Aevi Genomic Medicine, Inc. Announces Novel Co-crystal Version of AEVI-001
Aevi Genomic Medicine, Inc. (NASDAQ: GNMX) (the Company) today announced that it received positive feedback from the United States Food and Drug Administration (FDA) on an improved version of the Company's lead development molecule, AEVI-001, identified as AEVI-004.
Following 2016 U.S. Food and Drug Administration (the FDA) regulatory guidance on co-crystalization of active drugs, the Company created a co-crystal of fasoracetam (AEVI-001) with enhanced physical and chemical properties. The new molecule, AEVI-004, has comparatively greater stability and a higher melting point than AEVI-001. The molecule was engineered to maintain solubility, dissolution and pharmacokinetics substantially similar to AEVI-001.
The Company has received feedback from the FDA provisionally indicating that AEVI-004 is a co-crystal of AEVI-001 and a novel drug substance. The FDA also provisionally indicated that existing toxicology and pathology studies can support clinical development with AEVI-004.
Assuming positive results from the ongoing Phase 2 ASCEND clinical trial, and following minimal bridging preclinical and clinical pharmacological studies requested by FDA, the Company anticipates progressing the molecule directly into Phase 3 studies with minimal impact on the program timeline.
AEVI-004 is expected to have composition of matter patents extending to 2039 and should be listed as a novel drug substance in the FDA Orange Book.
"The successful engineering of the co-crystal molecule AEVI-004 provides great flexibility in the further development of fasoracetam for ADHD and other glutamate receptor-linked neuropsychiatric diseases," said Garry A. Neil, M.D., Chief Scientific Officer of the Company. "We are proud of the work done by our R&D team to develop AEVI-004, and we will continue to work closely with the FDA as we look to progress this molecule into Phase 3 studies. We look forward to presenting topline data from the ongoing Phase 2 ASCEND trial in the fourth quarter of this year."
AEVI-001 is an oral non-stimulant pan selective activator/modulator of mGluRs. The molecule has excellent pharmacokinetic and metabolic profiles and crosses the blood brain barrier.
AEVI-001 is being developed as a potential treatment for a sub-population of Attention Deficit Hyperactivity Disorder (ADHD) patients with genetic mutations that disrupt the mGluR network. In the United States, the Center for Disease Control estimates that 6.4 million children 4-17 years of age (11%) have ever been diagnosed with ADHD. Many ADHD patients remain unsatisfied with existing therapies, particularly with respect to safety, tolerability and treatment of comorbidities.
AEVI-001 is an investigational agent that has not been approved by the US FDA or any other regulatory agencies.
AEVI-004 is an oral non-stimulant pan selective activator/modulator of mGluRs. The molecule has excellent pharmacokinetic and metabolic profiles and crosses the blood brain barrier.
The molecule is a co-crystal of AEVI-001, crystallized with a pharmacologically inert conformer with a favorable toxicological profile. The molecule's pharmacological properties have been designed, and are expected, to be very similar to those of AEVI-001. As such, the Company believes that the molecule may progress directly to phase 3 studies with only minimal bridging preclinical and clinical pharmacological studies.
AEVI-004 has several distinct advantages over AEVI-001, including better stability and better manufacturability owing to a significantly higher melting point.
About Aevi Genomic Medicine, Inc.
Aevi Genomic Medicine, Inc. is dedicated to unlocking the potential of genomic medicine to translate genetic discoveries into novel therapies. Driven by a commitment to patients with pediatric onset life-altering diseases, the Company's research and development efforts leverage an internal genomics platform and an ongoing collaboration with the Center for Applied Genomics at The Children's Hospital of Philadelphia. Based on discoveries from the genomics collaboration, the Company currently has two programs in clinical development, AEVI-001 for mGLuR+ ADHD and AEVI-002 for severe pediatric onset Crohn's disease.
More information on the Company and pipeline is located on its website www.aevigenomics.com
This release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, Section 21E of the Securities Exchange Act of 1934 and as that term is defined in the Private Securities Litigation Reform Act of 1995, which include all statements other than statements of historical fact, including (without limitation) those regarding the expected timing of the closing of the private placement, the Company's development and business strategy, its product candidates and the plans and objectives of management for future operations. The Company intends that such forward-looking statements be subject to the safe harbors created by such laws. Forward-looking statements are sometimes identified by their use of the terms and phrases such as "estimate," "project," "intend," "forecast," "anticipate," "plan," "planning, "expect," "believe," "will," "will likely," "should," "could," "would," "may" or the negative of such terms and other comparable terminology. All such forward-looking statements are based on current expectations and are subject to risks and uncertainties. Should any of these risks or uncertainties materialize, or should any of the Company's assumptions prove incorrect, actual results may differ materially from those included within these forward-looking statements. Accordingly, no undue reliance should be placed on these forward-looking statements, which speak only as of the date made. The Company expressly disclaims any obligation or undertaking to disseminate any updates or revisions to any forward-looking statements contained herein to reflect any change in the Company's expectations with regard thereto or any change in events, conditions or circumstances on which any such statements are based. As a result of these factors, the events described in the forward-looking statements contained in this release may not occur.
Aevi Genomic Medicine, Inc.
SOURCE Aevi Genomic Medicine, Inc.
Company Codes: NASDAQ-NMS:GNMX