CMC holdup de­lays Io­vance fil­ing again — to 2022 — as FDA seeks more as­say da­ta for TIL ther­a­py

Re­mem­ber those po­ten­cy as­says that tripped up Io­vance’s prepa­ra­tions for a BLA fil­ing? It turns out they may take even longer than ex­pect­ed to re­solve.

In a brief state­ment, the com­pa­ny di­vulged that the FDA wants to see more da­ta on the as­says — used to en­sure each batch of its tu­mor-in­fil­trat­ing lym­pho­cyte ther­a­py is up to stan­dard — in ad­di­tion to what it’s al­ready sub­mit­ted. Io­vance is now plan­ning to file for ap­proval in the first half of 2022, which amounts to a de­lay of more than a year from the orig­i­nal time­line of sub­mit­ting in 2020.

Maria Fardis

CEO Maria Fardis high­light­ed the fact that li­fileu­cel, which is ini­tial­ly be­ing de­vel­oped for PD-1 re­frac­to­ry melanoma, rep­re­sent­ed the first cell ther­a­py in sol­id tu­mors to reach the fin­ish line. Al­though an­a­lysts still large­ly ex­pect them to get there even­tu­al­ly, some in­vestors fret­ted, dri­ving shares $IO­VA down 6.79% to $26.97 post­mar­ket.

“As such, TIL prod­uct is com­plex by na­ture and align­ment with FDA on a po­ten­cy as­say is an im­por­tant step to­ward BLA sub­mis­sion,” she said in a state­ment.

De­signed for one-time ad­min­is­tra­tion, Io­vance’s prod­uct con­sists of TILs ex­tract­ed from the pa­tients’ own tu­mors and then ex­pand­ed ex vi­vo — with the help of IL-2. The cur­rent man­u­fac­tur­ing process is de­scribed as a sec­ond-gen­er­a­tion it­er­a­tion to the pi­o­neer­ing ap­proach de­vel­oped by Steven Rosen­berg at the Na­tion­al Can­cer In­sti­tute, with its own fa­cil­i­ty to com­ple­ment some con­tract pro­duc­tion.

In tri­als across melanoma, cer­vi­cal can­cer and even lung can­cer, TIL ther­a­py spurred promis­ing over­all re­sponse rates.

While ex­ecs have pre­vi­ous­ly sug­gest­ed a speedy reg­u­la­to­ry path for the cer­vi­cal in­di­ca­tion, an­a­lysts ex­pect the fil­ing there will al­so be de­layed.

Io­vance is not alone. In a field where it’s of­ten said that your process is your prod­uct, even the lead­ing cell ther­a­py de­vel­op­ers have run in­to their share of chem­istry, man­u­fac­tur­ing and con­trol is­sues. Bris­tol My­ers Squibb and blue­bird learned it the hard way as the FDA slammed them with a sur­prise refuse-to-file let­ter cit­ing a faulty sec­tion on CMC — al­though they still se­cured pri­or­i­ty re­view and ul­ti­mate­ly ap­proval.

“We see this as main­tain­ing reg­u­la­to­ry risk, though our dis­cus­sions with ex­perts fa­mil­iar with po­ten­cy as­say de­vel­op­ment in cell and gene ther­a­pies see res­o­lu­tion of this type of is­sue as a mat­ter of when, not if,” Mizuho an­a­lyst Mara Gold­stein wrote in a note.

Un­lock­ing ESG strate­gies for growth with Gilead Sci­ences

RBC Capital Markets explores what is material in ESG for biopharma companies with the ESG leads at Gilead Sciences. Gilead has long focused on sustainability but recognized a more robust framework was needed. Based on a materiality assessment, Gilead’s ESG strategy today focuses first on drug access and pricing, while also addressing D&I and climate change. Find out why Gilead’s board is “acutely aware” of the contribution that ESG makes to firm’s overall success.

On the hunt for the next Mod­er­na, in­vestors have pumped 'plat­form plays' with cash. Can any­thing slow the run­away train?

It didn’t take an expert to see that mRNA platforms could be huge.

Julie Sunderland partnered with both Moderna and BioNTech about a decade ago while she was running program-related investments for the Bill & Melinda Gates Foundation — and even then the potential for their platforms was obvious despite some well-founded concerns about whether the next-gen tech would ever cross the finish line.

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Sen. Ron Wyden (D-OR) with reporters in the Senate Subway (Graeme Sloan/Sipa via AP Images)

Top Wyden pri­or­i­ty for drug price re­forms: Medicare ne­go­ti­a­tions

As the Biden administration tries to wrangle the details of its infrastructure bill, Senate Finance Committee Chair Ron Wyden (D-OR) took a concrete step forward on drug pricing reforms on Tuesday and unveiled five principles for such reforms, including providing Medicare with the ability to negotiate prices.

“Allowing the Secretary of HHS to negotiate the price Medicare will pay creates a much needed mechanism to achieve fairer prices when the market has failed to do so,” Wyden wrote.

Roche's Alzheimer's drug spurred bio­mark­er changes but no cog­ni­tive im­pact — pa­per; vTv out-li­cens­es for­mer lead pro­gram

More than a year ago, Roche and Eli Lilly were forced to contend with a Phase II/III failure of their respective Alzheimer’s drugs. But while Eli Lilly essentially threw in the towels, Roche wasn’t ready to give up yet.

The Swiss drugmaker now has some biomarker data to spotlight as investigators continue monitoring patients in an open-label extension study.

Dubbed DIAN-TU, the study had been designed to see whether Roche’s gantenerumab and Lilly’s solanezumab could spur a cognitive benefit for a group of patients who had a rare, inherited form of Alzheimer’s that’d tied to early-onset. In short, they didn’t: Both failed the primary endpoint.

Ken Frazier, Merck CEO (Bess Adler/Bloomberg via Getty Images)

Mer­ck­'s Keytru­da blazes a path in first-line cer­vi­cal can­cer, mak­ing good on drug­mak­er's push for ear­li­er pa­tients

In the years since I/O wonder drug Keytruda’s initial approval, Merck has struck an aggressive clinical trial program, which is now firmly focused on earlier lines of therapy. The drugmaker has scored some success there so far, and now it’s earned one of its biggest wins yet.

Keytruda plus chemotherapy with or without background Avastin significantly extended patients’ lives over those dosed with a placebo control in first-line patients with persistent, recurrent or metastatic cervical cancer, according to top-line data from the Phase III KEYNOTE-826 study revealed Tuesday.

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End­points News is now 5 years old. Here's how you can sup­port us for the next phase of growth

Endpoints News turned five years old over the weekend. I wanted to mark the happy occasion by extending our deepest gratitude to Endpoints’ premium subscribers while outlining several other ways to support us as we go broader and get bigger this year and beyond.

Same as any business, we’ve got to create value and get paid for delivering it. So if you depend on Endpoints to stay abreast on biopharma developments, we depend on you too.

Emma Walmsley, GlaxoSmithKline CEO (Credit: Fang Zhe/Xinhua/Alamy Live News)

Ac­tivist in­vestor El­liott in talks with oth­er Glax­o­SmithK­line in­vestors about re­plac­ing Em­ma Walm­s­ley, spin­ning off vac­cine busi­ness — re­port

As Emma Walmsley reveals details this Wednesday about the upcoming split of GlaxoSmithKline’s pharma and consumer units, some tough questions may be coming her way.

Elliott Management, the activist investor that’s previously threatened an attack on GSK (but eventually backed off), is floating more radical changes like replacing the CEO, further breaking up the company and spinning out the vaccine unit, or reviewing the focus on cancer drugs, the Financial Times reported.

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Covid-19 roundup: White House re­veals vac­cine do­na­tion plan; EU opts in for an­oth­er 150 mil­lion dos­es from Mod­er­na

President Joe Biden’s administration on Monday revealed the distribution list for 55 million of the 80 million doses of Covid-19 vaccines America plans to donate to lower-income nations:

Roughly 14 million doses will head to Latin America and the Caribbean, for a list of countries that includes Brazil, Argentina, Dominican Republic, Panama and Costa Rica.
Another 16 million doses are headed to Asia to help the following countries: India, Nepal, Bangladesh, Pakistan, Sri Lanka, Afghanistan, Maldives, Bhutan, Philippines, Vietnam, Indonesia, Thailand, Malaysia, Laos, Papua New Guinea, Taiwan, Cambodia, and the Pacific Islands.

Neu­rona Ther­a­peu­tics is dash­ing to the clin­ic with its cell ther­a­py for epilep­sy — but first, an­oth­er ven­ture round

Six years ago, a band of neuroscientists from the University of California, San Francisco combined decades of research and jumped into the hunt for an off-the-shelf cell therapy. Now, that team is sprinting toward the clinic with a treatment for epilepsy — but first, it’s making a pit stop at the venture well.

Neurona Therapeutics unveiled a $41.5 million round on Tuesday morning, bringing the San Francisco-based biotech’s total raise to $135 million. The cash will be used to advance the company’s pipeline, including an upcoming Phase I/IIa for its lead candidate, NRTX-1001, in chronic focal epilepsy.