Category - Developments

Developments

Mustang Bio MB-107 gets FDA rare pediatric disease designation

The FDA granted Mustang Bio’s (NASDAQ:MBIO) MB-107 rare pediatric disease designation for the treatment of X-linked severe combined immunodeficiency. Patients with X-linked severe combined immunodeficiency produce...

August 17, 2020

Developments

Imara doses first patient in Phase 2b sickle cell disease trial

Imara (NASDAQ:IMRA) dosed the first patient in its Phase 2b Ardent trial evaluating IMR-687 for the treatment of sickle cell disease. The trial will enrol some 99 patients with sickle cell disease, who will receive...

August 13, 2020

Developments

Scholar Rock gets FDA rare pediatric disease designation for spinal muscular atrophy

Scholar Rock (NASDAQ:SRRK) received FDA rare pediatric disease designation for SRK-015 for the treatment of spinal muscular atrophy. Spinal muscular atrophy is a rare genetic disease that leads to motor function...

August 13, 2020

Developments

AzurRx BioPharma reports positive interim data from Phase 2 cystic fibrosis trial

AzurRx BioPharma (NASDAQ:AZRX) reported positive results from the first five patients in its Phase 2 combination therapy trial of MS1819 for the treatment of severe exocrine pancreatic insufficiency (EPI) in patients...

August 12, 2020

Developments

FDA approves Lantheus’ VIALMIXRFID activation device

The FDA approved Lantheus’ (NASDAQ:LNTH) supplemental drug application for VIALMIXRFID, a device that activates the company’s DEFINITY injectable suspension. DEFINITY injectable suspension is an ultrasound imaging agent...

August 12, 2020

Developments

Hepion CRV431 antiviral benefits published in PLOS ONE

Hepion Pharmaceuticals (NASDAQ:HEPA) reported that the peer-reviewed journal, PLOS ONE, has published a paper highlighting the antiviral effects of CRV431. Co-authored by Hepion’s CSO, Dr. Daren Ure and CEO...

August 12, 2020

Developments

Ocugen OCU400 gets fourth FDA orphan drug designation for retinal disease

Ocugen’s (NASDAQ:OCGN) OCU400 received FDA orphan drug designation for the treatment of PDE6B gene mutation-associated retinal disease. OCU400 consists of a nuclear hormone receptor gene, NR2E3, which is delivered to...

August 10, 2020

Developments

Omeros reports positive results from COVID-19-associated ARDS trial

Omeros (NASDAQ:OMER) reported positive results from a study evaluating narsoplimab for the treatment of COVID-19-associated acute respiratory distress syndrome (ARDS). Narsoplimab is a human monoclonal antibody that is...

August 10, 2020

Developments

Seres SER-109 meets primary endpoint in Phase 3 study of C. difficile infection

Seres Therapeutics (NASDAQ:MCRB) reported positive topline results from its Phase 3 trial evaluating SER-109 for the treatment of recurrent C. difficile infection. The study enrolled 182 patients with multiply...

August 10, 2020

Developments

Kazia gets FDA rare pediatric disease designation for paxalisib

Kazia Therapeutics (NASDAQ:KZIA) received FDA rare pediatric disease designation for paxalisib for the treatment of diffuse intrinsic pontine glioma (DIPG). Paxalisib is currently in a Phase 1 trial for DIPG. Positive...

August 7, 2020

Category - Developments

Mustang Bio MB-107 gets FDA rare pediatric disease designation

Imara doses first patient in Phase 2b sickle cell disease trial

Scholar Rock gets FDA rare pediatric disease designation for spinal muscular atrophy

AzurRx BioPharma reports positive interim data from Phase 2 cystic fibrosis trial

FDA approves Lantheus’ VIALMIXRFID activation device

Hepion CRV431 antiviral benefits published in PLOS ONE

Ocugen OCU400 gets fourth FDA orphan drug designation for retinal disease

Omeros reports positive results from COVID-19-associated ARDS trial

Seres SER-109 meets primary endpoint in Phase 3 study of C. difficile infection

Kazia gets FDA rare pediatric disease designation for paxalisib

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