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.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

Alaa Halawaa, executive director at Mubadala’s US venture group

The ven­ture crew at Mubadala are up­ping their biotech cre­ation game, tak­ing care­ful aim at a new fron­tier in drug de­vel­op­ment

It started with a cup of coffee and a slow burning desire to go early and long in the biotech creation business.

Wrapping up a 15-year discovery stint at Genentech back in the summer of 2021, Rami Hannoush was treated to a caffeine-fueled review of the latest work UCSF’s Jim Wells had been doing on protein degradation — one of the hottest fields in drug development.

“Jim and I have known each other for the past 15 years through Genentech collaborations. We met over coffee, and he was telling me about this concept of the company that he was thinking of,” says Hannoush. “And I got immediately intrigued by it because I knew that this could open up a big space in terms of adding a new modality in drug discovery that is desperately needed in pharma.”

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'Band­ing to­geth­er': 50 fe­male biotech ex­ec­u­tives lay out plans for board di­ver­si­ty, new com­pa­nies and men­tor­ing founders

Earlier this month, during the Silicon Valley Bank meltdown, Angie You recalled the speed with which female biotech CEOs were helping each other connect with bankers, get their wires through and assuage concerns during a financial implosion.

This past weekend, 50 of about 125 women who are part of that Slack group and a broader coalition self-dubbed the Biotech Sisterhood met in person in Cancun for the second rendition of an annual summit connecting female biotech CEOs. The attendance list doubled that of the inaugural gathering in Arizona 12 months ago.

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Rohan Palekar, 89bio CEO

89bio’s PhII da­ta add to quick suc­ces­sion of NASH read­outs as field seeks turn­around

89bio said its drug was better than placebo at lessening fibrosis without worsening nonalcoholic steatohepatitis, or NASH, in two of three dose groups.

The San Francisco biotech said it thinks the Phase IIb data pave the way for a potential Phase III, following in the footsteps of another biotech in its drug class, Akero Therapeutics. To fund a late-stage study, CEO Rohan Palekar told Endpoints News 89bio “would need to raise additional capital,” with the company having about $188 million at the end of last year.

FDA in­di­cates will­ing­ness to ap­prove Bio­gen ALS drug de­spite failed PhI­II study

Ahead of Wednesday’s advisory committee hearing to discuss Biogen’s ALS drug tofersen, the FDA appeared open to approving the drug, newly released briefing documents show.

Citing the need for flexibility in a devastating disease like ALS, regulators signaled a willingness to consider greenlighting tofersen based on its effect on a certain protein associated with ALS despite a failed pivotal trial. The documents come after regulatory flexibility was part of the same rationale the agency expressed when approving an ALS drug last September from Amylyx Pharmaceuticals, indicating the FDA’s openness to approving new treatments for the disease.

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Roche and Lil­ly team up to de­vel­op blood test to de­tect ear­ly signs of Alzheimer's

Eli Lilly is teaming up with Roche to help develop a blood test to detect early signs of Alzheimer’s disease and determine whether a patient should go for further confirmatory testing.

Roche’s Elecsys Amyloid Plasma Panel (EAPP) measures pTau 181 protein assay and APOE E4 assay in human blood plasma – elevations in pTau 181 are present in the early stages of Alzheimer’s, while the presence of APO E4 is the most common genetic risk factor for the disease.

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Flare Therapeutics biochemists Yong Li (L) and Valerie Vivat

A $123M Flare will get Third Rock on­col­o­gy biotech in­to the clin­ic this year

Flare Therapeutics will start its first human trial this year with an investigational urothelial cancer drug after pulling together a $123 million Series B from Big Pharmas, VCs and its incubator, Third Rock Ventures.

Launched in 2021 on the idea that a biotech could finally succeed at drugging the much-sought-after but stubborn transcription factor, Flare Therapeutics said Wednesday it is now primed for the clinic after closing its large financing haul earlier this year. The raise is a relatively stark figure in a tough startup financing environment but further buoys the upbeat signals coming out of other Third Rock biotechs in recent weeks, including the $200 million CARGO Therapeutics and $100 million Rapport Therapeutics rounds.

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Francesco Marincola, newly-appointed Sonata Therapeutics CSO

Kite's head of re­search leaves for Flag­ship start­up Sonata

Another leader is departing Kite Pharma, and will to spend the “last part” of his career exploring how cancer evades the immune system.

Kite’s senior VP and global head of cell therapy research Francesco Marincola left the Gilead CAR-T unit last week for Sonata Therapeutics. Flagship last May unveiled the startup, which was pieced together from two fledgling biotechs Inzen and Cygnal Therapeutics. As CSO, Marincola will lead Sonata’s push to reprogram cancer cells to make them more immunogenic.

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Vipin Garg, Altimmune CEO

Al­tim­mune’s shares halved af­ter in­ter­im look at PhII weight loss drug da­ta

Altimmune’s attempt to catch up to Novo Nordisk and Eli Lilly’s GLP-1 drugs hit an investor snag Tuesday after the biotech shared interim Phase II weight loss data.

The Maryland biotech’s pemvidutide is a GLP-1/glucagon dual receptor agonist meant to activate GLP-1 receptors to squash appetite and glucagon to ramp up energy use. The 2.4 mg dose showed a placebo-adjusted weight loss of 9.7% at week 24 of 48, which Jefferies analysts said would be comparable to Novo Nordisk’s semaglutide (Wegovy) and Eli Lilly’s tirzepatide (Mounjaro).

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