This section highlights FDA-related milestones and regulatory updates for drugs developed by Moleculin Biotech (MBRX).
Over the past two years, Moleculin Biotech has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
Annamycin, Ara-C, MB-106, and WP1066. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Annamycin - FDA Regulatory Timeline and Events
Annamycin is a drug developed by Moleculin Biotech for the following indication: Soft tissue sarcoma (STS) lung metastases.
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:
- Annamycin
- Announced Date:
- June 18, 2025
- Indication:
- Soft tissue sarcoma (STS) lung metastases
Announcement
Moleculin Biotech, Inc announced it has received a written response from the Office of Oncologic Diseases - Pediatric Oncology, of the U.S. Food and Drug Administration (FDA) regarding the Company's Initial Pediatric Study Plan (iPSP), which was submitted after a June 2024 end-of-phase 1/2 meeting.
AI Summary
Moleculin Biotech, Inc. announced that it has received a written response from the FDA’s Office of Oncologic Diseases – Pediatric Oncology regarding its Initial Pediatric Study Plan (iPSP), which was submitted after a June 2024 end-of-phase 1/2 meeting. The FDA agreed to a single pediatric study evaluating Annamycin, in combination with Cytarabine, as a second line therapy for pediatric patients with relapsed/refractory acute myeloid leukemia. Importantly, the FDA recommended including patients as young as 6 months old, which is younger than Moleculin had initially proposed. The agency clarified that similar drug exposure and safety profiles compared to adult data could allow for efficacy extrapolation. Moleculin is now updating its iPSP to incorporate this feedback and expects to submit the revised plan later this quarter, with plans to begin the pediatric trial in the second half of 2027.
Read Announcement- Drug:
- Annamycin
- Announced Date:
- June 18, 2025
- Estimated Event Date Range:
- July 1, 2025 - December 31, 2025
- Target Action Date:
- 2025-H2
- Indication:
- Soft tissue sarcoma (STS) lung metastases
Announcement
Moleculin Biotech, Inc announced that Enrollment and dosing are ongoing in pivotal, adaptive design Phase 3 MIRACLE trial of Annamycin in adult patients with R/R AML; Initial data readout is on track for the second half of 2025
AI Summary
Moleculin Biotech, Inc. has announced progress in its pivotal Phase 3 MIRACLE trial for Annamycin. This adaptive design study focuses on treating adult patients with relapsed or refractory acute myeloid leukemia (R/R AML). Enrollment and dosing of patients in the trial are currently ongoing across multiple international sites, including locations in the United States, Europe, and the Middle East.
The company expects the initial data readout from this trial to be available in the second half of 2025. This data will help assess the treatment’s safety and effectiveness in managing R/R AML, potentially paving the way for advancements in therapy options for this hard-to-treat form of cancer.
Read Announcement- Drug:
- Annamycin
- Announced Date:
- April 29, 2025
- Indication:
- Soft tissue sarcoma (STS) lung metastases
Announcement
Moleculin Biotech, Inc., announced that an abstract and poster presentation regarding the Company's next-generation anthracycline, Annamycin, was presented at the American Association for Cancer Research (AACR) Annual Meeting 2025, on April 28, 2025, at the McCormick Place Convention Center in Chicago, IL.
AI Summary
Moleculin Biotech, Inc. announced that it presented an abstract and poster on its next-generation anthracycline, Annamycin, at the AACR Annual Meeting 2025. The presentation took place on April 28, 2025, at the McCormick Place Convention Center in Chicago, IL. The research highlighted Annamycin’s potential to work synergistically with a variety of FDA-approved anticancer drugs. Initial studies, carried out both in laboratory (in vitro) and animal (in vivo) models, suggest that combining Annamycin with standard anticancer therapies might significantly enhance treatment outcomes. These findings point to a possibility for broader clinical applications, potentially extending the use of Annamycin in treating not only blood cancers but also solid tumors such as sarcoma and pancreatic cancer. The Company's work at the AACR Annual Meeting reinforces its commitment to developing innovative cancer therapies and exploring effective drug combinations for challenging cancers.
Read Announcement- Drug:
- Annamycin
- Announced Date:
- January 27, 2025
- Indication:
- Soft tissue sarcoma (STS) lung metastases
Announcement
Moleculin Biotech, Inc., announced that Walter Klemp, Chairman and Chief Executive Officer of Moleculin participated in a Virtual Investor "What This Means" segment.
AI Summary
Moleculin Biotech, Inc. announced that its Chairman and CEO, Walter Klemp, recently took part in a Virtual Investor “What This Means” segment. In this online session, Klemp provided an update on the company's latest progress, focusing on the Phase 3 pivotal “MIRACLE” trial. This trial tests Annamycin in combination with Cytarabine to treat patients with acute myeloid leukemia (AML) who have not responded to or relapsed after initial therapy.
Klemp discussed how the global trial, which is being conducted across the US, Europe, and the Middle East, marks an important step forward for the company’s drug development efforts. He also explained what the recent advancements in the AML field could mean for Moleculin’s ongoing program with Annamycin, highlighting the significance of the trial’s progress for patients in need of new treatment options.
Read Announcement- Drug:
- Annamycin
- Announced Date:
- December 11, 2024
- Indication:
- Soft tissue sarcoma (STS) lung metastases
Announcement
Moleculin Biotech, Inc. announced the online publication of its abstract titled, "Annamycin, a non-cardiotoxic anthracycline, demonstrates unique organotropism and activity against Ara-C and Venetoclax resistant AML," as part of the ASH Annual Meeting held December 7-10, 2024, in San Diego, CA.
AI Summary
Moleculin Biotech, Inc. recently announced the online publication of its abstract titled "Annamycin, a non-cardiotoxic anthracycline, demonstrates unique organotropism and activity against Ara-C and Venetoclax resistant AML" as part of the ASH Annual Meeting held December 7–10, 2024, in San Diego, CA. The study focuses on preclinical data showing that Annamycin can effectively target acute myeloid leukemia (AML) cells that have become resistant to standard treatments such as Cytarabine (Ara-C) and Venetoclax. Researchers highlighted that Annamycin not only works synergistically with Ara-C but also avoids the cardiotoxic side effects common to other anthracyclines, making it a promising option for patients with relapsed or refractory AML. These encouraging results support ongoing clinical evaluations and further development of Annamycin as a potential treatment for hard-to-treat leukemia patients.
Read Announcement- Drug:
- Annamycin
- Announced Date:
- September 23, 2024
- Indication:
- Soft tissue sarcoma (STS) lung metastases
Announcement
Moleculin Biotech, Inc., announced the recent presentation of positive in vivo efficacy data of Annamycin in orthotopic and experimental lung metastatic models of sarcoma.
AI Summary
Moleculin Biotech, Inc. recently showcased promising results for its next-generation anthracycline, Annamycin, at the IASLC 2024 World Conference on Lung Cancer. The new data highlights that Annamycin significantly inhibits tumor growth and extends survival in orthotopic lung cancer models, as well as in experimental lung metastatic models of sarcoma. When compared to the commonly used anthracycline doxorubicin, Annamycin achieved 10- to 30-fold higher concentration levels in lung tissues, suggesting it has superior organotropic properties. In addition, Annamycin was confirmed to be 100% non-cardiotoxic, which is a critical benefit given the heart risks associated with other similar drugs. These encouraging results strengthen the potential of Annamycin as a treatment option for both primary and metastatic lung cancers, supporting further clinical evaluation in hard-to-treat cancers.
Read Announcement- Drug:
- Annamycin
- Announced Date:
- August 1, 2024
- Indication:
- Soft tissue sarcoma (STS) lung metastases
Announcement
Moleculin Biotech, Inc. announced the positive discussion in and outcome of its End of Phase 1B/2 (EOP1B/2) meeting with the US Food and Drug Administration (FDA) supporting the advancement of 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") to a Phase 3 pivotal trial for the treatment of AML patients who are refractory to or relapsed after induction therapy (R/R AML).,
AI Summary
Moleculin Biotech announced a positive outcome from its End of Phase 1B/2 meeting with the FDA. In this meeting, the agency supported moving forward with a pivotal Phase 3 trial for Annamycin when used together with Cytarabine (often called Ara-C, and in combination, “AnnAraC”). The trial, named MIRACLE, will focus on patients with relapsed or refractory acute myeloid leukemia (R/R AML) who did not respond well to initial treatment.
The FDA’s feedback included using an adaptive design that relies on achieving a complete remission by day 30 as the primary endpoint. This approach aims to support a potential accelerated approval. Additionally, the trial will be global, including sites in the United States, and will allow dosing above the currently approved lifetime maximum for anthracyclines. Moleculin is optimistic that the trial will generate key data needed for regulatory approval.
Read Announcement- Drug:
- Annamycin
- Announced Date:
- July 10, 2024
- Indication:
- Soft tissue sarcoma (STS) lung metastases
Announcement
Moleculin Biotech, Inc. announced the completion of its End of Phase 2 (EOP2) meeting with the U.S. Food and Drug Administration (FDA) for its Phase 1B/2 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 AML as both first line therapy and for subjects who are refractory to or relapsed after induction therapy (MB-106). ,
AI Summary
Moleculin Biotech, Inc. announced that it has completed its End of Phase 2 (EOP2) meeting with the U.S. Food and Drug Administration (FDA) for its Phase 1B/2 clinical trial. The trial evaluates the combination of Annamycin and Cytarabine—referred to as AnnAraC—for treating patients with acute myeloid leukemia (AML). The study targets both first line therapy patients as well as those who did not respond or relapsed after induction therapy under the MB-106 program.
The meeting with the FDA focused on evaluating the safety and efficacy data from the ongoing trial and outlining next steps for clinical development. The company expects to receive the official FDA meeting minutes by the end of Q3 2024, with further details to be reported before the end of August. Moleculin expressed gratitude for the productive discussions and is optimistic about advancing its AML treatment program.
Read Announcement- Drug:
- Annamycin
- Announced Date:
- May 7, 2024
- Indication:
- Soft tissue sarcoma (STS) lung metastases
Announcement
Moleculin Biotech, Inc., announced the presentation of positive interim data for Annamycin from its ongoing acute myeloid leukemia (AML) clinical development program. As previously announced, the Company will host a virtual AML Clinical Day, today, May 7, 2024, at 11:00 AM ET (details below) to discuss these results.
AI Summary
Moleculin Biotech, Inc. announced that it has achieved positive interim data for its drug candidate Annamycin from its ongoing acute myeloid leukemia (AML) clinical development program. This promising result could represent an important step forward in the treatment of AML, offering new hope in targeting this challenging cancer.
To provide further insights into these encouraging results, the company will host a virtual AML Clinical Day on May 7, 2024, at 11:00 AM ET. During this event, Moleculin Biotech plans to discuss the details of the interim data, outline the clinical implications, and share perspectives on the next steps in advancing Annamycin for AML patients.
Read Announcement
Ara-C - FDA Regulatory Timeline and Events
Ara-C is a drug developed by Moleculin Biotech for the following indication: For the treatment of AML patients who are refractory to or relapsed after induction therapy (R/R AML).
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:
- Ara-C
- Announced Date:
- July 9, 2025
- Indication:
- For the treatment of AML patients who are refractory to or relapsed after induction therapy (R/R AML).
Announcement
Moleculin Biotech, Inc., announced that the Regulation Agency for Medical and Pharmaceutical Activities (RAMPA) in the country of Georgia has approved its Clinical Trial Application (CTA) to conduct its pivotal Phase 2B/3, multi-center, randomized, double-blind, placebo-controlled, adaptive design study of 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 adult patients with acute myeloid leukemia (AML) who are refractory to or relapsed (R/R) after induction therapy (R/R AML).
AI Summary
Moleculin Biotech, Inc. announced that the Regulation Agency for Medical and Pharmaceutical Activities (RAMPA) in Georgia has approved its Clinical Trial Application (CTA) for its pivotal Phase 2B/3 trial. This study will test Annamycin in combination with cytarabine (known as AnnAraC) to treat adult patients with acute myeloid leukemia (AML) who have not responded to or have relapsed after initial therapy. The trial is designed as a multi-center, randomized, double-blind, placebo-controlled study with an adaptive design to assess the drug’s safety and effectiveness. Patient dosing in Georgia is expected to start by the end of August, and additional sites in Europe and the US are scheduled to join later, with over 30 sites planned for Part A. The study supports the search for better treatment options for R/R AML patients in need of new therapies.
Read Announcement- Drug:
- Ara-C
- Announced Date:
- May 12, 2025
- Indication:
- For the treatment of AML patients who are refractory to or relapsed after induction therapy (R/R AML).
Announcement
Moleculin Biotech, Inc., announced that the European Medicines Agency (EMA) has approved its Clinical Trial Application (CTA) to conduct its pivotal Phase 2B/3, multi-center, randomized, double-blind, placebo-controlled, adaptive design study of 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").
AI Summary
Moleculin Biotech, Inc. announced that the European Medicines Agency (EMA) has approved its Clinical Trial Application (CTA) to run a pivotal Phase 2B/3 study using its combination treatment, AnnAraC, which pairs Annamycin with cytarabine (Ara‐C). This multi-center, randomized, double-blind, placebo-controlled trial is designed with an adaptive structure, aimed at treating adult patients with relapsed or refractory acute myeloid leukemia (R/R AML). The EMA approval covers nine European Union countries—Belgium, Czechia, France, Germany, Italy, Lithuania, Poland, Romania, and Spain. This milestone approval enhances Moleculin’s recruitment and expands its global study efforts. The study is expected to help provide a much-needed treatment option for R/R AML patients, especially for those who have not responded to existing therapies, including recent venetoclax regimen failures.
Read Announcement - Drug:
- Ara-C
- Announced Date:
- April 1, 2025
- Indication:
- For the treatment of AML patients who are refractory to or relapsed after induction therapy (R/R AML).
Announcement
Moleculin Biotech, Inc., announced the first patient has been dosed in its Phase 3 pivotal 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 AML patients who are refractory to or relapsed after induction therapy (R/R AML).
AI Summary
Moleculin Biotech, Inc. announced that the first patient has been dosed in its Phase 3 pivotal trial for relapsed or refractory acute myeloid leukemia (AML). The trial is evaluating Annamycin in combination with Cytarabine—also known as Ara-C—a regimen referred to as AnnAraC. This global study, called the MIRACLE trial, is taking place in the United States, Europe, and the Middle East, and it uses an adaptive trial design. Early unblinding of safety and efficacy data is expected from the initial 45 subjects in the second half of 2025, with further data planned for release in the first half of 2026. Moleculin’s dosing design, which includes different dose levels of Annamycin alongside a standard high dose of Cytarabine, aims to provide a promising new treatment option for AML patients who do not respond to or relapse after induction therapy.
Read Announcement
MB-106 - FDA Regulatory Timeline and Events
MB-106 is a drug developed by Moleculin Biotech 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
WP1066 - FDA Regulatory Timeline and Events
WP1066 is a drug developed by Moleculin Biotech for the following indication: For the Treatment of Glioblastoma (NU 21C06).
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:
- WP1066
- Announced Date:
- September 9, 2024
- Indication:
- For the Treatment of Glioblastoma (NU 21C06)
Announcement
Moleculin Biotech, Inc., announced the enrollment and treatment of patients in an Investigator-initiated Phase 2 study evaluating WP1066 in combination with radiation therapy for the treatment of adults with glioblastoma (NU 21C06).
AI Summary
Moleculin Biotech, Inc. announced that patients with glioblastoma are now being enrolled and treated in an Investigator-initiated Phase 2 study at Northwestern University. This trial is testing WP1066 in combination with radiation therapy to treat adults with newly diagnosed glioblastoma, a highly aggressive brain tumor. The study, known as NU 21C06, is supported by funding from the NIH and BrainUp®, a non-profit focused on brain cancer awareness.
The trial aims to improve progression-free survival and includes tumor microenvironment analyses. Encouraging preclinical data showed that combining WP1066 with radiation not only increased survival rates in animal models but also triggered anti-tumor immune responses. Researchers are hopeful that this approach could address the unmet needs in glioblastoma treatment by providing a novel therapeutic option for patients with this challenging disease.
Read Announcement- Drug:
- WP1066
- Announced Date:
- May 15, 2024
- Indication:
- Ependymoma
Announcement
Moleculin Biotech, Inc., announced the commencement of an Investigator-initiated Phase 2 study evaluating WP1066 in combination with radiation therapy for the treatment of adults with glioblastoma (NU 21C06) in cooperation with the Company.
AI Summary
Moleculin Biotech, Inc. has announced the start of an important clinical study that could change the treatment options for adults with glioblastoma, a severe form of brain cancer. The company is initiating a Phase 2 trial—named NU 21C06—that will combine its drug candidate WP1066 with radiation therapy. This study is carried out through investigator initiative in cooperation with the company, aiming to explore and evaluate whether this combination can offer improved outcomes compared to current treatment methods.
The trial’s main focus is to determine the safety and effectiveness of using WP1066 alongside radiation therapy. Researchers are hopeful that this new approach could provide benefits to patients by enhancing the overall treatment strategy against glioblastoma. The results from this study are eagerly anticipated as they could pave the way for future advancements in cancer therapy.
Read Announcement
