Cyprium Therapeutics, Inc.

MIAMI, July 05, 2024 (GLOBE NEWSWIRE) -- Fortress Biotech, Inc. (Nasdaq: FBIO; FBIOP) (“Fortress”), an innovative biopharmaceutical company focused on acquiring and advancing assets to enhance long-term value for shareholders through product revenue, equity holdings and dividend and royalty revenue, today announced that its Board of Directors has paused the payment of dividends on the Company’s 9.375% Series A Cumulative Redeemable Perpetual Preferred Stock (the “Series A Preferred Stock”) until further notice. In accordance with the terms of the Series A Preferred Stock, dividends on the Series A Preferred Stock will continue to accrue and cumulate until such dividends are authorized or declared. The Company will begin accruing the dividend as of July 1, 2024, and no dividend payment will be issued on July 31, 2024. The Company believes pausing the dividend is in the best interest of the Company and its stakeholders to maintain financial flexibility ahead of potentially significant inflection points. The pausing of these dividends will defer approximately $0.7 million in cash dividend payments each month. The Board intends to revisit its decision regarding the monthly dividend regularly and will assess the profitability and cash flow of the Company to determine whether and when the suspension should be lifted. The Series A Preferred Stock trades on the Nasdaq Capital Market under the “FBIOP” stock ticker symbol. Lindsay A. Rosenwald, M.D., Fortress’ Chairman, President and Chief Executive Officer, said, “Across our portfolio, we could receive up to three regulatory approvals on NDAs and BLAs in the next 12 months for DFD-29, cosibelimab and CUTX-101, and potentially a fourth BLA filing as early as 2025 for CAEL-101. Based on its public statements, AstraZeneca has estimated that it expects the FDA to accept its BLA submission of CAEL-101 to treat AL amyloidosis for review as early as 2025, which could lead to approval and commercial milestone payments to Fortress. Additionally, Cyprium Therapeutics, our subsidiary company that developed CUTX-101, will retain 100% ownership over any priority review voucher that may be issued at NDA approval for CUTX-101. This is a pivotal time for the Company, and we are pausing the preferred dividend payments in order to maintain financial flexibility ahead of our multiple potential near-term milestones.” About Fortress Biotech Fortress Biotech, Inc. (“Fortress”) is an innovative biopharmaceutical company focused on acquiring and advancing assets to enhance long-term value for shareholders through product revenue, equity holdings and dividend and royalty revenue. The company has seven marketed prescription pharmaceutical products and over 20 programs in development at Fortress, at its majority-owned and majority-controlled partners and subsidiaries and at partners and subsidiaries it founded and in which it holds significant minority ownership positions. Fortress’ portfolio is being commercialized and developed for various therapeutic areas including oncology, dermatology, and rare diseases. Fortress’ model is focused on leveraging its significant biopharmaceutical industry expertise and network to further expand and advance the company’s portfolio of product opportunities. Fortress has established partnerships with some of the world’s leading academic research institutions and biopharmaceutical companies to maximize each opportunity to its full potential, including AstraZeneca, City of Hope, Fred Hutchinson Cancer Center, Nationwide Children’s Hospital and Sentynl. For more information, visit www.fortressbiotech.com. Forward-Looking StatementsStatements in this press release that are not descriptions of historical facts are “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, as amended. The words “anticipates,” “believes,” “can,” “continue,” “could,” “estimates,” “expects,” “intends,” “may,” “might,” “plans,” “potential,” “predicts,” “should,” or “will” or the negative of these terms or other comparable terminology are generally intended to identify forward-looking statements. These forward-looking statements are based on management’s current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock price. Factors that could cause actual results to differ materially from those currently anticipated include risks relating to: our growth strategy, financing and strategic agreements and relationships; the ongoing UTRF litigation and our indemnification of Caelum in connection therewith; our need for substantial additional funds and uncertainties relating to financings; our ability to identify, acquire, close and integrate product candidates successfully and on a timely basis; our ability to attract, integrate and retain key personnel; the early stage of products under development; the results of research and development activities; uncertainties relating to preclinical and clinical testing; uncertainties related to the timing and likelihood of a BLA being submitted for CAEL-101; our ability to obtain regulatory approval for products under development, including for DFD-29, cosibelimab and CUTX-101, for which submissions are pending; our ability to successfully commercialize products for which we receive regulatory approval; uncertainties related to the granting of any priority review voucher for CUTX-101; our ability to secure and maintain third-party manufacturing, marketing and distribution of our and our partner companies’ products and product candidates; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as may be required by law, and we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995. The information contained herein is intended to be reviewed in its totality, and any stipulations, conditions or provisos that apply to a given piece of information in one part of this press release should be read as applying mutatis mutandis to every other instance of such information appearing herein. Company Contact:Jaclyn JaffeFortress Biotech, Inc.(781) 652-4500ir@fortressbiotech.com Media Relations Contact:Tony Plohoros6 Degrees(908) 591-2839tplohoros@6degreespr.com

Record consolidated net revenue of $84.5 million for full-year 2023 Fortress may receive up to four regulatory decisions on NDAs and BLAs in the next 18 months FDA accepted New Drug Application filing for DFD-29 to treat inflammatory lesions and erythema of rosacea in adults; PDUFA goal date of November 4, 2024 MIAMI, March 28, 2024 (GLOBE NEWSWIRE) -- Fortress Biotech, Inc. (Nasdaq: FBIO) (“Fortress”), an innovative biopharmaceutical company focused on acquiring and advancing assets to enhance long-term value for shareholders through product revenue, equity holdings and dividend and royalty revenue, today announced financial results and recent corporate highlights for the full-year ended December 31, 2023. Lindsay A. Rosenwald, M.D., Fortress’ Chairman, President and Chief Executive Officer, said, “In 2023, we built a significant amount of momentum to position our Company to achieve multiple milestones in 2024. We also generated record consolidated net revenues of $84.5 million in 2023, the majority of which came from the sales and milestone payments from our dermatology and rare disease businesses.” Dr. Rosenwald continued, “We are pleased that the U.S. Food and Drug Administration (“FDA”) accepted the New Drug Application (“NDA”) filing for DFD-29 earlier this month and look forward to the Prescription Drug User Fee Act (“PDUFA”) goal date of November 4, 2024. Across our portfolio, we could receive up to four NDA and Biologics License Application (“BLA”) regulatory approvals over the next 18 months, while we continue to advance our 25 development stage programs in 2024.” 2023 and Recent Corporate Highlights1: Regulatory Milestones and Updates In January 2024, we submitted an NDA to the FDA seeking approval for DFD-29 (Minocycline Hydrochloride Modified Release Capsules, 40 mg) for the treatment of inflammatory lesions and erythema of rosacea in adults. In March 2024, the FDA accepted the NDA and has set a PDUFA goal date of November 4, 2024. If approved, DFD-29 has the potential to become the only oral, systemic therapy to address both inflammatory lesions and erythema (redness) from rosacea, as demonstrated in clinical trials. DFD-29 is currently in development at our partner company, Journey Medical Corporation (Nasdaq: DERM) (“Journey Medical”). We submitted a BLA to the FDA for cosibelimab, our investigational anti-PD-L1 antibody, as a treatment for patients with metastatic or locally advanced cutaneous squamous cell carcinoma who are not candidates for curative surgery or radiation, in January 2023. In December 2023, the FDA issued a complete response letter (“CRL”) for the cosibelimab BLA. The CRL only cited findings that arose during a multi-sponsor inspection of a third-party contract manufacturing organization as approvability issues to address in a resubmission. The CRL did not state any concerns about the clinical data package, safety or labeling for the approvability of cosibelimab. We believe we can address the feedback in a resubmission to enable marketing approval in 2024. We also secured additional U.S. patent protection for cosibelimab through at least May 2038. Cosibelimab is currently in development at our partner company, Checkpoint Therapeutics, Inc. (Nasdaq: CKPT) (“Checkpoint”). Based on its public statements, AstraZeneca plc (“AstraZeneca”) has estimated that it expects the FDA to accept its BLA submission of CAEL-101 (anselamimab) to treat AL amyloidosis for review in 2025. In 2021, AstraZeneca acquired Caelum Biosciences, Inc. (founded by Fortress) for an upfront payment of approximately $150 million paid to Caelum shareholders, of which approximately $56.9 million was paid to Fortress. The agreement also provides for additional potential payments to Caelum shareholders, including approximately $148 million to Fortress, payable upon the achievement of regulatory and commercial milestones. In December 2023, we completed the asset transfer of CUTX-101 (copper histidinate for Menkes disease) to Sentynl, a wholly owned subsidiary of Zydus Lifesciences Ltd. The CUTX-101 rolling NDA submission is ongoing and is expected to be completed by Sentynl in 2024. Cyprium Therapeutics, Inc. (“Cyprium”), our subsidiary company that developed CUTX-101, will retain 100% ownership over any FDA priority review voucher that may be issued at NDA approval for CUTX-101. In October 2023, we announced that the FDA accepted our Investigational New Drug application to initiate a Phase 1 open-label, multicenter clinical trial to assess the safety, tolerability and efficacy of MB-109, a novel combination of MB-101 (IL13Rα2‐targeted CAR-T cell therapy) and MB-108 (HSV-1 oncolytic virus), for the treatment of IL13Rα2+ recurrent GBM and high-grade astrocytoma. MB-109 is currently in development at our partner company, Mustang Bio, Inc. (Nasdaq: MBIO) (“Mustang Bio”). Commercial Product Updates In September 2023, our partner company, Journey Medical, entered into an exclusive license agreement with Maruho Co., Ltd. (“Maruho”), a Japanese company specializing in dermatology as well as Journey Medical’s exclusive licensing partner that developed and is commercializing Qbrexza® (Rapifort®) in Japan. Under the terms of a new license agreement, Journey Medical received a $19 million nonrefundable upfront payment and granted Maruho an exclusive license to develop and commercialize Qbrexza (glycopyrronium tosylate hydrate) for the treatment of hyperhidrosis in South Korea, Taiwan, Hong Kong, Macau, Thailand, Indonesia, Malaysia, Philippines, Singapore, Vietnam, Brunei, Cambodia, Myanmar and Laos (the “Territory”). Maruho is responsible for all development and commercialization costs for the program throughout the Territory. Journey Medical’s total net revenues for the year ended December 31, 2023, were $79.2 million, an increase of $5.5 million, or 7%, compared to total net revenues of $73.7 million for 2022. Journey Medical’s total product net revenues were $59.7 million for the year ended December 31, 2023, compared to total product net revenues of $71.0 million for the year ended December 31, 2022. General Corporate: Throughout 2023 and in January 2024, Fortress raised total gross proceeds of approximately $34.9 million in registered direct offerings priced at-the-market under Nasdaq rules and in a public offering. In October 2023, Fortress effected a 1-for-15 reverse stock split of its issued and outstanding common stock to bring the Company into compliance with Nasdaq’s minimum bid price requirement for continued listing. In April 2023, we announced the execution of an asset purchase agreement for 4D Molecular Therapeutics (“4DMT”) to acquire proprietary rights to our short-form human complement factor H asset for the treatment of complement-mediated diseases. Under the terms of the agreement, 4DMT will make cash payments totaling up to ~$140 million in potential late-stage development, regulatory and sales milestones. A range of single-digit royalties on net sales are also payable. Our short-form human complement factor H asset was in development at our subsidiary company, Aevitas Therapeutics, Inc. (“Aevitas”), prior to the asset purchase agreement with 4DMT. Financial Results: As of December 31, 2023, Fortress’ consolidated cash, cash equivalents and restricted cash totaled $83.4 million, compared to $74.7 million as of September 30, 2023, and $181.0 million as of December 31, 2022, an increase of $8.7 million for the fourth quarter and a decrease of $97.6 million for the full year. Fortress’ consolidated cash, cash equivalents and restricted cash, totaling $83.4 million as of December 31, 2023, includes $42.2 million attributable to Fortress and private subsidiaries, $1.8 million attributable to Avenue, $4.9 million attributable to Checkpoint, $7.0 million attributable to Mustang Bio and $27.4 million attributable to Journey Medical. Subsequent to the end of the fourth quarter, in January 2024, Fortress raised approximately $11.0 million in gross proceeds in a registered direct offering, Checkpoint raised approximately $14.0 million in gross proceeds in a registered direct offering and Avenue raised approximately $5.0 million in gross proceeds from warrant exercise transactions. Fortress’ consolidated net revenue totaled $84.5 million for the full year ended December 31, 2023, which included $59.7 million in net revenue generated from our marketed dermatology products. This compares to consolidated net revenue totaling $75.7 million for the full year ended 2022, which included $71.0 million in net revenue generated from our marketed dermatology products. Consolidated research and development expenses including license acquisitions totaled $106.1 million for the full year ended December 31, 2023, compared to $134.9 million for the full year ended December 31, 2022. Consolidated selling, general and administrative costs were $94.1 million for the full year ended December 31, 2023, compared to $113.7 million for the full year ended December 31, 2022. Consolidated net loss attributable to common stockholders was $(68.7) million, or $(8.47) per share, for the full year ended December 31, 2023, compared to net loss attributable to common stockholders of $(94.6) million, or $(15.97) per share for the full year ended December 31, 2022. About Fortress Biotech Fortress Biotech, Inc. (“Fortress”) is an innovative biopharmaceutical company focused on acquiring and advancing assets to enhance long-term value for shareholders through product revenue, equity holdings and dividend and royalty revenue. The company has seven marketed prescription pharmaceutical products and over 25 programs in development at Fortress, at its majority-owned and majority-controlled partners and subsidiaries and at partners and subsidiaries it founded and in which it holds significant minority ownership positions. Such product candidates span six large-market areas, including oncology, rare diseases and gene therapy, which allow it to create value for shareholders. Fortress advances its diversified pipeline through a streamlined operating structure that fosters efficient drug development. The Fortress model is focused on leveraging its significant biopharmaceutical industry expertise and network to further expand the company’s portfolio of product opportunities. Fortress has established partnerships with some of the world’s leading academic research institutions and biopharmaceutical companies to maximize each opportunity to its full potential, including AstraZeneca, City of Hope, Fred Hutchinson Cancer Center, St. Jude Children’s Research Hospital, Nationwide Children’s Hospital and Sentynl. For more information, visit . Forward-Looking Statements Statements in this press release that are not descriptions of historical facts are “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, as amended. The words “anticipates,” “believes,” “can,” “continue,” “could,” “estimates,” “expects,” “intends,” “may,” “might,” “plans,” “potential,” “predicts,” “should,” or “will” or the negative of these terms or other comparable terminology are generally intended to identify forward-looking statements. These forward-looking statements are based on management’s current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock price. Factors that could cause actual results to differ materially from those currently anticipated include risks relating to: our growth strategy, financing and strategic agreements and relationships; our need for substantial additional funds and uncertainties relating to financings; our ability to identify, acquire, close and integrate product candidates successfully and on a timely basis; our ability to attract, integrate and retain key personnel; the early stage of products under development; the results of research and development activities; uncertainties relating to preclinical and clinical testing; our ability to obtain regulatory approval for products under development; our ability to successfully commercialize products for which we receive regulatory approval; our ability to secure and maintain third-party manufacturing, marketing and distribution of our and our partner companies’ products and product candidates; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as may be required by law, and we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995. The information contained herein is intended to be reviewed in its totality, and any stipulations, conditions or provisos that apply to a given piece of information in one part of this press release should be read as applying mutatis mutandis to every other instance of such information appearing herein. Company Contact: Jaclyn Jaffe Fortress Biotech, Inc. (781) 652-4500 ir@fortressbiotech.com Media Relations Contact: Tony Plohoros 6 Degrees (908) 591-2839 tplohoros@6degreespr.com FORTRESS BIOTECH, INC. AND SUBSIDIARIES Consolidated Balance Sheets ($ in thousands except for share and per share amounts) December 31, 2023 2022 ASSETS Current assets Cash and cash equivalents $ 80,927 $ 178,266 Accounts receivable, net 15,222 28,208 Inventory 10,206 14,159 Other receivables - related party 167 138 Prepaid expenses and other current assets 10,500 9,661 Total current assets 117,022 230,432 Property, plant and equipment, net 6,505 13,020 Operating lease right-of-use asset, net 16,990 19,991 Restricted cash 2,438 2,688 Intangible asset, net 20,287 27,197 Other assets 4,284 973 Total assets $ 167,526 $ 294,301 LIABILITIES AND STOCKHOLDERS’ EQUITY (DEFICIT) Current liabilities Accounts payable and accrued expenses $ 73,562 $ 97,446 Income taxes payable 843 722 Common stock warrant liabilities 886 13,869 Operating lease liabilities, short-term 2,523 2,447 Partner company convertible preferred shares, short-term, net 3,931 2,052 Partner company line of credit — 2,948 Partner company installment payments - licenses, short-term, net 3,000 7,235 Other short-term liabilities 163 996 Total current liabilities 84,908 127,715 Notes payable, long-term, net 60,856 91,730 Operating lease liabilities, long-term 18,282 21,572 Partner company installment payments - licenses, long-term, net — 1,412 Other long-term liabilities 1,893 1,847 Total liabilities 165,939 244,276 Commitments and contingencies Stockholders’ equity (deficit) Cumulative redeemable perpetual preferred stock, $0.001 par value, 15,000,000 authorized, 5,000,000 designated Series A shares, 3,427,138 shares issued and outstanding as of December 31, 2023 and December 31, 2022, respectively, liquidation value of $25.00 per share 3 3 Common stock, $0.001 par value, 200,000,000 shares authorized, 15,093,053 and 7,366,283 shares issued and outstanding as of December 31, 2023 and December 31, 2022, respectively 15 7 Additional paid-in-capital 717,396 675,944 Accumulated deficit (694,870 ) (634,233 ) Total stockholders' equity attributed to the Company 22,544 41,721 Non-controlling interests (20,957 ) 8,304 Total stockholders' equity (deficit) 1,587 50,025 Total liabilities and stockholders' equity (deficit) $ 167,526 $ 294,301 FORTRESS BIOTECH, INC. AND SUBSIDIARIES Consolidated Statements of Operations ($ in thousands except for share and per share amounts) Year Ended December 31, 2023 2022 Revenue Product revenue, net $ 59,662 $ 70,995 Collaboration revenue 5,229 1,882 Revenue - related party 103 192 Other revenue 19,519 2,674 Net revenue 84,513 75,743 Operating expenses Cost of goods sold - product revenue 26,660 30,775 Research and development 101,747 134,199 Research and development - licenses acquired 4,324 677 Selling, general and administrative 94,124 113,656 Total operating expenses 226,855 279,307 Loss from operations (142,342 ) (203,564 ) Other income (expense) Interest income 3,003 1,398 Interest expense and financing fee (15,315 ) (13,642 ) Change in fair value of warrant liabilities 4,424 1,129 Other income (expense) (3,403 ) 1,215 Total other income (expense) (11,291 ) (9,900 ) Loss before income tax expense (153,633 ) (213,464 ) Income tax expense 521 449 Net loss (154,154 ) (213,913 ) Net loss attributable to non-controlling interests 93,517 127,338 Net loss attributable to Fortress (60,637 ) $ (86,575 ) Preferred A dividends declared and paid (8,032 ) (8,032 ) Net loss attributable to common stockholders $ (68,669 ) (94,607 ) Net loss per common share attributable to common stockholders - basic and diluted $ (8.47 ) $ (15.97 ) Weighted average common shares outstanding - basic and diluted 8,110,906 5,924,967 _________________________________ 1 The development programs depicted in this press release include product candidates in development at Fortress, at Fortress’ private subsidiaries (referred to herein as “subsidiaries”), at Fortress’ public subsidiaries (referred to herein as “partner companies”) and at entities with whom one of the foregoing parties has a significant business relationship, such as an exclusive license or an ongoing product-related payment obligation (such entities referred to herein as “partners”). The words “we”, “us” and “our” may refer to Fortress individually, to one or more of our subsidiaries and/or partner companies, or to all such entities as a group, as dictated by context.

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) (“Fortress”), today announced that the National Institute of Neurological Disorders and Stroke (“NINDS”) of the National Institutes of Health (“NIH”) has awarded a three-year grant totaling approximately $4.1 million to the Research Institute at Nationwide Children’s Hospital and Principal Investigator, Stephen G. Kaler, M.D., M.P.H., to fund completion of preclinical studies, manufacturing and preparation of an Investigational New Drug Application for a first-in-human clinical trial to advance adeno-associated virus (“AAV”)-ATP7A gene therapy, also known as AAV-ATP7A, for the treatment of Menkes disease. Often lethal if untreated, Menkes disease is an X-linked recessive disorder of copper metabolism caused by mutations in ATP7A, an evolutionarily conserved copper-transporting ATPase. The minimum birth prevalence for Menkes disease is believed to be 1 in 34,810 live male births, and potentially as high as 1 in 8,664 live male births, based on genome-driven ascertainment. In 2017, Cyprium entered into a worldwide, exclusive license agreement with the Eunice Kennedy Shriver National Institute of Child Health and Human Development (“NICHD”) to develop and commercialize AAV-ATP7A gene therapy to deliver working copies of the copper transporter defective in patients with Menkes disease, and to be used in combination with CUTX-101 (Copper Histidinate), which is being developed by Sentynl Therapeutics, Inc. (“Sentynl”). AAV-ATP7A was previously granted Orphan Drug Designation by the U.S. Food and Drug Administration (“FDA”). “By combining CUTX-101 with working copies of ATP7A delivered by AAV, we hope to enhance clinical outcomes in Menkes disease, a fatal rare pediatric disease. This funding allows us to further evaluate the preclinical safety, tolerability and dosing of AAV9-codon-optimized, reduced-size ATP7A, which we propose to administer in a first-in-human clinical trial for Menkes disease. Advances in viral gene therapy and newborn screening make it feasible to envision changing the natural history of this difficult illness, in combination with CUTX-101, potentially the first FDA-approved treatment for Menkes disease,” said Dr. Kaler, a physician-scientist in the Center for Gene Therapy in the Abigail Wexner Research Institute at Nationwide Children’s Hospital, Principal Investigator of the preclinical and clinical studies, and professor of Pediatrics and Genetics at The Ohio State University College of Medicine. Preclinical studies have demonstrated a synergistic effect of AAV-ATP7A and CUTX-101 in a reliable mouse model of Menkes disease. In early studies, cerebrospinal fluid (“CSF”)-directed AAV gene therapy rescued 22-53% of mice with a mutation in the human Menkes disease homolog (mottled-brindled) when combined with CSF or subcutaneous copper. In addition, mutant mice treated with CSF-directed AAV9- reduced-size ATP7A in combination with CUTX-101 showed higher brain copper levels, normalized brain neurochemicals, improvement of brain mitochondrial abnormalities, and near-normal growth and neurobehavioral outcomes. More recently, based on successful intravenous AAV9 gene therapy in mice and humans with spinal muscular atrophy at the Center for Gene Therapy, Nationwide Children’s Hospital, the Kaler Laboratory evaluated intravenous administration of AAV9-codon-optimized, reduced-size ATP7A combined with subcutaneous administration of CUTX-101 in Menkes disease mouse models. This regimen led to 95% long-term rescue of mottled-brindled mutant mice (n=19/20), using an AAV9 dose of 2.6 x 1013 vg/kg body weight, the most successful long-term rescue of this mouse model to date. Lindsay A. Rosenwald, M.D., Executive Chairman, President and Chief Executive Officer of Fortress, stated, “We are honored to have the NIH’s support, and Dr. Kaler’s and Nationwide Children’s leadership, to progress the development of AAV-ATP7A gene therapy, to help provide a more effective therapeutic option to patients with Menkes disease. As we continue the research and development of AAV-ATP7A in combination with CUTX-101, there is great potential for this next generation approach to enhance the treatment of Menkes disease, which currently has no FDA-approved treatment. We look forward to continuing to advance AAV-ATP7A with Dr. Kaler and supporting Sentynl as they finalize and complete the NDA submission for CUTX-101.” The content and research reported in this release are solely the responsibility of the authors and do not necessarily represent the official views of the NIH. About AAV-ATP7A Gene Therapy AAV-ATP7A is an adeno-associated virus (AAV)-based gene therapy in preclinical development for the treatment of Menkes Disease. AAV-ATP7A is being developed to deliver working copies of the copper transporter that is defective in Menkes patients and to be used in combination with CUTX-101 (Copper Histidinate). AAV-ATP7A was developed in part under an Exclusive License Agreement with the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), and in conjunction with the Research Institute at Nationwide Children’s Hospital and Principal Investigator, Dr. Stephen G. Kaler. About CUTX-101 (Copper Histidinate) CUTX-101 is a subcutaneous injectable formulation of Copper Histidinate in clinical development by Sentynl Therapeutics to treat patients with Menkes Disease. In a Phase 1/2 clinical trial conducted by Stephen G. Kaler, M.D., M.P.H., at the National Institutes of Health (“NIH”), early treatment of patients with Menkes disease with CUTX-101 led to an improvement in neurodevelopmental outcomes and survival. Cyprium previously reported positive topline clinical efficacy results for CUTX-101, demonstrating statistically significant improvement in overall survival for Menkes disease subjects who received early treatment (ET) with CUTX-101, compared to an untreated historical control cohort, with a nearly 80% reduction in the risk of death. Median overall survival (OS) was 177.1 months for CUTX-101 ET cohort compared to 16.1 months for the untreated historical control cohort. CUTX-101 has been granted FDA Breakthrough Therapy, Fast Track, Rare Pediatric Disease and FDA Orphan Drug Designations. Additionally, the European Medicines Agency granted Orphan Drug Designation for CUTX-101. An expanded access protocol, administered by Sentynl Therapeutics, for patients with Menkes disease is ongoing at multiple U.S. medical centers. Sentynl acquired the CUTX-101 program via asset purchase in December 2023 and will owe Cyprium milestone and royalty payments in connection with the development and commercialization of the product. About Menkes Disease Menkes disease is a rare X-linked recessive pediatric disease caused by gene mutations of copper transporter ATP7A. The minimum birth prevalence for Menkes disease is believed to be 1 in 34,810 live male births, and potentially as high as 1 in 8,664 live male births, based on recent genome-driven ascertainment (Kaler SG, Ferreira CR, Yam LS. Estimated birth prevalence of Menkes disease and ATP7A-related disorders based on the Genome Aggregation Database (gnomAD). Molecular Genetics and Metabolism Reports 2020 June 5;24:100602). The condition is characterized by distinctive clinical features, including sparse and depigmented hair (“kinky hair”), connective tissue problems, and severe neurological symptoms such as seizures, hypotonia, failure to thrive, and neurodevelopmental delays. Mortality is high in untreated Menkes disease, with many patients dying before the age of two years old. Milder versions of ATP7A mutations are associated with other conditions, including Occipital Horn Syndrome and ATP7A-related Distal Motor Neuropathy. Currently, there is no FDA-approved treatment for Menkes disease and its variants. About Cyprium Therapeutics Cyprium Therapeutics, Inc. (“Cyprium”) is focused on the development of novel therapies for the treatment of Menkes disease and related copper metabolism disorders. In March 2017, Cyprium entered into a Cooperative Research and Development Agreement with the Eunice Kennedy Shriver National Institute of Child Health and Human Development (“NICHD”), part of the NIH, to advance the clinical development of CUTX-101 (Copper Histidinate injection) for the treatment of Menkes disease. In 2023, Cyprium completed the transfer of its proprietary rights and assigned its FDA documents pertaining to CUTX-101 Copper Histidinate product candidate for the treatment of Menkes disease, to Sentynl Therapeutics, Inc. Cyprium and NICHD also previously entered into a worldwide, exclusive license agreement to develop and commercialize adeno-associated virus (AAV)-based gene therapy, called AAV-ATP7A, to deliver working copies of the copper transporter that is defective in patients with Menkes disease, and to be used in combination with CUTX-101; AAV-ATP7A is currently in pre-clinical development and has been granted FDA Orphan Drug Designation. Cyprium was founded by Fortress Biotech, Inc. (Nasdaq: FBIO) and is based in Miami. For more information, visit . About Fortress Biotech Fortress Biotech, Inc. (“Fortress”) is an innovative biopharmaceutical company focused on efficiently acquiring, developing and commercializing or monetizing promising therapeutic products and product candidates. The company has eight marketed prescription pharmaceutical products and over 25 programs in development at Fortress, at its majority-owned and majority-controlled partners and subsidiaries and at partners and subsidiaries it founded and in which it holds significant minority ownership positions. Such product candidates span six large-market areas, including oncology, rare diseases and gene therapy, which allow it to create value for shareholders. Fortress advances its diversified pipeline through a streamlined operating structure that fosters efficient drug development. The Fortress model is focused on leveraging its significant biopharmaceutical industry expertise and network to further expand the company’s portfolio of product opportunities. Fortress has established partnerships with some of the world’s leading academic research institutions and biopharmaceutical companies to maximize each opportunity to its full potential, including AstraZeneca, City of Hope, Fred Hutchinson Cancer Center, St. Jude Children’s Research Hospital, Nationwide Children’s Hospital and Sentynl. For more information, visit . Forward-Looking Statements This press release may contain “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, as amended. As used below and throughout this press release, the words “we”, “us” and “our” may refer to Fortress individually or together with one or more partner companies, as dictated by context. Such statements include, but are not limited to, any statements relating to our growth strategy and product development programs, ability to generate shareholder value, ability of our products to receive necessary approvals, including FDA approval, ability of our products and therapies to help patients and any other statements that are not historical facts. Forward-looking statements are based on management’s current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock price. Factors that could cause actual results to differ materially from those currently anticipated include, risks relating to: our growth strategy; financing and strategic agreements and relationships; our need for substantial additional funds and uncertainty relating to financings; our ability to identify, acquire, close and integrate product candidates successfully and on a timely basis; our ability to attract, integrate and retain key personnel; the early stage of products under development; the results of research and development activities; uncertainties relating to preclinical and clinical testing; risks relating to the timing of starting and completing clinical trials; the ability to secure and maintain third-party manufacturing, marketing and distribution of our and our partner companies’ products and product candidates; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as may be required by law, and we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995. The information contained herein is intended to be reviewed in its totality, and any stipulations, conditions or provisos that apply to a given piece of information in one part of this press release should be read as applying mutatis mutandis to every other instance of such information appearing herein. Company Contacts: Jaclyn Jaffe and Nicole McCloskey Cyprium Therapeutics, Inc. ir@cypriumtx.com Fortress Biotech, Inc. (781) 652-4500 ir@fortressbiotech.com Media Relations Contact: Tony Plohoros 6 Degrees (908) 591-2839 tplohoros@6degreespr.com