On April 3, 2024 Vopec Pharmaceuticals and Agastiya Biotech reported topline results from the Phase 1 study of AB001, an investigational small molecule, PDL1 immunotherapy for patients with metastasized and locally advanced solid tumors (Press release, Agastiya Biotech, APR 3, 2024, View Source [SID1234641730]).
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Unlike current standard of care immuno- and chemotherapy drugs AB001’s novel immunotherapy mechanism of action selectively destroys tumors without harming healthy cells.
Study Design
A total of 33 patients [3+3 Design], 7 with breast cancer, 5 with ovarian cancer, and 21 with other solid tumors, received AB001. AB001 was administered orally twice daily (at BID doses ranging from 40 to 800 mg) in patients with advanced or metastatic solid tumors. The study’s primary and secondary objectives were safety and efficacy. Safety parameters include MTD, PK, and other standardized toxicology protocols. The efficacy study included investigator-evaluated anti-tumor activity (PD) and biomarker responses correlated with pre- and post-treatment PET-CT and mRNA expression studies.
Toxicity Study
The Phase 1 study showed that AB001 was well-tolerated. The adverse events (AEs) were mild to moderate in severity. No dose-limiting toxic (DLT) effects or treatment-related deaths were reported. Treatment-related adverse events occurred in 9 patients at an instance rate of 2.9%; the most common adverse events were 5 patients with diarrhea (1%), 3 patients with vomiting (1%), and 1 patient with fatigue. Six adverse events were graded as mild in severity, and 3 adverse events were found to be moderate in severity. On investigation, the AEs were determined to be of unrelated causes to the treatment drug and resolved with medications, including over-the-counter medicines.
Efficacy Study
AB001 met secondary efficacy endpoints and demonstrated clinical benefit. The 14-day dose escalation studies showed notable improvements at all dosages in tumor biomarkers, immune markers, and tumor size, as confirmed by 21-day PET SCANs. Among patients with solid tumors, a favorable response was observed in 63.64%, a stable response in 19.39%, and disease progression in 16.36%. Partial responses (PR) were also observed in patients with other solid tumors. Serial assessment of tumor biomarkers showed a significant reduction from baseline. CA19.9 showed a 42% reduction (p=0.2), compared to a 38.4% drop in the CA125 markers (p=0.9), and the overall CEA markers revealed a reduction of 58.6% (p=0.6). mRNA studies showed significant changes in the expression of immunomodulation genes and Treg genes, which were associated with the anti-tumor response of AB001.
The superfast treatment impact within 21 days is attributed to the femto-scale metabolism and tumor DNA damage. MiRNA ad mRNA degradation add to the efficacy of the drug.
AB001 is the first Anti-cancer molecule to be released in India by Vopec Pharmaceutical with R&D and clinical collaboration with US-based Agastiya Biotech. Phase 2 is anticipated to begin in May 2024 in India.
The US FDA has awarded Orphan Drug Designation for AB001 for pancreatic, glioblastoma multiforme (GBM), and acute myeloid leukemia (AML) cancer indications. The companies will present the AB001 Phase 1 data at a future scientific forum.