Intellia Therapeutics Reports Financial Results for Second Quarter 2016
- Advancing proprietary and partnered pipeline candidates with
Novartis and Regeneron - Cash and cash equivalents of approximately
$300.7 million to accelerate CRISPR/Cas9 platform and pipeline development
“Intellia has made substantial progress with our science, financing and operations in the first half of 2016,” said
Recent Highlights
- On
April 11, 2016 , Intellia signed a multi-year research and development collaboration and licensing agreement withRegeneron Pharmaceuticals to advance CRISPR/Cas9 genome editing technology for in vivo therapeutic development. Regeneron has the exclusive rights to discover and develop CRISPR-based products against up to 10 targets, focused primarily on therapies for a broad range of diseases that may be treated by editing genes in the liver. Transthyretin amyloidosis (TTR) is the first target to be jointly developed and potentially commercialized by the companies. - The Company also strengthened its leadership team with the addition of
Perry Karsen as the Chairman of Intellia’s Board of Directors. Mr. Karsen brings decades of biopharmaceutical leadership experience to his role as Chairman. He most recently held senior leadership positions atCelgene Corporation , including Chief Operations Officer and Executive Vice President as well as Chief Executive Officer ofCelgene's cellular therapeutics division. - The Company, since its inception, has raised an aggregate of
$350.5 million , of which$170.5 million is from the initial public offering and concurrent private placements inMay 2016 ,$95 million is through collaboration agreements, and$85 million is from the sale of convertible preferred stock.
Second Quarter 2016 Financial Results
As of
Collaboration revenue was
Research and development expenses in the second quarter 2016 were
General and administrative expenses were
Research & Development Highlights
Intellia is advancing its pipeline through a risk-mitigated approach focused on sentinel indication development, platform delivery expansion, and preclinical and clinical scale up. The Company is focused on developing the following programs:
Programs | Partnerships | Type of Edit |
Delivery | Upcoming Milestones | ||||||||
In Vivo | ||||||||||||
Transthyretin Amyloidosis (ATTR) | Co-developing with Regeneron |
Knockout | LNP to Liver | Select 1 to 2 development candidates and advance to IND enabling studies in 2H2017/1H2018 |
||||||||
Alpha-1 Antitrypsin Deficiency (AATD) |
Proprietary | Knockout | LNP to Liver | |||||||||
Repair | ||||||||||||
Hepatitis B Virus (HBV) | Proprietary | Knockout | LNP to Liver | |||||||||
Inborn Errors of Metabolism (IEMs) |
Proprietary | Knockout | LNP to Liver | |||||||||
Repair | ||||||||||||
Insertion | ||||||||||||
Ex Vivo | ||||||||||||
Hematopoietic Stem Cells (HSCs) |
Selectively partnered with Novartis; proprietary |
Knockout | Electroporation | First Novartis IND expected to be submitted in 2018 | ||||||||
Repair | ||||||||||||
Insertion | ||||||||||||
CAR T Cells | Partnered with Novartis |
Knockout | Electroporation | Advance preclinical development | ||||||||
Insertion |
Upcoming Events
- Intellia will present delivery data utilizing CRISPR/Cas9 at the Cold Spring Harbor Laboratory Meeting, taking place from
August 17-20, 2016 . The oral presentation, Robust In Vivo Gene Editing in Mouse Hepatocytes with Systemic Lipid Nanoparticle Delivery of CRISPR/Cas9 Components, will be presented by Intellia’s Chief Technology Officer,David Morrissey , Ph.D. - Intellia’s CEO & Founder
Nessan Bermingham , Ph.D., will be presenting at theWedbush PacGrow Healthcare Conference inNew York onAugust 17, 2016 , theWells Fargo Healthcare Conference inBoston ,September 7-8, 2016 , theMorgan Stanley Global Healthcare Conference inNew York onSeptember 12, 2016 , and the Leerink Partners Rare Disease Roundtable Series inNew York onSeptember 28-29, 2016 .
About
Forward-Looking Statements
This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. These forward looking statements include, but are not limited to, statements regarding our ability to advance CRISPR/Cas9 into therapeutic products for severe and life-threatening diseases; the potential timing and advancement of our clinical trials; the impact of our collaborations with
Intellia Therapeutics, Inc. | |||||||||||||||||||
Consolidated Statements of Operations | |||||||||||||||||||
(unaudited) | |||||||||||||||||||
(in thousands) | |||||||||||||||||||
Three Months Ended June 30, | Six Months Ended June 30, | ||||||||||||||||||
2016 | 2015 | 2016 | 2015 | ||||||||||||||||
Collaboration revenue | $ | 4,206 | $ | 1,377 | $ | 5,983 | $ | 2,663 | |||||||||||
Operating expenses: | |||||||||||||||||||
Research and development | 7,422 | 1,966 | 12,647 | 3,337 | |||||||||||||||
General and administrative | 3,729 | 2,833 | 6,975 | 3,943 | |||||||||||||||
Total operating expenses | 11,151 | 4,799 | 19,622 | 7,280 | |||||||||||||||
Operating loss | (6,945 | ) | (3,422 | ) | (13,639 | ) | (4,617 | ) | |||||||||||
Interest income | 46 | - | 51 | - | |||||||||||||||
Loss before income taxes | (6,899 | ) | (3,422 | ) | (13,588 | ) | (4,617 | ) | |||||||||||
Income tax benefit | - | 382 | - | 484 | |||||||||||||||
Net loss | $ | (6,899 | ) | $ | (3,040 | ) | $ | (13,588 | ) | $ | (4,133 | ) |
Intellia Therapeutics, Inc. | ||||||||||
Consolidated Balance Sheets Data | ||||||||||
(unaudited) | ||||||||||
(in thousands) | ||||||||||
June 30, 2016 |
December 31, 2015 |
|||||||||
Cash and cash equivalents | $ | 300,687 | $ | 75,816 | ||||||
Total assets | 310,624 | 82,139 | ||||||||
Total liabilities | 87,022 | 14,783 | ||||||||
Convertible preferred stock | - | 88,557 | ||||||||
Total stockholders' equity (deficit) | 223,602 | (21,201 | ) |
Media Contact:Jennifer Mound Smoter Chief External Affairs & Communications Officer +1 224-804-4462 jenn.smoter@intelliatx.com Investor Contacts:John Graziano Trout Group 646-378-2942 jgraziano@troutgroup.comChad Rubin Trout Group 646-378-2947 crubin@troutgroup.com