[Press Release]PharmAbcine's PMC-403 Receives FDA Orphan Drug Designation for Idiopathic Systemic Capillary Leak Syndrome

20 Feb 2024
TitlePharmAbcine's PMC-403 Receives FDA Orphan Drug Designation for Idiopathic Systemic Capillary Leak Syndrome
CategoryPress Release

PharmAbcine's PMC-403 Receives FDA Orphan Drug Designation for Idiopathic Systemic Capillary Leak Syndrome

  • Orphan Drug Designation grant by the U.S. FDA for the treatment of Idiopathic Systemic Capillary Leak Syndrome (ISCLS) marks a significant milestone in addressing a rare and life-threatening disease with unmet medical needs.

PharmAbcine, Inc. ("PharmAbcine" or the "Company") (KOSDAQ: 208340), a clinical-stage public company developing next generation therapeutics to treat medical unmet needs, announced that on February 21st, 2023, PharmAbcine received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for PMC-403 for the treatment of a rare disease, Idiopathic Systemic Capillary Leak Syndrome (ISCLS).

PMC-403 is a novel TIE2 binding and activating human IgG with the mechanism of stabilizing pathological and leaky blood vessels. TIE2 receptors, expressed on endothelial cells, are involved in vessel normalization processes such as angiogenesis and intercellular adhesion. PMC-403 activates the TIE2 receptor to improve vascular structure and stabilize abnormal blood vessels. This function has shown potential to be applicable for the treatment of various diseases caused by pathological vasculature.

The Company conducted research collaboration with Dr. Kirk Druey, Chief of the NIH's Lung and Vascular Inflammation Section, to explore the efficacy of PMC-403 in ISCLS animal models. In this study, PMC-403 demonstrated efficacy in improving survival and alleviating vascular leakage in the mouse model of ISCLS induced by histamine. The study extends to the treatment of mice exposed to influenza, showing promising results in reducing vascular leakage.

ISCLS, or Clarkson Disease, is a severe systemic rare disorder characterized by the sudden and rapid exit of fluids and proteins from blood vessels into surrounding tissues due to the abnormal function of systemic vascular endothelial cells. This leads to symptoms such as hypotension, edema, and hypoalbuminemia, posing a significant threat with a 30% mortality rate upon acute onset.

ODD is a system that encourages the development of treatments for rare diseases or life-threatening diseases with fewer than 200,000 patients in the United States. Through this system, the development of orphan drugs is incentivized by various benefits such as subsidies, tax breaks, and market exclusivity due to the limited patient population, fostering rapid growth in this market. Additionally, the expedited review program for orphan drugs streamlines approval processes, incorporating FDA feedback and enabling faster development through smaller clinical trials.

According to Evaluate Pharma, the global orphan drug market is showing an average annual growth rate of 12%, from approximately $160 billion in 2021 to approximately $280 billion in 2026, and it is expected that orphan drug sales will account for 20% of the total prescription drug market by 2026.

About PharmAbcine Inc.

PharmAbcine is a clinical stage public company developing next generation IgG based therapeutics to treat cancer, neovascular eye diseases, and vascular related unmet needs.

The Company’s main pipeline include clinical assets olinvacimab, PMC-403, and PMC-309.

Olinvacimab, the Company’s lead asset, is ongoing a Phase II trial in combination with MSD’s pembrolizumab for mTNBC patients in Australia to reconfirm the encouraging result from Phase Ib olinvacimab plus pembrolizumab trial, delivering 50% ORR, 67% DCR, and clean safety profile.

PMC-403 is a novel TIE2-activating antibody that stabilizes dysfunctional leaky disorganized pathological vessels and can be used for vascular-related eye disease, including wet AMD (Age-related Macular Degeneration). PMC-403 is currently undergoing Phase I trial for neovascular AMD patients in Korea. PMC-403 is also being explored to expand into wider therapeutic areas related to pathological vessels including vessel-related rare diseases and non-rare diseases.

PMC-309, a novel anti-VISTA-antagonizing IgG in pan pH conditions, is an immune checkpoint regulator that targets MDSC (myeloid derived suppressor cells) and M2 macrophages which play pivotal role in maintaining immunosuppressive TME (Tumor Microenvironment). Phase I is ongoing at multicenter in Australia and Phase Ib/II in combination with MSD’s pembrolizumab PMC-309 + Pembrolizumab combo is in plan.

PMC-005, is an anti-EGFRviii IgG that only binds to EGFRviii expressed on cancer cells and can be applied to various modalities including CAR-T, CAR-NK, CAR-Macrophage, T cell/NK cell engager, and Radio-Immunotherapy.

For additional information about PharmAbcine, visit http://www.pharmabcine.com or follow on Youtube and LinkedIn.

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