Regeneron Pharmaceuticals (REGN) FY 2025 Annual General Meeting June 13, 2025 10:30 AM ET Speaker0 Good morning, and welcome to the twenty twenty five Annual Shareholder Meeting of Regeneron Pharmaceuticals. The meeting will begin after the following message. Speaker1 At Regeneron, we've always pursued bold science to drive our purpose, transforming lives through groundbreaking innovation. That principle is just as strong today as it was when the company was founded. Speaker2 We committed to not just treati ...

BURLINGTON, Mass., June 13, 2025 (GLOBE NEWSWIRE) -- Fractyl Health, Inc. (Nasdaq: GUTS) (the Company), a metabolic therapeutics company focused on pattern-breaking approaches to treat the root causes of obesity and type 2 diabetes (T2D), today announced it will present a poster of new preclinical data from its Rejuva Smart GLP-1 pancreatic gene therapy platform at the American Diabetes Association’s (ADA) 85th Scientific Sessions being held June 20-23, 2025, in Chicago, Illinois. Poster Presentation Detail ...

Pharvaris (PHVS) FY Conference June 11, 2025 04:00 PM ET Speaker0 All right. Good afternoon, and welcome to our, I think, the final session the conference. And it is my pleasure to introduce Maggie Beller, the head of IR for Verus. Maggie, welcome. I know this is the time I'm hosting you here. And before I go to the Q and A, let me just turn it to you for a quick introduction of the company, and maybe a brief overview about what's exciting ahead, and some of the catalysts and timing for that. Speaker1 Absol ...

Summary of 4D Molecular Therapeutics (FDMT) FY Conference Call Company Overview - **Company**: 4D Molecular Therapeutics (FDMT) - **Focus**: Gene therapy with a focus on ophthalmology and pulmonology, specifically targeting conditions like wet age-related macular degeneration (AMD), diabetic macular edema (DME), and cystic fibrosis. Key Points Industry and Market Opportunities - **Gene Therapy Potential**: The company believes it has developed best-in-class vectors for various delivery methods, which opens up large sustainable markets in gene therapy [1][2] - **Market Size**: The potential market opportunities are significant, with estimates of 4,150 million for wet AMD and DME, and 4,710 million for cystic fibrosis lung disease [1] Clinical Development and Trials - **Phase III Trials**: The focus for the next twelve months is on executing Phase III trials for FORWARD I and FORWARD II in wet AMD and initial dose data for cystic fibrosis [2] - **Physician Feedback**: Positive feedback from physicians regarding the durability of treatments and reduced injection burden, with expectations of an 80-90% reduction compared to existing therapies [6][7] - **Safety Profile**: The company reports a strong safety profile with no inflammation observed in DME trials, which is critical for large markets like wet AMD and DME [9][10] Commercial Strategy - **Pricing Flexibility**: The cost of goods for treatments is around $500, allowing for competitive pricing strategies that can benefit patients and shareholders [26] - **Durability Demand**: Surveys indicate that durability and extended treatment duration are the top desires for both patients and physicians, positioning gene therapy as a leading option [22] Regulatory and Financial Position - **Regulatory Engagement**: Ongoing discussions with the FDA regarding potential accelerated approval pathways for cystic fibrosis treatments, especially for patients with no available therapies [41][43] - **Financial Health**: The company ended Q1 with $458 million in cash, primarily allocated to fund Phase III trials for wet AMD, with a runway extending into 2028 [51] Future Directions - **Partnership Opportunities**: The company is open to partnerships outside the U.S. for its products, particularly in the lung franchise, while retaining U.S. rights for ophthalmology [48][49] - **Platform Development**: The gene therapy platform has shown broad utility, with six open INDs and a focus on developing a portfolio of products efficiently [45][54] Additional Insights - **Market Differentiation**: The company emphasizes that not all gene therapies are the same, highlighting its unique approach to local application and cost management, which differentiates it from other programs in the field [56] This summary encapsulates the key insights and strategic directions discussed during the conference call, providing a comprehensive overview of 4D Molecular Therapeutics' current status and future outlook in the gene therapy market.

-- PCRX-201 demonstrated sustained clinical efficacy with improvements in pain, stiffness, and function for up to three years – BRISBANE, Calif., June 11, 2025 (GLOBE NEWSWIRE) -- Pacira BioSciences, Inc. (NASDAQ: PCRX), the industry leader in the delivery of innovative, non-opioid pain therapies to transform the lives of patients, today announced new long-term follow-up data from its Phase 1 clinical trial evaluating PCRX-201 (enekinragene inzadenovec), a novel gene therapy candidate for osteoarthritis of ...

~ Proven biotech executive to lead commercialization of AMT-130 in Huntington’s disease ~ LEXINGTON, Mass. and AMSTERDAM, June 11, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the appointment of Kylie O’Keefe as Chief Customer and Strategy Officer, effective June 6, 2025. In this role, Ms. O’Keefe will lead the development and execution of uniQure’s global commercialization stra ...

-Ends- Enquiries: OXB: Sophia Bolhassan, Head of Investor Relations – T: +44 (0) 1865 509 737 / E: ir@oxb.com Oxford, UK – 11 June 2025: OXB (LSE: OXB) ("the Company"), a global quality and innovation-led cell and gene therapy CDMO, provides an update to coincide with its Annual General Meeting being held at 3pm BST today at Windrush Court, Oxford, UK. OXB has demonstrated strong commercial momentum and made good operational and commercial progress in the year 2025 to date, as it continues to provide viral ...

Summary of Ultragenyx Pharmaceutical (RARE) FY Conference Call Company Overview - Ultragenyx Pharmaceutical is at a significant inflection point, achieving revenues between $640 million to $670 million from four products: Crysvita, Nepsevi, Zolcivi, and Evkesa [2][4] - The company is experiencing a global commercial growth rate of approximately 20% annually [2] - Six late-stage programs are in development, with three expected to be filed within the year [4] Pipeline and Product Development - Key late-stage programs include: - Gene therapy for MPS IIIA, currently under review [2] - Gene therapy for GSD I, expected to be filed soon [2] - Osteogenesis Imperfecta (OI) program with high confidence in its transformative potential [5][6] - Angelman syndrome program (ASPIRE study) is on track to complete enrollment by the end of the year [49] - The company aims to become profitable by 2027, leveraging its existing products and new launches [4][68] Clinical Trials and Expectations - The OI program (cetrusumab) shows promising data from phase two trials, with a 67% reduction in fractures [6][19] - A significant threshold for commercial success is expected to be a fracture reduction of over 40%, with hopes for results above 50% [20][21] - The COSMIC trial aims to demonstrate the superiority of cetrusumab over bisphosphonates, which is crucial for market positioning [29][33] Market Strategy and Commercialization - The company plans to focus on key opinion leaders and centers with a high patient volume for initial launches [46] - A field force of 40 to 50 personnel is expected to support the launch, with an emphasis on home infusion models [42][48] - The commercial launch is anticipated to be easier due to a concentrated patient population in clinics [25] Gene Therapy Opportunities - The MPS IIIA program targets a severe disease with a small patient population but high urgency for treatment [59][60] - GSD I has a larger patient population, estimated at 1,500 to 8,000 in the US, with a significant need for treatment [63] - The company believes that urgency and the severity of conditions will drive adoption of gene therapies [66] Financial Outlook - The company reiterated its 2025 revenue guidance, projecting a 14% to 20% year-over-year growth, primarily driven by existing products [67][68] - Approximately 85% of revenue is expected to come from current products, with new launches contributing a smaller portion initially [68][69] Additional Insights - The company emphasizes the importance of patient quality of life and how treatments improve their daily activities, which will be a key driver for adoption [22][24] - Secondary endpoints in clinical trials are considered important for understanding the broader impact of treatments on patients [26][28] This summary encapsulates the key points discussed during the Ultragenyx Pharmaceutical FY Conference Call, highlighting the company's current status, pipeline developments, market strategies, and financial outlook.

Sana Biotechnology (SANA) FY Conference June 10, 2025 08:40 AM ET Speaker0 Great. Good morning, everyone. Thank you so much for joining us. Really pleased to have with us this morning Doctor. Steve Haar, who's the President and CEO of Sana Therapeutics. I'm Salveen Richter. I cover the biotechnology sector. Steve, before we jump into the programs here, perhaps level set us with where the company stands today regarding the portfolio strategy and key priorities and walk us through what we should look to from ...

Lexeo Therapeutics (LXEO) Conference June 05, 2025 02:00 PM ET Speaker0 Available to clients in this call. If the company is presenting, any recording may also be posted on their website. Usually, the opinion discussed by any experts speaking on this call are those who are speaking and not to check for Morgan. After this conference call, we will also be and participants now. I would now like to turn the call over to Tess Romero to begin. Please go ahead when you're ready. Speaker1 Thank you, operator, and t ...