Intellia Therapeutics(US:NTLA)2021-08-04 16:00Financial Overview - Intellia Therapeutics has raised approximately $1,166.1 million since inception, with $275.9 million from collaboration agreements and $438.3 million from follow-on public offerings[85]. - As of June 30, 2021, the company had raised approximately $1,166.1 million to fund operations, with $551.3 million in cash, cash equivalents, and marketable securities[144][145]. - The company closed a public offering in July 2021, raising estimated net proceeds of $648.1 million from the sale of 4,758,620 shares at $145.00 per share[145]. - Net cash used in operating activities was $105.4 million for the six months ended June 30, 2021, primarily due to increased research and development spending[157]. - Net cash provided by financing activities was $65.7 million during the same period, including $45.3 million from at-the-market offerings[159]. - The company expects to fund ongoing operating expenses and capital expenditures beyond the next twenty-four months with current cash and proceeds from the July 2021 offering[153]. - Interest income decreased by $1.5 million to $0.4 million for the six months ended June 30, 2021, compared to $1.9 million for the same period in 2020[143]. Research and Development - The lead in vivo candidate, NTLA-2001, has shown a mean reduction of 52% in serum TTR levels at a 0.1 mg/kg dose and 87% at a 0.3 mg/kg dose in a Phase 1 study[94]. - The ongoing Phase 1 trial for NTLA-2001 aims to enroll up to 38 participants and assess safety, tolerability, pharmacokinetics, and pharmacodynamics[93]. - The company plans to move to pivotal studies for both polyneuropathy and cardiomyopathy manifestations of ATTR amyloidosis after completing the Phase 1 trial[95]. - The company is developing NTLA-5001 for acute myeloid leukemia and NTLA-2002 for hereditary angioedema, with clinical trial applications pending[79]. - NTLA-2001 is the first CRISPR/Cas9-based therapy candidate administered systemically for precision editing of a gene in humans[86]. - The company has demonstrated over 95% reduction in serum TTR protein levels in non-human primate studies after a single systemic administration of LNPs[91]. - NTLA-2002 demonstrated greater reductions in serum kallikrein protein levels and activity compared to the current standard of care, sustained over 17 months following a single dose in an ongoing study[101]. - The company plans to enroll the first patient in the Phase 1 study for NTLA-2002 by year-end and is submitting additional regulatory applications for enrollment in other countries[101]. - NTLA-5001 targets the WT1 intracellular antigen, overexpressed in >90% of AML blasts, aiming to provide a broadly applicable treatment for AML[109]. - The company submitted its first CTA for NTLA-5001 to initiate a Phase 1 study, focusing on patients with persistent or recurrent AML[110]. Collaborations - Intellia's collaboration with Regeneron Pharmaceuticals involves shared development costs and profits, with Regeneron covering approximately 25% of worldwide costs for the ATTR amyloidosis program[96]. - Through June 30, 2021, the company recorded $145.0 million in upfront payments and $35.6 million for research and development services under the Regeneron collaboration[118]. - As of June 30, 2021, the company had deferred revenue of $62.8 million related to collaboration agreements[119]. - The collaboration with Novartis focuses on developing new ex vivo CRISPR/Cas9-edited therapies using CAR-T cells and HSCs[120]. - The company entered into a collaboration agreement with NewCo to develop allogeneic universal CAR-T cell therapies, with a commitment of up to $250 million from BXLS[124]. Financial Performance - Collaboration revenue decreased by $9.7 million to $6.6 million for the three months ended June 30, 2021, compared to $16.3 million for the same period in 2020[132]. - Research and development expenses increased by $21.1 million to $58.9 million for the three months ended June 30, 2021, compared to $37.8 million for the same period in 2020[133]. - Total operating expenses rose by $26.3 million to $75.6 million for the three months ended June 30, 2021, compared to $49.3 million for the same period in 2020[131]. - Net loss increased by $36.4 million to $68.8 million for the three months ended June 30, 2021, compared to $32.4 million for the same period in 2020[131]. - Collaboration revenue decreased by $16.2 million to $13.0 million for the six months ended June 30, 2021, compared to $29.2 million for the same period in 2020[139]. - Research and development expenses increased by $25.7 million to $98.2 million for the six months ended June 30, 2021, compared to $72.4 million for the same period in 2020[140]. - General and administrative expenses increased by $7.4 million to $30.3 million for the six months ended June 30, 2021, compared to $22.8 million for the same period in 2020[142]. - The company expects research and development expenses to continue to increase through 2021 as it grows its development team and progresses clinical trials[141]. - A one-time payment of $10.0 million was made to Novartis related to the amendment of the 2014 Novartis Agreement, impacting research and development expenses[134]. Strategic Goals - The company’s strategy focuses on leveraging a modular platform to advance in vivo and ex vivo therapies for diseases with high unmet need[85]. - The company aims to transform the lives of people with severe diseases by developing curative genome editing treatments using CRISPR/Cas9 technology[83]. - HAE is estimated to affect 1 in 50,000 people, with approximately 11,000 to 21,500 diagnosed patients in the U.S. and Europe[97]. - The company anticipates increased expenses in 2021 as it continues to grow its research and development team and advance additional programs into clinical development[151]. - The company has $47.4 million in shares of common stock remaining eligible for sale under the 2019 Sales Agreement[150]. - The company’s ability to generate revenue and achieve profitability depends on successful development and commercialization of product candidates, regulatory approvals, and market acceptance[154].