US-based biotherapeutics firm aTyr Pharma is expanding its Resolaris clinical programme in rare myopathies with an immune component (RMICs) with the start of a Phase Ib/II clinical trial in patients with limb girdle muscular dystrophy (LGMD) 2B.
The international, open-label, intra-patient dose escalation Phase Ib/II trial will evaluate the safety, tolerability, immunogenicity and activity of Resolaris in adult patients with LGMD2B, a severe genetic muscle disease with immune cell invasion in afflicted skeletal muscle.
The trial will include adult patients with facioscapulohumeral muscular dystrophy (FSHD) to further augment the company’s ongoing, blinded Phase Ib/II trial of Resolaris in adult patients with FSHD and to inform subsequent later-stage trial considerations.
Characterised by an immune component in the effected skeletal muscles, both LGMD2B and FSHD are progressive, debilitating muscle diseases.
CEO and executive chairman of aTyr Pharma John Mendlein said: "Our initiation of a LGMD2B patient trial represents our first indication expansion in RMICs with Resolaris.
"Although the genetic basis for LGMD2B is substantially different from FSHD, both patient groups develop an immune component as a part of the pathophysiology of the disease.
"The current standard of care potentially includes surgical interventions or use of devices, but no approved therapies.
"We believe Resolaris, derived from a protein released by human skeletal muscle cells, will promote the immune system’s natural homeostatic processes and will result in improved outcomes for those suffering from these rare diseases."
A total of 16 patients, including up to eight patients with LGMD2B and up to eight adult patients with FSHD are expected to be enrolled in this trial in the US and Europe.
The company intends to start a third Phase Ib/II clinical trial this month to specifically study patients with early onset FSHD.
In all three trials, adult patients with FSHD, early onset FSHD and LGMD2B, will evaluate Resolaris’ impact on the immune component using blood-borne markers and magnetic resonance imaging (MRI).