Pavan Cheruvu, Sio Gene Therapies CEO

Sio Gene Ther­a­pies posts pos­i­tive PhI/II da­ta in rare pe­di­atric dis­ease, the first read­out since its name change

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.”

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Even as public health officials remain guarded about their comments on the likelihood Omicron will escape the reach of the currently approved Covid-19 vaccines, there’s growing scientific consensus that we’re facing a variant that threatens to overwhelm the vaccine barricades that have been erected.

Stéphane Bancel, the CEO of Moderna, one of the leading mRNA players whose quick vault into the markets with a highly effective vaccine created an instant multibillion-dollar market, added his voice to the rising chorus early Tuesday. According to Bancel, there will be a significant drop in efficacy when the average immune system is confronted by Omicron. The only question now is: How much?

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Philip Dormitzer, new GSK global head of vaccines R&D

Glax­o­SmithK­line poach­es Pfiz­er's vi­ral vac­cines lead in rush to cap­i­tal­ize on fu­ture of mR­NA

GlaxoSmithKline has appointed Philip Dormitzer, formerly chief scientific officer of Pfizer’s viral vaccines unit, as its newest global head of vaccines R&D, looking to leverage one of the leading minds behind Pfizer and BioNTech’s RNA collaboration that led to Covid-19 jab Comirnaty, the British drug giant said Tuesday.

Dormitzer had been with Pfizer for a little more than six years, joining up after a seven-year stint with Novartis, where he reached the role of US head of research and head of global virology for the company’s vaccines and diagnostics unit.

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In­tro­duc­ing End­points Stu­dio, a new way to ad­ver­tise with End­points-craft­ed brand­ing cam­paigns

Since our start in 2016, Endpoints has grown fast while executing our mission to cover biopharma’s most critical developments for industry pros worldwide. As readership has grown, our advertising business has too. Endpoints advertising partners support the mission and engage their desired audiences through announcements on our email and web platforms, brand recognition in our event coverage and sponsorships of Endpoints daily and weekly reports.

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Barely a month after disappointing data shattered hopes for a major label expansion for the GI tumor drug Qinlock, Deciphera is making a major pivot — scrapping development plans for that drug and discarding another while it hunkers down and focuses on two remaining drugs in the pipeline.

As a result, 140 of its staffers will be laid off.

The restructuring, which claims the equivalent of 35% of its total workforce, will take place across all departments including commercial, R&D as well as general and administrative support functions, Deciphera said, as it looks to streamline Qinlock-related commercial operations in the US while concentrating only on a “select number of key European markets.”

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How to use reg­istry da­ta to sup­port FDA de­ci­sion mak­ing: Agency ex­plains in new guid­ance

Drugmakers looking to design a new registry or use an existing one to support a regulatory decision on a drug’s effectiveness or safety will need to consult with a new draft guidance released Monday by the FDA.

The agency’s reliance on registry data for regulatory decisions dates back more than two decades, at least, as in 1998 Bayer won approval for its anticoagulant Refludan (withdrawn from the market in 2013 for commercial reasons) based in part on a historical control group pulled from a registry.

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Tillman Gerngross, the rarely shy Dartmouth professor, biotech entrepreneur and antibody expert, has been warning for over a year that the virus behind Covid-19 would likely continue to mutate, potentially in ways that avoid immunity from infection and the best defenses scientists developed. He spun out a company, Adagio, to build a universal antibody, one that could snuff out any potential mutation.

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The chronic and painful skin condition results in lumps and abscesses caused by inflammation or infection of sweat glands, most often in the armpits or groin. Humira was first approved to treat HS in 2015 and remains the only FDA-approved drug for the condition. Two TV ads both note more than 30,000 people with HS have been prescribed Humira.