Antisoma’s novel leukaemia treatment fast-tracked by FDA
Cancer focused biotechnology company, Antisoma (LON:ASM, OTC: ATSMY) told investors that its novel leukaemia treatment – the AS1413 DNA intercalator – has been granted a Fast Track designation by the US Food and Drug Administration (FDA).
The news has been welcomed by investors as the company’s shares advanced over 6.5% on the London Stock Exchange this morning.
FDA Fast Track designated drugs ordinarily qualify for Priority Review - an expedited review process available to drugs that offer major advances in treatment or provide a treatment where no adequate therapy exists. The novel DNA intercalator, AS1413 (amonafide L-malate), is being developed for the treatment of secondary acute myeloid leukaemia (secondary AML).
“This drug could represent a major advance in the options available to patients with secondary AML, and we look forward to completing the ongoing phase III trial and sharing the data with FDA and other regulators", Antisoma Chief Executive Glyn Edwards commented. "We're very pleased to have gained FDA Fast Track status for AS1413”.
The FDA's Fast Track programme is designed to facilitate the development of new drugs that have shown the potential to address an unmet medical need in a serious or life-threatening disease. The drug already has orphan drug status in both the US and the EU for the treatment of AML.
Author(s): J. E. Rosenberg, H. A. Drabkin, P. Lara Jr., A. L. Harzstark, R. A. Figlin, G. W. Smith, F. Erlandsson, D. A. Laber; Dana-Farber Cancer Institute/Harvard Medical School, Boston, MA; Medical University of South Carolina, Charleston, SC; University of California, Davis, Sacramento, CA; University of California, San Francisco, San Francisco, CA; City of Hope, Duarte, CA; St. Francis Hospital, Beech Grove, IN; Antisoma Research Ltd., London, United Kingdom; James Graham Brown Cancer Center, University of Louisville, Louisville, KY
Abstract:
Background: AS1411 is a DNA aptamer that targets nucleolin. Although normally present in the nucleolus, nucleolin is overexpressed and shows localization to the plasma membrane in renal and other cancer cells. A dose-escalating phase I trial of AS1411 monotherapy reported 1 complete response (CR) and 1 partial response (PR) among 12 patients with advanced RCC. A randomized phase II trial in AML reported increased CR rates when AS1411 was added to high-dose cytarabine. Methods: This phase II single-arm study evaluated AS1411 monotherapy in patients with metastatic, predominantly clear cell, RCC who had failed, or shown intolerance to, =1 prior treatment, including a tyrosine kinase inhibitor. AS1411 was administered at 40 mg/kg/day CI days 1-4 of a 28 day cycle for 2 cycles. Response evaluation using RECIST occurred every 8 weeks from the start of therapy until disease progression (performed by investigators and by an independent radiologist). The primary endpoint was response rate (CR+PR); progression-free survival (PFS), duration of response, and safety were secondary endpoints. Pharmacokinetic (PK) analysis was performed. Results: 35 patients were enrolled and treated; the median no. prior therapies was 2 (range 1-7). 33 completed 2 cycles of treatment. Independent response assessment indicated 1 PR (3%) and 21 cases of stable disease (SD; 60%); investigator assessment indicated 1 PR (3%) and 12 SD (34%). The patient achieving a PR exhibited a decreased sum of unidimensional target lesion measurements of = 80%, and remains in PR at 5.9 months. Independently assessed median PFS was 3.9 months. No = grade 4 adverse events were recorded; fatigue and constipation were the most common grade 1-3 adverse events occurring in 29% and 34% of patients, respectively. No other toxicity was observed in more than 10% of patients. PK analysis demonstrated a median steady-state plasma concentration of 21 µg/mL, which is similar to the IC50 concentration identified for renal cancer cell lines in vitro. Conclusions: AS1411 has activity in RCC with minimal toxicity; PFS was comparable to that seen with active agents in the refractory setting. Further studies are needed to determine the optimal dosing and scheduling for this novel therapeutic.
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