Sang­amo team rush­es to de­fend first hu­man gene edit­ing da­ta as MPS II ther­a­py of­fers a hit — and a miss

Sang­amo $SG­MO rolled out its first glimpse at the clin­i­cal ef­fi­ca­cy of their gene edit­ing ther­a­py for Hunter syn­drome (MPS II). And while re­searchers were able to point to some clear ev­i­dence that their ap­proach had an im­pact on a key bio­mark­er for the dis­ease for 2 pa­tients, there was a crit­i­cal lack of proof that it was do­ing specif­i­cal­ly what it was de­signed for.

Un­der­stand­ably, Sang­amo con­cen­trat­ed on uri­nary gly­cosamino­gly­cans (GAGs), high­light­ing big drops for both pa­tients in the sec­ond co­hort of the tiny ex­plorato­ry study. 

Ed Con­ner

The gene edit­ing ap­proach they’ve de­vel­oped with zinc fin­ger tech is in­tend­ed to cut in­to the er­rant gene and “in­sert a new copy of the IDS gene in­to a pre­cise lo­ca­tion in the DNA of liv­er cells to en­able a pa­tient’s liv­er to pro­duce a con­tin­u­ous and sta­ble sup­ply of the miss­ing hu­man IDS en­zyme.”

The iduronate-2-sul­fa­tase (IDS) en­zyme is need­ed to break down or re­cy­cle gly­cosamino­gly­cans (GAGs) der­matan sul­fate and he­paran sul­fate. 

The en­zyme gene ther­a­py, if it works, would end the need for reg­u­lar en­zyme re­place­ment in­fu­sions. And the two pa­tients in co­hort two demon­strat­ed mean re­duc­tions in GAG, der­matan sul­fate and he­paran sul­fate of 51%, 32%, and 61%.

That’s good.

How­ev­er, there was al­so this:

At base­line and for the first 16 weeks post-dos­ing of SB-913, plas­ma IDS ac­tiv­i­ty (mea­sure­ments ob­tained at trough of week­ly ERT dos­ing) was be­low the lev­el of quan­tifi­ca­tion of the cur­rent as­say.

That’s what Twit­ter be­gan buzzing about, trig­ger­ing a rapid drop in Sang­amo’s shares.  

Sandy Macrae

In a call with an­a­lysts, Sang­amo ex­ecs rushed to fight back against the back­lash.

That lack of ev­i­dence that the ther­a­py cre­ates the en­zyme need­ed, “doesn’t mean IDS isn’t be­ing pro­duced,” said Sang­amo CMO Ed Con­ner. “Cells of pa­tients with MSP II are starv­ing for IDS.” And “low lev­els may be suf­fi­cient to sup­press GAG.”

Sang­amo CEO Sandy Macrae al­so fierce­ly de­fend­ed the re­sults, say­ing you can’t get that hit on ef­fi­ca­cy un­less the gene edit­ing was tak­ing ef­fect.

In ad­di­tion, they added that the third co­hort in the study is get­ting a much stronger dose that could have a big­ger ef­fect. And ex­ecs added that they need to de­vel­op a bet­ter as­say for their clin­i­cal work to de­tect IDS. 

That wasn’t enough to over­come the first re­ac­tion by in­vestors. Their stock was down 22% in mid-morn­ing trad­ing.

This tri­al is cru­cial for Sang­amo, which is look­ing for clear ev­i­dence that it can beat its ri­vals in the fast-grow­ing field of gene edit­ing. That ri­val­ry, which in­cludes a va­ri­ety of ap­proach­es to edit­ing genes, is just get­ting start­ed.

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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