John Butler, Akebia CEO

Ake­bi­a's ane­mia drug flunked Covid study — but count the bruised biotech 'ex­treme­ly en­cour­aged'

For as long as it’s ex­ist­ed — since 2007 and count­ing — Ake­bia has al­ways billed it­self as a kid­ney dis­ease spe­cial­ist. But as it grap­ples with a cost­ly FDA re­jec­tion of its lead ane­mia drug, the biotech is do­ing what­ev­er it can to stay afloat.

And that could mean hold­ing on­to a Covid-19 pro­gram even af­ter fail­ing a tri­al.

Re­port­ing ini­tial find­ings from a study test­ing vadadu­s­tat, its once-re­ject­ed ane­mia drug, for the pre­ven­tion and treat­ment of acute res­pi­ra­to­ry dis­tress syn­drome in pa­tients with Covid-19 and hy­pox­emia, Ake­bia said the drug failed the pri­ma­ry end­point, as mea­sured by the Na­tion­al In­sti­tute of Al­ler­gy and In­fec­tious Dis­ease Or­di­nal Scale.

But, ac­cord­ing to the biotech, the drug on­ly missed the goal by a hair: While the pri­ma­ry su­pe­ri­or­i­ty thresh­old over place­bo was set at 95%, re­searchers cal­cu­lat­ed a 94% prob­a­bil­i­ty that their drug was bet­ter than place­bo. And that means they won’t throw in the tow­el just yet.

“While the tri­al missed its pre­spec­i­fied pri­ma­ry end­point at Day14, we are ex­treme­ly en­cour­aged by the da­ta and be­lieve they sup­port fur­ther de­vel­op­ing vadadu­s­tat as a treat­ment for ARDS due to COVID-19 or oth­er caus­es,” CEO John But­ler said in a state­ment. “We will now work to re­view the full da­ta set more thor­ough­ly, con­sult with ex­perts in the field and ul­ti­mate­ly con­sult FDA on a po­ten­tial path for­ward.”

The tri­al was con­duct­ed by the Uni­ver­si­ty of Texas Health Sci­ence Cen­ter and par­tial­ly fund­ed by Ake­bia. It en­rolled 449 pa­tients.

Ake­bia has been grap­pling with a cas­cade of set­backs that be­gan in March, when the FDA served up a com­plete re­sponse let­ter to its ap­pli­ca­tion for vadadu­s­tat in ane­mia due to chron­ic kid­ney dis­ease. Reg­u­la­tors were con­cerned with safe­ty, specif­i­cal­ly in­creased risk of throm­boem­bol­ic events, and asked Ake­bia to do an­oth­er tri­al.

Just days af­ter the slap­down, the agency is­sued a par­tial clin­i­cal hold on the drug’s pe­di­atric stud­ies, trig­ger­ing a ma­jor round of lay­offs. Then in May, Ot­su­ka called off its years­long li­cens­ing and co-de­vel­op­ment pact with Ake­bia, quash­ing hopes of earn­ing more than $1 bil­lion in fu­ture mile­stones.

Ex­ecs are now bet­ting that rev­enues from their on­ly mar­ket­ed drug, Au­ryx­ia, will fund the com­pa­ny for the next few years while they try to turn things around by seek­ing Eu­ro­pean ap­proval of vadadu­s­tat and find­ing a new part­ner.

Shares $AK­BA, which al­ready nose­dived in­to pen­ny stock, slipped some more to $0.40 in pre-mar­ket trad­ing.

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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.

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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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 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.

David Reese, Amgen R&D chief

UP­DAT­ED: In a fresh dis­ap­point­ment, Am­gen spot­lights a ma­jor safe­ty is­sue with KRAS com­bo

Amgen had hoped that its latest study matching its landmark KRAS G12C drug Lumakras with checkpoint inhibitors would open up its treatment horizons and expand its commercial potential. Instead, the combo spurred safety issues that blunted efficacy and forced the pharma giant to alter course on its treatment strategy, once again disappointing analysts who have been tracking the drug’s faltering sales and limited therapeutic reach.

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