Axatilimab

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.

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.

FDA Approval of Axatilimab Marks New Era in Chronic GVHD Treatment

3 months ago

• Axatilimab, a CSF1R-blocking antibody, received FDA approval in August 2024 for chronic graft-versus-host disease treatment, demonstrating a 74% overall response rate in clinical trials. • Novel therapies including baricitinib, ixazomib, and teduglutide show promising results in treating steroid-refractory GVHD, offering new hope for patients resistant to conventional treatments. • Pharmacists play a crucial role in GVHD management through medication monitoring, patient education, and therapeutic drug optimization, contributing to improved patient outcomes.

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.

Pharma Industry Maintains Commitment to Clinical Trial Diversity Despite Political Headwinds

4 months ago

• Despite recent removal of federal DEI resources, pharmaceutical companies are expected to maintain their focus on clinical trial diversity due to strong scientific justification and established industry practices. • Industry experts express concern over potential impact on grant-receiving organizations and non-profits that collaborate with pharma companies pursuing diversity initiatives. • A new pan-European initiative launches with €66.8M funding and 73 stakeholders to advance clinical trial diversity over the next six years.

Galderma's Relfydess Shows Rapid, Long-Lasting Improvement for Frown Lines in Phase IIIb Trial

4 months ago

• Phase IIIb RELAX trial data demonstrates rapid onset of aesthetic improvement with Relfydess (RelabotulinumtoxinA) for frown lines, with 40% of subjects reporting improvement on Day 1. • The study showed long-lasting efficacy, with 69% of subjects satisfied at Month 6 and 60% at Month 12, highlighting sustained benefits of the treatment. • RelabotulinumtoxinA continues to be well-tolerated, with no treatment-related serious adverse events reported, reinforcing its safety profile. • Galderma's Relfydess has received approvals in 14 European countries, Australia, and the UK, expanding its availability for aesthetic use.

FDA Approves New Vial Sizes of Niktimvo for Chronic Graft-Versus-Host Disease

4 months ago

• The FDA has approved Niktimvo (axatilimab-csfr) in 9 mg and 22 mg vial sizes for chronic graft-versus-host disease (GVHD) after two prior systemic therapies. • Niktimvo is the first FDA-approved treatment targeting CSF-1R to reduce inflammation and fibrosis in chronic GVHD, offering a novel approach for patients. • AGAVE-201 trial data showed a 75% response rate at six months with the 0.3 mg/kg dose, demonstrating durable responses across various organs. • Incyte and Syndax anticipate Niktimvo will be available in early February, with patient support programs to ensure access and financial assistance.

Belumosudil Shows Promise in Treating Heavily Pretreated Chronic Graft-versus-Host Disease

4 months ago

• Real-world data shows belumosudil is often used in combination after multiple prior therapies for chronic graft-versus-host disease (cGVHD). • A retrospective study in Germany and Switzerland found a 42% overall response rate in patients with advanced cGVHD treated with belumosudil. • Belumosudil demonstrated a steroid-sparing effect and manageable safety profile in patients with severe cGVHD. • The ROCK2 inhibitor shows potential in earlier lines of cGVHD therapy, warranting further investigation in prospective trials.

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.

Ibrutinib and Rituximab Combination Shows Promise in Chronic Graft-vs-Host Disease

4 months ago

• Combination therapy with ibrutinib and rituximab is being explored to improve outcomes in chronic graft-vs-host disease (cGVHD). • Rituximab enhances the therapeutic effects of ibrutinib in treating cGVHD, potentially leading to better patient responses. • Recent trials have demonstrated promising efficacy and safety data for the ibrutinib and rituximab combination in cGVHD. • Clinical practice may shift towards incorporating this combination to optimize treatment strategies for cGVHD patients.

Qurient's Adrixetinib Receives FDA IND Approval for cGVHD Trial

5 months ago

• Qurient's Adrixetinib, a selective triple kinase inhibitor, has received FDA approval for a Phase 1b IND application to treat chronic graft-versus-host disease (cGVHD). • The Phase 1b trial will enroll 18 patients across five U.S. hospitals to assess Adrixetinib's safety, tolerability, pharmacokinetics, and efficacy as a monotherapy. • Adrixetinib targets Axl, Mer, and CSF1R, offering a dual-action mechanism that may benefit both cGVHD and leukemia, particularly in post-transplant settings. • Qurient is also exploring Adrixetinib in acute myeloid leukemia (AML) trials, aiming to establish it as a key drug for myeloid immune cell regulation.

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.

Graves' Disease and Graft-versus-Host Disease: Pipeline Therapeutics Update

6 months ago

• Several companies are actively developing novel therapies for Graves' Disease, targeting mechanisms like IGF-1R and FcRn. • Clinical trials are underway for Batoclimab (Phase III), VRDN-001 (Phase III), and Linsitinib (Phase II/III) to address Graves' Disease. • The Graft-versus-Host Disease pipeline includes over 65 drugs in development, with companies like MaaT Pharma and Syndax Pharmaceutical leading the way. • Therapies like MaaT013 and axatilimab are in advanced stages of clinical trials, offering potential new treatments for Graft-versus-Host Disease.

Incyte Highlights New Tafasitamab Data in Follicular Lymphoma at ASH 2024

6 months ago

• Incyte will present new data from its oncology portfolio at the 2024 American Society of Hematology (ASH) Annual Meeting in San Diego. • A late-breaking oral presentation will feature results from the Phase 3 inMIND study evaluating tafasitamab in relapsed or refractory follicular lymphoma (FL). • Incyte will host a virtual analyst and investor event on December 12, 2024, to discuss key data presentations from ASH.

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

6 months ago

• The FDA has approved Revuforj (revumenib), a menin inhibitor, for relapsed or refractory acute leukemia with KMT2A translocation in adults and pediatric patients. • The approval was based on the AUGMENT-101 trial, which demonstrated strong results in treating patients with this challenging form of leukemia. • Revuforj, developed by Syndax, is the first menin inhibitor to receive FDA approval, marking a significant advancement in leukemia treatment. • Syndax anticipates Revuforj will be available through specialty distributors in November, offering a new treatment option for patients with KMT2A-rearranged acute leukemias.

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.

Revumenib Shows Promise in Phase 2 Trial for Relapsed/Refractory AML

6 months ago

• Syndax Pharmaceuticals' revumenib demonstrated a 23% complete remission rate in patients with relapsed or refractory mutant NPM1 acute myeloid leukemia (AML). • The AUGMENT-101 trial also showed a 47% overall response rate in heavily pre-treated AML patients, including those who had prior venetoclax exposure. • Syndax plans to submit a supplemental New Drug Application (sNDA) in the first half of 2025 based on these positive Phase 2 results. • The favorable safety profile of revumenib, with only 5% of patients discontinuing due to treatment-related adverse events, supports its potential as a valuable option.

Syndax Highlights Revumenib and Niktimvo Progress in Q3 2024 Update

7 months ago

• Syndax's revumenib NDA for R/R KMT2Ar acute leukemia is under FDA review with a PDUFA date of December 26, 2024, potentially launching the drug by year-end. • Topline data from the AUGMENT-101 trial of revumenib in R/R mNPM1 AML is expected in Q4 2024, with a possible sNDA filing in H1 2025. • Niktimvo (axatilimab-csfr) has been approved by the FDA for chronic GVHD and is expected to launch in early Q1 2025, co-commercialized with Incyte. • A $350 million royalty funding agreement for Niktimvo is expected to fund Syndax through profitability, maximizing the potential of their pipeline.

Syndax Anticipates Key Data Readouts and FDA Decision for Revumenib

7 months ago

• Syndax Pharmaceuticals anticipates FDA approval for revumenib in R/R KMT2Ar acute leukemia by December 26, 2024, under Priority Review. • Topline data from the AUGMENT-101 trial for revumenib in R/R mNPM1 AML is expected in Q4 2024, potentially supporting an sNDA filing in 1H25. • Niktimvo (axatilimab-csfr) has been approved for chronic GVHD and is set for U.S. launch by early Q1 2025, co-commercialized with Incyte. • A $350 million royalty funding agreement for Niktimvo is expected to fund Syndax through profitability, maximizing pipeline potential.

Syndax Pharmaceuticals' Niktimvo Approved for cGVHD, Revumenib Awaits FDA Decision

7 months ago

• Syndax Pharmaceuticals' Niktimvo (anti-CSF1R) received FDA approval for chronic Graft versus Host Disease (cGVHD) treatment, marking a significant milestone for the company. • The PDUFA date for Syndax's revumenib, a menin inhibitor for KMT2A-rearranged acute myeloid leukemia (AML), is set for December 26, 2024. • Analysts are optimistic about Syndax's future, with price targets as high as $33.00, reflecting confidence in the company's growth trajectory following recent developments. • Syndax is also exploring revumenib's potential in colorectal cancer (CRC), with Phase 1 proof-of-concept data expected, potentially expanding the drug's market opportunities.

Drug Development Updates