Jazz Pharmaceuticals (www.jazzpharmaceuticals.com) has three new products, one recent acquisition, and $345 million in funding. Products include Xyrem (sodium oxybate) for patients with narcolepsy who suffer from excessive daytime sleepiness. In a Phase II trial of patients suffering from fibromyalgia, Xyrem also relieved pain and improved functioning. Antizol (fomepizole) injection is indicated as an antidote for ethylene glycol (such as antifreeze) or methanol poisoning. Cystadane (betaine anhydrous for oral solution) is the first agent for the treatment of homocystinuria, a rare genetic disorder.
Matthew Fust, senior vp and CFO, reported that the company recently completed its acquisition of Orphan Medical. Jazz is actively looking to expand its internal portfolio of products and development projects through in-licensing, acquisition, and co-development partnering transactions.
Amarin (www.amarincorp.com) is developing medications for major neurodegenerative disorders. The company’s drug, Miraxion, is synthesized from eicosapentaenoic acid, an omega-3 fatty acid, which is believed to stabilize cell membranes and mitochondrial integrity of stressed neurons, thereby preventing or slowing progression of neuronal degeneration.
Amarin currently is conducting large Phase III clinical trials of Miraxion in the U.S. and Europe to treat Huntington’s disease. The company recently acquired an oral formulation of apomorphine to treat Parkinson’s disease, which will broaden its neurology portfolio. Miraxion also was studied in a Phase II trial of depressed patients who failed to respond to current treatments, both as an adjunctive therapy and a monotherapy.
"As a result of encouraging clinical trial results, Amarin intends to further evaluate the clinical benefits of Miraxion in depression and will seek a development and marketing partner to accelerate this program," said Rick Stewart, CEO of Amarin.
Predix Pharmaceuticals(www.predixpharm.com), which recently merged with EPIX Pharmaceuticals, is focused on the discovery and development of novel, highly selective, small molecule drugs that target GPCRs and ion channels.
Using its drug discovery technology, Predix has advanced several drug candidates to clinical trials. The lead clinical-stage drug candidate, PRX-00023, a novel long-acting 5-HT1A agonist, completed enrollment for a Phase III trial in patients with generalized anxiety disorder. PRX-03140, a highly selective, small molecule agonist of a specific GPCR known as 5-HT4, completed Phase I trials indicating that the drug penetrated the brain and was well tolerated in patients with AD.
BrainCells (www.braincellsinc.com) applied the phenomenon of therapeutic neurogenesis to the field of psychiatry for the indication of treatment-resistant depression. The company is pursuing improved antidepressants by targeting small molecule drugs that specifically modulate the neurogenesis cascade leading to the development of new neurons.
Neurogenesis has been shown preclinically to play a role in the antidepressant efficacy of Prozac and other antidepressant medications. The goal of current research is to provide greater sensitivity, specificity, and predictive value in the treatment of depression and related mood disorders. Specifically, the neurogenesis-based approach to disorders of mood and anxiety may enable the discovery and development of next-generation antidepressants with greater efficacy, fewer side effects, and faster onset of action.
"The company is deploying its neurogenesis platform for profiling and selection of drug candidates," noted Jim Schoeneck, CEO of BrainCells. "We believe that the platform represents a major improvement in the predictive power of preclinical models for CNS disorders, in particular depression and mood disorders, which will facilitate a paradigm shift in CNS drug discovery."
NeuroNova (www.neuronova.com) is developing novel drugs to induce therapeutic neurogenesis. The drug development program is aimed at stimulating growth processes in the brain that lead to the formation of new endogenous neurons. NeuroNova uses its integrated platform of drug discovery technologies for neurogenesisthe Neurogenesis Qualifierto identify therapeutically relevant drug targets and to develop new drugs for these targets.
The company’s three leading drug candidates are sNN0029 for an undisclosed orphan drug indication, and sNN0031 and sNN0465 for Parkinson’s disease. Preclinical proof-of-concept has been demonstrated in animal models. Using ICV administration to deliver neurogenic drugs resulted in dose-dependent neurogenesis in vivo. Clinical trials are planned for 2006.
"We believe therapeutic neurogenesis can revolutionize the treatment of several currently incurable neurodegenerative diseases, such as Parkinson’s disease and Alzheimer’s disease, as well as certain orphan drug indications," states Anders Haegerstrand, M.D., Ph.D., CSO of NeuroNova.
Sound Pharmaceuticals (www.soundpharmaceuticals.com) is developing medications to prevent and treat hearing loss. Currently, there are no FDA-approved drugs that protect or restore hearing loss due to noise damage, ototoxic drugs, or aging.
The company is focusing on the role of cyclin-dependent kinase inhibitor 27 (p27Kip1), an enzyme shown to regulate cellular proliferation by interrupting the cell cycle. This research demonstrates for the first time that hair-cell regeneration is possible in mammals, and that cell-cycle regulation is important in controlling hair-cell regeneration.
Sound received a DARPA grant from the DoD to develop its novel regenerative therapies. Jonathan Kil, M.D., president and CEO of Sound, said that "based on promising research with p27Kip1, the company is currently optimizing its lead compound for further testing and expects to develop its patented formulations into oral drugs that will provide protection and regeneration."