Thumbs up: Can­cer drug R&D is ex­plod­ing, get ready for the pric­ing de­bate to fol­low

I ar­rived in Chica­go last night, still try­ing to sort out how to pri­or­i­tize the news com­ing out of AS­CO this week­end. In the lead up, I had a chance to do a pair of sto­ries that looked at new re­ports cov­er­ing the rapid pro­lif­er­a­tion of PD-(L)1 com­bos along with a 7.7% one-year growth rate in late-stage can­cer drug stud­ies, com­ing in at 631 piv­otal-stage ef­forts.

This didn’t hap­pen by ac­ci­dent.

Over the last few years we’ve seen a tru­ly rev­o­lu­tion­ary change in the way on­col­o­gy drugs are de­vel­oped. Backed in par­tic­u­lar by the FDA’s break­through pro­gram, now lib­er­al­ly ap­plied along with fast-track sta­tus and pri­or­i­ty re­views, the bio­phar­ma in­dus­try is com­pet­ing like nev­er be­fore to carve out new ter­ri­to­ry in can­cer treat­ment.

I well re­mem­ber a lot of the ini­tial hoot­ing from some an­a­lysts at the idea that the FDA’s BTD pro­gram would make a dif­fer­ence. But no one is hoot­ing now. The agency — and in par­tic­u­lar Richard Paz­dur — can­did­ly em­braced an open-door ap­proach on can­cer R&D. And that’s the path FDA com­mis­sion­er Scott Got­tlieb wants the rest of the FDA di­vi­sions to fol­low.

Richard Paz­dur

When a bu­reau­cra­cy sets new goals, things change. So I’ll be ex­pect­ing lots of fresh ac­tion on the BTD front over the next few years, for all kinds of dis­eases.

There are bil­lions of dol­lars at stake here, which is as it should be. The greater the pay­back, the greater the R&D in­vest­ment and the greater the re­wards for pa­tients, who are see­ing some im­por­tant and some­times dra­mat­ic im­prove­ment in the way many can­cers are treat­ed.

Out of this rev­o­lu­tion, though, will come plen­ty of added pres­sure on drug mak­ers to find a way to rea­son­ably price these new drugs and the com­bos com­ing down the pipeline. Af­ter get­ting side­lined at the Trump ad­min­is­tra­tion, changes on pub­lic pol­i­cy around drug pric­ing may well get lost in all the shout­ing in DC these days.

But you can be sure that pay­ers haven’t lost their fo­cus, es­pe­cial­ly as six-fig­ure com­bos start to make the rounds. So the in­dus­try will need to have a more co­her­ent ap­proach to pay for per­for­mance.

On­col­o­gy now rep­re­sents the po­ten­tial that drug R&D has in sig­nif­i­cant­ly im­prov­ing the stan­dard of care in a short pe­ri­od of time. Next, it has to lead the way on mar­ket-based so­lu­tions to the pric­ing de­bate.

That next part could prove to be much, much more chal­leng­ing.

Ryan Watts, Denali CEO

Bio­gen hands De­nali $1B-plus in cash, $1B-plus in mile­stones to part­ner on late-stage Parkin­son’s drug

Biogen is handing over more than a billion dollars cash to partner with the up-and-coming neurosciences crew at Denali on a new therapy for Parkinson’s. And the big biotech is ready to pile on more than a billion dollars more in milestones — if the alliance is a success.

For Biogen $BIIB, the move on Denali’s small molecule inhibitors of LRRK2 puts them in line to collaborate on a late-stage program for DNL151, which is scheduled to start next year.

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Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen scores a pri­or­i­ty re­view for its Alzheimer's drug ad­u­canum­ab, mov­ing one gi­ant leap for­ward in its con­tro­ver­sial quest

Biogen scored a big win at the FDA today as regulators accepted their application for the controversial Alzheimer’s drug aducanumab and gave it a priority review.

The PDUFA date is March 7, 2021.

Significantly, Biogen says it did not use its priority review voucher to win special treatment at the FDA. The agency handed that out gratis.

That’s the ideal scenario Biogen was looking for as disappointed analysts wondered aloud about the delayed application earlier in the year.

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Levi Garraway, Roche CMO (Source: Genentech)

UP­DAT­ED: FDA hands out a quick OK for po­ten­tial SMA block­buster ris­diplam, giv­ing Genen­tech and Roche a chance to chal­lenge ri­vals on the price

US regulators handed Roche and Genentech a big win Friday afternoon, one that has market-shaping potential for its high-priced rivals from Novartis and Biogen.

The FDA has green-lit the companies’ spinal muscular atrophy drug risdiplam, which will be marketed as Evrysdi in the US, for use in patients two months and older. It’s the first SMA drug that can be taken orally, as Biogen’s Spinraza is injected into the spine while Novartis’ Zolgensma is a gene therapy.

Moncef Slaoui, Getty Images

When will it end? Big Phar­ma's top vac­cine ex­pert at OWS of­fers a speedy time­line for a Covid-19 vac­cine — ei­ther be­fore or right af­ter the elec­tion

Moncef Slaoui hasn’t started making plans for his summer vacation next year. But he offers high odds that all Americans will be able to do that in the not too distant future.

In an interview with a pair of sympathetic podcasters at the conservative American Enterprise Institute, Slaoui provides an education to listeners on how any drug or vaccine can be sped through trials. And he leaves the door wide open to the notion that the leading vaccine developers can demonstrate efficacy and safety in a compelling fashion as early as October — or as late as the end of this year.

President Trump speaks with members of the media before boarding Marine One (AP Images)

'Oc­to­ber is com­ing,' and every­one still wants to know if a Covid-19 vac­cine will be whisked through the FDA ahead of the elec­tion

Right on the heels of a lengthy assurance from FDA commissioner Stephen Hahn that the agency will not rush through a quick approval for a Covid-19 vaccine, the President of the United States has some thoughts on timing he’d like to share.

In an exchange with Fox News’ Geraldo Rivera on Thursday, President Trump allowed that a vaccine could be ready to roll “sooner than the end of the year, could be much sooner.”

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Covid-19 roundup: Gates Foun­da­tion pours $150M in­to In­dia’s Serum In­sti­tute; Pfiz­er teams with Gilead on remde­sivir

By CEO and scion Adar Poonawalla’s estimation, the Serum Institute in India has already poured hundreds of millions of dollars into scaling up the unproven Covid-19 vaccine being developed by AstraZeneca and Oxford for use in low and middle income countries. It’s meant taking on a risk that other companies, including AstraZeneca, have mitigated with huge amounts of government funding.

Now, for the first time, Poonawalla is getting some outside help. The Gates Foundation has agreed to pay the institute $150 million to supply 100 million vaccines to India and other emerging economies next year, Reuters reported. That includes both the vaccine being developed by AstraZeneca and the one being developed by Novavax. Those vaccines will be available in 92 countries and be priced at $3 per dose.

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UP­DAT­ED: No­vavax her­alds the lat­est pos­i­tive snap­shot of ear­ly-stage Covid-19 vac­cine — so why did its stock briefly crater?

High-flying Novavax $NVAX became the latest of the Covid-19 vaccine players to stake out a positive set of biomarker data from its early-stage look at its vaccine in humans.

Their adjuvanted Covid-19 vaccine was “well-tolerated and elicited robust antibody responses numerically superior to that seen in human convalescent sera,” the company noted. According to the biotech:

All subjects developed anti-spike IgG antibodies after a single dose of vaccine, many of them also developing wild-type virus neutralizing antibody responses, and after Dose 2, 100% of participants developed wild-type virus neutralizing antibody responses. Both anti-spike IgG and viral neutralization responses compared favorably to responses from patients with clinically significant COVID‑19 disease. Importantly, the IgG antibody response was highly correlated with neutralization titers, demonstrating that a significant proportion of antibodies were functional.

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Bio­haven adds near­ly $1B in Nurtec deals with Roy­al­ty Phar­ma, Sixth Street

Biohaven just added nearly $1 billion to their balance sheet.

On Friday morning, the neuroscience biotech announced a pair of creative agreements with Royalty Pharma and the investment firm Sixth Street to bolster the commercial launch of their new migraine drug, Nurtec. Biohaven will sell a sliver of its royalties on Nurtec and 3% of the royalties on their experimental migraine drug zavegepant to Royalty Pharma as part of a larger agreeement that will pay $450 million. At the same time, the company announced they took out a $500 million loan from Sixth Street.

Ab­b­Vie set­tles in­sur­ance fraud suit, agrees to tweak nurse am­bas­sador pro­gram; CStone aims for NSCLC OK with pos­i­tive PhI­II da­ta

AbbVie has resolved a California lawsuit alleging insurance fraud in the promotion of its cash cow Humira, paying $24 million to settle things with the state’s insurance regulator.

The settlement comes almost four years after a whistleblower first reported AbbVie’s practice of deploying registered nurses to visit patients at home or call them by phone to ensure that Humira prescriptions are filled. AbbVie was also charged with providing illegal kickbacks to doctors in an attempt to encourage them to prescribe Humira for a range of anti-inflammatory diseases.