Braeburn Pharmaceuticals Achieves Primary Endpoint in Pivotal Phase 2/3 Study of BB0817, Risperidone 6-month Implant for Treatment of Schizophrenia
Phase 3 Safety Study Underway; NDA filing targeted for year-end 2017
Princeton, New Jersey — April 26, 2017 — Braeburn Pharmaceuticals announced today that a 6-month study of the safety, tolerability and pharmacokinetics of transferring patients diagnosed with schizophrenia or schizoaffective disorder and stabilized on oral risperidone to BB0817 (risperidone) implant has met its primary endpoint.
The trial was designed to demonstrate comparable average plasma concentrations of risperidone and its active metabolite, 9-hydroxy-risperidone between the risperidone implant and oral risperidone. The study enrolled more than 50 patients, diagnosed with schizophrenia or schizoaffective disorder according to DSM-V and stable on a daily 4 mg oral dose of risperidone for at least eight weeks. Enrolled patients received three risperidone implants just under the skin in their upper arm. The study met its objectives and demonstrated that plasma concentrations of oral risperidone and 9-hydroxy-risperidone were comparable to the plasma levels of BB0817 and remained consistent throughout the 6-month study.
“Schizophrenia is a potentially devastating disease, but it can be well-managed through compliance with antipsychotic medication. BB0817 may provide a compelling option to help patients manage schizophrenia, consistent with Braeburn’s commitment to develop long-acting implantable and injectable treatments for stigmatized diseases where adherence is critically important,” said Behshad Sheldon, President and CEO of Braeburn Pharmaceuticals. “This clinical program in schizophrenia represents a strong complement to our initial therapeutic focus in addiction and pain. Looking ahead, we expect results from a Phase 3 risperidone safety study later this year and are targeting a year-end 2017 filing of a New Drug Application seeking approval for the risperidone implant.”
Efficacy and safety of the risperidone implant were also assessed. During the trial, 100 percent of patients remained stable with no clinically meaningful change in Positive and Negative Symptom Scale (PANSS) scores from baseline over the course of the six months. The systemic adverse events were similar to those of oral risperidone and included akathisia (9%), EPS (6%) and anxiety (6%). Implant site pain was the most common adverse event related to the procedure (11%), and was generally mild in intensity. Nearly all (94%) patients who had the opportunity to enroll in an extension phase of the study chose to receive a second set of implants.
Risperidone is currently the leading agent used for treatment of schizophrenia, however the utility of risperidone and other schizophrenia agents is dependent on consistent administration of therapeutic doses. Daily dosing of risperidone is particularly challenging for patients with schizophrenia where improvement in symptoms often leads to discontinuation of medication. Long-acting formulations of schizophrenia treatments are prescribed for approximately 23 percent of these patients, and are viewed as essential to optimizing patient outcomes. Although injectable formulations of risperidone are available, no FDA-approved formulation is currently available as a 6-month treatment.
“Compliance with medication is a very important clinical issue, and without it, serious consequences including relapse and hospitalization are more likely. Preventing non-compliance is an important goal for any successful clinical treatment. The 6-month risperidone implant, if approved, would offer physicians and patients an innovative approach to the treatment of schizophrenia,” said Rishi Kakar, M.D., Associate Medical Director for Segal Institute for Clinical Research in Florida and Principal Investigator for the trial. “The implant is administered through a short, in-office procedure, and provides a treatment duration that is more than twice as long as currently-marketed injectables.”
Schizophrenia is a serious disease characterized by a distortion in the process of thinking and of emotional responsiveness. It most commonly manifests as hallucinations, paranoid or bizarre delusions, or disorganized speech and thinking, and is accompanied by significant social or occupational dysfunction. Onset of symptoms typically occurs in young adulthood and the condition is chronic, often requiring life-long treatment to mitigate symptoms.
It has been estimated that schizophrenia affects approximately one percent of the adult population in the U.S., and approximately 24 million people worldwide. In the U.S., schizophrenia is prevalent equally in both genders and accounts for approximately 20 percent of hospital bed stays. While there is no cure for the disease, symptoms and risk of relapse — the re-emergence or worsening of psychotic symptoms — can be managed in most patients with appropriate antipsychotic treatment. However, it is estimated that only 50 percent of patients who begin treatment with an antipsychotic remain on that treatment six months later, which can undermine long-term treatment outcomes.
Risperidone is an atypical (or second-generation) antipsychotic. It was first approved for treatment of schizophrenia by the FDA in 1994, and is marketed under the trade name Risperdal®. Risperidone is currently marketed in oral and injectable formulations, but there is no currently-available implantable risperidone product and no formulation that delivers a stable dose for six months. The World Health Organization includes risperidone on its List of Essential Medicines, a list of the most important medications needed in a basic health system.
About MedLaunch Technology
The implant platform technology enables subcutaneous insertion of a cylindrical, non-biodegradable, flexible polymer that can be used to deliver long-acting formulations of daily, oral drugs. This innovative technology, which was acquired by Braeburn in 2015, is designed to provide patients with a continuous and consistent level of medication over the course of a six-month to one-year treatment regimen. The polymer membrane controls the rate of diffusion of the drug substance, thereby providing immediate release while improving drug delivery via controlled release. This technology is currently being used in development of the six-month risperidone implant and a tizanidine implant.
About Braeburn Pharmaceuticals
Braeburn Pharmaceuticals, an Apple Tree Partners company, is a commercial-stage pharmaceutical company delivering individualized medicine in neuroscience. Long-acting therapeutic treatment options can be essential to improving patient outcomes and facilitating recovery in neurological and psychiatric disorders, which are often complicated by stigma and present significant public health challenges. Braeburn’s commercial product, Probuphine® (buprenorphine) implant was approved by the FDA in May 2016. Braeburn’s investigational product pipeline consists of long-acting implantable and injectable therapies for serious neurological and psychiatric disorders, including opioid addiction, pain, and schizophrenia. Braeburn’s pipeline products are at various stages of clinical development and include weekly and monthly CAM2038, subcutaneous injection depot formulations of buprenorphine, being investigated in opioid addiction and pain, BB0417 buprenorphine/granisetron injectable for acute pain, and BB0817, six-month risperidone implant being investigated in schizophrenia. More information on Braeburn can be found at www.braeburnpharmaceuticals.com.
Sherry Feldberg for Braeburn Pharmaceuticals