Ligand Pharmaceuticals Incorporated (Ligand) operates as a biopharmaceutical company.
The company is enabling scientific advancement through supporting the clinical development of high-value medicines. The company’s business model seeks to generate value for stockholders by creating a diversified portfolio of biopharmaceutical product revenue streams.
The company’s business model focuses on funding programs in mid- to late-stage drug development in return for economic rights, purchasing royalt...
Ligand Pharmaceuticals Incorporated (Ligand) operates as a biopharmaceutical company.
The company is enabling scientific advancement through supporting the clinical development of high-value medicines. The company’s business model seeks to generate value for stockholders by creating a diversified portfolio of biopharmaceutical product revenue streams.
The company’s business model focuses on funding programs in mid- to late-stage drug development in return for economic rights, purchasing royalty rights in development stage or commercial biopharmaceutical products and licensing its technology to help partners discover and develop medicines. The company’s partner with other pharmaceutical companies to leverage what they do best (late-stage development, regulatory management and commercialization) to generate its revenue. The company operates two infrastructure-light royalty-generating IP platform technologies. The company’s Captisol platform technology is a chemically modified cyclodextrin with a structure designed to optimize the solubility and stability of drugs. The company’s NITRICIL platform technology facilitates ‘tunable’ dosing, permitting an adjustable drug release profile to allow proprietary formulations that target a broad range of indications. The company has established multiple alliances, licenses and other business relationships with the world’s leading biopharmaceutical companies including Amgen, Merck, Pfizer, Jazz, Gilead Sciences and Baxter.
The company’s revenue consists of three primary elements: royalties from commercialized products, sales of its Captisol material to partners, and contract revenue from license fees and milestones payments.
Strategy
The company is a biopharmaceutical royalty aggregator. The company focuses on investing in differentiated late-stage assets and operating royalty-generating, infrastructure-light platform technologies. The company’s focus is to continue to expand its pipeline by aggregating royalty rights in mid- to late-stage development and commercial biopharma products
The company’s business model mitigates the high volatility and risk associated with building a business around a single or small number of assets. From a more tactical perspective, the company execute its strategy using four key approaches: royalty monetization, special situations, project finance, and IP technology platform investments. With royalty monetization, the company purchases rights on existing royalty contracts that are owned by inventors, academic institutions or companies.
Commercial and Clinical Stage Partnered Portfolio
The company has a large royalty portfolio including 12 major commercial-stage revenue-generating royalty assets and over 75 additional active programs with future revenue-generating potential, including over 85 that are programs that are fully funded by its partners.
Kyprolis (Amgen, Ono, BeiGene)
The company supplies Captisol to Amgen for use with Kyprolis (carfilzomib) and granted Amgen an exclusive product-specific license under its patent rights with respect to Captisol. Kyprolis is formulated with Ligand’s Captisol technology and is approved for the following:
In combination with dexamethasone, lenalidomide plus dexamethasone, daratumumab plus dexamethasone, or daratumumab and hyaluronidase-fihj and dexamethasone, or isatuximab and dexamethasone for the treatment of patients with relapsed or refractory multiple myeloma who have received one to three lines of therapy.
As a single agent for the treatment of patients with relapsed or refractory multiple myeloma who have received one or more lines of therapy.
Under this agreement, the company is entitled to receive revenue from clinical and commercial Captisol material sales. Amgen’s obligation to pay royalties does not expire until four years after the expiration of the last-to-expire patent covering Captisol. The company’s patents and applications relating to the Captisol component of Kyprolis are not expected to expire until 2033.
Qarziba (Recordati)
The company receives royalties on Qarziba (dinutuximab beta) sales through its acquisition of Apeiron Biologics AG (Apeiron), announced in July 2024.
Filspari (Travere, CSL Vifor, Renalys)
In early 2012, the company licensed the world-wide rights to Filspari (sparsentan) to Travere Therapeutics. In September 2024, Travere received full approval from the FDA for Filspari, which was previously under accelerated approval, for the treatment of immunoglobulin A nephropathy (IgAN). Filspari is the first and only dual endothelin and angiotensin II receptor antagonist in development for rare kidney diseases and is the first non-immunosuppressive treatment indicated for IgAN. In February 2024, Travere and its partner CSL Vifor received approval for Filspari for the treatment of IgAN in Europe. Additionally, Travere has partnered with Renalys Pharma to develop and commercialize Filspari in Japan and other Asian countries. Renalys completed enrollment in its registrational Phase III clinical trial of sparsentan for IgAN in January 2025.
In February 2025, Travere announced completion of its Type C meeting with the FDA and plans to submit a supplemental New Drug Application (sNDA) seeking traditional approval of Filspari for focal segmental glomerulosclerosis (FSGS). The sNDA will be based on existing data from the Phase 3 DUPLEX and Phase 2 DUET studies of Filspari and is expected to be submitted around the end of the first quarter of 2025.
Ohtuvayre (Verona, Nuance)
Ohtuvayre is a first-in-class selective dual inhibitor of the enzyme’s phosphodiesterase 3 and phosphodiesterase 4 (‘PDE3 and PDE4’) that combines bronchodilator and non-steroidal anti-inflammatory effects in one molecule. Ohtuvayre is delivered directly to the lungs through a standard jet nebulizer without the need for high inspiratory flow rates or complex hand-breath coordination. Ohtuvayre was approved by the FDA in June 2024 for the maintenance treatment of chronic obstructive pulmonary disease (‘COPD’) in adult patients. Ohtuvayre is the first inhaled product with a novel mechanism of action available for the maintenance treatment of COPD in adult patients in more than 20 years. Verona sublicensed the right to develop and commercialize Ohtuvayre in Hong Kong, Macau, Taiwan, and mainland China to Nuance Pharma.
Verona is conducting Phase 2 trials for indication expansion in non-cystic fibrosis bronchiectasis, as well as a fixed-dose combination of ensifentrine + Long-Acting Muscarinic Antagonist (LAMA) for maintenance treatment of COPD. In September 2024, Nuance Pharma (private), completed enrollment in its pivotal Phase 3 clinical trial evaluating Ohtuvayre for the maintenance treatment of COPD in China.
Rylaze (Jazz Pharmaceuticals)
In July 2021, Jazz announced the U.S. launch of Rylaze (asparaginase erwinia chrysanthemi (recombinant)-rywn), previously referred to as JZP458. Rylaze, which was approved by the FDA in June 2021, is a recombinant erwinia asparaginase used as a component of a multi-agent chemotherapeutic regimen for the treatment of acute lymphoblastic leukemia (ALL) and lymphoblastic lymphoma (LBL) in adult and pediatric patients one month or older who have developed hypersensitivity to E. coli-derived asparaginase. In September 2023, Jazz announced that the European Commission (EC) had granted marketing authorization for Rylaze, to be marketed as Enrylaze. Jazz began a rolling launch in the second half of 2023.
Vaxneuvance (Merck)
Vaxneuvance, a 15-valent pneumococcal conjugate vaccine, also known as V114, was approved in the U.S. in July of 2021 for the prevention of invasive disease caused by Streptococcus pneumoniae serotypes 1, 3, 4, 5, 6A, 6B, 7F, 9V, 14, 18C, 19A, 19F, 22F, 23F and 33F in adults 18 years of age and older, and subsequently in children 6 weeks through 17 years of age in June of 2022. Vaxneuvance was also approved in Europe in October 2022 for the prevention of invasive disease and pneumonia caused by Streptococcus pneumoniae in individuals 18 years and older and in infants, children and adolescents from 6 weeks to less than 18 years of age. Vaxneuvance utilizes CRM197 vaccine carrier protein, which is produced using the patent-protected Pelican Expression Technology platform.
Capvaxive (Merck)
Capvaxive, a 21 valent pneumococcal vaccine, also known as V116, was approved by the FDA in June 2024 for the prevention of invasive disease caused by Streptococcus pneumoniae serotypes 3, 6A, 7F, 8, 9N, 10A, 11A, 12F, 15A, 15C, 16F, 17F, 19A, 20A, 22F, 23A, 23B, 24F, 31, 33F, and 35B in adults 18 years of age and older. Capvaxive is the first pneumococcal conjugate vaccine specifically designed for adults, and its 21 covered serotypes account for approximately 85% of cases of invasive pneumococcal disease among individuals 50 and over, including 8 serotypes not covered by any approved vaccines. Capvaxive utilizes CRM197 vaccine carrier protein, which is produced using the patent-protected Pelican Expression Technology platform, which the company acquired in October 2020 through its acquisition of Pfenex.
Pneumosil (Serum Institute of India, SII)
SII began commercialization of its 10-valent pneumococcal conjugate vaccine, Pneumosil, which is produced using CRM197 made in the Pelican Expression Technology platform, in the second quarter of 2020. Pneumosil is designed primarily to help fight against pneumococcal pneumonia among children, with an advantage of targeting the most prevalent serotypes of the bacterium causing serious illness in developing countries. Pneumosil achieved WHO Prequalification in December 2019, allowing the product to be procured by United Nations agencies and Gavi, the Vaccine Alliance, and subsequently achieved Indian Marketing Authorization in July 2020, and SII announced commercial launch of the product in India in December 2020.
Teriparatide Injection Product (PF708) (Alvogen/Adalvo)
The company acquired the teriparatide injection product with the acquisition of Pfenex in October 2020. Teriparatide injection is a drug indicated for various uses including the treatment of osteoporosis in certain patients at high risk for fracture. Teriparatide injection was developed using the company’s Pelican Expression Technology and was approved by the FDA in 2019 in accordance with the 505(b)(2) regulatory pathway, with FORTEO as the reference product. The company’s commercialization partner, Alvogen, launched the product in June 2020 in the United States.
Alvogen has exclusively licensed the rights to commercialize and manufacture the teriparatide injection product in the U.S., while Adalvo has the rights to commercialize in the E.U. and other territories outside the U.S.
Evomela (Acrotech and CASI)
The company supplies Captisol to, and receive royalties from, Acrotech Biopharma for sales of Evomela in the United States, and CASI Pharmaceuticals for sales in China. Evomela is a Captisol-enabled melphalan IV formulation which is approved by the FDA for use in two indications:
a high-dose conditioning treatment prior to autologous stem cell transplantation (‘ASCT’) in patients with multiple myeloma; and
for the palliative treatment of patients with multiple myeloma for whom oral therapy is not appropriate.
The company’s patents and applications relating to the Captisol component of melphalan are not expected to expire until 2033. The company has entered into a settlement agreement with Teva and Acrotech Biopharma (the holder of the NDA for Evomela) which will allow Teva to market a generic version of Evomela in the United States in 2026, or earlier under certain circumstances. In December 2024, Acrotech issued a termination process letter to CASI alleging the Company materially breached the license agreement and failed to cure such breach, thus terminating the license agreement. CASI can continue to distribute Evomela in China for a reasonable wind down period not to exceed 24 months.
Nexterone (Baxter)
The company has a license agreement with Baxter, related to Nexterone, a Captisol-enabled formulation of amiodarone, which is marketed in the United States and Canada. The company supplies Captisol to Baxter for use in accordance with the terms of a license agreement and a separate supply agreement between it and Baxter.
Tzield (Sanofi)
The company acquired a royalty of less than 1% on net sales of Tzield through its acquisition of Tolerance Therapeutics (‘Tolerance’) in the fourth quarter of 2023. Tzield is the first disease-modifying therapy to be approved in type 1 diabetes (‘T1D’). It is a CD3-directed antibody indicated to delay the onset of Stage 3 T1D in adults and children aged 8 years and older with Stage 2 T1D. Tzield was granted Breakthrough Therapy Designation in 2019 and was approved by the FDA in November 2022.
Veklury (Gilead)
The company supplies Captisol to Gilead for sales of Veklury (remdesivir). Gilead received marketing approval from the FDA in October 2020. Veklury is an antiviral treatment for COVID-19. The product has regulatory approvals for the treatment of moderate or severe COVID-19 in over 70 countries. The company is supplying Captisol to Gilead under a 10-year supply agreement. The company is also supplying Captisol to Gilead’s voluntary licensing generic partners who are manufacturing remdesivir for 127 low- and middle-income countries. The company receives its commercial compensation for this program through the sale of Captisol.
Botensilimab and Balstilimab (BOT/BAL) (Agenus)
In May 2024, the company entered into the Agenus Agreement to support BOT/BAL clinical development, confirmatory Phase 3 trial, and launch readiness activities. Botensilimab is an investigational multifunctional anti-CTLA-4 immune activator (antibody) designed to boost both innate and adaptive anti-tumor immune responses. Its novel design leverages mechanisms of action to extend immunotherapy benefits to ‘cold’ tumors which generally respond poorly to standard of care or are refractory to conventional PD-1/CTLA-4 therapies and investigational therapies.
UGN-301
UGN-301 is an anti-CTLA-4 monoclonal antibody (zalifrelimab), prepared with reverse-thermal hydrogel for intravesical administration into the bladder. ntravesical administration of UGN-301 is designed to increase drug concentrations in the bladder without significant systemic exposure, potentially diminishing the systemic toxicity associated with CTLA-4 blockade. UroGen is evaluating UGN-301 as a monotherapy and as combination therapy for the intravesical treatment of high-grade non-muscle invasive bladder cancer.
TR-Beta - VK2809 and VK0214 (Viking)
The company’s partner, Viking, is developing VK2809, a novel selective thyroid hormone receptor beta (TR-beta) agonist with potential in multiple indications, including hypercholesterolemia, dyslipidemia and non-alcoholic steatohepatitis (NASH; also referred to as metabolic dysfunction associated steatohepatitis, MASH).
Ciforadenant – CPI-444 (Corvus)
The company’s partner, Corvus, is conducting a Phase 1b/2 clinical trial evaluating ciforadenant as a potential first line therapy for metastatic renal cell cancer (RCC) in combination with ipilimumab (anti-CTLA-4) and nivolumab (anti-PD-1). Under the terms of the company’s agreement with Corvus, it is entitled to development and regulatory milestones and tiered royalties on potential future sales.
Qtorin rapamycin (Palvella)
The company acquired economic rights to Qtorin 3.9% rapamycin anhydrous gel (Qtorin rapamycin, formerly PTX-022) from Palvella in December 2018. Qtorin rapamycin is a novel, topical formulation of high-strength rapamycin in development for the treatment of Microcystic Lymphatic Malformations (‘Microcystic LM’) and cutaneous venous malformations (‘VMs’).
Lasofoxifene (Sermonix)
The company’s partner, Sermonix has a license for the development of oral lasofoxifene, its lead investigational drug, for the United States and additional territories. Lasofoxifene is a selective estrogen receptor modulator in development for the treatment of breast cancer, discovered through the research collaboration between Pfizer and Ligand. Sermonix is conducting the Phase 3 ELAINE-3 clinical trial to assess the efficacy of lasofoxifene in combination with Eli Lilly and Company’s CDK4/6 inhibitor abemaciclib (Verzenio) compared to fulvestrant and abemaciclib in pre- and post-menopausal subjects with locally advanced or metastatic ER+/HER2- breast cancer with an ESR1 mutation.
Xinshumu (pradefovir) (Xi'an Xintong)
Xi'an Xintong received marketing approval from the Chinese National Medical Products Administration (NMPA) for Xinshumu (pradefovir mesylate tablets) in October 2024. Xinshumu is the first liver-targeted, novel Hepatitis B drug developed based on the HepDirect liver-targeting prodrug technology that utilizes a chemically modified prodrug of the active compound which is stable in blood and intestines and is activated by a liver specific CYP subtype enzyme to achieve high liver concentration of the active drug and low systemic exposure, reducing side effects in other organs for improved safety.
MB07133 (Xi'an Xintong)
Xi'an Xintong is also developing MB07133, a liver specific, HepDirect prodrug of cytarabine monophosphate, for the potential treatment of hepatocellular carcinoma and intrahepatic cholangiocarcinoma. MB07133 is in Phase 2 in China.
Patents and Proprietary Rights
Captisol
Patents and pending patent applications covering Captisol and methods of making Captisol are owned by the company. The company also owns several patents and pending patent applications covering drug products containing Captisol as a component. Globally, the company owns over 400 issued patents covering all of the foregoing Captisol compositions, methods and related technology.
Ten Captisol patents in several families are listed in the Orange Book in connection with one or more prescription drugs on the market. These Captisol-enabled drugs include Nexterone (Baxter), Kyprolis (Amgen), Noxafil (Merck), Evomela (Acrotech/CASI), Baxdela (Melinta) and Zulresso (Sage).
Kyprolis
Patents protecting Kyprolis include those owned by Amgen and those owned by the company.
Ligand UK Development Limited
Under the terms of the company’s sale of Vernalis (R&D) Limited to HitGen in December 2020, Ligand retained a portfolio of fully funded shots on goal, which include S65487, a Bcl-2 inhibitor, and S64315, an Mcl-1 inhibitor for the treatment of cancers, both of which are partnered with Servier in collaboration with Novartis. These programs and their IP are owned by Ligand UK Development Limited, which has a worldwide patent portfolio of approximately 100 granted patents in over 40 countries.
Novan
The company acquired a robust IP portfolio that consists of over 45 U.S. patents, 120 non-U.S. patents, and 25 pending patent applications worldwide along with substantial know-how and trade secrets. This IP portfolio provides material coverage for the company’s platform technologies, licensed products and product candidates, in addition to ZELSUVMI, which was approved by the FDA on January 5, 2024. Upon the initial approval of ZELSUVMI, the company applied for 1,280 days of patent term extension, or PTE, for the U.S. patent covering ZELSUVMI compositions.
Apeiron
In connection with the acquisition of Apeiron in July 2024, the company acquired a mature IP portfolio consists of more than 300 patents worldwide. This IP portfolio supports a number of licensed products and product candidates and consists of over 60 patents related to Qarziba.
Trademarks
Ligand, BEPro, Captisol, CyDex, LTP, LTP Technology, NITRICIL, and ZELSUVMI are protected under applicable intellectual property laws and are the company’s property. All other trademarks, trade names and service marks including, but not limited to Pelican Expression Technology, PeliCRM, Pfenex Expression Technology, OmniAb Kyprolis, Evomela, Veklury, Livogiva, Bonteo, Zulresso, Rylaze, Vaxneuvance, Pneumosil, Minnebro, Baxdela, Nexterone, Noxafil, Duavee, Filspari, Ohtuvayre, Qarziba and are the property of their respective owners.
History
Ligand Pharmaceuticals Incorporated was founded in 1987. The company was incorporated in 1987.
