On September 24, 2025 Artios Pharma Limited ("Artios"), a biopharmaceutical company committed to realizing the therapeutic power of targeting the DNA damage response ("DDR") in cancer, reported that the U.S. Food and Drug Administration (FDA) granted Fast Track designation to its ATR inhibitor, alnodesertib, in combination with a low dose of chemotherapeutic agent irinotecan, for the treatment of adult patients with ATM-negative metastatic colorectal cancer (mCRC) in the third-line setting (Press release, Artios Pharma, SEP 24, 2025, View Source [SID1234656187]).
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"The Fast Track designation for alnodesertib underscores its first-in-class potential for third-line mCRC patients with ATM-negative tumors," said Mike Andriole, Chief Executive Officer of Artios. "Approximately 3,000 patients with ATM-negative third-line mCRC succumb to this disease annually in the United States, with no treatment options that specifically address this protein deficiency. Alnodesertib has the potential to be the first treatment specifically for this invariably lethal disease. Additionally, we are encouraged by the durable responses this program has demonstrated across other tumor types, highlighting its ability to target replication stress across a range of solid tumors."
The designation is supported by encouraging results from the ongoing STELLA Phase 1/2a study, which is evaluating alnodesertib in combination with a low dose of irinotecan. Beyond third-line mCRC, clinical responses were observed in seven additional solid tumor types with ATM deficiency. The combination of alnodesertib plus low-dose irinotecan has a favorable safety profile to date, has been well tolerated, and has been shown to be suitable for long-term dosing.
"Patients with third-line colorectal cancer face a dismal prognosis, with current standards of care for third-line mCRC delivering response rates in the single digits. In our studies to date, alnodesertib has demonstrated compelling clinical activity in ATM-negative patients with mCRC as well as in other heavily pretreated cancer types with high endogenous replication stress," said Ian Smith, Chief Medical Officer of Artios. "These results, together with activity across other solid tumors, highlight alnodesertib’s potential to deliver meaningful benefit where treatment options are limited. The FDA’s Fast Track designation recognizes both the strength of our early clinical data and the urgent need for new therapies, while also providing the opportunity for enhanced interactions with the Agency."
The FDA’s Fast Track program is designed to facilitate the development and expedite the review of investigational drugs that demonstrate the potential to address unmet medical needs in serious or life-threatening conditions. Product candidates with Fast Track designation are eligible for priority review and accelerated approval if relevant criteria are met. The Fast Track designation for alnodesertib was granted based on early Phase 1/2 clinical studies that are currently ongoing in the United States. The designation will enable Artios to interact early and more frequently with the FDA to discuss alnodesertib’s development path.
About Alnodesertib
Alnodesertib, formerly known as ART0380, is a potential first-in-class, orally administered, selective small molecule inhibitor of ataxia-telangiectasia and Rad3-related protein (ATR). Artios’ differentiated approach combines alnodesertib with a low dose of the chemotherapy irinotecan, targeting cancers with high endogenous replication stress, such as those with ATM protein deficiency.