APDS

Pharming announces completion of enrollment in pediatric clinical trial of leniolisib

Retrieved on: 
Monday, April 8, 2024
Disease, Ageing, Patient, Euronext Amsterdam, Clinical trial, MPH, Index, Safety, FDA, APDS, Food, Pharmaceutical industry

Pharming plans to include data from this 4–11-year-old trial in regulatory filings worldwide for the approval of leniolisib for pediatric patients with APDS, beginning in 2025.

Key Points: 
  • Pharming plans to include data from this 4–11-year-old trial in regulatory filings worldwide for the approval of leniolisib for pediatric patients with APDS, beginning in 2025.
  • Anurag Relan, MD, MPH, Chief Medical Officer of Pharming, commented:
    “This is the first clinical trial for younger pediatric patients with APDS, who have a significant unmet need for a disease modifying treatment.
  • I am pleased with the progress made in our Phase III pediatric clinical program evaluating leniolisib in children with APDS.
  • Eligible patients enrolled in either of the pediatric trials will continue to receive leniolisib for a year after the initial 12-week treatment period through an open-label extension trial.

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.

Pharming Group announces development plans for leniolisib for additional primary immunodeficiencies (PIDs)

Retrieved on: 
Wednesday, December 13, 2023
APDS, Food, FRCPA, Infection, NIAID, IND, Epidemiology, FDA, Prevalence, Immunodeficiency, Autoimmune lymphoproliferative syndrome, CTLA4, Pharmacokinetics, Primary immunodeficiency, Euronext Amsterdam, Protocol, Lymphocyte, PTEN, Gene, Bangladesh Technical Education Board, Face, NIH, Patient, Safety, FAS, PID, Pharmaceutical industry

Leiden, The Netherlands, December 13, 2023: Pharming Group N.V. (“Pharming” or “the Company”) (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) announces today the expansion of its rare disease pipeline with plans to develop leniolisib for additional primary immunodeficiencies (PIDs) beyond activated phosphoinositide 3-kinase delta syndrome (APDS).

Key Points: 
  • Leiden, The Netherlands, December 13, 2023: Pharming Group N.V. (“Pharming” or “the Company”) (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) announces today the expansion of its rare disease pipeline with plans to develop leniolisib for additional primary immunodeficiencies (PIDs) beyond activated phosphoinositide 3-kinase delta syndrome (APDS).
  • Pharming has engaged with the US Food and Drug Administration (FDA) and has received feedback on its plans to develop leniolisib for PID disorders with immune dysregulation.
  • The Phase 2 clinical trial will evaluate leniolisib in PIDs with immune dysregulation linked to PI3Kẟ signaling in lymphocytes, with similar clinical phenotypes to APDS.
  • Building upon the success of leniolisib for APDS, we believe that leniolisib will continue to have efficient uses in rebalancing immune dysregulation in PIDs.

Pharming announces first patient dosed in pediatric clinical trial for children aged 1 to 6 years for leniolisib

Retrieved on: 
Tuesday, November 21, 2023
Associate, APDS, Food, FDA, Hope, Ageing, Rheumatology, MPH, Diagnosis, Safety, Patient, University, Pharmaceutical industry

At sites in the U.S., Japan, and the EU, the single-arm, open-label, multinational clinical trial will evaluate the safety, tolerability, and efficacy of leniolisib in 15 children, one to six years of age, who have a confirmed APDS diagnosis.

Key Points: 
  • At sites in the U.S., Japan, and the EU, the single-arm, open-label, multinational clinical trial will evaluate the safety, tolerability, and efficacy of leniolisib in 15 children, one to six years of age, who have a confirmed APDS diagnosis.
  • These patients will receive a specific, pediatric granulated formulation of leniolisib.
  • The study’s primary efficacy endpoints and secondary endpoints mirror those used to evaluate the clinical outcomes in previous leniolisib Phase II/III APDS trials for patients aged 12 and older.
  • While standard supportive therapies can ease some of the progressive, immune-related symptoms that characterize APDS, leniolisib is designed to help prevent those symptoms from arising.

APDS Announces New Name: Orijin™

Retrieved on: 
Monday, November 13, 2023
APDS, Employment, Curriculum

NEW YORK, Nov. 13, 2023 (GLOBE NEWSWIRE) -- Today, American Prison Data Systems (APDS), announces a major renaming effort to become Orijin.

Key Points: 
  • NEW YORK, Nov. 13, 2023 (GLOBE NEWSWIRE) -- Today, American Prison Data Systems (APDS), announces a major renaming effort to become Orijin.
  • Like APDS, Orijin’s mission is to prepare every justice-impacted individual for sustainable employment.
  • “American Prison Data Systems (APDS) was founded in 2012.
  • Nearly ten years later, we’re still focused on ‘changing corrections for good’ but revitalizing our dedication and work with a new name called Orijin,” stated Orijin’s Co-Founder Arti Finn.

APDS Wins 2023 D2L Excellence Award for Innovative Education Programs

Retrieved on: 
Wednesday, July 12, 2023
Knowledge, International Design Excellence Awards, Employment, Freedom, Education, Master Plan, Curriculum, Creation, Chances, Fusion, Liberal education, Tablet, Recidivism, Student, Educational consultant, D2L, Conference, Engagement, APDS, Tutoring, Nursing

New York, July 12, 2023 (GLOBE NEWSWIRE) -- On July 13th, tomorrow, APDS will receive the D2L 2023 Excellence Award at the 2023 Fusion Conference in Anaheim .

Key Points: 
  • New York, July 12, 2023 (GLOBE NEWSWIRE) -- On July 13th, tomorrow, APDS will receive the D2L 2023 Excellence Award at the 2023 Fusion Conference in Anaheim .
  • D2L , a global learning technology company, each year honors innovative educators, trainers, and leaders in employee engagement who have provided exceptional learning experiences using D2L Brightspace.
  • APDS, an education technology and public-benefit corporation providing free education and career readiness courses to incarcerated individuals statewide, has won the Excellence Award for their courses "The Master Plan" and "Liberation Education."
  • D2L CEO John Baker expressed his pride in the innovative leaders using D2L Brightspace to reach and engage more learners.

Study Demonstrates the Effectiveness of Rare Medical Network Content Marketing Programs in Rare Disease Education

Retrieved on: 
Thursday, April 27, 2023
Learning, Database, Gastroenterology, Research, PLD, Immunology, HCP, RMN, NPI, Patient, Physician, SAN, Hematology, Knowledge, Diagnosis, Rare, News, Social media, APDS, Nursing, Pharmaceutical industry

SAN DIEGO and THE WOODLANDS, Texas, April 27, 2023 /PRNewswire/ -- The Rare Medical Network (RMN) announced the results of a study designed to measure the educational value of the RMN specialty-specific websites to healthcare professionals. The study, which measured the impact of engagement with RMN content on 4 of the 16 RMN specialty sites (gastroenterology, immunology, hematology, pulmonology), found that those HCPs who visited the sites had significantly higher understanding and knowledge about a relatively unknown rare disease, APDS, than HCPs in a control group.

Key Points: 
  • Each RMN site contains relevant current news, articles, videos, and interactive educational exercises each focused on rare disease information specific to the specialty.
  • "Rare disease education is a growing need in our industry," said Jeffrey Sweeney, Co-Founder of Rare Expertise, "and this study demonstrates our ability to provide a more effective way to target and educate healthcare professionals about the rare diseases that are most relevant to them."
  • By raising awareness and educating physicians about rare diseases, the Rare Medical Network is designed to accelerate the diagnosis and treatment of rare diseases.
  • The objectives of the Rare Medical Network:
    "We believe by raising the level of overall rare disease knowledge by specialists, we are making an important contribution to closing the rare disease knowledge gap," said Jack Davis, Co-Founder of Rare Expertise.

D2L Announces 2023 Excellence Awards Winners

Retrieved on: 
Monday, April 24, 2023
Knowledge, Learning, Lawrenceville, Communication, Deakin University, Student, LMS, Master Plan, Launch pad, Teaching, Southern Alberta Institute of Technology, Business, Liberal education, Orientation, Competence, Georgia Gwinnett College, ITE, School, APDS, York Technical College, Faculty, International Design Excellence Awards, S3, Credit, Technological pedagogical content knowledge, Tepper School of Business, Nursing, Tutoring, Award, D2L

D2L acknowledges innovators delivering diverse features and tools to make learning more personal, engaging, and meaningful

Key Points: 
  • D2L acknowledges innovators delivering diverse features and tools to make learning more personal, engaging, and meaningful
    TORONTO, April 24, 2023 /PRNewswire/ - D2L, a global learning technology company, today announced the recipients of D2L's 2023 Excellence Awards , which recognize educators, trainers, and leaders in employee engagement who have used D2L Brightspace to deliver learning experiences that are innovative, collaborative, or have made an extraordinary impact on learning outcomes.
  • "We are inspired by the ways that institutions around the world are using D2L Brightspace, and proud to celebrate innovative leaders pushing to reach and engage more learners," says John Baker, CEO of D2L.
  • "The 2023 Excellence Awards showcase creative thinking that delivers better outcomes in orientation and registration, justice-impacted learning, and professional development.
  • The Institute for Teaching Excellence (ITE) at York Technical College (Rock Hill, South Carolina) has implemented multiple D2L Brightspace integrations and new tools and expanded the use of D2L Brightspace to faculty and staff areas across campus.

The Massachusetts Department of Corrections and APDS Strike Landmark Education Partnership

Retrieved on: 
Wednesday, April 19, 2023
Partnership, Employment, Massachusetts Department of Correction, Education, Nation, Curriculum, Rehabilitation, MA, APDS, Nursing, Management

New York, NY, April 19, 2023 (GLOBE NEWSWIRE) -- New York, NY - Wednesday, April 19, 2023 - APDS , an education technology and public-benefit corporation, announced a landmark partnership with the Massachusetts Department of Correction (MADOC) to provide nearly 6,000 incarcerated individuals with day-one access to best-in-class career-readiness courses.

Key Points: 
  • New York, NY, April 19, 2023 (GLOBE NEWSWIRE) -- New York, NY - Wednesday, April 19, 2023 - APDS , an education technology and public-benefit corporation, announced a landmark partnership with the Massachusetts Department of Correction (MADOC) to provide nearly 6,000 incarcerated individuals with day-one access to best-in-class career-readiness courses.
  • The education and career courses began rolling out to every state corrections institution in July 2022 and are now 100% complete.
  • Leveraging APDS, Massachusetts is taking an individualized and scalable approach to delivering education, rehabilitation, and workforce plans for each incarcerated individual across the state’s correctional facilities.
  • \
    Education is a crucial aspect of turning corrections into true rehabilitation centers.