Lilly Presents New Data on Retevmo® (selpercatinib) in Advanced RET Fusion-Positive Gastrointestinal and Other Cancers at 2021 American Association for Cancer Research (AACR) Annual Meeting

Confirmed Objective Response Rate of 47 Percent, with Responses Observed in Nine Unique Cancer Types

Median Duration of Response Not Reached at 13 Months Median Follow-Up, with 11 of 15 Responses Ongoing

Safety Consistent with Known Profile of Retevmo

New Data Expands on Retevmo's Established Data in Lung and Thyroid Cancers

INDIANAPOLIS, IN, USA I April 11, 2021 I Eli Lilly and Company (NYSE: LLY) today announced for the first time data from the Phase 1/2 LIBRETTO-001 trial showing treatment with Retevmo® (selpercatinib) demonstrated encouraging antitumor activity and safety across RET fusion-positive advanced solid tumors beyond lung and thyroid cancers, including multiple treatment-refractory gastrointestinal (GI) malignancies. The data were presented at the 2021 American Association for Cancer Research (AACR) Annual Meeting, held virtually April 10-15, 2021.

"We are excited to broaden the body of evidence for Retevmo in RET fusion-positive cancers beyond lung and thyroid tumors," said David Hyman, M.D., chief medical officer, oncology at Lilly. "These encouraging outcomes, including in difficult-to-treat GI malignancies, support a growing body of evidence that RET fusions are potentially actionable in a wide range of tumor types. These findings further demonstrate the importance of broad tumor profiling in advanced cancers. We look forward to discussing these new data with regulatory authorities this year."

In the Phase 1/2 LIBRETTO-001 trial, 32 adult patients with 12 unique RET fusion-positive advanced cancer types were enrolled by the efficacy cutoff date of September 19, 2020 (with follow-up through March 19, 2021). Cancer types treated included pancreatic, colon, breast, salivary, sarcoma, carcinoid, rectal neuroendocrine, small intestine, xanthogranuloma, ovarian, pulmonary carcinosarcoma, and unknown primary cancers. Among the 32 patients, 62.5 percent had gastrointestinal tumors (defined as pancreatic [n=9], colon [n=9], small intestine [n=1], and rectal neuroendocrine [n=1]). Across all 32 patients, the confirmed objective response rate (ORR) was 47 percent (95% CI: 26-65%). Confirmed responses were observed in nine unique RET fusion-positive advanced cancer types. The median duration of response (DoR) was not reached, with median follow-up of 13 months. Responses were ongoing in 73 percent (11/15) of responding patients.

Retevmo Efficacy
Objective Response Rate* % (95% CI) 47% (29-65), n=32
Median Duration of Response (range) Not Reached (2 -33+ months)
Responses Ongoing 73% (11/15)
Median Duration of Follow up 13 months

* per investigator assessment, + indicates patient ongoing

Safety among patients in this cohort was consistent with the known safety profile of Retevmo. In this cohort, the most common treatment-emergent adverse events of any grade (≥20%) were increased aspartate aminotransferase (AST)/increased alanine aminotransferase (ALT), dry mouth, hypertension, diarrhea, fatigue, nausea, and abdominal pain. No patients in this cohort discontinued treatment due to treatment-related adverse events.

"While uncommon, RET fusions occur in a 'long tail' of solid tumors beyond lung and thyroid cancers, and these patients do not yet have an approved targeted therapy option to address the underlying genomic driver of their cancer," said Vivek Subbiah, MD, associate professor in the Investigational Cancer Therapeutics Department and center clinical medical director of the Clinical Center for Targeted Therapy, of the Cancer Medicine Division, at The University of Texas MD Anderson Cancer Center. "These results demonstrate selpercatinib's potential for this patient population and reiterate the importance of broad-based genomic profiling to identify actionable oncogenic drivers, including RET fusions."

In May 2020, Lilly's first-in-class selective RET inhibitor Retevmo received Accelerated Approval from the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with metastatic RET fusion-positive non-small cell lung cancer (NSCLC), in adult and pediatric patients 12 years of age and older with advanced or metastatic RET-mutant medullary thyroid cancer (MTC) who require systemic therapy, and in adult and pediatric patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate). Retevmo was approved based on the Phase 1/2 LIBRETTO-001 trial's endpoints of ORR and DoR. Retevmo (marketed as Retsevmo® outside the U.S.) was approved by the European Commission in February 2021.

About LIBRETTO-001

The Phase 1/2 LIBRETTO-001 trial is the largest clinical trial of patients with RET-driven cancers treated with a RET inhibitor. The trial, which spans 16 countries and 89 sites, included a dose escalation phase (Phase 1) and a dose expansion phase (Phase 2). The Phase 2 portion of the trial had major efficacy outcomes of ORR and DoR, and prespecified secondary endpoints of central nervous system (CNS) ORR and CNS DoR, as determined by an independent review committee according to Response Evaluation Criteria in Solid Tumors (RECIST) v1.1.

LIBRETTO-001 continues to enroll patients with RET-altered tumors beyond lung cancer.

About Retevmo® (selpercatinib)

Retevmo (selpercatinib, formerly known as LOXO-292) (pronounced reh-TEHV-moh) is a selective and potent RET kinase inhibitor. 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 an 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.i Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials.

Please see full Prescribing Information for Retevmo.

SE HCP ISI All_25MAR2021

About Loxo Oncology at Lilly

Loxo Oncology at Lilly was created in December 2019, combining the Lilly Research Laboratories oncology organization and Loxo Oncology, which was acquired by Lilly in early 2019. Loxo Oncology at Lilly brings together the focus and spirit of a biotech with the scale and resources of large pharma, with the goal of rapidly delivering impactful new medicines for people with cancer. Our approach centers on creating new oncology medicines that unequivocally work early in clinical development and will matter to patients.

About Lilly Oncology

For more than 50 years, Lilly has been dedicated to delivering life-changing medicines and support to people living with cancer and those who care for them. Lilly is determined to build on this heritage and continue making life better for all those affected by cancer around the world. To learn more about Lilly's commitment to people with cancer, please visit www.LillyOncology.com.

About Eli Lilly and Company

Lilly is a global health care leader that unites caring with discovery to create medicines that make life better for people around the world. We were founded more than a century ago by a man committed to creating high-quality medicines that meet real needs, and today we remain true to that mission in all our work. Across the globe, Lilly employees work to discover and bring life-changing medicines to those who need them, improve the understanding and management of disease, and give back to communities through philanthropy and volunteerism. To learn more about Lilly, please visit us at lilly.com and lilly.com/newsroom.

i RETEVMO [package insert]. Indianapolis, IN: Eli Lilly and Company; 2021.

SOURCE: Eli Lilly

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