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BEL-0215

BEL-0215 (PEG-C1-INH) – Hereditary Angioedema.

Overview

Hereditary Angioedema (HAE) is a very rare and potentially life-threatening genetic condition that occurs in about 1 in 10,000 to 1 in 50,000 people. HAE symptoms include episodes of edema (swelling) in various body parts including the hands, feet, face and airway.

HAE patients have a defect in the gene that controls a blood protein called C1 Inhibitor. The genetic defect results in production of either inadequate or non-functioning C1-Inhibitor protein. Normal C1-Inhibitor helps to regulate the complex biochemical interactions of blood-based systems involved in disease fighting, inflammatory response and coagulation.

Although the past few years have seen the development of several new treatment options, remaining obstacles — high expense, short plasma half-lives (i.e., the amount of time therapeutically-active levels remain in the blood) of currently-approved therapeutics and the need for frequent IV infusions — still leave a significant unmet need for this indication.

BEL-0215 is a development candidate approaching the filing of an IND and Phase I clinical study for the maintenance treatment of HAE. Currently, approved treatments for maintenance HAE therapy require bi-weekly intravenous injections of a C1 Esterase Inhibitor. These IV injections of C1-INH help to prevent attacks and swelling in those suffering from HAE by providing the body with enough working C1 esterase inhibitor to help prevent the causes of swelling and is proven to reduce the frequency, duration, and severity of HAE attacks.

BEL-0215, a proprietary, long-acting form of C1 Esterase Inhibitor (C1-INH), offers a significant improvement over the currently approved C1 Esterase Inhibitor treatments, by eliminating the need for IV administration, reducing required dosing frequency, enhancing the side effect profile and improving patient compliance.

 

Mechanism of Action / Advantages

Advancing Development of Belrose’s PEG-C1 Esterase Inhibitor Candidate

Available data suggests that Belrose’s PEG-C1 Esterase Inhibitor candidate is suitable for further development:
  • The available drugs for both HAE prophylaxis and the treatment of acute HAE attacks are costly and require frequent administration
  • Given the need for the frequency of administration of the C1-INH, Belrose’s PEG-C1 Esterase Inhibitor candidate is a promising treatment option since data from animal models indicates that it reduces the required frequency of injection and may allow for a lower dose to achieve clinical efficacy
  • PEGylation appears to minimize the immunogenicity that may be problematic in other C1-Esterase inhibitor recombinant drugs as well as reducing the required frequency of dosing
  • Based on experiments with a range of potential PEGylation linking strategies, all compounds retained good functional activity based on homogeneous colorimetric assay, and it appears that the PEGylation had more impact on the kallikrein assay results when compared to the homogeneous functional assay data

 

Clinical Trial Overview

In May 2014, the FDA reviewed Belrose’s IND-enabling plan. BEL-0215 is being progressed through a final preclinical toxicology program and will then move into a Phase I human clinical trial, anticipated to be conducted in 2015.

Given the significant improvement to currently-approved maintenance HAE therapy, Belrose believes that it will be granted orphan drug designation by the FDA for the maintenance treatment of HAE, enabling an expedited regulatory approval pathway.