QURE Tshuag Rockets Tom qab FDA pom zoo nws cov tshuaj kim tshaj plaws puas tau

Food and Drug Administration nyuam qhuav pom zoo nws cov tshuaj kim tshaj plaws puas tau - hemophilia B noob kev kho mob los ntawm Carlisle tuam txhab uas muag (CSL) thiab UniQure (QURE) - thiab QURE Tshuag tau tshwm sim hnub Wednesday.

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Meanwhile, BioMarin (BMRN) kev sib koom tau tawg tom qab FDA tau hais tias nws yuav tsis tuav lub rooj sib tham pawg neeg tawm tswv yim los tham txog nws txoj kev kho hemophilia A noob. Cov kws pab tswv yim no ua cov lus pom zoo tsis cuam tshuam rau FDA tom qab sib tham txog cov txiaj ntsig thiab kev pheej hmoo ntawm kev sim tshuaj. BioMarin's Roctavian twb tau pom zoo nyob hauv Europe.

On hnub no lub khw lag luam, QURE Tshuag dhia 14.6% rau 26.36. Qhov ntawd txuas ntxiv cov khoom lag luam khiav los ntawm 7.1% nce rau hnub Tuesday. Shares topped ib muas point ntawm 25.91 tawm ntawm kev sib sau ua ke, raws li MarketSmith.com.

BioMarin Tshuag tau dhia 7.3% rau 97.79. Qhov ntawd tau thawb BioMarin Tshuag kom tawg tawm ntawm ib qho ob-hauv qab puag nrog kev nkag ntawm 92.86.

QURE Tshuag: Tus nqi yog tshaj qhov kev cia siab

UniQure's infusion tau pom zoo rau cov neeg mob hemophilia B tam sim no nyob rau hauv kev tiv thaiv kev tiv thaiv hu ua Factor IX therapy, lossis leej twg muaj keeb kwm ntawm qhov muaj peev xwm ua rau tuag taus lossis los ntshav hnyav. Ntawm tus nqi ib zaug ntawm $ 3.5 lab, Carlisle thiab UniQure vam tias cov tshuaj yuav kho tau.

Cov kws tshuaj ntsuam hais tias qhov kev pom zoo yog qhov xwm txheej zoo tshaj plaws rau UniQure, lub tuam txhab uas pib tsim Hemgenix ua ntej koom tes nrog Carlisle nyob rau theem tom ntej. Daim ntawv lo tsis txwv txoj kev kho rau ib lub pas dej tshwj xeeb ntawm cov neeg mob lossis suav nrog "lub thawv dub" ceeb toom.

"Vim qhov tsis paub meej txog kev pom zoo ntawm hemophilia noob kev kho mob thiab kev ceeb toom rau cov tub lag luam, peb xav tias qhov no yog qhov zoo rau UniQure thiab qhov tseem ceeb tsis txaus ntshai rau zaj dab neeg," UBS tus kws tshuaj ntsuam Eliana Merle tau hais hauv tsab ntawv ceeb toom hnub Wednesday.

Tus nqi $ 3.5 lab yog siab dua qhov kev pom zoo los ntawm Lub Tsev Haujlwm Saib Xyuas Kev Kho Mob thiab Kev Lag Luam. Cov nonprofit hais tias feem ntau hemophilia noob kev kho mob yuav raug nqi $2.5 lab. Daim ntawv tshaj tawm tau tshuaj xyuas tus nqi ntawm $ 2 lab txog $ 3 lab raws li cov thawj coj cov lus pom.

SVB Securities kws tshuaj ntsuam Joseph Schwartz tau hais tias tus nqi siab tshaj qhov kev cia siab yuav suav rau cov neeg mob hemophilia B me me. Tseem, nws pom qhov kev pom zoo yog qhov zoo rau cov neeg mob uas nquag los ntshav thiab xav tau "kev kho mob hnyav."

Cov neeg mob yuav raug saib xyuas rau peb teev tom qab Hemgenix infusion. Tom qab ntawd, lawv yuav tsum tau soj ntsuam txhua lub lim tiam rau peb lub hlis txhawm rau saib xyuas cov teeb meem ntawm daim siab. Schwartz sau ntawv tiv thaiv kev kho mob nrog Factor IX feem ntau yog muab txhua lub lim tiam.

"Peb ntseeg tias cov kev saib xyuas no ua rau muaj kev nkag siab zoo thiab yuav raug suav tias yog kev txi tsim nyog rau qhov yuav muaj kev ywj pheej los ntawm kev kho mob, los ntshav thiab kev puas tsuaj rau lub sijhawm ntev," nws hais hauv tsab ntawv ceeb toom.

Nws muaj qhov ua tau zoo tshaj plaws ntawm QURE Tshuag.

UniQure's Lwm Yam Kev Siv Tshuaj Kho Mob

Raws li cov lus pom zoo, Carlisle yuav yog lub luag haujlwm rau kev lag luam Hemgenix. Hauv kev xa rov qab, UniQure yuav tau txais txog li $ 1.5 nphom hauv kev them nyiaj tseem ceeb ntxiv thiab muaj nuj nqis ntawm kev muag khoom hauv nruab nrab mus rau qis-20%. UniQure kuj muaj cai tsim khoom.

Schwartz tau hais tias "Peb pom (qhov) kev pom zoo yog qhov kev pheej hmoo tseem ceeb rau UniQure lub peev xwm nyiaj txiag yav tom ntej," Schwartz tau hais. "Peb kuj xav tias qhov no muaj txiaj ntsig zoo rau kev pom zoo rau BioMarin's Roctavian hauv hemophilia A."

Qhov kev pom zoo kuj tseem yuav qiv kev lees paub rau UniQure kev siv zog hauv lwm qhov chaw kho noob caj noob ces. Cov tub ua lag luam tej zaum yuav ceev faj ntau ntawm lub tuam txhab kev ua haujlwm hauv Huntington tus kab mob. Peb ntawm 14 tus neeg mob hauv UniQure txoj kev tshawb fawb tau ntsib cov kev mob tshwm sim loj.

"Peb nco ntsoov Theem 1 thiab Theem 2 hloov tshiab tseem raug coj mus rau lub quarter thib ob ntawm 2023 thiab tuaj yeem yog cov ntaub ntawv tseem ceeb rau txoj haujlwm nrog thawj cov ntaub ntawv ntawm kev ua haujlwm (cov hom phiaj)," Merle, tus kws tshuaj ntsuam UBS tau hais.

Nws tau yuav qhov ntsuas ntawm QURE Tshuag thiab tsa nws lub hom phiaj rau 42 ntawm 40.

Ua raws li Allison Gatlin ntawm Twitter ntawm @IBD_AGatlin.

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