- Antisense Therapeutics (ANP) has received data from all nine participants having completed the trial for ATL1102 in DMD
- The phase II trial affirmed the drugs excellent safety profile and positive drug effects on disease progression endpoints
- ATL1102 is being developed as an original treatment for the inflammation that aggravates muscle fibre damage in DMD patients
- DMD affects one in every 3500 to 6000 male births and is described as the ongoing deterioration of muscles
- In a busy morning Antisense also announced it has entered into an agreement with Morgans Corporate Limited to underwrite the balance of options not yet exercised to raise $4.1 million
- Upon re-entering the ASX, Antisense’s share price is up 15.9 per cent with shares trading for 9.5 cents apiece
Antisense Therapeutics (ANP) has received data from all nine participants having completed the trial for ATL1102 in DMD.
The phase II, 24-week clinical trial affirmed the drug’s excellent safety profile and positive drug effects on disease progression endpoints at the low dose that was tested.
It also outlines Antisense’s plans to advance ATL1102 into a potentially pivotal phase IIb clinical trial.
The primary objective of the trial was to evaluate the safety and tolerability of 25 milligrams of ATL1102 administered once weekly, via injection, for 24 weeks in non-ambulatory DMD participants.
The trial showed ATL1102 to be generally safe and well-tolerated in non-ambulant (not being able to walk) boys with DMD.
No serious adverse events have been reported and there have been no safety concerns expressed by the Data Safety Monitoring Board.
The most commonly reported adverse events have related to the administration of the drug, mainly injection site erythema (skin redness) and skin discolouration.
The trial also assessed drug activity and efficacy including measuring the effects on immune cell numbers in the blood and measuring functional capacity in participants.
This was evaluated via performance of upper limb test and upper limb strength via MyoGrip and MyoPinch assessments.
“Seeing the efficacy signals of this study, conducted with a low dose in a small number of boys over a relatively short time period, is both gratifying and immensely encouraging,” Antisense Non-Executive Director William Goolsbee commented.
“DMD is a devestating disease where only a small handful of drugs have shown indications of efficacy so early in developments. In the context of DMD, we now look to have a drug,” he added.
DMD (Duchenne Muscular Dystrophy) is an X-chromosome disease that affects one in every 3500 to 6000 male births.
It occurs as a result of malnutrition in the dystrophin gene (a protein found in skeletal and heart muscles). This then causes a substantial reduction or absence of the protein.
Ongoing deterioration in the muscles can cause weakness and often affects the lower and upper limbs resulting in impaired ability, loss of function, and loss of self-caring ability.
There are currently no cures but medication such as corticosteroids and ongoing therapy can help control the symptoms and improve the patient’s quality of life.
“Disease stabilisation or indeed an improvement in functional scores in non-ambundant DMD boys is almost unheard of and a very encouraging result,” Professor Thomas Voit MD, Director of NIHR GOSH Biomedical Research Centre UK said.
“This is even more meaningful as these results have been obtained using different independent measures and over a relatively short time of 24 weeks,” he added.
Antisense’s ATL1102 is an inhibitor of CD49d expression on certain immune cells such as T cells (cells that hunt down and destroy infected and cancerous cells).
It has been reported that patients with DMD, who have a greater number of T cells with high levels of CD49d, have a more severe and rapid disease progression.
ATL1102 is the only drug in clinical development for DMD targeting CD49d and one of a very limited number of treatments being tested in wheelchair-bound DMD boys who are at a more advanced stage of the disease.
These positive results continue to be highly supportive of Antisense’s plans for a Phase IIb clinical trial of ATL1102 in DMD.
Scientific Advice meetings have been held over the recent months with three European regulatory authorities with a focus on the phase IIb trial plans, dose-escalation plans, the applicability of the study endpoints, and the study duration.
There was general acceptance by the agencies at the meetings regarding the trial endpoints, safety monitoring plan, dosing duration for 12 months, and the use of higher doses.
Encouraging clarification was provided by the agencies that this could be a path to an early regulatory approval on positive phase IIb results.
The next step will be to follow up the development plan with the European Medicines Agency and engage with the U.S. Food and Drug Administration on development plans for the U.S.
In a busy morning, Antisense also announced it has entered into an agreement with Morgans Corporate Limited to underwrite the balance of options not yet exercised or committed to raise $4.1 million.
This provides holders with an option to purchase shares in the company at the price of $0.08 per share.
Taking into account the underwritten amount and commitments received, Antisense’s understanding is that it will receive full options exercise of $5.5 million.
Upon re-entering the ASX, Antisense’s share price is up 15.9 per cent with shares trading for 9.5 cents apiece at 11:54 am AEDT.