Nkarta Announces IND Clearance and Enrollment for Myasthenia Gravis Trial

Nkarta, Inc., a biopharmaceutical firm listed on Nasdaq under the symbol NKTX, has announced the commencement of patient enrollment for its Ntrust-2 clinical trial. This trial aims to evaluate the efficacy of NKX019, the company’s innovative chimeric antigen receptor (CAR) NK-cell therapy, in treating various autoimmune diseases such as systemic sclerosis (SSc), idiopathic inflammatory myopathy (IIM), and ANCA-associated vasculitis (AAV). Additionally, the firm has received clearance for an investigator-sponsored trial (IST) to assess NKX019 in patients suffering from myasthenia gravis (MG).

NKX019 is a cutting-edge, off-the-shelf CAR NK-cell therapy designed to target and deplete CD19-positive cells, which play a role in B-cell mediated diseases. This therapy is intended to offer several benefits, including rapid B-cell elimination without the need for cell expansion, reduced risks of toxicities often associated with cell expansion, and the elimination of the need for fludarabine in lymphodepletion, thereby minimizing toxicity. Additionally, NKX019 supports on-demand dosing, allowing for repeated treatments as necessary.

Paul J. Hastings, the CEO of Nkarta, expressed enthusiasm for the promising potential of NKX019 in addressing a range of autoimmune diseases. He highlighted the ongoing efforts in clinical trials aimed at treating lupus nephritis and systemic lupus erythematosus, pointing to the broader applicability and potential benefits of NKX019 as a safe and accessible treatment option for autoimmune diseases.

The Ntrust-2 trial is a multi-center, open-label, dose-escalation study that builds upon previous academic research indicating the potential for durable, drug-free remissions in patients with autoimmune diseases following CD19-targeted cell therapy. This trial will evaluate NKX019 across three patient cohorts: those with SSc, IIM, and AAV. Participants will receive NKX019 on days 0, 3, and 7 after undergoing lymphodepletion with cyclophosphamide, a drug known for its safety in treating autoimmune diseases. The primary focus of the trial is to assess the safety and efficacy of NKX019 in promoting long-term remissions by effectively resetting the immune system through the eradication of pathogenic B cells.

The investigator-sponsored Phase 1 IST focuses on patients with myasthenia gravis and is led by Dr. Ali A. Habib, a Clinical Professor of Neurology at the University of California, Irvine, and other researchers. This trial will analyze the safety and clinical outcomes of NKX019, along with conducting translational and biomarker studies that include assessments of autoantibodies, cytokine profiles, and pharmacokinetics. Participants will receive NKX019 on days 0, 3, and 7 following lymphodepletion with cyclophosphamide.

Myasthenia gravis is an autoimmune disorder characterized by disrupted communication between nerves and muscles, leading to muscle weakness and fatigue. The condition is caused by the immune system producing antibodies that interfere with the neuromuscular junction. Currently, there is no cure for MG, and treatment often involves lifelong use of immunosuppressive medications. The development of cell therapies like NKX019 offers hope for changing the treatment landscape by potentially reducing the need for chronic treatments.

Preliminary data from the Ntrust-1 and Ntrust-2 studies are expected in 2025. Ntrust-1 focuses on patients with lupus nephritis, and both studies continue to enroll patients. The trials aim to leverage NKX019 to achieve long-term remissions in autoimmune diseases by resetting the immune system and eliminating pathogenic B cells.

Systemic sclerosis (SSc), a progressive autoimmune disease, is characterized by inflammation and hardening of the skin and other body parts. With approximately 100,000 people affected in the U.S., there are currently no treatments to halt or reverse the disease. IIM, including subtypes like dermatomyositis and immune-mediated necrotizing myopathy, affects around 50,000 U.S. residents and involves significant muscle damage and weakness. ANCA-associated vasculitis, impacting around 140,000 people in the U.S., causes severe damage to small blood vessels, often leading to life-threatening complications.

Overall, Nkarta is making significant strides in advancing NK cell therapies to address a range of autoimmune diseases, offering new hope for improved treatment outcomes and quality of life for patients.