AC Immune’s ACI-35.030 (now “JNJ-2056”) Granted FDA Fast Track Designation for Alzheimer’s Disease
AC Immune SA (NASDAQ: ACIU) announced that its active-immunotherapy candidate, ACI-35.030 (now called JNJ-2056), has received Fast Track designation from the FDA for Alzheimer's disease treatment. The therapy targets the pathologic form of the Tau protein, phosphorylated Tau (pTau). A Phase 2b clinical trial called ReTain is currently recruiting participants with preclinical Alzheimer's disease.
Key points:
- First active immunotherapy tested in a preclinical AD population
- Potential therapeutic advantages and benefits in convenience and access
- Shown to specifically target toxic form of Tau in Phase 1b/2a clinical testing
- Developed in partnership with Janssen Pharmaceuticals
- ReTain trial is fully funded and conducted by Janssen
AC Immune SA (NASDAQ: ACIU) ha annunciato che il suo candidato per l'immunoterapia attiva, ACI-35.030 (ora chiamato JNJ-2056), ha ricevuto la designazione Fast Track dalla FDA per il trattamento dell'Alzheimer. Questa terapia mira alla forma patologica della proteina Tau, la Tau fosforilata (pTau). Un studio clinico di Fase 2b chiamato ReTain sta attualmente reclutando partecipanti con Alzheimer preclinico.
Punti chiave:
- Primo trattamento immunoterapico attivo testato in una popolazione AD preclinica
- Potenziali vantaggi terapeutici e benefici in termini di comodità e accesso
- Dimostrato di mirare specificamente alla forma tossica della Tau nei test clinici di Fase 1b/2a
- Sviluppato in partnership con Janssen Pharmaceuticals
- Lo studio ReTain è completamente finanziato e condotto da Janssen
AC Immune SA (NASDAQ: ACIU) anunció que su candidato a inmunoterapia activa, ACI-35.030 (ahora llamado JNJ-2056), ha recibido la designación Fast Track de la FDA para el tratamiento del Alzheimer. Esta terapia se dirige a la forma patológica de la proteína Tau, la Tau fosforilada (pTau). Un ensayo clínico de Fase 2b llamado ReTain está actualmente reclutando participantes con enfermedad de Alzheimer preclínica.
Puntos clave:
- Primera inmunoterapia activa probada en una población de AD preclínica
- Posibles ventajas terapéuticas y beneficios en comodidad y acceso
- Se ha demostrado que apunta específicamente a la forma tóxica de Tau en pruebas clínicas de Fase 1b/2a
- Desarrollado en colaboración con Janssen Pharmaceuticals
- El ensayo ReTain está completamente financiado y conducido por Janssen
AC Immune SA (NASDAQ: ACIU)는 자사의 활성 면역요법 후보인 ACI-35.030(현재 JNJ-2056으로 명명됨)이 알츠하이머병 치료를 위한 신속 승인(Fast Track) 지정을 FDA로부터 받았다고 발표했습니다. 이 치료법은 병리학적 형태의 타우 단백질인 인산화 타우(pTau)를 표적으로 합니다. 현재 2b상 임상 시험인 ReTain이 전임상 알츠하이머병 환자를 모집하고 있습니다.
주요 사항:
- 전임상 AD 집단에서 시험된 첫 번째 활성 면역요법
- 치료상의 이점과 접근의 편리함
- 1b/2a상 임상 시험에서 독성 형태의 타우를 특정적으로 표적하는 것으로 나타났음
- 얀센 제약과의 파트너십으로 개발됨
- ReTain 시험은 얀센이 완전 자금 지원 및 수행
AC Immune SA (NASDAQ: ACIU) a annoncé que son candidat à l'immunothérapie active, ACI-35.030 (désormais appelé JNJ-2056), a reçu la désignation Fast Track de la FDA pour le traitement de la maladie d'Alzheimer. Cette thérapie cible la forme pathologique de la protéine Tau, la Tau phosphorylée (pTau). Un essai clinique de Phase 2b appelé ReTain recrute actuellement des participants atteints de la maladie d'Alzheimer préclinique.
Points clés :
- Première immunothérapie active testée dans une population AD préclinique
- Avantages thérapeutiques potentiels et bénéfices en termes de commodité et d'accès
- A montré qu'elle cible spécifiquement la forme toxique de Tau dans les essais cliniques de Phase 1b/2a
- Développé en partenariat avec Janssen Pharmaceuticals
- L'essai ReTain est entièrement financé et conduit par Janssen
AC Immune SA (NASDAQ: ACIU) hat bekannt gegeben, dass sein Kandidat für aktive Immuntherapie, ACI-35.030 (jetzt JNJ-2056 genannt), von der FDA die Fast-Track-Status für die Behandlung von Alzheimer erhalten hat. Die Therapie zielt auf die pathologische Form des Tau-Proteins, phosphoryliertes Tau (pTau), ab. Eine Phase-2b-Studie namens ReTain rekrutiert derzeit Teilnehmer mit präklinischer Alzheimer-Krankheit.
Wichtige Punkte:
- Erste aktive Immuntherapie, die in einer präklinischen AD-Population getestet wurde
- Potenzielle therapeutische Vorteile und Bequemlichkeit im Zugang
- In Phase 1b/2a klinischen Tests speziell auf die toxische Form von Tau abzielen
- In Partnerschaft mit Janssen Pharmaceuticals entwickelt
- Die ReTain-Studie wird vollständig finanziert und von Janssen durchgeführt
- FDA Fast Track designation granted for ACI-35.030 (JNJ-2056)
- Phase 2b ReTain trial initiated for preclinical Alzheimer's disease
- Potential therapeutic advantages and improved convenience
- Specific targeting of toxic Tau form demonstrated in Phase 1b/2a testing
- Partnership with Janssen Pharmaceuticals for development and commercialization
- ReTain trial fully funded by development partner
- None.
The FDA's Fast Track designation for ACI-35.030 (JNJ-2056) is a significant milestone for AC Immune and the field of Alzheimer's disease (AD) research. This designation acknowledges the potential of this active immunotherapy to address a critical unmet need in AD treatment. Here's why this news is particularly impactful:
- ACI-35.030 targets phosphorylated Tau (pTau), a key pathological protein in AD. This approach is differentiated from other therapies that target amyloid-beta, potentially offering a new avenue for treatment.
- The Phase 2b ReTain trial is pioneering in its focus on preclinical AD patients, aiming to intervene before symptoms manifest. This could be a game-changer in AD management if successful.
- The active immunotherapy approach may offer advantages in terms of convenience and accessibility, which could significantly impact patient compliance and treatment outcomes.
However, investors should note that while promising, the therapy is still in early-stage clinical trials. The road to potential approval remains long and uncertain, typical of AD drug development. The partnership with Janssen, a Johnson & Johnson company, provides financial backing and development expertise, which is a positive factor for the project's progression.
From a financial perspective, this FDA Fast Track designation for AC Immune's ACI-35.030 (JNJ-2056) is a positive catalyst for the company. Here's why:
- Fast Track status can potentially accelerate the development and review process, which could lead to earlier market entry if the drug proves successful. This could translate to faster revenue generation for AC Immune.
- The partnership with Janssen is crucial, as it means the Phase 2b ReTain trial is fully funded by the partner. This significantly reduces AC Immune's financial risk and burn rate.
- The Alzheimer's disease market is massive and growing, with hundreds of millions of potential patients. A successful therapy could generate substantial revenues.
However, investors should exercise caution. While this news is positive, AC Immune remains a clinical-stage company with no approved products. The company's financial health and stock performance will continue to be heavily influenced by clinical trial results and regulatory decisions. The Alzheimer's disease field is notoriously challenging, with many promising candidates failing in late-stage trials. Diversification and careful risk assessment are important when considering investments in this sector.
AC Immune’s ACI-35.030 (now “JNJ-2056”) Granted FDA Fast Track Designation for Alzheimer’s Disease
- Treatment of first randomized person with preclinical AD expected this quarter
- Phase 2b ReTain trial is fully funded and conducted by development partner
Lausanne, Switzerland, July 25, 2024 – AC Immune SA (NASDAQ: ACIU), a clinical-stage biopharmaceutical company pioneering precision medicine for neurodegenerative diseases, today announced that its active-immunotherapy candidate, ACI-35.030 (now called JNJ-2056), targeting the pathologic form of the Tau protein, phosphorylated Tau (pTau), has received Fast Track designation from the U.S. Food and Drug Administration (FDA). The recently initiated Phase 2b clinical trial ReTain is currently recruiting participants with preclinical Alzheimer’s disease, where individuals have yet to show clinical symptoms.
Dr. Andrea Pfeifer, CEO of AC Immune SA, commented: “Fast Track designation is an important recognition of the differentiation and potential value for patients of our anti-pTau active immunotherapy, ACI-35.030. The Phase 2b ReTain study is the first time any active immunotherapy is being tested in a preclinical AD population. We believe this modality has the potential to offer therapeutic advantages, as well as benefits in terms of convenience and access. Fast Track designation offers opportunities for more efficient development and regulatory review. More importantly, this underscores and validates the potential therapeutic impact of an active immunotherapy specifically targeting pTau, the key pathologic species of Tau protein. In Phase 1b/2a clinical testing, ACI-35.030 was shown to specifically target this toxic form of Tau and spare normal endogenous forms of the protein. We and our partners continue to drive innovation in the treatment and potential prevention of Alzheimer’s disease, developing new mechanisms of action and first-in-class potential therapeutics that are safe and simple to use for the hundreds of millions of people living with or at risk of the disease.”
JNJ-2056 is being developed pursuant to a global license, development and commercialization agreement with Janssen Pharmaceuticals, Inc. (Janssen), a Johnson & Johnson company. The ReTain trial is fully funded and conducted by Janssen.
About the ReTain Trial
The Phase 2b ReTain trial is a potentially registration-enabling randomized, multicenter, double-blind, placebo-controlled clinical study in participants with preclinical AD to assess the clinical effect of active immunization with JNJ-64042056. It is designed to test the hypothesis that JNJ-2056 has a disease-modifying effect that can delay or prevent the onset of cognitive impairment or other clinical symptoms in individuals with preclinical AD through inhibition of seeding and spreading of pathological Tau.
The study will include approximately 500 participants with preclinical AD (cognitively normal, Tau positive), who will be randomized in a 1:1 ratio to a single dose level of JNJ-2056 or placebo and administered as intramuscular injections for a maximum of 4 years. The primary endpoint will measure cognitive decline as assessed by the Preclinical AD Cognitive Composite 5 (PACC-5) score. The key secondary efficacy endpoint will assess the effect of JNJ-2056 on the propagation and/or accumulation of Tau pathology compared with placebo, as measured by Tau PET imaging.
About ACI-35.030 (JNJ-2056)
ACI-35.030, derived from AC Immune’s SupraAntigen® platform, has been shown in clinical studies to induce a strong polyclonal antibody response that matures and is maintained against key pathological forms of Tau believed to drive Tau aggregation and disease progression. ACI-35.030 is designed to enhance the formation of broad-spectrum protective antibodies against pTau. This investigational candidate has the potential to reduce pathological Tau spreading in the early stages of AD, and thereby may reduce or prevent disease progression.
About AC Immune SA
AC Immune SA is a clinical-stage biopharmaceutical company and a global leader in precision medicine for neurodegenerative diseases, including Alzheimer’s disease, Parkinson’s disease, and NeuroOrphan indications driven by misfolded proteins. The Company’s two clinically validated technology platforms, SupraAntigen® and Morphomer®, fuel its broad and diversified pipeline of first- and best-in-class assets, which currently features sixteen therapeutic and diagnostic programs, five of which are currently in Phase 2 clinical trials and one of which is in Phase 3. AC Immune has a strong track record of securing strategic partnerships with leading global pharmaceutical companies, resulting in substantial non-dilutive funding in potential milestone payments plus royalties.
SupraAntigen® is a registered trademark of AC Immune SA in the following territories: AU, EU, CH, GB, JP, RU, SG and USA. Morphomer® is a registered trademark of AC Immune SA in CN, CH, GB, JP, KR, NO and RU.
The information on our website and any other websites referenced herein is expressly not incorporated by reference into, and does not constitute a part of, this press release.
For further information, please contact:
| Head of Investor Relations & Corporate Communications Gary Waanders, Ph.D., MBA AC Immune Phone: +41 21 345 91 91 Email: gary.waanders@acimmune.com | U.S. Investors Corey Davis, Ph.D. LifeSci Advisors Phone: +1 212 915 2577 Email: cdavis@lifesciadvisors.com |
| International Media Chris Maggos Cohesion Bureau Phone: +41 79 367 6254 Email: chris.maggos@cohesionbureau.com |
Forward looking statements
This press release contains statements that constitute “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Forward-looking statements are statements other than historical fact and may include statements that address future operating, financial or business performance or AC Immune’s strategies or expectations. In some cases, you can identify these statements by forward-looking words such as “may,” “might,” “will,” “should,” “expects,” “plans,” “anticipates,” “believes,” “estimates,” “predicts,” “projects,” “potential,” “outlook” or “continue,” and other comparable terminology. Forward-looking statements are based on management’s current expectations and beliefs and involve significant risks and uncertainties that could cause actual results, developments and business decisions to differ materially from those contemplated by these statements. These risks and uncertainties include those described under the captions “Item 3. Key Information – Risk Factors” and “Item 5. Operating and Financial Review and Prospects” in AC Immune’s Annual Report on Form 20-F and other filings with the Securities and Exchange Commission. These include: the impact of Covid-19 on our business, suppliers, patients and employees and any other impact of Covid-19. Forward-looking statements speak only as of the date they are made, and AC Immune does not undertake any obligation to update them in light of new information, future developments or otherwise, except as may be required under applicable law. All forward-looking statements are qualified in their entirety by this cautionary statement.
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