Cynapsus Therapeutics Inc.
NASDAQ : CYNA
TSX : CTH

Cynapsus Therapeutics Inc.

June 29, 2015 06:00 ET

Cynapsus Therapeutics Enrolls First Patient in Pivotal Phase 3 Efficacy Study

Evaluating Sublingual, Film Strip Delivery of Apomorphine to Turn Parkinson's Disease Patients from the OFF to the ON State

CTH-300 Clinical Trial to Assess Effectiveness and Safety of APL-130277

TORONTO, ONTARIO--(Marketwired - June 29, 2015) - Cynapsus Therapeutics Inc. (NASDAQ:CYNA)(TSX:CTH) (the "Company") today announced enrollment of the first patient in the CTH-300 clinical trial, a pivotal Phase 3 study to examine the efficacy, safety and tolerability of APL-130277 for the acute treatment of OFF episodes in patients with Parkinson's disease (PD).

The CTH-300 trial is a double-blind, placebo-controlled, parallel-design study with an estimated enrollment of 126 PD patients in 35 centers who have at least one OFF episode every 24 hours, with total OFF time of at least two hours per day. The study objective is to evaluate the efficacy and safety of APL-130277 versus placebo in patients with PD. The 126 patients will each be observed for 12 weeks, with dosing at home and in clinic. The primary endpoint will be measured at week 12 in clinic and will be the mean change in the MDS-UPDRS Part III score at 30 minutes after dosing. The key secondary endpoint will be the percentage of patients who convert from the OFF to the ON state at or before 30 minutes of dosing with APL-130277 in clinic at the week 12 visit.

For additional information about the CTH-300 trial and to learn more about eligibility, patients can visit https://www.clinicaltrials.gov/ct2/show/NCT02469090.

"We are pleased to enroll the first patient in the CTH-300 Phase 3 study, which will provide an understanding of the role of APL-130277 for the on-demand treatment of OFF episodes associated with Parkinson's disease," said Dr. Albert Agro, Cynapsus' Chief Medical Officer. "We believe that the combination of Apomorphine as the only approved drug to rapidly treat OFF episodes and our sublingual film delivery technology could provide many patients with a rapid, reliable means of turning ON from the OFF state."

Anthony Giovinazzo, Cynapsus' President and Chief Executive Officer, said, "This is an important milestone for Cynapsus as we move into the first of our Phase 3 studies. We are another step closer to potentially bringing this new product to PD patients. Approximately 400,000 people with PD in the U.S. experience debilitating OFF episodes. When a PD patient is experiencing an OFF episode, they are often unable to perform simple daily tasks such as eating, bathing and dressing, thus becoming increasingly dependent on caregivers. We believe that APL-130277 can, if approved, change the way patients manage OFF episodes."

About Parkinson's Disease

Parkinson's is the second most common neurodegenerative disease worldwide. More than 1 million people in the United States and between four and six million people worldwide suffer from PD. There is no known cure or disease modifying treatment currently available for PD. Current medications and treatments only control the major symptoms of the disease, with most drugs becoming less effective over time as the disease progresses. PD results from the death of cells that produce dopamine, a neurotransmitter critical for movement. Current drugs and therapies either aim to supplement dopamine levels in the brain, mimic the effect of dopamine in the brain by stimulating dopamine receptors, referred to as dopamine agonists, or prevent the enzymatic breakdown of dopamine, prolonging its effect. The standard of care for the treatment of symptoms of PD remains oral levodopa, a drug approved nearly 50 years ago. While oral levodopa is efficacious, there are significant challenges for physicians in creating a dosing regimen of oral levodopa that consistently maintains levodopa levels within a patient's therapeutic range. Over time, the response to levodopa becomes less reliable and less predictable. As a result, the majority of PD patients experience OFF episodes despite taking PD medications.

About OFF Episodes

OFF episodes are thought to occur when brain dopamine levels fall below a critical threshold to sustain relatively normal motor function, or ON. It can be a period of time when a patient's PD medication is not working adequately to alleviate the patient's PD symptoms, when the medication has a delayed effect or does not work at all. When experiencing an OFF episode, a PD patient is unable to perform simple daily tasks such as eating, bathing and dressing, thus becoming increasingly dependent on caregivers. OFF episodes are considered one of the greatest unmet medical needs facing PD patients.

There are four main types of OFF episodes; however, all are the result of insufficient dopamine in the brain. Regardless of OFF type, it has been demonstrated that on average more than two-thirds of a patient's OFF time is actually spent waiting to turn ON after having taken levodopa. Current treatment options, other than apomorphine, consist of increasing the frequency of levodopa dosing or adding adjunctive PD medication, but these approaches do not address most of the OFF time (waiting to turn ON) and only can alleviate some of the end of dose wearing OFF. Apomorphine is currently the only approved acute, intermittent treatment to turn patients ON and provide rapid relief from OFF episodes.

About Apomorphine

Apomorphine is the most potent and fast-acting of all the dopamine agonists, which are used to stimulate dopamine receptors in place of naturally released dopamine, with similar efficacy to levodopa. No other dopamine agonist is potent enough to turn a levodopa-requiring patient from the OFF state to the ON state in which normal motor function is restored. Apomorphine is currently only available in the United States as a subcutaneous injection. Injectable apomorphine is painful to use, and administration is complex and inconvenient, requiring in excess of 15 steps. It is difficult for a PD patient to use while they are in the OFF state, and often requires administration by a caregiver. The injection may also cause scarring (nodules), irritation and injection site reactions. As a result, despite its highly efficacious profile, the adoption of injectable apomorphine has been limited.

About APL-130277

Cynapsus' APL-130277 is a "turning ON medication" designed to quickly and easily address all types of OFF episodes in PD patients, including morning OFF episodes, regardless of the stage of disease. APL-130277 is apomorphine administered orally as a sublingual thin film that the patient inserts under his or her tongue. It is designed to dissolve over the course of a few minutes, allowing apomorphine to rapidly enter the blood stream. The Company has studied APL-130277 in approximately 110 healthy volunteers and patients in five clinical studies. The most recent results from our Phase 2 clinical trial in PD patients met both the primary and secondary endpoints demonstrating a clinically meaningful improvement in motor function as early as 10 minutes and converting patients to full ON in as quickly as 15 minutes, with a durable effect lasting through 90 minutes. The study also demonstrated that APL-130277 treatment was safe and well-tolerated and has a safety profile comparable to all other dopamine agonists and levodopa. The sublingual delivery avoids the injection-related adverse events, or AEs, and the difficulty of use associated with the approved reference product, Apokyn. APL-130277 is designed to be easily administered and useable anywhere, anytime, generally with little or no assistance required, and is designed to make apomorphine accessible to all PD patients suffering from OFF episodes.

About Cynapsus

Cynapsus is a specialty central nervous system, or CNS, pharmaceutical company developing and preparing to commercialize a fast-acting, easy-to-use, sublingual thin film for the on-demand turning ON of debilitating OFF episodes associated with Parkinson's disease. The Company recently completed a Phase 2 clinical trial for its lead product candidate, APL-130277, a sublingual formulation of apomorphine hydrochloride, or apomorphine. Apomorphine is the only molecule approved for acute, intermittent treatment of OFF episodes for advanced PD patients, but it is currently only approved as a subcutaneous injection in the United States. APL-130277 is a "turning ON" medication designed to rapidly, safely and reliably convert a PD patient from the OFF to the ON state while avoiding many of the issues associated with subcutaneous delivery of apomorphine. It is designed to convert all types of OFF episodes, including morning OFF episodes, often considered the most difficult to treat. Cynapsus has initiated its Phase 3 clinical program for APL-130277, relying on the abbreviated Section 505(b)(2) regulatory pathway in the United States, and the Company intends to submit a new drug application, or NDA, in 2016.

Forward-Looking Statements

This announcement contains "forward-looking statements" within the meaning of applicable securities laws. These forward-looking statements include information about possible or assumed future results of the Company's business, financial condition, results of operations, liquidity, plans and objectives. In some cases, you can identify forward-looking statements by terminology such as "believe", "may", "estimate", "continue", "anticipate", "intend", "should", "plan", "expect", "predict", "potential", or the negative of these terms or other similar expressions. These forward-looking statements are based on the Company's current expectations and beliefs and inherently involve significant risks and uncertainties. Actual results and the timing of events could differ from those anticipated in such forward-looking statements as a result of risks and uncertainties, in include, but are not limited to those factors identified under the caption "Risk Factors" in the Company's filings and reports in the United States with the United States Securities and Exchange Commission (the "SEC") available on the SEC's web site, and in Canada with the various Canadian securities regulators, which are available online at www.sedar.com. Furthermore, unless otherwise stated, the forward-looking statements contained in this press release are made as of the date of this press release, and the Company has no intention and undertakes no obligation to update or revise any forward-looking statements, whether as a result of new information, future events, changes or otherwise, except as required by law. 

Neither the NASDAQ nor the TSX has approved or disapproved of the contents of this press release.

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