Patient registries are a hot topic of rare disease research and many organizations are taking advantage of this resource by signing up their patient communities and connecting with researchers. Eric Sid, MD, program officer for the Office of Rare Diseases Research (ORDR), said it is difficult to estimate how many registries exist in the U.S.…
Seelos Therapeutics is gearing up for a pilot study, in Australia, testing SLS-005 (trehalose) — its investigational therapy for people with Alzheimer’s disease — after receiving an acknowledgment letter from the country’s regulatory body for clinical trials. The letter, from the Australian Government Department of Health Therapeutic Goods Administration (TGA), was issued after the company…
Eisai has started submitting data to the Japanese Pharmaceuticals and Medical Devices Agency (PMDA) in support of an application for approval of its investigational lecanemab (BAN2401) to treat early Alzheimer’s disease. This submission followed the company’s prior assessment consultation with the PMDA, given in advance of an approval application and intended to accelerate its review by identifying and addressing…
The therapy Sarclisa (isatuximab) extended survival by nearly seven months in relapsed/refractory multiple myeloma patients also given Pomalyst (pomalidomide) and dexamethasone as standard treatment, according to updated three-year data from the ICARIA-MM Phase 3 trial. The data, “Isatuximab plus pomalidomide and low-dose dexamethasone versus pomalidomide and low-dose dexamethasone in patients with relapsed and refractory multiple myeloma…
Roche is launching a Phase 3 clinical trial to evaluate the ability of its investigational antibody therapy, gantenerumab, to slow Alzheimer’s progression in people with early signs of the disease, the company announced. Called SKYLINE (NCT05256134), the trial is now recruiting 1,200 cognitively unimpaired participants between ages 60 and 80 who show signs of brain…
Horizon Therapeutics has launched its #RAREis Representation program aimed at increasing diversity, equity, and inclusion among patients with rare diseases. There are about 400 million people worldwide living with a rare disease; for many of them, access to diagnosis, care, and treatments can be challenging. Accessing better care depends on several factors, including the social determinants of…
LX1001, a one-time gene therapy for Alzheimer’s disease being developed by Lexeo Therapeutics, raised levels of the protective APOE2 protein in the cerebrospinal fluid (CSF), early data from an ongoing Phase 1/2 trial shows. Findings in a first patient group treated at low dose also showed the gene therapy reduced the levels of tau protein — a hallmark…
The U.S. Food and Drug Administration (FDA) has given fast track designation to Harpoon Therapeutics’ HPN217 as a potential immunotherapy for people with relapsed, refractory multiple myeloma (RRMM) who have tried at least four prior therapies. Fast track status is given to therapies that show potential in addressing serious conditions for which available treatments fall short. It is…
A clinical trial application for varoglutamstat, or PQ912, an investigational therapy for Alzheimer’s disease, was approved in China, its developers, Vivoryon Therapeutics and Simcere Pharmaceutical Group, announced. “We are proud to have obtained [China’s Center for Drug Evaluation] CDE approval of our Clinical Trial Application of varoglutamstat which represents our strong commitment in the battle against…
A biomarker blood test called AlzoSure Predict that can help to identify people with mild cognitive impairment who are likely progress to Alzheimer’s disease — years before symptoms are evident — has been approved for use in the European Union (EU) and the U.K., its marketer, Diadem, announced. “This first regulatory approval for AlzoSure Predict … represents…
