Our Investment in Neumora: A Data Sciences-Driven Precision Approach to Treating Central Nervous System Diseases
Central nervous system (CNS) diseases, which include neurodegenerative disorders and neuropsychiatric illnesses, have placed a significant burden on healthcare systems worldwide. Reports show that neurological disorders accounted for 16% of total deaths globally in 2016, while psychiatric diseases and mental disorders were attributable to 14% of deaths worldwide in 2015. [1,2]
While these diseases account for a considerable proportion of mortality, there has been a significant lack of effective drugs available to patients. The pharma industry has had little success bringing CNS drugs to market. Compared to the 13% success rate for getting non-CNS drugs to market, the probability of success for CNS drugs is as low as 6%. 
There are several problems with the current paradigm of CNS drug development. In preclinical studies, animal models lack the complexity of human phenotypes, not capturing the entire spectrum of neurodegenerative and neuropsychiatric diseases. This leads to poor translatability in clinical trials. The present approach also uses traditional behavioral and symptomatic clinical endpoints that are highly subjective and non-quantitative.
At the center of these issues is the “one-size-fits-all” approach the current system uses for treating CNS diseases, where all patients are viewed as part of a large, homogeneous population and treated with a single drug. Within these diseases are sub-populations discretely characterized by differentiating features, including genetic and epigenetic, behavioral and environmental factors.
Overlooking the multifaceted nature of these disorders has led to the high failure rate in developing successful and effective CNS therapies. Therefore, there is an unmet need to address this heterogeneity and the unique biomarker sets for patient sub-types, and to define distinct populations that will benefit the most from targeted therapies. To shift away from this “one-size-fits-all” approach and improve CNS patient outcomes, we need to take a page from another disease — cancer.
Twenty years ago, all cancer patients received “shotgun” treatments with chemotherapy or radiation therapy. Patients weren’t segregated into different cancer sub-types, and this ultimately led to poor outcomes. But today, with the advances in molecular testing and next-generation sequencing, cancer patients are sub-typed on data from disease syndromes, phenotypes and genetics. This results in a personalized, targeted treatment strategy for each patient based on their individual disease profiles. The field of oncology has moved away from “one-size-fits-all” to precision medicine, which has significantly improved clinical success.
Leveraging this data-based methodology, the company Neumora Therapeutics is focused on shifting away from traditional CNS drug development and taking a precision approach to treating patients with neurodegenerative and neuropsychiatric diseases. Neumora’s mission is to use its unique, broadly applicable technology platform to identify distinct patient populations who will benefit most from targeted therapy. This innovative approach is why we decided to support Neumora and participate in their recent financing.
Neumora’s scalable data sciences platform uses large clinical and preclinical datasets on disease progression, genetic and epigenetic drivers, and environmental factors, to draw insights into defining homogeneous sub-types in CNS diseases. Through collaborations with leading clinical biomarkers, genomics and disease modeling companies, Neumora has grown its toolbox of open-source and proprietary datasets as well as AI/ML algorithm technologies to create multi-modal “data biopsies” that help identify homogeneous patient groups.
After distinct patient populations are defined, the company utilizes its platform to discover and develop targeted drugs that have the potential to benefit those particular patients, leading to de-risked clinical trials and creating new translational models. By treating those patients with precision therapies, Neumora can revolutionize the CNS drug development process by decreasing costs, increasing the probability of bringing effective drugs to market and improving patient outcomes.
While we are still in the early innings of building Neumora alongside ARCH Venture Partners and other leading biotech investors, the company has leveraged their novel approach to build a robust clinical and pre-clinical pipeline across a number of neuropsychiatry and neurodegenerative diseases.
At Mubadala Capital, our goal is to invest in innovative platform companies that will bring significant value to the healthcare industry. With a world-class leadership team and investor syndicate, Neumora has demonstrated its ability to build a scalable technology that will transform drug development for a wide range of neurological and neuropsychiatric diseases. Increasing access to targeted, personalized drugs is vital to improving patient care, and we are excited to support Neumora as they unlock the next generation of CNS medicines.
More on Neumora can be found here, here, here and here
1. GBD 2016 Neurology Collaborators. Global, regional, and national burden of neurological disorders, 1990–2016: a systematic analysis for the Global Burden of Disease Study 2016. Lancet Neurol. 2019;18(5):459–480. doi:10.1016/S1474–4422(18)30499-X
2. Walker ER, McGee RE, Druss BG. Mortality in mental disorders and global disease burden implications: a systematic review and meta-analysis. JAMA Psychiatry. 2015;72(4):334–341. doi:10.1001/jamapsychiatry.2014.2502
3. Kaitlin KI. CNS drugs take longer to develop, have lower success rates, than other drugs. In: Kaitin KI, editor. Tufts CSDD Impact Reports. Vol. 16. Tufts University, Tufts Center for the Study of Drug Development; 2014. pp. 1–4.