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.

Achiev­ing Dig­i­tal Trans­for­ma­tion: Un­lock­ing Cost Re­duc­tion, Clin­i­cal Ex­cel­lence & Pre­ci­sion Ther­a­peu­tics Man­u­fac­tur­ing

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End­points 20(+2) un­der 40, 2023; Bio­phar­ma's high­est-paid CEOs; N-of-1 CRISPR sto­ry goes on af­ter tragedy; 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.

We will be off Monday in observance of Memorial Day — and when we get back, it will be a straight march to ASCO, BIO and more. Enjoy the (long) weekend!

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Douglas Love, Annexon CEO

An­nex­on’s GA drug miss­es on pri­ma­ry goal but win on vi­su­al acu­ity will be fo­cus of planned late-stage tri­al

Annexon’s complement inhibitor didn’t prove better than sham at reducing lesion growth in a leading cause of blindness, but the biotech still plans to move forward on the back of secondary endpoints showing visual acuity preservation, which will “certainly” be the primary goal in a late-stage trial to be discussed shortly with the FDA, CEO Douglas Love told Endpoints News. 

The California biotech’s ANX007 was not statistically significant compared to pooled sham, the comparator, at 12 months in patients with geographic atrophy, per a Wednesday presentation. In every-month dosing, the GA lesion area changed about 6.2% from baseline (p=0.526) and 1.3% (p=0.896) in the every-other-month group. In a March note, Jefferies analyst Suji Jeong said a reduction of 20% to 30% would be “encouraging.”

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Teresa Bitetti, Takeda's president of the global oncology business unit

Take­da wins pri­or­i­ty re­view for $400M col­orec­tal can­cer drug, li­censed from Hutchmed in Jan­u­ary

Takeda and Hutchmed scored a priority review Thursday afternoon for a colorectal cancer drug, the companies announced.

The experimental drug in question is fruquintinib, previously approved in China in 2018 to treat metastatic colorectal cancer. Takeda and Hutchmed are aiming to bring fruquintinib to the US and other countries outside China in the same indication, and the FDA set its decision date for Nov. 30 of this year.

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Rich Horgan (R) with his late brother, Terry

Rich Hor­gan spear­head­ed a gene ther­a­py for his broth­er. The tri­al end­ed in tragedy, but the work con­tin­ues for more pa­tients

Rich Horgan’s quest to create a custom gene therapy for his brother, Terry, ended in tragedy. But Horgan doesn’t believe it’s the end of the story.

Terry, a 27-year-old patient with Duchenne muscular dystrophy, died last October just eight days after receiving the therapy in a clinical trial in which he was the only participant. The case raised questions about the safety of certain gene therapies and what would happen to other drug programs under a nonprofit that Horgan created, called Cure Rare Disease.

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Bio­phar­ma's 20 high­est-paid CEOs of 2022, each bring­ing in $20M+ pay­days

Even in a down year for much of the biopharma market, 20 CEOs brought in pay packages valued at more than $20 million, an Endpoints News analysis found.

Endpoints collected data on more than 350 CEO compensation packages, covering a wide range of pharma, biotech, and life sciences companies. All told, the 20 largest earners made over $725 million in 2022 — an average package of $36.4 million. Three brought in paydays over $50 million, and one CEO broke the $100 million mark.

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The 20(+2) un­der 40: Your guide to the next gen­er­a­tion of biotech lead­ers

This year’s list of 20 biotech leaders under the age of 40 includes a huge range of ambitions. Some of our honorees are planning to create the next big drug giant. Others are pushing the bounds of AI. One is working to revolutionize TB testing. All are compelling talents who are still young in age, but already far along in achievement.

And, as in years past, we went over. The 20 are actually 22 because of two double profiles that reflect how important teamwork is in the industry. As one of our honorees, Joe Illingworth of DJS Antibodies, told me in our interview, “It takes a village to raise a biotech.”

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FDA ap­proves Lex­i­con’s heart-fail­ure drug af­ter de­feat in di­a­betes

The FDA on Friday approved Lexicon’s heart failure drug sotagliflozin following a string of setbacks for the pharma company, including an FDA rejection in diabetes and the loss of a development deal with Sanofi.

The dual SGLT1 and SGLT2 inhibitor will be marketed as Inpefa and is a once-daily tablet. It’s been approved to reduce the risk of cardiovascular death and heart failure-related hospitalization or urgent visits in adults with heart failure or type 2 diabetes mellitus, chronic kidney disease, and other cardiovascular risk factors. The label spans the range of left ventricular ejection fraction, including preserved ejection fraction and reduced ejection fraction, as well as patients with or without diabetes, Lexicon said Friday.

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Eu­ro­pean Com­mis­sion to re­ceive few­er Pfiz­er-BioN­Tech vac­cine dos­es un­der amend­ed con­tract

The European Commission has made a few changes to its vaccine contract with Pfizer and BioNTech, reducing the dose volume while extending the delivery timeline to cope with “evolving public health needs.”

The Commission previously struck a contract in May 2021 for 900 million doses, with the option to purchase another 900 million. Of those, 450 million were expected to be delivered in 2023, though an amendment now calls for fewer doses. While neither the Commission nor Pfizer and BioNTech have revealed an exact amount, an unnamed source told Reuters that the amendment reduces the remaining expected doses by about a third.

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