– Company provides update following receipt of FDA minutes from Type B meeting –

– Conference call and webcast November 1 at 4:30 p.m. EDT –

 

CHICAGO, Nov. 01, 2016  -- AveXis, Inc. (AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced that the planned pivotal study of AVXS-101 in spinal muscular atrophy (SMA) Type 1 will reflect a single-arm design, using natural history of the disease as a comparator, and enroll approximately 20 patients. This update is based on the receipt of the minutes following the Type B meeting with the U.S. Food and Drug Administration (FDA) held on September 30, 2016.

MONTREAL, CANADA--(Oct 28, 2016) - Theratechnologies Inc. (Theratechnologies) (TH.TO) announced additional results related to the primary endpoint of the ibalizumab Phase III pivotal study, TMB-301. The initial results were announced on May 24, 2016.

 

Patients with multi-drug resistant (MDR) HIV-1 experienced a significant decrease in viral load after receiving a loading dose of ibalizumab 2,000 mg intravenously (IV) in addition to their failing antiretroviral therapies (ART) (or no therapy), according to new data from the TMB-301 study to be presented Saturday, October 29, 2016 by Theratechnologies' partner, TaiMed Biologics, in an oral presentation at IDWeek 2016™. A total of 40 patients were enrolled in the study. Seven days after the loading dose, 83% of patients achieved a ≥ 0.5 log10 decrease from baseline compared with 3% during the seven-day control period. These results were statistically significant (p<0.0001).

100% success rate on meeting clinical trial's primary end points required for Accelerated Approval Regulatory Pathway

100% clinical response rate with healing of lesions within weeks of treatment

Successful pre-BLA meeting held with FDA

ProMetic to commence filing plasminogen BLA modules with the FDA as planned in coming weeks

 

LAVAL, QC, Oct. 26, 2016 - ProMetic Life Sciences Inc. (TSX:PLI.TO - News) (OTCQX:PFSCF -News) ("ProMetic" or the "Corporation") announced today that its pivotal Phase 2/3 clinical trial in patients with plasminogen deficiency has met its primary and secondary endpoints with the intravenous plasminogen treatment.

In addition to being safe, well tolerated and without any drug related serious adverse events, ProMetic's plasminogen treatment achieved a 100% success rate of its primary end point, namely, a targeted increase in the blood plasma concentration level of plasminogen as a surrogate target. Moreover, all patients who had active visible lesions when enrolled in the trial had complete healing of their lesions within weeks of treatment, a 100% response rate for this secondary end point.

HOPE – A Phase 3 Clinical Trial to Initiate in December -- Company to Host Conference Call and Webcast Today at 1:30 p.m. PT/ 4:30 p.m. ET

 

SOUTH SAN FRANCISCO, Calif., Oct. 24, 2016 -- Global Blood Therapeutics, Inc. (GBT) (GBT), a biopharmaceutical company developing novel therapeutics for the treatment of grievous blood-based disorders with significant unmet need, today announced that it has reached agreement with the U. S. Food and Drug Administration (FDA) regarding the design of its pivotal trial for GBT440 in adults and adolescents with sickle cell disease (SCD).

 

The Phase 3 HOPE (Hemoglobin Oxygen Affinity Modulation to Inhibit HbSPolymErization) Study will be conducted at leading SCD sites globally and will enroll adults and adolescents with SCD who have had at least one episode of vaso-occlusive crisis (VOC) in the previous year. The HOPE Study is expected to begin screening patients by December with top-line data anticipated in the first half of 2019.