Investor call to be held January 8 at 4:30 pm ET to discuss data update and next stepsPITTSBURGH, Jan. 07, 2026 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS) today announced that it will host an investor conference call and webcast tomorrow, Thursday, January 8, 2026, at 4:30 pm ET, to disclose an interim clinical update from CORAL-1, the Company’s multi-center, dose escalation Phase 1 study evaluating KB407 in patients with cystic fibrosis. The interim clinical update will focus ...

TARRYTOWN, N.Y., Dec. 22, 2025 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) will webcast its presentation at the 44th Annual J.P. Morgan Healthcare Conference on Monday, January 12, 2026. The presentation is scheduled for 2:15 p.m. Pacific Time (5:15 p.m. Eastern Time) and may be accessed from the "Investors & Media" page of Regeneron's website at http://investor.regeneron.com/events-and-presentations. A replay and transcript of the webcast will be archived on the Company's website for ...

Clinically meaningful lung function activity, measured by ppFEV1 and LCI2.5, with follow-up through 1 year at dose selected for Phase 2 Durable CFTR transgene expression within target therapeutic range with follow-up through at least 1 year Data support 4D-710’s potential to be a durable, redosable, variant-agnostic, disease-modifying treatment for people with cystic fibrosis lung disease with high unmet needWebcast today at 8:00 a.m. ET with distinguished cystic fibrosis KOLs EMERYVILLE, Calif., Dec. 17, 2 ...

Summary of Krystal Biotech FY Conference Call Company Overview - **Company**: Krystal Biotech (NasdaqGS:KRYS) - **Product**: Vyjuvek, the first genetic medicine that can be dosed at home by patients or caregivers [2][3] Key Points Product Launch and Market Position - Vyjuvek has been launched in the U.S. for nearly two years and recently in France, Germany, and Japan [2] - The company has signed distribution agreements, indicating a strong global market entry strategy [2] - Positive experiences reported by patients and physicians regarding safety and efficacy in the U.S. [2] Manufacturing and Financial Position - Krystal maintains control over manufacturing, shipping, and packaging processes, which is expected to benefit future clinical pipelines [3] - The company has reported positive EPS for the last eight to nine quarters, indicating a strong financial position [3] Compliance and Growth Expectations - Compliance is expected to reach a steady state, but it may take longer than initially anticipated [4][5] - Annual vial consumption per patient is estimated to be between 26 and 30 vials, with an average price per patient projected at $450,000-$500,000 [8][9] - Targeting 1,200 identified patients in the U.S., with current patient count at approximately 615 [9] International Market Insights - Pricing in Japan is close to U.S. net pricing, with a 23% WAC discount compared to the U.S. [10][11] - The company believes that ex-U.S. sales could potentially exceed U.S. sales, with expectations of higher revenue percentages from Japan [16] Pipeline Developments - Ocular DEB study is ongoing with a controlled, blinded design, focusing on severe patients to increase chances of success [21][22] - The market for ocular DEB is estimated to be larger than the 25% of the RDEB population previously thought [36] - Pricing for ocular DEB will be determined post pivotal study outcomes [40] Future Studies and Regulatory Considerations - The NK side study is currently enrolling patients, with a focus on maintaining a clean safety profile [41][52] - Discussions with the FDA have been ongoing regarding safety data and study design [51][60] - Upcoming readouts for CF and AATD studies are anticipated, with a focus on moving quickly into redosing studies if data is positive [60] Unmet Medical Needs - Hailey-Hailey disease is highlighted as a significant unmet need with no current therapies available, representing a potential market opportunity [62] Additional Insights - The company is optimistic about the potential for growth in international markets and the success of its pipeline programs, particularly in areas with high unmet medical needs [16][60][62]

Core Insights - Regeneron Pharmaceuticals and Tessera Therapeutics have announced a global collaboration to develop and commercialize TSRA-196, a gene therapy for alpha-1 antitrypsin deficiency (AATD), which affects approximately 200,000 people in the U.S. and Europe [1][5][6] - TSRA-196 aims to correct the genetic mutation causing AATD, providing a one-time treatment option to restore functional alpha-1 antitrypsin protein production [1][6] - Tessera plans to file an Investigational New Drug application and multiple Clinical Trial Applications with the FDA by the end of the year [1][6] Collaboration Details - The collaboration will involve shared worldwide development costs and potential future profits, with both companies splitting these equally [2] - Tessera will receive $150 million from Regeneron, which includes an upfront cash payment and equity investment, along with potential milestone payments totaling $125 million [2][6] - Tessera will lead the initial first-in-human trial, while Regeneron will oversee subsequent global development and commercialization efforts [2] Scientific Background - AATD is caused by mutations in the SERPINA1 gene, leading to insufficient alpha-1 antitrypsin protein, which protects lung tissue from damage [5][7] - Current treatment options are limited, primarily consisting of weekly intravenous augmentation therapy for patients with lung disease [7] - Preclinical data for TSRA-196 has shown durable and high-fidelity genome editing in animal models, indicating its potential effectiveness and safety [3][6] Company Profiles - Regeneron is a leading biotechnology company focused on developing life-transforming medicines for serious diseases, leveraging its expertise in genetics and clinical development [8][9] - Tessera Therapeutics specializes in genome engineering through its Gene Writing platform, aiming to create transformative genetic medicines [11]

Core Insights - Eli Lilly and Company will participate in Citi's 2025 Global Healthcare Conference on December 2, 2025, with Ilya Yuffa, executive vice president and president of Lilly USA, engaging in a fireside chat [1] - A live audio webcast of the presentation will be available on Lilly's investor website, with a replay accessible for approximately 90 days [2] - Lilly has been a pioneer in medicine for nearly 150 years, focusing on significant health challenges such as diabetes care, obesity treatment, Alzheimer's disease, immune system disorders, and difficult-to-treat cancers [3] Company Developments - Eli Lilly announced the appointment of two new Executive Committee members and the expansion of leadership roles to prepare for future growth [5][6]

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]