Pharming announces the first commercial shipments of Joenja® (leniolisib) to patients in the U.S.

Rhea-AI Summary

Pharming Group N.V. (Euronext Amsterdam: PHARM, Nasdaq: PHAR) has announced the first commercial shipments of Joenja® (leniolisib) to patients in the United States. Joenja® is the first and only approved treatment for activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS), a rare primary immunodeficiency affecting adults and children aged 12 and older. The commencement of these shipments triggers a $10 million milestone payment to Novartis, under a 2019 licensing agreement. Joenja® was recently approved by the FDA, and the company aims to make it widely available across the U.S. Additionally, leniolisib is undergoing further regulatory reviews in Europe, the UK, Canada, Australia, and Japan, with ongoing clinical trials for younger patients suffering from APDS.

Positive

• First commercial shipments of Joenja® to U.S. patients mark a significant milestone.
• Triggers a $10 million milestone payment to Novartis, showcasing commercial success.
• Joenja® is the first and only approved treatment for APDS in the U.S., addressing a critical market need.

Negative

• None.

LEIDEN, Netherlands, April 11, 2023 /PRNewswire/ -- Pharming Group N.V. ("Pharming") (Euronext Amsterdam: PHARM) (Nasdaq: PHAR) announces the first commercial shipments of Joenja® (leniolisib) to patients in the United States. Joenja®, an oral, selective PI3Kδ inhibitor, is the first and only treatment approved in the U.S. for activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS), a rare and progressive primary immunodeficiency, in adult and pediatric patients 12 years of age and older.

Under the terms of Pharming's 2019 exclusive license agreement with Novartis for leniolisib, the corresponding first commercial sale of Joenja® triggers a $10 million milestone payment by Pharming to Novartis.

Stephen Toor, Chief Commercial Officer of Pharming, commented:

"We are pleased to announce that the first Joenja® shipments to patients, with payor reimbursement, were delivered approximately two weeks following FDA approval, achieving an important milestone for patients suffering with APDS. We look forward to making Joenja® widely available across the U.S. as the first and only approved treatment for patients with APDS."

About Activated Phosphoinositide 3-Kinase δ Syndrome (APDS)

APDS is a rare primary immunodeficiency that was first characterized in 2013. APDS is caused by variants in either one of two identified genes known as PIK3CD or PIK3R1, which are vital to the development and function of immune cells in the body. Variants of these genes lead to hyperactivity of the PI3Kδ (phosphoinositide 3-kinase delta) pathway, which causes immune cells to fail to mature and function properly, leading to immunodeficiency and dysregulation.^1,2,3 APDS is characterized by a variety of symptoms, including severe, recurrent sinopulmonary infections, lymphoproliferation, autoimmunity, and enteropathy.^4,5 Because these symptoms can be associated with a variety of conditions, including other primary immunodeficiencies, it has been reported that people with APDS are frequently misdiagnosed and suffer a median 7-year diagnostic delay.⁶ As APDS is a progressive disease, this delay may lead to an accumulation of damage over time, including permanent lung damage and lymphoma.^4-7 A definitive diagnosis can be made through genetic testing. APDS affects approximately 1 to 2 people per million worldwide. 

About Joenja® (leniolisib) 

Joenja® (leniolisib) is an oral small molecule phosphoinositide 3-kinase delta (PI3Kẟ) inhibitor approved in the US as the first and only targeted treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older. Joenja® inhibits the production of phosphatidylinositol-3-4-5-trisphosphate, which serves as an important cellular messenger and regulates a multitude of cell functions such as proliferation, differentiation, cytokine production, cell survival, angiogenesis, and metabolism. Results from a randomized, placebo-controlled Phase II/III clinical trial demonstrated clinical efficacy of Joenja® in the co-primary endpoints; demonstrating statistically significant impact on immune dysregulation and normalization of immunophenotype within these patients, and interim open label extension data has supported the safety and tolerability of long-term Joenja® administration.⁸ Leniolisib is currently under regulatory review by the European Medicines Agency, with plans to pursue further regulatory approvals in the UK, Canada, Australia and Japan. Leniolisib is also being evaluated in a Phase III clinical trial in children aged 4 to 11 with APDS, with a further trial planned in children aged 1 to 6 years with APDS.  For information about Joenja®, visit: Joenja.com 

About Pharming Group N.V. 

Pharming Group N.V. (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) is a global biopharmaceutical company dedicated to transforming the lives of patients with rare, debilitating, and life-threatening diseases. Pharming is commercializing and developing an innovative portfolio of protein replacement therapies and precision medicines, including small molecules, biologics, and gene therapies that are in early to late-stage development. Pharming is headquartered in Leiden, Netherlands, and has employees around the globe who serve patients in over 30 markets in North America, Europe, the Middle East, Africa, and Asia-Pacific.

For more information, visit www.pharming.com and find us on LinkedIn.

 Forward-looking Statements 

This press release may contain forward-looking statements. Forward-looking statements are statements of future expectations that are based on management's current expectations and assumptions and involve known and unknown risks and uncertainties that could cause actual results, performance, or events to differ materially from those expressed or implied in these statements. These forward-looking statements are identified by their use of terms and phrases such as "aim", "ambition", ''anticipate'', ''believe'', ''could'', ''estimate'', ''expect'', ''goals'', ''intend'', ''may'', "milestones", ''objectives'', ''outlook'', ''plan'', ''probably'', ''project'', ''risks'', "schedule", ''seek'', ''should'', ''target'', ''will'' and similar terms and phrases. Examples of forward-looking statements may include statements with respect to timing and progress of Pharming's preclinical studies and clinical trials of its product candidates, Pharming's clinical and commercial prospects, and Pharming's expectations regarding its projected working capital requirements and cash resources, which statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to the scope, progress and expansion of Pharming's clinical trials and ramifications for the cost thereof; and clinical, scientific, regulatory and technical developments. In light of these risks and uncertainties, and other risks and uncertainties that are described in Pharming's 2021 Annual Report and the Annual Report on Form 20-F for the year ended December 31, 2021, filed with the U.S. Securities and Exchange Commission, the events and circumstances discussed in such forward-looking statements may not occur, and Pharming's actual results could differ materially and adversely from those anticipated or implied thereby. All forward-looking statements contained in this press release are expressly qualified in their entirety by the cautionary statements contained or referred to in this section. Readers should not place undue reliance on forward-looking statements. Any forward-looking statements speak only as of the date of this press release and are based on information available to Pharming as of the date of this release. Pharming does not undertake any obligation to publicly update or revise any.

Inside Information

This press release relates to the disclosure of information that qualifies, or may have qualified, as inside information within the meaning of Article 7(1) of the EU Market Abuse Regulation.  

References

1.  Lucas CL, et al. Nat Immunol. 2014;15(1):88-97. 

2.  Elkaim E, et al. J Allergy Clin Immunol. 2016;138(1):210-218. 

3.  Nunes-Santos C, Uzel G, Rosenzweig SD. J Allergy Clin Immunol. 2019;143(5):1676-1687. 

4.  Coulter TI, et al. J Allergy Clin Immunol. 2017;139(2):597-606. 

5.  Maccari ME, et al. Front Immunol. 2018;9:543. 

6.  Jamee M, et al. Clin Rev Allergy Immunol. 2019;May 21. 

7.  Condliffe AM, Chandra A. Front Immunol. 2018;9:338. 

8.  RAO VK, et al Blood. 2023 Mar 2;141(9):971-983.  

For further public information, contact:

Pharming Group, Leiden, The Netherlands
Michael Levitan, VP Investor Relations & Corporate Communications
T: +1 (908) 705 1696

Heather Robertson, Investor Relations & Corporate Communications Manager
E: investor@pharming.com 

FTI Consulting, London, UK
Victoria Foster Mitchell/Alex Shaw/Amy Byrne
T: +44 203 727 1000

LifeSpring Life Sciences Communication, Amsterdam, The Netherlands
Leon Melens
T: +31 6 53 81 64 27
E: pharming@lifespring.nl

US PR
Ethan Metelenis
E: Ethan.Metelenis@precisionvh.com
T: +1 (917) 882 9038

EU PR
Claire Dobbs
E: claire.dobbs@solarishealth.com 
T: +44 7864 640093

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SOURCE Pharming Group

FAQ

What is Joenja® and what condition does it treat?

Joenja® (leniolisib) is the first and only approved treatment for activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in the U.S.

When did Pharming Group announce the first commercial shipments of Joenja®?

Pharming Group announced the first commercial shipments of Joenja® on April 11, 2023.

What financial impact did the shipment announcement have for Pharming Group?

The commencement of shipments triggers a $10 million milestone payment to Novartis as per their licensing agreement.

Is Joenja® available for children?

Yes, Joenja® is approved for patients aged 12 years and older, and is currently undergoing clinical trials for younger patients.

What are Pharming's future plans for Joenja®?

Pharming plans to pursue further regulatory approvals for Joenja® in Europe, the UK, Canada, Australia, and Japan.