Lib­er­tar­i­an ideas on FDA dereg­u­la­tion threat­en biotech; It's time for some re­al pow­er to re­form drug prices, rea­son­ably

Do you sin­cere­ly want to dereg­u­late drug de­vel­op­ment?

Sil­i­con Val­ley’s Bal­a­ji Srini­vasan had quite a habit of tack­ling the FDA on Twit­ter, crit­i­ciz­ing the agency for its slow and back­ward ways. But all that came back to haunt him af­ter re­porters picked up on the news that he and fel­low ‘seast­ead­er’ Jim O’Neill had been in to chat with Don­ald Trump about top jobs at the FDA. Sud­den­ly, that Twit­ter chan­nel van­ished in­to thin air, as we re­port­ed first. But his Tweets were saved, and live on, even as we hear that Srini­vasan may not be in line for that job any more. Trump’s fi­nal pick to run the FDA will have a huge in­flu­ence on how the agency plans to speed drug de­vel­op­ment. And we have a right and a need to suss things out for our­selves. So far, this episode ranks as an­oth­er rea­son to sus­pect that the FDA may soon be head­ed for a wrong turn, es­pe­cial­ly if the new fo­cus is on a sub­stan­tial dereg­u­la­tion of drug de­vel­op­ment born out of a dis­gust for the bu­reau­cra­cy.

Medicare price ne­go­ti­a­tions can ben­e­fit bio­phar­ma too.

Speak­ing of Trump, our new pres­i­dent got the chance to clar­i­fy his think­ing re­gard­ing drug prices in a week­end in­ter­view with The Wash­ing­ton Post. As we sus­pect­ed, his main weapon in the war against list prices will be Medicare ne­go­ti­a­tions — now banned by in­dus­try-friend­ly law­mak­ers. The in­dus­try des­per­ate­ly doesn’t want to see this, but in re­al­i­ty it makes for an ef­fec­tive way to get ac­tu­al dis­count prices on dis­play for all to see. The Amer­i­can pub­lic has a right to see what’s ac­tu­al­ly be­ing paid for ther­a­peu­tics, and this is one way to set a base­line dis­count price that is read­i­ly trans­par­ent. The free ride on ever-ris­ing drug prices is over, and we all know it. The soon­er this hap­pens, the bet­ter for every­one. If a price can’t sur­vive the light of day, it’s too high. In the mean­time, the in­dus­try can move more to­ward a per­for­mance-based pric­ing method.

Mallinck­rodt joins the price goug­ing club, and gets away with “mur­der.”

One of the rea­sons why drug prices are so con­tro­ver­sial is that the fed­er­al gov­ern­ment is vir­tu­al­ly help­less when it comes to pre­vent­ing a com­pa­ny from jack­ing up prices as­tro­nom­i­cal­ly overnight. Quest­cor proved that more than three years ago, when it brazen­ly bought a ri­val to Ac­thar to pre­vent com­pe­ti­tion. And Mallinck­rodt bought in to that rigged game by buy­ing Quest­cor. This week, Mallinck­rodt was cit­ed for il­le­gal­ly block­ing com­pe­ti­tion, but got away with on­ly a $100 mil­lion fine. The com­pa­ny paid for that with the in­creased rev­enue it earned on Ac­thar in just 2015, as its price con­tin­ued to soar. This is what Trump means when he says phar­ma is get­ting away with mur­der. It may take years for a ri­val to get ap­proved and on the mar­ket. And Mallinck­rodt is still free to jack prices. This has to end with some kind of ef­fec­tive le­gal mech­a­nism to pre­vent abus­es. Your next Mar­tin Shkre­li or Mallinck­rodt is sit­ting just around the cor­ner. And the longer we wait, the more like­ly it is that re­form will overzeal­ous­ly dam­age the way in­no­va­tion is fi­nanced in bio­phar­ma.

Our lat­est lists on ven­ture cash un­der­score sol­id fun­da­men­tals for biotech. Let’s not screw it up now.

Biotech is de­pen­dent on the flow of bil­lions of dol­lars in ven­ture cash. And we love noth­ing quite as much as track­ing where the mon­ey is com­ing from as well as what re­gions it is go­ing to. Al­ways enor­mous­ly pop­u­lar with read­ers, this year’s sto­ries paint a sim­ple tale of the dom­i­nant role played by two cru­cial hubs — Boston/Cam­bridge and the Bay Area — as well as a group of savvy in­vestors who are in­stru­men­tal in fos­ter­ing and fund­ing new in­no­va­tion in drug de­vel­op­ment. That ma­chine has been hum­ming along nice­ly now for sev­er­al years, point­ing to the fact that the fun­da­men­tals in pri­vate biotech are strong and durable. That bodes well for the years ahead, as ma­jor re­forms are be­ing planned, which we should all ap­pre­ci­ate. Re­form­ers might like to keep that in mind.

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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David de Graaf now has his $28.5M launch round in place, build­ing a coen­zyme A plat­form in his lat­est start­up

Long­time biotech ex­ec David de Graaf has the cash he needs to set up the pre­clin­i­cal foun­da­tion for his coen­zyme A me­tab­o­lism com­pa­ny Comet. A few high-pro­file in­vestors joined the ven­ture syn­di­cate to sup­ply Comet with $28.5 mil­lion in launch mon­ey — enough to get it two years in­to the plat­form-build­ing game, with­in knock­ing dis­tance of the clin­ic.

Canaan jumped in along­side ex­ist­ing in­vestor Sofinno­va Part­ners to co-lead the round, with par­tic­i­pa­tion by ex­ist­ing in­vestor INKEF Cap­i­tal and new in­vestor BioIn­no­va­tion Cap­i­tal.

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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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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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.