11 neuroscience biotech companies you should know about

Developing treatments for neurological diseases presents a huge challenge for neuroscience companies within the biotech industry. The central nervous system (CNS) is extremely complex, and many neurological diseases can be difficult to treat due to the limited regenerative capacity of the CNS, and the fact that the blood-brain barrier – a specialized system of cells that blocks harmful substances from entering the brain – prevents most drugs from reaching the brain tissue. These challenges have even led to big pharma companies shutting down their neuroscience drug development programs. 

Fortunately, plenty of biotechs have kept research in this area alive, as they attempt to take on the challenge of developing treatments for a variety of neurological disorders. In this article, we look at 11 of the top neuroscience companies around today.

Alzheon

• Technology: Small molecule inhibitor
• Lead neuroscience candidate: Valiltramiprosate (ALZ-801) for Alzheimer’s disease
• Recent news: Concluded that valiltramiprosate may still be helpful for patients in the early stages of mild cognitive impairment

Neuroscience company Alzheon is focused on developing the first oral treatment with the potential to slow or stop the progression of Alzheimer’s disease. Its candidate is called valiltramiprosate (ALZ-801) and is currently in phase 3 of development. Additionally, it received fast track designation from the U.S. Food and Drug Administration (FDA) in 2017 for the treatment of Alzheimer’s disease.

Valiltramiprosate is designed to block the formation of neurotoxic soluble beta-amyloid oligomers in the brain associated with the onset and progression of cognitive decline in Alzheimer’s patients. In mechanism of action studies, the drug was shown to fully inhibit the formation of neurotoxic soluble beta-amyloid oligomers. 

Although Alzheon announced in April that a pivotal phase 3 trial of valiltramiprosate failed to meet its primary endpoint of slowing cognitive decline in patients with early Alzheimer’s who carry two copies of the APOE4 gene – constituting approximately 15% of Alzheimer’s patients – the company concluded that the candidate may still be helpful for patients in the early stages of mild cognitive impairment, as the drug was found to be most effective for this subgroup of patients within the trial. 

In June 2024, Alzheon raised $100 million in series E financing to help advance the development and commercialization of ALZ-801. 

Annovis Bio

• Technology: Small molecule inhibitor
• Lead neuroscience candidate: Buntanetap for Alzheimer’s, Parkinson’s, and Lewy Body Dementia
• Recent news: Announced that all 84 sites for its phase 3 study of buntanetap in early Alzheimer’s disease are now fully activated

Focused on developing therapies for neurodegenerative disorders such as Alzheimer’s disease and Parkinson’s disease, Annovis’ mission is to develop effective treatments for some of the most challenging neurological conditions by restoring nerve cell function and improving cognitive and motor abilities in patients.

The company’s lead candidate is called buntanetap. It is an orally-administered small molecule inhibitor of several neurotoxic proteins, and is being investigated in the treatment of Alzheimer’s disease, Parkinson’s disease, and Lewy Body Dementia. The Alzheimer’s program is currently furthest ahead, as Annovis announced earlier this month that all 84 sites for its pivotal phase 3 study in early Alzheimer’s disease are now fully activated and enrolling participants, with the majority already treating patients across the U.S. Additionally, the first group of patients has completed the 6-month treatment period, meaning that the company is on track to deliver symptomatic data from the trial in the second half of 2026. 

In October 2025, the neuroscience company closed a $6 million registered direct offering and said it would use the proceeds from this toward working capital and general corporate purposes.

AviadoBio

• Technology: Gene therapy
• Lead neuroscience candidate: AVB-101 for FTD
• Recent news: Announced the completion of the second dose cohort in a phase 1/2 trial of AVB-101

AviadoBio is working on developing and delivering transformative gene therapies for patients living with debilitating and life-threatening neurodegenerative disorders, such as frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS), with the aim of slowing, arresting, preventing, and potentially reversing these types of disorders. 

The company has developed an adeno-associated virus (AAV) gene therapy platform focused on controlling gene expression, as well as a suite of blood-brain barrier-penetrant delivery approaches to deliver gene therapies directly to the brain and spinal cord. 

AVB-101 is the neuroscience company’s lead candidate. It is an investigational, one-time gene therapy for the treatment of FTD – a common and debilitating form of early-onset dementia – in patients with mutations in the progranulin (GRN) gene, and is designed to slow or arrest disease progression through the delivery of a functional copy of the GRN gene throughout the CNS, helping to restore normal progranulin levels.

In 2022, AVB-101 received orphan designation from the FDA and the European Commission (EC). Meanwhile, AviadoBio also announced an exclusive option and license agreement with Astellas in October last year for AVB-101.

AVB-101 is currently in a phase 1/2 trial for FTD with GRN mutations, for which the neuroscience company announced the completion of the second dose cohort in May. 

BioArctic

• Technology: Antibody therapies
• Notable neuroscience product: Leqembi for Alzheimer’s disease
• Recent news: Entered into a collaboration with Novartis regarding a potential new treatment 

Neuroscience company BioArctic is developing novel treatments in both common and rare neurodegenerative diseases, such as Alzheimer’s disease, Parkinson’s disease, ALS, and several other indications. The company’s focus lies in antibody treatments that target the misfolded and aggregated proteins underlying neurodegenerative diseases; or, more specifically, the company’s drugs target the soluble, and more toxic forms of the disease-causing proteins, known as oligomers or protofibrils. 

In fact, BioArtic was the original developer of Leqembi, the breakthrough Alzheimer’s treatment approved by the FDA that is now in the hands of Eisai and Biogen; Eisai collaborated on the development of the drug with BioArtic in 2005 before obtaining global rights to study, develop, manufacture, and market it in 2007, and subsequently including it in an Alzheimer’s collaboration with Biogen in 2014. 

As well as its successful partnership with Eisai, BioArctic has formed several other high-profile collaborations over the years. Most recently, it entered into an exclusive global licensing agreement with Bristol Myers Squibb for its PyroGlutamate-amyloid-beta (PyroGlu-Aβ) antibody program, as well as an option, collaboration, and license agreement with Novartis regarding a potential new treatment combining BioArctic’s BrainTransporter technology with an undisclosed target in neurodegeneration.

Biogen

• Technologies: Monoclonal antibodies, immunomodulators, etc
• Notable neuroscience product: Leqembi for Alzheimer’s disease
• Recent news: Leqembi was granted marketing authorization by the EMA

Founded in 1978, Biogen has been discovering, developing, and delivering treatments for neurological diseases for more than 40 years, making them pioneers in neuroscience. The neuroscience company works on developing treatments for diseases such as ALS, Alzheimer’s, depression, lupus, multiple sclerosis (MS), and spinal muscular atrophy (SMA).

Along with a number of already-approved therapies for these neurological disorders – including several treatments for MS, such as AVONEX, PLEGRIDY, and VUMERITY – the company also has a large drug development pipeline, with multiple ongoing phase 3 clinical trials, as well as phase 1 and 2 trials. Many of the drug candidates are being developed in collaboration with other companies. 

One of Biogen’s most notable collaborations is with Eisai; the two companies have been collaborating on the joint development and commercialization of Alzheimer’s treatments since 2014. This partnership led to the success of Leqembi, which has been heralded as a breakthrough for the treatment of Alzheimer’s after it was approved by the FDA in July 2023. The European Medicines Agency (EMA) also granted marketing authorization for Leqembi in April this year, after initially rejecting it in July 2024, and, similarly, the Therapeutic Goods Administration (TGA) approved it in Australia in September this year after it had previously rejected it. These initial rejections were due to concerns that the drug’s benefits may not outweigh the risks for many patients.

Capsida Biotherapeutics

• Technology: Gene therapy
• Lead neuroscience candidate: CAP-002 for STXBP1 developmental and epileptic encephalopathy
• Recent news: Received IND clearance from the FDA to initiate a phase 1/2 study for its Parkinson’s disease candidate, CAP-003

Capsida Biotherapeutics is a gene therapy platform company developing a new class of targeted, non-invasive gene therapies for patients of all ages with debilitating and life-threatening diseases through the intravenous (IV) delivery of engineered capsids that can target single or multiple organs simultaneously – including the CNS – while limiting exposure to non-targeted organs.

Capsida’s lead program, dubbed CAP-002, has received both FDA orphan drug designation and FDA fast track designation, and is currently in a phase 1/2 study for the treatment of children with syntaxin-binding protein 1 (STXBP1) developmental and epileptic encephalopathy, a brain disorder characterized by abnormal brain function and recurrent seizures. However, the company had a rocky start to life in the clinic when it chose to voluntarily pause the phase 1/2 study of CAP-002 in September after the death of the first patient. It said at the time that it had alerted the FDA and planned to submit a full report, but it has not yet provided an update on the status of the trial.

The company also announced in June that it had received Investigational New Drug (IND) clearance from the FDA to initiate a phase 1/2 study for its Parkinson’s disease candidate, CAP-003.

Back in 2021, Capsida debuted with $140 million of capital, which was made up of a $50 million series A funding from Versant Ventures and Westlake Village BioPartners, and an additional $90 million in upfront and equity investment capital as part of a multi-year strategic collaboration and option agreement with AbbVie to develop best-in-class, targeted gene therapies for three programs in serious neurodegenerative disorders. AbbVie exercised an option for the first neurodegenerative disease program under the ongoing collaboration in January this year, which led to Capsida receiving a $40 million license payment. 

Immunic Therapeutics

• Technology: Small molecules
• Lead neuroscience candidate: Vidofludimus calcium for MS
• Recent news: Reported new, positive long-term open-label extension data from its phase 2 trial of vidofludimus calcium

Working on the development of a clinical pipeline of orally administered, small molecule therapies for chronic inflammatory and autoimmune disorders, Immunic Therapeutics currently has three products in clinical development. Its lead candidate, vidofludimus calcium (IMU-838), is being developed for the treatment of multiple sclerosis (MS) – an autoimmune disorder that affects the brain, spinal cord, and optic nerves.

Vidofludimus calcium, which works by inhibiting dihydroorotate dehydrogenase (DHODH) and Nurr1, and has shown combined anti-inflammatory, anti-viral, and neuroprotective effects is being tested in several ongoing MS trials, including twin phase 3 trials in relapsing MS and a supportive phase 2 trial in progressive MS. Topline data from the twin phase 3 trials are expected by the end of 2026. 

In June, Immunic reported new, positive long-term open-label extension data from its phase 2 trial of vidofludimus calcium in patients with relapsing-remitting multiple sclerosis, which showed that, at week 144, 92.3% of patients remained free of 12-week confirmed disability worsening (CDW), with 92.7% remaining free of 24-week CDW. 

Also in June, Immunic announced that it had closed a $65 million public offering. The company said that the net proceeds from this would be used to fund its clinical trials and operations, as well as for other general corporate purposes.

MapLight Therapeutics 

• Technology: Targeted treatments
• Lead neuroscience candidate: ML-007C-MA for schizophrenia and Alzheimer’s disease psychosis
• Recent news: Priced a $258.9 million initial public offering

With the belief that currently available treatment options for brain disorders offer limited efficacy for patients and produce significant side effects, MapLight Therapeutics is on a mission to change the treatment paradigm for neurological diseases. To do so, the company is focusing on the potential of targeted treatments and has built a discovery platform that combines different technologies to uncover the individual circuits that misfire in brain disorders. These technologies target those circuits, enabling the company to develop innovative therapeutics with the potential to improve the treatment of multiple CNS conditions.

MapLight’s lead program, ML-007C-MA, is a fixed-dose combination of the investigational M1/M4 muscarinic agonist, ML-007, co-formulated with a peripherally acting anticholinergic (anticholinergics are substances that block the action of the acetylcholine neurotransmitter at synapses in the central and peripheral nervous system). The candidate is essentially designed to activate both M1 and M4 muscarinic receptors in the CNS to drive efficacy, while synchronizing the pharmacokinetics of the agonist and antagonist components to mitigate certain side effects. ML-007C-MA is currently being evaluated in phase 2 trials for the treatment of schizophrenia and Alzheimer’s disease psychosis.

In July 2025, MapLight managed to secure the biggest private biotech funding round of the month after bringing in $372.5 million in series D financing, which it said would help to advance ML-007C-MA through its ongoing phase 2 trials, as well as fund the exploration of other potential indications for the candidate. Furthermore, the company decided to go public last month, pricing a $258.9 million initial public offering (IPO). 

Muna Therapeutics

• Technology: Small molecules 
• Lead neuroscience candidate: MNA-001 for Alzheimer’s disease
• Recent news: Announced that the first patients had been dosed in a phase 1 trial of MNA-001

In 2021, Novo Holdings launched neuroscience company Muna Therapeutics with $73 million in series A funding to advance novel small molecule treatments specifically for neurodegenerative diseases, such as Alzheimer’s disease, MS, and Parkinson’s disease. The company was formed as a result of the combination of two European startup companies: Muna and K5 Therapeutics.

Muna’s aim is to enhance resilience to the effects of misfolded protein pathology to protect brain functions like cognition. To help it achieve this, its all-in-human discovery engine, MiND-MAP, identifies new therapeutic targets that can enhance the brain’s innate protective mechanisms. The platform applies spatial transcriptomics to brain samples from Alzheimer’s disease patients, cognitively resilient individuals, healthy controls, and centenarians with and without cognitive impairment.

Earlier this month, Muna announced that the first patients had been dosed in a phase 1 trial of its lead asset MNA-001, an oral TREM2 agonist intended for the treatment of early Alzheimer’s disease and other neurodegenerative diseases. Topline data from this trial is expected in mid-2026. 

Meanwhile, in December 2024, Muna entered into a strategic alliance with GSK to identify and validate novel drug targets for the treatment of Alzheimer’s disease. As part of the collaboration, the companies are exploring insights from Muna’s MiND-MAP platform. 

NRG Therapeutics

• Technology: Small molecules
• Lead neuroscience candidate: NRG5051 for ALS/motor neurone disease
• Recent news: Raised £50 million ($67 million) in series B funding

NRG Therapeutics describes itself as a neuroscience-focused drug discovery company building a pipeline of disease-modifying drug candidates, using therapeutic approaches to restore mitochondrial function while slowing or halting the progression of neurodegenerative diseases, such as ALS and Parkinson’s disease.

The company is developing novel inhibitors targeting the mitochondrial permeability transition pore (mPTP), a protein that plays a key pathological role in the neurodegeneration associated with conditions like Parkinson’s and ALS. Inhibition of the mPTP has been shown to protect neurones, reduce neuroinflammation, and improve motor function in preclinical disease models.

NRG Therapeutics has a preclinical pipeline of small molecule assets that help to prevent the pathological effects of misfolded TDP-43 and α-synuclein, which cause the degeneration of motor neurons and dopaminergic neurons in ALS and Parkinson’s, respectively. The company’s lead asset, NRG5051, has completed IND-enabling studies and is on track to enter the clinic in early 2026 for the treatment of ALS/motor neurone disease.

In November 2022, the neuroscience company closed a £16 million ($18.2 million) series A financing to advance its potential first-in-class brain-penetrant small molecules through IND-enabling studies. Shortly after this, in February 2023, the company was awarded a second $500,000 grant from The Michael J. Fox Foundation for Parkinson’s Research, which it said would support its lead discovery program and aid the development of a novel treatment for Parkinson’s. And, most recently, in September this year, NRG Therapeutics raised £50 million ($67 million) in series B financing, which it said would enable it to deliver clinical proof of concept in ALS/motor neurone disease with NRG5051. 

QurAlis

• Technology: Precision medicines
• Lead neuroscience candidate: QRL-201 for ALS 
• Recent news: Entered into a collaboration with Quiver Bioscience to advance a gene-targeted therapeutic approach for the treatment of Fragile X syndrome

QurAlis is advancing precision medicines for neurodegenerative diseases. Its two lead candidates are QRL-201 and QRL-101, both of which are in phase 1 clinical studies and are being developed for the treatment of ALS. 

QRL-201 is a first-in-class molecule for the treatment of ALS that aims to restore STATHMIN-2 (STMN2) expression in patients with the disorder. STMN2 is a protein that is important for the stabilization of microtubules that form an important part of the cytoskeleton of cells and axons. Reduced STMN2 is a hallmark of ALS. 

Meanwhile, QRL-101 is a selective Kv7.2/7.3 ion channel opener that aims to reduce hyperexcitability-induced neurodegeneration. Kv7.2/7.3 is a voltage-gated potassium channel in cell membranes and is the dominant component of the neuronal M-current in human motor neurons that stabilizes the membrane potential and controls neuronal excitability. Selecting Kv7.2/7.3 as a drug target for the treatment of ALS comes from research using human ALS motor neurons. Kv7 modulation has been shown to decrease spinal and cortical motor neuron excitability, both of which have been linked to patient survival. 

QurAlis also recently entered into a research collaboration with Quiver Bioscience to advance a novel gene-targeted therapeutic approach for the treatment of the inherited genetic disorder Fragile X syndrome. The goal of the collaboration is to combine Quiver’s “Genomic Positioning System” (GPS) drug discovery platform with QurAlis’ expertise in developing next-generation precision medicines for neurodegenerative and neurological diseases to build a foundational data package in support of advancing a potentially transformative therapeutic for the condition. 

Neuroscience: A challenging field 

Developing medicines in neuroscience is extremely challenging, and many companies have tried and failed to develop successful candidates. Just in the last year, there have been several notable failures in the field of candidates that once seemed to hold promise. For example, Asceneuron, which brought in $100 million in series C funding last summer, decided to shut down a phase 2 trial of its lead asset for Alzheimer’s disease in March. Additionally, Neumora Therapeutics lost more than 80% of its value in January when its most advanced asset failed a depression study.

Assets ultimately fail in this area due to the brain’s complexity – it is not yet fully understood, which makes it hard to pinpoint precise disease mechanisms, and many neurological conditions involve intricate networks of cells and signals, so targeting a single pathway is often not enough. Meanwhile, drug delivery is also a major hurdle, as the blood–brain barrier blocks most compounds from reaching their intended targets. Additionally, animal models frequently fail to mimic human neurological diseases, leading to high failure rates in clinical trials. As a result, progress in this area tends to be slower and riskier than in many other biotech fields. 

Nevertheless, these seemingly insurmountable hurdles do not stop companies like the ones mentioned in this article from coming up with an array of novel medicines with the potential to treat neurological diseases, ultimately offering hope to patients all over the world.