AUCATZYL Approved for R/R B-ALL; FDA Accepts NDA for Unicycive’s Oxylanthanum Carbonate; AstraZeneca and Amgen Report Positive Results in Chronic Rhinosinusitis; Nipocalimab Granted Breakthrough Designation for Sjögren’s Disease; AbbVie’s Schizophrenia Drug Fails Phase Studies

FDA Approves Autolus’s AUCATZYL for Relapsed/Refractory B-cell Acute Lymphoblastic Leukemia

Autolus Therapeutics has achieved a significant milestone with FDA approval for AUCATZYL (obecabtagene autoleucel), a next-generation CAR T-cell therapy for adults with relapsed or refractory B-cell acute lymphoblastic leukemia (R/R B-ALL). This approval, granted based on results from the FELIX clinical trial, highlights the therapy’s efficacy, with a 63% overall complete remission rate in the efficacy-evaluable patient cohort. Notably, AUCATZYL also demonstrated a favorable safety profile with low rates of severe Cytokine Release Syndrome and Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS), without requiring a Risk Evaluation and Mitigation Strategy (REMS).

In addition to its U.S. approval, Autolus has expanded its manufacturing capabilities to meet demand, establishing the Nucleus facility in Stevenage, UK. This facility has received certifications from both the MHRA and the FDA, allowing it to support the global distribution of AUCATZYL. Autolus has now begun collaborating with U.S. treatment centers to make AUCATZYL available to eligible patients and is actively seeking additional approvals in the EU and UK.

“Adult ALL is an extremely aggressive cancer, and there is a high unmet medical need for these patients once they relapse,” said Dr. Elias Jabbour, U.S. lead investigator of the FELIX study and professor at MD Anderson Cancer Center. “This milestone approval, based on the demonstrated clinical benefit of AUCATZYL, brings new hope for adult patients with relapsed/refractory B-ALL.”

Dr. Claire Roddie, Associate Professor at University College London Cancer Institute and lead investigator of the FELIX study, commented, “In the FELIX trial, AUCATZYL has shown long-term persistence and deep responses, which we believe are critical for achieving lasting remissions in B-ALL. Based on these results, AUCATZYL provides an attractive risk-benefit profile for patients with this aggressive cancer.”

Unicycive’s Oxylanthanum Carbonate NDA Accepted by FDA for Hyperphosphatemia in Dialysis Patients

Unicycive Therapeutics, Inc., a clinical-stage biotechnology company, announced that the FDA has accepted its New Drug Application (NDA) for Oxylanthanum Carbonate. The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of June 28, 2025. If approved, OLC could provide a significant improvement in the treatment of hyperphosphatemia in patients with chronic kidney disease on dialysis, reducing the treatment burden that these patients currently face.

“We are thrilled with the FDA acceptance of our first NDA, a significant milestone towards our efforts to bring this important treatment option to patients with kidney disease if approved,” said Shalabh Gupta, MD, Chief Executive Officer of Unicycive. “CKD patients on dialysis with hyperphosphatemia are often saddled with an onerous treatment regimen that includes having to take as many as 12 pills per day. OLC may have meaningful patient adherence benefits over currently available treatment options as it requires a lower pill burden for patients in terms of the number and size of pills per dose, and the pills are swallowed instead of chewed for added convenience. With our NDA now under review, we are preparing to commercialize and launch OLC in the second half of 2025, if approved.”

Unicycive is pursuing FDA approval of OLC via the 505(b)(2) regulatory pathway, with the NDA submission supported by data from three clinical studies and a robust preclinical research package. The company also highlighted that OLC is protected by a strong global patent portfolio, with exclusivity until 2031, potentially extending to 2035. Additionally, the FDA granted a waiver for PDUFA fees, providing Unicycive with savings of approximately $4 million.

AstraZeneca/Amgen Report Positive Chronic Rhinosinusitis Trial Results, But Face Regulatory Hurdles

AstraZeneca and Amgen announced that their subcutaneous antibody TEZSPIRE (tezepelumab) achieved the primary endpoint in the Phase III WAYPOINT study for chronic rhinosinusitis with nasal polyps, demonstrating a “statistically significant and clinically meaningful” reduction in nasal polyp size and congestion compared to placebo. Tezspire, which already has FDA approval for severe asthma, maintained a safety profile consistent with previous trials, according to the companies. Specific data was not disclosed, and no immediate plans to file for CRSwNP approval were mentioned.

Commenting on the announcement, William Blair analyst Matt Phipps noted that only one study has been conducted for Tezspire in CRSwNP, contrasting with Sanofi and Regeneron’s Dupixent (dupilumab), which was FDA-approved for CRSwNP in 2019 after two pivotal trials. “Ahead of the full data, we do not expect Tezspire to necessarily beat Dupixent on efficacy,” Phipps stated, highlighting that CRSwNP patients typically exhibit high type-2 inflammation—specifically targeted by Dupixent. In contrast, Tezspire targets TSLP cytokine, a key initiator in multiple inflammatory pathways associated with asthma, chronic obstructive pulmonary disease, and CRSwNP.

Sharon Barr, executive vice president of BioPharmaceuticals R&D at AstraZeneca, expressed enthusiasm, saying, “These findings reinforce that Tezepelumab’s first-in-class mode of action effectively addresses the multiple drivers of epithelial-driven inflammatory diseases.” The companies plan to submit these data to regulatory authorities and present them at an upcoming medical congress.

This recent success in the WAYPOINT study comes shortly after GSK’s depemokimab Phase III results in September, which showed significant efficacy in reducing asthma exacerbations and hospital visits. Like Tezspire, depemokimab is being developed for both severe asthma and CRSwNP, with coordinated regulatory filings planned for these indications.

Nipocalimab Receives FDA Breakthrough Therapy Designation for Moderate-to-Severe Sjögren’s Disease

Johnson & Johnson announced that the FDA has granted Breakthrough Therapy Designation to nipocalimab for the treatment of adults with moderate-to-severe Sjögren’s disease. This autoimmune disease affects multiple organs and lacks approved advanced treatments. Nipocalimab, the first investigational therapy to receive this designation for SjD, previously earned BTD for treating severe hemolytic disease of the fetus and newborn (HDFN) in alloimmunized pregnant individuals.

This FDA designation is supported by data from the Phase II DAHLIAS study, where nipocalimab demonstrated positive results in addressing SjD symptoms. A Phase 3 study is currently underway to further evaluate its safety and efficacy. Nipocalimab’s DAHLIAS study findings were also presented at the 2024 EULAR Congress, marking the first success of an investigational FcRn blocker for SjD.

“Today’s announcement represents a critical milestone for our work with nipocalimab,” said Terence Rooney, Vice President of Rheumatology at Johnson & Johnson Innovative Medicine. “With no treatments currently approved to address SjD’s underlying causes, there is an urgent need for innovation to improve patient outcomes.”

SjD can significantly impact life quality, with many experiencing systemic symptoms and a heightened risk of severe conditions like B-cell lymphomas. This breakthrough designation expedites nipocalimab’s development to potentially offer a targeted therapy for SjD, a disease associated with increased morbidity and mortality.

AbbVie’s Schizophrenia Drug Fails in Two Studies

AbbVie recently reported that its two Phase II EMPOWER trials, which investigated emraclidine as a once-daily oral monotherapy for schizophrenia patients experiencing acute psychotic symptoms, did not meet the primary endpoint. Specifically, the trials failed to show a statistically significant reduction in the Positive and Negative Syndrome Scale (PANSS) total score when compared to placebo after six weeks. Despite these disappointing results, AbbVie emphasized that emraclidine was well-tolerated, with adverse events comparable to those seen in earlier studies.

Roopal Thakkar, M.D., executive vice president of research and development at AbbVie, commented, “While we are disappointed with the results, we are continuing to analyze the data to determine next steps.” He added, “We would like to extend our gratitude to the study participants and their loved ones as well as to our network of clinical investigative sites for their participation in these trials. We are confident that our innovative pipeline will continue to bring meaningful therapies to patients, and we remain committed to finding better treatments for people living with psychiatric and neurological disorders.”

AbbVie’s neuroscience portfolio remains central to its research focus, particularly after the recent Cerevel acquisition, which brought multiple clinical-stage and preclinical candidates that align with AbbVie’s pipeline in psychiatry, migraine, and Parkinson’s disease. With promising options beyond emraclidine, AbbVie is optimistic about the potential for innovative therapies in treating complex neurological conditions.