Months af­ter IPO, No­var­tis spin­out resTOR­bio wins on PhI­Ib da­ta — stock soars

Just six months af­ter its de­but on the Nas­daq, resTOR­bio is trot­ting out late-stage da­ta this morn­ing for its lead drug can­di­date — a TORC1 in­hibitor they’re hop­ing will pre­vent res­pi­ra­to­ry in­fec­tions in el­der­ly folks. While it flopped in a com­bo tri­al, the com­pa­ny’s drug met its pri­ma­ry end­point when test­ed by it­self.

In­vestors are thrilled with the news, with resTOR­bio’s share price $TORC fly­ing up 161% as of press time. Yes­ter­day’s close was at $9.01 per share, and to­day its so far climbed to $23.50. If it holds, that’s a de­cent jump from its IPO price of $14 to $16 per share.

Joan Man­nick

The drug — spun out of No­var­tis in­to its own start­up just last year — is now called RTB101, al­though it once went un­der the code name BEZ235. In a Phase IIb tri­al, the 10 mg dose of the drug showed a sta­tis­ti­cal­ly sig­nif­i­cant re­duc­tion in res­pi­ra­to­ry in­fec­tions when tak­en by pa­tients once a day. That co­hort saw a 30.6% de­crease in the per­cent­age of pa­tients who de­vel­oped a res­pi­ra­to­ry in­fec­tion when com­pared to the place­bo co­hort: p=0.026.

In­ter­est­ing­ly, the drug flopped big time when test­ed in com­bi­na­tion with an al­ready-ap­proved im­muno­sup­pres­sant called everolimus, an mTOR in­hibitor of­ten used to pre­vent the re­jec­tion of or­gan trans­plants. When resTOR­bio test­ed RTB101 com­bi­na­tion with everolimus, they found no de­crease in the per­cent­age of pa­tients with res­pi­ra­to­ry in­fec­tions. The drug al­so saw lousy re­sults when the 10 mg dose was tak­en twice dai­ly in­stead of once.

“We’re find­ing that less TORC1 in­hi­bi­tion works bet­ter than more TORC1 in­hi­bi­tion,” the com­pa­ny’s co-founder and CMO Joan Man­nick tells me. They saw a sim­i­lar re­sponse in their Phase IIa tri­al, she said.

Chen Schor

The com­pa­ny’s CEO Chen Schor said he wasn’t too con­cerned that the com­bo and dou­ble dos­es didn’t per­form well. In fact, the drug work­ing as a monother­a­py is ben­e­fi­cial.

“We pre­fer to move for­ward with the monother­a­py, be­cause it’s eas­i­er to man­u­fac­ture,” he said. “That was our hope.”

The com­pa­ny al­so homed in on a few spe­cif­ic pa­tient groups in which they saw more promis­ing re­sults. For ex­am­ple, in asth­ma pa­tients, they saw a 68% re­duc­tion in res­pi­ra­to­ry in­fec­tions (p=0.0002), and in pa­tients 85 years or old­er they saw 67% (p=0.007). The tri­al in­volved some 650 pa­tients, ac­cord­ing to clin­i­cal­tri­als.gov, but when resTOR­bio whit­tles down the pa­tient group to these se­lect pop­u­la­tions, we’re talk­ing much few­er peo­ple. I asked Man­nick just how many we’re talk­ing here. In asth­ma, it was on­ly 47 pa­tients, she said, and a mere 27 peo­ple were 85 years old and old­er.

But Man­nick de­fend­ed the da­ta’s sig­nif­i­cance re­gard­less of the small­er pa­tient group. “When you have few­er pa­tients, it’s much hard­er to reach sta­tis­ti­cal sig­nif­i­cance,” she said. “You have to have a big ef­fect.”

The com­pa­ny is meet­ing with the FDA at the end of this year to dis­cuss how it should move for­ward with a Phase III tri­al. Man­nick and Schor said the com­pa­ny will be shoot­ing for a sim­i­lar tri­al de­sign, in which they test co­horts of nar­row pa­tient groups with­in the same tri­al.

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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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.”

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 Redford, 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.

CEO Markus Warmuth (Monte Rosa)

Monte Rosa rakes in $96M Se­ries B as it pre­pares 'mol­e­c­u­lar glue' plat­form for IND-en­abling stud­ies

About four months after completing an extension to its Series A, Monte Rosa Therapeutics is putting its next foot forward with another heap of cash.

The Boston-based biotech is back with $96 million in Series B financing with a goal to get its lead program ready for IND-enabling studies by the end of the year. Though Monte Rosa is keeping its specific target a secret for now, the company has been researching how to utilize its protein degradation technology in breast cancer and non-small cell lung cancer, among other areas.

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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