PDE

Phase 2b study will evaluate the safety and efficacy of gemlapodect (NOE-105) in patients with Tourette syndromeBASEL, Switzerland, Oct. 30, 2024 (GLOBE NEWSWIRE) -- Noema Pharma AG, a clinical-stage neuroscience-based company, today announced the first patients have been dosed in a global Phase 2b study evaluating the safety and efficacy of gemlapodect (NOE-105) in Tourette syndrome. NOE-TTS-201 (NCT06315751) is a Phase 2b, 12-week, randomized, double-blind, placebo-controlled study that is designed to examine the safety and efficacy of gemlapodect. The trial will enroll 140 patients with Tourette syndrome in multiple centers in the US and Europe. Patients will be treated with up to 15 mg of gemlapodect once daily, or a matching placebo. The primary endpoint is the Yale Global Tic Severity Scale (YGTSS) Total Tic Score. “Patients with Tourette syndrome carry an incredible burden related to the expression of motor and vocal tics with limited treatment options,” said Ilise Lombardo, M.D., Chief Executive Officer of Noema Pharma. “I am happy to announce the progression of the development of gemlapodect in Tourette syndrome with the dosing of the first patients in our Phase 2b trial.” About Tourette Syndrome Tourette syndrome is a neurodevelopmental disorder in the wider spectrum of tic disorders, consisting of motor and vocal abrupt and brief movement or sound which is preceded by a local or generalized urge/premonitory sensation. TS impacts over a half-million individuals and families in the US alone, as well as millions of individuals worldwide. About NOE-105/gemlapodect NOE-105/gemlapodect is a potential first-in-class phosphodiesterase-10A (PDE10A) inhibitor in mid-stage clinical development as a dopamine modulator for Tourette syndrome. NOE-105 has been well-tolerated in studies with healthy volunteers and in patients with Tourette syndrome. NOE-105 has the potential to achieve a more targeted effect by modulating dopamine signaling exclusively in the medium spiny neurons of the striatum in the brain, while limiting off-target effects compared to other dopamine antagonists. About Noema Pharma Noema Pharma is a clinical-stage biotech company advancing a portfolio of transformative therapeutics utilizing first-in-disease approaches targeting neuroscience-based conditions with high unmet need. Noema has four programs currently in active Phase 2 clinical trials evaluating pain in Trigeminal Neuralgia, seizures in Tuberous Sclerosis Complex, Tourette syndrome and vasomotor symptoms plus associated symptoms of menopause with readouts expected in 2025. Noema was founded by leading venture capital firm Sofinnova Partners and is supported by current investors including Forbion, Gilde Healthcare, Invus, Jeito Capital, Polaris Partners and UPMC Enterprises. Learn more at www.noemapharma.com. Investor Contact: Precision AQAlex Lobo212-698-8802alex.lobo@precisionaq.com

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

关注并星标CPHI制药在线 10月28日，康方生物宣布其IL-17靶向单抗古莫奇单抗（AK111）治疗中重度斑块型银屑病的关键3期研究（AK111-301）继2023年达到主要终点后，已于近期完成研究并获得优异的长期疗效和安全性结果。 数据显示：古莫奇单抗单药治疗52周，PASI75应答率近100%，sPGA0/1应答率近90%，PASI90应答率超90%，PASI100应答率超70%。而且，停药8周后，较第52周比较，PASI75、PASI90、sPGA0/1应答率约97%患者维持应答。 2023年公布的数据显示：古莫奇单抗单药治疗12周，PASI75应答率近95%，sPGA0/1应答率近90%；PASI90应答率超80%，PASI100应答率近50%。 安全性方面，古莫奇单抗治疗中重度斑块型银屑病不良事件发生率与安慰剂组相当，安全性良好。 对比12周和52周的数据可以发现，古莫奇单抗治疗中重度斑块型银屑病在长期维持以及进一步改善疗效方面的表现突出。 古莫奇单抗是康方生物自主研发的一款新型人源化IL-17A单抗，被开发用于治疗中重度斑块型银屑病、强直性脊柱炎等自免疾病。虽然古莫奇单抗治疗中重度斑块型银屑病疗效优异，但面对诸多竞争对手，未来前景如何还充满不确定性。 仅IL-17靶点，目前国内NMPA已批准5款IL-17A靶向单抗和1款IL-17R靶向单抗，详见下表。除了比吉利珠单抗，其他4款IL-17A靶向单抗和IL-17R靶向单抗均被批准用于治疗中重度斑块状银屑病，其中仅司库奇尤单抗、依奇珠单抗已进入国家医保。 疗效上，因未进行头对头研究，这5款单抗治疗斑块状银屑病药物无法直接比较。其中赛立奇单抗是全球唯一获批上市的全人源（IgG4亚型）IL-17A靶向单抗，已公布的其治疗中重度斑块状银屑病第12周的PASI90应答率为74.4%，PASI100应答率为50.2%。夫那奇珠单抗是我国首 个获批的国产重组抗IL-17A人源化单抗，已公布的其治疗中重度斑块状银屑病的数据显示：第12周，PASI75应答率为93.6%，sPGA0/1应答率为71.8%，PASI90应答率为76.8%，PASI100应答率为36.6%；第52周时，PASI100应答率达到63.1%，sPGA0应答率为63.3%。 给药频率上，司库奇尤单抗密集期需注射5次，维持期每4周给药一次。依奇珠单抗密集期需注射7次，维持期每4周给药一次。夫那奇珠单抗密集期仅需注射3次，维持期每4周给药一次。赛立奇单抗密集期需注射7次，维持期每4周给药一次。布罗利尤单抗密集期需注射3次，维持期每2周给药一次。 整体来看，已上市的IL-17靶向药各有优势，疗效、治疗成本、给药便捷性等均是影响其市场表现的因素。 此外，古莫奇单抗在IL-17领域还有不少潜在竞争者，如三生国健的SSGJ-608(IL-17A)、鑫康合生物/丽珠单抗的XKH004(IL -17A/17F)、君实生物的JS005(IL-17A)、华奥泰的HB0017（IL-17A）。这几款药物针对斑块状银屑病的临床试验均已进入3期阶段，其中三生国健SSGJ-608治疗成人中重度斑块状银屑病的关键注册性3期临床研究已完成揭盲及最终统计分析，所有主要疗效终点（PASI 75和sPGA 0/1）、关键次要疗效终点（PASI 90、PASI 100和sPGA 0）和所有次要疗效终点均成功达到。 银屑病是一种遗传、环境协同作用的免疫介导的慢性复发性疾病，其中斑块状银屑病是最常见类型，约占80%。银屑病病程长、易复发、治疗困难，患者需接受终生治疗。除了IL-17靶向药，中重度斑块状银屑病领域还批准了多款新药，详见下表。 从上述表格可以看出，中重度斑块状银屑病新药从TNF-α抑制剂逐步向白介素（IL）抑制剂过渡，而且还出现了一些小分子化药，如PDE4抑制剂和TYK2抑制剂。这些药物的迭代也改变了中重度斑块状银屑病患者的给药方式，包括皮下注射和口服给药。 此外，目前国内还有多款在研中重度斑块状银屑病新药进入临床后期，其中大部分为国产创新药。康方生物在该领域的另外一款药物——依若奇单抗对标强生的乌司奴单抗，2023年9月在国内申请上市。 总结 据弗若斯特沙利文数据，近年来中国银屑病药物市场规模增长强劲，已从2018年的6.04亿美元迅速增长至2022年的14.36亿美元，2025年有望达到32.55亿美元。生物制剂是中重度斑块状银屑病的主要系统用药，目前国内获批治疗中重度斑块状银屑病的生物制剂数量逐年攀升，但作用靶点相对集中，如IL-17、IL-23。此外，PDE4抑制剂、TYK2抑制剂也开始进军中重度斑块状银屑病市场。值得一提的是，除了生物制剂作用靶点的迭代，国产生物制剂的获批数量也在不断增加，这其中除了创新生物制剂，还包括生物类似物，而且在研产品中，国产创新药占比不断攀升。 END 来源：CPHI制药在线 声明：本文仅代表作者观点，并不代表制药在线立场。本网站内容仅出于传递更多信息之目的。如需转载，请务必注明文章来源和作者。 投稿邮箱：Kelly.Xiao@imsinoexpo.com ▼更多制药资讯，请关注CPHI制药在线▼ 点击阅读原文，进入智药研习社~