CELL AND GENE THERAPY, INDUSTRIAL IMPACT
Ligand Pharmaceuticals | February 21, 2023
In a recent announcement by biopharmaceutical firm, Ligand Pharmaceuticals, the U.S. Food and Drug Administration (FDA) has given accelerated approval for FILSPARITM (sparsentan). This will be helpful in treating adults with primary IgAN condition who are at risk of rapid disease progression, typically defined as a urine protein-to-creatinine ratio (UPCR) of less than 1.5 g/g.
FILSPARI, a once-daily oral medicine, is the first and only non-immunosuppressive therapy authorized for the treatment of IgAN. Two critical pathways are selectively targeted of IgAN (endothelin-1 and angiotensin II) to check disease progression. Up to 150,000 Americans may have the rare kidney illness IgAN, which is the main cause of kidney failure brought on by glomerular disease. Among these patients, it is predicted that 30,000 to 50,000 can be treated using the indication that has received accelerated approval. Travere has stated that it anticipates FILSPARI to be available from February 27, 2023, and it will offer a thorough patient support program all the way through the patient's therapeutic journey.
Eric Dube, Ph.D., President and CEO of Travere Therapeutics, said, “The accelerated approval of FILSPARI is a significant milestone on our path to advancing a transformative treatment for the IgA nephropathy community.” He further added, “As a first-of-its-kind, non-immunosuppressive therapy, we believe FILSPARI has the potential to ultimately become the new standard of care for IgA nephropathy and offer hope to those living with this condition who until now have had few treatment options. We are grateful to the patients, caregivers, clinical trial investigators, healthcare providers, and advocates who have worked alongside us to develop this innovative first-in-class therapy.”
(Source – Business Wire)
About Ligand Pharmaceuticals
Ligand Pharmaceuticals creates or acquires technologies that aid in the discovery and development of drugs by pharmaceutical firms. The company’s business model generates value for stockholders by offering a diverse portfolio of biotech and pharmaceutical product income streams, that are underpinned by an effective and low corporate cost structure.
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.
Quantum-Si | January 10, 2023
The leading Protein Sequencing Company™, Quantum-Si, recently announced that it signed a partnership and license agreement with Biovista to offer customers more significant proteomic insights beyond their protein sequence output.
Biovista utilizes artificial intelligence in multiple formats to analyze enormous data repositories and visualize non-obvious networks and connections between proteins, diseases, and drug mechanisms of action. The partnership enhances Quantium-Si's services significantly by making proteomic-to-drug workflows more efficient for researchers.
Biovista's VIZIT™ exploration tool will be incorporated into Quantum-Si's cloud-based suite of analytic tools, allowing researchers to visualize the connections between the protein sequences identified during their experiment to diseases, other proteins, and post-translational modifications. Thus, researchers can identify potential disease-specific mechanisms and biomarkers more efficiently and effectively.
"Quantum-Si's protein sequencing technology in concert with Biovista's database and visualization technology can aid in the discovery of new proteins for future therapeutic targets associated with disease," said Jeff Hawkins, CEO of Quantum-Si. He further stated, "The potential can also extend to discovering new biomarkers for clinical research and diagnostics."
(Source- Business Wire)
"Quantum-Si is leading the next generation of real-world meaningful sequencing," said Dr. Aris Persidis, Biovista's Co-Founder and President. "We are excited to see deep sequencing and insight generation now available in one integrated platform," he concluded.
(Source- Business Wire)
Quantum-Si, The Protein Sequencing CompanyTM, is dedicated to revolutionizing the rapidly expanding field of proteomics. It provides the first-of-its-kind, end-to-end, universal single-molecule detection solutions, allowing to build, experiment, and discover on its platform regardless of their existing products. In addition, its proprietary Time Domain Sequencing technology eliminates the reliance on color as an identification method, a barrier for any application outside of genomics, where only four colors are required.