[Aliaksandr Marko/Adobe Stock]
In mid-April, San Diego–based Pipeline Therapeutics announced a global license and development agreement with Janssen Pharmaceutica NV (NYSE:JNJ), focusing on PIPE-307, an investigational compound targeting nervous system disorders. As a pioneer in precision neuroscience, Pipeline Therapeutics aims to create targeted treatments for patients. PIPE-307, an oral, highly selective antagonist of the muscarinic M1 receptor, embodies this approach, having completed two phase 1 clinical trials and obtained FDA clearance for clinical development in relapsing-remitting multiple sclerosis (RRMS) patients. Janssen received a worldwide, exclusive license to research and develop PIPE-307 in all indications.
Under the agreement, Pipeline will receive $50 million upfront from Janssen, with the possibility of receiving an additional $25 million in equity investments from J&J and Pipeline’s existing investors.
PIPE-307 stimulates the maturation of oligodendrocyte precursor cells (OPCs) into myelin-producing oligodendrocytes. These cells produce myelin, the fatty substance that insulates nerve fibers and accelerates electrical signals in the nervous system. The drug candidate promotes the development of OPCs into fully functioning oligodendrocytes capable of producing myelin.
In an interview, Bill Martin, global therapeutic area head of neuroscience at Janssen, delves into the company’s approach to neuroscience and discusses their collaboration with Pipeline Therapeutics, a clinical-stage biopharmaceutical company focusing on neurodegenerative diseases. Martin also provides an overview of the partnerships with two other neuroscience-focused firms: Yumanity, a neurodegenerative disease-focused company that merged with Kineta Bio, and Rapport Therapeutics, a biotech firm specializing in neuroscience.
Martin emphasizes the importance of investing in biomarkers and collaborating with partners to reconceptualize the diagnosis and treatment of nervous system disorders. The company also addresses industry challenges and shares their aspirations for driving advances in precision neuroscience.
Can you give a summary of Janssen’s precision medicine evolution in neuroscience?
Bill Martin
Martin: Janssen’s approach to precision medicine both mirrors the evolution in neuroscience research and builds on our deeply rooted 60-year commitment to developing innovative medicines to treat disorders of the nervous system.
For example, advances in data science and biomarkers are enabling insights into human brain biology. We have invested in platform technologies to support the generation of proprietary insights, including in neurodegenerative diseases and neuropsychiatric conditions.
By leveraging external innovations, within the context of our strong heritage, we aim to reconceptualize how we diagnose and treat targeted disorders of the nervous system. We will do this by using precision approaches in how we identify targets of interest and the patients most suitable for particular treatments, as well as the way we modulate targets and measure therapeutic response.
How does the partnership with Pipeline Therapeutics align with Janssen’s strategy for advancing precision neuroscience?
Martin: The partnership with Pipeline Therapeutics exemplifies two pillars of our approach to innovation: humility and proprietary insights into human biology. The activity of muscarinic receptors contributes to the homeostatic balance of the nervous system and, with PIPE-307, Pipeline identified a molecule with unique properties against a target of high interest to Janssen Neuroscience.
The research and development of PIPE-307 aligns with Janssen’s precision medicine approach and we look forward to sharing more about the strategic positioning of this program as it advances.
What are the key milestones in Janssen’s collaborations with Yumanity and Rapport, and how do they support Janssen’s precision neuroscience vision?
Martin: The Yumanity and Rapport partnerships are completely different. Yumanity built a high-science company, distinguished by an innovative platform to identify novel targets around conserved biology. With the Yumanity deal, we acquired the discovery-stage neuroscience product candidates and targets, including an oral asset with potential disease-modifying properties for Parkinson’s disease which we look to progress through the next stages of clinical development.
By contrast, Rapport Therapeutics was conceived and created through a partnership between Third Rock Ventures and Janssen Neuroscience. The company is based on proprietary technologies developed at Janssen and the premise that these could support the expansion of precision neuromedicines with the right resources and team. Janssen contributed to Rapport a portfolio of neuroscience assets, along with a foundational discovery platform, associated intellectual property and a seasoned scientific team. Rapport is well-positioned for growth and we look forward to following their maturation.
Both partnerships align with our vision to lead the precision revolution in neuroscience to reduce the burden and disability caused by serious nervous system disorders.
What are the significant achievements in Janssen’s collaborations with Yumanity and Rapport, and how do these partnerships align with Janssen’s neuroscience strategy?
Martin: Challenges are part of any opportunity landscape. In drug development, clinical, regulatory and commercial hurdles must be cleared to ensure patients gain access to innovative medicines. The promise of precision is based on using emerging tools to surmount the challenges at each stage of the cycle, but to realize the potential of precision approaches means investing in the development of biomarkers to guide targeted development and commercialization. Janssen cannot do this alone. We aim to lead the neuroscience revolution to reduce the burden and disability caused by neuropsychiatric, neurological and neurodegenerative diseases through research by seeking partnerships with those companies who are involved in the next waves of innovation.
What are Janssen’s objectives in precision neuroscience, and how does the company plan to expand its impact in this area?
Martin: We have always and will continue to begin with the patients and the immense unmet needs that persist across brain diseases. Patients are our inspiration to lead the precision revolution in neuroscience. We will do this by delivering differentiated products. These may originate from our internal R&D teams or from compelling external partnership or most likely from a combination of both.
We will create and leverage advances in precision medicine to meet our ambitious goal: to ensure a future where we can alter the trajectory of nervous system disorders and eliminate the burden they place on global health.