Ver­tex CEO to UK Prime Min­is­ter: You’re de­valu­ing lives, pass­ing up a great of­fer and threat­en­ing the coun­try’s biotech in­dus­try

Ver­tex has scored big in the US with its new drugs for cys­tic fi­bro­sis, but the biotech still has to slug it out for every inch of mar­ket ter­ri­to­ry in Eu­rope — where its price for Orkam­bi is of­ten seen as way too high for the val­ue it of­fers. And af­ter get­ting snubbed in the UK last week — again — af­ter a long-run­ning cam­paign to win a re­im­burse­ment deal from the NHS, CEO Jef­frey Lei­den has de­cid­ed to pub­licly chas­tise British Prime Min­is­ter There­sa May for the stand­off.

This lat­est blast oc­curred af­ter UK health of­fi­cials de­cid­ed last week that the price Ver­tex is de­mand­ing for Orkam­bi is “un­sup­port­able,” de­spite a pro-re­im­burse­ment cam­paign backed by thou­sands of pa­tients with the dis­ease.

That de­ci­sion, Lei­den tells May, demon­strates how the gov­ern­ment puts “a low­er val­ue on the life of a CF pa­tient than oth­er coun­tries around the world.” He as­serts that the UK’s de­ci­sion amounts to “shut­ting the door” on a new gen­er­a­tion of pre­ci­sion med­i­cines. And the UK, he adds, is pass­ing up a great of­fer.

We have pro­vid­ed the most in­no­v­a­tive of­fer in the world to the NHS, yet have seen no re­cep­tiv­i­ty from NHS Eng­land. In one of the most pros­per­ous coun­tries in the world, NHS Eng­land’s lev­el of in­ter­est in our of­fer rep­re­sents a lack of com­mit­ment to chil­dren and young peo­ple with this dev­as­tat­ing dis­ease….

For the UK to de­vel­op its sci­ence and in­no­va­tion ecosys­tem post-Brex­it, these pol­i­cy state­ments and plans must trans­late to im­proved pa­tient ac­cess to in­no­v­a­tive ther­a­pies, such as the new gen­er­a­tion of pre­ci­sion med­i­cines which work on dif­fer­ent geno­types of rare dis­eases. This re­quires ap­praisal process­es to keep pace with the sci­ence; cur­rent process­es have sig­nif­i­cant lim­i­ta­tions in how they cap­ture and val­ue the full med­ical, so­ci­etal, eco­nom­ic and in­no­va­tion ben­e­fits of such pre­ci­sion med­i­cines. With­out this, any fu­ture biotech in­vest­ment in the UK is at sig­nif­i­cant risk.

Ver­tex took the gloves off with re­cal­ci­trant Eu­ro­pean sin­gle-pay­er groups long ago. In France, the biotech re­cent­ly goad­ed the lo­cal pa­tient pop­u­la­tion with its de­ci­sion to scrap any plans to re­cruit French pa­tients in­to a study, in light of the gov­ern­ment’s re­fusal to cov­er their drug at what they felt was an ap­pro­pri­ate amount. 

We’ll see if lec­tur­ing the British prime min­is­ter works any bet­ter.

Ver­tex Let­ter to Prime Min­is­ter 6 Ju­ly 2018 by Am­ber on Scribd


Im­age: Jef­frey Lei­den. VER­TEX

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

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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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Gilead re­leas­es an­oth­er round of murky remde­sivir re­sults

A month after the NIH declared the first trial on remdesivir in Covid-19 a success, Gilead is out with new results on their antiviral. But although the study met one of its primary endpoints, the data are likely to only add to a growing debate over how effective the drug actually is.

In a Phase III trial, patients given a 5-day dose of remdesivir were 65% more likely to show “clinical improvement” compared to an arm given standard-of-care. The trial, though, gave little indication for whether the drug had an impact on key endpoints such as survival or time-to-recovery. And in a surprising twist, a 10-day dosing arm of remdesivir didn’t lead to a statistically significant improvement over standard of care.

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Ken Frazier, AP Images

Why Mer­ck wait­ed, and what they now bring to the Covid-19 fight

Nicholas Kartsonis had been running clinical infectious disease research at Merck for almost 2 years when, in mid-January, he got a new assignment: searching the pharma giant’s vast libraries for something that could treat the novel coronavirus.

The outbreak was barely two weeks old when Kartsonis and a few dozen others got to work, first in small teams and then in a larger task force that sucked in more and more parts of the sprawling company as Covid-19 infected more and more of the globe. By late February, the group began formally searching for vaccine and antiviral candidates to license. Still, while other companies jumped out to announce their programs and, eventually and sometimes controversially, early glimpses at human data, Merck remained silent. They made only a brief announcement about a data collection partnership in April and mentioned vaguely a vaccine and antiviral search in their April 28 earnings call.

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Mark Genovese (Stanford via Twitter)

Gilead woos fil­go­tinib clin­i­cal in­ves­ti­ga­tor from Stan­ford to lead the charge on NASH, in­flam­ma­to­ry dis­eases

With an FDA OK for the use of filgotinib in rheumatoid arthritis expected to drop any day now, Gilead has recruited a new leader from academia to lead its foray into inflammatory diseases.

Mark Genovese — a longtime Stanford professor and most recently the clinical chief in the division of immunology and rheumatology — was the principal investigator in FINCH 2, one of three studies that supported Gilead’s NDA filing. In his new role as SVP, inflammation, he will oversee the clinical development of the entire portfolio.

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Stephen Isaacs, Aduro president and CEO (Aduro)

Once a high fly­er, a stag­ger­ing Aduro is auc­tion­ing off most of the pipeline as CEO Stephen Isaacs hands off the shell to new own­ers

After a drumbeat of failure, setbacks and reorganizations over the last few years, Aduro CEO Stephen Isaacs is handing over his largely gutted-out shell of a public company to another biotech company and putting up some questionable assets in a going-out-of-business sale.

Isaacs —who forged a string of high-profile Big Pharma deals along the way — has wrapped a 13-year run at the biotech with one program for kidney disease going to the new owners at Chinook Therapeutics. A host of once-heralded assets like their STING agonist program partnered with Novartis (which dumped their work on ADU-S100 after looking over weak clinical results), the Lilly-allied cGAS-STING inhibitor program and the anti-CD27 program out-licensed to Merck will all be posted for auction under a strategic review process.

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Hill­house re­casts spot­light on Chi­na's biotech scene with $160M round for Shang­hai-based an­ti­body mak­er

Almost two years after first buying into Genor Biopharma’s pipeline of cancer and autoimmune therapies, Hillhouse Capital has led a $160 million cash injection to push the late-stage assets over the finish line while continuing to fund both internal R&D and dealmaking.

The Series B has landed right around the time Genor would have listed on the Hong Kong stock exchange, according to plans reported by Bloomberg late last year. Insiders had said that the company was looking to raise about $200 million.

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Fangliang Zhang (Imaginechina via AP Images)

The big mon­ey: Poised to make drug R&D his­to­ry, a Chi­na biotech un­veils uni­corn rac­ing am­bi­tions in a bid to raise $350M-plus on Nas­daq

Almost exactly three years after Shanghai-based Legend came out of nowhere to steal the show at ASCO with jaw-dropping data on their BCMA-targeted CAR-T for multiple myeloma, the little player with Big Pharma connections is taking a giant step toward making it big on Wall Street. And this time they want to seal the deal on a global rep after staking out a unicorn valuation in what’s turned out to be a bull market for biotech IPOs — in the middle of a pandemic.

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Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

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