NEW YORK, July 18, 2025 (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 actio ...

Core Viewpoint - Ultragenyx Pharmaceuticals faces a regulatory setback as the FDA issued a Complete Response Letter (CRL) for its biologics license application (BLA) for UX111, a gene therapy for Sanfilippo syndrome type A (MPS IIIA) [1][6]. Regulatory Update - The CRL requested additional information and enhancements related to chemistry, manufacturing, and controls, stemming from recent facility inspections. The issues are facility- and process-related, not linked to product quality, and can be addressed quickly [2][6]. - Ultragenyx plans to collaborate closely with the FDA to resolve the observations and expects to resubmit the BLA, which will initiate a review period of up to six months [2]. Clinical Data and Impact - The FDA acknowledged the robustness of the neurodevelopmental outcome data and supportive biomarker findings. However, updated clinical data from current patients is required for the resubmission [3]. - The regulatory delay has pushed the potential approval of UX111 for MPS IIIA to 2026. Year-to-date, shares of Ultragenyx have decreased by 30.5%, contrasting with a 0.9% decline in the industry [3]. Disease Context - MPS IIIA is a rare, fatal lysosomal storage disease affecting the brain, with no approved treatments available. Approximately 3,000 to 5,000 patients are affected, with a median life expectancy of 15 years [9]. Pipeline Overview - Ultragenyx has several other gene therapy candidates in its pipeline, including UX143 for osteogenesis imperfecta and UX701 for Wilson disease, with ongoing studies showing promising results [11][12]. - The investigational gene therapy UX111 has received multiple designations in the U.S. and EU, including Regenerative Medicine Advanced Therapy and Orphan Drug designations [10].

Core Viewpoint - The FDA issued a Complete Response Letter (CRL) for Ultragenyx's Biologics License Application (BLA) for UX111, a gene therapy for Sanfilippo syndrome type A, citing specific chemistry, manufacturing, and controls (CMC) observations that are resolvable [1][2] Group 1: Company Overview - Ultragenyx Pharmaceutical Inc. is focused on developing therapies for rare and ultra-rare genetic diseases, with a diverse portfolio aimed at addressing high unmet medical needs [6] - The company is led by a management team experienced in rare disease therapeutics, emphasizing time- and cost-efficient drug development [7] Group 2: Product Information - UX111 is an in vivo gene therapy in Phase 1/2/3 development for Sanfilippo syndrome type A (MPS IIIA), a rare fatal lysosomal storage disease with no approved treatment [4] - The therapy targets the SGSH enzyme deficiency, which leads to the accumulation of heparan sulfate in the brain, causing neurodegeneration [4] - UX111 is administered via a one-time intravenous infusion using a self-complementary AAV9 vector to deliver a functional copy of the SGSH gene [4] Group 3: Regulatory Status - The FDA's CRL did not raise any issues regarding the clinical data package or clinical inspections, acknowledging the robustness of the neurodevelopmental outcome data and supportive biomarker data [3] - The company plans to address the CMC observations and resubmit the BLA, anticipating a review period of up to 6 months post-resubmission [2][3] Group 4: Disease Context - Sanfilippo syndrome type A (MPS IIIA) affects approximately 3,000 to 5,000 patients in commercially accessible areas, characterized by rapid neurodegeneration and a median life expectancy of 15 years [5] - The disease is caused by biallelic pathogenic variants in the SGSH gene, leading to a deficiency in the sulfamidase enzyme [5]

This article is published by Terry Chrisomalis, who runs the Biotech Analysis Central pharmaceutical service on Seeking Alpha Marketplace. If you like what you read here and would like to subscribe to, I'm currently offering a two-week free trial period for subscribers to take advantage of. My service offers a deep-dive analysis of many pharmaceutical companies. The Biotech Analysis Central SA marketplace is $49 per month, but for those who sign up for the yearly plan will be able to take advantage of a 33. ...

Company Overview - Proactive is a financial news publisher that provides fast, accessible, informative, and actionable business and finance news content to a global investment audience [2] - The company has a team of experienced news journalists who produce independent content across various financial markets [2] Market Focus - Proactive specializes in medium and small-cap markets while also covering blue-chip companies, commodities, and broader investment stories [3] - The content includes insights into sectors such as biotech and pharma, mining and natural resources, battery metals, oil and gas, crypto, and emerging digital and EV technologies [3] Technology Adoption - Proactive is committed to adopting technology to enhance its content creation and workflow processes [4] - The company utilizes automation and software tools, including generative AI, while ensuring that all published content is edited and authored by humans [5]

Core Insights - Ultragenyx Pharmaceutical (RARE) and Mereo BioPharma (MREO) have provided an update on the mid-to-late-stage Orbit study for UX143 (setrusumab) aimed at treating osteogenesis imperfecta (OI) in pediatric and young adult patients, with final analysis expected by the end of 2025 [1][5] - The shares of both companies experienced a significant decline in premarket trading following the announcement of the delay in new data from the Orbit study [2][5] - UX143 is a fully human monoclonal antibody that inhibits sclerostin, and interim safety data has shown an acceptable safety profile, allowing the study to proceed as planned [2][5] Study Details - The Orbit study is assessing the impact of UX143 on fracture rates in patients aged 5 to 25 with OI, with a pivotal phase III portion enrolling 159 additional patients [7] - The primary efficacy endpoint of the Orbit study is to reduce the annualized clinical fracture rate, with participants moving into an open-label extension period after the primary analysis [7] - The Cosmic study targets a younger population aged two to under seven years, comparing UX143 to intravenous bisphosphonates for fracture reduction, with 69 patients enrolled [8] Partnership and Market Context - Ultragenyx Pharmaceuticals and Mereo BioPharma entered a licensing agreement in 2020 for the joint development of UX143, with RARE holding exclusive rights in several regions [9] - The partnership was expanded in 2024 through a manufacturing and supply agreement, with RARE responsible for supplying setrusumab to MREO [9] - OI affects approximately 60,000 individuals in commercially accessible geographies, and there are currently no approved treatments available [11]

Company Overview - Ultragenyx (RARE) shares increased by 5.1% to $41.44 in the last trading session, with a notable trading volume [1] - The stock has also gained 5.1% over the past four weeks [1] Pipeline Progress - The rise in stock price is linked to positive investor sentiment regarding Ultragenyx's late-stage pipeline, particularly the regulatory application for FDA approval of UX111, a gene therapy for Sanfilippo syndrome type A, currently under Priority Review with a decision expected on August 18, 2025 [2] - Ultragenyx plans to submit a biologics license application for DTX401, an AAV8 gene therapy for glycogen storage disease type Ia, which could further enhance revenue if approved [2] Financial Expectations - The company is projected to report a quarterly loss of $1.27 per share, reflecting a year-over-year increase of 16.5%, with expected revenues of $161.61 million, up 9.9% from the previous year [3] - The consensus EPS estimate for the quarter has remained unchanged over the last 30 days, indicating that stock price movements may not sustain without trends in earnings estimate revisions [4] Industry Context - Ultragenyx operates within the Zacks Medical - Biomedical and Genetics industry, where another company, Fortress Biotech (FBIO), saw a 3.1% increase in its stock price, closing at $1.99, although FBIO has returned -7.2% over the past month [4] - Fortress Biotech's consensus EPS estimate for its upcoming report is -$0.31, which is a 57.5% improvement from the previous year, and it also holds a Zacks Rank of 3 (Hold) [5]

Core Viewpoint - Ultragenyx Pharmaceutical Inc. and Mereo BioPharma Group plc stocks are experiencing significant declines following the update on the Phase 3 Orbit study for UX143 (setrusumab) in patients with osteogenesis imperfecta (OI) [1][2]. Group 1: Study Progress and Analysis - The Phase 3 portion of the Orbit study is progressing towards a final analysis expected by the end of the year, in line with the original plan [2]. - The second interim analysis did not meet the minimal p-value threshold of p<0.01, which would have allowed the study to conclude early [3]. - The Data Monitoring Committee confirmed that UX143 has an acceptable safety profile, recommending the continuation of the study [3][4]. Group 2: Future Expectations - The final analyses for the Orbit and Cosmic studies will occur after patients have been on therapy for at least 18 months, with thresholds set at p<0.04 for Orbit and p<0.05 for Cosmic [5]. - Analysts express optimism regarding the final analysis, anticipating a positive outcome due to longer follow-up duration and a lower statistical significance threshold [6]. Group 3: Market Reaction - Following the update, RARE stock has dropped by 26.2% to $30.60, while MREO stock has decreased by 34.7% to $1.919 [7].

Core Insights - The Phase 3 Orbit study for UX143 (setrusumab) in pediatric and young adult patients with osteogenesis imperfecta (OI) is on track for final analysis by the end of the year [1][3] - The Data Monitoring Committee has confirmed an acceptable safety profile for UX143, allowing the study to proceed [2] - Ultragenyx and Mereo BioPharma are collaborating on the development of setrusumab, targeting OI sub-types I, III, and IV [5][12] Study Details - The Phase 3 Orbit study has enrolled 159 patients across 45 sites in 11 countries, with a primary efficacy endpoint focused on annualized clinical fracture rate [7] - The Cosmic study, which is also in Phase 3, has enrolled 69 patients aged 2 to <7 years, comparing setrusumab to intravenous bisphosphonates [8] - Both studies will conduct final analyses after patients have been on therapy for at least 18 months, with specific statistical thresholds set for each study [4] Background on Osteogenesis Imperfecta - OI is a genetic disorder affecting bone metabolism, primarily caused by mutations in the COL1A1 or COL1A2 genes, leading to increased bone brittleness and a high rate of fractures [9] - Approximately 60,000 individuals are affected by OI in commercially accessible regions, with no globally approved treatments available [9] Mechanism of Setrusumab - Setrusumab is a fully human monoclonal antibody that inhibits sclerostin, which negatively regulates bone formation, potentially increasing bone mass and strength [10] - Previous studies have shown that anti-sclerostin antibodies can significantly improve bone formation and density in OI models [11] Company Profiles - Ultragenyx is focused on developing therapies for rare genetic diseases, with a portfolio aimed at addressing high unmet medical needs [13] - Mereo BioPharma is also dedicated to innovative therapeutics for rare diseases, with setrusumab as one of its key candidates [15]

Pipeline and Approvals - Ultragenyx expects Phase 3 data readout for UX143 for Osteogenesis Imperfecta (OI) in 2025[21, 32] - Ultragenyx anticipates completing Phase 3 enrollment for GTX-102 for Angelman Syndrome (AS) in the second half of 2025[46, 49] - The company has a PDUFA date of August 18, 2025, for UX111 for Sanfilippo syndrome (MPS IIIA), with a potential commercial launch in the second half of 2025[63, 67] - Ultragenyx expects to submit a BLA for DTX401 for Glycogen Storage Disease Type Ia (GSDIa) in mid-2025, with a potential launch in 2026[78, 80] Financial Performance and Projections - Ultragenyx projects total revenue to grow by 14-20% in 2025, reaching $640-670 million[93, 95] - The company anticipates Crysvita revenue to be $460-480 million in 2025, representing a 12-17% increase[95] - Dojolvi revenue is projected to be $90-100 million in 2025, a 2-13% increase[95] - Ultragenyx aims to achieve full-year GAAP profitability in 2027, driven by revenue growth, new product launches, and expense management[2, 96, 99] Clinical Trial Results - Phase 2 data for UX143 in OI showed a 67% reduction in annualized fracture rate (AFR) [33] - Phase 3 data for DTX401 in GSDIa demonstrated a statistically significant 41% reduction in daily cornstarch intake at Week 48 (p < 0.0001)[83] - UX111 for MPS IIIA showed >80% of participants reduced CSF HS by 50% in efficacy set[71]