PR Newswire
10/05/04, 8:25a
(Copyright © 2004, PR Newswire)

CytRx Corporation has acquired all the clinical and pharmaceutical assets and related intellectual property of Hungary-based Biorex Research & Development RT. ("Biorex"), a privately held biotechnology company focused on the development of novel small molecules with broad therapeutic applications in neurology, diabetes, and cardiology. This acquisition dramatically accelerates CytRx's entry into clinical testing for amyotrophic lateral sclerosis (ALS; Lou Gehrig's Disease), with an anticipated initiation of a Phase II clinical trial for the drug arimoclomol during the second quarter of 2005. The acquisition of Biorex's assets also provides CytRx with a pipeline of at least two additional oral drug candidates at various stages of clinical development up to Phase II, a library of 500 small molecule drug candidates and an extensive intellectual property portfolio. In connection with the acquisition, CytRx paid a cash purchase price of US $3,000,000 and agreed to make milestone payments upon the occurrence of certain regulatory filings and approvals related to the acquired products.

Steven A. Kriegsman, CytRx's President and CEO, discussed the company's rationale for the transaction, "In the past several months, CytRx has refined its business strategy to focus on the development of therapeutics for ALS, obesity/type 2 diabetes, HIV and CMV. This acquisition significantly expands and accelerates our efforts in ALS by providing us with arimoclomol, a clinical-stage ALS drug with substantial revenue potential. We believe we can reach clinical proof-of-principle quickly with arimoclomol, since the drug has already demonstrated therapeutic efficacy in a pre-clinical model of ALS and was well absorbed and well tolerated in Phase I clinical trials. Due to the severity of ALS and the current lack of an effective treatment, we expect that arimoclomol will receive orphan drug status with the FDA. Therefore, a Phase II trial demonstrating efficacy could be sufficient to support registration for marketing. Additionally, the opportunity to obtain three small molecules in one transaction, each of which has substantial human safety data and therapeutic potential, further strengthens our product pipeline to complement our ongoing work in the development of RNAi therapeutics."

Dr. Robert H. Brown, Jr., Professor of Neurology at Harvard Medical School, founder of the Cecil B. Day Laboratory for Neuromuscular Research at Massachusetts General Hospital, and a recognized authority on ALS and member of CytRx's Scientific Advisory Board said, "The preliminary data makes arimoclomol an extremely important ALS drug candidate because of its demonstrated safety profile and the potential to treat both sporadic (spontaneous) and familial (inherited) forms of ALS."

Portfolio of Three Acquired Drug Candidates:

Biorex's small molecule drugs provide cellular protection from abnormal proteins by activating molecular "chaperone" proteins that can repair or degrade the damaged proteins that are believed to cause many diseases, including ALS.

Arimoclomol (ALS, Lou Gehrig's Disease)

Originally developed to treat diabetic complications, arimoclomol was recently discovered to significantly inhibit progression of ALS in an experimental animal model of the disease (Kierin et al., Nature Medicine, April 2004, Vol. 10(4), 402-5).

"The mechanism of these drugs accomplishes the same goal as RNAi, removal of toxic proteins, but does it at the protein level instead of at the RNA level," said Jack Barber, Ph.D., CytRx Senior Vice President of Drug Development.

The company's RNAi approach seeks to prevent the production of a toxic protein that causes disease in a subset of ALS patients that have inherited a muta|ion in the superoxide dismutase 1 (SOD1) gene. CytRx believes that arimoclomol would not only target the SOD1 protein, but also additional toxic proteins that may be involved in the more common ("sporadic") form of ALS as well. Arimoclomol would thus dramatically increase the number of treatable ALS patients, compared with the RNAi approach. "We plan to continue development of our RNAi approach to ALS concurrently with developing arimoclomol, but if both drugs prove to be effective, we would focus any immediate clinical development activities on the orally available arimoclomol, since it addresses a broader patient population," said Dr. Barber.