Achillion Pharmaceuticals (NASDAQ: ACHN) this morning said it has initiated patient dosing in a phase 2 open-label study of ACH-4471 for patients with paroxysmal nocturnal hemoglobinuria (PNH). ACH-4471 is the company’s first orally-administered, small molecule factor D inhibitor. The study will assess the efficacy, safety, and pharmacokinetics of ACH-4471 in untreated patients with PNH. “We are pleased to have begun this trial in PNH patients to evaluate ACH-4471, the first orally-administered factor D inhibitor to have demonstrated complement alternative pathway (AP) inhibition in humans, which represents a potentially novel and unique mechanistic approach to treating this life-threatening disease,” president and CEO Milind Deshpande, Ph.D., stated in the news release.
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About Achillion Pharmaceuticals
Achillion Pharmaceuticals, Inc. (NASDAQ: ACHN) is a science-driven, patient-focused company seeking to leverage its strengths across the continuum from discovery to commercialization in its goal of providing better treatments for people with serious diseases. The company employs a highly-disciplined discovery and development approach that has allowed it to pursue best-in-class oral antiviral therapy for chronic hepatitis C (HCV) and build a platform of potent and specific complement factor D inhibitors for AP-mediated diseases. Achillion is rapidly advancing its efforts to become a fully-integrated pharmaceutical company with a goal of bringing life-saving medicines to patients with rare diseases. More information is available at www.achillion.com.
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