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Taiho Oncology Announces New England Journal of Medicine Publication of First All-Oral Regimen in Newly Diagnosed Acute Myeloid Leukemia

On June 3, 2026 Taiho Oncology, Inc., a company developing and commercializing novel treatments for hematologic malignancies and solid tumors, reported the publication in the New England Journal of Medicine of results from the ASCERTAIN-V Phase 1/2 clinical trial, which evaluated the first all-oral regimen of decitabine-cedazuridine plus venetoclax in patients with newly diagnosed acute myeloid leukemia (AML) who are ineligible for intensive induction chemotherapy. The study demonstrated favorable response rates and survival with expected myelosuppressive effects.1

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Previously presented at the 2025 ASCO (Free ASCO Whitepaper) Annual Meeting and the 2025 European Hematology Association (EHA) (Free EHA Whitepaper) Congress, the results suggest this first all-oral regimen may provide outcomes consistent with existing hypomethylating agent–venetoclax–based approaches for patients with newly diagnosed AML who cannot receive intensive induction therapy.

Nearly 23,000 people in the U.S. are expected to be diagnosed with AML, a cancer of the blood and bone marrow, in 2026. 2 More than half of those patients will likely be deemed ineligible for intensive induction chemotherapy because of factors such as older age (75 or older) or poor health. 3 For these patients, the standard treatment often combines oral venetoclax with parenteral hypomethylating agents, requiring frequent injections or infusions and an average of 12.9 days per month engaging in healthcare visits. 4

Oral decitabine-cedazuridine is a hypomethylating agent that has demonstrated oral bioavailability and equivalent pharmacokinetic exposure to intravenous decitabine. 5

"We are committed to advancing therapies that improve the lives of patients, their families and their caregivers," said Harold Keer, MD, PhD, Chief Medical Officer, Taiho Oncology. "An all-oral regimen for AML could help reduce the significant time and cost burden associated with treatment in hospitals or infusion centers."

The ASCERTAIN-V trial evaluated the first all-oral regimen of oral decitabine-cedazuridine plus venetoclax in 189 patients (Phase 1, n=30; Phase 2a, n=58; Phase 2b, n=101). After an initial ramp-up period, patients were treated in 28-day cycles, receiving decitabine-cedazuridine on days one through five and venetoclax daily. Key results include complete response rates, durable remissions and survival, and a safety profile consistent with hypomethylating agent-venetoclax regimens.

In the Phase 2b of ASCERTAIN-V:
The complete response rate was achieved in 47% of patients (95% CI: 36-57%). The composite of complete response or complete response with incomplete hematologic recovery was 63% (95% CI: 53-73%).
The median time to marrow blasts of less than 5% was 28 days (range 22 to 57), and the median time to a complete response or complete response with incomplete hematologic recovery was 35 days (range 27 to 58).
Among patients achieving complete response, 80% (95% CI: 64%, 90%) had an ongoing response at six months and 75% (95% CI: 57%, 87%) at 12 months.
Median overall survival was 15.5 months (95% CI 7.6-not estimable). The 30- and 60-day mortality rates were 3% and 10%, respectively. The median follow-up period was 11.2 months.
Serious adverse events were reported in 84% of patients, consistent with known hypomethylating agent–venetoclax regimens. Common related Grade ≥3 adverse events were anemia (30%), neutropenia (26%) and febrile neutropenia (25%).

No drug–drug interactions were observed between decitabine-cedazuridine and venetoclax.
In the Phase 2b of ASCERTAIN-V, decitabine-cedazuridine and venetoclax dosing was modified once leukemic blast clearance was achieved as measured by bone marrow morphology assessments. Serious adverse events and febrile neutropenia decreased as venetoclax dosing days decreased with each treatment cycle. This information potentially supports the need to explore a treatment strategy of an initial induction followed by dose-adjusting ongoing cycles to enhance tolerability and minimize cytopenia-related complications in patients who have reached remission.

"The positive results of the trial — response rates, safety, pharmacokinetics and tolerability data — suggest that an all-oral regimen could potentially become a meaningful alternative to existing treatment protocols that involve parenteral hypomethylating agents for patients with AML who can’t undergo intensive induction chemotherapy," said Gail J. Roboz, M.D., the paper’s lead author, a professor of medicine, director of the Clinical and Translational Leukemia Program and a member of the Sandra and Edward Meyer Cancer Center at Weill Cornell Medicine and a hematologist oncologist at NewYork-Presbyterian/Weill Cornell Medical Center in New York City. "An all-oral treatment regimen is a highly anticipated addition to current treatment options for patients with AML."

About Decitabine and Cedazuridine Fixed-Dose Combination
This product is an orally administered, fixed dose combination of the approved anti-cancer DNA hypomethylating agent, decitabine, together with cedazuridine,6 an inhibitor of cytidine deaminase.7 By inhibiting cytidine deaminase in the gut and the liver, the fixed dose combination is designed to allow for oral delivery of decitabine over five days in a given cycle to achieve comparable systemic exposure to IV decitabine administered over five days.

(Press release, Taiho, JUN 3, 2026, View Source [SID1234666419])

ADC Therapeutics Announces Results From LOTIS-5 Phase 3 Confirmatory Clinical Trial of ZYNLONTA® in Combination with Rituximab in Relapsed or Refractory Diffuse Large B-Cell Lymphoma

On June 3, 2026 ADC Therapeutics SA (NYSE: ADCT) reported topline data from its Phase 3 LOTIS-5 confirmatory trial evaluating ZYNLONTA (loncastuximab tesirine-lpyl) in combination with rituximab in patients with relapsed or refractory diffuse large B-cell lymphoma (r/r DLBCL). ZYNLONTA plus rituximab achieved statistical significance on the trial’s primary endpoint of progression-free survival (PFS) and demonstrated no detrimental effect on the key secondary efficacy endpoint of overall survival (OS). In addition, a higher complete response (CR) rate and duration of CRs (DoCR) were observed with ZYNLONTA plus rituximab. Overall, treatment emergent adverse event (TEAE) rates were similar between arms. Similar rates of overall Grade ≥3 TEAEs greater than 5% were observed across both arms, with hematologic TEAEs higher in the control arm and infection, hepatotoxicity, and edema/effusion higher in the test arm. Serious adverse events (SAEs), TEAEs leading to study drug withdrawal, and Grade 5 events were higher in the test arm, with the majority of Grade 5 TEAEs in the test arm occurring in patients aged 75 years or older.

"In the context of a positive study, based on the totality of the data, we plan to discuss the benefit-risk profile of this combination with the U.S. FDA as we prepare for the planned supplemental Biologics License Application (sBLA) filing," said Ameet Mallik, Chief Executive Officer of ADC Therapeutics. "We would like to extend our thanks to the patients, investigators, and clinical teams who contributed to this important trial."

The LOTIS-5 trial is a randomized, open‐label, two‐arm, multicenter study evaluating ZYNLONTA plus rituximab versus the standard immunochemotherapy rituximab gemcitabine‐oxaliplatin (R‐GemOx), for the treatment of r/r DLBCL after one or more lines of systemic therapy. The study met the primary endpoint of PFS (per independent review committee) with statistical significance (HR = 0.73; p-value = 0.008 two sided), with a median PFS of 6.1 months for ZYNLONTA plus rituximab vs 4.7 months for R-GemOx. Overall survival showed no detrimental effect with ZYNLONTA plus rituximab compared to the control arm (HR = 0.96, impacted by the earlier use and a higher rate of new anti-lymphoma treatment switching in the control arm). Overall response rate (ORR) was 58.1% vs. 45.2%, CR rate was 39.5% vs. 26.7%, median duration of response (DOR) was 9.2 months vs. 7.7 months, and median DoCR was 16.8 months vs. 12.3 months for ZYNLONTA plus rituximab compared to R-GemOx, respectively. Of patients achieving CR, 48.5% vs. 16.7% remained in CR at 24 months in favor of ZYNLONTA plus rituximab. Of note, results in North America were consistent with the overall study results.

Overall, TEAE rates were similar between arms (98.5% vs. 97.5%). Higher rates of SAEs were seen in the test arm (49.0% vs. 34.5%). Grade ≥3 TEAEs observed in > 5% of patients were hematologic (40.7% vs. 59.4%), followed by infection/infestations (24.5% vs. 15.7%), then hepatotoxicity (17.2% vs. 8.1%) and oedema/effusion (7.4% vs. 0.5%) when comparing ZYNLONTA plus rituximab to R-GemOx. A higher rate of Grade 5 TEAEs was observed in the ZYNLONTA plus rituximab arm (27 pts/13.2%) vs. R-GemOx (9 pts/4.6%). Of note, the majority of Grade 5 TEAEs in the test arm occurred in patients aged 75 years or older. Higher rates of TEAEs leading to any drug withdrawal occurred in the ZYNLONTA plus rituximab arm (25.5% vs. 9.1%). In this study, the TEAE reporting window was defined as 105 days after the last dose of study treatment or the start of new anticancer therapy, whichever is earlier. The rates of TEAEs in this study were impacted by the longer overall TEAE observation time in the test vs. control arm. This difference is primarily driven by a higher rate of and earlier switching to subsequent therapies in the control arm.

"LOTIS-5 was designed to address a clear unmet need in r/r DLBCL in patients who cannot access or who progress on a CAR-T or other complex therapies," said Mehdi Hamadani, MD, Professor of Medicine, Associate Director of Clinical Research, Section Chief of Hematologic Malignancies at Medical College of Wisconsin and principal investigator for the trial. "Based on these results, I believe this combination may provide an additional option in treating relapsed or refractory DLBCL."

"Based on these topline results from LOTIS-5, we look forward to discussing next steps for this combination of ZYNLONTA plus rituximab with the U.S. FDA," said Mohamed Zaki, MD, PhD, Chief Medical Officer of ADC Therapeutics. "We intend to conduct a pre-sBLA meeting in August and are preparing for a planned sBLA submission in the fourth quarter of 2026."

In addition, the Company will continue to evaluate a broad range of value maximizing alternatives, including but not limited to near-term cost reduction initiatives.

For more information about LOTIS-5, please visit View Source (identifier: NCT04384484).

Conference Call Details
ADC Therapeutics management will host a conference call and live audio webcast to discuss the LOTIS-5 results today at 4:30 p.m. EDT. To access the conference call, please register here. Registrants will receive the dial-in number and unique PIN. It is recommended that you join 10 minutes before the event, though you may pre-register at any time. A live webcast of the call will be available under "Events & Presentations" in the Investors section of the ADC Therapeutics website at ir.adctherapeutics.com. The archived webcast will be available for 30 days following the call.

(Press release, ADC Therapeutics, JUN 3, 2026, View Source [SID1234666418])

Ingenix Raises €13m From Sofinnova Partners-Led Syndicate to Scale Modality Fusion, a Novel Architecture for Drug Development

On June 3, 2026 Ingenix, the AI and biology company built around a single question – "What would it take for AI to truly understand biology?" – reported a €13m seed-extension funding round led by Sofinnova Partners, with participation from Inovo VC and OTB VC.

The funding will scale development of Ingenix’s Biological Reasoning Engine and broaden its work with pharma and biotech partners through the Qualified Access Program, launching today.

The dominant approach in AI for drug development, which trains larger models on larger datasets assuming biology will yield to scale, faces a structural problem. Biology is profoundly complex, fundamentally non-linguistic, and runs across modalities and biological scales. No single model, however large, can capture that on its own. Moreover, no single company holds all the data, across every modality and biological scale, to train one. Ingenix has built a different architecture, Modality Fusion, which integrates best-in-class models across modalities and biological scales, then reasons across their representations directly. The Biological Reasoning Engine is what Modality Fusion enables.

"This funding lets us extend the Biological Reasoning Engine to the partners and questions where it can do the most useful work," said Piotr Surma, CEO and co-founder of Ingenix. "We built Ingenix on the conviction that biology needs an AI architecture designed for biology and not a general-purpose model retrofitted to it. The early results have been stronger than we forecast, and we’re excited to extend the Engine to a select number of partners through the Qualified Access Program."

"It is no longer enough to just build models," added Simon Turner, Partner at Sofinnova Partners. "Ingenix is building the reasoning layer, the part that actually connects the biology, the chemistry, and the clinical data into something a scientist can interrogate and act on. That’s the hard bit, and that’s where the value compounds. We’re thrilled to back a team that gets that."

The Engine in Action: ADC Payload Prioritization

In a recent engagement, Ingenix applied its Biological Reasoning Engine to a dual-payload ADC prioritization problem for an oncology biotech which had thousands of possible payload configurations but lacked the experimental capacity to test them.

The Engine produced 15 candidate combinations. Under blind expert review by the biotech’s translational science team, the predictions broke down as follows:

5 were publicly known hypotheses.
2 were supported in existing literature but not widely cited.
3 had been confirmed by the biotech through internal experiments but never published and never disclosed to Ingenix.
5 were novel hypotheses not previously considered by the biotech team. Of these, the biotech flagged 3 as actionable candidates.
The double-payload ADC space is too new for any AI system to have seen meaningful training data on it. The engine reached its predictions by reasoning from first principles about the underlying biology, rather than by pattern-matching against prior examples.

In short, insights that had taken the biotech several years of research and millions of euros to develop were accomplished by the Engine in a matter of minutes.

Applications to the Qualified Access Program are now open at ingenix.ai/qap.

(Press release, Ingenix, JUN 3, 2026, View Source [SID1234666417])

IDEAYA Biosciences Announces Clinical Collaboration with Roche in MTAP-Deleted RAS-Mutant Pancreatic Cancer

On June 3, 2026 IDEAYA Biosciences, Inc. (NASDAQ: IDYA), a precision medicine oncology company committed to the discovery and development of targeted therapeutics, reported it has entered into a clinical collaboration with Roche to evaluate the efficacy and safety of IDE892, its investigational, potential best-in-class PRMT5 inhibitor, in combination with Roche’s RG6505, a pan-RAS inhibitor, in patients with pancreatic ductal adenocarcinoma (PDAC) that carry an MTAP deletion. IDEAYA will sponsor the clinical trial combination study, and Roche will supply RG6505.

"We are pleased to evaluate the clinical combination of IDE892 with RG6505 in MTAP-deleted RAS-mutant PDAC," said Yujiro S. Hata, President and Chief Executive Officer, IDEAYA Biosciences. "This collaboration aligns with our broader clinical strategy to evaluate rational combinations with assets in our MTAP-deletion portfolio, and there remains especially high unmet need in PDAC."

IDE892 was designed to be a potential best-in-class PRMT5 inhibitor and has demonstrated robust monotherapy regressions in MTAP-deleted preclinical models. IDEAYA is evaluating IDE892 in a Phase 1 dose escalation clinical trial in MTAP-deleted solid tumors and plans to initiate Phase 1 combination cohorts, in PDAC with RG6505 as well as in NSCLC and other solid tumors with IDE397, IDEAYA’s proprietary MAT2A inhibitor.

MTAP deletion is estimated to occur in up to 40% of PDAC and almost all MTAP-deleted PDAC harbor co-occurring RAS mutations. Combining a PRMT5 inhibitor with a pan-RAS inhibitor may have the potential to drive deeper and more durable responses for MTAP-deleted PDAC patients who currently have no approved targeted treatment options.

Under the clinical collaboration, IDEAYA and Roche each retain all commercial rights to their respective compounds, including as monotherapy and as combination therapies. There will be joint IDEAYA and Roche governance to oversee the clinical combination study. The clinical collaboration also has the ability to evaluate a combination triplet with IDE892, RG6505, and IDE397, IDEAYA’s Phase 2 MAT2A inhibitor, upon joint IDEAYA and Roche approval.

(Press release, Ideaya Biosciences, JUN 3, 2026, View Source [SID1234666416])

TransCode Therapeutics Successfully Completes Phase 1a Clinical Trial, Reports Safety and Stabilization Data for TTX-MC138 in Metastatic Cancer

On June 3, 2026 TransCode Therapeutics, Inc. (NASDAQ: RNAZ) a clinical stage company pioneering immuno-oncology and RNA for the treatment of high risk and advanced cancer, reported further results of the Phase 1a dose escalation clinical trial. The trial met its primary endpoint of safety, with positive tolerability, combined with disease stabilization in multiple patients, and the absence of dose-limiting toxicities with its lead therapeutic candidate TTX-MC138. TTX-MC138, an investigational inhibitor of the key metastatic driver, microRNA-10b, has shown durable disease control. These findings support advancing TTX-MC138 into Phase 2a clinical development to assess efficacy in patients with circulating tumor DNA (ctDNA) positive colorectal cancer following curative–intent therapy.

TTX-MC138 has been administered to 16 patients who received 86 doses. The median treatment duration was 11.3 weeks, with a range of four to 52.4 weeks, representing 2 to 20 cycles of treatment.
Notably, three patients remain on trial, and continue to receive TTX-MC138. One patient is at 21 cycles of treatment, another is at 16 cycles, and the third one is at 14 cycles of treatment. (Table 1)

Table 1: Trial demographics, met safety primary endpoint

Cohort

Dose

Number of Patients

DLT’s1

0.8mg/kg

1.6mg/kg

3.2mg/kg2

4.8mg/kg

1 No significant treatment-related safety events or dose limiting toxicities were observed.
2 Optional backfill 3 with additional patients.
3 One patient in each of cohort 2, 3 and 4 currently on study.

TransCode believes these results support its selection of the recommended Phase 2a dose (RP2D) of 4.8mg/kg.

In addition, the assessment of the trial patient population underscored the potential for durable disease control in participants with metastatic cancer.

Based on Response Evaluation Criteria in Solid Tumors (RECIST) standardized criteria to measure tumor response to treatment using imaging to categorize lesions and assess changes in size over time, 9 out of 14 (64%) of evaluable patients achieved stable disease lasting six months, demonstrating a durable disease activity.

"From a clinical perspective, it is quite encouraging to see how well tolerated this agent has been at the exposures achieved through the Phase 1a dose-escalation study, without any dose-limiting toxicities. That, combined with the observation of disease stabilization in a population with such advanced disease supports continued clinical development" noted Keith Flaherty, MD, Director of Clinical Research at the Massachusetts General Hospital Cancer Center, Professor of Medicine at Harvard Medical School and TransCode’s Advisory Board member.

The pharmacokinetics profile from the analysis of plasma from patients receiving TTX-MC138 demonstrated evidence of drug bioavailability consistent with earlier preclinical studies.

One patient diagnosed with metastatic thyroid cancer was noted to have a dramatic decrease in their thyroglobulin levels, a tumor marker associated with cancer progression. The patient has now had demonstrated stable disease for the last 12 months and is one of the three patients who remain on study. We believe that the patient’s continued participation in the study, together with the decline in their thyroglobulin levels, provides further evidence of therapeutic activity from TTX-MC138.

A clinical study report is in process. Several presentations are planned at future scientific congresses.

"As the safety and tolerability primary objectives of the trial were met, the encouraging rates of disease stabilization provide the rationale to advance TTX-MC138 clinical development in our recently initiated Phase 2a trial. We continue to believe that TTX-MC138 may offer a promising therapeutic option, if approved, for patients with metastatic disease who have limited treatment alternatives," said Daniel Vlock, MD, TransCode Consulting Clinician.

Further information about the trial is available at www.clinicaltrials.gov, (NCT Identifier: NCT06260774).

About TTX-MC138

TTX-MC138 is a first-in-class therapeutic candidate designed to inhibit microRNA-10b (miR-10b), a microRNA widely believed to be critical to the emergence and progression of many metastatic cancers. TransCode’s Phase 0 clinical trial produced evidence of delivery of a radiolabeled version of TTX-MC138 to metastatic lesions.

(Press release, TransCode Therapeutics, JUN 3, 2026, View Source [SID1234666415])