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)

Big Phar­ma's Twit­ter ex­o­dus; Mer­ck wa­gers $1.35B on buy­out; $3.5M gene ther­a­py; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

As you start planning for #JPM23, we hope you will consider joining Endpoints News for our live and virtual events. For those who are celebrating Thanksgiving, we hope you are enjoying the long weekend with loved ones. And if you’re not — we’ll see you next week!

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Paul Perreault, CSL Behring CEO

CSL lands FDA ap­proval for he­mo­phil­ia B gene ther­a­py, sets $3.5M list price

The FDA has approved the world’s first gene therapy for hemophilia B, ushering into the market a treatment that’s historic in both what it promises to do and how much it will cost.

CSL will be marketing the drug, Hemgenix, at a list price of $3.5 million — which sets a new record for the most expensive single-use gene therapy in the US.

In a statement provided to Endpoints News, the Australian company noted that the current costs of treating people with moderate to severe hemophilia B can be significant over a lifetime. By some estimates, healthcare systems could spend more than $20 million per person.

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Image: Shutterstock

MIT re­searchers re­veal DNA "Paste" tech be­hind lat­est gene edit­ing start­up

MIT scientists have developed a tool that they say can insert large gene sequences where they want in the genome.

In a paper published Thursday in Nature Biotechnology, MIT fellows Omar Abudayyeh, Jonathan Gootenberg and colleagues detail a technology they call PASTE, which they say can potentially be used to insert long strands of DNA and treat genetic diseases caused by many different mutations, such as cystic fibrosis and Leber congenital amaurosis, a rare eye disorder that causes blindness.

Elon Musk (GDA via AP Images)

Biggest drug com­pa­nies halt­ed Twit­ter ad buys af­ter Lil­ly in­sulin spoof

Almost all of the drug industry’s biggest advertisers cut their spending on Twitter to zero or near-zero over the last two weeks amid worries about impersonation of their brands by pranksters and the future of the social media company.

Among 18 of the biggest pharmaceutical advertisers in the US market, 12 cut their Twitter ad spending to nothing for the week beginning Nov. 14, according to Pathmatics, which tracks data on prescription drug ad spending as well as general corporate advertising. The list of drugmakers cutting spending to zero includes Merck, AstraZeneca, Eli Lilly, Novartis, Pfizer and others.

J&J's Spra­va­to pulls a PhI­II win against Sero­quel XR in treat­ment-re­sis­tant de­pres­sion

A day before Thanksgiving, J&J’s Janssen has a new cut of Phase III Spravato data to be grateful for.

The pharma giant announced on Wednesday that its nasal spray, also known as esketamine, beat extended-release quetiapine, previously sold by AstraZeneca as Seroquel XR, in treatment-resistant depression (TRD). Of 676 adults, a significantly higher number of patients on Spravato were able to achieve remission and avoid relapse after 32 weeks, according to J&J.

Rob Davis, Merck CEO

Up­dat­ed: No Seagen here: 'Do more' means a small $1.35B pur­chase of Ima­go for Mer­ck

Merck is making an acquisition, the Big Pharma announced before Monday’s opening bell. No, Seagen is not entering the fold, as had been speculated for quarters.

Folding under Merck’s wings will be Pfizer-backed Imago BioSciences. For nearly a year, Merck CEO Rob Davis has been saying the pharma giant needs to “do more” on the business development front after its 2021 $11.5 billion acquisition of Acceleron.

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Isao Teshirogi, Shionogi president and CEO (Kyodo via AP Images)

Sh­ionogi's Covid an­tivi­ral lands first ap­proval in Japan's new emer­gency ap­proval path­way

Japanese regulators on Tuesday signed off on Shionogi’s homegrown antiviral for Covid-19, known as Xocova (ensitrelvir), making it the first approval under Japan’s emergency regulatory approval system.

The emergency approval, following a back-and-forth with regulators since last February, is based on a safety profile with more than 2,000 patients who have accessed the pill, and clinical symptomatic efficacy for five typical Omicron-related symptoms (primary endpoint) and antiviral efficacy (key secondary endpoint) in patients with mild to moderate SARS-CoV-2 infection, regardless of risk factors or vaccination status, and during the Omicron-dominant phase of the pandemic.

Dermavant Sciences' first consumer TV ad for its Vtama psoriasis med shows people ready for a new topical treatment.

Roivant’s Der­ma­vant de­buts first-ever TV com­mer­cial for pso­ri­a­sis cream Vta­ma

Dermavant Sciences has been marketing its first product, psoriasis med Vtama, to dermatologists for months, but on Tuesday it rolled out its first consumer campaign. The debut DTC effort including a streaming TV commercial encourages patients to a “Topical Uprising” in a nod to Vtama being a topical cream.

In the new commercial, a swell of people discards scarves and jacket coverings, gathering in the street to converge on a pharmacy to demand a steroid-free prescription. A moment of levity follows when a pharmacist says, “You know you can just talk to your doctor, right?” The gathered crowds collectively says, “Oh.”

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FDA preps for DMD drug gener­ics as Sarep­ta has yet to fin­ish its con­fir­ma­to­ry tri­al

The FDA typically releases guidance to help generic drug manufacturers develop new copycats of small molecule drugs, oftentimes in preparation for a brand name product’s patents or exclusivity to expire.

This week, FDA released such bioequivalence guidance for any generic drugmakers looking to take on Sarepta’s Duchenne muscular dystrophy (DMD) drug Exondys 51 (eteplirsen), even though the drug’s sponsor has yet to convert the accelerated approval to a full approval, showing clinical benefit.