Pfiz­er’s block­buster safe­ty is­sue jumps the At­lantic as EMA launch­es its own re­view of Xel­janz

Pfiz­er’s Xel­janz woes have spread to Eu­rope.

Af­ter the FDA an­nounced ear­li­er this week that it is in­ves­ti­gat­ing the in­creased risk of blood clots and death linked to their block­buster JAK in­hibitor seen in a post-mar­ket­ing study, the EMA says they’ll now make their own as­sess­ment on the safe­ty of the drug.

“EMA is as­sess­ing the re­sults of the study and will con­sid­er what reg­u­la­to­ry ac­tion is need­ed,” the agency said in a state­ment sent to End­points News. The EMA added that pa­tients should con­sult a doc­tor if they have any ques­tions.

Eu­ro­pean reg­u­la­tors had proved much tougher than the FDA on Xel­janz, wait­ing un­til 2017 to ap­prove the drug for rheuma­toid arthri­tis af­ter de­lay­ing the roll­out for more safe­ty da­ta — 6 years af­ter the FDA OK.

The drug en­tered the mar­ket with a black box warn­ing of a high­er risk of in­fec­tions, but the phar­ma gi­ant set off alarm bells when re­searchers said they were tak­ing peo­ple off the 10 mg dose used in their as­sess­ment of the drug’s safe­ty among pa­tients with an added car­dio risk af­ter the tri­al mon­i­tors con­clud­ed that the drug was as­so­ci­at­ed with high­er rates of blood clots and death.

Both the FDA and EMA note that the rec­om­mend­ed dose for Xel­janz in RA is 5 mg twice a day, but the drug is ap­proved at 10 mg for ul­cer­a­tive col­i­tis. Eli Lil­ly al­so faced its own chal­lenges af­ter the FDA ini­tial­ly re­ject­ed its JAK in­hibitor baric­i­tinib, fi­nal­ly ap­prov­ing it as Olu­mi­ant last year at a low­er dose than it had sought.

The new safe­ty alert comes at a bad time for Pfiz­er, which is de­pend­ing on ris­ing sales of its block­buster drug at a time when US law­mak­ers have been ham­mer­ing hard against an­nu­al price hikes. Oth­er JAKs are in the late-stage pipeline with their own claims on safe­ty and ef­fi­ca­cy that will now face fur­ther scruti­ny.

On the oth­er hand, any new RA ther­a­py en­ter­ing the field with a bet­ter safe­ty pro­file will be look­ing at a se­ri­ous­ly dis­rupt­ed mar­ket.

Patrick Soon-Shiong at the JP Morgan Healthcare Conference, Jan. 13, 2020 (David Paul Morris/Bloomberg via Getty Images)

Af­ter falling be­hind the lead­ers, dissed by some ex­perts, biotech show­man Patrick Soon-Sh­iong fi­nal­ly gets his Covid-19 vac­cine ready for a tri­al. But can it live up to the hype?

In January, when dozens of scientists rushed to start making a vaccine for the then-novel coronavirus, they were joined by an unlikely compatriot: Patrick Soon-Shiong, the billionaire doctor most famous for making big, controversial promises on cancer research.

Soon-Shiong had spent the last 4 years on his “Cancer Moonshot,” but part of his project meant buying a small Seattle biotech that specialized in making common-cold vectors, called adenoviruses, to train the immune system. The billionaire had been using those vectors for oncology, but the company had also developed vaccine candidates for H1N1, Lassa fever and other viruses. When the outbreak began, he pivoted.

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Jude Samulski, Marianne De Backer

Bay­er buys a biotech ‘race horse’ with a $4B deal — $2B in cash — aimed at go­ing big in­to gene ther­a­py

In the latest sign that Big Pharma wants a leading place in the push to develop a new generation of cell and gene therapies, Bayer is stepping up today with a $2 billion cash deal to buy out one of the fast-moving pioneers in the field, while adding up to $2 billion more in milestones if the new pharma subsidiary can deliver the goods.

As part of a continuing series of deals engineered by Bayer BD chief Marianne De Backer, the pharma player has snapped up Asklepios, more commonly referred to in more casual fashion as AskBio. And they are paying top dollar for a Research Triangle Park-based company that raised $225 million a little more than a year ago to back the brainchild of Jude Samulski, the gene therapy pioneer out of the University of North Carolina Gene Therapy Center.

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No­var­tis CEO Vas Narasimhan signs off on a $231M deal to try some­thing new in the R&D fight against SARS-CoV-2

Patrick Amstutz was baptized by pandemic fire early on.

He and colleagues attended the notorious Cowen conference in early March that included some of the top Biogen execs who helped trigger a superspreader event in Boston. Heading back to his post as CEO of Molecular Partners in Switzerland, the outbreak was sweeping through Italy, triggering near panic in some quarters and creeping into the voices of people he knew, including one friend on the Italian side of the country.

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Cedric Francois, Apellis CEO (Optum via YouTube)

UP­DAT­ED: So­bi bets $250M cash, about $1B in mile­stones for rights to a C3 ther­a­py be­ing pushed through 5 piv­otal tri­als

A couple years after licensing Novimmune’s emapalumab and turning around a quick FDA OK, Stockholm-based Sobi is betting up to $1.2 billion for rights to another rare disease drug.

The company is shelling out $250 million upfront and adding up to $915 million in milestones for rights to develop and commercialize Apellis Pharmaceuticals’ drug pegcetacoplan outside the US. Together, the companies will see the systemic C3 therapy through five registrational trials in hematology, nephrology and neurology.

Christian Rommel (via Roche)

Bay­er fol­lows R&D deal spree by raid­ing Roche's can­cer group for its new re­search chief

The day after Bayer signed off on a $4 billion deal designed to put the company among the leaders in gene therapy development, the pharma giant has recruited a new chief for its R&D division. And they opted for an expert in the cancer field.

Christian Rommel, Roche’s head of discovery and early-stage oncology development, has been tapped to take over the job. Joerg Moeller, who got the top research post after early- and late-stage development roles were combined 2 years ago, is hitting the exit “to pursue other career opportunities.”

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Once re­ject­ed, Kala's dry eye drug now gains en­try to a field where No­var­tis is groom­ing its own block­buster

When the FDA slapped a rejection on Kala Pharma’s dry eye drug last August, the biotech’s execs promised investors that a third Phase III study — they had already started at that point — would reverse their fortune.

Today they made good on that promise, clinching an approval for Eysuvis, an ocular corticosteroid being positioned as a first-line, short term treatment of dry eye disease.

Boasting a technology invented by Bob Langer out of MIT, Eysuvis is a corticosteroid, loteprednol etabonate, delivered by mucus-penetrating particles. It promises to enhance penetration into target tissue on the ocular surface, achieving an effect quicker than systemic corticosteroids and stronger than over-the-counter eye drops.

Albert Bourla, AP

UP­DAT­ED: Where's the Pfiz­er ef­fi­ca­cy read­out? CEO Bourla says 'soon,' but you're go­ing to have to wait for it

Pfizer CEO Albert Bourla had promised repeatedly that the pharma giant would know if its leading Covid-19 vaccine is effective by the end of this month — now just a few days away.

Instead, the company reported early Tuesday that it has yet to conduct any interim efficacy analyses. And it won’t now until sometime next month.

The news was included in a slide for their Q3 report.

In the morning Q3 call with analysts, Bourla says that they expect efficacy data “soon,” but noted that they wouldn’t be able to say anything until all the administrative work was done on the interim, which would take about a week. And he added that Pfizer isn’t going to say anything else about that hot topic until they have the data in hand.

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Charles Baum, Mirati CEO

UP­DAT­ED: Mi­rati plots a march to the FDA for its KRAS G12C drug, breath­ing down Am­gen’s neck with bet­ter da­ta

Mirati Therapeutics $MRTX took another closely-watched step toward a now clearly defined goal to file for an approval for its KRAS G12C cancer drug adagrasib (MRTX849), scoring a higher response rate than the last readout from the class-leading rival at Amgen but still leaving open a raft of important questions about its future.

Following a snapshot of the first handful of responses, where the drug scored a tumor response in 3 of 5 patients with non-small cell lung cancer, the response rate has now slid to 45% among a pooled group of 51 early-stage and Phase II patients, 43% — 6 of 14 — when looking solely at the Phase I/Ib. Those 14 patients had a median treatment duration of 8.2 months, with half still on therapy and 5 of 6 responders still in response.

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Covid-19 roundup: Sanofi and GSK pledge 200M vac­cine dos­es for a glob­al dis­tri­b­u­tion cam­paign

Sanofi and GSK have agreed to give 200 million doses of their vaccine candidate to the COVAX Facility, which is part of a program set up by CEPI, the WHO and Gavi to equitably distribute vaccines around the world.

The idea behind COVAX is to give all participating countries equal access to vaccines, regardless of income level. As of Oct. 14, more than 180 countries had signed agreements to the COVAX Facility, including France and the UK. China joined earlier this month, pledging to make its vaccines a “global public good.” One country notably off the list is the United States.