Results from a Phase 2 study of an Essential Tremor therapeutic shows improved upper limb tremor


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This increased inhibition is hypothesized to improve the symptoms of Essential Tremor.

New research shows promising results from a phase 2 trial of a drug for treatment of Essential Tremor, according to a study being presented at the MDS Virtual Congress 2021.

SAGE-324/BIIB124 is a positive allosteric modulator improving the ability of the inhibitory receptors to respond to a signal. This increased inhibition is hypothesized to improve the symptoms of Essential Tremor.

In this study, a Phase 2 trial of SAGE-324/BIIB124 has been performed to test the safety, efficacy, and tolerability of the neuroactive steroid. Patients with Essential Tremor were randomly assigned the drug or placebo group and tremor severity was assessed using the Essential Tremor Rating Assessment Scale (TETRAS) both before and after treatment. Upper limb tremor was found to be reduced in the treatment group relative to the placebo group.

Roongroj Bhidayasiri, MD, FRCP, Professor of Neurology & Director at Chulalongkorn University Hospital, commented on the study saying, “New therapeutic options for ET have been long awaited with classic orally approved medications consisting of propranolol and primidone. As GABA-A dysregulation is a well-known mechanism involved in ET, it is a potential target for new drug development that could be a future option for those who do not respond well to the first-line treatment or being considered an adjuvant therapy. SAGE-324/BIIB124 has recently been tested in a phase 2, placebo-controlled study, demonstrating significant results on a reduction of upper limb tremor performance subscale as shown by TETRAS item 4 score at 1-month when compared to placebo. These early findings, while promising, should be interpreted with caution as it is of a very short duration of 28 days involving a relatively small number of subjects without tremor characteristics and outcomes limited to a single rating scale. Moreover, a minority of subjects (23.5%) completed the study at the initial dosage of 60 mg, raising a possibility of intolerable adverse events at this dosage. Future studies should incorporate larger number of patients with detailed (clinical vs. digital) phenotypes and inclusion of various outcomes comprising of technology-based (preferably digital patient-reported outcome measures) and daily function assessments.”

The author of the study is employed by Sage Therapeutics.

About the MDS Virtual Congress 2021: Meeting participants gather to learn the latest research findings and state-of-the-art treatment options in Movement Disorders, including Parkinson’s disease. Over 8,000 physicians, scientists and medical professionals from more than 100 countries will participate virtually to view over 30 hours of educational content and 1,300 scientific abstracts submitted by colleagues from around the world.

About the International Parkinson and Movement Disorder Society:

The International Parkinson and Movement Disorder Society (MDS), an international society of over 11,000 clinicians, scientists, and other healthcare professionals, is dedicated to improving patient care through education and research. For more information about MDS, visit http://www.movementdisorders.org.

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