenne Muscular Dystrophy (DMD). This caused the shares to fall by over 47%. The trial of Sarepta's was the first placebo-controlled study of an experimental DMD drug.
The Sarepta results represent a significant change for Duchenne Muscular Dystrophy. Previously. the prevailing thought was that DMD is due to lack of dystrophin. However, the older boys in the Sarepta trial saw an increase in Dystrophin but no change over placebo! The younger boys improved because the inflammation had not yet started. There is a growing realisation throughout the whole DMD community (big pharma, investors and advocacy groups) that inflammation is just as important but maybe more important - and this represents a seismic shift now looking towarrds Antisense Therapeutics Limited.
This has now increased global interest in Antisense Therapeutics Limited which will soon commence Phase IIb trials in Europe for DMD after a successful Phase II trial in Australia. The open label six-month dosing trial of ATL1102 in nine non-ambulant patients with DMD aged between 10 and 18 years was conducted at the neuromuscular centre of the Royal Children’s Hospital in Melbourne which operates the largest clinic in the southern hemisphere treating children with DMD.
ATL1102 is being developed as a novel treatment for the inflammation that exacerbates muscle fibre damage in DMD patients, currently treated with corticosteroids. Corticosteroids have a range of serious side effects when used for a prolonged period as required in DMD. Currently there are a limited number of anti-inflammatory drugs in clinical development for DMD. The company announced in 2020 that ATL1102 had met the required safety standards and achieved "strong initial efficacy" as part of its Phase II clinical trial. the results were also supported by a team of scientists, researchers and medical professionals that were aware of the trial's endpoints.
Overall the study showed that administering ATL1102 resulted in "consistent improvements or stabilisation across the different measures of motor function and strength".
The European Phase IIb study is expected to be an approvable study, meaning commercialisation and revenue will likely result from a successful clinical trial.
The global market for DMD therapies is estimated at around EUR 3.5 billion.
The Australian broker, Morgans, released a report valuing Antisense at AUD $0.37 or €0.23 a share.
A copy of the report can be downloaded below
A research report in January 21 sets a 12-month price target of AUD $0.43 or €0.26 a shares
A February 2021 video interview with the CEO can be viewed below
On 25 february 2021, Antisense announced that the Paediatric Investigation Plan (PIP) for the development of ATL1102 for Duchenne Muscular Dystrophy (DMD) has been submitted to the European Medicines Agency. This plan addresses the entire paediatric development plan for ATL1102 in DMD.
A copy of the ASX Announcement can be downloaded below