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.”

MedTech clinical trials require a unique regulatory and study design approach and so engaging a highly experienced CRO to ensure compliance and accurate data across all stages is critical to development milestones.

In­no­v­a­tive MedTech De­mands Spe­cial­ist Clin­i­cal Tri­al Reg­u­la­to­ry Af­fairs and De­sign

Avance Clinical is the Australian CRO for international biotechs providing world-class clinical research services with FDA-accepted data across all phases. With Avance Clinical, biotech companies can leverage Australia’s supportive clinical trials environment which includes no IND requirement plus a 43.5% Government incentive rebate on clinical spend. The CRO has been delivering clinical drug development services for international biotechs for FDA and EMA regulatory approval for the past 24 years. The company has been recognized for the past two consecutive years with the prestigious Frost & Sullivan CRO Best Practices Award and a finalist in Informa Pharma’s Best CRO award for 2022.

Gold for adults, sil­ver for in­fants: Pfiz­er's Pre­vnar 2.0 head­ed to FDA months af­ter Mer­ck­'s green light

Pfizer was first to the finish line for the next-gen pneumococcal vaccine in adults, but Merck beat its rival with a jab for children in June.

Now, two months after Merck’s 15-valent Vaxneuvance won the FDA stamp of approval for kids, Pfizer is out with some late-stage data on its 20-valent shot for infants.

Known as Prevnar 20 for adults, Pfizer’s 20vPnC will head to the FDA by the end of this year for an approval request in infants, the Big Pharma said Friday morning. Discussions with the FDA will occur first and more late-stage pediatric trials are expected to read out soon, informing the regulatory pathway in other countries and regions.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,700+ biopharma pros reading Endpoints daily — and it's free.

No­var­tis re­ports two pa­tient deaths af­ter treat­ment with Zol­gens­ma

Two children with spinal muscular atrophy have died after receiving Novartis’ Zolgensma, a gene therapy designed as a one-time treatment for the rare fatal disease.

The deaths, which resulted from acute liver failure, occurred in Russia and Kazakhstan, Novartis confirmed in a statement to Endpoints News. Having notified health authorities across all the markets where Zolgensma is available, it will update the drug label “to specify that fatal acute liver failure has been reported,” a spokesperson wrote.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,700+ biopharma pros reading Endpoints daily — and it's free.

His­toric drug pric­ing re­forms pass; Pfiz­er ac­quires GBT; The long search for non-opi­oid pain drugs; 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.

The Endpoints Weekly has officially crossed the 60,000 mark on subscribers — thanks to all of your support. As the editorial team grows, we’ve been able to do a lot more, with many of those on display this week. Be sure to check out Lei Lei Wu’s deep dive on pain R&D. If you missed it, you may also rewatch her companion panel here.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,700+ biopharma pros reading Endpoints daily — and it's free.

Senate Finance Committee Chair Ron Wyden (D-OR) (Francis Chung/E&E News/POLITICO via AP Images)

Sen­ate Fi­nance chair con­tin­ues his in­ves­ti­ga­tion in­to phar­ma tax­es with re­quests for Am­gen

Amgen is the latest pharma company to appear on the radar of Senate Finance Committee Chair Ron Wyden (D-OR), who is investigating the way pharma companies are using subsidiaries in low- or zero-tax countries to lower their tax bills.

Like its peers Merck, AbbVie and Bristol Myers Squibb, Wyden notes how Amgen uses its Puerto Rico operations to consistently pay tax rates that are substantially lower than the U.S. corporate tax rate of 21%, with an effective tax rate of 10.7% in 2020 and 12.1% in 2021.

FDA ap­proves sec­ond in­di­ca­tion for As­traZeneca and Dai­ichi's En­her­tu in less than a week

AstraZeneca and Daiichi Sankyo’s antibody-drug conjugate Enhertu scored its second approval in less than a week, this time for a subset of lung cancer patients.

Enhertu received accelerated approval on Thursday to treat adults with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating HER2 (ERBB2) mutations, and who have already received a prior systemic therapy.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,700+ biopharma pros reading Endpoints daily — and it's free.

J&J to re­move talc prod­ucts from shelves world­wide, re­plac­ing with corn­starch-based port­fo­lio

After controversially spinning out its talc liabilities and filing for bankruptcy in an attempt to settle 38,000 lawsuits, Johnson & Johnson is now changing up the formula for its baby powder products.

J&J is beginning the transition to an all cornstarch-based baby powder portfolio, the pharma giant announced on Thursday — just months after a federal judge ruled in favor of its “Texas two-step” bankruptcy to settle allegations that its talc products contained asbestos and caused cancer. An appeals court has since agreed to revisit that case.

CSL is gathering its four business units under a unified brand identity strategy (Credit: CSL company site)

CSL brings Se­qirus, Vi­for un­der par­ent um­brel­la brand in iden­ti­ty re­vamp

CSL is gathering its brands under the family name umbrella, renaming its vaccine and newly acquired nephrology specialty businesses with the parent initials.

CSL Seqirus and CSL Vifor join CSL Plasma and CSL Behring as the four now uniformly branded business units of the global biopharma. The Seqirus vaccine division was formed in 2015 with the combination of bioCSL and its purchase of Novartis’ flu vaccine business. CSL picked up Vifor Pharma late last year in an $11.7 billion deal for the nephrology, iron deficiency and cardio-renal drug developer.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,700+ biopharma pros reading Endpoints daily — and it's free.

Winselow Tucker, Eli Lilly's new Loxo unit chief commercial officer

Eli Lil­ly plucks a new com­mer­cial chief from Bris­tol My­ers in man­age­ment shuf­fle as HR chief re­tires

Eli Lilly has found a new chief commercial officer from among the ranks at Bristol Myers Squibb, as it says farewell to its longtime head of human resources Stephen Fry.

Fry announced on Thursday his plans to retire after more than 35 years with Lilly. He’ll vacate his seat as SVP of human resources and diversity at the end of the year, and current Loxo CCO Eric Dozier is slated to take his place. As a result, BMS’ Winselow Tucker is joining the team as Loxo CCO at the end of the month.