Pharming Announces Agenda for Its Capital Markets Briefing to be Held on 21 June 2018 in New York

LEIDEN, Netherlands, June 5, 2018 /PRNewswire/ —

New forms of administration for RUCONEST(R) for HAE  

Expansion of rhC1INH clinical development in pre-eclampsia, contrast induced nephropathy and other potential large indications 

Clinical development of protein replacement products in Pompe and Fabry diseases  

Pharming Group N.V. (“Pharming” or “the Company”) announces the agenda for its Capital Markets Briefing to be held from 08:00 to 10:30 EST/14:00-16:30 CET on 21 June 2018 in New York, NY, and through a live webcast. The link to the live webcast and the subsequent recording will be available on Pharming’s website on the front page and under the News section.

During the Capital Markets Briefing, Pharming will discuss its ongoing activities and the strategy for its growing research and development pipeline both for its recombinant human C1 esterase inhibitor (rhC1INH) and new protein replacement products. The briefing is intended to inform shareholders, potential investors and other interested parties about Pharming’s current and planned activities in the areas of:

– new development of its lead product RUCONEST(R) (rhC1INH) within the hereditary
angioedema (HAE) space to meet patients’ needs;
– new development of rhC1INH outside the HAE space to tackle other major unmet medical
needs for which there are no current approved or effective therapies; and
– clinical development of new protein replacement products which address significant
shortcomings of existing therapies.

The Briefing will include presentations from top key opinion leaders in the main indications of interest. Of note, Professor Marc Riedl, Professor of Medicine at the University of California, San Diego and Clinical Director of the US HAEA Angioedema Center and a world expert on the diagnosis, treatment and etiology of hereditary angioedema, and Professor Gustaaf Dekker, of the school of Obstetrics and Gynaecology at the University of Adelaide and a world expert on the etiology and treatment of pre-eclampsia, will be presenting.

An outline of the agenda for the Capital Markets Briefing is detailed below:

– Introduction –  Dr Sijmen de Vries, Chief Executive Officer
– HAE therapeutics overview (acute and prophylaxis indications) – Professor Marc Riedl,
US HAEA Angioedema Center at UCSD
– HAE US market overview – Stephen Toor, Pharming US General Manager
– Building the RUCONEST(R) franchise- Professor Bruno Giannetti, Chief Operating Officer
– rhC1INH development for the treatment/prevention of pre-eclampsia – Professor Gustaaf
Dekker, University of Adelaide
– Contrast-induced nephropathy and cardiac protection – Professor Bruno Giannetti
– Protein replacement therapies in Pompe and Fabry diseases – Professor Bruno Giannetti
– Pharming’s new commercial potential –  Robin Wright, Chief Financial Officer
– Summary – Dr Sijmen de Vries

In the HAE therapeutics section, Professor Riedl will give his view of developments in treatment options for HAE patients.

The US HAE market overview section will focus on the Company’s perspective as to how we believe Pharming will be well positioned to compete in the future.

The RUCONEST(R) franchise section will expand on the new forms of administration for RUCONEST(R) for HAE, including small volume subcutaneous and intramuscular versions and a new, easy-to-use painless intradermal method of administration. This section will also detail Pharming’s plans to develop sufficient capacity to meet demand for rhC1INH in the new indications.

In his section, Professor Dekker will outline the latest scientific view of the development of pre-eclampsia and the rationale for expecting that a therapeutic intervention with C1 esterase inhibitor could have positive therapeutic effects in that condition.

The contrast-induced nephropathy segment will outline the endpoints and potential relevance for further clinical development of a Phase II double-blind placebo-controlled investigator-sponsored study which is ongoing in Basel, Switzerland.  

The protein replacement therapies section will explain the currently-expected advantages of Pharming’s two new proprietary recombinant products: alpha-glucosidase and alpha-galactosidase. alpha-glucosidase is an enzyme replacement therapy for Pompe disease; a rare lysosomal storage deficiency.  alpha-galactosidase, is an enzyme replacement therapy for Fabry disease (also a rare lysosomal storage disease). Timelines for the clinical development program for alpha-glucosidase will be outlined.

The commercial potential section will provide an outline of the markets for the main indications for which the Company intends to develop rhC1INH now and in the near future, and what this could mean in terms of Pharming’s future commercial development potential.

The Company presentations and the recording of the webcast of the briefing will be available on the Company’s website on the front page and under News.

To register to attend the event in person, contact Mike Biega, Solebury Trout: Advanced registration is required, as space is limited.

The Board of Management 

Sijmen de Vries, CEO

Bruno Giannetti, COO

Robin Wright, CFO

About Pharming Group N.V. 

Pharming is a specialty pharmaceutical company developing innovative products for the safe, effective treatment of rare diseases and unmet medical needs. Pharming’s lead product, RUCONEST(R) (conestat alfa) is a recombinant human C1 esterase inhibitor approved for the treatment of acute Hereditary Angioedema (“HAE”) attacks in patients in Europe, the US, Israel and South Korea. The product is available on a named-patient basis in other territories where it has not yet obtained marketing authorization.

RUCONEST(R) is distributed by Pharming in Austria, France, Germany, Luxembourg, the Netherlands, the United Kingdom and the United States of America. Pharming holds commercialisation rights in Algeria, Andorra, Bahrain, Belgium, Ireland, Jordan, Kuwait, Lebanon, Morocco, Oman, Portugal, Qatar, Syria, Spain, Switzerland, Tunisia, United Arab Emirates and Yemen. In some of these countries this is done in association with the HAEi Global Access Program (GAP).

RUCONEST(R) is distributed by Swedish Orphan Biovitrum AB (publ) (SS: SOBI) in the other EU countries, and in Azerbaijan, Belarus, Georgia, Iceland, Kazakhstan, Liechtenstein, Norway, Russia, Serbia and Ukraine.

RUCONEST(R) is distributed in Argentina, Colombia, Costa Rica, the Dominican Republic, Panama, and Venezuela by Cytobioteck, in South Korea by HyupJin Corporation and in Israel by Megapharm.

RUCONEST(R) is also being examined for approval for the treatment of HAE in young children (2-13 years of age) and evaluated for various additional follow-on indications.

Pharming’s technology platform includes a unique, GMP-compliant, validated process for the production of pure recombinant human proteins that has proven capable of producing industrial quantities of high quality recombinant human proteins in a more economical and less immunogenetic way compared with current cell-line based methods. Leads for enzyme replacement therapy (“ERT”) for Pompe and Fabry’s diseases are being optimized at present, with additional programs not involving ERT also being explored at an early stage at present.

Pharming has a long-term partnership with the China State Institute of Pharmaceutical Industry (“CSIPI”), a Sinopharm company, for joint global development of new products, starting with recombinant human Factor VIII for the treatment of Haemophilia A. Pre-clinical development and manufacturing will take place to global standards at CSIPI and are funded by CSIPI. Clinical development will be shared between the partners with each partner taking the costs for their territories under the partnership.

Pharming has declared that the Netherlands is its “Home Member State” pursuant to the amended article 5:25a paragraph 2 of the Dutch Financial Supervision Act.

Additional information is available on the Pharming website:

Forward-looking Statements 

This press release of Pharming Group N.V. and its subsidiaries (“Pharming”, the “Company” or the “Group”) may contain forward-looking statements including without limitation those regarding Pharming’s financial projections, market expectations, developments, partnerships, plans, strategies and capital expenditures. 

The Company cautions that such forward-looking statements may involve certain risks and uncertainties, and actual results may differ. Risks and uncertainties include without limitation the effect of competitive, political and economic factors, legal claims, the Company’s ability to protect intellectual property, fluctuations in exchange and interest rates, changes in taxation laws or rates, changes in legislation or accountancy practices and the Company’s ability to identify, develop and successfully commercialize new products, markets or technologies. 

As a result, the Company’s actual performance, position and financial results and statements may differ materially from the plans, goals and expectations set forth in such forward-looking statements. The Company assumes no obligation to update any forward-looking statements or information, which should be taken as of their respective dates of issue, unless required by laws or regulations. 


Pharming Group N.V.

Sijmen de Vries, CEO, Tel: +31-71-524-7400

Robin Wright, CFO, Tel: +31-71-524-7432

FTI Consulting:

Julia Phillips/ Victoria Foster Mitchell, Tel: +44-203-727-1136

Lifespring Life Sciences Communication

Leon Melens, Tel: +31-6-53-81-64-27