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Mustang Bio gains FDA Orphan Drug Status for glioma treatment

Published 11/07/2024, 08:36 AM
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WORCESTER, Mass. - Mustang Bio , Inc. (NASDAQ:MBIO), a clinical-stage biopharmaceutical company, has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for its MB-108, a herpes simplex virus type 1 (HSV-1) oncolytic virus, intended for the treatment of malignant glioma, including recurrent glioblastoma (GBM) and high-grade astrocytomas.

Orphan Drug Designation is awarded to treatments for rare diseases affecting fewer than 200,000 people in the U.S., offering incentives like tax credits for clinical trial costs, prescription drug user fee waivers, and potentially seven years of market exclusivity upon approval.

Mustang Bio's President and CEO, Manuel Litchman, M.D., expressed the significance of this designation, emphasizing the company's commitment to advancing MB-108 as a potential treatment option for patients with malignant glioma. The company also plans to request Orphan Drug Designation for MB-101, its IL13Rα2-targeted CAR-T cell therapy, in the treatment of malignant gliomas.

Preclinical data presented at the 2022 American Association for Cancer Research (AACR) Annual Meeting supported the combination of MB-108 and MB-101 to potentially enhance clinical outcomes for recurrent GBM. The strategy involves using MB-108 to modify the tumor microenvironment and improve the efficacy of MB-101 CAR-T cell therapy. Early clinical trials have shown that the administration of these therapies is well tolerated in patients with recurrent GBM.

The ongoing Phase 1 clinical trials for MB-101 at City of Hope and MB-108 at The University of Alabama at Birmingham are continuing to enroll patients. The development of the MB-109 program, which combines both MB-101 and MB-108, is contingent on additional funding or strategic partnerships.

Mustang Bio, founded by Fortress Biotech, Inc. (NASDAQ:FBIO), focuses on developing cell therapies for hard-to-treat cancers. The company's stock is registered with the SEC, and it partners with top medical institutions to advance the development of CAR-T therapies.

This news article is based on a press release statement.

In other recent news, Fortress Biotech's rosacea treatment, Emrosi™, received FDA approval, marking a significant milestone in the company's dermatological offerings. The drug, developed in partnership with Dr. Reddy’s Laboratories, demonstrated significant superiority in reducing total inflammatory lesion count in two Phase 3 clinical trials. The company expects Emrosi™ to be available in the U.S. market by late first quarter or early second quarter of 2025.

Fortress Biotech recently secured approximately $8 million from stock sales and private placements and entered into a $50 million loan agreement with Oaktree Capital Management. The company reported a second-quarter revenue of $14.9 million, slightly surpassing consensus forecasts.

In response to these developments, Roth/MKM analysts have raised Fortress Biotech's price target from $10.00 to $13.00, maintaining a Buy rating. The company's subsidiary, Mustang Bio, secured around $4 million from warrant exercises and announced a stock offering and private placement expected to yield approximately $2.5 million. Mustang Bio also reported positive results from a Phase 1/2 clinical trial of MB-106, a CAR T-cell therapy for a rare blood cancer. These are recent developments for Fortress Biotech and Mustang Bio.

InvestingPro Insights

To provide additional context to Mustang Bio's recent FDA Orphan Drug Designation for MB-108, it's worth examining the financial health of its parent company, Fortress Biotech, Inc. (NASDAQ:FBIO). According to InvestingPro data, Fortress Biotech has a market capitalization of $48.51 million, reflecting its position as a small-cap biopharmaceutical company.

The company's revenue for the last twelve months as of Q2 2023 stood at $82.62 million, with a revenue growth of 31.68% over the same period. This growth is particularly noteworthy given the challenging environment for biotech companies. However, it's important to note that Fortress Biotech is currently operating at a loss, with a negative gross profit of $15.1 million and an operating income margin of -115.07%.

InvestingPro Tips highlight some key aspects of Fortress Biotech's financial situation. The company is "quickly burning through cash," which is not uncommon for biopharmaceutical companies investing heavily in research and development. Additionally, analysts anticipate a sales decline in the current year, which could impact the company's ability to fund ongoing clinical trials, including those for Mustang Bio's promising therapies.

It's also worth noting that Fortress Biotech's stock price movements are quite volatile, which is typical for small-cap biotech stocks, especially those with pipeline products in clinical stages. This volatility could be influenced by news such as the recent Orphan Drug Designation for MB-108.

For investors interested in a more comprehensive analysis, InvestingPro offers 8 additional tips for Fortress Biotech, providing a deeper understanding of the company's financial health and market position.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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