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8/4/2022
Good morning and welcome to the Intellia Therapeutics' second quarter 2022 financial results conference call. My name is Andrew and I will be your conference operator today. Following formal remarks, we will open the call up for a question and answer session. This conference is being recorded at the company's request and will be available on the company's website following the end of the call. As a reminder, all participants are currently in listen-only mode. If anyone requires operator assistance during the conference, please press star then zero on your telephone keypad. I will now turn the conference over to Ian Karp, Senior Vice President of Investor Relations and Corporate Communications at Intelia. Please proceed.
Thank you, operator, and good morning, everyone. Welcome to Intelia Therapeutics' second quarter 2022 earnings call. Earlier this morning, Intellia issued a press release outlining the company's progress this quarter, as well as topics for discussion on today's call. This release can be found on the Investors and Media section of Intellia's website at intelliatx.com. This call is being broadcast live, and a replay will be archived on the company's website. At this time, I would like to take a minute to remind listeners that during this call, Intellia management may make certain forward-looking statements and ask that you refer to our SEC filings available at sec.gov for discussion of potential risks and uncertainties. All information presented on this call is current as of today, and Intelia undertakes no duty to update this information unless required by law. Joining me from Intelia are Dr. John Leonard, Chief Executive Officer, Dr. David Leblall, Chief Medical Officer, Dr. Laura Sepp-Lorenzino, Chief Scientific Officer, and Glenn Goddard, Chief Financial Officer. John will begin with an overview of recent business highlights David will provide an update on our clinical programs with a focus on NPLA 2001 and 2002. Laura will then provide an update to our ex vivo strategy. Glenn will then review Intellia's financial results from the second quarter, and John will provide some final remarks before we open the call up for Q&A. And with that, I'll now turn the call over to John.
Thank you, Ian, and thank you all for joining us this morning. At Intellia, We're building the industry's leading genome editing company. We're deploying the broadest and deepest toolbox, including novel editing and delivery solutions, to harness the immense power of CRISPR-based technologies for in vivo and ex vivo therapeutic applications, each with the potential to revolutionize medicine. During the second quarter, and more recently, we've continued to advance our full-spectrum pipeline and platforms. Starting with NTLA-2001, we've made excellent progress in the ongoing Phase 1 study. In June, we presented additional clinical evidence that NTLA-2001 led to deep and durable TTR reductions following a one-time administration across all therapeutically relevant doses. David will provide a recap of the data presented at EASL and elaborate on the latest findings from the study. In addition, dosing is now completed in the dose escalation portion of the cardiomyopathy arm. With the more expansive data set, we're finalizing selection of a fixed dose corresponding to the 0.7 mg per kg dose to evaluate in the dose expansion portion of the cardiomyopathy arm. Our second in vivo knockout candidate, NTLA-2002, has strong momentum. and we remain on track to share a first look at safety and activity data from the first in-human study later this year. This will represent an opportunity to not only demonstrate the potential of NTLA-2002 as a treatment for people living with hereditary angioedema, but also the reproducibility of our modular platform. With our ex vivo efforts, we're advancing unique and proprietary allogeneic technology, which leverages our LNP-based cell engineering platforms. Our novel approach is designed to overcome rejection by host T and NK cells, a key limitation to the durability of current allogeneic investigational therapies. Based on our compelling preclinical data, we are making a strategic shift to focus on exclusively developing allogeneic cell therapies. For our lead ex vivo program, NTLA-5001, which you may recall is an autologous program, we are pivoting to an allogeneic version, which is now in preclinical development. Laura will share more on our decision and the advantages of our allogeneic platform for patients shortly. Finally, Intelli remains in a strong financial position. We ended the quarter with $907 million in cash, which gives us the capacity to continue to prosecute the strategic priorities we set forth at the beginning of this year. I'll now turn it over to David.
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