Intellia Therapeutics Announces Fourth Quarter and Full-Year 2020 Financial Results
- Expects to report initial data from Phase 1 study of NTLA-2001, a potentially curative single-course therapy for transthyretin amyloidosis (ATTR), in 2021
- On track to submit an IND or IND-equivalent in mid-2021 for NTLA-5001 for the treatment of acute myeloid leukemia (AML)
- Intends to submit an IND or IND-equivalent in 2H 2021 for NTLA-2002 for the treatment of hereditary angioedema (HAE)
- Plans to nominate at least one new development candidate in 2021 from broad research efforts and continued platform innovation
- Ended 2020 with strong cash position of
“Intellia’s achievements in 2020 reflect important progress on both our full-spectrum strategy and our mission to deliver curative genome editing treatments for people with severe diseases. Dosing our first patient with NTLA-2001, the first-ever systemically delivered CRISPR-based therapy, was a major milestone for our team, as we completed our transition to a clinical-stage company in the fourth quarter,” said Intellia President and Chief Executive Officer,
Fourth Quarter 2020 and Recent Operational Highlights
- NTLA-2001 for ATTR: NTLA-2001 is the first systemically delivered CRISPR-based therapy dosed in a patient and could potentially be the first curative treatment for ATTR. By applying the Company’s in vivo lipid nanoparticle (LNP) delivery technology, NTLA-2001 offers the possibility of halting and reversing the disease with potent, lifelong transthyretin (TTR) protein reduction after a single course of treatment. NTLA-2001 is part of a co-development/co-promotion agreement between Intellia, the lead party, and Regeneron Pharmaceuticals, Inc. (Regeneron).
- The Company anticipates reporting interim clinical data from the Phase 1 study this year. These results are expected to characterize the emerging safety and activity profile of NTLA-2001 at the initial dose levels.
- In November, Intellia dosed the first patient in its global Phase 1 study evaluating NTLA-2001 in adults with hereditary ATTR with polyneuropathy (hATTR-PN). The Company continues to enroll patients in the study and is submitting additional regulatory applications in other countries as part of its ongoing, global development strategy.
- Intellia intends to evaluate NTLA-2001 in a broader ATTR population of both polyneuropathy and cardiomyopathy patients following its Phase 1 safety assessment and dose optimization.
- NTLA-5001 for AML: NTLA-5001 is a potential best-in-class engineered T cell therapy designed to treat all genetic subtypes of AML. This investigational candidate is an autologous T cell receptor (TCR)-T cell therapy targeting the Wilms’ Tumor 1 (WT1) antigen utilizing Intellia’s proprietary cell engineering process.
- Intellia plans to submit an Investigational New Drug (IND) application or equivalent regulatory application for NTLA-5001 in mid-2021. This first-in-human trial is expected to evaluate the safety and activity of NTLA-5001 in patients with persistent or recurrent AML who have previously received first-line therapies.
- NTLA-5001 shows high anti-tumor activity in proof-of-concept mouse models of acute leukemias. The preclinical data presented at the
American Society of Hematology(ASH) Annual Meeting in December highlighted faster expansion and superior function of T cells manufactured by Intellia’s proprietary approach, compared to standard T cell engineering approaches currently in use.
- The Company is also evaluating the potential use of NTLA-5001 to treat WT1-positive solid tumors in preclinical studies.
- NTLA-2002 for HAE: NTLA-2002 aims to prevent attacks for people living with HAE after a single course of treatment. Intellia is applying its modular LNP delivery system to develop NTLA-2002 to knock out the KLKB1 gene in the liver to permanently reduce plasma kallikrein activity. This approach is expected to provide continuous suppression of kallikrein activity and eliminate the significant treatment burden associated with currently available therapies for HAE patients.
- Intellia commenced clinical manufacturing activities to support the Company’s plans to submit an IND or equivalent regulatory application in the second half of 2021.
- The Company is applying insights gained from NTLA-2001 to expedite clinical development of NTLA-2002. The first-in-human trial is expected to evaluate safety, tolerability and activity in patients with HAE.
- The Company plans to present additional preclinical results in support of NTLA-2002 at the upcoming
American Academy of Allergy, Asthma & Immunology (AAAAI) 2021 Annual Meeting, taking place virtually from February 26– March 1, 2021.
- Modular Platform and Pipeline Expansion: Intellia is advancing its modular platform technologies to broaden the in vivo and ex vivo applications of genome editing. This includes developing capabilities for innovative CRISPR/Cas9-mediated in vivo editing in multiple tissue types, targeted transgene insertion and an allogeneic approach for the development of “off-the-shelf” T cell therapies. These efforts will support new therapeutic candidates for genetic diseases requiring removal and/or restoration of a protein, and next-generation engineered cell therapies for cancers and auto-immune diseases.
- Intellia plans to nominate at least one additional development candidate in 2021 and expects to present preclinical data at upcoming scientific conferences highlighting research advancements and platform innovations.
- Intellia demonstrated the modularity of its targeted insertion approach for a second target, in non-human primates, showing insertion of the SERPINA1 gene produced normal levels of human alpha-1 antitrypsin (AAT) after a single administration. In December, these results were presented at the Alpha-1 Foundation’s 20th
Gordon L. Snider Critical Issues Workshop: The Promise of Gene-Based Interventions of Alpha-1 Antitrypsin Deficiency. Intellia is advancing multiple genome editing strategies that may treat both lung and liver manifestations of AAT deficiency (AATD), which occur due to mutations in the SERPINA1 gene.
- Intellia is advancing preclinical validation of in vivo hematopoietic stem cell (HSC) genome editing using the Company’s proprietary non-viral delivery systems and CRISPR/Cas9 technology to potentially cure sickle cell disease. This research is supported by a grant from the
Bill & Melinda Gates Foundation.
- Financing: In December, Intellia closed an underwritten public offering of 5,513,699 shares of common stock, including the exercise in full of the underwriters’ option to purchase additional shares, at the public offering price of
$36.50per share. Intellia received aggregate gross proceeds of approximately $201 million, before underwriting discounts and commissions and offering expenses.
The Company will participate in the following events during the first quarter of 2021:
- AAAAI Annual Meeting, February 26–March 1, Virtual
- Keystone Symposium:
Precision Engineeringof the Genome, Epigenome and Transcriptome, March 8-10, Virtual Barclays Capital Global Healthcare Conference, March 8, Virtual
- Oppenheimer's 31st Annual Healthcare Conference,
March 16, Virtual Cold Spring Harbor Laboratoriesmeeting on Nucleic Acid Therapies, March 24 - 26, Virtual
The Company has set forth the following for pipeline progression:
- ATTR: Report initial clinical data from Phase 1 study of NTLA-2001 in 2021
- AML: Submit an IND or IND-equivalent for NTLA-5001 in mid-2021
- HAE: Submit an IND or IND-equivalent for NTLA-2002 in 2H 2021
- Nominate at least one new development candidate in 2021
Fourth Quarter and Full-Year 2020 Financial Results
- Cash Position: Cash, cash equivalents and marketable securities were
$597.4 millionas of December 31, 2020, compared to $284.5 millionas of December 31, 2019. The increase was driven by net proceeds of $296.6 millionfrom our follow-on public offerings, $100.0 millionupfront payment from the Regeneron collaboration expansion, which included a $30.0 millionequity investment, $49.5 millionof net equity proceeds raised from the Company’s “At the Market” (ATM) agreement, $18.2 millionfrom the Regeneronand Novartis Institutes for BioMedical Research, Inc.(Novartis) collaborations and $13.2 millionin proceeds from employee-based stock plans. These increases were offset in part by cash used to fund operations of approximately $164.6 million.
- Collaboration Revenue: Collaboration revenue decreased by
$4.3 millionto $6.6 millionduring the fourth quarter of 2020, compared to $10.9 millionduring the fourth quarter of 2019. The decrease was primarily driven by a decrease in Novartis revenue as the research portion of the collaboration ended in December 2019.
- R&D Expenses: Research and development expenses increased by
$6.5 millionto $38.2 millionduring the fourth quarter of 2020, compared to $31.7 millionduring the fourth quarter of 2019. This increase was primarily driven by the advancement of our lead programs, research personnel growth to support these programs and expansion of the development organization.
- G&A Expenses: General and administrative expenses increased by
$1.8 millionto $10.8 millionduring the fourth quarter of 2020, compared to $9.0 millionduring the fourth quarter of 2019. This increase was primarily related to employee related expenses, including stock-based compensation of $1.3 million.
- Net Loss: The Company’s net loss was
$42.2 millionfor the fourth quarter of 2020, compared to $28.3 millionduring the fourth quarter of 2019.
Intellia expects that its cash, cash equivalents and marketable securities as of
Conference Call to Discuss Fourth Quarter and Full-Year 2020 Earnings
The Company will discuss these results on a conference call today,
To join the call:
U.S.callers should dial 1-833-316-0545 and international callers should dial 1-412-317-5726, 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
Intellia Therapeutics is a leading clinical-stage genome editing company, focused on the development of proprietary, potentially curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by both producing therapeutics that permanently edit and/or correct disease-associated genes in the human body with a single treatment course, and creating enhanced engineered cells that can treat oncological and immunological diseases. Intellia’s combination of deep scientific, technical and clinical development experience, along with its leading intellectual property portfolio, puts it in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create new classes of therapeutic products. Learn more about Intellia and CRISPR/Cas9 at intelliatx.com. Follow us on Twitter @intelliatweets.
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 our ability to protect and maintain our intellectual property position; risks related to our relationship with third parties, including our licensors and licensees; risks related to the ability of our licensors to protect and maintain their intellectual property position; uncertainties related to regulatory agencies’ evaluation of regulatory filings and other information related to our product candidates; uncertainties related to the authorization, initiation and conduct of studies and other development requirements for our product candidates; the risk that any one or more of our 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 our collaborations with Regeneron or our 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
||Twelve Months Ended
|Research and development||38,231||31,731||150,408||108,413|
|General and administrative||10,763||8,976||44,169||41,058|
|Total operating expenses||48,994||40,707||194,577||149,471|
|Net loss per share, basic and diluted||$||(0.69||)||$||(0.57||)||$||(2.40||)||$||(2.11||)|
|Weighted average shares outstanding, basic and diluted||61,306||49,350||55,987||47,247|
|CONSOLIDATED BALANCE SHEET DATA (UNAUDITED)|
|(Amounts in thousands)|
|Cash, cash equivalents and marketable securities||$||597,371||$||284,472|
|Total stockholders' equity||527,072||269,881|
Chief Financial Officer
Interim Head of
Source: Intellia Therapeutics, Inc.