Intellia Therapeutics Announces First Quarter 2018 Financial Results
- New CEO puts in vivo and ex vivo genome editing on parallel tracks towards the clinic
- Company anticipates submitting its in vivo Investigational New Drug application by the end of 2019 for systemic lipid nanoparticle delivery of CRISPR/Cas9 to potentially cure transthyretin amyloidosis
- Consistently achieved 60 to 80 percent reduction in transthyretin protein production following a single dose of CRISPR/Cas9 delivered systemically via lipid nanoparticle to hepatocytes of non-human primates
- Achieved meaningful levels of genome editing using modular lipid nanoparticle platform technology against various additional liver targets in mice
$328 millionin cash and cash equivalents as of March 31, 2018
The Company announced today that it anticipates submitting an IND application for its lead indication, transthyretin amyloidosis (ATTR), by the end of 2019 and confirms plans to initiate IND-enabling studies in mid-2018. Over the past six months, ongoing NHP studies have demonstrated well-tolerated editing to therapeutically relevant levels of transthyretin (TTR) protein reduction (60 to 80 percent) after a single systemic administration via LNP delivery to NHP hepatocytes. Rates of editing were durable over the six-month period without re-dosing the animals. In support of the proposed IND submission, Intellia has narrowed the field of potential guides to its current development candidate for early human trials. The guide-optimization process used high-throughput screening to evaluate the entire TTR gene for those guides with high levels of activity and undetectable off-target cutting. The Company has completed studies to understand potential dosing regimens and is continuing studies on durability of the effect, both of which may expedite Phase I clinical trials. Intellia has also developed an enhanced LNP formulation through optimization campaigns that is currently being tested for multiple follow-on liver indications, and anticipates that this modular approach may minimize development timelines for each additional and subsequent liver-targeted product candidate.
Intellia has also demonstrated continued progression of its modular liver platform capability to knockout various targets of interest in the livers of mice, including SERPINA1 for alpha-1 antitrypsin deficiency (AATD) and HAO1 for primary hyperoxaluria type 1 (PH1), each of which has resulted in protein expression reductions believed to be therapeutically relevant. This initial knockout edit in AATD lays the groundwork for developing an approach that restores production of the missing protein in AATD, required for the amelioration of the disease.
The table below shows editing rates and corresponding protein reductions in the livers of mice for ATTR, AATD and PH1. ATTR and AATD both produce aberrant proteins hence treatment of these conditions requires reductions in the level of the disease-causing proteins. PH1 results from the low level activity of a particular protein for which treatment requires reducing the levels of substrate for that defective protein to metabolize, achieved by knocking out the gene that encodes HAO1. In each of these three cases, Intellia’s modular LNP delivery system achieved high levels of reduction of the targeted protein. These initial editing rates and corresponding protein reductions are evidence of Intellia’s ability to successfully target monogenic liver diseases by knocking out harmful genetic mutations.
|Target||Indication||% editing in
|SERPINA1||Alpha-1 antitrypsin deficiency||85||%||95||%|
|HAO1||Primary hyperoxaluria type 1||74||%||90||%|
Beyond the liver, the Company continues to advance its application of CRISPR/Cas9 technology to the central nervous system (CNS), including through its collaboration with
In ex vivo applications, Intellia seeks to develop allogeneic cellular therapies, which are cells derived from unmatched tissue donors, which are modified outside of the human body to allow them to be administered to an unrelated patient. This endeavor is supported through multiple efforts, including recently acquired access to intellectual property from researchers at the
In February of 2018, Cell Reports published Intellia’s first peer-reviewed paper entitled “A single administration of CRISPR/Cas9 lipid nanoparticles achieves robust and persistent in vivo genome editing.” This landmark paper documented Intellia’s delivery of Cas9 mRNA and single guide RNA using its proprietary LNPs to achieve a 97 percent reduction in mouse TTR protein levels in the liver, which was sustained for at least 12 months.
During the course of 2018, Intellia plans to share additional preclinical data on its TTR genome editing program, including the achievement of a near ten-fold reduction in the required dose, derived via improvements in potency, as well as other knockout targets and data on delivery via LNPs to the CNS of NHPs. Additionally, Intellia plans to share preclinical data on both immuno-oncology and autoimmune disease targets in 2018.
First Quarter 2018 Financial Results
Collaboration revenue was
Since inception through
Research and development expenses increased by
General and administrative expenses increased by
The Company’s net loss was
Cash and cash equivalents at
The Company’s primary uses of capital will continue to be for research and development programs, laboratory and related supplies, compensation costs for current and future employees, consulting, legal and other regulatory expenses, patent prosecution filing and maintenance costs for Intellia’s licensed intellectual property, and general overhead costs.
Upcoming Events During the Second Quarter 2018
The Company expects to make presentations at the following upcoming scientific and investor conferences:
The American Society of Gene and Cell TherapyAnnual Meeting, May 16, Chicago Jefferies Global Health Care Conference, June 5, New York City JMP Securities Life Sciences Conference, June 20, New York City
This press release contains "forward-looking statements" of 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 our ability to advance and expand the CRISPR/Cas9 technology to develop into human therapeutic products, as well as our CRISPR/Cas9 intellectual property portfolio; our ability to achieve stable or effective genome editing with a single treatment dose; the potential timing and advancement of our preclinical studies, including continuing non-human primate studies, and clinical trials; our ability to replicate results achieved in our preclinical studies in any future studies, including human clinical trials; the potential development of ex vivo cell therapeutics of all types using CRISPR/Cas9 technology; our ability to conduct successful IND-enabling studies of a lead ATTR development candidate and subsequently submitting an IND application by the end of 2019 that will be accepted by the regulatory agencies; our intent to present additional data for organs beyond the liver, additional insertion/repair data, and preclinical data in support of our first ex vivo programs on immuno-oncology and autoimmune/inflammation indications during 2018; our ability to nominate a development candidate for a second indication by late 2018; the intellectual property position and strategy of Intellia’s licensors; actions by government agencies; 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 through mid-2020. 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 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
|INTELLIA THERAPEUTICS, INC.|
|CONSOLIDATED STATEMENTS OF OPERATIONS (UNAUDITED)|
|(Amounts in thousands, except per share data)|
|Three Months Ended March 31,|
|Research and development||22,493||13,431|
|General and administrative||7,406||5,732|
|Total operating expenses||29,899||19,163|
|Net loss per share attributable to common stockholders, basic and diluted||$||(0.51||)||$||(0.36||)|
|Weighted average shares outstanding, basic and diluted||42,043||34,723|
|INTELLIA THERAPEUTICS, INC.|
|CONSOLIDATED BALANCE SHEET DATA (UNAUDITED)|
|(Amounts in thousands)|
|Cash and cash equivalents||$||327,778||$||340,678|
|Total stockholders' equity||295,499||300,597|
Vice President, Investor Relations
Senior Vice President,
Source: Intellia Therapeutics, Inc.