George Painter in his Atlanta Lab (DRIVE)
April 13, 2020 11:16 AM EDTUpdated 01:31 PM
R&D
Coronavirus
In Focus

Cre­ative or un­eth­i­cal? Re­searchers’ Covid-19 Go­FundMe cam­paign spurs crit­ics

Jason Mast

Associate Editor

It had been a business professor’s idea, back before Christmas, when the most infectious threat facing the US was a potentially harsh flu season. DRIVE Innovations, a non-profit biotech founded by a pioneering HIV researcher at Emory University, was looking for new ways of raising money and drawing press. Charles Goetz, who studied entrepreneurship, suggested GoFundMe.

What appeared online three months later had the smoothness of a VC pitch and the commanding lilt of a call to arms. That was the week the coronavirus moved from far-flung threat to looming menace for many Americans, when Vice President Pence was named to head the White House taskforce, the stock market suffered its worst fall since 2008 and the first cases of unknown origin appeared on the West Coast — a sign the virus was spreading within the US. GoFundMe put DRIVE’s campaign as the “Urgent Cause” at the center of its homepage. They promised a tantalizing antidote.

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Michel Vounatsos, Biogen CEO (via YouTube)
October 21, 2020 08:11 AM EDTUpdated 10:22 AM
R&D

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

John Carroll

Editor & Founder

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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SPONSORED
October 19, 2020 06:00 AM EDT

A new chap­ter in the de­cen­tral­ized clin­i­cal tri­al ap­proach

Alison Holland

Head of Decentralized and Remote trials

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)
October 20, 2020 10:21 AM EDTUpdated 10:48 AM
Regulatory
Coronavirus

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

John Carroll

Editor & Founder

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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Pfizer CEO Albert Bourla (Drew Angerer/Getty Images)
October 21, 2020 08:53 AM EDTUpdated 09:56 AM
Coronavirus

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

John Carroll

Editor & Founder

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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October 21, 2020 07:00 AM EDTUpdated 09:51 AM
R&D
Cell/Gene Tx

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

John Carroll

Editor & Founder

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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October 19, 2020 07:28 AM EDTUpdated 08:21 AM
R&D

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

John Carroll

Editor & Founder

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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October 21, 2020 01:35 PM EDTUpdated 02:30 PM
Coronavirus

UP­DAT­ED: Brazil­ian vol­un­teer in As­traZeneca Covid-19 vac­cine tri­al has died in the place­bo arm — re­ports

Max Gelman

Associate Editor

A volunteer in AstraZeneca’s Covid-19 vaccine trial in Brazil has died, Brazilian health authorities said Wednesday, triggering fresh alarms over the future of the Oxford program. But later reports noted that the death was in the placebo group and AstraZeneca issued word that there were no concerns about continuing the study.

The Brazilian health agency Anvisa said it had received data from an investigation into the issue, per a Reuters report. The report was then updated citing a Brazilian newspaper with unnamed sources saying that the volunteer was in the placebo arm of the trial.

David Hung (file photo)
October 21, 2020 01:16 PM EDT
People
Deals

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

John Carroll

Editor & Founder

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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October 21, 2020 11:03 AM EDT
R&D

Years af­ter a ma­jor tri­al set­back, No­var­tis switch­es gears with SMA drug. This time they're try­ing it for Hunt­ing­ton's

Nicole DeFeudis

Associate Editor

Four years after a Phase I/II setback in spinal muscular atrophy (SMA), Novartis is hoping its drug branaplam will find more success in a new neurological indication: Huntington’s disease.

The decision was announced a year after the head of research, Jay Bradner, said he did not see a “big opportunity” in SMA, according to Reuters. Novartis says it has preclinical data showing that branaplam reduces levels of mutant huntingtin protein, and SMA data showing patients on the drug had reductions in huntingtin mRNA. The FDA gave branaplam their orphan drug designation, and Novartis plans to move forth with a Phase IIb trial next year.

Cre­ative or un­eth­i­cal? Re­searchers’ Covid-19 Go­FundMe cam­paign spurs crit­ics

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

A new chap­ter in the de­cen­tral­ized clin­i­cal tri­al ap­proach

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

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

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

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

UP­DAT­ED: Brazil­ian vol­un­teer in As­traZeneca Covid-19 vac­cine tri­al has died in the place­bo arm — re­ports

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

Years af­ter a ma­jor tri­al set­back, No­var­tis switch­es gears with SMA drug. This time they're try­ing it for Hunt­ing­ton's