Martin Shkreli (AP Images)

The FDA OKs gener­ic Dara­prim, the Mar­tin Shkre­li ther­a­py that trig­gered an un­end­ing tem­pest over drug pric­ing

The FDA post­ed a new gener­ic drug ap­proval Fri­day af­ter­noon, and this one ar­rived with a lit­tle ex­tra rel­ish added to the cus­tom­ary pro­nounce­ment.

The agency has giv­en Cerovene the green light to sell gener­ic Dara­prim, Mar­tin Shkre­li’s treat­ment for tox­o­plas­mo­sis that det­o­nat­ed a world class brouha­ha over drug pric­ing that has taint­ed the in­dus­try. Shkre­li no soon­er ac­quired the old, once cheap drug than he in­stant­ly raised the price by more than 5,000%, some­thing that the gen­er­al pub­lic — and a big seg­ment of the bio­phar­ma in­dus­try — was pro­found­ly ap­palled by.

It’s tak­en more than 4 years for a gener­ic to come along, and giv­en the way drug pric­ing works in the U.S., there’s a good chance that re­al price re­lief may still be a long way off.

Shkre­li, al­ways ready to play the Jok­er in every phar­ma biopic, blast­ed back against the pub­lic scorn that he stirred, up un­til he was sen­tenced to 7 years for de­fraud­ing in­vestors in the hedge funds he had dri­ven in­to a brick wall be­fore he turned to biotech — fol­low­ing the path of buy­ing drugs and hik­ing the price.

Law­mak­ers put him in the spot­light, but there was no sham­ing Shkre­li. The price stayed high, and the agency even­tu­al­ly wound up us­ing the case to craft new rules that would help pre­vent Shkre­li wannabes from fol­low­ing in his foot­steps.

“Through the FDA’s Drug Com­pe­ti­tion Ac­tion Plan, we’ve worked to re­move bar­ri­ers in gener­ic drug de­vel­op­ment by not on­ly tak­ing ac­tions that im­prove the ef­fi­cien­cy of the de­vel­op­ment, re­view and ap­proval of gener­ic drugs, but al­so by clos­ing loop­holes that al­low brand-name drug com­pa­nies to ‘game’ the rules in ways that de­lay gener­ic com­pe­ti­tion that Con­gress in­tend­ed,” FDA com­mish Stephen Hahn said in a pre­pared state­ment.

Ac­cord­ing to a re­cent fed­er­al law­suit brought by the FTC and the state of New York, Shkre­li en­gaged in just such be­hav­ior, cook­ing up “an elab­o­rate an­ti­com­pet­i­tive scheme to pre­serve a mo­nop­oly” for Dara­prim. The al­leged of­fens­es ranged from keep­ing sam­ples out of reach for gener­ic drug­mak­ers to block­ing ac­cess to sales rev­enue.

There’s no im­me­di­ate word, though, on what Cerovene plans to charge for the drug. And typ­i­cal­ly, it takes sev­er­al gener­ic drug ri­vals to force the price down sharply. No mat­ter what hap­pens to Dara­prim, though, the drug pric­ing de­bate, and the fall­out that Dara­prim helped cre­ate, has be­come a fix­ture of the US po­lit­i­cal scene and the 2020 elec­tion cy­cle. And some of these can­di­dates are af­ter much big­ger fish than the im­pris­oned Shkre­li.

Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen spot­lights a pair of painful pipeline set­backs as ad­u­canum­ab show­down looms at the FDA

Biogen has flagged a pair of setbacks in the pipeline, spotlighting the final failure for a one-time top MS prospect while scrapping a gene therapy for SMA after the IND was put on hold due to toxicity.

Both failures will raise the stakes even higher on aducanumab, the Alzheimer’s drug that Biogen is betting the ranch on, determined to pursue an FDA OK despite significant skepticism they can make it with mixed results and a reliance on post hoc data mining. And the failures are being reported as Biogen was forced to cut its profit forecast for 2020 as a generic rival started to erode their big franchise drug.

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A new chap­ter in the de­cen­tral­ized clin­i­cal tri­al ap­proach

Despite the promised decentralized trial revolution, we haven’t yet moved the needle in a significant way, although we are seeing far bolder commitments to this as we continue to experience the pandemic restrictions for some time to come. The vision of grandeur is one thing, but operationalizing and execution are another and recognising that change, particularly mid-flight on studies, is worthy of thorough evaluation and consideration in order to achieve success. Here we will discuss one of the critical building blocks of a Decentralized and Remote Trial strategy: TeleConsent; more than paper under glass, it is a paradigm change and key digital enabler.

Philipp Spycher

Promis­ing bet­ter link­er tech to ADC field, Araris has 'very, very am­bi­tious' plans for the clin­ic

A couple months after raising CHF 2.5 million ($2.76 million) in initial seed funding, one-year-old Araris Biotech is topping off the round with another CHF 12.7 million ($14 million).

The Paul Scherrer Institute and ETH Zurich spinout now has CHF 15.2 million to work with, and CEO Philipp Spycher has big plans. He hopes to bring one of the company’s antibody-drug conjugates (ADC) to the clinic by late 2022 or early 2023. “It’s very, very ambitious, but we are very optimistic that we actually can make it,” he said.

Stephen Hahn, FDA commissioner (AP Images)

As FDA sets the stage for the first Covid-19 vac­cine EUAs, some big play­ers are ask­ing for a tweak of the guide­lines

Setting the stage for an extraordinary one-day meeting of the Vaccines and Related Biological Products Advisory Committee this Thursday, the FDA has cleared 2 experts of financial conflicts to help beef up the committee. And regulators went on to specify the safety, efficacy and CMC input they’re looking for on EUAs, before they move on to the full BLA approval process.

All of this has already been spelled out to the developers. But the devil is in the details, and it’s clear from the first round of posted responses that some of the top players — including J&J and Pfizer — would like some adjustments and added feedback. And on Thursday, the experts can offer their own thoughts on shaping the first OKs.

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Roche finds a home for a new, $500M man­u­fac­tur­ing lo­gis­tics hub, promis­ing 500 jobs

Roche is pouring $500 million into its Canadian headquarters in Mississauga, Ontario to set up a new hub that will coordinate logistics for its global supply chain.

Over the 5-year investment, the Swiss pharma giant expects to add 200 jobs over next year and another 300 by the end of 2023.

Introduced as a $190 million global pharmaceutical development site in 2011, the campus currently houses Roche’s Canadian commercial unit as well as product development, global procurement and pharma informatics. The new expansion will see it organize manufacturing across 13 plants and 11 sites, according to FiercePharma.

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David Hung (file photo)

Mas­ter deal­mak­er David Hung re­tools a SPAC sedan in­to a fi­nanc­ing mus­cle ve­hi­cle that leaves his can­cer start­up with $850M and a place on Wall Street

It’s only right that one of the industry’s top dealmakers just completed one of the biggest SPAC-related deals in the pipeline.

David Hung, of Medivation fame, has completed a back flip into the market, merging with EcoR1 Capital’s SPAC Panacea and landing neatly on Wall Street with an $NUVB stock ticker after filling out the blank check in his name. In addition to the $144 million held in the SPAC — provided none of the investors opt out — Hung is getting ahold of $500 million more being chipped in by a slate of institutional investors who feel that Hung could have the keys to another Medivation-style success.

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Pfizer CEO Albert Bourla (Drew Angerer/Getty Images)

Pfiz­er is on the verge of claim­ing a multi­bil­lion-dol­lar first-mover ad­van­tage with their Covid-19 vac­cine — an­a­lyst

From the beginning, Pfizer CEO Albert Bourla eschewed government funding for his Covid-19 vaccine work with BioNTech, willing to take all the $2 billion-plus risk of a lightning-fast development campaign in exchange for all the rewards that could fall its way with success. And now that the pharma giant has seized a solid lead in the race to the market, those rewards loom large.

SVB Leerink’s Geoff Porges has been running the numbers on Pfizer’s vaccine, the mRNA BNT162b2 program that the German biotech partnered on. And he sees a $3.5 billion peak in windfall revenue next year alone. Even after the pandemic is brought to heel, though, Porges sees a continuing blockbuster role for this vaccine as people around the world look to guard against a new, thoroughly endemic virus that will pose a permanent threat.

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UP­DAT­ED: CRISPR Ther­a­peu­tics gets a snap­shot of off-the-shelf CAR-T suc­cess in B-cell ma­lig­nan­cies — marred by the death of a pa­tient

Just days after scientific founder Emmanuelle Charpentier shared the Nobel prize for her work on CRISPR/Cas9, CRISPR Therapeutics $CRSP is showing off a snapshot of success in their early-stage study for an off-the-shelf CAR-T approach to CD19+ B cell malignancies — a snapshot marred by the death of a patient who had been given a high dose of the treatment.

Using their gene editing tech, researchers for CRISPR engineered cells from healthy donors into an attack vehicle aimed at cancer, something that has been achieved with great success using patients’ own cells — the autologous approach. But autologous CAR-T is hampered by the more complex vein-to-vein requirement that delays treatment, and now CRISPR Therapeutics along with other players like Allogene are determined to replace the pioneers with CAR-T 2.0.

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RBC's Bri­an Abra­hams holds a mock ad­comm on Bio­gen's iffy ad­u­canum­ab da­ta — and most of these ex­perts don't see a path to an ap­proval

As catalysts go, few loom larger than the aducanumab adcomm slated for Nov. 6.

With its big franchise under assault, Biogen is betting the ranch that its mixed late-stage Alzheimer’s data can squeak past the experts and regulators and get onto the market. And the topic — after a decade of Alzheimer’s R&D disasters in what still represents the El Dorado of drug markets — remains in the center ring of discussions around late-stage pipeline prospects.

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