On March 2, 2021 Sorrento Therapeutics, Inc. (Nasdaq: SRNE, "Sorrento") reported that the FDA has cleared Sorrento’s internally developed anti-CD47 monoclonal antibody, STI-6643, which was discovered from Sorrento’s G-MAB library, for an initial clinical trial (Press release, Sorrento Therapeutics, MAR 2, 2021, View Source [SID1234575920]). The initial clinical trial will be a basket trial entitled "A Phase 1B, Open-Label, Dose-Escalation Study of the Safety and Efficacy of STI-6643, an Anti-CD47 Human Monoclonal Antibody, in Patients with Selected Relapsed or Refractory Malignancies."

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Cluster of differentiation 47 (also known as integrin-associated protein) ("CD47") is a ubiquitously-expressed glycoprotein of the immunoglobulin superfamily that plays a critical role in self-recognition. Various solid and hematologic cancers exploit CD47 expression to evade immunological eradication, and its overexpression is clinically correlated with poor prognoses. One essential mechanism behind CD47-mediated immune evasion is that it can interact with signal regulatory protein-alpha (SIRPα) expressed on myeloid cells, causing phosphorylation of the SIRPα cytoplasmic immunoreceptor tyrosine-based inhibition motifs and recruitment of Src homology 2 domain-containing tyrosine phosphatases to ultimately result in delivering an anti-phagocytic "don’t eat me" signal. Given its essential role as a negative checkpoint for innate immunity and subsequent adaptive immunity, the CD47/SIRPα axis has been explored as a new target for cancer immunotherapy and its disruption has demonstrated great therapeutic promise.

In preclinical evaluations, STI-6643 displayed decreased red blood cell binding and hemolysis, while maintaining potent anti-tumor activity in solid tumor disease models. Clinical trials with anti-CD47 mAbs have historically experienced limitations due to significant anemia, hemagglutination, and thrombocytopenia due to CD47 expression on normal red blood cells, ultimately requiring employment of complicated clinical dosing regimens. These issues have not been seen to date with STI-6643 in preclinical studies conducted head-to-head against synthesized versions of other CD47 mAbs. Additionally, STI-6643 showed minimal T, B or NK cell depletion as opposed to other synthesized mAb clones, which could potentially result in improved efficacy by preserving infiltrating anti-tumor immune cells. STI-6643 has the potential to be a "best-in-class" product if these preclinical benefits are able to be reproduced in human trials. Sorrento is also conducting preclinical studies to compare the safety and efficacy of lymphatic delivery of STI-6643 to established parenteral routes of administration using Sorrento’s Sofusa technology. This study will be conducted at the Moffitt Cancer Center in Tampa, FL with Dr. David A. Sallman as the coordinating lead investigator.

STI-6643 is the second anti-CD47 antibody that has been developed from the G-MAB library. The other anti-CD47 antibody (IMC-002) discovered from the G-MAB library was previously cleared by the FDA, and is currently in Phase 1 human studies sponsored by ImmuneOncia Therapeutics, LLC, a joint venture company between Sorrento (35% ownership) and Yuhan Corporation.

Regarding the recent clearance for a clinical trial for STI-6643 by the FDA, Dr. Henry Ji, Chairman and CEO of Sorrento, commented, "We have seen great performance from STI-6643 in our IND-enabling studies. Our internal anti-CD47 program has now yielded two clinical candidates, a signal of our continued commitment to the development of innovative, safe and efficacious cancer treatments in addition to our commitment to fighting COVID-19."

On March 2, 2021 NANOBIOTIX (Euronext : NANO –– NASDAQ: NBTX – the ‘‘Company’’), a clinical-stage biotechnology company pioneering physics-based approaches to expand treatment possibilities for patients with cancer, reported positive new preclinical data investigating first-in-class radioenhancer NBTXR3, which is being evaluated as a tumor-agnostic, combo-agnostic product candidate across several tumor types (Press release, Nanobiotix, MAR 2, 2021, View Source [SID1234575936]). The data is being shared via poster presentation at the first American Association of Cancer Research (AACR) (Free AACR Whitepaper) Virtual Special Conference on Radiation Science and Medicine, held March 2-3, 2021, by researchers from the University of Texas MD Anderson Cancer Center (MD Anderson).

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PRECLINICAL DATA ON NBTXR3 COMBO

NBTXR3 is composed of sterile, functionalized, crystalline hafnium oxide nanoparticles that are delivered by intratumoral injection one time prior to radiotherapy (XRT). After activation by XRT, the product candidate has a physical primary mechanism of action through which a higher dose of radiation is delivered within the tumor, enhancing the tumor-killing effect of XRT without increasing the dose in surrounding healthy tissues. The subsequent biological secondary mechanism of action that we are evaluating is the potential activation of several immune pathways upon tumor cell destruction, generating adaptive immune response within the body.

NBTXR3 is being evaluated in an expansive global development plan both as a single agent activated by XRT and in combination with other anti-cancer therapies including chemotherapy and immune checkpoint inhibitors. This study examined NBTXR3 activated by XRT in combination with anti-PD-1 along with TIGIT and LAG3 inhibitors in an in vivo anti-PD-1 resistant mouse model (344SQR).

Key Findings Include:

The combination therapy of NBTXR3 + XRT + anti-PD-1 + anti-LAG3 + anti-TIGIT (Combo therapy) significantly promoted the proliferation activity of CD8+ T cells, improved local and distant tumor control, and increased survival rate
The anti-tumor efficacy of the Combo therapy was heavily dependent on CD4+ and CD8+ Tcells
The survivor mice from the groups treated with the Combo therapy were immune to re-injections of tumor cells
The survivor mice maintained significantly higher percentages of memory CD4+ and CD8+ T cells, as well as stronger anti-tumor immune activities than control
The Combo therapy augmented anti-tumor response in both irradiated and unirradiated (abscopal) tumors
POSTER PRESENTATION

Title: Integration of anti-TIGIT and anti-LAG3 with NBTXR3-mediated Immunoradiation Therapy Improved Abscopal Effect and Induces Long-term Memory Against Cancer
Timing: View on-demand starting at 9:30am EST on March 2, 2021 on Virtual Conference platform
Poster Number: PO-040
***

About NBTXR3

NBTXR3 is a first-in-class radioenhancer composed of sterile, functionalized, crystalline hafnium oxide nanoparticles. The product candidate is designed to increase the radiotherapy energy deposit inside tumor cells through the nanoparticles’ high atomic number core packaged in the space for interaction with ionizing radiation, and subsequently increase of tumor cell death when compared to radiotherapy alone—without adding toxicity to adjacent healthy tissues. NBTXR3 requires a single, intratumoral administration before the first radiotherapy treatment session, and has the ability to fit into current worldwide standards of radiation care. The primary physical mechanism of action of NBTXR3 activated by radiotherapy could be universal, making it potentially applicable across any solid tumor indication where radiotherapy is a part of standard of care including head and neck, lung, prostate, liver, colorectal, and esophageal cancers. The biological secondary mechanism of action of NBTXR3 activated by radiotherapy has been shown in preclinical studies to prime adaptive immune response, which would potentially bring a new dimension to cancer immunotherapies.

On March 2, 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 fourth quarter and twelve months ended December 31, 2020 (Press release, Novavax, MAR 2, 2021, View Source [SID1234575952]).

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"Novavax continues to make significant strides towards bringing NVX-CoV2373, our COVID-19 vaccine candidate, to market," said Stanley C. Erck, President and Chief Executive Officer of Novavax. "With positive efficacy results, including against evolving variant strains, NVX-CoV2373 offers a highly unique profile, including the ability to ship and store the vaccine at traditional refrigerated temperatures. We believe these attributes support emergency use authorization and have initiated dialogue with regulators to pursue appropriate regulatory authorization. In addition, we have secured agreements for the delivery of approximately 300 million doses of NVX-CoV2373. In our efforts to provide fair and equitable access to our vaccine around the world, we are proud to partner with the Serum Institute of India to jointly supply 1.1 billion doses of NVX-CoV2373 to Gavi through the COVAX Facility. We continue to work tirelessly to make final commercial preparations in advance of delivering our product across the globe."

Fourth Quarter 2020 and Recent Highlights

COVID-19 Program

Commenced regulatory process for authorization for NVX-CoV2373
Rolling submission initiated with the UK Medicines and Healthcare products Regulatory Agency; potential to file for authorization in the UK by early second quarter 2021
Engaged in ongoing dialogue with US Food and Drug Administration (FDA) through submissions to our open investigational new drug application, with potential for EUA filing in the second quarter of 2021
Rolling reviews initiated with:
European Medicines Agency
Health Canada
Australian Therapeutic Goods Administration
Medsafe New Zealand
Reported positive top-line data from Phase 3 UK clinical trial
Observed 95.6% efficacy against the original strain of COVID-19 and 85.6% against the UK variant strain
Overall primary endpoint met with a vaccine efficacy of 89.3%
Generally well-tolerated with a reassuring safety profile
Trial included 15,000 participants between 18-84 years of age, including 27 percent over the age of 65

Reported successful Phase 2b South Africa efficacy study
Observed 60% efficacy for HIV-negative portion of study population (94% of study participants were HIV-negative)
Demonstrated clinically meaningful protection from South Africa escape variant, which accounted for 93% of sequenced cases
Achieved primary efficacy endpoint in overall trial population of 49.4%
Trial included 4,404 participants, including 245 medically stable, HIV-positive participants
Supported in part by a $15 million grant from Bill & Melinda Gates Foundation (BMGF)
Completed enrollment in pivotal PREVENT-19 Phase 3 efficacy trial in the US and Mexico
30,000 participants enrolled in two-to-one study design, with highly diverse population
20% Latin American, 12% African American, 6% Native American, 5% Asian American and 13% adults over the age of 65
Interim data expected in the second quarter of 2021 dependent on the overall COVID-19 attack rate
Blinded crossover protocol, ensuring all participants are provided active vaccine, submitted to the FDA
Trial design harmonized to align with other Phase 3 clinical studies supported by the U.S. government
Ongoing clinical development of NVX-CoV2373
6-month boost dose as part of Phase 1/2 clinical trial in the US and Australia
Developing variant strain vaccines as standalone and bivalent candidates
Evaluation of candidates ongoing in non-human primates
Planning clinical evaluation of variant vaccine candidates in mid-2021

Secured cumulative funding of over $2 billion to date through US government, CEPI and BMGF for development of NVX-CoV2373
US government funding through partnership formerly known as Operation Warp Speed increased up to $1.75 billion
Coalition for Epidemic Preparedness Innovations (CEPI) funding up to $400 million

Increased projected global manufacturing capacity to over 2 billion annualized doses when at full-capacity, expected to occur in mid-2021
Approximately one billion doses to be manufactured by Serum Institute of India Private Limited (SIIPL)
Completed collaborations for global manufacturing, commercialization and distribution 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
Advanced joint commitment with SIIPL for the equitable access of 1.1 billion doses of NVX-CoV2373 for distribution by the COVAX Facility
Reached Memorandum of Understanding with Canadian government for plans to produce NVX-CoV2373 at the National Research Council’s Biologics Manufacturing Centre in Montreal
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

Secured agreements for approximately 200 million doses of NVX-CoV2373
Government of Canada to be supplied 52 million doses with an option for up to an additional 24 million
UK government to be supplied 60 million doses
Commonwealth of Australia to be supplied 51 million doses with an option for up to an additional 10 million
Government of New Zealand to be supplied 11 million doses
Government of Switzerland to be supplied 6 million doses
NanoFlu Program

Continued to advance NanoFlu program, including exploration of a combined NanoFlu/NVX-CoV2373 vaccine that could be used in a post-pandemic setting
Corporate Highlights

Appointed three individuals with extensive pharmaceutical industry experience to Novavax’ Board of Directors
Gregg Alton, J.D.
Brings extensive industry experience, including more than 20 years at Gilead Pharmaceuticals, serving in an array of leadership roles including Chief Executive Officer and Chief Patient Officer
Margie McGlynn, R. Ph.
Brings extensive pharmaceutical industry, vaccine and non-profit experience, including more than two decades at Merck; held roles of increasing responsibility, including President of Merck Vaccines and Infectious Diseases and President, US Hospital and Specialty Products Division
David Mott
Brings more than three decades of global management, board and investment experience across numerous biopharmaceutical companies, including previously having served as President and Chief Executive Officer of MedImmune
Financial Results for the Three and Twelve Months Ended December 31, 2020

Novavax reported a net loss of $177.6 million, or $2.70 per share, for the fourth quarter of 2020, compared to a net loss of $31.8 million, or $1.13 per share, for the fourth quarter of 2019. For the twelve months ended December 31, 2020, the net loss was $418.3 million, or $7.27 per share, compared to a net loss of $132.7 million, or $5.51 per share, for the same period in 2019.

Novavax revenue in the fourth quarter of 2020 was $279.7 million, compared to $8.8 million in the same period in 2019. The significant increase in revenue was comprised of revenue for services performed under the CEPI agreement and participation in OWS.

Research and development expenses increased to $401.2 million in the fourth quarter of 2020, compared to $29.3 million in the same period in 2019. The increase was primarily due to increased development activities relating to NVX-CoV2373 and increased employee-related costs, including stock-based compensation expense.

General and administrative expenses increased to $61.3 million in the fourth quarter of 2020, compared to $8.2 million for the same period in 2019. The increase was primarily due to increased employee-related costs, primarily stock-based compensation expense, and increased professional fees to support of our NVX-CoV2373 program.

As of December 31, 2020, Novavax had $806.4 million in cash, cash equivalents, marketable securities and restricted cash, compared to $82.2 million as of December 31, 2019. Net cash used in operating activities for the twelve months of 2020 was $42.5 million, compared to $136.6 million for same period in 2019.

Through utilization of At-the-market (ATM) offerings, Novavax raised net proceeds of $428 million and $874 million during the three and twelve months of 2020, respectively. In addition, in the second quarter of 2020, Novavax received gross proceeds of $200 million upon entering into an agreement to sell Series A Convertible preferred stock, convertible into 4,388,850 shares of common stock, to an investment fund affiliated with RA Capital Management (RA Capital) in a private placement. There shares were converted to common stock in the fourth quarter of 2020.

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 (877) 212-6076 (Domestic) or (707) 287-9331 (International), passcode 3797013. A replay of the conference call will be available starting at 7:30 p.m. ET on March 1, 2021 until 7:30 p.m. ET on March 8, 2021. To access the replay by telephone, dial (855) 859-2056 (Domestic) or (404) 537-3406 (International) and use passcode 3797013.

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 June 1, 2021.

About NVX-CoV2373

NVX-CoV2373 is a protein-based vaccine candidate engineered from the genetic sequence 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 block binding of the spike protein to cellular receptors and provided protection from infection and disease. It was generally well-tolerated and elicited robust antibody response numerically superior to that seen in human convalescent sera in Phase 1/2 clinical testing. NVX-CoV2373 is currently being evaluated in two pivotal Phase 3 trials: a trial in the U.K that demonstrated 89.3 percent overall efficacy and 95.6 percent against the original strain in a post-hoc analysis, and the PREVENT-19 trial in the U.S. and Mexico that began in December. It is also being tested in two ongoing Phase 2 studies that began in August: A Phase 2b trial in South Africa that demonstrated 50-60 percent efficacy against newly emerging escape variants, and a Phase 1/2 continuation in the U.S. and Australia.

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.

On March 2, 2021 GRAIL, Inc., a healthcare company whose mission is to detect cancer early, when it can be cured, reported a partnership with leading health system Providence to advance the science and delivery of cancer care (Press release, Grail, MAR 2, 2021, View Source [SID1234575968]). Providence, based in Renton, Washington, will be the first health system to offer Galleri, GRAIL’s multi-cancer early detection blood test, as a complement to recommended single cancer screening tests.

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The Galleri test initially will be used by Providence at its California, Washington, and Oregon points of care, and could eventually be available across Providence’s entire seven-state footprint that includes more than 50 hospitals and nearly 1,100 health clinics serving 5 million patients. Providence aims to pursue effective ways to identify cancer earlier in patient lives, and will integrate the Galleri test into clinical care to help drive earlier detection of multiple cancers and to try to improve and save lives.

"Today, the majority of cancers are found too late when outcomes are often fatal, and as a result, cancer is projected to become the leading cause of death worldwide," said Hans Bishop, chief executive officer at GRAIL. "Providence is a leading health system that shares our commitment to improving and saving lives, and our partnership moves us that much closer to making a tremendous and potentially life-altering difference for patients and providers. We are encouraged by interest in Galleri from health systems, medical practices, and self-insured employers, and excited by the progress in our mission to detect cancer early."

"At Providence, we continuously strive to deliver the most innovative medical advances to our patients, and we are thrilled to partner with GRAIL on implementation research that may bring Galleri to all the communities we serve," said Amy Compton-Phillips, MD, chief clinical officer at Providence. "When combined with the power of our genomics research, having a single blood test that can detect more than 50 cancers advances our goal of finding cancer early, enabling our providers to administer treatment when it’s most likely to be successful and giving our patients the opportunity to achieve the best possible outcomes."

GRAIL previously announced it expects to introduce Galleri in the second quarter of 2021. The blood test, which will be prescription only, will be available initially through partner health systems, medical practices, and self-insured employers.

An earlier version of Galleri demonstrated the ability to detect more than 50 types of cancers — over 45 of which lack recommended screening tests today — with a low false positive rate of less than 1%. When a cancer signal is detected, Galleri can determine where in the body the cancer is located with high accuracy, all from a single blood draw.

On March 2, 2021 Oscotec Inc. (039200: KOSDAQ), the Korean drug development company, and Beactica Therapeutics AB, the Swedish drug discovery company, reported a new research development and licensing agreement (Press release, Oscotec, MAR 2, 2021, View Source [SID1234575874]). Oscotec and Beactica will initially jointly collaborate concerning research and early preclinical development of novel anti-cancer drug candidates arising out of Beactica’s LSD1 programme. Oscotec may thereafter opt to take full responsibility for clinical development and commercialization.

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Under the terms of the agreement, Oscotec is entitled to gain exclusive global rights for further development and commercialization of Beactica’s programme. Beactica is eligible to receive up to EUR 149 million in potential preclinical, clinical and regulatory milestones, including an upfront and equity payment from Oscotec upon signing of the agreement. Furthermore, Beactica is eligible to receive double-digit royalties on commercial sales of the products resulting from the partnership. Beactica is also entitled to revenue shares from any related future licensing activities by Oscotec. Full financial details remain undisclosed.

"We are thrilled about the opportunity to work with Beactica on the LSD1 allosteric inhibitor programme that we hope opens up very unique and exciting possibilities," said Dr Taeyoung Yoon, CEO/CSO of Oscotec. "Not only could this opportunity fill our need of balancing the preclinical pipeline in the short term, we are convinced that the partnership will also bring to us added value of collaborative science where the two companies complement each other extremely well.""This is a landmark agreement for Beactica, and we are delighted to be working with Oscotec." said Dr Per Källblad, CEO of Beactica Therapeutics. "Their commitment to building a strong clinical pipeline of targeted therapeutics in immunology and oncology makes Oscotec an ideal partner for our programme."