Sarepta Therapeutics Announces Positive Results in Its Study Evaluating Gene Expression, Dystrophin Production, and Dystrophin Localization in Patients with Duchenne Muscular Dystrophy (DMD) Amenable to Skipping Exon 53 Treated with Golodirsen (SRP-4053)

-- Study achieved statistical significance on all primary and secondary biological endpoints --

-- Results further validate the Company’s exon-skipping platform for the treatment of DMD --

CAMBRIDGE, Mass., Sept. 06, 2017 -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicines to treat rare neuromuscular diseases, today announced muscle biopsy results from its 4053-101 study, a Phase 1/2 first-in-human study conducted in Europe to assess the safety, tolerability, pharmacokinetics, and efficacy of golodirsen in 25 boys with confirmed deletions of the DMD gene amenable to skipping exon 53.

Category: Biotech

Published: 06 September 2017

Written by Editor

Nasdaq Up on News Project Update

Catalyst Biosciences Announces Positive Factor IX Clinical Data

-- CB 2679d/ISU304 is approximately 22 times more potent than current hemophilia B therapy --

SOUTH SAN FRANCISCO, Calif., Sept. 06, 2017 -- Catalyst Biosciences, Inc. (Nasdaq:CBIO), today announced positive clinical data from the first Cohort of its ongoing Phase 1/2 proof-of-concept clinical trial in individuals with severe hemophilia B.

Cohort 1 results (N = 3) demonstrate:

An intravenous dose of CB 2679d is approximately 22 times more potent than an intravenous dose of BeneFIX® as measured by activity levels using a one-stage clotting assay; and

The average time that CB 2679d stayed in the circulation was significantly longer at 34 hours compared with BeneFIX at 25 hours.

Category: Biotech

Published: 06 September 2017

Written by Editor

Nasdaq Up on News Project Update

SEATTLE, WA - September 6, 2017 (Investorideas.com Newswire) SinglePoint, Inc. (OTC:SING) announces the acquisition of Dr. FeelGood, a profitable cannabis distribution company headquartered in Phoenix, Arizona. Dr. FeelGood is a leading distribution company with expansion plans to add proprietary delivery and ordering technology application. Per the agreement terms, SinglePoint will acquire fifty-one percent (51%) of the company in a combination of cash and stock, which will allow SinglePoint to recognize all revenue. The acquisition will add an additional revenue stream to SinglePoint, solidifying the company's revenue-by-acquisition strategy.

Read this news in full at http://www.investorideas.com/CO/SING/news/2017/09061Acquisition.asp

Category: Submitted Articles

Published: 06 September 2017

Written by Investor Ideas

Verastem Announces Positive Top-line Data from the Pivotal Phase 3 DUO ™ Study in Relapsed or Refractory Chronic Lymphocytic Leukemia and Small Lymphocytic Lymphoma

The Primary Outcome of Progression Free Survival (PFS) via Independent Review Committee (IRC) in the Intent to Treat (ITT) Population Significantly Favored Duvelisib Monotherapy Over Ofatumumab (Median PFS of 13.3 versus 9.9 Months, Respectively; Hazard Ratio (HR) of 0.52, p<0.0001)

Similar Efficacy Benefit for Duvelisib Monotherapy Over Ofatumumab for Patients with 17p Deletion (Median PFS of 12.7 versus 9.0 Months, Respectively; HR of 0.41, p=0.0011)

Oral Duvelisib Continues to Demonstrate a Consistent and Manageable Safety Profile

Category: Biotech

Published: 06 September 2017

Written by Editor

Nasdaq Up on News Project Update

Read more: Verastem Inc ( VSTM )

Integrated offering to be provided by new dedicated service group under PAREXEL® Access

BOSTON, September 6, 2017- PAREXEL International Corporation (NASDAQ: PRXL), a leading global biopharmaceutical services provider, today announced that it has launched a new end-to-end service offering to provide real-world data insights that help life science companies more effectively demonstrate product value.

“In an increasingly competitive market, leveraging data to demonstrate value is critical. However, unstructured data can make it difficult to overcome gaps in real-world evidence,” said Joshua Schultz, Senior Vice President and Worldwide Head of PAREXEL Access. “Our new service will allow clients to more seamlessly collect and communicate the specific data that payers, physicians, and patients require to establish that a new product offers greater benefits than the existing standard of care.”

Category: Submitted Articles

Published: 06 September 2017

Written by RealWire