Intellia Therapeutics Q2 2024 Financial Results and Company Progress

Intellia Therapeutics, Inc. (NASDAQ:NTLA), a pioneering company in the field of gene editing, recently disclosed its operational highlights and financial outcomes for the second quarter concluding on June 30, 2024. The company's mission is to transform medicine through CRISPR-based therapies.

Dr. John Leonard, Intellia’s President and CEO, announced that the Phase 2 study of NTLA-2002 met its primary and all secondary endpoints for treating hereditary angioedema (HAE). The study demonstrated significant efficacy with a 50 mg dose, which will proceed to a pivotal Phase 3 trial set to commence in the latter half of 2024. Intellia aims to present these findings in detail at a medical meeting in the fourth quarter, reinforcing their commitment to what could potentially be a functional cure for HAE.

The Phase 2 trial results showed that NTLA-2002, designed to target the KLKB1 gene in the liver, achieved deep reductions in HAE attacks with both 25 mg and 50 mg doses. Notably, the 50 mg dose was chosen for the Phase 3 trial due to its higher efficacy in reducing attacks and kallikrein protein levels. Furthermore, ongoing Phase 1 data revealed that most patients remained attack-free for over 18 months post-treatment, with a remarkable 98% reduction in monthly HAE attack rates.

In the realm of transthyretin (ATTR) amyloidosis, NTLA-2001, now called nexiguran ziclumeran (nex-z), is set for pivotal Phase 3 trials. The MAGNITUDE trial, focusing on ATTR amyloidosis with cardiomyopathy (ATTR-CM), is enrolling swiftly and has regulatory approval in over 12 countries, actively recruiting at more than 35 sites globally. Additionally, a Phase 3 trial for hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN) is planned for initiation by year-end, targeting around 50 patients in a placebo-controlled setting. Updated data from ongoing Phase 1 studies are expected in the second half of 2024.

Intellia is also progressing with NTLA-3001, a CRISPR-mediated gene insertion candidate aimed at addressing alpha-1 antitrypsin deficiency (AATD)-associated lung disease. The United Kingdom’s MHRA has authorized the initiation of a first-in-human study, with the first patient expected to be dosed in the second half of 2024. This innovative therapy aims to restore normal levels of functional AAT protein through precise gene insertion.

The company is advancing its in vivo platform, presenting data that show the potential for additive effects through redosing with CRISPR, enhancing the pharmacodynamic impact. This promising capability, illustrated in the NTLA-2001 program, could be significant for future therapies requiring an additive response.

Furthermore, Intellia is expanding its CRISPR-based technologies to target diseases in multiple tissues beyond the liver, including bone marrow, brain, muscle, lung, and eye. This expansion could dramatically enhance the scope of CRISPR-based treatments.

Their ex vivo programs are making strides in immuno-oncology and autoimmune diseases, leveraging their allogeneic platform to develop cell therapies that evade T cell and NK cell-mediated rejection. This platform, which enhances cell function, is being used to address both hematological and solid tumors.

Intellia has also seen notable corporate developments, with the appointments of Brian Goff to the board of directors and Edward Dulac as Executive Vice President, Chief Financial Officer, and Treasurer.

Financially, the company ended the second quarter of 2024 with approximately $940 million in cash, a slight decrease from the $1.0 billion at the end of 2023, partly due to operational funding needs. Revenue from collaborations was $7.0 million, down from $13.6 million in the same period the previous year. Research and development expenses were slightly lower than the previous year, while general and administrative expenses saw a modest increase. The net loss for the quarter was $147.0 million, compared to $123.7 million in the second quarter of 2023.

Intellia will participate in several healthcare conferences in the third quarter of 2024, including Morgan Stanley's and Wells Fargo's annual healthcare conferences, as well as the Cantor Global Healthcare Conference.