Capstone Therapeutics Corp. (the “Company” or “we”), is traded on the OTCQB under the ticker symbol “CAPS”. We are a biotechnology company committed to developing a pipeline of novel peptides and other molecules aimed at helping patients with underserved medical conditions.
Our primary asset is ownership in a joint venture development company, called LipimetiX Development, Inc. (“LipimetiX” or “JV”) based in Natick, Massachusetts. In 2012, Capstone invested $6 million in the joint venture. Our partners in the venture include three PhD drug developers, The University of Alabama at Birmingham Research Foundation (“UABRF”) and scientists at The University of Alabama at Birmingham (“UAB”). Both Capstone (with two full time employees) and the joint venture (managed under consulting contract by Benu Biopharma) operate virtual business models to maintain capital efficiency for our shareholders.
Through funding provided by Capstone, LipimetiX developed its first candidate peptide molecule, AEM-28, through pre-clinical and human clinical Phase 1a, 1b and 2a studies. 51 patients were enrolled in double-blinded, placebo-controlled studies at a clinical site in Perth, Australia. AEM-28 was delivered in single ascending dose and multiple ascending dose formats to test safety, tolerability and efficacy. The Medical Safety Committee, reviewing all safety-related aspects of the clinical trial, observed a generally acceptable safety profile. Of the first-in-man study, the primary endpoint was safety; yet, efficacy measurements analyzing pharmacodynamics yielded statistical significance in the pooled data set favoring AEM-28 vs. placebo in multiple lipid biomarker endpoints, especially VLDL cholesterol and triglycerides. Please see www.lipimetix.com
Concurrent with the clinical development activities of AEM-28, the JV has performed pre-clinical studies that have identified an analog of AEM-28, referred to as AEM-28-14, and a new formulation, that has the potential of increased efficacy, higher human dose toleration and an extended composition of matter patent life (application filed with the U.S. Patent and Trademark Office in 2015). The JV’s current intent is to prioritize the development of AEM-28-14.
Based upon AEM-28’s clinically proven ability to rapidly reduce triglycerides and other lipoproteins, we are targeting various atherosclerosis, heart and peripheral artery clinical indications.
Chimeric Apolipoprotein E Mimetic Peptides. Apolipoprotein E (Apo E) is in a class of protein that occurs throughout the body. Apo E is essential for the normal metabolism of cholesterol and triglycerides. After a meal, the postprandial (or post-meal) lipid load is packaged in lipoproteins and secreted into the blood stream. Apo E targets cholesterol and triglyceride rich lipoproteins to specific receptors in the liver, decreasing the levels in the blood. Elevated plasma cholesterol and triglycerides are independent risk factors for atherosclerosis, the buildup of cholesterol rich lesions and plaques in the arteries. Atherosclerosis is the major cause of cardiovascular disease, peripheral artery disease and cerebral artery disease, and can cause heart attack, loss of limbs and stroke. Defective lipid metabolism also plays an important role in the development of adult onset diabetes mellitus (Type 2 diabetes), and diabetics are particularly vulnerable to atherosclerosis, heart and peripheral artery diseases.
The UAB scientists patented the first chimeric Apo E mimetic peptide in 1999, reducing the 299 amino acid native Apo E into a smaller 28 amino acid, dual domain peptide that can be delivered therapeutically. One domain inserts into a lipoprotein surface and the second domain binds to the Apo E receptors in the liver. In 2010, our JV’s founding scientist, Dr. Dennis Goldberg, obtained worldwide right to patents for Apo E mimetic peptides from UABRF. The JV has an Exclusive License Agreement with UABRF for AEM-28 and its analogs.
Concurrent with the development activities with AEM-28, the JV has performed limited pre-clinical studies that have identified an analog of AEM-28, referred to as AEM-28-14 and a formulation, that has the potential of higher efficacy and higher dose toleration (in pre-clinical models). A fresh composition of matter patent application was filed in July 2015 with the U.S. Patent and Trademark Office for AEM-28-14 and a broad domain of analogs.
Previously, we were focused on the development and commercialization of two product platforms: AZX100 and Chrysalin (TP508). Since March 2012, we no longer have any interest in or rights to Chrysalin. In 2014, we terminated the License Agreement for AZX100 intellectual property and returned all interest in and rights to the AZX100 intellectual property to the Licensor (AzTE).
The JV and Company intend to explore fundraising, partnering or licensing to obtain additional funding to continue development activities of new leading candidate AEM-28-14. The JV and the Company do not have sufficient funding at this time to continue additional material development activities of AEM-28 and its analogs or other operations. The JV may conduct future clinical trials in Australia, the USA, and other regulatory jurisdictions if regulatory approvals, additional funding, and other conditions permit.
The Company intends, funding permitting, to continue limiting its internal operations to a virtual operating model while monitoring and participating in the management of LipimetiX’s development activities.