Vertex Provides Updates on Multiple Kidney Programs at the American Society of Nephrology (ASN) Annual Kidney Week Congress
Vertex Pharmaceuticals shared updates on multiple kidney disease programs at ASN Kidney Week Congress. New data shows povetacicept achieved 66% mean UPCR reduction at 48 weeks in IgA nephropathy patients, with 63% reaching clinical remission. In primary membranous nephropathy, povetacicept demonstrated 62% mean UPCR reduction at 24 weeks. The company launched the global Phase 3 RAINIER trial for IgA nephropathy and continues enrollment in Phase 3 AMPLITUDE trial of inaxaplin for APOL1-mediated kidney disease. Both treatments were well-tolerated with mostly mild to moderate adverse events.
Vertex Pharmaceuticals ha condiviso aggiornamenti su diversi programmi per malattie renali durante il Congresso ASN Kidney Week. Nuovi dati mostrano che povetacicept ha raggiunto una riduzione media dell'UPCR del 66% a 48 settimane nei pazienti con nefropatia da IgA, con il 63% che ha raggiunto la remissione clinica. Nella nefropatia membranosa primaria, povetacicept ha dimostrato una riduzione media dell'UPCR del 62% a 24 settimane. L'azienda ha avviato la fase globale del trial RAINIER di Fase 3 per la nefropatia da IgA e continua l'arruolamento nel trial di Fase 3 AMPLITUDE di inaxaplin per la malattia renale mediata da APOL1. Entrambi i trattamenti sono stati ben tollerati, con per lo più eventi avversi lievi o moderati.
Vertex Pharmaceuticals compartió actualizaciones sobre múltiples programas de enfermedades renales en el Congreso ASN Kidney Week. Nuevos datos muestran que povetacicept logró una reducción media del 66% en la UPCR a las 48 semanas en pacientes con nefropatía por IgA, con un 63% alcanzando remisión clínica. En la nefropatía membranosa primaria, povetacicept demostró una reducción media del 62% en la UPCR a las 24 semanas. La compañía lanzó el ensayo global de Fase 3 RAINIER para la nefropatía por IgA y continúa con el reclutamiento en el ensayo de Fase 3 AMPLITUDE de inaxaplin para la enfermedad renal mediada por APOL1. Ambos tratamientos fueron bien tolerados, presentando en su mayoría eventos adversos leves a moderados.
Vertex Pharmaceuticals는 ASN Kidney Week Congress에서 여러 신장 질환 프로그램에 대한 업데이트를 공유했습니다. 새로운 데이터에 따르면 povetacicept는 IgA 신병증 환자에서 48주 동안 평균 UPCR을 66% 감소시켰고, 63%가 임상 관해에 도달했습니다. 주요 외막 신병증에서 povetacicept는 24주 동안 평균 UPCR을 62% 감소시켰습니다. 이 회사는 IgA 신병증에 대한 글로벌 3상 시험 RAINIER를 시작했으며, APOL1 매개 신장 질환에 대한 inaxaplin의 3상 AMPLITUDE 시험에 대한 등록을 계속하고 있습니다. 두 치료법 모두 주로 경미한 또는 중등도의 부작용으로 잘 견딜 수 있었습니다.
Vertex Pharmaceuticals a partagé des mises à jour sur plusieurs programmes de maladies rénales lors du Congrès ASN Kidney Week. De nouvelles données montrent que povetacicept a atteint une réduction moyenne de 66% de l'UPCR après 48 semaines chez des patients atteints de néphropathie à IgA, avec 63% atteignant une rémission clinique. Dans la néphropathie membranée primaire, povetacicept a montré une réduction moyenne de l'UPCR de 62% après 24 semaines. L'entreprise a lancé l'essai mondial de phase 3 RAINIER pour la néphropathie à IgA et continue le recrutement dans l'essai de phase 3 AMPLITUDE de inaxaplin pour la maladie rénale médiée par APOL1. Les deux traitements ont été bien tolérés, avec des événements indésirables majoritairement légers à modérés.
Vertex Pharmaceuticals hat beim ASN Kidney Week Kongress Updates zu mehreren Programmen für Nierenerkrankungen geteilt. Neue Daten zeigen, dass povetacicept bei Patienten mit IgA-Nephropathie nach 48 Wochen eine mittlere UPCR-Reduktion von 66% erreichte, wobei 63% klinische Remission erzielten. Bei der primären membranösen Nephropathie zeigte povetacicept eine mittlere UPCR-Reduktion von 62% nach 24 Wochen. Das Unternehmen startete die globale Phase-3-Studie RAINIER für IgA-Nephropathie und setzt die Rekrutierung in der Phase-3-Studie AMPLITUDE von inaxaplin für APOL1-vermittelte Nierenerkrankung fort. Beide Behandlungen wurden gut vertragen, mit überwiegend milden bis moderaten Nebenwirkungen.
- Povetacicept showed 66% mean UPCR reduction in IgA nephropathy at 48 weeks
- 63% of IgAN patients achieved clinical remission with povetacicept
- 62% mean UPCR reduction in primary membranous nephropathy at 24 weeks
- 87% reduction in anti-PLA2R1 autoantibodies at week 20 in pMN patients
- Both 80mg and 240mg doses demonstrated stable renal function
- Small patient sample size (8 patients at 48 weeks for IgAN, 3 patients at 24 weeks for pMN)
Insights
The clinical data for povetacicept in kidney diseases shows remarkable promise. The
The dual inhibition of BAFF/APRIL pathways appears highly effective while maintaining a favorable safety profile. The initiation of the RAINIER Phase 3 trial and ongoing AMPLITUDE study for inaxaplin positions Vertex to potentially dominate the kidney disease market, particularly in areas with significant unmet needs.
This pipeline progress significantly strengthens Vertex's position in the nephrology space. The company is strategically expanding beyond its cystic fibrosis stronghold into multiple kidney diseases with large market potential. The robust efficacy data across different indications suggests povetacicept could become a multi-billion dollar asset.
The advancement of inaxaplin as a first-in-class APOL1 inhibitor also represents a major opportunity in AMKD, where no approved treatments exist. These developments could drive substantial revenue growth and market expansion for Vertex in the coming years.
- New data on povetacicept 80 mg SC Q4 weeks in IgA nephropathy shows mean UPCR reduction from baseline of
- First proteinuria data on povetacicept in primary membranous nephropathy shows mean UPCR reduction from baseline of
- Global Phase 3 RAINIER trial of povetacicept in IgA nephropathy now underway –
– Enrollment and dosing ongoing in Phase 3 portion of the global Phase 2/3 pivotal clinical trial of inaxaplin for the treatment of APOL1-mediated kidney disease –
“We are very pleased with the broadening of our innovative pipeline in renal medicine which now spans programs in AMKD, IgAN, pMN and polycystic kidney disease,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. “The new data in IgAN and pMN we shared at this year’s ASN congress continue to reinforce povetacicept as a potential best-in-class therapy and demonstrate its potential as a pipeline-in-a-product. We also continue to make progress in AMKD and autosomal dominant polycystic kidney disease (ADPKD) where we are advancing the first potential treatments to address the underlying cause of these diseases.”
Povetacicept in IgAN
Vertex presented data on 54 patients with IgAN who received povetacicept 80 mg or 240 mg subcutaneously every 4 weeks (SC Q4W). Treatment with povetacicept 80 mg SC Q4W demonstrated a clinically meaningful decrease in proteinuria, with a mean
Treatment with povetacicept 240 mg SC Q4W was associated with similar improvements in proteinuria along with stable renal function.
Both doses have been well tolerated in patients with IgAN. The majority of adverse events (AEs) were mild or moderate in severity, and there were no serious adverse events (SAEs) related to povetacicept.
Vertex has now initiated RAINIER, a global Phase 3 clinical trial of povetacicept 80 mg in IgAN.
A poster presentation #FR-PO854 entitled “Results from Longer Follow-Up with Povetacicept, an Enhanced Dual BAFF/APRIL Antagonist, in IgA Nephropathy (RUBY-3 Study)” was presented during the poster session on October 25 from 10:00 a.m. to 12:00 p.m. PDT.
Povetacicept in pMN
Vertex also presented emerging data in patients with pMN who received povetacicept 80 mg SC Q4W, with three patients having completed at least 24 weeks of treatment. Treatment with povetacicept demonstrated a mean
Povetacicept was well tolerated in patients with pMN, with AEs that were mild or moderate in severity. There were no SAEs related to povetacicept.
A poster presentation #TH-PO589 entitled “Updated Results with Povetacicept, an Enhanced Dual BAFF/APRIL Antagonist, in Primary Membranous Nephropathy (RUBY-3 Study)” was presented during the poster session on October 24 from 10:00 a.m. to 12:00 p.m. PDT.
AMKD
Vertex is developing inaxaplin, a potential first-in-class, investigational, oral small molecule inhibitor of APOL1, with the goal of targeting the underlying cause of AMKD. Enrollment and dosing are ongoing in the Phase 3 portion of the global Phase 2/3 pivotal AMPLITUDE clinical trial of inaxaplin.
Vertex has the following AMKD poster presentations at ASN:
- Poster presentation #TH-P01203 entitled “AMPLITUDE: A Phase 2/3 Adaptive Trial of Inaxaplin in APOL1-mediated Kidney Disease” was presented during the poster session on October 24 from 10:00 a.m. to 12:00 p.m. PDT.
- Poster presentation #SA-PO701 entitled “Small Molecule APOL1 Channel Inhibitor Reduces Proteinuria, Rescues Podocyte Injury, and Reverses eGFR Decline in an APOL1-Mediated Kidney Disease Mouse Model” will be presented during the poster session on October 26 from 10:00 a.m. to 12:00 p.m. PDT.
- Poster presentation #SA-PO700 entitled “Small Molecule Inhibition of APOL1 Channel Activity Protects Podocytes from Mitochondrial Dysfunction, Cell Death and Barrier Disruption Induced by APOL1 Risk Variants” will be presented during the poster session on October 26 from 10:00 a.m. to 12:00 p.m. PDT.
All accepted abstracts are available online on the ASN website.
About IgA Nephropathy (IgAN)
IgAN is a serious, progressive, life-threatening, B cell-mediated chronic kidney disease that is the most common cause of primary (idiopathic) glomerulonephritis, affecting people worldwide including approximately 130,000 people in the
About RAINIER
RAINIER is a global Phase 3 pivotal trial of povetacicept 80 mg vs. placebo on top of standard of care in approximately 480 people with IgAN. The study is designed to have a pre-planned interim analysis evaluating UPCR for the povetacicept arm versus placebo after a certain number of patients reach 36 weeks of treatment. If positive, the interim analysis may serve as the basis for Vertex to seek accelerated approval in the
About Primary Membranous Nephropathy (pMN)
Primary membranous nephropathy is a serious, progressive, life-threatening B cell-mediated chronic kidney disease affecting people worldwide, with approximately 60,000 people diagnosed in the
About RUBY-3
RUBY-3 is an ongoing, multiple ascending dose, multi-cohort, open label, Phase 1/2 basket study of povetacicept in autoimmune glomerulonephritis, including IgAN, pMN, lupus nephritis and ANCA-associated vasculitis with glomerulonephritis where povetacicept is being administered subcutaneously for up to 104 weeks.
About Povetacicept
Povetacicept is a dual antagonist of the BAFF (B cell activating factor) and APRIL (a proliferation inducing ligand) cytokines, which play key roles in pathogenesis of multiple autoimmune diseases via their roles in the activation, differentiation and/or survival of B cells, T cells and innate immune cells. Based upon an engineered TACI (transmembrane activator and CAML interactor) domain, povetacicept has higher binding affinity and greater potency in preclinical studies versus other inhibitors of BAFF and/or APRIL alone and has demonstrated potential best-in-class efficacy in a clinical trial in patients with IgA nephropathy and primary membranous nephropathy. Povetacicept is also in development for multiple serious diseases including other autoimmune kidney diseases and autoimmune cytopenias.
About APOL1-Mediated Kidney Disease (AMKD)
AMKD is a genetic kidney disease affecting approximately 100,000 people in the
About Inaxaplin
Inaxaplin is a potential first-in-class, investigational small molecule inhibitor of APOL1, and the first investigational therapy aimed at treating the underlying cause of AMKD.
About AMPLITUDE
AMPLITUDE is a global Phase 2/3 pivotal trial of inaxaplin for the treatment of AMKD, in which a 45 mg once daily oral dose is compared to placebo, on top of standard of care. The study is designed to have a pre-planned interim analysis at Week 48 evaluating estimated glomerular filtration rate (eGFR) slope, a measure of kidney function, supported by a percent change from baseline in proteinuria, in the inaxaplin arm versus placebo. If positive, the interim analysis may serve as the basis for Vertex to seek accelerated approval in the
About Autosomal Dominant Polycystic Kidney Disease (ADPKD)
ADPKD is the most common inherited kidney disease and one of the most common severe Mendelian genetic diseases, affecting approximately 250,000 diagnosed people in the
In most cases, ADPKD is caused by variants in the PKD1 and PKD2 genes, which express proteins known as polycystins. The majority of ADPKD patients (~
About Vertex
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has approved medicines that treat the underlying causes of multiple chronic, life-shortening genetic diseases — cystic fibrosis, sickle cell disease and transfusion-dependent beta thalassemia — and continues to advance clinical and research programs in these diseases. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including acute and neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes and myotonic dystrophy type 1.
Vertex was founded in 1989 and has its global headquarters in
Vertex Special Note Regarding Forward-Looking Statements
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, the statements by Carmen Bozic, M.D., in this press release, and statements regarding Vertex’s expectations and plans for the global Phase 3 RAINIER study of povetacicept, including the potential for an interim analysis to serve as the basis to seek accelerated approval in the
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