Intellia Therapeutics Announces Third Quarter 2020 Financial Results
- On track to dose first patient by year-end with NTLA-2001 for the treatment of transthyretin amyloidosis (ATTR), following regulatory authorization to initiate Phase 1 clinical trial
- Anticipates submitting an IND or IND-equivalent for lead TCR-T cell therapy, NTLA-5001 for the treatment of acute myeloid leukemia (AML), in 1H 2021
- Expects to submit an IND or IND-equivalent for NTLA-2002 for the treatment of hereditary angioedema (HAE) in 2H 2021
- Ended quarter with a strong cash position of $407.9 million
“We are very pleased with the recent regulatory authorization to begin our Phase 1 study of NTLA-2001, which keeps us on track to dose our first patient by year-end. This is an important step toward improving the lives of ATTR patients with a potentially curative treatment, and marks our transition into a clinical-stage company. Further, advancing NTLA-2001 is a major milestone for the field of genome editing, as this is the first clinical trial of a systemically delivered CRISPR/Cas9-based therapy,” said Intellia President and Chief Executive Officer
Third Quarter 2020 and Recent Operational Highlights
- ATTR Program: Intellia recently announced the authorization of its Clinical Trial Application (CTA) by the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a first-in-human clinical trial of NTLA-2001, an investigational therapy in development for the treatment of all clinical manifestations of ATTR. By applying the Company’s in vivo liver gene knockout technology, NTLA-2001 allows for the possibility of lifelong transthyretin (TTR) protein reduction after a single course of treatment. Intellia’s first-in-human study will evaluate NTLA-2001 in adults with hereditary ATTR with polyneuropathy (hATTR-PN). The Phase 1 study will be a two-part, open label, multi-center study to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of NTLA-2001, which will include the measurement of serum TTR levels following a single intravenous infusion. Intellia is on track to dose its first patient by the end of 2020, subject to the impact of the COVID-19 pandemic, and is submitting additional regulatory applications in other countries as part of its ongoing, global development strategy. Once safety and an optimal dose have been determined in the first-in-human study, Intellia intends to further evaluate NTLA-2001 in a broader ATTR patient population of both polyneuropathy and cardiomyopathy patients. NTLA-2001 is part of a co-development/co-promotion agreement between Intellia, the lead development and commercialization party, and Regeneron Pharmaceuticals, Inc. (Regeneron).
- AML Program: NTLA-5001 is a wholly owned, T cell receptor (TCR)-T cell therapy development candidate targeting the Wilms’ Tumor 1 (WT1) antigen for the treatment of AML. The Company seeks to develop NTLA-5001 as a broadly applicable treatment for AML patients, regardless of the mutational subtypes of the cancer. Intellia continues to advance Investigational New Drug application (IND)-enabling activities and remains on track to submit an IND or IND-equivalent for NTLA-5001 in the first half of 2021. At the upcoming 62nd
American Society of Hematology(ASH) Annual Meeting, taking place virtually from December 5– 8, 2020, the Company will present new preclinical results in support of NTLA-5001, showing high anti-tumor activity of its lead WT1-directed TCR-T therapy in a mouse model of AML. The preclinical data will also highlight the advantages of its proprietary T cell engineering process to produce multiple, highly efficient sequential edits in T cells that have superior function and minimal translocations compared to results from standard T cell engineering approaches. Additional efforts are underway to evaluate the potential use of NTLA-5001 to treat WT1-positive solid tumors.
- HAE Program: NTLA-2002 is a wholly owned, in vivo development candidate for the treatment of HAE. Today, Intellia announced results from its completed non-human primate (NHP) study of its lead lipid nanoparticle (LNP) formulation for NTLA-2002. Following a single dose, the knockout of the prekallikrein B1 (KLKB1) gene resulted in a year-long therapeutically relevant reduction of serum kallikrein protein levels and activity. Building on Intellia’s modular LNP delivery system, NTLA-2002 is designed to knock out the KLKB1 gene in the liver after a single course of treatment. This approach is expected to prevent improperly regulated bradykinin production and therefore reduce HAE attacks. During the third quarter, the Company initiated Good Laboratory Practices (GLP) toxicology studies in preparation for an IND or IND-equivalent submission for NTLA-2002, which remains on track for the second half of 2021.
- Modular Platform: Intellia continues to make significant progress across its platform technologies, broadening the in vivo and ex vivo application of genome editing. This includes developing innovative CRISPR/Cas9-mediated targeted transgene insertion and allogeneic cell solutions. At the 16th Annual Meeting of the
Oligonucleotide Therapeutics Society, held September 27-30, 2020, Intellia presented new data highlighting the potential to develop single-course therapies that may have a lifelong effect for a variety of genetic diseases. The data showed the persistence of both in vivo knockout and insertion CRISPR/Cas9 edits and corresponding durability of effect following a partial hepatectomy (PHx) and liver regrowth in a murine model. Unlike traditional gene therapy, for which a significant loss (over 80%) in transgene expression was observed in the insertion PHx model, Intellia’s targeted gene insertion approach yielded durable edits, with no significant loss in expression in the same model. Intellia and Regeneron are co-developing potential hemophilia A and B CRISPR/Cas9-based treatments using their jointly developed insertion capabilities. Intellia is also continuing to develop its proprietary platform to advance its wholly-owned programs.
- Board of Directors: In
October 2020, Intellia appointed John F. Crowleyto its Board of Directors. Mr. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, is a well-established leader in biotech and pharmaceuticals and a visionary advocate for the advancement of treatments for people living with rare diseases.
- Scientific Co-Founder Awarded
NobelPrize: Jennifer Doudna, Ph.D., one of Intellia’s scientific co-founders, was awarded the 2020 Nobel Prize in Chemistry for inventing the revolutionary CRISPR/Cas9 genome editing technology. Dr. Doudnashares the award with her research collaborator, Dr. Emmanuelle Charpentier.
The Company will participate in the following events during the fourth quarter of 2020:
Credit Suisse Healthcare Conference, November 11, Virtual
- Barclays Gene Editing and Therapy Summit,
November 16, Virtual
- 62nd ASH Annual Meeting,
December 5-8, Virtual
The Company has set forth the following for pipeline progression:
- ATTR: Dose first patient in Phase 1 study by year-end
- AML: Submit an IND or IND-equivalent for NTLA-5001 in 1H 2021
- HAE: Submit an IND or IND-equivalent for NTLA-2002 in 2H 2021
Third Quarter 2020 Financial Results
- Cash Position: Cash, cash equivalents and marketable securities were
$407.9 millionas of September 30, 2020, compared to $284.5 millionas of December 31, 2019. The increase was driven by net proceeds of $107.7 millionfrom the June follow-on public offering, $100.0 millionupfront payment from the Regeneron collaboration expansion, which included a $30.0 millionequity investment, $14.7 millionof net equity proceeds raised from the Company’s “At the Market” (ATM) agreement, $18.2 millionof funding received under the Regeneron and Novartis collaborations and $2.7 millionin proceeds from employee-based stock plans. These increases were offset in part by cash used to fund operations of approximately $119.8 million.
- Collaboration Revenue: Collaboration revenue increased by
$11.6 millionto $22.2 millionduring the third quarter of 2020, compared to $10.6 millionduring the third quarter of 2019. The increase was mainly driven by a $15.3 millionamount recognized for the transfer of control of the license to develop the Factor VIII target for hemophilia A associated with the extension of the Regeneron collaboration.
- R&D Expenses: Research and development expenses increased by approximately
$12.2 millionto $39.8 millionduring the third quarter of 2020, compared to $27.5 millionduring the third quarter of 2019. This increase was primarily driven by the advancement of our lead programs, research personnel growth to support these programs, and the expansion of the development organization.
- G&A Expenses: General and administrative expenses increased by approximately
$2.1 millionto $10.6 millionduring the third quarter of 2020, compared to $8.4 millionduring the third quarter of 2019. This increase was primarily related to employee related expenses, including stock-based compensation, of $2.0 million.
- Net Loss: The Company’s net loss was
$27.8 millionfor the third quarter of 2020, compared to $23.6 millionduring the third quarter of 2019.
Intellia expects that its cash, cash equivalents and marketable securities as of
Conference Call to Discuss Third Quarter 2020 Earnings
The Company will discuss these results on a conference call today,
To join the call:
U.S.callers should dial 1-877-317-6789 and international callers should dial 1-412-317-6789, approximately five minutes before the call.
- All participants should ask to be connected to the
Intellia Therapeuticsconference call.
A replay of the call will be available through the Events and Presentations page of the Investors & Media section on Intellia’s website at www.intelliatx.com, 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 its intellectual property position; risks related to Intellia’s relationship with third parties, including its licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; uncertainties related to regulatory agencies’ evaluation of regulatory filings and other information related to its product candidates; uncertainties related to the authorization, initiation and conduct of studies and other development requirements for its product candidates; the risk that any one or more of Intellia’s product candidates, including those that are co-developed, will not be successfully developed and commercialized; the risk that the results of preclinical studies or clinical 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 as well as discussions of potential risks, uncertainties, and other important factors in Intellia’s other filings with the
|CONSOLIDATED STATEMENTS OF OPERATIONS (UNAUDITED)|
|(Amounts in thousands, except per share data)|
|Three Months Ended
||Nine Months Ended
|Research and development||39,756||27,513||112,177||76,682|
|General and administrative||10,566||8,431||33,406||32,082|
|Total operating expenses||50,322||35,944||145,583||108,764|
|Net loss per share, basic and diluted||$||(0.47||)||$||(0.49||)||$||(1.70||)||$||(1.53||)|
|Weighted average shares outstanding, basic and diluted||58,754||48,554||54,218||46,547|
|CONSOLIDATED BALANCE SHEET DATA (UNAUDITED)|
|(Amounts in thousands)|
|Cash, cash equivalents and marketable securities||$||407,947||$||284,472|
|Total stockholders' equity||329,620||269,881|
Senior Vice President
Source: Intellia Therapeutics, Inc.