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FDA gives Loxo breakthrough tag for second cancer drug

US regulatory body to expect a NDA for LOXO-292 in late 2019


Loxo Oncology has picked up another breakthrough designation (BTD) from the FDA, this time for LOXO-292, a RET-targeting therapy that put in an impressive performance in a phase I/II study reported at this year’s American Society of Clinical Oncology (ASCO) meeting.

The BTD covers patients with metastatic RET-fusion-positive non-small cell lung cancer (NSCLC) who have progressed despite earlier therapy with chemotherapy and checkpoint inhibitors, as well as last-line treatment of RET-positive thyroid cancer.

At ASCO, Connecticut, US-based Loxo reported impressive data for the drug in heavily-pre-treated RET-positive tumours from the LIBRETTO-001 study, including a 77% response rate in NSCLC, including patients with brain metastases, and a 45% response rate in thyroid cancer.

Earlier this month, Loxo chief executive Josh Bilenker said ASCO data was a key milestone for the LOXO-292 programme as “the strength of the data helped raise awareness for RET, as an actionable target”.

RET mutations are seen in around 2% of NSCLC tumours, 10-20% of papillary and other thyroid cancers, and at low rates in other cancer types including pancreas, breast and colon cancers. LOXO-292 is designed to inhibit both RET signalling, as well as sidestepping common resistance mechanisms that occur after exposure to RET-targeting drugs.

Loxo has been building a position in ‘precision’ cancer therapies that target the genetic signature of the tumour and not the organ where the cancer originated. Its pipeline is headed by larotrectinib which has a BTD for patients with locally advanced or metastatic solid tumours harbouring an NTRK gene fusion mutation and has already been submitted for approval in the US, with an FDA decision due by 26 November.

Last year, Bayer paid an impressive $400m upfront with another $1.1bn in milestones for rights to larotrectinib and another tropomyosin receptor kinase (TRK) targeted drug called LOXO-195.

Loxo is hoping to submit a new drug application for LOXO-292 in late 2019 based on updated LIBRETTO-001 data and using overall response rate (OOR) as a primary endpoint, according to Bilenker. Additional dose-escalation data from the study will be presented later this year.

“We look forward to working with FDA to streamline the development of LOXO-292 in the two patient populations that have comprised the bulk of our initial clinical trial enrollment,” he said.

“Given the many available therapies for NSCLC and medullary thyroid cancer, we are pleased that LOXO-292 has shown encouraging data in refractory patients, and hope to demonstrate the full potential of this treatment in additional populations over time.”

Article by
Phil Taylor

6th September 2018

From: Regulatory



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