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Cytokinetics Reports Second Quarter 2021 Financial Results

On August 5, 2021 Cytokinetics, Incorporated (Nasdaq: CYTK) reported financial results for the second quarter of 2021. Net loss for the second quarter was $61.6 million, or $0.86 per share, compared to net loss for the second quarter of 2020 of $40.8 million, or $0.68 per share (Press release, Cytokinetics, AUG 5, 2021, View Source [SID1234585849]). Cash, cash equivalents and investments totaled $424.0 million at June 30, 2021. After the quarter, Cytokinetics raised approximately $297.3 million in net proceeds after deducting the applicable underwriting discounts and commissions through a public offering of common stock. Cytokinetics expects to end 2021 with more than $600 million in cash.

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"During the second quarter, we made progress in advancing our late-stage muscle biology-directed pipeline and are now preparing for our first NDA submission while two other programs are expected to proceed in pivotal Phase 3 trials this year," said Robert I. Blum, Cytokinetics’ President and Chief Executive Officer. "Results from REDWOOD-HCM and GALACTIC-HF demonstrate the potential of modulating cardiac myosin to improve outcomes in patients with HCM and heart failure for which there are no medical treatments that address underlying impaired contractility. Following our recent financing, we are also taking steps to build our commercial organization in anticipation of our first potential product launch next year in parallel with our continued clinical progress."

Q2 and Recent Highlights

Cardiac Muscle Programs

omecamtiv mecarbil (cardiac myosin activator)

Engaged with the U.S. Food and Drug Administration (FDA) in both a Type C meeting and a pre-NDA meeting to inform our plans to submit a New Drug Application (NDA) for omecamtiv mecarbil in 2H 2021. The submission will be based on GALACTIC-HF which demonstrated a positive effect on the primary composite endpoint of cardiovascular death or heart failure events in patients with heart failure and reduced ejection fraction who were receiving standard of care plus omecamtiv mecarbil.

Results from a secondary analysis of GALACTIC-HF (Global Approach to Lowering Adverse Cardiac Outcomes Through Improving Contractility in Heart Failure) were presented at the American College of Cardiology 70th Annual Scientific Session & Expo (ACC.21) showing that the treatment effect of omecamtiv mecarbil increased progressively as baseline ejection fraction decreased. The results were also published in the Journal of the American College of Cardiology.

Additional results from GALACTIC-HF were presented at Heart Failure 2021, an International Congress of the European Society of Cardiology demonstrating that the patients who derived greater treatment benefit from omecamtiv mecarbil included patients without atrial fibrillation or flutter, patients with higher baseline NT-proBNP and patients with severe heart failure based on modified criteria from the Heart Failure Association of the European Society of Cardiology (ESC-HFA) advanced heart failure position statement.

Completed enrollment in METEORIC-HF (Multicenter Exercise Tolerance Evaluation of Omecamtiv Mecarbil Related to Increased Contractility in Heart Failure), the second Phase 3 trial of omecamtiv mecarbil. We expect to complete conduct of METEORIC-HF by year end and report results in early 2022.

Expanded Medical Affairs team and activities. Hired medical directors and deployed Medical Science Liaisons in key U.S. locations. Organized framework for the Investigator Sponsored Study Program.

Established Go-to-Market-strategy and conducted commercial readiness activities, including organizational design, market research, forecasting, market access preparations and supply chain and logistics planning.
aficamten (CK-3773274, cardiac myosin inhibitor)

Received approval from the World Health Organization and the United States Adopted Name Council for aficamten to be used as the International Nonproprietary Name for CK-3773274.

Announced positive topline results from Cohorts 1 and 2 of REDWOOD-HCM (Randomized Evaluation of Dosing With CK-274 in Obstructive Outflow Disease in HCM) demonstrating that treatment with aficamten for 10 weeks resulted in statistically significant reductions from baseline compared to placebo in the average resting left ventricular outflow tract pressure gradient (LVOT-G) (p=0.0003, p=0.0004, Cohort 1 and Cohort 2, respectively) and the average post-Valsalva LVOT-G (p=0.001, p<0.0001, Cohort 1 and Cohort 2, respectively). The majority of patients treated with aficamten (78.6% in Cohort 1 and 92.9% in Cohort 2) achieved the target goal of treatment, defined as resting gradient <30 mmHg and post-Valsalva gradient <50 mmHg at Week 10 compared to placebo (7.7%). Treatment with aficamten in REDWOOD-HCM was generally well tolerated. The incidence of adverse events was similar between treatment arms. No serious adverse events were attributed to aficamten and no treatment interruptions occurred on aficamten, and no new cases of atrial fibrillation in patients treated with aficamten were reported.

Opened enrollment in Cohort 3 of REDWOOD-HCM for patients whose background therapy includes disopyramide. Activated the first site for enrollment in REDWOOD-HCM OLE, the open label extension clinical study designed to assess the long-term safety and tolerability of aficamten in patients with symptomatic obstructive HCM who have participated previously in REDWOOD-HCM.

Engaged FDA in a Type C meeting and subsequent end-of Phase 2 interaction to review the design of the planned Phase 3 clinical trial of aficamten in patients with obstructive HCM as well as the intended dosing strategy. Feedback was supportive of our objectives and progression.

Conducted preparations for a pivotal Phase 3 clinical trial of aficamten in patients with obstructive HCM, expected to begin in Q4 2021.

Ji Xing Pharmaceuticals continued enrolling patients in a Phase 1 study of aficamten in China and is preparing to participate in the planned Phase 3 clinical trial of aficamten in patients with obstructive HCM.

Presented scientific data related to the optimization of aficamten, including the first disclosure of its chemical structure, at the American Chemical Society Spring 2021 Virtual Meeting.
Skeletal Muscle Program

reldesemtiv (next-generation fast skeletal muscle troponin activator (FSTA))

Recently started COURAGE-ALS (Clinical Outcomes Using Reldesemtiv on ALSFRS-R in a Global Evaluation in ALS), the planned pivotal Phase 3 clinical trial of reldesemtiv in patients with ALS.
Pre-Clinical Development and Ongoing Research

Continued to advance new chemical entities and to conduct IND-enabling studies with expectation of our potentially advancing 1-2 potential drug candidates into clinical development over the next year.

Continued research activities directed to our other muscle biology research programs.
Corporate

Raised approximately $297.3 million in net proceeds, after deducting underwriting discounts and commissions from an underwritten public offering of 11,500,000 shares of common stock including the underwriter’s exercise of their overallotment option.

Executed agreements related to the termination of our Collaboration Agreement with Amgen and the transition to Cytokinetics of the development and commercialization rights for omecamtiv mecarbil and CK-136.

Announced the continuation of our partnership with The ALS Association in the fight against ALS.
Financials

Revenues for the three and six months ended June 30, 2021 were $2.8 million and $9.4 million, respectively, compared to $3.6 million and $7.4 million for the corresponding periods in 2020. The changes in revenues are due to changes in reimbursable collaborative activities with Amgen and Astellas.

Research and development expenses for the three and six months ended June 30, 2021 increased to $36.4 and $68.0 million, respectively, compared to $21.8 million and $43.5 million for the same periods in 2020. The changes were primarily due to increases in spending for COURAGE-ALS and our clinical development activities for our cardiac muscle inhibitor programs. In addition, this quarter, we incurred transition costs related to the termination of our collaboration with Amgen and the purchase from Amgen of approximately $7.3 million of materials including manufactured quantities of the active pharmaceutical ingredient for omecamtiv mecarbil.

General and administrative expenses for the three and six months ended June 30, 2021 increased to $21.2 million and $36.8 million from $14.2 million and $26.6 million in 2020 due primarily to an increase in personnel related costs including stock-based compensation and higher outside spending for commercial readiness.

Financial Guidance and Cash Runway

The company recently updated its financial guidance for 2021. We expect our revenues for 2021 will be in the range of $23 million to $28 million, our operating expenses will be in the range of $230 million to $250 million, and our net cash utilization will be in the range of $195 million to $215 million. This new guidance includes non-recurring new building construction costs of approximately $35 million and assumes receipt of $45 million under our funding agreement with RTW Investments, LP.

The company ended the second quarter with $424 million cash. With the common stock offering in July and our having raised approximately $297 million in net proceeds, we believe that we have more than three years of cash runway based on our revised 2021 cash utilization guidance.

Conference Call and Webcast Information

Members of Cytokinetics’ senior management team will review the company’s second quarter results via a webcast and conference call today at 4:30 PM Eastern Time. The webcast can be accessed through the Investors & Media section of Cytokinetics’ website at www.cytokinetics.com. The live audio of the conference call can also be accessed by telephone by dialing either (866) 999-CYTK (2985) (United States and Canada) or (706) 679-3078 (international) and typing in the passcode 7387377.

An archived replay of the webcast will be available via Cytokinetics’ website until August 19, 2021. The replay will also be available via telephone by dialing (855) 859-2056 (United States and Canada) or (404) 537-3406 (international) and typing in the passcode 7387377 from August 5, 2021 at 7:30 PM Eastern Time until August 19, 2021.

Bayer-strengthens-drug-discovery-platform-through-acquisition-of-Vividion-Therapeutics

On August 5, 2021 Bayer AG reported the acquisition of Vividion Therapeutics, Inc. (Vividion), a US-headquartered biopharmaceutical company utilizing novel discovery technologies to unlock high value, traditionally undruggable targets with precision therapeutics (Press release, Bayer, AUG 5, 2021, View Source [SID1234585848]). Vividion’s platform is able to produce a variety of small molecule therapies across indications, with initial focus on targets relevant to oncology and immunology. Vividion’s lead programs include multiple precision oncology targets and precision immunology targets, with ongoing efforts on a transcription factor NRF2 antagonist for the potential treatment of NRF2 mutant cancers, as well as NRF2 activators for various inflammatory diseases such as irritable bowel disease – among other pre-clinical programs.

Following closing of the acquisition, Bayer will own full rights to Vividion’s proprietary discovery platform, which comprises three integrated, synergistic components: a novel chemoproteomic screening technology, an integrated data portal, and a proprietary chemistry library. The acquisition of Vividion strengthens Bayer’s small molecule capabilities and expands Bayer’s reach into new modalities. Under the terms of the agreement, Bayer will pay an upfront consideration of USD 1.5 billion and potential success-based milestone payments of up to USD 500 million.

"This acquisition is a cornerstone of our strategy to fuel our pipeline with breakthrough innovation," said Stefan Oelrich, Member of the Board of Management, Bayer AG and President of the Bayer’s Pharmaceuticals Division. "Vividion’s technology is the most advanced in the industry, and it has demonstrated its ability to identify drug candidates that can target challenging proteins. Together with Bayer’s existing know-how, we will be able to develop first-in-class drug candidates, increasing the value of our pipeline. We want to provide innovative therapies for patients whose medical needs are not yet met by today’s treatment options."

Identification of drug candidates for proteins that are considered undruggable is a great challenge in drug discovery. Vividion’s chemoproteomic screening platform is able to identify previously unknown binding pockets on well-validated protein targets by screening chemical probes against the entire human proteome to assess selectivity. This yields highly potent and selective compounds that provide a wide therapeutic window for a variety of areas of high-unmet medical need. Vividion’s technology has already proven its applicability pre-clinically in oncology and immune-related diseases, and has the potential to expand into additional indications.

"Despite advances in genomics, structural biology and high-throughput screening, about 90% of disease-causing proteins cannot be targeted by current therapies due to the lack of a known addressable binding site. Our proprietary chemoproteomic platform technology addresses the key limitations of conventional screening techniques and allows us to discover previously unknown, or cryptic, functional pockets on the surface of proteins and identify small molecules that selectively bind to those targets," said Jeff Hatfield, Chief Executive Officer at Vividion. "When combined with Bayer’s expertise in the development of small molecules to market and patient, an unparalleled position comes into existence to unlock undruggable targets and generate first-in-class novel compounds for the benefit of patients."

To preserve its entrepreneurial culture as an essential pillar for nurturing successful innovation, Vividion will continue to operate as an independent organization on an arm’s length basis. Vividion will remain accountable to advance its technology and portfolio while benefiting from the experience, infrastructure and reach of Bayer as a global pharmaceutical company.

Closing of the transaction is contingent on customary closing conditions, including receipt of the required regulatory approvals, and is expected to take place in Q3 2021.

Credit Suisse is serving as financial advisor to Bayer, while Baker McKenzie is serving as legal counsel. Centerview Partners is serving as financial advisor to Vividion, while Cooley LLP is serving as legal counsel

FY2021 1Q Results

On August 5, 2021 Kureha Corporation reported that it FY2021 1Q Results (Press release, Kureha Corporation, AUG 5, 2021, View Source [SID1234585847])

Revenue recovered in Advanced Materials after the pandemic-related slump
• Higher core operating profit supported by Advanced Materials, Specialty Plastics and Other Operations
• Operating profit improved as a result of higher core operating profit
• Profit attributable to the Company increased as a result of higher profit before taxes

Advanced plastics Operating profit improved on higher sales volumes of PVDF, PPS and PGA: continued strong demand for PVDF binder in the automobile LiB market; enhanced production for PPS at a new facility; and PGA frac plugs sales steadily recovering in the post-pandemic shale oil and gas market Carbon products Operating profit remained flat despite higher sales volumes of carbon fiber insulation products for industrial high-heat furnacesAgrochemicals & Pharmaceuticals Sales volume growth of Kremezin, a therapeutic agent for chronic kidney failures, more than offset by a volume decline in agrochemical fungicides, together yielding lower operating profit Industrial chemicals Operating profit improved on higher sales volumes of organic chemicals

Home products / Fiber products Operating profit growth driven by higher ‘NEW Krewrap’ home products and ‘Seaguar’ fishing lines volumes Packaging materials Profit decreased due to higher raw materials cost despite improved revenue from heatshrink multilayer film, etc.

• The shale oil and gas market is gradually recovering as economic activity returns with rising oil prices
• Expanded the adaptation of PGA frac plugs in entire horizontal wells (full-bore use) at major customers by offering flexible volume price discounts since fall 2020
• Continues to promote the full-bore use and expand Kureha’s market share in high-/mid-temperature oilfields via flexible pricing and product design improvements, while developing improved PGA frac plugs for very low-temperature wells
• Aims to generate profit in the PGA business in FY2023 0 25 50 75 LiB binder application Other applications
• Robust PVDF sales continues on strong demand in the automobile LiB market since 3Q FY2020, driven by nations’ environment initiatives and subsidy policies promoting electric vehicles • Price negotiations underway to reflect rising raw materials cost
• Plans to increase the production of PVDF binder for automobile LiB applications at the Iwaki Factory, starting from February 2022
• Announces the decision to build a new PVDF plant (10,000tps) in Changshu, China on July 20, 2021

Applied DNA Schedules Fiscal 2021 Third Quarter Financial Results Conference Call and Webcast for Thursday, August 12, 2021

On August 5, 2021 Applied DNA Sciences, Inc. (NASDAQ: APDN) ("Applied DNA" or the "Company"), a leader in Polymerase Chain Reaction (PCR)- based DNA manufacturing and nucleic acid-based technologies, reported that it will report fiscal 2021 third quarter financial results after market close on Thursday, August 12, 2021 (Press release, Applied DNA Sciences, AUG 5, 2021, View Source [SID1234585846]). The Company’s management will discuss the results during a conference call and simultaneous webcast at 4:30 p.m. EDT that same day. Presentation slides will also be posted to the ‘IR Calendar and Corporate Presentations’ sub-page of the Company’s corporate website and embedded into the live webcast.

Conference Call and Webcast Information – Live
Date: Thursday, August 12, 2021, at 4:30 p.m. Eastern Daylight Time
Dial in: 844-887-9402, 412-317-6798 (international)
Hosts: Dr. James A. Hayward, chairman, president, and CEO, Beth Jantzen, chief financial officer
Webcast: View Source

Conference Call and Webcast Information – Replay
A telephonic replay of the conference call will be available for one week beginning one hour after the end of the live conference call.

Dial in: 877-344-7529, 412-317-0088 (international), Access Code: 10158254
Webcast: View Source
Availability: Telephonic replay: until Thursday, August 19, 2021; webcast replay: 1 year

The webcast and accompanying PowerPoint presentation will also be archived on the ‘IR Calendar and Corporate Presentations’ page listed under the Investor Relations drop-down menu on the Company’s website.

Moderna Reports Second Quarter Fiscal Year 2021 Financial Results and Provides Business Updates

On August 5, 2021 Moderna, Inc. (Nasdaq: MRNA), a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines, reported financial results and provided business updates for the second quarter of fiscal year 2021 (Press release, Moderna Therapeutics, AUG 5, 2021, View Source [SID1234585845]).

"I am proud of the progress our teams at Moderna have made in the past quarter in advancing our development pipeline while addressing a global pandemic and quickly establishing global manufacturing and commercial organizations," said Stéphane Bancel, Chief Executive Officer of Moderna. "We now have mRNA candidates in clinical trials across five therapeutic areas including infectious diseases, cardiovascular, oncology, rare disease and autoimmune disorders. We are pleased that our COVID-19 vaccine is showing durable efficacy of 93% through six months, but recognize that the Delta variant is a significant new threat so we must remain vigilant."

Looking forward, Bancel said, "We have begun preparing late stage studies for our flu vaccine and RSV vaccine, which received fast track designation from the FDA a few days ago and are looking forward towards our vision of a single dose annual booster that provides protection against COVID-19, flu and RSV for adults. I look forward to the start of our Phase 3 trial for CMV this year and to clinical proof of concept data in the coming quarters from our therapeutics pipeline. We believe this is just the beginning."

Updates and recent progress include:

COVID-19 Vaccine Development

Robust antibody responses have been observed from existing Moderna booster candidates against COVID-19 in Phase 2 studies
The Conditional Marketing Authorization (CMA) for Spikevax (Moderna’s COVID-19 Vaccine) in the European Union (EU) and authorization in Japan by the Ministry of Health, Labor and Welfare have been expanded to include adolescents 12 years of age and older
Moderna has initiated the rolling submission process for a Biologics License Application (BLA) for our vaccine in the U.S. and expects to complete its submission in August
Enrollment has completed for the Phase 1 study of mRNA-1283, Moderna’s next-generation COVID-19 vaccine, which is a potential refrigerator-stable vaccine that could facilitate easier distribution and storage
Infectious Diseases

Phase 3 study of cytomegalovirus (CMV) vaccine candidate (mRNA-1647) to begin in 2021
Dosing started in Phase 1/2 study of quadrivalent seasonal flu vaccine candidate (mRNA-1010)
Started dosing in Phase 2 study of Zika virus vaccine candidate (mRNA-1893)
Received Fast Track designation from U.S. Food and Drug Administration (FDA) for respiratory syncytial virus (RSV) vaccine candidate (mRNA-1345) for older adults (60 years of age and above)

Autoimmune Disorders

First participant dosed in the Phase 1 study of IL-2 mRNA program for autoimmune disorders (mRNA-6231)
Oncology

Discontinued further development of mRNA-2416, our standalone OX40L candidate; focus shifted to the development of mRNA-2752, which comprises mRNAs for OX40L plus two cytokines, IL23 + IL36γ.
Moderna currently has 23 mRNA development programs in its portfolio with 15 having entered clinical studies. The Company’s updated pipeline can be found at www.modernatx.com/pipeline. Moderna and collaborators have published more than 80 peer reviewed manuscripts.

Summary of Program Highlights by Modality

Core Modalities

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 threats to global public health. The Company’s global public health portfolio is focused on epidemic and pandemic diseases for which funding has been sought from governments and non-profit organizations.

COVID-19 Vaccine Development

Moderna COVID-19 Vaccine: Moderna has received emergency (or other conditional, interim or provisional) authorization for use of its COVID-19 vaccine in adults from health agencies in more than 50 countries and an Emergency Use Listing (EUL) from the World Health Organization (WHO), as well as authorization for use of its COVID-19 vaccine in adolescents age 12 and up in the European Union and Japan. On June 1, 2021, the Company initiated the rolling submission process for a Biologics License Application (BLA) for the vaccine in the U.S. and expects to complete its submission in August. Moderna is working with additional health agencies on the authorization and/or approval of its vaccine in additional jurisdictions. BARDA, part of the Office of the Assistant Secretary for Preparedness and Response (ASPR) within the U.S. Department of Health and Human Services (HHS), partially supported the research and development of the Moderna COVID-19 Vaccine with federal funding under Contract no. 75A50120C00034. Moderna retains worldwide rights to develop and commercialize the Moderna COVID-19 Vaccine.

Six months durability data: In final analysis of Phase 3 COVE study data, the Moderna COVID-19 Vaccine showed 93% efficacy, with the efficacy remaining durable through six months after administration of the second dose.
Addressing Variants of Concern: In a Phase 2 study, vaccination with 50 µg of three different Moderna mRNA booster candidates induced robust antibody responses against the wildtype D614G COVID-19 strain and against important variants of concern including Gamma (P.1); Beta (B.1.351); and Delta (B.1.617.2). The booster candidates included mRNA-1273, investigational mRNA-1273.351, and investigational mRNA-1273.211. Neutralizing antibody levels following the boost approached those observed after primary vaccination with two doses of 100 µg of mRNA-1273. These data have been submitted to a peer-reviewed journal for publication.
Further Clinical Studies of mRNA-1273

Phase 2/3 "TeenCOVE" study of mRNA-1273 in adolescents: The Phase 2/3 study of mRNA-1273 in adolescents ages 12-17 years completed enrollment in the U.S. An initial analysis of 3,732 participants randomized 2:1 in TeenCOVE Study showed a vaccine efficacy rate of 93% in seronegative participants who received at least one injection in a secondary analysis. The analysis included 15 cases (13 in the placebo group and 2 in the mRNA-1273 group) reported 14 days after first dose and based on the CDC definition of COVID-19, which requires one COVID-19 symptom and paired with a nasopharyngeal (NP) swab or saliva sample positive for SARS-CoV-2 by RT-PCR. The median duration for follow-up in this initial analysis was 53 days following the second dose. mRNA-1273 was generally well tolerated. The majority of adverse events were mild or moderate in severity. The most common solicited local adverse event was injection site pain. The most common solicited systemic adverse events after the second dose of mRNA-1273 were headache, fatigue, myalgia and chills. The Conditional Marketing Authorization (CMA) for Spikevax1 in the European Union (EU) has been expanded to include adolescents 12 years of age and older. In addition, the Japanese Ministry of Health, Labor and Welfare also approved Moderna Inc.’s COVID-19 vaccine for ages 12 to 17. Moderna has filed for an emergency use authorization (EUA) for adolescents with the U.S. Food and Drug administration as well as with additional regulatory agencies around the world.

Phase 2 "KidCOVE" study of mRNA-1273 in young children: The Phase 2 study of mRNA-1273 in pediatric population ages 6 months to 11 years is ongoing.

Phase 3 "COVE Transplant" study of mRNA-1273: The Phase 3 study of mRNA-1273 in adults with a kidney or liver transplant is ongoing, including the offer of a third vaccine dose to these immunocompromised participants

Next-generation vaccine against COVID-19 (mRNA-1283): The Phase 1 study of mRNA-1283 is fully enrolled and ongoing. mRNA-1283 is a next-generation vaccine candidate against COVID-19 that encodes for the portions of the SARS-CoV-2 spike protein critical for neutralization, specifically the Receptor Binding Domain (RBD) and N-terminal Domain (NTD). The encoded mRNA-1283 antigen is shorter than mRNA-1273 and is being developed as a potential refrigerator stable mRNA vaccine that will facilitate easier distribution and administration by healthcare providers.
Vaccines requiring complex antigens and against highly prevalent infections

Cytomegalovirus (CMV) vaccine (mRNA-1647): Positive seven-month data from the Phase 2 study assessing the safety, reactogenicity, and immunogenicity of different dose levels (50 μg, 100 μg and 150 μg) of mRNA-1647 were presented at Moderna’s annual Vaccines Day on April 14, 2021. Based on the interim analysis of the Phase 2 study, the 100 μg dose has been chosen for the Phase 3 pivotal study. The Company expects the Phase 3 study to begin in 2021. Moderna owns worldwide commercial rights for mRNA-1647.

Epstein-Barr virus (EBV) vaccine (mRNA-1189):mRNA-1189 is a vaccine against EBV containing five mRNAs that encode viral proteins (gp350, gB, gp42, gH and gL) in EBV. Similar to Moderna’s CMV vaccine (mRNA-1647), the viral proteins in mRNA-1189 are expressed in their native membrane-bound form for recognition by the immune system. Moderna is planning to begin a Phase 1 study of mRNA-1189 in 2021. There is no approved vaccine for EBV. Moderna owns worldwide commercial rights to mRNA-1189.
Vaccines against respiratory infections

Human metapneumovirus (hMPV) and parainfluenza type 3 (PIV3) vaccine (mRNA-1653): Moderna is enrolling seropositive pediatric participants (12-36 months of age) in the Phase 1 study of hMPV/PIV3 study (mRNA-1653). The first cohort in this study has been fully enrolled. Moderna owns worldwide commercial rights to mRNA-1653.

Respiratory syncytial virus (RSV) vaccine (mRNA-1345): mRNA-1345 is a vaccine against RSV encoding for a prefusion F glycoprotein, which elicits a superior neutralizing antibody response compared to the postfusion state. RSV is the leading cause of respiratory illness in young children. Older adults (65+) are at high risk for severe RSV infections. The Phase 1 study of mRNA-1345 to evaluate the tolerability and reactogenicity of mRNA-1345 in younger adults, women of child-bearing age, older adults and seropositive toddlers is ongoing. All four cohorts of younger adults (ages 18-49 years) are fully enrolled. Dosing in the older adult cohort (ages 65-79 years) is ongoing. The Company shared the first interim analysis of the Phase 1 study of mRNA-1345, through 1-month post-vaccination, of the younger adult cohorts at its annual Vaccines Day on April 14, 2021. The Company also intends to evaluate the potential of combinations of mRNA-1345 with its vaccines against other respiratory pathogens in children and separately in older adults. The FDA has granted Fast Track designation for mRNA-1345 in adults older than 60 years of age. There is no approved vaccine for RSV. Moderna owns worldwide commercial rights to mRNA-1345.
Seasonal influenza vaccine (mRNA-1010, mRNA-1020, mRNA-1030): Seasonal flu (type A and type B) epidemics occur seasonally and vary in severity each year, causing respiratory illnesses and placing substantial burden on healthcare systems. The World Health Organization (WHO) estimates approximately 3-5 million severe cases of flu each year globally, and 290,000-650,000 flu-related respiratory deaths. In the U.S., the estimated average economic burden of flu is approximately $11 billion per year. Current flu vaccines are only approximately 40-60% effective and their formulation is decided 9 months before the vaccines are intended to be used. The Company plans to explore potential combination vaccines against flu, SARS-CoV-2, RSV and human metapneumovirus (hMPV). The Company’s first-generation flu program will evaluate multiple candidates comprising multiple antigen combinations against the four seasonal viruses recommended by the WHO. The sentinel cohorts in the Phase 1/2 study of mRNA-1010 have been dosed.
Public health vaccines

Zika virus vaccine (mRNA-1893): The Phase 2 study of mRNA-1893 is enrolling participants. mRNA-1893 is being developed in collaboration with BARDA. Moderna owns worldwide commercial rights to mRNA-1893.
HIV vaccine (mRNA-1644 & mRNA-1574): HIV is the virus responsible for acquired immunodeficiency syndrome (AIDS), a lifelong, progressive illness with no effective cure. Approximately 38 million people worldwide are currently living with HIV with 1.2 million in the U.S. Approximately 2 million new infections of HIV are acquired worldwide every year and approximately 690,000 people die annually due to complications from HIV/AIDS. The primary routes of transmission are sexual intercourse and IV drug use, putting young adults at the highest risk of infection. From 2000 to 2015, a total of $562.6 billion globally was spent on care, treatment and prevention of HIV, representing a significant economic burden. mRNA-1644, a collaboration with the International AIDS Vaccine Initiative (IAVI) and the Bill and Melinda Gates Foundation, is a novel approach to HIV vaccine strategy in humans designed to elicit broadly neutralizing HIV-1 antibodies (bNAbs). A Phase 1 study for mRNA-1644 will use iterative human testing to validate the approach and antigens and multiple novel antigens will be used for germline-targeting and immuno-focusing. A second approach, mRNA-1574, is being evaluated in collaboration with the NIH and includes multiple native-like trimer antigens. The Company expects to begin Phase 1 studies for both mRNA-1644 and mRNA-1574 in 2021.

Nipah virus (NiV) Vaccine (mRNA-1215): NiV is a zoonotic virus transmitted to humans from animals, contaminated food, or through direct human-to-human transmission and causes a range of illnesses including fatal encephalitis. Severe respiratory and neurologic complications of NiV have no treatment other than intensive supportive care. The case fatality rate among those infected is estimated at 40-75%. NiV outbreaks cause significant economic burden to impacted regions due to loss of human life and interventions to prevent further spread, such as the slaughter of infected animals. NiV has been identified as the cause of isolated outbreaks in India, Bangladesh, Malaysia, and Singapore since 2000 and is included on the WHO R&D Blueprint list of epidemic threats needing urgent R&D action. mRNA-1215 was co-developed by Moderna and the NIH’s Vaccine Research Center (VRC).

Systemic Secreted & Cell Surface Therapeutics: In this modality, mRNA is delivered systemically to create proteins that are either secreted or expressed on the cell surface.

Antibody against the chikungunya virus (mRNA-1944): Positive interim data from the Phase 1 study evaluating escalating doses of mRNA-1944 in the 0.6 mg/kg dose with steroid premedication cohort and two doses of 0.3 mg/kg (without steroid premedication) given one week apart cohort were presented at Moderna’s annual R&D Day in September and demonstrated dose-dependent increases in levels of antibody against chikungunya. Safety and increased CHKV-IgG production in the two-dose regimen shows the platform’s ability for repeat dosing.

IL-2 (mRNA-6231): mRNA-6231 is an mRNA encoding for a long-acting tolerizing IL-2. This autoimmune development candidate is designed to preferentially activate and expand the regulatory T cell population. The first participant in the Phase 1 study of mRNA-6231 in healthy adult participants (between 18 and 50 years of age) has been dosed. mRNA-6231 uses the same LNP formulation as mRNA-1944. The Phase 1 study of mRNA-6231 will be the first clinical demonstration of subcutaneous administration of this delivery technology. Moderna owns worldwide commercial rights to mRNA-6231.

PD-L1 (mRNA-6981): mRNA-6981 is an mRNA encoding for PD-L1. This autoimmune development candidate is designed to augment cell surface expression of PD-L1 on myeloid cells to provide co-inhibitory signals to self-reactive lymphocytes. As an initial step to addressing a range of autoimmune indications, the Company intends to pursue proof-of-concept in a Phase 1 study of mRNA-6981 in type 1 autoimmune hepatitis (AIH), a condition that involves liver inflammation and can lead to cirrhosis and liver failure. mRNA-6981 uses the same LNP formulation as mRNA-1944. Moderna owns worldwide commercial rights to mRNA-6981.

Relaxin (AZD7970): Moderna has regained all rights to the Relaxin development candidate from AstraZeneca. Moderna now owns worldwide commercial rights to this development candidate.

Exploratory Modalities

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 vaccine (PCV) (mRNA-4157): The randomized Phase 2 study investigating a 1 mg dose of mRNA-4157 in combination with Merck’s pembrolizumab (KEYTRUDA), compared to pembrolizumab alone, for the adjuvant treatment of high-risk resected melanoma is ongoing. Phase 1 in multiple cohorts is ongoing. The upsized head & neck cohort is recruiting additional patients. Moderna shares worldwide commercial rights to mRNA-4157 with Merck.

Mutant KRAS vaccine (mRNA-5671 or V941): The Phase 1 open-label, multi-center study to evaluate the safety and tolerability of mRNA-5671 both as a monotherapy and in combination with pembrolizumab, led by Merck, is ongoing. Moderna shares worldwide commercial rights to mRNA-5671 with Merck.

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

OX40L/IL-23/IL-36γ (Triplet) (mRNA-2752): The Phase 1 trial evaluating mRNA-2752 as a single agent and in combination with durvalumab in patients with advanced solid tumor malignancies and lymphoma is ongoing. Moderna owns worldwide commercial rights to mRNA-2752.

IL-12 (MEDI1191): The Phase 1 open-label, multi-center study of intratumoral injections of MEDI1191 alone and in combination with durvalumab in patients with advanced solid tumors, led by AstraZeneca, is ongoing. MEDI1191 is an mRNA encoding for IL-12, a potent immunomodulatory cytokine. Moderna shares worldwide commercial rights to MEDI1191 with AstraZeneca.
Localized Regenerative Therapeutics: Localized production of proteins has the potential to be used as a regenerative medicine for damaged tissues.

VEGF-A (AZD8601): The Phase 2a study of AZD8601 VEGF-A, which is being developed for patients with ischemic heart disease undergoing coronary artery bypass grafting surgery with moderately impaired systolic function, led by AstraZeneca, is ongoing. Moderna has licensed worldwide commercial rights to AZD8601 to AstraZeneca.

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.

Propionic acidemia (PA) (mRNA-3927): The Phase 1/2 Paramount study of mRNA-3927 is ongoing. mRNA-3927 uses the same LNP formulation as mRNA-1944. This is the Company’s first development candidate in its systemic intracellular therapeutics modality to enter the clinic. Moderna owns worldwide commercial rights to mRNA-3927.

Methylmalonic acidemia (MMA) (mRNA-3705): Moderna received rare pediatric designation for its next generation MMA candidate (mRNA-3705). The Company plans to file new IND and CTA applications for mRNA-3705 and will focus development efforts on that candidate going forward. mRNA-3705 uses the same LNP formulation as mRNA-1944. Moderna owns worldwide commercial rights to mRNA-3705.

Phenylketonuria (PKU) (mRNA-3283): Individuals with PKU have a deficiency in phenylalanine hydroxylase (PAH) resulting in a reduced or complete inability to metabolize the essential amino acid phenylalanine into tyrosine. mRNA-3283 encodes human PAH to restore the deficient or defective intracellular enzyme activity in patients with PKU. mRNA-3283 is in preclinical development. Moderna owns worldwide commercial rights to mRNA-3283.
Glycogen storage disease type 1a (GSD1a) (mRNA-3745): Individuals with GSD1a have a deficiency in glucose-6-phosphatase resulting in pathological blood glucose imbalance. mRNA-3745 is an IV-administered mRNA encoding human G6Pase enzyme, designed to restore the deficient or defective intracellular enzyme activity in patients with GSD1a. mRNA-3745 is in preclinical development. Moderna owns worldwide commercial rights to mRNA-3745.
Information about each development candidate in Moderna’s pipeline can be found at investors.modernatx.com.

Second Quarter 2021 Financial Results

Revenue: Total revenue was $4.4 billion for the three months ended June 30, 2021, compared to $67 million for the same period in 2020. Total revenue was $6.3 billion for the six months ended June 30, 2021, compared to $75 million for the same period in 2020. Total revenue increased in 2021, resulting from commercial sales of the Company’s COVID-19 vaccine, and to a lesser extent, grant revenue. Product sales for the three and six months ended June 30, 2021 was $4.2 billion and $5.9 billion, respectively, from sales of 199 million and 302 million doses of the Company’s COVID-19 vaccine. The increases in grant revenue of $101 million and $291 million for the three and six months ended June 30, 2021, respectively, were primarily driven by increases in revenue from BARDA related to the Company’s COVID-19 vaccine development.
Cost of Sales: Cost of sales was $750 million, or 18%, of product sales for the three months ended June 30, 2021, including third-party royalties of $148 million. Cost of sales was $943 million, or 16%, of the Company’s product sales, for the six months ended June 30, 2021, including third-party royalties of $232 million. A portion of the inventory costs associated with the Company’s product sales for the six months ended June 30, 2021 was expensed as pre-launch inventory costs in 2020. At the end of the first quarter of 2021, the Company’s zero-cost COVID-19 vaccine inventory was substantially utilized. If inventory sold for the six months ended June 30, 2021 was valued at cost, the Company’s cost of sales for the period would have been $1.1 billion, or 19% of product sales.
Research and Development Expenses: Research and development expenses were $421 million for the three months ended June 30, 2021, compared to $152 million for the same period in 2020. Research and development expenses were $822 million for the six months ended June 30, 2021, compared to $267 million for the same period in 2020. The growth in spending in 2021 was mainly due to increases in clinical trial expenses, and to a lesser extent, personnel-related costs, manufacturing expenses, and consulting and outside services, largely driven by increased mRNA-1273 clinical development and headcount.
Selling, General and Administrative Expenses: Selling, general and administrative expenses were $121 million for the three months ended June 30, 2021, compared to $37 million for the same period in 2020. Selling, general and administrative expenses were $198 million for the six months ended June 30, 2021, compared to $61 million for the same period in 2020. The growth in spending in 2021 was mainly due to increases in consulting and outside services, personnel-related costs, and marketing expenses, primarily attributable to the Company’s COVID-19 vaccine commercialization-related activities and increased headcount.
Net Income (Loss):Net income was $2.8 billion for the three months ended June 30, 2021, compared to a net loss of $(117) million for the same period in 2020. Net income was $4.0 billion for the six months ended June 30, 2021, compared to a net loss of $(241) million for the same period in 2020.
Cash Position: Cash, cash equivalents and investments as of June 30, 2021 and December 31, 2020 were $12.2 billion and $5.2 billion, respectively.
Net Cash Provided By (Used In) Operating Activities: Net cash provided by operating activities was $7.0 billion for the six months ended June 30, 2021, compared to $(130) million used in operating activities for the same period in 2020. Net cash provided by operating activities increased significantly in 2021, mainly due to net income of $4.0 billion and additional customer deposits received during the period for the Company’s future COVID-19 vaccine supply.
Cash Used for Purchases of Property and Equipment: Cash used for purchases of property and equipment was $65 million for the six months ended June 30, 2021, compared to $25 million for the same period in 2020.
2021 Updated Financial Framework

For Expected Delivery in Fiscal Year (FY) 2021: Advance Purchase Agreements (APAs) signed for anticipated product sales of $20 billion, including sales already recorded in the six months ended June 30, 2021.
Dose Capacity for FY 2021: The Company expects dose capacity for its COVID-19 vaccine in FY 2021 to be between 800 million and 1 billion doses.
For Expected Delivery in FY 2022: The Company has already signed APAs for product sales of approximately $12 billion and options of approximately $8 billion. Numerous additional negotiations are still ongoing for 2022 APAs.
For Expected Delivery in 2023: The Company has also started to sign APAs for 2023 as forward-looking countries prepare for the endemic phase of COVID-19.
Dose Capacity for FY 2022: The Company expects dose capacity for its COVID-19 vaccine in FY 2022 to be between 2 billion to 3 billion doses, subject to dose level.
Cost of Sales: Cost of sales as percentage of product sales are expected to be between 18-20% for fiscal year 2021.
2021 Research & Development (R&D) and Selling, General & Administrative (SG&A) Expenses: Continue to expect quarter over quarter cost increases in R&D and SG&A expenses during 2021 as commercial and research and development activities and expenses ramp up.
Tax Rate: The Company now expects the effective tax rate for 2021 to be approximately 10% as a result of the forecasted global sales mix and utilization of the accumulated net operating loss carry-forward of $2.3 billion.
Capital Expenditures: Continue to expect $450-550 million of capital investments for 2021 including the planned capacity expansion announced in April 2021.
Share Repurchase Program: The Board of Directors has authorized a share repurchase program of up to $1 billion over a two-year period to return excess capital to shareholders.
Further Investments: The Company plans to continue to invest in its technology platform and in potential new modalities. The Company will also consider attractive strategic opportunities that further enable and complement our platform.
Management Updates

Shannon Thyme Klinger joined the Company as Chief Legal Officer and Corporate Secretary on June 1, 2021. Ms. Klinger joined Moderna from Novartis (NYSE: NVS), where she served as Chief Legal Officer and a member of the Novartis Executive Committee since 2018. Previously, she served as Chief Ethics, Risk & Compliance Officer. During her ten-year tenure at Novartis, she held other roles of increasing responsibility, including as Chief Ethics and Compliance Officer and Global Head of Litigation, General Counsel and Global Head of Legal at Sandoz, a Novartis division.
Paul Burton M.D., Ph.D., F.A.C.C, M.R.C.S joined the Company as Chief Medical Officer on July 6, 2021. Dr. Burton joined Moderna after spending sixteen years with Johnson & Johnson. Since March 2020, he served as Chief Global Medical Affairs Officer of Janssen Pharmaceuticals where he was responsible for Janssen’s worldwide medical affairs strategy and execution. Previously, he served as Janssen’s Vice President and Head, Cardiovascular and Metabolic Medical Affairs.

Kate Cronin joined Moderna as Chief Brand Officer on July 12, 2021. Ms. Cronin joined Moderna from Ogilvy Health, part of WPP plc., where she served as Global CEO and led the full spectrum of Ogilvy Health’s core capabilities including public relations and influence, brand strategy, advertising, medical education, market access, and patient and consumer engagement. Additionally, Ms. Cronin grew Ogilvy’s business in the health and wellness arena, encompassing a broad portfolio including pharmaceuticals, consumer health, insurance, hospitals, health technology and medical devices.
Corporate Updates

Full-Time Employees: As of June 30, 2021, Moderna had approximately 1,800 employees, compared to approximately 930 employees as of June 30, 2020.
Moderna Charitable Foundation: Moderna announced it is establishing a new charitable foundation with an initial endowment of $50 million to promote public health, healthcare and educational opportunities, particularly in underserved populations.
Moderna International Business Services (MIBS) Center: Moderna announced plans to establish an international business services hub in Warsaw, Poland, as the Company continues to build out a global infrastructure.
Science Day: Moderna hosted its fourth annual Science Day on May 27.
Job Creation in Massachusetts: Moderna reaffirmed its commitment to job creation in Massachusetts.
S&P 500: Moderna was added to the Standard & Poor’s (S&P) 500 index on Wednesday, July 21.
Company Recognition: Moderna was named number one on Fast Company’s 2021 Best Workplaces for Innovators list and number three on the Axios/Harris 2021 Poll of Corporate Reputation Rankings.
Key 2021 Investor and Analyst Event Dates

R&D Day – September 9
Investor Call and Webcast Information

Moderna will host a live conference call and webcast at 8:00 a.m. ET on Thursday, August 5, 2021. To access the live conference call, please dial 866-922-5184 (domestic) or 409-937-8950 (international) and refer to conference ID 5749439. A webcast of the call will also be available under "Events and Presentations" in the Investors section of the Moderna website at investors.modernatx.com. The webcast and slides are available directly at: View Source The archived webcast will be available on Moderna’s website approximately two hours after the conference call and will be available for one year following the call.