nipocalimab Archives - Medical Design Sourcing

Rare diseases, immense needs: J&J’s mission to change the landscape

Rare diseases may seem niche, but their impact is far from small. An estimated 7,000 rare diseases exist, collectively affecting a staggering 300 million people worldwide. This immense burden of disease, coupled with a profound lack of treatment options, underscores the urgent need for innovation. “Actually, the total burden of disease and unmet medical need [for rare disease] is really high,” emphasizes Dr. Katie Abouzahr, vice president, autoantibody portfolio and maternal fetal disease area leader at Johnson and Johnson Innovative Medicine. In recognition of Rare Disease Day on February 29 in 2024, we spoke with Abouzahr to explore how Johnson & Johnson Innovative Medicine aims to tackle these challenges.

The profound scarcity of treatments for the thousands of known rare diseases drives Johnson & Johnson’s commitment to this area. Abouzahr highlights that many of these diseases still lack advanced therapies, emphasizing the “incred…

A J&J veteran highlights listening to address overlooked maternal-fetal needs

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With nearly 20 years of experience at Johnson & Johnson, Cynthia Accuosti Jones now spearheads the company’s pioneering maternal-fetal medicine group tackling unmet needs in high-risk pregnancies. As vice president of global commercial strategy for autoantibody and maternal fetal health, she spotlights listening as the driver for patient impact. “You can’t have empathy if you don’t listen to people and it’s incredible how much people just need to be listened to,” Jones emphasized, underscoring the importance of addressing the often-neglected area of maternal health.

Jones’ longtime interest in healthcare stemmed from a desire to help people. She considered nursing but ultimately pursued finance and international business at Georgetown, building on childhood analytical skills tracking her father’s stocks. That experience piqued her interest in financial markets and data analysis. She…

Nipocalimab shows promise in RA subgroups in phase 2a IRIS-RA study

Johnson & Johnson’s nipocalimab, which works by targeting the neonatal Fc receptor (FcRn), has the potential to treat an array of autoimmune conditions. But the antibody recently hit a snag in the first-ever clinical study of an FcRn inhibitor in rheumatoid arthritis (RA), missing its primary endpoint. The development has sparked debate within the rheumatology community. “Now, there are some highly respected physicians who’ve said, ‘This is a failed trial,’” said Dr. Peter Taylor, the Norman Collisson Professor of Musculoskeletal Sciences at the University of Oxford. “I think that’s an incorrect interpretation.”

Johnson & Johnson is also pursuing a host of other autoimmune indications for nipocalimab. The FDA has granted it Fast Track designation for hemolytic disease of the fetus and newborn (HDFN) and orphan drug status for HDFN while EMA granted it orphan medicinal product designation for HDFN.

Breaking down the data from IRIS-RA

Johnson …

Nipocalimab gets positive results in Phase 2 HDFN clinical trial

Johnson & Johnson subsidiary Janssen (NYSE:JNJ) has revealed positive results from the proof-of-concept Phase 2 UNITY clinical trial of nipocalimab for pregnant adults with a high risk of developing severe hemolytic disease of the fetus and newborn (HDFN).

HDFN is a rare condition affecting between 3 and 80 out of 100,000 newborns or fetuses each year. The serious autoimmune disease can lead to life-threatening anemia in the fetus.

The UNITY trial met its primary endpoint with most pregnant patients who received nipocalimab giving birth to a live baby at or after 32 weeks of gestation, without the need for intrauterine transfusions.

The treatment was administered as weekly intravenous infusions to 14 participants and was well-tolerated over a 20-week period.

FDA granted nipocalimab Fast Track designation in July 2019 and orphan drug status in June 2020.

Janssen acquired nipocalimab as part of its $6.5 billion acquisition of Momenta Phar…

Why Janssen’s nipocalimab could represent a ‘pipeline in a pathway’

At the heart of Johnson & Johnson’s (NYSE:JNJ) $6.5 billion acquisition of Momenta Pharmaceuticals in 2020 is a portfolio of drug candidates for immune-mediated diseases.

One of the promising compounds in the acquisition is nipocalimab, which Janssen sees as a potential “pipeline in a pathway.” Nipocalimab holds promise for an array of diseases, ranging from rheumatoid arthritis and lupus and rare diseases such as myasthenia gravis. In all, Janssen is testing the drug in ten rare diseases as well as in rheumatoid arthritis and systemic lupus erythematosus.

When taken as a whole, rare diseases are not altogether rare, said Dr. Neely Mozaffarian, vice president and autoantibody pathway area leader at Janssen Immunology.

According to an analysis in the European Journal of Human Genetics, anywhere from 3.5% to 5.9% of the world’s population suffers from a rare disease, affecting 263–446 million people globally.

Rare autoant…