LEXINGTON, Mass., Feb. 26, 2015 /PRNewswire/ -- Synageva BioPharma Corp. (NASDAQ: GEVA), a biopharmaceutical company developing therapeutic products for rare disorders, today reported 2014 full year financial results and provided 2015 goals, other key objectives, and financial guidance. Synageva’s management team will host a conference call today at 4:30 p.m. EST to review the financial results and provide a general business update. To participate in today’s call by telephone, please dial (877) 445-4603 for U.S. callers or (443) 295-9270 for international callers. In addition, the conference call will be webcast live and may be accessed from the Investor Relations section of Synageva’s website at www.synageva.com.
Kanuma (sebelipase alfa) and LAL Deficiency
The company recently announced that the U.S. Food and Drug Administration (FDA) accepted for filing the Biologics License Application (BLA) for Kanuma for the treatment of lysosomal acid lipase deficiency (LAL Deficiency). The FDA granted the company’s request for Priority Review, which shortens the regulatory review period and is reserved for investigational therapies that treat serious conditions and, if approved, would provide a significant improvement in safety or effectiveness compared to available therapies. The FDA established a target action date of September 8, 2015 under the Prescription Drug User Fee Act (PDUFA). The FDA indicated that they are not currently planning to hold an advisory committee meeting for the Kanuma BLA. Synageva plans to expand its regulatory submissions for Kanuma beyond the BLA in the U.S. and the marketing authorization application in Europe to include Mexico and Japan during this year.
The company recently initiated an expanded access program in the U.S. for Kanuma for any person with a confirmed diagnosis of LAL Deficiency who does not otherwise qualify to participate in an ongoing clinical trial with Kanuma. The FDA allows expanded access to investigational products for people with serious and life-threatening illnesses who may not qualify to participate in a clinical trial. In addition to initiating the expanded access program in the U.S., the company recently established GevaCare, a global patient assistance program consisting of registered nurse case managers who, upon approval of Kanuma, will provide reimbursement information and supportive services for patients and their caregivers living with LAL Deficiency.
On February 13, 2015, the Japanese Patent Office issued Japanese Patent No. 5693728 relating to the treatment of LAL Deficiency with a patent term through 2031, not including any extension. This patent complements the patents recently issued in Europe (issued February 2015) and the U.S. (issued March 2014) relating to the treatment of LAL Deficiency, and is part of the planned global patent portfolio that covers the company’s LAL Deficiency program, including intellectual property directed to compositions of matter, methods of use and manufacturing.
SBC-103 for MPS IIIB
Dosing is underway in the Phase 1/2 trial with SBC-103 in patients with mucopolysaccharidosis IIIB (MPS IIIB, also known as Sanfilippo B syndrome). The open-label trial will enroll approximately nine patients, two years of age or greater but less than 12 years of age, with definitive diagnosis of MPS IIIB and developmental delay. Patients will be treated in one of three different dosing cohorts (0.3 mg/kg, 1.0 mg/kg or 3.0 mg/kg) with every other week intravenous administrations of SBC-103 for 24 weeks. Patients who meet qualifying criteria may continue dosing with SBC-103 for an extended period.
The primary endpoint of the trial is safety and tolerability of intravenous administration of SBC-103 in patients with MPS IIIB. The study will also determine the effects of dosing with SBC-103 on the onset, magnitude, and reversibility of changes in levels of total heparan sulfate (HS) in cerebral spinal fluid (CSF), serum, and urine as well as measure the effects of neurocognitive and developmental function and change in brain structures as assessed by magnetic resonance imaging. Exploratory biomarkers, SBC-103 concentration in CSF, MPS IIIB disease characteristics, symptoms, and quality of life will also be measured. The company plans to report preliminary data from this study during the second half of this year.
SBC-105 for GACI and other rare disorders of calcification
SBC-105 is Synageva’s third pipeline program and a first-mover enzyme replacement therapy in preclinical development for rare disorders of calcification, including the first planned indication for generalized arterial calcification of infancy (GACI). Patients with GACI suffer from calcification of the medium and large arteries leading to arterial narrowing and heart failure. The disease predominantly affects infants and children, and most patients with the disease die in early infancy.
In healthy individuals, an enzyme called ectonucleotide pyrophosphatase/phosphodiesterase-1 (ENPP1) generates pyrophosphate (PPi), which is a potent inhibitor of tissue calcification. Due to inherited mutations of ENPP1, patients with GACI have low levels of PPi, which results in increased calcification of the arteries and other tissues. SBC-105 is a recombinant enzyme that replaces the mutated ENPP1 enzyme and increases production of PPi. In a preclinical mouse model of GACI, SBC-105 improved survival, tissue calcification, and biomarkers associated with GACI. Beyond GACI, SBC-105 may have broader medical utility in other rare disorders of calcification including hypophosphatemic rickets and pseudoxanthoma elasticum, where low PPi levels contribute to pathological calcification with increased morbidity and mortality. The company plans to generate additional preclinical proof of concept data in disorders of calcification during this year.
Bio-superior pipeline programs
Synageva continues to explore opportunities to leverage the company’s manufacturing platform and other capabilities to create potentially bio-superior treatments for patient populations where there is still unmet medical need. The company has recently produced enzymes targeting Hunter syndrome, Fabry disease and Pompe disease with expression levels and activity that support further preclinical development. Ongoing preclinical development efforts are designed to generate proof of concept data including the potential for improved product characteristics for at least one of these programs this year.
Key Objectives for 2015:
- Advance the global regulatory process for Kanuma for LAL Deficiency in the U.S., Europe, Japan and Latin America.
- Continue global disease awareness programs to support identification of more patients with LAL Deficiency.
- Prepare for the global launch of Kanuma.
- Report preliminary data from the Phase 1/2 trial with SBC-103 in patients with MPS IIIB during the second half of 2015.
- Advance SBC-105 towards the clinic and generate preclinical proof of concept data for disorders of calcification.
- Advance bio-superior preclinical development efforts to generate proof of concept data including the potential for improved product characteristics for at least one of the Hunter syndrome/Fabry/Pompe disease programs.
2014 Full Year Financial Results
Revenue for the full year ended December 31, 2014 of $6.5 million included $6.0 million of Fuzeon royalties from Roche, as well as revenue of $0.5 million from collaborations and license fees. Total costs and expenses for the full year 2014, including research and development (R&D) expenses, sales, general and administrative (SG&A) expenses, and amortization of developed technology, totaled $198.6 million. This compares to total costs and expenses for the full year 2013 of $109.3 million.
Synageva had cash, cash equivalents and short-term investments totaling $446.9 million on December 31, 2014. The cash balance at December 31, 2014 does not include proceeds from the recent follow-on offering completed in January 2015 that resulted in $308.7 million in net proceeds to the company.
2015 Financial Outlook
Synageva currently expects total operating expenses between $280 million and $295 million for 2015. The total operating expense estimate is primarily due to investments supporting the global clinical development program for Kanuma, clinical development of SBC-103, preclinical development of SBC-105 and the potentially bio-superior programs, expansion of the global clinical, medical affairs and commercial infrastructure, expansion of manufacturing capabilities, as well as advancement of other preclinical pipeline 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.
Medical information regarding Kanuma and LAL Deficiency is available by email at medinfo@synageva.com.
Forward-Looking Statements
This news release contains “forward-looking statements”. Such statements generally can be identified by the use of words such as “anticipate,” “expect,” “plan,” “could,” “intend,” “believe,” “may,” “will,” “estimate,” “forecast,” “project,” or words of similar meaning.
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