Investigational Therapy
Cour Pharmaceuticals snags orphan drug designation for CNP-104 for the treatment of PBC
The FDA has granted Cour Pharmaceuticals orphan drug designation for CNP-104, a biodegradable nanoparticle in development for the treatment of primary biliary cholangitis, the company announced in a press release.
Dalzanemdor fails to meet endpoints in phase 2 study of patients with Huntington’s disease
A phase 2 clinical trial of dalzanemdor in those with cognitive impairment associated with Huntington’s disease failed to meet its primary and secondary endpoints, causing the manufacturer to halt development of the investigational therapy.
IV nipocalimab reduces disease expression in generalized myasthenia gravis
Treatment with IV nipocalimab was safe and led to significant reductions in disease expression at 24 weeks in adolescents with generalized myasthenia gravis, according to data presented at AANEM.
FDA grants breakthrough therapy designation to nipocalimab for Sjögren’s disease
The FDA has granted breakthrough therapy designation to nipocalimab, a novel neonatal Fc receptor blocker, for the treatment of moderate to severe Sjögren’s disease, according to a press release from Johnson & Johnson.
Data monitoring committee OKs continuation of phase 3 trial for vidofludimus calcium
An independent data monitoring committee has ruled that a phase 3 clinical trial investigating vidofludimus calcium for relapsing MS may continue as originally planned, according to the therapeutic’s manufacturer.
Rocatinlimab exhibits positive results in phase 3 study for atopic dermatitis
Rocatinlimab exhibited promising results in the phase 3 ROCKET HORIZON trial for the treatment of moderate to severe atopic dermatitis, Kyowa Kirin announced in a press release.
Once-weekly batoclimab lowers immunoglobulin for adults with uncontrolled Graves’ disease
Most adults who had uncontrolled Graves’ disease on antithyroid drugs responded to once-weekly subcutaneous batoclimab, according to top-line results released by Immunovant.
Phase 3 results for pegcetacoplan show reduced proteinuria, stabilized eGFR vs. placebo
Sobi and Apellis Pharmaceuticals Inc. announced results from the phase 3 Valiant study showing the drug pegcetacoplan reduced proteinuria and stabilized eGFR compared to placebo in patients with glomerulopathy or glomerulonephritis.
Partnership to advance novel therapeutic, drug delivery platform for Parkinson’s disease
A health care innovation company and a clinical-stage biotech company have announced a partnership to advance a novel apomorphine therapeutic in combination with a wearable drug delivery platform to treat Parkinson’s disease.
Gene therapy to treat congestive heart failure gains FDA fast track designation
Bayer AG and its subsidiary Asklepios BioPharmaceutical announced the FDA granted fast track designation for its investigational gene therapy for the treatment of congestive heart failure.