# Ultragenyx Pharmaceutical Inc. (RARE)

> Source: Nexqual · https://nexqual.com/RARE · data as of 2026-10-29 14:14 UTC · cite as "Nexqual, Ultragenyx Pharmaceutical Inc. (RARE), nexqual.com/RARE" · informational, not investment advice.

**Price:** USD 17.41 (+1.22% today) · US market open, as of October 29, 2026 at 14:14 UTC; quotes may be delayed. Prev close 17.2 · day 17.2–17.63 · 52w 12.73–39.89 · market cap $1.72B · NMS

## Key facts
- Last four reported quarters (through Jun 30, 2026): revenue $717M, net income -$586M (-81.7% margin), free cash flow -$489M (SEC filings).
- Analysts: Buy (mean 2.43 on a 1–5 scale) (21 analysts), mean target USD 26.25 (range 16–39).
- Total return with dividends: 1Y -49.7% vs S&P 500 +13.1%; 5Y -79.3% vs S&P 500 +79.1%.
- Next earnings: 2026-11-03, EPS estimate USD -0.75, revenue estimate $184M.

## Financials (SEC 10-K; TTM = last four 10-Q/10-K quarters)
| USD | FY2021 | FY2022 | FY2023 | FY2024 | FY2025 | TTM |
|---|---|---|---|---|---|---|
| Revenue | $351M | $363M | $434M | $560M | $673M | $717M |
| Operating income | -$382M | -$649M | -$569M | -$536M | -$535M | -$528M |
| Net income | -$454M | -$707M | -$607M | -$569M | -$575M | -$586M |
| EPS (diluted) | -$6.70 | -$10.12 | -$8.25 | -$6.29 | -$5.83 | — |
| Free cash flow | -$412M | -$497M | -$519M | -$421M | -$472M | -$489M |
| Operating margin | -108.6% | -178.6% | -131.1% | -95.7% | -79.5% | -73.6% |
| Net margin | -129.2% | -194.7% | -139.9% | -101.6% | -85.4% | -81.7% |
| Revenue growth | +29.7% | +3.4% | +19.5% | +29.0% | +20.2% | — |
| Cash + short-term investments | $740M | $748M | $578M | $610M | $680M | — |
Fiscal years end Dec. EPS is split-adjusted.

## Valuation (price ÷ last four quarters, through Jun 30, 2026)
P/S 2.4x · FCF yield -28.49% · Earnings yield -34.14%

## Analysts
Consensus Buy (mean 2.43 on a 1–5 scale), 21 analysts; mean target USD 26.25 (low 16, high 39).
Rating changes, last 120 days:
| Date | Firm | Action | Rating | Price target |
|---|---|---|---|---|
| 2026-10-15 | UBS | Initiated | Neutral | $16 |
| 2026-09-21 | Evercore ISI Group | Target raised | In-Line | $16 → $18 |
| 2026-09-21 | Barclays | Target raised | Overweight | $32 → $35 |
| 2026-09-18 | Morgan Stanley | Target raised | Equal-Weight | $18 → $20 |
| 2026-09-18 | Citigroup | Target raised | Buy | $31 → $32 |
| 2026-09-18 | Canaccord Genuity | Target raised | Buy | $37 → $39 |
| 2026-09-18 | Cantor Fitzgerald | Target raised | Overweight | $33 → $39 |
| 2026-09-17 | Citigroup | Target raised | Buy | $28 → $31 |
| 2026-09-04 | Goldman Sachs | Target lowered | Neutral | $28 → $19 |
| 2026-09-04 | Barclays | Target lowered | Overweight | $43 → $32 |
| 2026-09-03 | JP Morgan | Downgrade | Overweight → Neutral | $80 → $36 |
| 2026-09-03 | Jefferies | Target lowered | Buy | $77 → $28 |

## Total return (dividends reinvested)
|  | 1Y (from 2025-10-29) | 5Y (from 2021-10-25) |
|---|---|---|
| Total return | -49.7% | -79.3% |
| S&P 500 (SPY) | +13.1% | +79.1% |
| Per year | -49.7% | -26.9% |
| Volatility (annualized) | 96.4% | 56.2% |
| Max drawdown | -64.8% (Dec 2025→Sep 2026) | -83.3% (Apr 2022→Sep 2026) |

## Peers (Biotechnology)
RYTM (Rhythm Pharmaceuticals, Inc.) USD 93.38 +1.53% · ORKA (Oruka Therapeutics, Inc.) USD 96.95 +1.02% · IOVA (Iovance Biotherapeutics, Inc.) USD 10.26 -30.72% · IONS (Ionis Pharmaceuticals, Inc.) USD 45.43 +1.18% · AXSM (Axsome Therapeutics, Inc.) USD 144.78 +0.88% · PRAX (Praxis Precision Medicines, Inc) USD 286.55 +0.37%

## More
Full page: https://nexqual.com/RARE · Free keyless MCP server with the same data and citations: https://mcp.nexqual.com/mcp · Guide for agents: https://nexqual.com/agents · https://nexqual.com/llms.txt

## Supply chain and relationships (SEC annual reports)

Named in RARE's own 10-K filed 2026-02-18 (https://www.sec.gov/Archives/edgar/data/1515673/000119312526057329/rare-20251231.htm):

| Relationship | Company | Sentence in the filing |
|---|---|---|
| Partner | Kyowa Kirin | "Please see "—License and Collaboration Agreements—Approved Products— Kyowa Kirin Co., Ltd." for a description of our collaboration and license agreement with KKC." |
| Partner | REGENXBIO Inc. (RGNX) | "Please see "—License and Collaboration Agreements—Clinical Product Candidates—REGENXBIO Inc." for a description of our license agreement with REGENXBIO Inc." |
| Partner | GeneTx Biotherapeutics | "Please see "—License and Collaboration Agreements—Clinical Product Candidates—GeneTx" for a description of our license agreement with GeneTx Biotherapeutics LLC, or GeneTx." |
| Partner | Daiichi Sankyo | "We have in the past sought and may in the future seek funds through a sale of future royalty payments similar to our transactions with Royalty Pharma and OMERS or through collaborative partnerships, strategic alliances, …" |
| Partner | Cowen and Company | "In February 2024, we entered into a Sales Agreement with Cowen and Company, LLC, or Cowen, pursuant to which the Company may offer and sell shares of the Company's common stock having an aggregate offering proceeds up to…" |

Other companies that name RARE in their own reports (relationship from RARE's side):

| Relationship | Company | Their filing | Sentence |
|---|---|---|---|
| partner | Solid Biosciences Inc. (SLDB) | 10-K 2026-03-19 | "In October 2020, the license agreement was amended such that we were required to pay the University of Washington $375 thousand in connection with the execution of the collaboration and license agreem…" |
| partner | ABEONA THERAPEUTICS INC. (ABEO) | 10-K 2026-03-17 | "Under the terms of our agreement with Ultragenyx, we are eligible to receive payments based on the achievement of certain sales" |
| partner | REGENXBIO Inc. (RGNX) | 10-K 2026-03-05 | "Prior to the maturity date, interest and principal under the 2025 Royalty Bond shall be paid quarterly to HCR solely using proceeds received from certain specified royalties, milestone payments, licen…" |

## Company outlook (8-K filed 2026-08-04)

- "Guidance • Total revenue in the second quarter of 2026 was $214 million."
- "The company reaffirms its full year 2026 total revenue guidance of $730 million to $760 million, which excludes revenue from potential new product launches. • Crysvita revenue in the second quarter of 2026 was $156 million, consistent with expected seasonality in the U.S. and Canada and ordering patterns in Latin America."
- "The company reaffirms its full year 2026 Crysvita revenue guidance of $500 million to $520 million . • Dojolvi revenue in the second quarter 2026 was $27 million."
- "The company reaffirms its full year 2026 Dojolvi revenue guidance of $100 million to $110 million. • Evkeeza® revenue in the second quarter 2026 was $21 million, driven by increased demand from new country launches and early access. • Mepsevii® revenue in the second quarter 2026 was $10 million."

Analysts expect $183.7M for the current quarter, -75.3% vs the guidance midpoint $745.0M. Source: https://www.sec.gov/Archives/edgar/data/1515673/000119312526332802/rare-ex99_1.htm

## Analyst estimates and revisions

| Period | EPS est. | 90d ago | Change | Revenue est. | Analysts |
|---|---|---|---|---|---|
| Current quarter (ends 2026-09-30) | $-0.75 | $-0.73 | −$0.02 | $183.7M | 6 |
| Next quarter (ends 2026-12-31) | $-0.66 | $-0.39 | −$0.27 | $229.7M | 6 |
| Current fiscal year (ends 2026-12-31) | $-4.08 | $-4.19 | +$0.11 | $760.2M | 6 |
| Next fiscal year (ends 2027-12-31) | $0.23 | $1.55 | -85.4% | $997.0M | 6 |

## Options: expected move (Cboe, 15-min delayed)

Expected move by 2026-11-06 (includes earnings 2026-11-03): ±9.4%; last 8 reports averaged ±6.1%.

| Expiration | Days | ATM IV | Expected move |
|---|---|---|---|
| 2026-10-30 | 1 | 153.2% | ±8.2% |
| 2026-11-06 | 8 | 74.2% | ±9.4% |
| 2026-11-13 | 15 | 59.3% | ±9.9% |
| 2026-11-20 | 22 | 59.1% | ±11.7% |
| 2026-11-27 | 29 | 59.0% | ±13.5% |
| 2026-12-04 | 36 | 87.0% | ±21.9% |
| 2026-12-18 | 50 | 63.1% | ±19.0% |
| 2027-01-15 | 78 | 67.3% | ±25.2% |

## Earnings history and stock reaction
| Results filed | Quarter | EPS | Consensus | Surprise | Stock reaction |
|---|---|---|---|---|---|
| Aug 4, 2026 | Quarter ended Jun 30, 2026 | −$0.90 | −$1.00 | Beat +10.0% | -0.4% |
| May 5, 2026 | Quarter ended Mar 31, 2026 | −$1.54 | −$1.46 | Miss −5.5% | +4.5% |
| Feb 12, 2026 | Quarter ended Dec 31, 2025 | −$1.29 | −$1.17 | Miss −10.3% | -14.1% |
| Jan 12, 2026 | Quarter ended Dec 31, 2025 | −$1.29 | −$1.17 | Miss −10.3% | +7.1% |
| Nov 4, 2025 | Quarter ended Sep 30, 2025 | −$1.81 | −$1.21 | Miss −49.6% | -4.8% |
| Aug 5, 2025 | — | — | — | — | -1.4% |
| May 6, 2025 | — | — | — | — | -14.0% |
| Feb 13, 2025 | — | — | — | — | -2.2% |
EPS as reported against the analyst consensus before the release; surprise uses the cent-rounded figures shown (in dollars when EPS and consensus have opposite signs). Stock reaction = close before the results to close after (the first close following a release after the bell or before the open).

## Recent SEC filings (last 120 days)
| Filed | Form | What it says | Filing |
|---|---|---|---|
| 2026-10-20 | Schedule 13G | Madison Avenue International LP, Madison Avenue Partners, LP: 6.4% of shares, passive 5%+ holder | https://www.sec.gov/Archives/edgar/data/1515673/000101359426001062/0001013594-26-001062-index.htm |
| 2026-10-19 | 8-K | Other material event — “On October 19, 2026, Ultragenyx Pharmaceutical Inc. (the "Company") and Baylor Research Institute ("BRI") entered into a settlement agreement with Navinta LLC ("Navinta") resolving patent litigation related to patents covering the Company's DOJOLVI (triheptanoin) oral liquid. The litigation, which was filed in the United States District Court for the District of New Jersey, resulted from the submission by Navinta of an Abbreviated New Drug Application ("ANDA") to the U.S. Food and Drug Administration seeking…” | https://www.sec.gov/Archives/edgar/data/1515673/000119312526424739/0001193125-26-424739-index.htm |
| 2026-10-14 | 8-K | Entered a material agreement — “On October 8, 2026, Ultragenyx Pharmaceutical Inc. (the "Company") entered into an asset purchase agreement (the "PRV Asset Purchase Agreement"), pursuant to which the Company agreed to sell a Rare Pediatric Disease Priority Review Voucher ("PRV") to the buyer. The Company was awarded the PRV by the U.S. Food and Drug Administration ("FDA") on September 17, 2026 upon approval of FAYUVI TM (rebisufligene etisparvovec-hopf) also known as UX111, for the treatment of pediatric patients with mucopolysaccharidosis type…” | https://www.sec.gov/Archives/edgar/data/1515673/000119312526421079/0001193125-26-421079-index.htm |
| 2026-10-07 | 8-K | Entered a material agreement — “On October 6, 2026, Ultragenyx Pharmaceutical Inc. (the "Company") entered into an asset purchase agreement (the "PRV Asset Purchase Agreement"), pursuant to which the Company agreed to sell a Rare Pediatric Disease Priority Review Voucher ("PRV") to the buyer. The Company was awarded the PRV by the U.S. Food and Drug Administration ("FDA") on August 19, 2026 upon approval of GENGLYCOS (pariglasgene brecaparvovec-opnr), also known as DTX401, in adult and pediatric patients eight years and older with glycogen…” | https://www.sec.gov/Archives/edgar/data/1515673/000119312526416251/0001193125-26-416251-index.htm |
| 2026-09-17 | 8-K | Other material event — “On September 17, 2026, Ultragenyx Pharmaceutical Inc. ("Ultragenyx" or the "Company") announced that the U.S. Food and Drug Administration (the "FDA") granted standard full approval of FAYUVI (rebisufligene etisparvovec-hopf), also known as UX111, for the treatment of pediatric patients with mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome Type A). FAYUVI is the first-ever FDA-approved treatment for Sanfilippo syndrome Type A, a progressive and fatal neurodegenerative disease, and the second gene…” | https://www.sec.gov/Archives/edgar/data/1515673/000151567326000006/0001515673-26-000006-index.htm |
| 2026-09-02 | 8-K | Other material event — “On September 2, 2026, Ultragenyx Pharmaceutical Inc. ("Ultragenyx" or the "Company") announced results from the Phase 3 Aspire study for apazunersen (GTX-102) in Angelman syndrome. The study did not achieve the primary endpoint of change from Baseline in Bayley-4 cognitive raw score nor the key secondary endpoint of net response in Multidomain Responder Index ("MDRI"). The safety profile observed in Aspire was consistent with Phase 1/2. In Aspire , the randomized groups were comparable at baseline and consistent…” | https://www.sec.gov/Archives/edgar/data/1515673/000119312526380165/0001193125-26-380165-index.htm |
| 2026-08-19 | 8-K | Other material event — “On August 19, 2026, Ultragenyx Pharmaceutical Inc. ("Ultragenyx" or the "Company") announced the U.S. Food and Drug Administration (the "FDA") granted accelerated approval for GENGLYCOS (pariglasgene brecaparvovec-opnr), also known as DTX401, in adult and pediatric patients eight years and older with glycogen storage disease type Ia (GSDIa). Clinical Program and Post-Marketing Study Requirements Supporting Accelerated Approval of GENGLYCOS The approval of GENGLYCOS is based on positive data from the 48-week…” | https://www.sec.gov/Archives/edgar/data/1515673/000119312526357185/0001193125-26-357185-index.htm |
| 2026-08-14 | Schedule 13G | RTW Investments, LP, Roderick Wong: 7.9% of shares, passive 5%+ holder | https://www.sec.gov/Archives/edgar/data/1515673/000149321526000161/0001493215-26-000161-index.htm |
| 2026-08-13 | Schedule 13G | passive 5%+ holder | https://www.sec.gov/Archives/edgar/data/1515673/000116755726000169/0001167557-26-000169-index.htm |
| 2026-08-11 | 8-K | Other material event | https://www.sec.gov/Archives/edgar/data/1515673/000119312526343525/0001193125-26-343525-index.htm |
| 2026-08-07 | Schedule 13G | passive 5%+ holder | https://www.sec.gov/Archives/edgar/data/1515673/000009375126000549/0000093751-26-000549-index.htm |
| 2026-08-05 | 10-Q | Quarterly report · period ended Jun 30, 2026 | https://www.sec.gov/Archives/edgar/data/1515673/000119312526333233/0001193125-26-333233-index.htm |
| 2026-08-04 | 8-K | Quarterly results | https://www.sec.gov/Archives/edgar/data/1515673/000119312526332802/0001193125-26-332802-index.htm |
| 2026-07-31 | Schedule 13G/A | passive 5%+ holder (amended) | https://www.sec.gov/Archives/edgar/data/1515673/000210012126001001/0002100121-26-001001-index.htm |
Source: SEC EDGAR. Quotes are the opening words of each item as filed.