Neil Kumar, BridgeBio CEO (QED Therapeutics)

Eye­ing their first ap­proval, Bridge­Bio sends their first drug to the FDA and lands a speedy re­view

Over the last five years, Bridge­Bio has raised about $1 bil­lion, as­sem­bled over 20 dif­fer­ent com­pa­nies, ex­tend­ed net­works in­to Chi­na and Is­rael and scored part­ner­ships with a hand­ful of top aca­d­e­m­ic cen­ters. Now, for the first time, they’ll have a drug be­fore the FDA.

Bridge­Bio an­nounced to­day that the FDA has ac­cept­ed and hand­ed pri­or­i­ty re­view for fos­denopterin, an ex­per­i­men­tal med­i­cine for a rare, ir­re­versible neu­ro­log­i­cal con­di­tion that ap­pears in chil­dren and in­fants.  Picked up two years ago from Alex­ion, it was the lead mol­e­cule for Ori­gin Bio­sciences — a com­pa­ny they built specif­i­cal­ly for the sake of bring­ing it through late-stage tri­als.

The an­nounce­ment comes as Bridge­Bio pre­pares to show off their pipeline at their an­nu­al R&D day and as CEO Neil Ku­mar inch­es clos­er to de­clar­ing their port­fo­lio strat­e­gy a suc­cess. At the very least, it has al­ready wield­ed copy­cats. Atom­wise, a ma­chine learn­ing start­up, has said they want to be the Bridge­Bio of AI. Taysha launched, raised $125 mil­lion pri­vate­ly and got a $157 mil­lion IPO in less than 5 months on the promise of a port­fo­lio ap­proach to gene ther­a­py.

Bridge­Bio picked up the mol­e­cule from Alex­ion in 2018, af­ter the rare dis­ease biotech an­nounced it was re­fo­cus­ing on its core ar­eas and sell­ing off oth­er com­pounds. At the time it had al­ready been grant­ed FDA break­through sta­tus 5 years ear­li­er and put in­to what Bridge­Bio is now mak­ing two piv­otal stud­ies. The dis­ease, known as molyb­de­num co­fac­tor de­fi­cien­cy type A, is ul­tra-rare and af­fects in­fants, so the tri­als were al­most unique­ly small: 7 par­tic­i­pants in one and 5 in the oth­er.

Bridge­Bio says that its sub­mis­sion is based on da­ta from those two stud­ies, but they have not re­leased re­sults from ei­ther one.

The on­ly pub­licly re­leased find­ings on the mol­e­cule come from lim­it­ed stud­ies be­fore Ori­gin’s ac­qui­si­tion. In one com­pas­sion­ate use study in Ger­many, doc­tors ad­min­is­tered the drug to 16 new­borns with Type A and Type B of the dis­ease. Al­though type B pa­tients didn’t re­spond, 8 out of 11 type A pa­tients “rapid­ly im­proved,” with their con­vul­sions van­ish­ing al­most en­tire­ly. Three pa­tients treat­ed ear­ly re­mained seizure free, with “near-nor­mal long-term de­vel­op­ment,” ac­cord­ing to the Lancet study.

Al­though no PDU­FA date was an­nounced, the stan­dard 6-month time frame for pri­or­i­ty re­view would see Bridge­Bio get a de­ci­sion on their first drug by March of 2021. The com­pa­ny is try­ing to make it a big year. Ku­mar said in a state­ment that their sec­ond sub­mis­sion would come lat­er this year for in­fi­gra­tinib, an ex-No­var­tis com­pound Bridge­Bio sub­sidiary QED is now de­vel­op­ing for bile duct can­cer.

That would line up the would-be dis­rup­tor’s first two po­ten­tial ap­provals in a sin­gle year.

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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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.

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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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.”

Tom Riga, Spectrum Pharmaceuticals CEO

Spec­trum im­plodes af­ter a harsh pub­lic slap­down and now a CRL from Richard Paz­dur

The FDA has gone out of its way several times to flatten any expectations for Spectrum’s lung cancer drug poziotinib, including slamming the regulatory door in the biotech’s face four years ago when the their executive crew came calling for a breakthrough drug designation and encouragement from the oncology wing of the FDA.

That stinging early rebuke pointed straight down the path to a corrosive in-house agency review of Spectrum’s attempt to land an accelerated approval for the oral EGFR TKI and a public whipping that included a classic takedown by none other than Richard Pazdur, who slammed the company for “poor drug development” that led to confusion over the dose needed for a slice of NSCLC patients harboring HER2 exon 20 insertion mutations.

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