Calliditas nabs accelerated OK for rare kidney disease, beating two competitors to the punch

Renee Aguiar-Lucander, Calliditas CEO

Since its found­ing, Cal­lid­i­tas has wa­gered it could bring to mar­ket a new for­mu­la­tion of an oral steroid to treat a rare kid­ney dis­ease. And on Wednes­day af­ter­noon, its bet paid off.

The FDA grant­ed ac­cel­er­at­ed ap­proval to the biotech’s de­layed-re­lease budes­onide cap­sules, Cal­lid­i­tas an­nounced, wav­ing through a new treat­ment for the re­duc­tion of pro­tein­uria in pri­ma­ry IgA nephropa­thy. Wednes­day’s ap­proval came af­ter reg­u­la­tors de­layed the PDU­FA date by three months, as Cal­lid­i­tas said the agency re­quest­ed fur­ther analy­ses.

Cal­lid­i­tas will mar­ket the drug as Tarpeyo and ex­pects to launch it com­mer­cial­ly ear­ly next quar­ter. End­points News has reached out for com­ment on pric­ing and tim­ing for the drug’s con­fir­ma­to­ry study, and will up­date ac­cord­ing­ly.

Tarpeyo’s tri­al will like­ly mea­sure whether or not the drug can slow kid­ney func­tion de­cline, which has not yet been es­tab­lished. Re­duc­ing pro­tein­uria in IgAN pa­tients can low­er the amount of pro­tein in an in­di­vid­ual’s urine, serv­ing as a bio­mark­er stand-in for clin­i­cal ben­e­fit un­til the con­fir­ma­to­ry study can demon­strate clin­i­cal ben­e­fit.

Though Cal­lid­i­tas se­cured the first-ever US ap­proval in this dis­ease, it came in the midst of a fu­ri­ous race against two oth­er main com­peti­tors. Mar­tin Shkre­li’s for­mer biotech Tra­vere Ther­a­peu­tics (pre­vi­ous­ly Retrophin) and Seat­tle-based Chi­nook Ther­a­peu­tics are each work­ing on their own IgAN drugs, al­though nei­ther are steroids like Tarpeyo.

Tra­vere read out its own Phase III da­ta in Au­gust for sparsen­tan, where pa­tients saw a mean re­duc­tion in pro­tein­uria of 49.8% from base­line at 36 weeks com­pared to 15.1% in the con­trol group, good for a p-val­ue of p<0.0001. Cross-tri­al com­par­isons are tricky, par­tic­u­lar­ly in the ac­cel­er­at­ed ap­proval space, but Tarpeyo notched sim­i­lar re­sults with a 34% re­duc­tion from base­line af­ter 9 months vs 5% in the con­trol — a p-val­ue of p=0.0001.

Chi­nook, mean­while, is cur­rent­ly in a Phase III tri­al for its pro­gram known as atrasen­tan, an en­dothe­lin re­cep­tor an­tag­o­nist the com­pa­ny li­censed from Ab­b­Vie for an undis­closed price back in 2019. Ab­b­Vie had pre­vi­ous­ly been de­vel­op­ing atrasen­tan for di­a­bet­ic kid­ney dis­ease, but shut down a Phase III study in 2017.

Even if Cal­lid­i­tas won’t have much time to rest on its lau­rels, Wednes­day’s ap­proval is a big step to­ward prov­ing the biotech’s found­ing idea. Cal­lid­i­tas had spun out of Swe­den’s Up­p­sala Uni­ver­si­ty with a li­cense for the oral for­mu­la­tion and nev­er strayed from the rare kid­ney dis­ease.

The the­o­ry be­hind the drug is by sup­press­ing the im­mune re­ac­tion as­so­ci­at­ed with IgAN, Tarpeyo can pre­vent pa­tients from pro­gress­ing to end-stage re­nal dis­ease. Should the con­fir­ma­to­ry study prove suc­cess­ful, Cal­lid­i­tas re­searchers would os­ten­si­bly clinch that the­o­ry.

The FDA on Wednesday not only approved the first generic versions of the decades-old diabetes insipidus treatment vasopressin, but also simultaneously offered a particularly damning rebuke of a citizen petition attempting to block the generic, while promising to pass along the matter to the Federal Trade Commission.

The response could prove troublesome for the sponsor of the brand name version of the drug, Endo’s Par Sterile Products, which brought in more than $780 million in 2020 for its brand name version of the drug Vasostrict.

Sensor-based technology for clinical trial data collection represents the latest medical paradigm shift. There are more than 700 clinical studies involving wearable devices currently underway in the United States. A study from Intel IT projects their inclusion in clinical trials will surge to 70% by 2025.

Apps, biosensors and patient-centered technologies increase visibility of comprehensive patient data. Pharma leaders anticipate the benefits of wearables to include better data (58%), faster results (33%) and lower trial costs (10%).

Crowd gathering at the Westin St. Francis for JPM in 2019 (Endpoints News)

Well, see you in January 2023.

In a surprise about-face, #JPM22 will now be fully virtual after organizers of the popular biotech conference decided to pull the plug on a live event in San Francisco given fears over the Omicron variant and a growing chorus of drugmakers opting out.

The move is no big surprise after reports swirled about some of the industry’s biggest players nixing plans to attend live and pressuring the bank to reconsider the annual meet at the Westin St. Francis. STAT reported Tuesday that Moderna and Amgen, among other large drugmakers, had already pulled out.

Robert Califf (Graeme Sloan/Sipa via AP Images)

As Rob Califf likely makes his return as FDA commissioner next month, his confirmation hearing yesterday offered a peek into some of the larger obstacles he’s going to face in the coming months and years.

The pandemic isn’t going away anytime soon with Omicron, and some vaccines and therapeutics may need to be tweaked or pulled from the market entirely as they prove to be ineffective against the new variant. The FDA, meanwhile, needs to get back on even footing with some longer-term direction.

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For the first time, Biogen released details Thursday on how it intends to prove that its by turns celebrated and maligned drug Aduhelm can actually slow the decline of patients with Alzheimer’s, as required by the FDA.

The biotech said it will launch a global 1,300-person trial next May that will randomize early-stage Alzheimer’s patients to receive either Aduhelm or placebo. It will probably take about four years for the trial to generate results, the company said, pushing a final answer to 2026.

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A nurse administers a COVID-19 booster shot to Joe Rigdon at a vaccination site in Eastmonte Park, Altamonte Springs. (Photo by Paul Hennessy/SOPA Images/LightRocket via Getty Images)

When Laura Burns went to get her first Covid-19 shot last January, no one had warned her that the vaccines might not work for her.

Burns, the recipient of a double-lung transplant in 2016, knew to be careful about the medicines she took. She consulted with her transplant team when the Pfizer and Moderna shots were authorized and only signed up after being told the vaccines would likely be safe for her, which they were.

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Jay Bradner, President, Novartis Institutes for BioMedical Research

John Carroll: Well, hello everybody. This is John Carroll. I’m the editor of Endpoints News, the editor and founder of Endpoints News. I’m here with Jay Bradner, the president of the Novartis Institutes for BioMedical Research. Jay, we’re going to be talking about ASH in just a second, but you’ve just recently celebrated your sixth anniversary as president of NIBR. And I’m curious, it’s such a significant amount of time for anybody to spend in one career phase. And looking back over the last six years, is everything fundamentally different about the research process and the translational arena that you’re in?

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Thomas Lingelbach, Valneva CEO

Two weeks ago, Valneva pushed back against a study that found its candidate, VLA2001, was the only one out of seven tested vaccines that didn’t significantly boost protection when given to volunteers who had two doses of the Pfizer/BioNTech mRNA shot.

Valneva said the participants had been given booster doses after a shorter interval than usual — and vaccines made from inactivated viruses, such as its candidate, typically require a longer period of time to be effective.

From L-R: Luigi Naldini, Genenta CEO Pierluigi Paracchi and Genenta CMO Carlo Russo

After years of developing its gene therapy for cancer on the outskirts of biotech, Genenta has landed on Nasdaq as the first Italian biotech on the American exchange.

Genenta’s IPO is small compared to what you usually see these days, but the $36 million — or $40 million if all options are exercised — in gross proceeds will allow Pierluigi Paracchi’s crew to take its current proof-of-concept trial to fruition and pave the way to Phase II, fund long-term monitoring of patients, pay for preclinical research and manufacturing, while leaving enough money to run the actual Phase II trial slated to start in 2023 (the business development and admin team also gets some).
https://endpts.com/calliditas-nabs-accelerated-ok-for-rare-kidney-disease-beating-two-competitors-to-the-punch/