ARWR has been up 6.66% on moderate volume.
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It announced today that Calando will be presenting interim data from the PI of its CALAA-01, in a poster, at the ASCO Meeting, on June 6.
The PI of CAJAA-01 involves Calando’s delivery system, RONDEL.
Dr. Antoni Ribas of UCLA’s Jonnson Comprehensive Cancer Center will discuss the data.
An abstract with the clinical study data,will also be presented at the Developmental Therapeutics - Experimental Therapeutics Session on Sunday, June 6. The abstract can be accessed at ASCO’s site at: abstract.asco.org.
"These data represent an important milestone for our Company and for the broader RNAi industry", said ARWR's CEO, Dr. Christopher Anzalon.
"It is the first proof-of-concept data demonstrating systemic siRNA delivery and gene silencing via RNAi in humans and we believe that RONDEL will ultimately enable multiple RNAi therapeutics."
Dr. Ribas said:
“Given the longstanding hurdles with effective systemic delivery of siRNA in humans, these exciting data represent a significant step for the field of RNAi."
"Detection of RONDEL siRNA nanoparticles inside cells biopsied from tumors demonstrates that RONDEL is capable of shuttling siRNA into tumors after being infused into the bloodstream of patients."
"We have also seen mRNA and protein knockdown as well as the presence of RONDEL inside tumors in a dose-dependent manner, both of which are excellent indications of effective systemic delivery."
"These findings suggest that the RONDEL delivery system’s approach could be expanded for use as a means for drug delivery and treatment for many cancer therapy targets that are currently considered untreatable.”
The PI CALAA-001 is being conducted at the UCLA Jonsson Comprehensive Cancer Center in L.A., and at START (South Texas Accelerated Research Therapeutics) in San Antonio.
It is an open-label, dose-escalating study of intravenously administered CALAA-01 in adults with solid tumors, who have failed other standard-of-care treatments.
In 15 patients treated with CALAA-01 to date, no dose limiting toxicities were observed.
One patient at the highest dose level had stable metastatic melanoma for four months, a change from prior course.
Biopsies from three patients showed CALAA-01 nanoparticles in tumors in amounts that correlate with dose levels. Additionally, a reduction of target messenger RNA and target protein was observed and at the highest dose, the targeted protein was knocked down with confirmation of mechanism by specific cleavage sequence (RACE-PCR).
The study’s authors conclude that systemic delivery of siRNA via targeted nanoparticles has been well tolerated and can induce specific, siRNA-mediated gene silencing.
The study is ongoing and patients continue to be enrolled at escalating doses. The Company expects to complete the Phase I study by the end of the 2010 calendar year.
Calando's RONDEL delivery system extends the reach of RNAi therapeutics by answering the new field’s most pressing need — an effective and safe systemic delivery method.
The RONDEL system takes advantage of molecular forces that generate self-assembly of an siRNA containing nanoparticle therapeutic.
Comprised of three components and siRNA, the system is engineered to form targeted, stabilized, siRNA-containing nanoparticles of less than 100nm in diameter that target specific tissues and fully protect the siRNA from degradation in serum.
Upon delivery to the target cell, the nanoparticle binds to membrane receptors on the cell surface and the siRNA-containing nanoparticle is taken into the cell by endocytosis. There, chemistry built into the system unpacks the siRNA from the delivery vehicle. The siRNA is deposited into the cytoplasm of the cell where it can access the cellular machinery for RNA interference.
Benefits of the RONDEL system include more effective delivery, modular design to allow easy exchange of the active siRNA ingredient and targeting agent, fewer immune reactions and increased stability.
RONDEL is also designed to work with human physiology and cell biology to overcome the extra- and intra-cellular barriers to siRNA delivery.
CALAA-01, is Calando's leading drug candidate.
It is a combination of RONDEL and a patented siRNA targeting the M2 subunit of ribonucleotide reductase, a clinically-validated cancer target.
Ribonucleotide reductase catalyzes the conversion of ribonucleosides to deoxyribonucleosides and is necessary for DNA synthesis and replication; it is a critical component in the proliferation of cancer cells.
Calando's siRNA and CALAA-01 have demonstrated potent anti-proliferative activity across multiple types of cancer cells. The targeting agent in CALAA-01 is transferrin, a blood plasma protein for iron delivery. Transferrin receptors have been shown to be up regulated in many types of cancer cells.
The CALAA-01 PI is an open-label, dose-escalating study of the safety of intravenous CALAA-01 in adults with solid tumors refractory to standard-of-care therapies.
Patients who satisfy the inclusion and exclusion criteria receive two, 21-day cycles of CALAA-01. A cycle consists of four infusions administered on days 1, 3, 8, and 10 followed by 11 days of rest. If safe, a second 21-day cycle is administered consisting of infusions on days 22, 24, 29, and 31 followed by 11 days of rest.
As mentioned, ARWR was able to trim its loss significantly for 4th time on a roll, in the 2ndQ.<g>
The ACTAY is $9 but that is only one "analyst" opinion.
I will keep my target at the $3 level. <g>
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Bernard |
