Guest Column | April 19, 2024

Navigating Growth In Biotech: A First-Person Insight

By Lisa Ricciardi, CEO, Cognition Therapeutics

Lisa Ricciardi

As the CEO of Cognition Therapeutics, I've had the privilege of steering our company through many challenges and opportunities in the biotech industry. It's an industry where the stakes are high, the risks are numerous, and the potential for transformative impact on human health is immense. In this dynamic landscape, clinical stage biotech companies face a unique set of challenges, especially in the wake of the post-pandemic biotech recession that enveloped the industry in 2022 and 2023 and from which we are finally beginning to emerge.

Strategic investors eager to secure stakes in emerging innovations that drive their long-term business goals are approaching collaborations and acquisitions with a level of scrutiny that is both formidable and necessary. Encouraging breakthroughs in treatments for diseases like Alzheimer’s have been tempered by recent sobering realities adding to the already considerable baseline of due diligence these investors routinely conduct. A cautious stance from strategic investors signals a return to normalcy in a sector that briefly experienced a financing bonanza anomaly in the Covid-response period. So, it’s back to business as usual where building sustainable, long-term value is predicated on the quality of foundational science and the demonstrated ability to successfully navigate the many challenges of complex clinical trials.

Throughout my career in pharma and biotech, I've consistently observed the criticality of five attributes that are essential for emerging biotech companies to thrive and grow in what will be a challenging financing environment for the foreseeable future. These attributes are in many ways a proxy of the fundamentals needed to create long-term, sustainable value.

High Quality Science: The Bedrock Of Biotech Valuation And Partnership Opportunities

At the core of any successful biotech company, high-quality science is the foundation for successful clinical development. This seems self-evident, yet it's astonishing how often this foundational principle is overlooked in the rush to market. Scientific rigor must permeate every facet of our work, ensuring that only the most promising and thoroughly vetted therapies reach those in need. This rigorous approach to drug development not only maximizes patient benefits but also significantly enhances a company's market valuation. A company that is critical of its own science can withstand the scrutiny from the outside world with confidence.

Proven Target Engagement: Demonstrating Real Impact

In the complex field of drug development, proving that a therapeutic candidate can meet its endpoints is crucial. This involves a comprehensive strategy that encompasses strategic target assessment, sensitive detection methods, and a combination of phenotypic and mechanistic approaches to demonstrate both clinical and biologic activity. Such a robust testing framework is indispensable for validating drug targets and their biological activity, serving as a key indicator of the intrinsic value of our scientific endeavors.

Rigorous Phase 2 Clinical Trials: The Litmus Test For Efficacy

The road to bringing a drug to market is fraught with challenges, with many potential therapies failing to make it past crucial clinical milestones. It's essential to resist the temptation to rush into large-scale Phase 3 trials without a solid foundation of evidence from well-conducted, randomized placebo-controlled Phase 2 studies. These early trials are not merely procedural steps but are critical in demonstrating safety, efficacy, and patient benefit. Meticulously designed and seamlessly executed Phase 2 clinical trials make a compelling case to investors and partners about the viability of a therapy.

An area that often gets less attention but is critically important is finely tuning dose escalation in Phase 2 trials. Effective dose escalation in Phase 2 is foundational to a successful Phase 3 trial, as it ensures the chosen dose is both safe and an appropriate quantity to demonstrate potential efficacy. Another area to consider is the potential shortfalls of open-label clinical trial design. Open-label Phase 2 studies, or those based on post-hoc subset analysis, can increase the likelihood of failure in Phase 3. This is because open-label trials may compromise the reliability of the data collected, making it challenging to design an effective and unbiased Phase 3 trial. Open-label Phase 2 clinical trials may not fully capture the placebo effect, as the participants' knowledge of receiving a specific treatment can alter their responses, potentially skewing the results.

Enrolling Patients, Not “Subjects”: A Patient-Centered Approach

In clinical research, the way we view and treat patients can profoundly impact the outcomes of trials and, ultimately, patient care. Adopting a patient-centered approach that respects the dignity and autonomy of individuals is not just an ethical imperative but a strategic one. By building trust and prioritizing patient welfare, we can achieve better compliance and outcomes and, consequently, better therapeutics. This approach is particularly crucial for patients with neurodegenerative diseases, a group that has historically been underserved by our industry.

The company I lead, Cognition Therapeutics, is engaged in developing therapeutics with the patient in mind. Our lead candidate, CT1812, is a small molecule oral therapeutic currently in separate Phase 2 clinical trials for the treatment of mild-to-moderate Alzheimer’s disease and dementia with Lewy bodies respectively. Patients suffering from these neurogenerative diseases have few treatment options, all of which require intensive and time-consuming regimens. These regimens impose a considerable burden on patients and their loved ones, who often play a vital role in their care. Our work is aimed at developing a treatment regimen that does not add to the already immense burden those suffering from these neurodegenerative diseases already must endure.

Collaboration With The Scientific Community: Building Trust And Engagement

Clinical trials are not just scientific experiments; they are endeavors that require trust, collaboration, and engagement with a broad community of stakeholders. Principal investigators and clinical trial leaders play a pivotal role in this process. Their ability to identify and address issues, ensure patient safety, and maintain data integrity is invaluable. Moreover, their deep connections within professional and personal communities can significantly enhance patient engagement and awareness, driving the success of clinical trials and the development of quality drugs. We need to be willing to share our science, listen carefully to their input, and view the research community as partners who share the same goal of accelerating the advancement of beneficial treatments to patients and their families.

Building Long-Term, Sustainable Growth

As we navigate the complexities of the biotech landscape, these five attributes serve as guiding principles for Cognition Therapeutics and, I believe, for the industry at large. In a world where investment dollars are increasingly discerning, embodying these principles can set the foundation for long-term, sustainable growth. It's a journey filled with challenges, but with unwavering commitment to quality, efficacy, and patient-centricity, we can continue to drive innovation and bring life-changing therapies to those in need.

About The Author:

Lisa Ricciardi is the CEO of Cognition Therapeutics, a clinical-stage neuroscience company developing CT1812, an oral small molecule therapeutic to treat neurodegenerative diseases like Alzheimer’s disease and dementia with Lewy bodies. Lisa’s extensive biotech experience includes leadership roles at Suono Bio and Foundation Medicine, where she played a pivotal role in business development and global corporate strategy. She holds a BA from Wesleyan University and an MBA from the University of Chicago Booth School of Management.