Af­ter be­ing spurned at the FDA, PTC buys a con­tro­ver­sial Duchenne MD ther­a­py from Marathon

Af­ter be­ing stung by a fierce back­lash over its plans to sell a cheap, old steroid for Duchenne mus­cu­lar dy­s­tro­phy for $89,000 a year, Marathon Phar­ma­ceu­ti­cals is get­ting out. The com­pa­ny an­nounced this morn­ing that it is sell­ing de­flaza­cort (Em­flaza) to Duchenne MD play­er PTC Ther­a­peu­tics $PTCT, which has been strug­gling to get the FDA to pro­vide a se­ri­ous re­view for its own failed drug.

Marathon is get­ting $140 mil­lion in cash and stock in the deal, along with a shot at a one-time $50 mil­lion sales bonus on the ta­ble and a roy­al­ty stream that will ac­count for a low-to-mid 20s per­cent­age of the rev­enue.

PTC Ther­a­peu­tics CEO Stu­art Peltz

PTC CEO Stu­art Peltz doesn’t say in the re­lease how much he plans to charge for de­flaza­cort. The com­pa­ny’s stock slid 13% af­ter the news hit.

Marathon trig­gered a storm of crit­i­cism with its plans, which is con­tin­u­ing un­abat­ed now with a new fo­cus on the FDA’s role in the ap­proval. A large num­ber of Duchenne par­ents were able to buy gener­ic de­flaza­cort over­seas for around $1,000 a year, and crit­ics saw Marathon’s move as an­oth­er in a se­ries of price goug­ing scan­dals.

That scan­dal is now PTC’s to deal with.

“With our near­ly 20-year com­mit­ment to the Duchenne com­mu­ni­ty, it is deeply mean­ing­ful for us to bring this crit­i­cal ther­a­py to U.S. pa­tients,” said Peltz in a state­ment. “We be­lieve Em­flaza is a dis­ease-mod­i­fy­ing ther­a­py that has been shown to slow dis­ease pro­gres­sion. In keep­ing with PTC’s mis­sion, we are ex­cit­ed to work with the com­mu­ni­ty to raise the stan­dard of care for DMD pa­tients.”

Claim­ing that de­flaza­cort is a dis­ease mod­i­fy­ing ther­a­py will sur­prise many in the Duchenne com­mu­ni­ty. Like any steroid, it strength­ens pa­tients at a cost. Many of the par­ents came to pre­fer de­flazafort over pred­nisone be­cause it is as­so­ci­at­ed with less weight gain.

In ac­quir­ing the old steroid to be sold as a brand­ed ther­a­py, PTC is al­so adopt­ing Marathon’s claim that the ap­proval makes it pos­si­ble to ex­pand ac­cess — now “lim­it­ed to a small num­ber of pa­tients,” Peltz said in a call with an­a­lysts to­day — to all pa­tients over the age of 5.

Mark Rather, PTC CCO

“We plan to re­ex­am­ine the price of Em­flaza,” added Mark Rothera, PTC’s chief com­mer­cial of­fi­cer. “This is a clas­sic or­phan drug launch that we’re fac­ing in the Unit­ed States.”

“It re­al­ly wasn’t avail­able to pa­tients,” Peltz told an­a­lysts, es­ti­mat­ing that few­er than 10% of the pa­tients had ac­cess to the steroid.

Ac­cord­ing to Duchenne ad­vo­cate Chris­tine Mc­Sh­er­ry, though, quite a few fam­i­lies had no prob­lem get­ting over­seas sup­plies of the steroid. Mc­Sh­er­ry ini­tial­ly es­ti­mat­ed that 40% to 50% of the DMD kids are al­ready on de­flaza­cort and will now be forced to switch to the high­er priced US sup­pli­er, then ad­just­ed that down to a con­ser­v­a­tive 25%.

Law­mak­ers, in­clud­ing Sen­a­tor Bernie Sanders, have blast­ed Marathon’s claims that it need­ed to charge a high price to even­tu­al­ly re­gain the cost of de­vel­op­ment.

Peltz has tried and failed to prove that PTC’s drug ataluren — which failed back-to-back stud­ies and re­cent­ly flopped for cys­tic fi­bro­sis — could ben­e­fit Duchenne par­ents. He con­tin­ues to in­sist that the “to­tal­i­ty” of the da­ta proves its worth, but the FDA re­fused to even file their ap­pli­ca­tion, say­ing they didn’t have the da­ta need­ed for a re­view. PTC, though, re­cent­ly lever­aged the reg­u­la­tions to force a PDU­FA date for their drug. And ex­ecs styled this new deal as an open­ing to start US com­mer­cial­iza­tion ef­forts as it pre­pares to push ataluren in­to the mar­ket.

Eu­ro­pean reg­u­la­tors, mean­while, did give the drug a con­di­tion­al ap­proval, ex­tend­ing that with a new re­quire­ment to com­plete an ad­di­tion­al study in the next five years.

Brent Saunders [Getty Photos]

UP­DAT­ED: Ab­b­Vie seals $63B deal to buy a trou­bled Al­ler­gan — spelling out $1B in R&D cuts

Brent Saunders has found his way out of the current fix he’s in at Allergan $AGN. He’s selling the company to AbbVie for $63 billion in the latest example of the hot M&A market in biopharma.

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Turned back at the FDA, Im­muno­Gen is ax­ing 220 staffers, sell­ing pro­grams and hun­ker­ing down for a new PhI­II gam­ble

After being stymied by FDA regulators who were unconvinced by ImmunoGen’s $IMGN desperation shot at an accelerated OK based on a secondary endpoint, the struggling biotech is slashing its workforce, shuttering R&D projects and looking for buyers to pick up some of its experimental cancer assets as it goes back into a new Phase III with the lead drug.

We found out last month that the FDA had batted back their case for an accelerated approval of their antibody-drug conjugate mirvetuximab soravtansine, which had earlier failed a Phase III study for ovarian cancer. Now the other shoe is dropping.

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Bridge­Bio takes crown for biggest biotech IPO of 2019, as fel­low uni­corn Adap­tive rais­es of­fer­ing size and price

Bridge­Bio Phar­ma and Adap­tive Biotech­nolo­gies have not just up­sized IPO of­fer­ings — the pair of uni­corns have al­so raised their of­fer­ing prices above the range, haul­ing in a com­bined $648.5 mil­lion.

Neil Ku­mar’s Bridge­Bio Phar­ma, found­ed in 2015, has a sta­ble of com­pa­nies fo­cused on dis­eases that are dri­ven by de­fects in a sin­gle gene — en­com­pass­ing der­ma­tol­ogy, car­di­ol­o­gy, neu­rol­o­gy, en­docrinol­o­gy, re­nal dis­ease, and oph­thal­mol­o­gy — and can­cers with clear ge­net­ic dri­vers. The start­up mill birthed a pletho­ra of firms such as Ei­dos, Navire, QED Ther­a­peu­tics and Pelle­Pharm, which func­tion as its sub­sidiaries.

As­traZeneca chal­lenges Roche on front­line SCLC af­ter seiz­ing an in­ter­im win — and Mer­ck may not be far be­hind

The crowded playing field in the PD-1/L1 marketing game is about to get a little more complex.

This morning AstraZeneca reported that its CASPIAN study delivered a hit in an interim readout for their PD-L1 Imfinzi combined with etoposide and platinum-based chemotherapy options for frontline cases of small cell lung cancer, a tough target which has already knocked back Bristol-Myers’ shot in second-line cases. The positive data  — which we won’t see before they roll it out at an upcoming scientific conference — give AstraZeneca excellent odds of a quick vault to challenging Roche’s Tecentriq-chemo combo, approved 3 months ago for frontline SCLC in a landmark advance.

“This is the first trial offering the flexibility of combining immunotherapy with different platinum-based regimens in small cell lung cancer, expanding treatment options,” noted AstraZeneca cancer R&D chief José Baselga in a statement.

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Sanofi/Re­gen­eron mus­cle ahead of a ri­val No­var­tis/Roche team, win first ap­proval in key rhi­nos­i­nusi­tis field

Re­gen­eron and their part­ners at Sanofi have beat the No­var­tis/Roche team to the punch on an­oth­er key in­di­ca­tion for their block­buster an­ti-in­flam­ma­to­ry drug Dupix­ent. The drug team scored an ac­cel­er­at­ed FDA ap­proval for chron­ic rhi­nos­i­nusi­tis with nasal polyps, mak­ing this the first such NDA for the field.

An­a­lysts have been watch­ing this race for awhile now, as Sanofi/Re­gen­eron won a snap pri­or­i­ty re­view for what is now their third dis­ease in­di­ca­tion for this treat­ment. And they’re not near­ly done, build­ing up hopes for a ma­jor fran­chise.

Novotech CEO Dr. John Moller

Novotech CRO Award­ed Frost & Sul­li­van Best Biotech CRO Asia-Pa­cif­ic 2019

Known in the in­dus­try as the Asia-Pa­cif­ic CRO, Novotech is now lead CRO ser­vices provider for the grow­ing num­ber of in­ter­na­tion­al biotechs se­lect­ing the re­gion for their stud­ies.

Re­flect­ing this Asia-Pa­cif­ic growth, Novotech staff num­bers are up 20% since De­cem­ber 2018 to 600 in-house clin­i­cal re­search peo­ple across a full range of ser­vices, across the re­gion.

Novotech’s ca­pa­bil­i­ties have been rec­og­nized by an­a­lysts like Frost & Sul­li­van, most re­cent­ly with the pres­ti­gious Asia-Pa­cif­ic CRO Biotech of the year award for best prac­tices in clin­i­cal re­search for biotechs for the fifth year. See oth­er awards here.

Af­ter rais­ing $158M, this up­start's founders have star back­ers and plans to break new ground in gene ther­a­py

Back in 2014, Stephanie Tagliatela opted to take an early exit out of her PhD program after working in Mark Bear’s lab at MIT, where she specialized in the synaptic connections between neuronal cells in the brain. She never finished that PhD, but she and fellow MIT student Kartik Ramamoorthi — who was on the founding team at Voyager — came away with some ideas for a gene therapy startup.

Today, fully 5 years later, she and Ramamoorthi are taking the wraps off of a $104 million mega-round designed to take the cumulative work of their preclinical formative stage for Encoded Therapeutics into human studies. They’ve now raised $158 million since starting out in Illumina’s incubator in the Bay Area, and they believe they are firmly on track to do something unique in gene therapy.

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Richard Gonzalez testifying in front of Senate Finance Committee, February 2019 [AP Images]

Ab­b­Vie's $63B buy­out spot­lights the re­turn of ma­jor M&A deals — de­spite the back­lash

Big time M&A is back. But for how long?

Over the past 18 months we’ve now seen three major buyouts announced: Takeda/Shire; Bristol-Myers/Celgene and now AbbVie/Allergan. And with this latest deal it’s increasingly clear that the sharp fall from grace suffered by high-profile players which have seen their share prices blasted has created an opening for the growth players in big pharma to up their game — in sharp contrast to the popular bolt-on deals that have been driving the growth strategy at Novartis, Merck, Roche and others.

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Two biotech uni­corns swell pro­posed IPOs, eye­ing a $600M-plus wind­fall

We’ve been wait­ing for the ar­rival of Bridge­Bio’s IPO to top off the wave of new biotech of­fer­ings sweep­ing through Nas­daq at the end of H1. And now we learn that it’s been sub­stan­tial­ly up­sized.

Ini­tial­ly pen­ciled in at a uni­corn-sized $225 mil­lion, the KKR-backed biotech has spiked that to the neigh­bor­hood of $300 mil­lion, look­ing to sell 20 mil­lion shares at $14 to $16 each. That’s an added 5 mil­lion shares, re­ports Re­nais­sance Cap­i­tal, which fig­ures the pro­posed mar­ket val­u­a­tion for Neil Ku­mar’s com­pa­ny at $1.8 bil­lion.