• Isis Pharmaceuticals Inc., of Carlsbad, Calif., reported that three drugs were found to be safe and well tolerated in separate Phase I studies. The drugs, ISIS-PTP1B(Rx), ISIS-GCGR(Rx), and ISIS-GCCR(Rx), are in development for indications related to metabolic disorders including Type II diabetes. ISIS-PTP1B(Rx) is designed to target protein tyrosine phosphatase-1B, ISIS-GCGR(Rx) is designed to target the glucagon receptor and ISIS-GCCR(Rx) is designed to target the glucocorticoid receptor.

• Sarepta Therapeutics Inc., of Cambridge, Mass., said updated data from Study 202, its open-label, Phase IIb extension study of eteplirsen for the treatment of Duchenne's muscular dystrophy, showed that patients treated with eteplirsen for 62 weeks and evaluable on ambulatory measures (modified intent-to-treat population) maintained a statistically significant clinical benefit on the primary clinical outcome measure, the six-minute walk test, compared to patients who received placebo for 24 weeks followed by 38 weeks of eteplirsen treatment. As reported previously, Study 202 met its primary endpoint of increased novel dystrophin as assessed in muscle biopsies at week 48 and is now in the long-term extension phase in which patients continue to be followed for safety and clinical outcomes.

• Stemline Therapeutics Inc., of New York, released data from two studies of SL-401 in acute myeloid leukemia (AML) and other hematologic malignancies, including blastic plasmacytoid dendritic cell neoplasm (BPDCN) and preclinical activity against Hodgkin's lymphoma, scheduled for presentation at the 54th Annual Meeting of the American Society of Hematology in Atlanta. Updated clinical trial results for SL-401 suggested the therapy, which targets the interleukin-3 receptor, was well tolerated at clinically active doses and showed single agent antitumor activity in heavily pretreated patients with AML as well as in patients with high-risk myelodysplastic syndrome and relapsed/refractory BPDCN. A single cycle of SL-401 demonstrated single agent activity in patients with relapsed or refractory AML, including two durable complete responses of eight and > 25 months duration, respectively, and multiple additional cases of leukemia blast reductions. An overall survival benefit also was observed with only one cycle of SL-401 among the most treatment-resistant AML patients. In a second study, Stemline's SL-101, a monoclonal antibody-conjugate that targets CD123, demonstrated preclinical efficacy against lymphoid cancers, including Hodgkin's lymphoma.

• TG Therapeutics Inc., of New York, initiated a Phase I/II trial to evaluate the safety, tolerability and efficacy of TG-1101, its anti-CD20 monoclonal antibody, in combination with lenalidomide (Revlimid, Celgene Corp.) for patients with relapsed or refractory B-cell lymphoid malignancies previously treated with anti-CD20 antibody therapy. The multicenter trial will enroll up to 30 patients in the Phase I dose escalation component and up to an additional 30 patients, stratified by B-cell malignancy subtype, once the optimal dose is determined.