Cavion Announces Promising Phase 2 Data for Essential Tremor in T-CALM Proof-of-Concept Study

~Totality of Data Demonstrates CX-8998 is Well Tolerated with
Compelling Evidence of Efficacy Despite Missing Primary Endpoint
Measured Using Central Video Rating~

~Multiple Secondary and Exploratory Endpoints Showed Significant
Benefits in Functional, Patient-Reported and Clinical Outcome Measures~

HONG KONG–(BUSINESS WIRE)–#MDSCongress–Cavion, Inc., a leading clinical stage biotechnology company committed
to developing novel therapeutics for people with neurological diseases,
today announced promising results of the T-CALM Phase 2 clinical trial
of its first-in-class T-type calcium channel modulator CX-8998 in
essential tremor. The company plans to present results from this
proof-of-concept study as a late-breaking poster on Monday, October 8 at
the International Congress of Parkinson’s Disease and Movement Disorders®
in Hong Kong.


Multiple secondary and exploratory endpoints collectively showed that
treatment with CX-8998 10mg twice daily resulted in statistically
significant improvement versus placebo in activities of daily living and
patient-reported and clinical outcome measures, despite the study’s
primary endpoint being missed. In the study, CX-8998 demonstrated a
favorable safety and tolerability profile, with patients in the drug
treatment arm reporting higher levels of satisfaction with tremor
control medication compared to patients on placebo.

“After decades without new effective drug therapy options, we believe
that the neurostabilizing effects of CX-8998 provide hope for essential
tremor patients,” said Spyros Papapetropoulos, MD, PhD, Cavion’s
Executive Vice President of Research & Development and Chief Medical
Officer. “The data are particularly compelling given that about
two-thirds of patients in the study were on standard of care treatment
and significant improvement was achieved with CX-8998 on several
measures in a study lasting just one month.”

“Essential tremor is a challenge to treat, and patients often do not
respond favorably to the existing medications, many of which have been
available for decades,” said Karl Kieburtz, MD, MPH, Professor of
Neurology at the University of Rochester Medical Center. “Cavion’s
CX-8998 has a novel mechanism of action that may provide substantive
improvement, and it could be an important therapeutic contribution above
and beyond what we currently have for essential tremor. I look forward
to its clinical progress and further investigation. Better medications
are needed to treat this common and disabling movement disorder.”

T-CALM was a 28-day, double-blind, placebo-controlled Phase
2 proof-of-concept clinical trial in 95 patients with moderate to severe
essential tremor, conducted at 25 sites across the United States.
Patients were randomized to receive either placebo or CX-8998 titrated
up to 10mg dosed twice daily (BID). A majority of subjects (64 percent)
added study drug to their existing standard of care anti-tremor
medication.

The primary study endpoint using remote video rating of the Tremor
Research Group Essential Tremor Rating Assessment Scale – Performance
Subscale (TETRAS-PS), a measure of tremor severity, did not meet
significance at Day 28 (p=0.696). In contrast, the same TETRAS-PS scale
rated by the study investigators who observed the patients in person
resulted in a significant improvement in tremor severity at Day 28 for
the CX-8998 treated group compared to placebo (p=0.027). Independent
analysis of the video rating process uncovered unexpected complications
in implementation. Cavion is working closely with the scientific
community to enhance central video rating for use in interventional
studies.

Multiple secondary and exploratory endpoints collectively showed
statistical significance, with benefits after 28 days of treatment in
patients receiving CX-8998 10mg twice daily (BID) compared to placebo in
the following scales:

  • The TETRAS Activities of Daily Living (TETRAS-ADL) scale, a measure of
    tremor impact on daily activities (p=0.049)
  • The Clinician Global Impression of Improvement (CGI-I), a measurement
    of physician-reported global improvement (p=0.001)
  • The TETRAS Total Score (TETRAS ADL + TETRAS-PS rated by the study
    Investigators, p=0.029)
  • The Goal Attainment Scale (GAS), a measure of progress toward
    patient-selected goals (such as dressing oneself, writing a sentence,
    or drinking out of a cup) (p=0.034)

In addition, the Patient Global Impression of Change (PGI-C), a measure
of patient-reported improvement, trended towards significance favoring
CX-8998 (p=0.089). Kinesia One, an algorithm-based digital measure
developed for Parkinson’s disease, failed to detect a treatment
difference in Essential Tremor patients. Additional analyses, including
patient satisfaction with control by anti-tremor medication and
traditional and digital spirography, support a treatment benefit with
CX-8998. The drug showed a favorable safety and tolerability profile,
with the majority of adverse events mild to moderate, transient and
non-recurring. No major differences between CX-8998 and placebo were
observed in laboratory values, vital signs or electrocardiography.

In addition to the T-CALM study for essential tremor, CX-8998 is
currently being studied in T-WAVE, a multi-center Phase 2 clinical trial
for generalized epilepsy with absence seizures. “The body of evidence
supporting our ground-breaking approach is rapidly expanding,” said
Andrew Krouse, President and CEO of Cavion. “Our pipeline of T-type
calcium channel therapies extends beyond tremor to other neurological
diseases that share a common pathophysiology. We look forward to
initiating a Phase 3 study in tremor soon.”

About T-CALM

The T-CALM study is one of the largest Phase 2 clinical trials ever
conducted in essential tremor. This 28-day, double-blind,
placebo-controlled proof of concept study recruited 95
patients with moderate to severe essential tremor at 25 sites across the
United States. Patients were randomized to one of two treatment arms,
receiving either placebo or CX-8998 on top of certain standard of care
medications. The average age of patients in the study was 63, with an
average of 23 years since tremor onset. About two-thirds (64 percent) of
participants were on background standard of care anti-tremor medications
during the trial. The study design called for dosing of CX-8998 starting
at 4 mg BID (one week), followed by 8 mg BID (one week) and 10 mg BID
for the last 2 weeks, ending at day 28 of the study.

About Essential Tremor

About 10 million Americans and 42 million people worldwide are affected
by essential tremor (ET), the most common movement disorder. ET is a
progressive neurodegenerative disease that ranges from mildly to fully
debilitating, with significant effects on peoples’ quality of life and
activities of daily living, such as eating, drinking, dressing, writing
and typing. The condition can lead to social embarrassment, phobia,
anxiety and depression. With no new drugs approved for more than three
decades, there is an incredible need for new and effective options for
patients with ET (Haubenberger and Hallett. 2018. Essential Tremor. New
England Journal of Medicine.
378:19)

About Cavion, Inc.

Cavion,
Inc
. is a privately held clinical stage biotechnology company
creating therapies aimed at modulating the T-type calcium channel for
the treatment of chronic and rare neurological diseases. Cavion’s
portfolio of T-type calcium channel modulators is being developed to
restore the brain’s natural rhythms in a variety of neurological
diseases such as essential tremor, Parkinson’s disease tremor,
neuropathic pain, and epilepsy with absence seizures.

Forward-Looking Statement

This press release contains forward-looking statements that are subject
to risks and uncertainties and includes statements that are not
historical facts. Actual results could differ significantly from results
discussed. All p values in this release, other than the primary
endpoint, are considered nominal. Cavion, Inc. disclaims any intent or
obligation to update forward-looking statements, except as required by
law.

Contacts

Lynn Blenkhorn
Lynn.blenkhorn@ogilvy.com
508-851-0930
or
Susan
Klees
susanofcville@gmail.com
(434)
466-8930

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