A startup spun-out of Cincinnati Children’s Hospital in 2019 has cleared its first major hurdle with the U.S. Food and Drug Administration. The clearance paves the way for the company's first clinical trial.
Hyde Park-based biotech Kurome Therapeutics announced last week its IND, or investigational new drug application, for KME-0584 was cleared by FDA. KME-0584 is a first-in-class investigational drug for patients with relapsed/refractory acute myeloid leukemia (AML), a rare blood cancer, and high-risk myelodysplastic syndromes (MDS), or a group of blood disorders.
The notice means the company can proceed with a Phase 1 clinical trial. Kurome said it plans to initiate that study across multiple sites starting in the latter half of 2024.
Jan Rosenbaum, Kurome’s CEO and chief scientific officer, told me last year the trial will be held at both large university hospitals and smaller community hospital sites. Its patient population is one that’s hard to procure, and difficult to treat.
Rosenbaum spent 23 years as a scientist at Procter & Gamble and five years as the chief scientific officer at Airway Therapeutics, a biopharma company that's developing interventions for acute and chronic lung disease.
This is the first drug she’s ever put into the clinic.
“This is a major milestone for the Kurome team and our collaborators and validates our unique approach to AML and MDS as well as the suitability of KME-0584 for initial clinical testing,” Rosenbaum said in a news release. “We look forward to getting our clinical trial underway and testing our approach.”
KME-0584 targets certain proteins in the body called IRAK1 and IRAK4 as well all mutations of FLT3s, a type of protein receptor (FLT3s work to regulate cell growth and proliferation). These proteins, or the pathways they regulate, become over-activated in certain cancers. That helps cancer cells expand and survive.
Inhibiting, or blocking, signals from both IRAK1 and IRAK4 could interfere with that ability, Rosenbaum said.
The Phase I study will evaluate the safety and tolerability of KME-0584. Kurome said the drug is intended to be taken by mouth either alone or as a combination therapy with azacytidine, a type of chemotherapy drug, or venetoclax, a treatment for AML.
The study will include a dose escalation and an expansion phase with up to 100 total participants.
Kurome was founded in November 2019 as a spinoff of Cincinnati Children’s and landed its first round of funding in February 2020.
The company has raised $25 million in venture funding to date, including a $15 million Series A in 2021.
Kurome is backed by Avondale-based seed stage fund CincyTech and Queen City Angels locally as well as Medicxi, a European-based life sciences investment firm, and Affinity Asset Advisors, a New York hedge fund focused in the biotech and health care space.
