Atypical hemolytic uremic syndrome

4DMT Reports First Quarter 2024 Financial Results and Operational Highlights

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星期四, 五月 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.

The United States FDA Awards Orphan Drug Designation (ODD) to NM5072 for Treating Paroxysmal Nocturnal Hemoglobinuria (PNH)

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星期一, 四月 15, 2024
FDA, Pain, Mortality, Nephrology, Inflammation, Monocyte, Cell, Anemia, Alternative complement pathway, Safety, Ophthalmology, Red blood cell, Food, Hematology, Chelsea Handler, Dermatology, Half-life, Atypical hemolytic uremic syndrome, Blood, AP, Hyperlipidemia, Hemoglobinuria, Disease, Patient, Neurology, Fatigue, Platelet, Diagnosis, Infection, Hemolysis, Immune system, PNH, ODD, Quality of life, Chronic pain, LDH, Paroxysmal nocturnal hemoglobinuria, Pharmaceutical industry

The United States Food and Drug Administration (FDA) has awarded orphan drug designation (ODD) to NM5072 for the treatment of anemia in PNH patients.

Key Points: 
  • The United States Food and Drug Administration (FDA) has awarded orphan drug designation (ODD) to NM5072 for the treatment of anemia in PNH patients.
  • CLEVELAND, April 15, 2024 (GLOBE NEWSWIRE) -- NovelMed today announced that the Food and Drug Administration (FDA) has awarded Orphan Drug Designation (ODD) to NM5072, an Alternative Pathway (AP) blocker anti-Properdin antibody, for the treatment of patients with Paroxysmal Nocturnal Hemoglobinuria (PNH).
  • NM5072 is another drug in our pipeline that has received Orphan Drug Designation in the United States.
  • Among these, Paroxysmal Nocturnal Hemoglobinuria (PNH) emerges as a prominent indication for complement blockers heading towards FDA approval.

4DMT Reports Full Year 2023 Financial Results and Operational Highlights

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星期四, 二月 29, 2024
Therapy, Atypical hemolytic uremic syndrome, Chelsea Handler, Fabry disease, Respiratory disease, EMA, Injection, Research, CRISPR, Cystic fibrosis, FDA, Partnership, Inflammation, Retinitis pigmentosa, Security (finance), Overalls, Biopsy, Șaeș, PRISM, Conference, Cash, Diabetic retinopathy, Geographic atrophy, Astellas Pharma, CFTR, CNS, PRIME, AMD, Choroideremia, Patient, Safety, Clinical trial, Angiogenesis, VG, DME, AAV, Vaccine

We currently expect cash and cash equivalents to be sufficient to fund operations into the first half of 2027.

Key Points: 
  • We currently expect cash and cash equivalents to be sufficient to fund operations into the first half of 2027.
  • R&D Expenses: Research and development expenses were $97.1 million for 2023, as compared to $80.3 million for 2022.
  • G&A Expenses: General and administrative expenses were $36.5 million for 2023, as compared to $32.9 million for 2022.
  • Net Loss: Net loss was $100.8 million for 2023, as compared to net loss of $107.5 million for 2022.

Ruxoprubart (NM8074) Scores FDA Orphan Drug Designation for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment

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星期一, 二月 12, 2024
LDH, Infection, FDA, Food, Hemoglobinuria, Paroxysmal nocturnal hemoglobinuria, Alternative complement pathway, ODD, Safety, Atypical hemolytic uremic syndrome, ANCA, Death, Patient, Hyperlipidemia, AP, Diagnosis, AAV, PNH, Chelsea Handler, Hemolysis, Pharmaceutical industry

FDA grants orphan drug designation to Ruxoprubart (NM8074) for the treatment of PNH.

Key Points: 
  • FDA grants orphan drug designation to Ruxoprubart (NM8074) for the treatment of PNH.
  • The FDA has approved phase 1b/Phase II clinical trials for Paroxysmal Nocturnal Hemoglobinuria (PNH), C3 Glomerulopathy (C3G), Atypical Hemolytic Uremic Syndrome (aHUS), and most recently, ANCA vasculitis (AAV).
  • CLEVELAND, Feb. 12, 2024 (GLOBE NEWSWIRE) --  NovelMed today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Ruxoprubart, an alternative pathway (AP) blocker anti-Bb antibody, for the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH).
  • FDA's recognition of Ruxoprubart as an orphan drug for PNH underscores its potential to fulfill a crucial need for individuals grappling with this disease condition.

4DMT Presents Interim Data from 4D-310 INGLAXA Phase 1/2 Clinical Trials for Fabry Disease Cardiomyopathy at WORLDSymposium™ 2024

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星期一, 二月 12, 2024
C102, Atypical hemolytic uremic syndrome, Fabry disease, CPET, VO2, FDA, Quality of life, Cardiovascular disease, Data, GLS, Safety, MCID, Overalls, Death, Patient, Echocardiography, Chelsea Handler, Pharmaceutical industry

The data was presented in a late-breaking session at WORLDSymposium™ 2024 in San Diego, California on Friday, February 9, 2024.

Key Points: 
  • The data was presented in a late-breaking session at WORLDSymposium™ 2024 in San Diego, California on Friday, February 9, 2024.
  • “We are pleased to see 4D-310 continue to consistently demonstrate clinical activity across multiple important cardiac endpoints including cardiac function, exercise capacity and quality of life,” said Robert Kim, M.D., Chief Medical Officer of 4DMT.
  • “Current therapies do not adequately address Fabry-related cardiovascular manifestations, and cardiovascular disease is the most common cause of death in these patients.
  • “No approved therapy has been shown to definitively clear accumulated Gb3 from cardiomyocytes in patients with Fabry disease, which speaks to 4D-310’s highly differentiated profile.

4DMT Reports Third Quarter 2023 Financial Results and Operational Highlights

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星期四, 十一月 9, 2023
PRISM, Ophthalmology, D, European Medicines Agency, Cystic fibrosis, Cystic Fibrosis Foundation, Atypical hemolytic uremic syndrome, Security (finance), Risk, Clinical trial, Patient, Research, DME, Completeness, FDA, Cash, VG, AMD, ASRS, CFTR, AAV, Pharmaceutical industry

“The third quarter of 2023 was another period of tremendous progress across our large market product candidate portfolio,” said David Kirn, M.D., Co-founder and Chief Executive Officer of 4DMT.

Key Points: 
  • “The third quarter of 2023 was another period of tremendous progress across our large market product candidate portfolio,” said David Kirn, M.D., Co-founder and Chief Executive Officer of 4DMT.
  • R&D Expenses: Research and development expenses were $25.1 million for the quarter ended September 30, 2023 as compared to $18.9 million for the third quarter of 2022.
  • G&A Expenses: General and administrative expenses were $9.1 million for the quarter ended September 30, 2023 as compared to $8.1 million for the third quarter of 2022.
  • Net Loss: Net loss was $10.3 million for the quarter ended September 30, 2023, as compared to net loss of $25.7 million for the third quarter of 2022.

Amylyx Pharmaceuticals Appoints Camille L. Bedrosian, MD, as Chief Medical Officer

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星期二, 十一月 28, 2023
General Health, Finance, Health, Professional Services, Pharmaceutical, Health Canada, Paroxysmal nocturnal hemoglobinuria, Safety, Alexion, Executive, Atypical hemolytic uremic syndrome, Clinical trial, FDA, ALS, MBA, CMO, Survival, Retirement, Medication, Drug development, Pharmacovigilance, Pharmaceutical industry, Amylyx Pharmaceuticals, Ultragenyx, MD

Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the “Company”) today announced the appointment of Camille L. Bedrosian, MD, as Chief Medical Officer (“CMO”).

Key Points: 
  • Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the “Company”) today announced the appointment of Camille L. Bedrosian, MD, as Chief Medical Officer (“CMO”).
  • In this role, Dr. Bedrosian will lead global functions in medical affairs, regulatory, safety and pharmacovigilance, biometrics, clinical development, and clinical operations.
  • I’m excited to join Amylyx and contribute to bringing innovative, new treatment options to individuals and communities suffering from neurodegenerative diseases.
  • “We also want to thank our longtime Chief Medical Officer, Dr. Patrick Yeramian, for his many years of dedication and commitment to building Amylyx into the successful company it is today.

4DMT Gains Alignment with FDA on Plan to Lift Clinical Hold on Phase 1/2 INGLAXA Clinical Trial for 4D-310 for Fabry Disease Cardiomyopathy

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星期一, 十月 30, 2023
FDA, AAV, Chelsea Handler, Food, IV, Therapy, Fabry, Risk, Atypical hemolytic uremic syndrome, Safety, Patient, Pharmaceutical industry

“We are delighted to have reached an agreement with the FDA on our proposed plan to address the clinical hold and continue trial development,” said David Kirn, M.D., Co-founder and Chief Executive Officer of 4DMT.

Key Points: 
  • “We are delighted to have reached an agreement with the FDA on our proposed plan to address the clinical hold and continue trial development,” said David Kirn, M.D., Co-founder and Chief Executive Officer of 4DMT.
  • Consistent with the Company’s plans, the FDA subsequently notified the Company of a clinical hold in the U.S.
  • The Asia-Pacific clinical trial program was not placed on clinical hold by any regulatory agency.
  • In addition, the INGLAXA clinical trial protocol has been amended to minimize the risk of aHUS following IV 4D-310 dosing, including requiring the R/S immunosuppressive regimen.

4DMT Acquires Complement Pathway Inhibitor Payload for 4D-175 Product Candidate for Geographic Atrophy

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星期一, 四月 24, 2023
Retina, Injection, Perelman School of Medicine at the University of Pennsylvania, CFH, University, Chelsea Handler, Doctor of Philosophy, Attention deficit hyperactivity disorder predominantly inattentive, AMD, Cubic foot, Inflammation, CMC, Survival, Complement, End organ damage, Factor, University of Pennsylvania, Atypical hemolytic uremic syndrome, GA, AAV, Geographic atrophy, IVT, Patient, Ophthalmology, Therapy, Autoimmunity, Disease, Vaccine, Pharmaceutical industry, R100, Pharmacology

Geographic atrophy is a highly prevalent disease with a significant unmet medical need.

Key Points: 
  • Geographic atrophy is a highly prevalent disease with a significant unmet medical need.
  • According to published estimates, there are over one million individuals with GA in the U.S. alone as of 2022.
  • The first treatment for GA, complement inhibitor pegcetacoplan injection, was approved in the U.S. in February 2023 and is administered by intravitreal (IVT) injection once every 25 to 60 days.
  • Complement Factor H (CFH) is a master regulator of the complement system, functioning as a natural inhibitor of the alternative complement pathway.

4DMT to Present Interim Clinical Data from INGLAXA Phase 1/2 Trials of 4D-310 for Fabry Disease Cardiomyopathy at 19th Annual WORLDSymposium™

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星期四, 二月 16, 2023
AAV, Rheumatology, Atypical hemolytic uremic syndrome, Safety, Patient, Science, Webcast, Chelsea Handler, Stanford University, Fabry disease, Quality of life, Medicine, Pharmaceutical industry, Fabry

The webcast will include a summary of safety and efficacy data, including 12-month cardiac data on imaging, function, and quality of life endpoints.

Key Points: 
  • The webcast will include a summary of safety and efficacy data, including 12-month cardiac data on imaging, function, and quality of life endpoints.
  • In addition, detailed analysis of a cardiac biopsy will be presented.
  • The Company also announced cardiac efficacy data from the 4D-310 INGLAXA Phase 1/2 clinical trials will be presented in a platform presentation at the 19th annual WORLDSymposium™ in Orlando, Florida.
  • Cardiac effects of 4D-310 in adults with Fabry disease in a phase 1/2 clinical trial: Functional, quality of life, and imaging endpoints in patients with 12 months of follow up
    The presentation from WORLDSymposium™ will also be available on the 4D Molecular Therapeutics website under Scientific Presentations: https://4dmoleculartherapeutics.com/technology/scientific-presentations .