Viridian Therapeutics, Inc. (NASDAQ: VRDN), a biotechnology company
advancing new treatments for patients suffering from serious
diseases underserved by current therapies, today announced positive
topline clinical data from the first two cohorts in its ongoing
Phase 1/2 clinical trial of VRDN-001, an anti-IGF-1R antibody, in
patients with active thyroid eye disease (TED). TED is a rare
autoimmune disease in which the body’s own immune system attacks
the tissues around and behind the eyes causing inflammation,
swelling, and damage that develops into debilitating signs and
symptoms including double vision, bulging eyes, and ocular pain.
“These additional Phase 1/2 clinical data continue to support
the potential for VRDN-001 to be a new treatment option for
patients suffering from TED,” said Raymond Douglas, M.D., Ph.D.,
director of the Orbital and Thyroid Eye Disease Program,
Cedars-Sinai Medical Center and an investigator on the VRDN-001
trial. “The data show that a majority of patients experienced
meaningful improvements in proptosis and clinical activity score,
and complete resolution of diplopia after only two infusions of
VRDN-001, with initial data suggesting a prolonged duration of
benefit. This profile could offer substantial benefits to
patients.”
VRDN-001 – Phase 1/2 proof-of-concept trialThe
double-blind, placebo-controlled Phase 1/2 trial is evaluating two
infusions of VRDN-001 administered intravenously, three weeks
apart, with efficacy measured six weeks after the first dose. Each
dose is evaluated in a cohort of eight patients, with six patients
randomized to receive VRDN-001 and two patients randomized to
receive placebo. The inclusion and exclusion criteria and the
baseline patient characteristics for this trial are in line with
prior TED clinical trials. Efficacy measurements include proptosis
(bulging eyes), Clinical Activity Score (CAS), and diplopia (double
vision), which are the same endpoints as measured in the clinical
development of Tepezza®, the only approved therapy targeting IGF-1R
in patients with TED. The first cohort of the Phase 1/2 study
evaluated a dose of 10 mg/kg, with initial positive clinical data
reported on August 15, 2022, and with additional 12-week data
announced today. The second cohort evaluated a dose of 20 mg/kg
with 6-week data announced today. In addition, the Company plans to
report results from the third cohort evaluating a dose of 3 mg/kg
in early January 2023.
VRDN-001 – Safety data VRDN-001 was
well-tolerated by all patients treated at the 20 mg/kg dose, with
safety data consistent with the 10 mg/kg dose. There were no
reported serious adverse events (SAEs), no patient
discontinuations, no hearing impairment, no drug-related
hyperglycemia, and no infusion reactions as of November 8, 2022,
the most recent cut-off date for follow-up observation.
VRDN-001 – Clinical activity dataAll 12
VRDN-001 treated patients in the 10 mg/kg (n=6) and 20 mg/kg (n=6)
cohorts were treated for two full cycles and were evaluated for
proptosis, clinical activity score (CAS), and diplopia. Improvement
in proptosis, CAS, and diplopia was generally consistent across the
two cohorts. A similar IGF-1 response, a biomarker for target
engagement, was also observed across the two cohorts. The following
activity was observed across all patients at week 6:
Proptosis
- 75% proptosis responder rate (83% at 10 mg/kg and 67% at 20
mg/kg), defined as a ≥2mm reduction in proptosis from baseline as
measured by exophthalmometry
- 2.04mm mean reduction in proptosis from baseline (-2.4mm at 10
mg/kg and -1.7mm at 20 mg/kg) as measured by exophthalmometry
- 2.75mm mean reduction in proptosis from baseline when measured
by blinded, centrally reviewed MRI
Clinical Activity Score (CAS)
- 4.0 point mean reduction in CAS from baseline on a 7-point
measure of signs and symptoms of TED (4.3 points at 10 mg/kg and
3.7 points at 20 mg/kg reduction)
- 58% of patients achieved maximal or near-maximal therapeutic
effect on CAS (83% at 10 mg/kg and 33% at 20 mg/kg), defined as
reaching a CAS of 0 or 1 on the 7-point composite measure of signs
and symptoms of TED
Overall response
- 75% overall responder rate (83% at 10 mg/kg and 67% at 20
mg/kg), defined as a ≥2mm reduction in proptosis and a ≥2 point
reduction in CAS
Diplopia
- 75% complete resolution of diplopia, defined as patients with
baseline diplopia who achieved a score of 0 on the Gorman
subjective diplopia scale (75% at 10 mg/kg and 75% at 20
mg/kg)
The Company also reported 12-week data from the 10 mg/kg cohort,
showing that after two infusions, VRDN-001 maintained efficacy for
six additional weeks through week 12. This data further supports
the potential for a shorter course of treatment via a 5-infusion
schedule being studied alongside the standard 8 infusion arm in the
Company’s Phase 3 THRIVE program.
“We are delighted to deliver robust data from a second cohort of
TED patients, showing rapid, clinically meaningful improvements in
every measure of the signs and symptoms of TED. We were also
pleased with our recent Type C meeting with the U.S. Food and Drug
Administration (FDA) and two European Union (EU) scientific advice
meetings and now have initiated our global Phase 3 THRIVE clinical
trial in active TED patients,” said Barrett Katz, MD, MBA, Chief
Medical Officer at Viridian.
VRDN-001 – Upcoming milestones
- Topline data from 3 mg/kg cohort of active TED patients
expected in early January 2023
- First patient expected to be enrolled in chronic TED
proof-of-concept study in December 2022 with preliminary data
expected in the first half of 2023; patient screening underway in
November 2022
- First patient expected to be enrolled in Phase 3 THRIVE study
in active TED in December, with results expected in mid-2024;
patient screening underway in November 2022
SC Programs: VRDN-002 and VRDN-003Earlier this
year, Viridian initiated a first-in-human Phase 1 clinical trial of
VRDN-002, a novel monoclonal antibody that incorporates half-life
extension technology and is designed to support administration as a
convenient, low-volume, subcutaneous (SC) injection for the
treatment of TED patients. This single ascending dose trial
explored safety, tolerability, pharmacokinetics (PK) and
pharmacodynamics (PD) of intravenously administered VRDN-002 at
doses of 3, 10, and 20mg/kg in 12 healthy volunteers. In August
2022, interim VRDN-002 half-life data in healthy volunteers was
reported suggesting a potential half-life of 30-40 days, which
would support Q2W or Q4W dosing. Today, the Company is reporting
updated PK/PD data from the complete dataset in healthy
volunteers.
The following VRDN-002 healthy volunteers results were
observed:
- The data confirmed an extended half-life up to 43 days,
approximately four times the half-life of VRDN-001 and
teprotumumab, and an improvement over the interim results announced
in August 2022
- After a single IV dose of VRDN-002, plasma IGF-1 levels
increased approximately 2.5-fold and were sustained throughout the
measurement period of 84 days
- VRDN-002 was well tolerated with no reported serious adverse
events, hearing impairment, hyperglycemia, muscle spasms, or
infusion reactions reported
The Company is on track to deliver topline SC proof of concept
data for VRDN-002 in TED patients in the second half of 2023; this
trial will evaluate subcutaneous injections of 2mL 300mg dosed Q2W
or Q4W.
VRDN-003, which is VRDN-001 enhanced with the same half-life
extension as VRDN-002, is on track for an IND filing in the second
quarter of 2023, with non-human primate PK indicating a half-life
at least as long as VRDN-002. The Company continues to expect to
deliver topline PK/PD data from a healthy volunteer study in the
fourth quarter of 2023, and to select either VRDN-002 or VRDN-003
to advance to a pivotal Phase 3 trial in early 2024.
“The TED market, projected to exceed $4 billion globally, offers
a rare opportunity for a differentiated entrant to quickly capture
market share. Thus far, our VRDN-001 clinical data is remarkable
and bolsters our confidence in our TED programs. We are developing
a complete global portfolio, including best-in-class IV and SC
IGF-1R therapies for TED, designed to maximize the benefits we can
deliver for patients,” said Jonathan Violin, Ph.D., President and
CEO of Viridian Therapeutics.
Conference call and webcastThe Company will
host a conference call today at 8:00 a.m. ET to discuss the topline
data for VRDN-001 and VRDN-002. The dial-in number for the
conference call is 1-877-407-0789 for domestic participants and
1-201-689-8562 for international participants. The conference ID is
13732927. A live webcast of the conference call can be accessed
through the “Events” page in the Investors section of the Viridian
Therapeutics website. Following the live webcast, an archived
version of the call will also be available on the website.
About Viridian TherapeuticsViridian
Therapeutics is a biotechnology company advancing new
treatments for patients suffering from serious diseases underserved
by current therapies. Viridian’s most advanced program, VRDN-001,
is a differentiated monoclonal antibody targeting insulin-like
growth factor-1 receptor (IGF-1R), a clinically and commercially
validated target for the treatment of thyroid eye disease (TED).
VRDN-002 is a distinct anti-IGF-1R antibody and incorporates
half-life extension technology. VRDN-003 is an extended half-life
version of VRDN-001. Both VRDN-002 and VRDN-003 are designed for
administration as convenient, low-volume, subcutaneous
injections. TED is a debilitating autoimmune disease that
causes inflammation and fibrosis within the orbit of the eye which
can cause double vision, pain, and potential blindness. Viridian is
based in Waltham, Massachusetts.
Note Regarding Forward-Looking StatementsThis
press release contains forward-looking statements within the
meaning of the Private Securities Litigation Reform Act of 1995.
These statements may be identified by the use of words such as, but
not limited to, "anticipate," "believe," "continue," "could,"
"estimate," "expect," "intend," "may," "might," "plan,"
"potential," "predict," "project," "should," "target," "will," or
"would" or other similar terms or expressions that concern the
Company’s expectations, plans and intentions. Forward-looking
statements include, without limitation, statements regarding the
Company’s expectations, strategies, plans and intentions.
Forward-looking statements are neither historical facts nor
assurances of future performance. Instead, they are based on the
Company’s current beliefs, expectations, and assumptions. New risks
and uncertainties may emerge from time to time, and it is not
possible to predict all risks and uncertainties. No representations
or warranties (expressed or implied) are made about the accuracy of
any such forward-looking statements. Such forward-looking
statements are subject to a number of material risks and
uncertainties including but not limited to: the potential efficacy
and safety of VRDN-001 and VRDN-002 for the treatment of TED; the
relationship between the results from the positive data from the
ongoing Phase 1/2 clinical trial of VRDN-001 and the first-in-human
Phase 1 clinical trial of VRDN-002 and results of ongoing and
future clinical trials; the timing, progress and plans for the
Company’s ongoing and future research and clinical development
programs; trial protocols for ongoing clinical trials, including
the clinical trials for VRDN-001 and VRDN-002; expectations
regarding the timing for data, including the expected timing of
additional data from the ongoing Phase 1/2 clinical trial of
VRDN-001 and the first-in-human Phase 1 clinical trial of VRDN-002
and VRDN-003; uncertainty and potential delays related to clinical
drug development; the duration and impact of regulatory delays in
the Company’s clinical programs; manufacturing risks; competition
from other therapies or products; other matters that could affect
the sufficiency of existing cash, cash equivalents and short-term
investments to fund operations; the Company’s financial position
and its projected cash runway; the Company’s future operating
results and financial performance; the timing of pre-clinical and
clinical trial activities and reporting results from same;
potential addressable market size; the effects from the COVID-19
pandemic on the Company’s research, development and business
activities and operating results, including those risks set forth
under the caption “Risk Factors” in the Company’s Annual Report on
Form 10-K filed with the Securities and Exchange
Commission (SEC) on March 11, 2022 and other
subsequent disclosure documents filed with the SEC. Any
forward-looking statement speaks only as of the date on which it
was made. Neither the Company, nor its affiliates, advisors, or
representatives, undertake any obligation to publicly update or
revise any forward-looking statement, whether as a result of new
information, future events or otherwise, except as required by law.
These forward-looking statements should not be relied upon as
representing the Company’s views as of any date subsequent to the
date hereof.
Investor and Media ContactJohn JordanViridian
TherapeuticsVice President, Investor Relations& Corporate
Communications617-272-4691IR@viridiantherapeutics.com
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