On August 12, 2020 Cellular Biomedicine Group, Inc. (Nasdaq: CBMG) ("CBMG" or the "Company"), a biopharmaceutical firm engaged in the drug development of immunotherapies for cancer and stem cell therapies for degenerative diseases, reported its financial results and business highlights for the second quarter and six months ended June 30, 2020 (Press release, Cellular Biomedicine Group, AUG 12, 2020, View Source [SID1234563541]).

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"We held a virtual research & development day in July to provide an update on the six clinical programs we have in development," said Tony (Bizuo) Liu, Chief Executive Officer of CBMG. "Amid the global pandemic, we continue to enroll patients in investigator-initiated trials (IIT) for our clinical programs in China. This includes C-CAR088 anti-B-cell maturation antigen (BCMA) chimeric antigen receptor T cells (CAR-T) for relapsed or refractory multiple myeloma (MM), C-CAR039 anti-CD19/CD20 bi-specific CAR-T for Non-Hodgkin’s Lymphoma (NHL), and C-TCR055 alpha-fetoprotein (AFP) TCR-T in hepatocellular carcinoma (HCC). We look forward to the completion of our new Rockville facility later this year to support potential U.S. clinical development for C-CAR039 and C-TIL051 tumor-infiltrating lymphocytes (TIL) for non-small cell lung cancer (NSCLC). We also continue to enroll patients in China for our off-the-shelf AlloJoinTM knee osteoarthritis (KOA) Phase II trial. We plan to submit and present the C-CAR088 and C-CAR039 clinical data at a major conference later this year. We are happy to report that we have recently secured borrowings to support our near-term clinical development."

Clinical Highlights for First Half of 2020 and to Date:

C-CAR088 for MM*:
Infused 22 of the 25 enrolled patients; 17 patients with evaluable data for safety and clinical efficacy
No Grade 4 or higher cytokine release syndrome (CRS)
No Grade 2 or higher neurotoxicity and dose limiting toxicities
Cytopenia was mostly related to Cy/Flu lymphodepletion
17 patients with 100% best overall response; comprised of 5 complete response, 9 very good partial response and 3 partial response
C-CAR039 for NHL*:
Infused 10 of the 16 enrolled patients
No Grade 3 or higher CRS was observed
No Grade 2 or higher neurotoxicity
Cytopenia was mostly related to Cy/Flu lymphodepletion
Observed encouraging clinical efficacy with limited number of patients
C-TCR055 in HCC:
Initiated an early dose escalation study to evaluate the safety and efficacy
Conducted a poster presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting: "Selecting Clinical Lead of TCRs Targeting Alpha-Fetoprotein-Positive Liver Cancer on Balance of Risk and Benefit"
On July 13, 2020, the Company hosted a virtual Research & Development Showcase to present an overview and update on the current state of its clinical and pre-clinical programs.
*: As of June 15, 2020

Business Highlights for First Half of 2020 and to Date:

Executed a one-year $25 million convertible bridge loan
Extended the $16 million convertible bridge loan repayment schedule to August, 2021
Obtained $8.6 million lines of credit
Upcoming Milestones:

In the next nine months, present our key clinical assets update at major conferences
In 2021, execute our C-TIL051 to sponsor and initiate an IIT in the U.S. for stage IIIB and IV NSCLC patients refractory to anti-PD1 immunotherapy
Upon completion, qualify our Rockville facility to support U.S. clinical development
Financial Results for the Second Quarter and First Half 2020 as compared to the same periods in 2019:

Net loss allocable to common stockholders for the quarter and six months ended June 30, 2020 was $13.5 million and $25.1 million respectively, compared to $12.1 million and $21.4 million
General and administrative expenses for the quarter and six months ended June 30, 2020 were $3.3 million and $6.7 million, respectively, compared to $3.2 million and $6.6 million
Research and development expenses for the quarter and six months ended June 30, 2020 were $10.1 million and $17.8 million respectively, compared $9.1 million and $15.0 million
Net cash used in operating activities for first half of 2020 was $19.8 million, compared to $18.8 million
Our cash, cash equivalents and restricted cash decreased to $13,581,952 at June 30, 2020 compared to $32,443,649 at December 31, 2019. Subsequent to end of the second quarter, we arranged additional borrowings of $29.3 million to fortify our balance sheet.

On August 12, 2020 Antengene Corporation, a leading innovative hematology and oncology-focused biopharmaceutical company, reported the authorization of the first-in-human trial of ATG-017 (ERASER trial) by the Australian Therapeutic Goods Administration (TGA). ATG-017 is a potent and selective small molecule extracellular signal–regulated kinases 1 and 2 (ERK1/2) inhibitor (Press release, Antengene, AUG 12, 2020, View Source [SID1234563540]). The study will enroll patients with advanced solid tumors and hematological malignancies. Today’s milestone marks the first TGA trial authorization for Antengene and is the first clinical trial for ATG-017 globally.

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ATG-017 is an oral, potent and highly selective inhibitor of ERK1/2, which are related protein-serine/ threonine kinases that function as the terminal kinases in the RAS-RAF-MEK-ERK signal transduction cascade. This pathway participates in the control of numerous processes which include apoptosis, cell proliferation, and cellular immune response. In preclinical studies, ATG-017 has proven to regulate a large variety of cellular processes by targeting ERK1/2 and has shown to be effective in inhibiting the viability of tumor cell lines in vitro as well as the tumor growth in vivo.

"This first human trial for ATG-017 in Australia is a significant step for our company’s global clinical strategy and we will initiate overseas trials continuously for a number of novel drugs in our pipeline in the near future," said Dr. Jay Mei, M.D., Ph.D., Founder, Chairman and CEO of Antengene. "Our vision is to treat patients beyond borders. For Australian patients with life-threatening diseases and patients around the world, we are passionately working to continue to develop and commercialize more novel therapies to make a difference in patient lives."

"Aberrations in the MAPK pathway are amongst the most common in malignant cancer, so developing effective drugs targeting this pathway remains a high priority. We are excited to begin this trial with the ERK-targeting agent ATG-017, and bring together our group of highly experienced Australian investigators and sites to begin this collaboration with Antengene," said Associate Professor Jayesh Desai, Head of the Phase I/ Early Drug Development Program at the Peter MacCallum Cancer Centre.

"The RAS-MAPK pathway is a major player in a range of largely incurable hematological malignancies, so the potential to effectively target it with ATG-017 represents an exciting opportunity for Australian cancer patients. We very much look forward to collaborating with Antengene on this new trial," said Professor Andrew Spencer, Head of the Malignant Haematology and Stem Cell Transplantation Service at the Alfred Hospital, Melbourne.

In November 2019, Antengene entered into a licensing agreement with AstraZeneca (LSE/STO/NYSE: AZN) under which Antengene has been granted the exclusive global rights to further develop, manufacture and commercialize AZD0364 (ATG-017). ATG-017 is currently being studied in clinical trials for the treatment of various solid tumors and hematological malignancies.

About ATG-017

ATG-017 is a potent and selective small molecule extracellular signal–regulated kinases 1 and 2 (ERK1/2) inhibitor in clinical development for the treatment of various solid tumors, non-Hodgkin’s lymphoma, acute myeloid leukemia (AML) and multiple myeloma.

On August 12, 2020 QBiotics Group Limited (QGL), a life sciences company developing novel anticancer and wound healing pharmaceuticals, reported that it has entered into an agreement with MSD (tradename of Merck & Co., Inc., Kenilworth, NJ, USA), to evaluate use of its lead molecule tigilanol tiglate, in combination with Keytruda (pembrolizumab) in patients with unresectable melanoma (Press release, QBiotics, AUG 12, 2020, View Source [SID1234563539]).

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Dr Victoria Gordon, Managing Director and CEO of QBiotics, said, "We are delighted to announce this collaboration with MSD. Patients with unresectable melanoma who have received prior checkpoint inhibitors currently have limited effective treatment options. Through this program we hope to see that when combined, tigilanol tiglate and Keytruda may produce additive anti-tumour immune responses, and improve outcomes for patients."

The Phase I/II open label ‘QBC46-H06’ study is a dose escalation and expansion study with the primary objective of determining the maximum tolerated dose or maximum feasible dose of the combination therapy. Secondary measures include assessing tumour responses in both injected tumours and uninjected tumours, as well as clinical efficacy parameters. Patients with unresectable melanoma and who have had exposure to immune checkpoint inhibitors are eligible for the study.

Dr Gordon continued, "This study follows on from encouraging Phase I data where tigilanol tiglate as a monotherapy showed a 27% treatment response rate*, including an 18% complete response with full tumour destruction across a wide variety of solid tumour types[2]. Two patients with melanoma that had complete responses also had an abscopal (anenestic) response. Melanoma is the second human application we are pursuing for tigilanol tiglate following on from our Phase I/II clinical trial in patients with Head and Neck Squamous Cell Carcinoma (HNSCC) which commenced in December 2019".

Tigilanol tiglate is a small molecule administered by intratumoural injection directly into the solid tumour mass. Once injected, it has a multi-modal action including (i) rapid, but highly localised, inflammatory responses, (ii) increased permeability and destruction of tumour vascular endothelium, and (iii) rapid tumour cell death by oncosis[1].

On August 12, 2020 bioAffinity Technologies, a privately held biotech company advancing cutting-edge cancer diagnostics, and Precision Pathology Services, a CAP/CLIA-certified anatomic and clinical pathology laboratory, reported initiation of CLIA validation for CyPath Lung, a non-invasive flow cytometric test for early-stage lung cancer (Press release, BioAffinity Technologies, AUG 12, 2020, View Source [SID1234563538]). Precision Pathology has licensed bioAffinity’s intellectual property for development of CyPath Lung as a laboratory developed test (LDT).

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"With our development phase complete, we are very pleased to start the validation studies for certification of the CyPath Lung flow cytometry test as an LDT," said Roby Joyce, MD, President of Precision Pathology. "This is a very exciting time for us. Lung cancer is the leading cancer killer. Our test for early detection of this dreaded disease can help many people live longer and healthier lives."

The validation study is conducted in accordance with College of American Pathologists (CAP) guidelines and Clinical Laboratory Improvement Amendments (CLIA) regulations. The CAP/CLIA validation study is designed to establish and validate performance characteristics of CyPath Lung, including accuracy, precision, reproducibility and analytical sensitivity, that are necessary for commercialization. Following CyPath Lung certification as an LDT, physicians can order the test for their patients at high risk for lung cancer who receive a positive screening result or are otherwise suspected of having the disease.

"Precision Pathology rightfully enjoys an excellent reputation for quick turnaround times while providing accurate pathology diagnoses. The company is known for its exceptionally responsive and helpful service to the physicians and patients it serves," bioAffinity President Maria Zannes said. "Dr. Joyce and his team will bring the same very high quality to CAP/CLIA validation of CyPath Lung and its eventual sale later this year. CyPath Lung is in excellent hands."

CyPath Lung is a flow cytometric test to aid in the diagnosis of lung cancer. Patients collect sputum samples non-invasively at home, a particular benefit during the COVID-19 pandemic. The sample is shipped overnight to the laboratory for processing. Sample data is acquired by flow cytometry, a technique that can count, sort and profile individual cells with remarkable speed. Using an automated analysis with pre-set parameters, CyPath Lung profiles the lung environment including the presence of cancer-associated cells. Data acquisition and physician reports can be generated in minutes.

In a recent CyPath Lung test validation trial of 150 individuals at high risk for lung cancer including 28 people with diagnosed cancer, the test showed 88% specificity and 82% sensitivity, a positive predictive value of 62% and a negative predictive value of 95%. The test was 87% specific and 92% sensitive in detecting cancer in participants who had nodules of less than 20 mm in diameter, indicating CyPath Lung is highly accurate in finding lung cancer in its earliest stages.

The U.S. Preventive Services Task Force recommends that smokers and former smokers at high risk for lung cancer undergo annual screening by low dose computed tomography (LDCT). Screening by LDCT has been proven to detect lung cancer at earlier stages when it can be more successfully treated. The National Lung Cancer Screening Trial (NLCST) of more than 53,000 participants resulted in a 20% decrease in lung cancer-specific mortality when LDCT screening was performed in high-risk patients. However, screening by LDCT had a low 3.8% positive predictive value (PPV) which raises the risk of unnecessary, invasive and costly procedures for those who test positive. In the NLCST, for every 100 people who received a positive LDCT, less than four of those individuals actually had lung cancer.

"CyPath Lung can assist physicians in determining next steps after a patient presents with a positive LDCT result, particularly in many cases where the lung nodule is considered indeterminate. In our test validation trial, bioAffinity successfully tested the automated analysis program used by CyPath Lung and found it to be fast and very robust in predicting who has cancer and who was at high risk but did not have lung cancer," Zannes said. "Physician reports can be generated immediately after flow cytometry acquires sample data in approximately 20 minutes per sample. Looking beyond lung cancer, we believe our automated platform can be successfully applied to cancer diagnostic tests for several other cancers, which will improve overall survival rates for patients through early diagnosis and treatment. We look forward to working with Precision Pathology in the development of additional life-saving diagnostic tests."

On August 12, 2020 Personalis, Inc. (Nasdaq: PSNL), a leader in advanced genomics for cancer, reported that company management will participate in the 1-on-1 meetings at the upcoming Needham Virtual Cancer Diagnostics 1×1 Conference on Tuesday, August 18, 2020 (Press release, Personalis, AUG 12, 2020, View Source;1 [SID1234563536]).

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