This is a single dose, placebo-controlled study. Male subjects aged 18 to 40 years (inclusive) with a diagnosis of FXS. Eligible subjects may enroll in this study comprised of two in home and two in clinic visits each 14 days apart, for a total of four visits. Subjects will be given single dose gaboxadol (10 mg) or matched placebo at each of these visits to take orally. Thus, all enrolled subjects will receive placebo at home and in clinic and receive gaboxadol at home and in clinic in a blinded fashion.

Start Date29 Jan 2024

Sponsor / Collaborator

Children's Hospital & Medical Center

[+1]

NCT04823052

/ WithdrawnPhase 2

A Phase 2, Placebo-controlled, Adaptive Design Study to Explore the Safety and Efficacy of Sulindac (HLX-0201) and Gaboxadol (HLX-0206) and Possible Other Treatments in Adolescent and Adult Males With Fragile X Syndrome (FXS)

This study is to investigate the safety, tolerability and efficacy of Sulindac (HLX-0201) and Gaboxadol (HLX-0206) in males with Fragile X Syndrome (FXS) with confirmed full FMR1 mutation treated over a 10 week period in an outpatient setting.

Start Date25 May 2022

Sponsor / Collaborator

Healx Ltd.

NCT04106557

/ CompletedPhase 3

A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Phase 3 Study to Evaluate the Efficacy and Safety of OV101 in Pediatric Individuals With Angelman Syndrome

The purpose of this study is to assess the efficacy and safety of oral OV101 (gaboxadol) in pediatric subjects with Angelman syndrome.

Start Date09 Sep 2019

Sponsor / Collaborator

Ovid Therapeutics, Inc.

100 Clinical Results associated with Gaboxadol

100 Translational Medicine associated with Gaboxadol

100 Patents (Medical) associated with Gaboxadol

876

Literatures (Medical) associated with Gaboxadol

01 Jul 2025·PAIN

Molecular and cellular targets of GABAergic anesthetics in the mesopontine tegmentum that enable pain-free surgery

Article

Author: Baron, Mark ; Minert, Anne ; Ibraheem, Angham ; Devor, Marshall ; Vaso, Kristina

Abstract:

The mesopontine tegmental anesthesia area (MPTA) is a focal brainstem locus which, when exposed to GABAergic agents, induces brain-state transitioning from wakefulness to unconsciousness. Correspondingly, MPTA lesions render animals relatively insensitive to GABAergic anesthetics delivered systemically. Using chemogenetics, we recently identified a neuronal subpopulation within the MPTA whose excitation induces this same pro-anesthetic effect. However, very few of these “effector-neurons” express synaptic γ2-containing GABAA receptor isoforms and none express extrasynaptic δ-subunit containing receptors, suggesting that they are not the direct cellular target of GABAergic agents. Here we used pharmacological tools in rats to define the molecular target(s) of GABAergics in the MPTA. GABA microinjected into the MPTA at nanomolar concentrations, selective for GABAAδ-Rs, proved to be pro-anesthetic as was blocking GABA reuptake. Likewise, low-concentration gaboxadol/THIP, also selective for GABAAδ-Rs, was effective, whereas benzodiazepines and zolpidem, which selectively target GABAAγ2-Rs, were not. The GABAergic anesthetics pentobarbital and propofol proved pro-anesthetic when applied to the MPTA at the low concentrations present in the brain after systemic dosing. Glycinergic agonists which are inhibitory, but infective on GABAAδ-Rs, and other non-GABAergic agonists tested, were at most only marginally effective. We conclude that GABAAδ-Rs are the primary molecular target of GABAergic anesthetics in the MPTA. Immunolabeling revealed that this GABAA-R isoform is expressed exclusively by a distinct subpopulation of MPTA “δ-cells” that reside in close apposition to effector neurons. This suggests that during wakefulness, δ-cells serve as inhibitory interneurons which, when silenced by GABAergic agents, disinhibit (excite) the effector-neurons, triggering transition to unconsciousness.

01 Jul 2025·PEDIATRIC NEUROLOGY

Current and Emerging Precision Therapies for Developmental and Epileptic Encephalopathies

Review

Author: Keator, Cynthia G ; Nolan, Danielle ; Xiong, Katherine ; Sandoval Karamian, Amanda G ; Bhatia, Sonal ; Schreiber, John M ; Sattar, Shifteh ; Rao, Chethan K ; Samanta, Debopam ; Reeders, Puck C ; Axeen, Erika ; Hunter, Senyene E ; Karakas, Cemal ; Erdemir, Gozde ; Fine, Anthony L

Developmental and epileptic encephalopathies (DEEs) are severe neurological disorders characterized by childhood-onset seizures and significant developmental impairments. Seizures are often refractory to treatment with traditional antiseizure medications, which fail to address the underlying genetic and molecular mechanisms. This comprehensive review explores the evolving landscape of precision therapeutics for DEEs, focusing on mechanism-driven interventions across key pathophysiologic categories. Targeted approaches for channelopathies include antisense oligonucleotides and gene therapies, such as zorevunersen and ETX101 for SCN1A-related Dravet syndrome, alongside novel small molecules for other ion channel disorders. Advances in targeting neurotransmitter receptor dysfunctions, including γ-aminobutyric acid and glutamate receptor variants, highlight the use of modulators such as gaboxadol, radiprodil, and l-serine, alongside emerging gene therapies. For synaptic dysfunctions, innovative treatments such as chemical chaperones for STXBP1-related disorders and Ras-Raf-MEK-ERK inhibitors for SYNGAP1 pathologies are discussed. The review also examines precision interventions targeting cellular signaling pathways in tuberous sclerosis complex, epigenetic regulation in Rett syndrome, and metabolic interventions like ketogenic diets and targeted supplementation for specific genetic etiologies. Additionally, the importance of enhancing access to genetic testing, conducting robust natural history studies, and employing innovative clinical trial designs is emphasized. Future directions focus on addressing the challenges in developing and implementing gene-based therapies, integrating systems biology, leveraging artificial intelligence for data analysis, and fostering collaboration among stakeholders. The rapidly advancing field of precision therapeutics for DEEs holds promise to improve outcomes through tailored, equitable, and patient-centered care.

01 Apr 2025·JOURNAL OF NEUROPHYSIOLOGY

δ-Containing GABA_A receptors on parvalbumin interneurons modulate neuronal excitability and network dynamics in the mouse medial prefrontal cortex

Article

Author: Lambert, Peter M ; Mennerick, Steven ; Zorumski, Charles F ; Benz, Ann ; Shu, Hong-Jin ; Lu, Xinguo

We reveal the critical role of δ-containing GABAA receptors in parvalbumin interneurons in the medial prefrontal cortex, important for human neuropsychiatric disorders. We demonstrate these receptors’ importance in regulating neuronal excitability and network dynamics. δ-containing receptors act as a brake on interneuron activity, maintaining the excitation-inhibition balance in cortical circuits. Our findings provide insights into how disruptions in inhibitory signaling alter network function through a receptor subtype that is a target of neurotherapeutics.

News (Medical) associated with Gaboxadol

18 Mar 2026

·allsci.com

Ovid Therapeutics raises USD 60m in private placement

Ovid Therapeutics Inc. (Nasdaq: OVID), a New York-based biopharmaceutical company developing small molecule therapies for brain disorders, announced a USD 60 million private placement to fund the expansion of its lead clinical program, OV329, into additional neurological indications. The PIPE financing, expected to close on or about March 19, 2026, will support development of OV329 in tuberous sclerosis complex and infantile spasms, alongside general research and development activities. The company intends to combine the net proceeds with existing cash, cash equivalents, and marketable securities. Under the terms of the securities purchase agreement, Ovid is selling 19,154,321 shares of common stock at USD 2.01 per share and pre-funded warrants to purchase up to 10,701,710 additional shares at a purchase price of USD 2.009 per pre-funded warrant. The pre-funded warrants carry an exercise price of USD 0.001 per share and are immediately exercisable. Point72 led the financing, with participation from existing investors including Adage Capital Management, ADAR1 Capital Management, Affinity Asset Advisors, Ally Bridge Group, Balyasny Asset Management, Coastlands Capital, Eventide Asset Management, Janus Henderson Investors, and RA Capital Management. Leerink Partners served as lead placement agent, with Oppenheimer & Co. and LifeSci Capital acting as co-placement agents. Company overview and pipeline Ovid Therapeutics is focused on developing targeted small molecule candidates that modulate factors involved in neuronal hyperexcitability across multiple neurological and neuropsychiatric disorders. The company’s lead asset, OV329, is a next-generation GABA-aminotransferase inhibitor being developed as a potential therapy for treatment-resistant focal onset seizures and developmental and epileptic encephalopathies, including tuberous sclerosis complex and infantile spasms. The press release did not specify the current clinical phase of OV329 or provide a timeline for upcoming milestones in these new indications. Ovid’s broader pipeline includes OV4071 and a library of related compounds designed to directly activate the KCC2 transporter, targeting multiple central nervous system disorders. The company has previously advanced OV101 (gaboxadol) through Phase III testing in Angelman syndrome, with a completed randomized, double-blind study enrolling 104 patients and an open-label extension study (ELARA) enrolling 170 patients. OV101 was also evaluated in a completed Phase II trial in Fragile X syndrome. In July 2020, Ovid licensed exclusive European rights to OV101 for Angelman syndrome to Angelini Pharma. On the business development side, Ovid has undergone a period of portfolio restructuring. In June 2024, Takeda secured global development and commercialization rights to soticlestat (TAK-935), a cholesterol 24-hydroxylase inhibitor previously co-developed with Ovid for Dravet syndrome and Lennox-Gastaut syndrome. In June 2025, Ovid sold 100% of its ganaxolone royalty rights to Immedica Pharma AB for USD 7 million in cash. The company also holds a collaboration with Graviton Bioscience, established in May 2023, focused on developing brain-penetrant ROCK2 inhibitors for rare neurological diseases marked by seizures. Leave a Reply Cancel reply Your email address will not be published. Required fields are marked * Comment * Name * Email * Website

Phase 2Phase 3

12 Nov 2025

·endpoints.news

Exclusive: Ovid co-founder and CEO Levin plans leadership handoff in 2026

Ten years after founding Ovid Therapeutics, CEO Jeremy Levin plans to step down and hand over the top spot to COO Meg Alexander. Alexander will formally take the reins at the beginning of next year, the company told Endpoints News . Levin will lead the board as executive chairman. Equipped with new money and a package of neurology assets, Levin and Alexander said in a joint interview with Endpoints that the goal now is to reestablish a pipeline with best-in-class potential and to find future partners. “Everything we’re trying to do is essentially better, gentler medicines, and I think we’re on the verge of being able to prove to the world that we can do that,” Alexander said. Alexander joined Ovid as a vice president before being brought into the C-suite as the chief corporate affairs officer in February 2022, rising to strategy chief and then president and COO last year. Levin has worked in the biopharma sector for decades, ascending as a dealmaker at Novartis and Bristol Myers Squibb before becoming the president and CEO of Teva Pharmaceuticals in 2012. He’s also guided the wider industry at the trade group BIO, spending more than nine years in leadership roles, including chairing the group’s board during the pandemic. Levin helped launch Ovid in 2015, buying rights to an asset from Lundbeck and raising a $75 million Series B later that year. That asset, OV101, ultimately failed in a Phase 3 study in patients with Angelman syndrome. Ovid also bought the rights to seizure drug soticlestat from Takeda and developed it through Phase 2 before handing those rights back to the Japanese pharma in 2021. The soticlestat deal earned Ovid almost $200 million through an upfront payment. But the drug failed two Phase 3 trials in 2024 , albeit narrowly in one of the studies. In the executive chairman role, Levin will be around to help win more deals, which he says are very much in play, specifically for the KCC2 programs that Ovid acquired from AstraZeneca for $12.5 million in upfront cash and stock in January 2022. Ovid is competing with private biotech Axonis Therapeutics, which raised $115 million in 2024 to advance a KCC2-focused pipeline. “We are on the cusp now of demonstrating categorically that these are an open sesame to a portion of what goes on in the brain with hyper-excitability,” Levin said. “But that portion is a huge opportunity.” A GABA-aminotransferase inhibitor drug is running slightly ahead of the KCC2 programs. Phase 1 safety and biomarker data from healthy volunteers helped propel a PIPE financing earlier this year that brought in $81 million upfront and $94 million more in potential warrants, enough to last into the second half of 2028, according to the company’s latest earnings. When asked why he didn’t leave the company altogether as part of this transition, Levin said he trusted the partnership with Alexander and their ability to work together. “I don’t look over my shoulder, Meg doesn’t have to look over her shoulder,” Levin said. “We’ve worked together as a partnership here for a long time.”

Executive ChangePhase 1AcquisitionIPOPhase 3

15 Apr 2024

·firstwordpharma.com

Ultragenyx's Angelman syndrome hopeful shows early promise

Ultragenyx Pharmaceutical reported encouraging early data for its experimental Angelman syndrome treatment GTX-102, reinforcing the decision to buy out its development partner GeneTx Biotherapeutics for $75 million in 2022. However, despite the early success, investors may have been spooked by reports of serious adverse effects occurring in some patients, pushing Ultragenyx shares down by about 9% on Monday.The results, to be presented this week at the American Academy of Neurology (AAN) annual meeting, are from a Phase I/II study evaluating GTX-102 in 74 paediatric patients with a genetically confirmed diagnosis of full maternal UBE3A gene deletion.The antisense oligonucleotide, given by intrathecal injection, works by inhibiting expression of UBE3A-AS which in turn reactivates expression of the paternal UBE3A allele in central nervous system neurons. In preclinical models, this reactivation has been associated with improvements in some of the neurological symptoms that characterise Angelman syndrome, which include developmental delays, speech and motor disabilities, as well as sleep disturbances.The latest readout is drawn from 39 patients, including two expansion cohorts involving 24 subjects with data out to 170 days, and long-term data from dose-escalation cohorts comprising 15 patients with more than two years of data.Sustained long-term benefitAccording to Ultragenyx, results from the two expansion cohorts showed that GTX-102 led to "rapid and clinically meaningful improvement" across several domains, which included cognition, receptive communication, behaviour, gross motor function and sleep. The company also conducted a multi-domain responder index (MDRI) assessment that covers cognition, receptive communication, behaviour and sleep; GTX-102 resulted in a total net response of +2.0 on that scale.The dose-escalation cohorts showed long-term increasing and sustained clinical benefit "far exceeding" natural history data at day 758. Ultragenyx said there was a continuing long-term improvement with cognition, exceeding the threshold of clinical significance "by many-fold in many patients." Behaviour, sleep and gross motor function also showed sustained clinically meaningful benefits.Lower extremity weaknessWhile the company reported no unexpected serious adverse events, it did say three patients experienced mild-to-moderate lower extremity weakness considered to be related to GTX-102. "All resolved rapidly without sequelae and remain in the study without ongoing safety concerns," it added."Our next step is an end-of-Phase II meeting with the FDA and interactions with other health authorities to enable timely initiation of a Phase III pivotal study," stated chief medical officer Eric Crombez.Previous attempts by other developers to produce an effective therapy for Angelman syndrome have fallen flat. Ovid Therapeutics' lead candidate OV101 missed its primary endpoint in a Phase III study in 2020, and Roche last year scrapped its rugonersen (RO7248824) programme after it underperformed in early testing. Meanwhile, Biogen and Ionis Pharmaceuticals are partnered on BIIB121, an investigational antisense oligonucleotide treatment similar to GTX-102 that's currently in Phase I testing.

OligonucleotidePhase 3Phase 1Clinical ResultPhase 2

100 Deals associated with Gaboxadol

External Link

KEGG	Wiki	ATC	Drug Bank
D04282	Gaboxadol	-	DB06554

R&D Status

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Angelman Syndrome	Phase 3	United States	Ovid Therapeutics, Inc.	09 Sep 2019
Angelman Syndrome	Phase 3	Australia	Ovid Therapeutics, Inc.	09 Sep 2019
Angelman Syndrome	Phase 3	Germany	Ovid Therapeutics, Inc.	09 Sep 2019
Angelman Syndrome	Phase 3	Israel	Ovid Therapeutics, Inc.	09 Sep 2019
Angelman Syndrome	Phase 3	Netherlands	Ovid Therapeutics, Inc.	09 Sep 2019
Sleep Initiation and Maintenance Disorders	Phase 3	-	H. Lundbeck A/S	01 Jan 2003
Fragile X Syndrome	Phase 2	United States	Ovid Therapeutics, Inc.	17 Sep 2018
Depressive Disorder, Major	Phase 2	Austria	H. Lundbeck A/S	01 Nov 2008
Depressive Disorder, Major	Phase 2	Russia	H. Lundbeck A/S	01 Nov 2008
Epilepsy	Preclinical	United States	Ovid Therapeutics, Inc.	-

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03882918 (ELARA, CTgov)	Phase 3	Angelman Syndrome	141	OV101	toktfkvagu = pzbzdjnnkr lgazubwqbu (kgctdbdlfk, waglqljqng - ayjfedndrp) View more	-	18 Jul 2025
NCT03697161 (ROCKET, CTgov)	Phase 2	Fragile X Syndrome	36	OV101 (Gaboxadol) (OV101 (Gaboxadol) (5 mg QD))	skzxwenvwp = eeabbnelpa mgjbveroyg (ugpmrctzge, iwaaeiefrl - phxqxllguh) View more	-	23 Jan 2024
NCT03697161 (ROCKET, CTgov)	Phase 2	Fragile X Syndrome	36	OV101 (Gaboxadol) (OV101 (Gaboxadol) (5 mg BID))	skzxwenvwp = givlreraar mgjbveroyg (ugpmrctzge, ouypoluhgf - ugepasaikk) View more	-	23 Jan 2024
NCT02996305 (The STARS Phase 2 Study, CTgov)	Phase 2	Angelman Syndrome	88	Placebo	ghztcybwuk = yifrizpipq rwelhjxevn (wgvuxokavv, vfxhbpzyxy - biyoudnrlk) View more	-	31 Mar 2022
NCT03697161 (ROCKET, Pubmed \| Front Pharmacol) Manual	Phase 2	Fragile X Syndrome	23	OV101	fyoimsfmuj(ciomgzlcoh) = xmxrpmwsha ajrgwemgpy (ljraybpqtv ) View more	Positive	08 Oct 2021
NCT04106557 (NEPTUNE, GlobeNewswire) Manual	Phase 3	Angelman Syndrome	97	OV101	zqwhtwadnt(iqqbrezcjo) = mnmestbtln kkckrlksjg (rrpmndeflu )	Negative	01 Dec 2020
NCT04106557 (NEPTUNE, GlobeNewswire) Manual	Phase 3	Angelman Syndrome	97	Placebo	zqwhtwadnt(iqqbrezcjo) = zphuyqyokb kkckrlksjg (rrpmndeflu )	Negative	01 Dec 2020
STARS trial (AAN2020)	Phase 2	Angelman Syndrome	-	Gaboxadol	vbbusxhypa(gdnxtdcchz) = wsbdjubxsr pwfbptqkfe (styptyhadx ) View more	-	14 Apr 2020
Pubmed \| Int J Neuropsychopharmacol	Phase 2	Depressive Disorder, Major	490	Placebo	skttkoytpt(flarltmbfq) = The most common adverse events reported in the active treatment groups for which the incidence was higher than that in the placebo group, comprised anxiety ujkrrfnhun (evvhujmtgz ) View more	-	01 Jul 2012
Pubmed \| Int J Neuropsychopharmacol	Phase 2	Depressive Disorder, Major	490	Escitalopram 20 mg		-	01 Jul 2012
NCT00807248 (CTgov)	Phase 2	Depressive Disorder, Major	490	Placebo (Placebo (Orally, Once Daily))	bcfcgqmodq(thiflahtrj) = vycwvcdkbt zecykljyqw (bimypjkyyb, 1.1) View more	-	11 Aug 2011
NCT00807248 (CTgov)	Phase 2	Depressive Disorder, Major	490	Escitalopram+Placebo (Escitalopram 20 mg and Placebo (Orally, Once Daily))	bcfcgqmodq(thiflahtrj) = hjdqhquutu zecykljyqw (bimypjkyyb, 0.9) View more	-	11 Aug 2011
NCT00103818 \| NCT00095069 (0928-014, Pubmed)	Phase 3	Sleep Initiation and Maintenance Disorders	-	Gaboxadol 15 mg	mwrnjkwadf(nwjpyeloyh) = glyicuoxef ogwqzuqdbt (iecawqikkr ) View more	-	15 Feb 2010

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

AI Agents Built for Biopharma Breakthroughs

Accelerate discovery. Empower decisions. Transform outcomes.

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis