Cancer drug to treat dwarfism? Abandoned Novartis asset snagged by BridgeBio’s QED Therapeutics

BridgeBio is snatching a recently-abandoned cancer program off Novartis’ shelves, handing the asset to a spinout company with unusual plans for the drug.

The newly-licensed product — called infigratinib or BGJ398 — was in Phase II trials at Novartis for patients with bile duct cancer with FGFR fusions. Although the drug showed meaningful clinical activity early in the trial, Novartis decided to dump the drug last year.

Michael Henderson

“They were originally developing the FGFR inhibitor for a much broader spectrum of oncology,” said Michael Henderson, the guy in charge of finding new assets for BridgeBio. While two indications — bile duct cancer (cholangiocarcinoma) and urothelial carcinoma — saw an interesting response from the drug, it wasn’t enough to drive Novartis forward.

It’s enough for BridgeBio, which is spinning out QED Therapeutics to develop the drug with a gift of $65 million in seed money. A chunk of that cash will go straight back out to Novartis to pay for licensing fees, although details of the deal weren’t disclosed.

But weirdly, infigratinib was originally on BridgeBio’s radar for other reasons, which will play into where the drug goes from here.

Henderson, who co-founded PellePharm while still in medical school at the ripe age of 22, now works at BridgeBio scouting for promising assets. At 28, he tells me he spends his time scraping PubMed, reading abstracts, and sometimes pouring over Big Pharma’s financial statements looking for something worthwhile.

Just months ago, Henderson became intrigued with a paper about a form of genetic dwarfism called achondroplasia. The condition can cause brainstem compression and other complications as the bones fail to develop. In the paper that caught Henderson’s eye, researchers showed in mouse models that low doses of Novartis’ cancer drug infigratinib — which inhibits FGFR — could treat achondroplasia at its source by directly treating the mutation in FGFR3. In theory, this could mean big things for some patients suffering from the genetic disease. A correction at the source could help bones to grow more normally, perhaps decreasing serious complications of achondroplasia, such as cranial impingement, which can require surgery later in life. It might also decrease spinal stenosis, obstructive sleep apnea and ear infections.

Neil Kumar

“We’re really interested in the notion that targeted cancer therapies can be used to address other genetic conditions,” said BridgeBio’s CEO Neil Kumar.

Kumar tells me people are often nervous “and rightfully so” to use kinase inhibitors in pediatric conditions because they tend to be toxic. But Kumar thinks low doses may have “profound effects.”

So Henderson decides BridgeBio should try to snag the infigratinib program, and discovers (about 250 pages deep in a 500-page 10-K) that Novartis is already looking for buyers. In fact, Novartis already had term sheets from other companies hoping to snatch the drug.

“We sprinted to get [the bid] together within a month, assembled the right team, and within two months we were moving forward,” Henderson said.

BridgeBio made an appealing case: they would take over the Phase II cancer program, pursue trials in cholangiocarcinoma and urothelial carcinoma (good data permitting), and tack on some preclinical work in the brand new indication of achondroplasia.

The more patients the drug can reach, the better. So Novartis took them up on the deal.

QED is on the hunt for a CEO, and is being led in the interim by its R&D leaders and CMO Daniel Hoth, the former chief of the Investigational Drug Branch of the National Cancer Institute (NCI).

“We are committed to moving this compound forward in late-stage development and further proving the strong efficacy in cancer that has already been demonstrated across multiple trials,” Hoth said.

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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