Together We Discover
Reaching Patients Through
Immunology Innovation
"GO" Decision: ADHERE Trial in CIDP
FEBRUARY 2021
1
Forward Looking Statements
This presentation has been prepared by argenx se ("argenx" or the "company") for informational purposes only and not for any other purpose. Nothing contained in this presentation is, or should | |
be construed as, a recommendation, promise or representation by the presenter or the company or any director, employee, agent, or adviser of the company. This presentation does not purport to | ! |
be all-inclusive or to contain all of the information you may desire. This presentation also contains estimates and other statistical data made by independent parties and by us relating to market size | |
and growth and other data about our industry. This data involves a number of assumptions and limitations, and you are cautioned not to give undue weight to suchestimates. |
Safe Harbor: Certain statements contained in this presentation, other than present and historical facts and conditions independently verifiable at the date hereof, may constitute forward-looking statements. Examples of such forward-looking state- ments include those regarding our business and financial outlook and related plans; the therapeutic and commercial potential of our product candidates; the intended results of our strategy; the expected benefits of our collaborations, including with respect to our collaboration with Zai Lab; our and our collaboration partners' clinical development and regulatory plans, including the timing, design and outcome of ongoing and planned clinical trials and preclinical activities and the timing and outcome of regulatory filings and approvals; the timing, progress and benefits of marketing and commercialization activities; and the expected size of the markets for our product candidates. When used in this presenta- tion, the words "anticipate," "believe," "can," "could," "estimate," "expect," "intend,"
"is designed to," "may," "might," "will," "plan," "potential," "predict," "objective," "should," or the negative
of these and similar expressions identify forward-lookingstatements.
Such statements, based as they are on the current analysis and expectations of ma- nagement, inherently involve numerous risks and uncertainties, known and unknown, many of which are beyond the Company's control. Such risks include, but are not limited to: the impact of COVID-19 pandemic on our business, the impact of general economic conditions, general conditions in the biopharmaceutical industries, changes in the global and regional regulatory environments in the jurisdictions in which the Company does or plans to do business, market volatility, fluctuations in costsand
changes to the competitive environment. Consequently, actual future results may differ materially from the anticipated results expressed in the forward-looking statements. In the case of forward-looking statements regarding investigational product candidates and continuing further development efforts, specific risks which could cause actual results to differ materially from the Company's current analysis and expectations include: failure to demonstrate the safety, tolerability and efficacy of our product candidates; final and quality controlled verification of data and the related analyses; the expense and uncertainty of obtaining regulatory approval, including from the U.S. Food and Drug Administration and European Medicines Agency; the possibility of having to conduct additional clinical trials; our ability to obtain and maintain intellectual property protection for our product candidates; and our reliance on third parties such as our licensors and collaboration partners regarding our suite of technologies and product candidates. Further, even if regulatory approval is obtained, biopharmaceutical products are generally subject to stringent on-going govern- mental regulation, challenges in gaining market acceptance and competition. These statements are also subject to a number of material risks and uncertainties that are described in the Company's filings with the U.S. Securities and Exchange Commission ("SEC"), including in argenx's most recent annual report on Form 20-F filed with the SEC as well as subsequent filings and reports filed by argenx with the SEC. The reader should not place undue reliance on any forward-looking statements included in this presentation. These statements speak only as of the date made and the Company is under no obligation and disavows any obligation to update or revise such statements as a result of any event, circumstances or otherwise, unless required by applicable legislation.
2
Efgartigimod: Broad Pipeline Opportunity
Landscape of
IgG-mediated Severe
Autoimmune Diseases
(sampling)
Immune Lupus Thrombocytopenia
Guillain-Barré syndrome
Myasthenia Gravis
Scleroderma
Anca Vasculitis
Solid Biology | Feasible for |
Rationale: | Biotech: |
Predominantly | Orphan indication, |
mediated by | efficient clinical & |
pathogenic IgGs | regulatory pathway |
argenx Franchises & Indications
Efgartigimod to date achieved proof-of-concept in 4/4 indications; 2/2 in neuromuscular franchise
Myasthenia Gravis
Neuromuscular
Diseases | Chronic Inflammatory Demyelinating Polyneuropathy |
5th indication
Hematology
Epidermolysis Bullosa
Acquisita
Neuromyelitis
Pemphigus Optica
Disorders
Immune Thrombocytopenia
Skin Blistering
Diseases
Pemphigus Vulgaris
Multiple Sclerosis
Membraneous Hemolytic Anemia
Nephropathy
Rheumatoid
Arthritis
Thyroid Eye Disease
Kidney | TBD |
Bullous Pemphigoid
6th indication
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CIDP: Significant Unmet Need Exists for Patients
Rare, Chronic, and
Progressive
Symmetric proximal and distal weakness with sensory loss and decreased reflexes
Can progress quickly to severe disability (wheelchair)
50% of patients are severely disabled at some stage of illness
Pain and fatigue commonly reported
Prevalence &
Opportunity
~16,000 patients in the US
> $ 3Bn in IVIG Sales globally
Diagnosis/Metrics
Commonly misdiagnosed
Diagnosis often confirmed by physicians trying therapy for 3 months and reassessing
Treatment
70% of CIDP patients need ongoing treatment
Available treatments come with long infusion times and adverse effects
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arGEN-X SE published this content on 01 February 2021 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 01 February 2021 07:39:02 UTC.