COUR Pharmaceuticals | January 11, 2022
COUR Pharmaceuticals, a biotechnology company developing novel immune-modifying nanoparticles to treat immune disorders, today announced the U.S. Food and Drug Administration granted Fast Track Designation to the Company's investigational therapy CNP-104 for the treatment of Primary Biliary Cholangitis. CNP-104 aims to reprogram the immune system to address autoimmune causes of PBC.
"We are pleased to have received Fast Track Designation for CNP-104, which demonstrates the FDA recognizes the importance of this potential treatment for PBC and the need for improved of clinical outcomes for patients with this degenerative autoimmune disease, which currently has no treatment to stop progression of disease. Based on data from another COUR autoimmune trial with a CNP platform product, our Phase 2 PBC trial is designed to show that we can stop progression of PBC disease with improved liver function. If successful in Phase 2, COUR would seek breakthrough therapy designation in PBC given that current therapies treat only symptoms."
COUR CEO John Puisis
Fast Track Designation is an FDA process designed to facilitate the development and review of potential therapies that seek to fill an unmet medical need. The FDA created this designation to help get new therapies to patients earlier. A therapy that receives this designation is eligible for more frequent communication with the FDA and may be eligible for Accelerated Approval and Priority review, if relevant criteria are met.
The FDA accepted COUR's investigational new drug application for CNP-201 in October 2021 and COUR is currently opening the first human dosing in a proof-of-concept study for CNP-104 in early 2022 to evaluate the safety, tolerability, pharmacodynamics and efficacy of the therapy in PBC patients. CNP-104 uses COUR's proprietary nanoparticle platform, a novel system which combines disease specific pathogenic antigens with state-of-the-art pharmaceutical nanoparticles that mimic normal removal of dead or dying liver cells from the body.
In November 2021, Ironwood Pharmaceuticals, Inc., a GI-focused healthcare company entered into a collaboration and license option agreement with COUR for the development and commercialization of CNP-104. This agreement gives Ironwood an option to acquire an exclusive license to develop and commercialize, in the U.S., COUR's investigational therapy CNP-104 for PBC.
About COUR Pharmaceuticals
COUR Pharmaceuticals is developing first-in-class therapies designed to reprogram the immune system to achieve antigen-specific tolerance for immune-mediated disease. COUR's platform of immune-modifying nanoparticles is designed to treat the root cause of immune disease, unlike traditional approaches, which only minimize symptoms using toxic immune suppression. Data from clinical and preclinical settings demonstrate the opportunity for the COUR nanoparticle platform to address a wide range of immune and inflammatory conditions. The underlying technology was acquired from Northwestern University and draws from more than 30 years of research by the laboratory of Stephen D. Miller, Ph.D., the Judy E. Guggenheim Research Professor of Microbiology-Immunology.
Ascentage Pharma | January 05, 2021
Ascentage Pharma (6855.HK), an internationally engaged, clinical-stage biotechnology organization occupied with creating novel treatments for malignancies, persistent hepatitis B (CHB), and age-related sicknesses, today reported that the US Food and Drug Administration (FDA) has allowed an Orphan Drug Designation (ODD) to the organization's novel Bcl-2 inhibitor, APG-2575, for the therapy of patients with intense myeloid leukemia (AML). This is the fourth ODD allowed to APG-2575 by the US FDA, following the past ODDs for the therapy of Waldenström macroglobulinemia (WM), ongoing lymphocytic leukemia (CLL), and numerous myeloma (MM). Until this point, four of Ascentage Pharma's investigational drug up-and-comers have been allowed an aggregate of nine ODDs by the US FDA, a record number for any Chinese biopharmaceutical organization.
AML is an exceptionally heterogenous hematologic threat that is more normal in the old populace with a middle age at analysis of 68 years1. The latest information from the Surveillance, Epidemiology, and End Results Program (SEER) of the US National Cancer Institute (NCI) assessed 19,940 new instances of AML and 11,180 passings from this infection in the United States in 2020. Notwithstanding the critical advances in therapeutics as of late, the 5-year endurance pace of AML stays at 25%–30%, which actually speaks to a huge neglected clinical requirement for treatments with more solid viability and a superior wellbeing profile.
The expression "vagrant medications" alludes to drug items produced for the counteraction, finding, and treatment of uncommon illnesses or conditions. In the United States, a vagrant sign is characterized as a sickness or condition with a pervasiveness of under 200,000 patients in the nation. Since the Orphan Drug Act was passed in 1983, the US government has given motivators and strategy backing to support advancement of vagrant medications. Treatments conceded ODDs by the FDA are equipped for different advancement motivators, including a tax break on consumptions brought about in clinical examinations, a waiver of the New Drug Application (NDA) charge, conceivable exploration award granted by the FDA, and above all, 7 years of US market restrictiveness upon endorsement.
Panaxia Global, Neuraxpharm | July 22, 2020
Neuraxpharm, a leading European pharmaceutical company specialized in CNS, and Panaxia Global, the controlling owner of Panaxia Labs Israel (Panaxia Israel) (TASE: PNAX), Israel's largest medical cannabis manufacturer, announced today they have entered a definite agreement to start a business collaboration for marketing Panaxia's medical cannabis products in Germany, Europe's largest and fastest-growing market for cannabis in Europe. The agreement, which anchors a previous MOU entered by the companies, includes exclusivity for Panaxia products, as well as an option for scaling the distribution to additional countries, also including further major European countries.
The agreement is one of a kind in the cannabis industry. It represents the first time a global pharma company of Neuraxpharm caliber marketing medical cannabis products as part of its broad prescription and non-prescription product portfolio. On top of distributing the products to pharmacies through local distributors, the venture will focus on marketing efforts to the relevant target audiences in Germany, namely the prescribing physicians, and the patients. The marketing activity is critical for the success of commercial sales and will be implemented according to the standards used in the pharmaceutical industry.