Intellia Therapeutics(NTLA) - 2021 Q2 - Earnings Call Transcript

Financial Data and Key Metrics Changes - As of June 30, 2021, the company's cash, cash equivalents, and marketable securities totaled $551.3 million, a decrease from $597.4 million as of December 31, 2020, primarily due to cash used for operations of approximately $115.1 million [29] - Collaboration revenue decreased by $9.7 million to $6.6 million in Q2 2021 compared to $16.3 million in Q2 2020, driven by a one-time catch-up adjustment related to a previous collaboration agreement [31] - R&D expenses increased by $21.1 million to $58.9 million in Q2 2021 compared to $37.8 million in Q2 2020, influenced by a one-time payment related to a Novartis agreement and employee-related expenses [31] Business Line Data and Key Metrics Changes - NTLA-2001 showed a mean serum TTR reduction of 87% at day 28 in the second dose level, with a maximum reduction of 96%, indicating a strong therapeutic potential for ATTR amyloidosis [9][17] - NTLA-2002 and NTLA-5001 are progressing towards clinical trials, with CTAs submitted for both candidates, aiming to begin first-in-human trials by the end of the year [10][26] Market Data and Key Metrics Changes - The company is focusing on expanding its presence in the genetic disease treatment market, leveraging its CRISPR-based therapies to address unmet medical needs [7][11] - The collaboration with Cellex and Blackstone Life Sciences aims to enhance the development of differentiated immuno-oncology and autoimmune therapies, indicating a strategic move to broaden market reach [10][11] Company Strategy and Development Direction - The company aims to build a comprehensive toolbox for CRISPR-based medicines, focusing on both in vivo and ex vivo therapeutic applications [8] - Collaborations are seen as a key strategy to extend the reach of the company's technology while retaining rights across diverse areas [11] - The company plans to nominate at least one new development candidate by the end of the year and is working towards its first allogeneic development candidate by the first half of 2022 [28][35] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the potential of NTLA-2001 as a one-time treatment for ATTR amyloidosis, viewing the interim clinical data as a significant milestone [9][34] - The company is confident in its ability to engage with regulators regarding next steps for clinical trials without needing additional data [41][42] - Management highlighted the importance of achieving deeper TTR knockdown for improved clinical outcomes in both cardiomyopathy and polyneuropathy [73] Other Important Information - The company completed a successful follow-on offering in July, raising $648.1 million in net proceeds, strengthening its financial position to advance its pipeline [30] - The company is committed to sharing additional interim results and updates on its clinical programs at upcoming scientific and medical conferences [36] Q&A Session Summary Question: Plans for engaging with FDA regarding next step trials - Management indicated that they do not believe additional data is necessary to engage with regulators and are planning to do so soon [41][42] Question: Expectations for upcoming data from Cohorts 3 and 4 - Management stated that they will report additional data from the dose escalation portion of the trial before the end of the year [49] Question: Thoughts on re-dosing with NTLA-2001 - Management expressed confidence that one dose would be sufficient for meaningful clinical benefit, but noted that re-dosing could be an option if necessary [91] Question: Differences in T-cells from AML patients compared to B cell lymphoma patients - Management indicated that there are no expected issues with T-cell manufacturing from AML patients [95] Question: Regulatory path for 2001 in cardiomyopathy and polyneuropathy - Management stated that it is premature to conclude whether comparative studies will be required for regulatory approval [66]