Lumos Pharma’s Patient-Centric Strategy for Rare Disease Drug Development

Rare diseases, often overlooked in the broader medical research field, affect a small percentage of the population yet carry a significant burden for those diagnosed and their families. Among these, rare pediatric diseases present unique challenges and opportunities for rare disease drug development.

Pediatric growth hormone deficiency (PGHD) is one such condition, affecting a child’s growth and development. This rare endocrine disorder, characterized by inadequate secretion of growth hormone from the pituitary gland, has profound impacts on physical and sometimes cognitive development. Traditional treatment methods, primarily revolving around injectable therapies, have been the standard for decades. However, they come with their own set of challenges, both medical and psychological, for young patients.

Lumos Pharma is dedicated to transforming the treatment landscape for rare pediatric diseases like PGHD. Lumos Pharma’s approach is not just about creating new drugs; it’s about rethinking the entire patient experience, prioritizing ease of use and minimizing the treatment burden on young patients and their families. Their work exemplifies a broader shift in the pharmaceutical industry towards patient-centric drug development — a paradigm that places the needs and well-being of patients at the forefront of every stage of the drug development process.

Lumos Pharma’s endeavors in developing novel treatments for PGHD and other diseases underscore a broader mission: to provide hope and improved quality of life for patients who have long awaited effective and accessible treatments.

Xtalks spoke with Rick Hawkins, CEO and Chairman of Lumos Pharma, about the intricacies of rare disease drug development. Hawkins shares how Lumos Pharma adopted a patient-centric strategy to help shape the future of treatment for conditions like PGHD, and more.

Could you share how your previous experiences, especially founding Pharmaco and id2, have shaped your vision and approach as the CEO of Lumos Pharma?

Hawkins: I started my professional journey at Johnson & Johnson, gaining extensive experience in both the preclinical and clinical development of pharmaceuticals, as well as regulatory insights. This diverse background was instrumental in founding Pharmaco in Austin, Texas, which became one of the first contract research organizations (CROs) globally. My ten years there were marked by significant growth, as we expanded to about 2,000 employees, a legacy that continues today in PPD with 40,000 employees.

“Lumos Pharma’s main project is not just about creating a new drug but revolutionizing the treatment paradigm for pediatric growth hormone deficiency (PGHD) and potentially other growth hormone-related conditions.”

 

– Rick Hawkins

After leaving Pharmaco, I collaborated with Dr. John Kopchick from Ohio University. Together, we developed the world’s first growth hormone receptor antagonist, Somavert (pegvisomant), targeting acromegaly. This venture also led me to identify a gap in the market for contract manufacturing of recombinant proteins. To fill this gap, I established a facility for this purpose in Research Triangle Park in North Carolina, which was eventually acquired by AkzoNobel and is now part of FUJIFILM Diosynth Biotechnologies.

Another pivotal moment in my career was working with Dr. Michael Thorner, who was a renowned endocrinologist at the University of Virginia (UVA). We collaborated on developing MK-677 (LUM-201), an oral growth hormone secretagogue, after Merck decided to put it on the shelf. This project has now become a central focus at Lumos Pharma, where we are developing it as a novel oral treatment for PGHD. Our approach is unique in that it stimulates the natural pulsatile release of growth hormone, a method we believe is more aligned with the body’s physiology. We’ve conducted Phase II studies, which have met all primary and secondary endpoints and are now moving towards a pivotal Phase III trial.

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Lumos Pharma places a strong emphasis on being patient focused. How does this stance influence your decision-making and the company’s approach to drug development?

Hawkins: At Lumos Pharma, our patient-focused stance substantially shapes our decision-making and approach to drug development. This philosophy was influenced by my experience at Johnson & Johnson, where I worked on pimozide, a drug for Tourette’s syndrome. This experience underscored the importance of connecting with patient populations, particularly those suffering from rare diseases.

“We learned the value of this approach from Abbey Meyers, a mother of two children with rare diseases, who championed the Orphan Drug Act of 1983, which incentivized the development of drugs for rare diseases.”

 

– Rick Hawkins

Our commitment to being patient-focused means actively partnering with patient groups and families. For instance, in developing Somavert, conversations with patient advocacy groups were crucial. These interactions provided direct insights into the patients’ daily challenges and emphasized the need for dedicated research and development in this area.

This patient-centric approach contrasts significantly with my earlier experiences in big pharma, where direct patient interaction was rare.

At Lumos Pharma, we prioritize this connection, illustrated by our collaboration with pediatric endocrinologist Dr. Pisit “Duke” Pitukcheewanont, Chief Medical Officer of Lumos Pharma and President of the Human Growth Hormone Foundation. This partnership reflects our commitment to deeply understanding and addressing the needs of our patient communities.

What are the unique challenges in developing treatments for rare diseases, and how does Lumos Pharma address these challenges?

Hawkins: Developing treatments for rare diseases at Lumos Pharma presents unique challenges, chiefly due to the small patient populations. For instance, a rare disease in the US is defined as one affecting fewer than 200,000 people, but some conditions may only affect a few hundred or thousand individuals.

“The scarcity of rare disease patients makes identifying potential participants challenging, showing the importance of partnering with patient advocacy groups.”

 

– Rick Hawkins

In the case of PGHD, a rare disease we focus on, diagnosis is more feasible as pediatric endocrinologists are familiar with it, thanks to the availability of treatments for nearly 40 years. However, the rarity still necessitates conducting trials across many clinical sites worldwide, increasing the complexity and cost of development. Our recent global trial involved about 45 centers, even amidst the pandemic, which posed additional challenges.

Key to our success in addressing these challenges is our experienced team of 35 full-time professionals, who bring decades of expertise in rare diseases. Additionally, working with experienced investigators accustomed to injectable treatments has been advantageous. The introduction of an investigational oral treatment for PGHD has garnered significant interest, aiding in efficient patient recruitment and trial progression. We anticipate this effective approach will continue to benefit our upcoming pivotal trial.

Looking ahead, what are the key goals and aspirations for Lumos Pharma in the next five years?

Hawkins: Looking ahead, Lumos Pharma’s key goals and aspirations for the next five years include substantial advancements in our clinical trials. We’ve successfully met all primary and secondary endpoints in both of our recent studies, paving the way for a pivotal global trial in moderate PGHD, planned for the second half of 2024 following an End-of-Phase II meeting with the FDA in the first half of 2024.

Our approach is informed by historical precedents in growth hormone product reviews by the FDA, guiding our preparations for the Phase III study. This includes detailed planning in site selection, investigator discussions and addressing chemistry, manufacturing and control (CMC) aspects.

Moreover, we aim to build a substantial commercial presence around our product, leveraging the advantage of working in rare diseases. Our strategy involves closely working with pediatric endocrinologists and a targeted sales force, potentially enabling us to efficiently cover markets like the US. We are actively planning and discussing the commercialization phase, excited about the potential to significantly impact the rare disease sector and improve patient care.