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])

On December 8, 2025 Sumitomo Pharma America, Inc. (SMPA) reported new clinical data supporting further development of enzomenib, an investigational, oral selective menin inhibitor being researched for the treatment of relapsed or refractory acute leukemia, and nuvisertib, an oral investigational highly selective small molecule PIM1 kinase inhibitor, being researched for the treatment of relapsed or refractory myelofibrosis (MF), at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition.

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Updated Data Shared with the Use of Enzomenib in Acute Myeloid Leukemia
Updated preliminary data were presented from the ongoing Phase 1/2 study of enzomenib monotherapy, which as of October 4, 2025, included 116 total patients with acute leukemia, most of whom (93.1%, 108/116) had acute myeloid leukemia (AML) with a median of two prior regimens. Genomic abnormalities of leukemia subtypes including KMT2A rearrangement (KMT2Ar) were documented in 61 patients (52.6%), NPM1 mutation (NPM1m) in 34 patients (29.3%), and other HOXA9/MEIS1-driven abnormalities in 21 patients (17.7%).

Enzomenib was escalated from 40 mg twice a day (BID) to 400 mg BID with no dose-limiting toxicities (DLTs). Treatment-related adverse events (TRAEs) observed in at least 10% of patients were nausea (16.4%), and vomiting (11.2%). Differentiation syndrome (DS) was reported in 12.9% of patients and did not result in patient deaths, study discontinuations, or dose reductions of enzomenib. No treatment-related deaths were observed in the study. Dose-dependent increases in exposure were observed, particularly at doses greater than 140 mg BID. Little to no drug accumulation was observed, and CYP3A4 inhibitor azoles did not have a significant impact on exposure.

In patients with KMT2Ar, dose optimization of 200, 300, and 400 mg BID is complete, and the RP2D has been determined as 300 mg BID. At RP2D, in patients with KMT2Ar who had not received prior treatment with a menin inhibitor (n = 15), the objective response rate (ORR) was 73.3% and CR+CRh was 40%. Across the optimization dose levels, the duration of CR+CRh (n = 11) was 12.5 months and in all optimization patients (n = 39) median overall survival (mOS) was 11.8 months.

For patients with NPM1m AML, dose optimization is ongoing at 200, 300, and 400 mg BID with a focus on 200 and 300 mg BID in patients who have not received a prior menin inhibitor. In the NPM1m dose optimization population (n=25, pts who received ≥ 200 mg BID), ORR is 52% and CR+CR is 44% with a duration of CR+CRh of 5.7 months. The mOS was 8.5 months.

"Despite improved understanding of the genetic factors of certain high-risk subtypes in acute leukemias, poor prognosis for patients remains a significant unmet need," said Naval G. Daver, M.D., professor and director of the Leukemia Research Alliance Program in the Department of Leukemia at MD Anderson Cancer Center in Houston. "The data from this ongoing Phase 1/2 study continue to show that enzomenib exhibits promising clinical activity, with encouraging overall and complete response rates, duration of response, and no significant drug interactions with azoles in patients with relapsed or refractory KMT2Ar or NPM1m AML. As an intentionally designed oral therapy to inhibit menin and KMT2A protein interaction, these encouraging clinical results combined with a promising safety profile support the potential of enzomenib as a therapeutic option for relapsed or refractory acute leukemia patients with KMT2A-rearranged or NPM1-mutated subtypes of the disease."

Also presented were preliminary results of a Phase 1 study of enzomenib at dose levels of 140 mg, 200 mg, and 300 mg BID in combination with azacitidine and venetoclax (VEN/AZA) in patients with relapsed or refractory AML with KMT2Ar or NPM1m. VEN was administered on days 1-14 of a 28-day study cycle, AZA on days 1-7, and enzomenib was administered on days 1-28 with and without azole antifungal agents.

A total of 40 patients were enrolled, of which 18 had KMT2Ar (45%) and 22 (55%) had NPM1m. The median number of prior regimens was 2, with 15 patients having received prior venetoclax (37.5%) and 11 patients (27.5%) a prior menin inhibitor.

There were no DLTs observed in the 40 patients enrolled. Hematologic TRAEs related to any regimen component observed in at least 15% of patients included thrombocytopenia (45%), leukopenia (35%), neutropenia (30%), anemia (22.2%), and lymphopenia (15%). Non-hematologic TRAEs were nausea (25%), diarrhea (20%), and AST increased and constipation (15% each). Any-cause QT interval prolongation was reported in 4 patients (10%) with no grade 3 or higher events; no cases were considered related to enzomenib. There were also 4 events of non-serious DS with 0 events grade 3 or higher. Pharmacokinetic data indicated that there were no significant drug-drug interactions between enzomenib and VEN.

As of the clinical data cutoff on October 4, 2025, clinical activity data is available for 26 of the 40 total patients as 11 patients were still in Cycle 1 (n = 9) or Cycle 2 (n = 2), and 3 patients had cutaneous leukemia without measurable disease in the bone marrow (bone marrow blasts <5%).

Promising preliminary clinical activity has been observed, particularly in patients without prior VEN or menin inhibitor exposure (N=13). The ORR is 85% (11/13) and the composite complete remission (CRc) rate is 62% (8/13). Local MRD assessments were available in 9 patients and 7/9 (78%) achieved MRD negativity as of the cut-off.

These data support evaluating enzomenib with VEN/AZA in patients with newly diagnosed AML. Study arms are being added to investigate the combination regimen for patients with newly diagnosed disease. Enzomenib will be administered at 200 mg or 300 mg BID using VEN/AZA administered according to the FDA label. Enrollment of newly diagnosed patients with KMT2Ar or NPM1m AML will begin in early 2026.

Nuvisertib in Patients with Relapsed or Refractory (R/R) MF
For the first time, clinical data were presented from the ongoing global Phase 1/2 study evaluating the safety and efficacy of nuvisertib in combination with momelotinib (MMB) in 18 patients with R/R MF with anemia. All enrolled patients in the study had previously been treated with a JAK inhibitor, the current standard of care for patients with MF, and 61% of patients had high molecular risk mutation. Preliminary data showed that the treatment with nuvisertib and MMB combination appeared well tolerated, with early clinical activity observed, including ³50% total symptom score reduction (TSS50 response) in 58% of patients with an absolute reduction in all individual symptoms, a spleen volume reduction ³ 25% (SVR25 response) in 50% of patients, anemia improvement, and cytokine modulation in patients with relapsed or refractory MF with anemia. These preliminary data, collected as of October 15, 2025, support further development of nuvisertib in combination with MMB as a potential treatment option for patients with MF.

Additionally, data presented from the ongoing global Phase 1/2 study of nuvisertib in patients with relapsed or refractory MF (N=77) showed that nuvisertib monotherapy continued to be well tolerated with no DLTs and limited myelosuppression. The results showed that treatment with nuvisertib monotherapy led to SVR25 response in 20% of patients, TSS50 response in 45% of patients with absolute reduction in all individual symptoms. Data also showed that treatment with nuvisertib resulted in significant cytokine modulation over time, correlating with spleen and symptom responses and one-year overall survival rate of 81%.

"Patients living with myelofibrosis with anemia usually have a dismal prognosis, still continue to face limited treatment options," said John Mascarenhas, M.D., Director of the Center of Excellence for Blood Cancers and Myeloid Disorders, Icahn School of Medicine at Mount Sinai, New York. "The promising preliminary results from the combination of nuvisertib and momelotinib underscore the urgent need for new therapeutic approaches that may offer meaningful clinical benefits to a difficult to treat disease."

"We are excited to present the first-ever myelofibrosis data with a momelotinib-based combination, specifically nuvisertib in combination with momelotinib. The development of innovative therapies—both alone and in combination with other treatments—are critical for physicians and patients with blood cancers such as AML or MF who are in urgent need of new effective therapies," said Jatin Shah, M.D., Chief Medical Officer, Oncology, SMPA. "Based on these updated preliminary data presented at ASH (Free ASH Whitepaper), which continue to show promising clinical activity and safety profiles for both enzomenib and nuvisertib, we remain committed to accelerate the clinical development in these programs with the ultimate goal of improving patient outcomes."

About Leukemia
Leukemia is a type of cancer that forms in blood-forming tissue, characterized by the uncontrolled growth of blood cells, usually white blood cells, in the bone marrow. Acute leukemia, a form of leukemia, requires immediate treatment as blood cells multiply rapidly leading to a sudden onset of symptoms.1 Approximately 30% of patients with AML have NPM1 mutations,2 and 5%-10% of patients with AML have KMT2A rearrangements.3

About Myelofibrosis (MF)
MF is a rare type of blood cancer that is characterized by the buildup of fibrous tissue in the bone marrow, which can affect the production of blood cells. This buildup is caused by dysregulation in the JAK signaling pathway. MF is a serious and rare disease with 0.7 new cases per 100,000 people worldwide each year.4

About Enzomenib (DSP-5336)
Enzomenib is an investigational, oral, small molecule inhibitor of the menin and Lysine (K)-specific methyltransferase 2A (KMT2A) protein interaction, a key interaction in acute leukemia and other tumor cell proliferation and growth. Menin is a scaffold nuclear protein, which plays key roles in gene expression and protein interactions involved in many biological pathways, including cell growth, cell cycle, genomic stability, and hematopoiesis.5,6 In preclinical studies, enzomenib has shown selective growth inhibition in human acute leukemia cell lines with KMT2A rearrangements or NPM1 mutations.5,7 Enzomenib reduced the expression of the leukemia-associated genes HOXA9 and MEIS1 and increased the expression of the differentiation gene CD11b in human acute leukemia cell lines with KMT2A rearrangements and NPM1 mutation.8,9 The safety and efficacy of enzomenib is currently being clinically evaluated in a Phase 1/2 dose-escalation/dose-expansion study in patients with relapsed or refractory acute leukemia (NCT04988555). Additionally, the registrational Phase 2 Horizen-1 R/R mono AML/ALL (KMT2Ar + NPM1m) study is now open for enrollment. The FDA granted Orphan Drug Designation for enzomenib for the indication of acute myeloid leukemia in June 2022. The FDA granted Fast Track Designation for enzomenib for the indication of relapsed or refractory acute myeloid leukemia with KMT2Ar or NPM1m in June 2024. Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) granted Orphan Drug Designation for enzomenib for the indication of relapsed or refractory acute myeloid leukemia with KMT2Ar or NPM1m in September 2024.

About Nuvisertib (TP-3654)
Nuvisertib is an oral investigational selective inhibitor of PIM1 kinase, which has shown potential antitumor and antifibrotic activity through multiple pathways, including induction of apoptosis in preclinical models.10,11 Nuvisertib was observed to inhibit proliferation and increase apoptosis in murine and human hematopoietic cells expressing the clinically relevant JAK2 V617F mutation.10 Nuvisertib alone and in combination with ruxolitinib showed white blood cell and neutrophil count normalization and reduced spleen size and bone marrow fibrosis in JAK2 V617F and MPLW515L murine models of myelofibrosis.11 The safety and efficacy of nuvisertib is currently being clinically evaluated in a Phase 1/2 study in patients with intermediate- and high-risk myelofibrosis (NCT04176198). The FDA granted Orphan Drug Designation to nuvisertib for the indication of myelofibrosis in May 2022. The Japan Ministry of Health, Labour and Welfare (MHLW) granted Orphan Drug Designation to nuvisertib for the treatment of myelofibrosis in November 2024. The FDA granted Fast Track Designation to nuvisertib for the indication of myelofibrosis in July 2025.

(Press release, Sumitomo Pharmaceuticals, DEC 8, 2025, View Source [SID1234661282])

On December 8, 2025 Qihan Biotech, a clinical-stage biotechnology company developing off-the-shelf cell therapies for autoimmune diseases and cancer, including allogeneic CAR-T and in vivo CAR-T therapies, reported an oral and several poster presentations at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. The data included encouraging clinical results with the company’s allogeneic CAR-T program and foundational preclinical work supporting its emerging in vivo CAR-T program.

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Qihan’s oral presentation highlighted clinical data from 20 patients enrolled in three investigator-initiated trials evaluating QT-019B in multiple autoimmune diseases. Treatment was well tolerated, with no events exceeding Grade 1 CRS and no reported ICANS or serious infections.

Across multiple severe autoimmune indications, QT-019B demonstrated clinical benefit in patients dosed at therapeutically relevant levels (n=19), with one subtherapeutic-dose patient excluded from the efficacy summary.

SLE-ITP (n=6): 5 complete responses (CR) with normalized platelet counts and 1 partial response (PR)
APS-associated ITP (n=5): 4 CR with normalized platelet counts
AIHA (n=3): 2 CR with normalized hemoglobin
SLE (n=2): 1 DORIS remission and 1 SRI-4 response at Month 2
Neurological autoimmune diseases (NMOSD and MS; n=3): 2 EDSS stabilization; 1 too soon to assess
Pharmacokinetic and pharmacodynamic readouts reinforce the potential of Qihan’s immune-privileged platform to deliver durable, systemic immune reset.

Robust expansion of allogeneic CAR-T cells observed in 17 of 19 patients treated at therapeutic dose levels
Deep and sustained depletion of pathogenic autoantibodies, consistent with the intended immune-reset mechanism
In MS, early CSF analyses showed rapid and durable clearance of oligoclonal bands (OCB) and kappa free light chains (kFLC)
Qihan’s QT-019B program is supported by IND clearance for rSLE from both the US FDA and the China NMPA. The FDA has also granted Fast Track Designation for the SLE-ITP indication. Phase 1 enrollment is actively underway.

Qihan’s unique "immune-privileged" platform – built on the company’s world-leading capabilities in multiplexable gene editing and deep understanding of transplantation immunology – enables creation of next-generation cells capable of evading immune attack. The platform is uniquely designed to address the biological and translational barriers that have limited progress across the CAR-T field. A primary barrier is immune rejection, where host T and NK cells rapidly eliminate donor cells, limiting expansion and persistence, and preventing cells from surviving long enough to achieve durable clinical benefit. Another major challenge is the need for lymphodepletion, which is associated with organ damage and broad toxicities. Qihan is developing the next generation product QT-019C, which aims to overcome both immune rejection and the dependence on lymphodepletion. Across its oral and poster sessions, the company has demonstrated scientific rigor, clinical relevance, and platform scalability that collectively address these critical obstacles.

"Autologous therapies have shown what cell therapy can achieve, but they are not scalable for autoimmune disease. Efforts to develop off-the-shelf allogeneic approaches have been challenging due to immune rejection, limited expansion and persistence, and the need for lymphodepletion. Our data show a different path," said Luhan Yang, Ph.D., Chief Executive Officer of Qihan Biotech. "Our first-generation program demonstrates strong safety, robust in vivo expansion, meaningful efficacy, and evidence of immune reset. Our next-generation product, which is now in human testing in an IIT, goes further. By driving deeper hypoimmunity and enhanced expansion and persistence, it provides the possibility to eliminate lymphodepletion. We believe we are overcoming the key barriers that have constrained the field and opening the door to broadly accessible, off-the-shelf cell therapies."

Details of the oral and posters being presented are as follows:

Designing immune-evasive UCAR-T cells to overcome allogeneic barriers and advance off-the-shelf immunotherapy
Publication Number: 1045 (Oral Session)
Session Title: 711. Cell Collection and Manufacturing of HSPCs, CAR-T Cells, and Other Cellular Therapy Products: Refining CAR-T Cells and Engineered HSPCs; New Approaches to HSPC mobilization
Session Date & Time: December 8, 2025, at 4:30PM – 4:45 PM EST

Engineering Quiescent Viral Entry Pathways for in Vivo CAR-T Generation via Binder-Fusogen Combinatorics
Publication Number: 2404
Session Title: 711. Cell Collection and Manufacturing of HSPCs, CAR-T Cells, and Other Cellular Therapy Products: Poster I
Session Date & Time: December 6, 2025, at 5:30PM – 7:30 PM EST

Cytokine receptor armored CAR-T cells enable robust T cell expansion and function in the absence of lymphodepletion
Publication Number: 4104
Session Title: 702. CAR-T Cell Therapies: Basic and Translational: Poster II
Session Date & Time: December 7, 2025, at 6:00PM – 8:00 PM EST

Enhanced vsvg variants for binder‑dependent and high‑specificity transduction of primary T cells
Publication Number: 4181
Session Title: 711. Cell Collection and Manufacturing of HSPCs, CAR-T Cells, and Other Cellular Therapy Products: Poster II
Session Date & Time: December 7, 2025, at 6:00PM – 8:00 PM EST

Rapid normalization of platelet counts in patients with refractory thrombocytopenia associated with systemic lupus erythematosus (SLE) following treatment with allogeneic dual-target CD19/BCMA CAR T-cell therapy (QT-019B)
Publication Number: 3031
Session Title: 311. Disorders of Platelet Number or Function: Clinical and Epidemiological: Poster II
Session Date & Time: December 7, 2025, at 6:00PM – 8:00 PM EST

Allogeneic Dual-Target CD19/BCMA CAR T-Cell Therapy (QT-019B) for Refractory Autoimmune Hemolytic Anemia
Publication Number: 5922
Session Title: 704. Cellular Immunotherapies: Early Phase Clinical Trials and Toxicities: Poster III
Session Date & Time: December 8, 2025, at 6:00PM – 8:00 PM EST

About QT-019B
QT-019B is Qihan Biotech’s first-generation allogeneic CAR-T therapy designed to treat severe autoimmune diseases. Engineered using Qihan’s immune-privileged platform, QT-019B incorporates multiplex gene edits to enhance expansion, persistence, and resistance to immune rejection. QT-019B has demonstrated encouraging safety and clinical activity in multiple investigator-initiated trials and has been cleared by US FDA and China NMPA for further clinical development. QT-019B has received FDA Fast Track Designation for SLE-ITP.

QT-019B has been evaluated in the following autoimmune diseases:
SLE: Systemic Lupus Erythematosus
SLE-ITP: Systemic Lupus Erythematosus–associated Immune Thrombocytopenia
APS-ITP: Antiphospholipid Syndrome–associated Immune Thrombocytopenia
AIHA: Autoimmune Hemolytic Anemia
MS: Multiple Sclerosis
NMOSD: Neuromyelitis Optica Spectrum Disorder

(Press release, Qihan Biotech, DEC 8, 2025, View Source [SID1234661298])