In a come­back, Por­to­la scores FDA ac­cel­er­at­ed ap­proval for once-re­ject­ed An­dexxa

Some­how stay­ing on the good side of the FDA, Por­to­la $PT­LA has scored an elu­sive reg­u­la­to­ry win for their once-re­ject­ed drug an­dex­anet al­fa.

The an­ti-an­ti­co­ag­u­lant — to be mar­ket­ed as An­dexxa — won the OK via the ac­cel­er­at­ed ap­proval path­way, mean­ing that Por­to­la will have to run a post-mar­ket­ing tri­al to demon­strate an abil­i­ty to con­trol bleed­ing in pa­tients.  The ap­proval was based on da­ta from two Phase III stud­ies, con­duct­ed among healthy vol­un­teers, that showed An­dexxa could re­verse an­ti­co­ag­u­lant ac­tiv­i­ty of the Fac­tor Xa in­hibitors Xarel­to (ri­varox­a­ban) and Eliquis (apix­a­ban). The drug al­so comes with a black box warn­ing and is in­di­cat­ed for use when re­ver­sal of an­ti­co­ag­u­la­tion is need­ed due to life-threat­en­ing or un­con­trolled bleed­ing.

Bill Lis

Por­to­la has fought long and hard for this win. It first sub­mit­ted an NDA for An­dexxa late 2015, on­ly to be caught off guard with an FDA re­jec­tion. At the time, the South San Fran­cis­co biotech said the agency was look­ing for more in­for­ma­tion on man­u­fac­tur­ing and how the an­ti­dote works in oth­er blood thin­ning drugs, such as edox­a­ban (Savaysa) and enoxa­parin (Lovenox).

The team filed again in Au­gust 2017, ex­act­ly a year af­ter it got the com­plete re­sponse let­ter. Reg­u­la­tors have since pushed back the ac­tion date from Feb­ru­ary to May 4, and Por­to­la hint­ed in a re­cent earn­ings call that there could be fur­ther de­lays as the FDA re­quest­ed fur­ther da­ta, send­ing stock down. But noth­ing came out of that warn­ing, and Por­to­la spread word about the OK late Thurs­day — right be­fore the orig­i­nal ac­tion date.

A spokesper­son told me that the CRL Por­to­la re­ceived in 2016 “fo­cused al­most sole­ly on man­u­fac­tur­ing con­cerns, which Por­to­la ad­dressed.”

“An­dexxa is a break­through prod­uct, and as such, clin­i­cal was much faster than man­u­fac­tur­ing,” they wrote. “In oth­er words, at that time, man­u­fac­tur­ing had to catch up with clin­i­cal.”

In a lengthy press re­lease, Por­to­la did not elab­o­rate on how it re­solved the FDA’s pre­vi­ous con­cerns, choos­ing in­stead to high­light the mar­ket po­ten­tial of its re­ver­sal agent in an era when Fac­tor Xa in­hibitors have be­come in­creas­ing pop­u­lar. One of them is Por­to­la’s own Bevyxxa, whose ap­proval raised some eye­brows since it bare­ly failed a piv­otal tri­al. Eu­ro­pean reg­u­la­tors gave it a thumbs down.

“We are proud that An­dexxa is a first-in-class med­i­cine dis­cov­ered in our labs,” said CEO Bill Lis. “We re­main com­mit­ted to our sci­en­tif­ic lead­er­ship in the fields of throm­bo­sis and hema­to­log­ic can­cers.”

The com­pa­ny now plans to launch an ear­ly sup­ply pro­gram of “Gen­er­a­tion 1” An­dexxa in June, with broad­er com­mer­cial launch an­tic­i­pat­ed in ear­ly 2019, as­sum­ing the FDA green­lights the man­u­fac­tur­ing process of its Gen­er­a­tion 2 prod­uct.

Mean­while, the post-mar­ket­ing tri­al is slat­ed to be­gin in 2019 and be re­port­ed in 2023.

Por­to­la shares edged up 3% in pre-mar­ket trad­ing. Ac­cord­ing to Eval­u­atePhar­maAn­dexxa car­ries a 2022 sell­side rev­enue fore­cast of $749m.

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

President Trump walks past HHS secretary Alex Azar (Getty Images)

Azar falls in line un­der Trump again. Ex­perts say he's re­in­forc­ing a dark sig­nal sent to the FDA

In the latest incident where Alex Azar has steadfastly taken the side of President Donald Trump over that of the FDA, the HHS secretary was noncommittal this morning when asked if he supports the attempt by his subordinates at the FDA to strengthen guidelines for a vaccine EUA.

Appearing on NBC’s Today Show, the HHS secretary muddied the waters, stating that the guidance that matters is the one that is “actually already out there.”

CDC’s Robert Redfield, NIAID’s Anthony Fauci, Admiral Brett Giroir at HHS, and FDA’s Stephen Hahn prepare to testify at a House hearing on June 23 (Getty)

'Ex­treme­ly po­lit­i­cal' — Trump neuters FDA's at­tempt to strength­en vac­cine EUA

Stephen Hahn went before a Senate committee Wednesday and declared he’s fighting. “Every one of the decisions we have reached has been made by career FDA scientists based on science and data, not politics,” he exclaimed, adding that “FDA will not permit any pressure from anyone to change that. I will fight for science.”

A few hours later, he was undermined by President Donald Trump when a reporter asked if he was okay with stricter vaccine guidelines that the FDA was said to be cooking up. “That has to be approved by the White House. We may or may not approve it. That sounds like a political move,” he decided.

New York governor Andrew Cuomo (AP Images)

An­drew Cuo­mo says New York will un­der­take its own vac­cine re­view process, and wouldn’t rec­om­mend trust­ing the fed­er­al gov­ern­ment

The concerns keep mounting over President Donald Trump’s politicization of the FDA and other federal agencies guiding the development of a safe and effective vaccine. And today, the telegenic New York governor Andrew Cuomo appeared to introduce even more politics into the matter — latest in an ongoing series of incidents that have cast the proudly independent FDA in starkly political terms.

During his daily press conference Cuomo said that the state will review any coronavirus vaccines approved by the federal government, citing a lack of trust of the Trump administration. The announcement comes one day after Trump accused the FDA of making an “extremely political” move in proposing stricter vaccine guidance.

Covid-19 roundup: Op­er­a­tion Warp Speed's 7th vac­cine is live at­ten­u­at­ed; Small biotech touts big suc­cess where gi­ants have failed

Operation Warp Speed is stacking its vaccine portfolio with a “TBD” new candidate: a live attenuated vaccine that can be administered in a single dose, potentially as an oral formulation rather than an injection.

Sound familiar?

That could be because the unannounced candidate appears to match the profile of an inoculation being developed by Merck, according to Bloomberg, which first reported the development based on a presentation by Moncef Slaoui.

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On­ly five months af­ter a Se­ries A launch, Taysha goes pub­lic with $157M IPO

As has been the trend in 2020, Taysha Gene Therapies has become the latest biotech to make a quick ascent from a small, privately-funded company to enjoying its very own Nasdaq ticker.

The Dallas-based biotech raised $157 million for its IPO after pricing shares at $20 apiece Thursday, the high-point of its expected range. Initially pegging $100 million in financing, Taysha offered a little less than 8 million shares and will trade under the $TSHA symbol.

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David Berry (Flagship)

Flag­ship's next big tech­no­log­i­cal bet? The cloud

Earlier this month, Flagship announced their big bet on the software half the industry is talking about, launching the AI and machine learning startup. Now, they and a couple other investors are gambling $100 million on a software that much of the public generally thinks of as a cool, IT afterthought: cloud computing.

The idea, says founder and Flagship partner David Berry, is one of scale: The sheer magnitude of biological data that you can store on cloud technology is unprecedented. And that size, when leveraged properly, can allow you to ask questions and form insights that are similarly unprecedented.

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PhII Alzheimer's fail­ure deals new blow to Roche, AC Im­mune — but the tau hy­poth­e­sis is far from dead

The leading anti-tau antibody has failed its first Phase II testing, casting a shadow on a popular target (just trailing amyloid beta) for Alzheimer’s disease.

Roche and AC Immune are quick to acknowledge disappointment in the topline readout, which suggested that semorinemab did not reduce cognitive decline among patients with early Alzheimer’s disease, who are either just starting to have symptoms or have mild manifestations.

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Covid-19 roundup: J&J be­gins piv­otal Phase III tri­al for vac­cine; Con­tro­ver­sial hu­man chal­lenge tri­als to be­gin in Lon­don — re­port

Johnson & Johnson announced it’s beginning a pivotal Phase III trial for its Covid-19 candidate, JNJ-78436735 — the first single-dose vaccine in this stage.

The Phase III trial, dubbed ENSEMBLE, will enroll 60,000 patients worldwide, making it the largest Phase III study of a Covid-19 vaccine to date. J&J said the candidate achieved positive interim results in a Phase I/IIa study, which will be published “imminently.” There’s a possibility that the first batches will be ready for potential emergency use in early 2021, according to the company.

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