Phase 2 clinical trial data of argenx’s (ARGX) product Efgartigimod (ARGX-113) in adult patients with primary immune thrombocytopenia (ITP) showed a favorable safety and tolerability profile. The results also demonstrated that efgartigimod caused meaningful platelet count improvements across doses and ITP patient classifications, including newly diagnosed, persistent and chronic, and correlated with a consistent reduction in IgG levels.
Commenting on the news, Nicolas Leupin, Chief Medical Officer of argenx said that addressing ITP in a novel way across patient types was possible because Efgartigimod targets the source of the disease as it acts by eliminating IgGs and restoring platelet numbers. He attributed the durable product’s tolerance to the unique binding characteristics of the Fc fragment, viewing it as an important advantage in this new therapeutic class.
For detailed trials and results read the firm’s press release by clicking on the following link: http://www.argenx.com/en-GB/content/press-releases/37/
The primary endpoint of the Phase 2 trial
Safety and tolerability: Efgartigimod was well-tolerated in all patients. The most adverse events (AEs) are characterized as mild and one serious adverse event was reported in the primary study but was deemed unrelated to the study drug.
Post-hoc analyses around secondary endpoint measures demonstrated that efgartigimod treatment was associated with clinically meaningful improvements in platelet count’s magnitude of effect, onset of action and durability.
What’s More
A Subcutaneous Formulation: Argenx expects to initiate a Phase 2 trial in ITP using a subcutaneous formulation of Efgartigimod.
Other Diseases: Efgartigimod is currently being evaluated in a global Phase 3 registration trial in gMG and a Phase 2 proof-of-concept trial in pemphigus vulgaris (PV).
Future Plans: Argenx announced plans to initiate a Phase 2 proof-of-concept trial of efgartigimod (IV) in chronic inflammatory demyelinating polyneuropathy (CIDP) in the first half of 2019.
The Diseases
Primary Immune Thrombocytopenia (ITP)
In Adult patients with Primary Immune Thrombocytopenia (ITP) pathogenic IgGs destroy platelets’ producing cells in the bone marrow and blood platelets. Pathogenic IgGs drive disease progression in a multimodal approach: they accelerate platelet clearance, inhibit platelet production, directly induce platelet killing and interfere with platelet’s ability to perform their clotting function.
Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
CIDP is a chronic, progressive autoimmune disorder: It affects peripheral nerves and nerve roots. The disease is driven by an autoimmune-mediated destruction of the myelin sheath or myelin producing cells, which serve to insulate the axon of the nerves and enable speed of signal transduction. Demyelination and axonal damage in CIDP lead to loss of sensory and/or motor neuron function, which can cause weakness, sensory loss, imbalance and/or pain. CIDP is among the largest indications for IV/SC Ig in the United States.
The Drug
Efgartigimod (ARGX-113) is an investigational therapy for IgG-mediated autoimmune diseases and was designed to exploit the natural interaction between IgG antibodies and the recycling receptor FcRn.
Efgartigimod is the Fc-portion of an IgG1 antibody that has been modified by argenx proprietary ABDEG™ technology to increase its affinity for FcRn beyond that of normal IgG antibodies. As a result, efgartigimod blocks antibody recycling through FcRn binding and leads to fast depletion of the autoimmune disease-causing IgG autoantibodies. The development work on efgartigimod is conducted in close collaboration with Prof. E. Sally Ward (University of Texas Southwestern Medical and Texas A&M University Health Science Center, a part of Texas A&M University (TAMHSC)).
The Company
Argenx is a clinical-stage biotechnology company developing differentiated antibody-based therapies for the treatment of severe auto-immune diseases and cancer. The company is focused on developing first-in-class candidates aiming at novel targets or best-in-class candidate drugs against known, but complex, targets in order to treat diseases with a significant unmet medical need.
Prohost Observations
Argenx’s ability to execute its programs is enabled by its following technologies:
The SIMPLE ™ Platform: Based on the llama immune system, which enables exploiting novel and complex targets.
The three complementary Fc engineering technologies: These are designed to expand the therapeutic index of its product candidates.
There is no doubt that argenx has demonstrated solid scientific fundamentals and capability of creating novel products and developing them. The above is not the only news that boosted the value of this firm. The fruits from the efforts argenx has spent since its establishment are beginning to surface now recently.
Recent Good News:
– The announcement in August 29, 2018 that argenx has received feedback from the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan on the design of a Phase 3 trial and regulatory pathway towards potential marketing authorization of efgartigimod (ARGX-113) in patients with generalized myasthenia gravis (gMG).
In case positive data comes from the planned global Phase 3 registration trial, the trial results will serve as the basis FOR argenx to submit for approval and marketing in Japan.
– The announcement in August 22, 2018 of the exercise by AbbVie (ABBV) of its exclusive license option to develop and commercialize ARGX-115, an antibody targeting the novel immunotherapy glycoprotein A repetitions predominant (GARP).
Argenx and AbbVie entered into an option and license agreement for ARGX-115 in April 2016.
With the option exercise announced, AbbVie obtains a worldwide, exclusive license to develop and commercialize ARGX-115-based products. Argenx is now eligible to potentially receive development, regulatory and commercial milestone payments of up to $625 million, as well as tiered royalties on ARGX-115-based product sales, if approved. Argenx also has the right to co-promote ARGX-115-based products in the EU and Swiss Economic Area.
ARGX-115 is created through SIMPLE Antibody™ technology and binds specifically to the protein glycoprotein A repetitions predominant (GARP) that plays a key role in the regulation of production and release of active transforming growth factor beta (TGF-β).
Regulatory T-cell (TREGS)
ARGX-115 is believed to selectively limit the immunosuppressive activity of activated regulatory T-cells (Tregs), thereby stimulating the immune system to attack cancer cells.
We expect to here a lot from now on about Tregs, which prevent the immune system from recognizing and suppressing pathogenic cells including cancer cells. Argenx believes the selective inhibition of TGF-β release by Tregs is potentially superior to systemic inhibition of TGF-β activity or depletion of Tregs.
The Stock
The stock is trading today at around $94.65 with a market cap of $3.01 billion. In pre-market hours, the stock staged an intense rally that faded following the conference call. Regardless of the reason that investors decided to take profit as quickly, our eyes will remain on this firm.
Today’s abrupt selloff following the morning stock rally might have convinced us to buy ARGX at a bargain price which might still occur in the near-future. A selloff could happen following an announcement of good news, which has become a pattern followed blindly by professional traders.
News & Comments
September 17, 2018
Argenx’s product, Efgartigimod, demonstrates positive results in primary immune thrombocytopenia
Phase 2 clinical trial data of argenx’s (ARGX) product Efgartigimod (ARGX-113) in adult patients with primary immune thrombocytopenia (ITP) showed a favorable safety and tolerability profile. The results also demonstrated that efgartigimod caused meaningful platelet count improvements across doses and ITP patient classifications, including newly diagnosed, persistent and chronic, and correlated with a consistent reduction in IgG levels.
Commenting on the news, Nicolas Leupin, Chief Medical Officer of argenx said that addressing ITP in a novel way across patient types was possible because Efgartigimod targets the source of the disease as it acts by eliminating IgGs and restoring platelet numbers. He attributed the durable product’s tolerance to the unique binding characteristics of the Fc fragment, viewing it as an important advantage in this new therapeutic class.
For detailed trials and results read the firm’s press release by clicking on the following link: http://www.argenx.com/en-GB/content/press-releases/37/
The primary endpoint of the Phase 2 trial
Safety and tolerability: Efgartigimod was well-tolerated in all patients. The most adverse events (AEs) are characterized as mild and one serious adverse event was reported in the primary study but was deemed unrelated to the study drug.
Post-hoc analyses around secondary endpoint measures demonstrated that efgartigimod treatment was associated with clinically meaningful improvements in platelet count’s magnitude of effect, onset of action and durability.
What’s More
A Subcutaneous Formulation: Argenx expects to initiate a Phase 2 trial in ITP using a subcutaneous formulation of Efgartigimod.
Other Diseases: Efgartigimod is currently being evaluated in a global Phase 3 registration trial in gMG and a Phase 2 proof-of-concept trial in pemphigus vulgaris (PV).
Future Plans: Argenx announced plans to initiate a Phase 2 proof-of-concept trial of efgartigimod (IV) in chronic inflammatory demyelinating polyneuropathy (CIDP) in the first half of 2019.
The Diseases
Primary Immune Thrombocytopenia (ITP)
In Adult patients with Primary Immune Thrombocytopenia (ITP) pathogenic IgGs destroy platelets’ producing cells in the bone marrow and blood platelets. Pathogenic IgGs drive disease progression in a multimodal approach: they accelerate platelet clearance, inhibit platelet production, directly induce platelet killing and interfere with platelet’s ability to perform their clotting function.
Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
CIDP is a chronic, progressive autoimmune disorder: It affects peripheral nerves and nerve roots. The disease is driven by an autoimmune-mediated destruction of the myelin sheath or myelin producing cells, which serve to insulate the axon of the nerves and enable speed of signal transduction. Demyelination and axonal damage in CIDP lead to loss of sensory and/or motor neuron function, which can cause weakness, sensory loss, imbalance and/or pain. CIDP is among the largest indications for IV/SC Ig in the United States.
The Drug
Efgartigimod (ARGX-113) is an investigational therapy for IgG-mediated autoimmune diseases and was designed to exploit the natural interaction between IgG antibodies and the recycling receptor FcRn.
Efgartigimod is the Fc-portion of an IgG1 antibody that has been modified by argenx proprietary ABDEG™ technology to increase its affinity for FcRn beyond that of normal IgG antibodies. As a result, efgartigimod blocks antibody recycling through FcRn binding and leads to fast depletion of the autoimmune disease-causing IgG autoantibodies. The development work on efgartigimod is conducted in close collaboration with Prof. E. Sally Ward (University of Texas Southwestern Medical and Texas A&M University Health Science Center, a part of Texas A&M University (TAMHSC)).
The Company
Argenx is a clinical-stage biotechnology company developing differentiated antibody-based therapies for the treatment of severe auto-immune diseases and cancer. The company is focused on developing first-in-class candidates aiming at novel targets or best-in-class candidate drugs against known, but complex, targets in order to treat diseases with a significant unmet medical need.
Prohost Observations
Argenx’s ability to execute its programs is enabled by its following technologies:
The SIMPLE ™ Platform: Based on the llama immune system, which enables exploiting novel and complex targets.
The three complementary Fc engineering technologies: These are designed to expand the therapeutic index of its product candidates.
There is no doubt that argenx has demonstrated solid scientific fundamentals and capability of creating novel products and developing them. The above is not the only news that boosted the value of this firm. The fruits from the efforts argenx has spent since its establishment are beginning to surface now recently.
Recent Good News:
– The announcement in August 29, 2018 that argenx has received feedback from the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan on the design of a Phase 3 trial and regulatory pathway towards potential marketing authorization of efgartigimod (ARGX-113) in patients with generalized myasthenia gravis (gMG).
In case positive data comes from the planned global Phase 3 registration trial, the trial results will serve as the basis FOR argenx to submit for approval and marketing in Japan.
– The announcement in August 22, 2018 of the exercise by AbbVie (ABBV) of its exclusive license option to develop and commercialize ARGX-115, an antibody targeting the novel immunotherapy glycoprotein A repetitions predominant (GARP).
Argenx and AbbVie entered into an option and license agreement for ARGX-115 in April 2016.
With the option exercise announced, AbbVie obtains a worldwide, exclusive license to develop and commercialize ARGX-115-based products. Argenx is now eligible to potentially receive development, regulatory and commercial milestone payments of up to $625 million, as well as tiered royalties on ARGX-115-based product sales, if approved. Argenx also has the right to co-promote ARGX-115-based products in the EU and Swiss Economic Area.
ARGX-115 is created through SIMPLE Antibody™ technology and binds specifically to the protein glycoprotein A repetitions predominant (GARP) that plays a key role in the regulation of production and release of active transforming growth factor beta (TGF-β).
Regulatory T-cell (TREGS)
ARGX-115 is believed to selectively limit the immunosuppressive activity of activated regulatory T-cells (Tregs), thereby stimulating the immune system to attack cancer cells.
We expect to here a lot from now on about Tregs, which prevent the immune system from recognizing and suppressing pathogenic cells including cancer cells. Argenx believes the selective inhibition of TGF-β release by Tregs is potentially superior to systemic inhibition of TGF-β activity or depletion of Tregs.
The Stock
The stock is trading today at around $94.65 with a market cap of $3.01 billion. In pre-market hours, the stock staged an intense rally that faded following the conference call. Regardless of the reason that investors decided to take profit as quickly, our eyes will remain on this firm.
Today’s abrupt selloff following the morning stock rally might have convinced us to buy ARGX at a bargain price which might still occur in the near-future. A selloff could happen following an announcement of good news, which has become a pattern followed blindly by professional traders.
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