Achillion Announces Completion of Enrollment in Phase 2 PNH Combination Trial
- Completed enrollment in ACH-4471 Phase 2 PNH trial in combination with eculizumab -
- Interim data to be released in
- Targeting FDA end-of-Phase 2 meeting in second half of 2019 -
In the Phase 2 trial, patients with an inadequate response to eculizumab as monotherapy are being evaluated for increases in hemoglobin and reduced transfusions over 24-weeks. Early data released in
The company expects to present interim data for the first 10 patients on
“We are excited to present interim data on the first 10 patients enrolled and to discuss these results with the FDA,” said
The data from the ACH-4471 Phase 2 combination study along with the prior completed 10 patient Phase 2 monotherapy study will be submitted for review by regulatory authorities in an end of Phase 2 meeting in the second half of 2019.
About the ACH-4471 Phase 2 Combination Study
A Phase 2 open-label study of ACH-4471 in up to 12 patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who have an inadequate response to eculizumab as monotherapy. The purpose of this dose-ranging study is to determine the effectiveness of ACH-4471 in improving anemia, as measured by increased blood hemoglobin, when given with eculizumab (a C5 inhibitor commonly used for treatment of PNH) for 24 weeks. In
Cautionary Note Regarding Forward-Looking Statements
This press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that are subject to risks, uncertainties and other important factors that could cause actual results to differ materially from those indicated by such forward-looking statements. Achillion may use words such as “expect,” “anticipate,” “project,” “target,” “intend,” “plan,” “aim,” “believe,” “seek,” “estimate,” “can,” “could” “focus,” “will,” “look forward,” “continue,” “goal,” “strategy,” “objective,” “may,” “potential,” and similar expressions to identify such forward-looking statements. These forward-looking statements also include statements about: the potential benefits of factor D inhibition as a treatment for complement-mediated diseases; the potential benefits of, and indications for, Achillion’s compounds that inhibit factor D, including ACH-4471, ACH-5228 and ACH-5548; Achillion’s belief that its portfolio of compounds could expand factor D portfolio opportunities, provide strategic optionality or create significant value; Achillion’s expectations regarding the advancement of, and timeline for reporting results from, clinical trials of its product candidates as well as its ability to advance additional compounds; and other statements concerning Achillion’s strategic goals, efforts, plans, and prospects. Among the important factors that could cause actual results to differ materially from those indicated by such forward-looking statements are risks relating to, among other things, Achillion’s ability to: demonstrate in any current and future clinical trials the requisite safety, efficacy and combinability of its product candidates; advance the preclinical and clinical development of its complement factor D inhibitors under the timelines it projects in current and future preclinical studies and clinical trials; enroll patients in its clinical trials on its projected timelines; replicate in later stage clinical trials favorable data demonstrated in preclinical and early-stage clinical trials; obtain and maintain patent protection for its product candidates and the freedom to operate under third party intellectual property; obtain and maintain necessary regulatory approvals, and the granting of orphan designation does not alter the standard regulatory requirements and process for obtaining such approval; establish commercial manufacturing arrangements; identify, enter into and maintain collaboration and other commercial agreements with third-parties; compete successfully in the markets in which it seeks to develop and commercialize its product candidates and future products; manage expenses; manage litigation; raise the substantial additional capital needed to achieve its business objectives; and successfully execute on its business strategies. These and other risks are described in the reports filed by Achillion with the
In addition, any forward-looking statement in this press release represents Achillion's views only as of the date of this press release and should not be relied upon as representing its views as of any subsequent date. Achillion disclaims any duty to update any forward-looking statement, except as required by applicable law.
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Source: Achillion Pharmaceuticals, Inc.