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1rem

 Podcast, CEO and Founder of Sooth, Ian Baer, is back to discuss:

Why people trust friends and influencers over companies

Podcasts

Ian Baer, Founder and CEO of Sooth, discusses how the growing distrust in social media will impact industry marketing strategies and the relationships between pharmaceutical companies and the patients they aim to serve. He also explains dark social, how to combat misinformation, closing the trust gap, and more.

Navigating Distrust: Pharma in the Age of Social Media

AbbVie secures FDA approval for Rinvoq as the first oral Janus kinase inhibitor indicated for giant cell arteritis, expanding its immunology portfolio and signaling strategic growth opportunities in underserved autoimmune markets.

FDA Approves AbbVie’s Rinvoq as First Oral JAK Inhibitor for Giant Cell Arteritis

IQVIA’s Doug Long offers a lay of the land, including trends and developing issues that are shaping the current market. 

Asembia 2025: A General Landscape of the Pharmaceutical Industry

Approval of penpulimab-kcqx marks the company’s US regulatory debut and introduces a new immunotherapy option for advanced nasopharyngeal carcinoma.

FDA Approves Akeso’s PD-1 Monoclonal Antibody for Recurrent or Metastatic Non-Keratinizing Nasopharyngeal Carcinoma

The submission is backed by clinical data from over 2,100 patients, including two pivotal Phase III trials in patients treated with trenibotulinumtoxinE for moderate to severe glabellar lines.

AbbVie Submits Biologics License Application to FDA for Novel Serotype E Botulinum Neurotoxin in Moderate to Severe Glabellar Lines

The simplified requirements for Camzyos are expected to ease prior contraindications, thereby simplifying treatment and expanding eligibility in patients with symptomatic New York Heart Association Class II-III obstructive hypertrophic cardiomyopathy. 

FDA Updates Prescribing Information for Bristol Myers Squibb’s Camzyos to Reduce Echocardiography Monitoring Requirements

Dr. Dina Radenkovic, CEO of Gameto, discusses the key considerations for bringing iPSC-based fertility treatments to market from a regulatory and manufacturing perspective.

Gameto: Addressing Regulatory Hurdles in iPSC-Based Fertility

 video interview, Dr. Dina Radenkovic, CEO of Gameto, discusses the company's pioneering use of induced pluripotent stem cell (iPSC) technology in fertility treatments, particularly addressing ovarian aging. Its lead product, Fertilo, is an iPSC-derived ovarian support cell line in phase III clinical trials in the US with FDA clearance, and commercial availability in markets like Australia and Latin America. The company faces challenges including the scarcity of human eggs and regulatory hurdles. Radenkovic also highlights the need for better models to study female-specific conditions and the importance of political advocacy and funding to drive innovation in women's health.

Pharmaceutical Executive: From a regulatory and manufacturing perspective, what are the key considerations for bringing iPSC-based fertility treatments to market? What unique challenges does this present compared to other cell therapies, and how are you navigating them?


 We’re the first people pioneering iPSC therapy for use in women’s health and IVF. We’re also the first iPSC in Phase III in the US. When there is no precedent, you’re chartering new waters. There’s an additional layer of risk that we must deal with and successfully manage. Fortunately, we now have designations and we’re cleared to commercialize or are in the final stages of development for approval.

One of the challenges is just determining how this product is regulated. Given that our product is used in a dish and has no action inside of the body, it legally meets the definition of a device. Products for maturing eggs outside of the body (which is called in-vitro maturation) have been approved in the US and across the world as devices. On the other hand, our product is still made of living cells. We had to work with regulators around the world to get the proper designation and understand how to get it approved.

It took the longest to get the designation in Europe because they initially said it was not a pharmaceutical product. We went to get regulated as a device, but then they said that devices are not made of living cells. We just managed to obtain a designation with EMA, and we worked similarly with regulators across the world since our designation differs across jurisdictions.

In the United States, we’re considered a biologic, so we’re regulated by CBER. Our IND that was cleared for a Phase III trial was for a BLA submission. In other places where we are cleared for commercialization, we are classified as reproductive tissue or a device. The different designations result in different times to market for different jurisdictions.

When it comes to manufacturing, we have a lot of benefits. We are only treating a few cells and not the whole body. Some of the issues that present with cell therapy, like immunogenicity, are not as hard to solve when you’re only treating a couple of eggs outside of the body. We have already set up GMP manufacturing to supply the IVF cycles, and that is because of the lower dose compared to in-vivo cell therapies. There’s also a reduced scientific challenge when you treat the eggs outside of the body because there is no immune system.

We’ve partnered with REPROCELL, we’ve licensed the one of the world’s first female allergenic iPSC lines, we created a proprietary manufacturing differentiation protocol, we have our bank that should give us supplies for the next 40-to-50 years, we’ve developed potency assays and batch release criteria, and we can rapidly produce Fertilo vials in a scalable and efficient way.

It was challenging setting it up for the first time in iPSC. There were lots of problems that we had to solve, like figuring out how to fill vials with such a low cell dose. IVF is a great first application of cell therapy because it’s much easier to do clinically compared to in-vivo cell therapies, particularly when it comes to cost, scale of manufacturing, cell doses, and immune rejection.

Videos

Dr. Dina Radenkovic, CEO of Gameto, discusses the use of iPSCs in fertility treatments, the most significant scientific hurdles they've encountered in developing and scaling these therapies, particularly in relation to ovarian aging, and they're addressing them.

Gameto's iPSC Fertility: Overcoming Scientific Challenges

 video interview, Dr. Dina Radenkovic, CEO of Gameto, discusses the company's pioneering use of induced pluripotent stem cell (iPSC) technology in fertility treatments, particularly addressing ovarian aging. Its lead product, Fertilo, is an iPSC-derived ovarian support cell line in phase III clinical trials in the US. The company faces multiple challenges, such as the scarcity of human eggs and regulatory hurdles, with Gameto navigating different regions and their regulatory bodies. Radenkovic also highlights the need for better models to study female-specific conditions and the importance of political advocacy and funding to drive innovation in women's health.

Pharmaceutical Executive: Gameto is pioneering the use of iPSCs in fertility treatments. What are the most significant scientific hurdles you've encountered in developing and scaling these therapies, particularly in relation to ovarian aging, and how are you addressing them?


 The first problem is that we didn’t have good models to study ovarian aging that causes infertility (including loss of ovarian function, late-term menopause, and unique pathophysiological and medical conditions). A lot of the animal models that we use, like mice or primates, do not experience the same concept of ovarian aging. We decided to tackle that by building iPSC derived ovaries in a dish. We've done that with the sponsor research agreement with Harvard Medical School's Church Lab. We've published research that shows that our ovaries-in-a-dish beat previous mouse chimeric models. We've even collaborated with Johns Hopkins University and the organ transplant unit to receive cadaver organ donor ovaries and linked to primary tissue to truly replicate human biology in our ovaries-in-a-dish. This allows us to develop different treatments and rapidly test them.

That is what enabled us to bring Fertilo, which is an engineered iPSC-derived ovarian support cell line to mature eggs outside of the body, to phase III in the United States. Our trial is available on clinicaltrials.gov and is going to start recruitment at the end of March 2025. We are already clear for commercialization for therapeutic area agnostic and commercial fertility in several large markets like Australia, Latin America, and others. 

That was the ovary-in-a-dish model that helped us understand human biology and overcome the first scientific obstacle, which was: how do you develop novel treatments if you don’t have good models to study and test them in the lab?

The second challenge was really around our lead product, Fertilo, which is used for egg maturation. You need to get access to human eggs, which are a very scarce resource. This is why the IVF industry exists.

When we did our first wave of human studies, we looked at egg maturation as the end point. We wanted to see if Fertilo matures human eggs and are those eggs genetically, epigenetically, and morphologically healthy like the eggs that have been matured inside of the body with standard of care IVF. We've managed to partner with RMA of New York (one of the leading fertility clinics in Mount Sinai) and worked with one of their fertility specialist doctors. We worked with fertility clinics around the world to build the largest library of sequenced human eggs to truly show and be confident that our eggs are genetically and epigenetically normal.

Dr. Dina Radenkovic, CEO of Gameto, discusses the use of iPSCs in fertility treatments, the most significant scientific hurdles they've encountered in developing and scaling these therapies (particularly in relation to ovarian aging), and how they're addressing them.

Fred Aslan, MD, CEO, Artiva Biotherapeutics, explains what the FDA Fast Track designation for AlloNK in autoimmune diseases means for the acceleration of its development and potential approval of the therapy.

FDA Fast Track: Accelerating AlloNK's Development

 video interview, Fred Aslan, MD, CEO, Artiva Biotherapeutics, discusses the company’s current trials for autoimmune indications in the US. The first is a company-sponsored trial focusing on lupus, while the second is an investigator-initiated trial exploring four conditions: lupus, rheumatoid arthritis, pemphigus vulgaris, and vasculitis. This second trial is uniquely being conducted in a community setting to assess the practicality of outpatient treatment.

Pharmaceutical Executive: You've received FDA Fast Track designation for AlloNK in autoimmune diseases. How do you anticipate this designation will accelerate the development and potential approval of your therapy?


Fast Track designation indicates that FDA considers the therapy to be a novel therapy for an unmet need. There is a higher level of urgency for the agency, which allows for more prompt communication. Another advantage is that if you are approaching a market approval, it opens the optionality for you to have a rolling marketing application. This means that you can submit portions of your filings so the agency can start reviewing them earlier, as opposed to having to have your full dossier completed and then submitting it to FDA. It acknowledges that what you're doing has an important clinical unmet need, as well as increases your opportunities to interact with the agency to make progress a little faster.

Regulatory Involvement for DB-OTO in Hearing Loss

Last month, Regeneron announced updated results from the Phase I/II CHORD trial in children with otoferlin-related hearing loss. Out of 11 children who received DB-OTO, 10 showed notable hearing improvements at different decibel levels, with some achieving nearly normal or normal hearing thresholds. Pharmaceutical Executive spoke with Jonathon Witton, AuD, PhD, VP, auditory global program head, in more detail about the trial results, the performative potential of DB-OTO, and the company’s work with regulatory authorities.

DB-OTO has received multiple designations from the FDA and EMA—how do these designations accelerate the development and potential commercialization timeline?

We have received multiple designations from several regulatory agencies. I'll focus on the FDA. One of the most important designations we've received is Regenerative Medicine Advanced Therapy, which is essentially the breakthrough designation for cell and gene therapy. A key benefit of this designation is the opportunity to have additional meetings with the FDA to discuss your development program.

We’re working in a new area of medicine, and we’re trying to pioneer it. It’s crucial to have early, collaborative discussions with regulators to address the challenges in these types of development programs. For a program like DB-OTO, there’s an urgency to move forward for patients with high unmet needs. It's critical to have open, collaborative discussions with regulators about your development plan. I believe that’s the biggest value of these types of designations, as regulatory authorities are quite busy. They have a lot of work to do. When your programs can get in front of them and you have the chance to meet face-to-face and work through issues, it’s incredibly important for accelerating these programs.

 Regeneron envisions DB-OTO reshaping the standard of care for hearing loss by shifting from symptomatic management with prostheses, such as hearing aids and cochlear implants, to a molecularly targeted therapeutic approach. Historically, clinicians diagnosed hearing loss without understanding its genetic cause, but advances in genetic testing have revealed that over half of childhood hearing loss cases stem from genetic protein deficiencies. DB-OTO aims to correct this by delivering functional DNA to specific inner ear cells, enabling them to produce the missing protein, potentially restoring hearing to near-normal levels.

The transformative potential of DB-OTO is evident in early clinical trials, where 10 out of 11 patients demonstrated hearing improvements, with some achieving normal or near-normal sensitivity. This represents a paradigm shift—clinicians will now need to incorporate molecular diagnostics into routine care, ensuring that patients receive precise, targeted treatments rather than generalized symptom management.

Regeneron has secured multiple regulatory designations, including the FDA’s RMAT designation, which facilitates early and frequent discussions with regulators. These interactions are crucial in navigating the complexities of gene therapy development, accelerating timelines for approval and commercialization.

Challenges remain, particularly in clinical development and manufacturing. Ensuring scalable, high-quality production of gene therapies is as critical as generating robust clinical data. Regeneron has invested heavily in manufacturing capabilities to meet these demands.

Beyond clinical development, Regeneron actively engages with the hearing loss community, including advocacy groups, clinicians, and families. Insights from these discussions have highlighted practical concerns, such as ensuring continuous hearing for safety. This patient-centric approach shapes the development of DB-OTO and future gene therapies, with plans to expand treatments to broader populations, including adults with age-related and noise-induced hearing loss. Regeneron aims to pioneer gene therapy solutions for a currently untreatable public health challenge.

Jonathon Whitton, AuD, PhD, VP, auditory global program head, Regeneron, discusses how key regulatory designations, such as the FDA's Regenerative Medicine Advanced Therapy designation, facilitate accelerated development and commercialization of DB-OTO through increased collaboration with regulators.

FDA

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