Hillstream BioPharma Receives Orphan Drug Designation for Uveal Melanoma Treatment

Hillstream BioPharma Inc. recently announced the FDA granted Orphan Drug Designation to HSB-1216 for Uveal Melanoma (UM). Hillstream continues to build an attractive oncology pipeline and expand its oncology pipeline for devastating diseases with this significant milestone achievement for HSB-1216.

The Orphan Drug Designation program provides orphan status to drugs and biologics, which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US.

(UM is the most common primary intraocular tumor that originates from melanocytes of eye uveal tract accounting for 85% to 95% of primary ocular malignancies and 3% to 5% of all melanoma cases. Approximately 50% of patients with UM manifest distant organ metastasis, predominantly to the life-sustaining organs such as the liver, even when the primary tumors have been removed. Hepatic metastasis is among the major and direct causes of poor prognosis in these UM patients. The 50% mortality rate is unchanged despite treatment advances in treating the primary eye tumor. Hillstream is rapidly progressing towards a IND filing process and plans to initiate a clinical trial in solid tumor patients.

“This designation significantly expands our oncology efforts and diversifies our lead program” said Randy Milby, Founder and Chief Executive Officer of Hillstream. “The Orphan Drug Designation for the treatment of UV highlights the significant need for patients with this devastating disease and offers a potential solution with our ferroptosis inducing agent, HSB-1216, as a novel investigational treatment. We have an orphan drug designation in small cell lung cancer with HSB-1216 and rare pediatric designation in osteosarcoma with our follow-on program, HSB-888. We look forward to sharing our progress of these and future milestones as we progress our diverse pipeline into the clinic and eventually to help patients suffering from these devastating cancers.”

This combined portfolio enables Hillstream with the additional capability to address unmet needs in multiple high unmet need orphan tumors.

Hillstream BioPharma, Inc. is a biotechnology company developing novel therapeutic candidates targeting ferroptosis, an emerging new anti-cancer mechanism resulting in iron mediated cell death for drug resistant and devastating cancers. Hillstream’s most advanced candidate is HSB-1216, an IMCD modulator, targeting a variety of solid tumors. The active drug in HSB-1216 was found to be efficacious in a clinical pilot study in Germany in devastating cancers, including triple negative breast cancer and epithelial carcinomas. Hillstream intends to start a clinical study with HSB-1216 in 2022 to expand upon the existing early clinical proof-of-concept. Hillstream uses Quatramer, its proprietary tumor targeting platform, to enhance the uptake of HSB-1216 in the tumor microenvironment with an extended duration of action and minimal off-target toxicity. In addition, Trident Artificial Intelligence, Hillstream’s artificial intelligence precision medicine platform, is used to identify biomarkers in our clinical programs to target a specific patient segment most likely to benefit. For more information, visit www.hillstreambio.com.