Lib­er­tar­i­an ideas on FDA dereg­u­la­tion threat­en biotech; It's time for some re­al pow­er to re­form drug prices, rea­son­ably

Do you sin­cere­ly want to dereg­u­late drug de­vel­op­ment?

Sil­i­con Val­ley’s Bal­a­ji Srini­vasan had quite a habit of tack­ling the FDA on Twit­ter, crit­i­ciz­ing the agency for its slow and back­ward ways. But all that came back to haunt him af­ter re­porters picked up on the news that he and fel­low ‘seast­ead­er’ Jim O’Neill had been in to chat with Don­ald Trump about top jobs at the FDA. Sud­den­ly, that Twit­ter chan­nel van­ished in­to thin air, as we re­port­ed first. But his Tweets were saved, and live on, even as we hear that Srini­vasan may not be in line for that job any more. Trump’s fi­nal pick to run the FDA will have a huge in­flu­ence on how the agency plans to speed drug de­vel­op­ment. And we have a right and a need to suss things out for our­selves. So far, this episode ranks as an­oth­er rea­son to sus­pect that the FDA may soon be head­ed for a wrong turn, es­pe­cial­ly if the new fo­cus is on a sub­stan­tial dereg­u­la­tion of drug de­vel­op­ment born out of a dis­gust for the bu­reau­cra­cy.


Medicare price ne­go­ti­a­tions can ben­e­fit bio­phar­ma too.

Speak­ing of Trump, our new pres­i­dent got the chance to clar­i­fy his think­ing re­gard­ing drug prices in a week­end in­ter­view with The Wash­ing­ton Post. As we sus­pect­ed, his main weapon in the war against list prices will be Medicare ne­go­ti­a­tions — now banned by in­dus­try-friend­ly law­mak­ers. The in­dus­try des­per­ate­ly doesn’t want to see this, but in re­al­i­ty it makes for an ef­fec­tive way to get ac­tu­al dis­count prices on dis­play for all to see. The Amer­i­can pub­lic has a right to see what’s ac­tu­al­ly be­ing paid for ther­a­peu­tics, and this is one way to set a base­line dis­count price that is read­i­ly trans­par­ent. The free ride on ever-ris­ing drug prices is over, and we all know it. The soon­er this hap­pens, the bet­ter for every­one. If a price can’t sur­vive the light of day, it’s too high. In the mean­time, the in­dus­try can move more to­ward a per­for­mance-based pric­ing method.


Mallinck­rodt joins the price goug­ing club, and gets away with “mur­der.”

One of the rea­sons why drug prices are so con­tro­ver­sial is that the fed­er­al gov­ern­ment is vir­tu­al­ly help­less when it comes to pre­vent­ing a com­pa­ny from jack­ing up prices as­tro­nom­i­cal­ly overnight. Quest­cor proved that more than three years ago, when it brazen­ly bought a ri­val to Ac­thar to pre­vent com­pe­ti­tion. And Mallinck­rodt bought in to that rigged game by buy­ing Quest­cor. This week, Mallinck­rodt was cit­ed for il­le­gal­ly block­ing com­pe­ti­tion, but got away with on­ly a $100 mil­lion fine. The com­pa­ny paid for that with the in­creased rev­enue it earned on Ac­thar in just 2015, as its price con­tin­ued to soar. This is what Trump means when he says phar­ma is get­ting away with mur­der. It may take years for a ri­val to get ap­proved and on the mar­ket. And Mallinck­rodt is still free to jack prices. This has to end with some kind of ef­fec­tive le­gal mech­a­nism to pre­vent abus­es. Your next Mar­tin Shkre­li or Mallinck­rodt is sit­ting just around the cor­ner. And the longer we wait, the more like­ly it is that re­form will overzeal­ous­ly dam­age the way in­no­va­tion is fi­nanced in bio­phar­ma.


Our lat­est lists on ven­ture cash un­der­score sol­id fun­da­men­tals for biotech. Let’s not screw it up now.

Biotech is de­pen­dent on the flow of bil­lions of dol­lars in ven­ture cash. And we love noth­ing quite as much as track­ing where the mon­ey is com­ing from as well as what re­gions it is go­ing to. Al­ways enor­mous­ly pop­u­lar with read­ers, this year’s sto­ries paint a sim­ple tale of the dom­i­nant role played by two cru­cial hubs — Boston/Cam­bridge and the Bay Area — as well as a group of savvy in­vestors who are in­stru­men­tal in fos­ter­ing and fund­ing new in­no­va­tion in drug de­vel­op­ment. That ma­chine has been hum­ming along nice­ly now for sev­er­al years, point­ing to the fact that the fun­da­men­tals in pri­vate biotech are strong and durable. That bodes well for the years ahead, as ma­jor re­forms are be­ing planned, which we should all ap­pre­ci­ate. Re­form­ers might like to keep that in mind.

John Hood [file photo]

UP­DATE: Cel­gene and the sci­en­tist who cham­pi­oned fe­dra­tinib's rise from Sanofi's R&D grave­yard win FDA OK

Six years after Sanofi gave it up for dead, the FDA has approved the myelofibrosis drug fedratinib, now owned by Celgene.

The drug will be sold as Inrebic, and will soon land in the portfolio at Bristol-Myers Squibb, which is finalizing a deal to acquire Celgene.

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UP­DAT­ED: AveX­is sci­en­tif­ic founder was axed — and No­var­tis names a new CSO in wake of an ethics scan­dal

Now at the center of a storm of controversy over its decision to keep its knowledge of manipulated data hidden from regulators during an FDA review, Novartis CEO Vas Narasimhan has found a longtime veteran in the ranks to head the scientific work underway at AveXis, where the incident occurred. And the scientific founder has hit the exit.

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Ab­b­Vie gets its FDA OK for JAK in­hibitor upadac­i­tinib, but don’t look for this one to hit ex­ecs’ lofty ex­pec­ta­tions

Another big drug approval came through on Friday afternoon as the FDA OK’d AbbVie’s upadacitinib — an oral JAK1 inhibitor that is hitting the rheumatoid arthritis market with a black box warning of serious malignancies, infections and thrombosis reflecting fears associated with the class.

It will be sold as Rinvoq — at a wholesale price of $59,000 a year — and will likely soon face competition from a drug that AbbVie once controlled, and spurned. Reuters reports that a 4-week supply of Humira, by comparison, is $5,174, adding up to about $67,000 a year.

The top 10 fran­chise drugs in bio­phar­ma his­to­ry will earn a to­tal of $1.4T (tril­lion) by 2024 — what does that tell us?

Just in case you were looking for more evidence of just how important Amgen’s patent win on Enbrel is for the company and its investors, EvaluatePharma has come up with a forward-looking consensus estimate on what the list of top 10 drugs will look like in 2024.

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ICER blasts FDA, PTC and Sarep­ta for high prices on DMD drugs Em­flaza, Ex­ondys 51

ICER has some strong words for PTC, Sarepta and the FDA as the US drug price watchdog concludes that as currently priced, their respective new treatments for Duchenne muscular dystrophy are decidedly not cost-effective.

The final report — which cements the conclusions of a draft issued in May — incorporates the opinion of a panel of 17 experts ICER convened in a public meeting last month. It also based its analysis of Emflaza (deflazacort) and Exondys 51 (eteplirsen) on updated annual costs of $81,400 and over $1 million, respectively, after citing “incorrect” lower numbers in the initial calculations.

The key dates for KRAS watch­ers through the end of the year — the trail is nar­row and risks are ex­treme

There’s nothing quite like a big patent win when it comes to burnishing your prospects in the pipeline. And for Amgen, which seems to have rescued Enbrel for a run to 2029, the cheering section on Wall Street is now fixed on AMG 510 and a key rival.

And it didn’t take much data to do it. 

There was the first snapshot of a handful of patients, with a 50% response rate. Then came word that Amgen researchers are also tracking responses in different cancers, at least one in colorectal cancer and appendiceal too. 

Bain's Or­ly Mis­han joins Pfiz­er's neu­ro spin­out Cerev­el; On­colyt­ic virus biotech taps Sil­la­Jen ex­ec He­le­na Chaye as CEO

→ Bain Capital is deploying one of its top investors to Cerevel Therapeutics, steering a $350 million-plus neuro play carved out of Pfizer. Orly Mishan — a co-founder and principal of Bain’s life sciences unit — was involved in the partnership that birthed the biotech spinout in the first place. As Cerevel’s first chief business officer, she is tasked with corporate development, program management as well as technical operations. 

UP­DAT­ED: Sci­en­tist-CEO ac­cused of im­prop­er­ly us­ing con­fi­den­tial in­fo from uni­corn Alec­tor

The executive team at Alector $ALEC has a bone to pick with scientific co-founder Asa Abeliovich. Their latest quarterly rundown has this brief note buried inside:

On June 18, 2019, we initiated a confidential arbitration proceeding against Dr. Asa Abeliovich, our former consulting co-founder, related to alleged breaches of his consulting agreement and the improper use of our confidential information that he learned during the course of rendering services to us as our consulting Chief Scientific Officer/Chief Innovation Officer. We are in the early stage of this arbitration proceeding and are unable to assess or provide any assurances regarding its possible outcome.

There’s no explicit word in the filing on what kind of confidential info was involved, but the proceeding got started 2 days ahead of Abeliovich’s IPO.

Abeliovich, formerly a tenured associate professor at Columbia, is a top scientist in the field of neurodegeneration, which is where Alector is targeted. More recently, he’s also helped start up Prevail Therapeutics as the CEO, which raised $125 million in an IPO. And there he’s planning on working on new gene therapies that target genetically defined subpopulations of Parkinson’s disease. Followup programs target Gaucher disease, frontotemporal dementia and synucleinopathies.

But this time Abeliovich is the CEO rather than a founding scientist. And some of their pipeline overlaps with Alector’s.

Abeliovich and Prevail, though, aren’t taking this one lying down.

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Chi­na has be­come a CEO-lev­el pri­or­i­ty for multi­na­tion­al phar­ma­ceu­ti­cal com­pa­nies: the trend and the im­pli­ca­tions

After a “hot” period of rapid growth between 2009 and 2012, and a relatively “cooler” period of slower growth from 2013 to 2015, China has once again become a top-of-mind priority for the CEOs of most large, multinational pharmaceutical companies.

At the International Pharma Forum, hosted in March in Beijing by the R&D Based Pharmaceutical Association Committee (RDPAC) and the Pharmaceutical Research and Manufacturers of America (PhRMA), no fewer than seven CEOs of major multinational pharmaceutical firms participated, including GSK, Eli Lilly, LEO Pharma, Merck KGaA, Pfizer, Sanofi and UCB. A few days earlier, the CEOs of several other large multinationals attended the China Development Forum, an annual business forum hosted by the research arm of China’s State Council. It’s hard to imagine any other country, except the US, having such drawing power at CEO level.