Iritis

29岁的林女士，这一年多来总是双眼反复发红、刺痛、畏光，几乎每个月都要跑一次眼科门诊。她被诊断为“虹膜睫状体炎”，点了无数眼药水，症状稍缓后又反复。“是不是干眼症太严重？”、“是不是过敏？”、“是不是老熬夜？”她问过许多医生，也试过各种护眼方式，但病情始终反复。直到这一次来院复查，眼科医生发现她眼部异常并不仅仅是炎症那么简单。双眼表层巩膜呈散在结节状充血，后部角膜缘竟出现了新生血管和“影子血管”，更关键的是：她还提到了“最近常常头晕”！“有没有听力下降？耳鸣？耳闷？”医生一问，林女士一愣：“确实有点耳闷，没太在意。”种种线索拼在一起，医生高度怀疑：这不是单纯的眼病，而是罕见的Cogan综合征！累及眼、听觉-前庭系统的综合征1945年Cogan首次详细描述一种听觉-前庭障碍伴发非梅毒性基质性角膜炎的独立临床病例，将其命名为Cogan综合征。该病是一种累及眼、听觉-前庭系统的综合征，属于自身免疫性疾病，主要表现为非梅毒性角膜基质炎、前庭功能障碍、听力下降以及全身症状。该病临床上较罕见，好发于青壮年，无年龄差异，文献报道白种人居多。角膜基质炎大多数患者都会出现眼红、畏光及眼痛等，很少无症状。该病患者大多数都是双眼相继或同时发病，症状随病程进展逐日加重。分型Cogan综合征分为典型与非典型两种类型。典型Cogan综合征表现为非梅毒性角膜炎和美尼尔听觉-前庭障碍。诊断典型的综合征主要依据非梅毒性角膜基质炎、急性发作的感音神经性听力损失、类似美尼尔病的前庭症状及发病两年内听力进行性损失致聋。眼部症状除了基质性角膜炎外，还可有结膜炎、虹膜炎和睑板腺囊肿等。非典型的Cogan综合征眼部损伤多表现为不伴基质性角膜炎的其他病变。另一种非典型的Cogan综合征眼部表现具有典型的基质性角膜炎症状，但耳部症状为非美尼尔听觉-前庭障碍，或者眼耳症状不同时发生（一般2年内先后发生）。至今该病仍然没有确切的诊断标准，但是可以根据患者的症状体征及鉴别诊断对其进行确诊。临床表现该病首发症状可以是眼部炎症性反应，也可以是眼部症状伴发内耳症状。1.内耳症状：表现为骤然出现的类似美尼尔病样发作：眩晕，恶心，呕吐，共济失调，耳鸣，突发性感音神经性聋。内耳症状可与眼部症状同时出现，也可以晚于眼部症状数周或2年内先后出现。2.眼部表现：可出现较多变异，但最典型的眼部症状是非梅毒性间质性角膜炎，表现为眼红、畏光、眼睛不适、疼痛和视物模糊。在裂隙灯下可见斑片状颗粒状角膜浸润，反复发作患者可出现云状角膜变性。3.耳部损害：听力进行性下降为最主要的耳部损害，发病2年内，有54%典型的和37%非典型的Cogan综合征患者会发生单耳或双耳的耳聋。经过抗炎与全身激素治疗，听力较视力下降的改善较差。4.全身其他系统：引起其他系统症状。如，呼吸系统的上呼吸道感染，可见胸痛、呼吸困难等；心血管系统可见主动脉炎、心包炎或者心律失常、高血压等；消化系统可见腹部疼痛不适、消化不良、腹泻、便血、肝脾肿大等；以及皮肤可见红斑、风疹，也可出现多发性关节炎或关节痛。治疗糖皮质激素是治疗CS的基本药物，宜早期大量应用，起效后逐渐减量维持。糖皮质激素无效时可联用免疫抑制剂。结膜炎、巩膜炎、角膜炎、前葡萄膜炎局部应用糖皮质激素有效。无效时可用环孢霉素A滴眼液，严重眼部炎症需服用泼尼松。参考资料1. 范梓晰，靳荷，黄里佳,等. Cogan综合征二例. 中国实用眼科杂志，2015，33(02):200-201.2. 李雪静,陈楠.老年Cogan综合征1例[J].中国现代医学杂志,2019,29(09):126.3.毛秋月,杨涛,彭安全,等.Cogan综合征诊治的研究进展[J].中华耳科学杂志,2023,21(01):115-120.来源 | 梅斯医学编辑 | wanny神经系统罕见病交流群↓点击下方“阅读原文”，下载梅斯医学APP吧！

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

Reported $781.4 million in full year 2024 revenues, representing 97% year-over-year growth SYFOVRE® (pegcetacoplan injection) full year 2024 net product revenue of $611.9 million EMPAVELI® (pegcetacoplan) full year 2024 net product revenue of $98.1 million Submitted a supplemental new drug application (sNDA) for approval of EMPAVELI for C3G and primary IC-MPGN; U.S. launch anticipated in 2H 2025, if approved On track to initiate two Phase 3 trials of EMPAVELI in focal segmental glomerulosclerosis (FSGS) and in delayed graft function (DGF) in 2H 2025 Cash and cash equivalents of $411.3 million as of December 31, 2024; projected revenues and cash expected to be sufficient to fund operations to profitability Management to host conference call today at 8:30 a.m. ET SYFOVRE® (pegcetacoplan injection) full year 2024 net product revenue of $611.9 million EMPAVELI® (pegcetacoplan) full year 2024 net product revenue of $98.1 million WALTHAM, Mass., Feb. 28, 2025 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), today announced its fourth quarter and full year 2024 financial results and business highlights. “Apellis made significant strides in 2024, highlighted by the continued growth of SYFOVRE and the unprecedented phase 3 results for EMPAVELI in C3G and IC-MPGN,” said Cedric Francois, M.D., Ph.D., chief executive officer at Apellis. “With two potential blockbuster products, a promising pipeline to fuel long-term growth, and a strong financial foundation, we are well positioned for continued growth in 2025 and beyond.” Fourth Quarter and Full Year 2024 Business Highlights and Upcoming Milestones Transforming the treatment of geographic atrophy (GA) secondary to age-related macular degeneration SYFOVRE: Reported $167.8 million and $611.9 million in SYFOVRE U.S. net product revenue for the fourth quarter and full year 2024, respectively. Delivered approximately 94,000 SYFOVRE doses to physician practices in the fourth quarter, including approximately 89,000 commercial vials and 4,600 samples. More than 510,000 SYFOVRE injections are estimated to have been administered since launch through December 2024, including clinical trials. Presented 48-month data from the GALE extension study at The Macula Society Annual Meeting in February 2025 demonstrating that early treatment with SYFOVRE leads to preservation of retinal tissue at magnitudes of approximately 1.5 disc areas, which is equivalent to the size of 2 foveal areas, in nonsubfoveal patients dosed monthly. Received approval in Australia from Therapeutic Goods Administration (TGA) for every-other-month treatment of adult patients with GA with an intact fovea and when central vision is threatened by GA lesion growth. APL-3007 (siRNA) + SYFOVRE Expect initiation of a Phase 2 multi-dose study of APL-3007 + SYFOVRE in 2Q 2025; potential next generation treatment aimed at comprehensively blocking complement activity in the retina and choroid. Reported $167.8 million and $611.9 million in SYFOVRE U.S. net product revenue for the fourth quarter and full year 2024, respectively. Delivered approximately 94,000 SYFOVRE doses to physician practices in the fourth quarter, including approximately 89,000 commercial vials and 4,600 samples. More than 510,000 SYFOVRE injections are estimated to have been administered since launch through December 2024, including clinical trials. Presented 48-month data from the GALE extension study at The Macula Society Annual Meeting in February 2025 demonstrating that early treatment with SYFOVRE leads to preservation of retinal tissue at magnitudes of approximately 1.5 disc areas, which is equivalent to the size of 2 foveal areas, in nonsubfoveal patients dosed monthly. Received approval in Australia from Therapeutic Goods Administration (TGA) for every-other-month treatment of adult patients with GA with an intact fovea and when central vision is threatened by GA lesion growth. More than 510,000 SYFOVRE injections are estimated to have been administered since launch through December 2024, including clinical trials. Expect initiation of a Phase 2 multi-dose study of APL-3007 + SYFOVRE in 2Q 2025; potential next generation treatment aimed at comprehensively blocking complement activity in the retina and choroid. Maximizing EMPAVELI impact in rare diseases Paroxysmal nocturnal hemoglobinuria (PNH): Recorded $23.4 million and $98.1 million in EMPAVELI U.S. net product revenue for the fourth quarter and full year 2024, respectively. Continued high patient compliance rates of 97%. C3 glomerulopathy (C3G) and primary immune complex glomerulonephritis (IC-MPGN): Submitted a sNDA for approval of EMPAVELI based on positive Phase 3 VALIANT results at six months; if approved, U.S. launch expected in 2H 2025. Sobi received validation from the European Medicines Agency for its indication extension application for Aspaveli® (pegcetacoplan). Focal segmental glomerulosclerosis (FSGS) and delayed graft function (DGF) Expect initiation of two Phase 3 studies in 2H 2025, one in FSGS and one in DGF, two rare kidney diseases in which the complement pathway plays a significant role and there are no approved therapies. Recorded $23.4 million and $98.1 million in EMPAVELI U.S. net product revenue for the fourth quarter and full year 2024, respectively. Continued high patient compliance rates of 97%. Submitted a sNDA for approval of EMPAVELI based on positive Phase 3 VALIANT results at six months; if approved, U.S. launch expected in 2H 2025. Sobi received validation from the European Medicines Agency for its indication extension application for Aspaveli® (pegcetacoplan). Expect initiation of two Phase 3 studies in 2H 2025, one in FSGS and one in DGF, two rare kidney diseases in which the complement pathway plays a significant role and there are no approved therapies. Advancing innovative pipeline, leveraging complement expertise Advancing investigational pre-clinical research for one-time neonatal Fc receptor (FcRn) treatment using gene editing technology from Beam Therapeutics. Organizational Updates David Acheson, previously the North America senior vice president of commercial, is now serving as the executive vice president of commercial following the recent departure of Adam Townsend, chief operating officer. Mr. Acheson joined Apellis in 2019 and has led the successful U.S. launches of EMPAVELI and SYFOVRE. Keli Walbert was recently appointed to the Apellis Board of Directors. Ms. Walbert brings more than two decades of biopharmaceutical commercial leadership experience to the Board. She most recently served as executive vice president, U.S. commercial, at Horizon Therapeutics, where she was responsible for driving commercial strategy and organizational development. Fourth Quarter and Full Year 2024 Financial Results Total Revenue. Total revenue was $212.5 million for the fourth quarter of 2024, which consisted of $167.8 million in U.S. net product revenue of SYFOVRE, $23.4 million in U.S. net product revenue of EMPAVELI and $21.4 million in licensing and other revenue associated with the Sobi collaboration. Total revenue was $146.4 million for the fourth quarter of 2023, which consisted of $114.3 million in U.S. net product revenue of SYFOVRE, $24.4 million in U.S. net product revenue of EMPAVELI and $7.7 million in licensing and other revenue associated with the Sobi collaboration. For the full year 2024, total revenue was $781.4 million, which consisted of $611.8 million in U.S. net product revenue of SYFOVRE, $98.1 million in U.S. net product revenue of EMPAVELI and $71.4 million in licensing and other revenue associated with the Sobi collaboration. For the full year 2023, total revenue was $396.6 million, which consisted of $275.2 million in U.S. net product revenue of SYFOVRE, $91.0 million in U.S. net product revenue of EMPAVELI and $30.3 million in licensing and other revenue associated with the Sobi collaboration. Total revenue was $146.4 million for the fourth quarter of 2023, which consisted of $114.3 million in U.S. net product revenue of SYFOVRE, $24.4 million in U.S. net product revenue of EMPAVELI and $7.7 million in licensing and other revenue associated with the Sobi collaboration. For the full year 2023, total revenue was $396.6 million, which consisted of $275.2 million in U.S. net product revenue of SYFOVRE, $91.0 million in U.S. net product revenue of EMPAVELI and $30.3 million in licensing and other revenue associated with the Sobi collaboration. Cost of Sales. Cost of sales was $40.9 million for the fourth quarter of 2024, compared to $19.9 million for the fourth quarter of 2023. For the full year 2024, cost of sales was $117.7 million as compared to $58.5 million for the full year 2023. Cost of sales consists primarily of costs associated with the manufacturing of SYFOVRE and EMPAVELI, royalties owed to our licensor for such sales, costs associated with Sobi revenue, and certain period costs. Cost of sales consists primarily of costs associated with the manufacturing of SYFOVRE and EMPAVELI, royalties owed to our licensor for such sales, costs associated with Sobi revenue, and certain period costs. R&D Expenses. R&D expenses were $76.4 million for the fourth quarter of 2024, compared to $69.3 million for the fourth quarter in 2023. For the full year 2024, R&D expenses were $327.6 million compared to $354.4 million for the full year 2023. The decrease in R&D expenses for the full year ended December 31, 2024, was primarily attributable to a decrease in compensation and related personnel costs, which was partially offset by an increase in program specific external costs. The decrease in R&D expenses for the full year ended December 31, 2024, was primarily attributable to a decrease in compensation and related personnel costs, which was partially offset by an increase in program specific external costs. Selling, General and Administrative (SG&A) Expenses. SG&A expenses were $121.5 million for the fourth quarter of 2024, compared to $141.7 million for the fourth quarter in 2023. For the full year 2024, G&A expenses were $501.1 million compared to $500.8 million for the full year 2023. The increase was primarily attributable to an increase in office expenses, an increase in factoring fees, an increase in travel expenses, and an increase in insurance expenses, which were partially offset by a decrease in professional and consulting fees and a decrease in personnel-related costs. The increase was primarily attributable to an increase in office expenses, an increase in factoring fees, an increase in travel expenses, and an increase in insurance expenses, which were partially offset by a decrease in professional and consulting fees and a decrease in personnel-related costs. Net Loss. Apellis reported a net loss of $36.4 million and $197.9 million for the fourth quarter and full year 2024, respectively, compared to a net loss of $88.5 million and $528.6 million for the same periods in 2023. Cash. As of December 31, 2024, Apellis had $411.3 million in cash and cash equivalents, compared to $351.2 million in cash and cash equivalents as of December 31, 2023. Apellis anticipates its cash, combined with expected product revenues, will be sufficient to fund its projected operating expenses and capital expenditures to profitability. Conference Call and WebcastApellis will host a conference call and webcast to discuss its fourth quarter and full year 2024 financial results and business highlights today, February 28, 2025, at 8:30 a.m. ET. To access the live call by phone, please pre-register for the call here. A live audio webcast of the event and accompanying slides may also be accessed through the “Events and Presentations” page of the “Investors and Media” section of the company’s website. A replay of the webcast will be available for 30 days following the event. About SYFOVRE® (pegcetacoplan injection)SYFOVRE® (pegcetacoplan injection) is the first-ever approved therapy for geographic atrophy (GA). By targeting C3, SYFOVRE is designed to provide comprehensive control of the complement cascade, part of the body’s immune system. SYFOVRE is approved in the United States and Australia. About EMPAVELI®/Aspaveli® (pegcetacoplan)EMPAVELI®/Aspaveli® (pegcetacoplan) is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body’s immune system, which can lead to the onset and progression of many serious diseases. It is approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in the United States, European Union, and other countries globally. The therapy is also under investigation for several other rare diseases across hematology and nephrology. U.S. Important Safety Information for SYFOVRE® (pegcetacoplan injection) CONTRAINDICATIONS SYFOVRE is contraindicated in patients with ocular or periocular infections, in patients with active intraocular inflammation, and in patients with hypersensitivity to pegcetacoplan or any of the excipients in SYFOVRE. Systemic hypersensitivity reactions (e.g., anaphylaxis, rash, urticaria) have occurred. WARNINGS AND PRECAUTIONS Endophthalmitis and Retinal Detachments Intravitreal injections, including those with SYFOVRE, may be associated with endophthalmitis and retinal detachments. Proper aseptic injection technique must always be used when administering SYFOVRE to minimize the risk of endophthalmitis. Patients should be instructed to report any symptoms suggestive of endophthalmitis or retinal detachment without delay and should be managed appropriately. Retinal Vasculitis and/or Retinal Vascular Occlusion Retinal vasculitis and/or retinal vascular occlusion, typically in the presence of intraocular inflammation, have been reported with the use of SYFOVRE. Cases may occur with the first dose of SYFOVRE and may result in severe vision loss. Discontinue treatment with SYFOVRE in patients who develop these events. Patients should be instructed to report any change in vision without delay. Neovascular AMD In clinical trials, use of SYFOVRE was associated with increased rates of neovascular (wet) AMD or choroidal neovascularization (12% when administered monthly, 7% when administered every other month and 3% in the control group) by Month 24. Patients receiving SYFOVRE should be monitored for signs of neovascular AMD. In case anti-Vascular Endothelial Growth Factor (anti-VEGF) is required, it should be given separately from SYFOVRE administration. Intraocular Inflammation In clinical trials, use of SYFOVRE was associated with episodes of intraocular inflammation including: vitritis, vitreal cells, iridocyclitis, uveitis, anterior chamber cells, iritis, and anterior chamber flare. After inflammation resolves, patients may resume treatment with SYFOVRE. Increased Intraocular Pressure Acute increase in IOP may occur within minutes of any intravitreal injection, including with SYFOVRE. Perfusion of the optic nerve head should be monitored following the injection and managed as needed. Intravitreal injections, including those with SYFOVRE, may be associated with endophthalmitis and retinal detachments. Proper aseptic injection technique must always be used when administering SYFOVRE to minimize the risk of endophthalmitis. Patients should be instructed to report any symptoms suggestive of endophthalmitis or retinal detachment without delay and should be managed appropriately. Retinal vasculitis and/or retinal vascular occlusion, typically in the presence of intraocular inflammation, have been reported with the use of SYFOVRE. Cases may occur with the first dose of SYFOVRE and may result in severe vision loss. Discontinue treatment with SYFOVRE in patients who develop these events. Patients should be instructed to report any change in vision without delay. In clinical trials, use of SYFOVRE was associated with increased rates of neovascular (wet) AMD or choroidal neovascularization (12% when administered monthly, 7% when administered every other month and 3% in the control group) by Month 24. Patients receiving SYFOVRE should be monitored for signs of neovascular AMD. In case anti-Vascular Endothelial Growth Factor (anti-VEGF) is required, it should be given separately from SYFOVRE administration. In clinical trials, use of SYFOVRE was associated with episodes of intraocular inflammation including: vitritis, vitreal cells, iridocyclitis, uveitis, anterior chamber cells, iritis, and anterior chamber flare. After inflammation resolves, patients may resume treatment with SYFOVRE. Acute increase in IOP may occur within minutes of any intravitreal injection, including with SYFOVRE. Perfusion of the optic nerve head should be monitored following the injection and managed as needed. ADVERSE REACTIONS Most common adverse reactions (incidence ≥5%) are ocular discomfort, neovascular age-related macular degeneration, vitreous floaters, conjunctival hemorrhage. Please see accompanying full Prescribing Information for more information. U.S. Important Safety Information for EMPAVELI® (pegcetacoplan) BOXED WARNING: SERIOUS INFECTIONS CAUSED BY ENCAPSULATED BACTERIA EMPAVELI, a complement inhibitor, increases the risk of serious infections, especially those caused by encapsulated bacteria, such as Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type B. Life-threatening and fatal infections with encapsulated bacteria have occurred in patients treated with complement inhibitors. These infections may become rapidly life-threatening or fatal if not recognized and treated early. Complete or update vaccination for encapsulated bacteria at least 2 weeks prior to the first dose of EMPAVELI, unless the risks of delaying therapy with EMPAVELI outweigh the risks of developing a serious infection. Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for vaccinations against encapsulated bacteria in patients receiving a complement inhibitor. Patients receiving EMPAVELI are at increased risk for invasive disease caused by encapsulated bacteria, even if they develop antibodies following vaccination. Monitor patients for early signs and symptoms of serious infections and evaluate immediately if infection is suspected. Because of the risk of serious infections caused by encapsulated bacteria, EMPAVELI is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the EMPAVELI REMS. CONTRAINDICATIONS Hypersensitivity to pegcetacoplan or to any of the excipients For initiation in patients with unresolved serious infection caused by encapsulated bacteria including Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type B WARNINGS AND PRECAUTIONS Serious Infections Caused by Encapsulated Bacteria EMPAVELI, a complement inhibitor, increases a patient’s susceptibility to serious, life-threatening, or fatal infections caused by encapsulated bacteria including Streptococcus pneumoniae, Neisseria meningitidis (caused by any serogroup, including non-groupable strains), and Haemophilus influenzae type B. Life-threatening and fatal infections with encapsulated bacteria have occurred in both vaccinated and unvaccinated patients treated with complement inhibitors. The initiation of EMPAVELI treatment is contraindicated in patients with unresolved serious infection caused by encapsulated bacteria. Complete or update vaccination against encapsulated bacteria at least 2 weeks prior to administration of the first dose of EMPAVELI, according to the most current ACIP recommendations for patients receiving a complement inhibitor. Revaccinate patients in accordance with ACIP recommendations considering the duration of therapy with EMPAVELI. Note that, ACIP recommends an administration schedule in patients receiving complement inhibitors that differs from the administration schedule in the vaccine prescribing information. If urgent EMPAVELI therapy is indicated in a patient who is not up to date with vaccines against encapsulated bacteria according to ACIP recommendations, provide the patient with antibacterial drug prophylaxis and administer these vaccines as soon as possible. The benefits and risks of treatment with EMPAVELI, as well as the benefits and risks of antibacterial drug prophylaxis in unvaccinated or vaccinated patients, must be considered against the known risks for serious infections caused by encapsulated bacteria. Vaccination does not eliminate the risk of serious encapsulated bacterial infections, despite development of antibodies following vaccination. Closely monitor patients for early signs and symptoms of serious infection and evaluate patients immediately if an infection is suspected. Inform patients of these signs and symptoms and instruct patients to seek immediate medical care if these signs and symptoms occur. Promptly treat known infections. Serious infection may become rapidly life-threatening or fatal if not recognized and treated early. Consider interruption of EMPAVELI in patients who are undergoing treatment for serious infections. EMPAVELI is available only through a restricted program under a REMS. EMPAVELI REMS EMPAVELI is available only through a restricted program under a REMS called EMPAVELI REMS, because of the risk of serious infections caused by encapsulated bacteria. Notable requirements of the EMPAVELI REMS include the following: Under the EMPAVELI REMS, prescribers must enroll in the program. Prescribers must counsel patients about the risks, signs, and symptoms of serious infections caused by encapsulated bacteria, provide patients with the REMS educational materials, ensure patients are vaccinated against encapsulated bacteria at least 2 weeks prior to the first dose of EMPAVELI, prescribe antibacterial drug prophylaxis if patients’ vaccine status is not up to date and treatment must be started urgently, and provide instructions to always carry the Patient Safety Card both during treatment, as well as for 2 months following last dose of EMPAVELI. Pharmacies that dispense EMPAVELI must be certified in the EMPAVELI REMS and must verify prescribers are certified. Further information is available at www.empavelirems.com or 1-888-343-7073. Infusion-Related Reactions Systemic hypersensitivity reactions (e.g., facial swelling, rash, urticaria) have occurred in patients treated with EMPAVELI. One patient (less than 1% in clinical studies) experienced a serious allergic reaction which resolved after treatment with antihistamines. If a severe hypersensitivity reaction (including anaphylaxis) occurs, discontinue EMPAVELI infusion immediately, institute appropriate treatment, per standard of care, and monitor until signs and symptoms are resolved. Monitoring PNH Manifestations after Discontinuation of EMPAVELI After discontinuing treatment with EMPAVELI, closely monitor for signs and symptoms of hemolysis, identified by elevated LDH levels along with sudden decrease in PNH clone size or hemoglobin, or reappearance of symptoms such as fatigue, hemoglobinuria, abdominal pain, dyspnea, major adverse vascular events (including thrombosis), dysphagia, or erectile dysfunction. Monitor any patient who discontinues EMPAVELI for at least 8 weeks to detect hemolysis and other reactions. If hemolysis, including elevated LDH, occurs after discontinuation of EMPAVELI, consider restarting treatment with EMPAVELI. Interference with Laboratory Tests There may be interference between silica reagents in coagulation panels and EMPAVELI that results in artificially prolonged activated partial thromboplastin time (aPTT); therefore, avoid the use of silica reagents in coagulation panels. ADVERSE REACTIONS Most common adverse reactions in patients with PNH (incidence ≥10%) were injection site reactions, infections, diarrhea, abdominal pain, respiratory tract infection, pain in extremity, hypokalemia, fatigue, viral infection, cough, arthralgia, dizziness, headache, and rash. USE IN SPECIFIC POPULATIONS Females of Reproductive Potential EMPAVELI may cause embryo-fetal harm when administered to pregnant women. Pregnancy testing is recommended for females of reproductive potential prior to treatment with EMPAVELI. Advise female patients of reproductive potential to use effective contraception during treatment with EMPAVELI and for 40 days after the last dose. Please see full Prescribing Information, including Boxed WARNING regarding serious infections caused by encapsulated bacteria, and Medication Guide. About ApellisApellis Pharmaceuticals, Inc. is a global biopharmaceutical company that combines courageous science and compassion to develop life-changing therapies for some of the most challenging diseases patients face. We ushered in the first new class of complement medicine in 15 years and now have two approved medicines targeting C3. These include the first-ever therapy for geographic atrophy, a leading cause of blindness around the world. We believe we have only begun to unlock the potential of targeting C3 across many serious diseases. For more information, please visit http://apellis.com or follow us on X (formerly Twitter) and LinkedIn. Apellis Forward-Looking StatementStatements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute “forward-looking statements” within the meaning of The Private Securities Litigation Reform Act of 1995. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including whether the results of the Company’s clinical trials for EMPAVELI, SYFOVRE, or any of its future products will warrant regulatory submissions to the FDA or equivalent foreign regulatory agencies; whether systemic pegcetacoplan will receive approval from the FDA or equivalent foreign regulatory agencies for C3G and IC-MPGN or any other indication when expected or at all; rate and degree of market acceptance and clinical utility of EMPAVELI, SYFOVRE and any future products for which we receive marketing approval will impact our commercialization efforts; whether SYFOVRE will receive approval from foreign regulatory agencies for GA when expected or at all; whether the Company’s clinical trials will be completed when anticipated; whether results obtained in clinical trials will be indicative of results that will be generated in future clinical trials or in the real world setting; whether the period for which the Company believes that its cash resources will be sufficient to fund its operations; and other factors discussed in the “Risk Factors” section of Apellis’ Annual Report on Form 10-K with the Securities and Exchange Commission on February 28, 2025 and the risks described in other filings that Apellis may make with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Apellis specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise. Media Contact:Lissa Pavlukmedia@apellis.com617.977.6764 Investor Contact:Meredith Kayameredith.kaya@apellis.com617.599.8178