On May 27, 2026 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported that the New England Journal of Medicine has published the Phase 3 HERIZON-GEA-01 trial results, further characterizing the efficacy and safety profile of Ziihera (zanidatamab-hrii) in combination with chemotherapy, with and without the PD-1 inhibitor Tevimbra (tislelizumab), as first-line treatment for adults with HER2-positive (HER2+) locally advanced or metastatic gastroesophageal adenocarcinoma (GEA), including cancers of the stomach, gastroesophageal junction, and esophagus.

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The manuscript builds on the late-breaking oral presentation of the HERIZON-GEA-01 trial results at the 2026 ASCO (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium (ASCO GI), where zanidatamab-containing combinations demonstrated unprecedented progression-free survival (PFS) and overall survival (OS) outcomes in a global Phase 3 trial.

As previously reported, both zanidatamab-containing combinations significantly improved PFS compared with trastuzumab plus chemotherapy (median PFS 12.4 months versus 8.1 months; Hazard Ratio (HR) 0.63-0.65), and zanidatamab plus tislelizumab and chemotherapy demonstrated a statistically significant OS benefit (median OS 26.4 versus 19.2 months; HR 0.72).
At the first interim analysis, zanidatamab plus chemotherapy also demonstrated a median OS of more than two years. The OS for zanidatamab plus chemotherapy will be assessed at a second interim analysis expected in mid-2026.
The publication includes expanded prespecified subgroup analyses showing that PFS and OS results were generally consistent across clinically relevant patient characteristics, including PD-L1 status, geographic region, and Eastern Cooperative Oncology Group performance status, as well as PFS sensitivity analyses supporting the robustness of the findings.
Expanded safety analyses further characterize the safety profile of zanidatamab-containing regimens.
"The additional analyses from HERIZON-GEA-01 provide further support for using zanidatamab in clinical practice," said Kohei Shitara, M.D., director of the Department of Gastrointestinal Oncology, co-lead author of the New England Journal of Medicine publication, and principal investigator of the HERIZON-GEA-01 trial at the National Cancer Center Hospital East in Kashiwa, Japan. "For patients with HER2+ locally advanced or metastatic GEA, long-term outcomes have historically been limited. The expanded subgroup and sensitivity analyses indicate the durability and consistency of benefit observed with zanidatamab-containing regimens. Importantly, survival benefit with the addition of tislelizumab was suggested across PD-L1-defined subgroups, including in patients with PD-L1-negative tumors, a population that has historically derived limited benefit from PD-1-based approaches."

"We believe the HERIZON-GEA-01 results, together with the additional analyses now published, establish zanidatamab’s differentiated clinical profile in first-line HER2+ locally advanced or metastatic GEA," said Rob Iannone, M.D., M.S.C.E., executive vice president, global head of research and development, and chief medical officer of Jazz Pharmaceuticals. "In this global Phase 3 trial, zanidatamab demonstrated superior efficacy compared with trastuzumab-based standard of care, and the addition of tislelizumab enhanced overall survival. Taken together, these findings support the potential for zanidatamab-containing regimens to become a HER2-targeted agent of choice in this setting. Additionally, this marks the first immuno-oncology combination to show efficacy across both PD-L1–positive and PD-L1–negative tumors in this clinical setting, consistent with zanidatamab’s unique mechanism of action that enhances immune activation through complement-dependent cytotoxicity (CDC), antibody-dependent cellular cytotoxicity (ADCC), and antibody-dependent cellular phagocytosis (ADCP). These results are reshaping expectations for first-line treatment in this disease, and we continue to work with urgency to deliver this treatment option for patients."

Results of additional safety analyses of zanidatamab plus chemotherapy, with and without tislelizumab, were consistent with the known safety profiles of the individual components. Gastrointestinal events, including diarrhea, most commonly occurred early in treatment, were generally time-limited, and infrequently led to discontinuation of HER2-targeted therapy.

Analyses of subsequent anticancer therapies showed greater use of immune checkpoint inhibitors and HER2-targeted therapies in the trastuzumab plus chemotherapy group than in the zanidatamab-containing groups, reflecting that a higher proportion of patients in that arm experienced disease progression and subsequently received additional therapy. These analyses provide important context for interpreting OS outcomes.

Jazz has submitted the Phase 3 HERIZON-GEA-01 data, including this manuscript, to the National Comprehensive Cancer Network (NCCN) for inclusion in the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines).

PD-L1 Subgroup Oral Presentation at ASCO (Free ASCO Whitepaper) 2026

In addition, prespecified PD-L1 subgroup analyses from the Phase 3 HERIZON-GEA-01 trial to be presented at the 2026 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (May 29-June 2, 2026, in Chicago) provide further insight into the impact of zanidatamab-containing combinations across PD-L1-defined subgroups.

Zanidatamab in combination with tislelizumab and chemotherapy demonstrated meaningful improvements in PFS and OS in both PD-L1-positive and PD-L1-negative patients as determined by tumor area positivity (TAP) score and combined positive score (CPS).
With 26 months of follow-up, similarly prolonged PFS and OS were observed in both PD-L1-positive and PD-L1-negative patients compared with the control arm.
Notably, in PD-L1-negative patients (TAP <1%), median OS was 29.7 months with zanidatamab in combination with tislelizumab and chemotherapy compared with 15.8 months with trastuzumab plus chemotherapy, with findings consistent across PD-L1 assessment methods.
In PD-L1-positive patients (TAP≥1%), median OS was 26.4 months with zanidatamab in combination with tislelizumab and chemotherapy compared with 21.2 months with trastuzumab plus chemotherapy, with findings consistent across PD-L1 assessment methods.
About the Phase 3 HERIZON-GEA-01 Trial
HERIZON-GEA-01 (NCT05152147) is a global, randomized, open-label Phase 3 trial, conducted jointly with BeOne Medicines, to evaluate and compare the efficacy and safety of zanidatamab plus chemotherapy, with and without tislelizumab, to trastuzumab plus chemotherapy as first-line treatment for adult patients with advanced/metastatic HER2+ GEA. The trial randomized 914 patients from approximately 225 trial sites in more than 30 countries. Appropriate patients for this trial had unresectable locally advanced, recurrent or metastatic HER2+ GEA (adenocarcinomas of the stomach or esophagus, including the gastroesophageal junction), defined as 3+ HER2 expression by IHC or 2+ HER2 expression by IHC with ISH positivity per central assessment. Patients were randomized to the three trial arms: zanidatamab in combination with chemotherapy and tislelizumab; zanidatamab in combination with chemotherapy; and trastuzumab plus chemotherapy. The trial is evaluating dual primary endpoints, PFS per blinded independent central review (BICR) and OS.

About Gastroesophageal Adenocarcinoma
GEA, including cancers of the stomach, gastroesophageal junction, and esophagus, is the fifth most common cancer worldwide, and approximately 20% of patients have HER2+ disease.[1],[2],[3] HER2+ GEA has high morbidity and mortality, and patients are urgently in need of new treatment options. The overall prognosis for patients with GEA remains poor, with a global five-year survival rate of less than 30% for gastric cancer and about 19% for GEA.[4]

About Ziihera (zanidatamab-hrii)
Ziihera (zanidatamab-hrii) is a bispecific HER2-directed antibody that binds to two extracellular sites on HER2. Binding of zanidatamab- with HER2 results in internalization leading to a reduction in HER2 expression of the receptor on the tumor cell surface. Zanidatamab induces CDC, ADCC, and ADCP. These mechanisms result in tumor growth inhibition and cell death in vitro and in vivo.[5] In the United States, Ziihera is indicated for the treatment of adults with previously treated, unresectable or metastatic HER2-positive (IHC 3+) biliary tract cancer (BTC), as detected by an FDA-approved test.5 The FDA granted accelerated approval for this indication based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).5

Zanidatamab is being developed in multiple clinical trials as a targeted treatment option for patients with solid tumors that express HER2. Zanidatamab is being developed by Jazz and BeOne under license agreements from Zymeworks, which first developed the molecule.  

A supplemental biologics license application for zanidatamab is under FDA Real-Time Oncology Review in first-line HER2+ locally advanced or metastatic GEA. The FDA granted two Breakthrough Therapy designations for zanidatamab’s development: one as a single agent for previously treated HER2 gene-amplified BTC, and one in combination with fluoropyrimidine- and platinum-containing chemotherapy, with or without tislelizumab for first-line HER2+ unresectable locally advanced or metastatic gastric, gastroesophageal junction (GEJ), or esophageal GEA. The FDA also granted two Fast Track designations for zanidatamab: one as a single agent for refractory BTC and one in combination with standard-of-care chemotherapy for first-line GEA. Additionally, zanidatamab has received Orphan Drug designations from the FDA for the treatment of BTC, gastric (including GEJ) cancer, and esophageal cancer, as well as Orphan Drug designations from the European Medicines Agency for the treatment of BTC, gastric/gastroesophageal junction cancer, and esophageal cancer.

Important Safety Information for ZIIHERA

WARNING: EMBRYO-FETAL TOXICITY
Exposure to ZIIHERA during pregnancy can cause embryo-fetal harm. Advise patients
of the risk and need for effective contraception.

WARNINGS AND PRECAUTIONS

Embryo-Fetal Toxicity

ZIIHERA can cause fetal harm when administered to a pregnant woman. In literature reports, use of a HER2-directed antibody during pregnancy resulted in cases of oligohydramnios and oligohydramnios sequence manifesting as pulmonary hypoplasia, skeletal abnormalities, and neonatal death.

Verify the pregnancy status of patients of reproductive potential prior to the initiation of ZIIHERA. Advise pregnant women and females of reproductive potential that exposure to ZIIHERA during pregnancy or within 4 months prior to conception can result in fetal harm. Advise females of reproductive potential to use effective contraception during treatment with ZIIHERA and for 4 months following the last dose of ZIIHERA.

Left Ventricular Dysfunction

ZIIHERA can cause decreases in left ventricular ejection fraction (LVEF). LVEF declined by >10% and decreased to <50% in 4.3% of 233 patients. Left ventricular dysfunction (LVD) leading to permanent discontinuation of ZIIHERA was reported in 0.9% of patients. The median time to first occurrence of LVD was 5.6 months (range: 1.6 to 18.7). LVD resolved in 70% of patients.

Assess LVEF prior to initiation of ZIIHERA and at regular intervals during treatment. Withhold dose or permanently discontinue ZIIHERA based on severity of adverse reactions.

The safety of ZIIHERA has not been established in patients with a baseline ejection fraction that is below 50%.

Infusion-Related Reactions

ZIIHERA can cause infusion-related reactions (IRRs). An IRR was reported in 31% of 233 patients treated with ZIIHERA as a single agent in clinical studies, including Grade 3 (0.4%), and Grade 2 (25%). IRRs leading to permanent discontinuation of ZIIHERA were reported in 0.4% of patients. IRRs occurred on the first day of dosing in 28% of patients; 97% of IRRs resolved within one day.

Prior to each dose of ZIIHERA, administer premedications to prevent potential IRRs. Monitor patients for signs and symptoms of IRR during ZIIHERA administration and as clinically indicated after completion of infusion. Have medications and emergency equipment to treat IRRs available for immediate use.

If an IRR occurs, slow, or stop the infusion, and administer appropriate medical management. Monitor patients until complete resolution of signs and symptoms before resuming. Permanently discontinue ZIIHERA in patients with recurrent severe or life-threatening IRRs.

Diarrhea

ZIIHERA can cause severe diarrhea.

Diarrhea was reported in 48% of 233 patients treated in clinical studies, including Grade 3 (6%) and Grade 2 (17%). If diarrhea occurs, administer antidiarrheal treatment as clinically indicated. Perform diagnostic tests as clinically indicated to exclude other causes of diarrhea. Withhold or permanently discontinue ZIIHERA based on severity.

ADVERSE REACTIONS

Serious adverse reactions occurred in 53% of 80 patients with unresectable or metastatic HER2-positive BTC who received ZIIHERA. Serious adverse reactions in >2% of patients included biliary obstruction (15%), biliary tract infection (8%), sepsis (8%), pneumonia (5%), diarrhea (3.8%), gastric obstruction (3.8%), and fatigue (2.5%). A fatal adverse reaction of hepatic failure occurred in one patient who received ZIIHERA.

The most common adverse reactions in 80 patients with unresectable or metastatic HER2-positive BTC who received ZIIHERA (≥20%) were diarrhea (50%), infusion-related reaction (35%), abdominal pain (29%), and fatigue (24%).

USE IN SPECIFIC POPULATIONS

Pediatric Use

Safety and efficacy of ZIIHERA have not been established in pediatric patients.

Geriatric Use

Of the 80 patients who received ZIIHERA for unresectable or metastatic HER2-positive BTC, there were 39 (49%) patients 65 years of age and older. Thirty-seven (46%) were aged 65-74 years old and 2 (3%) were aged 75 years or older.

No overall differences in safety or efficacy were observed between these patients and younger adult patients.

The full U.S. Prescribing Information for ZIIHERA, including BOXED Warning, is available at: View Source

TEVIMBRA (tislelizumab) is a registered trademark of BeOne Medicines.

(Press release, Jazz Pharmaceuticals, MAY 27, 2026, View Source [SID1234666113])

On May 27, 2026 AbbVie (NYSE: ABBV) reported that the U.S. Food and Drug Administration (FDA) has approved DECNUPAZTM (pivekimab sunirine-pvzy) for the treatment of adult patients with BPDCN, an ultra-rare and aggressive hematologic malignancy with limited treatment options. The approval is supported by data from the Phase 1/2 CADENZA trial, a global study evaluating the safety and efficacy of DECNUPAZ for BPDCN.

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"For patients living with rare cancers, progress in research can be life‑changing," said Roopal Thakkar, executive vice president, research and development, chief scientific officer, AbbVie. "This approval delivers a new option for treating BPDCN and demonstrates our determination to drive meaningful advancements for patients affected by difficult-to-treat cancers."

Patients with BPDCN often present with skin lesions, and the disease can rapidly spread to the bone marrow, lymph nodes, and central nervous system. The disease typically affects adult men aged 60-70 years. Despite initial treatment with intensive chemotherapy, which may include stem cell transplantation, many patients experience relapse, underscoring the need for new treatment options.1

"BPDCN is an aggressive disease with historically limited therapeutic options, particularly for patients whose disease has relapsed or become refractory," said Naveen Pemmaraju, M.D., professor of leukemia, The University of Texas MD Anderson Cancer Center. "Pivekimab sunirine-pvzy is the first and only CD123 targeting ADC that can be initiated in an outpatient setting, offering a meaningful benefit for BPDCN patients in need of new treatment alternatives."

In the Phase 1/2 CADENZA trial, newly diagnosed patients with BPDCN who were treated with DECNUPAZ demonstrated clinically meaningful and durable responses. In newly diagnosed patients with BPDCN (n=33), researchers observed a composite complete response rate of 69.7% with a median duration of response of 9.7 months, with 13 patients (39.4%) who were able to receive post-study treatment stem cell transplant. Patients with relapsed or refractory disease (n=51) had a composite complete response rate of 15.7% with median duration of response rate of 9.2 months, with six patients (11.8%) who were able to receive post-study treatment stem cell transplant.2

Most common adverse reactions (≥20%) were edema, fatigue, musculoskeletal pain, hemorrhage, infusion-related reactions, nausea, and diarrhea. DECNUPAZ has a boxed warning for hepatotoxicity, including hepatic veno-occlusive disease, and warnings and precautions for infusion-related reactions, edema, sulfite allergic reactions, and embryo-fetal toxicity.

About the CADENZA Trial
CADENZA is a Phase 1/2, multicenter, open-label study designed to evaluate the safety, tolerability, pharmacokinetics, immunogenicity, and anti-leukemia activity of pivekimab sunirine-pvzy in patients with CD123-positive hematologic malignancies, including BPDCN. The study was also designed to determine the maximum tolerated dose, recommended Phase 2 dose and dosing schedule for pivekimab sunirine-pvzy monotherapy.3 Results published in the Journal of Clinical Oncology included a total of 84 patients who were part of the trial. Of these, 33 patients with no CNS involvement received DECNUPAZ as a frontline treatment and 51 had relapsed or refractory disease without evidence of active CNS disease. Eleven patients in the frontline treatment group had prior or concurrent cancer diagnoses in addition to BPDCN, making their treatment more complex and challenging.2

About DECNUPAZ (pivekimab sunirine-pvzy)
DECNUPAZ (pivekimab sunirine-pvzy) is a CD123-targeting ADC for the treatment of adult patients diagnosed with the hematological malignancy BPDCN. ADCs are designed to deliver potent cell death-inducing agents called ‘payloads’ directly to the cells expressing a specific protein. CD123 (IL-3Rα) is a protein overexpressed in BPDCN, making it an ideal target for therapy. The payload is a member of the indolinobenzodiazepine pseudodimer class that alkylates DNA and causes single-strand DNA breaks without crosslinking, leading to apoptosis and cell death.4

In October 2020, the FDA granted pivekimab sunirine-pvzy Breakthrough Therapy Designation for relapsed/refractory BPDCN.

DECNUPAZ U.S. Uses and Important Safety Information, Including Boxed Warning4

Use
What is DECNUPAZ?
DECNUPAZ is a prescription medicine used to treat adults with blastic plasmacytoid dendritic cell neoplasm (BPDCN).

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

IMPORTANT SAFETY INFORMATION

What is the most important information I should know about DECNUPAZ?
DECNUPAZ can cause serious side effects, including:

Liver problems (hepatotoxicity), including veno-occlusive disease (blockage of the small veins in the liver), which can be severe, life-threatening, or may lead to death. Your healthcare provider will do blood tests before each dose of DECNUPAZ and during treatment with DECNUPAZ to check for liver problems. Tell your healthcare provider right away if you develop signs or symptoms of liver problems, including:

− yellowing of the skin or eyes

− pain in your stomach (abdomen)

− fast weight gain

− swelling of your stomach

− dark urine

Your healthcare provider will check you for liver problems during your treatment with DECNUPAZ and may provide treatment for your side effects. Your healthcare provider may also delay or stop treatment with DECNUPAZ if you have severe liver problems.

What should I tell my healthcare provider before receiving DECNUPAZ?
Tell your healthcare provider about all of your medical conditions, including if you:

have liver problems
are allergic to sulfites
have asthma
have kidney problems
are pregnant or plan to become pregnant. DECNUPAZ can harm your unborn baby.
Females who are able to become pregnant:

Your healthcare provider will check for pregnancy before you start treatment with DECNUPAZ.
Use effective birth control (contraception) during treatment with DECNUPAZ and for 7 months after your last dose.
Tell your healthcare provider if you become pregnant or think that you may be pregnant during treatment with DECNUPAZ.
Males who have female partners who are able to become pregnant:

Use an effective birth control during treatment with DECNUPAZ and for 4 months after your last dose of DECNUPAZ.
are breastfeeding or plan to breastfeed. It is not known if DECNUPAZ passes into your breast milk. Do not breastfeed during treatment with DECNUPAZ and for 1 month after the last dose.
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Certain medicines may affect DECNUPAZ and increase your risk of side effects.

What are the possible side effects of DECNUPAZ?

DECNUPAZ can cause serious side effects, including:

Liver problems (hepatotoxicity), including veno-occlusive disease (blockage of the small veins in the liver), which can be severe, life-threatening, or may lead to death. Your healthcare provider will do blood tests before each dose of DECNUPAZ and during treatment with DECNUPAZ to check for liver problems. Tell your healthcare provider right away if you develop signs or symptoms of liver problems, including yellowing of the skin or swelling of your stomach.
Infusion-related reactions (IRR). DECNUPAZ can cause serious, life-threatening infusion related reactions. Your healthcare provider will give you medicines the day before and on the day of your infusion of DECNUPAZ to help reduce infusion-related reactions. Your healthcare provider will check you for symptoms of infusion related reactions during your infusion and for at least 4 hours or longer if needed, after your first infusion, and for at least 1 hour after each of your next infusions. Tell your healthcare provider right away if you develop signs or symptoms of infusion related reactions, including:

− shortness of breath

− nausea

− flushing

− chest pain

− fever

− feeling faint or lightheaded

− chills

− vomiting

Fluid retention (edema). DECNUPAZ can cause your body to hold too much fluid during treatment. Your healthcare provider may prescribe water pills (diuretic) if you develop edema. Tell your healthcare provider if you develop new or worsening edema, including:
swelling of your ankles or legs
shortness of breath or difficulty breathing
unusual weight gain
Sulfite allergic reactions. DECNUPAZ contains sodium metabisulfite, a sulfite that may cause severe, life-threatening allergic reactions in some people. Sulfite allergic reactions are more common in people with asthma than in people without asthma. Get medical help right away if you develop hives; itching; rash; swelling of the eyes, tongue, or lips; chest pain; trouble breathing or swallowing.
The most common side effects include:

fluid retention (edema)
feeling tired
muscle, bone, and joint pain

bleeding
infusion-related reactions
nausea
diarrhea
The most common severe abnormal laboratory test results with DECNUPAZ include:

decreased white blood cell counts
decreased platelet counts

decreased red blood cell counts
increased blood sugar level
Your healthcare provider may decrease your dose, delay your infusion, or permanently stop treatment with DECNUPAZ if you have side effects.

DECNUPAZ may cause fertility problems in males and females, which may affect your ability to have children. Talk to your healthcare provider if you have concerns about fertility.

These are not all the possible side effects of DECNUPAZ. Call your doctor for medical advice about side effects.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

Please see full Prescribing Information, including BOXED WARNING.

(Press release, AbbVie, MAY 27, 2026, View Source [SID1234666112])

On May 27, 2026 AstraZeneca reported that the US Food and Drug Administration (FDA) has informed that it will extend the Prescription Drug User Fee Act (PDUFA) date to review additional data requested to support the New Drug Application (NDA) for camizestrant in combination with a cyclin-dependent kinase (CDK) 4/6 inhibitor (palbociclib, ribociclib or abemaciclib) for the 1st-line treatment of patients with hormone receptor (HR)-positive, HER2-negative advanced breast cancer whose tumours have an emergent ESR1 mutation.

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The NDA is based on positive results from the pivotal SERENA-6 Phase III trial presented at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting and simultaneously published in The New England Journal of Medicine.1 The FDA granted Breakthrough Therapy Designation for the camizestrant combination in this setting in May 2025.

In April 2026, the FDA’s Oncologic Drugs Advisory Committee did not reach a majority vote in favour of the benefit of switching to camizestrant in combination with a CDK4/6 inhibitor after detection of an ESR1 mutation in circulating tumour DNA (ctDNA) prior to radiographic progression, based on the SERENA-6 Phase III trial. Subsequently, the Company has provided additional analyses requested by the FDA in support of the application, including ctDNA clearance data linked to longer-term efficacy outcomes that will be presented on 02 June at ASCO (Free ASCO Whitepaper) 2026.

Susan Galbraith, Executive Vice President, Oncology Haematology R&D, AstraZeneca, said: "We are committed to continuously advancing the clinical landscape in oncology in pursuit of improving outcomes for patients. The SERENA-6 treatment strategy epitomises this approach by monitoring patients for the emergence of ESR1 mutations in ctDNA and testing if a switch of endocrine backbone therapy at this point improves outcomes. We look forward to continuing the dialogue with the FDA in order to bring the benefits of camizestrant with this innovative treatment strategy to eligible patients in the US as quickly as possible."

On 22 May, the European Medicines Agency’s Committee for Medicinal Products for Human Use adopted a positive opinion recommending approval of the camizestrant combination in this setting based on the results of the SERENA-6 Phase III trial.

Camizestrant is approved in the United Arab Emirates and Saudi Arabia in this setting. Regulatory applications are also currently under review in Japan and several other countries based on the SERENA-6 Phase III trial.

Notes

HR-positive breast cancer
Breast cancer is the second most common cancer and one of the leading causes of cancer-related deaths worldwide.2 More than two million patients were diagnosed with breast cancer in 2022, with more than 665,000 deaths globally.2 In the US, breast cancer is the most common cancer in women, with more than 300,000 new cases of the disease diagnosed annually, and more than 42,000 deaths.3 While survival rates are high for those diagnosed with early breast cancer, only about 30% of patients diagnosed with or who progress to metastatic disease are expected to live five years following diagnosis.4

HR-positive breast cancer, characterised by the expression of estrogen or progesterone receptors, or both, is the most common subtype of breast cancer with 70% of tumours considered HR-positive and HER2-negative.4 Estrogen receptor (ERs) often drive the growth of HR-positive breast cancer cells.5

Globally, approximately 200,000 patients with HR-positive breast cancer are treated with a medicine in the 1st-line setting; most frequently with endocrine therapies that target ER-driven disease, which are often paired with CDK4/6 inhibitors.6-8 In the US, approximately 37,000 patients with HR-positive metastatic breast cancer are treated with these therapies in the 1st-line setting.6-8 However, resistance to these therapies develops in many patients.8 Once this occurs, treatment options are limited and survival rates are low with approximately 36% of patients anticipated to live beyond five years after diagnosis.4,8

Mutations in the ESR1 gene are a key driver of endocrine resistance and are associated with poor outcomes, emerging during treatment of the disease and becoming more prevalent as the disease progresses.9,10 Approximately 30% of patients with endocrine sensitive HR-positive disease develop ESR1 mutations during 1st-line treatment before disease progression.6

The optimisation of endocrine therapy and overcoming resistance to enable patients to continue benefiting from these treatments, as well as identifying new therapies for those who are less likely to benefit, are active areas of focus for breast cancer research.

SERENA-6
SERENA-6 is a Phase III, double-blind, randomised trial evaluating the efficacy and safety of camizestrant in combination with a CDK4/6 inhibitor (palbociclib, ribociclib or abemaciclib) versus treatment with an AI (anastrozole or letrozole) in combination with a CDK4/6 inhibitor (palbociclib, ribociclib or abemaciclib) in patients with HR-positive, HER2-negative advanced breast cancer (patients with either locally advanced disease, or metastatic disease) whose tumours have an emergent ESR1 mutation.

The global trial enrolled 315 adult patients with histologically confirmed HR-positive, HER2-negative advanced breast cancer, undergoing treatment with an AI in combination with a CDK4/6 inhibitor as 1st-line treatment. The primary endpoint of the SERENA-6 trial is PFS as assessed by investigator, with secondary endpoints including OS, and PFS2 by investigator assessment.

Camizestrant
Camizestrant is an investigational, potent, next-generation oral selective estrogen receptor degrader (SERD) and complete ER antagonist that is currently in Phase III trials for the treatment of HR-positive breast cancer.

AstraZeneca’s broad, robust and innovative clinical development programme, including the SERENA-6, SERENA-4, CAMBRIA-1 and CAMBRIA-2 trials, is evaluating the safety and efficacy of camizestrant when used as a monotherapy or in combination with CDK4/6 inhibitors to address a number of areas of unmet need in HR-positive, HER2-negative breast cancer.

Camizestrant has demonstrated anti-cancer activity across a range of preclinical models, including those with ER-activating mutations. In the SERENA-2 Phase II trial, camizestrant demonstrated a statistically significant and clinically meaningful improvement in PFS versus Faslodex (fulvestrant) in the overall trial population, including in patients with ESR1 tumour mutations irrespective of prior treatment with CDK4/6 inhibitors in patients with ER-positive locally advanced or metastatic breast cancer, previously treated with endocrine therapy. The SERENA-1 Phase I trial demonstrated that camizestrant is well tolerated and has a promising anti-tumour profile when administered alone or in combination with palbociclib, ribociclib and abemaciclib; three widely used CDK4/6 inhibitors.

(Press release, AstraZeneca, MAY 27, 2026, View Source [SID1234666110])

On May 27, 2026 BeOne Medicines Ltd. (Nasdaq: ONC; HKEX: 06160; SSE: 688235), a global oncology company, reported that data from HERIZON-GEA-01 were published in The New England Journal of Medicine and will be presented in an oral presentation (Rapid Oral Abstract: 4010) at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting on June 1, 2026, in Chicago. The HERIZON-GEA-01 clinical trial evaluated ZIIHERA (zanidatamab) plus chemotherapy, with and without TEVIMBRA (tislelizumab), compared with the control arm of trastuzumab plus chemotherapy as first-line treatment for advanced/metastatic HER2+ gastroesophageal adenocarcinoma (GEA).

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Dr. Sun Young Rha, Professor of Medical Oncology at the Yonsei Cancer Center, Yonsei University College of Medicine, Seoul, South Korea, senior author of the NEJM manuscript and first author of the ASCO (Free ASCO Whitepaper) abstract, said:

"Results from the HERIZON-GEA-01 published in The New England Journal of Medicine and presented in an oral presentation at ASCO (Free ASCO Whitepaper) provide new data about the regimen of tislelizumab added to zanidatamab plus chemotherapy, which demonstrated meaningfully improved outcomes for patients with HER2-positive gastroesophageal adenocarcinoma. In particular, the findings show that this regimen resulted in a survival benefit, even in patients with PD-L1 <1%. This combination has the potential to be an important new treatment option in areas of high unmet need in HER2+ GEA."

Key findings published in The New England Journal of Medicine

Overall survival (OS): A statistically significant and clinically meaningful improvement in OS with ZIIHERA plus TEVIMBRA and chemotherapy, reaching a median OS of 26.4 months; mOS of 24.4 months was reported with ZIIHERA plus chemotherapy, and 19.2 months with the control arm.
Progression-free survival (PFS): A statistically significant and clinically meaningful improvement in PFS in both ZIIHERA-containing arms with a median PFS of 12.4 months.
Duration of Response (DoR): Median DoR of 20.7 months with ZIIHERA and TEVIMBRA plus chemotherapy; median DoR of 14.3 months with ZIIHERA plus chemotherapy and 8.3 months with the control arm.
Dr. Geoffrey Ku, Associate Attending physician on the Gastrointestinal Oncology Service in the Department of Medicine at Memorial Sloan Kettering Cancer Center, author of the NEJM manuscript and the ASCO (Free ASCO Whitepaper) abstract, said:

"This practice-changing study demonstrates that zanidatamab is clearly superior to trastuzumab, with a manageable safety profile, in HER2-positive GEA. Moreover, the addition of tislelizumab contributes to the prolongation of overall survival and remarkable durability of responses, with benefit in both PD-L1 positive and negative tumors. If approved, the combination of zanidatamab, tislelizumab and chemotherapy should become the standard of care in untreated metastatic or locally advanced HER2-positive GEA patients irrespective of the tumor PD-L1 status."

Oral presentation with new data at ASCO (Free ASCO Whitepaper) 2026 demonstrates benefit regardless of PD-L1 status

With 26 months of follow-up, ZIIHERA plus TEVIMBRA and chemotherapy meaningfully improved PFS and OS were observed in both PD-L1-positive and PD-L1-negative patients compared with the control arm; data were consistent between tumor area positivity (TAP) and combined positive score (CPS).
In PD-L1 TAP <1% and ≥1% patients, the 18-month PFS was 50.3% and 42.6%, respectively, and the 24-month OS was 63.7% and 53.5% with ZIIHERA plus TEVIMBRA and chemotherapy.
In PD-L1-negative patients (TAP <1%), median OS was 29.7 months with ZIIHERA plus TEVIMBRA and chemotherapy compared with 15.8 months with the control arm. In PD-L1-positive patients (TAP≥1%), median OS was 26.4 months with ZIIHERA plus TEVIMBRA and chemotherapy compared with 21.2 months in the control arm. Findings were consistent across PD-L1 assessment methods.
In TAP<1%, the ZIIHERA plus TEVIMBRA and chemotherapy regimen resulted in a mPFS of 18.5 months compared with mPFS of 7.9 months in the control arm, while in TAP≥1% patients, the mPFS was 11.3 months vs. mPFS of 8.3 months in the control arm
Mark Lanasa, M.D., Ph.D., Chief Medical Officer, Solid Tumors at BeOne Medicines, said:

"The HERIZON-GEA-01 results – now published in The New England Journal of Medicine with a detailed sub-group analysis presented in an oral session at ASCO (Free ASCO Whitepaper) – strengthen the evidence supporting the role of TEVIMBRA in driving a sustained and statistically significant overall survival benefit. With a median OS of more than 26 months, unprecedented in this disease, the TEVIMBRA-containing arm is positioned as a compelling new therapeutic approach in a disease where there remains a critical unmet need."

The safety findings for the ZIIHERA plus TEVIMBRA and chemotherapy arm were generally consistent with the known effects of HER2-directed therapy and immunotherapy, and no new safety signals were identified. Diarrhea was the most common Grade ≥3 treatment-related adverse event (TRAE) in 24.5% of patients with ZIIHERA plus TEVIMBRA and chemotherapy, 20.0% of patients in the ZIIHERA plus chemotherapy arm, and 12.9% in the trastuzumab plus chemotherapy arm, noting that the median treatment duration was longest for the triplet arm at 43.1 weeks (vs. 31.0 weeks with ZIIHERA plus chemotherapy and 30.0 weeks in the control arm). A mandatory anti-diarrheal prophylaxis was established during the first cycle, and discontinuation rates due to drug-related diarrhea were relatively low at 4.1%, 1.3%, and 0% of patients, respectively, with most diarrhea events occurring early in the trial.

Regulatory Status

The U.S. FDA has accepted a supplemental Biologics License Applications (sBLA) for TEVIMBRA and has granted it priority review. In addition, the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA) has accepted sBLAs for ZIIHERA and for TEVIMBRA for the first-line treatment of advanced/metastatic HER2+ GEA. BeOne holds the rights to ZIIHERA in Asia (excluding India and Japan), Australia, and New Zealand, and intends to work with authorities in these markets to expedite regulatory submissions.

About the HERIZON-GEA-01 Phase 3 Trial

HERIZON-GEA-01 (NCT05152147) is a global, randomized, open-label Phase 3 trial, conducted jointly with Jazz Pharmaceuticals, to evaluate and compare the efficacy and safety of ZIIHERA plus chemotherapy, with and without TEVIMBRA, to the standard of care (trastuzumab plus chemotherapy) as first-line treatment for adult patients with advanced/metastatic HER2+ GEA. The trial randomized 914 patients from approximately 300 trial sites in more than 30 countries. Patients for this trial had unresectable locally advanced, recurrent or metastatic HER2+ GEA (adenocarcinomas of the stomach or esophagus, including the gastroesophageal junction), defined as 3+ HER2 expression by IHC or 2+ HER2 expression by IHC with ISH positivity per central assessment. Patients were randomized to the three trial arms: ZIIHERA in combination with chemotherapy and TEVIMBRA; ZIIHERA in combination with chemotherapy; and trastuzumab plus chemotherapy. The trial is evaluating dual primary endpoints, PFS per blinded independent central review (BICR) and OS.

About Gastroesophageal Adenocarcinoma

Gastroesophageal adenocarcinoma (GEA), which includes cancers of the stomach, gastroesophageal junction, and esophagus, is the fifth most common cancer worldwide. Approximately 20% of GEA patients have HER2-positive disease.1,2,3, which has high morbidity and mortality, and patients are urgently in need of new treatment options. The overall prognosis for patients with GEA remains poor, with a global five-year survival rate of less than 30% for gastric cancer and about 19% for GEA.4

About ZIIHERA (zanidatamab)

ZIIHERA (zanidatamab) is a bispecific human epidermal growth factor receptor 2, or HER2-directed antibody that binds to two extracellular sites on HER2. Binding of zanidatamab with HER2 results in internalization leading to a reduction in HER2 expression of the receptor on the tumor cell surface. Zanidatamab induces complement-dependent cytotoxicity (CDC), antibody-dependent cellular cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP). These mechanisms result in tumor growth inhibition and cell death in vitro and in vivo.5

Zanidatamab is being developed in multiple clinical trials as a targeted treatment option for patients with solid tumors that express HER2. Zanidatamab is approved in China for the treatment of patients who have unresectable, locally advanced, or metastatic HER2-high expression (IHC 3+) biliary tract cancer (BTC) and who have received prior systemic therapy. ZIIHERA has also been granted accelerated approval in the U.S. and conditional marketing authorization in the European Union for eligible BTC patients. Zanidatamab is being developed by Jazz and BeOne under license agreements from Zymeworks, which first developed the molecule. BeOne has licensed zanidatamab from Zymeworks in Asia (excluding India and Japan), Australia and New Zealand. Jazz Pharmaceuticals has rights in all other regions.

ZIIHERA is a registered trademark of Zymeworks BC Inc.

About TEVIMBRA (tislelizumab)

TEVIMBRA is a uniquely designed humanized immunoglobulin G4 (IgG4) anti-programmed cell death protein 1 (PD-1) monoclonal antibody with high affinity and binding specificity against PD-1. It is designed to minimize binding to Fc-gamma (Fcγ) receptors on macrophages, helping to aid the body’s immune cells to detect and fight tumors.

TEVIMBRA is the foundational asset of BeOne’s solid tumor portfolio and has shown potential across multiple tumor types and disease settings. The global TEVIMBRA clinical development program includes almost 15,000 patients enrolled to date in 30+ countries and regions across 71 trials, including 21 registration-enabling studies. TEVIMBRA is approved in over 50 countries, and more than 2 million patients have been treated globally.

Select Important Safety Information

Serious and sometimes fatal adverse reactions occurred with TEVIMBRA treatment. Warnings and precautions include severe and fatal immune-mediated adverse reactions, including pneumonitis, colitis, hepatitis, endocrinopathies, dermatologic adverse reactions, nephritis with renal dysfunction, and solid organ transplant rejection. Other warnings and precautions include infusion-related reactions, complications of allogeneic HSCT, and embryo-fetal toxicity.

Please see full U.S. Prescribing Information including the U.S. Medication Guide.

The information in this press release is intended for a global audience. Product indications vary by region.

(Press release, BeOne Medicines, MAY 27, 2026, View Source [SID1234666109])

On May 27, 2026 Precigen, Inc. (Nasdaq: PGEN), a commercial-stage biopharmaceutical company specializing in the advancement of innovative precision medicines to improve the lives of patients, reported that the US Food and Drug Administration (FDA) has granted orphan drug exclusivity for PAPZIMEOS (zopapogene imadenovec-drba) for the treatment of adults with recurrent respiratory papillomatosis (RRP). PAPZIMEOS was granted full approval by the FDA in August 2025, becoming the first and only approved treatment for adults with RRP, a rare, chronic and debilitating disease. PAPZIMEOS is commercially available in the US and is being prescribed nationwide across both major medical centers and community practices, with patients spanning a range of disease severities actively receiving treatment.

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Orphan drug exclusivity is granted to certain drugs and biologics approved for rare diseases or conditions that affect fewer than 200,000 people in the United States. The orphan drug exclusivity granted by the FDA for PAPZIMEOS for the treatment of adults with RRP is effective through August 14, 2032, providing seven years of market exclusivity in the US from the PAPZIMEOS approval date.

"We appreciate the FDA’s recognition of seven years of orphan drug exclusivity for PAPZIMEOS, which is a testament to the groundbreaking pivotal study data and the transformative potential of PAPZIMEOS in addressing the root cause of this rare disease," said Helen Sabzevari, PhD, President and CEO of Precigen. "This regulatory exclusivity, together with Precigen’s patent portfolio covering PAPZIMEOS and its therapeutic use, enhances the product’s value by strengthening market protection and long-term revenue potential, which in turn supports continued innovation for rare diseases."

About RRP
RRP is a rare, debilitating, and potentially life-threatening disease of the upper and lower respiratory tract caused by chronic HPV 6 or HPV 11 infection. RRP can lead to severe voice disturbance, compromised airways, and recurrent post-obstructive pneumonia. Although rare, RRP has the potential for transformation to malignant cancer and can be fatal. Management of RRP has primarily consisted of repeated surgeries, which do not address the underlying cause of the disease and can be associated with significant morbidity as well as significant patient and health system burden. As the number of lifetime surgeries increases, the risk for irreversible iatrogenic laryngeal injury increases with each surgery, and patients may undergo hundreds of these surgeries over their lifetimes. RRP can impact patients’ work and social lives, financial stability, and mental health. Patients with RRP can experience substantial impacts to daily living with decreased quality of life and high health care utilization. Based on an internal analysis of claims data and electronic health records, there are approximately 27,000 adult RRP patients in the US.

About PAPZIMEOS (zopapogene imadenovec-drba), for subcutaneous injection only
PAPZIMEOS is the first and only FDA-approved therapy for the treatment of adults with RRP and the first and only approved therapy to address the root cause of RRP. PAPZIMEOS is a non-replicating adenoviral vector-based immunotherapy designed to express a fusion antigen comprising selected regions of human papillomavirus (HPV) types 6 and 11 proteins. PAPZIMEOS is designed to generate an immune response directed against HPV 6 and HPV 11 proteins in patients with RRP. Discovered and designed in Precigen’s labs using Precigen’s proprietary AdenoVerse therapeutic platform, PAPZIMEOS represents a new therapeutic paradigm for RRP.

Indication and Important Safety Information

What is PAPZIMEOS?
PAPZIMEOS is a type of immunotherapy used to treat a condition called recurrent respiratory papillomatosis (RRP) in adults.

What is the most important information I should know about PAPZIMEOS?
Some people may have a reaction to the shot. Signs and symptoms may include redness, pain, swelling, itching, or warmth where the shot was given. After your first treatment, your healthcare provider will watch you for at least 30 minutes to make sure you’re feeling okay.

Please contact your doctor immediately if you develop an infection, the reaction to your shot worsens, or you experience any of the below symptoms, which may indicate a systemic allergic reaction:

Difficulty breathing
Widespread rash
Facial swelling
Thrombotic events (blood clots that block your blood vessels) may occur after your PAPZIMEOS shot. Please notify your doctor immediately if you have the following symptoms:

Shortness of breath
Chest pain
Leg swelling
Persistent abdominal pain
Severe or persistent headaches
Blurred vision
What should I know before taking PAPZIMEOS?
Before taking PAPZIMEOS, tell your healthcare provider about all of your medical conditions, including:

If you are pregnant or plan to become pregnant because it is not known if PAPZIMEOS will harm the unborn baby.
If you are breastfeeding or plan to breastfeed. It is unknown if PAPZIMEOS is present in breast milk, or how it affects the breastfeeding child or milk production. Talk to your healthcare provider about the best way to feed your baby during treatment with PAPZIMEOS.
What are the most common side effects of PAPZIMEOS?
The most common side effects include:

Pain, redness, or swelling where the shot was given
Feeling tired
Chills
Fever
Muscle aches
Nausea (feeling sick)
Headache
Increased heart rate
Diarrhea
Vomiting
Sweating a lot
These are not all of the possible side effects of PAPZIMEOS. Call your healthcare provider for medical advice about side effects. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You may also report side effects to Precigen, Inc. at 1-855-PGE-NRRP (1-855-743-6777).

(Press release, Precigen, MAY 27, 2026, View Source [SID1234666108])