Macro­Gen­ics touts a PhI­II win in a head-to-head with best-sell­er Her­ceptin — shares rock­et high­er

Macro­Gen­ics $MGNX has claimed suc­cess in their Phase III tri­al of their drug mar­ge­tux­imab for metasta­t­ic breast can­cer, putting them on the path to a BLA fil­ing. But they’re not of­fer­ing a lot of de­tail.

The top-line num­ber cleared the bar for sta­tis­ti­cal sig­nif­i­cance, but not by a wide mar­gin.

Scott Koenig

In a head-to-head with Her­ceptin (trastuzum­ab), the biotech re­ports that their drug won out in pro­long­ing pro­gres­sion-free sur­vival among heav­i­ly treat­ed third- and fourth-line HER2+ pa­tients. The p-val­ue on the pri­ma­ry end­point was 0.033 with a 24% risk re­duc­tion for dis­ease pro­gres­sion. A much bet­ter score of 0.005, though, was record­ed for the ma­jor­i­ty of pa­tients in the tri­al who were car­ri­ers of the CD16A (FcγRI­I­Ia) 158F al­lele, which is linked to re­sis­tance to Her­ceptin.

This was a close­ly watched cat­a­lyst for Macro­Gen­ics. Its shares shot up 166% on the top-line re­port Wednes­day morn­ing. Jonathan Chang at Leerink not­ed:

Many in­vestors we have spo­ken with ex­pect­ed the study to be neg­a­tive and we be­lieve lit­tle val­ue for Her2+ mBC was baked in­to the MGNX stock. We mod­eled a 60% prob­a­bil­i­ty of suc­cess.

We don’t know yet how those num­bers trans­late to months of added PFS, or the over­all sur­vival rate. Re­searchers are still pon­der­ing OS. The more ex­act da­ta, which will tell an­a­lysts a lot more about the mar­ket po­ten­tial for this drug, will be un­veiled at a lat­er can­cer con­fer­ence.

Re­searchers will be tak­ing the da­ta from the SOPHIA study, which en­rolled 536 pa­tients, to reg­u­la­tors some­time in the sec­ond half of this year.

The com­pa­ny has been tout­ing their an­ti­body’s en­gi­neer­ing, say­ing the bet­ter Fc do­main can en­gage the im­mune sys­tem. The drug is de­signed to tar­get the HER2 on­co­pro­tein. And there’s a long way to go on the de­vel­op­ment ef­fort.

“We look for­ward to ad­di­tion­al op­por­tu­ni­ties to de­vel­op mar­ge­tux­imab in oth­er HER2-pos­i­tive breast and gas­tric can­cer pop­u­la­tions,” Macro­Gen­ics CEO Scott Koenig re­marked. 

A side ben­e­fi­cia­ry in to­day’s news is Zai Lab, $ZLAB the Chi­nese biotech that in-li­censed re­gion­al rights to the drug for a low up­front pay­ment. Jef­feries not­ed:

Re­call, ZLAB in-li­censed marge 12/2/18 for an up­front of $25M for rights to breast and gas­tric can­cer in Chi­na (our note here). Ex­pec­ta­tions for the SOPHIA ph.III read­out were low, and we be­lieve ZLAB took a cal­cu­lat­ed risk/ben­e­fit ap­proach w/ marge that led to best-case sce­nario, giv­en to­day’s news.

The biotech hit a stum­bling block at the end of last year. Leery about signs of liv­er tox­i­c­i­ty among pa­tients tak­ing a B7-H3 × CD3 bis­pe­cif­ic un­der de­vel­op­ment at Macro­Gen­ics, the FDA slapped a par­tial hold on the ear­ly-stage pro­gram.

UP­DAT­ED: Mer­ck pulls Keytru­da in SCLC af­ter ac­cel­er­at­ed nod. Is the FDA get­ting tough on drug­mak­ers that don't hit their marks?

In what could be an early shot in the battle against drugmakers that whiff on confirmatory studies to support accelerated approvals, the FDA ordered Bristol Myers Squibb late last year to give up Opdivo’s approval in SCLC. Now, Merck is next on the firing line — are we seeing the FDA buckling down on post-marketing offenders?

Merck has withdrawn its marketing approval for PD-(L)1 inhibitor Keytruda in metastatic small cell lung cancer as part of what it describes as an “industry-wide evaluation” by the FDA of drugs that do not meet the post-marketing checkpoints on which their accelerated nods were based, the company said Monday.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 102,400+ biopharma pros reading Endpoints daily — and it's free.

Michael Shpigelmacher

Khosla joins bet on un­con­ven­tion­al start­up look­ing to send drug de­liv­er­ing ro­bots in­to the brain

When Michael Shpigelmacher started the project, he knew he’d have to fund it himself. Every other effort of its kind was academic, rejected as too risky by investors.

Shpigelmacher, a robotics geek and entrepeneur who had drifted into consulting for pharma, wanted to build the real-life equivalent of technology from the 1960s film Fantastic Voyage, the one where a submarine crew is shrunk to “about the size of a microbe” and sent on a mission to repair a scientist’s brain. He scanned the literature, found the lab that was working on the most advanced project — at the Max Planck Institute in Germany, it turned out — and started funding them with money from his own account, along with some seed cash from friends and family.

Paul Sekhri

The next big biotech su­per­star? Paul Sekhri has some thoughts on that

It occasionally occurs to Paul Sekhri that if they pull this off, his company will be on the front page of the New York Times and a lead story in just about every major news outlet on the planet. He tries not to dwell on it, though.

“I just want to be laser-focused on getting to that point,” Sekhri says, before acknowledging, “Yes, it absolutely crossed my mind.”

Sekhri, a longtime biopharma executive with tenures at Sanofi and Novartis, is now entering year three as CEO of eGenesis, the biotech that George Church protégé Luhan Yang founded to genetically alter pigs so that they can be used for organ transplants. He led them through one megaround and has just closed another, raising $125 million from 17 different investors to push the first-ever (humanized) pig to human transplants into the clinic.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 102,400+ biopharma pros reading Endpoints daily — and it's free.

Amit Munshi, Arena

One of Are­na's top drugs flops in a PhI­Ib study for IBS pain. But re­searchers tease out a pos­si­ble path for­ward as CEO ex­plores 's­trate­gic op­tion­s'

Four years ago, when Arena CEO Amit Munshi cut its ties to a troubled weight drug and doubled down on the pipeline, a cannabinoid receptor 2 agonist figured prominently in the biotech’s future. On Tuesday evening, however, Munshi’s high hopes for the drug took a nasty hit after it failed a Phase IIb study for patients with irritable bowel syndrome pain.

Put through a randomized pace with 273 patients, researchers said it flat failed the primary endpoint among the large group with abdominal pain. But they quickly went on to highlight subgroup data, always a tricky and controversial ploy, where they spotlighted a positive p value for patients with moderate to severe pain who received the high dose of the drug — one of 3 provided in the study.

Bob Nelsen (Photo by Michael Kovac/Getty Images)

With stars aligned and cash in re­serve, Bob Nelsen's Re­silience plans a makeover at 2 new fa­cil­i­ty ad­di­tions to its drug man­u­fac­tur­ing up­start

Bob Nelsen’s new, state-of-the-art drug manufacturing initiative is taking shape.

Just 3 months after gathering $800 million of launch money, a dream team board and a plan to shake up a field where he found too many bottlenecks and inefficiencies for the era of Covid-19, Resilience has snapped up a pair of facilities now in line for a retooling.

The company has acquired a 310,000-square-foot plant in Boston from Sanofi along with a 136,000-square-foot plant in Ontario to add to a network which CEO Rahul Singhvi says is just getting started on building his company’s operations up. The Sanofi deal comes with a contract to continue manufacturing one of its drugs.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 102,400+ biopharma pros reading Endpoints daily — and it's free.

CEO Marco Taglietti (Scynexis)

'N­ev­er been more ur­gent:' Scynex­is looks to tack­le su­per­bug cri­sis with late-stage read­out for an­ti­fun­gal hope­ful

As the superbug crisis heats up around the world, Scynexis says it has new data from two interim analyses that prove its antifungal has the potential to treat a broad range of infections.

“The need for new anti-infectives capable of fighting the most resistant pathogens has never been more urgent as we confront the ongoing COVID-19 global pandemic,” CEO Marco Taglietti said in a statement.

A spot­light schiz­o­phre­nia drug in Neu­ro­crine's $2B Take­da deal flunks its first ma­jor test. But it's not giv­ing up yet

When Takeda spun out a pipeline of experimental psychiatry drugs to Neurocrine in a $2 billion deal amid a post-merger shakeout, R&D chief Andy Plump described the therapies as “very interesting but still difficult.”

On Tuesday, we got some idea of how difficult.

San Diego-based Neurocrine revealed that one of the three spotlight clinical programs they’d acquired failed the primary endpoint in a Phase II trial for schizophrenia, registering a negative outcome on the change from baseline in the positive and negative syndrome scale/negative symptom factor score (PANSS NSFS).

Af­ter bail­ing on Covid-19 vac­cines, Mer­ck will team up with J&J to pro­duce its shot as part of un­usu­al Big Phar­ma pact

Merck took a big gamble when it opted to jump into the Covid-19 vaccine race late, and made an equally momentous decision to back out in late January. Now, looking to chip in on the effort, Merck reportedly agreed to team up with one of the companies that has already crossed the finish line.

President Joe Biden on Tuesday is expected to announce a partnership between drugmakers Merck and Johnson & Johnson to jointly produce J&J’s recombinant protein Covid-19 vaccine that received the FDA’s emergency use authorization Saturday, the Washington Post reported.

Ab­b­Vie tees up a biotech buy­out af­ter siz­ing up their Parkin­son's drug spun out of Ke­van Shokat's lab

AbbVie has teed up a small but intriguing biotech buyout after looking over the preclinical work it’s been doing in Parkinson’s disease.

The company is called Mitokinin, a Bay Area biotech spun out of the lab of UCSF’s Kevan Shokat, whose scientific explorations have formed the academic basis of a slew of startups in the biotech hub. One of Shokat’s PhD students in the lab, Nicholas Hertz, co-founded Mitokinin using their lab work on PINK1 suggesting that amping up its activity could play an important role in regulating the mitochondrial dysfunction contributing to Parkinson’s disease pathogenesis and progression.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 102,400+ biopharma pros reading Endpoints daily — and it's free.