"Fierce 15" for 2009 -Biotech companies with promising products

[Rich66]

June 30, 2009

FierceBiotech Announces "Fierce 15" for 2009

By Christopher P. Singer --
FierceBiotech published its annual "Fierce 15" list of what it considers to be among the most promising of the up-and-coming companies in the biotechnology industry. FierceBiotech, a daily biotech industry news and article provider, has been publishing the Fierce 15 since 2003. The companies below (listed alphabetically) were named to the Fierce 15 for 2009. The brief description of each company on this year's list has been adapted from information taken from each company's website.
Achaogen develops small molecule therapeutics to treat multi-drug resistant bacterial infections, which cause hundreds of thousands of deaths worldwide each year. The company is developing next-generation aminoglycosides called, "neoglycosides." Achaogen's lead compound, ACHN-490, has displayed efficacy in research and nonclinical studies against systemic infections caused by multi-drug resistant (MDR) Gram-negative bacteria (e.g., E. coli and K. pneumoniae) and MRSA. The compound entered a Phase 1 clinical trial in early 2009.
Agios Pharmaceuticals is a biopharmaceutical company focusing on cancer metabolism, providing an understanding of how cancer cells become addicted to using more nutrients than normal cells to ensure their survival and growth. Agios is developing a deep pipeline of drug candidates that disrupt the growth and survival of cancer. The company's initial focus is on cancer, particularly targeting three key metabolic pathways: glycolysis (sugar metabolism), fatty acid metabolism, and autophagy (self-metabolism), but the company's platform will also be applicable to other therapeutic areas including autoimmune, inflammatory, and neurological diseases.
Aileron Therapeutics is a biopharmaceutical company developing a new class of drugs called Stapled Peptides. This new class of drugs represents the first general solution for modulating intracellular protein-protein interactions, which have been identified as critical control points for most human diseases. Aileron's approach provides an opportunity to exploit potentially thousands of currently "undruggable" targets with applications in all human diseases. Aileron's Stapled Peptides are structured into an alpha-helical shape via an optimized cross-linking chemistry, mimicking the structure found at the interface of many protein-protein interactions. The resulting Stapled Peptide drugs are endowed with unique properties, including efficient cell penetration, high affinity binding to large target protein surfaces, and excellent stability within the body.
BioVex is a biotechnology company focusing on the development and commercialization of biological treatments for cancer and the prevention of infectious disease. The company's lead cancer technology platform, OncoVEXGM-CSF, is a first-in-class oncolytic, or cancer destroying, virus technology. OncoVEXGM-CSF works by replicating and spreading within solid tumors (leaving healthy cells unaffected), causing the death of cancer cells, and through stimulating the immune system to destroy uninjected metastatic deposits. BioVex's second major program is a vaccine for genital herpes, ImmunoVEXHSV2, which provides complete protection in animal models of the disease. The vaccine has been authorized to commence clinical testing in the UK.
Calistoga Pharmaceuticals is developing oral medicines targeting selected isoforms of the PI3 kinase pathway to improve the health of patients with cancer and inflammatory diseases. Calistoga Pharmaceuticals has a portfolio of proprietary compounds selectively targeting the isoforms of the PI3 kinase pathway. The pipeline includes a p110δ (delta) selective PI3 kinase inhibitor under clinical evaluation in patients with hematologic malignancies; a p110δ and p110β (beta) inhibitor under preclinical evaluation for the treatment of patients with solid tumor malignancies; and a p110δ selective PI3 kinase inhibitor advancing toward Phase 2 for treatment of patients with inflammatory diseases.
Clovis Oncology (website under development) has assembled a management team from Pharmion (purchased by Celgene) and a group of investors that are willing to provide $145 million in capital to fund Clovis, which is planned to follow a model similar to Pharmion's. The Clovis team will likely select five or six candidate cancer therapeutics from several developers and attempt to shepherd them toward regulatory approval.
Heptares Therapeutics is a drug discovery company focusing on novel small-molecule drugs targeting G-protein-coupled receptors (GPCRs), the largest family of druggable targets. The company has developed a technology for making purified, stabilized, and functional GPCRs (known as StaRs™, Stabilised Receptors), overcoming a major limiting factor to the development of new drugs targeting this group. Heptares' StaR platform is based on science from the MRC Laboratory of Molecular Biology (Cambridge, UK) and enables the company to engineer and purify GPCRs in stable and functional conformations that retain their drug-binding characteristics. For the first time, contemporary drug discovery approaches, such as crystallization and structure-based design, biophysical analysis of ligand interactions, and fragment screening can now be applied to GPCRs. This unique approach is expected to radically improve the chances of finding drugs to previously intractable targets and will enable the development of safer and more selective therapeutic agents.
Ironwood Pharmaceuticals is focused at the interface of pharmacology and chemistry. The company's programs have a pharmacologically validated mechanism along with robust readouts that are predictive of clinical outcomes. This methodology allows Ironwood to rapidly iterate between in vivo models and chemistry to efficiently find lead molecules with clinical promise. Linaclotide, is Ironwood's first-in-class compound, and is being evaluated in a comprehensive Phase 3 clinical program for the treatment of irritable bowel syndrome with constipation and chronic constipation. The company's pipeline of clinical candidates focuses on areas such as gastrointestinal and cardiovascular diseases, pain, and inflammation.
iZumi Bio is focusing on the application of cellular reprogramming, including the scientific breakthrough of induced pluripotent stem (iPS) cell technology for drug discovery. Because iPS cells have stem-cell-like properties, such as self-renewal and pluripotency (i.e., the ability to self replicate indefinitely and to differentiate into a variety of cell types), iPS cells are considered to be of great potential for providing insights into disease mechanisms, preclinical drug testing, and cell-based therapy. In addition, iPS cells avoid the ethical issues specific to human embryos and human embryonic stem cells. iZumi Bio's technology involves creating disease-relevant model systems from reprogrammed cells obtained in a non-invasive manner (i.e., skin cells) from patients with known disease.
KaloBios focuses on monoclonal antibody technology, using a proprietary platform to develop first-in-class human antibody therapeutics. The company has multiple programs that were in seven Phase 1 or 2 clinical trials in 2008-09, including an anti-infective for Pseudomonas aeruginosa infections being tested in cystic fibrosis and in intensive care patients on a ventilator; two molecules being evaluated in inflammatory conditions, such as rheumatoid arthritis and asthma; and a molecule in preclinical development for oncology. KaloBios' Humaneering™ technology offers advantages over other methods of human antibody creation in terms of immunogenicity, potency, and manufacturing yields.
Pacific Biosciences is developing a transformative DNA sequencing platform -- a single molecule real time (SMRTTM) technology that provides long reads, low costs, and fast cycle times. The technology allows for the observation of natural DNA synthesis by DNA polymerase as it occurs. The two key innovations behind the technology include the SMRT chip, which enables observation of individual fluorophores against a dense background of labeled nucleotides by maintaining a high signal-to-noise ratio; and phospholinked nucleotides, which produce a completely natural DNA strand through fast, accurate, and processive DNA synthesis.
Portola Pharmaceuticals is focused on therapeutics that target cardiovascular, inflammation, and oncology indications. Portola has a preclinical pipeline that includes molecules such as a FXa inhibitor antidote to address potential bleeding issues from use of a FXa inhibitor; a Syk and JAK inhibitor program developing compounds for the treatment of rheumatoid arthritis and certain cancers; and an aspirin replacement therapy for aspirin intolerant patients. Portola also has two compounds in Phase II clinical development in the area of thrombosis: betrixaban is an oral, once-daily Factor Xa inhibitor anticoagulant, and elinogrel is formulated as an i.v. and oral antiplatelet agent that is a direct-acting, reversible P2Y12 ADP receptor antagonist.
Proteon Therapeutics is developing a line of first-in-class pharmaceuticals for the treatment of renal and vascular diseases. Proteon possess unique knowledge of tissue remodeling mechanisms that allows it to develop its pipeline of therapeutics focusing on vascular access for hemodialysis and peripheral arterial disease (PAD). The company's first candidate molecule (PRT-201) is in development for the improvement of blood flow following vascular surgery procedures.
VaxInnate is developing novel vaccines targeted against pandemic and seasonal influenza. VaxInnate holds proprietary toll-like receptor technology which allows for rapid production and development of its fusion vaccines. These fusion vaccines also allow for the development of compositions that can generate an immune response to conserved regions of viral proteins that typically exhibit low immunogenicity, which may lead to a product that can be effective against a wider variety of strains and pandemic viruses.
Zafgen develops novel therapeutics for the treatment of obesity. Zafgen's research and development is based upon recent studies that demonstrate that the nature of adipose tissue, or fat cells, is a significant determinant of obesity, and that adipose tissue plays an active role in the disease. The company targets adipose vasculature in an attempt to shrink fat cells and help to maintain a more lean body composition.
For additional information regarding this topic, please see:
• "FierceBiotech Announces the 'Fierce 15' Biotech Companies for 2008," June 25, 2008


Posted at 11:14 PM in Biotech/Pharma Business | Permalink