Capricor Receives Rare Pediatric Disease Designation from FDA for CAP-1002 for Patients with Duchenne Muscular Dystrophy

Designation Covers Broad Treatment of Duchenne Muscular Dystrophy (DMD)

LOS ANGELES, July 18, 2017 -- Capricor Therapeutics, Inc. (NASDAQ: CAPR), a biotechnology company developing biological therapies for Duchenne muscular dystrophy and other rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to CAP-1002, Capricor's development candidate for the treatment of Duchenne muscular dystrophy, a debilitating genetic disorder characterized by progressive weakness and chronic inflammation of skeletal, heart, and respiratory muscles. The Rare Pediatric Disease Designation, as well as the Orphan Drug Designation previously granted to CAP-1002 by the FDA, covers the broad treatment of DMD. Upon receiving market approval for CAP-1002 by the FDA, Capricor would be eligible to receive a Priority Review Voucher.

Published: 18 July 2017

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CymaBay Announces Positive Interim Results from Its Ongoing Low-Dose Phase 2 Study of Seladelpar in Patients with Primary Biliary Cholangitis

Strong reduction in alkaline phosphatase of 39% (5 mg) and 45% (10 mg) at week 12

No safety signal for transaminase elevation or drug-induced itch

Potential for superior efficacy and better tolerability than existing second-line therapy

FDA agrees to extend dosing of 5 mg and 10 mg beyond six months

Conference call today at 8:30 a.m. ET

NEWARK, Calif., July 17, 2017 -- CymaBay Therapeutics, Inc. (Nasdaq:CBAY), today announced positive interim results from its ongoing low-dose Phase 2 study of seladelpar in patients with primary biliary cholangitis (PBC), a life-threatening and life-limiting chronic cholestatic liver disease. In the first part of the study, patients at high risk of disease progression, with an inadequate response to ursodeoxycholic acid (UDCA), as characterized by a persistent elevation in alkaline phosphatase (AP), or who were intolerant to UDCA, received either 5 mg or 10 mg of seladelpar once-daily.

Published: 17 July 2017

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Nasdaq Up on News Project Update

Data on Delivery of Tumor Treating Fields to be Presented at the 39th Annual International Conference of the IEEE Engineering in Medicine and Biology Society

Three oral presentations underscore Novocure’s commitment to further understanding the physical application of Tumor Treating Fields to enhance efficacy

Novocure (NASDAQ: NVCR) announced today that three oral presentations on Tumor Treating Fields (TTFields) will be given at the 39th Annual International Conference of the IEEE Engineering in Medicine and Biology Society (EMBS) on July 11 through July 15 in Jeju Island, Korea. The presentations include Novocure’s latest data on evaluating induced electric fields in human head models, evaluating electric field distribution in patients treated using human models, and determining the dielectric properties of human skin.

Published: 11 July 2017

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Nasdaq Up on News Project Update

U.S. FDA Confirms Amicus Therapeutics May Submit New Drug Application for Migalastat for Fabry Disease

Data Deemed Sufficient to Support NDA Submission --- NDA Submission Targeted for 4Q17

CRANBURY, N.J., July 11, 2017 -- Amicus Therapeutics (NASDAQ:FOLD) plans to submit a new drug application (NDA) to the U.S. FDA for the oral precision medicine migalastat for Fabry disease in the fourth quarter of 2017. Based on a series of discussions with and written communication received from the FDA, the Agency has informed Amicus that it may now submit an NDA for migalastat.

Amicus is preparing the NDA submission under Subpart H, which provides for accelerated approval. Amicus intends to base its NDA on existing data, including reduction in disease-causing substrate (GL-3), as well as the totality of data from completed clinical studies. Progressive accumulation of GL-3 is believed to lead to the morbidity and mortality of Fabry disease, including pain, kidney failure, heart disease and stroke. An additional Phase 3 study previously requested by the Agency to assess Gastrointestinal (GI) symptoms is no longer required prior to an NDA submission.

Published: 11 July 2017

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Nasdaq FDA HC Decision Up on News

Hemispherx Updates Status of Immuno-Oncology Program in Pancreatic Cancer

PHILADELPHIA, July 10, 2017 -- Hemispherx Biopharma (NYSE MKT:HEB) announced that 12 pancreatic patients are currently undergoing treatment with single-agent Ampligen immuno-oncology therapy in an Early Access Program (EAP) managed by Amsterdam-based myTomorrows, an international leader in providing physician access to experimental medicines.

Eight of the 12 patients entered the EAP with metastatic disease (late stage) and have been on drug for 12 weeks or longer. The four other patients entered the program in the last several weeks with earlier stage disease.

The program is being conducted at Erasmus Medical Center in Rotterdam under the supervision of lead clinician C.H.J. (Casper) van Eijck, MD, a noted Dutch oncologist specializing in pancreatic cancer.

Published: 10 July 2017

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AMEX Up on News Project Update

Capricor Therapeutics ( CAPR )

Capricor Receives Rare Pediatric Disease Designation from FDA for CAP-1002 for Patients with Duchenne Muscular Dystrophy

CymaBay Therapeutics Inc ( CBAY )

CymaBay Announces Positive Interim Results from Its Ongoing Low-Dose Phase 2 Study of Seladelpar in Patients with Primary Biliary Cholangitis

Novocure ( NVCR )

Data on Delivery of Tumor Treating Fields to be Presented at the 39th Annual International Conference of the IEEE Engineering in Medicine and Biology Society

Amicus Therapeutics ( FOLD )

U.S. FDA Confirms Amicus Therapeutics May Submit New Drug Application for Migalastat for Fabry Disease

Hemispherx Biopharma ( HEB )

Hemispherx Updates Status of Immuno-Oncology Program in Pancreatic Cancer