Ned Sharp­less weighs in on gene ther­a­py pric­ing de­bate, sug­gest­ing 'the mes­sag­ing got lost'

PHILADEL­PHIA — You know the drug pric­ing de­bate has reached a new height when the act­ing FDA com­mis­sion­er starts to chime in on a pub­lic fo­rum.

Ned Sharp­less NIH

In re­sponse to a ques­tion on gene ther­a­pies posed dur­ing a fire­side chat at the an­nu­al BIO con­ven­tion, Ned Sharp­less ac­knowl­edged the im­por­tance of dis­cussing cost — though it doesn’t fall un­der the FDA’s purview — but said he’s “a lit­tle dis­ap­point­ed” in the cov­er­age of the re­cent ap­proval of No­var­tis’ spinal mus­cu­lar at­ro­phy treat­ment, Zol­gens­ma. The treat­ment was priced at $2.12 mil­lion spread out over a 5-year in­stall­ment.

“This is a com­plete­ly nov­el, al­most mag­i­cal mir­a­cle that ends a dev­as­tat­ing dis­ease for lots of lit­tle kids and the thing you care the most about is the price? I mean, re­al­ly? If you’re so cyn­i­cal you can’t see how won­der­ful and great that is […] you need to re-wear your hap­py hat,” he said.

Then he hint­ed at how the price of these “shock­ing­ly ex­pen­sive” ther­a­pies might even­tu­al­ly come down as man­u­fac­tur­ers op­ti­mize their process­es.

“You have to re­mem­ber when we first made peni­cillin, it was so ex­pen­sive to make,” he said. “They would fil­ter the urine of the peo­ple who’d take it and crys­tal­lize it. We got bet­ter at mak­ing peni­cillin even­tu­al­ly.”

The top­ic was brought up again in the Q&A ses­sion when a pa­tient ad­vo­cate in the au­di­ence — whose 8-year-old son has re­ceived $3 mil­lion worth of oth­er treat­ments for SMA — asked how the “mes­sag­ing got lost.”

Here’s Sharp­less again:

Thank you so much for mak­ing that point. Be­cause I think I’m with you. I think the mes­sag­ing got lost. I was sort of imag­in­ing, it’s like […] at that wed­ding that Je­sus was at where he turned wa­ter in­to wine, there was prob­a­bly some­body who said hey, you know I want­ed Chardon­nay! This is red wine! […] But there is a le­git­i­mate is­sue here which is you know we have 40, 50, I don’t know how many INDs of these nov­el ther­a­pies in CBER and how are we go­ing to sup­port all of these things that get de­vel­oped for these ul­tra rare in­di­ca­tions and the so­ci­ety is go­ing to fig­ure that out.

The FDA is do­ing its part by co­or­di­nat­ing with the CMS on spe­cif­ic ar­eas such as an­tibac­te­ri­als and CAR-T, in­te­grat­ing their work to the ex­tent pos­si­ble, he men­tioned ear­li­er in a di­a­logue that ranged from opi­oids and stem cell clin­ics to Alzheimer’s and de­pres­sion.


Im­age: Ned Sharp­less. Tom Williams CQ Roll Call AP

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.

Bris­tol My­ers is clean­ing up the post-Cel­gene merg­er pipeline, and they’re sweep­ing out an ex­per­i­men­tal check­point in the process

Back during the lead up to the $74 billion buyout of Celgene, the big biotech’s leadership did a little housecleaning with a major pact it had forged with Jounce. Out went the $2.6 billion deal and a collaboration on ICOS and PD-1.

Celgene, though, also added a $530 million deal — $50 million up front — to get the worldwide rights to JTX-8064, a drug that targets the LILRB2 receptor on macrophages.

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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.

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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David Meline (file photo)

Mod­er­na’s new CFO took a cut in salary to jump to the mR­NA rev­o­lu­tion­ary. But then there’s the rest of the com­pen­sa­tion pack­age

David Meline took a little off the top of his salary when he jumped from the CFO post at giant Amgen to become the numbers czar at the upstart vaccines revolutionary Moderna. But the SEC filing that goes with a major hire also illustrates how it puts him in line for a fortune — provided the biotech player makes good as a promising game changer.

To be sure, there’s nothing wrong with the base salary: $600,000. Or the up-to 50% annual cash bonus — an industry standard — that comes with it. True, the 62-year-old earned $999,000 at Amgen in 2019, but it’s the stock options that really count in the current market bliss for all things biopharma. And there Meline did well.

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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.

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.