FDA Grants Orphan Drug Designation to AB001 for Pancreatic Cancers and AML

The FDA has granted orphan drug designation to AB001 for the treatment of patients with pancreatic cancer and acute myeloid leukemia.

The FDA has granted orphan drug designation (ODD) to AB001 for the treatment of patients with pancreatic cancer and acute myeloid leukemia, according to an announcement by Agastiya Biotech and Vopec Pharmaceuticals.1

"Chemo drugs are efficient in killing tumor cells. However, due to its high toxicity, it also ends up harming normal cells, weakening the patient's health and energy. AB001 preclinical studies have proved that it is able to kill cancer cells without harming normal cells. Additionally, AB001 targets cancer stem cells, thereby disrupting drug resistance and relapse mechanisms,” said Baskaran Pillai, MD, founder of Agastiya Biotech, in a press release.

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

In preclinical animal models, AB001showed success in treating solid and liquid tumors as well as non-PDL1-positive cancers. The preclinical research demonstrates the potential of AB001 to target the beta-catenin, which is currently considered to be an undruggable target.

In pancreatic cancers, the agent appears to have an advantage over standard-of-care chemotherapy agents like gemcitabine. Moreover, AB001 anti-leukemia activity in preclinical models of AML, potentially by inhibiting VEGFR2-mediated signaling pathways.

"On the advice of Dr. Pillai, I researched the preventative efficacy of AB001 on 5 different cancer cell lines. The prophylactic studies show that AB001 prevents various oncogenes from becoming mutated into tumors and maintains the tumor suppressor genes in the active state after induction with a chemical carcinogen," said MG Dinesh, chief scientific officer, Agastiya Biotech, in a statement.

ODDs from the FDA support the development of drugs that can prevent, diagnose, or treat a rare disease or condition. With an ODD, the developer is qualified to receive incentives from the FDA including Tax credits for qualified clinical trials of AB001, exemption from user fees, and a potential 7 years of market exclusivity after approval.2


1. Agastiya Biotech receives FDA orphan drug designation for AB001 for pancreatic cancer and acute myeloid leukemia (AML). News release. May 3, 2022. Accessed May 4, 2022. https://prn.to/3OVhziu

2. Designating an orphan product: Drugs and biological products. FDA website. Updated September 7, 2021. Accessed May 4, 2022. https://bit.ly/3MNcmr5

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