ALNY In Motion, Shire Finds New Use For Old Drug, TFX Snaps Up VASC

(RTTNews) - Alnylam Pharmaceuticals Inc. (ALNY), on Sunday, reported positive interim clinical results of its investigational RNAi therapeutic Fitusiran from an ongoing phase II open label extension study in patients with Hemophilia A or B without inhibitors.

According to the trial results, once-monthly subcutaneous administration of Fitusiran resulted in lowering of average antithrombin (AT) by approximately 80 percent and resulted in median Annualized Bleeding Rate (ABR) of 1.0 compared to a median pre-study ABR of 4.0.

In addition, Fitusiran was generally well tolerated, with no thromboembolic events or laboratory evidence of pathologic clot formation, noted the company.

ALNY closed Friday's trading at $41.57, down 0.02%.

Acceleron Pharma Inc. (XLRN) and Celgene Corp. (CELG), on Sunday, announced encouraging preliminary results from two phase II studies of Luspatercept for myelodysplastic syndromes - one in first-line, ESA treatment-naive MDS patients and the other in ring Sideroblast positive (RS+) and negative (RS-) MDS patients.

Acceleron and Celgene are jointly developing Luspatercept as part of a global collaboration.

XLRN closed Friday's trading at $36.29, up 3.57%.

bluebird bio Inc.'s (BLUE) phase 1/2 study of LentiGlobin in the treatment of patients with transfusion-dependent ß-thalassemia and severe sickle cell disease, dubbed HGB-205 study, has produced promising results.

The trial results show a stable clinical and biological effect in patients with TDT or severe SCD who have received a one-time treatment with LentiGlobin, noted the company.

BLUE closed Friday's trading at $69.75, up 1.60%.

Shire plc's (SHPG) phase III clinical trial of VONVENDI in patients with von Willebrand disease met the primary endpoint of effectively controlling bleeding and blood loss during an operation in adults undergoing major, minor and oral elective surgical procedures.

Von Willebrand disease, or VWD, is the most common inherited bleeding disorder, affecting up to 1 percent of the U.S. population.

VONVENDI is already approved by the FDA for on-demand treatment and control of bleeding episodes in adults (age 18 and older) diagnosed with von Willebrand disease, and it was launched in the U.S. as recently as August. The results from the new trial will form the basis of a supplemental new drug application to the FDA, requesting an expanded indication for the drug.

SHPG closed Friday's trading at $174.80, up 2.23%.

uniQure N.V. (QURE), on Saturday, announced promising follow-up results from its ongoing, dose-ranging phase I/II trial of AMT-060 in patients with severe hemophilia B. The data included up to 52 weeks of follow-up from the low-dose cohort and up to 31 weeks of follow-up from the second dose cohort.

While the low dose cohort showed sustained levels of Factor IX activity up to 1 year, with marked reduction of bleeding over time and a complete cessation of spontaneous bleedings in last 14 weeks of observation, the second-dose cohort demonstrated dose response - with all patients free of prophylactic Factor IX replacement therapy and only 1 spontaneous bleed being reported.

Given the encouraging results, the company looks forward to advancing AMT-060 into a late-stage clinical study.

QURE closed Friday's trading at $5.68, down 5.18%.

Vascular Solutions Inc. (VASC) has agreed to be acquired by Teleflex Inc. (TFX) for a purchase price of $56.00 per share in cash, equating to a total value of $1.0 billion.

The transaction is expected to close in the first half of 2017, subject to customary closing conditions. Upon completion of the transaction, Vascular Solutions will become a wholly owned subsidiary of Teleflex.

VASC closed Friday's trading at $56.00, up 1.63%.