Ver­tex deal for Scot­land — no deal for Eng­land

Cys­tic fi­bro­sis (CF) drug mak­er Ver­tex Phar­ma­ceu­ti­cals — which is still locked in ne­go­ti­a­tion with NHS Eng­land to en­dorse the use of its med­i­cines — has suc­cess­ful­ly ne­go­ti­at­ed a deal with Scot­tish au­thor­i­ties.

A month ago, the Scot­tish Med­i­cines Con­sor­tium spurned two of the com­pa­ny’s med­i­cines — Orkam­bi and Symke­vi — cit­ing un­cer­tain­ty over their long-term ef­fi­ca­cy in re­la­tion to their cost.

Each drug car­ries a list price of more than £100,000 per pa­tient per year. The UK has more than 10,400 cys­tic fi­bro­sis pa­tients – the largest CF pop­u­la­tion out­side the US, ac­cord­ing to the Cys­tic Fi­bro­sis Foun­da­tion.

But on Thurs­day, Ver­tex an­nounced a deal had been reached with Scot­land. The price ne­go­ti­at­ed for the treat­ments is con­fi­den­tial, but as part of the five-year agree­ment Ver­tex will col­lect re­al-world ev­i­dence on the drugs that sup­port any fu­ture sub­mis­sions, the com­pa­ny said, adding that about 400 out of the 900 CF pa­tients in Scot­land car­ry the mu­ta­tions ad­dress­able by Orkam­bi or Symke­vi.

David Rams­den Cys­tic Fi­bro­sis Trust

“We cel­e­brate the news in Scot­land to­day, but our cam­paign must con­tin­ue to fo­cus on Ver­tex and all par­ties in Eng­land, Wales and North­ern Ire­land. Those in need of the drugs have al­ready wait­ed too long and we must en­sure that thou­sands more peo­ple are not sub­ject­ed to a post­code lot­tery. Scot­land’s suc­cess must now be repli­cat­ed across the UK with­out fur­ther dam­ag­ing de­lay,” Cys­tic Fi­bro­sis Trust chief David Rams­den said in a state­ment.

The cys­tic fi­bro­sis drugs made by Ver­tex $VRTX are the first treat­ments that ad­dress the un­der­ly­ing ge­net­ic caus­es of cys­tic fi­bro­sis, which is char­ac­ter­ized by a thick sticky mu­cus in the lungs, di­ges­tive sys­tem and oth­er or­gans that re­duces life ex­pectan­cy. In its sec­ond-quar­ter re­sults — pub­lished in late Ju­ly — Ver­tex’s tri­fec­ta of CF med­i­cines com­bined gen­er­at­ed near­ly $1 bil­lion in sales, up 25% from the pre­ced­ing quar­ter. The FDA is al­so re­view­ing the Boston biotech’s three-drug cock­tail for CF, which is ex­pect­ed to treat 90% of CF pa­tients.

Jeff Lei­den Ver­tex

Ver­tex has been hag­gling with UK’s cost-ef­fec­tive­ness watch­dog NICE, which has re­fused to al­low the drug in­to Eng­land’s NHS un­til Ver­tex of­fers it a dis­count on the treat­ment’s price tag that would com­pel the agency to look fa­vor­ably up­on its cost-ef­fec­tive­ness. Ear­li­er this year, NHS Eng­land of­fered Ver­tex £500 mil­lion over five years and £l bil­lion over the next 10 years to ac­cess the com­pa­ny’s med­i­cines — but the US drug­mak­er re­ject­ed the of­fer. Ne­go­ti­a­tions are still on­go­ing.

To the hor­ror of UK cys­tic fi­bro­sis pa­tients, a Ver­tex ex­ec­u­tive dis­closed that last year close to 8,000 packs (each con­tain­ing a 28-day sup­ply) of the com­pa­ny’s treat­ment, Orkam­bi, were de­stroyed af­ter cross­ing their ex­piry date. In a stand­off with UK par­lia­ment in March, Ver­tex chief Jeff Lei­den stood his ground, de­spite be­ing chas­tised by a pletho­ra of MPs for Ver­tex’s pric­ing strat­e­gy, busi­ness mod­el and ethics.

So­cial im­age: Ver­tex

De­vel­op­ment of the Next Gen­er­a­tion NKG2D CAR T-cell Man­u­fac­tur­ing Process

Celyad’s view on developing and delivering a CAR T-cell therapy with multi-tumor specificity combined with cell manufacturing success
Overview
Transitioning potential therapeutic assets from academia into the commercial environment is an exercise that is largely underappreciated by stakeholders, except for drug developers themselves. The promise of preclinical or early clinical results drives enthusiasm, but the pragmatic delivery of a therapy outside of small, local testing is most often a major challenge for drug developers especially, including among other things, the manufacturing challenges that surround the production of just-in-time and personalized autologous cell therapy products.

Roger Perlmutter, Merck

#ASH19: Here’s why Mer­ck is pay­ing $2.7B to­day to grab Ar­Qule and its next-gen BTK drug, lin­ing up Eli Lil­ly ri­val­ry

Just a few months after making a splash at the European Hematology Association scientific confab with an early snapshot of positive data for their BTK inhibitor ARQ 531, ArQule has won a $2.7 billion buyout deal from Merck.

Merck is scooping up a next-gen BTK drug — which is making a splash at ASH today — from ArQule in an M&A pact set at $20 a share $ARQL. That’s more than twice Friday’s $9.66 close. And Merck R&D chief Roger Perlmutter heralded a deal that nets “multiple clinical-stage oral kinase inhibitors.”

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Game on: Re­gen­eron's BC­MA bis­pe­cif­ic makes clin­i­cal da­ta de­but, kick­ing off mul­ti­ple myelo­ma matchup with Bris­tol-My­ers

As J&J attempts to jostle past Bristol-Myers Squibb and bluebird for a landmark approval of its anti-BCMA CAR-T — and while GlaxoSmithKline maps a quick path to the FDA riding on its own BCMA-targeting antibody-drug conjugates — the bispecifics are arriving on the scene to stake a claim for a market that could cross $10 billion per year.

The main rivalry in multiple myeloma is shaping up to be one between Regeneron and Bristol-Myers, which picked up a bispecific antibody to BCMA through its recently closed $74 billion takeover of Celgene. Both presented promising first-in-human data at the ASH 2019 meeting.

FDA lifts hold on Abeon­a's but­ter­fly dis­ease ther­a­py, paving way for piv­otal study

It’s been a difficult few years for gene and cell therapy startup Abeona Therapeutics. Its newly crowned chief Carsten Thiel was forced out last year following accusations of unspecified “personal misconduct,” and this September, the FDA imposed a clinical hold on its therapy for a form of “butterfly” disease. But things are beginning to perk up. On Monday, the company said the regulator had lifted its hold and the experimental therapy is now set to be evaluated in a late-stage study.

Paul Hudson. Sanofi

New Sanofi CEO Hud­son adds next-gen can­cer drug tech to the R&D quest, buy­ing Syn­thorx for $2.5B

When Paul Hudson lays out his R&D vision for Sanofi tomorrow, he will have a new slate of interleukin therapies and a synthetic biology platform to boast about.

The French pharma giant announced early Monday that it is snagging San Diego biotech Synthorx in a $2.5 billion deal. That marks an affordable bolt-on for Sanofi but a considerable return for Synthorx backers, including Avalon, RA Capital and OrbiMed: At $68 per share, the price represents a 172% premium to Friday’s closing.

Synthorx’s take on alternative IL-2 drugs for both cancer and autoimmune disorders — enabled by a synthetic DNA base pair pioneered by Scripps professor Floyd Romesberg — “fits perfectly” with the kind of innovation that he wants at Sanofi, Hudson said.

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Roche faces an­oth­er de­lay in strug­gle to nav­i­gate Spark deal past reg­u­la­tors — but this one is very short

Roche today issued the latest in a long string of delays of its $4.3 billion buyout of Philadelphia-based Spark Therapeutics. The delay comes as little surprise — it is their 10th in as many months — as their most recent delay was scheduled to expire before a key regulatory deadline.

But it is notable for its length: 6 days.

Previous extensions had moved the goalposts by about 3 weeks to a month, with the latest on November 22 expiring tomorrow. The new delay sets a deadline for next Monday, December 16, the same day by which the UK Competition and Markets Authority has to give its initial ruling on the deal. And they already reportedly have lined up an OK from the FTC staff – although that’s only one level of a multi-step process.

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KalVis­ta's di­a­bet­ic mac­u­lar ede­ma da­ta falls short — will Mer­ck walk away?

Merck’s 2017 bet on KalVista Pharmaceuticals may have soured, after the UK/US-based biotech’s lead drug failed a mid-stage study in patients with diabetic macular edema (DME).

Two doses of the intravitreal injection, KVD001, were tested against a placebo in a 129-patient trial. Patients who continued to experience significant inflammation and diminished visual acuity, despite anti-VEGF therapy, were recruited to the trial. Typically patients with DME — the most frequent cause of vision loss related to diabetes — are treated with anti-VEGF therapies such as Regeneron’s flagship Eylea or Roche’s Avastin and Lucentis.

UP­DAT­ED: Ob­sE­va makes case for best-in-class hor­mone sup­pres­sive ther­a­py in pos­i­tive uter­ine fi­broid study

About a month after the Swiss biotech disclosed a failed late-stage study in its IVF program, ObsEva on Monday unveiled positive pivotal data on its experimental treatment for heavy menstrual bleeding triggered by uterine fibroids.

ObsEva in-licensed the drug, linzagolix, from Japan’s Kissei Pharmaceutical in 2015. Two doses of the drug (100 mg and 200 mg) were tested against a placebo in the 535-patient Phase III study, dubbed PRIMROSE 2, in patients who were both on and off hormonal add-back therapy (ABT).

Ear­ly-stage can­cer biotech nails $85M C round; Flem­ming Orn­skov's Gal­der­ma scores 'break­through' sta­tus

→ Zentalis Pharmaceuticals just nabbed an $85 million round from a syndicate that includes Matrix Capital, Viking Global Investors, Redmile Group, Farallon Capital, Perceptive Advisors, Surveyor Capital and Eventide Asset Management. Their lead drug is ZN-c5, which is currently in Phase I/II trials. The biotech describes that drug as a “potential best-in-class oral Selective Estrogen Receptor Degrader for estrogen receptor-positive, HER2-negative (ER+/ HER2-) breast cancer.”