CRBP Is One To Keep An Eye On, FDA Expands Use Of Promacta, OSUR Gets Funds

(RTTNews) - Alnylam Pharmaceuticals Inc.'s (ALNY) initial data from its ongoing phase 1/2 clinical trial with ALN-CC5 for the treatment of complement-mediated diseases has shown dose-dependent, durable, and statistically significant knockdown of serum C5 of up to 96% and has been found to be generally well tolerated to date.

Complement component C5 is predominantly expressed in liver cells, and is a genetically and clinically validated disease target. Silencing C5 represents a novel approach to the treatment of complement-mediated diseases, according to the company.

The trial results also demonstrated that single dose administration of ALN-CC5 was capable of achieving inhibition of serum complement activity of up to 92%, including an up to 61% inhibition of serum hemolytic activity, noted the company.

The phase 1/2 trial of ALN-CC5 is being conducted in the U.K., in three parts. Parts A and B are randomized (3:1, drug:placebo), double-blind, placebo-controlled, single-dose (Part A) and multi-dose (Part B), dose-escalation studies, designed to enroll up to a total of 60 healthy adult volunteers.

ALNY closed Friday's trading at $130.28, down 1.85%.

Corbus Pharmaceuticals Holdings Inc.'s (CRBP) lead drug candidate Resunab has been granted Orphan Drug Designation for the treatment of systemic sclerosis. The company is preparing to commence phase II studies of Resunab for systemic sclerosis.

Resunab is already being tested in a phase II study for dermatomyositis, and a phase II study of Resunab in adults with cystic fibrosis is expected to commence in the third quarter of this year.

CRBP closed Friday's trading at $3.15, down 11.52%.

CTI BioPharma Corp. (CTIC) and Baxter International's BioScience business on Friday reported encouraging data from a phase III clinical trial of Pacritinib, dubbed PERSIST-1, in patients with primary and secondary myelofibrosis.

According to the trial results, there was a significant reduction in the Total Symptom Score (TSS) (the proportion of patients with a 50 percent or greater reduction in TSS from baseline to Week 24), and in each individual common disease-related symptom, from baseline to Week 24, in patients treated with Pacritinib compared to best available therapy (exclusive of a JAK inhibitor) (BAT).

Pacritinib is currently being evaluated in two phase III clinical trials, known as the PERSIST I and PERSIST-2.

CTIC closed Friday's trading 2.39% down at $2.04.

The FDA has expanded the approved use of Promacta, co-discovered by GlaxoSmithKline plc (GSK) and Ligand Pharmaceuticals Inc. (LGND) for children six years and older with chronic immune thrombocytopenia who have had an insufficient response to corticosteroids, immunoglobulins or splenectomy. The drug was approved by the FDA in 2008 for use in adult patients with the same condition.

Promacta was acquired by Novartis in March 2015 from GlaxoSmithKline. Promacta had sales of $99 million in the first quarter of this year, and Ligand Pharma expects Promacta sales to be about half a billion dollars in the full year of 2015.

LGND closed Friday's trading at $93.93, down 0.24%.

MediciNova Inc.'s (MNOV) ongoing clinical trial of MN-166 in progressive multiple sclerosis has completed randomization of 255 subjects, which exceeds the goal of 250 subjects that were planned for participation.

An interim efficacy analysis will be conducted by the National Institute of Neurological Disorders and Stroke after half the subjects complete the 96-week treatment period, which analysis is expected to occur in the fall of 2016.

MNOV closed Friday's trading at $4.05, down 2.17%.

OraSure Technologies Inc. (OSUR) has been awarded a contract for up to $10.4 million in total funding from the U.S. Department of Health and Human Services to advance OraQuick Ebola Rapid Antigen Test.

The three-year, multi-phased contract includes an initial commitment of $1.8 million and options for up to an additional $8.6 million to fund certain clinical and regulatory activities, according to the company.

OSUR closed Friday's trading at $5.69, down 1.90%.