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The financing will be used to fund the upcoming Phase 3 studies of ColiFin (colistimethate sodium), an inhaled antibiotic therapeutic for the management of chronic infections in cystic fibrosis patients.
r-hIL-18BP (tadekinig alfa) is a recombinant human interleukin-18 binding protein inhibiting IL18. It captures excess free IL-18 mimicking the normal physiological situation, and therefore represents a well differentiated approach for the treatment of IL-18-opathies.
This collaboration is expected to provide capitalization through significant clinical milestones in ColiFin®’s Phase 3 development. ColiFin® (colistimethate sodium) is being developed for the treatment of chronic lung infections in cystic fibrosis (CF) patients.
Under the license agreement between Roche and Chugai, Chugai obtained exclusive marketing right in Japan for delandistrogene moxeparvovec (SRP-9001), an investigational gene therapy for Duchenne muscular dystrophy.
Lucerastat demonstrated a substantial reduction in levels of the Fabry-disease biomarker plasma Gb3 after 6 months of treatment. The potential effect of lucerastat on kidney function over 6 months of treatment was evaluated at the interim analysis of the extension study.
The safety, tolerability, pharmacodynamics, and pharmacokinetics of oral lucerastat were evaluated in study in adult patients with Fabry disease. In this single-center, open-label, randomized study, 10 patients with Fabry disease were randomized to lucerastat for 12 weeks.
STEER will build upon the Phase 1/2 study which showed that treatment with OAV-101 IT led to significant increases in Hammersmith Functional Motor Scale-Expanded scores and a clinically meaningful response in older patients between ≥2 years and <5 years old with SMA Type 2.
New clinical study to evaluate safety and efficacy of Zolgensma in children up to 21 kg, adding to real-world experience and regulatory approvals in Europe and Canada.
Tominersen, previously IONIS-HTTRx or RG6042, is an investigational antisense therapy designed to reduce the production of all forms of the huntingtin protein (HTT), including its mutated variant, mHTT.
Data from an interim analysis conducted by the independent Data and Safety Monitoring Board concluded that the Phase 3 SIDEROS study with Puldysa® (idebenone) was unlikely to meet its primary endpoint.