Da­ta sug­gest US, UK uni­ver­si­ties fall woe­ful­ly short on re­port­ing clin­i­cal tri­al re­sults

Clin­i­cal tri­al da­ta are used by pa­tients, doc­tors and pol­i­cy­mak­ers to make in­formed choic­es about the ben­e­fits and safe­ty of in­ter­ven­tions — while the meth­ods and re­sults of all tri­als are cru­cial to the pace and di­rec­tion of sci­en­tif­ic progress. How­ev­er, there is a large body of ev­i­dence that sug­gests that com­plet­ed clin­i­cal tri­als are com­mon­ly left un­re­port­ed, and ed­u­ca­tion­al in­sti­tu­tions in the Unit­ed States and the Unit­ed King­dom — ar­guably the two biggest re­gions that breed the bulk of med­ical in­no­va­tion — have emerged as one of the key cul­prits guilty of these vi­o­la­tions.

In the Unit­ed States, Con­gress passed a law in 2007 re­quir­ing tri­al spon­sors — in­clud­ing uni­ver­si­ties — to post the re­sults of cer­tain clin­i­cal tri­als on clin­i­cal­tri­als.gov with­in a year of tri­al com­ple­tion, and a decade lat­er in Jan­u­ary 2017 the rule was fi­nal­ized. Since 2017, 40 lead­ing US uni­ver­si­ties should have post­ed the re­sults of 450 clin­i­cal tri­als — but over a third (31%) of those re­sults are miss­ing, ac­cord­ing to an analy­sis by Uni­ver­si­ties Al­lied for Es­sen­tial Med­i­cines (UAEM) in part­ner­ship with non-prof­it re­search ad­vo­ca­cy group TranspariMED.

The vi­o­la­tors in­clude some of the most ac­tive tri­al spon­sors: For ex­am­ple the MD An­der­son Can­cer Cen­ter, which has on­ly re­port­ed 77% of due tri­als, Mayo Clin­ic (42%), UC San Fran­cis­co (37%), New York Uni­ver­si­ty (21%), and Co­lum­bia Uni­ver­si­ty (17%).

A sum­ma­ry of re­sults by per­cent­age of each uni­ver­si­ty eval­u­at­ed can be seen be­low:

Source: UAEM, TranspariMED

Click on the im­age to see the full-sized ver­sion

Over­all, 140 clin­i­cal tri­als are still miss­ing re­sults and five uni­ver­si­ties are re­spon­si­ble for half of the un­re­port­ed tri­als: Uni­ver­si­ty of Cal­i­for­nia San Fran­cis­co (17 tri­als with­out re­sults), Co­lum­bia (15 tri­als), Mayo Clin­ic (13), MD An­der­son Can­cer Cen­ter (12) and Chica­go (8), ac­cord­ing to the re­port.

These trans­paren­cy vi­o­la­tions are con­cern­ing con­sid­er­ing at least half of the valu­able med­i­cines that ex­ist to­day were orig­i­nal­ly de­vel­oped in uni­ver­si­ty labs with tax­pay­er fund­ing, in­clud­ing al­most all vac­cines, many HIV and tu­ber­cu­lo­sis drugs, and even in­sulin, the re­port not­ed. Be­tween 2010 and 2016, every sin­gle one of the 210 FDA-ap­proved med­i­cines can be traced back to fund­ing from the NIH, ac­cord­ing to a study pub­lished in the of­fi­cial jour­nal of the Na­tion­al Acad­e­my of Sci­ences.

Mean­while, these trans­paren­cy trans­gres­sions are echoed in the UK. Reg­u­la­tions in Eu­rope are sim­i­lar. Any tri­al of of any med­i­c­i­nal prod­uct con­duct­ed since 2004 in an EU coun­try has al­ready been re­quired to reg­is­ter on the Eu­ro­pean Union Clin­i­cal Tri­als Reg­is­ter (EU­C­TR) and since 2012, spon­sors must en­sure that all reg­is­tered tri­als since 2004 dis­close their re­sults to the EMA with­in 12 months of tri­al com­ple­tion. But the de­lays to the EMA’s soft­ware plat­form pushed the fi­nal date for re­sults post­ing by spon­sors to late De­cem­ber 2016.

Ben Goldacre

In a BMJ study pub­lished in 2018 — led by Ben Goldacre, a best-sell­ing au­thor, med­ical doc­tor and re­searcher who fo­cus­es on un­pack­ing the mis­use of sci­ence and sta­tis­tics in his books Bad Sci­ence and Bad Phar­ma — it was found that in Eu­rope, of the 7274 tri­als where re­sults were due, 49.5% re­port­ed re­sults. Tri­als with a com­mer­cial spon­sor (such as a drug de­vel­op­er) were sub­stan­tial­ly more like­ly to post re­sults than those with a non-com­mer­cial spon­sor (68.1% v 11.0%), the analy­sis sug­gest­ed.

Out of his labs at the Uni­ver­si­ty of Ox­ford, Goldacre set up an EU Tri­al­sTrack­er to con­tin­u­ous­ly mon­i­tor the re­port­ing of tri­als. As of 10 Jan­u­ary 2019, Goldacre and his team have iden­ti­fied 8,062 reg­is­tered tri­als that are ‘un­am­bigu­ous­ly’ due to re­port re­sults — but re­sults on just over half  (53.6%) have been post­ed to the reg­istry. The da­ta, which sug­gest­ed that UK uni­ver­si­ties were less re­li­able than drug de­vel­op­ers, sparked the in­ter­est of House of Com­mons Sci­ence and Tech­nol­o­gy Com­mit­tee. UK uni­ver­si­ties could be brought in front of the com­mit­tee if they fail to im­prove their track record, and the com­mit­tee will ask them to ex­plain them­selves in a fol­low-up ev­i­dence ses­sion if im­prove­ments are not made.

How to cap­i­talise on a lean launch

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

Turn­ing the cor­ner on treat­ing the root cause of sick­le cell dis­ease

Early in my career, as a medical resident, I saw first-hand the enormous challenges faced by children and adults with sickle cell disease (SCD), a genetic blood disorder that historically has lacked adequate treatment options. People living with this life-long disease are mainly those with ancestors from sub-Saharan Africa, as well as people of Hispanic, South Asian, Southern European and Middle Eastern descent. These patients suffer from devastating physical symptoms, including progressive, eventually fatal, organ damage and excruciating pain. In addition, they encounter emotional, mental and social burdens – non-physical aspects of living with SCD that also take a serious toll on patients and their caregivers.

Rev­o­lu­tion Med shoots for $100M+ IPO — and di­vulges some se­crets about that Warp Dri­ve buy­out

Biotech investors who like to wager on the race to the front of the KRAS market now have a new team to consider.

Revolution Medicines, which extended its reach on RAS with a deal to acquire Warp Drive Bio about 18 months ago, filed their S-1 in search of $100 million-plus. And they gave up a few secrets in the process.

The main clinical claim to fame that Revolution has centers on the SHP2 inhibitor RMC-4630, partnered with Sanofi back in the summer of 2018 — just after John Reed was named the incoming R&D chief. We already knew that the pharma giant handed over $50 million in cash plus a commitment of hundreds of millions more to align itself with Revolution as it makes a fresh foray into oncology. Now we know that Sanofi is also footing 80% of Revolution’s R&D bill on the program, while setting up a smorgasbord of $235 million in development milestones and $285 million in commercial bonuses.

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Olivier Brandicourt, AP Images

#JPM20 ex­clu­sive: Olivi­er Brandi­court fol­lows the Big Phar­ma CEO path to pri­vate eq­ui­ty, join­ing Black­stone ahead of a mam­moth fund de­but

Nick Galakatos Blackstone

Seven months after Olivier Brandicourt’s surprise “early retirement” from Sanofi, he’s back in the game, this time taking meetings at JP Morgan to discuss his new role at Blackstone, where he’s quietly begun work with Nick Galakatos and the life sciences crew.

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Video Re­play: End­points at #JPM20 — news­mak­ers on deal­mak­ing, pric­ing and man­u­fac­tur­ing

On Monday, we held our fourth annual #JPM event — and the team hit a key milestone that I’d like to share with the entire Endpoints News audience: We live-streamed the conversation and had nearly triple the number of executives watching online than we had in the sold-out crowd of 320.

For a media company on a mission to connect the biopharma world in bigger and better ways, we’re proud of how we were able to extend the reach of our franchise event. Paid subscribers were given access to the stream in real time, and now, two days later, we’re opening it up to everyone in this post.

Endpoints@JPM: (left to right) Steve Pearson, Nick Leschly, Bari Talente, Stephen Ubl, John Carroll

#JPM20: 'The NPV is al­ways wrong.' Take­da preps an­oth­er spin­out — this time on psych

Editor’s Note: Endpoints News is reporting live from #JPM20 after kicking things off with an action-packed event, which you can replay here. What follows is a stream of tidbits we have collected while wandering around Union Square in San Francisco. Check back in throughout the week for updates by John Carroll and Jason Mast.

SAN FRANCISCO — A year ago Takeda CEO Christophe Weber and R&D chief Andy Plump arrived at JP Morgan right on the heels of closing their big Shire buyout. Now they’re back after shaking up the portfolio, boosting R&D spending by about 50% to $4.5 billion and adjusting the pipeline — a task which isn’t quite finished yet.

Nick Leschly at Endpoints News' panel at the 2020 JP Morgan Healthcare Conference. Credit: Jeff Rumans

At #JPM20, two CEOs, two rad­i­cal­ly dif­fer­ent ther­a­pies, and a fight to chase down sick­le cell

SAN FRANCISCO – Few CEOs tell a story better than bluebird’s Nick Leschly.

He cuts a Jeff Bezos figure on stage at the Colonial Room, the JP Morgan presentation hall for A-list biotechs: lean and bald, fast-talking and vest-wearing. He explains in simple language, apologizing when he has to brush on the data. It helps that he has a good story to tell.

“We treated them one time,” Leschly tells a packed crowd, gesturing to the slide behind him. “Look what happened.”

The slide shows 9 horizontal bars studded with diamonds. Each bar, he explained, represented a sickle cell patient, and each diamond represented a severe medical event, such as a pain crisis. The diamonds stud one side – before the therapy – and vanish on the other, afterward.

“A 99% reduction in these events — this is a functional cure for sickle cell disease,” Leschly says. “This is unprecedented data.”

Upstairs and an hour later, Ted Love stands before a narrow conference room in his suit and polka-dot tie. Love, the CEO of Global Blood Therapeutics, is a 60-year-old physician. His voice trails off at the end of sentences, and the story he tells is less compelling. There are no cured patients.

“This is the first drug that addresses the root cause of sickle cell disease,” Love says, speaking in front of a slide showing a white pill bottle for GBT’s new drug Oxbryta. “Right in the label, it says that this drug inhibits polymerization.”

In the 60 years after scientists discovered the cause of sickle cell, almost no treatments emerged, even as the condition debilitated hundreds of thousands of Americans, most of them black or Hispanic. But the last few years have seen a resurgence of interest as new technologies have made the disease seem newly beatable.

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Neon Ther­a­peu­tics makes one last re­treat, sell­ing it­self cheap in a bar­gain base­ment M&A deal

Crushed by weak data for what had been their lead drug, Neon Therapeutics is being bought for parts this morning.

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Mark Pruzanski

#JPM20: Af­ter a year of NASH col­laps­es, all eyes on two biotechs

SAN FRANCISCO – It’s not quite Dewey defeats Truman, but Goldman Sachs calling 2019 “The Year of NASH” may well go down in the annals of worst biotech predictions.

Goldman Sachs slapped the label on weeks before 2019’s JP Morgan conference, projecting that long-discussed treatments for the obesity-driven condition suspected to lurk in millions of Americans would begin to bear fruit and investors would move accordingly. That did not quite happen.

“If you look at 2019, it was just a string of disappointing news,” Pascal Prigent, CEO of NASH-focused biotech Genfit, told Endpoints News in an interview.

The Year of NASH, or nonalcoholic steatohepatitis, became a year of NASH failures. Gilead failed two large Phase III trials. CymaBay went from a $1 billion company to a $100 million company after they found their drug was killing patients’ liver cells. Cirius withdrew an $86 million IPO bid after a disastrous readout. Industry-wide, there were few acqusitions in a market often projected to be worth $35 billion.

Gilead, after dominating the NASH discussion at the 2019 JPM, gave one quick mention to the program in their 2020 presentation before pivoting to other drugs.

“As promising as some of the mechanisms looked in earlier stages, when push comes to shove in large study settings, they just haven’t proven out,” Mark Pruzanski, CEO of the NASH-focused biotech Intercept, told Endpoints in an interview.

As biotech turns from 2019, the failures have refocused eyes away from Gilead and back toward two startups, both facing key events in the coming months: Intercept, which first alerted investors to NASH at JPM 2014, and the France-based Genfit.

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