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.

Op­ti­miz­ing Cell and Gene Ther­a­py De­vel­op­ment and Pro­duc­tion: How Tech­nol­o­gy Providers Like Corn­ing Life Sci­ences are Spurring In­no­va­tion

Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

Amid mon­key­pox fears, biotechs spring to ac­tion; Mod­er­na’s CFO trou­ble; Cuts, cuts every­where; Craft­ing the right pro­teins; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

It’s always a bittersweet moment saying goodbye, but as Josh Sullivan goes off to new adventures we are grateful for the way he’s built up the Endpoints Manufacturing section — which the rest of the team will now carry forward. If you’re not already, this may be a good time to sign up for your weekly dose of drug manufacturing news. Thank you for reading and wish you a restful weekend.

Bay­er sounds re­treat from a $670 mil­lion CAR-T pact in the wake of a pa­tient death

Two months after Atara Biotherapeutics hit the hold button on its lead CAR-T 2.0 therapy following a patient death, putting the company under the watchful eye of the FDA, its Big Pharma partners at Bayer are bowing out of a $670 million global alliance. And the move is forcing a revamp of Atara’s pipeline plans, even as research execs vow to continue work on the two drugs allied with Bayer 18 months ago, which delivered a $60 million cash upfront.

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Try­ing to shake up the Parkin­son's par­a­digm, Ab­b­Vie sub­mits NDA for con­tin­u­ous, 24-hour in­fu­sion ther­a­py

AbbVie is approaching the FDA with a new therapy to potentially treat Parkinson’s disease, using prodrugs of two medications commonly used for the condition.

The Big Pharma submitted its NDA for ABBV-951, a solution of levodopa and carbidopa prodrugs being evaluated in advanced Parkinson’s patients who don’t respond well to oral therapy, AbbVie announced Friday morning. Researchers are hoping a positive Phase III study that reads out in late October will help move things along quickly at the agency.

Martin Shkreli (Dennis Van Tine/MediaPunch/IPX)

In­fa­mous biotech ex­ec Mar­tin Shkre­li gets out of prison, hits the street

Martin Shkreli, the infamous biotech CEO who made headlines for his jeering assault on a legion of critics in and out of Congress, is back on the streets after 4 years inside a federal penitentiary.

Shkreli’s attorney put out a statement Wednesday afternoon saying that the “pharma bro” had been transferred to a halfway house in New York with a few more months to go under federal custody, slated to end September 14. Attorney Benjamin Brafman acknowledged the release and vowed that he and Shkreli are keeping quiet.

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Laurent Levy, Nanobiotix co-founder and CEO (via Nanobiotix)

UP­DAT­ED: An­oth­er biotech that went pub­lic in pan­dem­ic boom aims to weath­er the bear mar­ket through R&D cut­backs

The bear market is showing no signs of letting up, with a French biotech that IPO’ed during the pandemic boom feeling the heat.

Nanobiotix, a French biotech focusing on new treatments for cancer, is cutting back on preclinical research and amending some of its ongoing studies to stay afloat, the company said in its first quarter report late Wednesday night. The cuts are aimed at prioritizing its lead program for advanced head and neck squamous cell carcinoma, Nanobiotix said.

Sanofi and Re­gen­eron clear the fin­ish line in an in­flam­ma­to­ry esoph­a­gus dis­ease, leav­ing Take­da in the dust

With atopic dermatitis rivals breathing down Dupixent’s neck, Sanofi and Regeneron on Friday secured a first win in new territory in what Sanofi’s head of immunology and inflammation Naimish Patel called the fastest approval he’s ever seen.

The FDA approved Dupixent on Friday to treat patients 12 years and older with eosinophilic esophagitis (EoE), an inflammatory condition that causes swelling and scarring of the esophagus. The approval came just a couple months after regulators granted Dupixent priority review, and months ahead of its PDUFA date on Aug. 3.

Fu­ji­film con­tin­ues its biotech build­ing spree with new fa­cil­i­ty in Chi­na

A Japanese conglomerate is making a big play in China with the opening of a new facility, as it continues to expand.

Fujifilm Irvine Scientific has opened its new Innovation and Collaboration Center in Suzhou New District, China, an area in Jiangsu province specifically designated for technological and industrial development.

According to Fujifilm, the 12,000-square-foot site will be responsible for the company’s cell culture media optimization, analysis and design services. Cell culture media itself often requires customization of formulas and protocols to achieve the desired quantity and quality of therapeutic desired. Fujifilm Irvine Scientific is offering these services from its headquarters in California and Japan to its customers globally, as well as in China now.

Stéphane Bancel, Moderna CEO (AP Photo/Charles Krupa)

French pres­i­dent names Stéphane Ban­cel a Cheva­lier for Mod­er­na's Covid tri­umph

Moderna’s rapid fire development of its hugely successful mRNA vaccine for Covid saved lives, changed the vaccine industry forever and made CEO Stéphane Bancel a billionaire. But perhaps the sweetest reward came this week, when Bancel was named a Chevalier — basically knighted — by the president of France.

Prestigious European titles like this are rare in biopharma, but not unknown, as AstraZeneca’s Mene Pangalos could tell you after being knighted by the Queen, named on the honors list in 2020 for his contribution to science.