Full Press Release Details
Announces Positive Topline Results from KOMFORT Phase 2 Trial of Oral Difelikefalin for the Treatment of Pruritus in Patients with Notalgia
Study achieved primary endpoint of Worst Itch-Numeric Rating Scale score change from baseline at Week 8 (p=0.001) -
Onset of action seen at Week 1 and sustained through Week 8 -
Statistical significance
achieved on the WI-NRS 4-point responder analysis at Week 8 (p=0.007) -
Oral difelikefalin was well tolerated with a consistent safety profile -
Conference call today at 8:30 a.m. ET -
STAMFORD, Conn., June 30, 2022 - Cara
Therapeutics, Inc. (Nasdaq: CARA), a commercial-stage biopharmaceutical company leading a new treatment paradigm to improve
the lives of patients suffering from pruritus, today announced positive topline results from its Phase 2 proof-of-concept clinical trial
(KOMFORT) evaluating oral difelikefalin for the treatment of moderate-to-severe pruritus in patients with notalgia paresthetica (NP),
a nerve disorder characterized by chronic pruritus of the upper to middle back.
"We are pleased to have demonstrated clinical
proof of concept for oral difelikefalin in the treatment of pruritus associated with notalgia paresthetica," said Joana Goncalves,
M.D., Chief Medical Officer at Cara Therapeutics. "These topline results coupled with the results from our other programs support
the broad development of oral difelikefalin across disease areas regardless of the underlying cause of pruritus. We look forward to completing
our data analyses and discussing next steps with the U.S. Food and Drug Administration."
"With no approved treatments available
for notalgia paresthetica, the condition is challenging to manage and burdensome for patients," said Mark Lebwohl, M.D., the
lead investigator and Professor and Dean for Clinical Therapeutics and Chairman Emeritus of the Department of Dermatology at Icahn
School of Medicine at Mount Sinai. "These are encouraging results that underscore the potential for oral difelikefalin to be
the first treatment option to address pruritus associated with notalgia paresthetica."
Phase 2 Proof-of-Concept Trial Design &
The Phase 2 multicenter, randomized, double-blind,
placebo-controlled, 8-week study was designed to evaluate the efficacy and safety of oral difelikefalin for moderate-to-severe pruritus
in approximately 120 patients with NP. Patients were randomized to oral difelikefalin 2 mg taken twice daily versus placebo for 8 weeks,
followed by a 4-week active extension period.
The primary efficacy endpoint was the change
from baseline in the weekly mean of the daily 24-hour Worst Itch-Numeric Rating Scale (WI-NRS) score at Week 8. Other endpoints included
the 4-point responder analysis, itch-related quality of life scores, and safety assessments.
Patients treated with oral difelikefalin achieved
the primary endpoint (-4.0 difelikefalin vs. -2.4 placebo, p=0.001) with significant improvement observed as early as Week 1 and sustained
In addition, a statistically significantly
greater proportion of patients treated with oral difelikefalin achieved a 4-point improvement in WI-NRS score at Week 8 vs.
placebo (41% difelikefalin vs. 18% placebo, p=0.007).
Oral difelikefalin was generally well tolerated
with a safety profile consistent with that seen in earlier clinical trials. The most common treatment-emergent adverse events reported
in 5% of patients treated with oral difelikefalin and greater than placebo were: nausea, headache, dizziness, constipation and
urine output increased.
Conference Call & Webcast
Cara management will host a conference call and
live webcast today at 8:30 a.m. ET to discuss the positive topline results.
To participate in the conference call, please
dial (855) 445-2816 (domestic) or (484) 756-4300 (international) and refer to conference ID 6999079. A live webcast of the call can be
accessed under "Events & Presentations" in the News & Investors section of the Company's website at www.CaraTherapeutics.com.
An archived webcast recording will be available
on the Cara website beginning approximately two hours after the call.
About Pruritus Associated with Notalgia Paresthetica
Notalgia paresthetica (NP) is a common, although
under-recognized, chronic, sensory neuropathy affecting the upper back.1 It is estimated that chronic pruritus affects up
to 13% of the population in the United States, and about 8% of these patients suffer from neuropathic itch, including NP.2,3
One of the hallmark features of NP is chronic pruritus, which can be significantly burdensome and undermines the affected patients'
quality of life and overall well-being.3 The exact etiology of NP still has not been fully elucidated; however, it is widely
accepted that NP is a sensory neuropathy caused by alteration and damage to thoracic spinal nerves.3
The management of NP is challenging and is often
resistant to multiple therapies. There is currently no approved treatment for NP and conventional treatments for pruritus, such as antihistamines
and topical steroids, are largely ineffective.4
About Cara Therapeutics
Cara Therapeutics is a commercial-stage biopharmaceutical company
leading a new treatment paradigm to improve the lives of patients suffering from pruritus. The Company's novel KORSUVA (difelikefalin)
injection is the first and only FDA-approved treatment for moderate-to-severe pruritus associated with chronic kidney disease in adults
undergoing hemodialysis. The Company is developing an oral formulation of difelikefalin and has initiated Phase 3 programs for the treatment
of pruritus in patients with non-dialysis dependent advanced chronic kidney disease and atopic dermatitis. The Company has completed
the placebo-controlled phase of a Phase 2 proof-of-concept trial of oral difelikefalin for the treatment of moderate-to-severe pruritus
in patients with notalgia paresthetica. A Phase 2 proof-of-concept trial in primary biliary cholangitis patients with moderate-to-severe
pruritus is ongoing. For more information, visit www.CaraTherapeutics.com and follow the company on Twitter, LinkedIn
Forward-looking Statements
Statements contained in this press release regarding matters that
are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform
Act of 1995. Examples of these forward-looking statements include statements concerning the Company's planned future
regulatory submissions and potential future regulatory approvals, expected timing of the initiation, enrollment and data readouts
from the Company's planned and ongoing clinical trials, the potential results of ongoing clinical trials, timing of future
regulatory and development milestones for the Company's product candidates, the potential for the Company's product
candidates to be alternatives in the therapeutic areas investigated, including NP, and the potential for oral difelikefalin to
address additional pruritic indications, the size and growth of the potential markets for pruritus management, the Company's
expected cash reach, and the potential impact of COVID-19 on the Company's clinical development and regulatory timelines and
plans. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or
implied by such forward-looking statements. Risks are described more fully in Cara Therapeutics' filings with the Securities
and Exchange Commission, including the "Risk Factors" section of the Company's Annual Report on Form 10-K for
the year ending December 31, 2021 and its other documents subsequently filed with or furnished to the Securities and Exchange
Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. Cara
Therapeutics undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the
date on which they were made, except as required by law.
Iris Francesconi, Ph.D.