Less than a month after the U.S. FDA’s Peripheral and Central Nervous System Drugs Advisory Committee handed down a unanimous vote in favor of Eli Lilly and Co.’s Alzheimer’s disease candidate (AD), donanemab, the agency approved the drug as a once-monthly injection for adults with early symptomatic disease. Branded Kisunla, the beta-amyloid antagonist marks the second approved AD drug that has demonstrated in clinical trials an ability to slow cognitive decline, going up against Leqembi (lecanemab) from Biogen Inc. and Eisai Co. Ltd., which won full approval in July 2023, only six months after nabbing an accelerated nod.
At a time when much of the biopharma space can’t seem to get enough of antibody-drug conjugates (ADC), Bristol Myers Squibb Co. is backing away from a potential $3 billion-plus collaboration with Eisai Co. Ltd., handing back full rights to the latter’s folate receptor alpha-targeting ADC, farletuzumab ecteribulin (FZEC), citing a portfolio reprioritization.
At a time when much of the biopharma space can’t seem to get enough of antibody-drug conjugates (ADC), Bristol Myers Squibb Co. is backing away from a potential $3 billion-plus collaboration with Eisai Co. Ltd., handing back full rights to the latter’s folate receptor alpha-targeting ADC, farletuzumab ecteribulin (FZEC), citing a portfolio reprioritization. Global rights to FZEC now reside with Tokyo-based Eisai, which said it intends to accelerate work on the candidate, which is in three clinical studies, including two phase II trials in gynecological cancers and non-small-cell lung cancer.
Two days before the PDUFA date, the U.S. FDA handed down a complete response letter (CRL) for Rocket Pharmaceuticals Inc.’s Kresladi (marnetegragene autotemcel), delaying potential approval of the lentiviral-based gene therapy as the first therapeutic option for leukocyte adhesion deficiency type I, a rare, inherited immune disorder. But the Cranbury, N.J.-based company has suggested that delay won’t be long, as the CRL requests only “limited” chemistry manufacturing and controls (CMC) information – additional CMC data were also cited as the reason for the three-month review extension earlier this year.
Any skepticism that might have been lingering in the wake of Alnylam Pharmaceuticals Inc.’s February decision to tweak the analysis plan for the Helios-B trial testing vutrisiran in transthyretin amyloidosis with cardiomyopathy appeared to dissipate as the company reported top-line data showing the study met the primary and all secondary endpoints.
Sarepta Therapeutics Inc. CEO Douglas Ingram said he expects “ferocious” demand for gene therapy Elevidys (delandistrogene moxeparvovec), granted full approval by the U.S. FDA for Duchenne muscular dystrophy (DMD). Shares of the Cambridge, Mass.-based firm closed June 21 at $16.72, up $37.22, or about 30% on the news.
Coming off a presentation at the European Alliance of Associations for Rheumatology meeting, where a single report of disease recurrence in a lupus patient overshadowed promising early data for Kyverna Therapeutics Inc.’s autoimmune candidate, KYV-101, the Emeryville, Calif.-based company rallied on news of a case report describing the first use of the CD19-targeting CAR T-cell therapy in a patient with stiff-person syndrome, a rare, progressive neurological autoimmune disorder for which there is limited treatment.
Aerovate Therapeutics Inc. is shutting down the ongoing phase III portion of its Impahct study of AV-101, an inhaled version of PDGFR inhibitor imatinib, after reporting top-line data from the phase IIb portion failed to meet primary and secondary endpoints in patients with pulmonary arterial hypertension (PAH). It was a major blow for the single-product company, sending shares (NASDAQ:AVTE) falling 93% to close June 17 at $1.65, down $22.97.
Shares of Avidity Biosciences Inc. hit a 52-week high on reports of promising data from the first efficacy cohort of its phase I/II Fortitude study testing delpacibart braxlosiran (del-brax) in facioscapulohumeral muscular dystrophy (FSHD), including impressive biomarker results that could indicate a path for potential accelerated approval.
Ipsen SA picked up U.S. FDA accelerated approval for its Genfit SA-licensed elafibranor, making it the first new drug in eight years for treating primary biliary cholangitis, though a potential competitor lurks just around the corner.
