Pan­dem­ic trav­el re­stric­tions force FDA to de­lay a BLA re­view — in­volv­ing a site in­spec­tion in Ko­rea

In an un­usu­al move, the FDA is ap­par­ent­ly de­fer­ring ac­tion on a BLA be­cause it was un­able to con­duct a man­u­fac­tur­ing site in­spec­tion on time.

Spec­trum Phar­ma­ceu­ti­cals’ ap­pli­ca­tion for eflape­gras­tim is now in lim­bo as it works with the FDA to ex­pe­dite its trip to the Han­mi plant in South Ko­rea, where the drug sub­stance is man­u­fac­tured. Giv­en pan­dem­ic trav­el re­stric­tions, reg­u­la­tors were un­able to in­spect the site dur­ing the cur­rent re­view cy­cle, ac­cord­ing to a state­ment put out Mon­day morn­ing — two days af­ter the orig­i­nal PDU­FA date.

“Spec­trum has con­firmed with the FDA that this is not a Com­plete Re­sponse Let­ter,” it added in a re­lease.

As Covid-19 was rear­ing its head in the US — long be­fore the FDA was em­broiled in heat­ed de­bates about po­lit­i­cal in­ter­fer­ence in its drug and vac­cine re­view process — com­mis­sion­er Stephen Hahn had warned that with many staffers work­ing on Covid-19 ac­tiv­i­ties, the agency may slow down its re­view of oth­er drugs. But al­most six months lat­er, this ap­pears to be the first time it had to miss a dead­line due to the pan­dem­ic.

Al­ready chris­tened Rolon­tis, Spec­trum’s drug is a long-act­ing gran­u­lo­cyte colony-stim­u­lat­ing fac­tor (G-CSF) de­signed to boost a type of white blood cells in can­cer pa­tients re­ceiv­ing myelo­sup­pres­sive drugs. That makes it a ri­val to Am­gen’s Neu­las­ta, a $4 bil­lion fran­chise that has come un­der biosim­i­lar threats.

The two Phase III tri­als that went in­to the BLA test­ed eflape­gras­tim in pa­tients with ear­ly-stage breast can­cer who ex­pe­ri­enced neu­trope­nia due to chemother­a­py, sug­gest­ing non-in­fe­ri­or­i­ty and sim­i­lar safe­ty pro­file to Neu­las­ta.

Man­u­fac­tur­ing has been a sore point for the eflape­gras­tim pro­gram. Spec­trum chose to re­scind its mar­ket­ing ap­pli­ca­tion last March af­ter the FDA re­quest­ed more da­ta.

Joe Tur­geon

This time around, though, pres­i­dent and CEO Joe Tur­geon sees the ball in the oth­er court.

“The man­u­fac­tur­ing fa­cil­i­ty is ready for in­spec­tion and we are ea­ger to as­sist the FDA in com­plet­ing their as­sess­ment as soon as pos­si­ble,” he said.

What does this mean for oth­er NDAs or BLAs in the pipeline? We’re still wait­ing to hear back from the FDA, but Sal­im Syed — who’s been track­ing Bris­tol My­ers Squibb’s CVR like a hawk — doesn’t see a read-through to the all-im­por­tant ap­pli­ca­tion for Juno’s BC­MA-tar­get­ing CAR-T. His ra­tio­nale may of­fer some in­sight in­to who’s more or less like­ly to be af­fect­ed:

  1. JCAR017 has both break­through ther­a­py des­ig­na­tion and pri­or­i­ty re­view. Rolon­tis doesn’t. This im­plies that the FDA prob­a­bly has JCAR017 high­er up on the peck­ing or­der for re­views it needs to con­duct.
  2. Both of JCAR017’s man­u­fac­tur­ing fa­cil­i­ties (the JuMP fa­cil­i­ty in Both­well, WA and vec­tor CMO fa­cil­i­ty in TX) are in the US, so prob­a­bly eas­i­er to get to them than to the Rolon­tis South Ko­re­an fa­cil­i­ty.
  3. As far as we can tell, the JuMP fa­cil­i­ty has al­ready been in­spect­ed. We would sus­pect the TX fa­cil­i­ty may have al­ready been in­spect­ed as well or would be pri­or to the JCAR017 PDU­FA of No­vem­ber 16, 2020.

Im­ple­ment­ing re­silience in the clin­i­cal tri­al sup­ply chain

Since January 2020, the clinical trials ecosystem has quickly evolved to manage roadblocks impeding clinical trial integrity, and patient care and safety amid a global pandemic. Closed borders, reduced air traffic and delayed or canceled flights disrupted global distribution, revealing how flexible logistics and supply chains can secure the timely delivery of clinical drug products and therapies to sites and patients.

In fi­nal days at Mer­ck, Roger Perl­mut­ter bets big on a lit­tle-known Covid-19 treat­ment

Roger Perlmutter is spending his last days at Merck, well, spending.

Two weeks after snapping up the antibody-drug conjugate biotech VelosBio for $2.75 billion, Merck announced today that it had purchased OncoImmune and its experimental Covid-19 drug for $425 million. The drug, known as CD24Fc, appeared to reduce the risk of respiratory failure or death in severe Covid-19 patients by 50% in a 203-person Phase III trial, OncoImmune said in September.

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Pascal Soriot (AP Images)

UP­DAT­ED: As­traZeneca, Ox­ford her­ald 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine, tout­ing eas­i­er dis­tri­b­u­tion, low­er price

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.

With an average efficacy of 70%, the headline number may appear less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

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Carl Hansen, AbCellera CEO (University of British Columbia)

From a pair of Air Jor­dans to a $200M-plus IPO, Carl Hansen is craft­ing an overnight R&D for­tune fu­eled by Covid-19

Back in the summer of 2019, Carl Hansen left his post as a professor at the University of British Columbia to go full time as the CEO at a low-profile antibody shop he had founded called AbCellera.

As biotech CEOs go, even after a fundraise Hansen wasn’t paid a whole heck of a lot. He ended up earning right at $250,000 for the year. His compensation package included a loan — which he later paid back — and a pair of Air Jordan tennis shoes. His newly-hired CFO, Andrew Booth, got a sweeter pay packet than that — which included his own pair of Air Jordans.

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Leonard Schleifer, Regeneron CEO (Andrew Harnik/AP)

Trail­ing Eli Lil­ly by 12 days, Re­gen­eron gets the FDA OK for their Covid-19 an­ti­body cock­tail

A month and a half after becoming the experimental treatment of choice for a newly diagnosed president, Regeneron’s antibody cocktail has received emergency use authorization from the FDA. It will be used to treat non-hospitalized Covid-19 patients who are at high-risk of progressing.

Although the Rgeneron drug is not the first antibody treatment authorized by the FDA, the news comes as a significant milestone for a company and a treatment scientists have watched closely since the outbreak began.

Frank Zhang (AP Images)

Plot thick­ens around Leg­end Biotech, Gen­Script with founder Frank Zhang's ar­rest

Two months after Legend Biotech made the startling disclosure that founder and then-CEO Frank Zhang was placed under “residential surveillance,” its parent company revealed that he’s been formally arrested.

Zhang — who, since founding GenScript 18 years ago, has taken the CRO public and groomed Legend Biotech in-house until the J&J-partnered CAR-T player was mature enough for its own Nasdaq listing — is severing his final ties with both. He is resigning as board chair/non-executive director of GenScript and director of Legend.

Eiger nabs the first FDA ap­proval for Prog­e­ria, an ul­tra-rare pre­ma­ture ag­ing dis­ease, with an old Mer­ck drug

Eiger BioPharmaceuticals $EIGR has received an FDA OK for a drug Merck licensed to them at no cost — and now reportedly plans to charge a level consistent with other ultra-rare disease medicines.

The biotech announced Friday evening that regulators had approved lonafarnib for the treatment of Hutchinson-Gilford progeria syndrome, also known as Progeria, as well as some progeroid laminopathies in children older than one year. It’s the first approval granted for the condition, and the drug will be marketed and sold as Zokinvy.

Bahija Jallal (file photo)

TCR pi­o­neer Im­muno­core scores a first with a land­mark PhI­II snap­shot on over­all sur­vival for a rare melanoma

Bahija Jallal’s crew at TCR pioneer Immunocore says they have nailed down a promising set of pivotal data for their lead drug in a frontline setting for a solid tumor. And they are framing this early interim readout as the convincing snapshot they need to prove that their platform can deliver on a string of breakthrough therapies now in the clinic or planned for it.

In advance of the Monday announcement, Jallal and R&D chief David Berman took some time to walk me through the first round of Phase III data for their lead TCR designed to treat rare, frontline cases of metastatic uveal melanoma that come with a grim set of survival expectations.

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Vipin Suri, Catamaran Bio CSO

Cata­ma­ran Bio sails in­to the CAR-NK wa­ters with a $42M launch round

Catamaran Bio’s founding members decided to jump into the CAR-NK game last December over drinks at a trendy bar in Boston.

They were sitting around a table, discussing an MD Anderson study which provided some of the first clinical proof that natural killer (NK) cells can be reengineered to attack tumors, much like CAR-T therapies. It was a “long and lively” discussion, COO Mark Boshar recalls. And by the time it was over, they had a starting point to launch a company.