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Successful drug development requires a deep understanding of the disease of interest. In rare diseases, however, much of the information may be
unknown. Patient populations are small and historical data are collected
inconsistently. Patient registries and natural history studies are valuable sources of rare disease information for sponsors seeking to design reliable clinical trials - read our blog to explore their distinctions.
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Today's Big NewsFeb 2, 2023 |
| By Annalee Armstrong CEO David Ricks expressed a shrug over the FDA rejection of the accelerated review for Alzheimer’s med donanemab, while the company’s chief scientist batted back concerns that the FDA was worried about safety. |
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By Nick Paul Taylor Roche’s long-stuttering attempt to bring the cancer drug candidate ipatasertib to market has run out of steam. After multiple clinical setbacks, the Swiss drug developer has dropped a phase 3 prostate cancer trial—and sent its acute graft-versus-host disease (aGVHD) prospect packing at the same time. |
By James Waldron Once neck and neck with Valneva to get the first chikungunya vaccine to market, it now looks like Merck & Co. has admitted defeat as its French rival reaches the finish line. |
By Nick Paul Taylor Eli Lilly’s second stab at developing a KHK inhibitor in diabetes and nonalcoholic steatohepatitis (NASH) is over. Fifteen months after dropping its first candidate, Lilly has kicked the second program from its phase 1 pipeline, dealing another blow to a target that has attracted the attention of Pfizer. |
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April 3 - 4, 2023 | San Diego, CA Connect with leading communications professionals and have honest, open discussions about how to develop your career in the industry. Early Bird Rate Ends February 10th.
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By Max Bayer Bristol Myers Squibb has dumped nearly 10 early to mid-stage assets as part of a new year pipeline cleanout, including a phase 2 solid tumor candidate. The news comes just a day after the company ended a phase 2 trial testing its anti-TIGIT candidate. |
By James Waldron Fierce Biotech may have only broken the story of Goldfinch Bio’s demise last Friday, but Karuna Therapeutics has already raced to the closing down sale to take its pick of the shuttered biotech’s pipeline. |
By Annalee Armstrong With 93% of experimental pain drugs failing in phase 2 over the past five years, it’s not hard to see why pharma has been resistant to drug development in this area. A new report from the Biotechnology Innovation Organization catalogs the dearth of investment in new nonaddictive drugs to relieve pain despite an obvious unmet need. |
By Annalee Armstrong Purple Biotech is adding Immunorizon’s tri-specific antibodies to its paint palette in an acquisition that could reach $100 million in milestone payouts. |
By Max Bayer Mesoblast is giving it another go with regulators, submitting a second approval application for its off-the-shelf cell therapy. The treatment was originally rejected in 2020 due to a lack of convincing data. |
By Annalee Armstrong A day after imposing a second round of layoffs, Inovio revealed a small data set for its Ebola vaccine booster, which the company hopes could someday also boost its fortunes. |
By Joseph Keenan CVS Health Clinical Trial Services, a unit of the pharmacy giant CVS, signed up to begin enrolling patients for ProKidney’s phase 3 trial of its ReACT cell therapy for chronic kidney disease. |
By Helen Floersh By modulating a key circadian rhythm protein, the compounds potentially reduce scar tissue buildup and reduce the risk of hypertrophic scars after surgery. |
By Joseph Keenan IQVIA inked a partnership deal with Alibaba Cloud, a unit of Alibaba Group, to leverage Salesforce in a first-of-its-kind deal in mainland China. |
By Angus Liu As the incoming chair of the industry trade group PhRMA, Novartis CEO Vas Narasimhan has ideas about how the industry should respond to the consequential U.S. drug pricing law. |
By Frank Diamond Long lists of long COVID symptoms exacerbates the confusion surrounding the condition that makes diagnosing and treating patients difficult. A new study narrows the list of long COVID symptoms down to just seven. |
Fierce podcastsDon't miss an episode |
| | This week on "The Top Line," we hear from Stacie Dusetzina, Ph.D., a professor at Vanderbilt University's Department of Health Policy, about what the IRA means for the pharma industry. We'll also discuss what we can expect from the biotech industry in 2023. |
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May 1 - 3, 2023 | Jersey City, NJ Connect on current challenges and opportunities found within your medical affairs role to become better equipped in developing a unified scientific voice. Take advantage of the Early Bird Rates, Register Today!
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| Whitepaper Discover the Power of Small with NanoplasmidTM – the next-generation plasmid for manufacturing today’s cell and gene therapies. Sponsored by: Aldevron |
| WhitepaperThis paper explores the cost drivers behind gene therapies and how they are likely to evolve in the coming years. Sponsored by: Blue Matter |
| WhitepaperExploring the biology of G Protein-Coupled Receptors (GPCRs) and outlining a novel approach for developing therapeutics against previously undruggable GPCRs Sponsored by: Orion Biotechnology |
| WhitepaperLearn more about critical success factors for your next launch based on the experience of 100+ pharma product launches. Sponsored by: Herspiegel Consulting |
| WhitepaperRCTs with digital twins require fewer control patients and shorten trial timelines for Alzheimer's disease. Sponsored by: Unlearn AI |
| Whitepaper What makes nucleotides suitable for current good manufacturing practices? Sponsored by: Thermo Fisher Scientific |
| On-Demand Webinar See how high-throughput DNA synthesis combined with antibody engineering expertise provides end-to-end antibody discovery solutions. Sponsored by: Twist Bioscience |
| Whitepaper Download this paper to learn considerations on how to move a biologic through clinical trials faster, advantages of Parallel Processing, and more. Sponsored by: Catalent |
| WhitepaperWhat are key considerations for intranasal delivery in the treatment of acute and chronic conditions? Sponsored By: Catalent |
| WhitepaperLearn how Catalent’s extensive network across APAC can meet a wide range of clinical supply needs for sponsors conducting trials within the region. Sponsored by: Catalent |
| WhitepaperExplore Catalent’s newest clinical supply facility in Japan, including the comprehensive services & solutions it offers to help support trials both in-country & around the world. Sponsored by: Catalent |
| Case Study Learn more on an approach for process characterization for the production of a monoclonal antibody, which will improve the efficiency and effectiveness of the process by targeting the right design and number of experiments. Sponsored by: Catalent |
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