NEW YORK, Sept. 15, 2020 /PRNewswire/ -- Corino Therapeutics, Inc., a private, clinical-stage pharmaceutical company, announced today that Dr. John Berk, M.D., Assistant Director of the Amyloidosis Center at Boston University School of Medicine, is presenting positive findings from a proof-of-concept study of oral CRX-1008 in patients with hereditary leptomeningeal Transthyretin Amyloidosis (hATTR-leptomeningeal) at the XVII International Symposium on Amyloidosis. hATTR-leptomeningeal is an under recognized, progressive and fatal disease caused by accumulation in the brain of variant transthyretin (TTR) expressed by the choroid plexus, causing central nervous system (CNS) dysfunction. No available therapies exist for hATTR patients with amyloid-related CNS dysfunction.
The study, conducted by Dr. John Berk, M.D., evaluated both peripheral (blood) and central (cerebrospinal fluid) TTR stabilization in hATTR-leptomeningeal patients after oral dosing of CRX-1008 for 28 days. The results confirm that CRX-1008 is a potent TTR stabilizer in both plasma and CSF using established ex vivo assays. CRX-1008 normalized TTR concentration in plasma with an overall mean increase in plasma TTR tetramer concentration of 55%. Significant concentrations of drug penetrated into the CSF decreasing monomeric TTR concentration by a mean of 48% as measured under semi-denaturing conditions.
"These new clinical research findings suggest that CRX-1008 may be the first hope for patients with hATTR-leptomeningeal, treating both peripheral and central manifestations of hATTR", said Dr. John Berk, M.D., Associate Professor of Medicine at Boston University School of Medicine.
CRX-1008 was well tolerated in patients with no drug related adverse events or safety concerns.
"We are looking forward to conducting additional clinical trials designed to confirm the potential benefits of CRX-1008 in patients with both peripheral and central manifestations of hATTR, generating data needed for regulatory approval", stated Dr. Michael Roberts, Ph.D., Chief Executive Officer of Corino Therapeutics, Inc.
CRX-1008 is an investigational and potent small molecule stabilizer of TTR. Given orally, it inhibits TTR tetramer dissociation into monomeric TTR and prevents accumulation of amyloid in various tissues. CRX-1008 has unique and differentiated activity compared to other kinetic stabilizers, including the ability to cross an intact blood brain barrier.
Transthyretin Amyloidosis (ATTR) is a group of rare and potentially fatal diseases characterized by systemic deposition of a protein-derived material known as amyloid, in one or more organ systems (including the peripheral and autonomic nervous systems, the heart, the brain and the eyes). Transthyretin Amyloidosis occurs when wild type or mutant TTR becomes unstable, misfolds and aggregates into amyloid fibrils. The TTR amyloid deposits cause damage to the surrounding cells and tissues leading to a variety of clinical symptoms. There is a large unmet need for drugs that treat all forms of ATTR amyloidosis affecting major organs and the brain. Global prevalence of ATTR is estimated to be around 450,000.
About Corino Therapeutics, Inc.
Corino Therapeutics, Inc. is a private, development-stage biopharmaceutical company focused on acquiring, developing, and commercializing innovative products for the treatment of a variety of human diseases, particularly orphan indications. Corino develops technologies that address important unmet medical needs, and which offer improved, cost-effective alternatives to current methods of treatment. For more information, please visit www.corinorx.com.
Michael Roberts, Ph.D.
Corino Therapeutics, Inc.
SOURCE Corino Therapeutics, Inc.