On September 9, 2025 ARTBIO, Inc. ("ARTBIO"), a clinical-stage radiopharmaceutical company developing a new class of Pb212 alpha radioligand therapies (ARTs), reported that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for AB001, and confirmed the study may proceed (Press release, ARTBIO, SEP 9, 2025, View Source [SID1234655891]). The news follows a recent Series B capital raise to advance the company’s pipeline and proprietary technology platform for Pb212-based therapies.

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"AB001 is a novel PSMA-targeted and Pb212-based radioligand therapy designed to treat patients with metastatic prostate cancer, one that we believe has the potential to increase the radioligand dose rate to tumors without higher toxicity to normal tissues," said Margaret Yu, M.D., Chief Medical Officer of ARTBIO. "Following FDA review, our Phase 1 study may now move ahead. This is welcome news for patients who are eagerly awaiting new targeted treatment options and for investigators who are ready to begin screening patients for this trial."

AB001 had been previously administered to patients in a Phase 0 study conducted in Norway with encouraging biodistribution results in those participants. The report on this study was included as part of the IND.

"Despite the availability of many treatment options, metastatic prostate cancer is still not a curable disease, requiring additional innovative treatments," said Oliver Sartor, M.D., Director of the Transformational Prostate Cancer Research Center at East Jefferson General Hospital Cancer Center and ARTBIO Scientific Advisory Board Member. "I’m grateful for ARTBIO’s dedication and passion in this area and am excited about the difference that AB001 could make for patients."

ARTBIO plans to initiate the Phase 1 clinical trial for AB001 across multiple sites in the U.S. by leveraging its distributed manufacturing network of production hubs, including leading radiopharmaceutical CDMOs like SpectronRx, PharmaLogic, and Nucleus Radiopharma.

Clinical trial supply will be enabled by ARTBIO’s proprietary Pb212 generator technology, AlphaDirect, with precursor isotopes being sourced from the U.S. Department of Energy. The company plans to enroll patients outside of the U.S. once additional regulatory approvals are secured.

"This IND clearance represents critical progress in translating the unique biology of Pb212 into patient care," said Dr. Michael Morris, Professor of Medicine, Prostate Section Head at Memorial Sloan Kettering Cancer Center and ARTBIO Clinical Advisory Board Member. "With the ability to target radiation at the cellular level, this program has the potential to establish a new standard of care in metastatic prostate cancer."

About AB001
AB001 is an Alpha Radioligand Therapy (ART) consisting of a prostate-specific membrane antigen (PSMA)-targeted small molecule radiolabeled with Pb212. PSMA is commonly overexpressed in mCRPC and has become an attractive target for imaging agents and therapies.

On September 9, 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 updates to its active site status and recruitment for 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) (Press release, Moleculin, SEP 9, 2025, View Source [SID1234655877]). This Phase 3 "MIRACLE" trial (derived from Moleculin R/R AML AnnAraC Clinical Evaluation) is a global approval trial, including sites in the US, Europe and the Middle East.

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To advance this trial to recruiting the 45th subject in Q4 2025 for its first unblinding for early data insights into the trial’s efficacy and safety endpoints, the Company has the following expectations for September:

Global Expansion: Expanding recruiting beyond Ukraine to include Spain, Georgia, Poland, Romania, Italy, Lithuania, and the US;
Site Expansion: Adding 8 new active sites by the end of September, increasing the total to 20 sites recruiting;
Recruitment Goals: Treating, enrolling, or screening a total of 20 subjects by the end of September; and
Data Insights: Setting a solid footprint to recruit the 45th subject in Q4 2025, allowing for unblinded preliminary data assessment regarding efficacy and safety upon data lock.
Walter Klemp, Chairman and CEO of Moleculin, commented, "Our team is diligently expanding our active site footprint across the US, EU, and neighboring regions. We are confident in our target to recruit the 45th subject by Q4 2025 and achieve our first data unblinding shortly thereafter, especially since the consistent message we are receiving from investigators is that they are very eager to enroll patients in the MIRACLE trial. Moving from our end of Phase 2 meeting with the FDA to screening 13 subjects in just over a year exemplifies our operational efficiency. Of these 13, 10 have been dosed, further supporting our confidence in the speed of recruitment for MIRACLE."

Mr. Klemp continued, "We expect to increase to over 30 active sites by year-end, as we move toward a second data unblinding for the conclusion of Part A of the trial. That will only require an additional 30 to 45 subjects, so we expect to conclude Part A in the first half of 2026, before commencing Part B of the trial. This level of early visibility is, we believe, exceptionally rare for a Phase 2B/3 trial and highly advantageous for investors."

The MIRACLE study is a Phase 2B/3 clinical trial whereby data from the 2B (Part A) portion will be combined with the Phase 3 (Part B) 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) in combination with HiDAC and 15 subjects treated with just HiDAC plus placebo. The Company expects to reach the recruitment of the first 45 subjects in the second half of 2025 with unblinding shortly thereafter, 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.

As previously announced with regard to the EU, 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 CTA.

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.

For more information about the MIRACLE trial, visit clinicaltrials.gov and reference identifier NCT06788756. Additionally, the clinical trial in the EU is on euclinicaltrials.eu and the reference identifier there is 2024-518359-47-00.

Annamycin, also known by its non-proprietary name of 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. Annamycin also benefits from composition of matter patent protection through 2040 with the potential to extend that protection as far as 2045. Furthermore, Annamycin has Orphan Drug Designation for the treatment of relapsed or refractory acute myeloid leukemia from the EMA.

On September 9, 2025 DeuterOncology, a clinical-stage biotech company focused on developing best-in-class deuterated cancer therapies, reported compelling safety, pharmacokinetic and efficacy results from its ongoing Phase I dose escalation study of DO-2, a novel deuterated MET tyrosine kinase inhibitor (Press release, DeuterOncology, SEP 9, 2025, View Source [SID1234655892]). The results are being presented at the World Conference on Lung Cancer (WCLC) 2025 in Barcelona (Abstract ID-2859).

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The Phase I study enrolled 29 patients (26 RECIST 1.1 evaluable) with advanced solid tumors harboring any known driver alterations, with 86% having non-small cell lung cancer (NSCLC). The study’s primary findings demonstrate that DO-2 treated patients showed a remarkable 100% Disease Control Rate (DCR) and saw tumor shrinkage in 100% of MET exon 14 skip mutation-positive NSCLC patients without alternative oncogenic drivers and reached efficacious drug exposures (10/10 patients). RECIST 1.1 Partial responses (>30% reduction) were observed in 2 out of these 10 patients. Importantly, the study showed a markedly improved safety profile compared to approved MET inhibitors, with only one patient (5%) experiencing grade 1 peripheral oedema versus 68-82% seen with other agents in this class.

"These results represent a significant advancement in MET-targeted therapy," said Dr. Cecilia Ahlin, CMO of DeuterOncology. "Our deuteration strategy has successfully enhanced drug exposure while maintaining efficacy, but most importantly, we have achieved this without the debilitating peripheral oedema that forces many patients to discontinue current MET inhibitors —symptoms that can severely limit daily activities such as walking, dressing, or cooking. This addresses a critical unmet medical need for cancer patients."

The study’s key findings include:

Superior Safety Profile: Only 1 patient (5%) experienced peripheral oedema (grade 1) compared to 68-82% with approved competitors. No grade 3 or 4 peripheral oedema was observed versus ~17% in approved competitors
Enhanced Pharmacokinetics: Deuteration increased plasma exposure compared to the non-deuterated parent compound, enabling once-daily dosing at 60mg with food, versus 450-800mg daily with approved competitors
Promising Efficacy: A 100% disease control rate (DCR) was observed, including two partial responses, in treatment-naïve and previously treated MET exon 14 skipping NSCLC patients without known resistance mechanisms who achieved efficacious drug exposure. Notably, one patient with pleural effusion experienced full resolution and maintained stable disease for over 20 months
Excellent Tolerability: Only two grade 3 treatment-related adverse events (fatigue and reversible creatinine increase) were observed across all dose levels
Dr. Hans Prenen, Principal Investigator at University Hospital Antwerp (UZA), commented: "The clinical data for DO-2 are very encouraging. The fact that we’re seeing robust anti-tumor activity with minimal peripheral oedema represents a potential game-changer for MET-driven lung cancer patients, who currently face difficult treatment tolerability issues with existing therapies."

The study confirms DeuterOncology’s hypothesis that continuous 24/7 MET inhibition is not required for efficacy but is responsible for the class-effect toxicity of peripheral oedema. DO-2’s unique biochemical and pharmacokinetic profile allows for effective target engagement while providing recovery periods that minimize on-target toxicities.

Based on these promising Phase I results, DeuterOncology has initiated an expansion cohort in selected first-line MET exon 14 skip NSCLC patients to further validate the safety and efficacy of the 60mg once-daily regimen.

Details of the poster presentation are as follows:

Title: Preliminary Safety and PK of the MET-TKI DO-2 in Advanced Solid Tumors with MET Aberrations: Phase I Study

Authors: Hans Prenen MD, PhD, Rachel Galot MD, PhD, Alexis Cortot MD, PhD, Aurélie Swalduz MD, PhD, Bernd Dekeyser MD, Peter de Bruijn PhD, Francois Zammit MD, Jean-Pascal Machiels MD, PhD, Brant Delafontaine MD, Sylvie Rottey MD, PhD, Ingrid Desar MD, PhD, Marthe Paats MD, PhD, Barend J. Sikkema3MD, , Florence Wastelin MSc, Cecilia Ahlin MD PhD, Timothy Perera PhD, Jaap Verweij MD, PhD, Carla van Herpen MD, PhD, Debbie G.J. Robbrecht MD, PhD

Presenter: Timothy Perera

Session date and time: 09 September 2025. 10.00AM CEST

Poster ID number: Abstract ID-2859. P3.18.66

About MET-Driven Lung Cancer

Lung cancer remains the most common form of cancer worldwide, affecting approximately 2.2 million individuals annually. MET exon 14 skipping mutations occur in ~3% of NSCLC patients—equating to an estimated 60,000–70,000 new cases worldwide annually—and represent a validated therapeutic target. However, current approved MET inhibitors suffer from high rates of peripheral oedema (>50% of patients), leading to frequent dose reductions, treatment interruptions, and discontinuations that compromise patient outcomes.

On September 9, 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 updates to its active site status and recruitment for 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) (Press release, Moleculin, SEP 9, 2025, View Source [SID1234655877]). This Phase 3 "MIRACLE" trial (derived from Moleculin R/R AML AnnAraC Clinical Evaluation) is a global approval trial, including sites in the US, Europe and the Middle East.

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To advance this trial to recruiting the 45th subject in Q4 2025 for its first unblinding for early data insights into the trial’s efficacy and safety endpoints, the Company has the following expectations for September:

Global Expansion: Expanding recruiting beyond Ukraine to include Spain, Georgia, Poland, Romania, Italy, Lithuania, and the US;
Site Expansion: Adding 8 new active sites by the end of September, increasing the total to 20 sites recruiting;
Recruitment Goals: Treating, enrolling, or screening a total of 20 subjects by the end of September; and
Data Insights: Setting a solid footprint to recruit the 45th subject in Q4 2025, allowing for unblinded preliminary data assessment regarding efficacy and safety upon data lock.
Walter Klemp, Chairman and CEO of Moleculin, commented, "Our team is diligently expanding our active site footprint across the US, EU, and neighboring regions. We are confident in our target to recruit the 45th subject by Q4 2025 and achieve our first data unblinding shortly thereafter, especially since the consistent message we are receiving from investigators is that they are very eager to enroll patients in the MIRACLE trial. Moving from our end of Phase 2 meeting with the FDA to screening 13 subjects in just over a year exemplifies our operational efficiency. Of these 13, 10 have been dosed, further supporting our confidence in the speed of recruitment for MIRACLE."

Mr. Klemp continued, "We expect to increase to over 30 active sites by year-end, as we move toward a second data unblinding for the conclusion of Part A of the trial. That will only require an additional 30 to 45 subjects, so we expect to conclude Part A in the first half of 2026, before commencing Part B of the trial. This level of early visibility is, we believe, exceptionally rare for a Phase 2B/3 trial and highly advantageous for investors."

The MIRACLE study is a Phase 2B/3 clinical trial whereby data from the 2B (Part A) portion will be combined with the Phase 3 (Part B) 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) in combination with HiDAC and 15 subjects treated with just HiDAC plus placebo. The Company expects to reach the recruitment of the first 45 subjects in the second half of 2025 with unblinding shortly thereafter, 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.

As previously announced with regard to the EU, 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 CTA.

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.

For more information about the MIRACLE trial, visit clinicaltrials.gov and reference identifier NCT06788756. Additionally, the clinical trial in the EU is on euclinicaltrials.eu and the reference identifier there is 2024-518359-47-00.

Annamycin, also known by its non-proprietary name of 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. Annamycin also benefits from composition of matter patent protection through 2040 with the potential to extend that protection as far as 2045. Furthermore, Annamycin has Orphan Drug Designation for the treatment of relapsed or refractory acute myeloid leukemia from the EMA.

On September 9, 2025 Tempus AI, Inc. (NASDAQ: TEM), a technology company leading the adoption of AI to advance precision medicine, reported the publication of a study in JCO Precision Oncology validating the clinical utility of the company’s PurIST algorithmic diagnostic (Press release, Tempus, SEP 9, 2025, View Source [SID1234655894]). The study provides the largest real-world evidence to date supporting the integration of PurIST into routine clinical care for patients with advanced PDAC, with the aim of informing first-line chemotherapy selection and improving patient outcomes.

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Pancreatic cancer remains one of the most lethal malignancies, with limited therapeutic options and a five-year survival rate of just 12%. For patients with advanced, unresectable PDAC, the two most common first-line chemotherapy regimens, FOLFIRINOX (FFX) and gemcitabine plus nab-paclitaxel (GnP), have shown variable efficacy, and clinicians have lacked robust biomarkers to guide optimal therapy selection. To address this challenge, Tempus collaborated with GeneCentric to develop and deploy PurIST, a clinically validated, RNA-based algorithm test that classifies PDAC tumors as either "classical" or "basal" subtypes.

The Tempus-led study analyzed a real-world cohort of 931 patients with advanced PDAC, using the Tempus xR RNA sequencing platform to assign PurIST subtypes. Patients were treated with either first-line FFX or GnP, and clinical outcomes were assessed according to PurIST classification. The study’s findings establish PurIST as both a prognostic and predictive biomarker, enabling clinicians to personalize first-line therapy for advanced PDAC patients and maximize the likelihood of improved survival.

Prognostic Value: Among patients treated with FFX (N=536), those with the classical subtype had a significantly longer median overall survival (OS) of 11.8 months, compared to 7.0 months for basal subtype patients (Hazard Ratio [HR]=1.86; p<0.001).
Predictive Value: In patients with the classical subtype and good performance status (ECOG 0 or 1, N=311), treatment with FFX was associated with a 33% relative risk reduction in death compared to GnP (HR=0.67; p<0.009). No comparable benefit was observed in basal subtype patients.
"Pancreatic cancer is a challenging disease, and the ability to match patients to the most effective first-line therapy is critical. Prior research has suggested that certain molecular subtypes are associated with distinct prognoses and responses to therapy, but until now, large-scale real-world validation in a clinical setting has been lacking," said Ezra Cohen, MD, Chief Medical Officer, Oncology. "Our study demonstrates that PurIST subtyping enables a biomarker-driven approach to therapy selection, addressing a major unmet need in the management of pancreatic cancer."

"PurIST is a step forward for pancreatic cancer molecular testing, giving healthcare providers a new tool to better inform personalized treatment options for this devastating disease," said Michael Milburn, PhD, President and CEO of GeneCentric and co-author on the publication. "GeneCentric’s collaboration with Tempus allowed us to validate the algorithm leveraging the power of Tempus’ multimodal data to rapidly deliver a much-needed test for patients with PDAC."