On May 10, 2021 BWXT Medical Ltd. (BWXT Medical) reported that it has entered into a new long-term, mutually exclusive agreement to manufacture TheraSphere Y-90 Glass Microspheres for Boston Scientific, a leading global medical device company (Press release, BWXT Medical, MAY 10, 2021, View Source [SID1234579625]).

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Developed for the treatment of patients with hepatocellular carcinoma (HCC), TheraSphere treatment is comprised of millions of glass microspheres containing radioactive Yttrium-90, which are delivered directly to liver tumors via catheter and result in minimal exposure to surrounding healthy tissue.

BWXT Medical is a subsidiary of BWX Technologies, Inc. (NYSE: BWXT) and a global supplier of medical isotopes and radiopharmaceuticals.

Under the terms of the agreement, BWXT Medical will invest to automate the production process at its Ottawa, Canada facility, and thereby significantly increase capacity and dependability to support a growing global demand for TheraSphere.

"We congratulate Boston Scientific on its recent receipt of FDA PMA approval of TheraSphere, which expands patient access to this unique technology," said Martyn Coombs, president of BWXT Medical. "We share the aspiration of making a significant difference in the lives of people suffering with cancer, and we look forward to continuing to build a strong supply chain foundation for future growth."

On May 10, 2021 IDEAYA Biosciences, Inc. (NASDAQ: IDYA), a synthetic lethality-focused precision medicine oncology company committed to the discovery and development of targeted therapeutics, reported that dose expansion of the ongoing Phase 1/2 study (ClinicalTrials.gov Identifier: NCT03947385) evaluating the combination of darovasertib and crizotinib in metastatic uveal melanoma (MUM) (Press release, Ideaya Biosciences, MAY 10, 2021, https://www.prnewswire.com/news-releases/ideaya-announces-dose-expansion-in-phase-12-study-of-darovasertib-and-crizotinib-combination-based-on-early-clinical-efficacy-in-first-combination-cohort-301287055.html [SID1234579624]). IDEAYA is the sponsor of this combination study, which is being conducted pursuant to a clinical trial collaboration and supply agreement with Pfizer Inc. Darovasertib is IDEAYA’s clinical stage protein kinase C, or PKC, inhibitor and crizotinib is a cMET inhibitor to which Pfizer has exclusive worldwide rights.

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"We are encouraged to see the early deep partial response in the first cohort of the darovasertib and crizotinib combination. We look forward to the dose expansion phase and to continue dose exploration to clinically validate the preclinical combination hypothesis discovered by IDEAYA," said Dr. Marlana Orloff, M.D., Assistant Professor, Thomas Jefferson University Hospitals.

IDEAYA presented translational data at AACR (Free AACR Whitepaper) 2021 retrospectively evaluating cMET expression in clinical biopsies from MUM patients in a Darovasertib Phase 1 clinical trial, as well as preclinical data evaluating synergy between darovasertib and crizotinib in relevant cell models of a liver tumor microenvironment, a site of approximately 90% of uveal melanoma metastases. "We identified inhibition of the cMET signaling pathway in combination with PKC in the metastatic setting as a potential treatment regimen, and are excited to observe our first signal of clinical activity to validate this research finding," said Mick O’Quigley, Vice President, Head of Development Operations at IDEAYA Biosciences.

Darovasertib / Crizotinib Combination Therapy

IDEAYA is continuing patient enrollment into the darovasertib / crizotinib combination arm under the clinical trial collaboration and supply agreement with Pfizer. Highlights:

6 MUM patients have enrolled in the darovasertib and crizotinib combination study and 2 of these patients were evaluable for response with at least one post-baseline scan
Observed early clinical efficacy of the darovasertib and crizotinib combination in MUM. As of data and analyses cutoff on May 5, 2021 based on preliminary data from an unlocked database, these data showed:
tumor reduction in 2 of 2 evaluable patients in a first cohort
one unconfirmed partial response in a 3rd-line patient, with a 54% tumor reduction, which is the deepest response reported in the darovasertib clinical trial to date; this patient is awaiting a confirmatory scan
Drug-related adverse events observed in the darovasertib and crizotinib combination arm in MUM as of May 5, 2021, based on preliminary data from an unlocked database, primarily include: serious adverse events of syncope and hypotension, each of which resolved with patients continuing dosing; and adverse events that occurred in at least two of the six treated patients of nausea, diarrhea, vomiting, edema, decreased appetite, and syncope
Initiated dose expansion for a cohort of the Phase 1/2 darovasertib / crizotinib combination arm, with additional dose exploration ongoing
Observed preclinical synergies between darovasertib and crizotinib in relevant cellular models under conditions simulating a tumor microenvironment in the liver, the site of approximately 90% of uveal melanoma metastases, as reported at AACR (Free AACR Whitepaper) 2021
Correlated cMET expression and activation to observed clinical response based on a retrospective analysis of human clinical biopsies from the Novartis darovasertib Phase 1 clinical trial, supporting co-targeting PKC and cMET signaling

On May 10, 2021 Compugen Ltd. (Nasdaq: CGEN), a clinical-stage cancer immunotherapy company and a leader in predictive target discovery, reported that it has expanded its research collaboration with Johns Hopkins University to include studies investigating the biology of a novel myeloid target that was computationally-discovered by Compugen (Press release, Compugen, MAY 10, 2021, View Source [SID1234579623]). Initial preclinical studies demonstrate the potential of this target to serve as a novel myeloid immunomodulator, with significant tumor growth inhibition observed upon genetic deletion in in-vivo studies.

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The research program, headed by Jelani Chinelo Zarif, M.S., Ph.D., Assistant Professor of Oncology and Drew Pardoll, M.D., Ph.D., Professor of Oncology, both at Johns Hopkins University School of Medicine, will explore the biological function and mechanism of this novel target, which is expressed on myeloid cells and macrophages in various cancers. The expanded research plan is intended to further evaluate and validate the role of the target in various tumors.

"We continue to invest in our early-stage programs, which serve as our pipeline growth engine and remain a high priority for Compugen," said Anat Cohen-Dayag, Ph.D., President and CEO of Compugen. "Our collaboration with Johns Hopkins has played an important role in the development of our clinical stage asset, COM701, and we are thrilled to expand this collaboration to deepen our biological understanding of this novel myeloid target selected from our early-stage pipeline. We hope that these studies will provide a strong biological foundation to support the future development of a new, potentially first-in-class, therapeutic program, which may diversify our immuno-oncology pipeline programs and offer a new treatment opportunity for cancer patients."

On May 10, 2021 Irish based cell engineering company, Avectas reported the publication of ‘A novel non-viral delivery method that enables efficient engineering of primary human T cells for ex vivo cell therapy applications’ in leading international cell and gene therapy publication, Cytotherapy (Press release, Avectas, MAY 10, 2021, View Source [SID1234579622]).1

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Cytotherapy is the official journal of the International Cell & Gene Therapy Society (ISCT).

Dr Shirley O’Dea et al. examined the suitability of Avectas’ SOLUPORE non-viral delivery system for engineering primary human T cells for cell therapy applications. This publication describes how the next generation of immune cell therapy products will require complex modifications using engineering technologies that can maintain high levels of cell functionality and how non-viral engineering methods such as SOLUPORE have the potential to address limitations associated with viral vectors.

Data demonstrates efficient transfection of human primary T Cells with mRNA and CRISPR CAS9 RNP cargos while consistently maintaining high levels of cell viability. Gene expression profiling revealed minimal up or down regulation of genes, demonstrating the low level of perturbation experienced by the cells during this transfection process in contrast to electroporation which resulted in substantial changes in immune gene expression. CAR T cells engineered using the SOLUPORE system exhibited high cytotoxicity against target cancer cells in vitro and in vivo.

Avectas has previously published on the SOLUPORE Technology in PLOS ONE2 with citations in both Nature Biomedical Engineering3 and Chemical Reviews4.

Commenting on the publication, Dr Michael Maguire, CEO of Avectas, said: "It is impressive to see further published evidence showing the advantages of the SOLUPORE platform in preserving cellular functionality essential for cell therapies. Congratulations to Avectas CSO, Dr Shirley O’Dea and the Avectas R&D team on this additional publication of important work to support the efficacy and potential of SOLUPORE technology to the cell and gene therapy industry."

Using its patented SOLUPORE technology, Avectas partners with leading cell and gene therapy companies and research institutions to enable the next-generation of gene-modified cell therapies.

On May 10, 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 first quarter ended March 31, 2021 (Press release, Novavax, MAY 10, 2021, View Source [SID1234579621]).

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"Novavax made great strides over the first quarter to pave the path for our COVID-19 vaccine candidate, NVX-CoV2373, notably achieving statistically significant efficacy across our Phase 3 UK and Phase 2b South Africa trials," said Stanley C. Erck, President and Chief Executive Officer, Novavax. "In parallel, we have secured additional manufacturing and supply agreements, expanding our global supply chain to over 10 countries. In the coming months, we look forward to delivering on critical milestones, including announcing final data from our PREVENT-19 Phase 3 trial, completing our regulatory submissions, evaluating NVX-CoV2373 in younger populations and continuing to develop our booster strategy to address the evolving COVID-19 pandemic. As we continue our dialogue with regulatory authorities for authorization, we remain committed to promptly delivering our vaccine globally, ensuring equitable access and expansive distribution."

First Quarter 2021 and Recent Highlights

COVID-19 Clinical Development

Reported final efficacy analysis from UK Phase 3 trial and complete analysis from South Africa Phase 2b trial, expanding upon previously reported interim results
Demonstrated 100% protection against severe disease
Confirmed 96.4% efficacy against original strain of COVID-19 and 86.3% efficacy against B.1.1.7 variant strain in UK Phase 3 trial
Demonstrated efficacy of 88.9% in adults over the age of 65 and efficacy of 90.9% in adults with high-risk medical comorbidities in UK Phase 3 trial
Demonstrated efficacy of 55.4% among HIV-negative participants in South Africa Phase 2b trial, with the vast majority of cases due to the B.1.351 escape variant
South Africa Phase 2b results published in the New England Journal of Medicine
UK Phase 3 results submitted for publication to peer-reviewed journal and posted to medRxiv.org
Completed successful enrollment of 30,000 participants for PREVENT-19; final analysis to be reported in the second quarter of 2021
Initiated pediatric expansion of PREVENT-19 to evaluate efficacy, safety and immunogenicity of NVX-CoV2373 in adolescents
Enrolling up to 3,000 adolescent participants aged 12-17 across up to 75 sites in the U.S.
Blinded crossover expected to begin six months after initial set of vaccinations
Initiated crossovers and progressed booster studies of NVX-CoV2373
Initiated crossovers in PREVENT-19, UK Phase 3 and South Africa Phase 2b trials to ensure all trial participants have access to active vaccine
Completed 6-month booster doses in the U.S. and Australia Phase 2 study with immunology results expected in the third quarter of 2021
Com-COV2 Phase 2 clinical trial conducted by University of Oxford and UK Vaccines Taskforce
NVX-CoV2373 is one of four COVID-19 vaccines administered during trial to evaluate combined vaccine regimens in 1,050 participants
Top-line data expected in the third quarter of 2021
Advanced development of variant strain vaccines into preclinical studies
Preclinical data from study of B.1.351 variant strain vaccine candidate showed strong antibody response and functional immune response in non-human primates when boosted one year after receiving NVX-CoV2373
Expect to initiate clinical evaluation of one or more candidates
Successful collaboration on partner-initiated trials to advance clinical developmentof NVX-CoV2373
Takeda completed enrollment of 200 participants in Phase 1/2 clinical trial in Japan
Serum Institute of India (Serum Institute) completed enrollment of initial cohort in Phase 2/3 clinical trial in India, with total study including 1,600 participants
COVID-19 Manufacturing and Supply

Secured additional manufacturing capacity for NVX-CoV2373 globally, with continued progress toward achieving full manufacturing capacity
Anticipated capacity revised to 100 million doses per month by the end of the third quarter of 2021, with remainder of capacity expected to come online in the fourth quarter to support 150 million doses per month
Reached agreement in principle with GSK to support ‘fill and finish’ manufacturing of up to 60 million doses of NVX-CoV2373 for use in the UK
Established manufacturing presence in Canada through Memorandum of Understanding with Canadian government to produce NVX-CoV2373 at National Research Council’s Biologics Manufacturing Centre
Advanced purchase agreements globally, ensuring equitable access to low, middle and high income countries
Finalized advance purchase agreement with Government of Canada to supply 52 million doses with an option for up to an additional 24 million doses
Finalized 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 to participants of the COVAX Facility
Serum Institute to manufacture and distribute remaining balance of the 1.1 billion doses to low- and middle-income countries
Furthered existing partnerships to expand global access of NVX-CoV2373
Finalized exclusive license agreement with Takeda for the development, manufacturing and commercialization of NVX-CoV2373
Takeda to manufacture over 250 million doses of NVX-CoV2373 annually
Takeda in discussions with Government of Japan to potentially purchase 150 million doses of NVX-CoV2373
Expanded existing partnership with SK bioscience to include license agreement for the manufacturing and commercialization of NVX-CoV2373
SK bioscience to supply 40 million doses to the Republic of Korea
COVID-19 Regulatory Pathway

Progressed regulatory processes for authorization of NVX-CoV2373 with multiple regulatory agencies globally
Intend to file for authorization with the U.S. Food and Drug Administration (FDA), the UK Medicines and Healthcare products Regulatory Agency (MHRA) and the European Medicines Agency (EMA) in Europe in the third quarter of 2021
Rolling reviews initiated with regulatory authorities, including Health Canada, Australian Therapeutic Goods Administration, New Zealand Medsafe, MHRA and EMA
SK bioscience initiated regulatory submission process in collaboration with Novavax to the Republic of Korea’s Ministry of Food and Drug Safety
Combination Vaccine

NanoFlu/NVX-CoV2373 combination vaccine candidate induced protective responses in preclinical studies
Preclinical data manuscript submitted for publication to a peer-reviewed journal and posted on bioRxiv.org
Expect to initiate clinical evaluation later this year
Malaria R21 Vaccine / Matrix-M Adjuvant Collaboration

Phase 2b clinical trial results for the University of Oxford’s malaria vaccine candidate (R21), including Matrix-M adjuvant, published in Preprints with The Lancet
Trial included 450 participants aged 5-17 months in Burkina Faso
Demonstrated high efficacy of 77% when using 5 micrograms of antigen and 50 micrograms of Matrix-M adjuvant
Novavax to manufacture and supply Matrix-M adjuvant to Serum Institute for use in R21
Serum Institute has rights to use the vaccine, comprised of R21 with Matrix-M adjuvant, in endemic regions, while Novavax will have rights to sell and distribute the vaccine in travelers’ and military vaccine markets
Phase 3 licensure trial underway in 4,800 participants, aged 5-36 months, across four countries in Africa to evaluate the safety and efficacy of R21
Corporate Highlights

Strengthened corporate leadership with executive management promotions and hiring
Gale E. Smith, Ph.D. promoted to Senior Vice President, Discovery and Preclinical Research and Chief Scientist
Madelyn ‘Lyn’ Caltabiano, Ph.D. as Senior Vice President, Global Program Management
Troy Morgan, J.D. as Senior Vice President, Chief Compliance Officer
Henrietta Ukwu, M.D. as Senior Vice President, Chief Regulatory and Quality Officer
Financial Results for the Three Months Ended March 31, 2021

Novavax reported a net loss of $223 million, or $3.05 per share, for the first quarter of 2021, compared to a net loss of $26 million, or $0.58 per share, for the first quarter of 2020.

Novavax revenue in the first quarter of 2021 was $447 million, compared to $3 million in the same period in 2020. This significant 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 $593 million in the first quarter of 2021, compared to $17 million in the same period in 2020. The significant increase was primarily due to the development of NVX-CoV2373.

General and administrative expenses increased to $63 million in the first quarter of 2021, compared to $9 million for the same period in 2020. The increase was primarily due to increased employee-related costs, stock-based compensation expenses, and supporting our NVX-CoV2373 program.

As of March 31, 2021, Novavax had $2 billion in cash, cash equivalents, marketable securities and restricted cash, compared to $806 million as of December 31, 2020. Net cash provided by operating activities for the first three months of 2021 was $663 million, compared to net cash used in operating activities of $23 million for same period in 2020. The increase in cash provided was primarily due to $772 million 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 quarter 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 May 10, 2021 until 11:59 p.m. ET on May 17, 2021. To access the replay by telephone, dial (877) 344-7529 (Domestic) or (412) 317-0088 (International) and use passcode 10155684.

A webcast of the conference call can also be accessed on the Novavax website at View Source A replay of the webcast will be available on the Novavax website until August 10, 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 adjuvanted with Novavax’ patented saponin-based Matrix-M 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 100% protection against severe disease, efficacy of 96.4% against the original virus strain, 86.3% against the B.1.1.7/501Y.V1 variant and 89.7% overall; and the PREVENT-19 trial in the U.S. and Mexico that began in December 2020. 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 100% protection against severe disease 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

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.