Thursday, January 26, 2012

Maryland Stem Cell Companies Advance Therapies for Diabetes, Depression

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Two Maryland biotechs announced inroads in their efforts to develop stem cell treatments for depression and diabetes.

Neuralstem has gotten the regulatory go-ahead to advance to phase 1b in its ongoing clinical trial of its stem cell treatment for major depressive disorder.

NSI-189 stimulates new neuron growth in the brain’s hippocampus, which may be involved in depression and other conditions, including Alzheimer's disease and post-traumatic stress disorder, according to the Rockville biotech.

"Loss of hippocampal volume is a known characteristic in depressed patients,” Karl Johe, chairman and chief scientific officer, said in a statement. “NSI-189 stimulates neurogenesis and increases hippocampal volume in healthy adult mice, at the same time reversing behavioral symptoms in mouse depression models, so it could address depression at the source."

The next phase — expected to run six months — will test the safety of increasing doses in 28 daily administrations in 24 depressed patients.

Meanwhile, Osiris Therapeutics of Columbia reported that preliminary results from a phase 2 clinical trial of its stem cell therapy in patients with newly diagnosed type 1 diabetes showed the treatment to be well tolerated.

However, the treatment did not slow the progression of the disease, even though there was a trend toward fewer hypoglycemic events, compared with patients receiving a placebo, the company said in a statement.

The two-year trial, under way for a year, is being conducted with the Juvenile Diabetes Research Foundation. It involves infusions of the treatment, Prochymal, which is formulated from the adult mesenchymal stem cells of unrelated adults. A total of 63 adult and child patients are in the study.

“Importantly, no patients experienced a reaction to the infusions despite the cells being unrelated to the recipient, unmatched, and used without immunosuppression,” Osiris reported.

"The ability to safely use stem cells from unrelated donors is an important finding of this study and provides new possibilities for further development of stem cell therapies for type 1 diabetes," Jay S. Skyler, professor of medicine and deputy director of the Diabetes Research Institute at the University of Miami Miller School of Medicine, said in the statement.

Osiris is developing the treatment under a strategic alliance with Genzyme.

In other Maryland bioscience industry news:

Kenneth Carter, a veteran of Maryland’s bioscience industry who founded Noble Life Sciences more than a year ago, has transitioned into leading one of the startups that the Gaithersburg company was designed to help foster.

Carter is now president and CEO of NexImmune of Gaithersburg, which initially is focusing on developing a treatment for melanoma. The company also named Jonathan Schneck, a professor of pathology, medicine and oncology at Johns Hopkins School of Medicine, chairman of its clinical and scientific advisory board.

The startup launched in the fall, said Carter, who will remain chairman of Noble. He’s been succeeded as Noble’s president by Alain Cappeluti, its former CFO.

“It’s a close-knit environment,” Carter said.

Noble was established with two objectives: to operate as a contract research organization for the pharmaceutical and biotech industries and also to provide startup services and an incubator environment for startups with their own products.

“We’ve gotten down the path on that now,” Carter said.

He declined to disclose the startup’s backers, but said financing details will be announced later.

“There’s no big venture capital financing put together,” said Carter, who for a decade was CEO at Avalon Pharmaceuticals, before it was acquired by Clinical Data.

NexImmune also announced that it has licensed from Johns Hopkins the Artificial IMmune nanotechnology developed by Schneck and a colleague, Mathias Oelke.

"The AIM technology provides an exciting leap in immuno-therapy for a variety of cancer and other diseases whereby artificial cells direct specific immune responses against a specific disease,” Carter said in a statement. “I am very pleased to lead the team that will commercialize this exciting new technology.”

"It is terrific to work with Ken and his team on the commercial transformation of the AIM technology to a product development platform,” Schneck said in the statement. “If the products to be developed by NexImmune are successful in human clinical trials, I believe the AIM technology has the potential to provide a major step forward for immuno-therapy for a variety of diseases."

RegeneRx Biopharmaceuticals is exploring a handful of partnerships for its dry-eye drug candidate, RGN-259, after the Rockville biotech reported in November that the treatment failed to meet two primary outcome measures in a 72-patient, phase 2 clinical trial that ran 30 days.

Still, the data showed that the candidate “had statistically significant benefits over placebo in certain signs and symptoms of dry eye syndrome,” according to a RegeneRx statement, which also referred to the results as “very encouraging.”

“In this exploratory study with only 35 subjects per group, RGN-259 was shown to have clinically significant effects on dry eye, albeit not in the chosen pre-determined primary endpoints,” RegeneRx said in a later statement released Thursday. “The significant protective effect that RGN-259 was shown to have on ocular discomfort and corneal staining in response to adverse stimuli indicate that changes in response to the [controlled adverse environment] challenge are effective new endpoints for future clinical trials.”

“As we reported prior to the announcement of the dry eye data, we had signed a number of confidentiality agreements with ophthalmic companies prior to release of data from the trial,” CEO J.J. Finkelstein said in the earlier statement. “Since then, we have been engaged in discussions with two of these companies and we have been contacted by two additional companies that are evaluating our data and with whom we are also holding discussions. We have told all of them that our intended goal is to close a deal by Jan. 31.”

The company will “also be taking additional cost-cutting steps to preserve cash during this period, which we will announce in greater detail after Jan. 1,” Finkelstein said.

Citing information from Global Data, an industry market research firm, RegeneRx has said that the worldwide annual market for dry eye disorders was about $1.9 billion in 2010 and is estimated to reach $2.8 billion by 2017.

Twenty healthy adult volunteers have received Profectus BioSciences’ experimental HIV vaccine in a phase 1 study to test its safety and immunogenicity.

The first studies showing the potential of recombinant vesicular stomatitis virus as an HIV vaccine vector were performed at Yale University more than a decade ago, according to a statement from the Baltimore biotech. The recombinant version used in the new study can replicate in human cells, but has been attenuated so it won’t cause illness in animals or humans.

The trial is to involve 60 adults. The vaccine was found to be safe and immunogenic in non-human primates.

The vaccine program has been supported by a $22.5 million contract from the National Institutes of Health.

VirxSys of Gaithersburg reported raising $1 million of a new $30 million mixed offering from 10 investors.

The biotech is working on an HIV vaccine and genetic treatments for cardiovascular disease and hemophilia.

Novavax reported that CPL Biologics, its joint venture in India with Cadila Pharmaceuticals, is ready to begin clinical studies of flu and rabies vaccines this year and next.

Rabies is a “significant” public health hazard in India, where 36 percent of the world's rabies deaths occur, according to a statement from the Rockville biotech.

CPL Biologics also has made “rapid” progress with the validation of its vaccine manufacturing plant in Dholka, India, Novavax said. The facility uses the single-use vaccine bioprocessing system that Novavax uses at its pilot plant in Rockville.

Vanda Pharmaceuticals, which plans to move to Washington, D.C., by April, will recognize expenses totaling $1 million to $1.5 million by ending its current lease at 9605 Medical Center Drive, Rockville, by June 30, 2013, according to a regulatory filing.

The company also reported that the Internal Revenue Service has approved a change in its accounting method related to startup expenditures that will be reflected in a fourth-quarter tax benefit of about $0.3 million, an increase in its net operating loss carry forwards of about $35.7 million, and research and development credits of about $3.4 million.

Arcion Therapeutics of Baltimore reported that it received notice of allowance for a U.S. patent for its high-strength gel formulation of topical lidocaine, ARC-2022.

Arcion is developing the product for pain associated with post-herpetic neuralgia and shingles.

"With ARC-2022 our goal is to deliver lidocaine to the skin without the requirement of an occlusive patch,” James N. Campbell, president and CEO, said in a statement. “ With topical delivery, systemic toxicities and [central nervous system] side effects can be avoided and the abnormal pain signals are suppressed at the point of origination in the skin."

Stem Cells are advancing everyday bringing new hope to so many...........MrCordBlood

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