Investors in the pharma/biotech sector eagerly wait for pipeline updates as they play an important role in deciding whether or not to invest in a particular company. Additionally, with pipeline updates, companies sometimes provide updates on the regulatory front including events like new designations for their candidates, filing of marketing applications or acceptance of these applications for review by regulatory agencies.

These updates provide information on experimental drugs and at times give an insight on the commercial potential of the candidate once it is successfully developed and commercialized.

Earlier this week, Synageva BioPharma Corp. (GEVA) announced the commencement of patient dosing in a phase I/II study on its investigational candidate, SBC-103. The company is developing SBC-103 as an enzyme replacement therapy for mucopolysaccharidosis IIIB (MPS IIIB, also known as Sanfilippo B syndrome).

Synageva said this is the first study on an enzyme replacement therapy for MPS IIIB. In this study, the company will enroll approximately nine patients in the age group of 2 years to less than 12 years in three different dosing cohorts (0.3 mg/kg, 1.0 mg/kg or 3.0 mg/kg). Patients will be treated with intravenous administrations of SBC-103 every alternate week for 24 weeks. 

Those who meet the qualifying criteria may continue to receive SBC-103 for an extended treatment period of up to 128 weeks. The primary endpoint of the study is to evaluate the safety and tolerability of intravenous administration of SBC-103 in patients suffering from MPS IIIB. Preliminary data from the study should be out during the second half of 2015.

Additionally, Synageva reported that SBC-103 has been granted fast track status by the FDA. Fast track designation is usually granted to candidates that are considered to have the potential to treat serious or life-threatening conditions and which may fulfill an unmet medical need. This status should facilitate the development process for SBC-103 and expedite review of the candidate. SBC-103 also enjoys orphan drug designation in the U.S. and the EU.

We note that in Dec 2014, the FDA had granted orphan drug designation to BioMarin Pharmaceutical’s (BMRN) BMN 250. BioMarin intends to initiate studies on BMN 250 for the treatment of MPS IIIB by mid-2015.

Synageva carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the health care sector are Celgene Corp. (CELG) and Regado Biosciences, Inc. (RGDO). Both carry a Zacks Rank #1 (Strong Buy).

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