Intellia Therapeutics(US:NTLA)2022-08-03 16:00Funding and Financials - Intellia Therapeutics has raised approximately $1,861.1 million since inception through various funding methods, including IPOs and collaboration agreements[126]. - Collaboration revenue increased by approximately $7.5 million to $14.0 million for the three months ended June 30, 2022, compared to $6.6 million for the same period in 2021[176]. - Collaboration revenue for the six months ended June 30, 2022, increased by approximately $12.3 million to $25.3 million, compared to $13.0 million for the same period in 2021[181]. - The company reported a net cash provided by financing activities of $53.2 million during the six months ended June 30, 2022[204]. - Cash, cash equivalents, and marketable securities totaled $906.9 million as of June 30, 2022[189]. - Net cash used in operating activities was $164.9 million for the six months ended June 30, 2022, compared to $105.4 million in the same period of 2021[202]. - The company expects to fund ongoing operating expenses and capital requirements beyond the next 24 months with existing cash and collaboration funding[199]. - The company has significant milestone payments and royalties potential under collaborations with Novartis, SparingVision, and others, dependent on research outcomes[190]. - The company made a one-time payment of $10.0 million to Novartis as part of the amendment to the 2014 Novartis Agreement, recorded as research and development expense[169]. Research and Development - Research and development expenses rose by approximately $31.3 million to $90.2 million for the three months ended June 30, 2022, compared to $58.9 million for the same period in 2021, representing a 53% increase[177]. - Research and development expenses for the six months ended June 30, 2022, increased by $125.1 million to $223.3 million, compared to $98.2 million for the same period in 2021[182]. - The increase in research and development expenses was primarily driven by a $13.2 million increase in employee-related expenses due to workforce expansion[183]. - Research and development expenses increased by 127% to $223.3 million for the six months ended June 30, 2022, compared to $98.2 million in the same period of 2021[184]. - Employee-related expenses rose by 83% to $53.5 million, driven by the expansion of the development organization[186]. - The company incurred $56.0 million in expenses related to the acquisition of Rewrite Therapeutics, Inc.[186]. - The company anticipates an increase in expenses in the second half of 2022 due to ongoing research and development activities[197]. Clinical Development and Programs - The lead in vivo candidate, NTLA-2001, is being evaluated in a Phase 1 study, with over 30 patients treated, showing rapid and deep reductions in serum TTR protein[129]. - NTLA-2001 demonstrated an 86% to 98% maximum TTR reduction at day 28 in the polyneuropathy arm of the study[131]. - The ongoing Phase 1 study of NTLA-2001 has shown sustained TTR reductions for up to 12 months in certain cohorts[134]. - The company plans to file an IND or IND-equivalent for NTLA-3001, targeting AATD-associated lung disease, in 2023[137]. - NTLA-2002, for hereditary angioedema, is progressing through its first-in-human study, with interim data expected in the second half of 2022[136]. - The ATTR program is co-developed with Regeneron, which shares approximately 25% of worldwide development costs and commercial profits[135]. - NTLA-2003 is a wholly-owned in vivo knockout candidate aimed at treating AATD-associated liver disease, designed to inactivate the SERPINA1 gene to halt liver disease progression and potentially eliminate the need for liver transplants[138]. - In Q3 2021, the company and Regeneron nominated a Factor 9 gene insertion candidate for the Hemophilia B program, achieving circulating FIX levels at or above normal human plasma levels in preclinical studies[140]. - The company plans to advance at least one new in vivo development candidate by the end of 2022, continuing its focus on liver-targeted therapies[144]. - The company is pivoting its NTLA-5001 program to an allogeneic version, based on the potential for a high-quality, readily available cell product for aggressive cancers[149]. - NTLA-6001, an allogeneic CAR-T candidate targeting CD30, is in IND-enabling activities, representing the first candidate developed using the company's allogeneic cell engineering platform[150]. - The collaboration with Novartis has led to the initiation of clinical studies for two therapeutic candidates targeting sickle cell diseases, with Novartis currently recruiting patients for its Phase 1/2 study[155]. Technology and Innovation - The company is focused on developing curative CRISPR/Cas9-based medicines to address severe diseases with high unmet needs[125]. - The company is advancing its modular platform for genome editing, enhancing capabilities for both in vivo and ex vivo therapies[124]. - Intellia's strategy includes leveraging proprietary CRISPR/Cas9 technology to create engineered cell therapies for immuno-oncology and autoimmune diseases[126]. - The proprietary T cell engineering process using LNPs has shown improved cell properties and performance compared to traditional methods, supporting both autologous and allogeneic T cell candidates[153]. - The company demonstrated >90% T cell editing efficiency using its novel cytosine deaminase base editor technology, with no detectable increase in translocation above background levels[154]. Collaborations and Partnerships - The company has formed strategic alliances to enhance CRISPR/Cas9 therapeutic development, including a collaboration with SparingVision to develop genomic medicines for ocular diseases[161]. - The collaboration with AvenCell aims to develop allogeneic universal CAR-T cell therapies, with the company receiving a 33.33% equity interest in AvenCell[159][160]. - The company is eligible to receive up to $184 million per product in development and commercial milestone payments from ONK for engineered NK cell therapies[167]. - Kyverna will lead and fund preclinical and clinical development for KYV-201, with the company eligible for certain milestone payments and royalties on future sales[165]. Economic Factors - Inflation has generally impacted the company by increasing labor and program costs[210]. - The company does not believe inflation had a material effect on its results of operations during the six months ended June 30, 2022[210]. - The company does not utilize any foreign currency or derivative financial instruments[210].