- Biogen on Monday said it acquired rights to an experimental multiple sclerosis drug from China-based biotech Innocare, investing in a mainstay business for the biotech that has been challenged by competitive threats and patent losses.
- Per deal terms, Biogen will pay Innocare $125 million upfront for rights to orelabrutinib, a drug that's currently in Phase 2 testing in patients with the relapsing form of MS. Biogen could add as much as $812 million more to the deal if the drug hits certain development milestones and sales targets.
- The licensing deal is the latest indication of the industry's interest in aiming drugs known as BTK inhibitors at autoimmune diseases like MS. Sanofi, Roche and Merck KGaA are each testing BTK inhibitors in Phase 3 trials, and Biogen completed an early-stage study of a similar drug last year. The company claimed that Innocare's treatment has certain "unique characteristics" that could set it apart from the rest.
Ever since early June, the conversation surrounding Biogen has been centered on the approval of its Alzheimer's drug Aduhelm, and for good reason. Aduhelm's clearance was one of the most controversial in the history of the Food and Drug Administration, and the ripple effects of that decision on the U.S. healthcare system and on Biogen will be felt for years.
But well before Aduhelm's approval, Biogen had other concerns. Chief among them is the future of the multiple sclerosis business for which the company is best known.
Last year, a federal court invalidated a key patent covering Biogen's best-selling drug, Tecfidera, although an appeal is ongoing. Tecfidera sales have since sunk, but revenue from Biogen's other MS drugs have been slipping as well amid the emergence of new medicines. First quarter sales were down by roughly 26% compared to last year, a trend that's likely to continue as Biogen predicted continued "rapid erosion" of Tecfidera sales in the U.S.
Biogen gets royalties on sales of Roche's Ocrevus, which is fast becoming the top-selling MS drug globally. But the biotech company has been working to bolster its business in other ways, too. One of them involves turning a BTK inhibitor — a type of drug that's become a standard treatment for certain blood cancers — into a new medicine for MS.
Over the past few years, multiple drugmakers have begun testing in MS BTK inhibitors that, unlike the cancer medicines Imbruvica and Calquence, are meant to pierce the blood-brain barrier. The concept takes a page from Ocrevus, which targets a protein on the surface of the immune system's B cells. The approach has proven very effective at delaying MS symptoms, but wipes out B cells along the way.
BTK inhibitors, by comparison, block an enzyme that helps B cells mature and may spare healthy ones. The BTK enzyme is also involved in activating several types of immune cells, which could make the drugs helpful tools for treating multiple forms of MS — including the rarer "primary progressive" version — if they can effectively get to the brain.
That potential spurred Sanofi to pay $3.7 billion to buy Principia Biopharma and its brain-penetrating BTK blocker tolebrutinib last year. Other programs have emerged from Roche and Merck KGaA as well, and Biogen has a drug of its own, called BIIB091, that completed Phase 1 testing last year. According to the clinicaltrials.gov database, another Phase 1 study testing modified formulations of the drug is recruiting, with results expected later this year.
Adding olebrutinib to the mix, then, gives Biogen two similar drugs. But there are key differences between each of them. Chief medical officer Al Sandrock, for example, said in a statement that the "high selectivity and CNS penetrance" of Innocare's drug give it a profile that "may translate to potential clinical advantages" over similar drugs. BIIB091, while also meant to be selective, is a “reversible” BTK blocker, according to a Biogen spokesperson.
While BTK inhibition is an “exciting new frontier in MS,” there is only “limited data ... currently available for the class beyond proof of concept,” according to the spokesperson. Having both drugs, the spokesperson said, enables Biogen to assess their profiles and “potentially increase our chances of delivering a meaningful therapy.”
Even if so, Biogen is behind its rivals. A 160-patient, placebo-controlled Phase 2 study is underway, with primary results expected in 2024, according to clinicaltrials.gov. Sanofi, Roche and Merck KGaA could each have Phase 3 results in hand by then.
Deal terms give Biogen worldwide rights to the drug for use in MS and everywhere outside of China for other autoimmune diseases. Innocare still has full rights to the treatment for use in cancer.
Editor's note: This story has been updated to include comments from Biogen.