바이오스펙테이터

by Jongwon Jang

Avixgen actualizes its move of new drug development by securing 4 clinical pipelines, including new drugs for senile macular degeneration, xerophthalmia, and atopic dermatitis as well as for AIDS that entered into Phase 2 Clinical Trial in 2018. The company has been consolidating basic competence based on platform technology.

'AVI-CO-004' Phase 2 Clinical Trial for AIDS is in progress. It has two concurrent clinical trials in Vietnam and a domestic hospital (the Phase 2a Clinical Trials). The Phase 1 Clinical Trial for therapeutic agent of ‘AVI-3207’ known to inhibit VEGF-2 selectively for curing senile macular degeneration was approved in Australia last June following domestic trial. Its approval was acquired in August last year.

Therapeutic agent 'AVI-4015' for xerophthalmia was developed through drug repositioning process of oral anticancer therapeutic agent. The therapeutic agent for atopic dermatitis, 'AVI-3307', was approved with domestic Phase 1 Clinical Trial in last July.

AVI-4015 exhibits effect of improving inflammation on surface of eye and prevention of damage to cornea. AVI-3307 normalizes the damaged vascular permeability, thereby preventing the leakage of tissues of inflammatory factors to soothe symptoms that differentiates its mechanism from others.

Yu Ji-chang, the President of Avixgen, stated, “… technology transfer will be actively promoted to attain tangible results from next year upon confirmation of safety and efficacy of pipelines currently entered into clinical trials…”

◇ Clinical Trial of New Drug for AIDS targeting Virus NC Protein will be Expanded in U.S.A.

The leading pipeline of Avixgen is ‘AVI-CO-004’, the therapeutic agent for AIDS. It is currently in Phase 2a Clinical Trial.

AVI-CO-004 acts on NC (Nucleocapsid) protein selectively which acts on the stage of packaging of genetic information by virus particle, thereby inhibiting the combination with RNA gene and consequently incapacitating the infectiousness of HIV virus.

Avixgen was granted Phase 2a Clinical Trial domestically. In Vietnam, clinical trials are in progress currently in four medical institutions including Severance Hospital etc. A total of 36 male patients infected with HIV virus are subjected to repetitive administration of AVI-CO-004 for 10 days to assess safety, tolerance, and pharmacodynamic characteristics etc.

The company explained, “… since it was appointed as an orphan drug remaining in the stage of development, making inroads into domestic market is estimated to be enabled upon conditional approval after confirmation of results obtained from Phase 2a Clinical Trial …”, “… the drug has a mechanism different from existing medicine, rendering higher level of therapeutic efficacy as well as strong antivirus effect in combination with existing therapeutics, demonstrating sufficient competitiveness in market…”.

President Yu Ji-chang stated, “… we intend to conduct Phase 2 Clinical Trial in the United States next year. Phase 2 clinical trials are currently in progress domestically and in Vietnam, for which we are preparing for a Pre-IND meeting with FDA…”.

◇ Therapeutic Agents of VEGFR Targeted Macular Degeneration and Atopic Dermatitis Entered into Phase 1 Clinical Trial

Avixgen developed ‘ACP (Advanced Cell Penetrating-Peptide Technology)’, a cytopermeable peptide technology, by exploiting result of research on therapeutic agent for AIDS. Avixgen also established a platform technology. Cell penetrating peptide comprises 5~30 amino acid. It is a new drug delivery system of different concepts, enabling delivery of diverse cargoes into inside of cells such as DNA, RNA, protein, and low molecular compound etc.

President Yu Ji-chang stated, “… the development of medicine convenient for patients such as in forms of paste or instillation from conventional injection would be enabled as well as increased delivery of peptides enabled through ACP technology …’. He also added, “… beyond the candidate of new drug that we have excavated spontaneously, we also expect amelioration by applying ACP technology to materials of which efficacies are yet to be identified due to issues related to delivery …”.

Based on such platform technology, Avixgen currently develops candidate material of low molecular peptide targeting vascular epithelial growth factor receptor (VEGFR) as therapeutic agent for senile macular degeneration and atopic dermatitis.

President Yu Ji-chang elucidated, “… the development of pipeline for therapeutic agent aiming for intractable rare disease is in progress …”. The representative one is the therapeutic agent targeting severe immunodeficiency disease due to deficiency of adenosine deaminase (ADA) which is in charge of important functioning for the growth and function of lymphocyte.

Breakdown of enzyme due to mutation arising in the gene of corresponding enzyme causes death in infancy by serious disorder of immunity due to deficiency in humoral immunity and cell immunity ascribable to dysgenesis of stem cell. Avixgen develops an enzyme-replacement therapeutic agent capable of delivering corresponding enzyme to ACP.

President Yu Ji-chang explained, “… the application of gene therapeutic agent requires complex procedures comprising in vitro injection of normal enzyme to T-cell, culture thereof, and administration of the cultured enzyme to patient. However, our ACP technology enables direct attachment of enzyme, allowing intracellular functioning after administration to patient…”.

◇ Therapeutic agent for xerophthalmia was developed through Drug Repositioning Process … “Improvement in Inflammation and Protection of Corneal were realized by Inhibiting DDR1 Receptor”

The unique history of ‘AVI-4015’, the therapeutic agent for xerophthalmia developed by Avixgen, has attracted attention. The oral substance of anticancer agent which has been granted as a therapeutic agent for leukemia is being redeveloped as therapeutic agent for xerophthalmia. AVI-4015 is a candidate material with a mechanism that targets and inhibits DDR (Discoidin Domain Receptor)1 to improve inflammatory reaction simultaneously while protecting epithelial cells of corneal.

Avixgen was granted Phase 1 Clinical Trial which will be initiated within this year. The safety of AVI-4015 in the form of instillation will be appraised from healthy adults upon completion of studies on mechanism and efficacy test according to the redevelopment of drug.

President Yu Ji-chang stated, “… we are discussing collaborative research and development with several domestic and overseas pharmaceutical companies to exploit platform technology and technology transfer. We expect that we will be able to see tangible outcomes therefrom soon…”. Avixgen also promotes the IPO in KOSDAQ based on these accomplishments.