Leonard Schleifer, Regeneron CEO (AP Images)

What hap­pens when the N-of-1 is the pres­i­dent of the US? Re­gen­eron is about to find out

Pres­i­dent Trump is tak­ing a very per­son­al in­ter­est in an­oth­er ex­per­i­men­tal drug for Covid-19.

Fri­day af­ter­noon, af­ter he had test­ed pos­i­tive for coro­n­avirus, the White House put out a state­ment say­ing that Trump had tak­en an 8 gram dose of Re­gen­eron’s an­ti­body cock­tail — the high dose that ap­peared to do bet­ter than the low dose in terms of bat­ting down vi­ral loads.

Right af­ter word ar­rived that Trump had tak­en the ex­per­i­men­tal ther­a­py, he was trans­ferred to Wal­ter Reed — news that im­me­di­ate­ly height­ened fears about his con­di­tion. The Wash­ing­ton Post not­ed that Trump and his team de­cid­ed to make the move while he could still walk.

The pres­i­dent’s physi­cians didn’t stop with the cock­tail. Late Fri­day night, the pres­i­dent’s spokesper­son stat­ed that the doc­tors start­ed a course of remde­sivir, the Gilead drug that won the first emer­gency use au­tho­riza­tion from the FDA on mod­est­ly pos­i­tive tri­al re­sults.

Ear­li­er in the week Re­gen­eron — led by CEO Len Schleifer and chief sci­en­tist George Yan­copou­los — had stirred a mixed re­ac­tion to its first cut of the da­ta on their cock­tail, es­sen­tial­ly pro­vid­ing proof-of-con­cept da­ta for the drug with­out sol­id ev­i­dence of ef­fi­ca­cy in eas­ing the path of the pa­tients with ini­tial­ly mild cas­es of Covid-19.

While Re­gen­eron of­fered da­ta demon­strat­ing that the drug could re­duce the need for med­ical vis­its among pa­tients with a low im­mune re­sponse, the num­bers in­volved were far too low to of­fer any clear pic­ture reg­u­la­tors nor­mal­ly need for an OK. The use of the drug may in­di­cate that Trump is among the most vul­ner­a­ble group.

Trump, though, has a his­to­ry of tout­ing drugs based on lit­tle to no ev­i­dence they work. He took hy­droxy long af­ter the da­ta point­ed to its in­ef­fec­tive­ness. And he pushed the FDA to pro­vide an emer­gency OK for con­va­les­cent plas­ma on thin ev­i­dence that it could work to fight the virus.

Trump’s re­sponse to the Re­gen­eron cock­tail, re­al or sim­ply self-di­ag­nosed, could have enor­mous con­se­quences for the biotech — though adding remde­sivir could make it near im­pos­si­ble to tease out a re­sponse now. The star an­ti­body drug mak­er is seek­ing an emer­gency OK and may soon have the pres­i­dent in their cor­ner if he pulls through quick­ly.

Any se­ri­ous set­backs from here, though, could threat­en to turn Trump in­to an in­flu­en­tial op­po­nent.

So far right now, the pres­i­dent is a very big N of 1. And the ini­tial mar­ket re­sponse is all pos­i­tive. Re­gen­eron’s stock surged 5% af­ter hours Fri­day — not bad when you start with a mar­ket cap of $60 bil­lion.

Trump him­self of­fered a late-night thumbs up:

Bio­phar­ma com­pa­nies don’t typ­i­cal­ly like get­ting com­pas­sion­ate use re­quests for their drugs, see­ing lit­tle up­side when they work and a big down­side when they don’t. But as Schleifer told Katie Thomas at the New York Times: “When it’s the pres­i­dent of the Unit­ed States, of course, that gets — ob­vi­ous­ly — gets our at­ten­tion.”

Al­so of note: Trump had a choice in ex­per­i­men­tal an­ti­bod­ies. Eli Lil­ly tout­ed its ap­proach be­fore Re­gen­eron ar­rived on the scene, and their ther­a­py — which dis­ap­point­ed a num­ber of an­a­lysts on the first snap­shot of da­ta — didn’t make the cut. That can’t sit well at the phar­ma gi­ant.

In ad­di­tion to the cock­tail, Trump al­so is tak­ing zinc, vi­t­a­min D, famo­ti­dine, mela­tonin and a dai­ly as­pirin.

For a look at all End­points News coro­n­avirus sto­ries, check out our spe­cial news chan­nel.

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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Jean-Pierre Sommadossi, Atea president and CEO (file photo)

Roche wades deep­er in­to Covid-19 fight, ink­ing an­tivi­ral pact with $350M cash fol­low­ing Re­gen­eron deal

Roche is making its first bet on an antiviral against Covid-19 in style, shelling out $350 million in cash to grab ex-US rights.

The drug comes from Atea Pharmaceuticals, the 7-year-old biotech created by Pharmasset co-founder Jean-Pierre Sommadossi, which essentially rebranded itself as a Covid-19 fighter in May when it closed a whopping $215 million venture round. Over a dozen investors bought in, including marquee names like Bain Capital and RA Capital.

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.

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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Peter Kolchinsky and Raj Shah (file photo)

Pe­ter Kolchin­sky and Raj Shah's RA Cap­i­tal has $461M more to play with, af­ter 'rapid' in­vest­ment in the last 15 months

Just over 15 months after launching its first venture fund, RA Capital Management is ready for more. And this time the firm is bringing an even bigger load of cash to the table.

Announcing the close of its Nexus II fund on Wednesday, RA said it raised $461 million for investments in private companies across the biotech industry. The first venture fund, which raised $300 million, has churned through roughly 80% of its capital already, a pace that managing partner Raj Shah called unusually quick.

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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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.