A lit­tle over a month af­ter a full com­pa­ny re­brand, the Biotech For­mer­ly Known as Ax­o­vant has its first da­ta read­out un­der its new moniker.

Tues­day’s in­ter­im look comes from Sio Gene Ther­a­pies’ GM1 gan­gliosi­do­sis pro­gram, where the ex-Vant says it saw pos­i­tive safe­ty and ef­fi­ca­cy out­comes in a small Phase I/II tri­al. The re­sults mark what CEO Pa­van Cheru­vu hopes can pro­vide a new foun­da­tion in rare pe­di­atric dis­eases as Sio seeks to move past an epic Alzheimer’s fail just a few years ago.

“This is tru­ly a cor­ner­stone of the ap­proach we hope to take go­ing for­ward as we con­sid­er pipeline de­vel­op­ment and ex­pan­sion,” Cheru­vu told End­points News, “and is re­al­ly the first in­stance where we see promis­ing new da­ta com­ing out at, ad­mit­ted­ly, an ear­ly time point, but that we think builds up­on the strong sci­en­tif­ic foun­da­tion that we’ve put in place at Sio.”

In­vestors took the news in stride, with Sio $SIOX shares shoot­ing up about 45% af­ter the clos­ing bell Tues­day.

There are two types of GM1 gan­gliosi­do­sis, an in­her­it­ed ge­net­ic dis­ease that falls un­der the lyso­so­mal stor­age dis­or­der um­brel­la. If left un­treat­ed, the lack of a key en­zyme al­lows for waste ma­te­r­i­al to build up in cells and cause their ear­ly de­struc­tion.

The pro­gram, called AXO-AAV-GM1, is an AAV9-based gene ther­a­py Sio ac­quired back in De­cem­ber 2018 from the Uni­ver­si­ty of Mass­a­chu­setts Med­ical School as it was un­der­go­ing its shift in strat­e­gy. De­signed to in­tro­duce func­tion­al copies of the mu­tat­ed GLB1 gene, the can­di­date aims to re­verse the en­zyme de­fi­cien­cy, pre­vent­ing the pro­gres­sive death of cells over time.

Sio’s re­sults come from the six-month fol­low-up pe­ri­od for the low-dose co­hort of the tri­al, which en­rolled five pa­tients be­tween 32 and 68 months old. Cheru­vu was main­ly look­ing for good safe­ty re­sults as, giv­en the rare na­ture of the dis­ease, these were the first pa­tients dosed with a gene ther­a­py of any kind, he said. Sio found no se­ri­ous treat­ment-re­lat­ed side ef­fects and the ther­a­py was well-tol­er­at­ed.

In terms of ef­fi­ca­cy, Sio looked at the change in serum en­zyme ac­tiv­i­ty from base­line, or how con­cen­trat­ed an en­zyme is with­in a pa­tient’s cells, af­ter six months. The five pa­tients saw an av­er­age in­crease of 110%, which amount­ed to a restora­tion of 38% of nor­mal en­zyme ac­tiv­i­ty. Cheru­vu said that in oth­er sim­i­lar stor­age dis­eases, like Tay-Sachs and Sand­hoff dis­eases, restor­ing any­where from 10% to 20% typ­i­cal­ly cor­re­lates with long-term ben­e­fits.

“[These dis­eases] act as prox­ies for GM1 gan­gliosi­do­sis; they’re linked by an un­der­ly­ing bio­chem­i­cal and cel­lu­lar path­way,” Cheru­vu said.

With its first da­ta as Sio, the biotech will con­tin­ue to push AXO-AAV-GM1 through the dose es­ca­la­tion pe­ri­od and ul­ti­mate­ly in­to a larg­er tri­al. Sio has al­ready dosed the first two pa­tients in the high­er dose co­hort and is hop­ing to see safe­ty and ef­fi­ca­cy lev­els main­tained in the low-dose through the next read­out at the 12-month mark.

The re­sults al­so mark some­thing of a win for Cheru­vu, who took on the re­spon­si­bil­i­ty of clean­ing up the Alzheimer’s mess de­spite join­ing the com­pa­ny af­ter the fall­out. When Sio an­nounced its re­brand­ing last month, the CEO pro­claimed, “We’re not a vant any longer,” em­pha­siz­ing that Vivek Ra­maswamy’s Roivant biotech smor­gas­bord was no longer a ma­jor­i­ty stake­hold­er (it still owns a rough­ly 30% stake in the com­pa­ny and holds two board seats).

Sio’s pe­di­atrics pro­grams are part of a two-pronged fo­cus — Cheru­vu al­so not­ed the com­pa­ny is look­ing at some high-risk, high-re­ward can­di­dates in Parkin­son’s. And though AXO-AAV-GM1 is still in its ear­ly stages, Cheru­vu is con­fi­dent in the ther­a­py’s po­ten­tial.

“As we build the pro­gram, we hope to have a more ex­pe­dit­ed ap­proval process around this prod­uct,” Cheru­vu said. “We know there’s a pre­dictable nat­ur­al his­to­ry of de­cline in these kids, and if we can main­tain sta­bil­i­ty, main­tain mile­stones that have al­ready been gained, that could be a ba­sis for a sig­nif­i­cant dis­cus­sion with reg­u­la­tors.”

CRLs. 483s. CBER, CDER and RWE. For biopharma professionals, these acronyms command attention because of the fundamental role FDA plays in drug development. Now Endpoints is doubling down on regulatory coverage, and launching a weekly report focusing on developments out of White Oak, with analysis and insight into what it all means.

Coverage will be led by our new senior editor, Zachary Brennan. He joins Endpoints from POLITICO, where he covered pharma. Prior to that he was the managing editor for Regulatory Focus, a news publication from the Regulatory Affairs Professionals Society.

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