Trump needs to prove he's se­ri­ous about sci­ence; PTC's lat­est move is a jaw drop­per

End­points as­sess­es the big bio­phar­ma sto­ries of the week, with a lit­tle added com­men­tary on what they mean for the in­dus­try.

Two big steps back for the Trump ad­min­is­tra­tion

Elias Zer­houni

With Don­ald Trump in the White House, it’s al­ways one step for­ward and two steps back for bio­phar­ma. So last week we had Scott Got­tlieb’s nom for FDA com­mis­sion­er (a big step for­ward) fol­lowed by this week’s news that Trump’s bud­get plans to slash NIH spend­ing by close to $6 bil­lion, while dou­bling in­dus­try’s fees to the FDA (you guessed it.)

Both bud­get items are non­starters. Blast­ing the re­search bud­get at the NIH would gut the world’s most promis­ing dis­cov­ery ac­tiv­i­ties. In an in­ter­view with Forbes’  Matthew Her­p­er, for­mer NIH chief Elias Zer­houni not­ed that if you cut the NIH’s bud­get by 20%, you’d es­sen­tial­ly close the tap on new grants, a knock­out blow to a large group of in­ves­ti­ga­tors which would be es­pe­cial­ly dev­as­tat­ing for young sci­en­tists.

Com­ing as it does af­ter years of flat bud­get­ing, bro­ken on­ly re­cent­ly by an in­crease last year, Trump is play­ing ir­re­spon­si­ble pol­i­tics. The idea of sim­ply dou­bling in­dus­try fees is al­so a stick in the eye to drug de­vel­op­ers.

And the sur­vey says…

Speak­ing of Scott Got­tlieb.

We ran a snap poll over the week­end to get a bet­ter sense of what bio­phar­ma was think­ing in re­gards to his nom­i­na­tion. And the re­sponse was un­equiv­o­cal, with 87% giv­ing the move their ap­proval.

For Trump, it was a savvy choice. Rather than put in some­one who would like to de­stroy the agency, he went with a pick who would set out to make it bet­ter. On Thurs­day night, I met with a group of biotech ex­ecs in the Bay Area, and the same sup­port we saw in the poll per­me­at­ed the room. The FDA is a large agency and while some el­e­ments in it have en­thu­si­as­ti­cal­ly sought to ac­cel­er­ate ap­provals, it’s al­so clear that oth­ers on the in­side could do bet­ter.

Sev­er­al in the room, though, were open­ly skep­ti­cal that Got­tlieb or any new com­mis­sion­er can achieve a quick re­sponse in a fed­er­al agency like the FDA. One sug­gest­ed a time­line of one to two years. But I’ll take that. In the scheme of things, a two-year evo­lu­tion would be bet­ter than try­ing to shove overnight changes.

Steady and in­tel­li­gent will beat rash and crash every time.

PTC’s shame­ful de­ci­sion to buy de­flaza­cort and part­ner with Marathon is a telling move

For the past two years, PTC Ther­a­peu­tics has been stub­born­ly ham­mer­ing on the FDA’s doors in search of its ac­cep­tance of ataluren for re­view as a new ther­a­py for Duchenne mus­cu­lar dy­s­tro­phy. Not un­rea­son­ably the FDA has done what it could to bar en­try, re­fus­ing to ac­cept the in­ad­e­quate ap­pli­ca­tion. This drug, af­ter all, has now failed three straight stud­ies, prov­ing over and over that it’s a dud.

So now they’re tak­ing a back door in­to the mar­ket. And they seem con­tent to make a large num­ber of en­e­mies in the Duchenne com­mu­ni­ty to cap­i­tal­ize on the gam­bit. Their deal to buy Marathon’s rights to de­flaza­cort for $140 mil­lion — plus a stream of roy­al­ties —i demon­strates their con­tempt for pa­tients.

If PTC re­al­ly cared for pa­tients, they’d nev­er mar­ket de­flaza­cort. That would let Duchenne fam­i­lies con­tin­ue to buy the ex­act same old steroid from over­seas sources for a still hefty price of about $1,000 a year — high for a gener­ic. There’s no ques­tion about qual­i­ty or safe­ty. It’s the ex­act same thing.

Any price that PTC puts on de­flaza­cort in the US high­er than $1,000 a year will be too high. They’ll on­ly join the ranks of com­pa­nies brand­ed as price-goug­ing vil­lains. That’s why their stock plunged on Thurs­day. It was a reck­less and stu­pid move by a com­pa­ny prac­ticed in deny­ing re­al­i­ty. But shame­less­ness is PTC’s hall­mark. This is a com­pa­ny that sells a drug in Eu­rope that it has shown re­peat­ed­ly can’t help pa­tients. And it’s look­ing to gain pa­tient sup­port for the same drug in the US.

Reg­u­la­tors, mean­while, seem help­less to pre­vent any of this.

Some se­ri­ous biotech mon­ey is be­ing raised for po­ten­tial­ly break­through sci­ence

Ever since the 2008 fi­nan­cial cri­sis, the biotech ven­ture scene has emerged as a very sta­ble, grow­ing field. A line­up of well known VC groups staffed by ex­pe­ri­enced in­sid­ers have been pump­ing bil­lions of dol­lars in­to star­tups.

Now, all of a sud­den, we’re start­ing to see a few new play­ers jump in­to the game. Over the course of this week I pro­filed three new de­buts — a re­mark­able num­ber for biotech VCs. There was Bio­mat­ics, which has al­ready made its mark in the past eight months, Piv­otal bioVen­ture Part­ners and a new fund from Bill Maris, the ex-Google gu­ru. The Piv­otal mon­ey is com­ing from a Chi­nese group in­ter­est­ed in mak­ing glob­al bets.

What is ex­tra­or­di­nary is that all three of these new play­ers are keen to fund star­tups pur­su­ing break­through sci­ence. And that is help­ing seed an­oth­er big year for some as­ton­ish­ing plays. I il­lus­trat­ed that this week in my sto­ry on eGe­n­e­sis, which is out to make xenograft­ing a re­al­i­ty af­ter decades as a sci­fi sideshow.

It’s a great time to be cov­er­ing this busi­ness. Ob­vi­ous­ly quite a lot of this will nev­er make it in­to med­ical prac­tice. But some ra­zor edge sci­ence is get­ting fi­nanced and we’ll all ben­e­fit from that.

Fangliang Zhang, AP Images

UP­DAT­ED: Leg­end fetch­es $424 mil­lion, emerges as biggest win­ner yet in pan­dem­ic IPO boom as shares soar

Amid a flurry of splashy pandemic IPOs, a J&J-partnered Chinese biotech has emerged with one of the largest public raises in biotech history.

Legend Biotech, the Nanjing-based CAR-T developer, has raised $424 million on NASDAQ. The biotech had originally filed for a still-hefty $350 million, based on a range of $18-$20, but managed to fetch $23 per share, allowing them to well-eclipse the massive raises from companies like Allogene, Juno, Galapagos, though they’ll still fall a few dollars short of Moderna’s record-setting $600 million raise from 2018.

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As it hap­pened: A bid­ding war for an an­tibi­ot­ic mak­er in a mar­ket that has rav­aged its peers

In a bewildering twist to the long-suffering market for antibiotics — there has actually been a bidding war for an antibiotic company: Tetraphase.

It all started back in March, when the maker of Xerava (an FDA approved therapy for complicated intra-abdominal infections) said it had received an offer from AcelRx for an all-stock deal valued at $14.4 million.

The offer was well-timed. Xerava was approved in 2018, four years after Tetraphase posted its first batch of pivotal trial data, and sales were nowhere near where they needed to be in order for the company to keep its head above water.

Drug man­u­fac­tur­ing gi­ant Lon­za taps Roche/phar­ma ‘rein­ven­tion’ vet as its new CEO

Lonza chairman Albert Baehny took his time headhunting a new CEO for the company, making it absolutely clear he wanted a Big Pharma or biotech CEO with a good long track record in the business for the top spot. In the end, he went with the gold standard, turning to Roche’s ranks to recruit Pierre-Alain Ruffieux for the job.

Ruffieux, a member of the pharma leadership team at Roche, spent close to 5 years at the company. But like a small army of manufacturing execs, he gained much of his experience at the other Big Pharma in Basel, remaining at Novartis for 12 years before expanding his horizons.

Covid-19 roundup: Ab­b­Vie jumps in­to Covid-19 an­ti­body hunt; As­traZeneca shoots for 2B dos­es of Ox­ford vac­cine — with $750M from CEPI, Gavi

Another Big Pharma is entering the Covid-19 antibody hunt.

AbbVie has announced a collaboration with the Netherlands’ Utrecht University and Erasmus Medical Center and the Chinese-Dutch biotech Harbour Biomed to develop a neutralizing antibody that can treat Covid-19. The antibody, called 47D11, was discovered by AbbVie’s three partners, and AbbVie will support early preclinical work, while preparing for later preclinical and clinical development. Researchers described the antibody in Nature Communications last month.

President Donald Trump (left) and Moncef Slaoui, head of Operation Warp Speed (Alex Brandon, AP Images)

UP­DAT­ED: White House names fi­nal­ists for Op­er­a­tion Warp Speed — with 5 ex­pect­ed names and one no­table omis­sion

A month after word first broke of the Trump Administration’s plan to rapidly accelerate the development and production of a Covid-19 vaccine, the White House has selected the five vaccine candidates they consider most likely to succeed, The New York Times reported.

Most of the names in the plan, known as Operation Warp Speed, will come as little surprise to those who have watched the last four months of vaccine developments: Moderna, which was the first vaccine to reach humans and is now the furthest along of any US effort; J&J, which has not gone into trials but received around $500 million in funding from BARDA earlier this year; the joint AstraZeneca-Oxford venture which was granted $1.2 billion from BARDA two weeks ago; Pfizer, which has been working with the mRNA biotech BioNTech; and Merck, which just entered the race and expects to put their two vaccine candidates into humans later this year.

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

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Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

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Mer­ck wins a third FDA nod for an­tibi­ot­ic; Mereo tack­les TIG­IT with $70M raise in hand

Merck — one of the last big pharma bastions in the beleaguered field of antibiotic drug development — on Friday said the FDA had signed off on using its combination drug, Recarbrio, with hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia. The drug could come handy for use in hospitalized patients who are afflicted with Covid-19, who carry a higher risk of contracting secondary bacterial infections. Once SARS-CoV-2, the virus behind Covid-19, infects the airways, it engages the immune system, giving other pathogens free rein to pillage and plunder as they please — the issue is particularly pertinent in patients on ventilators, which in any case are breeding grounds for infectious bacteria.

RA Cap­i­tal, Hill­house join $310M rush to back Ever­est's climb to com­mer­cial heights in Chi­na

Money has never been an issue for Everest Medicines. With an essentially open tab from their founders at C-Bridge Capital, the biotech has gone two and a half years racking up drug after drug, bringing in top exec after top exec, and issuing clinical update after update.

But now other investors want in — and they’re betting big.

Everest is closing its Series C at $310 million. The first $50 million comes from the Jiashan National Economic and Technological Development Zone; the remaining C-2 tranche was led by Janchor Partners, with RA Capital Management and Hillhouse Capital as co-leaders. Decheng Capital, GT Fund, Janus Henderson Investors, Rock Springs Capital, Octagon Investments all joined.