On December 5, 2025 K36 Therapeutics, Inc. ("K36"), a clinical-stage biotechnology company developing first-in-class MMSET/NSD2 inhibitors for genetically defined cancers, reported that clinical data from its lead program, gintemetostat (KTX-1001) will be presented at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, taking place December 6 -9, 2025, in Orlando, Florida. The oral presentation will feature the results from the dose escalation part of the Phase 1 MMSET study NCT05651932.

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"I’m looking forward to sharing the clinical progress of gintemetostat (KTX-1001), a potent oral NSD2/MMSET inhibitor being developed for patients with t(4;14) multiple myeloma," said Saad Usmani, M.D., MBA, Chief of Myeloma Service at Memorial Sloan Kettering Cancer Center. "In the dose-escalation phase, gintemetostat monotherapy showed a favorable safety and tolerability profile and demonstrated disease control and efficacy. Pharmacodynamic data confirm target engagement, and we look forward to advancing into the dose-expansion phase to evaluate combinations with proteasome inhibitors, IMiDs, and next-generation CELMoDs such as mezigdomide."

In parallel with this important clinical milestone, K36 is continuing to strengthen its leadership team to support the advancement of its expanding clinical pipeline. The company also announced the appointment of Shinta Cheng, M.D., Ph.D., a seasoned clinical leader with more than 20 years oncology and hematology drug development experience, including in prostate cancer and multiple myeloma, as its new Chief Medical Officer.

"We are highly encouraged by the durable disease control achieved with oral, single-agent gintemetostat, and by the strength of our emerging clinical data, including patients who continue to experience long-standing benefit while remaining on monotherapy," said Dr. Cheng, Chief Medical Officer of K36 Therapeutics. "Gintemetostat is a first-in-class MMSET inhibitor with a novel mechanism of action and is being evaluated in combination with both standard and next-generation therapies. Its favorable safety and tolerability profile further support its potential as a foundational therapy for patients with t(4;14) multiple myeloma. We remain deeply grateful to the investigators, patients, and partners whose commitment enables our rapid clinical progress."

Dr. Cheng was most recently vice president, clinical development at SpringWorks Therapeutics, where he led clinical collaborations combining nirogacestat with therapies targeting BCMA in multiple myeloma as well as global registration programs for nirogacestat in desmoid tumors and mirdametinib in plexiform neurofibromas in NF1 patients. Previously, he led development of apalutamide and niraparib in prostate cancer at Johnson & Johnson Innovative Medicines. He was also Asia-Pacific immuno-oncology development lead for nivolumab and ipilimumab at Bristol-Myers Squibb, where he advanced early clinical studies of dasatinib and first-in-human androgen receptor antagonists in prostate cancer. Board-certified in internal medicine, hematology, and medical oncology, he trained at Beth Israel Deaconess Medical Center, Harvard Medical School. He holds an M.D. and Ph.D. in viral oncogenesis from the University of Rochester School of Medicine.

Oral presentation details are outlined below:

Title: Phase 1 study of KTX-1001, a first-in-class oral MMSET/NSD2 inhibitor, demonstrates clinical activity in relapsed/refractory multiple myeloma
Date & Time: December 6, 2025, 2:45 PM – 3:00 PM EST
Location: Orange County Convention Center – West Hall D1

"We are fortunate to welcome Shinta to K36 as we enter a period of significant clinical expansion," said Terry Connolly, Ph.D., Chief Executive Officer of K36 Therapeutics. "As we advance two first-in-class NSD2 inhibitor programs across multiple myeloma and prostate cancer, Shinta’s deep expertise in both diseases and his proven track record guiding therapies from early development through registration, comes at exactly the right moment. With our prostate cancer program now initiated and our multiple myeloma program expanding, his leadership will be pivotal in shaping this next phase of growth and strengthening our position in the emerging field of cancer epigenetics."

The full abstracts can be found at the ASH (Free ASH Whitepaper) Annual Meeting website at www.Hematology.org.

About Gintemetostat (KTX-1001)
Gintemetostat (KTX-1001) is a novel, first-in-class, potent, and selective inhibitor of the methyltransferase activity of MMSET/NSD2. It is an orally administered small molecule being developed for the treatment of relapsed and refractory multiple myeloma, with an initial focus on patients harboring the t(4;14) translocation. By targeting the underlying epigenetic driver of this high-risk subset, gintemetostat offers a promising avenue for patients with limited treatment options.

About Multiple Myeloma
Multiple myeloma (MM) is the second most common hematologic malignancy, driven by the uncontrolled proliferation of plasma cells in the bone marrow. According to the American Cancer Society, approximately 36,000 new cases are diagnosed each year. While recent therapeutic advances have extended survival, MM remains incurable, and most patients eventually relapse. High-risk MM, defined by genetic abnormalities such as t(4;14) and other adverse prognostic markers, is associated with aggressive disease biology, shorter survival, and limited benefit from standard-of-care regimens. Addressing this high-risk population represents one of the greatest unmet needs in myeloma research and treatment.

About the KTX-1001 Phase 1 MMSET Clinical Trial
The Phase 1 clinical trial is a single-arm, open-label study in participants with relapsed and refractory multiple myeloma. It is a multi-part study with dose escalation followed by an expansion cohort in patients with the genetic translocation t(4;14) to evaluate the safety, tolerability, and preliminary efficacy of different doses of KTX-1001 in combination with standard of care and mezigdomide. For more information and participating centers, visit NCT05651932 and EUCTR: 2022-500801-41-00.

(Press release, K36 Therapeutics, DEC 5, 2025, View Source [SID1234661176])

On December 5, 2025 Johnson & Johnson (NYSE:JNJ) reported that new data from the investigational Cohort 4 of the Phase 2b SunRISe-1 study show treatment with gemcitabine intravesical system resulted in high one-year disease-free survival (DFS), progression-free survival (PFS), and overall survival (OS) rates in patients with Bacillus Calmette-Guérin (BCG)-unresponsive, high-risk, papillary-only non-muscle invasive bladder cancer (NMIBC).1 These data were featured as a late-breaking oral presentation at the Society of Urologic Oncology (SUO) 2025 Annual Meeting and build upon data presented at the 2025 American Urological Association (AUA) Annual Meeting.

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"The findings are meaningful, as the majority of patients remained free of cancer recurrence at one year despite having papillary tumors that carry a high risk for recurrence and a significant risk of progression to a more aggressive, muscle-invasive stage of disease," said Siamak Daneshmand*, M.D., Professor of Urology, University of Southern California, and presenting author. "Bladder removal has traditionally been the primary path forward for these patients, a life-altering procedure that can have a significant impact on a patient’s quality of life."

"At Johnson & Johnson, we are committed to developing innovative treatments for patients with high-risk NMIBC who have few options beyond life-altering surgery," said Christopher Cutie, M.D., Vice President, Disease Area Leader, Bladder Cancer, Johnson & Johnson Innovative Medicine. "Those with papillary-only disease face particularly difficult decisions, as surgical removal of the bladder has long been the standard of care for patients who are unresponsive or resistant to BCG."

Cohort 4 of the Phase 2b SunRISe-1 study focused on 52 patients with papillary-only, high-risk NMIBC whose disease did not respond or stopped responding to BCG therapy and who were ineligible for or declined radical cystectomy. The therapy was administered every three weeks for six months, followed by every 12 weeks for up to an additional 18 months, to evaluate its potential to prevent the recurrence or progression of high-grade papillary tumors.1 The results support continued evaluation in the ongoing Phase 3 SunRISe-5 study (NCT06211764) comparing gemcitabine intravesical system to chemotherapy in patients with previously BCG-treated, papillary-only NMIBC.

At median follow-up of 15.9 months (range, 4-20 months), the one-year DFS rate was 74.3 percent (95 percent confidence interval [CI], 59.2-84.6), meaning nearly three out of four patients remained free from cancer recurrence. Results were similar across patients with high-grade Ta and T1 papillary tumors, 74.8 percent and 74.1 percent, respectively (95 percent CI, 54.3-87.1 and 48.5-88.3). At one year, PFS was 95.6 percent (95 percent CI, 83.5-98.9) and OS was 98 percent (95 percent CI, 86.6-99.7). Notably, 92.3 percent of patients did not undergo radical cystectomy, and median time to cystectomy was not reached. Overall Health Status and Physical Functioning scores were maintained during treatment with gemcitabine intravesical system.1

The therapy was generally well-tolerated. Most patients (80.8 percent) experienced treatment-related side effects that were low grade, such as mild urinary symptoms, including burning, frequency, or urgency. More serious side effects (13.5 percent) were uncommon and most often involved bladder pain. A small number of patients (7.7 percent) discontinued treatment due to side effects, and no treatment-related deaths were reported.1

About SunRISe-1, Cohort 4

SunRISe-1 (NCT04640623) is an ongoing Phase 2b, open-label, multicenter study evaluating the efficacy and safety of gemcitabine intravesical system in patients with BCG-unresponsive HR-NMIBC who are ineligible for, or elected not to undergo, radical cystectomy. Cohort 4 specifically enrolls patients with papillary-only disease. The primary endpoint of Cohort 4 is disease-free survival (DFS) rate at 12 months. Key secondary endpoints included safety and tolerability.2

About High-Risk Non-Muscle Invasive Bladder Cancer

High-risk non-muscle invasive bladder cancer is a type of non-invasive bladder cancer that is more likely to recur or spread beyond the lining of the bladder, called the urothelium, and progress to muscle invasive bladder cancer compared to low-risk NMIBC.3,4 HR-NMIBC makes up 15-44 percent of patients with NMIBC and is characterized by a high-grade, large tumor size, presence of multiple tumors, and carcinoma in situ.5 Radical cystectomy is currently recommended for HR-NMIBC patients who fail BCG therapy, with over 90 percent cancer-specific survival if performed before muscle-invasive progression.6,7 Given that NMIBC typically affects older patients, many may be unwilling or unfit to undergo radical cystectomy.8 The high rates of recurrence and progression can pose significant morbidity and distress for these patients.3,4

About INLEXZO (gemcitabine intravesical system)

INLEXZO is approved by the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with Bacillus Calmette-Guérin (BCG)-unresponsive, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS), with or without papillary tumors.

The safety and efficacy of INLEXZO is being evaluated in clinical trials in patients with MIBC in SunRISe-4, and HR-NMIBC in SunRISe-1, SunRISe-3, and SunRISe-5.

The legal manufacturer for INLEXZO is Janssen Biotech, Inc.

INLEXZO IMPORTANT SAFETY INFORMATION9

CONTRAINDICATIONS

INLEXZO is contraindicated in patients with:

Perforation of the bladder.
Prior hypersensitivity reactions to gemcitabine or any component of the product.
WARNINGS AND PRECAUTIONS

Risks in Patients with Perforated Bladder

INLEXZO may lead to systemic exposure to gemcitabine and to severe adverse reactions if administered to patients with a perforated bladder or to those in whom the integrity of the bladder mucosa has been compromised.

Evaluate the bladder before the intravesical administration of INLEXZO and do not administer to patients with a perforated bladder or mucosal compromise until bladder integrity has been restored.

Risk of Metastatic Bladder Cancer with Delayed Cystectomy

Delaying cystectomy in patients with BCG-unresponsive CIS could lead to development of muscle invasive or metastatic bladder cancer, which can be lethal. The risk of developing muscle invasive or metastatic bladder cancer increases the longer cystectomy is delayed in the presence of persisting CIS.

Of the 83 evaluable patients with BCG-unresponsive CIS treated with INLEXZO in Cohort 2 of SunRISe-1, 7 patients (8%) progressed to muscle invasive (T2 or greater) bladder cancer. Three patients (3.5%) had progression determined at the time of cystectomy. The median time between determination of persistent or recurrent CIS or T1 and progression to muscle invasive disease was 94 days.

Magnetic Resonance Imaging (MRI) Safety

INLEXZO can only be safely scanned with MRI under certain conditions. Refer to section 5.3 of the USPI for details on conditions.

Embryo-Fetal Toxicity

Based on animal data and its mechanism of action, INLEXZO can cause fetal harm when administered to a pregnant woman if systemic exposure occurs. In animal reproduction studies, systemic administration of gemcitabine was teratogenic, embryotoxic, and fetotoxic in mice and rabbits.

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 and for 6 months after final removal of INLEXZO. Advise male patients with female partners of reproductive potential to use effective contraception during treatment and for 3 months after final removal of INLEXZO.

ADVERSE REACTIONS

Serious adverse reactions occurred in 24% of patients receiving INLEXZO. Serious adverse reactions that occurred in >2% of patients included urinary tract infection, hematuria, pneumonia, and urinary tract pain. Fatal adverse reactions occurred in 1.2% of patients who received INLEXZO, including cognitive disorder.

The most common (>15%) adverse reactions, including laboratory abnormalities, were urinary frequency, urinary tract infection, dysuria, micturition urgency, decreased hemoglobin, increased lipase, urinary tract pain, decreased lymphocytes, hematuria, increased creatinine, increased potassium, increased AST, decreased sodium, bladder irritation, and increased ALT.

USE IN SPECIFIC POPULATIONS

Pregnancy

There are no available data on the use of INLEXZO in pregnant women to inform a drug-associated risk. Please see Embryo-Fetal Toxicity for risk information related to pregnancy.

Lactation

Because of the potential for serious adverse reactions in breastfed infants, advise women not to breastfeed during treatment and for 1 week after final removal of INLEXZO.

Females and Males of Reproductive Potential

Pregnancy Testing – Verify pregnancy status in females of reproductive potential prior to initiating INLEXZO.

Contraception – Please see Embryo-Fetal Toxicity for information regarding contraception.

Infertility (Males) – Based on animal studies, INLEXZO may impair fertility in males of reproductive potential. It is not known whether these effects on fertility are reversible.

Geriatric Use

Of the patients given INLEXZO monotherapy in Cohort 2 of SunRISe-1, 72% were 65 years of age or older and 34% were 75 years or older. There were insufficient numbers of patients <65 years of age to determine if these patients respond differently to patients 65 years of age and older.

Please read full Prescribing Information and Instructions for Use for INLEXZO.

(Press release, Johnson & Johnson, DEC 5, 2025, View Source;johnsons-inlexzo-gemcitabine-intravesical-system-delivers-74-percent-disease-free-survival-at-one-year-in-bcg-unresponsive-high-risk-papillary-only-nmibc-302634314.html [SID1234661175])

On December 5, 2025 ImCheck Therapeutics reported an oral presentation at the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting 2025, taking place from December 6 to December 9, in Orlando, FL, USA. The presentation will highlight the high remission rates and overall survival observed in EVICTION, ImCheck’s open-label, randomized Phase I/II study evaluating ICT01, a first-in-class γ9δ2 T-cell activator, in combination with azacitidine and venetoclax in older or unfit patients with newly diagnosed acute myeloid leukemia (AML).

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Details of the oral presentation at ASH (Free ASH Whitepaper) 2025 are:

Abstract Title: "γ9δ2 T-cell (γ9δ2TC) activation with ICT01 and azacitidine-venetoclax (Aza-Ven) induces high rates of remission and overall survival in patients with newly diagnosed (ND) acute myeloid leukemia (AML): Results from the phase 1/2 study eviction"

Presenter: Sylvain GARCIAZ, MD, PhD, Institut Paoli-Calmettes, Marseille, France

Session: 616. Acute Myeloid Leukemias: Investigational Drug and Cellular Therapies: Immunotherapy and chemotherapy combinations in AML

Room: OCCC – Chapin Theater (320)

Date: Sunday, December 7, 2025

Time: 5:15 p.m.- 5:30 p.m. ET

The ASH (Free ASH Whitepaper) presentation will be available on ImCheck’s corporate website after the presentation has been held.

(Press release, ImCheck Therapeutics, DEC 5, 2025, View Source [SID1234661174])

On December 5, 2025 XOMA Royalty Corporation ("XOMA Royalty") (Nasdaq: XOMA), the biotech royalty aggregator, reported it has successfully completed its previously announced acquisition of the entire issued and to be issued share capital of Mural Oncology plc ("Mural") (Nasdaq: MURA) (the "Acquisition") pursuant to an Irish High Court sanctioned scheme of arrangement under Chapter 1 of Part 9 of the Companies Act 2014 of Ireland (the "Scheme"). Mural shareholders received $2.035 in cash per share (the "Consideration").

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The Acquisition was approved by Mural shareholders at a special meeting of shareholders convened pursuant to section 450(1) of the Irish Companies Act 2014 and an extraordinary general meeting of shareholders held on October 24, 2025.

The Irish High Court sanctioned the Scheme on December 3, 2025. On December 5, 2025, the Scheme and the Acquisition became effective upon delivery of the court order to the Irish Companies Registration Office.

Prior to the opening of trading on December 5, 2025, all of Mural’s shares will cease trading on Nasdaq, and Mural intends promptly to cause such shares to be delisted from Nasdaq and deregistered under the Securities Exchange Act of 1934, as amended.

Advisors
XOMA Royalty was represented by Gibson, Dunn & Crutcher LLP and Mason Hayes & Curran LLP, who acted as U.S. and Irish legal advisors, respectively. Davy Corporate Finance UC acted as financial advisor to XOMA Royalty. Lucid Capital Markets, LLC acted as exclusive financial advisor to Mural, and Wilmer Cutler Pickering Hale and Dorr LLP and Arthur Cox LLP served as U.S. and Irish legal advisor, respectively, to Mural.

(Press release, Xoma, DEC 5, 2025, View Source [SID1234661173])

On December 5, 2025 Wugen, Inc., a clinical-stage U.S. biotechnology company developing allogeneic, off-the-shelf cell therapies for the treatment of hematological malignancies, reported an upcoming scientific presentation of the company’s Phase 1/2 study of WU-CART-007 (Soficabtagene Geleucel "Sofi-cel"). Researchers will present Phase 1/2 correlative data and long-term follow-up update for its investigational cell therapy this week at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition, Dec. 6-9 in Orlando.

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Sofi-cel is an investigational, potential first-in-class, allogeneic, anti-CD7 CAR-T cell therapy currently under evaluation in a pivotal trial (T-RRex) for patients with relapsed or refractory (R/R) T cell acute lymphoblastic leukemia or T cell lymphoblastic lymphoma (T-ALL/LBL).

In the Phase 1/2 Study, Sofi-cel exhibited over one-hundred-fold expansion following infusion and persisted in circulation for up to three months. The serum cytokine profile was consistent with the mechanism of action. In the Long-Term Follow-Up study, there were no reported late adverse-events of special interest or drug-related serious adverse effects. Three patients who received a successful allogeneic stem cell transplant in the Phase 1/2 study remain alive approximately two years after Sofi-cel infusion, highlighting the promise of this investigational therapy in a challenging patient population.

"The robust cellular pharmacokinetics and long-term survival observed in heavily pretreated patients with R/R T-ALL/LBL in the Phase 1/2 study give confidence in the therapeutic potential of Soficabtagene Geleucel," said Wugen Chief Medical Officer, Cherry Thomas, M.D. "The T-RRex investigators bring extensive experience in this challenging disease and are committed to delivering meaningful clinical benefit for patients with limited treatment options."

The pivotal Phase 2 T-RRex study (NCT06514794) is a single-arm trial evaluating the safety and efficacy of Sofi-cel in patients with R/R T-ALL/LBL. The study is currently enrolling patients aged ≥ 1 year with R/R T-ALL/LBL and will include an exploratory cohort assessing patients with minimal residual disease (MRD)-positive status.

Wugen Presentation at ASH (Free ASH Whitepaper)

4163 WU-CART-007, a CD7-directed allogeneic CAR-T cell therapy for R/R T-cell acute lymphoblastic leukemia/lymphoma: Phase 1/2 correlative data and long-term follow-up update

Presenter: Alexander S. Hamil, Ph.D., Wugen Inc., St. Louis

Presentation type: Poster

Time/location: Sunday, Dec. 7, 6:00-8:00 p.m. ET; OCCC – West Halls B3-B4
Session: 704. Cellular Immunotherapies: Early Phase Clinical Trials and Toxicities: Poster II

About Soficabtagene Geleucel (Sofi-cel)

Sofi-cel is an allogeneic, off-the-shelf, CD7-targeted CAR-T cell therapy engineered to overcome the technological challenges of harnessing CAR-T cells to treat T-cell cancers. Wugen is deploying CRISPR/Cas9 gene editing technology to delete CD7 and the T cell receptor alpha constant (TRAC) genes, thereby preventing CAR-T cell fratricide and mitigating the risk of graft-versus-host disease (GvHD). Sofi-cel is manufactured using healthy donor-derived T cells to eliminate the risk of malignant cell contamination historically observed in the autologous CAR-T setting. Sofi-cel is currently being evaluated in a global pivotal clinical trial for relapsed or refractory T-ALL/T-LBL. More information on the Phase 1/2 trial is available on clinicaltrials.gov, identifier NCT04984356 and on the pivotal trial on clinicaltrials.gov, identifier NCT06514794.

Sofi-cel has received Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the U.S. Food and Drug Administration and Priority Medicines (PRIME) Scheme designation in the European Union for the treatment of relapsed or refractory T-ALL/T-LBL. RMAT and PRIME designations provide increased agency support to expedite the development and review of promising therapies for patients in need.

(Press release, Wugen, DEC 5, 2025, https://wugen.com/wugen-to-present-correlative-data-and-long-term-follow-up-updates-for-off-the-shelf-allogeneic-cd7-targeted-car-t-cell-therapy-at-the-2025-ash-annual-meeting/ [SID1234661172])