Rare diseases are conditions that affect about 1 in 2,000 people. However, they are “rare” so to speak because if we consider the 700 million European inhabitants, rare diseases can potentially affect about 350,000 people.

A consistent part of rare diseases is represented by pulmonary ones. For example, cystic fibrosis (CF), which is the most common fatal genetic disease, and idiopathic pulmonary fibrosis (IPF), which is the most common and severe form of idiopathic interstitial pneumonia.^1,2

Caused by a CFTR gene mutation, cystic fibrosis is characterized by the reduction or loss of functional CFTR chloride channels, leading to the production of excessively thick mucus that obstructs the bronchus – causing infections, inflammations or respiratory failures – and other organs, such as the pancreas, interfering with the digestion.

Idiopathic pulmonary fibrosis, whose aetiology is still unknown, is characterized by scarring and hardening of pulmonary tissue which compromises lungs functionality. To this day there are no cures or treatments against this pathology to effectively reduce the scarring process.

Kither Biotech, a startup born from expertise in respiratory disease

Kither Biotech was founded in November 2011 as a spin-off of the University of Turin, initially providing a spectrum of in vitro and in vivo screening services to pharmaceutical companies.

Over the years, Kither Biotech has gained extensive expertise with murine preclinical models of respiratory diseases.

Pursuing this pathway, Kither Biotech became a biopharmaceutical startup and its members achieved significant scientific results by focusing on the modulation of signal transduction mediated through PI3K enzymes. They found two molecules with the potential to treat cystic fibrosis and idiopathic pulmonary fibrosis: KIT2014 and KITCL27, respectively.

Two molecules against cystic fibrosis and idiopathic pulmonary fibrosis

The patented peptide KIT2014 acts as a modulator of cyclic adenosine monophosphate (cAMP) levels, which are associated with cystic fibrosis pathogenesis. When inhaled, KIT2014 increases cAMP locally in bronchial epithelial cells to promote the opening of CFTR chloride channels, which are key to mucus hydration, whilst in lung smooth muscle and immune cells, cAMP elevation limits bronchoconstriction and neutrophil infiltration. In CF patients, treatment with KIT2014 is believed to restore the function of CFTR mutants by potentiating the effects of currently available CFTR modulators

The patented small molecule KITCL27 acts as a pan-inhibitor of class I PI3K enzymes, which are involved in the pathogenesis of idiopathic pulmonary fibrosis. Its action reduces the scarring of pulmonary tissue. KITCL27 offers two distinct advantages over classic PI3K inhibitors: it acts as a cell-permeable prodrug that is only active once it entered the target cells, with the active moiety staying within the cells because it loses its ability to cross cell membranes, and can be administered by inhalation to restrict its activity to the lungs.

State of the art

To this day, the two Kither Biotech molecules are in the pre-clinical development phase.

KIT2014 has already received the Orphan Drug Designation for the treatment of cystic fibrosis from the European Medicines Agency. Its promising proof-of-concept results have led Kither Biotech to raise in 2019 an initial Series A financing round worth €5.6 million to support its preclinical development.

In March 2022, a study describing the mechanism of action and proof-of-concept data of KIT2014 has been published in Science Translational Medicine. Also in March 2022, the company completed a Series B investment round of EUR 18.5 million. This financing round will enable Kither Biotech to carry on the development of KIT2014 into First-in-Human clinical trial and the pre-clinical development of KITCL27, and to expand and diversify the pipeline of products for the treatment of rare respiratory diseases.

“It is incredibly rewarding to work with the Kither Biotech team and to lead the company through preclinical development towards its first clinical trial in humans, with the potential to improve the condition of thousands of patients suffering from debilitating respiratory diseases.”

Dr Vincent Metzler, CEO of Kither Biotech

References

1. Cos’è la fibrosi cistica. Lega Italiana Fibrosi Cistica (LIFC), 2017.
2. Fibrosi polmonare idiopatica. Manuale MSD, 2021.

👉 Find more about Kither Biotech and others Bio4Dreams ecosystem companies.

Updates

May 2022

🌐 Kither Biotech joins Bio4Dreams network.