Drug Developers Expecting Significant Clinical and Economic Benefits by Targeting Newest Drug Therapies - Seite 2

In other pharma and biotech developments in the markets: 

Celgene Corporation (NASDAQ: CELG) and Acceleron Pharma Inc. (NASDAQ: XLRN) this week announced results from a phase III, randomized, double-blind, multi-center clinical study (BELIEVE). Luspatercept achieved a highly statistically significant improvement in the primary endpoint of erythroid response, which was defined as at least a 33 percent reduction from baseline in red blood cell (RBC) transfusion burden with a reduction of at least 2 units during the protocol-defined period of 12 consecutive weeks, from week 13 to week 24, compared to placebo. BELIEVE evaluated the efficacy and safety of luspatercept plus best supportive care versus placebo plus best supportive care in adults with transfusion-dependent beta-thalassemia.

Celgene Corporation (NASDAQ: CELG) also announced this week that the Phase III IMpassion130 study, which was sponsored by Roche, met its co-primary endpoint of progression-free survival (PFS). This is the first phase III study to demonstrate a statistically significant PFS improvement in first-line metastatic or unresectable locally advanced triple negative breast cancer (TNBC), a type of breast cancer with high unmet need. Results demonstrated that the investigational combination of TECENTRIQ® (atezolizumab) plus ABRAXANE® (paclitaxel protein-bound particles for injectable suspension) (albumin-bound) compared to ABRAXANE® monotherapy, as an initial (first-line) treatment, significantly reduced the risk of disease worsening or death (PFS) in patients with metastatic or unresectable locally advanced TNBC in the intention-to-treat (ITT) and PD-L1 positive populations.

BioCryst Pharmaceuticals, Inc. (NASDAQ: BCRX), a pharmaceutical company focused on the development and commercialization of treatments for rare diseases, recently announced that the Company has reached agreement on the design of a Phase 3 trial and regulatory requirements for marketing authorization of BCX7353 for Hereditary Angioedema ("HAE") with the Pharmaceuticals and Medical Devices Agency ("PMDA") in Japan. The Phase 3 trial design agreed upon for Japan, APeX-J, is a randomized, placebo-controlled double-blind trial of 24 weeks duration with a 28-week safety extension. Approximately 24 Japanese subjects with HAE will be enrolled. The APeX-J trial design closely follows the design of APeX-2, a Phase 3 clinical trial being conducted in the U.S., Canada and European countries. APeX-J tests the same dose levels of BCX7353 as in APeX-2, 110 mg daily and 150 mg daily, and the endpoints are identical to those in APeX-2. Data from the APeX-J and APeX-2 trials will be combined for regulatory submission in Japan.