Syndax Highlights Leadership in Menin Inhibition at ASH
– First real-world evidence for a menin inhibitor shows 77% ORR (10/13), 31% (4/13) CR/CRh, 75% (9/12) MRD negativity, and favorable tolerability among primarily R/R NPM1m, KMT2Ar, and NUP98r acute leukemia pts –
– Retrospective review shows revumenib was well tolerated as post-HSCT maintenance in children with KMT2Ar and NUP98r; all pts were alive and 90% (9/10) were relapse free at median follow-up of 19 months –
– Phase 2 SAVE trial of revumenib with venetoclax/HMA in newly diagnosed NPM1m and KMT2Ar AML shows 86% ORR (18/21), 76% CR (16/21), and 100% MRD negativity among responders –
– Phase 1 trial of revumenib with intensive chemotherapy in newly diagnosed NPM1m and KMT2Ar AML pts shows favorable tolerability and robust activity, including 92% CRc (24/26) –
NEW YORK, — Syndax Pharmaceuticals (Nasdaq: SNDX), a biopharmaceutical company focused on innovative cancer therapies, has showcased key findings for Revuforj (revumenib) at the recent American Society of Hematology (ASH) Annual Meeting. Syndax, along with its collaborators, presented a total of 12 Revuforj abstracts during this prominent event. Revuforj is a first-in-class, FDA-approved menin inhibitor administered orally.
“We are excited to share new data, including the first real-world evidence for this class, showing that revumenib demonstrates high efficacy with a favorable safety profile in various acute leukemia subtypes and treatment settings, including in combination with established therapies. Notably, our datasets revealed deep responses in R/R and frontline NPM1m and KMT2Ar acute leukemia, along with encouraging safety and preliminary efficacy in post-HSCT maintenance,” stated Nick Botwood, MBBS, Head of Research & Development and Chief Medical Officer at Syndax. “With the initiation of pivotal frontline trials earlier this year for patients unsuitable for intensive chemotherapy, and the commencement of our pivotal frontline trial for fit patients, we believe Syndax is well prepared to expand the clinical data supporting revumenib while leading the charge in the frontline treatment arena with such an innovative therapy.”
Overview of Key Revumenib Data Presented
Real-World Experience with Revuforj
Data was shared from a single-center retrospective review involving 17 patients treated with Revuforj outside of a clinical trial. The median age of patients was 54 years (ranging from 23 to 79). Among them, 47% had KMT2Ar, 29% had NPM1m, and 18% had NUP98r acute leukemia. The cohort had undergone a median of four prior lines of therapy, including 71% with prior venetoclax and 35% with prior hematopoietic stem cell transplant (HSCT).
Revuforj was administrated as part of combination therapy in 82% of cases, most commonly alongside venetoclax and a hypomethylating agent. An overall response rate (ORR) of 77% was noted, with a composite complete remission rate of 62% and 31% achieving CR/CRh. Additionally, 75% of patients were MRD negative at their best response. Of those treated, four patients were able to proceed with HSCT after undergoing revumenib therapy.
These real-world findings reinforce the safety and tolerability profile of Revuforj in clinical practice, with only 24% of patients experiencing Grade 3 or 4 non-hematological adverse events. Remarkably, no differentiation syndrome or QTc prolongation beyond Grade 3 was recorded, showing Revuforj to be a safe alternative for heavily pretreated patients.
Revumenib for Pediatric Patients
Another retrospective analysis focused on ten pediatric patients with HOX-driven AML, who received revumenib maintenance following HSCT. The median age of this cohort was 10 years, with 80% suffering from KMT2Ar and the remainder with NUP98r AML. Patients underwent a median of two cycles of revumenib pre-HSCT and commenced treatment post-transplant with plans for continuation spanning up to 12 months.
Remarkably, at a median follow-up of 19 months, all ten patients remained alive, with only one relapse, yielding a remarkable 90% relapse-free survival rate. The treatment was largely well-tolerated, with low-grade adverse effects dominating the profile.
Revolutionary SAVE Trial Results
Newly Diagnosed AML Patients
The Phase 2 SAVE trial, an investigator-sponsored study, evaluated revumenib’s oral combination with venetoclax and decitabine/cedazuridine across pediatric and adult patients with R/R or newly diagnosed AML harboring molecular alterations including NPM1m, KMT2Ar, or NUP98r. Eligibility was granted to patients over 12 years who were unqualified for high-intensity chemotherapy.
At the time of data cutoff, the trial had enrolled 21 newly diagnosed patients with a median age of 70 years. Among these patients, a robust 86% ORR was reported, along with a 76% complete remission rate and an astounding 100% MRD negativity among responders. A notable proportion progressed to HSCT following treatment, with approximately 57% resuming revumenib as post-HSCT maintenance.
Syndax's Commitments and Future Directions
Syndax Pharmaceuticals remains committed to advancing Revuforj through ongoing trials exploring its efficacy in combination with standard treatments for newly diagnosed patients with AML. Encouraging results and safety profiles pave the way for future exploration of Revuforj across various therapeutic settings.
Investors and stakeholders will gain further insights into Revuforj's pivotal role in cancer therapy at an event hosted by Syndax. The Company plans to discuss critical findings from the ASH Annual Meeting, offering an enlightening platform for dialogue about the future of cancer therapies.
Frequently Asked Questions
What is Revuforj?
Revuforj (revumenib) is an oral menin inhibitor that has received FDA approval for the treatment of relapsed or refractory acute leukemia with specific genetic mutations in adult and pediatric patients.
What were the key findings presented at the ASH Annual Meeting?
Syndax reported significant efficacy rates, including an ORR of 77% and a 90% relapse-free survival rate among pediatric patients receiving revumenib post-HSCT.
How is revumenib administered?
Revuforj is administered orally, making it a convenient option for patients compared to traditional treatment options.
Are there any notable safety concerns with revumenib?
Common safety concerns include differentiation syndrome and QTc prolongation. However, the overall safety profile appears favorable, with manageable adverse events reported.
What is the future direction for Syndax Pharmaceuticals?
Syndax aims to continue developing Revuforj and expand clinical trials in various treatment settings to enhance outcomes for patients with acute leukemia.
