Biogen ($BIIB) is aggressively pursuing neurodegenerative diseases, such as spinal muscular atrophy and Alzheimer’s disease. And it’s hoping its candidates there help to establish a new era for the company. But in its traditional stronghold of multiple sclerosis, the biopharma is struggling as it drops one late-stage candidate and advances another despite a Phase II failure this summer.
The company has discontinued development of amiselimod (MT-1303), a Phase III-ready, oral S1P receptor candidate that it just in-licensed a little more than a year ago from Mitsubishi Tanabe. Biogen paid $60 million up front under the deal, which also had up to $484 million in milestones.
“As we looked at how the landscape was evolving, both in terms of a regulatory competitive landscape, changing features there, and the corresponding fit with our strategic priorities about where we could best allocate resources, based on our own expertise and competency, we just determined that it wasn't as good of a fit as other things which we could allocate our resources towards,” said Biogen EVP of R&D Michael Ehlers on the Oct. 26 call about the decision.
This could seem like a vote of confidence for Celgene ($CELG), which has its own S1P modulator, ozanimod, that’s in Phase III testing for relapsing multiple sclerosis and ulcerative colitis. Celgene gained ozanimod under its $7.2 billion acquisition of Receptos last year.
Biogen is still advancing its opicinumab to treat MS, despite a Phase II trial failure for the candidate in June. The biopharma defended the continued development of the monoclonal antibody, which missed both a primary and secondary endpoint in the study.
The strategy is to narrow the population based on MRI imaging data; about one-quarter of patients in the study had MRI evidence that indicated reduced myelination and intact axons at the start of the study. It’s these patients that Biogen expects to continue to pursue.
“Within the 10 mg/kg dosing arm of the study, we identified a treatment effect in patients whose MRI scans had specific features suggesting evidence that reduced myelination and intact axons at study entry. This subset represented approximately one-quarter of all patients in the Synergy study,” said Ehlers.
He continued, “Based on these data, we hypothesize that patients with similar features may have the potential to benefit from treatment with opicinumab. We are in the final stages of designing the next study, and look forward to providing an update in the coming months.”
Biogen is also focused on another pair of high-profile late-stage programs: nusinersen to treat spinal muscular atrophy (SMA) and aducanumab to treat Alzheimer’s disease. For the former, which is partnered with Ionis Pharmaceuticals ($IONS), the biopharma has filed with the FDA and EMA for approval.
Aducanumab is under the spotlight after positive Phase Ib data, which was followed by Fast Track designation from the FDA in September. The drug is in a pair of ongoing Phase III trials, ENGAGE and EMERGE. Biogen plans to offer more detailed Phase 1b data at the Clinical Trials on Alzheimer's Disease conference in San Diego, CA, on Dec. 9.
“We're at the beginning of a transformative era in neurodegeneration, which could result in a new era for Biogen as well,” summed up outgoing Biogen CEO George Scangos, whose replacement has yet to be named. “We're already starting to see the types of discoveries that can be expected as science advances and innovative trial designs yield previously unseen results and insights.”
He added, “We believe the early data for aducanumab point to additional opportunities to invest in novel science, to address Alzheimer's disease and other neurodegenerative diseases. The insights we've gained from the Synergy trial are teaching us more about remyelination, and we're enthusiastic about moving opicinumab forward. And the encouraging results we're seeing from nusinersen point to the possibility that there can be entirely new mechanisms and modalities for treating the many daunting diseases of the central nervous system.”