In the wake of deep R&D cuts, Bay­er flags safe­ty con­cerns for a top PhI­II drug

Less than a week af­ter Bay­er an­nounced plans to carve down its in­ter­nal R&D group, lay­ing off 900 R&D staffers, the Ger­man com­pa­ny has tipped the mar­ket that one of its cru­cial late-stage de­vel­op­ment ef­forts has run in­to se­ri­ous trou­ble.

Jo­erg Moeller

R&D chief Jo­erg Moeller, who gath­ered the reins in re­search af­ter the de­par­ture of An­dreas Busch in late 2017, told an in­vest­ment group in Lon­don Mon­day that the com­pa­ny was halt­ing its de­vel­op­ment work on vi­laprisan, a uter­ine fi­broid drug that has been billed as a top block­buster prospect at the com­pa­ny.

“For vi­laprisan we have just some days ago put clin­i­cal de­vel­op­ment of our on­go­ing tri­als on hold,” he not­ed, ac­cord­ing to a re­port in Reuters. “That is due to very re­cent safe­ty find­ings in long-term tox­i­col­o­gy stud­ies … We have there­fore de­cid­ed as a pre­cau­tion­ary mea­sure to stop en­roll­ment in­to our on­go­ing pro­gram and eval­u­ate the da­ta.”

I queried Bay­er about the is­sue, and got this back mid-morn­ing:

We re­cent­ly ob­served safe­ty sig­nals in pre-clin­i­cal tox­i­co­log­i­cal stud­ies in ro­dents in­ves­ti­gat­ing long term safe­ty of vi­laprisan – a drug can­di­date we de­vel­op for the treat­ment of uter­ine fi­broids and en­dometrio­sis.

Al­though these are pre­lim­i­nary pre-clin­i­cal find­ings that have not been ob­served in oth­er vi­laprisan stud­ies we de­cid­ed as a pre­cau­tion­ary mea­sure to pause re­cruit­ment in the on­go­ing clin­i­cal stud­ies with vi­laprisan un­til a thor­ough analy­sis of the find­ings has been achieved.

No new pa­tients will be en­rolled in the pro­gram. Pa­tients cur­rent­ly on treat­ment will con­tin­ue their on­go­ing treat­ment course ac­cord­ing to study pro­to­col un­til fur­ther no­tice from Bay­er.

Bay­er went on to note that they had alert­ed the prop­er au­thor­i­ties and will mon­i­tor pa­tients for safe­ty is­sues.

This isn’t the first uter­ine fi­broid ef­fort to raise alarms.

The set­back oc­curs as Al­ler­gan is still deal­ing with the FDA’s re­jec­tion of its uter­ine fi­broid drug Es­mya af­ter Eu­ro­pean reg­u­la­tors cit­ed it for trig­ger­ing a rare liv­er dis­ease. The EMA ear­li­er this year put re­stric­tions on the use of the drug to pro­tect women on the ther­a­py.

Ab­b­Vie, mean­while, has been rolling out Orilis­sa af­ter that drug was first ap­proved for en­dometrio­sis last Ju­ly. The ther­a­py has passed muster for uter­ine fi­broids in Phase III as well, rais­ing hopes that Ab­b­Vie can gain sig­nif­i­cant rev­enue from this drug as they ex­pand the mar­ket.

Bay­er, mean­while, is tran­si­tion­ing its R&D op­er­a­tions as it works out a glob­al re­struc­tur­ing, with plans to cut back on what it spends on in­ter­nal re­search in fa­vor of more al­liances with out­side bio­phar­ma com­pa­nies. A cou­ple of years ago, though, they in­clud­ed vi­laprisan among a group of drugs they tout­ed as like­ly block­busters able to earn more than $6 bil­lion in peak sales.

What hap­pened with the rest?

Their PI3k can­cer drug Aliqopa was first ap­proved a year ago, go­ing in­to a third-line set­ting against heavy com­pe­ti­tion. They have a high-risk heart drug called veri­ciguat, ane­tum­ab rav­tan­sine failed a mid-stage study for mesothe­lioma and no longer fea­tures on the late-stage end of the pipeline re­port and finerenone is in Phase III for kid­ney dis­ease, along­side daro­lu­tamide for can­cer. Daro­lu­tamide hit its goal in Phase III for non-metasta­t­ic cas­tra­tion-re­sis­tant prostate can­cer a cou­ple of months ago, though they held back all the da­ta for a lat­er con­fer­ence.

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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Af­ter a posse of Wall Street an­a­lysts pre­dict a like­ly new win for Sarep­ta, we're down to the wire on a crit­i­cal FDA de­ci­sion

As Bloomberg notes, most of the Wall Street analysts that cover Sarepta $SRPT are an upbeat bunch, ready to cheer on the team when it comes to their Duchenne MD drugs, or offer explanations when an odd setback occurs — as happened recently with a safety signal that was ‘erroneously’ reported last week.

Ritu Baral Cowen
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