Company guidance, FDA catalysts, dilution risk and cash runway, built from SEC, FDA and ClinicalTrials.gov filings and updated every day.
$496MCash, Jun 2026
10moCash Runway (est.)
+2%Share Count, 1 Year
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📣 What RARE Says Is Next
first half of 2027 · “Data from the second primary endpoint, which evaluates reduction in treatment burden, including use of ammonia scavengers and dietary management, are expected in the first half of 2027.”
Company press release, Aug 4, 2026 · SEC filing
second half of 2026 · “UX016 novel prodrug for sialic acid used as a substrate replacement therapy for the treatment of GNE myopathy: The FDA cleared the Investigational New Drug (IND) application for UX016 and an externally funded Phase 1/2 study is expected to begin in the second half of 2026. 2”
Company press release, Aug 4, 2026 · SEC filing
fourth quarter of 2026 · “Data from this stage are expected in the fourth quarter of 2026.”
Company press release, Aug 4, 2026 · SEC filing
Quoted word for word from the company's press releases filed with the SEC. Timing is the company's own guidance, not a BioRadar estimate.
💰 Cash Runway & Dilution Risk
RARE reported $496M in cash and short-term investments (as of Jun 30, 2026) and used about $148.2M/quarter in operations, an estimated runway of 10 months at that rate (BioRadar estimate, not company guidance).
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Data: SEC EDGAR, FDA, ClinicalTrials.gov, Yahoo Finance • Informational purposes only, not investment advice. • All tracked stocks A–Z
This data is for informational purposes only, not investment advice. BioRadar does not provide buy/sell recommendations. Past performance does not guarantee future results. Always do your own due diligence.