Monday, June 28, 2010

ALS Drug Development Update

It can be difficult to keep abreast of all of the news that is relevant to research and treatment of ALS. Because of that, Prize4Life has designed tools to help keep researchers and the public up-to-date on what is happening in the research and drug development worlds (you can sign up for the free Weekly Digest and ALS Forum Newsletter if you want to get these alerts in your inbox). Below is a roundup of the most relevant ALS drug development news from the last six weeks. Check it out!

Invitrogen Contributes New Research for ALS
Invitrogen's stem cell business is participating in a new research program designed to find treatments for ALS. Life Technologies, Invitrogen's parent company, has partnered with the University of California San Diego and the Salk Institute for Biological Studies to research cell transplant therapies as a potential ALS treatment.

FoldRx Pharmaceuticals secures $29 million financing

FoldRx Pharmaceuticals has closed a $29 million financing deal to support a program in neurodegenerative disease. "With the addition of two top-tier venture firms to our already strong investor base, we believe we now have access to the resources necessary to commercialize tafamidis and build FoldRx into an independent, orphan-disease focused company," said Richard Labaudinière, Ph.D., President and CEO of FoldRx, in a FierceBiotech release. FoldRx Pharmaceuticals is focusing on disease-modifying small-molecule therapeutics to treat diseases of protein misfolding and aggregation, key pathological processes for many chronic neurodegenerative diseases, including ALS.

Aukera Therapeutics Named 2010 MIT Business Plan Contest Life Sciences Track Winner
Cambridge-based start-up biotech, Aukera Therapeutics, is developing Angiogenin, a novel protein therapy, to treat ALS. As the winner of MIT's Business Plan Contest, Aukera will receive seed funding and in-kind support from The Cambridge Innovation Center.

Stem Cell Therapeutics Wins US Patent
Stem Cell Therapeutics has received US patent rights for production of neural stem cells, a technology intensively investigated for its therapeutic use in ALS and other neurodegenerative diseases.

Amorfix Life Sciences and PREVENT Sign Licensing Agreement on ALS Vaccine
Amorfix has granted exclusive licensing rights to PREVENT for clinical development of its lead ALS vaccine for therapeutic purposes. Amorfix has retained the rights to develop the antibodies for diagnostic purposes.

Q Therapeutics’ Collaborators at Johns Hopkins Receive $1 Million to Study Glial Cells for Neurodegenerative Conditions
This infusion of funds is expected to accelerate characterization of Q Therapeutics’ proprietary glial cell-based therapeutic (Q-Cells®). This technology is being developed as a treatment for ALS and other neurodegenerative diseases.

ALS TDI: Changes in the Works
ALS TDI’s expertise using the SOD1 based model for target identification and drug discovery has made significant contributions to the ALS field. Recently, the non-profit ALS biotech has announced incorporation of a TDP-43 mouse model into its drug development program. This is a valuable effort that could bear dividends for the entire ALS research community.

Cytokinetics Announces Opening of a Phase IIa "Evidence of Effect" Clinical Trial of CK-2017357
Cytokinetics will begin a Phase II study of its fast skeletal muscle troponin activator, CK-2017357, for patients with peripheral artery disease and claudication. This announcement follows an ongoing Phase IIa trial in ALS patients. Cytokinetics believes this second study will further inform their understanding of possible therapeutic applications for this novel drug candidate for ALS and other skeletal muscle-associated diseases.

Neuralstem Updates Clinical Trial Progress
The first FDA approved stem cell clinical trial for ALS is reported to be on track. Neuralstem has completed safety evaluations of its first cohort of ALS patients and plans to move forward with the second cohort using a more extensive treatment regime.

Pfizer, Washington University announce collaboration
Pharma giant Pfizer grants Washington University unprecedented access to proprietary information regarding 500+ drug candidates. The collaboration is expected to uncover new uses for existing compounds.

Teva Reports Disappointing ALS Phase II Results for Talampanel
Teva’s talampanel is a no-go for ALS after discouraging Phase II data are reported. Despite demonstrating safety, talampanel did not reach its primary endpoint and will not be advanced as an ALS therapeutic.

New Virtual ALS-focused Company Aukera Therapeutics Wins MIT Award
Aukera, a finalist in the MIT $100K Business Plan Contest, wins the audience award for its development of a novel ALS therapeutic. The technology is based out of research from Harvard Medical School.

Repligen Files IND for Neurodegenerative Disease Treatment
Repligen has filed an investigational new drug application with the FDA for a Phase I study of its selective HDAC3 inhibitor, RG2833. While Repligen's initial drug development focus will be Friedreich's ataxia, RG2833 may have a broader therapeutic use for the treatment of ALS and other neurodegenerative diseases.

1 comment:

  1. My father died of ALS nearly 20 years ago. It's encouraging to read about new treatments and studies.