evidence for additional clinical development of this
We look forward to advancing the clinical
development of PXT864 as we strive to bring an
efficacious treatment to people suffering from
Alzheimer’s disease. PXT864 has also high potential
in other diseases such as Parkinson’s disease, ALS,
and other rare indications.
Q: What steps are taken to identify which
drugs in combination produce better results
than existing therapies?
Haij: Once we have identified and selected a
synergistic combination (PLEODRUG), the
comparison to existing therapies can be performed
like for any other new chemical entity. When
applicable, we study the effect of our PLEODRUG
combined with existing therapies/standard of care
(SOC) to seek an efficacy improvement versus
Q: Recent literature on PLEOTHERAPY states:
“two drugs can be better than one.” Are
combinations limited to two drugs, or is possible
to include three or more in combination?
Haij: Yes, for example, our lead PLEODRUG
PXT3003 is the combination of three known
molecules: baclofen, naltrexone, and sorbitol. Most
diseases are multifactorial (even when monogenic),
therefore monotherapy is reaching its limits as can
be witnessed by the very high number of failures in
late phase development.
Increasing the number of drugs in a combination
could overcome this multifactorial issue if they
target simultaneously several disease pathways.
Our PLEOTHERAPY approach is based on the
multifactorial nature of diseases and enables the
discovery and development of complex synergistic
combinations of drugs (PLEODRUG) targeting
simultaneously several disease pathways.
Q: How does the approval process for
combination drugs differ from a single drug?
What are some of the distinct challenges?
Haij: The process is similar in that all studies
must demonstrate safety and a positive intended
impact. The body of evidence required for potential
market approval is the same as for a single drug
development. However, additional data are also
required by regulatory agencies: for example, in
the case of a two-drug combination the efficacy of
the duo versus their respective single components is
Q: Have the FDA and European Medicines
Agency been supportive of the overall
concept, or are they viewing results on a case-by-case basis?
Haij: Regulatory agencies have watched the process
with great interest and with an appropriate amount
of scrutiny. In 2014, following positive Phase 2
results they granted the orphan drug designation for
PXT3003 in the treatment of CMT1A in adults. We
believe that the PLEOTHERAPY approach can lead
to better, more targeted treatments for a wide range
Q: The lower doses of already established
drugs are said to lower costs. How is that
possible? Don’t pharmaceutical companies
require royalty payments, including but not
limited to the samples needed for research?
Haij: With our PLEOTHERAPY approach we use
already known drugs. If such medications are off
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