Alzheon returns, with a $47M NIH grant and one last shot at a pivotal PhIII Alzheimer’s success
Alzheon has spent four years searching for investors, public or private, willing to back a large study for their once-failed Alzheimer’s drug. On Thursday, they finally found a benefactor.
The NIH’s Institute for Aging awarded Alzheon $47 million over the next five years for a pivotal Phase III trial on their lead drug, ALZ-801. It’s not the IPO that CEO and founder Martin Tolar wanted — and twice filed for — but the company says it will be enough to start study they have wanted to get off the ground for four years.
Alzheimer’s trials are notoriously long, difficult to run and expensive, but Tolar told Endpoints News in an email that the funding would be enough to cover the full study. In its first IPO attempt, Alzheon had tried to raise upwards of $100 million to launch two Phase III trials. The new raise is roughly in line with its second IPO attempt, when they said $40 million would be sufficient to start and get data from a Phase IIb study.
The Phase III trial will enroll 300 patients and begin in the first quarter of 2021, the company said. In 2016, Tolar said they would need 500 people for the pivotal studies. On top of the new filing, the company raised $6 million last year, according to an SEC filing.
Alzheon’s drug is built on the beta amyloid hypothesis — the one that, after multiple major failures, seemed dead in the water before Biogen announced last year that a re-analysis of one of its Phase III trials showed their beta amyloid drug aducanumab was successful. A version of ALZ-801 has also failed once before. Alzheon licensed it from Neurochem (now known as Bellus Health) six years after it failed in a Phase III study.
Rebecca Edelmayer, director of scientific engagement at the Alzheimer’s Association, acknowledged that treatments built around amyloid hypothesis have seen their share of setbacks, but she argued that didn’t foreclose the possibility for success with new medicines with different development and amyloid-targeting strategies.
“That doesn’t mean all approaches to target amyloid are created the same,” Edelmayer told Endpoints. “They don’t attack amyloid in the same way, they don’t remove amyloid or prevent it from building up in the same way. It’s important we test all the approaches.”
Alzheon has adjusted the molecule so it’s absorbed more easily in the blood. They say they can succeed by giving it to patients earlier — a common refrain among Alzheimer’s drug developers — and by giving it to the right patients. The study will enroll patients with two APOE4 mutations, putting them at a greatly elevated risk of developing amyloid plaque buildup and Alzheimer’s itself.
“This innovative Phase 3 study is the first to initially focus on symptomatic APOE4/4 patients,” said Anton Porsteinsson, a University of Rochester Alzheimer’s researcher who will serve as lead investigator. “If these biomarkers correlate with clinical efficacy, this would enable their use as potential surrogate outcomes in Alzheimer’s prevention trials, allowing more efficient trial design.”