SHR-1812

Y-mAbs Therapeutics is looking to capitalize on a second chance at winning approval for its potential treatment of pediatric neuroblastoma with CNS/cerebrospinal fluid cancer as the FDA has set up an adcomm meeting of outside experts for Oct. 28 to review the application. First developed by Memorial Sloan Kettering Cancer Center and licensed to Y-mAbs, omburtamab is one of the company’s two lead compounds focused on neuroblastoma, an area with high unmet need. But Y-mAbs, which saw its CEO leave abruptly in April, only recently re-filed its BLA for the anti-B7-H3 antibody after receiving a refuse-to-file letter from the FDA in 2020. The company at the time cited the need for more manufacturing and clinical data, noting that it provided supplementary data from a Phase II study regarding tumor response from the first 24 patients in the trial. Y-mAbs then resubmitted its BLA on March 31, and the FDA now has until November 30 to complete its priority review. The adcomm discussion in October is likely to go over clinical and safety data, as MSKCC’s Kim Kramer in 2020 presented planned interim results for 17 patients enrolled in the company’s pivotal multicenter study. The study showed a twelve-month OS of 87%, with a median follow-up of 26 weeks. This compares to an OS of about 30% in a historic control group previously disclosed by Y-mAbs. An approval in this pediatric setting would also come with a priority review voucher (PRV), which Y-mAbs can sell, as it did (for $105 million) after its first approval for the GD2 antibody Danyelza. Cowen analyst Joseph Thome said in a note to investors in April: Y-mAbs’ Danyelza brought in $32.9 million in its first year, and the biotech is expecting between $45 million and $50 million this year. Pediatric cancer has been a central focus for Y-mAbs founder Thomas Gad, whose daughter was diagnosed with high-risk neuroblastoma at age 2. She was treated with a version of Danyelza at the time, but later relapsed and began taking omburtamab about 13 years ago.