The Nobel Prize in Physics was awarded to John J. Hopfield of Princeton University and Geoffrey E. Hinton of the University of Toronto for their groundbreaking development of machine learning technology using artificial neural networks. Hinton, often revered as the “AI Godfather,” laid the foundational stones for the neural networks that power today’s AI revolution.

Simultaneously, the Nobel Prize in Chemistry recognized the transformative power of AI in solving one of biology’s most enduring mysteries. Demis Hassabis and John M. Jumper of Google DeepMind, alongside David Baker of the University of Washington, were honored for their breakthrough in protein structure prediction and design. Their creation of AlphaFold2 solved a 50-year-old scientific puzzle, enabling researchers to predict the three-dimensional structures of proteins with unprecedented accuracy.

These Nobel recognitions signal more than academic achievement – they indicate the current era where AI  becomes an indispensable tool in medical research, drug discovery, development of new treatments, our understanding of diseases, and fundamentally transform how we approach healthcare challenges in the 21st century.

However, while holding a transformative promise for healthcare, AI integration into medical practice faces significant obstacles and potential dangers that must be carefully addressed, the main concern include:

Data and Algorithmic Challenges

AI systems trained on biased datasets can preserve and amplify existing healthcare disparities. These biases can lead to substandard clinical decisions and worsen longstanding inequalities in healthcare outcomes among different demographic groups. This problem is compounded by AI trained on unstructured or biased data that might generate misleading results.

Patient Safety and Medical Errors

The most immediate concern involves direct patient harm. AI system errors pose risks of injuries to patients. Healthcare providers face the additional challenge of automation bias, where humans become overly reliant on AI systems despite their cognitive limitations, potentially leading to critical oversights in patient care.

Privacy and Data Protection

The digital transformation brings unprecedented risks to patient confidentiality. AI generates vast amounts of sensitive patient data, creating data privacy and security risks. More concerning, AI also magnifies existing cyber-security risks, potentially threatening patient privacy and confidentiality.

Implementation and Adoption Barriers

The path from laboratory to clinic presents multiple obstacles. Translating AI systems into healthcare include issues inherent to machine learning, logistical implementation hurdles, and adoption barriers. Healthcare providers struggle with concerns about data quality, bias, privacy, and accuracy of models, while many institutions lack the technical expertise needed for proper implementation.

The Regulatory Challenge

While over 1,000 AI/ML-enabled medical devices and technologies have been authorized by the FDA as of early 2025, regulatory agencies struggle to keep pace with technological advancement. The rapid deployment often outpaces comprehensive safety testing and standardization efforts.

In summary – while AI promises to revolutionize healthcare, its implementation faces significant obstacles that could undermine patient safety and healthcare equity. Success requires addressing these challenges proactively through improved data governance, bias mitigation strategies, enhanced cybersecurity measures, and comprehensive regulatory frameworks that balance innovation with patient safety.

The Israeli Perspective

Israel has emerged as a global leader in AI-powered healthcare innovation, with companies developing cutting-edge solutions that directly address many of the hurdles and risks discussed above. Here are five examples of how Israeli AI medical companies are tackling these challenges:

Aidoc – AI-Powered Medical Imaging for Critical Care: Aidoc is helping medical centers alert imaging technicians with AI-based insights of possible bleeding in the brain and other critical conditions in a patient’s scan within minutes. This directly addresses the challenge of integrating AI into clinical workflows without disrupting existing practices.

Ibex – AI-Powered Diagnostic Solutions: Ibex has led the way in AI-powered diagnostics for pathology, helping pathologists ensure better cancer care for patients around the world.  Developed by pathologists for pathologists, Ibex solutions
serve the world’s leading physicians, healthcare organizations and diagnostic providers.

MDClone – Revolutionary Privacy-Preserving Analytics: MDClone offers a new healthcare data paradigm, enabling fast and direct access to healthcare data while fully protecting patient’s privacy. The company addresses one of the most critical challenges in medical AI: accessing valuable healthcare data without compromising patient confidentiality.

CytoReason – AI-Driven Drug Development: CytoReason, an Israeli startup that uses artificial intelligence to develop computational disease models for drug discovery, secured funding backed by US chipmaker Nvidia and pharma giant Pfizer. This addresses the challenge of developing more effective and representative medical treatments.

Rhino Health – Privacy-Preserving Collaborative AI: Rhino Health is using federated learning to make AI development more collaborative while maintaining privacy. This innovative approach addresses both data sharing limitations and privacy concerns.

These examples represent only a small portion of Israel’s robust medical AI sector. Collectively, they and many other Israeli companies illustrate that the significant theoretical challenges facing medical AI can be effectively addressed through innovative design approaches and close alignment with actual clinical needs and workflows.

The post The AI Prescription: Separating Promise from Performance in Drug Development appeared first on ADRES biopharma regulatory consulting.

The Minovia Therapeutics SPAC deal, announced June 25, 2025, which values the Haifa-based company at $180 million pre-money, targets rare mitochondrial diseases and age-related decline. Its leadership is connected with Israel’s premier research institutions, including the Hebrew University, the Weizmann Institute, the Israel Institute for Biological Research, and Sheba Medical Center.

The $101 million+ XPRIZE Healthspan challenges competitors to develop therapeutic treatments that significantly extend healthy lifespan by 10 or 20 years by restoring muscle, cognitive, and immune function in individuals aged 65-80, within a treatment period of one year. This unprecedented prize pool has been assembled to incentivize the clinical development of highly robust aging interventions on actionable timelines.

ARPA-H’s PROSPR program in the US seeks contract parties to develop an Intrinsic Capacity composite score measurable at home that predicts long-term health outcomes based on physiological and biochemical measures, and repurposed or new drugs that increase healthspan. Unlike traditional disease-focused initiatives, PROSPR explicitly targets the pre-disease state, where geroscience research suggests the maximum impact for increasing healthspan.

This emerging sector, projected to become a trillion-dollar industry, faces a critical challenge: regulatory frameworks designed for traditional disease-specific therapeutics are not well-equipped to evaluate products targeting mechanisms in the biology of aging.

The Geroscience Revolution

Geroscience – the interdisciplinary field examining the intersection between aging biology and chronic diseases and health – has revealed that aging is not merely an inevitable decline but a malleable biological process. Evidence has been accumulating at an accelerating pace that targeting fundamental aging mechanisms may be able to simultaneously influence multiple age-related conditions, including cardiovascular disease, cancer, neurodegeneration, and metabolic disorders.

This scientific paradigm shift challenges the traditional “one drug, one disease” model that has dominated allopathic pharmaceutical development for decades. Instead of treating individual chronic diseases after they manifest, healthspan therapeutics aim to address their common underlying driver: aging itself.

Regulatory Roadblocks

The regulatory challenges facing healthspan therapeutics are structural and cultural. If a company today approaches the FDA claiming to have developed a pill that “cures aging,” this is unevaluable under current regulations.

Aging is not classified as a disease by regulatory agencies. FDA typically approves treatments for specific diseases in defined patient populations. Healthspan products target multiple chronic diseases across heterogeneous populations.

Moreover, the FDA typically requires a demonstration of clinical benefit in how patients ‘feel, function, or survive.’ This a fortiori limits evaluation of interventions to symptomatic disease states, and creates barriers for upstream interventions targeting molecular or cellular changes in pre-disease states – precisely where geroscience research suggests the greatest impact lies.

Further, vaccines, statins and contraceptives are exceptions that prove the rule, that FDA is not culturally well-equipped to evaluate a preventive therapeutic intervention for healthier, younger people who are not yet patients, and don’t want to be.

And, the timeframes required to demonstrate improved healthspan or longevity in human studies can span decades, creating practical and financial barriers that the private sector typically does not have the ability or stomach to overcome.

Clinical Development Complexities

Beyond regulatory hurdles, healthspan therapeutics face unique clinical development challenges. Traditional clinical trial designs, optimized for acute treatments in symptomatic patients, are poorly suited for interventions targeting healthy aging.

Biomarker validation represents a critical bottleneck. While researchers have identified numerous molecular signatures of aging, translating these into acceptable surrogate endpoints for regulatory development remains a multi-year or multi-decade challenge. The field lacks standardized, validated biomarkers that regulatory agencies will accept as meaningful indicators of healthspan improvement.

Patient recruitment poses additional complexity. Unlike traditional disease-focused trials that enroll patients with clear symptoms, healthspan studies must identify and enroll healthy individuals at risk for age-related decline – a population that may lack motivation or urgency to participate in lengthy clinical trials.

The heterogeneity of aging also presents statistical challenges. Individual variations in genetic background, lifestyle factors, and environmental exposures create substantial noise in clinical outcomes, requiring larger study populations and longer observation periods than traditional trials.

Looking Ahead

The THRIVE Act,* developed by the Kitalys Institute (www.kitalys.org), exemplifies a proposed solution to the regulatory challenge. This draft legislation would establish an optional regulatory pathway, escalating evidentiary requirements and incentives for therapeutic interventions, devices, and supplements targeting healthy longevity. A THRIVE Act version 2.0, incorporating feedback from multiple stakeholders, is now available at www.kitalys.org.

The convergence of advancing geroscience, increasing commercial interest, boosted by GLP-1 drugs, and healthy longevity entering mainstream conversations is driving healthspan products from experimental to mainstream medicine. As the field matures, we can expect standardized biomarkers, novel clinical trial designs, evolving regulatory, reimbursement, and other policies, a multitude of different approaches to increasing healthspan,** and the continuing explosion in longevity medicine. Regulation and policy can accelerate or delay the transition from “sick-care” to precision health, but forward-thinking companies will look for opportunities to get ‘ahead of the curve’ in this inevitable paradigm shift.

* The draft THRIVE Act proposes an optional regulatory pathway for healthspan products (those that can prevent, delay, reduce the risk of, or reverse chronic diseases of aging), with three tiers of escalating evidentiary requirements, and a period of market exclusivity per tier, to incentivize the generation of clinical evidence for extending healthspan. Tier 3 is analogous to full FDA approval, Tier 2 somewhat analogous to Accelerated Approval, and Tier 1 would afford qualifying products earlier access to the market than under current regulations, in order to generate increasingly rigorous clinical data on the capitalization of a commercial-stage company.

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Among the digital therapeutics evaluated to date, we can take the example of Moovcare®, which has accumulated more than a decade of clinical research across randomized trials and real-world studies in France, Spain, and the United States. Its approach is based on a simple principle: patients complete a weekly questionnaire, developed by physicians. Data are analyzed by an algorithm designed to detect early signs of relapses or complications. Notifications are then transmitted to a medical team, who reviews the information and, when necessary, contacts the patient. If a medical intervention is required, the oncologist is informed so that appropriate action can be taken before the patient’s condition deteriorates. It has already been used by thousands of patients. This approach complements conventional imaging and routine visits, supporting more timely clinical management.

Studies have shown an increase in overall survival of 7.6 months compared with standard follow-up, alongside improved quality of life, fewer hospitalizations, and adherence rates above 90%. Economic analyses also point to reductions of more than 10% in annual hospitalization costs. These results have been presented at international oncology congresses such as ASCO and published in leading peer-reviewed journals including JNCI, JTO, and JAMA.

In 2025, Moovcare® obtained CE Mark certification under the European Regulation (EU) 2017/745 for Moovcare® Toxicity, designed to monitor treatment-related toxicities across all cancer types. At the same time, Moovcare® Lung renewed its CE certification. This dual recognition reflects both regulatory compliance and the maturity of clinical validation achieved by the solutions.

Clinical practice feedback aligns with the trial results. Physicians report that this tool offers a structured approach to capturing patient-reported outcomes and supports earlier therapeutic decision-making. Patients describe the tool as easy to use and reassuring, with consistently high satisfaction across studies. Integration into clinical workflows has been feasible without compromising safety or oversight.

The broader lesson is clear: digital health in oncology can deliver real value. Long-term evaluation, regulatory approval, and integration into care pathways are critical for adoption at scale.

Moovcare® illustrates this pathway from concept to validated digital therapeutic. It is now entering a phase of international expansion. Its trajectory highlights how digital monitoring, grounded in robust evidence, can contribute to earlier relapse detection, better quality of life, and more efficient use of healthcare resources in cancer care.

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One major concern is the use of outdated or inaccurate content. As is probably already known, AI models are typically trained on historical data and may not reflect the most current versions of regulatory guidelines. For example, if I ask the AI about ICH Q2, it might reference an earlier draft or fail to incorporate recent Q&A updates. For example, when I used ChatGPT for a specific question relating to ICH Q2 (Validation of analytical procedures), even when I used its agent “WEB pilot”, which should access the most recent versions on the Internet, the answer I got was incorrect since it was based on version 1 instead of version 2 of the guideline. The risk is even higher for region-specific guidance – AI may inadvertently cite FDA expectations for an EMA dossier or vice versa. To mitigate this, I always verify outputs directly against official sources like EMA and FDA guidelines.

Another issue is source transparency. AI models can provide generalized summaries without clear citations or links to source documents. This lack of traceability is problematic in a regulated context, especially when we need to justify decisions during inspections or in Module 3 submissions. I’ve learned to treat AI outputs as pointers, not definitive sources, and as mentioned above, I always follow up by locating the original document myself.

There’s also the risk of hallucinated references-fabricated documents or clauses that sound convincing. These are dangerous in any regulatory context. I’ve seen AI create entirely fictional guidance sections or merge unrelated guidelines into one. As above, cross-referencing with validated regulatory databases (like RAPS, Cortellis, or agency portals) or relevant guidelines (EMA, FDA, ICH) helps ensure credibility. Additionally, I have noticed that some AI tools are less ‘hallucinating’ than others. E.g., Perplexity AI is usually more accurate. Alternatively, specific agents in ChatGPT (e.g., ‘ChatGMP’ and ‘Medical Device Regulatory Advisor’) are also usually more accurate.

A less obvious but serious risk is, of course, data protection. If I’m consulting on proprietary projects and use cloud-based AI tools that aren’t validated or private, there’s potential exposure of confidential sponsor data. This could breach both GDPR and confidentiality agreements. My solution: I never put sensitive information into unsecured platforms and additionally use enterprise-grade tools with appropriate data controls.

Last but not least, AI lacks regulatory-CMC nuance and the required expertise. It may present guidance without considering the clinical stage, product class, or regional nuances. If a prompt is developed by someone with no CMC expertise, it may lead to misleading, incomplete, or irrelevant outputs. Clear, context-specific questions-framed with an understanding of clinical stage, region, and technical nuance-are essential to guide the AI toward accurate and actionable responses. That’s where human expertise remains crucial.

In summary, AI is a valuable assistant-but not a substitute for CMC judgment. By combining AI’s efficiency with rigorous validation and oversight, we can use it responsibly and effectively in CMC consulting.

The post Using AI for Regulatory Referencing in CMC Consulting: Risks and Mitigations appeared first on ADRES biopharma regulatory consulting.

Today, more advanced approaches are emerging. There are now emerging AI platforms that, by analyzing RNA expression by digital pathology images alone can identify treatment options for more than 90% of patients. Compared with DNA-only testing, which requires weeks to obtain a result and costs thousands of dollars, these newer methods deliver results in just days and at a fraction of the cost. The impact is significant: more patients receiving effective treatments, quicker therapeutic decisions, and fewer wasted efforts on ineffective therapies.

Yet diagnostics are only the beginning. The real transformation will occur when these AI technologies are integrated into top-tier end-to-end AI-based care continuum ecosystems. These systems do more than just generate a test result. They can help guide patients and providers through the entire journey: identifying evidence-based treatment options, alerting patients and clinicians when therapies fail, matching individuals to clinical trials, securing access to off-label or compassionate-use drugs, and navigating the complexities of insurance and prior authorization. Patients will be supported not only with clinical intelligence but also with practical tools such as reminders, financial aid guidance, symptom tracking, and live chat support—to ensure continuity of care and the best possible patient outcomes.

This integrated approach establishes a powerful feedback loop: every patient outcome feeds back into the system, enhancing its predictive precision and continuously improving treatment matching. As data accumulates across thousands of patient journeys, it evolves into a dynamic engine—driving stronger scientific insights, accelerating clinical trial recruitment, and enabling more efficient payer decision-making.

However, technology alone is not enough. If technology integration is one side of the coin, sustainable reimbursement is the other. Without the right reimbursement model(s), even the most effective solutions risk underutilization. Current frameworks often fragment or silo coverage into individual services—a test, a consult, a lab fee—without recognizing the full clinical and economic value of coordinated AI-enabled solutions. This approach risks undermining the adoption of technologies aimed at improving outcomes and reduce costs across the entire journey.

What is required is a solution-based reimbursement model that spans the full continuum of care.  This means aligning payment with the total value delivered across the continuum, from diagnostic insights to treatment navigation, outcomes tracking, trial access, etc. Such models may include establishment of CPT codes for AI-enabled testing platforms, bundled payments that capture the full scope of services, and value-based pilots that tie reimbursement directly to outcomes such as survival, adherence, reduced hospitalizations.  Early payer engagement (health plans, health systems, employers, etc.) and evidence generation will be critical components of a successful strategy.

AI has demonstrated its ability to revolutionize cancer testing by providing faster, more affordable, and more comprehensive results than ever before. Its integration into patient navigation, trial enrollment, and insurance support ensures that insights are put into action. The final step is ensuring reimbursement keeps pace with this new reality. By funding solutions rather than silos, healthcare systems can realize the full potential of AI in oncology: more patients receive the right therapy the first time, resources are used more efficiently, and outcomes are improved on a larger scale.

The post From Insight to Access: How Integrated AI Solutions and Smarter Reimbursement Can Redefine Cancer Care appeared first on ADRES biopharma regulatory consulting.

And he’s been going for quite some time. A longtime entrepreneur with a background in health and science, Eran founded Immunity Pharma around a promising scientific discovery: a peptide that showed an unusual effect—it helped protect animals from radiation damage, even when administered post-exposure. “It wasn’t preventative. The primary damage had already happened,” he explained. “And it still worked, most of animals recovered. That’s when I knew we might have a true drug.”

The peptide was first developed by Professor Irun Cohen at the Weizmann Institute, who originally named it stressin,a nod to its effects on cellular stress. Eran quickly saw potential with much wider applications. He joined forces with Cohen and began exploring how the compound might be used to treat neurodegenerative diseases, which are often tied to cellular stress pathways.

After reviewing several disease candidates, the team chose to pursue the science in treating ALS, securing early preclinical funding from the Israeli ALS Association, IsrALS. Initial animal studies showed encouraging results, prompting the formal founding of Immunity Pharma. “We had a few options in parallel,” Eran said. “But the signal in ALS was strong, so we focused on it.”

IPL344 is now part of a family of peptides developed to activate the same stress-response mechanism. While ALS is the initial focus, the potential applications are extensive. “Today it is a platform,” Eran said. “We believe it could support a pipeline of therapies targeting other age-related diseases.”

Why ALS?

Amyotrophic lateral sclerosis (ALS) is a rare but devastating neurological disorder which causes progressive degeneration of motor neurons, leading to muscle weakness, difficulty speaking and swallowing, and eventually, respiratory failure. ALS is a highly challenging disease with a grim prognosis—the majority of ALS patients survive only two to five years after diagnosis, although life expectancy can vary greatly.

For a company like Immunity Pharma, ALS presented both a scientific challenge and an opportunity. “An advantage of focusing on ALS is that it’s one of the most aggressive diseases related to cellular stress,” Eran told me. “If it works there, we’ll know. And if it doesn’t, we’ll know that too. We won’t waste time and resources on futile dreams.”

That commitment to clarity shaped everything about Immunity’s lead candidate, IPL344. Most approved ALS therapies address symptoms or target a single process, such as oxidative stress or inflammation. IPL344 is designed to address six disease-driving mechanisms simultaneously, a distinction Eran believes is key.

The drug is a novel peptide designed to reactivate the AKT pathway, a crucial cellular signaling mechanism that supports neuron survival, reduces oxidative stress, and maintains metabolic stability. In ALS, this cellular pathway is often downregulated, and many drugs that attempt to stimulate it via cell membrane receptors fail because those receptors are themselves impaired.

Immunity Pharma’s approach is different. IPL344 bypasses the usual membrane receptor mediated pathways, instead entering the cell and activating a stress response that reawakens the AKT pathway from within. It’s a workaround, or as Eran put it, “a back door into the pro- survival system of the cell.”

That mechanism matters because ALS is a multi-pathology disease. “You’re not dealing with one problem,” Eran said. “You’re dealing with apoptosis, oxidative stress, misfolded proteins, disrupted metabolism, DNA damage, even immune dysfunction.” IPL344 addresses most of these, he believes, by unlocking AKT and, with it, the cell’s self-repair machinery.

Early results seem to back him up. In a Phase 1/2a open-label study, IPL344 was administered intravenously once daily, and preliminary data suggest about a 60% slower rate of disease progression in people living with ALS who received it, a rare, perhaps even unique, result. Additionally, the treatment was well tolerated and easily administered at home.

“We treated ten patients,” Eran said, “and we saw disease slow down in nine of them. Some were small, most of them significant, but there was a clear effect in multipledisease parameters.”

So, with these initial encouraging results, what are the next steps for the company?

“We’re now preparing for a large, pivotal trial that we expect to lead to drug approval,” Eran told me. But that next step is no small feat. “The science is there. The mechanism is solid. The clinical signs are encouraging. But bridging to the next phase takes enormous resources.”

Eran stressed that the company couldn’t have gotten to where it is today without the close partnership of scientists Prof. Irun Cohen, Dr. Ilana Cohen, and Dr. Oren Becker who cofounded the company with Eran, and the angels Rony Pfeifer, Gideon Stein and Yigal Tamir who joined him soon after, creating a small professional community around this endeavor. This core group has since expanded with dedicated clinical, scientific, professional, and business partners who have a shared vision and align with the business goals, maintaining long-term involvement in the company.

The Funding Journey

Like many early-stage biotech CEOs, Eran needs to spend a lot of his time and effort on fundraising. After good success with private investors, Immunity is now looking to include institutional partners to bring IPL344 to the finish line. That leap, from private to institutional backing, is particularly tough in Israel. “In the U.S., institutional investors are used to this stage. Here, it’s much harder to bring them in unless you are already past the finish line.”

If there’s a message here for other health tech founders, it might be this: storytelling matters. “Since I started the company, I’ve rewritten our presentation 200 times,” Eran said. While the science evolved gradually, the way he communicates it must constantly adapt to shifting business and scientific narratives. “You need to keep refining how you communicate the value of what you’re doing, maintaining a consistent core while demonstrating clear progress. That’s how you bridge the trust gap.”

Despite those hurdles, Eran stays focused. “This is a marathon, and we’re in the last ten kilometers,” he said. What helps him endure? A combination of incremental wins and belief in the mission of helping stop the progress of dreaded neurodegenerative diseases.   

As Immunity Pharma prepares for the next stage, Eran is clear-eyed about what lies ahead. “In five years, I hope we will have a drug on the market. I hope we’ve expanded the platform to treat other neurodegenerative diseases. But most of all, I hope we’ve helped people.”

It’s that final point—the patients—that keeps coming back in our conversation. “I know this drug helped the people who took it. I saw it,” he said. “Now we need to prove it, at scale. That’s the mission.”

Immunity Pharma began collaborating with ADRES.bio in 2012, advancing the company’s groundbreaking work to develop a treatment for ALS. Early in the partnership, ADRES.bio supported the production of the first manufacturing batches, enabling compassionate use treatment for a patient whose life was extended by 18 months. Over the years, the two teams have worked closely to navigate the complex regulatory path toward approval, sharing in the progress and discoveries that continue to reveal the therapeutic potential of Immunity Pharma’s product.

The post The AKT Awakening: Immunity Pharma’s Novel Approach to ALS and Neurodegenerative Disease appeared first on ADRES biopharma regulatory consulting.

 That’s where BDUK Therapeutics, an Israeli biotech startup poised to redefine TACE, comes into the picture. Co-founders Walter Wasser, MD and Yaron Suissa, PhD, MBA, contend that the fundamental flaw isn’t in the concept of targeting tumors by disrupting their blood supply, but rather in current methods that completely occlude vessels, inadvertently preventing the very chemotherapy they aim to deliver from reaching its cancerous target.

Their answer is EmboPore, a novel porous microsphere-based delivery technology developed by Professor Ofra Benny at Hebrew University, which is designed to maintain partial blood flow, allowing chemotherapy to be delivered directly to tumors over a sustained two-week period. Recent advancements in the field, showing improved progression-free survival when TACE is combined with targeted and immunotherapy drugs, further highlight the urgent demand for more effective TACE platforms—a need BDUK Therapeutics is uniquely positioned to address.

With approximately 900,000 new hepatocellular carcinoma diagnoses globally each year, and the TACE market alone valued at $11.8 billion in 2024 and projected to reach $16.4 billion by 2031, the moment for this innovation could not be more opportune. We recently sat down with BDUK co-founders Walter and Yaron to learn more about their approach to this immense challenge.

How did the idea for BDUK Therapeutics come together, and what made this particular approach to liver cancer stand out to you?

Walter: Yaron and I had been exploring multiple biotech opportunities, looking for something that addressed a major clinical gap. As a physician and a scientist, we were drawn to projects that could make a real difference.

After reviewing around ten technologies, we found Professor Benny’s work on porous microspheres designed to treat liver cancer especially compelling. The science was strong, the need was urgent, and it was clear this product  had the potential to change the standard of care. That’s when we decided to build BDUK Therapeutics around it.

Liver cancer accounts for more than 700,000 deaths annually worldwide, and TACE remains the standard of care for many patients. Why does TACE continue to fall short, and how does that shape the opportunity for innovation?

Walter: Liver cancer is a major global health challenge. The Lancet Commission’s July 2025 report on global hepatocellular carcinoma has already garnered significant attention, identifying liver cancer as the sixth most common cancer worldwide and third leading cause for cancer-related mortality globally. The commission projects new liver cancer cases will nearly double from 870,000 in 2022 to 1.52 million by 2050.

There are 60,000 to 70,000 new cases in the U.S. and Europe each year, with only 40% of these individuals are eligible for curative surgery. Another 20% have advanced disease with no treatment options. This leaves 40% of patients to be treated with TACE, transarterial chemoembolization.

TACE involves injecting beads mixed with chemotherapy into the hepatic artery to block blood flow and target the tumor. In theory, this starves the cancer and delivers the drug directly. But in practice, the occlusion is so complete that very little of the chemotherapy reaches the tumor. That’s the fundamental flaw.

As a result, outcomes remain deeply concerning. Nearly half of the patients who receive TACE die within two years of diagnosis, and only about one in five survive beyond five years. These are not just statistics, they reflect the limitations of current therapies and the urgent need for better options.

Your solution, EmboPore, was designed to overcome the core limitations of conventional TACE, namely, poor drug delivery and irreversible vessel occlusion. Can you explain how it works, and why it represents such a significant advance over the current standard of care?

Walter: EmboPore fundamentally reimagines how locoregional therapy is delivered. Traditional TACE uses embolic beads to block the hepatic artery and starve the tumor, but the complete occlusion means that very little of the chemotherapy actually reaches the cancer. That’s the root problem.

What Professor Benny developed is a porous microsphere that allows partial blood flow to continue, enabling embedded drugs —Doxorubicin and Tirapazamine (TPZ)—to reach the tumor over an extended two-week period. These microspheres are made of PLGA, a biodegradable polymer that rapidly breaks down in acidic environments, which are the conditions typically found in tumors. After an initial rapid degradation, the microspheres degrade in a gradual manner. This is a controlled degradation that releases the two drugs gradually and directly into the tumor microenvironment.

There is also a synergy between the two drugs: doxorubicin is a standard chemotherapeutic agent, while TPZ is only activated by hypoxia. The microspheres themselves induce localized hypoxia, activating TPZ which together with DOX provide the synergistic effect. Because EmboPore eventually dissolves, repeat treatments through the same arterial portal become a possibility—something conventional TACE doesn’t allow. The inability the catharize the same hepatic artery cause a significant negative impact on liver burden and patients’ capability to repeat the procedure.[RZ1] 

You can think of it like a drug-eluting stent, but for liver tumors: precise, sustained drug delivery at the site of disease with minimal systemic toxicity while enabling the required environment for optimal anti-tumor drug impact. In preclinical models, this resulted in significantly higher drug concentrations within tumors and liittle drug in the surrounding liver tissue.

From a pharma R&D and business development perspective, what makes this platform fundamentally different from other attempts to improve TACE?

Yaron: The key limitation in TACE is the complete blockage of blood vessels, which not only prevents drug penetration but also limits the potential for repeat dosing.. The field has optimized everything about the beads—size, material, drug binding—except the main problem: they completely occlude the vessel.

Our product solves that. The porous structure of the microsphere allows some blood to continue flowing through, improving drug exposure to the tumor and preserving vessel patency. It degrades faster in acidic environments like the tumor microenvironment, which further enhances drug release at the right time and place. It’s a delivery solution that works with the biology, not against it.

The TACE market is estimated at billions of dollars—how do you evaluate commercial potential and how does orphan drug designation factor into your strategy?

Yaron: The commercial potential is substantial. In 2023, the TACE market reached $11 billion globally, with over 1.3 million patients eligible for treatment. While the largest patient populations are in Asia, there are still 60,000 to 70,000 eligible patients each in the U.S. and Europe.

Given the patient population size for this condition, we anticipate qualifying for orphan drug designation in both the U.S. and Europe, which would unlock significant incentives such as market exclusivity, fee reductions, and development support.

We also believe we may be eligible to receive FDA breakthrough therapy designation (BTD), and PRIME status in Europe, which can accelerate regulatory review and potentially enable commercializing the drug before completing Phase 3 trials. From a business perspective, the market average for orphan drug ROI is compelling—even under conservative models the return is projected to reach 7x.

Additionally, we are currently observing a significant change in market trends. Whereas in the past startup interactions with big pharma were initiated only towards the end, or after completion of, a phase-2 clinical trial, it’s been reported that leading pharma companies are now investigating M&A deals much earlier, even after phase-1 studies with positive readout.

What’s your current funding timeline and how are you structuring the path to clinical trials?

Yaron: We’re in the process of raising funds to start Phase 1 over the next 18 months. That includes completing GLP-compliant rodent studies with secondary efficacy endpoints and initiating first-in-human trials. We’ve already started some preparatory work.

Interestingly, two of our three components, PLGA and Doxorubicin, are FDA approved, while the third, TPZ, is well known and previously tested. The vast existing safety data may allow us to expedite traditional preclinical steps and skip non-rodent toxicity studies or run them in parallel with the clinical study to save time.

While your lead indication is liver cancer, do you see broader applications for this platform beyond hepatocellular carcinoma (HCC)?

Yaron: Absolutely. While our initial focus is on primary liver cancer, EmboPore is equally relevant for secondary liver tumors—cancers that metastasize to the liver. These include colorectal, pancreatic, breast, and uveal melanoma, which often spread preferentially to the liver and are difficult to treat systemically.

The strength of EmboPore lies in its ability to deliver various chemotherapy locally, directly into the tumor’s blood supply, while preserving vessel patency. This allows for sustained drug exposure, reduced systemic toxicity, and the possibility of repeat treatment, thereby overcoming major limitations of conventional TACE.

Looking ahead, we also see potential applications in other solid tumors beyond the liver. Any cancer accessible through the arterial system—like kidney, prostate, or even brain tumors such as glioblastoma—could benefit from a targeted, locoregional drug delivery approach. While we’re not currently targeting non-oncology indications, the underlying technology may also have future relevance in areas like arterial inflammation or vascular diseases.

You’ve received some strong early interest from key clinical opinion leaders. How is that helping to shape your path forward?

Yaron: It’s been a strong indicator that we’re solving the right problem. Usually, it takes months to get KOLs to engage meaningfully, but we’ve had experts in the field want to get involved almost immediately.

We had a leading senior hepatologist interested in collaboration after presenting our supporting scientific data. We’ve also had interest from a translational oncology expert at a leading cancer center in New York as well as leading senior interventional radiologists both in Israel and the US. That kind of engagement, from individuals with deep experience in liver-directed therapies, confirms that we’re addressing a significant clinical gap.

We are confident that our mission to transform the treatment paradigm for individuals with intermediate-stage liver cancer will have far-reaching implications for patients worldwide, offering new hope and meaningful advances in care.

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For BDUK Therapeutics Ltd., the path ahead means proving their porous microsphere technology can succeed where standard TACE has failed, and clinical trials will provide the answer. With high liver cancer mortality rates and a clear market waiting, BDUK Therapeutics offers both hope for patients and potentially a blueprint for how academic research can be translated into life-saving therapies.

ADRES Bio is actively supporting BDUK Therapeutics on their regulatory path, including with CDMO selection, CMC activities, preclinical plans, grants, investor meetings, and regulatory discussions.

The post Beyond Vessel Occlusion. A Porous-Microsphere Approach to Reinventing TACE in HCC appeared first on ADRES biopharma regulatory consulting.

Israel works because three pieces line up. The Ministry of Health applies a risk-based review that allows sensible deferrals when patient safety is unchanged. The national HMOs provide digitized longitudinal records that support cohort pre-verification before you open recruitment. The sites are experienced with focused, early studies that prioritize clean execution over scale. Put together, you move from award to first visit in weeks rather than quarters, and you do not trade quality for speed.

Founders struggle most with timelines that drift.

In Israel, the path is clearer because key steps run in parallel. While your team finalizes the protocol and localizes documents, the MoH review can begin with a tight risk file and plain-English rationale for each change. In the same window, sites prepare logistics and query HMO data against your inclusion rules to identify likely matches. By the time approval lands, you are not starting from zero. You are ready to consent and screen with confidence that the pool exists.

Recruitment is the decisive edge.

Pre-verification against real records reduces screen failure, shortens the ramp, and narrows variance around your enrollment forecast. You know whether the cohort is there, where they receive care, and how to structure outreach that respects privacy and consent. This is not a shortcut. It is disciplined feasibility that removes guesswork before you spend on monitors, pharmacy, imaging, and idle site weeks.

Costs follow the same logic.

When cycle slips disappear, paid idle time drops. When you concentrate work in a few capable centers with proven access to the target population, you avoid the long tail of under-performing sites that enroll one patient each. Local rates for monitoring and select vendors are often lower than U.S. equivalents, which compounds the savings for Phase 1, 1b, and device feasibility programs. The important part is to capture these gains in contracts up front and to hold vendors to milestones that reflect the parallel work model.

Many teams now design patient-cohort add-ons that sit between a classic healthy-volunteer start and a larger Phase 2. The aim is an earlier signal in the target disease with biomarkers that de-risk dose, schedule, or device parameters. Keep these cohorts tight. Limit endpoints to those that change decisions. Blend EHR pulls for routine measures with focused study visits for what cannot be captured in care. You get sharper data without bloating the protocol.

Quality and oversight remain non-negotiable.

A monitoring plan should reflect actual risk and the specifics of your modality. Centralized review for safety labs, imaging, or device telemetry reduces variability and speeds detection of issues. Many operators run a brief enrollment pause after the first few patients to validate the consent flow, assess sample handling, and confirm endpoint capture. Small fixes at this stage prevent expensive rework later.

Data governance needs the same attention as science.

Work through HMO governance channels early. Start with de-identified feasibility queries to confirm that your criteria map to a real population. Align consent language so patients understand how routine care data link to study data, and so transfers remain minimal and auditable. If you keep data moves narrow and transparent, you protect patients and stay inspection-ready.

Israel is not a universal fit. If your indication is ultra-rare with minimal local prevalence, recruitment will not meet expectations. If your device requires complex hardware that is not available locally, setup time can erase the advantage. If your internal team cannot meet weekly decision cadence, the benefit of parallelism fades. In these cases, adapt the plan or select a different path rather than forcing a model that does not match your constraints.

A hybrid model often delivers the best of both worlds. Run a lean Israeli engine to reach first-patient data and pair it with one or two U.S. sites for KOL engagement and downstream regulatory alignment. Keep the protocol identical and the data systems unified to avoid splitting the study by accident. This structure supports both speed and credibility with partners who expect U.S. touchpoints.

If you need a framing for your next decision meeting, use five questions:

• What single decision must this study inform, and by what date?
• Does HMO feasibility show enough candidates under your criteria?
• Which elements of the submission can be safely deferred without changing patient risk?
• Where exactly will savings come from, and how will contracts lock them in?
• Which partners already have live HMO connections and MoH experience for your modality?

Clear answers to these questions prevent drift and protect milestones tied to your financing.

The recent webinar explored this operating model with voices from sites, HMOs, and startup clinical leaders who have delivered on these timelines. It was not a recap of guidelines. It was a working playbook with concrete examples of approvals, startup flow, and cohort management. If you want color on the mechanics or vendor choices, the session is a good companion to this article.

In short, Israel gives biomed startups a credible route to earlier human data with tighter timelines, faster recruitment, and thoughtful cost control. Plan parallel work, verify cohorts before you open, and keep quality measures precise. If you want to test whether this model fits your program, watch the webinar and then reach out with your specific constraints.

For questions or a quick fit check – contact us

The post A Faster, Leaner Path to First-Patient Data: Why Biomed Startups Choose Israel for Early Clinical Work appeared first on ADRES biopharma regulatory consulting.

Why Inspection Readiness Matters

For companies submitting BLA, NDA, or MAA applications, inspection outcomes often determine whether those filings will proceed smoothly or hit costly roadblocks. Regulatory authorities don’t just evaluate your data. They examine the integrity of your systems, the preparedness of your staff, and the reliability of your vendors. That’s why inspection readiness must be treated as an ongoing organizational capability, not a last-minute fire drill.

From Reactive to Proactive: What It Means to Be Inspection Ready

Being inspection ready means your systems, documentation, and people can withstand regulatory scrutiny at any time. It’s not just about compliance. It’s about creating a culture of quality aligned with global standards like ICH Q10, ICH E6(R2), 21 CFR Part 11, and EU GMP Annex 1 and 11.

This involves more than keeping documents in order. It means ensuring that clinical and manufacturing data are traceable, vendors meet your standards, and your team knows how to engage with regulators when the moment comes.

Key Steps to Building an Inspection Readiness Plan

A structured readiness strategy focuses on six core areas:

1. Closing the Gaps Before Inspectors Find Them

Start with a comprehensive gap analysis. Review processes across clinical operations, GMP manufacturing, data systems, and your Quality Management System (QMS). Use a risk-based approach in line with ICH Q9 to prioritize and remediate weaknesses.

This early assessment not only prevents regulatory surprises but also provides clarity on where to invest your resources for the biggest compliance impact.

2. Simulating the Real Thing with Mock Inspections

Nothing prepares teams better than a live drill. Mock inspections, ideally led by former FDA or EMA inspectors, simulate real-world audits. These exercises test everything from document access and SOP walkthroughs to how well your staff communicates with regulators.

A regulatory audit simulation reveals practical issues, such as poor documentation retrieval or inconsistent answers, that can derail an actual inspection.

3. Ensuring Data Integrity Through Source Data Verification

During inspections, regulators will trace reported results back to their original source documents—from batch records and lab notebooks to CRFs and electronic audit trails. That’s where Source Data Verification (SDV) comes in.

Using frameworks like ALCOA+ (Attributable, Legible, Contemporaneous, Original, Accurate, Complete, Consistent, Enduring, Available), SDV confirms that your data is credible, consistent, and submission-ready.

Lack of traceability is a frequent trigger for Form FDA 483 observations or even Complete Response Letters (CRLs). With SDV, you strengthen your inspection resilience and your case for approval.

4. Training Teams for Real Inspection Scenarios

You can’t fake readiness. Inspectors quickly sense whether your team understands GCP, GMP, and your internal SOPs. That’s why ongoing training is essential.

Go beyond basics. Ensure your staff knows how to manage deviations, document properly, and interact professionally with inspectors. Keep training logs current and retrain as systems or regulations evolve.

5. Oversight Beyond Your Walls: Managing Suppliers and CMOs

In the eyes of regulators, you are fully responsible for your third parties. That includes CMOs, CDMOs, CROs, and testing labs.

Your inspection readiness hinges on their compliance too. You’ll need robust Quality Agreements, vendor audits, and documented supplier qualifications that align with ICH Q10. Regulators will ask to see your vendor ratings, CAPA records, and oversight plans.

Any failure at the vendor level can jeopardize your submission. Don’t assume compliance. Demonstrate it.

6. Applying Risk-Based Vendor Qualification

Use tools like FMEA and heat maps to assess and prioritize vendor risks. This helps decide who to audit and when. It also documents rational oversight strategy regulators expect to see.

How Inspection Readiness Supports Submission Success

Most inspections are scheduled during the review window for submissions. If your site or supplier fails during this time, the consequences are immediate—delays, rejections, and lost opportunities for expedited pathways like FDA’s RTOR or EMA’s PRIME.

Investing in inspection readiness early ensures your application isn’t stalled by preventable issues.

What Documentation Must Be Inspection-Ready?

Inspectors typically request:

• Clinical Trial Records: Protocols, consent forms, CRFs, AE reports
• Manufacturing Documentation: Batch records, PPQ reports, trend analyses, stability logs
• Quality System Files: CAPAs, deviations, internal audits, SOP changes
• Third-Party Oversight Materials: Audit reports, Quality Agreements, vendor certifications
• Make sure all documents are easily accessible, traceable, and aligned with eCTD structures, particularly Modules 1.3 and 3.2.
• The Role of Expert Consultants
• Both FDA and EMA strongly recommend engaging GMP/GCP consultants, especially for novel therapies or emerging biotechs. They offer:
• Deep regulatory knowledge
• Independent assessments
• Customized training
• Direct support during inspections and audits
  

Working with a consultant ensures you aren’t navigating inspection risks alone. It signals to regulators that you’re serious about quality.

Why ADRES?

At ADRES, we provide integrated support across regulatory affairs, QA, and CMC to help life science companies meet inspection standards and accelerate approvals. Our offerings include:

End-to-end gap assessments

Mock inspections with ex-regulators

Source data verification and audit readiness

Training programs on GCP/GMP practices

Full-spectrum vendor qualification and monitoring

Partnering with ADRES reduces inspection-related delays, maintains data integrity, and ensures a smoother path to market.

The post How to Stay Ahead of Regulatory Expectations appeared first on ADRES biopharma regulatory consulting.

Clinical trials are inherently complex. Multiple stakeholders, systems, and regulatory expectations intersect, and this complexity increases the risk of inconsistency, delays, and compliance gaps. QA provides a structured, proactive way to manage these risks. It enforces process discipline and regulatory alignment across the entire trial lifecycle. Rather than being a final checklist, QA is a central pillar from day one.

Why QA Matters in Clinical Trials

At its core, Medical Quality Assurance ensures that clinical trials are conducted in a way that safeguards patients and produces reliable data. While Quality Control (QC) focuses on verifying outputs after they are generated, QA is about building sound processes that prevent problems from occurring in the first place.

This distinction has practical consequences. A well-functioning QA program lowers the likelihood of protocol deviations, data integrity problems, and regulatory challenges. When managed correctly, QA reduces rework, supports faster timelines, and keeps your trial on track for inspection readiness.

One area where QA shows measurable impact is protocol adherence. Regulatory inspections often highlight protocol deviations as a top issue. These deviations can usually be avoided through targeted QA measures, including early site oversight, clear staff training, and active tracking of quality metrics. Fewer deviations lead to better data and faster approvals.

The Regulatory Landscape for QA

QA in clinical trials must align with a wide array of national and international regulations. In the United States, the FDA provides key guidance through:

21 CFR Part 211, which outlines good manufacturing practices for finished pharmaceuticals

21 CFR Part 312, covering Investigational New Drug (IND) requirements

21 CFR Part 820, which governs quality systems for medical devices

21 CFR Parts 600 to 680, which apply specifically to biologics

In the European Union and globally, additional frameworks apply. These include:

Directive 2001/83/EC, which regulates medicinal products

EU MDR (2017/745) and IVDR (2017/746), which focus on medical devices and diagnostics

ICH Guidelines, including E6(R2), Q8, Q9, and Q10, which address good clinical practice, development principles, and pharmaceutical quality systems

ISO 13485 and ISO 14155, which govern medical device quality and clinical investigations

At ADRES, we continuously monitor regulatory changes and help sponsors build QA programs that stay in step with evolving global standards.

Responsibilities of Sponsors

Regulators expect sponsors to take full ownership of trial quality, not only for internal processes but also for outsourced activities. That includes work performed by CROs, laboratories, technology vendors, and other partners. To meet this expectation, sponsors must establish and maintain several core oversight practices:

Vendor qualification and selection based on objective, documented criteria

Defined roles and communication pathways through contracts and quality agreements

Routine performance evaluations, SOP reviews, and vendor audits

Corrective and Preventive Actions (CAPAs) that are tracked and verified for effectiveness

Data oversight guided by ALCOA+ principles and supported by risk-based monitoring

Validation of computerized systems, following 21 CFR Part 11 and EU Annex 11 requirements

QA helps sponsors demonstrate that they are in control, not just of their own processes but of the entire clinical trial ecosystem.

What an Effective QA Program Looks Like

A good QA program does not just satisfy compliance requirements. It supports operational efficiency and scientific integrity. Foundational elements typically include:

SOPs that reflect current regulatory expectations

Targeted training that clarifies roles and responsibilities

Risk-based quality management (RBQM) practices that prioritize oversight

Tracking of Key Quality Indicators (KQIs) and compliance metrics

Internal audits and Trial Master File (TMF) health checks

At ADRES, we focus on helping sponsors build QA frameworks that are flexible, effective, and grounded in everyday operations.

The Value of QA Audits

Quality Assurance audits offer a strategic advantage when designed well. They provide early visibility into risks and opportunities for improvement. Best practices for QA audits include:

A risk-focused audit plan that concentrates on critical areas such as informed consent, SAE reporting, and investigational product handling

Standardized checklists and workflows for consistent documentation

Interviews that verify procedural understanding among site and sponsor staff

CAPA documentation and follow-up assessments to ensure resolution

Our audit process at ADRES is built around practical value. We help clients move beyond box-checking to uncover real insights and implement meaningful improvements.

Supporting Patient Safety and Study Outcomes

At every stage, QA protects the integrity of your study and the safety of the patients involved. Key QA activities include:

Oversight of informed consent and inclusion criteria

Monitoring of Serious Adverse Event (SAE) reporting and follow-up

Verification of source data, dosing, and lab results

Readiness reviews to prepare for audits and inspections

These measures build trust, both internally and with regulators. They also help ensure that trial results are credible and reproducible. At ADRES, we specialize in helping sponsors establish QA programs that are practical, risk-based, and aligned with global requirements. Whether your team is preparing for a submission or managing a complex, multi-country study, a strong QA foundation supports better decisions and stronger outcomes throughout the clinical journey.

The post Building Quality from the Start: How Medical Quality Assurance Shapes Successful Clinical Trials appeared first on ADRES biopharma regulatory consulting.