On June 18, 2025 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a late-stage pharmaceutical company with a broad portfolio of drug candidates targeting hard-to-treat cancers and viral infections, reported 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 (Press release, Moleculin, JUN 18, 2025, View Source [SID1234653977]). The FDA has agreed to a single pediatric approval study in which Annamycin (also known as naxtarubicin) in combination with Cytarabine (also known as "Ara-C" and for which the combination of Annamycin and Ara-C is referred to as "AnnAraC") will be evaluated as second line therapy in pediatric patients with relapsed/refractory acute myeloid leukemia (R/R AML), a form of cancer.

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"We continue to be encouraged by the potential of Annamycin to change the game when it comes to the most prevalent cancer therapies in use today. This is particularly important in pediatric oncology, where about 60% of children with cancer are treated with anthracyclines that present a high risk of causing heart damage. An independent expert’s review of study data has shown no cardiotoxicity to date in 84 adult patients treated with Annamycin, which is why we’re excited about moving forward in young children with cancer. Receiving this written feedback from FDA is a crucial step in the development of Annamycin for pediatric use and provides us with helpful insight as we continue to prepare for the launch of a pediatric trial, planned for the second half of 2027," said Walter Klemp, Chairman and CEO of Moleculin. "As our team works to address and implement FDA’s feedback on our iPSP, we remain focused on the successful execution of our ongoing MIRACLE trial of AnnAraC in adult patients with R/R AML and look forward to initial data from that trial later this year."

As part of the iPSP, Moleculin proposed a single-arm study evaluating pharmacokinetics (PK), efficacy, and safety of AnnAraC in patients between 2 and 16 years of age. The FDA response calls for including patients from 6 months of age and older, with no minimum number of patients between 6 months and 2 years old. FDA also clarified that drug concentration exposure and safety profile that are comparable would allow extrapolation from efficacy in adults. Importantly, FDA said Moleculin would be able to start the pediatric trial before having the full two years of follow-up data from the adult trial.

Moleculin is in the process of updating its iPSP to incorporate FDA’s recommendations and intends to submit the revised plan to FDA later this quarter. Moleculin expects to initiate pediatric clinical study in the second half of 2027.

The Company is currently evaluating AnnAraC for the treatment of adult patients with R/R AML in the pivotal, adaptive Phase 3 MIRACLE trial (MB-108). This "MIRACLE" trial (derived from Moleculin R/R AML AnnAraC Clinical Evaluation) is a global trial, including sites in the US, Europe and the Middle East. Patient dosing has commenced, and the initial data readout is on track for the second half of 2025. For more information about the MIRACLE trial, visit clinicaltrials.gov and reference identifier NCT06788756

FDA has granted Annamycin Fast Track Status and Orphan Drug Designation for treating relapsed or refractory acute myeloid leukemia, and an additional Orphan Drug Designation for treating soft tissue sarcoma. The European Medicines Agency (EMA) has also granted Orphan Drug Designation for the treatment of relapsed or refractory acute myeloid leukemia.