Zenocutuzumab

The FDA has extended the Prescription Drug User Fee Act (PDUFA) goal date for Merus N.V.’s zenocutuzumab (Zeno) Biologics License Application (BLA), pushing the decision to February 4, 2025. The delay will allow the FDA additional time to assess recent information submitted by Merus regarding chemistry, manufacturing, and controls (CMC) for Zeno, a treatment under priority review targeting NRG1+ cancers. The FDA did not request any additional clinical data concerning this extension. Merus, headquartered in Utrecht, Netherlands, and Cambridge, Massachusetts, specializes in developing bispecific and trispecific antibody therapeutics, known as Multiclonics. These therapies, engineered to perform like traditional human monoclonal antibodies, have shown promise for durable therapeutic effects with minimal immunogenicity.

Taylor Tieden for BioSpace The FDA is set to decide on four promising therapies in the next two weeks, including a CAR T for acute lymphoblastic leukemia. In the coming weeks, the FDA has four big decisions lined up. Among these are verdicts for a gene therapy for a rare disease and another for a lung cancer biologic therapy under Priority Review. Read below for more. Journey’s Rosacea Drug Awaits FDA Verdict By November 4, the FDA will release its decision on Journey Medical Corporation’s DFD-29 (minocycline hydrochloride modified release capsules), which the company is proposing as an oral treatment for inflammatory lesions and erythema in adults with rosacea. Journey is backing its New Drug Application (NDA) with data from two Phase III studies, both of which hit all co-primary and secondary endpoints . Compared with the current standard of care—40-mg Oracea (doxycycline) capsules—DFD-29 showed significant superiority in treatment success, as per the Investigator’s Global Assessment. DFD-29 was likewise better than Oracea at reducing total inflammatory lesion count and erythema in rosacea patients. Both trials also found that DFD-29 significantly outperformed placebo. If approved, DFD-29 would be “the only oral medication to address both inflammatory lesions and erythema” in rosacea “and will be a preferred treatment option by physicians and their patients,” Journey president and CEO Claude Maraoui said in a company statement alongside the FDA’s acceptance of its NDA. Rosacea is an inflammatory dermatologic condition that affects around 16 million people in the U.S. The disease manifests as red, pus-filled bumps on the skin, alongside redness in the face. Merus Seeks Approval for Bispecific Antibody in NSCLC, Pancreatic Cancer Netherlands-based Merus is advancing the investigational bispecific antibody zenocutuzumab for the treatment of non-small cell lung cancer (NSCLC) and pancreatic ductal adenocarcinoma (PDAC) patients positive for fusions of the NRG1 gene. The FDA accepted Merus’ Biologics License Application (BLA) on May 6, 2024, and granted it Priority Review, which shortens the usual review period from 10 months to six months. In keeping with this schedule, the regulator is expected to release its decision no later than November 6. The NRG1 gene encodes for the HER3 ligand neuregulin. Though rare, fusion mutations between NRG1 and partner genes result in cancer-causing genomic events that are typically observed in certain malignancies, such as NSCLC and PDAC. Zenocutuzumab utilizes Merus’ proprietary Dock & Block technology to bind to HER2 and disrupt the interaction between the HER3 receptor and neuregulin or NRG1-fusion proteins. Preclinical studies have confirmed that zenocutuzumab effectively blocks the formation of HER2/HER3 heterodimers, in turn preventing cancer cell proliferation and survival. Merus’ data package for zenocutuzumab includes results from the Phase I/II eNRGy study, an October 2023 readout from which demonstrated a 37% objective response rate in NSCLC patients positive for NRG1 fusions. In PDAC patients carrying the same biomarker status, zenocutuzumab elicited a 42% objective response rate. Will FDA Follow Europe in Approving PTC’s Gene Therapy for AADC Deficiency? PTC Therapeutics is seeking U.S. approval for its gene therapy Upstaza (eladocagene exuparvovec) for the treatment of the ultrarare disease aromatic L-amino acid decarboxylase (AADC) deficiency in pediatric patients aged 18 months and up. The FDA’s decision is due on November 13. AADC deficiency is a genetic disorder caused by a mutation in the DDC gene, which leads to insufficient levels of the AADC protein. In turn, AADC deficiency is characterized by compromised production of dopamine, serotonin and other neurotransmitters, resulting in its key symptoms of developmental delays, muscle stiffness, movement problems and lethargy. Most patients with AADC deficiency need lifelong care, and the disease can be fatal. AADC deficiency affects between 1 in 64,000 and 1 in 90,000 births in the U.S. Upstaza targets the underlying cause of AADC deficiency by delivering a functional copy of the DDC gene. The gene therapy is delivered straight into the brain through a minimally invasive procedure and is meant to be a one-time treatment. In July 2022, the European Commission granted the gene therapy is Marketing Authorization, allowing its use in all 27 European Union member states, plus Iceland, Norway and Liechtenstein. The U.K.’s Medicines and Healthcare Products Regulatory Agency followed suit in November 2022. In March 2023, England’s National Institute for Health and Care Excellence recommended the use of Upstaza for the treatment of AADC deficiency, ensuring that the National Health Service will make the gene therapy available for patients who need it. FDA to Decide on Autolus’ CAR T Therapy for Acute Lymphoblastic Leukemia By November 16, the FDA will release its verdict for Autolus Therapeutics’ investigational CAR T therapy obecabtagene autoleucel (obe-cel) for the treatment for adult patients with relapsed or refractory B cell acute lymphoblastic leukemia (ALL). The London-based biotech is backing its BLA, which the FDA accepted in January 2024, with data from the pivotal Phase II FELIX study. An interim readout in December 2022 showed that the next-generation CAR T therapy could induce a 70% overall remission rate, alongside promising expansion and initial persistence at a median follow-up of 6.4 months. Obe-cel was also well-tolerated overall, with 3% of patients developing grade 3 or higher cytokine release syndrome, and 8% experiencing grade 3 or higher immune effector cell-associated neurotoxicity syndrome. If approved, obe-cel will join the limited roster of approved CAR T therapies alongside others such as Bristol Myers Squibb’s Abecma and Breyanzi and Gilead’s Tecartus and Yescarta. Obe-cel previously won the FDA’s Orphan Drug and Regenerative Medicine Advanced Therapy designations.