Treatment with highest dose pegtibatinase led to a clinically meaningful 55% mean reduction in total homocysteine from baseline and was generally well-tolerated after 12 weeks of treatment Results provide clinical proof of concept for first potential therapy targeting the underlying enzyme deficiency in classical homocystinuria SAN DIEGO – Travere Therapeutics,...
treatment News
SAN DIEGO – Travere Therapeutics, Inc. (NASDAQ: TVTX) today announced completion of patient enrollment in the Phase 3 PROTECT Study. The pivotal PROTECT Study is evaluating the safety and efficacy of sparsentan for the treatment of IgA nephropathy (IgAN), a rare kidney disorder that often progresses to end-stage kidney disease...
SAN DIEGO, Calif. — Travere Therapeutics, Inc. (Nasdaq: TVTX) today announced the submission of a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for conversion of the existing U.S. accelerated approval of FILSPARI® (sparsentan) in IgA nephropathy (IgAN) to full approval. In February 2023, the...
San Diego, Calif. – Travere Therapeutics on Friday released more data from the Phase III PROTECT and DUPLEX studies—in IgA nephropathy and focal segmental glomerulosclerosis, respectively—showing that treatment with Filspari (sparsentan) can preserve kidney function in these diseases. The results were presented at the American Society of Nephrology’s Kidney Week...
TORONTO, Ontario & SAN FRANCISCO, Calif. — Treadwell Therapeutics, a privately held clinical-stage biotechnology company pioneering and advancing novel first-in-class medicines for unmet needs in cancer, today announced the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to ocifisertib (CFI-400945), a first-in-class, investigational PLK4 inhibitor for the...
A wave of new therapies is starting to reshape the treatment landscape for people with rare anemias, but the authors of a new review also caution that more research is needed to better understand which therapies are right for which patients. Writing in Frontiers in Medicine, the authors began by...
Atlanta – In a post hoc analysis of the phase 2 NOBILITY trial, researchers found that treatment with obinutuzumab—an antibody that targets a protein expressed on certain immune cells—was superior to placebo for preserving kidney function and preventing flares in patients with lupus nephritis, a kidney condition associated with the...
ROCKVILLE, Md. – GlycoMimetics, Inc. (Nasdaq: GLYC) announced today that clinicians have treated the first patient in a Phase 1b/2 study evaluating the company’s lead drug candidate, uproleselan, added to cladribine plus low dose cytarabine (LDAC) in patients with treated secondary AML (ts-AML). The investigator-sponsored trial is being led by Tapan...
Paris, France – Testing for tumour DNA in the blood can successfully identify advanced bladder cancer patients who will not relapse following surgery, new research shows. This could allow doctors to target treatments more effectively to those who need it, and spare those patients for whom further treatment is unnecessary,...
SEATTLE and ORLANDO, Fla. — Trubion Pharmaceuticals, Inc. (Nasdaq: TRBN) announced today the presentation of encouraging Phase 1 safety and efficacy results following administration of low doses of TRU-016 in heavily pre-treated patients with high-risk genomic factors and relapsed or refractory chronic lymphocytic leukemia (CLL). TRU-016 is the Company’s proprietary...