What’s next for biotech? Q4 2023 funding trends point beyond the usual suspects

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Oncology may continue be one of the hottest sectors across the pharma sector, but other therapeutic areas are catching up in terms of innovation and investment. While oncology and hematology jointly accounted for about one-third of the new FDA approvals in 2023, investors are increasingly betting on precision medicine, advanced drug delivery systems and the use of AI and machine learning for drug discovery and development.

The shift is not entirely new. In a report from late 2023, McKinsey also notes ML-enabled drug discovery, cell therapies, and gene editing continue to attract significant funding, making up over two-thirds of biotech VC deals in 2022, constituting $15.5 billion in total. The consultancy also noted that immunology had displaced oncology as the hottest therapeutic area for asset-based biotech investments, also citing allogeneic cell therapies …

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Core trends in 2023 FDA drug approvals: Oncology, neurology and hematology dominate

2023 was a big year for hematology, neurology and oncology, with the medical specialties seeing the most FDA approvals. In terms of sponsors, Pfizer had the most approvals with six total, followed by UCB and Chiesi, each with three apiece.

When looking at commercial prospects, AstraZeneca’s respiratory syncytial virus antibody Beyfortus could be the biggest blockbuster from the 2023 cohort with expected peak sales of $1.9 billion. Alzheimer’s drug Leqembi could also be a major success, making up for the tepid demand for Aduhelm, which won conditional approval in 2021. While Aduhelm’s sales have sputtered, Leqembi could see peak revenue of $4.8 billion according to analysts.

Oncology continues to be a hot specialty with the most competition among the newly approved drugs, as the FDA continues to green light new entrants in crowded target classes such as PD-1 inhibitors.

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Zai Lab’s unified approach to tackling cancer, autoimmune, and neurological diseases

Zai Lab is a global biopharmaceutical company founded in 2014 and based in China and the U.S.. Led by Chairperson and CEO Dr. Samantha Du, the company now has more than 2,000 employees internationally.

Focusing on developing novel therapies for oncology, autoimmune disorders, infectious diseases, and neurological disorders, it has built a broad pipeline of late-stage drug candidates, five of which have been approved and launched in China. The company also has internal research and development capabilities to advance its discovery pipeline.

The company is strategically partnering with prominent biopharma organizations such as Amgen, GSK, Seagen, BMS, Mirati and Deciphera. Concurrently, Zai Lab is building a proprietary pipeline across therapeutic areas such asoncology, autoimmune disorders, infectious diseases, and neuroscience.

In this email Q&A, we heard from Josh Smiley, president and chief operating officer of Zai Lab, and Dr. Harald Reinhart, pre…

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Pfizer veteran pays it forward through AAPS mentorship 

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When Cindy Oksanen started out at Pfizer’s Groton, Connecticut research headquarters in 1992, she entered an environment where female Ph.D.s in leadership positions were rare. Starting out as a lab scientist, she developed formulations for new drugs including oral dosage forms like tablets and capsules. “We would develop the dosage forms and then transfer the manufacturing technology to sites around the world,” she recalled. At the time, there were roughly 10 men to every female Ph.D., Oksanen recalled. “It’s changed now, but back then, there weren’t many female role models in leadership roles,” she added.

Oksanen quickly assumed supervisory and leadership roles in analytical and manufacturing departments. Over time, she led development of more than 20 drugs from scratch through to regulatory approval. “I learned a lot about how to develop a drug product,&#…

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Pharma M&A activity primed for another high-flying year in 2024

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Despite challenging interest rates and regulatory environment, pharma M&A activity surged in 2023 with deal volume up more than 30% from the prior year based on a review of more than 200 acquisitions since 2018. The total disclosed deal value in 2023 also more than doubled the prior year’s tally to surpass $100 billion. While the pandemic boosted research into areas such as mRNA, it had something of a chilling effect on M&A. As pharma companies begin to put the pandemic in the rearview mirror, M&A activity has gained momentum. 

Analysts upbeat on biopharma M&A activity in 2024

According to PwC, the M&A activity in the pharmaceutical and life sciences sector could continue humming in 2024. Despite a challenging interest rate environment, PwC projects the sector to see deal values ranging from $225 billion to $275 billion. Deal volume in 2023 was in line with pre-pandemic levels. …

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Integrated bioanalysis strategies in nonclinical early in-vivo PK studies of siRNAs

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In molecular biology, small interfering RNAs (siRNAs) have emerged as pivotal tools for regulating gene expression through RNA interference (RNAi). These double-stranded RNA molecules, consisting of 19-23 base pairs, play a crucial role in research and drug development by inducing the degradation of target mRNA and inhibiting gene expression.

This article explores the complex world of siRNA drugs, highlighting their status in the market and their expanding role in clinical research. It explores the early in vivo pharmacokinetic (PK) studies of siRNAs, underscoring the necessity for diverse bioanalytical platforms. This diversity is essential due to the complex behavior of siRNAs in various biological environments. The article examines critical aspects of siRNA analysis, including exposure levels, tissue distribution, the concentration of RNA-induced silencing complexes, and the effectiveness in deg…

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A J&J veteran highlights listening to address overlooked maternal-fetal needs

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With nearly 20 years of experience at Johnson & Johnson, Cynthia Accuosti Jones now spearheads the company’s pioneering maternal-fetal medicine group tackling unmet needs in high-risk pregnancies. As vice president of global commercial strategy for autoantibody and maternal fetal health, she spotlights listening as the driver for patient impact. “You can’t have empathy if you don’t listen to people and it’s incredible how much people just need to be listened to,” Jones emphasized, underscoring the importance of addressing the often-neglected area of maternal health.

Jones’ longtime interest in healthcare stemmed from a desire to help people. She considered nursing but ultimately pursued finance and international business at Georgetown, building on childhood analytical skills tracking her father’s stocks. That experience piqued her interest in financial markets and data analysis. She…

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Wrangling medical imaging data: Strategies to streamline AI-powered workflows

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The value of artificial intelligence (AI) and machine learning (ML) in medical imaging is undeniable: more accurate diagnoses, predictive insights and streamlined workflows. However, as Big Pharma and medical research institutions amplify their AI and ML endeavors, they confront pivotal challenges. Chief among these challenges are the intricacies of labeling and annotating medical images.

Approximately 80% of the time it takes to prepare real-world data for downstream analysis is used on seemingly foundational tasks like locating, curating and structuring data. When we narrow our focus to medical imaging, the stakes rise significantly. The sheer volume of data required to do this work makes using human-only annotation close to impossible. And in this domain, the most minor details can critically influence diagnostic accuracy, making it imperative that data is not only accessible but clearly organiz…

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ConcertAI acquisition will boost CancerLinQ data capabilities with AI focus

The AI oncology startup ConcertAI recently acquired CancerLinQ, one of the largest oncology real-world data and quality of care technology service entities. Originally developed by the American Society of Clinical Oncology (ASCO) in 2013, CancerLinQ aims to use real-world data and technology to improve cancer care and advance evidence-based research. 

CancerLinQ has developed one of the “deepest, broadest, most generalizable, least biased single standalone data sources that exists in oncology,” according to Jeff Elton, CEO of ConcertAI. This real-world data asset encompasses clinical data across more than 7 million patients, 100 care sites and  more than 10 EMR systems.

Boosting CancerLinQ’s scope and capabilities

The acquisition will bring considerably more resources to CancerLinQ, which had a dedicated staff of between 50 and 60 people in comparison to ConcertAI, which has more than 1,100 employees. Under terms of the deal, ASCO will mainta…

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A nurse-turned-pharma-exec strives to advance care for those with an autoimmune liver disease

Intermediate magnification micrograph of primary biliary cirrhosis, from a liver biopsy with H&E stain. Image by Nephron, licensed under CC BY-SA 3.0, via Wikimedia Commons.

Now the vice president and head of medical affairs at CymaBay Therapeutics, Mary Standen‘s passion for healthcare was sparked in her high school years when her mother was diagnosed with breast cancer. Though her mother eventually recovered, Standen was galvanized to help provide both emotional support and practical assistance, such as administering weekly injections. The experience made Standen feel empowered to join her mother’s battle against cancer. “I felt I had it in my power to help with the fight,” Standen said.

The experience proved to be the initial formative experience that propelled Standen into a career dedicated to healthcare and patient advocacy. “After earning her bachelor’s in Psychology from Emory U…

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Using AI to unlock new uses for existing cancer medicines

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Repurposing is a drug development strategy that has been widely applied in cancer. This strategy, sometimes called label expansion, involves obtaining FDA approval to market a drug for the treatment of new indications, alone or in combination with other drugs. Not only can this approach extend the window of patent protection for a commercialized drug, but the path to regulatory approval can also be far less costly and risky than the traditional approach to drug development, largely because clinical evidence of safety already exists. And most importantly, the result is more and better treatment options for patients.

In keeping the law of unintended consequences, recent provisions introduced by the 2022 Inflation Reduction Act (IRA) blunted the potential benefits of drug repurposing. As a result of limiting the window of time during which pharmaceutical companies can recoup investments before…

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How a J&J exec found her calling in autoantibody drug development

Katie Abouzahr

Dr. Katie Abouzahr’s career, which began in the wards of the UK’s National Health Service (NHS) before extending into management consulting, paved the way for her leadership of autoantibody and maternal fetal medicine programs at Johnson & Johnson. “It’s not a typical pharma executive’s straight line path,” she acknowledged. “Careers can often be jungle gyms as opposed to ladders that you climb one rung at a time. It’s always a mixture of opportunity, timing, serendipity, and persistence.”

Those qualities helped pave the way for her current focus on autoantibody-based conditions marked by high unmet need. Drawing on her experience across clinical medicine, consulting, and drug development, Abouzahr focuses on advancing medicines for autoimmune and autoantibody-driven diseases, which are marked by high unmet need. The broader immunology area is a central …

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