Outlining their strategy of teaming up with top academic labs to identify novel antibody targets,
argenx (ARGX) noted that they are focused on treating orphan disease conditions with high
unmet need. Lead product, efgartigimod, targets the neonatal Fc receptor (FcRn) and is being
developed in several autoimmune disorders. The company is hailing this candidate as a first-inclass, best-in-class, and pipeline-in-a-product opportunity. Differentiating the drug using data on
the safety and tolerability profile, with no headache or gastrointestinal toxicity, and fast onset of
clinical benefit, the small size of this natural ligand of FcRn enables convenient subcutaneous
dosing, a formulation being considered for development in immune thrombocytopenia and
chronic inflammatory demyelinating polyneuropathy.
A Phase III trial evaluating efgartigimod was recently initiated what they noted as being the
largest myasthenia gravis trial and one which uniquely includes anti-acetylcholine receptor
antibody negative patients, whose disease is driven primarily by MuSK and LRP4 autoantibodies.
Favorable outcomes on secondary endpoints for this sub-set of patients may provide a
competitive edge for the product’s label over drugs approved exclusively for anti-acetylcholine
receptor antibody positive patients. A Phase III trial with the intravenous formulations is planned
for immune thrombocytopenia in the second half of 2019 while an ongoing Phase II trial
evaluating efgartigimod in pemphigus vulgaris has been modified so that the third cohort, to start
enrollment in the first half of 2019, can evaluate clinical remission.
The company recently entered into an alliance with Janssen for cusatuzumab, an anti-CD70
product being evaluated for the treatment of acute myeloid leukemia. As part of the deal, argenx
received a total of $500 million upfront, which includes a $200 million equity investment, and
retains the right to co-promote cusatuzumab in the US and share economics 50-50 on a royalty
basis, with no cap on the commercial upside. Strategically, this would allow argenx to establish an
acute myeloid leukemia sales force that could also work to promote efgartigimod in immune
thrombocytopenia.
CEO Tim Van Hauwermeiren made a point during the presentation to call out the primary
academic researchers critical to the development of their products. Likewise, he mentioned that
they are working with a leading global expert in complement on ARGX-117, to be discussed
further at the company’s R&D day in May. Out-licensed products include IL-22 receptor targeting
ARGX-122, from which argenx receives milestones from LEO Pharma, and which will be
developed for atopic dermatitis; GARP-targeting ARGX-115 moving forward with AbbVie; and a
partnership with Staten Bio to develop an anti-APOC3, ARGX-116, for dyslipidemia. Cash at year
end 2018 was estimated at 582.3m Euros (+$500m from the Janssen deal).
argenx (ARGX) noted that they are focused on treating orphan disease conditions with high
unmet need. Lead product, efgartigimod, targets the neonatal Fc receptor (FcRn) and is being
developed in several autoimmune disorders. The company is hailing this candidate as a first-inclass, best-in-class, and pipeline-in-a-product opportunity. Differentiating the drug using data on
the safety and tolerability profile, with no headache or gastrointestinal toxicity, and fast onset of
clinical benefit, the small size of this natural ligand of FcRn enables convenient subcutaneous
dosing, a formulation being considered for development in immune thrombocytopenia and
chronic inflammatory demyelinating polyneuropathy.
A Phase III trial evaluating efgartigimod was recently initiated what they noted as being the
largest myasthenia gravis trial and one which uniquely includes anti-acetylcholine receptor
antibody negative patients, whose disease is driven primarily by MuSK and LRP4 autoantibodies.
Favorable outcomes on secondary endpoints for this sub-set of patients may provide a
competitive edge for the product’s label over drugs approved exclusively for anti-acetylcholine
receptor antibody positive patients. A Phase III trial with the intravenous formulations is planned
for immune thrombocytopenia in the second half of 2019 while an ongoing Phase II trial
evaluating efgartigimod in pemphigus vulgaris has been modified so that the third cohort, to start
enrollment in the first half of 2019, can evaluate clinical remission.
The company recently entered into an alliance with Janssen for cusatuzumab, an anti-CD70
product being evaluated for the treatment of acute myeloid leukemia. As part of the deal, argenx
received a total of $500 million upfront, which includes a $200 million equity investment, and
retains the right to co-promote cusatuzumab in the US and share economics 50-50 on a royalty
basis, with no cap on the commercial upside. Strategically, this would allow argenx to establish an
acute myeloid leukemia sales force that could also work to promote efgartigimod in immune
thrombocytopenia.
CEO Tim Van Hauwermeiren made a point during the presentation to call out the primary
academic researchers critical to the development of their products. Likewise, he mentioned that
they are working with a leading global expert in complement on ARGX-117, to be discussed
further at the company’s R&D day in May. Out-licensed products include IL-22 receptor targeting
ARGX-122, from which argenx receives milestones from LEO Pharma, and which will be
developed for atopic dermatitis; GARP-targeting ARGX-115 moving forward with AbbVie; and a
partnership with Staten Bio to develop an anti-APOC3, ARGX-116, for dyslipidemia. Cash at year
end 2018 was estimated at 582.3m Euros (+$500m from the Janssen deal).
