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)

MedTech clinical trials require a unique regulatory and study design approach and so engaging a highly experienced CRO to ensure compliance and accurate data across all stages is critical to development milestones.

In­no­v­a­tive MedTech De­mands Spe­cial­ist Clin­i­cal Tri­al Reg­u­la­to­ry Af­fairs and De­sign

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Mathai Mammen (Rob Tannenbaum, Endpoints News at BIO 2018)

Math­ai Mam­men makes an abrupt ex­it as head of the big R&D group at J&J

In an after-the-bell shocker, J&J announced Monday evening that Mathai Mammen has abruptly exited J&J as head of its top-10 R&D group.

Recruited from Merck five years ago, where the soft-spoken Mammen was being groomed as the successor to Roger Perlmutter, he had been one of the top-paid R&D chiefs in biopharma. His group spent $12 billion last year on drug development, putting it in the top 5 in the industry.

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Robert Califf, FDA commissioner (Tom Williams/CQ Roll Call via AP Images)

Hop­ing to ex­pand mon­key­pox vac­cine sup­ply, US paves the way for new route of ad­min­is­tra­tion

After making it clear that the US’ current monkeypox vaccine supply is insufficient, the FDA on Tuesday authorized a new route of administration that should increase the number of available doses by five-fold.

Regulators cleared Bavarian Nordic’s Jynneos vaccine for intradermal injection in adults older than 18. Unlike subcutaneous injection — the current method by which vaccine is delivered under the skin — an intradermal jab goes directly into the skin. It’s believed that this method requires less vaccine, since the dermis is rich in dendritic cells which specialize in taking up foreign antigens and presenting them to the immune system, according to Daniel Kuritzkes, chief of infectious diseases at Brigham and Women’s Hospital in Boston.

Samantha Du, Zai Lab CEO

Any­one still look­ing for a CD47? Zai Lab shelves PhI pro­gram af­ter re­view­ing 'com­pet­i­tive land­scape'

Over the past few years, the promise of blocking CD47 — a “don’t eat me” signal co-opted by cancer cells — has sent drugmakers big and small into a frenzy. But one biotech is now bowing out.

Zai Lab is deprioritizing ZL-1201, its CD47 inhibitor, scrapping plans for a Phase II trial. It will now “pursue out-licensing opportunities,” the company said in its Q2 update. The decision was based on a review of the competitive landscape, it added, without going into further details.

Illustration: Kim Ryu for Endpoints News

Why non-opi­oid pain drugs keep fail­ing — and what's next for the field

In 1938, Rita Levi-Montalcini was forced to move her lab into her bedroom in Turin, as Mussolini’s facist government expelled Jewish people from studying or working in schools in Italy. Levi-Montalcini, then just a few years out of medical school and using sewing needles as scalpels in her makeshift lab, would soon discover nerve growth factor, or NGF, in chicken embryos.

Her discoveries formed the basis of our understanding of the peripheral nervous system and how cells talk to each other, and Levi-Montalcini went on to win the Nobel Prize in 1986. Much later, NGF was hailed as a promising target for new pain therapies, with some analysts quoting an $11 billion market. However, the latest anti-NGF candidate, Pfizer and Eli Lilly’s tanezumab, was rejected by the FDA last year because of a side effect that dissolved bone in some of its patients.

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Ted Love, Global Blood Therapeutics CEO

Up­dat­ed: Pfiz­er scoops up Glob­al Blood Ther­a­peu­tics and its sick­le cell ther­a­pies for $5.4B

Pfizer is dropping $5.4 billion to acquire Global Blood Therapeutics.

Just ahead of the weekend, word got out that Pfizer was close to clinching a $5 billion buyout — albeit with other potential buyers still at the table. The pharma giant, flush with cash from Covid-19 vaccine sales, apparently got out on top.

The deal immediately swells Pfizer’s previously tiny sickle cell disease portfolio from just a Phase I program to one with an approved drug, Oxbryta, plus a whole pipeline that, if all approved, the company believes could make for a $3 billion franchise at peak.

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Steve Paul, Karuna Therapeutics CEO (Third Rock)

Karuna's schiz­o­phre­nia drug pass­es a close­ly-watched PhI­II test, will head to FDA in mid-2023

An investigational pill that combines a former Eli Lilly CNS compound with an overactive bladder drug was better than placebo at reducing a scale of symptoms experienced by patients with schizophrenia in a Phase III trial.

Karuna Therapeutics’ drug passed the primary goal in EMERGENT-2, the Boston biotech said early Monday morning, alongside quarterly earnings. The study is the first of Karuna’s four Phase III clinical trials to read out in schizophrenia and will provide the backbone to the biotech’s first drug approval application, slated for mid-2023.

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HHS Secretary Xavier Becerra (Patrick Semansky/AP Images)

US weighs new route of ad­min­is­tra­tion for mon­key­pox vac­cine as cas­es climb — re­port

Less than a week after HHS Secretary Xavier Becerra declared monkeypox a national health emergency, reports have emerged that the US plans to extend its vaccine supply by opting for a different route of administration.

Officials are expected to call for intradermal injection of Bavarian Nordic’s Jynneos vaccine — the only shot approved specifically for monkeypox in the US — as opposed to subcutaneous injection, unnamed sources told both the New York Times and Washington Post on Tuesday.

'Messy at best': Is the US re­peat­ing the same Covid mis­steps with mon­key­pox mes­sag­ing?

When Kyle Planck first suspected he might have monkeypox in late June, he went to the CDC website and found six photos of different types of lesions. And that was about it for general public information.

Planck, who is a sixth-year PhD pharmacology researcher at Weill Cornell, kept looking though and found a separate part of the CDC website meant for healthcare professionals. There he found a medical slide deck with more pictures, professional journal articles and more details about symptoms and diagnosis.

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