Mosunetuzumab

Today, a brief rundown of news involving Ultragenyx and Bristol Myers Squibb, as well as updates from Roche, Longeveron and Rocket Pharmaceuticals that you may have missed.The Food and Drug Administration has approved the first gene therapy for the rare progressive neurological condition Sanfilippo syndrome Type A. Called Fayuvi and priced at $3.95 million, the treatment is now the second gene therapy Ultragenyx Pharmaceuticals has brought to market since August. Neither of those therapies are projected by analysts to become blockbuster products. But Ultragenyx obtained sellable drug review vouchers alongside both approvals, which should significantly bolster the companys balance sheet, wrote William Blair analyst Sami Corwin. Ultragenyx warned of coming cost cuts following the failure of an Angelman syndrome drug with bigger sales potential. Company shares climbed by double digits following Fayuvis approval. Ben FidlerBristol Myers Squibb has stopped development of an experimental cancer drug licensed from startup Orum Therapeutics three years ago. Bristol Myers paid Orum $100 million up front in 2023 for rights to the therapy, which is known as ORM-6151, involves a combination of antibody-drug conjugate and protein degradation technologies, and was being tested in acute myeloid leukemia. In a regulatory filing, though, Orum said that, after reviewing the data from a Phase 1 trial, it's shelving the drug along with obligations to make additional milestone payments. Orum had been eligible to receive as much as $80 million in conditional payouts in the deal. Ben FidlerRoche's double-barreled blood cancer drug Lunsumio has succeeded in a late-stage trial in follicular lymphoma. Roche didn't provide specifics, but said that a regimen involving Lunsumio and a standard therapy meaningfully improved progression-free survival compared to Rituxan and typical treatment in people who'd received at least one prior medication. Survival data were “immature“ upon an interim data check, Roche said. Lunsumio is currently approved in the third-line setting, but the latest Phase 3 study could convert that clearance to a standard nod and expand use. Roche intends to submit the findings to regulatory authorities. Ben FidlerLongeveron will begin a strategic review after its experimental cell therapy for heart disease failed a Phase 2 trial. After 12 weeks of follow-up, patients receiving that therapy, laromestrocel, didnt have a statistically significant change in a measure of the hearts ability to pump out blood. Longeveron plans to meet with the FDA to discuss potential steps forward, but in the meantime, the company will hire an investment bank to review all options and intends to cut costs. Ben FidlerRocket Pharmaceuticals has come to terms with U.S. regulatorson modifications to a pivotal study testing a treatment for the rare heart condition Danon disease. Rocket previously paused that trial following the death of a study participant and then resumed testing with an altered protocol that, so far, has proven safe in three other enrollees. On Tuesday, Rocket said the FDA endorsed that safety update as well as its path to completing the trial. The company will recruit 9 additional patients and hopes to complete dosing in the middle of 2027. Rocket will provide an update on the program during a virtual investor webinar in early October. Delilah Alvarado '

Roche’s T-cell engager portfolio has notched another milestone, as a phase 3 trial of the company’s Lunsumio met its primary endpoint in earlier-line follicular lymphoma. Without sharing any specific data, Roche and its subsidiary Genentech said Lunsumio’s combination with lenalidomide (Revlimid) demonstrated a statistically and clinically meaningful improvement in progression-free survival against rituximab (Rituxan) plus lenalidomide, known as R2, in the phase 3 Celestimo study. The trial enrolled follicular lymphoma (FL) patients who have received at least one prior line of treatment, and it serves as the confirmatory study required to convert Lunsumio’s existing accelerated approval as a monotherapy in third-line FL into a full approval, while also potentially moving the drug earlier into the second-line setting. Besides the positive PFS readout, Roche said overall survival data were immature but did not offer any further description. No new safety signals were identified beyond the known profile of each drug, the company added. Detailed data could help doctors and industry watchers understand the Lunsumio doublet’s position against Incyte’s Monjuvi and AbbVie and Genmab’s Epkinly , both of which won FDA approvals for second-line FL last year in three-drug regimens with R2. An in-class competitor to Lunsumio, Epkinly, used on top of R2, reduced second-line-plus patients’ risk of disease progression or death by 79% versus R2 alone, the phase 3 Epcore FL-1 trial showed. For its part, the addition of Monjuvi to R2 improved PFS 57% among second-line-plus patients in the phase 3 inMind trial. Both Lunsumio and Epkinly are CD20xCD3 T-cell engagers, while Monjuvi is an anti-CD19 antibody. The new Celestimo results “indicate that a two-drug outpatient Lunsumio regimen may offer these patients an effective new option earlier in their treatment journey,” Levi Garraway, M.D., Ph.D., Roche’s chief medical officer and head of global product development, said in a statement. In addition to the three antibody drugs, Gilead Sciences’ CD19 CAR-T, Yescarta, is being tested in the phase 3 Zuma-22 trial in second-line-plus FL. At the same time, BeOne Medicines is combining its small-molecule BTK inhibitor Brukinsa with Roche’s CD20 antibody Gazyva in the phase 3 Mahogany trial in previously treated FL and marginal zone lymphoma. Roche is targeting its CD20xCD3 portfolio across B-cell malignancies. The Swiss pharma’s combination of Lunsumio and its antibody-drug conjugate Polivy is awaiting an FDA decision in second-line diffuse-large B-cell lymphoma (DLBCL) by Feb. 9, 2027. Meanwhile, all eyes are on an upcoming readout from Roche’s Skyglo trial for the company’s other CD20xCD3 bispecific, Columvi, in first-line DLBCL. Lunsumio was also recently moved outside blood cancers and into a phase 1 systemic lupus erythematosus trial.