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Today's Rundown

Featured Story

Convalescent plasma fails phase 2 in moderate COVID-19 patients

A randomized phase 2 clinical trial has found convalescent plasma has no effect on progression to severe disease or all-cause mortality in COVID-19 patients. The data raise significant doubts about the efficacy of an intervention that has been given to more than 100,000 people in the U.S.

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Top Stories

Alnylam makes case for lumasiran in infants ahead of FDA ruling  

Alnylam has shared data from a phase 3 trial of its near-approval kidney disease drug lumasiran in children as young as three months. The RNAi therapy drove a 72% reduction in urinary oxalate from baseline, boosting Alnylam’s hopes of using the drug in a broad patient population.

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Retrophin buys up Orphan Technologies, spending $90M on rare disease drug

Retrophin has snapped up Orphan Technologies for a $90 million upfront payment, gaining access to the biotech's rare metabolic disorder drug OT-58.

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Ultragenyx dives into Duchenne with $40M Solid Bio deal

Solid Biosciences has had a roller coaster couple of years, with its gene therapy for Duchenne muscular dystrophy enduring multiple FDA holds due to safety concerns and manufacturing issues. Now, the biotech is getting a hand with its Duchenne work through a partnership with Ultragenyx worth $40 million upfront but could net it another $255 million in milestone payments.

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Sirnaomics nabs a major $105M funding round for next stage of its RNA work

After posting positive results from a midstage test for its RNAi candidate back in the spring, Sirnaomics has grabbed a $105 million series D round.

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Could a language test diagnose Alzheimer's? IBM is laying the groundwork

Doctors use brain scans and spinal taps to diagnose Alzheimer’s disease, but these methods and usually aren’t pursued until after a person has shown signs of cognitive decline. IBM thinks it has the beginnings of a solution: an artificial intelligence model, developed with Pfizer, that could eventually predict if a person will develop Alzheimer’s using a simple language test.

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FiercePharmaAsia—Dr. Reddy's under cyberattack; Insilico-Taisho AI collab; Fosun's Gland IPO

Dr. Reddy's detected a cyberattack as COVID-19 vaccine trial kicks off. Taisho tapped Insilico Medicine's AI drug discovery engine to identify drugs for age-related diseases. Gland Pharma, majority owned by Fosun Pharma, has reportedly won clearance for an $816 million Indian IPO. And more.

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Chutes & Ladders—Kite Pharma veteran Nowers takes the helm at ONK

Onk Therapeutics taps Nowers to take the helm; BD replaces chief technology officer post with insider Kaltenbach; Vaccitech nabs Marshall as CMO.

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Resources

Whitepaper: Patient Affordability Legislation: How This Frontier in the Pharmaceuticals Industry May Shape Your Business

This white paper outlines how one such law, Minnesota’s Alec Smith Insulin Affordability Act, has impacted the pharmaceutical industry and provides guidance about strategies businesses can employ to rapidly add support.

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: COVID-19 Vaccine Clinical Trials in Children: Challenges on the Horizon

Understanding the Challenges COVID-19 Pediatric Clinical Trials Face | White Paper

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.

Whitepaper: Exploring the patient dynamic from different perspectives

The culture of clinical research is evolving from one directed by researchers to one driven by the needs of patients and those who care for them.

Whitepaper: High Content Screening in 3D: Faster 3D Image & Multi-Parametric Data Analysis

Creating integrated HCS workflows in a single platform increases accuracy; reduces time and helps to identify the most appropriate hits or drug candidates. Learn more about a reliable method for creating a more efficient, faster, 3D image and multi-parametric data analysis workflow.

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.

Whitepaper: Go from Process R&D to Clinical APIs Quickly and Effectively

Struggling with complex chemistry to get APIs for clinical trials? Find out what to look for in an API CDMO partner to gain expertise and speed that lead to success.

Whitepaper: COMPLIANCE –THE COMPETITIVE DIFFERENTIATOR TO COMMERCIALIZATION: An Integrated Model Setting the New Global Standard

An integrated compliance model can be the competitive differentiator to commercialization and move the needle from cost center to value. Learn How.

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: How to Achieve Time and Cost Reductions in Clinical Trial Design and Build

Learn how to save time and reduce costs and complexity by streamlining the clinical trial process.

Whitepaper: Position Your Pharmaceutical Lab for Success

Want to leapfrog lengthy LIMS deployments and position your pharmaceutical lab for success? Find out how with LabVantage Solutions.

Whitepaper: Technology Transfers: Reaping Rewards, Reducing Risks

Learn how to safeguard supply, improve distribution and reduce program costs and risks

Whitepaper: Making Data Central to Your CNS and Pain Trials

Learn how the right data capture solution can prompt the patient to follow the correct procedures and automatically sort the responses, according to protocol.

Whitepaper: Research Reveals New Launch Standards for First-to-launch Pharma and Biotech Companies

While COVID-19 has shifted the rules of the healthcare industry, research shows there was already progress on changes being made for how pharma companies are launching products.

Whitepaper: 16 Biopharma Leaders Tell How COVID-19 Will Change Customer Engagement

Senior executives from 16 biopharma companies share their thoughts on how COVID-19 will change how the industry engages with its customers.

Report: State of Life Sciences | 2020 Trends Report

A complete Life Science Industry Snapshot in One Report

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