Atypical hemolytic uremic syndrome

4DMT Reports Full Year 2023 Financial Results and Operational Highlights

Retrieved on: 
Thursday, February 29, 2024

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

Retrieved on: 
Monday, February 12, 2024

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

Retrieved on: 
Monday, February 12, 2024

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

Retrieved on: 
Thursday, November 9, 2023

“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

Retrieved on: 
Tuesday, November 28, 2023

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

Retrieved on: 
Monday, October 30, 2023

“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

Retrieved on: 
Monday, April 24, 2023

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™

Retrieved on: 
Thursday, February 16, 2023

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 .

4D Molecular Therapeutics Announces Updates on Clinical Pipeline and Additional Preclinical Programs

Retrieved on: 
Monday, January 9, 2023

The doses to be evaluated in DME are expected to be similar to those used in the 4D-150 wet AMD clinical trial.

Key Points: 
  • The doses to be evaluated in DME are expected to be similar to those used in the 4D-150 wet AMD clinical trial.
  • It is estimated that there are over one million individuals with GA in the United States according to published data.
  • In addition, I’m excited by the potential of 4D-175, the new preclinical product candidate utilizing the same R100 vector.
  • Cardiac clinical endpoint data (MRI, echocardiography, cardiopulmonary exercise testing [CPET] and QOL assessment) from evaluations at baseline and 12 months after treatment were assessed.

Worldwide Rare Kidney Diseases Industry Report to 2035 - Key Market Trends and Forecasts - ResearchAndMarkets.com

Retrieved on: 
Monday, December 5, 2022

The "Rare Kidney Diseases Market by Target Indications, Type of Molecule, Route of Administration and Key Geographies: Industry Trends and Global Forecast, 2022-2035" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Rare Kidney Diseases Market by Target Indications, Type of Molecule, Route of Administration and Key Geographies: Industry Trends and Global Forecast, 2022-2035" report has been added to ResearchAndMarkets.com's offering.
  • This report features an extensive study of the current market landscape and the likely future potential associated with the rare kidney diseases market, over the next decade.
  • The term rare kidney diseases (RKD) represents around 150 different indications.
  • It is worth highlighting that the prevalence rate of rare kidney diseases is estimated to be 60-80 per 100,000 cases / individuals in the US and Europe.