Orphan Drug Status Granted for Treatment of Stargardt’s Macular Dystrophy, Advanced Cell Technology (OTC BB: ACTC.OB)
March 2nd, 2010
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The FDA has granted Orphan Drug designation for Advanced Cell Technology‘s (ACTC.OB) MA09-hRPE cells for use in the treatment of Stargardt’s Macular Dystrophy (SMD).
- As a result, ACTC.OB is eligible to receive a number of benefits, including tax credits, access to grant funding for clinical trials, accelerated FDA approval and allowance for marketing exclusivity after drug approval for a period of as long as 7 years.
Categories: Independent Equity Research
Merck KGaA agreed to buy Millipore (MIL), a supplier of drug-development equipment for biotechnology companies for about $6 B in cash, beating a rival offer from Thermo Fisher Scientific. BTIM’s ACTCellerate™ progenitor cell lines and ESpan™ cell growth media sell through MIL. Merck KGaA will pay $107 a share and is a Germany based company. The offer is 13 % more than Millipore’s closing price on 2/26/10 and 50 % higher. Millipore, based in Billerica, Massachusetts, put itself up for sale after getting an unsolicited takeover bid worth less than $95 a share from Thermo Fisher (Bloomberg).
GERN reported a Q4/09 net loss of $18.4 M, or $0.20 a share based on higher operating expenses. FY09’s loss was $70.4 M, or $0.80 a share. Q4/09 revenue was $605 K, with $1.7 M in revenue for FY09. Revenues in 2009 reflected royalty and license fee revenue and collaboration funding from various agreements. GERN in Q4 had operating expenses of $19 M, with R&D expenses of $15.3 M which increased primarily due to higher non-cash compensation expense for equity-based awards. Q4 G&A expenses were $3.6 M with FY09 operating expenses finishing at $72 M. Shares of Geron fell $0.25, or 4.3 % to $5.55 in Friday’s trading. Shares have traded between $3.67 and $9.24 over the last 52 weeks. Don’t count GERN down and out with $178.62 M in cash!
Programs to create a new center for the study of stem cells and to increase capacity to deal with global health issues were among 7 scientific initiatives announced 2/25/10 by NIH Director Francis S. Collins, MD, PhD.
Osiris Therapeutics is a stem cell therapeutic company focused on developing products in the inflammatory, orthopedic and cardiovascular areas. OSIR’s Q4/09 results reported achievement and data showing Prochymal achieved a 63% response rate when used as a rescue agent in children with end-stage GvHD with significant improvement in response rates over standard of care for both liver and gastrointestinal steroid-refractory GvHD – the two most deadly and difficult-to-treat forms of the disease. Graft-versus-host disease (GvHD) is a common complication of allogeneic bone marrow transplantation in which functional immune cells in the transplanted marrow recognize the recipient as “foreign” and mount an immunologic attack.
Aastrom reported results from interim analysis of the multi-center, randomized, double-blind, placebo-controlled U.S. Phase 2b clinical trial designated RESTORE-CLI. According to the interim analysis the safety profile was similar between the treatment and placebo arms. Based on a composite efficacy endpoint assessing time to treatment failure (including major amputations, wound size and gangrene), ASTMD’s autologous vascular repair cells (VRCs) were more effective than placebo (P<0.05). Other clinically meaningful endpoints (e.g., major amputation rate, complete wound closure) approached but did not reach statistical significance at interim analysis.
BCLI.OB announced that it has entered into agreements with 3 investors with each investing $500 K for a total of investment of $1.5 M with issuance to each of 3 investors, 2 M shares of stock based on the 30 day average share price as of 2/11/10 of $0.25 per share and 1 M warrants with an exercise price of $0.50 per share for a total issuance of 6 M shares and 3 M warrants. These investments provide BCLI.OB with the necessary funds to conduct its upcoming Phase I/II ALS clinical trial with a collaborative agreement with Hadassah Medical Center to conduct its ALS clinical trials at the Hadassah Ein Kerem Hospital, Israel.