Intellia Therapeutics(US:NTLA)2021-05-06 18:55Financial Data and Key Metrics Changes - As of March 31, 2021, the company's cash, cash equivalents, and marketable securities were $600.8 million, an increase from $597.4 million as of December 31, 2020, primarily driven by proceeds from various agreements and collaborations [49]. - Collaboration revenue decreased by $6.5 million to $6.4 million in Q1 2021 compared to $12.9 million in Q1 2020, attributed to a milestone payment from Novartis in the previous year [51]. - R&D expenses increased by $4.6 million to $39.3 million in Q1 2021 compared to $34.7 million in Q1 2020, driven by advancements in lead programs and expansion of the development organization [52]. - G&A expenses rose by $2.3 million to $13.6 million in Q1 2021 compared to $11.3 million in Q1 2020, related to employee-related expenses [53]. Business Line Data and Key Metrics Changes - The company received the European Union's orphan drug designation for NTLA-2001, indicating regulatory recognition of its potential benefits for ATTR patients [13][22]. - NTLA-2001 demonstrated over 95% TTR reduction in nonhuman primates after a single dose, suggesting potential for halting and reversing the disease [21]. - NTLA-5001, an engineered TCR T cell therapy for AML, is on track for a first-in-human regulatory submission mid-year [39]. Market Data and Key Metrics Changes - The global prevalence of hereditary ATTR is estimated at 50,000, with wild type ATTR affecting between 200,000 and 500,000 individuals [19]. - The treatment burden for HAE patients remains significant, with current therapies being costly and requiring ongoing management [31][113]. Company Strategy and Development Direction - The company aims to develop curative genome editing treatments, leveraging its CRISPR/Cas9 platform for both in vivo and ex vivo applications [10][11]. - Upcoming milestones include reporting interim clinical data for NTLA-2001 and submitting regulatory filings for NTLA-5001 and NTLA-2002 [18][57]. - The company is focused on expanding its pipeline and platform capabilities to deliver innovative genomic medicines [58]. Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the progress of NTLA-2001 and the potential for significant clinical validation in the near future [56]. - The company remains committed to addressing unmet medical needs and believes its innovative therapies can provide substantial benefits over existing treatments [22][113]. Other Important Information - The company welcomed Dr. Georgia Keresty to its board, bringing over 35 years of pharmaceutical industry experience [15]. - The company plans to share preclinical data at the ASGCT annual meeting, including updates on its research in alpha-1 antitrypsin deficiency [46]. Q&A Session Summary Question: What data should be expected from NTLA-2001 in mid-2021? - Management indicated that substantive data will be shared, but specifics on the number of cohorts were not disclosed [60][62]. Question: What is the status of the IND for NTLA-2001 in the US? - The first-in-human study is being conducted outside the US, with plans for a US IND for future studies involving cardiomyopathy [61]. Question: Any updates on safety in the clinical study? - Management stated that safety data will be shared in the mid-year update, emphasizing the study's progress [64]. Question: Will LNP design characteristics be discussed in the mid-year update? - Management suggested that while the focus will be on clinical data, there may be opportunities to discuss platform advantages later in the year [66]. Question: What is the target knockdown level for NTLA-2001? - The target is set at 80%, with the goal to surpass it for better clinical outcomes [68][69]. Question: Will there be redosing for patients not reaching target knockdown levels? - Management indicated that provisions for suboptimal doses may be addressed in the study [102]. Question: How does the company view competition in the sickle cell disease space? - The company aims to provide a solution that avoids the need for bone marrow transplants, focusing on in vivo delivery systems [86][87].