On May 14, 2025 MaaT Pharma (EURONEXT: MAAT – the "Company"), a clinical-stage biotechnology company and a leader in the development of Microbiome Ecosystem TherapiesTM (MET) dedicated to enhancing survival for patients with cancer through immune modulation, reported that updated data from its Early Access Program for 173 patients with aGvHD treated with MaaT013 have been selected for oral presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) Annual congress, taking place in Milan from June 12–15, 2025 (Press release, MaaT Pharma, MAY 14, 2025, View Source [SID1234653066]). The oral presentation of MaaT013 data at EHA (Free EHA Whitepaper)—Europe’s leading hematology conference—underlines the growing recognition of the drug’s clinical potential and the Company’s leadership in hemato-oncology using microbiome-based approach.

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These updated EAP results for 173 patients, to be presented at EHA (Free EHA Whitepaper) Congress, are consistent with the positive topline results of the Phase 3 trial announced in January 2025 and further confirm MaaT013’s high efficacy and favorable safety profile in treating patients with gastrointestinal aGvHD. There are currently no approved options for patients with GI-aGvHD who are refractory to steroids and either refractory or intolerant to ruxolitinib, despite the poor prognosis with one-year survival rates of 15% (Abedin et al., 2021).

MaaT Pharma has observed a 75% increase in physician demand with MaaT013 under the ongoing EAP in 2024 compared to 2023, across Europe and, more recently, in the United States. This steady demand for access to MaaT013 reflects its growing adoption by the medical community as a treatment option for patients with GI-aGvHD. To date, the Company has safely treated more than 300 patients with aGvHD across clinical trials and the Company’s EAP ongoing in both Europe and the U.S.

With upcoming regulatory milestones in Europe including a Marketing Authorization submission expected in June 2025, growing global physician interest, and continued clinical validation, MaaT013 has the potential to become the first approved third-line treatment for GI-aGvHD, significantly improving survival outcomes for approximately 3,000 third-line patients annually across the U.S., Canada, and Europe.

Details of the Oral Presentation:

Title: Pooled Fecal Allogeneic Microbiotherapy for Refractory Gastrointestinal Acute Graft-Versus-Host Disease: Results from the Early Access Program in Europe
Abstract number: S260
Presenting Author: Mohamad Mohty, Professor of Hematology and Head of the Hematology and Cellular Therapy Department at Saint-Antoine Hospital and Sorbonne University
Session title: s424 Stem cell transplantation – Session 2
Date & Time: 13/06/2025 (17:00 – 18:15 CEST) – Brown Hall 3
MaaT Pharma will also present its ongoing Phase 2b trial (PHOEBUS) design for MaaT033 developed as an adjunctive therapy to enhance overall survival in allo-HSCT. This international, multi-center trial (NCT05762211) is the largest randomized controlled study to date of a microbiome-based therapy in oncology, enrolling up to 387 patients across 60 sites. To date, the independent Data Safety Monitoring Board (DSMB) has conducted two safety reviews and one unblinded interim analysis, all of which concluded positively with the recommendation that the PHOEBUS trial proceed as planned.

On May 14, 2025 Arcellx, Inc. (NASDAQ: ACLX), a biotechnology company reimagining cell therapy through the development of innovative immunotherapies for patients with cancer and other incurable diseases, reported new positive data from its pivotal Phase 2 iMMagine-1 study of anitocabtagene autoleucel (anito-cel), in patients with relapsed or refractory multiple myeloma (RRMM) (Press release, Arcellx, MAY 14, 2025, View Source [SID1234653052]). These data will be presented during an oral presentation at the EHA (Free EHA Whitepaper)2025 Congress in Milan on June 14, 2025. Anito-cel is partnered with Kite, a Gilead Company.

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The Phase 2 iMMagine-1 data are from a May 1, 2025 data cutoff date, including all 117 patients with a median follow-up of 12.6 months and a minimum follow-up of four months after treatment with anito-cel. All patients received a single infusion of anito-cel (target dose of 115×106 CAR+ viable T cells). 101 of 117 patients (86%) were triple refractory, and 48 of 117 patients (41%) were penta refractory. Patients received a median of three prior lines of therapy, with 60 of 117 patients (51%) having received three prior lines.

Overall response rate (ORR) was 97% (114/117) with a complete response/stringent complete response (CR/sCR) rate of 68% (79/117) and a very good partial response or higher (≥VGPR) rate of 85% (100/117), per International Myeloma Working Group (IMWG) criteria as investigator-assessed. Of those evaluable for minimal residual disease (MRD) testing at the time of this data cut, 93.3% (70/75) achieved MRD negativity at a minimum of 10-5 sensitivity. Six-month progression-free survival (PFS) and overall survival (OS) rates were 91.9% and 96.6%, respectively, and 12-month PFS and OS rates were 78.8% and 95.2%, respectively. Median PFS and median OS have not been reached.

No delayed or non-immune effector cell-associated neurotoxicity syndrome (ICANS) neurotoxicities, including no Parkinsonism, no cranial nerve palsies, and no Guillain-Barré syndrome, and no immune-mediated enterocolitis have been observed to date with anito-cel. No additional treatment- or therapy-related deaths or Grade ≥3 cytokine release syndrome (CRS) or ICANS events have occurred since the previous data presentation in December 2024.

Conclusions

Preliminary results from the Phase 2 iMMagine-1 study demonstrate deep and durable responses with a predictable and manageable safety profile in a fourth-line or higher (4L+) RRMM population, including triple- and penta-class refractory disease. Notably, no delayed or non-ICANS neurotoxicities, including no Parkinsonism, no cranial nerve palsies, and no Guillain-Barré syndrome, and no immune-mediated enterocolitis have been observed with anito-cel to date.

"These clinical data from our registrational study continue to support our belief that anito-cel has the potential to address the needs of myeloma patients and the physicians who serve them," said Rami Elghandour, Arcellx’s Chairman and Chief Executive Officer. "There is no cure for multiple myeloma. We believe there remains an unmet medical need for CAR-T therapies that are efficacious, safe, and accessible. Anito-cel has the unique potential to address these needs thanks to our differentiated technology, our incredible and entrepreneurial team, the robust clinical data generated to date, and our strong partnership with Kite. Our 2026 commercial launch plans for anito-cel with our partners at Kite are well underway and we are excited for the opportunity to advance anito-cel in support of the myeloma community. We look forward to sharing these data with the clinical community at EHA (Free EHA Whitepaper) and are honored that the iMMagine-1 data will be presented during an oral presentation on Saturday, June 14."

EHA2025 Presentation Details

Phase 2 Registrational Study of Anitocabtagene Autoleucel for the Treatment of Patients with Relapsed and/or Refractory Multiple Myeloma: Preliminary Results from the iMMagine-1 Trial

Speaker: Gurbakhash Kaur, M.D., Assistant Professor of Internal Medicine, Mount Sinai Health System

Session Title: s431 Treatment of relapsed and/or refractory multiple myeloma (RRMM)

Session Date: June 14, 2025

Session Time: 17:00-18:15 CEST

Publication Number: S201

Presentation Title: S201 Phase 2 Registrational Study of Anitocabtagene Autoleucel for Relapsed and/or Refractory Multiple Myeloma (RRMM): Updated Results from iMMagine-1

About Multiple Myeloma

Multiple Myeloma (MM) is a type of hematological cancer in which diseased plasma cells proliferate and accumulate in the bone marrow, crowding out healthy blood cells and causing bone lesions, loss of bone density, and bone fractures. These abnormal plasma cells also produce excessive quantities of an abnormal immunoglobulin fragment, called a myeloma protein (M protein), causing kidney damage and impairing the patient’s immune function. MM is the third most common hematological malignancy in the United States and Europe, representing approximately 10% of all hematological cancer cases and 20% of deaths due to hematological malignancies. The median age of patients at diagnosis is 69 years with one-third of patients diagnosed at an age of at least 75 years. Because MM tends to afflict patients at an advanced stage of life, patients often have multiple co-morbidities and toxicities that can quickly escalate and become life-endangering.

About Anitocabtagene Autoleucel (anito-cel)

Anitocabtagene autoleucel (anito-cel, previously ddBCMA) is the first BCMA-directed CAR T-cell therapy to be investigated in multiple myeloma that utilizes Arcellx’s novel and compact binder known as the D-Domain. The small, stable D-Domain binder enables high CAR expression without tonic signaling and is designed to quickly release from the BCMA target. This combination may allow for the effective elimination of multiple myeloma cells without severe immunotoxicity. Anito-cel has been granted Fast Track, Orphan Drug, and Regenerative Medicine Advanced Therapy Designations by the U.S. Food and Drug Administration.

On May 14, 2025 Merck (NYSE: MRK), known as MSD outside of the United States and Canada, reported the U.S. Food and Drug Administration (FDA) has approved WELIREG (belzutifan), Merck’s oral hypoxia-inducible factor-2 alpha (HIF-2α) inhibitor, for the treatment of adult and pediatric patients 12 years and older with locally advanced, unresectable, or metastatic pheochromocytoma or paraganglioma (PPGL) (Press release, Merck & Co, MAY 14, 2025, View Source [SID1234653067]). Pheochromocytoma and paraganglioma are rare tumors that come from the same tissue, but pheochromocytoma form in the adrenal gland and paraganglioma form outside the adrenal gland. These tumors can be caused by certain genetic syndromes or mutations. The approval is based on data from the single-arm LITESPARK-015 clinical trial, where the primary endpoint was objective response rate (ORR).

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"PPGL, sometimes referred to as pheo para, is a rare condition affecting up to 2,000 people each year in the United States. Patients with these tumors, which arise from the adrenal glands and the extra-adrenal paraganglia, may require specialized care due to their complexity and rare nature, often posing significant challenges for both diagnosis and treatment," said Dr. Camilo Jimenez, professor, department of endocrine neoplasia and hormonal disorders, The University of Texas MD Anderson Cancer Center. "This approval, which is based on objective response rate data from the LITESPARK-015 trial, introduces belzutifan as the only approved and available non-surgical option for locally advanced, unresectable, or metastatic PPGL and could represent a change to the treatment paradigm for eligible patients."

"For patients with advanced PPGL, there has been a lack of approved systemic treatment options available to help manage their disease, underscoring the importance of this approval in the U.S.," said Dr. Marjorie Green, senior vice president and head of oncology, global clinical development, Merck Research Laboratories. "This approval marks the third indication for WELIREG in the U.S. and demonstrates our company’s commitment to providing innovative cancer therapies for patients in need, including those with rare diseases."

The WELIREG label contains a boxed warning that exposure to WELIREG during pregnancy can cause embryo-fetal harm. Verify pregnancy status prior to the initiation of WELIREG. Advise patients of these risks and the need for effective non-hormonal contraception. WELIREG can render some hormonal contraceptives ineffective. WELIREG can cause severe anemia that can require a blood transfusion. Monitor for anemia before initiation of and periodically throughout treatment with WELIREG. WELIREG can cause severe hypoxia that may require discontinuation, supplemental oxygen, or hospitalization. Monitor oxygen saturation before initiation of and periodically throughout treatment with WELIREG. For more information, see "Selected Safety Information" below.

Study design
LITESPARK-015 is an open-label, multicohort Phase 2 trial (ClinicalTrials.gov, NCT04924075) evaluating the efficacy and safety of WELIREG monotherapy. The study enrolled 72 patients in a single cohort (Cohort A1) who had measurable disease verified by blinded independent central review (BICR) per RECIST v1.1, documented histopathological diagnosis of PPGL, locally advanced or metastatic disease that was not amenable to surgery or curative treatment, and adequately controlled blood pressure (defined as BP <150/90 mm Hg, <135/85 mm Hg for adolescents) with no change in antihypertensive medications for patients with concomitant hypertension for at least two weeks prior to start of study treatment. Patients with carcinomatous meningitis were excluded. Patients received WELIREG at a dose of 120 mg once daily until disease progression or unacceptable toxicity.

The major efficacy outcome measure for the treatment of advanced PPGL was ORR measured by BICR using RECIST v1.1. Additional efficacy outcome measures included duration of response and time to response.

About pheochromocytoma and paraganglioma
Pheochromocytoma and paraganglioma (PPGL), sometimes referred to as pheo para, are rare tumors that can be caused by certain genetic syndromes or mutations. It is estimated that up to 2,000 new cases of PPGL are diagnosed each year in the U.S., and up to 52,800 new cases are diagnosed each year worldwide. Pheochromocytoma form in nerve tissue in the center of the adrenal gland, whereas paraganglioma form in nerve tissue near certain blood vessels and nerves outside the adrenal glands.

About WELIREG (belzutifan) 40 mg tablets, for oral use
Indications in the U.S.
Certain von Hippel-Lindau (VHL) disease-associated tumors
WELIREG is indicated for the treatment of adult patients with von Hippel-Lindau (VHL) disease who require therapy for associated renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas, or pancreatic neuroendocrine tumors (pNET), not requiring immediate surgery.

Advanced Renal Cell Carcinoma (RCC)
WELIREG is indicated for the treatment of adult patients with advanced renal cell carcinoma (RCC) with a clear cell component following a programmed death receptor-1 (PD-1) or programmed death ligand 1 (PD-L1) inhibitor and a vascular endothelial growth factor tyrosine kinase inhibitor (VEGF-TKI).

Pheochromocytoma or Paraganglioma (PPGL)
WELIREG is indicated for the treatment of adult and pediatric patients 12 years and older with locally advanced, unresectable, or metastatic pheochromocytoma or paraganglioma (PPGL).

Selected Safety Information for WELIREG
Warning: Embryo-Fetal Toxicity
Exposure to WELIREG during pregnancy can cause embryo-fetal harm. Verify pregnancy status prior to the initiation of WELIREG. Advise patients of these risks and the need for effective non-hormonal contraception as WELIREG can render some hormonal contraceptives ineffective.

Anemia
WELIREG can cause severe anemia that can require blood transfusion. Monitor for anemia before initiation of, and periodically throughout, treatment. Transfuse patients as clinically indicated. For patients with hemoglobin <8 g/dL, withhold WELIREG until ≥8 g/dL, then resume at the same or reduced dose or permanently discontinue WELIREG, depending on the severity of anemia. For life-threatening anemia or when urgent intervention is indicated, withhold WELIREG until hemoglobin ≥8 g/dL, then resume at a reduced dose or permanently discontinue WELIREG.

In LITESPARK-004 (N=61), decreased hemoglobin occurred in 93% of patients with VHL disease and 7% had Grade 3 events. Median time to onset of anemia was 31 days (range: 1 day to 8.4 months).

The safety of erythropoiesis-stimulating agents (ESAs) for treatment of anemia in patients with VHL disease treated with WELIREG has not been established.

In LITESPARK-005 (n=372), decreased hemoglobin occurred in 88% of patients with advanced RCC with a clear cell component and 29% had Grade 3 events. Median time to onset of anemia was 29 days (range: 1 day to 16.6 months). Of the patients with anemia, 22% received transfusions only, 20% received ESAs only, and 12% received both transfusion and ESAs.

In LITESPARK-015, anemia occurred in 96% of patients and 22% had Grade 3 events. Median time to onset of anemia was 29 days (range: 1 day to 22.1 months). Of the patients with anemia, 20% received transfusions only, 26% received ESAs only, and 6% received both transfusion and ESAs.

Hypoxia
WELIREG can cause severe hypoxia that may require discontinuation, supplemental oxygen, or hospitalization.

Monitor oxygen saturation before initiation of, and periodically throughout, treatment. For decreased oxygen saturation with exercise (e.g., pulse oximeter <88% or PaO2 ≤55 mm Hg), consider withholding WELIREG until pulse oximetry with exercise is greater than 88%, then resume at the same dose or a reduced dose. For decreased oxygen saturation at rest (e.g., pulse oximeter <88% or PaO2 ≤55 mm Hg) or when urgent intervention is indicated, withhold WELIREG until resolved and resume at a reduced dose or discontinue. For life-threatening hypoxia or recurrent symptomatic hypoxia, permanently discontinue WELIREG. Advise patients to report signs and symptoms of hypoxia immediately to a healthcare provider.

In LITESPARK-004, hypoxia occurred in 1.6% of patients.

In LITESPARK-005, hypoxia occurred in 15% of patients and 10% had Grade 3 events. Of the patients with hypoxia, 69% were treated with oxygen therapy. Median time to onset of hypoxia was 30.5 days (range: 1 day to 21.1 months).

In LITESPARK-015, hypoxia occurred in 13% of patients and 10% had Grade 3 hypoxia. Median time to onset of hypoxia was 35 days (range: 6 days to 23.9 months). Of the patients with hypoxia, 67% were treated with oxygen therapy.

Embryo-Fetal Toxicity
Based on findings in animals, WELIREG can cause fetal harm when administered to a pregnant woman.

Advise pregnant women and females of reproductive potential of the potential risk to the fetus. Advise females of reproductive potential to use effective non-hormonal contraception during treatment with WELIREG and for 1 week after the last dose. WELIREG can render some hormonal contraceptives ineffective. Advise male patients with female partners of reproductive potential to use effective contraception during treatment with WELIREG and for 1 week after the last dose.

Adverse Reactions

Adverse Reactions in LITESPARK-004
Serious adverse reactions occurred in 15% of patients, including anemia, hypoxia, anaphylaxis reaction, retinal detachment, and central retinal vein occlusion (1 patient each).

WELIREG was permanently discontinued due to adverse reactions in 3.3% of patients for dizziness and opioid overdose (1.6% each).

Dosage interruptions due to an adverse reaction occurred in 39% of patients. Those which required dosage interruption in >2% of patients were fatigue, decreased hemoglobin, anemia, nausea, abdominal pain, headache, and influenza-like illness.

Dose reductions due to an adverse reaction occurred in 13% of patients. The most frequently reported adverse reaction which required dose reduction was fatigue (7%).

The most common adverse reactions (≥25%), including laboratory abnormalities, that occurred in patients who received WELIREG were decreased hemoglobin (93%), fatigue (64%), increased creatinine (64%), headache (39%), dizziness (38%), increased glucose (34%), and nausea (31%).

Adverse Reactions in LITESPARK-005
Serious adverse reactions occurred in 38% of patients. The most frequently reported serious adverse reactions were hypoxia (7%), anemia (5%), pneumonia (3.5%), hemorrhage (3%), and pleural effusion (2.2%). Fatal adverse reactions occurred in 3.2% of patients who received WELIREG, including sepsis (0.5%) and hemorrhage (0.5%).

WELIREG was permanently discontinued due to adverse reactions in 6% of patients. Adverse reactions which resulted in permanent discontinuation (≥0.5%) were hypoxia (1.1%), anemia (0.5%), and hemorrhage (0.5%).

Dosage interruptions due to an adverse reaction occurred in 39% of patients. Of the patients who received WELIREG, 28% were 65 to 74 years, and 10% were 75 years and over. Dose interruptions occurred in 48% of patients ≥65 years of age and in 34% of younger patients. Adverse reactions which required dosage interruption in ≥2% of patients were anemia (8%), hypoxia (5%), COVID-19 (4.3%), fatigue (3.2%), and hemorrhage (2.2%).

Dose reductions due to an adverse reaction occurred in 13% of patients. Dose reductions occurred in 18% of patients ≥65 years of age and in 10% of younger patients. The most frequently reported adverse reactions which required dose reduction (≥1.0%) were hypoxia (5%) and anemia (3.2%).

The most common (≥25%) adverse reactions, including laboratory abnormalities, were decreased hemoglobin (88%), fatigue (43%), musculoskeletal pain (34%), increased creatinine (34%), decreased lymphocytes (34%), increased alanine aminotransferase (32%), decreased sodium (31%), increased potassium (29%), and increased aspartate aminotransferase (27%).

Adverse Reactions in LITESPARK-015
Serious adverse reactions occurred in 36% of patients. The most frequently reported serious adverse reactions were anemia and hypertension (4.2% each) and pyelonephritis, pneumonia, hypoxia, dyspnea and hemorrhage (2.8% each).

WELIREG was permanently discontinued due to adverse reactions in 2 patients (2.8%). Adverse reactions which resulted in permanent discontinuation were increased alanine aminotransferase and paraparesis (1.4% each).

Dosage interruptions due to an adverse reaction occurred in 40% of patients. Of the patients who received WELIREG, 13% were ≥65 years old and 4.2% were ≥75 years. Adverse reactions which required dosage interruption in >3% of patients were hypoxia, nausea and fatigue (4.2% each).

Dose reductions due to an adverse reaction occurred in 14% of patients. The most frequently reported adverse reaction which required dose reduction was hypoxia (4.2%).

The most common (≥25%) adverse reactions, including laboratory abnormalities, that occurred in patients were anemia (96%), fatigue (56%), musculoskeletal pain (56%), decreased lymphocytes (54%), increased alanine aminotransferase (51%), increased aspartate aminotransferase (42%), increased calcium (34%), dyspnea (33%), increased potassium (31%), decreased leukocytes (30%), headache (29%), increased alkaline phosphatase (25%), dizziness (26%) and nausea (25%).

Drug Interactions
Coadministration of WELIREG with inhibitors of UGT2B17 or CYP2C19 increases plasma exposure of belzutifan, which may increase the incidence and severity of adverse reactions. Monitor for anemia and hypoxia and reduce the dosage of WELIREG as recommended.

Coadministration of WELIREG with CYP3A4 substrates decreases concentrations of CYP3A4 substrates, which may reduce the efficacy of these substrates or lead to therapeutic failures. Avoid coadministration with sensitive CYP3A4 substrates. If coadministration cannot be avoided, increase the sensitive CYP3A4 substrate dosage in accordance with its Prescribing Information. Coadministration of WELIREG with hormonal contraceptives may lead to contraceptive failure or an increase in breakthrough bleeding.

Lactation
Because of the potential for serious adverse reactions in breastfed children, advise women not to breastfeed during treatment with WELIREG and for 1 week after the last dose.

Females and Males of Reproductive Potential
WELIREG can cause fetal harm when administered to a pregnant woman. Verify the pregnancy status of females of reproductive potential prior to initiating treatment with WELIREG.

Use of WELIREG may reduce the efficacy of hormonal contraceptives. Advise females of reproductive potential to use effective non-hormonal contraception during treatment with WELIREG and for 1 week after the last dose. Advise males with female partners of reproductive potential to use effective contraception during treatment with WELIREG and for 1 week after the last dose.

Based on findings in animals, WELIREG may impair fertility in males and females of reproductive potential and the reversibility of this effect is unknown.

Pediatric Use
The safety and effectiveness of WELIREG have been established in pediatric patients aged 12 years and older for the treatment of locally advanced, unresectable, or metastatic pheochromocytoma or paraganglioma.

Renal Impairment
For patients with severe renal impairment (eGFR 15-29 mL/min estimated by MDRD), monitor for increased adverse reactions and modify the dosage as recommended.

Hepatic Impairment
WELIREG has not been studied in patients with severe hepatic impairment (total bilirubin >1.5 x ULN and any AST). For patients with moderate and severe hepatic impairment, monitor for increased adverse reactions and modify the dosage as recommended.

On May 14, 2025 Azitra, Inc. (NYSE American: AZTR), a clinical stage biopharmaceutical company focused on developing innovative therapies for precision dermatology, reported that it will present a poster describing the Phase 1/2 clinical trial of ATR04-484 for EGFR inhibitor ("EGFRi")-associated rash at the 28th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) (Press release, Azitra, MAY 14, 2025, View Source [SID1234653053]). ASGCT (Free ASGCT Whitepaper) is being held May 13-17, 2025, in New Orleans, Louisiana.

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Details of the presentation are as follows:

Event:

American Society of Gene and Cell Therapy

Location:

New Orleans Ernest N. Morial Convention Center, New Orleans, Louisiana

When:

May 15, 2025; 5:30-7:00PM CT

Title:

A Novel Staphylococcus epidermidis Compound for the Topical Treatment Epidermal
Growth Factor Receptor (EGFR) Inhibitor-Induced Dermal Toxicity

Presenter:

Mary Spellman, MD, Chief Medical Officer

Registration:

View Source

"The Annual Meeting of ASGCT (Free ASGCT Whitepaper) is an important conference for researchers and industry leaders who are seeking to develop next-generation cell and gene therapies for patients in need," said Mary Spellman, MD, Chief Medical Officer of Azitra. "We are excited to present supporting evidence for the clinical development of topical ATR04-484 and our Phase 1/2 clinical study design, in anticipation of initiating dosing for the first patient in the coming month."

On May 14, 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 its financial results for the first quarter 2025 ended March 31, 2025 (Press release, Moleculin, MAY 14, 2025, View Source [SID1234653068]). As previously announced, the Company will host a conference call and live audio webcast to discuss the operational and financial results today, May 14, 2025 at 8:30 AM ET.

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"We are pleased with the continued progress of our pivotal, adaptive Phase 3 MIRACLE trial and remain encouraged by the Annamycin data demonstrated to date. In particular, with the sites opening in the US, the recent approval from the EMA, and the individual country committee and/or ethics approvals we have received for Belgium, Czechia, France, Germany, Italy, Lithuania, Poland, Romania, and Spain positions us to continue building momentum and remain on track with our expected enrollment and data milestones," commented Walter Klemp, Chairman and Chief Executive Officer of Moleculin.

Mr. Klemp continued, "In addition to the progress with our AML program, we are seeing advancements across our pipeline. We continue to be encouraged by the MB-107 trial data demonstrated by Annamycin for the treatment of STS lung mets and expect to report final data readouts from that trial before the end of June. Additionally, investigator-initiated clinical and preclinical work continues on WP1066, our STAT3 inhibitor."

Recent Highlights

Received European Medicines Agency (EMA) approval for its Clinical Trial Application (CTA) to conduct Phase 3 MIRACLE clinical trial in all nine countries submitted in the European Union (EU);
Announced the International Nonproprietary Names (INN) Expert Committee of the World Health Organization approved "naxtarubicin" for the non-proprietary names of the Company’s next-generation anthracycline in development, Annamycin;
Bolstered Annamycin intellectual property portfolio with granting of two new U.S. patents: U.S. patent number 12,257,261 titled, "Preparation of Preliposomal Annamycin Lyophilizate" and U.S. patent 12,257,262 titled "Method of Reconstituting Liposomal Annamycin";
Presented new pre-clinical data for Annamycin demonstrating market expansion potential including treatment for pancreatic cancer at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2025; and
Commenced patient dosing in its ongoing pivotal, adaptive design Phase 3 MIRACLE trial.
Clinical Development Update

Relapsed or Refractory (R/R) Acute Myeloid Leukemia (AML)

The Company is currently evaluating Annamycin (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") in a Phase 3 pivotal trial for the treatment of AML patients who are refractory to or relapsed after induction therapy (R/R AML). This Phase 3 "MIRACLE" trial (derived from Moleculin R/R AML AnnAraC Clinical Evaluation) will be global, including sites in the US, Europe and the Middle East. As of the end of April 2025, 38 sites have been selected in all of the regions targeted, with 5 sites in the US.

The MIRACLE study is a Phase 2B/3 clinical trial whereby data from the 2B portion will be combined with the Phase 3 portion for purposes of measuring its primary efficacy endpoint. MIRACLE is subject to appropriate future filings with and potential additional feedback from the FDA and their foreign equivalents, utilizes an adaptive design whereby the first 75 to 90 subjects will be randomized (1:1:1) in Part A of the trial to receive high dose cytarabine (HiDAC) combined with either placebo, 190 mg/m2 of Annamycin, or 230 mg/m2 of Annamycin, which Annamycin doses were specifically recommended by the FDA in the Company’s end of Phase 1B/2 meeting.

The protocol for the MIRACLE trial allows for the unblinding of preliminary primary efficacy data (Complete Remission or CR) and safety/tolerability of the three arms at 45 subjects, in addition to the conclusion of Part A (at 75 to 90 subjects). The first early unblinding will yield 30 subjects treated with Annamycin (190mg/m2 and 230 mg/m2) and HiDAC and 15 subjects treated with just HiDAC plus placebo. The Company expects to reach the first unblinding (45 subjects) in the second half of 2025, in addition to the second unblinding, which is expected in the first half of 2026. This accelerated estimated timeline is due in part to the positive response the Company received in meetings during December with potential investigators regarding recruitment for the trial.

The clinical trial approval with EMA was granted under the condition that the Company present results of appropriate nonclinical GLP studies before initiating the Phase 3 portion (Part B) of the study. Results will be submitted as a substantial modification to the existing approved protocol.

For Part B of the trial, approximately 220 additional subjects will be randomized to receive either HiDAC plus placebo or HiDAC plus the optimum dose of Annamycin (randomized 1:1). The selection of the optimum dose will be based on the overall balance of safety, pharmacokinetics and efficacy, consistent with the FDA’s new Project Optimus initiative.

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. Additionally, the clinical trial in the EU is on clinicaltrials.eu, and the reference identifier there is 2024-518359-47-00.

Expected Milestones for Annamycin AML Development Program

1Q – 3Q 2025 – Update on MIRACLE trial site selection/approvals by countries
2025 – Recruitment update for MIRACLE trial
2H 2025 – Data readout (n=45) unblinded efficacy/safety review
2H 2025 – 2026 – Impact of data readout (n=45) on regulatory pathway; Recruitment update
1H 2026 – Interim efficacy and safety data (n=~75-90) unblinded and Optimum Dose set for MIRACLE trial
2027 – Begin enrollment of 3rd line subjects in MIRACLE2
2027 – Enrollment ends in 2nd line subjects
2027 – Begin enrollment in pediatric AML trial
2028 – Primary efficacy data for 2nd line subjects in MIRACLE
2028 – Begin submission of a Rolling New Drug Application (NDA) for the treatment of R/R AML for accelerated approval on primary endpoint of CR from MIRACLE
2028 – Primary efficacy data for 2nd line subjects
2028 – Rolling NDA submission begins
Soft Tissue Sarcoma (STS) Lung Metastases

As previously announced, the Company completed enrollment in the Phase 2 portion of its U.S. Phase 1B/2 clinical trial evaluating Annamycin as monotherapy for the treatment of soft tissue sarcoma lung metastases. Subjects who had stable disease at the time of study discontinuation were followed for progression free response and overall survival. The clinical study report is finalized but has not yet been filed. The Company remains positive about the data and is expected to release the data by the end of June.

Expected Milestones for Annamycin STS Lung Mets Development Program

1H 2025 – Final MB-107 data readout
2025 – Identify next phase of development / pivotal IIT (investigator-initiated-trial) program
Annamycin (naxtarubicin) currently has Fast Track Status and Orphan Drug Designation from the FDA for the treatment of relapsed or refractory acute myeloid leukemia, in addition to Orphan Drug Designation for the treatment of soft tissue sarcoma. Furthermore, Annamycin has Orphan Drug Designation for the treatment of relapsed or refractory acute myeloid leukemia from the European Medicines Agency (EMA).

WP1066 & Brain Tumors

With regard to the Company’s WP1066 oral formula, the Company has an externally funded phase 1B/2 in combination with radiation treating glioblastoma (GBM), a form of brain cancer, at Northwestern University (Northwestern) that is actively recruiting. This is an investigator-initiated trial where Moleculin’s main cost is supplying drug product. To date Northwestern has recruited 7 subjects. No data has been released. Also, the Company has signed an agreement with Emory University enabling Emory to study various WP1066 IV formulations in preclinical studies with the goal of selecting the best molecule to move into a clinical setting towards, most likely, brain cancers such as GBM. Study drug was delivered in April 2025 to Emory with results from such studies expected in the second half of 2025.

Summary of Financial Results for the First Quarter 2025

Research and development (R&D) expense was $3.4 million and $4.3 million for the three months ended March 31, 2025 and 2024, respectively. The decrease of $0.9 million is mainly related to the clinical trials activity levels.

General and administrative expense was $2.5 million and $2.4 million for the three months ended March 31, 2025 and 2024, respectively. The increase of $0.1 million is mainly related to a slight overall increase in regulatory and legal fees.

As of March 31, 2025, the Company had cash and cash equivalents of $7.7 million and believes that the cash on hand is sufficient to fund planned operations into the third quarter of 2025.

Conference Call and Webcast

Moleculin management will host a conference call and live audio webcast to discuss the operational and financial results today, Wednesday, May 14, 2025 at 8:30 AM ET.

Interested participants and investors may access the conference call by dialing (877) 407-0832 (domestic) or (201) 689-8433 (international) and referencing the Moleculin Biotech Conference Call. The live audio webcast will be accessible on the Events page of the Investors section of the Moleculin website, moleculin.com, and will be archived for 90 days.