BUSINESS INSIGHTS, PHARMA TECH
Tonix Pharmaceuticals | February 14, 2023
On February 13, 2023, Tonix Pharmaceuticals, a leading clinical-stage biopharmaceutical firm, announced that it has exercised an option to acquire an exclusive license from Columbia University to develop a portfolio of fully human (TNX-3600) and murine (TNX-4100) monoclonal antibodies (mAbs) for the prophylaxis or treatment of SARS-CoV-2 infection. COVID-19 is caused by SARS-CoV-2. Tonix Pharmaceuticals and Columbia University announced a research collaboration and option agreement in 2020, which resulted in the development of the licensed mAbs.
Although the FDA granted emergency use authorization (EUA) to five mAb products containing seven distinct mAbs for either prophylaxis or treatment of COVID-19, none stayed available or functional since January 26, 2023, when the FDA stated that the last remaining mAb, Evusheld®, is no longer authorized. Previously, either FDA or the National Institutes of Health COVID Treatment Guidelines Panel had revoked recommendations or clearances for the other mAbs.
Until recently, the earlier EUA-approved products were obtained from the blood of humanized mice or COVID-convalescent patients. Columbia University's TNX-3600 fully human mAbs were generated utilizing a proprietary system that involved a human hybridoma fusion partner.
The firm feels that murine monoclonal antibodies, such as TNX-4100, have the capacity to generate antibodies with a high affinity that detect distinct epitopes on the SARS-CoV-2 spike protein. This is due to the fact that mice have a different antibodies repertoire than humans, and the technology for making antibodies optimizes the selection of relevant B cells via harvesting approach, immunization timing and screening platform.
About Tonix Pharmaceuticals
Founded in 2007, Tonix Pharmaceuticals is a clinical-stage biopharmaceutical firm committed to finding, acquiring, licensing, and developing therapies for the treatment and prevention of diseases and the alleviation of human suffering. Its pipeline is focused on rare diseases, immunology, infectious diseases, and central nervous system (CNS) product candidates. The company's immunology portfolio consists of biologics for the treatment of organ transplant rejection, cancer and autoimmunity. At the same time, its infectious disease portfolio includes next-generation COVID-19 vaccines and vaccinations against smallpox and monkeypox.
RESEARCH, PHARMA TECH
Ionis Pharmaceuticals, Inc. | March 10, 2023
Ionis Pharmaceuticals, a leading RNA-targeted therapy firm, recently announced that the USFDA has accepted for review a new drug application (NDA) for eplontersen. Eplontersen is an investigational antisense medicine designed to reduce the production of transthyretin (TTR) protein and treat hereditary and non-hereditary forms of ATTR amyloidosis (ATTR).
The NDA has been given a Prescription Drug User Fee Act (PDUFA) action date of December 22, 2023. The FDA has declared that no review concerns were detected and made no new data requests. The FDA also stated that it does not intend to conduct a meeting of the advisory committee to discuss the application.
ATTR amyloidosis is a systemic disease caused by the accumulation of TTR protein in organs, leading to progressive organ damage and eventually death. Patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) experience ongoing debilitating nerve damage throughout their bodies, resulting in the progressive loss of motor function.
The NDA for eplontersen is based on results from the global Phase 3 NEURO-TTRansform study showcased at the International Symposium on Amyloidosis (ISA). In the study, eplontersen showcased a statistically significant and clinically meaningful change relative to the baseline for its co-primary and critical secondary endpoints compared to the external placebo group. In addition, Eplontersen also demonstrated a favorable safety and tolerability profile.
Ionis and AstraZeneca announced a strategic partnership to develop and commercialize eplontersen in December 2021. Eplontersen is being developed and commercialized jointly by both businesses in the United States and will be created and commercialized by AstraZeneca throughout the rest of the globe, except Latin America.
The FDA granted eplontersen Orphan Drug Designation in the United States in January 2022. Eplontersen is also being studied in the worldwide Phase 3 CARDIO-TTRansform trial for transthyretin amyloid cardiomyopathy (ATTR-CM), a systemic, progressive, and deadly disorder leading to progressive heart failure and death within 3 to 5 years after disease start.
About Ionis Pharmaceuticals, Inc.
Ionis Pharmaceuticals, Inc. is a leading biopharmaceutical company headquartered in Carlsbad, California. Its unique antisense technology platform allows them to develop RNA-targeted therapies that modify the production of disease-causing proteins, leading to the development of treatments for rare genetic disorders, cardiovascular diseases, and neurodegenerative diseases. The company's most significant achievement is developing and commercializing Spinraza, the first approved treatment for spinal muscular atrophy. Ionis Pharmaceuticals has also developed drugs for other rare diseases, including transthyretin amyloidosis, Huntington's disease, and myotonic dystrophy type 1.
BioMarin Pharmaceutical Inc. | March 08, 2023
On March 7, 2023, BioMarin Pharmaceutical Inc., a global biotech company focused on improving lives via genetic discovery, announced that the US FDA has accepted its supplemental New Drug Application (sNDA) for VOXZOGO® (vosoritide) for injection to expand the treatment of achondroplasia in children under the age of 5.
Achondroplasia is a common form of disproportionate short stature. The FDA has set an action date of October 21, 2023, for the sNDA. The sNDA is supported by data from a Phase 2 double-blind, randomized, placebo-controlled clinical trial, which showed similar safety and efficacy profiles in children under five years of age compared to those aged five years and older.
The European Medicines Agency (EMA) also validated BioMarin's application for VOXZOGO in January to treat children under the age of 2. If approved, VOXZOGO could be prescribed for over 1,000 additional children from birth for achondroplasia.
VOXZOGO is the first EMA and FDA-approved treatment for achondroplasia with open bone growth plates. The treatment uses a new class of therapy, C-type natriuretic peptide (CNP) analog, which promotes bone growth by acting as a positive regulator of the signaling pathway downstream of the fibroblast growth factor receptor 3 gene (FGFR3).
The drug is approved in the EU, Brazil, and Australia for children aged two years and above with epiphyses (bone growth plates) and in Japan for children from birth who have achondroplasia with open growth plates. BioMarin has enrolled 250 children with achondroplasia in seven clinical studies in eight countries to evaluate the safety and efficacy of VOXZOGO.
About BioMarin Pharmaceutical Inc.
BioMarin is a leading biotechnology firm established in 1997 with a mission to transform people's lives by using genetic discovery. The company creates targeted therapies that address the underlying cause of genetic disorders to improve the quality of life for individuals with rare genetic diseases. It has developed eight first or best-in-class treatments and has a range of product candidates to address various genetic disorders using the same science-based approach. As BioMarin continues to create new and innovative solutions, it has the potential to impact the lives of even more people. The company is committed to prioritizing patients and their needs in its approach to drug development.