Now that Apellis Pharmaceuticals Inc.’s Empaveli pegcetacoplan has won FDA approval as the first targeted C3 therapy for treating paroxysmal nocturnal hemoglobinuria (PNH), the cost of treating the rare blood disorder is hefty.
Citing a lack of efficacy from adding its complement inhibitor Ultomiris (ravulizumab) to best supportive care for patients hospitalized with severe COVID-19, Alexion Pharmaceuticals Inc. paused further enrollment in a global phase III study of the drug. The move, recommended by the trial's independent data monitoring committee, suggested Ultomiris may soon join the list of other therapeutics once tested against COVID-19 but now no longer.
While everyone will be glad to see 2020 in their rearview mirror, the biopharmaceutical sector can count its blessings, as it emerges from a period where it has surprisingly received record public and private financings and enjoyed strong investor support. This is in sharp contrast to most of the global economy, which has been decimated by the ongoing pandemic.
With a $39 billion offer in hand, Alexion Pharmaceuticals Inc. is the target of what could be the third largest takeover to date of a pure play biotech company. At a 45% premium, the proposed transaction, which is expected to close in the third quarter of 2021, is largely applauded by analysts who point to three years of languishing stock prices despite the promise of the Boston-based company’s C5 complement franchise and a sturdy pipeline.
The share prices of blue-chip biopharmaceutical companies continued on their lackluster trajectory, which started in June and collectively lost ground last month, with the BioWorld Biopharmaceutical index slipping about 1.3% as investors took a break in order to digest the deluge of COVID-19 vaccine data released during the period and the impact on the bottom lines that the pandemic has exerted among those companies that have reported their second-quarter results.
Alexion Pharmaceuticals Inc., of Boston, will diversify its portfolio beyond its top-selling Soliris (eculizumab) and C5 inhibitor Ultomiris (ravulizumab) by acquiring Portola Pharmaceuticals Inc. for $1.4 billion. Alexion’s CEO, Ludwig Hantson, told investors May 5 that the acquisition immediately diversifies the company’s commercial-stage portfolio by adding Portola’s Andexxa (coagulation factor Xa [recombinant], inactivated-zhzo) to Alexion’s existing palette of hematology and neurology therapies. Alexion’s CEO, Ludwig Hantson, told investors May 5 that the acquisition immediately diversifies the company’s commercial-stage portfolio by adding Portola’s Andexxa (coagulation factor Xa [recombinant], inactivated-zhzo) to Alexion’s existing palette of hematology and neurology therapies. Andexxa is the only FDA-approved factor Xa inhibitor reversal agent in severe and uncontrolled bleeding.
Apellis Pharmaceuticals Inc.’s CEO, Cedric Francois, said his firm’s phase III study called Pegasus testing pegcetacoplan, or APL-2, in paroxysmal nocturnal hemoglobinuria (PNH) “finally established that there is an important unmet medical need in this disease.”
A medicine Stealth Biotherapeutics Inc. has been developing for the potential treatment of primary mitochondrial myopathy (PMM) missed the co-primary endpoints of a key phase III trial, sending company shares (NASDAQ:MITO) down 65.9% to close at $4.75 on Friday.
With Alexion Pharmaceuticals Inc.'s acquisition of Achillion Pharmaceuticals Inc. for about $930 million up front, Alexion expands and diversifies its pipeline into familiar territory – treating complement-mediated diseases. Blue Bell, Pa.-based Achillion is developing oral, small-molecule factor D inhibitors for treating complement alternative pathway-mediated rare diseases, including paroxysmal nocturnal hemoglobinuria and C3 glomerulopathy.
Alexion Pharmaceuticals Inc. and Stealth Biotherapeutics Corp. reached a deal for an option to co-develop and commercialize elamipretide for mitochondrial diseases.
