The study of investigators indicated that TMZ can up-regulate dopamine D2 receptor (DRD2) expression, and mediates Ferroptosis inhibition and chemoresistance of GBM. The clinical data also proved that the DRD2 expression in recurrent GBM is significantly higher than that in primary GBM. Moreover, the DRD2 antagonist haloperidol can attenuate the above function of DRD2, and increase the sensitivity of GBM to the TMZ by inducing fatal autophagy and ferroptosis. In xenograft mice, the combined usage of haloperidol and Temozolomide (TMZ) can significantly inhibit tumor growth and increase overall survival. The investigators' findings have been published in Clinical cancer research. Haloperidol known as a butylbenzene antipsychotic drug, has been widely used in several kinds of mental illnesses, such as depression, schizophrenia, and Bipolar disorder. And the safe dosage of the haloperidol is clear so far. So in this study, the investigators will recruit the patients who suffered from recurrent GBM, and evaluate the effectiveness of single TMZ chemotherapy or combined with haloperidol.

Start Date01 Dec 2024

Sponsor / Collaborator

Southern Medical University

CTRI/2024/10/075204 / Not yet recruitingNot ApplicableIIT

Comparison of effects of dexamethasone and haloperidol Vs dexamethasone and amisulpride on postoperative nausea and vomiting in laproscopic cholecystectomy Randomised, controlled, double blinded study. - NIL

Start Date20 Oct 2024

Sponsor / Collaborator

Sharda Hospital

TCTR20240417001 / RecruitingNot ApplicableIIT

A prospective observational study on QTc prolongation after receiving intravenous or intramuscular haloperidol injection for the management of acute agitation in hospitalized psychiatric patients

Start Date06 Aug 2024

Sponsor / Collaborator-

100 Clinical Results associated with Haloperidol

100 Translational Medicine associated with Haloperidol

100 Patents (Medical) associated with Haloperidol

20,283

Literatures (Medical) associated with Haloperidol

01 Mar 2025·BEHAVIOURAL BRAIN RESEARCH

Exploring the contribution of the dopaminergic and noradrenergic systems in the antidepressant-like action of 1-(2-(4-(4-ethylphenyl)-1H-1,2,3-triazol-1-yl)phenyl)ethanone in mice

Article

Author: Zuge, Narryman Pinto ; Goulart, Taís Barcelos ; da Rocha, Marcia Juciele ; Presa, Marcelo Heinemann ; Bortolatto, Cristiani Folharini ; Brüning, César Augusto ; Ledebuhr, Kauane Nayara Bahr ; Alves, Diego

1-(2-(4-(4-ethylphenyl)-1H-1,2,3-triazol-1-yl)phenyl)ethanone (ETAP) is a novel hybrid compound containing 1,2,3-triazole and acetophenone. It exhibits antidepressant-like effects in male mice, linked to modulation of serotonergic receptors and monoamine oxidase A (MAO-A) inhibition. This study aimed to evaluate the involvement of the dopaminergic and noradrenergic systems, as well as MAO-B activity inhibition, in the antidepressant-like effect of ETAP in male mice, and to evaluate the antidepressant-like effect of ETAP in female mice. Male mice were treated with different dopaminergic and noradrenergic receptors antagonists 15 min before administering ETAP (1 mg/kg, intragastrically, i.g.). The tail suspension test (TST) was performed 30 minutes later. Different male mice were treated with ETAP (1 mg/kg, i.g.), and 30 minutes later, were euthanized to assess MAO-B activity in the prefrontal cortex and hippocampus. To evaluate the antidepressant-like of ETAP in female mice, ETAP (1 mg/kg, i.g.) was administered, followed by the TST and the forced swimming test (FST) 30 minutes later. The dopaminergic antagonists haloperidol (0.05 mg/kg, intraperitoneally, i.p.), SCH23390 (0.01 mg/kg, subcutaneously, s.c.), and sulpiride (50 mg/kg, i.p.), as well the noradrenergic antagonists prazosin (1 mg/kg, i.p.), yohimbine (1 mg/kg, i.p.), and propranolol (2 mg/kg, i.p.), prevented the antidepressant-like effect of ETAP in the TST. MAO-B activity was unaffected by ETAP in both the prefrontal cortex and hippocampus. ETAP (1 mg/kg, i.g.) induced a significant antidepressant-like effect in female mice in the TST and FST. These findings provide valuable insights into the antidepressant-like effect of ETAP, highlighting its potential for developing more effective depression treatments.

01 Feb 2025·JOURNAL OF MOLECULAR HISTOLOGY

Neuroprotective effect of Bacillus subtilis in haloperidol induced rat model, targeting the microbiota-gut-brain axis

Article

Author: Kispotta, Sneha ; Sarangi, Prerna ; Dubey, Debasmita ; Prusty, Shakti Ketan ; Kar, Durga Madhab ; Rout, Monalisa

Functional microbes regulate Parkinson's disease (PD), according to contemporary research. The mechanism by which probiotics (PBT) improve PD was not fully explored yet. We examined the antioxidant impact and mechanism of PBT (Bacillus subtilis) on PD using gut-brain axis regulation. To establish a model of PD, rats were given haloperidol (HAL) intraperitoneally (i.p.) in this study. The standard group received L-DOPA for 21 days. After that, the motor function was assessed using different neurobehavioral tests. Further estimation comprehends the build up of alpha-synuclein, the manifestation of monoamine oxidase-B (MAO-B) activity, the deterioration of dopaminergic neurons and the induction of an oxidative stress reaction. In addition, the concentration of intestinal microbes was measured. These findings demonstrated that the administration of PBT in combination with L-dopa could alleviate motor impairments caused by HAL, the deterioration of dopaminergic neurons, and the build up of α-synuclein. Furthermore, the levels of superoxide dismutase (SOD) and dopamine were considerably raised by co-administration of L-dopa and PBT in the case of HAL-treated rats, whereas the levels of alpha-synuclein, MAO-B, and malondialdehyde (MDA) were reduced. Particularly, PBT administration reduced the gut microbial dysbiosis, which in turn raised the concentration of good bacteria i.e., Bifidobacterium and reduced the concentration of E. coli in experimental animals. These findings indicated that PBT might represent a promising candidate to inhibit the progression of Parkinson's disease by targeting the gut-brain axis.

01 Feb 2025·NEUROCHEMICAL RESEARCH

Glia-related Acute Effects of Risperidone and Haloperidol in Hippocampal Slices and Astrocyte Cultures from Adult Wistar Rats: A Focus on Inflammatory and Trophic Factor Release

Article

Author: Lissner, Lílian Juliana ; da Silva, Amanda ; de Souza Almeida, Rômulo Rodrigo ; Quincozes-Santos, André ; Dos Santos, Tiago Marcon ; Bobermin, Larissa Daniele ; Leite, Marina Concli ; Wyse, Angela T S ; Seady, Marina ; Santos, Camila Leite ; Gonçalves, Carlos-Alberto

Antipsychotics are drugs commonly prescribed to treat a variety of psychiatric conditions. They are classified as typical and atypical, depending on their affinity for dopaminergic and serotonergic receptors. Although neurons have been assumed to be the major mediators of the antipsychotic pharmacological effects, glia, particularly astrocytes, have emerged as important cellular targets for these drugs. In the present study, we investigated the effects of acute treatments with the antipsychotics risperidone and haloperidol of hippocampal slices and astrocyte cultures, focusing on neuron-glia communication and how antipsychotics act in astrocytes. For this, we obtained hippocampal slices and primary astrocyte cultures from 30-day-old Wistar rats and incubated them with risperidone or haloperidol (1 and 10 μM) for 30 min and 24 h, respectively. We evaluated metabolic and enzymatic activities, the glutathione level, the release of inflammatory and trophic factors, as well as the gene expression of signaling proteins. Haloperidol increased glucose metabolism; however, neither of the tested antipsychotics altered the glutathione content or glutamine synthetase and Na⁺K⁺-ATPase activities. Haloperidol induced a pro-inflammatory response and risperidone promoted an anti-inflammatory response, while both antipsychotics seemed to decrease trophic support. Haloperidol and risperidone increased Nrf2 and HO-1 gene expression, but only haloperidol upregulated NFκB and AMPK gene expression. Finally, astrocyte cultures confirmed the predominant effect of the tested antipsychotics on glia and their opposite effects on astrocytes. Therefore, antipsychotics cause functional alterations in the hippocampus. This information is important to drive future research for strategies to attenuate antipsychotics-induced neural dysfunction, focusing on glia.

News (Medical) associated with Haloperidol

12 Nov 2024

·www.globenewswire.com

Zevra Therapeutics Reports Third Quarter 2024 Financial Results and Corporate Updates

FDA approval of MIPLYFFA™ and product launch cap third quarter filled with multiple commercial and clinical development milestones Zevra received rare pediatric disease Priority Review Voucher Company ends quarter with cash, cash equivalents and investments of $95.5 million Company to host conference call and webcast today, Nov. 12, 2024, 4:30 p.m. ET CELEBRATION, Fla., Nov. 12, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on addressing unmet needs for the treatment of rare diseases, today reported its financial results for the third quarter ended Sept. 30, 2024, and provided corporate updates. “The third quarter was one of the most exciting and transformational periods in Zevra’s journey,” said Neil F. McFarlane, Zevra’s President and Chief Executive Officer. “After years of tireless effort, our team has achieved a major milestone with FDA approval of MIPLYFFA™ (arimoclomol), and we’re celebrating with the Niemann-Pick disease type C (NPC) community. We intend to achieve our goals with a high-performing team committed to execute, focus and innovate to drive our continued growth and long-term transformation.” Program Highlights On Sept. 20, the U.S. Food and Drug Administration (FDA) approved MIPLYFFA capsules as an orally delivered treatment for NPC. MIPLYFFA, the first NPC drug approved by the FDA, is indicated for use in combination with miglustat for the treatment of neurological manifestations of NPC in adult and pediatric patients 2 years of age and older. With the approval of MIPLYFFA, Zevra received a rare pediatric disease Priority Review Voucher (PRV) which the Company could monetize to help fund its growth. As of Oct. 31, Zevra has received 90 prescription enrollment forms for MIPLYFFA of which 30% are approved for reimbursement and are ready for fulfillment upon drug availability, consistent with the Company’s prior guidance of eight to 12 weeks post launch. Zevra has launched AmplifyAssist™, a comprehensive support program for caregivers and those eligible living with NPC or UCDs. OLPRUVA® had three new patient enrollments in the third quarter, and reimbursement coverage is at approximately 76% of covered lives. Improving OLPRUVA’s formulary position is a primary objective to make it the preferred treatment for UCDs. Zevra is refining its commercial strategy for OLPRUVA to focus on specific patient segments where there are fewer access barriers, and, together with The National Urea Cycle Disorders Foundation, launched a targeted patient education campaign highlighting the importance of prompt and accurate testing of blood ammonia levels, which, when elevated, may be a sign of a UCD. Campaign materials are available at www.checkammonia.com. The Company recently completed an End-of-Phase 2 meeting with the FDA for KP1077. The Agency indicated that a single pivotal study with appropriate confirmatory evidence will be sufficient to submit a new drug application (NDA). Zevra is evaluating strategic alternatives to advance clinical development and later, commercialization. The Phase 3 DiSCOVER trial of celiprolol for the treatment of Vascular Ehler-Danlos Syndrome (VEDS) is underway, and the Company has achieved its objective to restart enrollment and to begin dosing new patients, reporting that a total of 19 patients are now in the trial as of the end of the third quarter. Zevra is continuing its efforts to expand its partnerships with patient advocacy groups, treatment centers of excellence, and medical key opinion leaders. Corporate Highlights and Upcoming Milestones In August, Zevra completed an underwritten public offering of 10,615,385 shares of its common stock at an offering price of $6.50 per share for total net proceeds of approximately $64.5 million, after deducting underwriting discounts and commissions and offering expenses. Cantor and William Blair were joint book-running managers for the offering. Citizens JMP and H.C. Wainwright & Co. were co-managers. The Company completed a thorough portfolio assessment and developed a strategic plan for 2025 that builds from its 2024 accomplishments and furthers Zevra’s transformation towards becoming a leading rare disease company with a five-year vision to create value for patients and shareholders by organizing our priorities on four key pillars: Commercial Excellence, Pipeline and Innovation, Talent and Culture, and Corporate Foundation. Now focused on late-stage clinical development and commercial opportunities, Zevra has discontinued its in-house drug discovery activities and is closing its laboratory facilities in Iowa and Virginia. Future early research and development activities will be outsourced. Q3 2024 Financial Highlights Revenue, Net: $3.7 million, comprised of $2.6 million in net reimbursements from the French expanded access program (EAP) for arimoclomol, $1.1 million of royalties and other reimbursements under the AZSTARYS® license agreement, and de minimis OLPRUVA revenue. Cost of Goods Sold: $2.3 million, which includes recognition of $2.0 million in inventory obsolescence reserve expense related to OLPRUVA inventory that is nearing expiration. R&D Expenses: $10.9 million, which represents a decrease of $1.4 million compared to Q3 2023 due to the completion of the KP1077 phase 2 trial. SG&A Expenses: $16.2 million for Q3 2024, compared to $5.8 million in Q3 2023, which reflects the commercial team fully in place and actively engaged in commercial launch activities and patient services initiatives. This includes non-cash stock compensation of $6.1 million during the quarter, of which $2.5 million was performance-based upon the approval of MIPLYFFA. Net Loss: ($33.2) million, or ($0.69) per basic and diluted share for Q3 2024, compared to a net loss of ($10.4) million, or ($0.30) per basic and diluted share in Q3 2023. Cash Position: Cash, cash equivalents and investments were $95.5 million as of Sept. 30, 2024. Cash Runway Forecast: Based on the Company’s current operating plan, available cash, cash equivalents and investments are expected to extend Zevra’s cash runway into 2027, subject to continuing compliance with debt covenants. Cash runway forecast includes anticipated revenue from MIPLYFFA sales, reimbursements from the French EAP for arimoclomol, royalties under the AZSTARYS license agreement, and continued investments into our development pipeline programs. It does not include potential proceeds from a PRV sale. Common and Fully Diluted Shares O/S: As of Sept. 30, 2024, total shares of common stock outstanding were 53,227,364, and fully diluted common shares were 67,698,898, which included 5,483,537 shares issuable upon exercise of warrants. Upcoming Events Guggenheim Securities Healthcare Innovation Conference, with fireside chat on Wednesday, Nov. 13, 2024, at 4:00 p.m. ET. Management will be available for one-on-one meetings with registered attendees. Conference Call Information - UPDATED Zevra will host a conference call and audio webcast today at 4:30 p.m. ET to discuss its corporate update and financial results for the third quarter of 2024. The audio webcast will be accessible via the Investor Relations section of the Company’s website, http://investors.zevra.com/. An archive of the audio webcast will be available for ninety (90) days beginning at approximately 5:30 p.m. ET on Nov. 12, 2024. Additionally, interested participants and investors may access the conference call by dialing either: (785) 838-9345 (United States) -- updated dial-in +1 (203) 518-9814 (International)Conference ID: ZVRAQ324 About MIPLYFFA MIPLYFFA (arimoclomol) increases the activation of the transcription factors EB (TFEB) and E3 (TFE3) resulting in the upregulation of coordinated lysosomal expression and regulation (CLEAR) genes. MIPLYFFA has also been shown to reduce unesterified cholesterol in the lysosomes of human NPC fibroblasts. The clinical significance of these findings is not fully understood. MIPLYFFA was granted Breakthrough Therapy designation, Rare Pediatric Disease designation, Orphan Drug designation, and Fast Track designation by the FDA for the treatment of NPC. MIPLYFFA was further granted Orphan Medicinal Product designation by the European Medicines Agency (EMA) for the treatment of NPC. INDICATIONS AND USAGE MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older. IMPORTANT SAFETY INFORMATION Hypersensitivity Reactions: Hypersensitivity reactions such as urticaria and angioedema have been reported in patients treated with MIPLYFFA during Trial 1: two patients reported both urticaria and angioedema (6%) and one patient (3%) experienced urticaria alone within the first two months of treatment. Discontinue MIPLYFFA in patients who develop severe hypersensitivity reactions. If a mild or moderate hypersensitivity reaction occurs, stop MIPLYFFA and treat promptly. Monitor the patient until signs and symptoms resolve. Embryofetal Toxicity: MIPLYFFA may cause embryofetal harm when administered during pregnancy based on findings from animal reproduction studies. Advise pregnant females of the potential risk to the fetus and consider pregnancy planning and prevention for females of reproductive potential. Increased Creatinine without Affecting Glomerular Function: Across clinical trials of MIPLYFFA, mean increases in serum creatinine of 10% to 20% compared to baseline were reported. These increases occurred mostly in the first month of MIPLYFFA treatment and were not associated with changes in glomerular function. During MIPLYFFA treatment, use alternative measures that are not based on creatinine to assess renal function. Increases in creatinine reversed upon MIPLYFFA discontinuation. The most common adverse reactions in Trial 1 (≥15%) in MIPLYFFA-treated patients who also received miglustat were upper respiratory tract infection, diarrhea, and decreased weight. Three (6%) of the MIPLYFFA-treated patients had the following adverse reactions that led to withdrawal from Trial 1: increased serum creatinine (one patient), and progressive urticaria and angioedema (two patients). Serious adverse reactions reported in MIPLYFFA-treated patients were hypersensitivity reactions including urticaria and angioedema. To report SUSPECTED ADVERSE REACTIONS, contact Zevra Therapeutics, Inc. at toll-free phone 1-844-600-2237 or FDA at 1 800-FDA-1088 or www.fda.gov/medwatch. Before prescribing MIPLYFFA, please read the full Prescribing Information, including Instructions for Use. Drug Interaction(s): Arimoclomol is an inhibitor of the organic cationic transporter 2 (OCT2) transporter and may increase the exposure of drugs that are OCT2 substrates. When MIPLYFFA is used concomitantly with OCT2 substrates, monitor for adverse reactions and reduce the dosage of the OCT2 substrate. Use in Females and Males of Reproductive Potential: Based on animal findings, MIPLYFFA may impair fertility and may increase post-implantation loss and reduce maternal, placental, and fetal weights. Renal Impairment: The recommended dosage of MIPLYFFA, in combination with miglustat, in patients with an eGFR ≥15 mL/minute to <50 mL/minute is lower than the recommended dosage (less frequent dosing) in patients with normal renal function. MIPLYFFA capsules for oral use are available in the following strengths: 47 mg, 62 mg, 93 mg, and 124 mg. About OLPRUVA OLPRUVA (sodium phenylbutyrate) was approved for the treatment of certain UCDs in December 2022 and has recently been marketed under the brand name, OLPRUVA. OLPRUVA (sodium phenylbutyrate) for oral suspension is a prescription medicine used along with certain therapies, including changes in diet, for the long-term management of adults and children weighing 44 pounds (20 kg) or greater and with a body surface area (BSA) of 1.2 m2 or greater, with UCDs, involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). OLPRUVA is not used to treat rapid increase of ammonia in the blood (acute hyperammonemia), which can be life-threatening and requires emergency medical treatment. For more information, please visit www.OLPRUVA.com. Important Safety Information Certain medicines may increase the level of ammonia in your blood or cause serious side effects when taken during treatment with OLPRUVA. Tell your doctor about all the medicines you or your child take, especially if you or your child take corticosteroids, valproic acid, haloperidol, and/or probenecid. OLPRUVA can cause serious side effects, including: 1) nervous system problems (neurotoxicity). Symptoms include sleepiness, tiredness, lightheadedness, vomiting, nausea, headache, confusion, 2) low potassium levels in your blood (hypokalemia) and 3) conditions related to swelling (edema). OLPRUVA contains salt (sodium), which can cause swelling from salt and water retention. Tell your doctor right away if you or your child get any of these symptoms. Your doctor may do certain blood tests to check for side effects during treatment with OLPRUVA. If you have certain medical conditions such as heart, liver or kidney problems, are pregnant/planning to get pregnant or breast-feeding, your doctor will decide if OLPRUVA is right for you. The most common side effects of OLPRUVA include absent or irregular menstrual periods, decreased appetite, body odor, bad taste or avoiding foods you ate prior to getting sick (taste aversion). These are not all of the possible side effects of OLPRUVA. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088. About KP1077 Serdexmethylphenidate (SDX) is Zevra’s proprietary prodrug of d-methylphenidate (d-MPH) and is the sole active pharmaceutical ingredient (API) of KP1077. KP1077 has been granted Orphan Drug designation by the FDA, and by the European Commission, for the treatment of Idiopathic Hypersomnia, or IH. The U.S. Drug Enforcement Agency (DEA) has classified SDX 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. About Celiprolol Celiprolol is an investigational clinical candidate for the treatment of Vascular Ehlers-Danlos Syndrome (VEDS). Celiprolol has been granted Orphan Drug and Breakthrough Therapy designations by the FDA. Zevra recently restarted enrollment in the Phase 3 trial, known as the DiSCOVER trial being conducted under a Special Protocol Assessment (SPA) agreement with the FDA. Celiprolol’s mechanism of action is designed to reduce the mechanical stress on collagen fibers within the arterial wall through vascular dilation and smooth muscle relaxation. 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 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, including without limitation statements regarding the promise and potential impact of our preclinical or clinical trial data; the initiation, timing and results of any clinical trials or readouts, the content, information used for, timing or results of any NDA submissions or resubmissions for any products or product candidates for any specific disease indication or at any dosage; the potential benefits of any of our products or product candidates for any specific disease or at any dosage; the impact of meetings or communications with the FDA or any advisory committee; statements or decisions by the FDA or any other entity for any products or product candidates; our ability to monetize any PRV; future research and development activities; our strategic and product development objectives, including with respect to becoming a leading, commercially focused rare disease company; the potential benefits of our debt facility; our financial position, including our cash balance; our corporate governance objectives; potential revenues from our arimoclomol expanded access program in France; the potential for royalty and milestone contributions, the presentation of data at conferences; and the timing of any of the foregoing. 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 our 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 Dec. 31, 2023, Zevra’s Quarterly Report on Form 10-Q for the three months ended Sept. 30, 2024, and Zevra’s other filings with the Securities and Exchange Commission. While we may elect to update such forward-looking statements at some point in the future, except as required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Although we believe the expectations reflected in such forward-looking statements are reasonable, we cannot assure that such expectations will prove correct. These forward-looking statements should not be relied upon as representing our views as of any date after the date of this press release. Zevra Contact Nichol Ochsner+1 (732) 754-2545nochsner@zevra.com Russo Partners Contact David Schull+1 (858) 717-2310david.schull@russopartnersllc.com ZEVRA THERAPEUTICS, INC. UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS(in thousands, except share and per share amounts) Three months ended September 30, Nine months ended September 30, 2024 2023 2024 2023 Revenue, net $3,695 $2,895 $11,569 $14,541 Cost of product revenue (excluding $1,545 and $4,619 in intangible asset amortization for the three and nine months ended September 30, 2024, respectively shown separately below) 2,303 144 6,051 946 Intangible asset amortization 1,545 — 4,619 — Operating expenses: Research and development 10,945 12,297 33,743 28,385 Selling, general and administrative 16,208 5,818 38,743 19,657 Total operating expenses 27,153 18,115 72,486 48,042 Loss from operations (27,306) (15,364) (71,587) (34,447)Other (expense) income: Interest expense (2,312) (366) (5,157) (745)Fair value adjustment related to warrant and CVR liability (4,746) 3,678 4,660 4,253 Fair value adjustment related to investments 90 124 64 451 Interest and other income (expense), net 1,049 1,738 2,248 4,331 Total other (expense) income (5,919) 5,174 1,815 8,290 Loss before income taxes (33,225) (10,190) (69,772) (26,157)Income tax expense — (177) — — Net loss $(33,225) $(10,367) $(69,772) $(26,157) Basic and diluted net loss per share of common stock: Net loss $(0.69) $(0.30) $(1.59) $(0.76) Weighted average number of shares of common stock outstanding: Basic and diluted 47,808,817 34,724,614 43,843,851 34,364,075 ZEVRA THERAPEUTICS, INC. UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS (in thousands, except share and par value amounts) September 30, December 31, 2024 2023 Assets Current assets: Cash and cash equivalents $54,039 $43,049 Securities at fair value, current 35,337 24,688 Accounts and other receivables 7,790 17,377 Prepaid expenses and other current assets 2,276 1,824 Total current assets 99,442 86,938 Inventories 8,756 9,841 Securities at fair value, long-term 6,105 — Property and equipment, net 607 736 Operating lease right-of-use assets 820 790 Goodwill 4,701 4,701 Intangible assets, net 70,608 69,227 Other long-term assets 512 94 Total assets $191,551 $172,327 Liabilities and stockholders' equity Current liabilities: Accounts payable and accrued expenses $24,464 $28,403 Line of credit payable — 37,700 Current portion of operating lease liabilities 540 543 Current portion of discount and rebate liabilities 8,547 4,550 Other current liabilities 930 2,524 Total current liabilities 34,481 73,720 Long-term debt 58,904 5,066 Warrant liability 13,902 16,100 Operating lease liabilities, less current portion 483 456 Discount and rebate liabilities, less current portion 8,490 7,663 Other long-term liabilities 5,521 7,458 Total liabilities 121,781 110,463 Commitments and contingencies Stockholders’ equity: Preferred stock: Undesignated preferred stock, $0.0001 par value, 10,000,000 shares authorized, no shares issued or outstanding as of September 30, 2024, or December 31, 2023 — — Common stock, $0.0001 par value, 250,000,000 shares authorized, 54,803,056 shares issued and 53,227,364 shares outstanding as of September 30, 2024; 43,110,360 shares issued and 41,534,668 shares outstanding as of December 31, 2023 5 4 Additional paid-in capital 550,413 472,664 Treasury stock, at cost (10,983) (10,983)Accumulated deficit (469,550) (399,778)Accumulated other comprehensive income (loss) (115) (43)Total stockholders' equity 69,770 61,864 Total liabilities and stockholders' equity $191,551 $172,327

Phase 3Drug ApprovalBreakthrough TherapyOrphan Drug

06 Sep 2024

·www.globenewswire.com

Zevra Therapeutics Presented New Data for Arimoclomol and OLPRUVA® (Sodium Phenylbutyrate) at the Society for the Study of Inborn Errors of Metabolism (SSIEM) 2024 Annual Symposium

New clinical efficacy and safety data for arimoclomol as a possible treatment for Niemann-Pick disease type C, including from long-term and real-world settings, demonstrate clinically meaningful reduction in disease progression Arimoclomol was well tolerated during the Open Label Extension trial and Early Access Program with no safety signals identified PK modeling studies showed that administration of OLPRUVA while fasting vs fed results in higher drug exposure which may allow for lower effective dosages when taken without food CELEBRATION, Fla., Sept. 06, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics company, today announced the presentation of five posters at the Society for the Study of Inborn Errors of Metabolism (SSIEM) 2024 Annual Symposium. Four posters focused on data from multiple studies showing the efficacy and safety of arimoclomol as a treatment for people living with Niemann-Pick disease type C (NPC), and one poster highlighted data from pharmacokinetic modeling studies of OLPRUVA®, a therapy for the long-term management of certain adult and pediatric patients with urea cycle disorders (UCDs) involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC) or argininosuccinic acid synthetase (AS). “The data collected during the Phase 2/3 study of arimoclomol, including long-term data from Open Label Extension (OLE) and Early Access Program (EAP) participants, add to the large body of evidence that demonstrates arimoclomol’s clinical efficacy and safety as a treatment for people living with NPC,” said Adrian Quartel, M.D., FFPM, Chief Medical Officer of Zevra. “Additionally, we presented PK modeling data from OLPRUVA in both adult and pediatric virtual patients, showing an increase in drug exposure under fasting conditions.” Presentation Details The data for arimoclomol presented at SSIEM is summarized below: Poster Number: 21260 Title:Efficacy Results from a 12-month Double-blind Randomized Trial of Arimoclomol for Treatment of Niemann Pick Disease Type C – presenting an improved 4-Domain NPC Clinical Severity Scale Authors:Marc Patterson, Sven Guenther and Christine i Dali Summary:The treatment effect of arimoclomol was evaluated in a 12-month, double-blind, placebo-controlled clinical trial (NCT02612129) using the original 5-domain Niemann pick type C clinical severity scale (5DNPCCSS) and the modified 4-domain Niemann Pick type C clinical severity scale (4DNPCCSS). A statistically significant treatment effect was shown using the modified 4DNPCCSS and the prespecified 5DNPCCSS primary endpoint in the 12-month clinical trial, representing a clinically meaningful reduction in disease progression with arimoclomol treatment compared to placebo. Poster Number: 21271 Title:Long-term Efficacy and Safety Evaluation of Arimoclomol Treatment in Patients with Niemann Pick Type C – Data from 48 Months Open Label Trial Authors:Marc Patterson, Eugene Mengel, Sven Guenther and Christine i Dali Summary:The long-term safety and efficacy of arimoclomol in the 12-month double-blind, and 48-month open-label extension (OLE) portion of the clinical trial (NCT02612129) were presented using the 4-Domain Niemann Pick Type C Clinical Severity scale (4DNPCCSS) which evaluates ambulation, speech, swallowing and fine motor skills. For those patients transitioning from placebo to arimoclomol at the start of the open-label extension period, the mean annual rate of disease progression reduced from an annual rate of change of 1.9 points during the double-blind phase, to a rate of 0.3 in the first 12 months of treatment, remained numerically smaller for the rest of the trial, and was comparable between the double-blind phase of the trial and the open-label extension phase of the trial. Additionally, arimoclomol was well tolerated with no new safety signals observed. Poster Number: PO-212 Title:Arimoclomol for the Treatment of NPC in a Real-World Setting: Long-term Outcomes from an Expanded Access Program in the USA Authors:Walla Al-Hertani, Elizabeth M. Berry-Kravis, Raymond Wang, Marc Patterson, Can Ficicioglu, Loren Pena, Kristina Julich, Damara Ortiz, Paula Schleifer, Caroline Hastings, Paul Hillman, Ronan O'Reilly, Christine Dali and Daniel Gallo Summary:The long-term safety and efficacy of arimoclomol in a real-world setting were evaluated in a total of 56 adult and pediatric patients in the U.S. arimoclomol expanded access program (EAP) trial (NCT04316637). Data presented included over 3 years of U.S. EAP clinical outcomes and demonstrated that adult and pediatric patients treated with arimoclomol, including those with and without miglustat as a component of their routine clinical care, experienced relatively stable disease as measured by the 5DNPCCSS and 4DNPCCSS and show that arimoclomol was well tolerated. Poster Number: 20950 Title:Arimoclomol safety profile in the treatment of NPC in a Real-World setting: Long-term data from an Expanded Access program in the USA Authors:Can Ficicioglu, Elizabeth M. Berry-Kravis, Walla Al-Hertani, Raymond Wang, Marc Patterson, Loren Pena, Kristina Julich, Damara Ortiz, Paula Schleifer, Paul Hillman, Caroline Hastings, Ronan O'Reilly, Christine Dali and Daniel Gallo Summary:Safety data from 94 adult and pediatric participants in the arimoclomol US EAP were presented. Safety outcomes reported from exposure periods spanning as much as 46 months of treatment, demonstrated a safety profile consistent with the published clinical trial experience of arimoclomol in NPC. The data for OLPRUVA presented at SSIEM is summarized below: Poster Number: PO-609 Title:Modeling the Pharmacokinetics of Phenylbutyrate in Fed and Fasted States Authors:Steiner, Rebecca Baillie, Tongli Zhang, Christina Friedrich, Meredith Hart, Mike Reed Summary:Pharmacokinetic modeling evaluating whether sodium phenylbutyrate (NaPBA) could be safely and effectively administered while fasting showed greater absorption and bioavailability with increased drug exposure in fasted administration of NaPBA compared to fed administration of NaPBA and glycerol phenylbutyrate (GPB) in both adult and child virtual patients, which is predicted to increase efficacy in proportion to increased drug exposure, theoretically allowing for a 30% dose decrease compared to fed conditions. About the SSIEM 2024 Annual Symposium The Society for the Study of Inborn Errors of Metabolism (SSIEM) 2024 Annual Symposium took place September 3-6, 2024, in Porto, Portugal. The annual symposium is intended to foster the study of inherited metabolic disorders and promote the exchange of ideas between professionals in different disciplines who are researching inborn errors of metabolism (IEM). About Niemann-Pick Disease Type C (NPC) Niemann-Pick disease type C (NPC) is an ultra-rare, progressive, and neurodegenerative lysosomal storage disorder characterized by an inability of the body to transport cholesterol and other lipids within the cell, leading to an accumulation of these substances in various tissue areas, including brain tissue. The disease is caused by mutations in the NPC1 or NPC2 genes, which are responsible for making lysosomal proteins. Both children and adults can be affected by NPC with varying clinical presentations. Those living with NPC lose independence due to physical and cognitive limitations, with key neurological impairments presenting in speech, cognition, swallowing, ambulation, and fine motor skills. Disease progression is irreversible and can be fatal within months or take years to be diagnosed and advance in severity. About Arimoclomol Arimoclomol, Zevra’s orally-delivered, investigational drug product candidate for the treatment of NPC, has been granted Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, and Rare Pediatric Disease designation by the FDA, and Orphan Medicinal Product designation for the treatment of NPC by the European Medicines Agency (EMA). The FDA has accepted the resubmission of the NDA for arimoclomol and has set a user fee action date (PDUFA date) of September 21, 2024. About Urea Cycle Disorders UCDs are a group of rare, genetic disorders that can cause harmful ammonia to build up in the blood, potentially resulting in brain damage and neurocognitive impairments if ammonia levels are not controlled. Any increase in ammonia over time is serious. Therefore, it is important to adhere to any dietary protein restrictions and have alternative medication options to help control ammonia levels. About OLPRUVA® OLPRUVA (sodium phenylbutyrate) was approved for the treatment of certain UCDs in December 2022 and has recently been marketed under the brand name, OLPRUVA®. OLPRUVA (sodium phenylbutyrate) for oral suspension is a prescription medicine used along with certain therapies, including changes in diet, for the long-term management of adults and children weighing 44 pounds (20 kg) or greater and with a body surface area (BSA) of 1.2 m2 or greater, with UCDs, involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). OLPRUVA is not used to treat rapid increase of ammonia in the blood (acute hyperammonemia), which can be life-threatening and requires emergency medical treatment. For more information, please visit www.OLPRUVA.com. Important Safety Information Certain medicines may increase the level of ammonia in your blood or cause serious side effects when taken during treatment with OLPRUVA. Tell your doctor about all the medicines you or your child take, especially if you or your child take corticosteroids, valproic acid, haloperidol, and/or probenecid. OLPRUVA can cause serious side effects, including: 1) nervous system problems (neurotoxicity). Symptoms include sleepiness, tiredness, lightheadedness, vomiting, nausea, headache, confusion, 2) low potassium levels in your blood (hypokalemia) and 3) conditions related to swelling (edema). OLPRUVA contains salt (sodium), which can cause swelling from salt and water retention. Tell your doctor right away if you or your child get any of these symptoms. Your doctor may do certain blood tests to check for side effects during treatment with OLPRUVA. If you have certain medical conditions such as heart, liver or kidney problems, are pregnant/planning to get pregnant or breast-feeding, your doctor will decide if OLPRUVA is right for you. The most common side effects of OLPRUVA include absent or irregular menstrual periods, decreased appetite, body odor, bad taste or avoiding foods you ate prior to getting sick (taste aversion). These are not all of the possible side effects of OLPRUVA. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088. About Zevra Therapeutics, Inc. Zevra Therapeutics, Inc. is a 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 and its affiliates and are subject to the Company's Expanded Access Program (EAP) policy as published on its website at www.zevra.com. 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, including without limitation statements regarding the promise and zpotential impact of our preclinical or clinical trial data; the initiation, timing and results of any clinical trials or readouts, the content, information used for, timing or results of any NDA submissions or resubmissions for arimoclomol or any other product candidates for any specific disease indication or at any dosage; the potential benefits of any of our products or product candidates for any specific disease or at any dosage; our strategic and product development objectives, including with respect to becoming a leading, commercially focused rare disease company; potential revenues from our arimoclomol expanded access program; the potential for royalty and milestone contributions, the presentation of data at conferences; and the timing of any of the foregoing. 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 our 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, Zevra’s Quarterly Report on Form 10-Q for the three months ended June 30, 2024, and Zevra’s other filings with the Securities and Exchange Commission. While we may elect to update such forward-looking statements at some point in the future, except as required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Although we believe the expectations reflected in such forward-looking statements are reasonable, we cannot assure that such expectations will prove correct. These forward-looking statements should not be relied upon as representing our views as of any date after the date of this press release. Zevra Contact Nichol Ochsner+1 (732) 754-2545nochsner@zevra.com Russo Partners Contacts Adanna G. Alexander, Ph.D.+1 (646) 942-5603adanna.alexander@russopartnersllc.com Ignacio Guerrero-Ros, Ph.D.+1 (646) 942-5604ignacio.guerrero-ros@russopartnersllc.com

Clinical ResultOrphan DrugFast TrackBreakthrough TherapyDrug Approval

26 Aug 2024

·www.prnewswire.com

Schizophrenia Market: RISVAN's Approval Has Sparked a Competitive Race Among Pharmaceutical Companies | DelveInsight

The recent approval of Rovi Pharmaceuticals Laboratories' OKEDI/RISVAN (risperidone ISM) has spiked the competition among the other pharmaceutical companies that are developing novel therapies to improve the treatment landscape. RISVAN has the potential to become a blockbuster drug. LAS VEGAS, Aug. 26, 2024 /PRNewswire/ -- Schizophrenia is a severe mental illness affecting about 1% of Americans, cognitive functions, emotional responses, decision-making, and social interaction. Onset typically occurs in late teens to early 20s for men and late 20s to early 30s for women. Currently, as per the WHO, the vast majority of schizophrenia patients around the world are not receiving mental health care. Approximately 50% of people in mental hospitals have a schizophrenia diagnosis, and only 31.3% of people with psychosis receive specialist mental health care. As per DelveInsight analysis, in 2023 the total diagnosed prevalent cases of schizophrenia in the 7MM were found to be approximately 4 million cases out of which the US accounted for nearly 1.5 million cases. It is anticipated that these numbers will rise by 2034 due to several factors, including population growth, improved diagnostic techniques, increased awareness, and reduced stigma around mental health issues prompting more individuals to seek diagnosis, and potentially, environmental stressors contributing to a higher occurrence of the disorder. Schizophrenia treatment involves a blend of medication, psychological counseling, and social support. However, nearly one-third of patients do not respond sufficiently to standard treatments. Relapses, characterized by psychotic symptoms and functional impairments, are significant. Schizophrenia treatment mainly consists of antipsychotic medications that target dopamine neurotransmitters. Second-generation medications are favored because they lower the risk of severe side effects. Long-acting injectable antipsychotics can enhance adherence and improve treatment outcomes. High-dose regimens of specific medications may be advantageous for patients with severe, treatment-resistant schizophrenia. Additionally, hospitalization and electroconvulsive therapy might be necessary for patients who do not respond to medication therapy or during severe episodes. First-generation antipsychotics like chlorpromazine, fluphenazine, haloperidol, and perphenazine are effective in treating positive symptoms such as hallucinations and delusions but do not alleviate negative symptoms or cognitive impairments. Second-generation antipsychotics, including REXULTI/RXULTI (brexpiprazole), CAPLYTA (lumateperone), LATUDA (lurasidone hydrochloride), SAPHRIS (asenapine), ABILIFY MYCITE (aripiprazole tablets with sensor), VRAYLAR/REAGILA (cariprazine), SECUADO (asenapine), INVEGA SUSTENNA/TRINZA/HAYFERA (paliperidone palmitate), ARISTADA/ARISTADA INITIO (aripiprazole lauroxil), PERSERIS (risperidone), FANAPT (iloperidone), and LYBALVI (olanzapine and samidorphan), were developed to address both positive and negative symptoms, as well as cognitive symptoms. Although these medications typically have fewer extrapyramidal side effects, they can cause weight gain, sexual dysfunction, and other metabolic disorders. Additionally, elderly patients using FGAs and SGAs are at an increased risk of developing pneumonia. Learn more about the FDA-approved schizophrenia drugs @ Drugs for Schizophrenia Treatment REXULTI (brexpiprazole) is an oral tablet available in strengths from 0.25 mg to 4 mg, containing various inactive ingredients. While its exact mechanism of action is not fully understood, it work as a partial agonist at serotonin 5-HT1A and dopamine D2 receptors and as an antagonist at serotonin 5-HT2A receptors. REXULTI was first approved in the US in July 2015 for use in adults with MDD and schizophrenia. In January 2022, it received FDA approval for pediatric patients aged 13–17. The EMA approved it in July 2018, and Japan approved it in tablet form in January 2018 and as an oral-disintegrating dose in August 2021. Generic versions of REXULTI have now been approved, which may affect its market presence. Janssen Pharmaceutical's INVEGA SUSTENNA/INVEGA TRINZA/INVEGA HAFYERA (paliperidone palmitate) is an SGA agent that seems to act as a D2 partial agonist and a 5-HT-2A receptor antagonist. INVEGA SUSTENNA is administered monthly, INVEGA TRINZA every three months, and INVEGA HAFYERA twice yearly. Paliperidone is a benzisoxazole derivative. In the EU and Japan, INVEGA SUSTENNA is marketed as XEPLION. In the EU, INVEGA TRINZA is sold as TREVICTA, and in Japan as XEPLION TRI. INVEGA HAFYERA is a long-acting injectable treatment administered in the upper buttocks every six months, providing continuous treatment and symptom control over this period. Additionally, the recent approval of RISVAN (Risperidone ISM) has intensified the competition in the schizophrenia treatment space. Risperidone ISM is a long-acting injectable antipsychotic created and patented by ROVI for treating schizophrenia in adults. From the first injection, it delivers immediate and sustained plasma drug levels without the need for loading doses or additional oral risperidone. This approval is based on the positive outcomes of the pivotal PRISMA-3 study, which assessed the efficacy and safety of Risperidone ISM in patients with schizophrenia. The study's findings demonstrate that the two doses tested (75 mg and 100 mg administered once monthly) successfully met the predetermined primary and secondary efficacy endpoints for treating patients with moderate to severe schizophrenia symptoms. Furthermore, OKEDI (risperidone ISM) was launched in Germany, the UK, and Spain in 2022. The approval was based on the positive results of the pivotal PRISMA-3 study on the efficacy and safety of risperidone ISM in schizophrenia patients. As per the estimates by Delveinsight analysts, this drug has blockbuster potential in the schizophrenia market with medium-fast uptake. To know more about schizophrenia treatment options, visit @ New Treatment for Schizophrenia The schizophrenia market is crowded with so many companies working in the domain. Various potential therapies that are projected to enter during the forecast period include Boehringer Ingelheim's Iclepertin (BI-425809), Sumitomo Pharma/Otsuka's Ulotaront (SEP-363856), Reviva Pharmaceuticals' Brilaroxazine (RP5063), Minerva Neurosciences/Mitsubishi Tanabe Pharma's Roluperidone (MIN-101/MT-210), Karuna Therapeutics (Bristol Myers Squibb)/Royalty Pharma's KarXT (xanomeline-trospium), ACADIA Pharmaceuticals' NUPLAZID (pimavanserin), Teva Pharmaceutical/MedinCell/Royalty Pharma's Olanzapine LAI (mdc-TJK/TV-44749), Neurocrine Biosciences/Mitsubishi Tanabe Pharma's Valbenazine (NBI-98854), Lyndra Therapeutics' LYN-005 (long-acting oral risperidone), Newron Pharmaceuticals' Evenamide (NW-3509), Luye Pharma's LY03010 (ER paliperidone palmitate), and others. Discover which therapies are expected to grab major schizophrenia market share @ Schizophrenia Market Report Iclepertin (BI 425809) is an investigational drug and a glycine transporter 1 (GlyT1) inhibitor aimed at addressing the brain biology related to cognitive symptoms in schizophrenia (CIAS). Abnormalities in glutamatergic signaling downstream of neuronal NMDA receptors contribute to cognitive impairment in this condition. Enhancing NMDA receptor function could potentially improve synaptic plasticity and cognition. Glycine, an essential NMDA receptor co-agonist, is modulated by glycine transporters GlyT-1 and GlyT-2, which are located in presynaptic and astrocyte membranes. These transporters regulate glycine levels by taking it up into nerve terminals and adjacent glial cells, affecting synaptic cleft glycine concentration. By inhibiting these transporters, BI 425809 aims to increase glycine levels, thereby modulating NMDA receptor function. Administered orally, this potent and selective GlyT1 inhibitor is also under investigation for Alzheimer's disease. However, early 2020 Phase II trial results did not support its continued development for this indication, and the focus has now shifted to schizophrenia, with multiple Phase III trials currently underway. Since schizophrenia, patients frequently ignore certain symptoms, such as cognitive impairment. Therefore, if approved, BI-425809 will be the first approved pharmacotherapy for the treatment of CIAS. Ulotaront (SEP-363856), developed by Sumitomo Pharma and Otsuka Pharmaceuticals, is a trace amine-associated receptor 1 (TAAR1) agonist with serotonin 5-HT1A agonist activity. It is a small-molecule oral agent that does not bind to dopamine D2 or serotonin 5-HT2A receptors. Sunovion discovered Ulotaront in collaboration with PsychoGenics using the in vivo phenotypic SmartCube platform and associated artificial intelligence algorithms. Ulotaront is currently in late-stage development for schizophrenia and generalized anxiety disorder (GAD) in Japan and as an adjunctive treatment for major depressive disorder, schizophrenia, and GAD in the US. Top-line results from Phase III DIAMOND 1 and DIAMOND 2 trials in schizophrenia have been released, but the Phase III DIAMOND 3 trial in the US has been halted. Sumitomo Pharma has also stopped a Phase III trial for schizophrenia in Japan and another Phase II/III trial in China, Japan, Taiwan, and the Philippines, citing business strategy reasons. Despite this, two Phase III trials are still ongoing. Recently, Otsuka and Sumitomo revised their licensing agreement, excluding SEP-4199 and SEP-378614. Under this revision, Otsuka gained exclusive rights from SMPA to develop, manufacture, and commercialize ulotaront worldwide. If Otsuka successfully develops and commercializes ulotaront, they will pay up to 30 million USD (approximately 4.5 billion yen) in milestones, along with royalties based on sales, without any upfront payment. Starting January 2024, Otsuka will cover the full cost of studies conducted by both Sumitomo Pharma Group and Otsuka, except for certain studies. KarXT (xanomeline–trospium) is an oral modulator targeting muscarinic receptors found in both the central nervous system (CNS) and various peripheral tissues. This proprietary product combines xanomeline, a novel muscarinic agonist, with trospium, an approved muscarinic antagonist, to preferentially stimulate muscarinic receptors in the CNS. As a dual M1/M4 muscarinic acetylcholine receptor agonist, KarXT aims to improve the positive, negative, and cognitive symptoms of schizophrenia. The US FDA has accepted Karuna Therapeutics' NDA for KarXT for adult schizophrenia treatment, with a PDUFA date of September 26, 2024. Additionally, KarXT is undergoing multiple Phase III trials as an adjunctive treatment with standard-of-care agents for schizophrenia and is being evaluated for psychosis treatment in Alzheimer's disease patients. Following Bristol Myers Squibb's acquisition of Karuna Therapeutics, Karuna is now a wholly-owned subsidiary of BMS. At the Annual Congress of the Schizophrenia International Research Society (SIRS) in April 2024 in Italy, BMS presented promising results from the latest interim analysis of the Phase III EMERGENT-4 open-label extension trial, showing KarXT's efficacy in reducing symptoms in adults with schizophrenia. BMS plans to continue discussions with the US FDA and will release more data from the EMERGENT program later this year. Discover more about drugs for schizophrenia in development @ Schizophrenia Clinical Trials The anticipated launch of these emerging therapies for schizophrenia are poised to transform the market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the schizophrenia market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. DelveInsight estimates that the market size for schizophrenia is expected to grow from USD 2.6 billion in 2023 with a significant CAGR by 2034. This growth can be attributed to the introduction of upcoming therapies and the rising prevalence of the disease. The anticipated launch of these therapies is also expected to attract new entrants to the Schizophrenia market, resulting in increased competition and innovation. DelveInsight's latest published market report titled as Schizophrenia Market Insight, Epidemiology, and Market Forecast – 2034 will help you to discover which market leader is going to capture the largest market share. The report provides comprehensive insights into the schizophrenia country-specific treatment guidelines, patient pool analysis, and epidemiology forecast to help understand the key opportunities and assess the market's underlying potential. The schizophrenia market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Total Prevalent Cases of Schizophrenia Total Diagnosed Prevalent Cases of Schizophrenia Gender-specific Diagnosed Prevalent Cases of Schizophrenia Severity-specific Diagnosed Prevalent Cases of Schizophrenia The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM schizophrenia market. Highlights include: 11-year Forecast 7MM Analysis Epidemiology-based Market Forecasting Historical and Forecasted Market Analysis upto 2034 Emerging Drug Market Uptake Peak Sales Analysis Key Cross Competition Analysis Industry Expert's Opinion Access and Reimbursement Download this schizophrenia market report to assess the epidemiology forecasts, understand the patient journeys, know KOLs' opinions about the upcoming treatment paradigms, and determine the factors contributing to the shift in the schizophrenia market. Also, stay abreast of the mitigating factors to improve your market position in the schizophrenia therapeutic space. Related Reports Schizophrenia Pipeline Schizophrenia Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key schizophrenia companies, including Otsuka Pharmaceutical Development & Commercialization, Inc., Gedeon Richter Ltd., Lyndra Therapeutics, Neurocrine Bioscience, Takeda Pharmaceutical Company, Memory Pharmaceuticals, Hoffmann-La Roche, Neurocrine Biosciences, Boehringer Ingelheim, Meiji Seika Pharma Co., Ltd., SK Life Science, Inc., Alkermes, Inc. , Solvay Pharmaceuticals, Mitsubishi Tanabe Pharma Corporation, SyneuRx International (Taiwan) Corp, BioXcel Therapeutics Inc, Cognitive Research Corporation, Amarex Clinical Research, Forum Pharmaceuticals Inc, Amneal Pharmaceuticals, LLC, Recognify Life Sciences, Accutest Research Laboratories (I) Pvt. Ltd., Eli Lilly and Company, Johnson & Johnson Pharmaceutical Research & Development, L.L.C, Janssen, LP, Delpor, Inc., Jiangsu Hansoh Pharmaceutical, Cerevel Therapeutics, LLC, Zogenix, Inc., Luye Pharma, Reviva Pharmaceuticals, Alkermes, Inc., Karuna Therapeutics, Autifony Therapeutics, Neumora Therapeutics, Adex Therapeutics, among others. Schizophrenia Epidemiology Forecast Schizophrenia Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, and schizophrenia epidemiology trends. Dementia Market Dementia Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key dementia companies, including Hoffmann-La Roche, Forest Laboratories, Inc., Janssen Pharmaceuticals, Inc., Merck and Co., Inc., Novartis, among others. Bipolar Depression Market Bipolar Depression Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key bipolar depression companies, including Intra-Cellular Therapies, Sunovion Pharmaceuticals, COMPASS Pathways, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP

Phase 3Phase 2Clinical ResultDrug ApprovalLicense out/in

100 Deals associated with Haloperidol

External Link

KEGG	Wiki	ATC	Drug Bank
D00136	Haloperidol	N05AD01	DB00502

R&D Status

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Psychotic Disorders	US	Ortho-McNeil Pharmaceutical LLC	12 Apr 1967
Tourette Syndrome	US	Ortho-McNeil Pharmaceutical LLC	12 Apr 1967
Bipolar Disorder	JP	Sumitomo Pharma Co., Ltd.	24 Oct 1964
Schizophrenia	JP	Sumitomo Pharma Co., Ltd.	24 Oct 1964

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT01123408 \| NCT01164059 \| NCT00485901 \| NCT00534183 \| NCT01052389 \| NCT01193166 \| NCT00191555 \| NCT00419653 (Pubmed \| Cochrane Database Syst Rev)	Not Applicable	Schizophrenia	9,132	Haloperidol (oral)	fwbhfblikb(vesbvektpa): RR = 3.38 (95% CI, 2.28 - 5.02) View more	-	03 Jul 2024
				Olanzapine (oral)
NCT03392376 (AID-ICU, Pubmed \| Intensive Care Med) Manual	Phase 4	Delirium	1,000	Haloperidol	upgyyzslnc(ysyqspxovk): Difference (Mean) = 0.04 (95% CI, - 0.03 - 0.11), P-Value = 0.091 View more	Positive	01 Jan 2024
				Placebo
NCT03628391 (EuRIDICE, Pubmed \| Crit Care)	Phase 3	Delirium	132	Haloperidol 2.5 mg	dpazxrfxxb(ewzlmsjuxc): RR = 0.98 (95% CI, 0.73 - 1.31), P-Value = 0.87 View more	Negative	30 Oct 2023
				Placebo
NCT03021486 (CTgov)	Phase 2/3	Locally Advanced Malignant Neoplasm \| Delirium \| Recurrent Carcinoma ... View more	70	haloperidol (Escalation Group)	xpqeummlpr(txchcvbznl) = feihbbozpp wmqligpzcg (enoghtnasq, edolqegcgw - aslssobbwl) View more	-	01 Sep 2022
				chlorpromazine (Rotation Group)	xpqeummlpr(txchcvbznl) = nqroomnmas wmqligpzcg (enoghtnasq, xpcatmrxgp - zdbednhqma) View more
NCT02199743 (CTgov)	Phase 4	Schizoaffective disorder \| Schizophrenia	35	Perphenazine+Lurasidone+Haloperidol	xfrafwpeze(vkehczarjg) = ujjbccjwhl itlafnapba (dpwcejxonm, fzmdeurhhr - lbeymtikqn) View more	-	01 Mar 2021
NCT03056482 (Pubmed \| Ann Emerg Med)	Phase 4	syndrome; salt-losing, adrenogenital syndrome	33	Haloperidol	ppfypbafzg(jjdwgemwrq) = heybporipw qtvnupcffd (ushdbjsrho )	-	05 Nov 2020
				Ondansetron	ppfypbafzg(jjdwgemwrq) = lzetvwfhpf qtvnupcffd (ushdbjsrho )
NCT01639599 (CTgov)	Not Applicable	Postoperative Nausea and Vomiting \| Vomiting \| Genital Diseases, Female	150	Dexamethasone iv injection (Dexamethasone)	wyhxfnjmym(jwmigjsjol) = jomtldxwun nhbtwcvrjg (lmaiolbaag, detpjgvbby - yqysiddtkf) View more	-	24 Aug 2020
				Dexamethasone, haloperidol (Dexamethasone, Haloperiol 1mg)	wyhxfnjmym(jwmigjsjol) = dnopkkvhtw nhbtwcvrjg (lmaiolbaag, oqphjhpnpf - cgqmmopdox) View more
NCT02747511 (Pubmed \| J Emerg Med)	Phase 4	Headache	118	Haloperidol 2.5 mg i.v.	owwzzsdknf(nzxfcrcfiq) = gqxcffiela mukrcfkcew (ofuhawbccf )	Positive	10 May 2020
				Placebo	owwzzsdknf(nzxfcrcfiq) = jbvuwboljb mukrcfkcew (ofuhawbccf )
NCT01539733 (Pubmed \| Oncologist) Manual	Phase 3	Delirium \| Advanced cancer	98	Olanzapine	brmcschxex(afgvddyvhy) = vplcbmzuyn becyzvdmbk (jsdbpbsnvi, 31 - 59) View more	Non-superior	01 Mar 2020
				Haloperidol	brmcschxex(afgvddyvhy) = tdihfcvned becyzvdmbk (jsdbpbsnvi, 43 - 71) View more
NCT01211522 (MIND-USA, CTgov)	Phase 3	Cognitive Dysfunction \| Stress, Psychological \| Delirium ... View more	566	Haloperidol (Haloperidol)	zehhvhkkjk(akqwqscxip) = eygmrnofid xlrdcidroq (gojjxsnoej, ejdqmiapeo - gxmqksamdg) View more	-	20 Aug 2019
				Ziprasidone (Ziprasidone)	zehhvhkkjk(akqwqscxip) = cxqwstjgla xlrdcidroq (gojjxsnoej, nthnlicdzj - blejngfyyu) View more

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