NEW YORK, April 17, 2024 – Genscience LLC, a company applying genetic evidence and proprietary platforms for drug discovery and development and an affiliate of Deerfield Management Company (“Deerfield”), a healthcare investment firm, today announced a new study that shows incorporating genetic evidence into drug design significantly increases the probability of successful development by up to 2.6 times compared to when genetic evidence is absent. The new study appeared in the April 17, 2024 issue of the peer-reviewed journal Nature.¹ The study also documents new findings regarding how different characteristics of genetic evidence impact drug development success.

“These findings further demonstrate that genetic evidence is the most effective means to improve drug discovery and development success rates and R&D efficiency,” said Matt Nelson, PhD, Chief Executive Officer of Genscience and Vice President of Genetics and Genomics, Deerfield Discovery and Development (3DC). “This new data helps illustrate the power of genetic evidence to enrich our understanding of the relationship between a potential drug target and the disease we seek to treat.”

The new study builds on pivotal work Nelson published in 2015 ² and documents which genetic associations are more likely to differentiate successful from unsuccessful drug mechanisms across 18 therapy areas and among five discovery and development phases from preclinical to launched. Nelson and his colleagues found that the probability of success for drug mechanisms backed by genetic evidence is 2.6 times higher than those lacking such support, with a four-fold increase in hematology and metabolic therapy areas.

“This study provides a basis for optimism, that we can improve the success rate in drug discovery,” said Eric Vallabh Minikel, PhD, first author of the study, and senior group leader at the Broad Institute. “Human genetics can point us to the root molecular causes of disease and enable us to design fundamentally disease-modifying therapies.”

Nelson acknowledges that although many questions remain about the application and effectiveness of human genetics in drug discovery, the field of genetics identifies causal relationships between genes and disease in humans, providing a growing opportunity for novel target identification and improving indication selection for existing drugs and drug candidates. Increasing emphasis on drug mechanisms with supporting genetic evidence is expected to increase success rates and lower the cost of drug discovery and development.

“The implications of this study are profound, offering a promising genetic-informed approach for biotechnology and pharmaceutical companies to improve their drug development strategies and increase the likelihood of bringing life-saving medications to market,” said James Flynn, Managing Partner, Deerfield Management. “These findings also demonstrate the meaningful discoveries that advance health and clinical trials that Deerfield, 3DC and Genscience are committed to supporting.”

¹Minikel EV, Painter JL, Dong CC, Nelson MR. Refining the impact of genetic evidence on clinical success. Nature. 2024. DOI: 10.1038/s41586-024-07316-0.

²Nelson MR, Tipney H, Painter JL, et al. The support of human genetic evidence for approved drug indications. Nat Genet. 2015;47(8):856-860. doi:10.1038/ng.3314

About Genscience

Genscience, spun out of Deerfield Management Company and founded by Matthew R. Nelson, PhD, in 2023, is a platform and services company, developing state-of-the science genetics capabilities for the purpose of supporting drug discovery and development across disease areas.

About Deerfield Management

Deerfield is an investment management firm committed to advancing healthcare through investment, information and philanthropy. The Firm works across the healthcare ecosystem to connect people, capital, ideas and technology in bold, collaborative and inclusive ways. For more information, visit www.deerfield.com.

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Genscience contact:

Matthew R. Nelson, PhD

[email protected]

Deerfield contact:

Rachel Pelletier

[email protected]

+1 202-778-1000

NEW YORK– APRIL 9, 2024 – Cure.®, a healthcare innovation campus in New York City, has opened applications to the XSeed Award, an annual competitive program designed to disrupt the status quo by supporting traditionally underfunded and underrepresented minority- and women-led life science and healthcare startups working on drug development in New York City. Deerfield Management, a New York City-based healthcare investment firm, founded and supports the XSeed Award.

In 2024, two winning teams will become eligible to receive up to $250,000 each. They also will join the Cure ecosystem with their fellow awardees to receive entrepreneurshipmentoring and networking designed to help them move promising translational research to the marketplace and commercial success. The deadline for XSeed Award application submissions is May 31, 2024, and virtual finalist presentations will be June 28, 2024. The program will announce winners on Sept. 2, 2024, and they will receive honors at an official ceremony at Cure in October 2024. 

“The XSeed Award is designed to bring equity and diversity to life science and healthcare innovation, where ongoing barriers critically impact the cures developed for underserved communities and the careers of scientists from underrepresented communities. For example, Black and female scientists remain significantly less likely than their white or male colleagues to receive three or more NIH research grants, a threshold that academic institutions prioritize for faculty hires and retention,[i]” said James E. Flynn, Managing Partner at Deerfield and Cure Founder. “Deerfield is pleased to continue our collaboration on the transformational XSeed program, alongside the NYCEDC, Cure and the experts who comprise our enthusiastic XSeed Award Leadership Committee.”

In addition to advancing their research, the 2024 cohort of X-Seed Award winners will enhance their entrepreneurial skills, networks and visibility via the Cure ecosystem. Graduates of each cohort will then serve as mentors and coaches for future cohorts, further strengthening the entrepreneurial community in New York City.

“The Cure ecosystem of industry, investment and innovation experts is an ideal setting to mentor XSeed Award winners and hone their business knowledge to help their life science and healthcare achievements,” said Seema Kumar, CEO of Cure. “We look forward to welcoming the XSeed Award winners who, along with the Cure Xchange Challenge winners, are startups advancing health through collaboration and community building via Cure.”

Deerfield and the New York City Economic Development Corporation launched the XSeed Award as a yearly program in 2020 to provide funding to help address critical funding gaps for the diverse population of most talented researchers and entrepreneurs in New York City.  Advancium Health Network, an independent non-profit organization launched by Deerfield Management and dedicated to advancing healthcare and health equity, has helped operationalize the XSeed Awards since 2022.

“The XSeed Award program aims to break down barriers and create equal opportunity in the life sciences sector by supporting minority- and women-led research and early-stage ventures to help yield the medicines of tomorrow,” said New York City Economic Development Corporation (NYCEDC) Chief Strategy Officer Cecilia Kushner. “As Cure’s ecosystem expands, the XSeed Award program will continue to help establish New York City as both a premier and inclusive hub for the life sciences industry. We are thrilled to be working alongside Deerfield and Cure again to support this important effort.”

XSeed Leadership Committee 

Finalists will present their work to an esteemed panel comprised of the XSeed Award Leadership Committee, which includes Joseph Pearlberg, MD, PhD, Vice President of Scientific Affairs at Deerfield and committee co-chair; Claire Pomeroy, MD, President and CEO of the Albert and Mary Lasker Foundation; Christine Brideau, Head of Preclinical Pharmacology, Deerfield Management; Glennis Mehra, Ph.D., Director of Biolabs@NYULangone; and Jane Williams, MD, MPH, Vice President, head of Neuroscience, Rare Disease and Pediatrics at Syneos Health.

To learn more about the XSeed Award’s applicant eligibility criteria and to submit applications, visit https://wewillcure.com/xseed-award

About XSeed

Deerfield created the XSeed Award program in September 2019 through a partnership with the New York City Economic Development Corporation (NYCEDC) to aid the development of the Deerfield-founded healthcare innovation campus Cure. Deerfield and NYCEDC launched the XSeed Award in December 2020, with the first cohort of winners announced in April 2021.

Since its inception, the XSeed program has helped fill early-stage funding gaps within the life sciences industry by identifying and supporting promising translational research and commercial opportunities of underrepresented and underfunded startups, financially and through a mentorship cohort. To learn more about the XSeed Award, visit https://wewillcure.com/xseed-award.

About Deerfield Management

Deerfield is an investment management firm committed to advancing healthcare through investment, information and philanthropy. The Firm works across the healthcare ecosystem to connect people, capital, ideas and technology in bold, collaborative and inclusive ways. For more information, please visit www.deerfield.com.

About Cure.^®
Cure is a healthcare innovation campus in the heart of New York City that features laboratory and business facilities, a collaboration residency, office space and premium event venues, including an education center, conference center, and iconic rooftop facility, as well as tools, mentoring, networking, and other assistance to members of its ecosystem. Cure houses on-campus startup and established companies. Residents regularly create synergies and collaborative partnerships with peer organizations across the spectrum of healthcare, from academic or private to non-profit or government, and focus on diagnostic, device, drug or vaccine discovery, development and production as well as care delivery and public health. Cure also offers industry-leading event programming focused on critical health topics. Cure’s mission is to foster and accelerate advances in health. For more information, please visit wewillcure.com.

Cure Media Contact:
Caroline Drucker
[email protected]
(917) 588-3016

[i] Nguyen M, Chaudhry SI, Desai MM, Dzirasa K, Cavazos JE, Boatright D. Gender, Racial, and Ethnic and Inequities in Receipt of Multiple National Institutes of Health Research Project Grants. JAMA Netw Open. 2023;6(2):e230855. Published 2023 Feb 1. doi:10.1001/jamanetworkopen.2023.0855.

Genscience, an affiliate of Deerfield, works to integrate genetic insights into drug discovery and development decisions with differentiated experise, platforms, and analytics.

• Evaluate genetic evidence for drug targets and indications
• Characterize genetically defined patient populations and market size
• Conduct novel genetic R&D to fill in scientific gaps
• Identify potential therapeutic targets for indications of interest
• Assess genetic and genomic platform technologies

Genscience works closely with Deerfield and its portfolio companies to provide differentiated expertise, platforms and analytics. This integration aims to strengthen the probability of success and help guide the direction for new discoveries.

New York, NY —  Nov. 8, 2023 — Sfunga Therapeutics (“Sfunga”), a biotechnology company, and Deerfield Management Company (“Deerfield”), a healthcare investment firm, today announced the publication of data on a novel antifungal molecule AM2-19 in the scientific journal Nature.[i] AM2-19 is a derivative of amphotericin (AmB), rationally designed to mitigate toxicities and optimize antifungal activity. AM2-19, formulated as the development candidate, SF001, received Qualified Infectious Disease Product (QIDP) and Fast Track designations from the U.S. Food and Drug Administration (FDA) in 2023, and is in Phase 1 clinical development.

Fungal infections cause more than 150 million severe, life-threatening infections that result in about 1.7 million deaths each year globally.[ii] Invasive fungal infections (IFI) typically occur in people with compromised immune systems due to HIV/AIDS, cancer therapies and organ transplants. These infections also occur in people with severe lung disease, including that caused by influenza and SARS CoV-2. Despite this public threat, only four classes of antifungal medicines are available. Each class has known adverse effects with the potential to put patients’ safety and outcomes at risk. AmB is a naturally occurring small molecule, used since the 1950s as a treatment for people with IFI. The drug has a broad spectrum of activity, but AmB causes kidney impairment in about 80 percent of patients, limiting its use.[iii]

“Patients with invasive fungal infections have few available antifungal treatment options that are effective and safe. Sfunga is working to improve patient outcomes by developing a therapy with all of the antifungal properties of AmB, but with much better safety and tolerability,” said Kieren Marr, M.D., M.B.A., FIDSA, Sfunga co-founder and Chief Medical Officer and adjunct professor of medicine and business at Johns Hopkins University. “We are optimistic about the continued clinical development of SF001, the lead compound of the Sfunga pipeline already in Phase 1 clinical development.”

Sfunga was formed in 2019 as a collaborative venture between Martin Burke M.D., Ph.D., senior author of the Nature paper and professor of chemistry at the University of Illinois at Urbana-Champaign, and Deerfield.  Clinical development was initiated in 2023, under Marr’s direction. Sfunga has completed SF001’s first in human single-ascending dose study and is poised to begin a multiple-ascending dose study in 2024.

“The innovative development of SF001, published in Nature today, stems from leveraging highly valuable discoveries about the structure and function of AmB,” said James Flynn, Managing Partner, Deerfield Management and Cure^® Founder. “Our collaboration to create Sfunga furthers Deerfield’s commitment to providing tailored funding, infrastructure, and support needed for successful commercial development of novel discoveries that advance health.”

With nearly three decades of experience in healthcare investment, Deerfield generally maintains a portfolio of more than 150 private and public investments across the life science, medical device, diagnostic, digital health and health service industries at all stages of evolution from start-up to mature company.

SF001: Rationally designed for optimal safety and efficacy

To develop AM2-19, Burke and his colleagues determined how AmB kills fungi and damages kidney cells using solid-state nuclear magnetic resonance imaging. They found that AmB’s mechanism for both actions involves creating an extracellular ”sponge” that extracts critical sterol molecules from the membranes of cells. AmB binds to ergosterol in fungi and cholesterol in human kidney cells, ultimately contributing to cellular death.

Additional high-resolution atomic-level analyses provided insights into the binding interactions between AmB and each sterol. The resulting information guided the investigators’ development of AM2-19’s structure and shape as a new molecule that could rapidly extract fungal ergosterol without harmful toxicity to human cells.

In laboratory studies, AM2-19 bound to ergosterol but not cholesterol, demonstrating decreased toxicities, with potent efficacy against pathogenic yeasts and moulds in vitro and in animal studies.

The research reported in Nature received support from Sfunga; the National Institutes of Health under grants 5R01-AI135812-04, R35-GM118185, R01-GM112845 and R01-GM123455, R01-AI063503 and P4-GM136463; and the Roy J. Carver Charitable Trust.

Arun M, Soutar C, Zhang J, et al. Tuning sterol extraction kinetics yields a renal sparing polyene antifungal. Nature. 2023. doi: 10.1038/s41586-023-06710-4.

Fungal Infections Threaten Global Public Health

Fungi are organisms made of one cell, like yeasts, or many cells, such as moulds or mushrooms. Fungi naturally exist in the environment and the human body. The World Health Organization (WHO) designated 19 fungi as priority pathogens in 2022, naming four as critical and of the highest importance to public health: Cryptococcus neoformans, Candida auris, Aspergillus fumigatus and Candida albicans.[iv]

WHO reports that fungal diseases are expanding in their incidence and geographic range globally because of climate change, increased international travel and trade, and the impact of the COVID-19 pandemic, among other factors. Also, both yeasts and moulds are increasingly reported to develop resistance to available antifungals, limiting treatment options and contributing to poor outcomes.

About Sfunga

Sfunga Therapeutics is a cutting-edge biotechnology company dedicated to transforming the treatment of life-threatening fungal infections using a novel approach rooted in structural chemistry. Sfunga was founded under the belief that mechanistic insights enable targeted optimizations of natural products. Today, Sfunga is advancing the first polyene antifungal rationally designed to mitigate toxicities and increase efficacy. For more information, please visit www.sfunga.com.

About Deerfield Management

Deerfield is an investment management firm committed to advancing healthcare through investment, information and philanthropy. The Firm works across the healthcare ecosystem to connect people, capital, ideas and technology in bold, collaborative and inclusive ways. For more information, visit www.deerfield.com.

Sfunga contact:

Kieren Marr, M.D., M.B.A., FIDSA [email protected]

Deerfield contact:

Elise Wang [email protected] Phone: (212) 583-7267

Endnotes

[i] Arun M, Soutar C, Zhang J, et al. Tuning sterol extraction kinetics yields a renal sparing polyene antifungal. Nature. 2023. doi: 10.1038/s41586-023-06710-4.

[ii] Kainz K, Bauer MA, Madeo F, Carmona-Gutierrez D. Fungal infections in humans: the silent crisis. Microb Cell. 2020;7(6):143-145. Published 2020 Jun 1. doi:10.15698/mic2020.06.718.

[iii] Noor A, Preuss CV. Amphotericin B. National Library of Medicine. StatPearls. January 2023. Updated March 24, 2023. https://www.ncbi.nlm.nih.gov/books/NBK482327/.

[iv] WHO fungal priority pathogens list to guide research, development and public health action. Geneva: World Health Organization; 2022. License: CC BY-NC-SA 3.0 IGO. https://iris.who.int/bitstream/handle/10665/363682/9789240060241-eng.pdf?sequence=1.