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

Da­ta Lit­er­a­cy: The Foun­da­tion for Mod­ern Tri­al Ex­e­cu­tion

In 2016, the International Council for Harmonisation (ICH) updated their “Guidelines for Good Clinical Practice.” One key shift was a mandate to implement a risk-based quality management system throughout all stages of a clinical trial, and to take a systematic, prioritized, risk-based approach to clinical trial monitoring—on-site monitoring, remote monitoring, or any combination thereof.

Mer­ck scraps Covid-19 vac­cine pro­grams af­ter they fail to mea­sure up on ef­fi­ca­cy in an­oth­er ma­jor set­back in the glob­al fight

After turning up late to the vaccine development game in the global fight against Covid-19, Merck is now making a quick exit.

The pharma giant is reporting this morning that it’s decided to drop development of 2 vaccines — V590 and V591 — after taking a look at Phase I data that simply don’t measure up to either the natural immune response seen in people exposed to the virus or the vaccines already on or near the market.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 98,600+ biopharma pros reading Endpoints daily — and it's free.

News brief­ing: Jef­frey Lei­den to chair Tmu­ni­ty board of di­rec­tors; Op­di­vo wins new ap­proval in ad­vanced RCC

Longtime Vertex CEO Jeffrey Leiden is taking on a new role.

Leiden has been appointed chairman of Tmunity’s board of directors, the company announced Monday. The move comes about a year and a half after Leiden announced he’d be stepping down from his position at Vertex.

Vertex saw immense growth under Leiden, leading the company from its exit out of hepatitis C, when cures were moving in, and into cystic fibrosis. The company’s cystic fibrosis triple combo therapy Trikafta is already its best-seller, reaching the distinction just six weeks after launch and recording the strongest first quarter of sales for any drug, per some estimates.

Matt Gline (L) and Vivek Ramaswamy

Scoop: Vivek Ra­maswamy is hand­ing the CEO job to a top lieu­tenant at Roivant — but he’s not ex­act­ly leav­ing the biotech scene

Over the past 7 years since founding Roivant, Vivek Ramaswamy has been a constant blur of biotech building motion.

He launched his first biotech with an Alzheimer’s drug he picked up cheap, and watched the experiment implode in one of the highest profile pivotal disasters seen in the last decade. But it didn’t slow the 30-something exec down; if anything, he hit the accelerator. Ramaswamy blazed global paths and went on to raise billions to spur the creation of a large lineup of little Vants promising big things at a fast pace. He sold off a section of the Vant brigade to Sumitomo Dainippon for $3 billion. And more recently the relentless dealmaker has been building a computational discovery arm to add an AI-driven approach to kicking up new programs and companies, supplementing the in-licensing drive while pursuing advances that have created more than 700 jobs at Roivant, with $2 billion in reserves.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Ron Cooper, Albireo CEO

Al­bireo just ad­vanced down to the 10-yard line at the FDA. And Ron Coop­er’s team is get­ting prepped for the next big play

When Albireo Pharma’s board $ALBO moved to bring in Ron Cooper as the CEO more than 5 years ago, the development-stage company went with an experienced commercial player who had a big-time position on his resume after running Bristol Myers’ commercial ops in Europe.

Now, after successfully navigating a pivotal study, putting them in a foot race with a rival toward an FDA OK, Cooper is getting a boost from regulators on the last drive back to an arena he understands completely.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Hal Barron, GSK via YouTube

What does $29B buy you in Big Phar­ma? In Glax­o­SmithK­line’s case, a whole lot of un­com­fort­able ques­tions about the pipeline

Talk about your bad timing.

A little over a week ago, GSK R&D chief Hal Barron marked his third anniversary at the research helm by taking a turn at the virtual podium during JP Morgan to make the case that he and his team had built a valuable late-stage pipeline capable of churning out more than 10 blockbusters in the next 5 years.

And then, just days later, one of the cancer drugs he bet big on as a top prospect — bintrafusp, partnered with Merck KGaA — failed its first pivotal test in non-small cell lung cancer.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Can strug­gling Iterum turn the cor­ner to an an­tibi­ot­ic suc­cess sto­ry? They will know in six months

More than five years after Corey Fishman and Michael Dunne dusted sulopenem off Pfizer’s shelves — the second castoff antibiotic they’ve brought out of the pharma giant — and founded Iterum Therapeutics around that single drug, they have lined up a quick shot at approval with priority review from the FDA.

The decision, six months from now, will mark a make-or-break moment for a struggling biotech that has just enough cash to keep the lights on until the third quarter.

Bahija Jallal, Immunocore

Buried in Im­muno­core's IPO fil­ings? A kick­back scheme from a now for­mer em­ploy­ee

Immunocore spent much of 2019 dealing with the fallout of the Neil Woodford scandal, as the former star investor’s fall crashed the biotech’s valuation out of unicorn range. Now it turns out that the company spent 2020 dealing with another internal scandal.

The longtime UK biotech darling disclosed in their IPO filing last week that they had fallen victim to an alleged kickback scheme involving one of their employees. After a whistleblower came forward, they said in their F-1, they spent the summer and spring investigating, finding fraud on the part of an employee and two outside vendors.

Stéphane Bancel, Moderna CEO (Steven Ferdman/Getty Images)

Covid-19 roundup: Mod­er­na dou­bles down on Covid-19 with new boost­er tri­als; Aus­tralia plans do­mes­tic pro­duc­tion of As­traZeneca vac­cine amid dis­tri­b­u­tion lag

As Merck bows out of the global race to develop vaccines for Covid-19, Moderna is doubling down to make sure they can quell new variants that have recently emerged and quickly spread.

The Cambridge, MA-based biotech put out word on Monday that in vivo studies indicate their mRNA vaccine works well enough against two strains first detected in the UK and South Africa. But with a six-fold reduction in neutralizing titers observed against the latter strain, the company is launching a new study of a booster version to make sure it can do the job.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 98,600+ biopharma pros reading Endpoints daily — and it's free.