On May 3, 2021 Kiniksa Pharmaceuticals, Ltd. (Nasdaq: KNSA) ("Kiniksa"), a biopharmaceutical company with a portfolio of assets designed to modulate immunological pathways across a spectrum of diseases, reported first quarter 2021 financial results and recent corporate and portfolio activity (Press release, Kiniksa Pharmaceuticals, MAY 4, 2021, View Source [SID1234579103]).

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"The first quarter was transformational for Kiniksa with the approval of ARCALYST as the first and only FDA-approved therapy for patients with recurrent pericarditis. We are focused on the launch of ARCALYST and are confident in our commercialization strategy," said Sanj K. Patel, Chief Executive Officer and Chairman of the Board of Kiniksa. "Additionally, we are executing across our broader portfolio of immune-modulating assets. We recently reported positive data for mavrilimumab in severe COVID-19 pneumonia and hyperinflammation and remain engaged with the FDA and other government agencies to identify pathways for accelerated availability of mavrilimumab as a potential therapeutic option for this patient population. We also reported positive final Phase 1 data for our potentially best-in-class anti-CD40 program, KPL-404, and plan to initiate a Phase 2 proof-of-concept trial in the second half of 2021."

Portfolio Activity
ARCALYST (IL-1α and IL-1β cytokine trap)

Kiniksa received approval from the U.S. Food and Drug Administration (FDA) on March 18, 2021, for ARCALYST for the treatment of recurrent pericarditis and reduction in risk of recurrence in adults and children 12 years and older. The commercial launch of ARCALYST in recurrent pericarditis commenced in April 2021.
Kiniksa is responsible for sales and distribution of ARCALYST for all the approved indications in the United States, including cryopyrin-associated periodic syndromes (CAPS) and deficiency of IL-1 receptor antagonist (DIRA), and will evenly split profits with Regeneron Pharmaceuticals, Inc. (Regeneron).
Kiniksa is executing on its commercial strategy, including engagement with priority accounts and payers to enable rapid and broad access to ARCALYST for patients.
Kiniksa is enrolling pediatric and adult patients with recurrent pericarditis in the RESONANCE registry.
Mavrilimumab (monoclonal antibody inhibitor targeting GM-CSFRα)

Kiniksa expects to provide next steps for mavrilimumab, including for giant cell arteritis (GCA), in the second quarter of 2021.
Kiniksa announced data from the Phase 2 portion of the Phase 2/3 clinical trial of mavrilimumab in non-mechanically ventilated patients with severe COVID-19 pneumonia and hyperinflammation receiving local standard of care. Non-mechanically ventilated patients treated with mavrilimumab demonstrated a reduction in mechanical ventilation and death at Day 29 pooled across dose cohorts.
Kiniksa continues to advance its engagement activities with the FDA and other government agencies to identify pathways to potentially accelerate availability of mavrilimumab as a therapeutic option for severe COVID-19 patients. Enrollment in the Phase 3 portion of the trial is ongoing.
Vixarelimab (monoclonal antibody inhibitor of signaling through OSMRβ)

Kiniksa is conducting a placebo-controlled Phase 2b clinical trial of vixarelimab in prurigo nodularis, evaluating a range of once-monthly dose regimens via subcutaneous (SC) injection.
The primary efficacy endpoint is the percent change from baseline in the weekly-average Worst-Itch Numeric Rating Scale at Week 16.
KPL-404 (monoclonal antibody inhibitor of signaling between CD40 and CD154)

Kiniksa announced final data today from the KPL-404 Phase 1 clinical trial in healthy volunteers. KPL-404 was well tolerated and showed dose-dependent increases in concentration across cohorts. The data support further development in patients with optionality for intravenous and/or SC administration.
Kiniksa plans to initiate a Phase 2 proof-of-concept clinical trial of KPL-404 in rheumatoid arthritis in the second half of 2021. The planned trial will provide safety and characterization of chronic dosing with SC administration over 12 weeks as well as the potential to evaluate KPL-404 across a range of other autoimmune diseases.
Scientific Conference Presentations

Kiniksa plans to present additional data from the RHAPSODY, the pivotal Phase 3 trial of rilonacept, at the American College of Cardiology virtual scientific conference, which will be available starting on May 15, 2021 at 8:00 a.m. Eastern Time. Details of the presentations are as follows:
Antonio Brucato, MD, Department of Biomedical and Clinical Science, University of Milan, Fatebenefratelli Hospital, Milan, will present a poster entitled, Tapering and discontinuation of background therapies during the transition to rilonacept monotherapy in RHAPSODY, a phase 3 clinical trial of rilonacept in patients with recurrent pericarditis.
Paul Cremer, MD, Department of Cardiovascular Medicine, Cleveland Clinic, Cleveland, will present a moderated poster entitled, Cardiac magnetic resonance imaging for guiding decision-making on treatment duration: data from RHAPSODY, a phase 3 clinical trial of rilonacept in recurrent pericarditis.
Financial Results

Net loss for the first quarter of 2021 was $49.5 million, compared to a net loss of $26.4 million for the first quarter of 2020.
Total operating expenses for the first quarter of 2021 were $49.3 million, compared to $29.4 million for the first quarter of 2020.
Non-cash, share-based compensation expense for the first quarter of 2021 was $7.1 million, compared to $4.2 million for the first quarter of 2020.
Kiniksa made a $20.0 million milestone payment to Regeneron in the first quarter of 2021 upon the FDA approval of ARCALYST in recurrent pericarditis. The milestone payment was capitalized as an intangible asset and will be amortized through cost of goods sold on a straight-line basis over the 20-year life of the asset starting in the second quarter of 2021.
As of March 31, 2021, the company had cash, cash equivalents and short-term investments of $264.0 million and no debt.
Financial Guidance

Kiniksa expects that its cash, cash equivalents and short-term investments will fund its current operating plan into 2023.