On May 7, 2020 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, reported operational highlights and financial results for the first quarter ended March 31, 2020 (Press release, Intellia Therapeutics, MAY 7, 2020, View Source [SID1234557316]).
Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:
Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing
Schedule Your 30 min Free Demo!
"At Intellia, our mission is to develop curative, CRISPR/Cas9-based treatments for severe diseases. I am grateful to our team for remaining steadfast in our commitment to patients and putting us in the position to deliver on our goals despite the challenging circumstances presented by the global COVID-19 pandemic," said Intellia President and Chief Executive Officer John Leonard, M.D. "We look forward to advancing NTLA-2001 into clinical testing and dosing ATTR patients with the first systemically delivered CRISPR/Cas9-based therapy in the second half of 2020. Additionally, we continue to advance our full-spectrum pipeline, including IND-enabling activities for NTLA-5001, our engineered TCR-T cell therapy candidate for AML, and the nomination of NTLA-2002 as our development candidate for the treatment of HAE."
First Quarter 2020 and Recent Operational Highlights
ATTR Program: Intellia’s lead candidate, NTLA-2001, is designed to apply its in vivo liver knockout approach for the treatment of transthyretin amyloidosis (ATTR) in a single course of treatment. The Company has manufactured clinical-scale materials for a Phase 1 study of NTLA-2001 and remains on track to submit an Investigational New Drug application (IND) or IND-equivalent for this program in mid-2020. NTLA-2001 is anticipated to be the first systemically delivered CRISPR/Cas9 therapy to enter the clinic. Subject to the impact of COVID-19, the Company plans to dose the first patient in the second half of 2020. NTLA-2001 is part of a co-development/co-promotion (Co/Co) agreement between Intellia, which is the lead development and commercialization party, and Regeneron Pharmaceuticals, Inc. (Regeneron).
AML Program: NTLA-5001 for the treatment of acute myeloid leukemia (AML) is Intellia’s first T cell receptor (TCR)-T cell development candidate, which targets the Wilms’ Tumor 1 (WT1) antigen. The Company’s approach aims to develop a broadly applicable treatment for AML patients, regardless of mutational subtypes of a patient’s leukemia. As presented at Keystone Symposia’s Engineering the Genome Conference in February 2020, Intellia demonstrated that its CRISPR/Cas9-enabled approach results in an engineered T cell product capable of specific and potent killing of primary AML blasts. Intellia is continuing to advance IND-enabling activities, including process development to support clinical T cell manufacturing. The Company is on track to submit an IND or IND-equivalent for NTLA-5001 in the first half of 2021. Additional efforts are underway to evaluate the potential use of the WT1-targeted TCR construct to treat solid tumors.
HAE Program: Intellia has nominated its third development candidate, NTLA-2002 for the treatment of hereditary angioedema (HAE). Building on the modular lipid nanoparticle (LNP) delivery system developed for the ATTR program, NTLA-2002 is designed to knock out the prekallikrein B1 (KLKB1) gene in the liver after a single course of treatment. As part of an ongoing durability study of its lead LNP formulation in support of NTLA-2002, Intellia has now demonstrated six months of sustained therapeutically relevant reduction of serum kallikrein levels and activity following a single dose in non-human primates. Intellia expects to submit an IND or IND-equivalent for NTLA-2002 in the second half of 2021. NTLA-2002 is subject to an option by Regeneron to enter into a Co/Co agreement, which must be exercised within a limited time period after development candidate selection. Intellia would be the lead party if the option is exercised.
Modular Platform: Intellia continues to advance its differentiated genome editing and delivery strategies, including targeted insertion and consecutive editing, across its in vivo and ex vivo efforts. These platform capabilities enable the removal and/or restoration of a gene’s function in developing treatments for life-threatening diseases. The in vivo research builds on data previously shared by the Company demonstrating the first CRISPR-mediated, targeted transgene insertion in the liver of non-human primates, which generated circulating human FIX protein at or above normal levels. At the upcoming American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting, taking place virtually from May 12 – 15, 2020, the Company will present preclinical data related to certain programs and ex vivo platform capabilities. This will include an oral presentation featuring Intellia’s enhanced CRISPR/Cas9-based T cell engineering platform capable of highly efficient editing of multiple genes with levels of translocations indistinguishable from background levels, and favorable cell product attributes including high viability and expansion potential. This improved T cell engineering process has been incorporated into the NTLA-5001 program.
Novartis Collaboration: As announced in March 2020, the U.S. Food and Drug Administration (FDA) has accepted the IND application submitted by Novartis Institutes for BioMedical Research (Novartis), for a CRISPR/Cas9-based engineered cell therapy for the treatment of sickle cell disease (SCD) developed under collaboration with Intellia. The Phase 1/2 clinical trial will begin investigating OTQ923 in adult patients with severe complications of SCD. Novartis’ IND application triggered a milestone payment to Intellia, and the Company is eligible to receive additional downstream success-based milestones and royalties.
Upcoming Events
The Company will participate in the following events during the second quarter of 2020:
ASGCT 23rd Annual Meeting, May 12 – 15, Virtual
Jefferies Global Healthcare Conference, June 3, Virtual
Upcoming Milestones
The Company has set forth the following for pipeline progression:
ATTR:
Submit an IND or IND-equivalent for NTLA-2001 in mid-2020
Subject to the impact of COVID-19, plan to dose first patient in 2H 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
Modular Platform Advancements:
Present preclinical data at upcoming ASGCT (Free ASGCT Whitepaper) Annual Meeting
First Quarter 2020 Financial Results
Cash Position: Cash, cash equivalents and marketable securities were $250.3 million as of March 31, 2020, compared to $284.5 million as of December 31, 2019. The decrease was driven by cash used to fund operations of approximately $40.1 million, which was offset in part by $4.5 million of net equity proceeds raised from the Company’s "At the Market" (ATM) agreement, $1.0 million of funding received under the Novartis collaboration, and $0.3 million in proceeds from employee-based stock plans.
Collaboration Revenue: Collaboration revenue increased by $2.5 million to $12.9 million during the first quarter of 2020, compared to $10.4 million during the first quarter of 2019. The increase in collaboration revenue in 2020 was mainly driven by the $5.0 million milestone payment earned from Novartis for the IND submission of OTQ923.
R&D Expenses: Research and development expenses increased by approximately $10.9 million to $34.7 million during the first quarter of 2020, compared to $23.7 million during the first quarter of 2019. This increase was primarily driven by the advancement of our lead programs, research personnel growth to support these programs, as well as the expansion of the development organization.
G&A Expenses: General and administrative expenses increased by $0.8 million to $11.3 million during the first quarter of 2020, compared to $10.5 million during the first quarter of 2019. This increase was primarily driven by an increase in intellectual property related expenses due to increased patent-related activity.
Net Loss: The Company’s net loss was $31.8 million for the first quarter of 2020, compared to $21.9 million during the first quarter of 2019.
Financial Guidance
Intellia expects that its cash, cash equivalents and marketable securities as of March 31, 2020 will enable the Company to fund its anticipated operating expenses and capital expenditure requirements at least through the end of 2021. This expectation excludes any strategic use of capital not currently in the Company’s base-case planning assumptions.
Conference Call to Discuss First Quarter 2020 Earnings
The Company will discuss these results on a conference call today, May 7, 2020, at 8 a.m. ET.
To join the call:
U.S. callers should dial 1-877-317-6789 and use conference ID# 10141541, approximately five minutes before the call.
International callers should dial 1-412-317-6789 and use conference ID# 10141541, approximately five minutes before the 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 May 7, 2020 at 12 p.m. ET.