Lorigerlimab

With no buyer, BioMarin pulls gene therapy from shelvesGSK inks $1B kidney-focused siRNA pactFDA approves Immedica's ultra-rare metabolic disease therapyPatient death triggers partial hold of MacroGenics' mid-stage cancer studyWith no buyer, BioMarin pulls gene therapy from shelvesHaving failed to find a buyer for its haemophilia A gene therapy, BioMarin Pharmaceutical said Monday that it will withdraw Roctavian (valoctocogene roxaparvovec-rvox) from the market. The company announced plans to divest the programme in October, after the therapy posted third-quarter sales of just $3 million. According to BioMarin, it "undertook a comprehensive effort to identify a potential buyer."In connection with its decision to pull Roctavian from shelves, BioMarin said it will take a $240 million charge, including a $119-million inventory write-off. -Elizabeth EatonGSK inks $1B kidney-focused siRNA pact GSK has agreed to shell out $40 million upfront for global rights to two siRNA programmes from China's Frontier Biotechnologies in a deal that could net the Chinese drugmaker up to $963 million in milestones, plus tiered royalties (see – Vital Signs: Charting China's course westward into 2026).In return, the UK drugmaker obtains exclusive worldwide rights to the two assets; one candidate is at the IND stage, while the second remains preclinical. Frontier will advance one programme through a Phase I study in China and complete IND-enabling work on the other before GSK assumes responsibility for global clinical development, regulatory filings and commercialisation."This agreement further strengthens our immunology pipeline, adding two potential first-in-class oligonucleotide therapies with significant opportunity to improve patient outcomes across multiple kidney diseases," said Kaivan Khavandi, global head for respiratory, immunology and inflammation R&D at GSK.The Frontier pact follows GSK's licensing deal with Empirico last October for rights to a Phase I siRNA programme, EMP-012, in chronic obstructive pulmonary disease. Empirico received $85 million upfront and is eligible for up to $660 million in milestones.-Anna BratulicFDA approves Immedica's ultra-rare metabolic disease therapyThe FDA on Monday awarded Immedica Pharma's arginine specific enzyme therapy, Loargys (pegzilarginase-nbln), with an accelerated approval to treat hyperargininaemia in patients 2 years and older with arginase 1 deficiency (ARG1-D) in conjunction with dietary protein restriction. The treatment is expected to be available in the US starting in April. The clearance was based on findings from the Phase III PEACE trial, in which Loargys significantly reduced plasma arginine from baseline compared with placebo at 24 weeks. According to Immedica, the therapy is the first and only treatment to address persistently elevated levels of plasma arginine, the disease's main driver. ARG1-D is an ultra-rare progressive metabolic disease that affects an estimated 250 people living in the US.-Elizabeth EatonPatient death triggers partial hold of MacroGenics' mid-stage cancer studyMacroGenics said Monday that the FDA has placed a partial clinical hold on the Phase II LINNET study of its experimental cancer drug lorigerlimab following a patient death, pausing patient enrolment. The company reported safety events that occurred across four patients, including two cases of grade 4 thrombocytopenia, one report of grade 4 myocarditis, and one patient with grade 4 neutropenia and concurrent septic shock, which resulted in their death. MacroGenics has dosed 41 patients with platinum-resistant ovarian cancer or clear cell gynaecologic cancer with lorigerlimab, a bispecific DART molecule that targets PD-1 and CTLA-4. Though enrolment is paused, study participants can continue to receive lorigerlimab."MacroGenics is fully committed to working closely with the FDA to resolve the partial clinical hold and we intend to resume study enrollment as soon as possible," said CEO Eric Risser.-Elizabeth Eaton