INDUSTRIAL IMPACT, MEDICAL
Globenewswire | March 27, 2023
Iovance Biotherapeutics, Inc. a late-stage biotechnology company developing novel T cell-based cancer immunotherapies, announced it has completed its rolling Biologics License Application (BLA) submission to the U.S. Food and Drug Administration (FDA) for lifileucel. Lifileucel is a tumor infiltrating lymphocyte (TIL) therapy intended as a treatment for patients with advanced melanoma who progressed on or after prior anti-PD-1/L1 therapy and targeted therapy, where applicable. There are no FDA approved therapies in this treatment setting.
Frederick Vogt, Ph.D., J.D., Interim President and Chief Executive Officer of Iovance, stated, “Completing our BLA submission for lifileucel is a critical step forward in our journey to deliver the first individualized, one-time cell therapy for a solid tumor. I would like to acknowledge the patients and physicians who participated in the C-144-01 clinical trial and the FDA review team for their commitment and support, as well as our internal team for their tremendous effort in completing the first BLA submission for Iovance. Our preparations for commercialization remain on track to support a launch later this year. We look forward to continued collaboration with the FDA as they review this new class of treatment for advanced melanoma patients with limited options.”
The BLA submission for lifileucel is supported by positive clinical data from the C-144-01 clinical trial in patients with advanced post-anti-PD1 melanoma. Following a successful pre-BLA meeting with the FDA, Iovance is pursuing accelerated approval in this indication. Iovance also reached agreement with the FDA regarding the registrational trial design for the Phase 3 TILVANCE-301 trial of lifileucel in combination with pembrolizumab in frontline advanced melanoma. TILVANCE-301 is intended to support full approval of lifileucel in post-anti-PD-1 advanced melanoma and is also designed to support registration for lifileucel in combination with pembrolizumab as therapy for advanced melanoma in the frontline setting. Startup activities for TILVANCE-301 are ongoing and the trial is expected to be well underway at the time of potential accelerated approval for lifileucel in advanced post-anti-PD-1 melanoma.
Marc Hurlbert, Ph.D., CEO of the Melanoma Research Alliance (MRA), said, “MRA congratulates Iovance for completing the BLA submission and moving closer toward making TIL therapy an option for people with advanced melanoma who have progressed following prior treatments. We hope for an FDA approval as quickly as possible for patients with significant unmet need who have no approved treatment options.”
Following receipt of the complete rolling BLA submission for lifileucel, the FDA has 60 days to determine the acceptability of the BLA for review. The rolling BLA allowed Iovance to submit portions of the BLA to the FDA on an ongoing basis, enabling the FDA to begin review as early as possible as documents were received. The rolling BLA submission and eligibility for priority review are benefits available under the FDA’s guidance on expedited programs for serious conditions, which allow for an expedited six-month review from the time of BLA acceptance. In addition, the FDA previously granted a regenerative medicine advanced therapy (RMAT) designation for lifileucel in advanced melanoma.
About Iovance Biotherapeutics, Inc.
Iovance Biotherapeutics aims to be the global leader in innovating, developing and delivering tumor infiltrating lymphocyte (TIL) therapies for patients with cancer. We are pioneering a transformational approach to cure cancer by harnessing the human immune system’s ability to recognize and destroy diverse cancer cells in each patient. Our lead late-stage TIL product candidate, lifileucel for metastatic melanoma, has the potential to become the first approved one-time cell therapy for a solid tumor cancer. The Iovance TIL platform has demonstrated promising clinical data across multiple solid tumors. We are committed to continuous innovation in cell therapy, including gene-edited cell therapy, that may extend and improve life for patients with cancer.
MEDTECH, INDUSTRIAL IMPACT
Prnewswire | April 06, 2023
ConcertAI, LLC a leader in AI Software-as-a-Service (SaaS) technology and Real-world Evidence solutions for life sciences and healthcare, announced the release of its next generation of Clinical Trial Optimization (CTO 2.0).
"Trial sponsors have a range of new imperatives they're trying to meet: assuring that the trial population is reflective of the ultimate population that would have access to the drug; to set and meet diversity goals for those ethnic, racial and economic groups that are uniquely, negatively impacted by a disease; and meeting ever higher productivity imperatives for identifying and activating sites faster and working with those sites most likely to accrue to the pre-activation feasibilities," said Jeff Elton, Ph.D. CEO of ConcertAI. "Meeting these goals requires working with sites and investigators not historically preferred, designing trials for a minimum of burden on research sites and patients and, perhaps most importantly, moving more studies into community-based research settings."
CTO 2.0 now includes a range of features: (1) clinical depth and tools, such as line of therapy assessments, Kaplan-Meier survival curves, and cox-proportional hazard assessments that guide alternative designs with the largest underlying research dataset for oncology; (2) data integrating electronic medical-derived, medical claims, and social determinants of health; (3) site- and investigator-level information on current and previous clinical trials derived from public and private sources; and (4) operational trial metrics and site profile information to highlight performance and capabilities.
The solution's foundation is a study digitization layer that can handle any depth of inclusion and exclusion (I/E) criteria. Protocols can be assessed relative to standard-of-care in different settings and in different time periods, all the way down to the individual event or clinical activity level. There are APIs for integrating past study protocols from any legacy systems of records. Optimizer and recommendations engines can provide study-level optimizations and semi-automate site selection. Additional APIs can integrate past site survey data and investigator databases as a means of simplifying and unifying what are often non-integrated solutions within the clinical analytics and clinical operations organization. The SaaS technology will support researchers and clinicians in selecting the most appropriate sites for clinical trials, balancing potential patients available and previous trial performances at those sites to give the highest likelihood of success for a clinical trial. In assurance that trials can be conducted across settings and utilize the latest digital trial solutions that support direct EMR to EDC, CTO 2.0 leverages the latest clinical informatics integrations and data standards to ensure research can be conducted at scale and meeting, or exceeding, enhanced standards of care.
"In the past, clinicians used previously published protocols and recently approved studies in the same disease to inform trial designs for new studies," said Ronan Brown, ConcertAI's Chief Operating Officer. "Now that is changing. We'll now see greater reliance on large scale, multi-sourced, clinical datasets that have low latency, reflecting the latest treatments, supporting new designs, lower site burden, greater, diversity, and expanded views of relevant sites. It's a significant step forward in clinical trial planning and operations."
ConcertAI is a leader in Real-World Evidence (RWE) and AI technology solutions for life sciences and healthcare. Our mission is to accelerate insights and outcomes for patients through leading real-world data, AI technologies, and scientific expertise in partnership with the leading biomedical innovators, healthcare providers, and medical societies.
Puma Biotechnology, Inc. | March 16, 2023
Puma Biotechnology, Inc., a biopharmaceutical company, recently announced the online publication of the Phase II TBCRC041 randomized clinical trial results in JAMA Oncology. The trial investigated the use of alisertib, an adenosine triphosphate–competitive and reversible inhibitor of aurora kinase A, alone or in combination with fulvestrant in postmenopausal women with endocrine-resistant, HER2-negative metastatic breast cancer who were previously treated with fulvestrant.
The Phase II randomized clinical trial was conducted via the Translational Breast Cancer Research Consortium and included patients previously treated with CDK 4/6 inhibitors and everolimus.
The trial enrolled 91 evaluable patients, with baseline characteristics balanced between the two arms of the trial. However, more patients in the alisertib plus fulvestrant arm had previously received chemotherapy for metastatic disease (47.8% in the alisertib alone arm vs. 68.9% in the alisertib plus fulvestrant arm). In each arm of the trial, all patients had earlier been treated with CDK 4/6 inhibitors. Everolimus was previously administered to 37% of patients in the alisertib alone arm and 57.8% in the alisertib plus fulvestrant arm.
The trial's efficacy results indicated that nine partial responses were observed in the 46 evaluable patients in the alisertib alone arm, leading to an overall response rate of 19.6%. The median duration of response was 15.1 months, with a clinical benefit rate of 41.3% at 24 weeks. The projected median progression-free survival (PFS) was 5.6 months. Nine of the 45 evaluable patients in the alisertib plus fulvestrant arm of the study responded, for an overall response rate of 20.0%. There was one patient who had a complete response and eight patients who showed partial responses. The median duration of response was 8.5 months, with a clinical benefit rate of 28.9% at 24 weeks. The median PFS was expected to be 5.4 months.
The most prevalent grade 3 or higher adverse events in the alisertib alone arm of the trial were neutropenia (43.4%), anemia (19.6%) and leukopenia (17.4%). The most prevalent grade 3 or higher adverse events in the alisertib plus fulvestrant arm of the study were neutropenia (42.2%), leukopenia (31.1%), lymphopenia (15.6%), fatigue (11.1%), and anemia (8.9%).
About Puma Biotechnology, Inc.
Puma Biotechnology, Inc. is a leading biopharmaceutical company specializing in the development and commercialization of innovative treatments for cancer. Its flagship product is Nerlynx (neratinib), an oral tyrosine kinase inhibitor approved by the USFDA for the extended adjuvant treatment of HER2-positive early-stage breast cancer. In addition to Nerlynx, it has a robust pipeline of product candidates in various stages of development. The company is firmly committed to research and development and collaborates with leading academic institutions and research organizations to advance the understanding of cancer and develop new therapies.