• Alnylam Pharmaceuticals Inc., of Cambridge, Mass., presented several posters at the 7th Annual Meeting of Oligonucleotide Therapeutics Society related to systemic delivery of siRNA. Highlights included data regarding the discovery of third-generation lipid nanoparticles that improve the therapeutic index for delivery of RNAi, and advances related to the stability, potency and efficacy of novel GalNAc-conjugated siRNAs.

• EpiCept Corp., of Tarrytown, N.Y., said the FDA provided more definitive guidance on the clinical development of Ceplene (histamine dihydrochloride), the company's lead product administered with interleukin-2 (IL-2) for the remission maintenance and prevention of relapse of patients with acute myeloid leukemia in first complete remission. The FDA said that, as part of a registration study, the effect of Ceplene must be isolated from the effect of IL-2, so the preferred study design will be a comparison of Ceplene/IL-2 vs. IL-2 monotherapy. The agency also recommended that patients in the IL-2 monotherapy group receive the same IL-2 dosing regimen as patients receiving Ceplene/IL-2 in combination. The FDA reiterated the need to demonstrate a significant benefit of Ceplene/IL-2 vs. IL-2 monotherapy on overall survival as the primary endpoint, according to EpiCept, which plans to submit a new Phase III trial protocol to the agency. (See BioWorld Today, Dec. 13, 2010.)

• Hawthorn Pharmaceuticals Inc., of Madison, Miss., signed an exclusive global licensing deal with CoPlex Therapeutics LLC, of Louisville, Ky., for hawAD14, a preclinical oral small-molecule candidate for the treatment of Alzheimer's and other neurodegenerative diseases. Under terms of the agreement, Hawthorn will be responsible for funding all future development. Research indicated hawAD14 significantly decreases levels of amyloid beta and tau phosphorylation activity, the main constituents of amyloid plaques and paired helical filaments that make up neurofibrillary tangles in Alzheimer's disease. Hawthorn is conducting the necessary preclinical studies to file an investigational new drug application. Financial terms were not disclosed.

• Janssen Biotech Inc. (formerly Centocor Ortho Biotech Inc.), a Johnson & Johnson subsidiary, of Horsham, Pa., said the FDA issued a complete response letter for its Simponi (golimumab) supplemental biologics license application (sBLA) seeking an expanded label in the treatment of active psoriatic arthritis. The application, filed in November 2010, included data from a Phase III trial evaluating the effect of Simponi in inhibiting the progression of structural damage and maintaining improvement in signs and symptoms and physical function in the treatment of patients with active psoriatic arthritis. In July, the company received a CRL from the FDA regarding its sBLA seeking to expand the label for Simponi in rheumatoid arthritis to include a claim that the drug inhibits the progression of structural damage. (See BioWorld Today, April 27, 2009.)

• Oncothyreon Inc., of Seattle, licensed the preclinical Bcl-2 inhibitor sabutoclax from the Sanford-Burnham Medical Research Institute. Terms were not disclosed.

• Peregrine Pharmaceuticals Inc., of Tustin, Calif., presented preclinical data showing its phosphatidylserine (PS)-targeted antibodies, in addition to being used as anticancer therapeutics, can be used for tumor imaging. The antibodies may be effective in detection and monitoring of standard cancer therapies.

• PharmaGap Inc., of Ottawa, said it designed a liposome formulation of its lead cancer drug GAP-107B8, and will proceed to in vivo testing in bladder and ovarian cancer models. GAP-107B8 is a peptide that has been shown to be highly cytotoxic to numerous cancer types, and the liposomal formulations are intended to improve the therapeutic index.

• The Medicines Co., of Parsippany, N.J., on Monday launched a 50-mL ready-to-use formulation of Argatroban injection, a direct thrombin inhibitor indicated for prophylaxis or treatment of thrombosis in adult patients with heparin-induced thrombocytopenia (HIT) and as an anticoagulant in adult patients with or at risk for HIT undergoing percutaneous coronary intervention. TMC is supplying Argatroban in a pre-mixed ready-to-use, single-use 50-mL vial.