STORM Therapeutics Presented Interim Phase 1 Data on STC-15 at ASCO 2024

STORM Therapeutics Ltd. recently shared promising interim Phase 1 clinical data for its groundbreaking lead product, STC-15, at the American Society of Clinical Oncology (ASCO) Annual Meeting. The company's pioneering approach involves using RNA modifications to treat a variety of diseases.

The Phase 1 study aims to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and clinical activity of STC-15, a highly selective METTL3 inhibitor. METTL3 is an RNA methyltransferase enzyme, targeting which represents a novel approach in cancer treatment. The data presented involved 33 patients with advanced malignancies who were enrolled in five different dose escalation cohorts, ranging from 60mg to 200mg. These patients received either daily or thrice-weekly oral doses of STC-15.

According to the data, STC-15 was well tolerated across the tested dose ranges. Common treatment emergent adverse events (TEAEs) were manageable and included thrombocytopenia, rash, and pruritus. Out of the 27 patients evaluable for response assessment, 11% showed partial responses, and an additional 63% exhibited disease control. These findings underscore the drug's clinical activity.

Significant reductions in methylated polyA-RNA were observed across all cohorts, indicating effective METTL3 target engagement and a rapid PD effect. Further PD biomarker analysis revealed activation of interferon signaling and innate immune responses, bolstering the drug's potential efficacy.

The study identified the recommended Phase 2 doses as being between 60mg and 200mg, administered three times per week. The advancement of STC-15 into combination studies with checkpoint inhibitors is anticipated to begin later in 2024.

Jerry McMahon, CEO of STORM Therapeutics, emphasized the promising nature of the Phase 1 data, highlighting STC-15's excellent safety profile and pharmacologic properties. The company's strategy includes expanding clinical trial designs to involve patients with various types of cancer, such as lung, melanoma, head and neck, and endometrial cancer.

Chief Medical Officer Josi Holz expressed satisfaction with the progress indicated by the data, noting that STC-15 shows promising signs of tolerability and clinical activity. Holz also looked forward to completing the current Phase 1 study and integrating checkpoint inhibitors in future research.

Principal Investigator Justin Moser from HonorHealth Research Institute remarked on the encouraging preliminary results, suggesting that METTL3 inhibition presents a novel method to activate the innate immune system, potentially treating a range of tumor types.

The data supports the continued development of STC-15 for patients with advanced solid tumors and shows promise for further clinical studies. The ongoing Phase 1 study aims to determine optimized active doses, with details available on clinicaltrials.gov under identifier NCT05584111.

STORM Therapeutics is recognized for its pioneering work in cellular reprogramming through RNA modifications. The company's expertise in RNA modifying enzymes has led to the development of innovative small molecule drugs targeting cancer, inflammation, viral infections, and CNS diseases. STC-15 is the first RNA modifying enzyme inhibitor to reach human clinical trials and is currently being evaluated for safety and efficacy in patients with solid tumors. Preclinical data have also indicated that METTL3 inhibition induces immune responses and interferon pathways, contributing to tumor cell destruction and improving anti-tumor properties when combined with checkpoint inhibitors. STORM plans to advance additional RME inhibitor programs into clinical development by 2024.

The company's efforts are backed by prominent investors like M Ventures, Pfizer Ventures, and others, aiming to accelerate the development of these novel drugs. STORM seeks collaboration to extend its groundbreaking therapies beyond oncology, enhancing treatment possibilities for various diseases.

In conclusion, STC-15’s Phase 1 data suggest that the drug is safe, well-tolerated, and shows significant pharmacological activity in advanced malignancies. The next steps include further studies and combination trials with checkpoint inhibitors, aiming to offer new hope in cancer treatment.