Loading, Please Wait...
CAMBRIDGE, Mass., Feb. 27, 2019 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc.® (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology in both in vivo and ex vivo applications, today reported operational highlights and financial results for the fourth quarter and year ended December 31, 2018. In addition, Intellia highlighted select corporate milestones for 2019 and upcoming events for the first quarter of 2019.
“We are delivering on our full-spectrum strategy, and we expect to have two candidates in development in 2019. Our in vivo transthyretin amyloidosis program is on track for IND filing in 2020, and by the end of this year we anticipate having our first engineered cell therapy development candidate targeting acute myeloid leukemia,” said Intellia President and Chief Executive Officer John Leonard, M.D. “We have shown that a single lipid nanoparticle administration can produce very substantial transthyretin protein reduction in non-human primates, reaching levels that we believe hold great therapeutic promise for patients. We also look forward to sharing additional data on our acute myeloid leukemia program this year. We believe our TCR-based, CRISPR-engineered cell therapy will provide a much-needed option for patients.”
Recent Operational Highlights
The Company has set forth the following for 2019 pipeline progression:
The Company will participate in the following investor events:
Fourth Quarter and Full Year 2018 Financial Results
Intellia expects that its cash, cash equivalents and marketable securities as of December 31, 2018, as well as technology access and funding from Novartis and Regeneron, will enable Intellia to fund its anticipated operating expenses and capital expenditure requirements into the first half of 2021. This expectation excludes any potential milestone payments or extension fees that could be earned and distributed under the collaboration agreements with Novartis and Regeneron or any strategic use of capital not currently in the base-case planning assumptions.
About Intellia Therapeutics
Intellia Therapeutics® is a leading genome editing company focused on developing 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.
This press release contains “forward-looking statements” of Intellia Therapeutics, Inc. (“Intellia”) 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 ability to advance and expand the CRISPR/Cas9 technology to develop into human therapeutic products, as well as our intellectual property portfolio; our ability to achieve stable or effective genome editing; our ability to administer our CRISPR/Cas9 product candidates; the potential timing and advancement of our preclinical studies, including continuing non-human primate studies for our ATTR program and other programs (such as alpha-1 antitrypsin deficiency (AATD) and AML), and clinical trials; the timing and potential achievement of milestones to advance our pipeline including nominating development candidates and filing INDs; our ability to replicate or apply results achieved in our preclinical studies, including those in our ATTR, AATD and AML programs, in any future studies, including human clinical trials; the potential development of our proprietary LNP/AAV hybrid delivery system to advance our complex genome editing capabilities; the potential development of other in vivo or ex vivo cell therapeutics of all types, and those targeting WT1 in AML in particular, using CRISPR/Cas9 technology; our ability to conduct successful IND-enabling studies of a lead ATTR development candidate and subsequently submitting an IND application in 2020 that will be accepted by the regulatory agencies; our intent to present additional ATTR data, additional insertion/repair data, and other preclinical data during 2019 or thereafter; our ability to advance a development candidate for a second indication by late 2019 or thereafter; our plans to commence manufacturing efforts in 2019; the intellectual property position and strategy of Intellia’s licensors, or other parties from which it derives rights, as well as third-parties and competitors; actions by government agencies; our growth as a company and the anticipated contribution of the members of our board of directors and our executives to our operations and progress; the impact of our collaborations on our development programs; the potential timing of regulatory filings regarding our development programs; the potential commercialization opportunities, including value and market, for product candidates; our expectations regarding our uses of capital, expenses, future accumulated deficit and other 2018 financial results; and our ability to fund operations into the first half of 2021.
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 our intellectual property position; risks related to Intellia’s relationship with third parties, including our licensors; risks related to the ability of our licensors to protect and maintain their intellectual property position; uncertainties related to the initiation and conduct of studies and other development requirements for our 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 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 December 31,||Twelve Months Ended December 31,|
|Research and development||19,918||21,170||89,115||67,647|
|General and administrative||8,708||10,213||32,189||28,025|
|Total operating expenses||28,626||31,383||121,304||95,672|
|Net loss per share, basic and diluted||$||(0.43)||$||(0.61)||$||(1.98)||$||(1.88)|
|Weighted average shares outstanding, basic and diluted||44,215||39,155||43,069||36,006|
|INTELLIA THERAPEUTICS, INC.|
|CONSOLIDATED BALANCE SHEET DATA (UNAUDITED)|
|(Amounts in thousands)|
|Cash, cash equivalents and marketable securities||$||314,059||$||340,678|
|Total stockholders' equity||277,920||300,597|
Jennifer Mound Smoter
Senior Vice President
External Affairs & Communications