Apellis Pharmaceuticals, Inc.’s (APLS - Free Report) investigational therapy pegcetacoplan met the primary efficacy endpoint in a phase III head-to-head study, comparing it to Alexion’s (ALXN - Free Report) Soliris (eculizumab) in patients with paroxysmal nocturnal hemoglobinuria (PNH).
A rare blood disorder, PNH is associated with abnormally low hemoglobin levels as the disease destroys red blood cells. Top-line data from the PEGASUS study showed that patients treated with pegcetacoplan experienced a bigger increase in hemoglobin levels than those taking Soliris.
Pegcetacoplan demonstrated an improvement in adjusted means of 3.8 g/dL of hemoglobin at week 16, versus a 1.5 g/dL decrease in the Soliris arm, thereby meeting the study’s primary endpoint. The change observed in hemoglobin levels on treatment with pegcetacoplan bettered Apellis’ most optimistic expectation to see 2 g/dL or more change in hemoglobin. Meanwhile, the study also showed promising results in key secondary endpoints. Pegcetacoplan was non-inferior to Soliris on transfusion avoidance and absolute reticulocyte count. The safety profile of pegcetacoplan was comparable to eculizumab in the study. Apellis plans to meet regulators in the first half of the year to discuss the next steps.
Apellis’ stock shot up 28.3% on Tuesday in response to the news. The company’s shares have risen 178% in the past year against 2.4% decrease registered by the industry during this period.
Soliris, which is used to treat PNH as well as two other ultra-rare disorders, made almost $3.0 billion for Alexion in the first nine months of 2020. If approved by the FDA, pegcetacoplan would be Apellis’ first commercial product and should bring in significant revenues for the company. However, Alexion also markets long-acting C5 complement inhibitor, Ultomiris, a follow-up version of Soliris for PNH. The initial uptake of Ultomiris has been strong.
Alexion is also due to buy clinical-stage biopharmaceutical company, Achillion Pharmaceuticals, Inc. (ACHN - Free Report) for $930 million to strengthen its PNH franchise. Achillion’s danicopan is being developed in a phase II study in combination with Soliris, to treat patients with PNH, who are suboptimal responders to only Soliris. A potential approval of danicopan will make Alexion a market leader in the PNH space. Notably Amgen (AMGN - Free Report) is developing a biosimilar of Soliris. All these drugs could pose strong competition to pegcetacoplan on approval.
Apellis currently carries a Zacks Rank #4 (Sell).
You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
The Hottest Tech Mega-Trend of All
Last year, it generated $24 billion in global revenues. By 2020, it's predicted to blast through the roof to $77.6 billion. Famed investor Mark Cuban says it will produce ""the world's first trillionaires,"" but that should still leave plenty of money for regular investors who make the right trades early.
See Zacks' 3 Best Stocks to Play This Trend >>