Primary Biliary Cholangitis

CHICAGO, Oct. 30, 2024 (GLOBE NEWSWIRE) -- COUR Pharmaceuticals, a clinical-stage biotechnology company focused on the development of first-in-class, disease-modifying therapies designed to induce antigen-specific tolerance for immune-mediated diseases, today announced that the first patient has been dosed in its Phase 1b/2a proof-of-concept clinical trial evaluating CNP-106 as a potential treatment for people living with generalized myasthenia gravis (gMG). “Currently approved treatments for gMG work via broad immunosuppression and do not address the underlying autoimmunity of the disease,” said Dannielle Appelhans, President and Chief Executive Officer of COUR. “In preclinical studies to date, CNP-106 has demonstrated a unique ability to induce tolerance to acetylcholine receptor (AChR), leading to improved muscle function in an experimental model of autoimmune myasthenia gravis. These results indicate that CNP-106 has the potential to be a first-in-class treatment that offers people living with gMG a disease modifying therapy without burdensome immune system suppression.” The Phase 1b/2a double-blind, randomized, placebo-controlled clinical trial (NCT06106672) will enroll up to 54 patients with gMG, aged 18-75, to evaluate the safety, tolerability, pharmacodynamics, and efficacy of multiple ascending doses of CNP-106. About CNP-106CNP-106 is a biodegradable nanoparticle that encapsulates acetylcholine receptor (AChR). gMG is primarily driven by the production of antibodies against AChR in the bloodstream. These antibodies either block or destroy the receptors for acetylcholine, which is a crucial neurotransmitter for muscle contraction. As gMG progresses, it may lead to serious health issues, such as myasthenic crisis, tumors of the thymus gland, thyroid imbalances, and other autoimmune diseases. CNP-106 is designed to potentially induce the downregulation of AChR T cell populations and AChR antibodies, leading to improved muscle function. About COUR Pharmaceuticals:COUR Pharmaceuticals is a clinical-stage biotechnology company developing therapies to treat patients with autoimmune diseases. COUR’s first-in-class therapies are based on our proprietary antigen-specific immune tolerance platform and are designed to reprogram the immune system to address the underlying root cause of immune-mediated diseases. Data from multiple clinical and preclinical programs have demonstrated the ability of COUR’s product candidates to induce antigen-specific immune tolerance and have the potential to treat a wide range of autoimmune diseases. COUR is currently enrolling patients in a Phase 1b/2a double-blind, placebo-controlled, multicenter clinical study in Myasthenia Gravis and developing a product candidate in Type 1 Diabetes in addition to having completed first-in-human clinical studies in Celiac Disease (partnered with Takeda Pharmaceuticals), and Primary Biliary Cholangitis. For more information, please visit www.courpharma.com ContactsFor Investor RelationsBrian Bock, Chief Financial Officerbbock@courpharma.com For MediaJason Bracojbraco@lifescicomms.com

Detailed safety and efficacy results to be presented Data show potential of CNP-104 to slow disease progression as demonstrated by statistically significant decrease in liver stiffness compared with placebo Oct. 21, 2024 -- COUR Pharmaceuticals, a clinical-stage biotechnology company developing first-in-class, disease-modifying therapies designed to induce antigen-specific tolerance for autoimmune diseases, today announced it will present detailed safety and efficacy results from the Phase 2a study of CNP-104 in Primary Biliary Cholangitis (PBC) via a late-breaking poster at The Liver Meeting® 2024, being hosted by the American Association for the Study of Liver Disease (AASLD) from November 15-18 in San Diego, California. Presentation Details: Title: Tolerogenic treatment with CNP-104 results in regulation Th17 cells slowing progression of PBC on liver stiffness Presenter: Paul M. Peloso, M.D., COUR Chief Medical Officer Publication Number: 5039 Poster Session Date and Time: Monday, November 18, 2024, from 1:00 to 2:00 p.m. PT CNP-104 is a biodegradable nanoparticle encapsulating the E2 component of the mitochondrial pyruvate dehydrogenase complex (PDC), a key autoantigen in PBC. CNP-104 aims to address the root cause of PBC by inducing tolerance to pathogenic PDC-E2 T-cells that drive inflammation in bile ducts, resulting in clinically improved outcomes in liver health. In January 2022, CNP-104 received Fast Track Designation from the United States Food and Drug Administration (FDA), making it potentially eligible for Accelerated Approval and Priority Review, if relevant criteria are met. COUR Pharmaceuticals is a clinical-stage biotechnology company developing therapies to treat patients with autoimmune diseases. COUR’s first-in-class therapies are based on our proprietary antigen-specific immune tolerance platform and are designed to reprogram the immune system to address the underlying root cause of immune-mediated diseases. Data from multiple clinical and preclinical programs have demonstrated the ability of COUR’s product candidates to induce antigen-specific immune tolerance and have the potential to treat a wide range of autoimmune diseases. COUR is currently enrolling patients in a phase 1b/2a double-blind, placebo-controlled, multicenter clinical study in Myasthenia Gravis and developing a product candidate in Type 1 Diabetes in addition to having completed first-in-human studies in Celiac Disease (partnered with Takeda Pharmaceuticals), and Primary Biliary Cholangitis. The content above comes from the network. if any infringement, please contact us to modify.

Ipsen’s Iqirvo (elafibranor) has been recommended by the National Institute for Health and Care Excellence (NICE) to treat adults with the rare liver disease primary biliary cholangitis (PBC). The drug can now be used in combination with ursodeoxycholic acid (UDCA) in patients with an inadequate response to UDCA, or as a monotherapy in those who are unable to tolerate UDCA. The recommendation makes Iqirvo the first medicine for PBC to be approved for use on the NHS in nearly a decade and comes just two weeks after the Medicines and Healthcare products Regulatory Agency granted marketing authorisation to the drug in this setting. PBC is characterised by the build-up of bile and toxins (cholestasis) and chronic inflammation, which leads to irreversible scarring of the liver and destruction of the bile ducts. The disease affects approximately 25,000 people in the UK, the majority of whom are women, and is associated with symptoms such as persistent, debilitating itch, known as pruritus, and severe fatigue. The condition can also worsen over time and cause liver failure if not effectively treated. Taken orally once daily, Iqirvo is a peroxisome proliferator-activated receptor agonist that decreases bile toxicity and improves cholestasis by modulating bile acid synthesis, detoxification and transporters. NICE’s decision on the drug was supported by positive results from the phase 3 ELATIVE trial, in which 51% of patients receiving Iqirvo in combination with UDCA achieved a cholestasis response at week 52, compared to 4% of those in the placebo plus UDCA group. Additionally, 15% of Iqirvo-treated patients demonstrated ALP normalisation versus 0% on placebo at week 52, and a greater decrease in pruritus intensity was observed in Iqirvo-treated patients compared to placebo, but this did not reach statistical significance. Iqirvo will be made immediately available via the Innovative Medicines Fund, which facilitates faster access to new treatments while further data is collected. David Montgomery, UK and Ireland medical director at Ipsen, said: “NICE’s decision comes at a pivotal time as currently available treatments do not effectively manage both PBC disease progression and life-impacting symptoms like itch and sleep disturbances. This approval is an important step for us in improving outcomes for people living with PBC…”