• MDA Request for Applications: Drug Development in Neuromuscular Disease

    MDA Venture Philanthropy

    MDA Venture Philanthropy (MVP) is the Muscular Dystrophy Association's drug development program, which operates within MDA's Translational Research program. MVP is exclusively focused on funding the discovery and clinical application of treatments and cures for neuromuscular diseases.

    Adapting elements of the venture capital model, the MVP business plan is characterized by an emphasis on measurable results along with deep involvement by its scientific and industry advisers. MVP evaluates and makes targeted investments in for-profit and not-for-profit companies and academics developing therapeutics for neuromuscular diseases.

    Building upon MDA's long-term investment in research and health care, MVP is designed to complement MDA's ongoing programs of health care, lifesaving services, advocacy, basic and clinical research, and professional and public health information. MVP also benefits from MDA's other research programs that support basic research, clinical trials and research infrastructure.

    Business Plan

    A strategy for lowering barriers to therapeutic development: MVP leverages MDA's years of expertise in research into nerve and muscle biology to create a unique environment to foster translation of research into therapies. Dedicated staff scientists track research developments from MDA's basic research program and from the scientific community as a whole and match the most promising ideas with drug development companies capable of developing such therapies. Where companies have promising therapeutics, MVP helps match the company with disease experts to expedite and streamline development. Where academic projects need more data before they will be attractive to a corporate partner, MVP helps match the researcher with experts in drug development. As a "partner" and not just a funder, MVP will help to cultivate follow-on investors, while its access to patients, experts and research infrastructure can offset some costs of drug development.  

    Bridging the high-risk stages of therapy development: MVP makes targeted investments in projects for which the therapeutic of interest is unlikely to advance via traditional funding avenues due to the perceived risk of a small market, lack of a clear regulatory path or new technology. MVP funds are leveraged to aid getting projects through the critical stages of development to ensure that successful therapies attract funding from other sources and get to market. Co-investors and follow-on funders with complementary skills are cultivated actively in the form of venture capital groups, other nonprofits, and larger biotechnology and pharmaceutical companies. Exit points for MVP funding may include the situations in which 1) a therapeutic progresses to phase III or is approved; or 2) significant funding from follow-on investor(s) is received. Return on investment strategies for MVP may include revenue sharing from licensing agreements or commercialization of the therapeutic, or less commonly, sales of preferred stock.

    MVP uses elements of the venture capital model: MVP uses an iterative evaluation process that reviews the science, management, financials, legal issues and intellectual property of the company or project. MVP holds monthly teleconferences open to its advisers at which decisions to advance projects to the next stage of evaluation are made. In addition to the diverse qualifications of MVP's program staff, advisory committees and ad hoc scientific experts, MVP may utilize well-established outside contractors for formal financial and legal diligence. MVP manages its grants as if they were investments such that funding commitments are structured as milestone-driven contracts.

    A unique blend of staff, contractors and volunteers: MVP has access to a unique set of expertise that it uses to select projects to invest in and to manage those projects. The scientific advisers consist of experts in neuromuscular disease research, neuromuscular medicine, biotechnology venture capital investment and drug development. MVP staff scientists work with these volunteers to select projects with the best possible chance of success, and to act as advisers as those projects move forward. MVP uses its business advisers and outside contractors for legal and financial diligence and for contract negotiation, ensuring that MVP invests only in projects likely to have the greatest possible impact. The business advisers are volunteers with expertise in mergers and acquisitions, biotechnology investing, investment banking and corporate development.

    For information, visit the MDA website: https://www.mda.org/research/mda-venture-philanthropy

    For funding inquiries, contact Grace Pavlath, Ph.D., Senior Vice President & Scientific Program Director at gpavlath@mdausa.org.

  • The Lawrence and Isabel Barnett Drug Development Request For Proposal

    Significant Dates:

    RFP Open
    June 8, 2018

    Preliminary Proposal Due
    July 6, 2018

    Preliminary Proposal Review
    [review comments will not be available]

    Request To Submit Full Application Notification
    July 20, 2018

    Submission Of Full Application Due
    September 4, 2018

    Full Application Review
    [review comments will be available]

    Notification Of Awards With Grant Agreement
    November 2018

    Funding commences on receipt of all relevant signatures on the grant agreement

    Instructions for submission:

    Download Preliminary Proposal form here.

    Submit the preliminary proposal via email to researchgrants@alsa-national.org

    Program Description:

    There are currently three FDA-approved drugs for the treatment of Amyotrophic Lateral Sclerosis (ALS). Riluzole, (Rilutek®) which improves survival by two to three months which is marketed globally, and edaravone (Radicava™), which is approved in the United States and Japan. Edaravone lessens functional decline as measured by Amyotrophic Lateral Sclerosis Functional Rating Scale. The effect of edaravone on survival is unknown. Neudexta™ provides symptomatic relief of emotionality in ALS. Additional treatments are available that relieve the symptoms associated with ALS and improve the quality of life for patients living with the disease by providing comfort to the patient. However, there is an urgent need for new and improved therapies. With the recent progress in understanding ALS, the increased effort to develop tools to identify novel treatments for the disease and advances in technology, the opportunity to discover improved treatments for ALS could not be better.

    The ALS Association’s TREAT ALS™ program funds research from early target identification to preclinical research and early pilot clinical trials. As part of the program, The Association is requesting Preliminary Proposals for its Lawrence and Isabel Barnett drug development contract program to develop new treatments for ALS. This program compliments the Department of Defense ALS research program and the translational programs at the National Institutes of Health. The supporting funds help de-risk novel treatment approaches and aim to incentivize industry partners to further pursue novel targets.

    For questions regarding program fit please email Lucie Bruijn, Ph.D. MBA. Chief Scientist, The ALS Association lucie@alsa-national.org

    The Lawrence and Isabel Barnett Drug Development Program:

    This call supports the preclinical assessment of therapeutics for ALS pre-IND with a likely chance of reaching the clinic within 3 years. The proposed studies are expected to be product-driven and focused on therapeutics. It is anticipated that the agents and/or data generated from these awards will lead to the advancement of new therapies for ALS.

    The program is designed to support preclinical testing and development of therapeutics for ALS. Applicants must include preliminary data relevant to the phase(s) of the preclinical development process covered by the proposed research. Applicants must clearly and explicitly articulate what impact the project may have on therapeutic development for ALS. Clinical trials will not be supported with this funding opportunity.

    The contracts are limited to the areas of programmatic interest listed below. Applications must focus on one or more of these areas to be considered for funding. Applications that do not focus on at least one of the following areas will be administratively withdrawn. Preliminary data supporting the choice of target for drug development for ALS must be provided both in the Preliminary Proposal and the Full Application. Priority is given to applications focused on developing compounds directed towards the most attractive targets for ALS with significant data to support the relevance of the chosen target for ALS therapy. Academic- Industry partnered programs will be reviewed more favorably.

    Eligible Requests:

    Testing of compounds of lead molecules in mouse models of ALS. (Note: Design of mouse model study and approaches to measure target engagement will be reviewed and relevant details must be included in the Preliminary Proposal. Additional studies in human tissue and/or IPS lines are encouraged to strengthen mouse data).

    Development of pharmacologic agents through Adsorption, Distribution, Metabolism, Excretion, and Toxicity (ADMET) phase; This can be performed by a contract research organization in collaboration with the PI.

    Design and implementation of full-scale, pilot current Good Manufacturing Practice (cGMP) production of therapeutics and/or delivery systems for use in advanced preclinical and initial clinical trials.

    Industry partners and collaborative teams with appropriate expertise are strongly encouraged.

    The preclinical drug development process will likely require resources beyond those available at a single organization. Therefore, the contracts are open to investigators participating in synergistic collaborations, focused testing and developing lead agents for the treatment of ALS. Collaborations should be dedicated to a single, synergistic preclinical development project or study, rather than an additive set of subprojects (i.e., the combined efforts of the collaboration must provide greater benefit than the sum of individual research initiatives).

    If a collaboration is proposed, letters confirming/supporting the collaboration are required.If the collaboration is multi-organizational, participating organizations will ensure the success of the collaboration by resolving potential intellectual and material property issues and by removing organizational barriers that might interfere with achieving high levels of cooperation.

    Biotechnology or pharmaceutical companies are encouraged to apply. Whether a biotechnology or pharmaceutical company applies for this mechanism as an individual

    applicant or as part of a collaboration, the company is expected to leverage additional resources to complement the funding.

    Review Criteria for Preliminary Proposal:

    1. Scientific merit: Priority of therapeutic target as compared with other proposals. Design of mouse study
    2. Research Strategy and Objectives: How the scientific rationale supports the project objectives and feasibility.
    3. Impact How the project will make an important contribution to ALS therapeutic development and how does the approach fit with the current landscape of ALS treatments in development.
    4. Personnel: How the qualifications of the PI and key personnel are appropriate to perform the proposed research project.
    5. Consultants and Collaborators: How the overall collaboration will be most effective at achieving milestones and progressing the therapeutic towards clinical trials.
    6. Industry partner will be favorably reviewed.
    7. Timeline for development of therapeutic to be ready for phase I clinical trial.


    The maximum period of performance is 2 years.

    The maximum allowable direct and indirect cost (maximum of 10% allowed) for the entire period of performance is $500,000. $250,000 per year.

    Payment structure will be negotiated and based on milestone achievements. Time lines must be clearly described.

    More cost-effective studies that do not request the full available funding amount are encouraged.

    Regardless of the period of performance proposed, the applicant may not exceed the maximum allowable costs.

    Travel costs of up to $1,500 per year to attend scientific/technical meetings are allowed.

    Salary for PI will not be supported.


    For more information, visit The ALS Association website: http://www.alsa.org/research/our-approach/call-for-abstracts/barnett-drug-development-request-for-proposals-061118.html