`Oct, 2003
`
`Results in Year 1
`
`1. Determine the safety and efficacy of pharmacologic inhibiton of the microsomal
`transfer protein (MTP) in patients with homozygous FH, including the effects on in
`vivo lipoprotein metabolism and on atherosclerosis.
`
`We have initiated a phase I/ll clinical trial of an MTP inhibitor in patients with
`homozygous FH. Six patients have been enrolled and two have completed the dose
`escalation treatment phase of the protocol. Tolerability has been surprisingly good, and
`there have been no major safety issues; a few patients have had increased liver function
`tests that were dealt with by reduction in dose as per protocol. Excitingly, we have seen
`major reductions in plasma cholesterol levels, though we await more data before
`reporting on the efficacy results.
`In any case, we have already answered our major
`question with this phase I/II triaI—whether we can identify a dose of the MTP inhibitor in
`these patients that will be acceptably tolerated and result in substantial reduction in
`cholesterol levels. As a result, we have already begun planning for a larger and longer
`phase III trial. The current study could not have occurred without the support of the
`DDCF.
`I co-authored a review in the Journal of Clinical Investigation about genetic
`hypercholesterolemia that mentioned the use of MTP inhibition as a potential therapeutic
`strategy for homozygous FH.
`
`1 of 2
`
`PENN EX. 2081
`CFAD V. UPENN
`
`IPR2015-01835
`
`
`
`
`
`2 of 22 of 2
`
`
`PENN EX. 2081PENN EX. 2081
`
`CFAD V. UPENNCFAD V. UPENN
`
`
`
`IPR2015-01835IPR2015-01835
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