Intellia Therapeutics Announces Second Quarter 2020 Financial Results
- Submitted first regulatory application to the U.K.’s MHRA to initiate a Phase 1 study of NTLA-2001 for the treatment of transthyretin amyloidosis; on track to dose first patient by year-end
- Expanded Regeneron collaboration, receiving
$100 millionthrough upfront cash and equity investment and agreeing to co-develop treatments for hemophilia A and B using CRISPR-mediated targeted transgene insertion technology
- On track to submit an IND or IND-equivalent in 2021 for each of two wholly owned programs: NTLA-5001 for the treatment of acute myeloid leukemia and NTLA-2002 for the treatment of hereditary angioedema
- Ended quarter with strong cash position of
“We are very pleased to announce our first regulatory submission to initiate a Phase 1 study of NTLA-2001, which is an important milestone as we deliver on our mission to develop curative, CRISPR/Cas9-based treatments for severe diseases. Our CTA keeps us on track to dose our first ATTR patient by year-end and is a critical step in executing our global development strategy for the first systemically delivered CRISPR-based therapy to enter clinical trial,” said Intellia President and Chief Executive Officer,
Second Quarter 2020 and Recent Operational Highlights
- ATTR Program: Intellia announced today it has submitted its first Clinical Trial Application (CTA) to the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a Phase 1 study of NTLA-2001 for the treatment of transthyretin amyloidosis (ATTR). Based on the Company’s in vivo liver knockout approach, NTLA-2001 is a potential single-course therapy for patients living with ATTR. Pending CTA authorization and subject to the impact of COVID-19, the Company is on track to dose its first patient by the end of 2020 with the first systemically delivered CRISPR/Cas9-based therapy to enter the clinic. The Company is submitting additional regulatory applications to enable enrollment in other countries as part of its global development strategy. 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 acute myeloid leukemia (AML). The Company’s approach aims to develop a broadly applicable treatment for AML patients, regardless of the mutational subtypes of the cancer. At the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, held from
May 12-15, 2020, Intellia presented data on its proprietary T cell engineering process in support of NTLA-5001. The data showed that the Company’s proprietary process enables multiple, highly efficient, sequential edits in T cells, whether knocking out or inserting genes. This technology yields engineered cells with high anti-tumor activity and favorable attributes, including a desired memory phenotype, which is associated with longer-lasting treatment effects. Importantly, chromosomal translocations (i.e., undesired chromosomal rearrangements) were similar to background levels in untreated cells. 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. Additional efforts are underway to evaluate potential use of the WT1-targeted TCR construct to treat solid tumors.
- HAE Program: NTLA-2002 is a wholly owned, in vivo development candidate for the treatment of hereditary angioedema (HAE). Building on Intellia’s modular lipid nanoparticle (LNP) delivery system, NTLA-2002 is designed to knock out the prekallikrein B1 (KLKB1) gene in the liver after a single course of treatment, which is expected to prevent improperly regulated bradykinin production and therefore, reduce HAE attacks. In a non-human primate (NHP) study of its lead LNP formulation for NTLA-2002, the knockout of KLKB1 resulted in a therapeutically relevant reduction of serum kallikrein levels and activity following a single dose. Consistent with the durability achieved in earlier NHP studies for its lead in vivo program, the Company has now demonstrated sustained kallikrein activity reduction for 10 months in an ongoing study. Based on these results, Intellia believes NTLA-2002 could be efficacious and durable in preventing HAE attacks following a single course of treatment. Intellia continues to progress IND-enabling activities and is on track to submit an IND or IND-equivalent for NTLA-2002 in 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. Additionally, to supplement its ex vivo capabilities, the Company established research collaborations and license agreements with
TeneoBio and GEMoaB GmbHto develop engineered cell therapies for immuno-oncology and autoimmune diseases. Intellia’s broad platform innovations and expanded capabilities will drive the next wave of in vivo and ex vivo clinical candidates.
- Regeneron Collaboration: In
June 2020, Intellia and Regeneron announced an expansion of their existing collaboration to co-develop potential products for the treatment of hemophilia A and B. The collaboration expansion builds upon the jointly-developed targeted transgene insertion capabilities designed to durably restore a missing protein and to overcome the limitations of traditional gene therapy. The collaboration also provides Regeneron with rights to develop products for additional in vivo CRISPR/Cas9-based therapeutic liver targets. Under the expansion, the term of the collaboration has been extended until April 2024, at which point Regeneron has an option to renew for an additional two years. In addition, the expansion grants Regeneron a royalty-bearing, non-exclusive license to certain Intellia intellectual property to develop and commercialize up to 10 ex vivo CRISPR/Cas9 products in defined cell types. Intellia received $100 millionthrough an upfront payment of $70 millionand an additional $30 millionequity investment from Regeneron at $32.42per share.
- Financing: In
June 2020, Intellia closed an underwritten public offering of 6,301,370 shares of common stock, including the exercise in full of the underwriters’ option to purchase additional shares, at the public offering price of $18.25per share. Intellia received aggregate gross proceeds of approximately $115 million, before underwriting discounts and commissions and offering expenses.
The Company will participate in the following events during the third quarter of 2020:
Wedbush PacGrow Healthcare Conference, August 11, Virtual Baird Healthcare Conference, September 9, Virtual
The Company has set forth the following for pipeline progression:
- ATTR: Plan to dose first patient by end of 2020
- 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
Second Quarter 2020 Financial Results
- Cash Position: Cash, cash equivalents and marketable securities were
$436.8 millionas of June 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, $14.4 millionof funding received under existing collaborations with Regeneron and Novartis and $2.1 millionin proceeds from employee-based stock plans. These increases were offset in part by cash used to fund operations of approximately $86.5 million.
- Collaboration Revenue: Collaboration revenue increased by
$5.1 millionto $16.3 millionduring the second quarter of 2020, compared to $11.1 millionduring the second quarter of 2019. The increase in collaboration revenue in 2020 was mainly driven by an $8.4 millionone-time cumulative catch-up adjustment related to the extension of the Regeneron collaboration.
- R&D Expenses: Research and development expenses increased by
$12.3 millionto $37.8 millionduring the second quarter of 2020, compared to $25.5 millionduring the second quarter of 2019. This increase was primarily driven by IND-enabling activities related to our lead programs, research personnel growth to support these programs, and the expansion of the development organization.
- G&A Expenses: General and administrative expenses decreased by
$1.6 millionto $11.5 millionduring the second quarter of 2020, compared to $13.1 millionduring the second quarter of 2019. This decrease was primarily driven by a decrease in legal expenses, which were principally related to a decrease in certain activities related to intellectual property matters.
- Net Loss: The Company’s net loss was
$32.4 millionfor the second quarter of 2020, compared to $25.7 millionduring the second quarter of 2019.
Intellia expects that its cash, cash equivalents and marketable securities as of
Conference Call to Discuss Second 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 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 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
||Six Months Ended
|Research and development||37,771||25,460||72,421||49,169|
|General and administrative||11,526||13,118||22,840||23,651|
|Total operating expenses||49,297||38,578||95,261||72,820|
|Net loss per share, basic and diluted||$||(0.61||)||$||(0.56||)||$||(1.24||)||$||(1.05||)|
|Weighted average shares outstanding, basic and diluted||53,369||45,814||51,938||45,526|
|CONSOLIDATED BALANCE SHEET DATA (UNAUDITED)|
|(Amounts in thousands)|
|Cash, cash equivalents and marketable securities||$||436,803||$||284,472|
|Total stockholders' equity||351,587||269,881|
Senior Vice President
Source: Intellia Therapeutics, Inc.