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Neose’s various technologies evolve from the same core – the use of enzymes to complete and modify carbohydrate structures on glycoproteins. We have developed a unique expertise and a strong intellectual property position in this area. The technology core is broadly applicable and renewable, providing an opportunity for sustainable growth as new applications are developed.

Neose is using its GlycoAdvance and GlycoPEGylation technologies to develop proprietary drug candidates, as well as to partner with biotech and pharmaceutical companies. Our primary focus is developing next generation therapeutic proteins that are improved versions of currently marketed proteins with proven efficacy.

GlycoConjugation is an emerging application of the Company’s technology. It has the potential to produce novel therapeutic glycoproteins by conjugating bioactive or functional compounds to the glycans on antibodies and other glycoproteins.

Our Lead Candidates

GlycoPEG-GCSF -- Together with our partner, BioGeneriX AG, we are developing GlycoPEG-GCSF for treatment of neutropenia associated with myelosuppressive chemotherapy. In 2007, our partner completed two Phase I trials of GlycoPEG-GCSF described below.

Trial	Phase I Ascending Dose	Phase I Fixed Dose
Objectives	Safety and tolerability; dose response/change in absolute neutrophil count; pharmacokinetics
Study Design	Single center; open label; sequential, ascending dose	Single center; open label; fixed dose
Planned Dosing	25, 50, 100 and 200 ug/kg vs. Neulasta 100 ug/kg, SC	6 mg. vs. 6 mg. Neulasta, SC
Trial Size	n = 53 healthy volunteers	n = 36 healthy volunteers

In November 2007, Neose and BioGeneriX shared data from these trials. The first Phase I study evaluated a randomized, weight-based, single ascending dose of GlycoPEG-GCSF compared to Neulasta® in 53 healthy subjects. Conclusions from this trial are as follows:

• Single doses of GlycoPEG-GCSF demonstrated potent stimulation of neutrophils and mobilization of peripheral blood progenitor cells.
• GlycoPEG-GCSF demonstrated an approximately 30% greater absolute neutrophil count response versus Neulasta.
• GlycoPEG-GCSF demonstrated an approximately 60% increased bioavailability versus Neulasta®.
• Single doses of GlycoPEG-GCSF, up to 100 µg/kg, were generally well-tolerated with no serious adverse events.
• Adverse events were comparable to those for Neulasta® and there were no discontinuations due to adverse events.
• There were no antibodies to GlycoPEG-GCSF detected.

The second Phase I study evaluated a 6 mg dose of GlycoPEG-GCSF versus a 6 mg dose of Neulasta® in 36 healthy subjects. Conclusions from this trial are as follows:

• GlycoPEG-GCSF demonstrated potent stimulation of neutrophils and mobilization of peripheral blood progenitor cells.
• GlycoPEG-GCSF demonstrated an approximately 30% greater absolute neutrophil count response versus Neulasta.
• GlycoPEG-GCSF demonstrated an approximately 60% increased bioavailability versus Neulasta®.
• GlycoPEG-GCSF was generally well-tolerated with no serious adverse events.
• Adverse events were comparable to Neulasta, and there were no discontinuations due to adverse events.
• There were no antibodies to GlycoPEG-GCSF detected.

Click here for the full data presentation.

GlycoPEGylated Factors VIIa, VIII and IX -- We have licensed these coagulation factors for the treatment of hemophilia to Novo Nordisk A/S. In June 2007, Novo Nordisk commenced a Phase I trial to assess the safety and pharmacokinetics of GlycoPEGylated Factor VIIa in healthy volunteers. During 2007, preclinical data on Factor VIIa was presented at the annual meetings of the International Society on Thrombosis and Hemostasis and the American Society of Hematology. Copies of these poster presentations are available on our Presentations page. Factors VIII and IX are currently in research.

Capabilities

Neose’s technologies require recombinant enzymes (glycosyltransferases) and donor sugars (sugar nucleotides). These glycosyltransferases are highly specific and will add a sugar unit only to the target acceptor. Neose has developed a library of these enzymes that can be produced economically in fungal and other expression systems. Neose has also developed technology to produce sugar nucleotides at large-scale.

Co-Development Opportunities

Neose is interested in collaborating with biotechnology and pharmaceutical industry partners to use GlycoPEGylation in the development of their proprietary products. To learn more about how these technologies may be used to improve products in development or on the market, please call a business development representative at 866-251-6200 or submit an information request.