Large phar­ma com­pa­nies that have made “most fa­vored na­tion” deals with the White House will have un­til the end of Pres­i­dent Don­ald Trump’s cur­rent term to ful­fill some of their com­mit­ments to in­vest in the US, ac­cord­ing to a frame­work of the agree­ments re­viewed by End­points News.  

The doc­u­ment, ti­tled a “let­ter of agree­ment,” is a tem­plate that ap­pears to for­mal­ize agree­ments be­tween com­pa­nies and the US De­part­ment of Com­merce. Its au­then­tic­i­ty was con­firmed by mul­ti­ple sources in­volved in those ne­go­ti­a­tions.

The ex­act amounts com­pa­nies will have to in­vest in the US are be­ing ne­go­ti­at­ed in­di­vid­u­al­ly, ac­cord­ing to two drug­mak­ers in­volved in talks. At least one ex­pects to ful­fill the en­tire­ty of its multi­bil­lion-dol­lar pledge by the Jan. 1, 2029 dead­line.

A pi­o­neer­ing gene ther­a­py from Na­tion­wide Chil­dren's Hos­pi­tal is bring­ing rare op­ti­mism to a field that has long strug­gled to de­vel­op such treat­ments for cer­tain neu­rode­vel­op­ment dis­or­ders.

In Sep­tem­ber, 8-year-old Maxwell Freed be­came the first per­son to re­ceive a gene ther­a­py for SLC6A1, a rare con­di­tion that caus­es seizures, de­vel­op­men­tal de­lays and autism-like symp­toms, Na­tion­wide Chil­dren’s in Colum­bus, Ohio an­nounced Thurs­day. The med­i­cine has im­proved Maxwell’s mood, mus­cle tone and co­or­di­na­tion, en­abling a se­ries of firsts, in­clud­ing rid­ing a bike.

“He’s see­ing the world for the first time through a brain that is wak­ing up,” his moth­er, Am­ber Freed, told End­points News. “He’s much more in­ter­est­ed in the world around him.”

Cres­cent Bio­phar­ma made a set of sweep­ing moves Thurs­day morn­ing, no­tably with its strat­e­gy of bring­ing its PD-1xVEGF to Chi­na.

Boston-based Cres­cent signed a two-way deal with Kelun-Biotech, in which both com­pa­nies will have ac­cess to some of each oth­er­s' as­sets. Cres­cent said that clin­i­cal tri­al prepa­ra­tion is un­der­way and that it se­cured a $185 mil­lion pri­vate place­ment.

In­vestors ap­peared to ap­pre­ci­ate Cres­cen­t's moves. Its share price CBIO rose 15% in pre­mar­ket trad­ing.

The news be­gins with Cres­cent out-li­cens­ing the Greater Chi­na rights to its can­cer drug can­di­date CR-001 to Kelun-Biotech. Kelun will pay Cres­cent $20 mil­lion up­front and up to $30 mil­lion in mile­stones for the rights to the PD-1xVEGF drug can­di­date.

Plus, news about Pli­ant Ther­a­peu­tics, Repli­cate Bio­science, De­nali, Lai­go Bio and Aux­il­ius Phar­ma:

📉 UniQure's shares fall as it re­it­er­ates FDA's neg­a­tive feed­back on Hunt­ing­ton's study: The FDA said in its meet­ing min­utes to the com­pa­ny that the Phase 1/2 re­sults on its Hunt­ing­ton's dis­ease gene ther­a­py "are cur­rent­ly un­like­ly to pro­vide the pri­ma­ry ev­i­dence" to sup­port a fil­ing, echo­ing pre­vi­ous com­ments that uniQure shared from the agency in No­vem­ber. UniQure said it's re­quest­ing a fol­low-up meet­ing with the FDA for ear­ly next year. UniQure made waves when it re­port­ed in Sep­tem­ber that its Hunt­ing­ton's treat­ment suc­ceed­ed in a Phase 1/2 tri­al, mean­ing it had the po­ten­tial to be­come the first gene ther­a­py for the dis­ease. But those prospects were sub­se­quent­ly quashed by the FDA. The com­pa­ny's shares QURE fell 10% at mar­ket open Thurs­day. — Lei Lei Wu