Ultragenyx制药

📢 𝗝𝗨𝗦𝗧 𝗜𝗡: $Ultragenyx制药(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制药(RARE.US) | Guggenheim 维持 UltraGenexy Pharma 买入评级，目标价从 $43 下调至 $35

分析师认为 ASPIRE 结果具有二元性，若取得压倒性成功，股价将驱动至 $55–$65；若失败，RARE 将跌至十几美元低位。

📊 明日观察清单（非财务建议）

$戴尔科技-C(DELL.US) $捷普(JBL.US) $Pharvaris BV(PHVS.US) $GE Vernova(GEV.US) $Capricor治疗(CAPR.US) $Ultragenyx制药(RARE.US) $Vir Biotechnology(VIR.US) $Vertiv(VRT.US) $Nebius(NBIS.US) $Arm(ARM.US)

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