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Spinocerebellar Ataxia (SCA) is a rare, genetic, life-threatening neurodegenerative disease with no available treatment. Troriluzole demonstrated a 50-70% slowing of SCA disease progression on the primary and secondary outcome measures at the 3-year endpoint in a real-world evidence (RWE) study. Troriluzole has a well-established safety profile and if approved, would be the first and only FDA-approved treatment for SCA; subject to receipt of FDA approval, Biohaven is prepared to commercialize troriluzole for SCA in the US in 2025. NEW HAVEN, Conn. , Feb. 11, 2025 /PRNewswire/ -- Biohaven Ltd. (NYSE: BHVN) (Biohaven or the Company), today announced that the US Food and Drug Administration (FDA) has accepted for review the Company's New Drug Application (NDA) for troriluzole for the treatment of adult patients with spinocerebellar ataxia (SCA) and has granted Priority Review. This designation is assigned to applications for drugs that would offer a significant improvement over other available treatments for a given disorder or would provide a treatment option where none exists. In the case of SCA, a rare, genetic, neurodegenerative disease, troriluzole would be the first and only FDA-approved treatment for this life-threatening disorder. The FDA's decision regarding the NDA is expected within 6 months of filing (during 3Q2025). Based on FDA Priority Review timelines and if ultimately approved, Biohaven is prepared to commercialize troriluzole for SCA in the US in 2025. Melissa Beiner , M.D., SCA Clinical Development Lead at Biohaven , commented, "Our NDA filing is the culmination of over 8 years of clinical research and represents an important collaboration across the SCA community. The troriluzole NDA reflects rigorous scientific collaborations between advocacy groups, patients and their families, clinical experts in SCA and Biohaven . The FDA decision to grant Priority Review demonstrates the extremely high unmet need in this rare neurodegenerative disease. Time is of the essence for patients with SCA, who are suffering relentless and irreversible functional decline including impairments in coordination and balance leading to falls, loss of ambulation, and difficulties with vision, speech and swallowing." Dr. Beiner added, "The robust clinical data presented in the NDA demonstrate sustained and compelling treatment benefit in SCA patients treated with troriluzole, a once-daily, oral pill. We look forward to working closely with the FDA throughout the review process to bring the very first treatment to patients and families suffering from SCA." The NDA submission was based, in part, on positive topline results from Study BHV4157-206-RWE (NCT06529146), in which troriluzole 200 mg dosed orally in patients with SCA met the study's primary endpoint of change from baseline on the functional Scale for the Assessment and Rating of Ataxia (f-SARA), in all SCA genotypes, at 3 years compared to an external control arm. Troriluzole showed statistically significant superiority across 9 consecutive, prespecified primary and secondary endpoints with highly consistent, sustained, robust and clinically meaningful treatment effects. SCA patients treated with troriluzole showed a 50-70% slower rate of decline, representing 1.5-2.2 years delay in disease progression, over the 3-year study period (Figures 1-3 and Table 1). The NDA also includes confirmatory and supportive data from Studies BHV4157-201 and BHV4157-206, the first large, multi-center registrational trials in SCA. Notably, these data include disease stabilization in the SCA3 genotype (Figure 4) and a reduction in falls in all SCA genotypes (Figure 5), both compared to placebo over 48 weeks in Study BHV4157-206. Jeremy Schmahmann , M.D., Professor of Neurology at Harvard Medical School and Founding Director of the Ataxia Center and the Martha and Robert Fogelman Endowed Chair in Ataxia and Cerebellar Neurology at Massachusetts General Hospital commented, "The FDA acceptance for review of this NDA represents a critical milestone for SCA patients. Since the discovery of the first gene for SCA in 1993, patients and families affected by SCA have watched generation after generation suffer severe, progressive disability and premature death with no treatment options. The need for an intervention that can slow disease progression and help patients maintain their independence is urgent. The delay in disease decline shown in the real-world evidence study is a watershed in the history of the SCAs. This is what patients have been waiting for. It is what the doctors who have been powerless, have been waiting for. Additionally, the importance of troriluzole's effects on reducing falls in this patient population cannot be overstated. I applaud the FDA for recognizing this urgency by granting a Priority Review and look forward to using troriluzole in the clinic if approved." Biohaven's troriluzole clinical development program in SCA collected data over 8 years, including a robust long-term safety profile, and was the first industry trial conducted in SCA. The external control arms used in Biohaven's BHV4157-206-RWE Study were provided from objective, third-party data gathered from two independent natural history cohorts: one in the United States and one in Europe (EUROSCA Natural History Study). The National Ataxia Foundation (NAF) sponsored the Clinical Research Consortium for the Study of Cerebellar Ataxia (CRC-SCA) that served as the basis for the US natural history cohort. The data from the CRC-SCA is managed by the University of South Florida Health Informatics Institute . A total of 35 clinical sites provided data in the US and European natural history cohorts that served as the external controls in BHV4157-206-RWE. As per instructions from FDA on the real-world evidence study design and statistical analysis plan, the external control arm was determined using a Propensity Score Matching (PSM) method to ensure that untreated subjects from the comparator natural history cohort were rigorously matched to treated subjects from the troriluzole arm of Study BHV4157-206. PSM was used on all prognostic, demographic, and baseline characteristics known to be associated with disease progression in SCA, including baseline f-SARA, age, sex, age at symptom onset, genotype, and trinucleotide repeat length (by genotype). Andrew Rosen , Chief Executive Officer of the National Ataxia Foundation (NAF), stated, " Biohaven's SCA program reflects years of dedicated clinical research and collaboration with leading world experts and advocacy groups to advance the ataxia field. We are proud that our multi-year effort to fund and support the Clinical Research Consortium for the Study of Cerebellar Ataxia played such a critical role in providing the external control arm of Biohaven's study. The goal of CRC-SCA is to improve our understanding of SCA disease progression and to promote the development of disease-modifying therapies for SCA." Mr. Rosen added, "On behalf of patients and families, who have watched generations of family members succumb to this devastating disease and have been waiting for decades for a treatment that could slow disease progression, I thank the FDA for not only accepting this NDA for review, but recognizing the need for urgency for our community in the form of a Priority Review." Biohaven previously received both Fast-Track and Orphan Drug Designation (ODD) from the FDA, and ODD from the European Medicines Agency , where a troriluzole MAA is currently under review. An expanded access protocol (EAP) is currently enrolling patients with SCA who are eligible. EAPs are designed to give early access to potential therapies before they are approved by the FDA. More information about can be found at https://clinicaltrials.gov/study/NCT06034886. About Spinocerebellar Ataxia (SCA)Spinocerebellar ataxia is a group of dominantly inherited neurodegenerative disorders characterized by progressive loss of voluntary motor control and atrophy of the cerebellum and brainstem. SCA affects approximately 15,000 people in the United States and 24,000 in Europe and the United Kingdom . Patients experience significant morbidity, including impaired gait leading to falls, loss of ambulation and progression to a wheelchair, inability to communicate due to speech impairment, difficulty swallowing, and premature death. While signs and symptoms can appear anytime from childhood to late adulthood, SCA typically presents in early adulthood and progresses over a number of years. Currently, there are no FDA-approved treatments and no cure for SCA. About TroriluzoleTroriluzole is a new chemical entity (NCE) and third-generation novel prodrug that modulates glutamate, the most abundant excitatory neurotransmitter in the human body. The primary mode of action of troriluzole is reducing synaptic levels of glutamate. Troriluzole increases glutamate uptake from the synapse, by augmenting the expression and function of excitatory amino acid transporters located on glial cells that play a key role in clearing glutamate from the synapse. The glutamate modulating activity of troriluzole addresses the widely documented glutamate deregulation that underlies neurodegeneration and Purkinje cell dysfunction in patients with SCA. Troriluzole also has the potential to be developed in a number of other diseases associated with excessive glutamate. More information about troriluzole can be found at the Biohaven's website: https://www.biohaven.com/pipeline/clinical-programs/glutamate/. About Biohaven Biohaven is a biopharmaceutical company focused on the discovery, development, and commercialization of life-changing treatments in key therapeutic areas, including immunology, neuroscience, and oncology. The company is advancing its innovative portfolio of therapeutics, leveraging its proven drug development experience and multiple proprietary drug development platforms. Biohaven's extensive clinical and preclinical programs include Kv7 ion channel modulation for epilepsy and mood disorders; extracellular protein degradation for immunological diseases; TRPM3 antagonism for migraine and neuropathic pain; TYK2/JAK1 inhibition for neuroinflammatory disorders; glutamate modulation for OCD and SCA ; myostatin inhibition for neuromuscular and metabolic diseases, including SMA and obesity; antibody recruiting bispecific molecules and antibody drug conjugates for cancer. For more information, visit www.biohaven.com. Forward-looking StatementsThis news release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. The use of certain words, including "continue", "plan", "will", "believe", "may", "expect", "anticipate" and similar expressions, is intended to identify forward-looking statements. Investors are cautioned that any forward-looking statements, including statements regarding the future development, timing and potential marketing approval and commercialization of development candidates, , including the potential approval and commercialization of troriluzole for SCA, are not guarantees of future performance or results and involve substantial risks and uncertainties. Actual results, developments and events may differ materially from those in the forward-looking statements as a result of various factors including: the expected timing, commencement and outcomes of Biohaven's planned and ongoing clinical trials; the timing of planned interactions and filings with the FDA; the timing and outcome of expected regulatory filings; complying with applicable U.S. regulatory requirements; the potential commercialization of Biohaven's product candidates; the potential for Biohaven's product candidates to be first approved therapies; and the effectiveness and safety of Biohaven's product candidates. Additional important factors to be considered in connection with forward-looking statements are described in Biohaven's filings with the Securities and Exchange Commission, including within the sections titled "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations". The forward-looking statements are made as of the date of this news release, 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. Investor Contact: Jennifer Porcelli Vice President, Investor Relationsjennifer.porcelli@biohavenpharma.com+1 (201) 248-0741 Media Contact: Mike Beyer Sam Brown Inc. mikebeyer@sambrown.com+1 (312) 961-2502 View original content to download multimedia:https://www.prnewswire.com/news-releases/biohaven-announces-fda-acceptance-and-priority-review-of-troriluzole-new-drug-application-for-the-treatment-of-spinocerebellar-ataxia-302373056.html SOURCE Biohaven Ltd.

SAN FRANCISCO--(BUSINESS WIRE)--InCarda Therapeutics, Inc.: Analysis of the results of the RESTORE-1 Phase 3 trial following its premature termination demonstrated statistically significant cardioversion of PAF with orally inhaled flecainide, providing compelling rationale for the continued development of FlecIH-103 in the target population of PAF patients. — The RESTORE-1 trial was stopped prematurely due to lower-than-expected efficacy and plasma flecainide levels during a blinded review of the data — There were no safety concerns associated with the administration of FlecIH-103 A novel drug delivery platform has shown the potential to enable efficient and reliable delivery of flecainide to the heart through the lung, appropriate for both hospital and home use Bridging Phase 1 study data to date establishes a path for future registration trials Today, InCarda, a privately held biopharmaceutical company developing an inhaled flecainide acetate inhalation solution (FlecIH-103) for the acute conversion of paroxysmal atrial fibrillation (PAF) to sinus rhythm (SR), announced significant progress made on several fronts: 1) Results of the RESTORE-1 Phase 3 trial were presented at the 2024 European Society of Cardiology meeting in London 1 on September 2, 2024, and demonstrated strong “patient proof of concept” data for the safe and effective cardioversion of patients with PAF who were administered inhaled flecainide. These results were particularly striking in light of the fact that the trial was stopped prematurely. 2) Transition to a novel and more efficient drug-delivery platform is supported by recent in vitro and Phase 1 clinical data. 3) Preliminary interim results from a Phase 1 clinical study designed to establish the dose and dosing regimen with the new drug delivery platform warrant continuation of the study for the potential advancement of FlecIH-103 into registrational trials. RESTORE-1 Phase 3 results RESTORE-1 was a randomized, placebo-controlled, Phase 3 trial, employing a commercially available jet nebulizer, to assess the efficacy and safety of FlecIH-103 for the treatment of patients with PAF. The trial was stopped prematurely after 54 of the planned 400 patients had been enrolled due to an unexpectedly low rate of blinded cardioversion compared to the experience in the Phase 2 study (INSTANT). The rate of cardioversion in RESTORE-1 was 31% in the treated group versus 47% in the Phase 2 INSTANT study. This outcome was caused by lower-than-expected flecainide plasma levels and unexpected drug-device incompatibility not previously observed in the Phase 2 clinical trial. The premature halting of the trial was not due to any safety concerns. Despite the early termination of the trial and a small sample size (n=54), an analysis showed that the 31% cardioversion rate was statistically significant in FlecIH-103-treated patients compared to placebo (p=0.04), with a median time to restoration of normal sinus rhythm (NSR) of approximately 13 minutes from the start of dosing. The study captured statistically significant pharmacoeconomic benefits from early restoration of SR following administration of FlecIH-103: a) 80% of patients whose AF converted to sinus rhythm were eligible for discharge from the hospital within 2 hours of dose administration, compared to only 30% of patients whose PAF did not convert (p=0.002) b) 83% of patients who converted from PAF to NSR within 90 min of dosing were symptom-free, while only 28% of patients whose PAF did not convert to SR were symptom-free (p<0.0001) c) The need for electrical cardioversion to restore sinus rhythm was 49% in the active arm vs. 83% in the placebo arm (p<0.05). There were no safety concerns or SAEs associated with the administration of FlecIH-103 in this study and no patients discontinued the study due to an adverse event (AE) or had an AE that required treatment. In summary, these results indicate that orally inhaled flecainide can provide safe and rapid restoration of sinus rhythm in patients presenting to the emergency room with symptomatic acute PAF. We believe FlecIH-103 delivered via oral inhalation has the potential to be clinically meaningful and address the important unmet medical need for the estimated one million U.S. patients who visit ERs with PAF each year. Improved drug-delivery platform The lower-than-expected flecainide plasma levels observed in RESTORE-1 using a jet nebulizer motivated the transition to Aerogen’s novel, high-performance drug-delivery platform that enables more efficient and reliable inhaled delivery of FlecIH-103. This platform includes: a) A novel vibrating-mesh aerosol generation engine proven to deliver drugs to the deep lungs for rapid systemic absorption, provided by Aerogen Ltd., a world leader in acute care aerosol drug delivery located in Galway, Ireland, and b) A unique patient-facing breath-synchronized dosing system that helps optimize the patient’s breathing for the inhalation of FlecIH-103. The Aerogen drug-delivery platform employs the PDAP (Photo Defined Aperture Plate) technology, a breakthrough patented vibrating mesh for medical aerosol delivery developed by Aerogen Ltd. The PDAP technology effectively aerosolizes our flecainide formulation (FlecIH-103) into a fine-mist aerosol. In addition, the patient is guided by the dosing system to synchronize their breathing with the generation of the aerosol. We believe this increases the efficiency of the delivery of FlecIH-103 to the heart and may enable a low nominal dose needed for rapid and safe cardioversion. Bridging Phase 1 clinical data Preliminary interim results from a Phase 1 study being conducted in Australia with FlecIH-103 administered with this new delivery platform show encouraging results: a) Peak flecainide plasma concentrations (C max ) similar to those achieved in the Phase 2 (INSTANT) clinical trial, which showed a conversion rate of ~ 50% b) One-third of the amount of FlecIH-103 was needed in the inhaler to achieve similar C max when compared to the amount needed with the commercial jet nebulizer used in RESTORE-1, and c) Electrocardiographic (ECG) changes that are transient and consistent with the pharmacokinetics (PK) of the inhaled flecainide and are within a safe range [Max ΔQRS duration < 30 msec] No new safety signals have been observed, no SAEs reported and all AEs resolved without sequelae; the FlecIH-103 safety pro consistent with that observed in prior studies. We believe the novel delivery platform will easily be adaptable in all patient care settings including the hospital and at-home settings. Future plans InCarda plans to complete the ongoing Phase 1 study, including the investigation of additional dose regimens such as administration of a booster dose similar to current approved products for treating cardiac arrhythmias. Inhaled flecainide is being developed for use in both a medically supervised setting (e.g., in-hospital, emergency rooms and clinics) and as a portable treatment for self-administration outside the hospital. InCarda plans to reinitiate safety and efficacy trials in patients with PAF in 2025. Dr. Luiz Belardinelli, MD, InCarda’s Chief Medical Officer, said, “We believe that inhaled flecainide has the potential to be a valuable treatment for patients with recent-onset episodes of PAF. While RESTORE-1 did not achieve the level of efficacy desired and the trial was terminated early, the analysis showed a statistically significant higher conversion rate in the active arm compared to the placebo for the 54 patients evaluated, and the continued encouraging safety and tolerability pro FlecIH-103 suggest that this treatment could become an important option for patients with acute episodes of recent-onset of PAF. The trial also indicated that patients whose PAF converted to NSR had their symptoms relieved, required fewer interventions, and were discharged earlier from the ER, thus suggesting attractive pharmacoeconomic benefits of orally inhaled flecainide.” Carlos Schuler, Ph.D., co-founder and CEO of InCarda, said, “I am very excited by the ability of the new drug-delivery platform to deliver flecainide reliably. Given the positive Phase 1 results to date, I am optimistic that we will be able to achieve commercially attractive efficacy and safety and demonstrate pharmacoeconomic benefits in the registrational studies currently being planned.” About Atrial Fibrillation (AF) Atrial fibrillation (AF) is the most common sustained cardiac arrhythmia (abnormal heart rhythm) and is characterized by rapid and irregular heartbeats often resulting in palpitations and other symptoms that can be debilitating. A chronic, progressive condition, AF is estimated to affect six million people in the U.S., with that number expected to double by 2050 2 . This expected increase is partially due to the correlation between AF prevalence and an aging population, with approximately 9% of those aged 65 and older affected by the condition 2 . AF is associated with significant morbidity and a substantial reduction in quality of life, with the condition potentially resulting in exercise intolerance, congestive heart failure, tachycardia-induced cardiomyopathy, and stroke. The annual cost of AF to the U.S. healthcare system is estimated at more than $26 billion 2 . Paroxysmal AF (PAF) is a self-terminating type of AF in which episodes occur intermittently and resolve spontaneously in fewer than seven days. Approximately 25% of PAF patients progress to the permanent form of AF within five years 3 . Common symptoms of PAF can include a racing heartbeat, chest pain or pressure, a fluttering feeling in the chest, weakness, fatigue, dizziness, sweating, and lightheadedness. Current treatments for patients with PAF rely upon either chronic administration of oral antiarrhythmic drugs or acute hospital-based procedures such as intravenous drug administration of rate-control medicines and electrical cardioversion, neither of which adequately address the unmet need of patients for a rapid-acting treatment that can be administered whenever an episode of PAF occurs. There are currently no approved treatments that can be self-administered by patients whenever or wherever an episode of PAF occurs. About Inhaled Flecainide Inhaled Flecainide is a novel inhaled therapeutic candidate designed to rapidly deliver flecainide, a well-established oral antiarrhythmic agent, to the heart via the lungs to restore normal sinus rhythm and relieve the patient’s symptoms following the onset of new or recurrent episodes of paroxysmal AF (PAF). Previously completed Phase 1 clinical studies in healthy volunteers demonstrated that orally inhaled flecainide can rapidly and safely deliver the drug and result in ECG changes consistent with the potential to restore sinus rhythm in patients with PAF. A previously completed Phase 2 trial (INSTANT) in patients with recent-onset PAF showed that inhaled flecainide with an administration regimen of approximately eight minutes can safely and rapidly convert recent-onset PAF to normal sinus rhythm. InCarda started RESTORE-1, a Phase 3 clinical trial, but halted the study as mentioned above. With intellectual property protection now potentially extendable through 2045 with the inclusion of the new delivery system, orally inhaled flecainide continues to have potential as a first-in-class treatment for acute management of PAF. We estimate over a billion-dollar market potential for this product, in addressing this significant unmet medical need. About InCarda Therapeutics InCarda Therapeutics, Inc. is a privately held, clinical-stage biopharmaceutical company developing first-of-their-kind inhaled therapies for acute cardiovascular diseases and conditions. The company is leveraging the ability of inhaled therapy to deliver medicine in the “first pass” to cardiac tissue, presenting a ‘small,’ but effective dose of the drug directly to affected regions of the heart. This permits rapid-onset, lower off-target tissue exposure of the drug, lower exposure to cardiac tissue, and has the potential to be patient self-administered in an ambulatory care setting (e.g., at home care). InCarda employs a de-risked approach by using approved drugs as candidates for the new dosing paradigm via inhalation. The company’s lead development product, orally inhaled flecainide, is in development to treat acute episodes of symptomatic PAF, a prevalent cardiac arrhythmia. For more information, please visit: . LinkedIn: Twitter: @InCardaThera References: RESTORE-1 abstract, Dr. M. Rienstra, MD (Netherlands), ESC, London, Sep 02, 2024 J Am Coll Cardiol. 2014 Dec 2;64(21):2305-7 Am Heart J. 2005 Mar; 149(3):489-96 Contacts Carlos Schuler, Ph.D. CEO and Co-founder Email: schuler.carlos@incardatherapeutics.com Phone: 510-422-5522

Submitted a new drug application (NDA) for troriluzole in Spinocerebellar Ataxia Type 3 (SCA3) to U.S. FDA in 2Q2023, marking the team's fourth NDA in approximately 3 years Released additional data from Kv7 platform, including Phase 1 safety data by dose groups for BHV-7000 that further validates differentiated profile Projected Phase 3 Spinal Muscular Atrophy trial to complete enrollment in 2023 Initiated Phase 1 study of brain penetrant TYK2/JAK1 inhibitor, BHV-8000, and anticipate beginning Phase 2 trial in Parkinson's disease next year Highlighted robust pipeline with multiple INDs planned to be filed within the next year, including pan IgG degrader for multiple immune-mediated diseases in 2023 NEW HAVEN, Conn., May 31, 2023 /PRNewswire/ -- Biohaven Ltd. (NYSE: BHVN) ("Biohaven"), a global clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of life-changing therapies for people with debilitating diseases, including ultra-rare disorders, will provide an overview of clinical progress, regulatory updates, and pipeline developments at an in-person R&D Day today, concurrently with the Yale Ventures' Innovation Summit 2023 taking place on May 31-June 1. Members of Biohaven's senior management team and key opinion leaders will meet with investors and research analysts. The clinical progress, regulatory updates, and pipeline developments to include: Troriluzole in SCA: Submitted New Drug Application (NDA) to the U.S. FDA for troriluzole for the treatment of spinocerebellar ataxia type 3 (SCA3), an ultra-rare, genetically-defined, neurodegenerative disease associated with progressive disability, frequent falls, loss of ambulation, speech and swallowing impairment, and premature death that is the most common SCA genotype worldwide. NDA supported by consistent treatment benefits observed in patients with genotype SCA3 in Study BHV4157-206 across multiple outcome measures including the change from baseline f-SARA at Week 48, CGI-I total score at Week 48, and a robust reduction in fall risk over the study period. A rigorous analysis by genotype was possible as patients were randomized by genotype strata at baseline prior to randomization in the pivotal Phase 3 48-week, double-blind, placebo-controlled phase of Study BHV4157-206. SCA3 represented 41% of study participants, consistent with being the most common subtype. SCA3 affects approximately 10,600 people in North America and in the EU and Japan. NDA further supported by a composite scale development (SCACOMS) and analysis of Study BHV4157-206, and confirmatory evidence of efficacy provided by data from the 3-year, long-term open-label (OLE) extension phase of two studies (BHV4157-206 and BHV4157-201) using a Matching Adjusted Indirect Comparison (MAIC) to an external control group. Biohaven has previously received Fast-Track and Orphan drug designation (ODD) from the FDA, and ODD from the European Medicines Agency, for troriluzole in SCA. Kv7 Platform: Highlighted progress and broad potential of Kv7 platform, including ongoing and planned development of BHV-7000 (Kv7.2/3 activator): Phase 1 EEG study with BHV-7000 in 1H2023. Pivotal studies with BHV-7000 in focal epilepsy and bipolar disorder planned to initiate in 2H2023. Burgeoning evidence for therapeutic benefits of targeting Kv7 in diverse, high unmet need indications. Bispecific Platform: Provided updates regarding planned INDs for targeted extracellular protein degradation franchise (including IgG, IgA, and autoantibody-specific degrader programs) and next-generation antibody-drug conjugate (ADC) technologies; IND application for novel IgG degrader, BHV-1300, on track for submission in 2023. TYK2/JAK1 Inhibition in Immune-Mediated Brain Disorders: Began dosing with BHV-8000 (an oral, brain-penetrant, dual TYK2/JAK1 inhibitor) in a ­­­Phase 1 study in normal healthy volunteers. Taldefgrobep Alfa: In Spinal Muscular Atrophy: Enrollment of approximately 225 patients in global Phase 3 trial now anticipated to complete in 2023. In Metabolic Disorders: Planned Phase 2 trial initiation in 2H2023. Vlad Coric M.D., CEO and Chairman of Biohaven, commented, "Today's R&D Day review of our robust pipeline highlights Biohaven's continued dedication to advance novel therapeutics for brain disorders and builds upon our team's successes with the prior approvals of Nurtec® ODT and Zavzpret™ that have changed the treatment paradigm in migraine." "The NDA we submitted for troriluzole for SCA3 represents approximately seven years of effort by the Biohaven team to bring forward a potentially new treatment for this ultra-rare disease. Advancing a new investigational drug for a rare disease that has no current therapy is a multi-year process that is the culmination of not only our own internal efforts but also close coordination with patient advocacy groups including the National Ataxia Foundation (NAF), leading academic researchers and regulatory agencies. Rare brain diseases are particularly challenging to research given relatively small populations of patients to run / participate in clinical trials, the need to rely on real-world data and often a lack of standardized ratings scales or biomarkers. We could not have advanced the SCA program this far without the leadership from the National Ataxia Foundation that has supported basic science research as well as the development of natural history cohorts in SCA that serve as an external control for longitudinal studies. The Biohaven clinical trials in SCA were a first of its kind in this area and utilized a newly developed rating scale (the functional SARA or f-SARA) that was developed in close consultation with the FDA using standard regulatory pathways to elucidate this new scale. We look forward to interactions with the US FDA, EMA and other regulatory agencies across the globe as our submissions advance in the review process," Dr. Coric added. "We are pleased that our decades-long investment in ataxia research and understanding of disease progression has accelerated treatment development for SCA," said Andrew Rosen, Executive Director of the National Ataxia Foundation. "Thank you to all NAF members who participated in these important trials." Bruce Car PhD, Biohaven's CSO, stated, "Given the lack of efficacious therapies for many brain disorders, we must urgently evaluate new mechanisms and approaches to change the treatment paradigm in this therapeutic area. Our R&D Day today demonstrates the commitment that we have given to this effort with exciting new approaches ranging from our submission of a glutamate modulator in SCA to immune modulation using small molecule degraders to ion channels targeting agents for epilepsy and mood disorders, as well as a biologic treatment to enhance muscle growth in SMA. Our scientific and clinical team is poised to file multiple new INDs from our deep pipeline and complete late-stage clinical trials in the next couple of years. It is an exciting time at Biohaven for our passionate employees, patients and investors as we advance these novel investigational drugs in our pipeline." The Research & Development Day presentations will be made available following the conclusion of the program on . About Biohaven Biohaven is a global clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of life-changing therapies for people with debilitating diseases, including rare disorders. Biohaven's experienced management team brings with it a track record of delivering new drug approvals for products for diseases such as migraine, depression, bipolar disorder and schizophrenia. The company is advancing a pipeline of therapies for diseases with little or no treatment options, leveraging its proven drug development capabilities and proprietary platforms, including Kv7 ion channel modulation for epilepsy and neuronal hyperexcitability, glutamate modulation for obsessive-compulsive disorder and spinocerebellar ataxia, myostatin inhibition for neuromuscular diseases, and brain-penetrant TYK2/JAK1 inhibition for immune-mediated brain disorders. Biohaven's portfolio of early- and late-stage product candidates also includes discovery research programs focused on TRPM3 channel activation for neuropathic pain, CD-38 antibody recruiting, bispecific molecules for multiple myeloma, antibody drug conjugates (ADCs), and targeted extracellular protein degrader platform technology (MoDE™) with potential application in neurological disorders, cancer, and autoimmune diseases. For more information, visit . Forward-looking Statements This news release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. The use of certain words, including "continue", "plan", "will", "believe", "may", "expect", "anticipate" and similar expressions, is intended to identify forward-looking statements. Investors are cautioned that any forward-looking statements, including statements regarding the future development, timing and potential marketing approval and commercialization of development candidates are not guarantees of future performance or results and involve substantial risks and uncertainties. Actual results, developments and events may differ materially from those in the forward-looking statements as a result of various factors including: the expected timing, commencement and outcomes of Biohaven's planned and ongoing clinical trials; the timing of planned interactions and filings with the Food and Drug Administration; the timing and outcome of expected regulatory filings; complying with applicable U.S. regulatory requirements; the potential commercialization of Biohaven's product candidates; the potential for Biohaven's product candidates to be first in class therapies; and the effectiveness and safety of Biohaven's product candidates. Additional important factors to be considered in connection with forward-looking statements are described in Biohaven's filings with the Securities and Exchange Commission, including within the sections titled "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations". The forward-looking statements are made as of the date of this new release, 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. MoDEs is a trademark of Biohaven Therapeutics Ltd. Investor Contact: Jennifer Porcelli Vice President, Investor Relations [email protected] +1 (201) 248-0741 Media Contact: Mike Beyer Sam Brown Inc. [email protected] +1 (312) 961-2502 SOURCE Biohaven Ltd.