Apellis Pharmaceuticals, Inc. (APLS) came out with a quarterly loss of $0.46 per share versus the Zacks Consensus Estimate of a loss of $0.32. This compares to loss of $1.17 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of -43.75%. A quarter ago, it was expected that this company would post a loss of $0.33 per share when it actually produced a loss of $0.28, delivering a surprise of 15.15%.Over the last four quarters, the compa ...

文章核心观点 - 公司在第三季度取得了持续进展,包括SYFOVRE需求增长和C3G和IC-MPGN的III期VALIANT研究结果积极[2] - 尽管SYFOVRE净销售额因毛利率调整较高而低于预期,但公司仍专注于扩大地理性萎缩患者覆盖并巩固在该市场的领导地位[2] - 公司拥有两款潜在的重磅产品、一个前景看好的管线以及实现盈利的道路,未来增长前景良好[2] SYFOVRE(pegcetacoplan注射液) - 第三季度SYFOVRE美国净产品收入为1.52亿美元[3] - SYFOVRE商业瓶装需求环比增长约7%[3] - SYFOVRE净销售价格下降,主要由于毛利率调整增加[3] - 公司仍是地理性萎缩市场的领导者,第三季度向医疗机构提供了超过88,500剂SYFOVRE,其中包括约84,500瓶商业产品和4,000瓶样品[3] - 截至2024年9月,估计已有超过420,000剂SYFOVRE注射剂被给药,包括临床试验[3] - 2025年1月1日起,一家大型Medicare Advantage计划将SYFOVRE列为其处方集的唯一优先产品[3] - 公司在欧盟CHMP获得了负面意见,但预计将在2025年初获得英国、瑞士、加拿大和澳大利亚的监管决定[3] EMPAVELI(pegcetacoplan) - 第三季度EMPAVELI美国净产品收入为2,460万美元[4] - 患者依从性保持在97%的高水平[4] 研发进展 - 根据FDA反馈,公司计划于2025年初提交针对C3G和IC-MPGN的pegcetacoplan的补充新药申请,基于VALIANT研究6个月的积极结果[5] - FDA未要求公司等待52周完整数据再提交[5] - 在2024年肾脏周上公布了VALIANT研究的详细结果,显示pegcetacoplan在C3G和原发性IC-MPGN患者中的治疗效果很强[5] - Sobi计划于2025年初向欧洲药品管理局和日本卫生当局提交pegcetacoplan的上市申请[6] - Sobi完成了评估系统性pegcetacoplan在HSCT-TMA患者中疗效和安全性的II期研究的入组,预计2025年中期公布结果[6] - APL-3007(C3沉默的小干扰RNA)I期剂量递增研究预计2025年第一季度公布结果[6] 财务数据 - 第三季度总收入为1.968亿美元,其中SYFOVRE美国净产品收入为1.52亿美元,EMPAVELI美国净产品收入为2,460万美元,以及与Sobi合作的许可和其他收入2,030万美元[8] - 第三季度销售成本为3,360万美元,较上年同期增加,主要由于存货报废和终止PEG最低采购义务产生的费用增加,部分被销售里程碑费用减少所抵消[10] - 第三季度研发费用为8,860万美元,较上年同期增加,主要由于项目相关外部成本和非项目相关外部成本增加,部分被人员相关成本减少所抵消[11] - 第三季度销售及一般管理费用为1.22亿美元,较上年同期下降,主要由于人员相关成本、商业和营销活动、办公费用以及专业和咨询费用的减少[12] - 第三季度净亏损为5,740万美元,较上年同期的1.402亿美元有所改善[12] - 截至2024年9月30日,公司现金及现金等价物为3.969亿美元,预计未来收入和现金将足以支持公司实现正现金流[13]

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File Number: 001-38276 APELLIS PHARMACEUTICALS, INC. (Exact Name of Registrant as Specified in its Charter) Delaware 27-1537290 (State or othe ...

文章核心观点 - 该新药pegcetacoplan在治疗C3肾病(C3G)和原发性免疫复合膜性肾小球肾炎(IC-MPGN)方面取得了积极的临床试验结果 [1][2][3][4] - pegcetacoplan显示了显著的肾脏保护作用,包括显著降低蛋白尿、稳定肾小球滤过率(eGFR)、大幅减少C3c沉积等 [2][3] - pegcetacoplan在广泛的患者亚组中(包括青少年、成人、原发性肾脏和移植后肾脏)均显示了一致的疗效 [2][3] - pegcetacoplan的安全性和耐受性良好,没有出现严重的不良反应 [5] - 公司计划于2025年向FDA和EMA提交上市申请 [5] 公司信息 - Apellis Pharmaceuticals是一家全球性生物制药公司,专注于开发针对补体C3的创新疗法 [9] - Apellis与Sobi公司就pegcetacoplan的全球共同开发和商业化权利达成合作 [8] 行业信息 - C3肾病(C3G)和原发性免疫复合膜性肾小球肾炎(IC-MPGN)是罕见且严重的肾脏疾病,约50%的患者在诊断后5-10年内发展为肾衰竭,需要进行肾移植或终生透析 [7] - 目前尚无针对这些疾病根本原因的治疗方法,pegcetacoplan有望成为首个获批的治疗方案 [7] - 这些疾病在美国和欧洲的患病人数估计分别为5,000人和8,000人 [7]

William Blair has initiated coverage on Apellis Pharmaceuticals, Inc. APLS and notes that Apellis was the first company to successfully target complement C3, which has led to the approval of two therapies: Empaveli (systemic pegcetacoplan) for paroxysmal nocturnal hemoglobinuria and Syfovre (intravitreal pegcetacoplan) for geographic atrophy secondary to age-related macular degeneration.William Blair writes that although Empaveli has established a solid position in the PNH market, short-term challenges are ...

Hey there! I'm a biotech and healthcare investment analyst with several years of bedside clinical experience and a Master of Business Administration. I utilize risk-return charts and DCF analyses to highlight investment opportunities and risks. I'm a big fan of the barbell strategy: 90% in safe assets like Treasuries and ETFs, and 10% in high-growth stocks—keeping risk management front and center. Since markets are pretty efficient and returns can be random, I draw inspiration from 'Superforecasting' and 'A ...

Thank you for reading my research on Seeking Alpha. If you want to learn even more about my method and how I discover these investment opportunities, please check out my subscription marketplace service, Compounding Healthcare , and sign up for a free trial.It has been almost a year since my previous Apellis Pharmaceuticals, Inc. (NASDAQ: APLS ) article , where I discussed the company's C3 inhibitor technology, as well as the prospects for their flagship treatment, pegcetacoplan. Despite my trepidation abou ...

Apellis Pharmaceuticals (APLS) announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has confirmed its June 2024 negative opinion on the regulatory filing for intravitreal pegcetacoplan to treat geographic atrophy (GA) secondary to age-related macular degeneration (AMD). Shares of the company lost 11.5% on Friday in response.Management expressed deep disappointment in the outcome, stating that millions of GA patients in the EU remain without treatmen ...

WALTHAM, Mass., Sept. 20, 2024 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has confirmed its June 2024 negative opinion on the marketing authorization application of intravitreal pegcetacoplan for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration. “We are deeply disappointed by this outcome, which leaves millions of Europeans with GA ...

Met the primary endpoint, achieving statistically significant 68% (p<0.0001) reduction in proteinuria compared to placebo in a broad study population Positive results consistent across all subgroups, including C3G and IC-MPGN, adolescent and adult patients, and native and post-transplant kidneys Demonstrated favourable safety, consistent with established profile Companies plan to submit data for regulatory approval in the US and EU STOCKHOLM, Aug. 8, 2024 /PRNewswire/ -- Sobi® (STO: SOBI) and Apellis Pha ...