With new buyout, J&J jumps back in on a storied but troubled Holy Grail target
Over a decade ago, some pharma executives fell in love with a molecule called rapamycin and its genetic target, called mTOR. (Literally: “molecular target of rapamycin.”)
It was easy to see why. Originally identified in 1972 by an Indian-Canadian chemist, who hid it in ice cream tubs in his freezer and then smuggled it into the US after the company he worked for asked him to abandon the program, it was finally approved in 1999 as an immunosuppressant for organ transplant recipients.
But it showed a wide range of strange effects. Broadly speaking, it seemed to stop cells from dividing — a feature seen most notably in experiments showing mice, given the drug, lived about 12% longer.
It could also, it seemed, slow the growth of heart tissue on stents — an indication where it’s now used — and slow the growth of cancer cells. This was seen in rigorous animal models and, anecdotally, in the Indian-Canadian chemist himself, Surendra Nath Sehgal, who started taking rapamycin after being diagnosed with what doctors said was terminal colon cancer. He lived five years, only dying after he stopped.
So Merck, Novartis and others started developing new compounds to go after the same target. In the clinic, though, the effects were less pronounced. And the drugs could sometimes bring fatal side effects. In 2012, an FDA panel roundly rejected a Merck candidate in a rare sarcoma, saying the benefits didn’t outweigh the risks.
Although one Novartis mTOR-targeting drug, everolimus, was approved for several cancers, most drug companies moved on. Novartis itself offloaded its mTOR pipeline to PureTech in 2017.
Now, though, J&J is getting back in the game. The pharma announced on Wednesday it bought, for an undisclosed sum, a tiny biotech known as Anakuria Therapeutics.
Spun out of Navitor Pharmaceuticals, an mTOR-focused biotech that’s hung around quietly since its launch in 2009, Anakuria has a portfolio of rapamycin-like molecules. One, known as AT-20494, is ready for first-in-human trials, Navitor said in a statement.
J&J will test it in dominant polycystic kidney disease, a progressive genetic disorder where cysts grow to cover the kidneys, causing a range of symptoms.
Although researchers have advised against using rapamycin directly in such patients because of its extreme side effects, they’ve theorized some type of mTOR inhibitor could help slow cyst growth. Presumably, J&J sees the new molecule as safer and more selective.
“We are thrilled that the potential value and substantially differentiated profile of Anakuria’s mTORC1 inhibitor program can be explored with Janssen,” Navitor CEO Tom Hughes said in a statement.