On December 8, 2025 Kura Oncology, Inc. (Nasdaq: KURA, "Kura") and Kyowa Kirin Co., Ltd. (TSE: 4151, "Kyowa Kirin") reported new data demonstrating a favorable safety profile and encouraging antileukemic activity for KOMZIFTI (ziftomenib) in combination with venetoclax and azacitidine (ven/aza) for the treatment of acute myeloid leukemia (AML) harboring NPM1 mutations (NPM1-m) or KMT2A rearrangements (KMT2A-r). The ongoing KOMET-007 Phase 1a/1b trial evaluated patients in cohorts with newly diagnosed chemotherapy-ineligible AML and relapsed/refractory (R/R) AML. The new data are being reported today in two oral presentations at the 67th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) (ASH 2025).

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"The addition of ziftomenib to venetoclax and azacitidine has shown promising clinical activity, with 86% of newly diagnosed NPM1-mutated AML patients achieving composite complete remission and 68% attaining deep molecular MRD negativity, though median duration of response and overall survival remain immature," said Gail J. Roboz, M.D., the William S. Paley Professor in Clinical Medicine and Director of the Clinical and Translational Leukemia Program at Weill Cornell Medicine and a hematologist/oncologist at NewYork-Presbyterian/Weill Cornell Medical Center. "In relapsed/refractory NPM1-m and KMT2A-r AML, overall response rates of 65% and 41% were observed, rising to 83% and 70% in venetoclax-naïve patients, underscoring ziftomenib’s potential benefit even in challenging settings. Importantly, inclusion of ziftomenib was generally well tolerated, paving the way for its integration into front-line and relapsed/refractory regimens through ongoing registrational trials."

KOMZIFTI (ziftomenib), the first and only once-daily oral menin inhibitor for adult patients with R/R AML with a susceptible NPM1 mutation who have no satisfactory alternative treatment options, has been approved by the U.S. Food and Drug Administration (FDA) and is commercially available in the United States.

Ziftomenib + Venetoclax/Azacitidine in Newly Diagnosed NPM1-m AML

The ongoing KOMET 007 Phase 1a/b trial (NCT05735184) evaluated 40 patients with newly diagnosed NPM1-m AML as of the September 24, 2025 data cutoff date. Of these, 58% (23/40) had an ECOG performance status of 2 and 37 were response evaluable.

Robust activity was observed in newly diagnosed NPM1-m AML, including high rates of durable morphologic complete responses (CRc 86%; CR 73%).

68% of CRc responders achieved molecular MRD negativity by central next-generation sequencing (NGS).
Median duration of CR and OS were not reached at median follow-up of 26.1 weeks (range 1.6–54.1) as of the data cutoff.
68% of patients remained alive and on treatment or in long-term follow-up as of the data cutoff.
Five chemotherapy-ineligible patients received HSCT; three received ziftomenib maintenance therapy thereafter.
The triplet combination was generally well tolerated in newly diagnosed NPM1-m AML, with a safety profile consistent with that reported for ven/aza alone. Rates of ziftomenib-related myelosuppression were low, and the median times to neutrophil and platelet recovery were also consistent with those expected for ven/aza alone. One case each of grade 2 differentiation syndrome and grade 3 investigator-assessed QTc prolongation were successfully managed without treatment discontinuation.

Ziftomenib + Venetoclax/Azacitidine in R/R AML

The ongoing KOMET 007 Phase 1a/b trial (NCT05735184) evaluated 83 patients with R/R NPM1-m or KMT2A-r AML as of the September 24, 2025 data cutoff date. Of these, 58% (48/83) had received prior venetoclax and 80 were response evaluable.

Robust activity was observed in patients with R/R NPM1-m AML, including among those previously treated with venetoclax.

ORR was 65% and CRc rate was 48%, with CRc median duration of 39.9 weeks.
In venetoclax-naïve patients, ORR was 83% and CRc rate was 70%, compared with 48% and 28%, respectively, in venetoclax-exposed patients.
Median OS was 54.9 weeks (95% CI 32.0–NE).
14 patients received HSCT, five proceeded to ziftomenib maintenance therapy, and five were pending maintenance at time of data cutoff.
In patients with R/R KMT2A-r AML, encouraging activity was also observed.

ORR was 41% and CRc rate was 28%, with CRc median duration of 12.4 weeks.
In venetoclax-naïve patients, ORR was 70% and CRc rate was 60%.
Median OS was 21.1 weeks (95% CI 12.4–64.9).
Two patients received HSCT and both proceeded to ziftomenib maintenance therapy.
The combination was generally well tolerated in both R/R NPM1-m and R/R KMT2A-r AML. Rates of ziftomenib-related myelosuppression were low, with neutrophil and platelet recovery consistent with expectations for ven/aza alone. No ziftomenib-related QTc prolongation was reported. One grade 3 differentiation syndrome case (in an NPM1-m patient) was successfully resolved with protocol-specified measures, and the patient resumed treatment with ziftomenib.

"We’re truly encouraged by the consistent safety profile and the depth of responses observed with ziftomenib in combination with venetoclax and azacitidine across both newly diagnosed and relapsed/refractory NPM1-mutated and KMT2A-rearranged AML patients," said Mollie Leoni, M.D., Chief Medical Officer at Kura Oncology. "These compelling data reinforce our conviction that ziftomenib has the potential to become a foundational, best-in-class menin inhibitor for patients with AML. Importantly, we continue to activate sites in our pivotal KOMET-017 trials. The combination of a well-considered trial design and a compelling benefit-risk profile for ziftomenib gives us confidence in the pace and quality of enrollment of newly diagnosed and relapsed/refractory patients."

Presentations
Slides from the oral presentations will be available on Kura’s website at www.kuraoncology.com under the Posters and Presentations tab in the Ziftomenib section, and in the ASH (Free ASH Whitepaper) 2025 online program.

Virtual Investor Event
Kura will host a webcast and conference call today, December 8, 2025, at 12:30 p.m. ET / 9:30 a.m. PT featuring Kura management, Eunice Wang, M.D., Chief of Leukemia Service and Professor of Oncology at Roswell Park Comprehensive Center, and Amer Zeidan, M.B.B.S., M.H.S., Chief, Division of Hematologic Malignancies and Professor of Medicine at Yale School of Medicine. The live webcast and replay will be available on the on the Company’s website at www.kuraoncology.com under the Investors tab in the Events and Presentations section.

About KOMZIFTI (ziftomenib)
KOMZIFTI (ziftomenib) is an oral menin inhibitor approved for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with a susceptible NPM1 mutation who have no satisfactory alternative treatment options.

Ziftomenib is in development for the front-line treatment of AML harboring NPM1 mutations, KMT2A translocations and FLT3 mutations, with the potential to be combined with approved therapies and benefit a broad spectrum of patients earlier in their disease course.

IMPORTANT SAFETY INFORMATION FOR KOMZIFTI FROM THE U.S. PRESCRIBING INFORMATION

Boxed WARNING: DIFFERENTIATION SYNDROME

Differentiation syndrome, which can be fatal, has occurred with KOMZIFTI. Signs and symptoms may include fever, joint pain, hypotension, hypoxia, dyspnea, rapid weight gain or peripheral edema, pleural or pericardial effusions, pulmonary infiltrates, acute kidney injury, and rashes. If differentiation syndrome is suspected, interrupt KOMZIFTI, and initiate oral or intravenous corticosteroids with hemodynamic and laboratory monitoring until symptom resolution; resume KOMZIFTI upon symptom improvement.

WARNINGS AND PRECAUTIONS

Differentiation Syndrome

KOMZIFTI can cause fatal or life-threatening differentiation syndrome (DS). DS is associated with rapid proliferation and differentiation of myeloid cells. Symptoms of DS, including those seen in patients treated with KOMZIFTI, may include fever, hypoxia, joint pain, hypotension, dyspnea, rapid weight gain or peripheral edema, pleural or pericardial effusions, acute kidney injury, and rashes.

In the clinical trial, DS occurred in 29 (26%) of 112 patients with R/R AML with an NPM1 mutation who were treated with KOMZIFTI at the recommended dosage. DS was Grade 3 in 13% and fatal in two patients. In broader evaluation of all patients with any genetic form of AML treated with KOMZIFTI monotherapy in clinical trials, DS occurred in 25% of patients. Four fatal cases of DS occurred out of 39 patients with KMT2A-rearranged AML treated with KOMZIFTI. KOMZIFTI is not approved for use in patients with KMT2A-rearranged AML.

In the 112 patients with an NPM1 mutation, DS was observed with and without concomitant hyperleukocytosis, in as early as 3 days and up to 46 days after KOMZIFTI initiation. The median time to onset was 15 days. Two patients experienced more than one DS event. Treatment was interrupted and resumed in 15 (13%) patients, while it was permanently discontinued in 2 (2%) patients.

Prior to starting treatment with KOMZIFTI, reduce the WBC counts to less than 25 x 10⁹/L. If DS is suspected, interrupt KOMZIFTI, initiate oral or intravenous corticosteroids (e.g., dexamethasone 10 mg every 12 hours) for a minimum of 3 days with hemodynamic and laboratory monitoring. Resume treatment with KOMZIFTI at the same dose level when signs and symptoms improve and are Grade 2 or lower. Taper corticosteroids over a minimum of 3 days after adequate control or resolution of symptoms. Symptoms of DS may recur with premature discontinuation of corticosteroid treatment.

QTc Interval Prolongation

KOMZIFTI can cause QTc interval prolongation. In the clinical trial, QTc interval prolongation was reported as an adverse reaction in 12% of 112 patients treated with KOMZIFTI at the recommended dosage for R/R AML with an NPM1 mutation. QTc interval prolongation was Grade 3 in 8% of patients. The heart-rate corrected QT interval (using Fridericia’s method) (QTcF) was greater than 500 msec in 9% of patients, and the increase from baseline QTcF was greater than 60 msec in 12% of patients. KOMZIFTI dose reduction was required for 1% of patients due to QTc interval prolongation. QTc prolongation occurred in 14% of the 42 patients less than 65 years of age and in 10% of the 70 patients 65 years of age or older.

Correct electrolyte abnormalities, including hypokalemia and hypomagnesemia, prior to treatment with KOMZIFTI. Perform an ECG prior to initiation of treatment with KOMZIFTI, and do not initiate KOMZIFTI in patients with QTcF > 480 msec. Perform an ECG at least once weekly for the first four weeks on treatment, and at least monthly thereafter. Interrupt KOMZIFTI if the QTc interval is > 500 ms or the change from baseline is > 60 ms (Grade 3). In patients with congenital long QTc syndrome, congestive heart failure, electrolyte abnormalities, or those who are taking medications known to prolong the QTc interval, more frequent ECG monitoring may be necessary. Concomitant use of KOMZIFTI with drugs known to prolong the QTc interval may increase the risk of QTc interval prolongation, result in a greater increase in the QTc interval and adverse reactions associated with QTc interval prolongation, including Torsades de Pointes, other serious arrhythmias, and sudden death.

Embryo-Fetal Toxicity

Based on findings in animals and its mechanism of action, KOMZIFTI can cause embryo-fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to the fetus. Advise females of reproductive potential to use effective contraception during treatment with KOMZIFTI and for 6 months after the last dose. Advise males with female partners of reproductive potential to use effective contraception during treatment with KOMZIFTI and for 3 months after the last dose.

ADVERSE REACTIONS

Fatal adverse reactions occurred in 4 (4%) patients who received KOMZIFTI, including 2 with differentiation syndrome, 1 with infection, and 1 with sudden death. Serious adverse reactions were reported in 79% of patients who received KOMZIFTI. Serious adverse reactions occurring in ≥ 5% of patients included infection without an identified pathogen (29%), febrile neutropenia (18%), bacterial infection (16%), differentiation syndrome (16%), and dyspnea (6%).

Dosage interruption of KOMZIFTI due to an adverse reaction occurred in 54% of patients. Adverse reactions that required dose interruption in ≥ 2% of patients included infection without an identified pathogen (15%), differentiation syndrome (13%), febrile neutropenia (5%), pyrexia (4%), electrocardiogram QT prolonged (4%), leukocytosis (4%), bacterial infection (3%), cardiac failure (2%), cholecystitis (2%), diarrhea (2%), pruritus (2%), and thrombosis (2%). Dose reduction of KOMZIFTI due to an adverse reaction occurred in 4% of patients. Permanent discontinuation of KOMZIFTI due to an adverse reaction occurred in 21% of patients. Adverse reactions that required permanent discontinuation of KOMZIFTI in ≥ 2% of patients were infection without an identified pathogen (8%), bacterial infection (4%), cardiac arrest (2%), and differentiation syndrome (2%).

Most common (≥ 20%) adverse reactions, including laboratory abnormalities, were aspartate aminotransferase increased (53%), infection without an identified pathogen (52%), potassium decreased (52%), albumin decreased (51%), alanine aminotransferase increased (50%), sodium decreased (49%), creatinine increased (45%), alkaline phosphatase increased (41%), hemorrhage (38%), diarrhea (36%), nausea (35%), fatigue (34%), edema (30%), bacterial infection (28%), musculoskeletal pain (28%), bilirubin increased (27%), potassium increased (26%), differentiation syndrome (26%), pruritus (23%), febrile neutropenia (22%), and transaminases increased (21%).

DRUG INTERACTIONS

Drug interactions may occur when KOMZIFTI is concomitantly used with:

Strong or Moderate CYP3A4 Inhibitors: Monitor patients more frequently for KOMZIFTI-associated adverse reactions.
Strong or Moderate CYP3A4 Inducers: Avoid concomitant use of KOMZIFTI.
Gastric Acid Reducing Agents: Avoid concomitant use of KOMZIFTI with proton pump inhibitors (PPIs), H2 receptor antagonists (H2RAs), or locally acting antacids. If concomitant use with H2RAs or locally acting antacids cannot be avoided, modify KOMZIFTI administration time.
Take KOMZIFTI 2 hours before or 10 hours after administration of an H2 receptor antagonist.
Take KOMZIFTI 2 hours before or 2 hours after administration of a locally acting antacid.
Drugs that Prolong the QTc Interval: Avoid concomitant use of KOMZIFTI. If concomitant use cannot be avoided, obtain ECGs when initiating, during concomitant use, and as clinically indicated. Interrupt KOMZIFTI if the QTc interval is > 500 ms or the change from baseline is > 60 ms.
USE IN SPECIFIC POPULATIONS

Pregnancy: Based on findings in animals and its mechanism of action, KOMZIFTI can cause embryo-fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Verify pregnancy status in females of reproductive potential prior to starting KOMZIFTI.

Lactation: Because of the potential for adverse reactions in the breastfed child, advise women not to breastfeed during treatment with KOMZIFTI and for 2 weeks after the last dose.

Infertility: Based on findings in animals, KOMZIFTI may impair fertility in females and males of reproductive potential.

Please see full Prescribing Information, including Boxed WARNING.

(Press release, Kura Oncology, DEC 8, 2025, https://ir.kuraoncology.com/news-releases/news-release-details/kura-oncology-and-kyowa-kirin-report-combination-data-komziftitm [SID1234661264])

On December 8, 2025 The Menarini Group ("Menarini"), a leading international pharmaceutical and diagnostics company, and Stemline Therapeutics, Inc. ("Stemline"), a wholly-owned subsidiary of the Menarini Group, focused on bringing transformational oncology treatments to cancer patients, reported it will present updated meaningful median progression-free survival (mPFS) efficacy results from two combination regimens of the Phase 2 ELEVATE study in patients with estrogen receptor-positive (ER+), HER2-negative (HER2-) locally advanced or metastatic breast cancer (mBC). The ELEVATE study was designed to evaluate the safety and efficacy of oral-oral combination treatment options to overcome different resistance mechanisms observed in ER+/HER2- mBC with the goal of improving patient outcomes. These data will be presented at the 2025 San Antonio Breast Cancer Symposium (SABCS), December 9-12.

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"The encouraging progression-free survival data increase our confidence in the role elacestrant could play as an endocrine therapy backbone in the combination setting," said Virginia Kaklamani, MD, DSc, Professor of Medicine in the Division of Hematology/Oncology at UT Health San Antonio, and Leader of the Breast Cancer Program at UT Health San Antonio MD Anderson Cancer Center. "The safety profile of elacestrant in combination with everolimus or abemaciclib is consistent with the known safety profiles of each drug. No new safety signals have been observed."

The ELEVATE data to be presented at SABCS demonstrate that elacestrant in combination with everolimus or with abemaciclib shows a consistent PFS benefit, irrespective of ESR1 mutation status in patients with ER+/HER2- mBC, who experience disease progression on endocrine therapy (ET), with or without prior exposure to CDK4/6 inhibitors. These updated results also show that the safety of the combinations are consistent with the known safety profiles of each targeted therapy plus standard of care endocrine therapy.

Phase 2 mPFS in months (95% CI) in all patients and subgroups

Patient Population

Elacestrant 345 mg QD

+ Everolimus 7.5mg QD

(n=50)

Elacestrant 345 mg QD

+ Abemaciclib 150 mg BID

(n=60)

All patients

8.3 [4.0 – 10.2]

14.3 [7.3-16.6]

Visceral disease

7.7 [3.8 – 9.4]

14.3 [7.4-16.6]

No prior fulvestrant

8.3 [4.2 – 12.9]

14.8 [8.7-NR]

No primary endocrine resistance

8.3 [4.0-12.9]

14.3 [7.3-16.6]

ESR1mut

8.7 [3.5 – 12.9]

*

ESR1wt

9.0 [4.2 – 12.7]

*

PIK3CAmut

8.3 [3.6 – 10.2]

*

PIK3CAwt

9.6 [5.6 – NR]

*

*Maturity not reached for PFS (95% CI) for genomic subgroups (ESR1 / PIK3CA) in the elacestrant + abemaciclib cohort.

"The extensive evidence for elacestrant spans the monotherapy setting in our pivotal EMERALD study, now backed by two recent real-world data publications[1], [2], and in its growing potential in combination regimens, as highlighted by the data presented at SABCS," said Elcin Barker Ergun, CEO of the Menarini Group. "We remain deeply committed to fully exploring elacestrant’s potential benefit across multiple ongoing trials in both early stage and metastatic breast cancer."

Additionally, other elacestrant updates will be presented at SABCS, investigating its potential across the spectrum of breast cancer:

Presentation Title: Elacestrant in combination with everolimus or abemaciclib in patients with ER+/HER2-locally advanced or metastatic breast cancer (mBC): phase 2 results from ELEVATE, an open-label, umbrella study
Abstract Number: 1255
Presentation Date & Time: Thursday, December 11, 2025, 1:00 – 2:00 PM
Location: Hemisfair1-2
Presenting Author: Hope S. Rugo

Presentation Title: Elacestrant alone or in combination with triptorelin in premenopausal women with ER+/HER2-early breast cancer: primary analysis from the phase 2 SOLTI-2104-PremiÈRe trial
Abstract Number: 1123
Presentation Date & Time: Friday, December 12, 2025, 7:30 – 7:33 AM
Location: 301 ABC
Presenting Author: Mertixell Bellet

Presentation Title: ELEGANT: Elacestrant Versus Standard Endocrine Therapy (ET) in Women and Men With Node-positive, Estrogen Receptor-positive (ER+), HER2-negative (HER2-), Early Breast Cancer (eBC) With High Risk of Recurrence in a Global, Multicenter, Randomized, Open-label Phase 3 Study.
Abstract Number: 1276
Presentation Date & Time: Thursday, December 11, 2025, 12:30 – 2:00 PM
Location: Henry B. Convention Center
Presenting Author: Aditya Bardia

Presentation Title: The ADELA study: A Double-blind, Placebo-controlled, Randomized Phase 3 Trial of Elacestrant (ELA)+ Everolimus (EVE) Versus ELA + Placebo (PBO) in ER+/HER2-Advanced Breast Cancer (aBC) Patients with ESR1-mutated Tumors Progressing on Endocrine Therapy (ET) + CDK4/6i
Abstract Number: 1141
Presentation Date & Time: Wednesday, December 10, 2025, 12:30 – 2:00 PM
Location: Henry B. Convention Center
Presenting Author: Antonio Llombart-Cussac

Presentation Title: ERADICATE: A phase Ib/II study of elacestrant plus trastuzumab deruxtecan in patients with CDK4/6 inhibitor and endocrine-resistant HR+/HER2-low or HER2-ultralow metastatic breast cancer
Abstract Number: 2119
Presentation Date & Time: Friday, December 12, 2025, 12:30 – 2:00 PM
Location: Henry B. Convention Center
Presenting Author: Sara L. Sammons

Presentation Title: Hormonal receptor (HR)-positive HER2 negative breast cancer patients treated with preoperative Elacestrant and PULSAR adaptive radiotherapy: a phase II study (HELP Trial)
Abstract Number: 1071
Presentation Date & Time: Friday, December 12, 2025, 12:30 – 2:00 PM
Location: Henry B. Convention Center
Presenting Author: Luca Visani

About The Elacestrant Clinical Development Program

Elacestrant is also being investigated in several company-sponsored clinical trials in metastatic breast cancer disease, alone or in combination with other therapies. ELEVATE (NCT05563220) is a phase 1b/2 clinical trial evaluating the safety and efficacy of elacestrant combined with alpelisib, everolimus, capivasertib, palbociclib, ribociclib or abemaciclib. ELECTRA (NCT05386108) is an open-label phase 1b/2, multicenter study evaluating elacestrant in combination with abemaciclib in patients with ER+, HER2- breast cancer. The phase 2 portion evaluates this treatment regimen in patients with brain metastases. ELCIN (NCT05596409) is a phase 2 trial evaluating the efficacy of elacestrant in patients with ER+, HER2- advanced/metastatic breast cancer who received one or two prior hormonal therapies and no prior CDK4/6 inhibitors in the metastatic setting. ADELA (NCT06382948) is a phase 3 randomized, double-blinded trial evaluating elacestrant in combination with everolimus in patients with ER+, HER2- mBC with ESR1-mut tumors. Elacestrant is also being evaluated in additional investigator-led trials, in trials conducted in collaboration with other companies, in metastatic breast cancer as well as in early disease.

About ORSERDU (elacestrant)
U.S. Indication: ORSERDU (elacestrant), 345 mg tablets, is indicated for the treatment of postmenopausal women or adult men with estrogen receptor (ER)-positive, human epidermal growth factor receptor 2 (HER2)-negative, ESR1-mutated advanced or metastatic breast cancer with disease progression following at least one line of endocrine therapy.

Full prescribing information for the U.S. can be found at www.orserdu.com.

Important Safety Information
Warning and Precautions

Dyslipidemia: Hypercholesterolemia and hypertriglyceridemia occurred in patients taking ORSERDU at an incidence of 30% and 27%, respectively. The incidence of Grade 3 and 4 hypercholesterolemia and hypertriglyceridemia were 0.9% and 2.2%, respectively. Monitor lipid profile prior to starting and periodically while taking ORSERDU.

Embryo-Fetal Toxicity: Based on findings in animals and its mechanism of action, ORSERDU can cause fetal harm when administered to a pregnant woman. Advise pregnant women and females of reproductive potential of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with ORSERDU and for 1 week after the last dose. Advise male patients with female partners of reproductive potential to use effective contraception during treatment with ORSERDU and for 1 week after the final dose.

Adverse Reactions

Serious adverse reactions occurred in 12% of patients who received ORSERDU. Serious adverse reactions in >1% of patients who received ORSERDU were musculoskeletal pain (1.7%) and nausea (1.3%). Fatal adverse reactions occurred in 1.7% of patients who received ORSERDU, including cardiac arrest, septic shock, diverticulitis, and unknown cause (one patient each).
The most common adverse reactions (>10%), including laboratory abnormalities, of ORSERDU were musculoskeletal pain (41%), nausea (35%), increased cholesterol (30%), increased AST (29%), increased triglycerides (27%), fatigue (26%), decreased hemoglobin (26%), vomiting (19%), increased ALT (17%), decreased sodium (16%), increased creatinine (16%), decreased appetite(15%), diarrhea(13%), headache (12%), constipation (12%), abdominal pain (11%), hot flush (11%), and dyspepsia (10%).

Drug interactions

Concomitant use with CYP3A4 Inducers and/or inhibitors: Avoid concomitant use of strong or moderate CYP3A4 inhibitors with ORSERDU. Avoid concomitant use of strong or moderate CYP3A4 inducers with ORSERDU.

Use in specific populations

Lactation: Advise lactating women to not breastfeed during treatment with ORSERDU and for 1 week after the last dose.
Hepatic Impairment: Avoid use of ORSERDU in patients with severe hepatic impairment (Child-Pugh C). Reduce the dose of ORSERDU in patients with moderate hepatic impairment (Child-Pugh B).
The safety and effectiveness of ORSERDU in pediatric patients have not been established.

To report SUSPECTED ADVERSE REACTIONS, contact Stemline Therapeutics, Inc. at 1-877-332-7961 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

(Press release, Menarini, DEC 8, 2025, View Source;metastatic-breast-cancer-mbc-at-the-2025-san-antonio-breast-cancer-symposium-302635656.html [SID1234661281])

On December 8, 2025 Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), reported new data highlighting the potential of Lunsumio (mosunetuzumab-axgb) in earlier treatment lines for people living with different types of lymphoma, presented at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, December 6-9, 2025 in Orlando, Florida.

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"These data underscore the potential of Lunsumio to support more people living with lymphoma, building on the clinical benefit observed in later-stage follicular lymphoma," said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. "Moreover, the combinatorial potential of Lunsumio is evident in the two-drug regimens presented, which may enable outpatient treatment while preserving deep and durable efficacy."

Preliminary data support the potential for Lunsumio in combination with lenalidomide in relapsed or refractory (R/R) follicular lymphoma (FL)

First data from the single-arm extension of the Phase III CELESTIMO study, in 54 patients, demonstrated promising efficacy with this two-drug regimen in people with second-line or later (2L+) FL, including a complete response (CR) rate of 87.0% (95% confidence interval [CI]: 75.1–94.6). Cytokine release syndrome (CRS) events were reported in 27.8% of patients, and were predominantly low grade (Grade (Gr) 1: 22.2%; Gr 2: 3.7%; Gr 3: 1.9%), with all CRS events resolved. Neutropenia occurred in 40.7% of patients, and infections occurred in 57.4% of patients. These results indicate the potential of this combination to deliver meaningful outcomes earlier in the disease course. Primary analysis of the pivotal Phase III CELESTIMO study is anticipated in 2026.

Subcutaneous (SC) Lunsumio plus Polivy (polatuzumab vedotin-piiq) data demonstrate meaningful improvements for people with R/R large B-cell lymphoma (LBCL)

Long term follow-up data from the Phase Ib/II GO40516 study demonstrated sustained improvements in patients treated with investigational SC Lunsumio in combination with Polivy compared to those treated with Rituxan (rituximab) and Polivy in people with 2L+ LBCL. The overall response rate (ORR) was 77.5% (95% CI: 61.6–89.2) vs 50.0% (95% CI: 33.8–66.2) and median progression-free survival was 25.4 (95% CI: 9.2– not evaluable ) vs 6.4 months (95% CI:4.7–18.6). No new safety signals were identified. Adverse events (AE) included neutrophil count decreased/neutropenia (40%), febrile neutropenia (2.5%), infections (45%), and peripheral neuropathy (10%). Patient-reported outcomes from the Phase III SUNMO study investigating the same combination, demonstrated benefits across multiple aspects of health-related quality of life measures in comparison to Rituxan with gemcitabine and oxaliplatin particularly in maintaining or improving physical functioning, fatigue, lymphoma symptoms and peripheral neuropathy.

Results from these studies highlight the potential of this outpatient combination to prolong remission and improve outcomes for people living with this aggressive disease, without the need for conventional chemotherapy.

Long-term follow-up data show sustained responses with fixed-duration investigational SC Lunsumio and intravenous (IV) in third line or later (3L+) FL

Five-year follow-up data from the pivotal Phase II GO29781 study, the longest reported follow-up for a CD20xCD3 bispecific in R/R FL, showed durable remissions with IV Lunsumio, with a 5-year overall survival rate of 78.5% (95% CI: 69.6–87.4) and 54-month duration of CR rate (DOCR) of 52.0% (95% CI: 36.1-67.9). Furthermore, three-year follow-up data demonstrated durable responses with investigational SC Lunsumio with an ORR of 74.5%, CR rate of 62.8%, and 30-month DOCR of 53.0% (95% CI: 38.7-67.4). No new safety signals were observed in either study.

Lunsumio monotherapy is approved in over 60 countries for people with FL who have received at least two prior systemic therapies, with ongoing discussions with additional health authorities worldwide. SC Lunsumio was recently approved by the European Commission for FL after two or more lines of systemic therapy. A decision from the US Food and Drug Administration is expected soon.

Lunsumio, along with Columvi (glofitamab-gxbm), is part of Genentech’s industry-leading CD20xCD3 bispecific antibody portfolio. Continuing to explore new formulations and combinations of these medicines across different disease areas and lines of treatment is part of Genentech’s commitment to improve the patient experience and provide more choice to suit diverse patient and healthcare system needs.

About Lunsumio (mosunetuzumab-axgb)

Lunsumio is a first-in-class CD20xCD3 T-cell-engaging bispecific antibody designed to target CD20 on the surface of B cells and CD3 on the surface of T cells. This dual-targeting activates and redirects a patient’s existing T cells to engage and eliminate target B cells by releasing cytotoxic proteins into the B cells. A robust clinical development program for Lunsumio is ongoing, investigating the molecule as a monotherapy and in combination with other medicines, for the treatment of people with B-cell non-Hodgkin lymphomas, including follicular lymphoma, diffuse large B-cell lymphoma, and other indications.

About diffuse large B-cell lymphoma (DLBCL)

Diffuse large B-cell lymphoma (DLBCL) is an aggressive (fast-growing) blood cancer and is the most common form of non-Hodgkin’s lymphoma in the U.S. Approximately 160,000 people worldwide are diagnosed with DLBCL each year, with comparable incidence rates across regions. Medical practices, including pathological classification, diagnosis, staging, initial treatment and relapse management, are similarly approached worldwide. While it is generally responsive to treatment in the frontline, as many as 40% of people will relapse or have refractory disease, at which time salvage therapy options are limited and survival is short. Improving treatments earlier in the course of the disease and providing much-needed alternative options could help to improve long-term outcomes.

About follicular lymphoma (FL)

FL is the most common slow-growing (indolent) form of non-Hodgkin lymphoma, accounting for about one in five cases. It typically responds well to treatment but is often characterized by periods of remission and relapse. The disease typically becomes harder to treat each time a patient relapses, and early progression can be associated with poor long-term prognosis. It is estimated that more than 110,000 people are diagnosed with FL each year worldwide.

Lunsumio U.S. Indication

Lunsumio (mosunetuzumab-axgb) is a prescription medicine used to treat adults with follicular lymphoma whose cancer has come back or did not respond to previous treatment, and who have already received two or more treatments for their cancer.

It is not known if Lunsumio is safe and effective in children.

The conditional approval of Lunsumio is based on response rate. There are ongoing studies to establish how well the drug works.

What is the most important information I should know about Lunsumio?

Lunsumio may cause Cytokine Release Syndrome (CRS), a serious side effect that is common during treatment with Lunsumio and can also be severe or life-threatening.

Get medical help right away if you develop any signs or symptoms of CRS at any time, including:

fever of 100.4°F (38°C) or higher
chills
low blood pressure
fast or irregular heartbeat
tiredness or weakness
difficulty breathing
headache
confusion
feeling anxious
dizziness or light-headedness
nausea
vomiting
Due to the risk of CRS, you will receive Lunsumio on a "step-up dosing schedule."

The step-up dosing schedule is when you receive smaller "step-up" doses of Lunsumio on Day 1 and Day 8 of your first cycle of treatment
You will receive a higher dose of Lunsumio on Day 15 of your first cycle of treatment
If your dose of Lunsumio is delayed for any reason, you may need to repeat the step-up dosing schedule
Before each dose in Cycle 1 and Cycle 2, you will receive medicines to help reduce your risk of CRS
Your healthcare provider will check you for CRS during treatment with Lunsumio and may treat you in a hospital if you develop signs and symptoms of CRS. Your healthcare provider may temporarily stop or completely stop your treatment with Lunsumio, if you have severe side effects.

What are the possible side effects of Lunsumio?

Lunsumio may cause serious side effects, including:

neurologic problems. Lunsumio can cause serious and life-threatening neurological problems. Your healthcare provider will check you for neurologic problems during treatment with Lunsumio. Your healthcare provider may also refer you to a healthcare provider who specializes in neurologic problems. Tell your healthcare provider right away if you develop any signs or symptoms of neurologic problems during or after treatment with Lunsumio, including:
headache
numbness and tingling of the arms, legs, hands, or feet
dizziness
confusion and disorientation
difficulty paying attention or understanding things
forgetting things or forgetting who or where you are
trouble speaking, reading, or writing
sleepiness or trouble sleeping
tremors
loss of consciousness
seizures
muscle problems or muscle weakness
loss of balance or trouble walking
tiredness
serious infections. Lunsumio can cause serious infections that may lead to death. Your healthcare provider will check you for signs and symptoms of infection before and during treatment. Tell your healthcare provider right away if you develop any signs or symptoms of infection during treatment with Lunsumio, including:
fever of 100.4° F (38° C) or higher
cough
chest pain
tiredness
shortness of breath
painful rash
sore throat
pain during urination
feeling weak or generally unwell
hemophagocytic lymphohistiocytosis (HLH). Lunsumio can cause overactivity of the immune system, a condition called hemophagocytic lymphohistiocytosis. HLH can be life-threatening and has led to death in people treated with Lunsumio. Your health care provider will check you for HLH especially if your CRS lasts longer than expected. Signs and symptoms of HLH include:
fever
enlarged spleen
easy bruising
low blood cell counts
liver problems
low blood cell counts. Low blood cell counts are common during treatment with Lunsumio and can also be serious or severe. Your healthcare provider will check your blood cell counts during treatment with Lunsumio. Lunsumio can cause the following low blood cell counts:
low white blood cell counts (neutropenia). Low white blood cells can increase your risk for infection
low red blood cell counts (anemia). Low red blood cells can cause tiredness and shortness of breath
low platelet counts (thrombocytopenia). Low platelet counts can cause bruising or bleeding problems
growth in your tumor or worsening of tumor related problems (tumor flare). Lunsumio can cause serious or severe worsening of your tumor. Tell your healthcare provider if you develop any of these signs or symptoms of tumor flare during your treatment with Lunsumio:
chest pain
cough
trouble breathing
tender or swollen lymph nodes
pain or swelling at the site of the tumor
Your healthcare provider may temporarily stop or permanently stop treatment with Lunsumio if you develop severe side effects.

The most common side effects of Lunsumio include: tiredness, rash, fever, and headache.

The most common severe abnormal blood test results with Lunsumio include: decreased phosphate, increased glucose, and increased uric acid levels.

Before receiving Lunsumio, tell your healthcare provider about all of your medical conditions, including if you:

have ever had an infusion reaction after receiving Lunsumio
have an infection, or have had an infection in the past which lasted a long time or keeps coming back
have or have had Epstein-Barr Virus
are pregnant or plan to become pregnant. Lunsumio may harm your unborn baby. Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with Lunsumio
Females who are able to become pregnant:

your healthcare provider should do a pregnancy test before you start treatment with Lunsumio
you should use an effective method of birth control (contraception) during your treatment and for 3 months after the last dose of Lunsumio
are breastfeeding or plan to breastfeed. It is not known if Lunsumio passes into your breast milk. Do not breastfeed during treatment and for 3 months after the last dose of Lunsumio
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

What should I avoid while receiving Lunsumio?

Do not drive, operate heavy machinery, or do other dangerous activities if you develop dizziness, confusion, tremors, sleepiness, or any other symptoms that impair consciousness until your signs and symptoms go away. These may be signs and symptoms of CRS or neurologic problems.

These are not all the possible side effects of Lunsumio. Talk to your healthcare provider for more information about the benefits and risks of Lunsumio.

You may report side effects to the FDA at (800) FDA-1088 or View Source You may also report side effects to Genentech at (888) 835-2555.

Please see Important Safety Information, including Serious Side Effects, as well as the Lunsumio full Prescribing Information and Medication Guide or visit View Source

Polivy U.S. Indication

Polivy is a prescription medicine used with other medicines (a rituximab product, cyclophosphamide, doxorubicin, and prednisone) as a first treatment for adults who have moderate to high risk diffuse large B-cell lymphoma (DLBCL), not otherwise specified (NOS) or high-grade B-cell lymphoma (HGBL).

Polivy is a prescription medicine used with other medicines, bendamustine and a rituximab product, to treat DLBCL in adults who have progressed after at least 2 prior therapies.

Important Safety Information

Possible serious side effects

Everyone reacts differently to Polivy therapy, so it’s important to know what the side effects are. Some people who have been treated with Polivy have experienced serious to fatal side effects. Your doctor may stop or adjust your treatment if any serious side effects occur. Be sure to contact your healthcare team if there are any signs of these side effects.

Nerve problems in your arms and legs: This may happen as early as after your first dose and may worsen with every dose. Your doctor will monitor for signs and symptoms, such as changes in your sense of touch, numbness or tingling in your hands or feet, nerve pain, burning sensation, any muscle weakness, or changes to your walking pattern
Infusion-related reactions: You may experience fever, chills, rash, breathing problems, low blood pressure, or hives within 24 hours of your infusion
Low blood cell counts: Treatment with Polivy can cause severe low blood cell counts. Your doctor will monitor your blood counts throughout treatment with Polivy
Infections: If you have a fever of 100.4°F (38°C) or higher, chills, cough, or pain during urination, contact your healthcare team. Your doctor may also give you medication before giving you Polivy, which may prevent some infections
Rare and serious brain infections: Your doctor will monitor closely for signs and symptoms of these types of infections. Contact your doctor if you experience confusion, dizziness or loss of balance, trouble talking or walking, or vision changes
Tumor lysis syndrome: Caused by the fast breakdown of cancer cells. Signs include nausea, vomiting, diarrhea, and lack of energy
Potential harm to liver: Some signs include tiredness, weight loss, pain in the abdomen, dark urine, and yellowing of your skin or the white part of your eyes. You may be at higher risk if you already had liver problems or you are taking other medication
Side effects seen most often

The most common side effects during treatment were

Nerve problems in arms and legs
Nausea
Tiredness or lack of energy
Diarrhea
Constipation
Hair loss
Redness and sores of the lining of the mouth, lips, throat, and digestive tract
Polivy may lower your red or white blood cell counts and increase uric acid levels.

Polivy may not be for everyone. Talk to your doctor if you are

Pregnant or think you are pregnant: Data have shown that Polivy may harm your unborn baby
Planning to become pregnant: Women should avoid getting pregnant while taking Polivy. Women should use effective contraception during treatment and for 3 months after their last Polivy treatment. Men taking Polivy should use effective contraception during treatment and for 5 months after their last Polivy treatment
Breastfeeding: Women should not breastfeed while taking Polivy and for 2 months after the last dose
These may not be all the side effects. Talk to your healthcare provider for more information about the benefits and risks of Polivy treatment.

You may report side effects to the FDA at (800) FDA-1088 or View Source You may also report side effects to Genentech at (888) 835-2555.

Please see the full Prescribing Information and visit View Source for additional Important Safety Information.

Columvi U.S. Indication

Columvi (glofitamab-gxbm) is a prescription medicine to treat adults with certain types of diffuse large B-cell lymphoma (DLBCL) or large B-cell lymphoma (LBCL) that has come back (relapsed) or that did not respond to previous treatment (refractory), and who have received 2 or more prior treatments for their cancer.

It is not known if Columvi is safe and effective in children.

The conditional approval of Columvi is based on response rate and durability of response. There are ongoing studies to establish how well the drug works.

What is the most important information I should know about Columvi?

Columvi can cause Cytokine Release Syndrome (CRS), a serious side effect that is common during treatment with Columvi, and can also be serious and lead to death.

Call your healthcare provider or get emergency medical help right away if you develop any signs or symptoms of CRS, including:

fever of 100.4°F (38°C) or higher
chills or shaking
fast or irregular heartbeat
dizziness or light-headedness
trouble breathing
shortness of breath
Due to the risk of CRS, you will receive Columvi on a "step-up dosing schedule".

A single dose of a medicine called obinutuzumab will be given to you on the first day of your first treatment cycle (Day 1 of Cycle 1).
You will start the Columvi step-up dosing schedule a week after the obinutuzumab dose. The step-up dosing schedule is when you receive smaller "step-up" doses of Columvi on Day 8 and Day 15 of Cycle 1. This is to help reduce your risk of CRS. You should be hospitalized during your infusion and for 24 hours after receiving the first step-up dose on Day 8. You should be hospitalized during your infusion and for 24 hours after receiving the second step-up dose on Day 15 if you experienced CRS during the first step-up dose.
You will receive your first full dose of Columvi a week after the second step-up dose (this will be Day 1 of Cycle 2).
If your dose of Columvi is delayed for any reason, you may need to repeat the "step-up dosing schedule".
If you had more than mild CRS with your previous dose of Columvi, you should be hospitalized during and for 24 hours after receiving your next dose of Columvi.
Before each dose of Columvi, you will receive medicines to help reduce your risk of CRS and infusion-related reactions.
Your healthcare provider will monitor you for CRS during treatment with Columvi and may treat you in a hospital if you develop signs and symptoms of CRS. Your healthcare provider may temporarily stop or completely stop your treatment with Columvi if you have severe side effects.
Carry the Columvi Patient Wallet Card with you at all times and show it to all of your healthcare providers. The Columvi Patient Wallet Card lists the signs and symptoms of CRS you should get emergency medical help for right away.

What are the possible side effects of Columvi?

Columvi may cause serious side effects, including:

Cytokine Release Syndrome.
Neurologic problems. Columvi can cause serious neurologic problems that may lead to death. Your healthcare provider will monitor you for neurologic problems during treatment with Columvi. Your healthcare provider may also refer you to a healthcare provider who specializes in neurologic problems. Tell your healthcare provider right away if you develop any signs or symptoms of neurologic problems, including:
headache
confusion and disorientation
difficulty paying attention or understanding things
trouble speaking
sleepiness
memory problems
numbness, tingling, or weakness of the hands or feet
dizziness
shaking (tremors)
Serious Infections. Columvi can cause serious infections that may lead to death. Your healthcare provider will monitor you for signs and symptoms of infection and treat you as needed. Tell your healthcare provider right away if you develop any signs of an infection, including: fever, chills, weakness, cough, shortness of breath, or sore throat.
Growth in your tumor or worsening of tumor related problems (tumor flare).
Tell your healthcare provider if you get any of these signs or symptoms of tumor flare:

tender or swollen lymph nodes
pain or swelling at the site of the tumor
chest pain
cough
trouble breathing
The most common side effects of Columvi include: CRS, muscle and bone pain, rash, and tiredness.

The most common severe abnormal lab test results with Columvi include: decreased white blood cells, decreased phosphate (an electrolyte), increased uric acid levels, and decreased fibrinogen (a protein that helps with blood clotting).

Your healthcare provider may temporarily stop or completely stop treatment with Columvi if you develop certain side effects.

Before receiving Columvi, tell your healthcare provider about all of your medical conditions, including if you:

have an infection
have kidney problems
are pregnant or plan to become pregnant. Columvi may harm your unborn baby
Females who are able to become pregnant:
Your healthcare provider should do a pregnancy test before you start treatment with Columvi.
You should use effective birth control (contraception) during treatment and for 1 month after your last dose of Columvi. Talk to your healthcare provider about what birth control method is right for you during this time.
Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with Columvi.
are breastfeeding or plan to breastfeed. Columvi may pass into your breast milk. Do not breastfeed during treatment and for 1 month after your last dose of Columvi.
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

What should I avoid while receiving Columvi?

Do not drive, operate heavy machinery, or do other dangerous activities if you develop dizziness, confusion, shaking (tremors), sleepiness, or any other symptoms that impair consciousness until your signs and symptoms go away. These may be signs and symptoms of neurologic problems.

These are not all the possible side effects of Columvi. Talk to your health care provider for more information about the benefits and risks of Columvi.

You may report side effects to the FDA at (800) FDA-1088 or View Source You may also report side effects to Genentech at (888) 835-2555.

Please see Important Safety Information, including Serious Side Effects, as well as the Columvi full Prescribing Information and Medication Guide or visit View Source

(Press release, Genentech, DEC 8, 2025, View Source [SID1234661297])

On December 8, 2025 Amphista Therapeutics ("the Company" or "Amphista"), a leader in the discovery of next generation targeted protein degradation (TPD) medicines, reported that full details of its new preclinical data with its lead Targeted Glue AMX-883, an orally bioavailable, potent and selective degrader of BRD9 were presented on 6 December during the 67th American Society of Haematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in Orlando, Florida.

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The compelling findings support the potential of AMX-883 as a first-line treatment option in the earlier disease setting of acute myeloid leukaemia (AML), one of the most aggressive blood cancers where 5-year survival rates remain at just 33% and where resistance to standard of care treatments like venetoclax remains a major clinical challenge. Based on these data, the Company will advance AMX-883 as a karyotype-independent, pro-differentiation agent into the clinic for AML in H2 2026.

The key data presented at ASH (Free ASH Whitepaper) showed that:

AMX-883 demonstrates potent degradation as a monotherapy and synergistic benefit in combination with venetoclax

AMX-883 (30mg/kg twice daily and 100mg/kg once daily) significantly reduced cancer growth by achieving picomolar potency degradation of BRD9 across a panel of AML cell lines and significantly reduced leukemic burden in bone marrow and blood in vivo, compared to venetoclax alone.
Synergistic efficacy was observed when each dose of AMX-883 was combined with venetoclax (75mg/kg once daily), leading to significant blocking of tumour growth at therapeutically relevant concentrations.
AMX-883 prevents resistance to venetoclax when dosed in combination

The combination of AMX-883 with venetoclax in a venetoclax resistant cell line, prevented the emergence of resistance, and cells had comparable sensitivity to venetoclax from their first exposure.
AMX-883 actively degrades BRD9 in venetoclax resistant cells, prevents upregulation of key resistance markers, including MCL-1 and BCL-2, and increases levels of cell death markers.
Martin Pass, Chief Development Officer at Amphista Therapeutics said, "The important preclinical data presented for the first time show that our Targeted Glue AMX-883, a selective degrader of BRD9, extends the durability and efficacy of combination therapy with venetoclax and prevents AML cancer cells from becoming resistant to venetoclax. We are very pleased to receive positive feedback from experts in the field on this compelling profile, which gives us strong reason to believe that AMX-883 could be a viable treatment option for patients with AML. We look forward to commencing the first clinical trial for this devastating blood cancer in H2 2026."

To deliver the first clinical study of AMX-883, Amphista has built its development capabilities with three key strategic leadership appointments, including Dr. Lisa Butler, Senior Vice President of Clinical Operations who was previously Head of Study Leadership, early Oncology Clinical at AstraZeneca.

The abstract presented at ASH (Free ASH Whitepaper) is available on the congress website.

(Press release, Amphista Therapeutics, DEC 8, 2025, View Source [SID1234661249])

On December 8, 2025 MaaT Pharma (EURONEXT: MAAT – the "Company"), a clinical-stage biotechnology company and a leader in the development of Microbiome Ecosystem TherapiesTM (MET) dedicated to enhancing survival for patients with cancer through immune modulation, reported that Prof. Malard, MD, PhD, hematology professor at Saint-Antoine Hospital and Sorbonne University and ARES Trial lead investigator, presented the results for the pivotal ARES, single arm, open label trial evaluating MaaT013 (Xervyteg) in aGvHD during an oral session at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition currently taking place in Orlando, Florida, USA. In addition, the Company announced new data from the pivotal ARES trial including a 1-year overall survival rate of 54%, confirming the global clinical benefit of MaaT013 (Xervyteg).

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"These results confirm that MaaT013 (Xervyteg) offers a durable clinical benefit for patients with GI-aGvHD who have exhausted all currently approved treatment options. Achieving a 62% gastrointestinal response at Day 28, maintaining responses over time, and reaching a 54% one-year overall survival represent a meaningful step forward in addressing this critical unmet need," said Prof. Florent Malard, MD, PhD, Professor of Hematology at Saint-Antoine Hospital and Sorbonne University, and lead investigator of the ARES trial who presented the findings.

Prof. Malard detailed primary and secondary endpoints, noting that GI-Overall Response Rate (GI-ORR) at Day 28 (62% including 38% of complete response) remains high over time, indicating a durable response with a GI-ORR of 47% and all-organ ORR of 45% at Day 56. At three months, GI-ORR and all-organ ORR were both still high at 44%. These results indicate that responses to MaaT013 (Xervyteg) are durable and result in improved survival outcomes, which translates into a 54% 1-year overall survival rate in the study population.

Final efficacy data of MaaT013 (Xervyteg) in the ARES study are summarized below.
The ARES trial is a single-arm, open label trial evaluating MaaT013 (Xervyteg) as third-line treatment in 66 adult patients with severe GI-aGvHD refractory to corticosteroids and ruxolitinib across 50 sites in six European countries:

Patient profile:

91% (n=60) presented with Grade III–IV aGvHD with GI involvement
86% (n=57) were steroid-resistant and 14% (n=9) steroid-dependent; all were refractory to ruxolitinib
Male: 53%, Female: 47%
Final results:

GI-ORR at Day 28 occurred in 41/66 patients (62%) and prevalently consisted of complete response (CR) (38%, 25/66 patients), and very good partial response (VGPR) (20%, 13/66 patients).
All-organ ORR at Day 28 occurred in 42/66 patients (64%) patients and was similarly driven by high rates of CR (36%, 24/66 patients) and VGPR (18%, 12/66 patients).
GI-ORR at Day 56 was maintained at 47% (31/ 66 patients) and prevalently consisted of CR (35%, 23/66 patients).
All-organ ORR at Day 56 was 45% (30/66 patients) and prevalently consisted of CR (35%, 23/66 patients).
GI-ORR and all-organ ORR at 3 months were both 44% (29/ 66 patients), with a prevalence of CR (36%, 24/66 patients).
Overall survival (OS) at 12 months was 54% (median survival not reached), this confirms the 12-month probability of survival of 54% announced in January 2025 for the topline results.
Median overall survival was not reached, indicating that more than half of the patients were still alive at the end of the study. This suggests a durable survival benefit and reinforces the strong efficacy signal observed in the pivotal ARES study. The median OS of responders was not reached, while it was only 54 days for non-responders.
The OS was significantly higher in patients who had a GI response at Day 28 than those who did not respond: 68% vs 28% respectively (p <0.0001), indicating a strong association between early GI response and improved survival in refractory GI-aGvHD.
Safety data showed that MaaT013 (Xervyteg) was associated with an acceptable tolerability profile in severe aGvHD patient population (as reviewed continuously by a Data and Safety Monitoring Board).
The pivotal ARES trial results will soon be submitted for publication in a leading peer-reviewed medical journal. MaaT013 (Xervyteg) is currently under review by the European Medicines Agency (EMA) following the submission of a marketing authorization application in June 2025, with a decision expected in mid- 2026, as previously announced. If approved, MaaT013 (Xervyteg) would become the first microbiotherapy in oncology in the world and the first 3rd line therapy in aGvHD addressing a critical unmet need.

(Press release, MaaT Pharma, DEC 8, 2025, View Source [SID1234661265])