Driven by advances over the last 20 years in cancer biology, medicinal chemistry, and data sciences, the field of cancer therapy has begun to transition from non-targeted approaches, such as surgery, chemotherapy and radiation, to more targeted, genetic-based approaches.
For some patients, these targeted therapies have been transformative in allowing them to achieve long-term durable responses. However, for the majority of people with cancer, no highly effective precision therapies exist. We believe precision drug development has not yet fully harnessed all the scientific breakthroughs of the past two decades.
We have built a scalable precision oncology drug-hunting platform that fully integrates the most advanced technologies and is commanded by an experienced team of recognized leaders in their respective fields, to extend the frontiers of oncology research and drug development.
Our team of innovators includes experts in virtually all cutting-edge techniques used today in medicinal chemistry, computational chemistry, cancer biology, genetics, proteomics, and biophysics. We integrate these techniques, our expertise, and our insight in our quest for first-in-class or best-in-class oncology therapeutics for underserved patient groups.
Precision Oncology 2.0 aims to redefine the frontier of precision medicine to deliver optimized and transformational therapies for larger populations of people with cancer. We believe the full integration of the most advanced technologies across cancer biology, medicinal chemistry and data sciences into one platform and its customized application enables us to create exquisitely selective small molecule compounds consistently and rapidly against an unprecedented spectrum of targets. We use our platform to advance a broad pipeline of optimized compounds across three target categories: best-in-class molecules targeting clinically validated oncogenes; first-in-class molecules for previously undruggable targets; and first-in-class molecules for novel cancer targets. We plan to efficiently progress these novel compounds through the discovery process and into clinical trials with the goal of achieving rapid clinical proof-of-concept in well-defined patient populations.
Our approach provides key advantages to the discovery of novel medicines for people with cancer at an unparalleled scale, including:
- Increased discovery efficiency to deliver medicines to patients faster
- Increased chemical selectivity for a wider therapeutic window
- Increased spectrum of targets for drug discovery
Our Drug Hunting Platform
Our fully integrated, scalable precision oncology drug-hunting platform helps advance a broad pipeline of homegrown, exquisitely selective small molecule compounds against a wide variety of biologically validated targets. We use these next-generation drug discovery technologies to identify targets, lead compounds, and clinical candidates at a faster pace, with better molecule quality and against more targets than previously possible. Many of these next-generation technologies have only recently become available, and we believe we are a leader in successfully integrating all of these tools and advances to realize our vision of Precision Oncology 2.0.
Our lead programs are designed to be best-in-class, targeting clinically-validated, mutated oncogenes in patients with significant unmet medical need.
STX-H1047-PI3Kα: PI3Kα is an established cancer target and one of the most highly mutated targets in cancer, particularly in solid tumors. The mutations at the H1047 residue represent the highest frequency of mutations in PI3Kα. More than 55,000 people in the United States annually are diagnosed with cancers driven by mutations at this residue. Approved therapies targeting PI3Kα are limited by inhibition of the normal, or wild-type, version of PI3Kα in healthy tissues, leading to significant metabolic side effects that hinder the ability of patients to tolerate these therapies, and by an inability to treat tumors that have progressed into the central nervous system.
Using our drug-hunting platform, we have discovered a novel allosteric binding pocket that allows for specific targeting of the mutant over the wild-type form of PI3Kα with a small molecule. This may allow for maximal inhibition of the mutant protein in cancer cells compared to normal tissues, to avoid the off-target effects – such as metabolic dysfunction – that are associated with wild-type inhibition by commercially available options. STX-H1047-PI3Kα is designed to be a central nervous system-penetrant, oral pill. In preclinical studies, STX-H1047-PI3Kα has demonstrated exquisite in vitro selectivity and dose-dependent anti-tumor activity, without evidence of hyperglycemia in multiple model systems. Scorpion expects to submit an IND for STX-H1047-PIK3α in 2023.
STX-EGFR-EXON20: NSCLC is the most common form of lung cancer and EGFR mutations are one of the most common mutations in NSCLC. NSCLC tumors that express EGFR with Exon 20 insertion mutations, have an incidence of approximately 3,400 patients per year in the United States. Commercially-available therapies for NSCLC patient with EGFR Exon 20 insertion mutations are limited by significant toxicities associated with the inhibition of wild-type, EGFR protein in healthy tissues such as the skin and gut, leaving a significant unmet need for those patients.
Leveraging our discovery platform, we have identified highly differentiated chemical matter that provides exquisitely selective inhibition of Exon 20 insertion mutations compared to the wild-type form of the protein. This may allow for maximal inhibition of the mutant protein in cancer cells compared to normal tissues, thereby reducing the toxicities – often gastrointestinal or skin-related – that lead to dose limitations or reductions with existing EGFR Exon 20 inhibitors. STX-EGFR-EXON20 is designed as an oral pill, which in preclinical studies demonstrated best-in-class selectivity and dose-dependent anti-tumor activity in xenograft models at well-tolerated doses. Scorpion expects to submit an IND for STX-EGFR-EXON20 in 2023.