Eric Dube, Travere Therapeutics CEO

Mar­tin Shkre­li’s old com­pa­ny touts PhI­II win, guns for ac­cel­er­at­ed ap­proval

Three months af­ter re­brand­ing to shed the last of its ties to con­vict­ed “Phar­ma Bro” Mar­tin Shkre­li, Tra­vere Ther­a­peu­tics is putting its best foot for­ward with an up­beat Phase III analy­sis and plans to seek ac­cel­er­at­ed ap­proval in a rare kid­ney dis­or­der.

Tra­vere — for­mer­ly known as Retrophin — says its lead can­di­date sparsen­tan met the pre-spec­i­fied in­ter­im end­point in a study of 371 pa­tients who have fo­cal seg­men­tal glomeru­loscle­ro­sis, a dis­or­der where kid­neys’ fil­tra­tion breaks down and pro­teins leak in­to the urine. For many pa­tients, the dis­or­der leads to end-stage kid­ney dis­ease.

In the on­go­ing Phase III DU­PLEX study, pa­tients were ran­dom­ly giv­en sparsen­tan or Avapro, the cur­rent stan­dard of care. At 36 weeks, 42% of pa­tients in the treat­ment arm achieved the FS­GS par­tial re­mis­sion of pro­tein­uria end­point, ver­sus 26% in the Avapro group, ac­cord­ing to Tra­vere.

It’s still too soon to get a read on the con­fir­ma­to­ry pri­ma­ry end­point, which is the rate of change in es­ti­mat­ed glomeru­lar fil­tra­tion rate (eGFR) over 108 weeks. But there are enough da­ta to pur­sue ac­cel­er­at­ed ap­proval, Tra­vere added.

“For decades peo­ple liv­ing with FS­GS have faced dai­ly chal­lenges in con­trol­ling pro­tein­uria and a fear of pro­gress­ing to trans­plant or dial­y­sis be­cause cur­rent treat­ment op­tions are not enough,” CEO Er­ic Dube said in a state­ment. “As we move ahead, our or­ga­ni­za­tion will be fo­cused on main­tain­ing high qual­i­ty in this on­go­ing study, and on con­tin­u­ing our en­gage­ment with reg­u­la­tors to en­able sub­mis­sions for ac­cel­er­at­ed ap­proval with the avail­able da­ta set.”

DU­PLEX is ful­ly en­rolled and on track for a read­out on the con­fir­ma­to­ry pri­ma­ry end­point in the first half of 2023, ac­cord­ing to the com­pa­ny. The drug is al­so be­ing stud­ied in Phase III for IgA nephropa­thy, with an in­ter­im analy­sis ex­pect­ed in Q3 of this year.

Tra­vere has come a long way since it was found­ed by Shkre­li in 2011. The com­pa­ny gave Shkre­li the boot back in 2014, short­ly af­ter he bought the rights to Thi­o­la and jacked the price by 2,000%, ac­cord­ing to an FTC com­plaint. Af­ter be­ing oust­ed, Shkre­li went on a “war path,” threat­en­ing to harm the com­pa­ny and the rep­u­ta­tions of those in it, his suc­ces­sor Stephen Ase­lage tes­ti­fied, per a CN­BC re­port. 

The biotech filed a law­suit against Shkre­li in 2015, which the in­fa­mous biotech ex­ec fol­lowed with a coun­ter­suit. In 2019, he tacked on an­oth­er ac­cu­sa­tion against three board di­rec­tors seek­ing $30 mil­lion in dam­ages. Retrophin paid an undis­closed amount to Shkre­li lat­er that year to set­tle all of the dis­putes, and sev­er its ties with the “Phar­ma Bro,” who’s cur­rent­ly serv­ing a 7-year sen­tence for de­fraud­ing in­vestors of his hedge funds. Part of the case in­volved Shkre­li’s use of Retrophin stock to pay off those in­vestors.

The com­pa­ny changed its name to Tra­vere in No­vem­ber, as the old name “doesn’t re­flect who we are to­day, and the role that we want to play go­ing in­to the fu­ture,” Dube told End­points News. 

The new name is in­spired by the Latin roots of the words “path” (trac­tus) and “truth” (ver).

The biotech’s pipeline al­so in­cludes TVT-058, a Phase II can­di­date for clas­si­cal ho­mo­cystin­uria that it snagged in its Oc­to­ber buy­out of Or­phan Tech­nolo­gies. Tra­vere agreed to put down up to $517 mil­lion for the low-pro­file com­pa­ny and its en­zyme re­place­ment ther­a­py, in­clud­ing $427 mil­lion in mile­stones.

Biotech and Big Phar­ma: A blue­print for a suc­cess­ful part­ner­ship

Strategic partnerships have long been an important contributor to how drugs are discovered and developed. For decades, big pharma companies have been forming alliances with biotech innovators to increase R&D productivity, expand geographical reach and better manage late-stage commercialization costs.

Noël Brown, Managing Director and Head of Biotechnology Investment Banking, and Greg Wiederrecht, Ph.D., Managing Director in the Global Healthcare Investment Banking Group at RBC Capital Markets, are no strangers to the importance of these tie-ups. Noël has over 20 years of investment banking experience in the industry. Before moving to the banking world in 2015, Greg was the Vice President and Head of External Scientific Affairs (ESA) at Merck, where he was responsible for the scientific assessment of strategic partnership opportunities worldwide.

No­var­tis' sec­ond at­tempt to repli­cate a stun­ning can­cer re­sult falls flat

Novartis’ hopes of turning one of the most surprising trial data points of the last decade into a lung cancer drug has taken another setback.

The Swiss pharma announced Monday that its IL-1 inhibitor canakinumab did not significantly extend the lives or slow the disease progression of patients with previously untreated locally advanced or metastatic non-small cell lung cancer when compared to standard of-care alone.

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How Chi­na turned the ta­bles on bio­phar­ma's glob­al deal­mak­ing

Fenlai Tan still gets chills thinking about the darkest day of his life.

Three out of eight lung cancer patients who received a tyrosine kinase inhibitor developed by his company, Betta Pharma, died in the span of a month. Tan, the chief medical officer, was summoned to Peking Union Medical College Hospital, where the head of the clinical trial department told him that the trial investigators would be conducting an autopsy to see if the patients had died of the disease — they were all very sick by the time they enrolled — or of interstitial lung disease, a deadly side effect tied to the TKI class that’s been reported in Japan.

An­gion's or­gan dam­age drug strikes out again, this time in high-risk kid­ney trans­plant pa­tients

After flopping a test in Covid-19 earlier this year, Angion’s lead organ damage drug has now hit the skids again in kidney transplant patients.

Angion and partner Vifor Pharma’s ANG-3777 failed to beat out placebo in terms of improving eGFR, a measure of kidney function, in patients who had received a deceased donor kidney transplant and were at high risk of developing what is known as delayed graft function, according to Phase III results released Tuesday.

An image of Alzheimer's brain tissue. The red show gingipains, a protein from P. gingivalis, intermixing with neurons (yellow) and glial cells (green)

An Alzheimer's dark­horse fails its first big tri­al, but of­fers hope for a long-over­looked hy­poth­e­sis

Three years ago, Cortexyme emerged out of obscurity with some big-name backers and an unorthodox approach to treating Alzheimer’s.

They moved their drug into a pivotal study the next year, offering one of the first major tests for a hypothesis that has fluttered on the outskirts of Alzheimer’s research for decades: that, in many cases, the disease is driven by infectious agents — the havoc they wreak in the brain and the inflammation the body uses to try to fend them off. And that quashing the infection could slow patients’ cognitive decline.

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No­var­tis dumps AveX­is pro­gram for Rett syn­drome af­ter fail­ing re­peat round of pre­clin­i­cal test­ing

Say goodbye to AVXS-201.

The Rett syndrome gene therapy drug made by AveXis — the biotech that was bought, kept separate, then renamed and finally absorbed by Novartis into its R&D division — has been dropped by the biopharma.

In Novartis’ third quarter financial report, the pharma had found that preclinical data did not support development of the gene therapy into IND-enabling trials and beyond. The announcement comes a year after Novartis told the Rett Society how excited it was by the drug — and its potential benefits and uses.

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Peter Nell, Mammoth Biosciences CBO

UP­DAT­ED: Jen­nifer Doud­na spin­out inks a Mam­moth CRISPR deal with Ver­tex worth near­ly $700M

When a company gets its start in gene editing pioneer Jennifer Doudna’s lab, it’s bound to make headlines. But three years in, the fanfare still hasn’t died down for Mammoth Biosciences. Now, the Brisbane, CA-based company is cheering on its first major R&D pact.

Mammoth unveiled a nearly $700 million deal with Vertex on Tuesday morning, good for the development of in vivo gene therapies for two mystery diseases. The stars of the show are Mammoth’s ultra-small CRISPR systems, including two Cas enzymes licensed from Doudna’s lab over the past couple years, Cas14 and Casɸ.

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FDA is much worse than its reg­u­la­to­ry peers at proac­tive­ly dis­clos­ing da­ta, re­searchers find

The European Medicines Agency and Health Canada continue to outpace the FDA when it comes to proactively releasing data on drugs and biologics the agency has reviewed, leading to further questions of why the American agency can’t be more transparent.

In a study published recently in the Journal of Law, Medicine, & Ethics, Yale and other academic lawyers and researchers found that between 2016 and April 2021, the EMA proactively released data for 123 unique medical products, while Health Canada proactively released data for 73 unique medical products between 2019 and April 2021. What’s more, the EMA and Health Canada didn’t proactively release the same data on the same drugs. In stark contrast, the FDA in 2018 only proactively disclosed data supporting one drug that was approved that year.

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NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

No, sci­en­tists are not any clos­er to pig-to-hu­man trans­plants than they were last week

Steve Holtzman was awoken by a 1 a.m. call from a doctor at Duke University asking if he could put some pigs on a plane and fly them from Ohio to North Carolina that day. A motorcyclist had gotten into a horrific crash, the doctor explained. He believed the pigs’ livers, sutured onto the patient’s skin like an external filter, might be able to tide the young man over until a donor liver became available.