Ar­row­head touts Phase Ib RNAi da­ta for ccR­CC; PureTech lines up PD-1 from BeiGene for up­com­ing com­bo study

Just days af­ter get­ting wal­loped by the news that an­i­mal tox da­ta forced them to halt an ear­ly-stage cys­tic fi­bro­sis study, Ar­row­head is tout­ing some ini­tial snap­shots from their RNAi treat­ment for clear cell re­nal cell car­ci­no­ma.

Most of the pos­i­tive signs they’re point­ing to re­late to bio­mark­er ac­tiv­i­ty, with an av­er­age 48% re­duc­tion in HIF2α pro­tein among nine evalu­able biop­sy sam­ples. Those re­duc­tions var­ied wide­ly, though, start­ing at 9% and grow­ing to 82%.

There was one par­tial tu­mor re­sponse in the small group, though re­searchers not­ed the pa­tients had failed mul­ti­ple lines of ther­a­py. — John Car­roll

PureTech lines up a PD-1 from BeiGene for up­com­ing com­bo study

PureTech has struck a sup­ply deal with BeiGene to line up their PD-1 drug tislelizum­ab for a com­bi­na­tion study in­volv­ing LYT-200, an ex­per­i­men­tal an­ti­body tar­get­ing galectin-9 for dif­fi­cult-to-treat sol­id tu­mors.

This is part of a two-step ef­fort to test PureTech’s drug as a monother­a­py first, fol­lowed by the com­bi­na­tion with tislelizum­ab and oth­er agents.

“Af­ter a decade of op­ti­miz­ing use of im­muno-on­col­o­gy ther­a­pies, such as the check­point in­hibitors that have cer­tain­ly pro­vid­ed a par­a­digm shift in treat­ing ma­lig­nant dis­eases, we in the in­dus­try are ea­ger to ad­vance nov­el break­through agents and com­bi­na­tions to serve a wider range of can­cer pa­tients. Pa­tients need new op­tions as the first wave of im­munother­a­pies work on­ly in a small per­cent­age of them. Well thought-out com­bi­na­tion im­munother­a­py reg­i­mens may be that way for­ward,” said Alek­san­dra Fil­ipovic, head of on­col­o­gy at PureTech. — John Car­roll

Bio­haven re­ports bet­ter than ex­pect­ed sales on mi­graine drug, shares jump

Bio­haven $BHVN saw its shares tick up­ward Wednes­day morn­ing af­ter sharply beat­ing sales es­ti­mates for its mi­graine drug Nurtec, fol­low­ing a re­cent new ap­proval as a pre­ven­ta­tive.

Shares of the New Haven, CT-based biotech were trad­ing high­er by as much as 13% in ear­ly Wednes­day trad­ing af­ter Nurtec reaped near­ly $93 mil­lion in sales in the sec­ond quar­ter. Orig­i­nal­ly ap­proved in Feb­ru­ary 2020 to treat acute mi­graines, Nurtec tacked on pre­ven­ta­tive treat­ment this past May.

“Our sec­ond quar­ter net rev­enue per­for­mance re­flects the over­whelm­ing­ly pos­i­tive feed­back we are re­ceiv­ing from pa­tients and health­care providers about the im­pact of Nurtec ODT on mi­graine,” CCO BJ Jones said in a state­ment.

Bio­haven says its oral CGRP drug cov­ers about 95% of avail­able mi­graine pa­tients. It’s like­ly to face com­pe­ti­tion from Ab­b­Vie’s Ubrelvy, which launched just a cou­ple of months be­fore Nurtec in ear­ly 2020. — Max Gel­man

No­var­tis reshuf­fles its wild cards; Tough sell for Bio­gen? Googling pro­teins; Ken Fra­zier's new gig; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

If you enjoy the People section in this report, you may also want to check out Peer Review, my colleagues Alex Hoffman and Kathy Wong’s comprehensive compilation of comings and goings in biopharma.

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Demis Hassabis, DeepMind CEO (Qianlong/Imaginechina via AP Images)

Google's Deep­Mind opens its pro­tein data­base to sci­ence — po­ten­tial­ly crack­ing drug R&D wide open

Nearly a year ago, Google’s AI outfit DeepMind announced they had cracked one of the oldest problems in biology: predicting a protein’s structure from its sequence alone. Now they’ve turned that software on nearly every human protein and hundreds of thousands of additional proteins from organisms important to medical research, such as fruit flies, mice and malaria parasite.

The new database of roughly 350,000 protein sequences and structures represents a potentially monumental achievement for the life sciences, one that could hasten new biological insights and the development of new drugs. DeepMind said it will be free and accessible to all researchers and companies.

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In­side Bio­gen's scram­ble to sell Aduhelm: Pro­ject 'Javelin' and pres­sure to ID as many pa­tients as pos­si­ble

In anticipation of Aduhelm’s approval for Alzheimer’s in June, Biogen employees were directed to identify and guarantee treatment centers would administer the drug through a program called “Javelin,” a senior Biogen employee told Endpoints News.

The program identified about 800 centers for use, he said, and Biogen now pays for the use of bioassays to identify beta amyloid in potential patients having undergone a lumbar puncture procedure, the employee said — and one center preparing to administer the drug confirmed its participation in the bioassay program.

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Vas Narasimhan, Novartis CEO (Jason Alden/Bloomberg via Getty Images)

No­var­tis dis­cards one of its ‘wild card’ drugs af­ter it flops in key study. But it takes one more for the hand

Always remember just how risky it is to gamble big on small studies.

A little more than 4 years ago, Novartis reportedly put up a package worth up to $1 billion for the dry eye drug ECF843 after a small biotech called Lubris put it through its paces in a tiny study of 40 moderate to severe patients, tracking some statistically significant markers of efficacy.

By last fall, the program had risen up to become one of CEO Vas Narasimhan’s top “wild card” programs in line for a potential breakthrough year in 2021. These drugs were all considered high-risk, high-reward efforts. And in this case, risk won.

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Mod­er­na es­tab­lish­es pub­lic health-fo­cused char­i­ty; FDA ap­proves As­traZeneca di­a­betes drug for pe­di­atric use

To help promote public health and healthcare in underserved areas of the world, Moderna will establish a charity with a $50 million endowment.

The Cambridge, MA-based company announced the board of directors’ approval Thursday. The foundation will focus on “charitable, scientific and educational endeavors” with an emphasis on promoting public health and the access to healthcare, the press release said. The foundation will start operations once its status as a 501(c)(3) is approved.

EMA re­jects FDA-ap­proved Parkin­son's drug, signs off on Mod­er­na vac­cine use in ado­les­cents ahead of FDA

The European Medicines Agency on Friday rejected Kyowa Kirin’s Parkinson’s disease drug Nouryant (istradefylline), which the US FDA approved in 2019 under the brand name Nourianz.

EMA said it considered that the results of the clinical studies used to support the application “were inconsistent and did not satisfactorily show that Nouryant was effective at reducing the ‘off’ time. Only four out of the eight studies showed a reduction in ‘off’ time, and the effect did not increase with an increased dose of Nouryant.”

6 top drug­mak­ers of­fer per­spec­tives on FDA's new co­vari­ates in RCTs guid­ance

Back in May, the FDA revised and expanded a 2019 draft guidance that spells out how to adjust for covariates in the statistical analysis of randomized controlled trials.

Building on the ICH’s E9 guideline on the statistical principles for clinical trials, the 3-page draft was transformed into an 8-page draft, with more detailed recommendations on linear and nonlinear models to analyze the efficacy endpoints in RCTs.

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Al Sandrock, Biogen R&D chief (Biogen via YouTube)

Bio­gen has a shaky end to H1 with a $542M write-off adding to its woes — but an­a­lysts see big rev­enue ahead for Aduhelm

All eyes at Biogen’s Q2 earnings call Thursday were on Aduhelm, but investors also got a glimpse of what Biogen would have faced had the FDA not opted to approve their controversial Alzheimer’s drug.

That glimpse, revealing a combination of declining sales, growing competition and failed medicines, underscores the stakes of the big biotech’s Aduhelm efforts, as execs punch back at the criticism they’ve engendered in the political and medical world and vigorously pushes its sales staff to roll out the drug as fast as possible.

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Mol­e­c­u­lar Di­ag­nos­tics Can Trans­form Can­cer Care. Let’s Make It Hap­pen.

Like so many people around the world, my life has been profoundly shaped by cancer. Those personal experiences, along with a deep love of clinical laboratory science and a passion to apply the power of genomics in medicine, motivated me to launch a company that would improve cancer care through better diagnostics. Thirteen years later, I am proud that we are delivering more accurate information at multiple points along the patient journey, with a focus on eight of the 10 cancers that are most commonly diagnosed in the United States.