Leadiant Biosciences

Leadiant Biosciences

MD - Rockville
Biotechnology
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Focus: Orphan Disease Treatments

Leadiant Biosciences is a life sciences company focused on Orphan Disease Treatments.

Rare Diseases
Funding Stage
PUBLIC
Open Jobs
0

Products & Portfolio (5)

4 discontinued products not shown

CARNITOR
levocarnitine
LOE Approaching
INJECTION · INJECTABLE
CLINICAL PHARMACOLOGY CARNITOR (levocarnitine) is a naturally occurring substance required in mammalian energy metabolism. It has been shown to facilitate long-chain fatty acid entry into cellular mitochondria, thereby delivering substrate for oxidation and subsequent energy production. Fatty acids are utilized as an energy substrate in all tissues except the brain. In skeletal and cardiac muscle, fatty acids are the main substrate for energy production. Primary systemic carnitine deficiency is characterized by low concentrations of levocarnitine in plasma, RBC, and/or tissues. It has not been possible to determine which symptoms are due to carnitine deficiency and which are due to an underlying organic acidemia, as symptoms of both abnormalities may be expected to improve with CARNITOR . The literature reports that carnitine can promote the excretion of excess organic or fatty acids in patients with defects in fatty acid metabolism and/or specific organic acidopathies that bioaccumulate acylCoA esters. Secondary carnitine deficiency can be a consequence of inborn errors of metabolism or iatrogenic factors such as hemodialysis. CARNITOR may alleviate the metabolic abnormalities of patients with inborn errors that result in accumulation of toxic organic acids. Conditions for which this effect has been demonstrated are: glutaric aciduria II, methyl malonic aciduria, propionic acidemia, and medium chain fatty acylCoA dehydrogenase deficiency. Autointoxication occurs in these patients due to the accumulation of acylCoA compounds that disrupt intermediary metabolism. The subsequent hydrolysis of the acylCoA compound to its free acid results in acidosis which can be life-threatening. Levocarnitine clears the acylCoA compound by formation of acylcarnitine, which is quickly excreted. Carnitine deficiency is defined biochemically as abnormally low plasma concentrations of free carnitine, less than 20 µmol/L at one week post term and may be associated with low tissue and/or urine concentrations. Further, this condition may be associated with a plasma concentration ratio of acylcarnitine/levocarnitine greater than 0.4 or abnormally elevated concentrations of acylcarnitine in the urine. In premature infants and newborns, secondary deficiency is defined as plasma levocarnitine concentrations below age-related normal concentrations. End Stage Renal Disease (ESRD) patients on maintenance hemodialysis may have low plasma carnitine concentrations and an increased ratio of acylcarnitine/carnitine because of reduced intake of meat and dairy products, reduced renal synthesis and dialytic losses. Certain clinical conditions common in hemodialysis patients such as malaise, muscle weakness, cardiomyopathy and cardiac arrhythmias may be related to abnormal carnitine metabolism. Pharmacokinetic and clinical studies with CARNITOR have shown that administration of levocarnitine to ESRD patients on hemodialysis results in increased plasma levocarnitine concentrations.
1992
30
CARNITOR
levocarnitine
LOE Approaching
ORAL · SOLUTION
CLINICAL PHARMACOLOGY CARNITOR (levocarnitine) is a naturally occurring substance required in mammalian energy metabolism. It has been shown to facilitate long-chain fatty acid entry into cellular mitochondria, thereby delivering substrate for oxidation and subsequent energy production. Fatty acids are utilized as an energy substrate in all tissues except the brain. In skeletal and cardiac muscle, fatty acids are the main substrate for energy production. Primary systemic carnitine deficiency is characterized by low concentrations of levocarnitine in plasma, RBC, and/or tissues. It has not been possible to determine which symptoms are due to carnitine deficiency and which are due to an underlying organic acidemia, as symptoms of both abnormalities may be expected to improve with CARNITOR. The literature reports that carnitine can promote the excretion of excess organic or fatty acids in patients with defects in fatty acid metabolism and/or specific organic acidopathies that bioaccumulate acylCoA esters. Secondary carnitine deficiency can be a consequence of inborn errors of metabolism. CARNITOR may alleviate the metabolic abnormalities of patients with inborn errors that result in accumulation of toxic organic acids. Conditions for which this effect has been demonstrated are: glutaric aciduria II, methyl malonic aciduria, propionic acidemia, and medium chain fatty acylCoA dehydrogenase deficiency. Autointoxication occurs in these patients due to the accumulation of acylCoA compounds that disrupt intermediary metabolism. The subsequent hydrolysis of the acylCoA compound to its free acid results in acidosis which can be life-threatening. Levocarnitine clears the acylCoA compound by formation of acylcarnitine, which is quickly excreted. Carnitine deficiency is defined biochemically as abnormally low plasma concentrations of free carnitine, less than 20 µmol/L at one week post term and may be associated with low tissue and/or urine concentrations. Further, this condition may be associated with a plasma concentration ratio of acylcarnitine/levocarnitine greater than 0.4 or abnormally elevated concentrations of acylcarnitine in the urine. In premature infants and newborns, secondary deficiency is defined as plasma levocarnitine concentrations below age-related normal concentrations. PHARMACOKINETICS In a relative bioavailability study in 15 healthy adult male volunteers, CARNITOR Tablets were found to be bio-equivalent to CARNITOR Oral Solution. Following 4 days of dosing with 6 tablets of CARNITOR 330 mg b.i.d. or 2 g of CARNITOR oral solution b.i.d., the maximum plasma concentration (C max ) was about 80 µmol/L and the time to maximum plasma concentration (T max ) occurred at 3.3 hours. The plasma concentration profiles of levocarnitine after a slow 3 minute intravenous bolus dose of 20 mg/kg of CARNITOR were described by a two-compartment model. Following a single i.v. administration, approximately 76% of the levocarnitine dose was excreted in the ur
the treatment of primary systemic carnitine deficiency
1986
30
CARNITOR SF
levocarnitine
LOE Approaching
ORAL · SOLUTION
CLINICAL PHARMACOLOGY CARNITOR (levocarnitine) is a naturally occurring substance required in mammalian energy metabolism. It has been shown to facilitate long-chain fatty acid entry into cellular mitochondria, thereby delivering substrate for oxidation and subsequent energy production. Fatty acids are utilized as an energy substrate in all tissues except the brain. In skeletal and cardiac muscle, fatty acids are the main substrate for energy production. Primary systemic carnitine deficiency is characterized by low concentrations of levocarnitine in plasma, RBC, and/or tissues. It has not been possible to determine which symptoms are due to carnitine deficiency and which are due to an underlying organic acidemia, as symptoms of both abnormalities may be expected to improve with CARNITOR. The literature reports that carnitine can promote the excretion of excess organic or fatty acids in patients with defects in fatty acid metabolism and/or specific organic acidopathies that bioaccumulate acylCoA esters. Secondary carnitine deficiency can be a consequence of inborn errors of metabolism. CARNITOR may alleviate the metabolic abnormalities of patients with inborn errors that result in accumulation of toxic organic acids. Conditions for which this effect has been demonstrated are: glutaric aciduria II, methyl malonic aciduria, propionic acidemia, and medium chain fatty acylCoA dehydrogenase deficiency. Autointoxication occurs in these patients due to the accumulation of acylCoA compounds that disrupt intermediary metabolism. The subsequent hydrolysis of the acylCoA compound to its free acid results in acidosis which can be life-threatening. Levocarnitine clears the acylCoA compound by formation of acylcarnitine, which is quickly excreted. Carnitine deficiency is defined biochemically as abnormally low plasma concentrations of free carnitine, less than 20 µmol/L at one week post term and may be associated with low tissue and/or urine concentrations. Further, this condition may be associated with a plasma concentration ratio of acylcarnitine/levocarnitine greater than 0.4 or abnormally elevated concentrations of acylcarnitine in the urine. In premature infants and newborns, secondary deficiency is defined as plasma levocarnitine concentrations below age-related normal concentrations. PHARMACOKINETICS In a relative bioavailability study in 15 healthy adult male volunteers, CARNITOR Tablets were found to be bio-equivalent to CARNITOR Oral Solution. Following 4 days of dosing with 6 tablets of CARNITOR 330 mg b.i.d. or 2 g of CARNITOR oral solution b.i.d., the maximum plasma concentration (C max ) was about 80 µmol/L and the time to maximum plasma concentration (T max ) occurred at 3.3 hours. The plasma concentration profiles of levocarnitine after a slow 3 minute intravenous bolus dose of 20 mg/kg of CARNITOR were described by a two-compartment model. Following a single i.v. administration, approximately 76% of the levocarnitine dose was excreted in the ur
the treatment of primary systemic carnitine deficiency
1986
30
CYSTARAN
cysteamine hydrochloride
Peak
OPHTHALMIC · SOLUTION/DROPS
cystine-depleting agent by converting cystine to cysteine and cysteine-cysteamine mixed disulfides and reduces corneal cystine crystal accumulation.
corneal cystine crystal accumulation in patients with cystinosis
2012
30
MATULANE
procarbazine hydrochloride
LOE Approaching
ORAL · CAPSULE
CLINICAL PHARMACOLOGY The precise mode of cytotoxic action of procarbazine has not been clearly defined. There is evidence that the drug may act by inhibition of protein, RNA and DNA synthesis. Studies have suggested that procarbazine may inhibit transmethylation of methyl groups of methionine into t‑RNA. The absence of functional t‑RNA could cause the cessation of protein synthesis and consequently DNA and RNA synthesis. In addition, procarbazine may directly damage DNA. Hydrogen peroxide, formed during the auto‑oxidation of the drug, may attack protein sulfhydryl groups contained in residual protein which is tightly bound to DNA. Procarbazine is metabolized primarily in the liver and kidneys. The drug appears to be auto-oxidized to the azo derivative with the release of hydrogen peroxide. The azo derivative isomerizes to the hydrazone, and following hydrolysis splits into a benzylaldehyde derivative and methylhydrazine. The methylhydrazine is further degraded to CO 2 and CH 4 and possibly hydrazine, whereas the aldehyde is oxidized to N -isopropylterephthalamic acid, which is excreted in the urine. Procarbazine is rapidly and completely absorbed. Following oral administration of 30 mg of C-labeled procarbazine, maximum peak plasma radioactive concentrations were reached within 60 minutes. After intravenous injection, the plasma half-life of procarbazine is approximately 10 minutes. Approximately 70% of the radioactivity is excreted in the urine as N -isopropylterephthalamic acid within 24 hours following both oral and intravenous administration of C-labeled procarbazine. Procarbazine crosses the blood-brain barrier and rapidly equilibrates between plasma and cerebrospinal fluid after oral administration.
1969
30

Pipeline & Clinical Trials

Chenodeoxycholic Acid
Cerebrotendinous Xanthomatoses
N/A
Clinical Trials (1)
NCT05499026Safety and Efficacy of CDCA in CTX Chenodeoxycholic Acid (CDCA) in Patients Affected by Cerebrotendinous Xanthomatosis (CTX)
N/A
ERCP and Confocal imaging
Biliary Stricture
N/A
Clinical Trials (1)
NCT00892632Confocal Endomicroscopy for Biliary Strictures -Phase I
N/A
N/A
Clinical Trials (1)
NCT00351234Carnitine Levels and Carnitine Supplementation in Type I Diabetes
N/A
cysteamine hydrochloride
Cystinosis
N/A
Clinical Trials (1)
NCT00010426Randomized Study of New Formulation Ophthalmic Cysteamine Hydrochloride for Corneal Cystine Accumulation in Patients With Cystinosis
N/A
Phase 1
Clinical Trials (1)
NCT01954017STP206 for the Prevention of Necrotizing Enterocolitis (NEC)
Phase 1
PEG-L-asparaginase
T-Cell Lymphoma
Phase 1
Clinical Trials (1)
NCT01878708A Pilot Study of Oncaspar® + Dexamethasone in Patients With Relapsed or Refractory T-Cell Lymphoma
Phase 1
STP206 or vehicle control
Healthy
Phase 1
Clinical Trials (1)
NCT00922324Safety and Tolerability of STP206 in Healthy Adult Subjects
Phase 1
Phase 1
Valproic Acid and Levocarnitine
Spinal Muscular Atrophy Type I
Phase 1/2
Clinical Trials (1)
NCT00661453CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I
Phase 1/2
Valproic Acid and Levocarnitine
Spinal Muscular Atrophy
Phase 2
Phase 2
Clinical Trials (1)
NCT04231266Multi-Center Study of ManNAc for GNE Myopathy
Phase 2
Phase 2
Clinical Trials (1)
NCT02043587Chemotherapy With Liposomal Cytarabine CNS Prophylaxis for Adult Acute Lymphoblastic Leukemia & Lymphoblastic Lymphoma
Phase 2
Phase 2
Clinical Trials (1)
NCT00905034Methotrexate, Vincristine, Pegylated L-Asparaginase and Dexamethasone (MOAD) in Acute Lymphoblastic Leukemia (ALL) Salvage
Phase 2
Syntocinon Nasalspray
Female Sexual Dysfunction
Phase 2
Clinical Trials (1)
NCT02229721Effect of Chronic Intranasal Oxytocin Administration on Sexual Function in Pre- and Postmenopausal Women
Phase 2
acetyl-L-carnitine/statin
Diabetes Type 2
Phase 3
Clinical Trials (1)
NCT00984750Acetyl-L-Carnitine in Type 2 Diabetes
Phase 3
EZN-2279
ADA-SCID
Phase 3
Clinical Trials (1)
NCT01420627EZN-2279 in Patients With ADA-SCID
Phase 3
Chenodeoxycholic acid
Cerebrotendinous Xanthomatoses
Phase 3
Clinical Trials (1)
NCT06260748A Study of Chenodeoxycholic Acid (CDCA) in Newly Diagnosed Participants With Cerebrotendinous Xanthomatosis (CTX)
Phase 3

Open Jobs (0)

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Interview Prep Quick Facts
Founded: 2024
Portfolio: 9 approved products, 15 clinical trials
Top TAs: Rare Diseases, Oncology, Metabolic Diseases
Publications: 12 in PubMed
Portfolio Health
Peak1 (11%)
LOE Approaching8 (89%)
9 total products
Therapeutic Area Focus
Rare Diseases
4 marketed1 pipeline
Oncology
3 pipeline
Ophthalmology
1 marketed
Cardiovascular
1 pipeline
Marketed
Pipeline