Positive Phase 2b Trial Data: Zanidatamab in HER2-Amplified Biliary Tract Cancers Presented at ASCO 2023 by Jazz Pharmaceuticals and Zymeworks

prnewswire | June 05, 2023 | Read time : 01:53 min


Jazz Pharmaceuticals plc and Zymeworks Inc. (Nasdaq: ZYME) today presented positive pivotal trial data, including new data on progression-free survival (PFS), from the Phase 2b HERIZON-BTC-01 trial of the bispecific antibody zanidatamab in previously treated HER2-amplified biliary tract cancers (BTC). The data were featured as an oral presentation at the American Society of Clinical Oncology (ASCO) Annual Meeting, and the results were concurrently published in The Lancet Oncology. The abstract (4008) was also selected to be included in the 2023 Best of ASCO® program, which will be held this summer following the ASCO Annual Meeting.

For the trial's primary endpoint, data from 80 patients with HER2-amplified BTC (defined as in situ hybridization [ISH] positive and immunohistochemistry [IHC] 2+ or 3+) demonstrated a confirmed objective response rate (cORR) of 41.3% [95% confidence interval (CI): 30.4, 52.8] with a Kaplan Meier (KM) estimated median duration of response (DOR) of 12.9 months. The KM estimated median PFS was 5.5 months [95% CI: 3.7, 7.2] with a range of 0.3 to 18.5 months.

With a confirmed ORR of 41.3 percent, median DOR of 12.9 months and median PFS of 5.5 months, these results for zanidatamab are a significant step forward for second-line treatment of HER2-amplified BTC, where current chemotherapy treatments have been reported to provide only a 5 to 15 percent ORR and median PFS of 1.4 to 4 months," said Shubham Pant, M.D., professor of Gastrointestinal Medical Oncology and Investigational Cancer Therapeutics at The University of Texas MD Anderson Cancer Center. "The HERIZON-BTC-01 trial advances an exciting field of oncology research where we can leverage next-generation sequencing on BTC patients to understand genomic markers of the disease and choose the appropriate targeted therapies for these patients."

"We are thrilled to deliver an oral presentation on the pivotal HERIZON-BTC-01 study results demonstrating zanidatamab's meaningful clinical benefit and tolerable safety profile in patients with HER2-amplified BTC," said Rob Iannone, M.D., M.S.C.E., executive vice president, global head of research and development of Jazz Pharmaceuticals. "We are committed to advancing this program as rapidly as possible to potentially transform the lives of patients in critical need, with the goal of delivering a chemotherapy-free option that is the first-and-only therapy that targets HER2-amplified BTC."

About Jazz Pharmaceuticals plc

Jazz Pharmaceuticals plc is a global biopharmaceutical company whose purpose is to innovate to transform the lives of patients and their families. We are dedicated to developing life-changing medicines for people with serious diseases—often with limited or no therapeutic options. We have a diverse portfolio of marketed medicines and novel product candidates, from early- to late-stage development, in neuroscience and oncology. Within these therapeutic areas, we are identifying new options for patients by actively exploring small molecules and biologics, and through innovative delivery technologies and cannabinoid science. Jazz is headquartered in Dublin, Ireland and has employees around the globe, serving patients in nearly 75 countries. 


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PAN-TB Collaboration Announces the Start of a Phase 2 Clinical Trial to Evaluate Two Novel Tuberculosis Treatment Regimens

PRNewswire | August 10, 2023

The Project to Accelerate New Treatments for Tuberculosis (PAN-TB) collaboration announced today the start of a phase 2b/c clinical trial, sponsored by the Bill & Melinda Gates Medical Research Institute (Gates MRI). The trial will evaluate whether novel regimens that combine registered products and new chemical entities have the potential to effectively treat drug-sensitive TB (DS-TB) and inform the development of a "pan-TB" regimen capable of treating all forms of active pulmonary TB. The regimens under evaluation are designed to explore shorter treatment durations compared to existing drug regimens, without the need for accompanying drug-resistance testing for individuals. The goal is to identify a candidate regimen suitable for phase 3 development. Trial sites are being hosted in countries with a high burden of TB. The trial has started in South Africa with the first participant screened in the last week of July. Subsequent trial sites are expected to launch in the Philippines in August and in Peru in 2024, pending national approvals. The collaboration is evaluating two novel drug regimens comprising five antibacterial agents—bedaquiline, delamanid, pretomanid, quabodepistat (formerly known as OPC-167832), and sutezolid DBQS – delamanid, bedaquiline, quabodepistat and sutezolid PBQS – pretomanid, bedaquiline, quabodepistat and sutezolid The trial employs an innovative design that consists of two stages to assess the efficacy of the regimens and evaluate the potential to shorten treatment duration. In the initial stage, the trial will enroll approximately 129 participants. The safety, tolerability, efficacy, and pharmacokinetics of the complete DBQS and PBQS regimens, with each drug administered daily for four months (17 weeks), will be evaluated in participants 18 to 65 years old with DS pulmonary TB. Approximately 43 participants will receive the six-month (26 weeks) standard-of-care treatment for DS-TB1 in the countries where trial sites are located.2 If successful, the second stage will enroll approximately 400 participants to test the selected regimen at shorter treatment durations, ranging from two to four months, across five arms. An additional 30-40 participants will be enrolled in the standard-of-care arm for the second stage of this trial. To support participants during their treatment, the trial integrates a Stop Treatment and Watch (STrAW) Concilium, a group made up of expert clinical consultants that will evaluate trial participants' treatment response, blinded to the specific regimen they receive, and advise trial investigators on patient management. The trial will utilize the best available tools including regular engagement with treatment support workers, direct observation of therapy with video technology, and medication reminder monitors. A novel Biomarkers Strategy will inform promising new biomarkers with the aim of enabling faster access to data to monitor treatment outcomes for future trials. The need for novel TB treatment regimens Despite the introduction of new medicines in recent years, treatment remains a long, complex, and demanding process for people diagnosed with TB and can place significant costs on households and the health system. The most used drug regimen for the treatment of DS-TB requires people to take multiple drugs for at least six months, often with routine monitoring. TB treatment can place unmanageable financial strain on individuals and their households. The World Health Organization (WHO) estimates that nearly half of TB-affected households face catastrophic total costs associated with treatment, including costs of travel to clinics, loss of income during treatment, and more, fueling cycles of poverty in vulnerable communities. Further, accurate, rapid drug resistance testing is essential for early diagnosis of both drug-sensitive and drug-resistant TB, but access to testing can be limited due to high costs, technical challenges, and other barriers. Emilio Emini, PhD, CEO, the Bill & Melinda Gates Medical Research Institute, said: "The Bill & Melinda Gates Medical Research Institute is committed to developing biomedical interventions that address global health concerns for those in the greatest need. The team at the Gates MRI looks forward to working with our partners across the PAN-TB collaboration, to evaluate the potential of these novel TB treatment regimens, in the hope that it may offer a potentially easier pathway for all people diagnosed with pulmonary TB by offering well-tolerated, shorter and simpler treatment options." Masanori Kawasaki, Global TB Project Leader, Otsuka Pharmaceutical Co. Ltd, said: "We are immensely proud to be part of this remarkable, trailblazing, collaboration which is now launching a clinical trial for a new, and shorter, pan-TB treatment regimen. We are excited too to be working closely with key partners to bring new treatment options to patients. Otsuka is dedicated to life-saving research and development as demonstrated by our sustained hard work and investment in developing a new chemical entity called quabodepistat. We are most grateful to the Bill & Melinda Gates Medical Research Institute for bringing together leaders in the field of TB, towards the goal of ending TB for good." Ruxandra Draghia-Akli, MD, PhD, Global Head of Global Public Health R&D, Janssen Research & Development, LLC, said: "Our medicine for multidrug-resistant TB has reshaped TB treatment regimens over the past decade, but more must be done to develop the even shorter, simpler regimens needed to treat all forms of the disease and end TB once and for all. Today's milestone is another step in this direction, and we look forward to seeing the results of this important study." Mel Spigelman, MD, President and CEO of TB Alliance, said: "Sincere thanks and congratulations to all who have made this innovative and exciting new journey a reality. Bringing revolutionary, highly impactful new treatments to all TB patients around the world is a goal that deserves and requires all our concerted dedicated efforts." About the Project to Accelerate New Treatments for Tuberculosis The Project to Accelerate New Treatments for Tuberculosis (PAN-TB) is a first-of-its-kind collaboration among philanthropic, non-profit and private sectors that aims to accelerate the development of an investigational drug regimen capable of treating all forms of tuberculosis. The PAN-TB collaboration leverages members' collective assets, resources and scientific expertise to identify and evaluate new drug regimens with an acceptable safety profile, that have the potential to treat both drug-sensitive and drug-resistant TB and are well-tolerated, shorter in duration and simpler to use than existing options. The collaboration focuses on advancing research through phase 2 clinical efficacy studies to identify promising regimens for further development. The PAN-TB collaboration plans to work closely and transparently with the European Regimen Accelerator for Tuberculosis (ERA4TB), which was launched in January 2020. New molecular entities identified by ERA4TB that show promise in initial human studies could later be incorporated into the PAN-TB collaboration's later-stage, clinical research. Several organizations, including Evotec, GSK, TB Alliance and Janssen Pharmaceutica NV, are members of both projects, which will help to ensure coordination across collaborations toward the common goal of advancing TB drug and regimen development. The members of the PAN-TB collaboration are Evotec, GSK, Janssen Pharmaceutica NV, Otsuka Pharmaceutical Co., Ltd., based in Japan, TB Alliance, the Bill & Melinda Gates Medical Research Institute and the Bill & Melinda Gates Foundation. Additional members may be announced in the future. About Evotec SE Evotec is a life science company with a unique business model that delivers on its mission to discover and develop highly effective therapeutics and make them available to the patients. The Company's multimodality platform comprises a unique combination of innovative technologies, data and science for the discovery, development, and production of first-in-class and best-in-class pharmaceutical products. Evotec leverages this "Data-driven R&D Autobahn to Cures" for proprietary projects and within a network of partners including all Top 20 Pharma and over 800 biotechnology companies, academic institutions, as well as other healthcare stakeholders. Evotec has strategic activities in a broad range of currently underserved therapeutic areas, including e.g., neurology, oncology, as well as metabolic and infectious diseases. Within these areas of expertise, Evotec aims to create the world-leading co-owned pipeline for innovative therapeutics and has to-date established a portfolio of more than 200 proprietary and co-owned R&D projects from early discovery to clinical development. Evotec operates globally with more than 4,900 highly qualified people.

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Business Insights

Mitsubishi Tanabe Pharma America Highlights Latest Research in Parkinson's Disease at EAN and WPC 2023 Congresses

prnewswire | July 06, 2023

Mitsubishi Tanabe Pharma America, Inc. (MTPA) today announced the presentation of seven abstracts in Parkinson's disease (PD) at the 9th Congress of the European Academy of Neurology (EAN), which was held in Budapest, July 1-3, and the 6th World Parkinson Congress (WPC), being held in Barcelona, July 4-7. "Our presence at EAN and WPC this year underscores MTPA's dedication to investigating potential treatment options to tackle the current unmet needs and challenges of Parkinson's disease," said Gustavo A. Suarez Zambrano, M.D., Vice President of Medical Affairs at MTPA. "We're excited to share our progress with the global scientific community, including the presentation of key findings from a number of clinical trials evaluating ND0612 in people with Parkinson's disease experiencing motor fluctuations." Clinical development of investigational ND0612 is being led by NeuroDerm, Ltd., a wholly-owned subsidiary of Mitsubishi Tanabe Pharma Corporation (MTPC), MTPA's parent company. If regulatory approval for ND0612 is obtained, MTPA intends to commercialize the therapy in the U.S. Presentations at EAN Presentations highlighted findings from two ongoing studies of investigational ND0612 in people with PD with motor fluctuations, including topline results from the Phase 3 multi-center, randomized, double-blind double-dummy (DBDD) BouNDless trial evaluating the efficacy, safety and tolerability of ND0612, in addition to three-year outcomes from the Phase 2b open-label BeyoND study evaluating the long-term safety of ND0612. Additionally, results were shared from an open-label pharmacokinetic study assessing the relative bioavailability of levodopa when administered with ND0612 vs. oral levodopa/carbidopa (LD/CD). 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NeuroDerm, Ltd. is a wholly-owned subsidiary of Mitsubishi Tanabe Pharma Corporation (MTPC), based in Israel, inspired to reduce disease burden and improve the quality of life of patients and their families through innovative drug-device combination therapies and technologies. NeuroDerm is an integrated pharmaceutical and medical technology company developing central nervous system (CNS) product candidates.

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Pharma Tech

Multistakeholder Collaborations are Vital to Advancing Technologies in Parkinson's

PRNewswire | July 10, 2023

Critical Path Institute (C-Path) and the Center for Health + Technology (CHeT) at University of Rochester today announced the release of two seminal publications about digital health technologies for Parkinson's. These technologies, such as smartphones and wearable sensors, offer an opportunity for objective, frequent and remote assessment of people with Parkinson's. With 10 million people living with Parkinson's worldwide, collaboration and data sharing are crucial for driving innovation in drug development for the fastest-growing degenerative neurological condition. Research leaders at CHeT have joined forces with organizations and regulators through C-Path's Critical Path for Parkinson's (CPP) Consortium, a precompetitive public-private partnership. CPP aims to accelerate the regulatory endorsement of digital tools in Parkinson's clinical trials through its Digital Drug Development Tools (3DT) initiative. In June 2023, the Journal of Parkinson's Disease published two open access manuscripts, representing companion papers from this partnership that detail steps in conducting interviews of people with Parkinson's who participated in a clinical research study using smartphones. The manuscripts report on relevant symptoms in early Parkinson's and assess the relevance of digital measures from a smartphone application and smartwatch from the patient perspective. The study was led by Jamie Adams, M.D., Associate Professor of Neurology and Associate Director of CheT at the University of Rochester and Jennifer Mammen, Ph.D., Assistant Professor at the University of Rhode Island, with support from CPP. "This project pioneers a new path to efficiently advance tools aligned with the patient voice by engaging with regulatory agencies early and often," said Diane Stephenson, Ph.D., CPP Executive Director. "C-Path remains committed to learning what matters most to people living with Parkinson's." "These manuscripts describe a novel methodology that will help inform the use of digital health technologies in clinical trials for Parkinson's disease and support more objective and patient-centered measures. The goal is to enable efficient, data-driven, and patient-centric utilization of digital health technologies to speed therapeutic development," said Adams. "Sharing the results with the study participants and witnessing how this information helped them in new ways has been most rewarding." John Crawford, a CPP advisor, Parkinson's advocate, and manuscript coauthor states, "Digital health technologies have the potential to significantly impact Parkinson's management and have already revolutionized clinical trial conduct. Their advancement expedites trials, enhances intelligence, reduces costs, and ultimately accelerates the delivery of new therapies and technologies to patients." The new publications coincide with CPP's participation in the World Parkinson's Congress taking place in Barcelona, Spain July 4-7, 2023. CPP will present two posters, Recommendations for Parkinson's Clinical Trials using Digital Health Technologies and Worldwide Collaborative Framework for Optimizing New Parkinson's Treatment Trials with Patient Centric Outcome Measures, at the conference coauthored by people living with Parkinson's highlighting the importance of worldwide collaborations. About Critical Path Institute Critical Path Institute (C-Path) is an independent, nonprofit organization established in 2005 as a public and private partnership. C-Path's mission is to catalyze the development of new approaches that advance medical innovation and regulatory science, accelerating the path to a healthier world. An international leader in forming collaborations, C-Path has established numerous global consortia that currently include more than 1,600 scientists from government and regulatory agencies, academia, patient organizations, disease foundations, and hundreds of pharmaceutical and biotech companies. C-Path U.S. is headquartered in Tucson, Arizona and C-Path Europe is headquartered in Amsterdam, Netherlands with additional staff in multiple other locations. C-Path FDA Acknowledgement The Center for Health + Technology (CHeT) is an academic research organization within the University of Rochester Medical Center. We are comprised of six units, with expertise in clinical trials coordination, clinical materials services, patient and caregiver-reported outcome measures, tech research and innovation, data modeling and predictive analysis, and health policy. Our skilled team of consultants are readily available to provide guidance to academic institutions, pharmaceutical companies, technology firms, not-for-profit foundations, advocacy groups, and the federal government.

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