King can­cer: The top 10 ther­a­peu­tic ar­eas in bio­phar­ma R&D

It’s not go­ing to come as a sur­prise to any­one who’s been pay­ing at­ten­tion to drug R&D trends that can­cer is the num­ber 1 dis­ease in terms of new drug de­vel­op­ment projects. But it is amaz­ing to see ex­act­ly how much on­col­o­gy dom­i­nates the in­dus­try as nev­er be­fore.

At a time the first CAR-T looks to be on the thresh­old of a pi­o­neer­ing ap­proval and the first wave of PD-(L)1 drugs are spurring hun­dreds of com­bi­na­tion stud­ies, can­cer ac­count­ed for 8,651 of the to­tal num­ber of pipeline projects count­ed by the Analy­sis Group, crunch­ing the num­bers in a new re­port com­mis­sioned by PhRMA. That’s more than a third of the 24,389 pre­clin­i­cal through Phase III pro­grams tracked by Eval­u­atePhar­ma, which pro­vid­ed the data­base for this re­view.

That’s al­so more than the next 5 dis­ease fields com­bined, start­ing with num­ber 2, neu­rol­o­gy — a field that in­cludes Parkin­son’s and Alzheimer’s. Psy­chi­a­try, once a ma­jor fo­cus for phar­ma R&D, didn’t even make the top 10, with 468 projects.

Mov­ing down­stream, can­cer stud­ies are over­whelm­ing­ly in the lead. Sin­gling out Phase I projects, can­cer ac­count­ed for 1,757 out of a to­tal of 3,723 ini­tia­tives, close to half. In Phase II it’s the fo­cus of 1,920 of 4,424 projects. On­ly in late-stage stud­ies does can­cer start to lose its over­whelm­ing dom­i­nance, falling to 329 of 1,257 projects.

PhRMA com­mis­sioned this re­port to un­der­score just how much the in­dus­try is com­mit­ted to R&D and sig­nif­i­cant new drug de­vel­op­ment, a sub­ject that rou­tine­ly comes in­to ques­tion as an­a­lysts eval­u­ate how much mon­ey is de­vot­ed to de­vel­op­ing new drugs in­stead of, say, mar­ket­ing or share buy­backs.

The re­port makes a few oth­er points to un­der­score the na­ture of the work these days.

— Three out of four projects in the clin­ic were an­gling for first-in-class sta­tus, spot­light­ing the em­pha­sis on ad­vanc­ing new med­i­cines that can make a dif­fer­ence for pa­tients. Me-too drugs are com­plete­ly out of fash­ion, un­like­ly to com­mand much weight with pay­ers.

— Of all the projects in clin­i­cal de­vel­op­ment, 822 were for or­phan drugs look­ing to serve a mar­ket of 200,000 or less. Or­phan drugs have per­formed well, able to com­mand high prices and ben­e­fit­ing from in­cen­tives un­der fed­er­al law.

— There were 731 cell and gene ther­a­py projects in the clin­ic, with bio­phar­ma look­ing at pi­o­neer­ing ap­provals in CAR-T, with No­var­tis and Kite, as well as the first US OK for a gene ther­a­py, with the first ap­pli­ca­tion ac­cept­ed this week for a pri­or­i­ty re­view of a new ther­a­py from Spark Ther­a­peu­tics.


Dis­tri­b­u­tion of prod­ucts and projects by ther­a­peu­tic area and phase


Source: Analy­sis Group, us­ing Eval­u­atePhar­ma da­ta


Unique NMEs in de­vel­op­ment by stage (Au­gust 2016)

Paul Hudson, Getty Images

UP­DAT­ED: Sanofi CEO Hud­son lays out new R&D fo­cus — chop­ping di­a­betes, car­dio and slash­ing $2B-plus costs in sur­gi­cal dis­sec­tion

Earlier on Monday, new Sanofi CEO Paul Hudson baited the hook on his upcoming strategy presentation Tuesday with a tell-tale deal to buy Synthorx for $2.5 billion. That fits squarely with hints that he’s pointing the company to a bigger future in oncology, which also squares with a major industry tilt.

In a big reveal later in the day, though, Hudson offered a slate of stunners on his plans to surgically dissect and reassemble the portfoloio, saying that the company is dropping cardio and diabetes research — which covers two of its biggest franchise arenas. Sanofi missed the boat on developing new diabetes drugs, and now it’s pulling out entirely. As part of the pullback, it’s dropping efpeglenatide, their once-weekly GLP-1 injection for diabetes.

“To be out of cardiovascular and diabetes is not easy for a company like ours with an incredibly proud history,” Hudson said on a call with reporters, according to the Wall Street Journal. “As tough a choice as that is, we’re making that choice.”

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Psilocybin mushrooms (via The Denver Post)

In a key step for psy­che­del­ic re­search, mag­ic mush­room com­pound clears first clin­i­cal safe­ty hur­dle

Exasperated with the often-ineffective existing slate of antidepressants, COMPASS Pathways set up shop in London 2016 — and made a beeline for psilocybin, the psychoactive ingredient in magic mushrooms.

On Wednesday, the startup said its man-made version of the chemical — which is illegal across geographies in its natural fungi form — had been well-tolerated in an early-stage, placebo-controlled trial in 89 healthy volunteers.

Al­pham­ab On­col­o­gy rounds out HKEX's sec­ond biotech IPO year with $230M raise and high lo­cal in­ter­est

Alphamab Oncology has inspired a surge of local interest in what will likely be the Hong Kong Stock Exchange’s last biotech run of the year, pricing its IPO on the high end of the range and raising over $230 million (HK$1.83 billion).

After rejigging the offering structure and making up to 50% available for enthusiastic local investors, the biotech sold 179.4 million shares at $1.31 (HK$10.2) and saw its stock rise to $1.77 ($13.8) on the first day of trading.

For sale: Long-act­ing PhI­II GLP-1 di­a­betes drug that’s way be­hind ri­vals, now spurned by Sanofi

Almost exactly 4 years ago Sanofi came to the bargaining table with South Korea’s Hanmi bearing $434 million dollars in cash and offering about $4 billion in milestones to in-license their once-weekly GLP-1 injectable. The pact was intended to revive their ailing diabetes division. Instead, it turned into a very expensive grave to mark the end of Sanofi’s R&D ambitions in the field.

Sanofi CEO Paul Hudson used efpeglenatide’s demise — while committing to paying hundreds of millions of more dollars to push it through 5 late-stage studies — as a marker of the company’s determination to stay focused on first and best-in-class drugs.

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Parkin­son's trans­plants emerge as stem cell pi­o­neer Jeanne Lor­ing joins race

Jeanne Loring hadn’t studied Parkinson’s in 22 years when she got an email from a local neurologist.

The neurologist, Melissa Houser, didn’t know Loring had ever published on the disease. She was just looking for a stem cell researcher who might hear her out. 

“I think I was just picked out a hat,” Loring told Endpoints News. 

At a meeting in Loring’s Scripps Research office, Houser and a Parkinson’s nurse practitioner, Sherrie Gould, asked her why there was so much research done in stem cell transplants for other neurodegenerative diseases but not Parkinson’s. They wanted to know if she would work on one. 

What does $6.9B buy these days in on­col­o­gy R&D? As­traZeneca has a land­mark an­swer

Given the way the FDA has been whisking through new drug approvals months ahead of their PDUFA date, AstraZeneca and their partners Daiichi Sankyo may not have to wait until Q2 of next year to get a green light on trastuzumab deruxtecan (DS-8201).

The pharma giant this morning played their ace in the hole, showing off why they were willing to commit to a $6.9 billion deal — with $1.35 billion in a cash upfront — to partner on the drug.

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Paul Hudson, Sanofi

Paul Hud­son promis­es a bright new fu­ture at Sanofi, kick­ing loose me-too drugs and fo­cus­ing on land­mark ad­vances. But can he de­liv­er?

Paul Hudson was on a mission Tuesday morning as he stood up to address Sanofi’s new R&D and business strategy.

Still fresh into the job, the new CEO set out to convince his audience — including the legions of nervous staffers inevitably devoting much of their day to listening in — that the pharma giant is shedding the layers of bureaucracy that had held them back from making progress in the past, dropping the duds in the pipeline and reprioritizing a more narrow set of experimental drugs that were promised as first-in-class or best-in-class.  The company, he added, is now positioned to “go after other opportunities” that could offer a transformational approach to treating its core diseases.

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Large advertisements for the drug Vivitrol decorate the walls of Grand Central Station on June 15, 2017 in New York City. (Photo: Andrew Lichtenstein via Getty)

FDA slaps down Alk­er­mes for mis­lead­ing Viv­it­rol ads — don't for­get vul­ner­a­bil­i­ty to opi­oid over­dose

The ads piqued interest as soon as they started appearing in 2016: at Grand Central Station, on the Red Line in Cambridge, and on a billboard off the New Jersey Turnpike. All showed a young person, generally with his or her arms crossed, and the question, “what is Vivitrol?”

Vivitrol’s maker, Alkermes, was in the midst of a marketing and lobbying campaign to promote the anti-opioid addiction drug — a campaign that would face significant backlash for tarnishing competitors despite little evidence for Vivitrol’s superiority.

FDA in-house re­view spot­lights an is­sue with one of Hori­zon's end­points but notes ef­fi­ca­cy for lead drug

The FDA in-house review highlights a disagreement of investigators’ use of a key endpoint by Horizon Pharma in the late-stage trial for the top drug in its pipeline, but largely agreed that the antibody was effective.

Horizon submitted a BLA for thyroid eye disease (TED) drug teprotumumab in March, less than two years after they bought the drug (and the rest of a division) from Narrow River for $145 million upfront. With breakthrough status, priority review, orphan designation and in-house sales projections of up to $750 million, the one-time Roche reject became the marquee pipeline asset for a company that’s developed some of the world’s most expensive drugs.