Therapeutic Focus
Multiple Sclerosis
ALLOZYNE, has created AZ01 which is a PEGylated interferon β for the treatment of multiple sclerosis (MS), a chronic disease characterized by demyelination of nerve fibers, which leads to severe nerve damage. MS symptoms include fatigue as well as cognitive and visual impairment. There is no cure for MS which affects approximately 400,000 people in the US and 2.5 million people worldwide. Six drugs currently are approved for treating MS, including three brands of interferon β. These three interferon βs (Avonex™, Rebif™ and Betaseron™) in addition to glatimer acetate (Copaxone™) hold nearly 95% of the market with a combined annual sales of $8.59B and an annual growth rate of 12.3% in 2009. Despite their long standing commercial success, which has been built through stellar safety profiles, these therapies have significant limitations which lead to significant patient non compliance within the first 12 months of treatment. Dosing for these drugs is at least once weekly and can require up to seven injections per week. In addition, tolerability of these agents is somewhat limited due to formulation which can induce significant levels of injection site reactions. In addition, the interferon βs have been reported to induce flu like symptoms in as many as 50% of patients.
Leading MS clinicians believe there is significant need for new therapies that can offer improved efficacy and side effect profiles. In addition, there is a clear unmet medical need for more convenient dosing regimens and improved tolerability of these agents.
Considering the hypercompetitive arena that exists for long-acting formulations of IFNβ, we believe that AZ01 has several competitive advantages, including:
• Improved pharmacokinetic, safety and tolerability profile compared with currently available interferon β products approved for treatment of MS
• Significant dosing convenience through a potential monthly dosing regimen
• Efficient manufacturing process enabling high yields and competitive cost of goods at commercial scale
Crohn's Disease
ALLOZYNE, has created AZ01 which is a PEGylated interferon β for the treatment of multiple sclerosis (MS), a chronic disease characterized by demyelination of nerve fibers, which leads to severe nerve damage. MS symptoms include fatigue as well as cognitive and visual impairment. There is no cure for MS which affects approximately 400,000 people in the US and 2.5 million people worldwide. Six drugs currently are approved for treating MS, including three brands of interferon β. These three interferon βs (Avonex™, Rebif™ and Betaseron™) in addition to glatimer acetate (Copaxone™) hold nearly 95% of the market with a combined annual sales of $8.59B and an annual growth rate of 12.3% in 2009. Despite their long standing commercial success, which has been built through stellar safety profiles, these therapies have significant limitations which lead to significant patient non compliance within the first 12 months of treatment. Dosing for these drugs is at least once weekly and can require up to seven injections per week. In addition, tolerability of these agents is somewhat limited due to formulation which can induce significant levels of injection site reactions. In addition, the interferon βs have been reported to induce flu like symptoms in as many as 50% of patients.
Leading MS clinicians believe there is significant need for new therapies that can offer improved efficacy and side effect profiles. In addition, there is a clear unmet medical need for more convenient dosing regimens and improved tolerability of these agents.
Considering the hypercompetitive arena that exists for long-acting formulations of IFNβ, we believe that AZ01 has several competitive advantages, including:
• Improved pharmacokinetic, safety and tolerability profile compared with currently available interferon β products approved for treatment of MS
• Significant dosing convenience through a potential monthly dosing regimen
• Efficient manufacturing process enabling high yields and competitive cost of goods at commercial scale
Crohn's Disease
ALLOZYNE has created AZ17, which is a bispecific molecule for the treatment of inflammatory diseases such as Crohn’s disease. Crohn’s is a chronic inflammatory disease caused by an autoimmune attack mounted against tissues of the gastrointestinal tract. The disease typically progresses to chronic inflammation leading to fever, diarrhea, abdominal pain and permanent tissue damage. There is currently no cure for Crohn’s with a prevalence of over 400,000 in North America. The current standard of care for treatment of moderate to severe cases is an antibody based therapy known as a tumor necrosis factor (TNF) inhibitor. TNF inhibitors, such as Humira™, Remicade™ and Cimzia™, represented $8.6 billion or 63% of the 2006 autoimmune disease market and are expected maintain their pole position through 2016. Although TNF inhibitors hold a dominant market position, they are limited in their ability to treat inflammatory and autoimmune diseases by targeting this downstream inflammatory molecule.
AZ17 is composed of two scFvs linked together using our CAESAR platform and targets two upstream cytokines involved in the TH17 pathway. TH17 cells have been strongly associated with the development of inflammation and autoimmunity. These cells have a unique differentiation pathway, which provides specific points of intervention where their development and activation may be blocked.
We believe that AZ17’s novel mode of action will allow it to compete for this market space by offering the following competitive advantages:
• The independent inhibition of targeted cytokines is proven and safe
• Targeting multiple cytokines of the same pathway inherently increases responding patient population
• Low cost of goods due to expression in E. coli
• The nature of the linker affords customization of in vivo half-life to indication
Diabetes
Diabetes is one of the most challenging health problems of the 21st century. Current estimates for the US alone call for the doubling of the known patient population from today's 23 to 44 million over the next 25 years.
The standard care of therapy based on insulin injections has not changed meaningfully in the past 15 years. Although insulin has evolved from its bovine or porcine origin in the beginning to the new generations of recombinant human insulin and its analogs, it is thought that radical improvements in the treatment of diabetes will be achieved with next generation therapeutics targeting novel mechanisms of action.
Fibroblast Growth Factor 21 (FGF-21) belongs to the new class of therapeutics currently being explored for their ability to control blood glucose levels. Animal studies have shown that treatment with FGF21 lowers plasma glucose and increases insulin sensitivity. The beneficial effects of FGF21 are observed only after repeated dosing and prolonged exposure, making the FGF21 protein a challenging drug to develop and indicating an unmet medical need for a longer acting form.
ALLOZYNE has generated AZ21, a novel product candidate which is a long acting form of FGF21. AZ21 can overcome the need for multiple and frequent administration by its conjugation to a known and validated chemical moiety, polyethylene glycol (PEG), proven to elicit extended elimination half-life and reduced clearance.
AZ17 is composed of two scFvs linked together using our CAESAR platform and targets two upstream cytokines involved in the TH17 pathway. TH17 cells have been strongly associated with the development of inflammation and autoimmunity. These cells have a unique differentiation pathway, which provides specific points of intervention where their development and activation may be blocked.
We believe that AZ17’s novel mode of action will allow it to compete for this market space by offering the following competitive advantages:
• The independent inhibition of targeted cytokines is proven and safe
• Targeting multiple cytokines of the same pathway inherently increases responding patient population
• Low cost of goods due to expression in E. coli
• The nature of the linker affords customization of in vivo half-life to indication
Diabetes
Diabetes is one of the most challenging health problems of the 21st century. Current estimates for the US alone call for the doubling of the known patient population from today's 23 to 44 million over the next 25 years.
The standard care of therapy based on insulin injections has not changed meaningfully in the past 15 years. Although insulin has evolved from its bovine or porcine origin in the beginning to the new generations of recombinant human insulin and its analogs, it is thought that radical improvements in the treatment of diabetes will be achieved with next generation therapeutics targeting novel mechanisms of action.
Fibroblast Growth Factor 21 (FGF-21) belongs to the new class of therapeutics currently being explored for their ability to control blood glucose levels. Animal studies have shown that treatment with FGF21 lowers plasma glucose and increases insulin sensitivity. The beneficial effects of FGF21 are observed only after repeated dosing and prolonged exposure, making the FGF21 protein a challenging drug to develop and indicating an unmet medical need for a longer acting form.
ALLOZYNE has generated AZ21, a novel product candidate which is a long acting form of FGF21. AZ21 can overcome the need for multiple and frequent administration by its conjugation to a known and validated chemical moiety, polyethylene glycol (PEG), proven to elicit extended elimination half-life and reduced clearance.
