Boehringer dumps its $250M NASH drug; Bavar­i­an Nordic sells PRV for $95M; GBT inks deal with Sy­ros, gets $150M loan

→ Four years af­ter Boehringer hand­ed out a $250 mil­lion deal to get a NASH drug from Phar­max­is, they’re sweep­ing it out of the pipeline. The Ger­man phar­ma com­pa­ny not­ed to­day that the drug posed a threat of drug in­ter­ac­tions, scut­tling its as­pi­ra­tions for the pro­gram. BI 1467335 joins a long — and grow­ing — list of fail­ures in the NASH field, where drug de­vel­op­ers have been try­ing to field a ther­a­py that can bend the nee­dle on a dis­ease that af­flicts mil­lions of peo­ple world­wide.

→ It looks like $95 mil­lion is the go­ing price for pri­or­i­ty re­view vouch­ers these days. The lat­est sell­er is Bavar­i­an Nordic, which was award­ed the PRV in Sep­tem­ber fol­low­ing the ap­proval of its small­pox and mon­key­pox vac­cine, Jyn­neos. The Dan­ish biotech said it will be us­ing the new funds to ex­pand the man­u­fac­tur­ing and com­mer­cial op­er­a­tions. The buy­er — who can now shave six months off the re­view process on any drug they like — re­mains undis­closed. While this ex­pe­di­tion tick­et once com­mand­ed $350 mil­lion, the price has been steadi­ly de­creas­ing and As­traZeneca bought one from So­bi in Au­gust for just $95 mil­lion.

→ At one point about 4 years ago, Zaf­gen shares $ZFGN trad­ed north of $46 and ad­mir­ing in­vestors were stoked by its blitz of up­beat an­nounce­ments on a rad­i­cal new obe­si­ty drug that promised to rev­o­lu­tion­ize treat­ment in a va­ri­ety of large and small mar­ket nich­es. Then the ceil­ing caved in af­ter their lead drug killed a pa­tient, forc­ing the com­pa­ny to even­tu­al­ly scrap the ef­fort. To­day, af­ter lin­ger­ing on life sup­port for more than a year fol­low­ing their sec­ond crip­pling clin­i­cal hold by the FDA, the shell of what’s left is be­ing used by an­oth­er biotech to re­verse merge it­self on­to the Nas­daq. Chon­dr­i­al Ther­a­peu­tics goes in­to the re­verse and will come out as Lari­mar Ther­a­peu­tics with a lead pro­gram for Friedre­ich’s atax­ia.

Glob­al Blood Ther­a­peu­tics is not let­ting the launch of its first drug hold it back from de­vel­op­ing more treat­ments for sick­le cell dis­ease and be­ta tha­lassemia. The biotech is team­ing up with Sy­ros Phar­ma­ceu­ti­cals to dis­cov­er drugs that in­duce fe­tal he­mo­glo­bin on Sy­ros’ gene con­trol plat­form, with the ul­ti­mate goal of hit­ting po­ten­tial tar­gets that can switch on the gam­ma glo­bin gene with oral med­i­cines. GBT is kick­start­ing the col­lab­o­ra­tion with $20 mil­lion up­front and up to $40 mil­lion in pre­clin­i­cal re­search for at least three years — with up to $315 payable in op­tion fees and mile­stones.

To help fund all that, and more, the com­pa­ny $GBT is tak­ing a $150 mil­lion loan from Phar­makon Ad­vi­sors that would of­fer “fi­nan­cial flex­i­bil­i­ty” to dis­cov­er and de­vel­op po­ten­tial treat­ments with­out di­vert­ing re­sources from the launch of Oxbry­ta, their new­ly ap­proved in­hibitor of de­oxy­genat­ed sick­le he­mo­glo­bin poly­mer­iza­tion, said CFO Jeff Far­row in a state­ment.

→ Days af­ter show­cas­ing promis­ing da­ta on tu­ca­tinib, Seat­tle Ge­net­ics said the drug has now been des­ig­nat­ed a “break­through” ther­a­py by the FDA for lo­cal­ly ad­vanced or metasta­t­ic HER2-pos­i­tive breast can­cer. Added to the stan­dard reg­i­men of trastuzum­ab and capecitabine, the drug has been shown to ex­tend OS and PFS even in pa­tients with brain metas­tases. Fil­ings in the US and Eu­rope are planned for ear­ly 2020.

Eli Lil­ly CEO David Ricks is hit­ting back at Sen­a­tor Eliz­a­beth War­ren (D-MA) af­ter the De­mo­c­ra­t­ic pres­i­den­tial hope­ful ac­cused the com­pa­ny of fail­ing to de­liv­er on a promise to de­liv­er low­er-priced in­sulin to pa­tients. Based on a new sur­vey of phar­ma­cies, War­ren and Sen­a­tor Richard Blu­men­thal (D-CT) said the half-price in­sulin — an au­tho­rized gener­ic of Hu­ma­log dubbed In­sulin Lispro — was of­ten not in stock or avail­able to pa­tients. Ricks, speak­ing to CN­BC, said the claims are “non­sense” and shows “what’s bro­ken in the rest of the phar­ma­ceu­ti­cal sys­tem,” name­ly the mid­dle­men in the sup­ply chain such as phar­ma­cy ben­e­fit man­agers.

Da­ta Lit­er­a­cy: The Foun­da­tion for Mod­ern Tri­al Ex­e­cu­tion

In 2016, the International Council for Harmonisation (ICH) updated their “Guidelines for Good Clinical Practice.” One key shift was a mandate to implement a risk-based quality management system throughout all stages of a clinical trial, and to take a systematic, prioritized, risk-based approach to clinical trial monitoring—on-site monitoring, remote monitoring, or any combination thereof.

Mer­ck scraps Covid-19 vac­cine pro­grams af­ter they fail to mea­sure up on ef­fi­ca­cy in an­oth­er ma­jor set­back in the glob­al fight

After turning up late to the vaccine development game in the global fight against Covid-19, Merck is now making a quick exit.

The pharma giant is reporting this morning that it’s decided to drop development of 2 vaccines — V590 and V591 — after taking a look at Phase I data that simply don’t measure up to either the natural immune response seen in people exposed to the virus or the vaccines already on or near the market.

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News brief­ing: Jef­frey Lei­den to chair Tmu­ni­ty board of di­rec­tors; Op­di­vo wins new ap­proval in ad­vanced RCC

Longtime Vertex CEO Jeffrey Leiden is taking on a new role.

Leiden has been appointed chairman of Tmunity’s board of directors, the company announced Monday. The move comes about a year and a half after Leiden announced he’d be stepping down from his position at Vertex.

Vertex saw immense growth under Leiden, leading the company from its exit out of hepatitis C, when cures were moving in, and into cystic fibrosis. The company’s cystic fibrosis triple combo therapy Trikafta is already its best-seller, reaching the distinction just six weeks after launch and recording the strongest first quarter of sales for any drug, per some estimates.

Matt Gline (L) and Vivek Ramaswamy

Scoop: Vivek Ra­maswamy is hand­ing the CEO job to a top lieu­tenant at Roivant — but he’s not ex­act­ly leav­ing the biotech scene

Over the past 7 years since founding Roivant, Vivek Ramaswamy has been a constant blur of biotech building motion.

He launched his first biotech with an Alzheimer’s drug he picked up cheap, and watched the experiment implode in one of the highest profile pivotal disasters seen in the last decade. But it didn’t slow the 30-something exec down; if anything, he hit the accelerator. Ramaswamy blazed global paths and went on to raise billions to spur the creation of a large lineup of little Vants promising big things at a fast pace. He sold off a section of the Vant brigade to Sumitomo Dainippon for $3 billion. And more recently the relentless dealmaker has been building a computational discovery arm to add an AI-driven approach to kicking up new programs and companies, supplementing the in-licensing drive while pursuing advances that have created more than 700 jobs at Roivant, with $2 billion in reserves.

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Ron Cooper, Albireo CEO

Al­bireo just ad­vanced down to the 10-yard line at the FDA. And Ron Coop­er’s team is get­ting prepped for the next big play

When Albireo Pharma’s board $ALBO moved to bring in Ron Cooper as the CEO more than 5 years ago, the development-stage company went with an experienced commercial player who had a big-time position on his resume after running Bristol Myers’ commercial ops in Europe.

Now, after successfully navigating a pivotal study, putting them in a foot race with a rival toward an FDA OK, Cooper is getting a boost from regulators on the last drive back to an arena he understands completely.

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Hal Barron, GSK via YouTube

What does $29B buy you in Big Phar­ma? In Glax­o­SmithK­line’s case, a whole lot of un­com­fort­able ques­tions about the pipeline

Talk about your bad timing.

A little over a week ago, GSK R&D chief Hal Barron marked his third anniversary at the research helm by taking a turn at the virtual podium during JP Morgan to make the case that he and his team had built a valuable late-stage pipeline capable of churning out more than 10 blockbusters in the next 5 years.

And then, just days later, one of the cancer drugs he bet big on as a top prospect — bintrafusp, partnered with Merck KGaA — failed its first pivotal test in non-small cell lung cancer.

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Can strug­gling Iterum turn the cor­ner to an an­tibi­ot­ic suc­cess sto­ry? They will know in six months

More than five years after Corey Fishman and Michael Dunne dusted sulopenem off Pfizer’s shelves — the second castoff antibiotic they’ve brought out of the pharma giant — and founded Iterum Therapeutics around that single drug, they have lined up a quick shot at approval with priority review from the FDA.

The decision, six months from now, will mark a make-or-break moment for a struggling biotech that has just enough cash to keep the lights on until the third quarter.

Bahija Jallal, Immunocore

Buried in Im­muno­core's IPO fil­ings? A kick­back scheme from a now for­mer em­ploy­ee

Immunocore spent much of 2019 dealing with the fallout of the Neil Woodford scandal, as the former star investor’s fall crashed the biotech’s valuation out of unicorn range. Now it turns out that the company spent 2020 dealing with another internal scandal.

The longtime UK biotech darling disclosed in their IPO filing last week that they had fallen victim to an alleged kickback scheme involving one of their employees. After a whistleblower came forward, they said in their F-1, they spent the summer and spring investigating, finding fraud on the part of an employee and two outside vendors.

Stéphane Bancel, Moderna CEO (Steven Ferdman/Getty Images)

Covid-19 roundup: Mod­er­na dou­bles down on Covid-19 with new boost­er tri­als; Aus­tralia plans do­mes­tic pro­duc­tion of As­traZeneca vac­cine amid dis­tri­b­u­tion lag

As Merck bows out of the global race to develop vaccines for Covid-19, Moderna is doubling down to make sure they can quell new variants that have recently emerged and quickly spread.

The Cambridge, MA-based biotech put out word on Monday that in vivo studies indicate their mRNA vaccine works well enough against two strains first detected in the UK and South Africa. But with a six-fold reduction in neutralizing titers observed against the latter strain, the company is launching a new study of a booster version to make sure it can do the job.

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