Rare disease drug development companies battle with economic challenges and small patient populations, but new technology alongside a human connection are helping researchers and marketers identify patients, educate physicians and build networks. At Pharma USA, a Reuters Events meeting in Philadelphia March 16-17, executives from Astrazeneca plc and UCB SA discussed their successes with rare disease launches, presenting their playbooks for building trust and driving adoption among patients.
Belgian pharma giant UCB SA is putting skin in the bispecific T-cell engager (TCE) game, announcing a potential $1.1 billion deal to license Antengene Corp.’s ATG-201. ATG-201 is a CD19/CD3 bispecific TCE antibody aimed at autoimmune disorders, though specific indications were not disclosed.
UCB SA and Antengene Corp. Ltd. have entered into a license agreement that grants UCB a worldwide exclusive license to further develop, manufacture and commercialize ATG-201.
The U.S. FDA approved 18 drugs in November, down from 20 in October, bringing the total number of clearances to 199 through the first 11 months of the year. The number is about 5% lower than the 209 approvals recorded over the same period in 2024 but more than every prior year.
The U.S. FDA has approved UCB SA’s Kygevvi (doxecitine and doxribtimine), the first treatment for the ultra-rare, genetic and life-threatening mitochondrial disease thymidine kinase 2 deficiency. The approval comes as the company reaps a reward of rebuilding and reorganizing that it began little more than a year ago.
UCB SA has divulged compounds acting as NAD(+) hydrolase SARM1 (SAMD2; MyD88-5) inhibitors reported to be useful for the treatment of inflammation, injury, neurodegeneration, and eye, autoimmune and neurological disorders, among others.
A new version of a drug candidate that failed in a phase II for neuropathic pain 15 years ago has attracted $140 million in series D funds, plus an ex-North American deal worth up to $570 million, for Grin Therapeutics Inc.
Researchers from UCB SA and affiliated organizations presented the discovery and preclinical characterization of novel NUAK family SnF1-like kinase-1 (NUAK1) inhibitors.
Voyager Therapeutics Inc.’s recent selection of a lead development candidate, VY-1706, for its tau silencing gene therapy program in Alzheimer’s disease brought renewed attention to the target, which continues to intrigue a substantial lineup of developers. Bellwether data rolled out this fall from UCB SA and Roche AG at the Clinical Trials in Alzheimer’s Disease meeting in Madrid.
