FDA PDUFA Goal Date Set for October 17, 2026RESEARCH TRIANGLE PARK, N.C., Feb. 25, 2026 (GLOBE NEWSWIRE) -- Opus Genetics, Inc. (Nasdaq: IRD) (“Opus Genetics” or the “Company”), a clinical-stage biopharmaceutical company developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases (IRDs), today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the supplemental New Drug Application (sNDA) for phentolamine ophthalmic solution 0. ...

Group 1 - Beam Therapeutics (NASDAQ:BEAM) is identified as one of the 17 biotechnology stocks with more than 50% upside potential, with a Buy rating and a price target of $80, indicating an upside of over 197% [1] - The company's recent agreement with the U.S. FDA to expedite the approval process for BEAM-302 in alpha-1 antitrypsin deficiency is a significant factor in the positive rating, allowing the use of AAT biomarker data over a 12-month horizon for future BLA [2] - Beam Therapeutics has a strong financial position, with nearly $1.25 billion in cash and marketable securities as of the end of 2025, sufficient to support operations until 2029 and fund the development of BEAM-302 and the anticipated launch of risto-cel [3] Group 2 - UBS analyst Michael Yee assigned a Neutral rating with a $28 price target on January 8, suggesting that more certainty for investors could benefit the biotech sector in 2026, with expectations of major catalysts and attractive pipelines driving returns [4] - Beam Therapeutics focuses on developing precision genetic medicines, engaging in gene therapies and genome editing research, and is a leader in CRISPR-based editing for advanced genetic medicine development [5]

Company Overview - Opus Genetics is a clinical stage biopharmaceutical company focused on developing gene therapies for inherited retinal diseases [2] - The company trades under the ticker symbol IRD and has a multi-asset pipeline [2] Pipeline and Milestones - Opus Genetics has near-term data readouts and multiple milestones expected in 2026 [2]

Core Viewpoint - Ultragenyx Pharmaceutical Inc. shares fell 42% following disappointing late-stage data for their rare bone disease program in collaboration with Mereo BioPharma Group plc [1] Group 1: Study Results - The Phase 3 Orbit and Cosmic studies for setrusumab (UX143) in Osteogenesis Imperfecta (OI) did not achieve statistical significance for the primary endpoints of reducing annualized clinical fracture rate compared to placebo or bisphosphonates [2] - Both studies met secondary endpoints, showing improvements in bone mineral density (BMD) against comparators, with no changes in the safety profile observed [2] Group 2: Analyst Reactions - Analysts from William Blair expressed disappointment and surprise at the results, especially given setrusumab's efficacy in the Phase 2 portion of the Orbit study and the increased enrollment of patients with severe disease subtypes in the Phase 3 study [3] - Analyst Sami Corwin is cautious about the future of setrusumab, noting several unknowns regarding the data, including potential effects in pediatric patients and the unexpectedly low annualized fracture rate in the placebo arm [4] Group 3: Market Impact and Future Outlook - Following the news, Ultragenyx is trading at a value reflective of its approved products, with William Blair maintaining an Outperform rating for the stock [4] - There is potential for upside in the next 12 months, particularly with the pivotal Angelman syndrome readout expected in Q3 2026 and the potential approval of two gene therapies eligible for priority review vouchers [5]

Core Insights - Opus Genetics, Inc. is a clinical-stage biopharmaceutical company focused on developing gene therapies aimed at restoring vision and preventing blindness in patients with inherited retinal diseases [3] Company Overview - The company is developing durable, one-time treatments that target the underlying genetic causes of severe retinal disorders [3] - Opus Genetics has a pipeline that includes seven AAV-based programs, with key candidates such as OPGx-LCA5 for LCA5-related mutations and OPGx-BEST1 for BEST1-related retinal degeneration [3] - Additional candidates in development target RHO, RDH12, and MERTK mutations [3] - The company is also advancing Phentolamine Ophthalmic Solution 0.75%, which is an approved therapy for pharmacologically induced mydriasis, with further indications in late-stage development for presbyopia and low-light visual disturbances following keratorefractive surgery [3] - Opus Genetics is headquartered in Research Triangle Park, NC [3] Upcoming Events - George Magrath, M.D., the CEO of Opus Genetics, will present at the J.P. Morgan 2026 Healthcare Conference on January 15, 2026, at 8:15 a.m. PT [1]

Core Insights - Opus Genetics, Inc. is a clinical-stage biopharmaceutical company focused on developing gene therapies aimed at restoring vision and preventing blindness in patients with inherited retinal diseases [3] Company Overview - The company is developing durable, one-time treatments that target the underlying genetic causes of severe retinal disorders [3] - Opus Genetics has a pipeline that includes seven AAV-based programs, with key candidates such as OPGx-LCA5 for LCA5-related mutations and OPGx-BEST1 for BEST1-related retinal degeneration [3] - Additional candidates in development target RHO, RDH12, and MERTK mutations [3] - The company is also advancing Phentolamine Ophthalmic Solution 0.75%, which is an approved therapy for pharmacologically induced mydriasis, with further indications in late-stage development for presbyopia and low-light visual disturbances following keratorefractive surgery [3] - Opus Genetics is headquartered in Research Triangle Park, NC [3] Upcoming Events - George Magrath, M.D., the CEO of Opus Genetics, will present at the J.P. Morgan 2026 Healthcare Conference on January 15, 2026, at 8:15 a.m. PT [1]

Core Viewpoint - Opus Genetics has announced a registered direct offering to raise approximately $23 million to fund its gene therapy clinical programs for inherited retinal diseases and other ophthalmic disorders [1][2]. Group 1: Financing Details - The financing was led by Perceptive Advisors and Balyasny Asset Management, with participation from Nantahala Capital [1]. - The offering includes the sale of 3,827,751 shares of common stock at a price of $2.09 per share and pre-funded warrants to purchase up to 7,177,033 shares at a purchase price of $2.0899 per warrant [3]. - The offering is expected to close on November 7, 2025, subject to customary closing conditions [3]. Group 2: Use of Proceeds - The net proceeds from the offering will be used to advance the LCA5 and BEST-1 gene therapy clinical programs, as well as for working capital and general corporate purposes [2]. - The company expects its cash resources to fund operations into the second half of 2027, excluding potential proceeds from callable warrants or future milestone payments [2]. Group 3: Company Overview - Opus Genetics is a clinical-stage biopharmaceutical company focused on developing gene therapies for inherited retinal diseases and small molecule therapies for other ophthalmic disorders [6]. - The company's pipeline includes AAV-based gene therapies targeting conditions such as Leber congenital amaurosis (LCA) and bestrophinopathy, with lead candidates OPGx-LCA5 and OPGx-BEST1 currently in clinical trials [6].

Core Insights - Eli Lilly and Company (LLY) is set to report its Q3 2025 earnings on October 30, with sales and earnings estimates at $16.01 billion and $6.02 per share respectively [1][7] - Earnings estimates for 2025 have decreased from $23.15 per share to $22.73 per share over the past month [1] Earnings Performance - The company's performance has been mixed, exceeding earnings expectations in two of the last four quarters while missing in the other two, resulting in an average earnings surprise of negative 2.31% [3] - In the last reported quarter, LLY achieved a positive earnings surprise of 12.48% [3] Earnings Expectations - LLY has an Earnings ESP of -0.66% and holds a Zacks Rank 3 (Hold) [4] - Companies with a positive Earnings ESP and a Zacks Rank of 1, 2, or 3 have a higher likelihood of beating earnings estimates [5] Key Growth Drivers - The primary drivers for LLY's top-line growth in Q3 are expected to be its GLP-1 drugs, Mounjaro and Zepbound, which have seen high demand [6][7] - Sales estimates for Mounjaro and Zepbound are $5.48 billion and $3.45 billion respectively, with LLY's own estimates at $5.33 billion and $3.46 billion [8] Competitive Landscape - CVS Caremark has excluded Zepbound from its preferred drug list, which negatively impacted prescriptions in July and is expected to affect growth in Q3 [9] - Competitive dynamics have also hurt sales of Trulicity, with patient switches to Mounjaro and lower realized prices affecting revenues [11] Product Performance - Sales estimates for other key drugs include Trulicity at $1.05 billion, Taltz at $919 million, Verzenio at $1.58 billion, Jardiance at $687 million, Olumiant at $263 million, and Emgality at $196 million [11] - Newer products like Ebglyss, Jaypirca, Kisunla, and Omvoh are anticipated to contribute to sales growth [12] Strategic Initiatives - LLY is diversifying beyond GLP-1 drugs into cardiovascular, oncology, and neuroscience areas, with several M&A deals announced in 2025 [25] - The acquisition of Adverum Biotechnologies aims to enhance LLY's pipeline with gene therapies for vision loss [26] Market Position - LLY's stock has increased by 6.9% this year, outperforming the industry average of 5.6% [14] - Despite facing pricing pressures and competition, LLY remains the largest drugmaker with strong growth prospects [30]

Core Insights - Opus Genetics, Inc. is a clinical-stage biopharmaceutical company focused on developing gene therapies for inherited retinal diseases (IRDs) and small molecule therapies for other ophthalmic disorders [3] Company Overview - The company is based in Research Triangle Park, NC, and its pipeline includes AAV-based gene therapies targeting conditions such as Leber congenital amaurosis (LCA), bestrophinopathy, and retinitis pigmentosa [3] - Opus Genetics' lead gene therapy candidates are OPGx-LCA5, currently in an ongoing Phase 1/2 trial for LCA5-related mutations, and OPGx-BEST1, targeting BEST1-related retinal degeneration [3] - The company is also advancing Phentolamine Ophthalmic Solution 0.75%, which is approved for one indication and is being studied in two Phase 3 programs for presbyopia and reduced low light vision [3] Upcoming Events - Ash Jayagopal, PhD, MBA, Chief Scientific and Development Officer, will present a corporate update at Chardan's 9th Annual Genetic Medicines Conference on October 21, 2025, at 12:00 p.m. ET [1]

Core Insights - Opus Genetics, Inc. is set to present positive clinical results from its gene therapy and Phentolamine Ophthalmic Solution programs at various medical and industry conferences in October 2025 [1][2] Company Overview - Opus Genetics is a clinical-stage biopharmaceutical company focused on developing gene therapies for inherited retinal diseases (IRDs) and small molecule therapies for other ophthalmic disorders [5] - The company's pipeline includes AAV-based gene therapies targeting conditions such as Leber congenital amaurosis (LCA), bestrophinopathy, and retinitis pigmentosa [5] - Lead gene therapy candidates include OPGx-LCA5, currently in a Phase 1/2 trial for LCA5-related mutations, and OPGx-BEST1 for BEST1-related retinal degeneration [5] - The company is also advancing Phentolamine Ophthalmic Solution 0.75%, which is approved for one indication and is being studied in two Phase 3 programs for presbyopia and visual disturbances [5] Conference Participation - The company will participate in several key conferences, including: - Cell and Gene Meeting on the Mesa - Fierce Biotech Week - American Academy of Optometry Annual Meeting 2025 - Eyecelerator @ American Academy of Ophthalmology (AAO) [3][4] Presentation Details - Presentation on OPGx-LCA5 will include three-month pediatric and 18-month adult clinical data from the Phase 1/2 trial for Leber congenital amaurosis type 5 [6] - A fireside chat titled "Investing with Intention – How Early Vision and Partnership Shaped Opus Genetics" will feature key executives from the company [6] - A poster presentation on the pivotal Phase 3 trial of Phentolamine Ophthalmic Solution will also take place [6]