Intellia Therapeutics Announces Second Quarter 2018 Financial Results
- Continued to progress lead in vivo program in transthyretin amyloidosis targeting submission of an Investigational New Drug application by the end of 2019
- Announced first wholly owned ex vivo T cell receptor program targeting a unique epitope of Wilms’ Tumor 1 protein for the treatment of acute myeloid leukemia
- Strengthened foundational CRISPR/Cas9 genome editing intellectual property position through granting of new patent in the U.S.
$306 millionin cash and cash equivalents as of June 30, 2018
“In the first half of 2018, we focused on executing against our priorities, including initiating IND-enabling studies for our lead in vivo liver program, demonstrating that our lipid nanoparticle delivery platform has broad utility, and advancing our ex vivo R&D efforts across our key collaborations. We are excited about our progress, and we remain focused on achieving our 2018 goals,” said Intellia President and Chief Executive Officer
Second Quarter 2018 Operational Highlights
The Company achieved several key operational milestones during the second quarter of 2018, including the following:
- Intellia continued progress in non-human primate (NHP) dose-ranging studies that support the Company’s lead in vivo program in transthyretin amyloidosis (ATTR).
- At the 21st Annual Meeting of the
American Society of Gene and Cell Therapyin Chicago, Intellia and research collaborator, Ospedale San Raffaele, announced the identification of T cell receptors (TCRs) targeting a Wilms’ Tumor 1 protein (WT1) epitope, important for the treatment of acute myeloid leukemia as Intellia’s first ex vivo program. TCRs that target WT1 may also have applicability in solid tumors and other hematological malignancies, as WT1 is over-expressed on many tumor types. The Company plans to develop these TCRs as part of its first ex vivo product candidate. Intellia anticipates that this program will benefit from ongoing work to develop a truly allogeneic approach for engineered cellular therapy.
- In the second quarter, Intellia progressed ex vivo multiplexing efforts to achieve triple knockout edits with greater than 80 percent efficiency in human cells, and observed insertion efficacy of ~50 percent with simultaneous double knockout edits. Capabilities in multiplexing will support and advance ex vivo efforts within the Company’s expanding engineered cell therapy pipeline.
- Expanding on the in vivo liver editing achievements announced earlier this year, the Company progressed its primary hyperoxaluria type I (PH1) program utilizing a phenotypic mouse model of the disease. In PH1, excess oxalate produced in the liver crystallizes and accumulates in various organs eventually causing kidney failure. A knockout of the HAO1 gene reduces levels of glyoxylate, a precursor to urinary oxalate, and thereby reduces oxalate accumulation. In a mouse model of the disease, the Company achieved 74 percent editing of HAO1 leading to a ~90 percent protein reduction and a ~55 percent reduction in urinary oxalate after a single dose. This progress reinforces the value and speed of Intellia’s modular lipid nanoparticle delivery platform, including efficient and effective delivery to hepatocytes in the liver.
- Intellia continued to expand its fully automated, next-generation sequencing and bioinformatics platform to support the identification of highly active guides with limited to no off-target cutting or unforeseen deletions. In the second quarter, Intellia increased throughput capacity to process greater than 30,000 sequencing samples per week. This capability enables measuring of on- and off-target genome editing, including indels, translocations, excisions and inversions.
- Intellia, along with other licensees, announced in June that the U.S. Patent and Trademark Office (USPTO) granted U.S. Patent No. 10,000,772 (“the ’772 patent”) to The Regents of the University of
California, the University of Vienna and Emmanuelle Charpentier, Ph.D. (collectively, “UC”), co-owners of foundational intellectual property relating to CRISPR/Cas9 genome editing technology. The ’772 patent covers the use of single- and dual-guide RNA formats having certain structural motifs in a region that interacts with the Cas9 enzyme. These guide RNA formats are widely used in the CRISPR/Cas9 field. Intellia anticipates this is the first of many patents to be granted in the U.S. to UC for the CRISPR/Cas9 genome editing intellectual property. Outside of the U.S., UC continues to hold a strong global intellectual property position with applications from this patent estate issued in Europe, the United Kingdom, China, Japan, Australiaand various other countries worldwide. The ’772 patent is not involved in the appeal to the U.S. Court of Appeals for the Federal Circuit(CAFC) relating to the February 2017interference decision from the USPTO’s Patent Trial and Appeal Board. UC’s appeal was heard on April 30, 2018by the CAFC, and a decision is still pending.
- For the remainder of 2018, Intellia’s expected milestones include the following:
- Advance a second liver knockout target in NHPs;
- Advance candidates for a second liver indication;
- Present additional editing data supporting more complex edits such as insertion and repair;
- Prepare for a pre-Investigational New Drug meeting with the
U.S. Food and Drug Administrationfor ATTR;
- Expand preclinical data in support of Intellia’s first proprietary ex vivo autoimmune program; and
- Identify Intellia’s first hematopoietic stem cell target from the collaboration with
Second 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, intellectual property related costs, and general operating costs.
Upcoming Events During the Third Quarter 2018
The Company expects to make presentations at the following upcoming investor conferences:
Riley FBR Health Care Conference, Sept. 4, New York City Citi Biotech Conference, Sept. 5, Boston Wells Fargo Health Care Conference, Sept. 6, Boston
- Jefferies Gene Therapy Summit,
Sept. 27, 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; the potential timing and advancement of our preclinical studies, including non-human primate studies for our ATTR program and a second liver knockout program, 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 therapies of all types, and those targeting WT1 in particular, using CRISPR/Cas9 technology; our intent to present additional data for organs beyond the liver, ex vivo and in vivo therapeutics, additional insertion/repair data, and preclinical data in support of our first ex vivo programs on immuno-oncology and autoimmune/inflammation indications during 2018; the expansion of our fully automated bioinformatics platform; our ability to advance candidates for a second liver indication by late 2018; our potential ability to conduct a pre-IND meeting with the
|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,|
|Research and development||23,467||15,565||45,960||28,996|
|General and administrative||7,805||6,369||15,211||12,101|
|Total operating expenses||31,272||21,934||61,171||41,097|
|Net loss per share, basic and diluted||$||(0.52||)||$||(0.45||)||$||(1.03||)||$||(0.81||)|
|Weighted average shares outstanding, basic and diluted||42,836||34,916||42,441||34,820|
|INTELLIA THERAPEUTICS, INC.|
|CONSOLIDATED BALANCE SHEET DATA (UNAUDITED)|
|(Amounts in thousands)|
|Cash and cash equivalents||$||305,538||$||340,678|
|Total stockholders' equity||279,336||300,597|
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External Affairs & Communications
Source: Intellia Therapeutics, Inc.