BALTIMORE and SAN DIEGO, Nov. 23, 2015 /PRNewswire/ -- Kennedy Krieger Institute and Viking Therapeutics, Inc. ("Viking") (NASDAQ: VKTX), a clinical-stage biopharmaceutical company focused on the development of novel therapies for metabolic and endocrine disorders, today announced a sponsored research collaboration focused on evaluating the company's novel thyroid beta agonists for the treatment of X-linked adrenoleukodystrophy (X-ALD). Under terms of the agreement, Kennedy Krieger's X-ALD research scientists will study Viking's proprietary thyroid beta agonists, including VK0214, in in vivo models of X-ALD.
This research will build upon previous cell-based data demonstrating that the company's thyroid beta agonists positively affect the expression of specific genes relevant to X-ALD. In the planned work, researchers at the Kennedy Krieger will utilize an in vivo X-ALD model to assess the impact of VK0214 on key biomarkers of the disease. The successful completion of this work will support Viking's initiation of IND-enabling activities with the goal of advancing VK0214 into clinical studies.
"Collaborating with the Kennedy Krieger Institute is invaluable to the advancement of our thyroid beta agonists as potential treatments for this rare, often fatal, disease," said Brian Lian, Ph.D., chief executive officer of Viking. "The Institute's expertise in X-ALD is unrivalled, and the opportunity to evaluate our compounds in the group's in vivo models will generate critical scientific insight as we advance this program forward. X-ALD is a debilitating disease for which an adequate treatment is desperately needed. We're focused on understanding whether our proprietary thyroid agonists hold potential benefits for these patients."
"Scientific research suggests that thyroid beta agonists may play a role in the treatment of X-ALD and we are excited to collaborate with Viking to assess the potential of the company's novel collection of compounds in this area," stated Ali Fatemi, M.D., Director of Neurogenetics and the Moser Center for Leukodystrophies at Kennedy Krieger Institute. "This effort furthers our commitment to improving the lives of individuals with X-ALD, and all those living with disorders of the brain, spinal cord and musculoskeletal system."
Financial terms of the agreement have not been disclosed.
X-ALD is a rare and often fatal metabolic disorder characterized by a breakdown in the protective barriers surrounding brain and nerve cells; a process known as demyelination. The disease, for which there is no approved treatment, is caused by mutations in a peroxisomal transporter of very long chain fatty acids (VLCFA), known as ABCD1. As a result, transporter function is impaired and patients are unable to efficiently metabolize VLCFA. The resulting accumulation can trigger a rapid, inflammatory demyelination, which leads to cognitive impairment, motor skill deterioration, and even death. X-ALD is estimated to occur in approximately 1 in 17,000 births.
The thyroid beta receptor is known to regulate expression of an alternative VLCFA transporter, known as ABCD2. Various preclinical models have demonstrated that increased expression of ABCD2 can lead to normalization of VLCFA metabolism.
About Kennedy Krieger Institute
Internationally recognized for improving the lives of children and adolescents with disorders and injuries of the brain, spinal cord and musculoskeletal system, the Kennedy Krieger Institute in Baltimore, MD, serves more than 20,000 individuals each year through inpatient and outpatient clinics, home and community services and school-based programs. Kennedy Krieger provides a wide range of services for children with developmental concerns mild to severe, and is home to a team of investigators who are contributing to the understanding of how disorders develop while pioneering new interventions and earlier diagnosis. For more information on the Kennedy Krieger Institute, visit www.kennedykrieger.org.
About Viking Therapeutics, Inc.
Viking Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on the development of novel, first-in-class or best-in-class therapies for metabolic and endocrine disorders. The company's research and development activities leverage its expertise in metabolism to develop innovative therapeutics designed to improve patients' lives. Viking has exclusive worldwide rights to a portfolio of five therapeutic programs in clinical trials or preclinical studies, which are based on small molecules licensed from Ligand Pharmaceuticals Incorporated. The company's clinical programs include VK5211, an orally available, non-steroidal selective androgen receptor modulator, or SARM, in Phase 2 development for the treatment and prevention of lean body mass loss in patients who have undergone hip fracture surgery, VK2809, a small molecule thyroid beta agonist entering Phase 2 development for hypercholesterolemia and fatty liver disease, and VK0612, a first-in-class, orally available drug candidate in Phase 2 development for type 2 diabetes. Viking is also developing novel and selective agonists of the thyroid beta receptor for adrenoleukodystrophy, as well as two earlier-stage programs targeting metabolic diseases and anemia.
This press release contains forward-looking statements regarding Viking Therapeutics, including statements about Viking's expectations regarding its development activities, timelines and milestones, as well as the company's goals and plans regarding VK0214. Forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially and reported results should not be considered as an indication of future performance. These risks and uncertainties include, but are not limited to: risks associated with the success, cost and timing of Viking's product candidate development activities and clinical trials; and risks regarding regulatory requirements, among others. These forward-looking statements speak only as of the date hereof. Viking disclaims any obligation to update these forward-looking statements.
SOURCE Viking Therapeutics, Inc.; Kennedy Krieger