EIP Pharma flies into a high-risk PhIIb Alzheimer’s study fueled with $20.5M from billionaire Len Blavatnik’s Access Industries

John Alam

Undeterred by the string of failures plaguing Alzheimer’s drug development, a young startup based in Cambridge, MA has tapped an unconventional source for $20.5 million to advance a repurposed drug in the clinic.

The new funds — coming from billionaire Len Blavatnik’s Access Industries — will power EIP Pharma’s Phase IIb study of neflamapimod, a p38 kinase inhibitor in-licensed from Vertex Pharma. Named REVERSE-SD, the high-risk trial will measure improvement in episodic memory as a primary endpoint and have several secondary endpoints including Clinical Dementia Rating Scale Sum-of-Boxes, Wechsler Memory Scale, and spinal fluid biomarkers of disease progression.

The focus here is on cognition — a gold standard cheered on by the FDA in a set of guidance documents released in February. They came just in time for EIP Pharma, which launched REVERSE-SD in March.

“That’s the first time they’ve said that because they recognize that it’s really hard in the early stages of Alzheimer’s and in mild Alzheimer’s disease to show an effect on, for example, rates of going into a nursing home, because that happens later on,” CEO John Alam tells me. “What matters to patients — when they first get diagnosed — is the memory function.”

By targeting dysfunctional neurons and the synapses that connect them, Alam believes, their drug can directly address memory deficits. As such, their study will be “really well aligned with the new FDA guidance document in terms of how we define early Alzheimer’s disease and how we’re measuring drug effect,” says Alam, a former Vertex CMO who headed Sanofi’s aging unit before moving on to start EIP Pharma.

Len Blavatnik

By the time EIP was founded in 2014, Vertex was already well into mid-stage development with neflamapimod, albeit in non-CNS disorders. Having inherited full sets of animal testing, Phase I and Phase II clinical data — alongside an established manufacturing process — the biotech went straight to Phase IIa, which yielded positive results earlier this year.

That’s advanced compared to the newcomers working on novel pathways in Alzheimer’s — a high profile startup, Denali Therapeutics, has only recently began Phase I testing; Rodin Therapeutics, which is working on synaptic resilience, has yet to enter the clinic — and Alam was quick to point it out.

“The things that are in Phase III today, the idea for them and conception working on them started 10 or more years ago,” he says. “What we’re working on, and the mechanism we’re working on, is truly the state of the art science of our understanding of [how] actually memory deficits form.”

Groundwork laid by Vertex had also allowed EIP Pharma to be capital efficient prior to this point, enlisting a small group of 10 individual investors to back their earlier study. And it’s that private network that connected Alam’s team to Access Industries, the first institutional backer.

While Access had not been known for making a lot of life science investments, Blavatnik, the eccentric billionaire founder of Access, had backed biotechs through Clal Biosciences and dished out biomedical science grants at top institutions around the world. He was, however, not directly involved in the round.

Access could not be reached for comment. According to Alam, the two parties bonded over a vision for the long term — a working drug for Alzheimer’s patients, not a quick exit.

Down the line, EIP Pharma’s approach to synaptic dysfunction could touch “more or less every CNS disorder,” from Parkinsons and Huntington’s disease to autism and stroke recovery mechanisms. But for now, they will concentrate on chasing the success that’s eluded one big player after another.

“We have a really good shot,” Alam says.

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