On August 11, 2022 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 second quarter ended June 30, 2022 (Press release, Mustang Bio, AUG 11, 2022, View Source [SID1234618138]).
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Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, "We are very pleased with the significant clinical and regulatory milestones achieved in the first half of 2022 for our portfolio of cell and gene therapies. Interim Phase 1/2 data on MB-106, our CD20-targeted, autologous CAR T cell therapy for patients with relapsed or refractory B-cell non-Hodgkin lymphomas ("B-NHLs") and chronic lymphocytic leukemia ("CLL"), were presented at several prestigious medical meetings. MB-106 continues to demonstrate high efficacy and a favorable safety profile across all patients with a wide range of hematologic malignancies including follicular lymphoma ("FL"), CLL, diffuse large B-cell lymphoma ("DLBCL") and Waldenstrom macroglobulinemia ("WM"), with no cytokine release syndrome or immune effector cell-associated neurotoxicity syndrome greater than grade 2. Additionally, the U.S. Food and Drug Administration ("FDA") granted Orphan Drug Designation to MB-106 for WM, a rare type of B-NHL. We are excited by the continued progress of MB-106 and anticipate dosing the first patient shortly in a multicenter Phase 1/2 clinical trial evaluating the safety and efficacy of MB-106 for relapsed or refractory B-NHL and CLL under Mustang’s Investigational New Drug application ("IND"). Furthermore, we expect to enroll 3 to 6 patients in this trial by the end of 2022 and to disclose data from both the single-center Fred Hutch trial and the multicenter Mustang trial in the fourth quarter of this year. We also continued to advance our recurrent glioblastoma ("rGBM") clinical program. Data presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) ("AACR") Annual Meeting 2022 support the safety of administering our two clinical candidates, MB-108 (Nationwide Children’s herpes simplex virus type 1 oncolytic virus) and MB‐101 (City of Hope’s IL13Rα2‐targeted CAR T cell therapy), sequentially to optimize treatment in a regimen designated as MB-109, for which we plan to file an IND in 2023."
"Mustang is pleased to be a leader in the development of gene therapy treatments for severe combined immunodeficiency ("SCID") patients. Interim Phase 1/2 data on treatment with the same lentiviral vector used in MB-107, our ex vivo lentiviral gene therapy for X-linked SCID ("XSCID") in newly diagnosed infants under the age of two, presented at the American Society of Gene & Cell Therapy ("ASGCT") 25th Annual Meeting showed all 23 treated patients were alive at 2.6-year median follow-up without evidence of malignant transformation, and the treatment established a stable, functioning immune system in patients. We also announced that the first patient successfully received LV-RAG1 ex vivo lentiviral gene therapy to treat recombinase-activating gene-1 ("RAG1") SCID ("RAG1-SCID"), in an ongoing Phase 1/2 multicenter clinical trial taking place in Europe. Mustang has exclusively licensed LV-RAG1 for the development of MB-110, a first-in-class ex vivo lentiviral gene therapy for RAG1-SCID. XSCID and RAG1-SCID make up almost 60% of all SCID cases combined. We look forward to the continued advancement of our SCID clinical program, including our anticipated initiation in 2023 under Mustang’s IND of multicenter pivotal Phase 2 trials for both MB-107 in newborn XSCID patients and MB-207 in previously transplanted XSCID patients."
Recent Corporate Highlights:
In April 2022, Mustang announced that interim Phase 1/2 clinical trial data on MB-106, a CD20-targeted, autologous CAR T cell therapy for patients with relapsed or refractory B-cell NHL and CLL, were presented at the 2022 Tandem Meetings I Transplantation & Cellular Therapy Meetings of the American Society of Transplantation and Cellular Therapy and Center for International Blood & Marrow Transplant Research. Data demonstrated high efficacy and a favorable safety profile in all patients (n=25). Five dose levels were used during the study, and CRs were observed at all dose levels. Durable responses were observed in a wide range of hematologic malignancies including FL, CLL, DLBCL, and WM. An overall response rate ("ORR") of 96% and a complete response rate ("CR") of 72% were observed in all patients across all dose levels. Within the next 60 days, Mustang expects to dose the first patient in a multicenter Phase 1/2 clinical trial evaluating the safety and efficacy of MB-106 for relapsed or refractory B-NHL and CLL, and further expects to enroll 3 to 6 patients in this trial by the end of 2022.
Also in April 2022, MB-106 data focused on CLL were presented at the 4th International Workshop on CAR-T and Immunotherapies.
Additionally, in April 2022, Mustang announced interim data from two ongoing investigator-sponsored Phase 1 clinical trials evaluating two clinical candidates, MB‐101 (IL13Rα2‐targeted CAR T cell therapy licensed from City of Hope) and MB-108 (herpes simplex virus type 1 oncolytic virus licensed from Nationwide Children’s Hospital) for the treatment of recurrent glioblastoma ("rGBM"). The data were from a late-breaking poster presented at the AACR (Free AACR Whitepaper) Annual Meeting 2022. Preclinical data also presented support the safety of administering these two therapies sequentially to optimize treatment in a regimen designated as MB-109. Mustang expects to file an IND in 2023 to initiate an MB-109 Phase 1 clinical trial.
In May 2022, interim Phase 1/2 data on treatment with the same lentiviral vector used in MB-107, Mustang’s lentiviral gene therapy for XSCID, also known as bubble boy disease, in newly diagnosed infants under the age of two, were presented in an oral presentation during the Clinical Trials Spotlight Symposium at the ASGCT (Free ASGCT Whitepaper) 25th Annual Meeting. The presentation included updated data from a multicenter Phase 1/2 clinical trial for XSCID in newly diagnosed infants under the age of two at St. Jude Children’s Research Hospital, UCSF Benioff Children’s Hospital in San Francisco and Seattle Children’s Hospital. All 23 treated patients were alive at 2.6-year median follow-up without evidence of malignant transformation.
In June 2022, MB-106 CD20-targeted autologous CAR T cell therapy data were presented in an oral session at the European Hematology Association (EHA) (Free EHA Whitepaper) 2022 Hybrid Congress. Mazyar Shadman, M.D., M.P.H., Associate Professor and physician at Fred Hutchinson Cancer Center and University of Washington presented updated interim data from the ongoing Phase 1/2 clinical trial for B-NHL and CLL. Data presented included a 94% ORR and 78% CR in 18 patients with FL. Overall, for the 26 patients treated on the trial, there was a 96% ORR and 73% CR, including complete responses in both DLBCL patients, both WM patients, and both patients previously treated with CD19-targeted CAR-T therapy (1 DLBCL patient and 1 FL patient).
Also in June 2022, the FDA granted Orphan Drug Designation to MB-106 CD20-targeted autologous CAR T cell therapy for the treatment of WM, a rare type of B-NHL.
In July 2022, Mustang announced that the first patient successfully received LV-RAG1 ex vivo lentiviral gene therapy to treat RAG1-SCID, in an ongoing Phase 1/2 multicenter clinical trial taking place in Europe. LV-RAG1 is exclusively licensed by Mustang for the development of MB-110, a first-in-class ex vivo lentiviral gene therapy for the treatment of RAG1-SCID.
In 2023, Mustang expects to enroll the first patient in a pivotal multicenter Phase 2 clinical trial under Mustang’s IND to evaluate MB-107, a lentiviral gene therapy for the treatment of infants under the age of two with XSCID.
Mustang filed an IND application in December 2021 for its pivotal multicenter Phase 2 clinical trial of MB-207, a lentiviral gene therapy for the treatment of patients with XSCID who have been previously treated with hematopoietic stem cell transplantation ("HSCT") and for whom re-treatment is indicated. The trial is currently on hold pending CMC clearance from the FDA and, based on feedback from the Agency, Mustang expects to enroll the first patient in a pivotal multicenter Phase 2 clinical trial in 2023.
Financial Results:
As of June 30, 2022, Mustang’s cash and cash equivalents and restricted cash totaled $108.4 million, compared to $123.2 million at March 31, 2022 and $110.6 million as of December 31, 2021, a decrease of $14.8 million for the quarter and a decrease of $2.2 million year-to-date.
Research and development expenses were $15.2 million for the second quarter of 2022, compared to $11.9 million for the second quarter of 2021. Non-cash, stock-based expenses included in research and development were $0.4 million for the second quarter of 2022, compared to $0.3 million for the second quarter of 2021.
General and administrative expenses were $3.1 million for the second quarter of 2022, compared to $2.5 million for the second quarter of 2021. Non-cash, stock-based expenses included in general and administrative expenses were $0.2 million for the second quarter of 2022, compared to $0.2 million for the second quarter of 2021.
Net loss attributable to common stockholders was $19.1 million, or $0.19 per share, for the second quarter of 2022, compared to a net loss attributable to common stockholders of $14.4 million, or $0.16 per share, for the second quarter of 2021.