Intellia Therapeutics Inc. (NASDAQ: NTLA) has announced that the UK Medicines and Healthcare products Regulatory Agency (MHRA) has given approval for amendment of protocol for its current Phase 1 NTLA-2001 study to include ATTR amyloidosis with cardiomyopathy (ATT-CM).
ATTR-CM patients added to original Phase I group
Patients with ATTR-CM will now be included in additional dose expansion and escalation cohorts in the NTLA-2001 investigation. In addition, the ATTR-CM patients have been added to the original Phase 1 research cohort, which is now testing NTLA-2001 in patients with ATTR amyloidosis and polyneuropathy.
NTLA-2001 is an in vivo CRISPR/Cas9-based genome editing product being investigated as single-dose transthyretin (ATTR) amyloidosis treatment. It is the first investigational therapeutic of its sort. It is being developed to be the first single-dose treatment for potentially ATTR amyloidosis and reverse disease development in both ATTR-CM and ATTRv-PN patients by inactivating the TTR gene in liver cells block the generation of transthyretin (TTR) protein. Both the European Commission and the US have designated NTLA-2001 as an orphan medication for the treatment of ATTR amyloidosis. FDA
NTLA-2001 can reduce serum TTR levels
Intellia CEO and President John Leonard said, “ATTR amyloidosis is a chronic, fatal disease that can impact different organs and tissues within the body, often manifesting as either polyneuropathy or cardiomyopathy. At Intellia, our goal is to develop a potentially curative treatment that could benefit as many patients living with this disease as possible. We have already seen promising interim data supporting the ability of NTLA-2001 to significantly reduce serum TTR levels in ATTR patients with polyneuropathy. We are excited to now expand our Phase 1 study of NTLA-2001 to include ATTR patients with cardiomyopathy in order to advance this potentially first-of-its-kind, single-dose treatment for more patients.”
The Phase 1 protocol update allows up to 36 persons in the United Kingdom with inherited ATTR-CM or wild-type cardiomyopathy and New York Heart Association Class I–III heart failure to participate in the two-part research. The key goals of the study are to evaluate NTLA-2001’s safety, tolerability, pharmacokinetics, and pharmacodynamics.