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RXI Pharmaceuticals Corporation

19 Posts
ash_az
0
Op Leeuwenhoeck.com kwam ik Research Note van RXi Pharmaceuticals Corporation tegen (zie bijlage). Volgens Marcel Wijma zou USD 75 - 100 mio een goede waardering zijn. Dit is USD 4,50 tot 6,00 per aandeel. Als deze waardering maar half reeel is is het aandeel nog behoorlijk ondergewaardeerd. Gister mooie sprong van 13% (tussentijdse daguitschieter van zelfs 53%). Ik ben paar weken geleden ingestapt op USD 0,69. Huidige koers is USD 0.83. Voor mij een mooi casino aandeel dat dit jaar zou kunnen verdubbelen in waarde of de helft in waarde kan dalen. Echter volgens Research Note zijn er dit jaar verschillen milestones te verwachten die het aandeel behoorlijk van zijn plaats kan lanceren.

Zijn er meer mensen die dit aandeel volgen? Zo ja, wat zijn jullie verwachtingen?
Bijlage:
ash_az
0
Afgelopen half jaar heeft koers van RXi behoorlijke wat geleden aan gebrek aan (goed) nieuws. Af en toe wel een piek bij een nieuwsbericht, maar dat ebt dagen later weer weg. Bestuur is bezig om meer aandacht voor het fonds te creeren om de koers (blijvend) boven de USD 1,00 te brengen. Als de koers te lang onder de USD 1,00 blijft kan namelijk een ban van de NASDAQ volgen. Ik zelf heb het idee dat de koers nu wel uitgebodemd is en dat de geleidelijke weg omhoog is ingezet.

Verwachting is dat in 2021 een product de markt op gaat en tot die tijd zijn nog de nodige hobbels te nemen. Het lijkt er echter op dat door uitgifte van warrents en opties er voldoende cash zal zijn op product te ontwikkelen. Tot die tijd zijn er tevens nog voldoende (positieve) berichten te verwachten die de koers kunnen beinvloeden.

Hoewel er nog veel onzekerheid en dus risico is, verwacht ik dat dit een ideaal koopmoment is als je 5 jaar geduld hebt...
nightrider
0
RXII Security Details Other Company Securities
Share Structure
Market Value1 $13,492,755 a/o Oct 20, 2017
Authorized Shares Not Available
Outstanding Shares 23,247,338 a/o Aug 04, 2017
-Restricted Not Available
-Unrestricted Not Available
Held at DTC Not Available
Float Not Available
Par Value Not Available
Transfer Agent(s)
Shareholders
Shareholders of Record 106 a/o Mar 30, 2017
Security Notes
Capital Change=shs decreased by 1 for 30 split Ex-date=07/24/2013.
Capital Change=shs decreased by 1 for 10 split. Ex-date=04/18/2016.
Short Selling Data
Short Interest 0 (-100%)
Feb 14, 2014
Significant Failures to Deliver
nightrider
0
Is toch een riskant aandeel mi, het is zoals je schrijft, kan verdubbelen ( of meer), maar kan meer dan de helft dalen ook.
ash_az
0
Morgen na sluiten beurs in Amerika een update en cijfers Q3. Laatste is niet spannend (behalve stand cash en cash burn), maar mogelijk nieuws inzake voortgang ontwikkelingen. In november ook veel bijdrages op bio investor events. Is dat alleen om aandacht voor fonds te genereren, of ook om nieuw geld op te halen?
ash_az
0
MARLBOROUGH, Mass., Nov. 8, 2017 /PRNewswire/ -- RXi Pharmaceuticals Corporation (RXII), a clinical-stage RNAi company developing innovative therapeutics that address significant unmet medical needs, today reported its financial results for the third quarter ended September 30, 2017, and provided a business update.

Logo - mma.prnewswire.com/media/594748/RXi_P...

"RXi maintained a conservative spend rate in the third quarter in line with its projected budget as it continues to execute on its strategy with anticipated readouts this quarter from our dermatology and ophthalmology clinical programs. Data collection and analysis is ongoing, and the release of data is on track as previously reported," said Dr. Geert Cauwenbergh, President and CEO of RXi Pharmaceuticals. He further added that, "Our team has made notable progress with research efforts and data generation using our proprietary self-delivering RNAi technology platform (sd-rxRNA) for use in cancer therapeutics. sd-rxRNA compounds demonstrate high transfection efficiency with high cell viability in a number of immune cells. We believe that these assets have the potential to become a foundation for future growth opportunities, including several therapeutic approaches in the rapidly growing field of cell therapy for oncology. We look forward to successful and meaningful growth through our work as well as partnerships in this exciting and highly valuable medical field."

A live audio webcast will begin today at 4:30 p.m. ET. The webcast link is available under the "Investors – Events and Presentations" section of the Company's website, www.rxipharma.com. The event may also be accessed by dialing toll-free in the United States: +1 (844) 376-4678. International participants may access the event by dialing: +1 (209) 905-5958. An archive of the webcast will be available on the company's website approximately two hours after the presentation.

Select Third Quarter 2017 Financial Highlights

Cash Position

At September 30, 2017, the Company had cash of $5.4 million, compared with $12.9 million at December 31, 2016.

On August 8, 2017, the Company entered into a purchase agreement with Lincoln Park Capital Fund, LLC ("LPC"), pursuant to which the Company has the right to sell to LPC up to $15 million in shares of the Company's common stock over the 30-month term of the agreement. We expect to use proceeds from the purchase agreement for general corporate purposes, including but not limited to the advancement of our immunotherapy program, our clinical trials, and general and administrative expenses. As of September 30, 2017, there have been no purchases under the agreement with LPC.

Research and Development Expenses

Research and development expenses for the quarter ended September 30, 2017 were $1.5 million, as compared with $1.5 million for the quarter ended September 30, 2016. Research and development expenses were consistent quarter over quarter, with slight increases due to subject fees for the second cohort in the Samcyprone™ Phase 2 clinical trial and preclinical work in the Company's new immunotherapy program that was integrated into the Company with the acquisition of MirImmune in the first quarter of 2017, which were offset by a decrease in stock-based compensation expense.

General and Administrative Expenses

General and administrative expenses for the quarter ended September 30, 2017 were $1.0 million, as compared with $0.8 million for the quarter ended September 30, 2016. The increase in general and administrative expenses was primarily due to payroll-related expenses, including severance benefits, with the hire of the Company's former chief business officer in connection with the acquisition of MirImmune, resulting in a higher employee headcount as compared to the same period of the prior year, offset by a decrease in stock-based compensation expense.
ash_az
0
Net Loss

Net loss for the three months ended September 30, 2017 was $2.5 million, compared with $2.2 million for the three months ended September 30, 2016. The increase in net loss was primarily driven by the changes in general and administrative expenses, as discussed above.

Nasdaq Compliance

On August 2, 2017, the NASDAQ Stock Market provided written notice and granted the Company an additional 180 calendar days to regain compliance with the minimum bid price requirements set forth in the NASDAQ listing rules. As a result of this extension, the Company has until January 29, 2018 to regain compliance by maintaining a closing bid price of at least $1.00 for 10 consecutive business days. The NASDAQ written notice has no effect on the listing of the Company's common stock at this time.

Select Third Quarter 2017 and Recent Corporate Highlights

Select Business and Corporate Highlights

sd-rxRNA: Broadly applicable to numerous development areas providing continued and expanded growth

Grant Award – Development of self-delivering RNAi targeted to PTEN for treatment of spinal cord injury

The Company's proprietary self-delivering platform (sd-rxRNA) is broadly applicable to numerous therapeutic areas. For example, BioAxone Biosciences was recently awarded a grant from the National Institute of Neurological Disorders and Stroke (NINDS), part of the agency's SBIR Phase II funding program, to fund further development of BioAxone's preclinical candidate BA-434 in collaboration with RXi Pharmaceuticals. This two-year grant provides funding for further development of BA-434, a novel sd-rxRNA compound that targets PTEN for the treatment of spinal cord injury.

BioAxone has been awarded a total of $1,794,895 to fund the collaborative project over 24 months. For their contribution, RXi will receive approximately $129,000 in the first year with the potential to receive an additional $118,800 in the second year after achieving certain milestones.

Cell Therapy for Oncology

The Company's ongoing research programs with its sd-rxRNA platform have demonstrated robust cellular uptake in a number of immune cell types including, human T-cells, meso CAR-T, human NK, and dendritic cells. Our internal preclinical programs are focused on development of sd-rxRNA compounds for optimizing existing cell-based therapy treatment paradigms in oncology. In addition, the Company is actively seeking partnerships and collaborations with industry and academia to develop new technologies using engineered cells and our sd-rxRNA compounds. Our scientific team and advisors provide a strong foundation for the development of novel therapeutic treatment approaches using sd-rxRNA. This support positions RXi well with opportunities to provide meaningful growth for the Company.

Direct Therapeutic Use

RXI-109-1402 – Hypertrophic Scarring

The Company's ongoing Phase 2 clinical trial, RXI-109-1402, is being conducted to evaluate RXI-109, a sd-rxRNA compound targeting connective tissue growth factor (CTGF), a key regulator of scar formation. This open-label, multi-center study is designed to evaluate the effectiveness and safety of RXI-109 to reduce scar formation in healthy volunteers post scar revision surgery. The Company expects to share final study outcomes before the end of this year.

RXI-109-1501 – Retinal Scarring in Advanced Age-related Macular Degeneration (AMD)

Enrollment is complete in this Phase 1/2 study evaluating the safety and clinical activity of RXI-109 to prevent the progression of retinal scarring, a harmful component of numerous retinal diseases. This study is a multi-dose, dose escalation trial conducted in patients with advanced neovascular or wet age-related macular degeneration (AMD) where retinal scarring can result in continued vision loss. The primary endpoint for RXI-109-1501 is to evaluate the safety and tolerability of RXI-109. Additional endpoints will assess RXI-109's potential for clinical activity using numerous assessments to monitor ocular health and visual acuity. The Company expects to complete subject participation in the study by the end of 2017 and share top-line data in early 2018.
ash_az
0
RXI-SCP-1502 – Treatment of Cutaneous Warts

Samcyprone™, a proprietary topical formulation of the small molecule diphenylcyclopropenone (DPCP), is being evaluated in a Phase 2a clinical trial. RXI-SCP-1502 is a multi-center, multi-dose trial conducted in subjects with at least one cutaneous, plantar or periungual wart. The Company expects to share early read-outs before the end of this year.

Direct Consumer Care Use

RXI-231 - Consumer Care Products

The Company initiated a consumer testing program with RXI-231, a cosmetic ingredient based on sd-rxRNA that targets tyrosinase (TYR). The cosmetic product is a gel formulation designed to aid in the reduction of pigmentation and thereby improving skin appearance. The consumer testing program will evaluate the use and consumer acceptability of RXI-231.

There are three studies under this program. The first two studies in volunteers are performed to determine irritation and sensitization potential of the gel product containing RXI-231 when applied to the skin. A third study investigates the potential of the product to improve the appearance of skin pigmentation induced by UV exposure. The Company projects to report results before the end of this year.

Corporate Update

Management Team

On September 15, 2017, the Company announced the departure of Dr. Alexey Eliseev, former CEO of MirImmune Inc. Dr. Eliseev joined RXi on January 6, 2017 as Chief Business Officer of the Company in connection with its acquisition of MirImmune. MirImmune was a privately-held company developing cell-based therapeutics for cancer treatments based on a license to RXi's proprietary self-delivering RNAi technology. In his role as Chief Business Officer of RXi, Dr. Eliseev was responsible for the integration of this new therapeutic approach into the activities of RXi Pharmaceuticals, and for introducing the Company to several key industry and academic groups active in this emerging field.

Dr. James Cardia, RXi's current Director of Business Development and Intellectual Property now leads the management of the various ongoing activities in partnering and business development. During his tenure at RXi, Dr. Cardia's group was responsible for the discovery and optimization of "self-delivering" rxRNAs (sd-rxRNAs®) as well as the development and characterization of RXI-109, a promising anti-fibrotic agent currently in clinical trials for the treatment of both dermal and retinal scarring.

About RXi Pharmaceuticals Corporation

RXi Pharmaceuticals Corporation (RXII) is a clinical-stage company developing innovative therapeutics that address significant unmet medical needs. Building on the pioneering discovery of RNAi, scientists at RXi have harnessed the naturally occurring RNAi process which can be used to "silence" or down-regulate the expression of a specific gene that may be overexpressed in a disease condition. RXi developed a robust RNAi therapeutic platform including self-delivering RNA (sd-rxRNA®) compounds, that have the ability to selectively block the expression of any target in the genome, thus providing applicability to many therapeutic areas. Our current programs include dermatology, ophthalmology and cell-based cancer immunotherapy. RXi's extensive patent portfolio provides for multiple product and business development opportunities across a broad spectrum of therapeutic areas and we actively pursue research collaborations, partnering and out-licensing opportunities with academia and pharmaceutical companies. For additional information, visit the Company's website, www.rxipharma.com.
ash_az
0
RXI PHARMACEUTICALS ANNOUNCES POSITIVE RESULTS FROM PHASE 2 TRIAL WITH RXI-109 FOR DERMAL SCARRING
Dec 18, 2017
- RXI-109 is a self-delivering RNAi (sd-rxRNA) compound that targets connective tissue growth factor (CTGF), a key regulator of fibrosis and scar formation, and plays a key role in tissue regeneration and repair.
- Study successfully meets primary effectiveness objective with statistically significant outcomes for improved visual appearance for RXI-109 treated scar over control.
- RXI-109 meets secondary objective as it was shown to be safe and well tolerated.
- The exploratory endpoint for patient reported outcomes resulted in 88% of the patients indicating that RXI-109 treated scar looked better over control.
MARLBOROUGH, Mass., Dec. 18, 2017 /PRNewswire/ -- RXi Pharmaceuticals Corporation (NASDAQ: RXII) a clinical-stage company developing a new class of RNAi-based therapeutics today announced positive results with its lead clinical compound RXI-109 in a Phase 2 clinical trial. RXI-109-1402 is an open-label, multi-center, prospective, within-subject controlled study evaluating the effectiveness and safety of RXI-109 on the outcome of scar revision surgery for hypertrophic scars in healthy adults. The primary effectiveness objective was met as shown by a statistically significant improved visual appearance of revised scars after scar revision surgery and treatment with RXI-109 versus control, as assessed by the investigator. The full study results show that the product was safe and well tolerated for all dosage groups. Exploratory endpoint analysis furthermore shows that the cosmetic outcomes of RXI-109 treated scars were highly preferred over the untreated revised scars, by both investigators and patients.

mma.prnewswire.com/media/594748/RXi_P...

Joseph P. Hunstad, MD, FACSA, member of the Board of Directors for the American Society for Aesthetic Plastic Surgery and a Principal Investigator for the Company's Dermatology Clinical Program with RXI-109 stated that: "Today, scarring represents a high unmet medical need and based on our results; I believe that RXI-109 offers a clinically meaningful benefit for the treatment and prevention of hypertrophic scars following scar revision surgery. I look forward to providing further guidance in the development of this exciting new treatment option."

RXi's Chief Development Officer, Gerrit Dispersyn, Dr. Med. Sc., commented: "These results are very exciting, as they provide us with strong evidence that RXI-109, by preventing the overexpression of connective tissue growth factor, can result in a positive impact on the prevention of hypertrophic scars. The robustness of the data is shown by the fact that the effect of RXI-109 was significant by using both validated detailed scoring techniques, as well as simple qualitative assessments, and by the investigator driven data being further corroborated by an analysis of patient preferences. The fact that RXI-109 resulted in statistically significant improvements on scar outcomes at all follow-up time points for one of the dose groups, show that the effect of RXI-109 is both observed early on, and lasts at least throughout the entire follow-up time frame. In this study, we collected a lot of information that will allow us to perform additional analyses. Based on the current per protocol analysis, RXI-109 can be confidently moved to the next phase of clinical development."

Overall Efficacy Results

Using the Investigator Scar Assessment Scale, which enables a structured clinical evaluation of scar quality, the difference between RXI-109 treated scars and untreated (control) scars, was statistically significant at all follow-up time points, for one of the treatment arms. The improvement over untreated scars was a mean 6.88 point change at 3 months (p=0.028), a mean 8.00 point change (p=0.013) at 6 months and a mean 7.53 point change (p=0.013) at 9 months post revision surgery. The Investigator Scar Assessment Scale is a validated scoring tool which accounts for several parameters including surface area, pliability, relief, thickness, pigmentation and vascularity.
Using a qualitative measure of overall scar appearance (visual-analog-scale), the difference noted by the Investigator was also statistically significant for all follow-up time points, for the same treatment arm. The improvement over untreated scars was a mean 1.58 point change at 3 months (p=0.012), a mean 1.89 point change (p=0.004) at 6 months and a mean 1.55 point change (p=0.017) at 9 months post revision surgery.
As an exploratory endpoint, patient reported outcome for scar ranking was evaluated. Patients were asked to answer which scar segment (A or B) looks 'better' or if they thought they were 'not different'. For the treatment arm mentioned above, 88% of the patients and 86% of the investigators preferred the RXI-109 treated scar.
Safety and tolerability

Treatment with RXI-109 was well tolerated in treatment arms. There were no drug-related serious adverse events (SAEs) and most other treatment emergent adverse events (AEs) were those commonly found with intradermal injections, such as injection site pain (17.4%), and injection site erythema (11.8%).
Additional details from the Phase 2 study will be submitted for presentation at a future scientific conference.

Approximately 42 million surgeries occur in the United States alone each year.1 The American Society of Plastic Surgeons state that more than 170,000 patients receive scar revision therapy annually.2 Based on these populations, with costs primarily shouldered by patients, given the non-life-threatening nature of these indications, the resulting market potential could be worth as much as $1.5 billion. Given the current cost for cosmetic therapies such as Botox® and the fact that there are currently no approved treatment options for the prevention of hypertrophic scarring, premium pricing could be achieved with significant market penetration. Based on the study results, we are one step closer to having RXI-109 become the first solution for the prevention of hypertrophic scar formation in patients undergoing surgery or traumatic injury.

RXI-109 and RXi's sd-rxRNA technology platform are broadly protected in the United States and International regions. The Company's robust patent estate provides for multiple commercial and business development opportunities.

About Dr. Joseph Hunstad

Dr. Hunstad is one of the Principal Investigators for the Company's dermatology clinical program with RXI-109. His site has treated a large number of the subjects in the Company's clinical trials with RXI-109 to date. He received his Doctor of Medicine degree from the College of Human Medicine at Michigan State University and completed a General Surgery residency at Butterworth Hospital in Grand Rapids. He completed his Plastic Surgery residency at the Grand Rapids Area Medical Education Center, where he later served as Plastic Surgery Chief Resident for a year. He completed a Fellowship in Reconstructive Microsurgery at the MECOM Microsurgical Institute in Houston and is licensed to practice in North Carolina, Michigan, and Texas. Dr. Hunstad has been named one of the Best Plastic Surgeons in America by Castle Connolly, among numerous other awards and distinctions. Dr. Hunstad currently serves on the Board of Directors for the American Society for Aesthetic Plastic Surgery.

ash_az
0
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.

About RXi Pharmaceuticals

RXi Pharmaceuticals Corporation (NASDAQ: RXII) is a clinical-stage company developing innovative therapeutics that address significant unmet medical needs. Building on the pioneering discovery of RNAi, scientists at RXi have harnessed the naturally occurring RNAi process which can be used to "silence" or down-regulate the expression of a specific gene that may be overexpressed in a disease condition. RXi developed a robust RNAi therapeutic platform, including self-delivering RNA (sd-rxRNA®) compounds, that have the ability to highly selectively block the expression of any target in the genome, thus providing applicability to many therapeutic areas. Our current programs include dermatology, ophthalmology, and cell-based immunotherapy. RXi's extensive patent portfolio provides for multiple product and business development opportunities across a broad spectrum of therapeutic areas, and we actively pursue research collaborations, partnering and out-licensing opportunities with academia and pharmaceutical companies. For additional information, visit the Company's website, www.rxipharma.com.
ash_az
0
ash_az
0
Na de reverse stock split van 10:1 op 8 januari 2018 is de koers van RXi Pharmaceuticals gedaald van USD 4.51 tot USD 2.55 op 5 maart 2018. Vanaf maandag is er echter sprake van een opgaande lijn tot 4.30 op 9 maart (dinsdag USD 2.75-woensdag USD 3.06, donderdag USD 3.39 en vrijdag dus USD 4.30). Weet niet wat hier precies achter zit, maar met wellicht dat de presentatie van RXi zelf van 12 februari 2018 er iets mee te maken waarin ze zelf uitgaan van een return of investment van 3x tot 5x in 12 tot 18 maanden.

Daarnaast worden er ook een koersdoel van USD 20 afgegeven door Zacks (https://finance.yahoo.com/news/rxii-exploiting-natural-mechanism-rxi-160000783.html).

Ik blijf het aandeel volgen
De adviseur
0
Een mooi stuk over RXI Pharmaceuticals.

After Nasdaq woes, RXi pivots to promising cancer treatment field

In 1998, Craig C. Mello, a biologist at University of Massachusetts Medical School, and his colleague, Andrew Z. Fire, discovered a natural process known as RNA interference (or RNAi) that can be harnessed to make tiny, targeted changes in the way our genes transform our bodies. After winning the Nobel Prize for the discovery in 2006, Mello went on to cofound RXi Pharmaceuticals, where he now serves as chair of the Scientific Advisory Board.
In the two decades since the discovery, RXi has explored a variety of applications for RNAi, which can selectively prevent certain genetic information from being expressed in the body. Now, it's bringing the results of Mello's work into a burgeoning field of cancer research.
"We can, in essence, reprogram very elegantly a patient's own cells, or we can program engineered cells so that they are better armed—weaponized if you want—to attack cancer tissues more than the base-level immune system," said Gerrit Dispersyn, chief development officer at RXi.
A miracle treatment?
This type of process is part of immuno-oncology, a family of treatments that has brought new hope to people facing many types of cancer in recent years. In January, RXi announced that it was shifting its energy toward its work in oncology, and away from treatments for skin and eye diseases, where it had previously focused much of its energy.
The idea of using the immune system to treat cancers goes back more than a century, but it's made some huge leaps over the past decade. John Hallinan, chief business officer at the Massachusetts Biotechnology Council, said some immune system-based treatments have amazed doctors working in cancer treatment.
"The recovery is astounding, and some [patients] are relatively cancer-free after a regimen," he said. "It doesn't work for everybody, but it's very promising. Certain types of cancer where there's nothing else, it has proved to be nothing less than a godsend and a miracle."
Within the Massachusetts life sciences world, Hallinan said, immuno-oncology is now one of the top four most frequently researched areas.
Breakthroughs in the field didn't come out of nowhere, Hallinan said. As with many scientific advances, it depended on decades of painstaking research. But the payoff became clear over the past few years.
Immuno-oncology brings promise
For RXi, Dispersyn said, a clear signal about the practical viability of immuno-oncology research came last fall, when the FDA approved two other companies' cancer therapies using genetically engineered immune cells collected from patients. Given the promise of such methods, he said, regulators have begun greatly streamlining the process of getting them to market. Where a therapy might once have required studies on thousands of patients before it could move into clinical use, some can now reach the finish line with studies of less than 100 patients.
The federal "cancer moonshot," funded with $1.8 billion over seven years, has also accelerated immuno-oncology research, and increasing amounts of private money are going toward the work as well.
For RXi's business model, all this means that work on cancer has much greater promise in the short term than company leaders anticipated. Previously, they had anticipated that its first market-ready products would involve dermatology and ophthalmology since it already has related RNAi compounds in clinical development.
Now, though, the company—which was forced to use a reverse stock split earlier this year to boost its per-share price and regain compliance with Nasdaq Stock Market—sees oncology as its most likely path toward a viable therapy in the relatively near future. Dispersyn said it is looking for partners who can help continue the development of the dermatology and ophthalmology products.
That's not to say that the oncology applications of RXi's technology are brand new. Back in 2015, the company entered a licensing agreement with MirImmune LLC to develop cell-based cancer treatments. Then, in early 2017, it acquired the company, bringing the immunotherapy work in house.
The specific technology RXi brings to the table is a kind of RNAi compound that can deliver itself into a cell. Some other molecules that can be used to reprogram cells can't enter patients' cells by themselves because of their size or chemistry, so additional techniques, like lipid-based delivery vehicles or electric shocks, are needed to help administer the molecules. That, in turn, can make the process less efficient, or do damage to the cells, Dispersyn said.
Dispersyn said RXi's molecules are different. "We don't need any delivery mechanism or other delivery technique," he said. "These molecules get absorbed, get taken up by the cells without any other means."
That means they get into the cells with near 100-percent efficiency, and with no negative effects on the cells.
The process is relevant to a range of processes to treat diseases in which the body makes too much of a particular protein, or creates the wrong protein for a particular purpose.
Denmark testing underway
RXi is now working with several academic centers on practical applications for its technology in oncology. Inge Marie Svane of the Centre for Cancer Immunotherapy at Copenhagen University Hospital in Denmark said her organization is now testing the potential for RXi's technology in its work fighting melanoma and other cancers. Where some existing therapies inhibit proteins that cells have already produced, she said she hopes to be able to stop their production in the first place.
"It's a very new thing," she said.
"It's at a prior level, and maybe it will be more effective."
Svane said her center is beginning the testing on melanoma and will then move on to ovarian cancers and sarcoma—a type of cancer that affects connective tissues.
"I think this is a very promising principle, particularly that you can go down on the cellular level," she said.
Looking toward the future, Dispersyn said he hopes RXi will be able to get its first immuno-oncology compound into the clinical development phase in 12 to 18 months. At first, any uses of the technology will be in cases where more conventional cancer treatments have failed—something that's standard for any new therapy. Ultimately, though, the company hopes it will lead to more efficient first-line treatments that avoid the need for debilitating chemotherapy or radiation.

www.wbjournal.com/article/20180326/HE...
De adviseur
0
2018 Business Strategy
RXi will focus development initiatives on novel immuno-oncology therapeutics using its proprietary sd-rxRNA platform. Focusing the development portfolio on Immuno-oncology, with a near term focus on Adoptive Cell Transfer (ACT), will streamline and reduce our quarterly burn-rate for 2018. The Company is aiming to enter the clinic with at least one of our compounds in the next 18 months, targeting a large multi-billion dollar market. This will be achieved by:
-- Accelerating the development of RXI-762 and RXI-804, sd-rxRNA compounds targeting PD-1 and TIGIT respectively, for use in the treatment of solid tumors in the context of ACT. Finalize cGMP manufacturing of lead compound.
-- Expanding internal development efforts and external collaborations with various existing cell-based approaches, evaluating hematopoietic stem cells, NK cells, TILs, CAR T, TCR and engineered NK cells.
-- Developing immuno-oncology targets beyond checkpoint inhibition, i.e. cell differentiation.
-- Expanding our research efforts to further demonstrate that our technology is ideally suited to be combined with existing immune effector cell expansion/manufacturing.
Monetize Existing Dermatology and Ophthalmology Franchises:
Relating to the operational review, RXi intends to partner/out-license both its Dermatology and Ophthalmology Franchises. Successfully completing these transactions should provide non-dilutive funding for the Company's focused development path in Immuno-oncology.
Each of these Franchises is comprised of preclinical and clinical-stage assets broadly covered by a robust intellectual property estate. These assets include:

Dermatology
-- RXI-109: Phase 2 asset for dermal hypertrophic scarring with positive and statistically significant results addressing a USD 1-3B market.
-- RXI-231: Topical cosmetic ingredient in a proprietary formulation with positive results reducing a change of skin tone (pigmentation) triggered by UV (p< 0.04) addressing an estimated USD multi-billion dollar market.
-- RXI-185: Cosmetic ingredient that reduces collagenase in in vitro models. May be developed as a cosmetic to improve the appearance of aging skin.
-- Samcyprone(TM): Phase 2 small molecule topical immuno-therapy, with proven efficacy in cutaneous warts with an estimated market size USD 2-4B.
-- Access to the self-delivering platform for human therapeutic and diagnostic use in the field of dermatology, providing access to new compounds targeting proteins of relevance for the treatment of skin diseases.
The intellectual property estate for RXi's Dermatology Franchise is comprised of 14 patent families covering: RXI-109, RXI-231, RXI-185 and Samcyprone(TM). Importantly, this estate includes 18 granted patents broadly covering the sd-rxRNA platform, including RXI-109 in the US, Europe, Japan and China and Samcyprone(TM) in the US.

Ophthalmology
-- RXI-109: Phase 1/2 asset with proven safety, using intra-ocular injections, for wet AMD with estimated market size USD 3-5B. Readouts expected Q1 2018.
-- Preclinical data package supporting development of RXI-109 by intraocular injection or topical application to the eye for retinal or corneal scarring indications.
-- Patented set of sequences against a variety of relevant proteins for targeting ocular disorders. Also, sd-rxRNA compounds against targets which may be involved in retinoblastoma or retinal or corneal scarring.
-- Preclinical intra-ocular asset (sd-rxRNA targeting VEGF) with dose-dependent effects and tolerability demonstrated in a rodent model.
-- Access to the self-delivering platform for human therapeutic and diagnostic use in the field of ophthalmology, providing ready access to new compounds targeting proteins of relevance for the treatment of ophthalmic diseases.
RXi's Ophthalmology intellectual property estate includes 21 patent families. This estate includes 73 patents covering the composition and methods of use the sd-rxRNA platform and targets and sequences from the OPKO assets, including the use of RXI-109 for the treatment of ocular scarring in the US, Japan and China.
The webcast presentation took place on January 8, 2018, and the relating slides and audio may be found on the Company's website, www.rxipharma.com.
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 significant 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.
De adviseur
0
Inmiddels zijn we halverwege 2018, Geert Cauwenbergh heeft gezegd dat er in ieder geval interesse is in de Dermatology tak. Voor de Ophthalmology tak zal de interesse mede afhangen van de komende resultaten. Sinds 2013 werkt RXI hiervoor nauw samen met OPKO health. OPKO health heeft sinds 31 december 2017 een belang in RXI. Bij goede resultaten zullen zij ongetwijfeld interesse hebben.

De samenwerking met Iovance, absoluut geen kleine jongen met een marktwaarde van bijna 1,5 miljard is zeer positief. RXI komt terug op de radar, ze hebben teveel ijzers in het vuur en een te beloftevolle technologie in huis om ten onder te gaan. Daarbij heeft Geert Cauwenbergh de juiste contacten in de farmaceutische wereld, zeker niet onbelangrijk.

De risico/rendement verhouding is voor biotech begrippen zeker de moeite waard!

ash_az
0
Dat koersdoel is nog op basis van 2 4mio aandelen. Nu zijn het er 4.3mio... Evengoed nog voldoende koersdoelpotentieel over...
De adviseur
0
Hieronder de link naar een uitgebreid analisten rapport over RXI. Dateert uit mei 2017.
In de tussentijd heeft het bedrijf mooie vorderingen gemaakt. De analisten rechtvaardigen een marketcap van 144 miljoen dollar. Huidige marketcap: ruim 10 miljoen dollar.

Er zijn veel bedrijven met een enorm koerspotentieel, maar vaak is het risico daar ook naar. In het geval van RXI bestaat het risico (bij een marketcap van 10 miljoen) uit het op niets uitlopen van de ophthalmology, de dermatology én de immono-oncology tak.
In de dermatology tak is interesse getoond en heeft tevens al positieve fase 2 resultaten laten zien. Ook heeft het verkennende onderzoek naar de werking van TIL therapie (van samenwerkingspartner IOVANCE) en sd-rxRNA bemoedigende resultaten opgeleverd.

Oftewel ik schat de kans op een totale mislukking van al deze 3 componenten zéér klein. Daarbij is RNA op dit moment hot en is RXI de pionier op dit gebied. Wat mij betreft is dit een pareltje, op veilige afstand van de afgrond...
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