Latest news with #UltragenyxPharmaceuticalInc
Yahoo
7 days ago
- Business
- Yahoo
Ultragenyx to Participate at Goldman Sachs 46th Annual Global Healthcare Conference
NOVATO, Calif., June 03, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today announced that Emil Kakkis, M.D., Ph.D., the company's chief executive officer, will participate in a fireside at the Goldman Sachs 46th Annual Global Healthcare Conference on Tuesday, June 10, 2025, at 10:00 a.m. ET. The live and archived webcast of the panel will be accessible from the company's website at About Ultragenyx Pharmaceutical Inc. Ultragenyx 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: Contacts – Ultragenyx Pharmaceutical, Higa ir@
Yahoo
7 days ago
- Business
- Yahoo
Ultragenyx to Participate at Goldman Sachs 46th Annual Global Healthcare Conference
NOVATO, Calif., June 03, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today announced that Emil Kakkis, M.D., Ph.D., the company's chief executive officer, will participate in a fireside at the Goldman Sachs 46th Annual Global Healthcare Conference on Tuesday, June 10, 2025, at 10:00 a.m. ET. The live and archived webcast of the panel will be accessible from the company's website at About Ultragenyx Pharmaceutical Inc. Ultragenyx 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: Contacts – Ultragenyx Pharmaceutical, Higa ir@
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@
Yahoo
30-04-2025
- Business
- Yahoo
Ultragenyx to Host Conference Call for First Quarter 2025 Financial Results and Corporate Update
NOVATO, Calif., April 30, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that it will host a conference call at 5:00 p.m. ET on Tuesday, May 6, 2025, to discuss its financial results and corporate update for the quarter ending March 31, 2025. The live and replayed webcast of the call will be available through the company's website at The replay of the call will be available for three months. About Ultragenyx Pharmaceutical 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: Contact Ultragenyx InvestorsJoshua Higa(415) 475-6370ir@ MediaJoey Fleury (415) 946-1090media@ in to access your portfolio
Yahoo
15-04-2025
- Business
- Yahoo
Ultragenyx Pharmaceutical (RARE): Among Billionaire Stanley Druckenmiller's Top Stock Picks with Huge Upside Potential
We recently published a list of . In this article, we are going to take a look at where Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) stands against other Billionaire Stanley Druckenmiller's top stock picks with huge upside potential. Wall Street is overflowing with data. If companies are not releasing their earnings, economic data flows almost daily. This abundance of data can be overwhelming, making it easy to miss important signals. That's why investors monitor billionaires who've made and continue to make their fortunes on Wall Street. The list of these billionaires is long, but a few, such as Warren Buffett and Stanley Druckenmiller, stand out. Druckenmiller is a former Soros protégé who, after 12 years at Soros Fund Management, opted to exit and focus fully on his own hedge fund, Duquesne Capital, in 2000. He later transitioned this outfit into a family office – Duquesne Family Office – in 2010. Over the past eight quarters (up to Q4 2024), the family office has consistently outperformed the S&P 500. The top 20 holdings weighted have returned 81.63% in three years (cumulatively) and 22.01% (annualized). READ ALSO: Top 10 Growth Stocks in David Tepper's Portfolio and Billionaire Ken Fisher's Top 13 Growth Stock Picks. This billionaire is widely respected across Wall Street for many things, but most of all, his market acumen, integrity, and agility. One should recall that he was part of the Soros Fund Management crew that shorted the British pound in the early 1990s to the point of almost breaking the Bank of England. His genius in the market has been noticed by many. Ken Langone, another billionaire, described Druckenmiller as the best investor he's ever known. Druckenmiller is also influential. The Financial Times reports that the veteran investor could significantly influence the direction of the current US government economic policies. Scott Bessent, the current Treasury secretary, and Kevin Warsh, a possible successor to Jerome Powell at the Fed, are Druckenmiller's proteges. According to the FT, these three 'have a great relationship', which implies that Druckenmiller has a direct line to the country's most crucial economic thinkers. Druckenmiller's investing strategy is to chase value over hype. In other words, the 71-year-old billionaire focuses on investing in undervalued stocks rather than jumping on trendy ones. Simply put, he prefers solid opportunities over flashy ones. Just recently, he exited two of the most high-profile artificial intelligence (AI) stocks. This is an eye-brow-raising move, but if the billionaire's genius is anything to go by, there must be value he is chasing. To compile this list, we reviewed the Duquesne Family Office's SEC Q4 2024 13F filings. We picked 10 stocks that have the highest upside potential from their current levels based on average analyst price targets (as of April 11). Finally, we organized the stocks in ascending order based on their highest analyst upside potential while also outlining hedge fund sentiment regarding these stocks as of Q4 2024. Why are we interested in the stocks that hedge funds pile into? The reason is simple: our research has shown that we can outperform the market by imitating the top stock picks of the best hedge funds. Our quarterly newsletter's strategy selects 14 small-cap and large-cap stocks every quarter and has returned 373.4% since May 2014, beating its benchmark by 218 percentage points (). Stanley Druckenmiller of Duquesne CapitalUltragenyx Pharmaceutical Inc. (NASDAQ:RARE) is another biopharmaceutical company in Druckenmiller's portfolio. It develops treatments for rare and ultra-rare genetic diseases, which often have no approved therapies. The leading products include approved treatments like Burosumab (for a bone disorder called XLH), Triheptanoin (for certain metabolic disorders), and Vestronidase alfa (for a rare enzyme deficiency). In Q4 2024, Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) reported strong financial results, with total revenue of $560 million, exceeding previous guidance. For 2025, the company projects continued growth with total revenue between $640 million to $670 million. In light of this performance, CEO Emil D. Kakkis believes that they 'have created a next-generation rare disease company on a pathway to profitability with meaningful revenue growth from multiple global products.' The company is approaching several major catalysts in 2025. One of the major ones is a pending FDA decision for Ultragenyx Pharmaceutical Inc.'s (NASDAQ:RARE) gene therapy to treat Sanfilippo syndrome, which is expected in the second half of the year. The firm is also planning to submit a biologics license application for its Glycogen Storage Disease Type Ia (GSDIa) gene therapy in mid-2025. Additionally, the Phase 3 ORBIT study for setrusumab in osteogenesis imperfecta is progressing toward a second interim analysis in mid-2025. On March 27, JPMorgan raised Ultragenyx's (NASDAQ:RARE) price target from $104 to $117 and rated the shares as Overweight. It cites a 'very favorable' reward/risk profile for the company's clinical programs. Overall, RARE ranks 3rd on our list of Billionaire Stanley Druckenmiller's top stock picks with huge upside potential. While we acknowledge the potential of RARE as an investment, our conviction lies in the belief that AI stocks hold greater promise for delivering higher returns and doing so within a shorter time frame. There is an AI stock that went up since the beginning of 2025, while popular AI stocks lost around 25%. If you are looking for an AI stock that is more promising than RARE but that trades at less than 5 times its earnings check out our report about the READ NEXT: 20 Best AI Stocks To Buy Now and 30 Best Stocks to Buy Now According to Billionaires. Disclosure: None. This article is originally published at Insider Monkey. Sign in to access your portfolio
