SAGE Receives Special Protocol Assessment for Phase 3 STAT-Refractory Statusof SAGE-547; Ultragenyx Announces Initiation of Phase 2 Study of Recombinant Human Beta-Glucuronidase Print E-mail
By William Kent   
Thursday, 06 August 2015 18:54
Below is a look at some of the headlines for companies that made news in the healthcare sector on August 6, 2015. 
 
SAGE Therapeutics (NASDAQ: SAGE) announced it has reached agreement with the U.S. Food and Drug Administration (FDA) under a Special Protocol Assessment (SPA) for the STATUS Trial (SAGE-547 Treatment as Adjunctive Therapy Utilized in Status Epilepticus), a global, Phase 3, randomized, double-blind, placebo-controlled clinical trial to evaluate SAGE-547 as a treatment for patients with super-refractory status epilepticus (SRSE). The SPA provides agreement from the FDA that the Phase 3 STATUS Trial can adequately address objectives in support of a U.S. regulatory submission for approval of SAGE-547 for the treatment of patients with SRSE. SRSE is a rare, life-threatening condition of persistent, unremitting seizure, for which there are no approved therapies.
    
"This is a major accomplishment for SAGE Therapeutics. By obtaining FDA agreement on the Phase 3 STATUS Trial design, endpoints, patient population and statistical approach, we have a clear path on moving forward with our development and regulatory plan for SAGE-547 in SRSE. As first movers in seeking a treatment for this life-threatening disorder, we believe our SPA is an important mechanism to further de-risk our development pathway. We look forward to enrolling patients in the Phase 3 STATUS Trial imminently," said Jeff Jonas, M.D., chief executive officer of SAGE Therapeutics.
    
The STATUS Trial is designed to assess the efficacy and safety of SAGE-547 in approximately 126 patients with SRSE, aged two years or older, and will be conducted in the U.S., Canada and Europe. Patients will be randomized 1:1 to receive either SAGE-547 or placebo in addition to standard-of-care third-line anti-seizure agents for six days. The planned primary endpoint of the Phase 3 clinical trial will be successful resolution of status epilepticus (SE) after weaning the patient off all third-line agents, and SAGE-547 or placebo, without resumption of SE within 24 hours after completion of blinded SAGE-547 or placebo administration.
    
SAGE's Phase 3 open-label expanded access protocol, designated Study 302, was initiated in April 2015 and continues its enrollment. Study 302 is designed to make SAGE-547 available to patients in the U.S. who are affected by SRSE and who have not been admitted to, nor can be transferred to, a planned STATUS Trial clinical site.
    
In a completed Phase 1/2 open-label clinical trial, SAGE-547 demonstrated robust activity, with a 77 percent response rate in 22 evaluable patients with SRSE, and also a favorable tolerability profile. Independent of treatment response, six patient deaths occurred within the trial period, all driven by underlying conditions. Although 64 percent of patients reported serious adverse events, none were drug-related as determined by the Safety Review Committee.
    
About Special Protocol Assessments  -  The Special Protocol Assessment (SPA) process is a procedure by which the FDA provides official evaluation and
written guidance on the design and

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size of proposed protocols that are intended to form the basis for a new drug application.
    
Final marketing approval depends on the results of efficacy, the adverse event profile and
an evaluation of the benefit/risk of treatment demonstrated in the Phase 3 clinical program. The SPA agreement may only be changed through a written agreement between the sponsor and the FDA, or if the FDA becomes aware of a substantial scientific issue essential to product efficacy or safety.
   
   
   
   
   
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Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), announced the dosing of the first patient in a Phase 2 study of investigational recombinant human beta-glucuronidase (rhGUS, UX003) in patients under five years old with mucopolysaccharidosis 7 (MPS 7, Sly syndrome), potentially including patients with non-immune hydrops fetalis, a severe infantile presentation of the disease.
   
"This new Phase 2 study will enable us to assess the benefit of early intervention with rhGUS in very young patients as well as the potential to prevent some of the devastating consequences of this disease," said Sunil Agarwal, M.D., Chief Medical Officer of Ultragenyx. "The resulting data from patients under five years old, which can include infants with hydrops fetalis, will complement the data from the ongoing Phase 3 study in older patients with MPS 7."
   
The Phase 2 open-label, multi-center clinical study will assess the safety, tolerability, and efficacy of rhGUS in up to seven pediatric patients under five years old. Patients will receive 4 mg/kg of rhGUS treatment every other week for 48 weeks, followed by a long-term continuation period. The primary efficacy endpoint is the reduction in urinary GAG excretion. Additional efficacy measures include growth velocity, hepatosplenomegaly, functional development, and cardiac and pulmonary function. Interim data from the study are expected by the end of 2016.
   
This study is also intended to enroll patients with non-immune hydrops fetalis, a phenotype of MPS 7 in which patients are born with severe edema and often die within a few months to one year. Ultragenyx is currently treating one MPS 7 patient born with hydrops fetalis under compassionate use.
    
FDA Fast Track Designation  --  Ultragenyx also announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to the rhGUS program in MPS 7. Fast Track Designation is intended to facilitate the development and expedite the review of drugs for serious and life-threatening conditions that have the potential to address an unmet medical need. The designation allows for more frequent interaction with the FDA review team. It also enables eligibility for priority review and the potential for a rolling review of the Biologics License Application, when and if filed.
  
  
  
  
  
Also Thursday:
  
  
  
  
  
Aduro Biotech, Inc. (Nasdaq:ADRO) today announced that Stephen T. Isaacs, chairman, president and chief executive officer of Aduro, will present at the 35th Annual Canaccord Genuity Growth Conference on Wednesday, August 12, 2015, at 4:30 pm Eastern Time.
   
Air Methods Corporation (Nasdaq:AIRM), the global leader in air medical transportation, reported financial results for the quarter and six months ended June 30, 2015 and provided an update on preliminary July 2015 flight volume. 
    
Amyris, Inc. (Nasdaq:AMRS), the industrial bioscience company, today announced financial results for the second quarter ended June 30, 2015.
   
Athersys, Inc. (NASDAQ:ATHX) today announced its financial and operating results for the three months ended June 30, 2015.
    
Appriss Inc., a leading technology provider for risk assessment and analytics in public safety and health care, announced that Cerner (Nasdaq: CERN), a global leader in health care techonology, has integrated NARxCHECK into its electronic health record (EHR) system in an effort to fight prescription drug abuse and diversion nationwide.
  
Caladrius Biosciences, Inc. (NASDAQ:CLBS), a cell therapy leader with a Phase 3 clinical program in immuno-oncology and a portfolio of projects in immune modulation and regenerative medicine, announced today that it will report its financial results for the second quarter ended June 30, 2015 today, August 6, 2015. 
   
Caladrius Biosciences, Inc. (Nasdaq:CLBS), a company combining a leading cell therapy service provider with a development pipeline including a Phase 3 clinical program in immuno-oncology and a portfolio of projects in immune modulation and ischemic repair, announced today 2015 second quarter results.
   
CareDx, Inc. (Nasdaq:CDNA), a molecular diagnostics company focused on the discovery, development and commercialization of actionable diagnostic surveillance solutions for organ transplant recipients, and DNAnexus Inc., the leader in cloud-based genome informatics and data management, today announced that CareDx has selected DNAnexus' cloud genomics platform to support its ongoing R&D activities as well as clinical trials and registries. 
   
ChemoCentryx, Inc., (Nasdaq:CCXI), a clinical-stage biopharmaceutical company focused on autoimmune diseases, inflammatory disorders and cancer, today reported financial results for the second quarter ended June 30, 2015 and provided an update on the Company's corporate and clinical development activities.
    
Entellus Medical, Inc. (NASDAQ:ENTL), a medical technology company focused on the design, development and commercialization of products for the minimally invasive treatment of chronic and recurrent sinusitis patients in the physician office setting or operating room, today reported its financial results for the quarter ended June 30, 2015.
    
Fate Therapeutics, Inc. (NASDAQ:FATE), a biopharmaceutical company engaged in the development of programmed cellular immunotherapeutics for the treatment of severe, life-threatening diseases, today announced that management will be presenting at two upcoming investor conferences in August: 2015 Wedbush PacGrow Healthcare Conference. Christian Weyer, M.D., M.A.S., President and Chief Executive Officer, will present a company overview at 9:10 a.m. ET on Tuesday, August 11, 2015 in New York City;   Canaccord Genuity 35th Annual Growth Conference. Scott Wolchko, Chief Operating and Financial Officer, will present a company overview at 2:30 p.m. ET on Wednesday, August 12, 2015 in Boston.
    
FibroGen, Inc. (NASDAQ:FGEN), a research-based biopharmaceutical company, today announced that Jeffrey W. Henderson has joined the company's board of directors.
   
Five Prime Therapeutics, Inc. (Nasdaq:FPRX), a clinical-stage biotechnology company focused on discovering and developing novel protein therapeutics for cancer and inflammatory diseases, today provided a corporate update and reported financial results for the second quarter ending June 30, 2015.
    
Flexion Therapeutics, Inc. (Nasdaq:FLXN) today announced financial results for the second quarter ended June 30, 2015.
   
Galena Biopharma, Inc. (NASDAQ:GALE), a biopharmaceutical company developing and commercializing innovative, targeted oncology therapeutics that address major medical needs across the full spectrum of cancer care, today reported its financial results for the quarter ended June 30, 2015 and provided a business update.
    
HTG Molecular Diagnostics, Inc. (NASDAQ:HTGM), a provider of instruments and reagents for molecular profiling applications, today reported financial results for the three and six months ended June 30, 2015.
   
IASIS Healthcare┬« LLC today announced that it will provide an online listen-only Web simulcast of its fiscal third quarter 2015 conference call on Thursday, August 13, 2015. 
   
Kips Bay Medical, Inc. (OTCQB:KIPS), a medical device company that was focused on manufacturing and commercializing its external saphenous vein support technology, eSVS® Mesh, for use in coronary artery bypass grafting surgery, today provided a business update and announced financial results for its second quarter ended June 27, 2015.
   
Ohr Pharmaceutical, Inc. (Nasdaq:OHRP), an ophthalmology research and development company, today reported results for its third fiscal quarter ended June 30, 2015.
    
Oncothyreon Inc. (NASDAQ:ONTY) today reported financial results for the second quarter ended June 30, 2015.
   
Perseon Corporation (NASDAQ:PRSN) is pleased to announce results of data presented on Sunday, July 12 at the 57th annual American Association of Physicists in Medicine (AAPM) conference, in Anaheim, California. 
   
Raptor Pharmaceutical Corp. (NASDAQ:RPTP) today announced financial results for the three and six months ended June 30, 2015 and provided an update on recent corporate developments.
    
StemCells, Inc. (NASDAQ:STEM), a world leader in the research and development of cell-based therapeutics for the treatment of disorders of the central nervous system, today reported its financial results for the three months ended June 30, 2015. 
     
TearLab Corporation (NASDAQ:TEAR) (TSX:TLB) today reported its consolidated financial results for the second quarter ended June 30, 2015. All dollar amounts are expressed in U.S. currency and results are reported in accordance with United States generally accepted accounting principles.
   
TESARO, Inc. (NASDAQ:TSRO), an oncology-focused biopharmaceutical company, today reported operating results for second-quarter 2015 and provided an update on the Company's development programs.
    
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that it will host a conference call on Thursday, August 13, 2015 at 5pm ET to discuss second quarter 2015 financial results and provide a corporate update.
  
XOMA Corporation (Nasdaq:XOMA), a leader in the discovery and development of therapeutic antibodies, today provided a corporate update and reported its financial results for the quarter ended June 30, 2015.



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