BIO 2023 Takeaways: AI, Accelerated Approval, Patient Diversity

When it comes to the BIO International Convention, there’s likely no better place to be and no more diverse topics of discussion for leaders, researchers, and supporting professionals in biotech. Particular to clinical research, there was plenty of presentations and panel discussions — not to mention the opportunity for vendors and sponsors to sit down one on one — to hear from and engage with experts with know-how in drug discovery all the way through regulatory considerations to clinical trial design and execution.

Here, we’ve collated a handful of BIO 2023 impressions from attendees representing small and mid-size biotech/pharmas, consultancies, and professional organizations.

R. Nolan Townsend, CEO, LEXEO Therapeutics

“Prominently discussed across BIO was the accelerated approval pathway which came up across sessions with FDA Commissioner Califf, in the context of the often-discussed Alzheimer’s therapies (lecanemab) and Sarepta’s DMD gene therapy. This approval pathway represents the best manner in which patients with rare diseases or other diseases of high unmet need — like Alzheimer’s disease — can more rapidly access the lifesaving or life altering treatments they need. From a policy perspective, there are a number of evolving considerations around this pathway ranging from the link between accelerated approvals and the ultimate reimbursement of the medicine to the data required to receive an accelerated approval in the first place. Importantly, I think this pathway is a critical one for drug developers to consider because it fundamentally changes the regulatory context in which a medicine is advanced, allowing for a potentially less risky, more predictable and faster path to an approval. However, most importantly, this pathway allows the biopharmaceutical industry to deliver against the expectations that society has of us; developing therapies for diseases that have no treatment or insufficient treatments.”

Jonathan Steckbeck, Ph.D., MBA, CEO & Founder, Peptilogics

"One of the clearest trends at BIO 2023 was the buzz and interest in AI and machine learning. There is increasing receptiveness and sophistication toward the role that AI and machine learning can play in drug discovery and development and where it can have real impact. I noticed a significant shift in recognition of the importance of high-quality algorithms that can deal with biological complexity compared to the simple idea of ‘more data’ as a solution." 

Atul Deshpande, Ph.D., MBA, A-PMP, Chief Strategy Officer, Peptilogics

“A trend I noticed at BIO 2023 revolved around clinical research with patient-centric approaches. Companies that are incorporating next generation AI to recruit and retain patients are revolutionizing the field. Interestingly, some companies I met with are moving away from just collecting petabytes of data, to really building algorithms from smaller data sets to generate the right information to run effective clinical trials. Although they’re a few steps away from optimizing this transformative approach, I strongly believe they are paving the way for a novel approach to AI and not so Big Data.”

James F. Jordan, President, StraTactic

"Amidst the backdrop of the 2023 BIO International meeting in Boston, it becomes evident that the biotech industry is grappling with a critical challenge: the scarcity of drug candidates progressing through the pipeline, irrespective of disease area. Compounding this issue are the escalating costs associated with clinical trials, coupled with increased regulatory demands from the FDA. To illustrate this predicament, envision an apple pie. In the era of blockbuster drugs, one could divide the pie into thirds or fourths. However, today, the pie is sliced into eighths or sixteenths. With mounting expenses to develop a commercially viable drug, dividing the pie so thinly inevitably hampers profitability. Irrespective of the specific disease targets pursued by attending companies, the pressing questions emerge: How can we expand the size of the pie, and how can we mitigate the costs associated with bringing a potential commercial drug candidate to fruition? These questions underscore the paramount need for innovative approaches and collaborative efforts within the biotech industry to address these challenges head-on and pave the way for a more sustainable and prosperous future."

Jennifer Goldsack, CEO, Digital Medicine Society (DiMe)

“BIO 2023 solidified that we're in a new era of digital innovation in drug development. Presentations, panels, and networking discussions were all characterized by a fundamentally different and refreshing attitude about digital innovation. We saw appreciation for the slew of recent guidances, frameworks, and meetings offered by FDA. We heard numerous accounts of successful collaboration between sponsors and digital solutions providers. We listened to insights into sophisticated strategies capturing extensive ROI from digital innovation. This was a huge evolution from talk in recent years of 'just tossing a wearable into the trial.' It's clear that digital tools are being regarded thoughtfully, intentionally, and as a fundamental part of our ecosystem.”

Hamid Khoja, Chief Scientific Officer, FibroBiologics

"One striking feature of BIO2023 was the energy from attendees excited to meet in person, enhancing the possibilities for developing collaborations. The presentations were full of information, and with regards to clinical trials, I was most intrigued by the potential benefits of decentralization and in discussions of strategies for enhancing trial diversity. For early-stage companies planning for clinical trials, these are important considerations, and BIO2023 was a rich source of actionable insights." 

Shankar Musunuri, Co-founder and CEO, Ocugen Inc.

“The BIO International 2023 agenda was extremely comprehensive and covered a wide range of topics from advances in clinical trial design and health technologies to the economics surrounding today’s biotech industry and the need for further global expansion. We particularly benefitted from the presentations led by U.S. government agencies that provided insight into potential collaboration, which is very relevant to our current vaccines program. In addition to the learning opportunities, BIO International provides a forum for companies like Ocugen to engage with new businesses through their One-on-One partnering sessions. We made some important introductions that could ultimately have a meaningful impact on our pipeline development and potential collaborations.”

Robert Popovian, Pharm.D., MS, Chief Science Policy Officer, Global Healthy Living Foundation

“The 2023 Bio conference was exemplary in helping attendees learn about new research, development, and funding opportunities for new therapies. Unfortunately, the conference did not do a comprehensive deep dive for attendees to learn and advocate regarding challenges such as drug pricing, spending, affordability, and access. Without changing and influencing the marketplace, including [pharmacy benefit manager] and insurer tactics that hinder the ability of patients to access life-saving medicines, the promise of new drugs helping patients in need will not be fulfilled.”

Joseph Tucker, Ph.D., CEO, Enveric Biosciences

“Our takeaway from BIO2023 was that this year there seems to be a noticeably more serious review taking place by the larger companies of smaller company assets that they could possibly in license or acquire. It seems that the larger companies have available capital, recognize that they have holes in the early-stage parts of their pipelines that they’d like to fill, and a sense that difficult stock markets have cause valuations to decrease somewhat from the earlier lofty heights while also making capital raising tougher for earlier stage companies. Together these forces seem to be leading to a possible uptick in M&A over the coming 12 months.”

Juliet Williams, Ph.D., Senior Vice President, Head of Research, Kymera Therapeutics

“The industry’s expanding toolbox of modalities is giving us an opportunity to pursue high value, genetically validated targets that we have been unable to drug with traditional technologies. It’s exciting to imagine the impact that these programs will have on the treatment of disease and patients’ lives.”