Klotho Neurosciences, Inc. Granted FDA Orphan Drug Designation for KLTO-202 for Treatment of Amyotrophic Lateral Sclerosis ("ALS" or "Lou Gehrig's Disease")
Klotho Neurosciences (NASDAQ:KLTO) has received FDA Orphan Drug Designation for KLTO-202, its novel secreted-Klotho promoter gene therapy for treating Amyotrophic Lateral Sclerosis (ALS). This designation provides significant benefits including tax credits for clinical trials, GDUFA User Fee waivers, and 7 years of market exclusivity.
KLTO-202 targets motor neuron diseases using a muscle-specific promoter called "desmin" to express the s-KL gene and protein, specifically delivering therapy to the neuromuscular junction. The company has completed proof of concept studies in two animal models and is initiating manufacturing while planning regulatory meetings with the FDA and EMA.
Klotho Neurosciences (NASDAQ:KLTO) ha ottenuto la Designazione di Farmaco Orfano dalla FDA per KLTO-202, la sua nuova terapia genica che promuove il gene secreted-Klotho per il trattamento della Sclerosi Laterale Amiotrofica (SLA). Questa designazione offre importanti vantaggi, tra cui crediti d'imposta per gli studi clinici, esenzioni dalle tariffe GDUFA e 7 anni di esclusivit脿 sul mercato.
KLTO-202 agisce contro le malattie dei motoneuroni utilizzando un promotore specifico per i muscoli chiamato "desmina" per esprimere il gene e la proteina s-KL, fornendo la terapia in modo mirato alla giunzione neuromuscolare. L'azienda ha completato studi di prova del concetto su due modelli animali e sta avviando la produzione, pianificando inoltre incontri regolatori con FDA ed EMA.
Klotho Neurosciences (NASDAQ:KLTO) ha recibido la Designaci贸n de Medicamento Hu茅rfano por la FDA para KLTO-202, su novedosa terapia g茅nica promotora del gen secreted-Klotho para el tratamiento de la Esclerosis Lateral Amiotr贸fica (ELA). Esta designaci贸n ofrece beneficios importantes, incluyendo cr茅ditos fiscales para ensayos cl铆nicos, exenciones de tarifas GDUFA y 7 a帽os de exclusividad en el mercado.
KLTO-202 se dirige a enfermedades de las neuronas motoras usando un promotor espec铆fico para m煤sculos llamado "desmina" para expresar el gen y la prote铆na s-KL, entregando la terapia espec铆ficamente en la uni贸n neuromuscular. La compa帽铆a ha completado estudios de prueba de concepto en dos modelos animales y est谩 iniciando la fabricaci贸n mientras planea reuniones regulatorias con la FDA y la EMA.
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KLTO-202電� '雿办姢氙�(desmin)'鞚措澕電� 攴检湣 韸轨澊鞝� 頂勲氇劙毳� 靷毄頃橃棳 s-KL 鞙犾爠鞛愳檧 雼氨歆堨潉 氚滍槃鞁滌紲, 鞁犼步攴� 鞝戫暕攵�鞐� 旃橂鞝滊ゼ 韸轨澊鞝侅溂搿� 鞝勲嫭頃橂姅 鞖措彊 鞁犼步 歆堩櫂鞚� 響滌爜鞙茧 頃╇媹雼�. 須岇偓電� 霊� 臧�歆� 霃欕 氇嵏鞐愳劀 臧滊厫 歃濍獏 鞐瓣惮毳� 鞕勲頄堨溂氅�, 鞝滌“毳� 鞁滌瀾頃橁碃 FDA 氚� EMA鞕�鞚� 攴滌牅 須岇潣毳� 瓿勴殟 欷戩瀰雼堧嫟.
Klotho Neurosciences (NASDAQ:KLTO) a obtenu la d茅signation de m茅dicament orphelin par la FDA pour KLTO-202, sa nouvelle th茅rapie g茅nique promoteur du g猫ne secreted-Klotho pour le traitement de la scl茅rose lat茅rale amyotrophique (SLA). Cette d茅signation offre des avantages significatifs, notamment des cr茅dits d'imp么t pour les essais cliniques, des exon茅rations des frais GDUFA et 7 ans d'exclusivit茅 sur le march茅.
KLTO-202 cible les maladies des motoneurones en utilisant un promoteur sp茅cifique aux muscles appel茅 芦 desmine 禄 pour exprimer le g猫ne et la prot茅ine s-KL, d茅livrant la th茅rapie sp茅cifiquement 脿 la jonction neuromusculaire. La soci茅t茅 a achev茅 des 茅tudes de preuve de concept sur deux mod猫les animaux et lance la fabrication tout en planifiant des r茅unions r茅glementaires avec la FDA et l'EMA.
Klotho Neurosciences (NASDAQ:KLTO) hat von der FDA die Orphan-Drug-Designation f眉r KLTO-202 erhalten, eine neuartige secreted-Klotho-Promotor-Gentherapie zur Behandlung der Amyotrophen Lateralsklerose (ALS). Diese Designation bringt bedeutende Vorteile mit sich, darunter Steuergutschriften f眉r klinische Studien, GDUFA-Geb眉hrenbefreiungen und 7 Jahre Marktexklusivit盲t.
KLTO-202 zielt auf Motoneuronerkrankungen ab und verwendet einen muskel-spezifischen Promotor namens 鈥濪esmin鈥�, um das s-KL-Gen und -Protein zu exprimieren und die Therapie gezielt an der neuromuskul盲ren Verbindung zu verabreichen. Das Unternehmen hat Konzeptnachweise in zwei Tiermodellen abgeschlossen und beginnt mit der Herstellung, w盲hrend es regulatorische Gespr盲che mit der FDA und EMA plant.
- Received FDA Orphan Drug Designation providing 7 years of market exclusivity
- Successful completion of proof of concept studies in two animal models
- Tax credits and fee waivers granted through Orphan Drug Designation
- Targeting a rare disease market with 5,000 new cases annually in the US
- KLTO-202 is not yet approved for human use by any regulatory authority
- Early stage of development with clinical trials still pending
- Faces challenges in treating a universally fatal disease
Insights
FDA's Orphan Drug Designation for KLTO-202 provides Klotho significant regulatory advantages for their ALS treatment candidate, boosting development prospects.
The FDA's grant of Orphan Drug Designation (ODD) to Klotho Neurosciences for KLTO-202 represents a significant regulatory milestone in the company's ALS treatment development program. This designation offers substantial benefits including tax credits for clinical trial costs, GDUFA user fee waivers, and critically, seven years of market exclusivity upon approval鈥攊ndependent of patent protection.
KLTO-202 (s-KL-AAV.myo) utilizes a novel approach combining a secreted-Klotho promoter with gene therapy delivered to the neuromuscular junction. The therapy aims to address both the neurological damage and offer neuroprotection through therapeutic concentrations of the s-KL protein in blood, brain, and muscle tissues.
The company has completed proof-of-concept studies in two animal models and is initiating manufacturing of the candidate. Their regulatory strategy includes upcoming consultations with both FDA and EMA to establish the development pathway.
For context, ALS affects fewer than 200,000 people in the US with approximately 5,000 new diagnoses annually, qualifying it as a rare disease. The ODD validates Klotho's scientific approach while providing regulatory advantages to accelerate development for this universally fatal condition that currently has limited treatment options.
The FDA grants Orphan Drug Designation to drugs and biologics that are intended for safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the
"Receiving the Orphan Drug Designation for s-KL-AAV.myo for the early treatment of ALS underscores the importance of bringing new treatment options to patients suffering from this rare, universally fatal disease" said Dr. Joseph聽Sinkule, Klotho's Chief Executive Officer. "My cousin Karen died from this horrific disease. We aim to deliver the first gene replacement therapy addressing the neurologic insult resulting in motor neuron damage and the potential neurologic protection induced by providing therapeutic blood, brain, and muscle concentrations of the s-KL protein. After the FDA's review of the data leading to the Orphan Drug Designation, we believe this ODD designation provides strong validation of our science and our approach to treat this disease" concludes Dr. Sinkule.
ALS is sometimes referred to as Lou Gehrig's disease. Lou Gehrig, who played for the New York Yankees for 17 years in the 1920s and 1930s, stunned players and fans by retiring from baseball at the age of 36 after being diagnosed with ALS. Prior to this diagnosis, Gehrig played in a record-breaking 2,130 consecutive games, was referred to as the "Iron Horse," and was considered one of the greatest baseball players of all time. Less than two years later, at the age of 37, Gehrig died of complications from ALS. ALS is also referred to as Motor Neuron Disease in the
Klotho Neurosciences will have completed "proof of concept" studies in two animal models of human ALS and the Company is currently initiating manufacturing of the ALS-targeted product candidate, followed by meetings with the
KLTO-202, the company's lead product candidate targeting motor neuron diseases and muscular dystrophies, is composed of a muscle-specific promoter called "desmin," driving the expression of the s-KL gene transcript and s-KL protein, with targeted delivery of the gene therapy to the neuromuscular junction - the interface between the spinal cord and the muscles. At this time, KLTO-202 is not approved for human use by any regulatory authority.
About Klotho Neurosciences, Inc.
Klotho Neurosciences, Inc. (NASDAQ: KLTO), is a biogenetics company focused on the development of innovative, disease-modifying cell and gene therapies using a protein derived from a patented form of the "anti-aging" human Klotho gene (s-KL), and its novel delivery systems to transform and improve the treatment of neurodegenerative and age-related disorders such as ALS, Alzheimer's, and Parkinson's disease. The Company's current portfolio consists of its proprietary cell and gene therapy programs using DNA and RNA as therapeutics and genomics-based diagnostic assays. The Company is managed by a team of individuals and advisors who are highly experienced in biopharmaceutical product development and commercialization.
For more information, please visit the company's website at .
Investor Contact and Corporate Communications: - Jeffrey LeBlanc, CFO
[email protected]
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements. These statements are made under the "safe harbor" provisions of the
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FAQ
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