No, Pres­i­dent Trump, bio­phar­ma does not want you to dereg­u­late drug de­vel­op­ment

Nobody wants rad­i­cal changes at the FDA, ex­cept per­haps Pres­i­dent Trump and a few close as­so­ciates.

We’ve been track­ing the in­dus­try’s clear re­luc­tance to dis­card the FDA gold stan­dard in drug de­vel­op­ment. In our most re­cent sur­vey yes­ter­day, a plu­ral­i­ty of biotech ex­ecs vot­ed against sig­nif­i­cant changes at the agency, say­ing that re­forms over the past few years has al­ready sped up the process. And many of the ex­ecs that would like to see con­tin­ued change were clear­ly more in­ter­est­ed in specif­i­cal­ly tai­lored up­grades, not a whole­sale over­haul.

To­day, Reuters added a few more bio­phar­ma voic­es to that mix, most no­tably to me Roger Perl­mut­ter at Mer­ck.

Roger M. Perl­mut­ter, Mer­ck

“Peo­ple of­ten ar­gue that the FDA is too re­stric­tive,” Perl­mut­ter told the wire ser­vice. “We have the sense that the bal­ance is pret­ty right … you have to have a well-char­ac­ter­ized risk/ben­e­fit pro­file.”

Big Phar­ma’s big goal at the new ad­min­is­tra­tion has been tax re­form, and most of the CEOs have been no­to­ri­ous­ly qui­et in the face of Trump’s in­sis­tence that vast seg­ments of the reg­u­la­tions around drug de­vel­op­ment need to be in­cin­er­at­ed. So Perl­mut­ter’s po­si­tion here speaks loud­ly to the in­dus­try’s stand on dereg­u­la­tion.

Your run of the mill biotech fo­cused al­most ex­clu­sive­ly on pipeline de­vel­op­ment will have no prob­lem em­brac­ing Perl­mut­ter’s po­si­tion. And in­ter­est­ing­ly, even BIO is will­ing to sig­nal now — qui­et­ly — that it’s ad­vis­ing Trump against a rad­i­cal change in strat­e­gy at the FDA.

That’s why there’s been so much ac­tive sup­port for Scott Got­tlieb as the new com­mis­sion­er. Got­tlieb, a physi­cian, is seen as some­one who can help con­tin­ue stream­lin­ing the FDA with­out up­root­ing stan­dards that are es­sen­tial to do­ing drug R&D with in­tegri­ty.

Richard Paz­dur

A few years ago, I was im­pressed to hear Richard Paz­dur stand up at AS­CO and in no un­cer­tain terms em­brace the break­through ther­a­py des­ig­na­tion. Can any­one de­ny that his on­col­o­gy group has fol­lowed through, com­plete­ly over­haul­ing time­lines for new can­cer drugs?

Dif­fer­ent dis­eases call for dif­fer­ent stan­dards. Di­a­betes and heart dis­ease are not can­cer, and safe­ty con­cerns for can­cer drugs ini­tial­ly treat­ed in dy­ing pa­tients aren’t the same as safe­ty con­cerns for drugs de­signed to pre­vent an­oth­er heart at­tack or live with a chron­ic dis­ease. Still, the rest of the FDA can do more to fol­low Paz­dur’s lead.

Change is need­ed at the FDA. Just not the kind of bomb throw­ing that has been so loud­ly voiced by Trump. We hope he gets the mes­sage, soon.

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive PhI­II for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

Hal Barron has his second positive round of Phase III data in hand for his anti-BCMA antibody drug conjugate belantamab mafodotin (GSK2857916). And GSK’s research chief says the data paves the way for their drive in search of an FDA approval for treating multiple myeloma. 

It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,900+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,900+ biopharma pros reading Endpoints daily — and it's free.

We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at with any issues.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,900+ biopharma pros reading Endpoints daily — and it's free.

Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,900+ biopharma pros reading Endpoints daily — and it's free.

Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll

UP­DAT­ED: As­traZeneca’s Imfinzi/treme com­bo strikes out — again — in lung can­cer. Is it time for last rites?

AstraZeneca bet big on the future of their PD-L1 Imfinzi combined with the experimental CTLA-4 drug tremelimumab. But once again it’s gone down to defeat in a major Phase III study — while adding damage to the theory involving targeting cancer with a high tumor mutational burden.

Early Wednesday the pharma giant announced that their NEPTUNE study had failed, with the combination unable to beat standard chemo at overall survival in high TMB cases of advanced non-small cell lung cancer. We won’t get hard data until later in the year, but the drumbeat of failures will call into question what — if any — future this combination can have left.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,900+ biopharma pros reading Endpoints daily — and it's free.

Am­gen, Al­ler­gan biosim­i­lar of Roche's block­buster Rit­ux­an clears an­oth­er US piv­otal study 

Novartis $NVS may have given up, but Amgen $AMGN and Allergan $AGN are plowing ahead with their knockoff of Roche’s blockbuster biologic Rituxan in the United States.

Their copycat, ABP 798, was found to have a clinically equivalent impact as Rituxan — meeting the main goal of the study involving CD20-positive B-cell non-Hodgkin’s lymphoma patients. This is the second trial supporting the profile of the biosimilar. In January, it came through with positive PK results in patients with rheumatoid arthritis.