Partners Intrexon ($XON) and Ziopharm ($ZIOP) are cutting in on the fervor over a new class of immunotherapies for cancer, striking a deal with the University of Texas' MD Anderson Cancer Center.
Under the deal, the pair will give MD Anderson $100 million for a CAR-T technology, made through a process in which scientists remove T cells from a patient's blood and equip them with targeting mechanisms called chimeric antigen receptors (CARs), which seek out and bind to proteins expressed by cancer cells. The resulting cells are reinjected into the patient, at which point they track down malignancies and attack them as they would any commonplace infection.
MD Anderson's approach, dubbed Sleeping Beauty, uses a nonviral gene transfer system to get those modified T cells to their targets, in contrast to leading CAR-T therapies from Novartis ($NVS), Juno Therapeutics ($JUNO), Kite Pharma ($KITE) and others. That presents some safety advantages, researchers say, as MD Anderson's approach has thus far avoided some of the dangerous immune responses that have afflicted rival CAR-T programs.
Through the deal, Intrexon and Ziopharma expect to get 5 CAR-T candidates into the clinic this year, and the companies are pursuing both patient-derived immunotherapies and off-the-shelf treatments based on donor T cells.
|MD Anderson President Ron DePinho|
"Genetically engineering our patients' immune-system T cells to efficiently attack and destroy cancer cells represents one of the most exciting approaches with curative potential in oncology today," MD Anderson President Ron DePinho said in a statement. "We believe coupling MD Anderson's unique CAR-T cell approach with the powerful technologies of Ziopharm and Intrexon will allow us to build T cells that hit cancer harder, with greater precision, under tighter control and with potentially fewer side effects for patients."
CAR-T therapies have generated stellar results in early clinical trials, galvanizing widespread interest around the industry. Accordingly, Ziopharm's shares shot up more than 50% on the announcement of the MD Anderson deal, while Intrexon rose about 14%. The news is particularly promising for Ziopharm, which is still on the mend after the costly Phase III failure of its cancer treatment palifosfamide back in 2013.
Meanwhile, leading the way in CAR-T is Novartis ($NVS), which is working alongside the University of Pennsylvania. The fast-crowding space also includes Kite, which grossed more than $140 million in a June IPO on the strength of its approach to T cell modification, and Juno, which pulled of 2014's biggest biotech IPO thanks to the promise of its clinical candidates. Oncology heavyweight Celgene ($CELG) has jumped into the field with gene therapy pioneer bluebird bio ($BLUE), while latecomers Johnson & Johnson ($JNJ) and Pfizer ($PFE) have shouldered into the space with with high-dollar deals of their own, and Bellicum Pharmaceuticals ($BLCM) is wending toward the clinic with a novel approach to the field.
- read the announcement