Lilly Presents New Data on 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 Per Cent, 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 selpercatinib
• New Data Expands on selpercatinib's Established Data in Lung and Thyroid Cancers

TORONTO, April 20, 2021 /CNW/ - On April 11, 2021, Eli Lilly and Company announced data from the Phase 1/2 LIBRETTO-001 trial showing treatment with 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.

"Research into a broader set of RET fusion-positive cancers enables us to better understand the role of precision medicine in improving quality of life for patients with advanced disease," says Dr. Doron Sagman, vice president, R&D and Medical Affairs, Eli Lilly Canada. "Outcomes from the Phase 1/2 LIBRETTO-001 trial, are encouraging for difficult to treat GI and other RET-driven malignancies in patients treated with selpercatinib, a RET inhibitor."

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 cut-off 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 per cent 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 per cent (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 per cent (11/15) of responding patients.

selpercatinib 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 selpercatinib. 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.

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 selpercatinib
Selpercatinib (formerly known as LOXO-292) is a selective and potent RET kinase inhibitor. Selpercatinib 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. Selpercatinib 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. Selpercatinib is not approved for use in Canada.

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.

About Lilly Canada
Eli Lilly and Company is a global healthcare leader that unites caring with discovery to make life better for people around the world. We were founded more than a century ago by Colonel Eli Lilly, who was committed to creating high quality medicines that meet people's needs, and today we remain true to that mission in all our work. Lilly employees work to discover and bring life-changing medicines to people who need them, improve the understanding and management of disease, and contribute to our communities through philanthropy and volunteerism.

Eli Lilly Canada was established in 1938, the result of a research collaboration with scientists at the University of Toronto which eventually produced the world's first commercially available insulin. Our work focuses on oncology, diabetes, autoimmunity, neurodegeneration, and pain. To learn more about Lilly Canada, please visit us at www.lilly.ca.

For our perspective on issues in healthcare and innovation, follow us on twitter @LillyPadCA and @LillyMedicalCA

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 selpercatinib for the treatment of metastatic RET fusion-positive NSCLC, advanced or metastatic RET-mutation-positive MTC, and advanced or metastatic RET fusion-positive thyroid cancer, and as a potential treatment for other indications, and reflects Lilly's current beliefs and expectations. However, as with any pharmaceutical product, there are substantial risks and uncertainties in the process of development and commercialization. Among other things, there is no guarantee that future study results will be consistent with study findings to date or that selpercatinib will receive additional regulatory approvals. For further discussion of these and other risks and uncertainties, 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.

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

SOURCE Eli Lilly Canada Inc.

Media Contact: Samira Rehman, [email protected], 647-617-1994