British bil­lion­aire Jim Mel­lon and high-pro­file part­ners roll the dice on an an­ti-ag­ing up­start

When British bil­lion­aire Jim Mel­lon wants to map out an in­vest­ment strat­e­gy, he likes to write a book first. Out of that process came his most re­cent work — Ju­ve­nes­cence: In­vest­ing in the Age of Longevi­ty. Now he and some close as­so­ciates with some of the best con­nec­tions in biotech are us­ing the book as in­spi­ra­tion to launch a new com­pa­ny — al­so named Ju­ve­nes­cence — with plans to make a big splash in an­ti-ag­ing re­search.

Jim Mel­lon

And they’re plant­i­ng the first seeds now with a new joint ven­ture that will start to lay the foun­da­tion for the pipeline with ar­ti­fi­cial in­tel­li­gence tech­nol­o­gy.

“We are at an in­flec­tion point for the treat­ment of ag­ing,” says Greg Bai­ley, who likes to high­light some of the new cel­lu­lar path­ways that are point­ing to new ther­a­pies that can counter the ef­fects of ag­ing.

“I think this is go­ing to be the biggest deal I’ve ever done,” Bai­ley tells me in a phone in­ter­view, as his plane was prepar­ing for a take­off.  “It will need repet­i­tive fi­nanc­ing.  Five to $600 mil­lion was raised for Medi­va­tion. As we hit in­flec­tion points, we will need to raise a dra­mat­ic amount of mon­ey.”

Gre­go­ry Bai­ley

Bai­ley, the CEO of Ju­ve­nes­cence, was one of the ear­ly back­ers of Medi­va­tion, where he was a board di­rec­tor for 7 years — be­fore Pfiz­er stepped in to buy the biotech for $14 bil­lion. More re­cent­ly, he helped po­si­tion Bio­haven for an IPO, as­sem­bling a pipeline that in­cludes a late-stage drug in-li­censed from Bris­tol-My­ers Squibb be­fore rais­ing $190 mil­lion a few months ago in their maid­en of­fer­ing. The chair­man at Bio­haven is his long­time col­league De­clan Doogan, a for­mer top Pfiz­er re­search ex­ec who is com­ing in as a prin­ci­pal to the new ven­ture along­side Mel­lon and Bai­ley.

The pri­ma­ry game plan at Ju­ve­nes­cence, ex­plains Bai­ley, is to come up with var­i­ous op­er­a­tions en­gaged in de­vel­op­ing new an­ti-ag­ing drugs. Ju­ve­nes­cence AI is a joint ven­ture they’ve just set up with Alex Zha­voronkov, who runs In­sil­i­co Med­i­cine, based in Bal­ti­more. Mel­lon met Zha­voronkov while he was re­search­ing his book, says Bai­ley, and be­lieves that the tech the sci­en­tist de­vel­oped can il­lu­mi­nate new pro­grams with a bet­ter chance of suc­cess.

“They are go­ing to take up to 5 mol­e­cules from us every year for de­vel­op­ment,” says Zha­voronkov, an en­thu­si­as­tic ad­vo­cate of AI in drug re­search who’s al­so been work­ing on some al­liances with Big Phar­ma play­ers. The group has in­vest­ed about $7 mil­lion in the tech­nol­o­gy so far, he says, get­ting the JV set up. More will fol­low.

Alex Zha­voronkov

“We can gen­er­ate mol­e­cules with spe­cif­ic mol­e­c­u­lar prop­er­ties,” adds Zha­voronkov, who al­so has a spe­cial fo­cus on an­ti-ag­ing re­search.

“The mas­sive lib­er­a­tion of new da­ta needs to trans­form in­to knowl­edge,” says Doogan. “AI is the buzz word; can we take in­cre­men­tal steps, in an it­er­a­tive learn­ing process, cap­ture all knowl­edge?”

Ju­ve­nes­cence Bio will be charged with build­ing the pipeline, says Bai­ley, in part with the mol­e­cules that will be iden­ti­fied through the AI ven­ture. And Doogan will play a lead role in or­ga­niz­ing the team now, much as he was cred­it­ed with at Bio­haven.

Aside from the cel­lu­lar path­ways that have at­tract­ed their at­ten­tion, the biotech will look to ef­fect change in the mi­to­chon­dria, the cell’s pow­er­house, as well as clean up senes­cent cells that ac­cu­mu­late as the body grows old­er. And Bai­ley ex­pects he’ll be work­ing some Bio­haven-like deals to de­vel­op an ad­vanced pipeline at a rapid pace.

The prin­ci­pals chipped in the seed mil­lions for the com­pa­ny and in­vest­ed in the JV with Zha­voronkov. Bai­ley says you can ex­pect to see $20 mil­lion to $50 mil­lion more from a friends-and-fam­i­ly raise be­fore the end of the year. And it’s ex­pect­ed to grow from there.

De­clan Doogan

Doogan plans to re­cruit var­i­ous team lead­ers, in­di­vid­u­als who will be in charge of spe­cif­ic projects with 10 or few­er peo­ple on the crew. Like any biotech, he notes, they plan to re­ly on a se­mi-vir­tu­al struc­ture, with a ma­jor amount of out­sourc­ing in place of staff.

The biotech won’t just be a biotech, says Doogan. It will cov­er “mul­ti­ple do­mains: di­ag­nos­tics, con­sumer, con­ven­tion­al drug de­vel­op­ment — broad ideas to en­gage the con­sumer.”

The key, he says, is fo­cus­ing on not just a longer life, but a bet­ter one.

“Not just longer, but bet­ter longer,” is the way Doogan puts it. “Healthy ag­ing is the ob­jec­tive here.”

That leaves a lot of room.

“There are 52 ways to drop blood pres­sure, but we’ve done noth­ing for os­teoarthri­tis,” says Doogan by way of ex­am­ple.

“We have to be re­al­ly clever,” says Bai­ley. Drugs like No­var­tis’ mTOR in­hibitor everolimus, which con­trols cell growth and pro­lif­er­a­tion, can be a mod­el. Os­teoarthri­tis, a dis­ease as­so­ci­at­ed with ag­ing, can be the kind of dis­ease fo­cus that can dri­ve ear­ly work.

It’s ear­ly days yet for an­ti-ag­ing drug re­search. A few stal­warts like Bob Nelsen at Arch have backed the first few star­tups in the field. But Mel­lon and his col­leagues say now’s the time.

The longevi­ty in­dus­try, Mel­lon said re­cent­ly, is des­tined to grow “in­to the world’s largest in­dus­try.”

And he wants in.

2019 Trin­i­ty Drug In­dex Eval­u­ates Ac­tu­al Com­mer­cial Per­for­mance of Nov­el Drugs Ap­proved in 2016

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

How to cap­i­talise on a lean launch

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

Aymeric Le Chatelier, Ipsen

A $1B-plus drug stum­bles in­to an­oth­er big PhI­II set­back -- this time flunk­ing fu­til­i­ty test -- as FDA hold re­mains in ef­fect for Ipsen

David Meek

At the time Ipsen stepped up last year with more than a billion dollars in cash to buy Clementia and a late-stage program for a rare bone disease that afflicts children, then CEO David Meek was confident that he had put the French biotech on a short path to a mid-2020 launch.

Instead of prepping a launch, though, the company was hit with a hold on the FDA’s concerns that a therapy designed to prevent overgrowth of bone for cases of fibrodysplasia ossificans progressiva might actually stunt children’s growth. So they ordered a halt to any treatments for kids 14 and under. Meek left soon after to run a startup in Boston. And today the Paris-based biotech is grappling with the independent monitoring committee’s decision that their Phase III had failed a futility test.

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Roche's check­point play­er Tecen­triq flops in an­oth­er blad­der can­cer sub­set

Just weeks after Merck’s star checkpoint inhibitor Keytruda secured FDA approval for a subset of bladder cancer patients, Swiss competitor Roche’s Tecentriq has failed in a pivotal bladder cancer study.

The 809-patient trial — IMvigor010 — tested the PD-L1 drug in patients with muscle-invasive urothelial cancer (MIUC) who had undergone surgery, and were at high risk for recurrence.

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UP­DAT­ED: Eli Lil­ly’s $1.6B can­cer drug failed to spark even the slight­est pos­i­tive gain for pa­tients in its 1st PhI­II

Eli Lilly had high hopes for its pegylated IL-10 drug pegilodecakin when it bought Armo last year for $1.6 billion in cash. But after reporting a few months ago that it had failed a Phase III in pancreatic cancer, without the data, its likely value has plunged. And now we’re getting some exact data that underscore just how little positive effect it had.

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UP­DAT­ED: FDA’s golodirsen CRL: Sarep­ta’s Duchenne drugs are dan­ger­ous to pa­tients, of­fer­ing on­ly a small ben­e­fit. And where's that con­fir­ma­to­ry tri­al?

Back last summer, Sarepta CEO Doug Ingram told Duchenne MD families and investors that the FDA’s shock rejection of their second Duchenne MD drug golodirsen was due to some concerns regulators raised about the risk of infection and the possibility of kidney toxicity. But when pressed to release the letter for all to see, he declined, according to a report from BioPharmaDive, saying that kind of move “might not look like we’re being as respectful as we’d like to be.”

He went on to assure everyone that he hadn’t misrepresented the CRL.

But Ingram’s public remarks didn’t include everything in the letter, which — following the FDA’s surprise about-face and unexplained approval — has now been posted on the FDA’s website and broadly circulated on Twitter early Wednesday.

The CRL raises plenty of fresh questions about why the FDA abruptly decided to reverse itself and hand out an OK for a drug a senior regulator at the FDA believed — 5 months ago, when he wrote the letter — is dangerous to patients. It also puts the spotlight back on Sarepta $SRPT, which failed to launch a confirmatory study of eteplirsen, which was only approved after a heated internal controversy at the FDA. Ellis Unger, director of CDER’s Office of Drug Evaluation I, notes that study could have clarified quite a lot about the benefit and risks associated with their drugs — which can cost as much as a million dollars per patient per year, depending on weight.

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Gilead dusts off a failed Ebo­la drug as coro­n­avirus spreads; Ex­elix­is boasts pos­i­tive Ph I/II da­ta

→ Less than a year ago Gilead’s antiviral remdesivir failed to make the cut as investigators considered a raft of potential drugs that could be used against an Ebola outbreak. But it may gain a new mission with the outbreak of the coronavirus in China, which is popping up now around the world.

Gilead put out a statement saying that they’re now in discussions with health officials in the US and China about testing their NUC against the virus. It’s the latest in a growing lineup of biopharma companies that are marshaling R&D forces to see if they can come up with a vaccine or therapy to blunt the spread of the virus, which has now sickened hundreds, killed at least 17 people and led the Chinese government to start quarantining cities.

Alex Karnal (Deerfield)

Deer­field vaults to the top of cell and gene ther­a­py CD­MO game with $1.1B fa­cil­i­ty at Philadel­phi­a's newest bio­phar­ma hub

Back at the beginning of 2015, Deerfield Management co-led a $10 million Series C for a private gene therapy startup, reshaping the company and bringing in new leaders to pave way for an IPO just a year later.

Fast forward four more years and the startup, AveXis, is now a subsidiary of Novartis marketing the second-ever gene therapy to be approved in the US.

For its part, Deerfield has also grown more comfortable and ambitious about the nascent field. And the investment firm is now putting down its biggest bet yet: a $1.1 billion contract development and manufacturing facility to produce everything one needs for cell and gene therapy — faster and better than how it’s currently done.

Tri­fec­ta of sick­le cell dis­ease ther­a­pies ex­tend life ex­pectan­cy, but are not cost-ef­fec­tive — ICER

Different therapeutic traits brandished by the three approved therapies for sickle cell disease all extend life expectancy, but their impact on quality of life is uncertain and their long-term cost-effectiveness is not up to scratch according to the thresholds considered reasonable by ICER, the non-profit concluded in a draft guidance report on Thursday.

Sickle cell disease (SCD), which encompasses a group of inherited red blood cell disorders that typically afflict those of African ancestry, impacts hemoglobin — and is characterized by episodes of searing pain as well as organ damage.