Mike Raab, Ardelyx CEO

FDA grants ap­peal for Arde­lyx chron­ic kid­ney dis­ease drug af­ter Ju­ly 2021 re­jec­tion

The FDA be­stowed good tid­ings up­on Arde­lyx’s ex­per­i­men­tal chron­ic kid­ney dis­ease drug Thurs­day morn­ing.

Reg­u­la­tors told the biotech they would hear an ap­peal to its com­plete re­sponse let­ter, po­ten­tial­ly set­ting Arde­lyx up for an ap­proval re­sub­mis­sion in the first half of next year. The move fol­lows a post-CRL ad­vi­so­ry com­mit­tee meet­ing last month in which the FDA’s Car­dio­vas­cu­lar and Re­nal Drugs pan­elists vot­ed in fa­vor of both monother­a­py and com­bi­na­tion reg­i­men ap­provals.

“To say I’m ex­cit­ed would be an un­der­state­ment,” pres­i­dent and CEO Mike Raab said on an an­a­lyst call Thurs­day morn­ing. In­vestors didn’t feel the same, how­ev­er, as Arde­lyx shares $ARDX fell about 8%.

Arde­lyx al­so won’t have to sub­mit new da­ta, Raab said. Some of the analy­ses sub­mit­ted for the ad­vi­so­ry com­mit­tee “may very well be part of what they would want us to sub­mit,” he said. “But that’s da­ta that they al­ready have as part of the” drug ap­pli­ca­tion.

The news pro­vides new mo­men­tum for Arde­lyx’s roller-coast­er at­tempt to get its drug, known as tena­panor, across the fin­ish line. FDA orig­i­nal­ly re­ject­ed the drug back in Ju­ly 2021 de­spite three pos­i­tive Phase III read­outs. An­a­lysts at the time de­clared an ap­proval “all but cer­tain.”

Arde­lyx orig­i­nal­ly had fo­cused on treat­ing chron­ic kid­ney dis­ease writ large, but nar­rowed that aim to a sub­set of pa­tients with an over­abun­dance of phos­phate in the blood af­ter ear­li­er, broad­er stud­ies flopped. That con­di­tion, called hy­per­phos­phatemia, oc­curs in about 80% of pa­tients with end-stage kid­ney dis­ease on main­te­nance dial­y­sis.

The FDA wasn’t con­vinced, and Arde­lyx’s stock lost more than 70% of its val­ue, and the com­pa­ny made rounds of job cuts. As the ap­peal and ad­vi­so­ry com­mit­tee process­es played out, reg­u­la­tors even­tu­al­ly said “the mag­ni­tude of the treat­ment ef­fect is un­clear.”

When tena­panor was first re­ject­ed, the im­pli­ca­tion of the FDA’s re­sponse was that the agency want­ed Arde­lyx to con­duct a new study look­ing at clin­i­cal out­comes, Raab told End­points News Thurs­day. That wasn’t some­thing the biotech was will­ing to do, he said: “Giv­en the com­plex­i­ty of the dis­ease, the mori­bund na­ture and the speed at which they die of many things, makes an out­come study near­ly im­pos­si­ble.”

Now, the com­pa­ny will fo­cus on dis­cus­sions with the FDA, which will like­ly start with a Type A meet­ing, Raab added. Arde­lyx is be­ing in­clud­ed in dis­cus­sions for the drug’s po­ten­tial la­bel, and is ex­pect­ed to be brand­ed as Xphozah.

If the FDA ul­ti­mate­ly re­vers­es course and ap­proves tena­panor here, it will be the drug’s sec­ond green light af­ter a 2019 thumbs-up for ir­ri­ta­ble bow­el syn­drome with con­sti­pa­tion in adults.

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Who’s spend­ing and who’s cut­ting from Big Phar­ma’s $127B R&D bud­get? Here are the top 15 play­ers

A couple of the Big 15 biopharma companies in R&D hit the gas on research spending last year. Merck and Sanofi still have lots to prove in the pipeline, and they’re willing to gamble large sums to make a better future for themselves.

Doing nothing would be infinitely worse.

But collectively, the top players rang up a modest 2.4% increase in spending in 2022, which didn’t cover inflationary pressures. And that set the tone for an extraordinarily cautious year for the industry — even as it laid out about $127 billion to advance new drugs or up the ante on approved therapies.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Jeff Bluestone (R), Sonoma Biotherapeutics CEO

Jef­frey Blue­stone brings his start­up haul to $400M+, join­ing forces with Re­gen­eron on cell ther­a­pies

These days, when Jeffrey Bluestone gets together with his contemporaries in science, the conversation often turns to retirement plans.

But a little more than three years ago, Bluestone reached a momentous turning point in his career, exiting a prestigious post at UCSF, where he had spent decades in the scientific pursuit of new therapies. And it had nothing to do with retirement anytime in the near future.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Mihael Polymeropoulos, Vanda Pharmaceuticals CEO

Van­da wins court case against FDA over dis­clo­sure of CRL de­tails for sleep drug

DC District Court Judge Christopher Cooper today granted Vanda Pharma’s request to require the FDA to disclose more info on the complete response letter for its sleep disorder drug Hetlioz.

The melatonin receptor agonist is approved by the FDA to treat non-24-hour sleep-wake disorder, a circadian rhythm disorder. But in 2018 Vanda filed a supplemental application to market Hetlioz as a treatment for jet lag, which the FDA rejected in August 2019, with few details on what Vanda needed to correct course, according to the company.

Gun­ning for 2023 ap­proval, GSK de­tails PhI­II da­ta for Jem­per­li in front­line en­dome­tri­al can­cer

GSK has a new slate of data to offer on its PD-1 inhibitor, Jemperli — data that the pharma giant hopes will cement one of the four drug approvals it’s expecting this year.

While Jemperli (dostarlimab) is already approved for a subset of patients with second-line endometrial cancer, GSK set out in the Phase III RUBY trial to test it as an earlier line of treatment while also enrolling a broader group of patients. In an interim analysis, Jemperli was shown to extend progression-free survival for both the subset and the overall trial population when added to chemotherapy.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,800+ biopharma pros reading Endpoints daily — and it's free.

Chaim Lebovits, BrainStorm Cell Therapeutics CEO

Brain­Storm gets FDA ad­comm for ALS drug af­ter failed tri­al and RTF

BrainStorm Cell Therapeutics has had a rocky road trying to get its ALS treatment through to the FDA, including a particularly difficult setback after the FDA slapped the company with a refusal to file (RTF) letter in November after it submitted its original BLA in September last year.

But there’s a glimmer of hope for BrainStorm’s ALS treatment, NurOwn – the FDA will hold an advisory committee meeting to discuss the company’s BLA for the treatment, the company announced today.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,800+ biopharma pros reading Endpoints daily — and it's free.

Doug Williams, departing Codiak BioSciences CEO

Co­di­ak files for Chap­ter 11 bank­rupt­cy as most ex­ec­u­tives head for the ex­it

Codiak BioSciences has filed for Chapter 11 bankruptcy, spelling an end to the employment of most executives, including founder Doug Williams, as the biotech says it “expects to consummate a sale.”

The eight-year journey at Codiak is nearing an end with Williams; CFO Linda Bain; medical chief David Mauro; scientific head Sriram Sathyanarayanan; legal and compliance chief Yalonda Howze; and SVP of HR Nicole Barna all packing up their bags in the first few days of April. Chief technology officer Konstantin Konstantinov will stay.

Rep. Cathy McMorris Rodgers (R-WA) (Michael Brochstein/Sipa USA/Sipa via AP Images)

House com­mit­tee chair re­quests in­fo from FDA on fund­ing for tri­als in­volv­ing 'vi­ral ma­nip­u­la­tion'

Republican members of Congress want FDA Commissioner Robert Califf and his agency to provide more information on the research FDA has funded that manipulates certain viruses like the SARS-CoV-2 virus — after the FDA confirmed that certain studies it funded involved viral manipulation, but in standard ways that are common in molecular biology.

The three-page letter, signed Monday by House Energy and Commerce Committee Chair Cathy McMorris Rodgers (R-WA) and Subcommittee Chairs Brett Guthrie (R-KY) and Morgan Griffith (R-VA), kicked things off by saying that the committee was looking into FDA’s oversight of “FDA-funded research that may pose significant biosafety or biosecurity risks.”

FDA plans up­com­ing ad­comms for OTC con­tra­cep­tive, ep­i­neph­rine nasal spray, Intar­ci­a's last shot

The FDA has scheduled or planned for three advisory committee meetings in May: one for an over-the-counter birth control pill, one for a nasal spray version of the EpiPen, and another for  Intarcia Therapeutics, which has all but completely folded already as a company.

Perrigo will bring its daily contraceptive, Opill, to the joint Nonprescription Drugs Advisory Committee and the Obstetrics, Reproductive and Urologic Drugs Advisory Committee on May 9 and 10 for its over-the-counter birth control application.