An­oth­er Shkre­li-like drug price con­tro­ver­sy? Lea­di­ant finds it­self in the spot­light af­ter 500-fold price hike — FT

Mar­tin Shkre­li may be cool­ing his heels in a fed­er­al prison, but the phar­ma pric­ing strat­e­gy he pur­sued that got him in­to an in­tense na­tion­al con­tro­ver­sy ap­pears to be alive and well in Eu­rope this week.

The Fi­nan­cial Times con­cen­trat­ed its at­ten­tion over the week­end on the case of a drug called chen­odeoxy­cholic acid, or CD­CA. Lea­di­ant Bio­sciences, ear­li­er known as Sig­ma-Tau, gained con­trol of the cheap, old drug, once used to treat gall­stones, af­ter it had be­come rou­tine­ly used off-la­bel to treat a rare con­di­tion called cere­bro­tendi­nous xan­thomato­sis.

The biotech pulled it off the mar­ket and then gained an EMA OK for the ther­a­py. And then they jacked the price from €300 a year to more than €150,000, ac­cord­ing to the FT.

In the US, that kind of price hike may be con­tro­ver­sial, but it’s per­fect­ly le­gal. (Shkre­li’s prison sen­tence was for de­fraud­ing hedge fund in­vestors.) Marathon Phar­ma­ceu­ti­cals con­firmed that when it bought a cheap old steroid and jacked the price af­ter get­ting it ap­proved specif­i­cal­ly for Duchenne mus­cu­lar dy­s­tro­phy, large­ly on the ba­sis of some old tri­al da­ta it ac­quired. Af­ter that scan­dal erupt­ed, though, Marathon backed off and then sold the drug to PTC, which has main­tained a high price with­out gain­ing the same no­to­ri­ety.

Wilbert Ban­nen­berg

In Eu­rope, though, we hear that the Dutch Phar­ma­ceu­ti­cal Ac­count­abil­i­ty Foun­da­tion is set to file claims of mar­ket pow­er abuse with reg­u­la­tors.

“Some com­pa­nies are mis­us­ing the sys­tem to get high­er prof­its, and pa­tients are be­ing af­fect­ed when cheap, af­ford­able old prod­ucts are no longer avail­able,” foun­da­tion chief Wilbert Ban­nen­berg told the Fi­nan­cial Times. “This may be le­gal but it’s not so­cial­ly ac­cept­able.”

Lea­di­ant, which is led by CEO Mar­co Brughera, says it’s open to di­a­logue on the sub­ject of price. 

Tillman Gerngross, Adagio Therapeutics CEO

An­ti­body leg­end Till­man Gern­gross is el­bow­ing his way in­to the Covid-19 R&D cru­sade: 'I don’t see this end­ing any­time soon'

One of the most influential — and outspoken — scientists at work in the field of antibody discovery is jumping into the frenzied race to create new therapeutics to treat and prevent Covid-19. And he’s operating with the conviction that the current outbreak now once again spreading like wildfire will create plenty of demand for what he has in mind.

Dartmouth professor and Adimab CEO Tillman Gerngross tells me he’s raised $50 million from a group of close VCs to spin out a new company — Adagio Therapeutics — with a full C-suite team assembled to hire up a staff and keep rolling toward the clinic.

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Tal Zaks, Moderna CMO (Moderna via YouTube)

UP­DAT­ED: NI­AID and Mod­er­na spell out a 'ro­bust' im­mune re­sponse in PhI coro­n­avirus vac­cine test — but big ques­tions re­main to be an­swered

The NIAID and Moderna have spelled out positive Phase I safety and efficacy data for their Covid-19 vaccine mRNA-1273 — highlighting the first full, clear sketch of evidence that back-to-back jabs at the dose selected for Phase III routinely produced a swarm of antibodies to the virus that exceeded levels seen in convalescent patients — typically in multiples indicating a protective response.

Moderna execs say plainly that this first stage of research produced exactly the kind of efficacy they hoped to see in humans, with a manageable safety profile.

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Trans­port Sim­u­la­tion Test­ing for Your Ther­a­py is the Best Way to As­sure FDA Ex­pe­dit­ed Pro­gram Ap­proval

Modality Solutions is an ISO:9001-registered biopharmaceutical cold chain engineering firm with unique transport simulation capabilities that support accelerated regulatory approval for biologics and advanced therapeutic medicinal products (ATMP). Our expertise combines traditional validation engineering approaches with regulatory knowledge into a methodology tailored for the life sciences industry. We provide insight and execution for the challenges faced in your cold chain logistics network.

GSK’s Shin­grix leader Guil­laume Pfe­fer has jumped on board Flag­ship to helm a biotech hy­brid as Afeyan’s lat­est CEO-part­ner

After spending 4 years in a senior post with GlaxoSmithKline’s star team positioning Shingrix for a blockbuster approval, Guillaume Pfefer is headed back to the biotech world — in style.

Pfefer has signed on to join Noubar Afeyan’s busy group of partners at Flagship, and he’s taking the helm of an upstart — which today is being merged with another Flagship startup — with some grand plans of its own. The announcement this morning notes that Pfefer will run Kintai Therapeutics, one of the grads of the Flagship labs.

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Who are the women blaz­ing trails in bio­phar­ma R&D and lead­ing the fight against Covid-19? Nom­i­nate them for End­points' spe­cial re­port

One of the many inequalities the pandemic has laid bare is the gender imbalance in biomedical research. A paper examining Covid-19 research authorship wondered out loud: Where are the women?

It’s a question that echoes beyond our current times. In the biopharma world, not only are women under-represented in R&D roles (particularly at higher levels), their achievements and talents could also be undermined by stereotypes and norms of leadership styles. The problem is even more dire for women of color.

Donald Trump and Anthony Fauci (AP Images)

Covid-19 roundup: Fau­ci fires back at White House cam­paign to un­der­mine him

Anthony Fauci has called the White House campaign to discredit him “a bit bizarre” and said he stands by his previous statements, even if he has since changed his views.

The NIAID chief — who has received an outpouring of support following reports that the Trump administration has sent a document akin to opposition research to multiple news outlets — spoke with his usual candor in interviews with The Atlantic.

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John Furey, Imvax CEO

A neu­ro­sur­geon spent the past 30 years de­vel­op­ing a neoanti­gen tu­mor vac­cine. Now he has $112M for a piv­otal test

As a neurosurgeon, David Andrews knew there wasn’t much he could do for his glioma patients after resecting — rarely fully — their tumor. Even with the best treatment and care available, median overall survival is just somewhere between 14 and 16 months.

Then in the 1990s, his mentor at Thomas Jefferson University introduced him to Renato Baserga, a pathologist who had been studying the effect of using antisense oligonucleotide to knock out the insulin-like growth factor type 1 receptor in cancers. As IGF-R1 drives tumor growth and metastasis, the preclinical reasoning went, implanting a molecule targeting the receptor together with the tumor material near lymph nodes can slow down the spread of the cancer.

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The $1B Mer­ck-Bay­er drug that di­vid­ed car­di­ol­o­gists in March gets pri­or­i­ty re­view

Three months after Merck published in the New England Journal of Medicine data that left doctors and investors divided 0ver just how well its experimental heart drug worked, the FDA has handed that drug priority review. A decision is now due by January 20, 2021.

Merck first announced the drug, known as vericiguat, as a Phase III success last November. In 2016, Merck had paid $1 billion upfront for US rights to the Bayer-developed drug. Early projections foresaw a few hundred million a year in sales, but the unspecified late-stage success raised the possibility for far more. After all, Novartis’s flagship heart drug, Entresto, was earning $1.7 billion per year and was expected to reach up to $4 billion in annual sales.

New biotech Exalys, seek­ing to pre­vent post­op­er­a­tive delir­i­um, launch­es with $15 mil­lion in Se­ries A

An old group of former colleagues will be reuniting to lead a new biotech venture aimed at cultivating a portfolio to treat neuroinflammatory disorders.

Led by Rick Orr, who ran biotechs Adynxx and Alliqua Biomedical, the group is launching the startup Exalys on Thursday with $15 million in Series A funding from venture firms Catalys Pacific and Domain Associates. The nascent company’s first project will focus on preventing postoperative delirium, licensing a platform of EP4 receptors from Japanese pharma Eisai.