Apitope Ltd., of Bristol, UK, said its peptide therapy for the treatment of factor VIII intolerance, ATX-F8-117, has formally entered preclinical development. The drug is in development for possibly treating and preventing inactivating alloantibody development in hemophilia A patients treated with factor VIII.

Epizyme Inc., of Cambridge, Mass., said it started a phase Ib open-label study of EPZ-5676 in pediatric patients with acute leukemias bearing a rearrangement of the MLL gene (MLL-r). The study is designed to evaluate the safety, pharmacokinetics and pharmacodynamics of escalating doses of the drug in patients between the ages of 3 months and 18 years and to provide a preliminary assessment of efficacy. Epizyme also has additional studies ongoing, including one enrolling adults with MLL-r, with data expected in the second half of 2014. EPZ-5676, a small-molecule inhibitor of DOT1L, is partnered with Celgene Corp., of Summit, N.J., in a 2012 deal. (See BioWorld Today, April 26, 2012.)

Exelixis Inc., of South San Francisco, reported results from BRIM7, an ongoing phase Ib trial conducted by collaborator Roche AG, of Basel, Switzerland, and its Genentech subsidiary, on BRAF inhibitor vemurafenib in combination with MEK inhibitor cobimetinib in patients with locally advanced/unresectable or metastatic melanoma carrying a BRAFV600 mutation. As of Oct. 1, 2013, 129 patients had been treated, including 66 patients who had previously progressed on vemurafenib and 63 patients who were BRAF inhibitor-naïve. In the naïve patients, an 87 percent confirmed overall response (ORR) rate was achieved, including a 10 percent complete response rate and 78 percent partial responses. Results from vemurafenib-progressor patients showed a 15 percent confirmed ORR, 42 percent stable disease rate and a median progression-free survival of 2.8 months. Data were presented at the European Association of Dermato-Oncology meeting in Vilnius, Lithuania.

Intercept Pharmaceuticals Inc., of New York, reported results from the phase II OBADIAH trial at Digestive Disease Week in Chicago showing potential clinical benefits of obeticholic acid (OCA) for patients with bile acid diarrhea. Results showed that OCA, an agonist of farnesoid X receptor, produced an increase in fasting FGF19 after two weeks of treatment, the primary outcome measure, along with an improvement in clinical symptoms. Significant correlations were observed between reductions in serum bile acid levels and reductions in stool frequency (p = 0.004) and increases in fasting FGF19 (p = 0.02). OCA was well tolerated.