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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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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
Yahoo
07-05-2025
- Business
- Yahoo
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.'
Yahoo
29-03-2025
- Business
- Yahoo
Is Jazz Pharmaceuticals (JAZZ) the Best Mid Cap Biotech Stock to Buy?
We recently published a list of the . In this article, we are going to take a look at where Jazz Pharmaceuticals plc (NASDAQ:JAZZ) stands against the other best mid cap biotech stocks to buy. On March 6, Jared Holz, Mizuho health care equity strategist, appeared on CNBC's 'Power Lunch' to discuss investing in biotech and the oversaturation in the sector. He said that looking at the denominator of biotech stocks in the public market, we're standing at the 700-800 range, depending upon the day. Almost a quarter of these have enterprise values in negative territory. That basically means that investors and companies, in some respects, have exhausted options. For any value to be created, it is necessary to shift focus and devise another plan of attack through an asset they don't currently own, and resuscitate the company. In some respects, investors and analysts are always looking for a clear pathway for biotech as a sector to perform better. However, Holz said we are in a tough place if a quarter of the index is filled with companies with more cash than the market cap. That is why he suggested, which some people consider somewhat hyperbolic, getting rid of the tail here, as there are too many assets out there. Thousands of biotech companies are competing against each other for tiny market or revenue opportunities. Holz opined that venture funds should stop making so many companies that end up superseding each other in a few years. It would be helpful to dissolve all the biotech stocks with negative values and return the cash to shareholders. READ ALSO: and . Biotechs pose a tempting scenario, as a company's stock could be worth $5 with a potential blockbuster drug in the pipeline that could cure even cancer. The company's stock could surge to hundreds of dollars if the clinical trial succeeds. However, at the same time, it could plunge down to $0 if the trial doesn't. Holz thus says that the sector is incredibly tempting, and thousands of investors put their careers on the line daily to attempt to figure this out. He characterized biotech as one of the only sectors in all of equities where single stock picking is the only way to make money. Building on his years-long experience covering the industry, he said that the index has barely been up since the past decade. It has been essentially flat. Meanwhile, every other index that one can find has doubled or more. Therefore, there is an obvious issue with the broader complex. But if you find the right asset, you can kill it in the sector. We sifted through stock screeners, financial media reports, and ETFs to compile a list of 30 biotech stocks with a market cap between $2 billion and $10 billion. We then selected the top 10 with the highest number of hedge fund holders, as of Q4 2024, and ranked them in ascending order. We sourced the hedge fund sentiment data from Insider Monkey's database. Why are we interested in the stocks that hedge funds pile into? The reason is simple: our research has shown that we can outperform the market by imitating the top stock picks of the best hedge funds. Our quarterly newsletter's strategy selects 14 small-cap and large-cap stocks every quarter and has returned 373.4% since May 2014, beating its benchmark by 218 percentage points (). A biopharmaceutical scientist in their lab, studying a newly-diagnosed therapy-related acute myeloid leukemia. . Market Cap: $8.21 billion Number of Hedge Fund Holders: 49 Jazz Pharmaceuticals plc (NASDAQ:JAZZ) develops medicines for serious diseases. Its primary marketed products include Xywav, Xyrem oral solution, Epidiolex oral solution, Rylaze, Zepzelca, Defitelio, and Vyxeos liposome for injection. These medicines treat excess daytime sleepiness (EDS) in narcolepsy patients seven years of age or older, hepatic veno-occlusive disease (VOD), and other ailments. It ranks eighth among the 10 best mid cap biotech stocks to buy now. 2024 was a record year for the company, with its strong commercial execution leading to over $4 billion in total annual revenue and fiscal Q4 2024 revenue of around $1.1 billion, the highest ever for Jazz Pharmaceuticals plc (NASDAQ:JAZZ). The company attained several significant development and regulatory milestones in 2024 on the R&D front. For instance, in November, it attained the accelerated approval of Ziihera in the US to treat second-line HER2-positive biliary tract cancer. Strategic corporate development is a key focus for Jazz Pharmaceuticals plc (NASDAQ:JAZZ), which supports its plans to diversify its commercial and pipeline portfolio. It is evaluating oncology and neuroscience assets as part of its ongoing search and is also looking into other rare and orphan diseases. In a report released on March 14, Akash Tewari from Jefferies maintained a Buy rating on Jazz Pharmaceuticals plc (NASDAQ:JAZZ), with a price target of $183.00. Overall, JAZZ ranks 8th on our list of the best mid cap biotech stocks to buy. While we acknowledge the potential of JAZZ as an investment, our conviction lies in the belief that AI stocks hold greater promise for delivering higher returns and doing so within a shorter time frame. If you are looking for an AI stock that is more promising than JAZZ but that trades at less than 5 times its earnings, check out our report about the . READ NEXT: and . Disclosure: None. This article is originally published at .
Yahoo
05-03-2025
- Business
- Yahoo
Statutory Profit Doesn't Reflect How Good Jazz Pharmaceuticals' (NASDAQ:JAZZ) Earnings Are
Jazz Pharmaceuticals plc's (NASDAQ:JAZZ) strong earnings report was rewarded with a positive stock price move. Our analysis found some more factors that we think are good for shareholders. View our latest analysis for Jazz Pharmaceuticals One key financial ratio used to measure how well a company converts its profit to free cash flow (FCF) is the accrual ratio. The accrual ratio subtracts the FCF from the profit for a given period, and divides the result by the average operating assets of the company over that time. The ratio shows us how much a company's profit exceeds its FCF. Therefore, it's actually considered a good thing when a company has a negative accrual ratio, but a bad thing if its accrual ratio is positive. That is not intended to imply we should worry about a positive accrual ratio, but it's worth noting where the accrual ratio is rather high. Notably, there is some academic evidence that suggests that a high accrual ratio is a bad sign for near-term profits, generally speaking. For the year to December 2024, Jazz Pharmaceuticals had an accrual ratio of -0.10. That implies it has good cash conversion, and implies that its free cash flow solidly exceeded its profit last year. To wit, it produced free cash flow of US$1.3b during the period, dwarfing its reported profit of US$560.1m. Jazz Pharmaceuticals shareholders are no doubt pleased that free cash flow improved over the last twelve months. However, we can see that a recent tax benefit, along with unusual items, have impacted its statutory profit, and therefore its accrual ratio. That might leave you wondering what analysts are forecasting in terms of future profitability. Luckily, you can click here to see an interactive graph depicting future profitability, based on their estimates. Jazz Pharmaceuticals' profit was reduced by unusual items worth US$145m in the last twelve months, and this helped it produce high cash conversion, as reflected by its unusual items. In a scenario where those unusual items included non-cash charges, we'd expect to see a strong accrual ratio, which is exactly what has happened in this case. It's never great to see unusual items costing the company profits, but on the upside, things might improve sooner rather than later. We looked at thousands of listed companies and found that unusual items are very often one-off in nature. And that's hardly a surprise given these line items are considered unusual. Assuming those unusual expenses don't come up again, we'd therefore expect Jazz Pharmaceuticals to produce a higher profit next year, all else being equal. In addition to the notable accrual ratio, we can see that Jazz Pharmaceuticals received a tax benefit of US$91m. It's always a bit noteworthy when a company is paid by the tax man, rather than paying the tax man. The receipt of a tax benefit is obviously a good thing, on its own. However, our data indicates that tax benefits can temporarily boost statutory profit in the year it is booked, but subsequently profit may fall back. In the likely event the tax benefit is not repeated, we'd expect to see its statutory profit levels drop, at least in the absence of strong growth. In conclusion, both Jazz Pharmaceuticals' accrual ratio and its unusual items suggest that its statutory earnings are probably reasonably conservative, but the presence of a tax benefits may be inflating the numbers in a way that won't persist. Looking at all these factors, we'd say that Jazz Pharmaceuticals' underlying earnings power is at least as good as the statutory numbers would make it seem. In light of this, if you'd like to do more analysis on the company, it's vital to be informed of the risks involved. While conducting our analysis, we found that Jazz Pharmaceuticals has 2 warning signs and it would be unwise to ignore these. After our examination into the nature of Jazz Pharmaceuticals' profit, we've come away optimistic for the company. But there is always more to discover if you are capable of focussing your mind on minutiae. Some people consider a high return on equity to be a good sign of a quality business. While it might take a little research on your behalf, you may find this free collection of companies boasting high return on equity, or this list of stocks with significant insider holdings to be useful. Have feedback on this article? Concerned about the content? Get in touch with us directly. Alternatively, email editorial-team (at) article by Simply Wall St is general in nature. We provide commentary based on historical data and analyst forecasts only using an unbiased methodology and our articles are not intended to be financial advice. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. We aim to bring you long-term focused analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Simply Wall St has no position in any stocks mentioned. Sign in to access your portfolio
