This section highlights FDA-related milestones and regulatory updates for drugs developed by Mustang Bio (MBIO).
Over the past two years, Mustang Bio has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
MB-101, MB-106, MB-107, and MB-108. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
MB-101 - FDA Regulatory Timeline and Events
MB-101 is a drug developed by Mustang Bio for the following indication: Leptomeningeal brain tumors.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- MB-101
- Announced Date:
- July 7, 2025
- Indication:
- Leptomeningeal brain tumors
Announcement
Mustang Bio, Inc announced that the U.S. Food and Drug Administration ("FDA") has granted Orphan Drug Designation to Mustang for MB-101 (IL13Ra2-targeted CAR T-cells) for the treatment of recurrent diffuse and anaplastic astrocytoma (astrocytomas) and glioblastoma (GBM).
AI Summary
Mustang Bio, Inc. announced that the U.S. Food and Drug Administration has granted Orphan Drug Designation to its MB-101 therapy. MB-101 is an IL13Ra2-targeted CAR T-cell treatment aimed at recurrent diffuse and anaplastic astrocytomas as well as glioblastoma (GBM). This designation is given for drugs intended to treat rare diseases affecting fewer than 200,000 people in the United States. It offers valuable benefits, including tax credits for clinical trials, waiver of prescription drug user fees, and seven years of market exclusivity if the treatment is approved.
The grant validates Mustang Bio’s scientific approach and supports their ongoing efforts to develop MB-101 as a potential treatment for difficult-to-treat brain tumors. The company is keen on advancing this therapy, which may work even better when paired with their MB-108 oncolytic virus for patients battling malignant glioma and other high-grade astrocytomas.
Read Announcement- Drug:
- MB-101
- Announced Date:
- July 1, 2025
- Indication:
- Leptomeningeal brain tumors
Announcement
Mustang Bio Receives FDA Orphan Drug Designation For MB-101 Treatment Of Malignant Glioma
Read Announcement
MB-106 - FDA Regulatory Timeline and Events
MB-106 is a drug developed by Mustang Bio for the following indication: Relapsed or Refractory B-cell non-Hodgkin lymphomas (NHL) and Chronic Lymphocytic Leukemia (CLL).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- MB-106
- Announced Date:
- June 17, 2024
- Indication:
- Relapsed or Refractory B-cell non-Hodgkin lymphomas (NHL) and Chronic Lymphocytic Leukemia (CLL)
Announcement
Mustang Bio, Inc. announced that updated data from the ongoing Phase 1/2 clinical trial of MB-106, a CD20-targeted, autologous CAR T-cell therapy, show a favorable safety and efficacy profile in patients with Waldenstrom macroglobulinemia ("WM"), a rare form of blood cancer.
AI Summary
Mustang Bio, Inc. announced encouraging new data from its ongoing Phase 1/2 clinical trial of MB-106, a CD20-targeted, autologous CAR T-cell therapy. The treatment showed a favorable safety and efficacy profile in patients with Waldenstrom macroglobulinemia (WM), a rare blood cancer. In this small study, 90% of the patients responded to treatment, with three achieving complete responses, including one patient who has remained in complete remission for 31 months. The patients, all of whom had been heavily pretreated and refractory to BTK inhibitors, exhibited durable responses. Only one patient required additional anti-WM treatment after MB-106, and the therapy was feasible for outpatient administration. These promising findings highlight MB-106’s potential as a treatment option for WM, a field where no FDA-approved CAR T-cell treatments currently exist.
Read Announcement- Drug:
- MB-106
- Announced Date:
- June 14, 2024
- Indication:
- Relapsed or Refractory B-cell non-Hodgkin lymphomas (NHL) and Chronic Lymphocytic Leukemia (CLL)
Announcement
Moleculin Biotech, Inc., reported additional efficacy findings from the Company's ongoing Phase 1B/2 (MB-106) clinical trial evaluating Annamycin in combination with Cytarabine (also known as "Ara-C" and for which the combination of Annamycin and Ara-C is referred to as AnnAraC) for the treatment of subjects with acute myeloid leukemia (AML).
AI Summary
Moleculin Biotech, Inc. has released new efficacy findings from its ongoing Phase 1B/2 (MB-106) trial testing Annamycin combined with Cytarabine (AnnAraC) for treating acute myeloid leukemia (AML). In the study, 22 subjects were enrolled and 20 completed efficacy evaluations. Of these patients, 45% achieved a composite complete remission (CRc), including both complete remission (CR) and CR with incomplete blood count recovery (CRi). Notably, 89% of the CRc group had poor prognosis cytogenetics or mutations. In second line treatment, AnnAraC achieved an estimated median overall survival of about 6 months, with a similarly rising median durability of response for CRc patients. Importantly, the trial demonstrated no cardiotoxicity, and the combination was generally well tolerated, supporting its potential as a promising treatment option for AML patients.
Read Announcement- Drug:
- MB-106
- Announced Date:
- May 16, 2024
- Indication:
- Relapsed or Refractory B-cell non-Hodgkin lymphomas (NHL) and Chronic Lymphocytic Leukemia (CLL)
Announcement
Moleculin Biotech, Inc., announced its abstract has been accepted for poster presentation at the EHA2024 Hybrid Congress being held June 13-16, 2024 in Madrid, Spain and virtually.
AI Summary
Moleculin Biotech, Inc. recently announced that its abstract has been accepted for a poster presentation at the EHA2024 Hybrid Congress. The event will take place from June 13-16, 2024 in Madrid, Spain, and will also be accessible virtually. The poster, titled “LIPOSOMAL ANNAMYCIN (L-ANN) IN COMBINATION WITH CYTARABINE FOR TREATMENT OF PATIENTS WITH ACUTE MYELOID LEUKAEMIA (AML) REFACTORY TO OR RELAPSED (R/R) AFTER INDUCTION THERAPY (MB-106 STUDY),” will be presented by Dr. Wolfram C. M. Dempke, who serves as Moleculin’s European Chief Medical Officer.
This presentation highlights the Company’s efforts in developing new treatment approaches for challenging blood cancers. Attendees at the congress will have the opportunity to learn about Moleculin’s latest research, which may pave the way for improved therapies for patients with acute myeloid leukemia.
Read Announcement
MB-107 - FDA Regulatory Timeline and Events
MB-107 is a drug developed by Mustang Bio for the following indication: Newly diagnosed infants with X-linked severe combined immunodeficiency (XSCID) or bubble boy disease.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- MB-107
- Announced Date:
- June 5, 2025
- Indication:
- Newly diagnosed infants with X-linked severe combined immunodeficiency (XSCID) or bubble boy disease
Announcement
Moleculin Biotech, Inc., announced the release of its Soft Tissue Sarcoma (STS) Lung Mets KOL Webcast discussing the final data from its U.S. Phase 1B/2 clinical trial evaluating Annamycin as monotherapy for the treatment of soft tissue sarcoma lung metastases (MB-107).
AI Summary
Moleculin Biotech, Inc. recently released its Soft Tissue Sarcoma (STS) Lung Mets KOL Webcast featuring the final data from its U.S. Phase 1B/2 clinical trial. The webcast highlights the evaluation of Annamycin as a monotherapy for treating soft tissue sarcoma lung metastases (MB-107). During the presentation, key speakers including CEO Walter Klemp and Senior Chief Medical Officer Dr. Paul Waymack, along with several noted experts, discussed the promising results from the trial. The study focuses on the potential effectiveness of Annamycin, a next-generation anthracycline designed to avoid common issues such as multidrug resistance and cardiotoxicity, in managing a difficult-to-treat condition. The webcast is now available on the Moleculin website for a limited time, providing investors and stakeholders with detailed insights on the trial outcomes and the drug's future role in cancer treatment.
Read Announcement- Drug:
- MB-107
- Announced Date:
- June 4, 2025
- Indication:
- Newly diagnosed infants with X-linked severe combined immunodeficiency (XSCID) or bubble boy disease
Announcement
Moleculin Biotech, Inc today reported positive topline efficacy results from its completed U.S. Phase 1B/2 clinical trial evaluating Annamycin for the treatment of soft tissue sarcoma lung metastases ("STS lung mets") (MB-107).
AI Summary
Moleculin Biotech, Inc. today announced encouraging topline efficacy results from its U.S. Phase 1B/2 trial (MB-107) for Annamycin in treating soft tissue sarcoma lung metastases. The trial, which used a single-agent approach, established both the Maximum Tolerable Dose and the Recommended Phase 2 Dose while also evaluating safety and effectiveness. In the study, the clinical benefit rate was 59.4% among participants, with many patients showing stable disease and one achieving a partial response. Notably, patients who responded after two cycles experienced improvements in overall survival and progression-free survival, with some achieving approximately 4 months of progression-free survival and 20 months of overall survival. These results suggest that Annamycin could provide a valuable new treatment option, especially for patients who have undergone multiple prior chemotherapy regimens.
Read Announcement
MB-108 - FDA Regulatory Timeline and Events
MB-108 is a drug developed by Mustang Bio for the following indication: To Treat Malignant Glioma.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- MB-108
- Announced Date:
- February 11, 2025
- Estimated Event Date Range:
- January 1, 2025 - March 31, 2025
- Target Action Date:
- Q1 - 2025
- Indication:
- To Treat Malignant Glioma
Announcement
Moleculin Biotech, Inc. announced that Company remains on track for first subject treated in pivotal, adaptive Phase 3 clinical trial (the "MIRACLE" trial) in the first quarter of 2025,
AI Summary
Moleculin Biotech, Inc. announced that it remains on track to treat the first patient in its pivotal, adaptive Phase 3 clinical trial—known as the “MIRACLE” trial—in the first quarter of 2025. This study is designed to evaluate the combination of Annamycin and Cytarabine for treating relapsed or refractory acute myeloid leukemia, a form of cancer where patients do not respond to initial treatment.
The global trial, which includes clinical sites in the United States, Europe, and the Middle East, is set to enroll its first patient on schedule. The study protocol allows for an early unblinding of efficacy and safety data after 45 subjects, helping to guide further development steps. Moleculin’s commitment to swift enrollment and careful trial management supports its goals for a timely and effective study execution.
Read Announcement- Drug:
- MB-108
- Announced Date:
- February 11, 2025
- Indication:
- To Treat Malignant Glioma
Announcement
Moleculin Biotech, Inc., announced it has received first country regulatory approval in Europe to begin recruiting for its Phase 3 pivotal trial protocol evaluating Annamycin in combination with Cytarabine (also known as "Ara-C" and for which the combination of Annamycin and Ara-C is referred to as "AnnAraC") for the treatment of AML patients who are refractory to or relapsed after induction therapy (R/R AML) (MB-108).
AI Summary
Moleculin Biotech, Inc. announced it has received its first regulatory approval in Europe to start recruiting for a global Phase 3 pivotal trial. This trial will evaluate Annamycin in combination with Cytarabine—known together as AnnAraC—for treating AML patients who did not respond to induction therapy or who have relapsed (R/R AML). The study, called the MIRACLE trial, will soon include sites in the US, Europe, and the Middle East with the first subject expected to be treated in the first quarter of 2025. The trial design features an adaptive approach that allows for early unblinding of preliminary efficacy and safety data after enrolling 45 patients. This important milestone signifies progress toward expanding treatment options for challenging cases of AML, as Moleculin continues to advance its research with a focus on operational execution and effective patient recruitment.
Read Announcement
