Eric Dube, Travere Therapeutics CEO

Tra­vere fails to con­vince reg­u­la­tors to con­sid­er ac­cel­er­at­ed ap­proval for a rare kid­ney dis­ease — again

As the old say­ing goes, ‘If at first you don’t suc­ceed, try, try again.’ The ori­gin of this mantra is dis­put­ed but has been wide­ly at­trib­uted to Robert the Bruce, the first king of Scot­land, as he and his army did bat­tle with in­vad­ing Eng­lish forces in the ear­ly 14th cen­tu­ry.

But what hap­pens if you try a sec­ond time and again don’t suc­ceed?

Tra­vere Ther­a­peu­tics will be ask­ing it­self that ques­tion af­ter a sec­ond at­tempt to gun for ac­cel­er­at­ed ap­proval was re­ject­ed by the FDA, the com­pa­ny an­nounced Wednes­day af­ter­noon. The for­mer Mar­tin Shkre­li biotech had at­tempt­ed to push through its lead drug, sparsen­tan, to a speedy OK in a rare kid­ney dis­ease, but reg­u­la­tors pre­fer the more me­thod­i­cal, full ap­proval route.

The way Tra­vere tells it, in­ter­im da­ta from its Phase III DU­PLEX study and re­cent ad­di­tion­al eGFR da­ta do not meet the FDA’s “thresh­old to sup­port an ap­pli­ca­tion for ac­cel­er­at­ed ap­proval in FS­GS,” per a press re­lease. Reg­u­la­tors re­main open to ap­prov­ing sparsen­tan based on the DU­PLEX tri­al once it’s com­plete, the biotech added, rec­om­mend­ing the com­pa­ny “pur­sue tra­di­tion­al ap­proval based on two-year eGFR slope.”

An­a­lysts had been look­ing for an up­date here ahead of Tra­vere’s sec­ond quar­ter earn­ings re­port, which will take place Thurs­day morn­ing. Wednes­day’s out­come had been fore­cast un­der a po­ten­tial “bear case” sce­nario by Jef­feries’ Mau­ry Ray­croft last month.

But in­vestors didn’t ap­pear as con­cerned as Ray­croft, who had pre­dict­ed a rough­ly 30% stock drop should this up­date come through. While Tra­vere shares $TVTX did fall post-mar­ket, they on­ly dropped about 10% and re­mained there be­fore Thurs­day’s open­ing bell.

Tra­vere is de­vel­op­ing sparsen­tan to treat fo­cal seg­men­tal glomeru­loscle­ro­sis (FS­GS), a rare kid­ney dis­ease where scar tis­sue builds up on the kid­ney’s fil­ters and af­fects pa­tients’ abil­i­ty to re­move waste from their blood. In Feb­ru­ary 2021, Tra­vere read out a pos­i­tive, in­ter­im Phase III analy­sis and pledged to win the first ac­cel­er­at­ed ap­proval for FS­GS.

But that May, US reg­u­la­tors re­ject­ed the com­pa­ny’s ac­cel­er­at­ed ap­proval pitch say­ing da­ta from the in­ter­im read­out “would not be ad­e­quate” to sup­port a quick OK. At the time, Tra­vere had hoped ad­di­tion­al eGFR da­ta could per­suade the agency to re­con­sid­er, but that did not turn out to be the case.

In­stead, Tra­vere will now pur­sue the tra­di­tion­al ap­proval af­ter the DU­PLEX study reads out full da­ta, ex­pect­ed in the first half of next year. If every­thing turns out pos­i­tive, it could lead to an NDA fil­ing by the end of 2023, Tra­vere says.

“While we are dis­ap­point­ed that we will not be fil­ing for ac­cel­er­at­ed ap­proval of sparsen­tan in FS­GS, the eGFR da­ta in the DU­PLEX Study have con­tin­ued to progress in a man­ner con­sis­tent with the pro­file of sparsen­tan and we re­main con­fi­dent that the study can sup­port an ap­pli­ca­tion for full ap­proval in FS­GS next year,” CEO Er­ic Dube said in a state­ment.

Else­where, the com­pa­ny’s pitch for sparsen­tan in IgA nephropa­thy re­mains on track, Tra­vere added Wednes­day, with a PDU­FA date on track for Nov. 17. The biotech man­aged to pur­sue ac­cel­er­at­ed ap­proval in this in­di­ca­tion, and af­ter a re­cent FDA meet­ing, ex­ecs say no ad­vi­so­ry com­mit­tee is cur­rent­ly planned.

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Ted Love, Global Blood Therapeutics CEO

Up­dat­ed: Pfiz­er scoops up Glob­al Blood Ther­a­peu­tics and its sick­le cell ther­a­pies for $5.4B

Pfizer is dropping $5.4 billion to acquire Global Blood Therapeutics.

Just ahead of the weekend, word got out that Pfizer was close to clinching a $5 billion buyout — albeit with other potential buyers still at the table. The pharma giant, flush with cash from Covid-19 vaccine sales, apparently got out on top.

The deal immediately swells Pfizer’s previously tiny sickle cell disease portfolio from just a Phase I program to one with an approved drug, Oxbryta, plus a whole pipeline that, if all approved, the company believes could make for a $3 billion franchise at peak.

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BREAK­ING: Math­ai Mam­men makes an abrupt ex­it as head of the big R&D group at J&J

In an after-the-bell shocker, J&J announced Monday evening that Mathai Mammen has abruptly exited J&J as head of its top-10 R&D group.

Recruited from Merck 5 years ago, where the soft spoken Mammen was being groomed as the successor to Roger Perlmutter, he had been one of the top-paid R&D chiefs in biopharma. His group spent $12 billion last year on drug development, putting it in the top 5 in the industry.

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FDA commissioner Rob Califf (Tom Williams/CQ Roll Call via AP Images)

With drug pric­ing al­most done, Con­gress looks to wrap up FDA user fee leg­is­la­tion

The Senate won’t return from its summer recess until Sept. 6, but when it does, it officially has 18 business days to finalize the reauthorization of the FDA user fee programs for the next 5 years, or else thousands of drug and biologics reviewers will be laid off and PDUFA dates will vanish in the interim.

FDA commissioner Rob Califf recently sent agency staff a memo explaining how, “Our latest estimates are that we have carryover for PDUFA [Prescription Drug User Fee Act], the user fee funding program that will run out of funding first, to cover only about 5 weeks into the next fiscal year.”

Pascal Soriot, AstraZeneca CEO (David Zorrakino/Europa Press via AP Images)

As­traZeneca and Dai­ichi Sankyo sprint to mar­ket af­ter FDA clears En­her­tu in just two weeks

Regulators didn’t keep AstraZeneca and Daiichi Sankyo waiting long at all for their latest Enhertu approval.

The partners pulled a win on Friday in HER2-low breast cancer patients who’ve already failed on chemotherapy, less than two weeks after its supplemental BLA was accepted. While this isn’t the FDA’s fastest approval — Bristol Myers Squibb won an OK for its blockbuster checkpoint inhibitor Opdivo in just five days back in March — it comes well ahead of Enhertu’s original Q4 PDUFA date.

Steve Paul, Karuna Therapeutics CEO (Third Rock)

Karuna's schiz­o­phre­nia drug pass­es a close­ly-watched PhI­II test, will head to FDA in mid-2023

An investigational pill that combines a former Eli Lilly CNS compound with an overactive bladder drug was better than placebo at reducing a scale of symptoms experienced by patients with schizophrenia in a Phase III trial.

Karuna Therapeutics’ drug passed the primary goal in EMERGENT-2, the Boston biotech said early Monday morning, alongside quarterly earnings. The study is the first of Karuna’s four Phase III clinical trials to read out in schizophrenia and will provide the backbone to the biotech’s first drug approval application, slated for mid-2023.

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No­vavax shares shred­ded as Covid vac­cine sales fall more than 90% in Q2

Months after Novavax celebrated its first profitable quarter as a commercial company, the Gaithersburg, MD-based company is back in the red.

Sales for Novavax’s Covid-19 vaccine slipped to $55 million last quarter, down from $586 million in Q1, CEO Stanley Erck revealed on Monday after market close. The company’s stock $NVAX plummeted more than 32% in after-hours trading.

Upon kicking off the call with analysts and investors, Erck addressed the elephant in the room:

Uğur Şahin, BioNTech CEO (Kay Nietfeld/picture-alliance/dpa/AP Images)

De­spite falling Covid-19 sales, BioN­Tech main­tains '22 sales guid­ance

While Pfizer raked in almost $28 billion last quarter, its Covid-19 vaccine partner BioNTech reported a rise in total dose orders but a drop in sales.

The German biotech reported over $3.2 billion in revenue in Q2 on Monday, down from more than $6.7 billion in Q1, in part due to falling Covid sales. While management said last quarter that they anticipated a Covid sales drop — CEO Uğur Şahin said at the time that “the pandemic situation is still very much uncertain” — Q2 sales still missed consensus by 14%.

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FDA's vac­cine ad­comm to re­view first fe­cal trans­plant to treat C. dif­fi­cile in­fec­tions

Back in 2018, Swiss drugmaker Ferring Pharmaceuticals made a big bet on Minnesota-based Rebiotix, buying up the company for its experimental poop-based drug implant to treat an infection caused by C. difficile, a potentially dangerous bacteria, in a new way.

Four years later, Ferring’s fecal microbiota transplant, dubbed RBX2660 or Rebyota, will face the FDA’s adcomm of outside vaccine experts on Sept. 22, debating whether the agency should license the transplant as a treatment for adults following antibiotic treatment for recurrent C. difficile infection.