First Patient Dosed in Clinical Trial of Mutant-Selective PI3Kα Inhibitor to Combat Breast Cancer and other Solid Tumors, Developed by Scorpion Therapeutics

Scorpion Therapeutics, Inc. is proud to announce that the first patient has been dosed in their Phase 1/2 first-in-human clinical trial to evaluate the efficacy of STX-478, an allosteric and central nervous-system penetrant inhibitor of mutant phosphoinositide-3-kinase alpha.

This innovative drug has the potential to revolutionize precision medicine and provide an effective treatment for a variety of solid tumors, including breast and gynecological cancers, head and neck squamous cell carcinoma, and more. The trial will assess STX-478 as a monotherapy and as part of a combination therapy with approved agents for HR+/HER2- breast cancer.

STX-478 may offer a much-needed solution for the over 166,000 patients in the United States alone who are affected by tumors harboring PI3Kα mutations each year. Promising preclinical results have demonstrated the drug’s activity against both kinase and helical domain mutated tumors, making it an attractive target for drug discovery. With its potential to improve outcomes and address the scarcity of available treatment options, STX-478 is a promising development in the fight against cancer.

Scorpion is thrilled to have initiated clinical evaluation of STX-478, their mutant-selective PI3Kα inhibitor with a potentially superior profile, for the treatment of HR+/HER2- breast cancer and a selection of other solid tumors. It has only been three years since the company was founded, making this clinical trial a testament to their Precision Oncology 2.0 strategy and the capabilities of the scientific team.

The speed at which this program has advanced from target selection to clinical development is a testament to the quality of the compound and the expertise of the team in creating potentially life-changing therapies.

Treatment options for PI3Kα-mutated cancers are limited and often accompanied by significant side effects, such as hyperglycemia and rash, which can make long-term use difficult. Even more concerning is the lack of CNS penetrance of these treatments, despite the fact that up to half of all solid tumor patients face significant morbidity and mortality from brain metastases.

Scorpion’s Chief Medical Officer, Michael Streit, M.D., is excited to announce the initiation of a Phase 1/2 trial for STX-478, a wild-type-sparing, CNS-penetrant, oral inhibitor of mutant PI3Kα with excellent pre-clinical pharmacokinetic properties.

Results from this trial could potentially differentiate STX-478 from existing agents already on the market or in development—data from this trial will show the effect of STX-478 on potential biomarkers that could indicate anti-tumor activity, as well as its safety, pharmacokinetic, and pharmacodynamic properties.

If successful, STX-478 could provide a wider therapeutic window and greater efficacy for the treatment of solid tumors, especially HR+/HER2- breast cancer, and will be presented in 2024. Scorpion is looking forward to partnering with study investigators to evaluate STX-478 in patients with PI3Kα-mutated cancers.

About STX-478 Phase 1/2 Trial

Scorpion’s Phase 1/2 clinical trial is a pioneering study with the goal of assessing the safety and tolerability of STX-478 as a monotherapy and as a combination agent for multiple indications such as breast cancer, gynecological cancers, HNSCC, gastrointestinal cancers and other solid tumors, all harboring PI3Kα-mutations.

The trial is open-label, multi-center and designed to characterise the safety profile of STX-478 and identify a maximum tolerated dose (MTD) or a lower optimal-biologically active dose (RP2D). Secondary objectives for this bold venture include assessing the pharmacokinetic/pharmacodynamic effects and clinical response as measured by RECIST version 1.1. With this trial, Scorpion is striving to revolutionize the treatment of HR+/HER2- breast cancer driven by PI3Kα-mutations.

About Scorpion Therapeutics

Scorpion is revolutionizing the world of precision medicine with its pioneering platform of advanced cancer biology, medicinal chemistry, and data science technologies. Through this comprehensive system, they are consistently and rapidly creating small molecule compounds that are exquisitely selective and optimized to target an unprecedented spectrum of oncogenes and previously undruggable targets.

This leading-edge approach to cancer treatment, aptly named Precision Oncology 2.0, is aiming to develop a broad pipeline of wholly owned compounds that will transform the lives of larger populations of cancer patients.

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