Sen­a­tors grill Alex Azar on drug prices; Mor­phoSys sells rights to Darza­lex ri­val in $120M deal; Se­r­i­al deal­mak­er X-Chem en­ters new dis­cov­ery pact with Gilead

→ Alex Azar faced a group of skep­ti­cal sen­a­tors on Wednes­day, De­moc­rats and Re­pub­li­cans, as they re­viewed his nom­i­na­tion as chief of HHS un­der Pres­i­dent Don­ald Trump. And for the bulk of the first half of the dis­cus­sion, the sub­ject was drug prices; as in, the price of drugs is too damn high. Azar, who wrapped a lengthy stint as a se­nior ex­ec at Eli Lil­ly about a year ago, was not known for dri­ving down drug prices. But he re­peat­ed­ly agreed that drug prices need to come down now. In one of the sharpest at­tacks, Re­pub­li­can Rand Paul told Azar he has a lot of work to do to con­vince the Amer­i­can peo­ple that he’s work­ing on their be­half, rather than Big Phar­ma. And he vowed to with­hold his sup­port un­less Azar could come up with an ef­fec­tive reim­por­ta­tion plan. De­spite the back-and-forth, Azar will like­ly win con­fir­ma­tion and isn’t ex­pect­ed to do much be­yond en­cour­ag­ing more gener­ic drug ap­provals as a strat­e­gy to rein in drug prices.

Ger­man biotech Mor­phoSys has sold rights to its mul­ti­ple myelo­ma can­di­date MOR202 to Shang­hai’s I-Mab, nab­bing a $20 mil­lion up­front pay­ment with an ex­tra $100 mil­lion avail­able in po­ten­tial mile­stones. The re­gion­al li­cens­ing deal al­lows I-Mab’s af­fil­i­ate I-Mab Bio­phar­ma to de­vel­op and com­mer­cial­ize the C38 an­ti­body in Chi­na, Tai­wan, Hong Kong and Macao. MOR202 is a pur­port­ed ri­val to Janssen/Gen­mab’s Darza­lex (dara­tu­mum­ab), with Mor­phoSys’ drug can­di­date show­ing short­er in­fu­sion times in a Phase I/II study. Mor­phoSys is bring­ing on part­ners to sup­port clin­i­cal de­vel­op­ment. As a re­sult of this new deal, the com­pa­ny has upped its rev­enue pro­jec­tion by €15 mil­lion, push­ing it in­to the €63 mil­lion to €65 mil­lion range for 2017.

→ X-Chem has inked yet an­oth­er dis­cov­ery deal, this time with Gilead. In the agree­ment, which comes with an un­spec­i­fied up­front pay­ment, X-Chem will put its li­brary of “120 bil­lion DNA-en­cod­ed small mol­e­cules” to work in search of an­ti-vi­ral drugs and oth­er un­named ther­a­peu­tic tar­gets. Gilead has the op­tion to li­cense drug leads found un­der the col­lab­o­ra­tion, and will do fur­ther de­vel­op­ment and com­mer­cial­iza­tion of those pro­grams. The Waltham, MA com­pa­ny has inked a long line­up of re­search pacts with big play­ers in the in­dus­try, in­clud­ing Roche, As­traZeneca, Bay­er, Pfiz­er, Alex­ion, MD An­der­son Can­cer Cen­ter, Sanofi and J&J. Most re­cent­ly, the com­pa­ny’s an­nounced deals with Ono, Astel­las and Janssen. On this new pact with Gilead, X-Chem al­so has a shot at li­cens­ing fees and mile­stone pay­ments on leads that come out the agree­ment.

Shao-Lee Lin

→ Biotech vet­er­an Shao-Lee Lin will start at Hori­zon Phar­ma ear­ly next year as ex­ec­u­tive vice pres­i­dent, re­search and de­vel­op­ment and chief sci­en­tif­ic of­fi­cer. In this new po­si­tion, Lin joins CMO Jef­frey Sher­man in man­ag­ing the com­pa­ny’s grow­ing pipeline. As Hori­zon $HZNP gains a larg­er pres­ence in the or­phan drug field while and builds on its port­fo­lio of mar­ket­ed drugs, it could like use Lin’s ex­pe­ri­ence at Ab­b­Vie, Gilead and Am­gen lead­ing drug de­vel­op­ment across im­munol­o­gy, vi­rol­o­gy, in­flam­ma­tion and res­pi­ra­to­ry ther­a­peu­tics. Said Lin in a state­ment: “I’m im­pressed by Hori­zon’s rapid growth and its strat­e­gy for evo­lu­tion from pri­ma­ry care, with a fo­cus on mar­ket­ed med­i­cines, to di­ver­si­fy­ing in­to rare dis­eases and rheuma­tol­ogy and now com­mit­ted to the next stage in its trans­for­ma­tion of build­ing a ro­bust and sus­tain­able re­search and de­vel­op­ment port­fo­lio.”

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive PhI­II for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

Hal Barron has his second positive round of Phase III data in hand for his anti-BCMA antibody drug conjugate belantamab mafodotin (GSK2857916). And GSK’s research chief says the data paves the way for their drive in search of an FDA approval for treating multiple myeloma. 

It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at with any issues.

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Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll

UP­DAT­ED: As­traZeneca’s Imfinzi/treme com­bo strikes out — again — in lung can­cer. Is it time for last rites?

AstraZeneca bet big on the future of their PD-L1 Imfinzi combined with the experimental CTLA-4 drug tremelimumab. But once again it’s gone down to defeat in a major Phase III study — while adding damage to the theory involving targeting cancer with a high tumor mutational burden.

Early Wednesday the pharma giant announced that their NEPTUNE study had failed, with the combination unable to beat standard chemo at overall survival in high TMB cases of advanced non-small cell lung cancer. We won’t get hard data until later in the year, but the drumbeat of failures will call into question what — if any — future this combination can have left.

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Am­gen, Al­ler­gan biosim­i­lar of Roche's block­buster Rit­ux­an clears an­oth­er US piv­otal study 

Novartis $NVS may have given up, but Amgen $AMGN and Allergan $AGN are plowing ahead with their knockoff of Roche’s blockbuster biologic Rituxan in the United States.

Their copycat, ABP 798, was found to have a clinically equivalent impact as Rituxan — meeting the main goal of the study involving CD20-positive B-cell non-Hodgkin’s lymphoma patients. This is the second trial supporting the profile of the biosimilar. In January, it came through with positive PK results in patients with rheumatoid arthritis.