Latest news with #JazzPharmaceuticalsplc
Yahoo
12-06-2025
- Business
- Yahoo
Jazz Pharmaceuticals Releases Unexpected Phase 4 Results Highlighting Xywav®
Jazz Pharmaceuticals plc (NASDAQ:JAZZ) is one of the 13 Biotech Stocks with Huge Upside Potential. Jazz Pharmaceuticals plc (NASDAQ:JAZZ) is one of the stocks with the biggest upside. It presented late-breaking Phase 4 data on Xywav® at SLEEP 2025, which included findings from the XYLO and DUET trials. A biopharmaceutical scientist in their lab, studying a newly-diagnosed therapy-related acute myeloid leukemia. . In the XYLO study (n=43), moving from high-sodium to low-sodium oxybate resulted in a substantial decrease in 24-hour ambulatory systolic blood pressure (−4.1 mmHg; P=0.0019). SBP reduction during the day (−5.1 mmHg; P=0.0003) and while seated (−9.2 mmHg; P<0.0001) were secondary objectives. The TEAEs were moderate or mild. DUET data from 24 subjects taking 9-12g Xywav nightly displayed improvement in excessive daytime sleepiness and narcolepsy severity with an average dose of 11.2g during the stable period. Xywav is the sole FDA-approved low-sodium oxybate for cataplexy or EDS in narcolepsy and idiopathic hypersomnia. Xywav reduces nightly sodium intake by up to 92% compared to Xyrem®, lowering cardiovascular risk, which is a serious issue in narcolepsy patients. Both indications were awarded Orphan Drug Exclusivity by the FDA. Jazz Pharmaceuticals plc (NASDAQ:JAZZ)'s data underscores how crucial it is to lower sodium intake in long-term sleep disorder treatment plans. While we acknowledge the potential of JAZZ as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock. READ NEXT: 10 High-Growth EV Stocks to Invest In and 13 Best Car Stocks to Buy in 2025. Disclosure. None. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
09-06-2025
- Health
- Yahoo
Jazz Pharmaceuticals Presents Late-Breaking Phase 4 Data Showcasing Xywav® (calcium, magnesium, potassium, and sodium oxybates) Oral Solution Treatment Outcomes in Narcolepsy at SLEEP 2025
First presentation of the Phase 4 XYLO switch study reports blood pressure reductions in patients with narcolepsy when switching from twice-nightly high- to low-sodium oxybate Novel intermediate analysis from the DUET trial cohort of patients taking >9 grams evaluated safety and changes in daytime sleepiness in adults with narcolepsy taking Xywav dosages of 9-12 grams per night For U.S. media and investors only DUBLIN, June 9, 2025 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced late-breaking Phase 4 data evaluating treatment benefits of Xywav® (calcium, magnesium, potassium, and sodium oxybates) oral solution in people with narcolepsy. These results are two of Jazz's four late-breaking abstracts presented today as oral presentations at SLEEP 2025. The four late-breaking abstracts, selected for their scientific quality and innovation, comprise all industry-sponsored late-breaking oral presentations selected by the Associated Professional Sleep Societies (APSS). Xywav is the only low-sodium oxybate approved by the U.S. Food and Drug Administration for the treatment of cataplexy or excessive daytime sleepiness (EDS) in patients 7 years of age and older with narcolepsy and for adults with idiopathic hypersomnia. The Xywav label recommends a nightly dose of 6-9 grams per night. "People living with narcolepsy have an increased risk of developing cardiovascular and cardiometabolic comorbidities," said Richard J. Kovacs, MD, MACC, Chief Medical Officer, American College of Cardiology and Q.E. and Sally Russell Professor of Cardiology, Indiana University School of Medicine, and XYLO Steering Committee member. "Results from the XYLO study reinforce the importance of monitoring cardiovascular indicators, including blood pressure, and the need to minimize exposure to excess sodium in this at-risk population. Reducing cardiovascular risk and disease is an important goal for all healthcare providers." "People with narcolepsy live with a complex, severe disorder and often must combat excessive daytime sleepiness and associated symptoms — but the challenges they face are not only limited to sleep," said Kelvin Tan, MB BCh, MRCPCH, chief medical affairs officer of Jazz Pharmaceuticals. "Results from the XYLO interim analysis add to the overwhelming body of evidence demonstrating the positive implications of limiting unnecessary sodium intake, and emphasize how choosing low-sodium oxybate, Xywav, rather than twice-nightly high-sodium oxybate helps reduce excessive sodium burden, a modifiable risk factor for cardiovascular disease, in people with narcolepsy." Phase 4 XYLO Results Show Impact of Switching from Twice-Nightly High- to Low-Sodium Oxybate on Ambulatory Blood Pressure in People with NarcolepsyThe open-label, single arm Phase 4 XYLO switch trial (n=43) met its primary endpoint of change in mean 24-hour ambulatory systolic blood pressure (SBP) from baseline (taking twice-nightly high-sodium oxybate) to end-of-treatment (after six weeks on low-sodium oxybate, Xywav), with a −4.1 (−6.9, −1.4; P=0.0019) mmHg change. These results show that switching from twice-nightly high-sodium oxybate to the same dosage of low-sodium oxybate, Xywav, for approximately 6 weeks reduced daily treatment-related sodium intake and was associated with clinically meaningful blood pressure reductions in participants with narcolepsy, which was paralleled by 24-hour urinary sodium reduction. XYLO results are consistent with the extensive body of evidence on the benefits of reducing sodium intake. The study also achieved key secondary endpoints, including mean change in ambulatory daytime SBP (-5.1 mmHg; P=0.0003) and mean change in seated resting (in-office) SBP (-9.2 mmHg; (P<0.0001). The change in mean nighttime ambulatory SBP was −2.0 (P=0.1265) mmHg. Exploratory endpoints evaluated change in 24-hour, daytime ambulatory, seated resting, and nighttime ambulatory diastolic blood pressure. Overall, treatment-emergent adverse events (TEAEs) occurred in 32.8% of participants and were all mild or moderate in severity and consistent with the known safety profile of Xywav. Phase 4 DUET Data Evaluated Effectiveness and Safety of Xywav in Adults with Narcolepsy Taking Dosages of 9-12 GramsThis intermediate analysis, which evaluates a cohort of adults with narcolepsy from the DUET (Develop hypersomnia Understanding by Evaluating low-sodium oxybate Treatment) trial, demonstrated improvements in EDS on the Epworth Sleepiness Scale, the study's primary endpoint, in 24 participants taking 9-12 grams of Xywav twice-nightly, as compared to 9 grams at baseline. The current recommended dosage of Xywav for adults with narcolepsy is 6-9 grams per night. Following dose optimization, the average Xywav dose during the stable-dose period was 11.2 g/night. Participants with narcolepsy in this intermediate cohort analysis also experienced improvements on the Narcolepsy Severity Scale. Additionally, participants showed minimal changes in the number of central apnea events, mean oxygen saturation (SpO2) levels, or the mean percent of total sleep time with SpO2 <90% from BL to EOT. The DUET trial is a Phase 4, prospective, single-arm, open-label study to assess the effect of Xywav treatment on EDS, polysomnography parameters, and functional outcomes in adults with narcolepsy or IH. Overall, TEAEs were all mild or moderate and consistent with the known safety profile of Xywav at lower dosages. The full abstracts will be available online at About NarcolepsyNarcolepsy is a chronic, debilitating neurologic sleep disorder characterized by the inability to maintain continuous sleep at night and sustained wakefulness throughout the day. This leads to symptoms that can include fragmented or disrupted nighttime sleep, excessive daytime sleepiness, and cataplexy.1 Patients with EDS due to narcolepsy experience sleep attacks, called cataplexy, and, despite fighting the urge to sleep, may unintentionally fall asleep for short periods.2,3 These sleep attacks may happen at inappropriate or potentially dangerous times such as during driving, cycling, eating, or mid-conversation.4 There is no cure for narcolepsy, therefore this EDS is lifelong and has a substantial negative impact on a person's ability to function psychologically, socially and professionally.5 Patients with narcolepsy are at increased risk for hypertension, cardiometabolic morbidity, stroke, myocardial infarction, heart failure, cardiac arrest, and death.6,7,8,9 As narcolepsy is a chronic condition that requires lifelong, nightly treatment, early access to an effective, low-sodium treatment can transform lives and reduce the impact of narcolepsy on a person's physical and mental health.5 About Xywav® (calcium, magnesium, potassium, and sodium oxybates) oral solutionXywav is the only low-sodium oxybate approved by the U.S. Food and Drug Administration (FDA) for the treatment of cataplexy or excessive daytime sleepiness (EDS) in patients 7 years of age and older with narcolepsy. The FDA recognized seven years of Orphan Drug Exclusivity for Xywav for the treatment of cataplexy or EDS in patients 7 years of age and older with narcolepsy. The Office of Orphan Product Development (OOPD) at the FDA also published its summary of clinical superiority findings for Xywav for the treatment of cataplexy or EDS in patients 7 years of age and older with narcolepsy by means of greater cardiovascular safety compared to Xyrem® (sodium oxybate) oral solution. The decision of the OOPD is based on the FDA findings that Xywav provides a greatly reduced chronic sodium burden compared to Xyrem. Xywav has 131 mg of sodium at the maximum recommended nightly dose whereas other high sodium oxybates have 1640 mg at the equivalent dose. Xywav is comprised of a unique composition of cations resulting in 92% less sodium, or a reduction of approximately 1,000 to 1,500 mg/night at the recommended dose range of 6 g to 9 g/night. Xywav is the only oxybate therapy that does not carry a warning in the label related to use in patients sensitive to high sodium intake. Xywav is also the first and only U.S. FDA-approved treatment option for idiopathic hypersomnia in adults. The FDA recognized seven years of Orphan Drug Exclusivity for Xywav for the treatment of idiopathic hypersomnia in adults. Xywav is the only FDA-approved treatment studied across the multiple symptoms of idiopathic hypersomnia, such as EDS, sleep inertia (severe grogginess or confusion when waking up), long sleep duration and cognitive impairment. Xywav can be administered as a twice- or once-nightly regimen for the treatment of idiopathic hypersomnia in adults. The exact mechanism of action of Xywav in the treatment of adults with idiopathic hypersomnia and of cataplexy and EDS in narcolepsy is unknown. It is hypothesized that the therapeutic effects of Xywav are mediated through GABAB actions during sleep at noradrenergic and dopaminergic neurons, as well as thalamocortical neurons.10 The U.S. Drug Enforcement Agency (DEA) has designated Xywav as a Schedule III medicine. The DEA defines Schedule III drugs, substances, or chemicals as drugs with a moderate to low potential for physical and psychological dependence.10,11 Because of the risks of central nervous system (CNS) depression and abuse and misuse, Xywav is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the XYWAV and XYREM REMS. Important Safety Information for Xywav WARNING: CENTRAL NERVOUS SYSTEM DEPRESSION and ABUSE AND MISUSE. Central Nervous System DepressionXYWAV is a CNS depressant. Clinically significant respiratory depression and obtundation may occur in patients treated with XYWAV at recommended doses. Many patients who received XYWAV during clinical trials in narcolepsy and idiopathic hypersomnia were receiving CNS stimulants. Abuse and MisuseThe active moiety of XYWAV is oxybate or gamma-hydroxybutyrate (GHB). Abuse or misuse of illicit GHB, either alone or in combination with other CNS depressants, is associated with CNS adverse reactions, including seizure, respiratory depression, decreases in the level of consciousness, coma, and death. Because of the risks of CNS depression and abuse and misuse, XYWAV is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the XYWAV and XYREM REMS. ContraindicationsXYWAV is contraindicated in combination with sedative hypnotics or alcohol and in patients with succinic semialdehyde dehydrogenase deficiency. Warnings and PrecautionsCentral Nervous System DepressionThe concurrent use of XYWAV with other CNS depressants, including but not limited to opioid analgesics, benzodiazepines, sedating antidepressants or antipsychotics, sedating anti-epileptic drugs, general anesthetics, muscle relaxants, and/or illicit CNS depressants, may increase the risk of respiratory depression, hypotension, profound sedation, syncope, and death. If use of these CNS depressants in combination with XYWAV is required, dose reduction or discontinuation of one or more CNS depressants (including XYWAV) should be considered. In addition, if short-term use of an opioid (eg, post- or perioperative) is required, interruption of treatment with XYWAV should be considered. After first initiating treatment and until certain that XYWAV does not affect them adversely (eg, impair judgment, thinking, or motor skills), caution patients against hazardous activities requiring complete mental alertness or motor coordination such as operating hazardous machinery, including automobiles or airplanes. Also caution patients against these hazardous activities for at least 6 hours after taking XYWAV. Patients should be queried about CNS depression-related events upon initiation of XYWAV therapy and periodically thereafter. Abuse and MisuseXYWAV is a Schedule Ill controlled substance. The active moiety of XYWAV is oxybate, also known as gamma-hydroxybutyrate (GHB), a Schedule I controlled substance. Abuse of illicit GHB, either alone or in combination with other CNS depressants, is associated with CNS adverse reactions, including seizure, respiratory depression, decreases in the level of consciousness, coma, and death. The rapid onset of sedation, coupled with the amnestic features of GHB particularly when combined with alcohol, has proven to be dangerous for the voluntary and involuntary user (eg, assault victim). Physicians should carefully evaluate patients for a history of drug abuse and follow such patients closely. XYWAV and XYREM REMSBecause of the risks of central nervous system depression and abuse and misuse, XYWAV is available only through a restricted distribution program called the XYWAV and XYREM REMS. Notable requirements of the XYWAV and XYREM REMS include the following: Healthcare Providers who prescribe XYWAV are specially certified XYWAV will be dispensed only by the central pharmacy that is specially certified XYWAV will be dispensed and shipped only to patients who are enrolled in the XYWAV and XYREM REMS with documentation of safe use Further information is available at or 1-866-997-3688. Respiratory Depression and Sleep-Disordered BreathingXYWAV may impair respiratory drive, especially in patients with compromised respiratory function. In overdoses of oxybate and with illicit use of GHB, life-threatening respiratory depression has been reported. Increased apnea and reduced oxygenation may occur with XYWAV administration in adult and pediatric patients. A significant increase in the number of central apneas and clinically significant oxygen desaturation may occur in patients with obstructive sleep apnea treated with XYWAV. Prescribers should be aware that sleep-related breathing disorders tend to be more prevalent in obese patients, in men, in postmenopausal women not on hormone replacement therapy, and among patients with narcolepsy. Depression and SuicidalityIn Study 1, the randomized-withdrawal clinical trial in adult patients with narcolepsy (n=201), depression and depressed mood were reported in 3% and 4%, respectively, of patients treated with XYWAV. Two patients (1%) discontinued XYWAV because of depression. In most cases, no change in XYWAV treatment was required. In Study 2, the randomized-withdrawal clinical trial in adult patients with idiopathic hypersomnia (n=154), depression and depressed mood were reported in 1% and 3%, respectively, of patients treated with XYWAV. All patients continued XYWAV treatment. Two suicides and two attempted suicides occurred in adult clinical trials with oxybate (same active moiety as XYWAV). One patient experienced suicidal ideation and two patients reported depression in a pediatric clinical trial with oxybate. These events occurred in patients with and without previous histories of depressive disorders. The emergence of depression in patients treated with XYWAV requires careful and immediate evaluation. Monitor patients for the emergence of increased depressive symptoms and/or suicidality while taking XYWAV. Other Behavioral or Psychiatric Adverse ReactionsIn Study 1, confusion and anxiety occurred in 1% and 5% of patients with narcolepsy treated with XYWAV, respectively. One patient experienced visual hallucinations and confusion after ingesting approximately 9 grams of XYWAV. In Study 2, confusion and anxiety occurred in 3% and 16% of patients with idiopathic hypersomnia, respectively. One patient experienced visual hallucinations, which led to discontinuation of XYWAV. Other neuropsychiatric reactions reported with oxybate (same active moiety as XYWAV) in adult or pediatric clinical trials and in the postmarketing setting include hallucinations, paranoia, psychosis, aggression, agitation, confusion and anxiety. The emergence or increase in the occurrence of behavioral or psychiatric events in patients taking XYWAV should be carefully monitored. ParasomniasParasomnias can occur in patients taking XYWAV. In Study 1 and Study 2, parasomnias, including sleepwalking, were reported in 6% and 5% of adult patients treated with XYWAV, respectively. In a clinical trial of XYREM (same active moiety as XYWAV) in adult patients with narcolepsy, five instances of sleepwalking with potential injury or significant injury were reported. Parasomnias, including sleepwalking, have been reported in a pediatric clinical trial with sodium oxybate (same active moiety as XYWAV) and in postmarketing experience with sodium oxybate. Episodes of sleepwalking should be fully evaluated and appropriate interventions considered. Most Common Adverse ReactionsThe most common adverse reactions (occurring in ≥5% of XYWAV-treated patients in adult clinical trials in either narcolepsy or IH) were nausea, headache, dizziness, anxiety, insomnia, decreased appetite, hyperhidrosis, vomiting, diarrhea, dry mouth, parasomnia, somnolence, fatigue, and tremor. In the pediatric clinical trial with XYREM (same active moiety as XYWAV) that included pediatric patients 7 to 17 years of age with narcolepsy, the most common adverse reactions (≥5%) were nausea (20%), enuresis (19%), vomiting (18%), headache (17%), weight decreased (13%), decreased appetite (9%), dizziness (8%), and sleepwalking (6%). The overall adverse reaction profile of XYREM in the pediatric clinical trial was similar to that seen in the adult clinical trial program. The safety profile in pediatric patients with XYWAV is expected to be similar to that of adult patients treated with XYWAV and to that of pediatric patients treated with XYREM. Additional Adverse ReactionsAdverse reactions that occurred in 2-<5% of adult patients treated with XYWAV in the Open Label Titration and Stable Dose Periods of the randomized-withdrawal study in adult patients with narcolepsy with cataplexy (Study 1) were fatigue, dry mouth, depressed mood, enuresis, irritability, paresthesia, depression, tremor, somnolence, and muscle spasms. Adverse reactions occurring in 2-<5% of patients treated with XYWAV in the IH study include balance disorder, muscle spasms, fall, paresthesia, snoring, weight decreased, bruxism, confusional state, depressed mood, feeling drunk, and irritability. Adverse reactions that occurred in ≥2% of patients in clinical studies with oxybate (but not in Study 1) and which may be relevant for XYWAV, were pain, feeling drunk, pain in extremity, cataplexy, disturbance in attention, sleep paralysis, and disorientation. Discontinuation: In Study 1, 9 of 201 patients (4%) reported adverse reactions that led to withdrawal from the study (anxiety, decreased appetite, depressed mood, depression, fatigue, headache, irritability, nausea, pain in extremity, parasomnia, somnolence, and vomiting). The most common adverse reaction leading to discontinuation was nausea (1.5%). In Study 2, 17 of 154 (11%) patients across all study periods (excluding placebo during the DB RWP) (up to 42 weeks) reported adverse reactions that led to withdrawal from the study (anxiety, nausea, insomnia, vomiting, fatigue, feeling abnormal, fall, decreased appetite, dizziness, paresthesia, tremor, parasomnia, confusional state, hallucination visual, and irritability). The most common adverse reaction leading to discontinuation was anxiety (3.2%). In Study 1 and Study 2, the majority of adverse reactions leading to discontinuation began during the first few weeks of treatment. In the pediatric clinical trial with XYREM (same active moiety as XYWAV), 7 of 104 patients reported adverse reactions that led to withdrawal from the study (hallucination, tactile; suicidal ideation; weight decreased; sleep apnea syndrome; affect lability; anger, anxiety, depression; and headache). Drug InteractionsXYWAV is contraindicated in combination with alcohol or sedative hypnotics. Use of other CNS depressants may potentiate the CNS-depressant effects of XYWAV. Concomitant use of sodium oxybate with divalproex sodium results in an increase in systemic exposure to GHB, which was shown to cause a greater impairment on some tests of attention and working memory in a clinical study. A similar increase in exposure is expected with concomitant use of XYWAV and divalproex sodium; therefore, an initial dose reduction of XYWAV is recommended when used concomitantly with divalproex sodium. Prescribers are advised to monitor patient response closely and adjust dose accordingly if concomitant use of XYWAV and divalproex sodium is warranted. Pregnancy and LactationThere are no adequate data on the developmental risk associated with the use of XYWAV or sodium oxybate in pregnant women. XYWAV should be used during pregnancy only if the potential benefit justifies the potential risk to the fetus. GHB is excreted in human milk after oral administration of sodium oxybate. There is insufficient information on the risk to a breastfed infant, and there is insufficient information on milk production in nursing mothers. The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for XYWAV and any potential adverse effects on the breastfed infant from XYWAV or from the underlying maternal condition. Pediatric UseThe safety and effectiveness of XYWAV for the treatment of cataplexy or excessive daytime sleepiness in pediatric patients 7 years of age and older with narcolepsy have been established. XYWAV has not been studied in a pediatric clinical trial for narcolepsy or IH. Use of XYWAV in pediatric patients 7 years of age and older with narcolepsy is supported by evidence from an adequate and well-controlled study of sodium oxybate in pediatric patients 7 to 17 years of age, a study in adults showing a treatment effect of XYWAV similar to that observed with sodium oxybate, pharmacokinetic data of sodium oxybate from adult and pediatric patients, and pharmacokinetic data of XYWAV from healthy adult volunteers. Safety and effectiveness of XYWAV in pediatric patients below the age of 7 years with narcolepsy have not been established. Safety and effectiveness of XYWAV for the treatment of idiopathic hypersomnia in pediatric patients have not been established. Geriatric UseIn general, dose selection for an elderly patient should be cautious, usually starting at the low end of the dosing range, reflecting the greater frequency of decreased hepatic, renal, or cardiac function, and of concomitant disease or other drug therapy. Hepatic ImpairmentThe starting dose of XYWAV should be reduced in patients with liver impairment. Dosage Modification in Patients with Hepatic Impairment: The recommended starting dosage in patients with hepatic impairment is one-half of the original dosage per night, administered orally, divided into two doses. Dependence and ToleranceThere have been case reports of withdrawal, ranging from mild to severe, following discontinuation of illicit use of GHB at frequent repeated doses (18 g to 250 g per day) in excess of the recommended dosage range. Signs and symptoms of GHB withdrawal following abrupt discontinuation included insomnia, restlessness, anxiety, psychosis, lethargy, nausea, tremor, sweating, muscle cramps, tachycardia, headache, dizziness, rebound fatigue and sleepiness, confusion, and, particularly in the case of severe withdrawal, visual hallucinations, agitation, and delirium. These symptoms generally abated in 3 to 14 days. In cases of severe withdrawal, hospitalization may be required. In the clinical trial experience with XYREM in narcolepsy/cataplexy patients at recommended doses, two patients reported anxiety and one reported insomnia following abrupt discontinuation at the termination of the clinical trial; in the two patients with anxiety, the frequency of cataplexy had increased markedly at the same time. In the XYWAV clinical trial in adult narcolepsy/cataplexy patients at recommended doses, one patient reported insomnia following abrupt discontinuation of XYWAV. In the XYWAV clinical trial in adult idiopathic hypersomnia patients at recommended doses, six patients reported insomnia, two patients reported early insomnia, and one patient reported visual and auditory hallucinations following abrupt discontinuation of XYWAV. Tolerance to XYWAV has not been systematically studied in controlled clinical trials. There have been some case reports of symptoms of tolerance developing after illicit use at dosages far in excess of the recommended XYWAV dosage regimen. Please see full Prescribing Information, including BOXED Warning here: About Jazz PharmaceuticalsJazz Pharmaceuticals plc (Nasdaq: JAZZ) is a global biopharma company whose purpose is to innovate to transform the lives of patients and their families. We are dedicated to developing potentially life-changing medicines for people with serious diseases — often with limited or no therapeutic options. We have a diverse portfolio of marketed medicines, including leading therapies for sleep disorders and epilepsy, and a growing portfolio of cancer treatments. Our patient-focused and science-driven approach powers pioneering research and development advancements across our robust pipeline of innovative therapeutics in oncology and neuroscience. Jazz is headquartered in Dublin, Ireland with research and development laboratories, manufacturing facilities and employees in multiple countries committed to serving patients worldwide. Please visit for more information. Contacts: Media:Kristin BhavnaniHead of Global Corporate CommunicationsJazz Pharmaceuticals plcCorporateAffairsMediaInfo@ Ireland +353 1 637 2141U.S. +1 215 867 4948 Investors:Jeff MacdonaldExecutive Director, Investor RelationsJazz Pharmaceuticals plcInvestorInfo@ +353 1 634 3211U.S. +1 650 496 2717 References: Sateia, M. J. (2023). International Classification of Sleep Disorders-Third Edition, Text Revision (ICSD-3-TR). Chest, 146(5), 1387–1394. PubMed. Dauvilliers Y, Arnulf I, Mignot E. Narcolepsy with cataplexy. Lancet. 2007;369(9560):499-511. Colten HR, Altevogt BM, Institute of Medicine (US) Committee on Sleep Medicine and Research, eds. Sleep Disorders and Sleep Deprivation: An Unmet Public Health Problem. Washington (DC): National Academies Press (US); 2006. Peacock J, Benca RM. Narcolepsy: clinical features, co-morbidities & treatment. Indian Journal of Medical Research. 2010;131(2):338-349. National Health Service. Narcolepsy – Overview. 2019. Accessed June 2025. Ben-Joseph RH, Saad R, Black J, et al. Cardiovascular burden of narcolepsy disease (CV-BOND): a real-world evidence study. Presented at: 2022 AAN Annual Meeting; April 2-7; Seattle, Washington. Poster 1203. Black J, Reaven NL, Funk SE, et al. Medical comorbidity in narcolepsy: findings from the Burden of Narcolepsy Disease (BOND) study. Sleep Med. 2017;33:13-18. Ohayon MM, Black J, Lai C, et al. Increased mortality in narcolepsy. Sleep. 2014;37(3):439-444. Ohayon MM. Narcolepsy is complicated by high medical and psychiatric comorbidities: a comparison with the general population. Sleep Med. 2013;14(6):488-492. Xywav (calcium, magnesium, potassium and sodium oxybates) oral solution. Prescribing Information. Palo Alto, CA: Jazz Pharmaceuticals, Inc. 2021. United States Drug Enforcement Agency. Drug Scheduling. Accessed June 2025. View original content to download multimedia: SOURCE Jazz Pharmaceuticals plc Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
02-06-2025
- Business
- Yahoo
Jazz Pharmaceuticals Announces Zepzelca® (lurbinectedin) and Atezolizumab (Tecentriq®) Combination Significantly Improves Survival as First-Line Maintenance Therapy for Extensive-Stage Small Cell Lung Cancer
First-line maintenance combination therapy reduced the risk of disease progression or death by 46%, with a median overall survival of 13.2 months vs 10.6 months for atezolizumab alone from the point of randomization First Phase 3 study to demonstrate statistically significant and clinically meaningful improvements in both progression-free and overall survival in ES-SCLC first-line maintenance Results presented at the ASCO 2025 Annual Meeting and simultaneously published in The Lancet Jazz to host investor webcast on Tuesday, June 10 to review Zepzelca data For U.S. media and investors only DUBLIN, June 2, 2025 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced positive results from the Phase 3 IMforte study of Zepzelca® (lurbinectedin) in combination with atezolizumab (Tecentriq®) as a first-line maintenance treatment for people with extensive-stage small cell lung cancer (ES-SCLC), following induction therapy with carboplatin, etoposide and atezolizumab. The study met both primary endpoints, demonstrating statistically significant improvements in progression-free survival (PFS) and overall survival (OS) compared to atezolizumab alone. IMforte is the first global Phase 3 trial to demonstrate clinically meaningful PFS and OS benefits in the first-line maintenance setting for ES-SCLC and supports maintenance therapy with Zepzelca plus atezolizumab as a new standard of care for patients. The data were presented today in an oral session at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago and published simultaneously in The Lancet. Data from the trial served as the basis for the supplemental New Drug Application (sNDA) submission to the U.S. Food and Drug Administration (FDA). Following induction therapy with carboplatin, etoposide and atezolizumab, patients who did not have disease progression were randomized to receive Zepzelca plus atezolizumab or atezolizumab alone. From the point of randomization, the median PFS was 5.4 months for the Zepzelca plus atezolizumab combination versus 2.1 months for atezolizumab alone (stratified HR = 0.54, 95% CI: 0.43–0.67; p < 0.0001), and median OS was 13.2 months versus 10.6 months (stratified hazard ratio [HR] = 0.73; 95% CI: 0.57–0.95; p = 0.0174). The combination reduced the risk of disease progression or death by 46% and the risk of death by 27% compared to atezolizumab alone. The Zepzelca plus atezolizumab combination had no new or unexpected safety signals. "Small cell lung cancer is an aggressive and devastating disease; at the time of diagnosis, the large majority of patients have already progressed to extensive-stage disease and only one out of five survive longer than two years,1" said Luis Paz-Ares, M.D., Ph.D., Head of Medical Oncology at the Hospital Universitario 12 de Octubre in Madrid, Spain, and IMforte trial principal investigator. "The IMforte results are very encouraging showing a potentially practice-changing option that could improve survival for patients with a very high unmet need." "In the U.S., approximately 30,000 new cases of small cell lung cancer are diagnosed each year, and the IMforte results demonstrate a combination treatment approach that can meaningfully extend the survival benefit for people with extensive-stage small cell lung cancer who complete induction therapy without progression,2,3" said Stephen V. Liu, M.D., Associate Professor of Medicine, Lombardi Comprehensive Cancer Center, Georgetown University, and IMforte trial investigator. "Unfortunately, a significant number of patients are not able to receive any therapy at the time of progression. This combination gives oncologists a new evidence-based option to help patients before progression occurs and improve outcomes in a setting where options have been limited." "The IMforte trial results underscore the potential of Zepzelca with atezolizumab to deliver clinically meaningful benefit as a first-line maintenance option for patients with extensive-stage small cell lung cancer and is a significant advance for these patients," 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. "These results represent important progress in expanding Zepzelca's potential utility earlier in the treatment journey. We look forward to engaging with the FDA to bring this indication to market as quickly as possible." Phase 3 IMforte Trial Results These primary results are from the global Phase 3 IMforte trial, which evaluated Zepzelca plus atezolizumab as a first-line maintenance therapy in patients with ES-SCLC. 483 patients were randomized after completion of 4 cycles of induction therapy with atezolizumab plus carboplatin and etoposide. From the point of randomization, the median OS for the Zepzelca plus atezolizumab regimen was 13.2 months versus 10.6 months for atezolizumab alone (stratified hazard ratio [HR] = 0.73; 95% CI: 0.57–0.95; p = 0.0174). From the point of randomization, the median PFS by independent assessment was 5.4 months versus 2.1 months, respectively (stratified HR = 0.54, 95% CI: 0.43–0.67; p < 0.0001). Treatment duration for patients in the Zepzelca plus atezolizumab arm was twice as long as the atezolizumab arm, with a median maintenance treatment duration of 4.2 months versus 2.1 months, respectively. The Zepzelca plus atezolizumab combination as maintenance therapy was generally well tolerated with no new safety signals identified. In the Zepzelca plus atezolizumab and atezolizumab arms, respectively, treatment-related adverse events (TRAEs) occurred in 83.5% versus 40.0% of patients, with Grade 3-4 TRAEs in 25.6% versus 5.8% and Grade 5 TRAEs in 0.8% (two patients with sepsis and febrile neutropenia) versus 0.4% (one patient with sepsis). AEs led to treatment discontinuation in 6.2% of patients in the Zepzelca plus atezolizumab arm and 3.3% of patients in the atezolizumab arm. The Company will host an investor webcast on June 10 at 4:30 p.m. ET / 9:30 p.m. IST to review Zepzelca data. The webcast will include commentary from a leading small cell lung cancer expert and Company senior management. The webcast may be accessed from the Investors section of the Jazz Pharmaceuticals website at About the IMforte Phase 3 TrialIMforte (NCT05091567) is an ongoing Phase 3, randomized, multicenter maintenance trial evaluating the efficacy, safety and pharmacokinetics of Zepzelca plus atezolizumab, compared with standard-of-care first-line maintenance with atezolizumab alone, in adults (≥18 years) with ES-SCLC, following induction therapy with carboplatin, etoposide and atezolizumab. The primary endpoints for this study are OS and independent review facility (IRF)-assessed PFS in the maintenance phase. The trial consists of two phases: an induction phase and a maintenance phase. Participants were required to have an ongoing response or stable disease per the Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 after the induction phase of four cycles of carboplatin, etoposide, and atezolizumab to be considered for eligibility screening for the maintenance phase. Eligible participants were randomized in a 1:1 ratio to receive either lurbinectedin plus atezolizumab or atezolizumab in the maintenance phase. The trial is sponsored by Roche and co-funded by Jazz Pharmaceuticals. Additional information about the trial, including eligibility criteria and a list of clinical trial sites, can be found at: (Identifier: NCT05091567). About Small Cell Lung CancerIn the U.S., approximately 13 percent of lung cancers are small cell.2 Approximately 30,000 new cases of small cell lung cancer (SCLC) are reported in the U.S. each year.2,3 The risk for developing SCLC is much higher among current or former tobacco smokers; however, SCLC can also be caused by exposure to secondhand smoke, asbestos, some inhaled chemicals, radiation and air pollution. People with a family history of lung cancer may also be at a higher risk, too.4 SCLC is the most aggressive form of lung cancer and it tends to spread quickly to other parts of the body including the brain, liver and bone.5,6 A large percentage of SCLC patients on treatment briefly achieve a response, although the cancer often returns and is usually more aggressive and resistant to regimens that were previously effective.5 About Zepzelca® (lurbinectedin)Zepzelca is an alkylating drug that binds guanine residues within DNA. This triggers a cascade of events that can affect the activity of DNA binding proteins, including some transcription factors, and DNA repair pathways, resulting in disruption of the cell cycle and potentially cell death.4 The FDA approved Zepzelca under accelerated approval in June 2020 for the treatment of adult patients with metastatic small cell lung cancer (SCLC) with disease progression on or after platinum-based chemotherapy. The approval is based on overall response rate (ORR) and duration of response demonstrated in an open-label, monotherapy clinical study. In December 2021, Jazz and PharmaMar announced the initiation of LAGOON, a confirmatory Phase 3 clinical trial of Zepzelca for the treatment of patients with relapsed small cell lung cancer. If positive, LAGOON could confirm the benefit of Zepzelca in the treatment of SCLC when patients progress following 1L treatment with a platinum-based regimen and support full approval in the U.S. Zepzelca is a prescription medicine used to treat adults with SCLC that has spread to other parts of the body (metastatic) and who have received treatment with chemotherapy that contains platinum, and it did not work or is no longer working. Zepzelca is approved based on response rate and how long the response lasted. Additional studies will further evaluate the benefit of Zepzelca for this use. Important Safety Information Myelosuppression ZEPZELCA can cause myelosuppression. In clinical studies of 554 patients with advanced solid tumors receiving ZEPZELCA, Grade 3 or 4 neutropenia occurred in 41% of patients, with a median time to onset of 15 days and a median duration of 7 days. Febrile neutropenia occurred in 7% of patients. Sepsis occurred in 2% of patients and was fatal in 1% (all cases occurred in patients with solid tumors other than SCLC). Grade 3 or 4 thrombocytopenia occurred in 10%, with a median time to onset of 10 days and a median duration of 7 days. Grade 3 or 4 anemia occurred in 17% of patients. Administer ZEPZELCA only to patients with baseline neutrophil count of at least 1,500 cells/mm3 and platelet count of at least 100,000/mm3. Monitor blood counts including neutrophil count and platelet count prior to each administration. For neutrophil count less than 500 cells/mm3 or any value less than lower limit of normal, the use of G-CSF is recommended. Withhold, reduce the dose, or permanently discontinue ZEPZELCA based on severity. Hepatotoxicity ZEPZELCA can cause hepatotoxicity. In clinical studies of 554 patients with advanced solid tumors receiving ZEPZELCA, Grade 3 elevations of ALT and AST were observed in 6% and 3% of patients, respectively, and Grade 4 elevations of ALT and AST were observed in 0.4% and 0.5% of patients, respectively. The median time to onset of Grade ≥3 elevation in transaminases was 8 days (range: 3 to 49), with a median duration of 7 days. Monitor liver function tests prior to initiating ZEPZELCA, periodically during treatment, and as clinically indicated. Withhold, reduce the dose, or permanently discontinue ZEPZELCA based on severity. Extravasation Resulting in Tissue Necrosis Extravasation of ZEPZELCA resulting in skin and soft tissue injury, including necrosis requiring debridement, can occur. Consider use of a central venous catheter to reduce the risk of extravasation, particularly in patients with limited venous access. Monitor patients for signs and symptoms of extravasation during the ZEPZELCA infusion. If extravasation occurs, immediately discontinue the infusion, remove the infusion catheter, and monitor for signs and symptoms of tissue necrosis. The time to onset of necrosis after extravasation may vary. Administer supportive care and consult with an appropriate medical specialist as needed for signs and symptoms of extravasation. Administer subsequent infusions at a site that was not affected by extravasation. Rhabdomyolysis Rhabdomyolysis has been reported in patients treated with ZEPZELCA. Monitor creatine phosphokinase (CPK) prior to initiating ZEPZELCA and periodically during treatment as clinically indicated. Withhold or reduce the dose based on severity. Embryo-Fetal Toxicity ZEPZELCA can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise female patients of reproductive potential to use effective contraception during treatment with ZEPZELCA and for 6 months after the last dose. Advise male patients with female partners of reproductive potential to use effective contraception during treatment with ZEPZELCA and for 4 months after the last dose. Lactation There are no data on the presence of ZEPZELCA in human milk, however, because of the potential for serious adverse reactions from ZEPZELCA in breastfed children, advise women not to breastfeed during treatment with ZEPZELCA and for 2 weeks after the last dose. MOST COMMON ADVERSE REACTIONS The most common adverse reactions, including laboratory abnormalities, (≥20%) are leukopenia (79%), lymphopenia (79%), fatigue (77%), anemia (74%), neutropenia (71%), increased creatinine (69%), increased alanine aminotransferase (66%), increased glucose (52%), thrombocytopenia (37%), nausea (37%), decreased appetite (33%), musculoskeletal pain (33%), decreased albumin (32%), constipation (31%), dyspnea (31%), decreased sodium (31%), increased aspartate aminotransferase (26%), vomiting (22%), decreased magnesium (22%), cough (20%), and diarrhea (20%). DRUG INTERACTIONS Effect of CYP3A Inhibitors and InducersAvoid coadministration with a strong or a moderate CYP3A inhibitor (including grapefruit and Seville oranges) as this increases lurbinectedin systemic exposure which may increase the incidence and severity of adverse reactions to ZEPZELCA. If coadministration cannot be avoided, reduce the ZEPZELCA dose as appropriate. Avoid coadministration with a strong CYP3A inducer as it may decrease systemic exposure to lurbinectedin, which may decrease the efficacy of ZEPZELCA. GERIATRIC USEOf the 105 patients with SCLC administered ZEPZELCA in clinical studies, 37 (35%) patients were 65 years of age and older, while 9 (9%) patients were 75 years of age and older. No overall difference in effectiveness was observed between patients aged 65 and older and younger patients. There was a higher incidence of serious adverse reactions in patients ≥65 years of age than in patients <65 years of age (49% vs 26%, respectively). The serious adverse reactions most frequently reported in patients ≥65 years of age were related to myelosuppression and consisted of febrile neutropenia (11%), neutropenia (11%), thrombocytopenia (8%), and anemia (8%). Please see accompanying full Prescribing Information. ZEPZELCA is a trademark of Pharma Mar, S.A. used by Jazz Pharmaceuticals under license. Tecentriq (atezolizumab) is a registered trademark of Genentech, a member of the Roche Group. About Jazz PharmaceuticalsJazz Pharmaceuticals plc (Nasdaq: JAZZ) is a global biopharma company whose purpose is to innovate to transform the lives of patients and their families. We are dedicated to developing potentially life-changing medicines for people with serious diseases — often with limited or no therapeutic options. We have a diverse portfolio of marketed medicines, including leading therapies for sleep disorders and epilepsy, and a growing portfolio of cancer treatments. Our patient-focused and science-driven approach powers pioneering research and development advancements across our robust pipeline of innovative therapeutics in oncology and neuroscience. Jazz is headquartered in Dublin, Ireland with research and development laboratories, manufacturing facilities and employees in multiple countries committed to serving patients worldwide. Please visit for more information. Jazz Pharmaceuticals plc Caution Concerning Forward-Looking StatementsThis press release contains forward-looking statements, including, but not limited to, statements related to Zepzelca's potential as a first-line maintenance therapy for extensive-stage small cell lung cancer, the potential for Zepzelca in combination with atezolizumab to become a new standard of care for patients with ES-SCLC and other statements that are not historical facts. These forward-looking statements are based on Jazz Pharmaceuticals' current plans, objectives, estimates, expectations and intentions and inherently involve significant risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation, risks and uncertainties associated with the successful completion of regulatory activities and uncertain regulatory approval, and other risks and uncertainties affecting Jazz Pharmaceuticals and its development programs, including those described from time to time under the caption "Risk Factors" and elsewhere in Jazz Pharmaceuticals plc's Securities and Exchange Commission filings and reports (Commission File No. 001-33500), including Jazz Pharmaceuticals' Annual Report on Form 10-K for the year ended December 31, 2024, as supplement by Jazz Pharmaceuticals' Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, and future filings and reports by Jazz Pharmaceuticals. Other risks and uncertainties of which Jazz Pharmaceuticals is not currently aware may also affect Jazz Pharmaceuticals' forward-looking statements and may cause actual results and the timing of events to differ materially from those anticipated. The forward-looking statements herein are made only as of the date hereof or as of the dates indicated in the forward-looking statements, even if they are subsequently made available by Jazz Pharmaceuticals on its website or otherwise. Jazz Pharmaceuticals undertakes no obligation to update or supplement any forward-looking statements to reflect actual results, new information, future events, changes in its expectations or other circumstances that exist after the date as of which the forward-looking statements were made. Contacts: Media Contact:Kristin BhavnaniHead of Global Corporate CommunicationsJazz Pharmaceuticals plcCorporateAffairsMediaInfo@ +353 1 637 2141U.S. +1 215 867 4948 Investors: Jeff Macdonald Executive Director, Investor RelationsJazz Pharmaceuticals plcinvestorinfo@ Ireland +353 1 634 3211 U.S. +1 650 496 2717 1 Murray, Nevin, and Andrew T. Turrisi III. A review of first-line treatment for small-cell lung cancer. Journal of Thoracic Oncology 1.3 (2006): 270-278.2 Alvarado-Lunda G, Morales-Espinosa D. Treatment for small cell lung cancer, where are we now? – A review. Transl Lung Cancer Res. 2016;5(1):26-38.3 SEER Explorer Lung and Bronchus Cancer, Recent Trends in SEER Incidence Rates, 2000-2016, by Age, Updated June 27, 2024. Accessed May 30, 2025.4 American Cancer Society. Small cell lung cancer causes, risk factors, and prevention. Updated May 16, 2016. Accessed May 30, 2025.5 American Cancer Society. What is lung cancer? Updated October 1, 2019. Accessed May 30, 2025.6 American Cancer Society. Small cell lung cancer stages. Updated October 1, 2019. Accessed May 30, 2025. View original content to download multimedia: SOURCE Jazz Pharmaceuticals plc Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
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02-06-2025
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Jazz Pharmaceuticals Reports Clinically Meaningful Long-Term Median Overall Survival Data for Ziihera® (zanidatamab-hrii) in First-Line HER2-Positive Metastatic Gastroesophageal Adenocarcinoma at ASCO 2025
Phase 2 trial results continue to show clinically meaningful efficacy and durable responses, including 36.5-month median overall survival after four years of follow-up, with a manageable safety profile Findings presented today at ASCO 2025 and concurrently published in The Lancet Oncology For U.S. media and investors only DUBLIN, June 2, 2025 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced long-term data, including the first report of median overall survival (OS) from the Phase 2 trial evaluating Ziihera® (zanidatamab-hrii), a dual HER2-targeted bispecific antibody, in combination with chemotherapy for the investigational use in first-line HER2-positive (IHC 3+ or IHC 2+/FISH+) locally advanced nonresectable gastroesophageal adenocarcinoma (mGEA). The data were featured as a rapid oral presentation at the American Society of Clinical Oncology (ASCO) Annual Meeting, and the results were concurrently published in The Lancet Oncology. Among 41 patients with centrally confirmed HER2-positive tumors, treatment with Ziihera in combination with physician's choice of chemotherapy resulted in a median progression-free survival (PFS) of 15.2 months [95% CI: 9.5, 33.4], and a median overall survival (OS) of 36.5 months [95% CI: 23.6, not estimable (NE)]. Median PFS remained stable with the additional four-year follow-up, consistent with previously reported results. Among all 46 patients in the study with HER2-expressing mGEA, median PFS was 12.5 months [95% CI: 8.2, 21.8], and median OS also reached 36.5 months [95% CI: 23.6, NE], with the longest observed survival at 57.9 months (censored at data cutoff). Long-term follow-up also demonstrated low discontinuation rates, with no new safety signals observed. "Gastroesophageal adenocarcinoma remains a highly aggressive cancer with a poor prognosis, even with currently available treatment options," said Dr. Elena Elimova, lead trial investigator and a medical oncologist at Princess Margaret Cancer Centre, Toronto, Canada. "The long-term survival outcomes presented today at ASCO demonstrate the sustained antitumor activity achieved with zanidatamab plus chemotherapy over four years of follow-up. These results are especially encouraging given the high unmet need for better first-line treatment options for this patient population." "These long-term survival data from our Phase 2 trial build on previously reported results and further strengthen our belief in Ziihera as a transformative treatment option for patients with HER2-positive disease," 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. "An estimated median overall survival of 36.5 months in this patient cohort is very encouraging given recent observations with standard of care regimens in similar populations, where median survival has typically ranged from 15 to 20 months. The sustained 15.2-month progression-free survival in the centrally confirmed HER2-positive subgroup after four years is a meaningful indicator of durable clinical benefit. We look forward to the top line results of the pivotal Phase 3 HERIZON-GEA-01 trial later this year and remain committed to advancing Ziihera across multiple tumor types." Phase 2 mGEA Trial Results The data include four-year follow-up and the first report of median OS from an ongoing, open-label Phase 2 trial (NCT03929666) evaluating Ziihera in combination with chemotherapy as a first-line treatment for patients with HER2-expressing mGEA, which includes gastric, esophageal and gastroesophageal junction (GEJ) adenocarcinomas. Patients had not received prior HER2-targeted agents nor systemic treatment for mGEA. A total of 46 patients with HER2-expressing mGEA (41 patients with centrally confirmed HER2-positive mGEA) were enrolled from 14 sites across the United States, Canada and South Korea. Patients received Ziihera with physician's choice of chemotherapy, including fluoropyrimidine maintenance regimens. Chemotherapy-based regimens remain the current standard first-line treatment for mGEA. The longer-term data (median duration of follow-up of 48 months [range, 29-59]) demonstrate the promising antitumor activity of Ziihera combined with chemotherapy as a first-line treatment for HER2-positive mGEA. In a post-hoc subgroup analysis of the 41 treated patients with centrally confirmed HER2-positive tumors, median PFS was 15.2 months [95% CI: 9.5, 33.4], and median OS was 36.5 months [95% CI: 23.6, NE]. These survival outcomes were consistent with prior analyses, with PFS durability maintained at the four-year follow-up. The confirmed objective response rate (cORR), the study's primary endpoint, was 83.8% [95% CI: 68.0, 93.8], and median duration of response (DOR) was 20.4 months [95% CI: 8.3, 44.1]. These results further support the observed clinical benefit in this centrally confirmed population. Among all 46 patients in the study, median PFS was 12.5 months [95% CI: 8.2, 21.8], and the estimated 24-month PFS rate was 31% [95% CI: 17%, 46%]. Median OS was also 36.5 months [95% CI: 23.6, NE], with an estimated 24-month OS rate of 65% [95% CI: 49%, 77%]. The cORR was 76.2% [95% CI: 60.5, 87.9], and median DOR was 18.7 months [95% CI: 10.4, 44.1]. With additional follow-up, the safety and tolerability profile of Ziihera plus chemotherapy showed low discontinuation rates, with no new safety signals identified. Diarrhea (39%) and hypokalemia (22%) were the most common Grade 3-4 treatment-related adverse events (TRAEs); the incidence of Grade 3 diarrhea was reduced from 52% to 24% for patients enrolled after the implementation of mandated antidiarrheal prophylaxis. There were no treatment-related deaths. Five patients discontinued Ziihera due to TRAEs. Ongoing Phase 3 TrialThe Phase 3 randomized clinical trial, HERIZON-GEA-01 (NCT05152147), evaluating Ziihera in combination with standard of care chemotherapy with and without the addition of a PD-1 agent as a first-line treatment for HER2-expressing mGEA is currently underway. This is an events-based trial, and top-line results are expected to read out in the second half of 2025. About Gastroesophageal AdenocarcinomaGastroesophageal adenocarcinoma (GEA) is the fifth most common cancer worldwide, and approximately 20% of patients have HER2-positive disease.i,ii,iii HER2-positive 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 percent for gastric cancer and about 19 percent for About Ziihera® (zanidatamab-hrii) Ziihera (zanidatamab-hrii) is a bispecific HER2-directed antibody that binds to two extracellular sites on HER2. Binding of zanidatamab-hrii with HER2 results in internalization leading to a reduction in HER2 expression of the receptor on the tumor cell surface. Zanidatamab-hrii 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.v 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.v The U.S. Food and Drug Administration (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). v 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 BeiGene, Ltd. (BeiGene) under license agreements from Zymeworks, which first developed the molecule. The FDA granted Breakthrough Therapy designation for zanidatamab development in patients with previously treated HER2 gene-amplified BTC, and two Fast Track designations for zanidatamab: one as a single agent for refractory BTC and one in combination with standard-of-care chemotherapy for 1L gastroesophageal adenocarcinoma (GEA). Additionally, zanidatamab has received Orphan Drug designations from FDA for the treatment of BTC and GEA, as well as Orphan Drug designation from the European Medicines Agency for the treatment of BTC and gastric cancer. Important Safety Information for ZIIHERA WARNING: EMBRYO-FETAL TOXICITYExposure to ZIIHERA during pregnancy can cause embryo-fetal harm. Advise patientsof 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 females 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: About Jazz Pharmaceuticals Jazz Pharmaceuticals plc (Nasdaq: JAZZ) is a global biopharma company whose purpose is to innovate to transform the lives of patients and their families. We are dedicated to developing potentially life-changing medicines for people with serious diseases — often with limited or no therapeutic options. We have a diverse portfolio of marketed medicines, including leading therapies for sleep disorders and epilepsy, and a growing portfolio of cancer treatments. Our patient-focused and science-driven approach powers pioneering research and development advancements across our robust pipeline of innovative therapeutics in oncology and neuroscience. Jazz is headquartered in Dublin, Ireland with research and development laboratories, manufacturing facilities and employees in multiple countries committed to serving patients worldwide. Please visit for more information. Jazz Pharmaceuticals plc Caution Concerning Forward-Looking StatementsThis press release contains forward-looking statements, including, but not limited to, statements related to zanidatamab's potential as a transformative treatment option for patients with HER2-positive disease, expected timing of top-line results of the pivotal Phase 3 HERIZON-GEA-01 and other statements that are not historical facts. These forward-looking statements are based on Jazz Pharmaceuticals' current plans, objectives, estimates, expectations and intentions and inherently involve significant risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation, risks and uncertainties associated with the successful completion of regulatory activities and uncertain regulatory approval, risks related to failure or delays in successfully initiating or completing clinical trials and assessing patients and other risks and uncertainties affecting Jazz Pharmaceuticals and its development programs, including those described from time to time under the caption "Risk Factors" and elsewhere in Jazz Pharmaceuticals plc's Securities and Exchange Commission filings and reports (Commission File No. 001-33500), including Jazz Pharmaceuticals' Annual Report on Form 10-K for the year ended December 31, 2024, as supplement by Jazz Pharmaceuticals' Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, and future filings and reports by Jazz Pharmaceuticals. Other risks and uncertainties of which Jazz Pharmaceuticals is not currently aware may also affect Jazz Pharmaceuticals' forward-looking statements and may cause actual results and the timing of events to differ materially from those anticipated. The forward-looking statements herein are made only as of the date hereof or as of the dates indicated in the forward-looking statements, even if they are subsequently made available by Jazz Pharmaceuticals on its website or otherwise. Jazz Pharmaceuticals undertakes no obligation to update or supplement any forward-looking statements to reflect actual results, new information, future events, changes in its expectations or other circumstances that exist after the date as of which the forward-looking statements were made. Contacts: Media Contact:Kristin BhavnaniHead of Global Corporate CommunicationsJazz Pharmaceuticals plcCorporateAffairsMediaInfo@ +353 1 637 2141U.S. +1 215 867 4948 Investors: Jeff Macdonald Executive Director, Investor RelationsJazz Pharmaceuticals plcinvestorinfo@ Ireland +353 1 634 3211 U.S. +1 650 496 2717 i Abrahao-Machado I.F., et al. HER2 testing in gastric cancer: An update WorldJGastroenterol. 2016;22(19):4619-4625. ii Van Custem E., et al. HER2 screening data from ToGA: targeting HER2 in gastric and gastroesophageal junction cancer. Gastric Cancer. 2015;18(3):476-484. iii Stroes, C.I., et al. A systematic review of HER2 blockade for the curative treatment of gastroesophageal adenocarcinoma: Successes achieved and opportunities ahead. CancerTreatRev. 2021;99:102249. iv Battaglin F, et al. Molecular biomarkers in gastro-esophageal cancer: recent developments, current trends and future directions. Cancer Cell International. 2018;18(99). v ZIIHERA (zanidatamab-hrii) Prescribing Information. Palo Alto, CA: Jazz Pharmaceuticals, Inc.). View original content to download multimedia: SOURCE Jazz Pharmaceuticals plc
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07-05-2025
- Business
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Jazz Pharmaceuticals (JAZZ): Among Billionaire Ray Dalio's Bridgewater's Stock Picks with Huge Upside Potential
We recently published a list of Billionaire Ray Dalio's Bridgewater's 10 Stock Picks with Huge Upside Potential. In this article, we are going to take a look at where Jazz Pharmaceuticals plc (NASDAQ:JAZZ) stands against Billionaire Ray Dalio's Bridgewater's other stock picks with huge upside potential. Up until February 18, 2025, the stock market seemed poised to replicate the 2020-2022 bull run. Save for slight corrections in April and August of 2024, the market was in a general upside trend. And investors expected the trend to intensify, thanks to the Trump effect. But before President Trump's re-election, some voices, including that of Ray Dalio, pointed out the potential risks of this presidency to the market. Ray Dalio established Bridgewater Associates in 1975. But as of 2025, the billionaire investor isn't part of the hedge fund's key management. He currently acts as co-chief investment officer (CIO) mentor and member of the Bridgewater Board. Dalio also runs his family office, the Dalio Family Office, which manages over $15 billion in assets. He also does philanthropy work and he hasn't stepped away from sharing his economic and investment insights through books, interviews, and social media. Bridgewater has grown to become the largest and most renowned investment firm in the world. Over the past 10 years, the fund has returned 39.40%, and 17.30% over the past 12 months. However, just like Dalio predicted, the Trump administration's policies could jeopardize Bridgewater's ability to reward investors. Since taking office for the second term, Trump has made tariffs a key part of his government's economic policy. He issued an executive order on his first day in office and threatened 25% tariffs on all goods from China, Canada, and Mexico. The tariffs escalated through Liberation Day in April. All this time, the S&P 500 was taking one hit after another. For instance, the broad market index pulled back 12% between April 2 and April 8, 2025. April 2 happens to be the day of Trump's Liberation Day speech that marked a major tariff escalation. READ ALSO: Billionaire Jim Simons' RenTech's 10 Small-Cap Stock Picks with Huge Upside Potential and Billionaire Chase Coleman's 10 Stocks with Huge Upside Potential. Although the equity market appears to be correcting as of May 1, 2025 (the S&P 500 is up 12% since April 8), Dalio believes that Trump's tariffs have wrought irreversible damage. According to a post on X on Monday, Dalio said that many of his indicators tell him that 'we are on the brink of the monetary order, the domestic political and the international world orders breaking down due to unsustainable, bad fundamentals.'
