SYNCAR-001+STK-009(Synthekine)

MENLO PARK, Calif.--( BUSINESS WIRE )-- Synthekine Inc ., an engineered cytokine therapeutics company, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to SYNCAR-001 + STK-009, its CD19 CAR-T and orthogonal IL-2 investigational therapy, for the treatment of patients with severe, refractory systemic lupus erythematosus (SLE), without the use of lymphodepletion. SYNCAR-001 + STK-009 is a cytokine-inducible cell therapy regimen built on Synthekine’s proprietary ortho IL-2 technology. It is a two-component therapy consisting of SYNCAR-001, an autologous CD19-targeting chimeric antigen receptor T cell (CAR-T) which expresses an engineered IL-2 receptor allowing it to selectively receive a signal from STK-009, an engineered IL-2 cytokine. SYNCAR-001 + STK-009 has been administered to patients with CD19+ hematologic malignancies in an ongoing Phase 1 study ( NCT05665062 ). The FDA recently cleared an IND application for a Phase 1 study ( NCT06544330 ) of SYNCAR-001 + STK-009 in non-renal SLE and lupus nephritis (LN). “Recent clinical studies have shown CD19 targeted cell therapies can be a potentially effective treatment option for SLE. However, these therapies typically require lymphodepleting chemotherapy that introduce a wide range of toxicities, including cytopenias and infections, for this patient population,” said Debanjan Ray, chief executive officer of Synthekine. “Fast Track designation from the FDA allows us to more rapidly advance our cytokine-inducible CD19 CAR-T cell therapy. Eliminating lymphodepletion and its associated risks opens the door to a broader population of patients with lupus and other autoimmune diseases, and we look forward to accelerating our efforts to bringing SYNCAR-001 + STK-009 to those individuals in need.” The multi-center, dose escalation clinical trial will assess the safety and clinical activity of SYNCAR-001 + STK-009 in patients with non-renal SLE and LN. Patients will be treated with a one-time, fixed dose of SYNCAR-001 cells and a defined course of STK-009 via weekly subcutaneous injection. Fast Track designation is intended to aid the development and expedite the review of drugs to treat serious and life-threatening conditions with unmet medical needs, with the goal of reaching patients earlier. A therapeutic candidate that receives Fast Track designation may be eligible for more frequent interactions with the FDA to discuss the therapeutic candidate’s clinical trial designs and development plans. About Systemic Lupus Erythematosus (SLE) Systemic lupus erythematosus (SLE) is a chronic autoimmune disease that causes inflammation and potentially irreversible damage to affected organs. SLE manifests a wide range of symptoms with varying degrees of severity in about 200,000 U.S. adults, making it difficult to diagnose. Currently, there is no known cure for SLE and commonly used treatments including nonsteroidal anti-inflammatory drugs (NSAIDs), corticosteroids, and other immunosuppressants have limited benefit. About Lupus Nephritis (LN) Up to 50% of patients with SLE can develop lupus nephritis, a type of kidney disease that often starts at the same time or shortly after non-kidney related SLE symptoms. Between 10 to 30 percent of people who have lupus nephritis develop kidney failure. Current treatment options aim to reduce inflammation in the kidneys, however less than half of affected patients using these therapies achieve complete responses. About Synthekine Synthekine is harnessing the potential of cytokine therapeutics to develop selective immunotherapies designed to improve the treatment paradigm of cancer and inflammatory disease. Using insights on cytokine structure and function, the company engineers therapeutics designed to unlock the full efficacy potential of cytokines while avoiding their associated toxicities. Synthekine is applying principles of cytokine partial agonism and immunological specificity across multiple protein engineering platforms to create a broad and deep pipeline of product candidates. These novel immunotherapies include modified cytokines, cytokine-enhanced cell therapies and surrogate cytokine agonists. For more information, visit www.synthekine.com , and follow us on Twitter @ synthekine and LinkedIn .

SYNCAR-001 + STK-009 combination will be studied in two distinct lupus patient populations: non-renal systemic lupus erythematosus (SLE) and lupus nephritis (LN) MENLO PARK, Calif.--(BUSINESS WIRE)-- Synthekine Inc., an engineered cytokine therapeutics company, today announced that the U.S. Food and Drug Administration (FDA) has cleared an Investigational New Drug (IND) application to evaluate SYNCAR-001 + STK-009, its orthogonal IL-2 and CD19 CAR-T combination therapy, in patients with non-renal systemic lupus erythematosus (SLE) and lupus nephritis (LN), without the use of any lymphodepletion. SYNCAR-001 + STK-009 is a cytokine-inducible cell therapy regimen built on Synthekine’s proprietary orthoIL-2 technology. It is a two-component therapy consisting of SYNCAR-001, an autologous CD19-targeting chimeric antigen receptor T cell (CAR-T) which expresses an engineered IL-2 receptor allowing it to selectively receive a signal from STK-009, an engineered pegylated IL-2 cytokine. The SYNCAR-001 + STK-009 combination therapy is in a Phase 1 study (NCT05665062) in CD19+ hematologic malignancies. “Recent studies have shown that CD19 targeted cell therapies can make a meaningful impact on non-renal SLE, LN and other autoimmune conditions,” said Naiyer Rizvi, M.D., chief medical officer of Synthekine. “However, successful treatment with CD19 CAR-Ts requires patients to be lymphodepleted, a significant burden on the patient, physician and healthcare system. By leveraging Synthekine’s orthoIL-2 technology, STK-009 has the potential to drive SYNCAR-001 cell engraftment and controlled cell expansion without lymphodepletion. We look forward to bringing our highly differentiated treatment option to patients.” The multi-center, dose escalation clinical trial will assess the safety and clinical activity of SYNCAR-001 + STK-009 in patients with non-renal SLE and LN. Patients will be treated with a one-time, fixed dose of SYNCAR-001 cells and a limited course of STK-009 via weekly subcutaneous injection. The first patient is expected to be enrolled in the second half of 2024. “Lymphodepleting chemotherapy may increase the risk to patients with SLE who already have significant comorbidities. This requirement also limits access to many patients who have marrow and organ dysfunction related to their underlying SLE,” said Amit Saxena, M.D., associate professor of medicine at NYU Grossman School of Medicine. “Synthekine’s cytokine-inducible CD19 CAR-T cell therapy has the potential to address critical segments of the patient population who face these barriers and offer them the remarkable potential benefit of this breakthrough approach to treating a devastating disease.” About Systemic Lupus Erythematosus (SLE) Systemic lupus erythematosus (SLE) is a chronic autoimmune disease that causes inflammation and potentially irreversible damage to affected organs. SLE manifests a wide range of symptoms with varying degrees of severity in about 200,000 U.S. adults, making it difficult to diagnose. Currently, there is no known cure for SLE and commonly used treatments including nonsteroidal anti-inflammatory drugs (NSAIDs), corticosteroids, and other immunosuppressants have limited benefit. About Lupus Nephritis (LN) Up to 50% of patients with SLE can develop lupus nephritis, a type of kidney disease that often starts at the same time or shortly after non-kidney related SLE symptoms. Between 10 to 30 percent of people who have lupus nephritis develop kidney failure. Current treatment options aim to reduce inflammation in the kidneys, however less than half of affected patients using these therapies achieve complete responses. About Synthekine Synthekine is harnessing the potential of cytokine therapeutics to develop selective immunotherapies designed to improve the treatment paradigm of cancer and inflammatory disease. Using insights on cytokine structure and function, the company engineers therapeutics designed to unlock the full efficacy potential of cytokines while avoiding their associated toxicities. Synthekine is applying principles of cytokine partial agonism and immunological specificity across multiple protein engineering platforms to create a broad and deep pipeline of product candidates. These novel immunotherapies include modified cytokines, cytokine-enhanced cell therapies and surrogate cytokine agonists. For more information, visit , and follow us on Twitter @synthekine and LinkedIn.