Achillion Announces Enrollment Milestone in Phase 2 Trials for C3 Glomerulopathy
- Enrolled 23 patients in ACH-4471 Phase 2 C3G six and 12-month clinical trials -
- Targeting end-of-Phase 2 meeting with
“Enrolling our ACH-4471 Phase 2 trials is an important milestone for Achillion, and we would like to thank all of the patients, families, investigators and participating clinical trial sites for their outstanding contributions,” said
About the ACH-4471 Phase 2 C3G Trials
The objective of the six and 12-month proof-of-concept trials is to evaluate the safety and efficacy of the oral, small molecule factor D inhibitor, ACH-4471, in patients diagnosed with C3G. In the six-month trial, patients are blinded and randomized 1:1 to receive either ACH-4471 or placebo. The 12-month trial is open label. The measures being evaluated include changes in clinical manifestations of C3G including proteinuria and estimated glomerular filtration rate (eGFR) at the end of the treatment period and changes in kidney biopsy from baseline.
ACH-4471, Complement Factor D Inhibitor
The Company’s first-generation oral complement factor D inhibitor, ACH-4471, is currently being evaluated for efficacy with Phase 2 clinical programs in both paroxysmal nocturnal hemoglobinuria (PNH) and C3G and has demonstrated preliminary proof-of-concept in both indications. The PNH program consists of a Phase 2 clinical trial evaluating ACH-4471 in patients who are inadequately controlled or sub-optimally responding to eculizumab. Additionally, patients continue to dose in the PNH monotherapy extension trial. The C3G program consists of two Phase 2 clinical trials – a six-month blinded, placebo-controlled trial and a 12-month open-label trial, described above.
More information is available at http://www.achillion.com/patients-and-clinicians/
About C3 Glomerulopathy (C3G)
C3G is a devastating disease affecting the kidney for which there is no approved therapy. C3G affects persons of all ages with men and women equally affected. There are estimated to be approximately 4,000 C3G patients in
More information is available at http://www.achillion.com.
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; Achillion’s expectations regarding the timing of regulatory interactions and submissions; 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.
Senior VP, Chief Financial Officer
Tel. (215) 709-3032
Senior VP, Corporate Communications
Tel. (215) 709-3055
Source: Achillion Pharmaceuticals, Inc.