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Apellis Gains FDA Approval for Empaveli to Treat Chronic Blood Disorder

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Shares of Apellis Pharmaceuticals Inc. traded 20% higher after the company reported it received U.S. FDA approval for EMPAVELI (pegcetacoplan) for use in treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare, chronic life-threatening blood disorder.

Global biopharmaceutical company Apellis Pharmaceuticals Inc. (APLS:NASDAQ), which is focused upon pioneering targeted C3 therapies, announced that the U.S. Food and Drug Administration (FDA) "has approved EMPAVELI™ (pegcetacoplan), the first and only targeted C3 therapy for treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH)." The firm explained that "EMPAVELI is now approved for use in adults with PNH who are treatment naďve as well as patients switching from the C5 inhibitors Soliris® (eculizumab) and Ultomiris® (ravulizumab)."

Olga Frankfurt, M.D., PEGASUS study investigator and associate professor in the department of medicine, division of hematology and oncology at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University, commented, "EMPAVELI has the potential to improve the lives of patients with PNH by increasing hemoglobin and reducing blood transfusion requirements...Through my work as an investigator on the PEGASUS study, I was excited to see EMPAVELI providing broad control of PNH."

Apellis Pharmaceuticals' Co-Founder and CEO Cedric Francois, M.D., Ph.D., remarked, "As the first, FDA-approved targeted C3 therapy, EMPAVELI has the potential to redefine treatment for adults with PNH, including patients switching from any C5 inhibitor and treatment-naďve patients...This approval represents a major scientific advancement as EMPAVELI ushers in the first new class of complement medicine in almost 15 years. We look forward to exploring the full potential of targeting C3 and continue to advance registrational programs of this therapy across multiple complement-driven diseases with high unmet need."

The firm explained in the report that "PNH is a rare, chronic, life-threatening blood disorder caused by an acquired mutation, which leads to uncontrolled complement activation and the destruction of red blood cells through intravascular and extravascular hemolysis." This persistently low hemoglobin level condition often causes debilitating symptoms such as severe fatigue and difficulty breathing (dyspnea) and often requires patients to undergo frequent blood transfusions.

The company indicated that it has already filed a Marketing Authorization Application for pegcetacoplan for use in treating PNH with the European Medicines Agency (EMA). The firm noted that the EMA is currently reviewing application and that a decision could potentially be reached by the European Commission in H2/21.

The Phase 3 PEGASUS trial was a randomized, head-to-head study in 80 adult PNH patients conducted in collaboration with SFJ Pharmaceuticals. The trial was designed in order to test the efficacy and safety of EMPAVELI (pegcetacoplan) versus Soliris® (eculizumab). Over a 16-week period, participants were given either 1080 mg of EMPAVELI twice weekly or their current dose of Soliris. period.

The company identified EMPAVELI to be "the first and only approved therapy targeting C3, the central protein in the complement cascade that acts proximally in the complement cascade controlling both C3b-mediated extravascular hemolysis and terminal complement-mediated intravascular hemolysis."

Apellis advised that it entered into a co-development arrangement with Sobi in October 2020. Under the terms of the agreement, Apellis holds the exclusive commercial rights for systemic pegcetacoplan in the U.S. along with the global commercial rights for ophthalmological uses including treatment of geographic atrophy. The agreement in turn provides Sobi with exclusive commercial rights for systemic pegcetacoplan in areas outside the U.S.

Apellis Pharmaceuticals is based in Waltham, Mass., and is a global biopharmaceutical company that is engaged in pioneering targeted C3 therapies. The firm focuses in the areas of hematology, nephrology, neurology and ophthalmology.

Apellis Pharmaceuticals began the day with a market capitalization of around $3.6 billion with approximately 80.5 million shares outstanding and a short interest of about 9.6%. APLS shares opened almost 11% higher today at $49.01 (+$4.75, +10.73%) over Friday's $44.26 closing price. The stock has traded today between $48.96 to $54.25 per share and closed for trading at $53.06 (+$8.80, +19.88%).


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