Be­yond cold stor­age: Tech­nol­o­gy de­signed to keep or­gans warm, helps liv­er trans­plant pa­tients in key study

Since the 1960s, cold stor­age emerged as the pre­ferred method for or­gan preser­va­tion for trans­plan­ta­tion, but it didn’t tick all the box­es — lim­i­ta­tions such as the risk of ear­ly graft dys­func­tion still con­tribute to chron­ic com­pli­ca­tions. So an up­grade was in or­der. In­stead of flush­ing and pre­serv­ing the pro­cured or­gan at rough­ly freez­ing tem­per­a­tures, new tech­nol­o­gy ac­tu­al­ly keeps the or­gans warm and toasty for trans­plan­ta­tion in a bid to im­prove or­gan ac­ces­si­bil­i­ty and im­prove trans­plan­ta­tion out­comes.

The tech­nol­o­gy, pi­o­neered by An­dover, Mass­a­chu­setts-based Trans­Medics, ap­proved for use by the FDA for lung preser­va­tion, is un­der reg­u­la­to­ry re­view for the heart — and is be­ing test­ed for the liv­er. On Tues­day, the de­vel­op­er un­veiled late-stage da­ta that showed its prod­uct, called the Or­gan Care Sys­tem (OCS) Sys­tem, low­ered the in­ci­dence of ear­ly al­lo­graft dys­func­tion (EAD) — an ini­tial poor func­tion of the trans­plant­ed liv­er that typ­i­cal­ly por­tends poor al­lo­graft and pa­tient sur­vival.

The 300-pa­tient tri­al, chris­tened PRO­TECT, as­sessed the im­pact of the OCS sys­tem on pre­serv­ing and as­sess­ing donor liv­ers in­tend­ed for trans­plan­ta­tion — 153 pa­tients were trans­plant­ed with the OCS Liv­er and 147 pa­tients were on the con­trol group, which em­ployed cold stor­age meth­ods.

The pri­ma­ry ef­fec­tive­ness end­point was the in­ci­dence of EAD in the first 7 days fol­low­ing trans­plant pro­ce­dures, and that was met: 17.3% for OCS ver­sus 30.5% for con­trol (p=0.009). The main safe­ty goal, the av­er­age num­ber of liv­er graft-re­lat­ed se­ri­ous ad­verse events per pa­tient mea­sured over 30 days fol­low­ing trans­plan­ta­tion, was al­so achieved 0.046 for the OCS group, com­pared to 0.075 in the con­trol arm (p<0.0001).

Sec­ondary ef­fi­ca­cy end­points, in­clud­ing a sig­nif­i­cant­ly low­er in­ci­dence of is­chemic cholan­giopa­thy com­pli­ca­tions at 6 months post-trans­plan­ta­tion, were al­so met.

Mal­colm Mac­Con­mara UT South­west­ern

“To our knowl­edge, these re­sults from the OCS Liv­er PRO­TECT tri­al rep­re­sent the first time a new tech­nol­o­gy or ther­a­py has had a pos­i­tive im­pact on both EAD and is­chemic cholan­giopa­thy in liv­er trans­plan­ta­tion,” said Mal­colm Mac­Con­mara, the tri­al’s co-in­ves­ti­ga­tor and di­rec­tor of the or­gan re­search lab at UT South­west­ern Med­ical Cen­ter. “If ap­proved, this would safe­ly ex­pand the uti­liza­tion of donor liv­ers and sig­nif­i­cant­ly in­crease the num­ber of liv­ers avail­able for life-sav­ing trans­plan­ta­tion.”

For now, the OCS sys­tem is ap­proved for lung preser­va­tion — it is de­signed to re­duce the time dur­ing which the lungs lack blood and oxy­gen sup­port out­side of the body. It in­cludes a portable en­clo­sure with me­chan­i­cal and elec­tri­cal com­po­nents used to warm, ven­ti­late and per­fuse the lungs — as well as a lung preser­va­tion so­lu­tion com­bined with packed red blood cells. The trans­plant team can al­so mon­i­tor and as­sess the donor lungs while they are be­ing per­fused be­fore trans­plan­ta­tion.

Ini­tial­ly it was sanc­tioned for use in on­ly stan­dard-cri­te­ria donor lungs, but last year the FDA ex­pand­ed its use in donor lung pairs ini­tial­ly con­sid­ered un­ac­cept­able for trans­plan­ta­tion based on lim­i­ta­tions of cold stor­age preser­va­tion, for ex­am­ple for pa­tients that are ge­o­graph­i­cal­ly dis­tant.

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.

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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Kate Haviland, Blueprint Medicines CEO

Blue­print met all its end­points in bid for ex­pand­ed Ay­vak­it la­bel — but stock trends low­er any­way

Blueprint Medicines announced this morning that the second part of its study on Ayvakit in non-advanced systemic mastocytosis (SM) — a rare disease in which a type of white blood cells known as mast cells builds up — met all endpoints, but the biopharma left key questions unanswered.

In 212 patients, with 141 in the treatment arm and 71 in the control arm, patients who got Ayvakit saw an average 15.6-point decrease in their symptom scores compared to a 9.2-point decrease in the placebo arm at 24 weeks. In an extension study, those on Ayvakit saw their symptom scores drop by 20.2 points by week 48.

Paul Hudson, Sanofi CEO (Eric Piermont/AFP via Getty Images)

Up­dat­ed: Hit by an­oth­er PhI­II flop, Sanofi culls breast can­cer drug — sound­ing alarm for the class

Sanofi is officially giving up on its oral SERD.

The French drugmaker put out word Wednesday morning that it will discontinue the global development program of amcenestrant, the selective estrogen receptor degrader once billed as a top late-stage prospect. Having already failed a Phase II monotherapy test earlier this year, a combo with the drug also missed the bar in a second trial for breast cancer, triggering the decision to drop the whole program.

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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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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.”

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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Marisol Peron, Genmab SVP of communications and corporate affairs

Gen­mab launch­es cor­po­rate cam­paign am­pli­fy­ing its ‘knock your socks off’ an­ti­bod­ies

Genmab often talks about its “knock-your-socks-off” antibodies — and now the term is getting its own logo and corporate campaign.

The teal and purple logo for the acronym KYSO — Genmab pronounces it “ky-so” — debuts on Wednesday and comes on the heels of Genmab’s newly announced 2030 vision. That aspiration aims to expand Genmab’s drug development beyond oncology to include other serious diseases, while also doubling down on its own drug development.

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