Lilly’s $8 Billion Bet Pays Off With Dazzling Results in Lung Cancer Trial

Eli Lilly

When Eli Lilly and Company acquired Loxo Oncology for $8 billion, the company was gambling on several pipeline drugs in oncology. One was Vitrakvi (larotrectinib), which was approved by the U.S. Food and Drug Administration and is commercialized by Bayer.

Today, Lilly released data from another Loxo pipeline drug, LOXO-292, which was being investigated in the LIBRETTO-001 clinical trial. LIBRETTO-001 is a Phase I/II clinical trial. Phase I is a dose-escalation phase and Phase II is a dose-expansion phase. The primary endpoint of Phase II is overall response rate (ORR). Secondary endpoints are duration of response (DOR), progression-free survival (PFS) and safety.

LOXO-292 (selpercatinib) was being evaluated as a monotherapy for the treatment of RET fusion-positive non-small cell lung cancer (NSCLC). In the registration dataset made up of the first 105 patients with RET fusion-positive NSCLC with previous platinum-based chemotherapy, the drug showed a 68% ORR.

The patient group was heavily pretreated with a median of three previous systemic treatments—55% were treated with a checkpoint inhibitor and 48% received at least one multikinase inhibitor. The ORR was similar no matter which therapy they received previously.

“In this large cohort, selpercatinib’s response rate, durability, robust CNS activity, and safety shows promise,” said Alexander Drilon, lead investigator with the Memorial Sloan Kettering Cancer Center in New York City. “Furthermore, this continues to confirm that RET fusions are clinically targetable alterations, placing them in the company of activating EGFR/ALK/ROS1 alterations. We are encouraged by these data as there is currently an unmet need to provide genomically-tailored therapy to patients with RET fusion-positive NSCLCs.”

The company also released data of the drug in treatment-naive RET fusion-positive NSCLC. They analyzed 34 patients, and observed the drug had an 85% ORR. As yet, median duration of response (DOR) and progression-free survival (PFS) haven’t been reached in the patient population.

“We’re seeing the importance of precision medicines, designed for specific patients, grow in oncology,” stated Anne White, president of Lilly Oncology. “This data from LIBRETTO-001 shows that selpercatinib, also known as LOXO-292, represents an important new advance for patients with RET fusion-positive non-small cell lung cancer, emblematic of the kinds of new oncology medicines we hope to continue to bring forward at Lilly Oncology.”

The data was presented at the Presidential Symposium Session at the 2019 World Conference on Lung Cancer (WCLC) in Barcelona, Spain, which was hosted by the International Association for the Study of Lung Cancer (IASLC). The company expenses to use the data to support regulatory submissions to the FDA later this year.

RET mutations occur in about 2% of NSCLCs, in about 10% to 20% of papillary thyroid cancers, and in about 60% of medullary thyroid cancers. The company expects to present data on two kinds of thyroid cancers with RET mutations at the European Society for Medical Oncology meeting held in Barcelona later this month.

“At the beginning of this year, we put a bet on Loxo,” Daniel Skrovonsky, Lilly’s chief scientific officer, told Reuters. “The result today certainly validates that bet and exceeds what many people expected.”

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