Geographic atrophy

Lineage Cell Therapeutics Reports First Quarter 2024 Financial Results and Provides Business Update

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Jeudi, mai 9, 2024
Biotechnology, Health, Clinical Trials, AMD, Ophthalmology, City, Regenerative medicine, Research, Spinal cord, NanoString Technologies, Roche, MD, Doctor of Philosophy, Partnership, Conference, Trial of the century, Webcast, Corporate development, Beckman Research Institute, CIRM, DVM, Patient, Lineage, Geographic atrophy, Madrona Venture Group, Expense, RPE, Therapy, Delivery, G&A, Partner, Interest, Fine chemical

Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, today reported its first quarter 2024 financial and operating results and will host a conference call at 4:30 p.m. Eastern Time to discuss these results and to provide a business update.

Key Points: 
  • Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, today reported its first quarter 2024 financial and operating results and will host a conference call at 4:30 p.m. Eastern Time to discuss these results and to provide a business update.
  • “The quarter was highlighted by significant milestones and data updates on our lead program,” stated Brian M. Culley, Lineage CEO.
  • Under this new agreement, Lineage will provide additional clinical, technical, training and manufacturing services that further support the ongoing advancement and optimization of the OpRegen program.
  • Interested parties may access the conference call on May 9th, 2024, by dialing (800) 715-9871 from the U.S. and Canada and should request the “Lineage Cell Therapeutics Call”.

4DMT Reports First Quarter 2024 Financial Results and Operational Highlights

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Jeudi, mai 9, 2024
FDA, Regenerative medicine, Research, Choroideremia, Biopsy, European Medicines Agency, Injection, Overalls, Inflammation, Food, Alpha-1 antitrypsin, Cystic fibrosis, Chelsea Handler, Intravitreal administration, Conference, Retina, Atypical hemolytic uremic syndrome, DME, Angiogenesis, PRISM, AMD, Safety, Security (finance), Cash, Retinitis pigmentosa, Exercise, PRIME, EMA, NHP, Clinical trial, A1AT, ASRS, ARVO, ECFS, Șaeș, Cubic foot, Patient, Society, Geographic atrophy, Pharmaceutical industry

“The first quarter of 2024 kicks off another transformative year for 4DMT with exceptional progress across our product pipeline and platform in multiple therapeutic areas, particularly our lead program 4D-150 in large market VEGF-driven retinal diseases,” said David Kirn, M.D., Co-founder and Chief Executive Officer of 4DMT.

Key Points: 
  • “The first quarter of 2024 kicks off another transformative year for 4DMT with exceptional progress across our product pipeline and platform in multiple therapeutic areas, particularly our lead program 4D-150 in large market VEGF-driven retinal diseases,” said David Kirn, M.D., Co-founder and Chief Executive Officer of 4DMT.
  • R&D Expenses: Research and development expenses were $27.9 million for the first quarter of 2024, as compared to $22.4 million for the first quarter of 2023.
  • G&A Expenses: General and administrative expenses were $10.3 million for the first quarter of 2024, as compared to $8.0 million for the first quarter of 2023.
  • Net Loss: Net loss was $32.4 million for the first quarter of 2024, as compared to net loss of $28.7 million for the first quarter of 2023.

Oculis Announces Completion of Enrollment in Phase 2 Randomized Controlled ACUITY Trial with OCS-05 for Acute Optic Neuritis

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Mercredi, mai 8, 2024
Neuroprotection, Inflammation, Hospital, Safety, OCS, Diabetic retinopathy, Apoptosis, Optic neuritis, AON, IND, Disease, Patient, Paratriathlon, Geographic atrophy, Optic nerve, Neuron, Glaucoma, Ophthalmology, Neuro, Pharmaceutical industry

The Phase 2 ACUITY study is evaluating once-daily OCS-05 intravenous infusion in patients with AON.

Key Points: 
  • The Phase 2 ACUITY study is evaluating once-daily OCS-05 intravenous infusion in patients with AON.
  • OCS-05 has been granted orphan drug designation in both the United States and Europe, highlighting the importance of this unmet medical need.
  • In the meantime, we keep on working towards an IND submission for OCS-05 in the U.S. in 2024.
  • While corticosteroids are used to shorten the attack, there is no approved therapy for AON and unmet needs remain for therapies that can prevent vision loss after an acute episode of optic neuritis.

Annexon Presents New Neuroprotection Data Showing ANX007 Protects Vision and Vision-Associated Structures in Geographic Atrophy at ARVO 2024 Annual Meeting

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Mardi, mai 7, 2024
Brain, ARCHER, Complement component 1q, CNV, Research, Microglia, Incidence, Patient, Eye, Pivotal, Vasculitis, Paratriathlon, Retinal pigment epithelium, Geographic atrophy, RPE, Atrophy, Annual general meeting, Synapse, ARVO, European Medicines Agency, Overalls, Visual acuity, Medical imaging

BRISBANE, Calif., May 07, 2024 (GLOBE NEWSWIRE) -- Annexon, Inc. (Nasdaq: ANNX), a biopharmaceutical company advancing a late-stage clinical platform of novel therapies for people living with devastating classical complement-mediated neuroinflammatory diseases of the body, brain, and eye, today presented new analyses of ANX007 from the Phase 2 ARCHER trial in geographic atrophy (GA), and new preclinical data on the role of C1q in the pathogenic elimination of photoreceptor synapses and their protection with C1q blockade in GA. ANX007 is a first-in-class, non-pegylated antigen-binding fragment (Fab) designed to block C1q and activation of the classical complement cascade locally in the eye with an intravitreal formulation, and is the first therapeutic candidate for the treatment of GA to receive Priority Medicine (PRIME) designation by the European Medicines Agency. The data were presented at the Association for Research in Vision and Ophthalmology (ARVO) 2024 Annual Meeting.

Key Points: 
  • The data were presented at the Association for Research in Vision and Ophthalmology (ARVO) 2024 Annual Meeting.
  • “We are pleased to present additional clinical data from the ARCHER trial that are the first to show preservation of both vision and relevant anatomical structures following ANX007 treatment,” said Douglas Love, president and chief executive officer of Annexon.
  • “Importantly, the statistically significant preservation of photoreceptor anatomy measured by ellipsoid zone change highlights protection of key retinal structures associated with vision.
  • These two registrational trials are designed to confirm the Phase 2 ARCHER findings of protection against vision loss and underscore the unique neuroprotective mechanism of action of ANX007 and its competitive differentiation in visual function.

Belite Bio to Participate in Key Opinion Leader Webinar to Discuss Unmet Needs In Stargardt Disease (STGD1) and Geographic Atrophy (GA) and Perspectives On Tinlarebant

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Lundi, mai 6, 2024
Stargardt disease, MB BS, Geographic atrophy, FACS, Royal College of Ophthalmologists, MB, Therapy, Retina, Bachelor of Science, Eye, Research fellow, Macular degeneration, RBP4, Moorfields Eye Hospital, ARVO

SAN DIEGO, May 06, 2024 (GLOBE NEWSWIRE) -- Belite Bio, Inc. (NASDAQ: BLTE), a clinical-stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases that have significant unmet medical needs, today announced that the executive management team will participate in a Key Opinion Leader Webinar hosted by Jennifer Kim, Director, Equity Research, of Cantor Fitzgerald on Monday, May 13, 2024, at 2:00 p.m. ET.

Key Points: 
  • Webinar, moderated by Cantor Fitzgerald, will be held on Monday, May 13, 2024, at 2:00 p.m.
  • The event will feature Michel Michaelides, BSc, MB BS, MD(Res), FRCOphth, FACS (University College London Institute of Ophthalmology and Moorfields Eye Hospital), who will discuss an overview of the unmet need for treatments in Stargardt disease (STGD1) and Geographic Atrophy (GA).
  • Tinlarebant has been granted Fast Track Designation and Rare Pediatric Disease designation in the U.S., and Orphan Drug Designation in the U.S., Europe, and Japan for the treatment of STGD1.
  • A replay of the webinar will be available approximately one week after the event for 90 days under the "Events" tab on the investor relations section of the Belite Bio website at: https://investors.belitebio.com/presentations-events/events .

Belite Bio Presents Additional Analysis from Phase 2 Study of Tinlarebant in Stargardt Disease at the ARVO Annual Meeting

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Lundi, mai 6, 2024
Stargardt disease, University, Bias, Westmead, Stargardt, Research, Geographic atrophy, DRAGON, Adolescence, Growth, Observation, Disease, Therapy, Retina, Dry, Patient, Classification, CSO, Genotype, ABCA4, Annual general meeting, Belite, ARVO, SAN, Visual acuity, Pharmaceutical industry

Tinlarebant is Belite Bio’s orally administered tablet intended to slow disease progression in patients affected with STGD1 and Geographic Atrophy (GA) in advanced Dry Age-related Macular Degeneration (Dry AMD).

Key Points: 
  • Tinlarebant is Belite Bio’s orally administered tablet intended to slow disease progression in patients affected with STGD1 and Geographic Atrophy (GA) in advanced Dry Age-related Macular Degeneration (Dry AMD).
  • Genetic profiling was performed on the 13 adolescent STGD1 patients enrolled in a Phase 2 study of Tinlarebant.
  • “Data from the genotype and lesion growth analyses from our completed Phase 2 trial continues to demonstrate the efficacy of Tinlarebant,” said Dr. Nathan Mata, CSO of Belite Bio.
  • The presentation is now available in the “Presentations & Events” section of Belite Bio’s website: https://investors.belitebio.com/presentations-events/events.

Important Advances in Oxurion's R&D Program on Geographic Atrophy secondary to AMD

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Lundi, mai 6, 2024
AMD, Geographic atrophy, CRISPR, Brussels Stock Exchange

Leuven, BELGIUM – May 6, 2024 – 6:00 PM CET, Oxurion NV (Euronext Brussels: OXUR), an innovative biopharmaceutical company based in Leuven, today announces important progress in its preclinical program targeting Geographic Atrophy (GA), a severe and underserved form of Age-Related Macular Degeneration (AMD).

Key Points: 
  • Leuven, BELGIUM – May 6, 2024 – 6:00 PM CET, Oxurion NV (Euronext Brussels: OXUR), an innovative biopharmaceutical company based in Leuven, today announces important progress in its preclinical program targeting Geographic Atrophy (GA), a severe and underserved form of Age-Related Macular Degeneration (AMD).
  • This recently launched program has reached the first step with the identification of 50 targets that have the potential to provide better treatment options for GA.
  • The preclinical team successfully already confirmed the potential of several of these targets across various relevant in vitro models.
  • Next step is to continue the in vitro validation of the remaining identified targets and start their evaluation in GA animal models.

Boehringer Ingelheim shares positive results from the first study worldwide in diabetic macular ischemia

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Lundi, mai 6, 2024
Boehringer Ingelheim, Safety, Risk, Tissue, Diabetic retinopathy, Abstract, Research, Ingelheim am Rhein, Stanford School, Light, Principal, Master of Science, Paratriathlon, Geographic atrophy, Pediatrics, DMI, Quality of life, ARVO, DR, Medical device

Boehringer Ingelheim today presented positive data from the HORNBILL Phase I/IIa study of BI 764524, the first ever study exploring a potential treatment for people living with diabetic macular ischemia (DMI).

Key Points: 
  • Boehringer Ingelheim today presented positive data from the HORNBILL Phase I/IIa study of BI 764524, the first ever study exploring a potential treatment for people living with diabetic macular ischemia (DMI).
  • The current standard of care for advanced DR includes intravitreal anti-VEGF treatment or invasive laser treatment.
  • “Retinal non-perfusion is a key driver of vision loss in people living with diabetic retinopathy.
  • However, until the HORNBILL study, retinal non-perfusion has not been explored as a potential treatment target.”
    “Vision loss associated with retinal conditions such as diabetic retinopathy and DMI has a devastating impact on quality of life.

OpRegen® (RG6501) Phase 1/2a Clinical Study 24-Month Visual Acuity Results Featured at 2024 Retinal Cell & Gene Therapy Innovation Summit

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Lundi, mai 6, 2024
Biotechnology, Health, General Health, Pharmaceutical, Optical, Health Technology, Other Science, Research, Genetics, Science, Clinical Trials, AMD, Patient, Cell, MM2, Geographic atrophy, MD, Doctor of Philosophy, OCT, Lineage, Paratriathlon, Degenerative disease, RPE, Atrophy, Therapy, Visual impairment, ELM

The meeting was jointly organized by the Foundation Fighting Blindness and the Oregon Health & Science University Casey Eye Institute .

Key Points: 
  • The meeting was jointly organized by the Foundation Fighting Blindness and the Oregon Health & Science University Casey Eye Institute .
  • The presentation, “OpRegen® Retinal Pigment Epithelium (RPE) Cell Therapy for Patients with Geographic Atrophy (GA): Month 24 Results from the Phase 1/2a Trial,” was presented by David Telander, MD, PhD , Retinal Consultants Medical Group, on behalf of Roche and Genentech , a member of the Roche Group.
  • RG6501 (OpRegen) is a suspension of human allogeneic retinal pigment epithelial (RPE) cells currently in development for the treatment of GA secondary to AMD.
  • OpRegen subretinal delivery has the potential to counteract RPE cell loss in areas of GA lesions by supporting retinal cell health and improving retinal structure and function.

Johnson & Johnson Highlights Commitment to Transform Treatment of Retinal Diseases at ARVO 2024

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Vendredi, mai 3, 2024
AMD, Patient, Abstract (summary), Research, Johnson & Johnson, Collection, Paratriathlon, Abstract, Development, Genetic testing, Retina, Algorithm, Geographic atrophy, Diagnosis, Biomarker, Annual general meeting, ARVO, IRD, Pharmaceutical industry

RARITAN, N.J., May 3, 2024 /PRNewswire/ -- Johnson & Johnson today announced that eight company-sponsored presentations will be featured during the Association for Research in Vision and Ophthalmology (ARVO) 2024 Annual Meeting taking place in Seattle from May 5–9, 2024. The Company's two oral presentations will include one highlighting a real-world analysis on the economic value of early genetic testing in patients with inherited retinal diseases (IRDs), a group of rare eye disorders that can lead to serious vision impairment (Abstract #2154), and the second evaluating the role of automatic deep-learning based algorithms to measure precursors of geographic atrophy (GA), a late-stage and severe form of age-related macular degeneration (AMD) (Abstract #2770).1,2

Key Points: 
  • Johnson & Johnson to debut first-of-its-kind EYE-RD Global Registry, aiming to bridge knowledge gaps for inherited retinal diseases
    RARITAN, N.J., May 3, 2024 /PRNewswire/ -- Johnson & Johnson today announced that eight company-sponsored presentations will be featured during the Association for Research in Vision and Ophthalmology (ARVO) 2024 Annual Meeting taking place in Seattle from May 5–9, 2024.
  • "At Johnson & Johnson, we are committed to preserving vision, harnessing the best science to find solutions for those living with blinding retinal diseases," said Hideo Makimura, M.D., Ph.D., Vice President and Global Head, Specialty Ophthalmology Research and Development, Johnson & Johnson Innovative Medicine.
  • "These data at ARVO 2024 showcase our commitment to bringing innovative new therapies that preserve and restore vision for patients worldwide."
  • A complete listing of the Company-sponsored abstracts being featured at the ARVO Annual Meeting is provided below.