JERUSALEM, April 3, 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, non-CF bronchiectasis (NCFB) and idiopathic pulmonary fibrosis (IPF), today announced that the U.S. Food and Drug Administration (FDA) cleared the Investigational New Drug (IND) application for the initiation of a Phase 2 study for SPL84 in CF. SPL84 is the Company's lead antisense oligonucleotide (ASO) product for the treatment of people with CF carrying the 3849+10 kilobase (Kb) C->T splicing mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

The IND Clearance for the Phase 2 study initiation was granted following SPL84's favorable safety profile, supported by Phase 1 and preclinical toxicological and pharmacological data demonstrating the potential for full restoration of CFTR protein and activity, based on clinically predictive CF models.

The Company has also announced today that it has secured funding from the CF Foundation and other existing investors to support the SPL84 Phase 2 study and additional pulmonary programs. This represents the CF Foundation's third investment in SpliSense's SPL84 program.

"We are excited to initiate the Phase 2 study for SPL84 in CF with the support of the CF Foundation and other existing investors," said Gili Hart, PhD, Chief Executive Officer of SpliSense. " Although currently available CF therapies have managed to significantly increase life expectancy over the past few decades, there is still a significant unmet medical need for disease modifying treatments. Our lead product, SPL84, has been shown to fully restore CFTR activity in the CF gold standard pharmacological model. With the recent FDA IND clearance, we hope to be able to deliver, a life-changing treatment for people with CF carrying the 3849+10 Kb C->T mutation and expand our unique technology to additional CF mutations where there is a significant unmet need."

"In addition, SPL84 serves as proof-of-concept for our platform for the treatment of additional pulmonary diseases, including muco-obstructive diseases and IPF, which are expected to enter the clinic over the next 12 months," added Dr. Hart.

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.

About SPL5AC (Muco-Obstructive Diseases) and SPL5B (IPF)

SpliSense is advancing two preclinical ASO assets for the treatment of additional pulmonary diseases, including muco-obstructive diseases and IPF.  Patients with muco-obstructive lung disease are susceptible to severe mortality and morbidity, creating an unmet need for the development of an effective and potent agent in the treatment of mucin induced lung disease.  SPL5AC is an ASO that specifically lowers the level of MUC5AC expression and production in mucus, decreases mucus viscosity, eliminates accumulation in the airways, and reduces infection and inflammation as demonstrated in three proof of-concept disease animal models.

Chronic overexpression of MUC5B is suggested to be the single greatest risk factor for the development of IPF.  SPL5B is an ASO that lowers MUC5B expression and production avoiding accumulation of MUC5B mucin and plugs in the distil regions of the lungs.  By lowering MUC5B levels and plugs formation, SpliSense aims to slow or delay IPF progression and improve patient's clinical condition and quality of life.

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.

The Company's investors include Cystic Fibrosis Foundation, OrbiMed, Israel Biotech Fund, Integra Holdings and Biotel Limited.

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

Company contact:
Tsipi Haitovsky
Global Media Liaison
+972-52-5989-892
Tsipi@splisense.com

SOURCE: PR Newswire