CAMBRIDGE, Mass., Nov. 05, 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 third quarter ended September 30, 2020.

“We are very pleased with the recent regulatory authorization to begin our Phase 1 study of NTLA-2001, which keeps us on track to dose our first patient by year-end. This is an important step toward improving the lives of ATTR patients with a potentially curative treatment, and marks our transition into a clinical-stage company. Further, advancing NTLA-2001 is a major milestone for the field of genome editing, as this is the first clinical trial of a systemically delivered CRISPR/Cas9-based therapy,” said Intellia President and Chief Executive Officer John Leonard, M.D. “In parallel, we are progressing NTLA-5001 and NTLA-2002 for the treatment of AML and HAE, respectively, each to an IND or equivalent regulatory submission next year. We also continue to develop innovative capabilities across our platform, based on the Nobel Prize-winning CRISPR/Cas9 technology, to generate our next wave of therapeutic candidates.”

Third Quarter 2020 and Recent Operational Highlights

Upcoming Events

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

Upcoming Milestones

The Company has set forth the following for pipeline progression:

Third Quarter 2020 Financial Results

Financial Guidance

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

The Company will discuss these results on a conference call today, November 5, 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 November 5, 2020 at 12 p.m. ET.

About Intellia Therapeutics
Intellia Therapeutics is a leading genome editing company, focused on the development of proprietary, potentially curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by both producing therapeutics that permanently edit and/or correct disease-associated genes in the human body with a single treatment course, and creating enhanced engineered cells that can treat oncological and immunological diseases. Intellia’s combination of deep scientific, technical and clinical development experience, along with its leading intellectual property portfolio, puts it in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create new classes 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”, “we” or “our”) 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: being able to initiate clinical studies for NTLA-2001 for the treatment of transthyretin amyloidosis (“ATTR”) pursuant to its clinical trial applications (“CTA”), including dosing of a first patient by the end of 2020, and submitting similar regulatory applications in other countries; plans to submit an investigational new drug (“IND”) application or similar clinical trial application 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 submit an IND or similar clinical trial application for its hereditary angioedema (“HAE”) program in the second half of 2021; plans to advance and complete preclinical studies, including non-human primate studies for its HAE and other programs, and other animal studies supporting other in vivo and ex vivo programs, including its AML program; 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; further development of its proprietary cell engineering process for multiple sequential editing; 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; 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 develop other in vivo or ex vivo cell therapeutics of all types, and those targeting WT1 in AML in particular, using CRISPR/Cas9 technology; ability to optimize the impact of its collaborations on its development programs, including but not limited to its collaborations with Novartis Institutes for BioMedical Research, Inc. (“Novartis”) or Regeneron Pharmaceuticals, Inc. (“Regeneron”), including its co-development programs for hemophilia A and hemophilia B; statements regarding the timing of regulatory filings and clinical trial execution, including dosing of patients, regarding its development programs; the potential commercial opportunities, including value and market, for our product candidates; our expectations regarding our use of capital and other financial results during 2020; and our ability to fund operations for at least 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 regulatory agencies’ evaluation of regulatory filings and other information related to its product candidates; 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, including those that are co-developed, 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 (“SEC”). 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 September 30, Nine Months Ended September 30, 
       2020   2019   2020   2019  
 Collaboration revenue $22,220  $10,616  $51,399  $32,167  
 Operating expenses:         
  Research and development 39,756   27,513   112,177   76,682  
  General and administrative 10,566   8,431   33,406   32,082  
   Total operating expenses 50,322   35,944   145,583   108,764  
 Operating loss   (28,102)  (25,328)  (94,184)  (76,597) 
 Interest income   262   1,694   2,145   5,340  
 Net loss   $(27,840) $(23,634) $(92,039) $(71,257) 
 Net loss per share, basic and diluted$(0.47) $(0.49) $(1.70) $(1.53) 
 Weighted average shares outstanding, basic and diluted 58,754   48,554   54,218   46,547  
              


     
INTELLIA THERAPEUTICS, INC.
CONSOLIDATED BALANCE SHEET DATA (UNAUDITED)
(Amounts in thousands)
     
  September 30,
2020		 December 31,
2019
Cash, cash equivalents and marketable securities $407,947 $284,472
Total assets 458,555  334,280
Total liabilities 128,935  64,399
Total stockholders' equity 329,620  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