
Drug Developer

Developer of New Drugs for Rare Disease Treatment
On October 18, 2019, VCBeat (WeChat ID: vcbeat) learned from foreign media reports that Alexion Pharmaceuticals (Alexion) announced the acquisition of clinical-stage biopharmaceutical company Achillion Pharmaceuticals (Achillion) for $930 million. The acquisition is expected to be completed in the first half of 2020.
Upon completion of this acquisition, Alexion will expand its product portfolio and advance the clinical trials of two rare disease candidate therapies from Achillion, providing new treatment options for patients with complement-mediated immune diseases.
Achillion, founded in 1998 and headquartered in Pennsylvania, USA, is a clinical-stage biotechnology and pharmaceutical company. The company is dedicated to developing innovative drugs to improve clinical outcomes and quality of life for patients with complement system disorders. Achillion was listed on the NASDAQ Stock Exchange in the United States in November 2006, under the stock ticker symbol ACHN.
The complement system, also known as the complement activation pathways, includes the classical pathway, the alternative pathway, the lectin pathway, and the proteolytic pathway. It refers to the sequential assembly and activation of complement components, such as antigen-antibody complexes and ions, which triggers immunobiological phenomena including immune hemolysis and immune cytolysis. Scientific and clinical trials have demonstrated that the complement system is implicated in many difficult-to-treat rare diseases.
Currently, Achillion’s research focus is on the late-stage clinical trials and commercialization of danicopan, an oral complement factor D inhibitor. This small-molecule inhibitor can be used to treat rare diseases mediated by the complement activation pathway, such as paroxysmal nocturnal hemoglobinuria (PNH) and C3 glomerulopathy (C3G). Danicopan has potential therapeutic effects for various complement system disorders.
Furthermore, Achillion has two drug development programs in clinical stages, with danicopan currently undergoing Phase II clinical studies and ACH-5228 in Phase I clinical trials. Achillion will collaborate with key stakeholders, including healthcare professionals, researchers, regulatory agencies, and investors, to advance the late-stage clinical development and commercialization of its pipeline products.
Alexion, founded in 1992 and headquartered in Connecticut, USA, is a biotechnology and pharmaceutical company. The company is primarily engaged in the development and commercialization of drugs to treat severe conditions affecting the hematologic, renal, and nervous systems, helping patients with cancer, autoimmune diseases, and transplant rejection recover their health. Alexion was listed on the NASDAQ Stock Exchange in March 1996 under the ticker symbol ALXN.
Alexion focuses on treating rare diseases, and its monoclonal antibody immunotherapy eculizumab (Soliris) has gained significant market popularity, demonstrating favorable efficacy in patients with paroxysmal nocturnal hemoglobinuria (PNH). In April 2009, the U.S. Food and Drug Administration (FDA) granted orphan drug designation to eculizumab for the treatment of patients with atypical hemolytic uremic syndrome (aHUS).
Alexion CEO Dr. Ludwig Hantson stated, “Our R&D team has demonstrated that inhibiting complement factor C5 can improve therapeutic outcomes in multiple rare diseases, but this is merely a starting point for the development of complement inhibitors. Alexion will develop additional complement inhibitors to treat complement system-related disorders and help the body maintain normal immune function.”
Joe Truitt, President and Chief Executive Officer of Achillion, stated, “Achillion holds a significant advantage in the field of complement inhibitor development. We are currently conducting clinical trials for several small-molecule drugs to evaluate their therapeutic efficacy in immune diseases associated with the complement system.”
(Compiled by Xu Xiaoxue)