FDA sends No­vaVax back to the clin­ic; Stoke Ther­a­peu­tics sets terms for up­sized $101M IPO

→ A be­lea­guered No­vaVax has run in­to a ma­jor hur­dle on its RSV vac­cine’s quest to mar­ket. The FDA is ask­ing the com­pa­ny to con­duct a whole new Phase III tri­al to con­firm that ResVax can pro­tect in­fants from “med­ical­ly sig­nif­i­cant RSV dis­ease” if their moth­ers were vac­ci­nat­ed. While it’s sort­ing out the sit­u­a­tion in the US and Eu­rope, No­vaVax said it will con­tin­ue work­ing with its back­ers at the Bill & Melin­da Gates Foun­da­tion to in­tro­duce the vac­cine in low to mid­dle in­come coun­tries.

Cal­lid­i­tas Ther­a­peu­tics and Ever­est Med­i­cines have en­tered in­to a li­cense agree­ment to de­vel­op and com­mer­cial­ize Cal­lid­i­tas’ lead drug can­di­date, Ne­fe­con — which is cur­rent­ly be­ing run in a glob­al Phase III clin­i­cal tri­al for pa­tients with chron­ic au­toim­mune kid­ney dis­ease IgA Nephropa­thy (IgAN) — in Greater Chi­na and Sin­ga­pore. Swe­den-based Cal­lid­i­tas will re­ceive an up­front pay­ment of $15 mil­lion with po­ten­tial mile­stones up to $106 mil­lion.

Di­a­some Phar­ma­ceu­ti­cals, which is de­vel­op­ing He­pa­to­cyte Di­rect­ed Vesi­cle (HDV) tech­nol­o­gy that is de­signed to be mixed with any com­mer­cial­ly avail­able in­sulin to lessen the in­stances of blood glu­cose highs and lows in pa­tients liv­ing with di­a­betes, on Mon­day post­ed a snap­shot of da­ta from a six-month mid-stage study test­ing the drug, HDV-L, at the Amer­i­can Di­a­betes As­so­ci­a­tion meet­ing.

“In pa­tients with high­er base­line HbA1C, HDV-L led to a HbA1C re­duc­tion equiv­a­lent to con­ven­tion­al Lispro treat­ment, but these HDV-L treat­ed sub­jects re­duced their meal­time in­sulin dose by 25% and ex­pe­ri­enced a trend to­wards a 73% re­duc­tion in time spent in hy­po­glycemia. Pa­tients with low­er base­line HbA1C lev­els did not change their bo­lus meal­time in­sulin dos­es and spent in­creased time in hy­po­glycemia. These re­sults are con­sis­tent with the core hy­poth­e­sis that im­proved liv­er glu­cose stor­age at meal­time should re­duce both pe­riph­er­al glu­cose ex­po­sure and pe­riph­er­al in­sulin re­quire­ments,” said David Klonoff, the prin­ci­pal in­ves­ti­ga­tor of the study, in a state­ment.

→ Pre­clin­i­cal biotech Stoke Ther­a­peu­tics has bro­ken out the terms of its IPO. The Bed­ford, Mass­a­chu­setts-based com­pa­ny plans to of­fer 6.7 mil­lion shares in the price range of $14 to $16 to raise $101 mil­lion, it said in a fil­ing Fri­day. The com­pa­ny plans to list un­der the sym­bol $STOK. 

→ The genome se­quenc­ing ex­perts at Per­son­alis have al­so an­nounced an IPO. The com­pa­ny plans to of­fer 6.7 mil­lion shares in the price range of $14 to $16 to raise $100 mil­lion, it said in a fil­ing Fri­day and will trade as $PSNL. 

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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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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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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Levi Garraway. Broad Institute via Youtube

Roche raids Eli Lil­ly for its next chief med­ical of­fi­cer as San­dra Horn­ing plans to step down

We found out Monday morning where Levi Garraway was headed after he left Eli Lilly as head of oncology R&D a few days ago. Roche named Garraway as their new chief medical officer, replacing Sandra Horning, who they say is retiring from the company.

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AveX­is sci­en­tif­ic founder was axed — and No­var­tis names a new CSO in wake of an ethics scan­dal

Now at the center of a storm of controversy over its decision to keep its knowledge of manipulated data hidden from regulators during an FDA review, Novartis CEO Vas Narasimhan has found a longtime veteran in the ranks to head the scientific work underway at AveXis, where the incident occurred. And the scientific founder has hit the exit.

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