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While new regulatory requirements are often seen as a roadblock, they also present an opportunity to develop new and innovative solutions.



Turning Commercial Compliance Roadblocks into a Springboard for Innovation

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

Pharmaceutical companies’ direct-to-patient (DTP) programs have recently sparked conversations and criticism in fear of their potential to “cut out” or influence physicians, the truth is that physicians remain an irreplaceable cornerstone of healthcare—wherever that care occurs. These programs are designed to enhance healthcare access and speed the care pathway, not to sideline doctors—and studies support that.

, 83% across specialties believe at-home diagnostic tests help move patients through the care continuum faster, leading to earlier diagnosis and treatment to support better health. Also, 52% of clinicians believe they deliver more effective treatment and follow-ups via teleconsultations

, according to the American Journal of Managed Care.

As a highly regulated industry, pharma companies have been historically slower than most to adopt sweeping technological change. Even within the legal and regulatory parameters that are in place to ensure patient privacy, lack of undue influence and conflicts of interest, pharma has taken an innovative leap to support a better patient experience through DTP programs. For all those reasons, innovative pharma companies should be congratulated for their work to engage patients.

We are facing an unprecedented 58,000-95,000 physician shortage by 2030

, and access to specialists even now is especially challenging (86% of rural counties in the U.S. don’t have access to a single cardiologist).

 Physicians are still dedicated under a Hippocratic oath to care for their patients. 

 that happens, however, is rapidly changing. It’s changing because patients—not pharma—want it to. Consider this:

82% of consumers demand personalized web portals for common healthcare transactions

52% of consumers now expect to find whatever they need from a company in three clicks or less

80% have accessed care via telemedicine at least once in their lives

65% of consumers expect connected experiences

Our current healthcare workflows are also hurting physicians. According to the AMA, 48% of physicians have experienced at least one symptom of burnout. While this number is lower than other years, it’s still too high. It has been documented that physician burnout adversely impacts patient health, with the NIH reporting that physician burnout can double the risk to patient safety and contribute to 7%-10.6% of serious medical mistakes.

 As physician shortages rise and the over-65 population grows, integrating telehealth technology into physician workflows will help ease their 9 am to 5 pm workday and support clinical decision making.

In that same survey of US physicians, one respondent said, “Virtual care has absolutely made a positive impact on my patients, and I like how quick and easy it is to add these visits in at the end of my day or during a lunch break.”

Pharma isn’t causing the shift towards proactive consumer health management and technology adoption, they’re adapting to it and supporting it because ultimately, better care will be delivered with it. Not only will more patients have access to physicians and medication, they will have better access to the medications they need, earlier—saving and improving lives. It is well documented that earlier interventions make a difference:

 Early detection and management of type 2 diabetes can reduce the risk of complications by up to 50%.

 Regular monitoring and early intervention in cardiovascular conditions can decrease the likelihood of heart attacks by 25%.

Screening for CKD in individuals with diabetes can lead to early diagnosis, potentially reducing the progression to end-stage renal disease by 30%.

These statistics underscore the critical importance of early healthcare interventions in managing and mitigating the impact of various health conditions and the faster we can connect patients with doctors, the better.

The physician remains central even as DTP programs are implemented. The independent clinical decisions that happen during an in-person visit are still happening—but they are happening virtually. The idea that physicians that work in telehealth environments are less qualified or thoughtful than in-person physicians is unfounded. In fact, according to the CDC, most primary care physicians (76.7%) and medical specialists (73.1%) were able to provide a similar quality of care during telemedicine visits as in-person visits “to some extent or a great extent.”

Suggesting that physicians participating in DTP programs are incentivized by pharma to "sell" their drugs is not only an affront to the healthcare professionals dedicated to improving global health every day but also entirely false.

Technology is finally making end-to-end virtual care a reality. From telehealth to lab testing, digital appointment scheduling to Rx delivery—our industry is on the cusp of making real progress toward an easier healthcare journey. Through it all, the physician is central and will always have patient health as their primary objective, wherever they interact with them.


Source: ixlayer HCP at-home diagnostic testing attitudes survey, in partnership with The DHC Group and Sermo, April 2024 n=141

Patient and clinician Experience with Telehealth: 

https://www.researchgate.net/publication/330544973_Patient_and_Clinician_Experiences_With_Telehealth_for_Patient_Follow-up_Care

Source: *The Complexities of Physician Supply and Demand: Projections From 2021 ot 2036, AMC, 

https://www.aamc.org/media/75236/download?attachment

From Awareness ot Action: Tackling the Rising Burden of Common Chronic Diseases, FDA, 

https://www.fda.gov/news-events/fda-voices/awareness-action-tackling-rising-burden-common-chronic-diseases?utm_medium=email&utm_source=govdelivery

State of the Connected Consumer, Salesforce June 2019 

https://www.salesforce.com/content/dam/web/en_us/www/documents/reports/connected-healthcare-consumer-report.pdf

https://rockhealth.com/insights/consumer-adoption-of-digital-health-in-2022-moving-at-the-speed-of-trust/

https://pmc.ncbi.nlm.nih.gov/articles/PMC10266854/

https://www.ajmc.com/view/population-health-screenings-for-the-prevention-of-chronic-disease-progression?utm_source=chatgpt.com

https://www.england.nhs.uk/ourwork/prevention/secondary-prevention/cardiovascular-disease-high-impact-interventions/

https://bmcmedicine.biomedcentral.com/articles/10.1186/s12916-022-02438-6

https://www.cdc.gov/nchs/products/databriefs/db493.htm

Physicians remain central even as DTP programs are implemented. 

Engaging Patients: Why Pharma’s Direct-to-Patient Approach Should Be Applauded

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

IPF Progress

PE: Are there any ongoing collaborations with patient advocacy groups or organizations to raise awareness of IPF or facilitate patient participation in the clinical trial?

We’re working with a number of different pulmonary fibrosis advocacy groups right now, both in the United States and internationally. These include PF Warriors, the Pulmonary Fibrosis Foundation United States, Foundation Australia, EUPFF, and the Action for Pulmonary Fibrosis in the UK. We really need to do this, because even while IPF is a rare disease with around 150,000 patients in the United States, it’s not common. You don't have a patient on every corner. It's kind of weird, because if you talk to people, it doesn't seem like anyone's more than one to two degrees of separation away from an IPF patient. You really need to use the patient advocacy groups to let them know about the trial and bring the patients in and give them an opportunity to be on this drug. Our hope is that because the efficacy data is superior to current standard of care and standard of care is generally not very well tolerated, and this drug seems to be better tolerated, even more patients will come out, get on the study, and benefit from the drug. Advocacy groups can help with that. 

The initiation of the WHISTLE PF trial for ENV-101 aligns with the company’s broader strategic goals by advancing its priority of addressing idiopathic pulmonary fibrosis (IPF) with transformative therapies. ENV-101 targets the Hedgehog signaling pathway, directly addressing the disease’s underlying pathology by reducing fibrosis and potentially reversing lung damage, distinguishing it from current IPF treatments that only slow disease progression.

Phase IIa trials have demonstrated both clinical and statistical significance in improving lung function, structure, and reducing fibrosis. With a favorable safety profile observed across 230 patients, including oncology studies, the WHISTLE PF trial will further validate these findings in a larger, more diverse population. The Phase IIb trial will explore additional lower doses to optimize efficacy and tolerability while permitting standard-of-care therapies, ensuring comprehensive data collection. Safety monitoring will include an independent review board to oversee adverse events.

The company is also collaborating with patient advocacy organizations like the Pulmonary Fibrosis Foundation (USA), PF Warriors, and international groups in Australia, Europe, and the UK. Given IPF’s life-threatening nature and the drug’s promise to outperform standard care in both efficacy and tolerability, these partnerships are important for patient education and awareness.

While regulatory timelines remain uncertain, the company is committed to expediting the process, acknowledging the severe prognosis of IPF. The potential for accelerated approval, typically rare in pulmonary conditions, underscores the drug’s unique position as a game-changer for this devastating disease.

John Hood, CEO, Endeavor BioMedicines discusses partnering with advocacy groups to increase awareness of clinical trials for idiopathic pulmonary fibrosis. 

Guenter Kloucek

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