FDA to Chi­as­ma on their Phase III re­jec­tion: Come up with a bet­ter plan next time

The FDA re­al­ly, re­al­ly, re­al­ly doesn’t like Chi­as­ma’s Phase III study for its lead drug, oc­treotide. And the biotech isn’t mak­ing much head­way in find­ing a way around the reg­u­la­to­ry road­block with­out sat­is­fy­ing the agency’s de­mand for a new, far bet­ter de­signed Phase III tri­al.

Reg­u­la­tors at the agency bat­ted down the acromegaly drug, dubbed My­Capssa, last April af­ter con­clud­ing that the biotech sim­ply didn’t prove that the pill works with the sin­gle-arm Phase III on of­fer. The oral drug is in­tend­ed to re­place the in­ject­ed ther­a­pies cur­rent­ly used to treat the rare hor­mone ail­ment, in which the pi­tu­itary gland gen­er­ates too much hor­mone. And reg­u­la­tors don’t ap­pear to be too re­cep­tive to any al­ter­na­tive plans that Chi­as­ma has in mind. From the biotech’s 10Q:

In the End of Re­view meet­ing min­utes, the FDA re­it­er­at­ed its strong rec­om­men­da­tion for a ran­dom­ized, dou­ble-blind and con­trolled tri­al, and in­tro­duced the con­cept of a place­bo con­trol as a de­sign el­e­ment that could ad­dress some of the FDA’s con­cerns. While we ac­knowl­edge this feed­back, we con­tin­ue to eval­u­ate var­i­ous po­ten­tial paths for­ward, in­clud­ing a de­ter­mi­na­tion as to whether we can pro­duce da­ta suf­fi­cient to sat­is­fy the FDA of the ef­fi­ca­cy and safe­ty of My­capssa in adult pa­tients with acromegaly. The FDA stat­ed that it con­sid­ers path­ways al­ter­na­tive to its rec­om­men­da­tions to be less ide­al and ul­ti­mate­ly more risky to our ef­forts to se­cure ap­proval of our NDA for oc­treotide cap­sules in acromegaly. The FDA strong­ly rec­om­mend­ed that we work with the FDA to reach a com­mon un­der­stand­ing of ex­pec­ta­tions pri­or to ini­ti­at­ing and ex­e­cut­ing any al­ter­na­tive plans.

Chi­as­ma — a U.S./Is­raeli hy­brid with cor­po­rate head­quar­ters in Waltham, MA — does have an­oth­er Phase III run­ning for the planned Eu­ro­pean ap­pli­ca­tion, but it’s an­oth­er open-la­bel study that al­so doesn’t meet the U.S. agency’s de­mands. Their drug is a mim­ic of so­mato­statin.

In­ter­est­ing­ly, Chi­as­ma had good cause to won­der how the FDA would han­dle the NDA be­fore it sent it in. In its Q2 state­ment, the biotech ac­knowl­edged that Roche had bowed out of its col­lab­o­ra­tion af­ter con­fer­ring with reg­u­la­tors about the study. In a sub­se­quent meet­ing with the FDA:

The FDA al­so in­formed us that, in its view, a sin­gle-arm study was not as in­for­ma­tive as a con­trolled study such as an ac­tive con­trol tri­al us­ing a non-in­fe­ri­or­i­ty de­sign, and that the in­ter­pretabil­i­ty of the ef­fi­ca­cy find­ings we sub­mit­ted in our NDA from our sin­gle-arm study, and whether these find­ings would be ro­bust enough to war­rant ap­proval, would be re­view is­sues as the agency eval­u­at­ed our NDA.

The bot­tom line is that Chi­as­ma — run by long­time biotech vet Mark Leucht­en­berg­er — is caught with dwin­dling re­sources at a time its stock has been beat­en down to a frac­tion of its 12-month high. The com­pa­ny laid off about a third of its staff af­ter the re­jec­tion and has enough mon­ey to make it through 2017. But it al­so ac­knowl­edges that it doesn’t have enough cash for a new study that would meet the FDA’s de­mands.

 

Susan Galbraith, AstraZeneca EVP, oncology R&D, at EUBIO22 (Rachel Kiki for Endpoints News)

Up­dat­ed: As­traZeneca jumps deep­er in­to cell ther­a­py 2.0 space with $320M biotech M&A

Right from the start, the execs at Neogene had some lofty goals in mind when they decided to try their hand at a cell therapy that could tackle solid tumors.

Its founders have helped hone a new approach that would pack in multiple neoantigen targets to create a personalized TCR treatment that would not just make the leap from blood to solid tumors, but do it with durability. And they managed to make their way rapidly to the clinic, unveiling their first Phase I program for advanced tumors just last May.

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Ei­sai’s ex­pand­ed Alzheimer’s da­ta leave open ques­tions about safe­ty and clin­i­cal ben­e­fit

Researchers still have key questions about Eisai’s investigational Alzheimer’s drug lecanemab following the publication of more Phase III data in the New England Journal of Medicine Tuesday night.

In the paper, which was released in conjunction with presentations at an Alzheimer’s conference, trial investigators write that a definition of clinical meaningfulness “has not been established.” And the relative lack of new information, following topline data unveiled in September, left experts asking for more — setting up a potential showdown to precisely define how big a difference the drug makes in patients’ lives.

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iECURE CEO Joe Truitt and founder Jim Wilson

Jim Wil­son biotech iECURE gets fresh $65M to push pe­di­atric liv­er dis­ease gene ther­a­py in­to the clin­ic

Jim Wilson-founded biotech iECURE has wrapped a $65M Series A extension round to get its lead candidate — a gene replacement therapy for a rare inherited liver disease known as ornithine transcarbamylase deficiency, or OTC — into the clinic.

This round was co-led by Novo Holdings and LYFE Capital, followed by initial investors Versant and OrbiMed as well. In September 2021, iECURE raised a $50 million Series A led by the latter two. The new cash infusion will get iECURE through an initial in-human trial, which CEO Joe Truitt told Endpoints News iECURE hopes to read out in 2024.

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Illustration: Assistant Editor Kathy Wong for Endpoints News

Twit­ter dis­ar­ray con­tin­ues as phar­ma ad­ver­tis­ers ex­tend paus­es and look around for op­tions, but keep tweet­ing

Pharma advertisers on Twitter are done — at least for now. Ad spending among the previous top spenders flattened even further last week, according to the latest data from ad tracker Pathmatics, amid ongoing turmoil after billionaire boss Elon Musk’s takeover now one month ago.

Among 18 top advertisers tracked for Endpoints News, only two are spending: GSK and Bayer. GSK spending for the full week through Sunday was minimal at just under $1,900. Meanwhile, German drugmaker Bayer remains the industry outlier upping its spending to $499,000 last week from $480,000 the previous week. Bayer’s spending also marks a big increase from a month ago and before the Musk takeover, when it spent $16,000 per week.

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Vi­a­tris with­draws ac­cel­er­at­ed ap­proval for top­i­cal an­timi­cro­bial 24 years lat­er

After 24 years without confirming clinical benefit, the FDA announced Tuesday morning that Viatris (formed via Mylan and Pfizer’s Upjohn) has decided to withdraw a topical antimicrobial agent, Sulfamylon (mafenide acetate), after the company said conducting a confirmatory study was not feasible.

Sulfamylon first won FDA’s accelerated nod in 1998 as a topical burn treatment, with the FDA noting that last December, Mylan told the agency that it wasn’t running the trial.

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Catal­ent to cut about 200 jobs in Mary­land and Texas

Contract manufacturing company Catalent is cutting about 200 jobs in Maryland and Texas, according to WARN notices, trimming back some of its pandemic-era expansion.

The company will cut 77 jobs by Jan. 15 of next year at a cell therapy facility in Webster, TX, just outside of Houston. In Maryland, the company is reducing staff at two locations, with 82 jobs being eliminated at Catalent’s facility in Gaithersburg, and 53 in Rockville. The layoffs go into effect at those locations on Jan. 14.

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John Carroll with David Chang, Allogene CEO (Credit: Jeff Rumans Photography)

Al­lo­gene takes the stage in New York to go deep on its off-the-shelf cell ther­a­pies — de­clar­ing a first for sol­id tu­mors

NEW YORK — In most cases, a biotech like Allogene would wait until the next big science conference to offer its latest series of snapshots of its data. But most biotechs aren’t like Allogene, where the veteran leaders from Kite garnered a substantial number of kudos over the years for their in-depth reviews of the company’s progress.

So on Tuesday, the leaders at Allogene converged on Manhattan once again to give a detailed breakdown of their latest steps forward, looking to stay out front in the busy off-the-shelf cell therapy arena, keep a clean bill of health on the safety front and prove that they can not only match the autologous pioneers they helped create but make the all-important leap into solid tumors. It’s another step forward in a journey that has a long way to go before even the first big regulatory finish lines appear on the track. But for CEO David Chang, who spent some time with me running through the data ahead of the Tuesday session, it all amounts to forward momentum toward the desired goal.

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UK reg­u­la­tor warns of se­vere eye re­ac­tions fol­low­ing use of Sanofi and Re­gen­eron's Dupix­ent

The UK’s Medicines and Healthcare Regulatory Agency (MHRA) on Tuesday warned of some new and serious eye-related side effects following the use of Sanofi and Regeneron’s atopic dermatitis and asthma treatment Dupixent (dupilumab).

While Dupixent is already associated with cases of conjunctivitis and allergic conjunctivitis, dry eye and with infrequent cases of keratitis and ulcerative keratitis, the MHRA is calling on health professionals to be on the lookout for any of these eye-related side effects as “it is not currently possible to predict who may experience the rarer and most severe ocular adverse reactions, such as ulcerative keratitis.”

Jeb Keiper, Nimbus Therapeutics CEO

PhI­Ib win puts Nim­bus one step clos­er to chal­leng­ing Bris­tol My­ers in TYK2

Bristol Myers Squibb might be the first to clinch an FDA approval for a TYK2 inhibitor, but Nimbus Therapeutics is out to prove that it has the best drug in the class. The biotech says it now has positive mid-stage data to back up those claims — although it’s saving the hard numbers for now.

Topline results from a Phase IIb study involving 259 patients with moderate-to-severe plaque psoriasis showed that Nimbus’ drug, NDI-034858, hit the primary endpoint of helping more patients achieve PASI-75 than placebo at 12 weeks.