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. 

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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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.

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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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: Watch out Bay­er, Roche is com­ing for you with a dis­count price ri­val to the tu­mor ag­nos­tic drug you got from Loxo

Just ahead of schedule the FDA has come through with a key approval for Genentech’s tumor agnostic entrectinib — now headed to the market as Rozlytrek.

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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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