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Pharming and Santarus announce poster presentation of pivotal clinical data for RUCONEST(R) (recombinant human C1 esterase inhibitor)

June 25, 2013 - Leiden, The Netherlands And San Diego, CA

Pharming Group NV(NYSE Euronext: PHARM) and Santarus, Inc. (NASDAQ: SNTS), announced thatnew data from a pivotal Phase III clinical study with RUCONEST®(recombinant human C1 esterase inhibitor) for the treatment of acuteangioedema attacks in patients with hereditary angioedema (HAE) werefeatured in a poster presentation yesterday at the European Academy ofAllergy and Clinical Immunology (EAACI) & World Allergy Organization (WAO)World Allergy & Asthma Congress in Milan, Italy. The data indicate that thetime to beginning of relief of symptoms in patients experiencing an acuteattack of HAE was statistically significantly shorter with RUCONESTcompared with placebo.

"RUCONEST has the potential to be an important addition to the therapeuticoptions available for the treatment of acute attacks of HAE based on theencouraging clinical data observed to date," said Marc Riedl, M.D., SectionHead Department of Clinical Immunology and Allergy, University ofCalifornia, Los Angeles and lead author of the poster.

The poster is titled, Recombinant Human C1 Inhibitor for Treatment of AcuteAttacks of Hereditary Angioedema: A Randomized, Double-Blind, Placebo-Controlled Clinical Trial. The primary endpoint and safety data from thestudy are summarized below:

Time to Beginning of Relief of Symptoms

| | Median (95% CI), minutes |p-value*|
| |Placebo |RUCONEST | |
| | (N=31) | (N=44) | |
|Based on Treatment Effect | | | |
|Questionnaire | 152 (93, -) |90 (61, 150)| 0.031 |
|(TEQ) (Primary Endpoint) | | | |
|Based on Visual Analog Scale | | | |
|(VAS) Decrease >= 20 mm |303 (81, 720) |75 (60, 105)| 0.003|
| | | | |

*Based on log-rank test stratified by primary attack location; CI,
confidence interval: -, not calculable.

Treatment-Emergent Adverse Events Occurring Within 72 Hours in 5% or More
of Patients

| | Placebo | RUCONEST |
| | (N=18) | (N=56) |
| | n (%) | n (%) |
| Treatment-emergent adverse events | 4 (22%) | 4 (7%) |
| Sinus congestion | 1 (6%) | 0 |
| Vasomotor rhinitis | 1 (6%) | 0 |
| Diarrhea | 1 (6%) | 0 |
| Dyspepsia | 1 (6%) | 0 |

Note: Patients who received placebo (saline) followed by RUCONEST asrescue medications are summarized as placebo up to receipt of rescuemedication and as RUCONEST afterwards.

* No thromboembolic events, anaphylaxis or neutralizing antibodies were Observed

* One patient experienced a serious adverse event (abdominal hernia)approximately 79 days after RUCONEST administration

* No patients withdrew due to adverse events

Pharming and Santarus are seeking U.S. marketing approval of RUCONEST forthe treatment of acute angioedema attacks in patients with HAE. TheBiologics License Application (BLA) filing for RUCONESTis under review bythe U.S. Food and Drug Administration (FDA) with a response expected byApril 16, 2014. RUCONEST is approved in Europe for the treatment of acuteangioedema attacks in patients with HAE and is an investigational drug inthe U.S. that has been granted orphan drug designation by the FDA.

About RUCONEST and Hereditary Angioedema

RUCONEST (INN conestat alfa) is a recombinant version of the human proteinC1 esterase inhibitor, and is produced with Pharming's proprietarytransgenic technology. RUCONEST is approved in Europe for the treatment ofacute angioedema attacks in patients with HAE, a genetic disorder in whichthe patient is deficient in or lacks a functional plasma protein C1esterase inhibitor, resulting in unpredictable and debilitating episodes ofintense swelling. The swelling may occur in one or more anatomical areas,including the extremities, face, trunk, genitals, abdomen and upper airway.The frequency and severity of HAE attacks vary and are most serious whenthey involve laryngeal edema, which can close the upper airway and causedeath by asphyxiation. According to the U.S. Hereditary AngioedemaAssociation, epidemiological estimates for HAE range from one in 10,000 toone in 50,000 individuals. RUCONEST is an investigational drug in the U.S.and has been granted orphan drug designation by the FDA both for thetreatment of acute attacks of HAE and for prophylactic treatment of HAE.


The European Academy of Allergy and Clinical Immunology, EAACI, is a non-profit organisation active in the field of allergic and immunologicdiseases such as Asthma, rhinitis, eczema, occupational allergy, food anddrug allergy and anaphylaxis. EAACI was founded in 1956 in Florence andhas become the largest medical association in Europe in the field ofallergy and clinical immunology. It includes over 7,800 members from 121countries, as well as 42 National Allergy Societies.

About WAO

The World Allergy Organization (WAO) is an international alliance of 92regional and national allergy, Asthma and clinical immunology societies.Through collaboration with the Member Societies, WAO provides a wide rangeof educational and outreach programs, symposia, and lectureships toallergists/immunologists around the globe and conducts initiatives relatingto clinical practice, service provision, and physician training in order tobetter understand and address the challenges facingallergists/immunologists worldwide.

About Pharming Group NV

Pharming Group NV is developing innovative products for the treatment ofunmet medical needs. RUCONEST® is a recombinant human C1 esteraseinhibitor approved for the treatment of angioedema attacks in patients withHAE in all 27 EU countries plus Norway, Iceland and Liechtenstein, and isdistributed in the EU by Swedish Orphan Biovitrum. RUCONEST® ispartnered with Santarus, Inc. (NASDAQ: SNTS) in North America and aBiologics License Application for RUCONEST is under review by the U.S. Foodand Drug Administration. The product is also being evaluated for variousfollow-on indications. Pharming has a unique GMP compliant, validatedplatform for the production of recombinant human proteins that has provencapable of producing industrial volumes of high quality recombinant humanprotein in a more economical way compared to current cell basedtechnologies. Pharming now plans to utilise this platform for thedevelopment of rhFVIII for the treatment of Haemophilia A. Additionalinformation is available on the Pharming website,

About Santarus

Santarus, Inc. is a specialty biopharmaceutical company focused onacquiring, developing and commercializing proprietary products that addressthe needs of patients treated by physician specialists. The company'scurrent commercial efforts are focused on five products.UCERIS®(budesonide) extended release tablets for the induction ofremission in patients with active, mild to moderate ulcerative colitis andZEGERID® (omeprazole/sodium bicarbonate) for the treatment of certainupper gastrointestinal disorders are promoted to gastroenterologists.GLUMETZA® (metformin hydrochloride extended release tablets) andCYCLOSET® (bromocriptine mesylate) tablets, which are indicated asadjuncts to diet and exercise to improve glycemic control in adults withtype 2 diabetes, and FENOGLIDE® (fenofibrate) tablets, which isindicated as an adjunct to diet to reduce high cholesterol, are promoted toendocrinologists and other physicians who treat patients with type 2diabetes. Full prescribing and safety information for Santarus' productsis available at or by contacting Santarus at 1-888-778-0887.

Santarus' product development pipeline includes the investigational drugRUCONEST®(recombinant human C1 esterase inhibitor). A BiologicsLicense Application for RUCONEST for the treatment of acute angioedemaattacks in patients with hereditary angioedema is under review by the U.S.Food and Drug Administration with a response expected in April 2014.Santarus is also developing rifamycin SV MMX®, which is in Phase IIIclinical testing for the treatment of travelers' diarrhea. In addition,the company has completed a Phase I clinical program with SAN-300, aninvestigational monoclonal antibody. More information about Santarus isavailable at

Santarus and Pharming caution you that statements included in this pressrelease that are not a description of historical facts are forward-lookingstatements. The inclusion of forward-looking statements should not beregarded as a representation by Santarus or Pharming that any of its plansor objectives will be achieved. Actual results may differ materially fromthose set forth in this release due to the risks and uncertainties inherentin Santarus and Pharming's businesses, including, without limitation:whether the FDA will approve the RUCONEST BLA in a timely manner or at all;whether the FDA will concur with the clinical interpretation of the PhaseIII study results or the conduct of the study; whether the FDA ultimatelywill require additional clinical studies or other development programsbefore approving RUCONEST; risks related to Santarus' dependence onPharming for many functions related to RUCONEST, and Pharming's ability tocontinue to perform these functions based on its limited financialresources; risks related to the license and supply arrangements betweenSantarus and Pharming, including the potential for termination of thearrangements; other difficulties or delays in development, testing,manufacturing and marketing of, and obtaining and maintaining regulatoryapprovals for, Santarus and Pharming's products; and other risks detailedin prior press releases as well as in public periodic filings with theSecurities and Exchange Commission, including Santarus' Quarterly Report onForm 10-Q for the quarter ended March 31, 2013.

You are cautioned not to place undue reliance on these forward-lookingstatements, which speak only as of the date hereof. All forward-lookingstatements are qualified in their entirety by this cautionary statement andneither Santarus nor Pharming undertakes any obligation to revise or updatethis news release to reflect events or circumstances after the date hereof,except as may be required by law. This caution is made under the safeharbor provisions of Section 21E of the Private Securities LitigationReform Act of 1995.

Santarus®, FENOGLIDE®, UCERIS®, and ZEGERID® areregistered trademarks of Santarus, Inc. GLUMETZA® is a trademark ofBiovail Laboratories International S.r.l. licensed exclusively in theUnited States to Depomed, Inc. CYCLOSET® is a trademark of VeroScienceLLC. MMX® is a trademark of Cosmo Technologies Limited. RUCONEST®is a trademark of Pharming Group N.V.

# # #

Press release (PDF):

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(i) the releases contained herein are protected by copyright and other applicable laws; and

(ii) they are solely responsible for the content, accuracy and originality of the information contained therein.

Source: Pharming Group N.V. via Thomson Reuters ONE


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