Payback? Analysts say Sarepta was blindsided by an FDA rejection driven by regulatory revenge

August 19, 2019 06:15 PM EDTUpdated August 20, 09:53 AM

John Carroll

Payback? Analysts say Sarepta was blindsided by an FDA rejection driven by regulatory revenge

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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Scott Gottlieb, AP Images

September 17, 2019 08:59 AM EDTUpdated 09:14 AM

John Carroll

People

Scott Gottlieb is once again joining a team that enjoyed good times at the FDA under his high-energy stint at the helm

Right after jumping on Michael Milken’s FasterCures board on Monday, the newly departed FDA commissioner is back today with news about another life sciences board post that gives him a ringside chair to cheer on a lead player in the real-world evidence movement — one with very close ties to the FDA.

Aetion is reporting this morning that Gottlieb is joining their board, a group that includes Mohamad Makhzoumi, a general partner at New Enterprise Associates, where Gottlieb returned after stepping out of his role at the FDA 2 years after he started.

Gottlieb — one of the best connected execs in biopharma — knows this company well. As head of FDA he championed the use of real-world evidence to help guide drug developers and the agency in gaining greater efficiencies, which helped set up Aetion as a high-profile player in the game.

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Deborah Dunsire. Lundbeck

September 16, 2019 03:50 AM EDTUpdated 10:19 AM

John Carroll

Deals

UPDATED: Deborah Dunsire is paying $2B for a chance to leap directly into a blockbuster showdown with a few of the world's biggest pharma giants

A year after taking the reins as CEO of Lundbeck, Deborah Dunsire is making a bold bid to beef up the Danish biotech’s portfolio of drugs in what will likely be a direct leap into an intense rivalry with a group of giants now carving up a growing market for new migraine drugs.

Bright and early European time Monday morning the company announced that it will pay up to about $2 billion to buy Alder, a little biotech that is far along the path in developing a quarterly IV formulation of a CGRP drug aimed at cutting back the number of crippling migraines patients experience each month. In a followup call, Dunsire also noted that the company will likely need 200 to 250 reps for this marketing task on both sides of the Atlantic. And analysts were quick to note that the dealmaking at Lundbeck isn’t done, with another $2 billion to $3 billion available for more deals to beef up the pipeline.

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September 17, 2019 09:03 AM EDTUpdated 09:17 AM

Jason Mast

China

R&D

San Diego couple charged with stealing trade secrets, opening Chinese biotech as DOJ crackdown continues

A San Diego couple has been charged with stealing trade secrets from a US hospital and opening a business based off those secrets in China as the controversial industry-wide crackdown on alleged corporate espionage continues. On the same day, the Department of Justice announced they had arrested Beijing representative Zhongsan Liu for allegedly trying to obtain research visas for government recruiters.

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September 17, 2019 07:31 AM EDTUpdated 10:22 AM

Amber Tong

R&D

UPDATED: Biogen pulls the plug on prized IPF drug from $562M+ Stromedix buyout

One of Biogen’s attempts to branch out has flopped as the biotech scraps a mid-stage program for idiopathic pulmonary fibrosis.

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September 17, 2019 06:59 AM EDTUpdated 08:08 AM

Natalie Grover

R&D

Warts for the win: Aclaris' lead drug clears pivotal study

Aclaris Therapeutics has found a way to get rid of the warts and all.

The company — which earlier this month decided to focus on its arsenal of kinase inhibitors — on Monday unveiled positive data from a pivotal study testing its lead experimental drug for use in common warts.

The drug, A-101, was tested in a 502-patient study called THWART-2 — patients enrolled had one to six warts before qualifying for the trial. Patients either self-administered A-101 topical solution or a vehicle twice a week over a two-month period. A higher proportion of patients on the drug (a potent hydrogen peroxide topical solution) saw their warts disappear at day 60, versus the vehicle (p<0.0001) — meeting the main goal of the study. Each secondary endpoint also emerged in favor of A-101, the company said.

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Tower Bridge in London [Shutterstock]

September 16, 2019 03:59 PM EDT

John Carroll

Bioregnum

#UKBIO19: Join GSK’s Hal Barron and a group of top biotech execs for our 2nd annual biotech summit in London

Over the past 10 years I’ve made a point of getting to know the Golden Triangle and the special role the UK biopharma industry plays there in drug development. The concentration of world class research institutes, some of the most accomplished scientists I’ve ever seen at work and a rising tide of global investment cash leaves an impression that there’s much, much more to come as biotech hubs are birthed and nurtured.

September 13, 2019 08:33 AM EDTUpdated 08:47 AM

John Carroll

R&D

It’s finally over: Biogen, Eisai scrap big Alzheimer’s PhIIIs after a predictable BACE catastrophe raises safety fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

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Charles Nichols, LSU School of Medicine

September 17, 2019 05:00 AM EDTUpdated 09:39 AM

Jason Mast

R&D

Could psychedelics tackle the obesity crisis? A longtime researcher in the field says his latest mouse study suggests potential

Psychedelics have experienced a renaissance in recent years amid a torrent of preclinical and clinical research suggesting it might provide a path to treat mood disorders conventional remedies have only scraped at. Now a preclinical trial from a young biotech suggests at least one psychedelic compound has effects beyond the mind, and — if you believe the still very, very early hype — could provide the first single remedy for some of the main complications of obesity.

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Senior Associate - AgTech - Science & Technology

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September 16, 2019 07:24 PM EDT

John Carroll

R&D

Acceleron drops a development program as #2 drug fails to spark functional benefits in patients with a rare neuromuscular ailment

Acceleron is scrapping a muscular dystrophy development program underway for its number 2 drug in the pipeline after pouring over some failed mid-stage secondary data.

Gone is the ACE-083 project in patients with facioscapulohumeral muscular dystrophy. Their drug hit the primary endpoint on building muscle but flopped on key secondaries for functional improvements in patients, which execs felt was vital to the drug’s success.