• U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation and Rare Pediatric Disease designation for bexicaserin for the treatment of Dravet syndrome

Longboard Pharmaceuticals, Inc. (Nasdaq: LBPH), a clinical-stage biopharmaceutical company focused on developing novel, transformative medicines for neurological diseases, today announced that the FDA has granted Rare Pediatric Disease designation and Orphan Drug designation for its investigational drug bexicaserin for the treatment of Dravet syndrome.

“We are thrilled to receive both Rare Pediatric Disease and Orphan Drug designations for bexicaserin in Dravet syndrome. As the first company to receive Breakthrough Therapy designation (BTD) for the treatment of seizures associated with Developmental and Epileptic Encephalopathies (DEEs), we appreciate the regulatory support that BTD provides as we continue on our journey to study bexicaserin as a potential treatment option for DEE patients with significant unmet medical needs. Receiving Orphan Drug designation in Dravet syndrome provides us with further regulatory support as we progress the development of bexicaserin. Additionally, the granting of the Rare Pediatric Disease designation provides us with an opportunity to receive a Priority Review Voucher which can be used for priority review of another Longboard compound or monetized,” stated Dr. Randall Kaye, Longboard’s Chief Medical Officer. “We appreciate the FDA’s ongoing support and look forward to initiating our global Phase 3 DEEp program, starting with DEEp SEA in Dravet syndrome in the coming weeks.”

About Rare Pediatric Disease Designation and Priority Review Vouchers (PRVs)

The Rare Pediatric Disease program aims to incentivize drug development for rare pediatric diseases. Under this program, a sponsor who receives an approval for a drug or biological product for a rare pediatric disease may qualify for a Priority Review Voucher (PRV) that can be redeemed to receive priority review for a different product. The sponsor may also transfer or sell the PRV to another sponsor.

About Orphan Drug Designation

Supporting the development and evaluation of new treatments for rare diseases is a key priority for the FDA. The FDA has authority to grant Orphan Drug designation to a drug or biological product to prevent, diagnose or treat a rare disease or condition. Orphan Drug designation qualifies sponsors for incentives including tax credits for qualified clinical trials, exemption from user fees, and the potential for seven years of market exclusivity after approval.

ABOUT LONGBOARD PHARMACEUTICALS

Longboard Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on developing novel, transformative medicines for neurological diseases. Longboard is working to advance a portfolio of centrally acting product candidates designed to be highly selective for specific G protein-coupled receptors (GPCRs). Longboard’s small molecule product candidates are based on more than 20 years of GPCR research. Longboard plans to advance bexicaserin (LP352), an oral, centrally acting 5-hydroxytryptamine 2C (5-HT2C) receptor superagonist, with no observed impact on 5-HT2B and 5-HT2A receptor subtypes, into a global Phase 3 program. Earlier this year, Longboard reported positive topline data from a Phase 1b/2a clinical trial (the PACIFIC Study) evaluating bexicaserin in participants ages 12 to 65 years old with Developmental and Epileptic Encephalopathies (DEEs), including Lennox-Gastaut syndrome, Dravet syndrome and other DEEs. Longboard is also evaluating LP659, an oral, centrally acting, sphingosine-1-phosphate (S1P) receptor subtypes 1 and 5 modulator, which is in development for the potential treatment of rare neuroinflammatory conditions. Longboard recently completed a Phase 1 single-ascending dose (SAD) clinical trial for LP659 in healthy volunteers.

Bexicaserin and LP659 are investigational compounds that are not approved for marketing by the FDA or any other regulatory authority.

FORWARD-LOOKING STATEMENTS

Certain statements in this press release are forward-looking statements that involve a number of risks and uncertainties. In some cases, you can identify forward-looking statements by words such as “focus”, “potential”, “opportunity”, “can”, “look forward”, “aim”, “may”, “working to”, “designed to”, “plan”, or the negative, plural or other tenses of these words, references to future dates or time periods, or other comparable language, and they may include, without limitation, statements about the following: Longboard’s product candidates and programs (including bexicaserin’s potential, development, advancement into a global Phase 3 program, and Breakthrough Therapy, Rare Pediatric Disease and Orphan Drug designations and their potential benefits, including eligibility for a Priority Review Voucher (PRV)), plans, focus and work. For such statements, Longboard claims the protection of the Private Securities Litigation Reform Act of 1995. Actual events or results may differ materially from Longboard’s expectations. Factors that could cause actual results to differ materially from those stated or implied by Longboard’s forward-looking statements include, but are not limited to, the following: the rare pediatric disease PRV program will begin to sunset after September 30, 2024, and if the program is not reauthorized, we will only be eligible to receive a PRV if bexicaserin receives marketing approval by September 30, 2026; the standards for Breakthrough Therapy, Rare Pediatric Disease and Orphan Drug designations are not the same as the standard for drug approval, the clinical evidence supporting Breakthrough Therapy designation is preliminary, and not all drugs designated as Breakthrough Therapies ultimately will be shown to have substantial improvement over available therapies; the FDA may later decide to rescind a designation if it determines the designation is no longer supported by subsequent data; Longboard’s product candidates are in a lengthy research and development process, the timing, manner and outcome of research, development and regulatory review is uncertain, and Longboard’s product candidates, including bexicaserin and LP659, may not advance in research or development or be approved for marketing; results of clinical trials and other studies are subject to different interpretations and may not be predictive of future results; topline or interim data may not accurately reflect the complete results of a particular study or trial and remain subject to audit, and final data may differ materially from topline or interim data; enrolling participants in clinical trials is competitive and challenging; risks related to unexpected or unfavorable new data; nonclinical and clinical data is voluminous and detailed, and regulatory agencies may interpret or weigh the importance of data differently and reach different conclusions than Longboard or others, request additional information, have additional recommendations or change their guidance or requirements before or after approval; risks related to Longboard’s limited operating history, financial position and need for additional capital; Longboard will need additional managerial and financial resources to advance all of its programs, and you and others may not agree with the manner Longboard allocates its resources; risks related to the development and commercialization of Longboard’s product candidates; risks related to relying on licenses or collaborative arrangements; other risks related to Longboard’s dependence on third parties; competition; product liability or other litigation or disagreements with others; government and third-party payor actions, including relating to reimbursement and pricing; risks related to regulatory compliance; and risks related to Longboard’s and third parties’ intellectual property rights. Additional factors that could cause actual results to differ materially from those stated or implied by Longboard’s forward-looking statements are disclosed in Longboard’s filings with the Securities and Exchange Commission (SEC). These forward-looking statements represent Longboard’s judgment as of the time of this release. Longboard disclaims any intent or obligation to update these forward-looking statements, other than as may be required under applicable law.

CORPORATE CONTACT: Megan E. Knight VP, Head of Investor Relations IR@longboardpharma.com 858.789.9283

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