Specialty pipeline forecast as of June 2008

[diana.papshev]

NOTE: This forecast is updated on a monthly basis, please check Specialty Drug Pipeline by Month for the latest forecast post.

2-3Q08:

Nplate™ (romiplostim) by Amgen, expected approval in 2Q08
• Class/MOA: thrombopoiesis-stimulating protein
• Use: Immune Thrombocytopenic Purpura (ITP)
• Dosage form: SC injection
• Link to press releases:

o Amgen Press Release 6/08
o Amgen Press Release 1/08

• Link to clinicaltrials.gov:

o Phase III study compared to standard of care for ITP

• Link to published data:

o Efficacy of romiplostim in patients with chronic i...[Lancet. 2008] - PubMed Result

Cayston (aztreonam lysine) by Gilead; expected approval 9/08
• Class/MOA: monobactam
• Use: cystic fibrosis
• Dosage form: inhalation
• Link to press releases:

o Gilead press release 6/08
o Gilead press release 9/07

• Link to clinicaltrials.gov:

o Phase III trial for CF patients with P.Aeruginosa Airway Infection

• Link to published data:

o A phase 2 study of aztreonam lysine for inh…[Pediatr Pulmonol. 2008] - PubMed Result

Cinryze by Lev Pharmaceuticals; Approvable letter issued 1/08; FDA advisory committee unanimously recommends approval 5/08; expected approval 9/08
• Class/MOA: C1-esterase inhibitor
• Use: replacement therapy for the treatment of hereditary angioedema (HAE)
• Dosage form: IV injection
• Link to press releases:

o News release 5/08
o News release 3/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

Xenazine (tetrabenazine) by Prestwick; FDA advisory committee unanimously recommends approval
• Class/MOA: selective and reversible centrally-acting dopamine depleting drug, works by inhibiting a molecule known as VMAT2 (vesicular monoamine transporter 2)
• Use: treatment of chorea associated with Huntington’s disease (HD)
• Dosage form: oral
• Link to press releases:

o News release 12/07

• Link to clinicaltrials.gov:

o Trials registered with NIH

Actemra™ (tocilizumab) by Roche and Chugai, response to BLA expected 9/08; approved in Japan 4/08
• Class/MOA: first humanized interleukin-6 (IL-6) receptor-inhibiting monoclonal antibody
• Use: treatment of RA
• Dosage form: IV infusion
• Link to press releases:

o Roche Press Release RADIATE and AMBITION trials 6/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

• Link to recent literature:

o Juvenile Ideopathic Arthritis trial [Lancet. 2008] - PubMed Result
o OPTION trial: [Lancet. 2008] - PubMed Result

Promacta™ (eltrombopag) by GlaxoSmithKline, FDA granted priority review and advisory panel recommended approval in 5/08; expected approval 9/08
• Class/MOA: thrombopoietin-receptor
• Use: Immune Thrombocytopenic Purpura (ITP)
• Dosage form: oral agent
• Link to press releases:

o Press Release 5/08
o GSK Press Release 3/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

• Link to recent literature:

o Eltrombopag for the treatment of chronic idiopathic… N Engl J Med. 2007

Prograf MR (tacrolimus modified-release) by Astellas; NDA submitted 12/05; received approvable letter from the FDA in 1/07 for kidney and in 4/08 for liver transplats (and a non-approvable letter for heart transplants in 1/07)
• Class/MOA: immunosuppressant
• Use: prevention of organ rejection in patients receiving allogeneic liver/kidney transplants
• Dosage form: oral
• Link to press releases:

o Astellas press release 6/08
o News release 12/05

• Link to clinicaltrials.gov:

o Trials registered with NIH

• Link to published data:

o Two years postconversion from a prograf-based regimen… [Transplantation. 2007] - PubMed Result
o Once-daily tacrolimus extended release…[Transplantation. 2007] - PubMed Result

Tolvaptan by Otsuka; FDA advisory panel recommended approval in 6/08
• Class/MOA: selective vasopressin-receptor antagonist
• Use: hypervolemic/euvolemic hyponatremia
• Dosage form: oral
• Link to press releases:

o News article 6/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

4Q08:

Arcalyst (rilonacept) by Regeneron; approved 2/08; launch 2008
• Class/MOA: Interleukin-1 blocker
• Use: long term treatment of two Cryopyrin-Associated Periodic Syndromes (CAPS) disorders: Familial Cold Auto-Inflammatory Syndrome (FCAS) and Muckle-Wells Syndrome (MWS)
• Dosage form: SC injection
• Link to press releases:

o News release 2/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

• Link to Prescribing Information:

o PI

Cimzia™ (certolizumab pegol) by UCB, BLA filed in 12/07; FDA approved for Crohn's disease in 4/08; FDA response on RA expected in 10/08
• Class/MOA: anti-TNF agent
• Use: treatment of moderate to severe or active Crohn’s disease, moderate to severe or active RA, and moderate to severe psoriasis
• Dosage form: SC injection
• Link to press releases:

oUCB Press Release 2/07

• Link to clinicaltrials.gov:

oPhase III Open Label Long-Term Safety Study for RA

Denosumab by Amgen, FDA to complete review of postmenopausal osteoporosis (PMO) data by 2nd half of 2008 (in development for RA and multiple cancers)
• Class/MOA: fully-human monoclonal antibody that specifically targets RANK Ligand, the essential mediator of osteoclasts (the cells that break down bone)
• Use: treatment of postmenopausal osteoporosis (PMO) and treatment-induced bone loss
• Dosage form: SC injection
• Link to press release:

oAmgen Press Release 5/08
oAmgen Press Release 4/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

• Link to published studies:

o Effects of Denosumab on Bone Mineral Density and Bone Turnover in Postmenopausal Women [Journal of Clinical Endocrinology & Metabolism. 2008]

Fludara (fludarabine) by Xanthus (acquired by Antisoma); NDA filed 1/08; expected approval 4Q08
• Class/MOA: antimetabolite
• Use: treatment of relapsed B-cell chronic lymphocytic leukemia (CLL)
• Dosage form: oral
• Link to press releases:

o News release 1/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

Hycamtin (topotecan) by GSK; approved 10/07; launch in 2008
• Use: lung cancer
• Dosage form: oral
• Link to press releases:

o News release 10/07

o News release 10/07

• Link to clinicaltrials.gov:

o Trials registered with NIH

• Link to Prescribing Information:

o PI

Numax® (motavizumab) by MedImmune and AstraZeneca, BLA submitted 1/08; expected approval 4Q08, launch 2009
• Class/MOA: monoclonal antibody
• Use: potential to prevent serious lower respiratory tract disease caused by RSV in pediatric patients at high risk of RSV disease injection
• Dosage form: IM injection
• Link to press releases:

o Press Release 5/08
o News article 10/07

• Link to clinicaltrials.gov:

o Trials registered with NIH

Rebif® (interferon beta-1a) (new formulation for improved tolerability) by EMD Serono/Pfizer; sBLA submitted 4/06; expected approval 4Q08
• Class/MOA: interferon-beta 1a
• Use: MS
• Dosage form: SC injection
• Link to press releases:

o News release 3/08
o News release 4/06

• Link to clinicaltrials.gov:

o Trials registered with NIH

Provenge® (sipuleucel-T) by Dendreon, results of analysis due 2nd half 2008; currently under regulatory review for potential marketing approval
• Class/MOA: may represent the first in a new class of therapy known as Active Cellular Immunotherapies Active Cellular Immunotherapies (ACIs) An approach that uses live human cells to re-engage the patient's own immune system
• Use: treatment of metastatic hormone refractory prostate cancer (HRPC)
• Dosage form: IV infusion
• Link to press release:

o Press release 3/08
o News article 2/08

• Link to clinicaltrials.gov:

o Phase II trial in Metastatic Prostate Cancer After Failing Hormone Therapy

Tasigna (nilotinib) by Novartis; approved 10/07, with launch in 2008
• Class/MOA: Protein-tyrosine kinase inhibitor
• Use: reatment of chronic phase and accelerated phase Philadelphia chromosome positive chronic myelogenous leukemia (CML) in adult patients resistant or intolerant to prior therapy that included imatinib
• Dosage form: oral
• Link to press releases:

o News article 10/07

• Link to clinicaltrials.gov:

o Trials registered with NIH

• Link to published data:

o Nilotinib a highly selective BCR-ABL… [Blood. 2007] - PubMed Result
o Nilotinib a highly selective BCR-ABL…[Blood. 2008] - PubMed Result

• Link to Prescribing Information:

o PI

Vatalanib by Novartis/Schering AG
• Class/MOA: protein tyrosine kinase inhibitor
• Use: second-line treatment of patients with metastatic colorectal cancer who have failed standard chemotherapy treatment
• Dosage form: oral
• Link to press releases:

o News release 4/07

• Link to clinicaltrials.gov:

o Trials registered with NIH

Ustekinumab by Centocor, FDA advisory committee unanimously recommended approval in 6/08
• Class/MOA: human monoclonal antibody
• Use: treatment of adult patients with chronic moderate to severe plaque psoriasis
• Dosage form: SC injection
• Link to press release:

o News article 6/08
o Centocor Press Release 6/08

• Link to clinicaltrials.gov:

o Phase III safety and efficacy study in Patients With Severe Plaque-Type Psoriasis
o Phase III safety and efficacy study compared to Etanercept for the Treatment of Moderate to Severe Plaque Psoriasis

• Link to published data:

o PHOENIX 1 study [Lancet. 2008] – PubMed Results
o PHOENIX 2 study [Lancet. 2008] – PubMed Results

Surfaxin (lucinactant) by Discovery Laboratories, Inc.; approvable letter was received from the FDA in 6/2008, approval is expected in late 2008
• Class/MOA: precision-engineered version of natural human lung surfactant and contains Discovery Labs’ KL-4 peptide
• Use: respiratory distress syndrome
• Dosage form: inhalation
• Link to press releases:

o Discovery Labs press release 6/08

• Link to clinicaltrials.gov:

Trials registered with NIH

• Link to published data:

o One-year follow-up of very preterm infants… [Pediatrics. 2007] - PubMed Result

2009:

Cystic Fibrosis:

Denufosol by Inspire Pharmaceuticals, granted fast-track designation by the FDA; NDA expected to be filed in 2009
• Class/MOA: enhance the lung's innate mucosal hydration and mucociliary clearance through stimulation of the P2Y2 receptor
• Use: cystic fibrosis
• Dosage form: inhalation
• Link to press releases:

o Inspire Press Release 6/08 Pages

• Link to clinicaltrials.gov:

o Phase III study in Patients With CF Lung Disease

Hereditary angioedema (HAE) treatment:

Firazyr (icatibant) by Jerini; FDA has issued a not-approvable letter 4/08; the company plans to respond by 10/08
• Class/MOA: synthetic peptidomimetic, works by blocking the B2 receptor as an antagonist to the peptide hormone bradykinin
• Use: treatment of acute hereditary angioedema (HAE) attacks
• Dosage form: SC injection
• Link to press releases:

o News release 6/08

o News release 4/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

Berinert P (C1-esterase inhibitor) by CSL Behring; BLA filed 3/08; expected approval 1/09
• Class/MOA: C1-esterase inhibitor
• Use: hereditary angioedema
• Dosage form: IV
• Link to press releases:

o News article 3/08

• Link to clinicaltrials.gov:

o Trials registered at NIH

Hereditary antithrombin deficiency treatment:

ATryn (antithrombin alfa) by Ovation and GTC Biotherapeutics; approval expected in early 2009
• Class/MOA: antithrombin
• Use: treatment of hereditary antithrombin deficiency patients during high risk procedures
• Dosage form: IV injection
• Link to press releases:

o News release 6/08

• Link to clinicaltrials.gov:

o Phase III trial registered with NIH

• Link to published data:

o Antithrombin alfa in hereditary… [Thromb Haemost. 2008] - PubMed Result

Hypereosinophilic syndrome:

Bosatria (mepolizumab) by GSK; in Phase III trials; FDA filing expected in late 2008
• Class/MOA: humanized monoclonal antibody that inactivates IL-5
• Use: hypereosinophilic syndrome
• Dosage form: IV
• Link to press releases:

o News release 3/08

• Link to clinicaltrials.gov:

o Phase III trial registered with NIH

Chronic fatigue syndrome treatment:

Ampligen (poly I/poly C12U) by Hemispherx Biopharma, Inc.; NDA filed 10/07; received questions from FDA 12/07 and responded 3/08; expected approval 2009
• Class/MOA: RNA (nucleic acid) molecules
• Use: chronic fatigue syndrome
• Dosage form: IV
• Link to press releases:

o News release 1/08

o[] Hemispherx getting closer to satisfying application issues - Philadelphia Business Journal: News release 3/08[/url]

• Link to clinicaltrials.gov:

o Trials registered with NIH

Amyloid A amyloidosis :

Kiacta™ (eprodisate) by Bellus Health (formally Neurochem), FDA has recommended a second Phase III trial, which is expected to begin late 2008
• Class/MOA: delays the progression of AA amyloidosis and inhibits or reduces amyloid deposition
• Use: Amyloid A amyloidosis
• Dosage form: oral agent, orphan drug
• Link to press releases:

o Bellus Health Inc. Press Release 3/08

• Link to clinicaltrials.gov: none

Hepatitis treatment:

Telaprevir by Vertex; Phase IIb trial showed positive SVR rate, currently in Phase III; launch projected for 2009
• Class/MOA: HCV protease inhibitor
• Use: Hepatitis C
• Dosage form: oral
• Link to press releases:

o News release 6/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

HIV:

Vicriviroc by Schering-Plough, Phase III studies started 9/07
• Class/MOA: extracellular inhibitor of HIV infection designed to block entry of infectious virions into uninfected CD4 cells via antagonism of the CCR5 co-receptor
• Use: R5-type HIV infection in combination with other antiretroviral agents (which must include a protease inhibitor)
• Dosage form: oral
• Link to press releases:

o Schering-Plough Press Release 4/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

Systemic lupus erythematosus (SLE or lupus):

Orencia (abatacept) by BMS for SLE; currently in Phase II; approval estimated for 2009
• Class/MOA: fusion protein
• Use: SLE
• Dosage form: IV
• Link to clinicaltrials.gov:

o Trials registered with NIH

Riquent (abetimus) by La Jolla; FDA granted fast track status; estimated approval 2009
• Class/MOA: reduction of circulating levels of anti-dsDNA antibodies
• Use: SLE
• Dosage form: IV
• Link to press releases:

o La Jolla press release 4/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

Rituxan (rituximab) by Biogen Idec/Genentech; approval estimated for 2009
• Class/MOA: genetically engineered chimeric murine/human antibody that binds specifically to the CD20 antigen on normal and malignant-B-lymphocytes
• Use: SLE
• Dosage form: IV
• Link to press releases:

o Biogen/Genentech press release, 4/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

Exocrine pancreatic insufficiency (EPI):

Zentase (EUR 1008) by Eurand Pharmaceuticals Ltd.; NDA filed 6/07; priority review granted 2/08; approvable letter received from the FDA in 6/08, with expected approval 2009
• Class/MOA: porcine-derived, pancreatic enzyme replacement product (PEP)
• Use: treatment of exocrine pancreatic insufficiency (EPI)
• Dosage form: oral capsule
• Link to press releases:

o Eurand press release 6/08
o Eurand press release 4/07

• Link to clinicaltrials.gov:

o Phase III safety and efficacy trial for CF and EPI

Iron replacement:

Ferumoxytol Intravenous Injection by AMAG Pharmaceuticals, Inc.; NDA submitted 12/07 and accepted for review 2/08; expected approval 12/09
• Class/MOA: blood pool agent; true intravascular contrast agent that remains in the blood stream for an extended period of time
• Use: iron replacement therapy for chronic kidney disease (CKD) patients whether or not on dialysis
• Dosage form: IV
• Link to press releases:

o News release 4/08

o AMAG press release 5/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

Multiple Sclerosis:

Fampridine SR by Acorda; expected filing in early 2009
• Class/MOA: block potassium channels on ions
• Use: Multiple sclerosis
• Dosage form: oral
• Link to press releases:

o Acorda press release 6/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

Raynaud’s disease:

MQX-503 topical by MediQuest Therapeutics Inc; NDA submitted 4/08,
• Use: topical formulation designed to treat Raynaud 's Disease
• Dosage form: topical
• Link to press releases:

o News release 4/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

Botulism toxins:

Reloxin (botulinum toxin type A) by Ipsen and Medicis; BLA submitted in 3/08 and accepted by FDA 5/08, estimated date of approval 1/09
• Use: temporary improvement in the appearance of facial wrinkles
• Dosage form: SC injection
• Link to press releases:

o News release 5/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

Dysport (botulinum toxin type A) by Ipsen; BLA accepted by FDA 1/08
• Use: cervical dystonia
• Dosage form: SC injection
• Link to press releases:

o News release 1/08

Pulmonary Arterial Hypertension (PAH):

Viveta (treprostinil inhaled) by LungRx/United Therapeutics; in Phase III trials, approval expected in 2009
• Class/MOA: prostacyclin analog
• Use: PAH
• Dosage form: inhaled nebulizer
• Link to press releases:

o News article 5/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

Treprostinil by United Therapeutics
• Class/MOA: prostacyclin analogs
• Use: PAH
• Dosage form: oral
• Link to clinicaltrials.gov:

o Trials registered with NIH

Tadalafil by Lilly/Icos
• Class/MOA: Phosphodiesterase Type 5 Inhibitor
• Use: PAH
• Dosage form: oral
• Link to clinicaltrials.gov:

o Trials registered with NIH

Terguride by Ergonex Pharma; received orphan drug status 5/08
• Class/MOA: strong anti-serotoninergic activity by acting as a potent antagonist on 5-HT2B and 5-HT2A receptors
• Use: PAH
• Link to press releases:

o News article 5/08

Cancer treatments:

Axitinib by Pfizer; orphan drug for pancreatic cancer as of 5/07; in Phase III studies, expected approval 2009
• Class/MOA: small molecule tyrosine kinase inhibitor
• Use: pancreatic, thyroid cancer, melanoma, NSCLC, breast, colorectal cancer
• Dosage form: oral
• Link to press releases:

o News article 6/07

• Link to clinicaltrials.gov:

o Trials registered with NIH

Advexin by Introgen; BLA submitted in 6/08 for priority review
• Class/MOA: tumor suppressor p53 gene therapy
• Use: recurrent refractory head and neck cancer
• Dosage form: IV
• Link to press releases:

o Introgen press release 6/08

• Link to clinicaltrials.gov:

o Phase III study

Mozobil (plerixafor) by Genzyme; BLA submitted in 6/08, launch is anticipated in early 2009
• Class/MOA: CXCR4 chemokine receptor antagonist
• Use: enhanced mobilization of hematopoietic stem cells for collection and subsequent autologous transplantation in patients with lymphoma and multiple myeloma
• Dosage form: SC
• Link to press releases:

o Genzyme press release 6/08

• Link to clinicaltrials.gov:

o Phase III study

Motesanib diphosphate (AMG 706) by Amgen/Takeda; Phase III for NSCLC; Phase II studies versus bevacizumab in the treatment of metastatic breast cancer and NSCLC; in Phase Ib combination studies with multiple tumor types ongoing; estimated approval 2009
• Class/MOA: targets vascular endothelial growth factor receptors 1, 2 and 3 (VEGFR1-3); and in apoptosis, rhApo2L/TRAIL
• Use: metastatic breast cancer and NSCLC
• Dosage form: oral
• Link to press releases:

o Amgen press release 2/08

o Amgen pipeline 2008

• Link to clinicaltrials.gov:

o Trials registered with NIH

Panobinostat by Novartis; orphan drug status granted for topical treatment of cutaneous T-cell lymphoma (CTCL) 9/07; expected approval 2009
• Class/MOA: member of the hydroxamic acid group of histone deacetylase (HDAC) inhibitors that have been shown to impede multiple pathways implicated in cancer and reverse epigenetic events associated with cancer
• Use: lymphoma, prostate cancer
• Dosage form: topical, IV, po
• Link to press releases:

o News article 10/07

• Link to clinicaltrials.gov:

o Trials registered with NIH

Pazopanib by GSK
• Class/MOA: angiogenesis inhibitor targeting vascular endothelial growth factor receptor (VEGFR), platelet-derived growth factor receptor (PDGFR) and c-kit
• Use: advanced or metastatic renal cell carcinoma (RCC), ovarian cancer and soft tissue sarcoma (STS)
• Dosage form: oral
• Link to press releases:

o GSK press release, 5/08
o News article 6/07

• Link to clinicaltrials.gov:

o Trials registered with NIH

Zactima (vandetanib) by AstraZeneca; NDA submission expected in 2008, with potential approval in 2009
• Class/MOA: tyrosine kinase inhibitor which targets vascular endothelial growth factor receptor 2 (VEGFR-2), epidermal growth factor receptor (EGFR) and RET kinase
• Use: NSCLC
• Dosage form: oral
• Link to press releases:

o News article 3/08

• Link to clinicaltrials.gov:

o Trials registered with NIH]

Lestaurtinib by Cephalon, NDA filing for 1st half of 2008; granted orphan drug status 4/08
• Class/MOA: potent inhibitor of several tyrosine kinases including FLT-3 and TrkA
• Use: targeted agent against acute myeloid leukemia (AML) in patients at first relapse from standard induction
• Dosage form: oral
• Link to press releases:

o Cephalon News article 4/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

Xerecept® (corticorelin) by Celtic Pharma and Neurobiological Technologies, NDA filing planned for 2008
• Class/MOA: may present a steroid-sparing alternative to dexamethasone
• Use: treatment of peritumoral brain edema
• Dosage form: SC injection
• Link to press release:

o Celtic Pharma Press release 12/07

• Link to clinicaltrials.gov:

o Trials registered with NIH

Xyotax™ (paclitaxel poliglumex) by Cell Therapeutics, Phase III trial started 10/07, FDA granted Fast-track status
• Class/MOA: biologically-enhanced chemotherapeutic that links paclitaxel, the active ingredient in Taxol(R), to a biodegradable polyglutamate polymer, which results in a new chemical entity
• Use: treatment of advanced non-small cell lung cancer in women
• Dosage form: IV infusion
• Link to press releases:

o Cell Therapeutics, Inc. Press Release 4/07

o News release 1/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

Ipilimumab by Medarex/BMS; NDA likely to be submitted in 2009
• Class/MOA: fully human antibody against human CTLA-4
• Use: metastatic melanoma as a second-line monotherapy treatment, as a first-line treatment in combination with dacarbazine, and as a second-line treatment in combination with a melanoma-peptide vaccine
• Dosage form: IV infusion
• Link to press releases:

o News article 6/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

Ofatumumab (HuMax-CD20) by Genmab; last patient in pivotal Phase III trial analysis completed treatment
• Class/MOA: fully human, high-affinity antibody targeted at the CD20 molecule in the cell membrane of B-cells
• Use: refractory chronic lymphocytic leukemia (CLL), Non-hodgkins lymphoma, RA
• Dosage form: IV infusion
• Link to press releases:

o News article 5/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

Onconase (ranpirnase) by Alfacell; NDA submission estimated to be completed by end of 2008; fails to meet significance of survival in malignant mesothelioma 6/08
• Class/MOA: first-in-class product candidate based on Alfacell's proprietary ribonuclease (RNase) technology, triggering apoptosis
• Use: Malignant Mesothelioma
• Dosage form: IV
• Link to press releases:

o News article 6/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

Davanat (mannose and galactose) by Pro-Pharmaceuticals; NDA submitted in 5/08
• Class/MOA: polysaccharide polymer which binds to galectins that are over-expressed on cancer cells
• Use: targeted delivery of 5-FU in colorectal cancer
• Dosage form: IV
• Link to press releases:

o Pro-Pharmaceuticals press release 5/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

Mifamurtide by IDM Pharma; NDA is expected to be amended in first half of 2008
• Class/MOA: activates macrophages in vivo in order to increase their capacity to destroy cancer cells
• Use: osteosarcoma
• Dosage form: IV
• Link to press releases:

o News release 7/07

Gastrointestinal:

Gattex (teduglutide) by NPS; in Phase III; approval expected in 2009
• Class/MOA: glucagon-like peptide 2 (GLP-2) analog
• Use: Short bowel syndrome (SBS), gastrointestinal mucositis
• Dosage form: SC injection
• Link to press releases:

o News article 5/08

o News release 3/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

Respiratory diseases:

Bronchitol (in mannitol) by Pharmaxis; designated as an orphan drug 7/05; approval expected in 2009
• Class/MOA: increases mucous clearance
• Use: CF and COPD (bronchiectasis and chronic bronchitis)
• Dosage form: dry powder inhaler
• Link to press releases:

o Pharmaxis press release 6/08
o Pharmaxis press release 1/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

Tobramycin inhalation powder (TIP) by Nektar/Novartis; in Phase III; expected approval in 2009
• Class/MOA: aminoglycoside
• Use: CF
• Dosage form: dry powder inhaler
• Link to press releases:

o Nektar pipeline 2008

• Link to clinicaltrials.gov:

o Trials registered with NIH

Rheumatoid arthritis:

Golimumab by Centocor and Shering-Plough, BLA filed in 6/08
• Class/MOA: human, monoclonal antibody
• Use: treatment of RA, psoriatic arthritis, and ankylosing spondylitis
• Dosage form: SC injection
• Link to press releases:

o Centocor Press Release 6/08
o Centocor Press Release 6/08
o Centocor Press Release 3/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

Hepatorenal syndrome:
Lucassin (terlipressin) by Orphan Therapeutics; began rolling NDA process 5/08
• Class/MOA: synthetic vasopressin analogue that acts via the vasopressin V1 receptor as a systemic vasoconstrictor
• Use: treatment of hepatorenal syndrome (HRS) type 1 in patients with late-stage liver cirrhosis
• Dosage form: IV
• Link to press releases:

o News article 5/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

Long-term pipeline (2010 and beyond)

Systemic lupus erythematosus (SLE or lupus):

LymphoStat-B (belimumab)by CAT/HGS/GSK; expected BLA filing in early 2010, approval estimated for 2010
• Class/MOA: human monoclonal antibody that specifically recognizes and inhibits the biological activity of B-lymphocyte stimulator, or BLyS(R)
• Use: SLE
• Dosage form: IV
• Link to press releases:

o HGS press release 6/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

Atacicept by Zymogenics/Merck Serono; initiated second phase 2/3 study
• Class/MOA: TACI receptor that binds to the cytokines BLyS and APRIL.; these cytokines are members of the tumor necrosis factor family that promote B-cell survival and autoantibody production
• Use: systemic lupus erythematosus (SLE)
• Dosage form: SQ
• Link to press releases:

o Press release 6/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

Epratuzumab by UCB Pharma
• Class/MOA: humanized monoclonal antibody that targets the CD22 antigen found on B-cells
• Use: SLE
• Dosage form: IV
• Link to press releases:

o News release 8/07

• Link to clinicaltrials.gov:

o Trials registered with NIH

Rheumatoid arthritis:

SCIO469 by Scios; currently in Phase II trials
• Class/MOA: first-generation oral p38 MAP kinase inhibitor
• Use: RA
• Dosage form: oral
• Link to press releases:

o Scios press release 1/03

• Link to clinicaltrials.gov:

o Trials registered with NIH

Osteoporosis/hypoparathyroidism:

Preos (recombinant parathyroid hormone) by NPS; currently in Phase II-III trials; FDA issued approvable letter for osteoporosis in 3/06
• Class/MOA: parathyroid hormone
• Use: postmenopausal osteoporosis, hypoparathyroidism
• Dosage form: SC injection
• Link to press releases:

o NPS press release 9/07
o NPS press release 3/06

• Link to clinicaltrials.gov:

o Phase III trials registered with NIH

Cushing’s disease/acromegaly treatment:

Pasireotide by Novartis, filing of BLA planned in 2009
• Class/MOA: somatostatin analogue
• Use: treatment of cushing’s disease and acromegaly
• Dosage form: SC injection
• Link to clinicaltrials.gov:

o Trials registered with NIH

Hepatitis:

Albuferon® (albinterferon alfa-2b) by Human Genome Sciences and Novartis, with filing of BLA by Fall 2009
• Class/MOA: long-acting form of interferon alpha
• Use: treatment of chronic hepatitis C virus (HCV) infection
• Dosage form: SC injection
• Link to press release:

o Human Genome Sciences Press Release 5/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

Boceprevir by Schering-Plough; 2010
• Class/MOA: hepatitis C protease inhibitor
• Use: Hepatitis C
• Dosage form: oral
• Link to press releases:

o Schering-Plough press release 5/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

Celgosivir by Miginex; In Phase II studies, approximate approval 2010
• Class/MOA: alpha-glucosidase inhibitor
• Use: Hepatitis C to be used in combination with pegylated interferon and ribavirin
• Dosage form: oral
• Link to press releases:

o News article 11/06

• Link to clinicaltrials.gov:

o Trials registered with NIH

R1626 by Roche; in Phase II studies; approximate approval 2010
• Class/MOA: polymerase inhibitor
• Use: Hepatitis C
• Dosage form: oral
• Link to press releases:

o News article 4/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

Zadaxin (thymalfasin) by SciClone and Sigma-Tau
• Class/MOA: synthetic version of thymosin alpha 1, a substance found naturally in the circulation and produced in the body's thymus gland
• Use: Hepatitis C, Malignant melanoma
• Dosage form: SC injection, given 2x/week
• Link to press releases:

o News article 6/07

• Link to clinicaltrials.gov:

o Trials registered with NIH

Immunosuppressants/Transplantation:

Certican™ (everolimus) by Novartis, clinical trials ongoing
• Class/MOA: immunosuppressant; proliferation signal inhibitor
• Use: prevention of solid organ transplant rejection in combination with Neoral® (cyclosporine)
• Dosage form: oral agent
• Link to press release:

o Novartis Press release 7/03

• Link to clinicaltrials.gov:

o Trials registered with NIH

Anti-T-lymphocyte immune globulin (ATG) by Biotest, BLA filing expected early 2009
• Class/MOA: Anti-T-Lymphocyte Immune Globulin (rabbit)
• Use: prevention of graft-versus-host disease in lung transplantation
• Dosage form: IV infusion
• Link to press release: none
• Link to clinicaltrials.gov:

o Trials registered with NIH

Inhaled cyclosporine by APT Pharmaceuticals and Novartis, additional data gathered in response to FDA approvable letter in 6/05
• Class/MOA: immunosuppressant
• Use: prevention of graft-versus-host disease in lung transplantation
• Dosage form: inhalation
• Link to press release:

o APT press release 6/07

• Link to clinicaltrials.gov:

o Trial registered with NIH

Hereditary angioedema (HAE) treatment:

Rhucin (C1 esterase inhibitor) by Pharming; in Phase III
• Class/MOA: c1 esterace inhibitor
• Use: hereditary angioedema (HAE)
• Dosage form: IV injection
• Link to press releases:

o Pharming press release 6/08

• Link to clinicaltrials.gov:

o Trial registered with NIH

Gaucher disease:

Velaglucerase alfa by Shire; in Phase III, BLA filing expected in late 2009
• Class/MOA: glucocerebrosidase replacement
• Use: type 1 Gaucher disease
• Dosage form: IV injection
• Link to clinicaltrials.gov:

o Trials registered with NIH

Muckle-Wells Syndrome (MWS):

ACZ885 by Novartis; in Phase III, BLA filing expected in 2009 (studies are being conducted in other indications)
• Class/MOA: inhibits IL-1
• Use: Muckle-Wells syndrome
• Dosage form: SC injection
• Link to clinicaltrials.gov:

o Phase III trial registered with NIH

Multiple Sclerosis:

Fingolimod by Novartis, NDA filing by end of 2009
• Class/MOA: Immunomodulator
• Use: treatment of relapsing-remitting MS
• Dosage form: oral agent
• Link to press release:

o Novartis Press release 4/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

Teriflunomide by Sanofi-Aventis, submission scheduled for 2012
• Class/MOA: prevent the interaction of cells in the immune system involved in damage to the myelin sheath
• Use: treatment of relapsing-remitting MS
• Dosage form: oral agent
• Link to press release:

o News article 9/07

• Link to clinicaltrials.gov:

o Phase III Study vs Placebo in Patients With First Clinical Symptom of MS
o Phase III study reducing the Frequency of Relapses and Accumulation of Disability in Patients With MS

Laquinimod by Teva and Active Biotech, in Phase III trials
• Class/MOA: immunomodulator
• Use: treatment of relapsing-remitting MS
• Dosage form: oral agent
• Link to press release:

o Active Biotech Pipeline

• Link to clinicaltrials.gov:

o Trials registered with NIH

PAH:

Thelin™ (sitaxsentan) by Encysive Pharmaceuticals, planning additional Phase III study
• Class/MOA: sulfonamide class endothelin receptor antagonist (ERA)
• Use: treatment of pulmonary arterial hypertension (PAH)
• Dosage form: oral agent
• Link to press release:

o Encysive Pharmaceuticals Press release 6/07

• Link to clinicaltrials.gov:

o Phase III Safety and Efficacy Study in PAH

Cardiovascular disease/dyslipidemia:

Mipomersen by Genzyme and Isis, in Phase II-III studies, expected filing in 2010
• Class/MOA: antisense drug that reduces the production of apoB-100 protein
• Use: familial hypercholesterolemia
• Dosage form: SC injection
• Link to press release:

o Isis Press release 4/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

Cancer treatment:

Arenegyr (NGR-hTNF) by MolMed S.p.A; in Phase II
• Class/MOA: tumour homing peptide (NGR) selectively binding tumour blood vessels, fused to the powerful anticancer cytokine hTNF
• Use: Mesothelioma and Colorectal Cancer
• Dosage form: IV
• Link to press release:

o News article 5/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

Omnitarg (Pertuzumab) by Roche; in Phase III
• Class/MOA: humanized monoclonal antibody designed to bind to the HER2 receptor and inhibit the ability of HER2 to interact with other HER family members (HER1, HER2, HER3, and HER4) on the surface of cancer cells
• Use: Breast cancer in combination with Herceptin
• Dosage form: IV
• Link to press release:

o News article 6/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

Atamestane by Intarcia Therapeutics; Phase III studies ongoing
• Class/MOA: combined anti-estrogen therapy
• Use: first-line treatment of hormone-dependent breast cancer in combination with estrogen receptor blocker Fareston® (toremifene)
• Dosage form: oral agent
• Link to press release:

o News article 2/05

• Link to clinicaltrials.gov:

o Trials registered with NIH

Virulizin® by Lorus Therapeutics, orphan drug with fast-track status
• Class/MOA: immunotherapy that stimulates a patient's innate immune system through the activation of macrophages and the nfiltration of NK cells into tumors
• Use: first-line treatment of advanced pancreatic cancer in combination with Gemzar® (gemcitabine)
• Dosage form: IM injection
• Link to press release:

o News article 4/08
o News article 10/05

• Link to clinicaltrials.gov:

o Phase III efficacy study, given in Combination With Standard Chemo for Pancreatic Cancer

Voraxaze™ (glucarpidase) by Protherics PLC, BLA as a rolling submission starting in the first half of 2008; available through an expanded access
Program
• Class/MOA: recombinant enzyme
• Use: adjunctive therapy for cancer patients undergoing chemotherapy who are at risk for methotrexate toxicity
• Dosage form: IV infusion
• Link to press release:

o Protherics Press release 4/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

Saforis™ (glutamine in UpTec™) by MGA Pharma, FDA requested additional Phase III study; NDA submitted 2Q06
• Class/MOA: patented, topical, oral suspension of glutamine
• Use: prevention and treatment of chemotherapy-induced oral mucositis
• Dosage form: oral agent
• Link to press release:

o MGI Pharma Press release 10/06

• Link to clinicaltrials.gov:

o Phase III trial treating Mucositis Caused by Radiation Therapy in Patients With Newly Diagnosed Cancer of the Mouth or Throat

Larotaxel by Sanofi-Aventis, NDA filing planned for 2009
• Class/MOA: second-generation taxoid
• Use: 2nd line treatment of pancreatic cancer
• Dosage form: IV infusion
• Link to press release:

o Sanofi-Aventis Press Release9/07

• Link to clinicaltrials.gov:

o Trials registered with NIH

Phenoxodiol by Marshall Edwards, granted fast-track status
• Class/MOA: selectively limits plasma membrane electron transport in cancer cells, by binding to a cancer specific surface plasma membrane electron transport element on cancer cells thereby inhibiting their proliferation
• Use: treatment of HRPC in Taxotere® (docetaxel) nonresponders and recurrent chemotherapy-resistant, late-stage ovarian cancer
• Dosage form: IV infusion
• Link to press release:

o News article 5/07

• Link to clinicaltrials.gov:

o Phase III OVATURE Study: Weekly Carboplatin With and Without Phenoxodiol in Patients With Platinum-Resistant, Recurrent Epithelial Ovarian Cancer

Genasense (oblimersen sodium) by Genta Incorporated; FDA stated that available data are not adequate to support approval (3/08)
• Class/MOA: inhibits production of Bcl-2
• Use: relapsed or refractory chronic lymphocytic leukemia (CLL)
• Dosage form: IV
• Link to press releases:

o News release 3/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

Omacetaxine mepesuccinate by ChemGenex Pharmaceuticals; Fast Track designation granted 11/06 enables ChemGenex to file the NDA on a rolling basis as data become available (rolling NDA planned for mid-2008)
• Class/MOA: induces apoptosis by inhibition of protein synthesis, particularly Mcl-1
• Use: Chronic Myelogenous Leukemia
• Dosage form: SC injection
• Link to press releases:

o News release 4/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

Eribulin mesylate (E7389) by Company: Eisai Co., Ltd.; plans to submit an NDA to FDA in fiscal year 2009-2010
• Class/MOA: suppresses the growth of microtubule which is involved in various cellular processes in the body, such as cell division; synthetic analog of halichondrin B
• Use: third-line treatment of advanced breast cancer in patients who were pretreated with anthracycline, taxane and capecitabine.
• Dosage form:
• Link to press releases:

o News release 2/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

Sarasar (lonafarnib) by Schering-Plough; in Phase II, expected NDA submission 2009
• Class/MOA: farnesyl transferase inhibitor
• Use: Breast cancer, solid tumors
• Dosage form: oral
• Link to press releases:

o Schering Plough pipeline 2/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

Palifosfamide (ZIO-201) by Ziopharm; received orphan drug status for soft-tissue sarcoma (SAS); expected approval 2010
• Class/MOA: bi-functional alkylator that causes irreparable inter-strand DNA cross-linking, resulting in cell death
• Use: SAS
• Dosage form: IV injection (Phase II); oral form expected to go into Phase I in 2009
• Link to press releases:

o News article 5/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

Atiprimod by Callisto; Phase II; given orphan drug designation to treat carcinoid cancer
• Class/MOA: small molecule drug that displays a multiplicity of anti-cancer mechanistic activities
• Use: low to intermediate grade neuroendocrine carcinoma (advanced carcinoid cancer).
• Dosage form: oral
• Link to press releases:

o Callisto press release 5/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

Custirsen (OGX-011) by OncoGenex; in Phase II studies, expected approval >2010
• Class/MOA: block production of clusterin, a cell survival protein that is over-produced in several cancer indications and in response to many cancer treatments
• Use: metastatic hormone refractory prostate cancer
• Dosage form: IV injection
• Link to press releases:

o OncoGenex press release 2/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

AZD6244 by Array BioPharma/AstraZeneca; in Phase II trials, expected approval >2010
• Class/MOA: small molecule inhibitor that targets a key position in the Ras/Raf/MEK/ERK signalling pathway
• Use: advanced melanoma
• Dosage form: oral
• Link to press releases:

o News article 12/07

• Link to clinicaltrials.gov:

o Trials registered with NIH

Elesclomol (formerly STA-4783) by GlaxoSmithKline/Synta Pharmaceuticals; in Phase II, expected approval >2010
• Class/MOA: increasing the level of oxidative stress in cancer cells even further, beyond sustainable levels, inducing apoptosis
• Use: metastatic melanoma
• Dosage form: injectable
• Link to press releases:

o News article 5/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

Telintra by Telik; in Phase II, expected approval >2010
• Class/MOA: stimulate the production of blood cells in the bone marrow.
• Use: myelodysplastic syndrome (MDS) and in patients at risk for chemotherapy-induced neutropenia (CIN).
• Dosage form: oral
• Link to press releases:

o News article 5/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

BiovaxID by Biovest/Accentia; in Phase III
• Class/MOA: autologous tumor derived immunoglobulin idiotype vaccine
• Use: non-Hodgkin’s lymphoma
• Dosage form: SC injection
• Link to press releases:

o Accentia press release 6/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

Chemophase (recombinant human hyaluronidase) by Halozyme; in Phase I/II trials
• Class/MOA: hyaluronidase enzyme targeting extracellular matrix
• Use: chemoadjuvant in superficial bladder cancer
• Dosage form: intravesical instillation
• Link to press releases:

o Halozyme press release 6/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

Dimericine by AGI Dermatics; Fast track designation in 8/07, in phase III trials
• Class/MOA: DNA repair enzyme T4 endonuclease V in a liposome
• Use: solar and ultraviolet photosensitivity in patients with Xeroderma Pigmentosum (XP)
• Dosage form: topical lotion
• Link to press releases:

o AGI Dermatics press release 8/07

• Link to clinicaltrials.gov:

o Trials registered with NIH

Blood disorders:

Injectafer (ferric carboxymaltose) Injection by Luitpold Pharmaceuticals, Inc.
• Use: Iron deficiency anemia
• Dosage form: IV
• Link to press releases:

o News release 5/07

• Link to clinicaltrials.gov:

o Trials registered with NIH

CNS/Alzheimer's disease:

Bapineuzumab by Elan and Wyeth; in Phase II trials
• Class/MOA: human monoclonal antibody which binds to and clears beta amyloid peptide by generating antibodies
• Use: mild to moderate Alzheimer's disease
• Dosage form: SC injection
• Link to press releases:

o News article 6/08

• Link to clinicaltrials.gov:

o Trials registered with NIH

Note: Dates are estimated based on information provided by the published sources

Sources:

Drugs.com. Accessed at: New Drug Applications

Biotech100.com. Accessed at: Pipeline Drugs -- Biotechnology Drugs in the Pipeline

U.S. Food and Drug Administration. Accessed at: Drugs@FDA

U.S. National Institutes of Health. Accessed at: ClinicalTrials.gov

Walgreens Specialty. Accessed at: Pipeline Report 2Q08