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Retevmo Demonstrates Superior Progression-Free Survival for Adults with Newly-Diagnosed Non-Small Cell Lung Cancer

INDIANAPOLIS, Aug. 4, 2023. Eli Lilly and Company today announced topline results from the LIBRETTO-431 study evaluating Retevmo versus platinum-based chemotherapy plus pemetrexed – with or without pembrolizumab – as an initial treatment for patients with rearranged during transfection (RET) fusion-positive advanced or metastatic non-small cell lung cancer (NSCLC). The study met its primary endpoint, demonstrating a statistically significant and clinically meaningful improvement in progression-free survival (PFS). This result was based on a pre-specified interim efficacy analysis conducted by an independent data monitoring committee (IDMC). Adverse events observed on Retevmo were generally consistent with those identified across the previously reported Retevmo development program (LIBRETTO-001, LIBRETTO-121, LIBRETTO-321).

The labeling for Retevmo contains warnings and precautions for hepatotoxicity (evidence of liver dysfunction), interstitial lung disease (ILD)/pneumonitis, hypertension, QT interval prolongation, hemorrhagic events, hypersensitivity, tumor lysis syndrome, risk of impaired wound healing, hypothyroidism, and embryo-fetal toxicity.

LIBRETTO-431 is a Phase 3, randomized, open-label trial evaluating Retevmo versus platinum-based chemotherapy (cisplatin or carboplatin) plus pemetrexed with or without pembrolizumab, which is a current first-line standard of care option for patients with EGFR/ALK-negative NSCLC. LIBRETTO-431 is the first randomized trial comparing the safety and effectiveness of a targeted therapy to a PD-1 inhibitor plus chemotherapy in a biomarker selected patient population.

"The LIBRETTO-431 trial aims to answer an important question about the selection of initial treatment for people with advanced RET fusion-positive NSCLC and these results suggest Retevmo should be considered a first-line standard of care," said David Hyman, M.D., chief medical officer. "Additionally, this clinically meaningful achievement of improved outcomes underscores the importance of timely and comprehensive genomic testing to inform initial treatment decisions for all patients with NSCLC. The results of this study provide further confirmation that RET status – like EGFR, ALK, and others in the family of lung cancer oncogenic drivers – should be known prior to initiating therapy. We look forward to sharing these data in more detail with the oncology community."

These results build on the data from LIBRETTO-001, the largest clinical trial of patients with RET-driven cancers treated with a RET inhibitor, which spans 16 countries and 85 sites, and includes a dose escalation phase (Phase 1) and a dose expansion phase (Phase 2). In this trial, Retevmo demonstrated clinically meaningful and durable responses across a variety of tumor types in patients with RET-driven cancers.

NSCLC accounts for about 85 percent of all lung cancer diagnoses in the U.S., of which approximately 50 percent have actionable biomarkers. RET fusions have been identified in approximately one to two percent of all NSCLC cases.

Full results from the LIBRETTO-431 trial will be presented at an upcoming medical meeting, submitted to a peer-reviewed journal, and discussed with health authorities.

About LIBRETTO-431
LIBRETTO-431 is a randomized Phase 3 clinical trial of patients with treatment-naïve RET fusion-positive NSCLC. The trial enrolled 261 patients with advanced or metastatic RET fusion-positive NSCLC who had received no prior systemic therapy for metastatic disease. Enrolled trial participants were randomized 2:1 to receive either selpercatinib or platinum-based (carboplatin or cisplatin) and pemetrexed therapy with or without pembrolizumab as initial treatment of their advanced or metastatic RET fusion-positive NSCLC. RET fusions may be identified using local testing. This trial's primary endpoint is progression-free survival (PFS) and secondary endpoints include overall survival (OS), overall response rate (ORR), duration of response (DoR), and intracranial ORR. For patients randomized to the control arm, crossover was allowed at progression.

About Retevmo (selpercatinib)
Retevmo (selpercatinib, formerly known as LOXO-292) (pronounced reh-TEHV-moh) is a highly selective and potent RET kinase inhibitor with central nervous system (CNS) activity. Retevmo may affect both tumor cells and healthy cells, which can result in side effects. RET-driver alterations are predominantly mutually exclusive from other oncogenic drivers. Retevmo is a U.S. FDA-approved oral prescription medicine, 120 mg or 160 mg dependent on weight (<50 kg or ≥50 kg, respectively), taken twice daily until disease progression or unacceptable toxicity.

About Lilly
Lilly unites caring with discovery to create medicines that make life better for people around the world. We've been pioneering life-changing discoveries for nearly 150 years, and today our medicines help more than 51 million people across the globe. Harnessing the power of biotechnology, chemistry and genetic medicine, our scientists are urgently advancing new discoveries to solve some of the world's most significant health challenges, redefining diabetes care, treating obesity and curtailing its most devastating long-term effects, advancing the fight against Alzheimer's disease, providing solutions to some of the most debilitating immune system disorders, and transforming the most difficult-to-treat cancers into manageable diseases. With each step toward a healthier world, we're motivated by one thing: making life better for millions more people. That includes delivering innovative clinical trials that reflect the diversity of our world and working to ensure our medicines are accessible and affordable.

Retevmo is a registered trademark owned by or licensed to Eli Lilly and Company, its subsidiaries, or affiliates.

Lilly Cautionary Statement Regarding Forward-Looking Statements
This press release contains forward-looking statements (as that term is defined in the Private Securities Litigation Reform Act of 1995) about Retevmo® (selpercatinib) for the treatment of locally advanced and metastatic RET fusion-positive NSCLC and reflects Lilly's current beliefs and expectations. However, as with any pharmaceutical product, there are substantial risks and uncertainties in the process of drug research, development and commercialization. Among other things, there is no guarantee that planned or ongoing studies will be completed as planned, that future study results will be consistent with study results to date, or that Retevmo will receive additional regulatory approvals. For further discussion of these and other risks and uncertainties that could cause actual results to differ from Lilly's expectations, see Lilly's Form 10-K and Form 10-Q filings with the United States Securities and Exchange Commission. Except as required by law, Lilly undertakes no duty to update forward-looking statements to reflect events after the date of this release.

SOURCE Eli Lilly and Company

Posted: August 2023

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