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Apellis Pharma Shares Rise 20% on Positive Top-line Data in Phase 3 PNH Trial

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Shares of Apellis Pharmaceuticals opened 35% higher and established a new 52-week intraday high price today after the company announced positive top-line results from its Phase 3 head-to-head study of Pegcetacoplan (APL-2) versus Eculizumab in patients with Paroxysmal Nocturnal Hemoglobinuria.

Apellis Pharmaceuticals Inc. (APLS:NASDAQ) today announced that it had achieved positive results in the company's Phase 3 PEGASUS study, which is evaluating pegcetacoplan (APL-2) in adults with paroxysmal nocturnal hemoglobinuria (PNH).

The firm advised in the report that the top-line data show that pegcetacoplan met the study's primary efficacy endpoint and demonstrated superiority compared to patients who were treated with eculizumab. The company noted that pegcetacoplan also showed promising results in key secondary endpoints specifically meeting non-inferiority on transfusion avoidance and absolute reticulocyte count.

The company's Chief Medical Officer Federico Grossi, M.D., Ph.D., commented, "Pegcetacoplan is the first and only investigational therapy to demonstrate superiority compared to eculizumab on hemoglobin levels. We are also excited to see that 85% of patients treated with pegcetacoplan were transfusion free...At Apellis, we are focused on developing groundbreaking therapies, and these results show that pegcetacoplan has the potential to transform the lives of people with PNH. We look forward to meeting with regulators in the first half of the year to discuss next steps."

Peter Hillmen, M.B., Ch.B., Ph.D., professor of experimental hematology at the University of Leeds and an investigator in the PEGASUS study, added, "The majority of patients with PNH currently receiving treatment with eculizumab have continuing anemia...The PEGASUS results show that pegcetacoplanhas the potential to become a new standard of care for patients with PNH."

In regards to the PEGASUS study, Apellis Pharma's Co-Founder and CEO Cedric Francois, M.D., Ph.D., remarked, "Going into the study, our most optimistic expectation was to see a 2 g/dL or more change in hemoglobin and a trend on the key secondary endpoints. Needless to say, we are thrilled with these results...These data give us strong confidence in the further development of pegcetacoplan as a targeted C3 inhibitor in geographic atrophy and other serious complement-driven diseases."

The firm explained in the report that "PNH is a rare, chronic, life-threatening blood disorder associated with abnormally low hemoglobin levels due to the destruction of oxygen-carrying red blood cells (hemolysis) and that persistently low hemoglobin can result in frequent transfusions and debilitating symptoms such as severe fatigue and difficulty breathing (dyspnea)."

The company identifies pegcetacoplan (APL-2) as "an investigational, targeted C3 inhibitor designed to regulate excessive complement activation, which can lead to the onset and progression of many serious diseases." In addition to the study for use in PNH, the firm stated that it is also studying pegcetacoplan in several other clinical studies in geographic atrophy (GA), and C3 glomerulopathy and indicated that the FDA has granted Fast Track designation for pegcetacoplan in the treatment of both PNH and GA.

Apellis Pharmaceuticals is headquartered 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, ophthalmology and nephrology.

Apellis Pharmaceuticals has a market capitalization of around $1.9 billion with approximately 63.89 million shares outstanding and a short interest of about 8.3%. APLS shares opened 35% higher today at $40.89 (+$10.70, +35.49%) over yesterday's $30.19 closing price and reached a new 52-week high price this morning of $42.48. The stock has traded today between $34.61 and $42.48/share and is currently trading at $37.50 (+$7.31, +24.21%).


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