CMP-SCN / OPKO Health, CAMP4 Therap - LARVOL DELTA

SCN1A Upregulation by an Antisense Oligonucleotide Targeting SCN1ANAT for Treatment of Dravet Syndrome (AES 2022) - "We have shown that ASO targeting of SCN1A-NAT is a viable therapeutic approach for Dravet Syndrome, and we are advancing a lead ASO into clinical testing. Our findings will inform the design of IND-enabling GLP tox studies with CMP-SCN-001 targeting human SCN1A-NAT."

CNS Disorders • Epilepsy

CAMP4 Presents In Vivo Data Supporting Novel Therapeutic Strategy for Dravet Syndrome at American Epilepsy Society Annual Meeting (Businesswire) - "CAMP4 Therapeutics...announced the presentation of in vivo proof-of-concept data demonstrating the potential therapeutic utility of its lead oligonucleotide candidate for the treatment of Dravet Syndrome, a form of severe genetic epilepsy. The data, which show increases in SCN1A gene expression in non-human primates and reductions in seizures in mouse disease models, will be presented at the American Epilepsy Society (AES) Annual Meeting being held virtually and in person in Chicago, IL from December 3-7, 2021...Based on these data, CAMP4 will pursue clinical development for CMP-SCN with the expectation of filing an Investigational New Drug Application with the U.S. Food and Drug Administration by the end of 2022."

IND • Preclinical • CNS Disorders • Epilepsy

OPKO Health, CAMP4 Therapeutics Ink Oligonucleotide Platform With Focus On Drug-Resistant Epilepsy (Yahoo Finance) - "OPKO Health Inc...has entered into an exclusive worldwide agreement with privately-held CAMP4 Therapeutics Corporation (CAMP4) to develop, manufacture, and commercialize therapeutics utilizing the AntagoNAT technology...CAMP4 has prioritized OPKO's lead AntagoNAT compound to progress into clinical trials for Dravet syndrome...OPKO will be eligible to receive up to $93.5 million and additional shares as milestone payments. CAMP4 will also pay OPKO double-digit royalties on product sales."

Commercial • CNS Disorders • Epilepsy