While many large-cap biotech companies that presented during this year’s “ J.P. Morgan Healthcare” conference are retrenching and repurchasing stock, the small and medium cap biotechs are looking forward optimistically. Innovative firms are anticipating late-stage clinical trial results and, generally, good financial years.
BioTime’s agreement to acquire Geron’s embryonic stem cell program assets was one of the most-discussed conference highlights. “The concept of consolidating the intellectual property is strong enough to build a one-stop shop for the more than 600 patents and applications in this field,” said Michael West, Ph.D., CEO of BioTime.
The deal forms a new company, BioTime Acquisition Corporation (BAC), a wholly owned subsidiary of BioTime. “We know this is the first time we’ve had critical mass,” said Tom O’Karma, president and CEO of BAC, and former president and CEO of Geron.
As Dr. West stated, “We feel strongly about the power of pluripotency. With Geron’s assets, we can create a product model for cell therapy, so vials containing pure cells can be frozen and shipped like a pill. You can only do that if you have a pluripotent cell line.”
The company initially plans to address multiple sclerosis spinal conditions, enabling a single cell to treat many forms of MS spine and spinal injuries. “This has been demonstrated to be safe and feasible in humans, and we are ready to move it to other diseases,” O’Karma remarked.
At Vertex, “We’re sharpening our focus for the future to create transformative medicine for specialty markets—not drugs that are merely wanted, but drugs that are needed to transform lives,” said Jeff Leiden, M.D., Ph.D., chairman, president, and CEO. Dr. Leiden called innovation “the secret sauce to push the frontiers of personalized medicine.”
The company is interested in collaborative options for high-value targets that will help sustain growth. VX-509 for autoimmune diseases, for example, may be a platform for multiple disease therapeutics. Kalydeco™, currently a monotherapy for cystic fibrosis, also has the potential for label expansion. It was approved in the U.S. and Canada and will launch in Europe later in 2013.
The company just received breakthrough therapy designations from the FDA recently for VX-809 combined with ivacaftor for cystic fibrosis and for ivacaftor alone in people above age six who have the G551D mutation. Late-stage projects are under way for cystic fibrosis, hepatitis C, and autoimmune disease and, Dr. Leiden added, “early-stage research has an interesting molecule for genetic and specialty diseases.”