PIK3CD

Pharming Group reports fourth quarter and full year 2023 financial results

Retrieved on: 
Thursday, March 14, 2024
Patient, Health care, Epidemiology, Executive Committee, Laboratory, Microsoft Access, Lymphocyte, Clinical trial, European Commission, APDS, MHLW, AEG, Welfare, Ageing, Committee, EMA, Trial of the century, PID, CTLA4, Safety, Pharmacokinetics, Ministry, CHMP, ODD, U.S. FDA, Prevalence, PTEN, Disease, HAE, Immunodeficiency, VUS, Literature, PIK3R1, PMDA, FDA, DSM-IV codes, Diagnosis, Civil service commission, IRP, Haploinsufficiency, Genetic testing, MAA, Autoimmunity, World Games, Iberian languages, Marketing, MHRA, Euronext Amsterdam, PIK3CD, Physician, Pharmaceutical industry

The U.S. market contributed 97% of 2023 revenues, while the EU and Rest of World contributed 3%.

Key Points: 
  • The U.S. market contributed 97% of 2023 revenues, while the EU and Rest of World contributed 3%.
  • Revenues increased to US$7.9 million in the fourth quarter of 2023, driven by the continued increase in patients on paid therapy, and revenues were US$18.2 million for 2023.
  • Pharming made continued progress in the fourth quarter of 2023 on leniolisib regulatory filings for APDS patients 12 years of age and older in key global markets.
  • Pharming filed regulatory submissions in Canada and Australia in the third quarter of 2023, and Israel in the second quarter.

Pharming Group announces updated full year 2023 guidance and Joenja® U.S. launch update

Retrieved on: 
Monday, January 8, 2024
APDS, PIK3CD, VUS, FDA, Disease, Immunodeficiency, PIK3R1, Laboratory, Euronext Amsterdam, Growth, Patient, Pids, HAE, Pharmaceutical industry

For the full year 2023, total revenues are expected to increase by 19% to approximately US$245 million (preliminary and unaudited*).

Key Points: 
  • For the full year 2023, total revenues are expected to increase by 19% to approximately US$245 million (preliminary and unaudited*).
  • We expect 10% growth in RUCONEST® revenues, significantly exceeding our previous guidance for low single digit annual revenue growth.
  • In 2023, Pharming saw the first full-year, post-pandemic benefit of its first quarter 2020 restructuring and expansion of its U.S. salesforce.
  • Pharming expects to issue full financial results for the fourth quarter and full year 2023 in March 2024.

Cellectis and Imagine Institute Publish A Proof-Of-Concept Study of a Gene Surgery Candidate to Treat Activated Phosphoinositide 3-Kinase Δ Syndrome Type 1 (APDS1)

Retrieved on: 
Thursday, October 12, 2023
PIK3CD, TALEN, Euronext Growth, Research, Mythic Imagination Institute, Vaccine, Management, Cellectis

NEW YORK, Oct. 12, 2023 (GLOBE NEWSWIRE) -- Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, announced today the publication of a new research paper in Molecular Therapy – Methods & Clinical Development, demonstrating the efficacy of its TALEN-mediated gene correction of mutated PIK3CD gene in APDS1 T-cells.

Key Points: 
  • NEW YORK, Oct. 12, 2023 (GLOBE NEWSWIRE) -- Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, announced today the publication of a new research paper in Molecular Therapy – Methods & Clinical Development, demonstrating the efficacy of its TALEN-mediated gene correction of mutated PIK3CD gene in APDS1 T-cells.
  • The research work described in this article was jointly conducted by Imagine Institute and Cellectis teams.

PANTHERx® Rare Launches Drug for APDS

Retrieved on: 
Tuesday, March 28, 2023
Urinary tract infection, PIK3CD, Failure, Attention deficit hyperactivity disorder predominantly inattentive, Pharmacy, Immunodeficiency, Quality of life, Mutation, Medicine, Sinusitis, PI3K, PASLI disease, Patient, Bronchiectasis, Primary immunodeficiency, Rare, PIK3R1, Headache, Gene, APDS, Pharmaceutical industry, Vaccine

Through innovation and technology, we continue to make a positive impact on the quality of life of those living with rare diseases.

Key Points: 
  • Through innovation and technology, we continue to make a positive impact on the quality of life of those living with rare diseases.
  • We are pleased to add Joenja® to the list of medications in PANTHERx® portfolio," said Rob Snyder, president of PANTHERx® Rare.
  • Activated Phosphoinositide 3-Kinase-Delta Syndrome, or APDS, is a rare primary immunodeficiency affecting approximately one to two people per million with disease onset occurring as early as infancy.
  • Those with APDS may develop recurrent respiratory tract infections, bronchiectasis, lymphoproliferation as well as neurodevelopment delay and failure to thrive.

Pharming announces US FDA approval of Joenja® (leniolisib) as the first and only treatment indicated for APDS

Retrieved on: 
Friday, March 24, 2023
US Foods, PIK3CD, Fetus, Senior counsel, Birth, New Drug Application, Atopic dermatitis, Autoimmunity, Associate, Committee, Incidence, MD, Patient, Risk, Ageing, Disease, Priority review, Hope, EMA, Primary immunodeficiency, Diagnosis, FDA, Headache, Caregiver, Safety, Rheumatology, Liver, PASLI disease, Marketing Authorisation Application, Lymph, University, European Medicines Agency, MAA, PIK3R1, Splenomegaly, CHMP, Lymphoma, ANC, PRV, Euronext Amsterdam, Infection, Clinical trial, NDA, Sale, Sinusitis, Physician, Immunodeficiency, Genetic testing, Gene, Food, APDS, Pharmaceutical industry, Management, Vaccine, Pharming, Novartis, Medicine

LEIDEN, The Netherlands, March 24, 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 activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older. Joenja®, an oral, selective PI3Kδ inhibitor, is the first and only treatment approved in the US for APDS, a rare and progressive primary immunodeficiency. The FDA evaluated the Joenja® 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 for shipment in mid-April.

Key Points: 
  • Joenja®, an oral, selective PI3Kδ inhibitor, is the first and only treatment approved in the US for APDS, a rare and progressive primary immunodeficiency.
  • The FDA approval of a treatment option for one of the more than 450 primary immunodeficiencies is also a key moment for the broader primary immunodeficiency community.
  • Until now, management of APDS has relied on the treatment of the diverse symptoms associated with APDS.
  • With the approval of Joenja®, as a treatment for a rare pediatric disease, the FDA granted Pharming a priority review voucher ("PRV").

Pharming announces US FDA approval of Joenja® (leniolisib) as the first and only treatment indicated for APDS

Retrieved on: 
Friday, March 24, 2023
US Foods, PIK3CD, Fetus, Senior counsel, Birth, New Drug Application, Atopic dermatitis, Autoimmunity, Associate, Committee, Incidence, MD, Patient, Risk, Ageing, Disease, Priority review, Hope, EMA, Primary immunodeficiency, Diagnosis, FDA, Headache, Caregiver, Safety, Rheumatology, Liver, PASLI disease, Marketing Authorisation Application, Lymph, University, European Medicines Agency, MAA, PIK3R1, Splenomegaly, CHMP, Lymphoma, ANC, PRV, Euronext Amsterdam, Infection, Clinical trial, NDA, Sale, Sinusitis, Physician, Immunodeficiency, Genetic testing, Gene, Food, APDS, Pharmaceutical industry, Management, Vaccine, Pharming, Novartis, Medicine

LEIDEN, The Netherlands, March 24, 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 activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older. Joenja®, an oral, selective PI3Kδ inhibitor, is the first and only treatment approved in the US for APDS, a rare and progressive primary immunodeficiency. The FDA evaluated the Joenja® 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 for shipment in mid-April.

Key Points: 
  • Joenja®, an oral, selective PI3Kδ inhibitor, is the first and only treatment approved in the US for APDS, a rare and progressive primary immunodeficiency.
  • The FDA approval of a treatment option for one of the more than 450 primary immunodeficiencies is also a key moment for the broader primary immunodeficiency community.
  • Until now, management of APDS has relied on the treatment of the diverse symptoms associated with APDS.
  • With the approval of Joenja®, as a treatment for a rare pediatric disease, the FDA granted Pharming a priority review voucher ("PRV").

Pharming Group announces presentation of new leniolisib data at 2022 ASH Annual Meeting

Retrieved on: 
Wednesday, November 23, 2022
Human, Patient, Primary immunodeficiency, PDK1, AKT, Foundation for the National Institutes of Health, APDS, Immune system, Neutrophil, Euronext Amsterdam, Metabolism, COVID-19, PIP2, Genetic testing, Growth hacking, Immunity, PIP3, Exposition, PI3K, Immunodeficiency, B cell, Attention deficit hyperactivity disorder predominantly inattentive, Autoimmunity, T(D) v L(F) & Anor, Society, Macrophage, Phase, Multimedia, Disclosure, PIK3R1, Lymphocyte, PIK3CD, Annual report, Gene, ASH, Cell, Pharmaceutical industry, Online shopping, Vaccine, Mouse, MD, Pharming

LEIDEN, Netherlands, Nov. 23, 2022 /PRNewswire/ -- Pharming Group N.V. ("Pharming" or "the Company") (EURONEXT Amsterdam: PHARM) (Nasdaq: PHAR) announces today that new clinical data for leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, an investigational treatment for activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency, will be presented by V. Koneti Rao, MD, FRCPA, staff physician in the Primary Immune Deficiency Clinic at the National Institute of Health in Bethesda, Maryland, at the 64th American Society of Hematology (ASH) Annual Meeting and Exposition, in New Orleans, Louisiana taking place from Saturday, December 10 through  Tuesday, December 13, 2022.

Key Points: 
  • Information regarding Pharming's data presentation can be found below, and on the ASH conference website: https://www.hematology.org/meetings/annual-meeting .
  • APDS is caused by variants in either of two genes, PIK3CD or PIK3R1, that regulate maturation of white blood cells.
  • Leniolisib is a small-molecule inhibitor of the delta isoform of the 110 kDa catalytic subunit of class IA PI3K.
  • 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 Group announces presentation of new leniolisib data at 2022 ASH Annual Meeting

Retrieved on: 
Wednesday, November 23, 2022
Human, Patient, Primary immunodeficiency, PDK1, AKT, Foundation for the National Institutes of Health, APDS, Immune system, Neutrophil, Euronext Amsterdam, Metabolism, COVID-19, PIP2, Genetic testing, Growth hacking, Immunity, PIP3, Exposition, PI3K, Immunodeficiency, B cell, Attention deficit hyperactivity disorder predominantly inattentive, Autoimmunity, T(D) v L(F) & Anor, Society, Macrophage, Phase, PIK3R1, Disclosure, Lymphocyte, PIK3CD, Annual report, Gene, ASH, Cell, Pharmaceutical industry, Vaccine, Mouse, MD, Pharming

LEIDEN, Netherlands, Nov. 23, 2022 /PRNewswire/ -- Pharming Group N.V. ("Pharming" or "the Company") (EURONEXT Amsterdam: PHARM) (Nasdaq: PHAR) announces today that new clinical data for leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, an investigational treatment for activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency, will be presented by V. Koneti Rao, MD, FRCPA, staff physician in the Primary Immune Deficiency Clinic at the National Institute of Health in Bethesda, Maryland, at the 64th American Society of Hematology (ASH) Annual Meeting and Exposition, in New Orleans, Louisiana taking place from Saturday, December 10 through  Tuesday, December 13, 2022.

Key Points: 
  • Information regarding Pharming's data presentation can be found below, and on the ASH conference website: https://www.hematology.org/meetings/annual-meeting .
  • APDS is caused by variants in either of two genes, PIK3CD or PIK3R1, that regulate maturation of white blood cells.
  • Leniolisib is a small-molecule inhibitor of the delta isoform of the 110 kDa catalytic subunit of class IA PI3K.
  • 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 Announces European Medicines Agency (EMA) Validates its Marketing Authorisation Application under Accelerated Assessment for leniolisib

Retrieved on: 
Friday, October 28, 2022
Gene, Phase, MAA, PIK3R1, U.S. Securities and Exchange Commission, Company, Attention deficit hyperactivity disorder predominantly inattentive, Primary immunodeficiency, Marketing, Safety, Disclosure, Forward-looking statement, Goal, B cell, PDK1, PIP2, AKT, PIK3CD, European Economic Area, Cell, Immunodeficiency, Committee, PI3K, European Medicines Agency, Macrophage, MPH, Growth hacking, Genetic testing, Metabolism, Annual report, Patient, Phrase, Immune system, APDS, Euronext Amsterdam, Committee for Medicinal Products for Human Use, Review, Autoimmunity, PIP3, CHMP, Time, Marketing Authorisation Application, Neutrophil, Advanced Therapy Medicinal Product, EMA, Public health, Vaccine, Pharmaceutical industry, MD, Pharming

LEIDEN, Netherlands, Oct. 28, 2022 /PRNewswire/ -- Pharming Group N.V. ("Pharming" or "the Company") (EURONEXT Amsterdam: PHARM / Nasdaq: PHAR) announces today that its Marketing Authorisation Application (MAA) for leniolisib has been validated for scientific evaluation under an accelerated assessment by the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP). The application, submitted earlier in October 2022, is for the investigational drug, leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, as a treatment for activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency, in adolescents and adults 12 years or older.

Key Points: 
  • LEIDEN, Netherlands, Oct. 28, 2022 /PRNewswire/ -- Pharming Group N.V. ("Pharming" or "the Company") (EURONEXT Amsterdam: PHARM / Nasdaq: PHAR) announces today that its Marketing Authorisation Application (MAA) for leniolisib has been validated for scientific evaluation under an accelerated assessment by the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use ( CHMP ).
  • In August 2022, Pharming announced the leniolisib MAA was granted accelerated assessment by EMA's CHMP.
  • Marketing authorisation for leniolisib in the European Economic Area is anticipated in H1 2023.
  • This review constitutes a key milestone in Pharming's effort to give healthcare providers and their patients global access to leniolisib.

Pharming Announces European Medicines Agency (EMA) Validates its Marketing Authorisation Application under Accelerated Assessment for leniolisib

Retrieved on: 
Friday, October 28, 2022
Gene, Phase, MAA, PIK3R1, U.S. Securities and Exchange Commission, Company, Attention deficit hyperactivity disorder predominantly inattentive, Primary immunodeficiency, Marketing, Safety, Disclosure, Forward-looking statement, Goal, B cell, PDK1, PIP2, AKT, PIK3CD, European Economic Area, Cell, Immunodeficiency, Committee, PI3K, European Medicines Agency, Macrophage, MPH, Growth hacking, Genetic testing, Metabolism, Annual report, Patient, Phrase, Immune system, APDS, Euronext Amsterdam, Committee for Medicinal Products for Human Use, Review, Autoimmunity, PIP3, CHMP, Time, Marketing Authorisation Application, Neutrophil, Advanced Therapy Medicinal Product, EMA, Public health, Vaccine, Pharmaceutical industry, MD, Pharming

LEIDEN, Netherlands, Oct. 28, 2022 /PRNewswire/ -- Pharming Group N.V. ("Pharming" or "the Company") (EURONEXT Amsterdam: PHARM / Nasdaq: PHAR) announces today that its Marketing Authorisation Application (MAA) for leniolisib has been validated for scientific evaluation under an accelerated assessment by the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP). The application, submitted earlier in October 2022, is for the investigational drug, leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, as a treatment for activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency, in adolescents and adults 12 years or older.

Key Points: 
  • LEIDEN, Netherlands, Oct. 28, 2022 /PRNewswire/ -- Pharming Group N.V. ("Pharming" or "the Company") (EURONEXT Amsterdam: PHARM / Nasdaq: PHAR) announces today that its Marketing Authorisation Application (MAA) for leniolisib has been validated for scientific evaluation under an accelerated assessment by the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use ( CHMP ).
  • In August 2022, Pharming announced the leniolisib MAA was granted accelerated assessment by EMA's CHMP.
  • Marketing authorisation for leniolisib in the European Economic Area is anticipated in H1 2023.
  • This review constitutes a key milestone in Pharming's effort to give healthcare providers and their patients global access to leniolisib.