Arrowhead Pharmaceuticals Presents Interim Clinical Data on ARO-CFB for the Treatment of Complement Mediated Diseases
Arrowhead Pharmaceuticals (NASDAQ: ARWR) announced interim results from their Phase 1/2a clinical study of ARO-CFB, an RNAi therapeutic targeting complement factor B for complement mediated diseases. The data, presented at the 8th Complement-Based Drug Development Summit, showed significant efficacy with:
- Up to 90% reduction in circulating CFB protein with over 3 months duration
- Near complete inhibition of alternative pathway activity (100% mean reduction) at 200mg and 400mg doses
- Generally well-tolerated safety profile with mostly mild adverse events
The company plans to complete Part 1 of the study and proceed to Part 2, focusing on patients with immunoglobulin A nephropathy, the most common glomerular disease worldwide.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) ha annunciato risultati intermedi dal loro studio clinico di fase 1/2a di ARO-CFB, una terapia RNAi mirata al fattore B del complemento per le malattie mediate dal complemento. I dati, presentati all'8掳 Summit sullo Sviluppo di Farmaci Basati sul Complemento, hanno mostrato un'efficacia significativa con:
- Fino al 90% di riduzione della proteina CFB circolante per oltre 3 mesi
- Inibizione quasi completa dell'attivit脿 della via alternativa (riduzione media del 100%) a dosi di 200mg e 400mg
- Profilo di sicurezza generalmente ben tollerato con eventi avversi per lo pi霉 lievi
L'azienda prevede di completare la Parte 1 dello studio e procedere alla Parte 2, focalizzandosi su pazienti con nefrite da immunoglobulina A, la malattia glomerulare pi霉 comune al mondo.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) anunci贸 resultados intermedios de su estudio cl铆nico de fase 1/2a de ARO-CFB, una terapia de RNAi dirigida al factor B del complemento para enfermedades mediadas por complemento. Los datos, presentados en la 8陋 Cumbre sobre el Desarrollo de Medicamentos Basados en Complemento, mostraron una eficacia significativa con:
- Reducci贸n de hasta el 90% de la prote铆na CFB circulante con m谩s de 3 meses de duraci贸n
- Inhibici贸n casi completa de la actividad de la v铆a alternativa (reducci贸n media del 100%) a dosis de 200mg y 400mg
- Perfil de seguridad generalmente bien tolerado con eventos adversos mayormente leves
La empresa planea completar la Parte 1 del estudio y proceder a la Parte 2, centr谩ndose en pacientes con nefropat铆a por inmunoglobulina A, la enfermedad glomerular m谩s com煤n en el mundo.
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- 3臧滌洈 鞚挫儊 歆�靻嶋悩電� CFB 雼氨歆堨潣 靾滍櫂鞐愳劀 斓滊寑 90% 臧愳唽
- 200mg 氚� 400mg 鞖╇焿鞐愳劀 雽�觳� 瓴诫 頇滊彊鞚� 瓯办潣 鞕勳爠 鞏奠牅 (韽夑窢 100% 臧愳唽)
- 雽�攵�攵� 臧�氩检毚 攵�鞛戩毄鞙茧 鞚茧皹鞝侅溂搿� 鞛� 瓴敎 靾� 鞛堧姅 鞎堨爠靹� 頂勲頃�
須岇偓電� 鞐瓣惮 1攵�毳� 鞕勲頃橁碃 2攵�搿� 歆勴枆頃� 瓿勴殟鞚措┌, 靹戈硠鞐愳劀 臧�鞛� 頋旐暅 靷惮觳� 歆堩櫂鞚� 氅挫棴旮�搿滊秷毽� A 鞁犽硲歃� 頇橃瀽鞐� 歆戩頃� 瓴冹瀰雼堧嫟.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) a annonc茅 des r茅sultats interm茅diaires de son 茅tude clinique de phase 1/2a sur l'ARO-CFB, une th茅rapie RNAi ciblant le facteur B du compl茅ment pour les maladies m茅di茅es par le compl茅ment. Les donn茅es, pr茅sent茅es lors du 8猫me Sommet sur le D茅veloppement de M茅dicaments Bas茅s sur le Compl茅ment, ont montr茅 une efficacit茅 significative avec :
- R茅duction de jusqu'脿 90% de la prot茅ine CFB circulante durant plus de 3 mois
- Inhibition presque compl猫te de l'activit茅 de la voie alternative (r茅duction moyenne de 100%) 脿 des doses de 200mg et 400mg
- Profil de s茅curit茅 g茅n茅ralement bien tol茅r茅 avec surtout des effets ind茅sirables l茅gers
L'entreprise pr茅voit de compl茅ter la Partie 1 de l'茅tude et de passer 脿 la Partie 2, en se concentrant sur les patients atteints de n茅phropathie 脿 immunoglobuline A, la maladie glom茅rulaire la plus r茅pandue dans le monde.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) hat vorl盲ufige Ergebnisse aus ihrer Phase 1/2a-Studie zu ARO-CFB bekanntgegeben, einer RNAi-Therapie, die auf das Komplementfaktor B f眉r komplementvermittelte Krankheiten abzielt. Die Daten, die auf dem 8. Gipfel zur Entwicklung von komplementbasierten Arzneimitteln vorgestellt wurden, zeigten eine signifikante Wirksamkeit mit:
- Bis zu 90% Reduktion des zirkulierenden CFB-Proteins 眉ber mehr als 3 Monate
- Nahezu vollst盲ndige Hemmung der Aktivit盲t des alternativen Weges (Durchschnittsreduktion von 100%) bei 200mg und 400mg Dosen
- Allgemein gut vertr盲gliches Sicherheitsprofil mit 眉berwiegend milden Nebenwirkungen
Das Unternehmen plant, Teil 1 der Studie abzuschlie脽en und zu Teil 2 眉berzugehen, wobei der Fokus auf Patienten mit einer IgA-Nephropathie liegt, der weltweit h盲ufigsten glomerul盲ren Erkrankung.
- Achieved 90% reduction in CFB protein levels with single 400mg dose
- Demonstrated 100% mean reduction in alternative pathway activity by week 4
- Treatment effects showed duration greater than 3 months
- Safety profile shows good tolerability with mostly mild adverse events
- No treatment discontinuations due to adverse events
- None.
Insights
The interim Phase 1/2a data for ARO-CFB demonstrates remarkable efficacy in complement system modulation. The 90% reduction in circulating CFB protein and near-complete inhibition of alternative pathway activity are exceptional results that exceed typical early-phase expectations. The durability of effect lasting >3 months from a single dose is particularly noteworthy, suggesting potential for quarterly dosing regimens. The clean safety profile with mostly mild TEAEs and no discontinuations strengthens the therapeutic potential.
The progression to IgA nephropathy patients in Part 2 is strategically sound, as complement dysregulation is a key disease driver. If similar efficacy is demonstrated in patients, ARO-CFB could become a significant player in the
These compelling interim results significantly de-risk ARO-CFB's development program and enhance Arrowhead's position in the lucrative complement therapeutics space. The robust efficacy data, particularly the 100% reduction in alternative pathway activity at multiple dose levels, suggests potential best-in-class attributes. The favorable durability profile could translate to competitive advantages in pricing and market adoption.
For Arrowhead's
- Interim data from Phase 1/2a study demonstrate near complete inhibition in hemolytic activity and functional activity of alternative complement pathway
鈥淒ysregulated activation of the complement system can lead to progression of certain renal diseases, either by playing a directly pathogenic role, or by amplifying or exacerbating the inflammatory and damaging impact of non-complement disease triggers. In a Phase 1/2a clinical study, ARO-CFB treatment in healthy volunteers achieved deep and durable reductions in the liver production of complement factor B (CFB), which is involved in alternative complement pathway activation and associated with pathogenesis of diseases involving complement activation. Circulating levels of CFB protein were reduced by a mean of up to
Select ARO-CFB Results
In the ongoing AROCFB-1001 study, ARO-CFB achieved the following key results in normal healthy volunteers as of the interim data cutoff - 15 November 2024:
-
ARO-CFB led to dose dependent reductions in circulating CFB protein by up to
90% with greater than 3 months duration-
90% mean reduction achieved after a single dose of 400 mg -
90% mean reduction achieved after two doses of 100 mg
-
-
Single and multiple doses of ARO-CFB led to near complete inhibition of alternative pathway activity based on Wieslab AP
-
100% mean reduction achieved by week 4 after a single dose at both 200 mg and 400 mg doses -
92% and100% mean reductions were achieved after two doses at 100 mg and 200 mg, respectively
-
- Single and multiple doses of ARO-CFB led to near complete inhibition of alternative pathway hemolytic activity, measured by AH50
Safety and Tolerability Results
ARO-CFB has been generally well-tolerated to date with safety data supportive of further clinical development. There have been no treatment emergent adverse events (TEAE) leading to study or study drug discontinuation with most TEAEs being mild in severity.
About ARO-CFB
ARO-CFB is designed to reduce hepatic expression of complement factor B (CFB), which plays an important regulatory role in amplifying complement alternative pathway activation and has been identified as a promising therapeutic target. ARO-CFB is being developed as a potential treatment for complement mediated kidney diseases such as immunoglobulin A nephropathy (IgAN), which is the most common glomerular disease worldwide and carries a high lifetime risk of progression to end-stage renal disease. Additionally, ARO-CFB may have clinical applications in non-renal diseases involving complement activation.
About the AROCFB-1001 Phase 1/2 Study
AROCFB-1001 () is an ongoing Phase 1/2a dose-escalating study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ARO-CFB in up to 66 normal healthy volunteers (NHV) and patients with complement mediated kidney disease. In Part 1 of the study, NHVs will receive either one or two doses of ARO-CFB or placebo. In Part 2 of the study, adult patients with IgAN will receive 3 open-label doses of ARO-CFB. The study is designed to assess safety and tolerability and key pharmacodynamic parameters, including the change and percent change from baseline over time in serum CFB, and alternative complement pathway activity via AH50 and Wieslab AP.
About Arrowhead Pharmaceuticals
Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead鈥檚 RNAi-based therapeutics leverage this natural pathway of gene silencing.
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Source: Arrowhead Pharmaceuticals, Inc.
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Arrowhead Pharmaceuticals, Inc.
Vince Anzalone, CFA
626-304-3400
ir@arrowheadpharma.com
Investors:
LifeSci Advisors, LLC
Brian Ritchie
212-915-2578
britchie@lifesciadvisors.com
Media:
LifeSci Communications, LLC
Kendy Guarinoni, Ph.D.
724-910-9389
kguarinoni@lifescicomms.com
Source: Arrowhead Pharmaceuticals, Inc.
FAQ
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