📢 𝗝𝗨𝗦𝗧 𝗜𝗡: $Ultragenyx Pharma(RARE.US) Ultragenyx Phase 3 Aspire Study of Apazunersen Fails Primary and Key Secondary Endpoints
👉 𝗞𝗲𝘆 𝗛𝗶𝗴𝗵𝗹𝗶𝗴𝗵𝘁𝘀:➤ 𝗨𝗹𝘁𝗿𝗮𝗴𝗲𝗻𝘆𝘅's Phase 3 𝗔𝘀𝗽𝗶𝗿𝗲 study failed its primary endpoint.➤ Apazunersen showed no efficacy difference in 𝗕𝗮𝘆𝗹𝗲𝘆-𝟰 cognitive raw scores.➤ Study also missed the key secondary 𝗠𝗗𝗥𝗜 endpoint.➤ No efficacy support emerged across the 𝗳𝗶𝘃𝗲 individual MDRI endpoints.➤ Treatment groups were comparable and consistent with prior Phase 2 patients.➤ Safety remained consistent with previous 𝗣𝗵𝗮𝘀𝗲 𝟭/𝟮 findings.➤ Ultragenyx will evaluate the future of its 𝗮𝗽𝗮𝘇𝘂𝗻𝗲𝗿𝘀𝗲𝗻 program.➤ Company plans 𝘀𝗶𝗴𝗻𝗶𝗳𝗶𝗰𝗮𝗻𝘁 𝗲𝘅𝗽𝗲𝗻𝘀𝗲 𝗿𝗲𝗱𝘂𝗰𝘁𝗶𝗼𝗻𝘀 while maintaining commercial operations.➤ Angelman syndrome affects approximately 𝟲𝟬,𝟬𝟬𝟬 people in accessible markets.👉 𝗪𝗵𝘆 𝗧𝗵𝗶𝘀 𝗠𝗮𝘁𝘁𝗲𝗿𝘀:➤ Phase 3 failure creates major uncertainty around 𝗮𝗽𝗮𝘇𝘂𝗻𝗲𝗿𝘀𝗲𝗻's development future.➤ Missing both major endpoints substantially weakens the therapy's efficacy case.➤ Planned expense reductions could materially reshape 𝗨𝗹𝘁𝗿𝗮𝗴𝗲𝗻𝘆𝘅's operating priorities.➤ Outcome leaves a significant unmet need in 𝗔𝗻𝗴𝗲𝗹𝗺𝗮𝗻 𝘀𝘆𝗻𝗱𝗿𝗼𝗺𝗲 treatment.👉 𝗘𝘅𝗽𝗲𝗿𝘁 𝗦𝘁𝗮𝘁𝗲𝗺𝗲𝗻𝘁:𝗘𝗺𝗶𝗹 𝗞𝗮𝗸𝗸𝗶𝘀, 𝗠.𝗗., 𝗣𝗵.𝗗., Chief Executive Officer and President of Ultragenyx:"Based on everything we observed in the robust Phase 1/2 clinical development program and long-term extension study, we are disappointed by the Aspire result,"
Ultragenyx Pharma
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$Ultragenyx Pharma(RARE.US) | Guggenheim 𝗺𝗮𝗶𝗻𝘁𝗮𝗶𝗻𝘀 𝗕𝘂𝘆 on 𝗨𝗹𝘁𝗿𝗮𝗴𝗲𝗻𝘆𝘅 𝗣𝗵𝗮𝗿𝗺𝗮, cuts PT to $𝟯𝟱 from $𝟰𝟯
Analyst sees a binary ASPIRE outcome, with overwhelming success driving $55–$65 and failure sending RARE to the low teens. - M
📊 Watchlist for tomorrow (Not Financial Advice)
$Dell Tech(DELL.US) $Jabil(JBL.US) $Pharvaris BV(PHVS.US) $GE Vernova(GEV.US) $Capricor Therap(CAPR.US) $Ultragenyx Pharma(RARE.US) $Vir Biotechnology(VIR.US) $Vertiv(VRT.US) $Nebius(NBIS.US) $Arm(ARM.US)📌 See pinned tweet for how I use my WL - H
$Ultragenyx Pharma(RARE.US) | 𝐔𝐥𝐭𝐫𝐚𝐠𝐞𝐧𝐲𝐱: Cantor Fitzgerald maintains 𝐎𝐯𝐞𝐫𝐰𝐞𝐢𝐠𝐡𝐭, raises 𝐏𝐓 𝐭𝐨 $𝟗𝟔
Analyst sees growing confidence in GTX-102 for Angelman syndrome driving major upside. - N
New: Gil Cisneros (D) just filed up to $1,300,000 of new stock trades
The ones that stood out are 3 pharma picks: $Agios Pharmac(AGIO.US), $Bridgebio Pharma(BBIO.US), and $Ultragenyx Pharma(RARE.US)The crazy part is that all 3 had major FDA decisions go their way within weeks. every stock is up 10-26%Cisneros doesn't sit on a single health committee but 3/3 on FDA decisions is pretty good - G
Trump big bully, I will top-up on some banking stock due to encouraging earnings
Featured📈 Jan 14 U.S. Market Hotlist: Fed Under Fire, Banks Reporting, AI Stocks Surging👋 Hey brilliant investors! Another packed morning for U.S. markets. Here’s your quick breakdown with the juice you need:
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Brilliant!
Featured📈 Jan 14 U.S. Market Hotlist: Fed Under Fire, Banks Reporting, AI Stocks Surging👋 Hey brilliant investors! Another packed morning for U.S. markets. Here’s your quick breakdown with the juice you need:
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Diversifying more into hard assets as $ may quickly lose value with inflation staying high
Featured📈 Jan 14 U.S. Market Hotlist: Fed Under Fire, Banks Reporting, AI Stocks Surging👋 Hey brilliant investors! Another packed morning for U.S. markets. Here’s your quick breakdown with the juice you need:
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I believe the next leg up for AI is the monetisation phase. At the moment, they are still in the infrastructure stage hence the ‘bubble’ talk and FUD. There will still be pullbacks in AI names along the way but the main beneficiaries of the new economy moving forward IMO will be those associated with AI.
Featured📈 Jan 14 U.S. Market Hotlist: Fed Under Fire, Banks Reporting, AI Stocks Surging👋 Hey brilliant investors! Another packed morning for U.S. markets. Here’s your quick breakdown with the juice you need:
