Drugs in the Pipeline
ACX-362E is a targeted, narrow spectrum oral antibiotic that blocks the DNA replication process through inhibition of polymerase III, which has been shown to be bactericidal.
The NDA includes safety and efficacy data from two Phase 3 trials (REVIVE-1 and REVIVE-2; N=1190) which compared intravenous iclaprim to standard-of-care vancomycin in patients with ABSSSI.
SGT-001 is a novel adeno-associated viral (AAV) vector mediated gene transfer that could potentially address the underlying genetic cause of DMD, mutations in the dystrophin gene.
Mirvetuximuab soravtansine uses a humanized FRα-binding antibody to target antibody-drug conjugates specifically to FRα-expressing cancer cells and a potent anti-tumor agent, DM4, to kill the targeted cancer cells.
Fremanezumab, an anti-CGRP monoclonal antibody, is currently being reviewed by the Food and Drug Administration (FDA) as a quarterly or monthly injection for the preventive treatment of migraine in adults.
Current treatments for MPS 1 include bone marrow transplant and enzyme replacement with a recombinant form of human IDUA administered intravenously.
The pharmacokinetics trial, which included 60 participants with seasonal allergies, also demonstrated rapid drug absorption with the intranasal spray.
The film is a new formulation of the dopamine agonist apomorphine, intended for rapid conversion from the OFF to the ON state; it has been studied to treat motor OFF episodes up to 5 times a day.
The first patient has been enrolled in PEDFIC-1, a phase 3, single, randomized, double-blind, placebo-controlled trial designed to evaluate A4250 in 60 patients (aged 6 months-18 years) with PFIC subtype 1 or 2 who have elevated serum bile acid (sBA) levels and pruritus.
The trial included 594 individuals with open-angle glaucoma or ocular hypertension. Participants were randomized to receive 3 administration cycles (Day 1, Week 16 and Week 32) of Bimatoprost SR or twice daily timolol eye drops.
The independent data monitoring committee (IDMC) concluded that the 2 trials (AMARANTH and DAYBREAK-ALZ) investigating the treatment in mild Alzheimer's disease dementia, were not likely to meet their primary endpoints of change from baseline on the 13-item Alzheimer's Disease Assessment Scale - Cognitive Subscale.
GPLSCD01 is an investigational treatment made up of ex vivo expanded autologous human corneal epithelial cells containing stem cells.
Olinciguat is a soluble guanylate cyclase stimulator currently in Phase 2 development.
TransCon PTH is a long-acting prodrug of parathyroid hormone designed to restore PTH to physiologic levels, normalizing blood and urinary calcium levels, serum phosphate levels and bone turnover.
Brincidofovir is the Company's lead candidate, a nucleotide analog that has antiviral activity against variola virus, which causes smallpox.
Renal and Urology News Articles
NEPHROLOGY & UROLOGY NEWS
- Acute Kidney Injury (AKI)
- Chronic Kidney Disease (CKD)
- Contrast Nephropathy
- Cardiovascular Disease (CVD)
- Diabetic Nephropathy
- End-stage Renal Disease (ESRD)
- Lupus Nephritis
- Peritoneal Dialysis
- Secondary Hyperparathyroidism (SHPT)