Neuron23 completes Series C funding and Parkinson's disease drug candidates enter clinical trials

On March 30, Neuron23 Inc., an early-stage biotechnology company focused on developing precision drugs for genetically defined neurological and immune diseases, announced that it had completed a $100 million Series C funding round, led by SoftBank's SoftBank Vision Fund 2, bringing the company's total funding to date to $213.5 million. Historic investors Westlake Village BioPartners, Kleiner Perkins, Redmile Group, Cowen Healthcare Investments, Acorn Bioventures, HBM Partners, Perceptive Advisors and Surfeyor Capital also participated in the round.

Proceeds from the Series C round will be invested in the company's major projects: LRRK2 (leucine-rich repeat kinase 2) and TYK2 (tyrosine kinase 2). LRRK2 is a gene associated with Parkinson's disease and systemic inflammatory diseases, and TYK2 is a JAK family protein that plays a role in pathological immune signaling. The funding will also be used to build Neuron23's clinical development team, precision neuroimmunology platform, and data science capabilities.

Founded in 2018, Neuron23 is an early-stage biotechnology company focused on developing precision drugs for genetically defined neurological and immune diseases. Neuron23 leverages recent advances in human genetics, combined with its state-of-the-art AI drug discovery and biomarker platform, to advance the treatment of devastating diseases. The company's focus areas are neurodegenerative diseases, neuroinflammatory diseases, and systemic autoimmune and inflammatory diseases.

Parkinson's disease is the second most common neurodegenerative disease, the prevalence of Parkinson's disease over 60 years old in Europe and the United States has reached 1%, more than 4% over 80 years old, and the prevalence of people over 65 years old in China is 1.7%, similar to that of European and American countries. In the future, the number of people with Parkinson's disease in the continent will rise from 1.99 million in 2005 to 5 million in 2030, accounting for almost half of the world's Parkinson's disease patients.

A 2004 study by Paisan-Ruiz found that mutant LRRK2 is toxic to neurons, and mutations in this protein are associated with genetic forms of Parkinson's disease. The discovery of LRRK2 has attracted hundreds of millions of dollars in investment to support drug research aimed at stopping it. There is also new evidence that the activity of this mutated protein may also play a role in a subset of people with non-hereditary Parkinson's disease.

Neuron23 is designed to treat Parkinson's by blocking LRRK2. The main drug candidate, NEU-723, is a small molecule that will penetrate the brain and block the activity of mutant proteins.

Neuron23 has many competitors in the LRRK2 space.

Denali Therapeutics, based in San Francisco, USA, has a variety of brain penetration drugs for a range of neurological disorders, and Denali Therapeutics' targeted drug BIIB122/DNL151 for LRRK2, which will enter Phase 3 later this year.

Cerevel Therapeutics is also developing an LRRK2 candidate, which is licensed by Pfizer. The company said in its 2021 annual report that it aims to treat a specific variant of LRRK2 called G2019S, and that the company's LRRK2 inhibitor is in the discovery stage.

Neuron23 acknowledges the LRRK2 competition it faces. In an email, CEO Nancy Stagliano wrote that Neuron23's drug has two characteristics that make it potentially the best drug in the LRRK2 inhibitor class.

First, the drug is not only highly effective and capable of penetrating the brain, but the Neuron23 molecule is more selective in the way it hits its targets than its competitors. Second, the drug is taking a precision medicine approach to Parkinson's, using companion diagnostics to select patients who are most likely to respond to this treatment.

In addition to treating patients with LRRK2 mutations, Stagliano said Neuron23 will identify and treat a subset of patients with sporadic Parkinson's disease, whose disease is also driven by LRRK2, "which should increase our chances of clinical success later in life." ”

Neuron23 nominated NEU-723 as a clinical candidate for Parkinson's disease. Neuron23 is expected to begin clinical trials of NEU-723 by the end of 2022.

The new funding will also be used to support the development of a drug designed to block the immune-signaling protein TYK2, a part of the JAK protein family. These proteins have become the target of drugs to treat a range of autoimmune diseases. JAK inhibitors from companies such as Pfizer, Insett and AbbVie are approved for the treatment of conditions including rheumatoid arthritis, atopic dermatitis, psoriatic arthritis and ulcerative colitis.

TYK2 is an attractive target because hitting it does not affect limiting immune function and leads to immunodeficiency, which is a problem with many autoimmune disease drugs. Bristol Myers Squibb's goal is to block the enzyme as a way to treat autoimmune diseases; last October, the pharmaceutical giant reported that its TYK2 inhibitor, deucravacitinib, had failed in an interim clinical trial for ulcerative colitis.

Unlike other drug candidates for TYK2, Neuron23 says its preclinical drug can penetrate the blood-brain barrier, potentially addressing diseases characterized by neuroinflammation, such as multiple sclerosis. As the FDA's warnings about cancer and cardiovascular risk become more stringent, the entire category of JAK-blocking drugs has been affected. If Neuron23 can prove that its drug has better side effects, the molecule will be able to further differentiate itself from other areas.

Series C funding involves more than just getting more money. Neuron23 has also added two new board members: Jim Scopa and Valentin Barsan, MD, a veteran life sciences investor, and Barsan, a major investor in this round, SoftBank Investment Advisers.