On August 1, 2019 Intellia Therapeutics, Inc. (NASDAQ:NTLA), reported operational highlights and financial results for the second quarter ended June 30, 2019 (Press release, Intellia Therapeutics, AUG 1, 2019, View Source [SID1234538013]).

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"We continue to build our in vivo and engineered cell therapy pipeline in support of our mission to develop curative genome editing therapies for patients," said Intellia President and Chief Executive Officer John Leonard, M.D. "With IND-enabling toxicology studies underway for NTLA-2001, we are well-positioned to submit an IND in mid-2020 for the first systemically delivered CRISPR/Cas9-based therapy for transthyretin amyloidosis. Additionally, by the end of this year, we plan to select our first engineered cell therapy development candidate to treat acute myeloid leukemia."

Second Quarter 2019 and More Recent Operational Highlights

ATTR Program: Intellia’s first in vivo development candidate, NTLA-2001, is for the treatment of transthyretin amyloidosis (ATTR). As part of an ongoing durability study of the Company’s lead lipid nanoparticle (LNP) formulation in support of NTLA-2001, we have demonstrated six months of durable liver editing with sustained reduction of circulating transthyretin (TTR) protein (average reduction >95%) following a single dose in non-human primates (NHPs). The Company announced today it has conducted its pre-investigational new drug (IND) meeting with the U.S. Food and Drug Administration (FDA), has initiated IND-enabling toxicology studies and expects to submit an IND application for NTLA-2001 in mid-2020. Additionally, the Company’s supply chain operations are in place to support manufacturing of Phase 1 materials. NTLA-2001 is being co-developed with Regeneron Pharmaceuticals, Inc. (Regeneron), with Intellia as the lead development and commercialization party.
AML Program: Intellia’s lead T cell receptor (TCR)-based therapy uses engineered T cells to target Wilms’ Tumor 1 (WT1) for the treatment of acute myeloid leukemia (AML). During the second quarter of 2019, the Company and its research collaborators at IRCCS Ospedale San Raffaele initiated functional testing of multiple lead TCRs in patient-derived xenograft models and remain on track to nominate a development candidate by the end of 2019. In parallel, Intellia has begun establishing manufacturing capabilities to support clinical evaluation.

In Vivo Insertion in NHPs: As previously disclosed, Intellia demonstrated the first CRISPR/Cas9-mediated, targeted transgene insertion in the liver of NHPs, using Factor 9 (F9) as a model gene. F9 is a gene that encodes for the Factor IX (FIX) protein, a blood-clotting protein that is missing or defective in hemophilia B patients. The study is a collaborative effort between Intellia and Regeneron, using Intellia’s LNP delivery system of CRISPR/Cas9 with an adeno-associated virus (AAV) containing a proprietary bi-directional insertion template. Following a single dose of the hybrid LNP-AAV delivery system containing an F9 DNA template, the Company demonstrated that the circulating human FIX protein levels achieved at day 14 were durable through the two months of completed observation and were within the normal human range (source: Amiral et al, Clin. Chem., 1984). Additionally, the NHP data expands on the durability of clinically relevant human FIX protein levels achieved in mice for over 12 months. Intellia is currently working closely with Regeneron on moving the program forward and is also independently evaluating the hybrid LNP-AAV delivery system for targeted insertion across several other transgenes of interest in an in vivo setting.

Expanded Management Team: In May, Intellia announced the appointment of Laura Sepp-Lorenzino, Ph.D., as executive vice president and chief scientific officer. Dr. Sepp-Lorenzino brings decades of leadership and research and development experience and leads Intellia’s drug research organization.

Upcoming Milestones

The Company has set forth the following for pipeline progression:

ATTR:
° Commence manufacturing of NTLA-2001 Phase 1 materials by the end of 2019
° Submit IND application for NTLA-2001 in mid-2020
AML:
° Nominate first engineered cell therapy development candidate by the end of 2019
Platform:
° Present additional in vivo and engineered cell therapy data at upcoming scientific conferences by the end of 2019

Second Quarter 2019 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $275.8 million as of June 30, 2019, compared to $314.1 million as of December 31, 2018. The decrease was driven by cash used to fund operations of $59.8 million, which was offset in part by $7.9 million of net equity proceeds raised from the Company’s At The Market ("ATM") offerings, $7.0 million of funding received under the Novartis collaboration, $4.1 million of ATTR cost reimbursements made by Regeneron, and $2.6 million in proceeds from employee-based stock plans.
ATM Proceeds Due: The Company has $27.1 million in current assets on the balance sheet as of June 30, 2019 related to proceeds due from the ATM offerings that were received in the first week of July 2019.
Collaboration Revenue: Collaboration revenue increased by $3.4 million to $11.1 million during the second quarter of 2019, compared to $7.7 million during the second quarter of 2018. The increase in collaboration revenue in 2019 was primarily driven by amounts recognized from the expansion of the existing collaboration with Novartis, as well as by amounts recognized under the Company’s ATTR Co/Co agreement with Regeneron. As previously disclosed, Regeneron is obligated to fund approximately 50% of the development costs for the ATTR program.
R&D Expenses: Research and development expenses increased by $2.0 million to $25.5 million during the second quarter of 2019, compared to $23.5 million during the second quarter of 2018. This increase was primarily driven by the advancement of Intellia’s pipeline and the expansion of the research and development organization.
G&A Expenses: General and administrative expenses increased by $5.3 million to $13.1 million during the second quarter of 2019, compared to $7.8 million during the second quarter of 2018. This increase was primarily driven by personnel-related costs to support Intellia’s larger research and development organization, and an increase in legal and intellectual property (IP) costs.
Net Loss: The Company’s net loss was $25.7 million for the second quarter of 2019, compared to $22.2 million during the second quarter of 2018.
Financial Guidance

Intellia expects that its cash, cash equivalents, marketable securities and proceeds due from the ATM offerings as of June 30, 2019, as well as technology access and funding from Novartis and Regeneron, will enable Intellia to fund its anticipated operating expenses and capital expenditure requirements into the second half of 2021. This expectation excludes any potential milestone payments or extension fees that could be earned and distributed under the collaboration agreements with Novartis and Regeneron or any strategic use of capital not currently in the Company’s base-case planning assumptions.

Conference Call to Discuss Second Quarter 2019 Earnings

The Company will discuss these results on a conference call today, August 1, 2019, at 8 a.m. ET.

To join the call:

U.S. callers should dial 866-575-6539 and use conference ID# 6223506, approximately five minutes before the call.
International callers should dial +1 856-344-9299 and use conference ID# 6223506, approximately five minutes before the call.
A replay of the call will be available through the Events and Presentations page of the Investor Relations section of the Company’s website at www.intelliatx.com, beginning on August 1, 2019 at 12 p.m. ET.