Dario Eklund, Santhera

Months af­ter ax­ing a Duchenne pro­gram and lay­ing off dozens, San­thera claims a win there with a dif­fer­ent pro­gram

Last fall, San­thera Phar­ma­ceu­ti­cals un­cer­e­mo­ni­ous­ly dumped a Duchenne pro­gram af­ter it flunked a Phase III tri­al, forc­ing a wave of lay­offs that trimmed the biotech’s staff from 120 to 40. Now, the Swiss com­pa­ny be­lieves it’s found a path to a come­back with a dif­fer­ent can­di­date in Duchenne.

San­thera re­vealed topline re­sults from a Phase IIb study for its va­morolone pro­gram, say­ing two dif­fer­ent dos­es each hit the pri­ma­ry end­point com­pared with place­bo. Tues­day’s da­ta give the biotech con­fi­dence to po­ten­tial­ly seek ap­proval for both dosage lev­els with the aim of sub­mit­ting an NDA in the first quar­ter of next year.

“This was the last thing that need­ed to fall in­to place for us to be, what I’m col­lo­qui­al­ly call­ing, San­thera 2.0,” CEO Dario Ek­lund told End­points News. “It’s re­al­ly a new start for the com­pa­ny.”

The news sent San­thera shares sky­rock­et­ing in the Swiss stock ex­change more than 60%.

Back in Oc­to­ber, San­thera was forced to halt a Phase III study for its now-de­funct idebenone pro­gram af­ter it failed an in­ter­im analy­sis. The com­pa­ny had been ramp­ing up for a po­ten­tial com­mer­cial launch through­out Eu­rope and had to with­draw its mar­ket­ing au­tho­riza­tion ap­pli­ca­tion from the EMA.

Most of the staffers laid off in the sub­se­quent re­struc­tur­ing were sta­tioned over­seas in more than half a dozen Eu­ro­pean mar­kets, Ek­lund said. The launch had been slat­ed for the first quar­ter of this year, but the re­sult­ing fail­ure forced San­thera to strip “down to the bones.”

Idebenone was de­signed to im­prove lung func­tion in pa­tients by en­er­giz­ing weak­ened mus­cle cells, and had long faced road­blocks in its de­vel­op­ment. The FDA re­ject­ed its orig­i­nal ap­pli­ca­tion for the pro­gram back in 2016 and asked for a con­fir­ma­to­ry tri­al — the tri­al that San­thera ter­mi­nat­ed last fall.

But the va­morolone can­di­date func­tions sim­i­lar to a steroid, which is the stan­dard of care for many Duchenne pa­tients. The Phase IIb study ex­am­ined dosage lev­els of 2 mg/kg per day and 6 mg/kg per day, look­ing at how 121 pa­tients’ time to stand from a supine po­si­tion was short­ened af­ter 24 weeks of treat­ment.

In the high dose, pa­tients re­duced their time to stand from 6 sec­onds to 4.6 sec­onds, while the place­bo group went from 5.4 to 5.5 sec­onds. That equat­ed to a sparkling p-val­ue of p=0.002. San­thera didn’t re­veal the time dif­fer­ence for the low­er dose, but re­port­ed a p-val­ue here of p=0.02. There were “sub­tle dif­fer­ences” in the dose re­spons­es, va­morolone pro­gram lead Shabir Hasham told End­points.

Ide­al­ly, San­thera wants both dos­es to be ap­proved so that pa­tients have the op­tion of down-titrat­ing from the high­er lev­el if they ex­pe­ri­ence side ef­fects. There were no treat­ment-re­lat­ed side ef­fects that reg­is­tered at grade 3 or high­er, but Hasham said about one-third of tri­al par­tic­i­pants in the high­er dose group moved down to the low­er dose.

“Pa­tients can start on a high­er-dose ther­a­py, and those who are com­fort­able can stay while oth­ers come down and don’t lose the ef­fi­ca­cy,” Hasham said. “That’s re­al­ly the unique thing.”

Con­ver­sa­tions with reg­u­la­tors will dic­tate how San­thera ul­ti­mate­ly moves for­ward, Ek­lund said. He hopes that be­cause the can­di­date is an oral so­lu­tion rather than a pill, San­thera’s pitch will prove per­sua­sive — par­tic­u­lar­ly giv­en how typ­i­cal cor­ti­cos­teroid treat­ments used for Duchenne can be down-titrat­ed in a sim­i­lar man­ner.

Last Sep­tem­ber, San­thera picked up glob­al rights to va­morolone from Rever­a­Gen af­ter sign­ing on to the op­tion a few years ago. The com­pa­ny is look­ing to aug­ment the Phase IIb da­ta with re­sults from a pre­vi­ous­ly-re­port­ed Phase IIa study, which Hasham said now shows safe­ty and tol­er­a­bil­i­ty of the pro­gram over 30 months.

Big Phar­ma's Twit­ter ex­o­dus; Mer­ck wa­gers $1.35B on buy­out; $3.5M gene ther­a­py; 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.

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Paul Perreault, CSL Behring CEO

CSL lands FDA ap­proval for he­mo­phil­ia B gene ther­a­py, sets $3.5M list price

The FDA has approved the world’s first gene therapy for hemophilia B, ushering into the market a treatment that’s historic in both what it promises to do and how much it will cost.

CSL will be marketing the drug, Hemgenix, at a list price of $3.5 million — which sets a new record for the most expensive single-use gene therapy in the US.

In a statement provided to Endpoints News, the Australian company noted that the current costs of treating people with moderate to severe hemophilia B can be significant over a lifetime. By some estimates, healthcare systems could spend more than $20 million per person.

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Image: Shutterstock

MIT re­searchers re­veal DNA "Paste" tech be­hind lat­est gene edit­ing start­up

MIT scientists have developed a tool that they say can insert large gene sequences where they want in the genome.

In a paper published Thursday in Nature Biotechnology, MIT fellows Omar Abudayyeh, Jonathan Gootenberg and colleagues detail a technology they call PASTE, which they say can potentially be used to insert long strands of DNA and treat genetic diseases caused by many different mutations, such as cystic fibrosis and Leber congenital amaurosis, a rare eye disorder that causes blindness.

Elon Musk (GDA via AP Images)

Biggest drug com­pa­nies halt­ed Twit­ter ad buys af­ter Lil­ly in­sulin spoof

Almost all of the drug industry’s biggest advertisers cut their spending on Twitter to zero or near-zero over the last two weeks amid worries about impersonation of their brands by pranksters and the future of the social media company.

Among 18 of the biggest pharmaceutical advertisers in the US market, 12 cut their Twitter ad spending to nothing for the week beginning Nov. 14, according to Pathmatics, which tracks data on prescription drug ad spending as well as general corporate advertising. The list of drugmakers cutting spending to zero includes Merck, AstraZeneca, Eli Lilly, Novartis, Pfizer and others.

J&J's Spra­va­to pulls a PhI­II win against Sero­quel XR in treat­ment-re­sis­tant de­pres­sion

A day before Thanksgiving, J&J’s Janssen has a new cut of Phase III Spravato data to be grateful for.

The pharma giant announced on Wednesday that its nasal spray, also known as esketamine, beat extended-release quetiapine, previously sold by AstraZeneca as Seroquel XR, in treatment-resistant depression (TRD). Of 676 adults, a significantly higher number of patients on Spravato were able to achieve remission and avoid relapse after 32 weeks, according to J&J.

Rob Davis, Merck CEO

Up­dat­ed: No Seagen here: 'Do more' means a small $1.35B pur­chase of Ima­go for Mer­ck

Merck is making an acquisition, the Big Pharma announced before Monday’s opening bell. No, Seagen is not entering the fold, as had been speculated for quarters.

Folding under Merck’s wings will be Pfizer-backed Imago BioSciences. For nearly a year, Merck CEO Rob Davis has been saying the pharma giant needs to “do more” on the business development front after its 2021 $11.5 billion acquisition of Acceleron.

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Isao Teshirogi, Shionogi president and CEO (Kyodo via AP Images)

Sh­ionogi's Covid an­tivi­ral lands first ap­proval in Japan's new emer­gency ap­proval path­way

Japanese regulators on Tuesday signed off on Shionogi’s homegrown antiviral for Covid-19, known as Xocova (ensitrelvir), making it the first approval under Japan’s emergency regulatory approval system.

The emergency approval, following a back-and-forth with regulators since last February, is based on a safety profile with more than 2,000 patients who have accessed the pill, and clinical symptomatic efficacy for five typical Omicron-related symptoms (primary endpoint) and antiviral efficacy (key secondary endpoint) in patients with mild to moderate SARS-CoV-2 infection, regardless of risk factors or vaccination status, and during the Omicron-dominant phase of the pandemic.

Dermavant Sciences' first consumer TV ad for its Vtama psoriasis med shows people ready for a new topical treatment.

Roivant’s Der­ma­vant de­buts first-ever TV com­mer­cial for pso­ri­a­sis cream Vta­ma

Dermavant Sciences has been marketing its first product, psoriasis med Vtama, to dermatologists for months, but on Tuesday it rolled out its first consumer campaign. The debut DTC effort including a streaming TV commercial encourages patients to a “Topical Uprising” in a nod to Vtama being a topical cream.

In the new commercial, a swell of people discards scarves and jacket coverings, gathering in the street to converge on a pharmacy to demand a steroid-free prescription. A moment of levity follows when a pharmacist says, “You know you can just talk to your doctor, right?” The gathered crowds collectively says, “Oh.”

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FDA preps for DMD drug gener­ics as Sarep­ta has yet to fin­ish its con­fir­ma­to­ry tri­al

The FDA typically releases guidance to help generic drug manufacturers develop new copycats of small molecule drugs, oftentimes in preparation for a brand name product’s patents or exclusivity to expire.

This week, FDA released such bioequivalence guidance for any generic drugmakers looking to take on Sarepta’s Duchenne muscular dystrophy (DMD) drug Exondys 51 (eteplirsen), even though the drug’s sponsor has yet to convert the accelerated approval to a full approval, showing clinical benefit.