Curatis has revealed Corticorelin as the active substance in its investigational therapy C-PTBE-01, targeting peritumoral brain edema (PTBE) associated with brain tumors. The company also announced a significant upward revision of its market size estimates, now projecting a potential market opportunity exceeding USD 1 billion.
According to the company's recent epidemiological market analysis, the PTBE patient population is substantially larger than previously estimated. Curatis now projects more than 150,000 patients in the United States and approximately 500,000 patients globally who suffer from PTBE associated with primary and metastatic brain tumors.
Understanding PTBE and Current Treatment Limitations
PTBE occurs when fluid accumulates around brain tumors, causing significant neurological symptoms including headaches, vomiting, paralysis, speech disorders, visual problems, and altered mental status. The condition commonly develops in association with primary brain tumors and metastatic lesions, particularly those originating from lung cancer, breast cancer, melanoma, and colorectal cancer.
The current standard of care for PTBE relies heavily on corticosteroids, which effectively reduce inflammation but carry serious side effects. These include severe myopathy, impaired glucose metabolism, muscle wasting, abnormal weight gain, osteoporosis, gastritis, gastrointestinal bleeding, hypertension, and personality changes.
Perhaps most concerning for cancer patients, corticosteroids can counteract certain cancer therapies, including chemotherapy and emerging immunotherapies that depend on T-cell functionality, which steroids impair.
Corticorelin's Therapeutic Potential
Corticorelin has demonstrated a strong steroid-sparing effect in both preclinical studies and two clinical trials. The compound shows potential to significantly reduce or completely replace steroid use in PTBE patients, which could:
- Reduce or eliminate severe glucocorticoid-related side effects
- Improve patient quality of life
- Maintain the efficacy of novel tumor therapies that are currently compromised by steroid co-administration
Development Timeline and Strategic Appointments
Curatis is targeting a meeting with the U.S. Food and Drug Administration (FDA) in the third quarter of 2024 to gain agreement on the design of a pivotal Phase 3 trial. The company plans to initiate this trial in 2026.
To strengthen its development capabilities, Curatis has announced two key appointments:
Dr. Kirsty Crame, MD, joins the company with over 12 years of clinical development experience, including more than 10 years in immuno-oncology across various indications for both rare and common diseases. Dr. Crame previously served as Vice President of Clinical Strategy and Development at Medigene and as Director of Clinical Development at Gadeta. She will support Curatis in preparing and conducting the pivotal Phase 3 study for C-PTBE-01, leveraging her extensive regulatory affairs experience.
Dr. Timm Trenktrog, PhD, brings over 19 years of experience from Actelion Pharmaceuticals Ltd., where he most recently served as Vice President and Head of Technical Operations on the Executive Board. Dr. Trenktrog will lead technical development and manufacturing (CMC) for C-PTBE-01, ensuring high-quality production of the active ingredient, dosage form, and final product.
Market Implications
The revised market size estimate of over USD 1 billion highlights the significant commercial potential for C-PTBE-01. If successful in clinical trials and approved, the therapy could address a critical unmet need for PTBE patients who currently face difficult trade-offs between managing edema symptoms and enduring severe steroid-related side effects.
For cancer patients specifically, a steroid-sparing or steroid-replacing therapy could potentially improve outcomes by allowing full efficacy of concurrent cancer treatments, particularly immunotherapies that are increasingly important in the treatment of many cancers that commonly metastasize to the brain.
