Ruxolitinib Phosphate

With its blockbuster drug Jakafi (ruxolitinib) set to lose patent protection in 2028, Incyte is hoping a new experimental therapy will help fill the looming revenue gap. On Sunday, the company presented the first clinical data for a novel monoclonal antibody, called INCA033989, at the European Hematology Association (EHA) congress.The early-stage study focused on patients with high-risk essential thrombocythemia (ET) who carry a mutant calreticulin (mutCALR) gene, a common genetic driver of the disease. Current ET treatments can help improve symptoms and prevent vascular complications, but they but are limited by toxicity and tolerability issues, and no approved targeted therapies exist for these patients, noted lead author John Mascarenhas.In the Phase I trial, 86% of patients receiving at least 400 mg of the drug saw a complete or partial haematologic response, with 82% achieving complete response. Additionally, 89% of evaluable patients (34/38) showed a drop from baseline in their mutCALR variant allele frequency (VAF), and 21% experienced a partial molecular response, defined as >50% VAF reduction after only three treatment cycles.The data support the hypothesis that INCA033989 could "not only to normalise platelet counts and provide rapid and durable haematologic responses – but to induce molecular responses, which could potentially change the natural history of the disease," Mascarenhas said.Transient lipase increasesThe drug was generally well-tolerated across dose levels. One patient dropped out and another required a lower dose due to treatment-emergent adverse events (TEAEs). Thirteen patients had experienced Grade ≥3 TEAEs, with transient asymptomatic lipase increase as the most common (6%).Reviewing the abstract data released earlier this month, BMO Capital Markets analysts raised questions about safety signals with INCA033989, particularly Grade 3 lipase increases, though no clinical pancreatitis was reported. "Though interesting from an efficacy perspective, these data do little to address our concerns around the company's upcoming coming Jakafi (loss of exclusivity), with a launch not expected until 2028 and meaningful revenue contribution (if approved) likely not beginning until 2029," they said.Jakafi remains Incyte's cornerstone product, making up 77% of its total drug revenue last year. Despite its stronghold in myelofibrosis, growing competition and an upcoming loss of exclusivity add to pressure on the company. While doctors still view Jakafi as the go-to first-line therapy, prescribers may be quicker to switch to second-line options given the growing number that are now available. For more, see – Spotlight On: JAK inhibitors jockey for a share of the myelofibrosis pie.

Oral presentation highlights updated data in Early Access Program (EAP) for 173 patients with acute Graft-vs-Host Disease (aGvHD) treated with Xervyteg® Independent dataset from EAP reinforces the findings from the pivotal ARES trial and has also been included in the EMA Marketing Authorization Application submitted on June 2nd, 2025 EAP for Xervyteg® in aGvHD is currently running in 11 countries* providing expanded access to patients with high unmet medical needs Lyon, France, June 13, 2025 – 7:30am CET – MaaT Pharma (EURONEXT: MAAT – the “Company”), a clinical-stage biotechnology company and a leader in the development of Microbiome Ecosystem TherapiesTM (MET) dedicated to enhancing survival for patients with cancer through immune modulation, today announced that Professor Mohamad Mohty, Professor of Hematology and Head of the Hematology and Cellular Therapy Department at Saint-Antoine Hospital and Sorbonne University, will present updated data for Xervyteg® (MaaT013) in treating acute Graft-versus-Host Disease (aGvHD) under the Early Access Program (EAP) at the European Hematology Association (EHA) Annual Congress 2025. This independent EAP dataset further supports the efficacy and safety profile of Xervyteg® previously shown in the pivotal ARES trial. It also confirms the breakthrough potential of Xervyteg® for aGvHD patients with limited treatment options and it also serves as supportive data within the Marketing Authorization Application (MAA) recently submitted to the European Medicines Agency (EMA). Key highlights: aGvHD is a major cause of morbidity and mortality following allogeneic hematopoietic stem cell transplantation. The patients (N=173) treated in EAP previously failed 1 to 6 aGvHD systemic treatment lines and most had grade III (49%) or IV (38%) aGvHD. The real-world data presented underscores the favorable safety profile of Xervyteg® the strong and durable responses, translating into increased overall survival: Gastrointestinal Overall Response Rate (GI-ORR) of 53% at D28, with Complete Response (CR) observed in 30% of patients; all-organ Overall Response Rate (ORR) was 50% with 26% CR. Response is maintained at D56 indicating a long-term disease control with a GI-ORR of 47% and an ORR considering all organs of 46%. Overall Survival (OS) in all patients was 55% at 6 months, 48% at 12 months, 44% at 24 months. Xervyteg® displayed a good overall safety profile in the EAP population. OS was significantly higher in patients who responded to Xervyteg® (MaaT013) compared to non-responders (69% versus 25% at 12 months, and 61% versus 25% at 24 months). Median survival in all patients was 312 days. In responder patients, median survival was 834 days vs 69 days in non-responders. A subset of patients (n=70) failing both steroid resistant (SR) and ruxolitinib resistant (RR) and thus resembling the cohort enrolled in the pivotal Phase 3 ARES trial (NCT04769895), exhibited a significant and consistent efficacy profile: At both Day 28 and Day 56, Xervyteg® demonstrated durable efficacy in SR/RR aGvHD patients, with GI-ORRs of 57% and Complete Response (CR) observed in 44% of patients at D28 and 51% at D56. All-organs ORR was 54% with 41% CR at D28, and 55% with 48% CR at D56. OS was 55% at 6 months, 51% at 12 months, 40% at 24 months. OS was significantly higher in patients who responded to Xervyteg® compared to non-responders (77% versus 14% at 12 months, and 59% versus 14% at 24 months). Median survival in all 70 patients was 445 days. In responder patients, median survival was 834 days vs 53 days in non-responders. The complete data may be found here. “The consistency between the real-world Early Access Program data and our pivotal ARES trial underscores Xervyteg®’s clinical benefit for patients with severe, treatment-resistant aGvHD,” said Dr. Gianfranco Pittari, Chief Medical Officer at MaaT Pharma. “This is particularly meaningful for clinicians and patients, as it confirms the potential of microbiome therapies to deliver long-term survival benefits in a population with historically poor outcomes.” In comparison, historical data from Abedin et al. 2021 demonstrated that in a similar population of patients, i.e. third-line aGvHD patients receiving additional treatment after ruxolitinib failure, the median survival was only 28 days. “Among patients who responded by Day 28, the majority achieved a complete resolution of aGvHD symptoms — a strong predictor of sustained disease control over time. The overall safety profile is favorable in this high-risk patient population,” outlines Professor Mohty, Professor of Hematology and Head of the Hematology and Cellular Therapy Department at Saint-Antoine Hospital and Sorbonne University. Details of the Oral Presentation: Title: Pooled Fecal Allogeneic Microbiotherapy for Refractory Gastrointestinal Acute Graft-Versus-Host Disease: Results from the Early Access Program in Europe Abstract number: S260 Presenting Author: Mohamad Mohty, Professor of Hematology and Head of the Hematology and Cellular Therapy Department at Saint-Antoine Hospital and Sorbonne University Session title: s424 Stem cell transplantation – Session 2 Date & Time: 13/06/2025 (17:00 – 17:15 CEST) – Brown Hall 3 MaaT Pharma also presented a poster on the design of its ongoing Phase 2b trial (PHOEBUS) evaluating MaaT033 to enhance overall survival in allo-HSCT. This international, multi-center trial (NCT05762211) is the largest randomized controlled study to date of a microbiome-based therapy in oncology, enrolling up to 387 patients across 60 sites. * France, Austria, Belgium, Canada, Germany, Italy, Lebanon, Spain, Sweden, Switzerland & USA About MaaT Pharma MaaT Pharma is a leading, late-stage clinical company focused on developing innovative gut microbiome-driven therapies to modulate the immune system and enhance cancer patient survival. Supported by a talented team committed to making a difference for patients worldwide, the Company was founded in 2014 and is based in Lyon, France. As a pioneer, MaaT Pharma is leading the way in bringing the first microbiome-driven immunomodulator in oncology. Using its proprietary pooling and co-cultivation technologies, MaaT Pharma develops high diversity, standardized drug candidates, aiming at extending life of cancer patients. MaaT Pharma has been listed on Euronext Paris (ticker: MAAT) since 2021. Forward-Looking statements All statements other than statements of historical fact included in this press release about future events are subject to (i) change without notice and (ii) factors beyond the Company’s control. These statements may include, without limitation, any statements preceded by, followed by, or including words such as “target,” “believe,” “expect,” “aim”, “intend,” “may,” “anticipate,” “estimate,” “plan,” “project,” “will,” “can have,” “likely,” “should,” “would,” “could” and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Company’s control that could cause the Company’s actual results or performance to be materially different from the expected results or performance expressed or implied by such forward-looking statements.