FDA gives thumbs-down on hear­ing loss drug, cit­ing man­u­fac­tur­ing is­sues, and sends Fen­nec stock reel­ing

A small, North Car­oli­na-based biotech has run in­to some head­winds in its ef­forts to de­vel­op a com­pound that pre­vents hear­ing loss as­so­ci­at­ed with cis­platin chemother­a­py treat­ments.

The FDA hand­ed down a com­plete re­sponse let­ter to Fen­nec Phar­ma­ceu­ti­cals $FENC for its Ped­mark drug late Mon­day night, cit­ing man­u­fac­tur­ing is­sues with the com­pa­ny’s sup­pli­er. Fen­nec hopes to meet with reg­u­la­tors some­time with­in the next month or two, CEO Ros­ty Raykov said in a call with in­vestors Tues­day morn­ing, in or­der to re­solve the is­sue.

Raykov fur­ther em­pha­sized dur­ing the call that there were no clin­i­cal safe­ty or ef­fi­ca­cy prob­lems and an ad­di­tion­al tri­al was not re­quired.

Nonethe­less, the news rat­tled Wall Street, as the biotech’s stock was down more than 35% in morn­ing trad­ing.

Per Raykov, Fen­nec re­ceived a Form 483 from the agency, which list­ed con­di­tions or prac­tices that are re­quired to be re­solved pri­or to Ped­mark’s ap­proval. When asked by in­vestors what specif­i­cal­ly need­ed to be fixed, Raykov de­murred and re­it­er­at­ed that there were no safe­ty is­sues.

Though the com­pa­ny’s goal is to meet with the FDA some­time soon, Raykov couldn’t pro­vide an ex­act time­line due to the CRL be­ing is­sued late last night, he said, and Fen­nec is still hash­ing out a plan.

De­spite the is­sues cit­ed Mon­day night, Fen­nec is not plan­ning on switch­ing man­u­fac­tur­ers, Raykov said. Giv­en that the same fa­cil­i­ty pro­duces planned Ped­mark batch­es for both the Amer­i­can and Eu­ro­pean mar­kets, Fen­nec aims to iron out the headaches as quick­ly as pos­si­ble. Fen­nec’s Eu­ro­pean ap­pli­ca­tion for Ped­mark is still un­der EMA re­view.

There are cur­rent­ly no ap­proved drugs that pre­vent oto­tox­i­c­i­ty, or hear­ing loss, in plat­inum-based chemother­a­pies. Ped­mark, a for­mu­la­tion of sodi­um thio­sul­fate, was aim­ing for a la­bel in pe­di­atric pa­tients old­er than one month to 18 years old as the hear­ing loss stem­ming from such chemother­a­pies is of­ten long-last­ing and per­ma­nent. Typ­i­cal­ly, pa­tients who end up los­ing their hear­ing must opt for cochlear im­plants.

Ped­mark re­ceived fast track des­ig­na­tion in March 2018 fol­low­ing two Phase III stud­ies in the sur­vival and re­duc­tion of oto­tox­i­c­i­ty in five pe­di­atric can­cers that typ­i­cal­ly re­quire in­ten­sive cis­platin treat­ments.

Raykov reaf­firmed Tues­day that Ped­mark’s main func­tion is to “pre­serve hear­ing with­out re­duc­ing the ef­fec­tive­ness of cis­platin treat­ment,” and he is con­fi­dent the com­pa­ny can ap­pease the FDA in short or­der.

Janet Woodcock (AP Images)

Janet Wood­cock to be act­ing FDA com­mis­sion­er while Biden team fi­nal­izes nom­i­nee — re­ports

Janet Woodcock is set to be the most powerful person at the FDA in less than a week.

The veteran regulator and longtime director of the Center for Drug Evaluation and Research has been tapped as acting commissioner of the FDA, according to reports by BioCentury’s Steve Usdin and Pink Sheet’s Sarah Karlin-Smith.

The appointment was requested by the incoming Biden team, Karlin-Smith added, as they sort out the nomination of a permanent successor to Stephen Hahn — whose one-year tenure has been defined by Covid-19.

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Janet Woodcock (AP Images)

Janet Wood­cock is in the run­ning for FDA com­mis­sion­er — what does that mean for the agen­cy's fu­ture?

Just a day after reports emerged that Janet Woodcock will serve as interim chief of the FDA, word has gotten out that she is also in the running for the permanent job.

The decision, as the initial wave of reactions suggest, could have dramatic implications for where the agency is headed in the next four years — if not beyond.

Woodcock, the longtime CDER director, is being vetted alongside former FDA principal deputy commissioner Joshua Sharfstein, Bloomberg reported. Already tapped as acting head of the agency, she’s set to take over from Stephen Hahn right after Biden’s inauguration next week.

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Steve Harr (L) and Hans Bishop

Paint­ing by the num­bers, Sana founders carve up a gi­ant uni­corn-sized IPO — for a biotech that has­n't quite made it to the clin­ic

Sana Biotechnology is one of those startups that was sketched in on the chalkboard day one in the shape of a unicorn.

A giant unicorn.

And from the numbers the cell therapy 2.0 play spelled out in their S-1 $SANA, it’s clear that the company founders — led by a pair of major VCs aligned with some high-profile industry figures — are hunting a big chunk of that value for themselves.

The raise they penciled in — $150 million — isn’t likely what they actually have in mind, and it doesn’t do justice to the size of their ambitions.

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CEO Brett Monia (Ionis)

Can Brett Mo­nia push Io­n­is be­yond Spin­raza?

For 30 years, Brett Monia struggled as one of Ionis’ top scientists to get their antisense technology to work. Now, as CEO, he’s trying to use it to turn Ionis into one of the industry’s biggest biotechs.

Monia, one of the handful of young scientists who in 1989 followed Stanley Crooke across the country from SmithKline (now GSK) in Philadelphia to found Ionis in Northern California, replaced Crooke as CEO last January. By then, they had proven antisense, an RNA-based method for manipulating gene expression, could work dramatically well in at least some instances, transforming spinal muscular atrophy with the Biogen-partnered blockbuster Spinraza.

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David Kessler in April 2009 (Eric Risberg/AP Images)

Covid-19 roundup: Hack­ers start re­leas­ing 'ma­nip­u­lat­ed' Covid-19 vac­cine docs; Ex-FDA com­mish David Kessler to re­place Mon­cef Slaoui as Op­er­a­tion Warp Speed chief — re­port

There’s a new twist on the EMA Covid-19 hacking story.

Friday the European agency put out the 5th in a series of statements about the hackers who broke into their system, noting that some of the information on vaccines that was gleaned in the attack is showing up online — altered to raise questions about the Covid-19 vaccines now in use.

This included internal/confidential email correspondence dating from November, relating to evaluation processes for COVID-19 vaccines. Some of the correspondence has been manipulated by the perpetrators prior to publication in a way which could undermine trust in vaccines.

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Terry Rosen, Arcus CEO

Gilead part­ner Ar­cus earns an­a­lyst­s' plau­dits for ear­ly pan­cre­at­ic can­cer da­ta that 'ex­ceed­ed ex­pec­ta­tion­s'

Arcus’ small molecule CD73 inhibitor for pancreatic cancer got a standing ovation from analysts who said preliminary data “exceeded expectations”— making waves in a field that’s seen little progress in several years and proving the candidate could be worth the hundreds of millions Gilead provided upfront in a deal that included more than a billion dollars for opt-in rights and milestones.

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News brief­ing: Five Prime fi­nal­izes PhI­II plans for gas­tric can­cer; AI di­ag­nos­tics-fo­cused Paige ex­pands staff

Five Prime Therapeutics has finalized a plan to take their comeback gastric cancer drug into late-stage studies.

The South San Francisco-based biotech released full Phase II data for bemarituzumab on Friday, which Five Prime said in November met all of its pre-specified efficacy endpoints in a topline readout. Now, the company is announcing it plans to launch a Phase III trial for the program in 2021. Following November’s readout, the future of bemarituzumab had not yet been finalized.

Peter Thiel, Getty (Photographer: Kiyoshi Ota/Bloomberg)

Pe­ter Thiel's psy­che­delics-fo­cused ATAI ac­quires ma­jor­i­ty stake in Recog­ni­fy and its lead schiz­o­phre­nia can­di­date

Billionaire Peter Thiel has made significant and sometimes controversial pushes into life sciences over the past few years, and one of his startups out of Berlin has made a new acquisition less than two months after achieving unicorn status.

ATAI Life Sciences purchased a majority stake Tuesday in Recognify Life Sciences, a company focused on developing treatments for cognitive impairment associated with schizophrenia. The financial terms of the deal weren’t disclosed, but the acquisition follows up a $125 million Series C in November co-led by Thiel, leading to a post-money valuation of about $1 billion for ATAI.

Cog­nate dou­bles man­u­fac­tur­ing ca­pac­i­ties in Mem­phis, Eu­rope, as de­mand for cell and gene ther­a­pies sky­rock­ets

The marketplace for gene and cell manufacturing therapeutics continues to be scorching.

Cognate BioSciences, a leading CDMO specializing in gene and cell therapy technologies, announced plans Friday that will double its total manufacturing capacities at sites in both the US and Europe — in direct response to a “great demand of commercial capacity within the biologics industry.”

The company provided most details for its US expansion, which will take place at its current headquarters in Memphis, Tennessee near the Memphis International Airport — crucial, it said, as Memphis is one of the world’s busiest cargo airports. Cognate will add two separate facilities totaling 250,000-square-feet: a GMP distribution center to manage global supply chain needs, and a third site for commercial manufacturing.