News brief­ing: Io­n­is reads out pos­i­tive da­ta for prekallikrein-tar­get­ing drug; PPD col­lab­o­rates on lu­pus re­search

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

Late Fri­day ap­proval; Trio of biotechs wind down; Stem cell pi­o­neer finds new fron­tier; Biotech icon to re­tire; 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.

I hope your weekend is off to a nice start, wherever you are reading this email. As for me, I’m trying to catch the tail of the Lunar New Year festivities.

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Pfiz­er lays off em­ploy­ees at Cal­i­for­nia and Con­necti­cut sites

Pfizer has laid off employees at its La Jolla, CA, and Groton, CT sites, according to multiple LinkedIn posts from former employees.

The Big Pharma confirmed to Endpoints News it has let go of some employees, but a spokesperson declined to specify how many workers were impacted and the exact locations affected. Earlier this month, the drug developer had confirmed to Endpoints it was sharpening its focus and doing away with some early research on areas such as rare disease, oncology and gene therapies.

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Jake Van Naarden, Loxo@Lilly CEO

Lil­ly en­ters ripe BTK field with quick FDA nod in man­tle cell lym­phoma

Eli Lilly has succeeded in its attempt to get the first non-covalent version of Bruton’s tyrosine kinase, or BTK, inhibitors to market, pushing it past rival Merck.

The FDA gave an accelerated nod to Lilly’s daily oral med, to be sold as Jaypirca, for patients with relapsed or refractory mantle cell lymphoma.

The agency’s green light, disclosed by the Indianapolis Big Pharma on Friday afternoon, catapults Lilly into a field dominated by covalent BTK inhibitors, which includes AbbVie and Johnson & Johnson’s Imbruvica, AstraZeneca’s Calquence and BeiGene’s Brukinsa.

Filip Dubovsky, Novavax CMO

No­vavax gets ready to take an­oth­er shot at Covid vac­cine mar­ket with next sea­son plans

While mRNA took center stage at yesterday’s FDA vaccine advisory committee meeting, Novavax announced its plans to deliver an updated protein-based vaccine based on new guidance.

Vaccines and Related Biological Products Advisory Committee (VRBPAC) members voted unanimously in favor of “harmonizing” Covid vaccine compositions, meaning all future vaccine recipients would receive a bivalent vaccine, regardless of whether they’ve gotten their primary series.

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Eliot Forster, F-star CEO (Rachel Kiki for Endpoints News)

F-star gets down to the wire with $161M sale to Chi­nese buy­er as na­tion­al se­cu­ri­ty con­cerns linger

With the clock ticking on F-star Therapeutics’ takeover by a Chinese buyer, the companies are still scrambling to remove a hold on the deal from the US government’s Committee on Foreign Investment in the United States.

F-star and invoX Pharma said they are “actively negotiating” with CFIUS “about the terms of a mitigation agreement to address CFIUS’s concerns regarding potential national security risks posed by the transaction.”

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CBER Director Peter Marks (Susan Walsh/AP Images)

FDA ad­vi­so­ry com­mit­tee votes unan­i­mous­ly in fa­vor of bi­va­lent Covid shots re­plac­ing pri­ma­ry se­ries

The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) voted unanimously in favor of “harmonizing” Covid vaccine compositions, meaning all current vaccine recipients would receive a bivalent vaccine, regardless of whether they’ve gotten their primary series.

The vote marks an effort to clear up confusion around varying formulations and dosing schedules for current primary series and booster vaccines, as well as “get closer to the strains that are circulating,” according to committee member Paul Offit, professor of pediatrics at the Children’s Hospital of Philadelphia.

Rodney Rietze, iVexSol CEO

Bris­tol My­ers, Charles Riv­er join Se­ries A fund­ing for iVex­Sol

Massachusetts-based iVexSol has secured funding to the tune of $23.8 million in its latest Series A round. The new investors include Bristol Myers Squibb, manufacturer Charles River Laboratories and Asahi Kasei Medical.

iVexSol is a manufacturer of lentiviral vectors (LVV), used in making gene therapies, and this latest round of fundraising brings its total Series A total over $39 million, which will be used to recruit more employees and bolster its technology.

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John Rim, Samsung Biologics CEO (Samsung/PR Newswire)

Sam­sung Bi­o­log­ics spells out ex­pan­sion plans in South Ko­rea and US

The CDMO arm of one of South Korea’s largest conglomerates has posted its year-end results and plans for 2023, which include new construction.

Samsung Biologics netted north of KRW 3 trillion ($2.4 billion) in 2022 revenue and an operating profit of KRW 983.6 billion ($799 million), which the company touted on Friday as “record-high earnings.” The revenue boost was 55% compared to 2021.

No­var­tis' ap­proved sick­le cell dis­ease drug fails to beat place­bo in PhI­II

Novartis’ sickle cell drug, approved in 2019 and branded as Adakveo, has failed an ongoing Phase III, according to preliminary results.

The Swiss pharma giant unveiled early data from the ongoing STAND Phase III study on Friday, saying that crizanlizumab showed no statistically significant difference between the drug at two different dose levels compared to placebo in annualized rates of vaso-occlusive crises that lead to a healthcare visit over the first year since being randomized into the trial.