Intellia Therapeutics Announces Third Quarter 2018 Financial Results and Corporate Developments
- Enhanced in vivo CRISPR/Cas9 cargo components have led to substantially increased liver editing and protein reduction in non-human primates that could materially improve its transthyretin amyloidosis and other in vivo product profiles
- Integration of enhanced components into its lead transthyretin amyloidosis development candidate will move expected IND submission into 2020
- First to demonstrate robust CRISPR-mediated, site-specific insertion of transgenes in the liver of mice via proprietary LNP-AAV hybrid delivery approach
- Progressed transgenic TCR technology by showing that its engineered T cells efficiently recognize and kill myeloid leukemic blasts expressing WT1 antigen
- Broadened medical and regulatory science expertise by electing Dr.
Jesse Goodman, former Chief Scientist, Deputy Commissioner for Science and Public Healthand Director of the Center for Biologics Evaluation and Researchof the U.S. FDA, to its Board of Directors
$293 millionin cash and cash equivalents as of September 30, 2018
“We made excellent progress across our in vivo and ex vivo programs in the third quarter. In our ATTR program, Intellia previously reported that our proprietary LNP delivery technology could edit genes in the liver of non-human primates sufficiently to reduce TTR protein levels to a potential therapeutic range. Our continued in vivo delivery enhancement efforts have now produced striking improvements over these liver editing and protein knockdown levels. Data from the initial application of these enhancements in our ATTR program suggest that we may be able to double our previously reported liver editing results and more significantly reduce circulating protein beyond recognized therapeutic levels, at a lower, well-tolerated dose. We are applying these enhancements to develop a superior ATTR development candidate and we believe we can leverage these enhancements to significantly improve the performance of our development candidates for follow-on in vivo programs,” said Intellia President and Chief Executive Officer
“Furthermore, in our insertion programs, we successfully introduced functioning genes in mice using our modular LNP delivery system of CRISPR/Cas9 in combination with our proprietary AAV insertion templates developed in collaboration with Regeneron. In our presentation at ESGCT, Intellia was the first company to demonstrate technology that can insert genes in mice to produce, and fine tune, protein levels up to and greater than those required for normal human physiology.
“In parallel to these in vivo achievements, we are rapidly advancing our ex vivo immuno-oncology efforts, beginning with the development of best-in-class CRISPR-edited T cells for WT1,” added Dr. Leonard.
Third Quarter 2018 Operational Highlights and Recent Corporate Developments Include:
Transthyretin Amyloidosis Program Enhancements
Intellia announced today results from its transthyretin amyloidosis (ATTR) non-human primate (NHP) studies, conducted in collaboration with
Based on these new data, the Company is pursuing confirmatory studies with the goal of integrating enhanced cargo components in its Investigational New Drug (IND)-enabling studies and submission of an IND (previously planned for the end of 2019, but now targeted for 2020) for ATTR. In addition, the Company intends to apply these technology improvements to the rest of its in vivo product pipeline.
“We are elated to have achieved such impressive and compelling editing and protein knockdown results in our NHP studies. We believe that this approach, while introducing a relatively short delay to our previous IND timeline, will ultimately yield the best possible treatment option for patients – one that we hope will clearly advance the standard of care well beyond approved and potential therapies for the treatment of ATTR,” added Dr. Leonard.
Advancing Complex Genome Editing Capabilities
Intellia advanced its complex genome editing capabilities with the first robust demonstration of CRISPR-mediated, targeted insertion of transgenes in the liver of mice. The Company used its modular LNP delivery system of CRISPR/Cas9 in combination with Intellia’s proprietary modular adeno-associated virus (AAV) to insert donor template DNA into the albumin locus of mice. In collaboration with Regeneron, the Company used F9 as a model gene, which encodes Factor IX (FIX) protein, which is the clotting factor deficient in patients with hemophilia B. Using a proprietary bi-directional template platform technology, researchers showed that they can detect hybrid mAlb-hF9 transcripts in over 50 percent of hepatocytes following a single dose and measured circulating human FIX protein levels of more than 30,000 ng/mL. These levels are higher than those required in a clinical setting and correspond to levels predicted to be up to 40 to 300 times higher than those capable of preventing spontaneous bleeding episodes in hemophilia B patients, relative to wildtype or a hyperfunctional version of F9, respectively (sources: George, et al, NEJM, 2017; Simioni et al, NEJM, 2009). Furthermore, the Company observed that varying either the LNP or AAV dose modulated FIX levels. Protein levels remained stable after dosing throughout the 12-week observation period.
Intellia also applied the hybrid LNP-AAV delivery approach to its wholly owned in vivo preclinical program in alpha-1 antitrypsin deficiency (AATD). Alpha-1 antitrypsin (AAT), a protein that protects the lungs and is mutated or absent in patients with AATD, is produced by the SERPINA1 gene. To produce AAT protein, Intellia combined CRISPR/Cas9 delivery by LNP with the SERPINA1 DNA template delivered by AAV into the albumin locus. The targeted insertion generated gene expression levels in mice that correspond to levels that protect against the loss of pulmonary capacity in humans. This milestone underscores the utility of Intellia’s modular LNP-based delivery system and shows significant progress in developing an in vivo genome editing solution for AATD.
Progressing Transgenic T Cell Receptor Technology
Intellia and its research collaborator, Ospedale San Raffaele (OSR), presented data at the 26th Annual
Expanded Board of Directors and Management Team
Intellia announced today the appointment of
“We are thrilled to have someone with the broad scientific, public health, regulatory and clinical credentials of Jesse join Intellia’s board of directors,” said Intellia Chairman
Dr. Goodman is currently a professor of medicine at
Earlier this week, Intellia announced that
Strengthened Intellectual Property Position
Yesterday, the U.S. Patent and Trademark Office (USPTO) granted U.S. Patent No. 10,113,167 to The
Third 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 were
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.
Conference Call to Discuss Third Quarter 2018 Earnings and Corporate Developments
The Company will present third quarter 2018 results and corporate developments in a conference call on
To join the call:
- U.S. callers should dial 866-548-4713 and use conference ID# 5893807, approximately five minutes before the call.
- International callers should click here to access dial-in information and use conference ID# 5893807, approximately five minutes before the call.
A replay of the call will be available on Intellia’s website, beginning on
This press release contains “forward-looking statements” of
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 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 and quarterly reports on Form 10-Q filed with the Securities and Exchange Commission, 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 Therapeutics 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,|
|Research and development||23,237||17,481||69,197||46,477|
|General and administrative||8,270||5,711||23,481||17,812|
|Total operating expenses||31,507||23,192||92,678||64,289|
|Net loss per share, basic and diluted||$||(0.53||)||$||(0.44||)||$||(1.55||)||$||(1.25||)|
|Weighted average shares outstanding, basic and diluted||43,161||35,189||42,684||34,945|
|INTELLIA THERAPEUTICS, INC.|
|CONSOLIDATED BALANCE SHEET DATA (UNAUDITED)|
|(Amounts in thousands)|
|Cash and cash equivalents||$||293,248||$||340,678|
|Total stockholders' equity||263,270||300,597|
Jennifer Mound Smoter
Senior Vice President
External Affairs & Communications
Source: Intellia Therapeutics, Inc.