Anselamimab

Additional clinical and real-world data from the industry’s largest amyloidosis pipeline will advance understanding of underlying mechanisms of disease and unmet needs WILMINGTON, Del.--(BUSINESS WIRE)-- AstraZeneca and Alexion, AstraZeneca Rare Disease, will showcase 14 studies, including real-world evidence (RWE), from their portfolio and pipeline of investigational amyloidosis therapies at the International Symposium on Amyloidosis (ISA), in Rochester, MN from May 26–30, 2024. Presentations will include a new subgroup analyses of the Phase 3 NEURO-TTRansform study of WAINUATM (eplontersen), which was approved by the US Food and Drug Administration (FDA) in December 2023 for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults, commonly referred to as hATTR-PN or ATTRv-PN.1 Additionally, clinical data will be presented on ALXN2220 and anselamimab, which are being evaluated in Phase III clinical trials for ATTR and light chain (AL) amyloidosis, respectively. Sarah Walters, Vice President, US Cardiovascular, Renal & Metabolic Diseases, AstraZeneca, said: “AstraZeneca is dedicated to advancing the science and improving the lives of patients that are impacted by this devastating disease. Our data at the 2024 International Symposium on Amyloidosis demonstrate the leadership commitment AstraZeneca, Alexion and our partners Ionis and Neuroimmune have to develop best in class treatment options to serve a broad range of amyloidosis patients.” Christophe Hotermans, Senior Vice President, Head of Global Medical Affairs, Alexion, said: “With the largest and fastest-growing pipeline of investigational amyloidosis therapies, we are working to advance multiple modalities with the potential to halt, reduce or reverse organ damage. Key presentations at the ISA Annual Meeting include live-cell imaging and Phase I clinical data evaluating the ability of ALXN2220 to remove amyloid from cardiac tissue, to support its further study as a potential treatment for advanced ATTR cardiomyopathy. Additionally, findings on epidemiology and patient renal outcomes will reinforce the urgency for differentiated amyloidosis diagnosis and additional treatment options in AL amyloidosis.” Robust evidence program increases understanding of amyloidosis patient characteristics and treatment effectiveness. In addition to sub-analyses of the NEURO-TTRansform data, AstraZeneca will present US patient characteristics data, preliminary data from the OverTTuRe study measuring prevalence and characteristics of phenotypes in patients with ATTR amyloidosis in the United States and Japan; as well as the MaesTTRo study design, part of a global, multi-study, real-world evidence program designed to fill the evidence gaps of the effectiveness of treatments for ATTR. WAINUA is currently being evaluated in the Phase 3 CARDIO-TTRansform study for adults with cardiomyopathy of transthyretin-mediated amyloidosis (ATTR-CM), a systemic, progressive and fatal condition that typically leads to progressive heart failure and often death within three-to-five years from disease onset.2-5 The CARDIO-TTRansform Phase 3 study is fully enrolled with more than 1,400 patients–making it the largest study in this patient population to date.6 As part of a global development and commercialization agreement, AstraZeneca and Ionis are commercializing WAINUA for the treatment of hATTR-PN in the US and are seeking regulatory approval in Europe and other parts of the world.6 Novel investigational amyloid depleter assets demonstrate potential to improve outcomes for amyloidosis patients High-resolution live-cell imaging will offer new insight into the underlying cellular mechanism of antibody-mediated amyloid removal with ALXN2220. Results will show the addition of ALXN2220 to ATTR patient-derived cardiac tissue triggered phagocytosis, including recruitment of macrophages to amyloid deposits, detachment, internalization and degradation of ATTR amyloid. An encore poster presentation will report results from the first-in-human study of ALXN2220. Initial treatment with ALXN2220 was well tolerated with a transient increase in C-reactive proteins, suggesting initial targeted immune activation and recruitment of phagocytic immune cells. Additionally, an encore poster presentation will describe the pharmacokinetic and pharmacodynamic (PK/PD) model used for the selection of the dose range, dose escalation, dosing interval and study duration. Further, a poster will outline the baseline demographics and characteristics of the 406 participants of the Alexion CARES clinical program evaluating anselamimab in patients with Mayo Stage IIIa and IIIb AL amyloidosis, which includes the largest prospective AL amyloidosis study to date in advanced stage AL amyloidosis. Advancing understanding of the amyloidosis landscape A retrospective chart analysis from the US EMR TriNetX database will report outcomes in AL amyloidosis patients with renal involvement receiving bortezomib or daratumumab. Only half of patients were renal-responders and few achieved renal complete response (renCR, proteinuria ≤200 mg/day), underscoring the unmet medical need for this patient subset. Retrospective analyses will also be presented across three posters on the incidence and prevalence of AL and ATTR amyloidosis, reinforcing the importance of timely diagnosis of amyloidosis and continued monitoring. A comprehensive list of AstraZeneca key abstracts to be presented at the 2024 International Symposium on Amyloidosis includes7: Lead author Abstract title Presentation details Poster presentation session 1: 10:00-10:30 Poster presentation session 2: 14:45-15:45 Eplontersen Gillmore JD, Davis M, Hahn K, Smith G, Shivappa N, Papas M, Folkvaljon F, Pao C, Sundin AK, L Wittbrodt E, Grogan M Prospective, Real-World Data on the Characteristics, Treatment Patterns, and Outcomes of Patients With Transthyretin Amyloidosis: Design of the MaesTTRo Study Date: Wednesday, May 29 Abstract ID: 204 Waddington Cruz M, Berk J, Parman Y, Gertz M, Khella S, Weiler M, Kwoh J, Chen J, Reicher B, Nåtman J, Dasgupta N Eplontersen for Hereditary Transthyretin Amyloidosis With Polyneuropathy: An Exploratory Analysis of Treatment Effect in Male and Female Patients Date: Wednesday, May 29 Abstract ID: 202 Gillmore JD, Adams D, Weiler M, Masri A, Obici L, Kwoh J, Reicher B, Chen J, Waddington Cruz M, Natman J, Gertz M Eplontersen for Hereditary Transthyretin Amyloidosis With Polyneuropathy: An Exploratory Analysis in Patients With the V30M TTR variant and Early-onset or Late-onset Disease Date: Wednesday, May 29 Abstract ID: 230 Wixner J, Conceição I, Berk J, Adams D, Polydefkis M, Attarian S, Gillmore J, Dyck J, Coelho T, Chen J, Hardy E, Kwoh J, Nåtman J, Waddington Cruz M Neuropathy Impairment and Nutritional Status With Eplontersen in Patients With Hereditary Transthyretin-Mediated Amyloidosis Date: Wednesday, May 29 Abstract ID: 174 Alexander K, Alvarez C, Kumar N, Pao C, Wittbrodt E, Kohsaka S Prevalence and Descriptive Characteristics of Clinical Phenotypes in Patients With ATTR amyloidosis in the United States and Japan: Preliminary Results from the OverTTuRe Study Date: Monday, May 27 Abstract ID: 198 Bazell C, Alston M, Kumar N, Huang J, Venditto J, Grillis D, Eisenberg S, Nativi-Nicolau J Descriptive Characteristics of Patients Diagnosed With Transthyretin Amyloidosis in the Commercial and Medicare Populations Date: Wednesday, May 29 Abstract ID: 518 ALXN 2220 Michalon A, Huy, Mercuri M, Kahr P, Hock C, Grimm J ALXN2220: High-resolution Live-cell Imaging of Antibody-mediated Cardiac ATTR Amyloid Depletion Date: Tuesday, May 28 Abstract ID: 226 Michalon A, Renaud L, Machacek M, Cortijo C, Udaata C, Mercuri M, Buller F, Hock C, Nitsch R, Kahr P, Grimm J Prediction of Cardiac ATTR Depletion by NI006 Using Mechanistic PK/PD Modeling Date: Wednesday, May 29 Abstract ID: 291 Aus dem Siepen F, Van der Meer P, Damy T, Garcia-Pavia P, Donal E, Lairez O, Blank A, Kristen A, Quarta CC, Buchele G, Mercuri M, Hock C, Michalon A, Kahr P, Rey S, Tichy M ALXN2220: Targeted Immune Activation in Patients Undergoing Antibody-mediated Cardiac ATTR Amyloid Depletion Date: Wednesday, May 29 Abstract ID: 227 Amyloidosis Incidence and Analyses Laires PA, Evans J, Thompson J, Manwani R, Mudumby P, Field M, Fang S Prevalence, Incidence, and Characterization of Light Chain Amyloidosis in the USA: A Real-World Analysis Utilizing Electronic Health Records (EHR) Date: Monday, May 27 Abstract ID: 65 Manwani R, Yang F, Zhang Y, Laires PA Outcomes in Patients With AL Amyloidosis With Renal Involvement: Findings from the TriNetX Database Date: Monday, May 27 Abstract ID: 113 Laires PA, Li SXL, Uday A, Kumar P, Silva AM, Quarta C Prevalence and Incidence of ATTR Amyloidosis in the United States: Insights from Claims Database and Electronic Health Records Date: Tuesday, May 28 Abstract ID: 68 Laires PA, Zhang Y, Manwani R, Silva AM, Catini J, Thompson J, Dozier M, Yang F Subtype Distribution of Amyloidosis in the United States: Insights from an Electronic Health Records Database Analysis Date: Tuesday, May 28 Abstract ID: 112 The full list of ISA 2024 congress abstracts are available online. INDICATION for WAINUATM (eplontersen) WAINUA injection, for subcutaneous use, 45 mg is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. IMPORTANT SAFETY INFORMATION for WAINUATM (eplontersen) WARNINGS AND PRECAUTIONS Reduced Serum Vitamin A Levels and Recommended Supplementation WAINUA leads to a decrease in serum vitamin A levels. Supplement with recommended daily allowance of vitamin A. Refer patient to an ophthalmologist if ocular symptoms suggestive of vitamin A deficiency occur. ADVERSE REACTIONS Most common adverse reactions (≥9% in WAINUA-treated patients) were vitamin A decreased (15%) and vomiting (9%). Please see link to US Full Prescribing Information for WAINUA. Notes NEURO-TTRansform NEURO-TTRansform is a global, open-label, randomized trial evaluating the efficacy and safety of eplontersen in patients with hATTR-PN.8,9 The trial enrolled adult patients with hATTR-PN Stage 1 or Stage 2 compared to the external placebo group from the NEURO-TTR registrational trial that Ionis completed in 2017.8,9 The comparison of efficacy and safety for WAINUA versus external placebo was based on data up to week 66, and all patients were followed on treatment until week 85, when they had the option to transition into an open-label extension study, which is still ongoing.8,9 WAINUA WAINUATM (eplontersen) is a ligand-conjugated antisense oligonucleotide (LICA) medicine designed to reduce the production of transthyretin, or TTR protein.9,10 WAINUA has been approved in the US for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults (also referred to as hATTR-PN).1 AstraZeneca in amyloidosis Amyloidosis is a group of complex rare diseases caused by abnormal proteins that misfold and clump together to form toxic amyloids that deposit in tissues or organs, including the heart, kidneys and peripheral nerves.11-12 The build-up of these toxic amyloids can result in significant organ damage and organ failure that can severely impact quality of life and ultimately be fatal.11-12 AstraZeneca and its Rare Disease Unit, Alexion, are developing and evaluating multiple modalities with the potential to halt and reduce organ damage across various types of amyloidosis. The Company is uniquely positioned to lead therapeutic and diagnostic advances for people living with amyloidosis with the largest and fastest-growing pipeline of investigational amyloidosis therapies to address the spectrum of patient needs. Alexion Alexion, AstraZeneca Rare Disease, is focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and delivery of life-changing medicines. A pioneering leader in rare disease for more than three decades, Alexion was the first to translate the complex biology of the complement system into transformative medicines, and today it continues to build a diversified pipeline across disease areas with significant unmet need, using an array of innovative modalities. As part of AstraZeneca, Alexion is continually expanding its global geographic footprint to serve more rare disease patients around the world. It is headquartered in Boston, MA, US. About AstraZeneca AstraZeneca is a global, science-led biopharmaceutical company that focuses on the discovery, development and commercialization of prescription medicines in Oncology, Rare Diseases and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca operates in over 100 countries, and its innovative medicines are used by millions of patients worldwide. For more information, please visit and follow us on social media @AstraZeneca. References WAINUATM (eplontersen) [prescribing information]. Wilmington, DE: AstraZeneca Pharmaceuticals LP; 2023. Viney N, et al. Ligand conjugated antisense oligonucleotide for the treatment of transthyretin amyloidosis: preclinical and phase 1 data. ESC Heart Failure. 2021;8:652-661. Rintell D, et al. Patient and family experience with transthyretin amyloid cardiomyopathy (ATTR-CM) and polyneuropathy (ATTR-PN) amyloidosis: results of two focus groups. Orphanet J Rare Dis. 2021;16:70. Columbia University Irving Medical Center [Internet]. Drug Reduces Death from Underdiagnosed Form of Heart Failure [last accessed 11 December 2023]. Available from: . ClinicalTrials.gov. CARDIO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM) [last accessed 21 May 2024] Available from: . Ionis Pharmaceuticals [Internet]. WAINUA™ (eplontersen) granted regulatory approval in the U.S. for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis [last accessed 16 May 2024]. Available from: . Mayo Clinic. International Symposium on Amyloidosis: Abstracts/Posters [last accessed 21 May 2024] Available from: . Coelho T, et al. Design and Rationale of the Global Phase 3 NEURO-TTRansform Study of Antisense Oligonucleotide AKCEA-TTR-LRx(ION-682884-CS3) in Hereditary Transthyretin-Mediated Amyloid Polyneuropathy. Nerol Ther. 2021 Jun;10(1):375-389. Coelho T, et al. Eplontersen for Hereditary Transthyretin Amyloidosis With Polyneuropathy. JAMA. 2023;330(15):1448–1458. Coelho T, et al. Characteristics of Patients with Hereditary Transthyretin Amyloidosis-Polyneuropathy (ATTRv-PN) in NEURO-TTRansform, an Open-label Phase 3 Study of Eplontersen. Neurol Ther. 2023;12:267–287. Cleveland Clinic. Amyloidosis. Cleveland Clinic website. Accessed May 21, 2024. . Mayo Clinic. Amyloidosis. Mayo Clinic website. Accessed May 21, 2024. . US-89473 Last Updated 5/24

Fortress‘ late-stage pipeline continues to advance and may generate up to three regulatory approvals on NDAs and BLAs in the next 12 months and potentially a fourth BLA filing as early as 2025 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, May 15, 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 first quarter ended March 31, 2024. Lindsay A. Rosenwald, M.D., Fortress’ Chairman, President and Chief Executive Officer, said, “We achieved first quarter year-over-year product revenue growth of 7%, which was driven by greater than 20% year-over-year growth in our flagship products, Qbrexza® and Accutane®. In the first quarter of 2024, the U.S. Food and Drug Administration (“FDA”) accepted the New Drug Application (“NDA”) filing for DFD-29 and set a Prescription Drug User Fee Act (“PDUFA”) goal date of November 4, 2024. If approved, DFD-29 has the potential to be the only oral, systemic therapy to address inflammatory lesions and erythema (redness) from rosacea, differentiating it as a potential best-in-class solution for the millions of patients suffering from rosacea. We also dosed the first patient in a multi-center Phase 2 study for Triplex for control of cytomegalovirus (“CMV”) in patients undergoing liver transplantation and received grant funding from the National Institutes of Health (“NIH”) to further advance cell and gene therapy candidates for the potential treatment of adults living with HIV and children with Menkes disease. Looking ahead, our expansive portfolio of development-stage programs across multiple areas, including oncology, dermatology, and rare diseases, holds the potential for up to three NDA and Biologics License Application (“BLA”) regulatory approvals within the next 12 months and potentially a fourth BLA filing as early as 2025. Additionally, we anticipate multiple data readouts this year, including topline data from the Phase 1b/2a clinical trial of AJ201 to treat spinal and bulbar muscular atrophy (“SBMA”), data from the Phase 1b clinical trial of dotinurad for the treatment of gout and hyperuricemia and topline Phase 2 clinical data of Triplex, a CMV vaccine for adults co-infected with HIV and CMV. This sustained progress underscores the strength of Fortress’ business model, centered on acquiring and advancing assets that address unmet medical needs and enhance long-term value for shareholders through product revenues, equity holdings and royalties.” Recent Corporate Highlights1: Regulatory Milestones and Updates In March 2024, the FDA accepted the NDA for DFD-29 (Minocycline Hydrochloride Modified Release Capsules, 40 mg) and set a PDUFA goal date of November 4, 2024. We submitted the NDA to the FDA seeking approval for DFD-29 for the treatment of inflammatory lesions and erythema of rosacea in adults in January 2024. Both double blinded, randomized controlled DFD-29 Phase 3 clinical trials achieved their co-primary and all secondary endpoints with subjects completing the 16-week treatment with no significant safety issues. DFD-29 demonstrated statistical superiority compared to both Oracea capsules and placebo for Investigator’s Global Assessment (IGA) treatment success and the reduction in the total inflammatory lesion count in both clinical trials. Additionally, DFD-29 showed significantly superior reduction in Clinicians Erythema Assessment compared to placebo in both of the Phase 3 clinical trials. DFD-29 is currently in development at our partner company, Journey Medical Corporation (Nasdaq: DERM) (“Journey Medical”). The CUTX-101 rolling NDA submission is ongoing and is expected to be completed by our partner, Sentynl Therapeutics, Inc. in 2024. 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. 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 solely 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 intend to seek to address the feedback in a potential BLA resubmission, which is currently targeted for mid-year. Cosibelimab is currently in development at our partner company, Checkpoint Therapeutics, Inc. (Nasdaq: CKPT) (“Checkpoint”). Based on its public statements, AstraZeneca plc has estimated that it expects the FDA to accept its BLA submission of CAEL-101 (anselamimab) to treat AL amyloidosis for review as early as 2025. Clinical Updates The Phase 2 clinical trial of Triplex, a CMV vaccine, for adults co-infected with HIV and CMV is now fully enrolled with topline data anticipated in the fourth quarter of 2024. The study aims to show that vaccination with Triplex can safely elicit a CMV-specific immune response and reduce asymptomatic CMV replication in a population of people with HIV on suppressive antiretroviral therapy. The study will also evaluate whether this intervention might reduce chronic inflammation and immune activation, as compared to placebo, and thus, potentially reduce related mortality and morbidity. In May 2024, we announced that the first patient was dosed in a multi-center, placebo-controlled, randomized Phase 2 study of Triplex for control of CMV in patients undergoing liver transplantation. The trial is funded by a grant from the NIH’s National Institute of Allergy and Infectious Diseases of the (NIH/NIAID) that could provide over $20 million in non-dilutive funding. Triplex is currently in development at our subsidiary company, Helocyte, Inc. A Phase 1b clinical trial in patients with gout and hyperuricemia is ongoing in the U.S. to confirm the comparability of U.S. patients’ response to dotinurad (urate transporter (URAT1) inhibitor) with data generated in Japan, and to assess drug-drug interactions, if any, with allopurinol. We expect to announce data from this trial in mid-2024. Dotinurad is currently in development at our subsidiary company, Urica Therapeutics, Inc. (“Urica”). Commercial Product Updates Journey Medical’s total revenues for the first quarter ended March 31, 2024 were $13.0 million, an increase of $0.8 million, or 7%, compared to total net revenues of $12.2 million for the first quarter ended March 31, 2023. General Corporate: In January 2024, Fortress raised gross proceeds of approximately $11.0 million in a registered direct offering priced at-the-market under Nasdaq rules. In January 2024, Checkpoint raised gross proceeds of approximately $14.0 million in a registered direct offering, and Avenue raised approximately $5.0 million gross proceeds from warrant exercise transactions. Financial Results: As of March 31, 2024, Fortress’ consolidated cash, cash equivalents and restricted cash totaled $85.8 million, compared to $83.4 million as of December 31, 2023, an increase of $2.5 million during the quarter. Fortress’ consolidated cash, cash equivalents and restricted cash, totaling $85.8 million as of March 31, 2024, includes $45.6 million attributable to Fortress and the private subsidiaries, $3.2 million attributable to Avenue, $11.2 million attributable to Checkpoint, $1.7 million attributable to Mustang Bio and $24.1 million attributable to Journey Medical. Fortress’ consolidated cash, cash equivalents and restricted cash, totaled $83.4 million as of December 31, 2023, which included $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 first quarter, in May 2024, Avenue raised approximately $4.4 million in gross proceeds from warrant exercise transactions and Mustang raised approximately $4.0 million in gross proceeds from a public offering of common stock and warrants. Fortress’ consolidated net revenue totaled $13.0 million for the first quarter ended March 31, 2024, all of which was generated from our marketed dermatology products. This compares to consolidated revenue totaling $12.4 million for the first quarter of 2023, which included $12.2 million in revenue generated from our marketed dermatology products. Consolidated research and development expenses including license acquisitions totaled $24.8 million for the first quarter ended March 31, 2024, compared to $39.5 million for the first quarter ended March 31, 2023. Consolidated selling, general and administrative costs were $17.9 million for the first quarter ended March 31, 2024, compared to $25.3 million for the first quarter ended March 31, 2023. Consolidated net loss attributable to common stockholders was $(17.7) million, or $(1.03) per share, for the first quarter ended March 31, 2024, compared to net loss attributable to common stockholders of $(23.5) million, or $(3.47) per share for the first quarter ended March 31, 2023. All share and per share information has been retroactively adjusted to give effect to the Company’s October 2023 1-for-15 reverse stock split for all periods presented, unless otherwise indicated. 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 . 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 Unaudited Condensed Consolidated Balance Sheets ($ in thousands except for share and per share amounts) March 31, December 31, 2024 2023 ASSETS Current assets Cash and cash equivalents $ 83,774 $ 80,927 Accounts receivable, net 9,799 15,222 Inventory 10,580 10,206 Other receivables - related party 324 167 Prepaid expenses and other current assets 12,071 10,500 Total current assets 116,548 117,022 Property, plant and equipment, net 6,128 6,505 Operating lease right-of-use asset, net 16,462 16,990 Restricted cash 2,063 2,438 Intangible assets, net 19,473 20,287 Other assets 3,971 4,284 Total assets $ 164,645 $ 167,526 LIABILITIES AND STOCKHOLDERS’ EQUITY (DEFICIT) Current liabilities Accounts payable and accrued expenses $ 76,379 $ 73,562 Income taxes payable 843 843 Common stock warrant liabilities 689 886 Operating lease liabilities, short-term 2,601 2,523 Partner company convertible preferred shares, short-term, net 4,021 3,931 Partner company installment payments - licenses, short-term, net 3,000 3,000 Other short-term liabilities 163 163 Total current liabilities 87,696 84,908 Notes payable, long-term, net 61,420 60,856 Operating lease liabilities, long-term 17,619 18,282 Other long-term liabilities 1,847 1,893 Total liabilities 168,582 165,939 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 March 31, 2024 and December 31, 2023, respectively, liquidation value of $25.00 per share 3 3 Common stock, $0.001 par value, 200,000,000 shares authorized, 19,375,343 and 15,093,053 shares issued and outstanding as of March 31, 2024 and December 31, 2023, respectively 19 15 Additional paid-in-capital 733,290 717,396 Accumulated deficit (710,287 ) (694,870 ) Total stockholders' equity attributed to the Company 23,025 22,544 Non-controlling interests (26,962 ) (20,957 ) Total stockholders' equity (deficit) (3,937 ) 1,587 Total liabilities and stockholders' equity (deficit) $ 164,645 $ 167,526 FORTRESS BIOTECH, INC. AND SUBSIDIARIES Unaudited Condensed Consolidated Statements of Operations ($ in thousands except for share and per share amounts) Three Months Ended March 31, 2024 2023 Revenue Product revenue, net $ 13,030 $ 12,165 Collaboration revenue — 181 Revenue - related party — 35 Other revenue — 48 Net revenue 13,030 12,429 Operating expenses Cost of goods sold - product revenue 6,816 6,449 Research and development 24,839 35,276 Research and development - licenses acquired — 4,230 Selling, general and administrative 17,941 25,341 Total operating expenses 49,596 71,296 Loss from operations (36,566 ) (58,867 ) Other income (expense) Interest income 833 1,036 Interest expense and financing fee (2,602 ) (4,296 ) Gain (loss) on common stock warrant liabilities (667 ) 6,678 Other income (expense) (21 ) 304 Total other income (expense) (2,457 ) 3,722 Net loss (39,023 ) (55,145 ) Net loss attributable to non-controlling interests 23,606 33,608 Net loss attributable to Fortress $ (15,417 ) $ (21,537 ) Net loss attributable to common stockholders $ (17,731 ) $ (23,545 ) Net loss per common share attributable to common stockholders - basic and diluted $ (1.03 ) $ (3.47 ) Weighted average common shares outstanding - basic and diluted 17,151,945 6,792,376 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.