NEW YORK – Somerville, Massachusetts-based Bluebird Bio this week said it has entered into an advance agreement to sell a rare pediatric disease priority review voucher, if granted in connection with US regulatory approval of lovotibeglogene autotemcel (lovo-cel). The US Food and Drug Administration is currently deciding whether to grant the gene therapy market authorization as a treatment for patients with sickle cell disease ages 12 and older. Rare pediatric disease priority review vouchers are awarded as part of an FDA program to encourage sponsors to develop therapies for rare pediatric diseases. Vouchers can be redeemed by the awardee to receive priority review of a marketing application for a future product, sold, or transferred. The potential voucher, which Bluebird Bio would be eligible to receive if lovo-cel is approved for patients under the age of 18, would be sold for $103 million under the terms of the deal.
ArriVent Biopharma said this week that the US Food and Drug Administration has granted breakthrough therapy designation to furmonertinib as first-line treatment for advanced non-squamous non-small cell lung cancer (NSCLC) patients whose tumors harbor EGFR exon 20 insertion mutations. The designation is meant to expedite development and review for particularly promising drugs in difficult-to-treat diseases. ArriVent is studying furmonertinib for EGFR-mutated NSCLC in its ongoing Phase III FURVENT clinical trial and the Phase I FAVOUR trial.
Apollomics this week announced two new cohorts in its ongoing Phase II SPARTA study evaluating its c-MET inhibitor vebreltinib in patients with solid tumors harboring MET dysregulations. One cohort will explore vebreltinib plus an EGFR inhibitor in patients with EGFR-mutated non-small cell lung cancer that progressed on first-line EGFR inhibitor therapy due to acquired resistance through c-MET amplification. The second cohort will evaluate vebreltinib monotherapy in histology-agnostic solid tumors harboring wild-type MET gene and high expression of c-MET and hepatocyte growth factor (HGF).
Atara Biotherapeutics this week said it expanded a partnership with Pierre Fabre Laboratories to include the US and other remaining global commercial markets for its T-cell immunotherapy Ebvallo (tabelecleucel). Previously, Pierre Fabre secured rights to commercialize, distribute, and engage in regulatory activities for the product in Europe, the Middle East, Africa, and certain other markets. Under the terms of the expanded partnership, Pierre Fabre will pay Atara $30 million upfront and up to $640 million and double-digit tiered royalties on net sales. It will also reimburse Atara for anticipated global development costs for Ebvallo. Atara expects to submit a biologics license application for Ebvallo in post-transplant lymphoproliferative disease in Q2 2024. Concurrent with the partnership, Atara is restructuring, including a 30 percent reduction in workforce, to enable executions of its obligations under the Ebvallo partnership and to focus resources on advancing other programs. The restructuring is expected to fund Atara's operations into Q3 2025.
Bayer and the Broad Institute said this week that they have added five years to an ongoing research collaboration focused on developing treatments for new cancer targets. The precision oncology collaboration, which began in 2013, led to an EGFR/HER2 inhibitor that Bayer is currently evaluating in Phase I clinical trials for cancers harboring EGFR exon 20 insertion mutations.
In Brief This Week is a selection of news items that may be of interest to our readers but had not previously appeared in Precision Medicine Online.