• Researchers are studying a potential new treatment called taldefgrobep alfa for spinal muscular atrophy (SMA) in a clinical trial.
• This new medication may help people with SMA build stronger, healthier muscles.
• The trial is currently recruiting people with all SMA types who have all levels of mobility and have taken (or are taking) approved SMA treatments.

A new clinical study called the RESILIENT trial may help researchers develop a new medication for spinal muscular atrophy. During the study, researchers will learn more about the safety and effectiveness of the therapy. If you are interested in trying an additional treatment option, you may be able to enroll in the trial.

Which Medication Is Being Studied?

The RESILIENT trial is looking at a medication called taldefgrobep alfa. This medication works differently than other SMA therapies and may help people with the condition more effectively use their muscles.

Current SMA treatments target the nerves. SMA develops when mutations in the survival motor neuron 1 (SMN1) gene occur. This gene makes the SMN protein, which supports nerve health, so mutations can lead to nerve death. Current SMA treatments act as gene therapy that can reverse these problems and help the body make more SMN protein.

However, these treatments don’t directly promote muscle mass, which is also affected by SMA. This means that people with SMA often have problems with motor function and muscle weakness despite using SMA therapies. Taldefgrobep alfa is intended to work alongside these other gene therapy treatments to improve muscle function.

Muscles are controlled by many different proteins within the body. One of these proteins, myostatin, prevents muscles from getting bigger. Taldefgrobep alfa blocks myostatin, which may help the muscles grow. When this medication is used along with other SMA treatments, people with the condition may experience improvements of multiple symptoms.

In a press release about the new study, a lead researcher talked about the need for better SMA treatments. “There have been great strides in advancing therapeutics in this challenging disease,” said Dr. Irfan Qureshi, senior vice president of neurology at Biohaven, the manufacturer of taldefgrobep alfa. “Yet, there remains a significant unmet need to address the residual weakness and functional impairments, such as difficulty walking, that are caused by the disease.”

The RESILIENT Trial

The study aims to enroll between 180 and 225 people with SMA. Recently, researchers announced that they had enrolled their first participant.

RESILIENT is a phase 3 study, meaning it has already gone through testing in phase 1 trials, which aim to test whether a treatment is safe, and phase 2 trials, in which researchers measure whether the medication is effective and continue to look for possible side effects.

Phase 1 and 2 trials of taldefgrobep alfa were conducted in people with Duchenne muscular dystrophy (DMD), another type of neuromuscular disease. In these studies, the drug helped reduce myostatin levels and didn’t lead to any serious side effects. However, taldefgrobep alfa didn’t help improve motor function for people with DMD.

Researchers now want to see whether this drug may help block myostatin and relieve symptoms for people with SMA. If clinical trials show that taldefgrobep alfa is safe and effective in the treatment of SMA, the U.S. Food and Drug Administration (FDA) may approve the drug as a treatment to be used by people with the condition. At the moment, however, the drug is only available through the RESILIENT trial.

Who Can Join the Trial?

To qualify for the trial, potential participants:

• Must be between the ages of 4 and 21
• Must be currently taking a disease-modifying therapy (DMT), such as nusinersen (Spinraza) or risdiplam (Evrysdi), and/or have previously taken onasemnogene abeparvovec-xioi (Zolgensma)
• Can have any type of SMA
• Can have any level of mobility
• Can’t have previously used antimyostatin therapies (medications that block the myostatin protein)
• Can’t have a history of spinal fusion within six months of screening
• Can’t have a shunt that helps drain fluid from the central nervous system (brain or spinal cord), or a catheter implanted into the central nervous system

If you are interested in learning more about the trial or seeing if you can participate, you may want to speak with your health care team. You can also learn more at the National Institute of Health’s official web page for the study or contact the pharmaceutical company running the study at clinicaltrials@biohavenpharma.com.

Find Your Team

On mySMAteam, the social network for people with spinal muscular atrophy and their loved ones, more than 1,600 members come together to ask questions, give advice, and share their stories with others who understand life with SMA.

Do you have questions about clinical trials? Are you thinking about participating in one? Share your thoughts in the comments below, or start a conversation by posting on mySMAteam.