Treatment of fibrodysplasia ossificans progressiva by administration of an anti-activin a antibody
Inventors
Economides, Aristides • Neto, Eduardo Forleo • Trotter, Dinko Gonzalez • Herman, Gary • Rankin, Andrew • Mellis, Scott
Assignees
Interested in licensing this patent?
MTEC can help explore whether this patent might be available for licensing for your application.
Publication Number
US-12344665-B2
Publication Date
2025-07-01
Expiration Date
Abstract
Methods for treating fibrodysplasia ossificans progressiva (FOP) in human subjects are provided. Such methods involve administering to a subject having FOP a therapeutically effective amount of an activin A antagonist, such as an antibody against activin A.
Core Innovation
The invention describes treating fibrodysplasia ossificans progressiva (FOP) in a human subject by administering a therapeutically effective amount of an activin A antagonist to decrease the formation of new heterotopic ossification lesions. In particular, the activin A antagonist is an anti-activin A antibody, or an antigen-binding fragment thereof, and the approach is directed to reducing the incidence and/or formation of new heterotopic ossification lesion development in the human subject.
The invention further addresses decreasing lesion growth and mineralization rates associated with new heterotopic ossification lesions. The described findings include large reductions in new lesion incidence/volume and flare-ups, while not affecting pre-existing lesions.
The activin A antagonist is defined by specific complementarity determining region (CDR) sequences, including HCDR1, HCDR2, HCDR3, LCDR1, LCDR2, and LCDR3. The document also describes activin A signaling and biologic rationale in relation to FOP and includes a clinical-trial context for garetosmab.
Claims Coverage
The provided content includes one independent claim. The claim covers administering a therapeutically effective amount of an activin A antagonist that is an anti-activin A antibody, or antigen-binding fragment thereof, defined by six specific CDR sequences, to decrease the formation of new heterotopic ossification lesions in a human subject with fibrodysplasia ossificans progressiva (FOP).
Decreasing formation of new heterotopic ossification lesions in FOP via an anti-activin A antibody with specified six CDR sequences
A method of decreasing the formation of new heterotopic ossification lesions in a human subject with fibrodysplasia ossificans progressiva (FOP) by administering to the human subject a therapeutically effective amount of an activin A antagonist, thereby decreasing the formation of new heterotopic ossification lesions, wherein the activin A antagonist is an anti-activin A antibody, or antigen-binding fragment thereof, comprising six CDR sequences including HCDR1 GGSFSSHF (SEQ ID NO: 2), HCDR2 ILYTGGT (SEQ ID NO: 3), HCDR3 ARARSGITFTGIIVPGSFDI (SEQ ID NO: 4), LCDR1 QSVSSSY (SEQ ID NO: 6), LCDR2 GAS (SEQ ID NO: 7), and LCDR3 QQYGSSPWT (SEQ ID NO: 8).
The coverage is centered on decreasing new heterotopic ossification lesion formation in FOP by administering an activin A antagonist that is specifically defined as an anti-activin A antibody, or antigen-binding fragment, with six defined CDR sequences.
Stated Advantages
Decreases the formation of new heterotopic ossification lesions in a human subject with fibrodysplasia ossificans progressiva (FOP).
Decreases lesion growth and mineralization rates.
Produces large reductions in new lesion incidence/volume and flare-ups.
Does not affect pre-existing lesions.
Documented Applications
Treating fibrodysplasia ossificans progressiva (FOP) by administering an activin A antagonist to reduce and/or prevent new heterotopic ossification lesion formation and to decrease lesion growth/mineralization rates.
Clinical evaluation of treatment effects using 18F-NaF PET/CT, CT lesion volume, and pain assessed by daily average pain NRS, including phase II clinical trial results for garetosmab.
Interested in licensing this patent?