CAMBRIDGE, Mass., Aug. 06, 2020 (GLOBE NEWSWIRE) -- 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, today reported operational highlights and financial results for the second quarter ended June 30, 2020.

“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

Upcoming Events

The Company will participate in the following events during the third quarter of 2020:

Upcoming Milestones

The Company has set forth the following for pipeline progression:

Second Quarter 2020 Financial Results

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:

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.

About Intellia Therapeutics
Intellia Therapeutics is a leading genome editing company focused on developing proprietary, curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course, and through improved cell therapies that can treat cancer and immunological diseases, or can replace patients’ diseased cells. The combination of deep scientific, technical and clinical development experience, along with its leading intellectual property portfolio, puts Intellia in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Learn more about Intellia Therapeutics and CRISPR/Cas9 at intelliatx.com and follow us on Twitter @intelliatweets.

Forward-Looking Statements
This press release contains “forward-looking statements” of Intellia Therapeutics, Inc. (“Intellia” or the “Company”) within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellia’s beliefs and expectations regarding its: receiving authorization to initiate clinical studies for NTLA-2001 for the treatment of transthyretin amyloidosis (“ATTR”) pursuant to its clinical trial application (“CTA”) or similar regulatory applications, and the planned dosing of first patients by the end of 2020; plans to complete manufacturing activities and submit an investigational new drug application (“IND”) or equivalent regulatory filing for NTLA-5001, its first T cell receptor (“TCR”)-directed engineered cell therapy development candidate for its acute myeloid leukemia (“AML”) program in the first half of 2021; plans to complete manufacturing activities and submit an IND or equivalent regulatory filing for NTLA 2002, a development candidate for its hereditary angioedema (“HAE”) program in the second half of 2021; plans to advance and complete preclinical studies, including any necessary non-human primate studies, for its ATTR program, AML program, HAE program and other in vivo and ex vivo programs; development of a proprietary LNP/AAV hybrid delivery system, as well as its modular platform to advance its complex genome editing capabilities, such as gene insertion; presentation of additional data at upcoming scientific conferences, and other preclinical data in 2020; advancement and expansion of its CRISPR/Cas9 technology to develop human therapeutic products, as well as its ability to maintain and expand its related intellectual property portfolio; ability to demonstrate its platform’s modularity and replicate or apply results achieved in preclinical studies, including those in its ATTR, AML, and HAE programs, in any future studies, including human clinical trials; ability to develop other in vivo or ex vivo cell therapeutics of all types, and those targeting WT1 in AML in particular, using CRISPR/Cas9 technology; expectations of the potential impact of the coronavirus disease 2019 pandemic on strategy, future operations and timing of its clinical trials or IND submissions; ability to optimize the impact of its collaborations on its development programs, including but not limited to its collaborations with Novartis or Regeneron, including its co-development programs for hemophilia A and hemophilia B; statements regarding the timing of regulatory filings regarding its development programs; use of capital, expenses, future accumulated deficit and other 2020 financial results or in the future; and ability to fund operations at least through the next 24 months.

Any forward-looking statements in this press release are based on management’s current expectations and beliefs of future events, and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to Intellia’s ability to protect and maintain its intellectual property position; risks related to Intellia’s relationship with third parties, including its licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; uncertainties related to the authorization, initiation and conduct of studies and other development requirements for its product candidates; the risk that any one or more of Intellia’s product candidates will not be successfully developed and commercialized; the risk that the results of preclinical studies or clinical studies will not be predictive of future results in connection with future studies; and the risk that Intellia’s collaborations with Novartis or Regeneron or its other ex vivo collaborations will not continue or will not be successful. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellia’s actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in Intellia’s most recent annual report on Form 10-K as well as discussions of potential risks, uncertainties, and other important factors in Intellia’s other filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Intellia undertakes no duty to update this information unless required by law.


INTELLIA THERAPEUTICS, INC.
CONSOLIDATED STATEMENTS OF OPERATIONS (UNAUDITED)
(Amounts in thousands, except per share data)
          
   Three Months Ended June 30, Six Months Ended June 30,
    2020   2019   2020   2019 
Collaboration revenue$16,263  $11,118  $29,179  $21,551 
Operating expenses:       
 Research and development 37,771   25,460   72,421   49,169 
 General and administrative 11,526   13,118   22,840   23,651 
  Total operating expenses 49,297   38,578   95,261   72,820 
Operating loss (33,034)  (27,460)  (66,082)  (51,269)
Interest income 641   1,777   1,883   3,646 
Net loss$(32,393) $(25,683) $(64,199) $(47,623)
Net loss per share, basic and diluted$(0.61) $(0.56) $(1.24) $(1.05)
Weighted average shares outstanding, basic and diluted 53,369   45,814   51,938   45,526 
          


     
INTELLIA THERAPEUTICS, INC.
 
CONSOLIDATED BALANCE SHEET DATA (UNAUDITED)
 
(Amounts in thousands)
 
     
  June 30,
2020		 December 31,
2019
 
Cash, cash equivalents and marketable securities$436,803  $284,472  
Total assets  490,830   334,280  
Total liabilities  139,243   64,399  
Total stockholders' equity  351,587   269,881  
     


Intellia Contacts:

Investors:
Lina Li
Associate Director
Investor Relations
+1 857-706-1612
lina.li@intelliatx.com

Media:
Jennifer Mound Smoter
Senior Vice President
External Affairs & Communications
+1 857-706-1071
jenn.smoter@intelliatx.com