Beam Therapeutics also announced that it dosed the first patient in its Phase I/II sickle cell disease trial; Vyne stepped into the increasingly competitive vitiligo space, while Tracon moved further into sarcomas in its checkpoint inhibitor program, and Telix started a small trial of its drug in brain cancer.

At nearly $194,000, the wholesale acquisition cost for Tzield came in above analysts’ $100,000-$115,000 estimates, but payer pushback is not expected as the drug faces no competition.

Data showed efficacy limited to IVS26-mutant Leber congenital amaurosis 10, which affects only 300 patients in the US. The company is pausing enrollment as it seeks a partner.

The company plans to meet with the FDA before the end of the year to discuss the way forward, including a pivotal study it hopes to launch in 2023 in uncontrolled and resistant hypertension.

One analyst said efficacy data for roflumilast in INTEGUMENT-1 suggest limited competitiveness against Incyte’s topical JAK inhibitor, Opzelura, while others were more optimistic.

Industry experts at BioFuture 2022 highlighted the growing importance of digital technology in drug discovery and development, and also the limitations remaining.