{"filings":[{"id":494596,"accession_number":"0001193125-26-228639","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2026-05-18T18:15:14+00:00","items":["5.02","5.07","9.01"],"status":"ready","headline":"Ultragenyx shareholders OK 14.5M-share equity plan; re-elect directors","summary_kind":"llm","event_type":"other_material","confidence":"high","bullets":["Third Amended and Restated 2023 Incentive Plan approved; up to 14.5M new shares available plus shares from prior plans.","Emil Kakkis, Shehnaaz Suliman, Daniel G. Welch re-elected as Class I directors to serve until 2029 Annual Meeting.","Ernst & Young ratified as independent auditor; advisory 'say-on-pay' proposal passed with 70.9M votes for.","Director compensation limit set at $900k per year ($1.5M for first year or Chair/Lead Director)."],"consensus_eps_estimate":null,"consensus_eps_actual":null,"consensus_revenue_estimate":null,"consensus_revenue_actual":null,"consensus_period":null,"consensus_source":null,"sentiment":"neutral","materiality_score":0.6,"calibrated_materiality_score":0.6,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":93328,"accession_number":"0001193125-26-206368","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2026-05-05T23:59:59+00:00","items":["2.02","9.01"],"status":"ready","headline":"Ultragenyx Q1 revenue $136M, net loss $1.84/share; reaffirms 2026 guidance; GTX-102 Phase 3 data H2 2026","summary_kind":"llm","event_type":"earnings","confidence":"high","bullets":["Total revenue $136M (Q1 2025: $139M); Crysvita $93M, Dojolvi $18M, Evkeeza $18M, Mepsevii $7M.","Net loss $185M ($1.84/share) vs $151M ($1.57/share); operating expenses $305M including $30M restructuring.","Reaffirms 2026 total revenue guidance $730M-$760M; combined R&D and SG&A expenses flat to slightly down vs 2025.","DTX401 BLA for GSDIa granted Priority Review; PDUFA Aug 23, 2026. UX111 BLA resubmitted for MPS IIIA; PDUFA Sept 19, 2026.","GTX-102 Angelman Phase 3 data expected H2 2026; DTX301 Phase 3 showed significant ammonia reduction."],"consensus_eps_estimate":null,"consensus_eps_actual":-1.84,"consensus_revenue_estimate":null,"consensus_revenue_actual":136000000.0,"consensus_period":"2026-Q1","consensus_source":"xbrl_only","sentiment":"neutral","materiality_score":0.6,"calibrated_materiality_score":0.6,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":93327,"accession_number":"0001193125-26-139084","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2026-04-02T23:59:59+00:00","items":["8.01"],"status":"ready","headline":"FDA accepts resubmitted BLA for UX111 gene therapy; PDUFA date Sept 19, 2026","summary_kind":"llm","event_type":"regulatory","confidence":"high","bullets":["FDA accepted for review resubmitted BLA for UX111 (rebisufligene etisparvovec) AAV9 gene therapy.","UX111 is intended for Sanfilippo syndrome Type A (MPS IIIA) under accelerated approval pathway.","PDUFA action date set for September 19, 2026.","No new safety or efficacy data disclosed; review outcome remains uncertain."],"consensus_eps_estimate":null,"consensus_eps_actual":null,"consensus_revenue_estimate":null,"consensus_revenue_actual":null,"consensus_period":null,"consensus_source":null,"sentiment":"positive","materiality_score":0.65,"calibrated_materiality_score":0.65,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":104869,"accession_number":"0001193125-26-130498","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2026-03-30T23:59:59+00:00","items":["8.01"],"status":"ready","headline":"Ultragenyx receives FDA IND clearance for UX016 in GNE myopathy; Phase 1/2 to start H2 2026","summary_kind":"llm","event_type":"regulatory","confidence":"high","bullets":["FDA cleared IND for UX016, a prodrug of sialic acid for GNE myopathy, a rare inherited neuromuscular disorder.","UX016 is a small molecule prodrug designed to enhance sialic acid delivery to muscle cells.","Phase 1/2 study planned for H2 2026, enrolling ~24 adults aged 18–55 in the US; externally funded by a patient group.","Study will evaluate safety, pharmacokinetics, and efficacy including muscle strength and functional measures through 48 weeks."],"consensus_eps_estimate":null,"consensus_eps_actual":null,"consensus_revenue_estimate":null,"consensus_revenue_actual":null,"consensus_period":null,"consensus_source":null,"sentiment":"positive","materiality_score":0.55,"calibrated_materiality_score":0.55,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":104868,"accession_number":"0001193125-26-103167","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2026-03-12T23:59:59+00:00","items":["8.01"],"status":"ready","headline":"Ultragenyx DTX301 Phase 3 meets primary endpoint: 18% ammonia AUC reduction (p=0.018) in OTC deficiency","summary_kind":"llm","event_type":"other_material","confidence":"high","bullets":["DTX301-treated patients (n=18) showed 18% reduction in 24-hour plasma ammonia AUC vs placebo (n=19) at Week 36 (p=0.018).","8 of 9 patients with abnormal baseline ammonia reached normal levels; treated patients maintained normal ammonia despite 27% mean reduction in scavenger meds.","71% of treated patients reported much improvement on PGIC vs 0% for placebo at Week 24.","One SAE of acute hepatitis (resolved with steroids); 5 hyperammonemic crises in placebo (1 death) vs 1 in treated (no deaths).","Study continues to second primary endpoint on treatment burden; data expected H1 2027."],"consensus_eps_estimate":null,"consensus_eps_actual":null,"consensus_revenue_estimate":null,"consensus_revenue_actual":null,"consensus_period":null,"consensus_source":null,"sentiment":"positive","materiality_score":0.85,"calibrated_materiality_score":0.85,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":104867,"accession_number":"0001193125-26-062568","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2026-02-23T23:59:59+00:00","items":["8.01"],"status":"ready","headline":"FDA accepts Ultragenyx's BLA for DTX401 gene therapy for GSDIa with Priority Review, PDUFA Aug 23, 2026","summary_kind":"llm","event_type":"regulatory","confidence":"high","bullets":["FDA accepted Biologics License Application for DTX401 (pariglasgene brecaparvovec) to treat Glycogen Storage Disease Type Ia.","Granted Priority Review; Prescription Drug User Fee Act (PDUFA) action date is August 23, 2026.","DTX401 is an AAV-based gene therapy targeting a rare metabolic disorder with no approved therapies.","BLA acceptance triggers regulatory milestone; company may be eligible for priority review voucher if approved."],"consensus_eps_estimate":null,"consensus_eps_actual":null,"consensus_revenue_estimate":null,"consensus_revenue_actual":null,"consensus_period":null,"consensus_source":null,"sentiment":"positive","materiality_score":0.75,"calibrated_materiality_score":0.75,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":104866,"accession_number":"0001193125-26-048738","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2026-02-12T23:59:59+00:00","items":["2.02","2.05","9.01"],"status":"ready","headline":"Ultragenyx Q4 rev $207M (+25%), net loss $129M; restructures 10% headcount; UX111 gets IRL","summary_kind":"llm","event_type":"earnings","confidence":"high","bullets":["Full year 2025 total revenue $673M (+20% YoY); Crysvita $481M, Dojolvi $96M, Evkeeza $59M.","Q4 net loss $129M ($1.29/share); full year net loss $575M ($5.83/share).","Restructuring: 10% workforce cut (~130 employees), $50M charges; path to profitability in 2027.","2026 revenue guidance $730-760M; combined R&D+SG&A expenses flat to down low-single digits.","UX111 BLA resubmitted; received Incomplete Response Letter requesting additional CMC documentation."],"consensus_eps_estimate":null,"consensus_eps_actual":-5.83,"consensus_revenue_estimate":null,"consensus_revenue_actual":673000000.0,"consensus_period":"2025-FY","consensus_source":"xbrl_only","sentiment":"negative","materiality_score":0.85,"calibrated_materiality_score":0.85,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":104865,"accession_number":"0001193125-26-034474","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2026-02-03T23:59:59+00:00","items":["8.01"],"status":"ready","headline":"Ultragenyx UX111 gene therapy shows significant cognitive benefit in MPS IIIA; BLA resubmitted with PDUFA Q3 2026","summary_kind":"llm","event_type":"other_material","confidence":"high","bullets":["Children under 2 treated with UX111 showed +23.2 point improvement in Bayley-III cognitive raw score vs natural history (p<0.0001).","81.5% of overall efficacy set achieved ≥50% reduction in CSF heparan sulfate; median reduction 63.98% (p<0.001).","Functional retention observed: all older children retained communication; 9/10 retained ambulation and self-feeding.","UX111 well-tolerated with median follow-up 4.8 years; no TMA, myocarditis, DRG toxicity, or malignancy.","BLA resubmitted to FDA seeking accelerated approval; PDUFA date expected in Q3 2026."],"consensus_eps_estimate":null,"consensus_eps_actual":null,"consensus_revenue_estimate":null,"consensus_revenue_actual":null,"consensus_period":null,"consensus_source":null,"sentiment":"positive","materiality_score":0.85,"calibrated_materiality_score":0.85,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":104864,"accession_number":"0001193125-26-030590","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2026-01-30T23:59:59+00:00","items":["8.01"],"status":"ready","headline":"Ultragenyx resubmits UX111 BLA for Sanfilippo type A to FDA; PDUFA expected Q3 2026","summary_kind":"llm","event_type":"regulatory","confidence":"high","bullets":["Resubmitted BLA for UX111 gene therapy for Sanfilippo type A on Jan 30, 2026.","Submission addresses CMC observations from July 2025 CRL and includes additional year of clinical follow-up data.","FDA previously acknowledged robust neurodevelopmental data; updated data show durable treatment effect and acceptable safety.","PDUFA action date expected within a month; six-month review period suggests PDUFA in Q3 2026.","If approved, UX111 would be the first approved therapy for Sanfilippo syndrome type A."],"consensus_eps_estimate":null,"consensus_eps_actual":null,"consensus_revenue_estimate":null,"consensus_revenue_actual":null,"consensus_period":null,"consensus_source":null,"sentiment":"positive","materiality_score":0.8,"calibrated_materiality_score":0.8,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":104863,"accession_number":"0001193125-26-009791","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2026-01-12T23:59:59+00:00","items":["2.02","9.01"],"status":"ready","headline":"Ultragenyx prelim 2025 revenue $672-674M beats guidance; cash $735M; two gene therapy approvals possible in 2026","summary_kind":"llm","event_type":"earnings","confidence":"high","bullets":["Preliminary FY2025 total revenue $672-674M, ~20% YoY, exceeding top end of guidance.","Crysvita revenue $480-482M (17% YoY, above guidance); Dojolvi $95-97M (9% YoY, at guidance midpoint).","Cash and investments ~$735M at year-end 2025; full Q4/FY results and 2026 guidance due Feb 2026.","2026 catalysts: DTX401 (GSDIa) PDUFA Q3; UX111 (MPS IIIA) BLA resubmission early 2026; GTX-102 (Angelman) Phase 3 data H2 2026.","UX701 (Wilson disease) pivotal data H1 2026; UX143 (osteogenesis imperfecta) data presented at JPM conference."],"consensus_eps_estimate":null,"consensus_eps_actual":-5.83,"consensus_revenue_estimate":null,"consensus_revenue_actual":673000000.0,"consensus_period":"2025-FY","consensus_source":"xbrl_only","sentiment":"positive","materiality_score":0.75,"calibrated_materiality_score":0.75,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":121127,"accession_number":"0001193125-25-337099","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2025-12-30T23:59:59+00:00","items":["8.01"],"status":"ready","headline":"Ultragenyx completes rolling BLA submission for DTX401 gene therapy to FDA for GSDIa","summary_kind":"llm","event_type":"regulatory","confidence":"high","bullets":["BLA submission for DTX401 (pariglasgene brecaparvovec) completed for glycogen storage disease type Ia; based on 52 treated patients with up to 6 years follow-up.","Phase 3 GlucoGene study showed significant reductions in daily cornstarch intake while maintaining euglycemia and improved quality of life.","Rolling review began with non-clinical/clinical modules in August 2025; CMC module now submitted to complete the package.","DTX401 was well tolerated with an acceptable safety profile per the press release."],"consensus_eps_estimate":null,"consensus_eps_actual":null,"consensus_revenue_estimate":null,"consensus_revenue_actual":null,"consensus_period":null,"consensus_source":null,"sentiment":"positive","materiality_score":0.85,"calibrated_materiality_score":0.85,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":121126,"accession_number":"0001193125-25-332422","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2025-12-29T23:59:59+00:00","items":["8.01"],"status":"ready","headline":"Ultragenyx's setrusumab fails Phase 3 primary endpoints in OI; expense cuts planned","summary_kind":"llm","event_type":"other_material","confidence":"high","bullets":["Orbit and Cosmic studies did not meet primary endpoint of reducing annualized clinical fracture rate versus comparators.","Both studies achieved secondary endpoint of significant improvement in bone mineral density (BMD).","Company plans to implement significant expense reductions; evaluating next steps for the program.","Safety profile unchanged; no new safety signals observed."],"consensus_eps_estimate":null,"consensus_eps_actual":null,"consensus_revenue_estimate":null,"consensus_revenue_actual":null,"consensus_period":null,"consensus_source":null,"sentiment":"negative","materiality_score":0.85,"calibrated_materiality_score":0.85,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":121125,"accession_number":"0001193125-25-264707","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2025-11-04T23:59:59+00:00","items":["1.01","2.02","9.01"],"status":"ready","headline":"Ultragenyx Q3 revenue $160M (+15% YoY); sells 25% Crysvita royalty for $400M","summary_kind":"llm","event_type":"earnings","confidence":"high","bullets":["Total revenue $160M (+15% YoY); Crysvita $112M, Dojolvi $24M, Evkeeza $17M.","Net loss $180M ($1.81/share) vs $134M ($1.40) in Q3 2024; operating expense $331M.","Received $400M from OMERS for additional 25% Crysvita royalty; repurchase option at $540M.","FY2025 guidance reaffirmed: total rev $640-670M, Crysvita $460-480M, Dojolvi $90-100M.","UX143 OI Phase 3 data expected year-end; DTX401 BLA submission on track for Q4 2025; UX111 BLA resubmission early 2026."],"consensus_eps_estimate":null,"consensus_eps_actual":-4.55,"consensus_revenue_estimate":null,"consensus_revenue_actual":465721000.0,"consensus_period":"2025-Q3","consensus_source":"xbrl_only","sentiment":"neutral","materiality_score":0.85,"calibrated_materiality_score":0.85,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":121124,"accession_number":"0001193125-25-257538","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2025-10-30T23:59:59+00:00","items":["8.01"],"status":"ready","headline":"Ultragenyx doses first patient in Aurora study of GTX-102 for Angelman; Aspire Phase 3 fully enrolled","summary_kind":"llm","event_type":"other_material","confidence":"high","bullets":["First patient dosed in global Aurora study (NCT07157254) evaluating GTX-102 (apazunersen) for Angelman syndrome across all genotypes and ages 1 to <65.","Aurora will enroll ~60 participants in four cohorts based on age and genotype; primary endpoints include Bayley-4 and MDRI.","Phase 3 Aspire study fully enrolled with 129 participants (ages 4-17, UBE3A deletion), data expected H2 2026.","Aurora expands patient population beyond deletion-type to include UPD, ICD, and mutation genotypes.","Cohorts A, B, C single-arm; Cohort D randomized 2:1 GTX-102 vs no treatment with crossover at Week 24."],"consensus_eps_estimate":null,"consensus_eps_actual":null,"consensus_revenue_estimate":null,"consensus_revenue_actual":null,"consensus_period":null,"consensus_source":null,"sentiment":"neutral","materiality_score":0.65,"calibrated_materiality_score":0.65,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":137772,"accession_number":"0000950170-25-113553","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2025-09-08T23:59:59+00:00","items":["8.01"],"status":"ready","headline":"Ultragenyx reports positive longer-term Phase 3 data for DTX401 gene therapy in GSDIa","summary_kind":"llm","event_type":"other_material","confidence":"high","bullets":["DTX401 met primary endpoint at Week 48: mean cornstarch reduction 41% vs 10% placebo (p<0.0001).","At Week 96, DTX401 and crossover groups each achieved mean 61% reduction in daily cornstarch from baseline.","Two-thirds of participants eliminated at least one nighttime cornstarch dose; fasting tolerance improved through Year 2.","83% of DTX401 group and 95% of crossover reported improved disease burden at Week 96 per PGIC.","Open-label cohort (n=3) achieved 95% mean cornstarch reduction at Week 24; all discontinued cornstarch by Week 36."],"consensus_eps_estimate":null,"consensus_eps_actual":null,"consensus_revenue_estimate":null,"consensus_revenue_actual":null,"consensus_period":null,"consensus_source":null,"sentiment":"positive","materiality_score":0.8,"calibrated_materiality_score":0.8,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":137771,"accession_number":"0000950170-25-109570","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2025-08-18T23:59:59+00:00","items":["8.01"],"status":"ready","headline":"Ultragenyx starts rolling BLA submission for DTX401 gene therapy in GSDIa","summary_kind":"llm","event_type":"other_material","confidence":"high","bullets":["Initiated rolling BLA submission to FDA for DTX401 AAV gene therapy for Glycogen Storage Disease Type Ia.","Submitted non-clinical and clinical modules; plans to complete full BLA including CMC module in Q4 2025.","BLA includes 96-week Phase 3 data showing ~60-64% reduction in daily cornstarch use from baseline.","Updates address FDA observations from prior UX111 complete response letter related to CMC and manufacturing."],"consensus_eps_estimate":null,"consensus_eps_actual":null,"consensus_revenue_estimate":null,"consensus_revenue_actual":null,"consensus_period":null,"consensus_source":null,"sentiment":"positive","materiality_score":0.7,"calibrated_materiality_score":0.7,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":137770,"accession_number":"0000950170-25-103026","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2025-08-05T23:59:59+00:00","items":["2.02","9.01"],"status":"ready","headline":"Ultragenyx Q2 revenue $166M (+13% YoY); net loss narrows; UX111 BLA receives CRL","summary_kind":"llm","event_type":"earnings","confidence":"high","bullets":["Q2 total revenue $166M, Crysvita $120M, Dojolvi $23M; net loss $115M ($1.17/sh) vs $132M loss prior year.","Reaffirms 2025 guidance: total rev $640-670M, Crysvita $460-480M, Dojolvi $90-100M; path to profitability in 2027.","UX111 gene therapy for Sanfilippo type A received FDA CRL for CMC/manufacturing issues; clinical data package no issues; resubmission after Type A meeting.","GTX-102 for Angelman syndrome granted Breakthrough Therapy; Phase 3 Aspire fully enrolled (129 pts); data expected H2 2026.","UX143 Phase 3 final analysis expected end of 2025; DTX401 BLA submission planned Q4 2025; cash $539M as of June 30."],"consensus_eps_estimate":null,"consensus_eps_actual":-2.73,"consensus_revenue_estimate":null,"consensus_revenue_actual":305788000.0,"consensus_period":"2025-Q2","consensus_source":"xbrl_only","sentiment":"negative","materiality_score":0.7,"calibrated_materiality_score":0.7,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":137769,"accession_number":"0000950170-25-100918","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2025-07-31T23:59:59+00:00","items":["8.01"],"status":"ready","headline":"Ultragenyx fully enrolls Phase 3 Aspire study (129 patients) for GTX-102 in Angelman Syndrome","summary_kind":"llm","event_type":"other_material","confidence":"high","bullets":["Phase 3 Aspire study fully enrolled with ~129 patients ages 4-17 with maternal UBE3A deletion.","Participants randomized 1:1 to GTX-102 intrathecal injection or sham comparator for 48 weeks; sham can cross over after Week 48.","Primary endpoint: Bayley-4 cognitive raw score improvement; key secondary: MDRI across five domains.","Study completion expected H2 2026; company plans to move to topline data and regulatory submission."],"consensus_eps_estimate":null,"consensus_eps_actual":null,"consensus_revenue_estimate":null,"consensus_revenue_actual":null,"consensus_period":null,"consensus_source":null,"sentiment":"neutral","materiality_score":0.65,"calibrated_materiality_score":0.65,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":137768,"accession_number":"0000950170-25-095334","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2025-07-11T23:59:59+00:00","items":["8.01"],"status":"ready","headline":"FDA issues Complete Response Letter for Ultragenyx UX111 BLA; resubmission planned with 6-month review","summary_kind":"llm","event_type":"regulatory","confidence":"high","bullets":["FDA CRL received July 11, 2025, for UX111 (ABO-102) AAV gene therapy for Sanfilippo syndrome type A.","CRL requests additional CMC documentation and addresses observations from manufacturing facility inspections.","Company states observations are readily addressable and unrelated to product quality; clinical data package not cited.","FDA acknowledged robust neurodevelopmental outcome data and supportive biomarker evidence; updated clinical data requested.","Ultragenyx plans to resubmit BLA within months, expecting up to 6-month FDA review period."],"consensus_eps_estimate":null,"consensus_eps_actual":null,"consensus_revenue_estimate":null,"consensus_revenue_actual":null,"consensus_period":null,"consensus_source":null,"sentiment":"negative","materiality_score":0.75,"calibrated_materiality_score":0.75,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":137767,"accession_number":"0000950170-25-094806","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2025-07-09T23:59:59+00:00","items":["8.01"],"status":"ready","headline":"Ultragenyx's UX143 Phase 3 Orbit passes DMC safety review; final analysis year-end 2025","summary_kind":"llm","event_type":"other_material","confidence":"high","bullets":["DMC confirms acceptable safety profile for UX143 in pediatric/young adult OI patients; study continues to final analysis.","Final analysis of Phase 3 Orbit trial expected around end of 2025, with statistical threshold p<0.04.","Safety in younger patients consistent with prior studies; no data from Cosmic study analyzed at interim.","Patients continue dosing; final analyses after at least 18 months therapy."],"consensus_eps_estimate":null,"consensus_eps_actual":null,"consensus_revenue_estimate":null,"consensus_revenue_actual":null,"consensus_period":null,"consensus_source":null,"sentiment":"positive","materiality_score":0.6,"calibrated_materiality_score":0.6,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":155156,"accession_number":"0000950170-25-090616","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2025-06-27T23:59:59+00:00","items":["8.01"],"status":"ready","headline":"Ultragenyx receives FDA Breakthrough Therapy Designation for GTX-102 in Angelman syndrome","summary_kind":"llm","event_type":"regulatory","confidence":"high","bullets":["FDA granted Breakthrough Therapy Designation for GTX-102 (apazunersen) for Angelman syndrome.","Based on Phase 1/2 study in 74 patients (4-17 years) with full maternal UBE3A deletion showing consistent developmental gains.","Designation aims to expedite development and review; may enable rolling submission and enhanced FDA guidance.","Positive data shows rapid, sustained improvements across multiple symptom domains over up to three years."],"consensus_eps_estimate":null,"consensus_eps_actual":null,"consensus_revenue_estimate":null,"consensus_revenue_actual":null,"consensus_period":null,"consensus_source":null,"sentiment":"positive","materiality_score":0.75,"calibrated_materiality_score":0.75,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":155155,"accession_number":"0000950170-25-074529","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2025-05-19T23:59:59+00:00","items":["5.02","5.07","9.01"],"status":"ready","headline":"Ultragenyx shareholders approve amended incentive plan, elect directors, ratify auditor","summary_kind":"llm","event_type":"other_material","confidence":"high","bullets":["Stockholders approved Second Amended and Restated 2023 Incentive Plan, authorizing up to 11.5M new shares plus forfeited shares from prior plans.","Class III directors Matthew K. Fust and Amrit Ray, M.D. elected to Board for term expiring 2028.","Advisory vote on executive compensation received 55.1M for (68%) vs 26.1M against.","Ernst & Young ratified as fiscal 2025 independent auditor with 84.3M votes for.","Broker non-votes of 5.3M shares on director election, incentive plan, and say-on-pay proposals."],"consensus_eps_estimate":null,"consensus_eps_actual":null,"consensus_revenue_estimate":null,"consensus_revenue_actual":null,"consensus_period":null,"consensus_source":null,"sentiment":"neutral","materiality_score":0.55,"calibrated_materiality_score":0.55,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":155154,"accession_number":"0000950170-25-064473","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2025-05-06T23:59:59+00:00","items":["2.02","9.01"],"status":"ready","headline":"Ultragenyx Q1 revenue $139M, reaffirms FY2025 guidance; UX111 PDUFA Aug 18, 2025","summary_kind":"llm","event_type":"earnings","confidence":"high","bullets":["Q1 total revenue $139M (+28% YoY); net loss $151M ($1.57/share) vs $171M ($2.03) in Q1 2024.","Crysvita revenue $103M (+25% YoY); Dojolvi $17M; Evkeeza $11M; Mepsevii $8.4M.","Reaffirmed FY2025 guidance: total revenue $640-670M, Crysvita $460-480M, Dojolvi $90-100M.","UX111 BLA for Sanfilippo granted Priority Review; PDUFA date Aug 18, 2025; potential H2 launch.","DTX401 BLA for GSDIa expected mid-2025; GTX-102 Ph3 enrollment completion H2 2025; cash $563M."],"consensus_eps_estimate":null,"consensus_eps_actual":-1.57,"consensus_revenue_estimate":null,"consensus_revenue_actual":139292000.0,"consensus_period":"2025-Q1","consensus_source":"xbrl_only","sentiment":"neutral","materiality_score":0.6,"calibrated_materiality_score":0.6,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":173670,"accession_number":"0000950170-25-019854","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2025-02-13T23:59:59+00:00","items":["2.02","9.01"],"status":"ready","headline":"Ultragenyx Q4 revenue $165M, FY $560M; guides 2025 revenue $640-670M","summary_kind":"llm","event_type":"earnings","confidence":"high","bullets":["Q4 2024 total revenue $165M (+29% YoY); FY 2024 total revenue $560M (+29% YoY).","Crysvita FY revenue $410M (+25% YoY); Dojolvi FY revenue $88M (+25% YoY).","FY 2024 net loss $569M ($6.29/diluted share) vs $607M ($8.25) in 2023; cash used ops $414M.","2025 guidance: total revenue $640-670M, Crysvita $460-480M, Dojolvi $90-100M; lower cash ops expected.","PDUFA decision for UX111 gene therapy (Sanfilippo syndrome) expected H2 2025; BLA filing for DTX401 (GSDIa) mid-2025."],"consensus_eps_estimate":null,"consensus_eps_actual":-6.29,"consensus_revenue_estimate":null,"consensus_revenue_actual":560230000.0,"consensus_period":"2024-FY","consensus_source":"xbrl_only","sentiment":"neutral","materiality_score":0.75,"calibrated_materiality_score":0.75,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":173669,"accession_number":"0000950170-25-004587","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2025-01-13T23:59:59+00:00","items":["2.02","9.01"],"status":"ready","headline":"Ultragenyx preliminary 2024 revenue $555-560M above guidance; 2025 guide $640-670M","summary_kind":"llm","event_type":"earnings","confidence":"high","bullets":["Preliminary 2024 total revenue $555-560M, exceeding top end of guidance, ~29% YoY growth.","Crysvita revenue $405-410M (+24% YoY); Dojolvi revenue $87-89M (+25% YoY).","2025 total revenue guidance $640-670M; cash and investments ~$745M as of Dec 31, 2024.","UX143 Phase 3 OI study second interim analysis mid-2025; GTX-102 Phase 3 Angelman enrollment completion H2 2025.","UX111 BLA submitted (PDUFA H2 2025); DTX401 BLA filing mid-2025; DTX301 Phase 3 enrollment early 2025."],"consensus_eps_estimate":null,"consensus_eps_actual":-6.29,"consensus_revenue_estimate":null,"consensus_revenue_actual":560230000.0,"consensus_period":"2024-FY","consensus_source":"xbrl_only","sentiment":"positive","materiality_score":0.8,"calibrated_materiality_score":0.8,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":190632,"accession_number":"0000950170-24-138467","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2024-12-19T23:59:59+00:00","items":["8.01"],"status":"ready","headline":"First patient dosed in Ultragenyx Phase 3 Aspire study of GTX-102 for Angelman syndrome","summary_kind":"llm","event_type":"other_material","confidence":"high","bullets":["Global Phase 3 Aspire study will enroll ~120 children ages 4-17 with full maternal UBE3A deletion.","Patients randomized 1:1 to GTX-102 intrathecal injections or sham comparator for 48-week primary analysis.","Primary endpoint: cognition improvement (Bayley-4 cognitive raw score); key secondary: Multi-domain Responder Index.","Active group receives three monthly 8 mg loading doses then quarterly maintenance up to 14 mg; sham crossover after Week 48."],"consensus_eps_estimate":null,"consensus_eps_actual":null,"consensus_revenue_estimate":null,"consensus_revenue_actual":null,"consensus_period":null,"consensus_source":null,"sentiment":"neutral","materiality_score":0.75,"calibrated_materiality_score":0.75,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":190631,"accession_number":"0000950170-24-138250","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2024-12-19T23:59:59+00:00","items":["8.01"],"status":"ready","headline":"Ultragenyx submits BLA to FDA for UX111 gene therapy in Sanfilippo syndrome type A","summary_kind":"llm","event_type":"regulatory","confidence":"high","bullets":["Submitted BLA to FDA seeking accelerated approval for UX111 (AAV gene therapy) in Sanfilippo syndrome type A (MPS IIIA).","BLA supported by Transpher A study: rapid, sustained CSF heparan sulfate reduction correlated with improved cognitive development vs. natural history.","Most common adverse events were liver enzyme elevations; majority mild/moderate (Grade 1/2), all resolved.","In 2024, FDA agreed CSF heparan sulfate can serve as surrogate endpoint for accelerated approval."],"consensus_eps_estimate":null,"consensus_eps_actual":null,"consensus_revenue_estimate":null,"consensus_revenue_actual":null,"consensus_period":null,"consensus_source":null,"sentiment":"positive","materiality_score":0.7,"calibrated_materiality_score":0.7,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":190630,"accession_number":"0000950170-24-124458","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2024-11-12T23:59:59+00:00","items":["8.01"],"status":"ready","headline":"Ultragenyx reports positive Phase 1/2 data for GTX-102 in Angelman syndrome; Phase 3 Aspire study well powered","summary_kind":"llm","event_type":"other_material","confidence":"high","bullets":["Week 48 Bayley-4 Cognition GSV mean change +6.7 vs minimally important difference +5 (n=40).","Phase 3 primary endpoint Bayley-4 Raw score mean change +10.9 from baseline.","Study >95% powered to detect treatment effect even with up to 3x higher sham response.","MDRI net response +2.0 (p<0.0001); 80% of patients achieved net improvement in at least one domain.","Phase 3 Aspire will enroll ~120 patients with full maternal UBE3A deletion; 48-week primary analysis."],"consensus_eps_estimate":null,"consensus_eps_actual":null,"consensus_revenue_estimate":null,"consensus_revenue_actual":null,"consensus_period":null,"consensus_source":null,"sentiment":"positive","materiality_score":0.85,"calibrated_materiality_score":0.85,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":190629,"accession_number":"0000950170-24-121459","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2024-11-05T23:59:59+00:00","items":["2.02","9.01"],"status":"ready","headline":"Ultragenyx Q3 revenue $139M (+42% YoY); Breakthrough Therapy for setrusumab; DTX401 crossover 62% cornstarch reduction","summary_kind":"llm","event_type":"earnings","confidence":"high","bullets":["Total revenue $139M, up 42% YoY; Crysvita $98M (+31%), Dojolvi $21M (+29%), Evkeeza $11M.","Net loss $134M ($1.40/share) vs $160M ($2.23) YoY; operating cash burn $67M in Q3.","FY2024 guidance reaffirmed: total revenue $530-550M, Crysvita upper end $375-400M, Dojolvi $75-80M.","Setrusumab (UX143) granted FDA Breakthrough Therapy for osteogenesis imperfecta based on 67% fracture reduction.","DTX401 Phase 3 crossover patients showed 62% mean cornstarch reduction at week 30; BLA submission planned mid-2025."],"consensus_eps_estimate":null,"consensus_eps_actual":-4.91,"consensus_revenue_estimate":null,"consensus_revenue_actual":395353000.0,"consensus_period":"2024-Q3","consensus_source":"xbrl_only","sentiment":"positive","materiality_score":0.7,"calibrated_materiality_score":0.7,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":190628,"accession_number":"0000950170-24-113085","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2024-10-07T23:59:59+00:00","items":["8.01"],"status":"ready","headline":"Ultragenyx gets FDA Breakthrough Therapy Designation for setrusumab in osteogenesis imperfecta","summary_kind":"llm","event_type":"regulatory","confidence":"high","bullets":["Received FDA Breakthrough Therapy Designation for setrusumab (UX143) for OI Type I, III, IV in patients ≥2 years.","Designation based on Phase 2 data from Orbit (14-month) and Asteroid studies showing significant fracture rate reduction.","Breakthrough Therapy aims to expedite development and review for serious diseases with preliminary clinical evidence.","Ultragenyx collaborates with Mereo BioPharma on UX143 development.","Designation does not guarantee approval but may speed regulatory process."],"consensus_eps_estimate":null,"consensus_eps_actual":null,"consensus_revenue_estimate":null,"consensus_revenue_actual":null,"consensus_period":null,"consensus_source":null,"sentiment":"positive","materiality_score":0.75,"calibrated_materiality_score":0.75,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":190627,"accession_number":"0000950170-24-112402","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2024-10-03T23:59:59+00:00","items":["8.01"],"status":"ready","headline":"UX701 gene therapy shows clinical activity; 6 of 15 Wilson disease patients tapered off standard-of-care","summary_kind":"llm","event_type":"other_material","confidence":"high","bullets":["15 patients enrolled across 3 dose cohorts; 6 completely tapered off chelators/zinc therapy; 1 additional began tapering.","Non-ceruloplasmin bound copper stabilized to normal levels; ceruloplasmin-copper activity increased, indicating improved ATP7b function.","UX701 well tolerated with no unexpected treatment-emergent adverse events or significant immunologic safety events.","Company plans to enroll a fourth cohort at moderately increased dose with optimized immunomodulation regimen to enhance efficacy.","Dose selection for randomized placebo-controlled Stage 2 and long-term follow-up in Stage 3 are planned."],"consensus_eps_estimate":null,"consensus_eps_actual":null,"consensus_revenue_estimate":null,"consensus_revenue_actual":null,"consensus_period":null,"consensus_source":null,"sentiment":"positive","materiality_score":0.75,"calibrated_materiality_score":0.75,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":207123,"accession_number":"0000950170-24-089263","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2024-08-01T23:59:59+00:00","items":["2.02","9.01"],"status":"ready","headline":"Ultragenyx Q2 revenue $147M (+36% YoY), raises 2024 guidance; key FDA agreements advance pipeline","summary_kind":"llm","event_type":"earnings","confidence":"high","bullets":["Q2 total revenue $147M (Crysvita $114M, Dojolvi $19M); net loss narrows to $132M from $160M.","2024 revenue guidance raised to $530-550M (prev $500-530M); Crysvita expected towards upper end of $375-400M.","Cash $874M after June equity offering of $381M net; 2024 operating cash use guided below $400M.","FDA agrees CSF heparan sulfate as surrogate endpoint for UX111 Sanfilippo accelerated approval; BLA filing late-2024/early-2025.","Positive Phase 3 data for DTX401 in GSDIa (41.3% reduction in cornstarch, p<0.0001); Phase 2/3 OI setrusumab shows 67% fracture reduction."],"consensus_eps_estimate":null,"consensus_eps_actual":-3.54,"consensus_revenue_estimate":null,"consensus_revenue_actual":255859000.0,"consensus_period":"2024-Q2","consensus_source":"xbrl_only","sentiment":"positive","materiality_score":0.75,"calibrated_materiality_score":0.75,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":207122,"accession_number":"0000950170-24-084254","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2024-07-17T23:59:59+00:00","items":["8.01"],"status":"ready","headline":"Ultragenyx aligns with FDA on Phase 3 design for GTX-102 in Angelman syndrome","summary_kind":"llm","event_type":"regulatory","confidence":"high","bullets":["FDA alignment on EoP2 meeting for GTX-102; Phase 3 trial to enroll ~120 UBE3A deletion patients.","Primary endpoint: Bayley-4 cognitive raw score; 48-week double-blind, sham-controlled design.","Patients in control arm eligible to roll over to treatment after double-blind period.","EMA accepted Phase 3 design under PRIME; PMDA meeting expected in coming weeks.","Plans for open-label study to evaluate GTX-102 in other Angelman genotypes and age groups."],"consensus_eps_estimate":null,"consensus_eps_actual":null,"consensus_revenue_estimate":null,"consensus_revenue_actual":null,"consensus_period":null,"consensus_source":null,"sentiment":"positive","materiality_score":0.7,"calibrated_materiality_score":0.7,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":224478,"accession_number":"0000950170-24-076222","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2024-06-21T23:59:59+00:00","items":["5.02","5.07","9.01"],"status":"ready","headline":"Ultragenyx stockholders approve amended 2023 incentive plan and elect directors at annual meeting","summary_kind":"llm","event_type":"other_material","confidence":"high","bullets":["Class II directors Dunsire, Narachi, and Sanders elected with over 66M votes each for terms until 2027.","Amended 2023 Incentive Plan approved: 51.9M for, 22.3M against; adds 8.5M shares plus prior plan rollover shares.","Ernst & Young ratified as independent auditor for FY2024 with 77.2M votes for (98.2% of votes cast).","Advisory vote on executive compensation passed with 54.8M for vs 19.3M against; 4.3M broker non-votes."],"consensus_eps_estimate":null,"consensus_eps_actual":null,"consensus_revenue_estimate":null,"consensus_revenue_actual":null,"consensus_period":null,"consensus_source":null,"sentiment":"neutral","materiality_score":0.3,"calibrated_materiality_score":0.3,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":224477,"accession_number":"0001193125-24-162003","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2024-06-17T23:59:59+00:00","items":["1.01","9.01"],"status":"ready","headline":"Ultragenyx raises ~$330.7M in public offering of common stock and pre-funded warrants","summary_kind":"llm","event_type":"other_material","confidence":"high","bullets":["Offering of 7,435,898 shares at $39/share plus pre-funded warrants for 1,538,501 shares at $38.999.","Net proceeds expected ~$330.7M after underwriting discounts and expenses.","Underwriters granted 30-day option to purchase up to 1,346,153 additional shares.","Closing expected June 17, 2024; use of proceeds for general corporate purposes."],"consensus_eps_estimate":null,"consensus_eps_actual":null,"consensus_revenue_estimate":null,"consensus_revenue_actual":null,"consensus_period":null,"consensus_source":null,"sentiment":"neutral","materiality_score":0.7,"calibrated_materiality_score":0.7,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":224476,"accession_number":"0000950170-24-072414","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2024-06-12T23:59:59+00:00","items":["8.01"],"status":"ready","headline":"FDA agrees CSF heparan sulfate as surrogate endpoint for UX111 accelerated approval BLA in MPS IIIA","summary_kind":"llm","event_type":"regulatory","confidence":"high","bullets":["FDA agreed cerebral spinal fluid heparan sulfate is a reasonable surrogate endpoint supporting accelerated approval for UX111 gene therapy in Sanfilippo syndrome type A (MPS IIIA).","Company intends to file BLA late 2024 or early 2025 after a pre-BLA meeting with the FDA.","BLA filing will rely on data from the ongoing pivotal Transpher A study and long-term follow-up; results were presented at WORLDSymposium."],"consensus_eps_estimate":null,"consensus_eps_actual":null,"consensus_revenue_estimate":null,"consensus_revenue_actual":null,"consensus_period":null,"consensus_source":null,"sentiment":"positive","materiality_score":0.75,"calibrated_materiality_score":0.75,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":224475,"accession_number":"0000950170-24-072119","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2024-06-12T23:59:59+00:00","items":["8.01"],"status":"ready","headline":"Ultragenyx and Mereo report setrusumab cuts fracture rate 67% and boosts BMD 22% in OI Phase 2","summary_kind":"llm","event_type":"other_material","confidence":"high","bullets":["Median annualized fracture rate reduced 67% from 0.72 to 0.00 (p=0.0014) over mean 16 months in 24 patients.","Lumbar spine BMD increased 22% from baseline at month 12 (p<0.0001); Z-score normalized from -1.73 to -0.49.","No treatment-related serious adverse events or hypersensitivity reactions; most common AEs: infusion-related events, headache.","Phase 3 portion of Orbit study enrolled 158 patients; Cosmic study enrolled 69 patients; 14-month data to be presented at future meeting."],"consensus_eps_estimate":null,"consensus_eps_actual":null,"consensus_revenue_estimate":null,"consensus_revenue_actual":null,"consensus_period":null,"consensus_source":null,"sentiment":"positive","materiality_score":0.75,"calibrated_materiality_score":0.75,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":224474,"accession_number":"0000950170-24-066553","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2024-05-30T23:59:59+00:00","items":["8.01","9.01"],"status":"ready","headline":"Ultragenyx Phase 3 DTX401 gene therapy meets primary endpoint in GSDIa","summary_kind":"llm","event_type":"other_material","confidence":"high","bullets":["DTX401 reduced daily cornstarch intake by 41.3% vs 10.3% for placebo at Week 48 (p<0.0001).","68% of DTX401 patients achieved ≥30% reduction; 37% achieved ≥50% reduction.","Secondary endpoint met: 1.1 fewer cornstarch doses/day vs 0.2 for placebo (p=0.0011).","Safety profile acceptable; no dorsal root ganglion toxicity or thrombotic microangiopathy observed.","Company plans to discuss results with regulators and submit marketing application in 2025."],"consensus_eps_estimate":null,"consensus_eps_actual":null,"consensus_revenue_estimate":null,"consensus_revenue_actual":null,"consensus_period":null,"consensus_source":null,"sentiment":"positive","materiality_score":0.85,"calibrated_materiality_score":0.85,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":224473,"accession_number":"0000950170-24-052052","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2024-05-02T23:59:59+00:00","items":["2.02","9.01"],"status":"ready","headline":"Ultragenyx Q1 2024 revenue $109M (+8% YoY); net loss $171M; reaffirms guidance; pipeline advances","summary_kind":"llm","event_type":"earnings","confidence":"high","bullets":["Q1 total revenue $109M (+8% YoY); Crysvita $83M (+9%), Dojolvi $16M (+14%).","Net loss $171M ($2.03 per share) vs $164M ($2.33) in Q1 2023; operating expenses $274M.","Reaffirmed 2024 guidance: total revenue $500-530M, Crysvita $375-400M, Dojolvi $75-80M, cash use <$400M.","Positive interim Phase 1/2 GTX-102 data for Angelman syndrome; OI Phase 3 enrollment complete; DTX401 data expected Q2 2024.","Cash and equivalents $569M as of March 31, 2024; net cash used in operations $191M in Q1."],"consensus_eps_estimate":null,"consensus_eps_actual":-2.03,"consensus_revenue_estimate":null,"consensus_revenue_actual":108833000.0,"consensus_period":"2024-Q1","consensus_source":"xbrl_only","sentiment":"neutral","materiality_score":0.75,"calibrated_materiality_score":0.75,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":224472,"accession_number":"0000950123-24-003302","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2024-04-15T23:59:59+00:00","items":["8.01","9.01"],"status":"ready","headline":"Ultragenyx reports positive Phase 1/2 GTX-102 data in Angelman syndrome with rapid, sustained improvements","summary_kind":"llm","event_type":"other_material","confidence":"high","bullets":["Expansion Cohorts A&B (24 patients) at Day 170 showed clinically meaningful improvements across cognition, behavior, sleep, motor, and communication domains vs natural history.","Multi-domain responder index (MDRI) net response of +2.0 (p<0.0001) at Day 170; majority of patients achieved +2 to +4 domain improvement.","Dose-escalation Cohorts 4-7 (15 patients) at Day 758 showed sustained long-term benefit; MDRI net response +2.0 (p=0.0007) at Day 338.","No unexpected serious adverse events; three patients had mild-moderate lower extremity weakness (resolved, no recurrence on re-dosing).","FDA notified of safety events; no issues raised or additional actions required; data to be presented at AAN 2024."],"consensus_eps_estimate":null,"consensus_eps_actual":null,"consensus_revenue_estimate":null,"consensus_revenue_actual":null,"consensus_period":null,"consensus_source":null,"sentiment":"positive","materiality_score":0.85,"calibrated_materiality_score":0.85,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":243783,"accession_number":"0000950170-24-015856","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2024-02-15T23:59:59+00:00","items":["2.02","9.01"],"status":"ready","headline":"Ultragenyx FY2023 revenue $434M (+20% YoY); net loss narrows; 2024 guide $500-530M","summary_kind":"llm","event_type":"earnings","confidence":"high","bullets":["Total revenue 2023 $434M (+20% YoY); Crysvita $328M (+17%), Dojolvi $71M (+27%).","Net loss 2023 $607M ($8.25/sh) vs $707M ($10.12) in 2022; Q4 loss $123M ($1.52).","Cash $777M at Dec 31, 2023; 2024 net cash used in ops expected <$400M.","2024 guidance: total revenue $500-530M, Crysvita $375-400M, Dojolvi $75-80M.","Pipeline: DTX401 GSDIa Ph3 data H1 2024; GTX-102 Angelman expansion data H1 2024; setrusumab OI Ph3 enrollment Q1 end."],"consensus_eps_estimate":null,"consensus_eps_actual":-8.25,"consensus_revenue_estimate":null,"consensus_revenue_actual":434249000.0,"consensus_period":"2023-FY","consensus_source":"xbrl_only","sentiment":"positive","materiality_score":0.75,"calibrated_materiality_score":0.75,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":243782,"accession_number":"0000950170-24-002655","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2024-01-08T23:59:59+00:00","items":["2.02","9.01"],"status":"ready","headline":"Ultragenyx reports preliminary 2023 revenue $430-435M; guides 2024 revenue $500-530M","summary_kind":"llm","event_type":"other_material","confidence":"high","bullets":["Preliminary 2023 total revenue $430-435M; Crysvita $325-330M, Dojolvi $70-71M.","2024 total revenue guidance $500-530M; Crysvita $375-400M, Dojolvi $75-80M.","Year-end 2023 cash ~$776M; 2024 net cash used in ops guided <$400M.","Phase 3 data readouts expected H1 2024 for GSDIa gene therapy (DTX401) and OI study enrollment completion."],"consensus_eps_estimate":null,"consensus_eps_actual":null,"consensus_revenue_estimate":null,"consensus_revenue_actual":null,"consensus_period":null,"consensus_source":null,"sentiment":"neutral","materiality_score":0.65,"calibrated_materiality_score":0.65,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null},{"id":261187,"accession_number":"0000950170-23-072192","cik":1515673,"company_name":"Ultragenyx Pharmaceutical Inc.","ticker":"RARE","form_type":"8-K","filed_at":"2023-12-21T23:59:59+00:00","items":["5.03","9.01"],"status":"ready","headline":"Ultragenyx amends bylaws to align with Delaware law and SEC universal proxy card rules","summary_kind":"llm","event_type":"other","confidence":"high","bullets":["Board approved Second Amended and Restated Bylaws effective December 15, 2023.","Amendments reflect recent Delaware General Corporation Law changes and SEC universal proxy card rules.","Reserves white proxy card for company's exclusive use; clarifies ability to disregard non-compliant nominations.","Includes administrative, ministerial, clarifying and conforming changes."],"consensus_eps_estimate":null,"consensus_eps_actual":null,"consensus_revenue_estimate":null,"consensus_revenue_actual":null,"consensus_period":null,"consensus_source":null,"sentiment":"neutral","materiality_score":0.25,"calibrated_materiality_score":0.25,"market_cap_usd":2292829151.2817445,"market_cap_bucket":"not_microcap","market_cap_source":"finnhub_profile2","market_cap_as_of_date":"2026-05-28","daily_digest_date":null,"daily_digest_url":null,"daily_digest_rank":null}],"next_cursor":null}