Protalix BioTherapeutics, Inc. a yau ta sanar da sakamako na ƙarshe daga gwaji na asibiti na BRIGHT Phase III wanda ke kimanta pegunigalsidase alfa (PRX 102) don yuwuwar maganin cutar Fabry. Sakamakon ya nuna cewa jiyya tare da 2 mg / kg na PRX-102 da aka gudanar ta hanyar intravenous (IV) a kowane mako hudu yana da kyau a yi haƙuri, kuma cutar Fabry da aka kiyasta ta hanyar ƙwanƙwasa glomerular filtration rate (eGFR) da kuma plasma lyso-Gb3 maida hankali ya tabbata.

"Muna farin cikin raba bayanan karshe daga binciken BRIGHT, muhimmin ci gaba a ci gaban shirin mu na asibiti na PRX-102," in ji Dror Bashan, Shugaban Protalix da Babban Jami'in Gudanarwa. "Samun wannan bayanan don sake dubawa ta Cibiyar Abinci da Magunguna ta Amurka, Hukumar Kula da Magunguna ta Turai da sauran masu kula da ita wani mataki ne na gaba ga amincewar da ake tsammani na PRX-102 a matsayin mafi kyawun madadin ga manya Fabry marasa lafiya a cikin duka 1 MG na yau da kullun. \kg kowane mako biyu da kuma 2 mg \ kg kowane mako hudu tsarin.

PRX-102 shine mai bayyana tantanin halitta mai sake hadewa, PEGylated, mai haɗin giciye α galactosidase A ɗan takarar samfur. Gwajin asibiti na BRIGHT Phase III (NCT03180840) ya kasance cibiyar sadarwa da yawa, alamar buɗewa ta ƙasa da ƙasa, binciken canzawa da aka tsara don kimanta amincin, inganci da magunguna na jiyya tare da 2 mg / kg na PRX-102 ana gudanarwa kowane mako huɗu don makonni 52 ( jimlar 14 infusions). Binciken ya sanya 30 balagagge marasa lafiya da cutar Fabry (24 maza da 6 mata) tare da ma'ana (SD) shekaru 40.5 (11.3) shekaru, jere daga 19 zuwa 58 shekaru, wanda a baya samu wani yarda enzyme maye far (ERT) a kalla. shekaru uku akan tsayayyen kashi ana gudanarwa kowane mako biyu (agalsidase alfa - Replagal® ko agalsidase beta - Fabrazyme®). Mafi yawan alamun cutar Fabry a asali sune acroparesthesia, rashin haƙuri na zafi, angiokeratomas da hypohydrosis.

Duk marasa lafiya da suka shiga cikin binciken sun sami akalla kashi ɗaya na PRX-102, kuma marasa lafiya 29 sun kammala binciken. Daga cikin waɗannan marasa lafiya na 29, 28 sun karbi tsarin da aka yi nufi na 2 mg / kg na PRX-102 kowane mako hudu a duk tsawon binciken, yayin da aka canza mai haƙuri zuwa 1 mg / kg na PRX-102 kowane mako biyu a kowace yarjejeniya a jiko na 11th. . Ɗaya daga cikin majiyyaci ya janye daga binciken bayan jiko na farko saboda hadarin mota.

An gudanar da jiko na farko na PRX-102 a ƙarƙashin yanayin sarrafawa a wurin bincike. Dangane da ƙayyadaddun ƙayyadaddun ƙayyadaddun ƙayyadaddun ƙayyadaddun ƙa'idodin binciken, marasa lafiya sun sami damar karɓar jigon su na PRX-102 a saitin kulawar gida da zarar Mai Binciken da Mai Kula da Kula da Lafiya ya yarda cewa yana da lafiya don yin hakan.

Gabaɗaya, 33 na 182 jimlar jiyya-gaggawa mara kyau (TEAEs) da aka ruwaito a cikin marasa lafiya tara (30.0%) an ɗauke su da alaƙa da jiyya; duk sun kasance masu sauƙi ko matsakaici a cikin tsanani kuma yawancin an warware su a ƙarshen binciken. Babu TEAE mai tsanani ko mai tsanani da ke da alaƙa da magani kuma babu TEAE da ya haifar da mutuwa ko janyewar karatu. Daga cikin TEAE da ke da alaƙa da jiyya, 27 sun kasance halayen da ke da alaƙa da jiko (IRRs) kuma sauran abubuwan guda ɗaya ne na zawo, erythema, gajiya, rashin lafiya kamar mura, haɓakar furotin / creatinine na fitsari, da fitsari tabbatacce ga ƙwayoyin farin jini. An ba da rahoton 27 IRR a cikin marasa lafiya biyar (16.7%), duk maza. Duk IRRs sun faru a lokacin jiko ko a cikin sa'o'i biyu bayan jiko; ba a yi rikodin abubuwan da suka faru tsakanin sa'o'i biyu zuwa 24 bayan jiko ba. Babu ɗaya daga cikin marasa lafiya da ba tare da ƙwayoyin rigakafin ƙwayoyi ba (ADAs) a wurin nunin ADAs da suka haifar da jiyya bayan canzawa zuwa jiyya na PRX-102.

Ma'auni na sakamakon binciken ya nuna cewa plasma lyso Gb3 maida hankali ya kasance barga a lokacin binciken tare da ma'anar canji (± SE) na 3.01 nM (0.94) daga asali (19.36 nM ± 3.35) zuwa mako 52 (22.23 ± 3.60 nM). Ma'anar madaidaicin ƙimar eGFR ɗin sun tsaya tsayin daka yayin lokacin jiyya na mako 52, tare da ma'anar canji daga tushe na 1.27 mL/min/1.73 m2(1.39). Ma'ana (SE) eGFR gangara, a ƙarshen binciken, ga yawan jama'a, shine 2.92 (1.05) mL / min / 1.73m2 / shekara yana nuna kwanciyar hankali.

"Muna farin cikin sanar da sakamakon ƙarshe daga gwajin gwaji na BRIGHT Phase III kuma muna so mu gode wa masu binciken binciken, marasa lafiya na Fabry, da iyalansu waɗanda suka sadaukar da lokacinsu da ƙoƙarinsu ga wannan gagarumin bincike," in ji Giacomo Chiesi, shugaban Chiesi. Cututtukan da ba kasafai ba a Duniya. "Bisa ga waɗannan bayanan da ƙarin nazarin asibiti, mun yi imanin PRX-102 na iya zama wani muhimmin zaɓi na sabon magani ga marasa lafiya waɗanda ke karɓar infusions na ERT a kowane mako biyu, kuma muna sa ran ci gaba da aikinmu a duniya don samun amincewar doka kamar yadda ya kamata. da sauri da kuma ba da dama ga al'ummar cutar Fabry."

ABUBUWAN DA ZA KU GUDU DAGA WANNAN LABARI:

Gudanar da Abinci da Magunguna, Hukumar Kula da Magunguna ta Turai da sauran masu kula da ita wani mataki ne na gaba ga amincewar da ake tsammanin na PRX-102 a matsayin mafi kyawun zaɓi ga manya Fabry marasa lafiya a cikin 1 MG na yau da kullun kowane mako biyu da kuma 2 MG. \kg kowane mako hudu tsarin.
Daga cikin waɗannan marasa lafiya na 29, 28 sun karbi tsarin da aka yi nufi na 2 mg / kg na PRX-102 kowane mako hudu a duk tsawon binciken, yayin da aka canza mai haƙuri zuwa 1 mg / kg na PRX-102 kowane mako biyu ta hanyar yarjejeniya a jiko na 11th. .
Gwajin asibiti na BRIGHT Phase III (NCT03180840) ya kasance cibiyar sadarwa da yawa, alamar buɗewa ta ƙasa-da-ƙasa, binciken canzawa da aka tsara don kimanta aminci, inganci da magunguna na jiyya tare da 2 mg / kg na PRX-102 ana gudanarwa kowane mako huɗu don makonni 52 ( jimlar 14 infusions).