Intellia Therapeutics Announces Third Quarter 2017 Financial Results
- First company to demonstrate dose-dependent CRISPR/Cas9 liver editing in non-human primates, including editing of over 20 percent in certain animals, following systemic delivery of Intellia’s proprietary, non-viral, lipid nanoparticle delivery system
- Well-tolerated safety profile observed to date in non-human primates after single and repeat doses, with rapid clearance of all CRISPR/Cas9 cargo and cationic lipid components, similar to Intellia’s prior safety data in rodent studies
- Presented 52-week CRISPR/Cas9 editing durability data in mice, confirming long-term liver genome editing (approximately 70 percent) with sustained protein reduction (approximately 97 percent) after a single intravenous administration
- Demonstrated lipid nanoparticle-mediated delivery and editing with CRISPR/Cas9 in the central nervous system in mice
$222 millionin cash and cash equivalents as of September 30, 2017
The Company made considerable progress in its Transthyretin Amyloidosis (TTR) program using its proprietary lipid nanoparticle (LNP) delivery system. Based on interim, top-line data from Intellia’s first exploratory non-human primate (NHP) studies, the Company has demonstrated successful editing in the livers of individual animals with NHP guide RNAs (gRNAs) as well as with exploratory human cross-reactive gRNAs. These interim results, coupled with on-going studies and further optimization efforts, are anticipated to allow for the selection of a human guide development candidate as early as the first quarter of 2018. In addition, Intellia completed a 12-month mouse durability study, maintaining approximately 97 percent reduction in serum TTR protein levels, accompanied by sustained editing at the target DNA site in the liver after a single intravenous administration of its CRISPR/Cas9 system.
“We accomplished some significant milestones during the third quarter, demonstrating the first CRISPR/Cas9 liver-targeted genome editing with systemic delivery of LNPs in non-human primates, the third animal species in which we have achieved editing. Our delivery system was safe and well tolerated in the studies, enabling us to redose without adverse events observed. In our 52-week mouse study, we demonstrated editing durability after a single dose of CRISPR/Cas9 using our delivery system. Moving beyond the liver, we also demonstrated the potential adaptability of our approach by showing delivery and editing in the central nervous system in mice. These data further advance Intellia’s mission to develop curative genome editing therapies for current unmet medical needs,” said Chief Executive Officer and Founder,
Third Quarter 2017 Highlights
The Company achieved several key milestones during the third quarter of 2017, including:
- Produced interim, top-line NHP data demonstrating, for the first time, liver genome editing using CRISPR/Cas9 delivered through a LNP system, including observing over 20 percent editing in certain animals:
- Observed editing ranges of 0.10 percent up to 32.0 percent after a single dose, with multiple animals showing editing greater than 20 percent after a second dose. These results were observed in experiments with various exploratory gRNA, LNP formulations and dosing regimens, similar to prior results from Intellia’s initial rodent studies;
- Confirmed re-dosing in NHPs produced increased levels of editing, with no adverse events observed;
- Well-tolerated safety profile observed to date with both NHP-specific gRNA and exploratory human cross-reactive gRNAs. This was assessed by gross observation, as well as by clinical chemistry, hematology, and cytokine and complement levels;
- Animals with the highest levels of editing showed a higher reduction in TTR serum protein levels; and
- Initiated further studies to optimize editing efficiency and to explore dosing parameters, which will lead toward the selection of a development candidate.
- Presented 12-month data from the completed long-term mouse study, demonstrating robust and durable in vivo genome editing following a single, systemic intravenous administration:
- Achieved and maintained approximately a 97 percent reduction in serum TTR protein levels through one year, corresponding with approximately 70 percent editing at the target DNA site in the liver;
- Confirmed the transient nature of Intellia’s LNP delivery system with 99 percent clearance of mRNA within 10 hours and clearance of sgRNA within 72 hours in the liver; and
- The treatment was observed to be safe and well tolerated during the 52-week study.
- Made progress in evaluating in vivo delivery by LNPs to a second organ, with successful genome editing by CRISPR/Cas9 in the central nervous system (cerebellum and striatum) in mice.
- Continued to defend and enhance the Company’s CRISPR/Cas9 foundational and therapeutic intellectual property position through filing and prosecution of patent applications covering its internal, collaboration and in-licensed inventions.
Third Quarter 2017 Financial Results
Collaboration revenue was
Since its 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 research and development programs, laboratory and related supplies, compensation and related expenses, legal and other regulatory expenses, patent prosecution, filing and maintenance costs for Intellia’s licensed intellectual property, and general overhead costs.
During the remainder of 2017, the Company expects expenses to continue to increase compared to prior periods relating to its ongoing activities, particularly as certain research and development activities progress toward human clinical trials, and as the Company spends a full year in its new office and laboratory facility, which it has occupied since the fourth quarter of 2016.
Conference Call to Discuss Third Quarter 2017 Earnings
The Company will present third quarter 2017 results to investors and analysts in a conference call on
To participate on the day, U.S. callers should dial 888-752-0423 and use Conference ID# 9089048, approximately five minutes before the call. International callers should dial +1 918-398-4936 and use Conference ID# 9089048, approximately five minutes before the call.
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 Intellia’s 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 editing; 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 the ex vivo cell therapeutics through our eXtellia division, including the development of next-generation T cell therapies that address unmet needs in hematological and solid tumors, immuno-oncology and auto-immunity; the intellectual property position and strategy of Intellia’s licensors; actions by government agencies; the impact of our collaborations with Ospedale San Raffaele,
|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,|
|Research and development||17,481||7,861||46,477||20,509|
|General and administrative||5,711||4,705||17,812||11,680|
|Total operating expenses||23,192||12,566||64,289||32,189|
|Net loss per share attributable to common stockholders, basic and diluted||$||(0.44||)||$||(0.22||)||$||(1.25||)||$||(1.16||)|
|Weighted average shares outstanding, basic and diluted||35,189||34,316||34,945||18,098|
|INTELLIA THERAPEUTICS, INC.|
|CONSOLIDATED BALANCE SHEET DATA (UNAUDITED)|
|(Amounts in thousands)|
|Cash and cash equivalents||$||222,264||$||273,064|
|Total stockholders' equity (deficit)||175,847||209,837|
Vice President, Investor Relations
Senior Vice President,
Source: Intellia Therapeutics, Inc.