Mesentech and CureDuchenne have announced a partnership that includes generous funding from the Charles H. Hood Foundation. The partnership has developed a targeted therapy for reversing bone wastage in boys diagnosed with Duchenne muscular dystrophy.
CureDuchenne Ventures, the research funding arm of CureDuchenne, a leading global nonprofit dedicated to finding and funding a cure for Duchenne muscular dystrophy (DMD), announced an investment in Mesentech Inc., a regenerative medicine company with a prodrug platform that selectively delivers therapeutics to bone. The investment is part of a new joint funding collaboration with the Charles H. Hood Foundation (CHF) that looks to advance early-stage research for pediatric conditions.
The investment supports Mesentech’s lead program, MES-1007, into clinical development and its evaluation in Duchenne muscular dystrophy. Pediatric osteoporosis is a significant problem for DMD patients, leading to frequent fractures and premature loss of ambulation. There are currently no approved therapies for bone wastage for individuals affected by Duchenne. There is tremendous potential for MES-1007 to be the first targeted therapy to specifically address bone wastage in these individuals.
“We are excited about the potential impact Mesentech’s bone regenerative treatment will have for the Duchenne community,” said Debra Miller, founder, and CEO of CureDuchenne. “By collaborating with CHF on this investment, we can greatly increase the impact of our venture philanthropy model. Addressing bone morbidities that contribute to the loss of ambulation could significantly add quality of life to everyone affected by Duchenne.”
“The Hood Foundation is proud to join CureDuchenne in delivering capital at a critical juncture for a novel approach to address a great need for those living with Duchenne,” added John Parker, a CHF trustee and managing director of the foundation’s program-related investment fund. “Mesentech’s program fits perfectly with our investment objective of backing game-changing innovation that improves outcomes for children afflicted by disease.”
In addition to the funds provided, CureDuchenne will play an active role in helping the company advance their prodrug technology platform for Duchenne. As part of the transaction, CureDuchenne’s Chief Scientific Officer, Michael Kelly, PhD, will be added to Mesentech’s scientific advisory board.
“We are delighted that CureDuchenne and the Hood Foundation see the great potential to the DMD community of MES-1007 treatment and to welcome Dr. Kelly to our scientific advisory board,” said Jonathan Polak, Mesentech’s CEO. “The investment by CureDuchenne Ventures and the Hood Foundation supports the bone regenerative potential of MES-1007 and our bi-specific prodrug technology platform. We look forward to working with Dr. Kelly and the rest of the CureDuchenne team to evaluate MES-1007 bone growth potential in DMD.”
Dr. Kelly offered additional insight into MES-1007 and its bone growth potential via email:
MES-1007 is a bi-specific prodrug. What is the difference between bi-specific and mono-specific compounds (drugs)?
Dr. Kelly: MES-1007 is a bi-specific drug-conjugate consisting of two parts:
- A targeting moiety that uses a novel proprietary molecule that delivers the conjugate to bone, and
- A drug moiety that delivers a PGE2 prostaglandin analogue to activate the EP4 receptor specifically on bone when released from the conjugate.
Typical prodrugs are designed to improve specific properties of drugs such as bioavailability, pharmacokinetics, or half-life. The bi-specific drug conjugate combines this property with improved targeting to the tissue of interest.
What is the advantage of MES-1007 as a bi-specific prodrug?
This novel conjugate has no biological activity on its own, but is designed to only release its active drug component at the target site (bone). This specifically reduces the multiple side-effects associated with the activity of the drug in other (non-target) tissues and greatly improves its efficacy while reducing toxicity and mechanism-related side-effects.
What is it about your work on this program that is the most encouraging and/or what is most challenging?
Mesentech’s experimental drug still must undergo additional studies before it can be tried in individuals with Duchenne. But if successful, it could be the first bone-restoring drug to be tested in a Duchenne-specific clinical trial, and a significant opportunity to address an important component of the Duchenne disease process in an impactful way. When bones have reduced density, they become brittle and prone to fracture. Studies have indicated that in Duchenne, at least half of individuals will sustain bone fractures due to low bone density. Fractures in the vertebral bones of the spine are seen in up to 30% of boys with Duchenne, can be very painful, and if left untreated can lead to spine deformity. Fractures in one of the long bones in the arm or the leg are even more common, and in some cases can lead to permanent loss of ambulation.
How do you see the future for Duchenne muscular dystrophy?
Our focus is to treat the whole disease, improve existing therapies, and accelerate the development of new approaches that target the underlying cause of Duchenne to treat all patients independent of their genetic mutation.
The next few years could witness new drug approvals that target specific components of the disease such as, for example, micro-dystrophin gene therapy, novel anti-inflammatory agents, and “next-generation” improved exon-skipping drugs.
About Duchenne Muscular Dystrophy
Duchenne is a severe X-linked form of muscular dystrophy that affects approximately 1 in 5000 males. DMD is caused by the absence of the dystrophin protein. Dystrophin is a large protein that provides multiple cellular functions and helps to protect skeletal and cardiac muscle against injury, inflammation, and fibrosis. Individuals with Duchenne show progressive muscular degeneration and lose the ability to walk by early to mid-teens and progress to a full loss of upper body function. Cardiopulmonary complications are the primary cause of death.
About CureDuchenne
CureDuchenne is recognized as a global leader in research, patient care, and innovation for improving and extending the lives of those with Duchenne muscular dystrophy. As the leading genetic killer of young boys, Duchenne affects more than 300,000 individuals living today. CureDuchenne is dedicated to finding and funding a cure for Duchenne by breaking the traditional charitable mold through an innovative venture philanthropy model that funds groundbreaking research, early diagnosis, and community education. For more information on how to help raise awareness and funds needed for research, please visit www.cureduchenne.org.
Webinar: Mesentech and The Importance of Bone Health.
CureDuchenne will also be hosting a webinar with Mesentech on December 10th, entitled Mesentech and The Importance of Bone Health. The community is encouraged to join the webinar and learn directly from Mesentech about their novel therapeutic approach for improving bone density and strength.
About the Charles H. Hood Foundation
Since 1942, the Charles H. Hood Foundation has carried on the legacy of founder Charles H. Hood by funding groundbreaking and innovative pediatric research. After decades of successful grant programs, the Foundation broke new ground in 2015 by establishing its program-related investment fund to support entrepreneurial efforts to significantly improve the health outcome of children. Through both its grants and investments, the Foundation has made meaningful change possible by filling the gaps in the medical research and innovation funding marketplace. To learn more about the Hood Foundation please visit CharlesHoodFoundation.
