Intellia plots pivotal trials for lead programs as it lays out key objectives
Intellia has some big plans for the next two years, including pivotal trials for its first two in vivo CRISPR therapies.
The Cambridge, MA-based genome editing company on Thursday disclosed its game plan for 2023 and 2024, starting with INDs for the inclusion of US sites in studies for NTLA-2001 and NTLA-2002 in transthyretin (ATTR) amyloidosis with cardiomyopathy and hereditary angioedema (HAE), respectively.
ATTR amyloidosis with cardiomyopathy is a disease that affects the heart and tendons and is caused by the accumulation of a wild-type protein called transthyretin. Intellia added that the goal is to kick off the pivotal study by the end of this year, though those plans are still subject to regulatory feedback.
Another goal is to gear up for a Phase III study in ATTR amyloidosis with polyneuropathy, or ATTRv-PN.
If things go according to plan, a Phase II trial will start in the first half of this year for NTLA-2002 in HAE. An ongoing trial is being run in New Zealand, the Netherlands, and the UK.
Intellia offered a first look at its HAE treatment back in September, which directly edits the gene underlying the protein responsible for the swelling attacks in patients. At the time, the company reported that two out of three patients were attack-free, and a third was attack-free after 10 weeks. A few months later, CEO John Leonard claimed to Endpoints News that the treatment “may be a functional cure.”
And while it costs to run pivotal trials, Intellia has a substantial amount of cash on hand, ending 2022 with almost $1.3 billion in cash and marketable securities.