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Today's Rundown

Featured Story

Sarepta's DMD gene therapy fails phase 2 motor function test, sinking stock

Sarepta Therapeutics’ Duchenne muscular dystrophy gene therapy has failed to beat placebo in a phase 2 clinical trial. Functional motor ability scores in the SRP-9001 arm were statistically no better than in the placebo group, almost halving Sarepta’s stock price and raising doubts about a key program.  

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Thank you to Catalent for sponsoring this year's JPM 2021 coverage.

Top Stories

Matching Sinopharm, fellow Chinese biotech Sinovac sees 78% COVID-19 vaccine efficacy

A little over a week after Sinopharm nabbed the world’s first full approval from Chinese regulators on a 79% efficacy showing for its COVID-19 vax, fellow Chinese native Sinovac, using similar tech, has shown pretty much the same numbers.

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Buoyed by renewed clamor for infectious disease R&D, Affinivax nabs huge $226M funding round

After getting off a meaty $120 million round in April, the height of the first wave of COVID-19 infections in the West, Affinivax is a clear indicator that infectious disease and vaccine work is here to stay.

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DiCE scores $80M to roll oral IL-17 psoriasis med into the clinic

DiCE Molecules is setting its sights on the clinic. With $80 million raised from a laundry list of investors led by RA Capital Management, the company will push its lead program, an interleukin-17 antagonist, into a clinical trial in psoriasis and build out its preclinical programs.

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Aulos raises $40M to unlock the anti-cancer power of IL-2

ATP and Biolojic Design have teamed up to create IL-2-focused cancer biotech Aulos Bioscience. The startup begins life with IL-2-binding monoclonal antibodies designed by Biolojic Design and a $40 million funding commitment from ATP, positioning it to move into the clinic this year.

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Fierce Pharma Asia—China's PD-1 coverage; Fujifilm's $2B U.S. expansion; Sinopharm COVID-19 vaccine data

China's national drug reimbursement list saw local PD-1s cut prices by about 80% as foreign players failed to strike deals. Fujifilm will spend $2 billion on a new cell culture production site for its CDMO business. Sinopharm's inactivated COVID-19 vaccine showed 79% efficacy and became the first officially approved in China. And more.

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Chutes & Ladders—Pfizer CMO Rothenberg passes reins to Stanford's Habtezion

Pfizer CMO Rothenberg hits exit after 12 years. Nabel leaving Brigham and Women's General Hospital to join stealthy biotech startup. Axsome vet Marek joins Myovant as CEO two weeks after its first drug approval.

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Weigh in: Are the days of J.P. Morgan in San Francisco gone forever?

We're coming up on a new kind of J.P. Morgan Healthcare Conference next week, and as we do, some people—including us—are wondering whether the goodbye we’ve said to the old kind of J.P. Morgan Healthcare Conference is only temporary.  

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DBV Technologies laying off 200 staffers after nasty FDA reaction to its peanut allergy patch

DBV Technologies’ goal of delivering an anti-allergy skin patch has proven a tough nut to crack. After the FDA turned its patch away last year—saying its designs would need to be modified and retested—it's now planning to lay off two-thirds of its staff.

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BioNTech CEO applies COVID-19 vaccine's mRNA tech to multiple sclerosis

The new vaccine technology mRNA is making waves these days as COVID-19 shots based on it deliver efficacy that’s unrivaled by other platforms. BioNTech's Pfizer-partnered vaccine Comirnaty is one of those vaccines. Now, the German biotech’s CEO, Ugur Sahin, has led a study showing an mRNA vaccine was effective against multiple sclerosis in mice.

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Resources

Webinar: Using SPOT™ & CHO-BC® During Cell Line Development for Titers up to 9 g/L

Innovative cell lines and early process development are critical to generate a high producing cell lines alongside a USP strategy to meet high quality standards.

Whitepaper: Success in Rare Diseases: Understanding the Rare Disease Ecosystem

Biopharma companies in rare diseases: How to maximize the odds of success by becoming a valued member of the unique “rare disease ecosystem.”

Content Hub: Emerging Biotech Resource Center

A one-stop hub for Cytiva resources to support process development and manufacturing of your novel molecule

Whitepaper: Accelerate innovation and manufacturing to better serve patients

A significant number of new treatments have been approved so far, but some manufacturing and regulatory guidelines pose challenges for advanced therapies, leading to poor production yields.

Survey: Cell and Gene Clinical Supply Needs Market Research Survey

Cell and gene therapies hold the promise to offer novel therapeutic avenues for individuals facing serious medical conditions, but bringing them through the clinical trial process introduces a level of supply chain risk and complexity.

Whitepaper: Gene and Cell Therapy: A New Age of Medicine

Gene and cell therapy are shifting paradigms for manufacturers, patients, healthcare providers and pharmacies. We invite you to learn more by reading Gene and Cell Therapy: A New Age of Medicine.

Executive Summary: Manufacturing Strategies to Effectively Industrialize Autologous Cell Therapies

Autologous cell therapies have seen propelled growth since 2017, with the spotlight on the first FDA approval of a chimeric antigen receptor (CAR) T cell immunotherapy, Kymriah® (tisagenlecleucel), for the treatment of relapsed/refractory B-cell acute lymphoblastic leukemia.

Infographic: 6 Ways Phenotypic Data Improves Decision-Making in Your Drug Development Program

Learn six ways metabolomics provides greater confidence, higher success rates and complements the work of your in-house teams for your drug development pipeline.

Article: Process development and scale-up of pluripotent stem cell manufacturing

Human pluripotent stem cells (PSCs) have huge potential to produce cell therapies for many clinical areas. Here we describe a controlled, automated, and defined manufacturing process to fuel demand.

Whitepaper: The Future of Pharmacovigilance

Download the whitepaper to learn how your safety organization can implement automation technologies to enable process and cost efficiencies, improve resource allocation and ensure compliance and quality.

Whitepaper: Survey Reveals Demand for Clinical Trial Innovation in the Wake of COVID-19

The BBK Worldwide Study Voices 2020 survey gives voice to the healthcare consumer in the wake of COVID-19 and reveals important insights for clinical trial enrollment and engagement.

Research: Premier Research Resource Hub

Browse the latest learnings and insights from our clinical development professionals.

Infographic: Reducing Time to Clinic for Your Biomedical Applications

This infographic describes the benefits of GelMA in various biomedical applications and how X-Pure® GelMA can help you in your developments.

Whitepaper: Gene and Cell Therapy: Planning for Manufacturing Success Early

Gene and cell therapy organizations are asked to balance the need for rapid clinical progression and stringent quality expectations throughout development. In this document, Aldevron provides an in-depth look at preparing for plasmid manufacturing based on our years of experience.

Whitepaper: Companies Need An Effective Remote Workforce Cloud Strategy To Survive

A sound Cloud Strategy provides the flexibility and tools businesses need to quickly adapt and operate securely in an ever-changing remote work environment. Learn how your company can cost-effectively empower its employees to be productive and secure while working remotely.

eBrief: Drug Product Process Development: Ensuring a Consistent, High-quality Biologic

Learn about drug product process development challenges and risks, the difference between innovator and biosimilar drug product process development, and more during the drug product process development phase.

Q&A: Planning Successful Launches

Learn about proven strategies for a successful product launch, including cross-functional team collaboration, risk management and using the right project management tools.

eBook: CMC Considerations for Successful Early Drug Development

This eBook provides insights on key approaches and considerations for preparing your drug development program for long-term success.

Webinar: Phase Appropriate Expertise and Technologies to Accelerate Product Development to Commercialization

In this webinar, pharmaceutical scientists present real-life examples of challenges they’ve overcome in the transfer of oral small molecules from a dedicated early-phase development site to a dedicated late-phase manufacturing site.

Whitepaper: Why Your Business Needs a Ransomware Strategy To Avoid Being Hacked!

Cybercriminals Are Actively Exploiting The Coronavirus Pandemic To Gain Entry To Enterprise Networks. Don’t Let Your Company Become The Next Ransomware Headline -- Make These Changes Now!

Whitepaper: Achieving a Successful Drug Product Technology Transfer

Get insights on key considerations for a successful tech transfer process for manufacturing a biologic drug product, including a case study on overcoming challenges in a process transfer for a sterile diluent.

Executive Summary: Keys to a Successful Rapid Commercial Launch

Pharmaceutical and biotech companies are spending years developing drugs or promising new biologics, all with the hopes of saving or enhancing patients’ lives. It’s a “race to the finish” in some cases, and the quicker a company can get its product to patients, the better.

Whitepaper: The Art of Recognizing Clinical Supply Risk Factors

In a BioPharma Dive industry survey, planning and forecasting were identified as top concerns for trial sponsors. In response to this need, the CSM team at Catalent has developed a methodology for identifying, evaluating, and proactively managing the inherent risks involved in clinical trial supply chain management.

Case Study: Establishing Parameters for Success

Learn about a real-world example of how clinical supply management expertise was applied to overcome inventory challenges for a complex, global study.

Fact Sheet: Forecasting to Optimize Clinical Trial Supply Management

Whether a study is simple or highly complex, forecasting and simulation reports serve as data driven communication tools to help minimize risks and keep the study on track. Learn more about forecasting solutions for informed decision making and supporting contingency planning for clinical study.

eBook: Expecting the Unexpected: Strategies for Efficient Clinical Supply Management and Forecasting

Learn how to drive excellence within the forecasting process and utilize forecasting throughout the clinical study to better plan clinical supply budgets and project timelines. Explore how to identify potential supply-related issues before they can negatively impact your study.

Events