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.

Illustration: Assistant Editor Kathy Wong for Endpoints News

How Pur­due's $272M ad­dic­tion pay­out fund­ed a new home for its dis­card­ed non-opi­oid re­search

Don Kyle spent more than 20 years working for Purdue Pharma, right through the US opioid epidemic that led to the company’s rise and eventual infamy. But contrary to Purdue’s focus on OxyContin, Kyle was researching non-opioid painkillers — that is, until the company shelved his research.

As the company’s legal troubles mounted, Kyle found an unlikely way to reboot the project. In 2019, he took his work to an Oklahoma State University center that’s slated to receive more than two-thirds of the state’s $272 million settlement with Purdue over claims that the drugmaker’s behavior ignited the epidemic of opioid use and abuse.

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President Joe Biden at the State of the Union address with Vice President Kamala Harris and House Speaker Kevin McCarthy (Patrick Semansky/AP Images)

The drug pric­ing pres­i­dent: Biden warns of ve­to for any IRA re­peal at­tempts

President Joe Biden made clear in his “finish the job” State of the Union address last night that one of those jobs to be finished is insulin prices.

Biden’s push again to tackle insulin prices, after Republicans rebuffed the idea last summer and just after Biden won Medicare drug price negotiations/caps via the Inflation Reduction Act, shows how heavily he’s leaning into this work.

Rupert Vessey, Bristol Myers Squibb head of research and early development

Up­dat­ed: R&D tur­bu­lence at Bris­tol My­ers now in­cludes the end of a $650M al­liance and the de­par­ture of a top re­search cham­pi­on

This morning biotech Dragonfly put out word that Bristol Myers Squibb has handed back all rights to its IL-12 clinical-stage drug after spending $650 million to advance it into the clinic.

The news arrives amid a turbulent R&D stage for the pharma giant, which late last week highlighted Rupert Vessey’s decision to depart this summer as head of early-stage R&D following a crucial three-year stretch after he jumped to Bristol Myers in the big Celgene buyout. During that time he struck a series of deals for Bristol Myers, and also shepherded a number of Celgene programs down the pipeline, playing a major role for a lineup of biotechs which depended on him to champion their drugs.

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Utpal Koppikar, new Verily CFO

Ex­clu­sive: Ver­i­ly wel­comes Atara Bio­ther­a­peu­tics vet­er­an as new CFO

Verily, Alphabet’s life sciences outfit, has plucked a new CFO from the ranks of Atara Biotherapeutics, the company announced on Wednesday.

Utpal Koppikar joins Verily after a nearly five-year stint as CFO and senior VP at Atara, though his résumé also boasts roles at Gilead and Amgen.

The news follows a major reshuffling at Verily, including several senior departures earlier this year and a round of layoffs.

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Singer Nick Jonas is back at work for Dexcom, this time for its new G7 glucose monitor.

Dex­com's spokescelebri­ty Nick Jonas re­turns to Su­per Bowl in new glu­cose mon­i­tor com­mer­cial

Dexcom is going back to the Super Bowl with its pop singer and patient spokesperson Nick Jonas. Jonas takes center stage as the lone figure in the 30-second commercial showcasing Dexcom’s next-generation G7 continuous glucose monitoring (CGM) device.

Jonas’ sleight-of-hand tricks populate the commercial — he pinches his empty fingers together and pops them open to reveal the small CGM — even as he ends the ad, saying, “It’s not magic. It just feels that way.” Jonas then disappears in a puff of smoke.

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Richard Francis, newly-appointed Teva CEO (Novartis via Facebook)

New Te­va CEO Richard Fran­cis repri­or­i­tizes to 'get back to growth'

Six weeks into his new role at the helm of Teva Pharmaceutical, Richard Francis said it’s time to “get back to growth,” starting with a good look at the company’s priorities.

The chief executive has kicked off a strategic review, he announced during Teva’s quarterly call, which will continue over the next several months and produce results sometime in the middle of 2023. That means some pipeline cuts may be in store, he told Endpoints News, while declining to offer much more detail.

Bill Haney, Dragonfly CEO (Dave Pedley/Getty Images for SXSW)

Drag­on­fly chief: Bris­tol My­ers shouldn’t blame IL-12’s clin­i­cal per­for­mance for de­ci­sion to scrap the deal — eco­nom­ics played a key role

Bristol Myers Squibb says the IL-12 drug they were developing out of Dragonfly Therapeutics was scrubbed from the pipeline for a simple reason: It didn’t measure up on clinical performance.

But Bill Haney, the CEO of Dragonfly, is taking issue with that.

The early-stage drug, still in Phase I development, has passed muster with Bristol Myers’ general clinical expectations, advancing successfully while still in Phase I, he says.

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FDA Commissioner Robert Califf on Capitol Hill, Feb. 8, 2023 (Drew Angerer/Getty Images)

FDA com­mis­sion­er floats ideas on how to bet­ter han­dle the pan­dem­ic

FDA Commissioner Rob Califf joined the heads of the CDC and NIH in the hot seat today before a key House subcommittee, explaining that there needs to be a much faster, more coordinated way to oversee vaccine safety, and that foreign biopharma inspections, halted for years due to the pandemic, are slowly ramping up again.

Califf, who stressed to the House Energy and Commerce’s Subcommittee on Health that the CDC also needs better data, made clear that the FDA’s ability to monitor the safety of vaccines “would also benefit greatly by a coordinated federal public health data reporting authority.”

Sanofi is renewing its #VaccinesForDreams campaign with more stories, such as Juan's in Argentina (Sanofi)

Sanofi re­news so­cial cam­paign to re­mind that vac­cines let peo­ple ‘Dream Big’

Sanofi is highlighting people’s dreams — both big and small — to make the point that vaccines make them possible.

The renewed “Dream Big” global social media campaign’s newest dreamer is Juan, a teacher in the Misiones rainforest in Argentina whose story is told through videos on Instagram and Sanofi’s website with the hashtag #VaccinesForDreams.

The campaign ties to Sanofi’s broader umbrella initiative “Vaccine Stories” to promote the value of vaccines and drive awareness of the need for improved vaccination coverage.

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