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In the world of pharmaceutical names, the trademark has always been the star, and every other word has played a supporting role. But recent developments at FDA-and the industry's response to those developments-have set the stage for a new approach to nomenclature.

Also Known As...The Value of Generic Names

Casey McPherson, Founder and CEO of Chrysalis Genetics, talks about his journey with rare diseases and leveraging ASOs for treatment

Casey McPherson Shares His Journey With Rare Drug Development and Chrysalis Genetics

Casey McPherson, Founder and CEO of Chrysalis Genetics, has an interesting background for someone developing genetic technologies to help treat rare diseases. As a musician based in Austin, Texas, McPherson was inspired to enter the world of drug development and biotechnology after his daughter was diagnosed with a rare genetic condition. In this 

interview, McPherson talks about his journey toward developing a therapeutic for his daughter, as well as changing the overall landscape of therapeutic research for children with rare or ultra-rare genetic diseases.

00:00 - McPherson's background in music and learning about his daughter's rare genetic condition

03:47 - Drug development and the ASO platform

06:54 - The start of Everlum Bio, an ultra-rare genetic disease drug development CRO

08:06 - Commercializing rare disease drugs for global populations

11:10 - McPherson shares his thoughts on the future of the rare disease landscape, including shifting regulatory paths

14:23 - Lessons learned to guide other entrepreneurs in the biotech industry

17:00 - Advice for parents navigating the rare/ultra-rare disease space

Casey McPherson, Founder and CEO of Chrysalis Genetics, talks about his daughter's rare genetic condition, and how it led him to antisense oligonucleotides (ASOs) and therapeutic development for rare diseases.

AbbVie Pedro Valencia ADCs

PE: How is AbbVie using novel cancer biomarkers to develop potential first-in-class ADCs for the treatment of advanced solid tumors?

We're constantly looking for biomarkers that can help us to do two key things. The first is to tackle cancer cells. and spare normal cells. We’re looking for specific antigens that are over-expressing tumor cells, and you see limited expression in normal cells. We want to make sure that target that’s over-expressed is critical for the survival of the cancer cell. Once you block it, you can actually drive a cancer cell death. That’s what we're looking for. In that perspective, c-Met is one of those biomarkers that is over expressing multiple tumor types such as lung, colorectal, gastric, and other GA tumors. We as an industry have tried to tackle c-Met in the past but have been unsuccessful. It would actually be successful to tackle what we call met, which is a gene inside the cell.

There’s this thing called met amplification, where the cancer cell produces multiple copies of the gene, and around 30% of patients have it. We can actually tackle that with small molecules. But once the gene becomes a protein and gets over-expressed in the cell, that over-expression is something we haven’t been able to target. For example, we know that on lung cancer, c-Met over-expression is about 25% to 50% in colorectal cancer, and it could be anywhere from 30% to 70% depending how you measure it, so it’s quite prevalent.

With ADCs, we’re trying to tackle c-Met over-expression on the surface of cancer cells. That was novel, and we can do it through ADCs. 706 has a target called SEZ6, and it’s a target that is over expressed in neuroendocrine cells or neuroendocrine neoplasms. When you think about it, multiple tumor types kind of have a subset of cells that are neuroendocrine driven. Small cell lung cancer is one of the few that has everything driven by neuroendocrine cells. The beauty of SEZ6 is over-expressing those cells and limited expression in normal cells. So, we can tackle it with an ADC and drive the results that we're seeing today.

PE: ADCs have redefined the treatment of cancer in recent years. Can you please discuss the promise of this emerging class and what the future may hold?

At the end of the day, ADCs work like a trojan horse. You have a good antibody that cancer cells are happy to receive, but once you hook into the cancer cell receptor, you deliver the payload, which is pretty much chemotherapy. It’s actually a highly potent version of the regular chemotherapy cancer patients are given on a regular basis. So, from that perspective, our goal is to replace chemotherapy. Our grand goal with ADCs is to replace good old, decades old chemotherapy that is really toxic for some patients. That’s how we see the redefinition of cancer treatment in the future with ADCs. So, what do we see in the future? Well, if you think about it, chemotherapy is being given in combination with other agents. In the future, we see ADCs being given in combination with chemotherapy. There will be one with other agents such as immuno-oncology agents. So, if you combine the ADC and the PD1s, you can start seeing that synergy come together and kind of start really dreaming about cures for the patients. All in all, we're super excited about ADCs replacing chemotherapy. 

In an interview with Pharm Exec Associate Editor Don Tracy, Pedro Valencia, VP, Solid Tumor Pipeline Strategy & Execution, AbbVie, discusses using novel cancer biomarkers to develop potential first-in-class antibody drug conjugates (ADCs) and the promise that they offer. 

Utilizing Biomarkers to Develop First-in-Class Antibody Drug Conjugates to Treat Advanced Solid Tumors

Ash Shehata from KPMG discusses how investors are feeling about the pharma and life sciences industry heading into 2025.

What’s Driving Pharma Investors in 2025

Pharmaceutical Executive: What’s causing the increase in M&A in 2025?


 I think a lot of things. This year, it's really about smart diligence. At the end of the day, there's a lot of capabilities that we think are going to be driving that. Commercial success (operational and financial) and tax are driving a lot of the trends we're seeing. There's also some bigger groundwork underway for the healthcare and life sciences industry around global innovation, fueled by the future of AI.

Clearly there's an impact right now on the financial markets due to inflation lowering. That's going to drive deal activity a bit more this year, although people are maybe a little bit more cautious of what happens in the out years. At the end of the day there's a generally optimistic environment for the biopharma market. As we start to see capital market improvements, biotechs are clearly going to be back on the radar for this year, and we believe that will continue beyond this year. A lot of the portfolios that are sitting in private equity, with venture capitalists, as well as in corporates are also undergoing quite a bit of review. That's also going to drive a variety of deal activity. There's a lot of fundamentals, whether it's economics, some of it is just timing in the market trajectory. It's hard to even have this discussion, of course, without the political impact, but clearly that's also starting to get factored in as well. All in all, it's driving a pretty good level of activity.

PE: Do you think that the industry was waiting to see the results of the election before committing to a strategy for the coming year?


 There may have been a little of that, but I think a lot of the issues we've seen across the industry have generally been bipartisan. That’s true whether we talk about things like the IRA, transparency in pricing, negotiating contracts, and all of those things. Some of the pressures that we're seeing on the payment side, through the payers, the discourse around PBMs, all of those things have generally had somewhat of a bipartisan support going into the election year.

When those unknowns became knowns, as we got closer to election day, and clearly now, the industry showed a willingness to go forward. You have a combination of the financial markets easing and the election getting past us now, that's making for a pretty good and enticing deal market right now. That might go at least until the midterms, when we might see yet another turn of the election results. But I think that is creating a little bit of pressure right now to try to get some of these deal deals done, as long as the financial environment remains positive.

PE: What are the other factors that you think are most likely to impact deal activity?


 The big issue we start to see looking down the road is whether the true impact of technology and AI is going to start to take hold. We think that will take have a significant role in driving development around healthcare and life sciences deals and even acquiring innovative technologies. But at the end of the day, some of those impacts, at least in the pharmaceutical life sciences industry, will likely be delayed in 2025.

It's optimistic, but I think a lot of the novel and innovative therapies may not get the full benefit for some of the AI discovery programs.

Alcohol Use Disorder AD04

PE: Can you elaborate on the key design elements of the AD04 study and how they contributed to achieving positive topline results?

The purpose of the study was to evaluate the relative bioavailability of AD04. We compared low dose ondansetron, which is what AD04 is, to a reference standard marketed product, which is generic ondansetron at 4 mg. We also looked at the PK variability and the dose proportionality between two doses of AD04 through .33 mg and .99 mg. We also evaluated the food effect on the bioavailability of AD04 and of course, we looked at safety. 

PE: What do these results indicate about the pharmacokinetic profile of AD04, and how does this align with the drug’s intended therapeutic use for Alcohol Use Disorder?

We’re really happy to say that AD04 behaved as expected. It followed the normal dosing properties that we were expecting to see when we started the study, and it's not impacted at all by food intake or the timing of those meals.

The AD04 study aimed to assess the relative bioavailability, pharmacokinetics (PK), and safety of AD04, an ultra-low-dose ondansetron formulation, compared to a generic 4 mg ondansetron product. Key design elements included evaluating PK variability and dose proportionality between two AD04 doses (0.33 mg and 0.99 mg), assessing food effects on bioavailability, and confirming safety across patient demographics.

The results confirmed AD04's predictable pharmacokinetic profile, demonstrating that its bioavailability was unaffected by food intake or meal timing. These findings align with the drug's intended use for alcohol use disorder (AUD), particularly as a convenient treatment option. Notably, the study revealed enhanced efficacy in patients with specific genetic mutations in the 5-HT3 receptor region, supporting the drug’s targeted therapeutic approach.

The study addressed critical feedback from the FDA regarding bioavailability and PK data requirements for repurposed drugs. This milestone clears the path for initiating a Phase III study, expected to begin early to mid-next year, focusing on the genetically defined subgroup showing higher efficacy.

Safety results indicated strong tolerability across all tested groups, consistent with prior studies. The company is also exploring external collaborations for both development and commercialization, leveraging these findings to advance AD04’s clinical and market readiness. These efforts position the drug for meaningful progress toward FDA approval and broader adoption for AUD treatment.

Cary Claiborne, CEO, Adial Pharmaceuticals, discusses promising results of the AD04 study in patients with Alcohol Use Disorder

AD04 Study Highlights Consistent Bioavailability and Safety for Alcohol Use Disorder Treatment

Jennifer Hawks Bland, CEO of NewYorkBIO, shares her journey from litigation to biotech leadership, discusses fostering innovation in New York's thriving bioscience ecosystem, and outlines her vision for the state as a global biotech hub.

Biotech in the Empire State: Building a World-Class Bioscience Ecosystem in New York

Jennifer Hawks Bland, CEO of NewYorkBIO, shares her inspiring journey from a legal career to leading New York’s burgeoning bioscience ecosystem. Throughout her interview with 

, Jennifer discusses the unique strengths of New York as a biotech hub, including its world-renowned academic institutions, diverse population for clinical research, and robust financial infrastructure. She highlights NewYorkBIO’s role in fostering innovation, supporting early-stage companies, and connecting industry leaders. With her vision to position New York as a global leader in bioscience, Jennifer offers insights into the collaborative efforts shaping the future of biotechnology in the Empire State.

00:00 – Jennifer’s background in litigation and advocacy
2:57 – Jennifer’s approach to leadership at NewYorkBIO
4:11 – Advantages of forming a large bioscience ecosystem across New York State
6:29 – Leveraging partnerships between academia and research to fuel innovation
9:00 – Current advocacy priorities at NewYorkBIO
11:09 – NewYorkBIO’s approach to addressing current macroeconomic conditions in New York and beyond
14:19 – Significance of forging connections between Big Pharma and emerging biotech firms
15:36 – NewYorkBIO success stories demonstrating impact across the Empire State

Daniel Boring

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