Translational
Medicine
Translational medicine is where RxMD thrives. We thrive in unraveling the complexity of drug development – be it the understanding of enzyme substrate reactions, ligand receptor kinetics, pharmacokinetics, calculus, quantitative systems pharmacology, modelling and simulation, exposure response analysis, medicine or statistics.
We strongly believe that the earlier we are incorporated into a development team, the greater the contribution we can make to the development program.
Over the years, we have provided both strategic consulting and execution services to our partners.
Gene Therapy –
Development Strategy
through Asset Prioritization
RxMD called upon for assistance in the area of gene therapy (CAR-T therapy) for solid tumors. Sponsor working on developing next-generation T cell immunotherapy. RxMD provides critical inputs, helping client prioritize assets for oncology and immunology indications based on varied parameters viz. probability of efficacy, competitive landscape, path of regulatory approval, etc. RxMD also assists client in IND filing activities for lead assets (identified through prioritization) and formulating responses to regulatory questions. RxMD subsequently provides guidance in Phase 1 study start-up activities for lead assets.
RxMD approached for a due diligence of a new molecular entity for gastrointestinal (GI) motility disorders. RxMD reviews complete clinical and non-clinical package and flags hitherto unidentified adverse events pertaining to elevated liver enzymes. Partner reconsiders bidding for asset in the light of RxMD’s findings. Impressed with RxMD’s diligence capabilities, partner signs master services agreement for employing RxMD as preferred vendor for due diligence activities. RxMD later conducts diligence for partner across a range of drug-class indication combinations: antibacterial for community acquired bacterial pneumonia and skin/soft tissue infections; tyrosine kinase inhibitor for oncology indication; growth factor mimetic for renal injury; and antibacterial with gram negative activity. For all potential in-licensing assets, RxMD evaluates complete clinical trial and safety data to come up with recommendations on favorable and unfavorable aspects, which partner factors while bidding for the assets.
Due Diligence
Filing of INDs
A novel technology to expand ex-vivo umbilical cord blood hematopoietic stem cells developed by Sponsor. RxMD uses its pediatric and cell therapy expertise to author clinical sections of IND for the use of the novel technology in inborn errors of metabolism.
Sponsor develops a metal protein attenuating compound for a rare neurodegenerative condition. RxMD provides strategic medical, scientific input into the IND and the phase 1 program.
In parallel with the filing of an IND by sponsors, RxMD (in partnership with clinical pharmacologists) develops several novel phase 1 study designs for small molecules and biologics that incorporate both sequential dose escalation and a parallel study design with a lean minimalistic approach.
First in Human Studies
Innovative Study Design
Pharma company struggling to develop appropriate Proof-of-Concept study design for immunomodulator asset in chronic respiratory indication. RxMD employs innovative add-on design, using SoC drug concentration area under the curve (AUC) for an exposure-response analysis to probe a Proof of Concept
Client Speak
Usman "Oz" Azam, MD
In the cell and gene therapies world, RxMD have developed a very credible scientific, translational and development understanding. At Novartis during 2013-2016, my teams
Rajesh Krishna, PhD
This project could not have been done without your expert knowledge of pediatrics and, more importantly, PK/PD. I want to stress this aspect since over the course
Our Clientele
We have worked for U.S. and EU-based big pharma & biotech, for small molecules, biologics & cell therapy, across indications.
