Ex­perts of­fer a sol­id en­dorse­ment for Acel­Rx pain med, spurring a big ral­ly for their stock

The FDA forced Acel­Rx Phar­ma­ceu­ti­cals to go back to the draw­ing board to re­fash­ion a bet­ter safe­ty plan for its new pain ther­a­py Dsu­via (ARX-04). And that paid off Fri­day with a big en­dorse­ment from the out­side ex­perts the agency drew to­geth­er to re­view their mar­ket­ing ap­pli­ca­tion. 

Vince An­got­ti

The group vot­ed a lop­sided 10-3 in fa­vor of a green light, as these ex­perts were able to look past reg­u­la­to­ry con­cerns about the po­ten­tial for abuse and ac­ci­den­tal ex­po­sure posed by the 30 mcg sufen­tanil tablets.

That helped dri­ve a big ral­ly for their shares $ACRX, which are up 29% in pre-mar­ket trad­ing Mon­day.

To get an ap­proval through a vig­i­lant FDA, the biotech has re­peat­ed­ly em­pha­sized that the un­der-the-tongue med will not be avail­able in the com­mu­ni­ty, but on­ly used in the hos­pi­tal by trained per­son­nel. But sev­er­al of the ex­perts re­port­ed­ly shared the FDA’s con­cerns that the small pills could wind up on the floor, with­in reach of chil­dren.

“The FDA sug­gest­ed we mod­i­fy the di­rec­tions for use to bet­ter clar­i­fy the sub­lin­gual space, and they al­so want­ed more con­fir­ma­tion that the health care provider was the one ad­min­is­ter­ing the drug,” Acel­Rx CEO Vince An­got­ti told Healio. “We went back and at­tached in­struc­tions to each and every Dsu­via pack­et and had health care providers ad­min­is­ter the drug to mock pa­tients and achieved close to 100% ac­cu­ra­cy.”

We’ll soon see if the com­pa­ny can pass the fi­nal re­view by the FDA.

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,600+ biopharma pros reading Endpoints daily — and it's free.

As­traZeneca's di­a­betes drug Farx­i­ga helps pa­tients with heart dis­ease and with­out di­a­betes in land­mark tri­al

Months ago, data on J&J’s $JNJ Invokana indicated the diabetes drug conferred cardiovascular (CV) benefit in patients who do and do not have preexisting CV disease. On Tuesday, AstraZeneca’s $AZN rival treatment, Farxiga, was shown to cut the risk of CV death or the worsening of heart failure in patients with heart disease, in a landmark trial.

The treatments, in addition to Jardiance from Eli Lilly $LLY, belong to a class of diabetes drugs called sodium-glucose co-transporter 2 (SGLT2) inhibitors, which work by curbing the absorption of glucose via the kidneys so that surplus glucose is excreted through urination.

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,600+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: 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:

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,600+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,600+ biopharma pros reading Endpoints daily — and it's free.

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
Endpoints News

Basic subscription required

Unlock this story instantly and join 57,600+ biopharma pros reading Endpoints daily — and it's free.

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.
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.