Forty Seven, Inc. Announces Updated Data from Ongoing Clinical Trial of Magrolimab Showing Robust, Durable Activity in Patients with Myelodysplastic Syndrome and Acute Myeloid Leukemia
-- CR Rate of 50% and
-- Median Duration of Response Not Yet Reached, with Patients on Treatment for More than 6.4 and 8.8 Months for MDS and AML, Respectively --
-- Finalized Design of Clinical Program to Support Potential Accelerated Approval in Higher-Risk MDS --
-- Forty Seven to Host Investor Event and Webcast at 8:00 p.m. ET --
“As a clinician caring for patients with MDS, I am reminded daily of the limitations of existing standard-of-care medicines, which are appropriate for a small fraction of people and leave nearly 75% receiving only supportive care or watchful waiting,” said
“We are very encouraged by the tolerability and sustained clinical activity of the magrolimab and azacitidine combination observed thus far, which increase our confidence in the program and reinforce our commitment to advancing it as rapidly and as responsibly as possible,” 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
Clinical Activity Data
As of the data cutoff, 46 patients were evaluable for response assessment, including 24 patients with untreated higher-risk MDS and 22 patients with untreated AML, who are ineligible for induction chemotherapy.
- In higher-risk MDS, the overall response rate (
ORR) was 92%, with 12 patients (50%) achieving a complete response (CR), eight patients (33%) achieving a marrow CR and two patients (8%) achieving hematologic improvement. Additionally, two patients (8%) achieved stable disease.
- In untreated AML, the
ORRwas 64%, with nine patients (41%) achieving a CR, three patients (14%) achieving a CR with complete blood count recovery (CRi) and one patient (5%) achieving a morphologic leukemia-free state (MLFS). Additionally, seven patients (32%) achieved stable disease (SD) and one patient (5%) had progressive disease.
- The median time to response among MDS and AML patients treated with the combination was 1.9 months.
- No median duration of response or overall survival has been reached for either MDS or AML patients, with a median follow-up of 6.4 months (range 2.0 to 14.4 months) for MDS and 8.8 months (range 1.9 to 16.9 months) for AML.
Additionally, mutational analyses are ongoing to correlate subgroups with response. Seven of nine (78%) evaluable TP53 mutant AML patients achieved an objective response, with 44% achieving CR and 33% achieving CRi. TP53 mutations are often associated with a poor prognosis and patients with TP53 mutant disease are refractory to existing therapies.
Lastly, in AML patients who achieved an objective response, a significant increase in CD4 and CD8 T cell infiltration was observed in the bone marrow while on therapy, demonstrating that magrolimab and azacitidine can induce an adaptive T cell response.
As of the data cutoff, the combination of magrolimab and azacitidine was well-tolerated, with no evidence of increased toxicities compared to azacitidine alone. Adverse events (AEs) were consistent with prior clinical experience. No deaths were observed in the first 60 days on combination treatment and only one patient out of 62 (1.6%) discontinued treatment due to a treatment-related AE.
Clinical Development Plans for Magrolimab in MDS and AML
Forty Seven achieved alignment with the
Forty Seven has amended the protocol for its ongoing Phase 1b clinical trial to evaluate dosing every two weeks, and remains on track to complete enrollment of approximately 90 patients in the third quarter of 2020. ENHANCE, which will initiate in the first half of 2020, will enroll approximately 180 patients, randomized 1:1 to receive magrolimab and azacitidine or azacitidine alone. For both studies, the primary endpoint will be CR rate and duration of response, and either study could serve as the basis for an accelerated approval. ENHANCE will have an adaptive design, such that it can be extended to include additional patients evaluated for a longer period of time for overall survival benefit in support of a potential full approval based on maturing data in the ongoing Phase 1b trial. Additionally, given the decision to design ENHANCE as an RCT with standard, well-validated endpoints, the SPA process is no longer required.
Given the encouraging data observed in patients with TP53 mutant AML and the significant unmet medical need in this population, Forty Seven will continue to enroll additional patients with TP53 mutant disease in the ongoing Phase 1b trial, with potential future regulatory interactions to be considered pending analysis of the maturing data.
Investor Event and Webcast Information
Forty Seven will host an investor event on
Magrolimab 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. Forty Seven is initially developing magrolimab, an investigational medicine, for the treatment of patients with MDS, AML, non-Hodgkin’s lymphoma, ovarian cancer and colorectal carcinoma. Magrolimab has been granted Fast Track designation by the FDA for the treatment of MDS and AML, and for the treatment of relapsed or refractory diffuse large B-cell lymphoma (DLBCL) and follicular lymphoma, two forms of B-cell non-Hodgkin's lymphoma. Magrolimab has also been granted Orphan Drug designation by the FDA and European Medicines Agency for the treatment of AML.
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 “continue,” “could,” “expect,” “may,” “plan,” “potential,” "will," 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 utility of magrolimab in combination with azacitidine for the treatment of patients in MDS and AML, the data and results of Forty Seven’s ongoing Phase 1b clinical trial, the potential responses of patients with higher-risk disease to magrolimab in combination with azacitidine, the tolerability of magrolimab in combination with azacitidine, the potential for accelerated approval, the timing and potential filing of an initial BLA, the success of Forty Seven’s potential registration-enabling clinical development program for magrolimab in higher-risk MDS, continued enrollment in Forty Seven’s ongoing Phase 1b clinical trial, the initiation of a second clinical trial evaluating the combination of magrolimab and azacitidine compared to azacitidine alone, the outcome of future regulatory interactions, the results of clinical trials and the outlook for Forty Seven’s other programs.
Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. The product candidates that Forty Seven develops may not progress through clinical development or receive required regulatory approvals within expected timelines or at all. In addition, clinical trials may not confirm any safety, potency or other product characteristics described or assumed in this press release. Such product candidates may not be beneficial to patients or successfully commercialized. The failure to meet expectations with respect to any of the foregoing matters may have a negative effect on Forty Seven's stock price. Additional information concerning these and other risk factors affecting Forty Seven's business can be found in Forty Seven's periodic filings with the
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Source: Forty Seven, Inc.