RegulationOrphan Drug (United States), Priority Review (China), Orphan Drug (Japan), Orphan Drug (South Korea), Accelerated assessment (European Union)

Structure/Sequence

Molecular FormulaC8H12N4O5

InChIKeyNMUSYJAQQFHJEW-KVTDHHQDSA-N

CAS Registry320-67-2

914

Clinical Trials associated with Azacitidine

NCT07425808

/ Not yet recruitingPhase 2/3IIT

Gilteritinib in Combination With Azacitidine and Venetoclax Compared to Induction Chemotherapy "7+3" in Combination With a FLT3-inhibitor in Fit, Newly Diagnosed, FLT3-ITD Mutated Adult AML Patients: a Randomized Trial of the EORTC Leukemia Group and GIMEMA.

This international, multicenter, randomized (1:1), open-label phase II/III trial will evaluate the efficacy and safety of gilteritinib combined with azacitidine and venetoclax (experimental arm) versus standard "7+3" induction plus a FLT3inhibitor (quizartinib or midostaurin) (control arm) in newly diagnosed FLT3-ITD mutated AML patients eligible for intensive chemotherapy.

Start Date01 Dec 2026

Sponsor / Collaborator

European Organisation for Research & Treatment of Cancer

[+2]

NCT07463768

/ Not yet recruitingPhase 2IIT

A Single Arm Phase II Study Investigating the Efficacy and Safety of the Addition of Ivosidenib to Oral Azacitidine (Onureg®) in Patients Over 55 With Acute Myeloid Leukemia (AML) and IDH1 Mutation, in Complete Remission After Intensive Chemotherapy. A Study of the French AML Intergroup.

After remission post-induction and consolidation, maintenance therapy by an ivosidenib and oral azacitidine combination is susceptible to improve the prevention of AML relapse, which remains a major issue in the study population. We assume that the combination of ivosidenib with oral azacitidine will not be less well tolerated than in combination with the subcutaneous form, therapeutic regimen authorized until progression or toxicity. Ivosidenib and Onureg®, being already authorized treatments, it has been decided to use the classic administration schedules and dosages in combination.
The primary objective of the study is to evaluate relapse free survival (RFS) at 24 months in patients receiving oral azacitidine with ivosidenib.

Start Date01 Sep 2026

Sponsor / Collaborator

Servier Affaires Medicales SARL

NCT07439211

/ Not yet recruitingPhase 1IIT

Safety of Targeting PI3Kgamma Signaling With Azacitidine, Venetoclax and Eganelisib in Acute Myeloid Leukemia: A Phase 1 Study (GAVEL)

This study is to evaluate the safety and preliminary efficacy of adding the PI3K-gamma inhibitor, eganelisib, to a standard of care treatment option with combination venetoclax and azacitidine in participants with acute myeloid leukemia (AML).
The names of the study drugs involved in this research study are:
* Venetoclax (a type of BCL-2 inhibitor)
* Azacitidine (a type of Demethylating Agent)
* Eganelisib (a type of PI3K-gamma inhibitor)

Start Date01 Jul 2026

Sponsor / Collaborator

Dana-Farber Cancer Institute, Inc.

[+1]

100 Clinical Results associated with Azacitidine

100 Translational Medicine associated with Azacitidine

100 Patents (Medical) associated with Azacitidine

7,848

Literatures (Medical) associated with Azacitidine

01 Dec 2026·MOLECULAR BIOLOGY REPORTS

Dysregulated methylation of the human BOLL promoter hinders germ cell development from spermatogonia to spermatids

Article

Author: Ma, Hsiu-Yen ; Lin, Shih-Chieh ; Cheng, Yung-Hsuan ; Hou, Hsiu-Chiung ; Lin, Yung-Ming ; Lin, Tsung-Yen ; Lu, Chun-Wun ; Chen, Hsing-Yi ; Cheng, Yu-Sheng

PURPOSE:

BOLL, a highly conserved gene crucial for meiosis in spermatogenesis, is epigenetically regulated through DNA methylation in various species. This study aimed to determine whether BOLL promoter methylation contributes to its downregulation in azoospermic men with hypospermatogenesis (HS) and to explore its potential regulatory association with human spermiogenesis.

METHODS:

We conducted pyrosequencing to assess methylation levels at the putative BOLL promoter on testicular samples from azoospermic men with HS versus normal spermatogenesis (NR) and evaluate correlations with spermatogenic severity and gene expression. In silico CpG island (CGI) prediction and luciferase reporter assays were used to confirm methylation-sensitive regulatory regions. Pathway enrichment analysis identified biological processes associated with BOLL. Functional assays using BOLL overexpression in HEK293T cells and GC2 cells, as well as demethylation using 5-Aza-2'-deoxycytidine (5-AZA) in GC2 cells, were conducted to explore downstream transcriptional effects.

RESULTS:

Eight CpG sites (CpGs) within the BOLL promoter CGI were significantly hypermethylated in HS samples, with seven exhibiting a significant inverse correlation with the spermatogenic score, and three with BOLL transcript levels. The region from - 1434 to + 180 was confirmed as a methylation-sensitive promoter. Gene ontology analysis indicated that spermatid development and differentiation were the top BOLL-associated pathways. BOLL overexpression by plasmid transfection in HEK293T cells and GC2 cells upregulated spermatid-specific genes (TNP2 and GAPDHS), while concurrently suppressing spermatogonia-associated markers (ID4 and PIWIL4). In GC2 cells, 5-AZA treatment enhanced Boll expression and induced downstream spermatid-specific genes, including Prm2, which was not responsive to BOLL overexpression alone.

CONCLUSION:

We identified specific BOLL promoter CpGs that are hypermethylated in HS and associated with reduced gene expression. These findings suggest that promoter methylation may contribute to BOLL silencing and impaired spermatid differentiation, supporting its regulatory role in human spermiogenesis.

01 Apr 2026·LEUKEMIA RESEARCH

Venetoclax and azacitidine versus CAG for unfit patients with newly diagnosed acute myeloid leukemia: A propensity score-matched analysis

Article

Author: Fu, Qiang ; Wei, Yan ; Gao, Haitao ; Xu, Lanping ; Chang, Guanghui ; Jiang, Hao ; Zhang, Xiaohui ; Tang, Feifei ; Huang, Xiaojun ; Jiang, Qian

This study evaluated the clinical efficacy of venetoclax and azacitidine (VEN+AZA) versus CAG (cytarabine, aclarubicin, and granulocyte colony-stimulating factor) for newly diagnosed adult acute myeloid leukemia (AML) unfit patients. There were 248 patients received VEN+AZA and 104 patients received CAG. A PSM (Propensity Score-Matching) was conducted using the nearest neighbor method in a 1:2 ratio to minimize bias. As a result, 170 patients in the VEN+AZA cohort were matched with 85 patients in the CAG cohort. In propensity-matched group, VEN+AZA had higher CRc rate than the CAG (all cohort, 67.9 % vs 55.5 %, P = 0.04), especially for age ≥ 60 y (OR=1.085, P = 0.04), secondary AML(OR=1.802, P = 0.028), ELN intermediate-adverse karyotype(OR=1.33, P = 0.045), DNMT3A mutation (OR=1.32, P = 0.047) and IDH1/2 mutation (OR=1.952, P = 0.049), but patients with RUNX1::RUNX1T1 (OR=0.25, P = 0.044) favored CAG in CRc rate. The 3-year OS and EFS for VEN+AZA group and CAG group were 49.8 % VS. 49.8 % (P = 0.81) and 34.3 % VS. 31.7 % (P = 0.20). Patients with age ≥ 60 y (EFS, P = 0.032), ECOG≥ 2 (EFS, P = 0.047), secondary AML (OS, P = 0.015; EFS, P = 0.039), ELN intermediate-adverse karyotype (OS, P = 0.034; EFS, P = 0.044), RUNX1 mutation (OS, P = 0.003; EFS, P = 0.003) and IDH1/2 mutation (EFS, P = 0.039) showed a preference for VEN+AZA regarding OS, and patients with SRSF2 mutation favored CAG in OS (P = 0.031).

01 Apr 2026·EXPERIMENTAL HEMATOLOGY

Evaluation of dual BCL-2/BCL-XL inhibition in acute myeloid leukemia preclinical models with and without prior venetoclax therapy

Article

Author: Fabbri, Giulia ; Kingston, Nathan ; Andersen, Courtney ; Shah, Reecha ; Saeh, Jamal ; Cheung, Patricia

Acute myeloid leukemia (AML) is an aggressive hematologic malignancy that relies heavily on the antiapoptotic protein B-cell lymphoma 2 (BCL-2) for survival. Venetoclax, a BCL-2 inhibitor, exploits this dependency and is currently approved for treatment of elderly patients with AML. BCL-XL, another prosurvival protein in the BCL-2 family, has been identified as a key driver of both intrinsic and acquired resistance to venetoclax. Patients often develop resistance to BCL-2 inhibition through upregulation of BCL-XL. This study investigates the efficacy of a dual BCL-2/BCL-XL inhibitor and its combination with standard-of-care (SOC) agents. Dual BCL-2/BCL-XL inhibitor demonstrates robust activity in AML cell lines and patient-derived models, including activity in samples from patients who relapsed following venetoclax therapy. Its combination with SOC agents deepens the antileukemic activity both in vitro and in vivo. Among the combination regimens tested, cytarabine or hypomethylating agents (HMAs) drive strong blast reduction in patient samples previously exposed to venetoclax and yield improved survival in xenograft models derived from patients with AML who underwent prior venetoclax/5-azacytidine treatment. These preclinical findings support the clinical evaluation of dual BCL-2/BCL-XL inhibition in patients with AML, particularly, those who do not respond to venetoclax.

920

News (Medical) associated with Azacitidine

19 Mar 2026

·www.prnewswire.com

Curis Provides Fourth Quarter 2025 Business Update

Management to host conference call today at 4:30 p.m. ET LEXINGTON, Mass., March 19, 2026 /PRNewswire/ -- Curis, Inc. (NASDAQ: CRIS), a biotechnology company focused on the development of emavusertib (CA-4948), an orally available, small molecule IRAK4 and FLT3 inhibitor, today reported its business update and financial results for the quarter ended December 31, 2025. Operational Highlights TakeAim Lymphoma Lakshmi Nayak, MD, Director of the Center for CNS Lymphoma at Dana-Farber Cancer Institute in Boston, presented a poster with updated clinical data in Primary CNS Lymphoma (PCNSL) at the 30th Annual Meeting of the Society for Neuro-Oncology (SNO) in November 2025. The poster, titled Preliminary Safety and Efficacy of Emavusertib (CA-4948) in Combination with Ibrutinib in Relapsed/Refractory Primary Central Nervous System Lymphoma Patients, showed results for 24 patients who have received treatment with emavusertib and ibrutinib, a BTK inhibitor (BTKi), for at least 1 cycle (28 days): 5 of 5 BTKi-naïve patients achieved objective response (100% ORR) 7 of 19 BTKi-experienced patients achieved objective response (37% ORR) Median treatment duration for the 12 responders was 123 days (44 – 798 days) 9 additional patients were unable to complete 1 cycle of treatment; reasons for discontinuing treatment included adverse events unrelated to treatment, disease progression, and transition to hospice care. Cecilia Merrigan, DNP, Mayo Clinic in Rochester, presented a poster with initial clinical data in Secondary CNS Lymphoma (SCNSL) at the 30th Annual Meeting of the Society for Neuro-Oncology (SNO) in November 2025. The poster, titled Promising Efficacy Signal in Secondary CNS Lymphoma Patients Treated with Emavusertib and Ibrutinib, showed results for 2 patients who had previously progressed on a BTKi. Adding emavusertib to their ibrutinib treatment resulted in 1 complete response (CR) and 1 stable disease (SD) with a 38% reduction in disease burden. Curis continues to enroll PCNSL patients in the Company's TakeAim Lymphoma study of emavusertib in combination with ibrutinib which, as a result of discussions with FDA and EMA, is intended to support filings for accelerated approval in PCNSL in the US and Europe. Emavusertib has been granted orphan drug designation by both FDA and EMA in PCNSL. TakeAim CLL Curis initiated an open label Phase 2 clinical study of emavusertib in combination with zanubrutinib, a BTKi, in patients with Chronic Lymphocytic Leukemia (CLL). The goal of combining emavusertib with a BTKi is to enable a dual blockade of NF-kB, a key driver of disease in CLL and NHL, by inhibiting both the TLR and BCR pathways. The current standard of care is BTKi, which blocks the BCR pathway and can deliver high response rates, though typically only partial responses. Previous clinical studies have shown that adding emavusertib, which blocks the TLR pathway, to a BTKi regimen can enable patients with NHL to achieve deeper responses, including complete remission or undetectable minimal residual disease (MRD) and the potential for time-limited treatment, outcomes which represent the potential for a paradigm shift in the management of CLL. AML Christina Papayannidis, MD, IRCCS Azienda Ospedaliero Universitaria di Bologna, presented a poster with initial clinical data in frontline AML at the 67th ASH Annual Meeting in December. The AML triplet study (CA-4948-104) is evaluating the addition of emavusertib to the combination of azacitidine and venetoclax (aza-ven) in AML patients who have achieved complete remission on aza-ven but remain MRD-positive (MRD+). The first two cohorts in the study evaluate patients who received emavusertib for either 7 or 14 days in a 28-day cycle, in addition to their aza-ven treatment. The poster, titled Preliminary Pharmacokinetic and MRD Results from AML Patients Treated with 7- and 14-Day Dosing Schedule of Emavusertib added to Combination Therapy with Azacitidine and Venetoclax, showed results for 4 patients in the 7-day cohort and 6 patients in the 14-day cohort: 8 patients had central MRD samples 5 of 8 patients (62.5%) achieved MRD conversion (MRD+ to undetectable). Corporate On January 9, 2026, the Company announced the closing of a private placement (the "January 2026 PIPE Financing") with gross proceeds of up to $80.8 million, including initial gross proceeds of approximately $20.2 million with three series of warrants (A, B, and C) which can be exercised for up to $20.2 million each according to the terms and conditions of the financing agreement. All three series of warrants are currently exercisable at $0.75 per share and have the following termination conditions: Series A warrants terminate on January 8, 2031 Series B warrants terminate 30 days after the Company announces dosing of the fifth patient in the Phase 2 clinical trial in CLL, subject to conditions defined in the financing agreement Series C warrants terminate on January 8, 2027. Fourth Quarter 2025 Financial Results For the year ended December 31, 2025, Curis reported a net loss of $7.6 million, or $0.58 per share on both a basic and diluted basis, as compared to a net loss of $43.4 million, or $6.88 per share on both a basic and diluted basis in 2024. For the fourth quarter of 2025, Curis reported net income of $19.4 million or $1.23 per share on both a basic and diluted basis as compared to a net loss of $9.6 million or $1.25 on both a basic and diluted basis for the same period in 2024. Revenues, net were $9.4 million and $10.9 million for the years ended December 31, 2025 and 2024, respectively. Revenues are comprised of royalty revenues related to Genentech and Roche's net sales of Erivedge®. Revenues were $1.1 million and $3.3 million for the fourth quarters of 2025 and 2024, respectively. As previously announced, on November 6, 2025, the Company sold to TPC Investments Royalty LLC, a limited liability company managed by Oberland, its interest in Curis Royalty LLC. The sale included the Erivedge intellectual property, other assets associated with Erivedge and the License Agreement with Genentech ("Erivedge"), in exchange for upfront consideration of $2.5 million and a release of the Company's liability related to sale of future royalties to Oberland. In connection with such transaction, the Company transferred to Curis Royalty all rights to Curis Technology, Inventions and Joint Patents (each as defined in the License Agreement) and assigned the Company's rights, duties and obligations under the License Agreement to Curis Royalty. Following the sale, the Company is no longer entitled to revenues under the License Agreement. Research and development expenses were $28.3 million and $38.6 million for the years ended December 31, 2025 and 2024, respectively. The decrease was primarily attributable to lower employee-related, clinical, manufacturing and consulting costs. Research and development expenses were $5.8 million and $9.0 million for the fourth quarters of 2025 and 2024, respectively. General and administrative expenses were $14.0 million and $16.8 million for the years ended December 31, 2025 and 2024, respectively. The decrease was primarily attributable to lower employee-related and legal costs. General and administrative expenses were $2.9 million and $3.4 million for the fourth quarters of 2025 and 2024, respectively. Gain on release of liability related to sale of future royalties associated with sale of assets was the result of the sale of Erivedge. In the fourth quarter 2025, the Company recognized a non-cash $27.2 million gain and the liability related to sale of future royalties was extinguished. Other expense, net was $1.9 million for the year ended December 31, 2025 and other income, net was $1.2 million for the year ended December 31, 2024. The increase was partially attributable to an increase in expense related to the sale of future royalties and a decrease in interest income. Other expense, net was $0.3 million and $0.6 million for the fourth quarters of 2025 and 2024, respectively. As of December 31, 2025, Curis's cash and cash equivalents totaled $5.1 million, and the Company had approximately 12.9 million shares of common stock outstanding. Cash Runway Guidance Curis believes its cash and cash equivalents as of December 31, 2025, together with initial gross proceeds of $20.2 million received in January 2026 and expected gross proceeds of up to an additional $20.2 million from the exercise of the January 2026 PIPE Financing Series B Warrants upon the public announcement of dosing the 5th CLL patient in our TakeAim CLL study expected later this year, should enable the Company's planned operations into the second half of 2027. Conference Call Information Curis management will host a conference call today, March 19, 2026, at 4:30 p.m. ET, to discuss the business update and these financial results. To access the live conference call, please dial (800)-836-8184 from the United States or (646)-357-8785 from other locations, shortly before 4:30 p.m. ET. The conference call can also be accessed here on the Curis website in the Investors section. About Curis, Inc. Curis is a biotechnology company focused on the development of emavusertib, an orally available, small molecule IRAK4 and FLT3 inhibitor. Emavusertib is currently being evaluated in the TakeAim Lymphoma Phase 1/2 study (CA-4948-101) of emavusertib in combination with the BTK inhibitor, ibrutinib, in patients with relapsed/refractory primary central nervous system lymphoma (PCNSL) and in the TakeAim CLL Phase 2 study (CA-4948-203) of emavusertib in combination with the BTK inhibitor, zanubrutinib, in chronic lymphocytic leukemia (CLL). The Company's monotherapy and combination studies in acute myeloid leukemia (AML) are substantially complete, with additional funding the Company plans to continue development of emavusertib in AML. Emavusertib has received Orphan Drug Designation from the U.S. Food and Drug Administration for the treatment of PCNSL, AML and MDS and from the European Commission for the treatment of PCNSL. Curis, through its 2015 collaboration with Aurigene Discovery Technologies Limited, has the exclusive license to emavusertib (CA-4948). For more information, visit Curis's website at . Cautionary Note Regarding Forward-Looking Statements: This press release contains forward-looking statements within the meaning of the U.S. Private Securities Litigation Reform Act of 1995, including, without limitation, statements concerning Curis's cash runway or expectations with respect to the timing or exercise of the January 2026 PIPE Financing Series B Warrants; Curis's expectations with respect to the dosing of the fifth patient in the TakeAim CLL study, and the therapeutic potential of emavusertib in combination with zanubrutinib to improve treatment outcomes, achieve complete remissions and/or undetectable MRD, and/or reduce time on treatment for patients with CLL; Curis's expectations with respect to enrollment of BTKi naïve and BTKi experienced populations in the TakeAim Lymphoma study; statements regarding updated PCNSL data and the timing of such data from the TakeAim Lymphoma study, or the use of such data to support regulatory filings for approval of emavusertib in PCNSL, and the therapeutic potential and tolerability of emavusertib in patients with PCNSL. Forward-looking statements may contain the words "believes," "expects," "anticipates," "plans," "intends," "seeks," "estimates," "assumes," "predicts," "projects," "targets," "will," "may," "would," "could," "should," "likelihood", "continue," "potential," "opportunity," "focus," "strategy," "mission," or similar expressions. These forward-looking statements are not guarantees of future performance and involve risks, uncertainties, assumptions and other important factors that may cause actual results to be materially different from those indicated by such forward-looking statements. Curis may experience adverse results, delays and/or failures in its drug development programs and may not be able to successfully advance the development of its drug candidates in the time frames it projects, if at all. Curis's drug candidates may cause unexpected toxicities, fail to demonstrate sufficient safety and efficacy in clinical studies and/or may never achieve the requisite regulatory approvals needed for commercialization. Favorable results seen in preclinical studies and early clinical trials of Curis's drug candidates may not be replicated in later trials. Curis is dependent on the success of emavusertib and any delays in the development of emavusertib could have a material adverse effect on its business. There can be no guarantee that the collaboration agreement with Aurigene or the CRADA with NCI will continue for their full terms, that Curis or its collaborators will each maintain the financial and other resources necessary to continue financing its portion of the research, development and commercialization costs, or that the parties will successfully discover, develop or commercialize drug candidates under the collaboration. Curis will require substantial additional capital to fund its business. Based on its available cash resources, it does not have sufficient cash on hand to support current operations within the next 12 months from the date of this press release. Curis will require substantial additional funding to fund the development of emavusertib through regulatory approval and commercialization, and to support its continued operations. If it is not able to obtain sufficient funding, it will be forced to delay, reduce in scope or eliminate the development of emavusertib, including related clinical trials and operating expenses, potentially delaying the time to market for, or preventing the marketing of, emavusertib, which could adversely affect its business prospects and its ability to continue operations, and would have a negative impact on its financial condition and its ability to pursue its business strategies. Curis faces substantial competition. Curis and its collaborators face the risk of potential adverse decisions made by the FDA, EMA and other regulatory authorities, investigational review boards, and publication review bodies. Curis may not obtain or maintain necessary patent protection and could become involved in expensive and time-consuming patent litigation and interference proceedings. Unstable market and economic conditions, natural disasters, public health crises, political crises and other events outside of Curis's control, including its ability to regain and maintain its listing on the Nasdaq Capital Market, could significantly disrupt its operations or the operations of third parties on which Curis depends and could adversely impact Curis's operating results and its ability to raise capital. Other important factors that may cause or contribute to actual results being materially different from those indicated by forward-looking statements include the factors set forth under the captions "Risk Factor Summary" and "Risk Factors" in our most recent Form 10-K, and the factors that are discussed in other filings that we periodically make with the Securities and Exchange Commission. In addition, any forward-looking statements represent the views of Curis only as of today and should not be relied upon as representing Curis's views as of any subsequent date. Curis disclaims any intention or obligation to update any of the forward-looking statements after the date of this press release whether as a result of new information, future events or otherwise, except as may be required by law. SOURCE Curis, Inc. 21% more press release views with Request a Demo

Clinical ResultLicense out/inPhase 2Orphan DrugFinancial Statement

19 Mar 2026

·ir.sellaslifesciences.com

SELLAS Life Sciences Reports Full Year 2025 Financial Results and Provides Corporate Update

– Continued Advancement of Pivotal Phase 3 REGAL Trial of Galinpepimut-S (GPS) in Acute Myeloid Leukemia (AML) - Final Analysis upon Reaching 80 Events – – First Patient Dosed in Phase 2 Trial of SLS009 in Newly Diagnosed First Line AML – – Reported Positive Phase 2 Data of SLS009 in Relapsed/Refractory (r/r) AML at ASH 2025 – – $71.8 Million in Cash and Cash Equivalents as of December 31, 2025; Additional $42.6 Million in Proceeds Received in Q1 2026 to Date Through Warrant Exercises – NEW YORK, March 19, 2026 (GLOBE NEWSWIRE) -- SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) (“SELLAS’’ or the “Company”), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, today reported financial results for the full year ended December 31, 2025, and provided a corporate update. “Based upon continued progress in our GPS and SLS009 clinical programs in AML, we believe 2026 is shaping up to be a pivotal year for SELLAS,” said Angelos Stergiou, MD, ScD h.c., President and Chief Executive Officer of SELLAS. “We are advancing toward the final analysis of our Phase 3 REGAL trial evaluating GPS in AML patients who have achieved complete remission following second-line salvage therapy. Once we reach the required pre-specified 80th event, which will be announced, we will proceed with the necessary procedures towards database lock completion, statistical analysis, unblinding, and disclosure of topline results. If positive, REGAL could position GPS as a first- and best-in-class immunotherapeutic option in this AML population and serve as a significant value-inflection point for SELLAS.” Dr. Stergiou continued, “We also continue to make significant progress in our SLS009 clinical program for AML. Following the positive Phase 2 results of SLS009 in r/r AML presented at ASH, particularly in high-risk molecular subtypes, we have now dosed the first patient in the expansion cohort, which is evaluating SLS009 in newly diagnosed, first-line AML patients. Additionally, our preclinical data presented at ESMO in T-PLL demonstrated a statistically significant survival benefit of SLS009 both as monotherapy and in combination with venetoclax in a patient-derived xenograft model, further supporting the breadth of the CKD9 inhibition strategy. With a catalyst rich outlook, continued clinical execution, our strongest financial position in history, and expansion into earlier treatment settings, we look forward to an important year ahead.” Recent Corporate Highlights: European Collaboration to Advance SLS009 Clinical Program: In January 2026, SELLAS entered into a strategic collaboration with IMPACT-AML to expand SLS009 clinical program in Europe. Under the agreement, IMPACT-AML’s STREAM clinical network will conduct a study of SLS009 in combination with AZA/VEN in newly diagnosed AML patients. The enrollment of approximately 40 patients in Europe is anticipated in Q2 2026. Phase 3 REGAL Trial of GPS: On December 29, 2025, the Company provided an update on the pivotal Phase 3 REGAL trial, announcing that a total of 72 events had been recorded as of December 26, 2025, with the study remaining fully blinded. After reaching the required pre-specified 80th event, customary database lock and blinded data review procedures must be completed before statistical analysis, unblinding, and disclosure of topline results. Because the final analysis is event-driven, the timing of studies with overall survival as an endpoint can vary; SELLAS will announce the 80th event when it occurs. Phase 2 SLS009 in r/r AML: On December 7, 2025, the Company announced that clinical data from its ongoing Phase 2 study of SLS009, in combination with azacitidine (AZA) and venetoclax (VEN) for the treatment of patients with r/r AML with myelodysplastic syndrome-related changes (AML-MR) after prior VEN-based treatment were presented at the 67th American Society of Hematology (ASH) Annual Meeting and Exposition in December 2025. SLS009 in combination with AZA/VEN demonstrated clinically meaningful activity in patients with R/R AML-MR, and among the 35 evaluable patients, the overall response rate (CR/Cri/MLFS) was 46%, including 29% achieving CR/CRi. Patients harboring ASXL1 or TP53 mutations achieved response rates of 48% and 57%, respectively. The median overall survival (mOS) was exceedingly higher than the expected 2.6 months in this R/R AML patient population, and in the least pretreated cohort, mOS reached 8.9 months. Across all cohorts, patients with one prior line of therapy experienced the greatest benefit, with a 58% response rate and mOS not yet reached. No dose-limiting toxicities (DLTs) or treatment-related deaths were observed, and the combination was well tolerated. Expansion of SLS009 into Earlier-Line AML Treatment: Following the encouraging Phase 2 results of SLS009 in r/r AML particularly in patients with high-risk molecular subtypes, the Company has expanded the development program into earlier lines of therapy. After receiving constructive feedback from the FDA, SELLAS dosed the first patient in an 80-patient trial in newly diagnosed AML patients, as well as those who become refractory early to AZA/VEN treatment which we established through our extensive transcriptomics, genomics, and proteomics models. Preclinical Data on SLS009 in AML at the 2026 American Association for Cancer Research (AACR): The data shows that SLS009 induces apoptosis in AML cell lines, including those harboring high-risk ASXL1 and TP53 mutations. Pharmacodynamic changes were observed as early as 8 hours after treatment and became more pronounced over time, with reductions in MCL-1 and survivin, correlating with increased apoptosis. The poster, entitled, “Tambiciclib (SLS009), a CDK9 inhibitor, promotes apoptosis and suppresses MCL-1 levels in AML cell lines” will be presented on April 21, 2026. Preclinical Data on SLS009 in T-PLL Presented at ESMO 2025: In October 2025, preclinical data demonstrating statistically significant survival benefit of SLS009 were presented at the European Society for Medical Oncology (ESMO) Congress in Berlin, Germany. The results showed that SLS009, as a monotherapy and in combination with VEN, significantly prolonged survival compared to VEN alone in an in vivo patient-derived xenograft model of T-cell prolymphocytic leukemia (T-PLL). These findings further support the therapeutic potential of SLS009 to improve outcomes across multiple hematologic malignancies. Virtual R&D Day on Advancing Novel Therapies in AML: On October 29, 2025, SELLAS hosted a virtual R&D Day on “Advancing Novel Therapies in Acute Myeloid Leukemia (AML)” featuring key opinion leaders and Company management. The event provided a detailed review of the Company’s ongoing Phase 3 REGAL trial of GPS and the SLS009 program, underscoring each therapy’s potential to address multiple stages of disease progression along the AML treatment continuum. To access a replay of the R&D Day, please click here. Received $67.2 Million in Gross Proceeds from Warrant Exercises in 2025: In September and October 2025, SELLAS received a total of approximately $54.6 million in gross proceeds from the immediate exercise of existing warrants, including $23.6 million from warrants issued in January 2025 and $31.0 million from warrants issued in March and August 2024. An additional $12.6 million in proceeds was received during 2025 from the exercise of other previously outstanding warrants. Received Additional $42.6 Million in Proceeds from Warrant Exercises in Q1 2026: Subsequent to December 31, 2025, the Company received an additional $42.6 million in proceeds from the exercise of previously outstanding warrants. These additional proceeds bolster the reported $71.8 million cash and cash equivalents as of December 31, 2025, and provide the Company with the strongest financial position in its history. Financial Results for the Full Year 2025: Research and Development Expenses: Research and development expenses for the year ended December 31, 2025, were $16.0 million, compared to $19.1 million for the year ended December 31, 2024. The decrease was primarily due to decreases in clinical trial expenses and clinical and regulatory consulting costs, which were primarily driven by the completion of enrollment in the REGAL study in the first quarter of 2024. General and Administrative Expenses: General and administrative expenses for the year ended December 31, 2025, were $12.3 million, as compared to $12.4 million for the year ended December 31, 2024. The decrease was primarily attributable to a decrease in personnel related expenses. Net Loss: The net loss was $26.9 million for the year ended December 31, 2025, or a basic and diluted loss per share of $0.25, as compared to a net loss of $30.9 million for the year ended December 31, 2024, or a basic and diluted loss per share of $0.50. Cash Position: As of December 31, 2025, cash and cash equivalents totaled approximately $71.8 million. Subsequent to December 31, 2025, the Company received an additional $42.6 million in proceeds from the exercise of previously outstanding warrants. About SELLAS Life Sciences Group, Inc. SELLAS is a late-stage clinical biopharmaceutical company focused on the development of novel therapeutics for a broad range of cancer indications. SELLAS’ lead product candidate, GPS, is licensed from Memorial Sloan Kettering Cancer Center and targets the WT1 protein, which is present in an array of tumor types. GPS has the potential as a monotherapy and combination with other therapies to address a broad spectrum of hematologic malignancies and solid tumor indications. The Company is also developing SLS009 (tambiciclib) - potentially the first and best-in-class differentiated small molecule CDK9 inhibitor with reduced toxicity and increased potency compared to other CDK9 inhibitors. Data suggests that SLS009 demonstrated a high response rate in AML patients with unfavorable prognostic factors including ASXL1 mutation, commonly associated with poor prognosis in various myeloid diseases. For more information on SELLAS, please visit www.sellaslifesciences.com. Forward-Looking Statements This press release contains forward-looking statements. All statements other than statements of historical facts are “forward-looking statements,” including those relating to future events. In some cases, forward-looking statements can be identified by terminology such as “plan,” “expect,” “anticipate,” “may,” “might,” “will,” “should,” “project,” “believe,” “estimate,” “predict,” “potential,” “intend,” or “continue” and other words or terms of similar meaning. These statements include, without limitation, statements related to the GPS clinical development program, including the REGAL study and the timing of future milestones related thereto. These forward-looking statements are based on current plans, objectives, estimates, expectations, and intentions, and inherently involve significant risks and uncertainties. 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, risks and uncertainties with oncology product development and clinical success thereof, the uncertainty of regulatory approval, and other risks and uncertainties affecting SELLAS and its development programs as set forth under the caption “Risk Factors” in SELLAS’ Annual Report on Form 10-K filed on March 19, 2026 and in its other SEC filings. Other risks and uncertainties of which SELLAS is not currently aware may also affect SELLAS’ forward-looking statements and may cause actual results and the timing of events to differ materially from those anticipated. The forward-looking statements herein are made only as of the date hereof. SELLAS undertakes no obligation to update or supplement any forward-looking statements to reflect actual results, new information, future events, changes in its expectations, or other circumstances that exist after the date as of which the forward-looking statements were made. Investor Contact John Fraunces Managing Director LifeSci Advisors, LLC jfraunces@lifesciadvisors.com SELLAS LIFE SCIENCES GROUP, INC. CONSOLIDATED STATEMENTS OF OPERATIONS (Amounts in thousands, except share and per share data) SELLAS LIFE SCIENCES GROUP, INC. CONSOLIDATED BALANCE SHEETS (Amounts in thousands, except share and per share data)

Clinical ResultPhase 2ASHFinancial Statement

16 Mar 2026

·www.globenewswire.com

SELLAS Life Sciences Announces Enrollment of First Patient in Newly Diagnosed First-Line AML Trial of SLS009

- Study to Enroll 80 Patients Unlikely to Benefit from Venetoclax/Azacitide (Aza/Ven) Therapy in the US and Europe - - SELLAS’ Predictive Biomarker and AI Assisted Precision Medicine Models to be Utilized - - Topline Data Expected in Q4 2026 – March 12, 202 -- SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) (“SELLAS” or the “Company”), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, today announced the first patient has been enrolled in its randomized Phase 2 trial evaluating SLS009 (tambiciclib), a highly selective CDK9 inhibitor in newly diagnosed, first-line acute myeloid leukemia (AML) patients. The newly initiated NCT04588922 is designed to enroll approximately 80 patients and includes two AML cohorts with high unmet need and greatest potential benefit: Predictive biomarker cohort: Newly diagnosed patients unlikely to benefit from standard aza/ven therapy based on molecular profiling Early resistance cohort: Patients who initiate treatment with aza/ven but demonstrate a confirmed lack of any response after two treatment cycles “Enrolling the first patient in this first-line AML Phase 2 study represents an important milestone in the advancement of our SLS009 program and reflects our precision medicine strategy to address challenging subgroups of AML patients through our extensive transcriptomics, genomics, and proteomics models we have established,” said Angelos Stergiou, MD, ScD hc, President and Chief Executive Officer of SELLAS. “The strength of our Phase 2 data in r/r AML, particularly in high-risk molecular subtypes, together with the FDA's guidance, provided a strong foundation for moving into earlier lines of therapy. By targeting molecularly defined subgroups and those demonstrating early non-response, we aim to address a critical need in frontline AML and position SLS009 for potential registrational development.” This milestone follows the Company’s previously reported positive Phase 2 results in relapsed/refractory (r/r) AML and the FDA’s guidance recommending advancement into a study that includes newly diagnosed AML patients eligible for venectoclax and azacitidine (aza/ven) therapy. In the completed Phase 2 trial in r/r AML, SLS009 in combination with aza/ven met all endpoints, exceeding the pre-specified ORR threshold of 20%, demonstrating strong efficacy and favorable safety and tolerability with robust anti-tumor activity: 33% overall response rate (ORR) in all evaluable patients across all cohorts and dose levels, and 40% ORR for the recommended 30mg BIW dose level. ORR of 44% in AML patients with myelodysplasia-related changes (AML-MR) at the 30mg BIW dose ORR of 50% in ASXL1-mutated AML-MR at 30 mg BIW dose levels and 50% ORR in M4/M5 subtypes Median overall survival (mOS) of 8.8 months in patients treated with 30mg BIW, with a median of 1 prior line of therapy, and mOS of 8.9 months in AML MR patients vs 2.4 months with best available therapy. SELLAS is a late-stage clinical biopharmaceutical company focused on the development of novel therapeutics for a broad range of cancer indications. SELLAS’ lead product candidate, GPS, is licensed from Memorial Sloan Kettering Cancer Center and targets the WT1 protein, which is present in an array of tumor types. GPS has the potential as a monotherapy and combination with other therapies to address a broad spectrum of hematologic malignancies and solid tumor indications. The Company is also developing SLS009 (tambiciclib) - potentially the first and best-in-class differentiated small molecule CDK9 inhibitor with reduced toxicity and increased potency compared to other CDK9 inhibitors. Data suggests that SLS009 demonstrated a high response rate in AML patients with unfavorable prognostic factors including ASXL1 mutation, commonly associated with poor prognosis in various myeloid diseases. The content above comes from the network. if any infringement, please contact us to modify.

Clinical Result

100 Deals associated with Azacitidine

External Link

KEGG	Wiki	ATC	Drug Bank
D03021	Azacitidine	L01BC07	DB00928

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Juvenile Myelomonocytic Leukemia	United States	Bristol Myers Squibb Co.	20 May 2022
Myeloproliferative Disorders	European Union	betapharm Arzneimittel GmbH	24 Mar 2020
Myeloproliferative Disorders	Iceland	betapharm Arzneimittel GmbH	24 Mar 2020
Myeloproliferative Disorders	Liechtenstein	betapharm Arzneimittel GmbH	24 Mar 2020
Myeloproliferative Disorders	Norway	betapharm Arzneimittel GmbH	24 Mar 2020
Philadelphia chromosome positive chronic myelogenous leukemia	China	Bristol-Myers Squibb Pharma EEIG	24 Apr 2017
High Risk Myelodysplastic Syndrome	Australia	Celgene Pty Ltd.	30 Nov 2009
Acute Myeloid Leukemia	European Union	Bristol-Myers Squibb Pharma EEIG	17 Dec 2008
Acute Myeloid Leukemia	Iceland	Bristol-Myers Squibb Pharma EEIG	17 Dec 2008
Acute Myeloid Leukemia	Liechtenstein	Bristol-Myers Squibb Pharma EEIG	17 Dec 2008
Acute Myeloid Leukemia	Norway	Bristol-Myers Squibb Pharma EEIG	17 Dec 2008
Chronic Myelomonocytic Leukemia	European Union	Bristol-Myers Squibb Pharma EEIG	17 Dec 2008
Chronic Myelomonocytic Leukemia	Iceland	Bristol-Myers Squibb Pharma EEIG	17 Dec 2008
Chronic Myelomonocytic Leukemia	Liechtenstein	Bristol-Myers Squibb Pharma EEIG	17 Dec 2008
Chronic Myelomonocytic Leukemia	Norway	Bristol-Myers Squibb Pharma EEIG	17 Dec 2008
Myelodysplastic Syndromes	United States	Bristol Myers Squibb Co.	19 May 2004

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Immunoblastic Lymphadenopathy	Phase 3	Japan	Celgene Corp.	06 Nov 2018
Neoplasms	Phase 3	Italy	Celgene Corp.	26 Feb 2013
Thrombocytopenia	Phase 3	Belgium	Celgene Corp.	22 Nov 2012
Transfusion dependent anaemia	Phase 3	Belgium	Celgene Corp.	22 Nov 2012
Bone Marrow Neoplasms	Phase 3	Netherlands	Celgene Corp.	20 Feb 2009
Chronic Myelogenous Leukemia	Phase 3	Czechia	Pharmion Corp.	31 Jan 2005
Anemia, Refractory, With Excess of Blasts	Phase 3	United States	Celgene Corp.	01 Nov 2003
Anemia, Refractory, With Excess of Blasts	Phase 3	Australia	Celgene Corp.	01 Nov 2003
Anemia, Refractory, With Excess of Blasts	Phase 3	Bulgaria	Celgene Corp.	01 Nov 2003
Anemia, Refractory, With Excess of Blasts	Phase 3	Czechia	Celgene Corp.	01 Nov 2003

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05197426 (CTgov)	Phase 2	Acute Myeloid Leukemia	19	CC-486 (Treatment 1)	aeswqrdojk(lqnijftabp) = yqmvxytgcd yjpcslxvzq (ediszxjdqk, rgvckfpazk - scbcxgpmbb) View more	-	11 Feb 2026
				Placebo (Treatment 2)	aeswqrdojk(lqnijftabp) = ydjhdmawuu yjpcslxvzq (ediszxjdqk, bzuvovclgg - inkfaoejzs) View more
Tandem Meetings ASTCT and CIBMTR2026	Not Applicable	Myeloid Tumor	50	Azacitidine (AZA) + Azacitidine (AZA)Infusion (DLI)	gdpkuuzgfq(sjxzrqeowl) = yyzacvtvvr negkryzrny (pvecuccezh ) View more	Positive	04 Feb 2026
				Donor Lymphocyte Infusion (DLI)	gdpkuuzgfq(sjxzrqeowl) = ivqtrgopql negkryzrny (pvecuccezh ) View more
Tandem Meetings ASTCT and CIBMTR2026	Not Applicable	Acute Myeloid Leukemia	30	Azacitidine and Venetoclax	bxpbasnvvi(kgzzbmiirh) = vlpsrmoiwp fcozglhwvf (mmlioygxxn ) View more	Positive	04 Feb 2026
				Intensive Chemotherapy	bxpbasnvvi(kgzzbmiirh) = seakcuxpvy fcozglhwvf (mmlioygxxn ) View more
NCT01869114 (CTgov)	Phase 2	Adult acute myeloid leukemia with del(5q) \| Myelodysplastic Syndromes \| Acute Promyelocytic Leukemia	57	sirolimus+azacitidine (Arm A: Acute Myeloid Leukemia (AML))	rfbppixvox = hwzisbckha fbxqjhcmto (ixtjzfglqp, jyecmoreqc - ksnmtdoedq) View more	-	10 Dec 2025
				sirolimus+azacitidine (Arm B: High risk Myleodysplastic Syndrome (MDS))	rfbppixvox = tjfqxgbsmy fbxqjhcmto (ixtjzfglqp, zpaqkwtwoz - cgqkqlohqp) View more
ASH2025	Phase 2	acute leukemia Maintenance	58	Venetoclax 200 mgAzacitidine 32 mg/m2 + Venetoclax 200 mgAzacitidine 32 mg/m2	zigvlvqzfq(jtfqybezhw) = gqeewicxxp tqwhotacoa (cbhauvjgjo, 51.4 - 78.1) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	Acute Myeloid Leukemia with FLT3/ITD Mutation FLT3-ITD mutation	48	AzacitidineVenetoclaxGilteritinib + AzacitidineVenetoclaxGilteritinib + AzacitidineVenetoclaxGilteritinib (FLT3-ITD mutated AML + Refractory/Relapsed disease or persistent MRD)	qlzayclxtl(akfjlnjhrz) = kbyjexmsio delbasmpds (etegtschzs, 3.8 - 21.0) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	Acute Myeloid Leukemia	155	AZA-VEN	dwtapmqiba(ndlhdrbmft) = ytzmxapeym kegdlphvps (ijuhvwzcqv ) View more	Positive	06 Dec 2025
NCT06030089 (VENETACIBLE, ASH2025)	Phase 1	Acute Myeloid Leukemia First line Bcl-2 expression	20	Azacitidine + Venetoclax	ekhnyvwtav(nxaakkdsdi) = BH3 profiling predicted VEN resistance as early as day 5 of treatment. In the overall cohort, a significant increase in sensitivity to Mcl-1 (MS-1) (p < 0.01) and Bcl-xL (HRK) (p < 0.05) peptides was observed at day 5 post-treatment. Among responders, no significant changes were detected, whereas in non-responders, a significant increase in sensitivity to the Mcl-1 (MS-1) peptide was observed at day 5 post-treatment (p < 0.01) sqtjvjtpkb (lrzhgikuyz ) View more	Positive	06 Dec 2025
NCT06345365 (ASH2025)	Phase 2	Acute Myeloid Leukemia First line	22	Mitoxantrone liposome + cytarabine + azacitidine	mfeoioddbi(zcojxrwlcq) = sermyhtdac msiptwarxe (rcpjowynuf ) View more	Positive	06 Dec 2025
				daunorubicin + cytarabine + azacitidine	mfeoioddbi(zcojxrwlcq) = jfyelcdnwl msiptwarxe (rcpjowynuf ) View more
ASH2025	Not Applicable	Acute Myeloid Leukemia First line	870	CPX-351	fypughuput(vkhilyxpxj) = a consistently higher % time at home was observed in patients treated with CPX-351 (range: 80.4% to 97.6%) vs VEN-AZA (range: 74.7% to 90.2%), typically ~5-10% higher per month (monthly difference range: +3.5 to +11.9%). injtmtsini (malzkevxas ) View more	Positive	06 Dec 2025
				VEN-AZA

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