RELEASE: Pharming announces positive analysis data from leniolisib open-label extension study (1)

(Information sent by the signatory company).

RELEASE: Pharming announces positive analysis data from leniolisib open-label extension study (1)

(Information sent by the signatory company)

Pharming announces positive provisional analysis data from the open label extension study of leniolisib in presentation at the 64th annual meeting and exhibition of the American Society of Hematology (ASH)

V. Koneti Rao, MD, shared new evidence of long-term safety and hematologic response in patients receiving leniolisib to treat APDS, a rare primary immunodeficiency

Interim analysis demonstrated that leniolisib is well tolerated and indicated the durability of the efficacy results seen in the phase II/III randomized controlled trial

LEIDEN, The Netherlands, Dec. 15, 2022 /PRNewswire/ -- Pharming Group N.V. ("Pharming" or "the Company") (EURONEXT Amsterdam: PHARM) (Nasdaq: PHAR) announces data, including new evidence, from an interim analysis of its open-label extension study evaluating the investigational drug leniolisib, a drug Oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, for the treatment of adult and adolescent patients with activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency. Principal investigator V. Koneti Rao, M.D., a staff physician at the National Institutes of Health Primary Immunodeficiency Clinic in Bethesda, Maryland, USA, shared the positive findings in an oral presentation at the Society's Annual Meeting. 2022 American Hematology Award (ASH).

Dr Virgil Dalm, Principal Investigator, Consultant Clinical Immunology, Erasmus MC, Rotterdam, The Netherlands, commented:

"I am excited about the Pharming findings that further support leniolisib as a well-tolerated investigational treatment benefiting patients with APDS. The results demonstrate the long-term tolerability of leniolisib, with a median duration on study therapy of just over 2 years (102 weeks) and 5 subjects on treatment for 5 years or more.Persons with APDS frequently suffer from recurrent infections and lifelong Immunoglobulin Replacement Therapy (IRT) is required to reduce this burden.In particular , treatment with leniolisib demonstrated a significant reduction in the annualized infection rate, while 37% of study patients with ESRDs were able to reduce or completely discontinue their ESRD regimens.This is a remarkable result for any study of inborn errors. of immunity and, through the continued study of leniolisib, I hope to contribute to the knowledge of an improved treatment option for p people with APDS".

The ongoing extension study includes 37 APDS patients aged 12 years and older who, at the time of data cutoff for interim analysis, had received the selective PI3Kδ inhibitor leniolisib 70 mg twice daily for up to six years and three months, with a median duration on study therapy of 102 weeks. The study was designed primarily to assess the long-term safety and tolerability of leniolisib treatment in adolescent and adult patients with APDS who previously participated in a phase II/III study with leniolisib. The secondary endpoints of the extension study are intended to assess the efficacy and pharmacokinetics of long-term treatment with leniolisib in these patients.

The interim analysis found that leniolisib was well tolerated up to this point in the study. It also indicated the durability of the efficacy results seen in the randomized controlled trial, which showed a significant improvement over placebo in the co-primary endpoints of reduced lymph node size and increased naïve B cells. Interim results indicate a favorable long-term effect on immune dysregulation and deficiency often seen in patients with APDS, with clinical manifestations including infections, lymphoproliferation, autoimmunity, enteropathy, bronchiectasis, increased risk of lymphoma, and early mortality.

The majority of adverse events (AEs) reported in the interim analysis were grade 1 and 2 and included upper respiratory tract infection, headache, and pyrexia. Grade 1 AAs are the least serious and grade 5 the most serious. Overall, 13.5% of adverse events were study drug related; these affected five patients and included weight gain, arthralgia, hyperglycemia, and decreased neutrophil count. Of all AEs evaluated in the analysis, 16.2% were classified as severe, but none were identified as related to study treatment. There was one death among study participants that was identified as unrelated to study treatment.

Among the study participants, some experienced reductions in APDS disease markers, with response levels varying between individuals. Responses included:

Notably, 37% of participants who were on immunoglobulin replacement therapy (IRT) were able to reduce their IRT use while taking leniolisib. Six patients became IRT-independent, and four of these patients had been IRT-independent for 1 to 2.5 years at the data cutoff. From the data cutoff for the interim analysis, among the three patients who had a history of lymphoma prior to the trial, none had a recurrence or new lymphoma while participating in the study.

Anurag Relan, MD, MPH, Pharming's chief medical officer, commented:

"Pharming is pleased to share positive interim findings on the long-term safety and efficacy of leniolisib. The results announced at the 2022 ASH Annual Meeting build on the Phase II/III study findings announced earlier this year and published in Blood1 in November 2022, which highlighted the potential of leniolisib to control the development of immune-related symptoms of APDS.We are proud to help fill an unmet need by developing what could become the first drug approved to attack the cause of APDS".

The findings of the interim analysis are consistent with data, first reported on February 2, 2022, for the Phase II/III clinical trial investigating leniolisib as a treatment for adult and adolescent patients with APDS. Compared with placebo, patients in the phase II/III clinical trial achieved significant reductions in index lymph node size and increases in the percentage of peripheral blood naïve B cells.

Based on the Phase II/III clinical trial results and long-term open-label extension data, the US Food and Drug Administration (FDA) is conducting priority New Drug Application review. of Pharming for leniolisib as a treatment for adolescents and adults with APDS and has an assigned Prescription Drug User Fee Act (PDUFA) target date of March 29, 2023. In addition, the Marketing Authorization Application (MAA) of Pharming for leniolisib in the same patient population has been validated for evaluation under accelerated evaluation by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA). The marketing authorization for leniolisib in the European Union is scheduled for the first half of 2023.

About Activated Phosphoinositide 3-Kinase δ Syndrome (APDS)

APDS is a rare primary immunodeficiency that affects approximately 1 to 2 people per million. APDS is caused by variants in either of two genes, PIK3CD or PIK3R1, that regulate white blood cell maturation. Variants of these genes lead to hyperactivity of the PI3Kδ (phosphoinositide 3-kinase delta) pathway.2,3 Balanced signaling in the PI3Kδ pathway is essential for physiological immune function. When this pathway is overactive, immune cells do not mature and function properly, leading to immunodeficiency and dysregulation.2,4 APDS is characterized by severe and recurrent sinopulmonary infections, lymphoproliferation, autoimmunity, and enteropathy.5,6 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 diagnostic delay of 7 years.7 Because APDS is a progressive disease, this delay can lead to to an accumulation of damage over time, including damage and lymphoma.5-8 The only way to definitively diagnose this condition is through genetic testing.

Acerca de leniolisib

Leniolisib is a small molecule inhibitor of the delta isoform of the class IA PI3K 110 kDa catalytic subunit. PI3Kδ is expressed predominantly in hematopoietic cells and is essential for the normal functioning of the immune system through the conversion of phosphatidylinositol-4-5-triphosphate (PIP2) to phosphatidylinositol-3-4-5-triphosphate (PIP3). Leniolisib inhibits the production of PIP3, and PIP3 serves as an important cellular messenger that activates AKT (via PDK1) and regulates a multitude of cellular functions including proliferation, differentiation, cytokine production, cell survival, angiogenesis, and metabolism. Unlike PI3Kα and PI3Kβ, which are ubiquitously expressed, PI3Kẟ and PI3Kγ are mainly expressed in cells of hematopoietic origin. The central role of PI3Kẟ in the regulation of numerous cellular functions of the adaptive immune system (B cells and, to a lesser extent, T cells), as well as the innate immune system (neutrophils, mast cells, and macrophages), clearly indicates that PI3Kẟ is a target Valid and potentially effective therapeutics for immune diseases such as APDS. To date, leniolisib has been well tolerated during the Phase 1 first-in-human trial in healthy subjects and the Phase II/III registration clearance study in patients with APDS.

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. 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 undertakes 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. Rao VK, et al. Blood. 2022. https://doi.org/10.1182/blood.2022018546....2. Lucas CL, et al. Nat Immunol. 2014;15:88-97.3. Elkaim E, et al. J Allergy Clin Immunol. 2016;138(1):210-218.4. Nunes-Santos C, Uzel G, Rosenzweig SD. J Allergy Clin Immunol. 2019;143(5):1676-1687.5. Coulter TI, et al. J Allergy Clin Immunol. 2017;139(2):597-606.6. Maccari ME, et al. Front Immunol. 2018;9:543.7. James M, et al. Clin Rev Allergy Immunol. 2019;May 21.8. Condliffe AM, Chandra A. Front Immunol. 2018;9:338.

For further public information, please 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 Communications E: investor@pharming.com

FTI Consulting, Londres, Reino UnidoVictoria Foster Mitchell/Alex Shaw/Amy ByrneT: 44 203 727 1000

LifeSpring Life Sciences Communication, Ámsterdam, Países BajosLeon MelensT: 31 6 53 81 64 27E: pharming@lifespring.nl

RR. PP. of EE. UU.:Ethan MetelenisE: Ethan.Metelenis@precisionvh.com T: 1 (917)

RR. PP. of the EU:Dan CaleyE: Dan.caley@aprilsix.com T: 44(0)7875468942

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