8. Sponsored Research and Clinical Trial Agreements

Aevitas

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On January 25, 2018, Aevitas entered into a Sponsored Research Agreement with the University of Massachusetts (“UMass SRA”) for certain continued research and development activities related to the development of adeno-associated virus (“AAV”) gene therapies in complement-mediated diseases. The total amount to be funded by Aevitas under the UMass SRA is $0.8 million. Pursuant to the terms of the UMass SRA, Aevitas paid $0.8 million which was due upon execution. On May 31, 2020, a First Amendment to the UMass SRA was signed and the total amount to be funded was $0.7 million, including $0.4 million due within 30 days of execution. For the years ended December 31, 2020 and 2019, Aevitas recorded expense of approximately $0.4 million and nil, respectively, in connection with the UMass SRA. The expense was recorded in research and development expenses in the Company’s Consolidated Statement of Operations.

On July 24, 2018, Aevitas entered into a Sponsored Research Agreement with the Trustees of the University of Pennsylvania (“UPenn SRA”) for certain continued research and development activities related to the development of AAV gene therapies in complement-mediated diseases. The total amount to be funded by Aevitas under the UPenn SRA is $2.0 million. Pursuant to the terms of the UPenn SRA, Aevitas paid $0.3 million which was due upon execution. For the years ended December 31, 2020 and 2019, Aevitas recorded expense of approximately $0.6 million and $1.1 million, respectively, in connection with the UPenn SRA. The expense was recorded in research and development expenses in the Company’s Consolidated Statement of Operations.

On September 1, 2019, Aevitas entered into a Sponsored Research Arrangement (“SRA”) with Duke University School of Medicine (“Duke”). For the years ended December 31, 2020 and 2019, Aevitas recorded approximately nil and $0.1 million, respectively, for the purpose of conducting a study to identify a dose range for AAV8 vectors in Dry Age-related Macular Degeneration (“Dry AMD”) in research and development expense on the Consolidated Statement of Operations.  

Cellvation

In October 2016, Cellvation entered research funding agreement with the University of Texas in connection with the license for a method and apparatus for conditioning cell populations for cell therapies. In connection with this agreement Cellvation agreed to fund $0.8 million of research quarterly through March 31, 2018. The agreement was revised effective May 1, 2017, with quarterly payments extended through December 31, 2018. For the years ended December 31, 2020 and 2019, Cellvation recorded an expense of nil and $0.1 million, respectively, representing amounts due under this arrangement.

Mustang

For the years ended December 31, 2020 and 2019 Mustang recorded the following expense in research and development for sponsored research and clinical trial agreements:

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City of Hope Sponsored Research Agreement

In March 2015, in connection with Mustang’s license with COH for the development of CAR T, Mustang entered into a Sponsored Research Agreement in which Mustang will fund continued research in the amount of $2.0 million per year, payable in four equal annual installments, until 2020. The research covered under this arrangement is for IL13Rα2 (MB-101), CD123 (MB-102) and the Spacer technology. For the years ended December 31, 2020 and 2019, Mustang incurred expense of $0.5 million and $2.0 million, respectively and recorded as research and development expense in the Company’s Consolidated Statement of Operations.

CD123 (MB-102) Clinical Research Support Agreement

On February 17, 2017, Mustang entered into a Clinical Research Support Agreement for CD123. Pursuant to the terms of this agreement, Mustang made an upfront payment of approximately $20,000 and will contribute an additional $0.1 million per patient in connection with the on-going investigator-initiated study. Further, Mustang agreed to fund approximately $0.2 million over three years pertaining to the clinical development of CD123. For the years ended December 31, 2020 and 2019, Mustang recorded approximately $0.4 million and $1.2 million, respectively, in research and development expenses in the Company’s Consolidated Statements of Operations.

CS1(MB-104) Clinical Research Support Agreement

In June 2020, Mustang entered into a clinical research and support agreement with COH in connection with an Investigator-sponsored study conducted under an Institutional Review Board-approved, investigator-initiated protocol entitled: "Phase I Study to Evaluate Cellular Immunotherapy Using Memory-Enriched T Cells Lentivirally Transduced to Express a CS1-Targeting, Hinge-Optimized, 41BB-Costimulatory Chimeric Antigen Receptor and a Truncated EGFR Following Lymphodepleting Chemotherapy in Adult Patients with CS1+ Multiple Myeloma." The CAR T being studied under this protocol has been designated by Mustang as MB-104. Under the terms of the agreement Mustang will reimburse COH for costs associated with this trial not to exceed $2.4 million. The agreement will expire upon the delivery of the final study report or earlier. During the year ended December 31, 2020, Mustang recorded approximately $0.9 million in research and development expenses in the Company’s Consolidated Statement of Operations pursuant to this agreement.

IL13Rα2 (MB-101) Clinical Research Support Agreements

On February 17, 2017, Mustang entered into a Clinical Research Support Agreement for IL13Rα2 (the “IL13Rα2 GBM CRA”). Pursuant to the terms of this agreement Mustang made an upfront payment of approximately $9,300 and will contribute an additional $0.1 million per patient in connection with the on-going investigator-initiated study. Further, Mustang agreed to fund approximately $0.2 million over three years pertaining to the clinical development of IL13Rα2.

In October 2020, Mustang entered into a Clinical Research Support Agreement for the IL13Rα2 directed CAR T program for adult patients with Leptomeningeal Glioblastoma, Ependymoma or Medulloblastoma (the “IL13Rα2 Leptomeningeal CRA”). Pursuant to the terms of the IL13Rα2 Leptomeningeal CRA, Mustang made an upfront payment of $29,375 and will contribute an additional $0.1 million per patient in connection with the on-going investigator-initiated study. Further, the Company agreed to fund approximately $0.2 million annually pertaining to the clinical development of IL13Rα2.

For the years ended December 31, 2020 and 2019, Mustang recorded approximately $0.5 million and $0.9 million, respectively, in research and development expenses under the IL13Rα2 CRAs in the Company’s Consolidated Statement of Operations.

HER2 (MB-103) Clinical Research Support Agreement

In September 2020, Mustang entered into a clinical research support agreement with COH in connection with an Investigator-sponsored study conducted under an Institutional Review Board-approved, investigator-initiated protocol entitled: “Phase I Study of Cellular Immunotherapy using Memory-Enriched T Cells Lentivirally Transduced to Express a HER2-Specific, Hinge-Optimized, 41BB-Costimulatory Chimeric Receptor and a Truncated CD19 for Patients with Recurrent/Refractory Malignant Glioma.” The CAR T being studied under this protocol has been designated as MB-103. Under the terms of the agreement Mustang will pay COH $29,375 upon execution and will reimburse COH for costs associated with this trial not to exceed $3.0 million. The agreement will expire upon the delivery of a final study report or earlier. For the year ended December 31, 2020, Mustang recorded $1.5 million in research and development expenses in the Company’s Consolidated Statement of Operations pursuant to this agreement.

PSCA (MB-105) Clinical Research Support Agreement

In October 2020, Mustang entered into a clinical research support agreement with COH in connection with an Investigator-sponsored study conducted under an Institutional Review Board-approved, investigator-initiated protocol entitled: “A Phase 1b study to evaluate PSCA-specific chimeric antigen receptor (CAR)-T cells for patients with metastatic castration resistant prostate cancer.” The CAR T being studied under this protocol has been designated as MB-105. Under the terms of the agreement Mustang will pay COH $33,000 upon execution and will reimburse COH for costs associated with this trial not to exceed $2.3 million.  The agreement will expire upon the delivery of a final study report or earlier. For the year ended December 31, 2020, Mustang recorded $0.2 million in research and development expenses in the Company’s Consolidated Statement of Operations pursuant to this agreement.

City of Hope Sponsored Research Agreement - Manufacturing

On January 3, 2018, Mustang entered into a Sponsored Research Agreement (“SRA”) with COH to optimize and develop CAR T cell processing procedures. Pursuant to the SRA, Mustang funded continued research in the amount of $0.9 million for the program, with an initial term of two (2) years. The SRA expired in January 2020. For the years ended December 31, 2020 and 2019, Mustang recorded approximately nil and $0.5 million, respectively, in research and development expenses in the Company’s Consolidated Statements of Operations.

CRISPR Sponsored Research Agreement with Beth Israel Deaconess Medical Center, Inc.

On November 28, 2017, Mustang entered into a Sponsored Research Agreement with Beth Israel Deaconess Medical Center Inc. (“BIDMC”) to perform research relating to gene editing, via the use of CRISPR/Cas9, to be used in enhancing the efficacy of CAR T cell therapies for solid tumor indications and to generate universal off the shelf CAR T cell therapies for both liquid and solid tumor indications. Mustang agreed to fund approximately $0.8 million over a three-year period. Mustang recorded nil and $0.1 million in 2020 and 2019, respectively, related to this agreement in research and development expenses in the Company’s Consolidated Statements of Operations.  In January 2019, Mustang terminated the SRA with BIDMC due to the departure of key personnel from BIDMC.

CD20 (MB-106) Clinical Trial Agreement with Fred Hutch

On July 3, 2017, in conjunction with the CD20 Technology License from Fred Hutch, Mustang entered into an investigator-initiated clinical trial agreement (“CD20 CTA”) to provide partial funding for a Phase 1/2 clinical trial at Fred Hutch evaluating the safety and efficacy of the CD20 Technology in patients with relapsed or refractory B-cell non-Hodgkin lymphomas. In connection with the CD20 CTA, Mustang agreed to fund up to $5.3 million of costs associated with the clinical trial, which commenced during the fourth quarter of 2017. In November 2020, the CD20 CTA was amended to include additional funding of approximately $0.8 million for the treatment of five patients with chronic lymphocytic leukemia. For the years ended December 31, 2020 and 2019 Mustang recorded $1.8 million and $0.6 million of expense, respectively, related to this agreement in research and development expenses in the Company’s Consolidated Statements of Operations.

CD20 (MB-106) Sponsored Research Agreement – Manufacturing with Fred Hutch

On March 17, 2018, Mustang entered into a Sponsored Research Agreement (“SRA”) with Fred Hutch related to developing and optimizing processes and systems associated with CD20 cell processing. Pursuant to the SRA, Mustang funded research in the amount of $0.6 million during the term of the SRA, which expired in March 2019. For the years ended December 31, 2020 and 2019, Mustang recorded expense of nil and $0.2 million, respectively, in research and development expenses in the Statements of Operations pursuant to the terms of this agreement.

XSCID (MB-107) Data Transfer Agreement with St. Jude

In June 2020, Mustang entered into a Data Transfer Agreement with St. Jude under which Mustang will reimburse St. Jude for costs associated with St. Jude’s clinical trial for the treatment of infants with XSCID.  Pursuant to the terms of this agreement and for the year ended December 31, 2020, Mustang paid an upfront fee of $1.1 million, which was recorded in research and development expenses in the Company’s Consolidated Statement of Operations.  Mustang will continue to reimburse St. Jude for costs incurred in connection with this trial.

MB-107 (XSCID) Non-Interventional Services Agreement with Children’s CGMP

In December 2019, Mustang entered into a Non-Interventional Services Agreement with Children's CGMP, LLC ("Children’s"), an affiliate of St. Jude Children's Research Hospital, pursuant to which Children’s provides lentiviral vector for non-clinical XSCID research purposes, as well as related advisory services. Mustang agreed to fund approximately $0.8 million upon execution of the agreement, which was recorded in research and development expenses for the year ended December 31, 2019 in the Company's Consolidated Statement of Operations.

Oncogenuity

Columbia Sponsored Research Agreement

Pursuant to the terms of the Columbia License, Oncogenuity will make semi-annual research payments to Columbia over a five year period ending in November 2024; such payments not to exceed $4.8 million. For the year ended December 31, 2020, Oncogenuity recorded expense of $0.5 million in research and development in the Company’s Consolidated Statements of Operations. No expense was recorded in 2019.

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University of Oxford Sponsorship Agreement

On December 16, 2020 Oncogenuity entered into an agreement with The Chancellor Masters and Scholars of the University of Oxford (“Oxford”). Under the terms of the agreement Oxford will engage in preclinical development of antisense oligonulcleotides as a therapy in certain indications.  In connection with the agreement Oncogenuity agreed to fund research for approximately 18 months for up to of $0.6 million (£0.4 million). Oncogenuity made an up-front payment of $0.1 million (£0.1 million) in January 2021.

Tamid

On November 30, 2017, in connection with its three separate license agreements with UNC, Tamid entered into a Sponsored Research Agreement with UNC (“UNC SRA”) for certain continued research and development activities related to Nanodysferlin for treatment of Dysferlinopathy, and AAV-HLA-G for corneal transplant rejection. Total amount to be funded by Tamid under the UNC SRA is $2.3 million over a term of three years. Pursuant to the terms of the UNC SRA, Tamid paid $0.8 million which was due upon execution. For the years ended December 31, 2020 and 2019, Tamid recorded expense of nil and nil respectively in connection with the UNC SRA. The expense was recorded in research and development expenses in the Company’s Consolidated Statements of Operations. Effective December 2019, Tamid returned the license to UNC and ceased to incur costs associated with the development of products under this license.