Introduction

Neuroscience is undergoing a major resurgence. After more than a decade of limited successes and cautious investment by most big pharma, a new era innovation is unfolding. Driven by a confluence of factors – the vast unmet need (a 2021 Lancet Neurology study found 3.4 billion people worldwide suffer from nervous system disorders), and the dedication of patients, scientists, physicians, regulators, and companies have continued to de-risk the space with continued innovation on multiple fronts. We explore the latest advancements poised to reshape the future of neurosciences 2024 and beyond.  

Major advances are occurring in multiple fronts
  1. Better understanding of disease biology/improved models
  2. Continued elucidation of targets and approaches to address
  3. Optimized clinical trial design/better endpoints
  4. More flexible regulatory structure that understands the significant need for new therapeutics

These advances are not only fueling growth in the neuroscience pipeline (Lumanity analysis), but are also spurring a resurgence in big pharma interest and investment, evident in recent high-profile acquisitions:

  • AbbVie acquisition of Cerevel: In December 2023, AbbVie acquired Cerevel and its neuroscience pipeline of multiple clinical-stage and preclinical candidates with potential across several diseases including schizophrenia, Alzheimer’s disease psychosis, epilepsy, panic disorder, and Parkinson’s disease for $8.7 billion.
  • Bristol Myers acquires Karuna Therapeutics: In March 2024, Bristol Myers $14 billion buyout of Karuna Therapeutics driven by the company’s lead program, KarXT (xanomeline-trospium), an M1/M4-preferring muscarinic agonist antipsychotic with a novel mechanism of action and a differentiated efficacy and safety profile; touted as a potential first-in-class therapy with multi-billion-dollar sales potential across multiple indications

Lumanity has identified near-term transformational potential across multiple neuroscience areas including:

Looking toward the second half of 2024
  • FDA approval decision for Lykos Therapeutics’ MDMA-assisted therapy for PTSD is expected by the Prescription Drug User Fee Act (PDUFA) target action date of August 11, 2024, making it the first psychedelic-assisted therapy to be considered for approval.
    • *Breaking News: On June 4, 2024 the FDA’s Psychopharmacologic Drugs Advisory Committee (“PDAC”) voted 2 yes to 9 no on the question of whether the data for Lykos’ MDMA-assisted therapy for PTSD show that the drug is effective in patients with PTSD. They also voted 1 yes to 10 no on the question of whether the benefits with the FDA’s proposed risk evaluation and mitigation strategy (REMS) outweigh its risks for the treatment of patients with PTSD. The FDA is not bound by PDAC’s guidance but takes its advice into consideration (https://news.lykospbc.com/2024-06-04-Lykos-Therapeutics-Provides-Update-on-FDA-Advisory-Committee-Meeting-for-Investigational-MDMA-Assisted-Therapy-for-PTSD).
    • The impact of this on the growing pipeline of psychedelics for high unmet need indications including PTSD, generalized anxiety disorder and treatment-resistant depression is something the psychiatric community is closely watching.
  • KarXT (xanomeline-trospium) FDA approval announcement expected by Sept. 26, 2024, heralding a new generation of muscarinics for treatment of schizophrenia
  • Vertex NaV1.8 (suzetrigine) NDA for broad acute pain indication anticipated in H1 2024​ and potential broad peripheral neuropathic pain indication ~2025; could be first novel mechanism, non-opioid pain therapeutic in decades
  • Abbvie/Cerevel has three H2 2024 clinical readouts for epilepsy, psychiatric and neurodegenerative diseases
    • Darigabat (positive allosteric modulator of GABAA receptors) PII for epilepsy
    • Emraclidine (positive allosteric modulator of muscarinic acetylcholine 4 receptor subtype) PII for schizophrenia
    • Tavapadon (partial, selective agonist of dopamine D1/D5 receptor subtypes) PIII for Parkinson’s Disease
  • Neurocrine also has three H2 clinical events, all novel therapies for the treatment of schizophrenia
    • Luvadaxistat (selective d-amino acid oxidase (DAAO) inhibitor) PII for Cognitive Impairment Associated with Schizophrenia (CIAS)
    • Valbenazine (selective, orally active vesicular monoamine transporter 2 (VMAT2) inhibitor) PIII as adjunctive therapy for schizophrenia
    • NBI-1117568 (muscarinic M4 selective acetylcholine receptor agonist) PII for Cognitive Impairment Associated with Schizophrenia (CIAS)
Despite the resurgence and renewed interest, significant challenges remain

While advances in neurosciences mitigate some of the scientific and clinical/regulatory risk, meaningful commercial risk remains. Navigating this landscape requires companies to make strategic decisions at key inflection points during the development and commercialization of assets and portfolios. These decisions are crucial for defining, establishing, and demonstrating clinical relevance and value to a complex set of key stakeholders including physicians, patients and caregivers, and payers. Despite the inherent challenges of measuring and communicating value for novel neurological therapies, companies are making bold moves to identify these drivers in this commercially uncertain landscape.

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Lumanity offers extensive experience-based strategic guidance and insights in the field of neuroscience. Our goal is to provide our clients with actionable solutions that de-risk and optimize the development and commercialization of therapies, ultimately improving the lives of patients who are impacted across a range of conditions.

Contact us to learn more about how Lumanity can support your unique challenge today.