https://www.targetedonc.com/view/fda-grants-orphan-drug-designation-to-ab001-for-pancreatic-cancers-and-amlAccording to an announcement by Agastiya Biotech and Vopec Pharmaceuticals, AB001 has been granted orphan drug designation (ODD) by the FDA for the treatment of patients with pancreatic cancer and acute myeloid leukemia (AML).

In a press release, Baskaran Pillai, MD, founder of Agastiya Biotech, highlighted the limitations of traditional chemotherapy drugs, which can harm normal cells along with tumor cells, leading to weakened health and energy in patients. However, AB001’s preclinical studies have demonstrated its ability to selectively kill cancer cells without harming normal cells. The treatment also targets cancer stem cells, disrupting drug resistance and relapse mechanisms.

AB001 is a small molecule PD-L1 checkpoint inhibitor that specifically targets aberrations in intracellular signaling pathways within the tumor microenvironment, including Wnt/beta-catenin, PI3K, and RAS.

Promising results have been observed in preclinical animal models, where AB001 has shown efficacy in treating solid and liquid tumors, including non-PDL1-positive cancers. The research indicates that AB001 has the potential to target beta-catenin, which is currently considered difficult to address with drugs.

AB001 appears to hold advantages over standard-of-care chemotherapy agents like gemcitabine in the treatment of pancreatic cancer. Additionally, preclinical models of AML have demonstrated AB001’s anti-leukemia activity, potentially through inhibiting VEGFR2-mediated signaling pathways.

MG Dinesh, chief scientific officer at Agastiya Biotech, reported on the preventative efficacy of AB001 in various cancer cell lines. Prophylactic studies revealed that AB001 can prevent oncogenes from mutating into tumors and maintain the active state of tumor suppressor genes after exposure to a chemical carcinogen.

FDA’s orphan drug designation supports the development of drugs for rare diseases. Developers of drugs with ODD may receive incentives from the FDA, including tax credits for qualified clinical trials of AB001, exemption from user fees, and the potential for seven years of market exclusivity upon approval.

Source : https://www.targetedonc.com/view/fda-grants-orphan-drug-designation-to-ab001-for-pancreatic-cancers-and-aml