Pipeline
Two projects,
one breakthrough drug delivery platform
Our Pipeline
Two projects,
one breakthrough drug delivery platform
PK MED is dedicated to developing innovative, patented, safe and effective local therapies.
PK MED has developed a portfolio of early projects in indications with high unmet medical needs, starting with Gout flare (ARTHRELIS), Osteoarthritis (ARTHRELIS) and bone marrow transplantation enhancement (ENGRAFTIS).
The disease
Gout: a severe and debilitating inflammatory disease
More than 10 million patients in the U.S.
Many patients have comorbidities that limit the use of standard therapies
Current treatment options are often slow-acting, insufficiently effective, or contraindicated
A significant unmet medical need remains for difficult-to-treat patients
Our solution
An intra-articular combination
of a fast-acting anesthetic (ropivacaine) and a controlled-release form of a powerful anti-inflammatory agent (colchicine)
A fast-acting, highly effective and safe treatment
Administration
1
Single administration
per flare in all joints
Onset
IMMEDIATE
Minutes,
rapid onset of relief
Duration
7
Pain killer over the whole
flare duration (7 days)
Eligibility
ALL
Suitable for all refractory
gout patients
Derisked development program
Validated mechanism of action in humans
Colchicine: an oral treatment already approved
for gout flares
Ropivacaine: a recognized treatment already
on the market for providing rapid analgesia
High efficiency and transposition
Robust preclinical pharmacokinetic
and efficacy data
Local pathology, local mechanisms:
high probability of translatability to humans
Clinical Program
Clearance from FDA to initiate the clinical
program directly by a Phase 2 study
Support
Strongly supported by key opinion leaders
Key opinion leaders
"This product will fill a much needed gap in gout flare treatments: an effective, safe alternative for all patients but mainly for those who have contraindications to other therapies"
Pr. Angelo Gaffo
Rheumatoglogist
@Birmingham VA Hospital, USA
"An encapsulated colchicine with local release represents a major advance in the management of flares"
Pr. Hang-Korng Ea
Rheumatologist
@Hopital Lariboisière
Paris, France
"This new product is a breakthrough in the treatment of gout flares. The rapid onset should improve patient - physician confidence and adherence to treatments"
Pr. M-C. Boissier
Rheumatologist @AP-HP
Paris, France
"An injectable form of colchicine as a concept is innovative and would represent a very useful
form of therapy for acute gout"
Pr. Geraldine McCarthy
Rheumatologist @University College Dublin, Ireland
A potential extension of ARTHRELIS to osteoarthritis
The disease
A common degenerative disease affecting 600 million people worldwide
Few pharmacological options, with major limitations in efficacy and safety
Our approach
A long-acting intra-articular colchicine derived from ARTHRELIS
ENGRAFTIS
Changing the paradigm of bone marrow transplantation
The disease
Allogenic HSCT (Hematopoietic Stem Cell Transplantation) can cure and/or prolong life in a broad range of hematologic disorders
About 30,000 allogenic HSCT per year (US+EU)
However, many patients are at risks of HSCT complications leading to excess mortality
and/or increase of treatment costs
Our solution
A unique artificial bone marrow niche to enhance HSCT
-
Injectable therapeutic implant
-
Dual mechanism: restoration of signaling pathways and recreation of a favorable bone marrow environment
-
Patent protection through 2046,
ensuring a strong barrier to entry
A pioneer technology
First Preclinical validation completed
Preclinical
proof-of-concept:
improved engraftment
First indication: Poor Graft Function (PGF)
Major Medical need
Significant excess mortality: 1-year survival rate
of 20–50% vs. 90% in cases of successful graft*
Extension
Cell homing platform for other tissue
regeneration applications
* Zhao et al. Biology of Blood and Marrow Transplantation, 2019