Shoshana Shendelman, Applied Therapeutics CEO/founder

Biotech pulls plans to file ac­cel­er­at­ed ap­proval af­ter FDA 'change in di­rec­tion' — shares plum­met

When the FDA lift­ed a clin­i­cal hold on Ap­plied Ther­a­peu­tics’ lead pro­gram in galac­tosemia last Feb­ru­ary, the New York biotech sig­naled that they were then on a smooth road to­ward an ac­cel­er­at­ed ap­proval, with plans to file an NDA in the third quar­ter of 2021.

Reg­u­la­tors, though, ap­par­ent­ly changed their mind.

Ap­plied has de­cid­ed to hold on sub­mit­ting an NDA for AT-007 as a treat­ment for galac­tosemia, the com­pa­ny dis­closed, fol­low­ing dis­cus­sions with the FDA in which the agency in­di­cat­ed that “clin­i­cal out­comes da­ta will like­ly be re­quired for ap­proval.”

The com­pa­ny says that came as a sur­prise, as they had thought — based on pre­vi­ous con­ver­sa­tions — that they could pur­sue an ac­cel­er­at­ed ap­proval by show­ing its drug re­duces a key bio­mark­er called galac­ti­tol.

Shares $APLT plum­met­ed 30.50% to $6.22.

Pa­tients with galac­tosemia have trou­ble break­ing down the sug­ar galac­tose in their blood­streams and can suf­fer from tox­ic waste that builds up in tis­sues and or­gans, caus­ing long-term com­pli­ca­tions. Galac­ti­tol is one such tox­ic metabo­lite.

Ap­plied’s drug is de­signed to in­hib­it the en­zyme al­dose re­duc­tase and cross the blood-brain bar­ri­er to en­ter the cen­tral ner­vous sys­tem.

Fu­ture plans and tim­ing will be con­tin­gent on dis­cus­sions with the FDA, ac­cord­ing to the biotech.

“While dis­ap­point­ed by this change in di­rec­tion by the FDA, we re­main com­mit­ted to bring­ing this im­por­tant treat­ment to pa­tients with galac­tosemia,” founder/CEO Shoshana Shen­del­man said in a state­ment.

The orig­i­nal NDA in­volved the use of AT-007 among adults. But Ap­plied is al­so con­duct­ing a Phase III study with chil­dren, in which clin­i­cal out­comes over time are mea­sured, in­clud­ing cog­ni­tion, be­hav­ior and mo­tor skills. Af­ter a clin­i­cal hold and sub­se­quent pro­to­col amend­ments, the com­pa­ny said a fire­walled com­mit­tee will com­plete its first as­sess­ment of ef­fi­ca­cy in Q1 of this year (and, if it doesn’t hit sta­tis­ti­cal sig­nif­i­cance, re­peat the process every six months un­til it does).

Graphic: Alexander Lefterov for Endpoints News

Small biotechs with big drug am­bi­tions threat­en to up­end the tra­di­tion­al drug launch play­book

Of the countless decisions Vlad Coric had to make as Biohaven’s CEO over the past seven years, there was one that felt particularly nerve-wracking: Instead of selling to a Big Pharma, the company decided it would commercialize its migraine drug itself.

“I remember some investors yelling and pounding on the table like, you can’t do this. What are you thinking? You’re going to get crushed by AbbVie,” he recalled.

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Mar­ket­ingRx roundup: Pfiz­er de­buts Pre­vnar 20 TV ads; Lil­ly gets first FDA 2022 pro­mo slap down let­ter

Pfizer debuted its first TV ad for its Prevnar 20 next-generation pneumococcal pneumonia vaccine. In the 60-second spot, several people (actor portrayals) with their ages listed as 65 or older are shown walking into a clinic as they turn to say they’re getting vaccinated with Prevnar 20 because they’re at risk.

The update to Pfizer’s blockbuster Prevnar 13 vaccine was approved in June, and as its name suggests is a vaccine for 20 serotypes — the original 13 plus seven more that cause pneumococcal disease. Pfizer used to spend heavily on TV ads to promote Prevnar 13 in 2018 and 2019 but cut back its TV budgets in the past two fall and winter seasonal spending cycles. Prevnar had been Pfizer’s top-selling drug, notching sales of just under $6 billion in 2020, and was the world’s top-selling vaccine before the Covid-19 vaccines came to market last year.

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Albert Bourla (Photo by Steven Ferdman/Getty Images)

UP­DAT­ED: Pfiz­er fields a CRL for a $295M rare dis­ease play, giv­ing ri­val a big head start

Pfizer won’t be adding a new rare disease drug to the franchise club — for now, anyway.

The pharma giant put out word that their FDA application for the growth hormone therapy somatrogon got the regulatory heave-ho, though they didn’t even hint at a reason for the CRL. Following standard operating procedure, Pfizer said in a terse missive that they would be working with regulators on a followup.

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A new can­cer im­munother­a­py brings cau­tious hope for a field long await­ing the next big break­through

Bob Seibert sat silent across from his daughter at their favorite Spanish restaurant near his home in Charleston County, SC, their paella growing cold as he read through all the places in his body doctors found tumors.

He had texted his wife, a pediatric intensive care nurse, when he got the alert that his online chart was ready. Although he saw immediately it was bad, many of the terms — peritoneal, right iliac — were inscrutable. But she was five hours downstate, at a loud group dinner the night before another daughter’s cheer competition.

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Florida Gov. Ron DeSantis (AP Photo/Wilfredo Lee, File)

Opin­ion: Flori­da is so mAb crazy, Ron De­San­tis wants to use mAbs that don't work

Florida Gov. Ron DeSantis is trying so hard to politicize the FDA and demonize the federal government that he entered into an alternate universe on Monday evening in describing a recent FDA action to restrict the use of two monoclonal antibody, or mAb, treatments for Covid-19 that don’t work against Omicron.

Without further ado, let’s break down his statement from last night, line by line, adjective by adjective.

FDA slams Eli Lil­ly's 'mis­lead­ing' In­sta­gram ad for its type 2 di­a­betes in­jec­tion

In a first for 2022, the FDA’s Office of Prescription Drug Promotion has issued an untitled letter, which was recently sent to Eli Lilly over what the agency calls a “misleading” and “particularly concerning” Instagram ad the company posted for its type 2 diabetes drug Trulicity.

The questionable Instagram post, which has since been deleted by Lilly, failed to adequately communicate the indication and limitations of use associated with Trulicity, FDA says.

US re­stricts use of two mon­o­clon­al an­ti­bod­ies that don't work against Omi­cron

Two monoclonal antibody combos from Eli Lilly and Regeneron are no longer authorized in the US, and shipments to states have ceased because HHS said they are “highly unlikely” to work against the sweeping new variant Omicron.

The move by the FDA comes as states like Florida have become insistent that the mAbs need to be independently evaluated, although the federal Department of Health and Human Services, which has shipped hundreds of thousands of these two mAbs to states in recent weeks, did not ship any this week.

Not cheap­er by the dozen: Bris­tol My­ers be­comes the 12th phar­ma com­pa­ny to re­strict 340B sales

Bristol Myers Squibb recently joined 11 of its peer pharma companies in limiting how many contract pharmacies can access certain drugs discounted by a federal program known as 340B.

Bristol Myers is just the latest in a series of high-profile pharma companies moving in their own direction as the Biden administration’s Health Resources and Services Administration struggles to rein in the drug discount program for the neediest Americans.

Joaquin Duato, J&J CEO (Photo by Charles Sykes/Invision/AP)

New J&J CEO Joaquin Du­a­to promis­es an ag­gres­sive M&A hunt in quest to grow phar­ma sales

Joaquin Duato stepped away from the sideline and directly into the spotlight on Tuesday, delivering his first quarterly review for J&J as its newly-tapped CEO after an 11-year run in senior posts. And he had some mixed financial news to deliver today while laying claim to a string of blockbuster drugs in the making and outlining an appetite for small and medium-sized M&A deals.

Duato also didn’t exactly shun large buyouts when asked about the future of the company’s medtech business — where they look to be in either the top or number 2 position in every segment they’re in — even though the bar for getting those deals done is so much higher.

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