aTyr Pharma has started a Phase Ib/II trial of Resolaris for patients with early onset facioscapulohumeral muscular dystrophy (FSHD).
FSHD is a rare and severe genetic myopathy for which there are currently no approved treatments.
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This trial is part of an expansion of the company’s Resolaris clinical programme in rare myopathies with an immune component (RMICs).
The multicentre, open-label escalation trial is designed to evaluate the safety, tolerability, immunogenicity, and biological activity of Resolaris in early onset FSHD.
The Phase Ib/II trial will enrol about 16 FSHD patients who showed signs of the disease before they were ten years old.
The clinical trial sites are currently preparing to screen patients for inclusion in the study.
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By GlobalDataIn the trial’s first stage, about eight patients between the ages of 16 and 25 years old will be enrolled.
The second stage is expected to include up to eight patients between the ages of 12 and 15 years.
aTyr Pharma CEO and executive chairman John Mendlein said: "The initiation of our early onset FSHD Resolaris program is an important step in addressing the needs of some of the most severely affected FSHD patients.
"With this trial, we continue to advance our new class of Physiocrine-based therapeutics to harness the body’s natural immune processes in rare muscle disease."
Including both young adults and children, the early onset FSHD research programme is the third trial to be initiated by aTyr Pharma recently.
The US-based company also began a Phase Ib/II trial in limb girdle muscular dystrophy (LGMD2B) and a Phase Ib/II clinical trial evaluating Resolaris in adult FSHD patients.
All three trials will evaluate Resolaris’ impact on the immune component of the disease, using blood borne markers and magnetic resonance imaging (MRI) of skeletal muscle.
