On June 14, 2024 Sumitomo Pharma America, Inc. (SMPA) reported the oral presentation of data from the ongoing Phase 1/2 first-in-human study of DSP-5336 in patients with relapsed or refractory acute leukemia at the European Hematology Association (EHA) (Free EHA Whitepaper) 2024 Hybrid Congress (Press release, Sumitomo Pharmaceuticals, JUN 14, 2024, View Source [SID1234644347]). DSP-5336 is an investigational small molecule inhibitor of the menin and mixed-lineage leukemia (MLL) protein interaction, which plays key roles in biological pathways, including cell growth regulation, cell cycle control, genomic stability, bone development, and hematopoiesis.1,2,3

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Building on preliminary data presented at the 2023 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, authors presented updated data from the open-label, ongoing dose escalation and optimization portion of the Phase 1/2 study. Patients received oral DSP-5336 in repeating 28-day cycles at doses ranging from 40 mg to 300 mg twice-daily.

The oral presentation at EHA (Free EHA Whitepaper) included the results of 57 patients. Responses were more consistently observed in patients who received 140 mg twice-daily or higher, particularly in the 21 patients with either Nucleophosmin 1 (NPM1) mutation or KMT2A (MLL) rearrangement documented by local testing. Objective response was observed in both patient populations with 57% of patients (12/21), with complete remission or complete remission with partial hematologic recovery (CR/CRh) observed in 24% (5/21 patients).

To date, DSP-5336 remains well-tolerated with no dose limiting toxicity (DLT) observed and no significant cardiac signal nor treatment-related discontinuations or deaths. No significant drug-drug interactions with azoles have been identified and repeat dosing results in minimal to no pharmacokinetic accumulation. Importantly, no differentiation syndrome (DS) prophylaxis was needed, and the three cases of DS reported (5%) were manageable and did not result in intensive care unit (ICU) stays or discontinuation of DSP-5336.

"The response in patients previously untreated with menin inhibitors is encouraging, and competitive with greater than 50% objective response rate alongside a favorable safety profile in patients with relapsed or refractory acute leukemia," said Naval Daver, M.D., Director, Leukemia Research Alliance Program and Professor in the Department of Leukemia at The University of Texas MD Anderson Cancer Center, and lead author on the DSP-5336 poster at EHA (Free EHA Whitepaper). "Menin inhibitors have tremendous potential to improve the outcomes of certain types of acute leukemia, as they reverse the leukemogenic activity of MLL fusion and mutated NPM1 proteins. In addition to promising clinical activity the safety profile of DSP-5336 has been especially encouraging and may differentiate it from other menin inhibitors with no severe DS, DLTs nor treatment-related discontinuations."

Leukemia is a type of cancer that forms in blood-forming tissue, characterized by the uncontrolled growth of blood cells, usually white blood cells, in the bone marrow. Acute leukemia, a form of leukemia, requires immediate treatment as blood cells multiply rapidly leading to a sudden onset of symptoms.4 Approximately 30% of acute myeloid leukemia (AML) patients have NPM1 mutations7 and 5-10% of AML patients have KMT2A (MLL) rearrangements.5

"There remains a high unmet need in relapsed or refractory acute leukemia, as there are no approved targeted treatments for AML with KMT2A (MLL) rearrangements or NPM1 mutations," said Jatin Shah, M.D., Chief Medical Officer – Oncology at SMPA. "The biology of menin inhibition is clearly important and we are very early in the rapidly evolving field. We’re excited by these early results and aim to provide a needed option that is both efficacious and well-tolerated. We look forward to continuing to progress the study of DSP-5336 in the hopes of improving outcomes in AML and advancing patient care."

About DSP-5336
DSP-5336 is an investigational small molecule inhibitor of the menin and mixed-lineage leukemia (MLL) protein interaction. Menin is a scaffold nuclear protein that plays various key roles in biological pathways, including cell growth regulation, cell cycle control, genomic stability, bone development, and hematopoiesis.1,2 In preclinical studies, DSP-5336 has shown selective growth inhibition in human acute leukemia cell lines with KMT2A (MLL) rearrangements or NPM1 mutations.1,3 DSP-5336 reduced the expression of the leukemia-associated genes HOXA9 and MEIS1, and increased the expression of the differentiation gene CD11b in human acute leukemia cell lines with MLL rearrangements and NPM1 mutation.9,10 The safety and efficacy of DSP-5336 is currently being clinically evaluated in a Phase 1/2 dose escalation/dose expansion study in patients with relapsed or refractory acute leukemia (NCT04988555). The FDA granted Orphan Drug Designation for DSP-5336 for the indication of acute myeloid leukemia in June 2022.

On June 14, 2024 Ascentage Pharma (6855 HK), a global biopharmaceutical company engaged in developing novel therapies for cancer, chronic hepatitis B (CHB), and age-related diseases, reported the signing of an option agreement with Takeda to enter into an exclusive license agreement for olverembatinib, an oral, potentially best-in-class third-generation BCR-ABL tyrosine kinase inhibitor (TKI), which is currently in development for chronic myeloid leukemia (CML) and other hematological cancers (Press release, Ascentage Pharma, JUN 14, 2024, View Source [SID1234644346]). If exercised, the option would allow Takeda to license exclusive global rights to develop and commercialize olverembatinib in all territories outside of among others, mainland China, Hong Kong, Macau, Taiwan, China.

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Despite the impact TKIs have had in the treatment of patients with CML, there remains a significant unmet need for patients whose disease is resistant to these therapies or who develop hard-to-treat mutations following these treatments.

Under the terms of this option agreement, Ascentage Pharma is set to receive 100 million USD from Takeda to acquire the option to license olverembatinib. Ascentage will also be eligible for an option exercise fee and additional potential milestones of up to approximately 1.2 billion USD. Additionally, Ascentage Pharma is expected to receive a minority equity investment from Takeda.

As part of the agreement, Ascentage Pharma will continue to be solely responsible for all clinical development of olverembatinib before the potential exercise of the option to license olverembatinib. Olverembatinib is currently approved and marketed in China for the treatment of adult patients with TKI-resistant chronic-phase CML (CP-CML) or accelerated-phase CML (AP-CML) harboring the T315I mutation, and in adult patients with CP-CML resistant to and/or intolerant of first-and second-generation TKIs.

"We are thrilled to enter into this agreement with Takeda, which would allow us to leverage the global commercial expertise of an organization with a proven track record and global oncology footprint to potentially broaden the impact olverembatinib could have on patients in need around the world," said Dr. Dajun Yang, Chairman and CEO of Ascentage Pharma. "Olverembatinib has already had a significant impact for patients with CML in China. We are excited to further the development of olverembatinib in our ongoing global registrational Phase 3 studies."

"Throughout our history in oncology, Takeda has demonstrated a strong track record of working with companies that share our vision of bringing medicines to patients around the world with the highest unmet needs," said Teresa Bitetti, president of Takeda’s Global Oncology Business Unit. "Through this agreement with Ascentage Pharma, we believe there is great potential to continue to advance this mission. We are highly encouraged by the promising results olverembatinib has shown in the clinic to date, and excited to have the opportunity to further develop and deliver it to patients with chronic myeloid leukemia and other hematological cancers."

About Olverembatinib

Olverembatinib, the company’s core drug candidate developed for the treatment of drug-resistant chronic myeloid leukemia (CML) and the company’s first approved product, has been granted Priority Review Designations and Breakthrough Therapy Designations by the Center for Drug Evaluation (CDE) of China National Medical Products Administration (NMPA). To date, the drug has been included in the China 2022 National Reimbursement Drug List (NRDL). Furthermore, olverembatinib has been granted an Orphan Drug Designation (ODD) and a Fast Track Designation (FTD) by the US FDA, and an Orphan Designation by the EMA of the EU.

On June 14, 2024 Ryvu Therapeutics (WSE: RVU), a clinical-stage drug discovery and development company focusing on novel small molecule therapies that address emerging targets in oncology, reported clinical and preclinical data from RVU120 at the 2024 European Hematology Association (EHA) (Free EHA Whitepaper) Congress (EHA) (Free EHA Whitepaper), June 13-16, Madrid, Spain (Press release, Ryvu Therapeutics, JUN 14, 2024, View Source [SID1234644345]).

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"We are thrilled to share the advancements in RVU120 research and its clinical development, emphasizing its significant potential in addressing unmet needs in hematologic malignancies.", states Dr. Hendrik Nogai, Chief Medical Officer, Ryvu Therapeutics. "We are proud of the successful initiation of our Phase II studies. It is reassuring that the initial clinical safety data of RVU120 in combination with venetoclax, allow us to continue testing the compelling preclinical evidence of synergism in patients. Beyond AML and HR-MDS, we continue to work hard on new therapeutic strategies for patients with myeloproliferative neoplasms. Over the upcoming months, we are expecting a ramp up of patient enrollment and data readouts, and we remain committed to developing innovative treatments that can provide improved outcomes for patients in need." – underlines Dr. Hendrik Nogai, Chief Medical Officer, Ryvu Therapeutics.

Poster highlights:

Poster Title: RVU120, a first-in-class CDK8 inhibitor for the treatment of relapsed/refractory AML and high-risk MDS: preliminary results from two ongoing studies.
Poster Number: P600

Session date and time: Friday, June 14 (9:00 CET on the online platform, 18:00 CET for the poster presentation)

The poster includes data on 30 evaluable patients out of 38 total dosed patients in the Phase I trial (RIVER-51) and initial data from the Phase II trial (RIVER-52).

RVU120 as single agent showed clinical benefit in heavily pretreated patients with AML and HR-MDS in the Phase I trial CLI120-001 (RIVER-51). The strongest evidence of benefit was observed in patients with NPM1 and/or DNMT3A mutations, and in patients with HR-MDS.
At the poster presentation’s cut-off date, the data from the Phase II RIVER-52 trial of RVU120 as a monotherapy for patients with relapsed/refractory AML and HR-MDS were not yet mature enough for efficacy assessment in the target population, but preliminary signs of clinical benefit have been observed in ongoing patients.
The safety and tolerability of RVU120 at the RP2D of 250 mg administered every other day was confirmed in patients treated in both trials, with mild or moderate gastrointestinal events being the most frequently reported.
Poster Title: Synergistic potential of RVU120, a first-in-class CDK8/CDK19 inhibitor, with venetoclax in AML: preclinical and initial clinical insights.
Poster Number: P525

Session date and time: Friday, June 14 (9:00 CET on the online platform, 18:00 CET for the poster presentation)

Ryvu presents a mechanism of synergy between RVU120 and venetoclax in preclinical models of acute myeloid leukemia (AML).
The combination of RVU120 and venetoclax leads to caspase-dependent degradation of MCL-1 protein and represses inflammatory and AML oncogenic pathways at the transcriptomic level in AML cells.
RVU120, when combined with venetoclax, exerts cytotoxic and differentiating effects on leukemic stem cells (LSCs) from a hierarchical AML model, exceeding the efficacy of venetoclax alone.
By countering therapeutic failure caused by persistent LSCs and MCL-1-mediated venetoclax resistance, this combination offers hope to patients with AML in both the refractory and the frontline setting.
Initial data from the ongoing Phase II study RIVER-81 demonstrate the safety of RVU120 in combination with venetoclax at the initial dose level in patients with relapsed/refractory AML. Enrollment is currently ongoing in Cohort 2.
Poster Title: CDK8/19 Inhibition: A Promising Therapeutic Strategy in Myeloproliferative Neoplasms.
Poster Number: P1018

Session date and time: Friday, June 14 (9:00 CET on the online platform, 18:00 CET for the poster presentation)

In murine models of disease, RVU120 effectively attenuates myeloproliferative neoplasms (MPN) phenotypes (single-agent or combined with ruxolitinib (RUX)) partly through downregulation of pro-inflammatory cytokines.
RVU120 exhibits synergy with JAK inhibitors as a class and BET inhibitor – pelabresib. These findings open new potential therapeutic options for MPN patients, including myelofibrosis.
The combination of RVU120 and RUX acts synergistically by downregulating JAK/STAT signaling and inflammatory pathways at the transcriptomic level.
Based on compelling preclinical results, Ryvu is launching the clinical study POTAMI-61 (NCT06397313), evaluating RVU120 as a single agent or in combination with ruxolitinib in patients with myelofibrosis.
Investor Event:

Ryvu will host a webinar on Friday, June 14, at 9:30 CET, covering the latest data and potential of RVU120. To join the webcast, please register here: View Source

On June 14, 2024 Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, reported three abstracts to be presented at the European Hematology Association (EHA) (Free EHA Whitepaper) Congress, June 13-16, 2024 (Press release, Autolus, JUN 14, 2024, View Source [SID1234644344]).

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"We are looking forward to presenting longer-term data from adult patients with r/r ALL treated with obe-cel in the FELIX study," said Dr. Christian Itin, Chief Executive Officer of Autolus. "The data indicate that with a median follow up of 21.5 months long-term event free and overall survival are stabilizing at around 40%, suggesting that obe-cel can potentially deliver durable responses as a single agent."

Oral presentation:

Title: obecabtagene autoleucel in adult relapsed/refractory B cell acute lymphoblastic leukemia: Survival and potential impact of CAR T-cell persistence and stem cell transplantation in the FELIX study
Session Title: s419 Acute lymphoblastic leukemia – Clinical 1: Immunotherapy: antibodies and CAR-T cells
Session date and time: Friday, June 14 from 14:45 – 16:00 CEST
Session room: N104
Final Abstract Code: S114
Presenting Author: Dr. Claire Roddie
Summary of Findings: This is an encore presentation of the findings presented at ASCO (Free ASCO Whitepaper) 2024. The overall response rate (ORR) (Complete Response/CRi) in all patients who received obe-cel in the FELIX study was 78% (99/127 patients). At the February 7, 2024, data cut-off date, the majority of ongoing responders showed durable responses. Among the responding patients, at a median follow up of 21.5 months (range: 8.6–41.4), 40% were in ongoing remission without subsequent stem cell therapy (SCT) or other non-protocol specified therapy, while 18% proceeded to subsequent SCT while in remission, 5% started new anti-cancer therapy while in remission and 36% relapsed or died. The median event-free survival (EFS) was 11.9 months and median overall survival (OS) was 23.8 months and the estimated 12-month EFS and OS rates were 49.5% and 61.1% respectively.

These data support the potential of a long-term plateau of survival outcomes in patients receiving obe-cel. Ongoing CAR T persistence and B-cell aplasia were associated with improved EFS. This pattern is consistent with the Phase 1 ALLCAR19 data. Furthermore, SCT consolidation in remission following obe-cel did not appear to improve EFS or OS.
Poster presentations:

Title: obecabtagene autoleucel (obe-cel, AUTO1) for relapsed/refractory adult B-cell acute lymphoblastic leukemia (R/R B-ALL): The impact of inotuzumab (INO)-containing bridging therapy on treatment outcomes
Session Title: Poster session
Session date and time: Friday, June 14 from 18:00 – 19:00 CEST
Final Abstract Code: P418
Presenting Author: Dr. Jae H. Park
Summary of Findings: INO-containing bridging therapies were effective in reducing BM disease prior to lymphodepletion and administration of obe-cel. Our data suggests that reducing BM blasts as much as possible prior to lymphodepletion predicts EFS and OS outcomes; however, patients with high disease burden at screening are still at higher risk of relapse overall. Bridging therapy with INO was utilized in patients with higher disease burden (median 81.5% blasts at screening vs 40% in bridging therapy w/o INO group) and helped minimize the risk of CRS and ICANS without increasing liver toxicity. Choice of bridging therapy prior to obe-cel treatment, though influenced by clinical care variables, may impact outcomes for patients with R/R B-ALL. Further studies comparing bridging with INO-containing therapies or chemotherapy are warranted.

Title: Droplet digital PCR and flow cytometry sensitivity for measuring CAR T-cell kinetics in adult patients with relapsed/refractory B-cell acute lymphoblastic leukemia treated with obecabtagene autoleucel
Session Title: Poster session
Session date and time: Friday, June 14 from 18:00 – 19:00 CEST
Final Abstract Code: P1469
Presenting Author: Dr. Claire Roddie
Summary of Findings: A strong correlation was observed between flow cytometry and ddPCR assays for assessment of CAR T levels in peripheral blood. ddPCR is a more sensitive technology than flow cytometry for monitoring CAR T persistence. Flow cytometry assays developed specifically for CAR T monitoring may be sufficiently sensitive to be of clinical relevance. Our data suggest that loss of CAR T persistence is associated with shorter EFS and may be taken into consideration, together with other parameters such as measurable residual disease (MRD), to help inform clinical monitoring post-obe-cel infusion. A validation cohort using an appropriately developed CAR T marking flow cytometry assay versus a ddPCR assay would be required to conclude on the most appropriate methods to inform clinical outcomes.

On June 14, 2024 Syndax Pharmaceuticals (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported updated data from multiple combination trials of revumenib, the Company’s potent, selective, small molecule menin inhibitor, in patients with acute leukemias (Press release, Syndax, JUN 14, 2024, View Source [SID1234644341]). The updated data are being presented at the European Hematology Association (EHA) (Free EHA Whitepaper) 2024 Congress, being held June 13-16 in Madrid, Spain and virtually.

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"The growing body of data supports the potential for revumenib to have a meaningful impact in combination with current standard of care therapies," said Joshua F. Zeidner, M.D., Chief, Leukemia Research at the University of North Carolina, Lineberger Comprehensive Cancer Center. "Based on the BEAT AML data in the newly diagnosed setting which showed a high rate of MRD-negative responses coupled with a safety profile that enables combination use, revumenib has the potential to become a cornerstone of treatment as front-line therapy for newly diagnosed KMT2Ar and mNPM1 AML."

"We are committed to advancing revumenib across a spectrum of acute leukemia patients. As we prepare for the expected near-term approval of revumenib in the relapsed or refractory setting, we look forward to providing additional clinical data as monotherapy and in combination to support treatment in various acute leukemia treatment settings where novel treatment options are urgently needed," said Neil Gallagher, M.D., Ph.D., President, Head of Research and Development at Syndax.

BEAT AML Trial

The Company announced updated data from the BEAT AML trial of revumenib in combination with venetoclax/azacitidine in newly diagnosed mutant nucleophosmin (mNPM1) or KMT2A-rearranged (KMT2Ar) acute myeloid leukemia (AML) patients aged 60 years or older in an oral presentation titled "Phase 1b Study of Azacitidine, Venetoclax and Revumenib in Newly Diagnosed Older Adults with NPM1 Mutated or KMT2A Rearranged AML: Interim Results of Dose Escalation from the BeatAML Consortium."

The dose escalation phase of the trial tested revumenib at doses of 113 mg and 163 mg q12h in combination with a strong CYP3A4 inhibitor beginning on day 1 of a 28-day cycle in combination with on label doses of venetoclax and azacitidine. As of the data cutoff date of May 1, 2024, 26 newly diagnosed mNPM1 (n=17) or KMT2Ar (n=9) AML patients were enrolled. In the efficacy evaluable population, the composite complete remission1 (CRc) rate was 96% (23/24) and 92% (22/24) of patients also attained minimal residual disease (MRD) negative status as determined by central flow cytometry. Three patients proceeded to hematopoietic stem cell transplant (HSCT). The first cohort of patients treated in the trial, at 113 mg, had extended follow-up and an estimated 12-month survival of 100%.

Revumenib was dosed safely at both the 113 mg and 163 mg q12h dose in combination with venetoclax and azacitidine. 15% (4/26) of patients experienced differentiation syndrome with one (4%) Grade 3 event. 46% (12/26) of patients experienced QTc prolongation with three (12%) Grade 3 events. All DS and QTc prolongations were self-limiting and resolved without complication or the need for revumenib dose reductions. Venetoclax was dosed in accordance with its label and an analysis of the onset and extent of hematologic toxicities suggest a similar experience to what has been reported for the venetoclax/azacytidine doublet. Overall, there were no new or increased safety signals observed when revumenib was added to this triplet combination.

An expansion cohort is ongoing at both dose levels to establish the recommended dose for future trials. The Company plans to initiate a pivotal trial with this combination in front-line newly diagnosed patients by year-end 2024.

AUGMENT-102 Trial

The Company also announced a poster presentation featuring updated data from the AUGMENT-102 trial of revumenib in combination with fludarabine/cytarabine in a predominantly pediatric population of patients with relapsed/refractory (R/R) mNPM1 (n=2), NUP98-rearranged (NUP98r) (n=1) or KMT2Ar (n=23) AML titled "Safety and Activity of Revumenib in Combination with Fludarabine/Cytarabine (FLA) in Patients with Relapsed/Refractory Acute Leukemias."

As of the data cutoff date of January 15, 2024, 27 patients received revumenib plus FLA, including 9 patients treated at 113 mg q12h and 18 treated at 163 mg q12h. The patients enrolled had a median age of 6 years and had received a median of 3 prior lines of therapy. Eighteen (67%) patients had prior FLA containing regimens while 11 (41%) patients had prior HSCT. Five (56%) patients treated at the 113 mg q12h dose and nine (50%) patients treated at the 163 mg q12h achieved a CRc. Most patients who achieved a CRc and had evaluable data achieved (MRD) negative status (10/14; 71%) and 7 patients underwent HSCT while in remission following treatment.

Overall, revumenib was tolerable in heavily pretreated patients with KMT2Ar, NUP98r, or NPM1m acute leukemias without increased frequency or severity of AEs compared with historic FLA data or revumenib monotherapy. One DLT occurred at the 163 mg q12h dose that was a Grade 4 decreased neutrophil count in a patient with multiple prior transplants.

Grade 3 and above adverse events in over 40% of patients included decreased platelet count (17/27; 63%), anemia (15/27; 56%) and febrile neutropenia (13/27; 48%). Lower rates of cytopenias were reported at the 163 mg q12h dose than the 113 mg q12h dose, consistent with faster remission at the higher dose. Lower rates of nonhematologic adverse events were also observed at the higher dose level, which further suggests that the adverse event profile was not driven by revumenib. There was one adverse event leading to death (sepsis at the 113 mg q12h dose level) not related to revumenib. There were no cases of differentiation syndrome in the trial.

This study supports the selection of revumenib 163 mg q12h (95 mg/m2 q12h if weight <40 kg) combined with FLA and a strong cytochrome P450 inhibitor as the RP2D, in line with the dose of revumenib under FDA review as a monotherapy agent.

Additional Presentations at EHA (Free EHA Whitepaper) 2024

In addition to updated results from the BEAT AML and AUGMENT-102 studies, an encore presentation of results from the pivotal AUGMENT-101 study of revumenib in R/R KMT2Ar acute leukemia were also featured at the Congress during an oral session titled "Revumenib Monotherapy in Patients with Relapsed/Refractory KMT2Ar Acute Leukemia: Topline Efficacy and Safety Results from the Pivotal AUGMENT-101 Phase 2 Study."

Results from an exploratory analysis of immunophenotypic changes in AML blasts following treatment with revumenib were also featured in a poster presentation titled "Characterization of Immunophenotypic Changes Following Menin Inhibition in Acute Myeloid Leukemia."

Copies of EHA (Free EHA Whitepaper) posters and presentations will be available in the Publications and Meeting Presentations section of Syndax’s website.

About Revumenib

Revumenib is a potent, selective, small molecule inhibitor of the menin-KMT2A binding interaction that is being developed for the treatment of KMT2A-rearranged (KMT2Ar), also known as mixed lineage leukemia rearranged or MLLr, acute leukemias including ALL and AML, and mutant nucleophosmin (mNPM1) AML. Positive topline results from the Phase 2 AUGMENT-101 trial in R/R KMT2Ar acute leukemia showing the trial met its primary endpoint were presented at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, and data from the Phase 1 portion of AUGMENT-101 in acute leukemia was published in Nature. Revumenib was granted Orphan Drug Designation by the FDA and European Commission for the treatment of patients with AML and Fast Track designation by the FDA for the treatment of adult and pediatric patients with R/R acute leukemias harboring a KMT2A rearrangement or NPM1 mutation. Revumenib was granted Breakthrough Therapy Designation by the FDA for the treatment of adult and pediatric patients with R/R acute leukemia harboring a KMT2A rearrangement.