Duchenne Muscular Dystrophy Prevalence Increases, While Incidence Remains Steady

Uptick attributed to improved treatments and longevity

New York, NY, May 23, 2019—In the first study of its kind involving Duchenne Muscular Dystrophy (DMD) in the U.S., researchers from the Deerfield Institute found that while the number of new cases has remained stable, there has been an uptick in prevalence—largely attributed to enhanced treatments and longevity. The study, which is titled “Duchenne Muscular Dystrophy Prevalence in the U.S.: A Novel Incidence-Based Modeling Approach Using System Dynamics”, was featured in a recent poster session at the ISPOR 2019 annual meeting in New Orleans.

DMD, a genetic disorder characterized by progressive muscle degeneration and weakness, is caused by an absence of Dystrophin, a protein that helps keep muscle cells intact. Symptom onset is in early childhood, typically between ages 3 and 5. The disease primarily affects boys, but in rare cases it can affect girls.

Using a triangular distribution of incidence rates identified in the literature2,3, a sensitivity analysis was run to estimate the diagnosed incidence of DMD in the U.S. at 17.24 per 100,000 live male births, corresponding to approximately 362 incident cases in 2019; diagnosed prevalence was found to be 6.09 per 100,000 male population across all age groups, corresponding to about 10,015 prevalent cases in 2019.

The Deerfield Institute researchers found that while the majority (64.5%) of DMD patients are under the age of 20, there is a significant number of older DMD patients up to 45 years of age that were excluded from previous prevalence estimates. 4 The prevalence of DMD among males, aged 45 or younger, was found to be 10.0 per 100,000 vs previously estimated prevalence estimates of 1.38 per 10,000 among males 5 to 24 years of age.  

“We hypothesized that the prevalence of DMD has increased over the past few decades, due predominantly to improvements in treatment and care” said Emma Giegerich, MPH, an epidemiologist with the Deerfield Institute and co-author of the study. “Our incidence-to-prevalence model was built using system dynamics principles and birth-cohort-specific survival curves to get the most accurate picture of the disease landscape and its current burden. The results indicate that there is a larger than expected patient population that may benefit from novel treatment interventions, such as targeted gene therapies, potentially improving the viability of current or future drug development programs.”

The study was co-authored by Mark Stuntz, MPH, a former Deerfield Institute investigator.

1 Retrieved from https://www.mda.org/disease/duchenne-muscular-dystrophy.
2 Dooley J, Gordon EK, Dodds L, MacSween J. Duchenne muscular dystrophy: a 30-year population-based incidence study. Clinical Pediatrics 2010;49(2):177-179.
3 Mendell JR, Shilling C, Leslie ND, Flanigan KM, al-Dahhak R, Gastier-Foster J, Kneile K, Dunn DM, Duval B, Aoyagi A, Hamil C, Mahmoud M, Roush K, Bird L, Rankin C, Lilly H, Street N, Chandrasekar R, Weiss RB. Evidence-based path to newborn screening for Duchenne muscular dystrophy. Ann Neurol 2012;71:304-313. 
4 Romitti PA, Zhu Y, Puzhankara S, James KA, Nabukera SK, Zamba GK, Ciafaloni E, Cunniff C, Druschel CM, Mathews KD, Matthews DJ, Meaney FJ, Andrews JG, Conway KM, Fox DJ, Street N, Adams MM, Bolen J, STARnet MD. Prevalence of Duchenne and Becker muscular dystrophies in the United States. Pediatrics. 2015;135(3):513–21.

About the Deerfield Institute

The Deerfield Institute is the research division of Deerfield Management, a health care investment management firm committed to advancing health care through investment, information and philanthropy.

Contacts

Karen Heidelberger, [email protected], 212-551-1600

Deerfield Leads Investment of up to $50 Million in Sollis Therapeutics’ Innovative Solution for Sciatica Pain

New York, NY and Columbus, OH – April 12, 2018 – Deerfield Management and Sollis Therapeutics today announced a Deerfield‐led investment of up to $50 million in Sollis Therapeutics. The investment will support the development of an innovative non‐opioid and non‐steroid solution for pain associated with sciatica and other neuropathic pain syndromes. Sollis’ treatment is an extended release analgesic administered through a traditional epidural injection procedure.

The total equity financing of up to $50 million was led by Deerfield’s commitment of over $40 million and will fund the company through FDA approval. The funding will also support the development of other products in the Sollis pipeline.

“Deerfield’s support and partnership is a pivotal step in our development of a non‐opioid option for sciatica pain,” said Gregory Fiore, MD, CEO of Sollis Therapeutics. “This investment will allow us to bring to market a transformational treatment for sciatica, a condition with more than 5 million sufferers annually in the United States and with no FDA approved medications.”

“Sollis’ innovative treatment has the potential to provide immediate pain relief and long‐term efficacy for millions of patients suffering from sciatica and other neuropathic pain conditions. We are pleased to be partnering with the Sollis team toward the advancement of this innovative technology which will help eliminate the gateway to drug abuse and opioid addiction,” stated Andrew ElBardissi, MD, Principal of Deerfield Management.

About Sollis Therapeutics

Sollis Therapeutics is a Columbus Ohio based clinical‐stage biotechnology company focused on developing novel non‐steroid, non‐opioid analgesics. Sollis’ first product is an extended release combination analgesic and anti‐inflammatory medication purpose‐built for targeted local delivery for the treatment of sciatica and other neuropathic pain syndromes. The product will soon be entering into a pivotal clinical trial at multiple centers throughout the United States.

To learn more about Sollis, please visit www.SollisTX.com.

About Deerfield

Deerfield is an investment management firm committed to advancing healthcare through investment, information and philanthropy.
For more information, please visit www.deerfield.com.

Contacts

Sollis Therapeutics
[email protected]

Deerfield Management Company
Karen Heidelberger, 212‐692‐7140
[email protected]

Sollis Therapeutics

In April 2018, Deerfield invested in Sollis Therapeutics. Sollis is developing a drug / device combination consisting of Polylactic acid + Clonidine for the treatment of sciatica. Current treatment options consist of opioids, NSAIDs, and epidural injections, each of which have minimal therapeutic benefit. The PLA + Clonidine combination results in controlled bioresorbability and distribution over a 52 week period, creating a longer term benefit.

MD Anderson and Deerfield Management create Vescor LLC to develop novel therapeutics based on inhibiting autophagy for treatment of specific cancers

Houston and New York – January 11, 2017 ― Vescor LLC, a new company focused on discovery and development of autophagy targeted therapeutics for cancer treatment, has been formed by The University of Texas MD Anderson Cancer Center, Deerfield Management and two leading autophagy experts, Eileen White, Ph.D., deputy director and associate director for Basic Science, Rutgers Cancer Institute of New Jersey, and Alec Kimmelman, M.D., Ph.D., chairman,Department of Radiation Oncology at NYU Langone Medical Center and a member of the Perlmutter Cancer Center at NYU Langone.

Vescor, advised by its scientific founders White and Kimmelman, whose research has shown inhibition of autophagy can dramatically impact tumor growth in pre-clinical models, will develop small molecule inhibitors of a number of protein targets at critical nodes of the autophagy cascade, perform investigational new drug (IND) enabling studies, and move these into clinical development. MD Anderson’s Institute for Applied Cancer Science (IACS) in combination with Deerfield will provide drug discovery and development expertise, together with translational research focused at advancing autophagy therapeutics into trials in melanoma, lung and pancreatic cancers.

Autophagy is a cellular process that plays an important role during the development and maintenance of tumors, providing them with the metabolic plasticity to meet their energetic needs, and requirements for cellular building blocks. It allows tumors to scavenge nutrients to sustain growth and survival. Autophagy also has been demonstrated as critical to therapeutic resistance, and is upregulated during cancer treatment in response to chemotherapy and radiation.  

 “Nutrient scavenging by autophagy is a process that tumors hijack to meet their energetic requirements and to provide the necessary cellular building blocks for growth in a stressed tumor micro-environment”said White, “Preclinical models have demonstrated a critical role for autophagy in multiple cancer types.”

Autophagy has been known for over 50 years but its fundamental importance in physiology and medicine was only recognized after Yoshinori Ohsumi’s paradigm-shifting research in the 1990’s that allowed the detailed study of the process for which he was awarded this year’s Nobel Prize in physiology or medicine.

“Numerouspre-clinical studies have now shown a requirement for autophagy in sustaining tumor growth, including pancreatic cancer, as well as in providing a mechanism of resistance to a number of currently used therapies” said Kimmelman. “There is a need for potent and specific autophagy inhibitors to advance these important findings into clinical practice.”

Increating Vescor, Deerfield and MD Anderson aim to perform drug discovery and development in a markedly different way than traditional oncology start-ups.Core activities will be performed at IACS, which provides deep drug discovery and translational research expertise and capabilities, while managerial and operational expertise is provided by both IACS and Deerfield.

“We are excited to join forces with Drs. White and Kimmelman, two world-leading autophagy experts,” said Phil Jones, Ph.D., IACS’s executive director and head of drug discovery. “Our goal is to drive novel drug discovery paradigms and develop cancer therapeutics targeting unexplored mechanisms that might provide high clinical impact. We are delighted to partner in this effort to advance novel drugs towards the clinic.”

Vescor’s goal is to generate at least one IND-ready asset in a condensed time frame. This capital-efficient model is possible due to the availability of 100 percent of dedicated resources advancing product development efforts. 

“We believe Vescor will identify opportunities to significantly advance the treatment of cancer through manipulating autophagy and thus restore cellular homeostasis leading to the potential reduction of cancer disease burden,” stated William Slattery, Partner at Deerfield. “We look forward to working with Drs. White and Kimmelman and MD Anderson to expedite the novel biology of autophagy and to develop drugs that have the ability to impact the lives of patients.”

About Deerfield

Deerfield is an investment management firm committed to advancing healthcare through investment, information and philanthropy.

About MD Anderson

The University of Texas MD Anderson Cancer Center in Houston ranks as one of the world’s most respected centers focused on cancer patient care, research,education and prevention. The institution’s sole mission is to end cancer for patients and their families around the world. MD Anderson is one of only 45 comprehensive cancer centers designated by the National Cancer Institute (NCI).MD Anderson is ranked No.1 for cancer care in U.S. News & World Report’s“Best Hospitals” survey. It has ranked as one of the nation’s top two hospitals since the survey began in 1990, and has ranked first for nine of the past 10 years. MD Anderson receives a cancer center support grant from the NCI of the National Institutes of Health (P30 CA016672).

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Contacts

MD Anderson
Ron Gilmore
713-745-1898
[email protected]

Deerfield Management
Karen Heidelberger
212-692-7140
[email protected]

Vescor LLC

In January 2017, Deerfield and MD Anderson Cancer Center entered into a strategic collaboration to form Vescor Therapeutics. Vescor is focused on discovery and development of autophagy targeted therapeutics for cancer treatment. Autophagy is a cellular process that plays an important role during the development and maintenance of tumors. It allows tumors to scavenge nutrients to sustain growth and survival. Autophagy also has been demonstrated as critical to therapeutic resistance, and is upregulated during cancer treatment in response to chemotherapy and radiation.

Deerfield Announces Lead Investment in Visionary Anti-Fibrotic Company Blade Therapeutics

New York, New York – June 16, 2016 –DeerfieldManagement Company today announced its lead investment in the visionary anti-fibroticcompany Blade Therapeutics. Blade Therapeutics is developing cutting edge therapies to treat patients with diverse fibrotic diseasesfor which there are currently no remedies. The proceeds from the $45 millionSeries B financing will be used to advance the selection and development of a leadmolecule.

Blade Therapeutics exploits novel targetbiology to halt the progression of fibrosis. The technology is licensed fromJohns Hopkins University School of Medicine and is based on the research from thelaboratory of Harry (“Hal”) Dietz, M.D., the Victor A. McKusick Professor ofGenetics in the Institute of Genetic Medicine at Johns Hopkins University School of Medicine.

“Weare excited to support the ground-breaking science developed at Johns Hopkins.  It truly has the potential to change the courseof fibrosis and provide disease altering therapies to patients,” stated WilliamSlattery, partner at Deerfield Management.

“Fibrosis,in its many forms, is a clear unmet need and affects a diverse number ofpatients,” commented Cameron Wheeler, principal at Deerfield Management Company. “The chemistry and biology developed bythe Blade team has created a new path to a potential cure.”

WendyeRobbins, MD and CEO of Blade said “We are looking forward to having Deerfieldas an investor in Blade. Deerfield has shown an ability to provide supportbeyond capital which we believe will leverage our capabilities as Blade growsboth scientifically and operationally. We are also excited to welcome PfizerVentures, Bristol Meyers Squibb, and Novartis to the syndicate. We believe thatwith the leadership of this premier team of investors in addition to ourexisting shareholders we will be in a position to build the leading fibrosiscompany.”

About Blade Therapeutics

Blade Therapeutics is a privatebiopharmaceutical company revolutionizing the treatment of fibrotic disease.Blade is exploiting novel target biology to halt the progression of fibroticdisease. The company is committed to building the leading company in anti-fibrotic drug discovery anddevelopment.  Blade began operations inthe third quarter of 2015 with a license to ground-breaking research from Harry(“Hal”) Dietz, M.D.at Johns HopkinsUniversity School of Medicine and investment from MPM Capital. The company’s talentedand dedicated team of scientists are committed to the discovery and developmentof novel therapies that can modify the course of multiple fibroticdiseases.  Please visit www.blademed.com for more information.

About Deerfield

Deerfield is an investment management firmcommitted to advancing healthcare through investment, information andphilanthropy.

For more information, please visit www.deerfield.com

Contacts

DeerfieldManagement Company
Karen Heidelberger, 212-692-7140
[email protected]

Blade Therapeutics

Blade Therapeutics is developing cutting edge therapies to treat diverse fibrotic diseases for which there is currently no cure. The technology comes out of Johns Hopkins and is based on research from the laboratory of Hal Dietz, M.D., the Victor A. McKusick Professor of Genetics in the Institute of Genetic Medicine and the Johns Hopkins University School of Medicine.

Aziyo

Aziyo is a regenerative medicine platform company created by spinning out and capitalizing the musculoskeletal division of Tissue Banks International, a non-profit organization. The company brings together two seasoned management teams from the wound care industry from Advanced BioHealing and Osiris. Aziyo is a commercial stage company with multiple products.

Conventus Orthopaedics

Deerfield led and participated in Conventus Orthopaedics, Inc. $24 million series AA equity financing. Conventus is a device company solely focused on improving patient care through a platform technology with less invasive solutions to a broad range of challenging perarticular fractures.

Alphatec Spine

Alphatec Spine, Inc. is a medical device company that designs, develops, manufactures and markets physician-inspired products and solutions for the treatment of spinal disorders associated with trauma, congenital deformities, disease and degeneration. Deerfield entered into a financing with Alphatec to provide up to $50 million to satisfy certain obligations arising from litigation and growth capital for future commercial activities.