Lilly opens Phase 3 clinical trial in RET-mutant medullary thyroid cancer

Eli Lilly and CompanyEli Lilly and Company (NYSE: LLY) announced the opening of the LIBRETTO-531 clinical trial [NCT04211337] for selpercatinib, also known as LOXO-292, for treatment-naive RET-mutant medullary thyroid cancer (MTC) patients. This is the second Phase 3 trial to open for selpercatinib, a highly selective and potent, oral investigational new medicine in clinical development for the treatment of patients with cancers that harbor abnormalities in the rearranged during transfection (RET) kinase. Enrolled trial participants will be randomized to receive either selpercatinib or physician's choice of cabozantinib or vandetanib as initial treatment of their advanced or metastatic RET-mutant MTC.

"Approximately 60 percent of people with medullary thyroid cancer have an activating RET point mutation, yet the current therapeutic options are not ideal for many patients," said Lori Wirth, MD, medical director of the Center for Head and Neck Cancer, Massachusetts General Hospital Cancer Center. "This Phase 3 trial of selpercatinib in patients with advanced or metastatic RET-mutant MTC seeks to confirm a new standard of care that we hope will provide a more effective treatment option for this patient population."

"While medullary thyroid cancer is rare, the occurrence of RET mutations in MTC is high," said Gary Bloom, executive director for ThyCa: Thyroid Cancer Survivors' Association, Inc. "For that reason, we are very excited about the opening of this Phase 3 trial because it shows promise for patients with advanced and metastatic RET-mutant MTC. Due to new treatment options, it is imperative that MTC patients discuss with their medical doctors if and when they should undergo genomic testing of their tumors. This will ensure that people with RET tissue mutations have access to potential treatments and clinical trials such as this one for selpercatinib."

Trial Background

LIBRETTO-531 is a randomized Phase 3 clinical trial of patients with treatment-naïve RET-mutant MTC. The trial will enroll 400 patients with advanced or metastatic RET-mutant MTC who have received no prior systemic therapy for metastatic disease. Enrolled trial participants will be randomized 2:1 to receive either selpercatinib or physician's choice of cabozantinib or vandetanib as initial treatment of their advanced or metastatic RET-mutant MTC. RET mutations may be identified using local testing. This trial's efficacy endpoints are progression-free survival (PFS), treatment failure-free survival (TFFS), overall survival (OS), overall response rate (ORR), and duration of response (DoR). For patients randomized to the control arm, crossover is allowed at progression.

About Selpercatinib (LOXO-292)

Selpercatinib, also known as LOXO-292, is a highly selective and potent, oral investigational new medicine in clinical development for the treatment of patients with cancers that harbor abnormalities in the rearranged during transfection (RET) kinase. RET fusions and mutations occur across multiple tumor types with varying frequency. Selpercatinib was designed to inhibit native RET signaling as well as anticipated acquired resistance mechanisms.

Selpercatinib has received breakthrough designations in RET fusion-positive NSCLC, RET-mutant medullary thyroid cancer (MTC) and RET fusion-positive thyroid cancers.

About RET-Altered Cancers

Genomic alterations in RET kinase, which include fusions and activating point mutations, lead to overactive RET signaling and uncontrolled cell growth. RET fusions have been identified in approximately 2 percent of non-small cell lung cancer, 10-20 percent of papillary and other thyroid cancers and a subset of other cancers. Activating RET point mutations account for approximately 60 percent of MTC. RET fusion cancers and RET-mutant MTC are primarily dependent on this single activated kinase for their proliferation and survival. This dependency, often referred to as "oncogene addiction," renders such tumors highly susceptible to small molecule inhibitors targeting RET.

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 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.

Most Popular Now

BioMotiv and Bristol-Myers Squibb announce the lau…

BioMotiv, a mission-driven drug development accelerator associated with The Harrington Project for discovery and development, that advances breakthrough discoveries from ...

CEPI and GSK announce collaboration to strengthen …

CEPI, the Coalition for Epidemic Preparedness Innovations, and GSK announced a new collaboration aimed at helping the global effort to develop a vaccine for the 2019-nCoV...

Sandoz completes acquisition of Aspen's Japanese o…

Sandoz today announced that it has successfully completed the acquisition of the Japanese business of Aspen Global Incorporated (AGI), a wholly owned subsidiary of Aspen ...

Sanofi brain-penetrant BTK inhibitor meets primary…

The Sanofi Phase 2b study evaluating its investigational BTK (Bruton's tyrosine kinase) inhibitor (SAR442168), an oral, brain-penetrant, selective small molecule, achieve...

Roche reports very strong results in 2019

In 2019, Group sales rose 9% to CHF 61.5 billion and core EPS grew 13%, ahead of sales. The core operating profit increased 11%, reflecting the strong underlying business...

Merck donates one billionth praziquantel tablet

Merck, a leading science and technology company, today announced that it has already donated 1 billion tablets of praziquantel, the standard medication for the treatment ...

Bayer and Nuvisan create new research unit in Berl…

Bayer AG today announced that it entered into a definitive agreement to transfer a large part of its Berlin-based small molecule research unit to Nuvisan, an internationa...

Novartis announces MET inhibitor capmatinib (INC28…

Novartis announced that the US Food and Drug Administration (FDA) accepted and granted Priority Review to capmatinib’s (INC280) New Drug Application (NDA). Capmatinib is ...

WHO, China leaders discuss next steps in battle ag…

The Director-General of the World Health Organization (WHO), Dr Tedros Adhanom Ghebreyesus, met President Xi Jinping of the People's Republic of China in Beijing. They sh...

FDA approves first drug for treatment of peanut al…

Today the U.S. Food and Drug Administration approved Palforzia [Peanut (Arachis hypogaea) Allergen Powder-dnfp] to mitigate allergic reactions, including anaphylaxis, tha...

Can bilingualism protect the brain even with early…

A study by York University psychology researchers provides new evidence that bilingualism can delay symptoms of dementia. Alzheimer's disease is the most common form of d...

Poliovirus therapy shows potential as cancer vacci…

A modified form of poliovirus, pioneered at Duke Cancer Institute as a therapy for glioblastoma brain tumors, appears in laboratory studies to also have applicability for...