De­fend­ing its prize fran­chise, Ver­tex forks over $160M cash to grab Con­cert’s sim­pli­fied ver­sion of Ka­ly­de­co

James Cas­sel­la, Con­cert

Con­cert Phar­ma­ceu­ti­cals’ $CNCE chief claim to fame is us­ing deu­teri­um to re­for­mu­late drugs, stretch­ing ef­fects for pa­tients in ways that make them eas­i­er to use with few­er dos­es. And its mid-stage ef­fort on a longer-act­ing ver­sion of Ver­tex’s Ka­ly­de­co for cys­tic fi­bro­sis was too tempt­ing — or threat­en­ing — to pass up.

Ver­tex an­nounced this morn­ing that it is buy­ing the drug — CTP-656 — for $160 mil­lion in cash and an­oth­er $90 mil­lion in mile­stones, tak­ing over the Phase II tri­al. That’s enough mon­ey to fund Con­cert out an­oth­er four years, and in­vestors ral­lied to the news, dri­ving the biotech’s shares up a whop­ping 78% by mid­day.

James Cas­sel­la, the chief de­vel­op­ment of­fi­cer for Con­cert, not­ed af­ter the Phase I last sum­mer that the drug “pro­file sup­ports sim­pli­fied dos­ing, with the po­ten­tial for im­proved ef­fi­ca­cy.”

That’s an im­por­tant item for Ver­tex $VRTX, which has been de­vel­op­ing cock­tail ther­a­pies with Ka­ly­de­co to ex­pand its reach in the pa­tient pop­u­la­tion. Us­ing a sin­gle dai­ly dose ver­sion of the drug will make that task sim­pler as in­ves­ti­ga­tors work on a sin­gle-dose ver­sions that will help pa­tients stay com­pli­ant with their dos­ing.

Ver­tex isn’t leav­ing any­thing CF re­lat­ed on the ta­ble. In the deal it’s buy­ing all of Con­cert’s work in the field and pick­ing up de­vel­op­ment. And that helps flag Ver­tex’s plans to ag­gres­sive­ly pro­tect its CF fran­chise, says Ge­of­frey Porges. He notes:

CTP-656 was one of nu­mer­ous CF com­peti­tors in ear­ly clin­i­cal tri­als that have been ad­vanced in par­al­lel with Ver­tex’s race to con­coct a triple-ther­a­py reg­i­men for the ma­jor­i­ty of CF pa­tients, and to­day’s news sig­nals, in our view, the will­ing­ness of Ver­tex to de­fend its dom­i­nant and valu­able po­si­tion in the mar­ket cat­e­go­ry.

“With Ver­tex’s clin­i­cal and com­mer­cial ex­per­tise in CF, this agree­ment pro­vides the op­ti­mal path­way to rapid­ly ad­vance the de­vel­op­ment of CTP-656 for the ben­e­fit of cys­tic fi­bro­sis pa­tients,” said Con­ceret CEO Roger Tung in a state­ment. “The fi­nan­cial strength pro­vid­ed to Con­cert by this agree­ment will al­low us to ad­vance CTP-543 in­to piv­otal test­ing and broad­en our pro­pri­etary de­vel­op­ment pipeline.”

Tesla and SpaceX founder Elon Musk gestures to the audience after being recognized by President Trump following the successful launch of a Falcon 9 rocket at the Kennedy Space Center. (via Getty Images)

Tes­la chief Elon Musk teams up with Covid-19 play­er Cure­Vac to build 'R­NA mi­cro­fac­to­ries'

Elon Musk has joined the global tech crusade now underway to revolutionize vaccine manufacturing — now aimed at delivering billions of doses of a new mRNA vaccine to fight Covid-19. And he’s cutting right to the front.

In a late-night tweet Wednesday, the Tesla chief announced:

Tesla, as a side project, is building RNA microfactories for CureVac & possibly others

That’s not a lot to go on. But the tweet comes a year after Tesla’s German division in Grohmann and CureVac filed a patent on a “bioreactor for RNA in vitro transcription, a method for RNA in vitro transcription, a module for transcribing DNA into RNA and an automated apparatus for RNA manufacturing.” CureVac, in the meantime, has discussed a variety of plans to build microfactories that can speed up the whole process for a global supply chain.

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George Yancopoulos (Regeneron)

Re­gen­eron co-founder George Yan­copou­los of­fers a com­bat­ive de­fense of the po­lice at a high school com­mence­ment. It didn’t go well

Typically, the commencement speech at Yorktown Central School District in Westchester — like most high schools — is an opportunity to encourage students to face the future with confidence and hope. Regeneron president and co-founder George Yancopoulos, though, went a different route.

In a fiery speech, the outspoken billionaire defended the police against the “prejudice and bias against law enforcement” that has erupted around the country in street protests from coast to coast. And for many who attended the commencement, Yancopoulos struck the wrong note at the wrong time, especially when he combatively challenged someone for interrupting his speech with a honk for “another act of cowardness.”

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No­vavax snags Ben Machielse for CMC and pro­motes a trio of staffers; Mar­ty Du­vall lands an­oth­er CEO post at On­copep­tides

Novavax has been making waves recently by securing a $384 million commitment from CEPI to cover R&D and manufacturing for its Covid-19 vaccine while also spending $167 million on a 150,000 square-foot facility. The Maryland biotech continues to shore up its leadership team as well, bringing in Ben Machielse as their EVP of CMC just a couple weeks after nabbing AstraZeneca vet Filip Dubrovsky as their new CMO. Machielse was president and CEO of Vtesse from 2014-17, and before that, he also spent more than 11 years at MedImmune and was EVP of operations for the back half of his tenure.

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Dan Gold, MEI Pharma CEO

De­vel­op­ment part­ners at MEI, Helsinn dump a high-risk PhI­II AML study af­ter con­clud­ing it would fail sur­vival goal

Four years after Switzerland’s Helsinn put $25 million of cash on the table for an upfront and near-term milestone to take MEI Pharma’s drug pracinostat into a long-running Phase III trial for acute myeloid leukemia, the partners are walking away from a clinical pileup.

The drug — an HDAC inhibitor — failed to pass muster during a futility analysis, as researchers concluded that pracinostat combined with azacitidine wasn’t going to outperform the control group in the pivotal.

Elias Zerhouni (Photo by Vincent Isore/IP3/Getty Images)

Elias Zer­houni dis­cuss­es ‘am­a­teur hour’ in DC, the de­struc­tion of in­fec­tious dis­ease R&D and how we need to prep for the next time

Elias Zerhouni favors blunt talk, and in a recent discussion with NPR, the ex-Sanofi R&D and ex-NIH chief had some tough points to make regarding the pandemic response.

Rather than interpret them, I thought it would be best to provide snippets straight from the interview.

On the Trump administration response:

It was basically amateur hour. There is no central concept of operations for preparedness, for pandemics, period. This administration doesn’t want to or has no concept of what it takes to protect the American people and the world because it is codependent. You can’t close your borders and say, “OK, we’re going to be safe.” You’re not going to be able to do that in this world. So it’s a lack of vision, basically just a lack of understanding, of what it takes to protect the American people.

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No­var­tis los­es biosim­i­lar ap­peal as court up­holds a 31-year mo­nop­oly by Am­gen's En­brel

A new court ruling has strengthened Amgen’s grip on the IP estate around Enbrel, keeping biosimilars of the autoimmune and inflammatory drug at bay until 2029.

Novartis, the patent challenger, isn’t throwing in the towel yet. In a statement noting the failed appeal, its generics division Sandoz noted its reviewing options, “including potential appeal to US Supreme Court.”

It’s been almost four years since the FDA approved Erelzi, Sandoz’s copycat version of Enbrel. While sales of the Pfizer-partnered drug in the US — the market Amgen is in charge of — have dipped slightly during that time, it remains a solid megablockbuster with 2019 revenue slightly above $5 billion.

Douglas Love, Annexon CEO (Annexon)

IPO bound? A Bay Area biotech grabs a mega-round on the road to a piv­otal neu­rode­gen­er­a­tion pro­gram

South San Francisco-based Annexon has added $100 million to its cash reserves, along with a new roster of marquee investors backing their play on the classical complement pathway involved in neurodegeneration. And that may well fit the profile for an IPO — though right now everything seems to be working on that score.

Eighteen months after Bain and their syndicate partners put up $75 million to fuel clinical work, Annexon is back at the trough. And this time they’re adding Redmile Group for the lead role, with supporting investments from these new arrivals: BlackRock, Deerfield Management Company, Eventide Asset Management, Farallon Capital Management, Janus Henderson Investors and Logos Capital.

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An ex­pe­ri­enced biotech is stitched to­geth­er from transpa­cif­ic parts, with 265 staffers and a fo­cus on ‘new bi­ol­o­gy’

Over the past few years, different teams at a pair of US-based biotechs and in labs in Japan have labored to piece together a group of cancer drug programs, sharing a single corporate umbrella with research colleagues in Japan. But now their far-flung operations have been knit together into a single unit, creating a pipeline with 10 cancer drug development programs — going from early-stage right into Phase III — and a host of discovery projects managed by a collective staff of some 265 people.

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Pfiz­er shares surge on pos­i­tive im­pact of their mR­NA Covid-19 vac­cine — part­nered with BioN­Tech — in an ear­ly-stage study

Pfizer and their partners at the mRNA specialist BioNTech have published the first glimpse of biomarker data from an early-stage study spotlighting the “robust immunogenicity” triggered by their Covid-19 vaccine, which is one of the leaders in the race to vanquish the global pandemic.

Researchers selected 45 healthy volunteers 18-55 years of age for the study. They were randomized to receive 2 doses, separated by 21 days, of 10 µg, 30 µg, or 100 µg of BNT162b1, “a lipid nanoparticle-formulated, nucleoside-modified, mRNA vaccine that encodes trimerized SARS-CoV-2 spike glycoprotein RBD.” Their responses were compared against the effect of a natural, presumably protective defense offered by a regular infection.

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