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 Reed at JPM 2019. Jeff Rumans for Endpoints News

Sanofi's John Reed con­tin­ues to re­or­ga­nize R&D, cut­ting 466 jobs while boost­ing can­cer, gene ther­a­py re­search

The R&D reorganization inside Sanofi is continuing, more than a year after the pharma giant brought in John Reed to head the research arm of the Paris-based company.
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The top 10 block­buster drugs in the late-stage pipeline — Eval­u­ate adds 6 new ther­a­pies to heavy-hit­ter list

Vertex comes in for a substantial amount of criticism for its no-holds-barred tactical approach toward wresting the price it wants for its commercial drugs in Europe. But the flip side of that coin is a highly admired R&D and commercial operation that regularly wins kudos from analysts for their ability to engineer greater cash flow from the breakthrough drugs they create.

Both aspects needed for success in this business are on display in the program backing Vertex’s triple for cystic fibrosis. VX-659/VX-445 + Tezacaftor + Ivacaftor — it’s been whittled down to 445 now — was singled out by Evaluate Pharma as the late-stage therapy most likely to win the crown for drug sales in 5 years, with a projected peak revenue forecast of $4.3 billion.

The latest annual list, which you can see here in their latest world preview, includes a roster of some of the most closely watched development programs in biopharma. And Evaluate has added 6 must-watch experimental drugs to the top 10 as drugs fail or go on to a first approval. With apologies to the list maker, I revamped this to rank the top 10 by projected 2024 sales, instead of Evaluate's net present value rankings.

It's how we roll at Endpoints News.

Here is a quick summary of the rest of the top 10:

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UP­DAT­ED: Chica­go biotech ar­gues blue­bird, Third Rock 'killed' its ri­val, pi­o­neer­ing tha­lassemia gene ther­a­py in law­suit

Blue­bird bio $BLUE chief Nick Leschly court­ed con­tro­ver­sy last week when he re­vealed the com­pa­ny’s be­ta tha­lassemia treat­ment will car­ry a jaw-drop­ping $1.8 mil­lion price tag over a 5-year pe­ri­od in Eu­rope — mak­ing it the plan­et’s sec­ond most ex­pen­sive ther­a­py be­hind No­var­tis’ $NVS fresh­ly ap­proved spinal mus­cu­lar at­ro­phy ther­a­py, Zol­gens­ma, at $2.1 mil­lion. A Chica­go biotech, mean­while, has been fum­ing at the side­lines. In a law­suit filed ear­li­er this month, Er­rant Gene Ther­a­peu­tics al­leged that blue­bird and ven­ture cap­i­tal group Third Rock un­law­ful­ly prised a vi­ral vec­tor, de­vel­oped in part­ner­ship with the Memo­r­i­al Sloan Ket­ter­ing Can­cer Cen­ter (MSK), from its grasp, and thwart­ed the de­vel­op­ment of its sem­i­nal gene ther­a­py.

John Chiminski, Catalent CEO - File Photo

'It's a growth play': Catal­ent ac­quires Bris­tol-My­er­s' Eu­ro­pean launch pad, ex­pand­ing glob­al CD­MO ops

Catalent is staying on the growth track.

Just two months after committing $1.2 billion to pick up Paragon and take a deep dive into the sizzling hot gene therapy manufacturing sector, the CDMO is bouncing right back with a deal to buy out Bristol-Myers’ central launchpad for new therapies in Europe, acquiring a complex in Anagni, Italy, southwest of Rome, that will significantly expand its capacity on the continent.

There are no terms being offered, but this is no small deal. The Anagni campus employs some 700 staffers, and Catalent is planning to go right in — once the deal closes late this year — with a blueprint to build up the operations further as they expand on oral solid, biologics, and sterile product manufacturing and packaging.

This is an uncommon deal, Catalent CEO John Chiminski tells me. But it offers a shortcut for rapid growth that cuts years out of developing a green fields project. That’s time Catalent doesn’t have as the industry undergoes unprecedented expansion around the world.

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Arc­turus ex­pands col­lab­o­ra­tion, adding $30M cash; Ku­ra shoots for $100M raise

→  Rare dis­ease play­er Ul­tragenyx $RARE is ex­pand­ing its al­liance with Arc­turus $ARCT, pay­ing $24 mil­lion for eq­ui­ty and an­oth­er $6 mil­lion in an up­front as the two part­ners ex­pand their col­lab­o­ra­tion to in­clude up to 12 tar­gets. “This ex­pand­ed col­lab­o­ra­tion fur­ther so­lid­i­fies our mR­NA plat­form by adding ad­di­tion­al tar­gets and ex­pand­ing our abil­i­ty to po­ten­tial­ly treat more dis­eases,” said Emil Kakkis, the CEO at Ul­tragenyx. “We are pleased with the progress of our on­go­ing col­lab­o­ra­tion. Our most ad­vanced mR­NA pro­gram, UX053 for the treat­ment of Glyco­gen Stor­age Dis­ease Type III, is ex­pect­ed to move in­to the clin­ic next year, and we look for­ward to fur­ther build­ing up­on the ini­tial suc­cess of this part­ner­ship.”

Neil Woodford. Woodford Investment Management via YouTube

Wood­ford braces po­lit­i­cal storm as UK fi­nan­cial reg­u­la­tors scru­ti­nize fund sus­pen­sion

The shock of Neil Wood­ford’s de­ci­sion to block with­drawals for his flag­ship fund is still rip­pling through the rest of his port­fo­lio — and be­yond. Un­der po­lit­i­cal pres­sure, UK fi­nan­cial reg­u­la­tors are now tak­ing a hard look while in­vestors con­tin­ue to flee.

In a re­sponse let­ter to an MP, the Fi­nan­cial Con­duct Au­thor­i­ty re­vealed that it’s opened an in­ves­ti­ga­tion in­to the sus­pen­sion fol­low­ing months of en­gage­ment with Link Fund So­lu­tions, which tech­ni­cal­ly del­e­gat­ed Wood­ford’s firm to man­age its funds.

Gilead baits new al­liance with $45M up­front, div­ing in­to the busy pro­tein degra­da­tion field

Gilead is jump­ing on board the pro­tein degra­da­tion band­wag­on. And they’re turn­ing to a low-pro­file Third Rock start­up for the ex­per­tise. But if you were look­ing for a trans­for­ma­tion­al deal to kick up fresh en­thu­si­asm for Gilead, you’ll have to re­main pa­tient.

This one will have a long way to go be­fore they get in­to the clin­ic.

The big biotech said Wednes­day morn­ing that it is pay­ing $45 mil­lion up­front and re­serv­ing a whop­ping $2.3 bil­lion in biotech bucks if San Fran­cis­co-based Nurix can point the way to new can­cer ther­a­pies, as well as drugs for oth­er, un­spec­i­fied dis­eases.

A new num­ber 1 drug? Keytru­da tapped to top the 10 biggest block­busters on the world stage by 2024

Analysts may be fretting about Keytruda’s longterm prospects as a host of rival therapies elbow their way to the market. But the folks at Evaluate Pharma are confident that last year’s $7 billion earner is headed for glory, tapping it to beat out the current #1 therapy Humira as AbbVie watches that franchise swoon over the next 5 years.

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In­vestor day prep at Mer­ck in­cludes a new strat­e­gy to pick up the pace on M&A — re­port

Mer­ck’s re­cent deals to buy up two bolt-on biotechs — Ti­los and Pelo­ton — weren’t an aber­ra­tion. In­stead, both ac­qui­si­tions mark a new strat­e­gy to beef up its dom­i­nant can­cer drug op­er­a­tions cen­tered on Keytru­da while look­ing to ad­dress grow­ing con­cerns that too many of its eggs are in the one I/O bas­ket for their PD-1 pro­gram. And Mer­ck is go­ing af­ter more small- and mid-sized buy­outs to calm those fears.