Item 7.01. Regulation FD Disclosure.
On
The information contained in this Item 7.01, including in Exhibit 99.1 hereto, is being "furnished" and shall not be deemed "filed" for the purposes of Section 18 of the Securities Exchange Act of 1934, as amended, is not subject to the liabilities of that section and is not deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Securities Exchange Act of 1934, as amended, except as shall be expressly set forth by specific reference in such a filing.
Item 8.01 Other Events.
On
The MARINA trial is a randomized, double-blind, placebo-controlled, Phase 1/2 clinical trial expected to enroll approximately 44 adults with DM1. The primary objective of the trial is to evaluate the safety and tolerability of single and multiple ascending doses of AOC 1001 administered intravenously. The MARINA trial will assess the activity of AOC 1001 across key biomarkers, including spliceopathy, a key biomarker for DM1, and knockdown of DMPK mRNA, the disease-related mRNA responsible for DM1. Though the Phase 1/2 trial is not powered to assess functional benefit, it will explore the clinical activity of AOC 1001, including measures of mobility and muscle strength, as well as patient reported outcomes and quality of life measures. Patients will have the option to enroll in an open label extension study at the end of the post-treatment period. In the second half of 2022, the Company plans to conduct a preliminary assessment of safety, tolerability and key biomarkers in approximately half of the study participants.
Recently, the FDA also granted Orphan Drug Designation to AOC 1001 for the treatment of DM1. The FDA grants Orphan Drug Designation to novel drugs that seek to treat a rare disease or condition and, if the drug is approved for the designated orphan indication, provides seven years of market exclusivity, along with certain financial incentives, including tax credits, opportunities for grant funding towards clinical trial costs and FDA user-fee waivers.
Forward-Looking Statements
The Company cautions readers that statements contained in this report regarding
matters that are not historical facts are forward-looking statements. These
statements are based on the Company's current beliefs and expectations. Such
forward-looking statements include, but are not limited to, statements
regarding: the initiation of a Phase 1/2 clinical trial of AOC 1001 in patients
with DM1; and the expected benefits associated with Orphan Drug Designation. The
inclusion of forward-looking statements should not be regarded as a
representation by the Company that any of its plans will be achieved. Actual
results may differ from those set forth in this report due to the risks and
uncertainties inherent in the Company's business, including, without limitation:
the Company is early in its development efforts and all of its development
programs are in the preclinical or discovery stage; the Company's approach to
the discovery and development of product candidates based on its AOC platform is
unproven, and the Company does not know whether it will be able to develop any
products of commercial value; potential delays in the commencement, enrollment
and completion of clinical trials; disruption to the Company's operations from
the COVID-19 pandemic; risks that the benefits associated with Orphan Drug
Designation may not be realized, including that Orphan Drug exclusivity may not
effectively protect a product from competition and that such exclusivity may not
be maintained; the success of the Company's preclinical studies and clinical
trials for its product candidates; the results of preclinical studies and early
clinical trials are not necessarily predictive of future results; the Company's
dependence on third parties in connection with preclinical testing and product
manufacturing; unexpected adverse side effects or inadequate efficacy of the
Company's product candidates that may limit their development, regulatory
approval and/or commercialization, or may result in recalls or product liability
claims; regulatory developments in
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Item 9.01. Financial Statements and Exhibits.
(d) Exhibits Exhibit Number Description 99.1 Slide Presentation 104 Cover Page Interactive Data File (embedded within the Inline XBRL document)
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