SPL84, an antisense oligonucleotide treatment for cystic fibrosis patients with a specific mutation, gains FDA Fast Track designation.

RT’s Three Key Takeaways:

  1. SpliSense’s investigational drug SPL84 has received Fast Track designation from the FDA, aimed at expediting the development and review process to address an unmet medical need for cystic fibrosis patients with the 3849+10 Kb C->T mutation.
  2. SPL84 is an antisense oligonucleotide therapy that targets and corrects specific mutations in the CFTR gene, potentially allowing cells to produce fully functional CFTR proteins. It is administered via inhalation directly to the lungs.
  3. Following the recent IND clearance, SpliSense is set to commence a phase 2 clinical trial for SPL84.

Clinical-stage biotechnology company SpliSense announced that the US Food and Drug Administration (FDA) has granted Fast Track designation to SPL84 for cystic fibrosis (CF).  

SPL84 is the company’s lead antisense oligonucleotide 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.

Mechanism of Action

SPL84 utilizes short, precisely targeted RNA stretches called antisense oligonucleotides 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.

Significance of Fast Track Designation

“Fast Track Designation for SPL84 is an important acknowledgment 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,” says Gili Hart, PhD, CEO of SpliSense, in a release.

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.

Next Steps in Clinical Development

The designation comes after the company recently received Investigational New Drug (IND) clearance from the FDA, which will allow SpliSense to start a phase 2 trial for SPL84.

“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,” says Hart in a release.

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