CEO Conversations XXV: Ms. Meredith Manning, General Manager, PharmaEssentia (6446 TT) US subsidiary
PharmaEssentia’s ropeginterferon, branded BESREMi®, was granted US regulatory approval to treat polycythemia vera (PV) by the FDA on 12th Nov 2021. Called by some physicians a “Superinterferon”, BESREMi® is an innovative, long-acting form of interferon with a strong clinical profile. The drug was approved by the EU EMA as a frontline treatment for polycythemia vera in 2019. Now, it has become the only approved treatment for PV in the US regardless of prior treatment history.
Polycythemia vera (PV) is a rare blood disease. Along with essential thrombocytosis (ET) and myelofibrosis (MF), these diseases are categorized as myeloproliferative neoplasms (MPN). PV and ET each currently affect about 160,000 patients in the US, putting both diseases in the orphan disease category (hurdle = less than 200,000 patients). The excess red blood cells produced in the body from PV make the blood thicker than normal resulting in an increased risk of blood clots, heart attack, stroke, headaches, blurred vision, blind spots, gouty arthritis, and an enlarged spleen. The condition not only lowers one's quality of life but can also be life-threatening. Over time, PV can develop into myelofibrosis, and also sometimes leads to other blood cancers, like acute myeloid leukemia.
Ropeginterferon is a new, monopegylated long-acting interferon. Clinical data spanning over 7.5 years show Ropeginterferon is effective at controlling the disease. In clinical studies, 61% of patients achieved a complete hematological response and 80% of patients achieved a hematological response based on laboratory parameters, which are metrics physicians typically use to make treatment decisions. And the regimen was tolerable; most patients were able to stay on treatment.
PharmaEssentia has prepared well for its entry into the US market. The general manager of the US operation, Meredith Manning, is a University of Chicago Booth School of Business graduate, with two decades of experience as a product strategy specialist at top US pharmaceutical companies. She has been responsible for multi-billion dollar product lines and once successfully achieved US$1bn in sales for a new drug asset within eight months. The team Meredith has assembled for the BESREMi launch has deep knowledge of the most advanced developments in MPN treatment, as well as deep connections with key opinion leaders and treatment centers. The QIC team is very excited to have an interview with Ms. Manning and listen to her plan for the US launch.
QIC (Q): From Pfizer to Shire, you gained considerable experience as a senior marketing manager for several drugs, particularly in the blood disease segment. What did you learn from these positions? How does that help you with this new challenge?
Meredith Manning (M): I’m very gratified that my experiences throughout my career, particularly in roles with Pfizer, Vertex, Baxter and Shire, have allowed me to bring innovative pharmaceutical advancements to patients. Two critical learnings from these roles have been seminal in our efforts here. First and foremost is the data. In my roles, I’ve really understood how the data drives both the scientific understanding of the product, and the means to communicate the safety, efficacy, and benefit to the patient community. We focused a lot on rigorous data at Pfizer, diving into the analytics of the data to make smart decisions and develop a solid strategy.
But equally important is a keen understanding of the community we aim to serve and their needs. Having honed our market insight skills by listening to the patients to learn what they need, we are much better equipped to deliver a product that provides real value. In this community, that means understanding the clear unmet medical need. In PV, currently available therapeutics treat only the symptoms of the disease such as pruritus or an enlarged spleen. But we hear all too often of patient anxiety over the risk of progression. So our focus with BESREMi® is to treat the whole disease at the underlying cause in the bone marrow, not just the symptoms. This complete disease control may help to reduce the risk of progression over time, delivering reassurance to physicians and patients. My experience at Vertex taught me a lot about this, as we launched the company’s first product in hepatitis. The launch was at the time the most successful launch in the industry, hitting over a billion dollars in sales in under eight months. It was impressive in part because there were tremendous unmet needs, but also because we carefully listened to what doctors said they and their patients needed most.
My experience at Shire helped me understand the complex global dynamics that can shape a product’s success. There I was working as the product strategy head of seven different products in the hemophilia portfolio, selling in 65 countries around the world. Being able to sell a product to so many diverse communities requires understanding critical strategic levers and highly diverse community needs, like the patient engagement experience in Japan and the frequency they visit their doctor for a prescription, or like the insurance considerations in the highly varying regions in India. A thorough understanding of these market dynamics is absolutely paramount to our success.
Q: You have a lot of experience in hematology, so do you have a connection to this disease? What differences do you see among these patients as compared to those with other diseases?
M: My previous experience is in hemophilia, which is a different dynamic because it's a genetic disease that begins at birth. It can be a very devastating diagnosis for the parents. We should always be cognizant of the emotional stress that patients and parents feel at the time of a diagnosis as well as their medical needs. Beyond hemophilia, I think everyone, myself included, has a connection to the cancer community. Polycythemia vera, or Myeloproliferative neoplasms as a whole, is a combination of both of these communities. I was fascinated by the complexity and the science of MPNs, and was very excited to have the opportunity to leverage my previous experiences in hematology and oncology.
When I join a company, it's not just about bringing a product into the market, it's also about how can we transform the way in which care is being delivered around this very specific medical need. In PV, there hasn't been an FDA-approved product for patients across all lines of therapy in PV until now, so BESREMi® is a transformational new drug for this community. Our job now is to help the community understand how to diagnose and to use this product to treat PV. When I read about BESREMi® and what Dr. KC Lin and the team had invented, I was thrilled because this isn't just another interferon; this is the first time that we're going to be able to unleash the full potential of interferon for a community with a clear unmet need.
“Besremi is the first FDA-approved medication for polycythemia vera that patients can take regardless of their treatment history, and the first interferon therapy specifically approved for polycythemia vera.” – US FDA.
Q: Some people doubt that a small Taiwanese company like PharmaEssentia can pull off a major new drug launch in the US. How do you see it?
M: I have full confidence in our ability for a successful launch. Firstly, PharmaEssentia has really good science. When I met Dr. Lin and learned about the history of the product development, I was impressed by his deep knowledge and understanding of interferons and the technology of pegylation. As Dr. Lin says: “Better science, better lives.” So we’re bringing a really important new technology into the United States to advance care for patients. And just as important, we’re prepared in the U.S. to make an impact. We’ve convened a team with a deep understanding of market needs and drivers with the capability of communicating BESREMi®’s benefits.
Small companies with important science can be very powerful. I saw this first hand at Vertex – a great product and a prepared team meant we could compete with market leaders like Merck and be successful.
Q: Now that BESREMi® was approved by the US FDA, I’m sure your colleagues in the US are all very excited. Can you talk about the US team, and the follow-on plan?
M: We are truly excited about the launch in the U.S., and the morale is incredibly high. We talk about focusing on one team, one mission. We are all very collaborative, working together to bring this important therapy to the MPN community. We know that physicians deserve more therapeutic options, and PV patients deserve better care. Our team is very passionate about helping to bring better science to make a difference in people’s lives. We have attracted industry-best leadership in sales, medical affairs and other critical functions who bring us the best capabilities to navigate a complex market and deliver on our goals.
PharmaEssentia US team
Q: How do you see the competition in this space and what products might come to the market in the future? What makes BESREMi® stand out?
M: It’s important to know that we have a comprehensive clinical program with BESREMi® and PV and a deep understanding of how to operate across the different patient populations. Other interferons have not been as extensively studied as ours. It’s not just about understanding the dosing, the efficacy, and the safety profile. It’s also most important to study how patients at various stages of disease progression respond. The real benefit of BESREMi® is the fact that the mechanism of action targets the disease’s origin in the bone marrow. That's why we believe that we're seeing the high and durable complete hematological response. The medicine is not just treating symptoms and managing near-term complications; it is actually going to the source of the disease to achieve high and durable efficacy.
Q: Overall, how do you see the PV market in the US?
M: The community is still learning about the science of MPNs, and specifically PV, and that’s what makes this market so exciting. Researchers are still trying to understand why some patients progress faster than others. That is the reason we believe bringing BESREMi® to the market provides a phenomenal opportunity to help transform care. By bringing BESREMi® to the market, we can meet the long-neglected needs of the large patient group. We studied BESREMi® over a very broad patient population, many of which were early in their disease progression. We had patients on HU and treatment naïve patients. And we had high risk patients including 22% whom had a previous cardiovascular event. Another 16% of the patients were newly diagnosed. So we worked across a broad group of patients at various stages of their disease journey.
The recent study that Dr. Tiziano Barbui did in Italy is for an additional patient group: low risk or younger patients who were treated by the guidelines for only phlebotomy. He’s trying to determine if BESREMi® would be a better approach than phlebotomy in that patient population. As we gain more insight into those questions, we will have a clearer view of PV and of the potential of BESREMi®.
Q: I believe you have talked to a lot of opinion leaders, physicians and patients. How do they react to this product?
M: Over and over again, we hear how excited this community is to have access to BESREMi®. They understand the advance that it represents, and are grateful to have innovation that gives them an effective option when the limited options today haven’t been sufficient. One thought leader told us recently that PV patients haven’t been as much supported by the industry as they could be, and so they are excited that PharmaEssentia has been so dedicated to PV patients with comprehensive patient support programs. So often we hear how much BESREMi® is a great advancement for patients and for physicians in the MPN space.
Q: What is the current status of insurance reimbursement on the medicine?
M: We have invested heavily in ensuring that if a patient is deemed appropriate for this therapeutic, then they can get access to our product. We are actively working with payers to support coverage of BESREMi®, and in the next several months we will work to ensure BESREMi® is on formularies, the lists of products approved for reimbursement. In addition, we have comprehensive patient services to help overcome insurance barriers such as prior authorizations and co-pay issues.
Q: Currently HU is widely used to treat frontline PV and is cheaper than BESREMi®. Why would insurance companies want to support BESREMi®?
M: While cost is an important parameter, insurance companies also understand that a product with clear clinical benefits should be covered. Payers want to make sure that they are investing in a product that will be beneficial to their patient population. Our job is to communicate to the payers what benefit BESREMi® will bring to their patient population. It is a long-term disease, so a payer will prefer paying for a product that can control the disease over the long term. HU is actually not an FDA approved drug for PV, and while it is inexpensive, it’s clear from real world data that patients’ blood counts are not under control. Even if it is inexpensive, why would anyone pay for it if it is not working?
Insurance companies tend to follow the FDA approval and the guidelines of professional associations such as NCCN and American Cancer Society when making coverage decisions. We’ve already begun to engage providers of guidelines, sharing with them the FDA approval and the clinical literature so they can evaluate our product for inclusion in their guidelines. It is important for us to ensure that we communicate the benefit, and ensure that our services help patients.
Q: Is there anything you would like to say to the PV community, as BESREMi® is now officially approved in the US?
M: Personally, I would thank them for welcoming us into the PV community. Dr. Lin has been an important and longstanding member of this community. I’ve noticed that everyone I’ve met in the PV or MPN community very much welcomes working with PharmaEssentia. I think that starts with our leader who is so dedicated to bringing forward science that can transform the way we treat cancer. By bringing BESREMi® to the US, we are helping to raise awareness about PV and further advance the science of the disease. We look forward to continuing to work with and grow with the community for years to come.
More about PharmaEssentia:
CEO Conversations Installment VII: Dr. Ko-Chung Lin, Founder & CEO, PharmaEssentia (6446 TT), Part I
More about the approval of BESREMi®:
If you would like to arrange a meeting with PharmaEssentia’s Founder, Dr. KC Lin, or Ms. Meredith Manning, please contact yvonnehuang@qtumic.com.
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