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.

Cyclerion Therapeutics may have the design of a Phase IIb study ready to go, but it’s scrambling for a way to fund it.

The company said in a press release that it’s “actively evaluating the best combination of capital, capabilities, and transactions available to it to advance the development of zagociguat,” its lead candidate for a rare, genetic mitochondrial disease known as MELAS.

In a separate SEC filing, Cyclerion once again flagged “substantial doubt about (its) ability to continue as a going concern.” As of the end of 2022, it had cash and cash equivalents of only $13.4 million.

A small, Santa Ana-based biotech created in 2017 is looking to enter a SPAC deal as it lays out plans to begin trials in its lead cell therapy candidates and bring on new executives.

Graf Acquisition Corp. IV and NKGen Biotech announced Thursday, with few other details, that the two companies signed a non-binding letter of intent to “pursue a business combination.” Graf Acquisition II and III withdrew their IPOs last year.

The FDA has rejected Incyte’s extended-release formulation of ruxolitinib tablets, in a surprise setback for the company’s plans to build on its blockbuster Jakafi franchise.

The ruxolitinib XR tablets are designed to be taken once a day, whereas Jakafi is indicated for twice daily dosage (although some patients can take it once daily).

According to Incyte, the FDA acknowledged in its complete response letter that the study submitted in the NDA “met its objective of bioequivalence based on area under the curve (AUC) parameters but identified additional requirements for approval.”