RegulationFast Track (United States), Orphan Drug (United States), Orphan Drug (European Union), Orphan Drug (Japan), Orphan Drug (South Korea), Paediatric investigation plan (European Union), Conditional marketing approval (China), Priority Review (China)

Structure/Sequence

Sequence Code 37465

Source: *****

Clinical Trials associated with Luspatercept-AAMT

NCT07338344

/ RecruitingPhase 2IIT

Evaluation of the Clinical Efficacy and Safety of Luspatercept Combined With Low-dose Thalidomide Versus Luspatercept Alone in the Treatment of Adult Patients With Transfusion-dependent β-thalassemia

β-thalassemia is one of the most common inherited hemoglobinopathies worldwide and a major public health issue that severely impacts birth quality, human health, and social progress. Currently, there are limited clinical drugs specifically designed to treat patients with β-thalassemia. This clinical trial aims to evaluate the efficacy and safety of luspatercept combined with low-dose thalidomide compared with luspatercept alone in patients with thalassemia. Key questions to be answered include:
* Does luspatercept combined with low-dose thalidomide reduce the transfusion burden in patients with β-thalassemia major?
* What medical problems may occur when patients receive luspatercept combined with low-dose thalidomide? In this clinical trial, participants were randomly assigned in a 1:1 ratio to either an intervention group (luspatercept combined with low-dose thalidomide) or a control group (luspatercept combined with placebo) using a central randomization system. The clinical efficacy and safety of the two groups were evaluated. The primary outcome measure was the clinical efficacy of luspatercept combined with low-dose thalidomide in reducing the transfusion burden in patients with β-thalassemia major.

Start Date01 Feb 2026

Sponsor / Collaborator

First Affiliated Hospital of Guangxi Medical University

NCT07331818

/ Not yet recruitingPhase 2IIT

Luspatercept in Patients Affected With Rare Inherited Anemias

This is a prospective multicenter phase II basket trial evaluating Luspatercept in patients affected with rare inherited anemias

Start Date15 Jan 2026

Sponsor / Collaborator-

NCT07096297

/ RecruitingPhase 2IIT

A Phase II Study of Luspatercept Plus Darbepoetin Alfa in Non-mutated SF3B1 Lower-risk Myelodysplastic Syndromes

This is a single arm open-label Phase II trial of luspatercept and darbepoetin alfa in non-mutated SF3B1 , lower-risk, RBC transfusion dependent MDS participants with an endogenous erythropoietin (EPO) level < 500 IU/L.

Start Date01 Jan 2026

Sponsor / Collaborator

Yale University

[+1]

100 Clinical Results associated with Luspatercept-AAMT

100 Translational Medicine associated with Luspatercept-AAMT

100 Patents (Medical) associated with Luspatercept-AAMT

247

Literatures (Medical) associated with Luspatercept-AAMT

02 Jan 2026·Anti-inflammatory & anti-allergy agents in medicinal chemistry

Clinical Applications of Ligand Traps Targeting Activin Type II Receptors

Article

Author: Tsuchida, Kunihiro

This review summarizes recent advances in ligand trap therapies targeting activin type II receptors [ActRIIA/ACVR2A and ActRIIB/ACVR2B], which serve as shared receptors for members of the TGF-β family, including activins, GDF11, and myostatin [MSTN]. These receptors mediate Smad2/3 signaling and play critical roles in hematopoiesis, vascular homeostasis, and muscle regulation. Two peptide-based ligand traps have recently received clinical approval: luspatercept [ActRIIB-Fc], an erythroid maturation agent, and sotatercept [ActRIIA-Fc], a novel therapeutic agent for pulmonary arterial hypertension [PAH]. Luspatercept primarily inhibits activin B and GDF11, thereby promoting late-stage erythropoiesis and demonstrating efficacy in anemia associated with conditions such as myelodysplastic syndromes [MDS] and β-thalassemia. Sotatercept binds activins and GDFs to rebalance Smad2/3 and Smad1/5/8 signaling, thereby improving vascular remodeling in PAH. Although both agents have failed to increase skeletal muscle mass in clinical trials consistently, they represent significant advances in the treatment of hematopoietic and vascular disorders. Future studies should focus on optimal dosing strategies, long-term safety, and potential synergistic effects when combined with other therapeutic modalities.

01 Jan 2026·BRITISH JOURNAL OF HAEMATOLOGY

Achieving clinically meaningful changes in haemoglobin levels in patients with non‐transfusion‐dependent β‐thalassaemia treated with luspatercept: A post hoc analysis of the phase 2 BEYOND trial

Article

In this post hoc analysis of the phase 2 BEYOND trial, the majority of patients with non-transfusion-dependent β-thalassaemia achieved clinically meaningful haemoglobin levels ≥10.0 g/dL and increases from baseline ≥1.0 g/dL, thresholds associated with reduced risk of morbidity and mortality and recommended as an indication and target for treatment by international management guidelines, respectively.

31 Dec 2025·JOURNAL OF MEDICAL ECONOMICS

Healthcare resource utilization of patients with lower-risk myelodysplastic syndromes treated with luspatercept versus erythropoiesis-stimulating agents: a United States healthcare claims database study

Article

Author: Mohan, Manasi ; Yenikomshian, Mihran ; Huynh, Lynn ; Swanson, Ashley ; Tang, Derek ; Pinaire, Megan ; Slaff, Samantha ; Zanardo, Enrico ; Song, Rui ; Gavrilov, Svetlana ; Ball, Brian J.

OBJECTIVE:

This study compared the healthcare resource utilization (HRU) of patients with lower-risk myelodysplastic syndromes (LR-MDS) treated with luspatercept versus erythropoiesis-stimulating agents (ESAs).

METHODS:

This real-world retrospective cohort study used claims data from the Symphony Health Integrated Dataverse, a large nationally representative United States database, to identify patients with LR-MDS who initiated luspatercept or ESA between May 1, 2020, and June 30, 2022. Index date was defined as the date of the first claim for luspatercept or ESA. The follow-up period was from the index date to the earliest of the end of clinical activity or end of data availability. All-cause and MDS-related HRU were evaluated for both treatment cohorts and compared using generalized estimating equations with Poisson distribution and robust variance estimator.

RESULTS:

Overall, 243 and 3,515 patients were included in the luspatercept and ESA cohorts, respectively. Patients in both cohorts had a similar median (interquartile range [IQR]) age at index (luspatercept: 77.0 [70.0-79.0] years; ESA: 78.0 [72.0-79.0] years) and median (IQR) follow-up duration (luspatercept: 14.6 [10.4-22.6] months; ESA: 14.4 [9.5-20.9] months). Compared with patients treated with ESA, patients treated with luspatercept had a 26% lower rate of all-cause inpatient visits (adjusted incidence rate ratio [aIRR], 0.74; 95% confidence interval [CI], 0.58-0.93; p < .05) and a 31% lower rate of all-cause outpatient visits (aIRR, 0.69; 95% CI, 0.61-0.79; p < .001). The rate of MDS-related inpatient visits was also 25% lower among the patients treated with luspatercept versus ESA (aIRR, 0.75; 95% CI, 0.56-0.99; p < .05).

CONCLUSION:

This study showed that patients with LR-MDS treated with luspatercept required significantly less HRU than patients treated with ESA. Further research is warranted to evaluate the financial impact of this lower HRU burden.

191

News (Medical) associated with Luspatercept-AAMT

14 Jan 2026

·endpoints.news

Q&A: Bristol Myers CEO Chris Boerner on megamergers, Trump in 2026, and brain health as the 'next frontier'

SAN FRANCISCO — In his third year as CEO of Bristol Myers Squibb, Chris Boerner kicked off the JP Morgan Healthcare Conference with plans for six pivotal readouts of new drugs in 2026. This array of potential medicines for everything from heart disease to cancer to neuropsychiatry will be key in replacing revenues from current bestsellers Opdivo and Eliquis, which are expected to potentially face generic competition in 2028. The $114 billion pharma has been on a dealmaking run to bolster its pipeline and portfolio since Boerner took the helm, acquiring RayzeBio for $4.1 billion , Mirati Therapeutics for $4.8 billion , and Karuna Therapeutics for $14 billion . Boerner sat down with Endpoints News on Tuesday, the day after his JPM presentation, for a wide-ranging discussion of the year ahead and his take on the future of Bristol Myers and the pharma industry. The following transcript has been edited for length and clarity. Andrew Dunn: We’ll get to JPM stuff, but I wanted to actually start with your 2002 PhD dissertation on why some pharmas are better at developing drugs than others. Chris Boerner: Oh, wow. That’s going way back. Dunn: I figured that’d be an interesting place to start. What was your answer in 2002, and how does that hold up today in 2026? Boerner: I have to remember first. What prompted me to look at that question was that I read an article that talked about Bristol Myers Squibb being really great at developing oncology drugs. I called somebody who had graduated from Berkeley a few years before, and I said, ‘I don’t understand why BMS is so much better.’ It turns out nobody has done that work. If you boil it down, the core element is that experience matters. It matters a lot. The data suggested that BMS and other companies that were very good at developing drugs in a given therapeutic area, they had a lot of history of developing in that space. What that meant was they knew the science, they had access to physicians who could help them prosecute that science, they knew how to develop the medicines in terms of clinical trials, so the data showed very clearly they were faster and had a higher probability of success. If you’re doing business development, the idea at the time was these virtual pharmaceutical companies. You could have MBAs and PhDs, and they could go select assets and in-license them and create companies. It turns out that’s very difficult to do, because you don’t know which science to pick. Dunn: What do you think is the biggest change from ’02 to ’26 on that topic? Boerner: It begs the question of how do you ever get into something new. The most successful companies, in many respects, have taken two paths. One is they buy infrastructure, and they don’t screw it up. They keep the people and the knowledge base that exists. Or they follow the science down a path, and it leads them into a new area. A good example of that is how Genentech got into ophthalmology. They followed the science for VEGF in oncology and wove their way over to ophthalmology because that’s where the science takes them. Dunn: There’s not a dogmatic approach of “We don’t do that.” Boerner: Exactly. Companies can be overly strategic and just say, well, we’ll just put blinders on that. You’ve got to be careful about that. When we acquired Rayze right after I became CEO, we made the decision to keep that largely separate. As a result, we did the most important thing: We kept all of those people who had deep expertise. Fundamentally, we’ve kept that as a freestanding unit, and that very much ties into one of the insights I had coming out of that Berkeley work. Dunn: My first JPM was 2019, the year of the Bristol-Celgene deal. Boerner: That was a pretty big deal to happen. Dunn: How do you judge the success of that deal? What are the lessons learned, and anything you’d have done differently? Boerner: Bristol Myers Squibb going into that acquisition, we were a company heavily indexed on two medicines: Eliquis and Opdivo. Every earnings call was about those two medicines. What we accomplished with that deal is to broaden the discussion. I highlighted yesterday that within our growth portfolio, we’ve got four medicines young in the lifecycle, all annualizing to over $1 billion in sales: Camzyos, Breyanzi, Opdualag, and Reblozyl, which is actually analyzing to over $2 billion. That diversity in your business is a real strongpoint for us because you’re not beholden to one or two blockbuster medicines as you think about what the growth trajectory of the company is going to be in the long term. Lessons learned for the future: How you integrate a big acquisition like that has to be done very thoughtfully. There are places we probably could have integrated faster. Dunn: Is there an example? Boerner: Three or four years later, we were still surveying the employee base to say were you heritage Celgene or heritage BMS? But at the time we were doing that, the majority of the people at the company had joined after the Celgene acquisition. In some ways, we ended up distracting ourselves with that, which is one big challenge with any big acquisition. Dunn: $30 billion in deals over the past two years. Do you have a target deal size or general M&A capacity for 2026? Boerner: We are somewhat agnostic on size. Our criteria remain the same: One, it has to be an area of science we know. Two, it has to make strategic sense. And finally, perhaps obviously, but in some cases not obviously, it’s got to make financial sense. Dunn: Is the idea of a megamerger on the table in 2026? Boerner: That’s not our primary focus, to be honest with you. Our primary focus is on doing deals more akin to the ones you’ve seen us do over the last three or four years. Dunn: What led to increasing Bristol’s exposure to neurology, particularly with the anti-psychotic Cobenfy, and what do you see as the future of these diseases, given they feel like the hardest to make traction on? Boerner: They are hard, but the nice thing about the history of Bristol Myers Squibb is we focus on hard things. The next frontier in healthcare, I believe, is going to be brain health. We have some mid-stage programs in Alzheimer’s disease. I’m looking forward to seeing them continue to progress. When we made the decision to invest in Karuna, we were making the decision to do two things: first, commit to neuroscience, not just neurodegeneration, but neuropsych. Because of the nature of the asset we were acquiring and the potential to not just focus on schizophrenia but Alzheimer’s disease psychosis, agitation and cognition, it was a bridge between neuropsych and neurodegeneration. We had already made the commitment to be in this space, and this was an opportunity to expand, but also to bridge. Dunn: Biggest potential opportunity and risk in working with the Trump administration in 2026? Boerner: The biggest opportunity — one of the things that has been refreshing with this administration is they have recognized that access and affordability are absolutely critical. Every dollar associated with a branded medicine, about 50% of that dollar goes to somebody who didn’t take any risk in the discovery, development and certainly isn’t involved in the commercialization of that product. There’s a real opportunity for us to find ways to simplify this healthcare system. The risk, and it’s less of a risk associated with working with this administration, but it’s a risk for the United States in terms of the ecosystem that we have here. We have the best ecosystem for biopharmaceuticals in the world, and it is the envy of the world. This conference is an embodiment of a lot of that ecosystem that doesn’t exist in its form like this anywhere else in the world. But it is not a birthright. There are other places, China is notable among them, that have made a stated ambition and are making great progress in replicating that. The challenge we all have in the United States is how do you strengthen this ecosystem? We have a vested interest as a company that’s been in the United States for over 160 years to ensure that the future of the ecosystem here continues to be as attractive as it is today, if not more attractive. That is the fundamental challenge we have as an industry.

Acquisition

15 Dec 2025

·fibrogen.gcs-web.com

Roxadustat Granted Orphan Drug Designation for the Treatment of Myelodysplastic Syndromes by the U.S. Food and Drug Administration

Company is on track to file the Phase 3 protocol in the fourth quarter of 2025 SAN FRANCISCO, Dec. 15, 2025 (GLOBE NEWSWIRE) -- FibroGen, Inc. (NASDAQ: FGEN) today announced that the Office of Orphan Products Development of the U.S. Food and Drug Administration (FDA) has granted roxadustat Orphan Drug Designation for the treatment of myelodysplastic syndromes (MDS). “The Orphan Drug Designation granted to roxadustat for MDS underscores the significant treatment gap in this indication, and highlights patients’ need for additional convenient treatments that can provide durable response,” said Thane Wettig, Chief Executive Officer of FibroGen. “Roxadustat showed an improvement in transfusion-independence in a subset of patients with high transfusion burden in a post-hoc analysis from the Phase 3 MATTHERHORN trial, which along with its favorable tolerability profile and oral route of administration has the ability to set it apart from current second-line treatments. Our team is finalizing the Phase 3 protocol in this patient population for submission to the FDA in the fourth quarter of 2025.” There are approximately 58,000 patients diagnosed with LR-MDS in the U.S. with 85% of them suffering from anemia. Anemia in patients with MDS is associated with increased risk of cardiovascular complications and the need for blood transfusions. Transfusion-dependent patients suffer higher rates of complications and decreased quality of life. Current first-line treatments lead to transfusion independence in less than 50% of patients and relief is often temporary with limited options for second line and beyond treatments. In a post-hoc analysis from the Phase 3 MATTERHORN trial, roxadustat demonstrated transfusion independence benefits compared to placebo in patients with high transfusion burden. The FDA Orphan Drug Designation is granted to drugs intended for the treatment, diagnosis, or prevention of rare diseases that affect fewer than 200,000 people in the U.S. Benefits of the designation may include exemption from certain FDA fees, financial incentives for qualified clinical development, and seven years of market exclusivity in the U.S. following drug approval. About Myelodysplastic Syndromes Anemia Myelodysplastic syndromes (MDS) are a group of disorders characterized by dysfunctional progenitor blood cells and stem cells, resulting in chronic anemia in most patients. Annual incidence rates of MDS are estimated to be 4.9/100,000 adults in the U.S, of which 77% are considered lower-risk MDS. Approximately 80% of patients with MDS have anemia at the time of diagnosis, and around 60% of patients with MDS will experience severe anemia (hemoglobin <8 g/dL) at some point during the course of their disease. Anemia in patients with MDS is associated with increased risk of cardiovascular complications and the need for blood transfusion. Approximately 50% of patients with MDS require regular red blood cell transfusions. Transfusion-dependent MDS patients suffer higher rates of cardiac events, infections, and iron overload with the related complications. In addition, anemia frequently leads to significant fatigue, cognitive dysfunction, and decreased quality of life. Today, patients are routinely treated with erythropoiesis-stimulating agents (ESAs), luspatercept, imetelstat, or lenalidomide in lower-risk MDS with isolated del(5q), and hypomethylating agents (HMAs) in higher-risk disease. Only 35-40% of patients respond to current treatments and the durability of response is short. Moreover, these treatments are challenging to dose-calibrate and can only be administered via subcutaneous injection or through IV infusion. There remains a high unmet need for the treatment of anemia associated with MDS, and new strategies that provide durable response and the convenience of oral administration are highly desired in managing patients with MDS. About Roxadustat Roxadustat, an oral medication, is the first in a new class of medicines comprising HIF-PH inhibitors that promote erythropoiesis, or red blood cell production, through increased endogenous production of erythropoietin, improved iron absorption and mobilization, and downregulation of hepcidin. Roxadustat is approved in Europe, Japan, and numerous other countries for the treatment of anemia of CKD in adult patients on dialysis (DD) and not on dialysis (NDD). FibroGen has the sole rights to roxadustat in the United States, Canada, Mexico, and in all markets not held by AstraZeneca or licensed to Astellas. Astellas and FibroGen are collaborating on the commercialization of roxadustat for the treatment of anemia in territories including Japan, Europe, Turkey, Russia, and the Commonwealth of Independent States, the Middle East, and South Africa. About FibroGen FibroGen, Inc. is a biopharmaceutical company focused on development of novel therapies at the frontiers of cancer biology and anemia. Roxadustat (爱瑞卓®, EVRENZOTM) is currently approved in Europe, Japan, and numerous other countries for the treatment of anemia in chronic kidney disease (CKD) patients on dialysis and not on dialysis. The Company continues to evaluate the development plan for the Phase 3 trial of roxadustat in anemia associated with lower-risk myelodysplastic syndrome (LR-MDS) in the U.S. FG-3246 (also known as FOR46), a first-in-class antibody-drug conjugate (ADC) targeting CD46, is in Phase 2 development for the treatment of metastatic castration-resistant prostate cancer. This program also includes the development of FG-3180, an associated CD46-targeted PET biomarker. For more information, please visit www.fibrogen.com. Forward-Looking Statements This release contains forward-looking statements regarding FibroGen’s strategy, future plans and prospects, including statements regarding its commercial products and clinical programs and those of it and its collaboration partners Fortis and UCSF. These forward-looking statements include, but are not limited to, statements regarding the efficacy, safety, convenience, and potential clinical or commercial success of FibroGen products and product candidates, statements about regulatory interactions, and statements about FibroGen’s plans and objectives. These forward-looking statements are typically identified by use of terms such as “may,” “will”, “should,” “on track,” “could,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “predict,” “potential,” “continue” and similar words, although some forward-looking statements are expressed differently. FibroGen’s actual results may differ materially from those indicated in these forward-looking statements due to risks and uncertainties related to the continued progress and timing of its various programs, including the enrollment and results from ongoing and potential future clinical trials, and other matters that are described in FibroGen’s most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q, each as filed with the Securities and Exchange Commission (SEC), including the risk factors set forth therein. Investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this release, and FibroGen undertakes no obligation to update any forward-looking statement in this press release, except as required by law. For Investor Inquiries: David DeLucia, CFA Senior Vice President and Chief Financial Officer ir@fibrogen.com Source: FibroGen, Inc.

Phase 2Phase 3Orphan DrugDrug ApprovalClinical Result

07 Dec 2025

·www.prnewswire.com

Rigel Presents Updated Data from the Ongoing Phase 1b Study Evaluating R289 in Patients with Lower-Risk MDS at the 67th ASH Annual Meeting and Exposition

R289 continues to be generally well tolerated and at doses of ≥500 mg QD preliminary efficacy was observed in elderly, heavily pre-treated lower-risk MDS patients RBC-TI was achieved by 33% (6/18) of evaluable transfusion dependent patients receiving R289 doses ≥500 mg QD, including 40% (2/5) in the 500 mg BID dose group SOUTH SAN FRANCISCO, Calif., Dec. 7, 2025 /PRNewswire/ -- Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL), a commercial stage biotechnology company focused on hematologic disorders and cancer, today announced updated data from its ongoing Phase 1b study evaluating R2891, an oral prodrug of R835, a potent and selective dual inhibitor of interleukin receptor-associated kinases 1 and 4 (IRAK1/4), in patients with relapsed or refractory (R/R) lower-risk myelodysplastic syndrome (MDS). The data are being presented today in an oral session by Dr. Guillermo Garcia-Manero at the 67th American Society of Hematology (ASH) Annual Meeting and Exposition being held December 6-9, in Orlando, Florida and virtually. "New therapies are needed for patients with transfusion dependent lower-risk MDS. We're pleased to share these updated study results, which underscore the potential of R289 to become a treatment option for these patients," said Lisa Rojkjaer, M.D., Rigel's chief medical officer. "We look forward to concluding the dose expansion phase of the study and anticipate selection of the recommended Phase 2 dose for future clinical studies in the second half of 2026." Rigel's open-label Phase 1b study of R289 is evaluating the safety, tolerability, pharmacokinetics and preliminary efficacy in patients with R/R lower-risk MDS (NCT05308264). Enrollment in the dose escalation phase of the study was completed in July 2025. In October 2025, the first patient was dosed in the dose expansion phase where up to 40 patients will be randomized to either 500 mg once or twice daily to determine the recommended Phase 2 dose for future clinical trials. Key highlights from the updated data as of October 28, 2025, include: 33 patients were enrolled, representing a difficult-to-treat population. The median age was 75. The median number of prior therapies was 3 (range: 1-8); 76% (25) of patients had received luspatercept, 73% (24) had received an erythropoiesis stimulating agent (ESA), 67% (22) had received an hypomethylating agent (HMA) and 6% (2) had received imetelstat. 61% (20) of patients were high transfusion burden (HTB) at baseline. 67% (22) of patients were ring sideroblast (RS) negative. Median duration of treatment was 5.5 months (range: 0.9 - 27.7 months). R289 was generally well tolerated across all dose groups in this heavily pre-treated lower-risk MDS patient population, the majority of whom were HTB at baseline. The most common Grade 1/2 treatment-emergent adverse events (TEAEs) (in ≥18% of patients) were diarrhea (n=10, 30%), constipation and fatigue (each n=9, 27%), and creatinine increased and cough (each n=7, 21%). The most frequent Grade 3/4 TEAEs were anemia (n=6, 18%), neutrophil count decreased and pneumonia (each n=5, 15%), and alanine aminotransferase (ALT) increased and aspartate aminotransferase (AST) increased (each n=3, 9%). One (1) dose limiting toxicity (DLT) (Grade 4 AST increased/Grade 3 ALT increased) was reported in the 750 mg dose group. For evaluable transfusion dependent (TD) patients (≥16 weeks follow up) at dose levels of at least 500 mg QD and higher, 6/18 (33%) patients achieved durable red blood cell transfusion independence (RBC-TI) of >8 weeks [500 mg QD (1/3), 750 mg QD (2/5), 500/250 mg QD (1/5), 500 mg BID (2/5)]. Duration of RBC-TI was >16 weeks in 4 patients and >24 weeks in 3 patients. The median time to onset of RBC-TI was 1.9 months and the median duration of RBC-TI was 22.9 weeks. Peak hemoglobin increases of 2.9 to 6.1 g/dL compared to baseline occurred in patients achieving RBC-TI. Of the 6 patients achieving RBC-TI, 5 had received an HMA. At doses ≥500 mg QD, steady state R835 plasma concentrations reached or exceeded those associated with 50-90% inhibition of lipopolysaccharide (LPS)-induced cytokine release previously observed in healthy volunteers. Oral presentation details are as follows: Sunday, December 7, 2025, 9:45am to 10:00am ET Publication #: 489 Session Name: 637. Myelodysplastic Syndromes: Clinical and Epidemiological: Moving the Needle Through Novel Approaches in MDS and CMML Presentation Title: An Update of Safety and Efficacy Results from a Phase 1b Study of R289, a Dual IRAK 1/4 Inhibitor, in Patients with Relapsed/Refractory (R/R) Lower Risk Myelodysplastic Syndrome (LR-MDS) Presenter: Guillermo Garcia-Manero, M.D. R289 was previously granted Orphan Drug designation for the treatment of myelodysplastic syndromes and granted Fast Track designation for the treatment of previously-treated transfusion dependent lower-risk MDS by the FDA. About R289 R289 is a prodrug of R835, an IRAK1/4 dual inhibitor, which has been shown in preclinical studies to block inflammatory cytokine production in response to toll-like receptor (TLR) and interleukin-1 receptor (IL-1R) family signaling. TLRs and IL-1Rs play a critical role in the innate immune response and dysregulation of these pathways can lead to various inflammatory conditions. Chronic stimulation of both these receptor systems is thought to cause the pro-inflammatory environment in the bone marrow responsible for persistent cytopenias in lower-risk MDS patients.2 About Rigel Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL) is a biotechnology company dedicated to discovering, developing and providing novel therapies that significantly improve the lives of patients with hematologic disorders and cancer. Founded in 1996, Rigel is based in South San Francisco, California. For more information on Rigel, the Company's marketed products and pipeline of potential products, visit . R289 is an investigational compound not approved by the FDA. Sallman DA et al. Unraveling the Pathogenesis of MDS: The NLRP3 Inflammasome and Pyroptosis Drive the MDS Phenotype. Front Oncol. June 16, 2016. doi: Forward Looking Statements This press release contains forward-looking statements relating to, among other things, the potential outcomes of the dose escalation and expansion phase of the ongoing Phase 1b study of R289, the potential benefits of R289 as a therapeutic for MDS and lower-risk MDS, the existence of patients with an unmet medical need for such therapy, and Rigel's ability to further develop its clinical stage product candidates. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements and as such are intended to be covered by the safe harbor for "forward-looking statements" provided by the PSLRA. Forward-looking statements can be identified by words such as "plan", "potential", "may", "look to", "expects", "outcome", "will" and similar expressions in reference to future periods. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on Rigel's current beliefs, expectations, and assumptions and hence they inherently involve significant risks, uncertainties and changes in circumstances that are difficult to predict and many of which are outside of Rigel's control. Therefore, you should not rely on any of these forward-looking statements. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation, the risks and uncertainties of clinical trials and drug development; risks and uncertainties associated with the commercialization and marketing of R289; risks that the FDA or other regulatory authorities may make adverse decisions regarding R289; risks that clinical trials may not be predictive of real-world results or of results in subsequent clinical trials; risks that R289 may have unintended side effects, adverse reactions or incidents of misuses; the availability of resources to develop Rigel's product candidates; market competition; as well as other risks detailed from time to time in Rigel's reports filed with the Securities and Exchange Commission, including its Quarterly Report on Form 10-Q for the quarter ended September 30, 2025 and subsequent filings. Any forward-looking statement made by us in this press release is based only on information currently available to us and speaks only as of the date on which it is made. Rigel does not undertake any obligation to update forward-looking statements, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise, and expressly disclaims any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein, except as required by law. Contact for Investors & Media: Investors: Rigel Pharmaceuticals, Inc. 650.624.1232 [email protected] Media: David Rosen Argot Partners 646.461.6387 [email protected] SOURCE Rigel Pharmaceuticals, Inc. 21% more press release views with Request a Demo

Clinical ResultPhase 1Fast TrackOrphan DrugASH

100 Deals associated with Luspatercept-AAMT

External Link

KEGG	Wiki	ATC	Drug Bank
-	Luspatercept-AAMT	B03XA06	DB12281

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Non-transfusion dependent thalassaemia	European Union	Bristol-Myers Squibb Pharma EEIG	20 Mar 2023
Non-transfusion dependent thalassaemia	Iceland	Bristol-Myers Squibb Pharma EEIG	20 Mar 2023
Non-transfusion dependent thalassaemia	Liechtenstein	Bristol-Myers Squibb Pharma EEIG	20 Mar 2023
Non-transfusion dependent thalassaemia	Norway	Bristol-Myers Squibb Pharma EEIG	20 Mar 2023
Anemia	South Korea	Bristol Myers Squibb Co.	09 May 2022
Anemia, Sideroblastic	European Union	Bristol-Myers Squibb Pharma EEIG	25 Jun 2020
Anemia, Sideroblastic	Iceland	Bristol-Myers Squibb Pharma EEIG	25 Jun 2020
Anemia, Sideroblastic	Liechtenstein	Bristol-Myers Squibb Pharma EEIG	25 Jun 2020
Anemia, Sideroblastic	Norway	Bristol-Myers Squibb Pharma EEIG	25 Jun 2020
Myelodysplastic Syndromes	European Union	Bristol-Myers Squibb Pharma EEIG	25 Jun 2020
Myelodysplastic Syndromes	Iceland	Bristol-Myers Squibb Pharma EEIG	25 Jun 2020
Myelodysplastic Syndromes	Liechtenstein	Bristol-Myers Squibb Pharma EEIG	25 Jun 2020
Myelodysplastic Syndromes	Norway	Bristol-Myers Squibb Pharma EEIG	25 Jun 2020
Transfusion dependent anaemia	European Union	Bristol-Myers Squibb Pharma EEIG	25 Jun 2020
Transfusion dependent anaemia	Iceland	Bristol-Myers Squibb Pharma EEIG	25 Jun 2020
Transfusion dependent anaemia	Liechtenstein	Bristol-Myers Squibb Pharma EEIG	25 Jun 2020
Transfusion dependent anaemia	Norway	Bristol-Myers Squibb Pharma EEIG	25 Jun 2020
Transfusion-dependent Beta Thalassemia	European Union	Bristol-Myers Squibb Pharma EEIG	25 Jun 2020
Transfusion-dependent Beta Thalassemia	Iceland	Bristol-Myers Squibb Pharma EEIG	25 Jun 2020
Transfusion-dependent Beta Thalassemia	Liechtenstein	Bristol-Myers Squibb Pharma EEIG	25 Jun 2020

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Indication	Highest Phase	Country/Location	Organization	Date
Myelofibrosis	Phase 3	China	Celgene Corp.	23 Feb 2022
Polycythemia Vera	Phase 3	China	Celgene Corp.	23 Feb 2022
Thrombocythemia, Essential	Phase 3	China	Celgene Corp.	23 Feb 2022
Post-polycythemia vera myelofibrosis	Phase 3	United States	Celgene Corp.	25 Feb 2021
Post-polycythemia vera myelofibrosis	Phase 3	China	Celgene Corp.	25 Feb 2021
Post-polycythemia vera myelofibrosis	Phase 3	Japan	Celgene Corp.	25 Feb 2021
Post-polycythemia vera myelofibrosis	Phase 3	Argentina	Celgene Corp.	25 Feb 2021
Post-polycythemia vera myelofibrosis	Phase 3	Australia	Celgene Corp.	25 Feb 2021
Post-polycythemia vera myelofibrosis	Phase 3	Austria	Celgene Corp.	25 Feb 2021
Post-polycythemia vera myelofibrosis	Phase 3	Belgium	Celgene Corp.	25 Feb 2021

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Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ASH2025	Not Applicable	Myelodysplastic Syndromes First line	320	Luspatercept 1 mg/kg	tsbtlsyfoi(flxzlujlif) = rjsaziukah qjpzjnlsca (zjqnygbmxy ) View more	Positive	06 Dec 2025
				Luspatercept (TD)	intuhewbnm(yisebvbwzx) = wupvxxfdmg nkyjwmpgam (fmmgiaqodw )
NCT06045689 (MAXILUS, ASH2025)	Phase 3	Myelodysplastic Syndromes	105	Luspatercept 1.75 mg/kg	ligutctepo(rxufowgmgc) = oivzttwcfm olmydrwjfb (qypjvrjrho ) View more	Positive	06 Dec 2025
				Luspatercept 1.75 mg/kg	ligutctepo(rxufowgmgc) = ixrftzvjma olmydrwjfb (qypjvrjrho ) View more
ASH2025	Not Applicable	Hematologic Neoplasms	22	Luspatercept (refractory anemia associated with hematologic malignancies)	wcfdstnvtr(xnxawwejms) = vhbvfytecd onssosktag (dapivssgfx ) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	Myelodysplastic Syndromes First line	418	Luspatercept	scdryvlkfq(cqdczrtfrs) = qarvgdghhg ixlifsycwm (fkspsoppgo ) View more	Positive	06 Dec 2025
				ESAs	scdryvlkfq(cqdczrtfrs) = wfltjgruyh ixlifsycwm (fkspsoppgo ) View more
NCT04143724 (ASH2025)	Phase 2	Beta-Thalassemia	3	Luspatercept 1.0 mg/kg	uvzajvyfgi(elpgargpdl) = reported by 1 patient rgxnrjwvoq (jueaqqgstm ) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	Anemia, Myelophthisic	99	Luspatercept	zmzfxtevaf(dsyfptwigk) = itqbudtccw dmerlgenuh (rmryvylnot, 30.4 - NR) View more	Positive	06 Dec 2025
NCT02604433 (BELIEVE, ASH2025)	Phase 3	Transfusion-dependent Thalassemia	336	Luspatercept	ausbtlaqdk(gvsmgnopgd) = glbpfgbnrv khqevhvplh (wwyrpadlgb ) View more	Positive	06 Dec 2025
				Placebo	ausbtlaqdk(gvsmgnopgd) = jxshpzmswn khqevhvplh (wwyrpadlgb ) View more
ASH2025	Not Applicable	Hematologic Diseases	17	Luspatercept 1 mg/kg	juqtigfeat(xgwqfdyzjk) = vakipjykpa jtwhvhcxea (fbycggiexr ) View more	Positive	06 Dec 2025
				Luspatercept 1 mg/kg (patients with erythroid response)	icdbrcmxrr(qjpuhsjpbe) = uwpthefeld rgtpothtlo (jkbpkoprgl )
QOL-ONE Phoenix (ASH2025)	Phase 2	Anemia	22	Luspatercept 1.0 mg/kg	mgjevfrsva(aighjrnkul) = ttrkllvfiq ighznetnul (fwtlwudrxw ) View more	Positive	06 Dec 2025
NCT05664737 (ASH2025)	Phase 2	Alpha-Thalassemia	6	Luspatercept (Transfusion-dependent α-thalassemia HbH disease)	ajytnyzedv(nsnwzpidxa) = elzsytwvfs dprjxngptc (ltopuekdsy )	Positive	06 Dec 2025
				Luspatercept (Non-transfusion-dependent α-thalassemia HbH disease)	ajytnyzedv(nsnwzpidxa) = tsihhybaqs dprjxngptc (ltopuekdsy )

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