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.
CELL AND GENE THERAPY, MEDICAL
PRNewswire | May 03, 2023
BioCentriq, Inc. and panCELLa, a Pluristyx Company, announced that they have signed a research agreement designed to evaluate the ability of panCELLa's genetically-engineered, induced Pluripotent Stem Cell derived, feeder cells to activate and positively impact the expansion rate, total yield and potency of manufactured NK cells.
BioCentriq, a contract development and manufacturing organization focused exclusively on accelerating the development and manufacturing of cell therapies, will expand and cryopreserve the feeder cell bank for use as a reagent to activate adult and iPSC-derived NK cells and evaluate viable cell yield and potency in static and dynamic culture conditions.
panCELLa developed the feeder line from proprietary, genetically-modified, iPSCs to serve as a critical raw material for easy adoption by therapy developers and their partners to overcome the challenges associated with making NK cell therapies. The panCELLa feeder line provides a stable and easily scalable platform that can be fine-tuned to maximize NK cell activation, expansion, and manufacturability. Companies wishing to utilize panCELLa's feeder line can access a research version for evaluation and a clinical-grade format for therapeutic use through panCELLa and its sub-licensees.
"We envision the panCELLa-provided feeder line as an effective tool in our quest to accelerate the manufacturing of NK cell therapies and can be added to BioCentriq's proprietary LEAP™ platform," said Alex Klarer, Vice President of Business Strategy and Innovation at BioCentriq. "Our LEAP-NK™ platform is specifically designed to help biotech companies with promising NK cell therapies advance from lab to clinic in half the time it would normally take by leveraging proven technology, completed development work and established processes ready for GMP production."
Mahendra Rao, Chief Scientific Officer at Pluristyx, said, "We are pleased that BioCentriq selected our feeder line technology to better serve their cell therapy clients in solving the challenges of NK cell commercial manufacturing. We look forward to working with BioCentriq to bring the next generation of customized cell products and service solutions to speed the transition of these revolutionary therapies from the benchtop to the bedside."
BioCentriq is a full-service, New Jersey-based CDMO for cell and gene therapy, focusing on all stages of process development and clinical manufacturing. It was purchased by GC of South Korea for $73M. With over 90 scientists, engineers, analysts, and manufacturing specialists, the company has established quality systems and the infrastructure required to support the release of autologous and allogeneic drug products.
Premier Research | March 14, 2023
Premier Research has partnered with InSilicoTrials, a leader in using artificial intelligence (AI) and computational modeling and simulation (CM&S) to speed up the development of new therapies and medical devices. Premier Research helps biotech and device companies take their ideas from the idea stage to the commercialization stage. The aim of the partnership is to make it easier, faster, and safer for treatments for rare diseases to get approval from the government.
CM&S and AI have made it possible to use virtual patients in clinical trials in whole or in part, which the FDA approves of in some cases. In silico trials make it possible to create fake control or treatment arms, help figure out how to sign up patients, and predict the safety and effectiveness of new drugs and medical devices more accurately.
The use of synthetic control arms generated using in silico CM&S techniques represents a powerful tool for enabling non-feasible rare disease research, both preclinical and clinical research. In the last five years, Premier Research has been in charge of more than 240 rare disease studies for a wide range of indications. This shows that the company is committed to orphan drug development for a long time. Premier Research is continuing to put money into trials for rare diseases by partnering with InSilicoTrials.
One major advantage of in silico simulation is the ability to investigate 'what if' scenarios, particularly in rare diseases where a larger patient population does not exist. By using biological, molecular, or genetic data to describe rare diseases, in silico models can make synthetic control arms. This, along with Premier Research's expertise in clinical and preclinical development, execution, and regulatory aspects of small population trials, can shorten the time it takes to make effective treatments for rare diseases.
The partnership between Premier Research and InSilicoTrials will focus on smarter in silico study design, leading to a more effective pre-clinical review of trial design parameters and faster submission-ready studies. CEO of InSilicoTrials Luca Emili says that combining modeling and simulation with AI is the best way to improve the R&D process in drug development. This is because it allows sponsors to speed up the development of new treatments, make medical products safer, and lower R&D costs.
About Premier Research
Premier Research is a global company that does clinical research, product development, and consulting. It focuses on using new technologies to design and run smart studies and trials. With more than 2,500 employees in 75 countries, it has a lot of experience in certain therapeutic areas and uses. The company is committed to empowering its customers to meet the unmet needs of patients and offers great benefits and flexible working conditions.