Ultragenyx Pharmaceu (RARE)

$31.21 +0.31 (1.00%)
Market Cap 2.98B
Revenue (ttm) 560.23M
Net Income (ttm) -569.18M
EPS (ttm) N/A
PE Ratio 0.00
Forward PE N/A
Profit Margin -101.60%
Debt to Equity Ratio 0.00
Volume 1,364,500
Avg Vol 1,688,420
Day's Range N/A - N/A
Shares Out 96.37M
Stochastic %K 93%
Beta 0.21
Analysts Strong Sell
Price Target $86.05

Company Profile

Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in North America, Latin America, Europe, the Middle East, Africa, and the Asia-Pacific. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevi...

Industry: Biotechnology
Sector: Healthcare
Phone: 415 483 8800
Address:
60 Leveroni Court, Novato, United States
MadMaverick
MadMaverick Oct. 4 at 2:47 PM
Based on my weekly chart and my custom indicator. $RARE is setting up over $32.
0 · Reply
SweepCastApp
SweepCastApp Oct. 3 at 10:02 AM
$RARE: Unusual Options Activity Alerted CALL flow observed 100x contracts at Strike price of $40 Exp on 01/16/2026 with Premium of $27K and showing NEUTRAL Sentiment
0 · Reply
notreload_ai
notreload_ai Oct. 2 at 12:49 PM
JPMorgan is optimistic about $RARE's drug setrusumab (UX143) and expects big gains ahead of key study updates. https://notreload.xyz/ultragenyx-positive-catalyst-watch-jpmorgan-analysis/
0 · Reply
lidopete
lidopete Oct. 2 at 12:32 PM
$MREO $RARE Ultragenyx placed on 'Positive Catalyst Watch' at JPMorgan JPMorgan analyst Anupam Rama placed shares of Ultragenyx on "Positive Catalyst Watch" ahead of the company's ORBIT and COSMIC study updates. The firm has confidence in setrusumab's clinical success and keeps an Overweight rating on the shares with a $125 price target. At current share levels, JPMorgan sees a "very positive" reward/risk profile heading into the updates with more win scenarios than downside scenarios. Note, MREO is RARE's partner with setrusumab
1 · Reply
xbibackto65s
xbibackto65s Oct. 1 at 4:28 PM
$RARE what a pos always down even when $XBI is flying
1 · Reply
TimRegan_WTR
TimRegan_WTR Oct. 1 at 12:41 PM
$JLL $CRML $RARE $FEAM $ATLX
0 · Reply
research_WTR
research_WTR Oct. 1 at 9:58 AM
Did you catch Dmitry Silversteyn recap from Part 2 of his WTR Symposium Series? “Critical Elements Mining and Processing Infrastructure Buildout – A Matter of National Security”. Read here! Those interested can listen to this podcast on Apple Podcasts, Spotify, or on our website. Links are accessible in our full report! https://www.watertowerresearch.com/doc?docID=IR_ChemiMat_09302025 Part 2 included: @JindaleeL (JLL-ASX), @CriticalMetals_ (CRML), Tactical Resources Corp. (RARE-TSX), 5E Advanced Materials, Inc. (FEAM), and Atlas Lithium Corporation (ATLX) $JLL $CRML $RARE $FEAM $ATLX #SmallCap #Stocks #Investing
0 · Reply
LongerThanLong
LongerThanLong Sep. 30 at 2:32 PM
$AMGN together with $BIIB, $RARE, $DEO my favorite value plays these days. Won't get crazy rich but 10%+ should happen within a few months.
1 · Reply
highnihilism
highnihilism Sep. 30 at 8:28 AM
$VMEO Vimeo: 20 trades, $164K vs $24K avg (6.94x !!). $164K calls / $0 puts. $RARE Ultragenyx: 139 trades, $181K vs $563K avg (0.32x). $163K calls / $18K puts. $HSY Hershey: 463 trades, $291K vs $1.42M avg (0.21x). $161K calls / $130K puts. $BYRN Byrna Technologies: 147 trades, $182K vs $169K avg (1.08x). $160K calls / $22K puts.
0 · Reply
BigSqueezeComing
BigSqueezeComing Sep. 30 at 8:12 AM
$RARE Cantor Fitzgerald Reiterates a Buy Rating on Ultragenyx Pharmaceutical (RARE) By Noor Ul Ain Rehman | September 30, 2025, 4:00 AM
0 · Reply
Latest News on RARE
Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)

Oct 3, 2025, 4:30 PM EDT - 1 day ago

Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)

Ultragenyx Appoints Eric Olson as Chief Business Officer and Executive Vice President

Sep 30, 2025, 4:05 PM EDT - 4 days ago

Ultragenyx Appoints Eric Olson as Chief Business Officer and Executive Vice President

Ultragenyx Pharmaceutical Inc. (RARE) Presents at Bank of America Global Healthcare Conference 2025 Transcript

Sep 23, 2025, 10:13 AM EDT - 11 days ago

Ultragenyx Pharmaceutical Inc. (RARE) Presents at Bank of America Global Healthcare Conference 2025 Transcript

Ultragenyx Pharmaceutical Inc. (RARE) Presents At Morgan Stanley 23rd Annual Global Healthcare Conference Transcript

Sep 10, 2025, 12:17 PM EDT - 24 days ago

Ultragenyx Pharmaceutical Inc. (RARE) Presents At Morgan Stanley 23rd Annual Global Healthcare Conference Transcript

Ultragenyx Announces Positive Longer-term Data from Phase 3 Study of DTX401 AAV Gene Therapy for the Treatment of Glycogen Storage Disease Type Ia (GSDIa)

Sep 8, 2025, 4:05 PM EDT - 26 days ago

Ultragenyx Announces Positive Longer-term Data from Phase 3 Study of DTX401 AAV Gene Therapy for the Treatment of Glycogen Storage Disease Type Ia (GSDIa)

Ultragenyx Pharmaceutical Inc. (RARE) Presents At Cantor Global Healthcare Conference 2025 Transcript

Sep 4, 2025, 2:27 PM EDT - 4 weeks ago

Ultragenyx Pharmaceutical Inc. (RARE) Presents At Cantor Global Healthcare Conference 2025 Transcript

Ultragenyx to Participate at Investor Conferences in September

Aug 29, 2025, 4:30 PM EDT - 5 weeks ago

Ultragenyx to Participate at Investor Conferences in September

Ultragenyx Initiates Rolling Submission of Biologics License Application (BLA) to U.S. FDA for DTX401 AAV Gene Therapy for the Treatment of Glycogen Storage Disease Type Ia (GSDIa)

Aug 18, 2025, 8:00 AM EDT - 6 weeks ago

Ultragenyx Initiates Rolling Submission of Biologics License Application (BLA) to U.S. FDA for DTX401 AAV Gene Therapy for the Treatment of Glycogen Storage Disease Type Ia (GSDIa)

Ultragenyx Pharmaceutical Inc. (RARE) Q2 2025 Earnings Call Transcript

Aug 6, 2025, 12:42 AM EDT - 2 months ago

Ultragenyx Pharmaceutical Inc. (RARE) Q2 2025 Earnings Call Transcript

Ultragenyx Reports Second Quarter 2025 Financial Results and Corporate Update

Aug 5, 2025, 4:01 PM EDT - 2 months ago

Ultragenyx Reports Second Quarter 2025 Financial Results and Corporate Update

Ultragenyx to Host Conference Call for Second Quarter 2025 Financial Results and Corporate Update

Jul 30, 2025, 8:30 AM EDT - 2 months ago

Ultragenyx to Host Conference Call for Second Quarter 2025 Financial Results and Corporate Update

Ultragenyx: July Selloff Creates Buying Opportunity Ahead Of UX143 Catalyst

Jul 29, 2025, 9:10 AM EDT - 2 months ago

Ultragenyx: July Selloff Creates Buying Opportunity Ahead Of UX143 Catalyst

Ultragenyx Represents 'Intriguing Buying Opportunity,' Despite Recent Setbacks

Jul 28, 2025, 1:16 PM EDT - 2 months ago

Ultragenyx Represents 'Intriguing Buying Opportunity,' Despite Recent Setbacks

RARE Investors Have Opportunity to Join Ultragenyx Pharmaceutical Inc. Fraud Investigation with the Schall Law Firm

Jul 18, 2025, 3:07 PM EDT - 2 months ago

RARE Investors Have Opportunity to Join Ultragenyx Pharmaceutical Inc. Fraud Investigation with the Schall Law Firm

Ultragenyx Pharmaceutical, Apple And Other Big Stocks Moving Lower In Monday's Pre-Market Session

Jul 14, 2025, 8:14 AM EDT - 2 months ago

Ultragenyx Pharmaceutical, Apple And Other Big Stocks Moving Lower In Monday's Pre-Market Session

AAPL

US FDA declines to approve Ultragenyx's gene therapy for rare genetic disorder

Jul 11, 2025, 4:45 PM EDT - 3 months ago

US FDA declines to approve Ultragenyx's gene therapy for rare genetic disorder

Ultragenyx Receives Complete Response Letter from FDA for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA)

Jul 11, 2025, 4:30 PM EDT - 3 months ago

Ultragenyx Receives Complete Response Letter from FDA for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA)

Ultragenyx, Mereo Shares Dive As Brittle Bone Disease Drug Misses Early Win

Jul 10, 2025, 10:06 AM EDT - 3 months ago

Ultragenyx, Mereo Shares Dive As Brittle Bone Disease Drug Misses Early Win

Methode Electronics, Ultragenyx Pharmaceutical And Other Big Stocks Moving Lower In Thursday's Pre-Market Session

Jul 10, 2025, 8:00 AM EDT - 3 months ago

Methode Electronics, Ultragenyx Pharmaceutical And Other Big Stocks Moving Lower In Thursday's Pre-Market Session

MEI

Ultragenyx and Mereo BioPharma Announce UX143 Phase 3 Orbit Study for Osteogenesis Imperfecta Progressing to Final Analysis

Jul 9, 2025, 4:05 PM EDT - 3 months ago

Ultragenyx and Mereo BioPharma Announce UX143 Phase 3 Orbit Study for Osteogenesis Imperfecta Progressing to Final Analysis

Ultragenyx Receives Breakthrough Therapy Designation for GTX-102 in Angelman Syndrome

Jun 27, 2025, 8:00 AM EDT - 3 months ago

Ultragenyx Receives Breakthrough Therapy Designation for GTX-102 in Angelman Syndrome

Ultragenyx to Participate at Goldman Sachs 46th Annual Global Healthcare Conference

Jun 3, 2025, 5:00 PM EDT - 4 months ago

Ultragenyx to Participate at Goldman Sachs 46th Annual Global Healthcare Conference

Ultragenyx Underscores Continued Commitment to Rare Disease Innovation with 2024 Impact Report

May 14, 2025, 4:30 PM EDT - 5 months ago

Ultragenyx Underscores Continued Commitment to Rare Disease Innovation with 2024 Impact Report

Ultragenyx to Participate at Bank of America's 2025 Healthcare Conference

May 7, 2025, 5:00 PM EDT - 5 months ago

Ultragenyx to Participate at Bank of America's 2025 Healthcare Conference

Ultragenyx Pharmaceutical Inc. (RARE) Q1 2025 Earnings Call Transcript

May 6, 2025, 11:31 PM EDT - 5 months ago

Ultragenyx Pharmaceutical Inc. (RARE) Q1 2025 Earnings Call Transcript

Ultragenyx Reports First Quarter 2025 Financial Results and Corporate Update

May 6, 2025, 4:01 PM EDT - 5 months ago

Ultragenyx Reports First Quarter 2025 Financial Results and Corporate Update

Ultragenyx to Host Conference Call for First Quarter 2025 Financial Results and Corporate Update

Apr 30, 2025, 5:00 PM EDT - 5 months ago

Ultragenyx to Host Conference Call for First Quarter 2025 Financial Results and Corporate Update

Ultragenyx: Commercial Engine Fuels Rare Disease Pipeline

Apr 16, 2025, 9:49 AM EDT - 6 months ago

Ultragenyx: Commercial Engine Fuels Rare Disease Pipeline

Ultragenyx to Participate at Investor Conferences in March

Feb 24, 2025, 4:30 PM EST - 7 months ago

Ultragenyx to Participate at Investor Conferences in March

Ultragenyx Pharmaceutical Inc. (RARE) Q4 2024 Earnings Call Transcript

Feb 13, 2025, 9:53 PM EST - 8 months ago

Ultragenyx Pharmaceutical Inc. (RARE) Q4 2024 Earnings Call Transcript

Ultragenyx Reports Fourth Quarter and Full Year 2024 Financial Results and Corporate Update

Feb 13, 2025, 4:01 PM EST - 8 months ago

Ultragenyx Reports Fourth Quarter and Full Year 2024 Financial Results and Corporate Update

Ultragenyx to Host Conference Call for Fourth Quarter and Full-Year 2024 Financial Results and Corporate Update

Feb 6, 2025, 4:30 PM EST - 8 months ago

Ultragenyx to Host Conference Call for Fourth Quarter and Full-Year 2024 Financial Results and Corporate Update

Ultragenyx Announces New Data Demonstrating that Treatment with UX111 AAV Gene Therapy Significantly Improved Clinical Function Across Multiple Developmental Domains in Children with Sanfilippo Syndrome Type A (MPS IIIA) Correlated with Sustained Reductions in CSF-HS

Feb 5, 2025, 8:00 AM EST - 8 months ago

Ultragenyx Announces New Data Demonstrating that Treatment with UX111 AAV Gene Therapy Significantly Improved Clinical Function Across Multiple Developmental Domains in Children with Sanfilippo Syndrome Type A (MPS IIIA) Correlated with Sustained Reductions in CSF-HS

Ultragenyx's Multiple Commercial Assets And Candidates Impress Analyst; Sees Solid Upside

Jan 15, 2025, 2:05 PM EST - 9 months ago

Ultragenyx's Multiple Commercial Assets And Candidates Impress Analyst; Sees Solid Upside

Ultragenyx Reports Preliminary 2024 Revenue, Financial Guidance for 2025, Pipeline Updates, and 2025 Milestones

Jan 12, 2025, 11:00 AM EST - 9 months ago

Ultragenyx Reports Preliminary 2024 Revenue, Financial Guidance for 2025, Pipeline Updates, and 2025 Milestones

Ultragenyx to Present at the 43rd Annual J.P. Morgan Healthcare Conference

Jan 6, 2025, 4:30 PM EST - 9 months ago

Ultragenyx to Present at the 43rd Annual J.P. Morgan Healthcare Conference

European Commission (EC) Extends the Approval of Evkeeza® (evinacumab) to Children as Young as 6-months Old with Homozygous Familial Hypercholesterolemia (HoFH)

Jan 6, 2025, 2:00 AM EST - 9 months ago

European Commission (EC) Extends the Approval of Evkeeza® (evinacumab) to Children as Young as 6-months Old with Homozygous Familial Hypercholesterolemia (HoFH)

NHS England Rolls Out Evkeeza® ▼(evinacumab) for Eligible Adults and Adolescents Aged 12 Years and Older with Homozygous Familial Hypercholesterolaemia (HoFH)

Dec 20, 2024, 3:00 AM EST - 10 months ago

NHS England Rolls Out Evkeeza® ▼(evinacumab) for Eligible Adults and Adolescents Aged 12 Years and Older with Homozygous Familial Hypercholesterolaemia (HoFH)

Ultragenyx Announces First Patient Dosed in Pivotal Phase 3 Aspire Study Evaluating GTX-102 in Angelman Syndrome

Dec 19, 2024, 4:05 PM EST - 10 months ago

Ultragenyx Announces First Patient Dosed in Pivotal Phase 3 Aspire Study Evaluating GTX-102 in Angelman Syndrome

Ultragenyx to Participate in Investor Conferences in December

Nov 26, 2024, 4:30 PM EST - 11 months ago

Ultragenyx to Participate in Investor Conferences in December

Ultragenyx: Ready For More Growth After Q3 Earnings Beat

Nov 12, 2024, 11:12 AM EST - 11 months ago

Ultragenyx: Ready For More Growth After Q3 Earnings Beat

Ultragenyx Presents Positive Update on GTX-102 Angelman Syndrome Program at FAST's 17th Annual Global Science Summit

Nov 9, 2024, 10:30 AM EST - 11 months ago

Ultragenyx Presents Positive Update on GTX-102 Angelman Syndrome Program at FAST's 17th Annual Global Science Summit

Ultragenyx Pharmaceutical Inc. (RARE) Q3 2024 Earnings Call Transcript

Nov 5, 2024, 10:40 PM EST - 11 months ago

Ultragenyx Pharmaceutical Inc. (RARE) Q3 2024 Earnings Call Transcript

Ultragenyx Reports Third Quarter 2024 Financial Results and Corporate Update

Nov 5, 2024, 4:01 PM EST - 11 months ago

Ultragenyx Reports Third Quarter 2024 Financial Results and Corporate Update

Ultragenyx Provides Update on Stage 1 Cohorts in Pivotal Phase 1/2/3 Cyprus2+ Study Evaluating UX701 Gene Therapy for the Treatment of Wilson Disease

Oct 3, 2024, 4:30 PM EDT - 1 year ago

Ultragenyx Provides Update on Stage 1 Cohorts in Pivotal Phase 1/2/3 Cyprus2+ Study Evaluating UX701 Gene Therapy for the Treatment of Wilson Disease

Ultragenyx Announces Upcoming Setrusumab (UX143) Presentations at the ASBMR 2024 Annual Meeting

Sep 26, 2024, 8:00 AM EDT - 1 year ago

Ultragenyx Announces Upcoming Setrusumab (UX143) Presentations at the ASBMR 2024 Annual Meeting

Ultragenyx to Participate in Investor Conferences in September

Aug 29, 2024, 4:30 PM EDT - 1 year ago

Ultragenyx to Participate in Investor Conferences in September

Ultragenyx's Strong Pipeline And Valuation Concerns: A Balanced Perspective

Aug 22, 2024, 10:22 AM EDT - 1 year ago

Ultragenyx's Strong Pipeline And Valuation Concerns: A Balanced Perspective

MadMaverick
MadMaverick Oct. 4 at 2:47 PM
Based on my weekly chart and my custom indicator. $RARE is setting up over $32.
0 · Reply
SweepCastApp
SweepCastApp Oct. 3 at 10:02 AM
$RARE: Unusual Options Activity Alerted CALL flow observed 100x contracts at Strike price of $40 Exp on 01/16/2026 with Premium of $27K and showing NEUTRAL Sentiment
0 · Reply
notreload_ai
notreload_ai Oct. 2 at 12:49 PM
JPMorgan is optimistic about $RARE's drug setrusumab (UX143) and expects big gains ahead of key study updates. https://notreload.xyz/ultragenyx-positive-catalyst-watch-jpmorgan-analysis/
0 · Reply
lidopete
lidopete Oct. 2 at 12:32 PM
$MREO $RARE Ultragenyx placed on 'Positive Catalyst Watch' at JPMorgan JPMorgan analyst Anupam Rama placed shares of Ultragenyx on "Positive Catalyst Watch" ahead of the company's ORBIT and COSMIC study updates. The firm has confidence in setrusumab's clinical success and keeps an Overweight rating on the shares with a $125 price target. At current share levels, JPMorgan sees a "very positive" reward/risk profile heading into the updates with more win scenarios than downside scenarios. Note, MREO is RARE's partner with setrusumab
1 · Reply
xbibackto65s
xbibackto65s Oct. 1 at 4:28 PM
$RARE what a pos always down even when $XBI is flying
1 · Reply
TimRegan_WTR
TimRegan_WTR Oct. 1 at 12:41 PM
$JLL $CRML $RARE $FEAM $ATLX
0 · Reply
research_WTR
research_WTR Oct. 1 at 9:58 AM
Did you catch Dmitry Silversteyn recap from Part 2 of his WTR Symposium Series? “Critical Elements Mining and Processing Infrastructure Buildout – A Matter of National Security”. Read here! Those interested can listen to this podcast on Apple Podcasts, Spotify, or on our website. Links are accessible in our full report! https://www.watertowerresearch.com/doc?docID=IR_ChemiMat_09302025 Part 2 included: @JindaleeL (JLL-ASX), @CriticalMetals_ (CRML), Tactical Resources Corp. (RARE-TSX), 5E Advanced Materials, Inc. (FEAM), and Atlas Lithium Corporation (ATLX) $JLL $CRML $RARE $FEAM $ATLX #SmallCap #Stocks #Investing
0 · Reply
LongerThanLong
LongerThanLong Sep. 30 at 2:32 PM
$AMGN together with $BIIB, $RARE, $DEO my favorite value plays these days. Won't get crazy rich but 10%+ should happen within a few months.
1 · Reply
highnihilism
highnihilism Sep. 30 at 8:28 AM
$VMEO Vimeo: 20 trades, $164K vs $24K avg (6.94x !!). $164K calls / $0 puts. $RARE Ultragenyx: 139 trades, $181K vs $563K avg (0.32x). $163K calls / $18K puts. $HSY Hershey: 463 trades, $291K vs $1.42M avg (0.21x). $161K calls / $130K puts. $BYRN Byrna Technologies: 147 trades, $182K vs $169K avg (1.08x). $160K calls / $22K puts.
0 · Reply
BigSqueezeComing
BigSqueezeComing Sep. 30 at 8:12 AM
$RARE Cantor Fitzgerald Reiterates a Buy Rating on Ultragenyx Pharmaceutical (RARE) By Noor Ul Ain Rehman | September 30, 2025, 4:00 AM
0 · Reply
Airbags
Airbags Sep. 29 at 4:24 PM
$RARE does anyone know why this is popping today?
2 · Reply
SannieVerraderlijk
SannieVerraderlijk Sep. 29 at 3:38 PM
$RARE A sign of life
1 · Reply
SweepCastApp
SweepCastApp Sep. 29 at 8:49 AM
$RARE: Unusual Options Activity Alerted PUT flow observed 98x contracts at Strike price of $30 Exp on 12/19/2025 with Premium of $44K and showing NEUTRAL Sentiment
0 · Reply
Jphil07
Jphil07 Sep. 26 at 12:07 AM
$RARE price predictions if phase 3 orbit does well?
1 · Reply
TimRegan_WTR
TimRegan_WTR Sep. 24 at 1:02 PM
$JLL $CRML $RARE $FEAM $ATLX
0 · Reply
research_WTR
research_WTR Sep. 24 at 10:58 AM
Critical Elements Mining & Processing Symposium – Part 2 🎙 Hosted by Shawn Severson & Dmitry Silversteyn, this WTR Small-Cap Spotlight episode features leaders from Jindalee Lithium Limited, Critical Metals Corp., Tactical Resources Corp., 5E Advanced Materials, Inc., with insights from Atlas Lithium Corporation (Nasdaq: ATLX), covering: 🔹 Global demand trends, supply bottlenecks & pricing pressures 🔹 National security implications of reducing foreign dependence 🔹 Domestic infrastructure buildout: capital, regulations, workforce & government support 🔹 Actionable insights to accelerate U.S. & allied critical materials production Listen below! Apple: https://podcasts.apple.com/us/podcast/episode-151-critical-elements-mining-and-processing/id1693448305?i=1000728195984 Spotify: https://open.spotify.com/episode/2wFDONb1MJ7mIwpmdgWv66?si=2bb5173394a245fc Website: https://www.watertowerresearch.com/media-detail/2548/Media Others: https://pod.link/1693448305 #CriticalMetals #Mining #EnergyTransition #NationalSecurity #SupplyChain #WTRSmallCapSpotlight #CleanEnergy #Podcast $JLL $CRML $RARE $FEAM $ATLX
0 · Reply
Mercuria
Mercuria Sep. 22 at 11:29 PM
$RARE never heard of this stock before, know nothing of the company, but had a dream it’s going to fly so I’m interested. Anyone been following it and what to share thoughts?
1 · Reply
Longterm_Inv
Longterm_Inv Sep. 19 at 6:30 PM
$RARE Here's another stock that should not be overlooked. No reason to feel anything but optimism about this.
0 · Reply
Kaputt
Kaputt Sep. 19 at 3:28 PM
$RARE I’m writing this from a wet dream. This stock will pop like a jailbird’s first erection on his first date.
0 · Reply
SQUEAKSBANDIT
SQUEAKSBANDIT Sep. 18 at 2:55 PM
$RARE I topped off my position here just under $28.8 this am it could go lower short term, but with a longer term horizon $RARE is a winner of course we may not get to the long term as big pharma is likely already taking a good hard look at this one
0 · Reply
highnihilism
highnihilism Sep. 16 at 2:50 PM
Biotech → $CRNX Crinetics Pharmaceuticals $IONS Ionis Pharmaceuticals $PTCT PTC Therapeutics $RARE Ultragenyx Pharmaceutical $MDGL Madrigal Pharmaceuticals
0 · Reply
lidopete
lidopete Sep. 16 at 12:49 PM
AI Generated$MREO $RARE part 2=to 0.00 during treatment in the Phase 2 cohort, and that this reduction persisted in later interim readouts, using nearly identical language across several announcements. Meaning of Statements The company consistently indicates that interim clinical efficacy (fracture rate reduction) matches the effect observed in Phase 2. While the "quote" is not verbatim, the communicated message aligns with the meaning: interim data confirm fracture rate reductions previously seen in earlier trials. Sources for Reference Analyst Day transcripts ASBMR Annual Meeting late-breaker presentation (2023) Press releases (GlobeNewswire, Nasdaq) Earnings call discussions by Ultragenyx CMO Summary: Ultragenyx has repeatedly conveyed—both verbally and in writing—that interim clinical readouts for setrusumab are consistent with fracture rate reductions seen in Phase 2, matching the intent and factual content of the quote in question, despite not using those precise words.
0 · Reply