Core Viewpoint - Fortress Biotech and its subsidiary Helocyte are advancing the development of Triplex, a vaccine aimed at preventing and treating cytomegalovirus (CMV) in liver transplant recipients, with the first patient dosed in a Phase 2 clinical trial funded by a $9 million NIH/NIAID grant, expected to receive an additional $12 million over the next four years [1][2][3]. Company Overview - Fortress Biotech is focused on acquiring and advancing biopharmaceutical assets to enhance long-term shareholder value through product revenue, equity holdings, and dividend and royalty revenue, with seven marketed products and over 20 programs in development [6]. - Helocyte is a clinical-stage company developing immunotherapies for cancer and infectious diseases, including CMV and HIV, with a significant focus on transplant recipients who are at high risk for CMV complications [5]. Clinical Trial Details - The Phase 2 trial, named "CMV vaccine in Orthotopic Liver Transplant" (COLT), involves up to 416 CMV seronegative liver transplant recipients to evaluate the safety and efficacy of two doses of Triplex [2]. - The primary objective is to assess the impact of pre-transplant Triplex vaccination on the duration of CMV antiviral therapy within the first 100 days post-transplant [2]. Triplex Vaccine Information - Triplex is a recombinant Modified Vaccinia Ankara viral vector vaccine designed to induce a robust T cell response to CMV, with previous studies indicating it is safe, well-tolerated, and highly immunogenic [4]. - The vaccine is currently involved in multiple ongoing clinical trials for various transplant indications and HIV, with plans for additional studies, including a Phase 2 trial for kidney transplant recipients [4]. Market Context - CMV poses significant risks in organ transplantation, with over 46,000 organ transplants performed in the U.S. in 2023, primarily kidney and liver transplants, highlighting the unmet medical need for effective therapies [5].

WORCESTER, Mass., May 02, 2024 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. ("Mustang" or the "Company") (NASDAQ:MBIO), a clinical-stage biopharmaceutical company focused on translating today's medical breakthroughs in cell and gene therapies into potential cures for difficult-to-treat cancers, today announced the closing of its previously announced public offering for the purchase and sale of an aggregate of 16,877,638 shares of its common stock (or common stock equivalents in lieu thereof), Series A-1 warrants t ...

WORCESTER, Mass., April 29, 2024 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. ("Mustang" or the "Company") (NASDAQ:MBIO), a clinical-stage biopharmaceutical company focused on translating today's medical breakthroughs in cell and gene therapies into potential cures for difficult-to-treat cancers and rare genetic diseases, today announced the pricing of a public offering of an aggregate of 16,877,638 shares of its common stock (or common stock equivalents in lieu thereof), Series A-1 warrants to purchase up to 16,8 ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the Fiscal Year Ended December 31, 2023 Delaware 20-5157386 (State or Other Jurisdiction of Incorporation or Organization) (I.R.S. Employer Identification No.) 1111 Kane Concourse Suite 301 Bay Harbor Islands, FL 33154 33154 (Address of Principal Executive Offices) (Zip Code) Registrant's telephone number, including are ...

[Executive Summary](index=1&type=section&id=Executive%20Summary) Fortress Biotech achieved record $84.5 million net revenue in 2023, anticipating up to four regulatory decisions, including DFD-29's NDA acceptance, by November 2024 [2023 Performance and Strategic Outlook](index=1&type=section&id=2023%20Performance%20and%20Strategic%20Outlook) Fortress Biotech achieved record $84.5 million net revenue in 2023, with up to four regulatory decisions, including DFD-29's NDA acceptance, expected by November 2024 - Record consolidated net revenue of **$84.5 million** for full-year 2023[1](index=1&type=chunk)[2](index=2&type=chunk) - Up to **four regulatory decisions** (NDAs/BLAs) expected in the next 18 months[1](index=1&type=chunk)[2](index=2&type=chunk) - FDA accepted NDA filing for DFD-29 (rosacea treatment) with PDUFA goal date of **November 4, 2024**[1](index=1&type=chunk)[2](index=2&type=chunk) [Company Information](index=3&type=section&id=Company%20Information) Fortress Biotech is a biopharmaceutical company focused on acquiring and advancing assets across oncology, rare diseases, and gene therapy [About Fortress Biotech](index=3&type=section&id=About%20Fortress%20Biotech) Fortress Biotech acquires and advances assets to generate shareholder value, managing seven marketed products and over 25 development programs in oncology, rare diseases, and gene therapy - Core Business: Acquiring and advancing assets to generate product, equity, dividend, and royalty revenue for shareholders[8](index=8&type=chunk) - Portfolio Scope: **Seven marketed prescription products** and over **25 development programs** in oncology, rare diseases, and gene therapy[8](index=8&type=chunk) - Operational Strategy: Streamlined operating structure, leveraging biopharmaceutical expertise and network, with partnerships including AstraZeneca and City of Hope[8](index=8&type=chunk) [2023 and Recent Corporate Highlights](index=1&type=section&id=2023%20and%20Recent%20Corporate%20Highlights) This section details Fortress Biotech's recent regulatory milestones, corporate developments, and financial activities in 2023 and early 2024 [Regulatory Milestones and Updates](index=1&type=section&id=Regulatory%20Milestones%20and%20Updates) Fortress submitted DFD-29's NDA for rosacea in Jan 2024, accepted by FDA in March with a PDUFA goal date of November 4, 2024 - DFD-29 NDA Submission & Acceptance: Submitted **Jan 2024**, accepted **March 2024**, for rosacea treatment[3](index=3&type=chunk) - PDUFA Goal Date: **November 4, 2024**, for DFD-29[3](index=3&type=chunk) - Market Potential: DFD-29 could be the only oral, systemic therapy for both inflammatory lesions and erythema of rosacea[3](index=3&type=chunk) [General Corporate Developments](index=2&type=section&id=General%20Corporate) Key developments include a CRL for cosibelimab, anticipated CAEL-101 BLA in 2025, CUTX-101 asset transfer, Qbrexza licensing, and 2023 fundraising - Cosibelimab BLA: FDA issued CRL in **Dec 2023** due to third-party manufacturing findings; resubmission expected for **2024 marketing approval**[6](index=6&type=chunk) - CAEL-101 (AstraZeneca): BLA submission anticipated in **2025**, with potential for approximately **$148 million** in milestone payments to Fortress[6](index=6&type=chunk) - CUTX-101: Asset transfer to Sentynl completed in **Dec 2023**; Cyprium Therapeutics retains **100% ownership** of any FDA priority review voucher[6](index=6&type=chunk) - MB-109: FDA accepted Investigational New Drug application for a Phase 1 clinical trial for recurrent GBM and high-grade astrocytoma[6](index=6&type=chunk) - Journey Medical - Qbrexza Licensing: Entered exclusive license agreement with Maruho for hyperhidrosis treatment in South Korea and other Asian territories, receiving a **$19 million nonrefundable upfront payment**[6](index=6&type=chunk) Journey Medical Net Revenues (YoY) | Metric | 2023 (Millions) | 2022 (Millions) | Change (%) | | :-------------------- | :-------------- | :-------------- | :--------- | | Total Net Revenues | $79.2 | $73.7 | +7% | | Total Product Net Revenues | $59.7 | $71.0 | -15.9% | - Fortress Fundraising: Raised approximately **$34.9 million** in gross proceeds through registered direct offerings and a public offering in **2023 and Jan 2024**[6](index=6&type=chunk) - Reverse Stock Split: Effected a **1-for-15 reverse stock split** in **October 2023** to meet Nasdaq's minimum bid price requirement[6](index=6&type=chunk) - 4DMT Asset Purchase: Agreement for short-form human complement factor H asset, with potential payments up to **~$140 million** in late-stage development, regulatory, and sales milestones, plus single-digit royalties[6](index=6&type=chunk) [Financial Review](index=3&type=section&id=Financial%20Review) This section provides a detailed analysis of Fortress Biotech's financial performance, including revenues, expenses, and balance sheet changes for 2023 [Summary of Key Financials](index=3&type=section&id=Financial%20Results) Fortress Biotech reported $84.5 million net revenue in 2023, an 11.6% increase, with reduced operating expenses and an improved net loss of $(68.7) million Consolidated Cash, Cash Equivalents and Restricted Cash | Date | Amount (Millions) | Change (YoY) | Change (QoQ) | | :---------------- | :---------------- | :----------- | :----------- | | Dec 31, 2023 | $83.4 | -$97.6M | +$8.7M | | Sep 30, 2023 | $74.7 | - | - | | Dec 31, 2022 | $181.0 | - | - | Consolidated Net Revenue (YoY) | Metric | 2023 (Millions) | 2022 (Millions) | Change (%) | | :-------------------- | :-------------- | :-------------- | :--------- | | Total Net Revenue | $84.5 | $75.7 | +11.6% | | Net Revenue from Marketed Dermatology Products | $59.7 | $71.0 | -15.9% | Consolidated Operating Expenses (YoY) | Metric | 2023 (Millions) | 2022 (Millions) | Change (%) | | :-------------------- | :-------------- | :-------------- | :--------- | | R&D Expenses (incl. licenses) | $106.1 | $134.9 | -21.3% | | SG&A Costs | $94.1 | $113.7 | -17.2% | Consolidated Net Loss Attributable to Common Stockholders (YoY) | Metric | 2023 (Millions) | 2022 (Millions) | Change (%) | | :-------------------- | :-------------- | :-------------- | :--------- | | Net Loss | $(68.7) | $(94.6) | +27.4% | | Net Loss Per Share | $(8.47) | $(15.97) | +47.0% | - Subsequent Fundraising (Jan 2024): Fortress raised **~$11.0 million**, Checkpoint **~$14.0 million**, and Avenue **~$5.0 million** in gross proceeds[9](index=9&type=chunk) [Consolidated Balance Sheets](index=5&type=section&id=Consolidated%20Balance%20Sheets) Total assets decreased to $167.5 million as of December 31, 2023, driven by reduced cash, with total stockholders' equity declining to $1.6 million Balance Sheet Highlights (Dec 31, 2023 vs Dec 31, 2022) | Metric | 2023 (Thousands) | 2022 (Thousands) | Change (Thousands) | Change (%) | | :-------------------------------- | :--------------- | :--------------- | :----------------- | :--------- | | Cash and cash equivalents | $80,927 | $178,266 | $(97,339) | -54.6% | | Total current assets | $117,022 | $230,432 | $(113,410) | -49.2% | | Total assets | $167,526 | $294,301 | $(126,775) | -43.1% | | Total current liabilities | $84,908 | $127,715 | $(42,807) | -33.5% | | Total liabilities | $165,939 | $244,276 | $(78,337) | -32.1% | | Total stockholders' equity (deficit) | $1,587 | $50,025 | $(48,438) | -96.8% | [Consolidated Statements of Operations](index=6&type=section&id=Consolidated%20Statements%20of%20Operations) Net revenue increased 11.6% to $84.5 million in 2023, driven by collaboration and other revenues, with operating expenses decreasing by 18.8% Revenue Breakdown (2023 vs 2022) | Revenue Type | 2023 (Thousands) | 2022 (Thousands) | Change (Thousands) | Change (%) | | :-------------------- | :--------------- | :--------------- | :----------------- | :--------- | | Product revenue, net | $59,662 | $70,995 | $(11,333) | -15.9% | | Collaboration revenue | $5,229 | $1,882 | $3,347 | +177.8% | | Other revenue | $19,519 | $2,674 | $16,845 | +630.0% | | **Net revenue** | **$84,513** | **$75,743** | **$8,770** | **+11.6%** | Operating Expenses (2023 vs 2022) | Expense Type | 2023 (Thousands) | 2022 (Thousands) | Change (Thousands) | Change (%) | | :-------------------------------- | :--------------- | :--------------- | :----------------- | :--------- | | Cost of goods sold - product revenue | $26,660 | $30,775 | $(4,115) | -13.4% | | Research and development | $101,747 | $134,199 | $(32,452) | -24.2% | | Research and development - licenses acquired | $4,324 | $677 | $3,647 | +538.7% | | Selling, general and administrative | $94,124 | $113,656 | $(19,532) | -17.2% | | **Total operating expenses** | **$226,855** | **$279,307** | **$(52,452)** | **-18.8%** | Net Loss Attributable to Common Stockholders (2023 vs 2022) | Metric | 2023 (Thousands) | 2022 (Thousands) | Change (Thousands) | Change (%) | | :-------------------------------- | :--------------- | :--------------- | :----------------- | :--------- | | Net loss attributable to common stockholders | $(68,669) | $(94,607) | $25,938 | +27.4% | | Net loss per common share - basic and diluted | $(8.47) | $(15.97) | $7.50 | +47.0% | | Weighted average common shares outstanding | 8,110,906 | 5,924,967 | 2,185,939 | +36.9% | [Legal and Contact Information](index=4&type=section&id=Legal%20and%20Contact%20Information) This section outlines the company's forward-looking statement disclaimer and provides essential contact information for investor and media relations [Forward-Looking Statements](index=4&type=section&id=Forward-Looking%20Statements) All non-historical statements are forward-looking, subject to risks in growth, financing, development, and regulatory approval, with no obligation to update - Nature of Statements: Non-historical statements are forward-looking, subject to risks and uncertainties[10](index=10&type=chunk) - Risk Factors: Include growth strategy, financing, product development, regulatory approval, commercialization, manufacturing, intellectual property, and competition[10](index=10&type=chunk) - Disclaimer: Company disclaims obligation to update forward-looking statements, except as required by law[10](index=10&type=chunk) [Company Contact Information](index=4&type=section&id=Company%20Contact%3A) This section provides contact details for Fortress Biotech's investor relations and media relations - Company Contact: Jaclyn Jaffe, Fortress Biotech, Inc., **(781) 652-4500**, ir@fortressbiotech.com[11](index=11&type=chunk) - Media Relations Contact: Tony Plohoros, 6 Degrees, **(908) 591-2839**, tplohoros@6degreespr.com[11](index=11&type=chunk)

MIAMI, March 12, 2024 (GLOBE NEWSWIRE) -- Fortress Biotech, Inc. (NASDAQ:FBIO) ("Fortress"), an innovative biopharmaceutical company focused on efficiently acquiring, developing and commercializing or monetizing promising therapeutic products and product candidates, today announced that Lindsay A. Rosenwald, M.D., Chairman, President and Chief Executive Officer, will participate in a fireside chat at the 36th Annual ROTH Conference on Monday, March 18, 2024, at 2:00 p.m. PT in Dana Point, California. The Co ...

WORCESTER, Mass., March 11, 2024 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. ("Mustang" or the "Company") (NASDAQ:MBIO), a clinical-stage biopharmaceutical company focused on translating today's medical breakthroughs in cell and gene therapies into potential cures for difficult-to-treat cancers and rare genetic diseases, today announced financial results and recent corporate highlights for the full-year ended December 31, 2023. "2023 was an exciting year for Mustang filled with many highlights, including compelli ...

MB-101 was well-tolerated and 50% of patients achieved stable disease or better with two partial responses and two complete responses lasting 7.5 and 66+ months, respectively ~70% improvement in median overall survival compared to expected survival rate in cohort with dual intratumoral (ICT)/ intraventricular (ICV) delivery and an optimized manufacturing process This is the largest reported trial to date of CAR-T therapy for solid tumors WORCESTER, Mass., March 07, 2024 (GLOBE NEWSWIRE) -- Mustang Bio, Inc ...

LOS ANGELES--(BUSINESS WIRE)--A pioneering Phase 1 CAR T cell therapy trial for the treatment of glioblastoma at City of Hope, one of the largest cancer treatment and research organizations in the United States, demonstrates promising clinical activity against incurable brain tumors, according to research published today in Nature Medicine. The study, which is the largest reported trial to date of CAR T therapy for solid tumors, evaluated CAR T cells engineered to target the tumor-associated antigen inte ...

Encouraging preclinical studies demonstrate potential to combine AAV-ATP7A gene therapy with CUTX-101, which could be the first FDA-approved treatment for Menkes disease Cyprium Therapeutics, a majority-owned subsidiary of Fortress Biotech, is developing AAV-ATP7A gene therapy to be used in conjunction with CUTX-101 for the treatment of Menkes disease MIAMI, March 04, 2024 (GLOBE NEWSWIRE) -- Cyprium Therapeutics, Inc. ("Cyprium"), a majority-owned subsidiary of Fortress Biotech, Inc. (NASDAQ:FBIO) ("Fortre ...