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.