Geographic atrophy

Data and Safety Monitoring Board Approves Simultaneous Enrollment in Cohort 3 and Phase 2 Initiation in OCU410 ArMaDa study for Geographic Atrophy

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金曜日, 5月 31, 2024
MPH, Patient, Oxidative stress, Armada, Lipid metabolism, Clinical trial, Safety, Vaccine, FACS, DSMB, Data monitoring committee, Șaeș, Geographic atrophy, CMI, Inflammation, Pharmaceutical industry

Six subjects with GA were dosed in the Phase 1/2 clinical trial to date—three subjects were dosed with the low dose and three subjects were dosed with the medium dose.

Key Points: 
  • Six subjects with GA were dosed in the Phase 1/2 clinical trial to date—three subjects were dosed with the low dose and three subjects were dosed with the medium dose.
  • An additional three patients will be dosed with the high dose of OCU410 in the dose-escalation phase.
  • “No serious adverse events (SAEs) related to OCU410 have been reported to date in both low- and medium-dose cohorts.
  • OCU410 addresses multiple pathways causing dAMD, including complement, lipid metabolism, inflammation, and oxidative stress, providing long-term benefit to patients."

Ocugen to Present at BIO International Convention 2024

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水曜日, 5月 29, 2024
3D, Patient, Armada, Exhibition, Booth, Clinical trial, Stargardt disease, Vaccine, Stargardt, BIO, Geographic atrophy, Retinitis pigmentosa, Cartilage, Pharmaceutical industry

MALVERN, Pa., May 29, 2024 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines, today announced that the Company will present at the 2024 BIO International Convention taking place in San Diego, CA from June 3 – 6, 2024.

Key Points: 
  • MALVERN, Pa., May 29, 2024 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines, today announced that the Company will present at the 2024 BIO International Convention taking place in San Diego, CA from June 3 – 6, 2024.
  • “We are excited to return to BIO this year and showcase the significant advancements we've achieved both clinically and strategically,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen.
  • “BIO is an important opportunity to pursue collaborations that will propel the development of our first-in-class therapies while also enhancing shareholder value.
  • Ocugen’s presentation details are as follows:
    Location: Presentation Theater 1 - Hall A - Exhibition Hall, San Diego Convention Center
    Please visit Ocugen at Booth #3945 to learn more about the Company’s clinical programs and the potential they may hold for patients.

Akari Therapeutics Reports First Quarter 2024 Financial Results and Recent Highlights

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木曜日, 5月 16, 2024
Security (finance), HSCT, Therapy, Optimism, ADC, MD, M.D, Interim, Geographic atrophy, Research

Entered into a Definitive Merger Agreement with Peak Bio; Expected to Close in the Third Quarter of 2024

Key Points: 
  • Entered into a Definitive Merger Agreement with Peak Bio; Expected to Close in the Third Quarter of 2024
    BOSTON and LONDON, May 16, 2024 (GLOBE NEWSWIRE) -- Akari Therapeutics, Plc (Nasdaq: AKTX), a late-stage biotechnology company developing advanced therapies for autoimmune and inflammatory diseases, has reported financial results for the first quarter ended March 31, 2024 as well as recent company highlights.
  • “My first few weeks as Interim CEO of Akari have been filled with non-stop activity and optimism,” said Samir R. Patel, MD, Akari Interim President and CEO.
  • The combined entity will operate as Akari Therapeutics, Plc, which is expected to continue to be listed and trade on the Nasdaq Capital Market as AKTX.
  • The merger is expected to close in the third quarter of 2024.

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

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木曜日, 5月 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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木曜日, 5月 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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水曜日, 5月 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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火曜日, 5月 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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月曜日, 5月 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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月曜日, 5月 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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月曜日, 5月 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.