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Strong idea · still checking
Ultragenyx Pharmaceutical Inc.
Expected time 1 year is when we expect the main question to be answered. Full holding time 9-15 months is the longest the idea may stay open if the facts still support it.
Reviewed Sep 2, 2026
Long RARE on a 1-year fundamentals rerating.
Long RARE on a 1-year fundamentals rerating. The market is still pricing Ultragenyx as a high-burn rare-disease pipeline, but the company has a real commercial base, reaffirmed 2026 revenue guidance of $730 million to $760 million, and a stated 2027 profitability path. The gap can close if the September 2026 UX111 PDUFA and September/October GTX-102 Aspire readout keep the pipeline converting while GENGLYCOS begins to show access and launch execution. The thesis is not that every catalyst must work; it is that the existing revenue base plus one successful late-stage conversion supports a materially higher value than the current mid-$20s share price.
Expected holding period: 9-15 months
These are TickerYou's own possible outcomes—not Wall Street targets or copied internet forecasts. Lower is what may happen if important facts disappoint, middle is our main working case, and higher is what may happen if things go better than expected. These are checks for the research, not promises.
We will check this set through 6-12 months. The expected holding period (9-15 months) is the broader time we may follow the Idea, so the two dates do not need to match. We show longer-term estimates only when the supporting business and price assumptions are written down.
Where these numbers came from. Published Aug 24, 2026 against $25.78 using a saved Yahoo Finance price as of Aug 24, 2026.
| Time frame | Check by | Lower case | Middle case | Higher case | Source strength | What happened |
|---|
Loading company facts
Loading reported-quarter evidence
Checked through Sep 2, 2026
Each source says whether it supports the idea, challenges it, or adds background. A quick no-change check may update the review date without replacing these sources.
Ultragenyx reported Q2 2026 total revenue of $214 million, reaffirmed 2026 revenue guidance of $730 million to $760 million, reiterated a 2027 profitability path, and said UX111 PDUFA and GTX-102 Aspire data remained second-half 2026 catalysts.
Ultragenyx Reports Second Quarter 2026 Financial Results and Corporate Update (ir.ultragenyx.com)FDA granted accelerated approval for Genglycos on 2026-08-19 based on reduced daily cornstarch intake, while requiring confirmatory benefit and listing serious safety warnings including anaphylaxis, liver toxicity, adrenal insufficiency, and tumorigenicity risk.
FDA Approves First Therapy for Patients aged 8 years and older with Glycogen Storage Disease Type Ia (fda.gov)We rated 3 of 3 upcoming events. 0 are both likely and important.
51 of 100 = chance 0.85 × effect 0.6 × date nearness 1.00.
The date is not clear enough to calculate nearness.
33 of 100 = chance 0.55 × effect 0.6 × date nearness .
Checks are shown newest first. Repeated copies are folded together on screen, while every saved record remains unchanged.
Previously we believed RARE's revenue base, 2026 guidance and 2027 profitability path could support a rerating while UX111 and GTX-102 remained live. Searches found no cutoff-eligible dated evidence after 2026-08-30 usable for mutation; reopened 2026-08-04 and 2026-08-19 primary sources confirm the baseline but leave the major FDA and Aspire readouts unresolved.
We previously believed RARE could rerate if revenue guidance, the 2027 profitability path, UX111 timing and GTX-102 timing held. Searches found no material dated development after 2026-08-28; reopened primary releases still show 2026 revenue guidance of $730-$760 million, UX111 PDUFA on 2026-09-19, GTX-102 Aspire data expected in September or October 2026, and GENGLYCOS approved but dependent on launch execution and post-marketing evidence. The invalidation trigger has not fired; UX111 has not received a sourced CRL and GTX-102 has not produced a negative or inconclusive Aspire readout in the searched sources. GENGLYCOS access, GTX-102 data quality and the UX111 FDA action remain unresolved.
1 check left
Reopened primary sources dated 2026-08-04 and 2026-08-19 confirm revenue guidance, pipeline timing, and GENGLYCOS accelerated approval risk context.
UX111 FDA action, GTX-102 efficacy and safety, GENGLYCOS access, and cash burn remain unresolved.
No ready estimate is issued while checks are missing. The $40.00 research scenario remains below for context, without possible-move or sizing language.
UX111 could receive another Complete Response Letter or a narrow label if FDA is not satisfied with CMC, inspections, biomarker support, or accelerated-approval evidence.
Sell if FDA issues a Complete Response Letter for UX111 and GTX-102 Phase 3 Aspire is negative or clinically inconclusive before December 31, 2026.
| 1-3 monthsShort-term scenario | 2026-11-24 | $18.00 | $34.00 | $50.00 | medium evidence confidence | Open window |
| 3-6 monthsMedium-term scenario | 2027-02-24 | $17.00 | $40.00 | $60.00 | medium evidence confidence | Open window |
| 6-12 monthsLong-term scenario | 2027-08-24 | $16.00 | $45.00 | $70.00 | medium evidence confidence | Open window |
These are TickerYou's possible outcomes from the latest saved research. Source strength tells you how solid the supporting information is. It is not a chance of success or a recommendation score. These ranges stay visible as research, but we hide the comparison with today’s price until every required check is complete. We check every price range against what happened when its time frame ends—including the ones we got wrong.
Kept as the original record. These do not replace the current sources above.
Three checks come from TickerYou's research review. The app calculates the other three from dated prices, events, and sources. Each check keeps its published weight on every idea; the app never changes a weight to make an idea look better.
How strongly the research engine believes the thesis is right, judged 1-10. This pass judged it 8 out of 10.
Whether the price being paid helps or hurts, judged 1-10: 10 means you are paid to take the risk, 1 means the thesis must be right and the multiple must expand. This pass judged it 7 out of 10.
How contained the damage is if the thesis is wrong, judged 1-10: 10 means a real floor of assets or cash, 1 means a permanent loss. This pass judged it 5 out of 10.
The clearest catalyst still ahead opens in 7 days, on a single dated day (1 other date could not be read).
Price is -40.4% over the last 63 trading sessions — moving against it for this long view.
2 dated claim receipts average 10 out of 10 from explicit source tiers 1, 1; roles: 1 support, 1 challenge, 0 context. Roles establish claim linkage; only the stored tiers set reliability.
This confidence score of 71 out of 100 is the weighted average of the 6 ready checks above, on their 0-10 scales, adjusted to 100.
Previously we believed RARE could rerate if its commercial revenue base and 2027 profitability path held while UX111 and GTX-102 catalysts stayed on track. Live searches found no material cutoff-eligible post-review UX111 CRL, GTX-102 negative readout, guidance cut or GENGLYCOS launch break. Reopened primary sources confirm Q2 revenue guidance, the September 19, 2026 UX111 PDUFA, September/October GTX-102 timing, and the GENGLYCOS accelerated-approval risk baseline. The thesis remains intact but catalyst outcomes are unresolved.
Previously we believed RARE should rerate if 2026 revenue guidance, the 2027 profitability path, UX111 and GTX-102 stayed intact. Current searches found no cutoff-eligible material development after 2026-08-26T17:39:11.920Z; the reopened August 4 company update remains the newest usable primary baseline. This confirmed, but did not strengthen, the view; FDA and clinical readouts remain unresolved.
Previously we owned RARE for commercial-base durability plus clustered late-2026 pipeline catalysts. The only cutoff-eligible post-review company item found was the August 25 inducement grant to 23 non-executive employees, not a guidance, UX111 or GTX-102 development. Reopened August 4 and August 19 primary sources still show 2026 revenue guidance of $730-$760 million, the September 19 UX111 PDUFA and September/October GTX-102 Aspire timing, while GENGLYCOS remains an accelerated approval with confirmatory and safety obligations. Thesis intact; binary catalyst risk remains.
We previously believed RARE could rerate over one year if its revenue base, 2026 guidance, 2027 profitability path, UX111 PDUFA and GTX-102 Aspire catalyst cluster remained intact. A live search found no eligible post-review dated UX111 CRL, negative Aspire readout, or guidance change before the cutoff; the reopened August 4, 2026 company release still shows $214 million Q2 revenue, reaffirmed $730-$760 million 2026 revenue guidance, a 2027 profitability path, UX111 PDUFA on September 19, 2026 and GTX-102 Aspire data expected in September or October. The view is confirmed but not improved; the unresolved facts are the FDA action and Aspire efficacy/safety data.
Opened by the biotech hunt.
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TickerYou has not saved a quarterly report for this company yet. We show nothing instead of guessing.
Computed from SEC filings — never model output. Each quarter is named by the fiscal quarter the company itself reported and the month that quarter ended, through the same resolver the Earnings card beside this one uses; where no release established a fiscal label, the quarter is named by its end alone rather than by a quarter number guessed from the date. EPS is GAAP diluted, as filed, so it can legitimately differ from the adjusted figure in the Earnings card (consensus is quoted adjusted). Y/Y compares the same fiscal quarter a year earlier; a comparison that cannot be made honestly is an em-dash that says why.
Jun 2026: the filing establishes the quarter, but comparable year-ago growth and margin are not established.
Comparing the same fiscal quarter a year earlier controls for seasonality. Revenue direction and margin direction together show whether sales are translating into profit, but one quarter is evidence—not a complete thesis.
Watch for the next filing that supplies a same-quarter comparison rather than substituting an older period.
| (USD) | Jun 2026 | vs last year |
|---|---|---|
| Revenue | — | — |
| Profit after costs | $-92.00 M | — |
| Profit per share | -0.90 | — |
| Profit margin | — | — |
† derived: the fiscal year minus its three reported quarters — no 10-K reports a Q4 figure directly. For EPS this assumes per-share figures add across the year.
| Quarter | Revenue | Y/Y | Net income | Margin |
|---|---|---|---|---|
| Mar 2026 | — | — | $-185.00 M | — |
| Dec 2025 | — | — | $-128.59 M † | — |
| Sep 2025 |
| — |
| — |
| $-180.41 M |
| — |
| Jun 2025 | — | — | $-115.00 M | — |
| Mar 2025 | — | — | $-151.00 M | — |
| Dec 2024 | — | — | $-133.20 M † | — |
| Sep 2024 | — | — | $-133.52 M | — |
| Jun 2024 | — | — | $-131.60 M | — |