Interesting Drugs in Pipeline
Aclasta (Zoledronic acid)
Novartis — This drug is currently under investigation for the prophylactic treatment of post-menopausal osteoporosis in women. Its significant advantage over others in its class od drugs is that it is given in a once a year infusion. Clinical studies have shown it is effective in reducing the incidence of bone fractures with the effect sustained over 3 years. The incidence of side effects were similar to those of a placebo infusion.
Alzehmed for Alzheimer's Disease
Neurochem-Canada — Phase 2-3 Estimated marketing 2006-2007. Recently the company reported no significant improvement in a large group of patients treated with this drug.
Aztreonam Lysine for Pseudomonal infections in Cystic Fibrosis
Gilead — when given by inhalation, Aztreonam significantly improved respiratory symptoms and delayed the time when inhalation of tobramycin would be required.
Cetrorelix for Benign Prostatic Hypertrophy (BPH)
Aeterna Zentaris — this drug acts as a luteinizing-releasing hormone antagonist. Although it has to be given intramuscularly, preliminary studies have shown that cetrorleix may be a potential safe and effective drug to treat the symptoms of BPH.
Cladribine for Relapsing Multiple Sclerosis
Serono —
Ceplene (Histamine) for Acute Myeloid Leukemia (AML)
Maxim Pharma — Ceplene when combined with Interleukin, resulted in a significant improvement in cancer-free survival in a group of patients followed for a minimum of 3 years.
Dapoxetine for Premature Ejaculation (Johnson and Johnson)
USA — Phase 3
Dimebon (Medivation) for Alzheimer's disease
This drug in Phase 2-3 studies is being studied for its effect in mild to moderate Alzheimer's disease. When compared to placebo it appears to significantly slow down the progression of the disease. Its proposed mechanism is to increase mitochondrial function. It appears to be well-tolerated.
Entereg (Alvimopan) for Intestinal Paralysis
Postoperative ilius (intestinal paralysis) is common complication of gastrointestinal surgery. This is made worse by the administration of narcotics for the pain following surgery. Entereg blocks the opioid receptor in the intestine which when stimulated, slows intestinal peristalsis. Blocking this receptor from the effects of opioids on the intestine results in a more rapid recovery of intestinal function.
Genasense (Oblimersen) for Chronic Lymmphocytic Leukemia (CLL)
Genta — This new injectable drug has been studied in patients with relapsed or refractory CLL. The mechanism of action is to inhibit the production of bcl-2, a protein made by cancer cells which prevents their death after being exposed to antineoplastic drugs Patients receiving Genasense and standard chemotherapy were four times more likely to achieve complete remission compared to patients receiving chemotherapy alone.
Genasense (Oblimersen)
Genta Pharma — this novel new drug is being investigated for its value in the treatment of advanced melanoma. Studies to date have investigated the effect on the response rate of adding Genasense to dacarbazine (DTIC). The one major study to date showed the overall response rate increased from 9.3% to 17.2%. The overall progression-free survival increased from 1.6 months to 3.1 months and the overall survival from 9.7 months to 11.4 months. The most frequent adverse effects were fever, nausea, neutropenia (decreased WBC’s), thrombocytopenia (decreased platelets) and anemia.
Junovan (Mifamurtide) for Osteosarcoma
IDM Pharma — A NDA has been submitted for approval of Junovan in the treatment non-metastatic respectable osteosarcoma. In a large trial with a followup of 5 years, Junovan administration resulted in a significant improvement in disease-free survival and overall survival. At 6 years followup the probability of survival in patients given Junovan plus adjuvant chemotherapy was 77% compared to 66% in patients given adjuvant chemotherapy alone.
Lestaurtinib for Acute Mylogenous Leukemia (AML)
Cephalon — this drug in Phase II/III studies when combined with currently available therapy for AML resulted in complete remission in those patients resistant to lestaurtinib.
Nplate (Romiplostim) for chemotherapy-induced thrombocytopenia
Amgen — this drug under FDA priority review is indicated for the treatment of chemotherapy-induced thrombocytopenia. It is a thrombopoises-stimulating agent and in a Phase 3 study, 61% of treated patients had a positive platelet stimulus (>50,000) for greater than 6/8 study weeks. Nplate also resulted in less need for rescue medications such as steroids or IVIG.
Pirfenidone for Idiopathic Pulmonary Fibrosis
Intermune — this is an orally active drug which acts to inhibit collagen synthesis and reduce the components of the immune system which produce fibroblast stimulation. Fibroblast stimulation in the lung results in the production of collagen, the necessary precursor to the development of pulmonary fibrosis. Phase II/III
Panitumumab for Metastatic Colorectal Cancer
Genentech — this drug is under study for metastatic colorectal cancer. In preliminary studies it resulted in a 46% decrease in tumor progression rate in a group of patients who had failed on standard chemotherapy.
Pargluva (Muraglitazar) for Diabetes
Bristol-Myers — This drug joins the list of oral agents indicated for the treatment of Type II diabetes. In a large study, Pargluva produced a satistically significant better reduction in blood HbA1c values (indicator of diabetes control) than Actos, also an oral antidiabetic drug I in structurally-related class. Pargluva differs from Actos in that it is a dual peroxisome-activator receptor agonist. Side effects for Pargluva were 9.2% and for Actos 7.2%. There may be a higher incidence of heart failure associated with Pargluva than with Actos.
Promacta (Eltrombopag) for idiopathic thrombocytopenic purpura (ITP)
Glaxo — an NDA has been filed for this drug which has been studied for its ability to moderate idiopathic thrombocytopenic purpura (ITP). If approved it will be the first oral drug approved for ITP. It acts as an antagonist to the thrombopoietin receptor. In a recent study, 59% of treated patients achieved platelet counts <50,000/microliter of blood.
Provenge (Sipuleucel-T) for Prostate Cancer
This is a new form of treatment where the patients white blood cells are taken from the blood and then incubated with a fusion protein containing prostatic acid phosphatase. The white blood cells are then injected back into the patient. This process then activates killer T-lymphocytes which attach t an kill the prostate cancer dells.
Pulminiq (Inhaled Cyclosporine)
Chiron — this new form of cyclosporine will be indicated for the prevention of rejection in patients who had lung transplants. Although survival data are dramatic, the low number of patients tested may delay approval.
Rimonabant for Obesity
Sanofi — initial results are very positive with an average of 17 lbs lost in the first year of treatment and no apparent loss of effectiveness with continued dosing. There was also a 7-10% reduction in waist size. Available late 2005 or early 2006.
Riquent ® (Abetimus Sodium)
La Jolla Pharmaceutical — is being developed as a treatment for systemic lupus erythematosus (SLE). Lupus, which is a chronic, potentially life-threatening autoimmune disorder in which patients' diseased B cells produce antibodies to double-stranded DNA (dsDNA). These antibodies are believed to cause lupus kidney disease that can lead to kidney failure, dialysis, kidney transplantation, and death. Although many lupus patients may initially develop mild forms of kidney disease, it often progresses over time to become more severe, and can lead to renal failure. Riquent, is designed to arrest the production of antibodies to double-stranded DNA (dsDNA) in lupus patients and to arrest or delay lupus renal disease without suppressing the healthy functions of the immune system.
Rotigotine for Restless Legs Syndrome
Schwarz Pharma — Phase III trials currently underway with this cutaneous patch have shown a statistically significant reduction in RLS symptoms with no serious side effects.
Rufinamide for Lennox-Gastaut Syndrome (LGS)
Eisai — An NDA as been submitted for this medication with the indication Lennox-Gastaut Syndrome (LGS). It is an anticonvulsant with a structure different from those currently approved in the USA. 42% of patients with LGS had a 50% or more reduction in atonic Siezures. For all patients, there was a 33% reduction in seizure frequency.
Satraplatin for Hormone-Refractory Prostate Cancer
Spectrum — 950 men were randomized to receive either satraplatin plus prednisone or placebo pluus prednisone. The satraplatin group had a 40% reduction of risk of disease progression compared to the placebo-prednisone group. An NDA has been submitted and is currently under consideration by the FDA.
Solaris (Eculizumab) for Paroxysmal
Alexion Pharmaceuticals — Nocturnal Hemoglobinemia (PNH)-Completed Phase III studies with Solaris have shown a significant reduction of symptoms of PNH compared with placebo. This improvement translated to no need for transfusions compared with 10 units of blood in the placebo group. Stabilization of hemoglobin levels was achieved in49% of Solaris patients compared with 0% of placebo patients. Virtually all of the disease-related symptoms were significantly reduced. Side effects were: headache,back pain, and nausea.
Sparlon (Modafinil) for ADHD
This medication will be indicated for the treatment of attention-deficit/hyperactivity disorder (ADHD). It is a new dose form of Provigil which is used to treat narcolepsy. This drug taken once a day may be as effective as the amphetamines and methylphenidate without the same degree of cardiovascular risk.
Sugammadex (Bridion) for neuromuscular blockade
Schering — this novel agent is pending approval for the reversal of neuromuscular blockade by the skeletal muscle relaxant rocuronium. The main advantage of this drug over others designed to reverse neuromuscular blockade is that it does not act by depending on the inhibition of acetylcholinesterase. Shallow pancuronium blockade has also been successfully reversed by this medication. NOTE: FDA REFUSED APPROVAL
Torisel (Temsirolimus)
Wyeth — this drug is currently being studied in the treatment of advanced renal cell carcinoma. In a phase 3 study, Torisel improved overall survival time compared with treatment with interferon-alpha (10.9 months vs. 7.3 months). The mechanism of action is unique, an inhibition of mTOR kinase, a protein regulators of cell growth, proliferation, and cell survival. Wyeth has submitted an NDA to the US FDA and hopes to market the product in late 2007.
Tykerb (Lapatinib) for Advanced Breast Cancer
GSK — for advanced breast cancer who have HER2 receptor overexpression and whose disease has become refractory to Herceptin. Tykerb also blocks HER2 receptors but does so via a mechanism different from Herceptin. In a trial of 321 women with progressive disease the combination of Xyloda and Tykerb resulted in a temporary cessation of tumor growth with the time to tumor progression, significantly extended. The side effects of Tykerb, which is given by mouth, were minor and consisted of diarrhea and nausea.
Urocidin (Mycobacterium Cell Wall-DNA Complex) for Refractory Bladder Cancer
Bioniche — this drug is currently in Phase III testing in the United States where Urocidin is being compared to BCG vaccine. Early studies have shown that Urocidin is associated with a much lower incidence of side effects than BCG.
VEGF-Trap (Aflibercept) for Platinum-Resistant Ovarian Cancer
Sanofi-Aventis — this dug in Stage II-III testing prevents the development of new blood vessels needed by a growing tumor (antiangiogenesis). When studied in a group of 162 women with drug-resistant ovarian cancer, tumors shrank in 8% of patient and stopped growing in an additional 71%.
Xinlay (Atrasentan)
Abbott — for the treatment of metastatic hormone-refractory prostate cancer, an advanced stage of the disease that no longer responds to hormone treatment and has spread to other parts of the body, such as the bone. Phase II and III clinical trials in men with metastatic hormone-refractory prostate cancer showed that Xinlay could slow disease progression and delay in time to onset of bone pain. Xinlay is an investigational, oral, once-daily, non-hormonal, non-chemotherapy, anti-cancer agent that belongs to a class of compounds known as selective endothelin-A receptor antagonists (SERA™). SERAs antagonize the effect of endothelin-1 (ET-1), one of the proteins thought to be involved in the stimulation of the spread of cancer cells.
Zenvia (Dextromethorphan/Quinidine) for Diabetic Neuropathic Pain
Avanir Pharma — Significantly reduced pain associated with diabetic neuropathy as measured by Pain Relief and Pain Intensity Ratings Scale. Side effects were mild.