Data showed improvements across several key measures of visual function, including low luminance visual acuity and microperimetry Laru-zova was generally well-tolerated by DAWN participants at 6 months LONDON and CAMBRIDGE, Mass., May 06, 2025 (GLOBE NEWSWIRE) -- Beacon Therapeutics Holdings Limited (‘Beacon Therapeutics’ or ‘the Company’), a leading clinical-stage biotechnology company with a mission to save and restore the vision of patients with blinding retinal diseases, today announced 6-month interim ...

VYJUVEK Commercial Performance and Expansion - VYJUVEK has generated over $429 million in revenue since its launch[15] - Q1 2025 net revenue reached $88.2 million, a 95% increase compared to Q1 2024[16, 18] - The gross margin for Q1 2025 was 94%, with a gross to net of 17%[18] - Over 540 reimbursement approvals have been secured as of April 2025, with 45% from commercial payers and 39% from Medicaid[24, 25] - 97% of covered lives under commercial and Medicaid plans have positive access[25] Clinical Development Programs - Four clinical readouts are expected in 2025 for KB407 (cystic fibrosis), KB408 (AATD lung disease), KB304 (aesthetic skin conditions), and KB803 (ocular DEB)[19] - A Phase 1/2 study evaluating KB801 for neurotrophic keratitis is underway, with the first patient expected to be dosed this month[43] - Initial KB408 molecular data showed 35% AAT positive cells at baseline increasing to 39% after KB408 in patient 07[37] Financial Stability - The company's cash and investments totaled $7653 million as of March 31, 2025[54] - Non-GAAP R&D and SG&A expense guidance for the full year 2025 is $150 million to $175 million[55]

Abeona Therapeutics Inc.'s ABEO stock traded higher on Wednesday.The Food and Drug Administration (FDA) on Tuesday approved Abeona’s Zevaskyn (prademagene zamikeracel) gene-modified cellular sheets, also known as pz-cel, as the first and only autologous cell-based gene therapy for wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB).Zevaskyn is the only FDA-approved product to treat RDEB wounds with a single application.Zevaskyn consists of a patient's skin cells (ke ...

Company Overview - REGENXBIO Inc. is a biotechnology company focused on gene therapy with a mission to improve lives through its curative potential [3] - The company has been a pioneer in AAV gene therapy since its founding in 2009 [3] - REGENXBIO is advancing a late-stage pipeline of one-time treatments for rare and retinal diseases, including RGX-202 for Duchenne, RGX-121 for MPS II, and RGX-111 for MPS I, in partnership with Nippon Shinyaku [3] - The company is also collaborating with AbbVie on surabgene lomparvovec (ABBV-RGX-314) for wet AMD and diabetic retinopathy [3] - Thousands of patients have been treated with REGENXBIO's AAV platform, including those receiving Novartis' ZOLGENSMA® [3] Upcoming Events - REGENXBIO will host a conference call on May 12, 2025, at 4:30 p.m. ET to discuss its financial results for Q1 2025 and recent operational highlights [1] - Listeners can register for the webcast and analysts can participate in the Q&A session through provided links [2] - A replay of the webcast will be available on the company's investor website approximately two hours after the call [2]

Core Insights - Voyager Therapeutics announced eight presentations at the ASGCT annual meeting, focusing on advancements in gene therapies for neurological diseases, particularly Alzheimer's disease [1][2] Group 1: Gene Therapy Developments - The company highlighted its TRACER capsids, which have shown the ability to transduce 43%-98% of neurons and 87%-99% of astrocytes in non-human primates after a single intravenous dose [2] - The tau silencing gene therapy VY1706 demonstrated up to 73% knockdown of tau mRNA in non-human primates following a single intravenous dose of 1.3e13 vg/kg [2][6] - Presentations will include data on anti-amyloid gene therapy and enhancements to capsids aimed at immune evasion to broaden patient eligibility [2][6] Group 2: Presentation Details - Oral presentations will cover various topics, including the intravenous delivery of VY1706 and its effects on tau levels in the central nervous system [7] - Other presentations will focus on the discovery of AAV9-derived capsids that evade neutralizing antibodies and machine-learning applications for capsid screening [7][8] Group 3: Company Overview - Voyager Therapeutics is dedicated to leveraging human genetics to treat neurological diseases, with a pipeline that includes programs for Alzheimer's disease, Friedreich's ataxia, and Parkinson's disease [9] - The TRACER platform has enabled the development of novel capsids for gene therapy, enhancing the potential for high brain penetration following intravenous dosing [9][8]

Financial Data and Key Metrics Changes - Total revenues for the year ended December 31, 2024, were $28.9 million, an increase of $24.1 million, or 502.1%, compared to $4.8 million for the year ended December 31, 2023 [30] - Net loss for the year ended December 31, 2024, was $9.2 million compared to a net loss of $60.6 million for the year ended December 31, 2023 [34] - Cash, cash equivalents, and short-term bank deposits as of December 31, 2024, were $19.6 million, with approximately $29 million on a pro forma basis after financing completed in early January 2025 [34] Business Line Data and Key Metrics Changes - Revenues in 2024 included $15 million from upfront and milestone payments under the Gloria license agreement and $6 million of net APHEXDA product revenues [31] - Research and development expenses for the year ended December 31, 2024, were $9.2 million, a decrease of $3.3 million, or 26.4%, compared to $12.5 million for the year ended December 31, 2023 [32] - Sales and marketing expenses for the year ended December 31, 2024, were $23.6 million, a decrease of $1.7 million, or 6.7%, compared to $25.3 million for the year ended December 31, 2023 [33] Market Data and Key Metrics Changes - APHEXDA achieved a 10% market share of total CXCR4 inhibitor usage in the U.S. within less than 11 months of being on the market [26] - More than $6 million of net APHEXDA product revenue was generated in the U.S. through the closing of the Ayrmid transaction on November 21 [27] Company Strategy and Development Direction - The company has shifted its strategy to focus on evaluating early clinical stage and late pre-clinical stage therapeutic assets in oncology and rare diseases [6][7] - The exclusive license agreement with Ayrmid Pharma Ltd. for motixafortide allows the company to return to its roots in complex drug development while benefiting from Ayrmid's commercial potential [9][10] - The company aims to help as many patients as possible while creating enduring value for shareholders [20] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about evaluating promising candidates and making definitive announcements within the year [8] - The company has reduced its ongoing operating cash burn by approximately 70%, from over $40 million annually to approximately $12 million annually [18] - The company believes it is well-positioned to advance motixafortide in solid tumor indications while evaluating additional assets in oncology and rare diseases [19] Other Important Information - The Ayrmid agreement generated $10 million in upfront payments and potential milestones of $87 million, along with double-digit sales royalties ranging from 18% to 23% [16] - The company has shut down its U.S. operations and implemented a headcount reduction in Israel, allowing for a streamlined organization [17] Q&A Session Summary Question: Any more color on meaningful progress in evaluating assets? - Management indicated that while there are meaningful discussions ongoing, specific timelines for announcements cannot be provided [38] Question: How are internal programs, especially with WashU and sickle cell, looking? - Management noted that a significant win would be mobilizing enough cells in one cycle for gene therapy, reducing the number of apheresis sessions [43][44] Question: Commentary on increased traction of APHEXDA for multiple myeloma? - Management expressed confidence in Ayrmid's team and their marketing efforts, stating that the transition has gone smoothly [49] Question: How many assets have been looked at in the acquisition process? - Management stated that they have looked at thousands of molecules over the company's history, focusing on early clinical stage assets in oncology and rare diseases [56][58] Question: Allocation of expenses during the acquisition process? - Management confirmed that expenses would initially be higher during the search process, shifting towards R&D as acquisitions are made [59] Question: Internal versus external efforts in the acquisition process? - Management indicated that the majority of the work is done in-house, with external consultants used as needed [64]

Core Insights - Tenaya Therapeutics announced promising interim data from the MyPEAK-1 Phase 1b/2 clinical trial of TN-201, a gene therapy for MYBPC3-associated hypertrophic cardiomyopathy (HCM), presented at the 2025 American College of Cardiology Scientific Sessions [1][2][3] Group 1: Clinical Trial Data - TN-201 was well tolerated at a dose of 3E13 vg/kg, with treatment-emergent adverse events primarily mild and manageable [2][3] - All three patients in Cohort 1, who had severe disease at baseline, achieved NYHA Class I, indicating no limitations on physical activity [3] - Serial biopsies showed sustained presence of TN-201 DNA in the heart and robust RNA expression, increasing up to 13-fold from Week 8 to Week 52 post-dose [2][3] - MyBP-C protein levels increased from 56% to 59% and from 62% to 64% of normal between Week 8 and Week 52 for Patients 1 and 2, respectively [2][3] - Cardiac troponin levels decreased by more than 60% in two patients, returning to normal or near normal [2][3] Group 2: Future Expectations - Enrollment for Cohort 2 is expected to be completed in the first half of 2025, with initial data to be reported in the second half of 2025 [1][2][7] - The company anticipates sharing additional data from Cohort 1 and initial safety and biopsy data from Cohort 2 later this year [3][7] - Tenaya has updated its cash guidance into the second half of 2026, positioning itself to achieve important clinical data milestones for both TN-201 and TN-401 gene therapy programs [3] Group 3: Disease Context - MYBPC3-associated HCM is a severe condition affecting approximately 120,000 patients in the U.S., with no approved therapeutics addressing its underlying genetic cause [11][12] - Patients with MYBPC3 mutations are at higher risk for serious complications, including heart failure and sudden cardiac death, particularly if diagnosed before age 40 [10][11]

Core Insights - Opus Genetics is focused on developing innovative gene therapy treatments for inherited retinal diseases (IRDs) and has made significant progress in its transformation and pipeline [1][2] - The company raised $21.5 million through a public offering and private placement, enhancing its cash position to approximately $50.7 million, which supports key milestones for its gene therapy candidates [2][9] - The acquisition of Opus Genetics has strengthened the company's pipeline, which now includes seven adeno-associated virus (AAV)-based gene therapy assets targeting specific IRDs [4][17] Financial Performance - For the year ended December 31, 2024, Opus reported a net loss of $57.5 million, or $2.15 per share, compared to a net loss of $10.0 million, or $0.46 per share, in 2023 [15][25] - License and collaborations revenue decreased to $11.0 million in 2024 from $19.0 million in 2023, primarily due to the absence of a milestone payment received in 2023 [12][25] - General and administrative expenses increased to $18.2 million in 2024 from $12.0 million in 2023, attributed to transaction costs related to the acquisition and other operational expenses [13][25] Pipeline and Clinical Trials - The lead gene therapy candidate, OPGx-LCA5, is in a Phase 1/2 trial and has shown positive results, with visual improvement observed in all adult patients after one year [9][18] - OPGx-BEST1 is expected to enter clinical trials in 2025, targeting IRDs associated with mutations in the BEST1 gene [9][18] - The LYNX-2 Phase 3 trial for Phentolamine Ophthalmic Solution 0.75% has completed enrollment, with topline data expected in mid-2025 [9][18] Strategic Direction - The company aims to achieve several key milestones in 2025, including up to four clinical trial data readouts and the initiation of new trials for its gene therapy candidates [2][10] - A Type D meeting with the FDA was held to discuss the regulatory path for OPGx-LCA5, indicating proactive engagement with regulatory authorities [9][18] - The company is also seeking a strategic partner to advance the late-stage development of APX3330, a novel oral REF-1 inhibitor for diabetic retinopathy [9][18]

Core Insights - Defence Therapeutics Inc. has appointed Dr. Elias Theodorou as Chief Operating Officer, effective April 2025, to enhance its management team and accelerate development programs [2][3] - Dr. Theodorou brings over 25 years of experience in cancer research and innovative drug delivery systems, which aligns with Defence's mission to advance its proprietary Accum® technology [3][4] - The company has granted Dr. Theodorou 350,000 incentive stock options, with 100,000 vesting immediately and the remainder vesting in one year, exercisable at $1.07 per share for ten years [3] Company Overview - Defence Therapeutics is a clinical-stage biotechnology company focused on developing radio-immuno-conjugate and antibody-drug conjugate (ADC) products using its proprietary platform [4] - The core technology, Accum®, enables precision delivery of therapeutic agents to target cells, aiming to improve efficacy against cancer and infectious diseases [5]

Pz-cel (Prademagene zamikeracel) for RDEB - Pz-cel is anticipated to receive potential FDA approval for RDEB with a PDUFA date of April 29, 2025 [4, 74] - Phase 3 VIITAL study showed 81.4% of pz-cel treated wounds achieved >50% healing compared to control wounds at Week 24 [12] - Phase 3 VIITAL study showed 65.1% of pz-cel treated wounds achieved >75% healing compared to control wounds at Week 24 [12] - Phase 3 VIITAL study showed 16.3% of pz-cel treated wounds achieved complete healing compared to control wounds at Week 24 [12] - Pz-cel demonstrated a mean pain reduction from baseline at Week 24 [12] - The company estimates a peak annual US revenue of over $500 million for pz-cel [41] - The company anticipates gross margins of approximately 85-90% after the initial ramp-up [42] Pipeline and Partnerships - Ultragenyx's BLA for UX111 (ABO-102) in MPS IIIA has a PDUFA date of August 18, 2025 [4, 74] - The company is developing ABO-503 for X-linked retinoschisis (XLRS), with first-in-human opportunity in 1H 2026 [62, 65] Financial Resources - The company had $110 million in cash resources as of September 30, 2024, providing a runway through potential launch into 2026 [71, 72]