Latest news with #SATURN
Business Wire
4 days ago
- Business
- Business Wire
Halo Biosciences Announces Thorax Publication of Phase 2a SATURN Study Results in PH-ILD
PALO ALTO, Calif.--(BUSINESS WIRE)--Halo Biosciences ('Halo'), a clinical-stage biotechnology company developing extracellular matrix-targeted therapies, today announced publication of results from the Phase 2a SATURN study in Thorax. The study, conducted at Stanford University, evaluated 4-methylumbelliferone (4-MU) in patients with pulmonary hypertension, a highly progressive disease with significant unmet needs. The SATURN study, a Phase 2a randomized, double-blind, placebo-controlled trial, enrolled 16 patients with pulmonary hypertension. 4-MU was safe and well-tolerated throughout the 24-week treatment period. The primary hemodynamic measurement of change in pulmonary vascular resistance was not statistically significant. Among patients with PH-ILD, prespecified exploratory efficacy signals showed a mean improvement of 66 meters in six-minute walk distance and enhanced quality-of-life scores. These findings support further clinical evaluation of 4-MU as a potential disease-modifying therapy for inflammatory and fibrotic lung diseases. 'These clinical data reinforce the scientific rationale for targeting hyaluronan in fibrotic and inflammatory lung disease and highlight 4-MU's potential as a first-in-class, disease-modifying ECM-modulator for patients with serious conditions like PH-ILD,' said Paul Bollyky, M.D., professor of medicine at Stanford University and scientific co-founder of Halo Biosciences. 'This represents a meaningful step forward for individuals living with PH-ILD, a condition with limited treatment options and a high burden of disease.' HB-1614, Halo's lead investigational therapy, is a proprietary, oral formulation of 4-MU optimized for improved bioavailability and long-term use in patients with chronic lung conditions such as PH-ILD. By inhibiting hyaluronan synthesis, HB-1614 targets a key driver of extracellular matrix (ECM) remodeling involved in inflammation and fibrosis—processes central to disease progression in several debilitating diseases, including PH-ILD. 'We are proud to see the SATURN study featured in Thorax, validating our translational approach and marking a key milestone in our development of HB-1614,' said Anissa Kalinowski, chief executive officer of Halo Biosciences. 'We are thankful to Stanford University and sponsor investigators Roham Zamanian, M.D., and Vinicio de Jesus Perez, M.D., for their leadership of the SATURN trial, unlocking the potential of this new mechanism of action.' Halo Biosciences is progressing clinical development of HB-1614 and exploring partnership opportunities to accelerate its work in PH-ILD and other fibrotic conditions. The company holds exclusive intellectual property for its formulation and is positioned to optimize drug delivery, bioavailability and regulatory strategy. The full manuscript is now available online. To access the paper, visit: ABOUT HB-1614 HB-1614 is Halo Biosciences' lead investigational therapy, a proprietary formulation of 4-methylumbelliferone (4-MU) designed to inhibit hyaluronan synthesis, a key driver of inflammation and fibrosis in the ECM. By targeting this dysregulated pathway, HB-1614 offers a novel, disease-modifying approach for conditions like pulmonary hypertension associated with interstitial lung disease (PH-ILD). ABOUT PULMONARY HYPERTENSION Pulmonary hypertension (PH) is a progressive condition caused by elevated blood pressure in the arteries of the lungs, leading to reduced oxygen exchange, right heart strain, and eventual heart failure. i Symptoms include breathlessness, fatigue, and dizziness ii. PH diagnosis is often delayed and accompanied by comorbidities, with most patients diagnosed between the ages of 60 and 70 iii. When PH is associated with interstitial lung disease (PH-ILD), the course of disease is often more accelerated, with these patients facing a median survival of just 2 to 5 years. iv Currently, there is only one FDA-approved therapy for PH-ILD, iv leaving a significant unmet need for therapies that target the underlying mechanisms of disease progression. New approaches are urgently needed to improve outcomes and quality of life for this vulnerable patient population. ABOUT HALO BIOSCIENCES Halo Biosciences is a clinical-stage biopharmaceutical company targeting the extracellular matrix (ECM) to transform the treatment of diseases characterized by inflammation and fibrosis. It is headquartered in Palo Alto, CA. For more information, visit i Pulmonary Fibrosis Foundation. Pulmonary Hypertension Related to Interstitial Lung Disease (for Patients). Retrieved from ii Pulmonary Hypertension Association. Diagnosing Pulmonary Hypertensio n. Accessed on June 3, 2025 from iii Mount Sinai Health System. (n.d.). Idiopathic pulmonary fibrosis. Mount Sinai Health Library. Retrieved June 3, 2025, from iv Nathan, S. D., Stinchon, M. R., Atcheson, S., Simone, L., & Nelson, M. (2025). Shining a spotlight on pulmonary hypertension associated with interstitial lung disease care: The latest advances in diagnosis and treatment. Journal of Managed Care & Specialty Pharmacy, 31(1-a Suppl), S2–S29.
Yahoo
13-05-2025
- Business
- Yahoo
Mereo BioPharma Reports First Quarter 2025 Financial Results and Provides Corporate Highlights
Progress continues in Phase 3 Orbit study of setrusumab in osteogenesis imperfecta (OI) Cash of $62.5 million as of March 31, 2025, expected to fund operations into 2027 LONDON, May 13, 2025 (GLOBE NEWSWIRE) -- Mereo BioPharma Group plc (NASDAQ: MREO) ('Mereo' or the 'Company'), a clinical-stage biopharmaceutical company focused on rare diseases, today announced its financial results for the first quarter ended March 31, 2025, and provided recent corporate highlights. 'As we close out the first quarter of 2025, we continue to anticipate that this will be an important, milestone-rich year for Mereo. The Phase 3 Orbit study of setrusumab in osteogenesis imperfecta remains on track to read-out either at the second interim analysis in mid-2025 or at the final analysis in the fourth quarter. We are continuing to invest in the pre-commercial activities for setrusumab to enable a successful launch in our European territory, following potential regulatory approvals,' said Dr. Denise Scots-Knight, Chief Executive Officer of Mereo. "Further, alvelestat is now Phase 3 ready and we are finalizing the trial start-up activities to support our ongoing partnering process. Along with our late-stage pipeline, we believe that continued close management of our cash balance will enable us to support our operations into 2027.' First Quarter 2025 Highlights, Recent Developments, and Anticipated Milestones Setrusumab (UX143) Continued progress in the two global Phase 3 studies led by our partner Ultragenyx: The randomized, placebo-controlled Phase 3 portion of the Orbit study (in patients aged 5 to 25 years) is progressing toward the second interim analysis (IA2) in mid-2025 or a final analysis in the fourth quarter of 2025. All patients have now been on therapy for at least 12 months, conduct of the study is going well and patient safety in the Phase 3 portion of the study is consistent with safety observed in the Phase 2. Patients in the Cosmic study (aged 2 to <7 years) are being treated with either setrusumab or intravenous bisphosphonates (IV-BP) therapy and will be evaluated in parallel with the Orbit interim analysis. If Orbit progresses to full study completion in the fourth quarter of 2025, Cosmic will also continue to a data readout, to align with the Orbit readout without spending alpha at the mid-year interim assessment. Continued pre-commercial activities in Europe to support potential launch, including engagement with regulatory/HTA bodies and real-world data collection efforts through the SATURN program. Alvelestat (MPH-966) In the first quarter of 2025, the European Commission granted Orphan Designation to alvelestat for the treatment of alpha-1 antitrypsin deficiency-associated lung disease (AATD-LD). This adds to existing US FDA Orphan Drug and Fast Track designations. The start-up activities for the planned single, global Phase 3 pivotal study are ongoing. The Company remains in discussion with multiple potential development and commercialization partners. First Quarter 2025 Financial Results Total research and development ('R&D') expenses decreased by $0.1 million from $4.0 million in the first quarter of 2024 to $3.9 million in the first quarter of 2025. The decrease was primarily due to decreases of $1.2 million and $0.1 million in R&D expenses for alvelestat and etigilimab, offset by an increase of $1.3 million in R&D expenses for setrusumab. The decrease in program expenses for alvelestat was primarily due to undertaking reduced drug formulation and manufacturing activities in preparation for the Phase 3 study in the first quarter of 2025, compared to the first quarter of 2024. The increase in program expenses for setrusumab was primarily driven by amounts due under the manufacturing and supply agreement with our partner, Ultragenyx, ongoing activities related to real-world evidence programs and medical affairs activities in Europe and input into development, regulatory and manufacturing plans with Ultragenyx, who fund the global development of the program pursuant to our license and collaboration agreement. General and administrative expenses increased by $1.4 million from $5.9 million in the first quarter of 2024 to $7.3 million in the first quarter of 2025. The increase was primarily due to the recognition of a $1.7 million reduction in expenses in the first quarter of 2024 for amounts received from our depository to reimburse certain expenses incurred by us in respect of our ADR program, partially offset by a net decrease in employee-related expenses and professional fees. A reimbursement in respect of our ADR program is anticipated in 2025. Net loss for the first quarter of 2025 was $12.9 million, compared to $9.0 million during the first quarter of 2024, primarily reflecting an operating loss of $11.2 million and foreign currency translation loss. As of March 31, 2025, the Company had cash and cash equivalents of $62.5 million, compared to $69.8 million as of December 31, 2024. The Company's guidance remains unchanged, and it continues to expect, based on current operational plans, that its existing cash and cash equivalents balance will enable it to fund its currently committed clinical trials, operating expenses, and capital expenditure requirements into 2027. This guidance does not include any potential upfront payments associated with a partnership for alvelestat or business development activity around any of the Company's non-core programs. Total ordinary shares issued as of March 31, 2025 were 795,001,444. Total ADS equivalents as of March 31, 2025 were 159,000,288, with each ADS representing five ordinary shares of the Company. About Mereo BioPharmaMereo BioPharma is a biopharmaceutical company focused on the development of innovative therapeutics for rare diseases. The Company has two rare disease product candidates: setrusumab for the treatment of osteogenesis imperfecta (OI); and alvelestat for the treatment of severe alpha-1 antitrypsin deficiency-associated lung disease (AATD-LD). The Company's partner, Ultragenyx Pharmaceutical, Inc., has completed enrollment in the Phase 3 portion of a pivotal Phase 2/3 study in pediatrics and young adults (5 to 25 years old) for setrusumab in OI and in the Phase 3 study in pediatric patients (2 to <7 years old). The partnership with Ultragenyx includes potential additional milestone payments of up to $245 million and royalties to Mereo on commercial sales in Ultragenyx territories. Mereo has retained EU and UK commercial rights and will pay Ultragenyx royalties on commercial sales in those territories. Setrusumab has received orphan designation for osteogenesis imperfecta from the European Commission ('EC') and the FDA, PRIME designation from the EMA, and has Breakthrough Therapy designation and rare pediatric disease designation from the FDA. Alvelestat has received Orphan Designation for AATD from the EC and the FDA, and Fast Track designation from the FDA for AATD-LD. Following results from ASTRAEUS and ATALANTa in AATD-lung disease, the Company has aligned with the FDA and the EMA on the primary endpoints for a Phase 3 pivotal study which, if successful, could enable full approval in both the U.S. and Europe. In addition to the rare disease programs, Mereo has two oncology product candidates, etigilimab, an anti-TIGIT; and navicixizumab for the potential treatment of late-line ovarian cancer. Navicixizumab has been partnered with Feng Biosciences, Inc. in a global licensing agreement that includes milestone payments and royalties. Mereo has also entered into an exclusive global license agreement with ReproNovo SA, a reproductive medicine company, for the development and commercialization of leflutrozole, a non-steroidal aromatase inhibitor. Forward-Looking StatementsThis press release contains 'forward-looking statements' that involve substantial risks and uncertainties. All statements other than statements of historical fact contained herein are forward-looking statements within the meaning of Section 27A of the United States Securities Act of 1933, as amended, and Section 21E of the United States Securities Exchange Act of 1934, as amended. Forward-looking statements usually relate to future events and anticipated revenues, earnings, cash flows or other aspects of our operations or operating results. Forward-looking statements are often identified by the words 'believe,' 'expect,' 'anticipate,' 'plan,' 'intend,' 'foresee,' 'should,' 'would,' 'could,' 'may,' 'estimate,' 'outlook' and similar expressions, including the negative thereof. The absence of these words, however, does not mean that the statements are not forward-looking. These forward-looking statements are based on the Company's current expectations, beliefs and assumptions concerning future developments and business conditions and their potential effect on the Company. While management believes that these forward-looking statements are reasonable as and when made, there can be no assurance that future developments affecting the Company will be those that it anticipates. All of the Company's forward-looking statements involve known and unknown risks and uncertainties some of which are significant or beyond its control and assumptions that could cause actual results to differ materially from the Company's historical experience and its present expectations or projections. Such risks and uncertainties include, among others, the uncertainties inherent in the clinical development process; the Company's reliance on third parties to conduct and provide funding for its clinical trials; the Company's dependence on enrollment of patients in its clinical trials; and the Company's dependence on its key executives. You should carefully consider the foregoing factors and the other risks and uncertainties that affect the Company's business, including those described in the 'Risk Factors' section of its Annual Report on Form 10-K, as well as discussions of potential risks, uncertainties, and other important factors in the Company's subsequent filings with the Securities and Exchange Commission. The Company wishes to caution you not to place undue reliance on any forward-looking statements, which speak only as of the date hereof. The Company undertakes no obligation to publicly update or revise any of our forward-looking statements after the date they are made, whether as a result of new information, future events or otherwise, except to the extent required by law. Mereo BioPharma Contacts: Mereo +44 (0)333 023 7300 Denise Scots-Knight, Chief Executive Officer Christine Fox, Chief Financial Officer Burns McClellan (Investor Relations Adviser to Mereo) +01 646 930 4406 Lee Roth Investors investors@ BIOPHARMA GROUP PLCCONDENSED CONSOLIDATED BALANCE SHEETS(In thousands, except per share amounts)(Unaudited) March 31, December 31, 2025 2024 Assets Current assets: Cash and cash equivalents $ 62,483 $ 69,802 Prepaid expenses and other current assets 2,519 2,175 Research and development incentives receivables 1,897 2,786 Total current assets 66,899 74,763 Property and equipment, net 247 257 Operating lease right-of-use assets, net 622 727 Intangible assets, net 554 643 Total assets $ 68,322 $ 76,390 Liabilities Current liabilities: Accounts payable $ 2,924 $ 2,440 Accrued expenses 2,932 4,071 Convertible loan notes – current — 5,535 Operating lease liabilities – current 747 707 Other current liabilities 894 1,095 Total current liabilities 7,497 13,848 Warrant liabilities – non-current 419 821 Operating lease liabilities – non-current — 187 Other non-current liabilities 325 565 Total liabilities $ 8,241 $ 15,421 Shareholders' Equity Ordinary shares, par value £0.003 per share; 795,001,444 shares issued at March 31, 2025 (December 31, 2024: 775,728,034). $ 3,132 $ 3,059 Additional paid-in capital 544,266 539,642 Accumulated deficit (472,027 ) (462,883 ) Accumulated other comprehensive loss (15,290 ) (18,849 ) Total shareholders' equity 60,081 60,969 Total liabilities and shareholders' equity $ 68,322 $ 76,390 MEREO BIOPHARMA GROUP PLCCONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS(In thousands, except per share amounts)(Unaudited) Three Months Ended March 31, 2025 2024 Operating expenses: Research and development $ (3,930 ) $ (3,994 ) General and administrative (7,272 ) (5,906 ) Loss from operations (11,202 ) (9,900 ) Other income/(expenses) Interest income 659 617 Interest expense (180 ) (310 ) Changes in the fair value of warrants 416 (448 ) Foreign currency transaction (loss)/gain, net (2,765 ) 613 Benefit from research and development tax credit 185 477 Net loss before income tax (12,887 ) (8,951 ) Income tax benefit — — Net loss $ (12,887 ) $ (8,951 ) Loss per share – basic and diluted $ (0.02 ) $ (0.01 ) Weighted average shares outstanding – basic and diluted 784,279,387 700,263,490 Net loss $ (12,887 ) $ (8,951 ) Other comprehensive income/(loss) – Foreign currency translation adjustments, net of tax 3,559 (798 ) Total comprehensive loss $ (9,328 ) $ (9,749 )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
