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.”

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.

Luke Miels, GSK chief commercial officer

GSK picks up Scynex­is' FDA-ap­proved an­ti­fun­gal drug for $90M up­front

Editor’s note: This is a live story and will be updated.

GSK is dishing out $90 million cash to add an antifungal drug to its commercial portfolio, in a deal spotlighting the pharma giant’s growing focus on infectious diseases.

The upfront will lock in an exclusive license to Scynexis’ Brexafemme, which was approved in 2021 to treat a yeast infection known as vulvovaginal candidiasis, except in China and certain other countries where Scynexis already out-licensed the drug.

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Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

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Feng Zhang (Susan Walsh/AP Images)

In search of new way to de­liv­er gene ed­i­tors, CRISPR pi­o­neer turns to mol­e­c­u­lar sy­ringes

Bug bacteria are ruthless.

Some soil bacteria have evolved tiny, but deadly injection systems that attach to insect cells, perforate them and release toxins inside — killing a bug in just a few days’ time. Scientists, on the other hand, want to leverage that system to deliver medicines.

In a paper published Wednesday in Nature, MIT CRISPR researcher Feng Zhang and his lab describe how they engineered these syringes made by bacteria to deliver potential therapies like toxins that kill cancer cells and gene editors. With the help of an AI program, they developed syringes that can load proteins of their choice and selectively target human cells.

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Alec­tor cuts 11% of work­force as it dou­bles down on late-stage neu­ro pro­grams part­nered with GSK, Ab­b­Vie

A month after revealing plans to concentrate on its late-stage immuno-neurology pipeline, Alector is trimming its headcount by 11%.

The layoffs will impact around 30 employees across the organization, the company disclosed in an SEC filing, adding that the plan will “better align the company’s resources” with the new strategy. With $712.9 million in cash, cash equivalents and investments as of the end of 2022, Alector believes the reserves will now get it through 2025.

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CSL CEO Paul McKenzie (L) and CMO Bill Mezzanotte

Q&A: New­ly-mint­ed CSL chief ex­ec­u­tive Paul McKen­zie and chief med­ical of­fi­cer Bill Mez­zan­otte

Paul McKenzie took over as CEO of Australian pharma giant CSL this month, following in the footsteps of long-time CSL vet Paul Perreault.

With an eye on mRNA, and quickly commercializing its new, $3.5 million-per-shot gene therapy for hemophilia B, McKenzie and chief medical officer Bill Mezzanotte answered some questions from Endpoints News this afternoon about where McKenzie is going to take the company and what advances may be coming to market from CSL’s pipeline. Below is a lightly edited transcript.

Boehringer re­ports ro­bust sales led by type 2 di­a­betes and pul­monary drugs, promis­es more to come high­light­ing obe­si­ty

Boehringer Ingelheim reported human pharma sales of €18.5 billion on Wednesday, led by type 2 diabetes and heart failure drug Jardiance and pulmonary fibrosis med Ofev. Jardiance sales reached €5.8 billion, growing 39% year over year, while Ofev took in €3.2 billion, notching its own 20.6% annual jump.

However, Boehringer is also looking ahead with its pipeline, estimating “In the next seven years the company expects about 20 regulatory approvals in human pharma.”

FDA ap­proves Nar­can opi­oid over­dose re­ver­sal spray for over-the-counter sale

The FDA today approved Emergent BioSolutions’ Narcan brand naloxone nasal spray for over-the-counter sales. The nod was expected and comes on the heels of a unanimous 19-0 advisory committee vote in favor of approval last month.

The move to OTC means the opioid overdose reversal agent will now be available on grocery, convenience and gas stations shelves, as well as potentially for purchase online.

J&J bows out of RSV vac­cine race, end­ing PhI­II study and ced­ing to Pfiz­er, GSK

Johnson & Johnson announced Wednesday morning it is ending development of its adult RSV vaccine that was in the middle of a 27,200-patient trial, giving up a big slice of what’s expected to be the next multibillion-dollar pharma market.

The decision came down to the shifting RSV “competitive landscape,” a company spokesperson tells Endpoints News, adding the “breadth of options” was much different than when J&J first started its pivotal study. The spokesperson declined to comment on the Phase III data, saying only the shot is undergoing an “ongoing assessment.”

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