Geographic atrophy

Ocugen Announces Positive Data and Safety Monitoring Board Review and Initiation of Enrollment in Medium Dose for OCU410—a Modifier Gene Therapy—in Phase 1/2 ArMaDa Study for Geographic Atrophy

Retrieved on: 
Friday, April 5, 2024
Retina, Disease, Inflammation, Patient, Data, Cohort, Șaeș, Life, Clinical trial, Armada, MPH, Injection, Safety, DSMB, FACS, Vaccine, Lipid metabolism, Geographic atrophy, Oxidative stress, Pharmaceutical industry

Three subjects with geographic atrophy (GA) were dosed in the Phase 1/2 clinical trial to date.

Key Points: 
  • Three subjects with geographic atrophy (GA) were dosed in the Phase 1/2 clinical trial to date.
  • An additional three subjects will be dosed with the medium dose (Cohort 2) and three patients with the high dose (Cohort 3) of OCU410 in the dose-escalation phase.
  • “The DSMB has recommended moving forward to medium dose for dosing subjects with GA,” said Dr. Peter Chang, MD, FACS, DSMB Chair for the OCU410 clinical trial.
  • Phase 1 is a multicenter, open-label, dose-ranging study consisting of three dose levels [low dose (2.5×1010 vg/mL), medium dose (5×1010 vg/mL), and high dose (1.5 ×1011 vg/mL)].

Ocugen Provides Business Update with Certain Financials for the Year Ending 2023

Retrieved on: 
Tuesday, April 2, 2024
Stargardt disease, Disease, Safety, Webcast, Inflammation, Patient, CGMP, Data, Inhalation, Cohort, RP, Life, Bank statement, Investment, Clinical trial, NIAID, Injection, AMD, Vaccine, Nextgen, Geographic atrophy, RHO, FDA, Lipid metabolism, Government, Oxidative stress, Pharmaceutical industry

Completed Cohort 1 dosing for OCU410 and OCU410ST gene therapy clinical studies for geographic atrophy (GA) and Stargardt disease, respectively

Key Points: 
  • Completed Cohort 1 dosing for OCU410 and OCU410ST gene therapy clinical studies for geographic atrophy (GA) and Stargardt disease, respectively
    MALVERN, Pa., April 02, 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 provided a general business update with certain financials for the year ending 2023.
  • During the fourth quarter of 2023, the Company announced its mucosal vaccine candidate, OCU500, was chosen for the multi-billion-dollar NIAID Project NextGen initiative.
  • The first patient was dosed in the Phase 1/2 trials to assess the safety and efficacy of OCU410ST for Stargardt disease in November 2023.
  • Ocugen’s cash, cash equivalents, and investments totaled $39.5 million as of December 31, 2023, compared to $90.9 million as of December 31, 2022.

Akari Therapeutics Reports Full-Year 2023 Financial Results and Recent Highlights

Retrieved on: 
Monday, April 1, 2024
Diagnosis, Civil service commission, Research, AATD, Growth, Akari, C5, Patient, Choroidal neovascularization, Sale, B4, PRV, Mortality, Hot game, Clinical trial, Therapy, Priority review, Safety, NEI, European Commission, ADC, Geographic atrophy, Hematopoietic stem cell transplantation, CNV, Pharmaceutical industry

BOSTON and LONDON, April 01, 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 full year 2023 as well as recent company highlights.

Key Points: 
  • BOSTON and LONDON, April 01, 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 full year 2023 as well as recent company highlights.
  • 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.
  • Akari is continuing to recruit patients into the Part A portion of the Phase 3 clinical trial that has treated 10 patients to date.
  • During 2023, Akari also advanced the long-acting version of nomacopan (PASylated nomacopan) into the final stages of pre-clinical development as a treatment for geographic atrophy (GA).

Annexon Reports Fourth Quarter and Year-End 2023 Financial Results and Key Anticipated Milestones

Retrieved on: 
Tuesday, March 26, 2024
Diagnosis, Research, Brain, Geographic atrophy, Disease, RWE, International, Paratriathlon, Patient, ARCHER, G&A, Pivotal, EMA, Meta-analysis, Eye, European Medicines Agency, Medicine, Visual acuity, Immunoglobulin therapy, GBS, Facial muscles, Clinical trial, POC, Safety, Pharmacokinetics, Investment, SAD, FDA, BLA, AFL, CAD, Food, Pharmaceutical industry

BRISBANE, Calif., March 26, 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 highlighted portfolio progress and reported fourth quarter and full year 2023 financial results.

Key Points: 
  • Finally, we anticipate clinical proof-of-concept data from our novel oral inhibitor ANX1502 later in the year.
  • ANX005 in GBS: First-in-class monoclonal antibody designed to block C1q and the entire classical complement pathway in both the body and the brain.
  • ANX005 in GBS: Topline data from pivotal, randomized, placebo-controlled Phase 3 trial expected in the second quarter of 2024.
  • Cash and operating runway: Cash and cash equivalents and short-term investments were $259.7 million as of December 31, 2023.

Aviceda Therapeutics Announces Formation of Renowned European Clinical Advisory Board

Retrieved on: 
Monday, April 8, 2024
Technology, Research, Nanotechnology, Clinical Trials, Biotechnology, Health, Pharmaceutical, Optical, Science, Faculty, Medical University of Vienna, Part, Patient, Queen's University, Optometry, Queen's University Belfast, CAB, MD, Royal Victoria Hospital, Laborie (company), Medical school, Therapy, Retina, Safety, AMD, Geographic atrophy, Pharmaceutical industry

Aviceda Therapeutics , a private clinical-stage biotech company focused on developing next-generation immunomodulators by harnessing the power of glycobiology to alleviate chronic, non-resolving inflammation, announced today the formation of its European Clinical Advisory Board (CAB) made up of top internationally recognized thought leaders in retina to provide strategic consultation on the company’s clinical development of its lead asset, AVD-104, for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD).

Key Points: 
  • Aviceda Therapeutics , a private clinical-stage biotech company focused on developing next-generation immunomodulators by harnessing the power of glycobiology to alleviate chronic, non-resolving inflammation, announced today the formation of its European Clinical Advisory Board (CAB) made up of top internationally recognized thought leaders in retina to provide strategic consultation on the company’s clinical development of its lead asset, AVD-104, for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD).
  • The Company also announced that Tarek S. Hassan, MD will serve as Chair of the European and US CABs.
  • “We are excited that results from Part 1 of the US Phase 2/3 SIGLEC trial demonstrated functional and anatomic improvement in eyes with GA secondary to AMD after a single injection of AVD-104,” said Mohamed Genead, M.D., Aviceda’s Co-founder and CEO.
  • “With the contributions and support of this esteemed panel—and with Dr. Hassan as its chair—we look forward to bringing our clinical efforts to Europe later this year.”

US Retinal Specialists Highlight the Greatest Opportunity for Gene Therapies

Retrieved on: 
Tuesday, March 19, 2024
Retina, Stargardt disease, Macular edema, Retinitis pigmentosa, Eye, Intelligence, Paratriathlon, Hope, Stargardt, Adverum Biotechnologies, Safety, NAMD, Geographic atrophy, Ophthalmology, Diabetic retinopathy, Medical device

Unlike other organs, the eye's compact size and accessibility have made it an ideal application for gene therapies, offering targeted treatment with minimal systemic impact.

Key Points: 
  • Unlike other organs, the eye's compact size and accessibility have made it an ideal application for gene therapies, offering targeted treatment with minimal systemic impact.
  • As advancements continue, a plethora of gene therapies for retinal disorders are on the horizon.
  • Spherix Global Insights collaborated with 77 retinal specialists to understand their perspectives on adopting gene therapies, specifically where specialists were and were not willing to use gene therapy across diseases and patient types.
  • While retinal specialists express eagerness for gene therapies, dissatisfaction towards current treatments like Syfovre and Izervay underscores the need for innovation.

Clearside Biomedical Appoints Victor Chong, M.D., MBA as Chief Medical Officer

Retrieved on: 
Monday, March 18, 2024
DAS, Retina, University, Moorfields Eye Hospital, Pathology, Oligonucleotide, Ranibizumab, Data analysis, Partnership, Business, Boehringer Ingelheim, DME, Patient, Pharming, Research, Royal College of Ophthalmologists, Cell biology, Diabetic retinopathy, C3, University College London, MBA, Eye, Johnson & Johnson, Janssen, Drug development, John Radcliffe Hospital, AMD, CLS, University of Oxford, Royal college, Geographic atrophy, Stargardt disease, DMI, Pharmaceutical industry

ALPHARETTA, Ga., March 18, 2024 (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 that Victor Chong, M.D., MBA was appointed Chief Medical Officer on March 14, 2024.

Key Points: 
  • ALPHARETTA, Ga., March 18, 2024 (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 that Victor Chong, M.D., MBA was appointed Chief Medical Officer on March 14, 2024.
  • George Lasezkay, Pharm.D., J.D., President and Chief Executive Officer of Clearside, commented, “Victor is a well-known and well-respected retinal clinician and scientist, who is a strategically focused and visionary leader.
  • We are delighted to have him join the Clearside team.
  • “With the upcoming ODYSSEY data and extensive partnership portfolio, this is an exciting time to join Clearside.

Ocugen, Inc. Announces Dosing Completion of Subjects with Geographic Atrophy in Cohort 1 of Phase 1/2 Clinical Trial Evaluating the Safety and Efficacy of OCU410

Retrieved on: 
Wednesday, March 13, 2024
Diagnosis, Vision, Associate, Partnership, Fellow, Inflammation, Patient, Research, Hope, MD, United, Life, Clinical trial, Armada, Macular degeneration, Safety, Vaccine, Lipid metabolism, Geographic atrophy, Pharmaceutical industry

“We are very enthusiastic about the potential of OCU410 as a one-time treatment for life with a single sub-retinal injection,” said Dr. Shankar Musunuri, Chairman, CEO and Co-Founder of Ocugen.

Key Points: 
  • “We are very enthusiastic about the potential of OCU410 as a one-time treatment for life with a single sub-retinal injection,” said Dr. Shankar Musunuri, Chairman, CEO and Co-Founder of Ocugen.
  • “While there are currently two recently approved products for the treatment of GA, both require approximately 6-12 intravitreal injections annually and target only the complement system.
  • OCU410 addresses multiple pathways causing dAMD, including complement, lipid metabolism, inflammation, and oxidative stress.”
    Up to 13 leading retinal surgery centers across the United States are participating in the ArMaDa clinical trial.
  • The enrollment in the first cohort is now complete and 3 subjects received 200µL single subretinal administration of the low dose (2.5x1010 vg/mL) of OCU410.

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

Retrieved on: 
Wednesday, March 13, 2024
Stem Cells, Biotechnology, Health, Philanthropy, Pharmaceutical, Optical, Research, Genetics, Foundation, Science, Clinical Trials, Geographic atrophy, Patient, Visual impairment, Doctor of Philosophy, MD, Hyatt Regency Seattle, Therapy, RPE, AMD, Cell

Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, today announced that 24 month results from patients enrolled in a Phase 1/2a clinical study (ClinicalTrials.gov Identifier: NCT02286089 ) of RG6501 ( OpRegen ) in patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD), will be presented at the 2024 Retinal Cell & Gene Therapy Innovation Summit .

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 announced that 24 month results from patients enrolled in a Phase 1/2a clinical study (ClinicalTrials.gov Identifier: NCT02286089 ) of RG6501 ( OpRegen ) in patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD), will be presented at the 2024 Retinal Cell & Gene Therapy Innovation Summit .
  • The meeting will be held May 3, 2024, at the Hyatt Regency Seattle, and is 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,” will include anatomical and functional data from long-term follow-up in 10 of 12 patients and will be presented by David Telander, MD, PhD , Retinal Consultants Medical Group, on behalf of Roche and Genentech , a member of the Roche Group.

Akari Therapeutics and Peak Bio Announce Definitive Agreement to Merge as Equals Creating an Expanded Pipeline That Features a Novel Antibody Drug Conjugate (ADC) Toolkit

Retrieved on: 
Tuesday, March 5, 2024
Therapy, DLA Piper, Leukotriene B4, NEI, C5, CNV, OTC, Inflammation, Bayer, Choroidal neovascularization, ADC, Risk, AATD, American depositary receipt, Geographic atrophy, Haematopoietic system, Patient, Autoimmunity, McKinsey & Company, Hot game, LTB4, Cancer, Pharmaceutical industry

Following closing, the company will have an expanded pipeline that contains multiple compelling assets spanning early and late development stages.

Key Points: 
  • Following closing, the company will have an expanded pipeline that contains multiple compelling assets spanning early and late development stages.
  • An assessment of the pipeline is planned, including program prioritization, updated timelines, near-term value creation opportunities, and other considerations.
  • Key highlights of the merger include:
    The merged pipeline features a robust ADC toolkit with novel payload and linker technologies.
  • Goodwin Procter LLP is serving as legal advisor to Akari and DLA Piper LLP is serving as legal advisor to Peak Bio.