Vivoryon Therapeutics N.V. Presents Topline Phase 2 Data from VIVA-MIND Strongly Supporting Varoglutamstat’s Potential to Improve Kidney Function
Vivoryon Therapeutics announced topline Phase 2 data from the VIVA-MIND study of varoglutamstat in early Alzheimer's disease. The study showed a statistically significant improvement in kidney function, with patients treated with varoglutamstat 600mg BID showing an average improvement of >4mL/min/1.73m2 in estimated glomerular filtration rate (eGFR) versus placebo (p<0.001).
However, the study did not meet its primary and key secondary endpoints for Alzheimer's disease treatment, aligning with previous VIVIAD study results. The drug maintained a favorable safety profile with no new safety signals detected across over 400 participants in Phase 1 and 2 studies. The company plans to advance into a Phase 2 study in diabetic kidney disease, subject to additional funding/partnership.
Vivoryon Therapeutics ha annunciato i dati preliminari della Fase 2 dello studio VIVA-MIND riguardante il varoglutamstat nell'Alzheimer in fase iniziale. Lo studio ha evidenziato un improvement significativo nella funzione renale, con i pazienti trattati con varoglutamstat 600mg BID che mostrano un miglioramento medio >4mL/min/1.73m2 nella velocità di filtrazione glomerulare stimata (eGFR) rispetto al placebo (p<0.001).
Tuttavia, lo studio non ha raggiunto i suoi endpoint primari e secondari chiave per il trattamento dell'Alzheimer, in linea con i risultati precedenti dello studio VIVIAD. Il farmaco ha mantenuto un profilo di sicurezza favorevole senza nuovi segnali di sicurezza riscontrati in oltre 400 partecipanti negli studi di Fase 1 e 2. L'azienda pianifica di avanzare verso uno studio di Fase 2 nella malattia renale diabetica, soggetto a finanziamenti/partnership aggiuntivi.
Vivoryon Therapeutics anunció los datos preliminares de la Fase 2 del estudio VIVA-MIND sobre el varoglutamstat en la enfermedad de Alzheimer en etapa temprana. El estudio mostró una mejora estadísticamente significativa en la función renal, con pacientes tratados con varoglutamstat 600mg BID mostrando una mejora media de >4mL/min/1.73m2 en la tasa de filtración glomerular estimada (eGFR) en comparación con el placebo (p<0.001).
No obstante, el estudio no cumplió con sus objetivos primarios y secundarios clave para el tratamiento del Alzheimer, alineándose con los resultados previos del estudio VIVIAD. El fármaco mantuvo un perfil de seguridad favorable sin señales de seguridad nuevas detectadas en más de 400 participantes en los estudios de Fase 1 y 2. La empresa planea avanzar a un estudio de Fase 2 en enfermedad renal diabética, sujeto a financiamiento/socios adicionales.
Vivoryon Therapeutics는 초기 알츠하이머 병에 대한 varoglutamstat의 VIVA-MIND 연구의 2상 데이터를 발표했습니다. 이 연구는 신장 기능의 통계적으로 유의미한 개선을 보여주었으며, varoglutamstat 600mg BID로 치료받은 환자들이 위약 대비 추정 사구체 여과율(eGFR)에서 평균 >4mL/min/1.73m2의 개선을 보였습니다 (p<0.001).
그러나 이 연구는 알츠하이머 병 치료를 위한 주요 및 핵심 2차 목표를 달성하지 못했으며, 이는 이전 VIVIAD 연구 결과와 일치합니다. 이 약물은 1상 및 2상 연구에서 400명 이상의 참가자에서 새로운 안전 신호가 탐지되지 않으면서 우호적인 안전성을 유지했습니다. 회사는 추가 자금/파트너십을 조건으로 당뇨병성 신장 질환에 대한 2상 연구로 나아갈 계획입니다.
Vivoryon Therapeutics a annoncé les données préliminaires de la phase 2 de l'étude VIVA-MIND sur le varoglutamstat dans l'Alzheimer précoce. L'étude a montré une amélioration statistiquement significative de la fonction rénale, avec des patients traités par varoglutamstat 600mg BID montrant une amélioration moyenne de >4mL/min/1.73m2 du taux de filtration glomérulaire estimé (eGFR) par rapport au placebo (p<0.001).
Cependant, l'étude n'a pas atteint ses objectifs principaux et secondaires clés pour le traitement de la maladie d'Alzheimer, ce qui est en accord avec les résultats précédents de l'étude VIVIAD. Le médicament a conservé un profil de sécurité favorable sans nouveaux signaux de sécurité détectés chez plus de 400 participants dans les études de phase 1 et 2. L'entreprise prévoit d'avancer vers une étude de phase 2 dans la maladie rénale diabétique, sous réserve d'un financement ou d'un partenariat supplémentaire.
Vivoryon Therapeutics gab die vorläufigen Phase-2-Daten der VIVA-MIND-Studie zu Varoglutamstat bei frühzeitiger Alzheimer-Krankheit bekannt. Die Studie zeigte eine statistisch signifikante Verbesserung der Nierenfunktion, wobei Patienten, die mit Varoglutamstat 600 mg BID behandelt wurden, eine durchschnittliche Verbesserung von >4mL/min/1.73m2 bei der geschätzten glomerulären Filtrationsrate (eGFR) im Vergleich zu Placebo zeigten (p<0.001).
Die Studie erreichte jedoch nicht die primären und wesentlichen sekundären Endpunkte zur Behandlung der Alzheimer-Krankheit, was mit früheren Ergebnissen der VIVIAD-Studie übereinstimmt. Das Medikament wies ein günstiges Sicherheitsprofil auf, ohne dass neue Sicherheitszeichen in über 400 Teilnehmern der Phase-1- und 2-Studien festgestellt wurden. Das Unternehmen plant, in eine Phase-2-Studie zur diabetischen Nierenerkrankung überzugehen, vorbehaltlich zusätzlicher Finanzierung/Partnerschaften.
- Statistically significant improvement in kidney function (eGFR >4mL/min/1.73m2) versus placebo
- Favorable safety profile maintained with no new safety signals
- Consistent positive kidney function results across two independent Phase 2 studies
- Failed to meet primary and secondary endpoints for Alzheimer's disease treatment
- Early discontinuation of the VIVA-MIND study
- Additional funding/partnership required for planned Phase 2 kidney disease study
Insights
The topline Phase 2 VIVA-MIND data presents a significant finding in kidney function improvement, with varoglutamstat showing a statistically significant increase in eGFR of >4mL/min/1.73m2 compared to placebo (p<0.001). This replication of kidney benefits across two independent Phase 2 studies (VIVA-MIND and VIVIAD) strengthens the evidence for varoglutamstat's potential in treating diabetic kidney disease.
However, the drug failed to demonstrate efficacy in its primary Alzheimer's disease endpoints, showing no meaningful differences in CDR-SB and key secondary measures like CFC2 and ADAS-Cog 13. The safety profile remains favorable, with no new concerns identified at the 600mg BID dose across over 400 participants.
This pivot from Alzheimer's to kidney disease represents a potential strategic shift in the drug's development pathway, though future studies are contingent on additional funding or partnerships.
This clinical trial outcome presents a mixed financial picture for Vivoryon. While the failure in Alzheimer's disease endpoints is a significant setback for the company's primary development program, the unexpected positive kidney function data opens a potentially valuable alternative development path in diabetic kidney disease (DKD).
The global DKD market represents a substantial commercial opportunity, though the requirement for additional funding or partnerships to advance the Phase 2 DKD study suggests near-term capital constraints. The company's ability to pivot and leverage this unexpected benefit could be important for maintaining shareholder value, but will likely require significant additional capital investment and time to validate in a dedicated kidney disease trial.
The safety profile data across 400+ patients provides a valuable de-risking element for future development, potentially making the asset more attractive to potential partners.
Vivoryon Therapeutics N.V. Presents Topline Phase 2 Data from
VIVA-MIND Strongly Supporting Varoglutamstat’s Potential to
Improve Kidney Function
- VIVA-MIND Phase 2 data confirm results of varoglutamstat’s benefit on eGFR in VIVIAD; Company now has two independent double-blind placebo-controlled Phase 2 studies demonstrating a clinically meaningful treatment effect on kidney function
- Topline analysis of kidney function data in VIVA-MIND shows a statistically significant and clinically meaningful improvement of eGFR; average improvement of >4mL/min/1.73m2 with varoglutamstat versus placebo across all visits (weeks 4–72) and all patients (p<0.001)
- VIVA-MIND, a Phase 2 study in early Alzheimer’s disease (AD), was discontinued early and did not meet its primary and key secondary endpoints in early AD, in line with the previously reported results from VIVIAD
- Varoglutamstat continues to demonstrate a favorable safety and tolerability profile in VIVA-MIND with no new safety signals detected and a total of over 400 participants treated with varoglutamstat in Phase 1 and Phase 2 studies to date
- Further detail on the topline results from VIVA-MIND to be shared in the Company’s Q3 financial results webcast to be held on December 10, 2024, at 3:00 pm CET
Halle (Saale) / Munich, Germany, December 9, 2024 – Vivoryon Therapeutics N.V. (Euronext Amsterdam: VVY; NL00150002Q7) (Vivoryon), a clinical stage company focused on the discovery and development of small molecule medicines to modulate the activity and stability of pathologically altered proteins, today announced positive Phase 2 topline data from its U.S. VIVA-MIND study of varoglutamstat (PQ912), an investigational oral glutaminyl cyclase (QPCT/L) inhibitor, in early AD. Analysis of kidney function data revealed a statistically significant improvement of >4mL/min/1.73m2 in the estimated glomerular filtration rate (eGFR) in patients treated with varoglutamstat 600mg BID versus placebo across all visits and all patients (weighted average weeks 4-72; p<0.001; total treated n=109; varoglutamstat n=52, placebo n=57). Analysis of eGFR was prospectively defined as a safety parameter in VIVA-MIND. These data reinforce the previously reported beneficial effect of varoglutamstat on eGFR in the completed Phase 2b VIVIAD study and support the Company’s development strategy to advance into a Phase 2 study in diabetic kidney disease (DKD)1.
“In clinical development it is highly relevant to replicate results in two independent high-quality studies. The topline VIVA-MIND data showed a consistent, statistically significant and clinically meaningful improvement of kidney function, as measured by eGFR, in patients treated with varoglutamstat versus placebo and clearly corroborate previously reported results from VIVIAD,” said Frank Weber, M.D., CEO of Vivoryon.
Analysis of VIVA-MIND data in AD showed no clinically meaningful and no statistically significant differences between varoglutamstat 600mg BID and placebo for the primary endpoint of CDR-SB, and key secondary endpoints including CFC2, ADAS-Cog 13, in patients treated with varoglutamstat compared to placebo, in line with the previously reported results in AD from VIVIAD.
Frank Weber continued, “As for the results in AD, these confirm the data seen in VIVIAD and are therefore not unexpected. While we would have wished for a different outcome of this study, we would like to thank all patients, their families and caregivers, the Alzheimer's Disease Cooperative Study (ADCS) at the University of California San Diego School of Medicine and clinical study investigators as well as the U.S. National Institutes of Health – enabling the VIVA-MIND study through a grant – for their commitment to the study and the varoglutamstat program.”
VIVA-MIND met its safety endpoints with varoglutamstat being generally well tolerated with no new safety signals observed at the 600mg BID dose.
All topline VIVA-MIND results are preliminary and may be subject to change based on additional analysis and quality checks, however, the overall interpretation of the results is not expected to change significantly.
The Company will provide further detail on the topline VIVA-MIND data on December 10, 2024, in its Q3 financial results webcast.
Q3 Financial Results Conference Call and Webcast
Vivoryon will host a conference call and webcast on December 10, 2024, at 3:00 pm CET (9:00 am ET). A Q&A session will follow the presentation of the third quarter 2024 results and operational progress updates.
A live webcast and slides will be made available at: https://www.vivoryon.com/news-and-events/presentations-webcasts/
To join the conference call via phone, participants may pre-register and will receive dedicated dial-in details to easily and quickly access the call via the following website: https://register.vevent.com/register/BIf0be17873ad0409b83edd4eedbe3b7ac
It is suggested participants dial into the conference call 15 minutes prior to the scheduled start time to avoid any delays in attendance.
Approximately one day after the call, a slide-synchronized audio replay of the conference will be available on: https://www.vivoryon.com/news-and-events/presentations-webcasts/
Definitions and notes: 1.The timing and execution of the planned Phase 2 study is subject to additional funding / partnership. CDR-SB: Clinical Dementia Rating Sum of Boxes; CFC2: Cognitive-Functional Composite 2, ADAS-Cog-13: Alzheimer's Disease Assessment Scale cognitive subscale.
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About VIVA-MIND
VIVA-MIND is a Phase 2 study that was conducted in the U.S., coordinated by the Alzheimer's Disease Cooperative Study (ADCS) at the University of California San Diego (UCSD) School of Medicine and supported by the National Institute on Aging (NIA), part of the National Institutes of Health (NIH) with a
Varoglutamstat in Kidney Disease
Varoglutamstat (PQ912) is a proprietary, potent and selective inhibitor of human glutaminyl cyclases QPCT and QPCTL with therapeutic potential in indications including inflammatory and fibrotic diseases, neurodegenerative diseases, cancer and others. Initially advancing development aiming to treat Alzheimer’s disease (AD), varoglutamstat has been investigated in a number of different clinical studies, all of which have consistently demonstrated a favorable safety and tolerability profile both in healthy volunteers and patients with AD. Based on the known anti-inflammatory activity of varoglutamstat, the protocol for the Phase 2 VIVIAD study in AD, which was completed in the first half of 2024, included the investigation of kidney function and measurement of biomarkers of kidney inflammation and fibrosis to explore the role of QPCT/L inhibition on kidney function. Although patients in VIVIAD were selected for their AD status and not for their kidney function level, many of them had reduced kidney function due to age and/or comorbidities. Analysis showed a statistically significant benefit of varoglutamstat on a prospectively defined key kidney function endpoint (eGFR) and a significant reduction of the pro-inflammatory cytokine pE-CCL2. A substantially higher treatment benefit of varoglutamstat on eGFR was observed in a post-hoc diabetes subgroup, triggering plans to advance varoglutamstat into Phase 2 study in DKD, which is currently in planning.
About Vivoryon Therapeutics N.V.
Vivoryon is a clinical stage biotechnology company focused on developing innovative small molecule-based medicines. Driven by its passion for ground-breaking science and innovation, the Company strives to change the lives of patients in need suffering from severe diseases. The Company leverages its in-depth expertise in understanding post-translational modifications to develop medicines that modulate the activity and stability of proteins which are altered in disease settings. The Company has established a pipeline of orally available small molecule inhibitors for various indications including Alzheimer’s disease, inflammatory and fibrotic disorders, including of the kidney, and cancer. www.vivoryon.com.
Vivoryon Forward Looking Statements
This press release includes forward-looking statements, including, without limitation, those regarding the business strategy, management plans and objectives for future operations of Vivoryon Therapeutics N.V. (the “Company”), estimates and projections with respect to the market for the Company’s products and forecasts and statements as to when the Company’s products may be available. Words such as “anticipate,” “believe,” “estimate,” “expect,” “forecast,” “intend,” “may,” “plan,” “project,” “predict,” “should” and “will” and similar expressions as they relate to the Company are intended to identify such forward-looking statements. These forward-looking statements are not guarantees of future performance; rather they are based on the Management’s current expectations and assumptions about future events and trends, the economy and other future conditions. The forward-looking statements involve a number of known and unknown risks and uncertainties. These risks and uncertainties and other factors could materially adversely affect the outcome and financial effects of the plans and events described herein. The Company’s results of operations, cash needs, financial condition, liquidity, prospects, future transactions, strategies or events may differ materially from those expressed or implied in such forward-looking statements and from expectations. As a result, no undue reliance should be placed on such forward-looking statements. This press release does not contain risk factors. Certain risk factors that may affect the Company’s future financial results are discussed in the published annual financial statements of the Company. This press release, including any forward-looking statements, speaks only as of the date of this press release. The Company does not assume any obligation to update any information or forward-looking statements contained herein, save for any information required to be disclosed by law.
For more information, please contact:
Investor Contact
Vivoryon Therapeutics N.V.
Dr. Manuela Bader, Director IR & Communication
Email: IR@vivoryon.com
Media Contact
Trophic Communications
Valeria Fisher or Verena Schossmann
Tel: +49 175 8041816 / +49 151 219 412 77
Email: vivoryon@trophic.eu
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