Drugs in the Pipeline

Novel Antibiotic for C. Difficile Infection Gets FDA's QIDP Designation

Novel Antibiotic for C. Difficile Infection Gets FDA's QIDP Designation

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.

NDA Submitted for Novel, Targeted Antibiotic Iclaprim

NDA Submitted for Novel, Targeted Antibiotic Iclaprim

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.

FDA Lifts Clinical Hold on Trial for Duchenne Muscular Dystrophy Treatment

FDA Lifts Clinical Hold on Trial for Duchenne Muscular Dystrophy Treatment

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.

Mirvetuximab Gets Fast Track Status for Platinum-Resistant Ovarian Cancer

Mirvetuximab Gets Fast Track Status for Platinum-Resistant Ovarian Cancer

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 Chronic Cluster Headache Study Discontinued

Fremanezumab Chronic Cluster Headache Study Discontinued

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.

Gene Therapy for Mucopolysaccharidosis Type I Gets Fast Track Designation

Gene Therapy for Mucopolysaccharidosis Type I Gets Fast Track Designation

Current treatments for MPS 1 include bone marrow transplant and enzyme replacement with a recombinant form of human IDUA administered intravenously.

Intranasal Epinephrine for Anaphylaxis Looks Promising in Early Study

Intranasal Epinephrine for Anaphylaxis Looks Promising in Early Study

The pharmacokinetics trial, which included 60 participants with seasonal allergies, also demonstrated rapid drug absorption with the intranasal spray.

FDA to Review Sublingual Tx for Motor Fluctuations in Parkinson's Disease

FDA to Review Sublingual Tx for Motor Fluctuations in Parkinson's Disease

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.

A4250 Granted Rare Pediatric Disease Designation for Progressive Cholestasis

A4250 Granted Rare Pediatric Disease Designation for Progressive Cholestasis

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.

Implant to Reduce Intraocular Pressure in Glaucoma Shows Promise

Implant to Reduce Intraocular Pressure in Glaucoma Shows Promise

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.

Trials for Investigational Alzheimer's Tx Lanabecestat Discontinued

Trials for Investigational Alzheimer's Tx Lanabecestat Discontinued

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.

Treatment for Limbal Stem Cell Deficiency Gets Orphan Drug Designation

Treatment for Limbal Stem Cell Deficiency Gets Orphan Drug Designation

GPLSCD01 is an investigational treatment made up of ex vivo expanded autologous human corneal epithelial cells containing stem cells.

Olinciguat Granted Orphan Drug Status for Sickle Cell Disease

Olinciguat Granted Orphan Drug Status for Sickle Cell Disease

Olinciguat is a soluble guanylate cyclase stimulator currently in Phase 2 development.

Hypoparathyroidism Treatment Granted Orphan Drug Designation

Hypoparathyroidism Treatment Granted Orphan Drug Designation

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.

FDA Grant Orphan Drug Status to Brincidofovir for Smallpox

FDA Grant Orphan Drug Status to Brincidofovir for Smallpox

Brincidofovir is the Company's lead candidate, a nucleotide analog that has antiviral activity against variola virus, which causes smallpox.