LONDON--(BUSINESS WIRE)--The Loulou Foundation, a private foundation dedicated to the development of therapeutics for the neurodevelopmental condition CDKL5 Deficiency Disorder (CDD), announced today that the first patient has been enrolled in the three-year observational study with CDD patients, the Clinical Assessment of NeuroDevelopmental measures In CDD (CANDID) study. The CANDID study is directed by a novel consortium of seven biopharmaceutical industry partners, together with the Loulou Foundation, to enable the development of disease-modifying therapeutics for CDD. The seven companies are: Amicus Therapeutics [$FOLD]; Biogen Inc. [$BIIB]; Elaaj Bio; Marinus Pharmaceuticals Inc. [$MRNS]; PTC Therapeutics [$PTCT]; UCB SA [$UCB]; and Ultragenyx [$RARE]. The Loulou Foundation serves as the study coordinator, while the seven partners share the funding and governance of this study involving CDD clinical centers worldwide.
CDD is a genetic disorder whose symptoms include difficult-to-treat epilepsy and severe neurodevelopmental delay that affects patients' cognitive function, motor skills, and social interaction. With an incidence of one in 42,000 live births, CDD is one of the most common genetic pediatric epilepsies, affecting tens of thousands of patients worldwide. There are currently no approved treatments that address the disorder's neurodevelopmental symptoms. Most CDD patients require around-the-clock care and management of their complex symptoms.
The CANDID study will investigate the baseline and longitudinal performance of known and validated instruments and scales, measuring cognitive, motor, and developmental domains in up to 100 CDD patients. The CANDID study was the subject of a Critical Path Innovation Meeting with the U.S. Food and Drug Administration in December of 2020. The study design incorporates input from the agency. More information on the CANDID study can be found here.
"This first CDD patient enrollment in the CANDID study is an exciting milestone and a landmark first step in the development of disease-modifying therapeutics to treat this devastating disorder. The Loulou Foundation and our pre-competitive consortium partners are grateful for the sacrifices made by our CDKL5 community families participating in this observational study, which will be invaluable in moving forward therapies that go beyond management of seizures,” said Xavier Liogier, Ph.D., Chief of Translational Science at the Loulou Foundation.
Several new treatment options are entering clinical development that researchers hope will address both the seizures and the neurodevelopmental deficits of CDD. CANDID will provide researchers with information on appropriate tests evaluating disease-modifying therapeutics for CDD, such as gene therapies, enzyme replacement, genome editing, or small molecules. CANDID study results will be eventually shared with the entire community to aid CDD clinical trial design and inform therapeutic development for related neurodevelopmental disorders.
"The international CDD patient community fully supports this important study led by the Loulou Foundation and the industry consortium," said Mr. Jainu Jogani, Chair of the CDKL5 Alliance, an international organization of CDD patient family groups. "CDD families world-wide are excited by the potential for the CANDID study to help move the CDD field beyond measuring seizures to more comprehensive outcome measures that will support therapies targeting the underlying biology of CDD."
CDKL5 Deficiency Disorder (CDD) is a neurodevelopmental disorder caused by loss-of-function mutations in the CDKL5 gene, which encodes a kinase necessary for the proper function of neurons. CDD usually presents as seizures or infantile spasms within the first months of life, which later progresses to epilepsy. CDD patients display profound neurodevelopmental delay, particularly in cognitive, social, and motor function, and most CDD patients require constant care. With an incidence of approximately one in 42,000 live births, CDD is one of the most prevalent monogenic epilepsies, affecting tens of thousands of people worldwide. While some CDD patients can have partial control of their epilepsy with current anti-seizure medications, there are no approved treatments for the neurodevelopmental delay associated with CDD.
About the Loulou Foundation:
The Loulou Foundation is a private non-profit organization founded in 2015 to support the development of effective therapeutics and eventual cures for CDD. In 2016, the Loulou Foundation partnered with the Orphan Disease Center at the University of Pennsylvania’s Perelman School of Medicine to create the CDKL5 Program of Excellence within the Orphan Disease Center. Through this partnership, the Foundation funds grant and directed research programs to develop tools and resources to advance CDD therapeutic research. These programs include pre-clinical and clinical research into CDKL5 biology, CDD disease mechanisms, and proof-of-concept studies for gene therapy and genome modifying therapeutics.
About the CDKL5 Alliance:
The CDKL5 Alliance is an international organization that serves to unite and align the community of national and regional CDD patient family groups worldwide. Founded in 2017, the CDKL5 Alliance provides coordination and communication to its thirty-five member organizations and support to fledgling CDD patient family groups as they start to organize themselves. More information on the CDKL5 Alliance can be found at www.CDKL5Alliance.org.
University of Pennsylvania Financial Disclosure:
Penn has previously licensed certain CDD technologies to some of the industry members of the pre-competitive consortium.