Future Looks Bright For Ambitious Argenx
Executive Summary
Now that cash-rich Argenx has AbbVie fully onboard and a pipeline-in-a-product opportunity in efgartigimod moving smoothly through the clinic, the Belgian biotech is in for an interesting couple of years.
With AbbVie Inc. licensing a preclinical immuno-oncology drug, and strong data backing its lead candidate efgartigimod, argenx SE of Belgium's antibody technology platform has been validated.
The AbbVie deal, announced Aug. 22, saw the US major has exercised an option it took out in April 2016 to develop and commercialize ARGX-115, an antibody targeting glycoprotein A repetitions predominant (GARP) which plays a key role in the regulation of production and release of active transforming growth factor beta.
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. The premise is that ARCX-115 could prove useful as monotherapy or in combination with anti-PD1/L1 and/or anti-CTLA4 agents, or with cancer vaccines, to improve the activity and safety of those immunotherapies.
An argenx spokesperson told Scrip that in the two years since AbbVie took out the option, a lot of preclinical work had been done and the partners hoped to enrol the first patient in clinical trials next year. The company banked $40m when the deal was announced and received $20m more. It is eligible to receive milestone payments of up to $625m, as well as tiered royalties, if all goes well in the clinic.
Argenx, which has bases in Ghent, Belgium and Breda in the Netherlands, has also secured co-promotion rights for ARGX-115-based products in the EU and Switzerland. AbbVie seems convinced of the candidate's potential and Tom Hudson, its head of oncology early discovery and development, noted in a statement that "our collaboration with argenx over the past two years has been productive and we look forward to continue working together to fuel scientific progress."
CEO Tim Van Hauwermeiren said the deal highlighted the important of argenx's Innovative Access Program (IAP) as ARGX-115 is the result of research initially carried out by the de Duve Institute/ Université Catholique de Louvain in Belgium. He added that the IAP "remains a strategic priority for us [and] we continue to seek out cutting-edge research and targets while advancing our current collaborations, all with the potential to broaden our pipeline and demonstrate our discipline as a strategic partner."
Two additional IAP programs have started to bear fruit. One is ARGX-116, discovered in collaboration with disease biology experts from Staten Biotechnology, which specializes in the field of dyslipidemia and the other is a pact with Broteio Pharma of the Netherlands to develop therapies for severe autoimmune diseases.
The deal went down well with investors, as argenx shares went up 4.7%, while analysts at JMP Securities issued a note saying that the AbbVie collaboration "further supports our view that the company’s technologies are differentiated and important in the development of novel biologics to address unmet medical needs."
While getting AbbVie fully onboard is a validation of argenx' Simple Antibody technology, which employs llama immunization, the more immediate focus is on efgartigimod. The latter is a first-in-class antibody fragment designed for the treatment of patients with severe autoimmune diseases and the company is developing the drug in three indications.
Primary results from a Phase II trial for efgartigimod in idiopathic thrombocytic purpura are expected next month, with a more detailed set of data set to be presented at the American Society of Hematology meeting in San Diego in December. The company already has positive Phase II results from the drug in another indication, myasthenia gravis, and a Phase III trial is being prepared with 150 patients expected to be enrolled; it is also being tested in mid-stage studies for pemphigus vulgaris.
The argenx spokesperson told Scrip that the firm was looking to take efgartigimod into Phase III on its own and sees it as a pipeline-in-a-product opportunity. Argenx is focusing on the drug in rare diseases but the potential in bigger indications such as lupus and multiple sclerosis could make for partnering possibilities.
As for the rest of the pipeline, ARGX-110 is in a Phase II study for cutaneous T-cell lymphoma and in a Phase I/II trial in acute myeloid leukemia and myelodysplastic syndrome. Argenx also has a collaboration with Leo Pharma AS signed in May 2015 for ARGX-112 for inflammatory skin disorders.
In addition to an upfront and other payments, argenx could pocket up to approximately €100m as well as royalties on net sales of any product from the deal with the Danish company – a third preclinical milestone was banked in April this year following the approval of the clinical trial application (CTA) filing for ARGX-112. It also received a payment from Shire PLC triggered by the latter exercising its option to in-license an antibody. No details about the indication specifically being pursued have been disclosed but argenx rather vaguely notes on its website that it might be a therapy to treat a rare disease such as Hunter syndrome or Fabry disease, or for a specialist condition such as ADHD or ulcerative colitis.
Cashwise, argenx is in good shape after raising approximately $100m through a Nasdaq listing in May 2017 – its shares are also traded on Euronext Brussels. As of June 30, cash and equivalents amounted to €338.9m and the spokesperson noted that the firm has enough money to fund operations for at least the next couple of years.