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Antisense Therapeutics prepares for Phase IIb clinical trial of ATL1102 in DMD

Last updated: 05:15 13 Jan 2020 GMT, First published: 22:28 12 Jan 2020 GMT

Antisense Therapeutics Ltd - Antisense Therapeutics prepares for Phase IIb clinical trial of ATL1102 in DMD
The CEO Sessions will be in Sydney on Monday, February 3, and Melbourne on Tuesday, February 4

Antisense Therapeutics Limited (ASX:ANP) has raised $5.5 million from exercising options with funds to be used to advance preparations for Phase IIb clinical trial of ATL1102 in Duchenne Muscular Dystrophy (DMD).

The funds were received from the conversion and underwriting of listed options that expired on December 19, 2019.

Managing director and CEO Mark Diamond said the company thanked all option holders who converted their options and welcomed the support of major shareholders and new institutional investors and sophisticated clients of Morgans Corporate Ltd who participated in the underwriting.

MD and CEO to present

Diamond will outline the biopharmaceutical company’s story and future plans at Proactive’s first CEO Sessions for 2020 in Sydney on Monday, February 3, and Melbourne on Tuesday, February 4, along with four other companies.

Antisense, which has a market cap of around $41.5 million, last traded at 8.5 cents and in the past 12 months has traded between 2.6 cents and 14.5 cents.

Developing antisense pharmaceuticals

The company is developing and commercialising antisense pharmaceuticals for large unmet markets.

These products, including ATL1102 and ATL1103, are in-licensed from Ionis Pharmaceuticals Inc (NASDAQ:IONS), a leader in antisense drug development and commercialisation.

ATL1102 is an antisense inhibitor of CD49d, a subunit of VLA-4 (Very Late Antigen-4).

Antisense inhibition of VLA-4 expression has demonstrated activity in a number of animal models of inflammatory disease including asthma and MS with the MS animal data having been published in a peer-reviewed scientific journal.

ATL1102 was shown to be highly effective in reducing MS lesions in a Phase IIa clinical trial in RR-MS patients.

Clinical trial of ATL1102

The company is undertaking a clinical trial of ATL1102 in patients with DMD, an X-linked disease that affects 1 in 3600 to 6000 live male births.

This open label six-month dosing trial of ATL1102 in nine non-ambulant patients with DMD aged between 10 and 18 is being conducted at the neuromuscular centre of the Royal Children's Hospital (RCH).

Primary endpoints of the trial relate to the safety and tolerability of ATL1102 while the efficacy of ATL1102 will also be assessed in terms of its effects on disease processes and progression.

“Well placed”

Diamond said: “With additional funding received through options conversion and following successful completion of dosing in all patients in our Phase II clinical trial in DMD, we are well placed to advance preparations for ATL1102 to move into a potentially pivotal Phase IIb clinical trial.”

He said the trial work to date had affirmed the drug’s excellent safety profile and positive drug effects on disease progression endpoints at the low dose tested.

Dystrophin deficiency

DMD occurs as a result of mutations in the dystrophin gene which causes a substantial reduction in or absence of the dystrophin protein.

Children with DMD have dystrophin-deficient muscles and are susceptible to contraction-induced injury to muscle that triggers the immune system which exacerbates muscle damage.

Ongoing deterioration in muscle strength affects lower limbs leading to impaired mobility, and also affects upper limbs, leading to further loss of function and self-care ability.

The need for wheelchair use can occur in early teenage years for patients on corticosteroids with a mean age of 13, with respiratory, cardiac, cognitive dysfunction also emerging.

Patients with a greater number of immune T cells expressing high levels of CD49d have more severe and progressive disease and are wheelchair bound by the age of 10 despite being on corticosteroid treatment.

With no intervention, the mean age of life is approximately 19.

Management of the inflammation associated with DMD is currently addressed via the use of corticosteroids, however, they are acknowledged as providing insufficient efficacy and are associated with significant side effects.

Acknowledged high need

As a consequence, there is an acknowledged high need for new therapeutic approaches for the treatment of inflammation associated with DMD.

Diamond said: “The next stage of development will be in translating what we have learned into optimising clinical benefit for the non-ambulatory boys who comprise approximately 50% of the total DMD population and who have no effective treatment options.

“The company is moving forward with all deliberate speed to advance ATL1102 through the clinic, with specific view to a blinded controlled study in the EU which, based upon recent and ongoing guidance, may lead directly to early approval.”

Five presenting companies

The Sydney and Melbourne events will feature five presenting companies with others including Great Boulder Resources Ltd (ASX:GBR), Recce Pharmaceuticals Ltd (ASX:RCE) and Cynata Therapeutics Ltd (ASX:CYP). Also presenting in Sydney will be Reward Minerals Ltd (ASX:RWD) while the other presenting company in Melbourne will be Alice Queen Ltd (ASX:AQX). 

Register now for the CEO Sessions:

Sydney event, Monday, February 3

Melbourne event, Tuesday, February 4

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