Intellia Eyes Lonvo-z Launch as HAE PDUFA Nears, TTR Study Advances

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 Leonard said the FDA has accepted Intellia’s biologics license application for lonvo-z in hereditary angioedema, or HAE, and granted priority review. The company’s PDUFA date is March 10. The FDA is not currently planning to hold an advisory committee meeting, according to Leonard. “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%.