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RespireRx Pharmaceuticals Inc. (OTCQB:RSPI) is developing small molecule medicines for the treatment of respiratory disease with a focus on sleep apneas and respiratory depression and distress.
The Company believes that its translational approach will move forward fundamental scientific knowledge and apply it to human medical needs and incorporate the results into medical practice. RespireRx’s depth of knowledge and its data span from the molecular level, to the cellular level, to the organ level, to the whole non-human animal level, and now ultimately to the human level. Some people refer to this translational approach as taking discoveries from the bench to the bedside.
Sleep apnea is not just snoring, but rather a serious sleep disorder that occurs when a person’s breathing is interrupted repeatedly throughout the night. The disorder is a major cause of motor vehicle and industrial accidents and is co-morbid with cardiovascular disease, type 2 diabetes and other conditions. With a large patient population with few treatment options, sleep apnea represents a market potential of $3 billion to $9 billion per year for RespireRx’s drug candidates. Sleep apnea comes in a variety of forms, most commonly Obstructive Sleep Apnea (OSA), Central Sleep Apnea (CSA) and Mixed Sleep Apnea.
OSA is the most common form of sleep apnea. The American Academy of Sleep Medicine commissioned a study published in August 2016 entitled “Exploring the Economic Benefits of OSA Diagnosis and Treatment.” A few key points from that study are that there are an estimated 29.4 million Americans with OSA (Apnea/Hypopnea Index > 5) of which 23.5 million are undiagnosed and 5.9 million are diagnosed. Total costs of OSA in the United States are estimated at $162 billion. The costs associated with the 5.9 million diagnosed group are estimated to total $12.4 billion which breaks down as follows: (i) $6.2 billion for non-surgical treatments (CPAP device (Continuous Positive Airway Pressure), lifestyle, oral/dental devices, etc.), but no medicines as there are currently no pharmacotherapies for OSA, (ii) $5.4 billion for surgical treatments, and (iii) $800 million for diagnosis, testing and follow-up. The costs estimated for the undiagnosed group are $149.6 billion, of which $26.2 billion is related to motor vehicle accidents, $6.5 billion to workplace accidents, and $86.9 billion to lost productivity.
Part of the problem with OSA is that diagnosis is inconvenient and the treatment options are not particularly patient friendly or effective (CPAP is very effective but very patient unfriendly and compliance is very low). In addition, much of the population does not understand the symptoms. RespireRx believes that if a drug treatment were approved for OSA, not only would a significant number of non-surgically treated patients switch to the drug regimen, but a percentage of the currently undiagnosed population would get tested. In addition, there are companies pursuing less onerous diagnostic methods, including even home based tests that could increase the number of sufferers diagnosed.
The cause of CSA is very different from OSA, even though both result in the cessation of breathing for multiple short periods of time during sleep. Because the cause is different, the drug therapy would need to be different.
RespireRx is developing different drugs for each form of sleep apnea.
RespireRx has two Phase 2 clinical study medicines targeting Obstructive Sleep Apnea and Central Sleep Apnea. Phase 2B clinical data for dronabinol for OSA is expected to be announced before the end of 2016 and a Phase 2A clinical study of CX1739 is planned for CSA in chronic opioid users (see below). In a previous Phase 2A study of the three types of sleep apnea described above, CX1739 showed signals of efficacy in CSA and Mixed, but not OSA.
By the end of 2016, the Company is planning to release data from its Phase 2A clinical study of CX1739, conducted at the Duke University School of Medicine for another indication, opioid induced respiratory depression (RD), discussed in more detail below.
Pending additional funding, RespireRx is also looking to grow its portfolio of medicines to include product candidates for the treatment of respiratory distress in patients suffering from respiratory distress associated with spinal cord injury, Pompe Disease (an orphan disease and a niche market), and other indications.
In September 2016, RespireRx took the first step toward a NASDAQ (or other national exchange) uplisting by completing a reverse stock split.
- Two proprietary small molecule platforms—cannabinoids and ampakines
- Three Phase 2 or Phase 2-ready development programs
- Several pre-clinical programs
- Focus on large markets with unmet clinical needs
- More than 120 patents and patent applications
- Multiple opportunities for strategic collaborations
- Non-dilutive financing from National Heart, Lung and Blood Institute (NHLBI) and National Institute on Drug Abuse (NIDA) – more than $5 million to date
- Experienced and accomplished management team
- Positive Phase 2A efficacy results in RD, OSA and CSA
- Commercial and intellectual property (IP) protection for compounds and uses
“RespireRx is on to something unique. It is the only company focused on physiologically based sleep breathing disorders, and respiratory depression and distress and has a suite of IP-protected advanced clinical-stage medicines that have the potential to address these conditions,” stated Dr. James Manuso, President and CEO of RespireRx.
Amapakines: CX1739 and CX717
Dr. Robert M. Califf, in the February 5, 2016 edition of the FDA Voice, stated “…as all of you know, more Americans now die every year from drug overdoses than they do in motor vehicle crashes. Opioids were involved in 28,648 deaths in 2014, according to the CDC.”
Those deaths occur primarily from respiratory depression caused by opioids. And we are not speaking only about illegal street use, we are talking about people overdosing on prescribed opioids who are taking them legally. Chronic pain patients who are regular opioid users develop tolerance to the drugs for pain and need higher doses to achieve pain control (think Prince), but now run the risk of respiratory depression and the elderly can forget how much of their medications they have taken and the list goes on. Pain management in a post-surgical setting is limited by the dose of opioids that can be given due to the risk of causing respiratory depression. So there are acute, semi-acute and chronic applications for a drug, like CX1739, to either reverse or prevent respiratory depression caused by opioids.
A Phase 2A study of CX1739 was conducted at Duke University School of Medicine and was designed to determine the ability of CX1739 to reduce the respiratory depression caused by remifentanil, a potent opioid, and consisted of two stages. The first stage was a double-blind, placebo controlled, cross-over comparison of 300 mg of CX1739 and placebo, and the second was an open-label, dose ascending analysis of 600 mg and 900 mg of CX1739. Subjects were tested once a week for four weeks. During each of these weekly sessions, subjects were challenged with remifentanil in two ways, as a single, acute, bolus injection (REMI 1) and as an infusion (REMI 2). These procedures were designed, respectively, as models of acute opioid overdose and chronic opioid consumption in which opioid blood levels remain relatively constant. Data analysis is in process and the Company is planning to release data from this Phase 2A clinical study of CX1739 by the end of 2016.
In prior studies with another of RespireRx’s ampakines, CX717, the Company’s experimental medicine prevented respiratory depression caused by alfentanyl, another potent opioid, without offsetting the desirable pain-killing effects of the analgesic drug.
Based upon these results, and subject to available financing, the Company is currently planning to conduct a study of CX1739 in CSA patients who are chronic opioid users. Chronic opioid users’ respiratory depression is CSA measured during sleep. Approximately 70% of chronic opioid users have CSA and it is the primary risk factor for mortality.
One of RespireRx’s compounds, CX717, that is a candidate for further development in spinal cord injury patients suffering from respiratory distress. Of the approximately 276,000 spinal cord injury patients in the United States, approximately half of the injuries occur in the cervical (neck) region, 2/3 of those require respiratory support, usually a ventilator, and 40% require continued ventilator support after discharge. Animal studies support the notion that CX717 can improve respiration in spinal cord injury patients suffering from respiratory distress.
Ampakines have also shown efficacy in animal models of Pompe Disease, an orphan disease where patients may experience respiratory difficulties, and also respiratory distress in premature births.
Finally, RespireRx intends to develop a combination formulation of an ampakine, most likely CX717, with an opioid. The intent would be to create a “safer” opioid. As a person took more and more pills, in an attempt to achieve pain control, they would receive more and more of RespireRx’s ampakine, which in turn would prevent respiratory depression.
Dronabinol: Breakthrough Therapy for Obstructive Sleep Apnea
Dronabinol, RespireRx’s product candidate for the treatment of Obstructive Sleep Apnea, is the first medicine being develop to treat the physiological causes of the disorder. The standard treatment is the Continuous Positive Airway Pressure (CPAP) device, which for reasons made obvious by the photo below, has an extremely high non-compliance rate.
Not surprisingly, 30 percent of diagnosed patients never initiate CPAP treatment when prescribed a machine and over 50 percent of patients stop using CPAP in the first year
Dronabinol (delta 9-tetrahydrocannabinol) is approved by the U.S. Food and Drug Administration (FDA) for the treatment of anorexia associated with AIDS and for chemotherapy induced nausea and vomiting and has a long history of safety. RespireRx expects much greater patient compliance with this form of treatment over the CPAP device.
Phase 2A clinical trials for dronabinol successfully demonstrated a clear signal of activity in OSA patients.
Financed by a non-dilutive $5 million grant from the National Institutes of Health (NIH), a Phase 2B trial for the drug was conducted in 2016 at four major centers by the University of Illinois. The Phase 2B trial was completed in August 2016.
Upcoming Catalyst: Phase 2B data analysis is now underway and results are expected for release by the end of 2016. RespireRx plans to meet with the FDA to determine the path to registration.
RespireRx has obtained the licensed rights (expires 2025) from the University of Illinois to the use patents for the use of dronabinol (actually any cannabinoid, of which dronabinol is one) for the treatment of any sleep related breathing disorder, of which OSA is one. Further patents are pending on modified release formulations for dronabinol. As a Schedule III medicine, use is highly monitored by the U.S. government and is not covered by insurers for use outside of the existing FDA approved label, which discourages off label prescriptions of the generic versions of dronabinol. This provides market exclusivity via monitoring as well as via pricing, as third party reimbursement would only be available for a RespireRx brand of dronabinol, as RespireRx would be the only company with the FDA label for OSA.
James S. Manuso, Ph.D., MBA — CEO, President and Vice-Chairman of the Board of Directors
Dr. James Manuso is a biotechnology/pharmaceutical industry CEO and entrepreneur experienced in the foundation, management, financing, governance and sale of start-up, public, private, domestic and international companies with marketed and R&D-stage products. He has extensive, general management, business, financial, corporate, drug development expertise and well as experience in M&A and investment banking. Dr. Manuso has raised hundreds of millions of dollars for companies. He is also Chairman and CEO of Talfinium Investments, an investment entity and financial consultancy. He served as chairman and CEO of Astex Pharmaceuticals, formerly SuperGen, which was sold under his watch, to Otsuka Pharmaceuticals for $886 million.
Dr. Manuso is the author of over 30 chapters, articles and books on topics including health care cost containment and biotechnology company management. He has taught and lectured at Columbia, New York University, Georgetown, Polytechnic University, and Waseda University (Japan), and he served as a biotechnology consultant to the prime minister of New Zealand. He has delivered invited addresses at meetings of the American Management Association, the American Medical Association, the Securities Industry Association, the Biotechnology Industry Organization, and many other professional associations. He served previously as Vice President and a member of the Board of Trustees of the Greater San Francisco Bay Area Leukemia & Lymphoma Society.
Arnold S. Lippa, Ph.D. — Chief Scientific Officer and Executive Chairman of the Board
Dr. Arnold S. Lippa is a Managing Member and founder of T Morgen Capital LLC, which is an investment and management company specializing in the creation and management of biomedical companies. Since 2005, T Morgen Capital has been a significant equity owner and a managing member of Aurora Capital LLC, a life science focused FINRA member firm, where Dr. Lippa represents T Morgen Capital as a Manager. He is also Chairman of the Board of Xintria Pharmaceutical Corporation, which he co-founded in 2006. Dr. Lippa co-founded and currently is a manager and indirectly a member of Atypical BioCapital Management LLC and Atypical BioVentures Fund LLC, a life science fund management company and fund, respectively. Dr. Lippa also has an indirect ownership interest and managing membership in Aurora Capital LLC (see below).
Dr. Lippa was a founder of DOV Pharmaceuticals Inc. and a co-founder/co-manager of a number of life sciences companies, including Praxis Pharmaceutical, Inc. He has consulted for various pharmaceutical and biotechnology companies and was a graduate faculty professor at the New York University School of Medicine and the City University of New York. Dr. Lippa has held numerous scientific and editorial positions, has testified on the development of anti-AIDS drugs before a Congressional committee, and has served as a consultant and special reviewer for the National Institutes of Health and the National Science Foundation.
Richard Purcell — Senior Vice-President of Research and Development
Richard Purcell leads, with Dr. Lippa, the development of the Company’s portfolio of pipeline products for respiratory indications. He is a biopharmaceutical development specialist with extensive experience in providing consulting services to financial, venture capital, and start-up companies with a concentration on new business strategy and clinical development of novel compounds.
Mr. Purcell is also the President and CEO of intelliSantè, a private software development firm focused on patient-centric health information management systems. Previously, he was president of ClinPro, a clinical research organization where he led this full-service, technology driven CRO specializing in Phase I, II, and III clinical trial management. His work included the design and implementation of a number of early stage clinical development programs.
Prior to joining ClinPro, he worked for SCP Communications, a medical communications company, where he served as Corporate Vice President and General Manager of the Clinical Programs Division. He previously headed the Life Sciences Consulting Group for Kline and Company.
Mr. Purcell started his career as a molecular biologist, where he developed and patented a second generation TPA with increased half-life. He has also conducted primary research and published manuscripts on the topics of AIDS and immunomodulators. He graduated from Princeton University with a degree in Biochemical Sciences, and attended Rutgers Graduate School of Management focusing on marketing and finance.
Jeff Margolis — Vice-President, Treasurer, Secretary and Director
Jeff Margolis is the founder and President of Aurora Capital LLC (1994/1995), an SEC registered FINRA member (and SIPC and MSRB) investment bank and securities brokerage firm. All of Aurora Capital’s investment banking activities are directed at life science companies (biotechnology, biopharmaceutical, medical device, medical instrument, diagnostic and similar companies) and their investors. Mr. Margolis is the President and sole shareholder of Aurora Capital Corp., one of the managing members of Aurora Capital LLC and its predecessor.
Jeff Margolis is also Chief Financial Officer and a member of the Board Directors of Xintria Pharmaceutical Corporation, which he co-founded in 2006.
Mr. Margolis co-founded and currently is a manager and indirectly a member of Atypical BioCapital Management LLC and Atypical BioVentures Fund LLC, a life science fund management company and fund, respectively.
Robert Weingarten — Vice-President, CFO and Director
Mr. Weingarten is an experienced business consultant and advisor. Since 1979 he has provided financial consulting and advisory services to numerous public companies in various stages of development, operation or reorganization. Mr. Weingarten is a Certified Public Accountant (inactive) in the State of California. Mr. Weingarten was the Non-Executive Chairman of New Dawn Mining Corp. (“New Dawn”) from August 31, 2005 through September 30, 2010, and was named the Executive Chairman of New Dawn in October 2010. On July 8, 2010, Mr. Weingarten was appointed to the board of directors of Central African Gold Limited (formerly known as Central African Gold Plc and listed on the Alternative Investment Market of the London Stock Exchange at that time). Central African Gold Limited is an indirect, wholly-owned subsidiary of New Dawn. Both New Dawn and Central African Gold Limited have ceased to be publicly traded reporting companies in their respective jurisdictions. Mr. Weingarten was appointed as a director of Staffing 360 Solutions, Inc. on February 25, 2014 and resigned this position on April 20, 2014. Since June 30, 2015, Mr. Weingarten has served as a director of Guardion Health Sciences, Inc., a company that filed a registration statement on Form S-1 with the SEC on February 11, 2016.
James E. Sapirstein, RPh., M.B.A. – Director
James Sapirstein is considered a start-up and turnaround specialist, with 30 years of pharmaceutical and biotechnology industry experience. He is the CEO and Director of ContraVir Pharmaceuticals, a public company. Prior to joining ContraVir Pharmaceuticals, Mr. Sapirstein served as the CEO and a Director of Alliqua Biomedical. He was a founder, CEO, President and a member of the Board of Directors of Tobira Therapeutics. He has launched several HIV/AIDS agents worldwide. He served as the Head of the International HIV business at Bristol-Myers Squibb, as well as working in its Infectious Disease marketing teams. As Executive Vice President for Serono Laboratories, he led a team of over 100 professionals in the HIV and pediatric growth hormone business. He has held positions at Gilead Sciences, where he was responsible for the product Viread®, as well as Bristol-Myers Squibb, Hoffmann-LaRoche Ltd. and Eli Lilly and Company. He serves as a member of the Board of Directors of Panther Biotechnology and is a member of the Board of Directors of Clinical Supplies Management. He currently serves as an Advisory Board Director at the Fairleigh Dickinson School of Pharmacy. He serves as a Director of BioNJ and BIO’s Emerging Company Board. Mr. Sapirstein received his Pharmacy degree from the Ernest Mario School of Pharmacy at Rutgers University, and his Masters of Business Administration degree from Farleigh Dickinson University.
Kathryn MacFarlane, Pharm.D. – Director
Kathryn MacFarlane has over 25 years of experience in the pharmaceutical industry with expertise in marketing, new product planning, and commercialization. She is currently an owner and Managing Partner of SmartPharma LLC, a pharmaceutical consulting firm specializing in commercial consulting for emerging pharmaceutical companies. She also serves as Senior Vice President of Napo Pharmaceuticals. Her expertise includes market assessment and commercial planning for products in development, as well as evaluating products for licensing or acquisition. Her experience spans multiple therapeutic areas, including Women’s Health, CNS, Cardiology, Vaccines, and Dermatology. Prior to Napo Pharmaceuticals, Dr. MacFarlane was the Chief Commercial Officer of Agile Therapeutics. Before joining Agile Therapeutics, Dr. MacFarlane served as President and CEO at Xintria Pharmaceutical Corporation and also served as Vice President of Women’s Health and New Product Planning at Warner Chilcott. She had responsibility for the launches of Lipitor®, Celexa®, and Loestrin® 24.
In 1999, she was named a Distinguished Alumna, and in 2012, she was named the Eaton Entrepreneur of the Year by the Purdue University School of Pharmacy. She has completed a Postdoctoral Fellowship in Industrial Pharmacy Practice with Rutgers University and Hoffmann- LaRoche. Dr. MacFarlane currently serves on the Purdue University School of Pharmacy Dean’s Advisory Council and is a Founding Member and Advisor to IPhO. She also serves on the Board of Directors for INMED Partnerships for Children, an NGO dedicated to providing food security and health services to women and children. Dr. MacFarlane received her Bachelor of Science in Pharmacy and Doctor of Pharmacy degrees from Purdue University.
John J. Greer, Ph.D. – Scientific Advisor
Dr. John Greer is a former Director of Neuroscience and Mental Health Institute (NMHI) in University of Alberta. He is also a faculty member of the Department of Physiology, Perinatal Research Centre, and Women & Children’s Health Research Institute. Dr. Greer completed post-doctoral fellowships at the University of California, Los Angeles. He holds grants in two primary areas of research pertaining to neuromuscular control of breathing. The first funded initiative, regarding the neurochemical control of breathing, led to the development of ampakine therapy for overcoming respiratory depression caused by analgesics, anesthetics and certain disease states. The second funded initiative has provided the impetus for his laboratory to become prominent for its studies of the pathogenesis and etiology of the developmental anomaly, congenital diaphragmatic hernia. Dr. Greer is the inventor of the use patents licensed by RespireRx for the use of ampakines that the Company is developing for the treatment of drug-induced respiratory depression and central and mixed-sleep apnea.
Cautionary Note Regarding Forward-Looking Statements
This document contains certain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934 that are intended to be subject to the safe harbor created thereby. These might include statements regarding RespireRx’s financial position, business strategy and other plans and objectives for future operations, and assumptions and predictions about research and development efforts, including, but not limited to, preclinical and clinical research design, execution, timing, costs and results, future product demand, supply, manufacturing, costs, marketing and pricing factors, which are all considered forward-looking statements.
In some cases, forward-looking statements may be identified by words including “anticipates,” “believes,” “intends,” “estimates,” “expects,” “plans,” and similar expressions include, but are not limited to, statements regarding (i) future research plans, expenditures and results, (ii) potential collaborative arrangements, (iii) the potential utility of RespireRx’s proposed products, and (iv) the need for, and availability of, additional financing.
The forward-looking statements included herein are based on current expectations that involve a number of risks and uncertainties. These forward-looking statements are based on assumptions regarding RespireRx’s business and technology, which involve judgments by management with respect to, among other things, future scientific, economic and competitive conditions, and future business decisions, all of which are difficult or impossible to predict accurately and many of which are beyond RespireRx’s control. Although it is believed that the assumptions underlying the forward-looking statements are reasonable, actual results may differ materially from those set forth in the forward-looking statements. In light of the significant uncertainties inherent in the forward-looking information included herein, the inclusion of such information should not be regarded as a representation by RespireRx or any other person that RespireRx’s objectives or plans will be achieved.
Factors that could cause or contribute to such differences include, but are not limited to, regulatory policies or changes thereto, available cash, research and development results, competition from other similar businesses, and market and general economic factors. The information contained herein should be read in conjunction with the condensed consolidated financial statements (unaudited) and notes thereto included in Item 1 of the Company’s most recently filed Quarterly Report on Form 10-Q and the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2015, including the section entitled “Item 1A. Risk Factors.” The forward-looking statements contained herein will not be updated or revised to reflect new information, future events or otherwise.
Vice-President, Treasurer and Secretary
Telephone: (917) 834-7206