PharmaShots Interview: Insmed’s Martina Flammer Shares Insights on New Affordable Drugs for Rare Diseases

In an interview with PharmaShots, Martina Flammer, Chief Medical Officer at Insmed share her views on the R&D continuum to develop new drugs for rare diseases and also highlights its business pillars

Shots:

The idea of the continuum is that all department works together to develop the drugs which will be helpful not only for the company but for patients too
In the drug development process, quality of life is an important consideration. Additionally, the objective is to create the most relevant treatment for patients & to transform the lives of patients living with serious and rare diseases
Insmed’ business is focused on four pillars i.e., Arikayce (amikacin liposome inhalation suspension), brensocatib, TPIP, and translational medicine which has the potential to address unmet medical needs for patients

Tuba: Can you tell me about Insmed’s R&D continuum?

Martina Flammer: I’ve always viewed the development of a new treatment, drug, or any other intervention as a continuum that starts with scientific knowledge that is translated into a clinically relevant application for in-human studies, and into clinical practice. At Insmed, we take a similar approach to research and development where we first ask ourselves if our early scientific insights might be relevant for patients, and if so, “what clinical evidence do we need to see to continue on the path of delivering a new treatment that is impactful?” Data and insights are not the same. We continuously pressure test and form new insights based on data and environmental factors such as health care delivery to make sure we get where we need to go, with the patient experience as our guide every step of the way.

Tuba: How does Insmed’s R&D continuum help to combat silos?

Martina Flammer: When you look at how many organizations function, often their biggest challenge lies in the way they are structured and share information. At Insmed, we have a culture that fosters a more continuous exchange of information early on between functional experts across the organization. Regardless of function, everyone looks at the outcome we need to deliver to patients. Research and development are not separate entities working in a vacuum, but instead, in close collaboration and constant communication with each other. Our collective and ultimate objective is to create the most relevant treatment possible for patients. We also involve medical affairs to ask the research team why it’s important to develop this drug, what is the potential relevance for patients, why patients would choose it, why doctors would prescribe it, and why health insurers would grant access. This approach helps us avoid creating treatments that go to the drug “graveyard”— meaning those that may be approved but are not used because they’re not significantly contributing to unmet medical needs.

Tuba: What about this R&D continuum puts patients at the forefront of how Insmed approaches drug development?

Martina Flammer: Quality of life is an important consideration in drug development and at Insmed, we involve patient representatives, caregivers, and patients themselves so we can constantly listen and learn what is most important to them. Patients are the guiding force in everything we do, and their voices and experiences inform each stage of our R&D continuum, from protocol development to how to use a product. One example is the packaging. When developing packaging for our first product, a drug-device combination, we listened first-hand to patients’ needs and subsequently delivered a final product that takes into account the patient experience in terms of preparation, usage, and cleaning.

Tuba: What other strategies does Insmed implement to ease the burden of disease to patients?

Martina Flammer: Insmed’s commitment to serving patients with serious and rare diseases permeates every aspect of our business. Each department and each person within our organization has a role to play in serving patients and their families and we look at all functions as pieces of that puzzle working together for the common goal of bringing new and relevant therapies to people who need them. Early access and compassionate use are important considerations in rare disease drug development and once we bring a drug to market, we have a world-class patient services team whose role is to educate, inform, and support patients along every step of their journey.

Tuba: What motivates Insmed’s employees to continue working on rare diseases?

Martina Flammer: Our team is motivated by the patient experience. We regularly have patients come into our office or join us virtually to tell their stories first-hand. Whether it’s their journey to diagnosis, what influenced their decision to join a clinical trial, or how they support others in the patient community, we are inspired by people’s real perspectives, challenges, and successes. Our employees don’t view patients as an anonymous body; they know and hear from individual patients directly.

Tuba: Can you share more detail on Insmed’s work, its mission, pipeline, upcoming projects? Please share in detail with our readers.

Martina Flammer: Insmed’s mission is to transform the lives of patients living with serious and rare diseases. Today, our business is focused on four pillars that support this mission – ARIKAYCE® (amikacin liposome inhalation suspension), brensocatib, treprostinil palmitil inhalation powder (TPIP), and translational medicine. Each has significant potential to address unmet medical needs and transform the treatment landscape for patients.

Our recent accomplishments as we advance these four pillars include:

The approval and launch of ARIKAYCE in Japan earlier this year. With this launch, our lead product is now available in three major territories (Japan, Europe, and the United States).
Ongoing enrollment of patients in both the Phase 3 ASPEN study of brensocatib in patients with bronchiectasis and the ARIKAYCE frontline clinical trial program in patients with newly diagnosed nontuberculous mycobacterial (NTM) lung disease.
Positive Phase 1 data for TPIP, a treprostinil prodrug that potentially offers sustained presence in the lungs, reduced systemic exposure, and a PK profile that supports once-daily dosing. These data support advancement to Phase 2 development in both PAH and PH-ILD.
The growth of our early-stage translational medicine portfolio, including recent preclinical-stage acquisitions that we expect, will further augment our research capabilities

Source: US FDA

About Author:

Martina Flammer is the Chief Medical Officer at Insmed. She holds a medical degree from the University of Vienna Medical School, Austria, and completed post-doctoral training in internal and emergency medicine. She also holds a Master of Business Administration degree from New York University Stern School of Business.