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.”

Biogen CEO Michel Vounatsos (via Getty Images)

With ad­u­canum­ab caught on a cliff, Bio­gen’s Michel Vounatsos bets bil­lions on an­oth­er high-risk neu­ro play

With its FDA pitch on the Alzheimer’s drug aducanumab hanging perilously close to disaster, Biogen is rolling the dice on a $3.1 billion deal that brings in commercial rights to one of the other spotlight neuro drugs in late-stage development — after it already failed its first Phase III.

The big biotech has turned to Sage Therapeutics for its latest deal, close to a year after the crushing failure of Sage-217, now dubbed zuranolone, in the MOUNTAIN study.

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Pascal Soriot (AP Images)

As­traZeneca, Ox­ford on the de­fen­sive as skep­tics dis­miss 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.

With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

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John Maraganore, Alnylam CEO (Scott Eisen/Bloomberg via Getty Images)

Al­ny­lam gets the green light from the FDA for drug #3 — and CEO John Maraganore is ready to roll

Score another early win at the FDA for Alnylam.

The FDA put out word today that the agency has approved its third drug, lumasiran, for primary hyperoxaluria type 1, better known as PH1. The news comes just 4 days after the European Commission took the lead in offering a green light.

An ultra rare genetic condition, Alnylam CEO John Maraganore says there are only some 1,000 to 1,700 patients in the US and Europe at any particular point. The patients, mostly kids, suffer from an overproduction of oxalate in the liver that spurs the development of kidney stones, right through to end stage kidney disease.

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Jason Kelly, Ginkgo Bioworks CEO (Kyle Grillot/Bloomberg via Getty Images)

Af­ter Ko­dak de­ba­cle, US lends $1.1B to a syn­thet­ic bi­ol­o­gy com­pa­ny and their big Covid-19, mR­NA plans

In mid-August, as Kodak’s $765 million government-backed push into drug manufacturing slowly fell apart in national headlines, Ginkgo Bioworks CEO Jason Kelly got a message from his company’s government liaison: HHS wanted to know if they, too, might want a loan.

The government’s decision to lend Kodak three quarters of a billion dollars raised eyebrows because Kodak had never made drugs before. But Ginkgo, while not a manufacturing company, had spent the last decade refining new ways to produce materials inside cells and building automated facilities across Boston.

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Bax­ter con­tin­ues on-shoring push with $50M In­di­ana ex­pan­sion

It’s been a banner year for the once humdrum business of manufacturing drugs, particularly vaccines. Billions have been spent ramping up facilities for Covid-19 jabs, while individual CDMOs have expanded their facilities, apparently anticipating demand or responding to a government-led push to onshore drug manufacturing.

Now Baxter Biopharma Solutions, the CDMO wing of the many-armed healthcare giant Baxter, is getting in on the game. On Tuesday, they announced plans to spend $50 million to expand their flagship, 600,000 square-foot facility in Bloomington, IN.

Eu­ro­pean Union aims to es­tab­lish patent workaround in case of emer­gen­cies while try­ing to strength­en its own IP

The European Union is looking at ways to bypass patent protections and make it easier to make generic drugs in cases of emergency such as the Covid-19 pandemic, a new document says.

Normally, under WTO regulations, the practice known as “compulsory licensing” is allowed in exceptional circumstances and could be applied as a waiver to bypass patent holders. Wednesday’s document was published as part of the EU’s plan to shore up the intellectual property rights of its member states.

Pur­due Phar­ma pleads guilty in fed­er­al Oxy­Con­tin probe, for­mal­ly rec­og­niz­ing it played a part in the opi­oid cri­sis

Purdue Pharma, the producer of the prescription painkiller OxyContin, admitted Tuesday that, yes, it did contribute to America’s opioid epidemic.

The drugmaker formally pleaded guilty to three criminal charges, the AP reported, including getting in the way of the DEA’s efforts to combat the crisis, failing to prevent the painkillers from ending up on the black market and encouraging doctors to write more painkiller prescriptions through two methods: paying them in a speakers program and directing a medical records company to send them certain patient information. Purdue’s plea deal calls for $8.3 billion in criminal fines and penalties, but the company is only liable for a fraction of that total — $225 million.

PhRMA sues Trump gov­ern­ment over drug im­por­ta­tion rule — days be­fore it's set to be ef­fec­tive

Ever since President Donald Trump floated the idea of using state-sponsored importation to lower drug prices, PhRMA has made its opposition abundant. Not only is the proposal dangerous and futile,  but the trade group has also argued that it may even be illegal.

Now that the FDA has issued its final rule permitting states to bring certain drugs from Canada, PhRMA is taking the government to court — just a few days before the rule is slated to take effect.

The ad­u­canum­ab co­nun­drum: The PhI­II failed a clear reg­u­la­to­ry stan­dard, but no one is cer­tain what that means any­more at the FDA

Eighteen days ago, virtually all of the outside experts on an FDA adcomm got together to mug the agency’s Billy Dunn and the Biogen team when they presented their upbeat assessment on aducanumab. But here we are, more than 2 weeks later, and the ongoing debate over that Alzheimer’s drug’s fate continues unabated.

Instead of simply ruling out any chance of an approval, the logical conclusion based on what we heard during that session, a series of questionable approvals that preceded the controversy over the agency’s recent EUA decisions has come back to haunt the FDA, where the power of precedent is leaving an opening some experts believe can still be exploited by the big biotech.

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