The key dates for KRAS watch­ers through the end of the year — the trail is nar­row and risks are ex­treme

There’s noth­ing quite like a big patent win when it comes to bur­nish­ing your prospects in the pipeline. And for Am­gen, which seems to have res­cued En­brel for a run to 2029, the cheer­ing sec­tion on Wall Street is now fixed on AMG 510 and a key ri­val.

And it didn’t take much da­ta to do it. 

There was the first snap­shot of a hand­ful of pa­tients, with a 50% re­sponse rate. Then came word that Am­gen re­searchers are al­so track­ing re­spons­es in dif­fer­ent can­cers, at least one in col­orec­tal can­cer and ap­pen­diceal too. 

Nev­er mind that the drug doesn’t ac­tu­al­ly have to lose out in a clin­i­cal study to be dis­count­ed — a sig­nif­i­cant drop in re­spons­es in a big­ger group would be enough to dis­il­lu­sion in­vestors. Let’s re­mem­ber Nek­tar. But for now, this land­scape is be­ing paint­ed with block­buster num­bers to fill in.

This morn­ing Evan Seiger­man at Cred­it Su­isse jumped in with this:

We high­light that ear­ly da­ta have been en­cour­ag­ing, though we await de­tail on the reg­is­tra­tional tri­al de­sign, and ad­di­tion­al ef­fi­ca­cy and dura­bil­i­ty da­ta across all in­di­ca­tions. We project ini­tial ap­proval and use in 3L/2L by 2023, gen­er­at­ing sales of >$2.5B by 2030 to which we ap­ply a 40% PoS.

Not bad. 

AMG 510 is rec­og­nized as the lead­ing G12C drug aimed at a pock­et on KRAS that could bring this long-stud­ied tar­get in­to the range of a drug for the first time. And the phar­ma gi­ant is do­ing every­thing pos­si­ble to prove that it can ex­e­cute quick­ly and ef­fec­tive­ly in tak­ing it from Phase I to a reg­is­tra­tion study and on to the FDA. So ku­dos for grab­bing the in­dus­try’s at­ten­tion and keep­ing it. But it’s very risky at every step.

And every­thing could change over a mat­ter of months.

Michael Yee at Jef­feries wants to see more da­ta and is plug­ging some dates in­to his cal­en­dar. From ear­li­er in the week:

At World Lung (9/7-9/10) next up – 50-75% ORR is great? The AMG-510 ab­stract (re­leased 8/21) prob­a­bly wont be that in­ter­est­ing since sub­mis­sion was like­ly back in April. We ex­pect the up­dat­ed lung da­ta in the oral ses­sion on 9/8 to show: (1) ~3 ad­di­tion­al months of fol­low-up for the ini­tial 5 of 10 re­spon­ders (e.g. go­ing from April to per­haps late Ju­ly cut­off), so treat­ment du­ra­tion could ex­tend from 7-27 weeks to 19-39 weeks (e.g. 5-9 months), (2) ORR for high dose com­ing any­where from 50-75% but log­i­cal­ly it should come down from the per­fect 100% (n=3/3) per­haps down to ORR=75% (co not spe­cif­ic how many pts, est but n=10-15 per­haps), (3) no ear­ly re­laps­es or drug re­sis­tance. Ul­ti­mate­ly, we think 4-6 months of du­ra­tion of re­sponse on more pa­tients would be mean­ing­ful as 2L chemo has a low re­sponse with no dura­bil­i­ty…

An­drew Berens at SVB Leerink is al­so check­ing off dates on the 2019 cal­en­dar, with an eye on Mi­rati’s stock, which faces its own cat­a­lyst in Q4.

Mi­rati’s MRTX849 in 4Q19, po­ten­tial­ly at the Triple Meet­ing in Oc­to­ber. While we note re­cent pres­sure on MRTX shares fol­low­ing com­peti­tor down­grades cit­ing high ex­pec­ta­tions for the ini­tial ‘849 da­ta, we see the next two cat­a­lysts—’510 up­dat­ed lung can­cer da­ta at World Lung (9/7-10) and ‘510 da­ta at ES­MO (9/27-10/1)—as like­ly to be fur­ther val­i­dat­ing for the class, pro­vid­ing ad­di­tion­al ev­i­dence of monother­a­py dura­bil­i­ty in non-small cell lung can­cer (NSCLC) and bol­ster­ing con­fi­dence in util­i­ty of the class in tu­mor types be­yond lung can­cer. 

And here’s Yee again on the up­side for the two con­tenders:

In­vestor ex­cite­ment over the two lead­ing KRAS drugs (AMGN and MRTX) is like­ly to con­tin­ue w/ more da­ta over the next few months. If ALK+ lung drugs are $1-2B as a class yet KRAS is 4x big­ger, then it’s theroet­i­cal­ly a line of sight on a mul­ti-bil­lion po­ten­tial op­por­tu­ni­ty in lung can­cer…

Cat­a­lyst watch­ing is al­ways fun in biotech. And this one has lots of ea­ger fol­low­ers. Am­gen hasn’t stirred this much ex­cite­ment in its pipeline for years.

So­cial im­age: Am­gen, AP Im­ages

Scott Gottlieb, AP Images

Scott Got­tlieb is once again join­ing a team that en­joyed good times at the FDA un­der his high-en­er­gy stint at the helm

Right after jumping on Michael Milken’s FasterCures board on Monday, the newly departed FDA commissioner is back today with news about another life sciences board post that gives him a ringside chair to cheer on a lead player in the real-world evidence movement — one with very close ties to the FDA.

Aetion is reporting this morning that Gottlieb is joining their board, a group that includes Mohamad Makhzoumi, a general partner at New Enterprise Associates, where Gottlieb returned after stepping out of his role at the FDA 2 years after he started.

Gottlieb — one of the best connected execs in biopharma — knows this company well. As head of FDA he championed the use of real-world evidence to help guide drug developers and the agency in gaining greater efficiencies, which helped set up Aetion as a high-profile player in the game.

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Deborah Dunsire. Lundbeck

UP­DAT­ED: Deb­o­rah Dun­sire is pay­ing $2B for a chance to leap di­rect­ly in­to a block­buster show­down with a few of the world's biggest phar­ma gi­ants

A year after taking the reins as CEO of Lundbeck, Deborah Dunsire is making a bold bid to beef up the Danish biotech’s portfolio of drugs in what will likely be a direct leap into an intense rivalry with a group of giants now carving up a growing market for new migraine drugs.

Bright and early European time Monday morning the company announced that it will pay up to about $2 billion to buy Alder, a little biotech that is far along the path in developing a quarterly IV formulation of a CGRP drug aimed at cutting back the number of crippling migraines patients experience each month. In a followup call, Dunsire also noted that the company will likely need 200 to 250 reps for this marketing task on both sides of the Atlantic. And analysts were quick to note that the dealmaking at Lundbeck isn’t done, with another $2 billion to $3 billion available for more deals to beef up the pipeline.

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San Diego cou­ple charged with steal­ing trade se­crets, open­ing Chi­nese biotech as DOJ crack­down con­tin­ues

A San Diego couple has been charged with stealing trade secrets from a US hospital and opening a business based off those secrets in China as the controversial industry-wide crackdown on alleged corporate espionage continues. On the same day, the Department of Justice announced they had arrested Beijing representative Zhongsan Liu for allegedly trying to obtain research visas for government recruiters.

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UP­DAT­ED: Bio­gen pulls the plug on prized IPF drug from $562M+ Stromedix buy­out

One of Biogen’s attempts to branch out has flopped as the biotech scraps a mid-stage program for idiopathic pulmonary fibrosis.

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Warts for the win: Aclar­is' lead drug clears piv­otal study

Aclaris Therapeutics has found a way to get rid of the warts and all.

The company — which earlier this month decided to focus on its arsenal of kinase inhibitors — on Monday unveiled positive data from a pivotal study testing its lead experimental drug for use in common warts.

The drug, A-101, was tested in a 502-patient study called THWART-2 — patients enrolled had one to six warts before qualifying for the trial. Patients either self-administered A-101 topical solution or a vehicle twice a week over a two-month period. A higher proportion of patients on the drug (a potent hydrogen peroxide topical solution) saw their warts disappear at day 60, versus the vehicle (p<0.0001) — meeting the main goal of the study.  Each secondary endpoint also emerged in favor of A-101, the company said.

Tower Bridge in London [Shutterstock]

#UK­BIO19: Join GSK’s Hal Bar­ron and a group of top biotech ex­ecs for our 2nd an­nu­al biotech sum­mit in Lon­don

Over the past 10 years I’ve made a point of getting to know the Golden Triangle and the special role the UK biopharma industry plays there in drug development. The concentration of world class research institutes, some of the most accomplished scientists I’ve ever seen at work and a rising tide of global investment cash leaves an impression that there’s much, much more to come as biotech hubs are birthed and nurtured.

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

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Charles Nichols, LSU School of Medicine

Could psy­che­delics tack­le the obe­si­ty cri­sis? A long­time re­searcher in the field says his lat­est mouse study sug­gests po­ten­tial

Psychedelics have experienced a renaissance in recent years amid a torrent of preclinical and clinical research suggesting it might provide a path to treat mood disorders conventional remedies have only scraped at. Now a preclinical trial from a young biotech suggests at least one psychedelic compound has effects beyond the mind, and — if you believe the still very, very early hype — could provide the first single remedy for some of the main complications of obesity.

Ac­celeron drops a de­vel­op­ment pro­gram as #2 drug fails to spark func­tion­al ben­e­fits in pa­tients with a rare neu­ro­mus­cu­lar ail­ment

Acceleron is scrapping a muscular dystrophy development program underway for its number 2 drug in the pipeline after pouring over some failed mid-stage secondary data.

Gone is the ACE-083 project in patients with facioscapulohumeral muscular dystrophy. Their drug hit the primary endpoint on building muscle but flopped on key secondaries for functional improvements in patients, which execs felt was vital to the drug’s success.