San­thera ax­es half its em­ploy­ees and sees CMO de­part af­ter PhI­II DMD fail

Less than a month af­ter show­ing its lead Duchenne mus­cu­lar dy­s­tro­phy pro­gram out the door, San­thera Phar­ma­ceu­ti­cals is slash­ing half its staff.

Kristi­na Sjöblom Ny­gren

The Swiss biotech an­nounced the move Mon­day morn­ing as it con­tin­ues its re­struc­tur­ing around a sec­ond DMD can­di­date, va­morolone. Among the em­ploy­ees de­part­ing is San­thera’s now-for­mer CMO Kristi­na Sjöblom Ny­gren, though the com­pa­ny not­ed that she left for “fam­i­ly rea­sons and to pur­sue oth­er op­por­tu­ni­ties,” per a state­ment.

San­thera added that the re­duc­tions will save rough­ly $10.9 mil­lion in an­nu­al costs, fol­low­ing a one-time charge of about $3.26 mil­lion. In to­tal, San­thera is cut­ting more than 50 po­si­tions, bring­ing its to­tal work­force to 47 full-time equiv­a­lent em­ploy­ees.

Va­morolone be­came the com­pa­ny’s pri­ma­ry fo­cus af­ter San­thera re­port­ed that the oth­er DMD pro­gram, idebenone, failed a Phase III in­ter­im analy­sis last month. The next da­ta, a 6-month read­out in a piv­otal Phase IIb tri­al, is due some­time in the sec­ond quar­ter of 2021. If all goes well, San­thera said, an NDA could come as soon as next year’s fourth quar­ter.

That tri­al is mea­sur­ing im­prove­ment in time to stand over base­line af­ter 24 weeks, en­rolling DMD pa­tients that are not tak­ing steroid treat­ments. San­thera pre­vi­ous­ly not­ed that va­morolone has the po­ten­tial to be an al­ter­na­tive to such ther­a­pies al­to­geth­er by slow­ing dis­ease progress with­out steroidal side ef­fects. The drug it­self is a steroid de­signed to have sim­i­lar ef­fects as the cor­ti­cos­teroids Duchenne pa­tients cur­rent­ly take.

Va­morolone’s most re­cent da­ta comes from a Phase IIa back in Au­gust 2019, which hit the pri­ma­ry end­point in show­ing sta­tis­ti­cal­ly sig­nif­i­cant and pro­por­tion­al im­prove­ments from base­line on time to stand from supine.

San­thera said it ex­pects to be the first to mar­ket with a dis­so­cia­tive steroid in the US in 2022, ac­quir­ing glob­al rights to the ex­per­i­men­tal drug in Sep­tem­ber af­ter first sign­ing on two years ago. Orig­i­nal­ly de­vel­oped by Rever­a­Gen, va­morolone was ac­quired fol­low­ing an op­tion agree­ment with Acte­lion, now known as Idor­sia, in 2016. Idor­sia then sold the op­tion to San­thera in 2018.

Idebenone had pre­vi­ous­ly been held up as one of San­thera’s more promis­ing pro­grams, but it had of­ten run in­to trou­ble be­fore be­ing axed last month. De­signed to im­prove lung func­tion in pa­tients by en­er­giz­ing weak­ened mus­cle cells, idebenone was orig­i­nal­ly sub­mit­ted for FDA ap­proval back in 2016, but reg­u­la­tors were not im­pressed. The agency asked for a con­fir­ma­to­ry tri­al to prove ef­fi­ca­cy — the tri­al that ul­ti­mate­ly flopped in Oc­to­ber.

But idebenone al­so ran in­to trou­ble in Eu­rope when the EMA shot down a la­bel ex­pan­sion in­to DMD in 2017 and an ap­peal in 2018. Ap­proved on the con­ti­nent as Rax­one to treat Leber’s hered­i­tary op­tic neu­ropa­thy, the drug al­so failed an NIH-backed mul­ti­ple scle­ro­sis tri­al in March 2018 af­ter show­ing no dif­fer­ence from a place­bo.

In ad­di­tion to the re­struc­tur­ing around va­morolone, San­thera is al­so de­vel­op­ing lon­ode­le­stat for cys­tic fi­bro­sis and oth­er lung dis­eases.

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Illustration: Assistant Editor Kathy Wong for Endpoints News

As mon­ey pours in­to dig­i­tal ther­a­peu­tics, in­sur­ance cov­er­age crawls



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As the 10-year-old zooms through icy waters and targets flying creatures on the snow-capped planet Frigidus, she builds attention skills, thanks to Akili Interactive Labs’ video game EndeavorRx. She’s now less anxious and scattered, allowing her to stay on a low dose of ADHD medication, according to her mom Violet Vu.

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Eli Lil­ly’s Alzheimer’s drug clears more amy­loid ear­ly than Aduhelm in first-ever head-to-head. Will it mat­ter?

Ahead of the FDA’s decision on Eli Lilly’s Alzheimer’s drug donanemab in February, the Big Pharma is dropping a first cut of data from one of the more interesting trials — but less important in a regulatory sense — at an Alzheimer’s conference in San Francisco.

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Matt Gline, Roivant Sciences CEO (Photo by John Sciulli/Getty Images for GLG)

Pfiz­er and Roivant team up again for an­oth­er 'Van­t', set­ting up an­ti-in­flam­ma­to­ry show­down with Prometheus

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There’s no name for the startup yet, nor are there any employees. Thus far, the new company and Roivant can be considered “one and the same,” Roivant CEO Matt Gline tells Endpoints News. But Pfizer is so enthusiastic about the target that it elected to keep 25% of equity in the drug rather than take upfront cash from Roivant, Gline said.

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Roche HQ in Basel, Switzerland. (Image credit: Kyle LaHucik/Endpoints News)

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Paul Hudson, Sanofi CEO (Romuald Meigneux/Sipa via AP Images)

Sanofi and DN­Di aim to elim­i­nate sleep­ing sick­ness in Africa with promis­ing Ph II/III re­sults for new drug

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Lynn Baxter, Viiv Healthcare's head of North America

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