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.

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

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FDA de­lays de­ci­sion on No­var­tis’ po­ten­tial block­buster MS drug, wip­ing away pri­or­i­ty re­view

So much for a speedy review.

In February, Novartis announced that an application for their much-touted multiple sclerosis drug ofatumumab had been accepted and, with the drug company cashing in on one of their priority review vouchers, the agency was due for a decision by June.

But with June less than 48 hours old, Novartis announced the agency has extended their review, pushing back the timeline for approval or rejection to September. The Swiss pharma filed the application in December, meaning their new schedule will be nearly in line with the standard 10-month window period had they not used the priority voucher.

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Leen Kawas, Athira CEO (Athira)

Can a small biotech suc­cess­ful­ly tack­le an Ever­est climb like Alzheimer’s? Athi­ra has $85M and some in­flu­en­tial back­ers ready to give it a shot

There haven’t been a lot of big venture rounds for biotech companies looking to run a Phase II study in Alzheimer’s.

The field has been a disaster over the past decade. Amyloid didn’t pan out as a target — going down in a litany of Phase III failures — and is now making its last stand at Biogen. Tau is a comer, but when you look around and all you see is destruction, the idea of backing a startup trying to find complex cocktails to swing the course of this devilishly complicated memory-wasting disease would daunt the pluckiest investors.

GSK presents case to ex­pand use of its lu­pus drug in pa­tients with kid­ney dis­ease, but the field is evolv­ing. How long will the mo­nop­oly last?

In 2011, GlaxoSmithKline’s Benlysta became the first biologic to win approval for lupus patients. Nine years on, the British drugmaker has unveiled detailed positive results from a study testing the drug in lupus patients with associated kidney disease — a post-marketing requirement from the initial FDA approval.

Lupus is a drug developer’s nightmare. In the last six decades, there has been just one FDA approval (Benlysta), with the field resembling a graveyard in recent years with a string of failures including UCB and Biogen’s late-stage flop, as well as defeats in Xencor and Sanofi’s programs. One of the main reasons the success has eluded researchers is because lupus, akin to cancer, is not just one disease — it really is a disease of many diseases, noted Al Roy, executive director of Lupus Clinical Investigators Network, an initiative of New York-based Lupus Research Alliance that claims it is the world’s leading private funder of lupus research, in an interview.

Joseph Kim, Inovio CEO (Andrew Harnik, AP Images)

Caught in a stand­off with its con­tract man­u­fac­tur­er over Covid-19 vac­cine, In­ovio files suit in an at­tempt to break free while ri­vals race ahead

Inovio was one of the first vaccine developers to snag attention for a jab that their execs said promised to end the Covid-19 pandemic. Using their own unique DNA tech, CEO Joseph Kim said it took just 3 hours to work it out.

But while rivals are racing to the finish line with ambitious plans to make vast quantities of their vaccines with billions of dollars of deals, Inovio is still stuck at the starting line on manufacturing.

Gilead bol­sters its case for block­buster hope­ful fil­go­tinib as FDA pon­ders its de­ci­sion

Before remdesivir soaked up the spotlight amid the coronavirus crisis, Gilead’s filgotinib was the star experimental drug tapped to rake in billions competing with other JAK inhibitors made by rivals including AbbVie and Eli Lilly.

Now, long term data on the drug — discovered by Gilead’s partners at Galapagos and posted as part of a virtual medical conference — have solidified the durability and safety of filgotinib in patients with rheumatoid arthritis, spanning data from three late-stage trials. An FDA decision on the drug is expected this year.

Covid-19 roundup: Mod­er­na read­ies to en­ter PhI­II in Ju­ly, As­traZeneca not far be­hind; EU ready to ne­go­ti­ate vac­cine ac­cess with $2.7B fund

Moderna may soon add another first to the Covid-19 vaccine race.

In March, the mRNA biotech was the first company to put a Covid-19 vaccine into humans. Next month, they may become the first company to put their vaccine into the large, late-stage trials that are needed to prove whether the vaccine is effective.

In an interview with JAMA editor Howard Bauchner, NIAID chief Anthony Fauci said that a 30,000-person, Phase III trial for Moderna’s vaccine could start in July. The news comes a week after Moderna began a Phase II study that will enroll several hundred people.

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UP­DAT­ED: Es­ti­mat­ing a US price tag of $5K per course, remde­sivir is set to make bil­lions for Gilead, says key an­a­lyst

Data on remdesivir — the first drug shown to benefit Covid-19 patients in a randomized, controlled trial setting — may be murky, but its maker Gilead could reap billions from the sales of the failed Ebola therapy, according to an estimate by a prominent Wall Street analyst. However, the forecast, which is based on a $5,000-per-course US price tag, triggered the ire of one top drug price expert.