Yes, the FDA did just slap down PTC’s Duchenne MD drug for the third time

What­ev­er last shred of hope re­mained that PTC could turn things around and win a mir­a­cle ap­proval at the FDA for their Duchenne mus­cu­lar dy­s­tro­phy drug ataluren end­ed with a terse state­ment Wednes­day morn­ing not­ing that the agency has re­ject­ed their mar­ket­ing ap­pli­ca­tion.

Ac­cord­ing to PTC, the FDA has de­mand­ed more da­ta from a new clin­i­cal study, along with some CMC in­put along with ad­di­tion­al in­fo that the biotech says it is in the process of pro­vid­ing.

The FDA de­ci­sion marks the lat­est mis­step in a long and rocky road for PTC, which has been bang­ing on the FDA’s doors now for years with­out get­ting much more than re­peat­ed signs to move along. But PTC CEO Stu Peltz still won’t take re­peat­ed no’s for an an­swer.

“We are ex­treme­ly dis­ap­point­ed for the Duchenne com­mu­ni­ty and strong­ly dis­agree with the agency’s con­clu­sions,” said Peltz in a state­ment. “We be­lieve that this de­ci­sion fails to con­sid­er the ben­e­fit-risk of ataluren and the high un­met med­ical need. There­fore, we plan to file a for­mal dis­pute res­o­lu­tion re­quest next week.”

In­stead of see­ing its share price dive on bad news, though, PTC’s stock climbed 4%.

Stu Peltz

Twice the FDA re­fused to even file their ap­pli­ca­tion, slam­ming the biotech for fail­ing to put to­geth­er a cred­i­ble ap­pli­ca­tion af­ter a se­ries of tri­al fail­ures. As a re­sult, bet­ting that the agency might bend in the face of fam­i­ly sup­port for their drug — the way Sarep­ta ben­e­fit­ed de­spite fail­ing to demon­strate ef­fi­ca­cy — the com­pa­ny forced an agency re­view.

The sub­se­quent agency analy­sis of­fered an­oth­er slap-down for PTC, which has been forced to de­tail a se­ries of tri­al fail­ures for their drug. The FDA re­view not­ed:

Ul­ti­mate­ly, no pos­i­tive re­sults from any prospec­tive­ly planned analy­ses that are per­sua­sive have been pro­vid­ed with this ap­pli­ca­tion. The ap­pli­cant has pre­sent­ed on­ly the re­sults from nu­mer­ous post hoc and ex­plorato­ry analy­ses that are in­tend­ed to mit­i­gate two neg­a­tive clin­i­cal tri­als.

In a rare sign of their dis­plea­sure, the agency al­so of­fered their opin­ion that this is one drug that may nev­er be re­deemed by pos­i­tive re­sults from a clin­i­cal tri­al.

Aaron Kessel­heim of Har­vard Med­ical School, who sat on the FDA ex­pert pan­el that looked this drug over, con­clud­ed that PTC’s da­ta were the kind of re­sults that could eas­i­ly be mis­lead­ing, join­ing in a 10 to 1 vote against the mar­ket­ing ap­pli­ca­tion.

It could have gone worse for PTC. The FDA al­lowed the pan­el to con­sid­er a sum­ma­ry ex­e­cu­tion of PTC’s chances, but the pan­el pre­ferred to con­clude that the da­ta were sim­ply in­con­clu­sive thus far — al­low­ing PTC the chance to come back with new da­ta at some point.

Amaz­ing­ly, de­spite the re­peat­ed set­backs on ataluren, the Eu­ro­peans have al­lowed their ac­cel­er­at­ed ap­proval of the drug to stand as PTC is giv­en chance af­ter chance to come up with some­thing fa­vor­able.

PTC, though, is busy mar­ket­ing a Duchenne drug. Af­ter Marathon Phar­ma was gripped in a con­tro­ver­sy fol­low­ing an ap­proval for an old, cheap Eu­ro­pean steroid for the lethal dis­ease, Marathon opt­ed to shut down, hand­ing over their ther­a­py to PTC, which has con­tin­ued to charge even more than Marathon’s planned $84,000 an­nu­al whole­sale price for the larg­er boys in the Duchenne pop­u­la­tion.

The out­rage that greet­ed Marathon, though, has large­ly died down af­ter PTC man­aged to lim­it its pub­lic price to the ball­park dis­count that pay­ers would face.

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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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.

As dis­as­ter struck, Ab­b­Vie’s Rick Gon­za­lez swooped in on Al­ler­gan with an of­fer Brent Saun­ders couldn’t say no to

Early March was a no good, awful, terrible time for Allergan CEO Brent Saunders. His big lead drug had imploded in a Phase III disaster and activists were after his hide — or at least his chairman’s title — as the stock price continued a steady droop that had eviscerated share value for investors.

But it was a perfect time for AbbVie CEO Rick Gonzalez to pick up the phone and ask Saunders if he’d like to consider a “strategic” deal.

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As­traZeneca's jug­ger­naut PARP play­er Lyn­parza scoops up an­oth­er dom­i­nant win in PhI­II as the FDA adds a 'break­through' for Calquence

AstraZeneca’s oncology R&D group under José Baselga keeps churning out hits.

Wednesday morning the pharma giant and their partners at Merck parted the curtains on a successful readout for their Phase III PAOLA-1 study, demonstrating statistically significant improvement in progression-free survival for women with ovarian cancer in a first-line maintenance setting who added their PARP Lynparza to Avastin. This is their second late-stage success in ovarian cancer, which will help stave off rivals like GSK.

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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.