TickerTalks
Browse all tickers →
TickerTalks›$RARE
RARARE

Ultragenyx Pharmaceutical Inc.

Hot onTrending downwardX chatter spiked vs its recent normMoving on elevated volumeBacked by solid revenue growth
$RARE·$1.5B·Biotechnology·Healthcare
$15.30+3.0%1Y-51.1%
Mentions · last 7 days
2026-08-28: 37 posts2026-08-29: 11 posts2026-08-30: 20 posts2026-08-31: 43 posts2026-09-01: 40 posts2026-09-02: 116 posts2026-09-03: 238 posts506
Price updated 10h ago·X counts updated 1d ago
RARARE
$RAREUltragenyx Pharmaceutical Inc.
$15.30+3.03%506 posts
AI analysisFundamentalsVoices on X
Loading…

AI verdict & sentimentAI analysisGenerated by AI from underlying data

Top X posts

Today's AI verdict on what's driving $RARE, plus how loud the X conversation is and which way it's leaning.

AI analysis

TickerTalks’ read on the fundamentals and what’s driving the move.

Broken storySelling offAI verdict · as of 2026-09-04

Falling on heavy selling — points lower unless it turns around.

Ultragenyx's Angelman syndrome Phase 3 missed — the pipeline just lost its most anticipated readout.

Ultragenyx is a rare-disease biotech with a small commercial portfolio (Crysvita for XLH, Mepsevii, Dojolvi, Evkeeza) and a pipeline stacked with gene therapies. The Phase 3 Aspire study of GTX-102 in Angelman syndrome was the marquee readout — and it just failed the primary endpoint, taking the stock down 44% in a session.

  • The Angelman miss is the whole story: GTX-102 failed its Phase 3 primary endpoint, and long-time bulls are calling for CEO Emil Kakkis' resignation while Martin Shkreli publicly framed the company as an M&A target — the pipeline just lost its biggest single asset, and the equity is now valued primarily on the commercial base and the remaining pipeline (DTX401 for GSD1a, UX143 for OI, Sanfilippo).
  • The commercial base is small but growing: Q2 revenue landed at $214M (+29% year-over-year) with a 84% gross margin and negative operating margin — Crysvita and Evkeeza are compounding, and the last four earnings prints show revenue growth in the 25-30% range, so this is a base worth having even as the pipeline resets.
  • The near-term catalyst is now the Sanfilippo PDUFA on September 19: this is the specific dated event that decides whether the pipeline still has a positive tail — success adds a commercial line that partly offsets the Angelman miss; another miss into a stock at the 4th percentile of the 52-week range would extend the drawdown further.

The forward read: the September 19 Sanfilippo PDUFA is the near-term decision point — approval stabilizes the equity as an M&A target with modest downside; a CRL or delay resets the low and forces cost-cutting to be the whole story. Watch the FDA advisory-committee background documents that publish typically 2 business days before the PDUFA as the leading indicator.

Agrees with X sentimentAgree with the bearish anchor — a Phase 3 primary-endpoint miss on the most-anticipated pipeline asset is a legitimate broken-story event; the M&A/discount-value bull thread has real optionality but requires the Sanfilippo PDUFA to land before it becomes a base case.

What to watch: September 19 Sanfilippo PDUFA decision — approval stabilizes the equity as an M&A target; a CRL or delay resets the low; FDA advisory-committee background docs typically publish 2 business days before as the leading indicator.

On the calendar: 2026-09-19 — Sanfilippo PDUFA decision

X sentiment

What the X crowd is saying right now — descriptive, summarised from the day’s posts.

Bearish sentiment46 posts analyzed · as of 2026-09-04

$RARE (Ultragenyx) chatter is bearish after the Phase 3 Aspire study for apazunersen (GTX-102) in Angelman syndrome missed its primary endpoint — stock down 40-45% AH. Bulls try to salvage the thesis: 4 approved drugs, revenue potentially reaching $800M, next PDUFA Sept 19, cheap valuation could attract acquirers (BMRN). Traders opened bounce positions at €12.12. But the tone is dominated by 'gut punch' posts, criticism of the licensed-vs-foundational-IP pipeline, and multiple 'done with biotech' capitulations. Bearish overall with a small contrarian bounce trade.

Read the AI verdict + X sentiment for $RARE

  • One-line verdict on what's driving the move — fundamentals, momentum, both, or an event
  • Next dated catalyst when there is one (earnings, deal closing, activist clock)
  • X crowd read with bullish/bearish call + post volume
Free, forever. No credit card.

What it does

Plain-English summary of the business — what they sell and how they make money.

Ultragenyx Pharmaceutical Inc. is a biopharmaceutical firm dedicated to discovering, developing, and commercializing innovative treatments for rare and ultra-rare genetic conditions. Its operations span North America, Europe, and other international markets. The company's portfolio of marketed biologic products addresses several serious diseases. This includes Crysvita (burosumab), an antibody that targets fibroblast growth factor 23, used to treat X-linked hypophosphatemia and tumor-induced osteomalacia. Mepsevii offers enzyme replacement therapy for both pediatric and adult patients suffering from Mucopolysaccharidosis VII. Dojolvi is available for individuals with long-chain fatty acid oxidation disorders, while Evkeeza (evinacumab) provides a treatment option for homozygous familial hypercholesterolemia. Ultragenyx also boasts a robust pipeline of investigational therapies. Notable candidates include DTX401, an adeno-associated virus 8 (AAV8) gene therapy for glycogen storage disease type Ia, and DTX301, another AAV8 gene therapy aimed at ornithine transcarbamylase deficiency. Other promising developments feature UX143, a human monoclonal antibody for osteogenesis imperfecta; GTX-102, an antisense oligonucleotide designed for Angelman syndrome; UX701, targeting Wilson disease; and UX053, in development for glycogen storage disease type III. To advance its research and development efforts, Ultragenyx Pharmaceutical Inc. maintains strategic collaboration and licensing agreements with numerous partners, such as Kyowa Kirin Co., Ltd., Saint Louis University, REGENXBIO Inc., Bayer Healthcare LLC, GeneTx, Mereo, University of Pennsylvania, Arcturus Therapeutics Holdings Inc., Solid Biosciences Inc., and Daiichi Sankyo Co., Ltd. The company was founded in 2010 and operates from its headquarters in Novato, California.

Industry overviewAI analysisGenerated by AI from underlying data

Where Biotechnology sits in its cycle right now — and what that implies for $RARE.

Biotechnology · Healthcare

GLP-1 oral formulation competition — emerging clinical data from pipeline challengers — is the central bifurcation: platform holders (LLY, NVO) defend on efficacy breadth while precision oncology names decouple entirely. FDA catalyst cadence over the next 60 days sets the near-term tone.

Top industry ETF

$IBBiShares Biotechnology ETF
+12.1%YTD
+51.0%1Y

Fundamentals & catalyst

Profitability, valuation, and the next earnings event — at a glance, with rule-of-thumb signals.

Key ratios

P/E
-4.5How much investors are paying per dollar of profit the company actually earned in the last 12 months. Lower means the stock looks cheaper relative to earnings.~15–25 is typical for the S&P 500; high-growth names trade 30+; hyper-growth or speculative can be 100+ or negative.
ROIC
-53.5%What percentage return the business earns on every dollar of capital (equity + debt) deployed in operations. The cleanest measure of business quality.Above ~15% is high-quality; consistently above 25% suggests a real moat. Below the company's cost of capital is value-destroying.
Op margin
-73.8%Operating profit (after sales, marketing, R&D, and overhead but before interest and taxes) as a percentage of revenue. The clearest view of how well the underlying business is run.Mature business above 20% is healthy; software businesses can run 30%+; commodity / retail businesses operate in single digits.
FCF yield
-18.7%Free cash flow (operating cash flow minus capex) divided by the company's market cap. The cash-on-cash return you'd get owning the whole business at today's price.Above ~5% is attractive; below ~2% means you're paying up for growth. Capital-light businesses (software) run higher than capital-heavy ones (utilities).
P/S
3.6Same idea as P/E but per dollar of revenue. Useful for companies that aren't profitable yet, where P/E is meaningless.Under ~2 is cheap; software / SaaS often runs 8–15; well above 20 implies the market is pricing in very high future growth.
ROE
392%Net income as a percentage of shareholders' equity. Similar to ROIC but counts only the equity side.Above 20% is strong, but can be inflated by leverage — a heavily indebted company can show high ROE with weak underlying ROIC.
Gross margin
83.1%Revenue minus the direct cost of producing what was sold, as a percentage of revenue. The first read on whether the product is structurally profitable.Software / SaaS is typically 70%+; consumer goods 30–50%; commodity / hardware businesses can be under 20%.
D/E
-4.1Total debt divided by shareholders' equity. Measures how much the business runs on borrowed money versus owner capital.Under 1 is conservative; 1–2 is typical for mature businesses; over 2 is leveraged and more sensitive to interest rates.

Past earnings

QuarterReportedActualEstimateSurprise
Q2 2026Aug 4, 2026$-0.90$-1.22+26.2%
Q1 2026May 5, 2026$-1.84$-1.49-23.7%
Q4 2025Feb 12, 2026$-1.29$-1.20-7.5%
Q3 2025Nov 4, 2025$-1.81$-1.23-47.2%
Next earningsTue, Nov 3·consensus EPS $-0.63

Quarterly trend

QuarterRevenueYoYGrossOpEPSFCF
Q2 FY26$214.0M+28.5%84.1%-35.0%$-0.90$-98.0M
Q1 FY26$136.0M-2.4%77.9%-124%$-1.84$-198.0M
Q4 FY25$207.0M+25.5%86.0%-55.1%$-1.29$-100.8M
Q3 FY25$159.9M+14.7%82.5%-107%$-1.81$-92.7M

Forward consensus

5-year forecast · up to 14 analysts
FYRevenueRangeEPSRangeAnalysts
FY26$754.4M$739.0M – $787.7M-$4.05-$5.34 – -$1.0414
FY27$1.0B$752.2M – $1.5B-$0.63-$3.45 – $5.8614
FY28$1.3B$948.4M – $1.5B$0.60-$1.85 – $4.3814
FY29$1.6B$1.4B – $2.2B$2.64$2.07 – $3.878
FY30$2.2B$1.8B – $3.0B$5.33$4.18 – $7.8012

Setup & momentum

Volume, range, and moving-average position — the technical setup driving short-term moves.

Right now

Vol vs 30dToday's traded share volume divided by the average over the prior 30 trading days. ≥3× signals unusual interest; below 1× is quiet.3.7×Today's traded share volume divided by the average over the prior 30 trading days. ≥3× signals unusual interest; below 1× is quiet.
52w rangeWhere the latest close sits between the 52-week low (0%) and high (100%). Above 80% is extended; below 30% is basing or in a downtrend.6%Where the latest close sits between the 52-week low (0%) and high (100%). Above 80% is extended; below 30% is basing or in a downtrend.
vs 50d MALatest close vs the 50-day simple moving average. Positive = short-term trend is up.-44.8%Latest close vs the 50-day simple moving average. Positive = short-term trend is up.
vs 200d MALatest close vs the 200-day simple moving average. Positive = long-term trend is up.-41.1%Latest close vs the 200-day simple moving average. Positive = long-term trend is up.

Float & profile

FloatMid float · 94.6M shFree-float shares — the slice of issued stock actually available to trade. Lower buckets squeeze harder on a catalyst.Traded today12.1% of floatToday's volume as a percent of the free float. Above 5% on a single day is unusually high turnover for the available share count.β0.315-year weekly beta vs the S&P 500. Above 1.5 means the stock typically moves more than the index; below 0.8 moves less.

Insider activity

Recent open-market buys and sells by officers and directors — flagged when multiple insiders cluster.

Recent transactions

SellAug 3Parschauer Karah HerdmanEVP and Chief Legal Officer1.9K sh$47KSellAug 3Howard HornCFO4.7K sh$117KSellJul 1Howard HornCFO4.7K sh$156KSellJun 15Sanders Corazon (corsee) D.Director2.0K sh$50KSellJun 15Parschauer Karah HerdmanEVP and Chief Legal Officer1.9K sh$47KSellJun 1Howard HornCFO4.7K sh$111KSellMay 18Shehnaaz SulimanDirector5.7K sh$144KSellMay 5Eric CrombezEVP and Chief Medical Officer344 sh$9KSellMay 1Howard HornCFO4.7K sh$117KSellApr 1Howard HornCFO4.7K sh$98K
1–10 of 11
+ 15 other (15 awards) in window

See when $RARE insiders are putting their own money in

  • Real-time open-market buys and sells from Form 4 filings
  • Cluster-buy detection when multiple insiders pile in at once
  • 30 / 60 / 180-day windows so you can spot building conviction
Free, forever. No credit card.

Recent news

Latest headlines from major outlets, sourced and timestamped — context for whatever just moved.

Why Is Ultragenyx (RARE) Up 6.4% Since Last Earnings Report?zacks.com·2d agoRARE Stock Down 46% as Phase III Angelman Syndrome Study Misses Goalszacks.com·2d agoQUICK SPARK: Pharma Bro Martin Shkreli Says Ultragenyx Should Be M&A Targetbenzinga.com·2d agoUltragenyx Forced To Cut Costs After Disappointing Phase 3 Resultsbenzinga.com·2d agoHere Are Thursday’s Top Wall Street Analyst Research Calls: Broadcom, Deere & Company, Dell Technologies, Devon Energy, Moderna, Permian Resources, PG&E, Thermo Fisher, Viper Energy, and More247wallst.com·2d ago

More in Biotechnology

Peers in the same group — one click to compare setups, fundamentals, and chatter.

$AURA$MRNA$ALT$NWBO$SLS$IBRX$ABCL$ABVX
Voices on X · top 15 · last 7 days

TickerTalks is a research tool, not financial advice. We surface social-attention data; we do not make stock recommendations. Past attention is not predictive of future price movements.

PrivacyTermsSupport