5 Experimental SMA Therapies You Might Find Interesting
1. AVXS 101
Recently, AveXis’ gene therapy candidate AVXS-101, the only gene therapy for spinal muscular atrophy currently in human testing, has been granted FDA orphan drug status for treatment of all types of SMA. It was also granted FDA breakthrough therapy designation and fast track designation. Read about it and other SMA therapy developments here.
2. CK-2127107 (CK-107)
In collaboration with Astellas, Cytokinetics is developing CK-2127107 (CK-107), a new skeletal muscle troponin activator as a potential treatment for people living with SMA. CK-107 has completed five Phase 1 clinical trials in healthy volunteers, and is currently the subject of two Phase 2 clinical trials.
Biogen and Ionis Pharmaceuticals recently announced that nusinersen, their investigational drug for treating SMA, met the primary end point for the interim analysis of the ENDEAR Phase 3 trial evaluating nusinersen in infantile-onset SMA.
PTC’s more developed SMA drug candidate, RG7800, is the subject of a Phase 2 trial in adult and pediatric SMA patients, but dosing was suspended and the trial was placed on clinical hold to investigate a non-clinical safety finding observed in a longer-term animal study.
5. Gene Therapy
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