Antisense Therapeutics is using its Multiple Sclerosis drug to beat a rare form of muscular dystrophy because it believes the tiny market could be a multi-million dollar winner.

Antisense (ASX:ANP) is recruiting ten teenage boys with Duchenne Muscular Dystrophy (DMD) at Melbourne’s  Royal Children’s Hospital to undergo a Phase 2 clinical trial of its ATL1102 treatment.

DMD is a debilitating disease that affects one in every 3500 to 5000 boys and causes muscles to deteriorate and breakdown.

Patients are wheelchair-bound by the time they reach eight to 11 years and many do not survive past their 20s.

The usual treatment is a life-long course of corticosteroids — which are supposed to be used for short periods of time but for sufferers of DMD are a permanent option.

Long term use comes with nasty side effects like diabetes, thinning of the skin and poor wound healing, and weight gain.

Antisense chief Mark Diamond is hoping the drug will not only help with the symptoms but also slow the disease’s progression.

The DMD news sent Antisense’s share price rocketing.

The TREAT-NMD global DMD registry says there are about 13,500 sufferers in 31 countries around the world, so it’s not a huge market Antisense (ASX:ANP) is targeting.

But Mr Diamond says it’s a market that can make them money.

He’s basing his hopes on the outcome achieved by US therapeutics company Sarepta, which developed and now sells an effective DMD drug for $300,000 per dose a year.

Improved corticosteroids cost $100,000 per dose a year.

He says they would even be willing to commercialise the drug themselves if it comes through Phase 2 trials well, rather than licensing it out.


The DMD drug is a half-dose of the same treatment used in an MS trial last year.

The drug showed progress in reducing the number of brain lesions in MS patients, but suffered a setback when the powerful US Food and Drug Administration told them to go back and do a Phase 2b trial on a half dose, over safety concerns.

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The company is trialing the lower dose on DMD patients with the rationale that it treats the same harmful cell expressions in MS as in DMD.

They will use the DMD results as extra information for the MS treatment.

Rare diseases are profitable

Mr Diamond hopes to have some money coming in the door this year, 17 years after Antisense listed.

The company has another drug for a very rare disease called Acromegaly, which is when the pituitary gland produces too much growth hormone in adulthood causing symptoms like excessive bone growth in the hands and jaw.

Surgery to remove the pituitary tumour which usually sorts the condition out.

Antisense is targeting patients for whom surgery hasn’t worked — about 20,000 people in the Northern Hemisphere.

Mr Diamond says they have a partnership in Europe with a drug access outfit called MyTomorrows could mean they get pre-Phase 3 trial access to patients there.

He thinks they can sell their Acromegaly treatment of one to two injections a week for between $30,000-40,000 a year.

The only other one on the market, which requires one to two injections a day, goes for $200,000 a year.

The company has to finalise manufacturing but Mr Diamond believes that can have a treatment on the market by the fourth quarter of 2018.

Antisense shares closed on Wednesday down 7.5 per cent to 3.7c.