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UNLOXCYT™ (cosibelimab-ipdl) approved by U.S. FDA as first and only anti-PD-L1 treatment for advanced cutaneous squamous cell carcinomaWALTHAM, Mass., March 28, 2025 (GLOBE NEWSWIRE) -- Checkpoint Therapeutics, Inc. (“Checkpoint”) (Nasdaq: CKPT), a commercial-stage immunotherapy and targeted oncology company, today announced financial results for the fiscal year ended December 31, 2024, and recent corporate updates. Recent Corporate Updates: In March 2025, Checkpoint announced that it entered into an Agreement and Plan of Merger (the “Merger Agreement”) with Sun Pharmaceutical Industries, Inc. (“Sun Pharma”), and a wholly owned subsidiary of Sun Pharma, with Checkpoint continuing as the surviving corporation of the transaction and a wholly owned subsidiary of Sun Pharma (the “Merger”). The total transaction value of the Merger, including the upfront cash payment and the maximum value of the contingent value right (“CVR”), is up to approximately $416 million, and the Merger is expected to be completed in the second quarter of 2025. The transaction is subject to customary closing conditions, including required regulatory approvals and approval by the holders of a majority of the voting power of outstanding shares of Checkpoint common stock, and by the holders of a majority of the shares of Checkpoint common stock that are not held by Fortress Biotech, Inc. or by certain other affiliates of Checkpoint.In December 2024, Checkpoint announced that the U.S. Food and Drug Administration (“FDA”) approved UNLOXCYTTM (cosibelimab-ipdl) for the treatment of adults with metastatic cutaneous squamous cell carcinoma (“cSCC”) or locally advanced cSCC who are not candidates for curative surgery or curative radiation. UNLOXCYT is the first and only programmed death ligand-1 (“PD-L1”) blocking antibody to receive FDA marketing approval for this indication.In September 2024, Checkpoint presented longer-term data from its pivotal trial of cosibelimab in locally advanced and metastatic cSCC during the European Society for Medical Oncology (“ESMO”) Congress 2024. Longer-term results for cosibelimab presented at the ESMO Congress demonstrate a deepening of response over time, with higher objective response and complete response rates than initially observed at the primary analyses. A copy of the ESMO poster can be found on the Publications page of Checkpoint’s website. Financial Results: Cash Position: As of December 31, 2024, Checkpoint’s cash and cash equivalents totaled $6.6 million, compared to $4.9 million at December 31, 2023, an increase of $1.7 million. Subsequent to the end of the fiscal year, Checkpoint received approximately $38.1 million in cash proceeds through the exercise of existing warrants.R&D Expenses: Research and development expenses for the year ended December 31, 2024, were $36.2 million, compared to $43.6 million for the year ended December 31, 2023, a decrease of $7.4 million. Research and development expenses for the year ended December 31, 2024, included $12.9 million of non-cash stock expenses, compared to $4.6 million in non-cash stock expenses for the year ended December 31, 2023.G&A Expenses: General and administrative expenses for the year ended December 31, 2024, were $20.1 million, compared to $8.7 million for the year ended December 31, 2023, an increase of $11.4 million. General and administrative expenses for the year ended December 31, 2024, included $11.0 million of non-cash stock expenses, compared to $2.7 million in non-cash stock expenses for the year ended December 31, 2023.Net Loss: Net loss attributable to common stockholders for the year ended December 31, 2024, was $56.2 million, or $1.42 per share, compared to a net loss of $51.8 million, or $3.17 per share, for the year ended December 31, 2023. About UNLOXCYTTM (cosibelimab-ipdl) UNLOXCYT is a human immunoglobulin G1 monoclonal antibody that binds PD-L1 and blocks the interaction between PD-L1 and its T cell receptors, PD-1 and B7.1. This interaction releases the inhibitory effects of PD-L1 on the anti-tumor immune response. UNLOXCYT has also been shown to induce antibody-dependent cell-mediated cytotoxicity. INDICATION and IMPORANT SAFETY INFORMATION INDICATION UNLOXCYT (cosibelimab-ipdl) is indicated for the treatment of adults with metastatic cSCC or locally advanced cSCC who are not candidates for curative surgery or curative radiation. IMPORTANT SAFETY INFORMATION Severe and Fatal Immune-Mediated Adverse Reactions Immune-mediated adverse reactions listed herein may not include all possible severe and fatal immune-mediated adverse reactions. Immune-mediated adverse reactions, which can be severe or fatal, can occur in any organ system or tissue, and occur at any time after starting a PD-1/PD-L1–blocking antibody, including UNLOXCYT. While immune-mediated adverse reactions usually manifest during treatment, they can also manifest after discontinuation of PD-1/PD-L1–blocking antibodies. Immune-mediated adverse reactions affecting more than one body system can occur simultaneously.Monitor closely for signs and symptoms of immune-mediated adverse reactions. Evaluate liver enzymes, creatinine, and thyroid function tests at baseline and periodically during treatment. In cases of suspected immune-mediated adverse reactions, initiate appropriate workup to exclude alternative etiologies, including infection. Institute medical management promptly, including specialty consultation as appropriate.Withhold or permanently discontinue UNLOXCYT depending on the severity of the adverse reaction (see Dosage and Administration in Prescribing Information). In general, if UNLOXCYT requires interruption or discontinuation, administer systemic corticosteroids (1 to 2 mg/kg/day prednisone or equivalent) until improvement to Grade 1 or less. Upon improvement to Grade 1 or less, initiate corticosteroid taper and continue to taper over at least 1 month. Consider administration of other systemic immunosuppressants in patients whose immune-mediated adverse reaction is not controlled with corticosteroids. Immune-Mediated Pneumonitis UNLOXCYT can cause immune-mediated pneumonitis. In patients treated with other PD-1/PD-L1–blocking antibodies, the incidence of pneumonitis is higher in patients who have received prior thoracic radiation. Immune-mediated pneumonitis occurred in 1% (3/223, Grade 2) of patients receiving UNLOXCYT. Immune-Mediated Colitis UNLOXCYT can cause immune-mediated colitis, which may present with diarrhea, abdominal pain, and lower gastrointestinal bleeding. Cytomegalovirus infection/reactivation has occurred in patients with corticosteroid-refractory immune-mediated colitis treated with PD-1/PD-L1–blocking antibodies. In cases of corticosteroid-refractory colitis, consider repeating infectious workup to exclude alternative etiologies. Immune-mediated colitis occurred in 0.4% (1/223, Grade 1) of patients receiving UNLOXCYT. Immune-Mediated Hepatitis UNLOXCYT can cause immune-mediated hepatitis. Immune-Mediated EndocrinopathiesAdrenal Insufficiency UNLOXCYT can cause primary or secondary adrenal insufficiency. For Grade 2 or higher adrenal insufficiency, initiate symptomatic treatment per institutional guidelines, including hormone replacement as clinically indicated. Withhold or permanently discontinue UNLOXCYT depending on severity. Adrenal insufficiency occurred in 0.9% (2/223) of patients receiving UNLOXCYT, including Grade 2 in 0.4% (1/223) of patients. Hypophysitis UNLOXCYT can cause immune-mediated hypophysitis. Hypophysitis can present with acute symptoms associated with mass effect such as headache, photophobia, or visual field cuts. Hypophysitis can cause hypopituitarism. Initiate hormone replacement as clinically indicated. Withhold or permanently discontinue UNLOXCYT depending on severity. Thyroid Disorders UNLOXCYT can cause immune-mediated thyroid disorders. Thyroiditis can present with or without endocrinopathy. Hypothyroidism can follow hyperthyroidism. Initiate hormone replacement or medical management of hyperthyroidism as clinically indicated. Withhold or permanently discontinue UNLOXCYT depending on severity. Hypothyroidism occurred in 10% (22/223) of patients receiving UNLOXCYT, including Grade 2 in 5% (10/223) of patients. Hyperthyroidism occurred in 5% (12/223) of patients receiving UNLOXCYT, including Grade 2 in 0.4% (1/223) of patients. Type 1 Diabetes Mellitus, Which Can Present with Diabetic Ketoacidosis UNLOXCYT can cause type 1 diabetes mellitus, which can present with diabetic ketoacidosis. Monitor patients for hyperglycemia or other signs and symptoms of diabetes. Initiate treatment with insulin as clinically indicated. Withhold or permanently discontinue UNLOXCYT depending on severity. Immune-Mediated Nephritis with Renal Dysfunction UNLOXCYT can cause immune-mediated nephritis. Immune-Mediated Dermatologic Adverse Reactions UNLOXCYT can cause immune-mediated rash or dermatitis. Bullous and exfoliative dermatitis, including Stevens-Johnson syndrome (SJS), toxic epidermal necrolysis (TEN), and drug rash with eosinophilia and systemic symptoms (DRESS), have occurred with PD-1/PD-L1–blocking antibodies. Topical emollients and/or topical corticosteroids may be adequate to treat mild to moderate non-bullous/exfoliative rashes. Withhold or permanently discontinue UNLOXCYT depending on severity. Immune-mediated dermatologic adverse reactions occurred in 7% (15/223) of patients receiving UNLOXCYT, including Grade 3 in 0.9% (2/223) of patients and Grade 2 in 4% (9/223) of patients. Other Immune-Mediated Adverse Reactions The following clinically significant immune-mediated adverse reactions occurred in <1% of the 223 patients who received UNLOXCYT or were reported with the use of other PD-1/PD-L1–blocking antibodies. Severe or fatal cases have been reported for some of these adverse reactions. Cardiac/Vascular: Myocarditis, pericarditis, vasculitis.Nervous System: Meningitis, encephalitis, myelitis and demyelination, myasthenic syndrome/myasthenia gravis (including exacerbation), Guillain-Barre syndrome, nerve paresis, autoimmune neuropathy.Ocular: Uveitis, iritis, other ocular inflammatory toxicities. Some cases can be associated with retinal detachment. Various grades of visual impairment to include blindness can occur. If uveitis occurs in combination with other immune-mediated adverse reactions, consider a Vogt-Koyanagi-Harada–like syndrome, as this may require treatment with systemic steroids to reduce the risk of permanent vision loss.Gastrointestinal: Pancreatitis, including increases in serum amylase and lipase levels, gastritis, duodenitis.Musculoskeletal and Connective Tissue: Myositis/polymyositis, rhabdomyolysis and associated sequelae including renal failure, arthritis, polymyalgia rheumatica.Endocrine: Hypoparathyroidism.Other (Hematologic/Immune): Autoimmune hemolytic anemia, aplastic anemia, hemophagocytic lymphohistiocytosis, systemic inflammatory response syndrome, histiocytic necrotizing lymphadenitis (Kikuchi lymphadenitis), sarcoidosis, immune thrombocytopenia, solid organ transplant rejection, other transplant (including corneal graft) rejection. Infusion-Related Reactions UNLOXCYT can cause severe or life-threatening infusion-related reactions. Infusion-related infusion reactions were reported in 11% (24/223) of patients, including Grade 2 in 5.8% (13/223) of patients receiving UNLOXCYT.Monitor patients for signs and symptoms of infusion-related reactions. Interrupt or slow the rate of infusion or permanently discontinue UNLOXCYT based on severity of reaction. Consider premedication with an antipyretic and/or an antihistamine for patients who have had previous systemic reactions to infusions of therapeutic proteins. Complications of Allogeneic HSCT Fatal and other serious complications can occur in patients who receive allogeneic hematopoietic stem cell transplantation (HSCT) before or after being treated with a PD-1/PD-L1–blocking antibody. Transplant-related complications include hyperacute graft-versus-host disease (GVHD), acute GVHD, chronic GVHD, hepatic veno-occlusive disease after reduced intensity conditioning, and steroid-requiring febrile syndrome (without an identified infectious cause). These complications may occur despite intervening therapy between PD-1/PD-L1 blockade and allogeneic HSCT. Follow patients closely for evidence of transplant-related complications and intervene promptly. Consider the benefit versus risks of treatment with a PD-1/PD-L1–blocking antibody prior to or after an allogeneic HSCT. Embryo-Fetal Toxicity Based on its mechanism of action, UNLOXCYT can cause fetal harm when administered to a pregnant woman. Animal studies have demonstrated that inhibition of the PD-1/PD-L1 pathway can lead to increased risk of immune-mediated rejection of the developing fetus, resulting in fetal death. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with UNLOXCYT and for 4 months after the last dose. Common Adverse Reactions The most common adverse reactions (≥10%) were fatigue, musculoskeletal pain, rash, diarrhea, hypothyroidism, constipation, nausea, headache, pruritus, edema, localized infection, and urinary tract infection. Please see full Prescribing Information. About Checkpoint Therapeutics Checkpoint Therapeutics, Inc. is a commercial-stage immunotherapy and targeted oncology company focused on the acquisition, development and commercialization of novel treatments for patients with solid tumor cancers. Checkpoint has received approval from the FDA for UNLOXCYTTM (cosibelimab-ipdl) for the treatment of adults with metastatic cSCC or locally advanced cSCC who are not candidates for curative surgery or curative radiation. Additionally, Checkpoint is evaluating its lead investigational small-molecule, targeted anti-cancer agent, olafertinib (formerly CK-101), a third-generation epidermal growth factor receptor (“EGFR”) inhibitor, as a potential new treatment for patients with EGFR mutation-positive non-small cell lung cancer. Checkpoint is headquartered in Waltham, MA and was founded by Fortress Biotech, Inc. (Nasdaq: FBIO). For more information, visit www.checkpointtx.com. Forward‐Looking Statements This press release contains “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, each as amended, that involve a number of risks and uncertainties. For those statements, we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, express or implied, statements regarding the Merger and related matters, including the benefits of and timeline for closing the Merger, any payments under the CVRs, prospective performance and opportunities, post-closing operations and the outlook for the companies’ businesses; projections of or targets for revenues, costs, income (or loss), earnings per share, capital expenditures, dividends, capital structure, net financials and other financial measures; expectations for the timing and commercial launch and availability of UNLOXCYTTM (cosibelimab-ipdl) for the treatment of adults with metastatic cSCC or locally advanced cSCC who are not candidates for curative surgery or curative radiation; the commercial potential of UNLOXCYT; anticipated healthcare professional and patient acceptance and use of UNLOXCYT for the FDA-approved indication; and assumptions underlying or relating to such statements. Factors that may affect future results and may cause these forward-looking statements to be inaccurate include, but are not limited to: uncertainties as to the timing of completion of the Merger; uncertainties as to whether Checkpoint’s stockholders will vote to approve the transaction; the possibility that competing offers will be made; the possibility that various closing conditions for the transaction may not be satisfied or waived, including that a governmental entity may prohibit, delay or refuse to grant approval for the consummation of the transaction (or only grant approval subject to adverse conditions or limitations); the possibility that the proposed transaction may not be completed in the time frame expected by Checkpoint, or at all; failure to realize the anticipated benefits of the proposed transaction in the time frame expected, or at all; the effects of the transaction on relationships with employees, other business partners or governmental entities; potential adverse reactions or changes to business relationships resulting from the announcement or completion of the proposed transaction; significant or unexpected costs, charges or expenses resulting from the proposed transaction; negative effects of this announcement or the consummation of the proposed acquisition on Checkpoint’s common stock and/or Checkpoint’s operating results; the difficulty of predicting the timing or outcome of regulatory approvals or actions; the risks related to non-achievement of the CVR milestone and that holders of the CVRs will not receive payments in respect of the CVRs; other business effects, including the effects of industry, economic or political conditions outside of the companies’ control; transaction costs; actual or contingent liabilities; risk of litigation and/or regulatory actions related to the proposed acquisition; adverse impacts on business, operating results or financial condition in the future due to pandemics, epidemics or outbreaks, and their impact on Checkpoint’s business, operations, supply chain, patient enrollment and retention, clinical trials, strategy, goals and anticipated milestones; government-mandated or market-driven price decreases for Checkpoint’s products; the existence or introduction of competing products; reliance on information technology; Checkpoint’s ability to successfully market current and new products; Checkpoint’s and its collaborators’ ability to continue to conduct research and clinical programs; and exposure to product liability and legal proceedings and investigations. Further risks and uncertainties that could cause actual results to differ materially from the results anticipated by the forward-looking statements are detailed from time to time in Checkpoint’s periodic reports filed with the SEC, including the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2024 and the definitive proxy statement to be filed by Checkpoint with the SEC in connection with the proposed transaction. These filings, when available, are available on the investor relations section of Checkpoint’s website at https://ir.checkpointtx.com or on the SEC’s website at https://www.sec.gov. Any forward-looking statements set forth in this press release speak only as of the date of this press release, are made based on current beliefs and judgments, and are not predictions of actual performance. New risks and uncertainties arise from time to time, and it is impossible for us to predict these events or how they may affect us. We caution that a number of important factors, including those described in this document, could cause actual results to differ materially from those contemplated in any forward-looking statements. Unlisted factors may present significant additional obstacles to the realization of forward-looking statements. 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 required by law. This press release and prior releases are available at www.checkpointtx.com. The information found on our website is not incorporated by reference into this press release and is included for reference purposes only. Company Contact:Jaclyn JaffeCheckpoint Therapeutics, Inc.(781) 652-4500ir@checkpointtx.com Investor Relations Contact:Sandya von der WeidAssociate Director, LifeSci Advisors, LLC+41 78 680 05 38svonderweid@lifesciadvisors.com Media Relations Contact:Katie KennedyGregory FCA610-731-1045checkpoint@gregoryfca.com CHECKPOINT THERAPEUTICS, INC.CONDENSED BALANCE SHEETS(in thousands, except share and per share amounts)(Unaudited) December 31, 2024 2023 ASSETS Current Assets: Cash and cash equivalents $6,604 $4,928 Prepaid expenses and other assets 867 450 Total current assets 7,471 5,378 Total Assets $7,471 $5,378 LIABILITIES AND STOCKHOLDERS' EQUITY (DEFICIT) Current Liabilities: Accounts payable and accrued expenses $17,465 $15,485 Accounts payable and accrued expenses - related party 2,433 2,815 Common stock warrant liabilities 198 125 Total current liabilities 20,096 18,425 Total Liabilities 20,096 18,425 Commitments and Contingencies Stockholders' Equity (Deficit) Common Stock ($0.0001 par value), 175,000,000 and 80,000,000 shares authorized as of December 31, 2024 and 2023, respectively Class A common shares, 700,000 shares issued and outstanding as of December 31, 2024 and December 31, 2023 - - Common shares, 53,640,422 and 27,042,035 shares issued and outstanding as of December 31, 2024 and December 31, 2023, respectively 5 3 Common stock issuable, 2,386,808 and 1,492,915 shares as of December 31, 2024 and December 31, 2023, respectively 7,638 3,419 Additional paid-in capital 350,305 297,864 Accumulated deficit (370,573) (314,333)Total Stockholders’ Equity (Deficit) (12,625) (13,047)Total Liabilities and Stockholders’ Equity (Deficit) $7,471 $5,378 CHECKPOINT THERAPEUTICS, INC.CONDENSED STATEMENTS OF OPERATIONS(in thousands, except share and per share amounts)(Unaudited) For the year ended December 31, 2024 2023 Revenue - related party $41 $103 Operating expenses: Research and development 36,152 43,566 General and administrative 20,063 8,685 Total operating expenses 56,215 52,251 Loss from operations (56,174) (52,148) Other income (loss): Interest income 11 84 Gain (loss) on common stock warrant liabilities (73) 217 Foreign currency exchange loss (4) - Total other income (loss) (66) 301 Net Loss $(56,240) $(51,847) Loss per Share: Basic and diluted net loss per Class A common share and common share outstanding $(1.42) $(3.17) Basic and diluted weighted average number of Class A common shares and common shares outstanding 39,674,444 18,742,494

BETHESDA, Md., March 19, 2025 /PRNewswire/ -- Celebrated clinical geneticist Stephen Cederbaum, MD, PhD, FACMG, has been named the recipient of the 2025 ACMG Foundation for Genetic and Genomic Medicine's David L. Rimoin Lifetime Achievement Award in Medical Genetics. A founding member of the Society for Inherited Metabolic Disorders and the American College of Medical Genetics, Dr. Cederbaum is now a distinguished professor emeritus of Psychiatry, Pediatrics and Human Genetics at the University of California, Los Angeles (UCLA). During the height of his career at UCLA, he developed a clinical program in inborn errors of metabolism and, alongside it, became a leading expert on urea cycle disorders, most notably arginase deficiency, a disorder in which the body lacks the final enzyme in the urea cycle and is, therefore, unable to rid itself of excess ammonia. Dr. Cederbaum worked on enzyme therapy for the disorder, which remains a focus of ongoing research. From his first exposure to arginase deficiency in 1972, Dr. Cederbaum devoted decades to outlining the biochemistry and pathology that underlie the disorder, as well as identifying successful therapies to treat it. He developed animal models that enabled the exploration of gene replacement and other early therapies for arginase deficiency. "As a physician-scientist, [Dr. Cederbaum] identified the genetic basis and mechanistic consequences of arginase deficiency and the role of arginine metabolism in multiple disease conditions," said Brendan Lee, MD, PhD, chair of Molecular and Human Genetics at Baylor College of Medicine. "As such, he traversed classical biochemistry, mouse genetics, human genetics and clinical research/care. I had the privilege of meeting Dr. Cederbaum when I started my independent career at a meeting at the Urea Cycle Disorders research conference. His scientific insight—and wit—stood out to all, and his style of self-deprecating leadership in science and medicine has likewise been appreciated by both colleagues and trainees." Dr. Cederbaum did not limit his efforts to academic or clinical practice. He was also a keen advocate for public health, sponsoring a bill in California to include adults with inborn errors of metabolism in an existing Genetically Handicapped Persons Program. Dr. Cederbaum's efforts also spurred the broadening of mandated newborn screenings to include mass spectrometry, and he founded the California State Genetics Disease Branch Newborn Screening Guidelines Committee. Amid these accomplishments, he also found time to launch a support group for parents, which is now the ARD1-D Foundation; the organization now supports people with arginase deficiency and their families. He also served as an advisor to the National Urea Cycle Disorders Foundation, as well as mentoring and supporting students and colleagues. "His work ethic and humility stand out. Despite a long list of achievements, Dr. Cederbaum attributes success to his teams and colleagues. His dedication to patient care and advocacy is exemplary, with significant efforts to improve care in underserved regions, particularly in Latin America," said Marshall L. Summar, MD, ABP, ABMGG, CEO of Uncommon Cures LLC and Professor of Pediatrics at George Washington University. Dr. Cederbaum sees his connectedness more simply: "People mattered, and I always understood that one of the joys of clinical practice is just meeting people, getting to know people, the one-on-one," he says. "It may be that I'm an extreme example of someone for whom human interaction is very important, but I also always understood that my patients suffered from terrible disorders and that it really mattered how you interacted with them." A productive investigator, Dr. Cederbaum has authored or co-authored more than 180 peer-reviewed publications and more than 90 book chapters and reviews alongside his duties as a physician. Those publications include a study detailing the development of a mouse model for arginase deficiency (and gene transfer in those mice), cloning and characterizing the human and mouse types I and II arginase genes, and comparing the properties and functions of different arginases. "His combination of basic science and clinical research is almost unheard of in today's environment of ultra-specialization and sets him apart as both a pioneer and role model to the field," said Jerry Vockley, MD, PhD, chief of Genetic and Genomic Medicine and director of the Center for Rare Disease Therapy, UPMC Children's Hospital of Pittsburgh. Dr. Cederbaum's research has not only defined the diagnosis and treatment of arginase deficiency but also opened new avenues of understanding the role of arginase activity in endothelial and breast cancer cells. Outside arginase investigations, he is responsible for identifying Letterer-Siwe Disease as an immunodeficiency instead of a malignancy and for defining and developing effective therapies for pyruvate dehydrogenase deficiency, a rare disorder sparked by lactic acid buildup. Dr. Cederbaum built the foundation for his career with an undergraduate chemistry degree from Amherst College in Massachusetts in 1959, followed by a medical degree from New York University in 1963. A yearlong leave of absence before his MD allowed him to pursue bacterial genetics research at Massachusetts General Hospital. With his doctorate in hand, he completed a two-year internal medicine residency at Washington University, St. Louis, and then studied bacterial biochemistry at the National Institutes of Health. He returned to medical genetics with a 1970 fellowship at the University of Washington in Seattle. The following year, he joined UCLA, where he was a contemporary of the late Dr. Rimoin, for whom the current award is named. Dr. Cederbaum spent most of his five-decade career at UCLA, where he remains active today as a board member of the university's Faculty Club. Like many early-career researchers, Dr. Cederbaum knew the importance of choosing his research specialty. A lifelong lover of literature, he instinctively sought what Robert Frost termed "the road less taken." After meeting a few families impacted by arginase deficiency, he recognized that the disorder was poorly understood and began considering this new research path. "It was a road less traveled and an area in which I could make unique contributions. I started out having no real insight into how viable a long-term problem it was," he said. "It turned out to be one that could sustain a career for 50 years … so it turned out to be very fortunate, though I didn't have that kind of insight at the time." Dr. Cederbaum's mentees continue to pursue these studies. Before being honored by the ACMG, Dr. Cederbaum received the Sherman Mellinkoff Award, the UCLA School of Medicine's highest honor; the Edward A. Dickson Emeritus Professorship Award, UCLA; the National Organization for Rare Disorders Rare Impact Award; he was the Arno and Gretel Motulsky Lecturer, University of Washington; and was a distinguished visiting professor at the Universidad National Autónoma de México. "Dr. Stephen Cederbaum's remarkable contributions to the field of inborn errors of metabolism and newborn screening, with an emphasis on disorders of the urea cycle make him a well-qualified winner of the David L. Rimoin Lifetime Achievement Award," said Dr. Ann Garber-Rimoin, Dr. Rimoin's widow. "Using various forms of gene and mRNA therapy, his research has used both patient and animal models to improve clinical care, and to significantly impactnewborn screening policy. More recently, his research interests have been directed to the pre-symptomatic DNA screening for late-onset autosomal dominant disorders. Beyond his outstanding laboratory and clinical achievements, Dr. Cederbaum is known as a dedicated teacher and mentor, as well as a founder and supporter of patient support groups for patients with arginase deficiency. His kindness and genuine care for his patients further enhance his leadership role for his students and trainees. The Rimoin family is proud to recognize Dr. Stephen Cederbaum's outstanding career, and to honor him with the David L. Rimoin Lifetime Achievement Award." "Congratulations to Dr. Stephen Cederbaum for this well-deserved award. Dr. Cederbaum's life and work epitomizes the objective characteristics of the Rimoin award. His passion for teaching, mentoring, and care for patients and their families is admirable. He has championed the integration of clinical genomics care into the healthcare system. We are honored to be able to recognize his achievements," said Nancy J. Mendelsohn, MD, FACMG, president of the ACMG Foundation. Even after 50 years in the field, Cederbaum remains engaged with colleagues and community members. "To me, it's very important [to recognize] that you can always be better, and it's important to make yourself better," he said. "You're never finished with your obligations to society." About the David L. Rimoin Lifetime Achievement Award The David L. Rimoin Lifetime Achievement Award is the highest honor given by the ACMG Foundation. It honors professionals who have made career contributions to medical genetics. Its namesake, Dr. David L. Rimoin, was a pioneer in the field of medical genetics who advanced the understanding of skeletal dysplasia, diabetes and Tay-Sachs disease. A committee of past presidents of the ACMG selects the recipient following nominations from the general membership. About the ACMG Foundation for Genetic and Genomic Medicine The ACMG Foundation for Genetic and Genomic Medicine, a 501(c)(3) nonprofit organization, is a community of supporters and contributors who understand the importance of medical genetics and genomics in healthcare. Established in 1992, the ACMG Foundation supports the American College of Medical Genetics and Genomics (ACMG) mission to "translate genes into health." Through its work, the ACMG Foundation fosters charitable giving, promotes training opportunities to attract future medical geneticists and genetic counselors to the field, shares information about medical genetics and genomics, and sponsors important research. To learn more and support the ACMG Foundation's mission to create "Better Health through Genetics," visit . Barry Eisenberg [email protected] SOURCE American College of Medical Genetics and Genomics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

STOCKHOLM, March 19, 2025 /PRNewswire/ -- Sobi ® (STO: SOBI), today announced a research collaboration involving a new Phase 2a clinical trial for Gamifant® (emapalumab) for the potential treatment of interferon-gamma (IFNγ)-driven sepsis (IDS) which is to be presented at the International Symposium on Intensive Care and Emergency Medicine (ISICEM) Congress by Prof. Giamarellos-Bourboulis, from the Hellenic Institute for the Study of Sepsis. The study underscores Sobi's commitment to advancing medicine through targeted therapies for severe and complex conditions such as sepsis, a leading cause of mortality globally. Key Highlights IDS is a new endotype representing approximately 20 percent of sepsis patients and is characterised by detection of IFNγ (above lower limit of detection) and elevated levels of the chemokine CXCL9. The EMBRACE phase 2a study will investigate the potential of Gamifant® (emapalumab) in treating a subgroup of sepsis patients driven by the IDS endotype and absence of sepsis-induced immunoparalysis. IDS patients with a low human leukocyte antigen DR (HLA-DR) expression on monocytes characteristic of immunoparalysis will not be included into the EMBRACE study. The potential inclusion of this IDS sub-population into further studies will be evaluated based on the results of EMBRACE. The EMBRACE phase 2a trial (NCT06694701) was approved in March 2025. The first trial sites have been initiated, and patient screening to identify eligible patients with IDS has started. The trial design is to be presented at ISICEM by Prof. Evangelos Giamarellos-Bourboulis, MD, PhD, FISAC, principal investigator and sponsor of the EMBRACE study, from the Hellenic Institute for the Study of Sepsis (HISS): "The EMBRACE Phase 2a study will adopt a precision immunotherapy approach for the treatment of sepsis driven by the IDS endotype, a patient group currently having limited therapeutic choices." "We are looking forward to this research collaboration on the use of Gamifant® (emapalumab) in the new endotype of interferon gamma-driven sepsis," said Lydia Abad-Franch, MD, Head of Research & Development and Medical Affairs and Chief Medical Officer at Sobi. "We are committed to advancing science and study Gamifant® (emapalumab) in indications with a high unmet need and where excessive interferon gamma production plays a key role in driving hyperinflammation." Presentation details Emapalumab treatment for anticipated clinical benefit in sepsis driven by the interferon-gamma endotype (the EMBRACE trial): O Konstantinidou, E Olntasi, H Florou, A Reininger, E Giamarellos-Bourboulis, F EMBRACE STUDY GROUP. ISICEM 2025; Abstract number A173; Poster number P249. About sepsis and IFNγ-driven sepsis (IDS) Sepsis is a serious condition in response to an infection that can lead to organ failure and is a leading global cause of mortality. A recent large study, published in eBioMedicine in 20241, describes different sepsis endotypes, suggesting varying endotypes require differentiated treatment strategies. Approximately 20% of the patients studied are of the newly described IFNγ-driven sepsis (IDS) endotype. IDS is marked by elevated levels of CXCL9 and detection of IFNγ and poor clinical outcomes, with a 28-day mortality rate ranging from 40 to 43%. About the EMBRACE study The EMBRACE study (NCT06694701) is a Phase 2a, double-blind, randomized controlled trial planned to be conducted at 24 sites in Greece. The trial investigates whether Gamifant® (emapalumab), an anti-IFNγ antibody, can improve clinical outcomes in patients with the interferon-gamma-driven sepsis (IDS) endotype and absence of sepsis-induced immunoparalysis. IDS is characterized by elevated levels of CXCL9 and detectable IFNγ and is associated with poor patient outcomes. By targeting this inflammation pathway, the EMBRACE trial aims to reduce mortality, improve organ function, and accelerate recovery. The trial design includes three arms, and plans to enrol 75 patients, two groups receiving Gamifant® (emapalumab), (low and high doses) alongside standard-of-care treatment, and one group receiving placebo alongside standard-of-care treatment. The primary endpoint is a ≥1.4-point decrease in the Sequential Organ Failure Assessment (SOFA) score from baseline to the end of treatment (28 days). Secondary endpoints include 28-day mortality, safety, pharmacokinetics, and changes in key inflammatory biomarkers such as CRP, IL-6, ferritin, IFNγ, and CXCL9. About Gamifant® (emapalumab) Gamifant® (emapalumab) is an anti-interferon gamma (IFNγ) monoclonal antibody that binds to and neutralises IFNγ. In the USA, Gamifant® (emapalumab) is indicated for the treatment of adult and paediatric (newborn and older) patients with primary haemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy. Primary HLH is a rare syndrome of hyperinflammation that usually occurs within the first year of life and can rapidly become fatal unless diagnosed and treated. The FDA approval is based on data from the phase 2/3 studies (NCT01818492 and NCT02069899). Gamifant® (emapalumab) is indicated for administration through intravenous infusion over one hour twice per week until haematopoietic stem cell transplantation (HSCT) About Sobi® Sobi® is a specialised international biopharmaceutical company transforming the lives of people with rare and debilitating diseases. Providing reliable access to innovative medicines in the areas of haematology, immunology, and specialty care. Sobi has approximately 1,800 employees across Europe, North America, the Middle East, Asia, and Australia. In 2024, revenue amounted to SEK 26 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com and LinkedIn. 1: Interferon-gamma driven elevation of CXCL9: a new sepsis endotype independently associated with mortality Giamarellos-Bourboulis, Evangelos J. et al. eBioMedicine, Volume 109, 105414 Contacts For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. 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