Intellia Therapeutics Announces FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to Nexiguran Ziclumeran (nex-z) for the Treatment of Hereditary Transthyretin (ATTR) Amyloidosis with Polyneuropathy
Intellia Therapeutics (NASDAQ:NTLA) announced that its investigational therapy nexiguran ziclumeran (nex-z) has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for treating hereditary transthyretin amyloidosis with polyneuropathy. This single-dose CRISPR-based treatment aims to inactivate the TTR gene to prevent TTR protein production. The designation was granted based on promising Phase 1 data showing rapid, deep, and durable TTR reduction. This marks the third special regulatory designation for nex-z, following Orphan Drug Designations from both the FDA and European Commission.
Intellia Therapeutics (NASDAQ:NTLA) ha annunciato che la sua terapia sperimentale nexiguran ziclumeran (nex-z) ha ricevuto la designazione di Therapy Avanzata in Medicina Rigenerativa (RMAT) dalla FDA per il trattamento dell'amilodosi ereditaria da transtiretina con polineuropatia. Questo trattamento CRISPR a dose singola mira a inattivare il gene TTR per prevenire la produzione della proteina TTR. La designazione è stata concessa sulla base di promettenti dati di Fase 1 che mostrano una rapida, profonda e duratura riduzione della TTR. Questo segna la terza designazione normativa speciale per nex-z, dopo le designazioni di Farmaco Orfano sia dalla FDA che dalla Commissione Europea.
Intellia Therapeutics (NASDAQ:NTLA) anunció que su terapia experimental nexiguran ziclumeran (nex-z) ha recibido la designación de Terapia Avanzada en Medicina Regenerativa (RMAT) por parte de la FDA para el tratamiento de la amiloidosis hereditaria por transtiretina con polineuropatía. Este tratamiento basado en CRISPR, de dosis única, tiene como objetivo inactivar el gen TTR para prevenir la producción de la proteína TTR. La designación fue concedida en base a prometedores datos de la Fase 1 que mostraron una reducción rápida, profunda y duradera de TTR. Esta marca la tercera designación normativa especial para nex-z, después de las Designaciones de Medicamento Huérfano tanto de la FDA como de la Comisión Europea.
인텔리아 치료제 (NASDAQ:NTLA)는 실험적 치료제 넥시구란 지클루메란(nex-z)이 유전성 트랜스티레틴 아밀로이드증과 다발신경병증 치료를 위해 FDA로부터 재생의학고급치료(RMAT) 지정을 받았다고 발표했습니다. 이 단회 복용 CRISPR 기반 치료는 TTR 단백질 생성을 방지하기 위해 TTR 유전을 비활성화하는 것을 목표로 합니다. 이 지명은 TTR 감소의 빠르고 깊고 지속적인 데이터를 보여주는 유망한 1상 데이터에 기반하여 부여되었습니다. 이는 FDA와 유럽 위원회 모두에서 고아약 지정에 이어 넥스-z에 대해 세 번째 특별 규제 지정을 의미합니다.
Intellia Therapeutics (NASDAQ:NTLA) a annoncé que sa thérapie expérimentale nexiguran ziclumeran (nex-z) a reçu la désignation de Thérapie Avancée en Médecine Régénérative (RMAT) de la FDA pour traiter l'amylose héréditaire par transthyrétine avec polynévropathie. Ce traitement unique basé sur CRISPR vise à inactiver le gène TTR pour empêcher la production de la protéine TTR. Cette désignation a été accordée sur la base de données prometteuses de la Phase 1 montrant une réduction rapide, profonde et durable de TTR. Cela marque la troisième désignation réglementaire spéciale pour nex-z, après les désignations de médicament orphelin de la FDA et de la Commission européenne.
Intellia Therapeutics (NASDAQ:NTLA) gab bekannt, dass seine experimentelle Therapie nexiguran ziclumeran (nex-z) von der FDA die Regenerative Medicine Advanced Therapy (RMAT)-Einstufung für die Behandlung der erblichen Transthyretin-Amyloidose mit Polyneuropathie erhalten hat. Diese Einzeldosis-CRISPR-Behandlung zielt darauf ab, das TTR-Gen inaktiv zu machen, um die Produktion des TTR-Protein zu verhindern. Die Einstufung wurde basierend auf vielversprechenden Daten der Phase 1 gewährt, die eine schnelle, tiefe und nachhaltige Reduktion von TTR zeigen. Dies ist die dritte besondere regulatorische Einstufung für nex-z, nach den Orphan-Drug-Einstufungen sowohl von der FDA als auch von der Europäischen Kommission.
- Received FDA RMAT designation, potentially expediting development and review process
- Phase 1 data showed promising results with rapid, deep, and durable TTR reduction
- Therapy has secured three special regulatory designations, including US and EU Orphan Drug status
- Development partnership with Regeneron strengthens commercialization potential
- Still in early clinical phase (Phase 1) with significant development milestones ahead
- Faces competition in the ATTR amyloidosis treatment market
Insights
The RMAT designation for nex-z represents a significant regulatory milestone that could accelerate the therapy's path to market. The designation was granted based on compelling Phase 1 data showing rapid, deep and durable TTR reduction. This is particularly noteworthy as ATTR amyloidosis with polyneuropathy is a progressive, life-threatening condition with treatment options.
The key advantages of this designation include expedited development process, early FDA interactions, potential use of surrogate endpoints for accelerated approval and possible priority review. Combined with existing Orphan Drug Designations in both US and EU markets, this creates a favorable regulatory pathway that could significantly reduce time-to-market and development costs.
This single-dose CRISPR treatment approach, if successful, could revolutionize ATTR amyloidosis treatment by offering a one-time solution instead of current chronic therapies. The collaboration with Regeneron adds substantial development and commercialization expertise to advance this groundbreaking therapy.
This regulatory milestone significantly enhances NTLA's market position in the ATTR amyloidosis space. The commercial implications are substantial, as RMAT designation typically increases the probability of successful market entry and can reduce development timelines by months or years. The global ATTR amyloidosis market is projected to grow significantly and a one-time CRISPR treatment could capture substantial market share from existing chronic therapies.
The partnership with Regeneron provides important commercial infrastructure and expertise for eventual market launch. The therapy's potential to halt and reverse disease progression could command premium pricing, particularly given the current annual costs of existing treatments. This regulatory advancement strengthens NTLA's competitive position against other companies developing ATTR treatments.
CAMBRIDGE, Mass, Nov. 25, 2024 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to nexiguran ziclumeran (nex-z, also known as NTLA-2001) for the treatment of hereditary transthyretin (ATTR) amyloidosis with polyneuropathy (ATTRv-PN). Nex-z is an in vivo CRISPR-based investigational therapy designed as a single-dose treatment to inactivate the TTR gene and thereby prevent the production of TTR protein for the treatment of ATTR amyloidosis. Development and commercialization of nex-z is led by Intellia as part of a multi-target collaboration with Regeneron.
“This RMAT designation underscores the transformative potential of nex-z, our investigational in vivo CRISPR-based gene editing therapy for those living with hereditary ATTR amyloidosis with polyneuropathy,” said Intellia President and Chief Executive Officer John Leonard, M.D. “It was granted following the FDA’s review of our compelling interim Phase 1 data that indicated our one-time treatment led to rapid, deep and durable TTR reduction, which is expected to halt and potentially reverse the disease. We look forward to working closely with the FDA to bring this potential paradigm-shifting therapy to patients as quickly as possible.”
The RMAT designation was established under the 21st Century Cures Act to expedite the development and review of promising therapeutic candidates, including genetic therapies, that are intended to treat, modify, reverse or cure a serious or life-threatening disease. RMAT designation includes benefits, such as early interactions with the FDA, including discussions on surrogate or intermediate endpoints that could potentially support accelerated approval and satisfy post-approval requirements, and potential priority review of a product’s biologics license application (BLA).
This RMAT designation is the third special regulatory designation received by Intellia for nex-z. Nex-z was also granted Orphan Drug Designation by the U.S. FDA and European Union Orphan Drug Designation by the European Commission.
About nexiguran ziclumeran (nex-z, also known as NTLA-2001)
Based on Nobel Prize-winning CRISPR/Cas9 technology, nex-z has the potential to become the first one-time treatment for transthyretin (ATTR) amyloidosis. Nex-z is designed to inactivate the TTR gene that encodes for the transthyretin (TTR) protein. Interim Phase 1 clinical data showed the administration of nex-z led to consistent, deep and long-lasting TTR reduction. Intellia leads development and commercialization of nex-z as part of a multi-target discovery, development and commercialization collaboration with Regeneron.
About Transthyretin (ATTR) Amyloidosis
Transthyretin amyloidosis, or ATTR amyloidosis, is a rare, progressive and fatal disease. Hereditary ATTR (ATTRv) amyloidosis occurs when a person is born with mutations in the TTR gene, which causes the liver to produce structurally abnormal transthyretin (TTR) protein with a propensity to misfold. These damaged proteins build up as amyloid in the body, causing serious complications in multiple tissues, including the heart, nerves and digestive system. ATTRv amyloidosis predominantly manifests as polyneuropathy (ATTRv-PN), which can lead to nerve damage, or cardiomyopathy (ATTRv-CM), which can lead to heart failure. Some individuals without the genetic mutation produce non-mutated, or wild-type TTR proteins that become unstable over time, misfolding and aggregating in disease-causing amyloid deposits. This condition, called wild-type ATTR (ATTRwt) amyloidosis, primarily affects the heart. There are an estimated 50,000 people worldwide living with ATTRv amyloidosis and between 200,000 and 500,000 people with ATTRwt amyloidosis. There is no known cure for ATTR amyloidosis and currently available medications are limited to slowing accumulation of misfolded TTR protein.
About Intellia Therapeutics
Intellia Therapeutics, Inc. (NASDAQ:NTLA) is a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies. The company’s in vivo programs use CRISPR to enable precise editing of disease-causing genes directly inside the human body. Intellia’s ex vivo programs use CRISPR to engineer human cells outside the body for the treatment of cancer and autoimmune diseases. Intellia’s deep scientific, technical and clinical development experience, along with its people, is helping set the standard for a new class of medicine. To harness the full potential of gene editing, Intellia continues to expand the capabilities of its CRISPR-based platform with novel editing and delivery technologies. Learn more at intelliatx.com and follow us @intelliatx.
Forward-Looking Statements
This press release contains “forward-looking statements” of Intellia Therapeutics, Inc. (“Intellia” or the “Company”) within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellia’s beliefs and expectations regarding: the safety, efficacy, success and advancement of its clinical program for nexiguran ziclumeran (nex-z, also known as NTLA-2001) for the treatment of hereditary transthyretin (ATTR) amyloidosis with polyneuropathy (ATTRv-PN) pursuant to its clinical trial applications and investigational new drug application, including the potential of nex-z to halt and potentially reverse the disease and its potential to offer a paradigm-shifting treatment option for patients with ATTRv-PN.
Any forward-looking statements in this press release are based on management’s current expectations and beliefs of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to Intellia’s ability to protect and maintain its intellectual property position; risks related to valid third party intellectual property; risks related to Intellia’s relationship with third parties, including its licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; and uncertainties related to the authorization, initiation, enrollment and conduct of studies and other development requirements for its product candidates, including nex-z; risks related to the results of preclinical or clinical studies, including that they may not be positive or predictive of future results; the risk that one or more of Intellia’s product candidates, including nex-z, will not be successfully developed and commercialized; and risks related to Intellia’s reliance on collaborations, including that its collaboration with Regeneron will not continue or will not be successful. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellia’s actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in Intellia’s most recent annual report on Form 10-K as well as discussions of potential risks, uncertainties, and other important factors in Intellia’s other filings with the Securities and Exchange Commission, including its quarterly report on Form 10-Q. All information in this press release is as of the date of the release, and Intellia undertakes no duty to update this information unless required by law.
Intellia Contacts:
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FAQ
What is the RMAT designation granted to Intellia's nex-z (NTLA)?
How does Intellia's nex-z (NTLA) treat ATTR amyloidosis?
What regulatory designations has Intellia's nex-z (NTLA) received?
