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United Therapeutics has pulled its application to market one of its hypertension drugs in Europe after regulators there found problems with supporting records from two clinical trial sites.

Tyvaso, which is inhaled, is approved by the U.S. Food and Drug Administration to treat pulmonary arterial hypertension, and the Silver Spring drug-maker said it would eventually reapply for approval to sell it in the European Union.

"Our revenue growth targets are unaffected by the withdrawal," CEO Martine Rothblatt said in a statement. "We have long based our growth targets on the estimated 30,000 diagnosed [pulmonary arterial hypertension] patients in the U.S., including approximately 12,000 New York Heart Association Class III patients."

"It's disappointing, but it won't have a financial effect on United Therapeutics," Rothblatt added in a conference call with analysts. "We will prevail and get Tyvaso approved in Europe."

European regulators are "exacting a price from the fact that we got Remodulin approved in the European Union around them rather than through them," Rothblatt said, referring to the company's injected form of Tyvaso.

Rothblatt conceded her fault in the regulators objections, saying she supervised the Tyvaso project early on and "back then, some i's were not dotted, some t's not crossed. ... Those have been latched on" by the European regulators.

"They say good clinical practices [have] to be meticulous, whether it affects the data or not," she said.

The lapses occurred early in the Tyvaso testing project, "but nevertheless they were a legitimate reason for the [European Medicines Agency] to request a further study because of [its] hyperstrict rules on single-study approvals," Rothblatt said.

The errors concerned administrative issues related to patient consent and retention of source documents, said Roger Jeffs, president and COO, in the call. The errors were of "particular importance, given the filing was based on a single study."

"These didn't impact the findings of the study, but we couldn't convince" the European regulators of that, Jeffs said.

In fact, when the results from the patients whose records were in question — about 10 percent of the sample — were removed, "we got better results," he said.

"We definitely, positively, absolutely will do a new study," Rothblatt said. "It's important for European patients to have the benefit of Tyvaso. We will endeavor to get approval in Europe," where Tyvaso would have 10-year orphan drug exclusivity.

"It's hard to predict" how long it will take to conduct a new study, Jeffs said, but he estimated four years from start to finish.

In other Maryland bioscience industry news:

GenVec has signed a $3.8 million deal with the Department of Homeland Security to continue developing vaccines against foot-and-mouth disease. If renewals are exercised, the Gaithersburg company could receive an additional $700,000.

"This contract expands our ongoing efforts to develop adenovector-based [foot-and-mouth disease] vaccines," CEO Paul Fischer said in a statement. "This new contract with the DHS will support new vaccine discovery and technology improvement for this important threat."

Sanaria of Rockville has received a three-year, $3 million phase 2 Small Business Innovation Research grant from the National Institutes of Health to support its work on developing a malaria vaccine.

The grant will help fund research the company is conducting with Columbia University.

"Sanaria is uniquely positioned at this time to expand the pipeline of candidate sporozoite vaccines to include vaccines based on precisely gene-altered parasites that are highly potent in inducing protective immunity against malaria and are unable to cause disease," said company founder and chief scientist Stephen L. Hoffman in a statement.

Novavax reported positive results from a 1,000-patient study of its virus-like particle H1N1 flu vaccine at the World Health Organization in Geneva.

Healthy volunteers, ages 18 to 64, received various doses of the vaccine or a placebo to test the candidate's safety and immunogenicity, according to the Rockville biotech. Preliminary results after two weeks showed it was well tolerated at all three dose levels, with no systemic side effects and "robust serological immune responses that are considered protective."

The trial's second stage, with about 3,000 subjects, is ongoing.

"The data presented today suggest that our H1N1 [virus-like particle] vaccine is well tolerated and has a robust immune response even at a low dose of 5 mcg," CEO Rahul Singhvi said in a statement. "This is remarkable given that our vaccine does not contain any adjuvant."

Neuralstem of Rockville reported raising $5.23 million this year, including a previously reported $1.5 million private placement of 646,551 common shares and about $3.7 million from warrant exercises.

The stem-cell development company also announced that it received notice from the NYSE Amex that it is now in compliance with the exchange's listing requirements.

VIRxSYS Corp. of Gaithersburg said it is "encouraged" by results from a study of its HIV vaccine candidate in monkeys.

The vaccine "is capable of achieving significant control of viral load over the course of four months following a challenge with a highly pathogenic simian immunodeficiency virus, a virus found in non-human primates and similar to HIV," according to a company statement.

The company is preparing an investigational new drug application for therapeutic use of the candidate in HIV-infected patients.

"We are extremely encouraged by the results of this study. The combination of strong immune responses, viral control, and CD4 preservation is tremendous. In addition, contrary to most viral vectors currently in development, our lentiviral vector elicits nominal anti-vector responses and therefore can be successfully readministered," said Franck Lemiale, director of immunology, in the statement. "It will be very interesting to see how it performs as a therapeutic vaccine in humans."

Vaccinogen of Frederick, which develops treatments for tumors and tumor vaccine products, has completed a $4.0 million equity offering, according to a filing with the Securities and Exchange Commission.

Of the proceeds, $356,250 will be used for payments to at least one of the Vaccinogen principals.

PharmAthene of Annapolis, which recently won up to $78.4 million in a federal contract to continue developing its next-generation SparVax anthrax vaccine, reported encouraging data from a study of its Valortim to treat and prevent inhalation anthrax.

The new data show that Valortim enhances the human T cell response to B. anthracis and may also enhance the adaptive immune response to anthrax, according to a company statement. Preclinical studies suggest that Valortim could both prevent and treat an anthrax infection.

The latest data are from research supported by the Maryland Industrial Partnerships Program, which is designed to accelerate the commercialization of technology in the state by funding collaborative research and development projects between companies and University System of Maryland faculty.

Celsion, which is already testing its ThermoDox to treat primary liver cancer and recurrent chest wall breast cancer, said it will launch a phase 2 study this year to determine how well it can help treat colorectal liver metastases.

The Columbia company will test the candidate in tandem with radiofrequency ablation at Montefiore Medical Center in New York City, plus at least two other sites in North America and the Asian-Pacific region. Colorectal liver metastasis is currently treated by radiofrequency ablation.

"Celsion Corporation first began studying ThermoDox in combination with RFA for liver metastases in a phase 1 safety study of 24-patients, 15 of which had liver metastases from nine primary sites," CEO Michael Tardugno said in a statement. "The safety experience and dose response relationship we witnessed in the phase 1 study was evident in both primary and metastatic tumors."

There were about 147,000 new cases of colorectal cancer and about 50,000 colorectal cancer deaths last year in the U.S., according to the American Cancer Society. Liver metastases are presented in up to 25 percent of colorectal cancer patients, with an additional 50 percent developing it within five years. Their median survival is under a year, if untreated.

ThermoDox is a heat-activated liposomal encapsulation of doxorubicin, an approved and frequently used cancer drug, according to Celsion information. The drug is released by raising the body temperature where it is administered to about 106 degrees; this way, high concentrations are deposited in the targeted tumor.

The drug is being tested in a 600-patient phase 3 study at 70 clinical sites; enrollment is expected to be completed this year. Celsion said it expects to enroll about 100 patients in its study of ThermoDox for recurrent chest wall breast cancer and to complete the study by this summer.

Celsion also announced it will participate in a European study of ThermoDox and MRI-guided high-intensity focused ultrasound for liver and bone metastases.

The four-year project, led by University Medical Center Utrecht (Netherlands), has a budget of $8.7 million and is expected to begin in May.

An investigational new drug submission is planned for this year, pending successful completion of the preclinical studies.

"The drug/device combination of ThermoDox and MR-HIFU acts synergistically with the potential to provide an innovative non-invasive therapy for patients with aggressive and life-threatening cancers," said Nicholas Borys, Celsion's vice president and chief medical officer, in a statement. Philips Healthcare manufactures the ultrasound device.

Sequella reported raising $2 million of a $6 million private debt offering.

The Rockville biopharmaceutical company is developing tuberculosis treatments.

Cyto Pulse Sciences of Glen Burnie, which produces electrical DNA vaccine delivery systems, reported that the Swedish Medical Products Agency has approved a phase I clinical trial evaluating an HIV/DNA vaccine in collaboration with the Karolinska Institutet and the Swedish Institute for Infectious Disease Control.

The vaccine will be delivered to healthy volunteers by ZetaJet, a needle-free injection system provided by Bioject Medical Technologies, in combination Cyto Pulse's intradermal electroporation system, Derma Vax.

"We are optimistic the treatment will be effective and therefore have signed a letter of intent to participate in a follow-up phase 2 trial in Tanzania and Mozambique," said Cyto Pulse CEO Derin Walters in a statement.

STB Lifesaving Technologies of Rockville reported that $2.4 million was included in the Pentagon's fiscal 2010 appropriations bill to support development of its Fibrin Adhesive Stat dressing.

The product is designed to be effective against all kinds of blood loss, including severe arterial and venous bleeding, and could help save the lives of soldiers suffering from severe blood loss, according to company information.

"If awarded to STB, these funds, along with funds provided by our investors, will be used to accelerate the step up to cGMP manufacturing, and the necessary planning and preclinical work required to file for approval by the [Food and Drug Administration] to initiate human clinical trials," Ron Peele, STB president, said in a statement.

Osiris Therapeutics reported encouraging results from its phase 3 trial of Prochymal, its stem cell product for treating steroid-refractory acute graft vs. host disease.

Improvement was noted in patients treated with Prochymal who had skin, liver and gastrointestinal tract graft vs. host disease, the Columbia company said in a statement. The trial treated 244 patients from 72 bone-marrow transplant centers in the U.S., Canada, Europe and Australia.

Another new study showed that Prochymal had a response rate of 63 percent and improved survival when used as a rescue therapy in children suffering from severe treatment resistant graft vs. host disease.

Graft vs. host disease is a frequent complication of bone marrow transplantation and the leading cause of death in patients receiving allogeneic hematopoietic stem cell (bone marrow) transplants, according to company information. There is no approved treatment for the disease.

In yet another study, Osiris reported that Prochymal performed better than standard-of-care in a 53-patient double-blind, placebo controlled phase 2 trial studying the drug's effectiveness in treating heart attack patients.

Vanda Pharmaceuticals said the U.S. Patent and Trademark Office is allowing the Rockville company's patent application of a long-acting injectable formulation of its antipsychotic drug, Fanapt.

An oral form of Fanapt, which is indicated for treating schizophrenia, is marketed in the U.S. by Novartis Pharmaceuticals, under an agreement with Vanda in October.

Martek Bioscience, which develops nutritional products, said it renewed its lease on its 66,000-square-foot headquarters and expanded by 20,000 square feet in four buildings in the Columbia Business Center in Columbia.

Martek, with more than 525 employees, also has locations in Colorado, Kentucky and South Carolina.

BioElectronics wants to move forward by going after the back.

The Frederick company plans to introduce in the next few months a new design using elastic Velcro straps to attach its electronic healing patch to the body, with the back pain market a specific target.

"Considering that back pain is the single most often reported reason for physician office visits, and that the worldwide back pain market is valued in excess of many billions of dollars, we believe our opportunities with this new product are significant," CEO Andrew Whelan said in a statement.