Vivoryon Therapeutics N.V. Shares Highlights from Virtual R&D Update with KOL Speakers
Vivoryon Therapeutics N.V. held a virtual R&D update highlighting key developments for their drug varoglutamstat. The event featured presentations from experts discussing the drug's beneficial effects on kidney function demonstrated in two independent Phase 2 studies. Key findings showed that varoglutamstat improved kidney function (eGFR) with statistical significance, with over 70% of patients responding to treatment.
The company plans to initiate a Phase 2b study in patients with type 2 diabetes and CKD stages 3b and worse. The drug's unique positioning stems from its oral availability, novel mechanism of action targeting inflammation and fibrosis through QPCTL inhibition, and demonstrated long-term safety. Notably, Vivoryon has decided to discontinue varoglutamstat's investigation in Alzheimer's disease to focus resources on kidney disease development.
Vivoryon Therapeutics N.V. ha tenuto un aggiornamento virtuale sulla R&D evidenziando i principali sviluppi del loro farmaco varoglutamstat. L'evento ha visto presentazioni di esperti che hanno discusso gli effetti benefici del farmaco sulla funzione renale dimostrati in due studi indipendenti di fase 2. I risultati chiave hanno mostrato che varoglutamstat ha migliorato la funzione renale (eGFR) con significatività statistica, con oltre il 70% dei pazienti che ha risposto al trattamento.
L'azienda prevede di avviare uno studio di fase 2b in pazienti con diabete di tipo 2 e CKD stadi 3b e peggiori. La posizione unica del farmaco deriva dalla sua disponibilità orale, dal nuovo meccanismo d'azione che mira all'infiammazione e alla fibrosi attraverso l'inibizione di QPCTL, e dalla sicurezza a lungo termine dimostrata. È degno di nota che Vivoryon ha deciso di interrompere l'indagine su varoglutamstat nella malattia di Alzheimer per concentrare le risorse sullo sviluppo della malattia renale.
Vivoryon Therapeutics N.V. llevó a cabo una actualización virtual de I+D destacando los desarrollos clave de su medicamento varoglutamstat. El evento contó con presentaciones de expertos que discutieron los efectos beneficiosos del medicamento en la función renal demostrados en dos estudios independientes de fase 2. Los hallazgos clave mostraron que varoglutamstat mejoró la función renal (eGFR) con significación estadística, con más del 70% de los pacientes respondiendo al tratamiento.
La empresa planea iniciar un estudio de fase 2b en pacientes con diabetes tipo 2 y enfermedad renal crónica en estadios 3b y peores. La posición única del medicamento se debe a su disponibilidad oral, su nuevo mecanismo de acción que apunta a la inflamación y fibrosis a través de la inhibición de QPCTL, y la seguridad a largo plazo demostrada. Cabe destacar que Vivoryon ha decidido interrumpir la investigación de varoglutamstat en la enfermedad de Alzheimer para concentrar recursos en el desarrollo de enfermedades renales.
비보리온 테라퓨틱스 N.V.는 약물 바로글루탐스타트에 대한 주요 개발 사항을 강조하는 가상 R&D 업데이트를 개최했습니다. 이 행사에서는 두 개의 독립적인 2상 연구에서 입증된 약물의 신장 기능에 대한 유익한 효과를 논의하는 전문가들의 발표가 있었습니다. 주요 발견에 따르면, 바로글루탐스타트는 통계적으로 유의미하게 신장 기능(eGFR)을 개선하였으며, 70% 이상의 환자가 치료에 반응했습니다.
회사는 2형 당뇨병 및 CKD 3b 단계 이상의 환자를 대상으로 2b상 연구를 시작할 계획입니다. 이 약물의 독특한 위치는 경구 투여 가능성, 염증 및 섬유화를 목표로 하는 새로운 작용 기전(QPCTL 억제)을 통해 나타나며, 장기 안전성이 입증되었습니다. 특히, 비보리온은 알츠하이머병에서 바로글루탐스타트의 조사를 중단하고 신장 질환 개발에 자원을 집중하기로 결정했습니다.
Vivoryon Therapeutics N.V. a organisé une mise à jour virtuelle de R&D mettant en avant les développements clés de leur médicament varoglutamstat. L'événement a présenté des interventions d'experts discutant des effets bénéfiques du médicament sur la fonction rénale, comme le montrent deux études de phase 2 indépendantes. Les résultats clés ont montré que varoglutamstat améliorait la fonction rénale (eGFR) avec une signification statistique, plus de 70 % des patients ayant répondu au traitement.
L'entreprise prévoit de lancer une étude de phase 2b chez des patients atteints de diabète de type 2 et d'insuffisance rénale chronique aux stades 3b et plus. La position unique du médicament repose sur sa disponibilité orale, son nouveau mécanisme d'action ciblant l'inflammation et la fibrose par l'inhibition de QPCTL, ainsi que sur sa sécurité à long terme démontrée. Il est à noter que Vivoryon a décidé d'interrompre l'investigation de varoglutamstat dans la maladie d'Alzheimer pour concentrer les ressources sur le développement des maladies rénales.
Vivoryon Therapeutics N.V. hat ein virtuelles F&E-Update abgehalten, das wichtige Entwicklungen zu ihrem Medikament varoglutamstat hervorhob. Die Veranstaltung umfasste Präsentationen von Experten, die die vorteilhaften Wirkungen des Medikaments auf die Nierenfunktion, die in zwei unabhängigen Phase-2-Studien nachgewiesen wurden, diskutierten. Wichtige Erkenntnisse zeigten, dass varoglutamstat die Nierenfunktion (eGFR) statistisch signifikant verbesserte, wobei über 70 % der Patienten auf die Behandlung ansprachen.
Das Unternehmen plant, eine Phase-2b-Studie bei Patienten mit Typ-2-Diabetes und CKD im Stadium 3b und schlechter zu initiieren. Die einzigartige Positionierung des Medikaments ergibt sich aus seiner oralen Verfügbarkeit, dem neuartigen Wirkmechanismus, der auf Entzündung und Fibrose durch QPCTL-Inhibition abzielt, sowie der nachgewiesenen Langzeit-Sicherheit. Bemerkenswert ist, dass Vivoryon beschlossen hat, die Untersuchung von varoglutamstat bei Alzheimer zu beenden, um Ressourcen auf die Entwicklung von Nierenerkrankungen zu konzentrieren.
- Statistically significant improvement in eGFR over baseline in two Phase 2 studies
- High patient response rate (>70%) to treatment
- Strong meta-analysis results supporting Phase 2b study with lower patient numbers
- Demonstrated long-term safety data
- Discontinuation of Alzheimer's disease program
Insights
The R&D update reveals a strategic transformation for Vivoryon, marked by a decisive pivot from Alzheimer's disease to focus exclusively on kidney disorders. This repositioning is supported by compelling clinical evidence from two independent Phase 2 studies showing varoglutamstat's beneficial effects on kidney function.
The data presented carries exceptional weight for several reasons:
- A response rate exceeding 70% demonstrates remarkable consistency across patient populations, significantly reducing the risk of false positives and strengthening the drug's commercial potential
- The meta-analysis validation supports a smaller Phase 2b trial design, potentially accelerating the development timeline while reducing costs
- The novel mechanism targeting QPCTL addresses both inflammation and fibrosis, key drivers of kidney disease that have proven challenging to target effectively
The strategic focus on late-stage diabetic kidney disease (DKD) is particularly astute. Current treatments only reduce CKD progression risk by approximately
The introduction of VY2149, showing improved cellular uptake and pharmacokinetics, provides a valuable backup compound and potential next-generation asset, demonstrating pipeline depth beyond the lead program. The strong endorsement from key opinion leaders, particularly in study design optimization, significantly de-risks the clinical development pathway.
While discontinuing the Alzheimer's program may appear as a setback, it represents a pragmatic reallocation of resources toward a more promising therapeutic area with clearer clinical validation. This focus could accelerate time-to-market and improve the probability of regulatory success in the kidney disease space.
Vivoryon Therapeutics N.V. Shares Highlights from Virtual R&D Update with KOL Speakers
Halle (Saale) / Munich, Germany, February 19, 2025 – 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 key takeaways from its virtual R&D update with KOL (Key Opinion Leader) speakers held February 18, 2025. The event contextualized key data and statistical rigor supporting varoglutamstat’s beneficial effect on kidney function reported in two independent Phase 2 studies, provided an update on the Company’s clinical development plan for varoglutamstat in diabetic kidney disease (DKD), outlined varoglutamstat’s potential market positioning and provided further data on its new development compound, VY2149.
The event featured presentations from Tobias B. Huber, MD, Professor of Medicine, and Kevin Carroll, PhD, CEO, KJC Statistics, as well as from Vivoryon’s management.
“As we progress varoglutamstat through the next steps of clinical development in kidney disease, we are extremely encouraged to have the support from key experts in the field like Tobias and Kevin, whose counsel is instrumental in designing our clinical studies with the maximum chance of success in effectively addressing the great medical need in kidney disease, especially in our initial target population of stage 3b/4 DKD,” said Frank Weber, MD, CEO of Vivoryon. “With varoglutamstat, we are in the unique position of having substantial clinical evidence to support an entirely novel mechanism of action designed to overcome the persisting hurdles in the development of viable, safe and effective therapies to not only slow progression, but to stabilize or even improve kidney function.”
Highlights from Prof. Huber’s presentation include:
- Current standard of care only reduces risk of CKD progression by approx. 1/3, meaning a significant risk remains of disease progression or premature death in a growing and aging population
- To alleviate this burden for patients and healthcare providers, therapies are urgently needed that reduce or reverse risk of progression in CKD/DKD and rare kidney disorders
- Inhibition of QPCTL represents a potentially novel CKD target and varoglutamstat has reported a consistent efficacy signal and treatment effect in two independent Phase 2 studies in patients with diabetes at different timepoints, with both VIVIAD and VIVA-MIND showing a statistically significant improvement in eGFR over baseline
- A responder analysis confirmed that the consistent effect seen in both studies was not driven by a few outliners but that the majority of patients (>
70% ) responded to treatment - In an effort to further elucidate mechanistic details in underlying varoglutamstat activity on kidney function, human kidney organoids were created that will be tested in an in vitro system to investigate specific effects of varoglutamstat on renal tissue
Highlights from Dr. Carroll’s presentation include:
- The meta-analysis of VIVIAD and VIVA-MIND was conducted to provide the best overall assessment of efficacy of varoglutamstat and to statistically validate the homogeneity of outcomes
- The meta-analysis shows consistent results of high effect size and strongly supports viability of moving into a Phase 2b study in patients with diabetes with a comparatively low patient number, based on rigorous statistical planning and robust results
Highlights from presentations held by Vivoryon’s management include:
- Inflammation and fibrosis have long been known as key drivers of kidney disease, yet attempts to develop effective therapeutics selectively targeting key pathways have had limited success
- Inhibiting QPCTL, an enzyme that creates pro-inflammatory pE-versions of key inflammatory proteins, has the potential to halt the progressive course of kidney disease through its novel approach to tackle inflammation and fibrosis, making it a promising target
- Vivoryon has compiled robust evidence demonstrating inhibition of intracellular QPCTL decreases activity of pro-inflammatory cytokines and kidney fibrosis all the way from in vitro and in vivo data to human clinical results
- Varoglutamstat is uniquely positioned within the evolving kidney disease landscape through its one-of-a-kind combination of key characteristics: oral availability, novel MOA addressing key components of disease pathways, single agent activity and suitability for use in combination therapies, demonstrated beneficial long-term effect on eGFR, long-term safety data confirmed
- The initial target market for varoglutamstat in late-stage DKD represents an attractive patient opportunity with a potential for expansion to earlier stages of DKD / CKD as well as orphan diseases
- Future opportunities include new development compound VY2149 which has preclinically shown improved cellular uptake and pharmacokinetics
- Vivoryon plans to initiate a double-blind placebo-controlled Phase 2b study in patients with type 2 diabetes and CKD stages 3b and worse on top of standard of care
- In-line with focusing resources on advancing in kidney disease, Vivoryon has decided to discontinue investigation of varoglutamstat in Alzheimer's disease
- Overall, Vivoryon is poised to improve kidney health with varoglutamstat’s novel mechanism of action and breakthrough clinical study results
A replay of the webcast of the virtual R&D update will be available via the Presentations & Webcasts page in the Investor Relations section on the Company's website at www.vivoryon.com.
###
About Varoglutamstat
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 advanced development aiming to treat Alzheimer’s disease (AD), varoglutamstat has been investigated in a number of different clinical studies. Based on the known anti-inflammatory and anti-fibrotic activity of varoglutamstat, the protocol for the Phase 2b VIVIAD study in early AD included the investigation of kidney function (measured using eGFR) and measurement of biomarkers of kidney inflammation and fibrosis to explore the role of QPCT/L inhibition on kidney function. eGFR was also analyzed as a prospectively defined safety parameter in the VIVA-MIND Phase 2 study in the U.S.
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 Contacts
Vivoryon Therapeutics N.V.
Dr. Manuela Bader, Director IR & Communication
Email: IR@vivoryon.com
LifeSci Advisors
Sandya von der Weid
Tel: +41 78 680 05 38
Email: svonderweid@lifesciadvisors.com
Media Contact
Trophic Communications
Valeria Fisher or Verena Schossmann
Tel: +49 175 8041816 / +49 151 219 412 77
Email: vivoryon@trophic.eu
Attachment
FAQ
What were the key results of varoglutamstat's Phase 2 trials for kidney disease (VVY)?
Why did Vivoryon (VVY) discontinue varoglutamstat's Alzheimer's disease program?
What is the next development step for varoglutamstat (VVY) in kidney disease?
How does varoglutamstat (VVY) work in treating kidney disease?
