Perelman-backed vTv Therapeutics bets on anti-Alzheimer’s drug
vTv Therapeutics celebrates its 2015 initial public offering by ringing the Nasdaq stock market opening bell. Led by CEO Steve Holcombe, the drug developer had a market cap of $217 million in early February. Billionaire Ronald Perelman controls 75% of the stock.
By Mark Tosczak
It’s been 14 years since the Food and Drug Administration approved a new Alzheimer’s drug — a long, frustrating drought for more than 5 million U.S. patients and their families. Despite the injection of hundreds of millions of dollars into research — including Microsoft founder Bill Gates’ recent pledge of $100 million — there is no approved treatment to alter the relentless course of the disease.
Of some 17 drugs in the final stages of testing before the FDA considers approval, one is from vTv Therapeutics, a High Point-based company that expects to report the results of the first of two critical trials this spring. vTv is close to completing testing on the anti-Alzheimer’s drug Azeliragon that it has been developing for nearly 20 years, funded in large part by billionaire investor Ronald Perelman.
With Alzheimer’s and dementia care costing more than $250 billion a year in the U.S. alone, the payoff of a successful treatment is significant. So are the risks: Tested Alzheimer’s therapies between 2000 and 2012 had a 99.6% failure rate, while the failure of cancer drugs in the same period was 81%.
“There’s still a lot about the disease we don’t understand,” says James Hendrix, director of medical and scientific relations for Global Science Initiatives at the nonprofit Alzheimer’s Association, a Chicago-based research and advocacy group. “That makes it challenging to really design and develop therapeutics.”
Scientists don’t know exactly what causes Alzheimer’s, but they know more than ever about the effects of the disease. Proteins accumulate in the brain and damage brain cells, interfering with memory and other cognitive functions. Though Alzheimer’s isn’t the only cause of dementia, it’s the most common, responsible for an estimated 60% to 80% of all dementia cases.
“We have only a few treatments, and they’re not very good,” says Daniel Kaufer, chief of Cognitive Neurology & Memory Disorders at UNC Health Care and director of the UNC Memory Disorders Program. “The only real hope we have of turning this around is identifying an intervention early in the disease process.”
By the time cognitive impairments are evident and Alzheimer’s diagnosed, the disease is already several years into its course, says Kaufer, who has consulted for another North Carolina company that has pursued Alzheimer’s treatments, Chapel Hill-based Zinfandel Pharmaceuticals Inc.
Zinfandel had partnered with Japan-based Takeda Pharmaceutical Co. in an $87 million deal to test whether a diabetes drug, pioglitazone, might delay the onset of Alzheimer’s. The study attracted considerable interest among Alzheimer’s researchers because it had enrolled thousands of patients in a Phase 3 trial. But the Zinfandel-Takeda trial was called off in January after an interim analysis showed the drug was having little effect.
That leaves vTv as the only North Carolina company with a potential Alzheimer’s treatment that might be submitted for FDA approval in the next year or so, assuming its studies produce positive results.
vTv has high hopes in slowing the disease with Azeliragon. “This was the first potential therapy we discovered as a company,” CEO Steve Holcombe says. Azeliragon is designed to slow Alzheimer’s by blocking inflammation and protein buildup in patients’ brains. Its potential positive effects are based in part on the thesis that inflammation is a critical component of Alzheimer’s.
vTv also has candidates in the works for diabetes and other conditions, but Azeliragon is the furthest along, with two Phase 3 trials in progress, each with about 400 participants. Results from the first of those trials are expected in late March or April, Holcombe says.
Perelman, worth an estimated $18.2 billion according to Bloomberg, has funded much of Azeliragon’s development through his MacAndrews & Forbes investment firm. Best known for stakes in Revlon, Harland Clarke and Humvee maker AM General, he’s also injected about $250 million into vTv since the late 1990s, when the company was founded as TransTech Pharma. He put up another $10 million in December. Perelman controls more than 75% of the company’s common stock.
vTv has brought in more than $200 million over the years from partnerships with other drug companies and, in a 2015 IPO, raised $117 million. Eyes will be on its Phase 3 trials this year — if successful, Holcombe believes the company will attract more capital and perhaps big pharma suitors who might be interested in licensing the drug.
But Zinfandel’s study shows there are no guarantees in the bruising world of Alzheimer’s drug discovery. In the last 18 months, about half a dozen Phase 3 trials for Alzheimer’s drugs have failed. Pfizer, the world’s third-largest drug company, announced in January it would exit the field altogether.
Meanwhile, the toll of Alzheimer’s is expected to grow dramatically. By 2050, according to some estimates, as many as 14 million Americans could have the disease.
“This single disease,” says Hendrix, from the Alzheimer’s Association, “has the potential to bankrupt Medicare.”