Ongoing clinical trials are exploring new therapeutic avenues for spinal muscular atrophy (SMA), alongside studies aimed at optimizing existing treatments. These efforts span various companies and involve diverse approaches, from gene therapies to small molecule drugs.
Advancing SMA Therapies: A Look at the Pipeline
Currently, over 20 SMA treatment therapies are under development by more than 18 companies. These include therapies from Scholar Rock (apitegromab), Biogen (nusinersen), Astellas Pharma, Roche (GYM329, risdiplam), and others. These emerging therapies are anticipated to significantly impact the SMA market in the coming years.
Roche's Risdiplam Studies
In April 2024, Roche initiated a Phase IV open-label study to evaluate the effectiveness and safety of risdiplam (Evrysdi) when administered as an early intervention in pediatric patients with SMA who have previously undergone gene therapy. This study aims to provide insights into the potential benefits of sequential therapies in SMA management.
Genentech, a member of the Roche Group, also announced results from a multi-center, longitudinal, prospective, non-comparative study assessing the long-term safety and effectiveness of risdiplam in both adult and pediatric participants with SMA. Participants will be followed for up to 5 years to monitor the drug's long-term effects.
Biogen's Nusinersen Dosage Study
Biogen is conducting a study to examine the clinical efficacy of nusinersen (Spinraza) administered intrathecally at higher doses in SMA patients. The study's primary objective is to measure the change in the Children's Hospital of Philadelphia-Infant Test of Neuromuscular Disorders (CHOP-INTEND) total score. The trial also assesses the safety and tolerability of the higher nusinersen doses.
Novartis Evaluates OAV101 Intrathecally
Novartis is evaluating the safety, tolerability, and efficacy of OAV101 (onasemnogene abeparvovec, Zolgensma) administered intrathecally in participants aged 2 to <18 years with SMA who have discontinued treatment with nusinersen or risdiplam. This Phase IIIb study aims to determine if intrathecal administration of OAV101 can provide additional benefits in patients who have previously received other SMA therapies.
NMD Pharma Explores NMD670 for SMA Type 3
NMD Pharma A/S announced results from a Phase 2, randomised, double-blind, placebo-controlled, 2-way crossover study evaluating the efficacy, safety, and tolerability of NMD670 in ambulatory adults with Type 3 SMA. This study represents a novel approach to potentially improve muscle function in this patient population.
These ongoing clinical trials and studies reflect a continued commitment to improving treatment options and outcomes for individuals affected by SMA. The diverse therapeutic approaches and optimization strategies being explored offer hope for further advancements in SMA care.
