On November 2, 2023 Syndax Pharmaceuticals (Nasdaq: SNDX), a clinical-stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported that results from the pivotal AGAVE-201 trial of axatilimab, an anti-CSF-1R antibody, in adult and pediatric patients with chronic graft-versus-host disease (cGVHD), will be featured during the Plenary Scientific Session at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting being held December 9-12, 2023, in San Diego, California (Press release, Syndax, NOV 2, 2023, View Source [SID1234636796]).

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"Inclusion of AGAVE-201 in this year’s ASH (Free ASH Whitepaper) plenary session further supports our belief that axatilimab has the potential to serve as a highly differentiated therapeutic option for patients with chronic GVHD," said Michael A. Metzger, Chief Executive Officer. "We believe axatilimab’s best-in-category profile and unique mechanism of action positions it as an important addition to the chronic GVHD treatment armamentarium, if approved. We look forward to sharing the full dataset next month."

The Company and its partner, Incyte, previously announced positive topline data from the pivotal AGAVE-201 trial of axatilimab in patients with cGVHD following two or more prior lines of therapy. All three dose cohorts, 0.3 mg/kg every two weeks, 1.0 mg/kg every two weeks, and 3.0 mg/kg every four weeks, met the primary endpoint. The overall response rate within the first six months of treatment at the 0.3 mg/kg dose was 74%, and 60% of these patients were still responding at one year. Furthermore, axatilimab was generally well tolerated, and the most common adverse events were consistent with on-target effects and prior trials. Syndax and Incyte expect to submit a BLA filing by year-end 2023.

Abstract Number: 1
Title: Safety and Efficacy of Axatilimab at 3 Different Doses in Patients with Chronic Graft-Versus-Host Disease (AGAVE-201)
Presenter: Daniel Wolff, M.D.
Session Name: Plenary Scientific Session
Session Date: Sunday, December 10, 2023
Session Time: 2:00 – 4:00 PM PT
Presentation Time: 2:00 PM PT

Axatilimab Preclinical Data

In addition, preclinical data detailing the anti-inflammatory and anti-fibrotic mechanism through which axatilimab is thought to impact the disease process in cGVHD will be featured during a poster session.

Details for the presentation are as follows:

Abstract Number: 2540
Title: Axatilimab Ameliorates Inflammation and Fibrosis by Targeting the Macrophages in a Preclinical Model of Chronic GVHD
Presenter: Anamika Bajpai, Ph.D.
Session Name: 201. Granulocytes, Monocytes, and Macrophages: Poster II
Session Date: Sunday, December 10, 2023
Presentation Time: 6:00 – 8:00 PM PT

A copy of each abstract is now available online via the ASH (Free ASH Whitepaper) website at www.hematology.org.

About Chronic Graft-Versus-Host Disease

Chronic graft-versus-host disease (GVHD), an immune response of the donor-derived hematopoietic cells against recipient tissues, is a serious, potentially life-threatening complication of allogeneic hematopoietic stem cell transplantation which can last for years. Chronic GVHD is estimated to develop in approximately 40% of transplant recipients and affects approximately 14,000 patients in the U.S.1,2. Chronic GVHD typically manifests across multiple organ systems, with skin and mucosa being commonly involved, and is characterized by the development of fibrotic tissue3.

About Axatilimab

Axatilimab is an investigational monoclonal antibody that targets colony stimulating factor-1 receptor, or CSF-1R, a cell surface protein thought to control the survival and function of monocytes and macrophages. In pre-clinical models, inhibition of signaling through the CSF-1 receptor has been shown to reduce the number of disease-mediating macrophages along with their monocyte precursors, which has been shown to play a key role in the fibrotic disease process underlying diseases such as chronic GVHD and idiopathic pulmonary fibrosis (IPF). Axatilimab was granted Orphan Drug Designation by the U.S. Food and Drug Administration for the treatment of patients with chronic GVHD and IPF. In September 2021, Syndax and Incyte entered into an exclusive worldwide co-development and co-commercialization license agreement for axatilimab. Axatilimab is being developed under an exclusive worldwide license from UCB entered into between Syndax and UCB in 2016.

About AGAVE-201

The global Phase 2 AGAVE-201 dose-ranging trial evaluated the efficacy, safety, and tolerability of axatilimab in 241 adult and pediatric patients with recurrent or refractory active chronic GVHD whose disease had progressed after two prior therapies. Patients were randomized to one of three treatment groups that investigated a distinct dose of axatilimab administered at 0.3 mg/kg every two weeks, 1 mg/kg every two weeks or 3 mg/kg every four weeks. The trial’s primary endpoint is the proportion of patients in each dose group who achieved an objective response as defined by 2014 NIH Consensus Criteria for chronic GVHD by cycle 7 day 1. Secondary endpoints include duration of response, percent reduction in daily steroids dose, organ specific response rates and validated quality-of-life assessments using the Modified Lee Symptom Scale.

For more information about AGAVE-201, visit View Source