Vivek Ra­maswamy’s Urovant claims a big win in PhI­II — but ques­tions linger about a cheap gener­ic and their brand­ed ri­val

Af­ter the great Ax­o­vant im­plo­sion that swept away its high­ly tout­ed Alzheimer’s drug, Roivant chief Vivek Ra­maswamy need­ed one of the biotechs in his fast-grow­ing league of drug de­vel­op­ers to win a big Phase III tri­al. Based on to­day’s top-line re­lease for Urovant’s Phase III uri­nary in­con­ti­nence drug vibegron, the team is claim­ing that vic­to­ry and prep­ping an ap­pli­ca­tion for the FDA.

But there are some lin­ger­ing ques­tions about the cheap gener­ic that was used as an ac­tive com­para­tor in the study, and it’s dri­ving the biotech’s stock in­to the red Tues­day morn­ing.

Urovant’s drug hit sta­tis­ti­cal sig­nif­i­cance for both co-pri­ma­ry end­points and all 7 sec­on­daries when com­pared against a place­bo. But the drug was al­so test­ed along­side an old gener­ic drug called toltero­dine. And there the re­search showed that vibegron “achieved nu­mer­i­cal­ly bet­ter ef­fi­ca­cy that toltero­dine.”

In the slide show pre­sen­ta­tion this morn­ing, the com­pa­ny went on to il­lus­trate the specifics of the com­para­tor arms ver­sus place­bo, but re­searchers did not run a p val­ue for the Urovant drug against the gener­ic. But they were rel­a­tive­ly close, as you can see here:

Source: Urovant

Urovant’s drug cut in­stances of in­con­ti­nence by 2 from a base­line of 3.43, achiev­ing a clear win over the 1.4 drop in the place­bo arm. Toltero­dine, though, came much clos­er at 1.8. Both drugs beat the place­bo with sta­tis­ti­cal sig­nif­i­cance.

For uri­na­tion fre­quen­cy, there was a sim­i­lar spread. The place­bo arm hit a drop of 1.3, with a neg­a­tive 1.8 for vibegron and -1.6 for toltero­dine. Vibegron hit sta­tis­ti­cal dif­fer­ence ver­sus place­bo, toltero­dine did not. But there was no di­rect com­par­i­son of the ex­per­i­men­tal drug and the cheap place­bo.

In their an­nounce­ment back in 2012, Astel­las post­ed these re­spons­es for Myr­be­triq.

Myr­be­triq 25 mg, in­con­ti­nence episodes were re­duced by 1.36 episodes from a base­line of 2.65, a sta­tis­ti­cal­ly sig­nif­i­cant re­duc­tion of 0.40 vs. place­bo in 12 weeks. The num­ber of uri­na­tions was re­duced by 1.65 uri­na­tions from a base­line of 11.68, a sta­tis­ti­cal­ly sig­nif­i­cant re­duc­tion of 0.47 vs. place­bo in 12 weeks.

Myr­be­triq 50 mg, in­con­ti­nence episodes were re­duced by 1.49 episodes from a base­line of 2.71, a sta­tis­ti­cal­ly sig­nif­i­cant re­duc­tion of 0.40 vs. place­bo in 12 weeks.  Num­ber of uri­na­tions was re­duced by 1.75 uri­na­tions from a base­line of 11.70, a sta­tis­ti­cal­ly sig­nif­i­cant re­duc­tion of 0.55 vs. place­bo in 12 weeks.

You can be sure that an­a­lysts will al­so be fill­ing in the blanks by ex­trap­o­lat­ing the head-to-head com­par­isons them­selves.

Urovant’s pitch is built around demon­strat­ing a bet­ter safe­ty and ef­fi­ca­cy pro­file than the an­ti­cholin­er­gics — like toltero­dine, where there’s been a link with de­men­tia — and the block­buster drug Myr­be­triq (mirabegron), which was launched by Astel­las back in 2012. Vibegron is a be­ta-3 drug, like Myr­be­triq, which is backed by patents that ex­pire in 2023 — about 3 years af­ter Urovant could ex­pect an ap­proval.

Giv­en that this drug is in­tend­ed for a mass mar­ket with mil­lions of pa­tients, teas­ing out the specifics of how a new, brand­ed drug com­pares to well es­tab­lished mar­ket­ed drugs would be cru­cial to its longterm suc­cess or fail­ure on the mar­ket. 

Biren Amin

Urovant, which has a mar­ket cap of $442 mil­lion, is let­ting it all ride on this Phase III. Ra­maswamy nabbed the drug from Mer­ck with a small, $25 mil­lion up­front — one of a slate of deals he struck ear­ly on as he grabbed ther­a­pies that had been side­lined at the ma­jors and aimed them for quick cat­a­lysts.

Big gam­ble? Jef­feries’ Biren Amin not­ed that a clear win here would be worth a 100% spike in the stock, with a neg­a­tive out­come elim­i­nat­ing up to half of its mar­ket cap.

That’s not what hap­pened, though. Af­ter pop­ping up 20% be­fore the bell, the stock turned south, plung­ing to a mi­nus 17% as in­vestors tried to sort out the re­sults for them­selves.


Im­age: Vivek Ra­maswamy at the US-Chi­na Bio­phar­ma In­no­va­tion & In­vest­ment Sum­mit, 2018. PHARM­CUBE, END­POINTS

Op­ti­miz­ing Cell and Gene Ther­a­py De­vel­op­ment and Pro­duc­tion: How Tech­nol­o­gy Providers Like Corn­ing Life Sci­ences are Spurring In­no­va­tion

Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

David Baker working with a student on their protein design (Jason Mast)

Sci­en­tists are fi­nal­ly learn­ing how to de­sign pro­teins from scratch. Drug de­vel­op­ment may nev­er be the same

SEATTLE — It’s a cloudy Thursday afternoon in mid-July and David Baker is reclining into the futon in his corner office at the University of Washington, arms splayed out like a daytime talk show host as he coaches another one of his postdocs through the slings and arrows of scientific celebrity.

“Be jealous of your time,” he says, before plotting ways of sneaking her out of Zooms. “It’s this horrible cost to science that you’re tied up in some stupid meeting.”

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Pre­sent­ing a live End­points News event: Man­ag­ing a biotech in tur­bu­lent times

Biotech is one of the smartest, best educated industries on the planet. PhDs abound. We’ve had a long enough track record to see a new generation of savvy, experienced execs coming together to run startups.

And in these times, they are being tested as never before.

Biotech is going through quite a rough patch right now. For 2 years, practically anyone with a decent resume and some half-baked ideas on biotech could start a company and get it funded. The pandemic made it easy in many ways to pull off an IPO, with traditional road shows shut down in exchange for a series of quick Zoom meetings. Generalist investors flocked as the numbers raised soared into the stratosphere.

Amidst R&D reshuf­fle, Ver­tex ex­pands its pres­ence in Boston, aim­ing to be­come num­ber one

Vertex Pharmaceuticals has been one of the buzzier names in the bustling Boston biotech scene, but now the company is looking to vault to number one status — at least in terms of physical footprint.

At a ribbon cutting on Tuesday for its new Jeffrey Leiden Center for Cell and Genetic Therapies at the Boston Seaport, Vertex announced it would embark on a new project: The company will build a 344,000 square foot facility in the seaport to accommodate the company’s growing R&D needs, especially in its cell and gene therapies program.

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Clay Siegall (Photo by Dimitrios Kambouris/Getty Images for Gabrielle's Angel Foundation)

UP­DAT­ED: Clay Sie­gall re­signs from Seagen amid in­ves­ti­ga­tion in­to do­mes­tic vi­o­lence claims

A week after Seagen revealed that longtime CEO Clay Siegall was on leave due to an allegation of domestic violence, he has resigned.

Since that shocking revelation, more details about the claims have emerged into the public eye. As Endpoints News reported, Siegall was arrested on April 23. A police report about that night and a subsequent temporary restraining order described a pattern of abusive behavior against his wife and a physical altercation that left her with multiple bruises. Siegall denied the claims.

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Patty Murray, D-WA (Graeme Sloan/Sipa USA)(Sipa via AP Images)

Sen­ate user fee reau­tho­riza­tion bill omits ac­cel­er­at­ed ap­proval re­forms, shows wide gaps with House ver­sion

The Senate health committee on Tuesday released its first version of the bill to reauthorize all the different FDA user fees. But unlike the House version, there are only a few controversial items in the Senate’s version, which does not address either accelerated approval reforms or clinical trial diversity (as the House did).

While it’s still relatively early in the process of finalizing this legislation (the ultimate statutory deadline is the end of September), the House and Senate, at least initially, appear to be starting off in different corners on what should be included.

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Warren Buffett, Berkshire Hathaway CEO

Berk­shire Hath­away pulls out of Ab­b­Vie, Bris­tol My­ers Squibb in­vest­ments

It looks like Warren Buffett is sticking to ice cream and railroads for the moment.

The billionaire CEO of Berkshire Hathaway backed out of two major holdings in the pharma industry, Forexlive first reported, including a $410 million investment in AbbVie and a $324.4 million stake in Bristol Myers Squibb.

The move comes after Berkshire abandoned its Teva shares just last quarter, Bloomberg reported.

Long-ex­pect­ed UK lay­offs im­mi­nent for No­var­tis fol­low­ing sale

Nearly a year ago, more than 200 workers at Novartis’ Grimsby, UK, facility were able to hang on to their jobs after the pharma closed a Switzerland site as a part of its workforce restructuring plan. Now, it looks like those employees’ time is up, as the site has been sold, Grimsby Telegraph reported today.

The manufacturing site has been sold to Humber Industrials, a subsidiary of International Process Plants. None of the current staff members will be working with the new owners, however.

FDA lob­bies Con­gress over rare dis­ease court rul­ing with wide im­pli­ca­tions

Usually reserved for making decisions on drug applications or enforcing what Congress stipulates, the FDA is now dipping its toe into the wild world of congressional politics as it attempts to fix a major court decision that could have a chilling effect on rare disease R&D.

The case in question from last October saw a US appeals court overturn a prior FDA court win, saying that the agency never should’ve approved a rare disease drug because a previously approved but more expensive drug with the same active ingredient has orphan drug exclusivity barring such an approval.