As the Alzheimer’s disease world continues to closely follow the development of therapies that break up clumps of beta amyloid on patients’ brains, Roche is placing a bet on another approach via a deal that secures rights to a clinical-stage UCB compound that targets tau, another protein associated with the neurodegenerative disorder.
According to terms announced Wednesday, Roche is paying $120 million up front for global rights to the UCB drug, UCB0107. Belgium-based UCB remains responsible for conducting and funding a proof-of-concept study in patients with Alzheimer’s. After that study is complete, the Swiss pharmaceutical giant’s Genentech subsidiary may choose to continue the drug’s development or return its rights. If Roche develops the drug further, it will owe additional payments tied to the clinical and regulatory progress of the compound. The breakdown of those payments wasn’t disclosed but the companies say UCB could earn nearly $2 billion.
Tau is a protein that’s expressed in the central nervous system. In some neurodegenerative diseases, this protein misfolds and builds up into what look like tiny tangles on the brain. These tangles damage cells and lead to the death of neurons. Along with amyloid, tau is considered a hallmark sign of Alzheimer’s disease. One theory about Alzheimer’s posits that this pathological tau spreads from neuron to neuron, contributing to the progression of the disease. UCB0107 is an antibody drug designed to bind to the protein, stopping its spread.
Alzheimer’s disease drug research has focused mostly on drugs that target amyloid. Many of those drugs have failed in late-stage studies, including programs backed by Roche. But Biogen (NASDAQ: BIIB) and Eisai are keeping the amyloid hypothesis alive. Earlier this month, the partners completed the FDA submission for their amyloid-busting drug, aducanumab. That drug was initially shelved after appearing to fall short of its Phase 3 goal until Biogen revealed the analysis from a larger dataset, which showed it met the goal of one of two-late stage studies.
Eisai is leading the development of another drug covered by the partnership, BAN2401. That drug missed its Phase 2 goal at 12 months, but analysis after 18 months showed better results. Ivan Cheung, Eisai’s chairman and president of the company’s neurology business group, told Xconomy in an interview that the key for both aducanumab and BAN2401 was finding the right dose to show an effect. He added that there are many parts of the amyloid cascade that can be hit with a drug. “If you hit the wrong part, some [drugs] don’t work,” he said. “Aducanumab and BAN2401 are a different story.”
UCB isn’t alone in its pursuit of a tau-targeting alternative to amyloid Alzheimer’s drugs. AC Immune (NASDAQ: ACIU), is developing its small molecule, ACI-3024, under a partnership with Eli Lilly (NASDAQ: LLY). Last year, startup Pinteon Therapeutics revealed new financing and its antibody approach to blocking tau’s spread.
Tau is also associated with other brain diseases. In addition to Alzheimer’s, UCB is developing UCB0107 for progressive supranuclear palsy (PSP), a neurological disorder that causes problems with walking, balance, eye movements, and swallowing. UCB’s deal with Roche does not include PSP, and the Belgian company will continue to develop the drug for that disorder; a confirmatory Phase 3 study is expected to start in the second quarter of next year.
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