TG Ther­a­peu­tic­s' shares jump as um­bral­is­ib wins FDA's break­through sta­tus

About a month af­ter the pub­li­ca­tion of en­cour­ag­ing re­sults from an ear­ly-stage study eval­u­at­ing J&J’s Im­bru­vi­ca in com­bi­na­tion with its ex­per­i­men­tal drug um­bral­is­ib, TG Ther­a­peu­tics on Tues­day said the drug has been grant­ed the FDA’s break­through ther­a­py sta­tus for use in pa­tients with mar­gin­al zone lym­phoma (MZL), a type of can­cer with no specif­i­cal­ly ap­proved ther­a­pies.

The New York-based com­pa­ny’s stock $TGTX jumped about 12% in pre-mar­ket trad­ing.

The des­ig­na­tion was award­ed on the ba­sis of in­ter­im da­ta from a co­hort of MZL pa­tients, who have un­der­gone at least one pri­or ther­a­py, in an on­go­ing mid-stage study — UNI­TY-NHL — test­ing um­bral­is­ib, which is a PI3K delta in­hibitor. Re­sults from the co­hort are ex­pect­ed by the mid­dle of the year. MZL com­pris­es slow grow­ing B-cell non-Hodgkin lym­phomas that orig­i­nate in the mar­gin­al zone of lym­phoid tis­sue.

Once a hot prospect in can­cer R&D, the PI3K path­way has in­spired mul­ti­ple drug pro­grams but seen lim­it­ed de­vel­op­men­tal suc­cess due to study fail­ures, safe­ty con­cerns and dura­bil­i­ty is­sues. The first ever PI3K in­hibitor, Gilead’s Zy­delig, was ap­proved by the FDA in 2014, and car­ries a boxed warn­ing high­light­ing its po­ten­tial­ly dead­ly side ef­fects. Since then, the US agency has giv­en the nod to oth­er such drugs in­clud­ing Bay­er’s Aliqopa. A pletho­ra of PI3K in­hibitors are al­so in de­vel­op­ment.

TG’s um­bral­is­ib is al­so cur­rent­ly in late-stage de­vel­op­ment in com­bi­na­tion with the com­pa­ny’s oth­er ex­per­i­men­tal drug, ubli­tux­imab, for pa­tients with hema­to­log­ic ma­lig­nan­cies.

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.”

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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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.

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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Laura Shawver (Silverback Therapeutics)

Fol­low­ing a hefty Se­ries B, Sil­ver­back Ther­a­peu­tics quick­ly pulls in $85M for 'an im­por­tant growth phase'

Months after reeling in a $78 million Series B round, Silverback Therapeutics has hooked an even larger Series C.

The Seattle-based company announced Wednesday that it netted $85 million from a slate of new and previous investors. The quick boost could be a sign that an IPO is on the way.

In an email, Silverback CEO Laura Shawver told me she was “not able to provide any additional comments about Silverback” beyond what was shared in the company’s news release. In the prepared statement, she said the company is at “an important growth phase.”

News brief­ing: HHS chas­tis­es Eli Lil­ly for end­ing some dis­counts in a pan­dem­ic; Gilead to pay $97 mil­lion to set­tle kick­back al­le­ga­tions

Gilead agreed to pay $97 million to settle allegations that it illegally used a charity to pay thousands of Medicare patients’ co-payments for its pulmonary arterial hypertension drug Letairis.

The US government claimed that between June 2007 and December 2010, Gilead $GILD used the Caring Voice Coalition as a conduit to cover the costs of just its own drug. The alleged behavior goes against the False Claims Act.