FDA Ad­Com rec­om­mends GSK’s an­ti-BC­MA drug for ap­proval 12-0 de­spite sear­ing in-house re­view — does it mat­ter?

The FDA may have ripped Glax­o­SmithK­line for the safe­ty of its an­ti-BC­MA drug, but that didn’t stop an ad­vi­so­ry com­mit­tee from vot­ing 12-0 to rec­om­mend ap­proval for the mul­ti­ple myelo­ma treat­ment.

The de­ci­sion is in line with the com­mit­tee’s long-run­ning pref­er­ence to ap­prove can­cer drugs that show enough ef­fi­ca­cy and not over­whelm­ing safe­ty con­cerns, with the goal of giv­ing on­col­o­gists as many tools to work with as pos­si­ble as they treat in­di­vid­ual pa­tients. Yet while it sig­naled a strong like­li­hood of FDA ap­proval — and boost­ed GSK’s stock by a nifty 1.6% — the vote still leaves open the ques­tion of how use­ful the drug will ul­ti­mate­ly be and, ac­cord­ing­ly, how well it will sell for a com­pa­ny try­ing to claw back in­to the can­cer ther­a­peu­tics busi­ness.

The case for the ther­a­py, an an­ti­body drug con­ju­gate called be­lan­tam­ab, is sim­ple: It had a 31% re­sponse rate in mul­ti­ple myelo­ma pa­tients who had failed mul­ti­ple oth­er lines of ther­a­pies, in­clud­ing the J&J block­buster Darza­lex.

The case against the drug is al­so sim­ple: The FDA con­clud­ed that GSK, run­ning sin­gle-arm tri­als, failed to prove it helps pa­tients live longer.  And the down­sides were sig­nif­i­cant: Near­ly a fifth of pa­tients in one tri­al saw their vi­sion de­cline to lev­els at which they would not legal­ly be al­lowed to dri­ve in one state. One pa­tient on the ap­pli­ca­tion dose and two pa­tients on the high­er dose be­came legal­ly blind. “[I]t is not clear that the ben­e­fits out­weigh the risk of oc­u­lar tox­i­c­i­ty,” the FDA con­clud­ed in its re­view last week.

All that may not be enough to dra­mat­i­cal­ly cur­tail the drug’s com­mer­cial po­ten­tial if it weren’t for the deep well of ex­per­i­men­tal ther­a­pies that have shown far bet­ter re­sults in the clin­ic and are now near­ing ap­proval.

GSK will like­ly have to com­pete with the an­ti-BC­MA CAR-T ther­a­py from J&J-part­nered Leg­end Biotech that re­cent­ly showed a 100% re­sponse rate and a 69% com­plete re­sponse rate in pa­tients who had un­der­gone a me­di­an of five lines of ther­a­py. Blue­bird and Bris­tol My­ers Squibb have faced em­bar­rass­ing de­lays in bring­ing their so-called “ide-cel” CAR-T ther­a­py to mar­ket, but their da­ta are pret­ty clear: a 71% re­sponse rate and a 31% com­plete re­sponse rate in Phase II.

Then there’s the bis­pecifics from Re­gen­eron, Am­gen and Bris­tol My­ers, which are fur­ther be­hind but have been greet­ed with promise. In a dose-es­ca­la­tion study un­veiled at ASH in De­cem­ber, Re­gen­eron’s REGN5458 in­duced re­spons­es in 4 of 7 pa­tients who had al­ready re­ceived 7 lines of ther­a­py.

Be­lan­tam­ab was meant to be a cen­tral part of GSK’s re­turn to can­cer un­der CEO Em­ma Walm­s­ley, and it may soon get an ap­proval. How much it will mean for pa­tients or for a drug com­pa­ny look­ing to re-ce­ment its name re­mains un­clear.

In­side Track: Be­hind the Scenes of a Ma­jor Biotech SPAC

Dr. David Hung and Michelle Doig are no strangers to the SPAC phenomenon. As Founder and CEO of Nuvation Bio, a biotech company tackling some of the greatest unmet needs in oncology, Dr. Hung recently took the company public in one of this year’s biggest SPAC related deals. And as Partner at Omega Funds, Doig not only led and syndicated Nuvation Bio’s Series A, but is now also President of the newly formed, Omega-sponsored, Omega Alpha SPAC (Nasdaq: OMEG; oversubscribed $138m IPO priced January 6, 2021).

Aduhelm OK 'bit­ter­sweet' for ALS ad­vo­cates; Con­trast­ing Covid-19 vac­cine read­outs; GSK joins TIG­IT bat­tle; 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.

With the busiest days of June now behind us, we’re starting to think seriously about the second half of the year. In August, we have scheduled a special report where Endpoints will compile a list of the 20 most influential R&D executives in biopharma. Know a luminary who should definitely be included? Nominate them now.

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Who are the lu­mi­nar­ies dri­ving the biggest ad­vances in bio­phar­ma R&D? End­points News is ask­ing for your nom­i­na­tions for a spe­cial re­port

In biopharma, driving a drug to market is the ultimate goal — but none of that happens without a strong research and development program. At the most successful companies, those R&D efforts are spearheaded by true innovators in the field who are always looking for that next novel mechanism of action or breakthrough safety profile.

Now, Endpoints News is asking you to tell us who those guiding lights are.

Leen Kawas, Athira CEO

Biotech founder placed on leave as $400M Alzheimer's start­up idea comes un­der scruti­ny

Athira Pharma, the Alzheimer’s biotech that emerged out of obscurity last year and raised nearly $400 million for a dark-horse approach to treating neurodegeneration, has found itself in sudden turmoil.

On Tuesday evening, the company released a terse statement announcing that CEO and founder Leen Kawas had been placed on administrative leave while an independent review board investigated “actions stemming” from her doctoral research at Washington State University. Mark Litton, who joined the company as COO two years ago, will take over day-to-day operations, they said.

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Bris­tol My­ers breaks the bank on Ei­sai's fo­late re­cep­tor ADC drug, lay­ing out more than $3B+ for rights

For years, innovation in oncology has been a crapshoot with Big Pharma — the whales at the table — dropping the big bucks for the key to the next generation of tumor fighters. Bristol Myers Squibb hasn’t exactly made a name for being an innovator in the space, but that doesn’t mean it won’t splash in when it sees a potential winner.

Now, with a massive check in hand, the drugmaker is willing to put its intuition to the test.

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Michael Chambers (L) and John Ballantyne

Dana­her strikes deal to buy boom­ing next-gen man­u­fac­tur­er Alde­vron for $9.6B

Life sciences conglomerate Danaher Corp. $DHR has struck a deal to buy the fast-growing Aldevron, one of the world’s top manufacturers of hotly sought-after plasmid DNA, mRNA and recombinant proteins for the burgeoning world of vaccine and drugmakers pushing some game-changing technologies.

Buyout talks set the stage for Danaher to settle on a $9.6 billion cash pact to acquire the private Fargo, ND-based company — a key supplier for a disruptive new Covid vaccine as well as a host of gene and cell therapy and CRISPR gene editing players — founded by Michael Chambers and CSO John Ballantyne as a crew of 2 back in 1998.

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Spring reg­u­la­to­ry agen­da: What’s com­ing soon-ish from the FDA

The FDA’s lack of a permanent commissioner does not seem to be halting its progress to propose and finalize dozens of new regulations, with the latest batch covering everything from adverse event reporting to supplemental application submissions to annual reports for INDs.

Overall, FDA expects to release more than 40 new proposed regulations and finalize another 24 in the coming months and years.

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Sen. Joe Manchin (D-WV) on a call with HHS Secretary Xavier Becerra, June 9 (Al Drago/Pool via AP Images)

Sen. Manchin to Biden: Don't nom­i­nate Wood­cock for FDA com­mis­sion­er

While Sen. Joe Manchin’s opposition to Janet Woodcock as Biden’s choice for FDA commissioner has been known for months, the West Virginia Democrat spelled out more detailed concerns in a new letter to President Biden on Thursday.

“Dr. Woodcock is not the right person to lead the FDA,” Manchin, an influential centrist, writes, while noting the agency has been without permanent leadership for about five months.

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Med­ic­aid com­mis­sion to Con­gress: In­crease re­bates for ac­cel­er­at­ed ap­proval drugs

As the FDA continues to approve more new drugs under its accelerated approval pathway, the non-partisan Medicaid and CHIP Payment and Access Commission (MACPAC) is telling Congress to increase the statutory Medicaid rebates for such drugs until their clinical benefits have been verified.

Higher rebates for drugs with accelerated approvals, a move opposed by the biopharma industry, would mean lower net prices, lessening their financial burden on the health care system while incentivizing the companies to speed the verification of the drugs’ clinical benefits in confirmatory trials. Once those benefits are confirmed, the companies would return to the lower rebates when the accelerated approval is converted into a full approval, MACPAC suggests.