UK reg­u­la­tors green-light ear­ly ac­cess to San­thera’s Rax­one for Duchenne MD

Thomas Meier, CEO of San­thera

San­thera Phar­ma­ceu­ti­cals saw its shares {SIX: $SANN} jump 16% to­day af­ter the Swiss biotech re­port­ed that UK reg­u­la­tors will pro­vide ear­ly ac­cess to their drug Rax­one (idebenone) for use in fight­ing symp­toms of Duchenne mus­cu­lar dy­s­tro­phy.

British reg­u­la­tors say that the da­ta in­di­cate that this drug can pro­vide a longterm ben­e­fit for boys ex­pe­ri­enc­ing res­pi­ra­to­ry de­cline and not on or able to take glu­co­cor­ti­coids. They al­so en­dorsed the safe­ty pro­file of the drug.

That all sounds like an en­cour­ag­ing pre­lude to a CHMP de­ci­sion on this drug, which is on the mar­ket as a treat­ment for Leber’s Hered­i­tary Op­tic Neu­ropa­thy (LHON), a rare mi­to­chon­dr­i­al dis­ease. And Matthew Eck­ler at RBC says it bodes well for San­thera, in­di­cat­ing an in­creased like­li­hood of an ap­proval.

Notes Eck­ler:

Re­call that last month San­thera an­nounced a de­lay in the ex­pect­ed tim­ing of a CHMP rec­om­men­da­tion for Rax­one la­bel ex­pan­sion to Q3 vs. late Q1/ear­ly Q2 pri­or, as a re­sult of a sup­ple­men­tary in­for­ma­tion re­quest. We con­tin­ue to view this de­lay as a wrin­kle in the sto­ry, rather than fun­da­men­tal­ly al­ter­ing our the­sis. We ex­pect a pos­i­tive CHMP rec­om­men­da­tion and la­bel ex­pan­sion to DMD giv­en: 1) Rax­one is the on­ly DMD ther­a­py with a pos­i­tive Phase III tri­al; 2) CHMP re­newed Translar­na’s mar­ket­ing au­tho­riza­tion fol­low­ing fail­ure of its con­fir­ma­to­ry study; and 3) Rax­one is al­ready mar­ket­ed in the EU for LHON.

“We’re proud to re­ceive the pos­i­tive EAMS sci­en­tif­ic opin­ion for Rax­one in the UK and to have our lead com­pound des­ig­nat­ed as a promis­ing in­no­v­a­tive med­i­cine, the first for a drug in­tend­ed for the treat­ment of DMD,” said Thomas Meier, the CEO of San­thera, in a state­ment.

IDC: Life Sci­ences Firms Must Em­brace Dig­i­tal Trans­for­ma­tion Now

Pre-pandemic, the life sciences industry had settled into a pattern. The average drug took 12 years and $2.9 billion to bring to market, and it was an acceptable mode of operations, according to Nimita Limaye, Research Vice President for Life Sciences R&D Strategy and Technology at IDC.

COVID-19 changed that, and served as a proof-of-concept for how technology can truly help life sciences companies succeed and grow, Limaye said. She recently spoke about industry trends at Egnyte’s Life Sciences Summit 2022. You should watch the entire session, free and on-demand, but here’s a brief recap of why she’s urging life sciences companies to embrace digital transformation.

Bayer's first DTC ad campaign for chronic kidney disease drug Kerendia spells out its benefits

Bay­er aims to sim­pli­fy the com­plex­i­ties of CKD with an ABC-themed ad cam­paign

Do you know the ABCs of CKD in T2D? Bayer’s first ad campaign for Kerendia tackles the complexity of chronic kidney disease with a play on the acronym (CKD) and its connection to type 2 diabetes (T2D).

Kerendia was approved last year as the first and only non-steroidal mineralocorticoid receptor antagonist to treat CKD in people with type 2 diabetes.

In the TV commercial launched this week, A is for awareness, B is for belief and C is for cardiovascular, explained in the ad as awareness of the connection between type 2 and kidney disease, belief that something can be done about it, and cardiovascular events that may be reduced with treatment.

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James Mock, incoming CFO at Moderna

Mod­er­na taps new CFO from PerkinElmer af­ter for­mer one-day CFO oust­ed

When Moderna hired a new CFO last year,  it didn’t expect to see him gone after only one day. Today the biotech named his — likely much more vetted — replacement.

The mRNA company put out word early Wednesday that after the untimely departure of then brand-new CFO Jorge Gomez, it has now found a replacement in James Mock, the soon-to-be former CFO at diagnostics and analytics company PerkinElmer.

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Tom Barnes, Orna Therapeutics CEO

UP­DAT­ED: 'We have failed to fail': Mer­ck gam­bles $250M cash on a next-gen ap­proach to mR­NA — af­ter punt­ing its big al­liance with Mod­er­na

Merck went in deep on its collaboration with Moderna on new mRNA programs, and dropped them all over time, including their RSV partnership. But after writing off what turned out as one of the most successful infectious disease players in the business, Merck is coming in this morning with a new preclinical alliance — this time embracing a biotech that hopes to eventually outdo the famously successful mRNA in a new run at vaccines and therapeutics.

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Joe Jonas (Photo by Anthony Behar/Sipa USA)(Sipa via AP Images)

So­lo Jonas broth­er car­ries Merz's new tune in Botox ri­val cam­paign

As the lyrics of his band’s 2019 pop-rock single suggest, Joe Jonas is only human — and that means even he gets frown lines. The 33-year-old singer-songwriter is Merz’s newest celebrity brand partner for its Botox rival Xeomin, as medical aesthetics brands target a younger audience.

Merz kicked off its “Beauty on Your Terms” campaign on Tuesday, featuring the Jonas brother in a video ad for its double-filtered anti-wrinkle injection Xeomin.

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Paul Perreault, CSL Behring CEO

CSL CEO Paul Per­reault de­ter­mined to grow plas­ma col­lec­tion af­ter full-year sales dip

As the ink dries on CSL’s $11.7 billion Vifor buyout, the company posted a dip in profits, due in part to a drop in plasma donations amid the pandemic.

However, CEO Paul Perreault assured investors and analysts on the full-year call that the team has left “no stone unturned” when assessing options to grow plasma volumes. The chief executive also spelled out positive results for the company’s monoclonal antibody garadacimab in hereditary angioedema (HAE), though he isn’t revealing the exact numbers just yet.

Blaise Coleman, Endo International CEO

En­do files for Chap­ter 11 as it looks to fin­ish off its opi­oid lit­i­ga­tion

Irish drugmaker Endo International is entering into bankruptcy as it faces the weight of serious litigation related to its involvement in the opioid epidemic in the US.

The company has filed Chapter 11 proceedings in the US Bankruptcy Court for the Southern District of New York, with the company expected to file recognition proceedings in Canada, the UK and Australia. The company’s bankruptcy filing showed the company had assets and liabilities in the range of $1 billion to $10 billion.

Etleva Kadilli, director of UNICEF’s supply division

GSK lands first-ever UNICEF con­tract for malar­ia vac­cine worth $170M

GSK has landed a new first from UNICEF the first-ever contract for malaria vaccines, worth up to $170 million for 18 million vaccine doses distributed over the next three years.

The vaccine, known as Mosquirix or RTS,S, won WHO’s backing last October after a controversial start, but UNICEF said these doses will potentially save thousands of lives every year.

“We hope this is just the beginning,” Etleva Kadilli, director of UNICEF’s supply division, said. “Continued innovation is needed to develop new and next-generation vaccines to increase available supply, and enable a healthier vaccine market. This is a giant step forward in our collective efforts to save children’s lives and reduce the burden of malaria as part of wider malaria prevention and control programmes.”

Governor John Carney, Lieutenant Governor Bethany Hall-Long, Mayor Kenneth Branner, Jr., Delaware Prosperity Partnership President Kurt Foreman, WuXi AppTec Chairman and CEO Ge Li, WuXi AppTec Co-CEO and WuXi STA CEO Minzhang Chen, and others celebrate the groundbreaking for the WuXi STA Middletown campus.

WuXi breaks ground on Delaware fa­cil­i­ty, boost­ing its US pres­ence

While Middletown, Delaware’s main claim to fame was the site location for the film the Dead Poets Society, the city will soon play host to a massive manufacturing outfit.

WuXi AppTec’s contract research and manufacturing subsidiary WuXi STA, has broken ground on a 190-acre manufacturing campus in Middletown.

According to the company, this site will be WuXi’s second facility in the US, and it will create around 500 full-time jobs by 2026, but there are plans to kick off operations in 2025.