On February 23, 2021 Kiniksa Pharmaceuticals, Ltd. (Nasdaq: KNSA) ("Kiniksa"), a biopharmaceutical company with a pipeline of assets designed to modulate immunological pathways across a spectrum of diseases, reported fourth quarter and full-year 2020 financial results and corporate and pipeline activity (Press release, Kiniksa Pharmaceuticals, FEB 23, 2021, View Source [SID1234575440]).

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"Kiniksa delivered outstanding performance in 2020, with encouraging clinical data across our pipeline as well as Breakthrough Therapy designation or Orphan Drug designation for rilonacept, mavrilimumab and vixarelimab," said Sanj K. Patel, Chief Executive Officer and Chairman of the Board of Kiniksa. "Building on this momentum, we expect to launch rilonacept in recurrent pericarditis, pending FDA approval, in the first half of 2021. We also anticipate final Phase 1 data for KPL-404, our anti-CD40 program, in which data generated to-date support the best-in-class potential of this molecule and the opportunity for its evaluation in multiple devastating autoimmune diseases. Further, we expect data from the fully-enrolled mavrilimumab Phase 2 clinical trial in severe COVID-19 in the first half of this year. With these significant advances, it is important to highlight we are well capitalized with cash reserves of approximately $323 million."

Pipeline Activity
Rilonacept (IL-1α and IL-1β cytokine trap)

Kiniksa is preparing for the commercial launch of rilonacept in recurrent pericarditis in the first half of 2021, if approved by the U.S. Food and Drug Administration (FDA).
The FDA accepted the supplemental Biologics License Application (sBLA) for rilonacept in recurrent pericarditis with priority review and assigned a Prescription Drug User Fee Act (PDUFA) goal date of March 21, 2021.
Upon approval in recurrent pericarditis, Kiniksa will commence sales and distribution of rilonacept for the approved indications in the United States, including cryopyrin-associated periodic syndromes (CAPS), and deficiency of IL-1 receptor antagonist (DIRA), and evenly split profits with Regeneron Pharmaceuticals, Inc. (Regeneron).
Kiniksa is building commercial competencies in-house, including sales operations, value and access, and sales and marketing teams. In addition, the company is generating evidence on unmet need and disease burden, building disease awareness with payers, physicians, and advocacy groups, and establishing core capabilities such as distribution, patient services and data management.
Kiniksa achieved a regulatory milestone in the fourth quarter of 2020, which triggered a $7.5 million payment to Regeneron. The company is obligated to make a regulatory milestone payment of $20 million through the potential approval of rilonacept in recurrent pericarditis.
Mavrilimumab (monoclonal antibody inhibitor targeting GM-CSFRα)

Kiniksa expects to provide next steps for mavrilimumab, including for giant cell arteritis (GCA), in the first half of 2021.
Kiniksa is conducting a Phase 2/3 clinical trial of mavrilimumab in severe COVID-19 pneumonia and hyperinflammation.
Kiniksa completed enrollment in the Phase 2 portion of the clinical trial and expects data in the first half of 2021.
Kiniksa is enrolling patients in the Phase 3 portion of the clinical trial.
Vixarelimab (monoclonal antibody inhibitor of signaling through OSMRβ)

Kiniksa is conducting a Phase 2b dose-ranging clinical trial of vixarelimab in prurigo nodularis.
The Phase 2b clinical trial is expected to enroll approximately 180 patients experiencing severe pruritus. Patients will be randomized to receive vixarelimab or placebo subcutaneously once-monthly.
The primary efficacy endpoint is the percent change from baseline in the weekly-average Worst-Itch Numeric Rating Scale (WI-NRS) at Week 16.
Key secondary efficacy endpoints include the proportion of patients achieving a greater-than-or-equal-to 4-point weekly-average WI-NRS reduction at Week 16 and the proportion of patients achieving a 0/1 score (clear/almost clear) on the prurigo nodularis-investigator’s global assessment (PN-IGA) at Week 16.
KPL-404 (monoclonal antibody inhibitor of signaling between CD40 and CD40L)

Kiniksa expects final data and safety follow-up from all cohorts of the single-ascending-dose Phase 1 clinical trial of KPL-404 in the first half of 2021.
Preliminary Phase 1 data showed full receptor occupancy through Day 29 at the 3 mg/kg intravenous dose.
This data corresponded with complete suppression of the T-cell Dependent Antibody Response (TDAR) to the novel antigen keyhole limpet hemocyanin (KLH) through Day 29.
KPL-404 is a monoclonal antibody inhibitor designed to disrupt CD40-CD40 ligand (CD40L) signaling, a well-known pathway that plays a critical role in regulating B cell proliferation and T cell activation as well as antibody production.
Dysregulation of the CD40-CD40L pathway has been implicated in a broad range of autoimmune diseases including rheumatoid arthritis, Sjogren’s syndrome, Graves’ disease, systemic lupus erythematosus and the prevention of solid organ transplant graft rejection.
Financial Results

Net loss for the fourth quarter of 2020 was $53.7 million, compared to a net loss of $31.8 million for the fourth quarter of 2019. Net loss for the full-year 2020 was $161.4 million, compared to a net loss of $161.9 million for the full-year 2019.
Total operating expenses for the fourth quarter of 2020 were $52.9 million, compared to $32.6 million for the fourth quarter of 2019. Total operating expenses for the full-year 2020 were $157.4 million, compared to $170.0 million for the full-year 2019.
Non-cash, share-based compensation expense for the fourth quarter of 2020 was $6.3 million, compared to $5.0 million for the fourth quarter of 2019. Non-cash, share-based compensation expense for the full-year 2020 was $20.9 million, compared to $15.1 million for the full-year 2019.
Total operating expenses for the fourth quarter and full-year 2020 included a $7.5 million payment made to Regeneron related to our achievement of a regulatory milestone.
As of December 31, 2020, the company had cash, cash equivalents and short-term investments of $323.5 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.