NeurOp Receives FDA Orphan Drug Designation for NP10679 for Treatment of Subarachnoid Hemorrhage

Atlanta, GA – December 8, 2021 – NeurOp, Inc., a clinical-stage biotechnology company focused on neurological and psychiatric disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to the company’s investigational candidate NP10679 for the treatment of subarachnoid hemorrhage (SAH).

SAH is a life-threatening type of stroke caused by bleeding into the space surrounding the brain. In patients who experience SAH caused by a ruptured aneurysm, vasospasm producing ischemic damage occurs in over one-third of patients during the week following the event and often results in permanent disability or death.

The mechanism by which ischemia damages the brain involves overactivation of NMDA receptors. Ischemia also induces acidity of brain tissue. Together, these insights led NeurOp to develop NMDA receptor blockers with increased potency in acidic tissue. NeurOp’s lead compound NP10679 is a highly selective antagonist for NMDA receptors and has greater potency in acidic conditions. These properties will enable it to provide neuroprotection against ischemic injury caused by vasospasm in SAH with fewer negative side effects than currently available NMDA inhibitors and other treatments.

“NP10679 demonstrated safety, tolerability and positive pharmacokinetics in our Phase 1 studies that make it an attractive candidate for prophylactic treatment following SAH,” said James McNamara, M.D., Executive Chairman of NeurOp. “Based on its encouraging profile, we look forward to commencing a Phase 2 clinical trial of NP10679 for SAH in 2023.”

The FDA Office of Orphan Products Development grants orphan drug designation to investigational drugs and biologics intended for the treatment, diagnosis or prevention of rare diseases, which are defined as those that affect fewer than 200,000 people in the U.S. The program provides certain benefits that include tax credits and application fee waivers designed to offset some development costs, as well as seven-year marketing exclusivity to sponsors of approved orphan products.

About NP10679
NeurOp is developing NP10679 as a subunit-specific NMDA receptor inhibitor for CNS disorders. NMDA receptors are activated by the neurotransmitter glutamate, the predominant excitatory transmitter in the brain. Several neurological disorders, including pain, treatment-resistant depression and brain damage resulting from acute brain injury, such as stroke or SAH, are associated with overactivity of these receptors. NP10679 is selective for a specific NMDA subtype, GluN2B, and has increased potency in acidic conditions. Its enhanced selectivity and disease context-dependent target engagement may provide neuroprotection with fewer negative side effects than currently available NMDA inhibitors.

About NeurOp, Inc.
NeurOp, Inc. is a privately held, clinical-stage biopharmaceutical company based in Atlanta, Georgia that is developing small-molecule therapies for central nervous system disorders, including severe pain, treatment-resistant depression, subarachnoid hemorrhage (SAH) and stroke. Its proprietary compounds selectively inhibit the GluN2B subunit of neuronal NMDA receptors and are designed for potential therapeutic benefit with an improved safety and tolerability profile relative to other NMDA receptor antagonists. For more information, please visit www.neuropinc.com or follow us on LinkedIn.

NeurOp Announces Positive Topline Data from Phase 1 Studies for Lead Candidate NP10679 for CNS Disorders

Results show excellent half-life and positive safety profile to enable testing in stroke, severe pain, subarachnoid hemorrhage and treatment-resistant depression

Atlanta, GA – May 28, 2020 – NeurOp, Inc., a privately held, clinical-stage biotechnology company focused on neurological and psychiatric disorders, today announced the successful completion of Phase 1 studies of NP10679, a highly potent and selective GluN2B subunit-specific NMDA (N-methyl-D-aspartate) receptor inhibitor. NeurOp is investigating NP10679 for several CNS disorders such as stroke, severe pain, subarachnoid hemorrhage and treatment-resistant depression, which are associated with over-activity of NMDA receptors.

The primary objective of the studies was to evaluate the safety and tolerability of NP10679 in healthy subjects. Secondary objectives included assessment of pharmacokinetics and pharmacodynamics.

The initial Phase 1 trial was a first-in-human randomized, placebo-controlled, single ascending dose (SAD) study that assessed NP10679 in healthy volunteers. The study included six single ascending dose cohorts (5 to 200 mg). The study enrolled 48 subjects, 36 of whom received study drug while the remaining 12 subjects received placebo. A second Phase 1 study employed a multiple ascending dose (MAD) design assessing the effects of NP10679 on 24 subjects in three cohorts (25, 50 and 100 mg once daily) over five days.

The pharmacokinetic profile of NP10679 in humans indicated clear dose linearity across the dose ranges tested and an excellent half-life of approximately 17 hours. The results from both studies also revealed a benign safety profile that should allow adequate dosing to test the molecule in a number of indications.

“Development of NP10679 rests on a foundation of outstanding science. Based on the encouraging safety profile and positive pharmacokinetics demonstrated in our Phase 1 clinical studies, we look forward to advancing NP10679 into Phase 2 trials for stroke,” said James McNamara, M.D., Executive Chairman of NeurOp. “We plan to initiate Phase 2 studies in early 2021.”

The phase 1 NP10679 program was conducted in collaboration with Pharmaron (Baltimore). Data collected from this study will inform dose and schedule for further development of NP10679.

About NP10679
NP10679 is being developed as subunit-specific NMDA receptor inhibitor for CNS disorders. NMDA receptors are activated by the neurotransmitter glutamate, the predominant excitatory transmitter in brain. Several neurological disorders, including pain, treatment-resistant depression, and brain damage resulting from acute brain injury, such as stroke or subarachnoid hemorrhage (SAH), are associated with over-activity of these receptors that leads to significant acidification in brain tissues. NP10679 is selective for a specific NMDA subtype, GluN2B, and has increased potency in acidic conditions. This enhanced selectivity and disease context-dependent target engagement may provide neuroprotection with fewer negative side effects than currently available NMDA inhibitors.

About NeurOp, Inc.
NeurOp, Inc. is a clinical-stage biopharmaceutical company based in Atlanta, Georgia that is developing small-molecule therapies for central nervous system disorders, including severe pain, subarachnoid hemorrhage (SAH) and stroke. Its proprietary compounds selectively inhibit the GluN2B subunit of neuronal NMDA receptors for potential therapeutic benefit with fewer side effects than currently available NMDA receptor antagonists. For more information, please visit www.neuropinc.com.

NeurOp Initiates Phase 1 Clinical Trial of NMDA Receptor Inhibitor NP10679

NeurOp, Inc., a clinical-stage biotechnology company, today announced the initiation of a Phase 1 clinical trial of NP10679, a highly potent and selective GluN2B subunit-specific NMDA (N-methyl-D-aspartate) receptor inhibitor. NeurOp is investigating NP10679 for several neurological disorders that are associated with over-activity of these receptors.

The randomized, placebo-controlled, single ascending dose study will assess the safety, pharmacokinetics and pharmacodynamics of NP10679 in healthy volunteers. The study is being conducted in collaboration with Pharmaron (Baltimore) and is currently enrolling subjects in the United States. Data collected from the study will inform dose and schedule for further development of NP10679 for potential use in severe pain and the prevention of ischemic damage following subarachnoid hemorrhage (SAH).

Certain areas of the brain become acidified by metabolic insufficiency or increased neuronal activity. This condition or “context” exists in severe pain and SAH and may also exist in brain areas that are undergoing seizures or involved in nicotine or opioid addiction.

“NP10679 is a first-in-class therapy as a context-dependent NMDA receptor inhibitor,” said NeurOp’s Chief Scientific Officer, Robert Zaczek, PhD. “Preclinical data demonstrate that NP10679 is different from other NMDA therapies, because of its selectivity, safety profile and potency at low pH, a condition found in a number of central nervous system disorders.”

“We have achieved a significant milestone for NeurOp now that NP10679 has advanced to the clinic,” said James McNamara, MD, Executive Chairman at NeurOp. “We look forward to moving this compound through clinical development and are particularly encouraged by its potential as a treatment for severe pain. With the opioid epidemic facing the nation, the need for effective and safe medications for severe pain is greater than ever.”

NP10679 is bioavailable either orally or by IV, and it is currently being evaluated in this study by the IV route. An IND for NP10679 was opened in 2016.

About NeurOp
NeurOp, Inc. is a clinical-stage biopharmaceutical company based in Atlanta that is developing small-molecule therapies for central nervous system disorders, including severe pain, subarachnoid hemorrhage (SAH) and catastrophic juvenile epilepsies. Its proprietary compounds selectively inhibit the GluN2B subunit of neuronal NMDA receptors for potential therapeutic benefit with fewer side effects than currently available NMDA receptor antagonists. For more information, please visit www.neuropinc.com.

NeurOp contact:
Robert Zaczek, PhD
Phone: 860.853.0427

NIH Awards NeurOp $3.5 Million to Support Phase 1 Clinical Trial of NMDA Inhibitor NP10679

NP10679 is in development to prevent brain ischemia during stroke or subarachnoid hemorrhage

NeurOp, Inc. today announced that it has received a $3.5 million award from the National Institute of Neurological Disorders and Stroke (NINDS), a division of the NIH, to begin clinical testing of the Company’s drug candidate NP10679, a GluN2B subunit-specific NMDA (N-methyl-D-aspartate) inhibitor. NeurOp is investigating NP10679 for the prevention of ischemic damage during a stroke or subarachnoid hemorrhage (SAH).

“We designed NP10679 with great care to incorporate the attributes we believe differentiate this molecule from other NMDA inhibitors in development,” said Barney Koszalka, PhD, NeurOp CEO. “For example, the binding of the molecule is enhanced in an acidic environment, a property other NMDA inhibitors lack. This property of pH dependence improves the chance of achieving efficacy at dose levels devoid of side effects. We would like to partner with a pharmaceutical company in future trials to fully explore this advantage in an array of disorders such as stroke, treatment-resistant depression and neuropathic pain.”

NP10679 is bioavailable by either the oral or IV route, and it will initially be evaluated in a Phase 1 study in healthy human volunteers by the IV route. The study is expected to start in early 2018. Pre-clinical studies have shown efficacy in treating complications associated with SAH. An IND for NP10679 was opened in 2016.

NeurOp’s Chief Scientific Officer, Robert Zaczek, PhD, added, “The safety profile of NP10679 allows for prophylactic use in patients at risk for an ischemic event, such as those suffering an SAH. This is important because extensive data has shown that early intervention is key for robust efficacy of neuroprotective therapy. Our prophylactic intervention strategy will place NP10679 at its site of action before an SAH-driven delayed cerebral ischemia event takes place. This eliminates the time-of-dosing caveat that might, in part, have led to previous failures of clinical tests involving glutamatergic agents in stroke and head trauma.”

Note: Research reported in this news release is supported by the National Institute of Neurological Disorders and Stroke of the National Institutes of Health (NIH) under Award Number R44NS071657. The content is solely the responsibility of the authors and does not necessarily represent the official views of the NIH.

About NeurOp
NeurOp, Inc. is an Atlanta-based biopharmaceutical company developing new medicines for central nervous system disorders, including depression, neuropathic pain, ischemia (stroke), schizophrenia and Parkinson’s disease. Its research targets specific subunits of neuronal NMDA receptors to identify and evaluate small molecule modulators for potential therapeutic benefit. Multi-year funding from the NIH supports the Company’s research and development programs for NP10679 for the prevention of ischemic damage during a stroke or subarachnoid hemorrhage. For more information, please visit www.neuropinc.com.

NeurOp contact:
Barney Koszalka, PhD, CEO

Letter to Shareholders | 2015 Highlights

As we have in previous years, I’m writing to update you on the progress we have made this past year and the direction NeurOp is heading for the remainder of 2016 and into 2017.  We will continue to update our website with current information (www.neuropinc.com ) and events and also invite you to join our LinkedIn page. This year has proven to be a challenge, and our lead compound, NP10679, is on track to have an open IND by the summer’s end. The support for this program from the NIH remains strong, and we are confident of continued support from this agency to initiate Phase I trials. Feel free to reach out to me with any questions.

Regards,
Barney Koszalka, PhD
President and CEO
NeurOp, Inc.

Biotech Showcase 2016 | San Francisco

NeurOp will present at the 8th Annual Biotech Showcase 2016 in San Francisco, CA to be held January 11-13th. Biotech Showcase is an investor and networking conference devoted to providing private and public biotechnology and life sciences companies with an opportunity to present to, and meet with, investors and pharmaceutical executives at one of the industry’s largest annual healthcare investor conferences.

Tuesday, January 12, 2016
9:30 am PT
Track: A – Hearst

Biotech Showcase 2016
Parc 55 San Francisco – Union Square
55 Cyril Magnin St., 4th Floor
San Francisco, CA  94102

NeurOp’s Lee Latimer Elected to ACS 2016 Board of Directors

NeurOp, Inc. today announced that Lee H. Latimer, Ph.D., head of chemistry, has been elected as Director-at-Large for the American Chemical Society’s (ACS) 2016 Board of Directors. The Society announced election results on November 5.

Dr. Latimer has been a member of ACS since 1972 and became an ACS Fellow in 2012. He has held leadership positions at the local and national level of the organization and been recognized with a number of awards for his contributions.

“ACS is about opportunity for its members in so many ways. It has certainly been so for me. I enjoy meeting new challenges and colleagues in teams and applying my experience to make a difference,” said Latimer.

Dr. Latimer earned his Ph.D. in organic chemistry from the University of Wisconsin under the mentorship of B.M. Trost and held an NIH Fellowship with W.G. Dauben at the University of California at Berkeley and C.J. Sih at the University of Wisconsin at Madison. To learn more about Dr. Latimer’s extensive professional experience, click here.

About NeurOp
NeurOp, Inc. is an Atlanta-based biopharmaceutical company developing new medicines for central nervous system disorders, including depression, neuropathic pain, ischemia (stroke), schizophrenia, and Alzheimer’s and Parkinson’s diseases. Its research targets various subunits of neuronal NMDA receptors and their potential therapeutic benefit. The company has licensed its technology to Bristol-Myers Squibb for the development of a compound for treatment-resistant depression. Multi-year funding from the NIH supports the company’s research and development programs for schizophrenia, Alzheimer’s disease and NP10679, its drug candidate for the prevention of ischemic damage during a stroke or subarachnoid hemorrhage. For more information, please visit www.neuropinc.com.

NeurOp contact:
Barney Koszalka, CEO
Phone: (919) 260-5595

Letter to Shareholders | 2014 Highlights

Dear NeurOp Shareholder:

I want to update you on the substantial progress we made in the last year and the opportunities we see in front of us for 2015 and beyond. Our drug discovery efforts, focused on the NMDA receptor, have yielded two development candidates that will advance in 2015. I am pleased to share that we reached a significant milestone in our collaboration with Bristol-Myers Squibb (BMS) in the area of depression. BMS selected a development candidate, which triggered a milestone payment to NeurOp. In addition, the NIH continues to show strong support and enthusiasm for our subarachnoid hemorrhage (SAH) program, because they see intervention in this indication as a major unmet medical need.

In addition to moving these programs toward clinical testing, we made progress in identifying promising new applications for NMDA modulators outside those targeted for SAH and depression. NeurOp began a research collaboration with Janssen Pharmaceuticals to identify molecules that may yield further medical applications of NMDA receptor modulation.

Depression
Since BMS internalized the depression program in 2013, our access to information on the progression of the program is limited. However, in late June, BMS elevated compound NP11948 to development status. They are now evaluating the compound in the necessary experiments to file an Investigational New Drug (IND) application with the FDA. Typically, one would expect to this work to be completed in 2015, and we are hopeful that will be the case.

Ischemia & Addiction
NP10679, a compound meant to ameliorate or prevent brain damage and its clinical manifestations in those receiving treatment for SAH, met criteria set by us and the NIH to qualify it as a development candidate in 2013. Results from advanced pre-clinical studies performed in 2014 support the continued development of this molecule. Of significance, NP10679 showed a durable effect in animal studies. This observation, coupled with successful scale-up synthesis of drug substance and pre-IND cardiovascular studies, led us to a meeting with the FDA to review our planned submission to begin human testing.

The FDA review of the program reinforced our plan to move this program forward with all possible speed. The NIH granted additional funding of almost $400,000 to support our pre-IND studies, which brings the investment in this stage of the program to over $1.6 million for this year. We anticipate an IND filing in late 2015.

Last year, we mentioned an investigation of our compound’s ability to influence addiction – specifically, reducing the cravings associated with nicotine and opiate addiction. NeurOp conducted this research in collaboration with a leading researcher at the Medical University of South Carolina. The experimental findings in animals were positive in lessening cravings. These data suggest clinical application of NP10679 beyond SAH, and we are currently formulating an experimental strategy to enhance the compound’s value.

Further Leveraging the NMDA Platform
The NeurOp-Emory University collaborative research program helped us form another partnership with big pharma this past year. Janssen Pharmaceuticals and NeurOp entered into a short-term research agreement to find molecules that inhibit the NMDA receptor through GluN2C and/or GluN2D subunits. Although the program is in its early stages, we are encouraged and making progress to meet the yearly objectives for the program.

Looking Forward In 2015/16, we anticipate that the BMS depression program should reach a second milestone and trigger an additional payment to NeurOp. We will also continue our early drug discovery efforts by working to extend our agreement with Janssen and pursue an additional large pharma collaboration to identify NMDA modulators outside our current scope.

We expect that the ischemia program for NP10679 will have an approved IND and enter into clinical trials once we obtain funding. Despite having adequate funding to meet our 2015 objectives and complete the remaining studies required to file the IND, we will be unable to begin clinical trials without additional funds. NINDS, the division of the NIH that has supported the SAH program to date, has a limited budget for the initial Phase I trials we plan to start in 2016. While we will submit a grant application for this early clinical work, the total cost of the program is not likely to be covered. Securing additional funding remains a top priority for us.

The scientific news around modulating the NMDA receptor continues to be very positive, and NeurOp is entering a new and exciting phase in its evolution. We are energized by the potential of providing new medicines for unmet medical needs in many underserved areas of neuroscience, while at the same time growing value for our company.

I look forward to updating you throughout the year on our progress.

Very sincerely yours,

George Koszalka, Ph.D.
President and Chief Executive Officer, NeurOp, Inc.

NeurOp is committed to keeping you informed of our progress. We are working to launch a new website in the next few months that will allow us to share news, events and publications in a more timely, mobile-friendly format. You may sign up on our website to receive our latest news through an RSS feed. We also post news to our LinkedIn page, so you may wish to follow us there.