Eric Dube, Travere Therapeutics CEO

Sev­er­al months af­ter re­brand­ing, an old Mar­tin Shkre­li com­pa­ny touts promis­ing piv­otal da­ta in a rare kid­ney dis­or­der

If all goes well at the FDA, Cal­lid­i­tas could cross the fin­ish line with its in­flam­ma­to­ry kid­ney dis­ease treat­ment Ne­fe­con next month. But Tra­vere Ther­a­peu­tics isn’t far be­hind — and on Mon­day, the for­mer Mar­tin Shkre­li com­pa­ny un­veiled some promis­ing piv­otal da­ta that may give Cal­lid­i­tas a run for its mon­ey.

Af­ter 36 weeks of treat­ment with Tra­vere’s sparsen­tan, pa­tients with a rare con­di­tion called IgA nephropa­thy saw a mean re­duc­tion in pro­tein­uria (ex­ces­sive pro­tein in the urine) of 49.8% from base­line, the com­pa­ny said. That com­pares to a mean re­duc­tion of 15.1% in a group of pa­tients who took Avapro, a cur­rent stan­dard of care (p<0.0001).

“We con­tin­ue to hear from the nephrol­o­gy com­mu­ni­ty that there is a clear need for a ther­a­peu­tic op­tion to mean­ing­ful­ly re­duce pro­tein­uria over and above wide­ly used ACE and ARB treat­ments,” CMO Noah Rosen­berg said on a call with in­vestors.

IgAN is a con­di­tion that dam­ages the fil­ters, or glomeruli, in­side the kid­neys, caus­ing the or­gans to lose their func­tion. Tra­vere’s Phase III study, dubbed PRO­TECT, en­rolled 404 pa­tients who ex­pe­ri­enced per­sis­tent pro­tein­uria de­spite re­ceiv­ing ACE or ARB treat­ment. They were ran­dom­ized to re­ceive once-dai­ly dos­es of ei­ther sparsen­tan or Avapro, an ARB treat­ment.

The in­ter­im re­sults showed promise for the sec­ondary end­points, which mea­sure es­ti­mat­ed glomeru­lar fil­tra­tion rate (eGFR) af­ter about one and two years, ac­cord­ing to Tra­vere.

“The Com­pa­ny be­lieves that pre­lim­i­nary eGFR da­ta avail­able at the time of the in­ter­im analy­sis are in­dica­tive of a po­ten­tial clin­i­cal­ly mean­ing­ful treat­ment ef­fect af­ter two years of treat­ment,” it said in a state­ment, adding that topline re­sults from the con­fir­ma­to­ry end­point analy­sis are com­ing in the sec­ond half of 2023.

Mean­while, CEO Er­ic Dube plans on fil­ing for an ac­cel­er­at­ed ap­proval in the first half of next year.

Cal­lid­i­tas al­ready has its foot in the door at the FDA, af­ter sub­mit­ting an NDA for Ne­fe­con to treat IgAN back in March. Reg­u­la­tors set a Sept. 15 PDU­FA date — and if ap­proved, the drug would be­come the first ap­proved treat­ment in the US for the rare kid­ney dis­or­der.

But while it’s dif­fi­cult to com­pare tri­als, it’s look­ing like sparsen­tan may have the up­per hand in pro­tein­uria re­duc­tion. In No­vem­ber, Cal­lid­i­tas said da­ta from the first part of a Phase III study showed pa­tients who took Ne­fe­con had a 31% re­duc­tion in pro­tein­uria af­ter nine months, com­pared to a 5% re­duc­tion of those on place­bo (p=0.005).

Back in Feb­ru­ary, Tra­vere tout­ed a Phase III win in an­oth­er rare kid­ney dis­or­der called fo­cal seg­men­tal glomeru­loscle­ro­sis. A full read­out from that study is al­so ex­pect­ed in 2023, though Dube says there are enough da­ta now to pur­sue ac­cel­er­at­ed ap­proval.

Tra­vere was found­ed as Retrophin by the “Phar­ma Bro” back in 2011, but has since gone to great lengths to sev­er its ties to Shkre­li (in­clud­ing a name change back in No­vem­ber). 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.

Up­on re­brand­ing the com­pa­ny, Dube told End­points News that the old name “doesn’t re­flect who we are to­day, and the role that we want to play go­ing in­to the fu­ture.” The new name is in­spired by the Latin roots of the words “path” (trac­tus) and “truth” (ver).

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

No­var­tis de­vel­op­ment chief John Tsai: 'We go deep in the new plat­form­s'

During our recent European Biopharma Summit, I talked with Novartis development chief John Tsai about his experiences over the 3-plus years he’s been at the pharma giant. You can read the transcript below or listen to the exchange in the link above.

John Carroll: I followed your career for quite some time. You’ve had more than 20 years in big pharma R&D and you’ve obviously seen quite a lot. I really was curious about what it was like for you three and a half years ago when you took over as R&D chief at Novartis. Obviously a big move, a lot of changes. You went to work for the former R&D chief of Novartis, Vas Narasimhan, who had his own track record there. So what was the biggest adjustment when you went into this position?

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Covid-19 vac­cine boost­ers earn big thumbs up, but Mod­er­na draws ire over world sup­ply; What's next for Mer­ck’s Covid pill?; The C-suite view on biotech; 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.

You may remember that at the beginning of this year, Endpoints News set a goal to go broader and deeper. We are still working towards that, and are excited to share that Beth Snyder Bulik will be joining us on Monday to cover all things pharma marketing. You can sign up for her weekly Endpoints MarketingRx newsletter in your reader profile.

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Susan Galbraith, Executive VP, Oncology R&D, AstraZeneca

As­traZeneca on­col­o­gy R&D chief Su­san Gal­braith: 'Y­ou're go­ing to need or­thog­o­nal com­bi­na­tion­s'

 

Earlier in the week we broadcast our 4th annual European Biopharma Summit with a great lineup of top execs. One of the one-on-one conversations I set up was with Susan Galbraith, the oncology research chief at AstraZeneca. In a wide-ranging discussion, Galbraith reviewed the cancer drug pipeline and key trends influencing development work at the pharma giant. You can watch the video, above, or stick with the script below. — JC

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Amit Etkin, Alto Neuroscience CEO (Alto via Vimeo)

A star Stan­ford pro­fes­sor leaves his lab for a start­up out to re­make psy­chi­a­try

About five years ago, Amit Etkin had a breakthrough.

The Stanford neurologist, a soft-spoken demi-prodigy who became a professor while still a resident, had been obsessed for a decade with how to better define psychiatric disorders. Drugs for depression or bipolar disorder didn’t work for many patients with the conditions, and he suspected the reason was how traditional diagnoses didn’t actually get at the heart of what was going on in a patient’s brain.

Roche's Tecen­triq cross­es the fin­ish line first in ad­ju­vant lung can­cer, po­ten­tial­ly kick­ing off gold rush

While falling behind the biggest PD-(L)1 drugs in terms of sales, Roche has looked to carve out a space for its Tecentriq with a growing expertise in lung cancer. The drug will now take an early lead in the sought-after adjuvant setting — but competitors are on the way.

The FDA on Friday approved Tecentriq as an adjuvant therapy for patients with Stage II-IIIA non small cell lung cancer with PD-(L)1 scores greater than or equal to 1, making it the first drug of its kind approved in an early setting that covers around 40% of all NSCLC patients.

Rahul Singhvi, Resilience CEO

A Bob Nelsen start­up turns to Har­vard to help sharp­en its tech, in­spir­ing first spin­out

One of Bob Nelsen’s latest projects is headed to Harvard.

Resilience, a company started with the goal of establishing itself as a “one-stop-shop” for companies looking to scale manufacturing, including for hard-to-develop cell and gene therapies, is less than a year old. Friday, it announced a five-year R&D deal with Harvard University that includes $30 million to develop biologics, including vaccines, nucleic acids and cell and gene therapies.

Susan Galbraith speaking at Endpoints News' virtual EUBIO21 summit

Imfinzi/treme­li­mum­ab com­bo scores As­traZeneca an­oth­er OS win — this time in liv­er can­cer

Is the tide turning on AstraZeneca’s battered PD-L1/CTLA4 combo?

A single priming dose of the experimental tremelimumab, followed by Imfinzi every four weeks, beat Nexavar (sorafenib) in helping a group of liver cancer patients live longer in a Phase III study, the company reported, meeting the primary endpoint.

Specifically, the two drugs extended overall survival for patients with unresectable hepatocellular carcinoma who had not received prior systemic therapy and were not eligible for localized treatment.

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FDA+ roundup: Marks on Wood­cock­'s tenure as act­ing com­mis­sion­er; FDA lead­ers of­fer per­spec­tive on bar­ri­ers to di­ver­si­ty in re­search

CBER director Peter Marks praised Janet Woodcock’s work as acting FDA commissioner, and while noting that Biden needs to nominate someone to fill the role permanently by Nov. 16, he said he has “no idea” when that actually might occur.

“Dr. Woodcock has been at the agency for over three decades and she, during that time, has proven herself to be a remarkably capable manager,” Marks said at the Alliance for Regenerative Medicine’s meeting Tuesday. “And she’s been managing as if she’s commissioner, unlike some previous acting [commissioners] who are afraid to actually do things. She doesn’t appear to be afraid to do things. I have not felt any different now from when we had a commissioner in place,” he added.

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