As a promi­nent an­a­lyst pre­dicts (even­tu­al) drug price con­trols, bio­phar­ma braces for a blow while Trump balls his fist

Some­time this week, no one is sure when, Pres­i­dent Don­ald Trump is ex­pect­ed to give his long-await­ed speech on drug pric­ing. And a long line­up of key play­ers in the field has been scratch­ing their heads, anx­ious­ly try­ing to de­ter­mine just how far the na­tion’s most un­pre­dictable pres­i­dent could go.

Trump, of course, has been free with his pop­ulist brand of an­ti-in­dus­try rhetoric for more than a year now, af­ter es­tab­lish­ing the base­line with re­peat­ed as­ser­tions that phar­ma com­pa­nies have been get­ting away with mur­der for years. 

But there’s been lit­tle di­rect ac­tion, be­yond the work at the FDA to hus­tle up new gener­ic ap­provals while try­ing to use the pub­lic spot­light to whip up some fresh com­pe­ti­tion for the price gougers. 

FDA com­mis­sion Scott Got­tlieb like­ly tipped the ad­min­is­tra­tion’s hand on one point in a pre­view he of­fered on pric­ing last week, in­di­cat­ing that in­dus­try prac­tices in fight­ing a de­lay­ing ac­tion against gener­ics and biosim­i­lars will face some di­rect chal­lenges. But while sig­nif­i­cant, that by it­self won’t bend any longterm trends on pric­ing in fa­vor of pay­ers.

Mean­while, pub­lic anger over drug pric­ing is re­peat­ed­ly stoked with an un­end­ing se­ries of high pro­file as­saults on cer­tain play­ers that have used their pric­ing pow­er to jack up rev­enue from old drugs.

Just last night, Mallinck­rodt was back in the spot­light with Ac­thar, which has gone from $40 a vial in 2001 to $40,000, threat­en­ing the ba­sic po­lice and fire ser­vices pro­vid­ed by the town of Rock­ford, IL.

For­mer Rock­ford May­or Lar­ry Mor­ris­sey set the stage for a 60 Min­utes seg­ment with this quote: “‘Why is health­care so ex­pen­sive? Be­cause the fix is in. That’s the an­swer. That’s the short an­swer.”

And it’s not the kind of an­swer bio­phar­ma wants to hear right now, es­pe­cial­ly on 60 Min­utes.

The in­dus­try is still dis­grun­tled over the ad­min­is­tra­tion’s de­ci­sion to get phar­ma to cov­er part of the donut hole in Medicare drug prices. But they can live with that. There’s been a steady fo­cus at PhRMA on try­ing to shift the fo­cus from the man­u­fac­tur­ers to the in­sur­ers and PBMs that man­age the ben­e­fit. But it hasn’t worked. And re­cent­ly that tac­tic has pro­duced some painful­ly awk­ward sal­lies on so­cial me­dia that tends to at­tract as much spon­ta­neous laugh­ter as dis­cus­sion.

With pub­lic anger per­co­lat­ing at an un­prece­dent­ed rate — a ma­jor­i­ty told Kaiser Health that re­duc­ing drug pric­ing should be a top pri­or­i­ty of pol­i­cy mak­ers — some an­a­lysts be­lieve that what­ev­er Trump comes up with it won’t be enough to stop the one strat­e­gy that would make the bio­phar­ma in­dus­try reel: Price con­trols.

Se­nior Wells Far­go an­a­lyst David Maris put it in blunt terms a few days ago.

We be­lieve the US is on a longer-term arc to­ward price con­trols and low­er mar­gins, as there is an up­ris­ing that has been per­co­lat­ing for years against high drug prices. While the caus­es are many and the prob­lem much more com­pli­cat­ed and nu­anced than many oth­ers would lead read­ers to be­lieve, there is a re­bel­lion at hand. Ar­gu­ments about the cost to de­vel­op a drug or how oth­er sup­ply chain con­stituents are to blame are not ap­pre­ci­at­ed by the par­ent stand­ing at a phar­ma­cy counter aban­don­ing their child’s pre­scrip­tion be­cause they can­not af­ford it due to an out­ra­geous de­ducible or high co-pay. And ex­am­ples of un­con­scionable drug price in­creas­es have fur­ther hard­ened many con­sumer opin­ions. 

That day prob­a­bly won’t ar­rive this week. But if that’s the path we’re on, Trump is faced with ei­ther sat­is­fy­ing the anger with some grand com­pro­mis­es, or stok­ing it by pulling his punch­es.


Im­age: Pres­i­dent Don­ald Trump at a speech in Oc­to­ber, 2017. AP IM­AGES

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

No, sci­en­tists are not any clos­er to pig-to-hu­man trans­plants than they were last week

Steve Holtzman was awoken by a 1 a.m. call from a doctor at Duke University asking if he could put some pigs on a plane and fly them from Ohio to North Carolina that day. A motorcyclist had gotten into a horrific crash, the doctor explained. He believed the pigs’ livers, sutured onto the patient’s skin like an external filter, might be able to tide the young man over until a donor liver became available.

UP­DAT­ED: Agenus calls out FDA for play­ing fa­vorites with Mer­ck, pulls cer­vi­cal can­cer BLA at agen­cy's re­quest

While criticizing the FDA for what may be some favoritism towards Merck, Agenus on Friday officially pulled its accelerated BLA for its anti-PD-1 inhibitor balstilimab as a potential second-line treatment for cervical cancer because of the recent full approval for Merck’s Keytruda in the same indication.

The company said the BLA, which was due for an FDA decision by Dec. 16, was withdrawn “when the window for accelerated approval of balstilimab closed,” thanks to the conversion of Keytruda’s accelerated approval to a full approval four months prior to its PDUFA date.

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How to col­lect and sub­mit RWD to win ap­proval for a new drug in­di­ca­tion: FDA spells it out in a long-await­ed guid­ance

Real-world data are messy. There can be differences in the standards used to collect different types of data, differences in terminologies and curation strategies, and even in the way data are exchanged.

While acknowledging this somewhat controlled chaos, the FDA is now explaining how biopharma companies can submit study data derived from real-world data (RWD) sources in applicable regulatory submissions, including new drug indications.

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Marty Duvall, Oncopeptides CEO

On­copep­tides stock craters as it pulls can­cer drug Pepax­to from the mar­ket

Shares of Oncopeptides crashed more than 70% in early Friday trading after the company said it’s pulling its multiple myeloma drug Pepaxto (melphalan flufenamide) from the US market after failing a confirmatory trial. The move will force the company to close its US and EU business units and enact significant layoffs.

The FDA had scheduled an adcomm meeting next Thursday to discuss Pepaxto, which first won accelerated approval in February and costs about $19,000 per course of treatment. The committee was to weigh in on whether the confirmatory trial demonstrated a worse overall survival in the treatment arm compared to the control arm.

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Pfiz­er pitch­es its Covid-19 vac­cine for younger chil­dren ahead of ad­comm next week

Pfizer will present its case to the FDA’s vaccine adcomm next week, seeking authorization for a lower-dose version of its Covid-19 vaccine for kids ages 5 through 12, which the Biden administration said will likely begin rolling out early next month.

Two primary doses of the 10 µg vaccine (the dose for those ages 12 and up is 30 μg) given 3 weeks apart in this group of children “have shown a favorable safety and tolerability profile, robust immune responses against all variants of concern including Delta, and vaccine efficacy of 90.7% against laboratory-confirmed symptomatic COVID-19,” the company said in briefing documents ahead of next Tuesday’s meeting of the FDA’s Vaccines and Related Biological Products Advisory Committee.

David Lockhart, ReCode Therapeutics CEO

Pfiz­er throws its weight be­hind LNP play­er eye­ing mR­NA treat­ments for CF, PCD

David Lockhart did not see the meteoric rise of messenger RNA and lipid nanoparticles coming.

Thanks to the worldwide fight against Covid-19, mRNA — the genetic code that can be engineered to turn the body into a mini protein factory — and LNPs, those tiny bubbles of fat carrying those instructions, have found their way into hundreds of millions of people. Within the biotech world, pioneers like Alnylam and Intellia have demonstrated just how versatile LNPs can be as a delivery vehicle for anything from siRNA to CRISPR/Cas9.

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David Livingston (Credit: Michael Sazel for CeMM)

Renowned Dana-Far­ber sci­en­tist, men­tor and bio­phar­ma ad­vi­sor David Liv­ingston has died

David Livingston, the Dana-Farber/Harvard Med scientist who helped shine a light on some of the key molecular drivers of breast and ovarian cancer, died unexpectedly last Sunday.

One of the senior leaders at Dana-Farber during his nearly half century of work there, Livingston was credited with shedding light on the genes that regulate cell growth, with insights into inherited BRCA1 and BRCA2 mutations that helped lay the scientific foundation for targeted therapies and earlier detection that have transformed the field.

No­vo CEO Lars Fruer­gaard Jør­gensen on R&D risk, the deal strat­e­gy and tar­gets for gen­der di­ver­si­ty

 

I kicked off our European R&D summit last week with a conversation involving Novo Nordisk CEO Lars Fruergaard Jørgensen. Novo is aiming to launch a new era of obesity management with a new approval for semaglutide. And Jørgensen had a lot to say about what comes next in R&D, how they manage risk and gender diversity targets at the trendsetting European pharma giant.

John Carroll: I’m here with Lars Jørgensen, the CEO of Novo Nordisk. Lars, it’s been a really interesting year so far with Novo Nordisk, right? You’ve projected a new era of growing sales. You’ve been able to expand on the GLP-1 franchise that was already well established in diabetes now going into obesity. And I think a tremendous number of people are really interested in how that’s working out. You have forecast a growing amount of sales. We don’t know specifically how that might play out. I know a lot of the analysts have different ideas, how those numbers might play out, but that we are in fact embarking on a new era for Novo Nordisk in terms of what the company’s capable of doing and what it’s able to do and what it wants to do. And I wanted to start off by asking you about obesity in particular. Semaglutide has been approved in the United States for obesity. It’s an area of R&D that’s been very troubled for decades. There have been weight loss drugs that have come along. They’ve attracted a lot of attention, but they haven’t actually ever gained traction in the market. My first question is what’s different this time about obesity? What is different about this drug and why do you expect it to work now whereas previous drugs haven’t?

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