Tonix jumps on re­jigged phase III for PTSD drug; MeiraGTx scores an­oth­er NIH deal; Op­Gen, Qi­a­gen join hands to de­tect su­per­bugs

→ Af­ter a Phase III tri­al test­ing its PTSD drug, Ton­mya, in­volv­ing pa­tients that suf­fered trau­ma dur­ing mil­i­tary ser­vice since 2001, was pre­ma­ture­ly halt­ed fol­low­ing a fu­til­i­ty analy­sis, Tonix Phar­ma $TXNP went to the FDA armed with a sub­group analy­sis that showed pa­tients with fresh­er trau­ma had ben­e­fit­ted from the drug. On Wednes­day, the com­pa­ny said it had con­vinced the US health reg­u­la­tor to give it an­oth­er shot with a new late-stage study in this cat­e­go­ry of pa­tients, as well as those that have ex­pe­ri­enced civil­ian trau­ma. Tonix has al­so changed the tri­al’s goal­posts, say­ing it will de­ter­mine whether the pri­ma­ry end­point has been met at week 4, ver­sus week 12. Shares of tiny Tonix, whose break­through ther­a­py Ton­mya has en­dured mul­ti­ple tri­al set­backs, jumped about 22% on the an­nounce­ment. CEO Seth Le­d­er­man re­mains bull­ish about drug’s prospects, but told End­points News that he will need to raise more cap­i­tal to take the drug to the fin­ish line.

→ Gene ther­a­py de­vel­op­er MeiraGTx $MGTX has scored an ex­clu­sive li­cens­ing agree­ment with the NIH, in which the com­pa­ny gets the rights to an ex­per­i­men­tal drug for Sjö­gren’s syn­drome — a sys­temic au­toim­mune dis­or­der that im­pairs one’s abil­i­ty to se­crete flu­ids such as sali­va and tears that af­fects an es­ti­mat­ed 4 mil­lion Amer­i­cans. The two en­ti­ties have al­ready part­nered on two oth­er R&D pro­grams.

→ Ge­nom­ic analy­sis com­pa­ny Op­Gen $OPGN is col­lab­o­rat­ing with mol­e­c­u­lar di­ag­nos­tics com­pa­ny Qi­a­gen $QGEN to de­vel­op di­ag­nos­tics to de­tect mul­tidrug re­sis­tant bac­te­r­i­al pathogens. Op­Gen’s stock rose about 7% on the an­nounce­ment, in which fi­nan­cial terms of the deal were un­spec­i­fied.

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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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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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.
SpringWorks

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.

Jim Mellon [via YouTube]

Health­i­er, longer lifes­pans will be a re­al­i­ty soon­er than you think, Ju­ve­nes­cence promis­es as it clos­es $100M round

Earlier this year, an executive from Juvenescence-backed AgeX predicted the field of longevity will eventually “dwarf the dotcom boom.” Greg Bailey, the UK-based anti-aging biotech’s CEO, certainly hopes so.

On Monday, Juvenescence completed its $100 million Series B round of financing. The company is backed by British billionaire Jim Mellon — who wrote his 400-page guide to investing in the field of longevity shortly after launching the company in 2017. Bailey, who served as a board director for seven years at Medivation before Pfizer swallowed the biotech for $14 billion, is joined by Declan Doogan, an industry veteran with stints at Pfizer $PFE and Amarin $AMRN.

AveX­is sci­en­tif­ic founder was axed — and No­var­tis names a new CSO in wake of an ethics scan­dal

Now at the center of a storm of controversy over its decision to keep its knowledge of manipulated data hidden from regulators during an FDA review, Novartis CEO Vas Narasimhan has found a longtime veteran in the ranks to head the scientific work underway at AveXis, where the incident occurred. And the scientific founder has hit the exit.

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Ab­b­Vie gets its FDA OK for JAK in­hibitor upadac­i­tinib, but don’t look for this one to hit ex­ecs’ lofty ex­pec­ta­tions

Another big drug approval came through on Friday afternoon as the FDA OK’d AbbVie’s upadacitinib — an oral JAK1 inhibitor that is hitting the rheumatoid arthritis market with a black box warning of serious malignancies, infections and thrombosis reflecting fears associated with the class.

It will be sold as Rinvoq — at a wholesale price of $59,000 a year — and will likely soon face competition from a drug that AbbVie once controlled, and spurned. Reuters reports that a 4-week supply of Humira, by comparison, is $5,174, adding up to about $67,000 a year.