Updated Data for Indoximod Plus KEYTRUDA ® (pembrolizumab) Demonstrate Improvement of Response Rate for Patients with Advanced Melanoma

Pivotal Trial of Indoximod in Advanced Melanoma to Include Both PD-1 Inhibitors, KEYTRUDA (pembrolizumab) and OPDIVO® (nivolumab)
 
AMES, Iowa --  09-07-2017 - NewLink Genetics Corporation (NASDAQ: NLNK) today announced updated data from the ongoing Phase 2 NLG2103 study of indoximod, NewLink Genetics’ IDO pathway inhibitor, in combination with the PD-1 pathway inhibitor, KEYTRUDA (pembrolizumab). These data will be highlighted in an oral presentation at the Third International Cancer Immunotherapy Conference in Frankfurt/Mainz, Germany, on September 9, 2017 by Yousef Zakharia, M.D., Assistant Professor of Medicine, Division of Hematology, Oncology and Blood & Marrow Transplantation at the University of Iowa and Holden Comprehensive Cancer Center.
Published: 07 September 2017
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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. 
Published: 06 September 2017
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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.
Published: 06 September 2017
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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
Published: 06 September 2017
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FDA Grants Orphan Drug Status to Cellect's ApoGraft ™ for Acute GvHD and Chronic GvHD

ApoGraft™ aims to solve Bone Marrow Transplantation (BMT) and other transplant rejection associated diseases
Cellect's CEO, Dr. Shai Yarkoni commented "We may provide the answer to a great unmet clinical need causing severe morbidity and death in thousands of patients worldwide. The Orphan Drug status gives us 7 years exclusivity following approval as well as other advantages for ApoGraft™ commercialization."
 
TEL AVIV, Israel, Sept. 5, 2017 -- Cellect Biotechnology Ltd. (NASDAQ: APOP), a developer of stem cells selection technology, announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for Cellect's ApoGraft™ for the prevention of acute and chronic graft versus host disease(GvHD) in transplant patients.
Published: 05 September 2017
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