NEW YORK, Feb. 03, 2026 (GLOBE NEWSWIRE) -- Pomerantz LLP is investigating claims on behalf of investors of Ultragenyx Pharmaceutical Inc. (“Ultragenyx” or the “Company”) (NASDAQ: RARE). Such investors are advised to contact Danielle Peyton at newaction@pomlaw.com or 646-581-9980, ext. 7980. The investigation concerns whether Ultragenyx 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 acti ...

Core Insights - Ultragenyx Pharmaceutical (RARE) has resubmitted its biologics license application (BLA) to the FDA for accelerated approval of its gene therapy candidate UX111, aimed at treating Sanfilippo syndrome type A (MPS IIIA) [1][8] - The company’s shares have decreased by 44.6% over the past year, contrasting with a 15.2% increase in the industry [2] BLA Resubmission Details - The FDA issued a Complete Response Letter (CRL) in July 2025, requesting additional information regarding chemistry, manufacturing, and controls (CMC) elements, which were facility- and process-related issues not tied to product quality [3][10] - The resubmitted BLA addresses all CMC observations from the CRL and includes long-term data supporting neurological benefits, along with biomarker data in line with FDA agreements [4][11] Regulatory Timeline - A target action date from the FDA is expected within a month, with a review period of up to six months anticipated, aiming for a decision in the third quarter of 2026 [5][8] Clinical Data and Efficacy - The original BLA submission was supported by data from the phase I/II/III Transpher A study, which showed that UX111 treatment led to a significant reduction in heparan sulfate (HS) levels in cerebrospinal fluid (CSF) and improved long-term cognitive development [9][11] - The updated clinical data in the resubmission includes an additional year of patient follow-up, demonstrating durable treatment benefits and increasing separation from untreated outcomes [11] Disease Context - Sanfilippo syndrome type A is a rare, fatal lysosomal storage disorder affecting the central nervous system, with approximately 3,000 to 5,000 patients in commercially accessible areas and a median life expectancy of 15 years [14]

Core Viewpoint - Ultragenyx Pharmaceutical Inc. has resubmitted its Biologics License Application (BLA) for UX111, a gene therapy for Sanfilippo syndrome type A, to the FDA, aiming for accelerated approval based on new long-term clinical data demonstrating positive effects over 8.5 years [2][3][5]. Group 1: Product and Clinical Data - UX111 (rebisufligene etisparvovec) is an AAV9 gene therapy in Phase 1/2/3 development targeting Sanfilippo syndrome type A, a rare and fatal lysosomal storage disease with no approved treatment [6]. - The resubmitted BLA includes comprehensive responses to previous FDA observations and additional long-term clinical data, which support an intermediate clinical endpoint for accelerated approval [3][4]. - Updated clinical data show a durable treatment effect across multiple biomarkers and maintain an acceptable safety profile, with detailed updates to be presented at WORLDSymposium™ 2026 [4]. Group 2: Regulatory Process and Timeline - The FDA granted Priority Review for the UX111 BLA in February 2025, with a Prescription Drug User Fee Act (PDUFA) action date expected within a month of resubmission [5]. - The company anticipates a review period of up to six months from the resubmission date, with a PDUFA date expected in the third quarter of 2026 [5]. Group 3: Disease Background - Sanfilippo syndrome type A (MPS IIIA) is characterized by rapid neurodegeneration, with onset in early childhood, leading to cognitive, language, and motor decline, and has a median life expectancy of 15 years [7]. - The disease affects approximately 3,000 to 5,000 patients in commercially accessible geographies and is caused by biallelic pathogenic variants in the SGSH gene, leading to a deficiency in the sulfamidase enzyme [7][8]. Group 4: Company Overview - Ultragenyx is focused on developing novel therapies for serious rare and ultra-rare genetic diseases, with a diverse portfolio aimed at addressing high unmet medical needs [9]. - The company is led by a management team experienced in rare disease therapeutics, emphasizing time- and cost-efficient drug development [10].

Core Viewpoint - Ultragenyx Pharmaceutical Inc. is under investigation for potential violations of federal securities laws following the failure of its Phase III studies to meet primary endpoints related to fracture rate reduction [1][2]. Group 1: Study Results - The Phase III studies, Orbit and Cosmic, did not achieve their primary endpoint of reducing annualized clinical fracture rates compared to placebo and standard care, respectively [2][3]. - Despite showing improvements in bone density, the studies failed to correlate these results with a reduction in fracture rates [3]. Group 2: Company Response and Analyst Reactions - In response to the study results, Ultragenyx management cited a "low fracture rate in the placebo group" as a reason for the failed outcomes, claiming no "uncontrolled factors" were present in the study design [4]. - Following the announcement, analysts became cautious, with Barclays indicating limited opportunity for drug approval despite some clinical benefits observed [4].

Core Viewpoint - Ultragenyx Pharmaceutical Inc. has faced setbacks following the announcement of topline results from its Phase III Orbit and Cosmic Studies, which did not meet primary endpoints for the effectiveness of setrusumab (UX143) on Osteogenesis Imperfecta [1][2]. Group 1: Study Results - The Phase III Orbit and Cosmic Studies failed to achieve their primary endpoint of reducing annualized clinical fracture rates compared to placebo and standard care, respectively [2]. - Despite showing improvements in bone density among tested patients, the studies did not correlate these results with a reduction in fracture rates [3]. Group 2: Company Response and Analyst Reactions - In response to the study results, Ultragenyx management cited a "low fracture rate in the placebo group" as a reason for the failed outcomes, asserting that the study was designed to increase fracture occurrences to better observe differences between test groups [3]. - Following the announcement, analysts became cautious, with Barclays indicating limited opportunity for drug approval despite some clinical benefits observed [4].

Core Viewpoint - Tactical Resources Corp. has successfully closed a non-brokered private placement, raising a total of $1,308,037.50 through the issuance of 207,625 common shares at a price of $6.30 per share [1]. Group 1 - The private placement was conducted without a broker, indicating a direct approach to capital raising by the company [1]. - The shares issued are subject to a statutory hold period of four months plus one day, in compliance with applicable securities legislation [1].

Core Insights - The article emphasizes the author's extensive background in finance and medicine, highlighting a unique combination of skills that enhance expertise in biotech and pharma investing [1] - It outlines the role of the company in fostering a community for sharing innovative investment ideas within the biotech/pharma sector [1] - The author provides insights into both short-term and long-term investment strategies, focusing on income investing and potential takeover targets in the biotech/pharma industry [1] Company and Industry Summary - The company specializes in high-growth biotech and pharma investment ideas, offering exclusive stock analysis tailored to investor needs [1] - It provides regular portfolio trade alerts and an interactive chat feature, promoting dynamic engagement and support for investors navigating the biotech sector [1] - The author has published a top-selling book on biotech investing and teaches a course on the subject, indicating a commitment to educating investors [1]

Core Insights - Ultragenyx Pharmaceutical Inc. is under investigation for potential violations of federal securities laws following the failure of its Phase III studies for setrusumab (UX143) in Osteogenesis Imperfecta [1][2] - The studies did not meet their primary endpoints, leading to a significant drop in Ultragenyx's stock price [2][3] Group 1: Study Results - The Phase III Orbit and Cosmic studies failed to achieve their primary endpoint of reducing annualized clinical fracture rates compared to placebo and biophosphonates, respectively [2] - Although secondary endpoints showed improvements in bone material density, these did not correlate with a statistically significant reduction in fractures [2] Group 2: Market Reaction - Following the announcement of the study results, Ultragenyx's stock price fell by $14.87, a decrease of 43.49%, opening at $19.32 per share [3] - This decline has resulted in the stock reaching a new 52-week low, significantly below the previous low of $25.81 [3] Group 3: Company Response - Ultragenyx has indicated that it will implement significant expense reductions in response to the study outcomes [3]

Core Viewpoint - Ultragenyx Pharmaceutical Inc. is under investigation for potential violations of federal securities laws following the failure of its Phase III studies for setrusumab (UX143) in treating Osteogenesis Imperfecta, leading to a significant drop in stock price [1][2][3] Group 1: Study Results - The Phase III Orbit and Cosmic studies did not meet their primary endpoint of reducing annualized clinical fracture rates compared to placebo and biophosphonates, respectively [2] - Although secondary endpoints showed improvements in bone material density, these did not correlate with a statistically significant reduction in fractures in either study [2] Group 2: Financial Impact - Following the announcement of the study results, Ultragenyx's stock price fell by $14.87, a decrease of 43.49%, opening at $19.32 per share on December 29, 2025 [3] - The stock decline has resulted in a new 52-week low, significantly below the previous low of $25.81 [3] Group 3: Company Response - Ultragenyx has indicated it will implement significant expense reductions in response to the study outcomes [2]

Core Viewpoint - Ultragenyx is positioned as a leader in the development of treatments for rare diseases, focusing on innovative approaches to drug development and commercialization [1][2]. Group 1: Company Strategy - The company aims to support not only its own initiatives but also other companies and patient foundations in the rare disease drug development space, indicating a collaborative approach within the industry [2]. - Ultragenyx employs a differentiated strategy that emphasizes potent biology in challenging diseases, carefully selecting the best treatment modalities for each specific condition [2]. Group 2: Development Approach - The company adopts creative development methodologies, including adaptive trial designs and innovative endpoint choices, which are essential for studying diseases that have not been previously researched [2][3]. - Ultragenyx believes in a lean commercial model that is focused on patient needs, which reflects its commitment to efficient and effective commercialization of its treatments [3].