Market Overview

Regenacy Pharmaceuticals Announces Collaboration with the Charcot-Marie-Tooth Association (CMTA) to Advance Ricolinostat for the Treatment of Hereditary Neuropathy


-- Collaboration to explore role of HDAC6 in multiple forms of CMT
disease --

-- Company appoints Dr. David Herrmann to
Scientific Advisory Board --

Pharmaceuticals, LLC
, a clinical-stage biopharmaceutical company
developing breakthrough treatments for diabetic and other peripheral
neuropathies, today announced a collaboration with the
Charcot-Marie-Tooth Association (CMTA), a registered non-profit
organization serving the hereditary neuropathy patient community, to
validate the role of HDAC6 in multiple forms of Charcot-Marie-Tooth
(CMT) disease and evaluate the efficacy of ricolinostat, a selective
HDAC6 inhibitor, in animal models. In addition, the company announced
the appointment of David Herrmann, M.B.B.Ch., to its Scientific Advisory
Board. Dr. Herrmann is Chief of the Neuromuscular Division & Director of
the Peripheral Neuropathy Clinics and Cutaneous Innervation Laboratory
in the Department of Neurology at the University of Rochester, and a
member of the STAR (Strategy to Accelerate Research) advisory board for
the CMTA.

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"We are thrilled to have such a substantial collaboration to broaden our
programs for ricolinostat into inherited forms of neuropathy where there
is a tremendous unmet need," said Matt Jarpe, Ph.D., Vice President of
R&D of Regenacy Pharmaceuticals. "This alliance with the
Charcot-Marie-Tooth Association will enable us to expand our
understanding of the role of HDAC6 in neuropathic diseases and lead us
closer to initiating clinical trials in CMT disease."

CMT disease is a progressive and degenerative nerve disease that usually
appears in adolescence or early adulthood. Symptoms can include muscle
weakness, decreased muscle size, foot-drop, foot bone abnormalities,
fatigue, balance problems, neuropathic and/or musculoskeletal pain, loss
of feeling in the hands and feet and loss of coordination in the limbs.
There are no FDA approved treatments to stop or reverse the loss of
nerve function in CMT. Based on the terms of the collaboration, Regenacy
has an opportunity to expand on the groundbreaking work
of Dr. Ludo van den Bosch at University of Leuven and Dr. Andrew
Grierson at University of Sheffield to show the role of HDAC6 in several
forms of CMT Type 2. The collaboration will focus on evaluating the
efficacy of ricolinostat in animal models of CMT to support initiation
of clinical trials. This relationship is taking advantage of the
extensive suite of expert preclinical testing capabilities the CMTA has
assembled and makes available to groups such as Regenacy that want to
evaluate therapeutic potential in a CMT disorder.

"In parallel to announcing our collaboration with CMTA, we are pleased
to welcome Dr. David Herrmann to our Scientific Advisory Board," added
Dr. Jarpe. "Dr. Herrmann brings to Regenacy relevant and valuable
experience in clinical outcome measures in peripheral neuropathies,
particularly CMT disease. We are looking forward to his contributions
and proven strategic expertise as we continue to develop ricolinostat
for the treatment of CMT."

Dr. Herrmann is currently Professor of Neurology, Pathology and
Laboratory Medicine at the University of Rochester. He established one
of the first cutaneous innervation laboratories in the United States at
University of Rochester for diagnosis of small fiber neuropathy. In
addition to his role on the STAR advisory board for the CMTA, Dr.
Herrmann is a member of the Inherited Neuropathies Consortium. He is
currently Principal Investigator in Rochester for the NIH sponsored
Inherited Neuropathy Consortium Rare Disease Clinical Research Center.
Dr. Herrmann received his medical degree from The University of
Witwatersrand Medical School.

"I am personally passionate about inherited neuropathies for which there
is an urgency to develop treatments," said Dr. Herrmann. "Current
treatment options for Charcot-Marie-Tooth disease, and related
disorders, are very limited so each potential new therapy can make a
critical difference for patients in need. I am excited to collaborate
with the highly regarded Regenacy team, who are leaders in the field and
are committed to bringing forward new treatment options in various types
of peripheral neuropathy."

About Charcot-Marie-Tooth Association (CMTA)
Charcot-Marie-Tooth Association is a leading patient led nonprofit
organization dedicated to finding a cure for CMT. The CMTA's Strategy to
Accelerate Research (STAR) program brings top researchers together with
pharmaceutical and biotechnology partners to accelerate scientific
breakthroughs. The CMTA also offers community services including 70
local branches, Camp Footprint exclusively for teens with CMT, and
patient conferences. To learn more about the CMTA, please visit

About HDAC6 Inhibition
HDAC6 is an enzyme that regulates
mitochondrial function and intracellular transport along microtubules.
Peripheral nerves, due to their length, are uniquely sensitive to
metabolic and chemical influences that disrupt intracellular transport,
leading to debilitating pain, numbness and loss of muscle control,
partially caused by mitochondrial dysfunction. Recent studies in animal
models of diabetic, chemotherapy induced and inherited peripheral
neuropathy, have implicated HDAC6 as a promising therapeutic target to
restore nerve function in the periphery and significantly alleviate the
symptoms of pain, motor function loss and numbness.

About Regenacy
Regenacy Pharmaceuticals, LLC is a
clinical-stage biopharmaceutical company regenerating biological
function by protein acetylation for the treatment of diabetic and other
peripheral neuropathies and other chronic conditions. The company's
selective inhibition technology provides superior safety profiles and
potential enhanced efficacy compared to non-selective HDAC inhibitors.
Regenacy's programs selectively inhibit histone deacetylase 6 (HDAC6) to
restore normal intracellular protein and organelle transport in
peripheral neuropathies, and a platform of selective HDACs 1 and 2
inhibitors that have potential to treat major blood diseases such as
sickle cell disease, β-thalassemia, leukemia, and cognitive dysfunction
in neurological disorders.

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