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.”

Hal Barron and Rick Klausner (GSK, Lyell)

Ex­clu­sive: GSK’s Hal Bar­ron al­lies with Rick Klaus­ner’s $600M cell ther­a­py start­up, look­ing to break new ground blitz­ing sol­id tu­mors

LONDON — Chances are, you’ve heard little or nothing about Rick Klausner’s startup Lyell. But that ends now.

Klausner, the former head of the National Cancer Institute, former executive director for global health at the Gates Foundation, co-founder at Juno and one of the leaders in the booming cell therapy field, has brought together one of the most prominent teams of scientists tackling cell therapy 2.0 — highlighted by a quest to bridge a daunting tech gap that separates some profound advances in blood cancers with solid tumors. And today he’s officially adding Hal Barron and GlaxoSmithKline as a major league collaborator which is pitching in a large portion of the $600 million he’s raised in the past year to make that vision a reality.

“We’ve being staying stealth,” Klausner tells me, then adding with a chuckle: “and going back to stealth after this.”

“Cell therapy has a lot of challenges,” notes Barron, the R&D chief at GSK, ticking off the resistance put up by solid tumors to cell therapies, the vein-to-vein time involved in taking immune cells out of patients, engineering them to attack cancer cells, and getting them back in, and more. “Over the years Rick and I talked about how it would be wonderful to take that on as a mission.”

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First place fin­ish: Eli Lil­ly just moved to fran­chise leader with their sec­ond mi­graine drug OK in 1 year

In a rare twist for Eli Lilly’s historically slow-moving R&D group, the pharma giant has seized bragging rights to a first-in-class new drug approval. And all signs point to an aggressive marketing followup as they look to outclass some major franchise rivals hobbled by internal dissension.

The FDA came through with an OK for lasmiditan on Friday evening, branding it as Reyvow and lining it up — once a substance classification comes through from the DEA — for a major market release. The oral drug binds to 5-HT1F receptors and is designed to stop an acute migraine after it starts. That makes it a complementary therapy to their CGRP drug Emgality, which has a statistically significant impact on preventing attacks.

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Court green-lights Clo­vis case af­ter de­tail­ing ev­i­dence the board ‘ig­nored red flags’ on false safe­ty and ef­fi­ca­cy da­ta

Clovis investors have cleared a major hurdle in their long-running case against the board of directors, with a Delaware court making a rare finding that they had a strong enough case against the board to proceed with the action.

In a detailed ruling at the beginning of the month that’s been getting careful scrutiny at firms specializing in biotech and corporate governance, the Delaware Court of Chancery found that the attorneys for the investors had made a careful case that the board — a collection of experts that includes high-profile biotech entrepreneurs, a Harvard professor and well-known investigator as well as Clovis CEO Patrick Mahaffy — repeatedly ignored obvious warnings that Mahaffy’s executive crew was touting inflated, unconfirmed data for their big drug Roci. Serious safety issues were also reportedly overlooked while the company continued a fundraising campaign that brought in more than a half-billion dollars. And that leaves the board open to claims related to their role in the fiasco.

The bottom line:

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Allogene HQ Open House on September 17, 2019 in South San Francisco. (Jeff Rumans, Endpoints News)

The next 10 years: Where is biotech head­ed?

The last 10 years have seen a revolution in drug development. Timelines have shortened, particularly in oncology. Regulators have opened up. Investment has skyrocketed. China became a player. Biotechs have multiplied as gene and cell therapy has exploded — offering major new advances in the way diseases are treated, and sometimes cured.

So where are we headed from here? I journeyed out to San Francisco in September to discuss the answer to that question at Allogene’s open house. If the last 10 years have been an eye-opener, what does the next decade hold in store?

George Scangos / Credit: Cornell University

ARCH, Soft­Bank-backed Vir Biotech­nol­o­gy un­der­whelms with $143 mil­lion IPO

George Scangos went back to Wall Street, and came back 700 million pennies short.

Scangos’ vaunted startup Vir Biotechnology raised $143 million in an IPO they hoped would earn $150 million. Shares were priced at $20, the low-end of the $20-$22 target.

Launched with backing from ARCH Venture’s Robert Nelsen, Masayoshi Son’s SoftBank Vision Fund, and the Bill & Melinda Gates Foundation, the infectious disease startup was one of a new wave of well-resourced biotechs that emerged with deep enough coffers to pursue a full R&D line rather than slowly build their case by picking off a single lead program.

Ex-Ab­b­Vie part­ner Prin­cip­ia posts en­cour­ag­ing PhII re­sults for its BTK-in­hibitor

Months after their breakup with high-profile partner AbbVie, Principia announced positive preliminary results from the second half of a Phase II trial on their lead drug.

The San Francisco biotech announced data from part B of its Phase II open-label trial testing the BTK inhibitor PRN1008 on patients with pemphigus vulgaris, a rare autoimmune disease affecting the skin and mucous membranes. Of 15 enrolled patients, 6 achieved complete responses and 4 remain on the therapy.

Roche vice-chair: Let's re­pair the dam­age that short-term prof­it dri­ve has done to the plan­et

In his latest push for environmental advocacy, the vice chairman of Roche’s board of directors has told a group of business executives that “short-term profit maximization has destroyed the planet, environmentally and socially.”

Andre Hoffman
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In re­ver­sal, NICE backs Rubra­ca af­ter Clo­vis agrees to a price cut

NICE has changed its mind, agreeing to cautiously endorse Clovis Oncology’s Rubraca after the drugmaker agreed to cut its price — about two months after the UK cost-effectiveness agency’s initial rejection.

Rubraca, known chemically as rucaparib, is approved for use in the EU as monotherapy for the maintenance treatment of adult patients with high-grade epithelial ovarian, fallopian tube, or primary peritoneal cancer who have relapsed after platinum-based chemotherapy.

Bill Gates backs Gink­go Biowork­s' $350M raise to fu­el the buzzy syn­thet­ic bi­ol­o­gy 'rev­o­lu­tion'

If you want to understand Ginkgo Bioworks, the name should suffice: Bioworks, a spin off “ironworks,” that old industrial linchpin devoted to leveraging scale as a wellspring for vast new industries capable of remaking society. Ginkgo wants to be the ironworks for the revolution it’s heralded with as much fanfare as they can, playing off of one of the buzziest technologies in biotech.

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