Release Date: November 07, 2024
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Positive Points
- Intellia Therapeutics Inc (NTLA, Financial) reported unprecedented positive results from their phase two study of NTLA-2002 for hereditary angioedema, suggesting a potential functional cure.
- The FDA cleared the IND application for MAGNITUDE-2, a phase three trial for hereditary ATTR amyloidosis with polyneuropathy, indicating regulatory confidence in their pipeline.
- Intellia is leading the field with three active phase three studies expected by year-end, showcasing their strong position in in vivo CRISPR-based medicines.
- The company maintains a solid balance sheet with approximately $944.7 million in cash, cash equivalents, and marketable securities, providing financial stability to fund operations until late 2026.
- Intellia's NTLA-3001 program for alpha-1 antitrypsin deficiency is on track to dose the first patient by year-end, marking progress in their in vivo gene insertion programs.
Negative Points
- Collaboration revenue decreased by $2.9 million compared to the previous year, primarily due to a reduction in revenue related to the Advents License and Collaboration Agreement.
- R&D expenses increased by $9.7 million year-over-year, driven by the advancement of lead programs, indicating rising operational costs.
- The company experienced a decrease in cash from $1 billion at the end of 2023 to $944.7 million as of September 2024, reflecting significant cash usage.
- The phase three study for NTLA-2002 in hereditary angioedema is expected to have a primary completion date in December 2027, indicating a long timeline before potential market entry.
- The small study size of 50 patients for the phase three trial in ATTR polyneuropathy raises questions about the robustness of the data compared to larger silencer studies.
Q & A Highlights
Q: Could you provide more details on the pace of enrollment for the Haelo study, given the primary endpoint is event-driven?
A: John Leonard, CEO: The Haelo study has 765 patients with global sites activated, and enrollment is ahead of projections. We may update the timeline as we gain more insights, but things are progressing well.
Q: How are you prioritizing capital allocation given your pipeline and the focus on in vivo versus ex vivo programs?
A: Edward Dulac, CFO: We have $945 million in cash and are focused on three phase three studies, NTLA-3001, and platform investments. Our cash burn is around $100 million per quarter, and we have a conservative plan to fund operations until late 2026.
Q: Can you explain the design of the phase three study for ATTR polyneuropathy, particularly the use of a placebo-controlled study and the small study size?
A: John Leonard, CEO: The placebo-controlled design was agreed upon with the FDA and allows for a solid readout on the drug's performance. The study is conducted outside the US where other therapies are not available, facilitating prompt enrollment.
Q: Why is the Haelo study using a 2 to 1 randomization scheme, and how does it impact enrollment?
A: Dr. David Lebwohl, CMO: The 2 to 1 randomization is attractive to patients, offering a higher chance of receiving the active drug. This design, along with the option for crossover, is expected to accelerate enrollment.
Q: What are the major contributors to the ahead-of-schedule enrollment in the MAGNITUDE-2 ATTR-CM study?
A: John Leonard, CEO: Investigators are excited about the extent of TTR reduction and the safety profile. The deep, consistent, and enduring reductions achieved with the drug have generated enthusiasm among treating physicians globally.
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
