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Debra Miller co-founded CureDuchenne in 2003 with her husband, Paul, after their only son was diagnosed with Duchenne. Miller relies on her extensive background in sales and marketing to lead CureDuchenne. 

Her role includes overseeing all operations, research and financial resources as well as serving as the primary liaison to the Board of Directors. She is the decision maker, leader and manager in carrying out the mission of the organization. She also works closely with the Scientific Board of Advisors in designing and implementing the medical research strategic plan.

Miller is a member of the Department of Defense Duchenne Research review board and also a member of the TREAT-NMD international review board, TACT.  Miller has led CureDuchenne’s successful venture philanthropy programs and is the architect of CureDuchenne Ventures LLC, a new, for profit entity created to attract significant research funding to Duchenne research.

Prior to CureDuchenne, Miller had a career in publishing with positions at IDG Communications, Cahners Publishing, Ziff-Davis Publishing and Scholastic Publishing. She also worked in management at PC Magazine and was an independent stock trader.

Miller earned a Bachelor of Arts in Communication Studies from the University of California – Los Angeles. 

Diamond BioFund is an evergreen fund that was established in 2013 and is based in Taipei City, Taiwan. The fund is operating from a committed capital of USD 300 million. The fund makes equity investments in early- to late-stage private businesses. Typical allocations range from USD 1.5 million to north of USD 10 million. The fund likes to lead investments in Taiwan-based businesses and co-invest in businesses overseas. The fund is currently seeking new opportunities in Taiwan, Greater China, Southeast Asia, US, Canada, and EU.

Diamond BioFund takes a generalist approach when considering life science opportunities, including therapeutics (small molecule and biologics), medical devices, diagnostics, healthcare services, and nutraceuticals. The fund is seeking cutting edge, innovative, and platform technology. In therapeutics, the fund considers all stages from preclinical through pre-IPO. In medical devices, the fund prefers later stage projects with proof of concept. The fund is open to all disease indications.

Easton Capital Investment Group is a venture capital firm based in New York. Easton Capital invests broadly in the life science sector, and considers investment opportunities globally. The firm is likely to make 4-6 allocations in the next 6 months. Allocations typically vary from $250,000 to $5 million, in the form of equity or convertible debt. Allocations are typically made at the venture stage of development, but may be made in seed rounds if a company already has proof of their product's efficacy.

EIP Pharma, LLC is a private company based in Cambridge, MA.  The company has licensed-in and is developing a phase 2 clinical-stage oral small molecule that targets neuroinflammation and dysfunction of microglia, the major immune-cell in the brain.

Human genetic and other biologic data strongly indicate inflammation & microglial dysfunction are one of the major drivers of late-onset Alzheimer’s disease (AD), and likely the major driver in patients with symptomatic disease.

VX-745 (the licensed compound) specifically inhibits intra-cellular enzyme p38 mitogen activated protein kinase alpha (MAPKa).  In the brain, p38 MAPKa is a major regulator of inflammation through effects on microglia. P38 MAPKa is also expressed in neurons, where it is directly involved in memory formation and synaptic plasticity. 

VX-745 readily enters the brain, with brain concentrations in pre-clinical studies being approximately two-fold higher than in peripheral blood.  VX-745 had previously been clinically evaluated in non-CNS disorders, where already has demonstrated anti-inflammatory activity in patients. The combination of blood-brain-barrier penetration and previous clinical experience with demonstrated anti-inflammatory activity uniquely positions VX-745 to target inflammation to treat Alzheimer’s and other CNS disorders in which inflammation plays a role.

EIP Pharma licensed VX-745 from the originator company, Vertex Pharmaceuticals, and conducted animal studies to re-position the compound towards disease of the brain, including AD.  Vertex had previously completed a full chronic toxicology program and had demonstrated significant clinical and anti-inflammatory activity in a phase 2a 12-week treatment study of VX-745 in rheumatoid arthritis (RA).  An Investigational New Drug (IND) application remains open at FDA.

The animal studies conducted by EIP Pharma have demonstrated pro-cognitive effects, anti-inflammatory and other pharmacologic effects of VX-745 in the brain.  These data combined with dose response data in prior animal and clinical studies, have been utilized to identify doses that are highly likely to have positive activity in human brain.  The predicted doses are 2- to 5- fold lower than the dose that demonstrated anti-inflammatory activity and was well tolerated in RA, consistent with VX-745 preferentially distributing to the brain in animals.  Importantly, these dose levels should minimize risks of systemic toxicity that otherwise have hampered development of p38 MAPK inhibitors for peripheral disorders.  The animal studies have also generated new intellectual property (IP), including issuance of a US patent on 15 April 2014 (#8,697,627) for the use of VX-745 to lower brain amyloid plaque load.  This IP, along with additional IP that has been filed will support commercially the development of VX-745 beyond the primary composition of matter patent expiring in 2017.

Start-up activities (IRB submissions, CRO contracting, etc.) are underway for two phase 2a clinical studies in patients with AD; both studies are anticipated to be underway in early 2015.  Combined, the studies are designed to demonstrate anti-inflammatory and microglial modulatory activity in the brain.  Once demonstrated, VX-745 should have ability to demonstrate clinical proof-of-concept in AD and/or other disease indications in which neuroinflammation plays a role in disease pathogenesis.

Company was founded and is led by experienced biotech/pharma R&D executive (John Alam; former head of Alzheimer’s R&D at Sanofi, ex-Chief Medical Officer at Vertex Pharmaceuticals, led clinical development of AvonexÒ for Multiple Sclerosis) with extensive translational and drug development experience & expertise in both inflammation and CNS disorders.

Enso Ventures is a Venture Capital firm that was founded in 2010 and based in London with an additional office in New York. The firm is looking to provide equity capital to high growth companies in the life science space. The firm is currently interested in companies located in Europe, Russia and the US and is looking to make approximately 2-3 investments over the next 6-9 months.

Excel Venture Management is a venture capital firm that was founded in 2008 and is based in Boston, Massachusetts. The firm has managed one fund with committed capital of $125 million, and the firm is currently raising the second fund. Excel Venture Management seeks to make equity investments into life science companies from early to late stages. The typical investment size ranges from $3 million to $ 5million. The firm plans to invest in 4-5 companies over the next 6-9 months and prefers to invest in companies based in US.