Synageva BioPharma Corp. (“Synageva”) (NASDAQ:GEVA), a clinical stage biopharmaceutical company developing therapeutic products for rare disorders, today reported 2011 full year financial results and provided 2012 financial guidance.
“2011 marked a successful year for the company as we transitioned from a private to a public company. We achieved our goals of further developing our lead program, SBC-102 for LAL Deficiency, as well as advancing our other pipeline programs,” said Sanj K. Patel, President and Chief Executive Officer of Synageva BioPharma. “A strong financial position at the beginning of this year allows us to aggressively develop SBC-102 and our pipeline in order to stay true to our mission, which is to make a meaningful impact on the lives of patients suffering from rare diseases.”
Recent Company Highlights
Synageva reported progress on a number of key objectives during the past year including the following:
- Initiation and completion of enrollment of the Phase I/II trial of SBC-102 in adults with LAL Deficiency
- Report of encouraging interim results from the Phase I/II trial of SBC-102 in adults with LAL Deficiency at the 2012 LDN WORLD Symposium consistent with predicted pharmacodynamic effects
- Continued treatment of the early onset LAL Deficiency (Wolman) infant receiving SBC-102 initially through an expanded access program and now through the clinical study
- Preclinical data highlighting good cellular uptake of SBC-103, an enzyme replacement therapy under development for Sanfilippo B, at the 2012 LDN WORLD Symposium
- Strengthened balance sheet as a result of completing the reverse merger with Trimeris and a follow-on offering in January 2012 resulting in approximately $145 million in cash and cash equivalents
- Transition patients from the Phase I/II late onset LAL Deficiency trial with SBC-102 to the extension study
- Continue enrollment of the Phase I/II early onset LAL Deficiency trial with SBC-102
- Engage with US and European regulatory authorities on the design of the double-blind, placebo-controlled trial of SBC-102 for late onset LAL Deficiency
- Develop awareness about LAL Deficiency and continue to identify infants, children and adults who suffer from this devastating disorder
- Advance preclinical programs
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