Latest news with #osteogenesisimperfecta
Yahoo
14-05-2025
- Business
- Yahoo
Ultragenyx Underscores Continued Commitment to Rare Disease Innovation with 2024 Impact Report
NOVATO, Calif., May 14, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) released its 2024 Impact Report today, which covers the company's approach to corporate responsibility. The report highlights efforts to transform the lives of individuals and families affected by rare diseases, empower its employees, and make a meaningful impact on the communities it operates within and serves. 'Since founding Ultragenyx 15 years ago, I've sought to build a next-generation rare disease company that has a meaningful and sustained impact on the entire rare disease community,' said Emil Kakkis, M.D., Ph.D., founder, president, and chief executive officer of Ultragenyx. 'Guided by the principle of doing the right thing, we have engineered our own dynamic approach to drug development while working on broader education and policy initiatives that meaningfully support diagnosis, drug development, regulatory pathways, and clinical care. The sustainability and social impact implications of our actions and decisions are consistently considered and reflected in our 2024 Impact Report.' The company's approach to corporate responsibility is structured around six key pillars—Innovation, Patients, People, Communities, Planet, and Governance—aligned with its vision to lead the future of rare disease medicine and its mission to transform the lives of people with rare diseases. The 2024 Ultragenyx Impact Report features updates for all six pillars, as well as Sustainability Accounting Standards Board (SASB) and Global Reporting Initiative (GRI) indices. For the full report and more on Ultragenyx's Corporate Responsibility efforts, visit Highlights from the company's 2024 report include: Innovation:Ultragenyx is committed to developing and delivering transformative treatments where none exist. Successfully transferred the manufacturing process of our DTX401 gene therapy to our Gene Therapy Manufacturing Facility (GTMF) in Bedford, Massachusetts Advanced several key clinical programs to treat rare and ultra-rare diseases, including osteogenesis imperfecta (OI), Angelman syndrome, Sanfilippo syndrome type A (MPS IIIA), and glycogen storage disease type Ia (GSDIa) Supported more than 80 clinical and non-clinical investigator-sponsored trials (ISTs) globally Patients:Ultragenyx is committed to supporting the rare disease community through our efforts to develop novel therapies, share our science and expertise, achieve broad access to screening and treatment, and partner with policymakers for meaningful change. Supported more than 650 patients in 50 countries in gaining access to Ultragenyx treatments through various global expanded access and patient assistance programs since 2013 Successfully advocated for the passage of newborn screening legislation in two more U.S. states Hosted the ninth and tenth Rare Bootcamp to help patient families, foundations, and organizations seeking to develop novel treatments for rare diseases People:Ultragenyx is committed to maintaining an inclusive, safe, and healthy environment. The company is also committed to fair and equitable compensation practices that are transparent and free from bias. Launched the Executive Edge program to support the development of leadership skills at the vice president (VP) and senior VP levels Maintained a high engagement score of 88% in our employee engagement survey Communities:Ultragenyx is committed to supporting initiatives that provide meaningful impact for the rare disease community: public health and access to care, Science, Technology, Engineering, Arts, and Mathematics (STEAM) education, and local, at-risk communities. Approved more than $3 million in charitable donations, independent medical education, and health-related grants across more than 20 countries Hosted 2nd Annual Ultragenyx Global Days of Service, with employees volunteering in nearly 60 service projects Planet:Ultragenyx is committed to implementing an environmental strategy that helps minimize its environmental footprint across its business. Purchased 100% renewable electricity for our corporate headquarters campus in Novato, Calif. Achieved My Green Lab Green certification at our Translational Sciences lab in Novato, Calif. Governance:Ultragenyx is committed to strong corporate governance, ethics and integrity, compliance, data protection and security, and responsible procurement. More than 90% of responders to the annual compliance culture survey stated that the annual compliance training is adequate for them to confidently execute their responsibilities Maintained a formal Global Human Rights policy to hold ourselves and our vendors to its standards Ultragenyx Receives External Recognition in 2024Ultragenyx's achievements in 2024 were consistently recognized through several prestigious awards. In 2024, Ultragenyx was named one of the 100 Most Sustainable Companies by Barron's and one of the Top Places to Work in the USA for the second consecutive year, receiving six Cultural Excellence Awards from Top Workplaces. The company also received accolades from The Boston Globe and the San Francisco Chronicle. Additionally, the company was honored with the MassEcon Gold Award for Economic Impact in the Northeast region, celebrating its significant contributions to the Massachusetts economy, and was recognized as one of the Best Places for Working Parents in Massachusetts. About UltragenyxUltragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease. The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency. For more information on Ultragenyx, please visit the company's website at: Forward-Looking Statements and Use of Digital Media This press release and the 2024 Ultragenyx Impact Report and other materials cross-referenced in this press release contain statements that are aspirational or reflective of our views about the company's future performance that constitute 'forward looking statements' within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are generally identified through the inclusion of words such as 'aim,' 'anticipate,' 'aspire,' 'believe,' commit,' 'endeavor,' 'estimate,' 'expect,' 'goal,' 'intend,' 'may,' 'plan,' 'seek,' 'strive,' 'target,' 'will,' vision,' 'mission,' 'strategy,' 'commitment' and 'work,' or similar statements or variations of such terms and other similar expressions. The forward-looking statements in this document and the materials cross-referenced concern Ultragenyx's goals, progress or expectations with respect to corporate responsibility, sustainability, patients, products, product candidates, employees, environmental matters, policy and business risks and opportunities. Forward-looking statements inherently involve risks and uncertainties that could cause actual results to differ materially from those predicted in such statements. These statements are based on numerous assumptions that the company believes are reasonable but are open to a wide range of uncertainties and business risks. In addition, these statements may be based on standards for measuring progress that are still developing, controls and processes that continue to evolve, and assumptions that are subject to change in the future, and certifications, representations or data reviewed or provided by third parties Consequently, actual results may vary materially from what is contained in a forward-looking statement. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on May 7, 2025, and its subsequent periodic reports filed with the SEC. Forward-looking statements are aspirational and are not guarantees or promises that goals or targets will be met. Ultragenyx undertakes no obligation to update any forward-looking or other statements, whether as a result of new information, future events, or otherwise, and notwithstanding any historical practice of doing so. Ultragenyx may determine to adjust any goals and targets or establish new ones to reflect changes in its business. The information included in, and any issues identified as material for purposes of, the 2024 Ultragenyx Impact Report is not an indication that they are considered material to Ultragenyx, its investors or other stakeholders, or required to be disclosed in the company's filings, in each case under SEC reporting or any other laws or requirements that may apply to the company. In the context of this report, the term 'material' is distinct from, and should not be confused with, such term as defined for SEC or other mandatory reporting purposes. In addition to its SEC filings, press releases and public conference calls, Ultragenyx uses its investor relations website and social media outlets to publish important information about the company, including information that may be deemed material to investors, and to comply with its disclosure obligations under Regulation FD. Financial and other information about Ultragenyx is routinely posted and is accessible on Ultragenyx's Investor Relations website ( and LinkedIn website ( Ultragenyx Contacts Investors Joshua Higa(415) 475-6370ir@ MediaJoey Fleurymedia@
Associated Press
13-05-2025
- Business
- Associated Press
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) Alvelestat (MPH-966) 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 BioPharma Mereo 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 Statements This 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:
