- Ann & Robert H. Lurie Children's Hospital of Chicago, a top-ranked hospital, is ready to evaluate patients for ZEVASKYN treatment - - Abeona Assist™ comprehensive patient services program in place to offer personalized support for eligible patients and their families throughout ZEVASKYN treatment journey - CLEVELAND and CHICAGO, May 14, 2025 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) and Ann & Robert H. Lurie Children's Hospital of Chicago today announced that Lurie Children's is now acti ...

Core Insights - MeiraGTx announced a strategic collaboration with Hologen AI, involving a $200 million upfront payment and a joint venture, Hologen Neuro AI Ltd, with an additional $230 million committed to expedite the Phase 3 clinical development of AAV-GAD for Parkinson's disease [1][2][9] - The efficacy data for rAAV8.hRKp.AIPL1 for treating AIPL1-related retinal dystrophy was published, showing positive responses in all 11 children treated, leading to plans for Marketing Authorization Approval (MAA) in the UK and discussions with the FDA for a similar pathway in the US [1][2][10] Financial and Operational Highlights - As of March 31, 2025, MeiraGTx had cash and cash equivalents of approximately $66.5 million, with a net loss attributable to ordinary shareholders of $40.0 million for the quarter [21][31] - Service revenue increased to $1.9 million for the three months ended March 31, 2025, compared to $0.7 million for the same period in 2024, attributed to progress in process performance qualification services [23] - General and administrative expenses decreased to $9.4 million for the first quarter of 2025, down from $13.2 million in the same quarter of 2024, primarily due to reductions in share-based compensation and other costs [25] Clinical Development Updates - The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation for AAV-GAD for Parkinson's disease, allowing for expedited development and increased interaction with the FDA [4][9] - Positive discussions with the FDA have aligned on the requirements for ongoing Phase 2 studies for AAV-hAQP1 and AAV-GAD, supporting potential BLA filings [2][10] - The Phase 2 AQUAx2 study for AAV-hAQP1 continues to enroll participants, with a target for completion in Q4 2025 and potential BLA filing by the end of 2026 [13] Strategic Collaborations and Future Plans - The joint venture with Hologen Neuro AI Ltd aims to leverage AI technology to enhance the development of therapies targeting CNS disorders, with a focus on the AAV-GAD program [2][9] - MeiraGTx plans to initiate a Phase 3 study of AAV-GAD in the second half of 2025, continuing to work closely with regulatory agencies to expedite the development process [9][10]

Core Viewpoint - REGENXBIO Inc. announced that the FDA has accepted the Biologics License Application (BLA) for RGX-121, a potential one-time gene therapy for Mucopolysaccharidosis II (MPS II), with a target action date of November 9, 2025 [1][7]. Company Overview - REGENXBIO is a biotechnology company focused on gene therapy, founded in 2009, and has developed a late-stage pipeline for rare and retinal diseases [6]. - The company has pioneered AAV gene therapy and aims to improve lives through its curative potential [6]. Product Details - RGX-121 (clemidsogene lanparvovec) is designed to deliver the iduronate-2-sulfatase (IDS) gene to the central nervous system, potentially providing a permanent source of the I2S protein [4]. - The therapy aims to address both neurodevelopmental and systemic effects of Hunter syndrome, which currently relies on weekly enzyme replacement therapy [2]. Regulatory Designations - RGX-121 has received multiple designations from the FDA, including Orphan Drug Product, Rare Pediatric Disease, Fast Track, and Regenerative Medicine Advanced Therapy (RMAT) [2]. Commercialization Strategy - Following potential FDA approval, RGX-121 will be commercialized by NS Pharma, a subsidiary of Nippon Shinyaku, while REGENXBIO retains all rights and proceeds related to the potential sale of a Priority Review Voucher (PRV) [3].

Financial Data and Key Metrics Changes - REGENXBIO ended the quarter on March 31, 2025, with cash, cash equivalents, and marketable securities of $272 million, an increase from $245 million as of December 31, 2024, primarily driven by a $110 million upfront payment from the Nippon Shinyaku collaboration [19][20]. - R&D expenses for the quarter were $53 million, down from $54.8 million in the same quarter of 2024, mainly due to clinical trial expenses for RGX-314 and RGX-202 [20]. Business Line Data and Key Metrics Changes - RGX-121, a gene therapy for MPS II, is on track for potential FDA approval in the second half of 2025, with a BLA submitted under the accelerated approval pathway [5][6]. - RGX-202, a candidate for Duchenne muscular dystrophy (DMD), has surpassed 50% enrollment in its pivotal study and is expected to submit a BLA in mid-2026 [7][12]. - The retinal program, RGX-314, is advancing in two pivotal studies for wet AMD and is on track to be the first gene therapy for this condition [9][16]. Market Data and Key Metrics Changes - The DMD market is projected to have over half of the prevalent population untreated by 2027, highlighting a significant opportunity for RGX-202 [8]. - The wet AMD and diabetic retinopathy markets represent large multibillion-dollar commercial opportunities, with RGX-314 positioned to serve as a meaningful alternative to current treatments [9][16]. Company Strategy and Development Direction - The company is focused on transitioning to a commercial stage with in-house manufacturing capabilities and plans to secure non-dilutive funding [4][5]. - A strategic partnership with Nippon Shinyaku aims to commercialize the neurodegenerative franchise, including RGX-121 and RGX-111 [6]. - The company is preparing for commercial supply manufacturing of RGX-202 in anticipation of a 2027 launch [8][24]. Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the clinical progress and the potential for multiple first or best-in-class gene therapies to reach patients in the coming years [4][10]. - The company is optimistic about the FDA's review process for RGX-121 and believes the data supports a favorable benefit-risk profile for its gene therapies [30][46]. Other Important Information - The company has a robust cash runway expected to fund operations into the second half of 2026, with potential extensions through non-dilutive financing options [20][21]. - The manufacturing innovation center in Rockville, Maryland, is capable of producing up to 2,500 doses of RGX-202 annually, ensuring readiness for market needs upon approval [8][24]. Q&A Session Summary Question: Timing for the Hunter BLA - Management indicated that the BLA acceptance is imminent and they feel confident about the review process [30]. Question: Changes in Approvals and Biomarker Expectations - Management stated that they are prepared for an AdCom if required and are confident in their data supporting the accelerated approval pathway [32][82]. Question: Safety Profile Expectations - Management noted that they do not anticipate changes in the FDA's requirements regarding safety profiles and that enrollment for the pivotal study is on track [38][40]. Question: Updates on Diabetic Retinopathy Phase III Trial - Management confirmed that they are actively working with AbbVie on the final feedback for the trial and expect to begin site activation soon [51]. Question: Functional Data Updates for DMD - Management plans to release additional functional data for RGX-202 in the first half of the year, focusing on expanding the patient data set [57][84]. Question: Impact of Recent Pricing Announcements - Management believes it is too early to assess the impact of recent pricing discussions on their gene therapies [102].

Core Insights - REGENXBIO Inc. reported significant advancements in its gene therapy pipeline, with multiple late-stage assets showing differentiation from standard treatments, positioning the company for potential first- or best-in-class therapies for rare and retinal diseases [2] Program Highlights and Milestones - RGX-202 is a novel microdystrophin gene therapy for Duchenne muscular dystrophy, utilizing the NAV® AAV8 vector, and is the only construct including the C-Terminal domain found in natural dystrophin [3] - Clemidsogene lanparvovec (RGX-121) is being developed as a first-in-class treatment for MPS II (Hunter syndrome) in partnership with Nippon Shinyaku [4] - Surabgene lomparvovec (sura-vec, ABBV-RGX-314) is on track to be the first-in-class treatment for wet age-related macular degeneration (wet AMD), with pivotal trial enrollment ongoing and completion expected in 2025 [5][8] Financial Results - As of March 31, 2025, REGENXBIO's cash, cash equivalents, and marketable securities totaled $272.7 million, an increase from $244.9 million at the end of 2024, primarily due to a $110 million upfront payment from the Nippon Shinyaku partnership [11] - Revenues for the first quarter of 2025 were $89.0 million, a significant increase from $15.6 million in the same period of 2024, largely driven by $71.8 million in license and service revenue from the collaboration with Nippon Shinyaku [12] - Research and development expenses decreased to $53.1 million in Q1 2025 from $54.8 million in Q1 2024, while general and administrative expenses rose to $20.3 million from $18.3 million [13][14] - The company reported a net income of $6.1 million, or $0.12 per share, compared to a net loss of $63.3 million, or $1.38 per share, in the prior year [15] Corporate Updates - REGENXBIO's partnership with Nippon Shinyaku, finalized in March 2025, includes an upfront payment of $110 million and potential additional payments of up to $700 million based on milestone achievements [10] - The company is preparing for a Biologics License Application (BLA) submission for clemidsogene lanparvovec (RGX-121) expected in May 2025, with potential approval in the second half of 2025 [6]

CANbridge Pharmaceuticals Corporate Presentation Aug 2023 Disclaimer THIS DOCUMENT OR THE INFORMATION CONTAINED HEREIN IS NOT INTENDED TO AND DOES NOT CONSTITUTE ANY OFFER OR INVITATION, SOLICITATION, COMMITMENT OR ADV ERTISEMENT OF ANY OFFER FOR SUBSCRIPTION, PURHCASE OR SALE OF ANY SECURITIES, NOR SHALL ANY PART OF THIS DOCUMENT FORM THE BASIS OF OR BE RELIED ON IN CONNECTION WITH ANY CONTRACT OR COMMITMENT WHATSOEVER. This document contains strictly conf idential and proprietary inf ormation in relation ...

Financial Performance - Rocket Pharmaceuticals incurred a loss of 56 cents per share in Q1 2025, which is narrower than the Zacks Consensus Estimate of a loss of 59 cents and an improvement from a loss of 66 cents per share in the same quarter last year [1] - The company did not record any revenues in the reported quarter, missing the Zacks Consensus Estimate for total revenues of $8 million [2] - General and administrative expenses rose 28% year over year to $28.4 million, attributed to increased commercial preparation and legal expenses [2] - Research and development expenses were $35.9 million, down 21% from the previous year due to reduced manufacturing and development costs [3] - As of March 31, 2025, the company had cash, cash equivalents, and investments totaling $318.2 million, down from $372.3 million as of December 31, 2024, with expectations to fund operations into Q4 2026 [3] - Year to date, RCKT shares have declined 44%, compared to an 8% decline in the industry [4] Pipeline Developments - Kresladi, developed for treating severe leukocyte adhesion deficiency-I (LAD-I), received a complete response letter (CRL) from the FDA in June 2024, requesting limited additional information on the Chemistry Manufacturing and Controls (CMC) [7][8] - The company plans to file a complete BLA to resolve the CRL later in 2025 [8] - Rocket Pharmaceuticals is developing RP-L102 for treating Fanconi anemia (FA) and has initiated a rolling BLA, expecting to complete the submission in late 2025 or early 2026 [9] - Dosing is currently underway in a phase II pivotal study for RP-A501, targeting male patients with Danon disease, with clinical data readout expected in mid-2026 [10]

Financial Data and Key Metrics Changes - Revenue for Q1 2025 was $1.6 million, a decrease of $6.9 million compared to $8.5 million in Q1 2024, primarily due to a reduction in collaboration revenue and contract manufacturing revenue [17][18] - Research and development expenses were $36.1 million in Q1 2025, down from $40.7 million in the same period in 2024, mainly due to decreased employee-related and facility expenses [18] - Cash, cash equivalents, and investment securities totaled $409 million as of March 31, 2025, compared to $367.5 million as of December 31, 2024, reflecting the net proceeds from an $80.5 million follow-on offering [19][20] Business Line Data and Key Metrics Changes - The company is advancing its clinical pipeline with the initiation of three additional studies in refractory temporal lobe epilepsy, Fabry disease, and SOD1 ALS, while continuing enrollment in existing studies [8][9] - Enrollment for the SOD1 ALS trial's first two dose cohorts has been completed, and initial data from the Fabry disease study is expected in the second half of 2025 [9][16] Market Data and Key Metrics Changes - The FDA granted breakthrough therapy designation for AMT-130, highlighting the urgent need for treatments in Huntington's disease [8][12] - The company is preparing for a planned BLA submission and expects to provide a regulatory update later this quarter [21] Company Strategy and Development Direction - The company aims to submit a BLA for AMT-130 in 2025, which is seen as a transformational year with multiple milestones ahead [7][10] - The focus remains on delivering innovative therapies for Huntington's disease, with plans to engage with European regulators for potential commercialization [82] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the data supporting AMT-130 and its potential to slow disease progression, emphasizing the importance of clinical outcomes over surrogate endpoints [26][27] - The company remains optimistic about its interactions with the FDA and the path forward for its clinical programs [41][42] Other Important Information - The company has significantly reduced its cash burn through divestitures and restructuring, providing financial flexibility to advance its pipeline [9][10] - The management highlighted the importance of patient advocacy groups in the development of treatments for Huntington's disease [50][51] Q&A Session Summary Question: Confidence in three-year follow-up data on DUHDRS - Management is confident that the dose-dependent reduction in CUHDRS observed at two years will be maintained at three years [30][32] Question: Inclusion of propensity map scoring in third-quarter update - The analysis will be agreed upon with the FDA before locking the database, and top-line results will be shared accordingly [34] Question: Changes in key personnel after CMC meeting - No material changes in the review team have been noted, and the company remains encouraged about its pipeline [75] Question: Future confirmatory study discussions - The FDA is not ready to discuss confirmatory studies until the BLA submission data is reviewed, but the company does not expect this to delay the BLA filing [105]

Core Insights - uniQure N.V. has received Breakthrough Therapy designation from the FDA for AMT-130, indicating significant progress towards a potential disease-modifying treatment for Huntington's disease [3][4] - The company reported a cash position of approximately $409.0 million as of March 31, 2025, which is expected to fund operations into the second half of 2027 [7] - Financial results for Q1 2025 show a revenue decrease to $1.6 million from $8.5 million in Q1 2024, primarily due to reduced collaboration and contract manufacturing revenues [8] Company Developments - AMT-130 is advancing towards a planned Biologics License Application (BLA) submission, with regulatory updates expected in Q2 2025 [3][4] - Initial safety data from the third cohort of the Phase I/II study of AMT-130 indicate it is generally well-tolerated, with no treatment-related serious adverse events reported [5][6] - The company plans to present initial clinical data for AMT-260 at the Epilepsy Therapies & Diagnostics Development Symposium on May 29, 2025 [4][9] Financial Highlights - The cash position increased from $367.5 million at the end of 2024 to $409.0 million by March 31, 2025, largely due to net proceeds of $80.5 million from a follow-on offering [7] - Research and development expenses decreased to $36.1 million in Q1 2025 from $40.7 million in Q1 2024, reflecting a reduction in employee-related and facility expenses [11] - The net loss for Q1 2025 was $43.6 million, or $0.82 per share, compared to a net loss of $65.6 million, or $1.36 per share, in the same period of 2024 [16][25]

Core Insights - REGENXBIO Inc. is showcasing its advancements in gene therapy at the ASGCT 28th Annual Meeting, emphasizing its comprehensive capabilities in research, clinical development, and manufacturing [1][5] Presentations Overview - The company will present late-stage clinical trial data for RGX-121, aimed at treating MPS II, and RGX-202 for Duchenne muscular dystrophy, along with preclinical research on RGX-202's novel construct and manufacturing process [2][5] - Key oral presentations include: - Development of a commercial manufacturing process for RGX-202 [3] - Investigational gene therapy RGX-121 for neuronopathic MPS II [3] - Interim clinical data for RGX-202 [3] Poster Presentations - Various poster presentations will cover topics such as: - The impact of sample collection conditions on AAV endotoxin testing [4] - AAV-expressed microdystrophin's effects in a mouse model of Duchenne muscular dystrophy [4] - Development of in vitro methods for analyzing TLR9 stimulation by AAV vector genomes [4] - Characterization of oversized AAV vectors with high genome integrity [4] - Blood-brain barrier crossing AAV vectors targeting the transferrin receptor [4] Company Background - REGENXBIO, founded in 2009, focuses on gene therapy, particularly AAV gene therapy, with a late-stage pipeline targeting rare and retinal diseases [5] - The company has treated thousands of patients using its AAV platform, including those receiving Novartis' ZOLGENSMA® [5]