Larimar Therapeutics' New Drug Could Transform Treatment For Rare Neurological Disease, Analyst Sees Over 200% Upside For Stock
Larimar Therapeutics' New Drug Could Transform Treatment For Rare Neurological Disease, Analyst Sees Over 200% Upside For Stock
Oppenheimer has initiated coverage on Larimar Therapeutics (NASDAQ:LRMR), a clinical-stage biotechnology company focused on developing treatments for rare diseases.
Oppenheimer已經開始對larimar therapeutics(納斯達克:LRMR)進行覆蓋,這是一家專注於爲罕見疾病開發治療方案的臨床生物技術公司。
The analysts view the company's lead program, Nomlabofusp, as a differentiated, disease-modifying therapy that targets the key pathogenic mechanism of frataxin (FXN) deficiency in Friedreich's ataxia (FA).
分析師們認爲該公司的主導項目Nomlabofusp是一種獨特的、疾病修正療法,針對弗雷德雷希氏共濟失調(FA)中frataxin(FXN)缺陷的關鍵致病機制。
Lower FXN levels predict early onset, disease severity, and faster progression.
較低的FXN水平預示着早發病、疾病嚴重程度和快速進展。
Friedreich's ataxia is a rare neurodegenerative disease affecting ~20,000 patients globally.
弗雷德雷希氏共濟失調是一種罕見的神經退行性疾病,全球約有20,000名患者受影響。
Also Read: Larimar Therapeutics' Friedreich's Ataxia Investigational Drug Differentiated From Biogen's Marketed Drug.
更多閱讀:larimar therapeutics的弗雷德雷希氏共濟失調調查藥物與渤健公司市售藥物有所區別。
Oppenheimer expects open-label extension (OLE) interim data in the fourth quarter of 2024.
Oppenheimer預計於2024年第四季度公佈開放標籤擴展(OLE)中期數據。
Larimar intends to expand into the pediatric population and pursue accelerated approval with a potential FDA marketing application submission in the second half of 2025 under the FDA's START Pilot Program.
larimar打算擴展到兒童群體,並計劃在2025年下半年根據FDA的STARt試點計劃進行潛在的FDA營銷申請提交以加快批准過程。
Oppenheimer has initiated with an Outperform rating and a price target of $26.
Oppenheimer給予larimar therapeutics超越市場預期的評級和26美元的目標股價。
The analysts assume a 60% chance of accelerated approval, modeling the U.S. launch in 2027 and the European launch in 2028 for FA, estimating $1.2 billion in global revenues in 2030.
分析師假設加速批准的可能性爲60%,預計FA在2027年在美國上市,2028年在歐洲上市,預計2030年全球收入達12億美元。
Following early 2023 approval, Skyclarys, developed by Reata and acquired by Biogen Inc (NASDAQ:BIIB) for $7.3 billion in 2023, has generated ~$278 million in global sales.
在2023年初獲批後,由Reata開發並被渤健公司(納斯達克: BIIB)以73億美元收購的Skyclarys已在全球銷售額達到約2.78億美元。
Oppenheimer says that Nomlabofusp stands out from its competitors due to its potential to modify the disease.
Oppenheimer表示,Nomlabofusp憑藉其改變疾病潛力脫穎而出。
Although Reata/Biogen's Skyclarys has received approval for patients aged over 16 with FA and PTC Therapeutics Inc's (NASDAQ:PTC) vatiquinone is approved for patients over 7, Larimar's strategy to include pediatric and adolescent FA patients in its Phase 2 OLE trial enables it to address a larger patient demographic and tackle the progressive aspects of the disease.
儘管Reata/Biogen的Skyclarys已獲得16歲以上FA患者的批准,PTC Therapeutics Inc的(NASDAQ: PTC) vatiquinone已獲得7歲以上患者的批准,但Larimar在其第二階段OLE試驗中包括兒童和青少年FA患者的策略使其能夠面對更廣泛的患者群體並應對疾病的進展方面。
Price Action: LRMR stock is up 2.56% at $7.61 at last check Wednesday.
LRMR股價在週三最後一次檢查時上漲了2.56%,報7.61美元。
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Photo: Shutterstock
Photo: shutterstock
譯文內容由第三人軟體翻譯。