Dario Eklund, Santhera CEO (file photo)

San­thera kicks idebenone DMD pro­gram to the curb af­ter PhI­II tri­al fails in­ter­im analy­sis

An oft-trou­bled pro­gram at San­thera Phar­ma­ceu­ti­cals {SWX: $SANN} is head­ed for the chop­ping block.

The Swiss biotech an­nounced Tues­day morn­ing that it is dis­con­tin­u­ing a Phase III study for idebenone in pa­tients with Duchenne mus­cu­lar dy­s­tro­phy and re­ceiv­ing steroid treat­ment. In a re­lease, San­thera said that the tri­al failed an in­ter­im analy­sis, and as a re­sult will pull its EMA ap­pli­ca­tion and end glob­al de­vel­op­ment.

San­thera will al­so re­struc­ture its busi­ness plans to fo­cus on a sep­a­rate DMD can­di­date, va­morolone.

“While this is ob­vi­ous­ly not the out­come we ex­pect­ed, all our ef­forts in DMD will now be fo­cused on pro­gress­ing the promis­ing drug can­di­date va­morolone, which we re­cent­ly li­censed from Rever­a­Gen to its next in­flec­tion point,” CEO Dario Ek­lund said in a state­ment.

With the news, the biotech’s stock price cratered more than 43% as the Swiss stock ex­change opened, but has since lev­eled off to falling about 25 to 30%.

Idebenone was de­signed to im­prove lung func­tion in pa­tients by en­er­giz­ing weak­ened mus­cle cells, and the pri­ma­ry end­point for the Phase III tri­al sought to demon­strate an im­prove­ment in de­lay­ing the loss of res­pi­ra­to­ry func­tion af­ter 18 months. Though there were no safe­ty con­cerns not­ed by the tri­al in­ves­ti­ga­tors, the like­li­hood of meet­ing that end­point was deemed too small to mer­it con­tin­u­ing the tri­al. San­thera has not shared ex­act­ly what da­ta prompt­ed the shut­down.

Tues­day marks the end of an era as idebenone faced sev­er­al set­backs in its even­tu­al road to nowhere. San­thera orig­i­nal­ly ap­plied for US ap­proval back in 2016, but the FDA swat­ted down its ap­pli­ca­tion and asked for a con­fir­ma­to­ry tri­al — the tri­al which has now been ter­mi­nat­ed. At the time, that move proved con­sis­tent with the agency’s his­to­ry of pump­ing the brakes on com­pa­nies like Sarep­ta seek­ing swift OKs in the DMD field.

But idebenone al­so ran in­to trou­ble in Eu­rope when reg­u­la­tors 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.

With a re­newed fo­cus on va­morolone, San­thera is not giv­ing up its DMD ef­forts. The com­pound is cur­rent­ly in a Phase IIb tri­al look­ing to mea­sure im­prove­ment in time to stand over base­line af­ter 24 weeks. This tri­al en­rolled DMD pa­tients that are not tak­ing steroid treat­ments, and San­thera says va­morolone has the po­ten­tial to be an al­ter­na­tive to such ther­a­pies al­to­geth­er by slow­ing dis­ease progress with­out steroidal side ef­fects.

In ear­ly Sep­tem­ber, San­thera picked up glob­al rights to va­morolone af­ter sign­ing on to an op­tion from Rever­a­Gen and Idor­sia in 2018.

The re­struc­tur­ing will al­so al­low San­thera to fo­cus on lon­ode­le­stat for cys­tic fi­bro­sis and oth­er lung dis­eases, as well as its dis­cov­ery-stage gene ther­a­py ap­proach for con­gen­i­tal mus­cu­lar dy­s­tro­phy.

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.

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.

Emer Cooke, EMA director (AP Photo/Geert Vanden Wijngaert)

Ahead of FDA, EMA rec­om­mends au­tho­riz­ing new gene ther­a­py treat­ment for ul­tra-rare dis­ease

Aromatic amino acid decarboxylase (AADC) deficiency is an ultra-rare genetic disease that leaves patients unable to produce certain hormones in the brain, such as dopamine and serotonin, usually leading to developmental delays, weak muscle tone and inability to control the movement of the limbs. It can also lead to multiple organ failure.

To date, there have been no treatments approved for AADC deficiency, which has been identified in less than 150 patients.

Ather­sys tries to post-hoc-an­a­lyze its way out of an­oth­er tri­al fail for stroke stem cell ther­a­py

Athersys’ stem cell therapy has failed yet again.

In a 206-person trial conducted in Japan, Athersys’ stem cell therapy for stroke failed its primary endpoint of “excellent outcome,” a combined measure of three stroke recovery scores.

While a greater percentage of patients in the treatment group reached the primary endpoint compared to placebo, that difference was not statistically significant.

Siddhartha Mukherjee (Brian Ach/Getty Images for The New Yorker)

All Blue's $733M bid to ac­quire Zymeworks turns hos­tile as board bat­tles back — af­ter a biotech celebri­ty jumps in

Yesterday, the team at All Blue Capital — bent on the takeover of a badly battered Zymeworks — brought in celebrated oncologist, Pulitzer prize-winning writer and biotech exec Siddhartha Mukherjee to add some glitz to their proposed board. But they’re still not winning over any converts.

This morning, Zymeworks’ board officially turned this acquisition offer into a hostile showdown, rejecting the unsolicited offer and marshaling its forces to prevent a buyout at $10.50 per share.

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