Epilepsy - Pipeline Insight, 2025

This “Epilepsy - Pipeline Insight, 2025” report provides comprehensive insights about 75+ companies and 90+ pipeline drugs in Epilepsy pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Epilepsy: Understanding

Epilepsy: Overview

Epilepsy is one of the most common serious brain conditions, affecting over 50 million people worldwide. Epilepsy is a neurological disorder that is characterized by an enduring predisposition to generate epileptic seizures and the associated cognitive, psychological, and social consequences. Its incidence has a bimodal distribution with the highest risk in infants and older age groups. An epileptic seizure is a transient behavioural change that might be objective signs or subjective symptoms (such as loss of awareness, stiffening, jerking, a sensation that rises from the abdomen to the chest, a smell of burnt rubber or déjà vu), caused by abnormal excessive or synchronous neuronal activity in the brain.

Epilepsy is marked by recurrent seizures that are unpredictable in frequency is a chronic non communicable disease of the brain. A seizure is usually defined as a sudden alteration of behavior due to a temporary change in the electrical functioning of the brain. Normally, the brain continuously generates tiny electrical impulses in an orderly pattern. These impulses travel along neurons - the network of nerve cells in the brain - and throughout the whole body via chemical messengers called neurotransmitters. Seizure episodes are a result of excessive electrical discharges in a group of brain cells.

The pathophysiology of epilepsy involves conversion of a normal network into a hyper excitable network. It is associated with a group of processes which disturb extracellular ion homeostasis, alter energy metabolism, change receptor function and alter transmitter uptake.

In CNS, the brain consists of nerve cells and these nerve cells communicate and interact with each other through axons by discharging tiny electrical impulses. The brain along with nerve cells works on the phenomenon of electricity. The output of these electrical impulses is the release of chemicals called neurotransmitters from the axon end which in turn interacts with the next cell. These chemicals (neurotransmitters) can be excitatory or inhibitory. The balance of these excitatory and inhibitory impulses is very important to maintain the action potential of neurons. Release of excessive excitatory glutamate overactivates NMDA receptors resulting in excessive influx of Ca2+ ions. The overflow of Ca2+ levels caused deteriorate condition which activates cytoplasmic proteases (such as calpain I), which proteolysis cytoskeletal and other proteins, neuronal nitric oxide synthase (nNOS), which increases nitric oxide production in turn generating the free radical peroxynitrite that damages DNA which ultimately lead to neuronal cell death.

Accurate diagnosis of the type of epilepsy a person has is crucial for finding an effective treatment. There are many different ways to successfully control seizures. Drugs used to treat epilepsy work by decreasing the electrical activity of the brain, either by preventing neuronal depolarization by blocking sodium channels or calcium channels, enhancing potassium channel function, inhibiting excitation mediated by the neurotransmitter glutamate, or promoting inhibition mediated by GABA.

A neurologist’s decision to begin one seizure medication over another is based on seizure type, age, other medical conditions, and potential side-effect profile. As a general principle, medication should be started at a low dose to avoid side effects. Dose increases can be performed at regular intervals if needed. The goal is to control seizures with the lowest dose. When a first drug fails, most clinicians will choose to add on a second drug, later deciding whether or not to withdraw the initial medication. Because of the mechanism of action of drugs, all seizure medications have CNS side effects. For example, sleepiness is a common side effect of almost all AEDs. There are many different treatment that can prevent or stop seizures.

'Epilepsy- Pipeline Insight, 2025' report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Epilepsy pipeline landscape is provided which includes the disease overview and Epilepsy treatment guidelines. The assessment part of the report embraces, in depth Epilepsy commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Epilepsy collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Epilepsy R&D. The therapies under development are focused on novel approaches to treat/improve Epilepsy.

Epilepsy Emerging Drugs Chapters

This segment of the Epilepsy report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Epilepsy Emerging Drugs

EPX-100: Harmony Biosciences

EPX-100 is a potential new treatment being for people with Dravet syndrome and Lennox-Gastaut Syndrome. EPX-100 is a repurposed antihistamine, which was used in the 1950s and 1960s to treat itchiness. The medication is thought to be able to suppress seizures through its action on the serotonin signaling pathways, a mechanism that is different from its anti-histaminic properties. Serotonin is a chemical messenger present in many parts of the brain. Scientists think that people with Dravet syndrome and Lennox-Gastaut Syndrome may have alterations in the serotonin signaling pathway, but the exact problems are not known. Likewise, precisely how EPX-100 affects serotonin systems in the brain to reduce seizures is still unclear. According to company’s pipeline the drug is in the Phase III stage of its development for the treatment of Dravet syndrome and is in the Phase II stage of its development for the treatment of Lennox-Gastaut Syndrome.

BMB-101: Bright Minds Biosciences

BMB-101, a 5-HT2C selective and biased agonist, has demonstrated compelling activity in a host of in-vitro and in-vivo non-clinical tests. Compared to Locaserin, BMB-101 exhibits strong Gq signaling coupled with minimal beta-arrestin recruitment. Mechanistically, Serotonin (5- Hydroxytryptamine, 5-HT) is a monoamine neurotransmitter widely expressed in the central nervous system, and drugs modulating 5-HT have made a major impact in mental health disorders. Central 5-HT systems have long been associated with the control of ingestive behavior and the modulation of behavioral effects of psychostimulants, opioids, alcohol and nicotine. Results of clinical trials and animal studies indicate that 5-HT2C up receptor agonists may have therapeutic potential in the treatment of addiction by decreasing the intake of opioids as well as impulsive behavior that can escalate compulsive drug use. Currently the drug is in Phase II stage of Clinical trial evaluation for the treatment of Epilepsy.

STK-001: Stoke Therapeutics

STK-001 is an investigational new medicine for the treatment of Dravet syndrome currently being evaluated in ongoing clinical trials. Stoke believes that STK-001, a proprietary antisense oligonucleotide (ASO), has the potential to be the first disease-modifying therapy to address the genetic cause of Dravet syndrome. STK-001 is designed to upregulate NaV1.1 protein expression by leveraging the non-mutant (wild-type) copy of the SCN1A gene to restore physiological NaV1.1 levels, thereby reducing both occurrence of seizures and significant non-seizure comorbidities. Currently the drug is in Phase II stage of Clinical trial evaluation for the treatment of Dravet Syndrome.

NRTX-1001: Neurona Therapeutics

NRTX-1001 inhibitory neurons are targeted to the seizure-onset zone to enhance GABAergic inhibition, reduce seizure activity, and repair the affected neural network. NRTX-1001 is delivered as a single, one-time dose and is intended to persist and provide long-term seizure suppression. In preclinical studies, NRTX-1001 has provided seizure-freedom to the majority of the cell treatment group and has not shown signs of dose-limiting toxicity. The preclinical safety and efficacy data have led to an FDA-cleared Phase I/II clinical trial of NRTX-1001 for drug-resistant temporal lobe epilepsy.

IAMA-6: IAMA Therapeutics

IAMA-6 is an orally administered small molecule therapeutic designed to directly target and inhibit NKCC1-associated neuronal hyperexcitability. The elevated activity of NKCC1 is implicated in various pathological conditions, underscoring the significant potential of NKCC1 inhibition in the treatment of both idiopathic and secondary forms of autism (autism spectrum disorder, or ASD), refractory epilepsy, Dravet Syndrome, and other neurological disorders. With the capability to be applied across multiple central nervous system (CNS) indications, IAMA-6 has demonstrated its safety and favorable tolerability in pre-clinical studies. Currently the drug is in Phase I stage of Clinical trial evaluation for the treatment of Epilepsy.

Epilepsy: Therapeutic Assessment

This segment of the report provides insights about the different Epilepsy drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Epilepsy

• There are approx. 75+ key companies which are developing the therapies for Epilepsy. The companies which have their Epilepsy drug candidates in the most advanced stage, i.e. phase III include, Harmony Biosciences.

Phases

The report covers around 90+ products under different phases of clinical development like

• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

Route of Administration

Epilepsy pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical

Molecule Type

Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Epilepsy: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Epilepsy therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Epilepsy drugs.

Epilepsy Report Insights

• Epilepsy Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Epilepsy Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Epilepsy drugs?
• How many Epilepsy drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Epilepsy?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Epilepsy therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Epilepsy and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• UCB
• SK Life Science
• Stoke Therapeutics
• Xenon Pharmaceuticals
• Epygenix
• Bright Minds Biosciences
• Neurona Therapeutics
• Cevevel Therapeutics
• PTC Therapeutics
• Addex Pharmaceuticals
• Equilibre Biopharmaceuticals B.V.

ES Therapeutics Australia Pty Ltd

• Overseas Pharmaceuticals
• Anavex Life Sciences
• Eliem Therapeutics
• Ovid Therapeutics
• CAMP4 Therapeutics
• LifeSplice
• Virpax Pharmaceuticals
• Neuroene Therapeutics

Key Products

• ZX008
• Cenobamate
• XEN496
• XEN1101
• STK-001
• EPX-100
• CVL-865
• BMB-101
• NRTX 1001
• Ataluren
• JNJ-40411813
• EQU-001
• ES-481
• Lacosamide extended release
• ANAVEX 2-73
• ETX-155
• OV329
• CMP-SCN
• LSP-SCN8
• VRP324
• NT102

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