Boehringer Ingelheim’s Investigational Treatment for Cognitive Impairment Associated with Schizophrenia Receives FDA Breakthrough Therapy Designation

RIDGEFIELD, CT, May 24, 2021 – Boehringer Ingelheim today announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for BI 425809 for the treatment of Cognitive Impairment Associated with Schizophrenia (CIAS). The company also announced the planned initiation of the innovative CONNEX Phase III clinical trial program assessing the safety and efficacy of BI 425809 for improving cognition in adults with schizophrenia. BI 425809, a novel glycine transporter-1 (GlyT1) inhibitor, forms a key component of Boehringer Ingelheim’s Mental Health research program.

Together, this Breakthrough Therapy Designation and Phase III trial initiation are based on results from the Phase II 1346.9 clinical trial, which demonstrated that BI 425809 improved cognition in adult patients with schizophrenia¹. Central to many everyday tasks, impairment of cognitive function is a major burden for people with schizophrenia, and no pharmacological treatments are currently approved for CIAS².

“Cognition is a fundamental aspect of everyday life, including problem solving, memory and attention, which is why finding solutions for cognitive impairment is a key area of Boehringer Ingelheim Mental Health research,” said Vikas Mohan Sharma, M.D., Head of Medicine CNS, Retinopathies & Emerging Areas, Boehringer Ingelheim. “This Breakthrough Therapy Designation further highlights the urgent need for novel treatments for people living with schizophrenia. By combining traditional treatment approaches with new and innovative technologies, we are developing targeted therapies that will help to ease the burden of mental health conditions and enable people living with these conditions to create more meaningful connections to their lives, loved ones and society.”

The Breakthrough Therapy Designation process was established by the FDA to expedite the development and review of drugs for serious or life-threatening conditions where preliminary clinical evidence indicate that the therapy may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints.

The CONNEX trial program will incorporate disease specific speech biomarker technology from Aural Analytics that will be seamlessly embedded into VeraSci’s Pathway platform to provide a richer picture of trial participants’ cognition alongside more conventional clinical outcome measures. Additional information about the studies can be found at www.clinicaltrials.gov under the identifiers NCT04846868, NCT04846881, and NCT04860830.

“VeraSci is excited to take part in this landmark innovative trial in CIAS,” said VeraSci CEO Dr. Rich Keefe. “Innovative solutions are imperative to making meaningful progress in this difficult illness. We are hopeful that this research will be an important step forward for the field.”

"Interventions to improve patients’ lives are a key driver for our partnership with Boehringer Ingelheim and VeraSci as part of this first-of-a-kind trial program,” said Daniel Jones, Co-Founder & CEO from Aural Analytics. “Several of the symptoms of schizophrenia are generated by cognitive and emotional processes that can be identified through disruptions in the outward flow of speech. Using innovative speech analytics may help to objectively assess the downstream consequences of these disruptions."

As part of the broader CONNEX trial program, Boehringer Ingelheim will also use VeraSci’s Virtual Reality Functional Capacity Assessment Test (VRFCAT), an assessment that improves clinical trials by detecting functionally meaningful improvements in patients’ everyday lives, as a key secondary endpoint. The VRFCAT, which has been accepted into the FDA’s Clinical Outcome Assessment Qualification program, simulates key instrumental activities of daily living (iADLs) in a realistic and interactive environment and demonstrates sensitivity to basic functional capacity deficits.

About CONNEX Trial Program
CONNEX is a Phase III clinical trial program designed to assess the safety and efficacy of BI 425809 for improving cognition in adults with schizophrenia. The program is comprised of three clinical trials, including CONNEX-1, CONNEX-2 and CONNEX-3, all of which are Phase III randomized, double-blind, placebo-controlled parallel group trials to examine the efficacy and safety of BI 425809 once daily over a 26-week treatment period in patients with schizophrenia. The primary outcome measure is change from baseline in overall composite T-score of the Measurement and Treatment Research to Improve Cognition in Schizophrenia (MATRICS) consensus cognitive battery (MCCB).

BI 425809 is an investigational compound and has not been approved by the US Food and Drug Administration or any other regulatory authority. Safety and efficacy have not been established.

Boehringer Ingelheim Mental Health
At Boehringer Ingelheim Mental Health, we are redefining mental health to enable people to thrive. We link behavior to the underlying neurobiology to develop targeted therapies that can ease the burden of these conditions, not just the symptoms. By combining traditional treatment approaches with new and innovative approaches and technologies, we will enable those with mental health conditions to create more meaningful connections to their lives, loved ones and society.

About Boehringer Ingelheim Pharmaceuticals, Inc.
Making new and better medicines for humans and animals is at the heart of what we do. Our mission is to create breakthrough therapies that change lives. Since its founding in 1885, Boehringer Ingelheim is independent and family-owned. We have the freedom to pursue our long-term vision, looking ahead to identify the health challenges of the future and targeting those areas of need where we can do the most good.

As a world-leading, research-driven pharmaceutical company, with around 52,000 employees, we create value through innovation daily for our three business areas: Human Pharma, Animal Health, and Biopharmaceutical Contract Manufacturing. In 2020, Boehringer Ingelheim achieved net sales of around 22.33 billion USD (19.57 billion EUR). Our significant investment of over 4.2 billion USD (3.7 billion EUR) in 2020 (18.9% of net sales) in R&D drives innovation, enabling the next generation of medicines that save lives and improve quality of life.

We realize more scientific opportunities by embracing the power of partnership and diversity of experts across the life-science community. By working together, we accelerate the delivery of the next medical breakthrough that will transform the lives of patients now, and in generations to come.

Boehringer Ingelheim Pharmaceuticals, Inc., based in Ridgefield, CT, is the largest U.S. subsidiary of Boehringer Ingelheim Corporation and is part of the Boehringer Ingelheim group of companies. In addition, there are Boehringer Ingelheim Animal Health in Duluth, GA and Boehringer Ingelheim Fremont, Inc. in Fremont, CA.

Boehringer Ingelheim is committed to improving lives and strengthening our communities. Please visit www.boehringer-ingelheim.us/csr to learn more about Corporate Social Responsibility initiatives.

For more information, please visit www.boehringer-ingelheim.us, or follow us on Twitter @BoehringerUS.

About VeraSci
VeraSci is a clinical research company offering eClinical software, translation services, and expertise in endpoints and assessments. VeraSci brings scientific expertise, strategic innovation, and unwavering commitment to each project, helping clients worldwide develop novel therapies. Learn more at www.verasci.com.

About Aural Analytics, Inc.
Aural Analytics is the industry’s leading speech neuroscience company harnessing the clinical utility of the world’s speech across the age range and around the world. Their suite of applications and embeddable SDKs and APIs are available in up to 30 languages across Android, iOS, and the web are easy to use, secure, and provide robust, clinically relevant, interpretable, and validated metrics reflecting the neurological and respiratory health of its users. For more information, please visit auralanalytics.com or follow Aural Analytics on Twitter, LinkedIn, Medium, and Facebook.

MPR-US-101685

Media Contacts
VeraSci
Rebekkah Huss
Phone: 919-401-4642
Email: rebekkah.huss@verasci.com

Aural Analytics, Inc.
Media
Email: press@auralanalytics.com

References

1. Fleischhacker WW, Podhorna J, Gröschl M, et al. Efficacy and safety of the novel glycine transporter inhibitor BI 425809 once daily in patients with schizophrenia: a double-blind, randomised, placebo-controlled phase 2 study. Lancet Psychiatry. 2021;8(3):191-201. doi:10.1016/S2215-0366(20)30513-7
2. Van Rheenen TE, Lewandowski KE, Lipschitz JM, Burdick KE. Conducting clinical studies targeting cognition in psychiatry: guiding principles and design. CNS Spectr. 2019;24(1):16-21. doi:10.1017/S1092852918001074