A faster treat­ment for tu­ber­cu­lo­sis? TB Al­liance launch­es piv­otal tri­al for four-drug reg­i­men

Com­bin­ing some of the lat­est break­throughs in tu­ber­cu­lo­sis re­search with some of the old­est treat­ment op­tions, the TB Al­liance has launched a piv­otal tri­al to test how well their four-drug reg­i­men can treat tu­ber­cu­lo­sis in light of grow­ing re­sis­tance to cur­rent ther­a­pies.

Mel Spigel­man

The not-for-prof­it is con­tribut­ing an ex­per­i­men­tal com­pound, pre­tomanid, which rep­re­sents a class of nov­el an­ti-bac­te­r­i­al agents called ni­troim­i­da­zole. The rest of the reg­i­men calls for J&J’s be­daquiline — per­haps bet­ter known as Sir­turo — the off-patent an­tibi­ot­ic mox­i­floxacin, and pyraz­i­namide, a gener­ic med of­ten found in TB com­bos.

Dubbed Sim­plic­iTB, the tri­al will be con­duct­ed among 450 pa­tients across Africa, Asia, Eu­rope and Latin Amer­i­ca, with the first pa­tient al­ready en­rolled in Tbil­isi, Geor­gia.

There are two goals. First, re­searchers want to find out whether the BPaMZ reg­i­men can in­deed treat drug-sen­si­tive TB more quick­ly and ef­fec­tive­ly — some­thing they have ob­served in a small­er, short­er Phase II tri­al — by com­par­ing four months of that to six months of the stan­dard iso­ni­azid, ri­fampicin, pyraz­i­namide and etham­b­u­tol (HRZE) com­bo.

The sec­ond as­sess­ment is on the new reg­i­men’s po­ten­tial in mul­ti-drug re­sis­tant tu­ber­cu­lo­sis — cur­rent treat­ments tend to be ex­pen­sive, lengthy and in­volve in­jectable drugs.

“As re­sis­tance to cur­rent TB treat­ments con­tin­ues to grow, we need to in­tro­duce all-oral drug reg­i­mens that can treat every per­son with TB in six months or less, re­gard­less of their re­sis­tance pro­file,” said Mel Spigel­man, pres­i­dent and CEO of TB Al­liance. “If proven suc­cess­ful in Sim­plic­iTB, the BPaMZ reg­i­men would rep­re­sent a ma­jor step to­ward this goal.”

Tu­ber­cu­lo­sis is among the fa­tal ail­ments that con­tin­ue to af­flict low-in­come coun­tries but re­ceive lit­tle R&D ac­tion. Non-prof­its have large­ly been pick­ing up the charge; the Gates Foun­da­tion has made it one of its pri­or­i­ties by back­ing in­fec­tious dis­ease start­up Vir and, re­cent­ly, set­ting up a biotech-like in­sti­tute to test a tu­ber­cu­lo­sis pre­ven­tion pro­gram it­self.

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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Pay­back? Sarep­ta stunned as FDA spurns fol­lowup to Ex­ondys 51 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.