Intellia Therapeutics (NASDAQ:NTLA) executives outlined plans for a potential launch of its hereditary angioedema treatment, lonvo-z, while discussing the company’s ongoing Phase III program in transthyretin amyloidosis and its capital position during a Morgan Stanley event.
Chief Executive Officer John Leonard said Intellia has focused on in vivo gene editing, using lipid nanoparticle delivery technology to target the liver. The company’s longer-term opportunity includes developing targeted delivery approaches that could reach tissues outside the liver, including the brain and lungs, he said. For now, Intellia is focused on advancing its first products toward commercialization.
HAE filing accepted, PDUFA date set
“We think we’ve got a very, very strong package,” Leonard said, citing what he described as strong efficacy and a clean safety profile.
Intellia developed the HAE program as an all-comers study, excluding pediatric patients younger than 16 but without restrictions based on prior therapy, therapy type or disease severity, Leonard said. He noted that the Phase III population included patients using long-term prophylaxis therapies, patients using on-demand therapy and patients with varying levels of disease control.
Chief Financial Officer Ed Dulac highlighted data showing an 87% attack-rate reduction and a 62% attack-free rate during the 28-week primary observation period. He also said that, across more than 100 patients treated in Phase I, Phase II and Phase III studies, every patient eventually became free of long-term prophylaxis therapy and none returned to it. The earliest Phase I/II patients have more than four years of follow-up, he said.
Commercial preparations underway
Intellia has not set a price for lonvo-z. Dulac said payer discussions have considered pricing at multiples of the annual cost of long-term prophylaxis therapy, which he said can exceed $700,000 annually. He said there is precedent for premium pricing for a highly effective, one-time treatment, though payers show more resistance at higher multiples.
The company is building a focused commercial organization ahead of a potential approval. Dulac said roughly 70% of HAE covered lives are commercially insured, with Medicare accounting for about 20% and Medicaid for 10%. Intellia has field medical personnel, authorized-treatment-center liaisons and promotional field representatives in place, with training still required before a launch.
- The top 250 U.S. HAE prescribers cover about half of U.S. patients, according to Dulac.
- Intellia expects an initial launch to be measured by treatment-center penetration and patient start forms before revenue fully develops.
- Dulac said the company does not expect an “overnight flip to revenue” because patients receiving a one-time therapy cannot transition from clinical supply to commercial supply in the same way as users of chronic treatments.
Management characterized HAE as a relatively small market centered on patient switches from existing therapies. Dulac said the company believes a successful launch could help cover company operating costs and support investment in its TTR programs and earlier research efforts.
TTR program could be refined as enrollment continues
Intellia is also advancing nex-z in the MAGNITUDE Phase III study in transthyretin amyloidosis with cardiomyopathy and polyneuropathy. Leonard said the endpoint-driven trial has continued accruing events, including during a prior clinical hold, and remains largely in line with the company’s initial assumptions.
The company may make limited changes to the trial design as it incorporates insights from recent data in the TTR field. Leonard said Intellia is evaluating disease stage and NT-proBNP levels, potentially including caps on the distribution of patients across NT-proBNP subgroups. The company also plans to encourage investigators to enroll patients earlier in their disease course, while the study continues to evaluate therapy on top of standard care, including stabilizers.
Leonard said Intellia has identified an association between prior liver findings and a particular HLA allele. HLA testing is now part of screening in the study, although it is not intended to automatically exclude patients. About 12% of patients in the trial population carried the allele, though prevalence varies substantially by ethnicity, he said.
For the TTR asset, Intellia retains decision-making control and a 75% economic interest in its collaboration with Regeneron. Dulac said Regeneron has an option to co-promote the asset in the U.S. in a minority capacity roughly two years before anticipated approval, while commercialization profits would be split 75% to Intellia and 25% to Regeneron.
Cash runway and debt facility
Dulac said Intellia had approximately $630 million in cash during the second quarter and projects it can fund operations into 2028 without including any lonvo-z revenue. The company also announced a $300 million committed debt facility with OrbiMed, initially drawing $75 million. Another $75 million may become available upon lonvo-z approval, while three additional $40 million tranches are linked to lonvo-z sales performance.
Management said the financing structure provides flexibility as it prepares for a possible HAE launch and continues investing in the TTR portfolio and undisclosed research programs.
About Intellia Therapeutics (NASDAQ:NTLA)
Intellia Therapeutics, Inc is a biotechnology company focused on developing curative treatments using CRISPR-based gene-editing technology. The company’s approach is designed to make targeted changes to disease-associated genes, including through in vivo therapies that edit genes directly inside the body.
Intellia’s pipeline includes NTLA-2001, an investigational treatment designed to address transthyretin amyloidosis by editing the TTR gene, and NTLA-2002, an investigational therapy targeting hereditary angioedema through editing of the KLKB1 gene.
