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.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

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