Cytokinetics Inc. (CYTK), which is developing its spinal muscular atrophy (SMA) drug CK-2127107 (CK-107) with Astellas Pharma, reported positive results from a pre-clinical study. (See also, AveXis SMA Gene Drug Reports Positive Data.)

The study was performed to assess the drug's effects on skeletal muscle function in two mouse models. The subjects had varying degrees of SMA symptoms, similar to Type 2, Type 3, and Type 4 SMA.

Improved Muscle Function

Administering a single dose of Cthe K-2127107 drug resulted in increased isometric force, which is used as a measure for muscle function evaluation in mouse models for SMA. A significant increase was observed in force output at adequate frequencies across all types of subjects. The results indicate that CK-2127107 has the potential to improve muscle function in the SMA disease.

“The increased muscle force at sub-maximal nerve stimulation frequencies in mice inform the potential for CK-2127107 to increase muscle function in patients living with motor neuron dysfunction,” said Dr. Fady I. Malik, Cytokinetics' executive vice president of research and development.

The positive pre-clinical results complements Cytokinetics’ ongoing Phase 2 clinical trial of CK-2127107 in adolescent and adult patients with SMA, where it is being evaluated on multiple measures of muscle function in Type 2, Type 3, or Type 4 SMA patients.

Spinal muscular atrophy (SMA) is a genetic disorder caused by the deterioration of specialized nerve cells, called motor neurons, and the disease affects the control of muscle movement. It can lead to paralysis and difficulty in performing basic tasks like swallowing and breathing. Type 1 SMA starts in patients under the age of six months and is the most severe form of the disease. Type 2, 3 and 4 SMA afflicts patients in the higher age groups.

Other companies developing SMA drugs include early-stage players like AveXis Inc. (AVXS), whose AVXS-101 recently reported positive results from an early-stage trial, and established players like Biogen Inc. (BIIB) and Ionis Pharmaceuticals Inc. (IONS), which are jointly developing SMA therapies like nusinersen. (See also, Biogen Gets Competition for SMA Therapy.)

Along with SMA, Cytokinetics' CK-2127107 is also in a Phase 2 trial for treating chronic obstructive pulmonary disease, where it is being evaluated for its effect on physical function and exercise tolerance.

Following news of the positive trial results, Cytokinetics stock surged 11.5% during pre-market hours on Wednesday, and was trading 4.25% higher in the afternoon.