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.
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.
CELL AND GENE THERAPY, INDUSTRIAL IMPACT
Evaxion | February 10, 2023
Evaxion Biotech A/S, a clinical-phase biotech company specializing in enhancing AI-powered immunotherapies, and Pantherna Therapeutics GmbH, a biopharmaceutical company developing enhanced nanoparticle solutions for delivering and expressing mRNA therapeutics, announced a preclinical proof of concept for the combination of their key technologies.
Using Pantherna's unique lipid nanoparticle mRNA technology (PTXLNP®, PTXmRNA®), tumor neoantigens discovered by Evaxion's artificial intelligence (AI) platform drive a robust immune response and completely limit tumor development in a preclinical model.
Birgitte Rønø, CSO at Evaxion, said, “We are extremely pleased with the preclinical data that clearly demonstrate the potential for our combined technologies. The data provide further evidence that immunogenic sequences identified by Evaxion’s AI platforms are able to drive a relevant immune response on multiple vaccine platforms, including mRNA. This opens up many opportunities in our current and future immuno-oncology and infectious disease programs.”
(Source – GlobeNewswire)
CEO at Pantherna, Klaus Giese, stated, “We are encouraged by the data and see an enormous potential of combining Evaxion’s AI-based antigen discovery engines with our state-of-the-art mRNA-LNP platforms. The preclinical data substantiate the relevance of enhancing the efficiency of mRNA actions in the body and validates the ability of our platform to induce an immune response.”
(Source – GlobeNewswire)
Evaxion and Pantherna will continue investigating optimum LNP formulations for the efficient delivery of mRNA- and DNA-encoded antigens found by Evaxion's AI systems.
Evaxion Biotech A/S is a clinical-stage biotech firm working on AI-powered immunotherapies. Evaxion's patented and scalable artificial intelligence algorithms decode the human immune system to find and develop innovative immunotherapies for cancer, bacterial illnesses, and viral infections. It is developing a diverse pipeline of innovative product candidates, including three tailored cancer immunotherapies. It is listed on the Nasdaq New York Stock Exchange and is headquartered in Hørsholm, Denmark.