Human Anti-C5 Antibody BSI-061T used for paraoxysmal nocturnal hemoglobinuria (PHN) and atypical hemolytic uremic syndrome (aHUS)

Partnering objectives
Seeking a collaborative licensing opportunity with a company that has a strategic interest in the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS)

Field: Treatment of paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS)

Technology and MOA
MOA:a recombinant human monoclonal antibody that binds to the human C5 complement protein with high affinity and prevents cleavage of C5 into C5a and C5b.

C5 antibody binds to terminal complement protein C5, thereby blocking C5 cleavage into pro-inflammatory components and preventing the complement-mediated destruction of red blood cells (RBCs) as seen in paroxysmal nocturnal hemoglobinuria (PNH).

Marketing opportunity
Paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) are devastating medical conditions. There are no effective treatment to date; blood transfusion could improve quality life of patents. Although there have been some important advances in the treatment of these medical conditions with the advent of biological therapies, new treatments are still urgently needed. The current price of biological therapies are so expensive that many patients could not afford them.

Global market for PNH and aHUS should reach $5,335 M in 2025 from $3,563 M in 2018 at a compound annual growth rate (CAGR) of 5.9%, from 2018 to 2025; Global sales for targeting C5 reached $3,563 M in 2018 and estimated to reach $3,682 M in 2025, CAGR 0.5%.

Differentiable treatment with potential to be first/ best in class
BSI-061T is a fully human anti-C5 monoclonal antibody with high affinity to huC5, cynoC5 and mutant huC5R885H proteins and with more potent in vitro efficacy than all four benchmarks –eculizumab (2007), Ultomiris(2018), Crovalimab(Ph-II), and Pozelimab(Ph-III):

Indications
• Atypical Hemolytic Uremic Syndrome (Nondiarrhea – Associated Hemolytic Uremic Syndrome)
• Paroxysmal Nocturnal Hemoglobinuria
• Neuromyelitis Optica (Devic’s Syndrome)
• Myasthenia Gravis

Data
Key parameters of fully human anti-C5 monoclonal antibody BSI-061T as compared to benchmarks (Eculizumab, Ultomiris, Crovalimab, and Pozelimab):
• Exhibits higher affinity to human-C5 than all four benchmarks
• Cross-recognizes both cyno-C5 and human-C5R885H (not seen in Eculizumab/Ultomiris)
• Shows more potent in vitro efficacy than all four benchmarks
• Distinct epitope different from Eculizumab, but overlapped with Pozelimab
• An ideal candidate for next generation anti-C5 inhibitor development in competing with Ultomiris, Crovalimab, and Pozelimab

Intellectual property
For discussion under CDA.