ASH Roundup: CAR-T Shuffle, Hemophilia Updates, Checkpoint Progress & More

Xconomy National — 

Two years ago at the American Society of Hematology conference, excitement, not blood, was running in the streets of San Francisco. The cancer immunotherapy known as CAR-T was the star of the show, thanks to dazzling early data that showed more patients than not saw their leukemias and lymphomas knocked into remission by the living, genetically engineered cells.

For some developers the momentum continues, with two programs—one for non-Hodgkin lymphoma, one for kids with a type of leukemia—likely up for an FDA approval decision next year. But the field faces tough questions in months to come about patient deaths, in-house support, and—if the treatments come to market —tension over drug prices.

There was so much news about CAR-T and other T cell therapies at ASH, we’re rounding it up here. For everything else, read on.

Gene Therapy For Hemophilia

Potential gene therapies for hemophilia—an inherited disease that leaves patients unable to properly clot blood—continue to creep forward. Gene therapy offers the promise of a long-lasting treatment, and at ASH, Spark Therapeutics (NASDAQ: ONCE) and UniQure (NASDAQ: QURE) provided incremental updates.

Those data bring promise. At first exposure, patients are producing the clotting proteins the therapies are meant to trigger. Over time, patients are going longer without dangerous bleeding events and not relying on other drugs to boost clotting proteins.

Caveats remain. It’s more clear now that these gene therapies won’t work the same way for everyone. Some patients, for instance, develop an immune response that stifles the therapies and must be controlled with steroids. How long the gene therapy lasts in these patients, and others, is an important question.

At ASH, Spark said two patients in its Phase 1 trial have developed an immune response to its gene therapy, SPK-9001. The responses lowered both patients’ levels of replacement Factor IX, but so far those levels have not dropped low enough to cause bleeds or require other treatments. Succeeding on those measures without causing safety problems is what will ultimately make or break gene therapy in hemophilia. has more here, and here’s more on gene therapy in hemophilia.

Other Hemophilia News

—On its website, the Hemophilia Federation of America posts figures about the immune responses some patients develop against replacement clotting proteins like the ones sold by Baxalta and Bayer. The responses are most common in patients with severe hemophilia A—about 30 percent. The rates are much lower in people with mild or moderate hemophilia A (5 to 8 percent) and hemophilia B (2 to 3 percent).

Alnylam Pharmaceuticals (NASDAQ: ALNY) and Roche are developing drugs meant to help patients with these types of immune responses prevent bleeds. Roche’s emicizumab is in Phase 3 testing and has shown promise reducing bleeds, but has also led to dangerous clots. Alnylam is behind, presenting at ASH Phase 1 data for its RNA interference drug, fitusiran. So far, it’s shown the potential to reduce bleeds without significant safety problems. A few patients have seen a rise in liver enzymes, however, which could signal potential problems. Alnylam said the cases were reversible. Alnylam expects to begin a late-stage trial next year. Here’s more from Bloomberg.

Checkpoint Progress in Blood Cancer

—Cancer immunotherapy drugs known as “checkpoint” inhibitors, which help reveal tumors to the immune system, have started to change how cancers of the lung, skin, and bladder are treated. Progress in blood cancers has been slower. In May, the FDA approved Bristol-Myers Squibb’s nivolumab (Opdivo) for patients with a form of Hodgkin’s lymphoma who had failed prior treatment.  Merck’s pembrolizumab (Keytruda) could be approved for the same cancer in early 2017. And as OncLive reports, studies combining … Next Page »

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