|Krisztina Zsebo, CEO of Celladon|
Based: San Diego
CEO: Krisztina Zsebo, Ph.D.
Clinical Focus: Cardiovascular disease
The Scoop: You don't often find three Big Pharma venture arms clustered together in a single round for a mid-stage heart-failure drug. But there they were--Pfizer ($PFE), Novartis ($NVS) and Johnson & Johnson ($JNJ)--all backing Celladon's $53 million round earlier this year.
The money is being used to finance a Phase IIb confirmatory trial for Mydicar, an experimental gene therapy aimed at an enormous potential patient population. In a small, 39-patient mid-stage study, the treatment registered a dramatic 88% drop in the risk for heart failure. And if the pharma companies see clear confirmation of efficacy with a clean bill of health on safety, Celladon may well find itself in a position where it can write its own ticket.
What Makes it Fierce?
The journey has neither been short nor easy for Celladon, a development company CEO Krisztina Zsebo described as an "old style" biotech, building a program from the scientific ground up by a team of researchers led by company co-founder Roger J. Hajjar, the director of the Cardiovascular Research Center at Mount Sinai School of Medicine. From the beginning, Celladon has been in it for the long haul.
The company was launched on a "glimmer and a hope," says Zsebo. Starting with the disease pathway, Celladon went on to develop a gene therapy that replaces a key enzyme--SERCA2a--lost in heart failure. Restoring that enzyme can play a big role in boosting heart muscle contractions, the kind of medical advance that can keep patients out of the hospital and at home, with improved physical performance.
"Pharma would never develop a drug like this," says the CEO. "It's too high-risk."
Gene therapy, of course, has been on a long roller coaster ride over the past two decades. But Zsebo, who was the 80th staffer at Amgen ($AMGN) and completed a stint as venture partner at Enterprise Partners Venture Capital, says that new technology has paved the way for a renaissance in the field.
"We really have nailed it," she says. The old vector technology used in gene therapy caused havoc. But the recombinant adeno-associated viral vectors Celladon relies on have fixed that problem, she says, providing a direct delivery to heart muscles.
Investigators conservatively don't expect to see conclusive 12-month IIb data until early 2015. It's theoretically possible that if the data is clean enough, regulatory groups in the U.S. and Europe may clear it under new unmet-need approval criteria. Typically, though, regulators have in the past required major Phase III programs for new drugs aimed at a big heart market. It's possible that one of the Big Pharma companies that paid for a front-row seat could step up with an offer Celladon couldn't refuse, either after the data is in or before. Unless, of course, the biotech comes up empty-handed.
Zsebo says she's excited by Celladon's prospects, hoping to demonstrate in uncertain terms that an old school biotech that raised $120 million could persevere for a decade and produce a blockbuster.
"The current state of venture financing is really a shame. The model is broken," Zsebo says. "And we really do need this type of vehicle."
If she's successful, Celladon may help fix things.
Investors: Pfizer Venture Investments, Lundbeckfond Ventures, Novartis Venture Funds, H&Q Healthcare/Life Sciences Investors, and GBS Venture Partners, Enterprise Partners Venture Capital, Johnson & Johnson Development Corp., LSP Life Sciences Partners, MPM Capital and Venrock Associates.