Latest news with #RARE
Yahoo
04-08-2025
- Business
- Yahoo
Ultragenyx Pharmaceutical Inc. (RARE): A Bull Case Theory
We came across a bullish thesis on Ultragenyx Pharmaceutical Inc. on Valueinvesting subreddit by bluespacecolombo. In this article, we will summarize the bulls' thesis on RARE. Ultragenyx Pharmaceutical Inc.'s share was trading at $27.61 as of July 28th. A scientist in a laboratory coat looking into a microscope at human bone regeneration samples. Ultragenyx Pharmaceutical (RARE) has seen its share price halve in just two weeks, largely due to a halted trial and an FDA rejection, but the market appears to have overreacted. The company's Phase 3 trial for setrusumab, a potential blockbuster therapy for a rare bone disease, failed to meet an early 'stop-for-efficacy' milestone, yet the study remains on track, with safety intact and final results expected in late 2025 under less stringent endpoints. Meanwhile, the FDA issued a complete response letter for another gene therapy candidate, citing manufacturing issues rather than safety or efficacy concerns. The program remains viable, with a resubmission expected once production adjustments are made. Despite these setbacks, Ultragenyx continues to generate meaningful revenue, posting $139 million in Q1 and guiding for over $640 million in 2025, and maintains a robust cash position of around $560 million, with management projecting lower cash burn. Beyond setrusumab, the company's pipeline is well diversified, featuring DTX401, a gene therapy for glycogen storage disease that met Phase 3 goals and is slated for FDA submission this year, as well as an advanced Angelman syndrome program in Phase 3. None of the recent regulatory or clinical updates involved failures of safety or efficacy, leaving the scientific foundation of the pipeline intact. Analysts' average price targets remain near $100, implying a potential threefold upside from the current ~$26 level. With solid fundamentals, multiple near-term catalysts, and a temporarily depressed valuation, Ultragenyx represents a compelling contrarian opportunity as panic-driven selling unwinds. Previously we covered a bullish thesis on Capricor Therapeutics, Inc. (CAPR) by androidmj in March 2025, which highlighted the upcoming FDA decision on Deramiocel, strong financial backing, and significant upside on approval. The company's stock has depreciated about 46.32% since then as the thesis has yet to play out. The thesis still stands given CAPR's cash runway and de-risked catalyst. bluespacecolombo shares a similar view on the biotech space but emphasizes Ultragenyx's diversified pipeline, overreaction-driven selloff, and near-term catalysts. Ultragenyx Pharmaceutical Inc. is not on our list of the 30 Most Popular Stocks Among Hedge Funds. As per our database, 55 hedge fund portfolios held RARE at the end of the first quarter which was 57 in the previous quarter. While we acknowledge the potential of RARE as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock. READ NEXT: 8 Best Wide Moat Stocks to Buy Now and 30 Most Important AI Stocks According to BlackRock. Disclosure: None. Error while retrieving data Sign in to access your portfolio Error while retrieving data Error while retrieving data Error while retrieving data Error while retrieving data
Yahoo
30-07-2025
- Business
- Yahoo
Ultragenyx to Host Conference Call for Second Quarter 2025 Financial Results and Corporate Update
NOVATO, Calif., July 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, August 5, 2025, to discuss its financial results and corporate update for the quarter ending June 30, 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 Higair@ MediaJess Rowlands media@ 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
Hans India
26-07-2025
- Politics
- Hans India
TG caste survey ‘visionary': Rahul
Hyderabad: Close on the heels of Congress leader Sonia Gandhi's letter to Chief Minister Revanth Reddy hailing the Telangana government's Socio-Economic Caste Survey, Leader of Opposition Rahul Gandhi on Friday praised the Telangana Model survey as 'visionary, inclusive, and powered by 21st-century data. It is Social Justice 2.0 and it will define how India moves forward.' In his tweet on Friday, Rahul posted: 'Soniaji's fight to create the state of Telangana cemented our deep emotional connection to the people, the language and the culture of this beautiful state. I am filled with immense pride as I witness the remarkable efforts of the Congress government in conducting the Socio-Economic Caste Survey. Their commitment to a sincere, inclusive, and consultative process has set a new benchmark - one that should guide future Census exercises across the country. Based on the survey, the Telangana Government has taken a historic step in recommending 42 oerp OBC reservation in local body elections and educational institutions. The bill now awaits assent from the Hon'ble President. The Telangana Model is visionary, inclusive, and powered by 21st-century data. It is Social Justice 2.0 and it will define how India moves forward.' On Thursday, when Chief Minister Revanth Reddy made a presentation of the caste survey in Delhi, Rahul Gandhi said: 'When I first asked Revanth Reddy to conduct a caste survey in Telangana, I had my doubts. But they have exceeded my expectations. This is a milestone for social justice in the country,' Rahul said, adding that no other state in the country except Telangana got the opportunity to achieve targeted development based on the survey data. An enthused Chief Minister, calling the survey a 'RARE 'model, recalled that he was emotional and overjoyed when he received an appreciation letter from Sonia Gandhi. The Chief Minister remarked: 'The letter is worth a Nobel prize, Oscar and a lifetime achievement award', adding it would remain special for him, irrespective of whether he held a position or not in the future.
NDTV
25-07-2025
- Politics
- NDTV
Revanth Reddy Suggests Telangana Caste Census Be Called 'RARE' Model
Telangana Chief Minister A Revanth Reddy has dubbed a letter from All India Congress Committee (AICC) leader Sonia Gandhi, where she apologised for being unable to attend his presentation on Telangana's Backward Classes (BC) caste census to Congress Members of Parliament in Delhi, as a "letter of appreciation" equivalent to a "lifetime achievement award, a Nobel Prize, and an Oscar." He said Sonia Gandhi's acknowledgment of the state's BC caste census efforts was the "highest honour of his political career". Mr Reddy credited Rahul Gandhi for the initiative behind the caste census and urged the Congress party to advocate for 42 per cent BC reservations at the national level. During his presentation, Revanth Reddy also proposed a unique branding for Telangana's social justice initiatives, including the caste census, as the "RARE model". This acronym presumably stands for "Rahul-Revanth Model". Revanth Reddy presented the Telangana caste survey as a comprehensive socio-economic, educational, and political survey, highlighting that it involved the collection of 88 crore pages of self-declared data, deeming it "100 per cent foolproof". The Chief Minister asserted that the RARE model is not merely a Telangana initiative but a potential template for the nation's future in social justice. He also indicated plans for further enhancing reservations for Scheduled Castes (SCs) and Scheduled Tribes (STs) based on the findings of the state's socio-economic caste census. Mr Reddy said February 4, the date the survey began last year, would henceforth be observed as "Social Justice Day" in Telangana. Revanth Reddy's address also included a strong criticism of the BJP, accusing them of opposing caste census initiatives and claiming that Prime Minister Narendra Modi was "legally converted OBC" whose caste was included in the category only after he became Gujarat Chief Minister. He said he would stage a 'dharna' at Jantar Mantar if the Centre did not approve the 42 per cent BC quota bills.
Yahoo
13-07-2025
- Business
- Yahoo
Ultragenyx (RARE) Falls 26% as New Treatment Fails to Get FDA Green Light
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is one of the Ultragenyx fell by 26.05 percent week-on-week as investor sentiment was largely dragged down by the Food and Drug Administration's (FDA) rejection of its biologics license application (BLA) for its UX111 (ABO-102) treatment. In a regulatory filing, Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) said that the FDA requested additional information and improvements in relation to certain chemistry, manufacturing, and controls (CMC) aspects. It noted that the observations were not related to product quality concerns product, adding that many of the issues have already been addressed. UX111 was developed to treat a common type of Sanfilippo syndrome, a group of genetic conditions that begin in early childhood and causes severe brain damage and early death. A research team in a laboratory peering through microscopes at a biologic product. The treatment involves managing symptoms, as there are currently no approved disease-modifying medicines. Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) said it plans to resubmit updated clinical data from current patients after resolving the FDA's concerns. A new review could take another six months upon resubmission of a revised BLA application. While we acknowledge the potential of RARE as an investment, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and have limited downside risk. If you are looking for an extremely cheap AI stock that is also a major beneficiary of Trump tariffs and onshoring, see our free report on the . READ NEXT: 30 Stocks That Should Double in 3 Years and 11 Hidden AI Stocks to Buy Right Now. Disclosure: None. This article is originally published at Insider Monkey.
