Intellia Therapeutics(NTLA) - 2019 Q4 - Earnings Call Transcript

Financial Data and Key Metrics Changes - As of December 31, 2019, the company's cash, cash equivalents, and marketable securities were $284.5 million, a decrease from $314.1 million as of December 31, 2018, primarily due to approximately $125 million used for operations [30] - Collaboration revenue for Q4 2019 was $10.9 million, up from $7.9 million in the same period in 2018, driven mainly by the ATTR program collaboration with Regeneron [31][32] - R&D expenses increased to $31.7 million in Q4 2019 from $19.9 million in Q4 2018, attributed to IND-enabling activities for NTLA-2001 and research efforts for NTLA-5001 [33] Business Line Data and Key Metrics Changes - The company advanced its first development candidate, NTLA-2001, for transthyretin amyloidosis (ATTR) and is on track for an IND submission in mid-2020 [10][11] - NTLA-5001, targeting acute myeloid leukemia (AML), is also progressing with IND-enabling activities expected to lead to an IND submission in the first half of 2021 [22][26] - The HAE program aims to utilize a knockout approach for hereditary angioedema, with a development candidate nomination expected in the first half of 2020 [16][20] Market Data and Key Metrics Changes - The ATTR program targets a progressive and fatal disease with significant unmet medical need, as it is often underdiagnosed [13] - The HAE program is designed to address a rare genetic disease affecting approximately 1 in 50,000 people, with existing therapies having significant treatment burdens [17][18] Company Strategy and Development Direction - The company is focused on advancing a full spectrum genome editing strategy, with both in vivo and ex vivo programs aimed at addressing life-threatening diseases [7][8] - The modular approach allows for rapid development and adaptation of therapies based on prior experiences, enhancing the potential for successful outcomes [11][20] - The company aims to leverage its CRISPR technology to create engineered cell therapies for cancer and genetic diseases, with a strong emphasis on safety and efficacy [21][26] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the potential of CRISPR-based therapies to provide curative options for patients, particularly in the context of ATTR and HAE [11][15] - The company anticipates a significant year ahead with multiple milestones, including the initiation of clinical trials for NTLA-2001 and NTLA-5001 [36][37] - Management highlighted the importance of collaboration with key opinion leaders and patient groups to inform clinical development and ensure alignment with patient needs [45][46] Other Important Information - The company reaffirmed that its current cash balance is expected to fund operations through at least the end of 2021 [34] - The collaboration with Regeneron involves shared development costs and future commercial profits for the ATTR program, enhancing financial stability [32] Q&A Session Summary Question: Can you elaborate on consecutive editing and potential safety concerns? - The company explained that consecutive editing involves multiple LNP administrations targeting different genetic targets, with safety assessments similar to single administrations [39][42] Question: What feedback are you receiving regarding NTLA-2001 and its potential as a one-time treatment? - Management noted broad enthusiasm from key opinion leaders and patients for a one-time curative approach compared to chronic therapies [45][46] Question: What are your thoughts on the target TTR knockdown levels for the ATTR program? - The company aims for knockdowns in excess of 60% to achieve therapeutic value, with ongoing assessments to refine this target [55][56] Question: Can you provide an update on the alpha-1 antitrypsin program? - The company is actively working on optimizing the alpha-1 antitrypsin program and plans to share updates at future scientific meetings [60] Question: How will you approach the clinical development of the HAE program? - Management indicated that the clinical program will be designed to meet and improve upon existing standards of care, with a focus on patient safety [71][72]