Zevra Therapeutics to Present Data Demonstrating Relevance of Swallow Domain in Niemann-Pick Disease Type C Clinical Severity Scale at 53rd Child Neurology Society Annual Meeting
Zevra Therapeutics (NASDAQ: ZVRA) announced the presentation of data validating the swallow domain's significance in the Niemann-Pick Disease Type C Clinical Severity Scale (NPCCSS) at the 53rd Child Neurology Society Annual Meeting. The study, presented by Dr. Elizabeth Berry-Kravis, involved 12 NPC and swallow experts evaluating scoring categories and instructions.
The findings confirmed that NPCCSS swallow response categories can be consistently interpreted, with score changes reflecting actual changes in patient swallow function. The revised scoring algorithm improves linearity by re-ranking dysphagia categories and creating a separate category for tube-feeding. The R4DNPCCSS endpoint, which includes this revised swallow domain along with ambulation, fine motor skills, and speech domains, showed a favorable treatment difference of -1.51 (P=0.0413) for arimoclomol compared to placebo in the Phase 2/3 study.
Zevra Therapeutics (NASDAQ: ZVRA) ha annunciato la presentazione dei dati che convalidano l'importanza del dominio della deglutizione nella Scala di Gravità Clinica della Malattia di Niemann-Pick Tipo C (NPCCSS) durante il 53° Congresso Annuale della Child Neurology Society. Lo studio, presentato dalla Dott.ssa Elizabeth Berry-Kravis, ha coinvolto 12 esperti in NPC e deglutizione che hanno valutato le categorie di punteggio e le istruzioni.
I risultati hanno confermato che le categorie di risposta alla deglutizione dell'NPCCSS possono essere interpretate in modo coerente, con le variazioni di punteggio che riflettono cambiamenti reali nella funzione di deglutizione dei pazienti. L'algoritmo di punteggio rivisto migliora la linearità riordinando le categorie di disfagia e creando una categoria separata per l'alimentazione tramite tubo. L'end point R4DNPCCSS, che include questo dominio della deglutizione rivisto insieme ai domini del cammino, delle abilità motorie fini e del linguaggio, ha mostrato una differenza favorevole nel trattamento di -1.51 (P=0.0413) per arimoclomolo rispetto al placebo nello studio di Fase 2/3.
Zevra Therapeutics (NASDAQ: ZVRA) anunció la presentación de datos que validan la importancia del dominio de la deglución en la Escala de Severidad Clínica de la Enfermedad de Niemann-Pick Tipo C (NPCCSS) durante la 53ª Reunión Anual de la Child Neurology Society. El estudio, presentado por la Dra. Elizabeth Berry-Kravis, involucró a 12 expertos en NPC y deglución que evaluaron las categorías de puntuación e instrucciones.
Los hallazgos confirmaron que las categorías de respuesta de deglución del NPCCSS pueden ser interpretadas de manera consistente, con cambios en la puntuación que reflejan cambios reales en la función de deglución del paciente. El algoritmo de puntuación revisado mejora la linealidad reorganizando las categorías de disfagia y creando una categoría separada para la alimentación por sonda. El endpoint R4DNPCCSS, que incluye este dominio de deglución revisado junto con los dominios de ambulación, habilidades motoras finas y del habla, mostró una diferencia favorable de tratamiento de -1.51 (P=0.0413) para arimoclomol en comparación con placebo en el estudio de Fase 2/3.
Zevra Therapeutics (NASDAQ: ZVRA)는 니만-픽병 C형 임상 중증도 척도 (NPCCSS)에서 삼키기 영역의 중요성을 검증하는 데이터 발표를 53회 아동신경학회 연례 회의에서 발표했습니다. 이 연구는 엘리자베스 베리-크라비스 박사가 발표했으며, 12명의 NPC 및 삼키기 전문가가 점수 범주와 지침을 평가했습니다.
연구 결과는 NPCCSS 삼키기 반응 범주가 일관되게 해석될 수 있으며, 점수 변동이 환자의 삼키기 기능의 실제 변화를 반영한다는 것을 확인했습니다. 수정된 점수 알고리즘은 삼킴장애 범주를 재정렬하고 관급 급식을 위한 별도의 범주를 생성하여 선형성을 향상시킵니다. 이 수정된 삼키기 영역과 보행, 세밀한 운동 기술, 언어 영역을 포함하는 R4DNPCCSS 종점은 아리모클로몰이 위약에 비해 -1.51 (P=0.0413)의 유리한 치료 차이를 나타냈습니다.
Zevra Therapeutics (NASDAQ: ZVRA) a annoncé la présentation de données validant l'importance du domaine de la déglutition dans l'Échelle de Sévérité Clinique de la Maladie de Niemann-Pick de Type C (NPCCSS) lors de la 53e Réunion Annuelle de la Child Neurology Society. L'étude, présentée par le Dr Elizabeth Berry-Kravis, a impliqué l'évaluation des catégories de score et des instructions par 12 experts en NPC et en déglutition.
Les résultats ont confirmé que les catégories de réponse de déglutition de l'NPCCSS peuvent être interprétées de manière cohérente, les changements de score reflétant des modifications réelles de la fonction de déglutition du patient. L'algorithme de score révisé améliore la linéarité en réorganisant les catégories de dysphagie et en créant une catégorie séparée pour l'alimentation par sonde. L'endpoint R4DNPCCSS, qui inclut ce domaine de déglutition révisé ainsi que les domaines de la marche, des compétences motrices fines et de la parole, a montré une différence de traitement favorable de -1,51 (P=0,0413) pour l'arimoclomole par rapport au placebo dans l'étude de Phase 2/3.
Zevra Therapeutics (NASDAQ: ZVRA) kündigte die Präsentation von Daten an, die die Bedeutung der Schluckdomäne in der Niemann-Pick-Krankheit Typ C Klinischen Schweregradskala (NPCCSS) auf der 53. Jahrestagung der Child Neurology Society validieren. Die Studie wurde von Dr. Elizabeth Berry-Kravis vorgestellt und umfasste die Bewertung von Punktkategorien und Anweisungen durch 12 NPC- und Schluckexperten.
Die Ergebnisse bestätigten, dass die Schluckantwortkategorien der NPCCSS konsistent interpretiert werden können, wobei Punktänderungen tatsächliche Veränderungen in der Schluckfunktion der Patienten widerspiegeln. Der überarbeitete Punktalgorithmus verbessert die Linearität, indem er die Dysphagiekategorien neu anordnet und eine separate Kategorie für die enterale Ernährung schafft. Der R4DNPCCSS-Endpunkt, der diese überarbeitete Schluckdomäne zusammen mit den Bereichen Gehen, feine motorische Fähigkeiten und Sprache umfasst, zeigte im Phase-2/3-Studium einen günstigen Behandlungsunterschied von -1,51 (P=0,0413) für Arimoclomol im Vergleich zu Placebo.
- None.
- None.
Data show that the NPCCSS swallow score reflects the patient’s level of dysfunction
Study indicates that a change in score reflects actual improvement or worsening in a patient’s swallowing function
CELEBRATION, Fla., Nov. 11, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on addressing unmet needs for the treatment of rare diseases, today announced that Elizabeth Berry-Kravis, M.D., Ph.D., Professor of Pediatrics, Neurological Sciences, and Biochemistry at Rush University Medical Center in Chicago, is presenting data demonstrating the relevance of the swallow domain as part of the Niemann-Pick disease Type C Clinical Severity Scale (NPCCSS) at the 53rd Child Neurology Society Annual Meeting, which takes place Nov. 11-14 in San Diego.
The poster presentation includes a reassessment of individual swallow domain scoring categories, rater scoring instructions, and evaluation of the score linearity based on a qualitative study in which an independent research organization interviewed 12 NPC and swallow experts. Findings showed that the descriptions of the NPCCSS swallow response categories can be clearly and consistently interpreted and that a change in score reflects actual improvement or worsening in a patient’s swallow function.
This study and outside expert feedback were the basis for Zevra’s revised scoring algorithm of the swallow domain. This rescored swallow domain imparts improved linearity by re-ranking the scoring categories for dysphagia by frequency regardless of food texture. In addition, supplemental tube-feeding was placed in its own scoring category. The composite score of this revised swallow domain and the three NPCCSS domains for ambulation, fine motor skills, and speech, formed the primary 4-domain R4DNPCCSS endpoint used to evaluate the effectiveness of arimoclomol (MIPLYFFA™) in the Phase 2/3 study (NCT02612129). The treatment difference based on the R4DNPCCSS score change from baseline to 12 months or last visit while on treatment was -1.51 (P=0.0413) in favor of arimoclomol when compared to placebo.
Presentation Details
| Poster Number: | 279 | |
| Title: | Qualitative Assessment of the Validity and Standardization of the Swallow Domain in the 5-Domain Niemann-Pick Disease Type C (NPC) Clinical Severity Scale (5DNPCCSS) | |
| Date/Time: | Tuesday, Nov. 12, 2024, 12:30 p.m. – 1:45 p.m. PT, and 5:30 p.m. – 7:00 p.m. PT | |
| Presenter: | Elizabeth Berry-Kravis, MD, Ph.D., Professor of Pediatrics, Neurological Sciences, and Biochemistry at Rush University Medical Center in Chicago | |
Members of Zevra’s team will be available at the meeting in San Diego; visit Zevra at its commercial booth (#515) and medical booth (#614).
About Niemann-Pick Disease Type C (NPC)
Niemann-Pick disease type C (NPC) is an ultra-rare, progressive, and neurodegenerative lysosomal storage disorder characterized by an inability of the body to transport cholesterol and other lipids within the cell, leading to an accumulation of these substances in various cell types, including neurons. The disease is caused by mutations in the NPC1 or NPC2 genes, which are responsible for making the NPC1 and NPC2 lysosomal proteins. Both children and adults can be affected by NPC with varying clinical presentations. Those living with NPC can lose independence due to physical and cognitive limitations, with key neurological impairments presenting in speech, cognition, swallowing, ambulation, and fine motor skills. Disease diagnosis can often take years, with disease progression being irreversible and often leading to early mortality.
About Zevra Therapeutics, Inc.
Zevra Therapeutics, Inc. is a commercial-stage rare disease company combining science, data, and patient needs to create transformational therapies for diseases with limited or no treatment options. Our mission is to bring life-changing therapeutics to people living with rare diseases. With unique, data-driven development and commercialization strategies, the Company is overcoming complex drug development challenges to make new therapies available to the rare disease community.
Expanded access programs are made available by Zevra Therapeutics, Inc. and its affiliates and are subject to the Company's Expanded Access Program (EAP) policy, as published on its website. Participation in these programs is subject to the laws and regulations of each jurisdiction under which each respective program is operated. Eligibility for participation in any such program is at the treating physician's discretion.
For more information, please visit www.zevra.com or follow us on X and LinkedIn.
About MIPLYFFA™ (arimoclomol)
MIPLYFFA (arimoclomol) increases the activation of the transcription factors EB (TFEB) and E3 (TFE3) resulting in the upregulation of coordinated lysosomal expression and regulation (CLEAR) genes. MIPLYFFA has also been shown to reduce unesterified cholesterol in the lysosomes of human NPC fibroblasts. The clinical significance of these findings is not fully understood. MIPLYFFA was granted Breakthrough Therapy designation, Rare Pediatric Disease designation, Orphan Drug designation, and Fast Track designation by the FDA for the treatment of NPC. MIPLYFFA was further granted Orphan Medicinal Product designation by the European Medicines Agency (EMA) for the treatment of NPC.
For Full Prescribing and Safety Information for MIPLYFFA, please visit www.MIPLYFFA.com.
Cautionary Note Concerning Forward-Looking Statements
This news release may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include all statements that do not relate solely to historical or current facts, including without limitation statements regarding the promise and potential impact of our preclinical or clinical trial data; the potential benefits of any of our products or product candidates for any specific disease or at any dosage; our strategic and product development objectives, including with respect to becoming a leading, commercially focused rare disease company; the presentation of data at conferences; and the timing of any of the foregoing. Forward-looking statements are based on information currently available to Zevra and its current plans or expectations. They are subject to several known and unknown uncertainties, risks, and other important factors that may cause our actual results, performance, or achievements to be materially different from any future results, performance, or achievements expressed or implied by the forward-looking statements. These and other important factors are described in detail in the “Risk Factors” section of Zevra’s Annual Report on Form 10-K for the year ended December 31, 2023, Zevra’s Quarterly Report on Form 10-Q for the three months ended June 30, 2024, and Zevra’s other filings with the Securities and Exchange Commission. While we may elect to update such forward-looking statements at some point in the future, except as required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Although we believe the expectations reflected in such forward-looking statements are reasonable, we cannot assure that such expectations will prove correct. These forward-looking statements should not be relied upon as representing our views as of any date after the date of this press release.
Zevra Contact
Nichol Ochsner
+1 (732) 754-2545
nochsner@zevra.com
Russo Partners Contact
David Schull
+1 (858) 717-2310
david.schull@russopartnersllc.com
FAQ
What were the key findings of Zevra's NPCCSS swallow domain study presented at the 2024 Child Neurology Society Meeting?
What was the treatment difference for arimoclomol in Zevra's Phase 2/3 study using the R4DNPCCSS endpoint?
When and where is Zevra Therapeutics (ZVRA) presenting their NPCCSS data?
