Latest news with #ClinicalTrial
Yahoo
2 days ago
- Business
- Yahoo
Xenon Pharmaceuticals Inc (XENE) Q2 2025 Earnings Call Highlights: Strategic Advances and ...
Cash and Cash Equivalents: $624.8 million as of June 30, 2025. Marketable Securities: Included in the cash and cash equivalents total. Cash Burn: Decrease from $754.4 million as of December 31, 2024, indicating cash used in operations. Funding Outlook: Sufficient cash to fund operations into 2027. Warning! GuruFocus has detected 3 Warning Signs with XENE. Release Date: August 11, 2025 For the complete transcript of the earnings call, please refer to the full earnings call transcript. Positive Points Xenon Pharmaceuticals Inc (NASDAQ:XENE) completed patient recruitment for the X-TOLE2 Phase 3 clinical trial, a significant milestone for the development of azetukalner. The company anticipates having sufficient cash to fund operations into 2027, demonstrating strong fiscal management. Azetukalner has shown promising results in Phase 2b trials with compelling placebo-adjusted efficacy in focal onset seizure patients. Xenon Pharmaceuticals Inc (NASDAQ:XENE) has initiated multiple Phase 3 studies for azetukalner in major depressive disorder and bipolar depression, expanding its clinical development program. The company has a strong presence in the epilepsy community, with plans to present data at upcoming international conferences, enhancing its scientific outreach and educational efforts. Negative Points The timeline for filing the NDA after the X-TOLE2 data readout is approximately six months, which may delay commercialization. There is uncertainty regarding the final number of patients to be randomized in the X-TOLE2 study, which could impact study outcomes. The company has not yet established a compassionate use program for azetukalner, despite interest from the medical community. Xenon Pharmaceuticals Inc (NASDAQ:XENE) faces potential challenges in the epilepsy market, including overcoming the inertia of established treatment practices. There are concerns about potential safety issues with the Nav1.7 program, although no cardiovascular signals have been observed in preclinical data. Q & A Highlights Q: Can you remind us how quickly you think you'll be able to file on the back of the top line FOS data? Also, could you talk about your confidence in the Nav1.7 program in pain regarding safety issues? A: We estimate approximately six months from topline data to filing the NDA for the X-TOLE2 data. Regarding the Nav1.7 program, we believe we have a good handle on potential safety issues, such as cardiovascular signals seen in earlier molecules. We haven't observed any cardiovascular signals in preclinical safety data and will monitor this in human clinical development. Q: For X-TOLE2, when do you expect all patients to be randomized, and what was the patient recruitment split US versus ex-US? A: We have completed patient recruitment for X-TOLE2, and the last patients will go through an eight-week baseline period before randomization. We haven't finalized the specific breakdown of US versus ex-US recruitment or the number of sites yet, but we aim to maintain consistency with the X-TOLE study. Q: Have you seen any compassionate use interest for azetukalner? A: Yes, there is constant interest in compassionate use for azetukalner, not just within neurology and psychiatry but beyond. We are having active discussions about when a compassionate use program could be implemented. Q: How are you thinking about the potential difference in dose response for the 15 and 25 mg doses in the Phase 3 study versus the Phase 2? A: We expect to see a dose response between 15 mg and 25 mg in the Phase 3 program, similar to the clear dose response observed in Phase 2. Our discussions with the FDA suggest having multiple doses on label to provide flexibility to the epilepsy community. Q: On the Nav1.7 program, once you establish proof of concept, would you consider exploring combinations with a Nav1.8? A: We are excited about the leadership position in Nav1.7 and are focused on establishing proof of concept. Long-term, we may consider different combinations of non-opioid mechanisms, including Nav1.8, but it's early to start planning those combinations. For the complete transcript of the earnings call, please refer to the full earnings call transcript. This article first appeared on GuruFocus.
Medscape
2 days ago
- Health
- Medscape
Brain Stimulation Promising for Progressive Aphasia
Transcranial magnetic stimulation (TMS) may augment standard language therapy to help slow the progression of primary progressive aphasia (PPA), a neurodegenerative disorder that erodes communication. In a randomized sham-controlled clinical trial, 6 months of active intermittent theta-burst TMS plus language therapy improved or mitigated decline in regional brain metabolism, trained language abilities, functional impairment, and neuropsychiatric symptoms in adults with PPA. The study was published online on August 11 in JAMA Network Open . Added Value? PPA is a heterogeneous clinical syndrome marked by progressive speech and/or language impairment. Most cases stem from frontotemporal degeneration or Alzheimer's disease. There are currently no effective drug treatments, although speech-language therapy has proven to be helpful. TMS can induce changes in cortical excitability, potentially promoting the reorganization of language networks, and has shown promise as adjunctive treatment for post-stroke aphasia. Previous studies examining the short-term effects of TMS on PPA reported 'encouraging' results, but the longer-term effects, beyond more than a few weeks of intervention, have not been examined, until now. For the study, the researchers led by Jordi Matias-Guiu, MD, PhD, with the Department of Neurology, Hospital Clínico San Carlos, Madrid, Spain, recruited 63 adults with early-stage PPA (42 women; mean age, 72 years). Participants were randomized (2:1) to either active intermittent theta-burst TMS or sham TMS for 6 months, immediately followed by evidence-based language therapy for PPA. The trial consisted of a 24-week treatment that included a 2-week intensive course, in which active TMS or sham TMS combined with language therapy was applied daily, followed by a maintenance phase in which the same stimulation was applied weekly for 22 weeks. Participants were assessed at baseline, 3 months after the start of the treatment, and at the end of the treatment (6 months following baseline). The main outcome was the standardized uptake value ratio (SUVR) on FDG PET imaging in the left hemisphere, assessed at baseline and at 6 months (immediately following the intervention). Feasible, Effective Option The primary outcome was positive with higher adjusted mean SUVR in the active group than in the sham TMS group (0.78 vs 0.77; P = .046). Active TMS was also associated with significant improvement on all secondary language outcomes at 6 months, including the mini linguistic state examination, with an adjusted mean difference at 6 months of 7.71 ( P = .002). Patients in the sham group worsened on this measure compared with those in the active group. Active TMS also led to improvement in confrontation naming of trained words, which improved by a mean 23.8 points in active recipients compared with sham TMS recipients. Functional independence also benefited, with daily-living scores falling (indicating better performance) by 5.4 points in the active group compared with the sham TMS group. Neuropsychiatric symptoms eased as well, with a 4-point advantage on the neuropsychiatric inventory scale. There were no significant differences in the number of adverse events. Adherence to treatment protocol was high (92%). 'Overall, these findings suggest that the combination of TMS and language therapy is a feasible and effective treatment option for PPA,' the researchers concluded. They said future studies should investigate the potential for TMS paired with an evidence-based speech-language intervention to sustain or extend these benefits beyond 6 months.
Yahoo
2 days ago
- Business
- Yahoo
Neurogene Reports Second Quarter 2025 Financial Results and Highlights Recent Updates
Announced design of Embolden™ registrational clinical trial of NGN-401 gene therapy for Rett syndrome; trial initiation activities underway Completed dosing in Phase 1/2 NGN-401 trial, and remains on track to report updated clinical efficacy and safety data in the second half of 2025 Cash runway into early 2028 NEW YORK, August 11, 2025--(BUSINESS WIRE)--Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced second quarter 2025 financial results and highlighted recent corporate updates. "In the first half of 2025, we made significant progress in our NGN-401 program for Rett syndrome. We completed dosing of the last five participants in the Phase 1/2 trial and received written agreement from the U.S. FDA on the key elements of the Embolden™ registrational trial, in which we have already begun initiation activities," stated Rachel McMinn, Ph.D., Founder and Chief Executive Officer of Neurogene. "We are pleased to be moving forward with Embolden, which was purposefully and rigorously designed to differentiate NGN-401 by evaluating participants as young as three years of age with a primary endpoint that incorporates measures that KOLs, caregivers and payors believe to be clinically meaningful. Our previously announced reallocation of capital provides us runway into early 2028, enabling us to focus our resources on advancement of this potential therapy for the patients and families who urgently need new treatment options." Dr. McMinn continued, "In leveraging the continual dialogue under the START program, the FDA encouraged the analysis of the Embolden primary endpoint to remain at 12 months as the basis for full approval, noting that a 6-month endpoint may not be considered clinically meaningful. In further maintaining the rigorous design of the Embolden trial, we are electing to dose the last planned participant from the Phase 1/2 trial as part of the registrational Embolden trial and add one more participant to complete the proposed sample size at 20 patients." Second Quarter 2025 and Recent Highlights, and Anticipated Milestones NGN-401 Gene Therapy for Treatment of Rett Syndrome Received written agreement from the U.S. Food and Drug Administration (FDA) on key elements of the Embolden registrational trial of NGN-401 and refined the sample size to propose 20 participants Initiated Embolden clinical trial activities to support the conversion of the Phase 1/2 trial to a registrational trial Completed enrollment in the Phase 1/2 trial, with the last 5 additional participants dosed in the first half of 2025 Remains on track to report updated clinical efficacy and safety data from the Phase 1/2 trial in the second half of 2025 Presented at scientific conferences the hemophagocytic lymphohistiocytosis (HLH) monitoring and treatment algorithm incorporated into the NGN-401 clinical trial, which has been acknowledged as valuable information by the Rett syndrome and gene therapy communities There has been no evidence of HLH/hyperinflammatory syndrome in any NGN-401 trial participant at the 1E15 vg dose level, as of the date of this press release Upcoming Events Stifel Biotech Summer Summit: Management will participate in a fireside chat at 12:00 p.m. ET on August 12 (not webcast) H.C. Wainwright Annual Global Investment Conference: Management will participate in a fireside chat at 2:30 p.m. ET on September 8 and participate in 1x1 meetings Second Quarter 2025 Financial Results Cash, Cash Equivalents and Short-Term Investments: Cash, cash equivalents and short-term investments as of June 30, 2025 were $274.5 million. We currently expect cash, cash equivalents and short-term investments to fund planned operations into early 2028. Research & Development (R&D) Expenses: R&D expenses were $19.4 million for the three months June 30, 2025 compared to $15.7 million for the three months ended June 30, 2024. The increase in R&D expenses for the three months ended June 30, 2025 was primarily driven by an increase in Rett syndrome clinical trial costs and employee-related expenses due to an increase in R&D headcount. General & Administrative (G&A) Expenses: G&A expenses were $6.7 million for the three months ended June 30, 2025 compared to $5.3 million for the three months ended June 30, 2024. The increase in G&A expenses for the three months ended June 30, 2025 was primarily driven by an increase in employee-related expenses due to an increase in stock-based compensation, headcount and other corporate expenses. Net Loss: Net loss was $22.0 million for the three months ended June 30, 2025 compared to $18.5 million for the three months ended June 30, 2024. About Neurogene The mission of Neurogene is to treat devastating neurological diseases to improve the lives of patients and families impacted by these rare diseases. Neurogene is developing novel approaches and treatments to address the limitations of conventional gene therapy in central nervous system disorders. This includes selecting a delivery approach to maximize distribution to target tissues and designing products to maximize potency and purity for an optimized efficacy and safety profile. The Company's novel and proprietary EXACT™ transgene regulation platform technology allows for the delivery of therapeutic levels while limiting transgene toxicity associated with conventional gene therapy. Neurogene has constructed a state-of-the-art gene therapy manufacturing facility in Houston, Texas. CGMP production of NGN-401 was conducted in this facility and will support pivotal clinical development activities. For more information, visit About NGN-401 NGN-401 is an investigational AAV9 gene therapy being developed as a one-time treatment for Rett syndrome. It is the first clinical candidate to deliver the full-length human MECP2 gene under the control of Neurogene's EXACT™ transgene regulation technology. EXACT technology is an important advancement in gene therapy for Rett syndrome, specifically because the disorder requires a treatment approach that enables targeted levels of MECP2 transgene expression without causing overexpression-related toxic effects associated with conventional gene therapy. NGN-401 was selected by the U.S. Food and Drug Administration (FDA) for its START Pilot Program and has also received Regenerative Medicine Advance Therapy (RMAT) designation, orphan drug designation, Fast Track designation and rare pediatric designation from the FDA. Neurogene was previously granted an INTERACT meeting with the FDA regarding the EXACT technology. NGN-401 also received Priority Medicines (PRIME) designation, orphan designation and advanced therapy medicinal product designation from the European Medicines Agency (EMA) and the Innovative Licensing and Application Pathway (ILAP) designation from the United Kingdom (UK) Medicines and Healthcare products Regulatory Agency (MHRA). Cautionary Note Regarding Forward-Looking Statements Statements in this press release are made as of the date of this press release. Neurogene does not undertake any obligation to make any updates to these statements to reflect events that occur or circumstances that arise after the date of this press release, except as may be required under applicable U.S. securities law. Statements in this press release which are not historical in nature are intended to be, and hereby are identified as, forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current expectations and beliefs of the management of Neurogene, as well as assumptions made by, and information currently available to, management of Neurogene, including, but not limited to, statements regarding: the therapeutic potential and utility, efficacy and clinical benefits of NGN-401; trial designs, clinical development plans and timing for NGN-401, including elements of the registrational clinical study trial design subject to final approval of the FDA, such as the proposed number of participants in the Embolden trial, and the timing of the conversion of the NGN-401 Phase 1/2 clinical trial to a registrational clinical trial, anticipated timing of additional updates for the Embolden registrational trial of NGN-401 for Rett syndrome; expected timing for additional interim data from the Company's NGN-401 Phase 1/2 trial for Rett Syndrome, expected future interactions with or positions of the FDA; and the time period over which existing cash resources may be sufficient to fund the Company's operations. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as "may," "will," "should," "would," "expect," "anticipate," "plan," "likely," "believe," "estimate," "project," "intend," "on track," and other similar expressions or the negative or plural of these words, or other similar expressions that are predictions or indicate future events or prospects, although not all forward-looking statements contain these words. Forward-looking statements are based on current beliefs and assumptions that are subject to risks, uncertainties and assumptions that are difficult to predict with regard to timing, extent, likelihood, and degree of occurrence, which could cause actual results to differ materially from anticipated results and many of which are outside of Neurogene's control. Such risks, uncertainties and assumptions include, among other things: the expected timing of additional results from the NGN-401 clinical trial; the potential for negative impacts to participants in the Phase 1/2 clinical trial of NGN-401 for the treatment of Rett syndrome; the risk that the Company may not be able to report data on the predicted timeline; risks related to the Company's ability to obtain regulatory approval for, and ultimately commercialize, its product candidates, including NGN-401; risks related to timing of initiating the Embolden trial of NGN-401 for Rett syndrome; and other risks and uncertainties identified under the heading "Risk Factors" included in Neurogene's Quarterly Report on Form 10-Q for the quarter ended June 30, 2025, filed with the Securities and Exchange Commission ("SEC") on August 11, 2025, and other filings that the Company has made and may make with the SEC in the future. Nothing in this communication should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that the contemplated results of any such forward-looking statements will be achieved. Forward-looking statements in this communication speak only as of the day they are made and are qualified in their entirety by reference to the cautionary statements herein. Except as required by applicable law, Neurogene undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise. This communication contains hyperlinks to information that is not deemed to be incorporated by reference into this communication. - Financial Tables Follow - Neurogene Inc. Condensed Consolidated Balance Sheet Data (In thousands of U.S. dollars) June 30,2025 December 31,2024 Assets Cash and cash equivalents $ 58,813 $ 136,586 Short-term investments 215,706 175,819 Other current assets 4,467 3,518 Non-current assets 18,330 19,807 Total assets $ 297,316 $ 335,730 Liabilities Current liabilities 15,440 15,157 Non-current liabilities 8,621 10,198 Total liabilities 24,061 25,355 Stockholders' equity 273,255 310,375 Total liabilities and stockholders' equity $ 297,316 $ 335,730 Neurogene Inc. Condensed Consolidated Statements of Operations (In thousands of U.S. dollars, except share information) Three Months EndedJune 30, Six Months EndedJune 30, 2025 2024 2025 2024 Revenue under licensing agreements — 925 — 925 Operating expenses: Research and development expenses 19,366 15,744 37,131 29,285 General and administrative expenses 6,715 5,315 14,869 10,553 Total operating expenses 26,081 21,059 52,000 39,838 Loss from operations (26,081 ) (20,134 ) (52,000 ) (38,913 ) Other income, net 4,065 1,642 7,337 3,500 Net loss $ (22,016 ) $ (18,492 ) $ (44,663 ) $ (35,413 ) Per share information: Net loss per share, basic and diluted $ (1.05 ) $ (1.09 ) $ (2.12 ) $ (2.09 ) Weighted-average shares of common stock outstanding, basic and diluted 21,055,378 16,941,524 21,025,996 16,922,630 View source version on Contacts Company Contact: Cara MayfieldVice President, Corporate Investor Contact: Melissa ForstArgot PartnersNeurogene@ Sign in to access your portfolio
Yahoo
3 days ago
- Health
- Yahoo
‘Remarkable' pancreatic cancer jab offers longer survival hope for patients
Pancreatic cancer patients have been given fresh hope after a new vaccine appeared to slow disease progression and increase survival. Researchers described the findings as 'remarkable' and have already started testing the efficacy of the jab among a larger group of pancreatic and bowel cancer patients. Pancreatic cancer has some of the poorest cancer survival rates because it is often not detected until it is advanced. Many patients see their cancer unremitting, even after undergoing traditional treatments including chemotherapy and radiotherapy. Now a new vaccine has been developed to help harness the body's immune system to find and attack cancer cells. The jab has shown promise in an early trial. Pancreatic and bowel cancers frequently carry a mutation in a gene called KRAS. This mutation plays a key role in tumour growth and scientists developed the jab to recognise and attack KRAS-mutant cancer cells. The jab is a new type of immunotherapy vaccine designed specifically to improve vaccine delivery to the lymph nodes, which act as filters for foreign substances in the body including cancer cells and infections. The phase 1 trial involved 20 patients with pancreatic cancer and five patients with bowel cancer. After an average follow-up time of almost 20 months, 68% of patients had developed strong immune responses specific to mutant KRAS tumour proteins. Though the study showed that some responded more favourably compared to others. Patients who had the strongest immune responses lived longer and stayed cancer free for longer than those with weaker responses. On average pancreatic cancer patients survived about two years and five months after receiving the vaccine, according to the study, which has been published in the journal Nature Medicine. At present, just three in 10 people diagnosed with the condition survive for a year. Meanwhile the average time before the disease returned, also known as recurrence free survival, was more than 15 months. While some cancer jabs are personalised to each patient, this jab, ELI-002 2P, has a single version which can be given to all patients. This 'off-the-shelf' version means that it can be manufactured in bulk and given more rapidly. Study lead Dr Zev Wainberg, from the University of California, Los Angeles, in the US, told the PA news agency: 'Pancreas cancer (patients) even after all standard treatments, such as chemotherapy and radiation, still have very high risks of the cancer coming back. 'Our results show in the group of patients who had profound immune responses (17/25 68%) achieved had longer survival than we have expected in this cancer, quite a remarkable finding to occur in a phase 1 trial.' He added: 'This is the first trial using a new platform, called AMP technology. 'The technology was invented by a material scientist engineer and immunology, Darrel Irvine, in his lab at MIT, and the platform was designed to improve vaccine delivery to lymph nodes which play a special role in the immune response.' Asked about next steps, he added: 'We launched a randomised phase 2 based on the initial results from the Amplify-201 study in January 2024. 'The accrual of the 144 patients participating was very rapid for this trial, which completed enrolment last December, and we are looking forward to the results as follow up continues.' Dr Chris Macdonald, head of research at the charity Pancreatic Cancer UK, said: 'Immunotherapy, and in particular cancer vaccines, hold so much opportunity in the treatment of pancreatic cancer, but so far the opportunity has not been realised. 'However in recent years, this is changing. This study is the latest in a rapidly progressing field. 'This early-stage study takes an important step, showing the effectiveness of an 'off-the-shelf' cancer vaccine, potentially breaking a reliance on having to generate a vaccine specifically for every individual patient. 'This approach saves time and is less costly and resource heavy, meaning more people could potentially benefit. 'The future is bright and if the momentum in this field continues to grow, more people could have the opportunity to benefit from immunotherapy, helping them to live better for longer.' Dr Dani Edmunds, research information manager at Cancer Research UK, added: 'Although we've helped to double cancer survival in the UK in the past 50 years, progress has not been the same for pancreatic cancer which remains hard to treat. 'Therefore, it's promising to see that vaccines could help people with pancreatic and bowel cancer live cancer-free for longer. 'The results suggest that the vaccine can boost the immune system against cancer in some people following standard treatment. 'These people survived and stayed free from disease for longer than people who didn't get as strong an immune boost following vaccination. 'Larger controlled trials are needed to confirm these initial findings about the benefits of the vaccine. 'More research is needed to understand why some people benefit from the vaccine while others don't so that we can make sure we're beating cancer for everyone.'
Yahoo
17-05-2025
- Business
- Yahoo
Tectonic Therapeutic Presents Complete Results for Positive Phase 1b Clinical Trial of TX45 in Patients with Group 2 Pulmonary Hypertension in HFpEF in Late-Breaking Presentation at ESC Heart Failure 2025
Data confirmed TX45's tolerability profile and improvements in left ventricular function and pulmonary hemodynamics in patients with Group 2 Pulmonary Hypertension in Heart Failure with preserved Ejection Fraction ('PH-HFpEF') In new echocardiographic analysis, TX45 treatment resulted in sustained hemodynamic effects for 29 days TX45 improved cardiac and pulmonary hemodynamics in PH-HFpEF patients across a range of left ventricular ejection fractions ('LVEF'), including LVEF≥50% and LVEF 41-49% WATERTOWN, Mass., May 17, 2025 (GLOBE NEWSWIRE) -- Tectonic Therapeutic, Inc. (NASDAQ: TECX) ('Tectonic') today announced the complete results from Part A of the Phase 1b clinical trial of TX45 in patients with Group 2 Pulmonary Hypertension in Heart Failure with preserved Ejection Fraction ('PH-HFpEF'), which are being presented in a late-breaking, oral session at the European Society of Cardiology (ESC) Heart Failure 2025 Congress being held in Belgrade, Serbia. The results include the full cohort of 19 patients in Part A of the Phase 1b trial of TX45, Tectonic's lead asset and a long-acting relaxin therapy. Interim data for 16 patients in the Phase 1b trial was previously reported in a press release on January 30, 2025. The complete data from Part A of the Phase 1b clinical trial confirmed the tolerability and hemodynamic effects of TX45 in patients with PH-HFpEF previously reported in the interim data. Based on the complete dataset in PH-HFpEF, a single intravenous dose of TX45 was well tolerated, with no serious or severe adverse events. In the overall study population, TX45 achieved a 19.0% reduction in pulmonary capillary wedge pressure ('PCWP'), an endpoint known to correlate with exercise capacity, morbidity and mortality in patients with heart failure. In the subpopulation with combined pre- and post-capillary pulmonary hypertension ('CpcPH') who have an elevated Pulmonary Vascular Resistance ('PVR') and more severe disease, TX45 demonstrated >30% reduction in PVR, which along with PCWP is correlated to exercise capacity and mortality in this patient population. The Phase 1b trial enrolled a patient population and evaluated hemodynamic endpoints which are similar to the ongoing APEX Phase 2 clinical trial ( NCT06616974). APEX is a 24-week clinical trial in PH-HFpEF with topline results expected in 2026. New hemodynamic data reported today from the Phase 1b clinical trial include the following: Echocardiograms were evaluated at baseline and at Days 2, 15 and 29. Following the administration of TX45, sustained improvements in echocardiogram endpoints were observed consistent with improved hemodynamics. Increased tricuspid annular plane systolic excursion/systolic pulmonary artery pressure (TAPSE/SPAP), a marker of PVR, and right ventricular fractional area of change (RVFAC), a marker of right ventricular function, were observed on all days post treatment compared to baseline demonstrating a sustained effect for 29 days after single dose administration. In the clinical trial, hemodynamics were analyzed across a range of left ventricular ejection fractions ('LVEF'), including LVEF≥50% and LVEF 41-49%. Both subpopulations showed similar improvements in PCWP (19.7% and 18.4%, respectively) and cardiac output (18.3% and 18.7%, respectively), as well as improvement in pulmonary hemodynamics following administration of TX45. Cardiac output increased numerically more in response to TX45 in patients with higher baseline PVR, with 16.8% improvement in patients with baseline PVR<2 wood units, 20.5% with baseline PVR≥2 and 24.5% with baseline PVR≥3. 'We are enthusiastic about the potential of TX45 as a treatment for patients with Group 2 pulmonary hypertension. In particular, we are very encouraged that the echocardiographic analysis demonstrated sustained hemodynamic effects of TX45 out to 29 days,' said Alise Reicin, M.D., President and Chief Executive Officer of Tectonic. 'Additionally, the positive and consistent hemodynamic effects across a range of LVEF confirmed that TX45 is well positioned to address the full spectrum of PH-HFpEF patients. We look forward to the topline data from Part B of the Phase 1b study in another type of pulmonary hypertension patients, those with Heart Failure with reduced Ejection Fraction (HFrEF), expected in the second half of 2025.' Marcella K. Ruddy, M.D., Chief Medical Officer of Tectonic commented, 'These results from the Phase 1b study show the promising therapeutic profile of TX45, offering patients a potential best-in-class therapy for Group 2 pulmonary hypertension, a disease with high morbidity, mortality and no approved treatments. Additionally, these data continue to support our hypothesis that TX45 may provide the greatest benefit to patients with CpcPH, based on data showing strong improvement in pulmonary hemodynamics and cardiac output relative to baseline PVR.' Highlights from complete Phase 1b Part A results Within the cohort of 19 patients with PH-HFpEF enrolled in the Phase 1b open label clinical trial of TX45, 9 patients had CpcPH, as measured by PVR>2 Wood units. Hemodynamic measures evaluating left ventricular function included PCWP, Cardiac Output ('CO') and Stroke Volume ('SV'). Hemodynamic measures evaluating the pulmonary vasculature included PVR, Total Pulmonary Resistance ('TPR') and mean Pulmonary Artery Pressure ('mPAP'). Safety Results: TX45 was well tolerated with no serious or severe adverse events, discontinuations, infusion reactions or drug-related adverse events. There were no clinically significant changes in vital signs, physical exam or safety laboratory values. Transient asymptomatic decreases in blood pressure were observed over the first 24 hours after TX45 dosing. Hemodynamic Results: TX45 administration resulted in meaningful improvement in both left ventricular function and pulmonary hemodynamics, representing a differentiated profile for TX45 compared to other PAH drugs that are pulmonary vasodilators but have not shown improvement in left ventricular function and have not shown efficacy in PH-HFpEF. TX45 achieved the following improvements in left ventricular function: PCWP decreased 19.0% [95% CI, -26.1% to -11.9%]. CO increased 18.5% [95% CI, 10.2% to 26.9%]. TX45 achieved the following improvements in pulmonary hemodynamics: PVR decreased 32.0% [95% CI, -35.9% to -28.1%] and 35.5% (95%CI, -38.6% to -32.5%) in the subgroup of patients with baseline PVR≥2 and baseline PVR≥3, respectively. TPR decreased 28.7% [95% CI, -34.1% to -22.1%] in the overall population. Mean pulmonary artery pressure decreased 16.8% [95% CI, -20.8% to -12.8%] in the overall population. As a relaxin therapeutic, the differentiated mechanism of TX45 improved both left ventricular function and pulmonary hemodynamics, which most strongly matches the more severe pathophysiology of patients with CpcPH. About the TX45 Phase 1b clinical trial in Group 2 pulmonary hypertension The Phase 1b open label clinical trial is designed to evaluate the safety and hemodynamic effect of single doses of TX45 in patients with Group 2 pulmonary hypertension. Part A evaluated the effect of TX45 in PH-HFpEF and Part B will evaluate effects of TX45 in Pulmonary Hypertension in Heart Failure with reduced Ejection Fraction ('PH-HFrEF'). The design of the clinical trial is as follows: after obtaining informed consent, a right heart catheter, which is the gold standard for the measurement of cardiopulmonary hemodynamics, is inserted and baseline measurements are obtained, an intravenous dose of TX45 is administered, and hemodynamic effects are evaluated over 8 hours post dose. Participants are then followed for 45 days post dose for safety and exploratory biomarker endpoints. Part A of the trial has completed. Part B enrollment is ongoing with topline data expected in the second half of 2025. About Group 2 Pulmonary Hypertension in HFpEF The World Health Organization has defined 5 groups of pulmonary hypertension ('PH'). Tectonic is focused on the Group 2 subtype, a condition that develops due to left-sided heart disease, specifically Pulmonary Hypertension in Heart Failure with preserved Ejection Fraction ('PH-HFpEF'). In patients with PH-HFpEF, chronic heart failure leads to increased blood pressure in the pulmonary arteries, exerting severe strain on the right side of the heart, which adapts poorly to the increased pressure. This increased pulmonary pressure gradually causes worsening exercise capacity, shortness of breath and right-sided heart failure which can lead to death. PH-HFpEF is further segmented based on pulmonary hemodynamics into Isolated, post-capillary PH ('IpcPH') and Combined pre- and post-capillary PH ('CpcPH'). CpcPH is more severe, accounts for about one third to one half of the 1.4 million PH-HFpEF patients in the U.S. and is characterized by additional, abnormal changes to the pulmonary vasculature, leading to an increase in Pulmonary Vascular Resistance ('PVR'). Although several Group 1 PH (Pulmonary Arterial Hypertension, 'PAH') medications have been explored in Group 2 PH, to date, no medications have been approved for its treatment. About TX45, a long-acting Fc-relaxin fusion protein TX45 is an Fc-relaxin fusion protein with optimized pharmacokinetics and biophysical properties that activates the RXFP1 receptor, the G-protein coupled receptor target of the hormone relaxin. Relaxin is an endogenous protein, expressed at low levels in both men and women that is a pulmonary and systemic vasodilator with lusitropic, anti-fibrotic and anti-inflammatory activity. In normal human physiology, relaxin is upregulated during pregnancy where it exerts vasodilative effects, reduces systemic and pulmonary vascular resistance and increases cardiac output to accommodate the increased demand for oxygen and nutrients from the developing fetus. Relaxin also exerts anti-fibrotic effects on pelvic ligaments to facilitate delivery of the baby. About Tectonic Tectonic is a biotechnology company focused on the discovery and development of therapeutic proteins and antibodies that modulate the activity of G-protein coupled receptors ('GPCRs'). Leveraging its proprietary technology platform called GEODe™ (GPCRs Engineered for Optimal Discovery), Tectonic is focused on developing biologic medicines that overcome the existing challenges of GPCR-targeted drug discovery and harness the human body to modify the course of disease. Tectonic focuses on areas of significant unmet medical need, often where therapeutic options are poor or nonexistent, as these are areas where new medicines have the potential to improve patient quality of life. Tectonic is headquartered in Watertown, Massachusetts. For more information, please visit and follow on LinkedIn. Forward-Looking StatementsThis press release contains 'forward-looking statements' within the meaning of the 'safe harbor' provisions of the Private Securities Litigation Reform Act of 1995. All statements in this press release other than statements of historical facts are 'forward-looking statements.' These statements may be identified by words such as 'aims,' 'anticipates,' 'believes,' 'could,' 'estimates,' 'expects,' 'forecasts,' 'goal,' 'intends,' 'may,' 'plans,' 'possible,' 'potential,' 'seeks,' 'will' and variations of these words or similar expressions that are intended to identify forward-looking statements, although not all forward-looking statements contain these words. Forward-looking statements in this press release include, but are not limited to, statements regarding: the design, objectives, initiation, timing, progress and results of current and future preclinical studies and clinical trials of Tectonic's product candidates, including the ongoing Phase 1b and Phase 2 clinical trials for its lead program, TX45, in Group 2 PH-HFpEF. These forward-looking statements are based on Tectonic's expectations and assumptions as of the date of this press release. Each of these forward-looking statements involves risks and uncertainties that could cause Tectonic's clinical development programs, future results or performance to differ materially from those expressed or implied by the forward-looking statements. Many factors may cause differences between current expectations and actual results, including: the potential that success in preclinical testing and earlier clinical trials does not ensure that later clinical trials will generate the same results or otherwise provide adequate data to demonstrate the efficacy and safety of a product candidate; the impacts of macroeconomic conditions, including the conflict in Ukraine and the conflict in the Middle East, heightened inflation and uncertain credit and financial markets, on Tectonic's business, clinical trials and financial position; unexpected safety or efficacy data observed during preclinical studies or clinical trials; clinical trial site activation or enrollment rates that are lower than expected; Tectonic's ability to realize the benefits of its collaborations and license agreements; changes in expected or existing competition; changes in the regulatory environment; the uncertainties and timing of the regulatory approval process; and unexpected litigation or other disputes. Other factors that may cause Tectonic's actual results to differ from those expressed or implied in the forward-looking statements in this press release are identified under the heading 'Risk Factors' in Tectonic's Quarterly Report on Form 10-Q filed with the SEC on May 8, 2025, and in other filings that Tectonic makes and will make with the SEC in the future. Tectonic expressly disclaims any obligation to update any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise, except as otherwise required by law. For more information, please visit and LinkedIn. CONTACT: Contacts: Investors: Dan Ferry LifeSci Advisors daniel@ (617) 430-7576 Media: Kathryn Morris The Yates Network kathryn@ (914) 204-6412Error 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
