Sarep­ta shares yo-yo in the red on an­oth­er glimpse of stel­lar re­sults for Duchenne MD gene ther­a­py — what gives?

There’s just no telling what a stock will do — even when you stack the deck in your fa­vor.

Tonight Sarep­ta $SRPT came out with a GREAT piece of news. Adding to the stel­lar, but very ear­ly glimpse of pos­i­tive da­ta for their gene ther­a­py for Duchenne mus­cu­lar dy­s­tro­phy, the biotech up­dat­ed their da­ta with a set of new num­bers that would ap­pear to add to the over­all glow of the pro­gram.


And then its stock took a dive, falling close to 10% in a mat­ter of min­utes af­ter the de­layed re­lease hit the wires. Then it bounced back, on­ly a few points in the red. What gives?

Is this sell the news? And why would that be? Shorts cov­er­ing their po­si­tions? Sev­er­al in­s­ta-re­ac­tions in the Twit­ter­verse sus­pect­ed that the fourth pa­tient did too well, see­ing a 182% in­crease in dy­s­trophin, the much-want­ed el­e­ment that cre­ates hav­oc for pa­tients, whose mus­cles waste and die with­out it.

As mea­sured by West­ern blot, pa­tient 4 showed ro­bust lev­els of mi­cro-dy­s­trophin, with a mean of 182.7% com­pared to nor­mal uti­liz­ing Sarep­ta’s method, or 222% com­pared to nor­mal pur­suant to Na­tion­wide Chil­dren’s quan­tifi­ca­tion of Sarep­ta’s method that ad­justs for fat and fi­brot­ic tis­sue.

Was that too much? Or too vari­able?

Baird’s Bri­an Sko­r­ney, a strong Sarep­ta bull, heard the dis­tur­bance in the force, but isn’t about to crum­ble now.

We ex­pect to de­bate con­cerns over noisy CK da­ta and im­pli­ca­tions of over­ex­pres­sion but see these as very mi­nor points on an oth­er­wise very im­pres­sive da­ta set and re­it­er­ate our Out­per­form.

I asked Sko­r­ney — one of the more thought­ful an­a­lysts in the bunch — about the red num­bers. His re­ply:

I think the ini­tial neg­a­tive re­ac­tion is due to the high ex­pres­sion lev­els that are rem­i­nis­cent of the con­cerns around vari­abil­i­ty with Bio­Marin’s Fac­tor VI­II pro­gram and some re­bound in CK lev­els in the first three pa­tients. I don’t re­al­ly ex­pect any af­ter­hours down move to be sus­tained to­mor­row though.

Sev­er­al an­a­lysts and re­porters help­ful­ly pro­vid­ed the em­bar­goed thumbs up, with quotes. Yet there, but for the grace of the biotech Gods, go I. I was tipped to the re­lease… but nev­er ac­tu­al­ly got it as of­fered.

Again, an­oth­er mys­tery for those of us who cov­er the field.

On the face of it, Sarep­ta would seem to have every­thing go­ing in its fa­vor. The gene ther­a­py is pro­vid­ing the nec­es­sary ev­i­dence of a sol­id bio­mark­er re­sponse. Even some signs in the sin­gle arm study — with­out the com­par­i­son da­ta that would help prove that it makes a dif­fer­ence  — that it’s help­ing pa­tients im­prove phys­i­cal­ly.

And this is a ter­ri­ble dis­ease. An ac­tu­al cure would be a god­send.

Sarep­ta has an un­for­tu­nate his­to­ry when it comes to his­tor­i­cal com­par­isons, though, which raised sig­nif­i­cant doubts in the agency that eteplirsen works.

Any­way, be­fore the post-mar­ket trad­ing closed Wednes­day evening, the stock had edged back in­to the green, so the de­fend­ers of the faith had held the line, briefly. By mid-morn­ing Thurs­day, the stock was back in the red again.

I’ll wait for some ad­di­tion­al com­men­tary. In the mean­time, caveat emp­tor.

You’re on your own, as al­ways.

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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How small- to mid-sized biotechs can adopt pa­tient cen­tric­i­ty in their on­col­o­gy tri­als

By Lucy Clos­sick Thom­son, Se­nior Di­rec­tor of On­col­o­gy Pro­ject Man­age­ment, Icon

Clin­i­cal tri­als in on­col­o­gy can be cost­ly and chal­leng­ing to man­age. One fac­tor that could re­duce costs and re­duce bar­ri­ers is har­ness­ing the pa­tient voice in tri­al de­sign to help ac­cel­er­ate pa­tient en­roll­ment. Now is the time to adopt pa­tient-cen­tric strate­gies that not on­ly fo­cus on pa­tient needs, but al­so can main­tain cost ef­fi­cien­cy.

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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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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Af­ter watch­ing its share price soar on a Bloomberg re­port and heat­ed ru­mors, Bio­haven stock takes a bil­lion-dol­lar bath

Back in April, Biohaven Pharmaceutical became one hot biotech stock $BHVN based on a report in Bloomberg that some “potential bidders” had been kicking the tires at the biotech, which has a lead drug for migraines. Then the rumor mill really started to smoke when execs canceled a presentation at an investor conference a little more than a week ago.

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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.”

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.

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.