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Phase 2 Study of EPN-701 for Treatment of Calciphylaxis (IND: 129344; September 14, 2022)

Award Number: R01FD008525
ORGANIZATION: FOOD AND DRUG ADMINISTRATION
OPDIV: FDA
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 09/01/2026
PERIOD OF PERFORMANCE END DATE: 08/31/2030

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Phase 2 Study of EPN-701 for Treatment of Calciphylaxis (IND: 129344; September 14, 2022) - Abstract Calciphylaxis is a rare but serious disorder of vascular calcification, characterized by occlusion of microvessels in the subcutaneous adipose tissue and dermis, leading to painful ischemic skin lesions. Calciphylaxis predominantly affects dialysis-dependent patients and is associated with high morbidity and mortality. Currently, there is no approved therapy for calciphylaxis. In this application, we propose to examine the hypothesis that EPN-701 (IND# 129344)—a highly purified form of menaquinone-7 (MK-7), which belongs to the vitamin K2 family—will mitigate the progression of cutaneous calcification and promote clinical improvement in patients with calciphylaxis. This effect is hypothesized to occur via enhanced carboxylation and activation of matrix Gla protein (MGP), a potent inhibitor of vascular calcification. We will conduct a double-blind, placebo-controlled trial in dialysis-dependent patients with calciphylaxis (n = 60; 1:1 EPN-701 to placebo assignment) to evaluate the clinical efficacy, safety, pharmacodynamics, and pharmacokinetics of EPN-701 (10 mg/day administered orally for 24 weeks). Clinical efficacy will be assessed by pre-post (Baseline to Week 24) changes in pain intensity and the total surface area of calciphylaxis skin lesion(s) between treatment arms (alternate primary efficacy endpoints). We will also explore changes in pain interference, quality of life, and rates of lesion healing, hospitalization, infection, and mortality. Additionally, we will assess whether ongoing cutaneous calcification can be ameliorated and whether novel proteomic biomarkers of calciphylaxis improve with EPN-701 treatment. Safety will be evaluated through active monitoring of adverse events throughout the trial period. Pharmacodynamic effects will be assessed via changes in plasma levels of uncarboxylated MGP. EPN-701 plasma concentration-time profiles and other pharmacokinetic parameters will also be analyzed. This resubmission (RFA-FD-25-020) aligns with the FDA’s mission to advance treatments for rare diseases with substantial unmet need. Our team, composed of experts from academia and industry, brings extensive clinical and translational research experience and access to exceptional shared resources. The proposal is further strengthened by input from patient advisors. Findings from the proposed trial will inform endpoint selection and the design of a future phase 3 trial aimed at establishing a targeted, novel therapy for calciphylaxis.


Issue Date FY	Funding FY	Legal Entity Name	Legal Entity Address	Legal Entity City	Legal Entity State	Legal Entity Zip Code	Legal Entity COUNTY	Legal Entity COUNTRY	Assistance Listing	Award Code	Budget Year	Action Date	Action Type	Action Amount

Issue Date FY: 2026 ( Subtotal = $2,462,415 )
2026	2026	THE GENERAL HOSPITAL CORPORATION	55 FRUIT ST	BOSTON	MA	02114	SUFFOLK	USA	Food and Drug Administration Research	000	1	9/29/2026	NEW	$2,462,415
														Subtotal = $2,462,415

Grand Total All Awards = $2,462,415

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Phase 2 Study of EPN-701 for Treatment of Calciphylaxis (IND: 129344; September 14, 2022)

Award Number: R01FD008525

ORGANIZATION: FOOD AND DRUG ADMINISTRATION

OPDIV: FDA

AWARD CLASS: DISCRETIONARY

AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)

PERIOD OF PERFORMANCE START DATE: 09/01/2026

PERIOD OF PERFORMANCE END DATE: 08/31/2030

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