ATL1102 for Duchenne Muscular Dystrophy (DMD)

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

DMD is caused by a mutation in the muscle dystrophin gene leading to severe progressive muscle loss and premature death. One of the most common fatal genetic disorders, DMD affects approximately one in every 3,500 to 5,000 males worldwide. A key challenge in the management of DMD patients is to reduce the inflammation that exacerbates the muscle fibre damage. Corticosteroids are the only approved treatments for muscle inflammation, however they do not sufficiently suppress muscle inflammation, are not well tolerated and have serious side effects including adversely affecting growth rate. As a result, there is an acknowledged high unmet need for new therapeutic approaches for the treatment of inflammation associated with DMD.

Recently published clinical research on DMD patients has shown that patients who have a greater number of T cells (immune cells) in the blood that express high levels of CD49d (CD49dhiT-cell) are associated with both more severe and rapid disease progression, with an increase in the number of CD49dhi T cells associated with reduced walking capacity. Corticosteroids did not reduce these CD49dhi T cells. ATL1102 has been shown to block CD49d (VLA-4) expression on lymphocytes (including T cells), reduce immune cell numbers (including T cells), and to be highly effective in reducing inflammatory brain lesions in MS patients after only 8 weeks of dosing.

The Company is conducting a Phase II clinical trial of ATL1102 in DMD patients at the Royal Children’s Hospital Melbourne. For more details on the trial please click on the following link or paste in your browser http://www.ANZCTR.org.au/ACTRN12618000970246.aspx

For more details and updates please refer to specific company announcements

What is DMD?

Duchenne muscular dystrophy (DMD) is an X-linked disease that affects 1 in 3500 to 5000 live male births. DMD occurs as a result of mutations in the dystrophin gene which causes 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 (Pinto Mariz, 2015). 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, with respiratory, cardiac and cognitive dysfunction also emerging. With no intervention, the mean age of life is approximately 19 years. The 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. As a result, there is an acknowledged high unmet need for new therapeutic approaches for the treatment of inflammation associated with DMD.

Patient Stories

Jenn McNary, Patient Advocate

Jenn McNary is an educator and patient advocate with 15 years’ experience on the front lines of the rare disease clinical trial, drug approval and reimbursement process. A deeply informed, widely recognized voice for patients and their families and a trusted interpreter of patient experience for caregivers, clinicians, KOLs, regulators, legislators, and corporate stakeholders alike. Jenn also has two sons with DMD, Austin and Max.

Austin, Jenn McNary’s Son

A young adult living with DMD, Austin is an active member of the rare disease community. He is currently a student at Bunker Hill Community College with a goal of a Mechanical Engineering Degree. His accomplishments include an instrument of change award from the UCLA Center for Duchenne Muscular Dystrophy, a seat on the Parent Project Muscular Dystrophy (PPMD) Adult Advisory Committee, and multiple speaking engagements at rare disease events and congressional and regulatory briefings. Austin brings the team expertise in living with rare disease and mobility issues, participating in clinical trials and excelling in adaptive sports. In his free time, Austin is a soccer player on a local power soccer team and enjoys adaptive skiing.

Jake

“I would ask companies considering developing a therapy for non-ambulatory patients to please do so. It could make a huge difference in the quality of life of the non-ambulatory person and their caregivers/families. Non-ambulatory individuals seem to have been forgotten in the realm of research and developing therapies. People who are non-ambulatory are still important, productive individuals with a ton of living to do and much to offer the world.”Emily, Mother of Jake

Austin

“The most important things for Austin to maintain are the ability to self-feed, ability to use a keyboard, ability to lift a cell phone, ability to brush teeth, ability to adjust (put on, remove) headphones (gaming is a big deal in the Duchenne community).”– Trina, Mother of Austin

Jordan

“Any progress in helping improve or maintain quality of life is vitally important and would be a game changer for all of us. It would improve self-esteem, independence, and mental outlook/emotional well-being for the patient and the caregiver.” – Regina, Mother of Jordan

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