Syndax Pharmaceuticals
🇺🇸United States
- Country
- 🇺🇸United States
- Ownership
- -
- Employees
- 184
- Market Cap
- $1.7B
- Introduction
Syndax Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company engaged in the development of cancer therapies. Its product candidates include SNDX-5613 and SNDX-6352. The company was founded by Richard A. Heyman, Eckard Weber, Peter Ordentlich, Ronald M. Evans and Michael Downes on October 11, 2005 and is headquartered in Waltham, MA.
Syndax Pharmaceuticals Appoints Dr. Nicholas Botwood as New R&D Head and Chief Medical Officer
• 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
• 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
• 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
• 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.
InflaRx's Gohibic Receives EU Approval for COVID-19-Induced ARDS, Bolstering Market Potential
• InflaRx's Gohibic is the first and only treatment approved in the EU for SARS-CoV-2-induced ARDS, marking a significant milestone.
• The European Commission's approval was based on positive Phase 3 PANAMO trial results, demonstrating a 23.9% reduction in mortality.
• H.C. Wainwright reiterated a Buy rating for InflaRx, setting a price target of $8.00, citing Gohibic's market potential and pipeline.
• InflaRx is exploring partnership and distribution options in Europe and is in discussions with the FDA for potential US approval.
FDA Approves New Vial Sizes of Niktimvo for Chronic Graft-Versus-Host Disease
• 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.
Foghorn Therapeutics Advances Oncology Programs Targeting Chromatin Dysregulation
• Foghorn Therapeutics' FHD-909, a SMARCA2 inhibitor, is progressing in Phase 1 trials for SMARCA4-mutated cancers, with NSCLC as the primary target, and combination data with pembrolizumab and KRAS inhibitors will be presented at AACR.
• The company achieved selective degradation of ARID1B, a synthetic lethal target in solid tumors, and plans to update on its progress in 2025, while also advancing CBP and EP300 degrader programs.
• Foghorn Therapeutics is collaborating with Lilly to develop novel oncology medicines, including co-development of the SMARCA2 program and additional undisclosed targets.
• With a strong financial position of $243.8 million in cash reserves, Foghorn Therapeutics anticipates funding operations into 2027, supporting ongoing research and development efforts.
Syndax Highlights FDA-Approved Therapies and Anticipated Milestones at J.P. Morgan Healthcare Conference
• 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.
Sana Biotechnology's HIP Technology Shows Promise in Type 1 Diabetes Trial, Eliciting Analyst Upgrades
• Sana Biotechnology's HIP-modified islet cells demonstrated immune evasion and C-peptide production in a Type 1 Diabetes patient without immunosuppression.
• The positive Phase 1 data supports the potential of Sana's hypoimmune (HIP) cell platform, leading to increased price targets from analysts.
• A single patient in the trial showed stable C-peptide levels 4 weeks post-injection, indicating the HIP cells' survival and function without triggering an immune response.
• Sana Biotechnology's progress in Type 1 Diabetes treatment has been recognized, potentially setting a more favorable stage for its stock performance.
Qurient's Adrixetinib Receives FDA IND Approval for cGVHD Trial
• 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 Approvals in 2024: Novel Therapies for MASH, WHIM Syndrome, Pediatric Glioma, MDS, and Bladder Cancer
• The FDA approved Madrigal's Rezdiffra, the first treatment for metabolic dysfunction-associated steatohepatitis (MASH), addressing a significant unmet need in liver disease.
• X4 Pharmaceuticals' Xolremdi gained approval as the first targeted therapy for WHIM syndrome, a rare immunodeficiency, marking a milestone for the company.
• Day One Biopharmaceuticals' Ojemda secured approval for BRAF-altered pediatric low-grade glioma, offering a new treatment option for this common childhood brain tumor.
• Geron Corporation's Rytelo, a telomerase inhibitor, received FDA approval for lower- to intermediate-risk myelodysplastic syndromes (MDS), after 34 years in business.
• ImmunityBio's Anktiva, a novel IL-15 superagonist, was approved for non-muscle invasive bladder cancer, providing a new therapeutic approach for BCG-unresponsive patients.
FDA Wraps Up 2024 with Key Approvals for Drugs Targeting Various Conditions
• 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
• 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.
Kura Oncology and Kyowa Kirin Partner to Advance Ziftomenib for Acute Leukemias
• Kura Oncology secures a $330 million upfront payment through a collaboration with Kyowa Kirin to develop and commercialize ziftomenib for acute leukemias.
• Ziftomenib, a menin inhibitor, targets leukemias driven by KMT2A gene mutations and is expected to have an FDA submission in 2025 for advanced AML.
• The collaboration includes plans to investigate ziftomenib as an earlier line of therapy, in combination with other drugs, and as a post-transplant maintenance treatment.
• Kura Oncology is also developing tipifarnib for head and neck squamous cell carcinomas, with data from a Phase 1/2 study expected in the first half of next year.
Incyte Halts Urticaria Drug Trial After Toxicology Findings, Scraps Liver Disease Program
• Incyte paused enrollment for INCB000262, a drug being tested for chronic urticaria, due to concerning toxicology results from an animal study.
• The company discontinued development of INCB000547 after Phase 2 results showed it was ineffective for treating itching associated with liver disease.
• These setbacks impact Incyte's strategy to diversify beyond its top-selling drug, Jakafi, and could affect a potential $1.8 billion revenue opportunity.
• Despite these challenges, Incyte anticipates Phase 3 results for povorcitinib in hidradenitis suppurativa next year, which remains a key focus.
FDA Approves UCB's Bimzelx (bimekizumab-bkzx) for Hidradenitis Suppurativa
• 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 Revuforj (revumenib) as First Menin Inhibitor for KMT2A-Rearranged Leukemia
• The FDA has approved Revuforj (revumenib) as the first and only menin inhibitor for relapsed or refractory acute leukemia with KMT2A translocation in adults and children.
• The approval was based on the AUGMENT-101 trial, where 21% of patients achieved complete remission with partial hematological recovery after Revuforj treatment.
• Revuforj targets KMT2A rearrangements, present in approximately 10% of all leukemias, offering a new treatment option for a patient population with limited alternatives.
• Serious adverse reactions, including differentiation syndrome, were observed, necessitating careful monitoring and management during Revuforj treatment.
FDA Approves Revuforj (axatilimab) for Relapsed or Refractory Chronic Graft-versus-Host Disease
• The FDA has granted approval to Revuforj (axatilimab) for the treatment of adult and pediatric patients 12 years and older with relapsed or refractory chronic graft-versus-host disease (cGVHD) after failure of one or two prior lines of systemic therapy.
• Axatilimab, developed by Syndax Pharmaceuticals, is a novel, intravenously administered antibody that targets colony stimulating factor 1 receptor (CSF-1R), a key regulator of monocyte and macrophage function.
• The approval was based on the positive results from the AGAVE-201 trial, which demonstrated clinically meaningful improvements in overall response rate (ORR) and symptom burden in patients treated with axatilimab.
• Revuforj represents a significant advancement in the treatment landscape for cGVHD, offering a new therapeutic option for patients who have limited alternatives after failing prior treatments.
FDA Approves Syndax's Revuforj, a First-in-Class Menin Inhibitor for Acute Leukemia
• The FDA has approved Syndax Pharmaceuticals' Revuforj (revumenib) for adults and children with relapsed or refractory acute leukemia with KMT2A rearrangement.
• Revuforj, a menin inhibitor, showed a complete remission or complete remission with partial hematologic recovery in 21.2% of study participants, with a median response duration of 6.4 months.
• The drug's label includes a black box warning for differentiation syndrome, necessitating close monitoring and potential corticosteroid therapy.
• Syndax has priced Revuforj at $39,500 per month and is exploring its use in other leukemias, including those driven by mNPM1 mutations.
FDA Approves Revumenib for Relapsed/Refractory KMT2A-Rearranged Acute Leukemia
• 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.
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