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

 

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.

A lab technician works during research on coronavirus at Johnson & Johnson subsidiary Janssen Pharmaceutical in Beerse, Belgium, Wednesday, June 17, 2020. (Virginia Mayo/AP Images)

UP­DAT­ED: End­points News ranks all 28 play­ers in the Covid-19 vac­cine race. Here's how it stacks up to­day

(This piece was last updated on August 13. Endpoints News will continue to track the latest developments through the FDA’s marketing decisions.)

The 28 players now in or close to the clinical race to get a Covid-19 vaccine over the finish line are angling for a piece of a multibillion-dollar market. And being first — or among the leaders — will play a big role in determining just how big a piece.

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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.

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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Stéphane Bancel speaks to President Donald Trump at the White House meeting on March 2 (AP Images)

UP­DAT­ED: Mod­er­na of­fers steep dis­count in US sup­ply deal — but still takes the crown with close to $2.5B in vac­cine con­tracts

The US pre-order for Moderna’s Covid-19 vaccine is in.

Operation Warp Speed is reserving $1.525 billion for 100 million doses of Moderna’s Phase III mRNA candidate, rounding out to about $15 per dose — including $300 million in incentive payments for timely delivery. Given that Moderna has a two-dose regimen, it’s good for vaccinating 50 million people. The US government also has the option to purchase another 400 million doses for a total of $6.6 billion, or $16.5 per dose.

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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.

Qi­a­gen in­vestors spurn Ther­mo Fish­er’s takeover of­fer, de­rail­ing a $12B+ deal

Thermo Fisher Scientific had announced an $11.5 billion takeover of Dutch diagnostics company Qiagen back in March, but the deal apparently did not sit well with Qiagen investors.

After getting hammered by critics who contended that Qiagen $QGEN was worth a lot more than what Thermo Fisher wanted to spend, investors turned thumbs down on the offer — derailing the buyout even after Thermo Fisher increased its offer to $12.6 billion in July. Qiagen’s share price has been boosted considerably by Covid-19 as demand for its testing kits surged.

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Xuefeng Yu in Hong Kong, 2019 (Imaginechina via AP Images)

CanSi­no reaps $748M wind­fall from Shang­hai IPO — as it warns Covid-19 vac­cine won't be a huge mon­ey mak­er

CanSino began the year with a clear goal to secure a secondary listing on Shanghai’s STAR market. Then something more urgent came along: As a rising vaccine developer on a mission to bring global standard immunizations to China, it heeded the call to make a vaccine to protect against a virus that would paralyze the whole world.

Xuefeng Yu and his team managed to keep doing both.

More than a month after CanSino’s Covid-19 vaccine candidate is authorized for military use in China, the Hong Kong-listed company has made a roaring debut in Shanghai. It fetched $748 million (RMB$5.2 billion) by floating 24.8 million shares, and soared 88% on its first trading day.