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February 01, 2018

Today's Rundown

Featured Story

Roche dumps 4 eye, CNS and cancer drugs in Q4 clear-out

Swiss major Roche announced its financials today, and buried within its pipeline updates the company announced it was dumping a host of early- and late-stage candidates.

Top Stories

Avrobio pulls in $60M to push Fabry, Gaucher candidates through the clinic

With the financing, Avrobio plans to push its pipeline through the clinic "as aggressively as possible." Its Fabry treatment will enter phase 2, while its Gaucher and cystinosis programs will start phase 1 trials.

Voyager founder and CEO to step down, no replacement yet

A few months after Sanofi said it didn’t want to be involved with its gene therapy for Parkinson’s, Voyager Therapeutics’ chief and founder Steven Paul, M.D., is stepping down to become an “executive science advisor.”

Vertex picks drugs for mass-market cystic fibrosis combinations on back of strong midphase data

Vertex has picked the two next-generation correctors it will advance into phase 3 cystic fibrosis trials. VX-659 and VX-445 landed spots in experimental triple combinations designed to treat 90% of cystic fibrosis patients after delivering strong efficacy data in midphase trials.

AstraZeneca, Syndax ally to test PD-L1 drug and CSF1R inhibitor in solid tumor patients

AstraZeneca and Syndax have teamed up to test immune checkpoint inhibitor Imfinzi in combination with CSF1R inhibitor SNDX-6352. Syndax is set to put the cocktail through a phase 1b solid tumor trial to equip both companies to sponsor midphase trials to demonstrate its safety and efficacy. 

MIT study finds that 14% of trials see approvals—higher than first thought

A 14% approval rate for drugs coming out of clinical trials may not sound great, but researchers at the Massachusetts Institute of Technology say this is much higher than they thought, and that approvals are bouncing up again after a period of decline.

Repairing mutation ‘clusters’ with CRISPR could lead to broader Duchenne therapy

Targeting "clusters" of mutations in the dystrophin gene could lead to a treatment for as many as 60% of patients with Duchenne muscular dystrophy, compared to the 13% of patients for whom Sarepta's Exondys 51 is indicated. CRISPR is the key to achieving this, say UT Southwestern researchers.

Enrollment Showcase

Online Graduate Programs Designed for Your Career

Drexel University’s online graduate programs in biomedicine, clinical research, drug discovery, and molecular medicine were developed with working professionals in mind, providing you with the education, practical skills, and competitive edge you need to be successful in your career. Learn more.

Resources

[Whitepaper] Streamline your mAb Intact Protein Characterization: Download App Note

This Agilent intact protein analysis includes:

[Marketplace] Asia-Pacific Compliance Training for Life Sciences Professionals

Don’t miss this educational program trusted by multi-national companies as part of their employee’s compliance training. Explore legal, regulatory and ethical issues faced by pharmaceutical, biotech and medical device manufacturers operating in the Asia-Pacific region. Visit event page for session topics.

[Whitepaper] Is Your In-House Strategy Ready For The Uncertainties Of Biologic Drug Development?

Download Patheon’s whitepaper to discover two main areas where a drug developer can face significant obstacles during biologics development and how by evaluating these capabilities, it can answer the critical question of whether its in-house strategy is ready for these uncertainties.

[Webinar] How to Prevent, Identify, and Implement a Clinical Trial Rescue

What do you do when timelines are missed, data is dirty, and everyone is pointing fingers? Join this webinar to learn how to prevent, identify, and implement a successful clinical trial rescue.

 

[Whitepaper] Key to regulatory transformation is unified RIM

Expanding into new markets places a spotlight on inefficient processes.

[Whitepaper] Best Practices for Managing Regulatory Processes 

Regulatory leaders share challenges and best practices from a lack of standardization, manual processes, and fragmented systems.

[Whitepaper] Choosing the Right Solution: Improving the Site Feasibility Process

Selecting the right sites to conduct your clinical trials will determine the success or failure of meeting your enrollment goals.

[Whitepaper] 5 Steps to Managing Quality Globally

Read this guide if you’re a global life sciences company - conducting clinical trials across several countries, or manufacturing and distributing products in multiple regions - to learn five steps to align and improve quality oversight, as well as avoid common mistakes in harmonization initiatives.

[Whitepaper] How Do Sponsors Ensure Quality in Global Clinical Trials?

Global clinical trials are the trend and gaining in popularity, but running a global trial is not a straightforward process. This white paper explores some of the challenges associated with global clinical trials and considerations for ensuring that the collected data comply with the trial's overall objectives.

[Webinar] Validating a 7 Day Membrane Filtration Sterility Test using Celsis®

Join us as we discuss how Charles River Celsis® helped Labor L+S AG validate a 7-day membrane filtration sterility test using existing protocols.

[Infographic] Evolution of Flexible Commercial Biologics Manufacturing

Learn how Catalent Biologics is paving the way for flexible biologics development and manufacturing through advanced technologies and fully integrated multi-product facility.

[eBook] Developing Site-specifically Modified ADCs: Using a Chemoenzymatic Approach

Download the eBook to get articles from various Elsevier journals highlighting developments in ADC technology.

[Article] Optimizing titer and use of a specific enzyme for generating a fully processed antibody

Learn how cell development and process scale-up can be optimized for antibodies requiring additional processing.

[Webinar] Within-Subject Clinical Trials: Intro to New Methods & Stat. Models

In this webinar, we will introduce the rapidly-evolving within-subject clinical trial (WSCT) methods for early phase evidence generation that require fewer people, less time, and usually less funding than traditional early phase methods.

Events

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