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Apogenix GmbH
www.apogenix.com
(Apocept) APG101 |
Apogenix is a clinical stage biopharmaceutical company developing novel protein therapeutics for cancer treatment. The company's therapeutic candidates target the apoptotic pathway or tumor cell growth and migration. The lead product candidate, Apocept™ (APG101), is a CD95-Fc fusion protein therapeutic that blocks the interaction between CD95 and its ligand CD95L and therefore inhibits tumor cell growth. Apogenix recently announced that Apocept has reached all clinical endpoints in an actively controlled Phase 2 proof-of-concept study in patients with recurrent glioblastoma multiforme (GBM). The primary endpoint was progression free survival (PFS) at six months. Achieved secondary endpoints include PFS, overall survival (OS), safety and tolerability. The robust efficacy was combined with an excellent safety profile; no drug-related serious adverse events have been observed in any clinical studies conducted with Apocept. The current standard of care - which includes surgery, radiation and alkylating chemotherapy - is only moderately effective. Apocept is the first compound in more than 15 years to clearly deliver a PFS as well as an OS benefit in an actively controlled trial. Patients with GBM overexpressing a newly identified prognostic and therapeutic biomarker showed even more pronounced benefits. The inhibition of CD95L by Apocept holds great promise in other tumor indications such as, e.g.,myelodysplastic syndromes and pancreatic cancer. Apogenix was founded as a spin-out from the German Cancer Research Center (DKFZ). Since its inception in the fall of 2005, the company has raised more than €50 million in three financing rounds; the main investor is dievini Hopp BioTech Holding. In addition, the company has been awarded public funds totaling nearly €8.5 million. Apogenix is based in Heidelberg, Germany, and employs approximately 25 people. |
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BIND Biosciences
www.bindbio.com
BIND-014 |
BIND Biosciences is a clinical-stage company developing a new class of therapeutics, called Accurins, which are designed to create better patient outcomes by targeting therapeutic agents directly to areas of disease in concentrations unachievable by conventional approaches. Accurins are precisely engineered to accumulate and release their active agent in the target tissues thereby maximizing therapeutic activity while at the same time reducing toxicities associated with systemic delivery. In preclinical models, BIND's Accurin technology yields up to 10-fold higher drug concentrations at the site of disease resulting in significantly greater efficacy. Accurins can be developed both with already-approved active agents and with compounds currently in development to produce best-in-class drugs. BIND's most advanced product, BIND-014, is a targeted docetaxel Accurin currently showing promising results in a Phase 1 clinical trial in patients with advanced and metastatic cancers. BIND expects to initiate Phase 2 trials with BIND-014 in late 2012 in four important solid tumor indications. |
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Ceres Oncology
www.ceresoncology.com
VGX-100 |
Ceres Pty Ld is the newly created 100% subsidiary of Circadian Technologies Limited and has been granted exclusive rights to develop VGX-100 for oncology therapeutic applications. The company was formed on 4 September 2012 and became fully operational on 1 November 2012. VGX-100 is currently in Phase 1 clinical trials in the United States. |
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Cerulean
http://ceruleanrx.com
CRLX101 |
Cerulean (www.ceruleanrx.com) is the leading nanopharmaceutical company. We aspire to win the fight against cancer with dynamically tumor-targeted nanopharmaceuticals, which use the large pores of tumor blood vessels as entry portals to provide sustained drug release inside tumor cells. Dynamic tumor targeting results in high intracellular drug concentrations in tumors while sparing the rest of the body. Because of their tolerability, our nanopharmaceuticals facilitate a new cancer treatment paradigm in which incurable cancers are transformed into manageable chronic diseases treated with combinations of highly efficacious yet highly tolerable agents, providing patients hope for extended overall survival and improved quality of life. Our lead candidate, CRLX101, is a potent inhibitor of topoisomerase-1 and HIF-1α. We completed enrollment of a randomized 157-patient Phase 2b trial in non-small cell lung cancer with overall survival as the primary endpoint. Phase 2b data was expected in 4Q2012, but patients are living longer than expected, so the efficacy readout is delayed to 1Q2013. The second candidate from our platform, CRLX301, is a potent tubulin inhibitor. CRLX301 will enter the clinic in 2Q2013. Finally, our siRNA delivery program has the potential to unlock the enormous potential of RNAi by using nanoparticles to solve the RNAi delivery problem. |
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EnGeneIC
www.engeneic.com
EDV technology |
EnGeneIC's mission is to change the perception of cancer from life threatening to curable. We are a privately held Australian company with proprietary technology based on bacterial minicells. These very small, 400 nanometre particles (EnGeneIC Delivery Vehicles or EDVs), are capable of carrying a payload of anticancer drugs and other therapeutic molecules such as siRNA/microRNA and plasmids and are targeted to tumour cells using antibodies. |
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Jennerex Biotherapeutics
www.jennerex.com/
JX-929 |
Jennerex, Inc. is a clinical-stage biotherapeutics company focused on the development and commercialization of first-in-class, breakthrough targeted oncolytic immunotherapy products for cancer. The Company's lead product Pexa-Vec (JX-594) is currently in an international, randomized Phase 2b clinical trial (TRAVERSE) in patients with advanced primary liver cancer who have failed sorafenib therapy. Pexa-Vec is also in a Phase 1/2 clinical trials in patients with treatment-refractory colorectal cancer. Published studies designed to establish optimal dose levels and the safety profile of Pexa-Vec have shown its ability to selectively target and cause destruction of a variety of common solid tumor types and trigger a potent immune response. Pexa-Vec and other product candidates under development are designed to attack cancer tumors through three diverse mechanisms of action: the lysis of cancer cells through targeted viral replication, the ablation of the blood supply to tumors and the stimulation of the body's immune response against the cancer. Jennerex is headquartered in San Francisco and has related research and development operations in Ottawa, Canada and Pusan, South Korea. For more information about Jennerex, please visit www.jennerex.com. |
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KaloBios Pharmaceuticals, Inc.
www.kalobios.com
KB004 |
KaloBios is a biopharmaceutical company focused on the development of monoclonal antibody therapeutics. KaloBios has a portfolio of patient-targeted, first-in-class antibodies to treat serious medical conditions with a primary focus on respiratory diseases and cancer. In oncology, KaloBios is developing KB004, a Humaneered™, anti-EphA3 receptor tyrosine kinase monoclonal antibody. KB004 is currently in a Phase 1 clinical trial in patients with hematologic malignancies. KB004 is directed against EphA3, an oncofetal antigen involved in the positioning of cells during fetal development which is re-expressed on the surface of hematologic and solid tumor cells and the stem cell microenvironment, but not normal cells. As a result, KB004 may have the potential to disrupt the stem cell microenvironment, providing for long-term responses while sparing normal cells. KB004 is a high affinity, non-fucosylated antibody that can potentially kill tumor cells in three ways: (1) direct induction of programmed cell death (apoptosis); (2) enhanced antibody-dependent cell-mediated cytotoxicity (ADCC) activity; or (3) disruption of the tumor vasculature by binding to EphA3 on the endothelial cells that line the vasculature. KaloBios is currently enrolling patients in the Phase 1 dose escalation study. This will be followed by the expansion portion of the Phase 1 trial, which will pre-screen subjects for EphA3 expression and assess the safety and activity of KB004 at the selected dose. |
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Macrogenics
www.macrogenics.com
MGAH22 |
MacroGenics is a private, venture-backed biotechnology company focused on the discovery, development and delivery to patients of novel biologics for treatment of cancer, autoimmune disorders and infectious diseases. Its two lead clinical programs are MGAH22 (anti-HER2) and MGA271 (anti-B7-H3), both oncology therapeutics being studied in Phase 1 clinical trials. MacroGenics has built a fully-integrated set of capabilities in antibody-based product development which supports its innovative pipeline of clinical stage product candidates. MacroGenics' proprietary research is based on three core technology platforms, which include: (1) a leading research capability for screening and targeting cancer stem-like cells; (2) Dual-Affinity Re-Targeting (or DART) bi-specific technology, which allows the incorporation of multiple specificities within a single recombinant molecule; and (3) Fc optimization, which enhances antibody-dependent effector cell function. The company has multiple research and development collaborations with major pharmaceutical companies including Boehringer Ingelheim, Les Laboratoires Servier and Pfizer, Inc. For more information, visit www.macrogenics.com. |
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MEI Pharma
www.meipharma.com/
ME-344 |
MEI Pharma, Inc. (Nasdaq: MEIP) is a San Diego-based oncology company focused on the clinical development of novel therapies for cancer. The Company's clinical development pipeline includes two novel drug candidates derived from its isoflavone-based technology platform, ME-143 and ME-344, and a potential best-in-class, oral histone deacetylase (HDAC) inhibitor, Pracinostat. Results from a Phase I dose-escalation trial of lead NADH oxidase inhibitor ME-143 in heavily treated patients with solid refractory tumors were presented at the American Society of Clinical Oncology Annual Meeting in June 2012. Results from a Phase I clinical trial of lead mitochondrial inhibitor ME-344 in patients with solid refractory tumors are expected in the second quarter of 2013. Pracinostat has been tested in more than 150 patients in multiple Phase I and exploratory Phase II clinical trials, including advanced hematologic disorders such as acute myeloid leukemia, myelodysplastic syndrome and myelofibrosis. For more information, go to www.meipharma.com. |
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Shape Pharmaceuticals
www.shapepharma.com/
SHP 141 |
Shape Pharmaceuticals, Inc. is a focused company founded by James Bradner, M.D., and Ralph Mazitschek, Ph.D., and funded by HealthCare Ventures. Shape's lead product, SHP-141, is a topical HDAC inhibitor in clinical development for CTCL and psoriasis. SHP-141 was rationally designed as a "soft-drug" to act locally on CTCL and psoriasis lesions and to avoid the systemic toxicities associated with the currently-marketed HDAC inhibitors. The development of SHP-141 is being supported through an alliance with the Leukemia & Lymphoma Society. |
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Addex Therapeutics
www.addextherapeutics.com
Dipraglurant |
Addex Therapeutics is pioneering discovery and development of an emerging class of oral small molecule drugs, called allosteric modulators, which can be exquisitely selective for their intended target and confer significant therapeutic advantages over conventional “orthosteric” small molecule or biological drugs. The Company uses its proprietary discovery platform to address receptors and other proteins that have been undruggable for conventional drug discovery methods, including G-Protein Coupled Receptors (GPCRs), receptor tyrosine kinases (RTKs) and cytokine receptors, such as the TNF receptor superfamily. Many such targets have been widely recognized as attractive for modulation of important diseases with unmet medical needs, but have remained inaccessible to small molecule drug discovery. |
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Afferent Pharmaceuticals
www.afferentpharma.com/
AF-219 |
Afferent Pharmaceuticals is a clinical-stage biopharmaceutical company developing first-in-class, small molecule compounds that target P2X3 receptors for the treatment of chronic pain and other disorders. Afferent's lead P2X3 antagonist, AF-219, is currently in Phase 2 studies of osteoarthritis, interstitial cystitis/bladder pain syndrome, and idiopathic chronic cough.P2X3 receptors are preclinically well-validated targets, highly specific to unmyelinated, C fiber afferent nerves that have dense innervations in visceral organs, skin and joints. These small diameter fibers transmit sensations of pain and irritation via mechanisms that include ATP signaling (ATP is the endogenous ligand at P2X3 receptors). P2X3-containing fibers have been broadly implicated for treating chronic inflammatory, visceral and neuropathic pain, as well as migraine and cancer pain. By blocking pain signals at their source, P2X3 antagonists have the potential to transform the treatment of chronic pain and address some of the most important unmet medical needs in chronic pain management. This highly specific and targeted approach may minimize the likelihood of adverse effects in the brain or cardiovascular tissues, thereby providing a safer treatment option over existing, chronic pain therapies. |
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Allon Therapeutics
www.allontherapeutics.com
davunetide for Progressive Supranuclear Palsy |
Allon Therapeutics Inc. is a clinical-stage biotechnology company focused on bringing to market innovative central nervous system therapies. Allon's lead drug, davunetide, is proceeding in a fully enrolled pivotal Phase 2/3 clinical trial in an orphan indication, progressive supranuclear palsy (PSP), under a Special Protocol Assessment (SPA) with the Food and Drug Administration (FDA). This pivotal trial is based upon statistically significant human efficacy demonstrated in amnestic mild cognitive impairment, cognitive impairment associated with schizophrenia, and positive biomarker data. |
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BioAxone BioSciences Inc.
www.bioaxonebio.com
Cethrin for spinal cord injury |
BioAxone Biosciences Inc. is a privately owned US corporation with a late-stage clinical product and a focus on specialty pharmaceuticals for unmet medical needs. BioAxone has developed CethrinTM a Phase II drug to treat spinal cord injury. Cethrin is the most advanced drug in clinical development to treat spinal cord injury and is poised to be first to market for this unmet need. The company's second compound, BA-1049, is a small molecule Rho kinase inhibitor in preclinical development to treat glaucoma with a new mechanism of action. BioAxone owns significant intellectual property protecting Cethrin, BA-1049 and the Rho signaling area, with the key composition of matter patents issued in USA and Europe, and various medical use patents for further protection. Dr. McKerracher, Founder and CEO of BioAxone, led Cethrin's GMP manufacturing and clinical development until the program was licensed in 2007, and continued to oversee the program as a Board Member. Dr. McKerracher recaptured the program in 2011 in a unique business deal with the licensee and previous investors. From her experience in developing Cethrin, Dr. McKerracher has deep know-how to guide overall project management and ensure the clinical program meets its objectives on-time and on-budget. The company is seeking financing and a development partnership to accelerate commercialization of Cethrin and advance development of BA-1049. BioAxone is currently privately financed by the Founder and one Investor, and is in term sheet negotiation with one potential partner. |
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Cynapsus Therapeutics Inc.
www.cynapsus.ca
APL-130277 |
Cynapsus is a specialty pharmaceutical company developing the only oral (sublingual) delivery of the only approved drug (apomorphine) to treat the motor symptoms of Parkinson's disease. Over one million people in the United States and an estimated 5 million people globally suffer from Parkinson's disease. Parkinson's disease is a chronic and progressive neurodegenerative disease that impacts motor activity, and its prevalence is increasing with the aging of the population. Despite the use of very effective medications, motor symptoms appear to increase with the progression of Parkinson's disease. A rather debilitating collection of symptoms known as OFF episodes (motor freezing and impaired speech capabilities) can occur in up to 50% of patients. Cynapsus' lead drug candidate, APL-130277, is an easy-to-administer, fast-acting and oral reformulation of apomorphine. Apomorphine is currently only available as an injection, used to rescue patients from OFF episodes. Cynapsus is focused on rapidly maximizing the value of APL-130277 by completing clinical studies in advance of a New Drug Application expected to be submitted in 2015. The Company anticipates out-licensing to an appropriate global pharmaceutical partner before such an application is submitted. As a focused single project company it could also be the subject of an acquisition. |
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EnVivo Pharmaceuticals
www.envivopharma.com
EVP-6124 |
EnVivo Pharmaceuticals is a biotech company dedicated to discovering and developing small molecule therapeutics for disorders of the central nervous system (CNS). The company's focus is on building an integrated company and it is working to convert its broad pipeline into a range of CNS therapies that leverage novel mechanisms of action by altering the progression of disease and providing improvement in cognitive and overall function. EnVivo's lead product is an alpha-7 nicotinic acetylcholine receptor agonist that has successfully completed Phase 2b clinical trials in both schizophrenia and Alzheimer's disease. EnVivo's other development programs include an epigenetics program based on Histone Deacetylase inhibition (HDACi), a Gamma Secretase Modulator program and a potent and selective PDE10 inhibitor program. |
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Galenea Corp
www.galenea.com
MANTRA |
Galenea is a leader in the rapidly emerging field of synaptic transmission drug discovery. Synaptic transmission is the process by which neurons communicate with each other, and dysfunctions in this process are now known to play a central role in many psychiatric and neurological diseases. Modulators of synaptic transmission therefore have the potential to yield breakthrough treatments. Galenea has developed an innovative synaptic transmission drug discovery platform that integrates three components: the MANTRATM system, a proprietary, high throughput screening technology, to identify a new generation of small molecule modulators of synaptic transmission; preclinical network measures employing in vitro brain slice EEG assays, coupled with in vivo models using integrated EEG measures of animal behavior to more reliably determine CNS drug efficacy; and human EEG biomarkers, developed in tandem with and informed by our animal EEG data to greatly enhance CNS drug development. The company is advancing a novel pro-cognitive program derived from the platform; and has partnerships applying the platform to Huntington's disease and neurodegeneration. Galenea has also assembled a compelling scientific team, balancing academic aptitude with industry experience and entrepreneurship. For more information about Galenea, please visit the company's website at www.galenea.com. |
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Naurex
www.naurex.com
GlyX13/NRX-1074 |
Naurex Inc. is developing novel therapies for depression and other CNS disorders based on a new mechanism of action for modulating the NMDA receptor. Naurex researchers have generated novel chemical drug classes and a platform for discovering molecules that are partial agonists of the NMDAR. These partial agonists modulate the NMDAR in a different way than existing agents, exhibiting the remarkable efficacy potential of drugs such as ketamine, but without their prohibitive side effects. Naurex's lead drug candidate, GLYX-13, is in Phase II development as a therapy for treatment-resistant depression. GLYX-13 has exhibited rapid onset and long duration of effect with good safety. Other compounds from Naurex's novel chemistry platform have high potential for the treatment of mood, anxiety, cognitive, developmental, pain, and other CNS disorders. |
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Neuren Pharmaceuticals
www.neurenpharma.com
NNZ-2566 |
Neuren Pharmaceuticals is a New Zealand incorporated biopharmaceutical company with a well-differentiated pipeline built on a robust intellectual property portfolio in neurology and endocrinology. Clinical development, medical affairs and business development functions are all managed from the US. The company's lead clinical-stage compound is NNZ-2566, a synthetic analog of the n-terminal tripeptide of IGF-1, a neurotrophic and neuroprotective molecule naturally cleaved from endogenously expressed and exogenously administered IGF-1 both in the brain and in peripheral circulation. NNZ-2566 readily crosses the blood brain barrier and is also orally available. The intravenous form of the compound is currently in a Phase II clinical trial in patients with acute, moderate to severe traumatic brain injury. Two additional Phase II trials with the oral formulation - one in concussion or mild TBI and one in Rett Syndrome - are being initiated and will be fully operational in early 2013. The company has developed key collaborative relationships with the U.S. Army Walter Reed Army Institute of Research, the International Rett Syndrome Foundation, Autism Speaks, Autism Therapeutics Ltd. and the Blue Bird Circle Rett Center at Texas Children's Hospital/Baylor College of Medicine. Neuren is listed on the Australian Securities Exchange (ASX:NEU). |
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Zalicus
www.zalicus.com
Z160 |
Zalicus Inc. (Nasdaq: ZLCS) is a biopharmaceutical company that discovers and develops novel treatments for patients suffering from pain and immuno-inflammatory diseases. Zalicus has a portfolio of proprietary clinical-stage product candidates targeting pain and immuno-inflammatory diseases, including Z160 and Z944, and have entered into multiple revenue-generating collaborations with large pharmaceutical companies relating to other products, product candidates and drug discovery technologies. Zalicus applies its expertise in the discovery and development of selective ion channel modulators and its combination high throughput screening capabilities to discover innovative therapeutics for itself and its collaborators in the areas of pain, inflammation, oncology and infectious disease. To learn more about Zalicus, please visit www.zalicus.com.
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Ambit Biosciences Corporation
www.ambitbio.com
AC430 |
Ambit Biosciences is a privately held biopharmaceutical company engaged in the development of a robust pipeline of small molecule kinase inhibitors for the treatment of cancer, inflammatory disease and other indications. Ambit's lead compound, quizartinib (AC220), is a novel, potent, highly selective, orally bioavailable FMS-like tyrosine kinase-3 (FLT3) inhibitor, and is currently under clinical investigation in patients with relapsed or refractory AML. In addition to quizartinib, Ambit's clinical pipeline includes AC430, an oral JAK2 inhibitor, and CEP-32496, a BRAF inhibitor licensed to Teva. Ambit's preclinical portfolio includes a proprietary CSF1R inhibitor program. For more information, visit www.ambitbio.com. |
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Aquinox Pharmaceuticals Inc.
www.aqxpharma.com
AQX-1125 |
President & CEO , Aquinox Pharmaceuticals Inc. -Mr. Main has over 20 years experience in the pharmaceutical industry and brings a demonstrated ability to grow and finance pharmaceutical development companies and was a co-founder of Aquinox. Aquinox is the world leader in the discovery and development of novel, first-in-class, small molecule therapeutics targeting SHIP1 within the PI3K pathway for the treatment of inflammatory disease. Our lead clinical candidate, AQX-1125, is a highly active and selective small molecule allosteric activator of SHIP1 suitable for once-daily oral dosing as a pill. AQX-1125 recently met its primary endpoints in two Phase IIa proof-of-concept clinical studies (COPD, Asthma) and has demonstrated broad anti-inflammatory properties including in steroid-refractory indications. The company is currently planning additional Phase II clinical trials and is pursuing strategic partnerships. |
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Array BioPharma
www.arraybiopharma.com
ARRY 502 |
Array BioPharma Inc. is a biopharmaceutical company focused on the discovery, development and commercialization of targeted small-molecule drugs to treat patients afflicted with cancer and inflammatory diseases. Array is evolving into a late-stage development company, with two wholly-owned programs, ARRY-614 and ARRY-520, and three partnered programs, selumetinib partnered with AstraZeneca, MEK162 partnered with Novartis, and danoprevir, partnered with InterMune / Roche, having potential pivotal trial decisions by the end of calendar year 2013. For more information on Array, please go to www.arraybiopharma.com. |
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Avexxin AS
www.avexxin.com
AVX001 |
Avexxin is a pharmaceutical company focused on developing and commercializing novel small molecule therapeutics for patients with chronic inflammatory conditions. Avexxin's advanced understanding of the biology of the inflammatory process has resulted in a novel therapeutic approach for the treatment of psoriasis and other inflammatory disorders. |
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CoNCERT Pharmaceuticals, Inc.
www.concertpharma.com
CTP-499 |
Founded in 2006, Concert Pharmaceuticals is a clinical stage small molecule biotechnology company dedicated to creating differentiated new medicines through a novel scientific approach utilizing the safe, naturally-occurring element deuterium. Site-selective incorporation of deuterium has the unique ability to maintain the potency and selectivity of existing drugs, while in favorable cases improving important metabolic properties that have the potential to improve safety and tolerability characteristics. This approach provides the potential to substantially reduce the risk, timelines, and expense of drug research and development. Concert’s DCE Platform® (Deuterated Chemistry Entity) has led to a robust product pipeline with its most advanced compound, CTP-499, in Phase 2 clinical testing for diabetic kidney disease. The company has a broad research and development pipeline encompassing many therapeutic areas including renal disease, hematologic disorders and CNS disorders, among others. Since its inception, Concert has raised more than $110 million from venture capitalists, public institutional investors. In addition, the company has a broad patent estate and intellectual property position with approximately 26 issued US patents, as well as issued patents in Europe and Japan. Additional information about Concert is available online at www.concertpharma.com. |
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Kineta Inc.
www.kinetabio.com
ShK – 186 |
Kineta, Inc. is developing a pipeline of novel drug candidates designed to restore the immune system to normal function. With a focus in autoimmunity and viral disease, our new classes of therapeutic candidates are a response to the needs of millions of people facing disease around the world.
One of Kineta’s lead clinical programs is ShK-186 for autoimmune disease. ShK-186 is the first Kv1.3 potassium channel blocker to enter human clinical trials and is currently in phase one. With a novel mechanism that selectively targets effector memory T-cells, ShK-186 has been shown to significantly reverse clinical symptoms in animal models of multiple sclerosis and rheumatoid arthritis. It has also demonstrated a solid safety profile with very low side effects or immunosuppression in animals. Our near-term objectives are to demonstrate tolerability and efficacy in clinical trials.
Kineta is initially targeting ShK-186 for multiple sclerosis but is also exploring other autoimmune applications including Type 1 diabetes mellitus, lupus nephritis, rheumatoid arthritis, and psoriasis as well as smaller, orphan disease applications. |
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Selecta Biosciences
www.selectabio.com
nanoparticle vaccines and immunotherapies |
At Selecta Biosciences, we are developing an entirely new class of targeted immunotherapies that induce antigen-specific immune activation or antigen-specific immune tolerance. Our proprietary technology platform is based on innovations in nanotechnology and immunology discovered at MIT and Harvard Medical School. Selecta's platform is designed to create robust immune responses to a wide range of antigens while limiting adverse events. Unwanted auto-immune reactions are an important and growing health concern for which no or only symptomatic treatments exist. We are developing cutting edge immunotherapies that are rebalancing the immune response by inducing antigen-specific T regulatory cells and depleting T effector cells. Having achieved pre-clinical proof of concept, we are working with industry and academic partners to develop immune therapies in Type-1 diabetes, allergies and protein replacement. We are developing our novel targeted vaccines and immunotherapeutics for worldwide markets at our facilities in Watertown, Massachusetts, and Moscow, Russia. |
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Tigenix
www.tigenix.com
CX 621 |
TiGenix NV (Euronext Brussels: TIG) is a leading European cell therapy company with a marketed product for cartilage repair, ChondroCelect®,
and a strong pipeline with clinical stage allogeneic adult stem cell programs for the treatment of autoimmune and inflammatory diseases.
TiGenix is based out of Leuven (Belgium) and has operations in Madrid (Spain), and Sittard-Geleen (the Netherlands).
For more information please visit www.tigenix.com. |
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ViaCyte
www.viacyte.com
Encapsulated islet cell transplantation therapy |
ViaCyte is a private company focused on developing a novel cell therapy for the treatment of diabetes. The Company's technology is based on the production of pancreatic beta cell progenitors derived from human pluripotent stem cells. These cells are implanted using a durable and retrievable encapsulation device. Once implanted and matured, these cells secrete insulin and other regulatory factors in response to blood glucose levels. ViaCyte's goal is long term insulin independence without immune suppression, and without risk of hypoglycemia and other diabetes-related complications. ViaCyte is headquartered in San Diego, California with additional operations in Athens, Georgia. The Company is funded in part by the California Institute for Regenerative Medicine (CIRM) and JDRF. |
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Virobay, Inc.
www.virobayinc.com
cathepsin S inhibitor |
Virobay is a leader in the design, synthesis and development of small molecule inhibitors of cysteine proteases, a diverse class of enzyme proteases that are key mediators in a variety of diseases, including autoimmunity, neuropathic pain, liver disease, cancer, and cardiovascular disorders. Virobay was founded upon a rich industry legacy of intensive research and development focused on the cathepsin family of cysteine proteases. Today, Virobay possesses a trove of maturing assets, including an advancing clinical pipeline and well-characterized libraries of highly potent and selective inhibitors with drug-like pharmacokinetics consistent with the potential for oral, once-daily dosing in humans. Virobay's unique expertise in the structure-based design of this class of inhibitors includes extensive understanding of the properties of the cysteine protease active site, detailed knowledge of the cell biology of this enzyme family and a collection of pharmacodynamic biomarkers, as well as a deep understanding of the medicinal chemistry required to achieve superior levels of potency, pharmacokinetics, safety and specificity. Virobay's clinical pipeline currently includes product candidates in autoimmune disease, neuropathic pain and liver fibrosis. |
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Aastrom Biosciences, Inc.
www.aastrom.com
lxmyelocel-T |
Aastrom Biosciences is the leader in developing patient-specific, expanded multicellular therapies for use in the treatment of patients with severe, chronic cardiovascular diseases. The company's proprietary cell-processing technology enables the manufacture of ixmyelocel-T, a patient-specific multicellular therapy expanded from a patient's own bone marrow and delivered directly to damaged tissues. Aastrom has advanced ixmyelocel-T into late-stage clinical development, including a Phase 3 clinical program to study patients with critical limb ischemia and a planned Phase 2b clinical trial in patients with ischemic dilated cardiomyopathy. For more information, please visit Aastrom's website at www.aastrom.com. For more information on the pivotal REVIVE Phase 3 clinical trial, please visit the trial website at www.revivecli.com. |
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ARMGO Pharma, Inc.
www.armgo.com
Rycals |
ARMGO Pharma, Inc., is a privately held biopharmaceutical company whose small-molecule proprietary drugs, known as “Rycals”, are a new class of oral agents that act on a novel therapeutic target, the ryanodine receptor/calcium release channel (RyR) located on the sarcoplasmic/endoplasmic reticulum of the cell. Rycal compounds act to stabilize these channels and repair the calcium leak caused by stress associated with chronic and debilitating cardiac, muscular, and neurological diseases. ARMGO Pharma's lead program, ARM036, is in Phase 2 clinical studies for the treatment of heart failure and CPVT, an arrhythmogenic cardiomyopathy associated with mutations in RyR2. A second candidate, ARM210, has been selected for musculoskeletal disorders including Duchenne Muscular Dystrophy, and is currently in the pre-clinical phase. |
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Catabasis Pharmaceuticals, Inc.
www.catabasispharma.com
CAT 2000 |
Catabasis was formed in 2008 by industry veterans Jill Milne and Mike Jirousek around the idea of pathway pharmacology, an innovative approach to new drug development. Leveraging a proprietary technology platform, Catabasis seeks to develop pathway targeted molecules, which simultaneously modulate several targets on a particular pathway for a desired therapeutic effect. For each pathway, Catabasis identifies two safe and well-known agents that act on different aspects of the pathway, and then uses the proprietary SMART Linker platform to make a pathway targeted molecule. The SMART Linker platform conjugates the agents into a new chemical entity using a linker designed to be cleaved only by specific intracellular enzymes. The resulting conjugate is inactive until it enters the desired cells. This approach improves both the safety of the starting agents, and has demonstrated synergistic efficacy several of orders magnitude greater than simple combinations of the two agents.
CAT-2003, a conjugate of two well-known cardiovascular agents Niacin and Omega3 fatty acids, is Catabasis' second program. CAT2003 will enter Phase 1 testing for severe hypertriglyceridemia in Q4 of 2012. |
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Chanrx
www.bioenterprise.com/Companies/ChanRX-2
vanoxerine |
Venture backed ChanRx is pursuing a treatment for AF/AFL. A Phase IIb escalating dose trial for acute conversion of AF/AFL to sinus rhythm is commencing in November 2012. Vanoxerine has a long history of safety including 6 Phase I human trials by other companies. Vanoxerine does not prolong the QT interval in healthy human volunteers. ChanRx is the first to find an efficacious application of this drug which is not commercially available for any indication anywhere in the world. |
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Cortendo
www.cortendo.com
NormoCort (2S,4R ketoconazole or COR-003 |
Cortendo AB is a biopharmaceutical company headquartered in Göteborg, Sweden. Its stock is publicly traded on the NOTC-A-list (OTC) in Norway. Cortendo is a pioneer in the field of cortisol inhibition and has completed early clinical trials in patients with Type 2 diabetes. The development of the lead drug candidate NormoCort (COR003), the 2S,4R-enantiomer of ketoconazole, has been re-directed to Cushing's Syndrome. The company primarily focuses its resources where the path to commercialization or partnerships is clear and relatively near-term. Strategically, Cortendo's business model is to commercialize relevant opportunities in the United States while partnering its assets ex-US. Backed by a highly experienced leadership team Cortendo has plans to continue to implement its pipeline expansion efforts in osteoarthritis and diabetes, as well as other near term revenue opportunities. |
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Esperion
www.esperion.com
ETC-1002 |
Esperion Therapeutics is a biopharmaceutical company focused on developing innovative therapies to prevent, treat and reverse cardiovascular and metabolic diseases. We are developing therapies that enhance the body's ability to regulate lipid and carbohydrate metabolism as well as reduce inflammation, leading to improved health outcomes. Esperion is led by Roger Newton, co-discoverer and product champion of Lipitor, and funded by top tier venture capital investors. Esperion's most advanced product candidate, ETC-1002 -- a once-daily, oral drug demonstrating statin-like reductions in LDL-C of 41% -- is in Phase 2 clinical trials for patients who have, or are at risk for developing, cardiovascular and metabolic diseases. ETC-1002 is a metabolic regulator of imbalances in carbohydrate, lipid metabolism and inflammation, unique in its ability to modulate LDL and cholesterol, free fatty acids, glucose and other cardio-metabolic risk factors. |
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Resverlogix
www.resverlogix.com
RVX-208 |
Resverlogix is a clinical stage cardiovascular company with an epigenetic drug discovery platform technology that modulates protein production.
Atherosclerosis is the underlying cause of cardiovascular disease (CVD) which is responsible for more than 18 million deaths per year worldwide (reference: WHO)*. Current LDL lowering therapies generate nearly $35 billion a year in revenue. LDL lowering therapies stabilize atherosclerosis but do not regress the disease.
There are nearly 800,000 deaths yearly in the U.S. due to CVD. According to WHO, atherosclerosis is responsible for 78% of CVD cases. The projected direct and indirect medical costs of CVD in the U.S. will grow from $500 billion in 2012 to $1.2 trillion by 2030. The largest percentage of this cost is associated with the treatment of atherosclerosis (Heart Disease and Stroke Statistics--2012 Update: A Report from the American Heart Association). Resverlogix is developing RVX-208, a first-in-class BET bromodomain inhibitor, small molecule for the treatment of atherosclerosis. It functions by removing atherosclerotic plaque by a process called reverse cholesterol transport (RCT). Epigenetics is one of the most promising fields in biotech research and drug development. It is a biological system that regulates gene expression and therefore genetic phenotype without altering the genetic code. This control is achieved through relationships between certain proteins, DNA and DNA structure resulting in increased or decreased protein production. An understanding of this system allows for the discovery and development of targeted medicines that may treat specific diseases in specific individuals. Resverlogix is developing an epigenetic drug discovery platform with the potential to impact multiple diseases including cardiovascular, cancer, autoimmune, neurodegenerative , and others. |
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Synthonics, Inc.
www.synthonicsinc.com
metallo-furosemide |
Synthonics, Inc is a specialty pharmaceutical company focused on the discovery, development and licensing of drugs that incorporate our proprietary metal coordination chemistry. Metal coordination entails attaching a pharmaceutically acceptable metal to an active pharmaceutical agent to create a new and more effective drug: a metal coordinated pharmaceutical or MCP. Metal coordination offers tremendous flexibility, allowing us to "dial in" the desired pharmacokinetics by varying the metals and adjuvants used and the manner in which they are attached. |
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Trevena, Inc.
www.trevenainc.com
TRV027 |
Trevena, Inc. is a clinical stage drug discovery & development company with a new approach to drugs targeting G-protein coupled receptors (GPCRs) by selective signaling. Trevena's expertise lies in understanding which signaling pathways downstream of a GPCR are associated with beneficial versus adverse biological effects. We then utilize that information to discover and optimize "biased ligands" that activate only the beneficial pathways to unlock new biology and avoid drug adverse effects. Our novel technology has allowed us to develop differentiated drug candidates for acute heart failure (Phase 2a complete), acute and chronic pain (Phase 1 complete) and Parkinson's Disease. |
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Vitae Pharmaceuticals
www.vitaepharma.com
VTP-27999 |
Vitae is a clinical-stage biopharmaceutical company building a portfolio of novel, small molecule, first/best-in-class compounds that address large markets with significant unmet medical needs, including chronic kidney disease, diabetes, Alzheimer's disease and atherosclerosis. Vitae's lead compound, VTP-27999, is a novel, potent and selective renin inhibitor designed to offer a best-in-class therapeutic profile for kidney protection compared to current therapeutics (ACE, ARBs, aliskiren). VTP-27999 is expected to start Phase IIb in 2Q 2013. Vitae has partnered two of its programs with Boehringer Ingelheim, an 11beta-HSD-1 inhibitor for diabetes and metabolic syndrome that is in Phase I and a BACE inhibitor for Alzheimer's disease. To date, the two partnerships have generated more than $120 million for Vitae in upfront, research and milestone payments and are expected to enable a steady flow of additional, contingent milestone payments. Vitae's additional wholly owned preclinical programs are a LXR Atherosclerosis Program with an IND filing expected in 1Q 2013, a LXR Alzheimer's Disease Program with an IND filing expected in 3Q 2013, a LXR Dermatology Program with an IND filing expected 4Q 2013 and an early discovery stage Inflammation Program. Each of Vitae's programs was generated via our proprietary, structure-based drug discovery engine called Contour®. |
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3-V Biosciences
www.3vbio.com
FASN |
3-V Biosciences, Inc. is a privately held biopharmaceutical company that discovers and develops antiviral therapeutics designed to have broad-spectrum activity (including efficacy against viruses resistant to other classes of antiviral drugs) and a high barrier to resistance. The company's first programs target the fatty acid synthesis pathway (specifically, FASN). FASN is a cellular enzyme implicated in hepatitis C virus (HCV) replication and several cancers. First-generation FASN inhibitors have shown preclinical benefits in a variety of viral infections, including HCV, as well as in models of pancreatic, prostatic, breast and colorectal tumors, but their development has been hindered by poor bioavailability and systemic tolerability. 3-V has discovered and is advancing proprietary FASN inhibitors with pharmaceutical propertiesthat overcome the shortcomings of earlier generations of inhibitors, and have the potential to become significant new approaches to the treatment of chronic HCV and other indications. The company is planning to file the first IND from this program in the first quarter of 2013. |
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Agenus Bio
www.agenusbio.com
AG-707 |
Agenus Inc. is a biotechnology company working to develop treatments for cancers and infectious diseases. The company is focused on immunotherapeutic products based on strong platform technologies with multiple product candidates advancing through the clinic, including several product candidates that have advanced into late-stage clinical trials through corporate partners. Agenus’ flagship adjuvant, QS-21 Stimulon, has become a key component in the development of investigational preventive vaccine formulations across a wide variety of infectious diseases, and appears to be essential for several investigational therapeutic vaccines intended to treat cancer and degenerative disorders. QS-21 Stimulon adjuvant is being used in 17 vaccine programs and include GSK’s Phase 3 vaccine programs for RTS,S for malaria, MAGE-A3 cancer immunotherapeutic for non-small cell lung cancer and melanoma and HZ/su for shingles. In addition, Janssen’s QS-21 Stimulon adjuvant-containing vaccine candidate is in Phase 2 trials for the treatment of Alzheimer’s disease. HerpV, Agenus’ novel QS-21 containing therapeutic vaccine candidate for the treatment of genital herpes, is in a Phase 2 study. For more information, please visit www.agenusbio.com |
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Alios BioPharma
www.aliosbiopharma.com
early stage RSV project |
Alios BioPharma is a privately held clinical stage biotechnology company located in South San Francisco, California, that is developing novel medicines aimed at the treatment of viral diseases. In collaboration with Vertex Pharmaceuticals (NASDAQ: VRTX), Alios is developing ALS-2200 (VX-135), a uridine nucleotide analog, for the treatment of chronic hepatitis C. ALS-2200 (VX-135) is expected to enter Phase 2 all-oral clinical trials in Q4 2012. Alios has developed a novel proprietary nucleoside/tide library to identify therapeutics for the treatment of several viral infections including RSV, influenza, rhinovirus, and emerging viral diseases. Additionally, Alios is developing small molecule therapeutics that will complement its nucleoside/tide portfolio. The overall goal for the Alios therapeutic platform is to maximize patient benefits in areas of high unmet medical need through optimization of potency, safety and tolerability. |
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Biota
www.biota.com.au
RSV fusion inhibitor |
Biota is a leading anti-infective drug development company based in Maryland, USA with offices in Melbourne, Australia and Oxford, UK. Biota developed the first-in-class neuraminidase inhibitor, zanamivir, subsequently marketed by GlaxoSmithKline as Relenza®. Biota has a well balanced and advanced Research and Development (R&D) portfolio including: a) vapandavir - a class leading antiviral for the treatment/prevention of human rhinovirus (HRV) infection in patients at high risk of HRV associated complications (such as in asthmatics and transplant patients with compromised immune function). Vapendavir is the first and only oral antiviral to have successfully treated HRV disease in asthmatic patients (Phase IIb trial); b) the most advanced orally bioavailable respiratory syncytial virus (RSV) fusion inhibitor antiviral in development, which is expected to enter Phase I trials in 2013; c) broad spectrum and selective/gut flora sparing, novel antibacterials for the management of Gram positive and Gram negative bacterial disease; d) nucleoside and non-nucleoside pan-genotypic antivirals for use in interferon free combination therapy for the treatment of hepatitis C (HCV) virus infections. The HCV non-nucleoside candidate together with Biota's candidate antibacterial for the intravenous and oral treatment of Gram positive infections are undergoing regulatory safety testing in anticipation of Phase I trials. Biota's portfolio also includes conjugate vaccine technology as exemplified in the nicotine conjugate vaccine NicVax®, which provides a platform for the discovery and development of preventative and therapeutic vaccines. |
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Chimerix
www.chimerix.com
CMX001 |
Chimerix is developing novel oral antiviral therapeutics with the potential to improve outcomes for patients in multiple therapeutic areas, including transplant, oncology, acute care and global health. The company's proprietary lipid technology has given rise to two clinical-stage lipid acyclic nucleoside phosphonates, CMX001 and CMX157, which have demonstrated the potential for enhanced activity and safety compared with currently approved drugs. CMX001 is a novel, broad-spectrum, oral antiviral that inhibits double-stranded DNA (dsDNA) viruses, including cytomegalovirus (CMV), adenovirus (AdV), BK virus and herpes simplex virus. CMX001 has completed Phase 2 clinical development for the prophylaxis of CMV in hematopoietic stem cell transplant (HSCT) recipients and is in Phase 2 development for the preemption and treatment of AdV infection. To date, more than 800 patients have been dosed with CMX001 in controlled clinical trials and open-label treatment protocols. Chimerix has completed an End of Phase 2 meeting with the FDA and is preparing to initiate Phase 3 clinical development of CMX001 for the prophylaxis of CMV in HSCT recipients in 2013. In addition to its commercial use, CMX001 is being developed under a contract from the Biomedical Advanced Research and Development Authority (BARDA) as a medical countermeasure to protect against a bioterror threat in the event of a smallpox release. In July 2012, Chimerix granted an exclusive worldwide license to Merck for the development and commercialization of CMX157 for the treatment of HIV infection, further validating the company's propriety lipid technology. Chimerix is also leveraging its lipid technology and novel chemical library to pursue new treatments for other areas of high unmet medical need. |
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CrystalGenomics, Inc.
www.cgxinc.com/
antibiotic for MRSA |
CrystalGenomics Inc. is a clinical stage biopharmaceutical company with drug discovery and development capabilities, and headquartered in Seoul, Korea with a US subsidiary for multi-national clinical management (CG Pharmaceuticals, Inc.) in Emeryville, California and it is publicly traded on the KOSDAQ exchange (Korea). CrystalGenomics is dedicated to the discovery and development of novel pharmaceuticals in the areas of infectious disease, cancer and inflammatory diseases. The company's capabilities have been validated through highly respected publications including a cover page on nature for scientific accomplishment (2003), R&D collaborations with prominent pharmaceutical companies including AstraZeneca (2010), and a joint venture deal with ProQuest Investments involving a very early stage asset (2008). The Company's lead development candidate is a next generation NSAID, CG100649 with a novel mechanism of suppressing COX-2 as well as carbonic anhydrase enzymes for potentially improved safety profiles over existing products and it is Phase 3-ready for osteoarthritis. Additionally, its novel HDAC inhibitor, CG200745 for cancer is in Phase 1, and a novel antibiotic candidate, CG400549 for MRSA infection, is in Phase 2a Study in the US. |
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Novavax
www.novavax.com
VLP technology/vaccines |
Novavax, Inc. is a clinical-stage biopharmaceutical company creating recombinant protein nanoparticle vaccines to address a broad range of infectious diseases. The company is developing a portfolio of vaccine candidates targeting seasonal and pandemic influenza (H5N1), a vaccine designed to protect children and the elderly against respiratory syncytial virus (RSV), rabies and other candidates. |
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Okairos AG
www.okairos.com
genetic vaccines |
Okairos is a clinical-stage biopharmaceutical company, developing genetic vaccines for major infectious diseases - including malaria, hepatitis C, influenza, respiratory syncytial virus and cancer - using a novel proprietary technology. The company is headquartered in Basel, Switzerland and has laboratories in Rome and Naples, Italy.
Okairos' technology platform is centered on the development of new, potent adenovirus vectors to generate a pipeline of T-cell vaccines against a range of infectious diseases for which there is currently no effective vaccine. The company is also pursuing therapeutic vaccines to treat cancer. The company’s investors include BioMedInvest, Boehringer Ingelheim Venture Fund, LSP, Novartis Venture Funds and Versant Ventures.
For more information, visit www.okairos.com. |
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Rib-X
www.rib-x.com/
RX-04 |
Rib-X Pharmaceuticals℠ (Rib-X) is developing new antibiotics to provide superior coverage, safety and convenience for the treatment of serious and life-threatening infections. The Company's key competitive advantage is its focus on the three-dimensional properties of antibiotics. Rib-X has proprietary understanding of the atomic-level details of the bacterial ribosome— the target of most marketed antibiotics—and the ability to use those insights in the prospective design of next-generation and completely new classes of antibiotics, two factors central to the discovery process. In addition to designing for greater target potency, Rib-X uses this structural information to design antibiotics with efficacy against highly-resistant Gram-positive and Gram-negative bacteria. The Company's proprietary drug discovery platform, which is based on Nobel Prize-winning science, has generated a pipeline of potential products spanning multiple phases of research and clinical development. |
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Theraclone Sciences
http://theraclone-sciences.com
TCN 202 |
Theraclone Sciences, Inc. is a Seattle-based biotechnology company focused on the development of novel therapeutic antibodies for the treatment of infectious disease and cancer. The Company's I-STARTM discovery platform technology is used to interrogate the human memory B-cell repertoire and harnesses the power of the human immune system to identify rare, naturally evolved, broadly-neutralizing antibodies from immunologically relevant human subjects. Such human monoclonal antibodies can then be scaled for industrial production to combat disease across broad patient populations. In addition to antibody discovery collaborations with Pfizer, Zenyaku Kogo and IAVI, Theraclone has product candidates advancing in development. TCN-032 is a broadly protective, fully human monoclonal antibody being developed for the treatment of influenza A infections that has completed Phase 1. Data showed a favorable safety, tolerability and immunogenicity profile with pharmacokinetic properties consistent with human antibodies. TCN-032 recently entered a proof-of-concept Phase 2 study. TCN-202 is a broadly-neutralizing fully human monoclonal antibody, being developed for the treatment of human cytomegalovirus (HCMV) infections that began a Phase 1 study in 2Q 2012. Theraclone is a privately held company with venture investment from ARCH Venture Partners, Canaan Partners, Healthcare Ventures, MPM Capital, Amgen Ventures and Alexandria Real Estate Equities. For additional information, please visit www.theraclone-sciences.com. |
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Trellis Bioscience, LLC
www.trellisbio.com
mAb technology |
Trellis Bioscience is a private, venture funded therapeutic antibody company formed around a breakthrough discovery platform (CellSpot™) for isolating therapeutic grade antibodies directly from the blood cells of humans. Three mature IND candidate antiviral mAbs are available for partnering, targeting CMV, RSV, and Influenza. Each binds a highly conserved epitope, with neutralizing efficacy in model systems that exceeds the best published competing mAb. The CMV program was recently awarded a $3.3M grant from NIAID to support advancing the lead mAb to clinical testing, with primary utility in preventing birth defects arising from maternal transmission of virus to the fetus. Several additional infectious disease targets, including bacterial targets, are being pursued with SBIR funding. To facilitate expanding our focus to include cancer antigens, Trellis has established a collaboration with Open Monoclonal Technology, Inc (OMT), whose OmniRat™ technology expresses fully human mAbs in transgenic rats. Since the highest quality antibodies (those with high affinity and strict specificity) are present at extremely low levels even in hyper-immunized rats, CellSpot’s ability to find very rare clones is an effective complement to the OMT technology. Trellis seeks other partnerships that can leverage the platform technology’s ability to generate superlative mAbs. For example, we are collaborating with Integrated BioTherapeutics, Inc (IBT) on discovery of mAbs for a biodefense priority pathogen. Trellis management team is comprised of veterans of the biotechnology industry, with decades of experience in drug discovery and development. |
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Visterra Inc.
www.visterrainc.com
VIS410 |
Visterra discovers and develops novel drugs utilizing its proprietary platform based on the atomic interaction network analyses of proteins. The platform enables a more in depth, three-dimensional structural understanding of proteins as ‘networks’ of amino acids. Understanding and quantifying the connectivity of these amino acids – or how networked an individual amino acid is relative to all others in a particular protein – provides unique insights into both protein structure and function. This technology enables (i) a novel approach to characterize proteins, (ii) the identification of unique target epitopes, or drug target sites, on a given protein and (iii) the design of novel drugs to target these epitopes and effectively combat disease. Proof-of-concept has been established with VIS410, a fully human IgG1 antibody targeting influenza, with preclinical data to date continuing to confirm the product’s promise to rapidly eradicate any seasonal or pandemic influenza strains. Leveraging this success, Visterra is building a proprietary pipeline of additional preventive and therapeutic drugs targeting infectious diseases. |
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| (including Women's Health, Dermatology, Bone & Muscle Disease, Ophthalmology, Rare Diseases and other high value and niche areas) |
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Agile Therapeutics
www.agiletherapeutics.com
AG200-15 / AG890 contraceptive patch |
Agile Therapeutics is a pharmaceutical development company specializing in Women's Healthcare products, with an initial focus on providing women with more options and more convenient methods of hormonal contraception. The company's lead product, AG200-15, is a once-weekly contraceptive patch that recently completed Phase III clinical trials. In addition, Agile is also developing a low dose, progestin-only contraceptive patch, AG890 (formerly AG900). Both AG200-15 and AG890 incorporate proprietary transdermal delivery technology, Skinfusion®, developed by Agile, consisting of an active and peripheral adhesive system that allows stable drug delivery and dependable adhesion over seven days. For more information, please visit http://www.agiletherapeutics.com. |
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Akebia Therapeutics
www.akebia.com
P2 anemia drug |
Akebia Therapeutics, Inc. is a private, venture-backed biopharmaceutical company focused on the development of small molecules for the treatment of anemia. The company’s lead compound, AKB-6548, is a once daily, oral HIF2 stabilizing agent (HIF-PH inhibitor) in phase 2 clinical trials for chronic anemia. AKB-6548 is designed to increase the natural production of EPO, a glycoprotein hormone that controls red blood cell production, and causes a controlled rise in hemoglobin levels. Inadequate EPO production by the kidney is a common cause of anemia. Akebia has initially targeted pre-dialysis patients with chronic kidney disease, a large patient population that is currently undertreated for anemia. The results of its recent phase 2a trial of AKB-6548 in chronic kidney disease showed a highly significant, dose-responsive increase in hemoglobin (p<0.0001) and overall red blood cell production. In addition, there was a parallel increase in iron mobilization, with significant dose-related reductions in ferritin and hepcidin, as well as an increase in total iron binding capacity (predominantly transferrin). AKB-6548 was generally well tolerated, with no dose-related changes in adverse events, liver function or renal function as compared to placebo. Of particular note, there were no dose-related changes in vascular endothelial growth factor (VEGF) levels, a HIF responsive gene, at the end of 6 weeks of dosing. Together, these data strongly suggest that AKB-6548 could provide a highly efficacious and well-tolerated oral alternative to the currently approved injectable EPO stimulating agents. The market for chronic anemia drugs, which generated over $9 billion in worldwide sales in 2010, is dominated by injectable EPO stimulating agents. There are currently no approved orally dosed small molecule drugs for the treatment of chronic anemia. |
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AllTranz
www.alltranz.com
non-neuro for our smoking cessation, drug and alcoholism treatments |
AllTranz is a specialty pharmaceutical company developing a unique set of dermal delivery products utilizing two proprietary technology platforms consisting of prodrug development and microneedle formulations. AllTranz will be a leader in the development of innovative drug design strategies delivered via unique dermal product solutions to treat a variety of disorders. The broad range of offerings includes solutions for pain modulation, appetite stimulation, smoking cessation, substitution treatment for opiate addiction, as well as alcohol abuse, and abuse deterrent formulations for opiates. AllTranz will utilize its unique dermal drug delivery technology to develop its own products for broad market appeal, as well as partner with pharmaceutical companies seeking to leverage the AllTranz technology platform to improve delivery of existing drugs to form new chemical entities thereby extending patent franchises. |
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Cytokinetics
www.cytokinetics.com
Tirasemtiv |
Cytokinetics, Inc. (Nasdaq: CYTK) is a clinical-stage biopharmaceutical company focused on the discovery and development of novel small molecule therapeutics that modulate muscle function for the potential treatment of serious diseases and medical conditions. Cytokinetics' lead drug candidate from its cardiac muscle contractility program, omecamtiv mecarbil, is in Phase II clinical development for the potential treatment of heart failure. Amgen Inc. holds an exclusive license worldwide (excluding Japan) to develop and commercialize omecamtiv mecarbil and related compounds, subject to Cytokinetics' specified development and commercialization participation rights. Cytokinetics is independently developing tirasemtiv, a skeletal muscle activator, as a potential treatment for diseases and conditions associated with aging, muscle wasting or neuromuscular dysfunction. Tirasemtiv is currently the subject of a Phase II clinical trials program and has been granted orphan drug designation and fast track status by the U.S. Food and Drug Administration and orphan medicinal product designation by the European Medicines Agency for the potential treatment of amyotrophic lateral sclerosis, a debilitating disease of neuromuscular impairment in which treatment with tirasemtiv produced potentially clinically relevant pharmacodynamic effects in Phase II trials. All of these drug candidates have arisen from Cytokinetics' muscle biology focused research activities and are directed towards the cytoskeleton. The cytoskeleton is a complex biological infrastructure that plays a fundamental role within every human cell. Additional information about Cytokinetics can be obtained at www.cytokinetics.com. |
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GTx, Inc.
www.gtxinc.com
enobosarm |
GTx, Inc., headquartered in Memphis, Tenn., is a biopharmaceutical company dedicated to the discovery, development, and commercialization of small molecules for the treatment of cancer, cancer supportive care, and other serious medical conditions. GTx is developing enobosarm (Ostarine®;GTx-024), a selective androgen receptor modulator or SARM, for the prevention and treatment of muscle loss in patients with non-small cell lung cancer and for other muscle wasting diseases. GTx is currently enrolling subjects with advanced non-small cell lung cancer in two pivotal Phase III clinical trials, POWER 1 and POWER 2. These international Phase III studies are being conducted in clinical sites in the United States, Europe, and South America. In each of the placebo-controlled, double-blind clinical trials, 300 patients with Stage III or IV non-small cell lung cancer are being randomized to oral daily doses of placebo or enobosarm 3 mg at the time they begin first line standard chemotherapy. The studies are evaluating as co-primary endpoints at three months of treatment the response rates of enobosarm versus placebo on maintaining or improving total lean body mass (muscle) assessed by dual x-ray absorptiometry and improving physical function assessed by the Stair Climb Test. GTx is also developing Capesaris® (GTx-758), a selective ER alpha agonist, as a hormonal therapy for men with advanced prostate cancer. GTx has initiated an open-label clinical study of 75 men with metastatic castration resistant prostate cancer to test three lower doses of Capesaris (125 mg, 250 mg and 500 mg) sequentially in cohorts of 25 patients each. The Phase II 712 clinical trial is designed to assess the effect of Capesaris on serum prostate specific antigen response and prostate cancer progression. The study is expected to provide confirmation of the mechanism of drug action for Capesaris on lowering serum free testosterone levels by increasing serum SHBG in castrated men who failed primary androgen deprivation therapy. |
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Hygeia Therapeutics
hygeiatherapeutics.com/
HYH-102/HYG-440 |
Hygeia Therapeutics, Inc. is a specialty pharmaceutical company focused on the development of topical products for large under-served markets in women’s healthcare and dermatology. Our two lead products target a world-wide market opportunity of over $5B. Our technology creates so-called “soft” modulators of estrogen and androgen hormone receptors. We acquired our proprietary patent protected “soft” hormone receptor modulator technology through an exclusive license from Yale University. The licensed portfolio consists of ten (10) quickly deactivated estrogens and four (4) classes of anti-androgens. “Soft” drugs are designed by strategic placement of esters to yield predictive inactive metabolites. Designed and proven to convert rapidly to inactive metabolites, these novel and patent-protected molecules deliver strong local efficacy without the systemic side effects and drug-drug interactions seen with currently marketed estrogenic and anti-androgenic therapeutics. Our first lead product is HYG-102, a “soft” analog of the estrogen hormone 17β-estradiol, is a member of the 15a-carboxylic acid esters of estrogen for treatment of vulvar and vaginal atrophy. Our second lead product is HYG-440, a “soft” anti-androgen derivative of flutamide for treatment of androgen-related skin conditions including acne, seborrhea, androgenic alopecia and hirsutism. As topical products targeting well-understood mechanisms, they offer a relatively rapid, low cost and low risk development path. Hygeia was founded and is managed by veterans of the pharmaceutical industry. |
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OvaScience
www.ovascience.com
fertility technology |
OvaScience is a life sciences company focused on the discovery, development and commercialization of new treatments for infertility. The Company's
patented technology is based on the discovery of egg precursor cells (EggPCs), which are found in the ovaries. By applying proprietary technology to identify
and purify EggPCs, OvaScience's first product candidate, AUGMENTSM, aims to improve egg quality and increase the success of in vitro fertilization (IVF). The
Company plans to begin its AUGMENT clinical study by the end of 2012, with the potential to launch in the second half of 2014. OvaScience's team of scientists, physicians
and advisers includes recognized leaders in the field of reproductive medicine. |
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Prosensa
www.prosensa.com
PRO044 |
Prosensa is an innovative Dutch biopharmaceutical company focused on the discovery, development and commercialization of RNA-modulating therapeutics correcting gene expression in diseases with significant unmet need, in particular neuromuscular disorders. Prosensa's current focus is on developing treatments for Duchenne muscular dystrophy (DMD), Myotonic Dystrophy and Huntington's disease. In 2009 Prosensa entered into a strategic alliance for part of its DMD exon skipping program with GlaxoSmithKline. Prosensa's lead compound (drisapersen), being developed by GSK, is currently in phase III clinical trials. The company has the most advanced portfolio of DMD assets, and can potentially address over 85% of all DMD patients with its innovative exon-skipping technology. Prosensa is a privately held biopharmaceutical company, backed by a consortium of Abingworth, Gimv, Idinvest Partners, Life Sciences Partners, MedSciences Capital and New Enterprise Associates. Prosensa won the 2012 Emerging Star Award at the European Mediscience Awards and was listed as a Fierce 15 Biotech Company. |
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Sarepta Therapeutics
www.sareptatherapeutics.com
AVI-4658 |
Sarepta Therapeutics is focused on developing first-in-class RNA-based therapeutics to improve and save the lives of people affected by serious and life-threatening rare and infectious diseases. The Company's diverse pipeline includes its lead program eteplirsen, which recently met the primary efficacy endpoint, increase in novel protein production, and achieved a significant clinical benefit on the primary clinical outcome, over the placebo cohort in a Phase IIb extension trial in Duchenne muscular dystrophy (DMD) patients. In addition, Sarepta is developing next-generation RNA modulators as potential treatments for some of the world's most lethal infectious diseases. Sarepta aims to build a leading, independent biotech company dedicated to translating its RNA-based science to transformational therapeutics for patients who face significant unmet medical needs. |
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Xcovery Inc.
www.xcovery.com
X-82 |
Xcovery is a clinical-stage company focused on the development of next generation targeted therapeutics for cancer and ophthalmology. Founded by Sheridan G. Snyder and Chris Liang, PhD, Xcovery's goal is to develop innovative therapies to address unmet healthcare needs and optimize patient outcomes. Through innovative drug design, Xcovery has developed a comprehensive pipeline of therapies that target a wide range of advanced tumors and also have the potential to address basic mechanisms of eye disease, including angiogenesis, fibrosis and inflammation.
The company’s lead product, X-82, an oral VEGFR/PDGFR tyrosine kinase inhibitor (TKI), is currently being evaluated in clinical studies to treat both advanced solid tumors and age-related macular degeneration. Preliminary data from the first-in-human oncology trial presented at the ASCO 2012 Annual Meeting showed that X-82 is well tolerated and demonstrated preliminary signs of anti-tumor activity (7/16 on study ≥16wks; 1 complete response). In addition, X-82 has the proper pharmacokinetic properties that meet the pharmacodynamic requirements, differentiating it from all competitors’ compounds.Maximum tolerated dose and recommended Phase 2 dose have not been determined at this preliminary phase. In October 2012, Xcovery initiated a Phase I/II clinical study to evaluate the safety and preliminary biologic activity/efficacy of X-82 in patients with wet Age-related Macular Degeneration (AMD).
Xcovery is a holding company, which encompasses Xcovery, a clinical-stage company focused on the development of next-generation targeted therapeutics for cancer, and Xcovery Vision. X-82 was developed by Tyrogenex, an Xcovery-affiliated company funded by Biocatalyst International, a closely held early-stage venture fund. |
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