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CRISPR Gene Editing Therapy AllowsHereditary Angioedema Patients Experience 98% Reduction in Attack Rate
Intellia Therapeutics today announced long-term data from the Phase 1 portion of the ongoing Phase 1/2 study of investigational therapy NTLA-2002.NTLA-2002 is a CRISPR gene-editing therapy being developed as a one-time treatment for hereditary angioedema (HAE).HAE is a rare genetic disease that causes potentially life-threatening swelling attacks.
In all patients,At a median follow-up of 20.1 months, a single treatment with NTLA-2002 reduced patients' average monthly disease attack rate by 98% and moderate to severe attacks by an average of 99%.At each dose level tested, the HAE attack rate was significantly reduced. After a single infusion,The longest seizure-free period for the patient exceeded 26 months., and it is still ongoing. In addition, the effect of reducing attacks was also very significant in patients with the most severe HAE symptoms. The two patients with the highest monthly HAE attack rates (16.8 and 14.0 times/month, respectively) at the start of the study were attack-free at the end of the 16-week primary observation period and remained attack-free at the latest follow-up. The longest attack-free duration for these two patients was 23.5 months, and it is still ongoing. Additionally,100% of patients treated with NTLA-2002 no longer required prophylactic treatment after discontinuing other preventive therapies.NTLA-2002 is an in vivo CRISPR gene editing candidate therapy that delivers the CRISPR-Cas9 gene editing system in the form of mRNA via lipid nanoparticles (LNP), targeting the knockout of the plasma prekallikrein gene (KLKB1), to permanently reduce plasma kallikrein activity, thereby preventing hereditary angioedema attacks.
Eli Lilly Spends Over $600 Million to Acquire Potential "Best-in-Class" ASO Therapy for Neurodegenerative Diseases
QurAlis Corporation Announces Exclusive License Agreement with Eli Lilly and Company for Global Development and Commercialization of QRL-204, a Potential "Best-in-Class" Splicing Modulation Antisense Oligonucleotide (ASO) Therapy Designed to Restore UNC13A Function in Amyotrophic Lateral Sclerosis (ALS), Frontotemporal Dementia (FTD), and Other Neurodegenerative Diseases.
QurAlis to Receive $45 Million Upfront Payment and Additional Equity Investment. Furthermore, QurAlis is eligible for up to $577 million in milestone payments. The agreement includes a research and development collaboration utilizing QurAlis’ proprietary FlexASO splicing modulation platform to identify and develop additional UNC13A-targeted drug candidates. QurAlis’ FlexASO splicing modulation platform is designed to generate splicing modulating ASOs with higher potency and an improved therapeutic index.QurAlis' ASOs Can CorrectUNC13AThe erroneous splicing, restoring the production of UNC13A protein, and reducing the cryptic exons that may lead to disease progression.
Takeda's Oral Orexin Receptor 2 Agonist Meets Phase 2 Clinical Endpoint
Takeda released positive results from the Phase 2b clinical trial of its investigational therapy TAK-861 for the treatment of Type 1 Narcolepsy (NT1) today.TAK-861 is an oral orexin receptor 2 (OX2R) agonist.This randomized, double-blind, placebo-controlled clinical trial (n=112) met both primary and secondary endpoints.
The primary endpoint of the Phase 2b trial showed,In the Maintenance of Wakefulness Test (MWT), all dose groups showed statistically significant and clinically meaningful improvements in sleep latency compared to the placebo group (p≤0.001). The improvements were sustained over 8 weeks.
The study also achieved consistent results in key secondary endpoints, including the Epworth Sleepiness Scale (ESS) and weekly cataplexy rate (WCR), showing significant improvements in subjective sleepiness and cataplexy frequency compared to the placebo group, with sustained efficacy over 8 weeks. At the end of the 8-week treatment period, the MWT and ESS results for the majority of NT1 patients were within the normal range.
NT1 is a chronic, rare hypersomnia disorder of the central nervous system, characterized by significantly reduced levels of orexin neuropeptides in the brain and cerebrospinal fluid due to the substantial loss of orexin neurons. Patients with NT1 experience symptoms such as excessive daytime sleepiness (EDS), cataplexy, disrupted nighttime sleep, and hallucinations upon falling asleep or waking. These debilitating symptoms substantially decrease quality of life and severely impact work performance, academic achievement, and interpersonal relationships. TAK-861 aims to address the orexin deficiency in NT1 by selectively stimulating orexin receptors.
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[1] Intellia Therapeutics Announces Positive Long-Term Data from Ongoing Phase 1 Study of NTLA-2002, an Investigational In Vivo CRISPR Gene Editing Treatment for Hereditary Angioedema (HAE). Retrieved June 3, 2024, from https://ir.intelliatx.com/news-releases/news-release-details/intellia-therapeutics-announces-positive-long-term-data-ongoing[2] Takeda’s TAK-861 Phase 2b Late-Breaking Data Presentations at SLEEP 2024 Demonstrate Clinically Meaningful Impact of Oral Orexin Agonist in Narcolepsy Type 1 Compared to Placebo. Retrieved June 3, 2024, from https://www.takeda.com/newsroom/newsreleases/2024/tak-861-data-at-sleep-2024-on-narcolepsy-type-1/[3] QurAlis Grants Lilly Exclusive Global License for QRL-204, a Potentially First-in-Class Precision Therapy That Restores UNC13A Function in ALS and FTD. Retrieved June 3, 2024, from https://www.prnewswire.com/news-releases/quralis-grants-lilly-exclusive-global-license-for-qrl-204-a-potentially-first-in-class-precision-therapy-that-restores-unc13a-function-in-als-and-ftd-302156943.html
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