SOUTH SAN FRANCISCO, Calif., Oct. 09, 2025 (GLOBE NEWSWIRE) -- Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a clinical-stage biotechnology company with a mission to discover, develop and deliver potentially curative therapies that address the underlying causes of heart disease, today announced that Faraz Ali, Tenaya’s Chief Executive Officer, will participate in a fireside chat at the H.C. Wainwright Genetic Medicines Virtual Conference on Tuesday, October 14, 2025. Company management will host virtual 1x1 mee ...

MeiraGTx Holdings plc (NASDAQ: MGTX ) is a genetic medicines biotech with a late-stage pipeline and capabilities for end-to-end GMP manufacturing. Its main drug candidates are AAV-AIPL1 for congenital blindness, which is working toward exceptional-circumstances filings and has a plannedMy name is Myriam Hernandez Alvarez. I received the Electronics and Telecommunication Engineering degree from the Escuela Politecnica Nacional, Quito, Ecuador, the M.Sc. degree in computer science from Ohio University, Athens ...

MeiraGTx Holdings plc (NASDAQ: MGTX ) is a genetic medicines biotech with a late-stage pipeline and capabilities for end-to-end GMP manufacturing. Its main drug candidates are AAV-AIPL1 for congenital blindness, which is working toward exceptional-circumstances filings and has a plannedMy name is Myriam Hernandez Alvarez. I received the Electronics and Telecommunication Engineering degree from the Escuela Politecnica Nacional, Quito, Ecuador, the M.Sc. degree in computer science from Ohio University, Athens ...

Summary of Dyne Therapeutics (DYN) 2025 Conference Call Company Overview - **Company**: Dyne Therapeutics - **Focus**: Development of genetic medicines for neuromuscular diseases, specifically targeting DM1 (Myotonic Dystrophy Type 1) and DMD (Duchenne Muscular Dystrophy) [2][3] Key Milestones and Financials - **Upcoming Milestones**: - Top-line data for DMD by the end of 2025 - Top-line data for DM1 following DMD data - Filing of two Biologics License Applications (BLAs) [6][7] - Commercial launch of DMD expected in early 2027 [6] - **Financial Position**: - Pro forma cash of $900 million as of June 2025, expected to last into Q3 2027 [7] Product Development and Platform - **Platform**: - Utilizes an antibody fragment targeting the transferrin receptor for efficient delivery of genetic medicines to muscle and CNS [9][10] - Demonstrated ability to achieve functional improvement in DM1 and DMD within three to six months [10][11] - **Clinical Data**: - Preclinical studies showed significant dystrophin expression improvements compared to monoclonal antibody approaches, with 15x expression in cardiac tissue and 2.5x in quadriceps [13] - Clinical trials indicate positive patient-reported outcomes related to CNS effects [14][15] DMD Program (DYNE-251) - **Market Opportunity**: - Targets approximately 1,600 patients in the U.S. with exon 51 mutations, which represent a significant unmet need [17][19] - Current standard of care (Eteplisin) has limited efficacy and is burdensome for patients [18] - **Product Profile**: - DYNE-251 is a 20 mg/kg monthly dose, expected to show functional improvement and significant distance gains [21] - Clinical data presented showed 8.7% dystrophin levels at six months and various functional improvements [23][24] - **Regulatory Path**: - Plans to submit for accelerated approval based on dystrophin levels and functional improvements [25][32] DM1 Program (DYNE-101) - **Regulatory Strategy**: - Seeking accelerated approval based on VHOT as an intermediate clinical endpoint, with a registrational cohort of 60 patients [37][38] - Shifted focus from splicing correction to VHOT due to FDA feedback [39][40] - **Clinical Development**: - Ongoing multi-sending dose study with 56 patients, aiming for a phase three confirmatory trial in Q1 2026 [38] Competitive Landscape and Future Outlook - **Market Positioning**: - Positioned to compete effectively in the DMD and DM1 markets with a focus on functional improvements and CNS delivery [45] - **Pipeline Expansion**: - Plans to leverage the platform for additional exons and other neuromuscular diseases like FSHD and Pompe [46][47] - **Commercial Preparation**: - Building a commercial team with experience in rare diseases, preparing for the U.S. launch [35][36] Additional Insights - **Patient Engagement**: - Positive feedback from the DMD community regarding functional improvements observed in clinical trials [27] - **Regulatory Challenges**: - Acknowledgment of the complexities in navigating FDA requirements for novel endpoints [41][42] This summary encapsulates the key points discussed during the Dyne Therapeutics conference call, highlighting the company's strategic direction, product development, and market opportunities.

Core Viewpoint - Krystal Biotech, Inc. is advancing the development of KB707, a redosable immunotherapy targeting non-small cell lung cancer (NSCLC), with promising early efficacy data leading to an End of Phase 2 meeting with the FDA to discuss registration pathways [1][2][3] Group 1: Development Plans and Efficacy - The company has observed a 36% objective response rate in heavily pre-treated NSCLC patients as of April 15, 2025, with no Grade 4 or 5 adverse events reported, indicating a favorable safety profile [3] - Enrollment is ongoing in the KYANITE-1 study, a Phase 1/2 trial evaluating inhaled KB707 in patients with locally advanced or metastatic lung tumors [4] - The company has paused enrollment in the OPAL-1 study, which evaluates intratumoral KB707, to prioritize inhaled KB707, while continuing to monitor patients in OPAL-1 [5] Group 2: Company Overview - Krystal Biotech is a commercial-stage biotechnology company focused on genetic medicines for diseases with high unmet medical needs, with its first product, VYJUVEK, being the first redosable gene therapy approved in multiple regions [6]

Financial Performance & VYJUVEK Launch - VYJUVEK revenue since launch exceeds $525 million[8] - Second quarter 2025 product revenue reached $96 million, compared to $70.3 million in the second quarter of 2024[43] - Gross margin in the second quarter was 93%[10, 43] - Gross to net in the second quarter was 17%[10] VYJUVEK Expansion & Approvals - VYJUVEK received approval in Japan for DEB patients from birth, with launch expected in the fourth quarter of 2025[11, 14] - Over 575 reimbursement approvals in the U S[10] - U S compliance to weekly therapy while on drug at 82% as of end of 2Q 2025[10] - European launch is on track for later this quarter, with over 575 identified DEB patients in Germany and France[15, 16] Pipeline Development - 36% objective response rate was observed with KB707 monotherapy in advanced NSCLC patients[22] - 40% AAT positive cells after KB408[26] - Functional AAT delivery to the lungs was confirmed in the third patient dosed with KB408, showing a 79.3% reduction in free neutrophil elastase in ELF after a single dose[25, 26] Financial Guidance - Non-GAAP R&D and SG&A expense guidance for the full year 2025 is $150 million to $175 million[44]

PITTSBURGH, July 29, 2025 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the "Company") (NASDAQ: KRYS), a commercial-stage biotechnology company, today announced that it will report its second quarter 2025 financial results on Monday, August 4, 2025, prior to the open of U.S. markets. CONTACT Investors and Media: Stéphane Paquette, PhD Krystal Biotech spaquette@krystalbio.com The Company's management will also host a conference call and webcast at 8:30 am ET on Monday, August 4, 2025, to discuss the financial r ...

Neurotrophic Keratitis (NK) Market & KB801 Opportunity - The number of patients in the U S with at least one NK claim has increased by over 115% from 31,000 in 2020 to an estimated 68,000 in 2024[9, 10] - In 2023, U S Medicaid and Medicare spent over $540 million on Oxervate, the only FDA-approved therapy for NK[12] - An estimated over 410,000 days of Oxervate therapy were reimbursed in the U S in 2024[12] - KB801 is designed to address the shortcomings of Oxervate, aiming for superior NGF exposure with significantly reduced dosing frequency[11, 21] KB801 Preclinical Data - KB801 transduces primary human corneal epithelial cells in vitro, leading to dose-dependent expression and secretion of mature NGF[25, 26, 29] - In vitro studies confirmed functionality of secreted NGF using a growth factor starved cell proliferation assay[25, 30] - Topical administration of KB801 to wounded murine corneas was well-tolerated and resulted in localized NGF expression[25] - Head-to-head mouse PK studies demonstrated a clear durability advantage with KB801 compared to recombinant NGF, even against intensive recombinant dosing[43, 47] KB801 Clinical Development - The EMERALD-1 study is a Phase 1/2 double-masked, 2:1 randomized, placebo-controlled study in patients with moderate-to-severe NK, with the first patient dosed in July 2025[51, 53, 1] - Krystal is pursuing Platform Technology Designation with the FDA, potentially leading to expedited development of KB801[56, 57]

Company Overview - Tenaya Therapeutics Inc. is a clinical-stage biotechnology company focused on discovering, developing, and delivering potentially curative therapies for heart disease [3] - The company employs integrated capabilities such as target validation, capsid engineering, and manufacturing to create genetic medicines for both rare genetic disorders and common heart conditions [3] Recent Developments - On May 15, 2025, Tenaya granted stock options to purchase a total of 461,000 shares of common stock to three new non-executive employees as part of their employment commencement [1] - The stock options have an exercise price of $0.4373 per share, equal to the closing price on the grant date, and will vest over four years [1][2] Product Pipeline - Tenaya's pipeline includes: - TN-201: A gene therapy for MYBPC3-associated hypertrophic cardiomyopathy (HCM) - TN-401: A gene therapy for PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC) - TN-301: A small molecule HDAC6 inhibitor for heart failure with preserved ejection fraction (HFpEF) - Multiple early-stage programs in preclinical development [3]

Summary of Krystal Biotech (KRYS) Conference Call Company Overview - **Company Name**: Krystal Biotech - **Industry**: Biotechnology - **Focus**: Development and commercialization of genetic medicines for monogenic diseases - **Location**: Pittsburgh, with all manufacturing and intellectual property based in the US [4][5] Core Points and Arguments Commercial Stage and Pipeline - Krystal Biotech launched its drug, **BIJUVAK**, for dystrophic epidermolysis bullosa (DEB) in the US about 18 months ago and received approval in Europe, with plans to launch in Europe and Japan later in 2025 [4][5] - The company has been cash flow positive for the last seven quarters and does not plan to seek financing in the next few years [5][6] - The focus for 2025 includes expanding into lung and eye tissues, with early entry into oncology [5] Market Trends and Patient Dynamics - In Q1, patient healing led to temporary pauses in treatment, which is expected to result in a return of patients over the next 60-120 days [8][9] - The company has 17 sales representatives covering 52 states, facing challenges in prescription pull-through due to patient dispersion [10][11] - Insurance changes in Q1 impacted revenue but are expected to normalize in Q2 [12][13] European and Japanese Market Launch - The EU approval allows broader patient self-administration compared to the US, with launches expected in Germany and France in Q3 2025 [19][20] - Pricing in the EU is anticipated to be around 50-75% of the US price, with a conservative accrual strategy [27][28] Pipeline Developments - Upcoming focus on cystic fibrosis (CF) and alpha-one antitrypsin deficiency treatments, with strong value propositions for patients with null mutations [34][35] - The company is preparing for a lung cancer announcement at ASCO, reinforcing confidence in its lung delivery capabilities [37][38] Ophthalmology Programs - The company is initiating a registrational study for KBM-803 targeting ocular complications of DEB, with a market opportunity for about 10-15% of the RDEB population having eye lesions [48][49] - Another program, KB801, targets neurotropic keratitis, expected to be a significant market opportunity but not anticipated for launch until 2026 [50][51] Financial Outlook - The company is confident in its financial position, with plans to manage the launch of BIJUVAK and advance other pipeline assets without needing additional funding [53][55] Other Important Insights - The company emphasizes the importance of patient comfort and convenience in drug administration, with a high percentage of patients preferring home administration [30][31] - The management is focused on strategic partnerships for larger indications to enhance market reach and financial stability [55] This summary encapsulates the key points discussed during the conference call, highlighting the company's strategic direction, market dynamics, and pipeline developments.