Af­ter a slap­down on Orkam­bi price, Ver­tex is drop­ping French tri­al sites while warn­ing pa­tients on ac­cess

Ver­tex Phar­ma $VRTX is play­ing hard­ball with French drug reg­u­la­tors — and cys­tic fi­bro­sis pa­tients wait­ing for their next-gen triple are get­ting caught in the mid­dle.

Mired in a long-run­ning dis­pute over the re­im­burse­ment lev­el for their drug Orkam­bi, Ver­tex has de­cid­ed to shut out the tri­al sites it had se­lect­ed in France for the late-stage com­bi­na­tion stud­ies for its cys­tic fi­bro­sis drugs, in­clud­ing the ex­per­i­men­tal VX-659, which has raised hopes for a broad swath of pa­tients.

Ver­tex’s Orkam­bi was first ap­proved in France a lit­tle more than two years ago, trig­ger­ing ne­go­ti­a­tions over a re­im­burse­ment agree­ment as French of­fi­cials pur­sued a deep, 80% dis­count based on an old drug for CF — which Ver­tex has bucked against. Ver­tex has been sell­ing Orkam­bi in France at its list price as it ne­go­ti­ates the fi­nal price with the French gov­ern­ment. But of­fi­cials re­ject­ed that list price, say­ing that Orkam­bi pro­vid­ed on­ly a “mod­est” ben­e­fit — an at­ti­tude that has plagued Ver­tex’s ne­go­ti­a­tions in Eu­rope. The de­ci­sion puts Ver­tex on the hook for a re­im­burse­ment off the list price, and now it’s re­spond­ing by rais­ing a warn­ing about pa­tient ac­cess to their drugs and shut­ting out French tri­al sites.

The com­pa­ny of­fered this state­ment to End­points News in re­sponse to a query about the tri­al sites:

“Our goal is to cure CF and we have made sig­nif­i­cant ad­vances by dis­cov­er­ing and de­vel­op­ing the first med­i­cines to treat the un­der­ly­ing cause of this dis­ease. We want all pa­tients to have ac­cess to these and fu­ture med­i­cines we are de­vel­op­ing for those still wait­ing. We urge the French gov­ern­ment to join coun­tries like the U.S., Aus­tria, Ire­land, Ger­many and many oth­ers to pro­vide pa­tients per­ma­nent ac­cess to Orkam­bi.”

Bio­Cen­tu­ry got hold of an ex­cerpt of a let­ter Ver­tex sent to the labs which, they re­port­ed, stat­ed that:

(D)es­pite more than 18 months of “good faith” ne­go­ti­a­tions there has been no res­o­lu­tion on the re­im­burse­ment of Orkam­bi and that there­fore the path­way for CF pa­tients in France to gain ac­cess to the drug is “un­pre­dictable” and con­duct­ing the tri­als “would raise false hope.”

Bio­Cen­tu­ry got the ex­cerpt of the com­mu­ni­ca­tions from CF pa­tient groups in France, which are get­ting caught in the mid­dle of a fight. And it’s work­ing in Ver­tex’s fa­vor. Ac­cord­ing to the re­port they want Ver­tex to con­tin­ue ne­go­ti­a­tions and get the tri­al sites back in­to the late-stage stud­ies of the next-gen triples that promise to great­ly in­crease the num­ber of pa­tients that Ver­tex can reach.

A cou­ple of weeks ago Ver­tex picked VX-659 and VX-445 for two late-stage pro­grams, com­bin­ing each with teza­caftor and Ka­ly­de­co. Their drug ‘659 is slat­ed to be the first off the mark af­ter re­searchers demon­strat­ed that the high dose triple im­proved a mea­sure of lung func­tion by 13.3%.

Oxitec biologist releases genetically modified mosquitoes in Piracicaba, Brazil in 2016 [credit: Getty Images]

In­trex­on unit push­es back against claims its GM mos­qui­toes are mak­ing dis­ease-friend­ly mu­tants

When the hysteria of Zika transmission sprang into the American zeitgeist a few years ago, UK-based Oxitec was already field-testing its male Aedes aegypti mosquito, crafted to possess a gene engineered to obliterate its progeny long before maturation.

But when a group of independent scientists evaluated the impact of the release of these genetically-modified mosquitoes in a trial conducted by Oxitec in Brazil between 2013 and 2015, they found that some of the offspring had managed to survive — prompting them to speculate what impact the survivors could have on disease transmission and/or insecticide resistance.

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Pur­due threat­ens to walk away from set­tle­ment, asks to pay em­ploy­ees mil­lions in bonus­es

There are two updates on the lawsuit against Purdue Pharma over its role in fueling the opioid epidemic, as the Sackler family threatens to walk away from their pledge to pay out $3 billion if a bankruptcy judge does not stop outstanding state lawsuits against them. At the same time, the company has asked permission to pay millions in bonuses to select employees.

Purdue filed for chapter 11 bankruptcy this week as part of its signed resolution to over 2,000 lawsuits. The deal would see the Sackler family that owns Purdue give $3 billion from their personal wealth and the company turned into a trust committed to curbing and reversing overdoses.

As Nas­daq en­rolls the fi­nal batch of 2019 IPOs, how have the num­bers com­pared to past years?

IGM Biosciences’ upsized IPO haul, coming after SpringWorks’ sizable public debut, has revved up some momentum for the last rush of biotech IPOs in 2019.

With 39 new listings on the books and roughly two more months to go before winding down, Nasdaq’s head of healthcare listings Jordan Saxe sees the exchange marking 50 to 60 biopharma IPOs for the year.

“December 15 is usually the last possible day that companies will price,” he said, as companies get ready for business talks at the annual JP Morgan Healthcare Conference in January.

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Aerial view of Genentech's campus in South San Francisco [Credit: Getty]

Genen­tech sub­mits a plan to near­ly dou­ble its South San Fran­cis­co foot­print

The sign is still there, a quaint reminder of whitewashed concrete not 5 miles from Genentech’s sprawling, chrome-and-glass campus: South Francisco The Industrial City. 

The city keeps the old sign, first erected in 1923, as a tourist site and a kind of civic memento to the days it packed meat, milled lumber and burned enough steel to earn the moniker “Smokestack of the Peninsula.” But the real indication of where you are and how much has changed both in San Francisco and in the global economy since a couple researchers and investors rented out an empty warehouse 40 years ago comes in a far smaller blue sign, resembling a Rotary Club post, off the highway: South San Francisco, The Birthplace of Biotech.

A fa­vorite in Alex­ion’s C-suite is leav­ing, and some mighty sur­prised an­a­lysts aren’t the least bit hap­py about it

Analysts hate to lose a biotech CFO they’ve come to trust and admire — especially if they’re being blindsided by a surprise exit.

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David Grainger [file photo]

'Dis­con­nect the bas­tard­s' — one biotech's plan to break can­cer cell­s' uni­fied de­fens­es

Chemotherapy and radiotherapy are the current gladiators of cancer treatment, but they come with well-known limitations and side-effects. The emergence of immunotherapy — a ferocious new titan in oncologist’s toolbox — takes the brakes off the immune system to kill cancer cells with remarkable success in some cases, but the approach is not always effective. What makes certain forms of cancer so resilient? Scientists may have finally pieced together a tantalizing piece of the puzzle, and a new biotech is banking on a new approach to fill the gap.

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While No­var­tis ban­ish­es Zol­gens­ma scan­dal scars — Bio­gen goes on a Spin­raza 'of­fen­sive'

While Novartis painstakingly works to mop up the stench of the data manipulation scandal associated with its expensive gene therapy for spinal muscular atrophy (SMA) Zolgensma— rival Biogen is attempting to expand the use of its SMA therapy, Spinraza. 

The US drugmaker $BIIB secured US approval for Spinraza for use in the often fatal genetic disease in 2016. The approval covered a broad range of patients with infantile-onset (most likely to develop Type 1) SMA. 

Jason Kelly. Mike Blake/Reuters via Adobe

Eye­ing big ther­a­peu­tic push, Gink­go bags $290M to build a cell pro­gram­ming em­pire

Ginkgo Bioworks is on a roll. Days after publicizing a plan to nurture new startups via partnerships with accelerators Y Combinator and Petri, the Boston biotech says it has raised another $290 million for its cell programming platform to reach further and wider.

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UP­DAT­ED: Speak­er Nan­cy Pelosi to un­veil bill for fed­er­al­ly ne­go­ti­at­ed drug prices

After months of buzz from both sides of the aisle, Speaker Nancy Pelosi will today introduce her plan to allow the federal government to negotiate prices for 250 prescription drugs, setting up a showdown with a pharmaceutical industry working overtime to prevent it.

The need to limit drug prices is a rare point of agreement between President Trump and Democrats, although the president has yet to comment on the proposal and will likely face pressure to back a more conservative option or no bill at all. Republican Senator Chuck Grassley is reportedly lobbying his fellow party members on a more modest proposal he negotiated with Democratic Senator Ron Wyden in July.