On the cusp of PhI, An­gel­man-fo­cused biotech scores po­ten­tial buy­out deal from Ul­tragenyx

Ul­tragenyx has found a new rare dis­ease to de­vote it­self to, rop­ing in a young up­start as it be­gins a long trek to a new treat­ment for An­gel­man syn­drome.

Their new part­ners at GeneTx is work­ing to­ward an IND for GTX-102, an an­ti­sense oligonu­cleotide de­signed to in­hib­it the UBE3A-AS DNA strand and there­by turn on an al­lele in neu­rons that pro­duces a key pro­tein miss­ing in pa­tients with An­gel­man.

Emil Kakkis

The col­lab­o­ra­tion — and po­ten­tial ac­qui­si­tion — would pit Ul­tragenyx against Je­re­my Levin’s team at Ovid, which has show­cased some con­tro­ver­sial Phase II da­ta last year for its ex­trasy­nap­tic GABAA re­cep­tor ag­o­nist. An­gel­man is al­so among the slate of neu­ro­log­i­cal tar­gets that Bio­gen and Io­n­is are tack­ling in an an­ti­sense deal.

Once the FDA ac­cepts the drug for re­view, Ul­tragenyx will have 30 days to ex­er­cise an ex­clu­sive op­tion to buy the com­pa­ny out — an op­tion worth $20 mil­lion in the agree­ment. The duo has pen­ciled in a po­ten­tial ex­ten­sion, which would call for an­oth­er $25 mil­lion pay­ment.

“An­gel­man syn­drome is a dev­as­tat­ing neu­ro­log­i­cal con­di­tion with no treat­ment op­tions that rep­re­sents a very sig­nif­i­cant un­met med­ical need,” said Ul­tragenyx CEO Emil Kakkis. “GTX-102 is a nov­el and promis­ing po­ten­tial ther­a­py spe­cif­ic to the dis­ease mech­a­nism in these pa­tients, and we are pleased that GeneTx chose to part­ner with us based on our track-record of rapid, in­no­v­a­tive de­vel­op­ment in rare dis­eases.”

In ad­di­tion, GeneTx has a unique con­nec­tion to the pa­tient com­mu­ni­ty that made it ap­peal­ing: The Foun­da­tion for An­gel­man Syn­drome Ther­a­peu­tics, the non­prof­it group that launched GeneTx last Feb­ru­ary, is the largest pa­tient or­ga­ni­za­tion for the rare neu­rode­vel­op­men­tal dis­or­der, Ul­tragenyx CBO Thomas Kass­berg told Bio­Cen­tu­ry.

Paula Evans

FAST is al­so the ma­jor­i­ty own­er of GeneTx while Texas A&M Uni­ver­si­ty Sys­tem owns a mi­nor­i­ty share. Paula Evans, FAST chair­per­son, GeneTx CEO and an An­gel­man mom, said it is fit­ting to shift from do­na­tions to a pact with an es­tab­lished in­dus­try play­er.

The two sides will col­lab­o­rate on both the IND and man­age­ment of the Phase I/II study. For now, GeneTx con­tin­ues to fund all de­vel­op­ment ac­tiv­i­ties and take charge of reg­u­la­to­ry and sci­en­tif­ic ac­tiv­i­ties, while Ul­tragenyx of­fers strate­gic and clin­i­cal guid­ance.

Tile im­age: The Evans fam­i­ly, FAST

Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Novartis CEO Vas Narasimhan [via Bloomberg/Getty]

I’m not per­fect: No­var­tis chief Vas Narasimhan al­most apol­o­gizes in the wake of a new cri­sis

Vas Narasimhan has warily stepped up with what might pass as something close to a borderline apology for the latest scandal to engulf Novartis.

But he couldn’t quite get there.

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FDA to Sarep­ta: Your wide­ly an­tic­i­pat­ed fol­lowup to Ex­ondys 51 is not get­ting an ac­cel­er­at­ed OK for Duchenne MD

In one of the least anticipated moves of the year, the FDA has rejected Sarepta’s application for an accelerated approval of its Duchenne MD drug golodirsen after fretting over safety issues.

In a statement that arrived after the bell on Monday, Sarepta explained the CRL, saying:

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Levi Garraway. Broad Institute via Youtube

Roche raids Eli Lil­ly for its next chief med­ical of­fi­cer as San­dra Horn­ing plans to step down

We found out Monday morning where Levi Garraway was headed after he left Eli Lilly as head of oncology R&D a few days ago. Roche named Garraway as their new chief medical officer, replacing Sandra Horning, who they say is retiring from the company.

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Af­ter a posse of Wall Street an­a­lysts pre­dict a like­ly new win for Sarep­ta, we're down to the wire on a crit­i­cal FDA de­ci­sion

As Bloomberg notes, most of the Wall Street analysts that cover Sarepta $SRPT are an upbeat bunch, ready to cheer on the team when it comes to their Duchenne MD drugs, or offer explanations when an odd setback occurs — as happened recently with a safety signal that was ‘erroneously’ reported last week.

Ritu Baral Cowen
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UP­DAT­ED: No­var­tis spin­off Nabri­va fi­nal­ly scores its first an­tibi­ot­ic ap­proval

In May, Nabriva Therapeutics suffered a setback after the FDA rejected its antibiotic for complicated urinary tract infections — the Novartis spinoff has now had some better luck with the US agency, which on Monday approved its other drug for community-acquired bacterial pneumonia.

The drug, lefamulin, has been developed as an intravenous and oral formulation and been tested in two late-stage clinical trials. The semi-synthetic compound, whose dosing can be switched between the two formulations, is engineered to inhibit the synthesis of bacterial protein by binding to a part of the bacterial ribosome.

Saqib Islam. CheckRare via YouTube

Spring­Works seeks $115M to push Pfiz­er drugs across fin­ish line while Sat­suma sells mi­graine play in $86M IPO

SpringWorks and Satsuma — both biotech spinouts that have closed B rounds in April — are loading up with IPO cash to boost their respective late-stage plans.

Bain-backed SpringWorks is the better-known company of the two, and it’s gunning for a larger windfall of $115 million to add to $228 million from previous financings. In the process, the Stamford, CT-based team is also drawing the curtains on the partnerships it has in mind for the pair of assets it had initially licensed from Pfizer.

Mi­nor­i­ty racial groups con­tin­ue to be dis­mal­ly rep­re­sent­ed in can­cer tri­als — study

Data reveal that different racial and ethnic groups — by nature and/or nurture — can respond differently in terms of pharmacokinetics, efficacy, or safety to therapeutics, but this disparity is not necessarily accounted for in clinical trials. A fresh analysis of the last decade of US cancer drug approvals suggests the trend continues, cementing previous research that suggests oncology trials are woefully under-representative of the racial makeup of the real world.

Van­da shares slide af­ter FDA spurns their big end­point and re­jects a pitch on jet lag re­lief

Back in the spring of last year, Vanda Pharmaceuticals $VNDA served up a hot stew of mixed data for a slate of endpoints related to what they called clear evidence that their melatonin sleep drug Hetlioz (tasimelteon) could help millions of travelers suffering from jet lag.

Never mind that they couldn’t get a planned 90 people in the study, settling for 25 instead; Vanda CEO Mihael H. Polymeropoulos said they were building on a body of data to prove it would help jet-lagged patients looking for added sleep benefits. And that, they added, would be worth a major upgrade from the agency as they sought to tackle a big market.