Latest news with #Angiogenesis
Yahoo
08-02-2025
- Business
- Yahoo
4DMT Presents Positive 52-Week Results from Phase 2b Cohort of PRISM Wet AMD Study and Long-term Durability Data Supporting 4D-150 4FRONT Global Registration Program
3E10 vg/eye achieved an 83% reduction in injection burden vs. projected on-label aflibercept 2 mg Q8W, 70% required 0-1 supplemental injection, and 57% were injection-free through 52 weeks In the recently diagnosed subgroup, which most resembles the Phase 3 4FRONT-1 and 4FRONT-2 patient populations, 87% required 0-1 supplemental injection and 80% were injection-free through 52 weeks Durable and stable aflibercept expression demonstrated across all 3E10 vg/eye PRISM cohorts, with up to two years of follow-up 4D-150 continues to be well tolerated, with up to three years of follow-up 4FRONT-1 and 4FRONT-2 are on target to initiate in Q1 and Q3 2025, respectively Company to host webcast on Monday, February 10, 2025 at 8:00 a.m. ET EMERYVILLE, Calif., Feb. 08, 2025 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics (Nasdaq: FDMT; 4DMT or the Company), a leading clinical-stage company focused on unlocking the full potential of genetic medicines to treat large market diseases, today announced positive initial interim 52-week data from the Phase 2b Population Extension cohort of the PRISM clinical trial evaluating 4D-150 in a broad wet age-related macular degeneration (wet AMD) patient population. Additional data were provided on the durability of aflibercept expression for up to two years. The data were presented by Dante Pieramici, M.D., in an oral presentation titled 'Phase 2b Population Extension Cohort Evaluating 4D-150 in Neovascular Age-Related Macular Degeneration: 52-Week Results' at Angiogenesis, Exudation, and Degeneration 2025. 'We believe 4D-150 has paradigm-shifting potential. 4D-150 is designed to achieve favorable tolerability and robust and durable multi-year efficacy following routine intravitreal administration that enables seamless integration into retina clinics,' said David Kirn, M.D., Co-founder and Chief Executive Officer of 4DMT. 'The data from the PRISM clinical trial, which evaluated wet AMD patients with a broad range of disease severity and duration, and the initial data from SPECTRA in diabetic macular edema (DME), demonstrate 4D-150's potential to become the first backbone therapy forming the foundation for the treatment of vascular retinal diseases. The ability to deliver disease control with long-lasting freedom from frequent bolus injections addresses the primary unmet need for patients and physicians.' Topline 52-Week Efficacy Results for 4D-150 3E10 vg/eye (Planned Phase 3 Dose) from Phase 2b Population Extension Cohort of PRISM (Data Cut-Off January 15, 2025): Phase 2b (n=30): Broad Wet AMD Disease Activity Supplemental aflibercept injections: 83% reduction, representing 0.97 mean supplemental injections per patient over 52-weeks vs. 6.0 injections projected with on-label aflibercept 2 mg Q8W 70% 0-1 injection 57% injection-free Improved and maintained best corrected visual acuity (BCVA) of +2.2 letters Durable central subfield thickness (CST) improvement with fewer fluctuations, as measured by optical coherence tomography (OCT), of -11 µm; -13 µm in supplemental injection-free patients Phase 2b (n=15): Recently Diagnosed Subgroup Supplemental aflibercept injections: 94% reduction, representing 0.33 mean supplemental injections per patient over 52-weeks vs. 6.0 injections projected with on-label aflibercept 2 mg Q8W 87% 0-1 injection 80% injection-free Improved and maintained BCVA of +3.1 letters Durable CST improvement with fewer fluctuations, as measured by OCT, of -10 µm; -20 µm in supplemental injection-free patients 4D-150 Safety Update from PRISM (Data Cut-Off January 15, 2025): 4D-150 continues to be well tolerated during up to three years of follow up in all patients treated with 3E10 vg/eye 2.8% (2 of 71) had 4D-150–related 1+ intraocular inflammation (IOI) (SUN/NEI scales), which were transient 1+ vitreous cells noted at a single timepoint, as previously reported 99% (70 of 71) completed steroid prophylaxis taper on schedule 99% (70 of 71) remained completely off steroids No 4D-150–related hypotony, endophthalmitis, vasculitis, occlusive/non-occlusive retinal vasculitis, or choroidal effusions observed to date "The promise of 4D-150 for both patients and clinicians lies in its potential to tackle one of the most pressing unmet needs in vascular retinal diseases—providing a long-lasting, effective treatment option that reduces the frequent burden of bolus anti-VEGF injections,' said Dante Pieramici, M.D., a principal investigator of the PRISM study and member of the 4DMT Ophthalmology Advisory Board. '4D-150 offers a profound shift in how we manage our patients' care, potentially freeing them from the ongoing challenges of injection frequency while ensuring they maintain the vision improvement characteristic of current standard of care. The data from the PRISM study gives me great hope that 4D-150 can become the backbone of future retinal treatments for wet AMD, offering both clinical benefit and better quality of life for our patients." PRISM Durability Update from All 3E10 vg/eye Cohorts Aqueous humor concentrations were studied serially every three months Durable and stable aflibercept expression demonstrated, with up to two years of follow-up, with aqueous humor concentrations consistently within projected therapeutic range 4D-150 Program Milestones 4FRONT-1 and 4FRONT-2 expected to initiate in Q1 and Q3 2025, respectively Two-year Phase 1/2a and 18-month Phase 2b PRISM data expected in Q4 2025 Primary endpoint 52-week topline data from both 4FRONT-1 and 4FRONT-2 expected in H2 2027 Corporate Webcast Details Title: 4D-150 in Broad Wet AMD Population: Interim 52-week Data from Phase 2b & Program Durability Update for 3E10 vg/eye (Phase 3 Dose)Date/Time: Monday, February 10, 2025, at 8:00 a.m. ETRegistration: Link An archived copy of the webcast will be available for up to one year by visiting the 'Investors & Media' section of the 4DMT website: The presentation from Angiogenesis, Exudation, and Degeneration 2025 will also be available on the 4DMT website: About 4D-1504D-150 is a potential backbone therapy that is designed to provide multi-year sustained delivery of anti-VEGF (aflibercept and anti-VEGF-C) from the retina with a single, safe, intravitreal injection. 4D-150 utilizes our customized and evolved intravitreal vector, R100, which was invented at 4DMT through our proprietary Therapeutic Vector Evolution platform. 4D-150 is being developed for wet AMD and DME, which both affect millions of patients globally, with the goal of freeing patients from burdensome injections while preserving vision. About Wet AMDWet AMD is a highly prevalent disease with estimated incidence rate of 200,000 new patients per year in the United States. It is estimated that the total prevalence of wet AMD in certain major markets, including the United States and the European Union, and Japan, will be greater than 4 million individuals in the next five years. Wet AMD is a type of macular degeneration in which abnormal blood vessels (macular neovascularization or MNV) grow into the macula, the central area of the retina. As a consequence, MNV causes swelling and edema of the retina, bleeding, and scarring, and causes visual distortion and reduced visual acuity. The proliferation and leakage of abnormal blood vessels is stimulated by VEGF. This process distorts and can potentially destroy central vision and may progress to blindness without treatment. About 4DMT 4DMT is a late-stage biotechnology company focused on unlocking the full potential of genetic medicines to treat large market diseases in ophthalmology and pulmonology. 4DMT's proprietary invention platform, Therapeutic Vector Evolution, combines the power of directed evolution with approximately one billion synthetic AAV capsid-derived sequences to invent customized and evolved vectors for use in our wholly owned and partnered product candidates. Our lead program, 4D-150, is a potential backbone therapy that is designed to provide multi-year sustained delivery of anti-VEGF (aflibercept and anti-VEGF-C) targeted to the retina with a single, safe, intravitreal injection. Our second core program is 4D-710, which is the first known genetic medicine to demonstrate, in the lungs of people with cystic fibrosis (CF), successful delivery and expression of the CFTR transgene and initial clinical activity signals after aerosol delivery of a gene therapy. 4D Molecular Therapeutics™, 4DMT™, Therapeutic Vector Evolution™, and the 4DMT logo are trademarks of 4DMT. All of our product candidates are in clinical or preclinical development and have not yet been approved for marketing by the FDA or any other regulatory authority. No representation is made as to the safety or effectiveness of our product candidates for the therapeutic uses for which they are being studied. Learn more at and follow us on LinkedIn. Forward Looking Statements:This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, implied and express statements regarding the therapeutic potential, and clinical benefits and market potential of 4DMT's product candidates, including 4D-150, as well as the plans, announcements, and related timing for the clinical development of and regulatory interactions regarding 4D-150. The words "may," 'might,' "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," 'expect,' "estimate," 'seek,' "predict," 'future,' "project," "potential," "continue," "target" and similar words or expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including risks and uncertainties that are described in greater detail in the section entitled "Risk Factors" in 4D Molecular Therapeutics' most recent Quarterly Report on Form 10-Q filed on November 13, 2024 as well as any subsequent filings with the Securities and Exchange Commission. In addition, any forward-looking statements represent 4D Molecular Therapeutics' views only as of today and should not be relied upon as representing its views as of any subsequent date. 4D Molecular Therapeutics explicitly disclaims any obligation to update any forward-looking statements. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements. Contacts: Media:Jenn Gordondna CommunicationsMedia@ Investors:Julian PeiHead of Investor Relations and Corporate in to access your portfolio
Associated Press
28-01-2025
- Business
- Associated Press
Clearside Biomedical Announces Multiple Medical Meeting Presentations Focused on the Advantages of Suprachoroidal Delivery and Key Differentiators in its CLS-AX Clinical Program
- Upcoming Presentations at Angiogenesis and Macula Society Conferences to Feature CLS-AX Sub-Group Analyses from ODYSSEY Wet AMD Trial - - CLS-AX Targets Flexible Dosing of Biologics with Duration of Tyrosine Kinase Inhibitors - ALPHARETTA, Ga., Jan. 28, 2025 (GLOBE NEWSWIRE) -- Clearside Biomedical, Inc. (Nasdaq: CLSD), a biopharmaceutical company revolutionizing the delivery of therapies to the back of the eye through the suprachoroidal space (SCS®), announced today recent and upcoming presentations at ophthalmic medical meetings highlighting Clearside's suprachoroidal delivery technology and promising pipeline, including its Phase 3 ready CLS-AX program for the treatment of neovascular age-related macular degeneration (wet AMD). 'Our medical meetings this quarter continue to demonstrate the commercial, clinical and regulatory expertise that establishes Clearside as the leader in suprachoroidal delivery,' said Victor Chong, MD, MBA, Chief Medical Officer and EVP, Head of Research and Development. 'We are excited about the potential of our CLS-AX program and look forward to presenting additional data from our Phase 2b ODYSSEY trial at upcoming meetings. We believe suprachoroidal CLS-AX has the potential to deliver comparable 6-month therapeutic effect in most wet AMD patients, similar to other intravitreal TKIs in development, while allowing physicians to have more individualized and flexible dosing options, instead of rescue, for patients who need more frequent therapy. We also believe suprachoroidal delivery enables the precise application of therapy to the retina, which may result in improved safety over other intravitreal treatment options.' Recent Sessions on Suprachoroidal Drug Delivery Utilizing Clearside's SCS Microinjector®: Hawaiian Eye & Retina 2025 (January 18-24, 2025) Presentation: Where Are We with Suprachoroidal Delivery Presenter: Judy E. Kim, MD, University of Texas Southwestern Medical Center Presentation: Suprachoroidal Delivery of Investigational ABBV-RGX-314 for Diabetic Retinopathy: The Phase II ALTITUDE ® Study Presenter: Margaret Chang, MD, MS, Retinal Consultants Medical Group Presentation: Tyrosine Kinases Inhibitors Presenter: Rishi P. Singh, MD, Cleveland Clinic Presentation: Suprachoroidal Delivery of Triamcinolone Injectable Suspension for Post-Operative Cystoid Macular Edema Presenter: Irena Tsui, MD, Doheny Eye Center UCLA 3rd Annual Ophthalmic Drug Delivery Summit (January 28-30, 2025) Presentation: Advancing Targeted, Compartmentalized, & Long-Acting Depot Delivery Suprachoroidal Delivery of Particulate Formulations Presenter: Viral Kansara, PhD, Vice President, Preclinical Development, Clearside Biomedical Upcoming Sessions on CLS-AX Wet AMD Program Angiogenesis, Exudation, and Degeneration 2025 (Virtual; February 8, 2025) Presentation: Phase 2b CLS-AX ODYSSEY Trial Results Presenter: Roger Goldberg, MD, MBA, Bay Area Retinal Associates Medical Group The Macula Society 48th Annual Meeting (February 12-15, 2025) Presentation: Top Line Results from ODYSSEY: A Phase 2b Study of Suprachoroidally Administered CLS-AX in Participants with Neovascular Age-related Macular Degeneration Presenter: Thomas A. Ciulla, MD, MBA, Chief Medical Advisor-Retina and Chair, Scientific Advisory Board, Clearside Biomedical 5th Annual Wet AMD & Diabetic Eye Disease Drug Summit (March 18-20, 2025) Presentation: Transforming wAMD Treatment: Long-Lasting, Flexible Dosing with Suprachoroidal TKI Delivery Presenter: Victor Chong, MD, MBA, Chief Medical Officer, Clearside Biomedical About Clearside's Suprachoroidal Space (SCS®) Injection Platform and SCS Microinjector® Clearside's patent protected, proprietary suprachoroidal space (SCS®) injection treatment approach offers unprecedented access to the back of the eye, where sight-threatening disease often occurs. The Company's unique platform is inherently flexible and intended to work with established and new formulations of medications. Clearside's patented SCS Microinjector® can deliver a wide variety of drug candidates into the suprachoroidal space, providing targeted delivery to potentially improve efficacy and compartmentalization of medication to reduce or eliminate toxic effects on non-diseased cells. The SCS Microinjector is comprised of a syringe with a custom-designed hub and two 30-gauge hollow microneedles of varying lengths, each approximately one millimeter, optimizing insertion and suprachoroidal administration of drugs. About ODYSSEY Phase 2b Clinical Trial ODYSSEY was a randomized, double-masked, parallel-group, active-controlled, multicenter, 36-week, Phase 2b clinical trial in participants with wet AMD previously treated with intravitreal anti-vascular endothelial growth factor (VEGF) standard of care therapy. A total of 60 participants were treated for 36 weeks and randomized to either CLS-AX (1 mg) or aflibercept (2 mg) with a 2:1 randomization schedule (40 participants in CLS-AX arm and 20 participants in aflibercept arm). CLS-AX was administered via suprachoroidal injection using Clearside's SCS Microinjector, and aflibercept was administered via intravitreal injection. Participants in the trial were determined to have active disease with a median duration of wet AMD diagnosis of 9.9 months. The ODYSSEY trial achieved its objectives, including primary outcomes in mean change from baseline in best corrected visual acuity and safety and tolerability of CLS-AX, and secondary outcomes in visual function and ocular anatomy, the need for supplemental treatment, and treatment burden as measured by total injections over the trial duration. CLS-AX demonstrated compelling intervention-free rates with 100% of CLS-AX participants not requiring any additional treatment up to 3 months, 90% up to 4 months, 81% up to 5 months, and 67% up to 6 months after the initial CLS-AX dose. In the CLS-AX group, the injection frequency was reduced by approximately 84% compared to the average monthly injections in the 24 weeks prior to screening. About CLS-AX (axitinib injectable suspension) Clearside is developing CLS-AX as a longer-acting therapy for the treatment of retinal diseases. CLS-AX (axitinib injectable suspension) is a proprietary suspension of axitinib for suprachoroidal injection. Axitinib is a tyrosine kinase inhibitor (TKI), currently approved as an oral tablet formulation to treat advanced renal cell carcinoma, that achieves pan-VEGF blockade, directly inhibiting VEGF receptors-1, -2, and -3 with high potency and specificity. Clearside believes this broad VEGF blockade may have efficacy advantages over existing retinal therapies by acting at a different level of the angiogenesis cascade and may benefit patients who sub-optimally respond to current, more narrowly focused anti-VEGF therapies. Suprachoroidal injection of this proprietary suspension of axitinib has demonstrated meaningful potential in Phase 1/2a and Phase 2b wet AMD clinical trials in which CLS-AX was well tolerated and demonstrated a positive safety profile. With suprachoroidal administration of axitinib, there is the potential to achieve prolonged duration and targeted delivery to affected tissue layers by compartmentalizing axitinib behind the retina, thereby limiting drug exposure to the front of the eye. About Clearside Biomedical, Inc. Clearside Biomedical, Inc. is a biopharmaceutical company revolutionizing the delivery of therapies to the back of the eye through the suprachoroidal space (SCS®) to improve patient outcomes. Clearside's SCS injection platform, utilizing the Company's patented SCS Microinjector®, enables an in-office, repeatable, non-surgical procedure for the targeted and compartmentalized delivery of a wide variety of therapies to the macula, retina, or choroid to potentially preserve and improve vision in patients with sight-threatening eye diseases. Clearside is developing its own pipeline of small molecule product candidates for administration via its SCS Microinjector. The Company's lead program, CLS-AX (axitinib injectable suspension), is in development for the treatment of neovascular age-related macular degeneration (wet AMD). Planning for a Phase 3 program is underway. In addition, Clearside is evaluating various small molecules for the potential long-acting treatment of geographic atrophy (GA). Clearside developed and gained approval for its first product, XIPERE ® (triamcinolone acetonide injectable suspension) for suprachoroidal use, which is available in the U.S. through a commercial partner. Clearside also strategically partners its SCS injection platform with companies utilizing other ophthalmic therapeutic innovations. For more information, please visit or follow us on LinkedIn and X. Cautionary Note Regarding Forward-Looking Statements Any statements contained in this press release that do not describe historical facts may constitute forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements may be identified by words such as 'believe', 'expect', 'may', 'plan', 'potential', 'will', and similar expressions, and are based on Clearside's current beliefs and expectations. These forward-looking statements include statements regarding the potential benefits of CLS-AX, Clearside's suprachoroidal delivery technology and Clearside's SCS Microinjector®. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements. Risks and uncertainties that may cause actual results to differ materially include uncertainties inherent in the conduct of clinical trials, Clearside's reliance on third parties over which it may not always have full control and other risks and uncertainties that are described in Clearside's Annual Report on Form 10-K for the year ended December 31, 2023, filed with the U.S. Securities and Exchange Commission (SEC) on March 12, 2024, Clearside's Quarterly Report on Form 10-Q for the quarter ended September 30, 2024, filed with the SEC on November 12, 2024, and Clearside's other periodic reports filed with the SEC. Any forward-looking statements speak only as of the date of this press release and are based on information available to Clearside as of the date of this release, and Clearside assumes no obligation to, and does not intend to, update any forward-looking statements, whether as a result of new information, future events or otherwise. Source: Clearside Biomedical, Inc.
