Gala­pa­gos, Ab­b­Vie glimpse ear­ly progress in search of a cys­tic fi­bro­sis triple to ri­val Ver­tex

Piet Wiger­inck, Gala­pa­gos. Cred­it: Fe­lix Kalk­man

Ab­b­Vie $AB­BV and its part­ner Gala­pa­gos $GLPG are pleased with some of the ear­ly da­ta they’re see­ing for one piece of their triple drug puz­zle for cys­tic fi­bro­sis. And they’re plan­ning to give Ver­tex a run for its mon­ey on this front.

In a small study in­volv­ing 26 pa­tients with the G551D mu­ta­tion in CFTR, a dose-es­ca­lat­ing reg­i­men of the CFTR po­ten­tia­tor GLPG1837 pro­duced a sta­tis­ti­cal­ly sig­nif­i­cant drop in sweat chlo­ride con­cen­tra­tion.

That is an ear­ly snap­shot they need to con­tin­ue to blaze a trail for a new cys­tic fi­bro­sis cock­tail ther­a­py, tak­ing on mar­ket leader Ver­tex and its pi­o­neer­ing drug Ka­ly­de­co as it lines up a triple stan­dard of its own.

Just a month ago the two part­ners launched a Phase I study of their nov­el C2 cor­rec­tor GLPG2737, the first of many C2 cor­rec­tors be­ing de­vel­oped and “the fi­nal com­po­nent need­ed to com­plete a first triple com­bi­na­tion.” Po­ten­tia­tors GLPG1837 and GLPG2451 and C1 cor­rec­tor GLPG2222 are all in the clin­ic as they sort out the best can­di­dates for a cock­tail.

Set­ting up a ri­val­ry with Ver­tex, though, is prob­lem­at­ic at best. Bar­clays not­ed:

Over­all, we see these da­ta as in­ter­est­ing, yet high­light the ear­ly na­ture of the pro­gram and be­lieve the de­vel­op­ment time­line of a Gala­pa­gos triple com­bi­na­tion is ag­gres­sive with clin­i­cal tri­als slat­ed to be­gin 3Q17e. Even if Gala­pa­gos is able to main­tain time­lines, Ver­tex is rapid­ly pro­gress­ing with next-gen cor­rec­tors VX-661 in com­bi­na­tion with Ka­ly­de­co and VX-440 and VX-152 as part of a triple com­bi­na­tion, which could be­come new stan­dards. While to­day’s da­ta rep­re­sent a step for­ward for Gala­pa­gos and de­vel­op­ment part­ner Ab­b­Vie, it re­mains con­sid­er­ably ear­ly to eval­u­ate the po­ten­tial of ‘1837 as a com­peti­tor to Ver­tex.

Ab­b­Vie and Gala­pa­gos, though, seem de­ter­mined to give it a try.

“The suc­cess of this tri­al is an im­por­tant mile­stone in two re­gards; first­ly, GLPG1837 has shown safe­ty and sig­nif­i­cant ef­fi­ca­cy as a nov­el CFTR po­ten­tia­tor. Sec­ond­ly, it demon­strates that the CF com­mu­ni­ty is com­mit­ted to the fur­ther de­vel­op­ment of CFTR mod­u­la­tors de­spite the com­plex­i­ties re­lat­ed to evolv­ing stan­dards of care,” com­ment­ed Jane Davies of the Roy­al Bromp­ton & Hare­field NHS Trust in Lon­don and prin­ci­pal in­ves­ti­ga­tor for SAPHI­RA 1.

“The SAPHI­RA 1 re­sults show this is the first new po­ten­tia­tor since Ka­ly­de­co to demon­strate com­pet­i­tive re­sults in pa­tients har­bor­ing the G551D mu­ta­tion. Gala­pa­gos has a suite of po­ten­tia­tors in de­vel­op­ment. Gala­pa­gos and Ab­b­Vie will fur­ther study the da­ta be­fore de­cid­ing which po­ten­tia­tor will be in­clud­ed in the triple com­bi­na­tion,” said Dr Piet Wiger­inck, CSO of Gala­pa­gos. “The clin­i­cal val­i­da­tion of our in vit­ro sys­tems re­in­forces our be­lief in our ap­proach to get to a triple com­bi­na­tion ther­a­py.”

Regeneron CEO Leonard Schleifer speaks at a meeting with President Donald Trump, members of the Coronavirus Task Force, and pharmaceutical executives in the Cabinet Room of the White House (AP Photo/Andrew Harnik)

OWS shifts spot­light to drugs to fight Covid-19, hand­ing Re­gen­eron $450M to be­gin large scale man­u­fac­tur­ing in the US

The US government is on a spending spree. And after committing billions to vaccines defense operations are now doling out more of the big bucks through Operation Warp Speed to back a rapid flip of a drug into the market to stop Covid-19 from ravaging patients — possibly inside of 2 months.

The beneficiary this morning is Regeneron, the big biotech engaged in a frenzied race to develop an antibody cocktail called REGN-COV2 that just started a late-stage program to prove its worth in fighting the virus. BARDA and the Department of Defense are awarding Regeneron a $450 million contract to cover bulk delivery of the cocktail starting as early as late summer, with money added for fill/finish and storage activities.

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The home run count: The $100M+ mega-round boom in biotech in­spired a $6.7B feed­ing fren­zy — so far this year

Over the last 6 months there’s been a blizzard of money piling up drifts of the green stuff through the biotech landscape. And the forecast calls for more cash windfalls ahead.

Even as a global pandemic has killed more than half a million people, blighted economies and divided nations over the proper response, it’s also helped ignite an unprecedented burst of big-time investing. And not just in Covid-19 deals, as we’ve looked at before.

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UP­DAT­ED: Bio­gen shares spike as ex­ecs com­plete a de­layed pitch for their con­tro­ver­sial Alzheimer's drug — the next move be­longs to the FDA

Biogen is stepping out onto the high wire today, reporting that the team working on the controversial Alzheimer’s drug aducanumab has now completed their submission to the FDA. And they want the agency to bless it with a priority review that would cut the agency’s decision-making time to a mere 6 months.

The news drove a 10% spike in Biogen’s stock $BIIB ahead of the bell.

Part of that spike can be attributed to a relief rally. Biogen execs rattled backers and a host of analysts earlier in the year when they unexpectedly delayed their filing to the third quarter. That delay provoked all manner of speculation after CEO Michel Vounatsos and R&D chief Al Sandrock failed to persuade influential observers that the pandemic and other factors had slowed the timeline for filing. Actually making the pitch at least satisfies skeptics that the FDA was not likely pushing back as Biogen was pushing in. From the start, Biogen execs claimed that they were doing everything in cooperation with the FDA, saying that regulators had signaled their interest in reviewing the submission.

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Atul Deshpande, Harbour BioMed chief strategy officer & head, US operations (Harbour BioMed)

An­oth­er biotech IPO set-up? Multi­na­tion­al biotech leaps from round to round, scoop­ing up cash at a blis­ter­ing pace

A short four months after announcing a $75 million haul in Series B+ fundraising, the multinational biotech Harbour BioMed pulled in another round of investments and eclipsed the nine-digit mark in the process.

Harbour completed its Series C financing, the company announced Thursday morning, raising $102.8 million and bringing its total investment sum to over $300 million since its founding in late 2016. The biotech plans to use the money to transition early-stage candidates from the discovery phase, fund candidates already in the clinic, and prep late-stage candidates for commercialization.

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For­bion spot­lights late-stage plays, carves out new €250M growth fund

Having staked its rep on picking out a mix of biotech investment opportunities across the “build,” “enable,” “growth” continuum, Forbion is launching its first fund dedicated to late-stage opportunities.

Forbion Growth Opportunities Fund’s first close brought in €185 million ($208 million). Existing investors Pantheon, KfW Capital and the European Investment Fund came on board, joined by new backers Eli Lilly, Horizon Therapeutics, Belgian Growth Fund and New Waves Investments.

Mer­ck ex­pands scope of Zymeworks an­ti­body al­liance, adding close to $900M in mile­stones

Nearly a decade after first partnering with Merck, Vancouver-based biotech Zymeworks has expanded its collaboration with the pharma giant once again.

Zymeworks re-upped with Merck in a new licensing agreement, granting the New Jersey pharma giant the right to develop up to 3 additional multispecific antibody candidates. In exchange, the biotech will receive an undisclosed upfront payment — Merck is always loath to discuss cash terms — and nearly $900 million in combined regulatory ($411 million) and commercial ($480 million) milestones.

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Daniel O'Day, Gilead CEO (Kevin Dietsch/UPI/Bloomberg via Getty Images)

A new study points to $6.5B in pub­lic sup­port build­ing the sci­en­tif­ic foun­da­tion of Gilead­'s remde­sivir. Should that be re­flect­ed in the price?

By drug R&D standards, Gilead’s move to repurpose remdesivir for Covid-19 and grab an emergency use authorization was a remarkably easy, low-cost layup that required modest efficacy and a clean safety profile from just a small group of patients.

The drug OK also arrived after Gilead had paid much of the freight on getting it positioned to move fast.

In a study by Fred Ledley, director of the Center for Integration of Science and Industry at Bentley University in Waltham, MA, researchers concluded that the NIH had invested only $46.5 million in the research devoted to the drug ahead of the pandemic, a small sum compared to the more than $1 billion Gilead expected to spend getting it out this year, all on top of what it had already cost in R&D expenses.

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Ed Engleman (Stanford Blood Center)

Stan­ford star on­col­o­gy sci­en­tist Ed En­gle­man helped cre­ate the im­munother­a­py field. Now he wants to shake up neu­rode­gen­er­a­tion R&D

Over the last generation of drug R&D, Ed Engleman has been a standout scientist.

The Stanford professor co-founded Dendreon and provided the scientific insights needed to develop Provenge into a pioneering — though not particularly marketable — immunotherapy. He’s spurred a slate of startups, assisted by his well-connected perch as a co-founder of Vivo Capital, and took the dendritic cell story into its next chapter at a startup called Bolt.

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Nello Mainolfi (Kymera via YouTube)

Out to re­vive R&D, a resur­gent Sanofi pays $150M cash to part­ner up with a pi­o­neer­ing pro­tein degra­da­tion play­er

Frank Nestle was appointed Sanofi’s global head of immunology and inflammation research therapeutic area just days before dupilumab, the blockbuster-to-be IL-4 antibody, would be accepted for priority review. After four years of consolidating immunology expertise from multiple corners of the Sanofi family and recruiting new talents to build the discovery engine, he’s set eyes on a Phase I-ready program that he believes can grow into a Dupixent-sized franchise.