RARE Stock — Ultragenyx Pharmaceutical Inc.: Cash, EV, Pipeline

What they do

We are a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultra-rare genetic diseases. We have built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

Source: 10-K FY2025 via SEC EDGAR

Share price

$16.05

Market cap

$1,635.9M

Cash

$401.0M

Enterprise value

$2,420.9M

Balance sheet

Cash	$401.0M
Debt	$0.0M
Royalty-sale liability	$1,186.0M
Net cash	$401.0M
Enterprise value	$2,420.9M
Basic shares	101.9M shares
Fully diluted shares	123.0M shares
Fully diluted market cap	$1,974.0M
Cash per share	$3.93
Quarterly burn	$97.0M
Cash runway	4.1 quarters

Cash figures as of 2026-09-30. Database snapshot: 2026-11-13.

Royalty-sale liability as of 2026-09-30 (10-Q); included in enterprise value.

Share structure

Common

98.6M

Pre-funded warrants

0.0M

Options

18.2M

RSUs

—

Warrants

—

Converts

—

Conv preferred

0.0M

ESPP reserve

6.2M

FDSO

123.0M

Cap table as of 2026-10-29.

Price history

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Next catalyst

UX701 · Est. Nov 15, 2026 · quarter midpoint · guided Now

Recently reported

apazunersen Phase 3 Negative · 2026-09-02

Aspire · Angelman syndrome

Source release →

GTX-102 Phase 3 Missed primary · 2026-09-02

GTX-102 · angelman syndrome

Source release →

Upcoming catalysts

Expected	Drug	Phase	Indication	Mgmt guide
GUIDEDEst. Nov 15, 2026 Now quarter midpoint	UX701 Cyprus2+ pivotal study dose-finding stage	Not disclosed	Cyprus2+ pivotal dose-finding stage	Q4 2026
Feb 12 - Mar 26 in 13 wks	setrusumab	Phase 3	Osteogenesis Imperfecta	—
GUIDEDEst. Apr 1, 2027 in 7 wks half midpoint	DTX301 Enh3ance study second primary endpoint (treatment burden)	Not disclosed	Enh3ance second primary endpoint (treatment burden)	H1 2027
Sep 12 - Oct 24 in 10 mo	Adjuvant Immunomodulatory (IM) Therapy	Phase 3	Mucopolysaccharidosis IIIA; MPS IIIA; Sanfilippo Syndrome	—
Sep 12 - Oct 24 in 10 mo	Triheptanoin	Phase 3	Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)	—
Oct 13 - Nov 24 in 11 mo	Oral Corticosteroids	Phase 3	OTC Deficiency	—
Jan 12 - Feb 23 in 26 mo	UX016	Phase 2	GNE Myopathy	—
Mar 15 - Apr 26 in 28 mo	GTX-102	Phase 3	Angelman Syndrome	—
Apr 12 - May 24 in 29 mo	UX111	Phase 3	MPS IIIA; Sanfilippo Syndrome; Sanfilippo A	—
Apr 12 - May 24 in 29 mo	Standard of Care (SOC)	Phase 2	Wilson Disease	—
Feb 12 - Mar 26 in 40 mo	GTX-102	Phase 2	Angelman Syndrome	—

Estimated readout windows: registry primary-completion date + 6-12 weeks. Windows already open are shown from "Now". Drug names link to ClinicalTrials.gov.

Full readout calendar →

Pipeline assets

Crysvita

Approved · Active Modality: fully human monoclonal antibody

Target FGF23

Partnerships Kyowa Kirin (KKC): collaboration and license agreement (Aug 2013, as amended); KKC manufactures/supplies

Indications

X-linked hypophosphatemia (XLH), adults and pediatric 1+ (Approved)
Tumor-induced osteomalacia (TIO) (Approved)

Status note Targets FGF23; subcutaneous injection; approved in US, EU and certain other regions.

Evkeeza

Approved · Active Modality: fully human monoclonal antibody (IV)

Target ANGPTL3

Indications

Homozygous familial hypercholesterolemia (HoFH) (Approved)

Status note Binds and blocks ANGPTL3; approved product.

Dojolvi

Approved · Active

Indications

Japan (NHI listed; launched May 2026 under Conditional Approval System) (Approved)

Status note Listed on Japan National Health Insurance drug price list and officially launched May 2026 after manufacturing and marketing approval Mar 23, 2026 under the Conditional Approval System for Pharmaceuticals.

DTX401

Phase 3 · Active Modality: AAV8 gene therapy (one-time IV infusion)

Aliases pariglasgene brecaparvovec

Target G6Pase-α (G6PC)

Indications

Glycogen Storage Disease Type Ia (GSDIa) (Phase 3)

Catalysts

— August 23, 2026

Status note Designed to deliver stable expression and activity of G6Pase-α. BLA accepted for review Feb 2026 with Priority Review; FDA informed company (Apr 2026) an Advisory Committee meeting was planned.

UX111

Phase 3 · Active Modality: AAV9 gene therapy (one-time IV infusion)

Aliases ABO-102

Target SGSH (sulfamidase)

Partnerships Abeona Therapeutics (exclusive license agreement, announced May 2022)

Indications

Sanfilippo syndrome Type A (MPS IIIA) (Phase 3)

Catalysts

— September 19, 2026

Status note Resubmitted BLA accepted Apr 2026 after prior CRL.

UX701

Phase 3 · Active Modality: AAV9 gene therapy (single IV infusion)

Target ATP7B

Partnerships REGENXBIO Inc. (license agreement)

Indications

Wilson disease (pivotal Cyprus2+ study) (Phase 3)

Status note Delivers stable expression of truncated ATP7B copper transporter to improve copper distribution/excretion and reverse Wilson liver disease pathology. Ongoing pivotal Cyprus2+ study; Stage 1 dose-finding across four sequential dose cohorts (Cohort 4 enrollment of 5 patients completed Sep 2025).

DTX301

Phase 3 · Active Modality: AAV8 gene therapy (one-time IV infusion)

Target OTC

Indications

Ornithine transcarbamylase (OTC) deficiency (Enh3ance Phase 3) (Phase 3)

Status note Enh3ance study continues; second primary endpoint readout at 64 weeks of follow-up.

GTX-102

Phase 3 · Active Modality: antisense oligonucleotide (ASO; intrathecal)

Target UBE3A

Partnerships GeneTx Biotherapeutics LLC (license agreement)

Indications

Angelman syndrome (Aspire Phase 3) (Phase 3)
Angelman syndrome, other genotypes/ages (Aurora Phase 2/3) (Phase 2)

Catalysts

— H2 2026

Status note Inhibits expression of the paternal UBE3A antisense transcript. Aspire Phase 3 (129 patients aged 4-17 with full maternal UBE3A deletion, 48 weeks) fully enrolled Jul 2025. Aurora Phase 2/3 in other genotypes/ages: enrollment begun Oct 2025, expected complete H2 2026.

UX143

Phase 3 · Active Modality: fully human monoclonal antibody

Target sclerostin

Partnerships Mereo BioPharma 3 (Mereo): collaboration agreement

Indications

Osteogenesis imperfecta (OI) (Phase 3)

Status note Dec 2025: Phase 3 Orbit and Cosmic studies did not achieve their primary endpoints. Feb 2026: restructuring curtailed UX143 manufacturing. No discontinuation of development disclosed.

UX016

Preclinical · Active Modality: small molecule prodrug of sialic acid

Indications

GNE myopathy (Preclinical)

Status note Substrate replacement therapy for GNE myopathy; FDA cleared IND application Mar 2026; program is funded.

Per-asset detail extracted from the 10-Q filed 2026-08-05. Fields the filing does not state are marked "not disclosed" rather than filled from other sources.

Pipeline

Lead drug	Setrusumab
Lead indication	Osteogenesis Imperfecta
Lead phase	Phase 3
Active trials	18
Collaborator trials	5

Active clinical trials

Trial	Phase	Status	Role	Sponsor	BP	Condition(s)	Mgmt guide
NCT06636071	Phase 3	Active, not recruiting	Sponsor	RARE	—	Osteogenesis Imperfecta	—
NCT04360265	Phase 3	Enrolling by invitation	Sponsor	RARE	—	Mucopolysaccharidosis IIIA; MPS IIIA; Sanfilippo Syndrome	—
NCT05933200	Phase 3	Active, not recruiting	Sponsor	RARE	—	Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)	—
NCT05345171	Phase 3	Active, not recruiting	Sponsor	RARE	—	OTC Deficiency	H1 2027 Est. Apr 1, 2027 · half midpoint
NCT06415344	Phase 3	Active, not recruiting	Sponsor	RARE	—	Angelman Syndrome	—
NCT02716246	Phase 3	Recruiting	Sponsor	RARE	—	MPS IIIA; Sanfilippo Syndrome; Sanfilippo A	—
NCT05125809	Phase 3	Active, not recruiting	Sponsor	RARE	—	Osteogenesis Imperfecta	—
NCT05768854	Phase 3	Active, not recruiting	Sponsor	RARE	—	Osteogenesis Imperfecta	—
NCT06617429	Phase 3	Active, not recruiting	Sponsor	RARE	—	Angelman Syndrome	—
NCT07511556	Phase 2	Recruiting	Sponsor	RARE	—	GNE Myopathy	—
NCT04884815	Phase 2	Active, not recruiting	Sponsor	RARE	—	Wilson Disease	—
NCT07157254	Phase 2	Active, not recruiting	Sponsor	RARE	—	Angelman Syndrome	—

Sponsor-matched active trials from ClinicalTrials.gov (18 as sponsor, 5 as collaborator); "Sponsor" = company is the lead sponsor, "Collaborator" = company is a collaborator. The BP column flags big-pharma involvement as lead sponsor or collaborator (excluding the company itself). The Mgmt guide column shows what management guided for readout timing when a guided program matches the trial (hover for the quote, click for the source). Trial listings are not a company pipeline slide.

View all 23 active trials for Ultragenyx Pharmaceutical Inc on ClinicalTrials.gov

Specialist fund holders

Tracked specialist funds: ADAR1 Capital Management, Aisling Capital, Ally Bridge Group, Avoro Capital Advisors, BVF Partners, Baker Bros. Advisors, Boxer Capital, Braidwell LP, Casdin Capital, Cormorant Asset Management, DAFNA Capital, Deerfield Management, EcoR1 Capital, Frazier Life Sciences, Great Point Partners, Ikarian Capital, Logos Global Management, OrbiMed Advisors, Perceptive Advisors, RA Capital Management, RTW Investments, Sphera Biotech, Stonepine Capital, Tang Capital Partners, Vivo Capital.

Fund	Shares	Q/Q change	% of portfolio	Filing
RTW Investments	7,796,895	+4,148,100	2.22%	2026-08-14
Sphera Biotech	588,494	-220,839	2.81%	2026-08-14
Ikarian Capital	165,617	-316,954	0.91%	2026-08-14
DAFNA Capital	47,100	-14,000	0.30%	2026-08-14
Stonepine Capital	25,000	+25,000	0.64%	2026-08-17
ADAR1 Capital Management	0	-13,400	0.00%	2026-08-14

Specialist-fund positions from 13F filings. Q/Q change is vs the prior quarter's filing; % of portfolio is the fund's own reported portfolio weight.

Latest news

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RARE — Ultragenyx Pharmaceutical Inc. ☆

What they do

Balance sheet

Share structure

Price history

Next catalyst

Recently reported

Upcoming catalysts

Pipeline assets

Pipeline

Active clinical trials

Specialist fund holders

Latest news

RARE — Ultragenyx Pharmaceutical Inc.