Five of Six Evaluable Patients with TRK Fusion Cancers Achieve Confirmed RECIST Partial Responses; All Six Demonstrate Significant Tumor Regressions
All TRK Fusion Patients Remain on Study, With Longest Follow-up Beyond One Year
Company to Host Investor Conference Call and Webcast on
LOXO-101 Phase 1 study investigators reported that, as of the
Six patients with TRK fusion cancers had been on study sufficiently long for their first efficacy assessment, and all six exhibited significant tumor regressions. A seventh patient with a TRK fusion cancer was enrolled more recently and thus had not yet been evaluated for response as of the data cutoff date, though the patient remains on study. Five of the six efficacy evaluable patients achieved a confirmed partial response, as defined by standard RECIST criteria. The sixth patient demonstrated clear radiographic tumor regressions, including in the central nervous system, but has not met the threshold required for a RECIST response. Tumor regressions have been observed in five different anatomically-defined cancers: sarcoma, gastrointestinal stromal tumor, mammary analogue secretory cancer of the salivary glands, thyroid cancer and non-small cell lung cancer. No TRK fusion patients have progressed, with one patient in cycle 14, two patients in cycle 10 and three patients in cycle 7, as of the data cutoff date. In addition, LOXO-101 has been highly active and well-tolerated at doses that include the recommended Phase 2 dose of 100 mg BID. The majority of adverse events reported by the investigators have been mild to moderate. A maximum tolerated dose (MTD) has not been defined.
The responses, durability and safety data with LOXO-101 clearly suggest that this is an important drug candidate for patients with TRK fusion cancers, said
The consistent efficacy of LOXO-101 in patients with TRK gene fusions, independent of tumor type, is very exciting, said
LOXO-101 Phase 1 Results
LOXO-101 is currently being evaluated in an ongoing dose-escalation Phase 1 trial in patients with solid tumors refractory to standard therapy. As of
LOXO-101 has been well tolerated in the 43 patients treated, including 24 patients at a dose of 100mg BID. Adverse events reported regardless of attribution to study drug are generally consistent with those previously presented. Grade 1 and 2 adverse events include fatigue (33 percent), constipation (23 percent) and dizziness (23 percent). Grade 3 adverse events included fatigue, constipation, anemia, increased liver enzymes, dyspnea, abdominal pain, hypertension, hyperkalemia, delirium, pleural effusion, and syncope. No Grade 4 adverse events have been reported. The frequency of toxicities did not correlate with dose level. The maximum tolerated dose (MTD) has not yet been defined.
- Soft tissue sarcoma, LMNA-NTRK1 fusion: Confirmed partial response, remains on study in cycle 14 at a dose of 100 mg BID
- Gastrointestinal stromal tumor, ETV6-NTRK3 fusion: Confirmed partial response, remains on study in cycle 10 at a dose of 150 mg BID
- MASC, ETV6-NTRK3 fusion: Confirmed partial response, remains on study in cycle 10 at 100 mg BID
- MASC, ETV6-NTRK3 fusion: Confirmed partial response, remains on study in cycle 7 at 100 mg QD; this patient started therapy on 100 mg BID and dose-reduced early in cycle 1 to 100 mg QD due to transient dizziness possibly related to drug
- Papillary thyroid cancer, ETV6-NTRK3 fusion: Confirmed partial response, remains on study in cycle 7 at 100 mg BID
- Non-small cell lung cancer, TPR-NTRK1 fusion: 18% tumor regression of bone-only RECIST evaluable disease (stable disease), remains on study in cycle 7 at 100mg BID
All six patients remain on study as of
Upcoming Milestones for
- Continued enrollment of the LOXO-101 Phase 2 global, multi-center, single-arm, open-label basket trial in adult patients with solid tumors that harbor a TRK fusion, with an enrollment update expected in the second half of 2016.
- Initiation of a Phase 1 study of a selective RET inhibitor in late 2016 or early 2017.
- Initiation of a Phase 1 study of next-generation TRK inhibitor LOXO-195, addressing previously-treated patients with acquired resistance, in 2017.
Conference Call and Webcast Information
LOXO-101 is a potent, oral and selective investigational new drug in clinical development for the treatment of patients with cancers that harbor abnormalities involving the tropomyosin receptor kinases (TRKs). Growing research suggests that the NTRK genes, which encode for TRKs, can become abnormally fused to other genes, resulting in growth signals that can lead to cancer in many sites of the body. In an ongoing Phase 1 clinical trial, LOXO-101 has demonstrated encouraging preliminary efficacy. LOXO-101 is also being evaluated in a global Phase 2 multi-center basket trial in patients with solid tumors that harbor TRK gene fusions and a Phase 1 trial in pediatric patients. For additional information about the LOXO-101 clinical trials, please refer to www.clinicaltrials.gov. Interested patients and physicians can contact the Loxo Oncology Physician and Patient Clinical Trial Hotline at 1-855-NTRK-123.
About Loxo Oncology
Loxo Oncology is a biopharmaceutical company innovating the development of highly selective medicines for patients with genetically defined cancers. Our pipeline focuses on cancers that are uniquely dependent on single gene abnormalities, such that a single drug has the potential to treat the cancer with dramatic effect. We believe that the most selective, purpose-built medicines have the highest probability of maximally inhibiting the intended target, thereby delivering best-in-class disease control and safety. Our management team seeks out experienced industry partners, world-class scientific advisors and innovative clinical-regulatory approaches to deliver new cancer therapies to patients as quickly and efficiently as possible. For more information, please visit the company's website at www.loxooncology.com.
Forward Looking Statements
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