We are joined by Ivan Kairatov, a biopharma expert with deep insights into the intricate world of drug research and development. In our conversation, we’ll explore the high-stakes landscape of regulatory approvals, touching on Bristol Myers Squibb’s pivotal new cancer therapy, the challenging path for a rare disease drug from Disc Medicine, a new competitive dynamic in the cardiovascular space, and the heated race to develop next-generation treatments for inflammatory bowel disease.
The FDA accepted Bristol Myers Squibb’s protein degrader, iberdomide, with an August 17 decision date. Given its success in achieving minimal residual disease negativity, what could this new drug class mean for treating advanced multiple myeloma, and how crucial is this approval for the company?
Protein degraders represent a fundamentally new approach, so this is incredibly exciting. For patients with advanced multiple myeloma, reaching a state of minimal residual disease negativity is a profound goal; it means the cancer is essentially undetectable. Seeing a drug in this new class achieve that in a Phase 3 trial is a significant step forward. It offers hope for a deeper, more durable response for people who have already been through several lines of treatment. For Bristol Myers Squibb, the stakes are sky-high. After a difficult 2025 marked by cost cuts and clinical disappointments, a win here is more than just a new product—it’s a critical morale booster and a validation of their pipeline strategy. An approval by that August deadline would feel like a powerful turnaround.
For Disc Medicine’s bitopertin, the FDA acknowledged its effect on protoporphyrin IX levels but rejected it over “uncertainties” about clinical benefit. What specific evidence from the Phase 3 study could bridge this gap for a traditional approval, and what does this decision signal about the FDA’s current standards?
This situation is a classic “biomarker versus clinical benefit” dilemma. The FDA saw the science—the drug clearly lowers protoporphyrin IX, which is the root cause of the light sensitivity in EPP. However, they weren’t convinced this translated into a tangible, real-world improvement for patients, specifically regarding the sunlight exposure endpoints. To bridge that gap, the Phase 3 data will need to be crystal clear. Disc Medicine must show a direct, statistically significant, and clinically meaningful link between that reduction in protoporphyrin IX and patients’ ability to tolerate sunlight without pain. This decision signals that the FDA is holding a very high bar, even for rare diseases. They are demanding that companies prove their drug doesn’t just change a number on a lab test but actually improves a patient’s quality of life in a measurable way.
Cytokinetics’ Myqorzo is now approved in the EU to compete with Bristol Myers’ Camzyos. How might Myqorzo’s more flexible dosing and lack of a genotyping requirement translate into a tangible market advantage, and what practical steps should the company take to capitalize on these differences?
These differences could be game-changers in a real-world clinical setting. For a physician, having more flexible dosing options makes it easier to tailor the treatment to an individual patient, which can improve both safety and efficacy. Eliminating the need for a specific genetic test before starting treatment removes a significant logistical barrier and cost. It streamlines the whole process of getting the drug to the patient who needs it. To capitalize on this, Cytokinetics needs to launch a very focused educational campaign aimed directly at cardiologists in the EU. They must clearly articulate how these practical advantages simplify patient management and could lead to better outcomes compared to Camzyos. This isn’t just about competing on efficacy; it’s about competing on convenience and ease of use, which can be a powerful driver of adoption.
Teva and Sanofi’s duvakitug showed improved remission rates at 44 weeks for ulcerative colitis and Crohn’s. In the competitive TL1A inhibitor class, where rivals are already in Phase 3, how significant is this long-term data, and what metrics will be most critical for this drug to stand out?
This long-term data is extremely significant. In chronic conditions like ulcerative colitis and Crohn’s, achieving remission is one thing, but maintaining it is the ultimate goal. Seeing remission and response rates actually improve between the 14-week and 44-week marks is a very strong signal of the drug’s durability. While competitors like Merck and Roche are further ahead in development, having compelling long-term efficacy data can be a powerful differentiator. To truly stand out, the most critical metrics will be the depth of response—things like endoscopic healing—and a pristine safety profile. In a crowded class, being not only effective but also demonstrably safe and durable over the long haul will be what convinces doctors and payers to choose their drug.
What is your forecast for the development and approval landscape for novel drug classes, such as protein degraders and TL1A inhibitors, over the next few years?
My forecast is one of cautious but definite optimism. We are entering an era of truly innovative science, and these new classes are a prime example. For protein degraders, I expect the first approvals will open the floodgates for a wave of new therapies targeting previously “undruggable” proteins in oncology and beyond. For the TL1A inhibitors, the competition will be fierce, but this will ultimately be a huge win for patients with inflammatory diseases, who will soon have multiple highly effective new options. However, the regulatory bar will continue to rise. Regulators, as we saw with bitopertin, will demand not just novel mechanisms but clear, compelling evidence of real-world clinical benefit and long-term safety. The companies that succeed will be those that can master both groundbreaking science and rigorous, patient-focused clinical development.
