10 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 – the likes of GSK, Novartis, and Pfizer – shutting down their neuroscience drug development programs. 

Fortunately, plenty of smaller 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 take a look at 10 of the top neuroscience companies around today.

Alzheon

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 ALZ-801 and is currently in phase 3 of development. 

ALZ-801 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, ALZ-801 has been shown to fully inhibit the formation of neurotoxic soluble beta-amyloid oligomers. The initial phase 3 program for ALZ-801 focuses on early Alzheimer’s patients with two copies of the apolipoprotein ε4 allele (APOE4/4 homozygotes), who constitute approximately 15% of Alzheimer’s patients. The drug candidate received fast track designation from the U.S. Food and Drug Administration (FDA) in 2017 for the treatment of Alzheimer’s disease. 

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

Asceneuron

Asceneuron has come up with a unique approach to tackling Alzheimer’s that involves tau proteins, setting it apart from competitors targeting amyloid-beta aggregates. Tau proteins play a crucial role in maintaining the structure and function of neurons by stabilizing microtubules, which are essential for intracellular transport. In neurodegenerative diseases, tau proteins undergo abnormal phosphorylation, leading to the formation of neurofibrillary tangles. Ultimately, when this happens, it blocks communication and leads to neuron death, driving the symptoms of Alzheimer’s​. Unlike amyloid-beta plaques, tau ‘tangles’ form inside neurons rather than outside them, meaning they directly impact the health and function of neurons. 

Asceneuron’s lead asset for Alzheimer’s is called ASN51. It is a daily oral small molecule O-GlcNAcase (OGA) inhibitor (OGA is an enzyme that removes O-GlcNAc groups from proteins, including tau). The drug works by stabilizing tau proteins in a state that is less likely to form harmful aggregates. This helps to maintain normal neuronal function and potentially slow the progression of neurodegenerative disease.

Asceneuron has already completed five phase 1 clinical trials for ASN51, whereby the drug was found to be well-tolerated with a favorable safety profile. Pharmacokinetic data indicated that the drug is effectively absorbed when taken orally and achieves the desired therapeutic levels in the body​​. Upcoming phase 2 trials will now focus on evaluating the efficacy of ASN51 in patients with mild to moderate Alzheimer’s disease. 

Just last month, Asceneuron raised $100 million in a series C round led by Novo Holdings. The funds will help to accelerate the development of ASN51. 

AviadoBio

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 solutions to deliver gene therapies directly to the brain and spinal cord. It is also building additional platforms and capabilities as well as proprietary RNA silencing and subpial delivery technologies. 

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 U.S. Food and Drug Administration (FDA) and the European Commission (EC). AVB-101 is currently in a phase 1/2 trial for FTD with GNR mutations, in which the first patient was treated in April 2024. 

Biogen

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 development of the drug Leqembi, which has been heralded as a breakthrough for the treatment of Alzheimer’s after it was approved by the FDA in July last year. However, the therapy was recently rejected by the European Medicines Agency (EMA), who said that the risk the drug posed outweighed the benefit, leaving its future in the European Union (EU) uncertain. 

Capsida Biotherapeutics

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 therapeutic candidates are still in the preclinical stages. The company also has a few partnered programs after entering into several high-profile collaborations. 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. Last year, the two companies expanded their strategic collaboration to develop genetic medicines for eye diseases with high unmet need. 

Capsida also entered into a strategic collaboration with CRISPR Therapeutics in 2021 to research, develop, manufacture, and commercialize in vivo gene editing therapies delivered with engineered AAV vectors for the treatment of familial ALS and Friedreich’s ataxia. Additionally, in early 2023, Capsida announced another strategic collaboration, this time with Prevail Therapeutics – a subsidiary of Lilly – to develop best-in-class, IV-administered gene therapies directed to specific targets that are known to cause serious diseases affecting the CNS. 

Immunic Therapeutics

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 MS – an autoimmune disorder that affects the brain, spinal cord, and optic nerves.

Vidofludimus calcium, which works by inhibiting dihydroorotate dehydrogenase (DHODH) 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. The phase 2 trial produced positive interim data that was reported by the company in October 2023. The company said that this data showed biomarker evidence that vidofludimus calcium’s activity extends beyond the previously observed anti-inflammatory effects, thereby further reinforcing its neuroprotective potential.

In January 2024, Immunic announced a private placement of up to $240 million. The company said these funds would be used to fund the ongoing clinical development of its candidates, including vidofludimus calcium.

Muna Therapeutics

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. The company was formed as a result of the combination of two European startup companies: Muna and K5 Therapeutics.

Muna Therapeutics’ aim is to discover and develop therapies that will slow or stop the progression of debilitating neurodegenerative conditions, including Alzheimer’s, FTD, and Parkinson’s disease. To try and achieve this, it has a drug discovery platform that combines high-resolution target structural approaches, artificial intelligence (AI)-driven computational chemistry, and cell-based screening. It is advancing first-in-class small molecule programs focused on restoring neuron function in patients with progranulin pathway dysfunction – which leads to FTD – and resolving neuroinflammation and normalizing microglia function in patients with Alzheimer’s and other neurodegenerative diseases. 

In October 2022, Muna Therapeutics was awarded a $4.9 million grant from The Michael J. Fox Foundation to support the ongoing preclinical research and development of novel, brain-exposed, small molecule potassium channel type 1.3 (Kv1.3) blockers as a disease-modifying therapy for Parkinson’s disease. The blockers abrogate neuroinflammation driven by disease-associated microglia and increase neuroprotection in patients with the disease.

Neumora Therapeutics

Neumora Therapeutics is another recently founded neuroscience company, which launched in October 2021 with $500 million to develop targeted treatments for brain disorders. Having raised $400 million in a series A round, the company also received an additional $100 million in equity investment from Amgen. 

Neumora is focused on advancing medicines for therapeutically relevant targets implicated in CNS diseases, targeting novel mechanisms of action with best-in-class pharmacology, and leveraging a precision medicine approach to better understand the biological drivers of brain diseases and to identify targeted patient populations of interest.

The company’s current pipeline includes a broad range of novel medicines for both neuropsychiatric and neurodegenerative disorders, including one called navacaprant, which is in phase 3 of development to treat major depressive disorder (MDD). Navacaprant is an oral medication designed to modulate the dopamine and reward processing pathways, which play an important role in the regulation of mood, cognition, reward, and behavior. The company also recently announced the initiation of a phase 2 study of navacaprant in bipolar depression. Furthermore, in June 2024, the company also announced that it had initiated a phase 1b study of another candidate, NMRA-511, for the treatment of Alzheimer’s disease agitation. 

In September 2023, Neumora brought in $250 million from a public offering. 

NRG Therapeutics

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 Parkinson’s and ALS. 

The company is developing novel inhibitors targetting the mitochondrial permeability transition pore (mPTP), a protein that plays a key pathological role in the neurodegeneration associated with Parkinson’s and ALS. The company has discovered the first orally bioavailable and brain-penetrant mPTP inhibitors that help to prevent the pathological effects of misfolded TDP-43 and a-synuclein. TDP-43 and a-synuclein cause the degeneration of motor neurons and dopaminergic neurons in ALS and Parkinson’s, respectively. 

After NRG received a £2.68 million ($3.26 million) early-stage Biomedical Catalyst (BMC) award, it entered into a collaboration in 2022 with Domainex – an integrated medicines research services partner – to develop small molecule disease-modifying medicines for the treatment of Parkinson’s and motor neuron disease (MND). The ultimate aim of the collaboration was to nominate a preclinical candidate for these diseases.

In November 2022, NRG also 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.

QurAlis

Having closed an oversubscribed $88 million series B financing round in March 2023, neuroscience company QurAlis is advancing its 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 has two other programs in preclinical development – QRL-203 for the treatment of FTD, and QRL-204 for the treatment of ALS and FTD. The company recently granted Lilly exclusive global rights to develop and commercialize QRL-204. 

A high unmet need for CNS drugs 

According to a 2022 report from the World Health Organization (WHO), disorders of the nervous system are the second leading cause of death worldwide, accounting for nine million deaths per year. A big reason for this is that currently available therapies for neurological disorders are largely limited to the treatment of symptoms rather than the treatment of the disease itself, signaling an unmet medical need for disorders of this type. 

Thankfully, there has been a lot of much-needed recent activity in the CNS field. Some recent high-profile deals in this area include Bristol Myers Squibb’s $14 billion purchase of Karuna Therapeutics, Pfizer’s $11.6 billion acquisition of Biohaven, and AbbVie’s $8.7 billion buyout of Cerevel. Hopefully, this activity will soon lead to some breakthroughs in the treatments of several CNS disorders in which patients currently face a lack of options.