Pipeline

Our Branded Development Pipeline:

A diversified, durable, high-value portfolio of medicines

FILTER BY:
INDICATIONS UNDER STUDY    
  • INDICATIONS UNDER STUDY
  • ACUTE GRAFT VERSUS HOST DISEASE (US)
  • ACUTE PANCREATITUS
  • AMYOTROPHIC LATERAL SCLEROSIS
  • CHRONIC GRAFT VERSUS HOST DISEASE (JP)
  • CHRONIC NON-HEALING WOUNDS
  • DIABETIC FOOT ULCER
  • DUCHENNE MUSCULAR DYSTROPHY
  • HEPATIC ENCEPHALOPATHY
  • HEPATORENAL SYNDROME TYPE–1
  • NEONATAL HYPERBILIRUBINEMIA
  • POST-CARDIAC ARREST
  • SEVERE BURNS, DEEP PARTIAL THICKNESS
  • SEVERE BURNS, FULL THICKNESS
  • SKIN CANCER RECURRENCE
  • TRANSPLANT ORGAN PERFUSATE
  • NIEMANN-PICK DISEASE TYPE C
  • FAMILIAL ADENOMATOUS POLYPOSIS
All phases    
  • All phases
  • PRECLINICAL
  • PHASE 1
  • PHASE 2
  • PHASE 3
  • REGISTRATION

The safety and efficacy of these products for the indication under study have not yet been established by the relevant regulatory authorities.

 
 
 
 
 
 
therapy
preclinical
phase 1
phase 2
phase 3
registration
INDICATIONS
UNDER STUDY
           
  STANNSOPORFIN (HEME OXYGENASE INHIBITOR)
INDICATIONS UNDER STUDY: neonatal
hyperbilirubinemia
 

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Neonatal jaundice is a common condition in neonates that is associated with the yellowing of the skin and the whites of the eyes, typically in the first few days after birth. In most cases, it does not require treatment; however, elevated bilirubin levels in the blood (hyperbilirubinemia) can be toxic and lead to neurologic complications, including encephalopathy or irreversible brain damage. Currently, phototherapy is used to help the body clear bilirubin and bring down levels.

How this therapy could help

Stannsoporfin is a heme oxygenase inhibitor under investigation for its potential to reduce the production of bilirubin in infants at risk for severe neonatal jaundice. The safety and effectiveness of stannsoporfin have not yet been established by the FDA.

  UVADEX®
(METHOXSALEN) STERILE SOLUTION (THERAKOS)
INDICATIONS UNDER STUDY: chronic gvhd (jp)
 

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Graft versus Host Disease (GvHD) is a rare disorder that strikes bone marrow transplant or blood transfusion recipients whose immune system is deficient or suppressed. It can manifest as a skin rash, intestinal problems and liver dysfunction. It can be acute (sudden) or chronic (long lasting). Chronic GvHD lasts beyond 100 days and usually persists long after a bone marrow transplant.

How this therapy could help

Approximately 60% of bone marrow transplant patients develop GvHD, which is associated with high morbidity and mortality. Treatment with Extracorporeal Photopheresis for chronic GvHD is being studied in Japan.

  VTS-270 (2-HYDROXYPROPYL-B-CYCLODEXTRIN (HPßCD MIXTURE))
INDICATIONS UNDER STUDY: Niemann-Pick Disease Type C
 

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Niemann-Pick Type C (NPC) is a rare, neurodegenerative, and ultimately fatal disease that can present at any age. NPC is caused by mutations in either the NPC1 or NPC2 genes, resulting in the disruption of the trafficking of intracellular cholesterol, leading to intracellular lipid accumulation in various tissues, including the brain, liver, and spleen. Manifestations of the genetic disorder typically occur in childhood, with occasional late onset, and average diagnosis at ten years of age. NPC is usually fatal, and the majority of cases lead to death before age 20.

How this therapy could help

Results of a previous VTS-270 study showed the potential for a disease-modifying effect based on slowing of progression on neurological, disease-specific outcomes measures and promising clinical improvements in patients with NPC. VTS-270 is being studied further to assess safety and efficacy in patients with NPC.

  CPP-1X/SULINDAC ORAL COMBINATION
INDICATIONS UNDER STUDY: Familial Adenomatous Polyposis
 

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Familial Adenomatous Polyposis (FAP) results from a genetic mutation leading to uncontrolled growth of hundreds to thousands of polyps in the lower digestive tract. Left untreated, there is almost a 100% lifetime risk of developing colorectal cancer. The disease typically progresses without clear warning signs until reaching advanced stages. It can also lead to abnormal manifestations in other organs including bone, skin, retina, teeth and other malignant lesions.

How this therapy could help

Prior studies of CPP-1X/sulindac in in high-risk polyp formers demonstrated the potential the product may have in patients with FAP through targeting the underlying disease mechanism, preventing polyp growth and delaying disease progression. CPP-1X/sulindac is being studied further to assess safety and efficacy in patients with FAP.

  xenon (gas for inhalation)
INDICATIONS UNDER STUDY: post-cardiac
arrest
 

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A person experiencing cardiac arrest can die within minutes. Approximately 35-40% of patients are revived. Despite a return of heartbeat and blood circulation only 30-35% of resuscitated patients survive to hospital discharge. Survivors may have significant impairment of brain function and/or suffer coma. There are no pharmacological treatments approved to protect against brain injury in patients resuscitated after cardiac arrest.

How this therapy could help

Xenon is a noble gas that has been evaluated as an inhaled therapy in several studies. Its mechanism of action may contribute to reducing cell death in the brain, a cause of disability and death in patients resuscitated after cardiac arrest.

  terlipressin (VASOPRESSIN ANALOG)
INDICATIONS UNDER STUDY: hrs-1
 

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Hepatorenal Syndrome Type 1 (HRS-1) is a life-threatening condition that consists of rapid deterioration in kidney function in individuals with cirrhosis or liver failure. HRS-1 affects from 10,000 to as many as 30,000 patients in the U.S. This condition can lead to multi-organ failure including acute kidney failure, and is often fatal unless a liver transplant is performed or the condition is reversed. There is no FDA-approved pharmacological therapy for treatment of HRS-1 in the U.S.

How this therapy could help

Reversal of HRS has been shown to increase survival and improve clinical outcomes. Terlipressin is the most widely studied and clinically accepted pharmacological therapy globally for patients with HRS-1.

  stratagraft® (regenerative skin tissue)
INDICATIONS UNDER STUDY: severe burns, dpt
 

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Partial thickness burns (also known as second degree burns) affect the top two layers of skin. These burns can change over time and either heal or worsen into a full thickness (or third degree burn) after initial treatment. Partial thickness burns are serious and can become infected or be complicated by non-healing or abnormal scarring. The current standard of care for deep burns (partial or full thickness) includes skin-grafting (or autografting), which is the surgical harvest of healthy skin from an uninjured site on the patient’s body followed by transplant to the wound. It results in a donor site wound that requires medical management of pain, and may result in infection and/or scarring.

How this therapy could help

Results from a previous study in deep partial thickness burns demonstrated that StrataGraft skin tissue closed wounds and reduced the need for autografting. StrataGraft skin tissue is being studied as an alternative to autografting.

  uvadex® (METHOXSALEN) STERILE SOLUTION (THERAKOS)
INDICATIONS UNDER STUDY: acute gvhd (us)
 

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Graft versus Host Disease (GvHD) is a rare disorder that can strike bone marrow transplant or blood transfusion recipients whose immune system is deficient or suppressed. It can manifest as a skin rash, intestinal problems and liver dysfunction. It can be acute (sudden) or chronic (long lasting). Acute GvHD occurs in the first 100 days following bone marrow transplantation.

How this therapy could help

Multiple second-line treatments have been studied for management of unresponsive acute GvHD. Extracorporeal Photopheresis treatment for acute steroid-refractory GvHD is being studied in pediatric patients in the U.S.

  MNK-6105 (ORNITHINE PHENYLACETATE) INTRAVENOUS
INDICATIONS UNDER STUDY: HEPATIC ENCEPHALOPATHY
 

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Hepatic encephalopathy is a critical neurocognitive complication of chronic liver disease. It is associated with elevated circulating ammonia levels stemming from end stage liver disease (cirrhosis) and is generally reversible. Symptoms range from mildly altered mental status to coma or death. Once a patient suffers an acute hepatic encephalopathy episode, prevention of recurrence is critical and may require chronic therapy.

How this therapy could help

A goal of hepatic encephalopathy treatment is to lower blood ammonia levels. MNK-6105 has a novel mechanism of action that may help in eliminating excess ammonia in the bloodstream by excretion through the kidneys. An intravenous form of MNK-6105 is being studied in hospitalized patients with acute hepatic encephalopathy.

  stratagraft® (regenerative skin tissue)
INDICATIONS UNDER STUDY: severe burns, ft
 

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Full thickness burns (also known as third degree burns) affect all layers of the skin, and may cause damage down to underlying muscles. These burns carry severe risk for complications, such as infections, blood loss and shock, which could lead to organ failure or death. The current standard of care for severe burns is skin-grafting, or autografting, which is the surgical harvest of healthy skin from an uninjured site on the patient’s body followed by transplant to the wound. It results in a donor site wound that requires medical management of pain, and may result in infection and/or scarring.

How this therapy could help

A previous study in full thickness skin defects showed that StrataGraft skin tissue remained intact and viable throughout the seven-day placement period when it was used as a temporary cover to prepare the wound for skin-grafting. StrataGraft skin tissue is being studied further as a way to reduce the amount of autografting required in these patients.

  h.p. acthar® gel (repository corticotropin injection)
INDICATIONS UNDER STUDY: als
 

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Amyotrophic lateral sclerosis (ALS, often referred to as Lou Gehrig’s disease) is a progressive neurodegenerative disease that affects motor neuron cells in the brain and the spinal cord. Motor neurons reach from the brain and the spinal cord to the muscles throughout the body. The progressive degeneration of the motor neurons in ALS eventually leads to their demise and when the motor neurons die, voluntary and involuntary muscle movement is lost. With the progressive loss of motor neurons, people with ALS may lose the ability to speak, eat, move and breathe. There is increasing evidence that neuro-inflammation accompanies the death of motor neurons in ALS. Several inflammatory events that appear to accompany disease progression in ALS might be amenable to pharmacologic interventions as a component of disease management, and research in the field is investigating new approaches to implement an anti-inflammatory strategy for treating ALS.

How this therapy could help

H.P. Acthar Gel is being studied to assess its efficacy and safety as a treatment for ALS.

  MNK-6106 (ORNITHINE PHENYLACETATE) ORAL
INDICATIONS UNDER STUDY: HEPATIC ENCEPHALOPATHY
 

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Hepatic encephalopathy is a critical neurocognitive complication of chronic liver disease. It is associated with elevated circulating ammonia levels stemming from end stage liver disease (cirrhosis) and is generally reversible. Symptoms range from mildly altered mental status to coma or death. Once a patient suffers an acute hepatic encephalopathy episode, prevention of recurrence is critical and may require chronic therapy.

How this therapy could help

A goal of hepatic encephalopathy treatment is to lower blood ammonia levels. MNK-6105 has a novel mechanism of action that may help to eliminate excess ammonia in the bloodstream by excretion through the kidneys. An oral formulation of MNK-6105 is being developed, and will be studied in prevention of recurrence in patients with chronic hepatic encephalopathy.

  mnk-1411 (cosyntropin injection)
INDICATIONS UNDER STUDY: dmd
 

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Duchenne muscular dystrophy (DMD) is a rare genetic disorder characterized by weakness and wasting of the muscles. The disease is progressive and most affected individuals require a wheelchair by the teenage years. Serious life-threatening complications ultimately develop including breathing difficulties and disease of the heart muscle leading to premature death.

How this therapy could help

It is hypothesized that MNK-1411 may delay DMD progression by activating melanocortin receptors to reduce inflammation and diminish muscle damage. MNK-1411 is being evaluated for its potential effect on motor function and muscle strength.

  expressgraft
C9T1 skin tissue
INDICATIONS UNDER STUDY: DFU
 

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Diabetic foot ulcers (DFU) are open sores or wounds that occur on the bottom of the foot in approximately 15% of patients with diabetes and are the cause of more hospitalizations than any other complication of diabetes. Of those who develop a foot ulcer, approximately 6% will be hospitalized due to infection or other ulcer-related complication.

How this therapy could help

ExpressGraft (C9T1 skin tissue) is the first genetically engineered human skin substitute in development that expresses therapeutic proteins and may help reduce infection and accelerate wound healing. ExpressGraft (C9T1 skin tissue) has been shown in preclinical studies to augment one of the skin’s native antimicrobial defense mechanisms. It is being further evaluated for its potential to promote the healing of chronic wounds, including diabetic foot ulcers.

  inomax® (nitric oxide)
gas, for perfusion
INDICATIONS UNDER STUDY: transplant organ
perfusate
 

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A shortage of donor organs is one of the greatest challenges facing the field of organ transplantation. There is a high rate of rejection of donated organs, especially lungs and hearts, most of which do not meet the criteria for transplantation. There continues to be a high unmet need for increased organ supply worldwide.

How this therapy could help

Nitric oxide interrupts critical signaling pathways (e.g., immune response/inflammation) and may improve the viability of organ health. Nitric oxide is being studied as an addition to the organ perfusion procedure to evaluate organ viability prior to transplant and clinical outcomes post-transplantation.

  EXPRESSGRAFT
Pro-Angiogenic (VEGF)
INDICATIONS UNDER STUDY: TBD - CHRONIC NON-HEALING WOUNDS
 

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ExpressGraft (pro-angiogenic VEGF) is the first genetically engineered human skin substitute in development that expresses therapeutic proteins and may help reduce infection and accelerate wound healing. The pro-angiogenic properties of ExpressGraft provide sustained expression of human vascular endothelial growth factors (VEGF) that help to promote neovascularization and tissue regeneration within complex skin defects while providing a barrier function.

How this therapy could help

Potential applications include chronic wounds such as venous leg and/or diabetic foot ulcers, as well as acute wounds such as traumatic wounds that may require stimulation for neovascularization.

  EXPRESSGRAFT
Anti-Tumor (IL-12)
INDICATIONS UNDER STUDY: TBD - SKIN CANCER RECURRENCE
 

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ExpressGraft (IL-12) is the first genetically engineered human skin substitute in development that expresses therapeutic proteins and may help reduce infection and accelerate wound healing based on preclinical research. The anti-tumor properties of ExpressGraft (anti-tumor IL-12) provide sustained expression of human IL-12 and may suppress the growth of human skin cancer and secrete wound healing factors that may stimulate tissue regeneration.

How this therapy could help

A potential application may be following a surgical procedure for removal of a localized skin cancer.

  MP-3964
(TOLL-LIKE RECEPTOR-9 (TLR-9) ANTAGONIST)
INDICATIONS UNDER STUDY: transplant organ
perfusate & AP
 

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Acute pancreatitus is an inflammatory condition of the pancreas that is painful and at times fatal. The mortality rate of acute pancreatitis is about 10%. Sufferers often endure pain and malnutrition, and may be at higher risk of pancreatic cancer.

How this therapy could help

MP-3964, a TLR-9 antagonist, reduced serum markers of tissue injury, and reduced tissue inflammation and damage in both liver and pancreatic injury preclinical models. Further preclinical studies are ongoing to support a potential Investigational New Drug filing.

ALS: Amyotrophic Lateral Sclerosis, AP: Acute Pancreatitus, DFU: Diabetic Foot Ulcer, DMD: Duchenne Muscular Dystrophy, DPT: Deep Partial Thickness, FT: Full Thickness, GvHD: Graft vs Host Disease, HRS: Hepatorenal Syndrome, JP: Japan, VEGF: Vascular Endothelial Growth Factor