Biohaven Announces Obsessive-Compulsive Disorder (OCD) Proof Of Concept Phase 2/3 Study Results And Program Update

• Troriluzole 200 mg administered once daily as adjunctive therapy in OCD patients with inadequate response to standard of care treatment showed consistent numerical improvement over placebo on the Yale-Brown Obsessive Compulsive Scale (Y-BOCS) at all study timepoints (weeks 4 to 12) but did not meet the primary outcome measure at week 12, p < 0.05 at week 8 and p = 0.22 at week 12
• Given the strong signal in this proof of concept study, Biohaven plans to advance troriluzole to a full Phase 3 development program in OCD, with a larger sample size and the addition of a higher-dose arm
• Troriluzole was well tolerated with a safety profile consistent with past clinical trial experience

NEW HAVEN, Conn., June 24, 2020 /PRNewswire/ -- Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN) today reported topline results from its proof of concept study of troriluzole in the treatment of obsessive-compulsive disorder (OCD).

Table 1 shows the mean change in the Yale-Brown Obsessive-Compulsive Scale (Y-BOCS) total score over time. Troriluzole treated subjects had a mean Y-BOCS improvement of -5.1 points from baseline versus -3.6 for placebo-treated subjects [difference -1.5, p-value=0.041, 95% CI: -3.02, -0.06] at week 8, and -5.9 points versus -4.9 for placebo subjects [difference -1.0, p-value = 0.220, 95% CI: -2.59, 0.60] at week 12. Although the p-value in this proof of concept study did not reach statistical significance at the primary Y-BOCS endpoint at week 12, the results reveal a consistent treatment benefit of troriluzole over time and provide the appropriate data to power future studies.

Troriluzole treatment differences compared to placebo were greater in patients who were more severely ill at baseline (i.e., Y-BOCs total scores greater than the median score of 26, representing severe OCD symptoms), see Table 2. Troriluzole treated subjects (n=42) had a mean Y-BOCS change from baseline of -6.0 points versus -3.1 for placebo (n=45) subjects [difference -2.9, p=0.035, 95% CI: -5.49, -0.21] at week 8, and -7.0 points (n=44) versus -4.6 for placebo (n=43) subjects [treatment difference -2.4, p = 0.084, 95% CI: -5.18, 0.33] at week 12.

Vlad Coric, M.D., Chief Executive Officer of Biohaven, “While we have focused our primary investment to date on our lead CGRP program for migraine, we advanced troriluzole in a series of proof of concept trials to assess signal detection across disease states. Our strategy is to only further invest in troriluzole indications where we find an emerging drug signal. We are pleased to report these OCD study results that show a consistent and clinically meaningful drug effect at all study timepoints in patients who had an inadequate response to existing standard of care treatments. This study provides our R&D team with the necessary data to refine and adequately power subsequent trials to advance troriluzole in OCD. Based upon these results, we will have an End of Phase 2 meeting with the FDA and plan to initiate an appropriately powered pivotal Phase 3 study of two doses of troriluzole versus placebo.”

Obsessive-compulsive disorder is a serious psychiatric condition affecting over 2 million individuals in the U.S. and significantly impacts quality of life. Approximately 40% to 60% of OCD patients continue to experience significant residual symptoms despite approved therapies. Some refractory patients undergo psychosurgery (cingulotomy or deep brain stimulation) to alleviate their crippling symptoms.

Dr. Christopher Pittenger, M.D., Ph.D., Associate Professor of Psychiatry and Director of the Yale OCD Research Clinic, who was an investigator in the trial, commented, “As the first large scale, double-blind trial of troriluzole in OCD, this proof-of-concept study provides extremely encouraging initial data. OCD affects one person in 40 and can be debilitating. Fully a third of patients do not respond to current treatments, and many who do respond continue to suffer from residual symptoms. New therapies are urgently needed to alleviate this suffering and disability. There has not been a mechanistically novel medication approved for OCD in over 20 years. If troriluzole proves to be effective for these patients, it would be a huge advance. I look forward to working with Biohaven to explore the data from this proof-of-concept trial in more depth, to inform the design of future pivotal studies.”

Loren Aguiar, M.D., Biohaven’s Vice President of Research and Development stated, “The team conducted a well-designed, comprehensive proof of concept trial which has provided a wealth of data about the use of troriluzole as adjunctive therapy in OCD. We are continuing to analyze these data to investigate additional factors that may inform our next studies and further minimize placebo effect. The completion of the trial was complicated by the presence of restrictions surrounding the COVID-19 pandemic. We are grateful to the community of patients and researchers who persevered, allowing us to undertake and complete this challenging study.”

Biohaven has a deep and broad portfolio of assets in development with five ongoing Phase 3 trials across its calcitonin gene-related peptide (CGRP) antagonists, glutamate modulators and myeloperoxidase inhibitor platforms. Biohaven continues to focus resources on the NURTEC ODT launch for the acute treatment of migraine in adults, while preparing an sNDA submission for prevention. Earlier this year, troriluzole dosed at 280 mg successfully advanced past an interim futility analysis, performed after 100 subjects completed 6 months of treatment, in its pivotal Phase 2/3 trial in Alzheimer’s disease. Completion of the trial for troriluzole in Alzheimer’s Disease trial is expected in 4Q2020, and topline data from the troriluzole in SCA trial are anticipated in 2021. Additionally, Biohaven’s verdiperstat trial in Multiple System Atrophy (MSA) is enrolling ahead of schedule with topline results also expected in 2021.

About Troriluzole
Troriluzole is a third-generation prodrug and new chemical entity that modulates glutamate, the most abundant excitatory neurotransmitter in the human body. The primary mode of action of troriluzole is normalization of synaptic levels of glutamate. Troriluzole increases glutamate uptake from the peri-synaptic space, by augmenting the expression and function of excitatory amino acid transporters (i.e., EAAT2 also called GLT-1) located on glial cells that play a key role in clearing glutamate from the synapse. More information about troriluzole can be found on the Company’s website https://www.biohavenpharma.com/science-pipeline/glutamate/troriluzole

About Biohaven
Biohaven is a biopharmaceutical company focused on the development and commercialization of innovative best-in-class therapies to improve the lives of patients with debilitating neurological and neuropsychiatric diseases. Biohaven’s neuroinnovation portfolio includes FDA-approved NURTEC™ ODT (rimegepant) for the acute treatment of migraine and a broad pipeline of late-stage product candidates across three distinct mechanistic platforms: CGRP receptor antagonism for the acute and preventive treatment of migraine; glutamate modulation for obsessive-compulsive disorder, Alzheimer’s disease, and spinocerebellar ataxia; and myeloperoxidase (MPO) inhibition for multiple system atrophy and amyotrophic lateral sclerosis. For more information, visit www.biohavenpharma.com.

Forward-Looking Statements
This news release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements involve substantial risks and uncertainties, including statements that are based on the current expectations and assumptions of Biohaven’s management. All statements, other than statements of historical facts, included in this press release, including the expected enrollment for Biohaven’s Phase 2b/3 trial of troriluzole, the potential results of Biohaven’s Phase 2b/3 trial of troriluzole in OCD, the potential for the Phase 2b/3 trial to be a pivotal trial, the role of glutamate in OCD, the possible benefits of troriluzole as adjunctive therapy compared to current standard of care for OCD patients, as well as the timetable for the topline data and completion of trials, are forward-looking statements. The use of certain words, including the “believe” and “will” and similar expressions are intended to identify forward-looking statements. Biohaven may not actually achieve the plans and objectives disclosed in the forward-looking statements and you should not place undue reliance on Biohaven’s forward-looking statements. Various important factors could cause actual results or events to differ materially from those that may be expressed or implied by our forward-looking statements, including uncertainties relating to the future clinical success of troriluzole. Additional important factors to be considered in connection with forward-looking statements are described in the “Risk Factors” section of Biohaven’s Annual Report on Form 10-K filed with the Securities and Exchange Commission on February 26, 2020 and Biohaven’s Quarterly Report on Form 10-Q for the quarter ended March 31, 2020, filed with the Securities and Exchange Commission on May 7, 2020. The forward-looking statements are made as of this date and Biohaven does not undertake any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.

For further information, contact:
Dr. Vlad Coric
Chief Executive Officer
Vlad.Coric@biohavenpharma.com

NURTEC is a trademark of Biohaven Pharmaceutical Holding Company Ltd.

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SOURCE Biohaven Pharmaceutical Holding Company Ltd.

Company Codes: NYSE:BHVN