Radiopharm Theranostics Granted U.S. Food and Drug Administration Fast Track Designation for RAD101 Imaging in Brain Metastases

Rhea-AI Summary

Radiopharm Theranostics (RADX) has received FDA Fast Track Designation for RAD101, their novel imaging molecule designed to differentiate between recurrent brain metastases and treatment effects in solid tumors. RAD101 targets fatty acid synthase (FASN), a protein overexpressed in many solid tumors including cerebral metastases. The designation highlights the critical need for better diagnostic tools in managing brain metastases, which affects over 300,000 U.S. patients annually. Currently in Phase 2 clinical trials, Radiopharm expects to release topline results in H2 2025. The Fast Track status enables more frequent FDA communication, potential rolling review, and possible Priority Review eligibility.

Positive

• FDA Fast Track Designation granted for RAD101, potentially accelerating development and review process
• Addresses large market opportunity with over 300,000 U.S. patients diagnosed annually with brain metastases
• Phase 2 clinical trial results expected in second half of 2025
• Potential eligibility for Priority Review if criteria are met

Negative

• None.

Insights

Oncology Imaging Expert

FDA Fast Track for RAD101 accelerates potential breakthrough in brain metastasis diagnosis, addressing critical clinical uncertainty for 300,000+ annual US patients.

Radiopharm Theranostics has secured FDA Fast Track Designation for RAD101, a significant regulatory milestone that acknowledges both the serious nature of brain metastases and the unmet diagnostic need in this field. This designation carries substantial weight as it can potentially shorten the development timeline and expedite regulatory review for this innovative diagnostic agent.

The clinical significance of RAD101 cannot be overstated. Current imaging modalities often struggle to differentiate between actual tumor recurrence and treatment-related changes like radiation necrosis or pseudoprogression in brain metastases. This diagnostic uncertainty frequently leads to clinical dilemmas and potentially inappropriate treatment decisions.

RAD101's mechanism targets fatty acid synthase (FASN), an enzyme overexpressed in numerous solid tumors including cerebral metastases. This targeted approach potentially offers superior specificity compared to conventional imaging methods. The ongoing Phase 2 clinical trial will evaluate this diagnostic precision across brain metastases originating from various primary tumor types, including the particularly challenging leptomeningeal disease.

The market potential is substantial, with the company noting that over 300,000 patients are diagnosed with brain metastases annually in the U.S. alone. The anticipated timeline for topline results in the second half of 2025 suggests that if successful, this imaging agent could begin impacting clinical practice within a relatively short timeframe.

Beyond expedited review, Fast Track designation may afford Radiopharm more frequent FDA interactions and the possibility of rolling review for marketing applications. If the Phase 2 results demonstrate clinical utility, RAD101 could significantly improve treatment decision-making in this complex patient population while establishing Radiopharm as an innovator in the oncology imaging space.

RAD101 in U.S. Phase 2 clinical trial to distinguish between recurrent disease and treatment effect of brain metastases originating from solid tumors of different origin

SYDNEY, June 11, 2025 (GLOBE NEWSWIRE) -- Radiopharm Theranostics (ASX:RAD, “Radiopharm” or the “Company”), a clinical-stage biopharmaceutical company focused on developing innovative oncology radiopharmaceuticals for areas of high unmet medical need, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for RAD101 to distinguish between recurrent disease and treatment effect of brain metastases originating from solid tumors of different origin including leptomeningeal disease.

RAD101 is the Company’s novel imaging small molecule that targets fatty acid synthase (FASN), a multi-enzyme protein that catalyses fatty acid synthesis and is overexpressed in many solid tumors, including cerebral metastases.

“The FDA’s Fast Track Designation for RAD101 highlights the seriousness of recurrent brain metastases as a condition and the unmet medical need for innovative products that can differentiate between tumor recurrence and radiation necrosis or pseudprogression,” said Riccardo Canevari, CEO and Managing Director of Radiopharm Theranostics. “RAD101 represents a promising advancement in improving diagnostic precision for brain metastases, offering hope for more effective clinical decision-making in the over 300,000 patients diagnosed annually in the U.S. We are excited to advance our Phase 2 clinical trial and anticipate sharing topline results in the second half of 2025.”

The FDA’s Fast Track designation is designed to facilitate the development and expedite the review of drugs that are intended to treat serious or life-threatening conditions and demonstrate the potential to address an unmet medical need. A Sponsor that receives Fast Track designation may be eligible for more frequent meetings and communications with the FDA and rolling review of any application for marketing approval. A Sponsor’s drug receiving Fast Track designation also may be eligible for Priority Review if relevant criteria are met.

About the Phase 2 Clinical Trial of RAD101

The U.S. multicenter, open-label, single arm Phase 2b clinical trial is evaluating the diagnostic performance of 18F-RAD101 in 30 individuals with confirmed recurrent brain metastases from solid tumors of different origins. The primary objective of the study is concordance between 18F-RAD101 positive lesions and those seen in conventional imaging (MRI with gadolinium) in participants with suspected recurrent brain metastases. Secondary endpoints are accuracy, sensitivity and specificity of RAD101 in identifying tumor recurrence versus radiation necrosis in previously stereotactic radiosurgery (SRS)-treated brain metastases.

About RAD101

RAD101 is the Company’s novel imaging small molecule that targets fatty acid synthase (FASN), a multi-enzyme protein that catalyses fatty acid synthesis and is overexpressed in many solid tumors, including cerebral metastasis. Targeting FASN activity may allow for the more accurate detection of cancer cells, representing a clinically relevant method for the imaging of brain metastases. Positive data from the Imperial College of London’s Phase 2a imaging trial of 18F-RAD101 in patients with brain metastases (both SRS pre-treated and treatment naïve patients) showed significant tumor uptake that was independent from the tumor of origin. The study further indicated that PET-MRI may potentially represent a non-invasive prediction of overall-survival, warranting larger studies.

About Radiopharm Theranostics

Radiopharm Theranostics is a clinical stage radiotherapeutics company developing a world-class platform of innovative radiopharmaceutical products for diagnostic and therapeutic applications in areas of high unmet medical need. Radiopharm is listed on ASX (RAD) and on NASDAQ (RADX). The company has a pipeline of distinct and highly differentiated platform technologies spanning peptides, small molecules and monoclonal antibodies for use in cancer. The clinical program includes one Phase 2 and three Phase 1 trials in a variety of solid tumor cancers including lung, breast, and brain metastases. Learn more at radiopharmtheranostics.com.

Authorised on behalf of the Radiopharm Theranostics board of directors by Chairman Paul Hopper.

For more information:

Riccardo Canevari
CEO & Managing Director
P: +1 862 309 0293
E: rc@radiopharmtheranostics.com

Anne Marie Fields
Precision AQ (Formerly Stern IR)
E: annemarie.fields@precisionaq.com

Paul Hopper
Executive Chairman
P: +61 406 671 515
E: paulhopper@lifescienceportfolio.com

Media
Matt Wright
NWR Communications
P: +61 451 896 420
E: matt@nwrcommunications.com.au

FAQ

What is RAD101 and what is its purpose in brain metastases?

RAD101 is a novel imaging molecule that targets fatty acid synthase (FASN) to distinguish between recurrent brain metastases and treatment effects in solid tumors.

What does the FDA Fast Track Designation mean for Radiopharm Theranostics (RADX)?

The designation enables more frequent FDA communications, potential rolling review of applications, and possible Priority Review eligibility, potentially accelerating the development process.

When will Radiopharm Theranostics (RADX) release RAD101 Phase 2 trial results?

Radiopharm expects to share topline results from the Phase 2 clinical trial in the second half of 2025.

How many patients could potentially benefit from RAD101 in the US?

Over 300,000 patients are diagnosed annually in the U.S. with brain metastases who could potentially benefit from RAD101's diagnostic capabilities.

What specific problem does RAD101 address in brain metastasis treatment?

RAD101 addresses the challenge of differentiating between tumor recurrence and radiation necrosis or pseudoprogression in brain metastases, improving diagnostic precision for better clinical decision-making.