Warm autoimmune hemolytic anemia (wAIHA) research presented by Johnson & Johnson highlights profound disease burden and unmet need for targeted treatment options
Johnson & Johnson (NYSE: JNJ) presented research at ASH 2024 highlighting the significant burden of warm autoimmune hemolytic anemia (wAIHA), a rare condition affecting approximately 50,000 people in the U.S. The research emphasizes the lack of FDA-approved therapies and high unmet need for targeted treatments.
The findings reveal that wAIHA patients experience severe symptoms, including debilitating fatigue and shortness of breath, alongside high healthcare resource utilization. Patient council feedback indicated anxiety about the cyclical nature of the condition and dissatisfaction with current treatments. A sentiment analysis of 22,000 conversations showed negative experiences with rituximab, a common treatment approach.
The company is evaluating nipocalimab as a potential treatment in the Phase 2/3 ENERGY study, with results expected in 2025.
Johnson & Johnson (NYSE: JNJ) ha presentato una ricerca all'ASH 2024 che evidenzia il significativo onere dell'anemia emolitica autoimmune calda (wAIHA), una condizione rara che colpisce circa 50.000 persone negli Stati Uniti. La ricerca sottolinea la mancanza di terapie approvate dalla FDA e l'alto bisogno non soddisfatto di trattamenti mirati.
I risultati rivelano che i pazienti affetti da wAIHA sperimentano sintomi gravi, tra cui affaticamento debilitante e mancanza di respiro, oltre a un elevato utilizzo delle risorse sanitarie. I feedback del consiglio dei pazienti hanno indicato ansia riguardo alla natura ciclica della condizione e insoddisfazione per i trattamenti attuali. Un'analisi dei sentimenti di 22.000 conversazioni ha mostrato esperienze negative con il rituximab, un comune approccio terapeutico.
L'azienda sta valutando il nipocalimab come potenziale trattamento nello studio Phase 2/3 ENERGY, con risultati attesi nel 2025.
Johnson & Johnson (NYSE: JNJ) presentó una investigación en ASH 2024 que destaca la carga significativa de la anemia hemolítica autoinmune cálida (wAIHA), una condición rara que afecta a aproximadamente 50,000 personas en EE. UU. La investigación enfatiza la falta de terapias aprobadas por la FDA y la alta necesidad insatisfecha de tratamientos dirigidos.
Los hallazgos revelan que los pacientes con wAIHA experimentan síntomas severos, incluyendo fatiga debilitante y falta de aliento, junto con un alto uso de recursos de atención médica. Los comentarios del consejo de pacientes indicaron ansiedad sobre la naturaleza cíclica de la condición y descontento con los tratamientos actuales. Un análisis de sentimientos de 22,000 conversaciones mostró experiencias negativas con el rituximab, un enfoque de tratamiento común.
La empresa está evaluando el nipocalimab como un potencial tratamiento en el estudio Phase 2/3 ENERGY, con resultados esperados en 2025.
존슨 앤드 존슨 (NYSE: JNJ)은 ASH 2024에서 따뜻한 자가면역 용혈성 빈혈(wAIHA)의 상당한 부담을 강조하는 연구를 발표했습니다. 이 희귀 질환은 미국에서 약 50,000명에게 영향을 미치고 있습니다. 이 연구는 FDA 승인 치료제의 부족과 목표 치료에 대한 높은 unmet need를 강조합니다.
결과는 wAIHA 환자들이 심각한 증상, 즉 극심한 피로와 호흡 곤란을 경험하며, 높은 의료 자원 사용을 반영하고 있음을 보여줍니다. 환자 자문위원회의 피드백은 질병의 순환적 성격에 대한 불안과 현재 치료에 대한 불만을 나타냈습니다. 22,000건의 대화에 대한 감정 분석에서는 일반적인 치료 방법인 리툭시맙에 대한 부정적인 경험이 나타났습니다.
회사는 Phase 2/3 ENERGY 연구에서 nipocalimab을 잠재적 치료제로 평가하고 있으며, 결과는 2025년에 예상됩니다.
Johnson & Johnson (NYSE: JNJ) a présenté une recherche lors de l'ASH 2024 mettant en avant le poids significatif de l'anémie hémolytique auto-immune chaude (wAIHA), une condition rare touchant environ 50 000 personnes aux États-Unis. La recherche souligne le manque de thérapies approuvées par la FDA et le besoin insatisfait de traitements ciblés.
Les résultats révèlent que les patients atteints de wAIHA ressentent des symptômes sévères, y compris une fatigue débilitante et un essoufflement, ainsi qu'une forte utilisation des ressources de santé. Les retours du conseil des patients ont indiqué une anxiété vis-à-vis de la nature cyclique de la condition et une insatisfaction face aux traitements actuels. Une analyse des sentiments de 22 000 conversations a montré des expériences négatives avec le rituximab, une approche thérapeutique courante.
L'entreprise évalue le nipocalimab comme un traitement potentiel dans l'étude Phase 2/3 ENERGY, avec des résultats attendus en 2025.
Johnson & Johnson (NYSE: JNJ) präsentierte eine Forschung auf der ASH 2024, die die erhebliche Belastung durch die warme autoimmune hämolytische Anämie (wAIHA) hervorhebt, eine seltene Erkrankung, die etwa 50.000 Menschen in den USA betrifft. Die Forschung betont den Mangel an von der FDA zugelassenen Therapien und den hohen nicht gedeckten Bedarf an gezielten Behandlungen.
Die Ergebnisse zeigen, dass Patienten mit wAIHA schwere Symptome wie lähmende Müdigkeit und Atemnot sowie eine hohe Inanspruchnahme von Gesundheitsressourcen erleben. Feedback des Patientenrats deutete auf Ängste wegen der zyklischen Natur der Erkrankung und Unzufriedenheit mit den aktuellen Behandlungen hin. Eine Sentiment-Analyse von 22.000 Gesprächen zeigte negative Erfahrungen mit Rituximab, einem gängigen Therapieansatz.
Das Unternehmen bewertet Nipocalimab als potenzielle Behandlung in der Phase-2/3-Studie ENERGY, mit Ergebnissen, die 2025 erwartet werden.
- Company advancing Phase 2/3 ENERGY study for nipocalimab as potential wAIHA treatment
- None.
Insights
This research presentation at ASH 2024 highlighting warm autoimmune hemolytic anemia (wAIHA) reveals significant insights into an underserved rare disease market. The key findings demonstrate a substantial unmet medical need, with approximately 50,000 patients in the U.S. lacking FDA-approved treatment options. The research shows high healthcare resource utilization and patient dissatisfaction with current treatments, particularly rituximab.
Johnson & Johnson's nipocalimab, currently in Phase 2/3 ENERGY study with results expected in 2025, represents a potential first-to-market opportunity in wAIHA. If successful, this could establish J&J as a leader in this rare disease space. The comprehensive approach, including real-world evidence and patient advocacy collaboration, strengthens their market positioning and potential for regulatory success.
From a market perspective, this research positions J&J strategically in the rare disease therapeutics sector. With 1 in 8,000 people affected by wAIHA and no FDA-approved treatments, this represents a significant commercial opportunity. Rare disease drugs typically command premium pricing and given the demonstrated high healthcare utilization, there's strong potential for favorable pricing and reimbursement.
The research highlighting patient dissatisfaction with current treatments and high healthcare resource utilization strengthens the market entry case for nipocalimab. The 2025 readout timeline puts J&J in a position to potentially capture first-mover advantage in this untapped market, which could translate to substantial revenue potential despite the relatively small patient population.
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
"Approximately 50,000 people in the
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
"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. 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 2024U.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 2023U.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.
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2 Eaton WW et al. epidemiology of Autoimmune Disease in
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FAQ
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