New re­port her­alds a rev­o­lu­tion in can­cer R&D trends - and the stick­er shock that comes with it

There’s a rev­o­lu­tion play­ing out in can­cer R&D.

Over the last decade drug de­vel­op­ers fo­cused on on­col­o­gy have teamed with reg­u­la­tors on de­sign­ing a short­er R&D path for new can­cer drugs, us­ing tri­al de­signs that have sped clin­i­cal work and slashed the num­ber of pa­tients need­ed to demon­strate safe­ty and ef­fi­ca­cy.

With the num­ber of new check­point com­bos in the clin­ic es­ca­lat­ing rapid­ly, the late-stage pipeline of new can­cer drugs has ex­plod­ed, grow­ing 7.7% in just the last year to 631 new mol­e­cules. The two ini­tial PD-1 ap­provals of Op­di­vo and Keytru­da alone spawned 135 com­bi­na­tion stud­ies. And the work over­all is chang­ing the stan­dard of care in the field — at a high cost for the pay­ers foot­ing the bill.

Those are all con­clu­sions laid out in a new re­port from Quin­tiles­IMS, which con­ve­nient­ly de­tailed the ma­jor trends in can­cer R&D just a cou­ple of days ahead of the bell­wether AS­CO con­fer­ence in Chica­go.

Over the past 5 years, 68 new can­cer drugs have been ap­proved, mark­ing a sig­nif­i­cant im­prove­ment in out­comes for pa­tients, par­tic­u­lar­ly in the area of metasta­t­ic dis­ease, notes Mur­ray Aitken, the au­thor of the re­port and ex­ec­u­tive di­rec­tor of the Quin­tiles­IMS In­sti­tute.

Mur­ray Aitken

Aitken high­lights the role of the FDA’s 2012 Break­through Ther­a­py Des­ig­na­tion ini­tia­tive in help­ing change the path of can­cer drug R&D, along with read­ier use of fast track sta­tus and pri­or­i­ty re­views. With reg­u­la­tors will­ing to as­sist rapid drug de­vel­op­ment plans, the num­ber of pa­tients en­rolled in Phase III stud­ies dropped from 671 18 years ago to 188 in 2016 as in­ves­ti­ga­tors fo­cused on small­er sub­pop­u­la­tions.

Tri­al du­ra­tion in Phase III has been halved, drop­ping from 2000 days in the late ‘90s to a lit­tle more than 1000.

Drug de­vel­op­ers haven’t over­looked the op­por­tu­ni­ty. The num­ber of new drugs in the can­cer pipeline has swelled by 45% over the past decade, with a grow­ing num­ber of these ex­per­i­men­tal drugs us­ing bio­mark­ers to pre­dict pa­tient re­spons­es; 87% of these drugs count as tar­get­ed ther­a­pies.

None of this is free.

Glob­al spend­ing on can­cer drugs swelled from $107 bil­lion to $113 bil­lion in just one year. Over the past 5 years the an­nu­al growth rate in spend­ing on can­cer ther­a­pies has been 8.7% per year, sig­nif­i­cant­ly high­er than the 4.9% rate of the 5 years pri­or to that. And by 2021 to­tal glob­al spend­ing on can­cer drugs and sup­port­ive care will hit $147 bil­lion.

“We are see­ing pric­ing con­ces­sions,” Aitken told re­porters in a con­fer­ence call on Wednes­day, in­tro­duc­ing the re­port. Those con­ces­sions have re­duced the lev­el of net price growth for can­cer drugs to 3.6% last year, down from the 5% rate they record­ed in 2014 and 2015.

As new com­bi­na­tion drugs wind their way through the clin­ic, there’s been con­sid­er­able un­ease in bio­phar­ma about the cost im­pli­ca­tions of meld­ing ex­pen­sive new ther­a­pies in­to one pow­er­ful cock­tail. That de­bate is just be­gin­ning.

Aerial view of Genentech's campus in South San Francisco [Credit: Getty]

Genen­tech sub­mits a big plan to ex­pand its South San Fran­cis­co foot­print

The sign is still there, a quaint reminder of whitewashed concrete not 5 miles from Genentech’s sprawling, chrome-and-glass campus: South Francisco The Industrial City. 

The city keeps the old sign, first erected in 1923, as a tourist site and a kind of civic memento to the days it packed meat, milled lumber and burned enough steel to earn the moniker “Smokestack of the Peninsula.” But the real indication of where you are and how much has changed both in San Francisco and in the global economy since a couple researchers and investors rented out an empty warehouse 40 years ago comes in a far smaller blue sign, resembling a Rotary Club post, off the highway: South San Francisco, The Birthplace of Biotech.

Here comes the oral GLP-1 drug for di­a­betes — but No­vo Nordisk is­n't dis­clos­ing Ry­bel­sus price just yet

Novo Nordisk’s priority review voucher on oral semaglutide has paid off. The FDA approval for the GLP-1 drug hit late Friday morning, around six months after the NDA filing.

Rybelsus will be the first GLP-1 pill to enter the type 2 diabetes market — a compelling offering that analysts have pegged as a blockbuster drug with sales estimates ranging from $2 billion to $5 billion.

Ozempic, the once-weekly injectable formulation of semaglutide, brought in around $552 million (DKK 3.75 billion) in the first half of 2019.

As Nas­daq en­rolls the fi­nal batch of 2019 IPOs, how have the num­bers com­pared to past years?

IGM Biosciences’ upsized IPO haul, coming after SpringWorks’ sizable public debut, has revved up some momentum for the last rush of biotech IPOs in 2019.

With 39 new listings on the books and roughly two more months to go before winding down, Nasdaq’s head of healthcare listings Jordan Saxe sees the exchange marking 50 to 60 biopharma IPOs for the year.

“December 15 is usually the last possible day that companies will price,” he said, as companies get ready for business talks at the annual JP Morgan Healthcare Conference in January.

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Oxitec biologist releases genetically modified mosquitoes in Piracicaba, Brazil in 2016 [credit: Getty Images]

In­trex­on unit push­es back against claims its GM mos­qui­toes are mak­ing dis­ease-friend­ly mu­tants

When the hysteria of Zika transmission sprang into the American zeitgeist a few years ago, UK-based Oxitec was already field-testing its male Aedes aegypti mosquito, crafted to possess a gene engineered to obliterate its progeny long before maturation.

But when a group of independent scientists evaluated the impact of the release of these genetically-modified mosquitoes in a trial conducted by Oxitec in Brazil between 2013 and 2015, they found that some of the offspring had managed to survive — prompting them to speculate what impact the survivors could have on disease transmission and/or insecticide resistance.

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[via AP Images]

Pur­due threat­ens to walk away from set­tle­ment, asks to pay em­ploy­ees mil­lions in bonus­es

There are two updates on the lawsuit against Purdue Pharma over its role in fueling the opioid epidemic, as the Sackler family threatens to walk away from their pledge to pay out $3 billion if a bankruptcy judge does not stop outstanding state lawsuits against them. At the same time, the company has asked permission to pay millions in bonuses to select employees.

Purdue filed for chapter 11 bankruptcy this week as part of its signed resolution to over 2,000 lawsuits. The deal would see the Sackler family that owns Purdue give $3 billion from their personal wealth and the company turned into a trust committed to curbing and reversing overdoses.

David Grainger [file photo]

'Dis­con­nect the bas­tard­s' — one biotech's plan to break can­cer cell­s' uni­fied de­fens­es

Chemotherapy and radiotherapy are the current gladiators of cancer treatment, but they come with well-known limitations and side-effects. The emergence of immunotherapy — a ferocious new titan in oncologist’s toolbox — takes the brakes off the immune system to kill cancer cells with remarkable success in some cases, but the approach is not always effective. What makes certain forms of cancer so resilient? Scientists may have finally pieced together a tantalizing piece of the puzzle, and a new biotech is banking on a new approach to fill the gap.

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A fa­vorite in Alex­ion’s C-suite is leav­ing, and some mighty sur­prised an­a­lysts aren’t the least bit hap­py about it

Analysts hate to lose a biotech CFO they’ve come to trust and admire — especially if they’re being blindsided by a surprise exit.

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Scott Gottlieb, AP Images

Scott Got­tlieb is once again join­ing a team that en­joyed good times at the FDA un­der his high-en­er­gy stint at the helm

Right after jumping on Michael Milken’s FasterCures board on Monday, the newly departed FDA commissioner is back today with news about another life sciences board post that gives him a ringside chair to cheer on a lead player in the real-world evidence movement — one with very close ties to the FDA.

Aetion is reporting this morning that Gottlieb is joining their board, a group that includes Mohamad Makhzoumi, a general partner at New Enterprise Associates, where Gottlieb returned after stepping out of his role at the FDA 2 years after he started.

Gottlieb — one of the best connected execs in biopharma — knows this company well. As head of FDA he championed the use of real-world evidence to help guide drug developers and the agency in gaining greater efficiencies, which helped set up Aetion as a high-profile player in the game.

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Tower Bridge in London [Shutterstock]

#UK­BIO19: Join GSK’s Hal Bar­ron and a group of top biotech ex­ecs for our 2nd an­nu­al biotech sum­mit in Lon­don

Over the past 10 years I’ve made a point of getting to know the Golden Triangle and the special role the UK biopharma industry plays there in drug development. The concentration of world class research institutes, some of the most accomplished scientists I’ve ever seen at work and a rising tide of global investment cash leaves an impression that there’s much, much more to come as biotech hubs are birthed and nurtured.