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.

Australia’s Avance Clinical: no IND required and a 43.5% rebate on clinical spend for CGT biotechs

No IND Re­quired for Cell and Gene Ther­a­py Stud­ies with Aus­tralia’s Ac­cred­it­ed CRO Avance Clin­i­cal

Avance Clinical is the specialist Australian CRO, with CGT accreditation, for international biotechs that leverages Australia’s supportive clinical trials environment which includes no IND requirement plus a 43.5% Government incentive rebate on clinical spend.

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The cell and gene therapies (CGT) sector offers unprecedented opportunities for patient disease management across virtually all therapeutic areas. However, finding the right accredited clinical teams to take a therapy through to the clinic and manage the regulatory process can be a major challenge for biotechs with a CGT product.

Ursula von der Leyen, President of the European Commission

Omi­cron: Re­searchers scram­ble as new coro­n­avirus mu­ta­tion takes flight around the globe — Pfiz­er/BioN­Tech, Mod­er­na vow swift re­sponse

As Americans were waking up for their Black Friday rituals, they were greeted with the news that a new mutation of the Covid-19 virus has appeared and been sequenced — after it caught an international flight to Hong Kong. And two of the leading Covid-19 vaccine developers promised delivery of a new vaccine “within 100 days” if necessary while a third spelled out its 3-prong strategy hours later.

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Thanks­giv­ing edi­tion: Top 15 End­points sto­ries of 2021; Can you name that vac­cine?; Mer­ck­'s Covid an­tivi­ral dis­ap­points; FDA nom­i­nee's in­dus­try ties; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Happy Thanksgiving to all those who are celebrating it — although, if we are being honest, this week’s abbreviated edition is really for those who are not. Wherever you’re tuning in from, we appreciate your support, hope you find this recap helpful and we wish you a wonderful weekend.

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Mar­ket­ingRx roundup: Am­gen, Lil­ly, Bio­haven mi­graine brand re­call low, study says; No­var­tis looks to re­make drug launch mod­el

Forget the migraine marketing brand wars. When it comes to patients, many can’t even name one despite substantial advertising efforts, according to a new study from Phreesia that concludes CGRP migraine drugmakers still need to work on brand recognition.

Almost half (47%) of the patients Phreesia surveyed couldn’t name one preventative migraine brand. The best performer was Topamax, a small molecule anticonvulsant that’s been around since 2004, which 26% of migraine patients could recall. Among the new CGRP brand names recognized, Amgen’s Aimovig ranked highest with 8% recall, while Eli Lilly’s Emgality and Biohaven’s Nurtec tied at 7% and Teva’s Ajovy was remembered by 3% of patients.

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What were End­points read­ers tun­ing in­to this year? Here’s a look at our 15 most pop­u­lar re­ports of the year (so far)

At the beginning of this year, I laid out a basic objective for Endpoints News as we headed to our 5th anniversary. We’ve long been doing a fine job covering the breaking news in R&D — if I do say so myself — but we needed to expand our horizons on industry coverage, increase the staff and go much, much deeper when the stories demanded it.

In a phrase: broader and deeper.

It’s safe to say, based on our daily web traffic, that you all seemed to like this idea. We’ve doubled the staff — thanks to a growing group of paid subscribers — ramped up the daily report and now publish a regular slate of in-depth articles. And traffic — those clicks you always read about — have gone up in volume too. Monthly sessions are up 43%, to close to 1.5 million. Unique readers are up 63%, to 874,480 in October, after setting a record of close to a million the month before. Page views are running at 3 million-plus a month. And the overall number of subscribers has surged to 124,000.

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Name that vac­cine: From Comir­naty to Spike­vax to Nu­vax­ovid, Covid-19 shot­s' brand names re­main lit­tle-known

Most people know if they’re “Team Pfizer” or “Team Moderna,” but few know if they got the Comirnaty or Spikevax Covid-19 vaccine. Those are the brand names of Pfizer and Moderna vaccines, respectively, however they have yet to take hold with consumers, media or even medical professionals.

And there are others. Covid vaccine brand names also include AstraZeneca’s Vaxzevria, Novavax’s Nuvaxovid, and Sanofi and GlaxoSmithKline’s Vidprevtyn. J&J’s Janssen-developed Covid vaccine is the lone major holdout and is still yet to be named, if ever. In EMA filings approving its conditional use, the brand name is listed simply as “Covid-19 Vaccine Janssen.”

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Troy Wilson, Kura CEO

UP­DAT­ED: FDA hits the red light on an ear­ly-stage AML study af­ter a pa­tient dies

The FDA has slapped a clinical hold on the early-stage program for one of Kura Oncology’s cancer drugs following a patient’s death in a clinical trial.

The biotech $KURA reported early Wednesday that the Phase Ib study of KO-539 for acute myeloid leukemia would be halted, suspending enrollment, while researchers and the FDA probed the death. Patients already on the drug can continue taking it.

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Emma Walmsley, GlaxoSmithKline CEO (Fang Zhe/Xinhua/Alamy Live News)

Glax­o­SmithK­line places a risky bet on Ar­row­head­'s RNA drug in the fail­ure-strewn NASH field

As activist investors champ at the bit for change at drug giant GlaxoSmithKline, the pharma giant has turned over many rocks to find an R&D success to present to its detractors. In NASH, a field strewn with failures, GSK hopes a new license deal can churn out a much-needed winner.

GSK will pay $120 million in upfront cash and $910 million in downstream milestones to develop and sell ARO-HSD, Arrowhead Pharmaceuticals’ RNA interference drug targeting fatty liver disease nonalcoholic steatohepatitis (NASH), the companies said Monday.

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Joan Perelló, Sanifit CEO

Joan Perel­ló set out 17 years ago to de­vel­op a drug. And to­day he's be­ing re­ward­ed with a $424M biotech buy­out

Joan Perelló beat all the odds with his little Spanish biotech startup Sanifit.

Working on the far perimeter of the big US/European drug development scene, he took a drug born out of his PhD work and got enough seed cash to get started. That’s one near miracle. In the second near miracle he gathered a previously unheard of venture raise in Spain — helping build an industry ecosystem from scratch — to pursue a successful search for solid human data for his drug, SNF472. And while gathering a virtual team of developers from Europe and the US, the CEO/co-founder steered it into the late-stage arena.

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