PureTech Health presents deupirfenidone data at ATS conference
PureTech Health (PRTC) delivered a late-breaking, oral presentation at the 2025 American Thoracic Society international conference in San Francisco. The presentation provided further insights into the Phase 2b ELEVATE IPF trial of deupirfenidone, highlighting the strength and durability of deupirfenidone's treatment effect through at least 52 weeks while maintaining favorable tolerability in patients living with idiopathic pulmonary fibrosis. Data presented from PureTech's global Phase 2b randomized, double-blind, active- and placebo-controlled, dose-ranging ELEVATE IPF trial demonstrated the potential for deupirfenidone to offer a differentiated treatment option for patients with IPF. In the trial, patients treated with deupirfenidone 825 mg three times a day experienced a slower rate of lung function decline, as measured by Forced Vital Capacity, at 26 weeks versus those who were treated with placebo. This statistically significant difference represents a robust treatment effect versus placebo of 80.9% for deupirfenidone 825 mg TID as a monotherapy. This result compares favorably against the rate of decline in FVC observed in the trial among patients treated with pirfenidone 801 mg TID versus placebo, which was consistent with previously reported pirfenidone clinical trial data and represents a treatment effect of 54.1%. Taken together, these results indicate that the treatment effect with deupirfenidone 825 mg TID was approximately 50% greater than that of pirfenidone 801 mg TID, based on their respective reductions in lung function decline versus placebo. In addition to these findings, deupirfenidone 825 mg TID also demonstrated a statistically significant benefit in delaying time to IPF progression4 compared to placebo, further supporting the clinical relevance of the treatment effect. Importantly, the rate of FVC decline observed over 26 weeks with deupirfenidone 825 mg TID was similar to the expected natural decline in lung function in healthy older adults. Furthermore, preliminary data from the ongoing open-label extension study suggest that this treatment effect is durable out to at least 52 weeks. As of May 9, a total of 101 patients had received at least 52 weeks of treatment with deupirfenidone. Those in the deupirfenidone 825 mg TID arm experienced a decline in FVC of -32.8 mL over the 52-week period, which is similar to the expected natural decline in lung function in healthy older adults over one year. These new data provide additional support for the durability of the treatment effect observed with this dose and reinforce its potential to stabilize lung function decline over time, while maintaining favorable safety and tolerability. Additional details from the ongoing OLE are expected to be shared in a future scientific forum. These results are further supported by preliminary pharmacokinetic data, which underscore the differentiated profile of deupirfenidone. Compared to pirfenidone 801 mg TID, deupirfenidone 825 mg TID resulted in an approximately 50% increase in drug exposure. Notably, the dramatically increased drug exposure did not result in an increase in tolerability challenges, suggesting that the deuterated structure of deupirfenidone may overcome the dose-limiting adverse events associated with pirfenidone. PureTech believes these PK results are consistent with the enhanced efficacy and favorable tolerability seen with deupirfenidone 825 mg TID in the trial. Deupirfenidone was well tolerated at both doses studied. Safety analyses included identification of the 16 most common treatment-emergent adverse events, defined as occurring in more than 5% of participants in at least one treatment group, and characterized the arm with the highest relative incidence of each of these 16 TEAEs. The pirfenidone 801 mg treatment group had the highest relative incidence for 9 of these TEAEs, followed by deupirfenidone 825 mg (5), placebo (2), and deupirfenidone 550 mg. PureTech is targeting a meeting with the U.S. Food and Drug Administration by the end of the third quarter of 2025 to discuss the results of the Phase 2b trial and align on a potential registrational pathway, with the goal of initiating a Phase 3 trial by the end of 2025. PureTech anticipates providing further guidance later this year following the finalization of the trial design and FDA interactions.
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
Yahoo
19 hours ago
- Yahoo
Trevi Therapeutics Announces Closing of $115 Million Underwritten Offering and Full Exercise by Underwriters of Option to Purchase Additional Shares
NEW HAVEN, Conn., June 5, 2025 /PRNewswire/ -- Trevi Therapeutics, Inc. (Nasdaq: TRVI), a clinical-stage biopharmaceutical company developing the investigational therapy Haduvio™ (oral nalbuphine ER) for the treatment of chronic cough in patients with idiopathic pulmonary fibrosis (IPF) and in patients with refractory chronic cough (RCC), today announced the closing of its previously announced underwritten public offering of 20,010,000 shares of its common stock at a public offering price of $5.75 per share, which includes 2,610,000 additional shares issued upon the exercise in full by the underwriters of their option to purchase additional shares of common stock in the public offering at the public offering price, less underwriting discounts and commissions. The total proceeds of the public offering were approximately $115.1 million, before deducting underwriting discounts and commissions and expenses payable by Trevi. All of the shares in the offering were sold by Trevi. Morgan Stanley, Leerink Partners, Stifel and Cantor acted as joint book-running managers for the offering. The shares were offered by Trevi pursuant to a shelf registration statement on Form S-3 (File No. 333-273030), which was filed with the Securities and Exchange Commission (SEC) on June 29, 2023, amended on August 11, 2023 and declared effective by the SEC on August 15, 2023. This offering was made only by means of a prospectus supplement and the accompanying prospectus that form a part of the registration statement. The final terms of the offering are disclosed in a final prospectus supplement which has been filed with the SEC. Copies of the final prospectus supplement and the accompanying prospectus may also be obtained from Morgan Stanley & Co. LLC, Attention: Prospectus Department, 180 Varick Street, 2nd Floor, New York, New York 10014, or by email at prospectus@ Leerink Partners LLC, Attention: Syndicate Department, 53 State Street, 40th Floor, Boston, MA 02109, or by telephone at (800) 808-7525, ext. 6105, or by email at syndicate@ Stifel, Nicolaus & Company, Incorporated, Attention: Syndicate, One Montgomery Street, Suite 3700, San Francisco, CA 94104, by telephone at (415) 364-2720 or by email at syndprospectus@ or Cantor Fitzgerald & Co., Attention: Capital Markets, 110 East 59th Street, New York 10022, or by email at prospectus@ 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 jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction. About Trevi Therapeutics, Inc. Trevi Therapeutics, Inc. is a clinical-stage biopharmaceutical company developing the investigational therapy Haduvio™ (oral nalbuphine extended-release) for the treatment of chronic cough in patients with idiopathic pulmonary fibrosis (IPF) and in patients with refractory chronic cough (RCC). Haduvio acts on the cough reflex arc both centrally and peripherally as a kappa agonist and a mu antagonist (KAMA), targeting opioid receptors that play a key role in controlling chronic cough. Nalbuphine is not currently scheduled by the U.S. Drug Enforcement Agency. Investor Contact Jonathan CarlsonTrevi Therapeutics, Inc.(203) 654 3286carlsonj@ Media Contact Rosalia Scampoli914-815-1465 rscampoli@ View original content to download multimedia: SOURCE Trevi Therapeutics, Inc. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
a day ago
- Yahoo
Seyltx Announces Positive Non-Human Primate (NHP) and Additional In Vivo Data for its Centrally Acting Inhibitor of GluN2B in Chronic Cough, Paving the Way for Optimized Later-Stage Phase 2 Trials
- Results will be presented at the American Cough Conference on June 7, 2025 - NHP receptor occupancy studies indicate increasing the dose in Phase 2 from 20 mg to 40-80 mg is likely to significantly increase cough suppression before receptor saturation - Oral administration in the Guinea pig model shows cough suppression up to 74% from baseline while staying under the NOAEL - Durability of cough suppression over time promises cough control with TID dose regimen and opens opportunity for once daily reformulation CAMBRIDGE, Mass., June 05, 2025--(BUSINESS WIRE)--Seyltx, Inc. a clinical-stage biotherapeutics company focused on developing innovative therapies to treat chronic cough and other neuronal hypersensitivity indications, today announced promising preclinical results for its lead compound, Ifenprodil. These new findings, to be presented at the American Cough Conference on June 7, 2025, offer critical insights into optimal dosing strategies and therapeutic durability, which will significantly inform the design of upcoming Phase 2 crossover studies planned for 2026. Ifenprodil, a NMDA antagonist with >200x selectivity towards the GluN2B subunit, is being developed to address refractory chronic cough (RCC), a significant unmet medical need, with a follow-on opportunity in chronic cough associated with Idiopathic Pulmonary Fibrosis (IPF-Cough). The company has already completed a Phase 2a trial in IPF-Cough at an exploratory low dose (20 mg TID) which yielded statistically significant reductions in objective cough count of 39% from baseline (at 12 weeks) and statistically significant improvement on multiple patient-reported outcomes. A NHP receptor occupancy (RO) study, conducted in collaboration with Yale University, was performed whereby rhesus monkeys underwent 120-minute PET scans following the intravenous injection of 135 ± 64 MBq (S)-[18F]OF-NB1 (tracer molecule). Ifenprodil was administered via 10-minute intravenous infusion at doses from 0.025 to 1 mg/kg (corresponding to oral human equivalent doses (HED) of 10-400 mg), with PET acquisition commencing 15' post-infusion. Dose-dependent GluN2B receptor occupancy was observed with a calculated EC50 (50% occupancy) of 0.04 mg/kg (HED of ~20 mg) and approximately 80% receptor occupancy predicted at 0.2 mg/kg (HED of ~80 mg). Doses between 40-80 mg TID are therefore expected to achieve 60-80% receptor occupancy, significantly enhancing therapeutic effects compared to the 20 mg TID dose used in the prior Phase 2a trial. In vivo studies conducted in Guinea pigs (GPs; 6-16 animals per group) assessed the antitussive effect of oral doses of Ifenprodil ranging from 1.5 mg/kg to 12 mg/kg (human equivalent doses of 20 to 160 mg). Ifenprodil demonstrated statistically significant (p<0.01) cough count reduction across the dose range of 39-74% following exposure to a tussive agent (1M citric acid). The lowest tested dose of 1.5 mg/kg (HED of 20 mg) resulted in a 39.1% reduction in cough count from baseline, consistent with the 39% reduction seen in the Phase 2a trial. Doubling this dose to an HED of 40 mg TID, which is well-tolerated in humans, improved the cough suppression to 56.0% reduction from baseline. A maximal reduction of 74.4% from baseline was observed at 12 mg/kg (HED 160 mg), a dose under the estimated NOAEL. The therapeutic effect proved durable, lasting up to 8 hours, even as systemic levels of Ifenprodil were cleared. This robust target engagement at the GluN2B subunit supports durable cough control with a TID (three times daily) dose regimen and opens the door for potential once-daily reformulation. These combined preclinical findings suggest an optimal human dose range of 40 to 80 mg for maximal therapeutic benefit, with the 80 mg dose yielding approximately 80% receptor occupancy, safely below levels that have shown adverse events in preclinical models. "These compelling preclinical results represent a significant leap forward in our understanding of Ifenprodil's potential to provide meaningful relief for patients suffering from chronic cough," said Dr. Dietrich Stephan, CEO of Seyltx. "The data not only confirms Ifenprodil's robust antitussive properties but also provides invaluable guidance on optimizing our dosing strategy for future clinical trials. We are excited to integrate these learnings into the design of our upcoming Phase 2 studies, bringing us closer to delivering a truly impactful therapy for this debilitating condition." Dr. Stephan further emphasized, "The promise of addressing GluN2B in the brain is that this target appears to be a central node in the cough reflex, likely providing significant cough control as a monotherapy without addictive potential across the population independent of etiology, if the preclinical data is translatable. The agent also has the potential to be used in combination with future peripherally acting therapies that might be approved." Seyltx plans to incorporate these key learnings into the design of its Phase 2 crossover studies, slated to commence in 2026. About Seyltx: Seyltx ( is a clinical stage biotherapeutics company focused on developing innovative therapies to treat chronic cough and other neuronal hypersensitivity indications. Its lead indication is refractory chronic cough, a significant unmet medical need, with a follow-on opportunity in chronic cough associated with Idiopathic Pulmonary Fibrosis (IPF-Cough). Seyltx's lead compound is Ifenprodil, an NMDA antagonist highly selective towards the GluN2B subunit, has completed a Phase 2a trial in chronic cough associated with IPF. Forward-Looking Statements: Certain statements contained in this news release, other than statements of fact that are independently verifiable at the date hereof, may constitute "forward-looking statements" within the U.S. Private Securities Litigation Reform Act of 1995, as amended, and other applicable securities laws. Forward-looking statements are frequently, but not always, identified by words such as "expects," "anticipates," "believes," "intends," "estimates," "potential," "possible," "projects," "plans," and similar expressions. Such statements, based as they are on the current expectations of management, inherently involve numerous important risks, uncertainties and assumptions, known and unknown, many of which are beyond Seyltx's control. View source version on Contacts Parag ShahChief Operating Officerpshah@ Connectez-vous pour accéder à votre portefeuille
Associated Press
a day ago
- Associated Press
Mediar Therapeutics Doses First Patient in Phase 2 WISPer Trial of MTX-463 for Idiopathic Pulmonary Fibrosis
First-in-class agent targets WISP1 in patients with IPF Primary endpoint in WISPer trial is change in Forced Vital Capacity (FVC) at 24 Weeks BOSTON, June 5, 2025 /PRNewswire/ -- Mediar Therapeutics, Inc., a clinical-stage biotechnology company advancing first-in-class therapies designed to halt fibrosis, today announced the first patient has been dosed in its Phase 2 WISPer clinical trial evaluating MTX-463, an investigational therapy for idiopathic pulmonary fibrosis (IPF). The study's primary endpoint is the change from baseline in forced vital capacity (FVC)—an important measure of lung function—at 24 weeks. The study aims to advance the development of a potential novel treatment for patients living with IPF, and targets the myofibroblast, the key pathogenic cell in fibrosis. Mediar recently entered into a global licensing agreement with Lilly to advance MTX-463 through the Phase 2 WISPer trial. IPF is a rare and progressive lung disease characterized by scarring and thickening of lung tissue, which leads to increasing shortness of breath and a persistent dry cough. Despite available treatments, there remains a high unmet medical need, with an average life expectancy of three to five years post-diagnosis. The WISPer trial is a randomized, double-blind, placebo-controlled 24-week Phase 2 study designed to evaluate the efficacy, safety, and tolerability of MTX-463 in patients living with IPF. 'Dosing the first patient in the WISPer trial represents an important step forward in the pursuit of new options to address the urgent needs of individuals living with IPF,' said Toby Maher, MD, PhD, Director of Interstitial Lung Disease, University of Southern California, and lead investigator in the Phase 2 clinical study. 'IPF is a devastating disease with limited treatment options. We are eager to evaluate the potential of MTX-463 to slow or halt disease progression and look forward to the insights this study will provide for patients and their physicians.' 'Today marks an important milestone in our ongoing commitment to pioneering novel therapies to impact people living with fibrotic diseases. We extend our gratitude to Dr. Ryan Klein and the dedicated team at NewportNativeMD whose expertise and collaboration have been instrumental in reaching this important step,' said Jeff Bornstein, MD, Chief Medical Officer, Mediar. 'The first patient dosed with this first-in-class anti-fibrotic marks the beginning of a new chapter in our research, and we look forward to working with our clinical sites as we continue to enroll the Phase 2 program.' Mediar is also independently advancing its two wholly owned programs to treat other fibrotic disorders. MTX-474, a first-in-class human IgG1 antibody designed to neutralize EphrinB2 signaling, recently completed its Phase 1 clinical study. Mediar anticipates initiating a Phase 2 trial for MTX-474 in systemic sclerosis in the second half of 2025. Mediar's third novel fibrosis program, targeting SMOC2 for renal fibrosis, is also advancing with a plan to nominate a clinical candidate in the first half of 2025. About MTX-463 MTX-463 is a first-in-class human IgG1 antibody developed against WNT1-inducible signaling pathway protein-1 (WISP1). WISP1 is a secreted matricellular protein shown to have a relevant role in fibrosis progression, is measurable in human blood, and correlates with disease severity. Data indicates that MTX-463 neutralizes WISP1-mediated fibrotic signaling and significantly reduced fibrosis in vitro and in various preclinical models. A Phase 1 study was recently completed and a Phase 2 study in patients with IPF is now open (NCT06967805). More information can be found at: About Mediar Therapeutics Mediar Therapeutics is pioneering a new approach to fibrosis treatment that aims to halt the disease at a different source – the myofibroblast, the key pathogenic cell in fibrosis that drives scarring, disease progression, and ultimately organ failure. Mediar was founded based on a deep understanding of the complex science underlying fibrosis progression. By combining novel targets with reliable, easily detectable blood biomarkers and familiar modalities, Mediar's goal is to bring forward novel anti-fibrotic therapies that potentially have a precision medicine approach. For more information, contact [email protected] or follow us on LinkedIn. Media Contact [email protected] View original content to download multimedia: SOURCE Mediar Therapeutics
