Accropeutics´s RIPK1 inhibitor AC-003 capsule has been granted Orphan Drug Designation by the FDA

2023-12-25 11:18:17   views:

Suzhou China, December 25, 2023 —— Accro Bioscience (Suzhou) Limited (Accropeutics) announced that its RIPK1 inhibitor AC-003 capsules for the treatment of acute Graft-versus-Host Disease (aGvHD) has been granted Orphan Drug Designation (ODD) by the US Food and Drug Administration (FDA). 
Orphan Drug, also known as rare disease medicine, refers to drugs used for the prevention, treatment, and diagnosis of rare diseases. The orphan drug designation granted by the FDA applies to rare disease drugs and biologics with less than 200,000 cases per year in the United States, and provides relevant product policy support, including but not limited to tax credits for clinical trial costs, exemption from new drug application fees, and a 7-year market monopoly upon product approval.
Acute Graft versus Host Disease (aGVHD) is a common complication of allogeneic hematopoietic stem cell transplantation (alloHCT), with an incidence of approximately 30-50%, with 14-36% of patients developing III-IV grade aGVHD. aGVHD mainly affects the skin (rash or dermatitis), liver (hepatitis or jaundice), and gastrointestinal tract (abdominal pain or diarrhea). Severe aGVHD, if not controlled in a timely manner, can lead to death or develop into widespread chronic GVHD, affecting the patient's quality of life and long-term survival. The currently available GVHD treatment drugs include corticosteroids and JAK inhibitors (Ruxolitinib), which are strong immune suppressors with serious side effects, including hyperglycemia, hypertension, bone necrosis, and increased risk of opportunistic infections.
About AC-003. According to preclinical research from Accropeutics, AC-003 is a new, non immunosuppressive therapy for the treatment of aGVHD, which helps to control infection compared with the current treatment regimens for GVHD, while retains the graft-versus-leukemia (GVL) effect, and ultimately improves the success rate of transplantation. Phase I clinical data from China and the United States shows that AC-003 has good PK, safety and tolerability. The mechanism of RIPK1 kinase function in aGVHD has been previously published in Blood (
Dr. Xiaohu Zhang, Co-founder and CEO of Accropeutics, said: “We are excited to get the Orphan Drug Designation from the FDA. aGVHD is a terrible complication after hematopoietic stem cell transplantation with limited treatment options. Accropeutics will work diligently to move the program forward and hope AC-003 will one day benefit patients worldwide.”
About Accropeutics
Accropeutics is a clinical-stage biotech company with a core focus on molecular mechanisms of regulated cell death and related pathogenesis in human diseases. The Company has developed a robust portfolio with innovative compounds in various stages spanning from lead optimization to clinical trials. The company's RIPK1 inhibitor AC-003 has completed the Phase 1 clinical trial in China and the United States, and is ready to dose aGVHD patients. The company’s RIPK2 inhibitor AC-101 has finished phase 1 trial in healthy volunteers in Australia with excellent PK and safety profiles. AC-201, a selective TYK2/JAK1 inhibitor with huge potential for treating inflammatory and autoimmune diseases, was approved by HREC and NMPA in May 2023 and September 2023, respectively. Phase 1 data is expected in early 2024. The company has multiple compounds in the PCC and preclinical research and development stages. Accropeutics owns global rights of all its assets with more than 10 patents issued in China, Japan, Korea, US and EU.