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.

Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, which is nearly one in six deaths. Recently, we have seen incredible advances in novel cancer therapies such as immune checkpoint inhibitors, cell therapies, and antibody-drug conjugates that have revamped cancer care and improved survival rates for patients.

Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Ivan Cheung, Eisai US chairman and CEO

Bio­gen, Ei­sai re­fresh amy­loid hy­poth­e­sis with PhI­II show­ing Alzheimer's med slows cog­ni­tive de­cline

In the first look at Phase III data for lecanemab, Eisai and Biogen’s follow-up Alzheimer’s drug to the embattled Aduhelm launch, results show the drug passed with flying colors on a test looking at memory, problem solving and other dementia metrics.

One of the most-watched Alzheimer’s therapies in the clinic, lecanemab met the study’s primary goal on the CDR-SB — Clinical Dementia Rating-Sum of Boxes — giving the biotech the confidence to ask for full approval in the US, EU and Japan by next March 31. The experimental drug reduced clinical decline on the scale by 27% compared to placebo at 18 months, the companies said Tuesday night Eastern time and Wednesday morning in Japan.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,200+ biopharma pros reading Endpoints daily — and it's free.

Gilead names 'k­ing­pin­s' in coun­ter­feit HIV med law­suit

Gilead is mounting its counterfeit drug lawsuit, naming two “kingpins” and a complex network of conspirators who allegedly sold imitation bottles of its HIV meds, some of which ended up in US pharmacies.

The pharma giant on Wednesday provided an update on what it called a “large-scale, sophisticated counterfeiting conspiracy,” accusing two new defendants of “leading and orchestrating” a scheme to sell hundreds of millions of dollars in illegitimate drugs posing as meds such as Biktarvy and Descovy.

Vlad Coric, Biohaven CEO (Photo Credit: Andrew Venditti)

As Amy­lyx de­ci­sion waits in the wings, Bio­haven’s ALS drug sinks (again) in plat­form tri­al

The FDA’s decision on Amylyx’s ALS drug is set to come out sometime Thursday. In a space with few drugs, any approval would be a major landmark.

But elsewhere in the ALS field, things are a bit more tepid.

Thursday morning, Biohaven announced that its drug verdiperstat failed its arm of an ALS platform trial led by Massachusetts General Hospital. According to a press release, the drug did not meet its primary endpoint — improvement on an ALS functional status test — or any key secondary endpoints at 24 weeks. The trial had enrolled 167 patients, giving them either verdiperstat or placebo twice a day.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,200+ biopharma pros reading Endpoints daily — and it's free.

Nooman Haque, head of life sciences and healthcare at Silicon Valley Bank, and John Carroll

I’m head­ed to Lon­don soon for #EU­BIO22. Care to join me?

It was great getting back to a live ESMO conference/webinar in Paris followed by a live pop-up event for the Endpoints 11 in Boston. We’re staying on the road in October with our return for a live/streaming EUBIO22 in London.

Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.

Work taking place in the clean rooms at Vor (Credit: Vor)

Vor Bio opts to keep man­u­fac­tur­ing op­er­a­tions in-house for de­vel­op­ing stem cell, CAR-T ther­a­pies

While it is not uncommon for a biotech to go down the route of having the product manufactured by a contract organization, one small biotech is looking to keep its card close to its chest.

Vor Biopharma has started manufacturing operations at an in-house facility at its HQ in Cambridge, MA after beginning construction last summer.

According to the biotech, the facility aims to develop Vor’s hematopoietic stem cells (eHSCs) and CAR-T therapies for patients with blood cancers. The site will initially manufacture a clinical supply of its candidate VCAR33allo to support its IND, which is slated to be submitted in the first half of next year. It also plans to transfer the production of VOR33 to the facility. Vor is getting to work quickly as engineering runs for VCAR33allo has started this week.

Tar­sus looks to raise aware­ness of eye­lid mite dis­ease in cam­paign aimed at eye­care spe­cial­ists

Eyelid mite disease may be “gross” but it’s also fairly common, affecting about 25 million people in the US.

Called demodex blepharitis, it’s a well-known condition among eyecare professionals, but they often don’t always realize how common it is. Tarsus Pharmaceuticals wants to change that with a new awareness campaign called “Look at the Lids.”

The campaign and website debut Thursday — just three weeks after Tarsus filed for FDA approval for a drug that treats the disease.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,200+ biopharma pros reading Endpoints daily — and it's free.

Aligos co-founders Leonid Beigelman (L) and Lawrence Blatt

Ali­gos co-founders shoot back at J&J IP com­plaint with one of their own al­leg­ing fraud

This story goes all the way back to 2014.

In November of 2014, Johnson & Johnson acquired Alios BioPharma, an infectious disease biotech that was co-founded by Lawrence Blatt and Leonid Beigelman.

Following J&J’s 2014 acquisition, Blatt and Beigelman would become employees at J&J’s Janssen arm, with Blatt being the global head of infectious diseases and vaccines and Beigelman being Janssen’s VP of medicinal chemistry. But Blatt and Beigelman left Janssen in 2017, starting Aligos one year later.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,200+ biopharma pros reading Endpoints daily — and it's free.

Bill Hinshaw, Axcella CEO

Ax­cel­la touts in­ter­im NASH da­ta but needs cash to stay afloat for 2024 topline read­out

Axcella Therapeutics, with an empty bank account looming in the first quarter of next year and a recent setback in a mid-stage long Covid study, is out with some interim data on its NASH drug.

The biotech isn’t yet saying how the drug performed on the primary endpoint of improvement in the nonalcoholic fatty liver disease (NAFLD) Activity Score (NAS), but the first batch of results has sent shares $AXLA soaring nearly 15% before Thursday’s opening bell.