Edge Therapeutics Reports Initial Data from its Phase 1/2 NEWTON Trial for EG-1962 for Improvement of Outcomes after Ruptured Brain Aneurysm

February 11, 2015

Favorable response rate of 61% observed for EG-1962 patients in first two cohorts

No EG-1962-related hypotension reported

BERKELEY HEIGHTS, N.J.–(BUSINESS WIRE)– Edge Therapeutics, a clinical-stage biotechnology company that discovers, develops and seeks to commercialize novel, hospital-based therapies for the management of acute, life-threatening neurological conditions, today reported the progress of its ongoing Phase 1/2 NEWTON (Nimodipine microparticles to Enhance recovery While reducing TOxicity after subarachNoid hemorrhage) study. NEWTON is a multicenter, randomized, controlled, open label clinical study of the company’s lead product candidate, EG-1962, which is designed to treat patients that have suffered a subarachnoid hemorrhage from a ruptured brain aneurysm.

  • Edge has now completed enrollment of four cohorts of 12 patients (N = 48) in the NEWTON study, in which 36 were randomized to receive EG-1962 at 100 mg, 200 mg, 400 mg, and 600 mg and 12 were randomized to receive oral nimodipine, the current standard of care. Edge has also initiated enrollment of a fifth cohort of EG-1962 at an 800 mg dose.
  • 90-day efficacy data is available for 24 patients from the first two- NEWTON patient cohorts. The 90-day favorable outcome rate for patients treated with EG-1962 was 61% (N = 11) versus 17% (N = 1) for oral nimodipine.
  • Safety data is available for the first three cohorts (N = 36) and showed no evidence of hypotension in patients treated with EG-1962 while 33% (N = 3) of patients treated with oral nimodipine experienced hypotension.

Edge has collected safety and 90-day functional outcome efficacy data for 24 aneurysmal subarachnoid hemorrhage (aSAH) patients enrolled in the first two cohorts of NEWTON with 18 patients receiving 100 mg (cohort 1) or 200 mg (cohort 2) of EG-1962 and six patients receiving oral nimodipine. Of the 18 patients who received EG-1962, 61% (N = 11) experienced a favorable outcome on the extended Glasgow Outcome Scale (eGOS). The eGOS is a validated 8 point scale (1 = death, 8 = good recovery) used to assess recovery for patients who have suffered a ruptured aneurysm. A favorable outcome in the NEWTON study protocol is defined as an eGOS score between 6 and 8 as measured 90 days after treatment. By contrast, only 17% (N = 1) of the six patients treated with oral nimodipine in the first two cohorts of the NEWTON study had a favorable outcome on the eGOS.

Safety data from the third cohort (400 mg) who have not yet reached the 90-day functional outcome assessment are also available. Importantly, hypotension has not been observed among any patients who received EG-1962 in the first three cohorts (N = 27). By contrast, 33% (N = 3) of the 9 patients treated with oral nimodipine in the control group so far experienced hypotension. Safety and efficacy data for the fourth cohort are not yet available.

“Patients that have suffered a ruptured brain aneurysm are at great risk of death or severe brain damage as a result of delayed neurological complications for up to 14 days after the aneurysm has been treated,” said R. Loch Macdonald, MD, PhD and Chief Scientific Officer of Edge Therapeutics. “The improvement observed in patient outcomes associated with EG-1962 in the first two cohorts of the NEWTON study is very encouraging for patients and their clinicians.”

EG-1962 is a novel polymeric nimodipine microparticle that utilizes Edge’s proprietary PrecisaTM development platform and has the potential to improve patient outcome following aSAH. It is designed to avoid the dose-limiting side effects associated with standard of care oral nimodipine, including hypotension, by administering treatment directly to the site of the injury.

“We are pleased that EG-1962 has shown a consistent safety profile in the first three NEWTON cohorts,” said Brian Leuthner, President and Chief Executive Officer of Edge Therapeutics. “As we move closer to choosing an optimal dose for a Phase 3 study, we are also collecting important and very promising efficacy data that we anticipate will lead to clear endpoints for the next phase of EG-1962’s development. This preliminary data remain consistent with our goal at Edge to develop life-saving medicines for patients in very vulnerable conditions, such as following ruptured brain aneurysms. We look forward to providing top-line results from the NEWTON study during the first half of 2015 and plan to initiate Phase 3 activities in the second half of the year.”

The NEWTON study is evaluating the safety, tolerability and pharmacokinetics of EG-1962 compared to the current standard of care, oral nimodipine, in patients with aSAH. The company is also assessing patient functional outcomes at 90 days in each study cohort, which it believes will be indicative of the potential efficacy of EG-1962.

About Edge Therapeutics, Inc.

Edge Therapeutics is a clinical-stage biotechnology company that discovers, develops and seeks to commercialize novel, hospital-based therapies capable of transforming treatment paradigms in the management of acute, life-threatening neurological conditions. EG-1962, our lead product candidate, has the potential to fundamentally improve patient outcomes and transform the management of aneurysmal subarachnoid hemorrhage, or aSAH, which is bleeding around the brain due to a ruptured brain aneurysm. EG-1962 is a novel polymeric nimodipine microparticle that is administered directly into the brain ventricles. A single dose of EG-1962, administered initially at the time of aneurysm repair delivers a high concentration of nimodipine directly to the brain, with sustained drug exposure over 21 days. EG-1962 utilizes Edge’s proprietary, programmable, biodegradable polymer-based development platform, known as Precisa™. The Precisa™ platform allows Edge to create therapeutics capable of delivering medicines directly to the site of injury, providing a novel delivery mechanism that enables targeted and sustained drug exposure while potentially avoiding the systemic, dose-limiting side effects often associated with current standards of care. EG-1962 is currently being evaluated in the Phase 1/2 NEWTON study, a safety, pharmacokinetic and dose-escalation clinical trial. Our second product candidate, EG-1964, is being evaluated as a potential prophylactic treatment in the management of chronic subdural hematoma (cSDH), to prevent recurrent bleeding on the surface of the brain.

For more information on Edge Therapeutics, Inc., please visit: www.pdsbiotech.com.

Forward-Looking Statements

This press release and any statements of representatives and partners of the Company related thereto contain, or may contain, among other things, certain “forward-looking statements” as defined in the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve significant risks and uncertainties. Such statements may include, without limitation, statements with respect to the Company’s plans, objectives, projections, expectations and intentions, such as statements around the results of the NEWTON trial and timing of the Company’s Phase 3 trial, and other statements identified by words such as “projects,” “may,” “will,” “could,” “would,” “should,” “believes,” “expects,” “anticipates,” “estimates,” “intends,” “plans,” “potential” or similar expressions. These statements are based upon the current beliefs and expectations of the Company’s management and are subject to significant risks and uncertainties. Actual results may differ significantly from those set forth in the forward-looking statements. These forward-looking statements involve certain risks and uncertainties that are subject to change based on various factors (many of which are beyond the Company’s control), including, but not limited to, the timing and results of the Company’s clinical trials and the potential efficacy of EG-1962. The Company undertakes no obligation to publicly update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by applicable law.


Edge Therapeutics, Inc.
Gene Mack, 800-208-EDGE
Investor Relations