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%.

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

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FDA de­lays de­ci­sion on No­var­tis’ po­ten­tial block­buster MS drug, wip­ing away pri­or­i­ty re­view

So much for a speedy review.

In February, Novartis announced that an application for their much-touted multiple sclerosis drug ofatumumab had been accepted and, with the drug company cashing in on one of their priority review vouchers, the agency was due for a decision by June.

But with June less than 48 hours old, Novartis announced the agency has extended their review, pushing back the timeline for approval or rejection to September. The Swiss pharma filed the application in December, meaning their new schedule will be nearly in line with the standard 10-month window period had they not used the priority voucher.

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GSK presents case to ex­pand use of its lu­pus drug in pa­tients with kid­ney dis­ease, but the field is evolv­ing. How long will the mo­nop­oly last?

In 2011, GlaxoSmithKline’s Benlysta became the first biologic to win approval for lupus patients. Nine years on, the British drugmaker has unveiled detailed positive results from a study testing the drug in lupus patients with associated kidney disease — a post-marketing requirement from the initial FDA approval.

Lupus is a drug developer’s nightmare. In the last six decades, there has been just one FDA approval (Benlysta), with the field resembling a graveyard in recent years with a string of failures including UCB and Biogen’s late-stage flop, as well as defeats in Xencor and Sanofi’s programs. One of the main reasons the success has eluded researchers is because lupus, akin to cancer, is not just one disease — it really is a disease of many diseases, noted Al Roy, executive director of Lupus Clinical Investigators Network, an initiative of New York-based Lupus Research Alliance that claims it is the world’s leading private funder of lupus research, in an interview.

UP­DAT­ED: Es­ti­mat­ing a US price tag of $5K per course, remde­sivir is set to make bil­lions for Gilead, says key an­a­lyst

Data on remdesivir — the first drug shown to benefit Covid-19 patients in a randomized, controlled trial setting — may be murky, but its maker Gilead could reap billions from the sales of the failed Ebola therapy, according to an estimate by a prominent Wall Street analyst. However, the forecast, which is based on a $5,000-per-course US price tag, triggered the ire of one top drug price expert.

Leen Kawas, Athira CEO (Athira)

Can a small biotech suc­cess­ful­ly tack­le an Ever­est climb like Alzheimer’s? Athi­ra has $85M and some in­flu­en­tial back­ers ready to give it a shot

There haven’t been a lot of big venture rounds for biotech companies looking to run a Phase II study in Alzheimer’s.

The field has been a disaster over the past decade. Amyloid didn’t pan out as a target — going down in a litany of Phase III failures — and is now making its last stand at Biogen. Tau is a comer, but when you look around and all you see is destruction, the idea of backing a startup trying to find complex cocktails to swing the course of this devilishly complicated memory-wasting disease would daunt the pluckiest investors.

Covid-19 roundup: Mod­er­na read­ies to en­ter PhI­II in Ju­ly, As­traZeneca not far be­hind; EU ready to ne­go­ti­ate vac­cine ac­cess with $2.7B fund

Moderna may soon add another first to the Covid-19 vaccine race.

In March, the mRNA biotech was the first company to put a Covid-19 vaccine into humans. Next month, they may become the first company to put their vaccine into the large, late-stage trials that are needed to prove whether the vaccine is effective.

In an interview with JAMA editor Howard Bauchner, NIAID chief Anthony Fauci said that a 30,000-person, Phase III trial for Moderna’s vaccine could start in July. The news comes a week after Moderna began a Phase II study that will enroll several hundred people.

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José Basel­ga finds promise in new class of RNA-mod­i­fy­ing can­cer tar­gets, lock­ing in 3 pre­clin­i­cal pro­grams with $55M

Having dived early into some of the RNA breakthroughs of the last decades — betting on Moderna’s mRNA tech and teaming up with Silence on the siRNA front — AstraZeneca is jumping into a new arena: going after proteins that modify RNA.

Their partner of choice is Accent Therapeutics, which is receiving $55 million in upfront payment to steer a selected preclinical program through to the end of Phase I. After AstraZeneca takes over, the Lexington, MA-based startup has the option to co-develop and co-commercialize in the US — and collect up to $1.1 billion in milestones in the long run. The deal also covers two other potential drug candidates.

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Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

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