Forty Seven, Inc. Announces Updated Initial Data from Phase 1b Clinical Trial of 5F9 in Patients with Myelodysplastic Syndrome and Acute Myeloid Leukemia
-- 5F9 Well Tolerated with Clinical Activity as Monotherapy and in Combination with Azacitidine --
-- CR Rate of 55% and ORR of 100% Observed in Patients with Higher-Risk MDS --
-- Received FDA Feedback Suggesting Single Arm Pivotal Trial May Support Registration of 5F9 in Combination with Azacitidine in Higher-Risk MDS --
-- Forty Seven to Host Investor Event and Webcast at
“These new data for 5F9 show encouraging clinical activity in a broad population of patients with MDS and AML, who may be unfit for existing therapeutic options or at higher risk for developing rapidly-advancing disease,” said
“We are extremely pleased by the tolerability and clinical activity observed for 5F9 in combination with azacitidine to date, which validates the approach of combining 5F9 with other therapeutic agents that induce prophagocytic ‘eat me’ signals on tumor cells. This supports our belief in the potential of 5F9 as an immune checkpoint inhibitor,” said
Data from the Ongoing Phase 1b Clinical Trial
Forty Seven’s Phase 1b trial, which is being funded in part by the
As of the data cutoff of
Preliminary Safety Data
As of the data cutoff, 5F9 was well-tolerated both as a monotherapy and in combination with azacitidine, with no evidence of increased toxicities compared to azacitidine alone. Adverse events (AEs) were consistent with what has been previously seen with 5F9, and no significant cytopenias or autoimmune-related AEs were observed in patients treated with monotherapy 5F9. Overall, the most commonly reported treatment-related AEs were expected CD47-mechanism-based effects on red blood cells, which led to a temporary and reversible anemia, and many patients in the combination cohort experienced a hemoglobin improvement over the course of their treatment with a decrease in transfusions. Importantly, no treatment-related infections were observed, and only one patient out of 36 treated with the combination experienced neutropenic fever (3%). No deaths were observed in the first 60 days on combination treatment. Only one patient out of 46 (2%) discontinued treatment due to a treatment-related AE.
Preliminary Clinical Activity Data
As of the data cutoff, 35 patients were evaluable for response assessment, including 25 patients with untreated higher-risk MDS or AML who were treated with 5F9 and azacitidine (11 patients with higher-risk MDS and 14 patients with untreated AML) and 10 patients with r/r MDS or AML who were treated with monotherapy 5F9.
- In higher-risk MDS, the overall response rate (ORR) for the combination was 100%, with six patients (55%) achieving a complete response (CR), four patients (36%) achieving a marrow CR and one patient (9%) achieving hematologic improvement.
- In untreated AML, the ORR for the combination was 64%, with five patients (36%) achieving a CR, two patients (14%) achieving a complete response with incomplete blood count recovery (CRi), and two patients (14%) achieving a morphologic leukemia-free state (MLFS). Additionally, five patients (36%) achieved stable disease (SD).
- In r/r MDS or AML treated with monotherapy 5F9, the ORR was 10%, consisting of one patient who achieved a MLFS. Additionally, seven patients (70%) achieved SD.
- The median time to response among MDS and AML patients treated with the combination was 1.9 months.
- Six patients (30% of responders) receiving the combination who had an objective response have experienced deepening responses over time resulting in complete remissions. Five patients (25% of responders) have also successfully received allogeneic stem cell transplants.
- Historical response rates for single-agent azacitidine, show CR rates of approximately 15-20% in higher-risk MDS and untreated AML patients, with initial responses generally occurring after 4-6 months in most patients who respond.1
1 Azacitidine US package insert, Al-Ali et al., 2012; Dombretet al., 2015; Fenauxet al., 2010; Silverman et al., 2002
Clinical Development Plans for 5F9 in MDS and AML
Based on the favorable safety profile and encouraging clinical activity observed in this Phase 1b clinical trial to-date, expansion cohorts have been initiated in patients with both higher-risk MDS and untreated AML with 5F9 in combination with azacitidine.
Additionally, based on feedback from a Type B meeting with the
Investor Event and Webcast Information
Forty Seven will host an investor event on
5F9 is a monoclonal antibody against CD47 that is designed to interfere with recognition of CD47 by the SIRPα receptor on macrophages, thus blocking the "don't eat me" signal used by cancer cells to avoid being ingested by macrophages.
Forward Looking Statements
Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "may," "will," "expect," "anticipate," "estimate," "intend," “potential,” “believe” and similar expressions (as well as other words or expressions referencing future events, conditions, or circumstances) are intended to identify forward-looking statements. These statements include those related to the potential of 5F9 as a monotherapy and in combination with azacitidine for the treatment of MDS and AML; the potential of 5F9 as a tolerable treatment option for patients with MDS and AML as a monotherapy and in combination; the effectiveness of the Company’s dosing strategy to mitigate on-target anemia; the overall advancement of 5F9 in clinical trials; the likelihood of receiving
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Source: Forty Seven, Inc.