APDS (activated phosphoinositide 3-kinase delta syndrome (PI3Kδ)) is a rare and progressive primary immunodeficiency. Joenja® is a targeted treatment of APDS for adult and pediatric patients 12 years of age and older. is scheduled for early April. Pharming will host a conference call for investors and analysts on March 27 at 14:00 CEST/08:00 EST
LEIDEN, The Netherlands, March 27, 2023 /PRNewswire/ — Pharming Group N.V. (“Pharming” or “the Company”) (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) announces that the US Food and Drug Administration (FDA) has approved Joenja® (leniolisib) for the treatment of phosphoinositide syndrome Activated delta 3-kinase (PI3Kδ) (APDS) in adult and pediatric patients older than 12 years. Joenja®, a selective oral PI3Kδ inhibitor, is the first and only US-approved treatment for APDS, a rare and progressive primary immunodeficiency. The FDA evaluated Joenja®’s application for APDS under Priority Review, which is granted to therapies that have the potential to provide significant improvements in the treatment, diagnosis, or prevention of serious conditions. Joenja® is expected to launch in the US in early April and will be available to ship in the middle of the same month.
Eveline Wu, MD, MSCR, Head of the Division of Pediatric Rheumatology and Associate Professor of Pediatric Rheumatology and Allergy/Immunology at the University of North Carolina School of Medicine, stated:
“The FDA approval of Joenja® is an exciting time for the APDS community and offers to transform the treatment pathway for patients and families affected by this rare disease. This approval means that, for the first time, they will have access to a treatment approved, which has the potential to change the standard of care for the APDS patient population.”
Vicki Modell, Co-Founder of the Jeffrey Modell Foundation, an international non-profit organization dedicated to helping individuals and families affected by primary immunodeficiency disorders, commented:
“The approval of Pharming’s Joenja ® is an important step in making a difference in the lives of people living with APDS and experiencing severe, progressive and life-altering symptoms. FDA approval of a treatment option for a of the more than 450 primary immunodeficiencies is also a pivotal moment for the primary immunodeficiency community at large.The Jeffrey Modell Foundation’s mission of hope, advocacy, and action is dedicated to early diagnosis, genetic sequencing, treatments, and ultimately instance, future cures for primary immunodeficiencies”.
Sijmen de Vries, CEO of Pharming, commented:
“This FDA approval of Joenja® is an important milestone for people living with APDS, who will now have access to the first treatment option approved specifically for this debilitating disease. Until now, treatment of APDS has been based on treating the various symptoms associated with APDS. We are very grateful to the patients, caregivers and physicians who participated in the clinical trials that made today’s approval a reality. I would also like to thank the teams at Pharming and Novartis who have supported the development of Joenja® and can therefore be justifiably proud of this FDA approval.Today also marks a milestone for Pharming and demonstrates our commitment to transforming the lives of rare disease patients. approval and the short-term launch of Joenja®, our second commercial product, brings us closer to our goal of becoming a leading company global rare disease community dedicated to patient communities with unmet medical needs.
APDS is a rare primary immunodeficiency that was first characterized in 2013 and is currently estimated to affect between 1 and 2 people per million. It is caused by genetic variants in one of two identified genes, known as PIK3CD or PIK3R1, which are vital for the normal development and function of the body’s immune cells. Although people with APDS can experience a wide variety of symptoms, the most common are frequent and severe infections of the ears, sinuses, and upper and lower respiratory tract. Infections usually start in childhood. People with APDS are prone to swollen lymph nodes or an enlarged spleen (splenomegaly), as well as autoimmune and inflammatory symptoms. People with APDS may also be at increased risk of cancers such as lymphoma.
The FDA reviewed the New Drug Application (NDA) for Joenja® under priority review and has approved the drug based on the results of a phase II/III randomized, triple-blind, placebo-controlled, multinational clinical trial evaluating the efficacy of Joenja®. and safety in 31 patients diagnosed with APDS aged 12 years or older. Data from a long-term open-label extension clinical trial in which 38 patients received Joenja® for a median of two years were also submitted as part of the application.
Results from a 12-week randomized placebo-controlled study in 31 APDS patients aged 12 years and older demonstrated clinical efficacy of Joenja® 70 mg twice daily versus placebo, and was significant on the co-primary endpoints evaluating improvement in lymphoproliferation as measured by reduction in lymph node size and increase in naïve B cells, reflecting the impact on immune dysregulation and normalization of the immunophenotype in these patients, respectively. The adjusted mean change (95% CI) between Joenja® and placebo for lymph node size was -0.27 Mar. (-0.38, -0.12; P=0.0006; N=26) – and for the percentage of naïve B cells was 37.30 (24.06, 50.54; P=0.0002; N=13). The most common adverse reactions in the clinical trial (incidence >10%) were headache, sinusitis, and atopic dermatitis.
With the approval of Joenja®, as a treatment for a rare pediatric disease, the FDA granted Pharming a Priority Review Voucher (“PRV”). Pursuant to the terms of Pharming’s 2019 exclusive license agreement with Novartis for leniolisib, Novartis has the right to purchase the PRV from Pharming for a small minority portion of the PRV value. Under the agreement, Pharming will make milestone payments to Novartis and another party for the approval and first commercial sale for APDS totaling $10.5 million and we agree to make certain additional milestone payments to Novartis for an aggregate amount of up to $190 million upon achievement of certain leniolisib sales milestones. We also agree to pay Novartis escalating royalties, calculated in low-double to high-double-digit percentages on net sales of leniolisib.
For more information about APDS, visit AllAboutAPDS.com. Pharming, in partnership with Invitae Corporation, provides free access to genetic testing and counseling in the US and Canada through NavigateAPDS.com. For more information about Pharming and its dedication to the rare disease community, visit www.Pharming.com.
The Marketing Authorization Application (MAA) for leniolisib with the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) is currently under review. Pharming expects the CHMP to issue its opinion on the MAA in the second half of 2023.
Important safety information in EE. UU. for Joenja® (leniolisib)
INDICATIONS AND USE
Joenja® (leniolisib) is a kinase inhibitor indicated for the treatment of activated phosphoinositide 3-kinase delta syndrome (PI3Kδ) (APDS) in adult and pediatric patients 12 years of age and older.
IMPORTANT SAFETY INFORMATION
Verify the pregnancy status in women of reproductive potential before starting treatment with Joenja®.
Joenja® may cause fetal harm when administered to a pregnant woman. Advise patients of the potential risk to the fetus and to use highly effective contraception during treatment with Joenja® and for 1 week after the last dose of Joenja®.
Live attenuated vaccines may be less effective if administered during treatment with Joenja®.
Joenja® is not recommended for use in patients with moderate to severe hepatic impairment. There is no recommended dose for patients weighing less than 45kg.
The most common adverse reactions (incidence >10%) observed in clinical trials were headache, sinusitis, and atopic dermatitis.
Seven (33%) patients who received Joenja® developed an absolute neutrophil count (ANC) between 500 and 1,500 cells/microL. No patient developed an ANC <500 cells/microL and there were no reports of infections associated with neutropenia.
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 of two identified genes, PIK3CD or PIK3R1, which are vital for the development and function of the body’s immune cells. 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 infections severe and recurrent sinopulmonary disorders, lymphoproliferation, autoimmunity, and enteropathy.4,5 Because these symptoms can be associated with a variety of conditions, including other primary immunodeficiencies, people with APDS are frequently misdiagnosed and experience a median delay in diagnosis. than 7 years.6 Since APDS is a progressive disease, this delay can lead to an accumulation of damage over time, including permanent lung damage and lymphoma.4-7 The only way to definitively diagnose this condition is through tests genetics. APDS affects approximately 1 or 2 people per million worldwide.
Acerca de Joenja® (leniolisib)
Joenja® (leniolisib) is an oral small molecule inhibitor of phosphoinositide 3-kinase delta (PI3Kẟ) 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 and older. Joenja® inhibits the production of phosphatidylinositol-3-4-5-trisphosphate, which acts as an important cellular messenger and regulates a multitude of cellular functions including 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® on co-primary endpoints, with a statistically significant impact on immune dysregulation and normalization of immunophenotype in these patients. and interim open-label extension data have supported the safety and tolerability of long-term administration of Joenja®.8 Leniolisib is currently under regulatory review by the European Medicines Agency, with plans to obtain further regulatory approvals in UK, Canada, Australia and Japan. Leniolisib is also being evaluated in a Phase III clinical trial in children 4 to 11 years of age with APDS, and another trial in children 1 to 6 years of age with APDS is planned. For more information on Joenja®, visit: Joenja.com
Acerca de 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 precision medicines and protein replacement therapies, including small molecules, biologics and gene therapies that are in early or late stages of development. Pharming is headquartered in Leiden, The Netherlands, with employees around the world caring for patients in more than 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. statements. These forward-looking statements are identified by the use of terms and phrases such as “target”, “ambition”, “anticipate”, “believe”, “could”, “estimate”, “expect”, ”goals”, ” aim”, ”can”, ”milestones”, ”objectives”, ”outlook”, ”plan”, ”probably”, ”project”, ”risks”, “program,” “seek,” “should,” “target,” “will,” and similar terms and phrases. Examples of forward-looking statements may include statements regarding the 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, the statements of which 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 their cost ramifications yes; and clinical, scientific, regulatory and technical developments. In light of these risks and uncertainties, and other risks and uncertainties described in Pharming’s 2021 Annual Report and Annual Report on Form 20-F for the year ended December 31, 2021, filed with the Commission on In the US Securities and Exchange, 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 by such forward-looking statements. 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. All 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 press release. Pharming assumes no obligation to publicly update or revise any.
internal information
This press release concerns 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.
Conference access information for investors and analysts
March 27, 2023: 2:00 p.m. CEST/8:00 a.m. EST
Please note that the company will only answer questions from attendees who connect by phone.
Dialing details:
Netherlands (Local) 31 85 888 7233 United Kingdom 44 808 189 0158 United Kingdom (Local) 44 20 3936 2999 United States 1 855 979 6654 United States (Local) 1 646 664 1960
Global Dialing Numbers
Access code: 040991
Webcast link: https://webcast.openbriefing.com/pharming-mar23/
For more public information, contact:
Pharming Group, Leiden, The Netherlands Michael Levitan, Vice President Investor Relations and Corporate Communications T: 1 (908) 705 1696
Heather Robertson, Director of Investor Relations and Corporate CommunicationsE: investor@pharming.com
FTI Consulting, Londres, Reino Unido Victoria Foster Mitchell/Alex Shaw/Amy ByrneT: 44 203 727 1000
LifeSpring Life Sciences Communication, Ámsterdam, Países Bajos Leon MelensT: 31 6 53 81 64 27E: pharming@lifespring.nl
RR. PP. of EE. UU. Ethan MetelenisE: Ethan.Metelenis@precisionvh.comT: 1 (917)
RR. PP. of the EU Claire DobbsE: claire.dobbs@solarishealth.com T: 44 7864 640093
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