At some point, every biotech weighs whether to forge ahead alone or team up with a larger company that has the scientific know-how, clinical trial experience, or the cash to keep a program going. For early-stage developers of cell and gene therapies, these alliances are almost essential.
Bruce Levine been on both sides of this equation. The University of Pennsylvania professor has worked with companies seeking to advance their therapies through clinical development. But as an innovator, he’s also been in the position of looking for partners to continue his work. That’s the case with tisagenlecleucel (Kymriah), a cell therapy initially developed in Penn labs before going on to become the first FDA approved CAR-T treatment.
Speaking last week during Xconomy’s Xcelerating Life Sciences Philadelphia event, Levine said the university’s early research was supported by grants and philanthropic funding. While that research produced great results in a handful of patients, cell therapies are expensive to make, and the Penn scientists needed more cash.
Back in 2011 and 2012, there wasn’t a lot of venture capital support for startups researching cell therapies, Levine recalled. He hoped that publishing the data would spark support from a different source. He was right: The resulting media attention led to an assortment of suitors that ranged from small biotechs to large biopharmaceutical companies. Rather than build a startup around the cell therapy technology, the university decided to license it to one of the companies that came knocking: Novartis (NYSE: NVS).
“The startup might have been attractive, had there been funding,” Levine said. “Still, when you’re building something from the ground up, it takes a long time. At that point, nobody had global regulatory expertise or global clinical expertise.”
In 2012, Penn announced a licensing agreement with Novartis. The pharmaceutical giant had no experience manufacturing cell therapies, but Levine said that Penn brought to the alliance its cell therapy manufacturing expertise. Novartis brought cash and its experience running clinical trials and taking drugs through the regulatory process. Five years after the partnership began, the FDA approved Kymriah.
Levine has gone on to co-found Tmunity, a Philadelphia cancer cell therapy developer. Seed funding from Penn was followed by venture capital investment. The startup is led by CEO led by Usman Azam, a Novartis veteran. Levine says Tmunity represents an opportunity to do what the Penn team might have done with tisagenlecleucel back in 2012, had institutional financing been available.
Novartis isn’t the only big pharma company mining the sector for cell therapy innovation. GlaxoSmithKline (NYSE: GSK), which has a large presence in the Philadelphia area, has a cell therapy group of about 300 people split between the UK and the US. Delfi Krishna, director of GSK’s cell and gene therapy platform, said that a key consideration for any partnership with her company is finding technologies or assets that align with its areas of focus.
GSK established one such partnership last October with Lyell Immunopharma, a San Francisco startup run by Rick Klausner, former head of the National Cancer Institute. To date, T-cell therapies, including Kymriah, have been approved for blood-born cancers. The alliance with Lyell aims to use the startup’s technologies for improving T-cell function and cell “fitness” to develop cell therapies capable of treating solid tumors. The GSK-Lyell alliance includes the pharma giant’s lead cell therapy program, GSK337794, which is being evaluated in synovial sarcoma among other cancers. The partnership spans five years.
When considering a potential partnership, Krishna says GSK assesses the strength of preclinical and clinical data. GSK is also interested in how a potential partner’s assets match up to the pharma giant’s diseases of interest. Other considerations include the time it will take to develop the therapy and the investment that would be required.
“I would encourage people to think that GSK is absolutely open to innovative ideas in all areas of value chain—early, very early, late, and throughout,” Krishna said. “But it basically is a very thorough analysis, and it’s very data driven and science driven.”