On August 5, 2021 Novavax, Inc. (NASDAQ: NVAX), a biotechnology company developing next-generation vaccines for serious infectious diseases, reported its financial results and operational highlights for the second quarter ended June 30, 2021 (Press release, Novavax, AUG 5, 2021, View Source [SID1234586007]).

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"We are highly encouraged by the filing of regulatory submissions in multiple markets, made in partnership with Serum Institute of India. We view these submissions as the first of many filings to come, which will allow NVX-CoV2373 to be made available at a global scale," said Stanley C. Erck, President and Chief Executive Officer, Novavax. "Our clinical successes over the second quarter reaffirm our confidence in NVX-CoV2373’s differentiated efficacy profile. We continue to see the circulation of new variants and inequitable access to vaccine globally, demanding that we bring our COVID-19 vaccine to market as swiftly as possible."

Second Quarter 2021 and Recent Highlights

COVID-19 Vaccine Clinical and Non-Clinical Development

Reported final analysis from PREVENT-19 U.S. and Mexico Phase 3 trial
Achieved primary efficacy endpoint with overall efficacy of 90.4% against mild, moderate, and severe disease
Demonstrated 100% protection against moderate and severe disease
Demonstrated 91.0% efficacy among high-risk populations
Demonstrated 92.6% efficacy against Variants of Concern/Variants of Interest (VoC/VoI) and 100% efficacy against variants not considered VoC/VoI
Completed enrollment of pediatric expansion of PREVENT-19 Phase 3 trial
Enrolled 2,248 adolescents aged 12-17 across up to 75 sites in the U.S.
Blinded crossover expected to begin in August 2021 to ensure all participants have access to active vaccine
Published results from late-stage South Africa Phase 2b and UK Phase 3 trials in The New England Journal of Medicine
Completed UK Phase 3 crossover arms and initiated crossover studies in PREVENT-19 and South Africa Phase 2b trials to ensure all participants have access to active vaccine
Advanced clinical development of booster studies of NVX-CoV2373
Reported 6-month booster data from U.S. and Australia Phase 2 trial
Wild-type neutralizing antibodies increased more than 4-fold versus primary vaccination series
Cross-reactive functional antibodies to the Delta (B.1.617.2) variant strain detected after primary vaccination series and increased more than 6-fold following boosting
Analysis of sera from primary series immunization showed cross-reactive functional antibodies to the Alpha (B.1.1.7), Beta (B.1.351), and Delta (B.1.617.2) variant strains, all of which increased 6- to 10-fold with a booster dose
Participated in two UK Vaccines Taskforce-supported studies evaluating heterologous vaccination (Com-COV2) and heterologous boosting (CoV-Boost)
COVID-19 Vaccine Regulatory Pathway

Filed regulatory submissions in partnership with Serum Institute of India Pvt. Ltd. (SII) for emergency use authorization in multiple markets
Submitted regulatory filings with the Drugs Controller General of India (DCGI) and regulatory agencies in Indonesia and the Philippines
Expect to file for Emergency Use Listing with the World Health Organization in August 2021
Expect to complete regulatory filing with the UK Medicines and Healthcare products Regulatory Agency (MHRA) in the third quarter of 2021
Expect to complete additional regulatory filings in other markets within weeks of MHRA filing, including with the European Medicines Agency (EMA), Australian Therapeutic Goods Administration, Health Canada, and New Zealand Medsafe
Expect to submit for emergency use authorization to the U.S. Food and Drug Administration in the fourth quarter of 2021
COVID-19 Vaccine Manufacturing and Supply

Collaborated with partners globally to progress toward anticipated manufacturing capacity
On track to achieve capacity of 100 million doses per month by the end of the third quarter of 2021 and 150 million doses per month by the end of the fourth quarter 2021
Initiated technology transfer at National Research Council of Canada Biologics Manufacturing Centre to produce NVX-CoV2373
Expanded agreements globally to ensure equitable access to low-, middle-, and high-income countries
Entered into advance purchase agreement with Gavi, the Vaccine Alliance, to provide 1.1 billion doses to the COVAX Facility
Novavax to manufacture and distribute 350 million doses
SII to manufacture and distribute the remaining balance of the 1.1 billion doses to low- and middle-income countries
Finalized terms of advance purchase agreement with European Commission to supply up to 100 million doses with the option for an additional 100 million doses through 2023
Delivery of doses to begin following anticipated regulatory approval from EMA
NanoFluTM / NVX-CoV2373 Combination Vaccine (qNIV/CoV2373)

Announced preclinical data for qNIV/CoV2373 and expect to initiate Phase 1 clinical trial in Australia later this year
qNIV/CoV2373 induced strong functional antibodies, high levels of anti-S IgG, and neutralizing antibody titers
Preclinical data manuscript submitted for publication to a peer-reviewed journal and posted via the preprint server on bioRxiv.org
Announced data from sub-study in UK Phase 3 trial, supporting co-administration of NVX-CoV2373 with influenza vaccination
431 participants received approved seasonal influenza vaccine, while half of those participants were co-vaccinated with NVX-CoV2373
Co-administration did not negatively impact influenza immune response for any of the four influenza strains in the quadrivalent influenza vaccine
Confirmed efficacy trend of 87.5% against COVID-19
Data manuscript submitted for publication to a peer-reviewed journal and posted via the preprint server on medRxiv.org
Malaria Vaccine / Matrix-M Adjuvant Collaboration

Ongoing clinical development for R21, the University of Oxford’s malaria vaccine candidate formulated with Matrix-M adjuvant, in collaboration with SII
Phase 3 licensure trial underway in 4,800 participants, aged 5-36 months
Phase 2b clinical trial results demonstrated 77% efficacy and were published in Preprints with The Lancet
Financial Results for the Three Months Ended June 30, 2021

Novavax reported a net loss of $352 million, or $4.75 per share, for the second quarter of 2021, compared to a net loss of $18 million, or $0.30 per share, for the second quarter of 2020.

Novavax revenue in the second quarter of 2021 was $298 million, compared to $36 million in the same period in 2020. This increase was due to increased development activities relating to NVX-CoV2373 for services performed under the U.S. government and Coalition for Epidemic Preparedness Innovations agreements.

Research and development expenses increased to $571 million in the second quarter of 2021, compared to $35 million in the same period in 2020. The increase was primarily due to the development of NVX-CoV2373.

General and administrative expenses increased to $73 million in the second quarter of 2021, compared to $18 million for the same period in 2020. The increase was primarily due to increased employee-related costs, stock-based compensation expenses, and professional fees supporting our NVX-CoV2373 program.

As of June 30, 2021, Novavax had $2.1 billion in cash, cash equivalents and restricted cash, compared to $806 million as of December 31, 2020. Net cash provided by operating activities for the first six months of 2021 was $807 million, compared to $93 million for the same period in 2020. The increase in cash provided was primarily due to $1.1 billion in payments under advance purchase agreements recorded as deferred revenue and the timing of payments to third parties.

Through utilization of At-the-Market (ATM) offerings during the first six months of 2021, Novavax raised net proceeds of $565 million.

Conference Call

Novavax will host its quarterly conference call today at 4:30 p.m. ET. The dial-in numbers for the conference call are (866) 652-5200 (Domestic) or (412) 317-6060 (International). Participants will be prompted to request to join the Novavax, Inc. call. A replay of the conference call will be available starting at 7:30 p.m. ET on August 5, 2021 until 7:30 p.m. ET on November 12, 2021. To access the replay by telephone, dial (877) 344-7529 (Domestic) or (412) 317-0088 (International) and use passcode 10158313.

A webcast of the conference call can also be accessed on the Novavax website at novavax.com/events. A replay of the webcast will be available on the Novavax website until November 12, 2021.

About NVX-CoV2373

NVX-CoV2373 is a protein-based vaccine candidate engineered from the genetic sequence of the first strain of SARS-CoV-2, the virus that causes COVID-19 disease. NVX-CoV2373 was created using Novavax’ recombinant nanoparticle technology to generate antigen derived from the coronavirus spike (S) protein and is formulated with Novavax’ patented saponin-based Matrix-M adjuvant to enhance the immune response and stimulate high levels of neutralizing antibodies. NVX-CoV2373 contains purified protein antigen and can neither replicate, nor can it cause COVID-19. In preclinical studies, NVX-CoV2373 induced antibodies that blocked the binding of spike protein to cellular receptors and provided protection from infection and disease. It was generally well-tolerated and elicited robust antibody response in Phase 1/2 clinical testing.

NVX-CoV2373 is being evaluated in two pivotal Phase 3 trials: a trial in the U.K. that demonstrated efficacy of 96.4% against the original virus strain, 86.3% against the Alpha (B.1.1.7) variant and 89.7% efficacy overall; and the PREVENT-19 trial in the U.S. and Mexico that demonstrated 100% protection against moderate and severe disease and 90.4% efficacy overall. It is also being tested in two ongoing Phase 2 studies that began in August 2020: A Phase 2b trial in South Africa that demonstrated 55% efficacy overall in HIV-negative participants and 48.6% efficacy against a newly emerging escape variant first described in South Africa, and a Phase 1/2 continuation in the U.S. and Australia.

NVX-CoV2373 is stored and stable at 2°- 8°C, allowing the use of existing vaccine supply chain channels for its distribution. It is packaged in a ready-to-use liquid formulation in 10-dose vials.

About NanoFlu

NanoFlu is a recombinant hemagglutinin (HA) protein nanoparticle influenza vaccine produced by Novavax in its SF9 insect cell baculovirus system. NanoFlu uses HA amino acid protein sequences that are the same as the recommended wild-type circulating virus HA sequences. NanoFlu contains Novavax’ patented saponin-based Matrix-M adjuvant.

About Matrix-M Adjuvant

Novavax’ patented saponin-based Matrix-M adjuvant has demonstrated a potent and well-tolerated effect by stimulating the entry of antigen-presenting cells into the injection site and enhancing antigen presentation in local lymph nodes, boosting immune response.

Regeneron has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission .

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On August 5, 2021 CytomX Therapeutics, Inc. (Nasdaq: CTMX), a clinical-stage oncology-focused biopharmaceutical company pioneering a novel class of investigational conditionally activated therapeutics based on its Probody technology platform, reported second quarter 2021 financial results and provided a business update (Press release, CytomX Therapeutics, AUG 5, 2021, View Source [SID1234585977]).

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"In the second quarter of 2021, we continued to advance our broad pipeline of Probody therapeutics across multiple modalities and cancer types. Our Phase 2 studies, evaluating our two lead conditionally activated antibody-drug conjugates, praluzatamab ravtansine (CX-2009), targeting CD166, and CX-2029, targeting CD71, are ongoing," said Sean McCarthy, D.Phil., president, chief executive officer and chairman of CytomX Therapeutics. "The breadth of our progress and depth of our science have been highlighted by five clinical and scientific publications in peer-reviewed journals in 2021 to date, including a landmark preclinical publication demonstrating the power of Probody technology to increase the therapeutic window of an immune agonist," added Dr. McCarthy.

Business Highlights and Recent Developments

Patient enrollment is ongoing in the Phase 2 study of praluzatamab ravtansine (CX-2009), our wholly-owned CD166-directed conditionally activated antibody-drug conjugate (ADC), being evaluated as a monotherapy in patients with human epidermal growth factor receptor 2-non-amplified breast cancer and in combination with pacmilimab (CX-072), in patients with triple-negative breast cancer. Due primarily to impacts from the COVID-19 pandemic, including slower clinical site activation and patient enrollment, CytomX now anticipates initial data from this study in 2022.
In collaboration with AbbVie, the multi-cohort Phase 2 study of CX-2029, the CD71-directed conditionally activated ADC, continues to enroll patients into the expansion cohorts evaluating the following indications: squamous non-small cell lung cancer, head and neck squamous cell carcinoma, esophageal and gastro-esophageal junction cancers, and diffuse large B-cell lymphoma. Initial data from this study is anticipated in the fourth quarter of 2021.
Our collaboration partner, Bristol Myers Squibb, continues to study the combination of BMS-986249, a Probody version of ipilimumab, and nivolumab, the anti-PD-1 antibody, in four cancer types: metastatic melanoma, advanced hepatocellular carcinoma, metastatic castration-resistant prostate cancer, and advanced triple-negative breast cancer. Bristol Myers Squibb is also evaluating BMS-986288, a Probody version of non-fucosylated ipilimumab, as monotherapy or in combination with nivolumab, in a Phase 1 study.
CytomX submitted a pre-investigational new drug application (IND) meeting request to the U.S. Food and Drug Administration (FDA) for CX-904 in collaboration with Amgen and expects a written response from the FDA in the third quarter of 2021. CytomX will continue to discuss the program with Amgen and is working toward the filing of an IND in late 2021.
Ongoing research and pre-clinical development activities continue, towards the generation of conditionally activated cytokine therapeutics for the treatment of cancer, including interferon alpha-2b.
Five peer-reviewed manuscripts have been published highlighting progress across the CytomX pipeline and platform:
Pacmilimab (CX-072):
First-in-human biodistribution study using positron emission tomography imaging in Clinical Cancer Research. This is the first human clinical imaging report of a Probody therapeutic and further supports mechanistic aspects of platform performance including target engagement in the tumor and reduced target engagement in normal tissues. This article can be accessed using this link.
First-in-human monotherapy study in patients with advanced solid tumors in the Journal for ImmunoTherapy of Cancer. In this study, pacmilimab demonstrated single-agent activity in advanced solid tumors, including metastatic triple-negative breast cancer. This article can be downloaded using this link.
First-in-human study in combination with ipilimumab, the anti-cytotoxic T lymphocyte-associated antigen-4 antibody, in advanced solid tumors, also in the Journal for ImmunoTherapy of Cancer. The combination of pacmilimab and ipilimumab illustrates the potential for pacmilimab as a preferred checkpoint inhibitor for combination therapies. This article can be downloaded using this link.
CX-2029
First-in-human data in patients with advanced solid tumors in the peer-reviewed journal Clinical Cancer Research, demonstrating, for the first time, that CD71 can be a therapeutic cancer target for a masked drug-conjugated antibody . This article can be downloaded using this link.
Preclinical:
Preclinical studies of a novel Probody immuno-oncology agent targeting CD137 in Proceedings of the National Academy of Sciences. This is the first published application of the CytomX platform to agonist antibodies in immuno-oncology. This article can be accessed using this link.
Second Quarter 2021 Financial Results
Cash, cash equivalents and short- and long-term investments totaled $366 million as of June 30, 2021, compared to $316 million as of December 31, 2020.

Revenue was $16 million for the three months ended June 30, 2021, relatively flat when compared to the corresponding period in 2020.

Research and development expenses increased $2 million during the three months ended June 30, 2021 to $26 million compared to the corresponding period in 2020. The increase was driven mainly by timing of manufacturing and tissue sampling activities.

General and administrative expenses were $9 million for the three months ended June 30, 2021, essentially flat compared to the second quarter of 2020.

Conference Call & Webcast Information
CytomX management will host a conference call today at 5:00 p.m. ET (2:00 p.m. PT). Interested parties may access the live webcast of the conference call from the Events and Presentations page of CytomX’s website at www.cytomx.com or by dialing 1-877-809-6037 (U.S. and Canada) or 1-615-247-0221 (International) using the passcode 1488138. An archived replay of the webcast will be available on the Company’s website until August 12, 2021.

On August 5, 2021 Keros Therapeutics, Inc. ("Keros" or the "Company") (Nasdaq: KROS), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of novel treatments for patients suffering from hematological and musculoskeletal disorders with high unmet medical need, reported financial results for the quarter ended June 30, 2021 (Press release, Keros Therapeutics, AUG 5, 2021, View Source [SID1234585976]).

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"We have made substantial progress over the past quarter, including announcing our preliminary results from our Phase 2 clinical trial of KER-050," said Jasbir S. Seehra, Ph.D., President and Chief Executive Officer. "We continue to make notable progress advancing our pipeline, and remain focused on the execution of our near-term development plans. In that regard, we remain on track to initiate several clinical trials for KER-050, KER-047 and KER-012 over the balance of 2021."

Recent Corporate Highlights:

Executive leadership changes: In August 2021, the Company announced that Simon Cooper, M.B.B.S., has been appointed to serve as the Company’s Chief Medical Officer, effective as of August 2, 2021. Dr. Cooper succeeded Claudia Ordonez, M.D., who departed from the Chief Medical Officer position effective July 30, 2021. Dr. Ordonez will provide consulting services to the Company until September 15, 2021.
Issuance of Keros foundational U.S. patent: In May 2021, the United States Patent and Trademark Office issued U.S. Patent No. 11,013,785, which is a composition of matter patent directed to novel therapeutic proteins, including KER-050, that contains 20 claims and expires in November 2037.
Recent Program Highlights:

KER-050 for the treatment of ineffective hematopoiesis to address cytopenias
In June 2021, Keros announced preliminary results from Cohorts 1 and 2 (0.75 mg/kg and 1.5 mg/kg) of Part 1 of its Phase 2 clinical trial evaluating KER-050 in patients with myelodysplastic syndromes. Following Safety Review Committee recommendation, dosing for Cohort 3 of the trial was initiated at 2.5 mg/kg of KER-050, to be administered once every four weeks for 12 weeks.
The Company expects to report additional Part 1 data and initiate Part 2 of the trial by the end of 2021.
Second Quarter 2021 Financial Results

Keros reported a net loss of $15.6 million in the second quarter of 2021 as compared to a net loss of $10.8 million in the second quarter of 2020. The increase in net loss for the second quarter was largely due to increased research and development efforts as well as additional infrastructure expenses
to support our operations as a publicly traded company.

Research and development expenses were $10.0 million for the second quarter of 2021 as compared to $7.3 million for the same period in 2020. The increase of $2.7 million was primarily due to additional research and development efforts, manufacturing activities, and personnel expenses to support the advancement of our pipeline.

General and administrative expenses were $5.7 million for the second quarter of 2021 as compared to $3.7 million for the same period in 2020. The increase of $2.0 million was primarily due to increase in personnel expenses and other external expenses to support Keros’ organizational growth.

Keros’ cash and cash equivalents as of June 30, 2021 was $237.1 million compared to $265.9 million as of December 31, 2020. Keros expects that the cash and cash equivalents it had on hand at June 30, 2021 will fund its operating expenses and capital expenditure requirements into the fourth quarter of 2023.

On August 5, 2021 Athersys, Inc. (NASDAQ: ATHX) and HEALIOS K.K. (Healios) (TSE Mothers: 4593) jointly reported expansion and deepening of their partnership to optimize and better align the collaboration structure to drive therapeutic reach and commercial success in Japan for the MultiStem (invimestrocel) product following potential regulatory approval (Press release, Athersys, AUG 5, 2021, View Source [SID1234585953]). The changes and new agreements reflect improved clarity regarding Japanese regulatory, manufacturing, and commercial requirements gained in recent years and better enable the optimal investments and efforts in manufacturing and commercialization. The agreements will facilitate the regulatory approval process for MultiStem in Japan, prepare the companies for commercial manufacturing and supply and expand the overall scope of collaboration between the companies.

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"We are happy to have reached this Comprehensive agreement with Healios which strengthens the alignment, commitment, and motivation of both companies and improves the potential for MultiStem commercial success in Japan," stated Mr. William (B.J.) Lehmann, Jr., President, and Interim CEO of Athersys. "This is an important step for us as it brings us closer to achieving our first regulatory approval and providing supply for commercialization in a key market and meeting our goal of bringing life-saving therapies to patients worldwide."

Dr. Hardy TS Kagimoto, Chairman and CEO of Healios, commented, "We have come a long way in building a stronger, mutually beneficial, win-win partnership between Athersys and Healios. With the improved relationship and clarification of the roles, responsibilities, and incentives as reflected in the new agreements, we aim to accelerate the development, regulatory approval, and delivery of life-saving treatments for patients. We are committed to achieving our mission: ‘Life Explosion!’ by delivering cures for patients with unmet medical needs."

"I would like to compliment and express my appreciation to the boards and management of both Healios and Athersys for their dedication and commitment in working through challenging issues to achieve this important milestone for the benefit of patients dealing with severe medical issues such as acute respiratory distress syndrome and stroke and for the benefit of shareholders of both Athersys and Healios," added Dr. Ismail Kola, Chairman of the Board of Athersys. "This is truly a win-win enhancement to the partnership."

Key elements of the improved collaboration include:

Providing Healios access to Athersys’s manufacturing technology to enable Healios to manufacture MultiStem products, using a qualified manufacturer, for a potential commercial launch in Japan and allow Athersys to focus resources on advanced commercial manufacturing development.
Clarifying Athersys’s role in providing support services necessary for regulatory approvals, manufacturing readiness, and commercial launch in Japan.
Sharing investment in commercial preparation and product supply through planned investment by Healios in certain manufacturing preparation activities and additional production capacity for Japan and, through deferrals and certain adjustments to financial terms of the license agreement, including milestones and royalties, during the early commercial phase.
Expanding Healios’ license in Japan to include two new additional indications under certain conditions to enable Healios to further leverage its investment in MultiStem while providing Athersys the opportunity for additional revenues from this market.
Increasing alignment between the companies and creating incentives for accelerated execution and investment, through $8 million in new milestone payments available to Athersys tied to certain Japan commercial manufacturing activities and the establishment of large scale manufacturing relevant to Japan, and through warrants issued to Healios to purchase up to a total of 10 million shares of Athersys common stock at a premium to the current market price and exercisable for 60 days following regulatory approval for ARDS and ischemic stroke, respectively.