Urovan­t's over­ac­tive blad­der drug scores in mid-stage tri­al

Urovant Sci­ences said its ex­per­i­men­tal over­ac­tive blad­der drug vibegron im­proved symp­toms in a large mid-stage study, a wel­come sig­nal for the com­pa­ny as it eval­u­ates the drug fur­ther in a phase III ini­ti­at­ed ear­li­er this year.

The phase IIb two-stage tri­al en­rolled more than 1,300 pa­tients with OAB symp­toms. In the first stage, pa­tients ei­ther re­ceived once-dai­ly oral vibegron (3 mg, 15 mg, 50 mg or 100 mg), the uri­nary in­con­ti­nence drug toltero­dine (4 mg); or a place­bo for eight weeks. In the sec­ond stage, pa­tients were giv­en ei­ther vibegron (100 mg); toltero­dine (4 mg); a com­bi­na­tion vibegron (100 mg) and toltero­dine (4 mg); or place­bo for four weeks. The ob­jec­tive of the study was to eval­u­ate the safe­ty and ef­fi­ca­cy of vibegron as a once-dai­ly oral treat­ment for OAB pa­tients, re­gard­less of urge in­con­ti­nence.

Kei­th Katkin, Urovant CEO

Pa­tients giv­en the high­er dos­es of vibegron (50 mg and 100 mg) ex­pe­ri­enced sta­tis­ti­cal­ly sig­nif­i­cant de­creas­es in the dai­ly rate of uri­na­tion (p=0.007 and p<0.001 re­spec­tive­ly), ur­gency uri­nary in­con­ti­nence episodes (p<0.001 and p<0.001 re­spec­tive­ly), to­tal in­con­ti­nence episodes (p=0.005 and p=0.007) and ur­gency episodes (p=0.024 and p<0.001) at week 8. In the sec­ond tranche of the study, the ef­fi­ca­cy of vibegron monother­a­py on the pri­ma­ry end­point — re­duc­tion in uri­na­tion — was al­so sta­tis­ti­cal­ly sig­nif­i­cant, the com­pa­ny said.

Toltero­dine, sold as De­trol by Pfiz­er $PFE in the Unit­ed States, be­longs to a class of drugs called an­timus­carinics that are de­signed to in­hib­it mus­cle con­trac­ture and have been the on­ly avail­able treat­ments for decades. But they’re not de­signed specif­i­cal­ly for the blad­der and are as­so­ci­at­ed with a host of un­pleas­ant ad­verse-ef­fects, mak­ing them an un­pop­u­lar choice with pa­tients, es­pe­cial­ly in the el­der­ly.

Tri­al da­ta in­di­cat­ed that the mag­ni­tude of max­i­mal re­spons­es with the two high­er vibegron dos­es was of­ten nu­mer­i­cal­ly greater than toltero­dine, re­searchers wrote in the jour­nal Eu­ro­pean Urol­o­gy. “In ad­di­tion, these re­sults pro­vide the proof of con­cept that con­comi­tant dos­ing of vibegron and an an­timus­carinic can pro­duce mod­est symp­tom im­prove­ment ver­sus an an­timus­carinic alone”, they not­ed.

In the tri­al, all vibegron dos­es were well tol­er­at­ed. The in­ci­dence of dry mouth was high­er with pa­tients giv­en toltero­dine than those on vibegron monother­a­py, Urovant said.

The ex­act cause of OAB is un­known, which makes it dif­fi­cult to treat. More than 30 mil­lion over the age of 40 suf­fer symp­toms in the Unit­ed States ac­cord­ing to Urovant $UROV.

End­points News / Pharm­cube BI­IS18 Con­fer­ence in Shang­hai

Click on the im­age to see the full-sized ver­sion

Urovant is one of Vivek Ra­maswamy’s many “vants”, which are sub­sidiaries un­der the con­glom­er­ate Roivant Sci­ences brand. Last week Ra­maswamy told End­points News at the #BI­IS18 sum­mit that his strat­e­gy can be un­der­stood as an “Al­pha­bet for health­care”, a ref­er­ence to Google co-founder Lar­ry Page’s play­book of set­ting loose Google, YouTube, and even com­pa­nies like Cal­i­co and Ver­i­ly as se­mi-au­tonomous units that are led by the moth­er­ship. To date, Ra­maswamy has raised $3 bil­lion for his com­pa­nies and vibegron is one of 34 pro­grams in the “vant” clin­ic.

Urovant in-li­censed the glob­al rights, in­clud­ing Japan, to vibegron — an oral be­ta-3 adren­er­gic ag­o­nist — from Mer­ck $MRK. Vibegron is al­so be­ing test­ed for the treat­ment of OAB in men with be­nign pro­sta­t­ic hy­per­pla­sia and the treat­ment of pain as­so­ci­at­ed with ir­ri­ta­ble bow­el syn­drome. Mean­while, Urovant’s sec­ond ex­per­i­men­tal treat­ment, hMaxi-K, is a gene ther­a­py for pa­tients with OAB symp­toms who have failed oral phar­ma­co­log­ic ther­a­py.

Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology

ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development

CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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Therapists Marcela Ot'alora and Bruce Poulter are trained to conduct MDMA-assisted psychotherapy. In this reenactment, they demonstrate how they help guide and watch over a patient who is revisiting traumatic memories while under the influence of MDMA. (Photo: Multidisciplinary Association for Psychedelic Studies)

MD­MA, now in Phase III, shows promise as a PTSD treat­ment

The first time Lori Tipton tried MDMA, she was skeptical it would make a difference.

“I really was, at the beginning, very nervous,” Tipton said.

MDMA is the main ingredient in the club drug known as ecstasy or molly. But Tipton wasn’t taking pills sold on the street to get high. She was trying to treat her post-traumatic stress disorder by participating in a clinical trial.

After taking a dose of pure MDMA, Tipton lay in a quiet room with two specially trained psychotherapists. They sat next to her as she recalled some of her deepest traumas, such as discovering her mother’s body after Tipton’s mother killed two people and then herself in a murder-suicide.

Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll

UP­DAT­ED: As­traZeneca’s Imfinzi/treme com­bo strikes out — again — in lung can­cer. Is it time for last rites?

AstraZeneca bet big on the future of their PD-L1 Imfinzi combined with the experimental CTLA-4 drug tremelimumab. But once again it’s gone down to defeat in a major Phase III study — while adding damage to the theory involving targeting cancer with a high tumor mutational burden.

Early Wednesday the pharma giant announced that their NEPTUNE study had failed, with the combination unable to beat standard chemo at overall survival in high TMB cases of advanced non-small cell lung cancer. We won’t get hard data until later in the year, but the drumbeat of failures will call into question what — if any — future this combination can have left.

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Ted Ashburn. Oncorus

Cowen, Per­cep­tive lead $79.5M Se­ries B for 's­tand­out' biotech shep­herd­ing on­colyt­ic virus to clin­ic

As several Big Pharma players secure biotech partners in the oncolytic virus space for new immuno-oncology combos, Cowen and Perceptive Advisors have come out with their own bet on a startup that promises to shine.

The marquee investors are joining MPM, Deerfield, Celgene, Astellas, Arkin and UBS in backing the drug developer, Oncorus, which will now deploy the $79.5 million in Series B cash toward clinical development of its lead program. Other new investors include Surveyor Capital, Sphera Funds, IMM Investment, QUAD Investment Management, UTC Investment, SV Investment Corp and Shinhan Investment-Private Equity, the last five of which are Korean-based funds.

Fu­til­i­ty analy­sis au­gurs de­feat in piv­otal tri­al test­ing of Nu­Cana's lead drug in metasta­t­ic pan­cre­at­ic can­cer

Nearly two years after making its public debut, UK-based NuCana’s mission to make chemotherapies more potent and safer was dealt a blow, after a pivotal study testing its lead experimental drug halted enrollment in a hard-to-treat advanced form of cancer, following a futility analysis.

The drug, Acelarin, is being evaluated for use in metastatic pancreatic cancer patients who were not considered suitable for combination chemotherapy. In the late-stage ACELARATE study — which compared the experimental drug against the chemotherapy gemcitabine — 200 patients had been enrolled by the sponsor, Clatterbridge Cancer Centre, before an analysis from an independent safety and data monitoring panel suggested the study’s main goal would not be met.

UP­DAT­ED: Pay­back? An­a­lysts say Sarep­ta was blind­sided by an FDA re­jec­tion dri­ven by reg­u­la­to­ry re­venge

In one of the least anticipated moves of the year, the FDA has rejected Sarepta’s application for an accelerated approval of its Duchenne MD drug golodirsen after fretting over safety issues.

In a statement that arrived after the bell on Monday, Sarepta explained the CRL, saying:

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