Biotech Stock Roundup: BIOA Down on Study Update, QURE, CRDF Gain on Study Data & More

It was a busy week in the biotech sector with lots of data readouts. This apart, the key regulatory updates and pipeline news also continued to be in the spotlight.Recap of the Week’s Most Important Stories:BIOA Plummets on Study DataBioAge Labs’ BIOA shares plunged after the company announced that it would discontinue the ongoing mid-stage study of its investigational drug candidate, azelaprag.The STRIDES is a phase II randomized, double-blind, placebo-controlled study of azelaprag as monotherapy and in combination with Eli Lilly’s tirzepatide that planned to enroll approximately 220 individuals with obesity (aged 55 years and older). The study was designed to evaluate the efficacy as measured by body weight reduction and other outcomes, safety, and tolerability of two oral doses of azelaprag (300 mg, once or twice daily) in combination with tirzepatide (5 mg subcutaneous injection once weekly). A monotherapy arm was included to provide additional safety information.BIOA discontinued both arms after liver transaminitis without clinically significant symptoms was observed in some subjects receiving azelaprag. However, no transaminase elevations were observed in the tirzepatide only treatment group.QURE Up on Regulatory UpdateuniQure N.V. QURE announced that it has reached an agreement with the FDA on key elements of an accelerated approval pathway for AMT-130 in Huntington’s Disease. Shares gained 109.73% on the news.AMT-130 obtained the FDA’s Regenerative Medicine Advanced Therapy (“RMAT”) designation to treat Huntington’s disease.The deal was made at an RMAT meeting with the regulatory body in November 2024. The FDA agreed that the reported data from the ongoing early to mid-stage studies of AMT-130 for Huntington’s disease, compared to a natural history external control, are sufficient to serve as the primary basis for a biologics license application (BLA) for accelerated approval.Furthermore, the regulatory body has agreed that cUHDRS may be used as an intermediate clinical endpoint and that reductions in neurofilament light chain (NfL) measured in cerebrospinal fluid may serve as supportive evidence of therapeutic benefit in the application for accelerated approval.QURE initiated BLA readiness activities and looks forward to further engaging with the FDA in the first half of 2025 to discuss its statistical analysis plan and the technical requirements.CRDF Stock Up on Colorectal Cancer Study DataCardiff Oncology CRDF announced positive initial data from an ongoing mid-stage study evaluating two doses of its lead investigational candidate, onvansertib (20 mg and 30 mg), in combination with standard-of-care (SOC) chemotherapy to treat first-line RAS-mutated metastatic colorectal cancer (mCRC) patients. The stock surged on the news.Initial results from randomized phase II CRDF-004 demonstrated an objective response rate (ORR) of 64% in the 30mg onvansertib dose arm, almost double the 33% ORR observed in the control arm (SOC alone). The 30 mg dose arm also demonstrated a higher ORR than 50% observed in the 20 mg dose arm. Overall, patients treated with onvansertib demonstrated an ORR of 57%.The company also shared spider plots in the press release, which show that patients receiving the 30mg dose of onvansertib with SOC experienced greater tumor size reduction over time compared to those in the control and 20mg dose arms with similar safety profiles for both doses. The combo therapy was also well tolerated in the CRDF-004 study, and no major or unexpected toxicities were observed.Cardiff Oncology expects to share additional clinical updates from the mid-stage study in the first half of 2025.INCY’s Study Meets GoalsIncyte INCY announced positive results from the late-stage study on retifanlimab.The phase III POD1UM-304 study is evaluating retifanlimab in combination with platinum-based chemotherapy for the treatment of adults with previously untreated non-squamous and squamous metastatic non-small cell lung cancer (NSCLC) not harboring a driver mutation.Results from the POD1UM-304 study showed a clinically meaningful and statistically significant improvement over chemotherapy alone. Patients in the retifanlimab and chemotherapy combination treatment group achieved a median OS of 18.1 months compared to 13.4 months in the placebo and chemotherapy combination group.The POD1UM-304 study met the primary endpoint of OS and all secondary endpoints in patients with previously untreated metastatic NSCLC.Retifanlimab is marketed as Zynyz by INCY in the United States for Merkel cell carcinoma. In 2017, Incyte entered into an exclusive collaboration and license agreement with MacroGenics, Inc. for global rights to retifanlimab.Incyte currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.GSK Moves With Label Expansion of NucalaGSK plc GSK announced that the FDA has accepted its regulatory filing, seeking the approval of a new indication for Nucala (mepolizumab) in chronic obstructive pulmonary disease (COPD) treatment for review. A decision from the regulatory body is expected on May 7, 2025.GSK’s Nucala is a monoclonal antibody with a novel mechanism of action that targets IL-5, a key messenger protein (cytokine) in type 2 inflammation. Please note that Nucala is already approved in the United States for four IL-5-mediated conditions — severe eosinophilic asthma (aged 6+), chronic rhinosinusitis with nasal polyps in adults, eosinophilic granulomatosis with polyangiitis in adults and hypereosinophilic syndrome in patients aged 12+. Currently, the drug is not approved for COPD worldwide.GSK’s regulatory filing seeking the approval of Nucala as an add-on maintenance treatment for patients with COPD with an eosinophilic phenotype was supported by positive data from its phase III MATINEE study.PerformanceThe Nasdaq Biotechnology Index has gained 0.17% in the past five trading sessions and Regeneron’s shares have risen 3.86%. In the past six months, shares of MRNA have lost 72.06%. (See the last biotech stock roundup here: Biotech Stock Roundup: BBIO Stock Up on Drug Approval, SAVA, APLT Plunge on Setbacks & More)Image Source: Zacks Investment ResearchWhat's Next in Biotech?Stay tuned for more pipeline updates. Only $1 to See All Zacks' Buys and SellsWe're not kidding.Several years ago, we shocked our members by offering them 30-day access to all our picks for the total sum of only $1. No obligation to spend another cent.Thousands have taken advantage of this opportunity. Thousands did not - they thought there must be a catch. Yes, we do have a reason. We want you to get acquainted with our portfolio services like Surprise Trader, Stocks Under $10, Technology Innovators,and more, that closed 228 positions with double- and triple-digit gains in 2023 alone.See Stocks Now >>Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report GSK PLC Sponsored ADR (GSK): Free Stock Analysis Report Incyte Corporation (INCY): Free Stock Analysis Report BioAge Labs, Inc. (BIOA): Free Stock Analysis Report uniQure N.V. (QURE): Free Stock Analysis Report Cardiff Oncology, Inc. (CRDF): Free Stock Analysis ReportTo read this article on Zacks.com click here.Zacks Investment ResearchWeiter zum vollständigen Artikel bei Zacks Weiter zum vollständigen Artikel bei Zacks