uno Therapeutics is abandoning a cancer therapy that once looked like a revolutionary advance, reacting to a rash of patient deaths that made the customized treatment too risky to push forward.
The company’s decision changes the landscape in a blockbuster race to commercialize the first CAR-T technology, a promising new approach to cancer in which patients’ own immune cells are rewired to attack tumors. Juno, once a leader in the field, is now more than a year behind rivals Kite Pharma and Novartis.
Juno’s treatment, called JCAR015, was tied to five deaths in clinical trials on a rare form of leukemia, leading the Food and Drug Administration to twice suspend development in 2016. In each case, Juno’s therapy triggered a storm of immune activity that led to deadly brain swelling, a side effect the company struggled to explain.
Now, with its lead project sidelined, Juno is turning its focus to a second CAR-T treatment, JCAR017, which has shown promise in diffuse large B cell lymphoma, or DLBCL. In an early trial on 19 patients, Juno’s therapy had a positive effect on 42 percent of participants over three months, and the company plans to move JCAR017 into a larger study this year.
But the Seattle company will be playing from behind. In the coming weeks, Kite Pharma plans to file for FDA approval for its DLBCL treatment, and Novartis is on pace to do the same by the end of 2017. That means Juno, even in the best-case scenario, would enter a marketplace with two CAR-T therapies already in use.
DLBCL is the most common form of non-Hodgkin lymphoma, and CAR-T therapies, which would treat patients whose cancer endured standard treatment, present a blockbuster opportunity. Kite’s DLBCL treatment is in line for peak sales of $1.5 billion a year, according to Cowen analyst Eric Schmidt.
Such a competitive landscape creates a high-wire act for Juno, whose recent struggles have eroded investor confidence. If JCAR017 runs into serious safety problems or fails to measure up to its counterparts at Kite and Novartis, Juno could find itself shut out of a multibillion-dollar market.
Juno’s gradual decline comes in stark contrast with its glitzy beginnings.
The company made waves in biotech when it emerged in 2013 with a then-record $120 million in venture financing, brain power from leading cancer institutions, and promising data suggesting CAR-T could forever change the treatment of some particularly dire cancers. For years, Juno looked poised to win the first-ever approval in CAR-T.
But as the company has ceded ground to its rivals, its valuation has slipped. Juno’s share value is now just above its initial public offering price, roughly 65 percent down from its all-time high in 2015.