On January 18, 2018 Cytovation AS, a privately held biotech company developing CyPep-H1 for the treatment of cutaneous warts, reported that it has raised NOK 10m in a private funding round, completing the second stage of fundraising, having raised NOK 20m in June 2017 (Press release, Cytovation, JAN 18, 2018, View Source [SID1234561560]). Both financings were led by a group of private investors in Norway and the Company will now use these funds to advance lead product CyPep-H1 through the final stages of pre-clinical development and into and through a Phase I/IIa clinical trial in patients with warts caused by the human papilloma virus (HPV).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Cytovation is developing CyPep-H1 as a topical formulation of its proprietary lytic peptide, CyPep. CyPep is a highly-stable, purpose-engineered peptide, consisting of 27 amino acids, that selectively targets transformed or infected cells, destroying the cell membrane and killing the cells. The release of antigens from the lysed cells also triggers an immune response against the diseased cells, providing the possibility of long-term protection against warts.

The mode-of-action of CyPep presents a completely novel strategy to treat this common and potentially debilitating condition. Based on this mechanism, the Company believes that CyPep can have a number of applications in dermatological diseases, with CyPep-H1, for the treatment of cutaneous warts, being the first candidate under development. The Company is also investigating the potential of CyPep-derived peptides for the treatment of certain cancers.

Cytovation’s CEO, Kjell Inge Arnevig, commented on the financing: "The completion of this final tranche of fundraising now enables Cytovation to move through toxicology studies and formulation and into our planned Phase I/IIa clinical trial for CyPep-H1. We expect initial results from this study late 2018 and are confident that they will confirm the potential of this novel treatment approach for the many largely unsatisfied patients globally, who suffer from cutaneous warts."

On January 18, 2018 OncoSec Medical Incorporated ("OncoSec" or the "Company") (NASDAQ:ONCS), a company developing intratumoral cancer immunotherapies, reported preliminary clinical observations related to its pilot biomarker OMS-I140 clinical trial of ImmunoPulse IL-12 in patients with metastatic Triple Negative Breast Cancer (TNBC) (Press release, OncoSec Medical, JAN 18, 2018, View Source [SID1234523279]). The study is designed to assess whether a single cycle of ImmunoPulse IL-12 increases TNBC tumor immunogenicity by driving a pro-inflammatory cascade of events including activation of cytotoxic tumor-infiltrating lymphocytes (TILs).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

To date, five patients with TNBC have been treated with a single cycle of ImmunoPulse IL-12 (intratumoral pIL-12 [tavokinogene telseplasmid or "tavo"] with electroporation). Two of these five patients were subsequently treated with single agent nivolumab (Opdivo) – an anti-PD-1 checkpoint inhibitor treatment – as their immediate next therapy. Both of these patients, who were heavily pretreated metastatic TNBC patients with chemotherapy refractory disease, experienced robust objective responses in both ImmunoPulse IL-12 treated and untreated lesions. These clinical observations have prompted the Company to further commit to a more definitive evaluation of the combined therapies.

"Metastatic TNBC is a heterogeneous cancer with a poor prognosis where less than five percent of pre-treated patients achieve an objective response to PD-1/PD-L1 checkpoint treatments," explained Sharron Gargosky, Chief Clinical and Regulatory Officer of OncoSec. "The marked synergy shown in these patients strongly suggests that IL-12 may have primed the tumor microenvironment, impacting the clinical result. The combination of ImmunoPulse IL-12 and checkpoint inhibition represents a highly promising new therapeutic approach for TNBC and warrants a formal evaluation given the extremely low response rate in women who have failed multiple prior therapies."

Previous studies have demonstrated that breast cancer patients whose tumors are associated with markers of inflammation, such as the presence of TILs, achieve better clinical outcomes. In addition, the density of TILs is a key requirement for the anti-tumor activity of immune checkpoint inhibitors like anti-PD-1/PD-L1 antibodies. By augmenting the expansion of CD8+ tumor infilatrating T cells, ImmunoPulse IL-12 may be an ideal candidate to combine with checkpoint inhibitors, which has demonstrated low and variable activity as a monotherapy in TNBC.

Immunological examination of samples from all patients are currently being analyzed. These data, along with the full information regarding clinical observations and safety data, will be submitted for presentation at an upcoming medical meeting in 2018.

To learn more about the trial, visit www.oncosec.com. Additional details can also be found at www.clinicaltrials.gov via NCT02531425.

OPDIVO is a registered trademark of Bristol-Myers Squibb Company.

ImmunoPulse is a registered trademark of OncoSec Medical Incorporated, San Diego, CA, USA.

About Triple Negative Breast Cancer (TNBC)
Breast cancer cells that test negative for estrogen receptors (ER-), progesterone receptors (PR-), and HER2 (HER2-) means the cancer is triple negative.1 Approximately 15-20 percent of US breast cancer cases are triple negative breast cancer (TNBC),2 which disproportionately affects younger women as well as African-American women, followed by Hispanic women.3

TNBC remains a poor-prognosis breast cancer subtype, with limited treatment options for patients with advanced, recurrent disease. In the recurrent disease setting, chemotherapy remains the standard of care, and median survival is approximately 13 months from the time of disease recurrence.4 Emerging evidence shows immunotherapy options may play an important role in the treatment paradigm for TNBC. Preliminary data demonstrated the anti-PD-1 antibody, pembrolizumab, led to an objective response in approximately 18 percent of TNBC patients;5 and in the heavily pretreated population led to an objective overall response in approximately 4-8% of patients; 6 the anti-PD-L1 antibody, MPDL3280A, achieved an objective response in 33 percent of patients.7 There is increasing evidence that tumors need TILs for anti-PD-1/PD-L1 therapies to be most effective. Data also show TILs promote better responses to chemotherapy and improve clinical outcomes in breast cancer, including TNBC.8-13

On January 18, 2018 Amgen (NASDAQ: AMGN) and Allergan plc. (NYSE: AGN) reported that the European Commission (EC) has granted marketing authorization for MVASI (biosimilar bevacizumab) (Press release, Amgen, JAN 18, 2018, View Source;p=RssLanding&cat=news&id=2327358 [SID1234523289]). MVASI is the first biosimilar bevacizumab approved by the EC and is approved for the treatment of certain types of cancers, including in combination with fluoropyrimidine-based chemotherapy for metastatic carcinoma of the colon or rectum; in combination with paclitaxel for metastatic breast cancer; in combination with platinum-based chemotherapy for unresectable advanced, metastatic or recurrent non-squamous non-small cell lung cancer (NSCLC); in combination with erlotinib for unresectable advanced, metastatic or recurrent non-squamous NSCLC; in combination with interferon alfa-2a for advanced and/or metastatic renal cell cancer; in combination with carboplatin and paclitaxel, carboplatin and gemcitabine, and paclitaxel, topotecan, or pegylated liposomal doxorubicin for advanced, platinum-sensitive, or platinum-resistant recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer; and in combination with paclitaxel and cisplatin, or alternatively, paclitaxel and topotecan for persistent, recurrent, or metastatic carcinoma of the cervix.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The European Commission’s approval of MVASI marks a significant milestone for both Amgen and the oncology community, providing a biosimilar for a medicine which is used across multiple types of cancer," said Sean E. Harper, M.D., executive vice president of Research and Development at Amgen. "MVASI is the first targeted cancer biosimilar from Amgen’s portfolio approved in Europe, underscoring our commitment to delivering high-quality medicines that address some of the most serious illnesses."

Amgen and Allergan are committed to developing high-quality biosimilars with a robust analytic and clinical package. The EC approved MVASI based on a comprehensive data package that demonstrated MVASI and bevacizumab are highly similar, with no clinically meaningful differences in terms of the efficacy, safety and immunogenicity between the products. Clinical studies included results from a Phase 3 trial in patients with non-squamous NSCLC.

"MVASI is the first product from our collaboration with Amgen to receive marketing authorization from the European Commission, highlighting the success of our joint commitment to developing cancer biosimilars," said David Nicholson, chief research and development officer at Allergan. "We look forward to our continued work with Amgen and to providing important medicines to patients in the future."

Approval from the EC grants a centralized marketing authorization with unified labeling in the 28 countries that are members of the European Union (EU). Norway, Iceland and Liechtenstein, as members of the European Economic Area, will take corresponding decisions on the basis of the decision of the EC.

In September 2017, MVASI became the first anti-cancer biosimilar, as well as the first biosimilar bevacizumab, to be approved by the U.S. Food and Drug Administration (FDA). Amgen and Allergan are collaborating on the development and commercialization of four oncology biosimilars. Amgen has a total of 10 biosimilars in its portfolio, two of which have been approved by the EC.

About MVASI (biosimilar bevacizumab) in the EU

MVASI is a biosimilar to bevacizumab, a recombinant immunoglobulin G1 (IgG1) monoclonal antibody (mAb) that binds to vascular endothelial growth factor (VEGF) and inhibits the interaction of VEGF with its receptors, VEGF receptor-1 and VEGF receptor-2, thus inhibiting establishment of new blood vessels necessary for the maintenance and growth of solid tumors.

MVASI, in combination with fluoropyrimidine-based chemotherapy, is indicated for treatment of adult patients with metastatic carcinoma of the colon or rectum.

MVASI, in combination with paclitaxel, is indicated for first-line treatment of adult patients with metastatic breast cancer.

MVASI, in addition to platinum-based chemotherapy, is indicated for first-line treatment of adult patients with unresectable advanced, metastatic or recurrent NSCLC other than predominantly squamous cell histology.

MVASI, in combination with erlotinib, is indicated for first-line treatment of adult patients with unresectable advanced, metastatic or recurrent non-squamous NSCLC with Epidermal Growth Factor Receptor (EGFR) activating mutations.

MVASI, in combination with interferon alfa-2a, is indicated for first-line treatment of adult patients with advanced and/or metastatic renal cell cancer.

MVASI, in combination with carboplatin and paclitaxel, is indicated for the front-line treatment of adult patients with advanced (International Federation of Gynecology and Obstetrics (FIGO) stages IIIB, IIIC and IV) epithelial ovarian, fallopian tube, or primary peritoneal cancer.

MVASI, in combination with carboplatin and gemcitabine or in combination with carboplatin and paclitaxel, is indicated for treatment of adult patients with first recurrence of platinum-sensitive epithelial ovarian, fallopian tube or primary peritoneal cancer who have not received prior therapy with bevacizumab or other VEGF inhibitors or VEGF receptor-targeted agents.

MVASI, in combination with paclitaxel, topotecan, or pegylated liposomal doxorubicin, is indicated for the treatment of adult patients with platinum-resistant recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who received no more than two prior chemotherapy regimens and who have not received prior therapy with bevacizumab or other VEGF inhibitors or VEGF receptor-targeted agents.

MVASI, in combination with paclitaxel and cisplatin or, alternatively, paclitaxel and topotecan in patients who cannot receive platinum therapy, is indicated for the treatment of adult patients with persistent, recurrent, or metastatic carcinoma of the cervix.

MVASI EU Important Safety Information

The EU Summary of Product Characteristics for MVASI lists the following Special Warnings and Precautions: gastrointestinal (GI) perforations and fistulae, GI-vaginal fistulae in study GOG-0240, non-GI fistulae, wound healing complications, hypertension, posterior reversible encephalopathy syndrome (PRES), proteinuria, arterial thromboembolism, venous thromboembolism, haemorrhage, pulmonary haemorrhage/haemoptysis, congestive heart failure (CHF), neutropenia and infections, hypersensitivity reactions/infusion reactions, osteonecrosis of the jaw (ONJ), intravitreal use, eye disorders, systemic effects following intravitreal use, and ovarian failure/fertility.

About MVASI (bevacizumab-awwb) in the U.S.

MVASI is a biosimilar to bevacizumab, a recombinant IgG1 mAb that binds to VEGF and inhibits the interaction of VEGF with its receptors, VEGF receptor-1 and VEGF receptor-2, thus inhibiting establishment of new blood vessels necessary for the maintenance and growth of solid tumors.

MVASI is indicated for the treatment of metastatic colorectal cancer (mCRC), with intravenous 5-fluorouracil–based chemotherapy for first- or second-line treatment.

MVASI is indicated for the treatment of mCRC, with fluoropyrimidine- irinotecan- or fluoropyrimidine-oxaliplatin-based chemotherapy for second-line treatment in patients who have progressed on a first-line bevacizumab-containing regimen. MVASI is not indicated for adjuvant treatment of colon cancer.

MVASI is indicated for the treatment of non-squamous NSCLC, with carboplatin and paclitaxel for first line treatment of unresectable, locally advanced, recurrent or metastatic disease.

MVASI is indicated for the treatment of glioblastoma, as a single agent for adult patients with progressive disease following prior therapy.

The effectiveness of bevacizumab products in glioblastoma is based on an improvement in objective response rate. There are no data demonstrating an improvement in disease-related symptoms or increased survival with bevacizumab products.

MVASI is indicated for the treatment of metastatic renal cell carcinoma with interferon alfa.

MVASI is indicated for the treatment of cervical cancer, in combination with paclitaxel and cisplatin or paclitaxel and topotecan in persistent, recurrent, or metastatic disease.

MVASI is currently not available commercially. This is not an offer for sale. The following information is derived from the approved label in the U.S.

MVASI U.S. Important Safety Information

Boxed WARNINGS

Gastrointestinal (GI) Perforations

The incidence of gastrointestinal perforation, some fatal, in bevacizumab product-treated patients ranges from 0.3-3.2%. Fatal outcome was reported in <1% of bevacizumab-treated patients. Discontinue MVASI in patients with gastrointestinal perforation.

Surgery and Wound Healing Complications

The incidence of wound healing and surgical complications, including serious and fatal complications, is increased in bevacizumab product-treated patients. Discontinue MVASI in patients with wound dehiscence. The appropriate interval between termination of bevacizumab products and subsequent elective surgery required to reduce the risks of impaired wound healing/wound dehiscence has not been determined. Discontinue at least 28 days prior to elective surgery. Do not initiate MVASI for at least 28 days after surgery and until the surgical wound is fully healed.

Hemorrhage

Severe or fatal hemorrhage, including hemoptysis, gastrointestinal bleeding, central nervous system hemorrhage, epistaxis, and vaginal bleeding occur up to 5-fold more frequently in patients receiving bevacizumab products. Across indications, the incidence of grade ≥3 hemorrhagic events among patients receiving bevacizumab ranged from 0.4% to 6.9%. Do not administer MVASI to patients with serious hemorrhage or recent hemoptysis (≥1/2 tsp of red blood). Discontinue MVASI in patients with serious hemorrhage (ie, requiring medical intervention).

Additional serious adverse events

Additional serious and sometimes fatal adverse events with increased incidence in the bevacizumab product-treated arm vs control included
GI fistulae (up to 2% in metastatic colorectal cancer)
Non-GI fistulae (<1% in trials across various indications; 1.8% in a cervical cancer trial)
Arterial thromboembolic events (grade ≥3, 2.6%)
Proteinuria (nephrotic syndrome, <1%)
Additional serious adverse events with increased incidence in the bevacizumab product-treated arm vs control included
GI-vaginal fistulae occurred in 8.3% of patients in a cervical cancer trial
Venous thromboembolism (grade 3-4, up to 10.6%) in patients with persistent, recurrent, or metastatic cervical cancer treated with chemotherapy and bevacizumab product
Hypertension (grade 3-4, 5%-18%)
Posterior reversible encephalopathy syndrome (PRES) (<0.5%)
Infusion reactions with the first dose of bevacizumab product-treated patients were uncommon (<3%), and severe reactions occurred in 0.2% of patients
Inform females of reproductive potential of the risk of ovarian failure prior to starting treatment with MVASI
Pregnancy warning

Based on the mechanism of action and animal studies, bevacizumab products may cause fetal harm
Advise female patients that MVASI may cause fetal harm, and to inform their healthcare provider of a known or suspected pregnancy
Advise females of reproductive potential to use effective contraception during treatment with MVASI and for 6 months after the last dose of MVASI
Advise nursing women that breastfeeding is not recommended during treatment with MVASI
MVASI may impair fertility
Most Common Adverse Events

Across indications, the most common adverse reactions observed in bevacizumab product-treated patients at a rate of >10% and at least twice the control arm rate were: epistaxis, headache, hypertension, rhinitis, proteinuria, taste alteration, dry skin, rectal hemorrhage, lacrimation disorder, back pain, exfoliative dermatitis
Across all studies, bevacizumab product was discontinued in 8.4% to 21% of patients because of adverse reactions.
Please see full Prescribing Information, including Boxed WARNINGS, at www.Amgen.com.

About the Amgen and Allergan Collaboration

In December 2011, Amgen and Allergan plc. (then Watson Pharmaceuticals, Inc.) formed a collaboration to develop and commercialize, on a worldwide basis, four oncology antibody biosimilar medicines. This collaboration reflects the shared belief that the development and commercialization of biosimilar products will not follow a pure brand or generic model, and will require significant expertise, infrastructure, and investment to ensure safe, reliably supplied therapies for patients. Under the terms of the agreement, Amgen will assume primary responsibility for developing, manufacturing and initially commercializing the oncology antibody products.

About Amgen Biosimilars

Amgen Biosimilars is committed to building upon Amgen’s experience in the development and manufacturing of innovative human therapeutics to expand Amgen’s reach to patients with serious illnesses. Biosimilars will help to maintain Amgen’s commitment to connect patients with vital medicines, and Amgen is well positioned to leverage its more than 35 years of experience in biotechnology to create high quality biosimilars and reliably supply them to patients worldwide.

For more information, visit View Source and follow us on View Source

About Amgen’s Commitment to Oncology

Amgen Oncology is committed to helping patients take on some of the toughest cancers, such as those that have been resistant to drugs, those that progress rapidly through the body and those where limited treatment options exist. Amgen’s supportive care treatments help patients combat certain side effects of strong chemotherapy, and our targeted medicines and immunotherapies focus on more than a dozen different malignancies, ranging from blood cancers to solid tumors. With decades of experience providing therapies for cancer patients, Amgen continues to grow its portfolio of innovative and biosimilar oncology medicines.

On January 18, 2018 Cellular Biomedicine Group Inc. (NASDAQ: CBMG) (CBMG or the Company), a leading clinical-stage biopharmaceutical firm engaged in the development of immunotherapies for cancer, reported its plan to configure part of its facility in Shanghai with GE Healthcare’s FlexFactory platform, which is expected to be designed to speed up manufacturing timelines for its cell therapy clinical trials and commercial launch (Press release, Cellular Biomedicine Group, JAN 18, 2018, View Source [SID1234523290]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

There are more than 900 regenerative medicine trials underway globally, including trials in cell and gene therapy, a 19 percent increase since 2016.[1] Despite the increased number of precision medicine trials, gaps exist in how to manufacture these precise therapies to meet demand. Scalable integrated solutions to support the transition from clinical trials to commercialization have been limited. Many of the multiple cell therapy manufacturing process steps[2] remain largely unintegrated and manual, with open transfers between steps increasing contamination risk. To address these challenges and allow for reproducible manufacturing of cell therapies, GE Healthcare has developed FlexFactory for cell therapy, a scalable, semi-automated end-to-end platform.

"This is a productivity revolution in the CAR-T space – this new generation of semi-automated and standardized CAR-T manufacturing capabilities created by GE Healthcare and CBMG may allow cell therapy to provide an optimal platform and opportunity for general oncology patients. This long-term collaboration with GE could help us utilize digital technology, semi-automation and analytics, in an effort to reduce overall costs, and deliver treatments to patients more efficiently," said Tony (Bizuo) Liu, Chief Executive Officer, CBMG. GE Healthcare’s FlexFactory solution would support CBMG by providing process development and training services, cell processing equipment, semi-automation capabilities, and digital connectivity solutions – all of which support current good manufacturing practices (cGMP)-compliant manufacturing. CBMG plans to use its FlexFactory to speed up its timelines for commercializing its CAR T-cell therapies, targeting various blood and solid tumor cancers.

"With the rate in which cell therapies are moving through clinical trials, we understand how critentre for Commercialization of Regenerative Medicine (CCRM), a leader in developing and commercializing regenerative medicine technologies and cell and gene therapies, GE Healthcare expects to provide CBMG with process development services. The combined GE and CCRM process development team is comprised of 35 scientists and engineers with expertise in advanced therapeutic cell technologies, helping bridge the gap between research protocols and industrial manufacturing. GE and CCRM expects to support CBMG in increasing process efficiency by establishing a robust process development effort focused on simplifying, integrating and automating the manufacturing workflow.

"CCRM and GE Healthcare established the Centre for Advanced Therapeutic Cell Technologies, or CATCT, to industrialize cell manufacturing and accelerate the efforts of companies working with cell and gene therapies. The partnership between CBMG and GE is an exciting opportunity for the team at CCRM to demonstrate its process development skills and knowledge in overcoming cell therapy production challenges. We look forward to enabling CBMG in its efforts to commercialize its CAR T-cell therapy to treat patients with various blood and solid tumor cancers," said Michael May, President and CEO, CCRM.

On January 17, 2018 RXi Pharmaceuticals Corporation (NASDAQ: RXII) a clinical-stage company developing a new class of RNAi-based therapeutics reported that it will give an oral presentation highlighting the company’s proprietary therapeutic platform at the Immuno-Oncology Frontiers World conference (Press release, RXi Pharmaceuticals, JAN 17, 2018, View Source [SID1234523194]). This exclusive Immuno-oncology (I-O) partnering event provides direct access to decision makers and KOL’s working towards the next oncology blockbuster. Also, this is the only I-O event co-located with the leading Cell and Gene Therapy global meeting; providing access to the most commercially advanced technical expertise specifically for cellular immunotherapies. The conference will take place January 22-25, 2018 at the Hyatt Regency in Miami, Florida.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Title: Targeting Immune Checkpoints Using Self-Delivering RNAi (sd-rxRNA) Technology
Session: Stream 2- Emerging Science: Reprogramming the Tumor Microenvironment – Conquering Immune Suppressive Molecular Pathways
Date and Time: Tuesday, January 23, 2018, at 11:50 am ET
Presenter: James Cardia, Ph.D., Director of Business Development and Intellectual Property

The presentation will be available on the Company’s website approximately 1 hour after the presentation at www.rxipharma.com.

Logo – View Source

About RXi’s self-delivering RNAi (sd-rxRNA) technology platform

sd-rxRNA, RXi’s proprietary self-delivering RNAi platform, is a single chemically modified compound with delivery and therapeutic properties built directly into the compound itself. The compound is asymmetrical with a phosphorothioate backbone and contains chemical modifications that provide for efficient cellular uptake and gene silencing. These compounds are potent, stable and specific, and demonstrated to be safe and active in a clinical setting.

RXi’s novel sd-rxRNA technology differs from natural and most synthetic RNA interference (RNAi) molecules in that they are chemically modified to allow for efficient internalization of the compounds by cells and silencing of the targeted genes. Importantly, unlike other naked siRNA compounds, delivery of sd-rxRNAs are not limited to a specific cell type. For local delivery and ex vivo cell-based therapeutic applications, our compounds do not require delivery vehicles. This is a major advantage since delivery vehicles can have related toxicity that affects cell viability. sd-rxRNA has demonstrated nearly 100 percent transfection efficiency with high cell viability in numerous cell types.