In May 2025, the FDA accepted the RGX-121 BLA under the accelerated approval pathway; however, the Prescription Drug User Fee Act (PDUFA) goal date was extended from November 9, 2025, to February 8, 2026.The CatalystThe FDA issued a CRL for RGX-121, citing concerns about the study eligibility criteria and the appropriateness of a surrogate endpoint for predicting clinical benefit.The CRL lists several potential paths forward, including a new study, treating additional patients, conducting longer-term follow ...

Regenxbio said on Monday the U.S. Food and Drug Administration has declined to approve its drug for a rare disease. ...

Core Viewpoint - REGENXBIO Inc. is under investigation for potential violations of federal securities laws following the FDA's clinical holds on its RGX-111 and RGX-121 programs, which resulted in a significant decline in the company's share price by 30-35% after the announcement [1]. Regulatory Compliance - SEC disclosure rules mandate that public companies must provide material information for informed investment decisions, with Item 8.01 of Form 8-K allowing disclosure of material events not covered by other items [2]. - Rule 10b-5 under the Securities Exchange Act of 1934 prohibits material misstatements and omissions in securities transactions, covering both false statements and omissions of necessary facts [2]. Company Communication - During the Q3 2025 earnings call, CEO Curran Simpson highlighted positive regulatory interactions, noting that the FDA completed inspections with no observations, which may have created an asymmetric view of the company's regulatory status by not disclosing safety concerns being evaluated [3]. - The Q3 2025 earnings call transcript notably lacked any discussion regarding the RGX-111 program for MPS I, despite it being a significant pipeline asset that later faced the same FDA clinical hold, raising concerns about the completeness of information provided to shareholders [4].

Core Viewpoint - The Schall Law Firm is investigating claims against REGENXBIO Inc. for potential violations of securities laws related to misleading statements and undisclosed information affecting investors [1][2]. Group 1: Investigation Details - The investigation centers on whether REGENXBIO issued false or misleading statements or failed to disclose critical information to investors [2]. - A press release from REGENXBIO on January 28, 2026, revealed that the FDA placed a clinical hold on its gene therapy RGX-111 for treating MPS I due to a case of neoplasm in a participant from a Phase I/II study [2]. - The FDA also placed a clinical hold on RGX-121 for MPS II, citing similarities in products and shared risks between the clinical studies [2]. Group 2: Market Reaction - Following the announcement of the FDA's clinical hold, shares of REGENXBIO fell by 17.9% on the same day [2].

NEW YORK, Feb. 03, 2026 (GLOBE NEWSWIRE) -- Pomerantz LLP is investigating claims on behalf of investors of REGENXBIO Inc. (“Regenxbio” or the “Company”) (NASDAQ: RGNX).  Such investors are advised to contact Danielle Peyton at newaction@pomlaw.com or 646-581-9980, ext. 7980. The investigation concerns whether Regenxbio and certain of its officers and/or directors have engaged in securities fraud or other unlawful business practices.  [Click here for information about joining the class action] On January 28 ...

Core Viewpoint - REGENXBIO Inc. is under investigation for potential violations of federal securities laws following the FDA's clinical holds on its RGX-111 and RGX-121 programs, which resulted in a significant decline in the company's share price by 30-35% [1]. Regulatory Compliance - Public companies are required to disclose material information to investors as per SEC disclosure rules, specifically through Form 8-K, which allows for the reporting of material events not covered by other items [2]. - Rule 10b-5 under the Securities Exchange Act of 1934 prohibits both material misstatements and omissions in securities transactions, emphasizing the need for complete and accurate disclosures [2]. Company Communication - During the Q3 2025 earnings call, CEO Curran Simpson highlighted positive regulatory interactions, noting that the FDA completed inspections with no observations, which may have created an asymmetric view of the company's regulatory status by not disclosing safety concerns being evaluated [3]. - The Q3 2025 earnings call transcript notably lacked any discussion regarding the RGX-111 program for MPS I, despite it being a significant pipeline asset that later faced the same FDA clinical hold, raising concerns about the completeness of information provided to shareholders [4].

FDA Approvals & Rejections - Intellia Therapeutics has received FDA approval to resume its MAGNITUDE-2 Phase 3 trial for nexiguran ziclumeran (nex-z) targeting hereditary transthyretin amyloidosis with polyneuropathy, increasing target enrollment from 50 to 60 patients [2][4] - Outset Medical's next-generation Tablo Hemodialysis System has been granted FDA 510(k) clearance, making it the first dialysis device to meet enhanced cybersecurity standards, with shipping expected to begin in Q2 2026 [6][7] - OKYO Pharma has received positive feedback from the FDA for its Phase 2b/3 trial design for Urcosimod, a candidate for neuropathic corneal pain, with plans to start the trial in the first half of 2026 [8][9] - REGENXBIO has faced clinical holds on its RGX-111 and RGX-121 gene therapy programs due to a case of CNS tumor in a child treated with RGX-111, although no similar findings were reported in other patients [10][11] - Almirall has received NMPA approval for Seysara in China for treating moderate-to-severe acne vulgaris, expanding its dermatology portfolio in the region [12][13] Clinical Trials - Breakthroughs - Aprea Therapeutics reported early clinical activity for APR-1051 in endometrial cancer, achieving a 50% reduction in target lesion size in a patient with PPP2R1A-mutated uterine serous carcinoma [19][21] - Fractyl Health's Revita demonstrated positive results in weight maintenance after GLP-1 drug discontinuation, showing a 4.5% weight regain compared to 7.5% in the sham group [22][24] - Ascletis Pharma announced positive Phase 3 results for Denifanstat in moderate-to-severe acne vulgaris, focusing on long-term safety in a trial with 240 patients [25][26] - GRI Bio reported new gene expression data from its Phase 2a study of GRI-0621 in idiopathic pulmonary fibrosis, showing significant improvements in lung injury and fibrosis progression [27][28] - Cardiff Oncology announced encouraging results from its Phase 2 trial of Onvansertib in RAS-mutated metastatic colorectal cancer, with a well-tolerated regimen and plans to advance to a registrational program [31][32] - Genentech's CT-388 Phase 2 trial for obesity showed a significant placebo-adjusted weight loss of 22.5% at 48 weeks, with a high percentage of participants achieving significant weight loss [34][36] - Sarepta Therapeutics reported positive three-year results from its EMBARK study for ELEVIDYS in Duchenne muscular dystrophy, showing significant slowing of disease progression in treated patients [38][41] Deals - YD Bio Limited has signed a letter of intent to acquire Safe Save Medical for approximately $26.87 million, aiming to enhance its capabilities in advanced cellular therapeutics [14][15][17]

Company Overview - Regenxbio (RGNX) shares increased by 5.4% to close at $11.6, following a notable trading volume, despite a 23.5% loss over the past four weeks [1] - The stock's rebound is attributed to a market correction after a steep selloff due to the FDA's clinical hold on its gene therapy RGX-111 for MPS I and RGX-121 for MPS II, following a case of neoplasm in a patient [2] Financial Performance - Regenxbio is expected to report a quarterly loss of $0.99 per share, reflecting a year-over-year change of +2%, with revenues projected at $31.49 million, an increase of 48.5% from the previous year [3] - The consensus EPS estimate for the upcoming quarter has remained unchanged over the last 30 days, indicating a lack of upward trend in earnings estimate revisions [4] Industry Context - Regenxbio is part of the Zacks Medical - Biomedical and Genetics industry, where another company, CytomX Therapeutics (CTMX), experienced a 2.7% decline to $5.74, despite a 38.5% return over the past month [4] - CytomX Therapeutics' consensus EPS estimate has decreased by 0.5% to -$0.08, representing a significant year-over-year change of -136.4% [5]

NEW YORK, Jan. 29, 2026 /PRNewswire/ -- Pomerantz LLP is investigating claims on behalf of investors of REGENXBIO Inc. ("Regenxbio" or the "Company") (NASDAQ: RGNX). Such investors are advised to contact Danielle Peyton at [email protected] or 646-581-9980, ext. 7980. The investigation concerns whether Regenxbio and certain of its officers and/or directors have engaged in securities fraud or other unlawful business practices.  [Click here for information about joining the class action] On January 28, 2026, ...

Core Viewpoint - Regenxbio Inc. is under investigation for potential discrepancies between the information provided to investors and the actual safety developments in its gene therapy clinical programs, particularly following the FDA's clinical holds on its RGX-111 and RGX-121 programs due to safety concerns [1][2]. Group 1: Company Developments - On January 28, 2026, Regenxbio disclosed that the FDA placed clinical holds on its RGX-111 and RGX-121 programs after a tumor was identified in a trial participant, leading to a 30-35% decline in the company's share price [2]. - During the Q3 2025 earnings call on November 6, 2025, CEO Curran Simpson highlighted positive regulatory interactions, noting that the FDA completed inspections of clinical sites and the manufacturing facility with no observations, which may have created an asymmetric presentation of the company's regulatory standing [4]. - The Q3 2025 earnings call transcript did not mention the RGX-111 program for MPS I, despite it being a significant pipeline asset that would later face the same FDA clinical hold, raising concerns about the completeness of information provided to shareholders [5]. Group 2: Regulatory and Legal Context - SEC disclosure rules require public companies to provide material information necessary for informed investment decisions, and Rule 10b-5 prohibits material misstatements and omissions in securities transactions [3].