Ac­ne was a bust, but ex­ces­sive sweat­ing scores FDA OK for Der­mi­ra — shares climb

Af­ter a cat­a­stroph­ic fail­ure in ac­ne that oblit­er­at­ed its stock just months ago, Der­mi­ra $DERM had some re­deem­ing news this morn­ing — the FDA has OK’ed its an­ti-sweat­ing drug gly­copy­rro­ni­um.

Tom Wig­gans

The med now goes un­der the brand name Qbrexza, and it’s a once-dai­ly, top­i­cal treat­ment for peo­ple with ex­ces­sive un­der­arm sweat­ing (al­so called pri­ma­ry ax­il­lary hy­per­hidro­sis). The treat­ment is ap­plied di­rect­ly to the skin, block­ing sweat gland ac­ti­va­tion.

“For years, der­ma­tol­o­gists have been telling us of the need for new treat­ment op­tions that ad­dress pri­ma­ry ax­il­lary hy­per­hidro­sis giv­en the stig­ma and bur­den as­so­ci­at­ed with this con­di­tion,” said Tom Wig­gans, chair­man and chief ex­ec­u­tive of­fi­cer at Der­mi­ra. “From the start, our goal was to de­vel­op an ap­proach that went be­yond mask­ing a per­son’s ex­ces­sive un­der­arm sweat­ing and in­stead fo­cused on treat­ing the con­di­tion in a clin­i­cal­ly mean­ing­ful way. We part­nered with der­ma­tol­o­gists and the FDA dur­ing the de­vel­op­ment stage and lis­tened to the peo­ple who have been liv­ing with this con­di­tion to un­der­stand how they would de­fine a mean­ing­ful ben­e­fit. It is our hope that Qbrexza will not on­ly pro­vide the clin­i­cal ben­e­fit these suf­fer­ers have been seek­ing, but help to re­duce the over­all bur­den on their lives.”

Ever­core in­dus­try an­a­lyst Umer Raf­fat wrote a note this morn­ing about his take on the ap­proval, which was based on re­sults from two Phase III tri­als.

“I have to ad­mit: I was in the more skep­tic camp on un­met need for drug treat­ments in hy­per­hidro­sis … un­til I saw a work­shop host­ed by FDA where I lis­tened to many pa­tient sto­ries about the so­cial stig­ma/un­suc­cess­ful job in­ter­views etc. At present, Der­mi­ra es­ti­mates 5 (mil­lion) hy­per­hidro­sis pa­tients have had a dis­cus­sion with the doc, but on­ly ~15% treat­ed. Der­mi­ra will have to work hard on cre­at­ing this mar­ket.”

Raf­fat not­ed that Botox is cur­rent­ly ap­proved in hy­per­hidro­sis, but the drug has seen no sales growth in the in­di­ca­tion, hold­ing steady at about $70 mil­lion per year. Ever­core is mod­el­ing $200 mil­lion in peak sales, start­ing in 2019 with about $30 mil­lion. Qbrexza is ex­pect­ed to be avail­able na­tion­wide in phar­ma­cies be­gin­ning in Oc­to­ber 2018.

Der­mi­ra’s stock is up 27% on the news. But it’s not yet enough to un­do pre­vi­ous dam­age, as the com­pa­ny’s share price cratered over 65% in March (from $25 per share to $8.59 in a mat­ter of days) fol­low­ing news that its Phase III ac­ne drug failed in tri­als and would be scrapped. To­day in pre-mar­ket trad­ing, it’s at $11.04.

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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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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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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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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Am­gen, Al­ler­gan biosim­i­lar of Roche's block­buster Rit­ux­an clears an­oth­er US piv­otal study 

Novartis $NVS may have given up, but Amgen $AMGN and Allergan $AGN are plowing ahead with their knockoff of Roche’s blockbuster biologic Rituxan in the United States.

Their copycat, ABP 798, was found to have a clinically equivalent impact as Rituxan — meeting the main goal of the study involving CD20-positive B-cell non-Hodgkin’s lymphoma patients. This is the second trial supporting the profile of the biosimilar. In January, it came through with positive PK results in patients with rheumatoid arthritis.

BeiGene and Mus­tang nail down spe­cial FDA sta­tus for top drugs; Roche bags added cov­er­age for Hem­li­bra

→ BeiGene $BGNE is getting a boost in its drive to field a rival to Imbruvica. The FDA has offered an accelerated review to zanubrutinib, a BTK inhibitor that has posted positive results for mantle cell lymphoma. The PDUFA date lands on February 27, 2020. The drug scored breakthrough status at the beginning of the year.

→ BeiGene isn’t the only biopharma company to gain special regulatory status today. Mustang Bio $MBIO and St. Jude Children’s Research Hospital announced that MB-107, a lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency, also known as bubble boy disease, has been granted Regenerative Medicine Advanced Therapy status.