Accessibility StatementSkip Navigation INDIANAPOLIS, Nov. 18, 2025 /PRNewswire/ --Â Eli Lilly and Company (NYSE:LLY) will participate in the Citi's 2025 Global Healthcare Conference on December 2, 2025. Ilya Yuffa, executive vice president and president Lilly USA and Global Customer Capabilities, will take part in a fireside chat at 10:30 a.m., Eastern time. A live audio webcast will be available on the "Webcasts & Presentations" section of Lilly's investor website at https://investor.lilly.com/webcasts-and ...

Core Insights - 4D Molecular Therapeutics reported significant progress in Q3 2025, focusing on its lead product candidates 4D-150 and 4D-710, with a strong cash position extending operations into the second half of 2028 [2][5][9] Financial Performance - Cash, cash equivalents, and marketable securities totaled $372 million as of September 30, 2025, down from $505 million at the end of 2024, primarily due to operational cash usage [9][20] - Research and development expenses increased to $49.4 million in Q3 2025 from $38.5 million in Q3 2024, driven by Phase 3 trials for 4D-150 [10][18] - General and administrative expenses decreased to $11.8 million in Q3 2025 from $12.7 million in Q3 2024, attributed to reduced headcount [10][18] - The net loss for Q3 2025 was $56.9 million, compared to a net loss of $43.8 million in Q3 2024 [12][18] Strategic Partnerships and Investments - The company entered a strategic partnership with Otsuka Pharmaceutical, receiving an upfront payment of $85 million and expecting at least $50 million in cost-sharing over three years [5][6] - An equity investment of up to $11 million from the Cystic Fibrosis Foundation was announced to support the development of 4D-710 [5][11] Clinical Development Highlights - Positive long-term safety and efficacy data for 4D-150 in wet age-related macular degeneration (AMD) were reported from the Phase 1/2 PRISM trial, showing durable benefits over 1.5 to 2 years [5][11] - Enrollment for the 4FRONT Phase 3 trials in wet AMD is on track, with over 200 patients randomized and expected completion in Q1 2026 [11] - The 4D-710 program is advancing towards Phase 2, with interim safety and efficacy data expected by the end of 2025 [11] Leadership and Organizational Changes - The leadership team was strengthened with the promotion of Julie Clark, M.D., to Chief Medical Officer, and the addition of Liansheng Zhu, Ph.D., as SVP of Biometrics and Data Quality [6][11]

Core Insights - The FDA is open to pooling data from ongoing Phase I/II studies of LX2006 with data from a pivotal trial to support a Biologics License Application (BLA) [1][3] - Interim clinical data for LX2006 show significant improvements in cardiac and neurologic measures for Friedreich ataxia, including an 18% mean improvement in left ventricular mass index (LVMI) at 6 months and a 23% improvement at 12 months [1][3] - Lexeo Therapeutics completed a $154 million equity financing to support LX2006 registrational activities and further development of its cardiac pipeline, with cash expected to fund operations into 2028 [1][5][7] Regulatory Progress - The FDA approved a comparability report between LX2006 HEK and Sf9 manufacturing processes, endorsing the use of the Sf9 process for commercial manufacturing [1][7] - Lexeo plans to submit enhanced manufacturing comparability data and additional nonclinical data to support the BLA submission [3] Clinical Data - Interim clinical data from LX2006 show a 2.0-point mean improvement in the modified Friedreich Ataxia Rating Scale (mFARS) across all participants with over 6 months of follow-up [3] - In the HEROIC-PKP2 Phase I/II trial, ten participants have been dosed, with interim data from the low-dose cohort reported and additional data expected in January 2026 [1][4] Financial Results - For Q3 2025, Lexeo reported a net loss of $20.3 million, or $0.33 per share, compared to a net loss of $29.5 million, or $0.89 per share, in Q3 2024 [5][10] - Research and Development expenses decreased to $15.7 million in Q3 2025 from $23.4 million in Q3 2024 [5][9] Cash Position - As of September 30, 2025, Lexeo had cash, cash equivalents, and investments totaling $122.8 million, which, along with the recent financing, is expected to fund operations into 2028 [5][11]

Core Insights - BridgeBio Pharma, Inc. is a biopharmaceutical company focused on developing transformative medicines for genetic diseases [2] - The company will participate in healthcare investor conferences, hosting fireside chats to engage with investors [1][3] Company Overview - Founded in 2015, BridgeBio aims to discover, create, test, and deliver medicines for patients with genetic diseases [2] - The company's pipeline includes programs from early science to advanced clinical trials, showcasing its commitment to genetic medicine [2] Upcoming Events - BridgeBio will host a fireside chat at the UBS Global Healthcare Conference on November 10 at 1:15 pm EST [3] - The company will also participate in the Jefferies Global Healthcare Conference on November 18 at 11:00 am EST [3]

Core Insights - 4D Molecular Therapeutics Inc. has secured up to $11 million in additional funding from the Cystic Fibrosis Foundation to expedite the development of its genetic medicine, 4D-710, for cystic fibrosis [1][3] - The funding commitment includes an initial tranche of $7.5 million, bringing the total funding from the CF Foundation to approximately $32 million for 4DMT's cystic fibrosis programs [2][3] - 4D-710 has received both Rare Pediatric Disease Designation and Orphan Drug Designation from the FDA, highlighting its significance in treating cystic fibrosis, a disease affecting around 40,000 people in the US and 105,000 globally [3][4] Company Overview - 4D Molecular Therapeutics is a late-stage biotechnology company focused on developing adeno-associated virus vectors through its proprietary platform, Therapeutic Vector Evolution, based in the Netherlands and the US [4]

Summary of Avidity Biosciences FY Conference Call Industry Overview - The focus of the conference was on muscular dystrophies, particularly Duchenne Muscular Dystrophy (DMD) and Facioscapulohumeral Muscular Dystrophy (FSHD) [1][24] - DMD is a multibillion-dollar market projected to grow to $3 billion by 2033 [1] - Other muscular dystrophies like DM1 and FSHD currently lack approved therapies, but promising treatments are in development [1] Key Points on DMD Treatments - Current DMD treatments include first-generation PMO exon-skipping therapies, new gene therapies, and steroids, but significant unmet needs remain, especially for non-ambulatory patients [2][3] - The FDA's approval of the first PMO exon-skipping therapy is seen as a watershed moment that opened investment in the DMD space [3][4] - The approval set a low regulatory bar, leading to increased investment and development of new therapies [3][5] - New technologies, such as Antibody Oligonucleotide Conjugates (AOCs), are being developed to improve dystrophin production and functional benefits [9][10] Clinical Data and Efficacy - AOCs have shown promising results, with 40% exon skipping and a 25% increase in dystrophin levels [10][11] - Creatine kinase (CK) levels, a measure of muscle damage, have returned to normal for many patients, indicating functional improvement [11][14] - The FDA's low bar for approval has allowed for a variety of therapies to enter the market, but safety and efficacy remain critical [16][19] Gene Therapy Insights - Sarepta Therapeutics' Elevidys gene therapy faced scrutiny but initially showed strong uptake due to the unmet need in DMD [16] - Avidity's approach focuses on optimizing the gene therapy construct and manufacturing processes to improve safety and efficacy [18][19] - The company has developed a microdystrophin that closely resembles natural dystrophin, aiming for higher expression and longer half-life [17][18] FSHD Developments - FSHD is characterized by the aberrant expression of the DUX4 transcription factor, which is toxic to muscle cells [26][27] - Avidity is using AOC technology to deliver silencing RNA to inhibit DUX4 expression, showing early signs of clinical benefit [27][30] - The identification of a circulating biomarker (C-DUX) for DUX4 may facilitate accelerated approval processes [29][32] Market Dynamics and Future Outlook - The DMD market is expected to see multiple effective therapies, with opportunities for various players due to the large unmet need [41][42] - The potential for combination therapies is highlighted, with patients and physicians open to using multiple modalities for better outcomes [47][48] - The FDA's accelerated approval pathway is anticipated to remain in place to encourage ongoing innovation in the muscular dystrophy space [49] Conclusion - The conference highlighted the significant advancements in genetic medicine for muscular dystrophies, the importance of regulatory pathways, and the potential for new therapies to address unmet needs in DMD and FSHD [1][41][49]

Core Viewpoint - Lexeo Therapeutics, Inc. has announced a public offering and concurrent private placement aimed at raising approximately $135 million to support its clinical stage genetic medicine initiatives for cardiovascular diseases [1][4]. Group 1: Public Offering Details - Lexeo is offering 15,625,000 shares of common stock at a price of $8.00 per share in the public offering [2]. - The underwriters have a 30-day option to purchase an additional 2,343,750 shares at the public offering price [2]. - The public offering is expected to close on or about October 20, 2025, subject to customary closing conditions [4]. Group 2: Private Placement Details - Concurrently, Lexeo is selling pre-funded warrants to purchase 1,250,015 shares of common stock to Balyasny Asset Management at a price of $7.9999 per warrant [3]. - This private placement is exempt from the registration requirements of the Securities Act and is not contingent on the public offering [3][6]. Group 3: Company Overview - Lexeo Therapeutics is focused on pioneering treatments for cardiovascular diseases through genetic medicine [8]. - The company is advancing a portfolio of therapeutic candidates targeting genetic causes of conditions such as Friedreich ataxia cardiomyopathy and plakophilin-2 arrhythmogenic cardiomyopathy [8].

Core Insights - 4D Molecular Therapeutics has received up to $11 million in additional funding from the Cystic Fibrosis Foundation to accelerate the development of 4D-710 for cystic fibrosis lung disease [1][3][5] - The funding includes an initial tranche of $7.5 million and aims to enhance the strategic planning and coordination of 4D-710's development through a newly formed Joint Steering Committee [1][4][5] - The ongoing AEROW clinical trial is assessing the impact of 4D-710 on lung health, with enrollment in Phase 2 currently underway [5][6][8] Funding and Development Support - The CF Foundation's investment will be provided in two tranches, with the second tranche contingent on meeting specific clinical milestones [3][5] - The funding will support the advancement of 4D-710 into Phase 2 and prepare for Phase 3 readiness, including a second dosing expected to occur at least one year after the initial dose [5][6] - A Joint Steering Committee will be established to provide guidance and coordination for the clinical and operational progress of 4D-710 [4][5] Clinical Trial Details - The AEROW clinical trial includes a Phase 1 Redosing Cohort where selected participants will receive a second dose of 2.5E14 vg [6] - The trial has been amended to add additional clinical endpoints, including multiple-breath washout and high-resolution computed tomography to assess lung function and structure [6] - Interim Phase 1 data and program updates are expected to be shared by the end of 2025 [4][5] About Cystic Fibrosis and 4D-710 - Cystic fibrosis is a progressive genetic disease affecting nearly 40,000 people in the U.S. and over 105,000 globally, leading to severe lung function impairment [7] - 4D-710 is designed to be a durable, redosable, and variant-agnostic genetic medicine aimed at improving airway function and quality of life for individuals with cystic fibrosis [8] - The product has received Rare Pediatric Disease Designation and Orphan Drug Designation from the U.S. FDA, highlighting its potential significance in treating CF [8]

Group 1 - Chardan Capital sets a price target of $17 for Lexeo Therapeutics, indicating a potential increase of about 94% from its current trading price of $8.25 [1][6] - Lexeo is advancing with the FDA on an Accelerated Approval pathway for its treatment LX2006, which targets Friedreich ataxia cardiomyopathy [2][6] - Interim clinical data shows promising results, with an 18% mean reduction in left ventricular mass index (LVMI) at six months and a 23% mean reduction at twelve months, exceeding the FDA's target threshold of a 10% reduction [3][6] Group 2 - There is a clinically meaningful improvement in the modified Friedreich Ataxia Rating Scale (mFARS), indicating slowed disease progression and improved function, highlighting the potential of LX2006 as a life-changing therapy [4] - Currently, Lexeo's stock is trading at $8.34, reflecting a 28.90% increase, with a market capitalization of approximately $276.86 million and a trading volume of 9.42 million shares [5]

Core Viewpoint - The FDA has approved a label update for VYJUVEK, expanding its eligible patient population to include dystrophic epidermolysis bullosa (DEB) patients from birth, allowing for greater flexibility in application and wound management [2][3]. Group 1: Product and Application - VYJUVEK is a non-invasive, topical, redosable genetic medicine designed to deliver two copies of the COL7A1 gene directly to DEB wounds, addressing the disease-causing mechanism at the molecular level [5]. - The updated label allows DEB patients and caregivers to apply VYJUVEK at home, integrating it into daily routines and enhancing convenience [3][4]. - Wound dressings can now be removed during the next dressing change rather than waiting 24 hours, further streamlining the wound care process [3]. Group 2: Company and Market Position - Krystal Biotech believes these updates reinforce VYJUVEK's leadership position as a flexible and convenient treatment for DEB patients in the U.S. [3]. - The label update is based on real-world data and results from an open-label extension study, supporting the long-term safety and efficacy of VYJUVEK across all ages [4]. - Krystal Biotech is a fully integrated biotechnology company focused on developing genetic medicines for diseases with high unmet medical needs, with VYJUVEK being its first commercial product [12].