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

Image courtesy of The Janssen Pharmaceutical Companies of Johnson & Johnson.

Pro­tect­ing the glob­al phar­ma­ceu­ti­cal in­no­va­tion ecosys­tem – what’s at stake?

We are living in a new era of healthcare that is rapidly advancing progress impacting patient outcomes and experiences. We’ve seen a remarkable pace of transformational innovation, applied research, and advanced clinical development over the last decade.

Despite this tremendous progress, there is much more work to be done, and patients are counting on us – now more than ever – to continue that momentum. At the heart of our industry is a focus on developing and delivering medicines for some of the world’s most challenging diseases, including those that have few or no effective treatments today.

Eu­ro­pean Par­lia­ment calls mem­ber states to ac­tion on an­timi­cro­bial re­sis­tance

Members of the European Parliament have called on EU countries to develop national action plans against antimicrobial resistance (AMR), calling it a top-three priority health threat.

Parliament on Thursday announced recommendations for the fight against AMR, including national action plans that must be updated at least every two years, an EU-level database tracking AMR and antimicrobial use and increased partnership between the pharma industry, patient groups and academia.

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Bay­er buys li­cense to Cedil­la pre­clin­i­cal can­cer pro­gram; MEI Phar­ma says no to un­so­licit­ed of­fer

Bayer has acquired exclusive rights to Cedilla Therapeutics’ preclinical cancer drug candidates for an undisclosed amount.

Bayer announced Thursday morning that it is acquiring the license for Cedilla’s preclinical Cyclin E1/CDK2 complex inhibitors. Cyclin E1 activates CDK2, and the two drive cancer progression and are overexpressed in cancer cells.

Bayer did not disclose what indications it will be pursuing under this partnership. The deal is structured traditionally — Bayer will pay Cedilla an undisclosed amount upfront, and the latter is also eligible for potential development and commercial milestones and royalties. — Lei Lei Wu

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Roche plans to di­vest from lega­cy Genen­tech man­u­fac­tur­ing fa­cil­i­ty in Cal­i­for­nia

Roche is planning to make some changes to its subsidiary’s manufacturing network in California.

The Swiss pharma announced Wednesday that it plans to divest from Genentech’s manufacturing facility in Vacaville, CA, around 58 miles northeast of San Francisco. According to a statement from Roche, the move is part of a “broader strategy” to bring its manufacturing capabilities in line with its future pipeline. Roche is starting the process of finding a buyer for the site but has not named any candidates yet.

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Roger Perlmutter, Eikon Therapeutics CEO

Roger Perl­mut­ter builds Eikon's pipeline with deal-mak­ing flur­ry, rais­ing $106M more

Eikon Therapeutics announced three business development deals on Thursday, effectively dropping in a pipeline of cancer drugs alongside more than $100 million in fresh funding.

The Hayward, CA-based company has become one of biotech’s richest startups since its 2019 founding, having raised nearly $775 million. It’s developing a massive, automated research approach built around Nobel Prize-winning microscope science to peer inside cells and watch proteins in action. After its Series B last year, PitchBook reported a $3.02 billion valuation. And while CEO Roger Perlmutter declined to comment on that figure, he said its first tranche of nearly $106 million in Series C funding is a “meaningful step-up to our Series B valuation.”

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Peter van de Sande, Synaffix CEO

Lon­za shells out $107M cash to snap up Synaf­fix and its ADC plat­form

After lining up a string of partnerships over the years, Dutch antibody-drug conjugate specialist Synaffix has found a new home: Lonza, the contract development and manufacturing giant.

Lonza is paying about $107 million (€100 million) in cash to acquire Synaffix, with up to $64 million (€60 million) in “additional performance-based consideration” on the table. Synaffix’s ADC tech platform will now become part of Lonza’s offering for biopharma clients, lending its bioconjugate technologies to not just ADCs but also targeted gene therapy, immune cell engagers and other applications.

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Who's con­fi­dent­ly in­vest­ing in biotech star­tups dur­ing these tense days? We've got some an­swers

We’ve got a changeup to our event schedule in Boston next week, where we’ll be doing a mix of live/streaming events at our base at The Seaport Hotel as part of a two-day lineup of webinars, virtual firesides and a cocktail hour Q&A with a veteran of the biotech financing scene.

The 9:30-10:30 am ET live slot on Tuesday, June 6, will now feature a panel conversation on the current state of affairs for VC investing in biotech, focusing on what startups are getting cash — and how. Alaa Halawa, head of US ventures at Mubadala, is confirmed, along with Brian Goodman at MPM and Geoff von Maltzahn, a general partner at Moderna-buoyed Flagship. I have a couple of other invites out and will let you know how that plays out.

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The Modulo Bio team with CEO Michael Horowitz (fourth from right in semicircle)

Ex­clu­sive: With $8M, neu­ro start­up Mod­u­lo Bio emerges to test small mol­e­cules for ALS, de­men­tia in CEO’s per­son­al mis­sion

Embarking on a personal mission after his best friend’s mother was diagnosed with a mutation-driven case of frontotemporal dementia, Michael Horowitz has pulled together $8 million in venture funding at Modulo Bio to create small molecules for neurodegenerative diseases.

The San Diego and Bay Area biotech will select its lead development candidate and some backup options within six months and then raise a Series A to investigate therapeutics for C9orf72 mutation-driven cases of ALS and frontotemporal dementia, Horowitz told Endpoints News.

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Albert Bourla, Pfizer CEO (Michel Euler/AP Images, Pool)

FDA ap­proves Pfiz­er’s RSV shot for old­er adults, tee­ing up a com­pet­i­tive $17B vac­cine mar­ket

The FDA approved Pfizer’s RSV vaccine called Abrysvo for older adults on Wednesday, placing another Big Pharma onto the commercial stage ahead of the next RSV season.

Pfizer’s approval comes weeks after GSK won approval for its rival shot, Arexvy. Those two vaccines are both approved for use in adults 60 years and older and will be reviewed by a CDC panel in June before they’re expected to commercially launch this fall. Wall Street analysts see RSV as the next multibillion-dollar vaccine market, with Jefferies analysts recently forecasting the RSV market will grow to $17 billion over the next decade.

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