Corbus Pharmaceuticals Announces Topline Results from RESOLVE-1 Phase 3 Study of Lenabasum for Treatment of Systemic Sclerosis Topline data showed no significant differences in the primary and secondary endpoints when co

Pharmaceuticals Announces Topline Results from RESOLVE-1 Phase 3 Study of Lenabasum for Treatment of Systemic Sclerosis

MA, September 8, 2020 - Corbus Pharmaceuticals Holdings, Inc. (NASDAQ: CRBP) ("Corbus" or the "Company"),

a clinical-stage drug development company pioneering transformative medicines that target the endocannabinoid system, today announced topline results from the 52-week Phase 3 RESOLVE-1 study of lenabasum in patients with diffuse cutaneous

systemic sclerosis (SSc). SSc is a rare and life-threatening multi-system autoimmune disease for which there are currently no

U.S. Food and Drug Administration (FDA)-approved treatments for overall disease.

data showed no significant differences in the primary and secondary endpoints when comparing lenabasum to placebo, both added

to background drug therapy.

the primary endpoint, the median American College of Rheumatology Combined Response Index for Systemic Sclerosis (ACR

CRISS) scores at Week 52 were 0.887 in the placebo arm and 0.888 in the lenabasum 20 mg twice daily arm. ACR CRISS

is a composite endpoint that reflects

the probability of patient improvement. The

maximum achievable ACR CRISS score is 1.0.

is the first 52-week, randomized, placebo-controlled Phase 3 trial that tested the efficacy and safety of lenabasum in

365 patients with diffuse cutaneous SSc in a multinational, double-blind, randomized, placebo-controlled study, with dosing

of lenabasum at 20 mg twice daily, lenabasum at 5 mg twice daily, or placebo twice daily for 52 weeks. The majority of

enrolled patients (84%) were receiving background immunosuppressive drugs, reflecting recent trends in clinical practice.

proportions of placebo-treated and lenabasum-treated subjects had at least one treatment emergent adverse event (AEs), 86.2% in

the placebo arm and 91.7% in the lenabasum 20 mg twice daily arm. Serious AEs occurred in 14.6% of subjects in the control arm

and 9.2% of subjects in the lenabasum 20 mg twice daily arm. Severe AEs occurred in 13% of subjects in the control arm

and 5.8% of subjects in the lenabasum arm. No subjects receiving lenabasum withdrew from the study because of an AE-related

to study drug. Lenabasum treatment was well-tolerated in this study. No evidence of lenabasum-related immunosuppression or

new safety signals for lenabasum were observed.

analyses of these data are underway, and once Corbus has a fuller understanding of the data, the Company would

like to engage with the FDA to determine potential next steps in this clinical development program. The data

will be presented at upcoming medical conferences.

Cohen, Ph.D., Chief Executive Officer of Corbus said, "We are surprised and deeply disappointed that the RESOLVE-1

trial did not meet its primary endpoint. I would like to extend my gratitude to the participants in the study and

the clinical staff at the study sites, as well as to the Corbus employees, for their hard work and dedication.

We now look forward to upcoming topline results from our study of lenabasum in patients with cystic fibrosis."

Spiera, M.D., Co-Principal Investigator on RESOLVE-1 and Director of the Scleroderma, Vasculitis, and Myositis Program

at the Hospital for Special Surgery, Weill Cornell Medical College in New York City said,

"I am genuinely surprised by these results. Immunosuppressive drugs, alone or in combination, are increasingly becoming

a mainstay of treatment for patients with early diffuse cutaneous SSc. However, the impact of these drugs on disease has not previously

been studied systematically and clearly was underappreciated by the community of SSc experts. The high degree of efficacy of background

drug therapy in the control arm is well beyond what was expected."

Christopher Denton, PhD, FRCP, Co-Principal Investigator on RESOLVE-1 and Professor

of Experimental Rheumatology at UCL Medical School and Consultant Rheumatologist and Joint Director of the Centre for Rheumatology,

Royal Free Hospital, London said, "Whilst the immediate study results are disappointing, RESOLVE-1 provides a rich dataset

to understand for the first time how to better target treatments for SSc based upon clinical parameters and concomitant treatment.

We are already querying the data to understand the natural history of early diffuse cutaneous SSc and the potential benefits of

lenabasum in these subjects. Despite the efficacy of current immunosuppressive treatments in early diffuse cutaneous systemic

sclerosis, there is still major unmet need in this patient group. The potential value of a non-immunosuppressive treatment, added-to

or used instead of, additional immunosuppressive medications, remains exciting. The safety profile and tolerability of lenabasum

is very attractive for use in SSc patients."

White, M.D., Chief Medical Officer and Head of Research of Corbus commented, "We will now focus on further analyses of the

data to potentially identify groups of patients that may have responded to lenabasum."

was granted Orphan Drug designation and Fast Track designation for the treatment of SSc from the FDA and Orphan Designation for

the treatment of SSc from the European Medicines Agency.

is currently being evaluated in a Phase 3 DETERMINE study in dermatomyositis, a Phase 2 study in systemic

lupus erythematosus, and a Phase 2b study in cystic fibrosis.

Phase 3 Study Trial Design

RESOLVE-1 Phase 3 trial tested the efficacy and safety of lenabasum in people with diffuse cutaneous SSc on background drug therapy,

in North America, Europe, Asia, Israel, and Australia. This was a double-blind, randomized, placebo-controlled study, with dosing

of lenabasum at 20 mg twice daily, lenabasum at 5 mg twice daily, or placebo twice daily for 52 weeks.

hundred and sixty-five patients were dosed in the study. Baseline characteristics of patients across groups were balanced.

primary efficacy outcome for the Phase 3 RESOLVE-1 is a composite clinical trial endpoint known as the ACR CRISS score, assessed

at Week 52. The ACR CRISS score was also the primary efficacy endpoint in the preceding Phase 2 study published in Arthritis

& Rheumatology in April 2020. The ACR CRISS score is a composite clinical trial endpoint that assesses probability that

the subject has improved from baseline and integrates change from baseline in five endpoints selected by experts to be

the most relevant indicators of disease improvement in diffuse cutaneous SSc. Secondary efficacy endpoints include three

of the five core items of ACR CRISS assessed as change from baseline at Week 52 in mRSS, Health Assessment Questionnaire-Disability

index (HAQ-DI), and forced vital capacity (FVC) percent predicted.

will host a conference call and webcast presentation today, Tuesday, September 8th, 2020 at 8:30 a.m. ET.

participate in the call, please dial (877) 407-3978 (domestic) or (412) 902-0039 (international). The live webcast will be accessible

on the Events page of the investors section of the Corbus website, www.corbuspharma.com, and will be archived for

is a rationally designed, oral, small molecule that selectively binds as an agonist to the cannabinoid receptor type 2 (CB2),

resolves inflammation, and limits fibrosis. CB2 is preferentially expressed on activated immune cells and on fibroblasts, muscle

cells, and endothelial cells. In both animal and human studies conducted to date, lenabasum has induced the production of pro-resolving

lipid mediators that activate endogenous pathways which resolve inflammation and speed bacterial clearance without immunosuppression.

Data from animal models and human clinical studies suggest that lenabasum can reduce expression of genes and proteins involved

in inflammation and fibrosis. Lenabasum has demonstrated promising activity in animal models of skin and lung inflammation and

fibrosis in systemic sclerosis (SSc). Lenabasum is also active in animal models of lung infection and inflammation in cystic fibrosis

and joint inflammation and scarring in rheumatoid arthritis.

has demonstrated acceptable safety and tolerability profiles in clinical studies to date. Lenabasum treatment was associated with

improvement in multiple physician-assessed and patient-reported efficacy outcomes in Phase 2 studies in patients with diffuse

cutaneous SSc and patients with dermatomyositis with active skin involvement but not currently active muscle involvement. Lenabasum

treatment also was associated with a lower rate of and longer time to pulmonary exacerbations in a Phase 2 cystic fibrosis study.

is not approved for the treatment of systemic sclerosis, dermatomyositis, cystic fibrosis or systemic lupus erythematosus.

sclerosis, a form of scleroderma, is a chronic, rare, debilitating autoimmune disease affecting approximately 200,000 people in

the North America, EU and Japan.1 Systemic sclerosis is considered one of the most life-threatening rheumatic diseases.2

The disease affects affects the skin and internal organs and is driven by inflammation and fibrosis (scarring of tissue)

which can lead to severe damage and failure of multiple organs including the skin, joints, tendons, gastrointestinal tract, lungs,

heart, blood vessels and kidneys.3 There is no cure for systemic sclerosis, and current treatments address the clinical

manifestations of the disease, not the underlying mechanisms that drive inflammation and fibrosis.4

Pharmaceuticals Holdings, Inc. is a Phase 3 clinical-stage pharmaceutical company focused on the development and commercialization

of novel therapeutics to treat inflammatory and fibrotic diseases by leveraging its pipeline of rationally designed, endocannabinoid

system-targeting drug candidates. The Company's lead product candidate, lenabasum, is a novel, oral, selective cannabinoid

receptor type 2 (CB2) agonist rationally designed to resolve chronic inflammation and fibrotic processes. Lenabasum is currently

being evaluated in systemic sclerosis, cystic fibrosis, dermatomyositis and systemic lupus erythematosus.

is also developing a pipeline of drug candidates targeting the endocannabinoid system. The pipeline includes CRB-4001, a 2nd generation,

selective cannabinoid receptor type 1 (CB1) inverse agonist designed to be peripherally restricted. Potential indications for

CRB-4001 include nonalcoholic steatohepatitis (NASH), among others.

is not approved for the treatment of systemic sclerosis, dermatomyositis, cystic fibrosis or systemic lupus erythematosus. CRB-4001

is not approved for the treatment of NASH/NAFLD. For more information on Corbus' clinical programs, please visit here.

visit www.CorbusPharma.com and connect with the Company on Twitter, LinkedIn, and Facebook.

press release contains certain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and

Section 21E of the Securities Exchange Act of 1934 and Private Securities Litigation Reform Act, as amended, including those relating

to the Company's trial results, product development, clinical and regulatory timelines, market opportunity, competitive

position, possible or assumed future results of operations, business strategies, potential growth opportunities and other statement