San Diego startup DTx Pharma, which is developing technology for delivering RNA-based therapies to hard-to-treat diseases, has been acquired by drug giant Novartis for $500 million.
Founded in 2017, DTx Pharma has a chance to earn an additional $500 million if certain milestones are met, according to the companies.
The acquisition provides Novartis with DTx Pharma’s drug delivery platform called FALCON, an acronym for Fatty Acid Ligand Conjugated OligoNucleotide.
FALCON aims to overcome some of the barriers in delivering RNA-based treatments for difficult central nervous system, heart, muscular and brain diseases. For years, RNA-interference therapies have tended to be cleared by the kidneys and liver too quickly to be effective.
The first disease being targeted using FALCON is Charcot-Marie-Tooth Disease Type 1A — a genetic disorder that can result in progressive loss of muscle function.
About 150,000 people suffer from the condition in the U.S. and Europe. For now, there are no approved therapies for treating the underlying cause of the disease.
DTx Pharma’s treatment is in the early, preclinical stages. But initial research involving mice showed positive results, said co-founder and Chief Executive Arthur Suckow.
“The way FALCON technology or RNA interference technology works is that it operates at the gene level,” he said. “It essentially chops up the excess production of the gene. At least in rodent models, when we apply the technology, the mice go from not being able to balance, not being able to coordinate to all of a sudden walking and running.”
If DTx’s therapy for Charcot-Marie-Tooth Disease works for humans, it could unlock additional FALCON-based treatments for difficult diseases involving the nervous system, muscles and brain, said Suckow.
Novartis has not given a time frame for considering human clinical trials for the treatment of Charcot-Marie-Tooth Disease Type 1A. But Suckow said trials could start as early as 2024.
“This is a great outcome for the patients,” said Peter Condon, DTx Pharma’s Chief Business Officer. “The clinical infrastructure that Novartis brings will help accelerate this program.”
In addition to the treatment targeting Charcot-Marie-Tooth Disease, Novartis has acquired full rights to the FALCON platform and two other early-stage therapy programs.
“This acquisition underscores the Novartis commitment to bringing life-changing medicines forward for patients with neuromuscular diseases and other disorders of the nervous system,” said Robert Baloh, head of Neuroscience for the Novartis Institutes for BioMedical Research. “
The roughly 50-employee company previously raised $115 million in venture capital. Suckow, who started the company with a small personal investment, credited San Diego’s startup ecosystem, including the Connect Springboard program, with helping the fledgling company get off the ground.
This content was originally published here.
