BioReg­num: The mir­a­cle drug sto­ry that just won’t die

Bioreg­num
The view from End­points

Eight years ago resver­a­trol was the mir­a­cle drug of the hour. Linked to pre­clin­i­cal ev­i­dence that it could ex­pand the life spans of mice, Glax­o­SmithK­line paid a whop­ping $720 mil­lion in cold, hard cash to get its hands on Sir­tris, the biotech start­up that promised to de­liv­er an an­ti-ag­ing mir­a­cle by turn­ing an in­gre­di­ent in red wine in­to a med­ical re­al­i­ty.

Queue the jokes about drink­ing more red wine for bet­ter health. This is the stuff head­lines are made of, and I was hap­py to par­tic­i­pate.

GSK, which just demon­strat­ed its ap­petite for dis­cov­ery and ear­ly-stage de­vel­op­ment with its elec­tro­ceu­ti­cal part­ner­ship with Ver­i­ly, didn’t do very well with its new sub­sidiary. Progress was slow. Telling­ly, aca­d­e­mics be­gan to de­bate over whether the an­i­mal stud­ies GSK bought in­to could ever be repli­cat­ed. And the phar­ma gi­ant even­tu­al­ly de­cid­ed to dis­band the Sir­tris team in Cam­bridge, MA and ab­sorb the work in its Philly op­er­a­tions.

That’s the last I heard about it from GSK.

Resver­a­trol, though, is mak­ing a come­back as a mir­a­cle drug.

I read in the au­gust pages of the Wall Street Jour­nal Mon­day that sci­en­tists the world over “ap­pear to be get­ting clos­er to har­ness­ing one of red wine’s most elu­sive health-giv­ing in­gre­di­ents and putting it in­to a pill.”

Shaz­am.

To back that up, the Jour­nal cites a tiny hu­man study last fall that tracked a bio­mark­er for Alzheimer’s, an aw­ful dis­ease that is still poor­ly un­der­stood and re­mains as one of the sin­gle great­est chal­lenges in bio­phar­ma re­search to­day, along with new aca­d­e­m­ic re­search at Uni­ver­si­ty of New South Wales and a project at a small biotech com­pa­ny in Flori­da called Jupiter Or­phan Ther­a­peu­tics.

Out of such small serv­ings a great feast is made.

One of the chal­lenges at resver­a­trol, aside from ever prov­ing that it could do what some sci­en­tists be­lieved it could, was find­ing a safe con­cen­tra­tion of the drug. As an in­gre­di­ent in wine, you’d have to drink an ocean of it in or­der to get any kind of ther­a­peu­tic ef­fect.

Per­haps one day sci­en­tists will fig­ure out how to make resver­a­trol in­to a drug. It’s al­ways en­ter­tain­ing to track these dis­cov­ery projects. But it’s al­so im­por­tant to dis­tin­guish the kind of ex­tra­or­di­nary risks and epic time­lines that are in­volved — es­pe­cial­ly when a mir­a­cle drug re­turns to main­stream me­dia, dressed up once again as a pipeline con­tender to be reck­oned with. — John Car­roll.

The DCT-OS: A Tech­nol­o­gy-first Op­er­at­ing Sys­tem - En­abling Clin­i­cal Tri­als

As technology-enabled clinical research becomes the new normal, an integrated decentralized clinical trial operating system can ensure quality, deliver consistency and improve the patient experience.

The increasing availability of COVID-19 vaccines has many of us looking forward to a time when everyday things return to a state of normal. Schools and teachers are returning to classrooms, offices and small businesses are reopening, and there’s a palpable sense of optimism that the often-awkward adjustments we’ve all made personally and professionally in the last year are behind us, never to return. In the world of clinical research, however, some pandemic-necessitated adjustments are proving to be more than emergency stopgap measures to ensure trial continuity — and numerous decentralized clinical trial (DCT) tools and methodologies employed within the last year are likely here to stay as part of biopharma’s new normal.

Onno van de Stolpe, Galapagos CEO (Thierry Roge/Belga Mag/AFP via Getty Images)

Gala­pa­gos chops in­to their pipeline, drop­ping core fields and re­or­ga­niz­ing R&D as the BD team hunts for some­thing 'trans­for­ma­tive'

Just 5 months after Gilead gutted its rich partnership with Galapagos following a bitter setback at the FDA, the Belgian biotech is hunkering down and chopping the pipeline in an effort to conserve cash while their BD team pursues a mission to find a “transformative” deal for the company.

The filgotinib disaster didn’t warrant a mention as Galapagos laid out its Darwinian restructuring plans. Forced to make choices, the company is ditching its IPF molecule ’1205, while moving ahead with a Phase II IPF study for its chitinase inhibitor ’4617.

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Stéphane Bancel, Getty

Mod­er­na CEO brush­es off US sup­port for IP waiv­er, eyes more than $19B in Covid-19 vac­cine sales in 2021

Moderna is definitively more concerned with keeping pace with Pfizer in the race to vaccinate the world against Covid-19 than it is with Wednesday’s decision from the Biden administration to back an intellectual property waiver that aims to increase vaccine supplies worldwide.

In its first quarter earnings call on Thursday, Moderna CEO Stéphane Bancel shrugged off any suggestion that the newly US-backed intellectual property waiver would impact his company’s vaccine or bottom line. Still, the company’s stock price fell by about 9% in early morning trading.

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'Chang­ing the whole game of drug dis­cov­ery': Leg­endary R&D vet Roger Perl­mut­ter leaps back in­to work as a biotech CEO

Roger Perlmutter needs no introduction to anyone remotely involved in biopharma. As the R&D chief first at Amgen and then Merck, he’s built a stellar reputation and a prolific career steering new drugs toward the market for everything from cancer to infectious diseases.

But for years, he’s also held a less known title: science partner at The Column Group, where he’s regularly consulted about the various ideas the VCs had for new startups.

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Brent Saunders (Richard Drew, AP Images)

OcuWho? Star deal­mak­er turned aes­thet­ics czar Brent Saun­ders flips back in­to biotech. But who’s he team­ing up with now?

Brent Saunders went on a tear of headline-blazing deals building Allergan, merging and rearranging a variety of big companies into one before an M&A pact with Pfizer blew up and sent him on a bout of biotech drug deals. That didn’t work so well, so under pressure, he got his buyout at AbbVie — which needed a big franchise like Botox. And it was no big surprise to see him riding the SPAC wave into a recent $1 billion-plus deal that left him in the executive chairman’s seat at an aesthetics outfit — now redubbed The Beauty Health Company — holding a big chunk of the equity.

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Ngozi Okonjo-Iweala, Director general of WTO (AP Photo/Keystone/Alessandro Della Bella)

Opin­ion: Waiv­ing Covid-19 vac­cine IP could save lives, but where is the man­u­fac­tur­ing ca­pac­i­ty?

Droves of House Democrats in Washington and members of the European Parliament have now glommed onto a major push by India and South Africa at the WTO to abolish all IP around Covid-19 vaccines.

At first blush, waiving this IP sounds like an easy win: More Covid-19 vaccines made locally for more people means more lives saved. Simple enough, especially as low-income countries have received just a tiny fraction of the world’s vaccine allotment so far.

Ad­comm splits slight­ly in fa­vor of FDA ap­prov­ing Chemo­Cen­tryx’s rare dis­ease drug

The FDA’s Arthritis Advisory Committee on Thursday voted 10 for and 8 against the approval of ChemoCentryx’s $CCXI investigational drug avacopan as a treatment for adults with a rare and serious disease known as anti-neutrophil cytoplasmic autoantibody (ANCA)-vasculitis.

The vote on whether the FDA should approve the drug was preceded by a split vote of 9 to 9 on whether the efficacy data support approval, and 10 to 8 that the safety profile of avacopan is adequate enough to support approval.

Drug pric­ing watch­dog joins the cho­rus of crit­ics on Bio­gen's ad­u­canum­ab: What about charg­ing $2,560 per year?

As if Biogen’s aducanumab isn’t controversial enough, the researchers at drug pricing watchdog ICER have drawn up the contours of a new debate: If the therapy does get approved for Alzheimer’s by June, what price should it command?

Their answer: At most $8,290 per year — and perhaps as little as $2,560.

Even at the top of the range, the proposed price is a fraction of the $50,000 that Wall Street has reportedly come to expect (although RBC analyst Brian Abrahams puts the consensus figure at $11.5K). With critics, including experts on the FDA’s advisory committee, making their fierce opposition to aducanumab’s approval loud and clear, the pricing pressure adds one extra wrinkle Biogen CEO Michel Vounatsos doesn’t need as he orders full-steam preparation for a launch.

Biden ad­min­is­tra­tion backs a po­lar­iz­ing pro­pos­al to waive IP for all Covid-19 vac­cines

In a surprise U-turn, the Biden administration said Wednesday that it will support a proposal at the World Trade Organization to temporarily waive intellectual property protections on Covid-19 vaccines.

The proposal, backed by South Africa and India at the WTO, seeks to help developing countries with limited vaccine supplies. The US and Europe historically opposed the proposal, saying IP should be protected because it incentivizes new drug and vaccine development.

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