Revumenib

Syndax Pharmaceuticals Appoints Dr. Nicholas Botwood as New R&D Head and Chief Medical Officer

10 days ago

• Dr. Nicholas Botwood joins Syndax Pharmaceuticals from Bristol Myers Squibb, bringing 25 years of oncology drug development and commercialization experience to accelerate the company's cancer therapy pipeline. • The appointment comes as Syndax continues to expand its portfolio, which includes FDA-approved Revuforj (menin inhibitor) and Niktimvo (axatilimab-csfr), a monoclonal antibody targeting the CSF-1 receptor. • Dr. Botwood succeeds Dr. Neil Gallagher, who oversaw multiple positive data readouts, two product approvals, and the recent submission of Revuforj's supplemental New Drug Application for relapsed or refractory mNPM1 acute myeloid leukemia.

Lorlatinib Shows Impressive 5-Year PFS Results in ALK-Positive Advanced NSCLC

11 days ago

• Lorlatinib demonstrated a 60% progression-free survival rate at 5 years in patients with ALK-positive advanced non-small cell lung cancer, according to data from the phase 3 CROWN study presented at ASCO 2024. • The targeted therapy significantly reduced the risk of disease progression or death compared to crizotinib, with a hazard ratio of 0.19 (95% CI, 0.13-0.27), highlighting its superior efficacy in this patient population. • Investigators described the long-term progression-free survival results as "fantastic," positioning lorlatinib as a potentially practice-changing option for patients with ALK-positive NSCLC.

Ryvu Therapeutics Advances Synthetic Lethality Pipeline with Promising Preclinical Data at AACR 2025

2 months ago

• Ryvu's RVU305, a brain-permeable MTA-cooperative PRMT5 inhibitor, demonstrates significant tumor growth inhibition in MTAP-deleted cancer models and enhances responses when combined with anti-PD-1 antibodies. • The company's proprietary ONCO Prime platform has identified novel synthetic lethal targets for KRAS-driven colorectal cancer, potentially offering new personalized treatment options for patients based on their tumor's genetic profile. • Ryvu is advancing multiple preclinical programs simultaneously, including next-generation ADC payloads that will be presented at the upcoming ADC Payload Summit in Boston, with IND/CTA-enabling studies for RVU305 on track for completion in H2 2025.

Incyte Reports Strong 2024 Growth with $4.2B Revenue, Outlines Ambitious 2025 Pipeline Milestones

3 months ago

• Incyte achieved total revenues of $4.2 billion in 2024, marking a 15% year-over-year growth, driven by strong performance of Jakafi ($2.8B) and Opzelura ($508M). • The company anticipates four new product launches in 2025, including Niktimvo for chronic GVHD and expanded indications for existing therapies in atopic dermatitis and lymphoma. • Incyte's R&D pipeline shows significant advancement with plans for four pivotal study readouts, three Phase 3 study initiations, and seven proof-of-concept study results expected in 2025.

Syndax Highlights FDA-Approved Therapies and Anticipated Milestones at J.P. Morgan Healthcare Conference

4 months ago

• Syndax launched Revuforj (revumenib) for relapsed/refractory acute leukemia with KMT2A translocation, receiving NCCN guideline inclusion for AML and ALL. • Niktimvo (axatilimab-csfr) gained FDA approval for chronic GVHD treatment after two prior systemic therapies in patients weighing at least 40 kg. • A supplemental NDA filing for revumenib in R/R mNPM1 AML is expected in the first half of 2025, based on positive data from the AUGMENT-101 trial.

Blinatumomab Approved for Consolidation Therapy in CD19-Positive B-ALL, Regardless of MRD Status

4 months ago

• The FDA approved blinatumomab in June 2024 for CD19-positive, Philadelphia chromosome-negative B-ALL consolidation, expanding its use in both adult and pediatric patients. • ECOG-ACRIN E1910 trial data supported the approval, showing superior overall survival with blinatumomab plus chemotherapy compared to chemotherapy alone. • Blinatumomab is now a standard component of consolidation therapy, irrespective of a patient's minimal residual disease (MRD) status or backbone chemotherapy. • Research continues to refine blinatumomab's role, exploring optimal patient selection, treatment sequencing, and the necessity of allogeneic stem cell transplant.

FDA Approvals in 2024: Advancing Treatment Paradigms in Solid Tumors and Hematologic Malignancies

5 months ago

• The FDA granted over 65 approvals in 2024, significantly impacting treatment paradigms across various cancers, including breast, gynecologic, skin, and genitourinary malignancies. • Several tumor-agnostic approvals, such as fam-trastuzumab deruxtecan-nxki (Enhertu) for HER2-positive solid tumors and repotrectinib (Augtyro) for NTRK fusion-positive tumors, marked advancements in precision medicine. • Immunotherapies like nivolumab (Opdivo) and pembrolizumab (Keytruda) received multiple approvals, including combinations with chemotherapy for urothelial and endometrial carcinomas, improving patient outcomes. • Targeted therapies like vorasidenib (Voranigo) for low-grade glioma and selpercatinib (Retevmo) for RET-mutated thyroid cancers addressed unmet needs and demonstrated high efficacy and tolerability.

MSK Researchers Spearhead Advances in Cancer Treatment, Leading to 11 FDA Approvals in 2024

5 months ago

• Memorial Sloan Kettering Cancer Center (MSK) made significant strides in cancer treatment in 2024, including novel vaccines, drugs enhancing radiation, and advanced diagnostics. • A vaccine targeting KRAS mutations in pancreatic and colorectal cancers showed promising early results in stimulating the immune system. • Imlunestrant, combined with abemaciclib, demonstrated a significant reduction in cancer progression or death in ER+, HER2- advanced breast cancer patients resistant to standard therapy. • MSK played a pivotal role in clinical trials leading to 11 FDA approvals in 2024, spanning new uses for existing drugs, effective drug combinations, and novel targeted therapies.

Menin Inhibitor Combination Induces Remission in Pediatric AML Patient

5 months ago

• A 13-year-old with relapsed acute myeloid leukemia (AML) achieved full remission through a clinical trial at MD Anderson using a novel menin inhibitor combination. • The experimental regimen included revumenib (SNDX-5613), venetoclax, and decitabine/cedazuridine, leading to undetectable leukemia cells within two weeks. • Following remission, the patient underwent a successful stem cell transplant and is now recovering, with improving blood counts and renewed hope for the future. • This combined therapeutic approach may establish a new standard of care for KMT2A-mutated AML, addressing a critical unmet need in pediatric oncology.

Revumenib Combination Shows Promise in Relapsed/Refractory AML with Specific Genetic Alterations

5 months ago

• The phase 1/2 SAVE study evaluated an all-oral combination of revumenib, decitabine/cedazuridine, and venetoclax in relapsed/refractory AML patients. • The overall response rate was 82%, with notable efficacy in patients harboring _KMT2Ar_ (88%), _NPM1mt_ (67%), and _NUP98r_ (100%) genetic alterations. • A significant proportion of responders achieved minimal residual disease (MRD) negativity, particularly those with complete remission or complete remission with partial hematologic recovery. • The combination demonstrated a manageable safety profile, with common grade 3 or higher adverse events including febrile neutropenia and lung infection.

Triplet Therapies Show Promise in Treating Leukemias: ASH 2024 Highlights

6 months ago

• Novel triplet therapies are demonstrating significant positive results in treating relapsed/refractory and newly diagnosed leukemias, according to multiple clinical trials. • A revumenib-based triplet achieved an 82% overall response rate in relapsed/refractory AML patients with KMT2A or NUP98 rearrangements, offering an improved treatment option. • Ivosidenib, venetoclax, and azacitidine triplet showed a 94% overall response rate in IDH1-mutant hematologic malignancies, positioning it as a potential standard-of-care. • Pirtobrutinib, obinutuzumab, and venetoclax triplet yielded high rates of undetectable measurable residual disease in previously untreated CLL patients.

FDA Wraps Up 2024 with Key Approvals for Drugs Targeting Various Conditions

6 months ago

• The FDA approved Vertex's Alyftrek for cystic fibrosis, offering improved dosing and potential market exclusivity. • Novo Nordisk's Alhemo was approved for hemophilia A and B, providing a new option for patients with inhibitors. • Bristol Myers Squibb's Opdivo Qvantig gained approval as a subcutaneous formulation, offering faster administration for various solid tumors. • Eli Lilly's Zepbound secured approval for obstructive sleep apnea in obese adults, marking the first prescription medicine for this condition.

Ziftomenib Shows Promise in Treatment of Relapsed/Refractory AML

6 months ago

• Ziftomenib, a selective menin inhibitor, demonstrates promising clinical activity in relapsed/refractory AML patients, especially those with _NPM1_ mutations or _KMT2A_ rearrangements. • The KOMET-001 trial revealed a 25% complete remission rate in patients with _KMT2A_ rearrangement or _NPM1_ mutations at the 600 mg dose, with a 35% complete remission rate in _NPM1_ mutated patients. • Ziftomenib exhibits a manageable safety profile, with the most common severe adverse events including anemia, febrile neutropenia, and pneumonia, and a low incidence of _MEN1_ mutation development. • Ongoing clinical studies are exploring ziftomenib in combination with other therapies, such as 7+3 induction chemotherapy and azacytidine/venetoclax, to enhance its effectiveness in AML treatment.

FDA Approves UCB's Bimzelx (bimekizumab-bkzx) for Hidradenitis Suppurativa

6 months ago

• The FDA has approved Bimzelx (bimekizumab-bkzx) as the first IL-17A and IL-17F inhibitor for adults with moderate to severe hidradenitis suppurativa (HS). • Approval was based on Phase 3 trials (BE HEARD I and BE HEARD II) demonstrating significant improvement in HS signs and symptoms at Week 16 and sustained responses at Week 48. • Bimekizumab-bkzx showed a higher proportion of patients achieving HiSCR50 (50% improvement) compared to placebo, along with clinically meaningful improvements in HiSCR75. • This approval marks the fifth patient population in the U.S. that may benefit from Bimzelx, addressing a substantial unmet need in HS treatment.

FDA Approves Syndax's Revuforj as First Menin Inhibitor for KMT2A-Rearranged Leukemia

6 months ago

• The FDA has granted priority review and approval to Syndax Pharmaceuticals' Revuforj (revumenib) for relapsed or refractory acute leukemia with KMT2A translocations. • Revuforj is the first FDA-approved menin inhibitor, marking a significant advancement in treating this aggressive form of leukemia in patients aged one year and older. • Approval was based on the AUGMENT-101 trial, where Revuforj demonstrated a 21% complete remission rate or complete remission with partial hematological recovery. • Syndax plans to launch Revuforj this month, offering a new treatment option for patients with KMT2A-rearranged leukemia who have limited alternatives.

FDA Approves Revumenib for Relapsed/Refractory KMT2A-Rearranged Acute Leukemia

6 months ago

• The FDA has approved revumenib (Revuforj; Syndax Pharmaceuticals) as a first-in-class therapy for relapsed or refractory acute leukemia with KMT2A rearrangements. • Revumenib, a menin inhibitor, targets the interaction between menin and KMT2A protein, which is crucial for the leukemogenic process in KMT2Ar leukemia. • Clinical trials showed revumenib significantly improved remission rates compared to historical controls, with a complete remission rate of 22.8% in the AUGMENT-101 trial. • Pharmacists play a vital role in optimizing dosing, monitoring for adverse events like QTc prolongation and differentiation syndrome, and ensuring patient safety.

Revumenib Demonstrates Sustained Responses in Relapsed/Refractory KMT2Ar Acute Leukemia

6 months ago

• Revumenib shows continued clinically meaningful responses in patients with relapsed/refractory _KMT2Ar_ acute leukemia. • The Phase 2 AUGMENT-101 trial update reveals higher minimal residual disease negativity rates with revumenib. • Treatment with revumenib led to a significant percentage of patients proceeding to hematopoietic stem cell transplant. • The safety profile of revumenib remains manageable, with no discontinuations due to differentiation syndrome or QTc prolongation.

FDA Approves Revumenib for Relapsed/Refractory Acute Leukemia with KMT2A Translocation

6 months ago

• The FDA has approved revumenib (Revuforj) for relapsed or refractory acute leukemia with a lysine methyltransferase 2A (KMT2A) translocation in patients aged one year and older. • Revumenib's approval was based on the AUGMENT-101 trial, which demonstrated a complete remission (CR) plus CR with partial hematologic recovery (CRh) rate of 21.2%. • The median duration of CR+CRh was 6.4 months, and the median time to CR or CRh was 1.9 months in patients treated with revumenib in the clinical trial. • Common adverse reactions included hemorrhage, nausea, and increased phosphate, with the recommended dose varying by patient weight and CYP3A4 inhibitor use.

FDA Approves Revuforj (revumenib) for Relapsed/Refractory Acute Leukemia with KMT2A Translocation

6 months ago

• The FDA has approved Revuforj (revumenib) as the first menin inhibitor for relapsed or refractory acute leukemia with a KMT2A translocation in patients aged one year and older. • Efficacy was demonstrated in the AUGMENT-101 trial, with a complete remission rate of 21% in patients treated with Revuforj, and 23% proceeding to stem cell transplantation. • Revuforj's approval marks a significant advancement for patients with KMT2A-rearranged acute leukemia, who typically face poor prognoses and limited treatment options. • Syndax Pharmaceuticals is preparing to launch Revuforj, with plans to make various tablet strengths available to accommodate different patient populations.

Revuforj (Revumenib) Shows Promising Results in Acute Leukemia Trials

6 months ago

• Syndax Pharmaceuticals presented data from multiple trials of Revuforj (revumenib) at the ASH Annual Meeting, showcasing its efficacy in acute leukemias. • The SAVE trial showed an 82% overall response rate (ORR) and 48% complete remission rate (CR/CRh) when revumenib was combined with venetoclax and decitabine/cedazuridine in R/R AML. • In the AUGMENT-101 trial, revumenib demonstrated a 64% ORR and 23% CR/CRh in R/R KMT2Ar acute leukemia patients, with high rates of MRD negativity. • Initial results from the INTERCEPT trial suggest revumenib may effectively target MRD in AML patients, supporting further evaluation as a pre-emptive therapy.

Drug Development Updates