BridgeBio keeps FDA approval spree rolling, this time with nod for targeted bile duct cancer med

BridgeBio is keeping the momentum going in 2021. After winning a nod for its rare disease med Nulibry earlier this year, the FDA has handed the company an approval for its targeted cancer treatment.

The FDA blessed BridgeBio’s Truseltiq, previously known as infigratinib, with an accelerated approval in previously-treated, locally advanced or metastatic bile duct cancer with an FGFR2 fusion or rearrangement. The green light, won by BridgeBio's affiliate QED Therapeutics, marks the “hub and spoke” biotech’s first cancer drug approval and its second FDA approval ever after the regulator cleared its ultra-rare disease med Nulibry in late February.

Cholangiocarcinoma (CCA), a cancer of the bile ducts in the liver, affects some 20,000 people in the U.S. and European Union a year, and FGFR2 genetic aberrations are present in around 15% to 20% of those patients, BridgeBio said in a release. Currently, the median five-year survival rate is just 9%.

Regulators approved Truseltiq on results from a single-arm phase 2 study of 108 patients who’d tried at least one prior therapy for their advanced CCA. Patients received 125 mg of Trulsetiq daily for 21 days over 28-day cycles until toxicity or disease progression became too advanced.

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The trial yielded an overall response rate of 23%, with the median response lasting for five months, BridgeBio said.

Many CCA patients undergo surgery to remove their cancer, but in cases where that isn’t an option, there aren't many other treatment options. Still, Tuseltriq will face off with at least one rival in Incyte’s Pemazyre, which was approved last April to treat bile duct cancers with an FGFR2 fusion or other rearrangement.

Pemazyre rolled out with a list price of $17,000 per treatment cycle. With an average treatment duration of around six months and eight or nine cycles, the cost per patient lands somewhere between $136,000 and $153,000. The drug generated (PDF) $25.9 million in 2020 sales, falling short of the $50 million sales estimate RBC Capital Markets analysts predicted last year.

For its part, Truseltiq’s lower price tag could give the drug a cost edge over Pemazyre, especially in the first month of treatment, analysts at Mizuho Securities wrote to clients last week. Armed with an approval, BridgeBio aims to launch Truseltiq in about two weeks at a price of $21,500 per month, according to the analysts. The drug would cost around $64,500 every three months, marking a roughly 5% lower price than Pemazyre over the same period, the team said.

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The team predicts single-digit millions in Truseltiq sales this year, tempered by the fact that it’s second to market after Pemazyre and will essentially only capture the last two quarters of the year.

BridgeBio and Helsinn Therapeutics are on the hook to commercialize the drug in the U.S. and will split profits and losses equally, BridgeBio said. Helsinn Group, for its part, has exclusive marketing rights outside of the U.S., save for China, Hong Kong and Macau, where LianBio will tackle development and commercialization. Under the deal with Helsinn, BridgeBio is in line to receive tiered royalties and payments totaling up to $2.45 billion.

BridgeBio licensed the drug from Novartis back in early 2018, with plans to continue development in bile cancers and dwarfism. It spun out QED Therapeutics with $65 million in seed funding to tackle that work. The company didn't disclose the specifics of the deal but noted that the $65 million included a “substantial upfront payment” to Novartis.