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Pipeline (10 active programs)

Discontinued (2)

• apazunersen — Angelman Syndrome · strategic deprioritization; wind-down of all ongoing studies as part of expense reduction
• rivunatpagene miziparvovec — Wilson Disease · failed to achieve study goals; majority of patients did not achieve biochemical response

Capital (as filed)

Cash

$150M cash and cash equivalents · Sep 30, 2026 (10-Q)

Short-term investments

$116M Sep 30, 2026

Long-term investments

$135M Sep 30, 2026

Operating cash flow

−$43.0M quarter Jun 30, 2026 → Sep 30, 2026, year-to-date difference (10-Q)

As stated in coverage

$401M cash per the update of Nov 4, 2026 — the company's own words as read there, not the filed figure

Cash, investments and operating cash flow are the company's own XBRL facts as filed with the SEC, each shown with its period end and tag. As-stated figures come from the company's own words as read in Biotech Distilled coverage, dated, and routinely count securities and planned financing that a balance sheet does not. Offering rows are the SEC cover pages as filed. Not investment advice. Figures as of Nov 7, 2026.

Biotech Distilled coverage (15)

• 2026-11-04 UPDATE: Ultragenyx (RARE) - The Mall Closes a Wing 🔒 Paid
• 2026-10-15 UPDATE: Ultragenyx (RARE) - Two Vouchers, One Shrug 🔒 Paid
• 2026-10-08 UPDATE: Ultragenyx (RARE) - The Voucher Cashed at Double 🔒 Paid
• 2026-09-17 UPDATE: Ultragenyx (RARE) - The Last Card Flipped, and It Was Better Than the Deck Promised 🔒 Paid
• 2026-09-02 UPDATE: Ultragenyx (RARE) - The Crown Jewel Broke 🔒 Paid
• 2026-08-19 UPDATE: Ultragenyx (RARE) - Bedford Passed 🔒 Paid
• 2026-08-12 UPDATE: Ultragenyx (RARE) - One of My Three Sell Triggers Just Got Defused 🔒 Paid
• 2026-08-05 UPDATE: Ultragenyx (RARE) - The Burn Rate Just Halved, and the Angelman Clock Got a Month 🔒 Paid
• 2026-05-06 UPDATE: Ultragenyx (RARE) - The Angelman Safety Signal Just Got Quieter, and the Cash Got Lighter 🔒 Paid
• 2026-04-27 UPDATE: Ultragenyx (RARE) - Both PDUFA Dates Are Now on the Calendar 🔒 Paid
• 2026-03-14 UPDATE: Ultragenyx (RARE) - The Science Delivers (Again), But the Factory Remains the Boss 🔒 Paid
• 2026-03-01 UPDATE: Ultragenyx (RARE) - A Date With Destiny 🔒 Paid
• 2026-02-21 UPDATE: Ultragenyx (RARE) - 10-K Autopsy: Suing the CDMO and Mortgaging the Crown Jewels 🔒 Paid
• 2026-02-14 Update: Ultragenyx (RARE) - Q4 2025 Report 🔒 Paid
• 2026-02-09 Ultragenyx Pharmaceuticals (RARE): Scientific Deep Dive for Lead and Pipeline Products

Articles open on Substack. Some are subscriber-only; those marked ★ Distilled Pro are for Distilled Pro members.

Verdict history (15 calls)

Every verdict Biotech Distilled has published on Ultragenyx Pharmaceutical, oldest first — the full history, never a selection. How calls are scored: the track record.

1. 2026-02-09 Watch Ultragenyx Pharmaceuticals (RARE): Scientific Deep Dive for Lead and Pipeline Products Cash burn is too high relative to immediate revenue growth and CMC/manufacturing risk at Bedford is too elevated to justify a long position ahead of the gene therapy PDUFA dates.
2. 2026-02-14 Watch Update: Ultragenyx (RARE) - Q4 2025 Report Layoffs bridge the dilution gap but margin for error is zero; another CMC-related CRL would be fatal to the thesis, so waiting for Q3 PDUFA clearances remains the right posture.
3. 2026-02-21 Watch UPDATE: Ultragenyx (RARE) - 10-K Autopsy: Suing the CDMO and Mortgaging the Crown Jewels The Catalent lawsuit and Bedford facility history make manufacturing risk too glaring to take a long position ahead of FDA facility inspections, despite the cash runway being secured.
4. 2026-03-01 Watch UPDATE: Ultragenyx (RARE) - A Date With Destiny DTX401 clinical risk dropped with no AdCom, but manufacturing risk remains Code Red due to active Catalent litigation and prior UX111 CRL; keep powder dry until facility inspection updates.
5. 2026-03-14 Watch UPDATE: Ultragenyx (RARE) - The Science Delivers (Again), But the Factory Remains the Boss DTX301 Phase 3 win raises sum-of-parts value but CMC bottleneck is unchanged; remain on sidelines until Bedford is cleared.
6. 2026-04-27 Watch UPDATE: Ultragenyx (RARE) - Both PDUFA Dates Are Now on the Calendar Both PDUFA dates are now set but manufacturing risk is unchanged at Code Red; the 13% run-up since March eats into margin of safety with no new manufacturing data to underwrite it.
7. 2026-05-06 Watch UPDATE: Ultragenyx (RARE) - The Angelman Safety Signal Just Got Quieter, and the Cash Got Lighter GTX-102 safety risk improved meaningfully with 3+ years of clean LTE data, but manufacturing risk stays Code Red and the gating event remains the August 23 DTX401 PDUFA.
8. 2026-08-05 Watch speculative UPDATE: Ultragenyx (RARE) - The Burn Rate Just Halved, and the Angelman Clock Got a Month Halved burn rate, record revenue, and Aspire data narrowed to September/October shift the asymmetry enough to move off pure sidelines, but Bedford inspection remains the binary and pre-PDUFA weakness may offer a better entry.
9. 2026-08-12 Speculative buy speculative UPDATE: Ultragenyx (RARE) - One of My Three Sell Triggers Just Got Defused Esjay Dojolvi settlement pushes generic entry to 2033, removing one of three sell triggers and improving the commercial base, but manufacturing risk stays Code Red twelve days from the PDUFA.
10. 2026-08-19 Buy core UPDATE: Ultragenyx (RARE) - Bedford Passed FDA approved GENGLYCOS manufactured entirely at Bedford, retiring the existential manufacturing risk; with UX111 PDUFA and Angelman readout still ahead, asymmetry has flipped to building a position.
11. 2026-09-02 Hold UPDATE: Ultragenyx (RARE) - The Crown Jewel Broke Phase 3 Aspire missed primary and all secondary endpoints with zero separation across five domains, eliminating the Angelman rerating; the commercial base and approved gene therapy remain intact but the premium valuation case evaporated.
12. 2026-09-17 Buy core UPDATE: Ultragenyx (RARE) - The Last Card Flipped, and It Was Better Than the Deck Promised FAYUVI received full standard approval (not accelerated) with a 23.5-point cognitive separation at p<0.0001, Bedford cleared twice in 30 days, two PRVs in hand, and the stock is 45% below pre-Aspire levels — the floor got taller while the price dropped.
13. 2026-10-08 Buy core UPDATE: Ultragenyx (RARE) - The Voucher Cashed at Double GENGLYCOS PRV sold for $210M (~$168M net after NIH cut), roughly double the modeled ~$100M, closing the dilution gap with non-dilutive cash; second voucher still available.
14. 2026-10-15 Buy core UPDATE: Ultragenyx (RARE) - Two Vouchers, One Shrug Second PRV sold for $203M, bringing combined net voucher proceeds to ~$370M — nearly double the modeled amount — without diluting shareholders; balance sheet is now a cushion, not a countdown.
15. 2026-11-04 Buy core UPDATE: Ultragenyx (RARE) - The Mall Closes a Wing 20% layoff, wind-down of apazunersen and UX701, and R&D cut to under $350M transforms RARE into a commercial company with two de-risked gene therapy launches and ~$750M pro forma cash representing nearly 45% of market cap.

• Long call · opened Aug 12, 2026 at $26.86 · -42.6% vs XBI -4.5% (Nov 6, 2026)

Every verdict Biotech Distilled has published on this company, in order, with the post it came from — the full history, never a selection. Position figures are the same ones shown on /track-record, computed by the same method. Editorial opinion, not investment advice.

Timing, in the company's words (3 quotes from 2 filings)

Sentences quoted verbatim from the company's SEC filings, newest filing first. Each sits under the filing it appeared in; the "matched to" tag names the calendar row it was read for.

• 2026-11-04 10-Q in filings ↓

  • “UX016 for the treatment of GNEM In November 2026, we announced that the Phase 1/2 study has begun and is evaluating safety and dose of UX016 and its impact on muscle strength in adults with GNEM.”

    matched to DTX301 (OTC deficiency, second primary endpoint) · topline data · read as November 2026
• 2026-08-05 10-Q in filings ↓

  • “The FDA granted the BLA Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) action date of August 23, 2026.”

    matched to DTX401 · regulatory decision · read as August 23, 2026

  • “UX111 for the treatment of MPS IIIA In April 2026, we announced that the FDA had accepted our resubmitted BLA seeking accelerated approval for UX111 as a treatment for patients with Sanfilippo syndrome Type A. The FDA granted the BLA Priority Review and assigned a PDUFA action date of September 19, 2026.”

    matched to UX111 · regulatory decision · read as September 19, 2026

Timing sentences quoted from the company's own SEC filings, each under the filing it appeared in. The 'matched to' tag is the calendar row the sentence was read for; the filing is the source. Not investment advice.

Filings & releases (36)

SEC filings from EDGAR and press releases captured from the company's investor-relations site, newest first. Releases captured from May 6, 2026; filings from March 2026. Summaries come from the BD update that read the document; "BD extracted" counts point at the pipeline table above. All companies →

• Nov 6, 2026ReleaseUltragenyx Announces the Marketing Authorisation Application (MAA) for its Investigational Gene Therapy for MPS IIIA (Sanfilippo syndrome Type A) is Under Review with the UK Medicines and Healthcare Pvia aggregator
• Nov 4, 202610-QQuarterly reportThe Q3 10-Q puts real numbers on the post-Aspire thesis for the first time: a $43M operating cash draw, ~$750M of pro-forma liquidity once both PRV sales close, and zero gene therapy revenue yet — the launch ramp that determines whether the 2027 profitability target is real is now the only story left to watch.
• Nov 3, 2026ReleaseUltragenyx Reports Third Quarter 2026 Financial Results, Announces Substantial Expense Reductions, and Reaffirms Profitability in 2027→ BD updateSummary on the 8-K
• Nov 3, 20268-KCurrent report: acquisition / disposition, results of operations, exit / restructuring costs · EX-99.1→ BD updateQ3 2026 earnings confirm the transformation is real: operating cash burn collapsed to $43M for the quarter, a second 20% workforce reduction is underway, UX701 is terminated, Angelman is officially wound down, and R&D spending is being cut in half — but 2027 GAAP profitability is reaffirmed and the balance sheet now has ~$750M+ of cash plus PRV proceeds incoming.
• Oct 27, 2026ReleaseUltragenyx to Host Conference Call for Third Quarter 2026 Financial Results and Corporate Update
• Oct 19, 20268-KCurrent report: other events
• Oct 14, 2026ReleaseUltragenyx Enters into Agreement to Sell Rare Pediatric Disease Priority Review Voucher for $203 Million→ BD updateSummary on the 8-K
• Oct 14, 20268-KCurrent report: material agreement→ BD updateUltragenyx just sold its second Priority Review Voucher in one week — the FAYUVI PRV — for $203M gross, roughly double my ~$100M model. Two vouchers, $413M combined, and the Dilution Gap that dominated this year's coverage is now closed with real cash rather than modeled cash.
• Oct 7, 2026ReleaseUltragenyx Enters into Agreement to Sell Rare Pediatric Disease Priority Review Voucher for $210 Million→ BD updateSummary on the 8-K
• Oct 7, 20268-KCurrent report: material agreement→ BD updateUltragenyx just monetized the GENGLYCOS Priority Review Voucher for $210M — well above the ~$100M historical average I'd been penciling in — with a 20% NIH toll that still leaves ~$168M in net non-dilutive proceeds, meaningfully extending runway and reinforcing the post-Aspire commercial floor thesis.
• Oct 2, 2026ReleaseUltragenyx Announces Marketing Authorisation Application (MAA) Submission to the European Medicines Agency (EMA) for the First Investigational Gene Therapy for MPS IIIA (Sanfilippo Syndrome Type A)via aggregator
• Sep 17, 2026ReleaseUltragenyx Announces Approval of FAYUVI™ Gene Therapy, the First-Ever FDA-Approved Treatment for Sanfilippo Syndrome Type A (MPS IIIA)→ BD updateSummary on the 8-K
• Sep 17, 20268-KCurrent report: other events→ BD updateFAYUVI (UX111) just received standard full FDA approval for Sanfilippo syndrome Type A — a historic first-in-disease milestone that lands 15 days after the crown jewel Angelman failure cratered the stock 45%, and the disconnect between the approval quality and the price tells a story worth unpacking.
• Sep 2, 2026ReleaseUltragenyx Announces Phase 3 Aspire results in Angelman Syndrome→ BD updateSummary on the 8-KBD extracted 1 program update from this document → RARE pipeline
• Sep 2, 20268-KCurrent report: other events→ BD updateThe Phase 3 Aspire trial for apazunersen (GTX-102) in Angelman syndrome failed both the primary and key secondary endpoints today — the crown jewel missed, the 2026 bull case is broken, and more cost cuts are coming.
• Sep 1, 2026ReleaseUltragenyx Announces the Publication of a Successful 96-Week Randomized, Placebo-Controlled Trial with Crossover Treatment of GENGLYCOS™ (also known as DTX401) AAV Gene Therapy in GSDIa in The JournalBD extracted 1 program update from this document → RARE pipeline
• Aug 19, 2026ReleaseUltragenyx Announces U.S. FDA Approval of GENGLYCOS™ Gene Therapy, the First-Ever FDA-Approved Treatment Designed to Treat the Underlying Cause of Glycogen Storage Disease Type Ia (GSDIa)BD extracted 1 program update from this document → RARE pipeline
• Aug 19, 20268-KCurrent report: other events
• Aug 11, 20268-KCurrent report: other events
• Aug 5, 202610-QQuarterly report
• Aug 4, 2026ReleaseUltragenyx Reports Second Quarter 2026 Financial Results and Corporate UpdateBD extracted 1 program update from this document → RARE pipeline
• Aug 4, 20268-KCurrent report: results of operations · EX-99.1
• Jul 30, 2026ReleasePowered by Purpose: Celebrating our Emerging Talent
• Jul 28, 2026ReleaseUltragenyx to Host Conference Call for Second Quarter 2026 Financial Results and Corporate Update
• Jul 8, 2026ReleaseUltragenyx Releases 2025 Impact Report Emphasizing Commitment to Rare Disease Patients, Innovation, and Global Impact
• Jun 3, 2026ReleaseUltragenyx to Participate at Goldman Sachs 47th Annual Global Healthcare Conference
• May 18, 20268-KCurrent report: officer / director change, shareholder vote
• May 6, 2026ReleaseUltragenyx to Participate at Bank of America’s 2026 Healthcare Conference
• May 6, 202610-QQuarterly report
• May 5, 20268-KCurrent report: results of operations · EX-99.1
• Apr 30, 2026DEFA14AProxy soliciting material
• Apr 2, 20268-KCurrent report: other events
• Mar 30, 20268-KCurrent report: other events
• Mar 27, 2026DEFA14AProxy soliciting material
• Mar 27, 2026DEF 14AProxy statement
• Mar 12, 20268-KCurrent report: other events