Alternative complement pathway

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.

USAN Approves Generic Name “Ruxoprubart” for NM8074, an Antibody Therapy focused on Complement-Mediated Diseases

Retrieved on: 
Monday, January 8, 2024

Phase I Clinical Trial results in healthy subjects demonstrated the safety and well-tolerance of NM8074.

Key Points: 
  • Phase I Clinical Trial results in healthy subjects demonstrated the safety and well-tolerance of NM8074.
  • Across all cohorts, total Alternative Pathway (AP) inhibition was achieved, with the duration of AP inhibition showing a dose-dependent response.
  • The USAN Council has approved the nonproprietary name "Ruxoprubart" (pronounced Ruk” soe proo’ bart) for NM8074, designating it as a first-in-class Alternative Pathway selective investigational monoclonal antibody.
  • Ruxoprubart, a potent and highly selective investigational drug candidate, targets the complement protein Bb, playing a crucial role in benefiting several complement-mediated diseases.

Omeros’ OMS906 Data Presented at Complement-based Drug Development Summit

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Friday, October 16, 2020

Omeros Corporation (Nasdaq: OMER) presented data from its OMS906 program yesterday at the 4th Complement-based Drug Development Summit.

Key Points: 
  • Omeros Corporation (Nasdaq: OMER) presented data from its OMS906 program yesterday at the 4th Complement-based Drug Development Summit.
  • OMS906 is the companys lead human monoclonal antibody targeting mannan-binding lectin-associated serine protease-3 (MASP-3), the key activator of the complement systems alternative pathway.
  • Data also showed that lowest levels of detectable mature CFD correlated with complete inactivation of the alternative pathway.
  • Omeros expects OMS906 to have broad application in conditions involving inflammation and tissue damage as well as disorders associated with dysregulation of the alternative pathway.

Samsung Biologics signs strategic partnership with Kanaph Therapeutics to develop treatment for retinal diseases

Retrieved on: 
Thursday, September 24, 2020

INCHEON, South Korea, Sept. 23, 2020 /PRNewswire/ -- Samsung Biologics (207940.KS) and Kanaph Therapeutics have entered into a strategic partnership to develop KNP-301, a bi-specific Fc fusion protein intended to treat retinal diseases.

Key Points: 
  • INCHEON, South Korea, Sept. 23, 2020 /PRNewswire/ -- Samsung Biologics (207940.KS) and Kanaph Therapeutics have entered into a strategic partnership to develop KNP-301, a bi-specific Fc fusion protein intended to treat retinal diseases.
  • According to Kanaph Therapeutics, KNP-301 is designed to target autoimmune diseases by inhibiting the alternative pathway of the complement system.
  • With the benefits of its bi-specific approach, it aims to successfully treat complement-mediated and angiogenesis driven retinal diseases by suppressing both C3b and VEGF simultaneously.
  • "We are proud to announce our partnership with Kanaph Therapeutics for the development of their first biologics program," said Dr. Tae Han Kim, CEO of Samsung Biologics.

BioCryst Begins Proof of Concept Trial in PNH Patients With Oral Factor D Inhibitor, BCX9930

Retrieved on: 
Thursday, March 5, 2020

BCX9930 is an oral Factor D inhibitor discovered and developed by BioCryst for the treatment of complement-mediated diseases.

Key Points: 
  • BCX9930 is an oral Factor D inhibitor discovered and developed by BioCryst for the treatment of complement-mediated diseases.
  • The company expects to report data from the proof of concept study in PNH patients in the second quarter of 2020.
  • Because Factor D is essential for alternative pathway overactivity in PNH, and all other complement diseases of the alternative pathway, successful proof of concept data with BCX9930 would enable BioCryst to advance the program across other target indications.
  • In this proof of concept study of the safety and effectiveness of oral BCX9930, up to 16 PNH patients will receive BCX9930 twice daily (BID) for 28 days.