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Agios to Present New Data at EHA 2026 Reinforcing the Significant Therapeutic Impact of Mitapivat Across Multiple Rare Hemolytic Anemias

On May 12, 2026 Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, reported that new data on mitapivat, an oral pyruvate kinase (PK) activator, will be featured in oral and poster presentations during the 31st European Hematology Association (EHA) (Free EHA Whitepaper) Congress (EHA 2026) in Stockholm, Sweden, June 11-14, 2026.

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"Our presentations at EHA (Free EHA Whitepaper) 2026 build on over a decade of clinical and preclinical research that has consistently demonstrated the transformative potential of mitapivat as a disease-modifying oral medicine for hemolytic anemias, including sickle cell disease and thalassemia," said Sarah Gheuens, M.D., Ph.D., Chief Medical Officer and Head of R&D, Agios. "The plenary session for our RISE UP Phase 3 trial is an important opportunity to present new data showcasing the strong anti-hemolytic profile of mitapivat and its potential to address the urgent need for novel therapeutic options in sickle cell disease. We look forward to sharing these results and engaging with the hematology community as we continue to drive meaningful progress for patients with rare blood diseases."

Select presentations at EHA (Free EHA Whitepaper) 2026 will include:

An oral presentation during the Plenary Abstracts Session on detailed efficacy and safety data from the global 52-week, randomized, double-blind, placebo-controlled RISE UP Phase 3 trial of mitapivat in patients aged 16 years or older with sickle cell disease. Results demonstrate that treatment with mitapivat significantly improved hemoglobin response (a ≥1.0 g/dL increase from baseline in average hemoglobin from Week 24 through Week 52) and reduced markers of hemolysis (red blood cell destruction). Additional analyses, including measures of clinical benefit and patient-reported outcomes that were not previously disclosed in the company’s November 2025 topline report, will also be presented. The RISE UP abstract was one of only six selected for this distinguished plenary session from thousands of submissions.
An oral presentation on long-term data from the ENERGIZE Phase 3 trial of mitapivat in adult patients with non-transfusion-dependent alpha- or beta-thalassemia. Of the 192 patients who received at least one dose of mitapivat or placebo in the double-blind period of ENERGIZE, 95% (n=182) opted to transition into the corresponding open-label extension (OLE) period, during which all patients receive mitapivat. The results show the durability of mitapivat treatment, with clinically meaningful hemoglobin improvements sustained for up to 127 weeks across the double-blind and OLE periods. During the OLE, nearly half (n=30/61) of non-responders who switched from placebo achieved a hemoglobin improvement (a ≥1 g/dL increase from baseline in average hemoglobin during any two consecutive OLE study visits), supporting a consistent treatment effect with mitapivat. Additionally, about one-third (n=23/68) of non-responders who received mitapivat during the double-blind period achieved a hemoglobin improvement during the OLE, suggesting a potential benefit of continued long-term treatment.

A related poster will highlight a subset of patients with higher baseline hemoglobin levels (≥9.5 g/dL) in ENERGIZE. Among these patients, 38.9% (n=7/18) in the mitapivat arm achieved a hemoglobin response (a ≥1 g/dL increase from baseline in average hemoglobin from Week 12 through Week 24) in the double-blind period, compared with 0% (n=0/11) in the placebo arm. Additionally, patients in the mitapivat arm showed a 5.1-point improvement in patient-reported fatigue scores compared with 0.8 points among those in the placebo arm, as measured by the least squares mean change from baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue from Week 12 to Week 24.
An oral presentation on the collaborator-led SATISFY Phase 2 trial of mitapivat in 24 patients with one of three rare hemolytic anemias: hereditary spherocytosis, hereditary xerocytosis, or congenital dyserythropoietic anemia type II. Treatment resulted in sustained hemoglobin improvements over 56 weeks. Notably, patients who achieved a hemoglobin response (a ≥1 g/dL increase from baseline in average hemoglobin during at least two consecutive study visits) also showed significant decreases in liver iron content, an important marker of downstream complications in hemolytic anemias.
In total, 10 presentations and publications led by Agios and external collaborators will be shared at EHA (Free EHA Whitepaper) 2026.

EHA 2026 Accepted Abstracts

Title Number Date/Time Presenter Acceptance
Sickle Cell Disease
Efficacy and Safety of Mitapivat in Sickle Cell Disease: Results From the Global, Randomized, Phase 3 RISE UP Trial S102 Saturday, June 13, 12:00 – 1:30 p.m. CEST Biree Andemariam, M.D., University of Connecticut Health Oral Plenary Session
Metabolic Profiles Associated With Point of Sickling Reflect Immunoregulation, Oxidative Stress, and Compensatory Mechanisms in Patients With Sickle Cell Anemia PF1264 Friday, June 12, 6:45 – 7:45 p.m. CEST

Amira Idrizovic, Ph.D., Vall d’Hebron Research Institute, Barcelona, Spain Poster
Impaired Function of Pyruvate Kinase Is Associated With Hemolysis‑Related Inflammation, Ineffective Erythropoiesis, and Endothelial Dysfunction in Adults With Sickle Cell Anemia PF1269 Friday, June 12, 6:45 – 7:45 p.m. CEST Marissa J.M. Traets, M.D., University Medical Center Utrecht, Netherlands Poster
Thalassemia
Long‑Term Hemoglobin Improvements in Non-Transfusion‑Dependent Alpha‑ or Beta‑Thalassemia: Results From the Open‑Label Extension of the Ongoing Phase 3 ENERGIZE Trial S440 Saturday, June 13, 5:15 – 6:30 p.m. CEST Hanny Al‑Samkari, M.D., Mass General Brigham Cancer Institute

Oral
Efficacy of Mitapivat in Patients With Non-Transfusion‑Dependent Alpha‑ or Beta‑Thalassemia With Baseline Hemoglobin ≥9.5 g/dL: Subgroup Analysis From the Phase 3 ENERGIZE Trial PF1281 Friday, June 12, 6:45 – 7:45 p.m. CEST Khaled M. Musallam, M.D., Ph.D., Burjeel Medical City, Abu Dhabi, United Arab Emirates
Poster
ENERGIZEKIDS: Mitapivat in Pediatric Patients With Non-Transfusion‑Dependent Alpha‑ or Beta‑Thalassemia PB4160 N/A Kathryn Dickerson, M.D., University of Texas Southwestern Medical Center
Publication
ENERGIZEKIDS‑T: Mitapivat in Pediatric Patients With Transfusion‑Dependent Alpha‑ or Beta‑Thalassemia PB4162 N/A Janet Kwiatkowski, M.D., MSCE, Children’s Hospital of Philadelphia Publication
Other
The Patient Experience of Fatigue in Individuals With Sickle Cell Disease, Thalassemia, and Pyruvate Kinase Deficiency PB4417 N/A Biree Andemariam, M.D., University of Connecticut Health Publication
Effects of Mitapivat on Iron Burden and Spleen Size in Erythrocyte Membranopathies and Congenital Dyserythropoietic Anemia Type II: 56‑Week Follow‑Up Results From the SATISFY Study S304 Thursday, June 11, 4:45 – 6:00 p.m. CEST Thomas Doeven, M.D., University Medical Center Utrecht, Netherlands Oral
Diamond‑Blackfan Anemia Syndrome Erythroid Progenitors Display Abnormal Metabolic Profile PS1781 Saturday, June 13, 6:45 – 7:45 p.m. CEST Veronica Riccardi, M.D., University of Verona, Italy Poster

Please refer to the EHA (Free EHA Whitepaper) 2026 online program for full session details and data presentation listings, and visit the Agios booth (#A1.04) onsite.

EHA 2026 Investor Event
Agios will host a conference call and live webcast during the EHA (Free EHA Whitepaper) 2026 congress on Saturday, June 13, at 9:00 a.m. ET (3:00 p.m. CEST). The live webcast will be accessible on the Investors section of the company’s website (www.agios.com) under the "Events & Presentations" tab. A replay of the webcast will be available on the company’s website approximately two hours after the event.

(Press release, Agios Pharmaceuticals, MAY 12, 2026, View Source [SID1234665519])

Bristol Myers Squibb and Hengrui Pharma Announce Strategic Agreements to Advance Innovative Medicines Across Oncology, Hematology, and Immunology

On May 12, 2026 Bristol Myers Squibb (NYSE: BMY, "BMS") and Hengrui Pharma ("Hengrui") (600276.SH; 01276.HK) reported the companies have entered into global strategic collaboration and license agreements to advance a portfolio of 13 early stage programs in oncology, hematology and immunology, with the goal of accelerating discovery and development of innovative medicines for the benefit of patients worldwide.

The agreements include four oncology/hematology assets from Hengrui, four immunology assets from BMS, and five innovative assets to be jointly discovered and developed by both companies, leveraging Hengrui’s discovery engine and platform technologies across several innovative modalities. Hengrui has the option to co-develop select assets and the potential to conduct certain commercialization activities globally with BMS.

Under the collaboration, BMS obtains exclusive worldwide rights to the Hengrui‑originated assets outside Chinese mainland, Hong Kong SAR, and Macau SAR (the "Hengrui Territory"), while Hengrui obtains exclusive rights to the BMS‑originated assets within the Hengrui Territory, with BMS retaining rights for the rest of the world. Hengrui will be fully responsible for early clinical development to accelerate clinical proof of concept for these programs.

Aligned with the collaborative innovation strategies of both BMS and Hengrui, these agreements reflect the companies’ continued focus on advancing innovative science through partnership in areas of significant unmet medical need. The collaboration brings together BMS’s differentiated research and discovery strengths, global clinical development capabilities, regulatory expertise, and commercial scale with Hengrui Pharma’s discovery engine, platform technologies, and efficient early-stage development expertise, enabling the advancement of a broad portfolio of high-value programs.

"This strategic collaboration reflects our commitment to advancing innovative science while maintaining a disciplined approach to portfolio management," said Robert Plenge, MD, PhD, Executive Vice President and Chief Research Officer, Bristol Myers Squibb. "By leveraging complementary capabilities across geographies, we aim to accelerate early clinical learning and make informed decisions that support driving top tier growth in the next decade and, ultimately, our mission to deliver medicines that help patients prevail over serious diseases."

"This broad strategic collaboration reflects a highly synergistic collaboration between two global innovators with complementary strengths. By leveraging Hengrui’s growing R&D capabilities and proven efficiency in discovering and advancing innovative therapies, we are poised to advance the best of both pipelines," said Frank Jiang, MD, PhD, Executive Vice President and Chief Strategy Officer of Hengrui Pharma. "It also reflects Hengrui’s continued commitment to strengthen our global presence. Together, we aim to deliver meaningful benefits to patients worldwide."

Under the terms of the agreement, BMS will pay Hengrui up to $950 million, including a $600 million upfront payment, a $175 million first anniversary payment, and a second contingent anniversary payment of $175 million in 2028. The potential total value of the agreement is up to approximately $15.2 billion, including the exercise of available options for the joint discovery programs and the achievement of applicable development, regulatory, and commercial milestones for all programs. In addition, Hengrui is eligible to receive tiered royalties on net sales of products commercialized outside the Hengrui Territory.

The transaction is subject to review under the Hart‑Scott‑Rodino Antitrust Improvements Act and other customary closing conditions. The parties expect that the agreement will close in the third quarter of 2026.

(Press release, Bristol-Myers Squibb, MAY 12, 2026, View Source [SID1234665453])

CERo Therapeutics Completes Second Ascending Dose Cohort of Phase 1 CER-1236 Trial

On May 11, 2026 CERo Therapeutics Holdings, Inc. (OTCQB: CERO) ("CERo" or the "Company"), an innovative cellular immunotherapy company advancing next-generation engineered T cell therapeutics that employ phagocytic mechanisms, reported that it has completed dosing and the 28-day dose-limiting toxicity ("DLT") assessment period for all three patients in the second cohort of its Phase 1 CERTAIN-T clinical trial of CER-1236 in hematologic malignancies.

In the second cohort, patients received CER-1236 at a total dose of 4 × 10⁶ cells/kg, administered as a split dose on Day 0 and Day 2. No DLTs were observed through the DLT assessment window, supporting continued dose escalation in accordance with the clinical protocol. The observed safety profile remains consistent with clinical data presented at the Tandem Meetings in February, which reported no cytokine release syndrome ("CRS"), immune effector cell-associated neurotoxicity syndrome ("ICANS"), DLTs, or treatment-related severe adverse events during the DLT assessment window.

CERo is now escalating to the planned 1 × 10⁷ cells/kg split-dose cohort and is screening patients for enrollment. This next cohort is expected to include patients with myelodysplastic syndrome ("MDS") and myelofibrosis ("MF"), reflecting the Company’s strategy to evaluate CER-1236 in myeloid disease settings characterized by inflammatory bone marrow microenvironments and impaired hematopoiesis.

The Company has now treated six patients in its Phase 1 trial. In the most recent cohort of three patients, CER-1236 was administered at an increased dose level, with no dose-limiting toxicities observed during the DLT assessment period. As previously reported, CERo has also observed expansion of infused CER-1236 cells following administration, supporting continued evaluation of CER-1236’s pharmacokinetic and pharmacodynamic profile as the study advances through dose escalation.

Robert Sikorski, M.D., Ph.D., CERo Chief Medical Officer, stated, "We are encouraged by the continued progress of the CERTAIN-T study, including completion of the second dose cohort and advancement to the next planned dose level. As we proceed in accordance with the clinical protocol, our priority remains the careful evaluation of the safety, tolerability, pharmacokinetic profile, and preliminary clinical activity of CER-1236. The planned inclusion of patients with MDS and MF reflects our interest in exploring CER-1236 across additional hematologic malignancies with significant unmet need and further assessing the potential of its differentiated phagocytic CAR-T mechanism."

The first-in-human, multicenter, open-label Phase 1/1b study is designed to evaluate the safety and preliminary efficacy of CER-1236. The trial was initially focused on AML patients, including those with relapsed/refractory disease, measurable residual disease, or newly diagnosed TP53-mutated AML, and has since expanded to include transfusion-dependent MDS ("TD-MDS"), high-risk MDS ("HR-MDS"), and post-JAK inhibitor MF. Primary endpoints include safety and tolerability, while secondary endpoints include pharmacokinetics and measures of clinical response, including overall response rate (ORR), complete response (CR), composite complete response (cCR), and measurable residual disease (MRD).

CERo Chief Executive Officer Chris Ehrlich added, "The advancement of CER-1236 through dose escalation continues at a steady pace and we remain focused on evaluating its potential across hematologic malignancies with significant unmet need. We look forward to providing additional clinical updates as the study progresses."

(Press release, Cero Therapeutics, MAY 11, 2026, View Source [SID1234665481])

Ernexa Therapeutics Releases Virtual Investor KOL Connect Segment Highlighting Ovarian Cancer Landscape and Lead Candidate ERNA-101

On May 11, 2026 Ernexa Therapeutics (Nasdaq: ERNA), an industry innovator developing novel cell therapies for the treatment of advanced cancer and autoimmune disease, reported the release of a Virtual Investor KOL Connect segment featuring Dr. Robert Pierce, Chief Scientific Officer of Ernexa.
 
As part of the segment, Dr. Pierce provides insight into the complexities of ovarian cancer, one of the most challenging malignancies to treat, and discusses the limitations of current therapeutic approaches. The discussion highlights the patient journey from diagnosis through treatment, emphasizing the persistent unmet need for more effective and durable treatment options.

Dr. Pierce also outlines the evolving treatment landscape and shares his perspective on how Ernexa is approaching ovarian cancer differently at the biological level. He discusses the Company’s lead product candidate, ERNA-101, and its potential to improve outcomes and enhance the patient experience compared to existing therapies.

The Virtual Investor KOL Connect segment featuring Ernexa Therapeutics is now available here.

In addition, Ernexa announced that Sanjeev Luther, President & Chief Executive Officer, and Dr. Robert Pierce, Chief Scientific Officer, will participate in a Virtual Investor Closing Bell event on Wednesday, May 13, 2026, at 4:00 PM ET.

As part of the event, Mr. Luther and Dr. Pierce will provide a corporate overview and discuss Ernexa’s recently announced preclinical ovarian cancer data for ERNA-101, which demonstrated complete tumor elimination and 100% survival in ovarian cancer models when combined with PD-1 blockade. The discussion will also cover the potential for ERNA-101 to address immunologically "cold" tumors by reshaping the tumor microenvironment to support stronger immune responses.

For more information about ERNA-101 and the Company’s development plans, visit www.ernexatx.com

(Press release, Ernexa Therapeutics, MAY 11, 2026, View Source [SID1234665480])

ArriVent BioPharma Reports First Quarter 2026 Financial Results

On May 11, 2026 ArriVent BioPharma, Inc. (Company or ArriVent) (Nasdaq: AVBP), a clinical-stage company dedicated to accelerating the global development of innovative biopharmaceutical therapeutics, reported financial results for the first quarter ended March 31, 2026, and highlighted recent Company progress.

"Our two ongoing pivotal firmonertinib trials in uncommon EGFR-mutant non-small cell lung cancer (NSCLC) continue to advance, with topline monotherapy data for frontline EGFR exon 20 insertion mutations expected in mid-2026 and our global Phase 3 pivotal ALPACCA study continuing to enroll patients globally," said Bing Yao, CEO of ArriVent. "At American Association for Cancer Research (AACR) (Free AACR Whitepaper), we presented preclinical data highlighting the unique structural features of firmonertinib that improve binding and enhance activity against EGFR mutant proteins, further strengthening confidence in the broad activity of firmonertinib in EGFR-mutant NSCLC."

Dr. Yao continued, "We also presented preclinical data for our antibody-drug conjugate (ADC), ARR-002 at AACR (Free AACR Whitepaper). This novel MUC16/NaPi2b dual-targeting tetravalent ADC demonstrated synergistic anti-tumor activity compared to single-target and bivalent ADCs, along with a favorable tolerability profile, supporting its best-in-class potential. Following the recent clearance of our Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA), we plan to initially advance ARR-002 into the clinic for ovarian and endometrial cancers. Our balance sheet continues to be strong with projected cash runway into the fourth quarter of 2027, and we are focused on continued execution across our key registrational catalysts."

First Quarter 2026 and Recent Highlights

Firmonertinib

New preclinical data for firmonertinib presented at AACR (Free AACR Whitepaper). Preclinical findings for EGFR inhibitor firmonertinib showcased high resolution crystal structure data supporting the ongoing pivotal Phase 3 study in frontline EGFR exon 20 insertion mutant NSCLC at the 2026 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting.
Received National Medical Products Administration (NMPA) accelerated approval in China in second-line EGFR exon 20 insertion mutations. In February 2026, our partner Shanghai Allist Pharmaceutical Technology Co., Ltd., received NMPA accelerated approval for firmonertinib for adults with locally advanced or metastatic NSCLC who have progressed on or after prior platinum-based chemotherapy or who are intolerant to platinum-based chemotherapy and who have been tested for the presence of EGFR exon 20 insertion mutations.

Pipeline

Clinical advancement of ADC lead ARR-217 (MRG007). ArriVent received FDA IND clearance for ARR-217 and dosed its first patient in March 2026 and continues to advance the ongoing Phase 1 dose escalation for ARR-217, a CDH17 targeted ADC, in gastrointestinal malignancies in partnership with Lepu Biopharma Co., Ltd.
IND clearance for ARR-002 in endometrial and ovarian cancer. In May 2026, ArriVent received IND clearance from the FDA for ARR-002, a novel dual-target MUC16/NaPi2b tetravalent ADC, for ovarian and endometrial cancers. The Company plans to advance ARR-002 into the clinic through a first-in-human study evaluating safety, dosing, and early signals of efficacy.
New preclinical data for ARR-002 presented at AACR (Free AACR Whitepaper). ArriVent presented preclinical data on ARR-002, also known as AV-P138-ADC, characterizing its superior ADC potential in ovarian and endometrial cancers and planned advancement towards clinical evaluation. The data was presented with Aarvik Therapeutics, Inc., who also presented data for ARR-002 as part of an oral presentation at the Clinical Research Mini Symposium at AACR (Free AACR Whitepaper).

Upcoming Milestones

Firmonertinib pivotal EGFR exon 20 insertion data. Top-line firmonertinib monotherapy data from the global pivotal FURVENT Phase 3 (NCT05607550) study for first-line EGFR exon 20 insertion mutant NSCLC is projected to be in mid-2026.
Initiate Phase 1 dose optimization for ARR-217. Complete Phase 1 dose escalation and initiate dose optimization for ARR-217, a CDH17 targeting ADC program, in the second half of 2026.
Dosing of first patient with ARR-002. Dosing of first patient with ARR-002 in a Phase 1 trial expected in the second half of 2026.

2026 Financial Results

As of March 31, 2026, the Company had cash and investments of $326.4 million, which is expected to fund operations into 4Q 2027.
Net cash used in operations was $41.9 million and $68.0 million for the three months ended March 31, 2026 and 2025, respectively.
Research and development expenses were $37.6 million and $61.3 million for the three months ended March 31, 2026 and 2025, respectively.
General and administrative expenses were $8.5 million and $5.5 million for the three months ended March 31, 2026 and 2025, respectively.
Net loss was $43.3 million and $64.4 million for the three months ended March 31, 2026 and 2025, respectively.

(Press release, ArriVent Biopharma, MAY 11, 2026, View Source [SID1234665479])