Bris­tol My­ers Squibb and blue­bird bio re­turn to FDA; Take­da gets a break­through nod

More than two months af­ter the FDA slammed Bris­tol My­ers Squibb and blue­bird bio with a suprise refuse-to-file on their  “ide-cell” CAR-T ther­a­py, the pair of de­vel­op­ers have re­sub­mit­ted their ap­pli­ca­tion.

The new ap­pli­ca­tion comes just in time to keep the con­tin­gent val­ue rights from the Cel­gene deal alive. The “ide-cell” was one of the ther­a­pies Bris­tol My­ers picked up in the mon­ster ac­qui­si­tion and one of three ther­a­pies that have to be ap­proved by ear­ly next year to gain every Cel­gene share­hold­er $9. Mizuho an­a­lyst Sal­im Syed es­ti­mat­ed that Bris­tol My­ers would have to re­sub­mit by Ju­ly 31 — to­mor­row — to get an FDA de­ci­sion on time.

The FDA ini­tial­ly spurned the ap­pli­ca­tion over CMC is­sues. Such is­sues have dogged blue­bird for years, de­lay­ing ther­a­pies that have oth­er­wise looked promis­ing in the clin­ic, in­clud­ing their gene ther­a­py for sick­le cell ane­mia.

Take­da gets a break­through des­ig­na­tion in MDS

Take­da R&D chief Andy Plump has been ea­ger to bur­nish the com­pa­ny’s top drug prospects in the wake of the Shire ac­qui­si­tion and re­or­ga­ni­za­tion. And now he’s get­ting some help from the FDA.

The agency has stamped their “break­through ther­a­py des­ig­na­tion” on pevonedi­s­tat for high­er-risk myelodys­plas­tic syn­dromes, or HR-MDS. This is a NEDD8-ac­ti­vat­ing en­zyme in­hibitor and if it works as planned, will be the first drug for HR-MDS to come along in quite some time.

The agency was won over by Take­da’s Phase II da­ta on the drug.

“High­er-risk MDS is as­so­ci­at­ed with poor prog­no­sis, di­min­ished qual­i­ty of life and a high­er chance of trans­for­ma­tion to acute myeloid leukemia, an­oth­er ag­gres­sive can­cer. The com­bi­na­tion of pevonedi­s­tat and azac­i­ti­dine is a promis­ing ther­a­peu­tic ap­proach with the po­ten­tial to be the first nov­el treat­ment ad­vance­ment for high­er-risk MDS in more than 10 years,” said Christo­pher Arendt, who heads Take­da’s On­col­o­gy Ther­a­peu­tic Area Unit.

An­oth­er pri­or­i­ty re­view for an Ebo­la an­ti­body 

A day af­ter Re­gen­eron an­nounced a near­ly $350 mil­lion con­tract from BAR­DA to sup­ply the gov­ern­ment with their Ebo­la an­ti­body cock­tail, Ridge­back Bio­ther­a­peu­tics an­nounced that the FDA had ac­cept­ed an ap­pli­ca­tion for ap­proval for their Ebo­la an­ti­body and giv­en it pri­or­i­ty re­view.

Ridge­back’s an­ti­body — known as mAb114, an­su­vimab or sim­ply “the NIH an­ti­body” — and Re­gen­eron’s an­ti­body cock­tail were the two drugs that proved ef­fec­tive in last year’s NIH ebo­la tri­al, beat­ing out the ZMapp an­ti­body and the Gilead an­tivi­ral remde­sivir. Un­like Re­gen­eron, Ridge­back did not de­vel­op the an­ti­body but rather li­censed it from the NIH, which had de­vel­oped it in part­ner­ship with the In­sti­tute for Bio­med­ical Re­search in Switzer­land.

mAb114 re­ceived FDA break­through des­ig­na­tion last year. Ridge­back did not dis­close a PDU­FA date. Re­gen­eron is ex­pect­ing a de­ci­sion on their an­ti­body by Oc­to­ber 25.

Geno­cea an­nounces “pos­i­tive” can­cer vac­cine re­sults, stock falls

Neoanti­gen biotech Geno­cea an­nounced what it called “pos­i­tive pre­lim­i­nary re­sults” from its lead can­cer vac­cine, but in­vestors were ev­i­dent­ly unim­pressed, drop­ping the com­pa­ny’s stock $GN­CA 34%, from $4.97 to $3.28.

The com­pa­ny re­port­ed that three of five pa­tients had a RE­CIST re­sponse — an im­prove­ment in a score for sol­id tu­mors — in their Phase I/IIa study. Pa­tients were giv­en check­point in­hibitors plus the neoanti­gen vac­cine. Geno­cea said they saw an “ac­cel­er­a­tion of shrink­age be­yond that” seen in check­point in­hibitors alone, but the com­pa­ny did not in­clude a con­trol group for the tri­al, so it’s dif­fi­cult to draw ma­jor con­clu­sions.

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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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).

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.

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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FDA's con­tro­ver­sial Aduhelm de­ci­sion leaves ALS pa­tients feel­ing spurned

The FDA’s controversial approval of Biogen’s Aduhelm drug for Alzheimer’s disease has been met with fierce resistance from all corners of the biopharma industry, but few seem to be as upset with the decision as ALS patients and advocacy groups.

For all that’s already been written and discussed about the agency’s announcement, from the drug’s exorbitantly high price of $56,000 per year to criticism over lowered standards, ALS patients see something more. ALS patients and associations say they largely regarded Aduhelm’s approval as a bittersweet double standard: happy that those with Alzheimer’s have a new drug available, but questioning how the FDA evaluated Biogen’s drug compared to the experimental programs being studied for their own disease.

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Jim Green­wood opens new pol­i­cy shop, with an eye on drug price con­trols; Mod­er­na, CRISPR, Mus­tang re­ceive tax breaks in Mass­a­chu­setts

Capitol Hill heavyweight Jim Greenwood is taking his decades of experience in the intertwining worlds of biopharma and Congress to the law firm DLA Piper, where he’s opening up a new policy and regulatory practice alongside former Pfizer lawyer Geoffrey Levitt.

As Congress wrangles over how to bring down drug prices, the former BIO CEO told Endpoints News in an interview that his top three priorities will be, “Price controls, price controls and price controls.”

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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Christian Hogg, Hutchmed CEO

Hutchmed files for $600M+ IPO in Hong Kong as lead on­col­o­gy drug su­r­u­fa­tinib awaits FDA's good graces

In oncology, a flush of Chinese-developed drugs has the biopharma industry rethinking the poles of power in R&D as the blossoming nation continues to make a name for itself and pick up bundles of cash in the process. Now, as its lead drug faces a pivotal FDA review, the company formerly known as Chi-Med is planting its flag on home soil with a massive public offering.

Hutchmed — recently renamed from Chi-Med, or Hutchison China MediTech — will look to raise $603 million as part of a Hong Kong IPO that serves as a homecoming of sorts for the Chinese-based oncology player, which has listed on Nasdaq since 2016.