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Agenus First Quarter Results and Update

On May 7, 2020 Agenus Inc. (NASDAQ: AGEN), an immuno-oncology company with an extensive pipeline of agents designed to activate immune response to cancers provided a corporate update and reported financial results for the first quarter of 2020 (Press release, Agenus, MAY 7, 2020, View Source [SID1234557320]).

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"We accelerated the development of AGEN1181, advanced our plans to file our BLAs and filed two INDs for our allogeneic iNKT cell therapy to treat cancer and COVID-19," said Garo Armen, Ph.D., Chairman and CEO, Agenus. "Our ability to rapidly enroll in our bali and zali trials, in just 2 years, underscores the speed with which we can advance our AGEN1181 +/- bali and our innovative pipeline of products including allogeneic cell therapy programs."

AGEN1181, reveals clinical benefit1 in >70% of response-evaluable patients
Early data suggest that AGEN1181 could be a breakthrough in IO treatment:
Confirmed CR with AGEN1181 (1 mg/kg) in advanced endometrial cancer with a poor prognosis
Confirmed PR (significant tumor reduction) in advanced endometrial cancer with low-dose AGEN1181 + balstilimab
Disease stabilization in the majority of response-evaluable patients
Advisory board endorsed accelerated plans for AGEN1181 development

Balstilimab & zalifrelimab demonstrate ~26% response rates which are durable in an all-comer, non-biomarker selected population
Large cohort analysis (n=55) bali + zali demonstrate ~26% response rates (4 CRs, 10 PRs) in refractory cervical cancer
Responses are durable over 12 months

Allogeneic iNKTs advancing to clinic for the treatment of cancer and COVID-19
COVID-19: iNKTs designed to eliminate COVID-19 virus, dampen harmful inflammation, and promote protection from reinfection (FIM 1H2020)
CANCER: iNKTs designed to promote anti-tumor immunity in cancer & enable optimal combinations with Agenus checkpoint antibodies

Potential best and first in class TIGIT programs advancing to IND filing starting in 4Q2020
Fc enhanced TIGIT antibody (AGEN1327) has outperformed all tested competitor antibodies with superior T cell activation in PD-1 or LAG-3 combos
TIGIT bispecific (AGEN1777) demonstrated potent tumor killing in a difficult to treat colon cancer model where PD-1 antibodies alone are ineffective

Key Upcoming Milestones Expected
BLA filings and pre-commercial activities
IND filings for (2) TIGIT programs in 4Q2020 & 1H2021
Ongoing read-outs from lead clinical trials (AGEN1181, Balstilimab, Zalifrelimab)
Deliver new partnerships
Reduce annualized burn by $50M to mitigate any pandemic related business risk
First Quarter Financial Results

We ended the first quarter of 2020 with a cash balance of $92.3 million as compared to $61.8 million at December 31, 2019.

For the first quarter ended March 31, 2020, we reported a cash burn from our operations of $32 million. Net loss for the quarter was $45 million or $0.31 per share which included non-cash expenses of $16 million. We generated net income for the same period in 2019 of $17 million or $0.14 per share. In the first quarter of 2019 we recognized revenue of $80 million which included revenue related to the upfront license fee from our transaction with Gilead in addition to non-cash royalties earned. For the same period in 2020 we recorded revenue of $15 million primarily related to non-cash royalties earned.

To access the live call, dial 1-844-492-3727 (U.S.) or 1-412-317-5118 (International) and ask to be joined into the Agenus call. The call will also be webcast and will be accessible from the Company’s website at View Source or with this link View Source

A replay will be available on the Company’s website approximately one hour after the call and will remain available until July 7, 2020.

Lineage Cell Therapeutics Announces Early Exercise of Option with Cancer Research UK for Immuno-Oncology Cell Therapy Program

On May 7, 2020 Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs, reported that Cancer Research UK’s Commercial Partnerships has permitted Lineage to conduct an early exercise of its option to acquire data from Cancer Research UK’s ongoing Phase 1 clinical trial of VAC2 in non-small cell lung cancer (NCT03371485), and develop an allogeneic dendritic cell therapy platform ("VAC") (Press release, Lineage Cell Therapeutics, MAY 7, 2020, View Source [SID1234557319]). Lineage will assume responsibility for further development of the VAC2 product candidate as well as future development opportunities derived from the VAC platform, while Cancer Research UK concludes the ongoing trial. This decision was based on an early review of the data collected by Cancer Research UK in the VAC2 trial under a clinical trial and option agreement. In addition, Cancer Research UK will provide input on the potential use of VAC in the infectious disease space to develop a vaccine against SARS-CoV-2, the virus which causes COVID-19. Cancer Research UK is the world’s largest cancer charity dedicated to saving lives through research.

"Clinical data recently collected by Cancer Research UK in the ongoing trial of VAC2 has shown peripheral immunogenicity in patients with non-small cell lung cancer, providing validation of the underlying mechanism of using allogeneic dendritic cells to present antigens to the body’s immune system. Evidence of immunogenicity was a key clinical signal which was necessary before we would consider taking over the program," stated Brian M. Culley, Lineage CEO. "This encouraging evidence builds upon earlier clinical studies of VAC which supported not only the safety of the VAC platform, but also evidence that patients developed antigen-specific T cell immune responses. We are excited to assume responsibility for advancing this promising program and move Lineage more aggressively into the field of cancer immunotherapy. Given the scientific evidence supporting our approach, the opportunity to also apply our VAC program to SARS-CoV-2 vaccine development was another positive consideration and we are pleased that Cancer Research UK facilitated this decision. Presenting a viral antigen will require transferring the program from Cancer Research UK’s Centre for Drug Development to our cGMP facility, scaling the manufacturing process, and eventually submitting an IND for clinical testing. We also will be evaluating opportunities for non-dilutive funding for this new initiative."

Dr. Nigel Blackburn, Cancer Research UK’s Director of the Centre for Drug Development, said: "We are pleased that our development efforts on the VAC2 program over the past several years have generated initial evidence of an immune response in cancer patients and have resulted in an early exercise of the option by Lineage. We are excited to transfer the next phase of development to our partners and to assist the expansion of those efforts into SARS-CoV-2 vaccine development and other areas with significant unmet need."

About VAC2

VAC2 is an allogeneic, or non-patient specific, cancer vaccine product candidate designed to stimulate patient immune responses to an antigen commonly expressed in cancerous cells but not in normal adult cells. VAC2, which is produced from a pluripotent cell technology using a directed differentiation method, is comprised of a population of nonproliferating mature dendritic cells. As the most potent type of antigen presenting cell in the body, dendritic cells instruct the body’s immune system to attack and eliminate harmful pathogens and unwanted cells. Because the tumor antigen is loaded exogenously into the dendritic cells prior to administration, VAC2 is a platform technology that can be modified to carry any antigen, including patient-specific tumor neo-antigens or viral antigens. VAC2 is currently being tested in a Phase 1 study in adult patients with non-small cell lung cancer (NSCLC) in the advanced and adjuvant settings (NCT03371485), conducted by Cancer Research UK.

About Cancer Research UK’s Centre for Drug Development

Cancer Research UK has an impressive record of developing novel treatments for cancer. The Cancer Research UK Centre for Drug Development has been pioneering the development of new cancer treatments for 25 years, taking over 140 potential new anti-cancer agents into clinical trials in patients. It currently has a portfolio of 21 new anti-cancer agents in preclinical development, Phase I or early Phase II clinical trials. Six of these new agents have made it to market including temozolomide for brain cancer, abiraterone for prostate cancer and rucaparib for ovarian cancer. Two other drugs are in late development Phase III trials.

About Cancer Research UK’s Commercial Partnerships Team

Cancer Research UK is the world’s leading cancer charity dedicated to saving lives through research. Cancer Research UK’s specialist Commercial Partnerships Team works closely with leading international cancer scientists and their institutes to protect intellectual property arising from their research and to establish links with commercial partners. Cancer Research UK’s commercial activity operates through Cancer Research Technology Ltd., a wholly owned subsidiary of Cancer Research UK. It is the legal entity which pursues drug discovery research in themed alliance partnerships and delivers varied commercial partnering arrangements.

About Cancer Research UK

Cancer Research UK is the world’s leading cancer charity dedicated to saving lives through research.
Cancer Research UK’s pioneering work into the prevention, diagnosis and treatment of cancer has helped save millions of lives.
Cancer Research UK receives no funding from the UK government for its life-saving research. Every step it makes towards beating cancer relies on vital donations from the public.
Cancer Research UK has been at the heart of the progress that has already seen survival in the UK double in the last 40 years.
Today, 2 in 4 people survive their cancer for at least 10 years. Cancer Research UK’s ambition is to accelerate progress so that by 2034, 3 in 4 people will survive their cancer for at least 10 years.
Cancer Research UK supports research into all aspects of cancer through the work of over 4,000 scientists, doctors and nurses.
Together with its partners and supporters, Cancer Research UK’s vision is to bring forward the day when all cancers are cured.

Apellis Pharmaceuticals Announces Private Placement of Convertible Senior Notes

On May 7, 2020 Apellis Pharmaceuticals, Inc. (Nasdaq:APLS), a global biopharmaceutical company pioneering targeted C3 therapies, reported that it had entered into subscription agreements with a number of institutional investors for a private placement of $300.0 million aggregate principal amount of 3.500% convertible senior notes due 2026 (the "notes") for an aggregate purchase price of $328.9 million (Press release, Apellis Pharmaceuticals, MAY 7, 2020, View Source [SID1234557318]). The private placement is expected to close on May 12, 2020, subject to satisfaction of customary closing conditions.

The notes issuance is a reopening of, and will form a single series with, and have the same terms as, Apellis’ $220 million aggregate principal amount of 3.500% convertible senior notes due 2026 (the "existing notes") issued on September 16, 2019, but will have a different issue date, issue price, CUSIP number and different restrictions on transfer. The notes will be issued as additional notes under the indenture dated September 16, 2019 and will rank equal in right of payment to the existing notes.

The notes will be unsecured, senior obligations of Apellis, and will bear interest at a rate of 3.500% per annum, payable semi-annually in arrears on March 15 and September 15 of each year, beginning on September 15, 2020. The notes will mature on September 15, 2026, unless earlier repurchased, redeemed or converted in accordance with their terms.

The notes will be convertible into cash, shares of Apellis common stock, or a combination of cash and shares of Apellis common stock, at Apellis’ election. Prior to March 15, 2026, the notes will be convertible only upon the occurrence of certain events and during certain periods, and thereafter, at any time until the second scheduled trading day immediately preceding the maturity date.

The conversion rate for the notes will initially be 25.3405 shares of Apellis common stock per $1,000 principal amount of notes, which is equivalent to an initial conversion price of approximately $39.46 per share. The conversion rate will be subject to adjustment upon the occurrence of certain events.

Apellis estimates that the net proceeds of the offering will be approximately $323.0 million, after deducting placement agent fees and estimated offering expenses payable by Apellis. Apellis intends to use $43.1 million of the net proceeds from the offering to pay the cost of the capped call transactions entered into in connection with the private placement. Apellis intends to use the remainder of the net proceeds from the sale of the notes to fund clinical development of pegcetacoplan, including preparation of a new drug application submission, to support the potential commercialization of pegcetacoplan, if Apellis decides to commercialize on its own, including the build-out of a commercial infrastructure and sales force, to procure clinical and commercial supply of pegcetacoplan, to conduct research activities and for working capital and other general corporate purposes.

J. Wood Capital Advisors LLC is acting as sole placement agent in connection with the private placement.

The offer and sale of the notes and the shares of common stock issuable upon conversion of the notes, if any, have not been and will not be registered under the Securities Act of 1933, as amended, or the securities laws of any other jurisdiction, and the notes and any such shares may not be offered or sold in the United States absent registration or an applicable exemption from such registration requirements.

This press release shall not constitute an offer to sell, or a solicitation of an offer to buy the notes, nor shall there be any sale of, the notes in any state or jurisdiction in which such offer, solicitation or sale would be unlawful under the securities laws of any such state or jurisdiction.

Aldeyra Therapeutics Reports First-Quarter 2020 Financial Results and Announces New Clinical Programs

On May 7, 2020 Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra), a clinical-stage biotechnology company focused on the development of novel therapies with the potential to improve the lives of patients with immune-mediated diseases, today reported financial results for the first quarter ended March 31, 2020, reported new clinical trials in systemic inflammatory diseases, and provided an update on ocular disease programs (Press release, Aldeyra Therapeutics, MAY 7, 2020, View Source [SID1234557317]).

"Based on the success of the novel RASP inhibitor ADX-629 in Phase 1 clinical testing, we are pleased to announce a new comprehensive clinical initiative in systemic inflammatory diseases, complementing our late-stage pipeline in ocular disease," said Todd C. Brady, M.D., Ph.D., President and CEO of Aldeyra. "Although the COVID-19 pandemic has affected clinical trial enrollment timelines, our cash position remains strong, and we are extending our projected cash runway guidance into 2022."

New Clinical Programs in Systemic Inflammatory Diseases

Aldeyra plans to assess the activity of ADX-629 in three types of severe inflammation: cytokine release syndrome, autoimmune disease, and allergy. In preclinical models, ADX-629 treatment reduced levels of TH1, TH2, and TH17-related cytokines, suggesting potential activity across a broad array of inflammatory diseases. The timing of clinical trial initiation depends, in part, on restrictions related to COVID-19, the availability of clinical research facilities and staffing, and the ability to recruit patients.

COVID-19 Respiratory Compromise: A Phase 2 clinical trial of ADX-629 is expected in subjects with COVID-19-associated respiratory compromise, defined as hypoxia and pulmonary radiographic involvement, immediately following admission to the hospital. Severe inflammation, characterized in part by cytokine release syndrome, leads to acute respiratory distress syndrome and other conditions that require mechanical ventilation. The clinical trial, contingent on

FDA review of information submitted via the Coronavirus Treatment Acceleration Program (CTAP), is expected to begin in the third quarter of 2020.

Autoimmune Disease: A Phase 2a clinical trial of ADX-629 in patients with psoriasis, an autoimmune condition associated with TH1 cytokines, is expected to begin in the second half of 2020.

Allergy: A Phase 2a allergen-challenge clinical trial of ADX-629 in patients with atopic asthma, an allergic inflammatory disease associated with TH2 cytokines, is expected to begin in the second half of 2020.

Late-Stage Ocular Disease Programs

Reproxalap, a first-in-class RASP inhibitor for topical ocular administration, continues to advance towards a new drug application (NDA) filing in allergic conjunctivitis and dry eye disease. The Phase 3 GUARD Trial of ADX-2191, a novel formulation of methotrexate for intravitreal administration, in patients with proliferative vitreoretinopathy currently remains active, although enrollment has been significantly delayed due to the COVID-19 pandemic.

Dry Eye Disease: A Type C meeting with the U.S. Food and Drug Administration (FDA) is scheduled for mid-2020 to discuss remaining NDA requirements for reproxalap in dry eye disease. Reproxalap has demonstrated clinically relevant improvement from baseline in two well-controlled clinical trials: Part 1 of the Phase 3 RENEW Trial announced late last year and a Phase 2 formulation trial announced earlier this year. Dry eye disease remains poorly served by available therapies, and represents one of the largest markets in ophthalmology, affecting an estimated 34 million patients in the United States. Aldeyra plans to provide an update on dry eye disease clinical development plans following receipt and review of FDA feedback.

Allergic Conjunctivitis: Based on delays primarily associated with an extended allergy season, results from the Phase 3 INVIGORATE Trial of reproxalap are currently expected in the first half of 2021. Based on the successful Phase 3 ALLEVIATE Trial announced in 2019, and assuming continued clinical success and positive regulatory review, reproxalap has the potential to be the first new mechanistic approach in decades for the treatment of allergic conjunctivitis. The current therapeutic landscape of allergic conjunctivitis is generally limited to antihistamines, which do not lead to satisfactory activity in up to one-third of patients, and corticosteroids, which cannot be used chronically due to potentially serious adverse events. Allergic conjunctivitis is one of most common ocular surface diseases, affecting an estimated 66 million patients in the United States, and is often associated with dry eye disease.

Proliferative Vitreoretinopathy (PVR): Patient enrollment in Part 1 of the adaptive the Phase 3 GUARD Trial of ADX-2191 for the prevention of PVR has been significantly delayed due to lack of clinical site availability and staffing resulting from the COVID-19 pandemic. PVR is a rare but vision-threatening retinal disease associated with recurrent retinal detachments. There is no approved therapy for PVR. Aldeyra expects to update the enrollment and completion timeline by year-end, and is also exploring additional indications for ADX-2191, including primary intraocular lymphoma, a rare but serious ocular cancer that can affect the retina, uvea, optic nerve, and other ocular structures.

Financial Review for the Quarter Ended March 31, 2020

For the quarter ended March 31, 2020, Aldeyra reported a net loss of $9.9 million, compared with a net loss of $15.6 million for the quarter ended March 31, 2019. Net loss per share was $0.34 for the quarter ended March 31, 2020, compared with $0.58 for the same period in 2019. Losses have resulted from the costs of Aldeyra’s clinical trials and research and development programs, as well as from general and administrative expenses.

Research and development expenses were $6.6 million for the quarter ended March 31, 2020, compared with $7.8 million for the same period in 2019. The decrease of $1.2 million is primarily related to the decreases in clinical and preclinical development and manufacturing costs. Expenses for the 2019 period also included $6.6 million of in-process research and development expenses incurred in connection with the acquisition of Helio Vision.

General and administrative expenses were $3.0 million for each of the quarters ended March 31, 2020 and 2019. Increases in personnel related costs, including stock-based compensation, were offset by a decrease in legal and other miscellaneous administrative costs.

For the quarter ended March 31, 2020, total operating expenses were $9.6 million, compared with total operating expenses of $17.4 million for the same period in 2019.

Cash, cash equivalents, and marketable securities were $61.4 million as of March 31, 2020. Based on current operating plans, Aldeyra believes that its cash, cash equivalents, and marketable securities as of March 31, 2020 will be sufficient to fund currently anticipated operating expenses into 2022, including the completion of the Phase 3 INVIGORATE Trial for reproxalap, as well as the Phase 2 clinical trials of ADX-629 in COVID-19-associated respiratory compromise, atopic asthma, and psoriasis; the commencement of one or more additional clinical trials in dry eye disease, subject to the outcome of the FDA meeting scheduled for mid-year 2020; and the continuation of Part 1 of the adaptive Phase 3 clinical trial in PVR contingent on patient enrollment.

Conference Call & Webcast Information

Aldeyra will host a conference call today at 8:00 a.m. ET to announce new clinical trials in systemic inflammatory diseases, provide an update on ocular disease programs, and report first-quarter 2020 financial results. The dial-in numbers are (866) 211-4098 for domestic callers and (647) 689-6613 for international callers. The Conference ID number is 6982314. Due to the expected high demand on our conference provider, please plan to dial in to the call at least 30 minutes prior to the start time.

A live webcast of the conference call will also be available on the investor relations page of the company’s corporate website at View Source After the live webcast, the event will remain archived on the Aldeyra Therapeutics website for 90 days.

Intellia Therapeutics Announces First Quarter 2020 Financial Results

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]).

"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.