PTC Therapeutics gears up gene therapy capacity in lease deal with Bristol-Myers

PTC Therapeutics is aiming for its first gene therapy launch and amping up its pipeline, and it needed more manufacturing capacity to make it all happen. It found that—and lab space, too—in a facility Bristol-Myers Squibb was looking to unload.

PTC inked a long-term lease (pdf) with Bristol-Meyers, gaining about 185,000 square feet of space that includes a state-of-the-art biologics production facility as well as supporting research and operations buildings.

The lease comes as PTC nears FDA filings for two new products: risdiplam, a small molecule treatment for spinal muscular atrophy that will compete with Biogen's Spinraza, and its first gene therapy, which targets AADC deficiency, a rare enzyme disorder.

Part of BMS’s Hopewell, New Jersey campus, the new site will allow PTC to support its gene therapy production, the South Plainfield, New Jersey-based company said. PTC plans to "further develop" the existing biologics set-up to produce gene therapies, CEO Stuart Peltz said during the company's second quarter earnings call. It will also move its research operations to a newly renovated building on the Hopewell campus.

"The existing GMP suites on this site were recently renovated and will be used to produce material to serve multiple preclinical and clinical gene therapy programs," Peltz said, adding that the new site will complement the company's existing preclinical production facility in Bridgewater, New Jersey.

“Obtaining this facility allows PTC to execute on our plans of rapidly advancing our gene therapy pipeline,” Peltz said in a statement about the lease.

Its AADC gene therapy manufacturing is one key piece of the FDA application for approval, PTC execs said during the call. The company is working toward releasing a commercial batch now, COO Marcio Souza said, based on agreements with the agency.

"[T]he key thing is on getting manufacturing completed and done," Peltz said in discussing the BLA submission, going on to say, "[O]ur process thus far in the manufacturing is moving along, and we anticipate the submission with the completion of the manufacturing this year."

In addition to the two upcoming launches, PTC markets Emflaza, a Duchenne Muscular Dystrophy treatment, in the U.S. It sells another DMD drug, Translarna, in Europe and Tegsedi, which treats polyneuropathy of hereditary transthyretin-mediated amyloidosis, in Latin America.