Press Releases

Loxo Oncology Initiates Phase 1 Trial of LOXO-101, a Selective Trk Inhibitor

Released on May 01, 2014

Stamford, CT (May 1, 2014) – Loxo Oncology, Inc., a biopharmaceutical company focused on targeted cancer therapies for genetically-defined populations, announced today that it has initiated a Phase 1 Study for its lead compound, LOXO-101, a potent and selective inhibitor of the tropomyosin kinase (TRK) family of receptors. The Phase 1 study will evaluate the safety, pharmacokinetics and pharmacodynamics of escalating doses of LOXO-101 and, in a second stage, will also provide a preliminary assessment of anti-tumor activity in cancer patients preselected to have an alteration in genes affecting the TRKA, TRKB or TRKC proteins. “We are very excited to begin the clinical evaluation of LOXO-101, which has been specifically designed to block TRK signaling,” said Lori A. Kunkel, MD, chief medical officer of Loxo Oncology. “Increasingly, TRK alterations are being described across human cancer, and in many cases, alongside known, validated oncogenes in a range of malignancies, including lung cancer, thyroid and neural crest-derived tumors. We believe these patients need more specific and effective treatments.”

“The advancement of LOXO-101 into the clinic is an important milestone for us. It exemplifies our approach to targeted drug development – the application of compelling chemistry to a promising target in a genetically-defined patient population,” said Josh Bilenker, MD, chief executive officer and president at Loxo Oncology. “We look forward to working with patients and key thought leaders to evaluate the potential of this and other exciting developmental therapeutics.”

Loxo Oncology retains all development and commercialization rights to its TRK inhibitor program, which includes LOXO-101. About TRK The genes NTRK1, NTRK2 and NTRK3 encode the receptor tyrosine kinases TRKA, TRKB and TRKC, respectively. These receptors bind ligands known as neurotrophins, which are important to the development and function of the nervous system. Translocations involving the TRK kinase domain, mutations in the TRK ligand-binding site, amplifications of NTRK, TRK splice variants and autocrine/paracrine signaling have been described in diverse tumor types such as non-small cell lung carcinoma, papillary thyroid carcinoma, glioblastoma and acute myelogenous leukemia. Research suggests that targeted inhibition of TRK has therapeutic potential in many of these tumor types. In a recent article, Oncogenic and drug-sensitive NTRK1 rearrangements in lung cancer, published in Nature Medicine, Vaishnavi et al describe novel TRK fusions in lung cancer and demonstrate potent growth inhibition in relevant models with one of the many TRK inhibitors licensed to Loxo Oncology.

About Loxo Oncology

Loxo Oncology was incorporated in Delaware in May 2013 and was founded by Josh Bilenker, MD Loxo Oncology is committed to bringing targeted cancer therapies rapidly into the clinic that have an opportunity for clinical success in genetically defined patient populations. Loxo Oncology derives its company name from an attendant of the Greek goddess Artemis, who represented the concept of trajectory in the sport of archery. For more information, visit

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