From depression to ulcers and anthrax infections, a handful of Maryland biotechs have been given the regulatory OK to either launch or resume clinical trials of their experimental treatments.
Neuralstem reported that the Food and Drug Administration has approved its investigational new drug application to launch a phase 1a safety trial of its experimental small-molecule compound to treat major depression.
The compound, NSI-189, was discovered by Neuralstem, a Rockville biotech that also focuses on stem cell treatments for nervous system disorders such as amyotrophic lateral sclerosis.
The new product works by stimulating new neuron growth in the brain's hippocampus, which may be involved in depression and other ailments, such as Alzheimer's disease, according to a Neuralstem statement.
"Today's antidepressants are based on a theory of serotonin deficiency," said Karl Johe, the company's chairman and chief scientific officer, in the statement. "a new theory is emerging that chronic stress can lead to hippocampal atrophy and eventually to depression. NSI-189 appears to help the brain repair itself, generating new neurons and protecting against damage. this neurogenic approach is completely novel in the treatment of CNS diseases."
NSI-189 showed promising results in mice, the company said.
The treatment is a new tack for the biotech, according to CEO Richard Garr.
"In addition to our ongoing programs in cell therapeutics, we are now advancing a new class of orally administered drugs that recruit endogenous neural stem cells," Garr said in the statement. "NSI-189 is the first in this class."
The trial will test a single oral dose of NSI-189 in healthy patients. If its safety endpoints are met, the trial will move to a second phase, testing the safety of daily escalating doses for 28 days in depressed patients. The company expects the entire phase 1 trial to run about a year.
Another Rockville biotech, Sequella, also filed an investigational new drug application with the FDA and received permission to start a phase 2 trial of SQ109 to treat duodenal ulcers caused by the helicobacter pylori bacterium.
"H. pylori is a highly infectious pathogen that causes 95 percent of duodenal and 85 percent of gastric ulcers, and is implicated in most gastric cancers," CEO Carol a. Nacy said in a statement. "we are quite pleased with the clinical development of SQ109 as we progress through phase 2 trials for tuberculosis and look forward to achieving similar success against H. pylori."
The new phase 2 study is based on results from phase 1 studies of SQ109 for treating tuberculosis. it will be conducted at the Baylor College of Medicine in Houston.
"H. pylori is an important human pathogen and the cause of gastric cancer. Worldwide, increasing antibiotic resistance has resulted in a marked reduction in the ability to cure the infection, and new drugs are urgently needed," David Y. Graham, who will help conduct the study, said in the statement.
about 14.5 million Americans have ulcers, according to federal data cited by Sequella. a new drug that simplifies the current regimen of three or four drugs could generate more than $500 million in annual sales, the company said.
Meanwhile, Annapolis biodefense company PharmAthene announced that it has received FDA clearance to resume clinical testing of its candidate to treat anthrax infections.
The FDA had partially suspended the testing program about a year ago after the company reported two adverse reactions in the four subjects who were dosed in a phase 1 clinical trial.
"We're very pleased to resume clinical testing of Valortim," said Thomas Fuerst, senior vice president and chief scientific officer, in a statement. "The accumulating data suggest that Valortim may have unique characteristics, which could position it as a leading candidate for future procurement in the nation's biodefense stockpile. a presumed different mechanism of action, relative to other anthrax monoclonal antibodies and encouraging efficacy data in animal studies at low doses, suggest that Valortim may offer important advantages in the treatment of symptomatic anthrax infection."
PharmAthene plans to begin a phase 1 dose-escalation study of Valortim within several weeks, Fuerst said. Valortim is a fully human anti-toxin monoclonal antibody being developed to prevent and treat inhalational anthrax.
Another Rockville biotech, Novavax, reported that the FDA has lifted its hold on the company's phase 1 clinical trial to study a new vaccine candidate to prevent respiratory syncytial virus infection. last month, Novavax reported that the FDA had a question about the candidate's chemistry, manufacturing and controls, a matter that has been resolved.
The virus can cause lower respiratory infections, sometimes resulting in infant deaths and illness in the elderly. Novavax has tested the vaccine, based on its virus like-particle technology, in cotton rats.
"this is a significant accomplishment and represents the second major internally discovered vaccine program based on our core platform technologies," CEO Rahul Singhvi said in a statement.
a Gaithersburg biotech, GenVec, also is developing a vaccine for the disease. it recently reported encouraging preclinical proof-of-concept findings of its vaccine program from studies in multiple animal models.
"these data are promising and show that a vaccine utilizing our technology has significant potential to prevent" respiratory syncytial virus, said Jason Gall, senior research director at the Gaithersburg biotech, in a statement. "currently, there is no approved RSV vaccine and we are excited to be moving toward filling the need for a safe and effective RSV vaccine."
Respiratory syncytial virus causes lower respiratory infections in infants and young children. Infections typically produce cold-like symptoms, but can result in severe lower respiratory tract infections, causing up to 130,000 pediatric hospitalizations per year in the U.S., according to GenVec information.
In other Maryland bioscience industry news:
MedImmune of Gaithersburg, the biologics division of AstraZeneca of London, has entered into a license and collaboration deal with Evotec of Hamburg, Germany, to develop a diabetes treatment.
The work will focus on regenerating insulin-producing beta cells, according to an Evotec statement. MedImmune will have exclusive access to research programs.
Evotec will receive an upfront payment of $6.6 million, with payments potentially reaching $335.9 million, if certain milestones are achieved and sales royalties are realized.
United BioSource, a contract research organization in Bethesda and wholly owned subsidiary of Medco Health Solutions, has acquired Total Healthcare Group of London, an international consultancy with clients in the biopharmaceutical industry.
Total Healthcare, along with United BioSource's value strategy and research services, will be led by the London company's founder, Robert Hollamby, who has been named senior vice president at United.
"Demonstrating value and providing evidence-based research that allows stakeholders in the health care market to make important economic decisions surrounding biopharmaceuticals and devices is crucial in the market place," said UBC President Mark Clein. "UBC helps clients respond effectively to the evolving health care environment by researching, communicating, and demonstrating value in medicines.
"The addition of [Total Healthcare] significantly expands our capabilities and geographic reach and allows us to launch a comprehensive practice that we expect to be a leader in the global biopharmaceutical industry," said Mark Clein, president of United BioSource, in a statement.
Medco of Franklin Lakes, N.J., which provides pharmacy benefits management services, acquired United BioSource in September for $730 million in cash. Medco reported a third-quarter profit of $371.5 million on revenues of $16.32 billion.
Osiris Therapeutics of Columbia, which develops stem cell treatments, settled its patent infringement suit against Orthofix of Lewisville, Texas, according to an Orthofix statement.
Osiris and the licensee for its patented treatment, NuVasive of San Diego, sued Orthofix in U.S. District Court in New Jersey in April, claiming patent infringement. NuVasive paid Osiris $85 million for the license to the treatment, which can be used to repair defective bones, according to the suit.
Orthofix reported that it would record a fourth-quarter charge of $2 million related to the settlement, but said other terms were confidential.
Micromet of Bethesda reported that 21 adult patients with minimal residual disease positive acute lymphoblastic leukemia who were treated by its lead candidate, blinatumomab, in a phase 2 trial experienced prolonged remissions.
Blinatumomab is the first of a new class of agents called BiTE antibodies, designed to harness the body's T cells to kill cancer cells, according to Micromet.
Of 20 evaluable patients, 16 achieved a complete molecular response, all within the first cycle of treatment, the company reported.
"Nine of the patients received an allogeneic stem cell transplant, a toxic procedure that typically carries a high short-term risk of mortality," the company said. "Notably, all nine transplanted patients were alive 100 days following the transplant."
Micromet also reported encouraging results from a phase 1 trial of blinatumomab to treat relapsed non-Hodgkin's lymphoma. Blinatumomab continues to produce "a high response rate and duration of response in a number of different [non-Hodgkin's lymphoma] subtypes."
Emergent BioSolutions of Rockville reported positive data from a phase 1 dose escalation study of TRU-016, its candidate to treat chronic lymphocytic leukemia.
"Based on favorable results observed to date, Emergent and our development partner Abbott are in the process of initiating additional combination studies of TRU-016" in chronic lymphocytic leukemia and non-Hodgkin's lymphoma, said W. James Jackson, chief scientific officer at Emergent, in a statement.
Rexahn Pharmaceuticals has won a U.S. patent for its method of using beta-lactam compounds to enhance cognitive function and treat cognitive disorders such as dementia, amnesia and Altzheimer's disease.
The patent "further broadens our [central nervous system]-related portfolio and strengthens our intellectual property position with potential treatments for neurodegeneration," President Rick Soni said in a statement.
Champions Biotechnology of Baltimore was selected by Teva Pharmaceutical Industries of Israel to provide its technology to test Teva's novel anti-angiogenic compound for treating cancer.
such compounds are designed to work by blocking the tumor's blood supply, according to a joint statement. Champions will use its platform to test the effectiveness of Teva's compound in various cancer indications to help in clinical development.
"The use of our highly predictive Champions Tumorgraft platform will provide Teva with the data necessary for critical drug development decisions and help focus and accelerate the development program for this compound," said Elizabeth Bruckheimer, vice president of scientific operations with Champions, in the statement.
VIRxSYS of Gaithersburg reported positive results from a study of the simian version of its HIV vaccine candidate in monkeys with highly pathogenic simian immunodeficiency virus.
The immunized animals showed sustained viral load reduction, preservation of the immune system and increased survival compared to controls, the company said in a statement.
"If successfully translated to humans, our results would indicate that the therapeutic vaccine VRX1273 has the potential to lower levels of circulating virus, diminish its damage to the immune system and improve the lives of individuals living with HIV," Franck Lemiale, the company's senior director of vaccines and immunology, said in the statement.
RegeneRx Biopharmaceuticals reported receiving a Mexican patent for using its thymosin beta 4 and related products to prevent or heal damage from a heart attack.
RegeneRx has similar patents pending in several countries, including the U.S., the Rockville biotech said in a statement.
an adjuvant developed by Profectus Biosciences has significantly boosted the efficacy of an experimental DNA vaccine to prevent HIV infection, according to the Baltimore company.
In a phase clinical human trial, the Genevax IL-12 pDNA adjuvant produced response rates and magnitude of responses "significantly higher than those seen previously with other DNA vaccine trials," the company reported at the annual HIV Vaccine Trials Network Conference last month in Seattle.
In the trial, 48 healthy, HIV-negative volunteers were vaccinated to assess the safety and levels of immune responses generated by an experimental HIV DNA vaccine — Pennvax-B, provided by Inovio Pharmaceuticals — delivered with or without the Profectus BioSciences adjuvant. The multi-center study is sponsored by the National Institute of Allergy and Infectious Diseases.
"we thank NIAID for supporting the clinical trials … and for the award of the $32 [million] … contract that has supported 60 percent of the research, development and manufacturing costs of the Profectus HIV DNA vaccine and Genevax IL-12 program, while the remainder of the cost has been provided through a contribution-in-kind," John Eldridge, chief scientific officer, said in a statement.
Akonni Biosystems, a Frederick diagnostics company, has raised $912,598 of a new $8 million equity offering, according to its filing with the Securities and Exchange Commission.
In March, Akonni raised $5 million of a $6.3 million debt offering. The company also recently won three grants totaling almost $4 million from the National Institutes of Health and National Institute of Justice.
The company also reported winning a $150,000 phase 1 National Science Foundation grant to investigate the feasibility of developing a lab-on-a-film microarray device that could be manufactured in a reel-to-reel format.
"The benefit of this manufacturing approach is that lab-on-a-film microarray production and assembly can be automated at very high speeds, resulting in 10- to 100-fold savings in costs," Christopher Cooney, director of engineering, said in a statement.
The PATH Malaria Vaccine Initiative, a Bethesda nonprofit, has launched a new collaboration with Merck and the New York University Langone Medical Center to help develop a new vaccine.
The group will evaluate an approach targeting a novel part of a major surface protein — the circumsporozoite protein — on the malaria parasite, according to a joint statement. by blocking a critical function of the protein, researchers hope to prevent the parasite from entering the liver.
"With the availability of a first-generation malaria vaccine on the horizon, we are ramping up our efforts to seek out and invest in scientific approaches for malaria vaccines that could potentially be even more effective and protect more people," said Christian Loucq, director of the Malaria Vaccine Initiative, in the statement. "we are very pleased that one of the world's largest pharmaceutical companies and a major academic medical center have committed to testing a promising new way to defend children against malaria."
Malaria kills almost 900,000 people annually, mostly children younger than 5 in sub-Saharan Africa, according to the group.