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Olipudase alfa is an investigational enzyme replacement therapy designed to replace deficient or defective ASM, allowing for the breakdown of sphingomyelin.
ANX005 is a a monoclonal antibody (mAb) designed to fully block C1q and the entire classical complement pathway with the aim of preserving functioning synapses to slow or halt neurodegeneration. It is beig evaluated for GBS autoimmune disoder.
The active ingredient in QRX003 is a broad-spectrum serine protease inhibitor, whose mechanism of action is intended to down-regulate the hyperactivity of skin kallikreins, leading to a more normalized rate of skin shedding.
The active ingredient in QRX003 is a broad-spectrum serine protease inhibitor, whose mechanism of action is intended to down-regulate the hyperactivity of skin kallikreins, leading to a more normalized rate of skin shedding.
Following the results from the QR-110 (sepofarsen) Illuminate trial, the EMA has recommended an additional clinical trial be conducted for sepofarsen prior to submitting a Marketing Authorisation Application (MAA).
The acquisition of arimoclomol aligns perfectly with the strategy to build KemPharm’s value via the advancement and commercialization of novel treatments that address rare CNS conditions, including the lead clinical candidate, KP1077 in idiopathic hypersomnia.
Data showed OK-432,2, the originator compound for TARA-002 was clinically successful in treating lymphatic malformations and support a favorable safety profile.
ANAVEX®2-73 is an orally available drug candidate that restores cellular homeostasis by targeting sigma-1 and muscarinic receptors. Preclinical studies demonstrated its potential to halt and/or reverse the course of Alzheimer’s disease.
Sepofarsen (QR-110) is an investigational RNA therapy designed to restore vision in Leber congenital amaurosis 10 due to the c.2991+1655A>G mutation (p.Cys998X) in the CEP290 gene.
The study did not meet primary endpoint nor notable secondary endpoints for QR-110 (sepofarsen) an investigational RNA therapy and, no benefit observed in either treatment arm versus sham for the treatment of CEP290-mediated LCA10.
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