Biogen Strikes Deal for Orelabrutinib, Now in Phase 2 Trial

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by Marta Figueiredo PhD |

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Biogen has signed a deal with InnoCare Pharma to acquire global commercialization and licensing rights to orelabrutinib, an experimental oral BTK inhibitor (BTKi) now being tested in people with relapsing-remitting multiple sclerosis (RRMS) in a Phase 2 clinical trial.

Under the terms of the agreement, Biogen will have exclusive rights to the therapy in the field of MS worldwide and of certain autoimmune diseases outside of China (including Hong Kong, Macau, and Taiwan), while InnoCare will retain the rights to it for certain autoimmune diseases in China.

InnoCare will also maintain orelabrutinib’s exclusive worldwide rights in the field of cancer, where the therapy is already approved for two indications in China.

The company will receive an upfront cash payment of $125 million from Biogen and is eligible for additional royalty payments, as well as up to $812.5 million if certain development, commercial milestones, and sales thresholds are met.

“Biogen is a recognized leader in neuroscience and we believe this transaction will help advance the development of orelabrutinib in MS,” Jasmine Cui, PhD, InnoCare’s co-founder, chairwoman, and CEO, said in a press release.

Alfred Sandrock, Jr., MD, PhD, Biogen’s head of research and development, said that “for over 30 years, Biogen has led in MS research and today has a leading portfolio of MS products.”

“We are focused on developing next-generation approaches that we hope will improve outcomes for those living with progressive and relapsing forms of MS,” Sandrock said, adding that the “unique characteristics of orelabrutinib, combining high selectivity and CNS [central nervous system] penetrance, may translate to potential clinical advantages relative to other BTKi programs.”

Other BTK inhibitors, such as Sanofi’s tolebrutinib (previously known as SAR442168), Roche’s fenebrutinib, and EMD Serono’s evobrutinib, are currently being evaluated as potential MS therapies in Phase 3 trials, with promising Phase 2 results. EMD Serono is known as Merck KGaA outside North America.

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Orelabrutinib is an orally available small molecule that potently and selectively blocks the activity of Bruton’s tyrosine kinase (BTK), an enzyme important for the activation of B-cells and microglia — immune cells that drive the abnormal immune responses in MS.

By suppressing BTK, orelabrutinib is designed to prevent the activation of these cells, thereby lowering inflammation and potentially slowing progression of all forms of MS.

According to InnoCare, the therapy was shown to block BTK’s activity by more than 90%, without interfering with the function of other enzymes, and to cross the blood-brain barrier. This highly selective membrane tightly regulates what substances from the bloodstream can access the CNS (the brain and spinal cord), and crossing it is often a challenge for CNS-targeting therapies.

The therapy also showed a favorable pharmacological, safety, and efficacy profile, allowing it to be given once daily at a low dose without compromising its ability to specifically and strongly suppress BTK’s activity.

“We are excited about the potential of orelabrutinib for the treatment of patients with all forms of MS given the potential efficacy and safety profile, plus a promising level of blood-brain barrier penetration,” Cui said.

In March, InnoCare launched a Phase 2 trial (NCT04711148) to evaluate orelabrutinib’s safety, pharmacokinetics, biological activity, and effectiveness in up to 160 adults with RRMS. Pharmacokinetics refers to the therapy’s movement into, through, and out of the body.

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Participants will be randomly assigned to receive a daily oral capsule of either one of three doses of orelabrutinib or a placebo for 24 weeks (six months), after which all will be given the experimental therapy for up to 120 weeks (more than 2 years).

The trial is currently enrolling patients at two U.S. sites, with additional sites in European countries planned. The study is expected to end in March 2024.

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