Second Quarter 2014 Financial Results
For the quarter ended
Synageva reiterates its previous net loss guidance of between
Updates to Ongoing Clinical Trials with Sebelipase Alfa for LAL Deficiency
Synageva continues to progress additional clinical trials to expand the clinical experience with sebelipase alfa in LAL Deficiency. The data from these trials are not required for the planned regulatory submissions for sebelipase alfa for LAL Deficiency. Eligible patients may include those who were not able to enroll into one of the previously completed studies based on completion of enrollment, age, disease presentation, previous treatment by hematopoietic stem cell or liver transplantation, or disease characteristics that would have precluded participation in a placebo-controlled study.
Study LAL-CL06 is an open-label trial to assess the safety of sebelipase alfa in a broad LAL Deficiency patient population. Infants (greater than eight months of age), children and adults with LAL Deficiency with evidence of advanced liver disease or disease recurrence in patients with past liver or hematopoietic transplant, among other disease manifestations can be included in this study. Clinical trial site initiation began in
Study LAL-CL08 is an open-label trial to expand the clinical experience with sebelipase alfa in additional infants. The first of these patients was initially dosed under compassionate use in
Updates to SBC-103 for MPS IIIB
The company plans to initiate clinical trial sites later this year and dose the first patient with intravenous administration of SBC-103 in a Phase 1/2 study in patients with mucopolysaccharidosis IIIB (MPS IIIB) by late 2014 or early 2015. This is based upon longer-term preclinical toxicology data expected during the second half of 2014 that could support a longer Phase 1/2 clinical trial than originally planned.
Sebelipase Alfa for LAL Deficiency
LAL Deficiency is a rare autosomal recessive lysosomal storage disease (LSD) caused by a marked decrease in LAL enzyme activity. LAL Deficiency presenting in children and adults, historically called Cholesteryl Ester Storage Disease, is an underappreciated cause of cirrhosis and accelerated atherosclerosis. These complications are due to the buildup of fatty material in the liver, blood vessel walls and other tissues as a result of the decreased LAL enzyme activity. Infants presenting with LAL Deficiency, historically called Wolman disease, show very rapid progression with death, usually in the first six months of life. Affected infants develop severe liver complications, malabsorption, and growth failure.
Sebelipase alfa is a recombinant form of the human LAL enzyme being developed by Synageva as an enzyme replacement therapy for LAL Deficiency. Sebelipase alfa has been granted orphan designation by the
SBC-103 for MPS IIIB and Other Pipeline Programs
The mucopolysaccharidoses (MPS) consist of a group of rare LSDs caused by a deficiency of enzymes needed to break down complex sugars called glycosaminoglycans. The MPS III syndromes (also known as Sanfilippo syndromes) share complications with other MPS diseases but represent a clinically distinct subset with marked central nervous system degeneration. MPS IIIB, also known as Sanfilippo B syndrome, is caused by a decrease in alpha-N-acetyl-glucosaminidase (NAGLU) enzyme activity which leads to the buildup of abnormal amounts of heparan sulfates (HS) in the brain and other organs. The accumulation of abnormal HS, particularly in the central nervous system, leads to severe cognitive decline, behavioral problems, speech loss, increasing loss of mobility, and premature death.
SBC-103 is a recombinant form of the human NAGLU enzyme being developed by Synageva as an enzyme replacement therapy for MPS IIIB. Using various dosing approaches, SBC-103 reduced HS substrate storage in the brain, liver and kidney in an MPS IIIB animal model. SBC-103 has been granted orphan designation by the
Synageva continues to plan to advance two additional programs into clinical trials by the end of 2016. The company's pipeline programs include proteins targeting rare diseases at various stages of preclinical development. These diseases are characterized by significant morbidity and mortality and are selected based on scientific rationale, high unmet medical need, potential to impact disease course and strategic alignment with our corporate and commercial focus. Synageva owns the worldwide commercial rights to these programs.
Synageva routinely posts information that may be important to investors in the "Investor Relations" section of the company's website at www.synageva.com. Synageva encourages investors and potential investors to consult this website regularly for important information about the company.
Further information regarding Synageva is available at www.synageva.com.
Keywords for this news article include: Therapy, Pediatrics,
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