Snubbed at the FDA, PTC files their three-time los­er for Duchenne MD over protest

PTC Ther­a­peu­tics $PTCT isn’t go­ing to let a slate of three con­sec­u­tive, failed stud­ies for ataluren block its re­view at the FDA. Us­ing the agency’s rules for ap­pli­ca­tions on drugs reg­u­la­tors have re­fused to file, the biotech says it filed over protest, us­ing reg­u­la­to­ry pro­ce­dures to force a PDU­FA date of Oc­to­ber 24.

PTC CEO Stu­art Peltz was frank about why the FDA wouldn’t ac­cept their ap­pli­ca­tion ear­ly last year. Reg­u­la­tors felt that the com­pa­ny had flat failed to make a case for the drug that was wor­thy of a re­view. And he’s been beat­ing on the FDA’s door ever since, de­spite the agency’s de­ci­sion to re­ject its first ap­peal, claim­ing once again that the “to­tal­i­ty” of the da­ta war­rant its ap­proval.

In a state­ment, the CEO said he would be “work­ing close­ly with the FDA and the DMD com­mu­ni­ty to bring this much-need­ed ther­a­py to pa­tients.”

Cur­rent­ly, the EU al­lows the drug to be sold af­ter grant­i­ng a con­di­tion­al ap­proval that now re­quires PTC to con­duct a new clin­i­cal tri­al over the next 5 years. PTC’s move to­day un­der­scores a dif­fer­ent set of stan­dards that reg­u­la­tors have ap­plied to drugs in the field.

While the FDA spurned PTC and re­ject­ed Bio­Marin’s dris­apersen, the FDA’s Janet Wood­cock over­rode the stren­u­ous ob­jec­tions of nu­mer­ous sub­or­di­nates who felt that Sarep­ta had nev­er made its case for eteplirsen. Pa­tients and fam­i­lies or­ga­nized a co­or­di­nat­ed cam­paign to win an ap­proval, though, and the drug is now sold as Ex­ondys 51 with a la­bel that says ef­fi­ca­cy of the drug has nev­er been es­tab­lished.

Jef­feries’ Gena Wang doesn’t give PTC very good odds.

We not­ed that the ‘fil­ing over protest’ has been rarely pur­sued and would be con­sid­ered as a new ap­pli­ca­tion pro­ce­du­ral­ly. We found two prece­dents – Phar­ma­cyclics’ Xcytrin in lung can­cer brain metas­tases (fil­ing over protest in Apr ’07, PDU­FA date of Dec 31 ’07 and non-ap­prov­able let­ter on Dec 21 ’07), and GSK’s Bexxar in Non-Hodgkin’s Lym­phoma (ini­tial fil­ing in 1999, and ac­cel­er­at­ed ap­proval af­ter fil­ing over protest in June ’03; with­drawn by GSK in 2013 due to in­com­plete post-mar­ket­ing tri­als).

“The bull ar­gu­ment for PTCT has been that eteplirsen’s ap­proval “opens the door” for ataluren,” not­ed RBC’s Simos Sime­oni­dis. “We def­i­nite­ly agree that both ap­pli­ca­tions are “less-than-per­fect” and, in our view, nei­ther one should have been ap­proved. But even though we dis­agreed with FDA on eteplirsen, we can un­der­stand their ra­tio­nale for ac­cel­er­at­ed ap­proval. The key and very sig­nif­i­cant dif­fer­ence be­tween the two ap­pli­ca­tions is that we would char­ac­ter­ize the eteplirsen dataset as in­com­plete and the ques­tion of whether eteplirsen “works” in DMD as still unan­swered. This can­not be said about ataluren, where mul­ti­ple large, well-con­trolled, ran­dom­ized tri­als have been con­duct­ed. ” They all failed.

PTC ac­knowl­edged just days ago that its drug failed a late-stage pro­gram for cys­tic fi­bro­sis, adding to the ev­i­dence that the drug doesn’t work. Ear­li­er it failed a Phase IIb for Duchenne MD fol­lowed by an­oth­er flop in Phase III, de­signed to over­come the prob­lems the biotech saw ear­li­er.

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

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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

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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?

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UP­DAT­ED: Sci­en­tist-CEO ac­cused of im­prop­er­ly us­ing con­fi­den­tial in­fo from uni­corn Alec­tor

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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

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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.