Kiniksa Pharmaceuticals Stock

Kiniksa Pharmaceuticals, Ltd. operates as a commercial-stage biopharmaceutical company. The company focuses on discovering, acquiring, developing and commercializing therapeutic medicines for patients suffering from debilitating diseases with significant unmet medical need.

The company’s portfolio of immune-modulating assets, ARCALYST (rilonacept), abiprubart (also known as KPL-404) and mavrilimumab, is based on strong biologic rationale or validated mechanisms, targets a spectrum of underserved cardiovascular and autoimmune conditions, and offers the potential for differentiation.

ARCALYST is an interleukin-1a and interleukin-1ß cytokine trap. In 2017, the company licensed ARCALYST from Regeneron, which discovered and initially developed the drug. The company’s exclusive license to ARCALYST from Regeneron includes worldwide rights, excluding the Middle East and North Africa, for all applications other than those in oncology and local administration to the eye or ear. In February 2022, the company granted Hangzhou Zhongmei Huadong Pharmaceutical Co., Ltd. (Huadong) exclusive rights to develop and commercialize ARCALYST in the Asia Pacific region, excluding Japan.

The company received FDA approval of ARCALYST for the treatment of recurrent pericarditis and reduction in risk of recurrence in adults and children 12 years and older in March 2021. Recurrent pericarditis is a painful inflammatory cardiovascular disease with an estimated United States prevalent population of approximately 40,000 patients seeking and receiving medical treatment. ARCALYST is commercially available across the United States through a select network of distributors. ARCALYST is also approved in the United States for the treatment of Cryopyrin-Associated Periodic Syndromes (CAPS), including Familial Cold Autoinflammatory Syndrome (FCAS) and Muckle-Wells Syndrome in adults and children 12 years and older, and the maintenance of remission in Deficiency of Interleukin-1 Receptor Antagonist (DIRA) in adults and children weighing 10 kg or more. The company is responsible for sales and distribution of ARCALYST in all approved indications in the United States, and evenly split profits on sales, as well as third party proceeds with Regeneron. In March 2023, Regeneron initiated a technology transfer of the manufacturing process for ARCALYST drug substance, and the company is working with Regeneron to qualify a new CDMO.

Abiprubart (KPL-404) is an investigational monoclonal antibody inhibitor of CD40-CD154 costimulatory interaction. In 2019, the company acquired all of the outstanding securities of Primatope Therapeutics, Inc. (Primatope), the company that owned or controlled the intellectual property related to abiprubart. In connection with its acquisition of Primatope, the company acquired an exclusive world-wide license to abiprubart from Beth Israel Deaconess Medical Center, Inc. (BIDMC).

The CD40-CD154 interaction is a key T-cell co-stimulatory signal critical for B-cell maturation, immunoglobulin class switching and Type 1 immune response. Disrupting the CD40-CD154 interaction is an attractive approach to address multiple autoimmune disease pathologies.

In December 2021, the company initiated a Phase 2 clinical trial of abiprubart in rheumatoid arthritis (RA), which is designed to evaluate pharmacokinetics, safety and efficacy with subcutaneous administration. In January 2024, the company announced topline clinical data from Cohorts 1, 2 and 3 of the trial, and that the trial met its primary efficacy endpoint in Cohort 3 at the weekly dose level. The company expects to announce data from Cohort 4 of the trial in the second quarter of 2024.

Mavrilimumab is an investigational monoclonal antibody inhibitor targeting granulocyte-macrophage colony stimulating factor receptor alpha (GM-CSFRa). In 2017, the company licensed exclusive worldwide rights in all indications to mavrilimumab from MedImmune, Limited (MedImmune). In February 2022, the company granted Huadong exclusive rights to develop and commercialize mavrilimumab in the Asia Pacific region, excluding Japan.

The company is evaluating potential partnership opportunities to advance mavrilimumab’s development. The company previously evaluated mavrilimumab in giant cell arteritis (GCA), a chronic inflammatory disease of the medium-to-large arteries, and COVID-19-related acute respiratory distress syndrome (ARDS).

Using a data-centric approach, its team considers a wide variety of metrics to drive informed capital allocation strategies and generate value from the company’s portfolio of immune-modulating assets, including by analyzing potential additional indications for its products and product candidates, being opportunistic in its business development activities to in-license or acquire programs, considering appropriate opportunities to partner or out-license its programs and conducting internal research to discover and develop molecules to expand its portfolio.

Strategy

The core of the company’s strategy is the identification, development and commercialization of therapeutic medicines for patients suffering from debilitating diseases with significant unmet medical need. the company puts patients first and live by its motto: every second counts. The key elements of the company’s strategy are to explore opportunities to drive value and maximize the potential of its existing portfolio; commercialize ARCALYST and its product candidates, if approved; advance its product candidates through the development process; and work to identify, discover, acquire and develop new therapies.

Products

ARCALYST

ARCALYST is an interleukin-1a and interleukin-1ß cytokine trap. Cytokines are small proteins that play a role in cell signaling. The company received FDA approval of ARCALYST for the treatment of recurrent pericarditis and reduction in risk of recurrence in adults and children 12 years and older in March 2021. ARCALYST is also approved in the United States for the treatment of CAPS, including FCAS and Muckle-Wells Syndrome in adults and children 12 years and older, and the maintenance of remission in DIRA in adults and children weighing 10 kg or more. ARCALYST was sold by Regeneron in the United States for the treatment of CAPS from 2008 and DIRA from 2020 until the company assumed responsibility for sales in such indications in March 2021.

In June 2020, the company reported results from RHAPSODY, its global, double-blind, placebo-controlled, randomized-withdrawal design, pivotal Phase 3 clinical trial of ARCALYST in subjects with recurrent pericarditis. RHAPSODY met its prespecified primary and all major secondary efficacy endpoints with statistical significance, showing that ARCALYST improved clinically meaningful outcomes associated with unmet medical need in recurrent pericarditis. Subsequent data from the long-term extension portion of RHAPSODY, reported in 2022, demonstrated, among other things, that continued ARCALYST treatment beyond 18 months resulted in continued treatment response.

In February 2022, the company granted Huadong exclusive rights to develop and commercialize ARCALYST in the Asia Pacific region, excluding Japan.

Clinical Trials

In June 2020, the company reported results from RHAPSODY, its global, double-blind, placebo-controlled, randomized-withdrawal design, pivotal Phase 3 clinical trial of ARCALYST in subjects with recurrent pericarditis. RHAPSODY met its prespecified primary and all major secondary efficacy endpoints with statistical significance.

Product Candidates

Abiprubart

Abiprubart is an investigational monoclonal antibody inhibitor of CD40-CD154 costimulatory interaction. CD40-CD154 interaction is a key T-cell co-stimulatory signal critical for B-cell maturation, immunoglobulin class switching, and Type 1 immune response.

In 2021, the company reported positive final data from its randomized, double-blind, placebo-controlled, single-ascending-dose Phase 1 clinical trial of abiprubart in healthy volunteers which evaluated safety and pharmacokinetics, as well as receptor occupancy (RO) and T-dependent antibody response (TDAR) in these subjects.

The company is conducting a Phase 2 clinical trial of abiprubart in RA, which is designed to enable its potential development in a spectrum of autoimmune diseases believed to be mediated by the CD40-CD154 co-stimulatory interaction. In January 2024, the company announced topline clinical data from Cohorts 1, 2 and 3 of the trial and that the trial met its primary efficacy endpoint in Cohort 3 at the weekly dose level. The company expects to announce data from Cohort 4 of the trial in the second quarter of 2024. The company plans to make further development decisions based upon the totality of the data following the receipt of data from Cohort 4 of its Phase 2 clinical trial.

Phase 2 Trial in Rheumatoid Arthritis

In December 2021, the company commenced a Phase 2 clinical trial of abiprubart in RA. The Phase 2 trial is a randomized, double-blind, placebo-controlled study designed to provide pharmacokinetic data and early signal of efficacy with chronic administration, and optionality to evaluate abiprubart across a range of other autoimmune diseases. The trial enrolled participants with active RA who had an inadequate response to or were intolerant to a Janus kinase inhibitor (a JAKi) or at least one biologic disease-modifying anti-rheumatic drug (a bDMARD).

Mavrilimumab

Mavrilimumab is a fully-human monoclonal antibody that is designed to antagonize GM-CSF signaling by binding to the alpha subunit of the GM-CSF receptor. The company is evaluating potential partnership opportunities to advance mavrilimumab’s development. In February 2022, the company granted Huadong exclusive rights to develop and commercialize mavrilimumab in the Asia Pacific region, excluding Japan.

Solution

Mavrilimumab is designed to inhibit the signaling of GM-CSF, a growth factor that stimulates the production of certain types of white blood cells. In an ex vivo GCA artery culture model, mavrilimumab inhibited inflammatory molecules characteristic of GCA pathophysiology.

Discovery Activities

The company conducts internal discovery activities directed toward wholly owned molecules for the treatment of debilitating disease targets where there to be a strong mechanistic rationale and potential for clear differentiation from existing approved agents or those in development.

Commercial Operations

The company’s commercial team combines dozens of years of pharmaceutical commercial leadership experience with a passion for helping patients with significant unmet medical need. The company’s salesforce is complemented by its medical affairs, payor and patient services teams. The company has also built an efficient digital marketing effort by targeting prescribers treating recurrent pericarditis to complement the reach of its salesforce. The company intends to expand its capabilities in parallel with the development path of its product candidates.

License and Acquisition Agreements

Out-Licensing Agreements

Genentech Agreement

In August 2022, the company entered into a license agreement (the Genentech License Agreement) with Genentech, Inc. and F. Hoffmann-La Roche Ltd. (collectively, Genentech), pursuant to which it granted Genentech exclusive worldwide rights to develop, manufacture and commercialize vixarelimab and related antibodies (each, a Genentech Licensed Product). The Genentech License Agreement became effective in September 2022 following termination of the statutory waiting period under the Hart-Scott Rodino Act.

Huadong Collaboration Agreements

In 2022, the company entered into two collaboration and license agreements (each, a Huadong Collaboration Agreement and together, the Huadong Collaboration Agreements) with Huadong, pursuant to which it granted Huadong exclusive rights to develop and commercialize ARCALYST and develop, manufacture and commercialize mavrilimumab (each, a Huadong Licensed Product and together, the Huadong Licensed Products) in the following countries: People’s Republic of China, Hong Kong SAR, Macao SAR, Taiwan Region, South Korea, Indonesia, Singapore, The Philippines, Thailand, Australia, Bangladesh, Bhutan, Brunei, Burma, Cambodia, India, Laos, Malaysia, Maldives, Mongolia, Nepal, New Zealand, Sri Lanka, and Vietnam (collectively, the Huadong Territory). The company otherwise retains its rights to the Huadong Licensed Products outside the Huadong Territory.

In-Licensing Agreements

License Agreement with Regeneron

In 2017, the company entered into a license agreement with Regeneron (the Regeneron Agreement). Pursuant to the Regeneron Agreement, the company has an exclusive license under certain intellectual property rights controlled by Regeneron to develop and commercialize ARCALYST worldwide, excluding the Middle East and North Africa, for all indications other than those in oncology and local administration to the eye or ear. Upon receiving positive data in RHAPSODY, the company’s pivotal Phase 3 clinical trial of ARCALYST, Regeneron transferred the biologics license application (BLA) for ARCALYST to it. In March 2021, when the FDA granted approval of ARCALYST for the treatment of recurrent pericarditis and reduction in risk of recurrence in adults and children 12 years and older, the company assumed the sales and distribution of ARCALYST for CAPS and DIRA in the United States.

License Agreement with MedImmune

In December 2017, the company entered into a license agreement with MedImmune (the MedImmune Agreement), pursuant to which MedImmune granted it an exclusive, sublicensable, worldwide license to certain intellectual property rights to make, use, develop and commercialize mavrilimumab and any other product containing an antibody to the GM-CSF receptor alpha that is covered by certain MedImmune patent rights for all indications. The company also acquired non-exclusive licenses to other MedImmune technology for use in exploiting licensed products. The company also acquired reference rights to relevant manufacturing and regulatory documents, and MedImmune’s existing inventory of mavrilimumab drug substance and product. The company is obligated to use commercially reasonable efforts to develop and commercialize the licensed products.

Beth Israel Deaconess Medical Center License Agreement

In 2019, the company acquired all of the outstanding securities of Primatope Therapeutics, Inc. (Primatope), the company that owned or controlled the intellectual property related to abiprubart. In connection with its acquisition of Primatope, the company acquired the rights to an exclusive license to certain intellectual property rights controlled by BIDMC to make, use, develop and commercialize abiprubart under the BIDMC license agreement (the BIDMC Agreement).

Commercial Operations

The company’s commercial team combines dozens of years of pharmaceutical commercial leadership experience with a passion for helping patients with significant unmet need.

Intellectual Property

ARCALYST

The company has a field-specific exclusive license under the Regeneron Agreement to granted patents and pending applications in the United States and numerous other jurisdictions relating to ARCALYST. As of December 31, 2023, the patent rights in-licensed under the Regeneron Agreement relating to its program include three granted patents in the United States and a patent granted in Japan. In addition, the patent rights in-licensed under the Regeneron Agreement relating to the company’s program include patent applications that are pending in the United States, Canada, Europe and selected countries in Asia. A United States patent covering ARCALYST as a composition of matter expired in 2020, and relevant composition of matter patents issued outside of the United States expired in 2023. Three patents covering methods of using ARCALYST in the treatment of recurrent pericarditis have issued in the United States and have a statutory term that expires in 2038, not including any patent term adjustment. In March 2021, the FDA granted approval for ARCALYST for the treatment of recurrent pericarditis and reduction in risk of recurrence in adults and children 12 years of age and older, which granted it seven years of marketing exclusivity in the United States.

Abiprubart

The company owns, via its acquisition of Primatope, granted patents and pending patent applications in the United States and numerous other jurisdictions relating to abiprubart. The company also has an exclusive license with BIDMC to granted patents and pending patent applications in the United States and numerous other jurisdictions relating to abiprubart. These patents and patent applications cover abiprubart as a composition of matter and its use. As of December 31, 2023, the patent rights acquired from Primatope include four patents granted in the United States and 32 patents granted in other jurisdictions, including Australia, Brazil and selected countries in Europe and Asia. In addition, the patent rights acquired from Primatope include patent applications pending in the United States, Australia, Europe, Canada, and selected countries in Asia. The issued composition of matter patents acquired from Primatope have statutory expiration dates in 2036, not including any patent term extensions or adjustments. As of December 31, 2023, the patent rights licensed from BIDMC include two patents granted in the United States and 33 patents granted in other jurisdictions, including Canada and selected countries in Europe and Asia. In addition, the patent rights licensed from BIDMC include patent applications pending in the United States, Europe and Canada. The issued composition of matter patents licensed from BIDMC have statutory expiration dates in 2032, not including any patent term extensions or adjustments. Patent term extension could extend the expiration date of one patent in the United States and patents in certain other jurisdictions, each in accordance with applicable law.

Mavrilimumab

The company has an exclusive license under the MedImmune Agreement to granted patents and pending patent applications in the United States and numerous other jurisdictions relating to mavrilimumab. These patents and patent applications cover mavrilimumab as a composition of matter and its use. As of December 31, 2023, the patent rights in-licensed under the MedImmune Agreement relating to the company’s program include three granted patents in the United States and 113 patents granted in other jurisdictions, including Canada, Australia and selected countries in Europe and Asia. In addition, the patent rights in licensed under the MedImmune Agreement relating to its program include patent applications that are pending in the United States, Europe, Canada, and selected countries in Asia and Latin America.

Other Intellectual Property

In addition to the above, the company maintains certain other intellectual property, including trademarks and know-how, related to its pre-clinical development and broader Kiniksa brand.

Research and Development

Research and development expenses were $76.1 million for the year ended December 31, 2023.

Government Regulation

The company is subject to federal laws, including the Medicaid Drug Rebate Program (the MDRP). The company’s products are eligible to be reimbursed by Medicaid. Medicaid is a joint federal and state program that is administered by the states for low-income and disabled beneficiaries. Under the MDRP, participating manufacturers are required to pay a rebate for each unit of product reimbursed under the state Medicaid programs. The amount of the rebate for each product is set by law and depends in part on the prices at which its products are sold to certain other purchasers and may be subject to an additional discount if certain pricing increases more than inflation.

History

Kiniksa Pharmaceuticals, Ltd. was founded in 2015. The company was incorporated under the laws of Bermuda in 2015.