The pay­er back­lash against PTC’s de­flaza­cort for Duchenne MD be­gins with some damn­ing com­par­i­son shop­ping

PTC Ther­a­peu­tics CEO Stu­art Peltz

PTC Ther­a­peu­tics has a few weeks to go be­fore it wraps its $190 mil­lion deal to buy the con­tro­ver­sial drug de­flaza­cort from Marathon Phar­ma­ceu­ti­cals and starts mar­ket­ing the old steroid with a new FDA ap­proval for Duchenne mus­cu­lar dy­s­tro­phy. But even be­fore the biotech de­cides its price, the pay­er kick­back against this drug has al­ready be­gun.

Un­less it’s vir­tu­al­ly free, it’s clear that for now some don’t want to pay for it.

A new analy­sis from the Wash­ing­ton State Health­care Au­thor­i­ty al­so rais­es fresh ques­tions about PTC’s re­peat­ed as­ser­tions — echo­ing Marathon — that they were in­vest­ing in a de­vel­op­ment pro­gram in or­der to make the steroid more wide­ly avail­able, be­yond the small group that they said had been im­port­ing it from over­seas sup­pli­ers.

Of­fi­cials at the Wash­ing­ton State Health­care Au­thor­i­ty start­ed their pol­i­cy re­view by do­ing some com­par­i­son shop­ping be­tween de­flaza­cort and pred­nisone, a gener­ic steroid that sells in the US for about 5 cents per pill, or $55 a year. And that’s the price that the au­thor­i­ty is con­sid­er­ing in de­cid­ing the af­ford­abil­i­ty and cov­er­age on de­flaza­cort for state Med­ic­aid pa­tients.

When Marathon Phar­ma­ceu­ti­cals priced de­flaza­cort at $294 a pill, or $89,000 a year — af­ter Duchenne par­ents had been pay­ing about $1,000 a year to im­port a sup­ply of the drug — the news trig­gered the lat­est in a se­ries of em­bar­rass­ing drug price scan­dals.

Marathon CEO Jeff Aronin de­cid­ed to duck and cov­er, “paus­ing” the launch, and then PTC came along with $140 mil­lion in cash, plus mile­stone mon­ey, and a so­lu­tion to the cri­sis. Now PTC CEO Stu­art Peltz is in the hot seat, lin­ing up a new whole­sale price, adopt­ing the same ar­gu­ment that de­flaza­cort has been avail­able to on­ly a small per­cent­age of the pa­tients in the US.

Once he prices and launch­es, US pa­tients will no longer have le­gal ac­cess to the drug at dis­count prices from a UK sup­pli­er.

The WSHA, though, de­cid­ed to do some fact check­ing ahead of PTC’s de­flaza­cort launch as they con­sid­er their pol­i­cy de­ci­sion.

In their re­port, dat­ed for an April 19 re­view, the au­thor­i­ty found three small stud­ies com­par­ing de­flaza­cort and pred­nisone. These stud­ies pro­vid­ed “low qual­i­ty” ev­i­dence that there is no ev­i­dence of any dif­fer­ence be­tween pred­nisone and de­flaza­cort in im­prov­ing mus­cle strength and some low qual­i­ty ev­i­dence from one tiny and pos­si­bly bi­ased study that de­flaza­cort would trig­ger less weight gain than pred­nisone — though there has nev­er been a study on that end­point to pro­vide sta­tis­ti­cal sig­nif­i­cance.

What about Marathon’s claim, now echoed by PTC, that few­er than 10% of the pa­tient pop­u­la­tion have ac­cess to de­flaza­cort?

The au­thor­i­ty’s pol­i­cy re­port cites CDC fig­ures in­di­cat­ing that 22% of the boys take de­flaza­cort and 14% al­ter­nat­ed be­tween pred­nisone and de­flaza­cort. The rest take pred­nisone. Those fig­ures al­so fall more close­ly to an es­ti­mate by pa­tient ad­vo­cate Chris­tine Mc­Sh­er­ry that at least 25% of the boys with Duchenne MD take de­flaza­cort, pos­si­bly reach­ing as high as 50%.

These pa­tients have been pay­ing more for de­flaza­cort large­ly be­cause they be­lieve their chil­dren do bet­ter, with less weight gain, than they would ex­pe­ri­ence with pred­nisone — a be­lief which has al­so dri­ven use out­side the US. But many are in­censed that Marathon took a cheap al­ter­na­tive and put it through a de­vel­op­ment pro­gram that left them with the ap­proval and a pri­or­i­ty re­view vouch­er as a bonus — prob­a­bly for far less cost than the com­pa­ny had im­plied in claim­ing that it would take years to cov­er the cost of re­search.

“Pred­nisone is the low­er cost, equal­ly ef­fec­tive al­ter­na­tive,” con­clud­ed Ryan Pi­s­tore­si, Phar­mD, As­sis­tant Chief Phar­ma­cy Of­fi­cer at the au­thor­i­ty. And that’s why pred­nisone should be the pre­ferred cor­ti­cos­teroid, with a case-by-case re­view for any spe­cial re­quest for de­flaza­cort.

PTC may have an ap­proved drug to mar­ket in the US, af­ter the FDA re­peat­ed­ly spurned their drug ataluren. But their ex­ec­u­tive team faces a steep mar­ket climb.

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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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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A uni­corn stalks Wall Street in search of IPO cash; CASI Phar­ma in-li­cens­es CD19 ther­a­py from Chi­na’s Ju­ven­tas

→ A herd of up­start biotechs will look to Wall Street for some ma­jor wind­falls this week as a burst of new of­fer­ings con­tin­ues to feed cash in­to the R&D sys­tem. To­day we learned that Bridge­Bio will look to raise in the neigh­bor­hood of $225 mil­lion by of­fer­ing 15 mil­lion shares for $14 to $16 each. And they have a string of joint bookrun­ners: J.P. Mor­gan, Gold­man Sachs, Jef­feries, SVB Leerink, KKR, Piper Jaf­fray, Mizuho Se­cu­ri­ties, BMO Cap­i­tal Mar­kets and Ray­mond James. If suc­cess­ful, Bridge­Bio will emerge with a mar­ket cap of around $1.7 bil­lion. There are 5 biotechs look­ing to IPO this week, in­clud­ing Akero and Pre­vail.

UP­DAT­ED: Sanofi Gen­zyme deserts gene ther­a­py de­vel­op­er Voy­ager Ther­a­peu­tics

While gene ther­a­py com­pa­nies re­joice as the sec­tor gains trac­tion with ap­provals and a flur­ry of M&A ac­tiv­i­ty, one play­er is feel­ing the heat.

Back in 2015, Voy­ager Ther­a­peu­tics joined forces with Sanofi Gen­zyme in a deal worth up to $845 mil­lion ($100 mil­lion up­front + a po­ten­tial $745 mil­lion in mile­stones) to co-de­vel­op gene ther­a­pies for se­vere cen­tral ner­vous sys­tem dis­or­ders. But two years lat­er, the French drug­mak­er re­treat­ed, elect­ing to not pick up the op­tion to work on Voy­ager’s Parkin­son’s dis­ease pro­gram. (Last year, the FDA dis­ap­point­ed Voy­ager, telling the com­pa­ny that it was not open to an ac­cel­er­at­ed fil­ing on the Parkin­son’s drug on the ba­sis of Phase II da­ta — in­stead of re­quir­ing an ad­di­tion­al piv­otal study.)

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.

John Oyler, Founder & CEO of BeiGene, at the US-China Biopharma Innovation and Investment Summit in Shanghai on October 23, 2018; Credit: Endpoints News, PharmCube

UP­DAT­ED: As Bris­tol-My­ers/Cel­gene tie up loose ends, BeiGene pock­ets $150M from PD-1 breakup

As soon as Bristol-Myers Squibb announced its $74 billion buyout for Celgene, BeiGene emerged as a prominent example of a player whose pact with the big biotech could sour, as its PD-1 candidate seems to overlap with Opdivo. After six months of suspense, the partners say they are finally bringing the 2-year-old deal to an amicable end.

BeiGene $BGNE gets $150 million for the termination in addition to full global rights to tislelizumab. In 2017 Celgene had paid $263 million in upfront license fees to develop the PD-1 inhibitor for solid cancers in the US, Europe, Japan and the rest of the world outside Asia. It also threw in a $150 million equity investment in exchange for BeiGene handling its commercial operations — think Abraxane, Revlimid and Vidaza — in China.

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Exterior of the 1 million square foot Discovery Labs in Upper Merion, PA (PR Newswire)

Philadel­phia cham­pi­ons life sci­ences 'co-work­ing,' re­viv­ing for­mer GSK cam­pus in $500M makeover

In a boost to Philadel­phia’s thriv­ing life sci­ences scene, a for­mer Glax­o­SmithK­line cam­pus and a near­by site has been turned in­to what its de­vel­op­er calls “the largest cowork­ing ecosys­tem” for health­care com­pa­nies in the coun­try.

The Dis­cov­ery Labs, a com­pa­ny spawned by MLP Ven­tures, has se­lect­ed two lo­ca­tions in the King of Prus­sia area as the $500 mil­lion test case for its strat­e­gy of ac­quir­ing and con­vert­ing old phar­ma­ceu­ti­cal R&D fa­cil­i­ties world­wide. The sites add up to 1.64 mil­lion square feet.

Gene ther­a­pies seize the top of the list of the most ex­pen­sive drugs on the plan­et — and that trend has just be­gun

Anyone looking for a few simple reasons why the gene therapy field has caught fire with the pharma giants need only look at the new list of the 10 most expensive therapies from GoodRx.

Two recently approved gene therapies sit atop this list, with Novartis’ Zolgensma crowned the king of the priciest drugs at $2.1 million. Right below is Luxturna, the $850,000 pioneer from Spark, which Roche is pushing hard to acquire as it adds a gene therapy group to the global mix.

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Ted Love. HAVERFORD COLLEGE

Glob­al Blood Ther­a­peu­tics poised to sub­mit ap­pli­ca­tion for ac­cel­er­at­ed ap­proval, with new piv­otal da­ta on its sick­le cell dis­ease drug

Global Blood Therapeutics is set to submit an application for accelerated approval in the second-half of this year, after unveiling fresh data from a late-stage trial that showed just over half the patients given the highest dose of its experimental sickle cell disease drug experienced a statistically significant improvement in oxygen-wielding hemoglobin, meeting the study's main goal.

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