Shoot­ing for a new stan­dard on os­teoarthri­tis, Mer­ck KGaA out­lines promis­ing da­ta for a po­ten­tial game-chang­er

Mer­ck KGaA’s phar­ma group is tak­ing one more step in a long jour­ney to­ward re­hab­bing its rep in the R&D field this week­end. Com­pa­ny ex­ecs turned up at the ACR sci­en­tif­ic con­fer­ence in San Diego to roll out a promis­ing look at some key Phase II os­teoarthri­tis da­ta — which comes with a crit­i­cal caveat.

The drug is sprifer­min, which Mer­ck Serono has had in its pipeline for the past 13 years af­ter bag­ging it from Zy­mo­Ge­net­ics, now a part of Bris­tol-My­ers Squibb. In the first two-year ef­fi­ca­cy as­sess­ment of the five-year study, re­searchers are now proud­ly point­ing to a slight in­crease of car­ti­lage in the knee, as op­posed to the re­morse­less de­cline that leads to ex­pen­sive knee re­place­ment surgery.

Lu­ciano Ros­set­ti

By Mer­ck re­search chief Lu­ciano Ros­set­ti’s reck­on­ing, this is the first time a drug has ac­tu­al­ly trig­gered a re­ver­sal of this dis­ease — rather than just man­ag­ing the symp­toms of the dis­ease — leav­ing the Ger­man Mer­ck with a po­ten­tial­ly ground­break­ing drug in a field that ac­counts for 5% of the world’s pop­u­la­tion, with 237 mil­lion peo­ple whose lives have been crimped by this dis­ease.

“The most strik­ing re­sponse is the dis­ease re­sponse,” Ros­set­ti tells me. And what’s par­tic­u­lar­ly ex­cit­ing, he added, is that the drug — re­com­bi­nant hu­man FGF-18 pro­tein — is not just block­ing the de­cline of car­ti­lage, but sig­nif­i­cant­ly build­ing nor­mal car­ti­lage in a 2-year time frame.

At the high­est dosage, re­searchers tracked a 0.05 mm in­crease in car­ti­lage thick­ness, with a step down in dose to a 0.04 mm in­crease. Con­sid­er­ing that a 0.04 mm loss puts pa­tients at a great­ly high­er risk of knee re­place­ment, he says, they’re look­ing at a ma­jor treat­ment shift.

If the da­ta are borne out in Phase III, Mer­ck KGaA be­lieves it can line up an­oth­er drug be­hind its big PD-1 drug avelum­ab, part­nered with Pfiz­er in the biggest up­front ever record­ed in biotech. Mer­ck KGaA al­so re­cent­ly won a Eu­ro­pean ap­proval for the MS drug cladrib­ine, ini­tial­ly aban­doned in 2010 af­ter it was spurned by reg­u­la­tors on both sides of the At­lantic. That marked a low point for Mer­ck KGaA, which went for more than a decade with­out a sin­gle ma­jor win on the R&D side of the busi­ness.

Ros­set­ti and the rest of the team want to put the bad old days be­hind the com­pa­ny for good, and sprifer­min could help.

But there’s a big catch. The drug failed to reg­is­ter any kind of no­tice­able im­prove­ment over place­bo on pa­tients’ symp­toms in­volv­ing pain or func­tion, an es­sen­tial part of the sec­ondary end­point pro­file.

Just get­ting an in­jec­tion of ei­ther place­bo or drug in the knee had a big im­pact, says Ros­set­ti, mak­ing it im­pos­si­ble to tease out the drug’s ef­fect. They now have to see if reg­u­la­tors are open to us­ing a mark­er on car­ti­lage thick­ness rather than an ef­fect on symp­toms as a Phase III pri­ma­ry end­point.

There is no set plan for Phase III, he adds, with up­com­ing reg­u­la­to­ry ex­changes need­ed to sort out tri­al de­sign and length. You can be sure it won’t be easy or quick, though. Glob­al­Da­ta re­cent­ly es­ti­mat­ed that the drug could hit the mar­ket in 2021, with fair­ly lim­it­ed near-term com­mer­cial prospects in a mar­ket dom­i­nat­ed by cheap gener­ics.

Still, it’s for­ward progress for Mer­ck KGaA in the clin­ic. And that is big.

Sarep­ta was stunned by the re­jec­tion of Vyondys 53. Now it's stun­ning every­one with a sur­prise ac­cel­er­at­ed ap­proval

Sarepta has a friend in the FDA after all. Four months after the agency determined that it would be wrong to give Sarepta an accelerated approval for their Duchenne MD drug golodirsen, regulators have executed a stunning about face and offered the biotech a quick green light in any case.

It was the agency that first put out the news late Thursday, announcing that Duchenne MD patients with a mutation amenable to exon 53 skipping will now have their first targeted treatment: Vyondys 53, or golodirsen. The biotech said the new drug has been priced on par with their other marketed drug, Exondys 51 — which for an average patient costs about $300,000 per year, but since pricing is based on weight, that sticker price can even cross $1 million.

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UP­DAT­ED: Sanofi CEO Hud­son lays out new R&D fo­cus — chop­ping di­a­betes, car­dio and slash­ing $2B-plus costs in sur­gi­cal dis­sec­tion

Earlier on Monday, new Sanofi CEO Paul Hudson baited the hook on his upcoming strategy presentation Tuesday with a tell-tale deal to buy Synthorx for $2.5 billion. That fits squarely with hints that he’s pointing the company to a bigger future in oncology, which also squares with a major industry tilt.

In a big reveal later in the day, though, Hudson offered a slate of stunners on his plans to surgically dissect and reassemble the portfoloio, saying that the company is dropping cardio and diabetes research — which covers two of its biggest franchise arenas. Sanofi missed the boat on developing new diabetes drugs, and now it’s pulling out entirely. As part of the pullback, it’s dropping efpeglenatide, their once-weekly GLP-1 injection for diabetes.

“To be out of cardiovascular and diabetes is not easy for a company like ours with an incredibly proud history,” Hudson said on a call with reporters, according to the Wall Street Journal. “As tough a choice as that is, we’re making that choice.”

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Paul Biondi (File photo)

Bris­tol-My­er­s' strat­e­gy, BD chief Paul Bion­di ex­it­ed the com­pa­ny -- just ahead of the $74B Cel­gene deal close

Paul Biondi, who orchestrated billions of dollars in deals for Bristol-Myers Squibb over the 5 years he’s run their business development team, has exited the company. Biondi left last month, according to a company spokesperson, in pursuit of another — unspecified — external opportunity.
After 17 years with Bristol-Myers Squibb, Paul Biondi, Head of Strategy and Business Development, decided to leave the company to pursue an external opportunity. The company wishes him well in his new endeavors. Bristol-Myers Squibb  is actively searching for Paul’s successor, and will make an announcement, as appropriate.
Biondi specialized in opportunity at Bristol-Myers. Named to the head of BD in 2015, 13 years after he first joined the company, he played lead roles in striking a variety of licensing deals and buyouts. That includes the sweeping $1.85 billion cancer drug alliance with Nektar, which has come under considerable scrutiny over some recent setbacks.

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Arie Belldegrun at UKBIO 2019. Shai Dolev for Endpoints News

Kite Phar­ma's ex-CEO con­tra­dicts founder as CAR-T patent tri­al heats up, with con­flict­ing val­u­a­tions

Two days after Kite Pharma founder Arie Belldegrun told a federal courtroom that a meeting he had with a Memorial Sloan Kettering executive wasn’t about licensing their immunotherapy patent, Kite’s ex-CEO Aya Jakobovits said it was.

The admission came Tuesday during cross-examination in a patent infringement case that features two of the biggest cancer biotechs and some of the most well-known names in American medicine.

Jakobovits initially said she was not in attendance, didn’t know it was going to happen and didn’t know what took place, according to Law360. But then the plaintiff’s lawyer handed her a document – whose contents were not publicly revealed – and asked again if she learned after-the-fact that the meeting involved a potential patent license.

“Yes,” Jakobovits eventually said.

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On the heels of promis­ing MCL da­ta, Kite hus­tles its 2nd CAR-T to the FDA as the next big race in the field draws to the fin­ish line

Three days after Gilead’s Kite subsidiary showed off stellar data on their number 2 CAR-T KTE-X19 at ASH, the executive team has pivoted straight to the FDA with a BLA filing and a shot at a near-term approval.

In a small, 74-patient Phase II trial reported out at the beginning of the week, investigators tracked a 93% response rate with two out of three mantle cell lymphoma patients experiencing a complete response.

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FDA commissioner Stephen Hahn (AP Images)

Stephen Hahn con­firmed as new FDA chief

The Senate has voted 72-18 to confirm Stephen Hahn as the nation’s 24th commissioner of food and drugs, ushering in a longtime researcher and medical executive that faced little backlash in the confirmation process but was widely seen as a secondary choice for those around the agency.

Four ex-FDA chiefs and dozens of high-profile groups had publicly endorsed for the job acting commissioner Ned Sharpless, who served from Scott Gottlieb’s surprise departure through November 1, when President Donald Trump officially nominated Hahn. Gottlieb also gave his support to Sharpless on Twitter.

House pass­es Pelosi drug pric­ing bill on par­ty-line vote

House Democrats on Thursday fell in line behind Speaker Nancy Pelosi’s (D-CA) drug pricing bill, voting for its passage 230-192, with two Republicans voting for it. The bill is not likely to be taken up in the Senate, and was derided by House Republicans as being too partisan and putting the development of many new treatments at risk.

President Donald Trump, who praised the Pelosi effort when it was first released, later turned against the bill, further ensuring its defeat.

What does $6.9B buy these days in on­col­o­gy R&D? As­traZeneca has a land­mark an­swer

Given the way the FDA has been whisking through new drug approvals months ahead of their PDUFA date, AstraZeneca and their partners Daiichi Sankyo may not have to wait until Q2 of next year to get a green light on trastuzumab deruxtecan (DS-8201).

The pharma giant this morning played their ace in the hole, showing off why they were willing to commit to a $6.9 billion deal — with $1.35 billion in a cash upfront — to partner on the drug.

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KRAS analy­sis vaults Mer­ck­'s flag­ship Keytru­da back in­to the spot­light

The jewel in Merck’s crown — Keytruda — just got more precious.

On Thursday, the US drugmaker broke out an exploratory analysis showing the checkpoint inhibitor helped patients live longer as the first line of defense in patients with a form of non-small cell lung cancer (NSCLC) whose tumors expressed PD-L1, regardless of KRAS status.

KRAS mutations occur in roughly a fifth of patients diagnosed with non-small cell lung cancer, and data suggest that these mutations are associated with poor response to treatment, noted Jonathan Cheng, vice president of oncology clinical research at Merck Research Laboratories, in a statement.