On August 6, 2020 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, reported operational highlights and financial results for the second quarter ended June 30, 2020 (Press release, Intellia Therapeutics, AUG 6, 2020, View Source [SID1234563137]).

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"We are very pleased to announce our first regulatory submission to initiate a Phase 1 study of NTLA-2001, which is an important milestone as we deliver on our mission to develop curative, CRISPR/Cas9-based treatments for severe diseases. Our CTA keeps us on track to dose our first ATTR patient by year-end and is a critical step in executing our global development strategy for the first systemically delivered CRISPR-based therapy to enter clinical trial," said Intellia President and Chief Executive Officer, John Leonard, M.D. "With our recently expanded collaboration with Regeneron and subsequent financing, we are well-capitalized to advance our initial wave of development programs and continue our work developing innovative modular platform capabilities."

Second Quarter 2020 and Recent Operational Highlights

ATTR Program: Intellia announced today it has submitted its first Clinical Trial Application (CTA) to the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a Phase 1 study of NTLA-2001 for the treatment of transthyretin amyloidosis (ATTR). Based on the Company’s in vivo liver knockout approach, NTLA-2001 is a potential single-course therapy for patients living with ATTR. Pending CTA authorization and subject to the impact of COVID-19, the Company is on track to dose its first patient by the end of 2020 with the first systemically delivered CRISPR/Cas9-based therapy to enter the clinic. The Company is submitting additional regulatory applications to enable enrollment in other countries as part of its global development strategy. NTLA-2001 is part of a co-development/co-promotion agreement between Intellia, the lead development and commercialization party, and Regeneron Pharmaceuticals, Inc. (Regeneron).

AML Program: NTLA-5001 is a wholly owned, T cell receptor (TCR)-T cell therapy development candidate targeting the Wilms’ Tumor 1 (WT1) antigen for the treatment of acute myeloid leukemia (AML). The Company’s approach aims to develop a broadly applicable treatment for AML patients, regardless of the mutational subtypes of the cancer. At the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting, held from May 12-15, 2020, Intellia presented data on its proprietary T cell engineering process in support of NTLA-5001. The data showed that the Company’s proprietary process enables multiple, highly efficient, sequential edits in T cells, whether knocking out or inserting genes. This technology yields engineered cells with high anti-tumor activity and favorable attributes, including a desired memory phenotype, which is associated with longer-lasting treatment effects. Importantly, chromosomal translocations (i.e., undesired chromosomal rearrangements) were similar to background levels in untreated cells. Intellia continues to advance Investigational New Drug application (IND)-enabling activities and remains on track to submit an IND or IND-equivalent for NTLA-5001 in the first half of 2021. Additional efforts are underway to evaluate potential use of the WT1-targeted TCR construct to treat solid tumors.

HAE Program: NTLA-2002 is a wholly owned, in vivo development candidate for the treatment of hereditary angioedema (HAE). Building on Intellia’s modular lipid nanoparticle (LNP) delivery system, NTLA-2002 is designed to knock out the prekallikrein B1 (KLKB1) gene in the liver after a single course of treatment, which is expected to prevent improperly regulated bradykinin production and therefore, reduce HAE attacks. In a non-human primate (NHP) study of its lead LNP formulation for NTLA-2002, the knockout of KLKB1 resulted in a therapeutically relevant reduction of serum kallikrein levels and activity following a single dose. Consistent with the durability achieved in earlier NHP studies for its lead in vivo program, the Company has now demonstrated sustained kallikrein activity reduction for 10 months in an ongoing study. Based on these results, Intellia believes NTLA-2002 could be efficacious and durable in preventing HAE attacks following a single course of treatment. Intellia continues to progress IND-enabling activities and is on track to submit an IND or IND-equivalent for NTLA-2002 in the second half of 2021.
CRISPR/Cas9-mediated targeted transgene insertion and allogeneic cell solutions. Additionally, to supplement its ex vivo capabilities, the Company established research collaborations and license agreements with TeneoBio and GEMoaB GmbH to develop engineered cell therapies for immuno-oncology and autoimmune diseases. Intellia’s broad platform innovations and expanded capabilities will drive the next wave of in vivo and ex vivo clinical candidates.

Regeneron Collaboration: In June 2020, Intellia and Regeneron announced an expansion of their existing collaboration to co-develop potential products for the treatment of hemophilia A and B. The collaboration expansion builds upon the jointly-developed targeted transgene insertion capabilities designed to durably restore a missing protein and to overcome the limitations of traditional gene therapy. The collaboration also provides Regeneron with rights to develop products for additional in vivo CRISPR/Cas9-based therapeutic liver targets. Under the expansion, the term of the collaboration has been extended until April 2024, at which point Regeneron has an option to renew for an additional two years. In addition, the expansion grants Regeneron a royalty-bearing, non-exclusive license to certain Intellia intellectual property to develop and commercialize up to 10 ex vivo CRISPR/Cas9 products in defined cell types. Intellia received $100 million through an upfront payment of $70 million and an additional $30 million equity investment from Regeneron at $32.42 per share.

Financing: In June 2020, Intellia closed an underwritten public offering of 6,301,370 shares of common stock, including the exercise in full of the underwriters’ option to purchase additional shares, at the public offering price of $18.25 per share. Intellia received aggregate gross proceeds of approximately $115 million, before underwriting discounts and commissions and offering expenses.

Modular Platform: Intellia continues to make significant progress across its platform technologies, broadening the in vivo and ex vivo application of genome editing. This includes developing innovative

Upcoming Events

The Company will participate in the following events during the third quarter of 2020:
Wedbush PacGrow Healthcare Conference, August 11, Virtual

Baird Healthcare Conference, September 9, Virtual

Upcoming Milestones

The Company has set forth the following for pipeline progression:

ATTR: Plan to dose first patient by end of 2020

AML: Submit an IND or IND-equivalent for NTLA-5001 in 1H 2021

HAE: Submit an IND or IND-equivalent for NTLA-2002 in 2H 2021Second Quarter 2020 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $436.8 million as of June 30, 2020, compared to $284.5 million as of December 31, 2019. The increase was driven by net proceeds of $107.7 million from the June follow-on public offering, $100.0 million upfront payment from the Regeneron collaboration expansion, which included a $30.0 million equity investment, $14.7 million of net equity proceeds raised from the Company’s "At the Market" (ATM) agreement, $14.4 million of funding received under existing collaborations with Regeneron and Novartis and $2.1 million in proceeds from employee-based stock plans. These increases were offset in part by cash used to fund operations of approximately $86.5 million.

Collaboration Revenue: Collaboration revenue increased by $5.1 million to $16.3 million during the second quarter of 2020, compared to $11.1 million during the second quarter of 2019. The increase in collaboration revenue in 2020 was mainly driven by an $8.4 million one-time cumulative catch-up adjustment related to the extension of the Regeneron collaboration.

R&D Expenses: Research and development expenses increased by $12.3 million to $37.8 million during the second quarter of 2020, compared to $25.5 million during the second quarter of 2019. This increase was primarily driven by IND-enabling activities related to our lead programs, research personnel growth to support these programs, and the expansion of the development organization.

G&A Expenses: General and administrative expenses decreased by $1.6 million to $11.5 million during the second quarter of 2020, compared to $13.1 million during the second quarter of 2019. This decrease was primarily driven by a decrease in legal expenses, which were principally related to a decrease in certain activities related to intellectual property matters.

Net Loss: The Company’s net loss was $32.4 million for the second quarter of 2020, compared to $25.7 million during the second quarter of 2019.

Financial Guidance

Intellia expects that its cash, cash equivalents and marketable securities as of June 30, 2020 will enable the Company to fund its anticipated operating expenses and capital expenditure requirements at least through the

next 24 months. This expectation excludes any strategic use of capital not currently in the Company’s base-case planning assumptions.

Conference Call to Discuss Second Quarter 2020 Earnings

The Company will discuss these results on a conference call today, August 6, 2020, at 8 a.m. ET.

To join the call:

U.S. callers should dial 1-877-317-6789 and international callers should dial +1-412-317-6789, approximately five minutes before the call.

All participants should ask to be connected to the Intellia Therapeutics conference call.

A replay of the call will be available through the Events and Presentations page of the Investors & Media section on Intellia’s website at www.intelliatx.com, beginning on August 6, 2020 at 12 p.m. ET.