Retina

Orphan designation: Sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate Treatment of long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency, 21/08/2020 Withdrawn

Retrieved on: 
Thursday, April 18, 2024
Eurostat, Diagnosis, Civil service commission, Atrophy, Brain, Enzyme, DSM-IV codes, Cell, Patient, Committee, Knowledge, Mitochondrion, PPAR, Regulation, Fatigue, EMA, IRIS, Eye, Diet, European, Retina, COMP, Heart, Sponsor, European Commission, Hypoglycemia, Safety, LCHAD, Dicarboxylic acid, Marketing, Liver, Medicine, Pharmaceutical industry

Orphan designation: Sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate Treatment of long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency, 21/08/2020 Withdrawn

Key Points: 


Orphan designation: Sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate Treatment of long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency, 21/08/2020 Withdrawn

4BIO Capital Portfolio Company Ascend Acquires Florida-Based GMP Manufacturing Capacity

Retrieved on: 
Tuesday, April 9, 2024
Retina, AAV, QC, Partnership, Patient, Research, Acquisition, CMC, Protein, GMP, Investment, Therapy, San Francisco Bay Area, Retinitis pigmentosa, Pharmaceutical industry

London, UK, 9 April 2024 – 4BIO Capital (“4BIO” or “the Group”), an international venture capital firm unlocking the treatments of the future by investing in advanced therapies and other emerging technologies, today announces that its portfolio company, Ascend Advanced Therapies (Ascend), an end-to-end gene therapy development partner, has acquired the Contract, Manufacturing and Controls (CMC) team and site in Alachua, Florida, from Beacon Therapeutics (Beacon).

Key Points: 
  • London, UK, 9 April 2024 – 4BIO Capital (“4BIO” or “the Group”), an international venture capital firm unlocking the treatments of the future by investing in advanced therapies and other emerging technologies, today announces that its portfolio company, Ascend Advanced Therapies (Ascend), an end-to-end gene therapy development partner, has acquired the Contract, Manufacturing and Controls (CMC) team and site in Alachua, Florida, from Beacon Therapeutics (Beacon).
  • The transaction brings an operational good manufacturing practice (GMP) facility, process and analytical development capabilities, and additional experts to the Ascend team.
  • This key acquisition brings Ascend its first US manufacturing site, a huge milestone for the Company.
  • The facility is highly synergistic to a growing global network of Ascend sites that has been built via critical acquisitions.

Opthea Appoints John Han, PharmD, as VP Medical Affairs

Retrieved on: 
Monday, April 8, 2024
Retina, Amgen, Organization, Regeneron Pharmaceuticals, Doctor of Pharmacy, Chiron Corporation, Patient, ISTA Pharmaceuticals, Bayer, Shore, AMD, Ophthalmology, Pharmaceutical industry

Dr. Han will report to Chief Executive Officer (CEO), Frederic Guerard, PharmD, and will join the Opthea Executive Management Team.

Key Points: 
  • Dr. Han will report to Chief Executive Officer (CEO), Frederic Guerard, PharmD, and will join the Opthea Executive Management Team.
  • Dr. Han brings over 20 years of experience building medical affairs programs and partnering across organizations to lead pre- and post-launch initiatives for medicines in ophthalmology and retinal disease.
  • Over the course of his career, Dr. Han has served in senior leadership positions in medical and scientific affairs at leading biopharmaceutical companies.
  • He joins Opthea from Adverum Biotechnologies, Inc. where he led the development of the Medical Affairs function.

Inozyme Pharma Announces Positive Topline Data from Ongoing Phase 1/2 Trials of INZ-701 in Adults with ABCC6 Deficiency (PXE) and ENPP1 Deficiency

Retrieved on: 
Monday, April 8, 2024
Patient, Cerebrovascular disease, Stroke, Accelerated approval (FDA), European Medicines Agency, Doctor of Philosophy, Osteomalacia, PROMIS, Therapy, Conditional, Natural history, CTX, Pros, ADAS, Risk, DSM-IV codes, Cohort, Aes, Alkaline phosphatase, Rickets, EMA, PXE, Safety, Epilepsy, Clinical trial, Food, Hospital, Disability, BSAP, Cardiovascular disease, Disease, PD, Paresis, Consultant, Hypophosphatemia, Fatigue, Survivor, Sclera, FDA, Observational study, Pyrophosphate, Choroid, Incidence, Retina, Biomarker, Life, Event, Medical imaging

BOSTON, April 08, 2024 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY) (“the Company” or “Inozyme”), a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of pathologic mineralization and intimal proliferation, today announced positive topline safety, pharmacokinetic (PK), pharmacodynamic (PD) and exploratory efficacy data from the Company’s ongoing Phase 1/2 clinical trials of INZ-701 in adults with ABCC6 Deficiency (PXE, pseudoxanthoma elasticum) and ENPP1 Deficiency.

Key Points: 
  • “We are excited by the excellent safety and preliminary efficacy profile of INZ-701 in adults with ABCC6 Deficiency,” said Douglas A. Treco, Ph.D., CEO of Inozyme Pharma.
  • The patients were assigned to three dose cohorts of INZ-701: 0.2 mg/kg (n=3), 0.6 mg/kg (n=3), and 1.8 mg/kg (n=4).
  • For trial design details, please see the section entitled “INZ-701 in ABCC6 Deficiency Phase 1/2 Clinical Trial Design” below.
  • For trial design details, please see the section entitled “INZ-701 in ENPP1 Deficiency Phase 1/2 Clinical Trial Design” below.

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)].

Opthea to Participate in the 23ʳᵈ Annual Needham Virtual Healthcare Conference 

Retrieved on: 
Thursday, April 4, 2024
Retina, Conference, Pharmaceutical industry

MELBOURNE, Australia and PRINCETON, N.J., April 04, 2024 (GLOBE NEWSWIRE) -- Opthea Limited (ASX/NASDAQ: OPT “Opthea”, the “Company”), a clinical-stage biopharmaceutical company developing novel therapies to treat highly prevalent and progressive retinal diseases, including wet age-related macular degeneration (wet AMD), today announced that management will present at the 23rd Annual Needham Virtual Healthcare Conference and participate in one-on-one investor meetings on April 11, 2024.

Key Points: 
  • MELBOURNE, Australia and PRINCETON, N.J., April 04, 2024 (GLOBE NEWSWIRE) -- Opthea Limited (ASX/NASDAQ: OPT “Opthea”, the “Company”), a clinical-stage biopharmaceutical company developing novel therapies to treat highly prevalent and progressive retinal diseases, including wet age-related macular degeneration (wet AMD), today announced that management will present at the 23rd Annual Needham Virtual Healthcare Conference and participate in one-on-one investor meetings on April 11, 2024.
  • 23rd Annual Needham Virtual Healthcare Conference Presentation Details:

Opthea Appoints Sujal Shah to the Board of Directors

Retrieved on: 
Wednesday, April 3, 2024
MB, Retina, Audit, Patient, Gilead Sciences, Therapy, AMD, Management

MELBOURNE, Australia and PRINCETON, N.J., April 03, 2024 (GLOBE NEWSWIRE) -- Opthea Limited (ASX/NASDAQ: OPT “Opthea”, the “Company”), a clinical-stage biopharmaceutical company developing novel therapies to treat highly prevalent and progressive retinal diseases, including wet age-related macular degeneration (wet AMD), today announced the appointment of Sujal Shah to the Company’s Board of Directors (the “Board”), effective April 4, 2024.

Key Points: 
  • MELBOURNE, Australia and PRINCETON, N.J., April 03, 2024 (GLOBE NEWSWIRE) -- Opthea Limited (ASX/NASDAQ: OPT “Opthea”, the “Company”), a clinical-stage biopharmaceutical company developing novel therapies to treat highly prevalent and progressive retinal diseases, including wet age-related macular degeneration (wet AMD), today announced the appointment of Sujal Shah to the Company’s Board of Directors (the “Board”), effective April 4, 2024.
  • Concurrent with his appointment as non-Executive Director, Mr. Shah will also become Chairman of the Audit and Risk Committee.
  • Mr. Shah is an accomplished biopharmaceutical executive with extensive leadership and product development experience and a track record in capital formation that complements the deep expertise in retinal disease, especially wet AMD, of the Opthea Board.
  • “We are delighted to welcome Sujal to our Board of Directors as we rapidly advance the registrational program for sozinibercept in wet AMD and prepare for key upcoming clinical, regulatory and commercial milestones,” said Jeremy Levin, D.Phil, MB BChir, Chairman of the Opthea Board of Directors.

KALA BIO Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Corporate Update

Retrieved on: 
Friday, March 29, 2024
Retina, Chair, Three Months, Biologics license application, Secretome, Disease, Infection, Incidence, Paratriathlon, Inflammation, Patient, Sale, Limbal stem cell, Research, CIRM, Acquisition, Commercial, Eye, Safety, Retinitis pigmentosa, Food, FDA, BLA, Stargardt disease, Physician, Contingency, Protease, Cryptocurrency

ARLINGTON, Mass., March 29, 2024 (GLOBE NEWSWIRE) -- KALA BIO, Inc. (NASDAQ:KALA), a clinical-stage biopharmaceutical company dedicated to the research, development and commercialization of innovative therapies for rare and severe diseases of the eye, today reported financial results for the fourth quarter and full year ended December 31, 2023 and provided a corporate update.

Key Points: 
  • “In 2023, we focused on clinical execution, advancing our Phase 2b CHASE trial of KPI-012 for the treatment of PCED.
  • In December 2023 and March 2024, KALA announced private placement financings with an institutional investor, priced at-the-market under Nasdaq rules.
  • Cash Position: As of December 31, 2023, KALA had cash and cash equivalents of $50.9 million, compared to $56.1 million as of September 30, 2023.
  • Operating Loss: For the quarter ended December 31, 2023, loss from operations was $9.6 million, compared to $10.3 million for the same period in 2022.

Opus Genetics Announces Completion of Dosing in First Cohort of Phase 1/2 Trial of Gene Therapy OPGx-LCA5 in Patients with Rare Inherited Retinal Disease LCA5

Retrieved on: 
Tuesday, March 26, 2024
Opus, Retina, Leber congenital amaurosis, University of Pennsylvania, VR, Genetics, LCA5, University, Patient, Ophthalmology, LCA, Penn Presbyterian Medical Center, Pharmaceutical industry

RESEARCH TRIANGLE PARK, N.C., March 26, 2024 (GLOBE NEWSWIRE) -- Opus Genetics, a patient-first, clinical-stage gene therapy company developing treatments for inherited retinal diseases, today announced that the first cohort has completed dosing in its open-label, dose-escalation Phase 1/2 clinical trial evaluating the subretinal delivery of OPGx-LCA5, an adeno-associated virus 8 (AAV8) vector designed to precisely deliver a functional LCA5 gene to the outer retina in patients with Leber congenital amaurosis (LCA) resulting from biallelic mutations in the LCA5 gene (LCA5).

Key Points: 
  • Based on positive safety and efficacy data from the first cohort of three adult patients, the Company will advance OPGx-LCA5 into the next highest dose.
  • Opus anticipates initiating the next cohort mid-2024.
  • There are also future plans to expand the study population to include subjects 13 years or older.
  • “Based on these early clinical data, we’re excited for the potential of OPGx-LCA5 to transform the lives of patients affected by LCA5,” said Ben Yerxa, Ph.D., chief executive officer of Opus.

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.”