P2X3 receptor

First Patient Dosed in Phase 2A Dose-Finding, Proof-of-Concept RESTEADY Trial Evaluating SASS-001 in a Sub-Population of Patients with Sleep Apnea with a Central Component; Top-line Data Expected in 1H 2026 SASS-001, a Novel Drug Combination of Sivopixant and a second compound, is the first clinical stage asset from Apnimed's Joint Venture with Shionogi Both Phase 3 Trials (SynAIRgy and LunAIRo) evaluating AD109 in Mild, Moderate and Severe Obstructive Sleep Apnea (OSA) patients of all body types remain on track for top-line results expected in Q2 and Q3 2025, respectively CAMBRIDGE, Mass., April 7, 2025 /PRNewswire/ -- Apnimed, Inc., a pharmaceutical company building the industry-leading portfolio of first-in-class oral drug candidates that address the root causes of obstructive sleep apnea (OSA) and other sleep-related breathing diseases, today announced that the first patient has been dosed in the Phase 2A RESTEADY trial evaluating the novel oral drug combination, SASS-001, in an underserved population of patients suffering from sleep apnea with a central component. Top-line results from the study are expected in the first half of 2026. SASS-001 represents the first clinical stage asset from Shionogi-Apnimed Sleep Science, LLC (SASS), Apnimed's joint venture with Shionogi & Co., Ltd. (Shionogi). "With the first patient dosed in the Phase 2A RESTEADY trial, both Apnimed and SASS have reached an important milestone – the advancement of a scientific hypothesis to clinical development," commented Larry Miller, MD, Chief Executive Officer of Apnimed. "Today's announcement represents the combined efforts of the Apnimed and Shionogi teams, highlighting their respective strengths in sleep medicine drug development and drug discovery." RESTEADY is a Phase 2A, multicenter, double-blind randomized, controlled, dose escalation, parallel-arm, safety and efficacy study evaluating SASS-001 in adult patients suffering from sleep apnea with a central component (NCT06776432). The study is expected to enroll approximately 60 patients from up to 10 U.S. clinical sites. Patients will be randomized in a 3:1 ratio to either dose-escalating SASS-001 or a placebo arm. Patient participation in the study is approximately 10 weeks, with trial measures to include sleep-clinic polysomnography and at-home sleep evaluations. Apnimed's most advanced clinical program, AD109, also continues to progress following the conclusion of enrollment in both Phase 3 clinical trials, LunAIRo and SynAIRgy. The Company continues to anticipate the availability of top-line data from both trials in Q2 and Q3 2025, respectively. AD109 is being studied in obese and non-obese adult patients with mild, moderate and severe OSA. About SASS and SASS-001 SASS is a joint venture of Apnimed and Shionogi focused on accelerating the discovery, preclinical and clinical development of novel pharmacologic solutions designed to address the complex pathology of sleep apnea and other sleep related diseases. SASS-001 is an investigational oral clinical candidate under development by SASS for sleep apnea with a central component. The candidate is a combination of sivopixant, a selective P2X3 receptor antagonist, and a second compound. About Sleep Apnea with a Central Component Sleep apnea with a central component is characterized by the concomitant presence of obstructive and central apneas. In central apneas, breathing temporarily stops due to absence of the signal from the nervous system to respiratory muscles to initiate a breath. This mechanism is different from the neuromuscular dysfunction that is thought to cause OSA, in which a breath is initiated but airflow is prevented by a physical occlusion of the airway despite contraction of respiratory muscles. Central and obstructive apneas often coexist in an individual patient, particularly in those with concomitant cardiovascular diseases such as atrial fibrillation and heart failure. About Obstructive Sleep Apnea Obstructive sleep apnea (OSA) is a serious, chronic sleep-related breathing disease in which the upper airway repeatedly collapses during sleep, leading to intermittent oxygen deprivation. It is caused by two overlapping mechanisms: neuromuscular dysfunction during sleep and predisposing anatomic abnormalities. It has been demonstrated that OSA affects people of all body types, ages and genders —there is no single "face" of the disease. An estimated 80 million people in the United States and 1 billion people worldwide suffer from OSA. An individual with OSA can experience hundreds of sleep apnea events in a single night, each one reducing the blood oxygen levels and negatively impacting cellular functions vital to normal health and function. Failure to effectively treat OSA increases the risk of serious long-term health consequences, such as high blood pressure, cardiovascular disease and type 2 diabetes, along with impacts on quality of life, including daytime fatigue and impaired judgement. Yet, the majority of those diagnosed with OSA refuse, abandon, or underutilize treatment. Currently, no available pharmacological treatments directly address the underlying neuromuscular dysfunction that is present in all people with OSA. About Apnimed Apnimed is a privately held clinical-stage pharmaceutical company dedicated to transforming the treatment landscape for sleep-related breathing diseases. We believe the introduction of simple, once-nightly oral drugs will dramatically expand diagnosis and the reach of treatment for people with OSA. OSA, like other common chronic diseases such as diabetes or hypertension, would benefit from having multiple drugs with differing mechanisms to more fully address the heterogeneity of disease pathophysiology. Apnimed envisions a new era where novel oral therapies simplify intervention, expand the reach of diagnosis and treatment, and help more people get the oxygen and restorative sleep needed to thrive. Based in Cambridge, Mass., Apnimed is advancing its growing pipeline of oral pharmaceutical product candidates designed to improve oxygenation in individuals living with OSA and other sleep-related breathing disorders. Our lead candidate AD109 could become the catalyst for a new oral treatment paradigm for OSA that has been historically limited to cumbersome devices or invasive surgeries. AD109 is currently in two Phase 3 clinical trials for the treatment of mild, moderate and severe OSA. Apnimed is developing several therapies through our joint venture with Shionogi & Co., Ltd., Shionogi Apnimed Sleep Science. Learn more at apnimed.com or follow us on X and LinkedIn. Media Contact: [email protected] Investor Contact: Ramzi Benamar [email protected] SOURCE Apnimed, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

With no FDA-approved treatments for refractory chronic cough (RCC), patients have long faced limited options – but Trevi Therapeutics is hoping to change that. On Monday, the company announced that its investigational oral therapy Haduvio (nalbuphine extended-release) met its primary endpoint in the Phase IIa RIVER trial, showing a statistically significant 57% placebo-adjusted reduction in 24-hour cough frequency.The crossover study, which enrolled 66 patients with RCC, demonstrated that Haduvio reduced cough frequency by 67% from baseline, compared to 10% with placebo. The therapy also showed similar efficacy in both moderate (10-19 coughs/hour) and severe (20+ coughs/hour) patient subgroups, with reductions of 68% and 66%, respectively.Trevi plans to discuss next steps with the FDA while continuing its parallel Phase IIb CORAL trial in chronic cough associated with idiopathic pulmonary fibrosis (IPF), which recently completed enrollment. Topline results there are due this half."The RIVER trial demonstrated that Haduvio was highly effective, and worked quickly at the lowest dose tested across a broad range of cough counts," said CEO Jennifer Good. "These results, coupled with our existing IPF chronic cough results, are further evidence of the effectiveness of Haduvio's central and peripheral KAMA mechanism in treating these difficult neurological cough conditions." Company shares were up over 41% on the news Monday.While Trevi's success may be capturing investor attention, other players have struggled to gain traction in the space. In 2023, Merck & Co. received a second complete response letter from the FDA for its oral P2X3 receptor antagonist gefapixant, despite the drug being approved in Europe and Japan. The US regulator had concluded that Merck's application "did not meet substantial evidence of effectiveness" for treating refractory or unexplained chronic cough.Meanwhile, GSK is hoping camlipixant, another P2X3 antagonist, can help turn the company's fortunes around (see – Vital Signs: Pharma's worst performers of 2024, and how they can turn the tide in 2025). Phase III results for the drug, which GSK acquired through its $2-billion deal for Bellus Health, are expected at the end of this year.In Phase IIb SOOTHE trial results published earlier this month in the American Journal of Respiratory and Critical Care Medicine, camlipixant demonstrated a 34% placebo-adjusted reduction in 24-hour cough frequency.Before the RIVER readout, Oppenheimer analysts said they "lean optimistic that Haduvio's performance in RIVER will open the door to a sizable second opportunity in RCC patients inadequately addressed by other therapies." Specifically, they highlighted that efficacy in the severe cough segment would "best inform its prospects for those in whom alternatives, including GSK's Phase III-stage camlipixant, are insufficient."

-- IkT-001Pro to be developed as a New Molecular Entity as the first oral, potentially disease-modifying treatment for a rapidly fatal disease primarily afflicting women between the ages of 30 and 60 -- Oct. 21, 2024 -- Inhibikase Therapeutics, Inc. (Nasdaq: IKT) (“Inhibikase” or “Company”), a clinical-stage pharmaceutical company developing protein kinase inhibitor therapeutics to modify the course of Cardiopulmonary and Neurodegenerative disease through Abelson Tyrosine Kinase inhibition, today announced the closing of a private placement of approximately $110 Million from the issuance and sale of shares of the Company’s common stock and accompanying warrants with potential aggregate financing of up to approximately $275 Million upon the full cash exercise of the warrants issued in the private placement, before deducting placement agent fees and offering expenses. The financing will fund execution of the Phase 2b ‘702’ trial in Pulmonary Arterial Hypertension (PAH) and for general corporate purposes. “This transformational financing for Inhibikase is a testament to the Company’s persistent scientific and medical focus to develop disease-modifying therapeutics that could improve the lives of millions of patients afflicted with cardiopulmonary and neurodegenerative diseases,” noted Dr. Milton Werner, President and Chief Executive of Inhibikase. “Imatinib, the active ingredient in IkT-001Pro, had previously been shown to provide significant clinical benefit and improve heart health as an oral, once a day therapy, albeit with an unacceptable safety and tolerability profile. We believe imatinib delivered as IkT-001Pro has the potential to ameliorate the safety and tolerability profile that precluded approval of imatinib more than 10 years ago. With this investment, top tier dedicated healthcare investment funds recognized the potential of IkT-001Pro to improve the lives of patients afflicted with PAH. As with risvodetinib in Parkinson’s-related diseases, IkT-001Pro is a product of the Company’s innovative medicinal chemistry program that designs and develops novel kinase inhibitor therapeutics.” Concurrent with the closing of this financing, three highly accomplished leaders in biopharmaceutical development and a Partner of Soleus Capital with deep experience in financing cardiopulmonary therapeutics development will join the Board of Directors at the closing of this transaction. Roberto Bellini (former Chief Executive of BELLUS Health Inc. and current Managing Partner of BSQUARED Capital), Amit Munshi (current Chief Executive of Orna Therapeutics and former CEO of Arena Pharmaceuticals), Arvind Kush (current CFO of Candid Therapeutics and former CFO of RayzeBio) and David Canner (Partner at Soleus Capital) have been appointed to the Inhibikase Board of Directors. In addition to joining the Board of Directors, Messers. Bellini, Munshi and Kush will each participate in the transaction. Mr. Bellini has been appointed Independent Chairperson of the Inhibikase Board coincident with the closing. In addition to these new Board members, Ms. Gisele Dion, Chair of the Audit Committee and Dr. Paul Grint, Chair of the Compensation Committee, have resigned from their service on the Board. The Company is grateful for their dedicated service, their thoughtful guidance and efforts that were an integral part of the program advancements leading to this investment. “I am excited about IkT-001Pro as a potential treatment for PAH patients,” noted Mr. Bellini. “I look forward to leveraging my recent experience at BELLUS Health to support the company’s strategic growth.” “I want to express my excitement to join the Inhibikase Board at this transformative moment for the company,” said Mr. Munshi. “Inhibikase, under Dr. Werner’s leadership, is poised to create a fundamental shift in the lives of patients suffering from these debilitating and fatal diseases,” said Mr. Munshi. “This financing is an important milestone for Inhibikase, and I’m thrilled to be joining the Board,” added Mr. Kush. “The capital from top tier investors is transformative and will enable the rapid development of IkT-001Pro for PAH.” Roberto Bellini is currently a Managing Partner at BSQUARED Capital, a family office biotech fund. He previously served as President and CEO of BELLUS Health between January 2010 and July 2023. Under his leadership BELLUS Health developed camlipixant, a P2X3 antagonist for chronic cough from preclinical to Phase 3. BELLUS Health was subsequently acquired by GSK plc in 2023. Mr. Bellini holds a Bachelor of Science in Biochemistry from McGill University. Amit Munshi is a 30-year industry veteran who is currently Chief Executive Officer of Orna Therapeutics and former President, Chief Executive Officer and board member of ReNAgade Therapeutics. Previously Mr. Munshi served as President and CEO of Arena Pharmaceuticals Inc., which was subsequently sold to Pfizer, Inc. Arvind Kush, the Chief Financial and Chief Business Officer of Candid Therapeutics, has over 15 years of finance and investment banking experience. Prior to Candid, Arvind was Chief Financial Officer at RayzeBio, where he helped the company raise over $160 million in a private financing in 2022, complete its $350+ million IPO in 2023 and close its $4.1B acquisition Bristol Meyers Squibb in February 2024. Arvind holds a Bachelor in Engineering in Computer Science from Visvesvaraya Technological University and an MBA from Goizueta Business School, Emory University. David Canner is a Partner at Soleus Capital. David has served on the board for a number of companies. Prior to joining Soleus in 2018, David completed his PhD at MIT in Biology after finishing his undergraduate studies at Princeton University where he received a BA in Chemistry. IkT-001Pro is a prodrug formulation of imatinib mesylate and has been developed to improve the safety of the first FDA-approved Abelson (Abl) kinase inhibitor, imatinib (originally marketed as Gleevec®). Imatinib is commonly taken for hematological and gastrointestinal cancers that arise from Abl kinase mutations found in the bone marrow or for gastrointestinal cancers that arise from c-Kit and/or PDGFRa/b mutations in the stomach; c-Kit, PDGFRa/b and Abl are all members of the Abelson Tyrosine Kinase protein family. In a previously published Phase 3 trial (the IMPRES trial, published in 2013 in the journal Circulation, volume 127, pages 1128-1138), imatinib was shown to induce a clinically meaningful improvement in 6 minute walking distance (6MWD) of approximately 40 meters and reduce pulmonary vascular resistance by more than 300 dyne-sec/cm, improve other measures of heart health and approximately 30% of participants actually got better with imatinib added to their standard-of-care at a dose of 400 mg once daily. IkT-001Pro has the potential to be a safer, better tolerated alternative for imatinib therapy in patients with PAH. In preclinical studies, IkT-001Pro was shown to be as much as 3.4 times safer and/or better tolerated than imatinib in non-human primates, reducing burdensome gastrointestinal and other on-dosing side effects that occur following oral administration. The 702 trial evaluating IkT-001Pro in PAH was IND-cleared for clinical entry at Phase 2b by the FDA on September 9, 2024. Imatinib delivered as IkT-001Pro was granted Orphan Drug Designation for Stable-Phase CML in 2018, and Orphan Drug Designation is presently under review for PAH. The securities (including the shares of common stock underlying the pre-funded warrants and warrants) were offered in a private placement under Section 4(a)(2) of the Securities Act of 1933, as amended (the “Securities Act”) and have not been registered under the Securities Act, or any state or other applicable jurisdictions’ securities laws, and may not be offered or sold in the United States absent registration or an applicable exemption from the registration requirements of the Securities Act and applicable state or other jurisdictions’ securities laws. This press release shall not constitute an offer to sell or a solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction. Any offering of the securities under the resale registration statement will only be made by means of a prospectus. Inhibikase Therapeutics, Inc. (Nasdaq: IKT) is a clinical-stage pharmaceutical company developing Abelson Tyrosine Kinase inhibitor therapeutics for Cardiopulmonary and Neurodegenerative disease. Inhibikase's multi-therapeutic pipeline includes its neurodegenerative disease portfolio led by risvodetinib in Parkinson’s disease, Multiple System Atrophy and other diseases that may arise from alpha-synuclein aggregate formation. Inhibikase’s cardiopulmonary disease portfolio is led by IkT-001Pro, a prodrug of imatinib mesylate, for Pulmonary Arterial Hypertension that will deliver imatinib in a form that the Company believes will provide a better patient experience with fewer on-dosing side-effects. The Company's RAMP™ medicinal chemistry program has identified several follow-on compounds to risvodetinib that could potentially be applied to other cognitive and motor function diseases of the brain. Inhibikase is headquartered in Atlanta, Georgia with offices in Lexington, Massachusetts. The content above comes from the network. if any infringement, please contact us to modify.