Applied's trial conduct under fire from FDA

Today’s Big News

Dec 4, 2024

GSK taps Rgenta to develop new RNA-targeting treatments, offering up to $500M per target 


Novo Holdings-backed Antag raises $84M to send complementary obesity candidate to the clinic


GSK axes hepatitis B vaccine trial after missing efficacy goal 


Applied Therapeutics' trial conduct scrutinized in FDA warning letter


GSK goes East again, snagging option on preclinical DualityBio ADC for $30M


Maze Therapeutics looks to steer lead kidney disease assets through the clinic with $115M series D 


Relay passes baton to Elevar, handing off NDA-ready rare cancer asset for up to $500M 


RNA-targeting molecule from Arrakis allows muscles to relax in mice with myotonic dystrophy 

The Top Line Podcast: Don’t miss out on the newest episode. Listen now.

 

Featured

GSK taps Rgenta to develop new RNA-targeting treatments, offering up to $500M per target

Pharma giant GSK is buying into Rgenta Therapeutics’ mission to develop a pipeline of oral RNA-targeting small molecules. The new partners aim to develop new splice modulators for multiple disease areas, including oncology, with Rgenta able to receive up to nearly $500 million biobucks per target.
 

Top Stories

Novo Holdings-backed Antag raises $84M series A to send complementary obesity candidate to the clinic

It will take a unique offering to make a significant dent in the multibillion-dollar obesity market. But Antag has some star power on its side, with GLP-1 co-discoverer Jens Holst, M.D., one of the biotech's co-founders.

GSK axes hepatitis B vaccine trial after missing efficacy goal

GSK has terminated a phase 1/2 hepatitis B trial after the therapeutic vaccine regimen failed to meet the efficacy endpoint, denting the company’s hopes of creating a functional cure for the infectious disease.

Applied Therapeutics' trial conduct scrutinized in FDA warning letter

In a warning letter published Tuesday, the FDA scolded Applied Therapeutics on two counts related to its 47-patient study of govorestat in kids with classic galactosemia, specifically raising issues related to data deletion and a dosing error. The write-up was issued around the same time that the agency rejected govorestat's approval application.

GSK goes East again, snagging option on preclinical DualityBio ADC for $30M

GSK has made another trip to China to build out its antibody-drug conjugate pipeline, returning with an option on a preclinical Duality Biologics candidate that could ultimately cost the Big Pharma $1 billion.

Maze Therapeutics looks to steer lead kidney disease assets through the clinic with $115M series D

Human genetics biotech Maze Therapeutics has navigated the biotech world’s tricky fundraising landscape to land a $115 million series D round.

Relay passes baton to Elevar, handing off NDA-ready rare cancer asset for up to $500M

After pausing work on a rare cancer asset, Relay Therapeutics is passing the baton to Elevar Therapeutics. The two have signed a deal worth up to $500 million biobucks that gives Elevar licensing rights to an investigational oral small molecule designed to treat solid tumors.

RNA-targeting molecule from Arrakis allows muscles to relax in mice with myotonic dystrophy

Dune-inspired Arrakis Therapeutics has tested its spice—an RNA-targeting small molecule—in a mouse model of myotonic dystrophy type 1 (DM1), with the drug reversing the prolonged muscle contraction that is characteristic of the disease.

UnitedHealthcare CEO Brian Thompson fatally shot in Manhattan: reports

UnitedHealth Group abruptly ended its investor day event on Wednesday morning as news reports surfaced that the CEO of its health plan division had been fatally shot.

Eli Lilly's Zepbound defeats Novo Nordisk's Wegovy in head-to-head weight loss trial

Eli Lilly revealed results from a study that showed its obesity drug Zepbound was 47% more effective than Novo’s Wegovy in helping patients lose weight. In the SURMOUNT-5 study, while participants taking Zepbound lost an average of 20.2% of their weight after 72 weeks, those on Wegovy lost 13.7% of their weight.

Health tech company myTomorrows cuts ribbon at India operations hub, looks to expand clinical trial access in Asia

Amsterdam-based health tech company myTomorrows is eyeing expansion into the Asia-Pacific region, opening an operations hub in India that will help match patients with clinical trials.

Philips, Mayo Clinic team up on AI for cardiac MRI scans

The project will look to combine separate AI technologies currently under development by both the Mayo Clinic and Philips, which the company said could one day aid radiographers in completing difficult heart scans with less training.
 
Fierce podcasts

Don’t miss an episode

Meet 2024’s fiercest women in life sciences

This week on "The Top Line," Fierce editors discuss our special report celebrating 10 women driving change in clinical research, business development, venture capital, and more
 

Resources

Whitepaper

Building a “Best Practice” R&D Organization in Biopharma

We interviewed 14 R&D biopharma leaders on the critical success factors for building a Best Practice R&D organization. This report shares what we learned.
Whitepaper

Single Cell RNA Profiling FFPE Tissue

Explore the robust cross-platform agreement between scRNA-seq and bulk RNA-seq, ensuring consistent and reproducible data on immune cell presence.
Whitepaper

Straight to Market in an Autoinjector

The use of prefilled syringes to administer biotherapeutics is beginning to gain traction because they offer a range of stability, efficacy, and patient safety benefits. Download this white paper to learn how Lonza has developed a novel workflow to ensure confidence regarding functionality in PFS and autoinjectors.
Whitepaper

7 “Make or Break” Factors for Emerging Biopharma Companies

This e-book explores 7 factors that are critical to the success of emerging biopharma companies, whether they plan to out-license, partner, or launch & commercialize products. It’s an essential resource for emerging companies with products in all stages of development.
Whitepaper

Planning for success in cell-based manufacturing

Learn how to evaluate quality documentation to help you select the right raw materials at early decision points for your cell-based manufacturing processes.
 

Industry Events

On Helix

Date: ‌4 ‌July ‌2024 ‌- ‌Location: ‌Babraham ‌Research ‌Campus, ‌Cambridge ‌UK

 

Upcoming Fierce Events

4
Dec
Free Virtual Event
5
Dec
The Lighthouse at Pier 61 in New York City
14-15
Jan
San Francisco, CA
22-24
Jan
Virtual Event
29-1
Apr-May
San Diego, CA

View all events