On March 10, 2020 Cellectis (Euronext Growth: ALCLS; Nasdaq: CLLS), a clinical-stage biopharmaceutical company focused on developing immunotherapies based on gene-edited allogeneic CAR T-cells (UCART), reported that a new patent from the US Patent and Trademark Office (USPTO) had been granted to Cellectis for methods of preparing allogeneic T-cells for immunotherapy with CRISPR-Cas9 technology (Press release, Cellectis, MAR 10, 2020, https://www.cellectis.com/en/press/us-patent-covering-crispr-edited-allogeneic-car-t-cells-granted-to-cellectis/ [SID1234555360]).

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The patent US10,584,352 issued today claims "a method of preparing and administering T-cells for immunotherapy comprised of providing primary human T-cells from a healthy donor and genetically modifying the primary human T-cells to eliminate expression of the T-cell receptor (TCR), which contains expression on the Cas9 endonuclease fused to a nuclear localization signal (NLS) and guide RNA that directs said endonuclease to at least one targeted locus encoding the TCR in the T-cell genome, and further the expansion of the genetically modified T-cells, as well as the administration of at least 10,000 of the expanded genetically modified T-cells to a patient."

This patent complements the European patent EP3004337, claiming a method of preparing T-cells for immunotherapy using the CRISPR-Cas9 system, initially granted on August 2, 2017 and upheld by the EPO in November 2019 following an opposition procedure initiated in May 2018.

In January 2020, Cellectis was also granted European Patent EP3116902, which claims "an engineered isolated CAR T-cell, which expression of beta 2-microglobulin (B2M) is inhibited, while at least one gene encoding a component of the T-cell receptor (TCR) is inactivated."

"These patents highlight Cellectis’ long-term expansion of expertise in various gene-editing technologies, including CRISPR-Cas9," said Dr. André Choulika, Chairman and CEO, Cellectis. "Cellectis invented the gene-edited CAR-T allogeneic approach, and over the past decade, we have remained committed to developing the most safe and efficacious therapeutic products on a global scale."

The Cellectis inventors of US10,584,352 and EP3004337 patents are Dr. André Choulika, Chairman and CEO, Dr. Philippe Duchateau, Chief Scientific Officer and Dr. Laurent Poirot, VP, Immunology Department. Inventors from Cellectis of the EP3116902 patent include Dr. Laurent Poirot, VP, Immunology Department, Dr. David Sourdive, EVP, Strategic Initiatives, Dr. Philippe Duchateau, Chief Scientific Officer and Dr. Jean-Pierre Cabaniols, Head of Analytical Department.

On March 10, 2020 Delphinus Medical Technologies, Inc., the leader in advanced breast ultrasound technology, has reported that results of a study comparing the use of whole breast ultrasound tomography (UST) and mammography to assess breast cancer risk have been published in the Journal of Clinical Medicine (Press release, Delphinus Medical Technologies, MAR 10, 2020, View Source [SID1234555376]). The study was conducted by researchers at the National Cancer Institute, the Karmanos Cancer Institute, Wayne State University, Henry Ford Health Systems, The Mayo Clinic and George Washington University and was supported by a contract from the National Cancer Institute, part of the National Institutes of Health.

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Mammographic percent density (MPD) is an independent risk factor for developing breast cancer. MPD only modestly improves breast cancer risk prediction and is not typically assessed in women under 40 because of ionizing radiation concerns. Previous studies have shown that tissue sound speed, derived from UST, a non-ionizing modality, is a potential surrogate marker of breast density. But prior to this study, sound speed has not been directly linked to breast cancer risk. The study evaluated the relationship of sound speed, using Delphinus’ SoftVue 3D whole breast ultrasound system, and MPD with breast cancer risk in a case-control study. This study compared 61 patients with a recent breast cancer diagnosis with 165 women with no personal history of breast cancer.

This study demonstrated that increasing quartiles of whole breast volume-averaged sound speed were consistently and more strongly associated with increasing breast cancer risk than quartiles of mammographic percent density. These findings were statistically significant and suggest future opportunities for utilizing UST-breast cancer risk assessment, particularly in younger women with the absence of ionizing radiation.

"It is well-established that dense breast tissue is a breast cancer risk factor. This study suggests that whole breast ultrasound tomography may provide stronger and more specific information about that risk than mammography, which may ultimately help to stratify the risk in order to suggest more personalized screening and interventions," said Dr. Rachel Brem, Director, Breast Imaging and Intervention at The George Washington University and the Program Leader, Breast Cancer, at The George Washington Cancer Center. "We are encouraged by the study results that indicate the potential use of whole breast ultrasound to improve the accuracy of breast cancer risk assessment with a non-ionizing breast imaging modality."

Delphinus Chief Technology Officer, Dr. Neb Duric, added, "This study expands the potential application of our platform SoftVue technology beyond diagnostic imaging and breast cancer screening to cancer risk stratification for women at virtually any age, including the approximately 70 million women in the U.S. that are below screening age. We believe that SoftVue imaging may enable individual risk assessment and intervention at an early age, when interventions are the most effective, as well as personalized screening regimens that take into account risk levels."

On March 10, 2020 China Immunotech Biotech of Beijing completed a $6.5 million Series A financing, led by Jianxin Capital with Grower Venture Capital and Huacheng Group participating (Press release, Immunotech, MAR 10, 2020, View Source [SID1234555396]). Founded in March 2018, China Immunotech is developing TCR-T and CAR-T products that target hematological tumors, solid tumors and virus-related diseases. It has two unique technology platforms, STAR-T and TCR-T. The STAR-T platform uses a proprietary structure of antigen receptor complexes. The company believes the platform provides multi-targeted molecules with better efficacy, fewer side effects and easier development than traditional CAR-T products.

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On March 10, 2020 Axial Biotherapeutics, a biotechnology company dedicated to building a unique class of gut-targeted programs for neurodegenerative diseases and neurodevelopmental disorders, reported that David H. Donabedian, Ph.D., Co-founder and Chief Executive Officer of Axial Biotherapeutics will present a virtual company overview and participate in a virtual panel on "Modulating the Microbiome" at the Chardan 2nd Annual Microbiome Medicines Summit on Monday, March 16, 2020 at 8:45 AM ET and 12:40 PM ET, respectively (Press release, Axial Biotherapeutics, MAR 10, 2020, View Source [SID1234555563]).

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On March 10, 2020 ChemoCentryx, Inc., (Nasdaq: CCXI), reported financial results for the fourth quarter and full year ended December 31, 2019 and provided an overview of the Company’s recent corporate highlights (Press release, ChemoCentryx, MAR 10, 2020, View Source [SID1234555361]).

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"The power of data to transform the world was evident in the results of our Phase III ADVOCATE trial of avacopan in ANCA-associated vasculitis," said Thomas J. Schall, Ph.D., President and Chief Executive Officer of ChemoCentryx. "The ADVOCATE data marked a critical turning point, in my view, in the treatment of this debilitating disease, with avacopan demonstrating superiority over the current standard of care which employs daily dosing of glucocorticoids. Those results transformed not just thousands of lives potentially, but changed the face of our enterprise as well. We intend to file an NDA for avacopan with the FDA by the middle of this year, and are laying the groundwork for US commercialization. Furthermore, the success of ADVOCATE not only validated the biology of C5a receptor inhibition by avacopan as a powerful therapy but it surpassed expectations and may open doors to additional renal disease opportunities in underserved indications such as lupus nephritis. The momentous readout in ADVOCATE now sets the stage for a data-rich 2020, during which we expect to share topline results from four additional ongoing clinical trials. An epochal year for CCXI was 2019, and we expect 2020 to be extraordinary as well."

Key Highlights

Exceeded expectations for avacopan with topline data from the ADVOCATE Phase III pivotal clinical trial announced in the fourth quarter of 2019, which demonstrated avacopan’s statistical superiority in sustaining remission at 52 weeks over the prednisone-containing standard-of-care, eliminating the need for daily noxious steroids. Avacopan was shown to lower all four aspects of the total burden of disease: stopping active vasculitis; significantly lowering current therapy-induced illness, with a statistically significant reduction in the Glucocorticoid Toxicity Index (GTI) and other accepted assessments of glucocorticoid toxicity; significantly improving kidney function as evidenced by a marked improvement in Estimated Glomerular Filtration Rate, or eGFR; and statistically significant improvements in quality of life, assessed by the SF-36 QOL instrument and the EuroQOL-5D-5L instrument (Visual Analogue Scale and EQ Index).

On track to announce topline data from the LUMINA-1 Phase II randomized clinical trial of CCX140, an inhibitor of the chemokine receptor known as CCR2, in patients with sub-nephrotic primary Focal Segmental Glomerulosclerosis (FSGS), another rare kidney disease, in the second quarter of 2020. The single-arm, open label LUMINA-2 study continues to enroll, evaluating CCX140 in primary FSGS patients with the more severe nephrotic levels of proteinuria, and we expect to announce topline data by year-end 2020.

Advanced enrollment in our ACCOLADE Phase II clinical trial of avacopan in patients with the rare kidney disease C3 Glomerulopathy (C3G). C3G often afflicts the young, requiring dialysis and often kidney transplant with recurring disease common. There is no approved effective treatment for C3G. In 2019 we were awarded a two-year $1 million grant from the orphan drug office of the FDA to support advancement in the ongoing ACCOLADE trial. Topline data from ACCOLADE are expected in the second half of 2020.

Surpassed 90 percent target enrollment in our AURORA Phase IIb clinical trial of avacopan for the treatment of the chronic disabling skin disease Hidradenitis Suppurativa (HS). Topline data from AURORA are expected in the third quarter 2020.

Maintained a strong balance sheet, with reported cash and investments exceeding $202 million at December 31, 2019, and an additional credit facility of up to $100 million secured in January 2020.

Fourth Quarter and Full Year 2019 Financial Results

Cash and investments, totaled $202.2 million at December 31, 2019, excluding the credit facility of up to $100 million that was secured in January 2020.

Revenue was $10.0 million for the fourth quarter of 2019, compared to $9.3 million for the same period in 2018. For the full year ended December 31, 2019, revenue was $36.1 million, compared to $42.9 million for 2018. The quarterly increase in revenue from 2018 to 2019 was primarily due to the full enrollment of the CCX140 LUMINA-1 Phase II trial in patients with FSGS in 2019. Revenue in 2019 also included $176,000 of grant revenue from the FDA to support the clinical development of avacopan in patients with C3G.

Research and development expenses were $19.2 million for the fourth quarter of 2019, compared to $15.1 million for the same period in 2018. Full year 2019 research and development expenses were $70.3 million compared to $62.7 million in 2018. The increase in research and development expenses from 2018 to 2019 was primarily attributable to (i) an increase in Phase II clinical study expense driven by patient enrollment in the avacopan AURORA trial in patients with HS and the two CCX140 LUMINA trials in patients with FSGS, and (ii) an increase in Phase I clinical study expense due to the initiation of avacopan ancillary studies. These increases were partially offset by decreases in 2019 in research and drug discovery expenses and expenses for the avacopan ADVOCATE Phase III pivotal trial as the study was fully enrolled in 2018.

General and administrative expenses were $7.0 million for the fourth quarter of 2019, compared to $5.6 million for the same period in 2018. Full year 2019 general and administrative expenses were $24.2 million, compared to $20.4 million in 2018. The increase from 2018 to 2019 was primarily due to higher employee-related expenses, including those associated with our commercialization planning efforts, and higher professional fees.

Net loss for the fourth quarter of 2019 was $15.5 million, compared to net loss of $10.8 million for the same period in 2018. Full year 2019 net loss was $55.5 million, compared to net loss of $38.0 million in 2018.

Total shares outstanding at December 31, 2019 were approximately 60.2 million shares.

We expect to utilize cash and investments in the range of $85 million to $95 million in 2020.

Conference Call and Webcast

The Company will host a conference call and webcast today, March 10, 2020 at 5:00 p.m. Eastern Time / 2:00 p.m. Pacific Time. To participate by telephone, please dial (877) 303-8028 (Domestic) or (760) 536-5167 (International). The conference ID number is 5494029. A live and archived audio webcast can be accessed through the Investors section of the Company’s website at www.ChemoCentryx.com. The archived webcast will remain available on the Company’s website for fourteen (14) days following the conference call.