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raise $120 million from an all-star cast, a round that laid the groundwork for a $94 million IPO in 2016. That foundation has put Editas in position to possibly be the first U.S. company to test CRISPR-Cas9 drugs in human beings next year.
Nancy Simonian, Syros Pharmaceuticals
Simonian is a true Boston life sciences veteran, starting out at Harvard University and Massachusetts General Hospital before crossing to industry for roles at Biogen (NASDAQ: BIIB) and Millennium Pharmaceuticals. But readers nominated Simonian for her work growing a startup, Syros Pharmaceuticals (NASDAQ: SYRS), into a public company on the verge of its biggest tests yet.
Simonian has helped build a platform that the company has claimed can find drug targets others can’t—a “lens,” Simonian has said, to look into how genes are regulated and go awry in disease. Using that, the company is developing drugs that control the regulation of genes involved in disease. Along the way Syros raised over $120 million in private cash, another $58 million in an IPO, and now has two drugs in clinical testing. Readers nominated Simonian for those and other reasons. “Her leadership style is one of openness and collaboration,” says one.
The task ahead for Simonian is to prove that all this work can lead to an effective drug; Phase 2 data for Syros’s leukemia treatment, SYR-1425, are expected in the fall.
Jeff Albers, Blueprint Medicines
This past year, for the first time, the FDA approved a cancer therapy to treat tumors with a specific genetic signature, regardless of where in the body they originated. It was a landmark decision, a victory for the drug’s developer, Merck (NYSE: MRK), and the latest step forward for precision medicine. But the advance also bodes well for others with similar ideas, among them Blueprint Medicines (NASDAQ: BPMC), which is developing drugs for subgroups of patients with genomically defined cancers. Its CEO, Jeff Albers, is among our nominees.
Albers, a former Algeta executive, took over Blueprint in July 2014 when it was privately held, had gone through two CEOs in a year, and didn’t have a drug in clinical testing. He assembled the investor base for a $50 million crossover round, followed that up by forging partnerships with Alexion Pharmaceuticals and Roche, and led Blueprint to a $169 million IPO in April 2015. The company has doubled in size, has three drugs in clinical testing, and is currently worth close to three times its $18 IPO price. Shares have also doubled over the past year.
CROSSING THE FINISH LINE
Ed Kaye, Sarepta Therapeutics
In September 2016, the FDA approved Sarepta Therapeutics’s (NASDAQ: [[ticker:SRPT]) Duchenne muscular dystrophy drug eteplirsen (Exondys 51), largely off of evidence from a tiny, 12-person clinical trial. The decision was so controversial that it caused a rift within the highest levels of the agency.
We likely won’t know for a few more years whether the FDA made a great decision or a colossal blunder by approving eteplirsen, the first ever approved Duchenne drug. Setting that question aside though, Ed Kaye, who shifted from Sarepta’s chief medical officer to CEO in 2015, guided the company through a particularly volatile time to get eteplirsen over the finish line. Sarepta’s relationship with the FDA was in tatters at the time, and it desperately needed a win. As Kaye told Xconomy last year, Sarepta would’ve had to scrap eteplirsen, cut a bunch of jobs, and essentially become a preclinical company again if it had failed to gain FDA approval. But Kaye was able to strengthen Sarepta’s relationship with the agency, and steer the company to its first approval instead. Now Sarepta has the money to develop other Duchenne treatments, and just had its best sales quarter to date. And Kaye is a big reason why. The caveat to his nomination: Kaye stepped aside in June.
Lonnie Moulder, Tesaro
One strategy has picked up steam in biotech circles over the years: Assemble a veteran group of drug hunters, and have them find and acquire assets from elsewhere and successfully develop them. That sounds great on paper, but it hasn’t often been executed in practice. Tesaro (NASDAQ: TSRO) is showing what happens when the plan works, and that has earned its CEO a nomination from readers.
Lonnie Moulder is a veteran of MGI Pharma, which was sold to Eisai for $3.9 billion in 2008. In 2010, Moulder and former MGI colleague Mary Lynne Hedley signed on to lead Tesaro; he as CEO, she as president and chief operating officer. The plan: Sift through the vast universe of experimental cancer drugs to find and acquire treatments that may have fallen through the cracks, and develop them. Seven years later, Tesaro is a publicly traded company with two approved drugs and a market capitalization of $7 billion. In March, one of those drugs, niraparib (Zejula), became the first ever “maintenance” therapy approved for ovarian cancer, meaning it’s used to help keep cancers from recurring. Tesaro’s shares are now trading nearly 10 times their IPO price in 2012, and have nearly tripled in value since May 2016.