ASH Roundup: Immunotherapy Stars, Gene Therapy Leaps Ahead

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“off the shelf” procedure for manufacturing genetically engineered donor cells; its first trial will include patients with CD19-positive B cell leukemia.

—Cambridge, MA-based Foundation Medicine (NASDAQ: FMI) kept building the case for its FoundationOne Heme, a diagnostic that analyzes the genomic profile of blood cancers. (Its other test, FoundationOne, does the same for solid tumors.) Foundation presented three studies: In a 112-patient study, 96 percent had genetic alterations with “therapeutic relevance”—meaning, a marketed or experimental drug exists that targets those genetic abnormalities. In a second study, 42 of 84 patients with various blood cancers had relevant genetic alterations; 12 of those cases (14 percent) led to a change in the patient’s diagnosis. Foundation added a third study purporting to show that FoundationHeme led to a clinical benefit for patients with relapsed multiple myeloma. The company is conducting studies like these to convince insurers to cover its tests.

—Cambridge-based Epizyme (NASDAQ: EPZM) provided an early look at preclinical data for its third drug, a PRMT5 inhibitor it’s developing with GlaxoSmithKline (NYSE: GSK). Shares trended downward last month after Epizyme released the abstract for a Phase 1 study one of its two lead compounds, EPZ-5676. It updated that data this week as well, revealing one additional partial response to the drug, though investors have shown more interest in another drug—EPZ-6438—that is also in early studies. Epizyme is one of the few recently public, Celgene-partnered biotechs that hasn’t taken off yet. It priced at $15 last year, and currently trades at roughly $21.

—Investigators affiliated with Juno presented or updated five Phase 1 or 1/2 clinical trials. The results included 24 of 27 complete remissions in adults with acute lymphoblastic leukemia after CD19 CAR-T cell therapy; and six of six patients with relapsed/refractory B cell non-Hodgkin’s lymphoma remained alive and in remission at a median follow-up of 9 months after high-dose chemotherapy, autologous stem cell transplant, and CD19 CAR-T cell therapy. Juno lists all five updates here.

—Shares of Newton, MA-based Karyopharm Therapeutics (NASDAQ: KPTI) have roughly doubled in 2014 on the strength of data for the company’s prospective blood cancer drug selinexor. At ASH, Karyopharm released results from a study testing the drug in combination with carfilzomib and dexamethasone, plus more results from a non-Hodgkin’s lymphoma study. Karyopharm’s drugs are supposed to work by keeping the body’s tumor suppressor proteins in the nucleus of cells. You can read more about the approach here.

—Lexington, MA-based Promedior updated numbers from the Phase 2 trial it’s running for PRM-151, a myelofibrosis drug prospect. It’s the latest data from the trial we detailed back in May, and privately-held Promedior said patients’ overall response rate of 43 percent after six months were good enough to move forward.

Alex Lash contributed to this report.

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