Kuur Therapeutics Announces Publication of Interim Phase 1 Data for CAR-NKT Cell Therapy KUR-501 in Nature Medicine

Kuur Therapeutics, a leader in the development of off-the-shelf CAR-NKT cell immunotherapies for the treatment of solid and hematological malignancies, today announced the publication in Nature Medicine of interim findings from its ongoing phase 1 GINAKIT2 clinical trial collaboration with Baylor College of Medicine and Texas Children’s Hospital, in high risk relapsed refractory (R/R) patients with neuroblastoma, a form of childhood cancer.

The interim results demonstrated that expressing the CAR with interlukin-15 (IL-15), a natural protein that supports NKT survival, enhanced the tumor-fighting capabilities and in vivo persistence of autologous NKT cells. Two of three patients studied showed tumor reduction following CAR-NKT infusion: one classified as stable disease and the other as a partial response. Imaging revealed a dramatic reduction in the size and metabolic activity of bone metastases in the patient with the partial response. CAR-NKT cells demonstrated a favorable safety profile and localized to the site of the neuroblastoma tumors.

Kuur is the first company to test CAR-NKT cell therapy in patients. The company’s revolutionary platform engineers CARs on invariant NKT cells, a subset of T lymphocytes. NKT cells represent the next generation of CAR therapy, because this innovative approach harnesses the innate tumor-homing properties of NKT cells, a specialized type of lymphocyte that eliminates tumor-supportive macrophages, activates anti-tumor NK, dendritic and CD8 T cells, and does not induce graft versus host disease when used in an allogeneic setting.

“These results validate the biology of CAR-NKT cells in that they home to tumor and marrow, expand and have tumor killing properties. They also demonstrate safety and enhanced tumor homing capabilities, offering distinct advantages over other cell types for the treatment of solid and hematological tumors,” said Kurt C. Gunter, MD, CMO of Kuur. “Using our novel engineering platform, we have manufactured CAR-NKT cells with high purity and added IL-15 to the CAR construct, which further increases in vivo persistence and anti-tumor activity. We look forward to continuing our research with the experts in cellular and gene therapy at Baylor as we aim to leverage the CAR-NKT cell approach to create more precise and effective therapies for cancer patients, including allogeneic therapies.”

The results published were derived from the three heavily pre-treated, R/R metastatic neuroblastoma patients in dose level 1 (3×106 CAR-NKTs/m2) of the GINAKIT2 clinical study. These data were originally presented at the 23rd Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) in May 2020. The trial is currently enrolling at the fourth and highest dose level.

About KUR-501

KUR-501 is an autologous product in which natural killer T (NKT) cells are engineered with a chimeric antigen receptor (CAR) targeting GD2, which is expressed on almost all neuroblastoma tumors. KUR-501 is also designed to address key limitations of current CAR immune cell therapies by secreting the cytokine IL-15, which has been shown in nonclinical studies to increase the persistence of CAR-NKT cells and improve their efficacy within the immunosuppressive tumor microenvironment. KUR-501 is being tested in the phase 1

Better Buy: CRISPR Therapeutics vs. Editas Medicine

You can’t use traditional means to compare CRISPR Therapeutics (NASDAQ:CRSP) and Editas Medicine (NASDAQ:EDIT) because they are emerging biotech companies with little revenue, and several years between their current efforts and any form of profitability.

Both of these companies are developing medicines that edit genes, using the same CRISPR technology. The system is capable of eliminating mutated DNA that cause a particular disease, such as sickle-cell anemia, some types of blindness, or Alzheimer’s.

The potential for CRISPR gene-editing is enormous. A study by Market Insights puts the global gene therapy market at $18.1 billion by 2027, giving it a compound annual growth rate (CAGR) of 25.7%. Figuring out which gene-editing biotech to invest in depends less on their fundamentals and more on which one seems most capable of developing marketable therapies based on the available technology.

Hands of scientists working with test tubes in a laboratory.

Image source: Getty Images.

The case for CRISPR

CRISPR Therapeutics has certainly been kinder to shareholders since January. Its share price is up over 37% year to date, while the share price for Editas has been flat. Much of the enthusiasm surrounding CRISPR Therapeutics stems from two of its therapies that have shown progress this year.

The first is CTX001, which CRISPR has been developing with Vertex Pharmaceuticals (NASDAQ:VRTX) as a treatment for two genetic blood disorders: severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). According to CRISPR Therapeutics, CTX001 edits a patient’s stem cells from within their bone marrow and instructs those cells to produce high levels of fetal hemoglobin (HbF) in their red blood cells. HbF, a hemoglobin that carries oxygen, is present at birth but is replaced with the adult form of hemoglobin by the body as we age. The thought is fetal HbF will eliminate the need for transfusion in TDT patients and will help reduce the number of sickle crises experienced by SCD patients.

CTX001 is in two phase 1/2 trials: CLIMB-111 and CLIMB-121. CLIMB-111 involves five TDT patients and CLIMB-121 has enrolled two SCD patients. While the sample sizes are obviously small, the company says that all seven patients so far have achieved blood platelet engraftment. Based on those results, the U.S. Food and Drug Administration (FDA) has given CTX001 Orphan Drug and Fast Track Designation for both SCD and TDT.

CRISPR Therapeutics listed only $44,000 in collaborative revenue in the second quarter, but the important number is the company’s $945 million in cash reserves. At the company’s cash burn rate, these reserves can last nearly three years before the company needs an infusion of cash or a profitable therapy.

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The case for Editas Medicine

On Aug. 25, the FDA granted Editas Medicine’s therapy, EDIT-301, rare pediatric disease (RPD) designation to treat sickle cell disease. That means the company gets a voucher from the FDA to receive a priority review on a different drug or therapy. The FDA does this to encourage research into medications that affect fewer than 200,000 people in the United States.

Like CRISPR, Editas is getting help from