CAMBRIDGE, Mass.--(BUSINESS WIRE)--Triplet Therapeutics, Inc., a biotechnology company leveraging insights from the human genetics of repeat expansion disorders (REDs), today announced it has achieved its target enrollment in the SHIELD HD natural history study of Huntington’s disease (HD). The study, initiated in May of this year, exceeded its target enrollment of 60 Huntington’s disease gene expansion carriers ahead of schedule. It will now expand to secure further enrollment from European sites due to the high level of interest from the HD community.
“We are pleased to have completed enrollment of this natural history study faster than expected in just six months, despite the challenges posed by the COVID-19 pandemic,” said Irina Antonijevic, M.D., Ph.D., Triplet’s chief medical officer. “We are especially encouraged by the HD community’s enthusiasm for the study and incredibly grateful to the participants and investigators who are helping to accelerate research that can inform and advance into disease-modifying therapies.”
“Triplet’s approach to HD and other REDs is uniquely focused on the underlying genetic cause of somatic instability, which in turn drives disease onset and progression,” said Nessan Bermingham, Ph.D., Triplet’s chief executive officer, president, and founder. “This sets our approach apart from other HD therapies in development. Based on what we have seen with SHIELD HD, we believe patients and families share the excitement and confidence in our approach, and we are so pleased to partner with them to drive the study forward and learn more about the early clinical and biological signs of the disease.”
SHIELD HD is a prospective, longitudinal natural history study that will follow early manifest and premanifest individuals at nine clinical sites, spread across five countries and five U.S. states. It is on track for completion within two years.
The study assesses a range of clinical outcomes and biomarkers including cognitive, motor, and functional measures such as the composite Unified HD Rating Scale (“cUHDRS”), brain MRI, DNA Damage Response (DDR) gene expression, and the blood and spinal fluid protein biomarker neurofilament light chain. The duration of SHIELD HD is designed to observe a rate of change in key clinically meaningful outcomes. SHIELD HD results will augment data from upcoming clinical trials and inform their interpretation.
More information about the study is available on ClinicalTrials.gov (NCT04406636).
About Huntington’s disease
Huntington’s disease is a genetic disorder linked to a mutation in the HTT gene characterized by an increase in the number of CAG repeats within the gene. The presence of these repeats, beyond a certain threshold, wreaks havoc on brain function, affecting mood, cognition, and motor skills, ultimately leading to death. Thanks to the contributions of thousands of patients – whose participation in genetic research has built a fundamentally new understanding of the cause of REDs – it is now known that the number of repeat sequences expands over time in patients, increasing the toxic impact on cells, particularly neurons. The DDR pathway plays a central role in driving this process.
About Triplet Therapeutics
Triplet Therapeutics is a biotechnology company developing transformational treatments for patients with repeat expansion disorders (REDs) – a group of more than 50 known genetic diseases including myotonic dystrophy type 1 (DM1), Huntington’s disease (HD), spinocerebellar ataxias (SCAs), fragile X syndrome, and familial amyotrophic lateral sclerosis (ALS) – leveraging insights from patient genetics. Triplet designs and develops potential therapeutics for REDs using its proprietary thRED Engine, which enables the Company to develop a single oligonucleotide targeting the DNA Damage Response (DDR) pathway to potentially treat multiple REDs.
Triplet is headquartered in Cambridge, Mass. For more information, please visit www.triplettx.com.