Swiss pharmaceutical giant Novartis has acquired DTx Pharma for $500 million upfront, with the potential for an additional $500 million in milestones. DTx Pharma has developed an RNA therapy that targets the liver to treat high cholesterol and has a lead drug candidate ready for Phase 1 testing in Charcot-Marie-Tooth (CMT) disease, a rare neuromuscular disorder. Novartis is interested in DTx Pharma’s technology platform, which uses small interfering RNA (siRNA) to stop disease-causing proteins from being produced. The acquisition gives Novartis the opportunity to expand its RNA research to more diseases throughout the body.
The first gene-silencing therapies using siRNA were designed to target diseases in the liver. Novartis’ Leqvio and Alnylam Pharmaceutical’s Onpattro are two examples of such therapies. However, RNA drug developers are now looking to expand the use of siRNA therapies beyond the liver. DTx Pharma uses combinations of fatty acids to improve the uptake of siRNA by different cell types. In preclinical research, DTx Pharma’s therapy for CMT1A, which targets Schwann cells in the peripheral nervous system, was able to successfully knock down disease-causing proteins and improve mobility in mice.
As DTx Pharma prepared to advance into Phase 1 testing, Novartis became interested in the startup’s lead program approach for addressing the root cause of CMT. Novartis also sees potential applications for DTx Pharma’s technology platform in neurodegenerative diseases and central nervous system disorders. While Novartis is focused on muscle disorders, other companies like Sarepta Therapeutics, Avidity Biosciences, and Dyne Therapeutics are also pursuing genetic medicines for similar conditions.
In addition to its work in muscle-targeting siRNAs, DTx Pharma is also conducting discovery-stage research on undisclosed central nervous system targets and other cell types like skin and cardiac tissue. The company’s first clinical test will be in CMT, which Novartis considers an important area due to the significant disability caused by the disease. Robert Baloh, global head of neuroscience for the Novartis Institutes for BioMedical Research, has personal experience with CMT from his time as a professor of neurology and believes that bringing the program to the clinic would be a significant achievement.
The acquisition of DTx Pharma by Novartis demonstrates the continued interest and investment in RNA therapies and the potential to expand their use in treating various diseases throughout the body. Novartis sees the value in DTx Pharma’s technology platform and believes it can be applied beyond the liver to address other diseases and cell types. The development of siRNA therapies holds promise for the future of genetic medicine and could revolutionize the treatment of a wide range of conditions.
July 19, 2023
Artificial Intelligence
FDAcop
