Hillstream BioPharma Receives Orphan Drug Designation for HSB-1216 for the Treatment of Uveal Melanoma

Rhea-AI Summary

Hillstream BioPharma has announced that the US FDA has granted Orphan Drug Designation for HSB-1216, targeting uveal melanoma, a rare and aggressive cancer. This designation highlights the unmet need in treating this condition, where 50% of patients develop metastatic disease. HSB-1216 is a ferroptosis inducer that aims to improve treatment outcomes. The company plans to progress toward an IND filing and initiate clinical trials in solid tumor patients, further diversifying its oncology pipeline.

Positive

• FDA granted Orphan Drug Designation for HSB-1216, enhancing its oncology portfolio.
• HSB-1216 targets a significant unmet need in treating uveal melanoma.
• Plans to initiate clinical trials signal progress and potential future revenue.

Negative

• Uveal melanoma has a poor prognosis with a 50% mortality rate.
• The lengthy and expensive clinical trial process poses risks for successful outcomes.

-Uveal melanoma is an aggressive intraocular malignancy with a poor prognosis in which 50% of patients develop metastatic disease-

-Evidence implicates ferroptosis, an emerging iron-mediated cell death, as a pathway to effectively eradicate uveal melanoma in proof-of-concept animal models

-HSB-1216, a novel ferroptosis inducer against solid tumors has been granted an Orphan Drug Designation for the treatment of uveal melanoma by the US FDA

Bridgewater, NJ, Feb. 16, 2022 (GLOBE NEWSWIRE) -- Hillstream BioPharma Inc. (Nasdaq: HILS) (“Hillstream”), 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, 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.¹

(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” stated 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.

About Hillstream BioPharma Inc.
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, please visit www.hillstreambio.com.

Forward Looking Statements

Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified using words such as "anticipate," "believe," "forecast," "estimated" and "intend" or other similar terms or expressions that concern Hillstream's expectations, strategy, plans or intentions. These forward-looking statements are based on Hillstream's current expectations and actual results could differ materially. There are several factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, clinical trials involve a lengthy and expensive process with an uncertain outcome, and results of earlier studies and trials may not be predictive of future trial results; our clinical trials may be suspended or discontinued due to unexpected side effects or other safety risks that could preclude approval of our product candidates; risks related to business interruptions, including the outbreak of COVID-19 coronavirus, which could seriously harm our financial condition and increase our costs and expenses; dependence on key personnel; substantial competition; uncertainties of patent protection and litigation; dependence upon third parties; and risks related to failure to obtain FDA clearances or approvals and noncompliance with FDA regulations. Investors should read the risk factors set forth in our registration statement on Form S-1 and our periodic reports filed with the Securities and Exchange Commission. While the list of factors presented here is considered representative, no such list should be considered to be a complete statement of all potential risks and uncertainties. Unlisted factors may present significant additional obstacles to the realization of forward-looking statements. Forward-looking statements included herein are made as of the date hereof, and Hillstream does not undertake any obligation to update publicly such statements to reflect subsequent events or circumstances.

Investor Relations Contact:

Email: investorrelations@hillstreambio.com
www.hillstreambio.com

Source: Hillstream BioPharma Inc.

1. US Food and Drug Administration. Developing Products for Rare Diseases & Conditions. https://www.fda.gov/industry/developing-products-rare-diseases-conditions Accessed on 2/15/22
   

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FAQ

What was the recent development for Hillstream BioPharma regarding HSB-1216?

Hillstream BioPharma announced that the FDA granted Orphan Drug Designation for HSB-1216, which targets uveal melanoma.

Why is the Orphan Drug Designation significant for HSB-1216?

It highlights the significant unmet medical need in treating uveal melanoma, a rare and aggressive cancer.

What are the future plans for HSB-1216 after receiving Orphan Drug Designation?

Hillstream plans to progress toward IND filing and to initiate clinical trials for HSB-1216 in solid tumor patients.

What is the prognosis for patients with uveal melanoma?

Approximately 50% of patients with uveal melanoma develop metastatic disease, contributing to a poor prognosis.