Biogen initiates phase 3 trial of omaveloxolone for children with FA

CAMBRIDGE, Mass. - Biogen Inc. (NASDAQ:BIIB), a prominent biotechnology company with a market capitalization of $18.54 billion and an impressive gross profit margin of 75.65%, has begun dosing in the BRAVE study, a global Phase 3 clinical trial evaluating omaveloxolone in children aged 2 to under 16 years with Friedreich ataxia (FA), a rare neurodegenerative disorder. According to InvestingPro analysis, Biogen maintains a strong financial health score, positioning it well for continued research investment.

The study will include approximately 255 children with FA, both ambulatory and non-ambulatory, randomized 2:1 to receive either omaveloxolone or placebo once daily for 52 weeks. Participants will then have the opportunity to enter an open-label extension lasting up to week 104.

Omaveloxolone is currently marketed as SKYCLARIS in over 40 countries for treating FA in adults and adolescents aged 16 and older. This trial aims to address the unmet need in younger patients, as symptoms typically begin in childhood with earlier onset associated with faster disease progression.

"Building on the work of Reata we have been urgently advancing the pediatric development plan for omaveloxolone and are thrilled that the Phase 3 BRAVE study has now begun," said Stephanie Fradette, Head of the Neuromuscular Development Unit at Biogen.

The primary outcome measure for the first part of the study is change from baseline in Upright Stability Score, a subscale of the modified FA rating scale that measures disease progression.

"Early onset patients often have the most aggressive and fast progressive form of Friedreich ataxia," said Susan Perlman, Professor of Neurology at UCLA. "This vulnerable population faces significant unmet need, with no approved treatments currently available."

Enrollment has begun in the United States with plans to open study sites globally. The information is based on a press release statement from Biogen.

In other recent news, Biogen has been the focus of several analyst evaluations, emphasizing its financial and strategic developments. Goldman Sachs maintained a Buy rating with a $196 price target, noting the strong performance of Biogen’s partner, Eisai, whose Alzheimer’s treatment Leqembi reported fiscal year-ending revenues of approximately $299 million. This exceeded Eisai’s forecast of $280 million, driven by robust sales in Japan and China. Meanwhile, H.C. Wainwright also retained a Buy rating but adjusted Biogen’s price target to $187, citing a 395% year-over-year increase in Leqembi sales. The firm highlighted the growing acceptance of anti-amyloid therapies and the establishment of specialized clinics like Stanford’s Alzheimer’s Care and Treatment clinic.

UBS reiterated a neutral rating on Biogen, maintaining a $119 price target, reflecting cautious optimism about Biogen’s Alzheimer’s pipeline despite timeline uncertainties. H.C. Wainwright expressed optimism about Biogen’s nephrology potential, particularly with the development of felzartamab, a promising anti-CD38 antibody. The firm emphasized the potential advantage of Biogen’s shift toward rare kidney diseases, supported by encouraging Phase 2 results. These developments underscore Biogen’s strategic focus on expanding its therapeutic areas and leveraging its existing expertise in rare diseases.

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