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.
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 9 million deaths per year. A big reason for this is because 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.
Although in recent years big pharma has been seen to pull away from investments in neurosciences research, there are still several biotechs attempting to take on the challenge of developing treatments for a variety of neurological disorders. Here, listed in alphabetical order, are nine of the top neuroscience companies around today.
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AviadoBio
Headquarters: London
Founded: 2019
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 a neuroanatomy-led approach to viral vector distribution, and aims to deliver its gene therapies to the right place in order to maximize their potential, targeting the genetic drivers, modifiers, or pathways of monogenic and complex neurodegenerative diseases.
AVB-101 is the neuroscience company’s lead candidate. It is an investigational, one-time adeno-associated virus (AAV) 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 central nervous system, 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).
Biogen
Headquarters: Cambridge, U.S.
Founded: 1978
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 disease (AD), 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 Co. Ltd; the two companies have been collaborating on the joint development and commercialization of Alzheimer’s disease (AD) treatments since 2014, which last year resulted in a potential breakthrough for the treatment of AD after a global phase 3 confirmatory Clarity AD clinical trial of lecanemab – a humanized monoclonal antibody to treat early-stage AD – showed a reduction in the disease in the brain while slowing memory decline. This was the first time a drug being trialed for AD had shown these kinds of results.
Capsida Biotherapeutics
Headquarters: Thousand Oaks, U.S.
Founded: 2019
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.
In 2021, Capsida debuted with $140 million of capital, with $50 million series A funding from Versant Ventures and Westlake Village BioPartners, as well as an additional $90 million in up front 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. The two companies recently 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 amyotrophic lateral sclerosis (ALS) and Friedreich’s ataxia.
Earlier this year, the company announced another strategic collaboration, this time with Prevail Therapeutics, 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
Headquarters: New York City, U.S.
Founded: 2016
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, IMU-838, is being developed for the treatment of MS – an autoimmune disorder that affects the brain, spinal cord and optic nerves.
IMU-838, which works by inhibiting dihydroorotate dehydrogenase (DHODH) and has shown combined anti-inflammatory, anti-viral and neuroprotective effects, is currently in phase 3 clinical trials, after Immunic’s phase 2 EMPhASIS trial showed that long-term open-label treatment with IMU-838 was associated with a low rate of disability worsening over time. It was also shown to compare favorably to historical trial data for currently available MS medications.
Immunic’s other two programs are IMU-935, for the treatment of psoriasis and castration-resistant prostate cancer, and IMU-856, for the treatment of celiac disease. Both are currently in phase 1 trials.
Muna Therapeutics
Headquarters: Copenhagen, Denmark
Founded: 2020
In 2021, Muna Therapeutics launched with $73 million 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 start-up companies: Muna and K5 Therapeutics.
The neuroscience company’s aim is to discover and develop therapies that will slow or stop the progression of debilitating neurodegenerative conditions, including AD, FTD and Parkinson’s disease. To try and achieve this, it has a drug discovery platform that combines high-resolution target structural approaches, 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 who have AD and other neurodegenerative diseases.
Furthermore, in October last year, 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
Headquarters: Watertown, U.S.
Founded: 2020
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 an equity investment from Amgen.
Neumora believes it is time to develop precision medicines for the treatment of brain disorders, as the traditional one-size-fits-all approach – that has been seen in many clinical trials for neurological disorders throughout the years – regularly leads to underwhelming efficacy and clinical trial failures.
Taking inspiration from the recent revolution in precision oncology, the company aims to redefine neuroscience research and development with a data-driven precision neuroscience platform that will cut through brain heterogeneity to match the right patient populations to targeted therapeutics.
The company’s current pipeline includes a broad range of novel medicines for both neuropsychiatric and neurodegenerative disorders, including one that is in a phase 2 clinical trial to treat major depressive disorder (MDD). Each candidate will be paired with a proprietary precision phenotype to ensure a targeted approach to development.
NRG Therapeutics
Headquarters: Stevenage, U.K.
Founded: 2018
NRG Therapeutics describes itself as a neuroscience-focused drug discovery company building a pipeline of disease-modifying drug candidates, using therapeutic approaches that will restore mitochondrial function while slowing or halting the progression of neurodegenerative diseases, such as Parkinson’s and ALS.
The company is developing novel, drug-like ‘second-generation’ mitochondrial permeability transition pore (mPTP) inhibitors, and 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 is to nominate a preclinical candidate for these diseases.
In November 2022, NRG 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 will support its lead discovery program and aid the development of a novel treatment for Parkinson’s.
QurAlis
Headquarters: Cambridge, U.S.
Founded: 2016
Having recently 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 STMN2 – a protein that is important for the stabilization of microtubules that form an important part of the cytoskeleton of cells and axons – expression in patients with the disorder.
Meanwhile, QRL-101 is a first-in-class Kv7 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.
QurAlis also has three other programs in preclinical development – QRL-203 for the treatment of FTD, QRL-204 for the treatment of ALS and FTD, and QRL-202 for the treatment of ALS and FTD.
UCB
Headquarters: Brussels, Belgium
Founded: 1928
UCB was created by Emmanuel Janssen in the 1920s, with its first therapeutic breakthroughs coming in the 1950s, followed by research in the field of biotechnology in the 1960s. Nowadays, the company is committed to improving the lives of people living with severe neurological and immunological diseases. Its neurology-based disease areas currently include epilepsy, Dravet syndrome, restless legs syndrome and Parkinson’s disease.
Most recently, the company had FINTEPLA, its oral solution for the treatment of seizures associated with Lennox-Gastaut syndrome – a severe form of epilepsy characterized by seizures that begin in early childhood – approved by the EC based on safety and efficacy data from a global, randomized placebo-controlled phase 3 clinical trial. FINTEPLA also received approval from the FDA for use in the U.S. last year.
Some of UCB’s other approved products for neurological conditions include BRIVIACT and VIMPAT for epilepsy, and Neupro for Parkinson’s disease and restless legs syndrome. The company also has several approved products in the area of immunology.
There are currently more than 100 UCB-sponsored clinical studies in different disease areas, such as epilepsy, psoriasis, rheumatoid arthritis and hidradenitis suppurativa.