Con­cert crushed on PhII schiz­o­phre­nia flop; Am­gen's so­tora­sib gets BTD in Chi­na

Con­cert Phar­ma­ceu­ti­cals stock tanked near­ly 40% on Mon­day morn­ing when a ma­jor Phase II flop left the biotech no choice but to ax its schiz­o­phre­nia pro­gram.

The move leaves Con­cert with just one Phase III can­di­date for alope­cia area­ta in the pipeline, which CEO Roger Tung says is now the fo­cus of their ef­forts. Shares $CNCE hov­ered at around $6.90 on the news — a sharp drop from Fri­day’s close at $10.47.

The can­di­date, a deuter­at­ed form of the en­doge­nous amino acid D-ser­ine dubbed CTP-692, failed both the pri­ma­ry and sec­ondary end­points in a proof-of-con­cept study with 325 pa­tients, Con­cert said. The par­tic­i­pants, who were al­ready on a sta­ble course of an­tipsy­chot­ic med­ica­tion, ran­dom­ly re­ceived ei­ther 1, 2 or 4-gram dos­es of CTP-692 once dai­ly. The pri­ma­ry end­point was mea­sured as change in the Pos­i­tive and Neg­a­tive Symp­tom Scale (PANSS) to­tal score at 12 weeks com­pared to base­line. But un­for­tu­nate­ly, no sig­nif­i­cant im­prove­ment over place­bo was seen at any of the dos­es.

“Un­for­tu­nate­ly, we didn’t see the re­sults we hoped for to sup­port con­tin­u­a­tion of this pro­gram,” Tung said in a state­ment. He laid out plans to shelf CTP-692, and in­stead fo­cus on the com­pa­ny’s Phase III alope­cia area­ta can­di­date, CTP-543.

While CTP-543 has seen some set­backs, the JAK in­hibitor scored a win when it re­ceived break­through ther­a­py des­ig­na­tion back in Ju­ly. The drug, which blocks JAK1 and JAK2, was placed on a brief clin­i­cal hold back in 2017 so the FDA could re­view “cer­tain re­cent­ly com­plet­ed non-clin­i­cal tox­i­col­o­gy stud­ies.” Con­cert fixed the is­sue and com­plet­ed a Phase IIa study in 2018. It’s now rac­ing to beat Pfiz­er’s PF-06651600, which in­hibits JAK3, and PF-06700841, which tar­gets TYK2 and JAK1. — Nicole De­Feud­is

Am­gen’s so­tora­sib gets BTD in Chi­na af­ter lat­est KRAS da­ta

On­ly a day af­ter pre­sent­ing the lat­est round of da­ta for its KRAS in­hibitor so­tora­sib, Am­gen has re­ceived a good sign from the Chi­nese gov­ern­ment.

The in­ves­ti­ga­tion­al drug notched a break­through ther­a­py des­ig­na­tion from Chi­na’s NM­PA for the treat­ment of KRAS G12C-mu­tat­ed lo­cal­ly ad­vanced or metasta­t­ic non-small cell lung can­cer, Am­gen an­nounced Fri­day. The BTD is for pa­tients who have re­ceived at least one pri­or sys­temic ther­a­py.

Last week’s da­ta came in ad­vance of the sched­uled re­lease for the World Con­fer­ence on Lung Can­cer, cour­tesy of an em­bar­go break on the PR for the da­ta. Ac­cord­ing to Am­gen, their one-year fol­lowup on the piv­otal Phase II found a 37.1% over­all re­sponse rate among 126 pa­tients, with 3 com­plete re­spons­es and 43 par­tials. The me­di­an du­ra­tion of re­sponse was 10 months.

So­tora­sib had pre­vi­ous­ly re­ceived a break­through des­ig­na­tion from the FDA, and the Phase II da­ta lined up right along­side the Phase I num­bers. — Max Gel­man

Gilead fronts $220M for Ar­cus in ex­change for larg­er own­er­ship stake

Gilead is in­creas­ing its own­er­ship stake in Ar­cus Bio­sciences just a few weeks af­ter its stock price hit a record high.

The cash in­fu­sion will to­tal rough­ly $220 mil­lion, the com­pa­nies said Mon­day. Gilead’s stake will in­crease to 19.5%, from ap­prox­i­mate­ly 13%, with the pur­chase of 5,650,000 ad­di­tion­al Ar­cus shares at a price of $39 per share.

Some of the fund­ing will go to­ward Ar­cus’ CD73 in­hibitor for pan­cre­at­ic can­cer, for which Ar­cus pre­sent­ed new da­ta that earned rave re­views from an­a­lysts a few weeks ago. Re­searchers tracked a 41% over­all re­sponse rate across all dose-es­ca­la­tion co­horts, ac­cord­ing to Ar­cus. And on Mon­day, Mizuho’s Mara Gold­stein said the Gilead buy-in fur­ther val­i­dates the strength of the da­ta.

“We see the deal in­creas­ing our con­fi­dence for this to be an in­flec­tion-po­ten­tial year for RCUS ahead of key da­ta read­outs in 2021 from mul­ti­ple stud­ies,” Gold­stein wrote to in­vestors.

Gilead’s abil­i­ty to in­crease its stake stems from a May 2020 agree­ment that al­lows the com­pa­ny to raise its Ar­cus own­er­ship to 35% over a 5-year pe­ri­od. — Max Gel­man

Il­lu­mi­na and Se­quoia launch new ge­nomics-fo­cused in­cu­ba­tor 

Il­lu­mi­na and Se­quoia Cap­i­tal Na­tion­al are un­veil­ing an in­cu­ba­tor to build new Chi­nese com­pa­nies in the bustling ge­nomics field.

The com­pa­nies an­nounced on Mon­day the Se­quoia Cap­i­tal Chi­na In­tel­li­gent Health­care Ge­nomics In­cu­ba­tor, Pow­ered by Il­lu­mi­na, which will be­gin its in­au­gur­al fund­ing cy­cle this fall.

Dur­ing two six-month fund­ing cy­cles a year, se­lect­ed star­tups will get ac­cess to in­vest­ment and busi­ness guid­ance from Se­quoia Cap­i­tal Chi­na, and ac­cess to Il­lu­mi­na se­quenc­ing sys­tems and reagents and ful­ly op­er­a­tional lab space in Shang­hai.

“Il­lu­mi­na has a suc­cess­ful track record of cre­at­ing ven­ture-backed ge­nomics start­up com­pa­nies through Il­lu­mi­na Ac­cel­er­a­tor in the U.S. and UK,” Il­lu­mi­na CEO Fran­cis deS­ouza said in a state­ment. — Nicole De­Feud­is 

Ap­pli­ca­tions are open un­til June 1.

Tar­get­ing a Po­ten­tial Vul­ner­a­bil­i­ty of Cer­tain Can­cers with DNA Dam­age Re­sponse

Every individual’s DNA is unique, and because of this, every patient responds differently to disease and treatment. It is astonishing how four tiny building blocks of our DNA – A, T, C, G – dictate our health, disease, and how we age.

The tricky thing about DNA is that it is constantly exposed to damage by sources such as ultraviolet light, certain chemicals, toxins, and even natural biochemical processes inside our cells.¹ If ignored, DNA damage will accumulate in replicating cells, giving rise to mutations that can lead to premature aging, cancer, and other diseases.

Fol­low biotechs go­ing pub­lic with the End­points News IPO Track­er

The Endpoints News team is continuing to track IPO filings for 2021, and we’ve designed a new tracker page for the effort.

Check it out here: Biopharma IPOs 2021 from Endpoints News

You’ll be able to find all the biotechs that have filed and priced so far this year, sortable by quarter and listed by newest first. As of the time of publishing on Feb. 25, there have already been 16 biotechs debuting on Nasdaq so far this year, with an additional four having filed their S-1 paperwork.

Steve Cutler, Icon CEO (Icon)

In the biggest CRO takeover in years, Icon doles out $12B for PRA Health Sci­ences to fo­cus on de­cen­tral­ized clin­i­cal work

Contract research M&A had a healthy run in recent years before recently petering out. But with the market ripe for a big buyout and the Covid-19 pandemic emphasizing the importance of decentralized trials, Wednesday saw a tectonic shift in the CRO world.

Icon, the Dublin-based CRO, will acquire PRA Health Sciences for $12 billion in a move that will shake up the highest rungs of a fragmented market. The merger would combine the 5th- and 6th-largest CROs by 2020 revenue, according to Icon, and the merger will set the newco up to be the second-largest global CRO behind only IQVIA.

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Tom Barnes (Orna)

The mR­NA era is here. MPM be­lieves the fu­ture be­longs to oR­NA — and Big Phar­ma wants a seat at the ta­ble

If the ultra-fast clinical development of Covid-19 vaccines opened the world’s eyes to the promises of messenger RNA, the subsequent delays in supply offered a crash course on the ultra-complex process of producing them. Even before the formulation and fill-finish steps, mRNA is the precious end product from an arduous journey involving enzyme-aided transcription, modification and purification.

For Bristol Myers Squibb, Novartis Institutes for Biomedical Research, Gilead’s Kite and Astellas, it’s time to rethink the way therapeutic RNA is engineered.

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Xen­cor, UCLA to part­ner for new ther­a­peu­tic an­ti­bod­ies; Schrödinger ex­pands part­ner­ship with Google Cloud

Seeking to build on the 20 clinical-stage drug candidates already in its pipeline, California oncology and autoimmune disease-focused biotech Xencor announced on Thursday a partnership with nearby UCLA and the university’s Technology Development Group.

The two will collaborate to develop novel therapeutic antibodies, pairing novel targets proposed by scientists at UCLA with Xencor’s XmAb technology platforms that engineer monoclonal antibodies to create new protein structures. No financial terms of the agreement were disclosed, but any selected drug candidates resulting from the partnership will use a framework of “predefined terms to enter sponsored research agreements and potential license agreements,” the organizations said.

S&P ex­pects steady ero­sion in Big Phar­ma's cred­it pro­file in 2021 as new M&A deals roll in — but don't un­der­es­ti­mate their un­der­ly­ing strength

S&P Global has taken a look at the dominant forces shaping the pharma market and come to the conclusion that there will be more downgrades than upgrades in 2021 — the 8th straight year of steady decline.

But it’s not all bad news. Some things are looking up, and there’s still plenty of money to be made in an industry that enjoys a 30% to 40% profit margin, once you factor in steep R&D expenses.

Tal Zaks, Moderna CMO (AP Photo/Rodrique Ngowi, via still image from video)

CMO Tal Zaks bids Mod­er­na a sur­prise adieu as biotech projects $18.4B in rev­enue, plots post-Covid ex­pan­sion

How do you exit a company after six years in style? Developing one of the most lucrative and life-saving products in pharma history is probably not the worst way to go.

Tal Zaks, Moderna’s CMO since 2015, will leave the mRNA biotech in September, the biotech disclosed in their annual report this morning. The company has already retained the recruitment firm Russell Reynolds to find a replacement.

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Ken Frazier, Merck CEO (Bess Adler/Bloomberg via Getty Images)

UP­DAT­ED: Mer­ck takes a swing at the IL-2 puz­zle­box with a $1.85B play for buzzy Pan­dion and its au­toim­mune hope­fuls

When Roger Perlmutter bid farewell to Merck late last year, the drugmaker perhaps best known now for sales giant Keytruda signaled its intent to take a swing at early-stage novelty with the appointment of discovery head Dean Li. Now, Merck is signing a decent-sized check to bring an IL-2 moonshot into the fold.

Merck will shell out roughly $1.85 billion for Pandion Pharmaceuticals, a biotech hoping to gin up regulatory T cells (Tregs) to treat a range of autoimmune disorders, the drugmaker said Thursday.

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Glax­o­SmithK­line re­thinks strat­e­gy for Covid-19 an­ti­body — not the Vir ones — af­ter tri­al flop. Is there hope in high-risk pa­tients?

In the search for a better Covid-19 therapeutic, GlaxoSmithKline and Vir have partnered up on two antibodies they hope have a chance. GSK is also testing its own in-house antibody, and early results may have shut the door on its widespread use.

A combination of GSK’s monoclonal antibody otilimab plus standard of care couldn’t best standard of care alone in preventing death and respiratory failure in hospitalized Covid-19 patients after 28 days, according to data from the Phase IIa OSCAR study unveiled Thursday.