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Can we make the an­tibi­ot­ic mar­ket great again?

The stan­dard for-prof­it mod­el in drug de­vel­op­ment is straight­for­ward. Spend mil­lions, even bil­lions, to de­vel­op a med­i­cine from scratch. The re­turn on in­vest­ment (and ide­al­ly a tidy prof­it) comes via vol­ume and/or price, de­pend­ing on the dis­ease. But the string of big phar­ma ex­its and slew of biotech bank­rupt­cies in­di­cate that the mod­el is sore­ly flawed when it comes to an­tibi­otics.

The in­dus­try play­ers con­tribut­ing to the ar­se­nal of an­timi­cro­bials are fast dwin­dling, and the pipeline for new an­tibi­otics is em­bar­rass­ing­ly sparse, the WHO has warned. Drug­mak­ers are en­ticed by green­er pas­tures, com­pared to the long, ar­du­ous and ex­pen­sive path to an­tibi­ot­ic ap­proval that of­fers lit­tle fi­nan­cial gain as treat­ments are typ­i­cal­ly priced cheap­ly, and of­ten lose po­ten­cy over time as mi­crobes grow re­sis­tant to them.

Mean­while, doc­tors — of­ten con­fined by hos­pi­tal bud­gets — pre­fer to use old­er, cheap­er an­tibi­otics in their first re­sponse, re­serv­ing fresh, more tar­get­ed al­ter­na­tives for acute cas­es.

Lance Price

Doc­tors should pre­scribe as few an­tibi­otics as pos­si­ble, ex­cept in the cas­es where they can re­al­ly help — but bio­phar­ma com­pa­nies need many pa­tients treat­ed to re­coup their in­vest­ment.

“They’re sell­ing more of the drugs, they’re re­coup­ing at cost, but they’re al­so then dri­ving up re­sis­tance to that drug,” not­ed Lance Price, found­ing di­rec­tor of George Wash­ing­ton Uni­ver­si­ty’s An­tibi­ot­ic Re­sis­tance Ac­tion Cen­ter (ARAC), in an in­ter­view with End­points News.

The cur­rent frame­work, which large­ly re­lies on for-prof­it drug­mak­ers, is there­fore part­ly to blame for the loom­ing su­per­bug cri­sis, he said. “So if we de­vel­op new drugs, and we just put them in­to the same sys­tem, we know where we’re go­ing to end up right?”

“I think we re­al­ly have to take a sys­tem­at­ic ap­proach and just re­vamp the whole thing.”

A PUB­LIC OP­TION?

Drug-re­sis­tant in­fec­tions are fast be­com­ing the norm. Ex­trav­a­gant an­tibi­ot­ic use has re­sult­ed in the tsuna­mi of mul­ti-drug re­sis­tant bac­te­ria. The WHO es­ti­mates drug-re­sis­tant bac­te­r­i­al, vi­ral and fun­gal in­fec­tions al­ready kill 700,000 peo­ple each year. Un­less ac­tion is tak­en, the avalanche of an­timi­cro­bial re­sis­tance (AMR) could cause 10 mil­lion deaths an­nu­al­ly by 2050, ac­cord­ing to the World Bank.

“If we reach that point, then we’re go­ing to start shelling out tons of mon­ey, right?” Price, who al­so serves as a pro­fes­sor at the George Wash­ing­ton Uni­ver­si­ty’s Milken In­sti­tute School of Pub­lic Health, ex­claimed. “We’re go­ing to hit a kind of des­per­a­tion, we’re go­ing to start writ­ing gi­ant checks…”

“So rather than wait­ing till that point — why don’t we just mod­el what hap­pens at that point, how much it’s go­ing to cost…and say, all right, why don’t we take a frac­tion of that and let’s cre­ate a whole new sys­tem for de­vel­op­ing new drugs and make them a pub­lic good,” he sug­gest­ed. “I would imag­ine it would come out to be cheap­er that way.”

Jo­hannes Frue­hauf

There may be some mer­it to that idea, but that means gov­ern­ments will be re­spon­si­ble for fund­ing, not­ed Jo­hannes Frue­hauf, doc­tor, co-founder of Mis­sion Bio­Cap­i­tal, a seed-stage life sci­ences in­vest­ment fund, and the Bi­o­labs net­work, an in­cu­ba­tor for US-based biotech star­tups.

“I ac­tu­al­ly doubt that will have the po­lit­i­cal will,” he said. “Un­til we have made it very clear to the every­day man how big a risk this is for him and his fam­i­ly — I think that’s cur­rent­ly not clear — I don’t think there will be enough po­lit­i­cal sup­port for a mas­sive ef­fort.”

Isaac Ston­er, chief of Oc­ta­gon Ther­a­peu­tics — a com­pa­ny that piv­ot­ed away from an­tibi­ot­ic de­vel­op­ment to au­toim­mune dis­ease af­ter en­coun­ter­ing the pit­falls of the ‘bro­ken’ an­tibi­ot­ic mar­ket, echoed his skep­ti­cism.

“When you see large, bloat­ed, pub­lic or­ga­ni­za­tions come to­geth­er and try and solve the prob­lem like that. It’s not al­ways the most ef­fi­cient way to do it,” he said. “I fun­da­men­tal­ly be­lieve that treat­ing very sick peo­ple and sav­ing lives should be good busi­ness on its own with­out gov­ern­ment in­ter­ven­tion.”

But good busi­ness it is not. Big phar­ma has large­ly re­treat­ed from an­tibi­otics, on­ly a hand­ful re­main in the space — in­clud­ing Mer­ck and GSK — down from more than twen­ty in the 1980s. For one of the biggest threats to glob­al health, the li­on’s share of an­tibi­ot­ic de­vel­op­ment is tak­ing place in a hand­ful of labs of small bio­phar­ma com­pa­nies as a ma­jor­i­ty of their larg­er coun­ter­parts fo­cus on more lu­cra­tive en­deav­ors.

Ted Schroed­er

A host of small an­tibi­ot­ic com­pa­nies that have man­aged to get their drugs ap­proved haven’t had much luck stay­ing afloat ei­ther. In re­cent years, an­tibi­ot­ic de­vel­op­ers — in­clud­ing Achao­gen, Tetraphase, and Melin­ta — have seen their val­ue go up in smoke as fee­ble sales frus­trat­ed growth.

“Right now, if we don’t do some­thing to sup­port the small com­pa­nies that have the in­no­v­a­tive prod­ucts, we run a re­al risk of not on­ly com­pa­nies go­ing bank­rupt — but los­ing the an­tibi­ot­ic de­vel­op­ment ex­per­tise that’s res­o­nant with­in those com­pa­nies, be­cause the sci­en­tists tend to move on and do oth­er things,” No­var­tis spin­off Nabri­va chief Ted Schroed­er told End­points.

LEG­IS­LA­TION

In the Unit­ed States, in­cen­tives are al­ready in place to push drug­mak­ers to de­vel­op an­tibi­otics, such as fund­ing sup­port through the Bio­med­ical Ad­vanced Re­search and De­vel­op­ment Au­thor­i­ty (BAR­DA) and reg­u­la­to­ry re­forms such as the Lim­it­ed Pop­u­la­tion Path­way for An­tibac­te­r­i­al and An­ti­fun­gal Drugs (LPAD)  — but the in­dus­try is clam­or­ing for the pas­sage of “pull in­cen­tives,” or pol­i­cy mea­sures to in­crease the val­ue of a mar­ket­ed an­tibi­ot­ic by re­ward­ing drug­mak­ers on­ly af­ter their an­tibi­ot­ic is ap­proved.

Seema Ver­ma

Ex­ist­ing in­cen­tives, “while well-in­ten­tioned…ap­pear to have been in­suf­fi­cient, as they fo­cused ex­clu­sive­ly on bol­ster­ing the de­vel­op­ment pipeline with­out re­mov­ing the block­age cre­at­ed by is­sues with pay­ment,” CMS ad­min­is­tra­tor Seema Ver­ma con­ced­ed last Au­gust.

For­mer FDA com­mis­sion­er Scott Got­tlieb, in 2018, sug­gest­ed a “li­cens­ing mod­el” in which acute care in­sti­tu­tions that pre­scribe an­timi­cro­bial med­i­cines pay a fixed li­cens­ing fee for ac­cess to these drugs, grant­i­ng them the right to use a cer­tain num­ber of an­nu­al dos­es.

Last year, the CMS un­veiled a pro­pos­al to re­struc­ture the pay­ment ap­pa­ra­tus to res­cue ex­ist­ing an­tibi­ot­ic man­u­fac­tur­ers, by clas­si­fy­ing drug re­sis­tance in a way that would com­pel high­er pay­ments to hos­pi­tals treat­ing pa­tients with an­timi­cro­bial re­sis­tance, and craft­ing a path­way for doc­tors to pre­scribe ap­pro­pri­ate new an­tibi­otics with­out dis­rupt­ing hos­pi­tal bud­gets. Un­der the cur­rent sys­tem, hos­pi­tals bun­dle to­geth­er the costs of all the ser­vices for a giv­en di­ag­no­sis, which tends to in­cen­tivize hos­pi­tals to pre­scribe cheap­er, gener­ic an­tibi­otics that are not en­gi­neered to tack­le drug-re­sis­tant in­fec­tions.

The bi­par­ti­san DIS­ARM (De­vel­op­ing an In­no­v­a­tive Strat­e­gy for An­timi­cro­bial Re­sis­tant Mi­croor­gan­isms) leg­is­la­tion al­so has wide­spread sup­port from the in­dus­try and oth­er stake­hold­ers. The bill is de­signed to com­pel re­im­burse­ment by Medicare of an­tibi­otics that treat stub­born in­fec­tions away from the bun­dled pay­ment sys­tem, with­in which all an­tibi­otics cur­rent­ly re­side. “I think ul­ti­mate­ly, that’s the best so­lu­tion,” Nabri­va’s Schroed­er said.

Mean­while, across the At­lantic, the Unit­ed King­dom al­so un­veiled a key step in 2019 to re­sus­ci­tate an­timi­cro­bial de­vel­op­ment by woo­ing man­u­fac­tur­ers with the in­cen­tive of a ‘sub­scrip­tion’ style pay­ment: the drugs will be paid for, even if they’re just stored in re­serves. In 2018, a Eu­ro­pean pub­lic-pri­vate con­sor­tium al­so rec­om­mend­ed in­sti­tut­ing a mar­ket en­try re­ward of $1 bil­lion per an­tibi­ot­ic glob­al­ly to re­sus­ci­tate the an­tibi­ot­ic pipeline.

Aleks En­gel

These are ex­am­ples of ini­tia­tives that re­ward drug mak­ers not by the vol­ume of goods sold, but for the val­ue they bring, which is need­ed; along with an in­jec­tion of pri­vate funds, said Aleks En­gels, di­rec­tor of the RE­PAIR im­pact fund.

The fund, which has in­vest­ed $48 mil­lion in 8 com­pa­nies so far, was cre­at­ed by the in­vest­ment arm of No­vo Nordisk’s No­vo Hold­ings in 2018 to fund de­vel­op­ers fo­cused on tar­get­ing drug-re­sis­tant pathogens.

“If there is a pay­ment of some sort, for ac­tu­al­ly get­ting all the way to the end de­liv­er­ing nov­el an­tibi­otics to the pub­lic — and if that in­cen­tive is suf­fi­cient­ly cer­tain and suf­fi­cient­ly large — then the mar­ket­place would go back to func­tion­ing,” he said.

But Oc­ta­gon’s Ston­er was not con­vinced. These steps, while in­cre­men­tal­ly pos­i­tive, don’t quite solve the is­sue at hand, he said.

“What we need is for there to be a mar­ket where you can go you can make your in­vest­ment back plus a mul­ti­ple,” he em­pha­sized. “It might take an­oth­er decade for san­i­ty to be re­stored. And we’re not go­ing to wait around for that — we quite sim­ply can’t.”

PUZ­ZLING PRIC­ING

A study pub­lished in 2018 found that an­tibi­ot­ic re­sis­tance added $1,383 to the cost of treat­ing a pa­tient with a bac­te­r­i­al in­fec­tion in the Unit­ed States, which amounts to a na­tion­al cost of $2.2 bil­lion an­nu­al­ly on the ba­sis of the num­ber of in­fec­tions re­port­ed in 2014. Still, the US health­care sys­tem is used to pay­ing very lit­tle for an­tibi­otics.

Bac­te­r­i­al in­fec­tions can re­sult in the loss of limbs, even brain func­tion. “If a drug can cure that, the drug should be paid (for),” said Frue­hauf, who al­so co-found­ed Lab­Cen­tral, the non-prof­it co-work­ing lab/in­cu­ba­tor in Kendall Square. “If you com­pare that to sur­gi­cal in­ter­ven­tion or oth­er long term care, it’s tens of thou­sands of dol­lars in the US…if you can achieve the same with a drug or two, then it ought to be ac­cept­able that the drug is priced high.”

Isaac Ston­er

In 2015, when Oc­ta­gon’s Ston­er first met his co-founders, he thought the first $50,000 an­tibi­ot­ic would be soon a re­al­i­ty, giv­en the rise in drug re­sis­tance rates.

When you look at rare or ge­net­ic dis­eases, pay­ers deal with prices like $350,000 $850,000, Ston­er said. “Why can’t we get $100,000 for an an­tibi­ot­ic?”

“Why is sav­ing some­one from an ag­gres­sive metasta­t­ic can­cer some­how worth more than sav­ing some­one from an ag­gres­sive mul­tidrug-re­sis­tant bac­te­ria? I don’t un­der­stand that.”

If an­tibi­otics were priced in line with their ther­a­peu­tic val­ue, doc­tors and hos­pi­tals will be forced to be­come more re­spon­si­ble pre­scribers, he sug­gest­ed.

“This is the last re­sort drug…that’s go­ing to save the pa­tient, and is ex­pen­sive enough that I’m go­ing to think twice be­fore throw­ing it around willy-nil­ly,” he said. “I think that is the so­lu­tion, we need to see two more ze­ros added to the cur­rent re­im­burse­ment par­a­digm.”

In­creas­ing US drug prices — even life-sav­ing med­i­cines — in the cur­rent pric­ing en­vi­ron­ment, where pa­tients, pol­i­cy­mak­ers, and politi­cians are up in arms against the pace and mag­ni­tude of price hikes is like­ly a fool’s er­rand. The phar­ma­ceu­ti­cal in­dus­try al­ready holds the crown for the least fa­vored sec­tor by Amer­i­cans, falling be­hind the fed­er­al gov­ern­ment it­self.

Mean­while, law­mak­ers on ei­ther side of the aisle are work­ing on leg­is­la­tion to low­er US drug prices, but so far no­body can agree on just how to make the US health care sys­tem great again. The in­dus­try, which has long thrived in a lais­sez-faire pric­ing en­vi­ron­ment in the Unit­ed States, has ve­he­ment­ly ar­gued that gov­ern­ment in­ter­ven­tion will sti­fle in­no­va­tion.

“It’s po­lit­i­cal­ly un­ten­able — ar­gu­ing right now in fa­vor of pric­ing in­creas­es…quite sad­ly, that could do it.” Ston­er said. “If I ruled the world, I would just say, okay, you know, we’re go­ing to have nov­el an­tibi­otics now re­im­bursed at the cost that an in­pa­tient ICU stay costs.”

PRE­CI­SION AN­TIBI­OTICS

The ac­ci­den­tal dis­cov­ery of peni­cillin, the world’s first an­tibi­ot­ic, by Scot­tish re­searcher Alexan­der Flem­ing opened the flood­gates in 1928, in­spir­ing the gold­en age of an­tibi­ot­ic dis­cov­ery, in which half the an­tibi­otics in pop­u­lar use to­day were dis­cov­ered. That heady pace of dis­cov­ery has since thawed — since the 1980s, there have been no new class of an­tibi­otics ap­proved.

Joan But­ter­ton

“The bac­te­r­i­al genome — it’s very small,” Joan But­ter­ton, as­so­ciate VP of clin­i­cal re­search, in­fec­tious dis­eases at Mer­ck, told End­points

“So there’s on­ly a cer­tain num­ber of tar­gets that we can po­ten­tial­ly go af­ter — and all the easy ones have been tak­en. And a lot of what we do with try­ing to de­vel­op new drugs is come up with mol­e­cules that just work bet­ter.”

Mean­while, the lib­er­al use of broad-spec­trum an­tibi­otics — pre­cip­i­tat­ed by the lack of time-sen­si­tive di­ag­nos­tics — can rav­age the pa­tient’s mi­cro­bio­me, erad­i­cat­ing the tox­ic bac­te­ria in tan­dem with the healthy gate­keep­ers of the gut that play a vi­tal role in pathogen re­sis­tance, nu­tri­ent ac­qui­si­tion and mod­u­lat­ing the im­mune sys­tem.

So some an­tibi­ot­ic de­vel­op­ers have tak­en in­spi­ra­tion from ad­vances in on­col­o­gy. The emer­gence of pre­ci­sion can­cer drugs, tai­lored to fit the nuts and bolts of the pa­tient and their strain of dis­ease, ush­ered in a fresh era in the treat­ment of the dead­ly ill­ness. Pre­ci­sion an­tibi­otics — en­gi­neered to maim or kill spe­cif­ic bac­te­ria — are en­vi­sioned as the next fron­tier in the bat­tle against su­per­bugs.

Manos Per­ros

Decades ago, the in­tro­duc­tion of No­var­tis’ Gleevec thrust the tar­get­ed ap­proach in­to gear and trans­formed the treat­ment of can­cer. That is how the an­tibi­ot­ic space is evolv­ing, Manos Per­ros, chief of As­traZeneca spin­off En­ta­sis, told End­points.

“So we don’t see our­selves as a bet­ter chemother­a­py com­pa­ny or the equiv­a­lent,” he said. “We see our­selves as the next CAR-T com­pa­ny (in the field of an­tibi­otics) that is now look­ing at pa­tient pop­u­la­tions where the med­ical need is great.”

One of the Mass­a­chu­setts-based com­pa­ny’s lead ther­a­pies is in late-stage de­vel­op­ment for a hos­pi­tal pathogen called Acine­to­bac­ter, which is mul­ti-drug re­sis­tant and is as­so­ci­at­ed with high mor­tal­i­ty rates. Akin to the CAR-T ther­a­pies, which car­ry pre­mi­um prices for their abil­i­ty to treat a sub­set of pa­tients with cer­tain forms of can­cer where stan­dard ther­a­pies haven’t kept the dis­ease at bay, En­ta­sis is hop­ing to ad­dress the 30,000 to 50,000 US pa­tients that do not re­spond to stan­dard an­tibi­otics.

In or­der to do that, the com­pa­ny has em­ployed a phar­ma­coeco­nom­ic ap­proach in its Phase III de­sign, where the ex­per­i­men­tal drug will be test­ed head-to-head against a com­para­tor in the num­ber of days the pa­tient stays in the ICU or is bound to a ven­ti­la­tor.

Er­ic Kim­ble

“We need to be able to price this drug…where we would be able to jus­ti­fy the re­turn on the in­vest­ment that we’ve made in this pro­gram,” En­ta­sis’ chief com­mer­cial of­fi­cer Er­ic Kim­ble ar­gued. If the En­ta­sis drug reigns supreme on these met­rics, he said, “we can eas­i­ly quan­ti­fy the cost sav­ings to an in­sti­tu­tion.”

UK-based Sum­mit Ther­a­peu­tics, whose lead pro­gram is tar­get­ing the stub­born C. diff in­fec­tion, is tak­ing the pre­ci­sion ap­proach one step fur­ther.

Most re­cent­ly launched an­tibi­otics have been im­proved ver­sions of reg­i­mens with ex­ist­ing mech­a­nisms of ac­tion us­ing the non-in­fe­ri­or­i­ty tri­al de­sign, which bac­te­ria will find a way to sub­vert soon­er rather than lat­er, chief Glyn Ed­wards told End­points.

Glyn Ed­wards

“The an­tibi­ot­ic space has not been great… but we think we can make great again,” he said, by choos­ing to de­vel­op prod­ucts for in­di­ca­tions where the ef­fi­ca­cy of stan­dard an­tibi­otics is want­i­ng, and work­ing on prov­ing su­pe­ri­or­i­ty in clin­i­cal tri­als (and not just non-in­fe­ri­or­i­ty) where pos­si­ble and us­ing health-eco­nom­ic cal­cu­la­tions to demon­strate sav­ings achieved by low­er re­cur­rence rate.

“I don’t think that’s any dif­fer­ent from any oth­er ther­a­peu­tic area,” he said, as­sert­ing that if the da­ta shows the an­tibi­ot­ic is bet­ter than ex­ist­ing treat­ments and re­duces the rate of re­cur­rence, doc­tors and hos­pi­tals will be will­ing to pay a pre­mi­um price.

“If you can show that the hos­pi­tal health­care sys­tem will save mon­ey…even if the ac­qui­si­tion costs are high­er, then that’s a very at­trac­tive mar­ket,” he said.

Sum­mit’s ex­per­i­men­tal C. diff ther­a­py, which Sum­mit says kills spe­cif­ic bac­te­ria by thwart­ing cell di­vi­sion, is in late-stage de­vel­op­ment.

“I think it’s wrong to just look at the re­cent prod­uct launch­es, and then throw your hands in the air and say you can’t make mon­ey out of this,” Ed­wards added. “If you do have a good prod­uct, and you show it is a bet­ter prod­uct, and it’s an area of high un­met need, and there are lots of pa­tients — you can make a lot of mon­ey from it.”

But test­ing for su­pe­ri­or­i­ty in an­tibi­ot­ic tri­als is no walk in the park. “It’s one of those things that aca­d­e­mics like to fo­cus on,” Schroed­er of Nabri­va told End­points.

The guid­ance from the FDA is to gen­er­al­ly con­duct non-in­fe­ri­or­i­ty tri­als, he said; “that is a reg­u­la­to­ry ar­ti­fact.”

But giv­en the need to elim­i­nate pa­tients who are re­sis­tant to the com­para­tor drug, it’s very dif­fi­cult to do su­pe­ri­or­i­ty tri­als, he ar­gued. “The prac­ti­cal re­al­i­ty of do­ing drug de­vel­op­ment doesn’t usu­al­ly al­low for a su­pe­ri­or­i­ty de­sign when you have to drop pa­tients out, be­cause they’re like­ly to be non-re­spon­ders based on their mi­cro­bi­o­log­i­cal pro­file.”

“So you’re kind of stack­ing the deck against su­pe­ri­or­i­ty, just be­cause it would be un­eth­i­cal to treat some­one with an an­tibi­ot­ic for an or­gan­ism that was show­ing re­sis­tance.”

But whether pre­ci­sion an­tibi­ot­ic de­vel­op­ers are able to find ways to prove su­pe­ri­or­i­ty takes a back­seat to the plagu­ing is­sue of di­ag­nos­tics, which can take days to iden­ti­fy the of­fend­ing pathogen. When a pa­tient presents with symp­toms of in­fec­tious dis­ease, doc­tors don’t have weeks or days to de­cide what ther­a­py to use.

The tech­nol­o­gy en­gi­neered to im­prove di­ag­no­sis time­lines al­ready ex­ists, En­ta­sis’ Per­ros said.

“If you get the kinds of drugs that would ben­e­fit from that di­ag­no­sis, there is no com­mer­cial dri­ver for a com­pa­ny to de­vel­op the tech­nol­o­gy in­to a com­mer­cial prod­uct, and vice ver­sa…its a chick­en and egg sit­u­a­tion,” he sug­gest­ed. “But over the last cou­ple of years that vi­cious cy­cle has been bro­ken — there are com­mer­cial prod­ucts on the mar­ket, and we’ve been work­ing with a (di­ag­nos­tic) com­pa­ny to run our Phase III tri­al.”

The Big Phar­ma dis­card pile; Lay­offs all around while some biotechs bid farewell; New Roche CEO as­sem­bles top team; and more

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Sen. Ron Wyden (D-OR) (Francis Chung/E&E News/Politico via AP Images)

In­fla­tion re­bates in­com­ing: Wyden calls on CMS to move quick­ly as No­var­tis CEO pledges re­ver­sal

Senate Finance Chair Ron Wyden (D-OR) this week sent a letter to the head of the Centers for Medicare & Medicaid Services seeking an update on how and when new inflation-linked rebates will take effect for drugs that see major price spikes.

The newly signed Inflation Reduction Act requires manufacturers to pay a rebate to Medicare when they increase drug prices faster than the rate of inflation.

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Sanofi scraps PhI­II tri­al for Prin­cip­ia drug af­ter re­view­ing com­pe­ti­tion

Months after the FDA placed Phase III trials of Sanofi’s BTK inhibitor on hold, the company is winding down one of the studies.

Sanofi reported in its Q4 earnings that the URSA study “was discontinued after careful evaluation of the emerging competitive treatment landscape in” myasthenia gravis, a rare disease that causes muscle weakness.

The Phase III, placebo-controlled trial was testing tolebrutinib in patients with the moderate-to-severe form of the disease. It started in late 2021, according to records on clinicaltrials.gov, and was originally designed to recruit 154 participants who were receiving the standard of care.

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Raymond Stevens, Structure Therapeutics CEO

Be­hind Fri­day's $161M IPO: A star sci­en­tist, GPCR drug dis­cov­ery and a plan to chal­lenge phar­ma in di­a­betes

What does it take to pull off a $161 million biotech IPO these days?

In Structure Therapeutics’ case, it means having a star scientist co-founder paired with the computational drug discovery company Schrödinger, $198 million in private funding from blue-chip investors, almost six years of research work on G protein-coupled receptors and a slate of oral, small-molecule drugs, with an eye on the huge and growing diabetes and weight-loss market.

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Af­ter 13 years, Ramy Mah­moud steps in­to CEO seat at Opti­nose; Ru­pert Vessey set to ex­it Bris­tol My­ers in Ju­ly

After 13 years as president and COO at Optinose, Ramy Mahmoud has stepped into a new role as its CEO. He is taking the place of Peter Miller, who stepped down earlier this week, though Miller is still staying with the company as a consultant.

In 2010, the two business partners joined Optinose to take it in a new direction, transforming it from a delivery platform to product company. They previously worked together at Johnson & Johnson, when Miller was president at Janssen and Mahmoud headed medical affairs. Miller said after he learned about Optinose, “I did what I always do, which is find people smarter than me to talk with about the idea. And the first person I called was Ramy … and I said, ‘Hey, Ramy, what do you think of this technology?’”

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How to use ex­ter­nal con­trols: FDA spells out think­ing in new draft guid­ance

The use of real-world evidence to inform the FDA’s decision-making continues apace, with the agency releasing new draft guidance yesterday on how sponsors can compare outcomes of trial participants receiving a test treatment with outcomes in a group of people external to the trial.

The practice of externally controlled trials is common, particularly in oncology or other difficult areas where it’s not ethical or feasible to use internal controls.

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The Big Phar­ma axe: Mer­ck cuts chikun­gun­ya vax, Bris­tol My­ers drops Cy­tomX-part­nered pro­gram, and more

As fourth quarter earnings come in, Big Pharmas are disclosing changes to their pipelines during their investor calls, and sometimes more quietly in presentation appendices.

Merck dropped its chikungunya vaccine candidate, which completed a Phase II study. Merck acquired the vaccine through its purchase of Themis Bioscience in 2020. In developing a vaccine for chikungunya, a mosquito-borne virus, Valneva is the frontrunner, as it submitted its vaccine to the FDA at the end of December.

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