On March 24, 2026 Karyopharm Therapeutics Inc. (Nasdaq: KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, reported topline results from its Phase 3 SENTRY trial, a randomized, double-blind, placebo-controlled trial of 60 mg selinexor in combination with ruxolitinib in frontline myelofibrosis (n=353). The trial met the first co-primary endpoint, demonstrating statistically significant improvement in spleen volume reduction of 35% or more (SVR35) for patients treated with the combination of selinexor plus ruxolitinib, with rapid, deep and sustained spleen volume reduction rates seen in the combination arm. The mean change in absolute total symptom score (Abs-TSS) at week 24 relative to baseline was comparable across the two arms with similar symptom improvement relative to baseline; the difference across the two arms was not statistically significant. Importantly, the topline results suggest a promising signal in overall survival (OS) for the combination arm.

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!

Spleen Volume: 50% of patients who received the combination of selinexor plus ruxolitinib achieved a statistically significant improvement in SVR35 at week 24 compared to 28% of patients who received ruxolitinib alone (one-sided p<0.0001). Patients on the combination achieved rapid spleen reduction with 49% already achieving SVR35 at week 12 compared to 20% who received ruxolitinib alone. Spleen volume reduction was sustained, with 47% of patients on the combination achieving SVR35 at week 36 compared to 23% who received ruxolitinib alone.

Symptoms: Similar symptom improvement from baseline was observed in patients who received the combination of selinexor plus ruxolitinib compared to ruxolitinib alone as measured by Abs-TSS at week 24. Patients who received the combination reported a 9.89 point improvement in Abs-TSS compared to a 10.86 point improvement in patients who received ruxolitinib alone.

Overall Survival: Promising OS signal was observed in patients who received the combination of selinexor plus ruxolitinib compared to ruxolitinib alone with a hazard ratio of 0.43 (95% CI [0.19, 1.00] nominal one-sided p=0.0222). The Company intends to continue to follow OS to maturity to further evaluate this signal.

Overall Survival Associated with SVR35: Post-hoc landmark analyses at weeks 12 and 24 suggest SVR35 may predict overall survival.

Variant Allele Frequency (VAF) Reduction: Evidence of potential disease modification from a pre-specified exploratory endpoint was observed at week 24 from baseline in the combination arm as 32% of patients who received the combination achieved a ≥20% reduction in VAF for JAK2, MPL, and CALR compared to 24% of patients who received ruxolitinib alone (n=261).

Other Secondary and Exploratory Endpoints: Across other secondary and exploratory endpoints of progression-free survival, hemoglobin stabilization, and bone marrow fibrosis improvement, no meaningful difference was observed between the trial arms as of the data cut-off of February 20, 2026. The Company intends to further evaluate these endpoints as they mature.
Patients were randomized 2:1 to 60 mg of selinexor once weekly plus ruxolitinib or placebo plus ruxolitinib. The ruxolitinib dose was determined based on the patients’ baseline platelet count per the drug’s prescribing information. All data presented are as of the data cut-off of February 20, 2026.

"The results from SENTRY are an important development for patients as the combination of selinexor plus ruxolitinib meaningfully improved spleen response and we observed a promising signal in overall survival. Reducing spleen volume remains one of the most important treatment goals in myelofibrosis since achieving SVR35 is associated with improvement in overall survival," 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. "While the symptom endpoint did not reach statistical significance, patients treated in both arms achieved similar symptom improvement relative to baseline. Importantly, while JAK inhibitors have been the backbone of therapy, continued progress requires new therapies that target additional biological pathways. Inhibition of XPO1 represents a differentiated mechanism that has the potential to address these pathways and evolve treatment beyond JAK inhibition alone."

"For patients with myelofibrosis, improvements in spleen and symptoms are expected outcomes from JAK inhibitors such as ruxolitinib. The SENTRY topline results suggest that the combination of selinexor and ruxolitinib delivers superior spleen reduction, which may predict overall survival, while offering similar symptom improvement, and may offer an important advance for our patients," said Dr. Claire Harrison, Professor of Myeloproliferative Neoplasms and Deputy Chief Medical Officer of Research, Data, and Analytics at Guy’s and St. Thomas’ NHS Foundation Trust in the United Kingdom.

"Selinexor’s differentiated mechanism provides a complementary approach to JAK inhibition and highlights the importance of targeting additional biological pathways beyond JAK signaling to further advance outcomes for patients with myelofibrosis. I am encouraged by the speed and magnitude of spleen response, and the promising overall survival signal and evidence of potential disease modification. In totality, these data underscore selinexor’s potential to meaningfully improve clinical outcomes for patients with myelofibrosis," said Reshma Rangwala, MD, PhD, Chief Medical Officer and Head of Research of Karyopharm. "On behalf of Karyopharm, I would like to thank the patients, families, caregivers, investigators, and clinical trial team who participated in this trial. We are excited to share our results with regulatory authorities, key opinion leaders and patient advocacy organizations."

"The myelofibrosis community is waiting for new treatment options that can build upon the benefit of JAK inhibitors. Improving overall survival is the ultimate goal for people living with myelofibrosis and I am incredibly encouraged by these results," said Kapila Viges, Chief Executive Officer of the MPN Research Foundation. "These results are an exciting development for the myelofibrosis community."

Safety and Tolerability

The combination demonstrated a manageable safety and tolerability profile consistent with the known profile of selinexor and ruxolitinib individually. No new safety signals were observed.

The five most common all-grade treatment emergent adverse events (TEAEs) in the selinexor plus ruxolitinib arm were thrombocytopenia (selinexor plus ruxolitinib arm: 59%; placebo plus ruxolitinib arm: 43%), anemia (57%; 58%), nausea (57%; 17%), constipation (32%; 36%) and neutropenia (27%; 9%) (n=234; n=116). The rate of grade 3+ TEAEs was 70% in the selinexor plus ruxolitinib arm compared to 50% in the placebo plus ruxolitinib arm. The rate of TEAEs leading to treatment discontinuation was 15% in the selinexor plus ruxolitinib arm and 9% in the placebo plus ruxolitinib arm. The rate of confirmed leukemic transformations was the same across both arms of the trial at 1.7%.

Next Steps

The Company will be meeting with the U.S. Food and Drug Administration (FDA) to discuss the totality of the data from the SENTRY trial and its supplemental new drug application (sNDA) filing plan.

The Company plans to share additional data from the Phase 3 SENTRY trial at an upcoming medical meeting and expects to submit a manuscript to a peer-reviewed medical journal. The Company believes that potential inclusion in relevant compendia could occur in the second half of 2026.

Conference Call Information

Karyopharm will host a conference call today, March 24, 2026, at 8:00 a.m. Eastern Time, to discuss the results of its Phase 3 SENTRY trial in myelofibrosis. To access the conference call, please dial (800) 836-8184 (local) or (646) 357-8785 (international) at least 10 minutes prior to the start time and ask to be joined into the Karyopharm Therapeutics call. A live audio webcast of the call, along with accompanying slides, will be available under "Events & Presentations" in the Investor section of the Company’s website. An archived webcast will be available on the Company’s website approximately two hours after the event.

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. 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.

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.

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 in the U.S. and marketed by Karyopharm 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; (ii) in combination with dexamethasone in adult patients with heavily pre-treated multiple myeloma; and (iii) under accelerated approval in adult patients with diffuse large B-cell lymphoma (DLBCL), including DLBCL arising from follicular lymphoma, after at least two lines of systemic therapy. 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, including in myelofibrosis and endometrial cancer.

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).

For the treatment of adult patients with relapsed or refractory diffuse large B‐cell lymphoma (DLBCL), not otherwise specified, including DLBCL arising from follicular lymphoma, after at least two lines of systemic therapy. This indication is approved under accelerated approval based on response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).
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%.

The most common adverse reactions (incidence ≥20%) in patients with DLBCL, excluding laboratory abnormalities, are fatigue, nausea, diarrhea, appetite decrease, weight decrease, constipation, vomiting, and pyrexia. Grade 3‐4 laboratory abnormalities (≥15%) are thrombocytopenia, lymphopenia, neutropenia, anemia, and hyponatremia. In the SADAL trial, fatal adverse reactions occurred in 3.7% of patients within 30 days, and 5% of patients within 60 days of last treatment; the most frequent fatal adverse reactions was infection (4.5% of patients). Serious adverse reactions occurred in 46% of patients; the most frequent serious adverse reaction was infection (21% of patients). Discontinuation due to adverse reactions occurred in 17% of patients.
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, MAR 24, 2026, View Source,-Demonstrating-Statistically-Significant-Improvement-in-Spleen-Volume-Reduction [SID1234663863])

On March 24, 2026 Fennec Pharmaceuticals Inc. (NASDAQ:FENC; TSX: FRX), a specialty pharmaceutical company, reported its financial results for the fiscal year ended December 31, 2025 and provided a business update.

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!

"Our 2025 results validate that our strategy is clear and the foundation we built over the past year is now propelling Fennec into its next chapter of growth. We delivered record net product sales, achieved significant growth within our Fennec HEARS program, and advanced independent clinical evidence generation for PEDMARK – all while driving quarter-over-quarter growth in every quarter in 2025. These results demonstrate increasing PEDMARK adoption across key accounts and patient segments, effective field execution, and sustained progress across the organization," said Jeff Hackman, chief executive officer of Fennec Pharmaceuticals. "Concurrently, we strengthened our financial position through prudent operating decisions and strategic financial initiatives, including the closing of public and private offerings and the completion of full debt redemption."

Business Highlights:

● Continued Growth Within Key PEDMARK Accounts: Adoption continues to accelerate across new and existing accounts, including multiple Adolescent and Young Adult (AYA) patients across several tumor types receiving PEDMARK. Strong adoption trends reflect growing confidence in PEDMARK’s clinical value and reinforce its potential to help reshape the standard of care for patients receiving cisplatin-based treatment, demonstrating that the Company’s growth strategies are well aligned with market opportunities.

● Expanded Field Team to Accelerate Growth: In the fourth quarter, given the positive momentum Fennec observed over 2025, the Company made the strategic decision to further enhance its execution by increasing its customer facing team to achieve greater reach and frequency with its customers so the organization can ultimately help more cancer patients protect their hearing.

● New Real-World Data in Adults with Head and Neck Cancer (HNC): In February 2026, Fennec announced it presented the first new data since the pivotal clinical program at the 2026 Multidisciplinary Head and Neck Cancers Symposium (MHNCS). Findings supporting the potential use of PEDMARK in adults with head and neck cancers (HNC) were observed in a multi-institutional retrospective review of 15 adults with HNC. The data showed that PEDMARK could be safely given ≥ six hours after cisplatin dosing and was easy to incorporate into the real-world care plan for adults with HNC. This strict post-cisplatin timing is a validated approach intended to preserve cisplatin antitumor activity and no disruption to curative-intent cisplatin-based treatment delivery was observed as part of the study review.

● Initiation of Two Institution-Led Clinical Studies: In December 2025, Fennec announced that City of Hope, a U.S. cancer research and treatment organization, is evaluating PEDMARK for the prevention of cisplatin-induced ototoxicity (CIO) in adult men with stage II-III metastatic testicular germ cell tumors. In March 2026, Fennec announced that Tampa General Hospital (TGH) Cancer Institute is initiating a study evaluating the real-world clinical utility of PEDMARK in reducing the risk of ototoxicity in AYA and adult cancer patients receiving cisplatin-based treatment. Additional investigator-initiated studies supporting the use of PEDMARK in additional tumor types and patient populations, including AYA cancer, have been submitted to Fennec and are currently under review.

● STS-J01 in Japan: In December, Fennec announced positive topline results from the investigator-initiated Phase 2/3 STS-J01 clinical trial evaluating PEDMARK for the reduction of cisplatin-induced ototoxicity in pediatric and adolescent and young adult (AYA) patients with non-metastatic solid tumors in Japan. The results were from the first large-scale pediatric and adolescent and young adults (AYA) trial in Japan and demonstrated that PEDMARK can protect hearing without compromising cisplatin’s efficacy or introducing any concerning side effects. The Company is pursuing registration in Japan and is also exploring partnering or licensing opportunities for PEDMARK.

Upcoming Events:

● Piper Sandler Spring Biopharma Symposium: The management team will host one-on-one investor meetings at the annual Piper Sandler Spring Biopharma Symposium being held April 15–April 16, 2026 at the Convene One Boston Place.

Financial Results for the Fourth Quarter and Full Fiscal Year Ended December 31, 2025

● Net Product Sales – For the fourth quarter of 2025, the Company recorded net product sales of $13.8 million compared to $7.9 million in the fourth quarter of 2024, representing an increase of approximately 75%. For the full fiscal year (FY) 2025, the Company recorded net product sales of approximately $44.6 million compared to $29.6 million in 2024, representing an increase of approximately 50%. The increase in net product sales is attributable to growth across both new and existing accounts with notable success in conversion and adherence of PEDMARK patients.
● Selling and Marketing Expenses – The Company recorded $6.1 million in selling and marketing expenses in the fourth quarter of 2025 compared to $3.9 million in the fourth quarter of 2024. The increase in selling and marketing expenses is largely related to increased payroll and additional marketing expenses as we focused on expanding our commercial team and preparing for additional outreach to community oncology centers and the adolescent and young adult (AYA) population. For the FY 2025, the Company recorded $18.6 million in selling and marketing compared to $18.4 million in fiscal year 2024. The year- over-year slight increase is largely related to increased payroll and marketing expenses in the comparable period offset by the elimination of European expenses after the announcement of the Norgine transaction in March 2024.

● General and Administrative (G&A) Expenses – The Company recorded $8.9 million in G&A expenses fourth quarter of 2025 compared to $4.2 million in the fourth quarter of 2024. For the FY 2025, the Company recorded $28.8 million in G&A expenses compared to $23.1 million in fiscal year 2024. G&A expenses increased in both the comparable quarterly and fiscal years due to increased intellectual property-related legal expenses, increased payroll expenses as headcount increased and increased non-cash expenses associated with equity-based remuneration.

● Cash Position – Cash and cash equivalents were $36.7 million as of December 31, 2025. For the FY 2025, there was a $10.2 million increase in cash and cash equivalents between December 31, 2024 and December 31, 2025. The net increase in cash was primarily due to the approximately $42.0 million in net proceeds from equity offerings and net cash collected from net product sales offset by operating expenses and the $21.5 million debt paydown in November of 2025. As of December 31, 2025 the company had $0 in debt outstanding.

Fourth Quarter and Full-Year 2025 Conference Call Information

Date:Tuesday, March 24, 2026

Time:8:30 a.m. Eastern Time

Webcast Link: View Source

Participant Link: View Source

1Financial Update
The selected financial data presented below is derived from our unaudited condensed consolidated financial statements, which were prepared in accordance with U.S. generally accepted accounting principles. The complete unaudited condensed consolidated financial statements for the period ended December 31, 2025, and management’s discussion and analysis of financial condition and results of operations will be available via www.sec.gov and www.sedar.com. All values are presented in thousands unless otherwise noted.

About Cisplatin-Induced Ototoxicity

Cisplatin and other platinum-based chemotherapies are widely used to treat solid tumors and have been vital in improving survival rates. Unfortunately, these life-saving treatments often result in permanent, irreversible hearing loss, also known as ototoxicity.i

Hearing loss from cisplatin treatment is not rare. Studies show that between 60-90% of patients treated with cisplatin may develop hearing loss, depending upon the dose and duration of chemotherapy.ii Many of those treated with cisplatin will require lifelong hearing aids or cochlear implants, which can be helpful for some, but do not reverse the hearing loss and can be costly over time.iii Treatment-induced hearing loss can reduce quality of survivorship as it impacts many aspects of life, such as speech and language skills, academic performance, social-emotional development, career potential and the ability to live independently.iv,v While audiologic monitoring is recommended to help manage ototoxicity, it is currently underutilized in certain cancer patient populations.

PEDMARK (sodium thiosulfate injection)

PEDMARK is the first and only U.S. Food and Drug Administration (FDA) approved therapy indicated to reduce the risk of ototoxicity associated with cisplatin treatment in pediatric patients 1 month of age and older with localized, non-metastatic, solid tumors. It is a unique formulation of sodium thiosulfate in single-dose, ready-to-use vials for intravenous use in pediatric patients. PEDMARK is also the first and only therapeutic agent with proven efficacy and safety data with an established dosing regimen, across two open-label, randomized Phase 3 clinical studies, the Children’s Oncology Group (COG) Protocol ACCL0431 and SIOPEL 6.

Additionally, PEDMARK is recommended for the adolescent and young adult (AYA) population by the National Comprehensive Cancer Network, or NCCN, with a 2A endorsement.

Approximately 500,000 patients in the U.S. are diagnosed annually with cancers that could be treated with a platinum-based chemotherapy.vi,vii The incidence of ototoxicity depends upon the dose and duration of chemotherapy, and many of those treated will require lifelong hearing aids. Until the FDA approval of PEDMARK, there were no preventative agents for this hearing loss. Patients with hearing loss resulting from cancer treatment have a statistically significant worse quality of life compared with peers who have no hearing loss.viii,ix

PEDMARK has been studied by co-operative groups in two Phase 3 clinical studies of survival and reduction of ototoxicity, COG ACCL0431 and SIOPEL 6. Both studies have been completed. The COG ACCL0431 protocol enrolled childhood cancers typically treated with intensive cisplatin therapy for localized and disseminated disease, including newly diagnosed hepatoblastoma, germ cell tumor, osteosarcoma, neuroblastoma, medulloblastoma, and other solid tumors. SIOPEL 6 enrolled only hepatoblastoma patients with localized tumors.

Indications and Usage
PEDMARK (sodium thiosulfate injection) is indicated to reduce the risk of ototoxicity associated with cisplatin in pediatric patients 1 month of age and older with localized, non-metastatic solid tumors.

Limitations of Use
The safety and efficacy of PEDMARK have not been established when administered following cisplatin infusions longer than 6 hours. PEDMARK may not reduce the risk of ototoxicity when administered following longer cisplatin infusions, because irreversible ototoxicity may have already occurred.

Important Safety Information
PEDMARK is contraindicated in patients with history of a severe hypersensitivity to sodium thiosulfate or any of its components.

Hypersensitivity reactions occurred in 8% to 13% of patients in clinical trials. Monitor patients for hypersensitivity reactions. Immediately discontinue PEDMARK and institute appropriate care if a hypersensitivity reaction occurs. Administer antihistamines or glucocorticoids (if appropriate) before each subsequent administration of PEDMARK. PEDMARK may contain sodium sulfite; patients with sulfite sensitivity may have hypersensitivity reactions, including anaphylactic symptoms and life-threatening or severe asthma episodes. Sulfite sensitivity is seen more frequently in people with asthma.

PEDMARK is not indicated for use in pediatric patients less than 1 month of age due to the increased risk of hypernatremia or in pediatric patients with metastatic cancers.

Hypernatremia occurred in 12% to 26% of patients in clinical trials, including a single Grade 3 case. Hypokalemia occurred in 15% to 27% of patients in clinical trials, with Grade 3 or 4 occurring in 9% to 27% of patients. Monitor

serum sodium and potassium levels at baseline and as clinically indicated. Withhold PEDMARK in patients with baseline serum sodium greater than 145 mmol/L.

Monitor for signs and symptoms of hypernatremia and hypokalemia more closely if the glomerular filtration rate (GFR) falls below 60 mL/min/1.73m2.

Administer antiemetics prior to each PEDMARK administration. Provide additional antiemetics and supportive care as appropriate.

The most common adverse reactions (≥25% with difference between arms of >5% compared to cisplatin alone) in SIOPEL 6 were vomiting, nausea, decreased hemoglobin, and hypernatremia. The most common adverse reaction (≥25% with difference between arms of >5% compared to cisplatin alone) in COG ACCL0431 was hypokalemia.

Please see full Prescribing Information for PEDMARK at: www.PEDMARK.com.

(Press release, Fennec Pharmaceuticals, MAR 24, 2026, View Source [SID1234663862])

On March 24, 2026 BioNTech SE (Nasdaq: BNTX, "BioNTech" or "the Company") reported that it will present data from its diversified portfolio in the field of lung cancer at the European Lung Cancer Congress ("ELCC") held in Copenhagen, Denmark, from March 25-28, 2026. The data updates covered in both oral and poster presentations highlight progress across late-stage immunomodulator candidates pumitamig and gotistobart, as well as antibody-drug conjugate ("ADC") programs, across various lung cancer subtypes and lines of treatment. BioNTech’s clinical portfolio encompasses both monotherapies and combinations with standard of care treatments, as well as novel-novel combination regimens aimed at delivering differentiated therapeutic profiles for the treatment of patients with lung cancer across all stages of the disease.

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 data we will present at this year’s ELCC further define the potential of our late-stage portfolio in lung cancer. With updates on pumitamig and gotistobart, as well as first clinical data for our HER3-targeted ADC, we continue to advance differentiated treatment approaches across lung cancer settings while building the clinical evidence to guide their further development," said Prof. Özlem Türeci, M.D., Co-Founder and Chief Medical Officer at BioNTech. "Our aim is to offer patients with lung cancer transformative treatment options that help provide meaningful long-term benefit across all stages of the disease."

Highlights of BioNTech’s lung cancer programs to be presented at ELCC 2026:

Pumitamig (BNT327/BMS986545) – a bispecific immunomodulator candidate combining PD-L1 checkpoint inhibition and VEGF-A neutralization, developed in collaboration with Bristol Myers Squibb Company ("BMS"):

1L ES-SCLC: Updated follow-up data from a single-arm Phase 2 clinical trial (NCT05844150) conducted in China continued to show encouraging preliminary antitumor activity and survival outcomes, together with a manageable tolerability profile for pumitamig plus chemotherapy as first-line therapy in patients with extensive-stage small cell lung cancer ("ES-SCLC"), an aggressive subtype of lung cancer. The data support the ongoing pivotal global Phase 3 ROSETTA Lung-01 clinical trial (NCT06712355) in first-line ES-SCLC.

1L NSCLC: New findings from a Phase 1b/2a clinical trial (NCT05918445) conducted in China showed preliminary antitumor activity irrespective of PD-L1 expression levels and a manageable safety profile for pumitamig as first-line monotherapy in both squamous and non-squamous advanced non-small cell lung cancer ("NSCLC"). The results complement the ongoing global Phase 2/3 ROSETTA Lung-02 clinical trial (NCT06712316) evaluating the combination of pumitamig with chemotherapy in first-line NSCLC.

EGFR-mutant NSCLC: Data from a Phase 2 clinical trial (NCT05756972) conducted in China showed clinically meaningful survival outcomes and a manageable safety and tolerability profile for pumitamig combined with chemotherapy in patients with EGFR-mutant advanced or metastatic NSCLC, regardless of PD-L1 expression level. These data highlight its potential in patients progressing on EGFR tyrosine kinase inhibitors.

Gotistobart (BNT316/ONC-392) – a tumor microenvironment-selective regulatory T cell depletion candidate targeting CTLA-4 and developed in collaboration with OncoC4, Inc. ("OncoC4"):

2L+ squamous NSCLC: Data from the non-pivotal, dose-confirmation stage 1 portion of the global Phase 3 PRESERVE-003 clinical trial (NCT05671510) showed clinically meaningful antitumor activity, an overall survival benefit with a 54% reduction in the risk of death compared with standard of care chemotherapy, and a manageable safety profile for gotistobart in patients with squamous NSCLC who have progressed on prior immunotherapy plus chemotherapy. The pivotal stage of the Phase 3 clinical trial is ongoing.

BNT326/YL202 – a HER3-targeted ADC candidate developed in collaboration with MediLink Therapeutics (Suzhou) Co., Ltd. ("MediLink"):

NSCLC: First clinical data from the NSCLC cohort of a Phase 2 clinical trial with BNT326/YL202 (NCT06107686) conducted in China showed antitumor activity and a favorable safety profile in patients with advanced or metastatic NSCLC who progressed after standard of care therapy. The findings support the ongoing Phase 1b/2 clinical trial (NCT07070232) evaluating the novel combination of pumitamig and BNT326/YL202.

Lung cancer is among BioNTech’s tumor focus areas, as the Company aims to address the significant unmet medical needs in the treatment of patients with lung cancer. BioNTech is advancing a diversified and robust clinical development approach in lung cancer spanning investigational next-generation immunomodulators, antibody-drug conjugates, mRNA cancer immunotherapies, and their combinations. With 16 ongoing clinical trials across various lung cancer subtypes and lines of treatment, including four ongoing pivotal Phase 3 clinical trials and five ongoing novel-novel combination trials, BioNTech is focused on developing innovative approaches to address the challenges of lung cancer treatment from early to late-stage conditions.

The abstracts are available on the ELCC Congress website. Click here for further information on BioNTech’s lung cancer portfolio.

Full presentation details:

Medicine Abstract Title Abstract Number/Presentation Details
Pumitamig First-Line Pumitamig (PD-L1 × VEGF-A bsAb) Monotherapy in PD-L1+ Non-Squamous and Squamous Non-Small Cell Lung Cancer: Data from a Phase 1b/2a Trial in China Abstract #69P
Poster
March 27, 2026; 1:00 – 2:00pm CET
Progression-Free Survival and Overall Survival with Pumitamig (PD-L1 × VEGF-A bsAb) Plus Chemotherapy in Patients With EGFR-Mutated Advanced Non-Small Cell Lung Cancer Following Progression with EGFR TKI in China: Phase 2 Study Results Abstract #21P
Poster
March 27, 2026; 1:00 – 2:00pm CET
Phase 2 Study of First-Line Pumitamig (PD-L1 × VEGF-A bsAb) Plus Chemotherapy for Extensive-Stage Small-Cell Lung Cancer (ES-SCLC): Updated Efficacy and Safety Results Abstract #426P
Poster
March 26, 2026; 1:00 – 2:00pm CET
ROSETTA Lung-01: A Phase 3, Two-Stage Trial of Pumitamig, a PD-L1 × VEGF-A Bispecific Antibody, Plus Chemotherapy Versus Atezolizumab + Chemotherapy as First-Line Treatment in Patients with Extensive-Stage Small Cell Lung Cancer Abstract #439TiP
Poster
March 26, 2026; 1:00 – 2:00pm CET
ROSETTA Lung-02: A Global Phase 2/3, Randomized, Open-Label Trial of Pumitamig, a PD-L1 × VEGF-A Bispecific Antibody, in Combination with Chemotherapy in Patients (pts) With First-Line Non-Small Cell Lung Cancer Abstract #149TiP
Poster
March 27, 2026; 1:00 – 2:00pm CET
Gotistobart Anti-Tumor Activity of Gotistobart Compared to Docetaxel in Patients with Metastatic Squamous Non-Small Cell Lung Cancer (sqNSCLC) Progressing on PD-(L)1 Inhibitors: Stage 1 PRESERVE-003 Phase 3 Trial Abstract #3O
Proffered paper session
March 27, 2026; 3:35 – 3:45pm CET
BNT326/YL202 First Disclosure of Efficacy and Safety Data for YL202/BNT326 (HER3 ADC) From a Phase 2 Trial in Patients (pts) with Non-Small Cell Lung Cancer (NSCLC) Abstract #11MO
Mini oral session
March 27, 2026; 09:15 – 09:20am CET
BNT326-02: A Phase 1b/2 Trial of BNT326/YL202 (HER3 ADC) with Pumitamig (PD-L1 × VEGF-A bsAb) in Non-Small Cell Lung Cancer (NSCLC) Abstract #6147TiP
Poster
March 27, 2026; 1:00 – 2:00pm CET

(Press release, BioNTech, MAR 24, 2026, View Source [SID1234663860])

On March 23, 2026 Daiichi Sankyo reported ENHERTU (trastuzumab deruxtecan) has been approved in Japan for the treatment of adult patients with HER2 positive (HER2 [ERBB2] gene amplification or immunohistochemistry [IHC] 3+) advanced or recurrent solid cancers refractory or intolerant to standard treatments.

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!

ENHERTU is a specifically engineered HER2 directed DXd antibody drug conjugate (ADC) discovered by Daiichi Sankyo (TSE: 4568) and being developed and commercialized by Daiichi Sankyo in Japan.

The approval by Japan’s Ministry of Health, Labour and Welfare (MHLW) is based on results from four phase 2 trials, including HERALD, an investigator-initiated trial conducted in Japan, DESTINYPanTumor02, DESTINY-CRC02 and DESTINY-Lung01 where ENHERTU demonstrated clinically meaningful responses across a broad range of tumors. A companion diagnostic (CDx) to test liquid biopsy for HER2 amplification is approved for this new tumor agnostic indication of ENHERTU in Japan. A separate CDx submission to test tumor tissue for HER2 expression (IHC 3+) is planned.

In HERALD, ENHERTU demonstrated a confirmed objective response rate (ORR) of 56.5% (95% confidence interval [CI]: 43.3-69.0) as assessed by investigator in patients (n=62) with unresectable, advanced or recurrent HER2 positive solid cancers refractory or intolerant to standard treatments, including biliary tract, cervical, colorectal, endometrial, esophageal, gastric, melanoma, non-small cell lung (NSCLC), ovarian, pancreatic, prostate, salivary gland, small intestine, urothelial cancer or other tumors.

In DESTINY-PanTumor02, ENHERTU demonstrated a confirmed ORR of 61.3% (95% CI: 49.4-72.4) as assessed by investigator in a subgroup of previously treated patients (n=75) with HER2 positive unresectable advanced or recurrent solid tumors, including biliary tract, bladder, cervical, endometrial, ovarian, pancreatic or other tumors. In DESTINY-Lung01, ENHERTU demonstrated a confirmed ORR of 52.9% (95% CI: 27.8- 77.0) in a subgroup of patients (n=17) with HER2 positive unresectable or metastatic NSCLC. In DESTINY-CRC02, ENHERTU demonstrated a confirmed ORR of 46.9% (95% CI: 34.3-59.8) in a subgroup of patients (n=64) with HER2 positive locally advanced, unresectable or metastatic colorectal cancer.

"This is the first tumor agnostic approval for a HER2 directed medicine and antibody drug conjugate in Japan and marks the sixth approved indication for ENHERTU," said Yuki Abe, PhD, Head of R&D Division in Japan and Head of Research, Daiichi Sankyo. "Based on the clinically meaningful efficacy seen with ENHERTU across numerous types of metastatic cancer, this milestone validates the importance of testing for HER2 across a broad range of tumors

In HERALD, adverse reactions occurred in 59 patients (95.2%) treated with ENHERTU. The most common adverse reactions were nausea (58.1%), decreased appetite (53.2%), fatigue (46.8%), anemia (38.7%), decreased neutrophil count (32.3%), decreased white blood cell count (32.3%), decreased platelet count (24.2%) and stomatitis (22.6%). In DESTINY-PanTumor02, adverse reactions occurred in 65 patients (86.7%) treated with ENHERTU. The most common adverse reactions were fatigue (50.7%), nausea (46.7%), decreased neutrophil count (45.3%), diarrhea (33.3%), anemia (28.0%) and decreased appetite (21.3%). In DESTINY-Lung01, adverse reactions occurred in 17 patients (100%), including two Japanese patients, treated with ENHERTU. The most common adverse reactions were nausea (82.4%), fatigue (52.9%), decreased appetite (35.3%), vomiting (23.5%), diarrhea (23.5%) and anemia (23.5%). In DESTINY-CRC02, adverse reactions occurred in 61 patients (93.8%) treated with ENHERTU. The most common adverse reactions were nausea (56.9%), fatigue (43.1%), decreased neutrophil count (30.8%), diarrhea (23.1%), decreased appetite (23.1%), alopecia (23.1%) and vomiting (20.0%).

ENHERTU is approved in Japan with a Warning in its prescribing information for interstitial lung disease (ILD). ILD occurred in 262 patients (11.6%) treated with ENHERTU across multiple clinical trials. As cases of ILD, including fatal cases, have occurred in ENHERTU-treated patients, ENHERTU is to be used in close collaboration with a respiratory disease expert. Patients should be closely observed during therapy by monitoring for early signs or symptoms of ILD (such as dyspnea, cough or fever) and performing regular peripheral artery oxygen saturation (SpO2) tests, chest X-ray scans and chest CT scans. If abnormalities are observed, discontinue administration of ENHERTU and take appropriate measures, such as corticosteroid administration. Prior to initiation of ENHERTU therapy, a chest CT scan should be performed and medical history taken to confirm the absence of any comorbidity or history of ILD with the patient and carefully consider the eligibility of the patient for ENHERTU therapy.

About HERALD
HERALD is a multicenter, open-label, single-arm, investigator-initiated, phase 2 trial evaluating the efficacy and safety of ENHERTU (5.4 mg/kg) in patients with unresectable, advanced or recurrent solid tumors refractory or intolerant to standard treatments and have HER2 gene amplification in circulating tumor DNA, including biliary tract, cervical, colorectal, endometrial, esophageal, gastric, melanoma, NSCLC, ovarian, pancreatic, prostate, salivary gland, small intestine, urothelial cancer or other tumors.

The primary endpoint of HERALD is confirmed ORR as assessed by investigator. Secondary endpoints include duration of response (DOR), disease control rate (DCR), progression-free survival (PFS), time to treatment failure, overall survival (OS), ORR by independent central review and safety. Results from HERALD were published in the Journal of Clinical Oncology.

HERALD enrolled 62 patients at seven sites in Japan. For more information about the trial, visit Japan’s Registry of Clinical Trials.

About DESTINY-PanTumor02
DESTINY-PanTumor02 is a global, multicenter, multi-cohort, open-label, phase 2 trial evaluating the efficacy and safety of ENHERTU (5.4 mg/kg) for the treatment of previously treated HER2 expressing tumors, including biliary tract, bladder, cervical, endometrial, ovarian, pancreatic cancer or other tumors.

The primary endpoint of DESTINY-PanTumor02 is confirmed ORR as assessed by investigator. Secondary endpoints include DOR, DCR, PFS, OS, safety, tolerability and pharmacokinetics. Results from DESTINYPanTumor02 were published in the Journal of Clinical Oncology.

On March 23, 2026 Daiichi Sankyo reported the Japan Pharmaceuticals and Medical Devices Agency (PMDA) has accepted the update of the ENHERTU (trastuzumab deruxtecan) prescribing information following review of data from the DESTINY-Gastric04 phase 3 trial, which now expands the use of ENHERTU in Japan to include the second-line treatment of patients with HER2 positive (immunohistochemistry [IHC] 3+ or IHC 2+/in-situ hybridization [ISH]+) unresectable advanced or recurrent gastric cancer. ENHERTU previously was approved as a third-line treatment based on the results from the DESTINY-Gastric01 phase 2 trial.

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!

ENHERTU is a specifically engineered HER2 directed DXd antibody drug conjugate (ADC) discovered by Daiichi Sankyo (TSE: 4568) and being developed and commercialized by Daiichi Sankyo in Japan.

Gastric cancer is the third most common cancer in Japan.1 More than 125,000 cases of gastric cancer were diagnosed in Japan in 2022, with more than 43,000 deaths.1 Approximately one in five gastric cancers are considered HER2 positive. 2,3 Prior to the results of DESTINY-Gastric04, no other HER2 directed medicine has demonstrated a survival benefit in the second-line metastatic setting in a randomized clinical trial.4

In DESTINY-Gastric04, ENHERTU demonstrated a 30% reduction in risk of death compared to ramucirumab plus paclitaxel in patients with second-line HER2 positive unresectable and/or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma (hazard ratio [HR]: 0.70; 95% confidence interval [CI]: 0.550-0.896; p=0.0044). Median overall survival (OS) was 14.7 months with ENHERTU (n=246; 95% CI: 12.1-16.6) compared to 11.4 months with ramucirumab plus paclitaxel (n=248; 95% CI: 9.9-15.5). DESTINY-Gastric04 was presented as a late-breaking oral presentation at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) (#ASCO25) Annual Meeting and simultaneously published in The New England Journal of Medicine.

"Gastric cancer can be particularly challenging to treat and is associated with a poor prognosis, especially in the metastatic setting where outcomes are notably worse for patients with disease progression after first-line treatment," said Yuki Abe, PhD, Head of R&D Division in Japan and Head of Research, Daiichi Sankyo. "The impressive survival results seen in DESTINY-Gastric04 support the expanded use of ENHERTU in Japan, making it available as a new second-line option for patients with HER2 positive metastatic gastric cancer."

In DESTINY-Gastric04, the safety profile of ENHERTU was consistent with previous clinical trials with no new safety concerns identified. Adverse reactions occurred in 227 patients (93.0%) treated with ENHERTU (6.4 mg/kg), including 26 Japanese patients. The most common adverse reactions were fatigue (48.0%), decreased neutrophil count (48.0%), nausea (44.3%), anemia (31.1%), decreased appetite (29.1%), decreased leukocyte count (26.6%), decreased platelet count (26.6%), diarrhea (25.8%), alopecia (24.2%), increased transaminase (21.7%) and vomiting (20.1%).

ENHERTU is approved in Japan with a Warning in its prescribing information for interstitial lung disease (ILD). ILD occurred in 262 patients (11.6%) treated with ENHERTU across multiple clinical trials. As cases of ILD, including fatal cases, have occurred in ENHERTU-treated patients, ENHERTU is to be used in close collaboration with a respiratory disease expert. Patients should be closely observed during therapy by monitoring for early signs or symptoms of ILD (such as dyspnea, cough or fever) and performing regular peripheral artery oxygen saturation (SpO2) tests, chest X-ray scans and chest CT scans. If abnormalities are observed, discontinue administration of ENHERTU and take appropriate measures, such as corticosteroid administration. Prior to initiation of ENHERTU therapy, a chest CT scan should be performed and medical history taken to confirm the absence of any comorbidity or history of ILD with the patient and carefully consider the eligibility of the patient for ENHERTU therapy.

About DESTINY-Gastric04
DESTINY-Gastric04 is a global, randomized, open-label, phase 3 trial evaluating the efficacy and safety of ENHERTU (6.4 mg/kg) versus ramucirumab and paclitaxel in patients with HER2 positive (IHC 3+ or IHC 2+/ISH+) unresectable and/or metastatic gastric or GEJ adenocarcinoma with disease progression on or after a trastuzumab-containing regimen.

The primary endpoint is OS. Secondary endpoints include investigator-assessed progression-free survival, objective response rate, duration of response, disease control rate and safety.

At disclosure of the topline results, an Independent Data Monitoring Committee recommended unblinding DESTINY-Gastric04 based on the superior efficacy of ENHERTU seen at a planned interim analysis.

DESTINY-Gastric04 enrolled 494 patients in Asia, Europe and South America. For more information about the trial, visit ClinicalTrials.gov.

About HER2 Positive Gastric Cancer
Gastric (stomach) cancer is the fifth most common cancer worldwide and the fifth leading cause of cancerrelated death, with a five-year global survival rate of 5% to 10%. 5,6 Approximately one million cases of gastric cancer were diagnosed in 2022. 5 Gastric cancer is the third most common cancer in Japan.1 More than 125,000 cases of gastric cancer were diagnosed in Japan in 2022, with more than 43,000 deaths.1

HER2 is a tyrosine kinase receptor growth-promoting protein expressed on the surface of many types of tumors, including gastric cancer.3,7 Approximately one in five gastric cancers are considered HER2 positive. 2,3

Prior to the results of the DESTINY-Gastric04 trial of ENHERTU, no other HER2 directed medicine has demonstrated a survival benefit in the second-line metastatic setting in a randomized clinical trial

About ENHERTU
ENHERTU (trastuzumab deruxtecan; fam-trastuzumab deruxtecan-nxki in the U.S. only) is a HER2 directed ADC. Designed using Daiichi Sankyo’s proprietary DXd ADC Technology, ENHERTU is the lead ADC in the oncology portfolio of Daiichi Sankyo and the most advanced program in AstraZeneca’s ADC scientific platform. ENHERTU consists of a HER2 monoclonal antibody attached to a number of topoisomerase I inhibitor payloads (an exatecan derivative, DXd) via tetrapeptide-based cleavable linkers.

ENHERTU (5.4 mg/kg) in combination with pertuzumab is approved in the U.S. as a first-line treatment for adult patients with unresectable or metastatic HER2 positive (IHC 3+ or ISH+) breast cancer, as determined by an FDA-approved test, based on the results from the DESTINY-Breast09 trial.

ENHERTU (5.4 mg/kg) is approved in more than 90 countries/regions worldwide for the treatment of adult patients with unresectable or metastatic HER2 positive (IHC 3+ or ISH+) breast cancer who have received a prior anti-HER2-based regimen, either in the metastatic setting or in the neoadjuvant or adjuvant setting, and have developed disease recurrence during or within six months of completing therapy based on the results from the DESTINY-Breast03 trial.

ENHERTU (5.4 mg/kg) is approved in more than 90 countries/regions worldwide for the treatment of adult patients with unresectable or metastatic HER2 low (IHC 1+ or IHC 2+/ISH-) breast cancer who have received a prior systemic therapy in the metastatic setting or developed disease recurrence during or within six months of completing adjuvant chemotherapy based on the results from the DESTINY-Breast04 trial.

ENHERTU (5.4 mg/kg) is approved in more than 60 countries/regions worldwide for the treatment of adult patients with unresectable or metastatic hormone receptor (HR) positive, HER2 low (IHC 1+ or IHC 2+/ ISH-) or HER2 ultralow (IHC 0 with membrane staining) breast cancer, as determined by a locally or regionally approved test, that have progressed on one or more endocrine therapies in the metastatic setting based on the results from the DESTINY-Breast06 trial.

ENHERTU (5.4 mg/kg) is approved in more than 70 countries/regions worldwide for the treatment of adult patients with unresectable or metastatic NSCLC whose tumors have activating HER2 (ERBB2) mutations, as detected by a locally or regionally approved test, and who have received a prior systemic therapy based on the results from the DESTINY-Lung02 and/or DESTINY-Lung05 trials. Continued approval in China and the U.S. for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

ENHERTU (6.4 mg/kg) is approved in more than 80 countries/regions worldwide for the treatment of adult patients with locally advanced or metastatic HER2 positive (IHC 3+ or IHC 2+/ISH+) gastric or GEJ adenocarcinoma who have received a prior trastuzumab-based regimen based on the results from the DESTINY-Gastric01, DESTINY-Gastric02 and/or DESTINY-Gastric04 trials.

ENHERTU (5.4 mg/kg) is approved in more than 15 countries/regions worldwide for the treatment of adult patients with unresectable or metastatic HER2 positive (IHC 3+) solid tumors who have received prior systemic treatment and have no satisfactory alternative treatment options based on efficacy results from the DESTINY-PanTumor02, DESTINY-Lung01, DESTINY-CRC02 and/or HERALD trials. Continued approval in the U.S. for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

About the ENHERTU Clinical Development Program
A comprehensive global clinical development program is underway evaluating the efficacy and safety of ENHERTU as a monotherapy or in combination or sequentially with other cancer medicines across multiple HER2 targetable cancers.

About the Daiichi Sankyo and AstraZeneca Collaboration
Daiichi Sankyo and AstraZeneca entered into a global collaboration to jointly develop and commercialize ENHERTU in March 2019 and DATROWAY in July 2020, except in Japan where Daiichi Sankyo maintains exclusive rights for each ADC. Daiichi Sankyo is responsible for the manufacturing and supply of ENHERTU and DATROWAY.

About the ADC Portfolio of Daiichi Sankyo
The Daiichi Sankyo ADC portfolio consists of eight ADCs in clinical development crafted from ADC technology discovered in-house by Daiichi Sankyo.

The DXd ADC Technology platform of Daiichi Sankyo consists of seven ADCs in clinical development where each ADC is comprised of a monoclonal antibody attached to a number of topoisomerase I inhibitor payloads (an exatecan derivative, DXd) via tetrapeptide-based cleavable linkers. The DXd ADCs include ENHERTU and DATROWAY, which are being jointly developed and commercialized globally with AstraZeneca, and ifinatamab deruxtecan (I-DXd), raludotatug deruxtecan (R-DXd) and patritumab deruxtecan (HER3-DXd), which are being jointly developed and commercialized globally with Merck & Co., Inc, Rahway, NJ, USA. DS-3939 and DS3790 are being developed by Daiichi Sankyo.

An additional ADC being developed by Daiichi Sankyo is DS3610, which consists of an antibody attached to a novel payload that acts as an agonist of STING.

Ifinatamab deruxtecan, raludotatug deruxtecan, patritumab deruxtecan, DS-3939, DS3610 and DS3790 are investigational medicines that have not been approved for any indication in any country. Safety and efficacy have not been established.

(Press release, Daiichi Sankyo, MAR 23, 2026, View Source [SID1234665016])