CEO Eric Dube (Travere Therapeutics)

Retrophin no more: Shkre­li's lega­cy dis­ap­pears as for­mer com­pa­ny be­comes Tra­vere Ther­a­peu­tics

Retrophin is shed­ding the last of its ties to con­vict­ed “Phar­ma Bro” Mar­tin Shkre­li and his failed neu­ro­log­i­cal drug — with a name change.

“That name doesn’t re­flect who we are to­day, and the role that we want to play go­ing in­to the fu­ture. So we felt that it was be­fit­ting to have a name that every sin­gle one of us came to work and can iden­ti­fy with,” CEO Er­ic Dube told End­points News.

The biotech, which was found­ed in 2011 by Shkre­li, now goes by Tra­vere Ther­a­peu­tics, in­spired by the Latin roots of the words “path” (trac­tus) and “truth” (ver). Dube said the re­brand­ing has been in the works since he joined the com­pa­ny last year, and rep­re­sents “the tire­less jour­ney that many rare dis­ease fam­i­lies em­bark on to reach a di­ag­no­sis and ul­ti­mate­ly a treat­ment or cure.”

A few weeks be­fore it took a new iden­ti­ty, Retrophin agreed to buy low-pro­file Or­phan Tech­nolo­gies for up to $517 mil­lion, bol­ster­ing its rare dis­ease pipeline with OT-58 for the treat­ment of clas­si­cal ho­mo­cystin­uria, a con­di­tion that’s trig­gered by in­suf­fi­cient lev­els of the en­zyme cys­tathio­n­ine be­ta syn­thase. And Dube is ex­pect­ing to read out Phase III da­ta for its can­di­date sparsen­tan in fo­cal seg­men­tal glomeru­loscle­ro­sis (FS­GS) and IgA nephropa­thy in Q1 and Q3 of next year, re­spec­tive­ly.

“Next year is go­ing to be trans­for­ma­tion­al for us with the two Phase III da­ta read­outs,” Dube said. “We al­so ex­pect to have a da­ta read­out from our Phase I/II from the as­set from Or­phan Tech­nolo­gies and we’ll con­tin­ue to pre­pare for the launch of sparsen­tan as­sum­ing suc­cess from our Phase II­Is.”

Last Au­gust, Retrophin’s lead drug — which was co-in­vent­ed by Shkre­li for a rare neu­ro­log­i­cal dis­ease called pan­tothen­ate ki­nase-as­so­ci­at­ed neu­rode­gen­er­a­tion — flunked the pri­ma­ry and key sec­ondary end­points in a piv­otal Phase III tri­al. Up­on the news, the com­pa­ny’s stock plunged 30% ahead of the bell, knock­ing $250 mil­lion off the mar­ket cap.

Retrophin gave Shkre­li the boot back in 2014, short­ly af­ter he bought the rights to Thi­o­la then jacked the price by 2,000%, ac­cord­ing to an FTC com­plaint. Af­ter be­ing oust­ed from the com­pa­ny, he went on a “war path,” his suc­ces­sor Stephen Ase­lage tes­ti­fied, per a CN­BC re­port. Ase­lage said Shkre­li threat­ened to “harm the com­pa­ny and dam­age the rep­u­ta­tions of the peo­ple in the com­pa­ny.”

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.

While Retrophin’s name has changed, Dube said its mis­sion re­mains the same: “be­ing ded­i­cat­ed ex­clu­sive­ly to rare dis­ease and de­vel­op­ing and de­liv­er­ing ther­a­pies for rare dis­ease.” He said be­ing di­ag­nosed with a rare can­cer in his ear­ly 30s changed his per­spec­tive on the field, and while he was lucky to have caught it ear­ly enough, many oth­ers aren’t.

“I was very for­tu­nate, hav­ing now been a sur­vivor when there was not an ap­proved ther­a­py,” he said. “But the oth­er thing that that ex­pe­ri­ence taught me is that time re­al­ly mat­ters … I know that the work that we do has a di­rect im­pact on our pa­tients and so we have to work with very good dis­ci­pline and with speed.”

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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So what hap­pened with No­var­tis' gene ther­a­py group? Here's your an­swer

Over the last couple of days it’s become clear that the gene therapy division at Novartis has quietly undergone a major reorganization. We learned on Monday that Dave Lennon, who had pursued a high-profile role as president of the unit with 1,500 people, had left the pharma giant to take over as CEO of a startup.

Like a lot of the majors, Novartis is an open highway for head hunters, or anyone looking to staff a startup. So that was news but not completely unexpected.

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David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

Vicente Anido (University of West Virginia via YouTube)

Aerie fires CEO af­ter lead pro­gram flop, com­ments about pri­ma­ry end­points be­ing 'not re­quired'

Aerie Pharmaceuticals CEO Vicente Anido has left the company less than a week after trying to chart a Phase III study in the wake of a serious Phase IIb flop.

Anido’s last day at Aerie was Friday, the biotech announced in a news release Tuesday morning, and Benjamin McGraw is taking his place in an interim role. The now former CEO was terminated without cause, according to an SEC filing.

The board has started looking for a full-time chief to take his place.

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When ef­fi­ca­cy is bor­der­line: FDA needs to get more con­sis­tent on close-call drug ap­provals, agency-fund­ed re­search finds

In the exceedingly rare instances in which clinical efficacy is the only barrier to a new drug’s approval, new FDA-funded research from FDA and Stanford found that the agency does not have a consistent standard for defining “substantial evidence” when flexible criteria are used for an approval.

The research comes as the FDA is at a crossroads with its expedited-review pathways. The accelerated approval pathway is under fire as the agency recently signed off on a controversial new Alzheimer’s drug, with little precedent to explain its decision. Meanwhile, top officials like Rick Pazdur have called for a major push to simplify and clarify all of the various expedited pathways, which have grown to be must-haves for sponsors of nearly every newly approved drug.

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Lat­est news: It’s a no on uni­ver­sal boost­ers; Pa­tient death stuns gene ther­a­py field; In­side Tril­li­um’s $2.3B turn­around; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Next week is shaping up to be a busy one, as our editor-in-chief John Carroll and managing editor Kyle Blankenship lead back-to-back discussions with a great group of experts to discuss the weekend news and trends. John will be spending 30 minutes with Jake Van Naarden, the CEO of Lilly Oncology, and Kyle has a brilliant panel lined up: Harvard’s Cigall Kadoch, Susan Galbraith, the new head of cancer R&D at AstraZeneca, Roy Baynes at Merck, and James Christensen at Mirati. Don’t miss out on the action — sign up here.

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Jay Bradner (Jeff Rumans for Endpoints News)

Div­ing deep­er in­to in­her­it­ed reti­nal dis­or­ders, No­var­tis gob­bles up an­oth­er bite-sized op­to­ge­net­ics biotech

Right about a year ago, a Novartis team led by Jay Bradner and Cynthia Grosskreutz at NIBR swooped in to scoop up a Cambridge, MA-based opthalmology gene therapy company called Vedere. Their focus was on a specific market niche: inherited retinal dystrophies that include a wide range of genetic retinal disorders marked by the loss of photoreceptor cells and progressive vision loss.

But that was just the first deal that whet their appetite.

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