Rare Daily Staff

The U.S. Food and Drug Administration said it awarded 15 new clinical trial research grants totaling more than $22 million over the next four years to boost the development of products for patients with rare diseases.

These new grants were awarded to principal investigators from academia and industry across the country.

“Given the often small number of patients facing certain rare diseases, there can be limited resources devoted to researching new drugs and unique challenges with recruiting and conducting the clinical trials needed to develop medicines targeted to rare conditions,” said FDA Commissioner Scott Gottlieb. “By helping to support the cost of development of these potential new drugs, and reduce some of the financial risk, we also hope that these grants will lower the cost of the capital needed to develop these products, boost competition, and translate into lower prices for successful medicines.”

The FDA awarded the grants through the Orphan Products Clinical Trials Grants Program. The grants are intended for clinical studies evaluating the safety and effectiveness of products that could either result in, or substantially contribute to, the FDA approval of products targeted to rare diseases.

The grants awarded this year will support needed research in a range of rare diseases that have little, or no, treatment options for patients.

A total of 76 grant applications were received for this fiscal year, with a funding rate of 20 percent. The grant recipients for fiscal year 2017 are the following:

AADi, LLC (Pacific Palisades, California), Neil Desai, Phase 2 Study of ABI-009 for the Treatment of Advanced Perivascular Epithelioid Cell Tumors – about $2 million over four years

Albert Einstein College of Medicine (Bronx, New York), Caterina Minniti, Phase 2 Study of Topical Sodium Nitrite for the Treatment of patients with Sickle Cell Disease & Leg Ulcers – about $2 million over four years

Albert Einstein College of Medicine (Bronx, New York), Eric Hollander, Phase 2 Study of Oxytocin for the Treatment of Hyperphagia in Prader-Willi Syndrome – about $1.5 million over three years

Alkeus Pharmaceuticals, Inc. (Cambridge, Massachusetts), Leonide Saad, Phase 2 Study of ALK-001 for the Treatment of Stargardt Disease – about $250,000 over one year

CereNova, LLC (Durham, North Carolina), Daniel Laskowitz, Phase 2A Study of CN-105 for the Treatment of Intracerebral Hemorrhage – about $1 million over two years

Columbia University Medical Center (New York), Elizabeth Shane, Phase 2 Study of Teriparatide for the Treatment of Idiopathic Osteoporosis in Premenopausal Women – about $1.9 million over four years

Columbia University Medical Center (New York), Gulam Manji, Phase 2 Study of PLX3397 + Sirolimus for the Treatment of Malignant Peripheral Nerve Sheath Tumors – $2 million over four years

Dana-Farber Cancer Institute (Boston), Steven Dubois, Phase 1 Study of dual PI3K/BRD4 Inhibitor SF1126 for the Treatment of Neuroblastoma – $750,000 over three years

Duke University (Durham, North Carolina), Allan Kirk, Phase 2 Study of Belatacept, Alemtuzumab, and Sirolimus in Renal Transplantation – about $1 million over three years

Johns Hopkins University (Baltimore), Susan Dorman, Phase 2a Study of Rifampin, Merrem and Augmentin for the Treatment of Pulmonary Tuberculosis – about $2 million over four years

New York Medical College (Valhalla, New York), Mitchell Cairo, Phase 2 Defibrotide for the Prevention of Complications in High-Risk Sickle Cell Disease Patients Following Allogeneic Stem Cell Transplantation – about $1.75 million over four years

Protalex, Inc (Florham Park, New Jersey), Richard Francovitch, Phase 1/2 Study of PRTX-100 for the Treatment of Immune Thrombocytopenia – about $500,000 over two years

Sloan-Kettering Institute for Cancer Research (New York), Ping Chi, Phase 2 Study of MEK162 & Imatinib for the Treatment of Gastrointestinal Stromal Tumors – $2 million over four years

Tocagen Inc. (San Diego), Asha Das, Phase 2/3 Study of Toca 511 +Toca FC versus SOC in Recurrent Glioblastoma and Anaplastic Astrocytoma – $2 million over four years

University of California, San Francisco (San Francisco), Marshall Stoller, Phase 2 Study of Lipoic Acid for the Treatment of Cystine Nephrolithiasis – about $2 million over four years.

October 6, 2017

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