Ionis' Rare Development Disorder Drug Candidate 'Could Be Differentiated' From Peer Ultragenyx: Analyst
Ionis' Rare Development Disorder Drug Candidate 'Could Be Differentiated' From Peer Ultragenyx: Analyst
Ionis Pharmaceuticals Inc (NASDAQ:IONS) released results Monday from the completed multiple ascending dose (MAD) portion of the Phase 1/2 open-label study of ION582 in people with Angelman syndrome (AS).
愛奧尼斯製藥公司（納斯達克股票代碼：IONS）週一公佈了針對安格爾曼綜合症（AS）ION582 的1/2期開放標籤研究中已完成的多重遞增劑量（MAD）部分的結果。
AS is a rare neurodevelopmental disorder caused by a loss of function in the maternal UBE3A gene. It affects an estimated 1 in 21,000 people worldwide.
AS 是一種罕見的神經發育障礙，由母體 UBE3A 基因功能喪失引起。據估計，全球每21,000人中就有1人受到影響。
The data demonstrated consistent and encouraging clinical improvement on assessing all functional domains, including communication, cognition, and motor function measures.
數據顯示，在評估所有功能領域（包括溝通、認知和運動功能測量）方面，臨床表現持續且令人鼓舞。
Also Read: Ionis Pharmaceuticals Reveals Late-Stage Donidalorsen Data, Analyst Says Results Show Competitive Hereditary Angioedema Profile.
另請閱讀：分析師稱愛奧尼斯製藥公司公佈了多尼達洛森的後期數據，結果顯示遺傳性血管性水腫狀況具有競爭力。
Overall, 97% of people in the medium and high dose groups saw an improvement in overall AS symptoms.
總體而言，在中、高劑量組中，97% 的人的整體AS症狀有所改善。
In the 51-patient HALOS study, ION582 showed favorable safety and tolerability at all dose levels.
在 51 名患者 HALOS 研究中，ION582 在所有劑量水平下均顯示出良好的安全性和耐受性。
Ionis plans to meet with regulators later this year to review and confirm its Phase 3 study design. This puts the company on track for a pivotal study initiation in the first half of 2025.
Ionis計劃在今年晚些時候與監管機構會面，審查並確認其3期研究設計。這使該公司有望在2025年上半年啓動一項關鍵研究。
William Blair writes that the update is a clear positive for Ionis, positioning ION582 as a key asset in its wholly owned portfolio after recently reacquiring sole rights from Biogen Inc (NASDAQ:BIIB).
威廉·布萊爾寫道，此次更新對愛奧尼斯來說顯然是一個積極的利好消息，在最近從百健公司（納斯達克股票代碼：BIIB）手中重新收購了獨資權後，將 ION582 定位爲其全資投資組合中的關鍵資產。
William Blair reiterates the Outperform rating on Ionis.
威廉·布萊爾重申了對愛奧尼斯的跑贏大盤評級。
The analyst notes that the consistent improvements across various metrics in the robust dataset, which will likely be used as a primary endpoint for approval, boost confidence in the drug's potential benefits.
該分析師指出，強大的數據集中各種指標的持續改進增強了人們對該藥物潛在益處的信心，該數據集可能被用作批准的主要終點。
While slightly behind a peer program from Ultragenyx Pharmaceutical Inc (NASDAQ:RARE), Ionis has largely closed the development timeline gap and could be differentiated from a safety perspective, making ION582 an attractive asset for future growth given lack of approved therapies for the indication currently.
儘管略微落後於Ultragenyx Pharmaceutical Inc（納斯達克股票代碼：RARE）的同行計劃，但愛奧尼斯在很大程度上縮小了開發時間差距，從安全角度來看，可以區分開來，這使得 ION582 成爲未來增長的有吸引力的資產，因爲目前該適應症缺乏批准的療法。
In the meantime, investors are expected to focus on the launch of Wainua (eplontersen) in collaboration with AstraZeneca Plc (NASDAQ:AZN), and the potential approval and launch of the wholly-owned therapies olezarsen for familial chylomicronemia syndrome (PDUFA December 19, 2024) and donidalorsen for the prophylactic treatment of HAE (NDA expected to be filed this year).
同時，預計投資者將重點關注與阿斯利康公司（納斯達克股票代碼：AZN）合作推出的Wainua（eplontersen），以及治療家族性乳糜微粒血癥綜合徵的全資療法olezarsen（PDUFA，2024年12月19日）和用於HAE預防性治療的多尼達洛森（預計將於今年提交保密協議）的可能批准和推出年）。
The analyst believes the next 12 to 18 months could be transformative for Ionis due to the expansion of its marketed products. They also consider the company undervalued, especially in light of the potential of TTR silencers for TTR-cardiomyopathy, following Alnylam Pharmaceuticals Inc's (NASDAQ:ALNY) positive HELIOS-B trial results for vutrisiran.
這位分析師認爲，由於其上市產品的擴張，未來12至18個月可能會對Ionis產生變革。他們還認爲該公司的估值被低估了，特別是考慮到在Alnylam製藥公司（納斯達克股票代碼：ALNY）對伏特利西蘭的Helios-B試驗結果呈陽性之後，TTR消音器有可能治療TTR心肌病。
Price Action: IONS stock is up 4.50%, trading at $49.97 at last check Monday.
價格走勢：IONS股票上漲4.50％，週一最後一次檢查時交易價格爲49.97美元。
- Tale Of Two Ionis-Partnered Neurology-Focused Early Stage Assets-Biogen Discontinues One, Decides Not To Exercise Licensing Option For Another.
- Tale Of Two Ionis合作的專注於神經病學的早期資產——Biogen停產了一家資產，決定不對另一家資產行使許可權。