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Melbourne-based biotech submits Duchenne Muscular Dystrophy drug for US and EU Orphan Drug designation

Sophia Katsinas
1 September 2020

Antisense Therapeutics (ANP) is a biotech small cap currently focused on developing an antisense treatment for a devastating genetic muscle wasting disorder that affects boys from birth with serious complications developing as young as 5-years-of age. This fatal muscular disorder is known as Duchenne Muscular Dystrophy (DMD).

The bigger picture goal for ANP is to create a series of antisense drugs for diseases whose needs are currently unmet in the medical research and technology space.

Read more: Meet the ASX-listed biotech tapping into unclaimed medical territory

This month Antisense submitted an application to the FDA for Orphan Drug designation of its drug ATL1102.

Orphan Drug designation is granted by the FDA to drugs that have proven to be safe and effective and aim to treat rare diseases that affect fewer than 200,000 people in the US.

The FDA’s director of the Office of Neuroscience in their Centre for Drug Evaluation and Research, Billy Dunn MD said in a news release on August 12: “The FDA is committed to fostering drug development for serious neurological disorders like Duchenne muscular dystrophy (DMD).”

ANP is partnered with the world’s leading antisense drug development and commercialisation company, Ionis Pharmaceuticals, which is listed on the Nasdaq and has a market capitalisation of US$8 billion.

ANP’s footprint in the US has increased with the appointment of US based Medical Director Dr Gil Price who is overseeing the company’s active engagement with key DMD advocacy groups and thought leading physicians.

ANP has also recently applied for Orphan Drug designation for ATL1102 in DMD to the European Medicines Agency Committee for Orphan Medicinal Products.  Orphan status with EMA brings development and marketing incentives, such as reduced fees, scientific advice and market exclusivity for 10 years upon regulatory approval.

Mark Diamond, ANP’s Managing Director and CEO said: “This is an important regulatory and commercial milestone in the further development of ATL1102 for DMD and represents another key step forward towards bringing this potentially transformative therapy to patients with a significant unmet need”.

Further to feedback already received from three EU national authorities, in July 2020, the European Medicines Agency (EMA) assessed Antisense’s clinical trial plans for Phase IIb/III clinical trial in DMD patients and provided additional constructive feedback on the trial design.

In parallel and as a priority, Antisense is actively progressing interactions with US based key opinion leaders, patient advocacy groups and expert regulatory consultants to define clinical path for ATL1102 in DMD in the US. Antisense Therapeutics (ANP) has a market cap of over A$40 million, with $4 million in cash, with leading healthcare institutional investors Australian Ethical investment and Platinum Asset Management holding 6.65% and 5.15% of the company respectively.

This article is sponsored content. The supplier of this content has a commercial arrangement with Switzer Financial Group.

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