Cancer Wrap: Blueprint, Checkmate, More AACR News & Phase 3 Blues

Xconomy National — 

The big headlines from the American Association for Cancer Research meeting in Chicago were all about the battle over the latest lung cancer data, and for good reason. Lung cancer remains the deadliest cancer, but the Phase 3 data suggest that treatment options for some of the direst cases could soon rapidly expand.

There were results from earlier stages of clinical studies and preclinical research, which is the traditional material for AACR, as well as a couple of late-stage letdowns that came to light. Here’s a sample of other cancer news blowing in the Windy City and beyond this week.


Drugs designed to target specific mutations in particular types of cancer—HER2-positive breast cancer or EGFR-positive lung cancer, for example—have been around a long time, with most approved in the past two decades.

But the FDA gave the field a big jolt last year when it approved Merck’s pembrolizumab (Keytruda) to treat tumors with a specific genetic alteration regardless of origin—the first decision of its kind. Loxo Oncology (NASDAQ: LOXO) and Ignyta (NASDAQ: RXDX) are leading similar efforts and could have drugs on the market in coming years. Their commercial success will depend on whether DNA testing to find patients with more mutations, even rare ones, becomes more widely adopted (and reimbursed by insurance).

At AACR, Blueprint Medicines (NASDAQ: BPMC) of Cambridge, MA, provided a first look at human data for its BLU-667, which targets so-called RET fusions that can drive cancers of the lung, thyroid, and more. So far, 7 of 14 patients (50 percent) with non-small cell lung cancer and 10 of 25 (40 percent) with medullary thyroid cancer who have failed other drugs have responded to BLU-667.

Why is such an interim look noteworthy? Blueprint CEO Jeff Albers says the company will pursue “expedited development” of its drugs in genetically defined patient subgroups.”It’s early to speculate about an accelerated approval pathway,” Albers told Xconomy, but there’s precedent. Loxo filed for accelerated FDA approval based on three studies, totaling 55 patients with TRK fusions. Blueprint will see where it stands after adding more patients with different cancer types to the Phase 1 study and focusing more on how long their responses last, rather than just whether they respond at all. “Ultimately, our registration strategy will be guided by additional data from the ongoing trial,” Albers says.

Wall Street wasn’t impressed and sent Blueprint shares down 13 percent. Blueprint will soon face competition from Loxo, whose own RET fusion-targeting drug is in Phase 1. Albers, who notes Blueprint will test a higher dose of BLU-667 in further testing, brushed off the “day-to-day movements in the market.”

“We believe the data reported at AACR give us a very solid foundation to rapidly advance the program,” he says.

—Poseida Therapeutics released very early data from a Phase 1 study of its CAR-T cell therapy for multiple myeloma. The live T cells are drawn from a patient, engineered to target the protein BCMA, and infused back to the patient.

It’s only three patients on a low dose of cells, but San Diego-based Poseida said the results—activity against the cancer and no “cytokine storm” side effects that often come with CAR-T therapy—were good enough to step up to the next dose. Bluebird Bio (NASDAQ: BLUE) and Novartis (NYSE: NVS) have the most advanced CAR-T programs for myeloma, with Bluebird and partner Celgene (NASDAQ: CELG) planning a pivotal study this year. Poseida’s study is backed with a $20 million grant from California’s regenerative medicine agency.


—Checkmate Pharmaceuticals of Cambridge, MA, has nudged its way into the combination immunotherapy game with promising early data presented Monday. It’s well documented that checkpoint inhibitors only help a minority of patients. With its drug CMP-001 Checkmate wants to make resistant cancers vulnerable to checkpoint therapy. Its first test: CMP-001 and pembrolizumab in melanoma patients who are failing treatment with pembrolizumab.

The early numbers at AACR: 15 of 69 evaluable patients (22 percent) who had failed a median of two treatments, including checkpoint therapy, responded to the CMP-001-pembrolizumab combination. Six of the responses have lasted … Next Page »

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