Clinical TrialsInformation and insight on the latest clinical trials
- Phase 3 Trial Results of Endari in Sickle Cell Disease Published
Emmaus Life Sciences announced that the New England Journal of Medicine (NEJM) has published the results of its 48-week phase 3 clinical trial of Endari (L-glutamine oral powder) which supported the FDA approval in 2017 to reduce the acute complications of sickle cell disease in adult and pediatric patients 5 years of age and older.
- Preliminary Results from Phase 2 Trial in Patients with Charcot-Marie-Tooth Disease Show Promise
There seems to be some good news on the horizon for individuals with Charcot-Marie-Tooth (CMT) disease. Acceleron Pharma announced positive preliminary results from Part 1 of the Phase 2 clinical trial with ACE-083 in patients with CMT disease at the Peripheral Nerve Society (PNS) annual meeting.
- The Right to Try Act
Mr. Robert (Bob) E. Ward, Chairman of the Board and Chief Executive Officer of Eloxx Pharmaceuticals discusses Right To Try, which is aimed at allowing terminally ill Americans to try medicines that have passed Phase 1 of the FDA approval process and remain in clinical trials but are not yet on pharmacy shelves.
- Dilanubicel Receives Orphan Drug Status for Reduction of Morbidity and Mortality Associated With HSCT
The U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to dilanubicel (Nohla Therapeutics) for the reduction of morbidity and mortality associated with hematopoietic stem cell transplant (HSCT).
- Human Gene Therapy for Rare Diseases: Draft Guidance for Industry
Statement from FDA Commissioner Scott Gottlieb, MD on agency’s efforts to advance development of gene therapies
- Scioto Biosciences: Focused on Reducing Necrotizing Enterocolitis
Jim Schulz, Co-founder and Chief Financial Officer of Scioto Biosciences, discusses his company’s focus on developing therapies devoted to having a impact on the delivery of microbiome therapeutics with the goal of reducing the incidence of necrotizing enterocolitis (NEC).
- ArQule: Focused on Targeted, Biomarker-Defined Patient Population
Brian Schwartz, Chief Medical Officer of ArQule, discusses his company and it’s clinical-stage pipeline consistlng of five drug candidates, all of which are in targeted, biomarker-defined patient populations.
- APOLLO Phase 3 Clinical Study Results for Hereditary Transthyretin-Mediated Amyloidosis
Alnylam Pharmaceuticals announced the publication of the pivotal study results from the APOLLO Phase 3 trial of patisiran in The New England Journal of Medicine (NEJM).
- FDA Grants Fast Track Designation to CUTX-101 for Classic Menkes Disease
The U.S. Food and Drug Administration (FDA) granted Fast Track Designation to Cyprium Therapeutics’ Copper Histidinate, (CUTX-101), for patients diagnosed with classic Menkes disease who have not demonstrated significant clinical progression.
- Alnylam’s Pending Approval of Patisiran
Barry Greene, President of Alnylam, discusses his company and it’s pending approval of Patisiran, an investigational RNAi therapeutic targeting transthyretin (TTR) in development for the treatment of hereditary ATTR amyloidosis (hATTR amyloidosis).