Macro­Gen­ics scores first FDA ap­proval with breast can­cer med Mar­gen­za in third-line pa­tients

In the sus­tain­ably hot on­col­o­gy field, breast can­cer has at­tract­ed some of the biggest play­ers on the mar­ket, in­clud­ing block­buster meds from Roche and Mer­ck. Now, a new play­er has tak­en the field af­ter the FDA gave a thumbs-up to new­com­er Macro­Gen­ics and its HER2 ex­press­ing tu­mor fight­er.

CEO Scott Koenig

Macro­Gen­ics $MGNX scored its first FDA ap­proval Wednes­day for Mar­gen­za (mar­ge­tux­imab-cmkb) to treat metasta­t­ic HER2-pos­i­tive breast can­cer pa­tients who have al­ready re­ceived two or more pri­or HER2 reg­i­mens, in­clud­ing at least one in the metasta­t­ic set­ting, the Mary­land biotech said.

The phar­ma was trad­ing up around 5% Thurs­day morn­ing at rough­ly $26 per share.

The FDA based its re­view on da­ta from the Phase III Sophia tri­al, a head-to-head study pit­ting a com­bi­na­tion of Mar­gen­za and chemo to a com­bo of Roche’s Her­ceptin and chemo. In that 536-pa­tient study, Mar­gen­za post­ed a 24% re­duc­tion in risk of dis­ease pro­gres­sion or death over Her­ceptin, with a me­di­an pro­gres­sion-free sur­vival of 5.8 months on Macro­gen­ics’ drug.

Macro­Gen­ics will re­lease pric­ing in­for­ma­tion clos­er to Mar­gen­za’s March launch date, ex­ec­u­tives said on a call with in­vestors Thurs­day morn­ing. The drug­mak­er is al­so await­ing fur­ther OSS da­ta for Mar­gen­za that is ex­pect­ed to come through in the sec­ond half of 2021.

Mar­gen­za is hop­ing to take its place in a sud­den­ly bustling mar­ket for lat­er-line breast can­cer that in­cludes rough­ly 7,000 U.S. pa­tients per year. Scor­ing a head-to-head win against Her­ceptin, which VP of com­mer­cial strat­e­gy and plan­ning Paul Nor­ris called the “gold stan­dard” and “a high bar” in com­ments to in­vestors, as well as show­ing flex­i­bil­i­ty with chemother­a­py op­tions could help Mar­gen­za rise above the fray.

“One of the crit­i­cal ben­e­fits of the study we did is you can com­bine (Mar­gen­za) with dif­fer­ent chemother­a­pies and give the physi­cian high­er con­fi­dence,” Nor­ris said.

With fol­low-up OSS da­ta on the way, Macro­gen­ics is look­ing in­to its pipeline for a big­ger mar­ket op­por­tu­ni­ty for Mar­gen­za, in­clud­ing as a first-line ther­a­py for gas­tric can­cer and as a com­bo reg­i­men with oth­er can­di­dates in the drug­mak­er’s pipe. The com­pa­ny re­leased da­ta in May show­ing 43% — 6 of 14 — of pa­tients with ad­vanced HER2 tu­mors demon­strat­ed an ob­jec­tive re­sponse to a com­bi­na­tion of mar­ge­tux­imab and MGD013 with 4 con­firmed re­spons­es and ev­i­dence of tu­mor shrink­age that could dri­ve that re­sponse rate even high­er.

“We see this as an en­try point for the use of this mol­e­cule,” CEO Scott Koenig said.

Un­like oth­er drug­mak­ers who build their com­mer­cial team in-house, Macro­Gen­ics will re­ly on an an “end-to-end” com­mer­cial­iza­tion deal with Ever­sana signed in No­vem­ber that will help it rapid­ly roll out with mak­ing a mas­sive in­vest­ment on its own. The com­pa­nies will split com­mer­cial­iza­tion rights in the US and Ever­sana will win a 125% roy­al­ty for sales over its li­cens­ing fees for the pact.

The part­ners will wait un­til March to launch, a de­lay that Macro­Gen­ics por­trayed as need­ed to get the drug’s pack­ag­ing up to speed. Nor­ris said on the call the com­pa­ny de­layed its com­mer­cial­iza­tion plans as dis­cus­sions with the FDA ramped up.

“We’ve been very fo­cused on mak­ing sure we didn’t in­vest too soon too much,” Nor­ris said. “We wait­ed un­til we had stronger in­ter­ac­tions with the FDA be­fore we start­ed to en­gage on the oth­er com­mer­cial­iza­tion ac­tiv­i­ties.”

Ed­i­tor’s Note: This sto­ry has been up­dat­ed to cor­rect an er­ror. Paul Nor­ris, VP of com­mer­cial strat­e­gy plan­ning, spoke on be­half of Macro­Gen­ics on the Thurs­day in­vestor call.

The Price of Re­lief: Ex­plor­ing So­lu­tions to the Ris­ing Costs of On­col­o­gy Drugs

In 2020, The National Cancer Institute estimated about 1.8 million new cases of cancer diagnosed in the United States, while the costs associated with treatment therapies continued to escalate. Given the current legislative climate on drug pricing, it’s never been more important to look at the evolution of drug pricing globally and control concerns of sustainable and affordable treatments in oncology.

A $3B+ peak sales win? Pfiz­er thinks so, as FDA of­fers a tardy green light to its JAK1 drug abroc­i­tinib

Back in the fall of 2020, newly crowned Pfizer chief Albert Bourla confidently put their JAK1 inhibitor abrocitinib at the top of the list of blockbuster drugs in the late-stage pipeline with a $3 billion-plus peak sales estimate.

Since then it’s been subjected to serious criticism for the safety warnings associated with the class, held back by a cautious FDA and questioned when researchers rolled out a top-line boast that their heavyweight contender had beaten the champ in the field of atopic dermatitis — Dupixent — in a head-to-head study.

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Lat­est news on Pfiz­er's $3B+ JAK1 win; Pacts over M&A at #JPM22; 2021 by the num­bers; Bio­gen's Aduhelm reck­on­ing; The sto­ry of sotro­vimab; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

For those of you who attended #JPM22 in any shape or form, we hope you had a fruitful time. Regardless of how you spent the past hectic week, may your weekend be just what you need it to be.

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Robert Califf, FDA commissioner nominee (Graeme Sloan/Sipa USA/Sipa via AP Images)

Rob Califf ad­vances as Biden's FDA nom­i­nee, with a close com­mit­tee vote

Rob Califf’s second confirmation process as FDA commissioner is already much more difficult than his near unanimous confirmation under the Obama administration.

The Senate Health Committee on Thursday voted 13-8 in favor of advancing Califf’s nomination to a full Senate vote. Several Democrats voted against Califf, including Sen. Bernie Sanders and Sen. Maggie Hassan. Several other Democrats who aren’t on the committee, like West Virginia’s Joe Manchin and Ed Markey of Massachusetts, also said Thursday that they would not vote for Califf. Markey, Hassan and Manchin all previously expressed reservations about the prospect of Janet Woodcock as an FDA commissioner nominee too.

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Michel Vounatsos, Biogen CEO (World Economic Forum/Ciaran McCrickard)

Bio­gen vows to fight CM­S' draft cov­er­age de­ci­sion for Aduhelm be­fore April fi­nal­iza­tion

Biogen executives made clear in an investor call Thursday they are not preparing to run a new CMS-approved clinical trial for their controversial Alzheimer’s drug anytime soon.

As requested in a draft national coverage decision from CMS earlier this week, Biogen and other anti-amyloid drugs will need to show “a meaningful improvement in health outcomes” for Alzheimer’s patients in a randomized, placebo-controlled trial to get paid for their drugs, rather than just the reduction in amyloid plaques that won Aduhelm its accelerated approval in June.

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CRO own­er pleads guilty to ob­struct­ing FDA in­ves­ti­ga­tion in­to fal­si­fied clin­i­cal tri­al da­ta

The co-owner of a Florida-based clinical research site pleaded guilty to lying to an FDA investigator during a 2017 inspection, revealing that she falsely portrayed part of a GlaxoSmithKline pediatric asthma study as legitimate, when in fact she knew that certain data had been falsified, the Department of Justice said Wednesday.

Three other employees — Yvelice Villaman Bencosme, Lisett Raventos and Maytee Lledo — previously pleaded guilty and were sentenced in connection with falsifying data associated with the trial at the CRO Unlimited Medical Research.

‘Skin­ny la­bels’ on gener­ics can save pa­tients mon­ey, re­search shows, but re­cent court de­ci­sions cloud fu­ture

New research shows how generic drug companies can successfully market a limited number of approved indications for a brand name drug, prior to coming to market for all of the indications. But several recent court decisions have created a layer of uncertainty around these so-called “skinny” labels.

While courts have generally allowed generic manufacturers to use their statutorily permitted skinny-label approvals, last summer, a federal circuit court found that Teva Pharmaceuticals was liable for inducing prescribers and patients to infringe GlaxoSmithKline’s patents through advertising and marketing practices that suggested Teva’s generic, with its skinny label, could be employed for the patented uses.

UP­DAT­ED: CMS to re­strict cov­er­age of Bio­gen's con­tro­ver­sial Alzheimer's drug to on­ly clin­i­cal tri­als

The Centers for Medicare and Medicaid Services on Tuesday said it will only pay for Biogen’s Aduhelm and other FDA-approved anti-amyloid monoclonal antibodies for Alzheimer’s disease under CMS-approved randomized controlled trials.

The draft national coverage decision, which insurers nationwide are likely to follow, makes clear that CMS will be looking for randomized controlled trials that “demonstrate a clinically meaningful benefit in cognition and function.” That will be a tough task for Biogen, which previously showed conflicting benefits from past Aduhelm trials that were initially cut short due to futility and then resurrected for the accelerated approval.

Susan Galbraith, AstraZeneca EVP, Oncology R&D

Can­cer pow­er­house As­traZeneca rolls the dice on a $75M cash bet on a buzzy up­start in the on­col­o­gy field

After establishing itself in the front ranks of cancer drug developers and marketers, AstraZeneca is putting its scientific shoulder — and a significant amount of cash — behind the wheel of a brash new upstart in the biotech world.

The pharma giant trumpeted news this morning that it is handing over $75 million upfront to ally itself with Scorpion Therapeutics, one of those biotechs that was newly birthed by some top scientific, venture and executive talent and bequeathed with a fortune by way of a bankroll to advance an only hazily explained drug platform. And they are still very much in the discovery and preclinical phase.

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