We are dedicated to developing novel therapies and solutions for a wide variety of patients in need. Our pipeline of 20+ programs developed across our partner companies spans several large-market therapeutic areas and includes multiple late-stage product candidates.
47% Journey Medical Corporation
Acne
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Fungal Infections
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Wounds
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Recalcitrant Nodular Acne
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47% Journey Medical Corporation
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CONTRAINDICATIONS AND WARNINGS
Accutane® must not be used by patients who are or may become pregnant. There is an extremely high risk that severe birth defects will result if pregnancy occurs while taking Accutane in any amount, even for short periods of time. Potentially any fetus exposed during pregnancy can be affected. There are no accurate means of determining whether an exposed fetus has been affected.
Rosacea
Rosacea is a chronic, relapsing, diverse skin condition that most commonly presents with symptoms such as deep facial redness, spider veins (telangiectasia) and acne-like inflammatory lesions (papules and pustules). Based on research in the British Journal of Dermatology, it is estimated that 415 million people suffer with rosacea worldwide.1 Rosacea is most frequently seen in adults between 30 and 50 years of age. Surveys conducted by The National Rosacea Society report more than 90 percent of rosacea patients said their condition had lowered their self-confidence and self-esteem, and 41 percent reported that it had caused them to avoid public contact or cancel social engagements. Among rosacea patients with severe symptoms, 88 percent said the disorder had adversely affected their professional interactions, and 51 percent said they had missed work because of their condition.
1. Gether L, Overgaard LK, Egeberg A, Thyssen JP. Incidence and prevalence of rosacea: a systematic review and meta-analysis. Br J Dermatol 2018 Feb 25. doi: 10.1111/bjd.16481
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Cosibelimab is a fully-human monoclonal antibody of IgG1 subtype that directly binds to programmed death ligand-1 (PD-L1) and blocks the PD-L1 interaction with the programmed death receptor-1 (PD-1) and B7.1 receptors.
View Clinical TrialRecurrent or Metastatic Cancers
Cutaneous squamous cell carcinoma (cSCC)
Cutaneous squamous cell carcinoma (cSCC) is the second most common type of skin cancer in the United States. cSCC starts in squamous cells within the surface of the skin, and upon progression, can invade deeper layers of the skin or spread to lymph nodes or other parts of the body. Surgery is the first option for patients with cSCC. In advanced stages, some patients will be treated with radiation or systemic drug therapy; however, certain patients may not be able to be cured with surgery or radiation. It is estimated that 7,000 patients in the United States die each year from cSCC.
10% Checkpoint; 4.5% Royalty | 2.5% Equity Dividend
CUTX-101 is a copper histidinate injection for the treatment of Menkes disease.
View Clinical Trial View Clinical TrialMenkes Disease
Menkes disease is an X-linked genetic disorder of copper transport caused by mutations of the ATP7A gene. Copper is an essential trace element required for human development and health. In Menkes disease, patients are born without the ability to absorb copper from diet, leading to severe developmental delays and significant neurological symptoms. Without treatment, Menkes disease could lead to death by three years of age. There is currently no FDA-approved therapy for the treatment of Menkes disease.
75% of Cyprium; 3% - 12.5% royalty payable to Cyprium from partner Sentynl3 | 2.5% Annual Equity Dividend
IV Tramadol is intended for the management of moderate to moderately severe postoperative pain.
View Clinical Trial View Clinical Trial View Clinical TrialPost-Operative Acute Pain Management
Many patients who undergo surgical procedures experience severe postoperative pain. IV Tramadol is being developed as a potential treatment for pain management following surgery, including abdominoplasty and bunionectomy.
Abdominoplasty, or more commonly referred to as a “tummy tuck,” is a cosmetic surgical procedure in which excess skin and fat is removed from the abdomen, resulting in a smoother and firmer abdomen. A bunionectomy is a surgical procedure to remove a bunion from a patient’s foot. Depending on the complexity of these surgeries, some patients will experience severe postoperative pain.
6% Avenue; 4.5% Royalty | 2.5% Equity Dividend
Olafertinib is a small-molecule, targeted anti-cancer agent for the treatment of patients with epidermal growth factor receptor (EGFR) mutation-positive non-small cell lung cancer (NSCLC).
View Clinical TrialFrontline NSCLC with EGFR Mutations
Lung cancer is the leading cause of cancer death among men and women. There are two main types of lung cancer: non-small cell lung cancer (NSCLC) and small cell lung cancer (SCLC). The American Cancer Society estimates that roughly 80% to 85% of lung cancers are NSCLC. Approximately 20% of newly diagnosed NSCLC patients have activating mutations in EGFR, which can be selectively targeted with an EGFR inhibitor.
10% Checkpoint; 4.5% Royalty | 2.5% Equity Dividend
CAEL-101 is a first-in-class anti-amyloid antibody designed to improve organ function by reducing or eliminating amyloid deposits in patients with amyloid light chain (AL) amyloidosis.
View Clinical Trial View Clinical Trial View Clinical TrialAL Amyloidosis
AL amyloidosis is a rare systemic disorder caused by an abnormality of plasma cells in the bone marrow. Misfolded amyloid proteins produced by plasma cells cause buildup in and around tissues, nerves and organs, gradually affecting their function. This can cause progressive and widespread organ damage and high mortality rates.
42% of future proceeds to Caelum from AstraZeneca
Triplex is a universal, multi-antigen T-cell immunotherapeutic vaccine for controlling cytomegalovirus (CMV) in stem cell and solid organ transplant recipients.
View Clinical Trial View Clinical Trial View Clinical TrialCytomegalovirus (CMV)
CMV is a common virus that is typically asymptomatic in healthy individuals but can cause life-threatening disease in those with weakened or uneducated immune systems. Patients undergoing allogeneic stem cell and solid organ transplantation are at particularly high risk of experiencing complications associated with CMV.
CMV can be transmitted to seronegative transplant recipients through infected donor grafts or reactivated in seropositive transplant recipients (particularly those with low baseline immunity) as a result of their immune-suppressed condition. In either instance, CMV can cause severe and life-threatening complications, including pneumonia, gastroenteritis and retinitis.
83% Helocyte; 4.5% Royalty | 2.5% Equity Dividend
MB-106 is a CD20-specific chimeric antigen receptor (CAR) T Program for B-cell non-Hodgkin lymphoma (NHL) and chronic lymphocytic leukemia (CLL).
View Clinical Trial View Clinical TrialB-cell Non-Hodgkin Lymphoma and Chronic Lymphocytic Leukemia (CLL)
There are several forms of NHL, including follicular lymphoma, mantle cell lymphoma, marginal zone lymphoma, lymphoplasmacytic lymphoma and small lymphocytic lymphoma, which account collectively for ~45% of all cases of NHL. Most types of NHL are incurable with available therapies, except for allogenic hematopoietic stem cell transplant (allo-SCT). More than 70,000 new cases of B-cell NHL are diagnosed each year in the United States, and more than 19,000 patients die annually due to this group of diseases.
8% Mustang; 4.5% Royalty | 2.5% Equity Dividend
Dotinurad is being developed as a urate lowering therapy for the treatment of gout.
Gout and Chronic Kidney Disease
Gout is a serious, progressive and debilitating inflammatory arthritis caused by deposits of uric acid crystal in and around the connective tissue of joints, tendons and the kidneys. There are nearly 20 million diagnosed patients with gout in the US, Europe and Canada as of 2021, and it is estimated that two to three million U.S. patients are unsatisfied with their urate-lowering therapy and their serum uric acid levels remain inadequately controlled.
68% of Urica; 4.5% Royalty | 2.5% Equity Dividend
AJ201 was designed to modify spinal and bulbar muscular atrophy (“SBMA”), also known as Kennedy's Disease, through multiple mechanisms including degradation of the abnormal AR protein and by stimulating Nrf1 and Nrf2, which are involved in protecting cells from oxidative stress which can lead to cell death. AJ201 completed a Phase 1 clinical trial in 2021, which demonstrated the safety of the molecule. It is currently being studied in a Phase 1b/2a multicenter, randomized, double-blind clinical trial in six clinical sites across the U.S., and screening of patients has begun. This first-in-human study aims to evaluate the safety and clinical response of AJ201 in patients suffering from SBMA. AJ201 has been granted Orphan Drug Designation (“ODD”) by the U.S. Food and Drug Administration for the indications of Kennedy’s Disease, Huntington’s Disease and Spinocerebellar Ataxia.
Spinal and Bulbar Muscular Atrophy (SBMA)
Spinal and bulbar muscular atrophy (“SBMA”), also known as Kennedy's Disease, is a rare, inherited, X-linked genetic neuromuscular disease primarily affecting men. The condition is caused by a polyglutamine expansion in the androgen receptor (“AR”) which leads to production of an abnormal AR protein that forms aggregates responsible for muscle atrophy focused in the spinal-bulbar region of the body. The weakening of the bulbar muscles affects chewing, speech and swallowing, with patients prone to choking or inhaling foods or liquids, resulting in airway infection. SBMA also affects muscles in the limbs, leading to difficulty walking and injury caused by falling. Although there is a range of cited prevalence rates in the literature, a recent study used genetic analysis to estimate disease prevalence of 1:6,887 males[i]. Currently, there is no effective treatment for SBMA.
6% Avenue; 4.5% Royalty | 2.5% Equity Dividend
MB-101 is a chimeric antigen receptor (CAR) T therapy for glioblastoma (GBM).
View Clinical Trial View Clinical TrialRecurrent Glioblastoma (GBM)
GBM is the most common brain and central nervous system (CNS) cancer, accounting for 45.2% of malignant primary brain and CNS tumors, 54% of all gliomas, and 16% of all primary brain and CNS tumors. There were an estimated 12,390 new GBM cases predicted in 2017 in the U.S. Malignant brain tumors are the most common cause of cancer-related deaths in adolescents and young adults aged 15-39 and the most common cancer occurring among 15-19-year-olds in the U.S. Although GBM is a rare disease (2-3 cases per 100,000 persons per year in the US and EU), it is quite lethal, with five-year survival rates historically under 10%. Standard of care therapy consists of maximal surgical resection, radiation and chemotherapy with temozolomide, which, while rarely curative, is shown to extend median overall survival from 4.5 to 15 months. GBM remains difficult to treat due to the inherent resistance of the tumor to conventional therapies.
8% Mustang; 4.5% Royalty | 2.5% Equity Dividend
MB-108 is a next-generation oncolytic virus (C134) that can replicate in tumor cells, but not in normal cells. Replication of MB-108 in the tumor itself not only kills the infected tumor cells but also causes the tumor cell to act as a factory to produce new virus.
View Clinical TrialRecurrent Glioblastoma (GBM)
GBM is the most common brain and central nervous system (CNS) cancer, accounting for 45.2% of malignant primary brain and CNS tumors, 54% of all gliomas and 16% of all primary brain and CNS tumors. There were an estimated 12,390 new GBM cases predicted in 2017 in the U.S. Malignant brain tumors are the most common cause of cancer-related deaths in adolescents and young adults aged 15-39 and the most common cancer occurring among 15-19-year-olds in the U.S. Although GBM is a rare disease (2-3 cases per 100,000 persons per year in the US and EU), it is quite lethal, with five-year survival rates historically under 10%. Standard of care therapy consists of maximal surgical resection, radiation and chemotherapy with temozolomide, which, while rarely curative, is shown to extend median overall survival from 4.5 to 15 months. GBM remains difficult to treat due to the inherent resistance of the tumor to conventional therapies.
8% Mustang; 4.5% Royalty | 2.5% Equity Dividend
BAER-101 is a novel α2/3–subtype-selective GABA A positive allosteric modulator (“PAM”).
CNS Disorders
Central nervous system diseases, also known as central nervous system disorders, are a group of neurological disorders that affect the structure or function of the brain or spinal cord, which collectively form the central nervous system (CNS).
6% Avenue; 4.5% Royalty | 2.5% Equity Dividend
MB-109 combines MB-101 (IL13Rα2-targeted CAR-T cells) CAR T cell therapy with MB-108 (C134 oncolytic virus). The combination is designed to leverage MB-108 to make cold glioblastoma tumors “hot,” and thereby may improve the efficacy of MB-101 CAR-T cell therapy. Clinical data from separate trials, together with results from the in vivo combination studies currently underway, will support the first ever industry-sponsored trial of an oncolytic virus with a CAR-T for the treatment of cancer patients.
Recurrent GBM and Anaplastic Astrocytoma
8% Mustang 4.5% Royalty 2.5% Equity Dividend
AAV.sFH is an adeno-associated virus (AAV) gene therapy that restores lasting production of regulatory proteins, potentially providing a curative treatment for diseases with high unmet need.
Complement-Mediated Diseases
Asset sold to 4DMT in April 2023 ~$140M in potential future milestone proceeds plus royalties4
Adeno-associated virus (AAV)-based ATP7A is a gene therapy intended for use in combination with CUTX-101 for the treatment of Menkes disease and related copper transport disorders.
Menkes Disease
Menkes disease is an X-linked genetic disorder of copper transport caused by mutations of the ATP7A gene. Copper is an essential trace element required for human development and health. In Menkes disease, patients are born without the ability to absorb copper from diet, leading to severe developmental delays and significant neurological symptoms. Without treatment, Menkes disease could lead to death by three years of age. Currently, there is no FDA-approved therapy for treatment of Menkes disease.
75% of Cyprium; 2.5% Annual Equity Dividend
CK-103 is a BET inhibitor that is designed to inhibit BRD4, a member of the BET domain for cancer treatment.
Multiple Solid Tumors
10% Checkpoint; 4.5% Royalty | 2.5% Equity Dividend
CEVA-102 is the first cell product produced by CEVA-D and is being developed for the treatment of severe traumatic brain injury (TBI) in adults and children.
Traumatic Brain Injury (TBI)
TBIs are associated with 33% of all trauma-related deaths. There are no effective therapies to treat secondary brain injury and the post-injury response of central nervous system apoptosis and neuroinflammation.
79% of Cellvation; 4.5% Royalty | 2.5% Equity Dividend
CK-302, an anti-GITR monoclonal antibody, is being developed as a novel treatment for patients with multiple solid tumors.
Multiple Solid Tumors
10% Checkpoint; 4.5% Royalty | 2.5% Equity Dividend
CK-303, an anti-CAIX monoclonal antibody, is being developed as a treatment for multiple solid tumors in patients.
Multiple Solid Tumors
10% Checkpoint; 4.5% Royalty | 2.5% Equity Dividend
CEVA-D is a novel bioreactor device that enhances the anti-inflammatory potency of bone marrow-derived cells without genetic manipulation, using wall shear stress (WSS) to suppress tumor necrosis factor-a (TNF-a) production by activated immune cells.
Traumatic Brain Injury (TBI)
TBIs are associated with 33% of all trauma-related deaths. There are no effective therapies to treat secondary brain injury and the post-injury response of central nervous system apoptosis and neuroinflammation.
79% Cellvation; 4.5% Royalty | 2.5% Equity Dividend
We have established relationships with some of the world's leading academic research institutions and biopharmaceutical companies.
In January 2019, Caelum Biosciences, Inc., a Fortress Biotech partner company, signed an agreement with Alexion Pharmaceuticals, Inc. to advance the development of CAEL-101 for light chain (AL) amyloidosis. The collaboration leverages Alexion’s expertise in rare disease antibody development and commercial franchise in hematology.
AstraZeneca is a global, science-led biopharmaceutical company that focuses on the discovery, development and commercialization of prescription medicines used by millions of patients worldwide. In December 2019, we entered into an exclusive worldwide licensing agreement with AstraZeneca for BAER-101 (formerly known as AZD7325), a novel α2/3–subtype-selective GABA A positive allosteric modulator (“PAM”). BAER-101 is currently in development at our partner company, Baergic Bio, Inc., a biopharmaceutical company focused on the development and commercialization of novel therapeutics for the treatment of central nervous system (“CNS”) disorders.
City of Hope is a world-renowned independent cancer research and treatment center. Our partner company Mustang Bio, Inc. entered into exclusive, worldwide licensing agreements with City of Hope on patents covering five novel CAR T cell therapies in development for multiple forms of cancer: MB-101 (IL-13Rα2 CAR T), MB-102 (CD123 CAR T), MB-103 (HER2 CAR T), MB-104 (CS1 CAR T) and MB-105 (PSCA CAR T). These CAR T cell therapies are currently in clinical development at City of Hope and supported by Mustang Bio, Inc. Another partner company, Helocyte, Inc., entered into exclusive, worldwide licensing agreements with City of Hope for the use of Triplex and ConVax, two novel immunotherapies to prevent and control cytomegalovirus (CMV).
In September 2017, we entered into an exclusive, worldwide licensing agreement with Fred Hutchinson Cancer Research Center for the use of MB-106, a CAR T therapy related to autologous T cells engineered to express a CD20-specific chimeric antigen receptor (CD20 Technology). MB-106 is currently in development at our partner company, Mustang Bio, Inc.
In February 2019, we entered into an exclusive worldwide license agreement with Nationwide Children’s Hospital to develop MB-108, an oncolytic virus (C134) for the treatment of glioblastoma multiforme. MB-108 is currently in development at our partner company, Mustang Bio, Inc.
In November 2016, we entered into an exclusive worldwide license agreement with The University of Texas Health Science Center at Houston (UTHealth) to secure rights to three programs for the treatment of traumatic brain injury (TBI), including two Phase 2 cell therapies (CEVA-101 pediatric and adult), CEVA-102 and a next-generation bioreactor called CEVA-D that enhances the anti-inflammatory potency of bone marrow‐derived cells without genetic manipulation. CEVA-101 is currently in development at our partner company, Cellvation, Inc.
Cincinnati Children’s is one of the oldest and most distinguished pediatric hospitals in the United States. In December 2019, we entered into an agreement with Cincinnati Children’s to advance clinical development of BAER-101, a novel α2/3–subtype-selective GABA A positive allosteric modulator (“PAM”), in select central nervous system (“CNS”) disorders. BAER-101 is currently in development at our partner company, Baergic Bio, Inc., a biopharmaceutical company focused on the development and commercialization of novel therapeutics for the treatment of CNS disorders.
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