CFTR

Study to evaluate the safety and tolerability of KIT2014, an inhaled peptide therapy designed to enhance Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) function, act as a bronchodilator, and reduce inflammation KIT2014 is being developed as a CFTR enhancer, acting as an add-on treatment to existing CFTR modulators, targeting improved lung function and reduced inflammation in Cystic Fibrosis patients TURIN, Italy, Oct. 30, 2024 /PRNewswire/ -- Kither Biotech, a clinical-stage biotech company focused on innovative therapies for respiratory diseases, today announced the start of a Phase 1 clinical study of KIT2014, a novel inhaled peptide therapy for Cystic Fibrosis (CF). KIT2014 is designed as an add-on therapy to existing CFTR modulators and works by enhancing CFTR gating, promoting bronchodilation, and reducing inflammation through PDE3/4 inhibition. "The initiation of this Phase 1 study marks a significant step forward for Kither Biotech and the CF community," said Dr. Dimitrios Goundis, CEO of Kither Biotech. "KIT2014 addresses mucus obstruction, chronic inflammation, and bronchoconstriction-three critical challenges that affect CF patients even with current CFTR modulators. We are excited to begin assessing the potential of KIT2014 to improve treatment outcomes." About the KIT2014 Study This Phase 1 double-blind, placebo-controlled study will primarily evaluate the safety and tolerability of single and multiple doses of KIT2014 in healthy adult volunteers, followed by an assessment of pharmacokinetics. For more information, visit clinicaltrials.gov (NCT06659757). About Cystic Fibrosis Cystic fibrosis is a life-limiting genetic disease that affects approximately 130,000 people worldwide. It is caused by mutations in the CFTR gene, which lead to defective chloride ion transport across cell membranes, resulting in the buildup of thick, sticky mucus in the lungs. This mucus impairs mucociliary clearance, promotes chronic bacterial infections, and triggers persistent inflammation, leading to progressive lung damage. Unmet Medical Need for KIT2014 Despite significant advances from CFTR modulators, many CF patients continue to experience persistent inflammation and recurring infections, severely impacting lung function and overall health. Neutrophil-driven inflammation, marked by elevated levels of neutrophil elastase, remains a major contributor to lung damage in CF patients. KIT2014 addresses these issues with an innovative solution that directly targets this inflammation, reduces neutrophil activation, and provides an additional therapeutic benefit that complements existing CF treatments. KIT2014 has the potential to improve significantly long-term outcomes for CF patients. Mechanism of Action of KIT2014 KIT2014 is a peptide-based therapy designed to target these fundamental issues by acting on multiple pathways in CF lung disease. Delivered through inhalation, it allows direct targeting of the lungs. By inhibiting phosphodiesterase 3/4 (PDE3/4), it elevates cyclic adenosine monophosphate (cAMP) levels. In bronchial epithelial cells, this mechanism enhances CFTR gating and improves chloride ion transport, while in airway smooth muscle cells, it acts as a bronchodilator. Additionally, KIT2014 reduces neutrophil-driven inflammation through PDE3/4 inhibition. Together, these mechanisms position KIT2014 as a promising add-on to existing CFTR modulator therapies. About Kither Biotech Kither Biotech is a clinical-stage biotech company focused on developing innovative therapies for respiratory diseases. Kither Biotech's lead program, KIT2014, is an innovative inhaled peptide therapy aimed at enhancing CFTR function, providing bronchodilation, and reducing inflammation in CF patients. By targeting multiple pathways in CF lung disease, Kither Biotech is committed to addressing unmet medical needs and improving the quality of life for patients suffering from severe respiratory conditions. The company has successfully raised $26 million to date from investors including Claris Ventures, 2Invest, 3B Future Health, CDP Venture Capital, Italian Angels for Growth, Club degli Investitori, Ersel and ACE Venture. For more information, please visit and follow us on LinkedIn. SOURCE Kither Biotech WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

The US Food and Drug Administration (FDA) has granted rare paediatric disease (RPD) designation to Enterprise Therapeutics’ investigational cystic fibrosis therapy. Estimated to affect more than 100,000 people globally, cystic fibrosis is a genetic disease that causes sticky mucus to build up in the lungs, digestive system and other organs in the body. Symptoms usually begin in early childhood and vary between patients, but the condition gets progressively worse over time and causes increasing damage to the affected organs. Enterprise’s ETD001, which targets the sodium channel in the airway epithelium to increase the hydration and clearance of mucus, has already demonstrated a well-tolerated profile in a phase 1 trial of healthy participants and has been shown to be long-acting in pre-clinical studies. A phase 2a trial is now aiming to assess the safety and efficacy of the candidate in patients who are ineligible for, or are not receiving, cystic fibrosis transmembrane conductance regulator modulator therapy. FDA RPD designation aims to incentivise drug development for serious or life-threatening diseases that affect fewer than 200,000 people in the US and primarily impact patients aged under 18 years. Under the programme, a sponsor that receives an approval for a RPD product may qualify for a priority review voucher (PRV) that can be redeemed to expedite the review of a different product, or be sold or transferred to another sponsor. John Ford, Enterprise’s chief executive officer, said: “As we progress through our phase 2a trial of ETD001, this RPD designation will further support our mission to advance this novel approach for treating people with cystic fibrosis with the highest unmet medical need, as rapidly as possible.” Annabella Amatulli, head of regulatory affairs at the company, added: “The RPD designation will give Enterprise access to valuable incentives and support from the FDA during the development of ETD001, including the eligibility to request a PRV at the time of marketing approval.” The FDA’s decision comes just two months after Enterprise announced that the first cystic fibrosis patient had been dosed in its phase 2a trial, which Ford said represented “an incredible milestone”.

– Phase 1b study will evaluate the safety and tolerability of multiple ascending doses of RCT2100 in people with cystic fibrosis – MENLO PARK, CA, USA I September 26, 2024 I ReCode Therapeutics, a clinical-stage genetic medicines company using tissue-specific delivery to power the next wave of mRNA and gene correction therapeutics, today announced that the first patient has been dosed in a Phase 1b study evaluating RCT2100, an investigational inhaled mRNA therapy for people with cystic fibrosis (CF). CF is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, impacting approximately 130,000 people worldwide. Although recent advancements in CFTR modulator therapies have improved outcomes for many people with CF, those with certain mutations remain underserved because these mutations prevent the production of functional CFTR protein, making current treatments ineffective. “The initiation of our Phase 1b study of RCT2100 in cystic fibrosis marks a significant milestone for us and is a step closer to offering new hope to those who cannot benefit from existing CF therapies,” said Shehnaaz Suliman, M.D., MBA, M.Phil., chief executive officer of ReCode Therapeutics. “We are immensely grateful for the ongoing support and partnership of the CF patient community and are focused on accelerating enrollment to bring this potential new treatment to those in need as quickly as possible.” Formulated using ReCode’s proprietary Selective Organ Targeting (SORT) lipid nanoparticle (LNP) platform, RCT2100 is designed to deliver CFTR mRNA directly to target cells in the lungs, instructing them to produce a functional version of the CFTR protein that is missing in some individuals with CF. By providing this CFTR mRNA, the therapy aims to address the root cause of CF, potentially restoring CFTR protein function in the lungs rather than just managing the symptoms. ReCode recently received global regulatory authorization to proceed with the RCT2100 Phase 1b study in the United States (U.S.), United Kingdom (UK), and Europe. The study is currently enrolling at sites in the Netherlands, with future expansion planned for France, the U.S. and the UK. For more information, please visit CF-Clinical-Studies.com . About Cystic Fibrosis Cystic fibrosis (CF) is a progressive genetic disease that causes persistent lung infections and respiratory failure. CF is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. An estimated 105,000 people have been diagnosed with CF across 94 countries, according to the Cystic Fibrosis Foundation. The absence or dysfunction of the CFTR protein results in a defect in airway hydration, which leads to excessive mucus buildup in the lungs. It also creates a mucociliary clearance defect, recurrent infections, inflammation, respiratory failure, and other complications. Despite advancements in CFTR modulator treatments, approximately 10% of the CF community have genetic mutations that do not benefit from these life-changing therapeutics. About ReCode Therapeutics ReCode Therapeutics is a clinical-stage genetic medicines company using precision delivery to power the next wave of mRNA and gene correction therapeutics. ReCode’s proprietary Selective Organ Targeting (SORT) lipid nanoparticle (LNP) platform enables highly precise and targeted delivery of genetic medicines directly to the organs, tissues, and cells implicated in disease, enabling improved efficacy and potency. ReCode’s lead programs include RCT2100 for the treatment of the 10% of cystic fibrosis patients who have genetic mutations in the CFTR gene that do not respond to currently approved CFTR modulators and RCT1100 for the treatment of primary ciliary dyskinesia caused by pathogenic mutations in the DNAI1 gene. RCT1100 and RCT2100 are inhaled disease-modifying mRNA-based therapies formulated using the SORT LNP delivery platform. ReCode is expanding its pipeline to develop potential therapies for other rare and common genetic diseases, including musculoskeletal, central nervous system, liver, and infectious disease indications. ReCode has been recognized by the San Francisco Business Times and Silicon Valley Business Journal as a Best Place to Work. For more information, visit www.recodetx.com and follow us on LinkedIn and Instagram . SOURCE: ReCode Therapeutics