Strug­gling Sarep­ta bounces back as an­a­lysts cheer the first trick­le of rev­enue for DMD drug

Sarep­ta CEO Ed Kaye

Shares of Sarep­ta shot up 21% on Tues­day, buoyed by some en­thu­si­as­tic re­marks for the biotech’s first round of sales of its Duchenne mus­cu­lar dy­s­tro­phy drug Ex­ondys 51, which were talked up by CEO Ed Kaye at the JP Mor­gan con­fer­ence. And a deal to kick off a gene ther­a­py re­search pact may have helped.

The sales fig­ure for Ex­ondys 51 didn’t amount to much. Sales hit $5.4 mil­lion for the first three months the drug was on the mar­ket, which fell be­hind Leerink’s es­ti­mate and gen­er­al con­sen­sus — pegged by Joseph Schwartz at $7.9 mil­lion and $6 mil­lion. But with more than 250 DMD pa­tients amenable to an ex­on-skip­ping ther­a­py seek­ing cov­er­age, and a pos­i­tive as­sess­ment that Sarep­ta could man­age the back­lash that’s grown among pay­ers, the biotech earned a 21% spike in its stock price.

This is one of the most con­tro­ver­sial drugs to ever hit the mar­ket. The FDA’s la­bel spec­i­fies that there’s no proof of clin­i­cal ben­e­fit, and the in­ter­nal war at the FDA over this drug en­cour­aged a slate of in­sur­ers to ei­ther refuse to cov­er it or lim­it it, caus­ing the big share price spike that greet­ed an OK to melt away.

But Sarep­ta’s stock has been on a roller coast­er ride for years now, lurch­ing from big gains to big loss­es with each turn of the card.

Bri­an Sko­r­ney, Baird

Baird’s Bri­an Sko­r­ney was and is an op­ti­mist.

“While we ex­pect hur­dles to get­ting in­sur­ance au­tho­riza­tion and putting a plan in place for each pa­tient to have a week­ly in­fu­sion, we be­lieve the vast ma­jor­i­ty of these pa­tients will con­vert to rev­enue in 1H17,” he wrote. “We al­so be­lieve this is a strong in­di­ca­tor of ini­tial de­mand and ex­pect new start forms to con­tin­ue to in­crease over 2017.”

Gena Wang at Jef­feries, who nev­er ex­pect­ed to see this drug on the mar­ket, still ex­pects plen­ty of stub­born push­back from pay­ers. She writes:

Mgmt guid­ance on Ex­ondys 51 ac­cess and re­im­burse­ment con­sis­tent with our ex­pec­ta­tion of strict pay­er push­back. On­ly 13% of cov­ered lives have a fa­vor­able pol­i­cy, while 8% of cov­ered lives are deny­ing cov­er­age, per mgmt. Rest (79%) are ei­ther pend­ing pol­i­cy de­ci­sions, cov­er­ing with re­stric­tions, and/or re­view­ing pts case by case, in line with our ex­pec­ta­tions of strict pay­er push­back.

The drug is slat­ed to cost more than $300,000 a year.

The Cam­bridge, MA-based Sarep­ta, mean­while, signed up to sup­port a mi­crody­s­trophin gene ther­a­py pro­gram at Na­tion­wide Chil­dren’s Hos­pi­tal run by Jer­ry Mendell and Louise Rodi­no-Kla­pac. Sarep­ta gets an op­tion to ac­quire the pro­gram, with a Phase I/IIa tri­al start­ing lat­er this year.

Sarep­ta al­so struck a deal to part­ner with Ox­ford, UK-based Sum­mit in Oc­to­ber on a ther­a­py that fol­lows a com­ple­men­tary path­way to treat DMD.

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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Right back at you, Pfiz­er: BeiGene and a Pfiz­er spin­out launch a new­co to de­vel­op a MEK/BRAF in­hibitor that could ri­val $11.4B com­bo

A day af­ter Pfiz­er bought Ar­ray and its ap­proved can­cer com­bo, BeiGene and Pfiz­er spin­out Spring­Works have part­nered in launch­ing a new biotech that has an eye on the very same mar­ket the phar­ma gi­ant just paid bil­lions for. And they’re plan­ning on us­ing an ex-Pfiz­er drug to do it.

In a nut­shell, Chi­na’s BeiGene is toss­ing in a pre­clin­i­cal BRAF in­hibitor — BGB-3245, which cov­ers both V600 and non-V600 BRAF mu­ta­tions — for a big stake in a new, joint­ly con­trolled biotech called Map­Kure with Bain-backed Spring­Works.

Step­ping on Roche's toes, Mer­ck cuts in­to SCLC niche with third-line Keytru­da OK

In the in­creas­ing­ly crowd­ed check­point race, small cell lung can­cer has been a rare area where Roche, a sec­ond run­ner-up, has a lead over the en­trenched lead­ers Mer­ck and Bris­tol-My­ers Squibb. But Mer­ck is fi­nal­ly mak­ing some head­way in that di­rec­tion with the lat­est ap­proval for its PD-1 star.

The lat­est green light en­dors­es Keytru­da in the third-line treat­ment of metasta­t­ic SCLC, where it would be giv­en to pa­tients whose dis­ease ei­ther don’t re­spond to or re­lapse af­ter chemother­a­py, which would have fol­lowed at least one pri­or line of ther­a­py.

Sanofi aligns it­self with Google to stream­line drug de­vel­op­ment

Tech­nol­o­gy is bleed­ing in­to health­care, and big phar­ma is rid­ing the wave. Sanofi $SNY ap­point­ed its first chief dig­i­tal of­fi­cer this Feb­ru­ary, fol­low­ing the foot­steps of its peers. By May, the French drug­mak­er and some of its big phar­ma com­pa­tri­ots joined forces with Google par­ent Al­pha­bet’s Ver­i­ly unit to aug­ment clin­i­cal tri­al re­search. On Tues­day, the Parisian com­pa­ny tied up with Google to ac­cess its cloud com­put­ing and ar­ti­fi­cial in­tel­li­gence tech to spur the de­vel­op­ment of new ther­a­pies.

UP­DAT­ED: Roche fields first ap­proval for Ro­z­lytrek in the run-up to a show­down with Bay­er, Pfiz­er

While it’s wait­ing to hear back from FDA reg­u­la­tors, Roche is be­gin­ning the vic­to­ry lap for en­trec­tinib in Japan.

Roche is giv­ing Bay­er a run for their mon­ey with this tu­mor-ag­nos­tic drug, which tar­gets NTRK gene fu­sions. Now dubbed Ro­z­lytrek, it’s sanc­tioned to treat adult and pe­di­atric pa­tients in Japan with neu­rotroph­ic ty­ro­sine re­cep­tor ki­nase fu­sion-pos­i­tive, ad­vanced re­cur­rent sol­id tu­mors.

In­vestors fret as VBI's hep B vac­cine fails key sec­ondary PhI­II study goal

Sobered by mount­ing costs, Dy­navax $DVAX last month made the de­ci­sion to fo­cus all its re­sources on its 2017-ap­proved he­pati­tis B vac­cine Hep­lisav-B, which ri­vals and su­per­sedes the ef­fi­ca­cy and con­ve­nience pro­file of GSK’s $GSK es­tab­lished En­ger­ix-B. The Cal­i­for­nia-based com­pa­ny will be on the look­out for an­oth­er com­peti­tor — VBI Vac­cines, which on Mon­day un­veiled late-stage da­ta on its hep B vac­cine: Sci-B-Vac.

Image: Shutterstock

Gene ther­a­py R&D deals turn red hot as Big Phar­ma steps up to play

This September will mark the 20th anniversary of the death of Jesse Gelsinger, a young man suffering from X-linked genetic disease of the liver. He was killed in a gene therapy study conducted by Penn’s James Wilson, and the entire field endured a lengthy deep freeze as the field grappled with the safety issues inherent in the work.

Some thought gene therapy R&D would never survive. But it did. And this year marked a landmark approval for Zolgensma, a new gene therapy for spinal muscular atrophy Novartis priced at $2.1 million.

“Gene therapy is the hottest item on the block now. But there was a time when we first got into this trial, where there wasn’t a person in the world who believed that gene therapy would work. We have to remember that,” noted gene therapy investigator Jerry Mendell told SMA News Today.

We’re still right on the pioneering frontier when it comes to getting approvals for gene therapies and launching marketing campaigns with the European green light for bluebird's leading program last Friday underscoring the nascent nature of the field. But gene therapy R&D is booming, and has been for several years now.

The rapid growth of gene therapy clinical development is well known, but we decided to put some numbers on it, to quantify what’s going on. DealForma chief Chris Dokomajilar took a lot over the past 10 years, as the number of deals, R&D partnerships and buyouts steadily gained steam, spiking last year and on track to maintain the surge in 2019.

The upfronts and totals for the dollars on deals so far in 2019 is already close to the 2018 mark, underscoring a new phase of negotiations as the major players step up to gain a piece of the late-stage and commercial action.

Once again, we’re looking at an “overnight” biotech success story, decades in the making.

At some point, that may start to brake the numbers we’re seeing. But for now, as rivals line up to compete for frontline prominence across a range of diseases, the arrows are all pointed north.

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Albert Bourla appears before the Senate Committee on Finance for a hearing on prescription drug pricing on Capitol Hill in Washington, DC, February 26, 2019. Chris Kleponis for CNP via AP Images

UP­DAT­ED: Pfiz­er CEO Al­bert Bourla is back in the M&A game, but why is he pay­ing $11.4B for Ar­ray?

Pfiz­er $PFE has cut short its time on the side­lines of bio­phar­ma M&A.

Mon­day morn­ing the phar­ma gi­ant un­veiled an $11.4 bil­lion deal to ac­quire Ar­ray Bio­Phar­ma, beef­ing up its on­col­o­gy work and adding a new re­search hub in Boul­der, Col­orado to its glob­al op­er­a­tions.

At $48 a share, Ar­ray $AR­RY in­vestors will be get­ting a 62% pre­mi­um off the Fri­day close of $29.59.

Pfiz­er, which has strug­gled to gain all the up­side promised in past buy­outs like Medi­va­tion, high­light­ed the ac­qui­si­tion of 2 ap­proved drugs in the deal — Braftovi (en­co­rafenib) and Mek­tovi (binime­tinib).

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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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