On November 13, 2018 Mustang Bio, Inc. ("Mustang") (NASDAQ: MBIO), a company focused on the
development of novel immunotherapies based on proprietary chimeric antigen receptor engineered T cell ("CAR T")
technology and gene therapies for rare diseases, reported financial results and recent corporate highlights for the third quarter ended September 30, 2018 (Press release, Mustang Bio, NOV 13, 2018, View Source [SID1234531346]).
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Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, "The third quarter of 2018 and recent months have been marked by clinical progress and a key addition to our management team with the appointment of Martina A. Sersch, M.D., Ph.D., as Chief Medical Officer. Notably, we expanded our pipeline into gene therapy by securing an exclusive worldwide license agreement with St. Jude Children’s Research Hospital ("St. Jude") for the development of a first-in-class, ex vivo, clinical-stage lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency ("X-SCID"). Data from a multicenter Phase 1/2 trial led by St. Jude in infants under the age of two years old are extremely encouraging. Eight patients under the age of two with X-SCID have been treated to date, with results presented at the 21st Annual Meeting of the American Society of Gene & Cell Therapy in May 2018. The therapy was well tolerated. In addition, six patients achieved reconstituted immune systems within three to four months following treatment, with the remaining two patients continuing to progress favorably in earlier stages of recovery. Four of these six patients have discontinued monthly infusions of intravenous immunoglobulin, and the remaining patients, at earlier stages of recovery, continue to progress favorably. In three patients who had disseminated infections prior to therapy, all infections resolved completely. In addition, the therapy is being investigated in patients over the age of two in a second Phase 1/2 trial at the National Institutes of Health ("NIH"), with equally encouraging data and an excellent safety profile to date. The two patients with the longest follow-up have seen sustained restoration of antibody production after immunization, and all five patients treated experienced a decrease in viral infections and overall clinical improvement."
Dr. Litchman continued, "We also recently announced updates on two Phase 1 clinical trials at City of Hope using our
HER2-specific CAR T cell therapy, including a first-of-its-kind trial using intraventricular delivery of CAR T cells to brains of patients with HER2-positive breast cancer with brain metastases. As we look ahead to the fourth quarter of 2018, we look forward to filing our first Investigational New Drug (IND) application to support a Phase 1/2 trial of MB-102 in acute myeloid leukemia ("AML"), blastic plasmacytoid dendritic cell neoplasm and high-risk myelodysplastic syndrome."
Recent Corporate Highlights:
• In July 2018, Mustang completed a pre-IND meeting with the U.S. Food and Drug Administration ("FDA") for MB102 (CD123 CAR T). Based on the meeting, Mustang expects to file an IND in the fourth quarter of 2018 to support a Phase 1/2 trial of MB-102 in AML, blastic plasmacytoid dendritic cell neoplasm and high-risk myelodysplastic
syndrome.
• In August 2018, Mustang announced that it entered into an exclusive worldwide license agreement with St. Jude
for the development of a first-in-class ex vivo lentiviral gene therapy for the treatment of X-SCID, also known as
bubble boy disease. The therapy is currently being evaluated in a Phase 1/2 multicenter trial in infants under the
age of two. This study is the world’s first lentiviral gene therapy trial for infants with X-SCID. The therapy is also
being investigated in patients over the age of two in a second Phase 1/2 trial at the National Institutes of Health
("NIH").
• In October 2018, Mustang appointed Martina A. Sersch, M.D., Ph.D., as Chief Medical Officer.
• Also in October 2018, Mustang announced that a first-of-its-kind Phase 1 clinical trial evaluating the safety and
effectiveness of intraventricular delivery of CAR T cells to the brains of patients with HER2-positive breast cancer
with brain metastases has been initiated at City of Hope. In addition, Mustang announced that City of Hope dosed
the first patient in a Phase 1 clinical trial of HER2-specific CAR T cells in treating recurrent or refractory grade IIIIV
glioma. The trial will evaluate the side effects and best dose of HER2-specific CAR T cells in treating patients
with grade III-IV glioma that has come back or does not respond to treatment.
Financial Results:
• As of September 30, 2018, Mustang’s consolidated cash, cash equivalents, short-term investments (certificates of
deposit) and restricted cash totaled $41.3 million, compared to $47.2 million as of June 30, 2018, and $61.5 million
as of December 31, 2017, a decrease of $5.9 million for the quarter and a decrease of $20.2 million year-to-date.
• Research and development expenses were $5.3 million for the third quarter of 2018, compared to $2.2 million for
the third quarter of 2017. Non-cash, stock-based compensation expenses included in research and development
were $0.7 million for third quarter of 2018, compared to $0.3 million for the third quarter of 2017.
• Research and development expenses from license acquisitions were $1.0 million for the third quarter of 2018,
compared to $0.3 million for the third quarter of 2017.
• General and administrative expenses were $1.3 million for the third quarter of 2018, compared to $4.6 million for
the third quarter of 2017. Non-cash, stock-based compensation expenses included in general and administrative
expenses were $0.2 million for the third quarter of 2018, compared to $0.5 million for the third quarter of 2017.
• Net loss attributable to common stockholders was $7.5 million, or $0.28 per share, for the third quarter of 2018,
compared to $6.9 million, or $0.27 per share, for the third quarter of 2017. Net loss attributable to common
stockholders was $18.9 million, or $0.70 per share, for the first nine months of 2018, compared to $15.7 million,
or $0.63 per share, for the first nine months of 2017.