• Abstracts presented at ASH 2024 provide insight into the patient experience given the unpredictable nature of wAIHA, a rare autoantibody disease, and the uncertainty of current treatment approaches used to manage the condition
• There are no FDA-approved therapies indicated for wAIHA, and patients living with this condition need targeted treatment options with a proven safety and efficacy profile
• Johnson & Johnson is evaluating nipocalimab for the potential treatment of wAIHA in the Phase 2/3 ENERGY study, which is expected to read out in 2025

SAN DIEGO, Dec. 9, 2024 /PRNewswire/ -- Johnson & Johnson (NYSE: JNJ) announced today findings from several online abstracts and posters on patients living with warm autoimmune hemolytic anemia (wAIHA), a rare, life-threatening condition where autoantibodies lead to the premature destruction of red blood cells, at the 2024 American Society of Hematology (ASH) Annual Meeting. These findings underscore the significant disease burden for the estimated one in 8,000 people living with wAIHA, including the high unmet need for targeted therapies with proven safety and efficacy profiles and the associated impact the disease has on healthcare utilization.¹  

"Approximately 50,000 people in the U.S. are living with wAIHA and grappling with devastating physical symptoms such as debilitating fatigue, dizziness, shortness of breath, and jaundice and in severe cases, chest pain or loss of consciousness, often with profound implications to their mental health," said Karen Jones, Executive Director of the patient advocacy group wAIHA Warriors and an abstract co-author.*² "Our findings demonstrate the continued need for research into new treatment approaches for people living with this condition with the potential to maintain immune–function and reduce the need for invasive surgeries and repeat blood transfusions." 

Johnson & Johnson is collaborating with patient advocates and the scientific community to generate real-world evidence and harness the patient perspective to inform their clinical development process.³ Members of a global patient council shared their diagnosis and treatment journeys, the emotional and financial impacts of the disease, and their views about opportunities for improved management. These patients expressed high levels of anxiety related to the cyclical nature of the condition and lack of sustained disease control as well as dissatisfaction with current treatment management options.^3,4 All council members experienced side effects from at least one treatment prescribed to them, none of which are indicated specifically for the treatment of wAIHA.³ This was also demonstrated in a sentiment analysis, which looked at over 22,000 conversations among adults who self-identified as having wAIHA, revealing that the most common negative sentiment themes for rituximab, a common treatment approach for the condition, were lack of efficacy and side effects.⁵ 

Two of the accepted posters established that wAIHA is associated with significant long-term healthcare resource utilization – including ongoing need for emergency and inpatient care – as demonstrated through observational studies on the healthcare utilization of individuals living with wAIHA in the United States and Sweden.^6,7 These findings highlight the need to achieve greater disease control after initial wAIHA diagnosis.^6,7 

"Through observational and qualitative research in wAIHA, we are learning more about the unmet need for novel treatment approaches that address the underlying cause of disease and address the serious health consequences faced by the many people living with wAIHA," said Katie Abouzahr, M.D., Vice President, Autoantibody and Maternal Fetal Immunology Disease Area Leader, Johnson & Johnson Innovative Medicine. "We are excited about the potential of our ongoing clinical program for investigational nipocalimab in this condition and look forward to sharing the results of our Phase 2/3 study next year." 

ABOUT WARM AUTOIMMUNE HEMOLYTIC ANEMIA (wAIHA) 

Warm autoimmune hemolytic anemia (wAIHA) is a rare, life-threatening condition where autoantibodies lead to the premature destruction of red blood cells (RBCs), resulting in anemia.⁸ Approximately 1-3 new people per 100,000 are affected by wAIHA per year, and about 1 in 8,000 individuals are living with the condition.^1,8 This condition affects both women and men, and can affect people at any age with incidence increasing over the age of 50.^1,9 Additionally, people with wAIHA are at increased risk of other serious complications such as venous thrombotic events, acute renal failure, and infection.¹⁰

There are no Food and Drug Administration (FDA)-approved drugs indicated for wAIHA, and treatment typically consists of corticosteroids, broad immunosuppressants and B-cell directed therapies.⁶ With an unmet need for treatment in wAIHA, continued research for evidence-based potential therapies is critical.¹⁰

ABOUT NIPOCALIMAB

Nipocalimab is an investigational monoclonal antibody, designed to bind with high affinity to block FcRn and reduce levels of circulating immunoglobulin G (IgG) antibodies potentially without impact on other immune functions. This includes autoantibodies and alloantibodies that underlie multiple conditions across three key segments in the autoantibody space including Rare Autoantibody diseases, Maternal Fetal diseases mediated by maternal alloantibodies and Prevalent Rheumatology.^{11,12,13,14,15,16,17,18,19} Blockade of IgG binding to FcRn in the placenta is also believed to limit transplacental transfer of maternal alloantibodies to the fetus.^20,21

The U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) have granted several key designations to nipocalimab including:  

• U.S. FDA Fast Track designation in hemolytic disease of the fetus and newborn (HDFN) and warm autoimmune hemolytic anemia (wAIHA) in July 2019, gMG in December 2021 and fetal neonatal alloimmune thrombocytopenia (FNAIT) in March 2024  
• U.S. FDA Orphan drug status for wAIHA in December 2019, HDFN in June 2020, gMG in February 2021, chronic inflammatory demyelinating polyneuropathy (CIDP) in October 2021 and FNAIT in December 2023  
• U.S. FDA Breakthrough Therapy designation for HDFN in February 2024 and for Sjögren's disease in November 2024 
• EU EMA Orphan medicinal product designation for HDFN in October 2019 

ABOUT JOHNSON & JOHNSON

At Johnson & Johnson, we believe health is everything. Our strength in healthcare innovation empowers us to build a world where complex diseases are prevented, treated, and cured, where treatments are smarter and less invasive, and solutions are personal. Through our expertise in Innovative Medicine and MedTech, we are uniquely positioned to innovate across the full spectrum of healthcare solutions today to deliver the breakthroughs of tomorrow, and profoundly impact health for humanity.  

Learn more at https://www.jnj.com or at www.innovativemedicine.jnj.com.

Follow us at @JanssenUS and @JNJInnovMed.  

Janssen Research & Development, LLC, Janssen Biotech, Inc. and Janssen Global Services, LLC are Johnson & Johnson companies. 

Cautions Concerning Forward-Looking Statements

This press release contains "forward-looking statements" as defined in the Private Securities Litigation Reform Act of 1995 regarding product development and the potential benefits and treatment impact of nipocalimab. The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialize, actual results could vary materially from the expectations and projections of Janssen Research & Development, LLC, Janssen Biotech, Inc., Janssen Global Services, LLC and/or Johnson & Johnson. Risks and uncertainties include, but are not limited to: challenges and uncertainties inherent in product research and development, including the uncertainty of clinical success and of obtaining regulatory approvals; uncertainty of commercial success; manufacturing difficulties and delays; competition, including technological advances, new products and patents attained by competitors; challenges to patents; product efficacy or safety concerns resulting in product recalls or regulatory action; changes in behavior and spending patterns of purchasers of health care products and services; changes to applicable laws and regulations, including global health care reforms; and trends toward health care cost containment. A further list and descriptions of these risks, uncertainties and other factors can be found in Johnson & Johnson's Annual Report on Form 10-K for the fiscal year ended December 31, 2023, including in the sections captioned "Cautionary Note Regarding Forward-Looking Statements" and "Item 1A. Risk Factors," and in Johnson & Johnson's subsequent Quarterly Reports on Form 10-Q and other filings with the Securities and Exchange Commission. Copies of these filings are available online at www.sec.gov, www.jnj.com or on request from Johnson & Johnson. None of Janssen Research & Development, LLC, Janssen Biotech, Inc., Janssen Global Services, LLC nor Johnson & Johnson undertakes to update any forward-looking statement as a result of new information or future events or developments.

*Karen Jones, Executive Director of the patient advocacy group wAIHA Warriors and an abstract co-author has provided consulting, advisory, and speaking services to Johnson & Johnson; she has not been paid for any media work.

¹ Tranekær S, Hansen DL, Frederiksen H. Epidemiology of Secondary Warm Autoimmune Haemolytic Anaemia-A Systematic Review and Meta-Analysis. J Clin Med. 2021 Mar 17;10(6):1244. doi: 10.3390/jcm10061244. PMID: 33802848; PMCID: PMC8002719.
² Eaton WW et al. epidemiology of Autoimmune Disease in Denmark 2007. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2717015/. 
³ Güttinger et al. Insights on the Lived Experience of Warm Autoimmune Hemolytic Anemia (wAIHA) from an Ongoing Patient Council. Blood (2024) 144 (Supplement 1): 7693.https://doi.org/10.1182/blood-2024-205079.
⁴ Piatek, et al. Qualitative Examination of Treatment Experiences Among Individuals Living with Warm Autoimmune Hemolytic Anemia (wAIHA). Blood (2024) 144 (Supplement 1): 7721. https://doi.org/10.1182/blood-2024-208446.
⁵ Piatek, et al. Sentiment Analysis Applied to Digital Conversations Among Warm Autoimmune Hemolytic Anemia (wAIHA) Patients Receiving Rituximab and/or Blood Transfusion. American Society of Hematology 2024 Annual Meeting. December 2024.
⁶ Jackson et al. A Retrospective Database Analysis of Healthcare Resource Utilization in Patients with Warm Autoimmune Hemolytic Anemia in the United States. American Society of Hematology 2024 Annual Meeting. December 2024.
⁷ Kjellander et al.  Health Resource Utilization Among Patients with Warm Autoimmune Hemolytic Anemia in Sweden: A Retrospective Registry-Based Study. American Society of Hematology 2024 Annual Meeting. December 2024. 
⁸ National Organization for Rare Disorders, Warm autoimmune Hemolytic Anemia, July 30, 2024. https://rarediseases.org/rare-diseases/warm-autoimmune-hemolytic-anemia/.
⁹ Cherif, H., Cai, Q., Crivera, C., Leon, A., Rahman, I., Leval, A., Noel, W. and Kjellander, C. (2024), Overall Survival and Treatment Patterns Among Patients With Warm Autoimmune Hemolytic Anemia in Sweden: A Nationwide Population-based Study. Eur J Haematol. https://doi.org/10.1111/ejh.14311.
¹⁰ Fattizzo B, Barcellini W. New Therapies for the Treatment of Warm Autoimmune Hemolytic Anemia. Transfusion Medical Reviews, Vol. 36, Issue 4. October 2022 https://doi.org/10.1016/j.tmrv.2022.08.001.
¹¹ ClinicalTrials.gov Identifier: NCT04968912. Available at: https://clinicaltrials.gov/study/NCT04968912. Last accessed: December 2024.
¹² ClinicalTrials.gov Identifier: NCT04951622. Available at: https://clinicaltrials.gov/ct2/show/NCT04951622. Last accessed: December 2024.
¹³ ClinicalTrials.gov Identifier: NCT05327114. Available at: https://www.clinicaltrials.gov/study/NCT05327114. Last accessed: December 2024.
¹⁴ ClinicalTrials.gov Identifier: NCT04119050. Available at: https://clinicaltrials.gov/study/NCT04119050. Last accessed: December 2024.
¹⁵ ClinicalTrials.gov Identifier: NCT05379634. Available at: https://clinicaltrials.gov/study/NCT05379634. Last accessed: December 2024.
¹⁶ ClinicalTrials.gov Identifier: NCT05912517. Available at: https://www.clinicaltrials.gov/study/NCT05912517. Last accessed: December 2024.
¹⁷ ClinicalTrials.gov Identifier: NCT06028438. Available at: https://clinicaltrials.gov/study/NCT06028438. Last accessed: December 2024.
¹⁸ ClinicalTrials.gov Identifier: NCT04882878. Available at: https://clinicaltrials.gov/study/NCT04882878. Last accessed: December 2024.
¹⁹ ClinicalTrials.gov. NCT03842189. Available at: https://clinicaltrials.gov/ct2/show/NCT03842189. Last accessed: December 2024.
²⁰ Lobato G, Soncini CS. Relationship between obstetric history and Rh(D) alloimmunization severity. Arch Gynecol Obstet. 2008 Mar;277(3):245-8. DOI: 10.1007/s00404-007-0446-x. Last accessed: December 2024.
²¹ Roy S, Nanovskaya T, Patrikeeva S, et al. M281, an anti-FcRn antibody, inhibits IgG transfer in a human ex vivo placental perfusion model. Am J Obstet Gynecol. 2019;220(5):498 e491-498 e499.