Helixmith

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Helixmith
FormerlyByromedica Pacific Co. LTD.
(1996–1999)
ViroMed
(1999–2019)
Company type Public
Industry Biotechnology
FoundedNovember 1996;28 years ago (1996-11)
FounderSunyoung KIM, D. Phil
Headquarters21, Magokjungang 8-ro 7-gil, Gangseo-gu,
Magok, Seoul
,
South Korea
Key people
Sunyoung Kim (CEO), Seungshin Yu (CTO), Sinyoung Kim (COO)
Number of employees
121 (2019)
Website helixmith.com

Helixmith Co. LTD. is a biotechnology company located in Seoul, South Korea with US presence in San Diego. The company has an extensive gene therapy pipeline, including a non-viral plasmid DNA program for neuromuscular and ischemic disease, a CAR-T program targeting several different types of solid tumors, and an AAV vector program targeting neuromuscular diseases. Helixmith’s lead gene is Engensis (VM202), currently in phase III diabetic peripheral neuropathy (DPN) in the US. Engensis (VM202) is a plasmid DNA designed to simultaneously express two isoforms of hepatocyte growth factor (HGF), HGF 728 and HGF 723. In addition to DPN, Engensis is also being studied in diabetic foot ulcers (DFU), amyotrophic lateral sclerosis (ALS), coronary artery disease (CAD), claudication, and Charcot-Marie-Tooth disease (CMT).

Contents

Helixmith headquarters located in Magok, Seoul Helixmith.jpg
Helixmith headquarters located in Magok, Seoul

History

Helixmith Co. LTD. (prev. ViroMed) was established in 1996 as the first on-campus startup at Seoul National University in 1996, and later renamed to ViroMed in 1999. The company has been listed on the Korean Securities Dealers Automated Quotations (KOSDAQ: 084990) since 2006. In April 2019, the company was renamed to Helixmith, and moved its headquarters from its previous research facility in Seoul National University to Magok, Seoul.

Overview

Helixmith’s main business area is in gene therapy development. Helixmith’s lead gene therapy product is Engensis (VM202), a non-viral plasmid DNA that encodes the therapeutic gene called hepatocyte growth factor (HGF). Engensis is being developed for diabetic peripheral neuropathy (DPN, phase 3) in the US. The product is also being studied for diabetic foot ulcers (DFU, phase 3), amyotrophic lateral sclerosis (ALS, phase 2), coronary artery disease (CAD, phase 2), claudication (phase 2) and Charcot-Marie-Tooth disease (CMT, phase 1/2a).

Helixmith’s pipeline extends to CAR-T cell therapy and AAV gene therapy. In CAR-T cell therapy, the company aims at eradicating various solid tumors. The CAR-T program is in pre-clinical stage through a separate subsidiary called Cartexell. In AAV gene therapy, the company has a number of early stage products targeting neuromuscular diseases such as ALS and multiple sclerosis. Helixmith also has an antibody pipeline including VM507, an antibody that can detect and activate c-MET, receptor of hepatocyte growth factor (HGF).

Helixmith is also developing phytotherapeutics based on natural plant extracts with therapeutic potential. The company has unique experience in areas including natural medicine, health functional food and cosmetic products using botanical sources.

Products

Engensis

Helixmith’s non-viral plasmid DNA product, Engensis, is designed to express recombinant HGF protein in nerve and Schwann cells to promote nerve system regeneration and induce the formation of microvascular blood vessels.

HGF has a short half-life (5 minutes or less) and is quickly removed from the body by the liver, creating an obstacle to effective treatment with previous injectable recombinant HGF protein products.

A single injection of Helixmith’s proprietary plasmid DNA product expresses the HGF gene at levels 30-40 times higher than conventional plasmid DNA and provides sustained gene expression in mouse models for 2 weeks, with peak protein expression at Day 7 and a gradual decrease over the next week To date, more than 500 patients have been treated with Engensis across ten clinical trials in six different diseases and conditions. Data from previous clinical studies suggest that Engensis is well tolerated and has the potential to provide durable analgesic and/or symptomatic relief in a variety of disease settings. Beyond potentially alleviating pain, Engensis is designed to target the underlying causes of neuropathy through its predicted angiogenic and neuroregenerative properties.

The US FDA recognized the potential for Engensis to meet the unmet need for this condition in 2018 by designating it as a Regenerative Medicine Advanced Therapy (RMAT), making it the first RMAT-designated gene therapy for a prevalent disease with over one million patients. This designation grants all the benefits afforded by the fast track and breakthrough designations, including priority review, to Engensis. Helixmith currently has multiple target indications under the Engensis pipeline: diabetic peripheral neuropathy (DPN, phase 3), diabetic foot ulcers (DFU, phase 3), amyotrophic lateral sclerosis (ALS, phase 2), coronary artery disease (CAD, phase 2), claudication (phase 2) and Charcot-Marie-Tooth disease (CMT, phase 1/2a).

The US FDA granted a RMAT (Regenerative Medicine Advanced Therapy) designation to VM202-DPN in 2018. This is the first RMAT designation for a drug product based in Korea, and the first and the only RMAT designation worldwide in pain area. Engensis has been attracting huge attention in painful DPN because of its big market size. The US FDA granted orphan drug and fast track designation for Engensis (VM202-ALS) in 2016. Engensis (VM202) is currently under development as a possible treatment for chronic DFU with the hope to potentially heal the ulcer by supplying sufficient blood through new blood vessel formation around occluded or narrowed blood vessels towards the lower extremities.

VM507

VM507, Helixmith’s leading antibody treatment, is an antibody that can detect and activate c-MET (HGF receptor). An antibody is an immune protein that binds to an antigen to inhibit its activity or stimulate neutralization or activation. Although it is a protein generated in the immune system originally, an antibody is available to be mass-produced, purified, and analyzed into monoclonal antibody, regarding specific antibodies with selectivity and specificity against specific antigens.

VM507 is an antibody that can detect and activate c-MET, receptor of hepatocyte growth factor (HGF). As a fully human antibody, it has the potential to be safe immunologically, transmissible via blood vessel injection or local injection to other various tissues and organs, and the longer half-life may contribute to improved efficacy.

The c-Met level is especially high in patients with chronic/acute renal disease. VM507 showed therapeutic efficacies such as inhibition of renal fibrosis and improvement of functional index by binding with c-MET receptor in the renal tissue when injected intravenously in the mouse model of renal disease.

Status of Clinical Trials

According to its website, the company is involved in the following clinical trials:

Phytotherapeutics

Helixmith currently has two target indications under its phytotherapeutics pipeline: PG201 (Osteoarthritis), and HX204 (Inflammatory bowel disease). PG201 is a prescription drug for osteoarthritis and is the 7th botanical drug that has ever been approved by the MFDS (Ministry of Food and Drug Safety) in 2012. It is being sold under the brand name “LAYLA Tab” and has been generating a domestic annual revenue of 20 billion KRW since it has been licensed out to PMG Pharma.

PG201 showed significant improvement in various animal models of osteoarthritis and rheumatoid arthritis. In addition, it has been founded that it can prevent cartilage destruction by regulating the expression of cartilage degradation enzymes unlike conventional anti-inflammatory analgesic drugs such as NSAIDs. PG201 has proved its safety and efficacy on patients with osteoarthritis by conducting phase II and phase III clinical trials.

HX204 is currently under pre-clinical development and is expected to enter clinical phase in 2022.[ citation needed ]

Related Research Articles

Diabetic neuropathy includes various types of nerve damage associated with diabetes mellitus. The most common form, diabetic peripheral neuropathy, affects 30% of all diabetic patients. Symptoms depend on the site of nerve damage and can include motor changes such as weakness; sensory symptoms such as numbness, tingling, or pain; or autonomic changes such as urinary symptoms. These changes are thought to result from a microvascular injury involving small blood vessels that supply nerves. Relatively common conditions which may be associated with diabetic neuropathy include distal symmetric polyneuropathy; third, fourth, or sixth cranial nerve palsy; mononeuropathy; mononeuropathy multiplex; diabetic amyotrophy; and autonomic neuropathy.

This is a list of AIDS-related topics, many of which were originally taken from the public domain U.S. Department of Health Glossary of HIV/AIDS-Related Terms, 4th Edition.

<span class="mw-page-title-main">Human Genome Sciences</span> Former American pharmaceutical company, acquired by GlaxoSmithKline.

Human Genome Sciences (HGS) was a biopharmaceutical corporation founded in 1992 by Craig Venter, Alan Walton and Wally Steinberg. It uses the human DNA sequence to develop protein and antibody drugs. It had drugs under development to treat such diseases as hepatitis C, systemic lupus erythmatosis, anthrax, and cancer. It collaborated with other biotechnology and pharmaceutical companies for development partnerships and licensing.

<span class="mw-page-title-main">Pentoxifylline</span> Chemical compound

Pentoxifylline, also known as oxpentifylline, is a xanthine derivative used as a drug to treat muscle pain in people with peripheral artery disease. It is generic and sold under many brand names worldwide.

<span class="mw-page-title-main">Hepatocyte growth factor receptor</span> Mammalian protein found in Homo sapiens

Hepatocyte growth factor receptor is a protein that in humans is encoded by the MET gene. The protein possesses tyrosine kinase activity. The primary single chain precursor protein is post-translationally cleaved to produce the alpha and beta subunits, which are disulfide linked to form the mature receptor.

<span class="mw-page-title-main">Arimoclomol</span> Medication

Arimoclomol, sold under the brand name Miplyffa, is a medication for the treatment of Niemann–Pick disease type C. It is taken by mouth.

A helper dependent virus, also termed a gutless virus, is a synthetic viral vector dependent on the assistance of a helper virus in order to replicate, and can be used for purposes such as gene therapy. Naturally-occurring satellite viruses are also helper virus dependent, and can sometimes be modified to become viral vectors.

<span class="mw-page-title-main">Hepatocyte growth factor</span> Mammalian protein found in Homo sapiens

Hepatocyte growth factor (HGF) or scatter factor (SF) is a paracrine cellular growth, motility and morphogenic factor. It is secreted by mesenchymal cells and targets and acts primarily upon epithelial cells and endothelial cells, but also acts on haemopoietic progenitor cells and T cells. It has been shown to have a major role in embryonic organ development, specifically in myogenesis, in adult organ regeneration, and in wound healing.

Ocrelizumab, sold under the brand name Ocrevus, is a medication used for the treatment of multiple sclerosis. It is a humanized anti-CD20 monoclonal antibody. It targets CD20 marker on B lymphocytes and is an immunosuppressive drug. Ocrelizumab binds to an epitope that overlaps with the epitope to which rituximab binds. It is administered by intravenous infusion. The fixed-dose combination ocrelizumab/hyaluronidase is administered by subcutaneous injection.

<span class="mw-page-title-main">Glutamate carboxypeptidase II</span> Enzyme

TAH molecule, also known as N-acetyl-L-aspartyl-L-glutamate peptidase I, NAAG peptidase, or prostate-specific membrane antigen (PSMA) is an enzyme that in humans is encoded by the FOLH1 gene. Human GCPII contains 750 amino acids and weighs approximately 84 kDa.

<span class="mw-page-title-main">Eva Feldman</span> American neurologist

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<span class="mw-page-title-main">Diabetic foot</span> Medical condition

A diabetic foot disease is any condition that results directly from peripheral artery disease (PAD) or sensory neuropathy affecting the feet of people living with diabetes. Diabetic foot conditions can be acute or chronic complications of diabetes. Presence of several characteristic diabetic foot pathologies such as infection, diabetic foot ulcer and neuropathic osteoarthropathy is called diabetic foot syndrome. The resulting bone deformity is known as Charcot foot.

c-Met inhibitors are a class of small molecules that inhibit the enzymatic activity of the c-Met tyrosine kinase, the receptor of hepatocyte growth factor/scatter factor (HGF/SF). These inhibitors may have therapeutic application in the treatment of various types of cancers.

Human HGF plasmid DNA therapy of cardiomyocytes is being examined as a potential treatment for coronary artery disease, as well as treatment for the damage that occurs to the heart after MI. After MI, the myocardium suffers from reperfusion injury which leads to death of cardiomyocytes and detrimental remodelling of the heart, consequently reducing proper cardiac function. Transfection of cardiac myocytes with human HGF reduces ischemic reperfusion injury after MI. The benefits of HGF therapy include preventing improper remodelling of the heart and ameliorating heart dysfunction post-MI.

Ozanezumab is a monoclonal antibody designed for the treatment of ALS and multiple sclerosis.

<span class="mw-page-title-main">Diabetic foot ulcer</span> Medical condition

Diabetic foot ulcer is a breakdown of the skin and sometimes deeper tissues of the foot that leads to sore formation. It is thought to occur due to abnormal pressure or mechanical stress chronically applied to the foot, usually with concomitant predisposing conditions such as peripheral sensory neuropathy, peripheral motor neuropathy, autonomic neuropathy or peripheral arterial disease. It is a major complication of diabetes mellitus, and it is a type of diabetic foot disease. Secondary complications to the ulcer, such as infection of the skin or subcutaneous tissue, bone infection, gangrene or sepsis are possible, often leading to amputation.

Total contact casting (TCC) is a specially designed cast designed to take weight off of the foot (off-loading) in patients with diabetic foot ulcers (DFUs). Reducing pressure on the wound by taking weight off the foot has proven to be very effective in DFU treatment. DFUs are a major factor leading to lower leg amputations among the diabetic population in the US with 85% of amputations in diabetics being preceded by a DFU. Furthermore, the five-year post-amputation mortality rate among diabetics is estimated at 45% for those with neuropathic DFUs.

Lawrence Steinman is an American neurologist, neuroimmunologist and an academic. He is a Professor of Neurology and Neurological Sciences and Pediatrics at Stanford University.

Regenerative Medicine Advanced Therapy (RMAT) is a designation given by the Food and Drug Administration to drug candidates intended to treat serious or life-threatening conditions under the 21st Century Cures Act. A RMAT designation allows for accelerated approval based surrogate or intermediate endpoints.

References