Lidiya Rizova

Lidiya Rizova inquired about the commercial strategy for lonvo-z in HAE, specifically targeting the youngest or sickest patients, and sought an update on the timing for the PTR program's clinical hold resolution and the understanding of the underlying event that led to a patient's death.

Answer

President and CEO John Leonard addressed the PTR program, stating the polyneuropathy study is off clinical hold and accruing, while the MAGNITUDE (cardiomyopathy) study remains under FDA review due to its larger size and different patient population. He clarified the patient death was from a ruptured duodenal ulcer, possibly unrelated to treatment, and the LFT elevations were an outlier. Edward Dulac discussed the HAE commercial strategy, emphasizing lonvo-z's simple administration and durable effects, appealing to patients and physicians. He noted plans to scale field teams, engage payers, and finalize distribution and pricing, anticipating a strong value proposition.

Lidiya Rizova inquired about Intellia's commercial strategy for lonvo-z in HAE, focusing on the types of patients most amenable to gene therapy, and sought an update on the timing for resolving the MAGNITUDE clinical hold and any evolution in understanding the underlying event that led to a patient death.

Answer

President and CEO John Leonard addressed the TTR program, stating the PN study is off clinical hold and expected to be fully accrued by year-end, while MAGNITUDE (CM study) is larger with ongoing FDA engagement and substantial progress. He clarified the patient death was from a ruptured duodenal ulcer, potentially unrelated to treatment, and described the patient as an outlier. EVP and CFO Edward Dulac discussed the HAE commercial strategy, emphasizing the focus on treatment effect over modality, the unique profile of lonvo-z offering freedom from attacks and drug therapy, and ongoing efforts in physician education, payer engagement, and scaling commercial teams.