Warren Huff, Reata CEO

Rea­ta sug­gests Friedre­ich's atax­ia pro­gram could be de­layed, send­ing stock plung­ing

Rea­ta Phar­ma­ceu­ti­cals $RE­TA made waves last Oc­to­ber when its drug omavelox­olone pro­duced pos­i­tive tri­al re­sults in treat­ing a rare neu­ro­log­i­cal dis­or­der, but the can­di­date’s path for­ward be­came much murki­er Mon­day.

In a re­port of quar­ter­ly earn­ings, the biotech di­vulged that the FDA is con­sid­er­ing de­lay­ing omavelox­olone’s NDA pend­ing com­ple­tion of a sec­ond tri­al. That could push back ap­proval by at least a year giv­en that the tar­get pop­u­la­tion, in­di­vid­u­als with Friedre­ich’s atax­ia, is lim­it­ed and pro­gres­sion of the hard-to-treat ill­ness is no­to­ri­ous­ly slow. The Covid-19 pan­dem­ic would al­so hin­der Rea­ta’s abil­i­ty to com­plete an ad­di­tion­al tri­al.

The news shook Wall Street as Rea­ta stock closed down a lit­tle over 33% on the day.

Rea­ta’s pro­posed so­lu­tion to keep the de­lay as short as pos­si­ble in­volves sub­mit­ting a “crossover study” that would mea­sure the ef­fect of omavelox­olone in pa­tients who were pre­vi­ous­ly in the place­bo group and are be­ing treat­ed with the can­di­date in an open-la­bel ex­ten­sion study. Should the FDA ac­cept that ap­proach, Rea­ta ex­pects to com­plete the crossover study be­fore the end of the year and then sub­mit the NDA in the first quar­ter of 2021.

If the pro­pos­al is re­ject­ed or the da­ta do not re­turn pos­i­tive re­sults, how­ev­er, the biotech will re-eval­u­ate whether or not a sec­ond tri­al as out­lined is fea­si­ble. Re­gard­less of the out­come, Rea­ta not­ed it will con­tin­ue to pur­sue ap­proval out­side the Unit­ed States.

An­a­lysts did not im­me­di­ate­ly know what to make of the up­date. SVB Leerink’s Joseph Schwartz wrote to in­vestors that while Rea­ta seemed con­fi­dent it could per­suade the FDA to ac­cept its crossover tri­al, the biotech gave no in­di­ca­tion to which way reg­u­la­tors were lean­ing and “would not pro­vide guid­ance on how they ex­pect these dis­cus­sions to progress.” Schwartz fur­ther said that the pos­si­bil­i­ty of a first-quar­ter NDA “re­mains to be seen” and that their mod­el for Rea­ta’s stock pro­jec­tion is un­der­go­ing re­view.

With­in the quar­ter­ly up­date, Rea­ta al­so dis­closed that the FDA raised ques­tions about its oth­er lead pro­gram bar­dox­olone re­gard­ing Phase III tri­al re­sults with­in the sec­ond year of the study. If those re­sults are avail­able be­fore the year is out, the biotech could sub­mit the da­ta be­fore reg­u­la­tors make a de­ter­mi­na­tion on ac­cel­er­at­ed ap­proval, push­ing back the PDU­FA date but al­so re­sult­ing in full ap­proval in­stead. If full ap­proval is in­deed the route, the NDA fil­ing would be de­layed to the first quar­ter of 2021.

Again, Schwartz not­ed that Rea­ta did not hint at when the FDA be­gan ques­tion­ing the da­ta for this pro­gram. He went on to write that the biotech had po­ten­tial­ly spooked in­vestors with “less as­sertive” lan­guage in their 2019 10-K com­pared to their 2018 fil­ing, and the com­pa­ny would again not say whether this change co­in­cid­ed with reg­u­la­tors’ con­cerns.

Baird an­a­lyst Bri­an Sko­r­ney took a dif­fer­ent view, how­ev­er, call­ing the nose­dive an “over­re­ac­tion.” He not­ed that while con­cerns about omavelox­olone are “valid” and the drug’s ap­proval could be pushed as far back as 2023, bar­dox­olone is like­ly to pull a big­ger haul.

This is not the first time Rea­ta has faced head­winds in the last 12 months. Short­ly be­fore re­veal­ing their pos­i­tive omavelox­olone da­ta, Rea­ta un­cer­e­mo­ni­ous­ly de­cou­pled from a part­ner­ship with Ab­b­Vie that cost the biotech $330 mil­lion. Rea­ta was al­so forced to halt some tri­als due to Covid-19 com­pli­ca­tions, but con­tin­ued oth­ers by adapt­ing to at-home blood draws as op­posed to clin­ic vis­its.

The deal­ings with Ab­b­Vie go back sev­er­al years, as the com­pa­nies signed two part­ner­ships in rapid suc­ces­sion to the tune of $850 mil­lion in the ear­ly 2010s. But Ab­b­Vie sud­den­ly halt­ed its chron­ic kid­ney dis­ease stud­ies on bar­dox­olone in 2012 af­ter heart-re­lat­ed ad­verse ef­fects be­gan pop­ping up. Those ef­fects es­sen­tial­ly took the com­pound out of the run­ning in the CKD field, where CEO War­ren Huff had once vowed to make it a block­buster.

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.