Warren Huff, Reata CEO

Rea­ta con­tin­ues to hit FDA road­blocks with its Friedre­ich’s atax­ia can­di­date, not­ing reg­u­la­tor 'con­cern­s'

Rea­ta Phar­ma­ceu­ti­cals is fac­ing an FDA wall on its small mol­e­cule can­di­date to treat Friedre­ich’s atax­ia, omavelox­olone. Ac­cord­ing to the com­pa­ny’s Q2 re­port, the FDA might still not be sold on the drug.

“[The FDA] con­tin­ues to have con­cerns re­gard­ing the strength of the ef­fi­ca­cy ev­i­dence. The FDA did not iden­ti­fy any sig­nif­i­cant clin­i­cal safe­ty is­sues. The FDA stat­ed that the safe­ty re­view is on­go­ing, and they are con­tin­u­ing to eval­u­ate the car­diac safe­ty of omavelox­olone in pa­tients with Friedre­ich’s atax­ia. They have not iden­ti­fied any oth­er ma­jor safe­ty con­cerns at this stage of their re­view,” the Q2 re­port said.

Rea­ta re­port­ed that it went to sev­er­al lengths to damp­en the FDA’s mis­giv­ings. The com­pa­ny up­dat­ed its re­sults, which now con­tain both new and lat­er time points in the tri­al, as well as bumped up the num­bers of pa­tients. Rea­ta al­so gave the reg­u­la­tor a new analy­sis and new ev­i­dence in the set­ting of the dis­ease’s patho­phys­i­ol­o­gy. The com­pa­ny said that the new da­ta points got the all-clear and could move for­ward in the NDA process.

“We look for­ward to con­tin­u­ing to work with FDA on its re­view of our NDA for omavelox­olone for the treat­ment of pa­tients with Friedre­ich’s atax­ia, a rare, ge­net­ic, de­bil­i­tat­ing, and de­gen­er­a­tive neu­ro­mus­cu­lar dis­or­der with no ap­proved ther­a­pies. We have sub­mit­ted these ad­di­tion­al da­ta and analy­ses to the FDA and are con­tin­u­ing to pre­pare for the up­com­ing Ad­vi­so­ry Com­mit­tee meet­ing,” said War­ren Huff, Rea­ta’s CEO.

The FDA is ex­pect­ed to make its de­ci­sion by Nov. 30. Rea­ta stressed it’s still wait­ing to re­ceive for­mal min­utes from its mid-cy­cle re­view meet­ing with reg­u­la­tors.

This sit­u­a­tion with omavelox­olone is not the first time Rea­ta and the FDA have crossed swords. In 2021, the FDA’s Car­dio­vas­cu­lar and Re­nal Drugs Ad­vi­so­ry Com­mit­tee vot­ed 13-0 that the agency should not ap­prove its bar­dox­olone methyl cap­sules as a treat­ment to slow the pro­gres­sion of chron­ic kid­ney dis­ease in those with Al­port syn­drome af­ter pan­elists felt that the drug had no ef­fect in treat­ing the dis­ease.

In­vestors were ini­tial­ly bull­ish on the drug as it showed pos­i­tive da­ta in 2019. How­ev­er, in 2020, the FDA de­layed omavelox­olone’s NDA pend­ing com­ple­tion of a sec­ond tri­al, de­lay­ing progress and caus­ing its stock price to drop.

The lat­est news is still not what in­vestors want to hear as the com­pa­ny has been fac­ing a 70% drop in its price $RE­TA since Au­gust of last year. And in ear­ly Mon­day trad­ing, Rea­ta shares were down more than 30%.

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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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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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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Uğur Şahin, BioNTech CEO (ddp images/Sipa USA/Sipa via AP Images)

BioN­Tech bets on dif­fi­cult STING field via small mol­e­cule pact with a Pol­ish biotech

BioNTech is beefing up its relatively thin small molecule pipeline by adding weight to a clinically difficult corner of oncology R&D: STING agonists. To do so, BioNTech is teaming up with a 15-year-old Polish biotech and doling out €40 million, about $41.5 million, to start.

The deal is broken into two parts: First, BioNTech obtains an exclusive global license to develop and market Ryvu Therapeutics’ STING agonist portfolio as small molecules, whether alone or in combination with other agents.

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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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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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.”