Washington Editor

PTC Therapeutics Inc. received a $20 million financial boost from Celgene Corp. in exchange for exclusive development rights to orally bioavailable small molecules for two undisclosed oncology targets.

As part of the agreement, PTC is granting Celgene access to Gene Expression Modulation by Small Molecules (GEMS), a screening technology used to identify small molecules that modulate post-transcriptional control mechanisms - the sequence of events in the cell that ultimately regulates the rate and timing of protein production.

Summit, N.J.-based Celgene is the fourth company to gain access to PTC's GEMS technology, which has been used in drug discovery programs in oncology, infectious diseases, cardiovascular diseases and neuromuscular disorders. The other firms are Pfizer Inc., CV Therapeutics Inc. and Schering-Plough Corp.

If Celgene exercises its research option under the agreement, PTC may receive funding for research efforts and earn milestone payments per target for achieving certain discovery, development, regulatory and commercial objectives.

Celgene would receive exclusive worldwide rights and pay PTC royalties on worldwide sales of any products resulting from the collaboration. No other details of the deal were disclosed.

PTC CEO Stuart Peltz told BioWorld Today that his firm plans to use the $20 million equity investment to advance its development and clinical trial programs.

The nearly 10-year-old South Plainfield, N.J.-based firm has raised $132 million in equity, Peltz said.

The company has successfully completed Phase I trials of its GEMS-based product PTC299, an orally administered small molecule designed to inhibit the production of vascular endothelial growth factor in tumors, and is set to soon start enrolling participants in a Phase Ib/IIa study.

The Phase Ib/IIa trial, which is being funded in part by the Department of Defense and conducted in the U.S., will test PTC299 in 38 patients with advanced breast cancer, Peltz said.

PTC also plans to test the compound in other solid tumor types starting early next year, he added.

PTC299 also may prove clinically useful in other diseases in which VEGF levels play a key role, such as in age-related macular degeneration, according to PTC.

GEMS is an "exciting" technology, Peltz said, because it can be used to inhibit or enhance protein expression.

"It opens up a door against a number of targets," he said.

The fact that four firms have sought collaborations to gain access to the GEMS technology for use in multiple therapeutic areas, Peltz said, is a "testament to the breadth of the technology."

PTC also continues to advance its genetic disorder product PTC124, he noted. The product is being developed to treat Duchenne muscular dystrophy (DMD) and cystic fibrosis due to nonsense mutations and has been granted fast-track and orphan-drug status by the FDA for both indications.

Interim data reported in May from a Phase II trial of patients with DMD due to a nonsense mutation showed that treatment with PTC124 was associated with increases in muscle dystrophin expression and reductions in serum creatinine kinase values in at least 50 percent of evaluable patients.

Results of two Phase II trials reported in November 2006 showed that PTC124 can restore function of the cystic fibrosis transmembrane conductance regulator protein in airway cells and significantly reduce blood neutrophil counts.

PTC plans to soon start pivotal studies of PTC124 in CF and DMD, Peltz noted.

Shares of Celgene (NASDAQ:CELG) closed at $69.01 Thursday, down 28 cents.