Cel­lec­tis re­cruits ex-Servi­er re­search chief De­pil; Are­na inks $224M deal on new drugs; JLABS heads to Shang­hai; As­traZeneca vet takes Goldfinch helm

→ French CAR-T de­vel­op­er Cel­lec­tis has re­cruit­ed Stéphane De­pil, the for­mer R&D chief of Servi­er, to lead the com­pa­ny’s clin­i­cal de­vel­op­ment. De­pil will serve as se­nior vice pres­i­dent of R&D and chief med­ical of­fi­cer. The new ap­point­ment brings De­pil back to the in­dus­try af­ter a break while he di­rect­ed the can­cer im­munother­a­py pro­gram at the Léon Bérard Can­cer Cen­ter in Ly­on, France. But back when he was at Servi­er, he over­saw 20 can­cer pro­grams, sev­er­al of which were in clin­i­cal tri­als. Cel­lec­tis hopes to ben­e­fit from De­pil’s track record in the field to move its CAR-T as­sets in­to the clin­ic af­ter a rough start in­volv­ing a pa­tient death. “Stéphane De­pil’s deep med­ical, aca­d­e­m­ic, and clin­i­cal reg­u­la­to­ry on­col­o­gy ex­pe­ri­ence – specif­i­cal­ly in R&D for all phas­es with­in the phar­ma­ceu­ti­cal, biotech­nol­o­gy, and clin­i­cal re­search spaces – will be in­valu­able as he leads Cel­lec­tis’ strat­e­gy and pro­motes aware­ness of the break­through work that we are do­ing as a leader and in­no­va­tor in the gene-edit­ing field,” said An­dré Chouli­ka, Cel­lec­tis’ CEO, in a state­ment. “His strate­gic al­liance-build­ing, col­lab­o­ra­tion skills, un­der­stand­ing of the glob­al en­vi­ron­ment with on­co­log­i­cal clin­i­cal re­search, and first­hand ex­pe­ri­ence run­ning a phar­ma com­pa­ny all add a great de­gree of abil­i­ty and depth to our lead­er­ship team.”

→ Are­na Phar­ma­ceu­ti­cals, the San Diego drug mak­er best known for its di­et pill Belviq, has inked a deal worth up to $224M with Ever­est Med­i­cines to de­vel­op and com­mer­cial­ize its two new pipeline drugs in main­land Chi­na, Tai­wan, Hong Kong, Macau, and South Ko­rea. The two drugs, ra­linepag and es­trasi­mod, are a far cry from Are­na’s ear­ly work in obe­si­ty drugs. Ra­linepag is a Phase III-ready oral, se­lec­tive prosta­cy­clin re­cep­tor ag­o­nist for the treat­ment of pul­monary ar­te­r­i­al hy­per­ten­sion. The drug’s Phase II da­ta got Are­na’s in­vestors pret­ty jazzed ear­li­er this year. The drug is de­signed to be more po­tent and ef­fec­tive than Acte­lion’s Up­travi. Then there’s etrasi­mod, a Phase II oral S1P re­cep­tor mod­u­la­tor be­ing eval­u­at­ed for mul­ti­ple au­toim­mune dis­eases, in­clud­ing ul­cer­a­tive col­i­tis, a form of in­flam­ma­to­ry bow­el dis­ease. Ear­li­er this year, Amit Mun­shi said the drug was safer and po­ten­tial­ly more ef­fi­ca­cious than Ozan­i­mod, the drug de­vel­oped by San Diego’s Re­cep­tos (pur­chased by Cel­gene for $7.2 bil­lion). In the new deal with Ever­est, a com­pa­ny backed by $50M from Chi­na’s C-Bridge Cap­i­tal, Are­na gets $12M in an up­front pay­ment and is el­i­gi­ble to re­ceive up to $212M in mile­stones, along with roy­al­ties.

John­son & John­son is launch­ing a JLABS in Shang­hai — the first lo­ca­tion out­side of North Amer­i­ca — af­ter reach­ing agree­ments with the Shang­hai Mu­nic­i­pal Gov­ern­ment, Pudong New Area Gov­ern­ment and Shang­hai Phar­ma En­gine Com­pa­ny. Sched­uled to open in Q2 2019, the fa­cil­i­ty in Zhangjiang Hi-Tech Park will span 4,400 sq. me­ter (47,000+ sq. ft.) and can host up to 50 life sci­ence and health care star­tups. “Chi­na, and Shang­hai specif­i­cal­ly, have be­come a glob­al hotspot for health­care in­no­va­tion,” said Vladimir Makat­saria, John­son & John­son Chi­na Group chair­man, in a state­ment. J&J is cur­rent­ly ac­cept­ing ap­pli­ca­tions from biotech, phar­ma­ceu­ti­cal, med­ical de­vice, con­sumer and dig­i­tal health com­pa­nies to this site as well as its eight oth­er JLABS lo­ca­tions.

→ In­dus­try vet An­tho­ny John­son is tak­ing the helm at Goldfinch Bio, a Cam­bridge, MA-based biotech look­ing to de­vel­op new drugs for ge­net­i­cal­ly de­fined kid­ney dis­eases. Goldfinch launched a year ago with $55 mil­lion in Third Rock mon­ey. John­son is the lat­est in a long line of Big Phar­ma R&D ex­ecs mak­ing a move to a start­up, leav­ing his post as head of ear­ly clin­i­cal de­vel­op­ment for the In­no­v­a­tive Med­i­cines and Ear­ly De­vel­op­ment Biotech Unit. His bio in­cludes 25 pos­i­tive proof-of-con­cept stud­ies. In ad­di­tion, Michael Brox­son is join­ing Goldfinch as chief busi­ness and op­er­at­ing of­fi­cer.

Shire $SH­PG has struck a col­lab­o­ra­tion deal with Rani Ther­a­peu­tics that gives the rare dis­ease gi­ant ex­clu­sive rights to re­search, eval­u­ate and pur­sue a world­wide li­cense for the oral de­liv­ery of fac­tor VI­II to pa­tients with he­mo­phil­ia A. This in­vest­ment, the terms of which were not dis­closed, comes just a few weeks af­ter ri­val Roche’s Hem­li­bra nabbed an FDA ap­proval, some­thing that an­a­lysts said could help shrink Shire’s share of the he­mo­phil­ia mar­ket from 49% to 29%. The Rani Pill tech­nol­o­gy brings a nov­el in­testi­nal de­liv­ery ap­proach to the treat­ment of he­mo­phil­ia, which is dom­i­nat­ed by in­jecta­bles, though the com­pa­ny is al­so do­ing work on rheuma­toid arthri­tis, can­cer and Crohn’s dis­ease. As part of the pact, Shire has al­so made an eq­ui­ty in­vest­ment in­to Rani Ther­a­peu­tics.

Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen spot­lights a pair of painful pipeline set­backs as ad­u­canum­ab show­down looms at the FDA

Biogen has flagged a pair of setbacks in the pipeline, spotlighting the final failure for a one-time top MS prospect while scrapping a gene therapy for SMA after the IND was put on hold due to toxicity.

Both failures will raise the stakes even higher on aducanumab, the Alzheimer’s drug that Biogen is betting the ranch on, determined to pursue an FDA OK despite significant skepticism they can make it with mixed results and a reliance on post hoc data mining. And the failures are being reported as Biogen was forced to cut its profit forecast for 2020 as a generic rival started to erode their big franchise drug.

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A new chap­ter in the de­cen­tral­ized clin­i­cal tri­al ap­proach

Despite the promised decentralized trial revolution, we haven’t yet moved the needle in a significant way, although we are seeing far bolder commitments to this as we continue to experience the pandemic restrictions for some time to come. The vision of grandeur is one thing, but operationalizing and execution are another and recognising that change, particularly mid-flight on studies, is worthy of thorough evaluation and consideration in order to achieve success. Here we will discuss one of the critical building blocks of a Decentralized and Remote Trial strategy: TeleConsent; more than paper under glass, it is a paradigm change and key digital enabler.

Stephen Hoge, Moderna president (Moderna)

On morn­ing of FDA Covid-19 ad­comm, Mod­er­na com­pletes PhI­II en­roll­ment, putting them neck-and-neck with Pfiz­er

Weeks away from a potential EUA application, Moderna announced they have completed enrollment in their 30,000-person Phase III Covid-19 vaccine trial, with over a third of volunteers non-white and a quarter over the age of 65.

The announcement caps what has been the most closely-watched recruitment race in the history of drug development, as Pfizer and Moderna rushed to get enough volunteers to prove whether or not experimental vaccines could actually protect people from contracting Covid-19. Pfizer reached that mark on Sept. 15. Moderna said around the same time that they would slow down enrollment to ensure they enrolled enough participants from minority and at-risk groups.

News brief­ing: UK biotech 4D phar­ma heads for Nas­daq via SPAC; Dr. Red­dy's shuts down man­u­fac­tur­ing af­ter cy­ber­at­tack

Another pharma company is intending to use a SPAC to join the Nasdaq.

4D pharma, a UK-based biotech, is reverse-merging with a blank check company in a deal worth up to $37.6 million. The move will give 4D pharma a new Nasdaq ticker, which will be $LBPS, using the American Depositary Share program.

As a result of the move, 4D pharma will gain $14.6 million in cash held by the blank check company, dubbed Longevity. The merger is expected to be completed in early 2021, after which shares will be immediately tradeable.

Pfiz­er scoops up an an­tibi­ot­ic in rare M&A deal, bag­ging a vir­tu­al start­up op­er­at­ing on a shoe­string bud­get

Pfizer is stepping up with a rare antibiotics buyout deal today, grabbing Palo Alto, CA-based Arixa Pharmaceuticals in a bid to add a new oral version of avibactam, a beta lactamase inhibitor — or BLI — approved back in 2015 as part of the IV treatment Avycaz.

The Arixa acquisition follows some encouraging Phase I responses demonstrating that 60% to 80% of the oral drug is absorbed into the bloodstream. Only 7% of the IV version is absorbed orally, far below the 30% threshold Arixa has pointed to as a therapeutic threshold. The buyout gives Pfizer’s hospital group a line on a new oral combo with antibiotics like ceftibuten to go after drug-resistant cases of urinary tract infections and other ailments.

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John Maraganore, Alnylam CEO (Scott Eisen/Bloomberg via Getty Images)

With lumasir­an on the FDA's doorstep, Al­ny­lam reads out new PhI­II da­ta in PH1

Just over a month away from its December PDUFA date, Alnylam flaunted new data from two Phase III studies to back lumasiran in primary hyperoxaluria type 1 (PH1), a rare liver condition.

The Cambridge, MA-based biotech snagged a priority review for the candidate back in June, and got positive feedback from the EMA’s Committee for Medicinal Products for Human Use just last week. Lumasiran uses RNA interference (RNAi) to silence the gene for glycolate oxidase, an enzyme used in the production of oxalate.

Can B cells break the bound­aries of cell ther­a­py? Long­wood start­up has $52M to prove a new en­gi­neer­ing tech

Back in December 2017, as the cell therapy world was still basking in the virtually back-to-back approvals of two pioneering CAR-Ts, researchers at Seattle Children’s Research Institute reported a scientific first in a different corner of the field: engineer B cells to treat disease.

The team, led by David Rawlings and Richard James, eventually worked with Longwood Fund to start a biotech around those findings. And now Atlas Venture and RA Capital Management are coming on board to lead a $52 million launch round, joined by Alta Partners, for Be Biopharma.

Bo Cumbo, AavantiBio CEO (file photo)

Bo Cum­bo jumps from the top com­mer­cial post at Sarep­ta to the helm of a gene ther­a­py start­up with some in­flu­en­tial back­ers, big plans and $107M

After a 7-year stretch building the commercial team at Sarepta, longtime drug salesman Bo Cumbo is jumping to the entrepreneurial side of the business, taking the helm of a startup that’s got several deep-pocket investors. And he’s not just bringing his experience in selling drugs.

He tells me that when he told Sarepta CEO Doug Ingram about it, his boss got excited about the venture and opted to jump in with a $15 million investment from Sarepta to add to the launch money, alongside 3 of the busiest investors in biotech.

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HHS secretary Alex Azar (at the podium) and FDA commissioner Stephen Hahn (Pete Marovich/Getty Images)

Covid-19 roundup: Azar open­ly plan­ning Hahn ouster — re­port; Vul­ner­a­ble pop­u­la­tions like­ly to get vac­cines by Jan­u­ary

The relationship between HHS secretary Alex Azar and FDA commissioner Stephen Hahn has deteriorated to the point where Azar has suggested replacing Hahn, according to a Politico report.

Azar was angered by the FDA’s pushback of the Trump administration’s proposals for authorizing Covid-19 vaccines, so much so that he began openly floating potential replacements for Hahn. The report cited six unnamed sources that said Azar discussed bringing up Hahn’s removal to the White House.

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