NOVATO, Calif. - Ultragenyx Pharmaceutical Inc. (NASDAQ: NASDAQ:RARE) has reached an agreement with the U.S. Food and Drug Administration (FDA) regarding the use of cerebral spinal fluid (CSF) heparan sulfate (HS) as a surrogate endpoint for accelerated approval of its gene therapy, UX111 (ABO-102), for Sanfilippo syndrome (MPS IIIA). This agreement is based on existing clinical study data, which the company believes is sufficient for a biologics license application (BLA) submission.
The pivotal decision was announced today, following discussions between Ultragenyx and the FDA. The company plans to finalize the BLA details in a forthcoming pre-BLA meeting, with the aim of submitting the application later this year or early next year.
Emil D. Kakkis, M.D., Ph.D., CEO and president of Ultragenyx, expressed gratitude towards the FDA for recognizing the urgency in providing treatments for children with neurologically devastating diseases. He also thanked the patient and caregiver advocates, scientists, and industry leaders for their collaboration in providing the necessary evidence to support this decision.
UX111, currently in Phase 1/2/3 development, is an in vivo gene therapy designed for a one-time intravenous infusion to deliver a functional copy of the SGSH gene to cells. The therapy aims to address the underlying enzyme deficiency responsible for the abnormal accumulation of heparan sulfate in the brain, which leads to progressive cell damage and neurodegeneration. The UX111 program has received several designations in the U.S. to expedite its development, including Regenerative Medicine Advanced Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug designations. In the EU, it has received PRIME and Orphan medicinal product designations.
Sanfilippo syndrome type A is a rare, fatal lysosomal storage disease with no approved treatment, primarily affecting the central nervous system and characterized by rapid neurodegeneration beginning in early childhood. It is estimated to affect approximately 3,000 to 5,000 patients in commercially accessible geographies and is caused by pathogenic variants in the SGSH gene.
Ultragenyx is a biopharmaceutical company focused on developing products for the treatment of rare and ultra-rare genetic diseases with high unmet medical need. This news is based on a press release statement from Ultragenyx Pharmaceutical Inc.
In other recent news, Ultragenyx Pharmaceutical Inc. has been the subject of several analyst firm updates following positive outcomes from its Phase 3 GlucoGene study. Goldman Sachs (NYSE:GS) upgraded Ultragenyx shares from Neutral to Buy, citing increased conviction in the company's monoclonal antibody setrusumab and the diverse rare disease pipeline. The firm also projected a potential 2026 approval and launch for gene therapy DTX401 in GSD1a, forecasting that Ultragenyx will reach GAAP profitability in 2027.
Baird, Stifel, Piper Sandler, and Canaccord Genuity also revised their outlook on Ultragenyx. Baird raised the price target for Ultragenyx shares to $72, citing the Phase 3 results as a strong indicator of a likely approval for DTX401. Stifel increased the price target to $127, noting the significant reduction in the need for cornstarch, a dietary supplement used to manage GSDIa. Piper Sandler maintained an Overweight rating and a $135.00 price target on Ultragenyx, pointing to sustained glucose control and significant reductions in daily cornstarch intake. Lastly, Canaccord Genuity raised the price target to $111, signaling confidence in Ultragenyx's prospects.
These recent developments underline the growing confidence in the potential success of Ultragenyx's DTX401 therapy and setrusumab. The positive clinical trial results and analyst upgrades reflect the company's progress in rare disease treatment.
InvestingPro Insights
As Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) forges ahead with its commitment to addressing rare genetic diseases, the company's financial health and market performance provide additional context for investors. Notably, Ultragenyx has a substantial market capitalization of $3.73 billion, which underscores its significant presence in the biopharmaceutical sector. However, the company's financial metrics indicate some challenges. With a negative P/E ratio of -5.62 and an adjusted P/E ratio for the last twelve months as of Q1 2024 at -5.9, Ultragenyx is not currently profitable. This aligns with an InvestingPro Tip indicating that analysts do not expect the company to be profitable this year.
The company's gross profit margin presents another area of concern, standing at -60.26% for the last twelve months as of Q1 2024. This figure suggests that Ultragenyx is currently operating at a loss, which is supported by an InvestingPro Tip highlighting weak gross profit margins. Despite these financial headwinds, Ultragenyx has a robust liquidity position, with liquid assets exceeding short-term obligations, which may provide some financial flexibility as it continues to invest in its product pipeline.
For those closely watching the company's stock performance, Ultragenyx's shares are trading at a high Price/Book multiple of 25.97, which could suggest a premium valuation compared to its book value. Investors interested in a deeper dive into Ultragenyx's financials and prospects can explore additional InvestingPro Tips at https://www.investing.com/pro/RARE. There are 5 more tips available that can offer further insights into the company's performance and potential. Moreover, interested investors can take advantage of a special offer using the coupon code PRONEWS24 to get an additional 10% off a yearly or biyearly Pro and Pro+ subscription, providing access to a wealth of data and analysis to inform investment decisions.
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