On May 8, 2019 argenx (Euronext & Nasdaq: ARGX), a clinical-stage biotechnology company developing a deep pipeline of differentiated antibody-based therapies for the treatment of severe autoimmune diseases and cancer, reported that Tim Van Hauwermeiren, Chief Executive Officer, will present at the Bank of America Merrill Lynch 2019 Health Care Conference on Wednesday, May 15, 2019 at 9:20 a.m. PT in Las Vegas, NV (Press release, argenx, MAY 8, 2019, View Source [SID1234535949]).

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!

A live webcast of the presentation will be available on the Company’s website at www.argenx.com. A replay of the webcast will be available for 90 days following the presentation.

On May 8, 2019 Nektar Therapeutics (Nasdaq: NKTR) reported its financial results for the first quarter ended March 31, 2019 (Press release, Nektar Therapeutics, MAY 8, 2019, View Source [SID1234535966]).

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!

Cash and investments in marketable securities at March 31, 2019 were approximately $1.8 billion as compared to $1.9 billion at December 31, 2018.

"Nektar continues to advance our immuno-oncology and immunology pipeline with clinical trials initiating for multiple drug candidates across multiple indications," said Howard W. Robin, President and CEO of Nektar. "We are working with our partner, Bristol-Myers Squibb, to execute on our broad joint development program for bempegaldesleukin in combination with nivolumab, with registrational trials in melanoma, RCC, urothelial and non-small cell lung cancer underway and additional trials planned to begin in the coming months."

"NKTR-181 is under review with the FDA and we are planning for a potential launch later this year," continued Robin. "With respect to NKTR-358, we will report the first data for this exciting drug candidate in an oral presentation at the EULAR Congress in June and our multiple-ascending dose trial in lupus patients is continuing. With our partner Lilly, we continue to advance development of NKTR-358 with two new Phase 1b studies in additional auto-immune disorders planned to start in 2019. We are also completing our IND-enabling activities for our next immuno-oncology candidate, NKTR-255, which activates the IL-15 pathway."

Revenue in the first quarter of 2019 was $28.2 million as compared to $38.0 million in the first quarter of 2018. Revenue in the first quarter of 2019 was lower primarily due to the recognition of $10.0 million received in the first quarter of 2018 from Takeda for the approval of Adynovi in Europe.

Total operating costs and expenses in the first quarter of 2019 were $148.9 million as compared to $124.8 million in the first quarter of 2018. Total operating costs and expenses increased primarily as a result of increased research and development (R&D) expense.

R&D expense in the first quarter of 2019 was $118.5 million as compared to $99.4 million for the first quarter of 2018. R&D expense was higher in the first quarter of 2019 as compared to the same period in 2018 primarily because of expenses for our pipeline programs, including the continued development of bempegaldesleukin in Phase 2 and registrational studies and related manufacturing costs, costs related to Phase 1 clinical studies of NKTR-358 and IND-enabling activities for NKTR-255.

General and administrative expense was $25.0 million in the first quarter of 2019 as compared to $18.7 million in the first quarter of 2018 and increased primarily due to costs related to commercialization readiness activities for NKTR-181 and increased non-cash stock-based compensation.

In the first quarter of 2019, net loss was $118.5 million, or $0.68 loss per share as compared to net loss of $95.8 million, or $0.60 loss per share in the first quarter of 2018.

2019 Business Highlights

In April, Nektar presented positive preclinical data on its immuno-oncology pipeline candidates, bempegaldesleukin and NKTR-255, an IL-15 receptor agonist, at the 2019 AACR (Free AACR Whitepaper) Annual Meeting.
In March, Nektar presented preliminary immune activation, safety and clinical activity data from the ongoing dose-escalation stage of the REVEAL study at the 2019 ASCO (Free ASCO Whitepaper)-SITC Meeting. The REVEAL Phase 1/2 study is evaluating the safety and efficacy of NKTR-262, a novel TLR agonist, in combination with bempegaldesleukin.
In February, Nektar presented clinical data from first-line Stage IV urothelial carcinoma patients enrolled in the PIVOT-02 study of bempegaldesleukin with nivolumab at the 2019 ASCO (Free ASCO Whitepaper) Genitourinary Cancers Symposium.
The company also announced upcoming presentations at the following scientific congresses during the second quarter of 2019:

4th Drug Discovery Nexus, Boston, MA:

Presentation: "Harnessing cytokines to develop immune therapeutic agents"
Presenter: Loui Madakamutil, Ph.D., Nektar Therapeutics
Date: Friday, May 17, 2019, 9:30 a.m. Eastern Daylight Time
4th Annual Advances in Immuno-Oncology Congress 2019, London, U.K.:

Presentation: "Bempegaldesleukin (NKTR-214): Targeting the IL-2 Pathway for Immuno-Oncology Applications"
Presenter: Jonathan Zalevsky, Ph.D., Nektar Therapeutics
Session: Immuno-Oncology Therapeutic Approaches, Clinical Research & Clinical Trials
Date: Tuesday, May 21, 2019, 16:30 British Summer Time
American Society for Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2019 Annual Meeting, Chicago, IL:

Oral Abstract # 11010: "Pilot study of bempegaldesleukin (NKTR-214) and nivolumab in patients with sarcomas"
Presenter: Sandra D’Angelo, M.D., Memorial Sloan-Kettering Cancer Center
Session: Clinical Science Symposium: Emerging Combinations in Sarcoma Immunotherapy
Date: Monday, June 3, 2019, 11:30 a.m. – 1:00 p.m. Central Daylight Time
Poster #228/Abstract # 2584: "Overcoming genetically-based resistance mechanisms to PD-1 blockade", Torrejon, D., et al.
Session: Developmental Immunotherapy and Tumor Immunobiology
Date: Saturday, June 1, 2019, 8:00 a.m. – 11:00 a.m. Central Daylight Time
Poster #267/Abstract # 2623: "Baseline tumor immune signatures associated with response to bempegaldesleukin (NKTR-214) and nivolumab", Hurwitz, M., et al.
Session: Developmental Immunotherapy and Tumor Immunobiology
Date: Saturday, June 1, 2019, 8:00 a.m. – 11:00 a.m. Central Daylight Time
Poster #416b/Abstract # TPS4595: "A phase III randomized open label study comparing bempegaldesleukin (NKTR-214) plus nivolumab to sunitinib or cabozantinib (investigator’s choice) in patients with previously untreated advanced renal cell carcinoma", Tannir, N., et al.
Session: Genitourinary (Nonprostate) Cancer
Date: Monday, June 3, 2019, 1:15 p.m. – 4:15 p.m. Central Daylight Time
Poster 168b/Abstract # TPS9601: "A phase III, randomized, open-label study of bempegaldesleukin (NKTR-214) plus nivolumab (NIVO) versus NIVO monotherapy in patients (pts) with previously untreated, unresectable or metastatic melanoma (MEL)", Khushalani, N., et al.
Session: Melanoma/Skin Cancers
Date: Monday, June 3, 2019, 1:15 p.m. – 4:15 p.m. Central Daylight Time
Pharmaceutical & Bioscience Society Symposium: Advances in Immuno-Oncology, Foster City, CA:

Presentation: "Bempegaldesleukin (NKTR-214), a first-in-class, CD122-preferential IL-2 pathway agonist"
Presenter: Willem Overwijk, Ph.D., Nektar Therapeutics
Date: Tuesday, June 11, 2019, 8:45 a.m. Pacific Daylight Time
24th Congress of European Hematology Association (EHA) (Free EHA Whitepaper), Amsterdam, Netherlands:

Abstract # PS1208: " Effects Of NKTR-255, A Polymer Conjugated Human IL-15, on Efficacy of CD19 CAR T Cell Immunotherapy in a Preclinical Lymphoma Model"
Presenter: Cassie K. Chou, M.D., Ph.D., Fred Hutchinson Cancer Research Center
Session: Gene therapy, cellular immunotherapy and vaccination – Biology & Transitional Research
Date: Saturday, June 15, 2019, 17:30 – 19:00 Central European Summer Time
Poster Pitch: Saturday, June 15, 2019, 16:30 – 16:45 Central European Summer Time
Location: Hall G106
Annual European Congress of Rheumatology (EULAR) 2019, Madrid, Spain:

Abstract # OP0195: "Selective Expansion of Regulatory T-Cells in Humans by a Novel IL-2 Conjugate T-reg Stimulator, NKTR-358, Being Developed for the Treatment of Autoimmune Diseases"
Presenter: Brian Kotzin, M.D., Nektar Therapeutics
Session: Genetics, epigenetics and immunity
Date: Thursday, June 13, 2019, 11:15 Central European Summer Time
Conference Call to Discuss First Quarter 2019 Financial Results
Nektar management will host a conference call to review the results beginning at 5:00 p.m. Eastern Daylight Time/2:00 p.m. Pacific Daylight Time, Wednesday, May 8, 2019.

This press release and a live audio-only Webcast of the conference call can be accessed through a link that is posted on the home page and Investors section of the Nektar website: View Source The web broadcast of the conference call will be available for replay through Monday, June 10, 2019.

To access the conference call, follow these instructions:

Dial: (877) 881-2183 (U.S.); (970) 315-0453 (international)
Passcode: 8249707 (Nektar Therapeutics is the host)

In the event that any non-GAAP financial measure is discussed on the conference call that is not described in the press release, or explained on the conference call, related information will be made available on the Investors page at the Nektar website as soon as practical after the conclusion of the conference call.

On May 8, 2019 Jounce Therapeutics, Inc. (NASDAQ: JNCE), a clinical-stage company focused on the discovery and development of novel cancer immunotherapies and predictive biomarkers for patient enrichment, reported financial results and provided a corporate update for the first quarter ended March 31, 2019 (Press release, Jounce Therapeutics, MAY 8, 2019, View Source [SID1234535905]).

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!

"We have made significant progress in recent months by applying our Translational Science Platform and reverse translational approach to advance both our ongoing clinical and preclinical programs. Most importantly, at AACR (Free AACR Whitepaper), we presented promising new progression free and overall survival data from the ICONIC trial for patients stratified by our vopratelimab pharmacodynamic biomarker, ICOS hi CD4 T cells in the blood. Additionally, we presented validating preclinical data from our lead tumor-associated macrophage program, JTX-8064," said Richard Murray, Ph.D., chief executive officer and president of Jounce Therapeutics. "With these key accomplishments in hand, we look forward to advancing our broader pipeline with the goal of three immunotherapies in the clinic in 2019. We remain focused on the underlying mechanistic science of our immunotherapies and understanding of the characteristics of responding patients in our mission to bring meaningful and long-lasting benefit to cancer patients with unmet needs."

Pipeline Highlights:
Vopratelimab (JTX-2011)

Key data presented at AACR (Free AACR Whitepaper) 2019: In April 2019, Jounce presented two posters on vopratelimab at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. Highlights from the poster presentations include:

Patients in the ICONIC trial with the emergence of ICOS hi CD4 T cells demonstrated improved progression free survival (PFS) and overall survival (OS) compared to patients with ICOS lo CD4 T cells, based on an analysis of a subgroup of patients with multiple solid tumor types including PD-1 inhibitor naive and PD-1 inhibitor experienced patients.

The characteristics of ICOS hi CD4 T cells associated with vopratelimab treatment via translational analyses demonstrated that vopratelimab stimulates only primed CD4 T cells with high levels of ICOS. The translational data shows that vopratelimab, unlike PD-1 inhibitors, leads to expansion and activation of peripheral CD4 T effector cells, and that these are observed in patients with clinical benefit.

On track for planned initiation of Phase 2 clinical studies: Based on the recently-presented AACR (Free AACR Whitepaper) data, Jounce plans to initiate additional Phase 2 clinical studies focusing on settings in which ICOS hi CD4 T effector cells exist or emerge and are primed to respond to vopratelimab, potentially leading to clinical benefit. The first of these studies will be a clinical trial of

jouncelogosmaller.jpg

vopratelimab in combination with ipilimumab in PD-1 inhibitor experienced patients in two tumor types, non-small cell lung cancer and bladder cancer. Additionally, Jounce expects to initiate a clinical trial of vopratelimab in combination with ipilimumab in PD-1 inhibitor naive patients with bladder cancer and a separate predictive biomarker study. Jounce expects to report preliminary efficacy data in 2020.

JTX-4014

On track to complete enrollment of Phase 1 study: Jounce remains on track to assess safety and select the recommended Phase 2 dose for JTX-4014, its PD-1 inhibitor, in 2019.

JTX-8064

New validating preclinical data presented at AACR (Free AACR Whitepaper) 2019: In April 2019, Jounce presented new preclinical data demonstrating the properties of JTX-8064, Jounce’s lead tumor associated macrophage candidate. JTX-8064 is an inhibitor of LILRB2 (leukocyte immunoglobulin like receptor B2; ILT4) and is believed to reprogram tumor-associated macrophages within the tumor microenvironment. Highlights from the poster presentation include:

When LILRB2 binds to its ligands, it maintains macrophages in the M2 or immuno-suppressive state.

When JTX-8064 blocks ligand binding to LILRB2, it induces an immune activating state in macrophages that may lead to the enhancement of the anti-tumor immune response.

Inhibiting LILRB2 induces pro-inflammatory cytokine secretion and a unique transcriptional profile suggestive of an M1-like shift in human macrophages to an immune stimulatory state.

On track to file IND and initiate Phase 1 clinical trial: Jounce expects to file an investigational new drug (IND) application and initiate a Phase 1 clinical trial of JTX-8064 in 2019.

First Quarter 2019 Financial Results:

Cash Position: As of March 31, 2019, cash, cash equivalents and investments were $173.2 million, compared to $195.9 million as of December 31, 2018. The decrease in cash, cash equivalents and investments was primarily due to operating costs incurred during the period.

Collaboration Revenue: Collaboration revenue was $11.0 million for the first quarter of 2019, compared to $11.2 million for the same period in 2018. Collaboration revenue represents non-cash revenue recognition relating to the $225.0 million upfront payment received in July 2016 upon the execution of Jounce’s global strategic collaboration with Celgene.

Research and Development Expenses: Research and development (R&D) expenses were $17.3 million for the first quarter of 2019, compared to $18.2 million for the same period in 2018. The decrease in R&D expenses was primarily due to $1.5 million of decreased external research and development costs attributable to JTX-4014 IND-enabling expenses incurred during the first quarter of 2018, partially offset by $0.5 million of increased employee compensation costs.

General and Administrative Expenses: General and administrative (G&A) expenses were $7.2 million for the first quarter of 2019, compared to $6.8 million for the same period in 2018. The increase in G&A expenses was primarily due to $0.7 million of increased employee compensation costs, including $0.3 million of increased stock-based compensation expense.

jouncelogosmaller.jpg

Net Loss: Net loss was $12.4 million for the first quarter of 2019, or a basic and diluted net loss per share of $0.38. Net loss was $13.0 million for the same period in 2018, or a basic and diluted net loss per share of $0.40. The decrease in net loss and net loss per share was primarily attributable to the decrease in operating expenses from the first quarter of 2018 to the first quarter of 2019.

Financial Guidance:
Jounce reiterates its expectation that cash burn on operating expenses and capital expenditures for the full year 2019 will be approximately $80.0 million to $95.0 million. Jounce expects to record approximately $50.0 million to $60.0 million in non-cash collaboration revenue in 2019 from the recognition of the Celgene upfront payment received in 2016.

Conference Call and Webcast Information:
Jounce Therapeutics will host a live conference call and webcast today at 8:00 a.m. ET. To access the conference call, please dial (866) 916-3380 (domestic) or (210) 874-7772 (international) and refer to conference ID 5789371. The live webcast can be accessed under "Events & Presentations" in the Investors and Media section of the company’s website at www.jouncetx.com. The webcast will be archived and made available for replay on the company’s website approximately two hours after the call and will be available for 30 days.

Cautionary Note Regarding Forward-Looking Statements:
Various statements in this release concerning Jounce’s future expectations, plans and prospects, including without limitation, Jounce’s expectations regarding operating expenses, capital expenditures, collaboration revenue and other financial results; the timing, progress and release of data for Phase 2 clinical studies of vopratelimab; the timing, progress and results of the Phase 1 trial of JTX-4014; the filing of an IND and initiation of a Phase 1 trial of JTX-8064 and the timing, progress and results of preclinical studies and clinical trials for Jounce’s product candidates and any future product candidates may constitute forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "anticipate," "believe," "estimate," "expect," "intend," "may," "on track," "plan," "predict," "target," "potential" or similar terms, variations of such terms or the negative of those terms. Although the Company believes that the expectations reflected in the forward-looking statements are reasonable, the Company cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Jounce’s ability to successfully demonstrate the efficacy and safety of its product candidates and future product candidates; the preclinical and clinical results for its product candidates, which may not support further development and marketing approval; the potential advantages of Jounce’s product candidates; the development plans of its product candidates and any companion or complementary diagnostics; actions of regulatory agencies, which may affect the initiation, timing and progress of preclinical studies and clinical trials of Jounce’s product candidates; Jounce’s ability to obtain, maintain and protect its intellectual property; Jounce’s ability to manage operating expenses; Jounce’s ability to maintain its collaboration with Celgene and those risks more fully discussed in the section entitled "Risk Factors" in Jounce’s most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission as well as discussions of potential risks, uncertainties, and other

jouncelogosmaller.jpg

important factors in Jounce’s subsequent filings with the Securities and Exchange Commission. All such statements speak only as of the date made, and the Company undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

On May 8, 2019 Athersys, Inc. (NASDAQ: ATHX) reported its financial results for the three months ended March 31, 2019 (Press release, Athersys, MAY 8, 2019, View Source [SID1234535933]).

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!

Highlights of the first quarter of 2019 and recent events include:

Announced positive results from our exploratory clinical study of MultiStem cell therapy for treatment of acute respiratory distress syndrome ("MUST-ARDS"), showing reduced mortality and greater ventilator-free and ICU-free days in patients with moderate to severe ARDS;

The MUST-ARDS study has been selected for presentation at the American Thoracic Society International Conference on May 20, 2019;

Our partner in Japan, HEALIOS K.K. ("Healios"), announced its first patient enrolled in the ONE-BRIDGE study in Japan, evaluating MultiStem cell therapy treatment of patients who suffer from pneumonia-induced ARDS;

Advanced ischemic stroke program, supporting Healios’ TREASURE study and ramping up our MASTERS-2 Phase 3 registrational study;

Announced plans to host an investor day in New York City on May 14, 2019 that will highlight the Company’s capabilities and technologies, as well as progress made developing the MultiStem product platform and future plans;

Participated in several investor conferences and media interviews, including "The Stem Cell Podcast" and the "Forbes Book Podcast" as the featured guest; also featured live on TD Ameritrade’s "The WatchList";

Launched a revamped website with a simplified design, improved functionality and enhanced content to help educate our visitors;

Recognized revenues of $1.4 million for the quarter ended March 31, 2019 and net loss of $13.0 million, or $0.09 net loss per share, for the quarter ended March 31, 2019; and

Ended the 2019 first quarter with $51.0 million of cash and cash equivalents.
"We made continued progress in the first quarter of 2019 in advancing our key initiatives, reflected by the release of results from our ARDS study and the continued progress of enrollment in our Phase 3 MASTERS-2 trial and the TREASURE registrational study being conducted by our partner in Japan for treating ischemic stroke," commented Dr. Gil Van Bokkelen, Chairman & CEO at Athersys. "We are working collaboratively with Healios in multiple areas, which includes supporting their initiation of the ONE-BRIDGE study for ARDS, and we further strengthened our core capabilities."
"In addition, we have maintained a solid balance sheet as we continue to implement our strategic plan, working toward the achievement of our key goals on behalf of our shareholders and the patients we are committed to helping," concluded Dr. Van Bokkelen.

First Quarter Results
Revenues increased to $1.4 million for the three months ended March 31, 2019 compared to $1.1 million for the three months ended March 31, 2018. Our revenues are generally derived from license fees, manufacturing-related activities for Healios, other royalty and related contract revenue from our collaborations, and grant revenue. Revenues from our collaboration with Healios increased quarter-over-quarter related to our manufacturing services. Royalty revenues from RTI Surgical, Inc. have concluded with its discontinuation of distribution of the licensed product.
Research and development expenses increased to $11.4 million for the three months ended March 31, 2019 from $8.9 million for the comparable period in 2018. The $2.5 million increase is primarily associated with increased clinical trial and manufacturing process development costs of $1.9 million, increased personnel costs of $0.4 million, and increased stock compensation costs of $0.1 million. Included in our clinical costs are costs associated with providing manufacturing services to Healios, which are invoiced to Healios in accordance with the collaboration agreements.
General and administrative expenses increased to $3.1 million for the three months ended March 31, 2019 from $2.7 million in the comparable period in 2018. The $0.4 million increase was due primarily to increased legal and professional fees, consulting services, and stock compensation costs compared to the same period last year.
Net loss for the first quarter was $13.0 million in 2019 compared to a net loss of $10.2 million in 2018. The difference of $2.8 million reflects the above variances, as well as a decrease of $0.2 million in other income.
In the three months ended March 31, 2019, net cash used in operating activities was $5.5 million compared to $5.7 million in the three months ended March 31, 2018. At March 31, 2019, we had $51.0 million in cash and cash equivalents, compared to $51.1 million at December 31, 2018.

Conference Call
William (B.J.) Lehmann, President and Chief Operating Officer, and Laura Campbell, Senior Vice President of Finance, will host a conference call today to review the results as follows:

Date

May 8, 2019
Time

4:30 p.m. (Eastern Time)
Telephone access: U.S. and Canada

(877) 396-3286
Telephone access: International

(647) 689-5528
Access code

6595362
Live webcast

www.athersys.com, under the Investors section

We encourage shareholders to listen using the webcast link and to use the phone line if you intend to ask a question. A replay will be available at www.athersys.com under the Investors section approximately two hours after the call has ended. Shareholders may also call in for on-demand listening shortly after the completion of the call until 11:59 PM Eastern Time on May 15, 2019 by dialing (800) 585-8367 or (416) 621-4642 and entering Encore passcode 6595362.

On May 8, 2019 Moderna, Inc. (Nasdaq: MRNA), a clinical stage biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for patients, reported financial results for the first quarter of 2019 and provided business updates (Press release, Moderna Therapeutics, MAY 8, 2019, View Source/news-releases/news-release-details/moderna-reports-first-quarter-2019-financial-results-and" target="_blank" title="View Source/news-releases/news-release-details/moderna-reports-first-quarter-2019-financial-results-and" rel="nofollow">View Source [SID1234535950]).

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!

"We continue to execute against our corporate objectives as we progress clinical studies across our development portfolio, introduce a new mRNA rare disease development candidate and focus on identifying additional modalities and disease targets," said Stéphane Bancel, Moderna’s chief executive officer. "We are excited to pursue a treatment to potentially address the underlying cause of glycogen storage disease type 1a, and we believe this candidate also reflects the continued productivity of our mRNA platform. At yesterday’s annual Science Day event, we presented new insights into our mRNA and delivery science, including the potential delivery of mRNA to white blood cells. While our team has additional research to perform in this area, we look forward to being able to bring new candidates into development as we continue working to help patients with a wide range of serious diseases."

Moderna currently has 21 mRNA development candidates in its portfolio with 11 in clinical studies. Across Moderna’s pipeline, more than 1,000 subjects have been enrolled in clinical studies. The Company’s updated pipeline can be found at www.modernatx.com/pipeline.

Summary of Recent Highlights by Modality

Prophylactic vaccines: Moderna is developing vaccines against viral diseases where there is unmet medical need – including complex vaccines with multiple antigens for common diseases, as well as vaccines against epidemic and pandemic threats to global public health.

Respiratory syncytial virus (RSV) vaccine (mRNA-1777 and mRNA-1172): Merck has filed an investigational new drug (IND) application with the U.S. Food and Drug Administration (FDA) and plans to run a Phase 1 study for a follow-on development candidate (mRNA-1172), a vaccine for RSV which has shown enhanced potency in preclinical studies and uses a Merck proprietary formulation. As a result, further development of mRNA-1777 has been paused and next steps will be determined based on data from the new mRNA-1172 Phase 1 study.
Varicella zoster virus (VZV) vaccine (mRNA-1278): Based on an assessment of the commercial opportunity, research priorities and other factors, Merck has discontinued preclinical development of mRNA-1278, an investigational vaccine for VZV (the virus that causes shingles). Merck has returned rights to Moderna, and the Company will not continue development at this time.
Cytomegalovirus (CMV) vaccine (mRNA-1647): The first three dose levels in Moderna’s ongoing study of mRNA-1647 are fully enrolled, and the study is currently enrolling subjects into the fourth (300μg) dose cohort. The Phase 1 study is randomized, observer-blind and placebo-controlled with the goal of evaluating the safety and immunogenicity of mRNA-1647, a vaccine against the pentamer and gB complexes of CMV.
Presentation of note: Moderna presented data from its Phase 1 studies of vaccines against viruses that cause respiratory diseases at the European Society for Pediatric Infectious Disease (ESPID) meeting held in Ljubljana, Slovenia.
Cancer Vaccines: These programs focus on stimulating a patient’s immune system with antigens derived from tumor-specific mutations to enable the immune system to elicit a more effective anti-tumor response.

Personalized cancer vaccines (PCVs) (mRNA-4157, NCI-4650): Two abstracts for Moderna PCV programs were accepted for presentation at the 2019 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. Moderna will present new updates to clinical data from the Company’s Phase 1 study of mRNA-4157, a PCV being studied alone in patients with resected solid tumors and in combination with Merck’s pembrolizumab in patients with unresectable solid tumors. The National Cancer Institute (NCI) will also present data from its Phase 1 study of PCV NCI-4650, a monotherapy for patients with advanced metastatic cancers. The two abstracts are:
Moderna: A Phase 1 multi-center study to assess the safety, tolerability and immunogenicity of mRNA-4157 alone in patients with resected solid tumors and in combination with pembrolizumab in patients with unresectable solid tumors. Selected for a Poster Discussion, Saturday, June 1, 1:15 PM-2:45 PM CST.
NCI: Immunogenicity and tolerability of personalized mRNA vaccine mRNA-4650 encoding defined neoantigens expressed by the autologous cancer. Selected for a Poster Session, Saturday, June 1, 8:00 AM-11:00 AM CST.
Moderna and NCI PCVs are designed and manufactured individually based on the DNA sequence of a patient’s tumor, encoding for peptides containing mutations found in their cancer in order to deliver multiple unique and personalized neoantigens in a single vaccine. Moderna’s PCV now includes up to 34 neoantigens, up from 20. The NCI program uses Moderna’s mRNA technology but uses a different neoantigen selection process and study design.

Intratumoral Immuno-Oncology: These programs aim to drive anti-cancer T cell responses by injecting mRNA therapies directly into tumors.

OX40L (mRNA-2416): Moderna continues to enroll patients in its Phase 1/2 trial evaluating mRNA-2416, an intratumoral injection of mRNA encoding OX40L, for the treatment of advanced relapsed/refractory solid tumor malignancies and lymphomas and is also preparing to start enrollment of a Phase 2 expansion cohort of the study in patients with advanced ovarian carcinoma.
OX40L + IL23 + IL36γ (Triplet) (mRNA-2752): Moderna continues to dose a second cohort (0.5 mg) of patients in its ongoing Phase 1 study evaluating the safety and tolerability of mRNA- 2752 for the treatment of advanced or metastatic solid tumor malignancies or lymphoma. mRNA-2752 is an investigational mRNA immuno-oncology therapy that encodes a novel combination of three immunomodulators designed to activate the immune system to recognize and eradicate tumors that are resistant to checkpoint inhibitors. It is being studied both as a single agent and in combination with AstraZeneca’s durvalumab or tremelimumab.
IL12 (MEDI1191): At the American Academy of Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in March, Moderna’s strategic collaborator AstraZeneca shared preclinical data that supported advancing MEDI1191 into a Phase 1 clinical study. MEDI1191 is an mRNA encoding for IL12, a potent immunomodulatory cytokine, which aims to enhance immune response in immunologically "cold" tumors. AstraZeneca is now leading an open-label, multi-center study of intratumoral injections of MEDI1191 alone and in combination with a checkpoint inhibitor.
Systemic Secreted Therapeutics: In this modality, mRNA is delivered systemically to create proteins that are secreted outside the cell with the aim of producing pharmaceutically active proteins with therapeutic effects across the human body.

Antibody against the chikungunya virus (mRNA-1944): Moderna has completed enrollment of the second dose level cohort (0.3 mg/kg) of its Phase 1 study evaluating the safety and tolerability of escalating doses of mRNA-1944 via intravenous infusion in healthy adults. This is the first monoclonal antibody encoded by mRNA to be dosed in a human and the first development candidate from the Company’s systemic secreted therapeutics modality to start clinical testing. The formulation used for mRNA-1944 is also utilized in Moderna’s MMA program.
Systemic Intracellular Therapeutics: These programs aim to deliver mRNA into cells within target organs as a therapeutic approach for diseases caused by a missing or defective protein.

Methylmalonic acidemia (MMA) (mRNA-3704): Site initiation activities are ongoing for the Phase 1 study of mRNA-3704, Moderna’s program for the rare metabolic disease MMA.
Propionic acidemia (PA) (mRNA-3927): The European Commission (EC) has adopted the recommendation from the Committee for Orphan Medicinal Products for orphan drug designation for mRNA-3927, a development candidate for propionic acidemia (PA).

Additionally, enrollment continues in the Company’s global natural history study of MMA and PA (MaP study). This is a global, multi-center, non-interventional study for patients with confirmed diagnosis of MMA due to methylmalonyl-CoA mutase (MUT) deficiency or PA and is designed to identify and correlate clinical and biomarker endpoints for these disorders.
Glycogen storage disease type 1a (GSD1a) (mRNA-3745): Moderna has selected a new development candidate for the rare inherited metabolic disease GSD1a. GSD1a results in a buildup of glycogen in tissues and an inability to regulate glucose, due to mutations within the enzyme glucose 6-phosphatase (G6Pase), leading to life-threatening hypoglycemia and long-term liver and kidney damage. Patients with this disease incur metabolite buildup associated with hepatomegaly (enlarged livers), which can lead to benign and malignant liver tumors. mRNA-3745 is an IV-administered mRNA encoding G6Pase enzyme, designed to restore deficient or defective intracellular enzyme activity. This is expected to increase blood glucose levels, while decreasing levels of uric acid, lactic acid and triglycerides. In a mouse model, Moderna has shown the ability to improve hypoglycemia and other metabolic abnormalities associated with GSD1a, and mice treated with G6Pase mRNA showed a dose-dependent improvement in fasting glycemia and a reduction in both serum triglycerides and liver weight. There are approximately 6,500 GSD1a patients in the United States and the European Union. Disease management requires a strict diet to maintain blood glucose levels with some patients requiring a liver transplant. Preclinical data from this program were shared at the 22nd Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper).
Other systemic therapeutic preclinical research: Also at ASGCT (Free ASGCT Whitepaper) 2019, Moderna and academic collaborators gave oral presentations and shared data from seven preclinical animal studies using mRNA-based therapies for several rare diseases including: ornithine transcarbamylase deficiency (OTC) and maple syrup urine disease (MSUD) (in collaboration with the Gene Therapy Program at the Perelman School of Medicine, University of Pennsylvania); arginase-1 (ARG1) deficiency (in collaboration with the University of California, Los Angeles Department of Surgery and Department of Molecular and Medical Pharmacology); factor VIII deficiency (hemophilia A) (in collaboration with the Seattle Children’s Research Institute); adult-onset type II citrullinemia (CTLN2); and progressive familial intrahepatic cholestasis type 3 (PFIC3). These programs are early research initiatives and presently are not Moderna development candidates.
Publication of note: In March, Moderna published data in the American Journal of Human Genetics that showed preclinical proof-of-concept for administering mRNA encoding human ɑ-Gal, across species, as a potential systemic mRNA therapy for the treatment of Fabry disease.
Information about each development candidate in Moderna’s pipeline, including those discussed in this press release, can be found on the investor relations page of its website View Source/." target="_blank" title="View Source/." rel="nofollow">View Source

First Quarter 2019 Financial Results

Cash Position: Cash, cash equivalents and investments as of March 31, 2019 and December 31, 2018 were $1.55 billion and $1.69 billion, respectively.
Net Cash Used in Operating Activities: Net cash used in operating activities was $143.9 million for the three months ended March 31, 2019 compared to $111.4 million for the three months ended March 31, 2018. Net cash used in operating activities includes $22.0 million and $25.0 million in the first quarter of 2019 and 2018, respectively, of in-licensing payments to Cellscript, LLC and its affiliate, mRNA RiboTherapeutics, Inc., to sublicense certain patent rights. After 2019, we have no further in-licensing payment obligation to Cellscript and its affiliate.
Cash Used for Purchases of Property and Equipment: Cash used for purchases of property and equipment was $7.6 million for the three months ended March 31, 2019 compared to $31.9 million for the three months ended March 31, 2018.
Revenue: Total revenue was $16.0 million for the three months ended March 31, 2019 compared to $29.0 million for the three months ended March 31, 2018. On January 1, 2019, we adopted Accounting Standards Codification (ASC) Topic 606, Revenue from Contracts with Customers (ASC 606), using the modified retrospective transition method applied to those contracts which were not completed as of January 1, 2019. The total revenue decrease was mainly attributable to the decrease in collaboration revenue from all of our strategic alliances, particularly AstraZeneca and Merck, largely driven by the adoption of ASC 606. Total revenue under the previous revenue recognition standard would have been $37.9 million for the first quarter of 2019.
Research and Development Expenses: Research and development expenses were $130.6 million for the three months ended March 31, 2019 compared to $90.1 million for the three months ended March 31, 2018. The increase was primarily due to an increase in personnel related costs, including stock-based compensation, mainly driven by an increase in the number of employees supporting research and development programs, an increase in clinical trial and manufacturing costs, an increase in lab supplies and materials for our preclinical studies and clinical trials, and an increase in consulting and outside services costs.
General and Administrative Expenses: General and administrative expenses were $27.3 million for the three months ended March 31, 2019 compared to $16.3 million for the three months ended March 31, 2018. The increase was mainly attributable to an increase in personnel related costs, including stock-based compensation, primarily driven by an increase in the number of employees, and consulting and outside services costs, both of which were related to operating as a publicly traded company.
Net Loss: Net loss was $132.7 million for the three months ended March 31, 2019 compared to $72.4 million for the three months ended March 31, 2018.
Other Corporate Updates

Moderna Annual Science Day: On May 7, Moderna hosted its annual Science Day, which featured presentations from Stephen Hoge M.D., president and Melissa Moore Ph.D., chief scientific officer of Moderna’s mRNA research platform, and focused on some of the Company’s latest advances in basic and applied mRNA science. This included improvements in the potency and delivery of potential mRNA medicines, and a new research program focused on delivery of mRNA to the immune system. The archived webcast of Science Day is available under "Events & Presentations" on the Investors section of the Moderna website at View Source and will be available there for approximately 30 days.
Company Management:
Moderna’s mRNA platform chief scientific officer, Dr. Melissa Moore, was recently elected to the American Academy of Arts and Sciences.
Moderna’s chief human resources officer, Annie Drapeau, left the Company in April to return to a human resources leadership role in the technology industry.
Investor Call and Webcast Information

Moderna will host a live conference call and webcast at 8:00 a.m. ET on Wednesday, May 8, 2019. To access the call, please dial 866-922-5184 (domestic) or 409-937-8950 (international) and refer to conference ID 8273939. A webcast of the call will also be available under "Events & Presentations" in the Investors section of the Moderna website at View Source/." target="_blank" title="View Source/." rel="nofollow">View Source The archived webcast will be available on Moderna’s website approximately two hours after the conference call and will be available for 30 days following the call.