SpliSense Receives FDA Fast Track Designation for SPL84 for the Treatment of Cystic Fibrosis

2024-05-29

临床2期快速通道寡核苷酸

JERUSALEM, May 29, 2024 /PRNewswire/ -- SpliSense, a clinical-stage biotechnology company focused on the development of transformative RNA-based therapies for pulmonary diseases including cystic fibrosis (CF), muco-obstructive diseases like COPD, asthma and NCFB and idiopathic pulmonary fibrosis (IPF), today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to SPL84 for CF.

SPL84 is the Company's lead antisense oligonucleotide (ASO) product for the treatment of patients with CF carrying the 3849+10 kilobase (Kb) C->T splicing mutation in the transmembrane conductance regulator (CFTR) gene.

"Fast Track Designation for SPL84 is an important acknowledgement by the FDA of the critical need to find an effective treatment for CF patients carrying the 3849+10 kilobase (Kb) C->T mutation, a serious and life-threatening condition with very poor treatment options," said Gili Hart, PhD, CEO of SpliSense. "The recent IND clearance we received from the FDA together with this Fast Track designation for SPL84, currently being evaluated in a global Phase 2 study, will allow us to expedite the development of a potentially life-changing treatment for people with CF carrying the 3849+10 Kb C->T mutation, and expand our unique technology to additional pulmonary indication where there is a significant unmet need."

About Fast Track Designation

The FDA grants Fast Track designation to facilitate the development and expedite the review of medicines to treat serious conditions and fill an unmet medical need. Fast Track status allows for enhanced communication and collaboration between the FDA and drug developers, potentially speeding up the delivery of life-saving treatments to patients.

About Cystic Fibrosis (CF)

CF is a genetic multisystem disorder that originates from various mutations in the CFTR gene, which is responsible for the production of the CFTR protein, a chloride channel expressed in the lungs as well as in other tissues. The past decade has seen a dramatic change in CF care with the approval of new CFTR modulators. However, approved CFTR modulators do not support all people with CF and do not offer a cure for the disease. Thus, new strategies of therapeutic development are essential to address partially responsive and non-responsive people with CF, specifically those carrying the 3849+10 Kb C->T splicing mutation.

About SPL84

SpliSense utilizes short, precisely targeted proprietary RNA stretches called Antisense Oligonucleotides (ASOs) to modulate specific mRNA sequences or correct various mutations in the target mRNA. SPL84 binds specifically to the mutated CFTR RNA in the targeted sequence, leading to the modulation of the mutated region in the mRNA, potentially allowing the cell to produce fully functional CFTR proteins. SPL84 is administered directly and preferentially to the lungs via inhalation, where it is designed to be taken up by the lung cells and to drive the production of corrected CFTR mRNA and eventually fully functional CFTR proteins. SPL84, has been shown to fully restore CFTR activity in the CF gold standard pharmacological model. SPL84 is currently being evaluated in a global Phase 2 study for the treatment of patients with CF carrying the 3849+10 kilobase (Kb) C->T by weekly inhalation regimen.

About SpliSense

SpliSense is a biotechnology company focused on the development of RNA-based treatments for pulmonary diseases. The Company's pioneering platform harnesses ASOs for treatment of unmet cystic fibrosis mutations and large pulmonary diseases including muco-obstructive diseases and IPF. Through its novel ASO pulmonary approach, SpliSense aims to address specific genetic mutations or deficiencies, by restoring or reducing protein function, thus targeting the root cause of the disease.

For additional information, please visit our website at: https://splisense.com/

Company contact:

Tsipi Haitovsky

Global Media Liaison

+972-52-5989-892

[email protected]

SOURCE SpliSense

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