Oral Therapy
VIDEO: Combination treatments can increase compliance for PI3K inhibitors
In this video, Aruna Padmanabhan, MD, discusses the evaluation of PI3K inhibitor combinations as first-line treatment for patients with metastatic breast cancer.
Linsitinib for thyroid eye disease shows proptosis reduction, safety in phase 2b/3 trial
Linsitinib, an oral small molecule under development for the treatment of thyroid eye disease, demonstrated proptosis reduction and safety in the phase 2b/3 LIDS trial, according to a press release from Sling Therapeutics.
Etavopivat could offer ‘great benefit’ in sickle cell disease
SAN DIEGO — Etavopivat reduced the annualized vaso-occlusive crises rate vs. placebo for people with sickle cell disease, according to study data presented at ASH Annual Meeting and Exposition.
FDA grants rare pediatric disease, fast track designations to Stargardt disease therapy
The FDA granted rare pediatric disease and fast track designations to gildeuretinol, an investigational oral therapy for the treatment of Stargardt disease, according to a press release from Alkeus Pharmaceuticals.
First investigational oral therapy lowers Lp(a) in phase 2 study
CHICAGO — Adults with elevated liproprotein(a) at high risk for a CV event saw substantial reductions over 12 weeks with muvalaplin, an investigational oral small molecule Lp(a) inhibitor, data from a phase 2 study show.
VIDEO: Oral Stargardt’s disease treatment focuses on reducing vitamin A dimerization
CHICAGO — In this Healio Video Perspective from Eyecelerator@AAO, Michel Dahan, CEO of Alkeus Pharmaceuticals discusses ALK-001, the company’s oral therapy for the treatment of Stargardt’s disease and age-related macular degeneration.
Oral treatment aims to reduce GA progression by slowing vitamin A dimerization
CHICAGO — Decreasing vitamin A dimerization may be a promising treatment avenue for treating geographic atrophy, according to a speaker here.
NIH awards $3.3 million for oral disease-modifying Alzheimer’s, Parkinson’s therapeutic
The NIH has awarded a $3.3 million grant to advance a novel, oral silent allosteric modulator intended to treat Alzheimer’s and Parkinson’s diseases, according to the manufacturer.
FDA grants fast track designation to brepocitinib for noninfectious uveitis
The FDA granted fast track designation to brepocitinib for the treatment of nonanterior noninfectious uveitis, according to a press release from Priovant Therapeutics.
FDA approves Miplyffa for Niemann-Pick disease type C
The FDA announced in a press release that it has approved Miplyffa to treat neurological symptoms associated with Niemann-Pick disease type C in patients aged 2 years and older.