Sivakumar, Kumaraswamy, Cochrane, Thomas I., Sloth, Birgitte, Ashar, Hardik, Laurent, Didier, Tanko, Laszlo and Amato, Anthony A. (2020) Long-term Safety, Tolerability, and Efficacy of Bimagrumab (BYM338) in Sporadic Inclusion Body Myositis: Results of an Open-label Extension Study. Neurology, 95. ISSN 1526-632X

Abstract

Objective: To assess the long-term safety, tolerability and efficacy of multiple doses of bimagrumab in participants with sporadic inclusion body myositis (sIBM) who completed previous core study.

Methods: In this multicenter, open-label extension study, 10 adults received bimagrumab 10 mg/kg IV every 4 weeks up to 2 years. Safety (primary endpoint) was assessed by recording adverse events (AEs). Efficacy was assessed by change from baseline in thigh muscle volume (TMV), lean body mass (LBM), 6-minute walk distance (6MWD), hand grip and quadriceps strength.

Results: Participants had a mean (standard deviation [SD]) age of 70.1 (10.4) years. All participants (n=10) discontinued the treatment due to early termination of the study (n=7) or AEs (n=3; myocardial infarction, esophageal carcinoma, and dementia, none of which were treatment related). The most common AEs were muscle spasms and falls (both 9/10, 90%), followed by diarrhea (6/10, 60%), acne and skin abrasion (both 5/10, 50%). At Weeks 8 and 16, mean TMV increased from baseline by 4.1% (4.3) and 4.5% (6.3). Mean LBM increased from baseline and was sustained at 6.9% (3.9) at Week 76. Means of 6MWD showed a progressive decline from baseline to Week 104 accompanied by modest numerical increase in hand grip strength and no significant changes in quadriceps strength.

Conclusion: Treatment of up to 2 years with bimagrumab had a good safety profile and was well-tolerated in participants with sIBM. Despite an increase in muscle mass and maintenance of strength measures noted on a group level, heterogeneity of treatment responses prevent us from drawing firm conclusion about impact on mobility.

Item Type:	Article
Keywords:	Bimagrumab, sporadic inclusion body myositis, thigh muscle volume, lean body mass, 6-minute walk distance
Date Deposited:	24 Dec 2020 00:45
Last Modified:	24 Dec 2020 00:45
URI:	https://oak.novartis.com/id/eprint/40848