Predict your next investment

Maastricht University company logo
CONSUMER PRODUCTS & SERVICES | Education & Training (non-internet/mobile) / Colleges & Universities
maastrichtuniversity.nl

See what CB Insights has to offer

Founded Year

1976

About Maastricht University

Maastricht University (UM) is an educational institute that offers graduate degree programs. It is located in Maastricht, Netherlands.

Maastricht University Headquarter Location

Minderbroedersberg 4-6

Maastricht, 6211,

Netherlands

Latest Maastricht University News

Biogen thinks a CMS-mandated trial is a death sentence. Others see a chance to change the future of Alzheimer’s trials

Jan 19, 2022

Jason Mast Editor Bio­gen has gone full blitz since Medicare an­nounced it would on­ly cov­er its new Alzheimer’s drug when used in clin­i­cal tri­als, ac­cus­ing the agency of dis­crim­i­nat­ing against Alzheimer’s pa­tients and try­ing to get physi­cians to change reg­u­la­tors’ minds. Far less at­ten­tion, though, has gone to what a Medicare-fund­ed clin­i­cal tri­al would ac­tu­al­ly look like. Bio­gen has op­er­at­ed as if it would be a stan­dard late-stage Alzheimer’s tri­al, en­rolling a cou­ple thou­sand pa­tients and giv­ing half place­bo. Cer­tain­ly that would se­vere­ly cur­tail ac­cess to a drug Bio­gen hoped would treat hun­dreds of thou­sands and mor­tar-and-pes­tle any prof­its. But the Cen­ters for Medicare & Med­ic­aid Ser­vices, or CMS, of­fers a fair bit of lat­i­tude for what a study could look like. The key cri­te­ria are sim­ply that the tri­al be place­bo-con­trolled, en­roll a de­mo­graph­i­cal­ly rep­re­sen­ta­tive sam­ple of Alzheimer’s pa­tients in the US and be suf­fi­cient­ly pow­ered — i.e. have enough vol­un­teers — to give a de­ci­sive an­swer as to whether Aduhelm can slow pa­tients’ cog­ni­tive de­cline. And that, sta­tis­ti­cian Kert Viele ar­gues, gives Bio­gen a win­dow to pro­pose some­thing nov­el: A study that would both give a larg­er num­ber of pa­tients ac­cess to the amy­loid-clear­ing an­ti­body and make Alzheimer’s re­search more or­ga­nized and rig­or­ous for years to come. “This is one of those ex­am­ples of nev­er let­ting a good cri­sis go to waste,” said Viele, who helps phar­ma com­pa­nies de­sign clin­i­cal tri­als at Berry Con­sul­tants. Kert Viele The idea is sim­ple: Bio­gen pro­pos­es a “re­al­ly big” tri­al — at least 10,000 pa­tients, maybe far more. Be­cause you have so many vol­un­teers, you don’t have to give half place­bo. You could give a fourth, a sixth, or even a tenth place­bo and still get a ro­bust com­para­tor arm. That means more pa­tients get the drug. You would al­so de­sign it with min­i­mal re­quire­ments, such as fol­low-up as­sess­ments, so it’s easy for pa­tients out­side of big-city re­search in­sti­tu­tions where Alzheimer’s tri­als are gen­er­al­ly con­duct­ed, al­low­ing for a gen­uine­ly di­verse pa­tient group. (De­spite Black Amer­i­cans be­ing up to twice as like­ly to de­vel­op the dis­ease, Bio­gen en­rolled on­ly 0.6% Black pa­tients in its piv­otal Aduhelm stud­ies; most oth­er com­pa­nies do lit­tle bet­ter .) Bio­gen has shown no in­cli­na­tion to­ward such a study — a spokesper­son told End­points News it was “too pre­lim­i­nary” to dis­cuss tri­al de­tails, and on a call with an­a­lysts, CEO Michel Vounatsos didn’t ex­plic­it­ly rule out sim­ply pulling Aduhelm from the mar­ket. But it’s a use­ful ex­er­cise for two rea­sons. First, un­der CMS rules, any­one could the­o­ret­i­cal­ly pro­pose a tri­al and get re­im­burse­ment. Sec­ond, it of­fers a back-of-the-en­ve­lope blue­print for what Alzheimer’s re­search could look like if it were more or­ga­nized, join­ing a broad­er pan­dem­ic-era push to make clin­i­cal tri­als for all dis­eases larg­er, more co­or­di­nat­ed and more rig­or­ous. For Viele, the coup de grâce of such a study is that it could lay the ground­work for a large, adap­tive tri­al that would even­tu­al­ly test oth­er drugs in par­al­lel, al­low­ing re­searchers to test more agents in hu­mans more cheap­ly and quick­ly. A start­up with a nov­el mol­e­cule, for ex­am­ple, could plug it in­to the ex­ist­ing net­work, in­stead of hav­ing to set up their own. Drugs that showed lit­tle signs of ac­tiv­i­ty would be swapped out for the next con­tender. That could in­clude new amy­loid-clear­ing drugs that Roche, Bio­gen and Eli Lil­ly are like­ly to re­port da­ta on next year, should they, like ad­u­canum­ab, show mud­dled re­sults. Al­though re­searchers have used these kinds of so-called plat­forms of tri­als in ALS and can­cer, they’ve tak­en greater promi­nence for the role they played in Covid-19. Most no­tably, in the UK, the RE­COV­ERY study used the coun­try’s Na­tion­al Health Ser­vice to en­roll 40,000 pa­tients and gen­er­ate an­swers that in­formed treat­ment around the globe. That’s Viele’s mod­el: “Stream­lined,” he said, “big, sim­ple, ef­fi­cient, fast-en­rolling.” Of course, the US fa­mous­ly lacks the na­tion­wide health net­work that made RE­COV­ERY pos­si­ble. But Viele notes Amer­i­can re­searchers set up their own plat­form tri­als that proved — and dis­proved — sev­er­al covid treat­ments. Lon Schnei­der Lon Schnei­der, an Alzheimer’s ex­pert at USC, not­ed that CMS has backed big, sim­ple stud­ies be­fore. To test whether PET scans, a pricey di­ag­nos­tic tool for Alzheimer’s, can ac­tu­al­ly help pa­tients, the agency is fund­ing a na­tion­wide study to track sim­i­lar pa­tients who did and did not re­ceive a scan and see how their symp­toms change over time. “CMS is an in­sur­ance com­pa­ny, so CMS wants to look at health out­comes and costs,” he said. “It doesn’t mind spend­ing a lot of mon­ey if it gets some­thing of val­ue out of it.” Viele, though, is not the first to sug­gest a plat­form tri­al for Alzheimer’s. But so far, ex­cept for a small study on fa­mil­ial Alzheimer’s, none have launched. “It’s a good idea,” said PJ Viss­er, a neu­ropsy­chol­o­gist at Maas­tricht Uni­ver­si­ty in the Nether­lands, who was re­cent­ly in­volved in a large Eu­ro­pean ef­fort to launch an Alzheimer’s plat­form tri­al. “But it’s dif­fi­cult to re­al­ize.” In­deed, some Alzheimer’s ex­perts say that while they have long hoped for an Alzheimer’s plat­form tri­al, Viele’s strat­e­gy is flawed. Robert Howard, a pro­fes­sor of old age psy­chi­a­try at Uni­ver­si­ty Col­lege Lon­don, has twice un­suc­cess­ful­ly pe­ti­tioned the UK gov­ern­ment to fund a plat­form tri­al test­ing re­pur­posed psy­chi­atric drugs like lithi­um on Alzheimer’s pa­tients. But he ques­tioned the wis­dom of run­ning such a large study on Aduhelm. Rob Howard Viele’s pro­pos­al, he said, amount­ed to us­ing a clin­i­cal tri­al as a Tro­jan horse to give thou­sands of pa­tients ac­cess to a drug that on­ly might boost cog­ni­tion a tiny bit but def­i­nite­ly caus­es dan­ger­ous side ef­fects, such as brain bleeds . The CMS-man­dat­ed Aduhelm tri­al should be as small as pos­si­ble to get a de­ci­sive re­sult, he said. “Why would you want to ex­pose any more peo­ple than you ab­solute­ly need to, to treat­ment that we know has ad­verse ef­fects, and may not have any pos­i­tive ef­fects?” Howard said. In dis­eases that progress rapid­ly, such as Covid-19 or can­cer, ex­cep­tion­al­ly large tri­als can al­so bring ex­cep­tion­al­ly fast re­sults. If you en­roll 2,000 Covid-19 pa­tients in April, you’d be able to see how a giv­en drug af­fect­ed the en­tire course of their dis­ease be­fore sum­mer. The same doesn’t hold true for Alzheimer’s, a mal­a­dy fa­mous for its slow pre­cip­i­ta­tion. A 2,000-per­son tri­al and a 10,000-per­son tri­al will pro­duce re­sults around the same time. “There’s no short­cut to hav­ing 18 months’ fol­low-up time,” said Howard. And then there’s the con­straint vir­tu­al­ly every re­searcher hop­ing to start a plat­form study runs in­to: Phar­ma­ceu­ti­cal com­pa­nies are of­ten re­luc­tant to take part. They of­ten don’t want to give up con­trol over the da­ta. And they of­ten don’t want to see their drugs put head-to-head with a ri­val’s drug, for fear it may prove in­fe­ri­or. PJ Viss­er The tri­al Viss­er tried to set up, called EPAD, fell apart af­ter years of work and thou­sands of pa­tients en­rolled when no com­pa­nies want­ed to join, even though many had helped spon­sor its cre­ation. Scott Berry, pres­i­dent of Berry Con­sul­tants and an EPAD or­ga­niz­er, said drug de­vel­op­ers pulled out even af­ter cal­cu­la­tions clear­ly showed it would save them time and mon­ey. Viele coun­ters that com­pa­nies will sign on to plat­form stud­ies once oth­ers have al­ready joined. They save de­vel­op­ers the con­sid­er­able cost and time of set­ting up their own stud­ies, ad­van­tages that are too great to ig­nore. Berry agreed. He thinks EPAD might turn out dif­fer­ent­ly now, af­ter RE­COV­ERY. At the time, he said, com­pa­nies were re­luc­tant to turn over their “crown jew­els” to a rel­a­tive­ly un­known idea. “Had that project been cre­at­ed af­ter Covid, I think the dis­cus­sions would have been very, very dif­fer­ent with phar­ma,” he said. Tools have al­so come on­line that could make plat­form tri­als eas­i­er, said Viss­er. His­tor­i­cal­ly, re­searchers have had to give pa­tients cost­ly, in­va­sive scans to show they have plaques or tan­gles in their brain be­fore en­rolling them in most Alzheimer’s stud­ies. Slow­ly, though, blood tests are near­ing ap­proval on­line that could al­low physi­cians to screen pa­tients far more eas­i­ly. But does that mean Aduhelm is the right drug to start with? Schnei­der, the USC neu­rol­o­gist, isn’t so sure. Any study, he not­ed, could be up­end­ed if any of the new amy­loid drugs re­port de­ci­sive re­sults next year. And the da­ta Bio­gen has al­ready put out sug­gest that even if Aduhelm works, its ef­fects are min­i­mal, like­ly be­low what a pa­tient could no­tice. Plat­form tri­als could be use­ful, he said, but Aduhelm might not be worth the ef­fort. If “you’re ask­ing me per­son­al­ly, I’d say nah, give it up Bio­gen,” he said. “This ain’t go­ing to be a block­buster.” AUTHOR Zachary Brennan Senior Editor As President Biden’s Build Back Better Act — and, with it, potentially the Democrats’ last shot at major drug pricing reforms in the foreseeable future — remains on life support, the Congressional Budget Office isn’t helping their case. The CBO last week released a new slide deck , outlining an update to its model on how Medicare negotiations might take a bite out of new drugs making it to market. The new model estimates a 10% long-term reduction in the number of new drugs, whereas a previous CBO report from August estimated that 8% fewer new drugs will enter the market over 30 years. Read More John Carroll Editor & Founder Dyne Therapeutics’ stock took a nasty hit this morning after the biotech put out word that the FDA had slapped a clinical hold on their top program for Duchenne muscular dystrophy. And now speculation is bouncing around Biotwitter that there could be a class effect at work here that would implicate other drug developers in the freeze. Dyne execs didn’t have a whole lot to say about why the FDA sidelined their IND for DYNE-251 in DMD while “requesting additional clinical and non-clinical information for” the drug. Keep reading Endpoints with a free subscription Unlock this story instantly and join 129,100+ biopharma pros reading Endpoints daily — and it's free. SIGN UP John Carroll Editor & Founder In a stunning switch, GlaxoSmithKline put out word early Wednesday that R&D chief Hal Barron is exiting the company after 4 years — a relatively brief run for the man chosen by CEO Emma Walmsley in late 2017 to turn around the slow-footed pharma giant. Barron is being replaced by Tony Wood, a close associate of Barron’s who’s taking one of the top jobs in Big Pharma R&D. He’ll be closer to home, though, for GSK. Barron has been running a UK and Philadelphia-based research organization from his perch in San Francisco. Keep reading Endpoints with a free subscription Unlock this story instantly and join 129,100+ biopharma pros reading Endpoints daily — and it's free. SIGN UP John Carroll Editor & Founder Three years after Royalty Pharma chief Pablo Legorreta led a group of investors to buy up a pair of biotechs and create a new startup called ProKidney, the biotech is jumping straight into an $825 million public shell created by SPAC king and tech billionaire Chamath Palihapitiya. ProKidney was founded 6 years ago but really got going at the beginning of 2019 with the $62 million acquisition of inRegen, which was working on an autologous — from the patient — cell therapy for kidney disease. After extracting kidney cells from patients, researchers expand the cells in the lab and then inject them back into patients, aiming to restore the kidneys of patients suffering from CKD. Keep reading Endpoints with a free subscription Unlock this story instantly and join 129,100+ biopharma pros reading Endpoints daily — and it's free. SIGN UP LOG IN

Predict your next investment

The CB Insights tech market intelligence platform analyzes millions of data points on venture capital, startups, patents , partnerships and news mentions to help you see tomorrow's opportunities, today.

Maastricht University Patents

Maastricht University has filed 1 patent.

The 3 most popular patent topics include:

  • Cell biology
  • Human proteins
  • Intracellular receptors
patents chart

Application Date

Grant Date

Title

Related Topics

Status

9/18/2020

Transcription factors, Molecular biology, Intracellular receptors, Cell biology, Human proteins

Application

Application Date

9/18/2020

Grant Date

Title

Related Topics

Transcription factors, Molecular biology, Intracellular receptors, Cell biology, Human proteins

Status

Application

Maastricht University Web Traffic

Rank
Page Views per User (PVPU)
Page Views per Million (PVPM)
Reach per Million (RPM)
CBI Logo

Maastricht University Rank

CB Insights uses Cookies

CBI websites generally use certain cookies to enable better interactions with our sites and services. Use of these cookies, which may be stored on your device, permits us to improve and customize your experience. You can read more about your cookie choices at our privacy policy here. By continuing to use this site you are consenting to these choices.