[Translation] A multicenter, randomized, double-blind, parallel, placebo-controlled clinical trial on the efficacy and safety of dexmethylphenidate hydrochloride extended-release capsules in the treatment of attention deficit hyperactivity disorder

采用多中心、随机、双盲、安慰剂平行对照的方法，评估盐酸右哌甲酯缓释胶囊用于治疗6~17周岁患者注意缺陷多动障碍的有效性和安全性。

[Translation]

A multicenter, randomized, double-blind, placebo-controlled, parallel-controlled method was used to evaluate the efficacy and safety of dexmethylphenidate hydrochloride extended-release capsules in the treatment of attention deficit hyperactivity disorder in patients aged 6 to 17 years.

Start Date21 Sep 2024

Sponsor / Collaborator

Yichang Humanwell Pharmaceuticals Co., Ltd.

CTR20233755 / CompletedNot Applicable

盐酸右哌甲酯缓释胶囊人体生物等效性研究

[Translation] Study on the bioequivalence of dexmethylphenidate hydrochloride sustained-release capsules in healthy volunteers

主要研究目的：研究空腹/餐后/拌食状态下单次口服受试制剂盐酸右哌甲酯缓释胶囊（规格：20mg，宜昌人福药业有限责任公司生产）与参比制剂盐酸右哌甲酯缓释胶囊（商品名：FocalinXR，规格：20mg；Novartis Pharmaceuticals Corporation持证）在健康受试者体内的药代动力学，评价空腹/餐后/拌食状态口服两种制剂的生物等效性。次要研究目的：研究受试制剂盐酸右哌甲酯缓释胶囊20mg和参比制剂FocalinXR20mg在健康受试者中的安全性。

[Translation]

The main purpose of the study is to study the pharmacokinetics of the test preparation dexmethylphenidate hydrochloride sustained-release capsules (specification: 20 mg, produced by Yichang Renfu Pharmaceutical Co., Ltd.) and the reference preparation dexmethylphenidate hydrochloride sustained-release capsules (trade name: FocalinXR, specification: 20 mg; licensed by Novartis Pharmaceuticals Corporation) in healthy subjects after a single oral administration in the fasting/postprandial/mixed state, and to evaluate the bioequivalence of the two preparations in the fasting/postprandial/mixed state. Secondary purpose of the study: to study the safety of the test preparation dexmethylphenidate hydrochloride sustained-release capsules 20 mg and the reference preparation FocalinXR 20 mg in healthy subjects.

Start Date17 Dec 2023

Sponsor / Collaborator

Yichang Humanwell Pharmaceuticals Co., Ltd.

CTR20232453 / CompletedNot Applicable

盐酸右哌甲酯缓释胶囊人体生物等效性研究（预试验）

[Translation] Bioequivalence study of dexmethylphenidate hydrochloride sustained-release capsules in humans (preliminary study)

主要研究目的：研究空腹/餐后/拌食状态下单次口服受试制剂盐酸右哌甲酯缓释胶囊（规格：20mg，宜昌人福药业有限责任公司生产）与参比制剂盐酸右哌甲酯缓释胶囊（商品名：FocalinTMXR，规格：20mg；Novartis Pharmaceuticals Corporation持证）在健康受试者体内的药代动力学，评价空腹/餐后/拌食状态口服两种制剂的生物等效性，为正式试验设计提供参考依据。次要研究目的：研究受试制剂盐酸右哌甲酯缓释胶20mg和参比制剂FocalinTMXR 20mg在健康受试者中的安全性。

[Translation]

The main purpose of the study is to study the pharmacokinetics of the test preparation dexmethylphenidate hydrochloride sustained-release capsules (specification: 20 mg, produced by Yichang Renfu Pharmaceutical Co., Ltd.) and the reference preparation dexmethylphenidate hydrochloride sustained-release capsules (trade name: FocalinTMXR, specification: 20 mg; licensed by Novartis Pharmaceuticals Corporation) in healthy subjects after a single oral administration under fasting/postprandial/mixed food conditions, and to evaluate the bioequivalence of the two preparations taken orally under fasting/postprandial/mixed food conditions, and to provide a reference for the design of the formal trial. Secondary purpose of the study: to study the safety of the test preparation dexmethylphenidate hydrochloride sustained-release gel 20 mg and the reference preparation FocalinTMXR 20 mg in healthy subjects.

Start Date28 Aug 2023

Sponsor / Collaborator

Yichang Humanwell Pharmaceuticals Co., Ltd.

100 Clinical Results associated with Dexmethylphenidate Hydrochloride

100 Translational Medicine associated with Dexmethylphenidate Hydrochloride

100 Patents (Medical) associated with Dexmethylphenidate Hydrochloride

290

Literatures (Medical) associated with Dexmethylphenidate Hydrochloride

15 Jul 2023·BMC chemistry

Chromatographic reversed HPLC and TLC-densitometry methods for simultaneous determination of serdexmethylphenidate and dexmethylphenidate in presence of their degradation products-with computational assessment.

Article

Author: Kelani, Khadiga M ; Talaat, Wael ; Elsonbaty, Ahmed ; Nassar, Ahmed M W ; Morshedy, Samir ; Omran, Gamal A

Two Chromatographic methods have been established and optimized for simultaneous determination of serdexmethylphenidate (SER.DMP) and dexmethylphenidate (DMP) in the presence of their degradation products. The first method is a reversed phase high performance liquid chromatography with diode array detection (HPLC-DAD). Isocratic separation was carried out on Waters X-bridge Shield RP₁₈ column (150×3.9×5 μm particle size) using a mixture of 5 mM phosphate buffer (pH 5.5): acetonitrile (40:60, v/v) as a mobile phase, flow rate 1 mL/min and detection at 220 nm. The second method is a thin-layer chromatography (TLC)-densitometry method using methanol: chloroform (70:30, v/v) as a mobile phase and UV scanning at 220 nm. In HPLC method, the linearity range of SER.DMP was (2.5-25 μg/mL); with LOD (0.051 μg/mL) and LOQ (0.165 μg/mL) while for DMP was (2.5-25 μg/mL); with LOD and LOQ of (0.098 μg/mL) and (0.186 μg/mL), respectively. For TLC method the sensitivity range of SER.DMP was (5-25 μg/mL), LOD was (0.184 μg/spot), while LOQ was (0.202 μg/ spot) whereas for DMP the sensitivity range was (5-25 μg/mL) with LOD of (0.115 μg/ spot) and LOQ of (0.237 μg/ spot), respectively. SER.DMP was found to be equally labile to acidic and alkaline hydrolysis, whereas DMP was sensitive to acidic hydrolysis only. Both drugs were successfully determined in presence of acidic and basic degradants by the two developed methods (stability indicating assay method). Chromatographic separation of the degradation products was carried out on TLC aluminum silica plates 60 F254, as a stationary phase, using methanol: dichloroethane: acetonitrile (60:20:20 v/v), as a mobile phase. The degradation pathway was confirmed using TLC, IR, ¹H-NMR and mass spectroscopy; moreover, the separation power was correlated to the computational results by applying molecular dynamic simulation. The developed methods were validated according to the International Conference on Harmonization (ICH) guidelines demonstrating good accuracy and precision. They were successfully applied for quantitation of SER.DMP and DMP in pure and capsule forms. The results were statistically compared with those obtained by the reported method in terms of accuracy, precision and robustness, and no significant difference was found.

01 Jul 2023·Journal of clinical psychopharmacology

Risk of Major Malformations in Infants After First-Trimester Exposure to Stimulants

Article

Author: Viguera, Adele C ; Rossa, Ella T ; Gaccione, Peter ; Cohen, Lee S ; Chitayat, David ; Freeman, Marlene P ; Szpunar, Mercedes J ; Kobylski, Lauren A

Abstract:

Purpose/Background:

The prevalence of attention-deficit/hyperactivity disorder in adult females is 3% to 4%. Attention-deficit/hyperactivity disorder is highly comorbid with other psychiatric disorders such as mood, anxiety, and substance use disorders. For reproductive-aged women, the treatment of attention-deficit/hyperactivity disorder with stimulant medications may be considered during pregnancy or breastfeeding, although historically, data are lacking to inform these decisions. The aim of this investigation was to determine the risk of major malformations in infants after first-trimester prescription stimulant exposure in a small but rigorously characterized sample.

Methods/Procedures:

The Massachusetts General Hospital National Pregnancy Registry for Psychiatric Medications systematically ascertains information from pregnant females including demographic information, medical and psychiatric history, use of prescription medications, and other information relevant to fetal outcomes. Participants provide verbal informed consent and are interviewed twice during gestation and again at approximately 3 months postpartum. The primary outcome of interest is the presence of a major malformation identified within 6 months after birth. Redacted cases of major malformations are reviewed by a dysmorphologist blinded to medication exposure.

Findings/Results:

A total of N = 1988 women were eligible for this analysis, including the following exposures: n = 173 to mixed amphetamine salts; n = 40 to lisdexamfetamine; n = 45 to methylphenidate; n = 3 to dexmethylphenidate; and n = 1755 controls. The odds ratio of a major malformation among infants after first-trimester exposure to any stimulant was 0.39 (95% confidence interval, 0.09–1.61) compared with controls. There were no major malformations observed in infants exposed to lisdexamfetamine, methylphenidate, or dexmethylphenidate.

Implications/Conclusions:

Although preliminary, this analysis from an ongoing pregnancy registry provides reassurance that these stimulants do not appear to have major teratogenic effects.

Trial Registration:

ClinicalTrials.gov identifier: NCT01246765.

01 May 2023·Journal of child and adolescent psychopharmacology

Analysis of Growth Velocity in Children with Attention-Deficit/Hyperactivity Disorder Treated for up to 12 Months with Serdexmethylphenidate/Dexmethylphenidate.

Article

Author: Oh, Charles ; Cutler, Andrew J ; Childress, Ann C ; Patel, Maitrey

Objective: Serdexmethylphenidate/dexmethylphenidate (SDX/d-MPH) is approved for the treatment of patients aged ≥6 years with attention-deficit/hyperactivity disorder (ADHD). A 12-month, open-label safety study with SDX/d-MPH in children with ADHD showed that SDX/d-MPH was well tolerated and comparable with other methylphenidate products. In this post hoc analysis of the 12-month study, the objective was to characterize the effect of SDX/d-MPH on growth in children with ADHD over 12 months. Methods: This was a post hoc analysis of a dose-optimized, open-label, phase 3 safety study of SDX/d-MPH in children aged 6-12 years with ADHD (NCT03460652). Weight and height Z-score analyses were conducted. Z-score change from baseline was calculated based on the baseline values for the subjects remaining in the study at the observation time point. Results: Subjects (N = 238) from the treatment-phase safety population included all enrolled subjects who received ≥1 dose of study drug and had ≥1 postdose safety assessment. During treatment, the mean weight and height Z-scores decreased over time from their respective baselines. At the 12-month time point, mean (standard deviation [SD]) Z-score changes from baseline for weight and height for the subjects remaining in the study were -0.20 (0.50) and -0.21 (0.39), respectively; however, these mean changes in Z-scores were not clinically significant (change <0.5 SD). Long-term treatment with SDX/d-MPH was associated with modest reductions in expected weight and lower-than-expected increases in height: effects that plateaued or diminished later in treatment. Conclusion: The overall effects of SDX/d-MPH on growth velocity (the change in weight and height from one time point to the next) were minimal, and the range of changes was not considered clinically significant. ClinicalTrials.gov identifier: NCT03460652.

News (Medical) associated with Dexmethylphenidate Hydrochloride

07 Nov 2024

·www.globenewswire.com

Cingulate Reports Third Quarter 2024 Financial Results Reflecting $19.5 Million Increase in Working Capital to Advance ADHD Drug to Market

On Target for Mid-2025 New Drug Application (NDA) Submission of lead ADHD asset CTx-1301KANSAS CITY, Kan., Nov. 07, 2024 (GLOBE NEWSWIRE) -- Cingulate Inc. (NASDAQ: CING), a biopharmaceutical company utilizing its proprietary Precision Timed Release™ (PTR™) drug delivery platform technology to build and advance a pipeline of next-generation pharmaceutical products, today announced its financial results for the three months ended September 30, 2024, and provided a clinical and business update. “The capital raised this quarter has allowed us to focus on advancing the remaining activities required for NDA submission of our lead ADHD asset, CTx-1301, targeted for mid 2025,” said Cingulate Chairman and CEO Shane J. Schaffer. “The granting of European patents for CTx-1301 in up to 30 territories, including the United Kingdom, helps Cingulate expand its reach beyond the United States and makes a positive impact on the growing ADHD markets abroad. Overall, Cingulate finds itself in a stronger position financially and looks forward to filing its first NDA next year.” Cingulate Initiates Final Study for Lead ADHD Asset CTx-1301 In September, Cingulate commenced its final FDA-required study, which is a food effect study, for CTx-1301. A data readout from the study is expected by the end of 2024. European Patent Granted for Lead Asset CTx-1301 for the Treatment of ADHD Cingulate was issued a European patent for its lead asset CTx-1301 for the treatment of ADHD during the third quarter of 2024. This patent will include up to 30 European territories, including the United Kingdom. In addition to the European patent, Cingulate has patents in Australia, Canada and Israel, as well as pending patents in Hong Kong, the Republic of Korea, and the United States. Nasdaq Listing Update On September 9, 2024, the Nasdaq Hearings Panel notified Cingulate that it had regained compliance with the $1.00 minimum bid price requirement set forth in Nasdaq Listing Rule 5550(a)(2). Over $12 Million of Capital Raised in the Third Quarter Cingulate raised approximately $12.5 million of capital in the third quarter of 2024. The capital raised provides the Company the cash runway to fund the clinical, manufacturing, and regulatory activities, as well as operating activities, into the third quarter of 2025, based on planned expenditures. Cingulate is targeting mid-2025 for the NDA submission of CTx-1301 (dexmethylphenidate) for the treatment of Attention Deficit Hyperactivity Disorder (ADHD). Third Quarter Results Cash Position: As of September 30, 2024, Cingulate had approximately $10 million in cash and cash equivalents, a significant increase from December 31, 2023, providing the Company with an extended cash runway into the third quarter of 2025, as noted above. Working Capital: As of September 30, 2024, Cingulate had approximately $9.8 million in working capital, an increase of $19.5 million from December 31, 2023. This increase in working capital is reflective of a significant strengthening of the Company’s balance sheet resulting from capital raised in 2024. Liabilities: As of September 30, 2024, total liabilities were $1.5 million, a decrease from December 31, 2023 of $8.8 million, including the conversion of the related party note payable in the amount of $3.3 million which occurred in the first quarter of 2024. Stockholders’ Equity: As of September 30, 2024, total stockholders’ equity was $12.0 million, an increase of $18.9 million from the end of 2023. R&D Expenses: R&D expenses were $1.4 million for the three months ended September 30, 2024, a decrease of $2.5 million from the three months ended September 30, 2023. This change was primarily the result of decreased clinical activity in the three months ended September 30, 2024 as compared to the same period in 2023. During the third quarter of 2023, we incurred significant costs relating to two Phase 3 studies for CTx-1301, the fixed dose pediatric and adolescent safety and efficacy study and the pediatric dose optimization and duration study. Enrollment in these two studies was completed in early 2024 and we are progressing with the remaining close-out and analytical activities required for an NDA submission. Manufacturing costs also decreased as the activity in 2023 was more significant for the manufacture of clinical supply for the Phase 3 studies. In 2024, manufacturing activity included the completion of registration batches of CTx-1301. G&A Expenses: Total G&A expenses were $1.9 million for the three months ended September 30, 2024, which was relatively consistent to the three months ended September 30, 2023. Net Loss: Net loss was $3.2 million for the three months ended September 30, 2024, compared to $6.0 million for the same period in 2023. The decrease in the net loss primarily related to a decrease in R&D and G&A expenses described above. Cingulate Inc. Consolidated Balance Sheet Data September 30, December 31, 2024 2023 Cash and cash equivalents $10,040,149 $52,416 Total assets $13,580,104 $3,491,436 Working Capital $9,801,070 $(9,647,172) Total liabilities $1,542,541 $10,360,865 Accumulated deficit $(102,357,201) $(92,943,443) Total stockholders' equity $12,037,563 $(6,869,429) Cingulate Inc. Consolidated Statements of Operations Three Months Ended September 30, Six Months Ended September 30, 2024 2023 2024 2023 Operating expenses: Research and development $1,428,504 $3,923,852 $5,116,582 $10,508,395 General and administrative 1,853,583 1,825,822 4,319,902 5,453,643 Operating loss (3,282,087) (5,749,674) (9,436,484) (15,962,038) Interest and other income (expense), net 50,483 (229,380) 22,726 (638,212) Loss before income taxes (3,231,604) (5,979,054) (9,413,758) (16,600,250) Income tax benefit (expense) - - - - Net loss (3,231,604) (5,979,054) (9,413,758) (16,600,250) About Cingulate® Cingulate Inc. is a biopharmaceutical company utilizing its proprietary Precision Timed Release™ (PTR™) drug delivery platform technology to build and advance a pipeline of next-generation pharmaceutical products, designed to improve the lives of patients suffering from frequently diagnosed conditions characterized by burdensome daily dosing regimens and suboptimal treatment outcomes. With an initial focus on the treatment of Attention Deficit/Hyperactivity Disorder (ADHD), Cingulate is identifying and evaluating additional therapeutic areas where its PTR technology may be employed to develop future product candidates, such as anxiety disorders. Cingulate is headquartered in Kansas City, KS. For more information visit Cingulate.com. Forward-Looking Statements This press release contains “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These forward-looking statements include all statements, other than statements of historical fact, regarding our current views and assumptions with respect to future events regarding our business, including statements with respect to our plans, assumptions, expectations, beliefs and objectives with respect to product development, clinical studies, clinical and regulatory timelines, market opportunity, competitive position, business strategies, potential growth opportunities and other statements that are predictive in nature. These statements are generally identified by the use of such words as “may,” “could,” “should,” “would,” “believe,” “anticipate,” “forecast,” “estimate,” “expect,” “intend,” “plan,” “continue,” “outlook,” “will,” “potential” and similar statements of a future or forward-looking nature. Readers are cautioned that any forward-looking information provided by us or on our behalf is not a guarantee of future performance. Actual results may differ materially from those contained in these forward-looking statements as a result of various factors disclosed in our filings with the Securities and Exchange Commission (SEC), including the “Risk Factors” section of our Annual Report on Form 10-K filed with the SEC on April 1, 2024 and our other filings with the SEC. All forward-looking statements speak only as of the date on which they are made, and we undertake no duty to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except to the extent required by law. Investor & Public Relations: Thomas Dalton Vice President, Investor & Public Relations, Cingulate tdalton@cingulate.com (913) 942-2301 Matt Kreps Darrow Associates mkreps@darrowir.com (214) 597-8200

Financial StatementNDA

08 Oct 2024

·www.globenewswire.com

Cingulate’s Shane Schaffer Joins Cast of Nationally Syndicated Big Biz Show

KANSAS CITY, Kan., Oct. 08, 2024 (GLOBE NEWSWIRE) -- Shane J. Schaffer, Chairman and CEO of Cingulate Inc. (NASDAQ: CING), a biopharmaceutical company utilizing its proprietary Precision Timed Release™ (PTR™) drug delivery platform technology to build and advance a pipeline of next-generation pharmaceutical products, recently joined the cast of the Big Biz Show, a business and finance broadcast, to discuss Cingulate’s lead Phase 3 asset CTx-1301 for the treatment of ADHD, its plans for commercialization, and other therapeutic areas where the company’s PTR™ technology may be applied. The Big Biz Show is nationally syndicated and can be seen and heard around the globe across a multitude of broadcast partners including iHeart Radio and the American Forces Network. Dr. Schaffer’s interview with Big Biz host Bob “Sully” Sullivan and his cast may be viewed here. For more information about the Big Biz Show visit bigbizshow.com. About Cingulate®Cingulate Inc. (NASDAQ: CING), is a clinical-stage biopharmaceutical company utilizing its proprietary Precision Timed Release™ (PTR™) drug delivery platform technology to build and advance a pipeline of next-generation pharmaceutical products, designed to improve the lives of patients suffering from frequently diagnosed conditions characterized by burdensome daily dosing regimens and suboptimal treatment outcomes. With an initial focus on the treatment of Attention Deficit/Hyperactivity Disorder (ADHD), Cingulate is identifying and evaluating additional therapeutic areas where PTR technology may be employed to develop future product candidates, including to treat anxiety disorders.Cingulate is headquartered in Kansas City. For more informationvisit cingulate.com. Investor RelationsThomas DaltonVice President, Investor & Public Relations, CingulateTDalton@cingulate.com913-942-2301 Matt KrepsDarrow Associatesmkreps@darrowir.com214-597-8200

Phase 3Executive Change

24 Sep 2024

·www.globenewswire.com

Zevra Therapeutics to Present Top-Line Data from the Phase 2 Clinical Trial of KP1077 for Idiopathic Hypersomnia at Sleep Europe 2024

KP1077 demonstrates clinically meaningful benefits for key IH symptoms Top-line data provide key information for the design of a Phase 3 study CELEBRATION, Fla., Sept. 24, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company) a commercial-stage rare disease therapeutics company, today announced that Rene Braeckman, Ph.D., Zevra’s Senior Vice President of Clinical Development, will present top-line data from the placebo-controlled, double-blind, randomized withdrawal Phase 2 clinical trial (NCT05668754) evaluating the safety and tolerability of KP1077 (serdexmethylphenidate, or SDX) in patients with idiopathic hypersomnia (IH) at Sleep Europe 2024, the 27th Congress of the European Sleep Research Society (ESRS) being held in Seville, Spain, September 24-27, 2024. Presentation Details Oral PresentationTitle: Safety and Efficacy of KP1077 in a Phase 2, Placebo-Controlled, Double-Blind, Randomized Withdrawal Study in Patients with Idiopathic HypersomniaDate/Time: Friday, September 27, 2024, 8:20 a.m. – 8:30 a.m. CETLocation: Auditorium 1 Poster PresentationTitle: Safety and Efficacy of KP1077 in a Phase 2, Placebo-Controlled, Double-Blind, Randomized Withdrawal Study in Patients with Idiopathic HypersomniaDate/Time: Thursday, September 26, 2024, 12:00 noon – 1:30 p.m. CET, and 5:30 p.m. – 6:45 p.m. CETCategory: NeurologyNumber: P771 About Sleep Europe 2024 The biennial congress of the European Sleep Research Society “Sleep Europe” is Europe’s leading congress on sleep medicine and sleep research. Every two years, thousands of clinicians, researchers, scientists, students & trainees come together to learn the latest sleep science news and network with their peers. For more information you can access the full program online here: https://esrs.eu/sleep-congress/scientific-programme/ About the KP1077 Phase 2 Trial The Phase 2 clinical trial (NCT05668754) was a double-blind, placebo-controlled, randomized-withdrawal, dose-optimizing, multi-center study that evaluated the safety and efficacy of KP1077 for the treatment of IH. Part 1 of the trial consisted of a 5-week open-label dose titration phase during which patients were optimized to one of four doses of KP1077 (80, 160, 240, or 320 mg/day). Part 2 of the trial entailed a two-week randomized, double-blind, withdrawal phase, during which two-thirds of the trial participants continued to receive their optimized dose while the remaining one-third received placebo. Participants were assigned into two evenly divided cohorts. The first cohort received a single daily dose just before bedtime, and the second cohort received half the daily dose shortly after awakening and the second half prior to bedtime. Zevra enrolled 66 adult patients with IH in 24 centers in the U.S. into the open-label titration phase of the study and 50 of those patients continued into the double-blind phase. The primary endpoint was the safety and tolerability of KP1077. The major secondary efficacy endpoint was the change in Epworth Sleepiness Scale (ESS) total score. Additional secondary endpoints included the IH Severity Scale (IHSS), the Sleep Inertia Visual Analog Scale (SIVAS), and a new scale to assess the symptoms and severity of brain fog. About Idiopathic Hypersomnia Idiopathic hypersomnia (IH) is a rare sleep disorder characterized by excessive daytime sleepiness (EDS). Patients with IH experience daytime lapses into sleep, or an irrepressible need to sleep that persists even with adequate or prolonged nighttime sleep. Additionally, those with IH have extreme difficulty waking, otherwise known as sleep inertia, severe brain fog, and often fall asleep unintentionally or at inappropriate times. These symptoms of IH often lead to further, even more debilitating problems such as memory lapses, difficulty maintaining focus, and depression. It is estimated, based on claims data, that approximately 37,000 patients in the United States are currently diagnosed with IH, although the total patient population may be much larger due to some patients who have not yet been diagnosed, have been misdiagnosed, or are not currently seeking treatment. About KP1077 KP1077 (serdexmethylphenidate or SDX) is Zevra’s proprietary prodrug of d-methylphenidate (d-MPH) and its sole active pharmaceutical ingredient (API). KP1077 has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA), and by the European Commission, for the treatment of IH. The U.S. Drug Enforcement Agency (DEA) has classified SDX, the sole API in KP1077, as a Schedule IV controlled substance based on evidence suggesting SDX has a lower potential for abuse when compared to d-MPH, a Schedule II controlled substance. In addition, KP1077 has intellectual property protection through 2037 and potentially beyond. About Zevra Therapeutics, Inc. Zevra Therapeutics, Inc. is a commercial-stage rare disease company combining science, data, and patient needs to create transformational therapies for diseases with limited or no treatment options. Our mission is to bring life-changing therapeutics to people living with rare diseases. With unique, data-driven development and commercialization strategies, the Company is overcoming complex drug development challenges to make new therapies available to the rare disease community. Expanded access programs are made available by Zevra Therapeutics, Inc. and its affiliates and are subject to the Company's Expanded Access Program (EAP) policy, as published on its website. Participation in these programs is subject to the laws and regulations of each jurisdiction under which each respective program is operated. Eligibility for participation in any such program is at the treating physician's discretion. For more information, please visit www.zevra.com or follow us on X (formerly Twitter) and LinkedIn. Cautionary Note Concerning Forward-Looking Statements: This press release may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include all statements that do not relate solely to historical or current facts, and which can be identified by the use of words such as “may,” “will,” “would,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplate,” “believe,” “estimate,” “predict,” “assume,” “intend,” “potential,” “continue” or other similar words or the negative of these terms, although not all forward-looking statements contain these identifying words or expressions. Forward-looking statements are not guarantees of future actions or performance. Forward-looking statements include all statements that do not relate solely to historical or current facts, including without limitation statements regarding anticipated consummation of the proposed offering, market conditions, the satisfaction of customary closing conditions related to the proposed offering, the completion of the offering on the anticipated terms or at all and general economic conditions. Forward-looking statements are based on information currently available to Zevra and its current plans or expectations. They are subject to several known and unknown uncertainties, risks, and other important factors that may cause Zevra’s actual results, performance, or achievements to be materially different from any future results, performance, or achievements expressed or implied by the forward-looking statements. These and other important factors are described in detail in the “Risk Factors” section of Zevra’s Annual Report on Form 10-K for the year ended December 31, 2023, subsequent Quarterly Reports on Form 10-Q, and other filings with the SEC. While Zevra may elect to update such forward-looking statements at some point in the future, except as required by law, Zevra disclaims any obligation to do so, even if subsequent events cause Zevra’s views to change. Although Zevra believes the expectations reflected in such forward-looking statements are reasonable, Zevra cannot assure that such expectations will prove correct. These forward-looking statements should not be relied upon as representing Zevra’s views as of any date after the date of this press release. Zevra Contact Nichol Ochsner+1 (732) 754-2545nochsner@zevra.com Russo Partners ContactsDavid Schull+1 (858) 717-2310david.schull@russopartnersllc.com Ignacio Guerrero-Ros, Ph.D.+1 (646) 942-5604Ignacio.guerrero-ros@russopartnersllc.com

Phase 2Orphan DrugClinical Result

100 Deals associated with Dexmethylphenidate Hydrochloride

External Link

KEGG	Wiki	ATC	Drug Bank
D03721	Dexmethylphenidate Hydrochloride	N06BA11	DB06701

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Attention Deficit Disorder With Hyperactivity	US	Sandoz, Inc.	13 Nov 2001

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Fatigue	Phase 2	US	Celgene Corp.	01 Jun 2002
Neoplasms	Phase 2	US	Celgene Corp.	01 Jun 2002
Neurobehavioral Manifestations	Phase 2	US	Celgene Corp.	01 Jun 2002

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05631626 (Corporate Publications) Manual	Phase 3	Attention Deficit Disorder With Hyperactivity	21	CTx-1301	lstamwhecf(rdvmaluutq) = Subjects who were randomized to their optimized dose of CTx-1301 showed improvements on the Permanent Product Measure of Performance (PERMP) (effect size 0.88 to 2.6; with an average of 1.79) compared to subjects randomized to placebo. dyotwlbmpt (pndpqvynki )	Positive	11 Jul 2023
				placebo
NCT00393042 (Pubmed \| J Child Adolesc Psychopharmacol)	Phase 3	Attention Deficit Disorder With Hyperactivity	77	Extended Release Dexmethylphenidate	oyxqmlzwru(mlxcezkyjr) = more common at higher dose levels for both stimulants ogzammwlii (bkibofbarr ) View more	-	01 Dec 2011
				Mixed Amphetamine Salts
NCT00714688 (CTgov)	Phase 3	Attention Deficit Disorder With Hyperactivity	279	placebo (Placebo)	flkfjmkmde(ipjzswiybn) = yavfsqfiyq vbqltpfxts (lawhuwihyj, nnnzdrwprj - kzjwnlxlol) View more	-	21 Apr 2010
				OROS MPH (54 mg PR OROS MPH)	flkfjmkmde(ipjzswiybn) = jtgvhoolek vbqltpfxts (lawhuwihyj, dlixpruear - mcvrxvbkbd) View more
NCT00829673 (CTgov)	Phase 1	-	24	Dexmethylphenidate (Dexmethylphenidate HCl)	kxajzyozmz(ygcgnqxkzy) = bxsqbyeexd wsleeghqej (mmnhjnpivp, bryissttug - vffvnavxzn) View more	-	04 Aug 2009
				Focalin (Focalin®)	kxajzyozmz(ygcgnqxkzy) = surwhxfvig wsleeghqej (mmnhjnpivp, unjiievfdk - bvrtgynlms) View more
NCT00829712 (CTgov)	Phase 1	-	24	Dexmethylphenidate (Dexmethylphenidate HCl)	redjwszwct(mxtggzmmku) = nqgshslnye kfoucfcmij (anlmsnjgfc, jwmaozvogg - iambpiwuzv) View more	-	04 Aug 2009
				Focalin (Focalin®)	redjwszwct(mxtggzmmku) = mdcpsplrul kfoucfcmij (anlmsnjgfc, uatpbgsjog - nqwvdwrxhn) View more
NCT00361387 (Pubmed \| Chest)	Phase 4	Fatigue	-	dexmethylphenidate hydrochloride	ldcjokpzaw(gssthuydkr) = ouorqfflsl bwcodulnwx (mttmenwcbs ) View more	Positive	01 May 2008
				placebo	ldcjokpzaw(gssthuydkr) = apyybolmes bwcodulnwx (mttmenwcbs ) View more

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