Three years, a clinical hold and a death later, Sarepta abandons partnered pivotal-stage gene therapy

January 13, 2022 07:00 AM EST

Three years, a clinical hold and a death later, Sarepta abandons partnered pivotal-stage gene therapy

Amber Tong

Senior Editor

Sarepta is breaking off a three-year licensing pact for a rare disease gene therapy candidate, leaving its former partner on its own as it prepares for a pivotal readout in mid-year.

According to Lysogene, Sarepta returned the global commercial rights for LYS-SAF302 after “unsuccessful discussions for transferring back to Lysogene the responsibility for the global commercial supply of LYS-SAF302.”

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Graphic: Alexander Lefterov for Endpoints News

January 25, 2022 01:19 PM EST

In Focus

Marketing

Small biotechs with big drug ambitions threaten to upend the traditional drug launch playbook

Amber Tong

Senior Editor

Of the countless decisions Vlad Coric had to make as Biohaven’s CEO over the past seven years, there was one that felt particularly nerve-wracking: Instead of selling to a Big Pharma, the company decided it would commercialize its migraine drug itself.

“I remember some investors yelling and pounding on the table like, you can’t do this. What are you thinking? You’re going to get crushed by AbbVie,” he recalled.

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January 25, 2022 12:49 PM EST

Pharma

Marketing

MarketingRx roundup: Pfizer debuts Prevnar 20 TV ads; Lilly gets first FDA 2022 promo slap down letter

Beth Snyder Bulik

Senior Editor

Pfizer debuted its first TV ad for its Prevnar 20 next-generation pneumococcal pneumonia vaccine. In the 60-second spot, several people (actor portrayals) with their ages listed as 65 or older are shown walking into a clinic as they turn to say they’re getting vaccinated with Prevnar 20 because they’re at risk.

The update to Pfizer’s blockbuster Prevnar 13 vaccine was approved in June, and as its name suggests is a vaccine for 20 serotypes — the original 13 plus seven more that cause pneumococcal disease. Pfizer used to spend heavily on TV ads to promote Prevnar 13 in 2018 and 2019 but cut back its TV budgets in the past two fall and winter seasonal spending cycles. Prevnar had been Pfizer’s top-selling drug, notching sales of just under $6 billion in 2020, and was the world’s top-selling vaccine before the Covid-19 vaccines came to market last year.

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Albert Bourla (Photo by Steven Ferdman/Getty Images)

January 24, 2022 06:54 AM ESTUpdated 11:11 AM

R&D

UPDATED: Pfizer fields a CRL for a $295M rare disease play, giving rival a big head start

John Carroll

Editor & Founder

Pfizer won’t be adding a new rare disease drug to the franchise club — for now, anyway.

The pharma giant put out word that their FDA application for the growth hormone therapy somatrogon got the regulatory heave-ho, though they didn’t even hint at a reason for the CRL. Following standard operating procedure, Pfizer said in a terse missive that they would be working with regulators on a followup.

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ENDPOINTS CAREERS

Associate Director, Program Management - Cell & Gene Therapy Manufacturing

Lonza

Portsmouth, NH, USA

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Alexander Lefterov/Endpoints News

January 25, 2022 07:10 AM ESTUpdated 01:20 PM

R&D

In Focus

A new cancer immunotherapy brings cautious hope for a field long awaiting the next big breakthrough

Jason Mast

Editor

Bob Seibert sat silent across from his daughter at their favorite Spanish restaurant near his home in Charleston County, SC, their paella growing cold as he read through all the places in his body doctors found tumors.

He had texted his wife, a pediatric intensive care nurse, when he got the alert that his online chart was ready. Although he saw immediately it was bad, many of the terms — peritoneal, right iliac — were inscrutable. But she was five hours downstate, at a loud group dinner the night before another daughter’s cheer competition.

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Florida Gov. Ron DeSantis (AP Photo/Wilfredo Lee, File)

January 25, 2022 01:48 PM EST

R&D

Opinion: Florida is so mAb crazy, Ron DeSantis wants to use mAbs that don't work

Zachary Brennan

Senior Editor

Florida Gov. Ron DeSantis is trying so hard to politicize the FDA and demonize the federal government that he entered into an alternate universe on Monday evening in describing a recent FDA action to restrict the use of two monoclonal antibody, or mAb, treatments for Covid-19 that don’t work against Omicron.

Without further ado, let’s break down his statement from last night, line by line, adjective by adjective.

January 25, 2022 11:13 AM ESTUpdated 11:40 AM

Cell/Gene Tx

FDA slams door to pivotal trial for bubble boy disease gene therapy as Mustang Bio runs into another hold

Josh Sullivan

Associate Editor

Mustang Bio is in familiar territory, but that isn’t a place it necessarily wants to be.

The FDA has placed a hold on Mustang Bio’s pivitol trial for its gene therapy to treat patients with bubble boy disease, citing issues surrounding chemistry, manufacturing and controls clearance. It’s the second hold due to CMC issues the company has received in roughly 18 months.

An investigational new drug application was submitted in December 2021. If granted an IND, a Phase II study will then assess safety, tolerability and efficacy of MB-207. If approved by the FDA, the therapy would one day be eligible for a rare pediatric disease voucher.

January 25, 2022 12:56 PM EST

Pharma

Not cheaper by the dozen: Bristol Myers becomes the 12th pharma company to restrict 340B sales

Zachary Brennan

Senior Editor

Bristol Myers Squibb recently joined 11 of its peer pharma companies in limiting how many contract pharmacies can access certain drugs discounted by a federal program known as 340B.

Bristol Myers is just the latest in a series of high-profile pharma companies moving in their own direction as the Biden administration’s Health Resources and Services Administration struggles to rein in the drug discount program for the neediest Americans.

ENDPOINTS CAREERS

Director, Business Development, Cell & Gene Therapies

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Joaquin Duato, J&J CEO (Photo by Charles Sykes/Invision/AP)

January 25, 2022 12:50 PM EST

Pharma

New J&J CEO Joaquin Duato promises an aggressive M&A hunt in quest to grow pharma sales

John Carroll

Editor & Founder

Joaquin Duato stepped away from the sideline and directly into the spotlight on Tuesday, delivering his first quarterly review for J&J as its newly-tapped CEO after an 11-year run in senior posts. And he had some mixed financial news to deliver today while laying claim to a string of blockbuster drugs in the making and outlining an appetite for small and medium-sized M&A deals.

Duato also didn’t exactly shun large buyouts when asked about the future of the company’s medtech business — where they look to be in either the top or number 2 position in every segment they’re in — even though the bar for getting those deals done is so much higher.

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Roy Baynes, Merck

January 24, 2022 07:19 AM ESTUpdated 08:15 AM

R&D

FDA bats back Merck’s ‘pipeline in a product,’ demands more efficacy data

John Carroll

Editor & Founder

Despite some heavy blowback from analysts, Merck execs maintained an upbeat attitude about the market potential of its chronic cough drug gefapixant. But the confidence may be fading somewhat today as Merck puts out news that the FDA is handing back its application with a CRL.

Dubbed by Merck’s development chief Roy Baynes as a “pipeline in a product” with a variety of potential uses, Merck had fielded positive late-stage data demonstrating the drug’s ability to combat chronic cough. The drug dramatically reduced chronic cough in Phase III, but so did placebo, leaving Merck’s research team with a marginal success on the p-value side of the equation.

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