On July 30, 2026 One Biosciences, a techbio company pioneering clinical-grade single-cell tumor profiling, reported data establishing that single-cell RNA expression profiles can be generated from the standard, formalin-fixed paraffin-embedded (FFPE) pathology specimens obtained in routine cancer care using the company’s proprietary platform, OneMap.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The study, conducted with collaborators at Centre Léon Bérard, Hôpital Bichat–Claude Bernard, Institut Curie, and Memorial Sloan Kettering Cancer Center, was released as a preprint on BiorXiv.

"Every tumor is a unique ecosystem of many different types of cells, some cancerous, some not. Single cell profiling enables us to understand what those cells are, what they’re doing and potentially how that may impact treatment outcomes," said Helena Yu, MD, thoracic medical oncologist at Memorial Sloan Kettering Cancer Center. "Until now that type of analysis required different types of samples and workflows than we deploy in clinical care. This study shows we can do that analysis from the same samples we already collect, paving the way to more deeply explore how we can leverage this technology to guide treatment."

The study included multiple institutions from multiple regions and varied sample and tumor types to reflect the diversity encountered in real world clinical care. The data include findings from 88 single-nucleus experiments spanning bladder, breast, colon, lung, ovarian, and pancreatic cancers, including 36 core biopsies. The assay produced reproducible measurements of cellular composition and cell-type-resolved gene expression. Technical reproducibility was high (median correlation > 0.92 in cell-type proportions across replicates), and automated estimates of immune infiltration closely matched pathologists’ assessments of tumor-infiltrating lymphocytes. The workflow succeeded on archival blocks up to roughly a decade old and on ultra-low-input biopsies, and it quantified clinically relevant antibody-drug-conjugate targets.

The assay uses single-nucleus RNA sequencing to measure gene activity inside individual cells present in thin slices of tissue prepared for standard pathologic analyses. OneMap, an A.I.-powered software engine, identifies which cells are present and measures what each type is doing. OneMap then produces a single standardized report for each sample, eliminating the batch processing that older single-cell methods required.

"Single-cell analysis is a valuable addition to current molecular profiling, revealing important information about a tumor that standard tests cannot. Previously, it required the wrong kind of samples, too much tissue and analysis that only worked in large batches, making it impractical for real-world patient care," said Vincent Miller, executive chairman of One Biosciences. "This study validates our approach, which runs on ordinary pathology slides every hospital already produces, needs only a fraction of the tissue, and returns a standardized report for one patient at a time, creating a true sample-to-report solution that can enable more targeted clinical trials and ultimately improve patient care."

Standard tumor diagnostics average signal across millions of cells, potentially obscuring subpopulations of cells that might drive resistance, including immune cells. Single-cell profiling analyzes tumors one cell at a time, providing critical insights for both researchers and clinicians. Ongoing studies will explore whether single cell profiles can better select patients. In the clinic, single cell profiles offer an important addition to standard bulk profiling that grounds treatment decisions in the composition of a patient’s own tumor.

(Press release, One Biosciences, JUL 30, 2026, View Source [SID1234669554])

On July 30, 2026 Karyopharm Therapeutics Inc. (Nasdaq: KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, reported topline results from its Phase 3 XPORT-EC-042 trial evaluating selinexor as a maintenance-only therapy compared to placebo in adult patients with TP53 wild-type advanced or recurrent endometrial cancer. The trial did not meet its primary endpoint of progression free survival. A trend favoring the selinexor arm was observed in the modified intent to treat (mITT) population (n=236), with a median PFS of 12.75 months in the selinexor arm compared to 7.43 months in the placebo arm (hazard ratio=0.76 [95% CI: 0.51, 1.12]; one-sided p-value=0.0791). The safety and tolerability profile of selinexor was consistent with its established safety profile, with no new safety signals observed. Karyopharm intends to complete a full evaluation of the data from the XPORT-EC-042 trial and plans to present the data at a future medical meeting. The results of the XPORT-EC-042 trial do not affect ongoing trials of selinexor in other potential indications.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"These results are meaningful for a patient population lacking effective maintenance therapies that can delay disease progression," said Professor Ignace Vergote, MD, gynecologic oncologist at the Catholic University Leuven in Belgium, European Network for Gynaecological Oncological Trial groups (ENGOT) and global lead principal investigator. "The trend for a longer progression-free survival observed in the mITT population of the selinexor arm continues to highlight the potential of XPO1 inhibition in patients with TP53 wild-type/pMMR endometrial cancer."

"Delaying the progression of cancer by five months at the median is a meaningful and encouraging outcome," said Dr. Robert Coleman, M.D., FACOG, FACS, of Texas Oncology and the Gynecologic Oncology Group (GOG) and lead principal investigator in the United States. "Although I am disappointed that the PFS improvement was not statistically significant, I look forward to continuing to follow these results over time and presenting the data from this important trial at an upcoming medical meeting. This patient population who have TP53 wild-type/pMMR advanced or recurrent endometrial cancer remains in need of new treatment options."

"While disappointed by these unexpected results, we believe they advance the scientific understanding of XPO1 inhibition for tens of thousands of endometrial cancer patients worldwide. We are deeply committed to further investigating these data," said Reshma Rangwala, MD, PhD, Chief Medical Officer and Head of Research at Karyopharm. "I would like to thank all of the patients, their families and the clinical trial investigators and their staff, as well as ENGOT and GOG, for participating in this trial."

"While the results we are announcing today fell short of our expectations, they do not diminish our confidence in the broader potential of selinexor and benefit of XPO1 inhibition," said Richard Paulson, President and Chief Executive Officer of Karyopharm. "We remain focused on maximizing our opportunity in myelofibrosis and continuing to build on our profitable multiple myeloma business. Looking ahead, we expect several important milestones in our myelofibrosis program over the next year, including the submission of our sNDA, the potential addition of selinexor to relevant compendia guidelines and topline data from the 60 mg cohort of the Phase 2 SENTRY-2 trial, each anticipated in the second half of 2026."

About the Phase 3 XPORT-EC-042 Trial

EC-042 (XPORT-EC-042; ENGOT-EN20; GOG-3083; NCT05611931) is a global, Phase 3, randomized, double-blind, placebo-controlled clinical trial evaluating selinexor as a maintenance-only therapy following chemotherapy or chemotherapy plus a checkpoint inhibitor in patients with TP53 wild-type advanced or recurrent endometrial cancer (N=257). Patients were randomized 1:1 to receive either a 60 mg, once-weekly, administration of oral selinexor or placebo until disease progression. The trial includes two patient populations, for which the primary endpoint of progression free survival was tested sequentially: 1) a modified intent to treat population (mITT) that includes patients with either, a) TP53 wild-type tumors with proficient mismatch repair status (pMMR); or, b) TP53 wild-type tumors with deficient mismatch repair status (dMMR), who are medically ineligible to receive checkpoint inhibitors; and, 2) the trial’s original intent to treat (ITT) population, which includes all patients enrolled in the trial whose tumors are TP53 wild-type, regardless of MMR status. Overall survival is a key secondary endpoint. The mITT population enrolled 236 patients. As of the data cut-off, 106 progression free survival events as assessed by the investigator had been observed in the mITT population. In connection with the EC-042 trial, Karyopharm entered into a global collaboration with Foundation Medicine, Inc. to develop FoundationOneCDx, a tissue-based comprehensive genomic profiling test to identify and enroll patients whose tumors are TP53 wild-type. The trial is being conducted in collaboration with the European Network of Gynaecological Oncological Trial groups (ENGOT) and the GOG Foundation, Inc.

About Endometrial Cancer

Endometrial cancer (EC) is the most common gynecologic malignancy in the U.S.1 In 2026, approximately 68,000 uterine cancers (predominantly endometrial) are expected to be diagnosed, with approximately 14,000 deaths.1 Worldwide there were about 420,368 cases with 97,723 deaths in 2022.2 Both incidence and mortality have continued to rise.3,4 Key risk factors include obesity, type 2 diabetes, high-fat diets, tamoxifen or oral estrogen use, and delayed menopause.5 TP53 is a well-recognized prognostic marker for EC; >50% of advanced or recurrent EC tumors are TP53wt (gene for tumor protein P53; wild-type), and ~40%-55% are both TP53wt and mismatch repair-proficient (pMMR).6-8 While immune checkpoint inhibitors have shown benefit in patients with mismatch repair–deficient (dMMR) and pMMR, the magnitude of benefit is greater for patients with dMMR tumors versus pMMR tumors.9-10 There remains an unmet need for targeted therapies for patients with pMMR EC.11

1. American Cancer Society. Cancer Facts & Figures 2026. View Source Accessed February 8, 2026

2. IARC GLOBOCAN 2022, Global Estimates

3. Lu KH, et al. N Engl J Med. 2020;383:2053-2064

4. NCI. Cancer stat facts: uterine cancer. View Source Accessed October 7, 2025

5. American Cancer Society, Endometrial Cancer Risk Factors, 2025

6. Leslie KK, et al. Gynecol Oncol. 2021;161(1):113-121.

7. Vergote I, et al. J Clin Oncol. 2023;41(35):5400-5410.

8. Mirza MR, et al. Presentation at: ESMO (Free ESMO Whitepaper) Congress; October 20-24, 2023

9. Mirza MR, et al. N Engl J Med. 2023; 388:2145-2158.

10. Eskander RN, et al. N Engl J Med. 2023;388:2159-2170.

11. Makker V, et al. Gynecol Oncol. 2024 Jun:185: 202-211

About XPOVIO (selinexor)

XPOVIO is a first-in-class, oral exportin 1 (XPO1) inhibitor compound for the treatment of cancer. XPOVIO functions by selectively binding to and inhibiting the nuclear export protein XPO1. XPOVIO is approved and marketed by Karyopharm in the U.S. in multiple oncology indications, including: (i) in combination with VELCADE (bortezomib) and dexamethasone (XVd) in adult patients with multiple myeloma after at least one prior therapy; and (ii) in combination with dexamethasone in adult patients with heavily pre-treated multiple myeloma. XPOVIO (also known as NEXPOVIO in certain countries) has received regulatory approvals in various indications in a growing number of ex-U.S. territories and countries, including but not limited to the European Union, the United Kingdom, Mainland China, Taiwan, Hong Kong, Australia, South Korea, Singapore, Israel, and Canada. XPOVIO/NEXPOVIO is marketed in these respective ex-U.S. territories by Karyopharm’s partners: Antengene, Menarini, Neopharm, and FORUS. Selinexor is also being investigated in several other mid- and late-stage clinical trials across multiple high-unmet need cancer indications.

For more information about Karyopharm’s products or clinical trials, please contact the Medical Information department at: Tel: +1 (888) 209-9326; Email: [email protected]

XPOVIO (selinexor) is a prescription medicine approved:

In combination with bortezomib and dexamethasone for the treatment of adult patients with multiple myeloma who have received at least one prior therapy (XVd).
In combination with dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti‐CD38 monoclonal antibody (Xd).
SELECT IMPORTANT SAFETY INFORMATION

Warnings and Precautions

Thrombocytopenia: Monitor platelet counts throughout treatment. Manage with dose interruption and/or reduction and supportive care.
Neutropenia: Monitor neutrophil counts throughout treatment. Manage with dose interruption and/or reduction and granulocyte colony‐stimulating factors.
Gastrointestinal Toxicity: Nausea, vomiting, diarrhea, anorexia, and weight loss may occur. Provide antiemetic prophylaxis. Manage with dose interruption and/or reduction, antiemetics, and supportive care.
Hyponatremia: Monitor serum sodium levels throughout treatment. Correct for concurrent hyperglycemia and high serum paraprotein levels. Manage with dose interruption, reduction, or discontinuation, and supportive care.
Serious Infection: Monitor for infection and treat promptly.
Neurological Toxicity: Advise patients to refrain from driving and engaging in hazardous occupations or activities until neurological toxicity resolves. Optimize hydration status and concomitant medications to avoid dizziness or mental status changes.
Embryo‐Fetal Toxicity: Can cause fetal harm. Advise females of reproductive potential and males with a female partner of reproductive potential, of the potential risk to a fetus and use of effective contraception.
Cataract: Cataracts may develop or progress. Treatment of cataracts usually requires surgical removal of the cataract.

Adverse Reactions

The most common adverse reactions (≥20%) in patients with multiple myeloma who receive XVd are fatigue, nausea, decreased appetite, diarrhea, peripheral neuropathy, upper respiratory tract infection, decreased weight, cataract and vomiting. Grade 3‐4 laboratory abnormalities (≥10%) are thrombocytopenia, lymphopenia, hypophosphatemia, anemia, hyponatremia, and neutropenia. In the BOSTON trial, fatal adverse reactions occurred in 6% of patients within 30 days of last treatment. Serious adverse reactions occurred in 52% of patients. Treatment discontinuation rate due to adverse reactions was 19%.
The most common adverse reactions (≥20%) in patients with multiple myeloma who receive Xd are thrombocytopenia, fatigue, nausea, anemia, decreased appetite, decreased weight, diarrhea, vomiting, hyponatremia, neutropenia, leukopenia, constipation, dyspnea, and upper respiratory tract infection. In the STORM trial, fatal adverse reactions occurred in 9% of patients. Serious adverse reactions occurred in 58% of patients. Treatment discontinuation rate due to adverse reactions was 27%.
Use In Specific Populations
Lactation: Advise not to breastfeed.

For additional product information, including full prescribing information, please visit www.XPOVIO.com.

To report SUSPECTED ADVERSE REACTIONS, contact Karyopharm Therapeutics Inc. at 1‐888‐209‐9326 or FDA at 1‐800‐FDA‐1088 or www.fda.gov/medwatch.

(Press release, Karyopharm, JUL 30, 2026, View Source [SID1234669535])

On July 30, 2026 CEL-SCI Corporation (NYSE American: CVM) reported that a manuscript highlighting the latest findings from its Phase 3 study titled "A Novel Neoadjuvant Immunotherapy Confers Improved Overall Survival in Oral Cancer Patients with Low Tumor PD-L1 Expression: The IT-MATTERS Clinical Trial – Prognostic Role of Tumor PD-L1 Expression," has been accepted for publication in Oral Oncology, one of the world’s leading peer-reviewed journals dedicated to head and neck cancer research and treatment.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The manuscript, authored by Eyal Talor, Ph.D. (CEL-SCI Chief Scientific Officer, the developer of Multikine) and colleagues, is based on data from CEL-SCI’s landmark Phase 3 IT-MATTERS clinical trial evaluating Multikine (Leukocyte Interleukin, Injection)* in the treatment of newly diagnosed treatment naïve, resectable, locally advanced head and neck cancer patients. In accordance with the journal’s publication policies, CEL-SCI will disclose the scientific findings following publication in Oral Oncology.

"The acceptance of this manuscript by Oral Oncology represents another important validation of the quality and significance of our clinical data," said Geert Kersten, CEO of CEL-SCI. "Publication in leading peer-reviewed journals expands awareness of Multikine among oncologists, head and neck surgeons and cancer researchers around the world. As we prepare to begin patient enrollment in our Confirmatory Registration Study, we believe growing recognition within the scientific community will further strengthen the foundation supporting Multikine’s potential to become an important new treatment option for patients with low PD-L1 head and neck cancer."

In CEL-SCI’s Phase 3 study, a well-defined population of newly diagnosed, previously untreated, locally advanced resectable head and neck cancer patients with low tumor PD-L1 expression and no lymph node involvement treated with Multkine before receiving standard of care had a 73% five-year overall survival rate compared to 45% in patients receiving standard of care alone.

Unlike currently available immune checkpoint inhibitor therapies that primarily target patients with higher PD-L1 expression, Multikine is being developed specifically for patients with low PD-L1 tumor expression—an underserved patient population with limited treatment options and a recognized unmet medical need.

CEL-SCI is preparing to commence enrollment in its global 212-patient Confirmatory Registration Study, which is designed to confirm the previously observed overall survival benefit in this predefined target patient population. The registration study aims to enroll patients globally at clinical centers across the United States, Europe and Asia and is designed based on the approximately 97% statistical power to detect the previously observed overall survival hazard ratio of 0.34 in the same selected population of the completed Phase 3 study.

About Multikine

Multikine is a novel cancer immunotherapy administered before surgery as a treatment for newly diagnosed previously untreated locally advanced head and neck cancer. Its goal is to activate a person’s immune system to fight cancer before the ravages of surgery, radiation and chemotherapy have weakened the immune system. In the world’s largest head and neck cancer Phase 3 study in the same selected population, Multikine increased the 5-year survival rate of the target patient population to 73% vs 45% in patients treated with standard of care alone and halved the risk of death from 55% to 27%.

(Press release, Cel-Sci, JUL 30, 2026, View Source [SID1234669555])

On July 30, 2026 Karyopharm Therapeutics Inc. (Nasdaq: KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, reported that it plans to submit a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) in August 2026 seeking accelerated approval of selinexor in combination with ruxolitinib for the treatment of patients with myelofibrosis.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The planned submission follows productive engagements with the FDA, including written feedback that spleen volume reduction ≥ 35% (SVR35) appears to qualify as a reasonably likely surrogate endpoint (RLSE) to predict overall survival and can be used to support an sNDA under the accelerated approval pathway. The Company plans to use overall survival data from long-term follow-up of the ongoing Phase 3 SENTRY trial to verify clinical benefit. Overall survival is a pre-specified secondary endpoint of SENTRY. The trial does not permit patient crossover; patients, investigators and the Karyopharm study team remain blinded to treatment assignment during ongoing follow-up.

"The SENTRY trial generated one of the most compelling frontline datasets in myelofibrosis to date," said Dr. John Mascarenhas, Professor of Medicine at the Icahn School of Medicine at Mount Sinai and Director of the Center of Excellence for Blood Cancers and Myeloid Disorders. "The combination of selinexor and ruxolitinib demonstrated compelling spleen responses across a broad range of subgroups. The spleen responses were rapid, deep and sustained with promising overall survival findings and important evidence of disease modification. These results have the potential to redefine frontline treatment and establish a new treatment paradigm for patients with myelofibrosis."

"Patients with myelofibrosis have waited too long for meaningful innovation," said Richard Paulson, President and Chief Executive Officer of Karyopharm. "We are grateful to the FDA for its thoughtful and collaborative engagement in helping define a rigorous path forward. If approved, selinexor in combination with ruxolitinib has the potential to become the first approved combination therapy for patients with myelofibrosis, incorporating a novel class of therapy. We believe this represents a potentially transformative opportunity for patients and a defining moment in Karyopharm’s history as we work with urgency toward our planned August sNDA submission."

The planned sNDA will be based on results from the randomized, double-blind, Phase 3 SENTRY trial that compared selinexor in combination with ruxolitinib against placebo in combination with ruxolitinib, including the statistically significant improvement in SVR35 at week 24, the rapid, deep and sustained nature of the spleen responses, a promising overall survival signal, reductions in variant allele frequency and the overall safety data package.

"The SENTRY trial generated a substantial body of evidence showing consistent improvements across multiple measures of clinical activity, including spleen response and a promising signal of overall survival," said Reshma Rangwala, M.D., Ph.D., Chief Medical Officer and Head of Research of Karyopharm. "Together, these findings reinforce the biologic rationale for combining XPO1 and JAK inhibition and support the potential of this novel combination to deliver meaningful long-term benefits for patients with myelofibrosis."

Karyopharm intends to request Priority Review at the time of submission of the sNDA, which, if granted, would result in a Prescription Drug User Fee Act (PDUFA) target action date approximately six months following the FDA’s receipt of the application.

About the Phase 3 SENTRY Trial

SENTRY (XPORT-MF-034; NCT04562389) is a Phase 3 clinical trial evaluating a once-weekly dose of 60 mg of selinexor in combination with ruxolitinib compared to placebo plus ruxolitinib in JAKi-naïve myelofibrosis patients with platelet counts >100 x 109/L (N=353). Patients were randomized 2-to-1 to the selinexor arm. The co-primary endpoints for this trial are spleen volume reduction ≥ 35% (SVR35) at week 24 and the average change in absolute total symptom score (Abs-TSS) over 24 weeks relative to baseline. The results from the Phase 3 SENTRY trial were presented at the 2026 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting and were simultaneously published in the peer-reviewed Journal of Clinical Oncology. In addition, the results were presented at the 2026 European Hematology Association (EHA) (Free EHA Whitepaper) Congress, where the presentation was recognized as one of the six best abstracts at the meeting.

About Myelofibrosis

Myelofibrosis is a rare blood cancer that affects approximately 20,000 patients in the United States and 17,000 patients in the European Union1. The disease causes bone marrow fibrosis (scarring in the bone marrow), which makes it difficult for the bone marrow to make healthy blood cells, splenomegaly (enlarged spleen), progressive anemia which often leads to symptoms like fatigue and weakness, and other disease associated symptoms including abdominal discomfort, pain under the left ribs, early satiety, night sweats and bone pain. The only approved class of therapies to treat myelofibrosis are JAK inhibitors, including ruxolitinib.

1. Clarivate/DRG (2023)

About XPOVIO (selinexor)

XPOVIO is a first-in-class, oral exportin 1 (XPO1) inhibitor compound for the treatment of cancer. XPOVIO functions by selectively binding to and inhibiting the nuclear export protein XPO1. XPOVIO is approved and marketed by Karyopharm in the U.S. in multiple oncology indications, including: (i) in combination with VELCADE (bortezomib) and dexamethasone (XVd) in adult patients with multiple myeloma after at least one prior therapy; and (ii) in combination with dexamethasone in adult patients with heavily pre-treated multiple myeloma. XPOVIO (also known as NEXPOVIO in certain countries) has received regulatory approvals in various indications in a growing number of ex-U.S. territories and countries, including but not limited to the European Union, the United Kingdom, Mainland China, Taiwan, Hong Kong, Australia, South Korea, Singapore, Israel, and Canada. XPOVIO/NEXPOVIO is marketed in these respective ex-U.S. territories by Karyopharm’s partners: Antengene, Menarini, Neopharm, and FORUS. Selinexor is also being investigated in several other mid- and late-stage clinical trials across multiple high-unmet need cancer indications.

For more information about Karyopharm’s products or clinical trials, please contact the Medical Information department at: Tel: +1 (888) 209-9326; Email: [email protected]

XPOVIO (selinexor) is a prescription medicine approved:

In combination with bortezomib and dexamethasone for the treatment of adult patients with multiple myeloma who have received at least one prior therapy (XVd).
In combination with dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti‐CD38 monoclonal antibody (Xd).
SELECT IMPORTANT SAFETY INFORMATION

Warnings and Precautions

Thrombocytopenia: Monitor platelet counts throughout treatment. Manage with dose interruption and/or reduction and supportive care.
Neutropenia: Monitor neutrophil counts throughout treatment. Manage with dose interruption and/or reduction and granulocyte colony‐stimulating factors.
Gastrointestinal Toxicity: Nausea, vomiting, diarrhea, anorexia, and weight loss may occur. Provide antiemetic prophylaxis. Manage with dose interruption and/or reduction, antiemetics, and supportive care.
Hyponatremia: Monitor serum sodium levels throughout treatment. Correct for concurrent hyperglycemia and high serum paraprotein levels. Manage with dose interruption, reduction, or discontinuation, and supportive care.
Serious Infection: Monitor for infection and treat promptly.
Neurological Toxicity: Advise patients to refrain from driving and engaging in hazardous occupations or activities until neurological toxicity resolves. Optimize hydration status and concomitant medications to avoid dizziness or mental status changes.
Embryo‐Fetal Toxicity: Can cause fetal harm. Advise females of reproductive potential and males with a female partner of reproductive potential, of the potential risk to a fetus and use of effective contraception.
Cataract: Cataracts may develop or progress. Treatment of cataracts usually requires surgical removal of the cataract.
Adverse Reactions

The most common adverse reactions (≥20%) in patients with multiple myeloma who receive XVd are fatigue, nausea, decreased appetite, diarrhea, peripheral neuropathy, upper respiratory tract infection, decreased weight, cataract and vomiting. Grade 3‐4 laboratory abnormalities (≥10%) are thrombocytopenia, lymphopenia, hypophosphatemia, anemia, hyponatremia and neutropenia. In the BOSTON trial, fatal adverse reactions occurred in 6% of patients within 30 days of last treatment. Serious adverse reactions occurred in 52% of patients. Treatment discontinuation rate due to adverse reactions was 19%.
The most common adverse reactions (≥20%) in patients with multiple myeloma who receive Xd are thrombocytopenia, fatigue, nausea, anemia, decreased appetite, decreased weight, diarrhea, vomiting, hyponatremia, neutropenia, leukopenia, constipation, dyspnea and upper respiratory tract infection. In the STORM trial, fatal adverse reactions occurred in 9% of patients. Serious adverse reactions occurred in 58% of patients. Treatment discontinuation rate due to adverse reactions was 27%.
Use In Specific Populations
Lactation: Advise not to breastfeed.

For additional product information, including full prescribing information, please visit www.XPOVIO.com.

To report SUSPECTED ADVERSE REACTIONS, contact Karyopharm Therapeutics Inc. at 1‐888‐209‐9326 or FDA at 1‐800‐FDA‐1088 or www.fda.gov/medwatch.

(Press release, Karyopharm, JUL 30, 2026, View Source [SID1234669536])

On July 30, 2026 Sumitomo Pharma America, Inc. (SMPA) reported that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to enzomenib (DSP-5336) for the treatment of patients with acute lymphoblastic leukemia (ALL). 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.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Orphan drug designation is granted by the FDA to a drug or biological product to prevent, diagnose, or treat a rare disease or condition.1 The FDA previously granted orphan drug designation to enzomenib for acute myeloid leukemia (AML) in June 2022. 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) and the registrational Phase 2 Horizen-1 R/R mono AML/ALL (KMT2Ar + NPM1m) study.

"The availability and selection of treatment choices is a major clinical and logistical challenge for patients with acute lymphoblastic leukemia, a challenge underscored by the complexity of sequencing therapies," said Tsutomu Nakagawa, President and CEO of Sumitomo Pharma America (SMPA). "Receiving Orphan Drug Designation for enzomenib for the treatment of ALL is an exciting development that reinforces the molecule’s potential. We will work closely with the FDA to advance clinical research of enzomenib in the hopes of bringing an innovative new treatment option to people living with ALL."

ALL, also known as acute lymphocytic leukemia, progresses quickly if untreated. It is characterized by an acute onset and occurs when the bone marrow produces an overabundance of lymphocytes. In ALL, impaired blood cell production can increase susceptibility to infections and may result in anemia and an increased risk of bleeding. ALL cells may spread to other bodily areas including the brain and spinal cord.2

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 that plays key roles in gene expression and protein interactions involved in many biological pathways, including cell growth, cell cycle, genomic stability, and hematopoiesis.3,4 In preclinical studies, enzomenib has shown selective growth inhibition in human acute leukemia cell lines with KMT2A rearrangements or NPM1 mutations.3,5 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 or NPM1 mutation.6,7 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) and the registrational Phase 2 Horizen-1 R/R mono AML/ALL (KMT2Ar + NPM1m) study. 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 Ministry of Health, Labour and Welfare (MHLW) granted Orphan Drug Designation for enzomenib for the indication of relapsed or refractory acute myeloid leukemia with KMT2Ar or NPM1m in September 2024.

(Press release, Sumitomo Dainippon Pharma, JUL 30, 2026, View Source [SID1234669556])