In a com­pas­sion­ate-use pi­lot study for hered­i­tary an­gioede­ma (HAE), Io­n­is’ PKKRx and PKK-LRx were shown to re­duce the rate of break­through at­tacks in two pa­tients.

PKK-LRx is cur­rent­ly in Phase II de­vel­op­ment to treat the rare ge­net­ic dis­ease, which caus­es rapid and painful in­flam­ma­tion at­tacks in the hands, feet, limbs, face, ab­domen, lar­ynx and tra­chea. The lig­and-con­ju­gat­ed an­ti­sense (LI­CA) oligonu­cleotide is de­signed to re­duce the pro­duc­tion of prekallikrein (PKK).

Two pa­tients in the com­pas­sion­ate-use study — 24- and 27-year-old women with se­vere bradykinin-me­di­at­ed forms of an­gioede­ma — were giv­en the un­con­ju­gat­ed par­ent drug PKKRx, fol­lowed by lig­and-con­ju­gat­ed PKK-LRx.

The mean month­ly at­tack rate de­creased from 1.2 to 0.25 for Pa­tient 1, and from 7.9 to 1.0 in Pa­tient 2, ac­cord­ing to a re­port in the New Eng­land Jour­nal of Med­i­cine. And no ad­verse ef­fects were re­port­ed.

“These find­ings sup­port con­tin­ued de­vel­op­ment of the lig­and-con­ju­gat­ed ASO in­hibitor of he­pat­ic prekallikrein ex­pres­sion for the treat­ment of pa­tients with se­vere hered­i­tary an­gioede­ma who have lim­it­ed re­sponse to or un­ac­cept­able side ef­fects from cur­rent ther­a­pies,” the re­port states. — Nicole De­Feud­is 

PPD part­ners with Lu­pus Re­search Al­liance af­fil­i­ate to ad­vance lu­pus re­search

PPD has en­tered in­to a col­lab­o­ra­tion deal with Lu­pus Ther­a­peu­tics, an af­fil­i­ate of the Lu­pus Re­search Al­liance, to ex­pand lu­pus re­search for the CRO’s glob­al cus­tomer base.

Karen Kau­cic

With the deal, PPD will be able to op­ti­mize its tri­als and pro­to­cols thanks to LT’s own clin­i­cal in­ves­ti­ga­tor net­work. LT in turn gets ac­cess to bet­ter med­i­cines and ther­a­pies for its own pa­tients, the com­pa­nies said. Fi­nan­cial terms of the deal were not dis­closed.

Through that net­work, PPD will get ac­cess to more than 50 re­search sites with over 200 af­fil­i­at­ed in­ves­ti­ga­tors, who col­lec­tive­ly see more than 20,000 ac­tive sys­temic lu­pus ery­the­mato­sus (SLE) pa­tients.

PPD’s CMO Karen Kau­cic said in a state­ment that the CRO is ex­cit­ed to part­ner with a com­pa­ny that has “site staff who have ex­pe­ri­ence across mul­ti­ple stud­ies, a known, re­li­able net­work of in­ves­ti­ga­tors and sites ready to ini­ti­ate clin­i­cal tri­als award­ed work.”

Lu­pus is a chron­ic con­di­tion in which a per­son’s im­mune sys­tem at­tacks its own tis­sues and or­gans, caus­ing in­flam­ma­tion that can dam­age any part of the body. Lu­pus is dif­fi­cult to di­ag­nose be­cause its signs and symp­toms can im­i­tate a wide va­ri­ety of oth­er ail­ments. — Max Gel­man

Ma­gen­ta CFO hits the ex­it as the CEO brings in 2 new top ex­ecs for the team

Ja­son Gard­ner

There’s a shake­up un­der­way in the top team run­ning Ma­gen­ta Ther­a­peu­tics.

CEO Ja­son Gard­ner is now look­ing for a re­place­ment for chief op­er­at­ing and fi­nan­cial of­fi­cer Ja­son Ryan, who’s switch­ing to a con­sul­tan­cy role for per­son­al rea­sons.

Then there are two new ex­ecs: Lisa Ol­son, a long­time Ab­b­Vie vet, is tak­ing the CSO po­si­tion while Kevin John­son moves in­to a post run­ning reg­u­la­to­ry and qual­i­ty con­trol. He had been in pret­ty much the same job at Imara and got his PhD at the UNC School of Med­i­cine. — John Car­roll

Xen­cor col­labs with MD An­der­son on tu­mor treat­ments

Cal­i­for­nia-based biotech Xen­cor and the Uni­ver­si­ty of Texas MD An­der­son Can­cer Cen­ter are team­ing up on the treat­ment for a va­ri­ety of can­cers.

The deal will run for the next five years as the two par­ties study Xen­cor’s port­fo­lio of XmAb drug can­di­dates. This in­cludes bis­pe­cif­ic an­ti­bod­ies as well as en­gi­neered cy­tokines, and the biotech cur­rent­ly has nine bis­pecifics and one cy­tokine in Phase I tri­als.

Among Xen­cor’s bis­pe­cif­ic can­di­dates are po­ten­tial treat­ments for acute myeloid leukemia, B cell ma­lig­nan­cies and neu­roen­docrine tu­mors and gas­troin­testi­nal stro­mal tu­mors. The biotech is al­so de­vel­op­ing bis­pecifics that ac­ti­vate with­in the tu­mor mi­croen­vi­ron­ment. — Max Gel­man