RespireRx Pharmaceuticals

Novel Medicines for Respiratory Diseases

Overview

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.

Investment Highlights

  • 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