SAN DIEGO - Kura Oncology, Inc. (NASDAQ:KURA), a clinical-stage biopharmaceutical company, has announced that the U.S. Food and Drug Administration (FDA) has awarded Breakthrough Therapy Designation to its drug candidate ziftomenib for the treatment of relapsed/refractory NPM1-mutant acute myeloid leukemia (AML). This designation is based on preliminary clinical evidence suggesting ziftomenib may offer substantial improvement over existing therapies for this serious condition.
The FDA's decision is rooted in data from the ongoing KOMET-001 clinical trial, which explores the efficacy of ziftomenib in patients with this specific AML mutation. Acute myeloid leukemia is a prevalent type of acute leukemia in adults, characterized by the rapid growth of abnormal white blood cells, red blood cells, or platelets in the bone marrow.
NPM1-mutant AML accounts for around 30% of new AML cases each year. Despite high initial response rates to frontline therapy, patients often experience relapse, leading to poor survival outcomes. The median overall survival for patients in second, third, and fourth-line treatments is approximately 7.8, 5.3, and 3.5 months, respectively. Currently, no targeted therapies approved by the FDA are available for NPM1-mutant AML.
Ziftomenib is a once-daily, oral investigational drug that inhibits the menin-KMT2A/MLL protein-protein interaction, a pathway known to drive various genetic alterations in AML. In Phase 1 of the KOMET-001 study, ziftomenib has shown an encouraging safety profile and tolerability. A 35% complete remission rate was observed in patients treated at the recommended Phase 2 dose.
Kura Oncology is on track to complete the registration-directed trial of ziftomenib by mid-2024. The drug also holds Orphan Drug Designation from the FDA for the treatment of AML. Ziftomenib is being evaluated in combination with standard treatments in other ongoing clinical trials.
This breakthrough designation aims to expedite the development and review of ziftomenib by allowing more intensive guidance from the FDA, organizational commitment involving senior managers, and eligibility for rolling review and priority review.
The information in this article is based on a press release statement from Kura Oncology, Inc.
InvestingPro Insights
Amid the significant milestone achieved by Kura Oncology, Inc. (NASDAQ:KURA) with the FDA's Breakthrough Therapy Designation for ziftomenib, investors might be interested in the company's current financial health and market performance. As per real-time data from InvestingPro, Kura Oncology holds a market capitalization of roughly $1.34 billion USD. Despite the positive news, the company's Price/Earnings (P/E) Ratio stands at -8.14, reflecting its current lack of profitability. Furthermore, with an Operating Income of approximately -$165.8 million USD for the last twelve months as of Q4 2023, the financials indicate challenges in generating profits.
On the brighter side, two InvestingPro Tips highlight potential optimism for investors. Firstly, Kura Oncology holds more cash than debt on its balance sheet, which could provide financial flexibility and resilience. Secondly, three analysts have revised their earnings upwards for the upcoming period, suggesting a potentially improving outlook for the company's financial performance. Additionally, the Relative Strength Index (RSI) indicates that the stock is in oversold territory, which might attract investors looking for a potential rebound.
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