Notable Trends and Companies to Follow in Neuroscience

by Irina Bilous          Biopharma insight

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Historically, the approval rate for medicines treating central nervous system (CNS) disorders was low. By 2022, the success rate for CNS assets from phase 1 through to regulatory submission was only 5–6%.

The failure of pharmaceutical companies to bring CNS medications to the market can be attributed to factors such as a limited understanding of disease mechanisms and challenges in delivering drugs across the blood-brain barrier. Nevertheless, recent technological advancements and an improved understanding of disease pathophysiology led to expanding the CNS pipeline.

A recent report shows that the CNS pipeline has grown by 31%, constituting 14% of the overall industry R&D pipeline. Moreover, the approval of the first drug for spinal muscular atrophy (SMA) developed by Ionis Pharma and Biogen was a significant stride that revitalized interest in the field and demonstrated the ability of innovative treatments to address complex neurological disorders.

Hot Deals of 2023 Involving Modulators of Muscarinic Receptors

In December 2023, Bristol Myers Squibb expanded its neuroscience portfolio by acquiring Karuna Therapeutics for $14.0 billion. Karuna’s leading candidate, KarXT (xanomeline-trospium), has a dual action on M1 and M4 muscarinic receptors without affecting the dopaminergic pathway.

KarXT is efficient in patients with schizophrenia as it reduces both positive and negative symptoms of the condition, which affects about 24 million people worldwide. When the FDA approves KarXT, it will become the first novel treatment for schizophrenia in decades. 

The same month, AbbVie boosted its neurological and psychiatric disorder pipeline by purchasing Cerevel Therapeutics for $8.7 billion. This move will advance AbbVie's capabilities in addressing Alzheimer's, Parkinson's, psychosis, epilepsy, and panic disorders.

Cerevel's promising drug, emraclidine, is in the Phase 2 trial for schizophrenia. Emraclidine acts on M4 receptors, and similar to KarXT, it doesn’t directly affect dopamine D2 receptors, which hypothetically will prevent adverse effects that are common for available antipsychotic drugs. 

Muscarinic receptors are part of the cholinergic pathway in the brain. Therefore, drugs targeting this pathway affect brain chemistry differently from currently available medicines that act on receptors of dopamine or serotonin, the two other well-known neuromediators. Because dopamine is essential for multiple functions in the brain, e.g., motor control, dampening D2 receptors leads to adverse effects. 

The market for schizophrenia treatments targeting muscarinic receptors alone is estimated at $10 billion. With the acquisition mentioned, AbbVie and BMS strive to take leading positions in the market for muscarinic receptor drugs. Other companies in this space include Neurocrine Biosciences, Sosei Heptares, Neumora Therapeutics, MapLight Therapeutics, and Anavex Life Science.


Companies Bringing Innovations to Improve the Psychiatric and Mental Health 

In September 2023, Otsuka acquired  Mindset Pharma for $59.1 million to accelerate the development of the new generation of serotonin 5-HT2A receptor agonists to treat treatment-resistant depression and post-traumatic stress disorder (PTSD). 

Another acquisition of a brain chemistry modifying asset was accomplished by AbbVie in 2022 when the pharma giant purchased Syndesi. ABBV-552 is a small molecule that acts upon targeting synaptic vesicle glycoprotein 2A (SV2A), which enhances synaptic transmission. It’s currently in Phase 2 clinical trials for Alzheimer’s disease (AD), with the potential to improve cognitive impairments and other symptoms concurrent with AD.

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Topics: Industry Trends   

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