Staff-devoid Acasti says it's still on track

Today's Big News

Jun 23, 2023

Roche drops antisense drug after seeing efficacy data, leaving Biogen, Ionis and Ultragenyx to duke it out


Apogee takes aim at Regeneron, Lilly as inflammation-focused biotech readies IPO


Weeks after laying off 'substantially all' employees, Acasti Pharma insists its approval plans remain on track


Alphabet's quantum spinoff starts working with AstraZeneca, Sanofi to accelerate R&D


Fierce Biotech Fundraising Tracker '23: Tagworks raises $65M series A; Flagship launches Empress with $50M


Chutes & Ladders—Bayer promotes Sebastian Guth to helm all US operations


'The Top Line': The most expensive drugs in the US, plus this week's headlines

The Top Line Podcast: don’t miss out on the newest episode where we discuss ASCO data, rising health tech stars, and more. Listen now.

 

Featured

Roche drops antisense drug after seeing efficacy data, leaving Biogen, Ionis and Ultragenyx to duke it out

Roche has bowed out of the race to develop an antisense oligonucleotide (ASO) for Angelman syndrome. After getting a look at early efficacy data, the Swiss drugmaker opted against advancing the candidate, switching its Angelman focus to another asset and leaving the ASO space to Biogen, Ionis and Ultragenyx.
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July 18-19, 2023 | Jersey City, NJ
 

Top Stories

Apogee takes aim at Regeneron, Lilly as inflammation-focused biotech readies IPO

Barely six months after launching, Paragon Therapeutics’ first spinout has set its sights on the public markets as well as taking on some of the biggest names in immunology and inflammation.

Weeks after laying off 'substantially all' employees, Acasti Pharma insists its approval plans remain on track

Laying off “substantially all” your workforce is normally a sign that a biotech is in a death spiral, but Acasti Pharma’s CEO is insistent that the move has “energized” the company as it gears up for a phase 3 study of its lead asset.

Puerto Rico on Gene & Cell Therapy: An Unparalleled Proposal for Non-Dilutive Funding

Puerto Rico is becoming the best place in the world to conduct R&D activities for emerging technologies. Could your organization maximize growth potential by capitalizing on Puerto Rico’s unique tax incentives & culture of innovation?

Alphabet's quantum spinoff starts working with AstraZeneca, Sanofi to accelerate R&D

The Alphabet spinoff SandboxAQ has signed up AstraZeneca and Sanofi as users of its drug discovery and development tools. Fresh from raising $500 million, the startup has secured the big-name customers for a newly established biopharma molecular simulation division that aims to accelerate R&D.

Fierce Biotech Fundraising Tracker '23: Tagworks raises $65M series A; Flagship launches Empress with $50M

Alongside the new year, we've launched a new Fierce Biotech Fundraising Tracker to keep the pulse on the industry's financing.

Chutes & Ladders—Bayer promotes Sebastian Guth to helm all US operations

When Bayer named former Roche pharma chief Bill Anderson as its new CEO, it was clear the company would be leaning into its pharmaceutical business focus moving forward. That’s been further cemented with the promotion of Sebastian Guth as head of the company’s U.S. operations.

'The Top Line': The most expensive drugs in the US, plus this week's headlines

This week on "The Top Line," we discuss the most expensive drugs in the U.S., plus Eli Lilly's recent acquisition, the latest company to challenge the Inflation Reduction Act, and the rest of the week's headlines.

UPDATED: After delays, Sarepta's DMD gene therapy Elevidys finally crosses FDA finish line at $3.2M

After the FDA said it needed "modest additional time" to review the therapy, the agency has ultimately approved the Duchenne muscular dystrophy gene therapy under the accelerated approval pathway. It's now branded as Elevidys.

Medtronic signs on to distribute Allurion's swallowable balloon, AI platform for weight loss

The next year is set to be a busy one for Allurion: Not only is the weight-loss technology maker planning to go public this summer, but it has also now tapped Medtronic to help bring its technology to even more healthcare providers and patients around the world.

UPDATED: Intercept restructures as Ocaliva's NASH hopes dashed again with FDA rejection

Even after a second application attempt, Intercept’s Ocaliva wasn’t the fatty liver disease breakthrough that the company had hoped it would be. Intercept is now swinging into restructuring mode, and the NASH baton passes on to Madrigal Pharma.

What CVS' exit from clinical trials means for other retail players

Last month, CVS Health announced plans to sunset its research recruitment business unit just two years after its launch. What does CVS' exit spell out for rival Walgreens and other new players in the clinical trials space?

Fierce Pharma Asia—AstraZeneca China spinoff report; Takeda's Vyvanse shortage; Enzene's New Jersey plant

AstraZeneca has reportedly worked out a contingency plan to spin out its China business amid increasing geopolitical risks. The British pharma is still forming collaborations in the country, the latest being an R&D pact focused on hypercholesterolemia. Takeda is experiencing a shortage of ADHD med Vyvanse. And more.
 
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Don't miss an episode

'The Top Line': Most expensive drugs, plus this week's headlines

This week on "The Top Line," we discuss the most expensive drugs in the U.S., plus Eli Lilly's recent acquisition, the latest company to challenge the Inflation Reduction Act, and the rest of the week's headlines.

 

Resources

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Enhance the cost-effectiveness of your GMP chemical supply chain

Learn how to develop a high-performing supply chain for cGMP chemicals used throughout your bioprocessing workflows.
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Autoantibodies: Powerful Biomarkers in Cancer Precision Medicine

Autoantibody screening is promising for early cancer detection with the potential to improve the whole treatment decision tree, providing a less invasive, powerful approach.
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Designing an Optimal APAC Clinical Supply Chain

Discover key considerations that every trial sponsor should know when navigating the complex clinical supply distribution landscape across Asia-Pacific.
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Running Decentralized Trials at Scale

Explore how clinical supply can be scaled up as decentralized programs progress, and how technological solutions can accelerate trial deployment.
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Questions You Need to Ask Your CRO About Clinical Supply

Planning on using a CRO to support your next clinical study? This infographic outlines critical areas to address at the start of the relationship in order to ensure that budget and timelines keep on track for the duration of the trial.
eBook

Strategies to Optimize Clinical Trial Enrollment Timelines

See how clinical trial sponsors can build stronger strategies to address challenges across the patient enrollment funnel, starting with four necessary steps.
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Commercializing Gene Therapies, Part 4 – Market Entry

This paper outlines various market archetypes and key considerations leaders must address when prioritizing new potential markets for gene therapies.
 

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