On May 11, 2020 Mustang Bio, Inc. ("Mustang") (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, reported financial results and recent corporate highlights for the first quarter ended March 31, 2020 (Press release, Mustang Bio, MAY 11, 2020, View Source [SID1234557553]).

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Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, "We were pleased to announce several important milestones in the first few months of 2020. Most notably, we were excited to submit our Investigational New Drug ("IND") application with the U.S. Food and Drug Administration ("FDA") for MB-107, our lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency ("XSCID"), also known as bubble boy disease, and we look forward to commencing a multi-center Phase 2 clinical trial in newly diagnosed infants with XSCID under the age of two. We also anticipate filing a second IND in the third quarter of this year for a multi-center trial for the treatment of previously transplanted XSCID patients. Additionally, the European Medicines Agency ("EMA") granted Advanced Therapy Medicinal Product ("ATMP") classification to MB-107, which is an important step in establishing our path to market approval and commercialization in Europe."

"Among our other first quarter accomplishments was the complete response achieved at the lowest dose level in the first subject treated following process optimization in our Phase 1/2 clinical trial of MB-106, our CD20-targeted, autologous CAR T cell therapy, for patients with relapsed or refractory B-cell non-Hodgkin lymphomas. This trial is ongoing, and we hope to announce additional interim data later this year. We are also very pleased to report that MB-105, a PSCA-directed CAR T currently under investigation in a Phase 1 trial at City of Hope, appeared to be active in the first patient to receive the therapy following a standard CAR T conditioning regimen. As we progress through 2020, we look forward to advancing our gene and CAR T cell therapies toward additional potentially value-creating regulatory and clinical milestones in the months ahead," Dr. Litchman concluded.

Recent Corporate Highlights:

·In May 2020, Mustang submitted an IND application with the FDA to initiate a multi-center Phase 2 clinical trial of MB-107 in newly diagnosed infants with XSCID who are under the age of two. The trial is expected to enroll 10 patients who, together with 15 patients enrolled in the current multicenter trial led by St. Jude Children’s Research Hospital, will be compared with 25 matched historical control patients who have undergone hematopoietic stem cell transplant ("HSCT"). The primary efficacy endpoint will be event-free survival. The initiation of this trial is currently on hold pending CMC clearance by the FDA. Mustang is targeting topline data from the trial in the second half of 2022.
·Mustang further expects to file an IND in the third quarter of 2020 for a registrational multi-center Phase 2 clinical trial of its lentiviral gene therapy in previously transplanted XSCID patients. This product will be designated MB-207. Mustang anticipates enrolling 20 patients and comparing them to matched historical control patients who have undergone a second HSCT. Mustang is targeting topline data for this trial in the second half of 2022.
·In the ongoing Phase 1 trial at City of Hope with MB-105, a PSCA-directed CAR T administered systemically to patients with PSCA-positive castration resistant prostate cancer, the first patient to receive the therapy following a standard CAR T conditioning regimen experienced a significant reduction in his prostate-specific antigen ("PSA") at day 28. This PSA response was associated with radiographic improvement of the patient’s metastatic disease.

In April 2020, Mustang announced that the EMA granted ATMP classification to MB-107 for the treatment of XSCID.
·In February 2020, Mustang announced that the first subject treated with the optimized MB-106 (CD20-targeted, autologous CAR T cell therapy) manufacturing process, developed in collaboration between Mustang and Fred Hutchinson Cancer Research Center, achieved a complete response at the lowest starting dose in an ongoing Phase 1/2 clinical trial. The trial is evaluating the safety and efficacy of MB-106 in subjects with relapsed or refractory B-cell non-Hodgkin lymphomas.

Financial Results:

·As of March 31, 2020, Mustang’s cash, cash equivalents and restricted cash totaled $56.8 million, compared to $62.4 million as of December 31, 2019, a decrease of $5.6 million for the first quarter.
·Research and development expenses were $9.3 million for the first quarter of 2020. This compares to $7.0 million for the first quarter of 2019. Non-cash, stock-based compensation expenses included in research and development were $0.4 million for the first quarter of 2020, compared to $0.1 million for the first quarter of 2019.
·Research and development expenses from license acquisitions totaled $0.3 million for the first quarter of 2020, compared to $0.5 million for the first quarter of 2019.
·General and administrative expenses were $2.0 million for the first quarter of 2020. This compares to $2.3 million for the first quarter of 2019. Non-cash, stock-based compensation expenses included in general and administrative expenses were $0.4 million for the first quarter of 2020, compared to $0.7 million for the first quarter of 2019.
·Net loss attributable to common stockholders was $11.9 million, or $0.28 per share, for the first quarter of 2020, compared to a net loss attributable to common stockholders of $9.6 million, or $0.34 per share, for the first quarter of 2019.

On May 10, 2020 Hummingbird Bioscience, an innovative biotherapeutics company focused on the discovery and development of new breakthrough therapies, reported that it has closed an extended Series B funding round of US$25 million (Press release, Hummingbird Bioscience, MAY 10, 2020, View Source [SID1234557436]). This brings the total capital raised through financing activities and strategic partnerships to more than US$65 million to date.

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Leading the Series B extension is new investor SK Holdings, with participation from existing shareholders including Heritas Capital and SEEDS Capital, the investment arm of Enterprise Singapore.

The Series B round was extended to US$25 million due to significant over-subscription, with the addition of select quality and value-add investors. Hummingbird will use the new funds to accelerate development of new candidates into clinical trials and strengthen its scientific and research and development capabilities.

"We are delighted to have SK Holdings join our investor base. Hummingbird is building a strong portfolio of promising new therapies that we believe can deliver very meaningful benefit for patients across a broad spectrum of disease. These new funds give us further resources to develop our early stage pipeline, and support the clinical development of our lead programs," said Dr Piers Ingram, Chief Executive Officer and co-founder, Hummingbird Bioscience.

"Heritas Capital is pleased to continue our backing of the Hummingbird team since leading its Series A extended round," commented Chik Wai Chiew, Executive Director and Chief Executive Officer, Heritas Capital Management. "Even as the COVID-19 pandemic has resulted in a slow-down in investing, we are mindful that backing leading innovative biotech companies, especially players such as Hummingbird, to develop cures for addressing patients’ needs remains our priority."

Earlier this year Hummingbird announced publication of positive data on its lead candidate, HMBD-001, a first-in-class HER3 antibody; and manufacturing of HMBD-002, a first-in-class VISTA antibody. Regulatory submissions to initiate Phase I studies for these two candidates are expected in the second half of 2020.

On May 10, 2020 Ascentage Pharma (6855.HK), a globally focused, clinical-stage biotechnology company engaged in developing novel therapies for cancers, chronic hepatitis B (CHB), and age-related diseases, reported that latest clinical data of the company’s drug candidates, including the MDM2-p53 inhibitor APG-115, novel Bcl-2/Bcl-xL dual inhibitor APG-1252, and IAP inhibitor APG-1387, will be presented in three poster discussions and a poster at the 2020 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (Press release, Ascentage Pharma, MAY 10, 2020, View Source [SID1234557437]). Because of concerns about the current coronavirus (COVID-19) pandemic, this year’s annual meeting will be held in a virtual format from May 29 through May 31, 2020.

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The ASCO (Free ASCO Whitepaper) Annual Meeting is the most authoritative meeting in the oncology space. The purpose of this meeting is to disseminate premier scientific research and cutting-edge treatment approaches in oncology to the world.

"We are excited to present the clinical advances of three of our drug candidates," said Dr. Yifan Zhai, Chief Medical Officer of Ascentage Pharma. "Through these results, Ascentage Pharma is demonstrating its capabilities of global clinical development in apoptosis. We are striving to offer more treatment options for patients with unmet medical needs."

Four posters will be presented at ASCO (Free ASCO Whitepaper)20, including:

Poster Discussion Sessions

Phase Ib study of a novel, small-molecule MDM2 inhibitor APG-115 combined with pembrolizumab in U.S. patients with metastatic solid tumors
Time: May 29th, 8:00–11:00am, EST

Abstract: #3512

Session: Developmental Therapeutics—Molecularly Targeted Agents and Tumor Biology

First-in-human study of palcitoclax (APG-1252), a novel dual Bcl-2/Bcl-xL inhibitor, demonstrated advantages in platelet safety while maintaining anticancer effect in U.S. patients with metastatic solid tumors
Time: May 29th, 8:00–11:00am, EST

Abstract: #3509

Session: Developmental Therapeutics—Molecularly Targeted Agents and Tumor Biology

Phase Ib study of a novel bivalent IAP antagonist APG-1387 in combination of pembrolizumab for patients with advanced solid tumors
Time: May 29th, 8:00–11:00am, EST

Abstract: #3508

Session: Developmental Therapeutics—Molecularly Targeted Agents and Tumor Biology

Poster Session

Phase I study results of APG-115, a MDM2-p53 antagonist, in Chinese patients with advanced liposarcoma and other solid tumors
Time: May 29th, 8:00–11:00am, EST

Abstract: #11542

Session: Sarcoma

On May 9, 2020 Avalon GloboCare Corp. (NASDAQ: AVCO), a clinical-stage global developer of cell-based technologies and therapeutics, reported that it has achieved significant milestones, advancing its next generation immune cell therapy using FLASH-CAR technology co-developed with the Company’s strategic partner Arbele Limited (Press release, Avalon, MAY 9, 2020, View Source [SID1234609546]). The adaptable FLASH-CAR platform can be used to create personalized cell therapy from a patient’s own cells, as well as off-the-shelf cell therapy from a universal donor.
Currently available Chimeric Antigen Receptor T (CAR-T) cellular immunotherapy involves a patient’s own T-cells—a type of white blood cell that protects against infections and other diseases including cancer—that are turned into personalized cancer fighting cells. The T-cells are removed from the patient, reprogrammed in the lab using a viral vector to target cancer cells, and infused back into the patient as a cancer immunotherapy.

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In contrast to these existing therapies, Avalon’s FLASH-CAR uses next generation CAR technology to modify patients’ T-cells and natural killer (NK) cells using a ribonucleic acid (RNA)-based platform rather than a viral vector. Similar to T-cells, NK cells are a type of white blood cell, also able to attack cancer cells, but utilize different mechanisms. By using RNA molecules rather than a viral vector, Avalon’s RNA-based CAR technology is designed to rapidly create personalized CAR therapies in 1 to 2 days compared to the 10- to 14-day bio-manufacturing time necessary to generate currently available CAR-T cellular immunotherapy. Avalon’s FLASH-CAR technology is also designed to reprogram the immune cells to hone in on multiple crucial cancer cell targets, called tumor antigens, to potentially achieve superior therapeutic effect. Avoiding the use of viral vectors and complicated bio-processing procedures significantly reduces manufacturing costs, resulting in a more affordable and potentially breakthrough therapy for cancer patients. The FLASH-CAR technology can also be used to generate "off-the-shelf", universal cell therapy that has the potential to reach even more patients.

Avalon’s first FLASH-CAR platform candidate, AVA-011, targets both CD19 and CD22 tumor antigens on cancer cells. Pre-clinical research on AVA-011, including tumor cytotoxicity studies, has been successfully completed and Avalon is immediately entering the process development stage to generate clinical-grade CAR-T and CAR-NK cells for use in human clinical trials. Avalon and Arbele have jointly filed for USPTO provisional and PCT patents for this RNA-based CAR platform cellular therapy and for other applications.

Avalon expects to begin a first-in-human clinical trial with AVA-011 for the treatment of relapsed or refractory B-cell lymphoblastic leukemia (B-ALL) and non-Hodgkin lymphoma in the first quarter of 2021. The goal is to use AVA-011 as a bridge to bone marrow stem cell transplant therapy, currently the only curative approach for patients with these blood cancers.

"Avalon GloboCare is committed to decreasing the time it takes to deliver cellular immunotherapies to cancer patients, as well as lowering the cost of manufacturing by building on our unique RNA-based CAR platform that does not require using a viral vector," stated David Jin, M.D., Ph.D., President and Chief Executive Officer of Avalon GloboCare. "We are accelerating our innovative discovery and development plan, as well as delivering precise clinical execution and leadership in cellular immunotherapy. Our pre-clinical studies are encouraging and we are excited for AVA-011 to enter the clinical development stage, including multi-center clinical trials following completion of process development to generate the cell therapy," said Dr. Jin.

"Through this strategic partnership with Avalon GloboCare, we envision an accelerated scientific and clinical development of the RNA-based FLASH-CAR technology platform with great potential to generate "off-the-shelf" immune effector cell therapies to treat both hematologic and solid malignancies," said John Luk, Ph.D., EMBA, President and Chief Executive Officer of Arbele Limited.

On May 9, 2020 Cinda Biopharmaceutical (Hong Kong Stock Exchange: 01801), an innovative drug dedicated to the development, production and sale of major diseases such as cancer, autoimmunity, metabolic diseases, etc. The biopharmaceutical company, and Eli Lilly (NYSE: LLY) reported : A randomized, innovative, and innovative PD-1 inhibitor Daboshu (Sindilimumab Injection) jointly developed by the two parties (Press release, Innovent Biologics, MAY 9, 2020, View Source [SID1234557375]). Double-blind, phase III controlled clinical study (ORIENT-12)-Daboshu (sindilimumab injection, hereinafter referred to as sinidiumab) combined with Gemcitabine (gemcitabine for injection, hereinafter referred to as gemcitabine) Platinum-based chemotherapy is used in the first-line treatment of advanced or metastatic squamous non-small cell lung cancer (NSCLC) to reach the primary study endpoint. This is the world ’s first randomized, double-blind, phase III clinical study evaluating PD-1 antibody combined with Gemcitabine (gemcitabine) and platinum for first-line treatment of squamous NSCLC.

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Studies have shown that Sindilimum combined with gemcitabine and platinum compared with placebo combined with gemcitabine and platinum, significantly prolonged progression-free survival (PFS), reached the preset study endpoint, PFS significantly benefited, safety characteristics and previous reports The research results of xindilimumab were consistent and there was no new safety signal. Detailed research results will be announced in subsequent academic conferences.

Cinda and Eli Lilly plan to communicate with the Drug Evaluation Center (CDE) of the State Drug Administration on the submission of a new indication for the application of cindilimumab combined with gemcitabine and platinum for first-line treatment of squamous NSCLC.

Professor Zhou Caicun, director of the Department of Oncology, Shanghai Pulmonary Hospital, said: "Lung cancer accounts for the first cause (25.2%) of all cancer deaths, of which NSCLC accounts for approximately 80% to 85%. About 35% of NSCLC are squamous NSCLC. Over the past two decades, the development of drugs for the treatment of NSCLC has mainly focused on non-squamous NSCLC. Due to the lack of driver genes and special tumor biological characteristics, drug development has been slow. The emergence of anti-PD-1 antibodies is This type of patient brings a new treatment. We see that the study of Sindilimum reached the preset primary study end point. The ORIENT-12 study compared with the KEYNOTE-407 study of pabolizumab Using different combination chemotherapy regimens, it is the first randomized controlled study in the world to confirm that PD-1 antibody combined with gemcitabine and platinum can significantly improve the benefit of progression-free survival in patients with first-line squamous NSCLC. "

Dr. Hui Zhou, Vice President of the Medical Science and Strategic Oncology Department of Cinda Biopharmaceutical Group, said: "Currently Cindylimumab is the only anti-PD-1 monoclonal antibody drug listed in the National Medical Insurance List. It was awarded the National Drug Administration in 2018 Approved by the Administration for the treatment of relapsed or refractory classic Hodgkin’s lymphoma after at least second-line chemotherapy. We are currently conducting a number of phase III randomized controlled studies on Sindrinimab. ORIENT-12 The results of the study are encouraging, and we foresee the potential of Sindilimumab to benefit more lung cancer patients. This is also the main study of Cinda Biological following ORIENT-11 (first-line non-squamous NSCLC randomized, double-blind, phase III study, NCT03607539) After the end point, the second successful randomized controlled study of lung cancer. "

Dr. Li Wang, senior vice president of Eli Lilly China and head of the Center for Drug Development and Medical Affairs, said, "Not long ago, the Drug Evaluation Center (CDE) of the State Drug Administration accepted Cindilimum for non-squamous non-small The application of new indications for first-line treatment of cell lung cancer (nsqNSCLC). And the exciting research results of ORIENT-12 have reflected the potential of Sindilimumab in the treatment of squamous non-small cell lung cancer. Here we think Thanks to all the patients and their families, researchers and clinical trial centers, and Cinda colleagues who participated in the study. We look forward to bringing this new treatment to lung cancer patients in China as soon as possible. "

About ORIENT-12 research

The ORIENT-12 study is a randomized evaluation of the efficacy and safety of Daboshu (sintilimab) or placebo in combination with Gemcitabine (gemcitabine) and platinum for first-line treatment of advanced or metastatic squamous NSCLC , Double-blind, Phase III controlled clinical study (ClinicalTrials.gov, NCT03629925). The primary study endpoint was progression-free survival (PFS) assessed by the Independent Imaging Review Committee (IRRC) according to the RECIST v1.1 standard. Secondary study endpoints include overall survival (OS) and safety.

A total of 357 subjects were enrolled in this study. They were randomized 1: 1 and received Daboshu (Sintilimab injection) 200 mg or placebo in combination with Gemcitabine (gemcitabine for injection) and platinum Treatment, given once every 3 weeks, after completing 4 or 6 cycles of combination therapy, enter Daboshu (sindilimumab injection) or placebo for maintenance therapy until disease progression, toxicity intolerance or other Circumstances where treatment needs to be terminated. After the disease progresses in the control group, it can be conditionally crossed to monotherapy of daboshu (sintilimab injection).

About squamous non-small cell lung cancer (sqNSCLC)

Lung cancer is the first malignant tumor in China that ranks first in both morbidity and mortality. NSCLC accounts for about 80% to 85% of all lung cancers, and about 70% of NSCLC patients are diagnosed as locally advanced or metastatic tumors that are not suitable for radical surgery. At the same time, a considerable proportion of early-stage NSCLC patients undergoing surgery will have recurrence or distant metastasis, and then die due to disease progression. About 35% of Chinese patients with NSCLC are squamous NSCLC. Squamous NSCLC lacks driver genes, and the effectiveness of first-line chemotherapy is about 30%. At present, only paclizumab combined with carboplatin and paclitaxel has been approved by the FDA and the National Drug Administration (NMPA) for this population. Treatment is still limited, and there are huge unmet medical needs.

About Daboshu (Sintilimab Injection)

Daboshu (Sintilimab Injection) is an innovative biopharmaceutical of international quality jointly developed by Cinda Biopharma and Eli Lilly in China. The first indication for its approval is relapsed / refractory classic Hodgkin’s lymphoma, which was included in the 2019 edition of the Chinese Society of Clinical Oncology (CSCO) lymphoma diagnosis and treatment guidelines. In April 2020, the National Drug Administration (NMPA) formally accepted the application for indications for the first-line treatment of non-squamous non-small cell lung cancer (nsqNSCLC) with dabershu (sintilimab injection). In the medical insurance country talks in 2019, Dabex (Sintilimab Injection) is the only PD-1 inhibitor that enters the National Health Insurance.

Dabshu (Sindilimumab Injection) is a human immunoglobulin G4 (IgG4) monoclonal antibody that specifically binds to PD-1 molecules on the surface of T cells, thereby blocking tumor immune tolerance The PD-1 / Programmed Death Receptor Ligand 1 (Programmed Death-Ligand 1, PD-L1) pathway reactivates the anti-tumor activity of lymphocytes to achieve the purpose of treating tumors. There are currently more than twenty clinical studies (of which more than 10 are registered clinical trials) are underway to explore the anti-tumor effect of sundilimumab on other solid tumors. Cinda Biotech is also conducting clinical research work on Cindylimumab injections worldwide.