“We are quite encouraged by the interim data generated with ANX005 in HD. We are particularly excited to see a heightened clinical response in patients with excess baseline complement activity, suggesting that the classical complement pathway plays a key role in the neurodegenerative disease process and that ANX005 has the potential to provide meaningful benefit to HD patients,” remarked Douglas Love, Esq., president and chief executive officer of Annexon. “These early data in HD patients, coupled with prior proof-of-concept data in GBS, provide a growing body of evidence for the potential role of anti-C1q in treating complement mediated neurodegenerative and autoimmune diseases, and we look forward to continuing to assess the full potential of our approach in several ongoing trials in diseases of high unmet need.”
The Phase 2 trial remains ongoing, and Annexon anticipates reporting full data from all patients enrolled, including data from the three-month follow-up period, in the second quarter of 2022. Pending results from the full dataset, Annexon plans to evaluate the opportunity for a Phase 3 trial of ANX005 in HD patients.
Conference Call Information
Annexon management will host a conference call today at 4:30 p.m. ET. To participate in the conference call, please dial (833) 649-1187 (domestic) or (281) 206-0036 (international) and refer to conference ID 6297344. The webcast and accompanying slides can be accessed under the ‘Events & Presentations’ section on the Investors page at www.annexonbio.com. A replay of the webcast will be archived on the Annexon website for 30 days.
About the Phase 2 Trial (ANX005-HD-01)
The Phase 2 multi-center, open-label trial is evaluating ANX005 administered intravenously for a 24-week (six-month) dosing period in patients with, or at risk for, early manifest HD. The study enrolled a total of 28 patients in May of 2021, and 23 patients completed the 24-week treatment period. The interim data reported today include safety data for all 28 patients enrolled, efficacy data as measured by UHDRS in all 23 evaluable patients, pharmacokinetics (PK) and pharmacodynamics (PD) data for the first 13 patients, and NfL for the first 16 patients who completed the 24-week treatment period based on cutoff dates required to obtain data for this interim analysis.
About Annexon
Annexon (Nasdaq: ANNX) is a clinical-stage biopharmaceutical company developing a new class of complement medicines for patients with classical complement-mediated autoimmune, neurodegenerative, and ophthalmic disorders of the body, brain and eye. The company’s pipeline is based on its platform technology addressing a broad spectrum of well-researched classical complement-mediated autoimmune and neurodegenerative diseases triggered by aberrant activation of C1q, the initiating molecule of the classical complement pathway. Annexon is advancing a portfolio of innovative product candidates designed to block the activity of C1q and the entire classical complement pathway: ANX005 (intravenous administration), ANX007 (intravitreal administration), and ANX009 (subcutaneous administration). Annexon is deploying a disciplined, biomarker-driven strategy designed to improve the probability of technical success of its portfolio. For more information, visit www.annexonbio.com.
Forward Looking Statements
This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. In some cases, you can identify forward-looking statements by terminology such as “aim,” “anticipate,” “assume,” “believe,” “contemplate,” “continue,” “could,” “design,” “due,” “estimate,” “expect,” “goal,” “intend,” “may,” “objective,” “plan,” “positioned,” “potential,” “predict,” “seek,” “should,” “suggest,” “target,” “on track,” “will,” “would” and other similar expressions that are predictions of or indicate future events and future trends, or the negative of these terms or other comparable terminology. All statements other than statements of historical facts contained in this press release are forward-looking statements. These forward-looking statements include, but are not limited to, statements about: initial findings and observations related to the interim data from the company’s ongoing, open-label Phase 2 clinical trial of ANX005 in patients with HD; the potential benefits from treatment with anti-C1q therapy; timing of full data from the Phase 2 trial of ANX005 in HD patients; plans to evaluate the opportunity for a Phase 3 trial of ANX005 in HD patients; and continuing advancement of the company’s innovative portfolio. Forward-looking statements are not guarantees of future performance and are subject to risks and uncertainties that could cause actual results and events to differ materially from those anticipated, including, but not limited to, risks and uncertainties related to: the company’s history of net operating losses; the company’s ability to obtain necessary capital to fund its clinical programs; the early stages of clinical development of the company’s product candidates; the effects of COVID-19 or other public health crises on the company’s clinical programs and business operations; the company’s ability to obtain regulatory approval of and successfully commercialize its product candidates; any undesirable side effects or other properties of the company’s product candidates; the company’s reliance on third-party suppliers and manufacturers; the outcomes of any future collaboration agreements; and the company’s ability to adequately maintain intellectual property rights for its product candidates. These and other risks are described in greater detail under the section titled “Risk Factors” contained in the company’s Annual Report on Form 10-K and Quarterly Reports on Form 10-Q and the company’s other filings with the SEC. Any forward-looking statements that the company makes in this press release are made pursuant to the Private Securities Litigation Reform Act of 1995, as amended, and speak only as of the date of this press release. Except as required by law, the company undertakes no obligation to publicly update any forward-looking statements, whether as a result of new information, future events or otherwise.
