Apellis Initiates Phase 1/2 Study of APL-9 in Patients with Severe COVID-19
May 28, 2020
WALTHAM, Mass., May 28, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company pioneering targeted C3 therapies, today announced that the company has initiated a Phase 1/2 clinical study of APL-9, an investigational C3 inhibitor designed for acute interventions, in COVID-19 patients with respiratory failure including acute respiratory distress syndrome (ARDS). ARDS is a life-threatening form of lung failure that involves overactivation of complement, a part of the body’s immune system.1
In COVID-19, complement activation has also been found to be strongly associated with organ failure and thrombotic events, which involve the abnormal formation of blood clots and are an emerging cause of mortality in COVID-19 patients.2,3,4 Apellis initiated the Phase 1/2 study following preliminary results from its ongoing, non-interventional study in patients with ARDS secondary to COVID-19, which showed a substantial increase in the activation of the complement cascade.
The randomized, multi-center, controlled Phase 1/2 study is expected to enroll 66 hospitalized patients with COVID-19 and respiratory failure who require oxygen supplementation or mechanical ventilation. The primary objective of the study is to assess the safety of APL-9 as an add-on to the current standard of care. Secondary objectives include evaluating length of stay in the hospital, overall survival, time on oxygen therapy or mechanical ventilation, and markers of complement activation, organ failure, and coagulation (blood clotting). Apellis initiated the study upon clearance by the U.S. Food and Drug Administration, which expedited the review of the investigational new drug application for APL-9 in COVID-19.
“ARDS and harmful blood clots are associated with mortality in COVID-19, and we believe that dysregulation of complement plays a key role in both of these conditions,” said Lukas Scheibler, Ph.D., Chief Innovation Officer at Apellis. “We look forward to evaluating the potential of APL-9 to improve clinical outcomes in COVID-19 patients by controlling complement centrally, at the level of C3.”
For more information about the Phase 1/2 study APL9-COV-201, visit www.clinicaltrials.gov (NCT04402060).
APL-9 is an investigational drug designed to control the complement cascade centrally at C3 and may have the potential to treat a range of diseases caused by excessive or uncontrolled activation of complement. APL-9 is a second-generation C3 inhibitor that leverages the same mechanism of action as Apellis’ lead compound, pegcetacoplan (APL-2), but has a lower molecular weight and shorter half-life. APL-9 is designed to be intravenously administered for acute use.
Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. By pioneering targeted C3 therapies, we aim to develop best-in-class and first-in-class therapies for a broad range of debilitating diseases that are driven by uncontrolled or excessive activation of the complement cascade, including those within hematology, ophthalmology, and nephrology. For more information, please visit http://apellis.com.
Apellis Forward-Looking Statement
Statements in this press release about future expectations, plans and prospects, such as the statements regarding the timing of enrollment of clinical trials, 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. These statements include, but are not limited to, statements relating to the implications of preliminary clinical data. 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 company’s clinical trials will be fully enrolled and completed when anticipated; uncertainties regarding the impact of the COVID-19 pandemic on the company’s business and operations, including the timing and conduct of its ongoing clinical trials; whether preliminary or interim results from a clinical trial will be predictive of the final results of the trial or of other trials; whether results obtained in preclinical studies and clinical trials will be indicative of results that will be generated in future clinical trials; whether the results of the company’s clinical trials will warrant regulatory submissions and whether APL-9 will receive approval from the FDA or equivalent foreign regulatory agencies for respiratory failure secondary to COVID-19 or any other indication when expected or at all; whether, if Apellis’ products receive approval, they will be successfully distributed and marketed; and other factors discussed in the “Risk Factors” section of Apellis’ Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on April 29, 2020 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.
1. Robbins RA, Russ WD, Rasmussen JK, Clayton MM. Activation of the complement system in the adult respiratory distress syndrome. Am Rev Respir Dis. 1987;135(3):651-658. doi:10.1164/arrd.19184.108.40.2061.
2. Magro C, Mulvey JJ, Berlin D, et al. Complement associated microvascular injury and thrombosis in the pathogenesis of severe COVID-19 infection: A report of five cases. Transl Res. 2020. doi:10.1016/j.trsl.2020.04.007.
3. Cugno M, Meroni PL, Gualtierotti R, et al. Complement Activation in Patients with Covid-19: A Novel Therapeutic Target. J Allergy Clin Immunol. 2020. doi:10.1016/j.jaci.2020.05.006.
4. Giannis D, Ziogas IA, Gianni P. Coagulation disorders in coronavirus infected patients: COVID-19, SARS-CoV-1, MERS-CoV and lessons from the past. J Clin Virol. 2020;127:104362. doi:10.1016/j.jcv.2020.104362.