Vivek Ramaswamy (Jeff Rumans/JPM 2020)

Urovan­t's lead drug dis­ap­points in mid-stage study as first big FDA de­ci­sion looms

Just as Urovant gets ready for its first big FDA de­ci­sion on vibegron, the drug has flopped in what would’ve been a fol­low-on in­di­ca­tion.

In a Phase IIa tri­al in­volv­ing women with ab­dom­i­nal pain due to ir­ri­ta­ble bow­el syn­drome, vibegron failed to meet the bar on im­prov­ing “av­er­age worst ab­dom­i­nal pain” over 12 weeks, com­pared to place­bo, among IBS-D pa­tients.

There were ac­tu­al­ly slight­ly more re­spon­ders in the place­bo group than in the drug arm, with on­ly 40.9% of those ran­dom­ized to vige­bron achiev­ing at least a 30% de­crease in “worst ab­dom­i­nal pain” in the past 24 hours. The tri­al en­rolled 222 women but on­ly 189 com­plet­ed the study.

Even though the drug did some­what bet­ter in a key sec­ondary end­point — name­ly re­spon­ders on the Glob­al Im­prove­ment Scale — the 42.4% to 33.3% dif­fer­ence wasn’t enough to qual­i­fy for sta­tis­ti­cal sig­nif­i­cance.

Cor­nelia Haag-Molken­teller

“Dis­ap­point­ed,” Urovant CMO Cor­nelia Haag-Molken­teller is ready to shift fo­cus to the rest of the de­vel­op­ment plan.

“We look for­ward to ad­vanc­ing our Phase 3 pro­gram for vibegron in men with over­ac­tive blad­der and be­nign pro­sta­t­ic hy­per­pla­sia (BPH), as well as our Phase 2a pro­gram for URO-902 in OAB,” she said in a state­ment.

The biotech said it will con­tin­ue to an­a­lyze the full dataset, with­out com­mit­ting to any next steps.

A once dai­ly pill de­signed to ag­o­nize be­ta-3, vibegron is the key pro­gram for Urovant, which re­cent­ly ful­ly mi­grat­ed from Roivant to Sum­i­to­vant, the new um­brel­la struc­ture Vivek Ra­maswamy set up with Sum­it­o­mo Dainip­pon, in a deal val­ued at $584 mil­lion.

It claimed a Phase III win back in March 2019 when it hit both co-pri­ma­ry end­points and 7 sec­ondary end­points com­pared to place­bo. But ques­tions re­mained over its po­ten­tial ef­fi­ca­cy over a cheap gener­ic called toltero­dine used as an ac­tive com­para­tor.

Ra­maswamy, a flam­boy­ant deal en­gi­neer with a check­ered record, orig­i­nal­ly bought the drug from Mer­ck for $25 mil­lion.

5AM Ven­tures: Fu­el­ing the Next Gen­er­a­tion of In­no­va­tors

By RBC Capital Markets
With Andy Schwab, Co-Founder and Managing Partner at 5AM Ventures

Key Points

Prescription Digital Therapeutics, cell therapy technologies, and in silico medicines will be a vital part of future treatment modalities.
Unlocking the potential of the microbiome could be the missing link to better disease diagnosis.
Growing links between academia, industry, and venture capital are spinning out more innovative biotech companies.
Biotech is now seen by investors as a growth space as well as a safe haven, fuelling the recent IPO boom.

Janet Woodcock (AP Images)

End­points poll: Janet Wood­cock takes the (in­ter­im) helm at the FDA. And a large ma­jor­i­ty of our read­ers want her to stay there

It’s official: Janet Woodcock is now the acting chief of the FDA.

And — according to an Endpoints poll — most industry readers would like her to stay there, although a significant minority is strongly opposed.

To recap: Joe Biden is reportedly choosing between Woodcock and former deputy FDA commissioner Joshua Sharfstein as his nominee for the permanent position. Given their respective track records, the decision is set to determine the agency’s lodestar for years to come.

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What’s next for End­points — and how to sup­port our in­de­pen­dent bio­phar­ma news mis­sion

The firehose of biopharma news is gushing these days.

That’s why broader and deeper is the theme for 2021 at Endpoints. You can expect new coverage outside our core R&D focus, with deeper reporting in some key areas. When John Carroll and I launched Endpoints nearly five years ago, we were wading in waist-high waters. Now we’re a team of 25 full-time staffers (and growing) with plans to cover the flood of biopharma news, Endpoints-style.

Eli Lil­ly's an­ti­body cuts risk of Covid-19 by up to 80% among the most vul­ner­a­ble — but will it have a place next to vac­cines?

Eli Lilly says bamlanivimab lowered the risk of contracting symptomatic Covid-19 in a first-of-its-kind trial involving nursing home residents and staff, paving the way for a new option to protect against the virus.

But how big of an impact it might have, and what role it will play, at a time vaccines are being rolled out to the exact population it is targeting still remains unclear.

Among 965 participants in the study — all of whom tested negative for the coronavirus at baseline — the number of symptomatic cases reported in the bamlanivimab arm was 57% lower than that in the placebo arm (odds ratio 0.43, p=0.00021). In addition to that primary endpoint, all secondary endpoints reached statistical significance.

Hal Barron, GSK R&D chief (GSK via YouTube)

Glax­o­SmithK­line's $4B bis­pe­cif­ic can­cer drug al­liance with Mer­ck KGaA hit by big set­back with a PhI­II fail­ure on NSCLC

Close to 2 years ago, GSK’s R&D team eagerly agreed to pay up to $4 billion-plus to ally itself with Merck KGaA on a mid-stage bispecific called bintrafusp alfa, which intrigued them with the combination of a TGF-β trap with the anti-PD-L1 mechanism in one fusion protein.

But today the German pharma company says that their lead study on lung cancer was a bust, as independent monitors said there was no reason to believe that the experimental drug — targeting PD-L1/TGF-Beta — could beat Keytruda.

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With patent con­cerns loom­ing, Roche gets a new pri­or­i­ty re­view on block­buster IPF drug

Seven years after the FDA first approved Esbriet, the blockbuster Roche IPF drug is getting an expedited review for a second indication.

On Thursday, the agency gave Esbriet priority review for unclassified interstitial lung diseases, or forms of pulmonary inflammation and scarring that don’t fit easily into the over 200 known types of ILD. The move comes 10 months after Esbriet received breakthrough designation and sets Roche up for a decision by May.

Michelle McMurry-Heath, BIO CEO (BIO via YouTube)

BIO looks to re­struc­ture, lay­ing off staff amid chal­lenge to the trade org's nor­mal face-to-face style

The biopharma industry, on the whole, had a red-letter year in 2020 amid Covid-19, with fundraising at an all-time high and major players speeding vaccines ahead to approval. But for BIO, the industry’s leading trade organization, the pandemic has prompted a reconsideration of the game plan.

BIO will pivot to digital as the Covid-19 pandemic continues to rage, making “some staff reductions” as it looks to bring its roughly 37,000 in-person meetings each year to the web, the organization said Thursday.

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Covid-19 roundup: Italy won­ders aloud if it can sue Pfiz­er for vac­cine short­falls; US recom­mits to WHO un­der Biden ad­min­is­tra­tion

As reports crop up that deliveries of Pfizer and BioNTech’s Covid-19 vaccine are being unexpectedly cut, Italy wonders if it can take the vaccine developers to court, according to the Wall Street Journal. 

After its shipment for this week was cut by 29%, the Italian government consulted its attorney general about taking legal action, the WSJ reported. Pfizer and BioNTech had warned the EU and Canada last week that their allocations would be reduced as Pfizer upgrades its Belgium factory. What Italy says it doesn’t appreciate, though, is the short notice.

Janet Woodcock and Joshua Sharfstein (AP, Images)

Poll: Should Joshua Sharf­stein or Janet Wood­cock lead the FDA from here?

It’s time for a new FDA commissioner to come on board, a rite of passage for Joe Biden’s administration that should help seal the new president’s rep on seeking out the experts to lead the government over the next 4 years.

As of now, the competition for the top job appears to have narrowed down to 2 people: The longtime CDER chief Janet Woodcock and Joshua Sharfstein, the former principal deputy at the FDA under Peggy Hamburg. Both were appointed by Barack Obama.