Take­da takes a $200M hit af­ter No­var­tis is forced to yank its mar­ket­ing ap­pli­ca­tion for Xi­idra

Now that the EMA has el­bowed No­var­tis’ ap­pli­ca­tion for Xi­idra off the ta­ble, the re­ver­ber­a­tions are be­ing felt at Take­da.

The phar­ma gi­ant not­ed ear­ly Mon­day that it will rec­og­nize a loss of $200 mil­lion for the cur­rent quar­ter af­ter the news hit late last week that the Eu­ro­pean reg­u­la­tor had de­cid­ed that the eye drug they sold to No­var­tis for $3.4 bil­lion in cash and $1.9 bil­lion in mile­stones hadn’t made its case on ef­fi­ca­cy. The drug has been mar­ket­ed in the US now for 4 years, and Take­da picked it up in the $62 bil­lion Shire buy­out and then prompt­ly sold it off to No­var­tis.

That de­ci­sion at the EMA is hit­ting the $1.9 bil­lion in po­ten­tial sales mile­stones, now that the Eu­ro­pean mar­ket is off the ta­ble. The con­ti­nen­tal reg­u­la­tor was clear­ly unim­pressed by the da­ta for Xi­idra, and be­came skep­ti­cal over the way the con­trol arm was han­dled by Shire.

In ex­plain­ing the with­draw­al, the EMA not­ed:

The Agency con­sid­ered that the ef­fec­tive­ness of Xi­idra was not demon­strat­ed across dif­fer­ent symp­toms of dry eye dis­ease. Al­though some ef­fect was seen in the re­duc­tion of eye dry­ness, the im­prove­ment was not con­sid­ered clin­i­cal­ly sig­nif­i­cant. In ad­di­tion, al­though Xi­idra was in­tend­ed to be used in pa­tients with more se­vere dis­ease in whom ar­ti­fi­cial tears had not been suf­fi­cient in im­prov­ing the con­di­tion, the Agency had some con­cerns about how these pa­tients were to be se­lect­ed, and not­ed that the stud­ies had com­pared Xi­idra with the ve­hi­cle, and had not used ar­ti­fi­cial tears in an op­ti­mal way. The Agency al­so not­ed that there were no da­ta on the ef­fect of long-term treat­ment with Xi­idra de­spite eye dry­ness be­ing a chron­ic (long-last­ing) dis­ease.

The drug, as No­var­tis ex­plained, is de­signed to block the in­ter­ac­tion be­tween LFA-1 1 and ICAM-1 2, in­hibit­ing T cell re­cruit­ment and ac­ti­va­tion and re­duc­ing pro-in­flam­ma­to­ry cy­tokine re­lease.

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.

Vas Narasimhan (AP Images)

UP­DAT­ED: Still held down by clin­i­cal hold, No­var­tis' Zol­gens­ma falls fur­ther be­hind Bio­gen and Roche as FDA asks for a new piv­otal study

Last October, the FDA slowed down Novartis’ quest to extend its gene therapy to older spinal muscular atrophy patients by slapping a partial hold on intrathecal administration. Almost a year later, the hold is still there, and regulators are adding another hurdle required for regulatory submission: a new pivotal confirmatory study.

The new requirement — which departs significantly from Novartis’ prior expectations — will likely stretch the path to registration beyond 2021, when analysts were expecting a BLA submission. That could mean more time for Biogen to reap Spinraza revenues and Roche to ramp up sales of Evrysdi in the absence of a rival.

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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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Rep. Andy Harris (R-MD) (Tasos Katopodis/Pool via AP Images)

The mi­cro-cap that tapped a mask-skep­tic con­gress­man for their Covid DSMB is ap­ply­ing for an EUA. Their ev­i­dence? 21 pa­tients

NeuroRx, the tiny biotech that came under fire last week after Politico reported they selected a congressman and two other acquaintances of the CEO to supervise their Covid-19 drug trial, announced today that they will ask the FDA to authorize their drug based on the results of just 21 patients.

Such an application would test the agency’s standards of evidence for an EUA, which have already come under scrutiny after controversial authorizations for convalescent plasma and hydroxychloroquine. The only other company to discuss their intention to file for an EUA, Eli Lilly, did so after results came back from a randomized control study testing their antibody in over 450 patients.