BOSTON, Feb. 05, 2026 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global commercial-stage biopharmaceutical company focused on transforming the lives of patients living with rare neuroendocrine diseases, today announced that Hunter C. Smith, Chief Financial Officer, will participate in a fireside chat at the upcoming Guggenheim Emerging Outlook: Biotech Summit 2026 conference on Wednesday, February 11 at 11:00 a.m. ET in New York. The fireside chat will be webcasted and available unde ...

Company Overview - Phathom Pharmaceuticals, Inc. is a biopharmaceutical company focused on developing and commercializing novel treatments for gastrointestinal diseases [3] - The company has in-licensed exclusive rights to vonoprazan, a first-in-class potassium-competitive acid blocker (PCAB), for the U.S., Europe, and Canada [3] - Phathom markets vonoprazan in the U.S. as VOQUEZNA® for various gastrointestinal conditions, including heartburn associated with Non-Erosive GERD and H. pylori infection treatment [3] Upcoming Events - Members of Phathom's management team will participate in a fireside chat at the Guggenheim Emerging Outlook Biotech Summit 2026 on February 12, 2026 [1] - The conference will take place from February 11-12, 2026, in New York, NY, and will include one-on-one meetings [2] - A live webcast and archived recordings of the event will be available on Phathom's website for 90 days following the meeting [2]

Core Viewpoint - Cognition Therapeutics, Inc. has extended the duration of its expanded access program for dementia with Lewy bodies, allowing participants several more months of treatment beyond the initial twelve months [1]. Group 1: Expanded Access Program (EAP) - The EAP has been operational since June 2025 and aims to gather additional long-term safety data [2]. - Feedback from participants and their care partners has been favorable, highlighting the program's operational aspects that reduce travel for patients [2][3]. - A total of 32 individuals are enrolled across eight sites, each receiving 100 mg of oral zervimesine daily [2]. Group 2: Clinical Development and Regulatory Engagement - The EAP is part of a broader clinical development program for dementia with Lewy bodies, which includes the Phase 2 SHIMMER study [3]. - Cognition Therapeutics held a Type C meeting with the FDA on January 21, 2026, to discuss findings from the SHIMMER study, focusing on clinically meaningful endpoints for future studies [3]. Group 3: Company Overview - Cognition Therapeutics is a clinical-stage biopharmaceutical company focused on developing small molecule therapeutics for age-related neurodegenerative disorders [5]. - The company has completed Phase 2 studies for its lead candidate, zervimesine, in various conditions including dementia with Lewy bodies and mild-to-moderate Alzheimer's disease [5].

Core Viewpoint - The Ultragenyx class action lawsuit alleges that the company and its executives made misleading statements regarding the efficacy of setrusumab in treating Osteogenesis Imperfecta, leading to significant stock price declines when the truth was revealed [4][5][6]. Company Overview - Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company focused on developing treatments for rare and ultra-rare genetic diseases [3]. Allegations of the Lawsuit - The lawsuit claims that Ultragenyx misrepresented the reliability of information regarding setrusumab's effects and downplayed the risks associated with the Phase III Orbit study, which ultimately failed to show statistically significant results [4]. - On July 9, 2025, Ultragenyx disclosed that the Phase III Orbit study did not achieve statistical significance, resulting in a stock price drop of over 25% [5]. - On December 29, 2025, the company announced that both the Phase III Orbit and Cosmic studies failed to meet primary endpoints, causing a further stock price decline of more than 42% [6]. Legal Process - The Private Securities Litigation Reform Act of 1995 allows investors who purchased Ultragenyx stock during the class period to seek appointment as lead plaintiff in the lawsuit, representing the interests of all class members [7]. Law Firm Background - Robbins Geller Rudman & Dowd LLP is a leading law firm in securities class action recoveries, having recovered over $916 million for investors in 2025 alone, and a total of $8.4 billion over the past five years [8].

Core Viewpoint - CRISPR Therapeutics is identified as a stock that has the potential to double by 2030, with analysts maintaining positive ratings despite some adjustments in price targets [1][2]. Group 1: Analyst Ratings and Price Targets - Citizens maintained an Outperform rating on CRISPR Therapeutics while reducing the price target from $86 to $80, citing an undervalued pipeline and potential late-stage development for up to seven opportunities in 2027 [1]. - Bank of America analyst Alec Stranahan reduced the price target for CRISPR Therapeutics from $93 to $90 while maintaining a Buy rating, reflecting adjustments across its US Biopharmaceuticals coverage [2]. Group 2: Market Context and Trends - Various factors are aligning positively for the biotech sector, including rewards for positive data, large-cap biopharma investing in M&A and licensing, an increasing backlog of private companies, better access to capital, and minimal effects from drug price regulation [4]. - The firm suggests that while biotech has returned, the primary uncertainty lies in the longevity of this trend [4]. Group 3: Company Overview - CRISPR Therapeutics is a gene editing company that develops gene-based medicines for serious human diseases using its CRISPR/Cas9 platform [5].

Company Overview - EyePoint, Inc. is a clinical-stage biopharmaceutical company focused on developing and commercializing innovative therapeutics for serious retinal diseases [3][4] - The company's lead product candidate, DURAVYU™, is an investigational sustained delivery treatment combining vorolanib, a selective tyrosine kinase inhibitor, with next-generation bioerodible Durasert E™ technology [3][5] - EyePoint has a history of four approved drugs over three decades and has treated tens of thousands of eyes with its innovations [4] Product Development - DURAVYU™ is currently undergoing Phase 3 pivotal trials for wet age-related macular degeneration, with topline data expected in mid-2026 [3] - The first patient dosing in the pivotal Phase 3 clinical trials for diabetic macular edema is anticipated in the first quarter of 2026 [3] Corporate Events - George O. Elston, Executive Vice President and Chief Financial Officer of EyePoint, will participate in a fireside chat at the Guggenheim Emerging Outlook: Biotech Summit 2026 on February 12, 2026 [1] - A webcast and archived replay of the fireside chat will be available on the company's website [2]

Core Insights - Cullinan Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing first- or best-in-class therapies for autoimmune diseases and cancer [3] Group 1: Company Overview - Cullinan Therapeutics is advancing high-impact therapies leveraging expertise in T cell engagers, which are currently established in oncology and expanding into autoimmune diseases [3] - The company has a clinical-stage pipeline built on a rigorous scientific approach and innovation aimed at delivering new standards of care for patients [3] Group 2: Upcoming Events - The CEO Nadim Ahmed and CMO Jeffrey Jones will participate in a fireside chat at the Guggenheim Emerging Outlook: Biotech Summit 2026 on February 12, 2026, at 11:00 a.m. ET [1] - A webcast of the fireside chat will be available on the company's investor relations website [2]

Core Insights - Ocular Therapeutix is preparing to present topline results from the SOL-1 Phase 3 superiority trial for AXPAXLI™ in wet AMD at the 49 Macula Society Annual Meeting scheduled for February 25-28, 2026 [1][3] - The company plans to submit a New Drug Application (NDA) for AXPAXLI based on the SOL-1 52-week data, contingent on positive results and discussions with the FDA [1][3] - The SOL-R Phase 3 non-inferiority trial has completed randomization of 631 subjects, with topline results expected in Q1 2027 [1][3] - Ocular has a cash balance of $737.1 million as of December 31, 2025, which is projected to support operations into 2028 [1][5] Business Highlights - The SOL-1 trial is designed to evaluate the safety and efficacy of AXPAXLI in treating wet AMD, with a primary endpoint focused on maintaining visual acuity [3][16] - The SOL-R trial aims to demonstrate non-inferiority compared to aflibercept, with a primary endpoint at Week 56 [3][19] - The HELIOS-3 trial for diabetic retinopathy is currently underway, with a focus on subjects with moderately severe to severe non-proliferative diabetic retinopathy [4][24] Financial Performance - Total net revenue for Q4 2025 was $13.3 million, a decrease of 22.4% from $17.1 million in Q4 2024 [6] - For the full year 2025, total net revenue was $52.0 million, down 18.5% from $63.7 million in 2024 [6] - Research and development expenses increased to $50.8 million in Q4 2025 from $41.0 million in Q4 2024, reflecting ongoing clinical trial costs [7] - The company reported a net loss of $(64.7) million for Q4 2025, consistent with a net loss of $(48.4) million in Q4 2024 [10] Future Developments - The SOL-X open-label extension trial is expected to begin in Q2 2026, allowing subjects from SOL-1 and SOL-R to enroll for additional safety follow-up [3][21] - The company raised approximately $475 million from an equity offering in September 2025, with net proceeds supporting operations into 2028 [4]

Core Insights - Syndax Pharmaceuticals is a commercial-stage biopharmaceutical company focused on innovative cancer therapies [3] - The CEO, Michael A. Metzger, will participate in a fireside chat at the 2026 Guggenheim Emerging Outlook: Biotech Summit on February 12, 2026 [1] - The company has notable products in its pipeline, including Revuforj (revumenib) and Niktimvo™ (axatilimab-csfr), both of which are FDA-approved [3] Company Overview - Syndax Pharmaceuticals is dedicated to advancing cancer care through its innovative therapies [3] - The company is conducting several clinical trials to maximize the potential of its product pipeline [3] - Revuforj is a menin inhibitor, while Niktimvo™ is a monoclonal antibody targeting the CSF-1 receptor [3]

Core Insights - Exicure, Inc. announced positive results from a Phase 2 clinical study of burixafor, a selective CXCR4 inhibitor, demonstrating rapid lymphocyte mobilization and potential therapeutic applications beyond transplantation [1][2] Study Results - The Phase 2 study involved 12 participants with multiple myeloma, non-Hodgkin's lymphoma, and Hodgkin disease undergoing autologous stem cell transplantation, with 92% achieving the primary endpoint of collecting ≥5.0 × 10⁶ CD34+ cells/kg within two leukapheresis sessions [2][3] - Notably, 6 participants achieved the target in a single session, with median times to neutrophil and platelet engraftment recorded at 12 and 22 days, respectively [3] Mobilization Profile - Burixafor demonstrated peak mobilization of CD34+ cells within one hour of administration, significantly faster than existing CXCR4 inhibitors, which peak at 10-14 hours [4] - This rapid mobilization allows for same-day leukapheresis, potentially simplifying treatment logistics and reducing hospital resource utilization [4] Clinical Implications - The combination of burixafor and G-CSF resulted in substantial increases in circulating lymphocyte counts, with increases of up to 11-fold observed in multiple myeloma patients [5] - The study highlights burixafor's potential applicability in gene therapy and CAR-T cell therapies, which require efficient peripheral blood cell collection [5] Future Development - Exicure is advancing burixafor's clinical development, having completed an additional Phase 2 study combining burixafor with G-CSF and propranolol, with positive topline data presented at recent hematology meetings [6] - The company aims to leverage burixafor's capabilities in various hematologic diseases, including plans for a chemosensitization trial in acute myeloid leukemia [8]