Despite a litany of high profile failures among drugs to fight Alzheimer’s disease, biotechs and pharma companies continue to pour billions of dollars into the fight. The latest firm to get a financial boost is a startup from Cambridge, MA, that until today has been known as Neurophage Pharmaceuticals.
The company has raised $47 million from current backer Merieux Development and a group of high net worth individuals, and changed its name to Proclara Biosciences. The new name, according to CEO Franz Hefti, comes in conjunction with a key milestone: Proclara will for the first time test one of its drugs in Alzheimer’s patients.
The trial is a long time coming for Proclara. It was formed out of Harvard University by Jonathan Solomon and Hampus Hillerstrom in 2007, and has since raised $110 million from Merieux, Shire, and others including Smedvig Capital founder Peter Smedvig. (Shire is no longer involved, according to Hefti.)
The company has yet to be sold or go public, meaning investors have been waiting a while for returns. Proclara will now need a bigger windfall to make those returns materialize.
“We are very fortunate to have these investors,” Hefti says. “They are very patient, very supportive, and would like to contribute to finding effective therapy for [Alzheimer’s].”
Solomon is no longer CEO, though he remains a consultant; Hillerstrom is still chief financial officer and executive vice president. Hefti says that Solomon stepped back at the end of 2015 because his family wanted to move back to Israel. Hefti has been involved with Proclara for years, first as a member of the scientific advisory board and most recently as its chief operating officer.
The prize for a successful Alzheimer’s drug, of course, would be significant. More than 5 million Americans currently have the disease, estimated by the Alzheimer’s Association estimates to reach about 14 million by 2050. But there are only two approved drugs—both help temporarily boost cognition in some patients—and a much larger list of clinical failures.
Most Alzheimer’s drugs have failed because, it seems, they aimed to clear deposits of the protein beta-amyloid from the brains of patients who already had full-blown disease—too late to help change their outlook. There is still debate how much those unusual deposits, called plaques, are the cause or the effect of the disease.
Researchers are now making big bets on tests to see if more rigorous patient selection, based on genetics and a less advanced disease state—that is, before the deposits have built up as much—might work better.
Answers to those questions could come over the next few years, as Biogen (NASDAQ: BIIB) and Eli Lilly (NYSE: LLY) present data from Phase 3 trials of two Alzheimer’s drugs, aducanumab and solanezumab. In an early stage, randomized, placebo controlled trial, Biogen’s drug was the first to ever show an effect on cognition. Biogen moved it into in late-stage trials that should wrap up in 2018. Lilly’s solanezumab failed an earlier study but is now being tested in patients with a less advanced form of Alzheimer’s. Data are due later this year.
Proclara is trying to hit simultaneously two types of misfolded proteins—beta amyloid and tau, the latter of which is also implicated in Alzheimer’s. Its lead drug, NPT088, is supposed to help clear up excess deposits of both in the brain, according to chief scientific officer Richard Fisher. Preclinical studies have also shown the drug might help clear tangles of alpha synuclein, a protein implicated in Parkinson’s disease.
To this point, NPT088 has only been tested in healthy volunteers. With the new funding, Proclara has begun its first test of NPT088 in Alzheimer’s patients, a randomized, placebo-controlled study. According to Hefti, Proclara will enroll up to 66 patients with “probable” Alzheimer’s disease, whom Hefti described as patients with cognitive decline and whose brain scans reveal amyloid and tau buildup.
Patients will get a dose of NPT088 or placebo once a month, for six months, and then get brain scans at the end of the study—data are expected by early 2018, according to Hefti.
If the follow-up scans show a reduction in the tangled proteins, Proclara could move NPT088 into two bigger, longer studies—one in Alzheimer’s, the other in Parkinson’s—designed to measure whether the drug is actually slowing the progression of patients’ disease, not just clearing out tau and amyloid deposits.
The cost of longer-term studies will be significant; Proclara is talking to institutional investors about its next round of funding. The hope, Hefti says, is to get a deal done next year.
The company’s name change is meant to help with the fundraising. Neurophage came from a fluky discovery by Beka Solomon, the mother of founding CEO Jonathan Solomon. She is a life sciences professor at Tel Aviv University, and in an experiment using filamentous bacteriophages—one kind of virus that infects bacteria—she found that the virus seemed to help break up amyloid plaques.
But Proclara is making protein-based drugs based on that discovery, not viruses. NPT088 is an engineered version of a protein found on that bacteriophage that the firm has fused to an antibody. The old name, Neurophage, was causing too much confusion, says Hefti.
“If you talk to investors, they’ll say, ‘Oh you’re the company with the phage,’” Hefti says. “We have a pretty sophisticated biologic [drug].”