Verdiperstat

iStock/ higyou Launched in 2020 to more quickly bring to market an effective medicine for amyotrophic lateral sclerosis, the HEALEY Platform Trial has generated disappointing results for many but also continuing programs from Clene and Prilenia. Clinical wins have been few and far between for the HEALEY ALS Platform Trial . A heterogeneous patient population and delays have posed challenges, with several biotechs discontinuing programs based on underwhelming results. But optimism persists among the trial’s leaders and early participants, with two companies, Clene Nanomedicine and Prilenia Therapeutics, moving their investigational amyotrophic lateral sclerosis (ALS) treatments forward. The first seed for an ALS platform trial was planted when Massachusetts General Hospital’s Merit Cudkowicz read an article on master platform trials by former FDA Principal Deputy Commissioner Janet Woodcock. “I read that one and thought it might be appropriate for ALS,” Cudkowicz told BioSpace . Then, along came Sean Healey, a prominent American businessman who had recently been diagnosed with ALS, with an attractive opportunity. He said, “I might not save myself from this illness, but I want to make a difference. Give me a proposal,” Cudkowicz recalled. So she and her team worked around the clock, ultimately pitching the platform trial, and “[Healey] loved it. He just got it right away, that it was efficient and good for patients.” Healey, in partnership with his global asset management firm, Affiliated Managers Group (AMG), donated $40 million to launch the Sean M. Healey & AMG Center for ALS at Mass General, and set in motion a new chapter in ALS research. Cudkowicz serves as the center’s director. Biopharma quickly jumped on board with the platform trial, with dozens of companies—in addition to Clene and Prilenia, UCB, Biohaven Pharmaceutical and Seelos Therapeutics—vying to be a part of it. Fast forward four years and none of these drugs has hit the trial’s primary endpoints. But Clene CEO Rob Etherington said that doesn’t reflect a failure of the study itself. The HEALEY trial was designed to “fast fail” assets that don’t have utility, he explained, “and it worked in that respect, for a few of the regimens.” Indeed, Cudkowicz noted that Biohaven, which ultimately opted against moving forward with the program, also viewed the trial as a success because “they got a quick answer, a clear answer.” Biohaven did not respond to BioSpace ’s request for comment. But perhaps most notably, Clene and Prilenia showed positive data on secondary endpoints that prompted those companies to progress their candidates, Cudkowicz said. “So, I really actually view that as a success.” The First Five To select the initial round of participants in the trial, Cudkowicz and her team decided to host a competition. She noted that many aspiring platform trials seemed to be stuck at the funding stage and believed a call for proposals would speed up the process. The prize: entry into the Healey ALS Platform Trial, where some costs for the first companies would be covered. The team received 30 proposals from all over the world. Cudkowicz and her colleagues assessed each therapy based on clinical potential, mechanism of action, and diversity of approach. “We did want to pick drugs with different targets for the first five,” Cudkowicz said. Finally, they selected five programs. UBC’s zilucoplan inhibits complement component 5, the dysregulation of which is implicated in ALS, while Biohaven’s verdiperstat blocks the enzyme that reduces oxidative stress and neuroinflammation. Clene’s CNM-Au8, by contrast, is an oral, liquid suspension of gold nanocrystals designed to improve the survival, function, and communication of nerve cells. Prilenia’s pridopidine is also a neuroprotective agent, and Implicit Bioscience’s IC14 is an anti-CD14 monoclonal antibody devised to regulate the pathogenic inflammatory responses to infection and injury. Of these, Clene and Prilenia fared the best. Although both companies’ assets missed their primary endpoints, positive biomarker data and benefit to a subgroup of patients in Prilenia’s case supported moving the programs forward. With Prilenia’s candidate, a subgroup of patients with early-stage ALS who were rapidly declining had “substantially less decline” in ALS Functional Rating Scale-Revised (ALSFRS-R) total score compared to placebo. The company has stated plans to launch a Phase III trial. Meanwhile, Clene has a briefing book in front of the FDA based on HEALEY data and additional biomarker results gleaned from the RESCUE-ALS trial and survival data from ongoing compassionate use protocols. While the agency originally said the data were insufficient to support a new drug application, Clene has since submitted more information, including mechanistic and ALS-specific biomarkers and data on a super-responder group, which Etherington said is now under review. Clene also gathered promising survival and biomarker data from its arm of the HEALEY study. Treatment with CNM-Au8 for up to 133 weeks decreased the risk of death by 49% compared to PRO-ACT database–matched placebo, according to Clene, while also reducing levels of neurofilament light chain (NfL), which is gaining ground as a predictor of clinical outcomes in neurodegenerative diseases. “Without the survival secondary endpoint win and the exploratory endpoint win and the neurofilament biomarker win that HEALEY gave Clene, we’d be in a very different position today,” Etherington said. “We certainly wouldn’t have a briefing book under consideration at the FDA without HEALEY.” The other companies have either shut down their programs or are still determining next steps. Implicit never moved forward to the design phase. UCB’s arm of the trial was stopped early after an interim analysis demonstrated futility, according to a Massachusetts General press release . Meanwhile, Biohaven’s verdiperstat failed to statistically differentiate from placebo, and the drug is no longer listed in the company’s pipeline . Seelos joined after Implicit departed and got mixed results. The company stated in a March press release that while its drug trehalose failed to meet statistical significance in the primary and secondary endpoint in the HEALEY study, it showed a “potential signal of efficacy” in a subgroup of patients not taking Amylyx’s Relyvrio, which was given to some trial participants after its approval in September 2022 . And the HEALEY trial marches on. In July 2022, Calico Life Sciences signed on to test ABBV-CLS-7262, and Denali Therapeutics announced its participation with DNL343 in December of the same year. Data from these arms of the trial are expected in early 2025. Another company, Australian outfit PharmAust, recently committed to studying its drug monepantel, and Cudkowicz said talks are ongoing with several more companies. Cudkowicz said that no matter what the result of the various arms, “you can learn a lot about [ALS].” Patient- and Business-Centric Platform trials are a fairly new phenomenon, and Cudkowicz shared that she learned a lot from the DIAN Alzheimer’s trial at Washington University. The model offers myriad benefits, including efficiency. While a clinical trial typically takes more than a year to get off the ground, the HEALEY study is a perpetual trial , meaning a new drug regimen can be added in just 30 days. It’s also patient-centric. The use of a shared placebo between all regimens means that more patients receive a potentially efficacious drug. This also means companies have to recruit fewer patients. The fact that some of their costs were covered was a significant benefit for the smaller companies in the HEALEY trial. Prilenia, for example, had six employees at the time it applied to join the trial. “The good news was they paid for the drug, so we didn’t have to raise money for [that],” Hayden told BioSpace . The infrastructure build and support were also partially funded by several foundations, Cudkowicz said. “I’m grateful. Without HEALEY, we would never have done this trial,” Hayden said. “We didn’t have the money.” The companies involved also have access to elite experience in the ALS space. Cudkowicz is widely considered to be the dean of ALS drug development, and both Etherington and Hayden referenced the gravitas associated with the HEALEY trial, which includes 75 research sites. “The advantage of HEALEY is undeniably the fact that you have an easy network of top expert centers . . . so you’re really able to access the top centers in the United States of America,” Etherington told BioSpace . “That is a tremendous advantage for young startup companies to have access to and to be involved with.” Etherington added that acceptance into the trial gave CNM-Au8 credibility “because it really was a testament of others’ stamp of approval that are well respected in the space.” Growing Pains Still, a complex structure with so many moving parts is bound to take time to perfect. Etherington noted that every drug is unique, and “a platform study does not enable an individual drug and its unique aspects of mechanism or side effects or administration to actually make any changes” to the trial’s protocol. For example, each cohort in the HEALEY trial has run for six months. For CNM-Au8, this was too short of a window to prove efficacy, Etherington said. “To have everybody come in and have to fit the framework with no exceptions, that’s a challenge.” Notably, Cudkowicz shared that the team is currently considering amending the protocol based on learnings from the first five regimens. “This includes possibly longer duration of follow up on the randomized controlled trial period to address the possibility that some medications need more time to achieve biological and clinical effects,” she said. Cudkowicz added that a voice app developed by Aural Analytics and used in the first four regimens is “turning out to be a very good digital biomarker. We also did home spirometry (a measure of lung function), which looks pretty much just as good as in clinic.” Another challenge for a trial with multiple centers across the U.S., Etherington said, was an initial delay in receiving data from every center, though Clene is now in possession of the final datasets from the HEALEY trial. Because every regimen starts at a different time, they all finish at different times, slowing down the rate at which results are available. For a company conducting a single study, “all hands are on deck,” Etherington said. By contrast, in a platform trial, “we have to be more ecumenical. We have to share, as it were, the load and the timeline, and we need to recognize that the company’s individual interests need to be subjugated, to some degree, to the whole of the platform study.” Hayden agreed, saying that the HEALEY team was sometimes “overwhelmed” with running what amounts to eight trials, and that there sometimes is “a little frustration in terms of the speed of response, the ability to get statistical analysis done. All of that can be slower than we would like.” Cudkowicz acknowledged that the trial has faced challenges in this regard. “Since the idea of platform trials is efficiency and speed, we want all parts of it to be efficient,” she said. “Some of the structural, operational things that slow down trials in general are still in play in platform trials [and] how trials are started up at academic institutions is still too slow, in my opinion. We have some new initiatives to improve this dramatically for the next several new regimens,” she said in an email. Cudkowicz added that interest in the trial overwhelmed the capacity of some of the trial sites. “There’s high interest in it from the companies, from the patients, but now we’ve gotta do it even faster.” A heterogeneous patient population was another challenge, Hayden said. “The study included essentially everybody with ALS, and that was maybe . . . both positive and negative. The positive was that this gave hope to everybody.” However, ALS has a “very heterogeneous population.” The HEALEY trial included patients with definite, probable and possible ALS, as well as those more than three years out from diagnosis. “We were concerned about that right in the beginning because we knew that all the trials that had been successful in ALS had a different population” that included patients with only definite and probable ALS, and a shorter disease duration, Hayden said. Prilenia ultimately prespecified that it wanted to also study pridopidine in patients with early-stage ALS, diagnosed less than 18 months before the trial’s start, and HEALEY investigators were able to recruit from such a population for the arm of the trial testing the drug. And while pridopidine failed the primary endpoint in the full analysis set, in patients with early ALS, “what we saw is that we had a very wonderful positive effect,” Hayden said. “We delayed progression for the first time ever seen in an ALS trial at eight weeks.”

Acquired exclusive license for oral, brain-penetrant, dual TYK2/JAK1 inhibitor for immune-mediated brain disorders in March 2023 covering global rights (excluding China) Appointed Nick Kozauer, M.D. as SVP of Clinical Development and Regulatory Strategy following his tenure as Director of the Division of Neurology 2 in the Office of New Drugs of the U.S. Food and Drug Administration Taldefgrobep alfa granted Fast Track Designation in SMA Driving strong and consistent progress across six robust drug development platforms in 2023: in Kv7 activation, targeting Phase 2/3 study start in focal epilepsy and bipolar disorder in the second half of 2023; Phase 1 study initiation planned with potentially first-in-class TYK2/JAK1 brain-penetrant inhibitor in immune-mediated brain disorders; three Phase 3 studies gaining momentum with regulatory engagement and enrollment completion expected in programs spanning glutamate modulation in SCA and OCD, respectively, and enrollment ongoing in myostatin inhibition program in SMA; three IND submissions anticipated in 2023 spanning Kv7 activation, TRPM3 antagonism, and IgG degradation offer potential to drive long-term value creation beyond 2023 NEW HAVEN, Conn., May 12, 2023 /PRNewswire/ -- Biohaven Ltd. (NYSE: BHVN) ("Biohaven" or the "Company"), a global clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of life-changing therapies for people with debilitating neurological and neuropsychiatric diseases, including ultra-rare disorders, today reported financial results for the first quarter ended March 31, 2023, and provided a review of recent accomplishments and anticipated upcoming milestones. Vlad Coric, M.D., Chairman and Chief Executive Officer of Biohaven, commented, "We have set forth ambitious goals for the balance of 2023, as we continue advancing what we believe is one of the most innovative and exciting neuroscience platform in development. We were energized by the preliminary safety, tolerability and pharmacokinetic data we reported from our SAD/MAD Phase 1 study of Kv7.2/Kv7.3 activator BHV-7000 earlier in the quarter, supported by the preponderance of preclinical efficacy data generated to date with BHV-7000 demonstrating potent anti-seizure efficacy, concentration-dependent hyperpolarization of the resting membrane potential, meaningful increases in action potential threshold, and encouragingly low GABAA activity. We also look forward to unlocking Kv7 platform expansion opportunities across a spectrum of indications with high unmet medical need." Dr Coric continued, "With our glutamate platform, we look to gain clarity on the regulatory path forward for SCA and expect to complete enrollment in our Phase 3 OCD program in 2023, which we believe are two critically important milestones given the dearth of approved therapies for these distinct patient populations. Regarding our myostatin platform, we continue to activate sites and drive enrollment in our ongoing Phase 3 SMA study. With our recently acquired TYK2/JAK1 platform, we remain focused on starting Phase 1 studies in 2023 and exploring BHV-8000's potential to address an array of immune-mediated brain disorders. And finally, we look forward to submitting IND applications across three separate programs, including BHV-7010, a next-gen Kv7 activator targeting epilepsy and mood disorders, BHV-2011, a TRPM3 inhibitor targeting chronic pain, and BHV-1300, our IgG degrader, following the report of robust preclinical data earlier in the year. We remain as committed as ever to driving data-driven, efficient results for patients and the shareholders who support our continued work, and look forward to executing and delivering continued value in the year ahead." First Quarter 2023 and Recent Business Highlights Acquired BHV-8000, a brain-penetrant inhibitor of TYK2/JAK1, from Highlightll - As previously reported, in March 2023, the Company acquired exclusive rights (excluding China) to a novel, oral, first-in-class, brain-penetrant, dual inhibitor of TYK2/JAK1 offering wide therapeutic index with TYK2 inhibition and high selectivity for JAK1 inhibition without the severely limiting adverse class effects of JAK2/JAK3 inhibitors. The Company expects to commence Phase 1 development in 2023, exploring its potential to address immune-mediated brain disorders. Delivered oral and poster presentations demonstrating preclinical efficacy and initial Phase 1 safety and tolerability of BHV-7000 on ASENT 2023 virtual platform - In March 2023, the Company presented data at the 2023 American Society for Experimental Neurotherapeutics Annual Meeting (ASENT 2023) demonstrating that activating Kv7.2/7.3 with BHV-7000, a structurally and pharmacologically distinct compound from ezogabine, produced concentration-dependent hyperpolarization of the resting membrane potential, potentiated meaningful increases in action potential threshold, exhibited significantly lower GABAA activity than ezogabine, and delivered potent anti-seizure efficacy in the maximal electroshock seizure (MES) model, without negatively impacting neurobehavior. The Company also presented data from the Phase 1 SAD/MAD clinical trial, demonstrating BHV-7000 was well-tolerated at single doses up to 100 mg and multiple doses up to 40 mg per day for 15 days. Most adverse events were mild and resolved spontaneously, and there were no serious or severe adverse events or dose-limiting toxicities reported. In BHV-7000 treated subjects in the MAD study, CNS-related adverse events typically associated with other anti-seizure medications were not reported. Taldefgrobep Alfa Granted Fast Track Designation and Orphan Drug Designation - As previously reported, Taldefgrobep alfa, an anti-myostatin adnectin in development for SMA, was granted Fast Track designation and Orphan Drug designation by the U.S. Food and Drug Administration (FDA) in February 2023, and December 2022, respectively. Phase 3 studies are ongoing; the Company expects to enroll approximately 180 patients in this randomized, double-blind, placebo-controlled global trial. Reported preclinical data with extracellular target degrader platform technology (MoDE™), a pan-IgG degrader - As previously reported, in January 2023, the Company evaluated the effect of a single dose of IgG degrader, BHV-1300, in cynomolgus monkeys. The Company reported 75% reduction of IgG levels from baseline and noted the observation occurred in three days; the data in this pre-clinical study compares favorably to standard of care therapy efgartigimod, where reduction of IgG levels with efgartigimod was observed to be 50% and had taken 5-7 days. The Company expects BHV-1300 will be ready for IND submission in the second half of 2023. Reported preclinical data with second MoDE in bispecific platform targeting IgA Nephropathy - As previously reported, in January 2023, the Company reported preclinical data with a second MoDE targeting galactose deficient IgA (Gd-IgA), which is believed to play a pathogenic role in IgA Nephropathy. Specific removal of pathogenic Gd-IgA with preservation of normal IgA potentially permits disease remission without incurring an infection risk. The Company shared preliminary data demonstrating the chimeric antibody-ASGPR ligand conjugate specifically mediated endocytosis of Gd-IgA, as opposed to normal IgA, in an endocytosis assay with HepG2 cells. Leadership team expanded with key appointment - In April 2023, Biohaven announced the appointment of Nick Kozauer, M.D. as SVP of Clinical Development and Regulatory Strategy following his tenure as Director of the Division of Neurology 2 in the Office of New Drugs of the FDA. Dr. Kozauer had supervised and reviewed the approval of over 15 marketed drugs during his tenure at the FDA. Upcoming Expected Milestones: Biohaven is progressing its product candidates through clinical programs in a number of common and rare disorders. The Company expects to reach significant pipeline milestones in the coming periods. Biohaven expects to: Initiate EEG study with BHV-7000 in the first half of 2023: Following Phase 1 study completion, Biohaven expects to initiate pivotal trials in patients with epilepsy and patients with bipolar disorder in the second half of 2023. Submit IND with BHV-7010 in epilepsy and mood disorders: The Company expects to submit an IND with next-generation Kv7 activator BHV-7010 in epilepsy in the second half of 2023. Submit IND with BHV-2100 in chronic pain: The Company expects to submit an IND with BHV-2100, a TRPM3 antagonist in the Company's ion channel platform targeting a pain disorder in the second half of 2023. Submit IND with BHV-1300: The Company expects to submit an IND with pan-IgG degrader BHV-1300 in the second half of 2023. Commence Phase 1 studies with BHV-8000: The Company expects to commence Phase 1 studies with BHV-8000, an oral, brain-penetrant, dual TYK2/JAK1 inhibitor for immune-mediated brain disorders in 2023. Complete enrollment in Phase 3 study of troriluzole in OCD in 2023: Two Phase 3 randomized, double-blind, placebo-controlled studies are expected to enroll up to 700 patients (in each trial) across nearly 200 global study sites. The Company anticipates completing enrollment in year-end 2023. Provide an update on troriluzole in SCA: The Company intends to interact with the FDA and/or European Medicines Agency in the first half of 2023 on next steps. Continue advancing Phase 3 clinical studies of taldefgrobep alfa in SMA: The Company expects to enroll approximately 180 patients in the study through patient enrollment in up to 60 clinical sites. Continue advancements across multiple neuroscience and immunoscience indications: The Company's preclinical pipeline includes molecular degraders of extracellular proteins, CD38 targeting antibody recruiting molecules (ARMs), TRP channels, and other undisclosed targets, including those with disease-modifying potential. Capital Position: Cash, cash equivalents and marketable securities as of March 31, 2023 was $392.0 million, including $4.0 million of restricted cash, and excluding $61.5 million of cash payable to Biohaven Pharmaceutical Holding Company Ltd. (the "Former Parent"), compared to $467.9 million, including $2.5 million of restricted cash, and excluding $35.2 million of cash payable to the Former Parent, as of December 31, 20221. First Quarter 2023 Financial Highlights: Research and Development (R&D) Expenses: R&D expenses, including non-cash share-based compensation costs, were $63.5 million for the three months ended March 31, 2023, compared to $70.1 million for the three months ended March 31, 2022. The decrease of $6.6 million was primarily due to a decrease of $14.7 million in personnel-related costs including non-cash share-based compensation costs, and a decrease in expenses for verdiperstat, BHV-2100 and BHV-1200, partially offset by an increase in expenses for our clinical programs for Kv7 (BHV-7000 and 7010), troriluzole and BHV-2000. The decrease in personnel-related costs is due to non-cash share-based compensation expense for the first quarter of 2022 being allocated from the Former Parent equity plan based on equity awards with higher grant date fair values, which was partially offset by increased personnel costs related to an increase in headcount for our discovery operations. Non-cash share-based compensation expense was $2.2 million for the three months ended March 31, 2023, a decrease of $22.3 million as compared to the same period in 2022. General and Administrative (G&A) Expenses: G&A expenses, including non-cash share-based compensation costs, were $14.3 million for the three months ended March 31, 2023, compared to $19.7 million for the three months ended March 31, 2022. The decrease of $5.4 million was primarily due to decreased non-cash share-based compensation costs. This was partially offset by increased personnel costs in the first quarter of 2023 compared to the same period in 2022, due to a majority of the personnel costs in the first quarter of 2022 being allocated to the Former Parent. Non-cash share-based compensation expense was $1.5 million for the three months ended March 31, 2023, a decrease of $14.1 million as compared to the same period in 2022. The decrease in non-cash share-based compensation expense is due to the first quarter of 2022 expense being allocated from the Former Parent equity plan based on equity awards with higher grant date fair values. Other Income (Expense), Net: Other income (expense), net was a net income of $8.2 million for the three months ended March 31, 2023, compared to net expense of $4.0 thousand for the three months ended March 31, 2022. The increase of $8.2 million was primarily due to an increase in net investment income and an increase of $3.9 million in other income related to our transition services provided to our Former Parent, which is largely non-recurring. Net Loss: Biohaven reported a net loss for the three months ended March 31, 2023, of $70.5 million, or $1.03 per share, compared to $97.0 million, or $2.46 per share, for the same period in 2022. Non-GAAP adjusted net loss for the three months ended March 31, 2023 was $66.7 million, or $0.98 per share, compared to $56.9 million, or $1.45 per share for the same period in 2022. These non-GAAP adjusted net loss and non-GAAP adjusted net loss per share measures, more fully described below under "Non-GAAP Financial Measures," exclude non-cash share-based compensation charges. A reconciliation of the GAAP financial results to non-GAAP financial results is included in the tables below. For periods prior to Biohaven's spin-off from the Former Parent on October 3, 2022 (the "Spin-Off"), net loss per share and non-GAAP adjusted net loss per share were calculated based on the 39,375,944 common shares of Biohaven distributed to the Former Parent shareholders at the time of the distribution, including common shares issued in connection with the Former Parent share options that were settled on October 3, 2022 and common shares issued in connection with the Former Parent restricted share units that vested on October 3, 2022. The same number of shares is being utilized for the calculation of basic and diluted earnings per share for all periods presented prior to the Spin-Off. Non-GAAP Financial Measures This press release includes financial results prepared in accordance with accounting principles generally accepted in the United States (GAAP), and also certain non-GAAP financial measures. In particular, Biohaven has provided non-GAAP adjusted net loss and adjusted net loss per share, which are adjusted to exclude non-cash share-based compensation, which is substantially dependent on changes in the market price of common shares. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. However, Biohaven believes the presentation of non-GAAP adjusted net loss and adjusted net loss per share, when viewed in conjunction with GAAP results, provides investors with a more meaningful understanding of ongoing operating performance and can assist investors in comparing Biohaven's performance between periods. In addition, these non-GAAP financial measures are among those indicators Biohaven uses as a basis for evaluating performance, and planning and forecasting future periods. These non-GAAP financial measures are not intended to be considered in isolation or as a substitute for GAAP financial measures. A reconciliation between these non-GAAP measures and the most directly comparable GAAP measures is provided later in this news release. 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 neurological and neuropsychiatric 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 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 extracellular target degrader platform technology (MoDE™) with potential application in neurological disorders, cancer, and autoimmune diseases. 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 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 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.