SpliSense Announces FDA and EMA Grant Orphan Drug Designation to SPL84-23-1 for the Treatment of Cystic Fibrosis

JERUSALEM, Jan. 4, 2022 /PRNewswire/ -- SpliSense, a biopharmaceutical company developing transformative mRNA-altering therapies for cystic fibrosis (CF) and other pulmonary diseases, today announced that the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have both granted orphan drug designation (ODD) to SPL84-23-1, an antisense oligonucleotide (ASO) targeting the 3849 mutation in the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) gene associated with a severe form of CF lung disease.

Orphan-drug designation is granted by the FDA to a drug or biologic intended to treat a rare disease or condition, which generally includes a disease or condition that affects fewer than 200,000 individuals in the U.S. ODD granted therapies entitle companies to development incentives including tax credits for qualified clinical trials, exemptions from certain FDA application fees, and the potential of seven years of marketing exclusivity in the event of regulatory approval.

EMA is a decentralised agency of the EU responsible for the scientific evaluation, supervision and safety monitoring of medicines in the EU. Orphan designation is a status assigned to a medicine intended for use against a rare condition. Orphan medicines benefit from ten years of market exclusivity and additional incentives. 

"The orphan designations we received for our lead drug candidate both from the FDA and EMA are important milestones toward addressing the urgent, unmet needs of patients living with cystic fibrosis," said Gili Hart, PhD, CEO, SpliSense. "We are looking forward to initiating the clinical trial with SPL84-23, that is expected to commence in mid 2022 in multiple centers in the US, Europe and Israel."

About SPL84-23-1

SPL84-23-1 is an antisense oligonucleotide (ASO) targeting a specific splicing defect in CFTR, the 3849 mutation, which generates an aberrant splice site in the CFTR pre-mRNA, resulting in the inclusion of 84 intronic nucleotides as a cryptic exon in the CFTR mRNA. This 84bp cryptic exon contains an in-frame stop codon that leads to degradation of a significant fraction of the mRNA as well as to the production of truncated non-functional CFTR protein. Treatment with SPL84-23-1 ASO has shown in preclinical models to enable production of a fully functional CFTR protein and therefore has the potential to treat the CF disease associated with the 3849 mutation.

SpliSense utilizes short, precisely targeted proprietary RNA stretches called ASOs to correct various mutations in the CFTR mRNA. In particular, the ASO binds to the mutated CFTR pre-mRNA in the desired spot, leading to the elimination/masking of the mutated region in the mRNA and allowing the cell to produce functional CFTR proteins. The ASOs are administered directly and preferentially to the lungs via inhalation where it is taken up by the lung cells and drive the production of corrected CFTR mRNA and eventually functional CFTR proteins. SpliSense utilizes proprietary algorithms to support the design of optimized ASOs, thereby maximizing efficiency and reducing the potential for undesired off-target effects.

About Cystic Fibrosis

Cystic fibrosis (CF) is a genetic disease that leads to respiratory infections and disabilities and affects over 90,000 people worldwide. It is caused by mutations in the CFTR gene, leading to dysfunctional CFTR proteins. Disease-causing CFTR protein variants create irregularities in the transport of ions, generating mucus obstructions, chronic infection, inflammation and even respiratory failure. Over 2000 variants in the CFTR gene have been identified, and over 400 are known to cause disease. The past decade has seen a dramatic change in CF care. However, approved CFTR modulators do not support all patients with CF or offer a cure for the disease. Thus, new strategies of medication development are essential to address non-responsive mutations.

About SpliSense

SpliSense is a biopharmaceutical company focused on transformative mRNA-altering treatments targeting unmet needs in pulmonary diseases. by its pioneering platform harnesses ASOs. The Initial of focus SpliSense is on high unmet need, orphan indications (CF) with subsequent expansion to larger, non-orphan pulmonary indications (muco- obstructive diseases). SpliSense's pipeline includes innovative therapies in various development stages, from discovery to IND enabling studies. For additional information, please visit our website at www.splisense.com.

Company contact:
Tsipi Haitovsky
Global Media Liaison
SpliSense
+972-52-5989-892
[email protected]

SOURCE SpliSense