Intellia Therapeutics (NTLA) investor relations material

Recent progress and achievements

• Completed the first phase III program for an in vivo CRISPR product, demonstrating pioneering work in genome editing and lipid nanoparticle delivery.

• Phase III data for hereditary angioedema (HAE) confirmed earlier results, with most patients essentially free of disease after outpatient IV therapy.

• Initiated rolling BLA submissions for HAE, aiming for completion in the second half of the year, leveraging RMAT designation for frequent FDA engagement and potential priority review.

• Rebooted phase III programs for transthyretin amyloidosis (TTR), targeting both polyneuropathy and cardiomyopathy indications.

• Ongoing research efforts continue to explore innovative approaches beyond standard methodologies.

Competitive landscape and value proposition

• HAE patients still experience attacks and burdens despite existing therapies, with over 80% reporting attacks in the past year.

• Current therapies are expensive and require ongoing authorizations, creating uncertainty and barriers for patients.

• One-and-done CRISPR therapy offers the potential to eliminate disease burden, reduce attacks, and remove the need for ongoing medication.

• Nearly all patients in trials discontinued long-term prophylaxis, with improved patient experience and reduced anxiety about drug access.

• Pricing strategy aims to avoid record-high prices, focusing on resource savings for payers, patients, and physicians.

Payer engagement and pricing considerations

• One-time therapy pricing expected to be higher than annual costs of current agents, typically 2x-5x reference products, justified by durable outcomes and high response rates.

• No patients have lost therapeutic effect after up to four years of follow-up, supporting long-term value.

• Payers generally prefer standard reimbursement models over value-based agreements due to administrative complexity.

• Efforts are made to ensure pricing does not create excessive resistance, with most patients already meeting step-edit requirements.