Little Deciphera will hustle positive PhIII survival data on lead cancer drug to the FDA in search of its first OK

August 13, 2019 07:02 AM EDTUpdated 10:05 AM

John Carroll

Little Deciphera will hustle positive PhIII survival data on lead cancer drug to the FDA in search of its first OK — shares soar

Deciphera has spent the last 5 years positioning itself as another commercial player-to-be in the hectic oncology arena, steering its way through a $127 million IPO $DCPH in 2017, hiring Pfizer vet Mike Taylor as CEO — and then replacing him with someone with marketing experience last spring.

Basic subscription required

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

Brian Kaspar. AveXis via Twitter

August 19, 2019 06:07 PM EDTUpdated 5 hours ago

John Carroll

People

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.

Basic subscription required

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

Novartis CEO Vas Narasimhan [via Bloomberg/Getty]

August 19, 2019 06:33 AM EDTUpdated 07:12 AM

John Carroll

Pharma

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.

Basic subscription required

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

ENDPOINTS CAREERS

Structural Biologist/Biomolecular NMR Investigator

Skyhawk Therapeutics

Waltham, MA

view job offer post your job now

August 19, 2019 06:15 PM EDT

John Carroll

R&D

Regulatory

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:

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

August 19, 2019 11:45 AM EDT

John Carroll

People

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.

Basic subscription required

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

August 19, 2019 09:36 AM EDTUpdated 09:57 AM

John Carroll

R&D

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

Basic subscription required

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

ENDPOINTS CAREERS

Bioinformatics Software Developer

Skyhawk Therapeutics

Waltham, MA, USA

view job offer post your job now

August 19, 2019 03:57 PM EDTUpdated 06:31 PM

Natalie Grover

Pharma

R&D

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

August 19, 2019 09:24 AM EDT

Amber Tong

IPOs

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.

August 19, 2019 08:56 AM EDT

Natalie Grover

R&D

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.

ENDPOINTS CAREERS

Recruiting Manager/Sr.Manager/Associate Director

Skyhawk Therapeutics

Waltham, MA, USA

view job offer post your job now

August 19, 2019 07:55 AM EDTUpdated 09:49 AM

John Carroll

R&D

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.