Actinium Pharmaceuticals Inc (NASDAQ:ATNM) stock shot up Wednesday after it said positive FDA discussions for its cancer drug Actimab, for Myelodysplastic Syndrome (MDS), a bone marrow failure disorder, enabled accelerated pivotal trials and broadened the target patient population.
Actinium highlighted the outcome of its discussions with the FDA for the Actimab-MDS program, which resulted in guidance for an accelerated pathway to a pivotal trial after a short dose-finding Phase 1 portion.
The initial proposal to the FDA was to conduct a larger Phase 2 trial prior to a pivotal trial in patients with a TP53 mutation, however, the company was encouraged by the FDA to expand the trial to include all high-risk patients with poor or very poor and complex cytogenetics.
The company said the program will use the ARC Ac-225-lintuzumab for targeted conditioning in combination with a reduced intensity dose of fludarabine and melphalan prior to a bone marrow transplant in patients with high-risk Myelodysplastic Syndrome.
"In our studies to date, we've seen that Ac-225–lintuzumab can achieve remissions with minimal extramedullary toxicities even in patients progressing to acute myeloid leukemia from MDS. Typically, high-risk MDS patients undergoing a bone marrow transplant do poorly with standard conditioning resulting in poor outcomes,” said Actinium's Chief Medical Officer Dr Mark Berger.
As many as 21,000 patients are diagnosed with acute myeloid leukemia yearly in the US and over 350,000 cases of acute myeloid leukemia exist worldwide.
Berger said the goal of the Actimab-MDS program is to "use our ARC AC-225–Lintuzumab to target progenitor cells" with the goal of achieving improved conditioning and bone marrow transplant outcomes, as successful bone marrow transplant is the only potentially curative treatment option for these patients. He said the biotech was building on the data it already had from its CD33 program and was excited to have a quicker pathway to a pivotal trial that targets a larger patient population than originally envisaged.
Actinium's two combination trials with Venetoclax, a BCL-2 inhibitor that was jointly developed by AbbVie and Genentech, will leverage Actimab-A's unique mechanism of action to explore synergies between the two agents for patients with relapsed acute myeloid leukemia.
Actinium shares shot up 4.8% to $0.64.
Contact Uttara Choudhury at [email protected]