This is it? GSK rolls out a buzzy new R&D plan long on as­pi­ra­tions and short on per­for­mance goals

Glax­o­SmithK­line set out to kick­start some en­thu­si­asm for its pipeline this morn­ing. But CEO Em­ma Walm­s­ley and her new R&D chief Hal Bar­ron clear­ly have a lot more work cut out for them on that crit­i­cal point.

Its quar­ter­ly re­port high­lights a strat­e­gy in R&D that will be heav­i­ly fo­cused on the im­mune sys­tem and hu­man ge­net­ics — two well es­tab­lished fields that long ago at­tract­ed the at­ten­tion of just about every­one work­ing in drug de­vel­op­ment these days. And they signed up 23andMe — pay­ing $300 mil­lion to buy in and gain ex­clu­sive ac­cess to its da­ta base — to help ex­plore dis­ease rel­e­vant genes, herald­ing a “ma­jor ad­vance” while adding to a slate of al­liances on ge­net­ics the Lon­don-based gi­ant has al­ready com­plet­ed.

The phar­ma gi­ant says it will fo­cus more on CRISPR tech, with an em­pha­sis on ma­chine learn­ing and com­pu­ta­tion­al de­sign — a cou­ple of buzzy fea­tures that have al­so been mak­ing the rounds late­ly.

There’s a com­ment on cul­ture:

GSK al­so in­tends to pro­mote a cul­ture of in­creased ac­count­abil­i­ty and smart risk-tak­ing. This will in­clude re­defin­ing suc­cess and fos­ter­ing a cul­ture of truth-seek­ing ver­sus pro­gres­sion- seek­ing, and op­ti­mised port­fo­lio de­ci­sion-mak­ing, along­side im­ple­men­ta­tion of a new ro­bust gov­er­nance mod­el. Tar­get­ed busi­ness de­vel­op­ment to strength­en the Group’s pipeline and tech­nol­o­gy ca­pa­bil­i­ties will al­so be part of the new R&D ap­proach. 

And then there’s the po­ten­tial. Look­ing to make a come­back in phar­ma R&D, GSK tout­ed a BC­MA ther­a­py in de­vel­op­ment — hard­ly the first. HIV al­so war­rants a men­tion, though that will be cen­tered in its ma­jor­i­ty-owned sub­sidiary Vi­iV,  which has been mak­ing sig­nif­i­cant ad­vances along­side the vac­cines group.

With lit­tle in the way of specifics to of­fer from the phar­ma side in the near term, GSK is vague­ly promis­ing big things be­yond 2020.

In a con­fer­ence call with re­porters to­day — at least the ones that weren’t al­lowed a pre­view — Bar­ron and 23andMe chief Anne Wo­j­ci­c­ki hit the dis­cov­ery an­gle hard, high­light­ing what they see as an op­por­tu­ni­ty to find gene vari­ants for Parkin­son’s and can­cer and oth­er dis­eases that would make good new pro­grams.

I asked Bar­ron for his as­sess­ment of the R&D op­er­a­tion he had tak­en on. He em­pha­sized the pos­i­tive, but al­so ac­knowl­edged the weak­ness­es.

“I think it’s im­por­tant to look back at what GSK has done well,” he replied. There have been a num­ber of new ap­provals, he said, but with­out the kind of com­mer­cial im­pact that can gen­er­ate the num­bers the com­pa­ny needs. This new al­liance with 23andMe, he said, of­fered the chance to get at that — with new tar­gets that could be suit­able for mul­ti­ple ap­provals and bet­ter life cy­cle man­age­ment of im­por­tant new drugs.

I tried to fol­low up with a ques­tion on whether GSK had a dis­cov­ery or late-stage drug prob­lem, but got cut off on the call.

GSK brought in the leg­endary Genen­tech vet to turn around what is per­haps the slow­est, most woe­ful Big Phar­ma R&D group in the world. A ge­net­ics data­base deal with 23andMe will of­fer no short-term help, of­fer­ing a leg up for dis­cov­ery work that will be years in the mak­ing. And Bar­ron will try to or­ches­trate the turn­around from his base in the Bay Area, far from GSK’s cen­tral re­search op­er­a­tions around Philadel­phia and Steve­nage in the UK.

De­spite a bud­get of $6 bil­lion, Glax­o­SmithK­line has had lit­tle in the late-stage pipeline to boast about — and the late-stage pipeline is what is used to judge a com­pa­ny’s po­ten­tial in turn­ing out block­busters.

GSK’s phar­ma R&D group doesn’t make block­busters, though. Or hasn’t in years. And adopt­ing a strat­e­gy that any busi­ness school grad­u­ate could have whipped up one af­ter­noon won’t gin up miss­ing ex­cite­ment in the in­vest­ment com­mu­ni­ty.


Im­age: Hal Bar­ron. GSK

Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

Franz-Werner Haas, CureVac CEO

UP­DAT­ED: On the heels of a snap $1B raise, Cure­Vac out­lines plans to seek emer­gency OK for Covid-19 vac­cine -- shares rock­et up

CureVac is going from being one of the quietest players in the race to develop a new vaccine to fight the worst public health crisis in a century to a challenger for the multibillion-dollar market that awaits the first vaccines to make it over the finish line. Typically low-key at a time of brash comments and incredibly ambitious development timelines from the leaders, CureVac now is jumping straight into the spotlight.

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Inside FDA HQ (File photo)

The FDA just ap­proved the third Duchenne MD drug. And reg­u­la­tors still don’t know if any of them work

Last year Sarepta hit center stage with the FDA’s controversial reversal of its CRL for the company’s second Duchenne muscular dystrophy drug — after the biotech was ambushed by agency insiders ready to reject a second pitch based on the same disease biomarker used for the first approval for eteplirsen, without actual data on the efficacy of the drug.

On Wednesday the FDA approved the third Duchenne MD drug, based on the same biomarker. And regulators were ready to act yet again despite the lack of efficacy data.

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US gov­ern­ment re­port­ed­ly be­gins prepar­ing for Covid-19 chal­lenge tri­als. Are they eth­i­cal?

Controversial human challenge trials for potential Covid-19 vaccines reportedly have a new booster — the US government.

Scientists working for the government have begun manufacturing a strain of the novel coronavirus that could be used in such studies, Reuters reported Friday morning. The trials would enroll healthy volunteers to be vaccinated and then intentionally infected with a weakened coronavirus.

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Cal­lid­i­tas bets up to $102M on a biotech buy­out, snag­ging a once-failed PBC drug

After spending years developing its oral formulation of the corticosteroid budesonide, Sweden’s Calliditas now has its sights set on the primary biliary cholangitis field.

The company will buy out France-based Genkyotex, and it’s willing to bet up to €87 million ($102 million) that Genkyotex’s failed Phase II drug, GKT831, will do better in late-stage trials.

Under the current agreement, Calliditas $CALT will initially pay €20.3 million in cash for 62.7% of Genkyotex (or €2.80 a piece for 7,236,515 shares) in early October, then circle back for the rest of Genkyotex’s shares under the same terms. If nothing changes, the whole buyout will cost Calliditas €32.3 million, plus up to  €55 million in contingent rights.

James Wilson, WuXi Global Forum at JPM20

FDA puts up a red light for Pas­sage Bio’s first gene ther­a­py pro­gram, de­lay­ing a pro­gram from James Wilson's group at Penn

Gene therapy pioneer James Wilson spearheaded animal studies demonstrating the potential of new treatments injected directly into the brain, looking to jumpstart a once-and-done fix for an extraordinarily rare disease called GM1 gangliosidosis in infants. His team at the University of Pennsylvania published their work on monkeys and handed it over to Passage Bio, a Wilson-inspired startup building a pipeline of gene therapies — with an IND for PBGM01 to lead the way.

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Phase III read­outs spell dis­as­ter for Genen­tech’s lead IBD drug

Roche had big plans for etrolizumab. Eyeing a hyper-competitive IBD and Crohn’s market where they have not historically been a player, the company rolled out 8 different Phase III trials, testing the antibody for two different uses across a range of different patient groups.

On Monday, Roche released results for 4 of those studies, and they mark a decided setback for both the Swiss pharma and their biotech sub Genentech, potentially spelling an end to a drug they put over half-a-decade and millions of dollars behind.

Trevor Martin (Mammoth)

Eye­ing in-vi­vo edit­ing, Mam­moth li­cens­es Jen­nifer Doud­na’s new CRISPR en­zyme

Last month, Jennifer Doudna revealed in Science a new, “hyper-compact” CRISPR enzyme that was half the size of traditional CRISPR enzymes and could, she suspected, offer a new, more versatile tool for gene editing.

Now, the University of California-Berkeley has licensed that enzyme, known as Casφ, exclusively to a biotech startup she and two former students set up three years ago: Mammoth Biosciences. It’s the second new CRISPR protein Mammoth has licensed from Doudna’s lab, after they licensed Cas14 in 2019.

Clockwise from left: Canaccord Genuity principal Michelle Gilson, Canaccord Genuity CSO Brian Mueller and BioMarin CSO Hank Fuchs (Canaccord Genuity webcast)

Bio­Marin CSO diss­es ri­vals for the he­mo­phil­ia A gene ther­a­py crown: Way be­hind, fac­ing big re­cruit­ment chal­lenges and at best a .6 on the gen-one scale

The leader in the race to a hemophilia A gene therapy does not like to be compared unfavorably to the competition. And when their top execs do the comparing, don’t look for any modesty — BioMarin, they say, owns the lead.

As Factor VIII expression wanes over time, quite a few analysts have raised questions about the kind of future BioMarin’s gene therapy — a supposed once-and-done treatment — faces if it stops working. But just 7 days away from their PDUFA date, with high odds of success, the top execs clearly feel that they are way out front, while promising their rivals will discover there’s a tough slog ahead trying to pursue trials where large numbers of patients are ineligible for new therapies.

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