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13 stories mentioning RAREUpdated 6d ago

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Wire

Ultragenyx Shares Rise After FDA Approval for Sanfilippo Syndrome Treatment

Ultragenyx Pharmaceutical (RARE) shares rose 11% after the company won FDA approval for Fayuvi, the first treatment for children with Sanfilippo syndrome type A, a rare genetic condition that progressively damages the brain and nervous system.Delivered as a one-time IV infusion, Fayuvi uses a modified virus to deliver a healthy gene, replacing the enzyme the patients lack. the FDA said Thursday in a statement. In clinical trials, treated children aged 2 to 5 maintained or improved their cognitive abilities compared to historical patient outcomes, the agency said.The FDA noted common side effects like fever, nausea, and elevated liver enzymes. The label also warns of potential long-term risks, including blood clotting issues and tumor formation, requiring patients to take immune-suppressing steroids around the time of treatment.Price: $14.34, Change: $+1.46, Percent Change: +11.34%

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Wire

Sector Update: Healthcare Stocks Edge Higher Late Afternoon

Healthcare stocks were slightly higher late Thursday afternoon, with the NYSE Healthcare Index increasing 0.2% and the State Street Health Care Select Sector SPDR ETF (XLV) up 0.1%.The iShares Biotechnology ETF (IBB) eased 0.3%.In corporate news, Haleon (HLN) has negotiated better shelf placement at Walmart (WMT) and Target (TGT), Reuters reported. The company reportedly said it has offered major retailers better prices and promotions, as well as exclusivity on new products, in exchange for more prominent shelf space. Haleon shares were down 1.1%.Hutchmed (HCM) shares jumped 17% after the company said its unit licensed rights to GSK (GSK) to develop and commercialize experimental cancer treatment HMPL-A830 outside greater China. GSK shares added 0.8%.AbbVie (ABBV) said Thursday that it has completed its acquisition of Apogee Therapeutics (APGE), valuing the clinical-stage biotechnology company at about $10.9 billion, or $135.11 per share. AbbVie shares were down 0.5%.Ultragenyx Pharmaceutical (RARE) shares fell 44% after the firm said a phase 3 study of apazunersen in Angelman syndrome did not achieve the primary endpoint nor a key secondary endpoint.Eli Lilly (LLY) unit Centessa Pharmaceuticals has achieved a late-stage development milestone related to its selective orexin receptor 2 agonist portfolio, triggering an $8.6 million payment to research collaboration partner Nxera Pharma, Nxera said Thursday. Lilly shares were down 0.2%.

$ABBV$HCM$HLN$LLY$RARE
Wire

Top Midday Decliners

Ultragenyx Pharmaceutical (RARE) said late Wednesday a phase 3 study of apazunersen in Angelman syndrome did not meet its primary endpoint nor a key secondary endpoint.Shares slumped 45% as intraday trading volume catapulted to over 19.8 million from a daily average of about 2.4 million.Tyson Foods (TSN) reduced its fiscal 2026 revenue growth guidance range to 1.5% to 2% from 2.5% to 3.5% previously, the company said Thursday.Shares fell 7.2%, with intraday trading volume of over 4.1 million, versus a daily average of about 3.3 million.Campbell's (CPB) shares dropped 8% after the company guided for a soft fiscal 2027 outlook amid persistent inflation worries and cut its quarterly dividend on Thursday.Trading volume grew to more than 27.2 million from a daily average of almost 8.0 million.Price: $14.72, Change: $-11.82, Percent Change: -44.53%

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Sectors

Sector Update: Healthcare Stocks Higher Thursday Afternoon

Healthcare stocks were slightly higher Thursday afternoon, with the NYSE Healthcare Index increasing 0.3% and the State Street Health Care Select Sector SPDR ETF (XLV) up 0.1%.The iShares Biotechnology ETF (IBB) eased 0.3%.In corporate news, Hutchmed (HCM) shares jumped 17% after the company said its unit licensed rights to GSK (GSK) to develop and commercialize experimental cancer treatment HMPL-A830 outside greater China. GSK shares added 0.8%.AbbVie (ABBV) said Thursday that it has completed its acquisition of Apogee Therapeutics (APGE), valuing the clinical-stage biotechnology company at about $10.9 billion, or $135.11 per share. AbbVie shares were down 0.3%.Ultragenyx Pharmaceutical (RARE) shares fell 45% after the firm said a phase 3 study of apazunersen in Angelman syndrome did not achieve the primary endpoint nor a key secondary endpoint.

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Sectors

Sector Update: Healthcare Stocks Edge Higher Pre-Bell Thursday

Healthcare stocks were edging higher pre-bell Thursday, with the State Street Health Care Select Sector SPDR ETF (XLV) up 0.3% and the iShares Biotechnology ETF (IBB) advancing by 0.5%.Hutchmed (HCM) shares were up more than 11% after the company said its unit licensed rights to GSK (GSK) to develop and commercialize experimental cancer treatment HMPL-A830 outside greater China.Ultragenyx Pharmaceutical (RARE) shares were down more than 46% after the company said its phase 3 study of apazunersen in Angelman syndrome failed to achieve the primary endpoint or a key secondary endpoint.Liquidia (LQDA) was up more than 1% after the company said the US Food and Drug Administration has granted fast-track designation to Yutrepia inhalation powder to treat Raynaud's phenomenon associated with systemic sclerosis.

$GSK$HCM$IBB$LQDA$RARE$XLV
Research

Evercore ISI Downgrades Ultragenyx Pharmaceutical to In Line From Outperform, Adjusts Price Target to $16 From $34

Ultragenyx Pharmaceutical (RARE) has an average rating of overweight and mean price target of $33.95, according to analysts polled by FactSet.

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Research

William Blair Downgrades Ultragenyx Pharmaceutical to Market Perform From Outperform

Ultragenyx Pharmaceutical (RARE) has an average rating of overweight and mean price target of $33.95, according to analysts polled by FactSet.

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Research

JPMorgan Downgrades Ultragenyx Pharmaceutical to Neutral From Overweight, Lowers Price Target to $36 From $80

Ultragenyx Pharmaceutical (RARE) has an average rating of overweight and mean price target of $33.95, according to analysts polled by FactSet.

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Research

Baird Downgrades Ultragenyx Pharmaceutical to Neutral From Outperform, Adjusts Price Target to $16 From $40

Ultragenyx Pharmaceutical (RARE) has an average rating of overweight and mean price target of $33.95, according to analysts polled by FactSet.

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Wire

Ultragenyx Says Phase 3 Study on Rare Neurogenetic Disorder Missed Primary, Secondary Endpoints; Shares Fall After-Hours

Ultragenyx Pharmaceutical (RARE) said late Wednesday the phase 3 Aspire study of apazunersen in Angelman syndrome, a rare, neurogenetic disorder, did not achieve its primary endpoint or its key secondary endpoint.The late-stage study did not reach the primary endpoint of change from Baseline in Bayley-4 cognitive raw score nor the key secondary endpoint of net response in Multidomain Responder Index, according to a statement.The safety profile for the phase 3 trial remained consistent with phase 1 and phase 2, the company said.Ultragenyx said it will assess the apazunersen program following this outcome and make a decision on its disposition.Shares of Ultragenyx were down about 45% in after-hours trading.

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Sectors

Sector Update: Healthcare Stocks Fall Pre-Bell Thursday

Healthcare stocks were falling pre-bell Thursday, with the State Street Health Care Select Sector SPDR ETF (XLV) down 0.6% and the iShares Biotechnology ETF (IBB) declining by 0.9%.Ultragenyx Pharmaceutical (RARE) has received accelerated approval from the US Food and Drug Administration for Genglycos to treat glycogen storage disease type Ia, a rare genetic metabolic disorder due to an enzyme deficiency, the drugmaker said. Ultragenyx Pharmaceutical shares were up more than 7% premarket.Nuvation Bio (NUVB) stock was up more than 2% after the company said it has received US Food and Drug Administration fast-track designation for Safusidenib for the treatment IDH1-mutant glioma.AC Immune (ACIU) shares were up more than 2% after the company said preliminary results from a first-in-human Phase 1 trial of ACI-19764 showed the drug was safe and well tolerated in healthy volunteers.

$ACIU$IBB$NUVB$RARE$XLV
Sectors

Sector Update: Healthcare

Healthcare stocks were leaning lower pre-bell Thursday, with the State Street Health Care Select Sector SPDR ETF (XLV) down 0.5% and the iShares Biotechnology ETF (IBB) declining by 1%.Ultragenyx Pharmaceutical (RARE) has received accelerated approval from the US Food and Drug Administration for Genglycos to treat glycogen storage disease type Ia, a rare genetic metabolic disorder due to an enzyme deficiency, the drugmaker said. Ultragenyx Pharmaceutical shares were up more than 6% premarket.

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Research

Wedbush Lifts Price Target on Ultragenyx Pharmaceutical to $27 From $25, Keeps Neutral Rating

Ultragenyx Pharmaceutical (RARE) has an average rating of buy and mean price target of $53.40, according to analysts polled by FactSet.

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