Lambert-Eaton Myasthenic Syndrome

New compelling results demonstrate 18 months of both sustained reduction in immunoglobulin G antibodies and sustained improvement in gMG symptoms in pivotal Vivacity-MG3 study and open-label extension phase Up to 128 weeks and 180 patient years of follow-up in the open-label extensiona confirm a safety profile consistent with the Phase 3 Vivacity-MG3 study 45% of the patients receiving steroids at open-label extension baseline were able to decrease or discontinue their steroid use Additionally, the nipocalimab plus standard of care (SOC) group demonstrated four times greater odds of improving and maintaining the strength and function of different muscle groups as measured by QMGb response versus placebo plus SOC in the 24-week double blind phase of the study SPRING HOUSE, Pa., April 8, 2025 /PRNewswire/ -- Johnson & Johnson (NYSE: JNJ) today announced results from additional analyses of the Phase 3 Vivacity-MG3 double-blind study and the ongoing open-label extensiona (OLE), evaluating the long-term efficacy and safety of investigational nipocalimab in a broad population of antibody-positive (anti-AChR+, anti-MuSK+, anti-LRP4+) adults with generalized myasthenia gravis (gMG).1,2 Patients treated with nipocalimab plus standard of care (SOC) maintained improvements in their MG-ADLc and QMGb scores over 84 weeks with sustained reductions in total immunoglobulin G (IgG).1 These data are included in a presentation (Session 7 #022) and are among 12 abstracts that Johnson & Johnson will present at the American Academy of Neurology (AAN) 2025 Meeting in San Diego, California, which includes an oral presentation on QMG score improvements from the double-blind phase of the Phase 3 Vivacity-MG3 study. "The sustained disease control seen over 84 weeks for nipocalimab is a key result given the chronic course of generalized MG and the significant burden on people living with this condition," said Constantine Farmakidis M.D., Associate Professor of Neurology at University of Kansas Medical Centerd. "Overall, I am encouraged by these results that show improvement in disease control as measured by the MG-ADL and QMG scores across a broad population seropositive for AChR, MuSK, or LRP4 autoantibodies." Nipocalimab demonstrated a mean change in MG-ADL of -5.64 (p<0.001) from the double-blind baseline after 60 weeks in the OLE for study participants receiving nipocalimab and SOC, and -6.01 (p<0.001) mean change for study participants who transitioned from placebo and SOC to nipocalimab and SOC.1 In the antibody-positive population, 45% of patients receiving steroids at the OLE baseline were able to decrease or discontinue steroids at the time of this data cut by more than half of the baseline dose.1 Among these patients, the mean dose of prednisone decreased from 23 to 10 mg per day.1 Nipocalimab had a consistent and tolerable safety profile throughout the OLE phase.1 Additional findings from the Phase 3 Vivacity-MG3 double-blind study indicate that patients treated with nipocalimab plus SOC achieved statistically significant improvements in their QMG score by -4.9 versus placebo plus SOC (p<0.001) over weeks 22 and 24.2 Patients in the nipocalimab plus SOC treatment group were four times more likely to sustain symptom improvement at 20 weeks compared to the placebo plus SOC group, as measured by a three or greater point improvement on the QMG score.2 Results show significantly more patients treated with nipocalimab (36.4%,) versus placebo (10.5%, p<0.001) spent greater than 75% of study duration demonstrating improvements in the QMG score.2 A reduction of more than three points in the QMG score indicates a decrease in the severity of the patient's symptoms as a result of improvements in muscle strength, allowing patients to carry out important daily activities such as swallowing and chewing.3,4 "People living with generalized MG around the world endure daily challenges, such as difficulties swallowing, impaired speech and muscle weakness. They deserve additional, effective treatment options that help address these challenges and provide sustained disease control and stability over time," said Katie Abouzahr, M.D., Vice President, Autoantibody Portfolio and Maternal Fetal Immunology Disease Area Leader, Johnson & Johnson Innovative Medicine. "These positive data underscore our commitment to helping develop potential innovative therapeutic options for patients living with autoantibody diseases, including gMG." Editor's notes: ABOUT GENERALIZED MYASTHENIA GRAVIS (gMG) Myasthenia gravis (MG) is an autoantibody disease in which the immune system mistakenly makes antibodies (e.g., anti-acetylcholine receptor [AChR], anti-muscle-specific tyrosine kinase [MuSK] or anti-low density lipoprotein-related protein 4 [LRP4]), which target proteins at the neuromuscular junction, and can block or disrupt normal signaling from nerves to muscles, thus impairing or preventing muscle contraction.5,6,7 The disease impacts an estimated 700,000 people worldwide.5 The disease affects both men and women and occurs across all ages, racial and ethnic groups, but most frequently starts in young women and older men.8 Roughly 50 percent of individuals diagnosed with MG are women, and about one in five of those women are of child-bearing potential.9,10,11 Approximately 10 to 15% of new cases of MG are diagnosed in adolescents (12 – 17 years of age).12,13,14 Among juvenile MG patients, girls are affected more often than boys with over 65% of pediatric MG cases in the U.S. diagnosed in girls.15,16,17 Initial disease manifestations are usually ocular, but 85 percent of MG patients experience additional advancements to the disease manifestations – referred to as generalized myasthenia gravis (gMG). This is characterized by severe muscle weakness of the skeletal muscles and difficulties in speech and swallowing.18,19,20,21,22 Approximately 100,000 individuals in the U.S. are living with gMG.23 Vulnerable gMG populations, such as pediatric patients, have more limited therapeutic options.24 Currently, SOC treatments for adolescents with gMG are extrapolated from adult trials.14 Other than symptomatic treatments, there are no approved FcRn blockers for adolescents with gMG in the United States.14 ABOUT THE PHASE 3 VIVACITY-MG3 STUDY The Phase 3 Vivacity-MG3 study (NCT04951622) was specifically designed to measure sustained efficacy and safety with consistent dosing in this unpredictable chronic condition where unmet need remains high. Antibody positive or negative adult gMG patients with insufficient response (MG-ADL ≥6) to ongoing SOC therapy were identified and 199 patients, 153 of whom were antibody positive, enrolled in the 24-week double-blind placebo-controlled trial.25,26 Randomization was 1:1, nipocalimab plus current SOC (30 mg/kg IV loading dose followed by 15 mg/kg every two weeks) or placebo plus current SOC.25 Baseline demographics were balanced across arms (77 nipocalimab, 76 placebo).25 The primary endpoint of the study was mean change in MG-ADLb score from baseline over Weeks 22, 23 and 24 in antibody positive patients. A key secondary endpoint included change in QMG score. Long-term safety and efficacy were further assessed in an ongoing open-label extension (OLE) phase.26 ABOUT NIPOCALIMAB Nipocalimab is an investigational monoclonal antibody, designed to bind with high affinity to block FcRn and reduce levels of circulating immunoglobulin G (IgG) antibodies potentially without impact on other immune functions. This includes autoantibodies and alloantibodies that underlie multiple conditions across three key segments in the autoantibody space including Rare Autoantibody diseases, Maternal Fetal diseases mediated by maternal alloantibodies and Rheumatic diseases.26,27,28,29,30,31,32,33,34 Blockade of IgG binding to FcRn in the placenta is also believed to limit transplacental transfer of maternal alloantibodies to the fetus.35,36 The U.S. FDA and EMA have granted several key designations to nipocalimab including: U.S. FDA Fast Track designation in hemolytic disease of the fetus and newborn (HDFN) and warm autoimmune hemolytic anemia (wAIHA) in July 2019, gMG in December 2021 and fetal neonatal alloimmune thrombocytopenia (FNAIT) in March 2024 and Sjögren's disease (SjD) in March 2025 U.S. FDA Orphan drug status for wAIHA in December 2019, HDFN in June 2020, gMG in February 2021, chronic inflammatory demyelinating polyneuropathy (CIDP) in October 2021 and FNAIT in December 2023 U.S. FDA Breakthrough Therapy designation for HDFN in February 2024 and for Sjögren's disease in November 2024 U.S. FDA granted Priority Review in gMG in Q4 2024 EU EMA Orphan medicinal product designation for HDFN in October 2019 ABOUT JOHNSON & JOHNSON At Johnson & Johnson, we believe health is everything. Our strength in healthcare innovation empowers us to build a world where complex diseases are prevented, treated, and cured, where treatments are smarter and less invasive, and solutions are personal. Through our expertise in Innovative Medicine and MedTech, we are uniquely positioned to innovate across the full spectrum of healthcare solutions today to deliver the breakthroughs of tomorrow and profoundly impact health for humanity. Learn more at or at Follow us at @JNJInnovMed. Janssen Research & Development, LLC and Janssen Biotech, Inc. are Johnson & Johnson companies. CAUTIONS CONCERNING FORWARD-LOOKING STATEMENTS This press release contains "forward-looking statements" as defined in the Private Securities Litigation Reform Act of 1995 regarding product development and the potential benefits and treatment impact of nipocalimab. The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialize, actual results could vary materially from the expectations and projections of Janssen Research & Development, LLC, Janssen Biotech, Inc. and/or Johnson & Johnson. Risks and uncertainties include, but are not limited to: challenges and uncertainties inherent in product research and development, including the uncertainty of clinical success and of obtaining regulatory approvals; uncertainty of commercial success; manufacturing difficulties and delays; competition, including technological advances, new products and patents attained by competitors; challenges to patents; product efficacy or safety concerns resulting in product recalls or regulatory action; changes in behavior and spending patterns of purchasers of health care products and services; changes to applicable laws and regulations, including global health care reforms; and trends toward health care cost containment. A further list and descriptions of these risks, uncertainties and other factors can be found in Johnson & Johnson's most recent Annual Report on Form 10-K, including in the sections captioned "Cautionary Note Regarding Forward-Looking Statements" and "Item 1A. Risk Factors," and in Johnson & Johnson's subsequent Quarterly Reports on Form 10-Q and other filings with the Securities and Exchange Commission. Copies of these filings are available online at , or on request from Johnson & Johnson. None of Janssen Research & Development, LLC, Janssen Biotech, Inc. nor Johnson & Johnson undertakes to update any forward-looking statement as a result of new information or future events or developments. 1 Antozzi, C et al., Long-Term Safety and Efficacy of Nipocalimab in Generalized Myasthenia Gravis: Vivacity-MG3 Open-Label Extension Phase Results. Abstract #022 for poster presentation at 2025 American Academy of Neurology Congress. April 2025. 2 Nowak R et al., Efficacy of Nipocalimab, a Novel Neonatal Fragment Crystallizable Receptor Blocker, as Measured using Quantitative Myasthenia Gravis Assessment: Findings from the Phase 3 Placebo-Controlled Vivacity-MG3 Study. Abstract #001 for oral presentation at 2025 American Academy of Neurology Congress. April 2025. 3 Wolfe GI Myasthenia gravis activities of daily living profile. Neurology. 1999;22;52(7):1487-9. doi: 10.1212/wnl.52.7.1487. 4 Yin J, et al. A multicenter, randomized, open-label, phase 2 clinical study of telitacicept in adult patients with generalized myasthenia gravis. Eur J Neurol. 2024 Aug;31(8):e16322. doi: 10.1111/ene.16322. 5 Chen J, Tian D-C, Zhang C, et al. Incidence, mortality, and economic burden of myasthenia gravis in China: A nationwide population-based study. The Lancet Regional Health - Western Pacific. 2020;5(100063). . 6 Bacci ED et al. Understanding side effects of therapy for myasthenia gravis and their impact on daily life. BMC Neurol. 2019;19(1):335. 7 Wiendl, H., et al., Guideline for the management of myasthenic syndromes. Therapeutic advances in neurological disorders, 16, 17562864231213240. . Last accessed: April 2025. 8 Bubuioc A, et al. The epidemiology of myasthenia gravis. Journal of Medicine & Life (2021). Jan-Mar;14(1):7-16. doi: 10.25122/jml-2020-0145. 9 Ye, Yun et al. Epidemiology of myasthenia gravis in the United States. Frontiers in neurology vol. 15 1339167. 16 Feb. 2024, doi:10.3389/fneur.2024.1339167. 10 Dresser, Laura et al. Myasthenia Gravis: Epidemiology, Pathophysiology and Clinical Manifestations. Journal of clinical medicine vol. 10,11 2235. 21 May. 2021, doi:10.3390/jcm10112235. 11 J&J. Data on file. 12 Evoli A, Batocchi AP, Bartoccioni E, Lino MM, Minisci C, Tonali P. Juvenile myasthenia gravis with prepubertal onset. Neuromuscul Disord. 1998 Dec;8(8):561-7. doi: 10.1016/s0960-8966(98)00077-7. 13 Evoli A. Acquired myasthenia gravis in childhood. Curr Opin Neurol. 2010 Oct;23(5):536-40. doi: 10.1097/WCO.0b013e32833c32af. 14 Finnis MF, Jayawant S. Juvenile myasthenia gravis: a paediatric perspective. Autoimmune Dis. 2011;2011:404101. doi: 10.4061/2011/404101. 15 Haliloglu G, Anlar B, Aysun S, Topcu M, Topaloglu H, Turanli G, Yalnizoglu D. Gender prevalence in childhood multiple sclerosis and myasthenia gravis. J Child Neurol. 2002 May;17(5):390-2. doi: 10.1177/088307380201700516. 16 Parr JR, Andrew MJ, Finnis M, Beeson D, Vincent A, Jayawant S. How common is childhood myasthenia? The UK incidence and prevalence of autoimmune and congenital myasthenia. Arch Dis Child. 2014 Jun;99(6):539-42. doi: 10.1136/archdischild-2013-304788. 17 Mansukhani SA, Bothun ED, Diehl NN, Mohney BG. Incidence and Ocular Features of Pediatric Myasthenias. Am J Ophthalmol. 2019 Apr;200:242-249. doi: 10.1016/j.ajo.2019.01.004. 18 National Institute of Neurological Disorders and Stoke. Myasthenia Gravis. Available at: . Last accessed: April 2025. 19 Bever, C.T., Jr, Aquino, A.V., Penn, A.S., Lovelace, R.E. and Rowland, L.P. (1983), Prognosis of ocular myasthenia. Ann Neurol., 14: 516-519. . 20 Kupersmith MJ, Latkany R, Homel P. Development of generalized disease at 2 years in patients with ocular myasthenia gravis. Arch Neurol. 2003 Feb;60(2):243-8. doi: 10.1001/archneur.60.2.243. PMID: 12580710. 21 Myasthenia gravis fact sheet. Retrieved April 2024 from . 22 Myasthenia Gravis: Treatment & Symptoms. (2021, April 7). Retrieved April 2024 from . 23 DRG EPI (2021) & Optum Claims Analysis Jan 2012-December 2020. 24 O'Connell K, Ramdas S, Palace J. Management of Juvenile Myasthenia Gravis. Front Neurol. 2020 Jul 24;11:743. doi: 10.3389/fneur.2020.00743. PMID: 32793107; PMCID: PMC7393473. 25 Antozzi, C et al., Nipocalimab in Generalized Myasthenia Gravis: Results from a Double-Blind, Placebo-Controlled, Randomized Phase 3 Study. Abstract at 2024 European Academy of Neurology Congress. June 2024. 26 ClinicalTrials.gov Identifier: NCT04951622. Available at: . Last accessed: April 2025. 27 ClinicalTrials.gov. NCT03842189. Available at: . Last accessed: April 2025. 28 ClinicalTrials.gov Identifier: NCT05327114. Available at: . Last accessed: April 2025. 29 ClinicalTrials.gov Identifier: NCT04119050. Available at: . Last accessed: April 2025. 30 ClinicalTrials.gov Identifier: NCT05379634. Available at: . Last accessed: April 2025. 31 ClinicalTrials.gov Identifier: NCT05912517. Available at: . Last accessed: April 2025. 32 ClinicalTrials.gov Identifier: NCT06028438. Available at: . Last accessed: April 2025. 33 ClinicalTrials.gov Identifier: NCT04968912. Available at: . Last accessed: April 2025. 34 ClinicalTrials.gov Identifier: NCT04882878. Available at: . Last accessed: April 2025. 35 Lobato G, Soncini CS. Relationship between obstetric history and Rh(D) alloimmunization severity. Arch Gynecol Obstet. 2008 Mar;277(3):245-8. DOI: 10.1007/s00404-007-0446-x. 36 Roy S, Nanovskaya T, Patrikeeva S, et al. M281, an anti-FcRn antibody, inhibits IgG transfer in a human ex vivo placental perfusion model. Am J Obstet Gynecol. 2019;220(5):498 e491-498 e499. SOURCE Johnson & Johnson WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

CORAL GABLES, Fla., April 08, 2025 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. ("Catalyst" or "Company") (Nasdaq: CPRX), a commercial-stage biopharmaceutical company focused on in-licensing, developing, and commercializing novel medicines for patients living with rare and difficult-to-treat diseases, today reported that its sub-licensee in Canada, Kye Pharmaceuticals, Inc. (Kye), has announced that Health Canada has accepted the New Drug Submission (NDS) for AGAMREE®, a novel corticosteroid treatment for Duchenne muscular dystrophy (DMD), for review. The submission has been granted Priority Review, expediting the regulatory process with the potential for marketing authorization before the end of 2025. If approved, AGAMREE would be the first and only treatment option indicated for patients diagnosed with DMD in Canada. “We are pleased that Health Canada has accepted the New Drug Submission for AGAMREE with a Priority Review, recognizing its potential to address a significant unmet need for patients with Duchenne muscular dystrophy in Canada,” stated Richard J. Daly, President and CEO of Catalyst. “In collaboration with our Canadian sub-licensee, Kye, we remain committed to bringing this innovative therapy to DMD patients and ensuring access, particularly for those in underserved communities.” AGAMREE (40 mg/mL oral suspension) received U.S. Food and Drug Administration (FDA) approval on October 26, 2023, for the treatment of DMD in patients aged two years and older and became commercially available in the U.S. on March 13, 2024. Kye currently markets FIRDAPSE®, Catalyst’s flagship product for the treatment of Lambert-Eaton myasthenic syndrome (LEMS), in Canada. LEMS is a rare neuromuscular disorder characterized by debilitating and progressive muscle weakness and fatigue. About Duchenne Muscular Dystrophy Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by progressive muscle degeneration and weakness. It primarily affects males, with symptoms typically appearing in early childhood, around ages three to five. DMD is caused by mutations in the gene that encodes dystrophin, a protein that plays a crucial role in maintaining the structure and function of muscle fibers. Without dystrophin, muscle cells become fragile and easily damaged, leading to progressive muscle degeneration. Symptoms of DMD usually begin with difficulty in walking, frequent falls, and muscle weakness, particularly in the legs and pelvis. As the disease progresses, individuals may experience difficulty standing, climbing stairs, and eventually, complete loss of mobility. Other complications can include respiratory and cardiac issues due to muscle weakness. About AGAMREE® (vamorolone) AGAMREE's unique mode of action is based on differential effects on glucocorticoid and mineralocorticoid receptors and modifying further downstream activity. As such, it is considered a novel corticosteroid designed to achieve dissociative properties while maintaining efficacy that has the potential to demonstrate comparable efficacy to steroids, with the potential for a better-tolerated side effect profile. This mechanism of action may allow AGAMREE to emerge as an effective alternative to the current standard of care corticosteroids in children, adolescents, and adult patients with DMD. In the pivotal VISION-DMD study, AGAMREE met the primary endpoint Time to Stand (TTSTAND) velocity versus placebo (p=0.002) at 24 weeks of treatment and showed a good safety and tolerability profile. The most commonly reported adverse events versus placebo from the VISION-DMD study were cushingoid features, psychiatric disorders, vomiting, weight increases, and vitamin D deficiency. Adverse events were generally of mild to moderate severity. About Kye Pharmaceuticals Kye Pharmaceuticals is a growth-stage Canadian specialty pharmaceutical company committed to bringing value to Canadians by identifying, licensing, and commercializing novel prescription medicines that may not otherwise be available to patients across Canada. With a growing pipeline of novel medicines, Kye's portfolio spans a range of therapeutic areas, including cardiology, psychiatry, pediatrics, rare diseases, hematology, and neurology. Kye Pharmaceuticals is a private company headquartered in Toronto focused on bringing medications to the Canadian market which fulfill clinically significant unmet needs. Kye is committed to licensing and launching medicines that matter by delivering better outcomes to our partners, Canadian healthcare professionals, and, most importantly, patients across Canada. For more information about the company, its management, portfolio and pipeline, please visit www.kyepharma.com About Catalyst Pharmaceuticals Catalyst Pharmaceuticals, Inc. (Nasdaq: CPRX) is a biopharmaceutical company committed to improving the lives of patients with rare diseases. With a proven track record of bringing life-changing treatments to the market, we focus on in-licensing, commercializing, and developing innovative therapies. Guided by our deep commitment to patient care, we prioritize accessibility, ensuring patients receive the care they need through a comprehensive suite of support services designed to provide seamless access and ongoing assistance. Catalyst maintains a well-established U.S. presence while actively seeking to expand its global commercial footprint through strategic partnerships. Catalyst, headquartered in Coral Gables, Fla., was recognized on the Forbes 2025 list as one of America's Most Successful Mid-Cap Companies and on the 2024 Deloitte Technology Fast 500™ list as one of North America’s Fastest-Growing Companies. For more information, please visit Catalyst's website at www.catalystpharma.com. Forward-Looking Statements This press release contains forward-looking statements, as that term is defined in the Private Securities Litigation Reform Act of 1995. Forward-looking statements involve known and unknown risks and uncertainties, which may cause Catalyst's actual results in future periods to differ materially from forecasted results. A number of factors, including (i) whether Health Canada will approve AGAMREE either in the anticipated timeline or at all, (ii) if approved, whether Kye can successfully commercialize AGAMREE in Canada, and (iii) those factors described in Catalyst's Annual Report on Form 10-K for the fiscal year 2024 and its subsequent filings with the U.S. Securities and Exchange Commission (“SEC”), could adversely affect Catalyst. Copies of Catalyst's filings with the SEC are available from the SEC, may be found on Catalyst's website, or may be obtained upon request from Catalyst. Catalyst does not undertake any obligation to update the information contained herein, which speaks only as of this date. Source: Catalyst Pharmaceuticals, Inc. Source: Catalyst Pharmaceuticals, Inc.

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