Cytokinetics to Present Latest Updates on Therapy Candidate at Cure SMA Conference


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AVXS-101 update

Cytokinetics will present the latest developments on its drug candidate CK-2127107, also known as CK-107, for spinal muscular atrophy (SMA) during the Cure SMA 2017 Annual SMA Conference June 29 to July 2 in Orlando, Florida.

Developed in collaboration with Astellas, CK-107 was designed to increase muscle function and improve physical performance in people with SMA.

A comprehensive Phase 1 clinical evaluation of CK-107 in healthy subjects has been completed, with a total of five studies and 123 participants. These studies provided positive safety and tolerability profiles, as well as positive drug distribution and activity results, supporting further testing.

Results of a preclinical study with different types of SMA mouse models similar to SMA type 2 and to SMA types 3 and 4 showed that single and ascending doses of CK-107 administrated locally increased muscle response to nerve stimulation, according to a company presentation. This observation supported the therapeutic potential of CK-107 for improving muscle function in SMA patients.

These findings will be the subject of a poster presentation titled “The Fast Skeletal Muscle Troponin Activator, CK-2127107, Improves Muscle Function in Mouse Models of Spinal Muscular Atrophy,” presented by Eva Chin, PhD, director of pharmacology in Muscle Biology & Therapeutics at Cytokinetics, at the Annual SMA Conference.

An oral presentation titled “Clinical Trial Update of CY 5021: CK-2127107, a Selective Activator of the Fast Skeletal Muscle Troponin Complex, for the Potential Treatment of Spinal Muscular Atrophy,” will also be presented by Stacy Rudnicki, MD, director of clinical research and development at Cytokinetics, at the conference.

From the Phase 2 clinical trial (NCT02644668) of CK-2127107 in SMA types 2, 3, and 4 patients, Rudnicki will provide an overview on the participants’ baseline characteristics, and she will explain the reasons for patient screen failure observed in the trials’ first cohort.

Rudnicki will also participate in a panel discussion on SMA therapies in development.

In May 2017, the FDA granted orphan drug status to CK-2127107.

CK-2127107 is also under evaluation in a Phase 2 study (NCT02662582) for the treatment of patients with chronic obstructive pulmonary disease (COPD), to improve exercise tolerance and physical capacity.