10126 Torino (TO)
Contact person: Marco Malisani
Phone: 0039 0116706425
Founding Year: 2011
Employees in R&D: 0
Fields of business: Pharma / Biopharma
PI3K, cystic fibrosis, lung inflammation. KIT2014
KITHER is an academic spin-off of the University of Turin, located in Turin in the 2i3T Incubator of the University
of Turin, hosted by the Molecular Biotechnology Centre (MBC) of Turin, and associated to the BioPMed Piemonte Innovation cluster. Our mission consists in the development of new products for targeted therapies, in particular to fight
airway disease and targeting PI3Ks with safety. The team includes Emilio Hirsch (Full
Professor at the University of Torino, with high knowledge in studying the role of PI3K enzymes in chronic inflammation and cancer), Marco Kevin Malisani (Business executive with extensive experience in funding and managing start-up companies
in Italy, Great Britain and South East Asia), Alberto Bardelli (Professor at the University of Torino, Researcher and co-founder of Horizon Discovery, a successful British biotech company), Gian Cesare Tron (Professor at the University of Piemonte Orientale, chemist with experience in multicomponent reactions drug synthesis), and Alessandra Ghigo (Assistant Professor at the
University of Turin, with strong expertise on the role of PI3K in lung pathology).
Cystic Fibrosis (CF) is a lethal congenital disease, affecting 75 000 people worldwide with a prevalence in Europe of 1/8000. The underlying cause of CF is a mutation in the “cystic fibrosis transmembrane conductance regulator” (CFTR) gene, encoding a channel
expressed by epithelial secretory cells. Life expectancy of CF patients is around 30 years due to a progressive dysfunction of the respiratory system, in which the reduced activity of the channel results in airway obstruction, infection and inflammation eventually leading to respiratory failure. Therapies mainly delay symptoms, but the situation is changing due to the recent approval of drugs that restore
expression of function of CFTR. However, the clinical efficacy is still unsatisfactory, as patients carrying the most prevalent mutation, F508del only show small improvements in lung function. Therefore, there is urgent need to invest in development of new effective means of treating CF. Kither Biotech recently patented a cell permeable peptide derived from the enzyme PI3K? (WO/2016/103176) that functions
as a CFTR activator for inhaled therapy and that is significantly more effective than the gold-standard currently available CFTR openers.
The preclinical assessment of the compound named KIT2014 shows that it is safe and effective in animal models as well as
in human cell-based organoids. Strikingly, the peptide not only opens the CFTR but, at the same time, also reduces airway obstruction and inflammation, two effects of critical benefit to CF patients. KIT2014 (that received the Orphan Drug Designation by EMA in 2017), in combination or even alone, will contribute to prolonging life-expectancy and improvement of quality of life across the life
span.Therefore, we intend to bring this potential new orphan drug to a combined phase I/IIa-b studies in CF patients involving key clinical sites in Europe as well as leading patient organizations. If successful, patients will not only benefit in terms of life quality, life-long treatment with the drug will very likely also have a significant effect on life expectancy.
The cystic fibrosis therapeutics market size was USD 3,560.5 million in 2016. CFTR modulators are are the largest segment. The market is expected to reach USD 13.9 billion by 2025, mainly due to fast growth of the CFTR-modulators segment.
CF market Current treatment regimens for CF are complex and time consuming. Therefore, adherence is often not complete. Moreover, most of the available therapies achieve only symptomatic relief and lack a mechanistic rationale. The two main products on the market, developed by Vertex Pharma, are extremely expensive and their efficacy is limited.
Thus,the demand for more effective treatment based on CFTR modulators among CF patients is rapidly increasing, and high-throughput
screening of compounds is time demanding and cost consuming. On the contrary, approaches based on an experimentally-confirmed mechanistic rationale (as in the case of KIT2014) are much more affordable.