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.

The FDA has been insisting for months that a Covid-19 vaccine had to be at least 50% effective – a measure of transparency meant to shore public trust in the agency and in a vaccine that had been brought forward at record speed and record political pressure. But now, with concerns of a Trump-driven authorization arriving before the election, the agency may be raising the bar.

The FDA is set to release new guidance that would raise safety and efficacy requirements for a vaccine EUA above earlier guidance and above the criteria used for convalescent plasma or hydroxychloroquine, The Washington Post reported. Experts say this significantly lowers the odds of an approval before the election on November 3, which Trump has promised despite vocal concerns from public health officials, and could help shore up public trust in the agency and any eventual vaccine.

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

How does a biotech celebrate its two-year anniversary? For Immetas Therapeutics, it’s with an $11 million Series A round and a game plan to fight age-related disease.

Co-founders Gene Wang and David Sinclair came together years ago around the idea that inflammation is the ultimate process driving age-related illnesses, including cancer. The duo launched Immetas in 2018 and packed the staff with industry experts. Wang, who says he’s always had an entrepreneurial spirit, has held lead roles at Novartis, GSK, Bristol Myers Squibb and Merck. He’s worked on blockbuster drugs like Humira, Gardasil, Varubi and Zolinza. And now, he’s channeling that spirit as CEO.

Having worked in parallel for years to move their respective PD-1 inhibitors up to the first-line treatment of gastrointestinal cancers, Merck and Bristol Myers Squibb finally have the data at ESMO for a showdown.

Comparing KEYNOTE-590 and CheckMate-649, of course, comes with the usual caveats. But a side-by-side look at the overall survival numbers also offer some perspective on a new frontier for the reigning checkpoint rivals, both of whom are claiming to have achieved a first.