Akari Therapeutics Reports Breakthrough Preclinical Data Demonstrating Synergistic Activity of AKTX-101 with KRAS Inhibition in KRAS-Mutated Pancreatic Cancer Models

On May 21, 2026 Akari Therapeutics, Plc (Nasdaq: AKTX), an oncology biotechnology company developing antibody drug conjugates (ADCs) with a novel RNA splicing modulator payload, reported positive preclinical data featured in an online abstract released in connection with the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting 2026 highlighting synergistic cytotoxic activity of AKTX-101 in combination with KRAS inhibition in KRAS-mutated pancreatic cancer models. Access the abstract here.

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The data further expands the potential opportunity for Akari’s lead TROP2-targeting ADC, AKTX-101, and its proprietary RNA spliceosome-modulating payload beyond the Company’s current Phase 1 development plan and supports the broader applicability of targeting RNA splicing as a potential way to treat difficult-to-treat KRAS-driven cancers.

The ASCO (Free ASCO Whitepaper) abstract evaluated the activity of AKTX-101 in combination with adagrasib, a KRAS inhibitor, across pancreatic cancer cell lines harboring KRAS G12D and KRAS G12C. In these studies, the combination of AKTX-101 and adagrasib demonstrated synergistic cell killing in KRAS mutant cell lines driven by G12C and G12D. This synergistic inhibition was not observed with the comparator first-in-class topoisomerase I-targeting TROP2 ADCs. Instead, these ADCs exhibited antagonism when paired with adagrasib. These results suggest that AKTX-101 synergy with KRAS inhibitor may be linked to the novel biology of PH1 targeting RNA splicing. The synergy was explained by PH1’s unique ability target pre-mRNA transcripts for degradation, including those bearing KRAS mutations driving these cancer models.

"KRAS has long been considered one of oncology’s most important but difficult targets, and while recent KRAS inhibitors have represented meaningful progress, there remains a significant opportunity to further enhance activity and broaden therapeutic impact," said Abizer Gaslightwala, President and Chief Executive Officer of Akari Therapeutics. "These data suggest that our PH1 RNA splicing modulator payload may offer a fundamentally differentiated mechanism capable of enhancing KRAS inhibitor activity in ways not observed with conventional ADC payloads. We believe this represents an exciting opportunity not only for AKTX-101, but potentially for RNA splicing modulation as a new therapeutic strategy across KRAS-driven tumors."

Dr. Satyajit Mitra, Executive Director and Head of Oncology, stated, "Historically, KRAS mutations have been incredibly difficult to drug, and one may need to approach this problem with drugs of different modalities and mechanisms of action. AKTX 101 synergy with an approved drug, like adagrasib, in an unapproved KRAS setting offers exciting combination possibilities. The PH1 spliceosome modulator payload ADC has the potential to unlock efficacy of cancer therapeutics in cancers with oncogene dependency. We have previously demonstrated combination with AR-v7 oncogenes with enzalutamide and now KRAS oncogenes with adagrasib."

The data featured in the ASCO (Free ASCO Whitepaper) 2026 online abstract build upon Akari’s previously reported AACR (Free AACR Whitepaper) 2026 findings demonstrating differentiated cytotoxicity and superior potency of AKTX-101 versus current TROP2 ADCs utilizing Topoisomerase I inhibitor payloads across multiple tumor models, including bladder, lung (including KRAS G12V mutated NSCLC) and breast cancers.

Akari’s lead program, AKTX-101, is a TROP2-targeting ADC powered by the Company’s proprietary PH1 RNA spliceosome-modulating payload. Unlike traditional ADC payloads that primarily rely on microtubule or DNA-damaging mechanisms, PH1 is designed to disrupt RNA splicing within cancer cells while also activating innate and adaptive immune responses.

Akari has initiated IND-enabling studies for AKTX-101 and is targeting initiation of a Phase 1 first-in-human clinical trial by mid-2027.

For more information about the ASCO (Free ASCO Whitepaper) Annual Meeting 2026, please visit www.asco.org.

(Press release, Akari Therapeutics, MAY 21, 2026, View Source [SID1234665940])

Zentalis Pharmaceuticals to Present Phase 1b MUIR Trial Data Showing Encouraging Clinical Activity and Manageable Safety Profile of Azenosertib Plus Paclitaxel in Platinum-Resistant Ovarian Cancer at ASCO 2026

On May 21, 2026 Zentalis Pharmaceuticals, Inc. (Nasdaq: ZNTL), a clinical oncology innovator advancing late-stage development of investigational first-in-class WEE1 inhibitor azenosertib as a biomarker-driven treatment approach for ovarian cancer, reported that data from Part 1 of the Phase 1b MUIR trial will be presented at the 2026 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, being held May 29 – June 2, 2026, in Chicago, Illinois. The poster, titled "Azenosertib Plus Paclitaxel for Platinum-Resistant Ovarian Cancer: Results From a Phase 1b Study," will be presented in the Gynecologic Cancer Poster Session (Abstract #5529, Poster Board #195) on June 1, 2026, from 9:00am–12:00pm CDT.

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"Paclitaxel is one of the most widely used chemotherapy agents across tumor types, including in ovarian cancer, and these Phase 1b data show encouraging clinical activity and tolerability of adding azenosertib to paclitaxel in an all-comer platinum-resistant ovarian cancer (PROC) setting," said Ingmar Bruns, M.D., Chief Medical Officer of Zentalis Pharmaceuticals. "Establishing the safety, combinability, and efficacy signal of azenosertib with paclitaxel is an important proof of concept — one that we believe speaks to the broad potential of azenosertib across multiple lines of ovarian cancer and other tumor types where taxanes are commonly used. With our core strategic focus on advancing azenosertib in registration-intended trials as a monotherapy in the Cyclin E1-positive PROC population, the MUIR trial represents an important and complementary part of our broader indication expansion strategy."

"In a heavily pre-treated, all-comer patient PROC population where all patients had received prior paclitaxel, we observed meaningful tumor reductions and durable responses with this azenosertib paclitaxel combination, with an overall response rate of 39% and a median PFS of 7.3 months" said Joyce F. Liu, M.D., MPH, Associate Professor at Dana-Farber Cancer Institute and a study investigator. "At the 250 mg 5:2 intermittent dose — the dose thought to offer the optimal therapeutic index — half of patients achieved a response with a median duration of response of 9.2 months. Interestingly, the clinical activity appears similar in both Cyclin E1-positive and Cyclin E1-negative tumors, suggesting that Cyclin E1 status may not be as important in the azenosertib combination setting where the combination agent is inducing replication stress. These results support continued evaluation of azenosertib-taxane combinations in broader ovarian cancer settings and other tumor types where taxanes are part of the standard of care."

MUIR is a multi-part, open-label Phase 1b clinical trial evaluating azenosertib in combination with chemotherapy in patients with ovarian cancer. Part 1 evaluated azenosertib in combination with four chemotherapy regimens in patients with PROC, with data from the paclitaxel arm presented at ASCO (Free ASCO Whitepaper) as paclitaxel is commonly used across multiple tumor types, including ovarian cancer. Data from the other combination arms will be presented separately at a later date. The findings reflect a December 1, 2025 data cutoff and include 46 patients who received azenosertib across four dose cohorts — 200 mg QD continuously or 200 mg, 250 mg, or 300 mg QD intermittently (5 days on, 2 days off) — in combination with paclitaxel 80 mg/m². All patients had received prior paclitaxel.

Encouraging Activity in All-Comer PROC Population with Activity Across 4 Dose Groups (n=46)
•Overall Response Rate (ORR): 39.1% (95% CI: 25.1–54.6)
•Clinical Benefit Rate (CBR): 58.7% (95% CI: 42.2–73.0)
•Median Duration of Response (DOR): 5.6 months (95% CI: 5.6–9.2)
•Median Progression-Free Survival (PFS): 7.3 months (95% CI: 3.7–7.5)
These results are encouraging in the context of the historical efficacy of paclitaxel monotherapy in PROC, with an ORR of approximately 30% and a median PFS of approximately 4 months.
Clinical activity was broadly comparable in Cyclin E1-positive patients (ORR: 41.4% [95% CI: 23.5-61.1]; median PFS: 7.3 months [95% CI: 3.7-9.1]) and Cyclin E1-negative patients (ORR: 35.7% [95% CI: 12.8-64.9]; median PFS: 5.4 months [95% CI: 1.7-NE]), suggesting that Cyclin E1-positive biomarker status may not be required to derive benefit when azenosertib is combined with a cytotoxic agent.
At the 250 mg intermittent (5:2) dose cohort (n=12), which demonstrated the potential optimal therapeutic index:
•ORR: 50.0% (95% CI: 21.1–78.9), including one complete response
•CBR: 66.7% (95% CI: 34.9-90.1)
•Median DOR: 9.2 months (95% CI: 3.8–NE)
•Median PFS: 5.5 months (95% CI: 1.7–12.9)

Manageable Safety Profile with Low Rate of High-Grade Events Across 4 Dose Cohorts (n=46)
•Most common all-grade treatment-related adverse events (TRAEs): fatigue (60.9%), anemia (58.7%), nausea (52.2%), and neutropenia (50.0%).
•Most frequent Grade ≥3 TRAEs: neutropenia (30.4%) and anemia (19.6%); rates of high-grade fatigue and nausea were less than 10%.
•Serious TRAEs occurred in approximately 20% of patients; the most frequent were fatigue, diarrhea, and neutropenia, each occurred in 2 patients.
•Of 15 patients (32.6%) who discontinued due to adverse events, approximately half discontinued paclitaxel only and were able to continue on azenosertib monotherapy until disease progression
•One G5 event due to sepsis was assessed as related to azenosertib by the investigator (previously reported in June 2024). While the role of azenosertib cannot be excluded, the event may have been attributable to the patient’s advanced disease, given the absence of neutropenia and negative blood cultures at the time of the event.
The poster will be available at View Source after ASCO (Free ASCO Whitepaper).
About MUIR Clinical Trial
MUIR (ZN-c3-002) is a multi-part, open-label Phase 1b clinical trial (NCT04516447) evaluating the safety, efficacy, and preliminary clinical activity of azenosertib in combination in patients with ovarian cancer.

Part 1 enrolled patients with platinum-resistant ovarian cancer (PROC) treated with azenosertib in combination with one of four chemotherapy regimens: carboplatin, gemcitabine, pegylated liposomal doxorubicin, or paclitaxel. Primary objectives are safety and tolerability, with key secondary objectives including clinical activity assessed by objective response rate, duration of response, and progression-free survival per RECIST v1.1.

Part 2 is evaluating azenosertib plus bevacizumab as maintenance regimen (first [1L] or second line [2L]) in patients with advanced ovarian, peritoneal, or fallopian tube cancer following platinum-based chemotherapy. The dose expansion portion will evaluate azenosertib at the recommended dose in combination with bevacizumab in patients with platinum-sensitive ovarian cancer in 2L who progressed while on a PARP inhibitor for 1L maintenance. The primary objective is safety and tolerability; secondary objectives include preliminary clinical activity of the combination as assessed by progression-free survival for the dose expansion portion.

About Azenosertib
Azenosertib is an investigational, potentially first-in-class, selective, and orally bioavailable inhibitor of WEE1 currently being evaluated in clinical studies in ovarian cancer and additional tumor types. WEE1 acts as a master regulator of the G1-S and G2-M cell cycle checkpoints, through negative regulation of both CDK1 and CDK2, to prevent replication of cells with damaged DNA. By inhibiting WEE1, azenosertib enables cell cycle progression, despite high levels of DNA damage, thereby resulting in the accumulation of DNA damage and leading to mitotic catastrophe and cancer cell death.

Azenosertib is in late-stage development as a potential treatment for Cyclin E1-positive platinum-resistant ovarian cancer (PROC). There is currently no approved treatment option specifically for this biomarker-selected population which comprises approximately 50% of PROC patients. Cyclin E1 protein overexpression has been established as a sensitive and specific predictive biomarker for identifying patients who could potentially derive benefit from azenosertib treatment, based on retrospective analysis of azenosertib studies in PROC. Validation of the Cyclin E1 companion diagnostic assay is ongoing in the DENALI and ASPENOVA trials.

Azenosertib has been granted Fast Track Designation by the U.S. FDA for the treatment of patients with Cyclin E1-positive platinum-resistant ovarian cancer. Fast Track Designation is intended to facilitate the development and expedite the review of therapies that have the potential to treat serious conditions and address unmet medical needs.

(Press release, Zentalis Pharmaceuticals, MAY 21, 2026, View Source [SID1234665939])

OPTIMA Trial Results to Be Presented at ASCO Provide New Evidence Supporting Prosigna-Guided Chemotherapy Decisions in Breast Cancer

On May 21, 2026 Veracyte, Inc. (NASDAQ: VCYT), a leading cancer diagnostics company, reported that data from two significant phase III clinical trials using its Prosigna Breast and Decipher Prostate tests will be presented at the 2026 ASCO (Free ASCO Whitepaper) Annual Meeting in Chicago, taking place May 29 – June 2. The OPTIMA and ENZAMET trial presentations are expected to provide practice-changing evidence demonstrating how Veracyte’s genomic tests can guide treatment decisions in both early-stage breast cancer and metastatic prostate cancer.

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The OPTIMA study is a prospective, randomized trial led by University College London (UCL) and supported by the National Institute for Health Research (NIHR). The study enrolled more than 4,400 patients and was designed to address a key clinical question: which patients would benefit from chemotherapy, and which may be able to safely avoid it, and its long-term toxicities. The study results demonstrating how the Prosigna test can guide adjuvant chemotherapy decisions in patients with high-risk breast cancer will be presented by Dr. Rob Stein of UCL and OPTIMA Trial Chief Investigator, on Saturday, May 30 during the breast cancer session.

"The OPTIMA trial results represent a major milestone in precision breast oncology and will provide Level 1A evidence supporting Prosigna-guided treatment decisions," said Kelly Marcom, M.D., Veracyte’s medical director, Breast Cancer. "These findings have the potential to transform how clinicians treat a large population of patients with breast cancer, helping them to personalize their patient’s treatment choices using the genomic insights that the Prosigna test provides."

The ENZAMET trial is an international, prospective, randomized study conducted by the Australian and New Zealand Urogenital and Prostate Cancer Trials Group (ANZUP). Dr. Christopher Sweeney of South Australian Immunogenomics Cancer Institute, Adelaide University, will present data on Saturday, May 30 during the Genitourinary Cancer session on Decipher Prostate’s ability to predict treatment benefit with the chemotherapy docetaxel in metastatic, hormone-sensitive prostate cancer. The independent analysis evaluated how the Decipher test can help identify which patients are most likely to benefit from treatment intensification with triplet therapy (docetaxel to standard of cancer hormonal therapy).

"Together, these studies provide practice-changing evidence supporting the use of Veracyte’s tests in guiding treatment decisions across cancer types," said Phillip Febbo, M.D., Veracyte’s chief scientific and medical officer. "In practice, this means clinicians can more confidently match treatment intensity to individual patient risk, helping ensure the right level of care while avoiding unnecessary treatment and its side effects, for both breast and prostate cancer patients."

Additional research to be presented at ASCO (Free ASCO Whitepaper) supports the company’s mission to transform cancer care for patients all over the world.

Oral Presentations at ASCO (Free ASCO Whitepaper) 2026 Annual Meeting

Title: First results from the OPTIMA phase III randomized non-inferiority trial of test-directed chemotherapy in patients with high clinical risk ER-positive HER2-negative early breast cancer
Presenter: Robert Stein, PhD, MBBChir, FRC, National Institute for Health Research University College London Hospitals Biomedical Research Centre
Session Title: Breast Cancer—Local/Regional/Adjuvant
Date/Time: Saturday, May 30, 2026, 1:15 PM CT
Location: Hall B1
Title: Clinico-Transcriptomic Risk Stratification to Guide Abiraterone Treatment Intensification in High-Risk Prostate Cancer: A Combined Analysis of NRG/RTOG 9202, 9413, 9902, and 0521
Presenter: Krishnan R Patel, MD, MHS, Radiation Oncology Branch, National Cancer Institute, NIH
Session Title: Genitourinary Cancer—Prostate, Testicular, and Penile
Date/Time: Saturday, May 30, 2026, 3:00 PM CT
Location: Hall D1
Title: Assessment of the ability of Decipher Prostate Genomic Classifier (DGC) >0.85 to identify patients who benefit from adding docetaxel (DOC) to androgen deprivation therapy (ADT) plus enzalutamide (ENZ): Level 1B evidence from the ENZAMET study
Presenter: Christopher Sweeney, MBBS, South Australian Immunogenomics Cancer Institute, Adelaide University
Session Title: Genitourinary Cancer—Prostate, Testicular, and Penile
Date/Time: Saturday, May 30, 2026, 3:12 PM CT
Location: Hall D1
Title: Genomic classifier–driven NCCN risk reclassification to track distinct transcriptomic signatures in early prostate cancer
Presenter: Daniel Keizman, MD, Tel Aviv Sourasky Medical Center, Israel
Session Title: Genitourinary Cancer—Prostate, Testicular, and Penile
Date/Time: Sunday, May 31, 2026, 4:36 PM CT
Location: Hall D1
Additional Posters at ASCO (Free ASCO Whitepaper)

Abstract #1660 | Poster bd #578: Titled: Practice patterns and outcomes by genomic risk in octogenarians with high-risk localized prostate cancer: a national real-world data analysis. Presenter: James Janopaul-Naylor – Saturday May 30, 2026 at 9:00 AM CDT, Hall A
Abstract #4619 | Poster bd #98: Titled: Neoadjuvant sacituzumab govitecan in patients with muscle-invasive bladder cancer: Final results and biomarker analyses of the SURE-01 trial. Presenter: Brigida A. Maiorano – Sunday, May 31, 2026 at 9:00 AM CDT, Hall A
Abstract #5114 | Poster bd #209: Titled: Assessing the Clinical and Biological Associations Between Artera Multimodal Artificial Intelligence (MMAI) and Decipher Genomic Classifier (GC) in Localized Prostate Cancer (PCa). Presenter: Boon Hao Hong – Sunday, May 31, 2026 at 9:00 AM CDT, Hall A
Abstract #4617 | Poster bd #96: Titled: Molecular characterization of residual disease post-neoadjuvant sacituzumab govitecan (SG), pembrolizumab, or their combination in patients with muscle-invasive bladder cancer (MIBC). Presenter: Andrea Necchi – Sunday, May 31, 2026 at 9:00 AM CDT, Hall A
For more information, stop by Veracyte’s booth #13069 at the ASCO (Free ASCO Whitepaper) Annual Meeting or visit the company’s website here.

(Press release, Veracyte, MAY 21, 2026, View Source [SID1234665938])

Rigel Announces Oral and Poster Presentations at the 2026 ASCO Annual Meeting and EHA2026 Congress

On May 21, 2026 Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL), a commercial stage biotechnology company focused on hematologic disorders and cancer, reported the final data from the Phase 3 AcceleRET-Lung clinical trial of GAVRETO (pralsetinib) as first-line treatment of rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC) will be presented in an oral session at the 2026 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting on Friday, May 29, 2026.

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In addition, the ASCO (Free ASCO Whitepaper) Annual Meeting and European Hematology Association (EHA) (Free EHA Whitepaper) 2026 Congress will feature poster presentations that include additional data for pralsetinib and data for REZLIDHIA (olutasidenib) for the treatment of relapsed or refractory (R/R) isocitrate dehydrogenase-1 (mIDH1)-mutated acute myeloid leukemia (AML). The ASCO (Free ASCO Whitepaper) Annual Meeting is being held in Chicago, Illinois and virtually from May 29 to June 2, 2026 and the EHA (Free EHA Whitepaper)2026 Congress is being held in Stockholm, Sweden and virtually from June 11 to June 14, 2026.

"Having an oral presentation at ASCO (Free ASCO Whitepaper) as well as multiple additional data presentations at both upcoming medical conferences underscores the continued clinical relevance of our oncology portfolio. We’re pleased that data from the AcceleRET-Lung clinical study will be presented for the first time and the real-world outcome data for olutasidenib will be presented for patients with relapsed/refractory mIDH1 AML previously treated with venetoclax, further validating its role as a treatment option for these patients," said Raul Rodriguez, Rigel’s president and CEO. "Together, these data reinforce our ability to deliver targeted therapies that significantly improve the lives of patients with difficult-to-treat cancers. We look forward to sharing these data with the medical community."

Key highlights from the presentations at ASCO (Free ASCO Whitepaper) and EHA (Free EHA Whitepaper) include:

In the Phase 3 AcceleRET clinical trial, pralsetinib met the primary progression-free survival (PFS) endpoint and had a significantly greater overall response rate and more durable response vs. standard of care, demonstrating the clinical utility of pralsetinib in RET fusion-positive NSCLC. In terms of safety, there were 32 (30.0%) and 26 (25.0%) deaths in the pralsetinib and standard of care groups, respectively, with 8 (7.4%) and 0 due to infection. Increased monitoring suggests severe infection risk can be effectively managed.
In the Phase 1/2 ARROW clinical study of pralsetinib in patients with RET altered thyroid cancer and medullary thyroid cancer, pralsetinib yielded clinically meaningful and durable responses with a manageable safety profile, consistent with prior reports.
In an analysis of the patient, disease and molecular characteristics of long-term (LT) complete remission (CR)/CR with partial hematologic recovery (CRh) (>12 months duration of response) in the registrational Phase 2 clinical trial evaluating olutasidenib in patients with R/R mIDH1 AML, olutasidenib enabled LT CR/CRh in half of patients with CR/CRh without transplant (longest response >54 months). Estimated 48-month overall survival was 74% (95% CI: 51%, 88%).
In a separate real-world assessment evaluating treatment patterns, safety and effectiveness of adults with R/R mIDH1 AML receiving olutasidenib following a venetoclax-based therapy in routine practice (73% of patients received olutasidenib as second-line therapy), a subgroup of patients that historically has very poor outcomes, 51 charts were collected from 18 physicians. Olutasidenib demonstrated robust real-world effectiveness with a CR/CRh rate of 60.8% and CR rate of 57% and a median response duration of 30.3 months. Substantial reductions in transfusion dependence further support olutasidenib as a viable post-venetoclax therapeutic option. These findings suggest that earlier sequencing of olutasidenib in the treatment paradigm may optimize patient outcomes.
ASCO Annual Meeting abstracts may be accessed online via View Source EHA (Free EHA Whitepaper)2026 Congress abstracts may be accessed online via the EHA (Free EHA Whitepaper) Library.

ASCO Presentations

Abstract Title

Lead Author / Presenter

Presentation Type / Abstract #

Session Title

Session Date / Time (CT)

GAVRETO (pralsetinib)

Efficacy and safety of pralsetinib as first-line treatment of RET fusion-positive advanced or metastatic non-small cell lung cancer (NSCLC): The phase 3 AcceleRET-Lung study

Sanjay Popat, MBBS, FRCP, PhD

Oral presentation #8504

Lung Cancer—Non-Small Cell Metastatic

Friday, May 29, 2026 1:00pm – 4:00pm

Efficacy and safety of pralsetinib in advanced or metastatic RET-altered thyroid cancer (TC): Final analysis of the phase 1/2 ARROW study

Vivek Subbiah, MD

Poster presentation #6028

Head and Neck Cancer

Saturday, May 30, 2026

1:30pm – 4:30pm

Efficacy and Safety of Pralsetinib in RET Fusion-Positive Solid Tumors: Data From the TAPISTRY Trial

Chia-Chi Lin, MD, PhD

Poster presentation #3144

Developmental Therapeutics—Molecularly Targeted Agents and Tumor Biology

Saturday, May 30, 2026

1:30pm – 4:30pm

Clinical factors driving use of RET inhibitor pralsetinib and associated real-world outcomes in RET fusion–positive NSCLC: A retrospective chart review

Makenzi Evangelist, MD

Publication #e20731

Lung Cancer—Non-Small Cell Metastatic

REZLIDHIA (olutasidenib)

Acute myeloid leukemia (AML) patient, disease, and molecular characteristics associated with a long-term (LT) response to olutasidenib

Justin M. Watts, MD

Poster presentation #6523

Hematologic Malignancies—Leukemia, Myelodysplastic Syndromes, and Allotransplant

Monday, June 1, 2026

9:00am – 12:00pm

Real-World Treatment Patterns and Outcomes with Olutasidenib After Venetoclax in IDH1-Mutated AML Using EHR Data

Yasmin Abaza, MD

Publication #e18514

Hematologic Malignancies—Leukemia, Myelodysplastic Syndromes, and Allotransplant

Treatment Patterns and Outcomes with Olutasidenib After Venetoclax in IDH1-Mutated AML Using Real-World Data from Chart Review

Pinkal Desai, MD, MPH

Publication #e18527

Hematologic Malignancies—Leukemia, Myelodysplastic Syndromes, and Allotransplant

Evaluating the cost per month of response for olutasidenib (OLU) versus ivosidenib (IVO) for patients with relapsed/refractory (R/R) mutant IDH1 (mIDH1) acute myeloid leukaemia (AML)

Yasmin Abaza, MD

Publication #e18504

Hematologic Malignancies—Leukemia, Myelodysplastic Syndromes, and Allotransplant

EHA Presentations

Abstract Title

Lead Author / Presenter

Presentation Type / Abstract #

Session Date / Time (CEST)

REZLIDHIA (olutasidenib)

Treatment Patterns and Outcomes with Olutasidenib After Venetoclax in IDH1-Mutated AML Using Real-World Data from Chart Review

Pinkal Desai, MD, MPH

Poster presentation #PS1502

Saturday, June 13, 2026 (18:45 – 19:45 CEST)

Acute myeloid leukemia (AML) patient, disease, and molecular characteristics associated with a long-term (LT) response to olutasidenib

Stéphane de Botton, MD, PhD

Poster presentation #PS1625

Saturday, June 13, 2026 (18:45 – 19:45 CEST)

Delphi Consensus on Optimal Treatment Strategies Using IDH1 Inhibitors in Patients with R/R mIDH1 AML

Justin M. Watts, MD

Publication #PB2795

Real-World Treatment Patterns and Outcomes with Olutasidenib After Venetoclax in IDH1-Mutated AML Using EHR Data

Yasmin Abaza, MD

Publication #PB2610

TAVALISSE (fostamatinib disodium hexahydrate)

Health-Related Quality of Life (HRQoL) Among Patients With Immune Thrombocytopenia (ITP) Treated With Fostamatinib in the FORTE Study

Amber Afzal, MD, MSCI

Publication #PB4295

About NSCLC
It is estimated that over 229,000 adults in the U.S. will be diagnosed with lung cancer in 2026. Lung cancer is the leading cause of cancer death in the U.S., with non-small cell lung cancer (NSCLC) being the most common type accounting for 77% of all lung cancer diagnoses.1 RET fusions are implicated in approximately 1-2% of patients with NSCLC.2

About AML
Acute myeloid leukemia (AML) is a rapidly progressing cancer of the blood and bone marrow that affects myeloid cells, which normally develop into various types of mature blood cells. AML occurs primarily in adults and accounts for about 1 percent of all adult cancers. The American Cancer Society estimates that there will be about 22,720 new cases in the United States, most in adults, in 2026.3

Relapsed AML affects about half of all patients who, following treatment and remission, experience a return of leukemia cells in the bone marrow.4,5 Refractory AML, which affects between 10 and 40 percent of newly diagnosed patients, occurs when a patient fails to achieve remission even after intensive treatment.6 Quality of life declines for patients with each successive line of treatment for AML, and well-tolerated treatments in relapsed or refractory disease remain an unmet need.

About ITP
In patients with immune thrombocytopenia (ITP), the immune system attacks and destroys the body’s own blood platelets, which play an active role in blood clotting and healing. Common symptoms of ITP are excessive bruising and bleeding. Patients suffering with chronic ITP may live with an increased risk of severe bleeding events that can result in serious medical complications or even death. Current therapies for ITP include steroids, blood platelet production boosters (TPO-RAs), and splenectomy. However, not all patients respond to existing therapies. As a result, there remains a significant medical need for additional treatment options for patients with ITP.

About GAVRETO (pralsetinib)

INDICATIONS

GAVRETO (pralsetinib) is indicated for the treatment of:

Adult patients with metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA-approved test
Adult and pediatric patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate)*
*This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).

IMPORTANT SAFETY INFORMATION

WARNING: SERIOUS INFECTIONS, INCLUDING OPPPORTUNISTIC INFECTIONS

GAVRETO may increase the risk for serious infections, including bacterial, fungal, viral and opportunistic infections, which can lead to hospitalization or death. Withhold, reduce the dose or permanently discontinue GAVRETO based on severity.

WARNINGS AND PRECAUTIONS

Serious Infections, Including Opportunistic Infections: GAVRETO may increase the risk for serious infections, including fatal and opportunistic infections. In the AcceleRET-Lung trial, infections occurred in 72% of patients who received GAVRETO, including 18% with Grade 3 and 3.7% with Grade 4 and 7% with fatal outcomes. Among the patients who received chemotherapy/immunotherapy, infections occurred in 52%, including 10% with Grade 3. Infections in the GAVRETO arm included pneumonia, urinary tract infection, opportunistic infections (such as pneumocystis jirovecii pneumonia and fungal infections) and others. Monitor patients for signs and symptoms of infection and treat appropriately. Withhold, reduce the dose, or permanently discontinue GAVRETO based on severity.
Interstitial Lung Disease (ILD)/Pneumonitis: Severe, life-threatening, and fatal ILD/pneumonitis can occur in patients treated with GAVRETO. Pneumonitis occurred in 12% of patients who received GAVRETO, including 3.3% with Grade 3-4, and 0.2% with fatal reactions. Monitor for pulmonary symptoms indicative of ILD/pneumonitis. Withhold GAVRETO and promptly investigate for ILD in any patient who presents with acute or worsening of respiratory symptoms (e.g., dyspnea, cough, and fever). Withhold, reduce dose or permanently discontinue GAVRETO based on severity of confirmed ILD.
Hypertension: Occurred in 35% of patients, including Grade 3 hypertension in 18% of patients. Overall, 8% had their dose interrupted and 4.8% had their dose reduced for hypertension. Treatment-emergent hypertension was most commonly managed with anti-hypertension medications. Do not initiate GAVRETO in patients with uncontrolled hypertension. Optimize blood pressure prior to initiating GAVRETO. Monitor blood pressure after 1 week, at least monthly thereafter and as clinically indicated. Initiate or adjust anti-hypertensive therapy as appropriate. Withhold, reduce dose, or permanently discontinue GAVRETO based on the severity.
Hepatotoxicity: Serious hepatic adverse reactions occurred in 1.5% of patients treated with GAVRETO. Increased aspartate aminotransferase (AST) occurred in 49% of patients, including Grade 3 or 4 in 7% and increased alanine aminotransferase (ALT) occurred in 37% of patients, including Grade 3 or 4 in 4.8%. The median time to first onset for increased AST was 15 days (range: 5 days to 2.5 years) and increased ALT was 24 days (range: 7 days to 3.7 years). Monitor AST and ALT prior to initiating GAVRETO, every 2 weeks during the first 3 months, then monthly thereafter and as clinically indicated. Withhold, reduce dose or permanently discontinue GAVRETO based on severity.
Hemorrhagic Events: Serious, including fatal, hemorrhagic events can occur with GAVRETO. Grade ≥3 events occurred in 4.1% of patients treated with GAVRETO including one patient with a fatal hemorrhagic event. Permanently discontinue GAVRETO in patients with severe or life-threatening hemorrhage.
Tumor Lysis Syndrome (TLS): Cases of TLS have been reported in patients with medullary thyroid carcinoma receiving GAVRETO. Patients may be at risk of TLS if they have rapidly growing tumors, a high tumor burden, renal dysfunction, or dehydration. Closely monitor patients at risk, consider appropriate prophylaxis including hydration, and treat as clinically indicated.
Risk of Impaired Wound Healing: Impaired wound healing can occur in patients who receive drugs that inhibit the vascular endothelial growth factor (VEGF) signaling pathway. Therefore, GAVRETO has the potential to adversely affect wound healing. Withhold GAVRETO for at least 5 days prior to elective surgery. Do not administer for at least 2 weeks following major surgery and until adequate wound healing. The safety of resumption of GAVRETO after resolution of wound healing complications has not been established.
Embryo-Fetal Toxicity: Based on findings from animal studies and its mechanism of action, GAVRETO can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective non-hormonal contraception during treatment with GAVRETO and for 2 weeks after the last dose. Advise males with female partners of reproductive potential to use effective contraception during treatment with GAVRETO and for 1 week after the last dose.
ADVERSE REACTIONS

Common adverse reactions (≥25%) were musculoskeletal pain, constipation, hypertension, diarrhea, fatigue, edema, pyrexia, and cough. Common Grade 3/4 laboratory abnormalities (≥2%) were decreased lymphocytes, decreased neutrophils, decreased hemoglobin, decreased phosphate, decreased leukocytes, decreased sodium, increased aspartate aminotransferase (AST), increased alanine aminotransferase (ALT), decreased calcium (corrected), decreased platelets, increased alkaline phosphatase, increased potassium, decreased potassium, and increased bilirubin.
DRUG INTERACTIONS

Avoid coadministration of GAVRETO with strong or moderate CYP3A inhibitors, P-gp inhibitors, or combined P-gp and strong or moderate CYP3A inhibitors. If coadministration cannot be avoided, reduce the GAVRETO dose. Avoid coadministration of GAVRETO with strong or moderate CYP3A inducers. If coadministration cannot be avoided, increase the GAVRETO dose.
Lactation: Advise women not to breastfeed during treatment with GAVRETO and for 1 week after the last dose.

Pediatric Use: Monitor open growth plates in adolescent patients. Consider interrupting or discontinuing GAVRETO if abnormalities occur.

Click here for Important Safety Information and Full Prescribing Information, including Boxed WARNING.

About REZLIDHIA

INDICATION
REZLIDHIA is indicated for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test.

IMPORTANT SAFETY INFORMATION

WARNING: DIFFERENTIATION SYNDROME

Differentiation syndrome, which can be fatal, can occur with REZLIDHIA treatment. Symptoms may include dyspnea, pulmonary infiltrates/pleuropericardial effusion, kidney injury, hypotension, fever, and weight gain. If differentiation syndrome is suspected, withhold REZLIDHIA and initiate treatment with corticosteroids and hemodynamic monitoring until symptom resolution.

WARNINGS AND PRECAUTIONS

Differentiation Syndrome
REZLIDHIA can cause differentiation syndrome. In the clinical trial of REZLIDHIA in patients with relapsed or refractory AML, differentiation syndrome occurred in 16% of patients, with grade 3 or 4 differentiation syndrome occurring in 8% of patients treated, and fatalities in 1% of patients. Differentiation syndrome is associated with rapid proliferation and differentiation of myeloid cells and may be life-threatening or fatal. Symptoms of differentiation syndrome in patients treated with REZLIDHIA included leukocytosis, dyspnea, pulmonary infiltrates/pleuropericardial effusion, kidney injury, fever, edema, pyrexia, and weight gain. Of the 25 patients who experienced differentiation syndrome, 19 (76%) recovered after treatment or after dose interruption of REZLIDHIA. Differentiation syndrome occurred as early as 1 day and up to 18 months after REZLIDHIA initiation and has been observed with or without concomitant leukocytosis.

If differentiation syndrome is suspected, temporarily withhold REZLIDHIA and initiate systemic corticosteroids (e.g., dexamethasone 10 mg IV every 12 hours) for a minimum of 3 days and until resolution of signs and symptoms. If concomitant leukocytosis is observed, initiate treatment with hydroxyurea, as clinically indicated. Taper corticosteroids and hydroxyurea after resolution of symptoms. Differentiation syndrome may recur with premature discontinuation of corticosteroids and/or hydroxyurea treatment. Institute supportive measures and hemodynamic monitoring until improvement; withhold dose of REZLIDHIA and consider dose reduction based on recurrence.

Hepatotoxicity
REZLIDHIA can cause hepatotoxicity, presenting as increased alanine aminotransferase (ALT), increased aspartate aminotransferase (AST), increased blood alkaline phosphatase, and/or elevated bilirubin. Of 153 patients with relapsed or refractory AML who received REZLIDHIA, hepatotoxicity occurred in 23% of patients; 13% experienced grade 3 or 4 hepatotoxicity. One patient treated with REZLIDHIA in combination with azacitidine in the clinical trial, a combination for which REZLIDHIA is not indicated, died from complications of drug-induced liver injury. The median time to onset of hepatotoxicity in patients with relapsed or refractory AML treated with REZLIDHIA was 1.2 months (range: 1 day to 17.5 months) after REZLIDHIA initiation, and the median time to resolution was 12 days (range: 1 day to 17 months). The most common hepatotoxicities were elevations of ALT, AST, blood alkaline phosphatase, and blood bilirubin.

Monitor patients frequently for clinical symptoms of hepatic dysfunction such as fatigue, anorexia, right upper abdominal discomfort, dark urine, or jaundice. Obtain baseline liver function tests prior to initiation of REZLIDHIA, at least once weekly for the first two months, once every other week for the third month, once in the fourth month, and once every other month for the duration of therapy. If hepatic dysfunction occurs, withhold, reduce, or permanently discontinue REZLIDHIA based on recurrence/severity.

ADVERSE REACTIONS
The most common (≥20%) adverse reactions, including laboratory abnormalities, were aspartate aminotransferase increased, alanine aminotransferase increased, potassium decreased, sodium decreased, alkaline phosphatase increased, nausea, creatinine increased, fatigue/malaise, arthralgia, constipation, lymphocytes increased, bilirubin increased, leukocytosis, uric acid increased, dyspnea, pyrexia, rash, lipase increased, mucositis, diarrhea and transaminitis.

DRUG INTERACTIONS

Avoid concomitant use of REZLIDHIA with strong or moderate CYP3A inducers.
Avoid concomitant use of REZLIDHIA with sensitive CYP3A substrates unless otherwise instructed in the substrates prescribing information. If concomitant use is unavoidable, monitor patients for loss of therapeutic effect of these drugs.
LACTATION
Advise women not to breastfeed during treatment with REZLIDHIA and for 2 weeks after the last dose.

GERIATRIC USE
No overall differences in effectiveness were observed between patients 65 years and older and younger patients. Compared to patients younger than 65 years of age, an increase in incidence of hepatotoxicity and hypertension was observed in patients ≥65 years of age.

HEPATIC IMPAIRMENT
In patients with mild or moderate hepatic impairment, closely monitor for increased probability of differentiation syndrome.

Click here for Important Safety Information and Full Prescribing Information, including Boxed WARNING.

About TAVALISSE
Indication
TAVALISSE (fostamatinib disodium hexahydrate) tablets is indicated for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment.

Important Safety Information

Warnings and Precautions

Hypertension can occur with TAVALISSE treatment. Patients with pre-existing hypertension may be more susceptible to the hypertensive effects. Monitor blood pressure every 2 weeks until stable, then monthly, and adjust or initiate antihypertensive therapy for blood pressure control maintenance during therapy. If increased blood pressure persists, TAVALISSE interruption, reduction, or discontinuation may be required.
Elevated liver function tests (LFTs), mainly ALT and AST, can occur with TAVALISSE. Monitor LFTs monthly during treatment. If ALT or AST increase to ≥3 x upper limit of normal, manage hepatotoxicity using TAVALISSE interruption, reduction, or discontinuation.
Diarrhea occurred in 31% of patients and severe diarrhea occurred in 1% of patients treated with TAVALISSE. Monitor patients for the development of diarrhea and manage using supportive care measures early after the onset of symptoms. If diarrhea becomes severe (≥Grade 3), interrupt, reduce dose or discontinue TAVALISSE.
Neutropenia occurred in 6% of patients treated with TAVALISSE; febrile neutropenia occurred in 1% of patients. Monitor the ANC monthly and for infection during treatment. Manage toxicity with TAVALISSE interruption, reduction, or discontinuation.
TAVALISSE can cause fetal harm when administered to pregnant women. Advise pregnant women the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment and for at least 1 month after the last dose. Verify pregnancy status prior to initiating TAVALISSE. It is unknown if TAVALISSE or its metabolite is present in human milk. Because of the potential for serious adverse reactions in a breastfed child, advise a lactating woman not to breastfeed during TAVALISSE treatment and for at least 1 month after the last dose.
Drug Interactions

Concomitant use of TAVALISSE with strong CYP3A4 inhibitors increases exposure to the major active metabolite of TAVALISSE (R406), which may increase the risk of adverse reactions. Monitor for toxicities that may require a reduction in TAVALISSE dose.
It is not recommended to use TAVALISSE with strong CYP3A4 inducers, as concomitant use reduces exposure to R406.
Concomitant use of TAVALISSE may increase concentrations of some CYP3A4 substrate drugs and may require a dose reduction of the CYP3A4 substrate drug.
Concomitant use of TAVALISSE may increase concentrations of BCRP substrate drugs (eg, rosuvastatin) and P-Glycoprotein (P-gp) substrate drugs (eg, digoxin), which may require a dose reduction of the BCRP and P-gp substrate drug.
Adverse Reactions

Serious adverse drug reactions in the ITP double-blind studies were febrile neutropenia, diarrhea, pneumonia, and hypertensive crisis, which occurred in 1% of TAVALISSE patients. In addition, severe adverse reactions occurred including dyspnea and hypertension (both 2%), neutropenia, arthralgia, chest pain, diarrhea, dizziness, nephrolithiasis, pain in extremity, toothache, syncope, and hypoxia (all 1%).
Common adverse reactions (≥5% and more common than placebo) from FIT-1 and FIT-2 included: diarrhea, hypertension, nausea, dizziness, ALT and AST increased, respiratory infection, rash, abdominal pain, fatigue, chest pain, and neutropenia.
Click here for Important Safety Information and Full Prescribing Information.

To report side effects of prescription drugs to the FDA, visit www.fda.gov/medwatch or call 1-800-FDA-1088 (800-332-1088).

GAVRETO, REZLIDHIA and TAVALISSE are registered trademarks of Rigel Pharmaceuticals, Inc.

(Press release, Rigel, MAY 21, 2026, View Source [SID1234665937])

Olema Oncology Announces Encouraging Initial Clinical Data from the Phase 1 Study of OP-3136, a KAT6 Inhibitor, at 2026 ASCO Annual Meeting

On May 21, 2026 Olema Pharmaceuticals, Inc. ("Olema" or "Olema Oncology", Nasdaq: OLMA), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of targeted therapies for breast cancer and beyond, reported preliminary clinical data from the Phase 1 study of OP-3136, a potent lysine acetyltransferase 6 (KAT6) inhibitor. The data will be presented in a poster presentation on May 30, 2026 at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting taking place in Chicago, Illinois. Olema will also present a trial-in-progress poster for the Phase 3 OPERA-02 trial of palazestrant in combination with ribociclib in frontline estrogen receptor-positive, human epidermal growth factor receptor 2-negative (ER+/HER2-) metastatic breast cancer.

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"We are pleased to share the initial Phase 1 data for OP-3136, which demonstrated acceptable tolerability and promising anti-tumor activity as a monotherapy across multiple dose levels in various advanced solid tumor types," said Sean P. Bohen, M.D., Ph.D., President and Chief Executive Officer of Olema Oncology. "The decreases in tumor size observed in over two-thirds of evaluable patients and evidence of on-target engagement reinforce our confidence in OP-3136 as a potential best-in-class KAT6 inhibitor and a potentially differentiated option for difficult-to-treat cancers. We look forward to progressing OP-3136 in development, particularly in combination with palazestrant in metastatic breast cancer."

The Phase 1 study evaluates dose escalation followed by dose expansion of OP-3136 in patients with ER+/HER2- advanced breast cancer (ABC), metastatic castration-resistant prostate cancer (mCRPC), and metastatic non-small cell lung cancer (mNSCLC). In Part 1A, OP-3136 monotherapy was administered orally once daily in 28-day cycles across dose levels from 2 mg to 45 mg. As of the March 2, 2026 data cut-off, 32 heavily pretreated patients who became resistant or intolerant to standard of care treatments were enrolled in this cohort.

Key Findings
Safety and Tolerability

OP-3136 monotherapy was well-tolerated with no dose-limiting toxicities observed across the evaluated daily dose range up to 45 mg per day orally.
Most treatment-related adverse events (TRAEs) were grade 1 or 2; no grade 4 or 5 TRAEs were observed. TRAEs were manageable with dose modifications; no treatment discontinuations occurred due to TRAEs.
The most common TRAEs were dysgeusia (81% any grade; 56% grade 1, 25% grade 2), anemia (38% any grade; 6% grade 3), and neutropenia (34% any grade; 28% grade 3).

Efficacy and Target Engagement

Among 19 response-evaluable patients across dose levels and tumor types, tumor shrinkage was observed in 13 patients; partial responses (PR) were observed in 3 patients with measurable disease, with 2 confirmed PRs and 1 unconfirmed PR.
The longest duration of treatment is 62 weeks.
11 patients remain on treatment, including 9 with ABC and 2 with mCRPC.
Across all doses tested, OP-3136 demonstrated rapid, sustained, and significant reduction in levels of lysine 23 of histone H3, a direct target of KAT6, consistent with on-target KAT6 inhibition.

Pharmacokinetics

OP-3136 exhibited predictable, dose-proportional plasma exposure across all doses tested.
At doses of 6 mg and above, steady-state concentrations exceeded efficacy targets based on preclinical models.

"These initial results from the Phase 1 study of OP-3136, including confirmed and durable responses and a manageable safety profile in a heavily pretreated population, underscore the potential of KAT6 inhibition as a therapeutic strategy in different solid tumor types," said Amita Patnaik, MD, FRCPC, Principal Investigator, Co-Founder, and Co-Director of Clinical Research at the START Center for Cancer Research. "Supported by evidence of target engagement and predictable pharmacokinetics across all doses evaluated to date, I am excited to further evaluate this novel therapy, both as a monotherapy and in combination with multiple agents, as Phase 1 development continues."

OP-3136 Poster Presentation Details
Title: A phase 1, first-in-human study of OP-3136, a novel oral selective KAT6A/B inhibitor, as monotherapy in advanced solid tumors and in combination with endocrine therapy in ER+, HER2- advanced breast cancer: preliminary results
Abstract Number: 3088
Poster Number: 225
Date/Time: May 30, 2026 from 1:30pm-4:30pm CT / 2:30pm-5:30pm ET

OPERA-02 Trial-in-Progress Poster Presentation Details
Olema will also present a trial-in-progress poster for the Phase 3 OPERA-02 trial of palazestrant in combination with ribociclib in frontline ER+/HER2- metastatic breast cancer.

Title: OPERA-02: A phase 3 study of palazestrant plus ribociclib as first-line treatment of ER+, HER2- advanced breast cancer
Abstract Number: TPS1152
Poster Number: 261b
Date/Time: June 1, 2026 from 1:30pm-4:30pm CT / 2:30pm-5:30pm ET

Copies of these posters will be available on the Publications page of Olema’s website in alignment with the ASCO (Free ASCO Whitepaper) embargo. Additional information, including abstracts, is available on the ASCO (Free ASCO Whitepaper) Annual Meeting website.

(Press release, Olema Oncology, MAY 21, 2026, View Source [SID1234665936])